Conditions: Vasculitis
Allocation: RANDOMIZED | Intervention model: PARALLEL
Enrollment: 67
Healthy volunteers: no

Brief summary:
The aim of this trial is to optimize the treatment to induce remission for patients with non-life-threatening anti-neutrophil cytoplasmic antibody vasculitis (AAV). The intent is to reduce the toxicity of induction therapy by reducing the overall exposure to or eliminating entirely the use of systemic corticosteroids during the induction period with an inhibitor of the complement C5a receptor plus cyclophosphamide or rituximab.

Detailed description:
The primary safety objective of this study is to evaluate the safety and tolerability of CCX168 in subjects with AAV on background cyclophosphamide or rituximab treatment.

The primary efficacy objective is to evaluate the efficacy of CCX168 based on the Birmingham Vasculitis Activity Score (BVAS) version 3.

The secondary objectives of this study include assessment of the feasibility of reducing or eliminating the use of corticosteroids in the treatment of subjects with ANCA-associated vasculitis without the need for rescue corticosteroid measures and the effect of CCX168 on several disease parameters.

Study acquired by Amgen and all disclosures were done by previous sponsor ChemoCentryx.

Arms:
arm 1: Placebo plus a full dose of oral glucocorticoids for steps 1 and 2 of the study arm 2: 30 mg Active study medication, plus either two-thirds reduced dose of oral glucocorticoids for step 1 of the study, or no oral glucocorticoids for step 2 of the study

Interventions: intervention 1: Placebo intervention 2: CCX168
Intervention descriptions: intervention 1: BID for 84 days intervention 2: BID for 84 days

Protocol document:
Prot_000.pdf: 
C he m o Ce ntr y x, I n c.  
C O N FI D E N TI A L  
Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8 
 
 A me n d me nt 4. 0 
                                                                   Pa ge 1 of 1 1 3                                   1 8 Se pte m ber 2 0 1 5 
 
P R O T O C O L 
Pr ot oc ol A me n d me nt 4 . 0 
TI T L E P A G E  
St u d y Title:  A Ra n d o mize d, D o u ble- Bli n d, Place b o- C o ntr olle d, P hase 2 St u d y 
t o E val uate t he Safet y a n d Efficac y of C C X 1 6 8 i n S u bjects wit h 
A nti - Ne utr o p hil C yt o plas mic A nti b o d y ( A N C A)-Ass ociate d 
Vasc ulitis o n Bac k gr o u n d C ycl o p h os p ha mi de or Rit u xi ma b  
Treat me nt  
Pr ot oc ol N u m ber:  C L 0 0 2 _ 1 6 8 
I n vesti g ati o n al Pr o d uct: C o m ple me nt 5a Rece pt or A nta g o nist C C X 1 6 8 
I n dic ati o n: A N C A -Ass ociate d Vasc ulitis  
S p o ns or:  C he m o Ce ntr y x, I n c.  
De vel o p me nt P h ase:  2  
E U D R A C T n u m ber  2 0 1 1- 0 0 1 2 2 2- 1 5 
S p o ns or's Res p o nsi ble 
Me dic al Officer:  
 
C he m o Ce ntr y x, I n c.  
 
S p o ns or Si g n at or y: 
 
A p pr o v al D ate: 
3 1 M arc h 2 0 1 1 — FI N A L  
2 3 A pril 2 0 1 2 — Pr ot oc ol A me n d me nt 1. 0  
1 4 M arc h 2 0 1 3 — Pr ot oc ol A me n d me nt 2. 0  
3 0 M a y  2 0 1 4 —Pr ot o c ol A me n d me nt 3. 0  
1 8 Se pte m ber 2 0 1 5 — Pr ot oc ol A me n d me nt 4. 0 
  
 
C o nfi de nti al  
T he i nf or mati o n c o ntai ne d herei n is t he pr o pert y of t he S p o ns or a n d ma y n ot be re pr o d u ce d, 
p u blis he d, or discl ose d t o ot hers wit h o ut writte n a ut h orizati o n of t he S p o ns or. 
T his st u d y will be c o n d u cte d acc or di n g t o t he pri n ci ples of G o o d Cli nical Practice as descri be d 
i n I nter nati o nal C o nfer e n ce o n Har m o nizati o n g ui deli nes, i ncl u di n g t he arc hi vi n g of ess e ntial 
d oc u me nts. 
P P D
P P D
P P D


C he m o Ce ntr y x, I n c.  
C O N FI D E N TI A L  
Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8 
 
 A me n d me nt 4. 0 
                                                                   Pa ge 2 of 1 1 3                                   1 8 Se pte m ber 2 0 1 5 
 
I N V E S TI G A T O R SI G N A T O R Y P A G E 
Pr ot oc ol N u m ber:  C L 0 0 2 _ 1 6 8 
Pr ot oc ol Title:  A R a n d o mize d, D o u ble- Bli n d, Place b o- C o ntr olle d, P hase 2 St u d y t o E v al uate 
t he Safet y a n d Efficac y of C C X 1 6 8 i n S u bjects wit h A nti-Ne utr o p hil C yt o plas mic A nti b o d y 
( A N C A)-Ass ociate d V as c ulitis o n Bac k gr o u n d C ycl o p h os p h a mi de or Rit u xi ma b  Treat me nt  
I a gree: 
•  t o ass u me res p o nsi bilit y f or t he pr o per c o n d u ct of t he st u d y at t his site. 
•  t o c o n d uct t he st u d y i n c o m plia nce wit h t his pr ot oc ol, a n y f ut ure a me n d m e nts, a n d wit h 
a n y ot h er st u d y c o n d uct pr oce d ures pr o vi de d b y C he m o Ce ntr y x, I n c.  
•  n ot t o i m ple me nt a n y de viati o ns fr o m or c ha n ges t o t he pr ot oc ol wit h o ut a gree me nt fr o m 
t he s p o ns or a n d pri or r e vie w a n d writte n a p pr o val fr o m t he I nstit uti o nal Re vie w B oar d 
(I R B)/ Et hics C o m mittee ( E C), e x ce pt w here necessar y t o eli mi nate a n i m me diate hazar d 
t o t he s u bjects, or f or a d mi nistrati ve as pects of t h e st u d y ( w here per mitte d b y all 
a p plica ble r e g ulat or y r e q uire me nts).  
•  t hat I a m t h or o u g hl y fa miliar wit h t he a p pr o priate use of t he i n vesti gati o nal dr u g(s), as 
descri be d i n t his pr ot oc ol, a n d a n y ot her i nf or mati o n pr o vi de d b y t he s p o ns or i ncl u di n g, 
b ut n ot li mite d t o t he f oll o wi n g: t he c urre nt v ersi o n of t he Cli nical I n v esti gat or’s 
Br oc h ure pr e par e d b y C h e m o Ce ntr y x, I nc. a n d a p pr o ve d pr o d uct la b el, if a p plica ble. 
•  t hat I a m a ware of a n d will c o m pl y wit h c urre nt I C H/ F D A g o o d cli nical practices 
g ui deli nes ( G C P) a n d all re g ulat or y re q uire me nts. 
•  t o e ns ure t hat all pers o ns assisti n g me wit h t he st u d y are a d e q uatel y i nf or me d a b o ut t he 
i n vesti gati o nal dr u g(s) a n d t heir st u d y-relate d d uti es a n d f u ncti o n as d escri be d i n t he 
pr ot oc ol. 
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _         
 _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ 
Pri nci pal I n v esti gat or     
  
  
  
  
  
  
 
Date  
 
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ 
Pri nte d Na me  
A d dress * _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ 
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ 
P h o ne N u m ber * _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ 
* If t he a d dr ess or p h o ne n u m ber nee ds t o be c ha n ge d d uri n g t he c o urs e of t he st u d y, t his will be 
d o ne b y t h e I n vesti gat or, wit h writte n n otificati o n t o t he S p o ns or, a n d will n ot re q uire (a) 
pr ot oc ol a me n d me nt(s). 


C he m o Ce ntr y x, I n c.  
C O N FI D E N TI A L  
Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8 
 
 A me n d me nt 4. 0 
                                                                   Pa ge 3 of 1 1 3                                   1 8 Se pte m ber 2 0 1 5 
 
S P O N S O R C O N T A C T I N F O R M A TI O N  
Pr ot oc ol N u m ber:  C L 0 0 2 _ 1 6 8 
Pr ot oc ol Title:  A R a n d o mize d, D o u ble- Bli n d, Place b o- C o ntr olle d, P hase 2 St u d y t o E v al uate 
t he Safet y a n d Efficac y of C C X 1 68 i n S u bjects wit h A nti- Ne utr o p hil C yt o plas mic A nti b o d y 
( A N C A)-Ass ociate d V as c ulitis o n Bac k gr o u n d C ycl o p h os p h a mi de or Rit u xi ma b  Treat me nt  
 
Me dical Offi cer    
 
C hief Me dic al Officer  
C he m o Ce ntr y x, I n c.  
 
 
 
 
 
Cli nical      
  
 
 
O perati o ns   
  
 
Cli nical C o ns ulta nt   
Ma na ger     
  
 
C he m o Ce ntr y x, I n c.  
 
 
 
 
 
P P D
P P D
P P D
P P D




C he m o Ce ntr y x, I n c.  
C O N FI D E N TI A L  
Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8 
 
 A me n d me nt 4. 0 
                                                                   Pa ge 5 of 1 1 3                                   1 8 Se pte m ber 2 0 1 5 
 
P R O T O C O L A M E N D M E N T 4 . 0: S U M M A R Y O F C H A N GE S 
1.  T he statistical met h o d ol o g y secti o n i n t he S y n o psis  a n d secti o n 7. 1 3. 3 was re vise d t o i n dicate 
t hat t he differ e nce i n pr o p orti o ns of s u bjects ac hie vi n g t he cate g orical e n d p oi nts will be use d 
i nstead of t he o d ds rati o. T he rati o nale f or t his c ha n ge is t o f oll o w t he prece de nce set b y a 
pri or cli nical trial c o m pari n g rit u xi ma b wit h c ycl o p h os p ha mi de i n A A V (St o ne et al,  2 0 1 0). 
A mi x e d m o del f or re peate d meas ures ( M M R M) a nal ysis f or c o nti n u o us varia bles was als o 
a d de d. 
2.  T he St u d y Sc he ma  was c orrect e d t o i n dicate t hat t he Ste p 3 e nr oll me nt tar get was 3 6 
s u bjects, n ot 1 8 0. 
3.  T he S y n o psis , Ti me a n d E ve nts ta ble , a n d secti o ns 1. 2. 3, 1. 3, 5. 9 , 6. 1 4 , a n d 7. 2. 3 were 
re vise d t o c o ns oli date pr e vi o us c o u ntr y-s pecific a me n d me nts. 
4.  A d diti o n of a st o p pi n g criteri o n f or C C X 1 6 8/ place b o d osi n g t o Secti o n 7. 2. 3 of t he pr ot oc ol 
re gar di n g W B C a n d n e utr o p hil c o u nts: If a s u bject de vel o ps gra d e t w o or w orse le u k o pe nia 
( W B C < 3 x 1 09/ L) O R a n a bs ol ute ne utr o p hil c o u nt < 1 x 1 09/ L, d osi n g wit h C C X 1 6 8 or 
place b o m ust be cease d i n t his s u bject. St u d y dr u g ma y b e res u me d o nl y if W B C a n d 
a bs ol ute ne utr o p hil c o u nt b ot h e x cee d t he l o wer li mit of t he res pecti ve n or mal ra n ge, t he 
I n v esti gat or dee ms r es u m pti o n t o be a p pr o priate, a n d t he W B C a n d A N C are m o nit ore d 
cl osel y t hereafter. T his r ec o m me n dati o n was bas e d o n fi n di n gs of le u k o p e nia/ ne utr o pe nia i n 
t w o cases i n a n ot her st u d y i n patie nts wit h A N C A- ass ociate d v asc ulitis ( st u d y C L 0 0 3 _ 1 6 8). 


C he m o Ce ntr y x, I n c.  
C O N FI D E N TI A L  
Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8 
 
 A me n d me nt 4. 0 
                                                                   Pa ge 6 of 1 1 3                                   1 8 Se pte m ber 2 0 1 5 
 
T A B L E O F C O N T E N T S  
I N V E S TI G A T O R SI G N A T O R Y P A G E ......................................................................................... 2 
S P O N S O R C O N T A C T I N F O R M A TI O N  ....................................................................................... 3 
S P O N S O R SI G N A T U R E F O R A P P R O V A L  ................................................................................. 4 
P R O T O C O L A M E N D M E N T 3. 0: S U M M A R Y O F C H A N G E S  ................................................... 5 
T A B L E O F C O N T E N T S  ................................................................................................................. 6 
LI S T O F T A B L E S  ......................................................................................................................... 1 0 
S T U D Y S Y N O P SI S  ...................................................................................................................... 1 1 
S T U D Y S C H E M A ......................................................................................................................... 2 7 
TI M E A N D E V E N T S T A B L E ...................................................................................................... 2 8  
LI S T O F A B B R E VI A TI O N S A N D A C R O N Y M S  ...................................................................... 3 1 
1.  
I N T R O D U C TI O N ...................................................................................................... 3 3 
1. 1.  
Bac k gr o u n d ................................................................................................................. 3 3  
1. 2.  
St u d y Dr u g D e vel o p me nt ........................................................................................... 3 4  
1. 2. 1.  
N o n -Cli nical P har mac ol o g y  ....................................................................................... 3 4 
1. 2. 1. 1.  
I n-Vitr o Effi cac y a n d Sel ecti vit y f or C 5a R  ................................................................ 3 4 
1. 2. 1. 2.  
Efficac y M o dels  .......................................................................................................... 3 4 
1. 2. 2.  
N o n - Cli nical Safet y a n d T o xic ol o g y .......................................................................... 3 5 
1. 2. 3.  
N o n -Cli nical A D M E  ................................................................................................... 3 8 
1. 3.  
Pri or H u ma n E x perie nce ............................................................................................. 4 0  
1. 4.  
Rati o nale f or t he St u d y ............................................................................................... 4 5  
2.  
O BJ E C TI V E S  ............................................................................................................. 4 6 
2. 1.  
Pri mar y O bj ecti ve  ....................................................................................................... 4 6 
2. 2.  
Sec o n dar y O bj ecti ves  ................................................................................................. 4 6 
3.  
S T U D Y D E SI G N  ....................................................................................................... 4 7 
4.  
S T U D Y P O P U L A TI O N  ............................................................................................. 5 0 
4. 1.  
Size of t he P o p ulati o n ................................................................................................. 5 0  
4. 2.  
I n cl usi o n Criteria ........................................................................................................ 5 0 
4. 3.  
E x cl usi o n Criteria  ....................................................................................................... 5 1 
4. 4.  
Re m o val of S u bjects fr o m T hera p y of Assess me nt  .................................................... 5 2 


C he m o Ce ntr y x, I n c.  
C O N FI D E N TI A L  
Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8 
 
 A me n d me nt 4. 0 
                                                                   Pa ge 7 of 1 1 3                                   1 8 Se pte m ber 2 0 1 5 
 
5.  
S T U D Y M E DI C A TI O N/ T R E A T M E N T  ................................................................... 5 2 
5. 1.  
Pr o d uct C haracteris tics ............................................................................................... 5 2 
5. 2.  
Ra n d o mizati o n a n d Met h o d of Treat me nt Assi g n me nt .............................................. 5 3  
5. 3.  
D oses a n d Re gi me ns ................................................................................................... 5 3  
5. 4.  
Rati o nale f or D ose Sel ecti o n  ...................................................................................... 5 7 
5. 5.  
Dr u g S u p pl y ................................................................................................................ 5 7  
5. 5. 1.  
Pac ka gi n g a n d La b eli n g  .............................................................................................. 5 7 
5. 5. 2.  
St ora ge  ........................................................................................................................ 5 8 
5. 6.  
Bli n di n g ...................................................................................................................... 5 8  
5. 7.  
Dr u g Acc o u nta bilit y ................................................................................................... 5 9  
5. 8.  
Treat me nt C o m plia nce  ................................................................................................ 5 9 
5. 9.  
C o nc o mita nt Me dicati o ns a n d Restricti o ns ................................................................ 5 9  
5. 1 0.  
Cr iteria f or Resc u e C ortic oster oi d Use ....................................................................... 6 0 
6.  
S T U D Y P R O C E D U R E S  ............................................................................................ 6 1 
6. 1.  
Scree ni n g a n d E nr oll me nt  ........................................................................................... 6 1 
6. 2.  
St u d y Da y 1  ................................................................................................................ 6 2 
6. 3.  
St u d y Da y 2  ................................................................................................................ 6 4 
6. 4.  
St u d y Da y 8  ................................................................................................................ 6 5 
6. 5.  
St u d y Da y 1 5 .............................................................................................................. 6 6  
6. 6.  
St u d y Da y 2 2 .............................................................................................................. 6 7  
6. 7.  
St u d y Da y 2 9 .............................................................................................................. 6 8  
6. 8.  
St u d y Da y 4 3 .............................................................................................................. 7 0  
6. 9.  
St u d y Da y 5 7 .............................................................................................................. 7 1  
6. 1 0.  
St u d y Da y 7 1 .............................................................................................................. 7 2  
6. 1 1.  
St u d y Da y 8 5 .............................................................................................................. 7 3  
6. 1 2.  
St u d y Da y 9 9 .............................................................................................................. 7 4  
6. 1 3.  
St u d y Da y 1 1 3 ............................................................................................................ 7 5  
6. 1 4.  
St u d y Da y 1 4 1 ............................................................................................................ 7 6  
6. 1 5.  
St u d y Da y 1 6 9 ............................................................................................................ 7 7  
7.  
S T U D Y A S S E S S M E N T S  .......................................................................................... 7 8 
7. 1.  
Efficac y Assess me nts  ................................................................................................. 7 8 


C he m o Ce ntr y x, I n c.  
C O N FI D E N TI A L  
Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8 
 
 A me n d me nt 4. 0 
                                                                   Pa ge 8 of 1 1 3                                   1 8 Se pte m ber 2 0 1 5 
 
7. 1. 1.  
C ortic oster oi d Resc ue Us e  .......................................................................................... 7 8 
7. 1. 2.  
Esti mate d Gl o mer ular Filtrati o n Rate  ......................................................................... 7 8 
7. 1. 3.  
Uri nar y Assess me nts  .................................................................................................. 7 8 
7. 1. 4.  
Bir mi n g h a m Vasc ulitis Acti vit y Sc ore ( B V A S) ......................................................... 7 9 
7. 1. 5.  
Vasc ulitis Da ma ge I n d e x ( V DI)  ................................................................................. 7 9 
7. 1. 6.  
A nti -Ne utr o p hil C yt o plas mic A nti b o d y ( A N C A) Assess me nts  ................................. 7 9 
7. 1. 7.  
Ser u m C -Reacti ve Pr otei n ( C R P)  ............................................................................... 7 9 
7. 1. 8.  
Uri nar y M C P -1 Assess m e nts  ...................................................................................... 7 9 
7. 1. 9.  
Healt h -Relate d Q ualit y of Life Assess me nts  .............................................................. 7 9 
7. 1. 1 0.  
Hist ol o g y ..................................................................................................................... 7 9 
7. 2.  
Safet y Assess me nts  ..................................................................................................... 8 0 
7. 2. 1.  
P h ysical E x a mi nati o ns a n d Vital Si g ns ...................................................................... 8 0  
7. 2. 2.  
Cli nical Safet y La b orat or y Assess me nts  .................................................................... 8 0 
7. 2. 3.  
Re p orti n g of A d vers e E v e nts ...................................................................................... 8 1  
7. 2. 4.  
Re p orti n g of Seri o us A d verse E ve nts ......................................................................... 8 3  
7. 3.  
P har mac o ki netic Assess me nts  .................................................................................... 8 4 
7. 4.  
P har mac o d y na mic Ass ess me nts  ................................................................................. 8 4 
7. 5.  
St u d y C o m pleti o n a n d Wit h dra wal ............................................................................ 8 4  
7. 6.  
Data M o nit ori n g C o m mittee  ....................................................................................... 8 5 
7. 7.  
Statistical Met h o ds  ...................................................................................................... 8 6 
7. 8.  
S u bject P o p ulati o ns .................................................................................................... 8 6  
7. 9.  
Safet y E n d p oi nts  ......................................................................................................... 8 6 
7. 1 0.  
Efficac y E n d p oi nts ...................................................................................................... 8 6  
7. 1 1.  
P har mac o ki netic E n d p oi nts ........................................................................................ 8 8  
7. 1 2.  
P har mac o d y na mic E n d p oi nts ..................................................................................... 8 8  
7. 1 3.  
Statistical A nal ysis Met h o d ol o g y  ............................................................................... 8 8 
7. 1 3. 1.  
Baseli ne C haracteristics  .............................................................................................. 8 8 
7. 1 3. 2.  
Safet y A nal yses  .......................................................................................................... 8 9 
7. 1 3. 3.  
Efficac y A nal yses  ....................................................................................................... 8 9 
7. 1 3. 4.  
C o variates  ................................................................................................................... 9 0 
7. 1 3. 5.  
P har mac o ki netic A n al ysis  .......................................................................................... 9 1 


C he m o Ce ntr y x, I n c.  
C O N FI D E N TI A L  
Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8 
 
 A me n d me nt 4. 0 
                                                                   Pa ge 9 of 1 1 3                                   1 8 Se pte m ber 2 0 1 5 
 
7. 1 4.  
Sa m ple Size J ustificati o n  ............................................................................................ 9 1 
7. 1 5.  
I nteri m A nalysis  .......................................................................................................... 9 2 
7. 1 6.  
Pr ot oc ol De viati o ns .................................................................................................... 9 2  
8.  
S T U D Y C O M P L E TI O N A N D  T E R MI N A TI O N  ...................................................... 9 2 
8. 1.  
St u d y C o m pleti o n  ....................................................................................................... 9 2 
8. 2.  
St u d y Ter mi nati o n  ...................................................................................................... 9 2 
9.  
R E G U L A T O R Y A N D A D MI N I S T R A TI V E R E Q UI R E M E N T S ............................ 9 3 
9. 1.  
I n v esti gat or Res p o nsi bilities ....................................................................................... 9 3 
9. 2.  
I nstit uti o nal Re vie w B oar d or Et hics C o m mittee ....................................................... 9 3 
9. 3.  
I nf or me d C o nse nt ....................................................................................................... 9 3 
9. 4.  
Pr ot oc ol M o dificati o ns ............................................................................................... 9 4  
9. 5.  
Re g ulat or y D oc u me ntati o n  ......................................................................................... 9 4 
9. 6.  
S u bject I d e ntificati o n Re gister  ................................................................................... 9 4 
9. 7.  
Rec or d Rete nti o n  ........................................................................................................ 9 4 
9. 8.  
Case Re p ort F or m C o m pleti o n  ................................................................................... 9 5 
9. 9.  
M o nit ori n g .................................................................................................................. 9 5  
9. 1 0.  
O n -site A u dits ............................................................................................................. 9 5 
9. 1 1.  
Use of I nf or mati o n a n d P u blicati o n ............................................................................ 9 5  
1 0.  
R E F E R E N C E S  ........................................................................................................... 9 6 
1 1.  
A P P E N DI C E S  ............................................................................................................ 9 7 
1 1. 1.  
State me nt of O bli gati o ns of S p o ns or, M o nit or, a n d Cli nical I n vesti gat or ................. 9 7  
1 1. 2.  
I nf or me d C o nse nt F or m ............................................................................................ 1 0 0 
1 1. 3.  
Bir mi n g h a m Vasc ulitis Ac ti vit y Sc ore ( B V A S) Versi o n 3 ...................................... 1 0 1 
1 1. 4.  
Vasc ulitis Da ma ge I n d e x  .......................................................................................... 1 0 3 
1 1. 5.  
Pre d nis o ne D ose Sc he d ul e ........................................................................................ 1 0 4  
1 1. 6.  
C ycl o p h os p ha mi de a n d Azat hi o pri ne D osi n g  .......................................................... 1 0 7 
1 1. 7.  
Rit u xi ma b D osi n g  ..................................................................................................... 1 0 9 
1 1. 8.  
A d verse E v e nts Re p orte d wit h C ycl o p h os p ha mi de ................................................. 1 1 0  
1 1. 9.  
A d verse E v e nts Re p orte d wit h C ortic oster oi d Use .................................................. 1 1 1  
1 1. 1 0.  
A d verse E v e nts Re p orte d w it h Rit u xi ma b Use ........................................................ 1 1 3 
1 1. 1 1.  
A d verse E v e nts Re p orte d wit h Azat hi o pri ne Use .................................................... 1 1 3  


C he m o Ce ntr y x, I n c.  
C O N FI D E N TI A L  
Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8 
 
 A me n d me nt 4. 0 
                                                                   Pa ge 1 0 of 1 1 3                                   1 8 Se pte m ber 2 0 1 5 
 
 
LI S T O F T A B L E S  
Ta ble  1: 
O ver vie w of T o xic ol o g y St u dies Perf or me d wit h C C X 1 6 8 ....................................... 3 6  
Ta ble  2: 
O ver vie w of Safet y P har mac ol o g y St u dies Perf or me d wit h C C X 1 6 8 ....................... 3 7  
T a ble 3: 
P har mac o ki netic Par a meters of C C X 1 6 8 i n C D- 1 Mice, S pra g ue- Da wl e y 
Rats, Bea gle D o gs, a n d C y n o m ol g us M o n k e ys after A d mi nistrati o n of a 
Si n gle Or al Ga va ge or a n I ntra ve n o us D os e of C C X 1 6 8 ........................................... 3 9  
Ta ble  4: 
S u m mar y of Treat me nt -e mer ge nt A d verse E ve nts O bser ve d i n t he Si n gle-
d ose Peri o d, C L 0 0 1 _ 1 6 8 ............................................................................................ 4 1  
Ta ble  5: 
S u m mar y of K e y P har mac o ki netic Res ults fr o m t he Si n gle - d os e Peri o d, 
C L 0 0 1 _ 1 6 8 ................................................................................................................. 4 2  
Ta ble  6: 
S u m mar y of all t he treat me nt -e mer ge nt a d vers e e ve nts o bser ve d i n t he 
m ulti ple - d ose peri o d of t he st u d y, C L 0 0 1 _ 1 6 8 .......................................................... 4 2 
Ta ble  7: 
S u m mar y of P har mac o ki netic Res ults ( mea n, S D) fr o m t he M ulti- d ose 
Peri o d, O nce D ail y D ose Gr o u ps, C L 0 0 1 _ 1 6 8 .......................................................... 4 4  
Ta ble  8: 
S u m mar y of P har mac o ki netic Res ults ( mea n, S D) fr o m t he M ulti- d os e 
Peri o d, T wice D ail y D os e Gr o u ps, C L 0 0 1 _ 1 6 8 ......................................................... 4 4  
Ta ble  9: 
Perce nt Pr o ba bilit y of C o nti n uati o n t o Ne xt Ste p ...................................................... 9 1  
 


C he m o Ce ntr y x, I n c.  
C O N FI D E N TI A L  
Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8 
 
 A me n d me nt 4. 0 
                                                                   Pa ge 1 1 of 1 1 3                                   1 8 Se pte m ber 2 0 1 5 
 
S T U D Y S Y N O P SI S  
N a me of S p o ns or 
C he m o Ce ntr y x, I n c.  
N a me of Acti ve I n gre die nt  
C C X 1 6 8  
St u d y n u m ber:  
C L 0 0 2 _ 1 6 8 
Title  
A Ra n d o mize d, D o u ble- Bli n d, Place b o- C o ntr olle d, P hase 2 St u d y t o E val uate t he Safet y a n d 
Efficac y of C C X 1 6 8 i n S u bjects wit h A nti- Ne utr o p hil C yt o plas mic A nti b o d y ( A N C A) -
Ass ociate d Vas c ulitis o n Bac k gr o u n d C ycl o p h os p ha mi de or Rit u xi ma b  Treat me nt  
I n vesti g at ors 
Se veral  
St u d y ce nters  
M ulti -ce nter  
St u d y peri o d  
6 0 m o nt hs 
P h ase of de vel o p me nt  
P hase 2  
Ai m  
T he ai m of t his trial is t o o pti mize t he treat me nt t o i n d uce re missi o n f or pati e nts wit h a nt i-
ne utr o p hil c yt o plas mic a nti b o d y vasc ulitis ( A A V). T he i nte nt is t o re d uce t he t o xicit y of 
i n d ucti o n t hera p y b y re d uci n g t he o v erall e x p os ure t o or eli mi nati n g e ntirel y t he use of s yste mic 
c ortic oster oi ds d uri n g t he i n d ucti o n peri o d wit h a n i n hi bit or of t he c o m ple me nt C 5a rece pt or 
pl us c ycl o p h os p h a mi de or rit u xi ma b . 
O bjecti ves  
T he pri mar y safet y  o bjecti ve of t his st u d y is t o e v al uate t he safet y a n d t olera bilit y of C C X 1 6 8 
i n s u bjects wit h A AV o n bac k gr o u n d c ycl o p h os p h a mi de or rit u xi ma b  treat me nt. 
T he pri mar y efficac y o bj ecti ve is t o e val uate t h e efficac y of C C X 1 6 8 base d o n t he Bir mi n g ha m 
Vasc ulitis Acti vit y Sc ore  ( B V A S) v ersi o n 3. 
T he sec o n d ar y o bjecti ves of t his st u d y i ncl u de: 
1.  E val uati o n of t he efficac y of C C X 1 6 8 c o m p are d t o sta n dar d of care ( S O C) base d o n 
c ha n ges i n r e nal diseas e acti vit y para meters:  
a. e G F R ( M D R D s er u m cr eati ni ne e q uati o n); 
b.  He mat uria ( ce ntr al la b or at or y micr osc o pic c o u nt of uri nar y R B Cs); a n d  
c. A l b u mi n uria (first m or ni n g uri nar y al b u mi n:creati ni ne rati o); 
2.  Assess me nt of c ha n ges i n re nal i nfla m ma t or y acti vit y base d o n uri nar y m o n oc yt e 
c he m oattracta nt pr otei n -1 ( M C P -1):creati ni ne rati o a n d ser u m C -reacti ve pr otei n 
c o nce ntr ati o n wit h C C X 1 6 8 c o m pare d t o S O C ; 


C he m o Ce ntr y x, I n c.  
C O N FI D E N TI A L  
Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8 
 
 A me n d me nt 4. 0 
                                                                   Pa ge 1 2 of 1 1 3                                   1 8 Se pte m ber 2 0 1 5 
 
3.  Assess me nt of t he feasi bilit y of re d u ci n g or eli mi nati n g t he use of c ortic ost er oi ds i n t he 
treat me nt of s u bj ects wit h A N C A-ass ociat e d vasc ulitis ( A A V) wit h o ut t he nee d f or resc u e 
c ortic oster oi d meas ures  wit h C C X 1 6 8 c o m pare d t o S O C ; 
4.  Assess me nt of healt h -rel ate d q ualit y- of-life c ha n ges base d o n S h ort F or m- 3 6 versi o n 2 ( S F-
3 6 v 2) a n d E ur o Q O L- 5 D- 5 L ( E Q - 5 D- 5 L) wit h C C X 1 6 8 c o m pare d t o S O C ; 
5.  Assess me nt of c ha n ges i n Vasc ulitis Da ma ge I n d e x ( V DI) wit h C C X 1 6 8 c o m pare d t o S O C ; 
6.  Assess me nt of c ha n ges i n A N C A (a nti - P R 3 a n d a nti- M P O) wit h C C X 1 6 8 c o m pare d t o 
S O C ; 
7.  Assess me nt of c ha n ges i n p har mac o d y n a mics mar kers i n plas ma a n d uri ne wit h C C X 1 6 8 
c o m pare d t o S O C ; 
8.  E val uati o n of t he p har m ac o ki netic pr ofile of C C X 1 6 8 i n s u bjects wit h A A V. 
Met h o d ol o g y 
A A V sta n dar d t h era p y i n cl u des c ycl o p h os p ha mi d e or rit u xi ma b a n d oral c ortic oster oi ds, 
ta pere d o v er a p eri o d of ti me. Se vere disease warra nts a d diti o n of I V c ortic oster oi ds a n d/ or 
plas ma e x c ha n ge. Base d o n c o m pelli n g res ults fr o m precli nical st u dies i n a m o use m o del of 
A N C A -ass ociat e d re n al vasc ulitis,  C C X 1 6 8 has t he p ote ntial t o be a c ortic oster oi d s pari n g or 
c ortic oster oi d r e place m e nt t hera p y f or t his diseas e. He nce, t he cli nical h y p ot hesis of t he trial is 
t o test t he feasi bilit y of usi n g C C X 1 6 8 as a c ortic oster oi d s pari n g or re place me nt t hera p y d uri n g 
i n d ucti o n of re missi o n. T his h y p ot hesis will be teste d i n a t hree-ste p ma nner i n t his ra n d o mize d, 
d o u ble- bli n d, place b o- c o ntr olle d, P hase 2 cli nical trial i n u p t o a p pr o xi matel y 6 0 s u bjects wit h 
ne w or rela pse d A A V. A n e xter nal data m o nit ori n g c o m mittee ( D M C) will re vie w safet y data, 
i ncl u di n g res c ue I V or or al c ortic oster oi d use o v er t he c o urse of t h e st u d y a n d a d vise t he 
S p o ns or re gar di n g pr o gressi o n fr o m eac h ste p t o t he ne xt i n t he st u d y. Ste ps 1 a n d 2 of t he 
st u d y ha v e bee n c o m plete d. 
S T E P 1  
I n Ste p 1 of t h e trial, u p t o a p pr o xi matel y 1 2 s u bjects will be stratifie d t o o ne of t w o strata, 
eit her ne wl y dia g n os e d A A R V or rel a pse d A A R V, a n d t he n ra n d o mize d t o C C X 1 6 8 or place b o 
(i n a 2: 1 rati o). A t w o-t hir ds re d uce d d ose of oral c ortic oster oi ds will be gi ve n t o s u bjects 
ra n d o mize d t o C C X 1 6 8 a n d a f ull d ose of oral c ortic oster oi ds t o s u bjects ra n d o mize d t o 
place b o. All 1 2 s u bjects will recei ve I V c ycl o p h os p ha mi de treat me nt, w hi c h is part of sta n dar d 
t hera p y f or A A R V. If n ecessar y, resc u e I V met h yl pre d nis ol o ne or oral resc ue ster oi ds after Da y 
1 s h o ul d be gi ve n t o s u bj ects wit h w orse n i n g disease.  
S T E P 2   
Ste p 2 will be o pe ne d f or e nr oll me nt if b ot h of t he f oll o wi n g criteria are met: 
1.  N ot m ore t ha n 1 S U S A R m ost li kel y relate d t o C C X 1 6 8, as assesse d b y t h e D M C, is 
o bser ve d i n s u bjects r ecei vi n g C C X 1 6 8 i n Ste p 1; 
2.  A A R V disease acti vit y is c o ntr olle d i n t he maj orit y of s u bjects ( > 5 0 %) recei vi n g C C X 1 6 8 
i n Ste p 1, wit h o ut t he nee d f or I V or oral resc ue c ortic oster oi d t hera p y, as assesse d b y t he 


C he m o Ce ntr y x, I n c.  
C O N FI D E N TI A L  
Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8 
 
 A me n d me nt 4. 0 
                                                                   Pa ge 1 3 of 1 1 3                                   1 8 Se pte m ber 2 0 1 5 
 
D M C.    
I n Ste p 2 of t h e trial, u p t o a p pr o xi matel y 1 2 s u bjects will be stratifie d t o o ne of t w o strata, 
eit her ne wl y dia g n os e d A A R V or rel a pse d A A R V, a n d t he n ra n d o mize d t o C C X 1 6 8 or place b o 
(i n a 2: 1 rati o). Or al c orti c oster oi ds will n ot be gi v e n t o s u bjects ra n d o mize d t o C C X 1 6 8, b ut a 
f ull d ose of oral c ortic oster oi ds will be gi v e n t o s u bjects ra n d o mize d t o place b o. All 1 2 s u bjects 
will recei ve I V c ycl o p h os p ha mi de treat me nt. If necessar y, resc u e I V or oral met h yl pre d nis ol o ne 
s h o ul d be gi ve n t o s u bjects wit h w orse ni n g diseas e.  
S T E P 3  
I n Ste p 3, a p pr o xi matel y 3 6 s u bjects will be stratifie d pri or t o ra n do mizati o n base d o n t he 
f oll o wi n g stratificati o n fact ors: 
1.  Eit her ne wl y dia g n ose d A A V or rela pse d A A V; 
2.  Eit her M P O or P R 3 A N C A p ositi vit y; 
3.  Will recei ve eit her c ycl o p h os p ha mi de or rit u xi ma b as part of sta n d ar d of care treat me nt.  
F oll o wi n g str atificati o n, s u bje cts will be ra n d o mize d 1: 1: 1 t o o ne of t hree gr o u ps: 
Gr o u p A: C C X 1 6 8 pl us c ycl o p h os p h a mi de/rit u xi ma b pl us n o oral c ortic oster oi ds;  
Gr o u p B: Place b o pl us c ycl o p h os p h a mi de/rit u xi ma b pl us a f ull starti n g d ose of oral 
c ortic oster oi ds; 
Gr o u p C: C C X 1 6 8 pl us c ycl o p h os p h a mi de/rit u xi ma b pl us a t w o-t hir ds re d uce d starti n g d ose of 
oral c ortic oster oi ds;  
If necessar y, resc u e I V m et h yl pr e d nis ol o ne or oral resc ue ster oi ds s h o ul d be gi v e n t o s u bjects 
wit h w orse ni n g dis ease. 
Ste p 3 will be o pe ne d f or e nr oll me nt if b ot h of t he f oll o wi n g criteria are met: 
1.  N ot m ore t ha n 1 S U S A R m ost li kel y relate d t o C C X 1 6 8, as assesse d b y t h e D M C, is 
o bser ve d i n s u bjects r ecei vi n g C C X 1 6 8 i n Ste p 2; 
2.  A A V disease acti vit y is c o ntr olle d i n t he maj orit y of s u bjects ( > 5 0 %) recei vi n g C C X 1 6 8 i n 
Ste p 2, wit h o ut t he nee d f or I V or oral resc ue c ortic oster oi d t hera p y. 
T he C C X 1 6 8/ place b o d osi n g peri o d f or all t hree ste ps of t he st u d y is 8 4 da ys, f oll o we d b y a n 
8 4 -da y f oll o w - u p peri o d.  
I n or der t o pr ot ect t he bli n di n g, a d o u ble-d u m m y desi g n will be utilize d:  
S T E P 1  
I n Ste p 1, t he st u d y dr u g a n d ot her me dicati o n f or re nal vas c ulitis will be ta ke n as f oll o ws b y 
st u d y s u bjects: 
•  Gr o u p A ( 3 0 m g C C X 1 6 8 t wice dail y):  
− T hree 1 0 -m g C C X 1 6 8 ca ps ules i n t he m or ni n g a n d 3 ca ps ules i n t he e v e ni n g, 


C he m o Ce ntr y x, I n c.  
C O N FI D E N TI A L  
Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8 
 
 A me n d me nt 4. 0 
                                                                   Pa ge 1 4 of 1 1 3                                   1 8 Se pte m ber 2 0 1 5 
 
a p pr o xi matel y 1 2 h o urs  after t he m or ni n g d ose, d ail y f or 8 4  d a ys.  
− Pre d nis o ne 2 0 m g orall y per da y if t he s u bject’s b o d y w ei g ht is ≥5 5 k g or 1 5 m g per 
da y if t h e s u bject’s b o d y wei g ht is < 5 5 k g, starti n g o n Da y 1, wit h ta peri n g 
acc or di n g t o t he pr ot oc ol- s pecifie d s c he d ule. 
− Pre d nis o ne matc hi n g place b o ca ps ules e q ui vale nt t o 4 0 m g orall y per da y if t he 
s u bject’s b o d y wei g ht is ≥5 5 k g or 3 0 m g per da y if t he s u bject’s b o d y wei g ht is < 5 5 
k g, starti n g o n D a y 1, wit h ta peri n g acc or di n g t o a pr ot oc ol- s pecifi e d sc he d ule. 
− C ycl o p h os p ha mi de I V will be gi ve n o n D a y 1, a n d als o o n Da ys 1 5, 2 9, a n d 5 7; 
d oses o n Da ys 8 5, 1 1 3, 1 4 1, a n d 1 6 9 will be gi v e n at t he discreti o n of t he Pri nci pal 
I n v esti gat or.  
− Resc ue I V met h yl pr e d nis ol o ne or oral c ortic oster oi ds  s h o ul d be gi ve n t o s u bjects 
wit h w orse ni n g dis ease.  
•  Gr o u p B ( Place b o t wice dail y):  
− T hree place b o C C X 1 6 8 ca ps ules i n t he m or ni n g a n d 3 ca ps ules i n t he e v e ni n g, 
a p pr o xi matel y 1 2 h o urs after t he m or ni n g d ose, d ail y f or 8 4 d a ys. 
− Pre d nis o ne 6 0 m g orall y per da y if t he s u bject’s b o d y w ei g ht is ≥5 5 k g or 4 5 m g per 
da y if t h e s u bject’s b o d y wei g ht is < 5 5 k g, starti n g o n Da y 1 wit h ta peri n g acc or di n g 
t o a pr ot oc ol-s pecifie d sc he d ule. 
− C ycl o p h os p ha mi de I V will be gi ve n o n D a y 1 a n d als o o n Da ys 1 5, 2 9, a n d 5 7; 
d oses o n Da ys 8 5, 1 1 3, 1 4 1, a n d 1 6 9 will be gi v e n at t he discreti o n of t he Pri nci pal 
I n v esti gat or.  
− Resc ue I V met h yl pr e d nis ol o ne or oral c ortic oster oi ds  s h o ul d be gi ve n t o s u bjects 
wit h w orse ni n g dis ease.  
S T E P 2  
I n Ste p 2, st u d y dr u g a n d ot her me dicati o n f or v as c ulitis will be ta ke n as f oll o ws b y st u d y 
s u bjects: 
•  Gr o u p A ( 3 0 m g C C X 1 6 8 t wice dail y):  
− T hree 1 0 - m g C C X 1 6 8 ca ps ules i n t he m or ni n g a n d 3 i n t he e ve ni n g, a p pr o xi matel y 
1 2 h o urs after t h e m or ni n g d ose, d ail y f or 8 4 da ys.  
− Pre d nis o ne matc hi n g place b o ca ps ules e q ui vale nt t o 6 0 m g orall y per da y if t he 
s u bject’s b o d y wei g ht is ≥5 5 k g or 4 5 m g per da y if t he s u bject’s b o d y wei g ht is < 5 5 
k g, starti n g o n D a y 1 wit h ta peri n g acc or di n g t o a pr ot oc ol- s pecifi e d sc he d ule. 
− C ycl o p h os p ha mi de I V will be gi ve n o n D a y 1 a n d als o o n Da ys 1 5, 2 9, a n d 5 7; 
d oses o n Da ys 8 5, 1 1 3, 1 4 1, a n d 1 6 9 will be gi v e n at t he discreti o n of t he Pri nci pal 
I n v esti gat or.  


C he m o Ce ntr y x, I n c.  
C O N FI D E N TI A L  
Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8 
 
 A me n d me nt 4. 0 
                                                                   Pa ge 1 5 of 1 1 3                                   1 8 Se pte m ber 2 0 1 5 
 
− Resc ue I V met h yl pr e d nis ol o ne or oral c ortic oster oi ds  s h o ul d be gi ve n t o s u bjects 
wit h w orse ni n g dis eas e.    
•  Gr o u p B ( Place b o t wice dail y):  
− T hree place b o C C X 1 6 8 ca ps ules i n t he m or ni n g a n d 3 i n t he e ve ni n g, a p pr o xi matel y 
1 2 h o urs after t h e m or ni n g d ose, d ail y f or 8 4 da ys. 
− Pre d nis o ne 6 0 m g orall y per da y if t he s u bject’s b o d y w ei g ht is ≥5 5 k g or 4 5 m g per 
da y if t h e s u bject’s b o d y wei g ht is < 5 5 k g, starti n g o n Da y 1 wit h ta peri n g acc or di n g 
t o a pr ot oc ol-s pecifie d sc he d ule. 
− C ycl o p h os p ha mi de I V will be gi ve n o n D a y 1 a n d als o o n Da ys 1 5, 2 9, a n d 5 7; 
d oses o n Da ys 8 5, 1 1 3, 1 4 1, a n d 1 6 9 will be gi v e n at t he discreti o n of t he Pri nci pal 
I n v esti gat or.  
− Resc ue I V met h yl pr e d nis ol o ne or oral c ortic oster oi ds  s h o ul d be gi ve n acc or di n g t o 
s u bjects wit h w orse ni n g disease. 
S T E P 3  
I n Ste p 3, st u d y dr u g a n d ot her me dicati o n f or v as c ulitis will be ta ke n as f oll o ws b y st u d y 
s u bjects:  
•  Gr o u p A ( 3 0 m g C C X 1 6 8 t wice dail y wit h n o c ortic oster oi ds):  
− T hree 1 0 - m g C C X 1 6 8 ca ps ules i n t he m or ni n g a n d 3 i n t he e ve ni n g, a p pr o xi matel y 
1 2 h o urs after t h e m or ni n g d ose, d ail y f or 8 4 da ys.  
− Pre d nis o ne matc hi n g place b o ca ps ules e q ui vale nt t o 6 0 m g orall y per da y if t he 
s u bject's b o d y w ei g ht is ≥5 5 k g or 4 5 m g per da y if t he s u bject's b o d y wei g ht is < 5 5 
k g, starti n g o n D a y 1 wit h ta peri n g acc or di n g t o a pr ot oc ol- s pecifi e d sc he d ule.  
− If i n t he c ycl o p h os p ha mi de strat u m, c ycl o p h os p ha mi de I V will be gi v e n o n Da y 1 
a n d als o o n Da ys 1 5, 2 9, 5 7, a n d 8 5 ; starti n g o n D a y 9 9 t hr o u g h Da y 1 6 8, all 
s u bjects will recei ve oral azat hi o pri ne at a tar get d ose of 2 m g/ k g/ da y. 
− If i n t he rit u xi ma b strat u m, rit u xi ma b I V will be gi ve n o n Da ys 1, 8, 1 5, a n d 2 2 ( 3 7 5 
m g / m2 at eac h ti me p oi nt).  
− Resc ue I V met h yl pr e d nis ol o ne or oral ster oi ds s h o ul d be gi ve n t o s u bjects wit h 
w orse ni n g disease.  
•  Gr o u p B ( Place b o t wice dail y): 
− T hree place b o C C X 1 6 8 ca ps ules i n t he m or ni n g a n d 3 i n t he e ve ni n g, a p pr o xi matel y 
1 2 h o urs after t h e m or ni n g d ose, d ail y f or 8 4 da ys. 
− Pre d nis o ne 6 0 m g orall y per da y if t he s u bject's b o d y w ei g ht is ≥5 5 k g or 4 5 m g per 
da y if t h e s u bject's b o d y wei g ht is < 5 5 k g, starti n g o n Da y 1 wit h ta peri n g acc or di n g 


C he m o Ce ntr y x, I n c.  
C O N FI D E N TI A L  
Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8 
 
 A me n d me nt 4. 0 
                                                                   Pa ge 1 6 of 1 1 3                                   1 8 Se pte m ber 2 0 1 5 
 
t o t he pr ot oc ol -s pecifi e d sc he d ule. 
− If i n t he c ycl o p h os p ha mi de strat u m, c ycl o p h os p ha mi de I V will be gi v e n o n Da y 1 
a n d als o o n Da ys 1 5, 2 9, 5 7, a n d 8 5 ; starti n g o n D a y 9 9 t hr o u g h Da y 1 6 8, all 
s u bjects will recei ve oral azat hi o pri ne at a tar get d ose of 2 m g/ k g/ da y. 
− If i n t he rit u xi ma b strat u m, rit u xi ma b I V will be gi ve n o n Da ys 1, 8, 1 5, a n d 2 2 ( 3 7 5 
m g / m2 at eac h ti me p oi nt).  
− Resc ue I V met h yl pr e d nis ol o ne or oral ster oi ds s h o ul d be gi ve n t o s u bjects wit h 
w orse ni n g disease.  
•  Gr o u p C ( 3 0 m g C C X 1 6 8 t wice dail y wit h re d uce d c ortic oster oi ds):  
− T hree 1 0 - m g C C X 1 6 8 ca ps ules i n t he m or ni n g a n d 3 ca ps ules i n t he e v e ni n g, 
a p pr o xi matel y 1 2 h o urs after t he m or ni n g d ose, d ail y f or 8 4 d a ys.  
− Pre d nis o ne 2 0 m g orall y per da y if t he s u bject's b o d y w ei g ht is ≥5 5 k g or 1 5 m g per 
da y if t h e s u bject's b o d y wei g ht is < 5 5 k g, starti n g o n Da y 1, wit h ta peri n g acc or di n g 
t o t he pr ot oc ol-s pecifi e d sc he d ule. 
− Pre d nis o ne matc hi n g place b o ca ps ules e q ui vale nt t o 4 0 m g orall y per da y if t he 
s u bject's b o d y w ei g ht is ≥5 5 k g or 3 0 m g per da y if t he s u bject's b o d y wei g ht is < 5 5 
k g, starti n g o n D a y 1, wit h ta peri n g acc or di n g t o a pr ot oc ol- s pecifi e d sc he d ule. 
− If i n t he c ycl o p h os p ha mi de strat u m, c ycl o p h os p ha mi de I V will be gi v e n o n Da y 1, 
a n d als o o n Da ys 1 5, 2 9, 5 7, a n d 8 5 ; starti n g o n D a y 9 9 t hr o u g h Da y 1 6 8, all 
s u bjects will recei ve oral azat hi o pri ne at a tar get d ose of 2 m g/ k g/ da y. 
− If i n t he rit u xi ma b strat u m, rit u xi ma b I V will be gi ve n o n Da ys 1, 8, 1 5, a n d 2 2 ( 3 7 5 
m g / m2 at eac h ti me p oi nt).  
− Resc ue I V met h yl pr e d nis ol o ne or oral c ortic oster oi ds s h o ul d be gi ve n t o s u bjects 
wit h w orse ni n g dis ease. 
F oll o wi n g t he 8 4- da y d osi n g peri o d, t he re will be a n 8 4- da y f oll o w- u p peri o d. All s u bjects will 
visit t he st u d y ce nter d uri n g t he scree ni n g p eri o d, a n d o n Da ys 1, 2, 8, 1 5, 2 2, 2 9, 4 3, 5 7, 7 1, 8 5, 
9 9, 1 1 3, 1 4 1, a n d 1 6 9. S u bjects will n ot ta ke st u d y me dicati o n i n t he m or ni n g of t he d a ys of 
visits t o t he st u d y ce nter, a n d will be i nstr ucte d t o ta ke t he me dicati o n w hile at t he st u d y ce nter. 
F or t he ot her st u d y d a ys, st u d y me dicati o n will be ta ke n at h o me i n t he m or ni n g wit hi n 1 h o ur 
after br ea kfast a n d i n t he e ve ni n g wit hi n 1 h o ur aft er di n ner.  
T he  scree ni n g peri o d will be ke pt as s h ort as p ossi ble i n or der n ot t o dela y i nitiati o n of 
treat me nt. T he scree ni n g peri o d m ust n ot e x cee d 1 4 da ys. Scree ni n g pr oce d ures will i ncl u de 
de m o gr a p hics, me dical hist or y, me dicati o n hist or y, p h ysical e x a mi nati o n a n d vital si g ns, ser u m 
c he mistr y, h e mat ol o g y, uri nal ysis (i ncl u di n g he m at uria, pr ote i n uria [A C R ]), E C G, c hest X ra ys, 
viral scree ni n g, s er ol o g y a n d c o m ple me nt meas ure me nts (if n ot d o ne wit hi n t he pre vi o us 1 2 
m o nt hs), esti mate d gl o m er ular filtr ati o n rate (e G F R) assess me nt, A N C A m eas ure m e nt (i n direct 
i m m u n ofl u oresce nce test f or P-A N C A a n d C -A N C A, as well as E LI S A tests f or a nti - P R 3 a n d 
a nti-M P O), re nal bi o ps y (if necessar y f or st u d y eli gi bilit y), a n d B V A S assess me nt. T o e x pe dite 


C he m o Ce ntr y x, I n c.  
C O N FI D E N TI A L  
Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8 
 
 A me n d me nt 4. 0 
                                                                   Pa ge 1 7 of 1 1 3                                   1 8 Se pte m ber 2 0 1 5 
 
t he scree ni n g pr ocess, bl o o d a n d uri ne tests will be d o ne at t he l ocal la b orat ories f or t he 
Scree ni n g visit. La b orat or y res ults fr o m t he l ocal la b orat ories o btai n e d wit hi n 7 2 h o urs of 
scree ni n g are acce pta ble i n or der t o a v oi d u n necessar y bl o o d dra ws. Eli gi ble s u bjects m ust be 
A N C A - p ositi ve a n d m ust ha ve at least o ne “ maj or” ite m, or at least 3 n o n- maj or  ite ms, or at 
least t w o re nal ite ms o n t he B V A S  versi o n 3.  
Eli gi ble s u bjects will visit t he st u d y ce nt er o n D a y 1, after a n o ver ni g ht fast of at least 8 h o urs, 
f or p h ysical e x a mi nati o n a n d vital si g ns, ser u m c h e mistr y, he m at ol o g y, uri nal ysis (i ncl u di n g 
he mat uria, pr otei n uri a ( A C R), a n d M C P- 1:creati ni ne rati o asse ss me nt), e G F R, A N C A 
meas ure me nt ( a nti - P R 3 a n d a nti- M P O), a B V A S a n d V DI assess me nt, S F - 3 6 v 2 a n d E Q- 5 D-5 L 
assess me nt,  hs C R P, baseli ne p har mac o ki netics a n d p har mac o d y n a mics ( P K/ P D) bl o o d sa m ple 
c ollecti o n, a n d ra n d o mizati o n. Me dicati o n will be a d mi nistere d (I V) a n d dis pe nse d (f or oral 
me dicati o ns). T he s u bjects will ta ke t he first d ose of C C X 1 6 8 or place b o, a n d pre d nis o ne or 
place b o w hil e at t he st u d y ce nter. T h e s u bjects will sta y at t he cli nic f or at least 6 h o urs after t he 
first d ose o n Da y 1 f or safet y o bs er vati o n a n d P K sa m ple c ollecti o n. A s u bj ect c o ul d be k e pt 
o ver ni g ht i n t he h os pital o n Da y 1, if necessar y. T his h os pital sta y w o ul d n ot be c o nsi dere d a 
seri o us a d vers e e v e nt, u nless ot her S A E criteri a are met. 
T wice dail y d osi n g of C C X 1 6 8 or place b o will c o nti n ue f or 8 4 da ys. At p ost- Da y 1 st u d y visits, 
st u d y me dicati o n will be a d mi nistere d acc or di n g t o t he pr ot oc ol sc he d ule, bl o o d a n d uri ne 
sa m ples will be c ollecte d f or saf et y a n d efficac y a n d P K/ P D meas ure me nts. B V A S assess me nts 
will be ma de o n Da ys 1,  2 9, 8 5, 1 1 3, a n d 1 6 9. V DI assess me nt will be ma d e o n Da ys 1, 8 5, a n d 
1 6 9. S F - 3 6 v 2 a n d E Q-5 D -5 L i nstr u me nts will be c o m plete d o n Da y s 1, 2 9, 8 5, a n d 1 6 9 i n Ste p 
3.  P h ysical e x a mi nati o ns, b o d y s yste m re vie ws, a n d vital si g n assess me nts will be perf or me d 
t hr o u g h o ut t he st u d y. C o nc o mita nt me dicati o n a n d a d verse e ve nt ass ess me nts will be ma de at 
e ver y st u d y visit.  
S u bjects will be disc har ge d fr o m t he st u d y w he n all t he St u d y D a y 1 6 9 visit pr oce d ures ha ve 
bee n c o m plete d. T h e s u bject’s c o n diti o n will be e val uate d b y t he I n v esti gat or at t he e n d of t he 
cli nical trial ( Da y 1 6 9) a n d a p pr o priate sta n d ar d of care me dical treat me nt will be p r o vi de d t o 
all s u bjects as nee d e d.  
T o t he e xte nt p ossi ble, a n y a d v erse e ve nts t hat are dee me d st u d y dr u g -rel ate d a n d are o n g oi n g 
at disc har ge will be f oll o we d -u p t o res ol uti o n or u ntil a deter mi nati o n is ma de t hat t he 
u nres ol ve d e v e nt is sta ble.  


C he m o Ce ntr y x, I n c.  
C O N FI D E N TI A L  
Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8 
 
 A me n d me nt 4. 0 
                                                                   Pa ge 1 8 of 1 1 3                                   1 8 Se pte m ber 2 0 1 5 
 
N u m ber of S u bjec ts 
A p pr o xi matel y 6 0 m ale or p ost me n o pa usal or s ur gicall y sterile fe mal e s u bjects wit h A A V will 
be ra n d o mize d f or t his st u d y. S u bjects w h o dr o p o ut of t he st u d y pre mat urel y will n ot be 
re place d. T hese s u bj ects will be part of t he i nte nt-t o-treat p o p ulati o n. S u bjects will n ot be 
all o we d t o partici pate i n m ore t ha n o ne Ste p of t h e st u d y.  
M ai n Criteri a f or I ncl usi o n  
1.  
Cli nical dia g n osis of gra n ul o mat osis wit h p ol ya n giitis  ( We ge ner’s), micr osc o pic 
p ol ya n giitis or re n al li mite d vasc ulitis, c o nsiste nt wit h C ha pel - Hill c o nse ns us defi niti o ns 
(J e n nette et al., 2 0 1 3 ); 
2.  
Male a n d p ost me n o pa usal ( l ac k of me nses f or at least 2 years  wit h o ut a n alter n ati ve 
e x pl a n ati o n ) or s ur gicall y sterile fe male s u bj ects, a ge d at least 1 8 years , wit h ne w ( wit hi n 
4 we e ks pri or t o scree ni n g) or rela ps e d A A V w here treat me nt wit h c ycl o p h os p ha mi de or 
rit u xi ma b w o ul d be re q uire d; If fe m ale u n der 5 0 ye ars, t he p ost me n o p a us al st at us 
s h o ul d be c o nfir me d b y t he rele v a nt h or m o n al test. Male s u bj ects wit h part ners of 
c hil d beari n g p ote ntial ma y partici pate i n t he st u d y if t he y h a d a vas ect o m y at least 6 
m o nt hs pri or t o ra n d o mizati o n or if a de q u ate c o ntrace pti o n is use d d uri n g, a n d f or at least 
t he t hree m o nt hs after st u d y c o m pleti o n; A de q u ate c o ntrace pti o n is defi ne d as res ulti n g i n 
a fail ure rate of less t ha n 1 % per year;  a cce pt a ble met h o ds i ncl u de c o m bi ne d estr o ge n 
a n d pr o gest o ge n ( or al, i ntr a v a gi n al, or tr a ns der m al), or pr o g est o ge n- o nl y h or m o n al 
c o ntr ace pti o n ( o r al, i nject a ble, or i m pl a nt a ble), i ntr a -uteri ne de vice, i ntr a uteri ne 
h or m o ne rele asi n g s yste m, bil ater al t u b al occl usi o n, v asect o mize d p art ner, or se x u al 
a bsti ne nce;  
3.  
P ositi ve i n direct i m m u n ofl u oresce n ce (II F) test f or P -A N C A or C - A N C A, or p ositi ve 
E LI S A test f or a nti - pr ot ei nase- 3 ( P R 3) or a nti-m yel o per o xi dase ( M P O) at scree ni n g; If 
o nl y t he II F assa y is p ositi ve at scree ni n g, a n d n o ne of t he E LI S A tests, t h ere m ust be 
d oc u me ntati o n i n t he st u d y r ec or ds of a p ositi ve E LI S A assa y i n t he past;  
4.  
Ha ve at least o ne “ maj or ” ite m , or at least 3 n o n- maj or  ite ms, or at least 2 re nal ite ms o n 
t he B V A S versi o n 3; 
5.  
Esti mate d gl o mer ular filtrati o n rate ≥ 2 0 m L per mi n ute ( M D R D); 
6.  
Willi n g a n d a ble t o gi ve writte n I nf or me d C o nse nt a n d t o c o m pl y wit h t he re q uire me nts of 
t he st u d y pr ot oc ol; a n d 
7.  
J u d ge d t o be ot her wise h ealt h y b y t h e I n vesti gat or, base d o n me dical hist or y, ph ysical 
e x a mi nati o n (i ncl u di n g electr ocar di o gra m [ E C G]), a n d cli nical la b orat or y assess me nts. 
S u bjects wit h cli nical la b orat or y val ues t hat are o utsi de of n or mal li mits ( ot her t ha n t h ose 
s pecifie d i n t he E x cl usi o n Criteria) a n d/ or wit h ot her a b n or m al cli nical fi n di n gs t hat are 
j u d ge d b y t he I n vesti gat or n ot t o be of cli nical si g nifica nce, m a y be e nt ere d i nt o t he st u d y.     
M ai n Criteri a f or E xcl usi o n  
1.  
Se vere disease as d eter mi ne d b y ra pi dl y pr o gressi ve gl o mer ul o ne p hritis s uc h t hat 
c o m me nce me nt of re n al re pl ace me nt t hera p y c o ul d be a ntici pate d wit hi n 7 da ys, al ve olar 


C he m o Ce ntr y x, I n c.  
C O N FI D E N TI A L  
Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8 
 
 A me n d me nt 4. 0 
                                                                   Pa ge 1 9 of 1 1 3                                   1 8 Se pte m ber 2 0 1 5 
 
he m orr ha ge lea di n g t o Gra de 3 or hi g h er h y p o xia (i.e., decreas e d o x y ge n s at urati o n at 
rest, e. g., p ulse o xi meter < 8 8 % or Pa O2 ≤5 5 m m H g), he m o pt ysis, ra pi d-o nset 
m o n o ne uritis m ulti ple x ( Gra de 3 o r hi g h er, lea di n g t o se vere s y m pt o ms t hat li mit self care 
acti vities of dail y li vi n g or re q uiri n g a n assisti ve de vice), or ce ntral ner v o us s yste m 
i n v ol ve me nt; 
2.  
A n y ot her m ulti -s yste m a ut oi m m u ne disease i ncl u di n g e osi n o p hilic gra n ul o mat osis wit h 
p ol ya n giitis  (C h ur g Stra uss), s yste mic l u p us er yt h e mat os us, I g A vasc ulitis (He n oc h -
Sc h ö nlei n p ur p ura), r he u mat oi d vasc ulitis, Sj ö gre n’s disease, a nti- gl o mer ular base me nt 
me m bra ne disease, or cr y o gl o b uli ne mia; 
3.  
Me dical hist or y of c o a g ul o pat h y or blee di n g dis or der; 
4.  
Recei ve d c ycl o p h os p ha mi de wit hi n 1 2 wee ks pri or t o scree ni n g; if o n azat hi o pri ne, 
m yc o p h e n olate m ofetil, or met h otre x ate at t he ti me of scree ni n g, t hese dr u gs m ust be 
wit h dra w n pri or t o r ecei vi n g t he c ycl o p h os p ha mi de  or rit u xi ma b  d ose o n Da y 1;  
5.  
Recei ve d i ntra v e n o us c ortic oster oi ds, > 3 0 0 0 m g m et h yl pre d nis ol o ne e q ui vale nt, wit hi n 1 2 
wee ks pri or t o scree ni n g; 
6.  
Ha ve bee n ta ki n g a n or al dail y d ose of a c ortic oster oi d of m ore t ha n 1 0 m g pre d nis o ne-
e q ui vale nt f or m or e t ha n 6 wee ks c o nti n u o usl y pri or t o t he scree ni n g visit. If o n a n oral 
c ortic oster oi d at a d ail y d ose of m ore t ha n 1 0 m g pre d nis o ne e q ui val e nt at t he ti me of 
scree ni n g, t he oral d ose n ee ds t o be re d uce d t o a d ail y d ose n ot e x cee di n g 1 0 m g 
pre d nis o ne-e q ui vale nt pri or t o Da y 1; 
7.  
Recei ve d rit u xi ma b or ot her B-cell a nti b o d y wit hi n 5 2 wee ks of s cree ni n g or 2 6 wee ks 
pr o vi de d B cell rec o nstit uti o n has occ urre d (i.e., C D 1 9 c o u nt > 0. 0 1 x 1 0 9/ L); recei ve d a nti-
T N F treat me nt, a bat ace pt, ale mt uz u ma b, I VI g, beli m u ma b, t ociliz u ma b,  or plas ma 
e x c ha n ge wit hi n 1 2 wee ks pri or t o scree ni n g; 
8.  
S y m pt o matic c o n gesti ve heart fail ure re q uiri n g prescri pti o n me dicati o n, cli nicall y e vi de nt 
peri p her al e de ma  of car diac ori gi n, p o orl y-c o ntr olle d h y p erte nsi o n (s yst olic bl o o d 
press ure > 1 6 0 or diast olic bl o o d press ure > 1 0 0), hist or y of u nsta ble a n gi na, m y ocar dial 
i nfarcti o n or str o ke wit hi n 6 m o nt hs pri or t o scree ni n g; 
9.  
Hist or y or prese n ce of a n y f or m of ca n cer wit hi n t he 5 years pri or t o scree ni n g, wit h t he 
e x ce pti o n of e x cise d basal cell or s q ua m o us cell carci n o ma of t h e s ki n, or cer vical 
carci n o ma i n sit u or br east carci n o ma i n sit u t hat has bee n e x cise d or resecte d c o m pletel y 
a n d is wit h o ut e vi de nce of l ocal rec urre nce or met astasis;  
1 0.  E vi de nce of t u b erc ul osis base d o n c hest X ra ys perf or me d d uri n g s cree ni n g as part of t he 
B V A S assess me nt;  
1 1.  P ositi ve H B V, H C V, or HI V viral scree ni n g test; 
1 2.  A n y i nf ecti o n re q uiri n g a nti bi otic treat me nt wit hi n 4 wee ks pri or t o scree ni n g (e x ce pt f or 
pr o p h ylactic treat me nt f or P ne u m ocystis c ari nii  p ne u m o nia [ P C P] or treat me nt f or 
s us pecte d i nfecti o n t hat i nstea d t ur ns o ut t o be a c o nse q ue nce of A N C A v asc ulitis, e. g., 
p ne u m o nitis); 


C he m o Ce ntr y x, I n c.  
C O N FI D E N TI A L  
Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8 
 
 A me n d me nt 4. 0 
                                                                   Pa ge 2 0 of 1 1 3                                   1 8 Se pte m ber 2 0 1 5 
 
1 3.  Recei ve d a li ve v acci ne wit hi n 4 wee ks pri or t o s cree ni n g;  
1 4.  W B C c o u nt less t ha n 4 0 0 0/ µ L , or ne utr o p hil c o u nt less t ha n 2 0 0 0/µ L, or l y m p h oc yte 
c o u nt less t ha n 1 0 0 0/ µ L;  
1 5.  He m o gl o bi n less t ha n 9 g/ d L ( or 5. 5 6 m m ol/ L) at s cree ni n g;  
1 6.  E vi de nce of h e patic disease; A S T, A L T, al kali ne p h os p hatase, or bilir u bi n > 3 x t he u p per 
li mit of n or mal; 
1 7.  Pr ot hr o m bi n ti me ( P T) or partial t hr o m b o plasti n ti me ( P T T) a b o ve t he n or mal refere nce 
li mit; 
1 8.  Cli nicall y si g nifica nt a b n or mal E C G d uri n g s cree ni n g, e. g., Q Tc F greater t ha n 4 5 0 msec; 
1 9.  Partici pate d i n a n y cli nical st u d y of a n i n vesti gati o nal pr o d uct wit hi n 3 0 da ys pri or t o 
scree ni n g or wit hi n 5 half-li ves after ta ki n g t he last d ose; a n d 
2 0.  Hist or y or prese n ce of a n y me dical c o n d iti o n or disease w hic h, i n t he o pi ni o n of t he 
I n v esti gat or, ma y place t he s u bject at u nacce pt a ble ris k f or st u d y partici p ati o n. 
Test Pr o d uct  
C C X 1 6 8 will be a d mi nistere d via har d gelati n ca ps ules c o ntai ni n g 1 0 m g C C X 1 6 8. T he 
C C X 1 6 8 ca ps ules will be s u p plie d t o t he st u d y ce nters i n plastic b ottles c o ntai ni n g 3 0 ca ps ules.  
S u bjects i n Gr o u p A i n Ste ps 1 a n d 2, a n d Gr o u ps A a n d C i n Ste p 3  ( 3 0 m g C C X 1 6 8) will 
recei v e o ne kit c o ntai ni n g 2 b ottles of C C X 1 6 8 ca ps ules o n Da ys 1, 8, 1 5, a n d 2 2. S u bjects i n 
Gr o u p A i n Ste ps 1 a n d 2, a n d Gr o u ps A a n d C i n Ste p 3  will recei ve t w o kits c o ntai ni n g 2 
b ottles eac h of C C X 1 6 8 ca ps ules o n Da ys 2 9, 4 3, 5 7, a n d 7 1. S u bjects will be as ke d t o ta ke 3 
ca ps ules e v er y m or ni n g a n d 3 ca ps ules e v er y e v e ni n g, a p pr o xi matel y 1 2 h o urs after t h e 
m or ni n g d ose, as i nstr ucte d. St u d y me dicati o n will be ta ke n wit hi n 1 h o ur after brea kfast i n t he 
m or ni n g a n d wit hi n 1 h o ur after di n n er i n t he e v e ni n g f or 8 4 d a ys c o nti n u o usl y. Ca ps ules will 
be ta ke n wit h wat er, pr ef era bl y wit h 5 0 m L, b ut n ot t o e x cee d 1 0 0 m L. Place b o a n d C C X 1 6 8 
b ottles a n d ca ps ules will be i de ntical i n a p peara n ce.    
Ref ere nce T her a p y, D ose a n d M o de of A d mi nistr ati o n  
Gr o u p B will be t he pl ace b o c o ntr ol gr o u p. Place b o ca ps ules will be s u p plie d t o t he st u d y 
ce nters i n plastic b ottles c o ntai ni n g 3 0 ca ps ules.  
S u bjects i n Gr o u p B ( place b o) will recei ve 2 b ottl es of place b o ca ps ules o n Da ys 1, 8, 1 5, a n d 
2 2. S u bjects i n Gr o u p B will recei ve 4 b ottles of place b o ca ps ules o n Da ys 2 9, 4 3, 5 7, a n d 7 1. 
S u bjects will be as ke d t o ta ke 3 ca ps ules e ver y m or ni n g a n d 3 ca ps ules e ver y e v e ni n g, 
a p pr o xi matel y 1 2 h o urs after t he m or ni n g d ose, as i nstr ucte d. St u d y me dicati o n will be ta ke n 
wit hi n 1 h o ur after br ea kfast i n t he m or ni n g a n d wit hi n 1 h o ur after di n ner i n t he e ve ni n g f or 8 4 
da ys c o nti n u o usl y. Ca ps ules will be ta ke n wit h w ater, prefera b l y wit h 5 0 m L, b ut n ot t o e x cee d 
1 0 0 m L.  
Pre d nis o ne will be gi v e n as ta blets, o vere n ca ps ulat e d wit h har d gelati n ca ps ules i n or der t o 
mai ntai n t he bli n di n g. T w o d ose stre n gt hs of pre d nis o ne will be pr o vi de d, 2 0 m g a n d 5 m g. 
Place b o f or pr e d nis o ne will be gi ve n as mat c hi n g har d gelati n ca ps ules wit h i nert filler.  


C he m o Ce ntr y x, I n c.  
C O N FI D E N TI A L  
Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8 
 
 A me n d me nt 4. 0 
                                                                   Pa ge 2 1 of 1 1 3                                   1 8 Se pte m ber 2 0 1 5 
 
D ur ati o n of Tre at me nt a n d O bser v ati o n   
S u bjects will be scree n e d wit hi n a peri o d n ot t o e xcee d 1 4 d a ys pri or t o St u d y D a y 1 (t he first 
da y of d osi n g). T he tr eat me nt peri o d is 8 4 da ys a n d all s u bjects will be f oll o we d f or 8 4 da ys 
after t he d osi n g peri o d.  
T o t he e xte nt p ossi ble, a n y a d v erse e ve nts t hat are dee me d st u d y dr u g -rel ate d a n d are o n g oi n g 
at disc har ge will be f oll o we d - u p t o res ol uti o n or u ntil a deter mi nati o n is ma de t hat t he 
u nres ol ve d e v e nt is sta ble. T he s u bject’s c o n diti o n will be e val uate d b y t h e I n vesti gat or at t he 
e n d of t he cli nical trial a n d a p pr o priate sta n d ar d of care me dical treat me nt will be pr o vi de d t o 
all s u bjects as nee d e d.  
S af et y As sess me nts 
Safet y assess me nts i ncl u de a d vers e e v e nts, p h ysical e x a mi nati o n a b n or malities, vital si g ns, a n d 
cli nical la b orat or y tests (i ncl u di n g bl o o d c h e mistr y, he mat ol o g y, a n d uri nal ysis), a n d E C Gs.  
Effic ac y Assess me nts  
E fficac y assess me nts i ncl u de: 
1.  B V A S versi o n 3; 
2.  e G F R b y M o dificati o n of Diet i n Re nal Disease ( M D R D) f or m ula;  
3.  He mat uria a n d pr otei n uri a ( A C R);  
4.  C ortic oster oi d I V or or al resc ue us e ( d ose a n d d urati o n of d osi n g); 
5.  V DI;  
6.  A N C A (a nti - P R 3 a n d a nti-M P O b y E LI S A);  
7.  Ser u m C -reacti ve pr otei n c o nce ntrati o n meas ure d b y hi g h se nsiti vit y C R P assa y;  
8.  Uri nar y M C P -1:creati ni ne rati o , a n d 
9.  S F - 3 6 v 2 a n d E Q- 5 D-5 L meas ure me nts . 
P h ar m a c o ki netic Assess me nts  
C o nce ntrati o ns of C C X 1 6 8 a n d p ossi ble meta b olites will be deter mi ne d i n plas ma fr o m bl o o d 
sa m ples c ollecte d i n E D TA t u bes o n Da ys 1, 8, 1 5, 2 2, 2 9, 4 3, 5 7, 7 1,  a n d 8 5. O n Da y 1, 
sa m ples will be ta ke n at pre- d ose, 0. 5, 1, 2, 3, 4, a n d 6 h o urs after d osi n g.  
Uri ne will be c ollect e d o n Da y 1 starti n g after a c o m plete v oi d pri or t o d osi n g, a n d c ollecti o n of 
all uri ne u p t o t he 6- h o ur ti me p oi nt f oll o wi n g d osi n g. C o nce ntrati o ns of C C X 1 6 8 a n d p ossi ble 
meta b olites will be meas ure d i n a r e pres e ntati ve s a m ple fr o m t his 6 - h o ur c ollecti o n. 
P h ar m a c o d y n a mi c Ass ess me nts  
A plas ma sa m ple will be c ollecte d o n D a ys 1, 2 9, 8 5, a n d 1 6 9 f or p har mac o d y na mic m ar ker 
meas ure me nts, i ncl u di n g f or e x a m ple c ystati n C, c o m ple me nt fra g m e nts, a n d i nfla m mat or y 
c yt o ki ne a n d c h e m o ki ne le vels. T he P K plas ma sa m ples ma y als o b e use d f or t hese 


C he m o Ce ntr y x, I n c.  
C O N FI D E N TI A L  
Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8 
 
 A me n d me nt 4. 0 
                                                                   Pa ge 2 2 of 1 1 3                                   1 8 Se pte m ber 2 0 1 5 
 
p har mac o d y n a mics mar k er meas ur e me nts.  
Uri ne sa m ples will als o b e c ollecte d o n Da ys 1, 8, 1 5, 2 9, 5 7, 8 5, 1 1 3, a n d 1 6 9 f or bi o mar ker 
assess me nts i ncl u di n g f or e x a m ple c o m ple me nt fra g m e nts, i nfla m mat or y c he m o ki ne a n d 
c yt o ki ne l e vels.  
A sali va sa m ple will be c ollecte d o n Da y 1 fr o m s u bjects w h o ha ve pr o vi de d i nf or me d c o ns e nt 
f or assess me nt of ge netic mar kers of A N C A disease as well as t he c o m ple me nt pat h wa y. 
St atistic al Met h o ds    
De m o gr a p hics a n d B aseli ne C h ar acteristics  
All s u bject baseli ne c h ar acteristics a n d d e m o gra p hic data (a ge, se x, race, et h nicit y, wei g ht, 
hei g h t, b o d y mass i n de x, s m o ki n g stat us, E C G, T B scree n res ults, viral test res ults, A N C A, 
ser ol o g y t est res ults, vasc ulitis disease d urati o n (fr o m ti me of first i n d ucti o n treat me nt), B V A S, 
V DI, S F - 3 6 v 2 s c ore, E Q- 5 D-5 L sc ore,  hs C R P, e G F R, he mat uria, pr otei n uria ( A C R), 
gl o mer ular hist o pat h ol o g y (if bi o ps y w as ta ke n), uri nar y M C P- 1:creati ni ne rati o, p h ysical 
e x a mi nati o n a b n or malities, me dical hist or y, pre vi o us ( wit hi n 6 m o nt hs of scree ni n g) a n d 
c o nc o mita nt me dicati o ns (i ncl u di n g vas c ulitis me dicati o n use) at st u d y  e ntr y will be liste d b y 
treat me nt gr o u p, st u d y ce nter, a n d s u bject n u m ber, a n d will als o be s u m marize d b y treat me nt 
gr o u p a n d ste p of t he st u d y.  
S afety A n alysis  
T he pri mar y safet y e n d p oi nt is t he s u bject i nci de nce of a d v erse e ve nts.  
Ot her saf et y e n d p oi nts i ncl u de: 
1.  S u bject i nci de nce of e ve nts p ossi bl y ass ociate d wit h gl uc oc ortic oi d us e: seri o us i nfecti o ns, 
ne w -o nset dia b etes mellit us/ h y per gl yce mia, b o ne fract ure, pe ptic ulcer disease, cat aracts, 
ne w o nset/ w orse ni n g h y p erte nsi o n, wei g ht gai n m ore t ha n 1 0 k g, a n d ps yc hiatric dis or ders; 
2.  S u bject i nci de nce of i nfecti o ns, seri o us i nfecti o ns, se vere i nfecti o ns (i.e., Gra de 3), a n d 
i nfecti o ns lea di n g t o s u bject wit h dra w al fr o m t he st u d y; 
3.  C ha n ge fr o m bas eli ne i n all safet y la b orat or y para meters;  
4.  C ha n ge fr o m bas eli ne i n vital si g ns; 
5.  I n ci de nce of cli nicall y si g nifica nt E C G c ha n ges fr o m baseli ne. 
All s u bjects w h o are ra n d o mize d a n d recei ve d at least o ne d ose of st u d y me dicati o n will be 
i ncl u de d i n t he safet y p o p ulati o n.  
All cli nical safet y a n d t ol era bilit y d ata will be  liste d b y treat me nt gr o u p a n d b y s u bject, a n d will 
be s u m marize d b y tr eat m e nt gr o u p. All re p orte d a d verse e v e nts will be c o d e d usi n g Me d D R A 
a n d liste d b y S yste m Or ga n Class, preferre d ter m, a n d ver bati m ter m. Treat me nt -e mer ge nt 
a d verse e ve nts will be list e d a n d s u m marize d b y S yste m Or ga n Class, b y relate d ness a n d b y 
ma xi m u m se verit y a n d c o m pare d acr oss treat me nt gr o u ps. Seri o us a d v erse e ve nts a n d a d v erse 
e ve nts lea di n g t o wit h dra wal will be s u m marize d a n d c o m pare d acr oss treat me nt gr o u ps. 
I n di vi d ual vital si g ns a n d c ha n ge fr o m baseli ne i n vital si g ns will be liste d b y treat me nt gr o u p, 
s u bject, a n d st u d y visit, a n d s u m marize d descri pti vel y. La b orat or y d ata (act ual val ues a n d 


C he m o Ce ntr y x, I n c.  
C O N FI D E N TI A L  
Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8 
 
 A me n d me nt 4. 0 
                                                                   Pa ge 2 3 of 1 1 3                                   1 8 Se pte m ber 2 0 1 5 
 
c ha n ge fr o m baseli ne) will be liste d b y treat me nt gr o u p, s u bject, a n d st u d y visit. A b n or mal 
la b orat or y val u es will be fla g ge d. La b orat or y d ata will als o be s u m marize d b y treat me nt gr o u p 
a n d st u d y visit. E C Gs will be ac q uire d at Baseli ne ( Da y 1) a n d Da y 2 9. E C Gs will be e val uate d 
f or a n y a b n or malities, w hic h will be assesse d f or cli nical si g nifica nce. 
T he i nci de nce of a d v erse e ve nts a n d seri o us a d verse e ve nts will be c o m pare d bet wee n C C X 1 6 8 
a n d sta n dar d of care c o ntr ol gr o u ps f or all 3 ste ps.  
Effic acy A n alysis  
T he pri mar y efficac y e n d p oi nt is t he pr o p orti o n of s u bjects ac hie vi n g disease res p o nse at Da y 
8 5 defi ne d as B V A S perce nt re d ucti o n fr o m b aseli ne of at least 5 0 % pl us n o w orse ni n g i n a n y 
b o d y s yste m c o m p o ne nt.  
Ot her efficac y e n d p oi nts i ncl u de: 
1.  I n p atie nts wit h he mat uri a a n d al b u mi n uria at b aseli ne, t he pr o p orti o n of s u bjects ac hie vi n g 
re nal r es p o nse at D a y 8 5; re nal res p o nse is defi ne d as a n i m pr o ve me nt i n para meters of 
re nal vas c ulitis: 
a. a n i ncrease fr o m baseli ne t o Da y 8 5 i n e G F R ( M D R D ser u m creati ni ne e q u ati o n), pl us 
b.  a decr eas e fr o m bas eli ne t o Da y 8 5 i n h e mat uria (ce ntral la b orat or y micr os c o pic c o u nt 
of uri nar y R B Cs), pl us 
c. a decr eas e fr o m bas eli ne t o Da y 8 5 i n al b u mi n uria (first m or ni n g uri nar y 
al b u mi n:creati ni ne rati o).  
2.  Pr o p orti o n of s u bjects ac hie vi n g diseas e re missi o n at Da y 8 5 defi ne d as B V A S of 0 or 1 
pl us n o w orse ni n g i n e G F R a n d uri nar y R B C c o u nt < 1 0/ h pf; 
3.  P erce nt c h a n ge fr o m bas eli ne t o Da y 8 5 i n B V A S; 
4.  C ha n ge a n d perce nt c ha n ge fr o m baseli ne t o D a y 8 5 i n e G F R; 
5.  I n s u bjects wit h baseli ne he mat uria > 5 R B Cs/ h pf, t he pr o p orti o n of s u bjects a n d ti me t o 
first ac hie vi n g uri nar y R B C c ou nt ≤ 5/ h pf at a n y ti me d uri n g t he 8 4- da y treat me nt peri o d; 
6.  I n s u bjects wit h baseli ne he mat uria ≥ 3 0 R B Cs/ h pf, t he pr o p orti o n of s u bjects a n d ti me t o 
first ac hie vi n g uri nar y R B C c o u nt < 3 0/ h pf at a n y ti me d uri n g t he 8 4- d a y treat me nt peri o d; 
7.  I n s u bjects with he mat uri a at baseli ne, t he p erce nt c ha n ge fr o m baseli ne t o Da y 8 5 i n 
uri nar y R B C c o u nt; 
8.  I n s u bjects wit h al b u mi n uria at baseli ne, t h e perce nt c ha n ge fr o m baseli ne t o Da y 8 5 i n 
uri nar y A C R;  
9.  Perce nt c h a n ge fr o m bas eli ne t o Da y 8 5 i n uri nar y M C P -1:creati ni ne rati o;  
1 0.  Pr o p orti o n of s u bjects re q uiri n g resc ue I V or oral gl uc o c ortic oi d treat me nt;  
1 1.  C ha n ge fr o m bas eli ne t o Da y 8 5  i n t he Vas c ulitis Da ma ge I n d e x ( V DI); 
1 2.  C ha n ge fr o m bas eli ne t o Da y 8 5  i n healt h-relate d q ualit y- of-life as meas ure d b y t h e S h ort 
F or m - 3 6 versi o n 2. 0 ( S F- 3 6 v 2) a n d E ur o Q O L- 5 D-5 L ( E Q - 5 D-5 L);  


C he m o Ce ntr y x, I n c.  
C O N FI D E N TI A L  
Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8 
 
 A me n d me nt 4. 0 
                                                                   Pa ge 2 4 of 1 1 3                                   1 8 Se pte m ber 2 0 1 5 
 
Ot her e n d p oi nts i ncl u de:  
1.  T otal c u m ulati ve st u d y -s u p plie d pre d nis o ne d ose a n d d urati o n of d osi n g d uri n g t he 8 4-da y 
treat me nt peri o d; 
2.  T otal c u m ulati ve s yste mi c c ortic oster oi d d os e (a n y use) a n d d urati o n of d osi n g d uri n g t he 
8 4- da y d osi n g p eri o d; 
3.  T otal c u m ulati ve c ycl o p h os p ha mi de or rit u xi ma b d ose a n d d urati o n of d osi n g d uri n g t he 8 4-
da y d osi n g p eri o d;  
4.  Perce nt c h a n ge fr o m bas eli ne i n hs C R P,  
5.  Perce nt c h a n ge fr o m bas eli ne i n A N C A (a nti - P R 3 a n d a nti-M P O) at D a y 8 5,  
6.  Pr o p orti o n of patie nts bec o mi n g A N C A n e gati ve at Da y 8 5, a n d  
7.  C ha n ge a n d perce nt c ha n ge fr o m baseli ne i n plas ma a n d uri ne bi o mar k ers. 
T he 8 4- da y f oll o w- u p peri o d res ults f or t he e n d p oi nts liste d a b o ve will als o be s u m marize d.   
S u m mar y statistics will be  calc ul ate d f or eac h of t he effi cac y e n d p oi nts. F or cate g orical 
e n d p oi nts, n u m bers a n d perce nt a ges will be calc ulate d. F or c o nti n u o us varia bles, n u m bers, 
mea ns, me dia ns, ra n ges, a n d sta n dar d d e viati o ns will be calc ulate d. Ge o m etric mea ns will be 
calc ulate d  f or uri nar y A C R, uri nar y R B C c o u nt, uri nar y M C P- 1:creati ni ne, a n d hs C R P. S hift 
ta bles will be ge ner ate d f or uri nar y para met ers s uc h as he mat uria a n d al b u mi n uria. Res ults will 
be prese nt e d se paratel y f or eac h st e p, a n d c o m bi n e d f or t hree gr o u ps: ( 1) t he pl ace b o s u bjects 
(sta n dar d of car e gr o u p) acr oss all t hree ste ps, ( 2) t he C C X 1 6 8 s u bjects recei vi n g a l o w d os e 
st u d y-s u p plie d pr e d nis o ne d ose fr o m Ste ps 1 a n d 3, a n d ( 3) t he C C X 1 6 8 s u bjects recei vi n g n o 
st u d y-s u p plie d pr e d nis o ne fr o m Ste ps 2 a n d 3. Res ults will als o be prese nte d b y strat u m f or 
eac h of t h e t hree stratificati o n fact ors, ne wl y di a g n ose d vs. rela pse d p atie nts, rit u xi ma b vs. 
c ycl o p h os p ha mi de use, a n d P R 3 vs. M P O p ositi ve A N C A.   
T he mai n effi cac y h y p ot hesis i n t his st u d y is t hat C C X 1 6 8 treat me nt will i n d uce treat me nt 
res p o nse w hile b ei n g c ortic oster oi d-s pari n g, i.e., A A V disease acti vit y will be c o ntr olle d i n t he 
maj orit y of s u bjects recei vi n g C C X 1 6 8 wit h o ut t he nee d f or res c ue I V or oral c ortic oster oi ds.  
T he pri mar y efficac y e n d p oi nt is t he pr o p orti o n of s u bjects ac hie vi n g disease res p o nse at Da y 
8 5 defi ne d as B V A S perce nt re d ucti o n fr o m b aseli ne of at least 5 0 % pl us n o w orse ni n g i n a n y 
b o d y s yste m c o m p o ne nt.  T he pr o p orti o n of s u bjects ac hie vi n g dise ase res p o nse a n d t he 1-
si de d 9 5 % c o nfi de n ce i nt erval  f or t he differe nce i n pr o p orti o n ( C C X 1 6 8 mi n us S O C) will be 
esti mate d f or t he c o m p aris o n bet wee n eac h C C X 1 6 8 gr o u p a n d t he S O C gr o u p. If t he l o wer 
b o u n d of t he 9 5 % c o nfi de nce i nter v al is great er t h a n - 0. 2 0, t he res pecti ve C C X 1 6 8 gr o u p will 
be c o nsi der e d n ot i nferi or t o t he S O C gr o u p. If t h e l o wer b o u n d of t h e c o nfi de nce i nter val is 
gr eater t ha n  0. 0, t he r es p ecti ve C C X 1 6 8 gr o u p will be c o nsi dere d s u peri or t o t he S O C gr o u p i n 
ac hie vi n g t he diseas e res p o nse .  
C o nti n u o us varia bles will be a nal yz e d usi n g mi x e d effects m o del f or re pe ate d me as ures 
( M M R M) wit h treat me nt gr o u p , visit, tre at me nt-b y -visit i nter acti o n,  a n d r a n d o miz ati o n 
strata ( ne wl y dia g n os e d A A V or r ela pse d A A V, rit u xi ma b or c ycl o p h os p h a mi de, P R 3 or M P O 


C he m o Ce ntr y x, I n c.  
C O N FI D E N TI A L  
Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8 
 
 A me n d me nt 4. 0 
                                                                   Pa ge 2 5 of 1 1 3                                   1 8 Se pte m ber 2 0 1 5 
 
A N C A)  as f act ors , a n d b aseli ne as c o variate.  S u bjects will be c o nsi dere d as re pe ate d 
me as ure u nits o ver visits. P oi nt esti mates a n d c orres p o n di n g 9 5 % c o nfi d e nce i nter vals will be 
esti mate d f or t he diff er e n ce bet w ee n eac h C C X 1 6 8 gr o u p a n d t he place b o gr o u p usi n g si m ple 
c o ntr ast f r o m t he m o del . A d diti o n all y, a n al ysis of c o v ari a nce ( A N C O V A) wit h t he s a me 
f act ors a n d c o v ari ates will be a p plie d f or t he bet w ee n gr o u p c o m p aris o n at e ac h visit. 
C o nti n u o us varia bles i ncl u de c ha n ge a n d/ or perce nt c ha n ge fr o m baseli ne i n B V A S, e G F R, 
hs C R P, uri nar y A C R, uri nar y M C P - 1:creati ni ne rati o, V DI, S F- 3 6 v 2 a n d E Q- 5 D- 5 L (t otal a n d 
s u bsc ores), p h ar mac o d y n a mics mar kers, a n d A N C A (a nti - P R 3 a n d a nti-M P O). Data t hat are n ot 
n or mall y distri b ute d, e. g., uri nar y A C R will be l o g- tra nsf or me d bef ore a n al ysis. 
Cate g orical varia bles wi ll be a nal yz e d usi n g t he C M H  test. Ot her t ha n t he pri mar y efficac y 
e n d p oi nt, t hese i ncl u de t he pr o p orti o n of s u bjects ac hie vi n g re n al res p o ns e at Da y 8 5. Re nal 
res p o nse is defi n e d as a n i m pr o ve me nt fr o m baseli ne i n para met ers of re nal vasc ulitis, base d o n 
a n i ncrease i n e G F R, a d ecrease fr o m baseli ne i n he mat uria, a n d a decreas e fr o m baseli ne i n 
uri nar y al b u mi n uria. Ot h er cat e g orical para m eters, i.e., pr o p orti o n of s u bjects re q uiri n g res c ue 
I V or or al ster oi ds, ac hie vi n g diseas e re missi o n, uri nar y R B Cs ≤5/ h pf, a n d uri nar y R B Cs 
< 3 0/ h pf will be a nal yz e d si milarl y.    
S u bjects recei vi n g r esc ue ster oi ds after Da y 1 b ut bef ore Da y 8 5 + 7 d a ys (i.e. Da y 9 2) will be 
c o nsi dere d as treat me nt f ail ures.  
All statistical testi n g will be o ne-si de d a n d wit h t he t y p e I err o r rate at α = 0. 0 5. I n t he c o nte xt of 
t his bei n g a P hase 2 tri al, n o a dj ust me nt f or m ulti plicit y will be ma de. If t he t w o C C X 1 6 8 
gr o u ps s h o w e d a si milar res p o nse, t he t w o C C X 1 6 8 gr o u ps ma y b e c o m bi ne d f or testi n g a gai nst 
t he c o ntr ol gr o u p. 
T he mai n effi cac y a nal ysis will be i n t he i nte nt -t o-treat p o p ulati o n. T his i ncl u des all s u bjects 
w h o ha ve si g ne d i nf or me d c o nse nt t o partici pate i n t he st u d y, w h o w ere ra n d o mize d i n t his 
st u d y, ha v e recei ve d at least o ne d ose of st u d y dr u g, a n d h a ve at least o ne p ost baseline B V A S 
assess me nt . 
A sa m ple size of 1 2 s u bjects ( 8 acti ve a n d 4 place b o) per ste p i n t he first 2 ste ps was c h ose n 
base d o n feasi bilit y. A sa m ple size of 3 6 s u bjects i n Ste p 3, 1 2 i n eac h of t h e C C X 1 6 8 gr o u ps 
a n d 1 2 i n t he place b o, will pr o vi de a t otal of a p pr o xi matel y 6 0  s u bjects acr oss all t hree ste ps, 
a n d ~ 2 0 s u bjects i n eac h of t he treat me nt gr o u ps. Ass u mi n g a c o ntr ol gr o u p B V A S res p o ns e of 
4 4 % at Da y 8 5 a n d a C C X 1 6 8 gr o u p res p o nse of 8 6 %, a sa m ple size of 2 0 i n eac h gr o u p will 
pr o vi de a p pr o xi matel y 9 0 % p o wer f or t he pri mar y efficac y a n al ysis .  
P h ar m ac o ki netic a n alysis 
Plas ma sa m ples will be c ollecte d at Baseli ne ( Da y 1) a n d Da ys 8, 1 5, 2 2, 2 9, 4 3, 5 7, 7 1,  a n d 8 5 
t o deter mi ne t he P K pr ofile of C C X 1 6 8 (a n d p ote ntial meta b olites) . I n di vi d ual plas ma 
c o nce nt r ati o ns of C C X 1 6 8 (a n d p ote ntial meta b olites)  will be liste d, pl otte d, a n d s u m marize d 
descri pti vel y a n d gr a p hicall y. T he f oll o wi n g p ara meters will be deter mi ne d, w here p ossi ble: 
C ma x  
Ma xi m u m plas ma c o nce ntrati o n  
T ma x  
Ti me of ma xi m u m plas ma c o nce ntrati o n  
A U C 0- 6 Area u n d er t he plas ma c o nce ntrati o n -ti me c ur ve fr o m Ti me 0 t o H o ur 6 o n Da y 1 


C he m o Ce ntr y x, I n c.  
C O N FI D E N TI A L  
Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8 
 
 A me n d me nt 4. 0 
                                                                   Pa ge 2 6 of 1 1 3                                   1 8 Se pte m ber 2 0 1 5 
 
C mi n  
Tr o u g h l e vel plas ma c o n ce ntrati o ns at p ost -D a y 1 visits       
It is of i nter est t o e val uat e w het her t h e P K pr ofile of s u bjects wit h A N C A-ass ociate d 
vasc ulitis is si mil ar t o t he pr ofile i n healt h y v ol u nteers. T he relati o ns hi p bet wee n P K 
para meters a n d r e nal f u n cti o n base d o n e G F R will be e val uat e d. T he dat a ma y als o be 
use d t o e val uate t he P K/ P D relati o ns hi p of C C X 1 6 8 treat me nt. T o t his e n d, t he c ha n ge 
a n d/ or per ce nt c h a n ge  fr o m baseli ne i n B V A S, e G F R, uri nar y A C R, uri nar y M C P-
1:creati ni ne r ati o , ser u m hs C R P, he mat uria, a n d ot her bi o mar kers m a y be use d as P D 
mar kers.  
Uri nar y c o n ce ntr ati o ns of C C X 1 6 8 a n d p ossi ble meta b olites i n a 6- h o ur uri ne sa m ple 
c ollecte d o n D a y 1 will als o be meas ure d. Res ults will be liste d a n d s u m marize d 
descri pti vel y.  
Plas ma  c o nce ntr ati o ns of pre d nis ol o ne, c ycl o p h os p ha mi de a n d its meta b olites , a n d 
rit u xi ma b ma y als o b e meas ure d i n t he P K plas ma sa m ples t o e val uate p ote ntial dr u g -
dr u g i nteracti o n. 




C he m o Ce ntr y x, I n c.                                                             C O N FI D E N TI A L                                                            Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8 
 
                                                                                  A me n d me nt 4. 0 
 
                                                                  Pa ge 2 8 of 1 1 3                                                                    1 8 Se pte m ber 2 0 1 5 
TI M E A N D E V E N T S T A B L E 
 
Scree ni n g
1 
St u d y D a y
2 
 
-1 4 t o -1  
1
3 
2   8  
1 5  2 2  
2 9  
4 3  5 7   7 1  8 5  
9 9  
1 1 3  1 4 1  
1 6 9  
I nf or med C o nse nt  
X  
 
 
 
 
 
 
 
 
 
 
 
 
 
 
De m o gra p hics, M e dical Hist or y, Pri or 
Me dicati o ns  
X  
 
 
 
 
 
 
 
 
 
 
 
 
 
 
P h ysical E x a mi nati o n
4  
X  
X 5 
 
 
X  
 
X  
 
X  
 
X  
 
X  
 
X  
B o d y S yste m Re vie w  
 
 
X  
X  
 
X  
 
X  
 
X  
 
X  
 
X  
 
Vital Si g ns
6  
X  
X 5  
X  
X  
X  
X  
X  
X  
X  
X  
X  
X  
X  
X  
X  
1 2 -lea d E C G 
X  
 
 
 
 
 
X  
 
 
 
 
 
 
 
 
C hest X ra ys
7 
X  
 
 
 
 
 
( X) 
 
 
 
( X) 
 
( X) 
 
( X) 
HI V, H B V, H C V T esti n g  
X  
 
 
 
 
 
 
 
 
 
 
 
 
 
 
A N A, a nti -G B M a nti b o dies, C 3, C 4, 
I g G, I g M, a n d I g A
8 
X  
 
 
 
 
 
 
 
 
 
 
 
 
 
 
P T a n d a P T T  
X  
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Ser u m C he mistr y, He m at ol o g y  
X  
X 5 
X  
X  
X  
 
X  
X  
 
X  
X  
X  
 
X  
X  
W B C c o u nt (l ocal la b)
9 
 
X 5 
 
 
X  
 
X  
 
X  
 
X  
X  
( X) 
 
 
Ser u m Creati ni ne ( w h e n f ull 
C he mistr y n ot d o ne) 
 
 
 
 
 
 
 
 
X  
 
 
 
X  
 
 
Uri nal ysis
1 0 
X  
X 5 
X  
X  
X  
X  
X  
X  
X  
X  
X  
X  
X  
X  
X  
Uri ne al b u mi n, M C P -1 a n d creati ni ne 
assa ys  
 
X 5 
 
X  
X  
 
X  
 
X  
 
X  
 
X  
 
X  
A N C A meas ur e me nt
1 1 
X  
X 5 
 
 
 
 
X  
 
 
 
X  
 
X  
 
X  
Re nal bi o ps y ( o pti o nal pr oce d ure )  
X  
 
 
 
 
 
 
 
 
 
X
1 2 
 
 
 
 
Ra n d o mizati o n  
 
X 5 
 
 
 
 
 
 
 
 
 
 
 
 
 
C C X 1 6 8 or Place b o Dis pe nsi n g  
 
X  
 
X  
X  
X  
X  
X  
X  
X  
 
 
 
 
 


C he m o Ce ntr y x, I n c.                                                             C O N FI D E N TI A L                                                            Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8 
 
                                                                                  A me n d me nt 4. 0 
 
                                                                  Pa ge 2 9 of 1 1 3                                                                    1 8 Se pte m ber 2 0 1 5 
 
Scree ni n g
1 
St u d y D a y
2 
 
-1 4 t o -1  
1
3 
2   8  
1 5  2 2  
2 9  
4 3  5 7   7 1  8 5  
9 9  
1 1 3  1 4 1  
1 6 9  
C C X 1 6 8 or Place b o Acc o u nta bilit y  
 
 
 
X  
X  
X  
X  
X  
X  
X  
X  
 
 
 
 
Pre d nis o ne or Place b o Di s pe nsi n g 
 
X  
 
X  
X  
 
X  
X  
X  
 
X  
X  
X  
 
 
Pre d nis o ne or Place b o A cc o u nta bilit y  
 
 
 
X  
X  
 
X  
X  
X  
 
X  
X  
X  
X  
( X)
1 3 
C ycl o p h os p ha mi de I V d ose
1 4 
 
X  
 
 
X  
 
X  
 
X  
 
X  
 
 
 
 
Azat hi o pri ne d osi n g
1 5 
 
 
 
 
 
 
 
 
 
 
 
X → 
→ 
→ 
→ 
Rit u xi ma b I V d ose
1 6 
 
X  
 
X  
X  
X  
 
 
 
 
 
 
 
 
 
B V A S  
X  
X 5 
 
 
 
 
X  
 
 
 
X  
 
X  
 
X  
V DI  
 
X 5 
 
 
 
 
 
 
 
 
X  
 
 
 
X  
S F -3 6 v 2 a n d E Q -5 D -5 L  
 
X 5 
 
 
 
 
X  
 
 
 
X  
 
 
 
X  
hs C R P  
 
X 5 
 
X  
X  
 
X  
 
X  
 
X  
 
X  
 
X  
P K Plas ma Sa m ple C ollecti o n  
 
X 5 ,1 7 
 
X  
X  
X  
X  
X  
X  
X  
X  
 
 
 
 
P D Plas ma Sa m ple C ollecti o n  
 
X 5 
 
 
 
 
X  
 
 
 
X  
 
 
 
X  
Sali va sa m ple c ollecti o n  
 
X  
 
 
 
 
 
 
 
 
 
 
 
 
 
Uri ne sa m ple c ollecti o n f or C C X 1 6 8 
a nal ysis  
 
X
1 8 
 
 
 
 
 
 
 
 
 
 
 
 
 
C o nc o mita nt Me dicati o ns  
X  
X  
X  
X  
X  
X  
X  
X  
X  
X  
X  
X  
X  
X  
X  
I nstr u ct s u bjects w h o are o n 
mai nte na nce c ort ic oster oi ds  
( ≤ 1 0 m g pr e d nis o ne e q ui vale nt) at 
scree ni n g r e gar di n g t a peri n g o ver ≤ 6 
wee ks  
X  
 
 
 
 
 
 
 
 
 
 
 
 
 
 
A d verse E v e nt Assess me nt  
 
X  
X  
X  
X  
X  
X  
X  
X  
X  
X  
X  
X  
X  
X  
1 Scree ni n g m ust occ ur e x pe diti o usl y ( n ot t o  e xcee d 1 4 da ys) i n or der n ot t o dela y start of tre at me nt; scree ni n g la bs will be d o ne at t he l ocal la b orat or y t o 
e x pe dite eli gi bilit y assess me nt; res ults fr o m a re nal bi o ps y, if perf or me d wit hi n 4 wee ks of Da y 1 will be c ollecte d o n t he Hist ol o g y F or m pr ovi de d . La b orat or y 
res ults fr o m t he l ocal la b orat ories o btai ne d wit hi n 7 2 h o urs of scree ni n g are acce pta ble i n or der t o a v oi d u n necessar y bl o o d dra ws.  
2 Visit Da ys 1, 2, 8, a n d 1 5 m ust occ ur o n t he sc he d ule d st u d y da ys. Visit Da ys 2 2, 2 9, 4 3, 5 7, 7 1, a n d 8 5 ma y occ ur wit hi n a +/ - 2-da y wi n d o w of t he sc he d ule d 
visit. Visit Da ys 9 9, 1 1 3, 1 4 1, a n d 1 6 9 ma y occ ur wit hi n a +/ - 4-da y wi n d o w of t he sc he d ul e d visit.  
3 A s u bj ect c o ul d be ke pt o ver ni g ht i n t he h os pital o n Da y 1, if necessar y. T his h os pital sta y w o ul d n ot be c o nsi dere d a seri o u s a d verse e ve nt, u nless ot her S A E 
criteria are met.  


C he m o Ce ntr y x, I n c.                                                             C O N FI D E N TI A L                                                            Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8 
 
                                                                                  A me n d me nt 4. 0 
 
                                                                  Pa ge 3 0 of 1 1 3                                                                    1 8 Se pte m ber 2 0 1 5 
4 P h ysical e xa mi nati o n will i ncl u de b o d y wei g ht meas ure me nt; Hei g ht will o nl y be meas ure d at Scree ni n g.  
5 T hese pr oce d ures m ust be d o ne B E F O R E ta ki n g t he first d os e of st u d y me dicati o n.  
6 Assess me nt of heart rate, b o d y te m perat ure, a n d bl o o d press ure (s u pi ne, after at least 3 mi n utes of rest)  
7 C hest X ra ys will be ac q uire d  a n d e xa mi ne d at Scree ni n g t o e xcl u de t he prese nce of T B a n d f or baseli ne B V A S a n d V DI. C hest X ra ys at s u bse q ue nt st u d y 
visits will O N L Y be ac q uire d if i n t he cli nical o pi ni o n of t he Pri nci pal I n vesti gat or t here is p ul m o nar y disease i n v ol ve me nt t hat nee ds t o be assesse d f or safet y, 
B V A S, or V DI.  
8 T hese tests d o n ot nee d t o be perf or me d if res ults are a vaila ble fr o m tests d o ne wit hi n t he past 1 2 m o nt hs pri or t o t he Scree ni n g visit.  
9 F or s u bj ects recei vi n g c ycl o p h os p ha mi de,  a bl o o d sa m ple m ust be ta ke n earl y d uri n g t he visit f or W B C c o u nt at t he l ocal la b orat or y; t he W B C c o u nt is 
necessar y f or c ycl o p h os p ha mi de d ose decisi o ns o n t hese da y s. F or s u bjects recei vi n g az at hi o pri ne, l oc al W B C c o u nt will be d o ne at t he D a y 1 1 3 visit.  
1 0 S u bj ects will be as ke d t o v oi d t heir bla d ders c o m pletel y, a n d a re prese ntati ve clea n catc h, mi dstrea m  uri ne sa m ple will b e c ollecte d. D uri n g scree ni n g, t he l ocal 
la b orat or y will perf or m a uri nal ysis f or he mat uria a n d pr otei n uria f or eli gi bilit y assess me nt. F or t he rest of t he visits, a uri ne sa m ple will be se nt t o t he ce ntral 
la b orat or y f or uri nal ysis i ncl u di n g p H, s pecific gra vit y, gl uc ose, nitrite, ket o nes, bilir u bi n, ur o bili n o ge n, R B Cs, a n d W B Cs. Uri nar y A C R a n d M C P -
1:creati ni ne will be meas ure d b y t he ce ntral la b orat or y at st u d y visits i n dicate d i n t he ta ble.  
1 1 At scree ni n g, a n i n direct i m m u n ofl u oresce nce test f or P -A N C A a n d C -A N C A, as well as E LI S A tests f or a nti -pr otei nas e -3 ( P R 3) a n d a nti -m yel o per o xi dase 
( M P O) will be perf or me d f or st u d y eli gi bilit y; f or t he rest of t he st u d y visits, o nl y E LI S A t ests f or a nti - P R 3 a n d a nti -M P O will be perf or me d.  
1 2 If p ossi ble, re nal bi o ps y s h o ul d be o btai ne d fr o m s u bj ects wit h a pre-st u d y bi o ps y. 
1 3 T his acc o u nta bilit y is o nl y nec essar y f or s u bj ects w h o disc o nti n ue bet wee n Da y 8 5 a n d 1 6 9, a n d c o me f or a n earl y ter mi nati o n visit.  
1 4 A p plica ble o nl y t o c ycl o p h os p ha mi de strat u m. C ycl o p h os p ha mi de d oses gi ve n fr o m Da y 1 t hr o u g h 5 7 m ust be gi ve n acc or di n g t o directi o ns pr o vi de d i n t he 
pr ot oc ol . T he date a n d start a n d e n d ti me of t he c ycl o p h os p ha mi de d ose will be rec or de d. 
1 5 Azat hi o pri ne m ust be ta k e n fr o m Da y 9 9  t hr o u g h Da y 1 6 8 b y all s u bj ects i n t he c ycl o p h os p ha mi de b ut n ot t he rit u xi ma b  strat u m. 
1 6 A p plica ble o nl y t o t he rit u xi ma b strat u m. A rit u xi ma b d osi n g re gi me n of 3 7 5 m g / m2 I V o n Da ys 1, 8, 1 5, a n d 2 2 s h o ul d be use d. 
1 7 P K bl o o d sa m ple will be c ollecte d pri or t o t he m or ni n g d ose o n Da y 1 a n d at 0. 5, 1, 2, 3, 4, a n d 6 h o urs f oll o wi n g d osi n g.  
1 8 S u bj ects will v oi d pri or t o t he m or ni n g d ose of C C X 1 6 8/ place b o o n t his da y, a n d t he n A L L uri ne will be c ollecte d o ver t he fir st 6 h o urs f oll o wi n g 
C C X 1 6 8/ place b o d osi n g. T he 6 -h o ur uri ne v ol u me will be  meas ure d a n d a re prese ntati ve s a m ple will be se nt t o meas ure C C X 1 6 8 c o nce ntrati o n i n t he uri ne.  


C he m o Ce ntr y x, I n c.                            C O N FI D E N TI A L                                Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8                                                                                                                    A me n d me nt 4. 0 
                                                                   Pa ge 3 1 of 1 1 3                               1 8 Se pte m ber 2 0 1 5 
LI S T O F A B B R E VI A TI O N S A N D A C R O N Y M S  
A A V  
a nti-ne utr o p hil c yt o plas mic a nti b o d y ass o ciate d vasc ulitis  
A A R V  
a nti- ne utr o p hil c yt o plas mic a nti b o d y ass o ciate d re nal vasc ulitis 
A C R  
al b u mi n:creati ni ne rati o  
A E  
a d verse e ve nt  
A L T  
ala ni ne a mi n otra nsf eras e (als o calle d S G P T)  
A N A  
a nti- n uclear a nti b o dies 
A N C A  
a nti- ne utr o p hil c yt o plas mic a nti b o dies 
A PI  
acti ve p har m ace utical i n gre die nt  
a P T T 
acti vate d partial t hr o m b o plasti n ti me  
A S T  
as partate a mi n otra nsferas e (als o calle d S G O T)  
A U C 0- 6  
area u n d er t he c ur ve fr o m h o ur 0 t o 6 
B L Q  
bel o w li mit of q ua ntificati o n  
B U N   
bl o o d urea nitr o ge n 
B V A S  
Bir mi n g h a m Vasc ulitis Acti vit y Sc ore v ersi o n 3  
C 3  
c o m ple me nt 3  
C 4  
c o m ple me nt 4  
C 3a 
c o m ple me nt 3a 
C 4a 
c o m ple me nt 4a 
C 5a 
c o m ple me nt 5a 
C 5a R  
c o m ple me nt 5a rece pt or  
C 5 b 9  
c o m ple me nt 5 b 9 
C A  
c o m pete nt a ut h orit y 
C- A N C A  
c yt o plas mic -a nti- ne utr o p hil c yt o plas mic a nti b o d y 
c G M P  
c urre nt g o o d cli nical practice  
C ma x  
ma xi m u m ( plas ma) c o nce ntrati o n  
C P K   
creati ni ne p h os p h o ki nas e 
C R A  
Cli nical Researc h Ass o ciate (als o k n o w n as t he St u d y M o nit or)  
C R F  
case r e p ort f or m  
C R O  
c o ntract r esearc h or ga nizati o n  
D M C  
data m o nit ori n g c o m mittee  
E C  
et hics c o m mittee 
E C 5 0  
5 0 % eff ecti ve c o n ce ntrati o n  
E C G  
electr ocar di o gr a m  
e G F R  
esti mate d gl o mer ular filtrati o n rate  
E Q - 5 D-5 L   
E ur o Q ol- 5 D-5 L  
F A C S  
fl u oresce n ce acti vate d cell s orti n g 
F D A   
F o o d a n d Dr u g A d mi nistrati o n 
F LI P R  
Fl u or o metric I m a gi n g Pl ate Rea der  
g 
gr a m  
G B M   
gl o mer ular base m e nt me m bra ne 
G C P  
g o o d cli nical practice  
G G T   
ga m ma -gl uta m yl tr a ns pe pti das e 
G P A  
gr a n ul o mat osis wit h p ol ya n giitis ( We ge ner’s)  
G P C R   
G pr otei n-c o u ple d rece pt or  


C he m o Ce ntr y x, I n c.                            C O N FI D E N TI A L                                Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8                                                                                                                    A me n d me nt 4. 0 
                                                                   Pa ge 3 2 of 1 1 3                               1 8 Se pte m ber 2 0 1 5 
H E D   
h u ma n e q ui vale nt d ose 
H E E N T   
hea d, e yes, ears, n ose, t hr oat 
HI V  
h u ma n i m m u n o deficie nc y vir us 
h pf 
hi g h p o wer fiel d 
I C5 0 
c o nce ntr ati o n t o i n hi bit 5 0 % 
I C H 
I nter nati o nal C o nfere nce o n Har m o nisati o n 
I N R 
I nter nati o nal N or m alize d Rati o 
II F 
I n dir ect i m m u n ofl u oresce nce 
I R B 
I nstit uti o nal Re vie w B oar d 
I VI g  
i ntra ve n o us i m m u n o gl o b uli n 
K 3E D T A  
tri-p otassi u m et h yle n e dia mi ne tetra -acetic aci d  
k g 
kil o gra m 
KI M - 1  
ki d ne y i nj ur y m olec ul e- 1 
L D H  
lactate de h y dr o ge nas e 
M A C  
me m bra ne attac k c o m ple x  
M C H  
mea n cell he m o gl o bi n  
M C H C  
mea n cell he m o gl o bi n c o nce ntrati o n  
M D R D  
M o dificati o n of Diet i n Re nal Diseas e  
Me d D R A  
Me dical Dicti o nar y f or Dr u g Re g ul at or y Affairs  
m g  
milli gra m  
m L  
milliliter  
M P A  
micr osc o pic p ol ya n giitis  
M P O   
m yel o per o xi dase 
N 
n u m ber 
N G A L   
ne utr o p hil gel ati nase-ass ociate d li p ocali n  
N O A E L  
N o o bser ve d a d vers e effect le vel  
P- A N C A  
peri n uclear -a nti-n e utr o p hil c yt o plas mic a nti b o d y  
P C P  
P ne u m ocystis c ari nii  p ne u m o nia 
P D  
p har mac o d y n a mic(s)  
P K  
p har mac o ki n etic(s)  
P R 3  
pr otei nase 3  
P T  
pr ot hr o m bi n ti me 
R B C   
re d bl o o d cell 
S A E  
seri o us a d vers e e v e nt 
S F - 3 6 v 2  
S h ort F or m- 3 6 versi o n 2 
S G P T  
ser u m gl uta mic p yr u vic tra nsa mi nase (als o calle d A L T) 
S G O T  
ser u m gl uta mic o x al oacetic tra nsa mi nase (als o calle d as A S T) 
S L E  
s ystemic l u p us er yt h e mat os us 
S O P  
sta n dar d o per ati n g pr oce d ure 
S U S A R  
s us pecte d u ne x pecte d seri o us a d verse reacti o n 
T ma x  
ti me of ma xi m u m ( plas ma) c o nce ntrati o n 
T P M T   
t hi o p uri ne S-met h yltra nsferas e  
V DI  
vasc ulitis da ma ge i n de x  
W B C   
w hite bl o o d cell 
W G  
We ge n er’s gr a n u l o mat osis 


C he m o Ce ntr y x, I n c.                            C O N FI D E N TI A L                                Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8                                                                                                                    A me n d me nt 4. 0 
                                                                   Pa ge 3 3 of 1 1 3                               1 8 Se pte m ber 2 0 1 5 
1.  
I N T R O D U C TI O N 
1. 1.  
B ac k gr o u n d  
T he acti vati o n of t he c o m ple me nt pat h wa y ge n erates bi ol o gicall y acti ve fra g m e nts of  
c o m ple me nt pr otei ns, e. g. C 3a, C 4a a n d C 5a a na p h ylat o xi ns a n d C 5 b- 9 me m bra ne attac k 
c o m ple x es ( M A C), all of w hic h me diate i nfla m mat or y re s p o nses b y i n d uci ng le u k o c yt e 
c he m ota xis, acti vati n g m acr o p ha ges, ne utr o p hils, platelets, mast cells a n d e n d ot helial cells a n d 
b y i ncr easi n g vas c ular p er mea bilit y, c yt ol ysis a n d tiss ue i nj ur y.  
C 5a is o ne of t he m ost p ote nt pr o- i nfla m mat or y m e diat ors of t he c o m ple m e nt s yste m, bei n g at 
least 10 0 ti mes m ore p ote nt t ha n C 3a. T his 1 9 0 k D p ol y pe pti de, al o n g wit h a C 5 b fra g me nt, is 
pr o d uce d b y e nz y matic clea va ge of a C 5 prec urs or d uri n g acti vati o n of a n y of t he 3 c o m ple me nt 
pat h wa ys. C 5a i n d uces e x pressi o n of a d he si o n m olec ules a n d c h e m otactic mi grati o n of 
ne utr o p hils, e osi n o p hils, bas o p hils a n d m o n oc yt es. It als o me di ates i nfla m mat or y reacti o ns b y 
ca usi n g s m o ot h m uscle c o ntracti o n, i ncreasi n g v asc ular per m ea bilit y, i n d u ci n g b as o p hil a n d 
mast cell de gra n ul ati o n a n d  i n d uci n g releas e of l ys os o mal pr oteases a n d o xi dati ve free ra dicals. 
T he a na p h ylactic a n d c he m otactic eff ects of C 5a are me diate d t hr o u g h its i nteracti o n wit h t he 
C 5a rece pt or ( C 5a R), a G pr otei n-c o u ple d rece pt or ( G P C R) e x presse d o n h u ma n ne utr o p hils, 
m o n oc ytes, b as o p hils, e osi n o p hils, re nal gl o mer ul ar tiss ues, a n d l u n g s m o ot h m uscle a n d 
e n d ot helial cells.  
Rece ntl y, s e ver al re p orts ha ve s h o w n t hat a nti -n e utr o p hil c yt o plas mic a nti b o d y ( A N C A) -i n d uce d 
gl o mer ul o ne p hritis i n mice (a m o d el t hat cl osel y reca pit ul ates t he hist ol o gi cal feat ures of h u m a n 
pa uci -i m m u ne necr otizi n g cresce ntic gl o mer ul o ne p hritis i n gra n ul o mat osis wit h p ol ya n giitis 
[ G P A; We ge ner’s] a n d micr osc o pic p ol ya n giitis [ M P A]) is dra maticall y a meli orate d b y ge netic 
deleti o n of eit her C 5 or C 5a R ( Sc hrei ber et al., 2 0 0 9 ). T he de v el o p me nt of s yste mic l u p us 
er yt h e mat os us ( S L E) is ass ociate d wit h t he de p ositi o n of I g G-c o ntai ni n g i m m u ne c o m ple x es i n 
vari o us tiss ues/ or ga ns, wit h t he e ns ui n g acti vati o n of t he c o m ple me nt casca de a n d pr o d ucti o n of 
i nfla m mat or y sti m uli s uc h as C 5a. Gl o mer ular e x pressi o n of C 5a R m R N A a n d pr otei n was 
s h o w n t o c orrel ate p ositi vel y wit h t he d e gree of mesa n gi al h y p ercell ularit y a n d l e vel of ser u m 
creati ni ne i n mesa n gial gl o mer ul o ne p hritis, i ncl u di n g l u p us ne p hritis ( A be et al., 2 0 0 1 ). Rece nt 
st u dies s h o we d t hat C 5a R-deficie nt mice a n d mice treate d wit h a s mall pe pti dic a nti -C 5a R 
a nta g o nist are pr ot ecte d fr o m tiss ue i nj ur y i n d uce d b y i m m u ne c o m ple x f or mati o n. I n a d diti o n, 
use of a C 5 m A b i n a s p o nta ne o us m o use m o del of l u p us- li ke a ut oi m m u ne disease res ulte d i n 
si g nifica nt a meli orati o n of t he c o urse of gl o mer ul o ne p hritis a n d i n mar ke dl y i ncrease d s ur vi val. 
A ge netic versi o n of t he disease ( M R Ll pr mice) is als o atte n uate d si g nifica ntl y w he n t he C 5a R 
rece pt or is delete d fr o m t hat ge netic bac k gr o u n d. 
T he t hera p e utic i n dicati o n  bei n g p urs ue d i nitiall y f or C C X 1 6 8, a p ote nt a n d selecti ve C 5a R 
a nta g o nist, is i n t he treat me nt of A N C A -ass o ciate d vasc ulitis  ( A A V).  
A A V  c urre ntl y is treate d wit h gl uc o c ortic oster oi ds a n d c ycl o p h os p ha mi de, azat hi o pri ne, 
m yc o p h e n olate m ofetil, rit u xi ma b, a n d plas ma e x c ha n ge i n se vere cases. 


C he m o Ce ntr y x, I n c.                            C O N FI D E N TI A L                                Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8                                                                                                                    A me n d me nt 4. 0 
                                                                   Pa ge 3 4 of 1 1 3                               1 8 Se pte m ber 2 0 1 5 
1. 2.  
St u d y Dr u g De vel o p me nt  
1. 2. 1.  
N o n - Cli nic al P h ar m ac ol o g y 
1. 2. 1. 1.  
I n- Vitr o Effic ac y a n d Selecti vit y f or C 5 a R  
C C X 1 6 8 is a p ote nt a nta g o nist of t he h u m a n C 5a rece pt or ( h C 5a R). As meas ure d i n vitr o wit h a 
m yel oi d h u ma n cell li ne, C C X 1 6 8 f u ncti o nal l y i n hi bits C 5a-me diate d c he m ota xis wit h a p ote nc y 
(I C5 0) of 0. 9 2 n M. A d diti o nall y, C C X 1 6 8 dis places 1 2 5I- C 5a fr o m h C 5a R wit h a p ote nc y (I C5 0) 
of 0. 6 5 n M. W he n teste d o n fres hl y is olate d h u ma n ne utr o p hils, C C X 1 6 8 i n hi bits t he C 5a-
me diate d i ncrease i n c yt o plas mic calci u m le vels wit h a p ote nc y (I C 5 0) of 0. 2 n M.  
C C X 1 6 8 has bee n e v al uate d f or its a bilit y t o i n hi bit t he C 5a- me diate d c he m ota xis of ne utr o p hils 
i n fres hly is olat e d h u ma n w h ole bl o o d. C C X 1 6 8 pr o d uce d 5 0 % i n hi biti o n (I C 5 0) of C 5a-
me diati o n ne utr o p hil mi grati o n i n t his assa y at a c o nce ntrati o n of 1. 7 n M; 9 0 % i n hi biti o n ( A 1 0 
val ue) was d eter mi ne d i n h u ma n w h ole bl o o d at a C C X 1 6 8 c o nce ntrati o n of 1 5. 4 n M. C C X 1 6 8 
als o i n hi bits C 5a R i n c y n o m ol g us m o n ke ys a n d ha msters wit h p ote ncies si milar t o t hat o bser ve d 
wit h h u ma n w h ole bl o o d. H o we ver, C C X 1 6 8 p ossesses m o derat e p ote nc y f or ra b bit C 5a R (I C 5 0 
~ 1. 4 µ M) a n d lac ks affi nit y f or m o use, rat or d o g C 5a R (I C 5 0 > 1 0 µ M). 
C C X 1 6 8 dis pla ys greater t ha n 1 0, 0 0 0- f ol d selecti vit y f or h C 5a R relati ve t o ot her c he m otactic 
rece pt ors, i ncl u di n g C C R 1, C C R 2, C C R 4, C C R 5, C C R 6, C C R 7, C C R 8, C C R 9, C C R 1 0, C C R 1 2, 
C X C R 1, C X C R 2, C X C R 3, C X C R 4, C X C R 6, C X C R 7, C 5 L 2, C 3a R, C he m R 2 3, G P R 1 a n d 
F P R L 1. C C X 1 6 8 has bee n f urt her e v al uate d a gai nst a pa nel of 5 5 u nrelate d rece pt ors a n d 
me m bra ne - ass ociate d pr otei ns. Wea k le vels of acti vit y ( > 1, 0 0 0-f ol d s electi vit y relati ve t o 
h C 5a R) were n ot e d a gai nst t he site 2 s o di u m c ha n nel ( 5 9 % i n hi biti o n wit h 1 0 µ M C C X 1 6 8 ). 
1. 2. 1. 2.  
Effic ac y M o dels  
C C X 1 6 8 has bee n e v al uate d i n viv o utilizi n g m o dels t hat are rel e va nt t o t he i nte n de d t hera p e utic 
use i n h u ma ns. W he n C 5a is ge n er ate d l ocall y i n t he bl o o dstrea m, C 5a R- beari n g l e u k oc ytes i n 
t he vici nit y i m me diatel y u pre g ulate a d h esi o n m olec ules a n d a d here t o t he i n ner face of t h e bl o o d 
vessel. If C 5a is i ntr o d uce d s yste micall y b y i ntra v e n o us i njecti o n, le u k oc yt e a d here nce occ urs 
i m me diatel y t hr o u g h o ut t he vasc ulat ure a n d, as a res ult, t he n u m ber of le u k oc yt es still fl o wi n g i n 
t he bl o o dstrea m dr o ps tra nsie ntl y b y a s u bsta ntial a m o u nt. I n ge neral, e val u ati o n of C 5a R 
a nta g o nists i n a ni mal m o dels p oses a c h alle n ge beca use C 5a R a nta g o nists t hat are p ote nt f or 
h u ma n C 5a R, i ncl u di n g C C X 1 6 8, are less p ote nt f or C 5a R ort h ol o gs i n m ost ot her m o del s pecies 
(s uc h as mice, rats a n d r a b bits). F or t his reas o n, C C X 1 6 8 has bee n e v al uate d i n C 5a-i n d uce d 
le u k o pe nia m o dels usi n g tra ns ge nic mice i n w hi c h t he m o use C 5a R ge n e has bee n re pl ace d wit h 
t he h u ma n C 5a R ge ne a n d n o n- h u ma n pri mates. 
C he m o Ce ntr y x ge n er ate d a h u ma n C 5a R k n oc k-i n ( h C 5a R KI) m o use strai n i n w hic h t he m o use 
C 5a R ge n e has bee n re pl ace d wit h t he h u ma n C 5a R ge ne. T he i n nate i m m u ne cells of t hese mice 
res p o n d n or mall y t o C 5a, i n a ma n ner hi g hl y se nsiti ve t o C C X 1 6 8. I n vitr o, C C X 1 6 8 bl oc ks 
h C 5a- me d iate d c h e m ota xis of le u k oc yt es fres hl y is olate d fr o m t hese h C 5a R KI mice wit h hi g h 
p ote nc y (I C 5 0 = 0. 5 n M i n 1 0 0 % m o use plas ma). T his val ue is nearl y i de ntical t o t he p ote nc y 
( 1. 7 n M) e x hi bite d b y C C X 1 6 8 i n its i n hi biti o n of ne utr o p hil mi grati o n t o h C 5a i n w h ole h u ma n 
bl o o d, i n dicati n g t hat t he h C 5a R KI mice are s uita ble f or p har m ac o d y na mi c e val uati o n of 
C C X 1 6 8. I n t he h u ma n C 5a R k n oc k-i n mice, a n i ntra ve n o us d ose of 2 0 µ g/ k g h C 5a r o b ustl y 


C he m o Ce ntr y x, I n c.                            C O N FI D E N TI A L                                Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8                                                                                                                    A me n d me nt 4. 0 
                                                                   Pa ge 3 5 of 1 1 3                               1 8 Se pte m ber 2 0 1 5 
i n d uces t his le u k o pe nia wit hi n o ne mi n ute after i njecti o n. Pretreat me nt of t he mice wit h a n oral 
d ose of 0. 3 m g/ k g C C X 1 6 8, w hic h res ulte d i n a pl as ma c o nce ntrati o n of a p pr o xi matel y 7 5 n M at 
6 0 mi n. p ost- d ose, al m ost c o m pletel y bl oc k e d t he C 5a-i n d uce d le u k o p e nia. A d ose of 
0. 0 3 m g/ k g C C X 1 6 8, pr o d uci n g a plas ma c o n ce ntrati o n of 1 5 n M, res ulte d i n a 5 0 % re d u cti o n i n 
t he C 5a-i n d uce d le u k o p e nic res p o nse. 
I n c y n o m ol g us m o n ke ys, it was deter mi ne d t hat a n i ntra ve n o us h C 5a d ose of 1 0 µ g/ k g r o b ustl y 
i n d uces a dr o p i n ne utr o p hils ( ne utr o pe nia) wit hi n o ne mi n ute. Pre-treat me nt of t he cy n o m ol g us 
m o n ke ys wit h a 3 0 m g/ k g or al d ose of C C X 1 6 8 c o m pletel y bl oc k e d t he C 5a-i n d uce d 
ne utr o pe nia. T his d ose of C C X 1 6 8 res ulte d i n a plas ma c o nce ntrati o n of a p pr o xi matel y 2 3 0 n M 
at t he ti me of h C 5a a d mi nistrati o n. A d ose of 3 m g/ k g res ulte d i n greater t ha n 5 0 % re d u cti o n of 
t he h C 5a res p o nse, a n eff ect t hat was ass o ciate d wit h a C C X 1 6 8 plas ma c o nce ntrati o n of 
a p pr o xi matel y 3 8 n M. 
T he effi cac y of C C X 1 6 8 i n a m o use m o del of A N C A- ass ociate d gl o mer ul o ne p hritis was 
e val uate d i n or d er t o assess t he cli nical p ot e ntial of C C X 1 6 8 i n t he treat me nt of A N C A-
ass ociate d vas c ulitis. I n t hese st u dies, i ntra ve n o us i njecti o n of m o use a nti- m yel o per o xi dase (a nti-
M P O) I g G i nt o t he h u ma n C 5a R k n oc k-i n mice ca use d gl o mer ul o ne p hritis i n a ma n ner 
mi mic ki n g A N C A diseas e i n h u ma ns. At dail y oral d oses of 3 0 m g/ k g C C X 1 6 8, a mar ke d 
i n hi biti o n of a nti- M P O i n d uce d gl o mer ul o ne p hritis was d oc u me nte d hist ol o gicall y, as assesse d 
b y t he n u m ber of necr oti c ( 8. 2 % wit h ve hicl e, 1. 1 % wit h C C X 1 6 8; p < 0. 0 0 0 1) a n d cresce nt-
c o ntai ni n g gl o mer uli ( 2 9. 3 % wit h ve hicle, 3. 3 % wit h C C X 1 6 8; p < 0. 0 0 0 1). T hese res ults w ere 
c o nsiste nt wit h re d uce d pr otei n, le u k oc ytes a n d R B C le vels i n t he uri ne a n d re d uce d s er u m B U N 
a n d creati ni ne i n mice recei vi n g C C X 1 6 8. S o me t hera pe utic b e nefit ( 3 0 % re d ucti o n i n t he 
n u m ber of gl o mer uli wit h cresce nts) was n ot e d at C C X 1 6 8 d oses as l o w as 0. 1 m g/ k g/ da y. 
A d mi nistrati o n of 4 m g/ k g C C X 1 6 8 t wice d ail y w as i de ntifie d as t he l o west d osi n g re gi me n t hat 
pr o d uce d a n ear- ma xi mal t hera pe utic b e nefit. At t his d ose, plas ma le vels ra n ge d fr o m 3 5 n g/ m L 
( Cmi n ) t o 2 0 0 n g/ m L ( Cma x ) t hr o u g h o ut t he da y. T h e sa me bl o o d bi o mar ker of C 5a R bl oc ka de 
use d i n t he P hase 1 cli nical trial was als o use d wit h t he h C 5a R KI mice; C C X 1 6 8 ha d si milar 
C 5a R a nta g o nist p ote nc y o n h u ma n a n d h C 5a R KI m o use n e utr o p hils (i n hi biti o n of C 5a-i n d uce d 
C D 1 1 b u pre g ulati o n i n bl o o d, I C 5 0 4 n M). T he e xte nt of f u ncti o nal C 5a R bl oc ka de o n bl o o d 
ne utr o p hils ass ociate d wit h t he plas ma le vels of 4 m g/ k g C C X 1 6 8 t wice dail y was d eter mi ne d t o 
ra n ge fr o m 9 9 % ( at Cma x ) t o 9 5 % (at Cmi n ), wit h a ti me-a vera ge d le v el of rece pt or bl oc ka de of 
9 7 %. 
T hese mec h a nis m - base d p har mac ol o g y st u dies, ta ke n t o get her, s u p p ort o ur esti mate t hat 
mai ntai ni n g h u ma n plas ma C C X 1 6 8 c o nce ntrati o ns s ufficie ntl y hi g h t o pr o vi de ≥ 9 5 % rece pt or 
c o vera ge will pr o vi de si g nifica nt cli nical be n efits i n i nfla m mat or y c o n diti o ns ass ociate d wit h 
C 5a R acti vati o n.  
1. 2. 2.  
N o n - Cli nic al S af et y a n d T o xic ol o g y 
T he t o xic ol o g y pr o gra m was desi g ne d t o s u p p ort t his P hase 2 st u d y t o assess t he safet y, 
t olera bilit y, p har mac o ki n etics, a n d efficac y of C C X 1 6 8 i n s u bjects wit h A A V. I n t his re gar d, a 
c o m pre he nsi ve t o xic ol o g y pr o gra m has b ee n c o n d ucte d i n li g ht of e xisti n g I C H n o n cli nical 
t o xic ol o g y g ui da nce (i ncl u di n g M 3 N o ncli nical Safet y St u dies f or t he C o n d uct of H u ma n 
Cli nical trials f or P har mace uticals; S 2 B S pecifi c As pects of Re g ulat or y G e n ot o xicit y Tests f or 
P har mace uticals; a n d S 7 A Safet y P har mac ol o g y St u dies f or  H u ma n P har mace uticals).   


C he m o Ce ntr y x, I n c.                            C O N FI D E N TI A L                                Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8                                                                                                                    A me n d me nt 4. 0 
                                                                   Pa ge 3 6 of 1 1 3                               1 8 Se pte m ber 2 0 1 5 
T he n o ncli nical t o xic ol o g y pr o gra m wit h C C X 1 6 8 c o nsiste d of a series of ac ute (si n gle- d os e) 
a n d/ or m ulti ple- d ose t o xic ol o g y or al st u dies c o n d ucte d i n rats a n d c y n o m ol g us m o n ke ys. Oral 
re peat- d ose r a n ge fi n di n g st u dies were s u bse qu e ntl y f oll o we d b y f or mal m ulti ple - d ose ( 2 8-da y) 
st u dies utilizi n g t he oral r o ute of a d mi nistrati o n. T he defi niti ve 2 8-da y a n d 1 3/ 2 0 -wee k st u dies 
i n v ol ve d t he a d mi nistrati o n of C C X 1 6 8 at dail y d oses u p t o 1 0 0 m g/ k g/ da y i n rats a n d u p t o 5 0 
m g/ k g/ da y i n c y n o m ol g us m o n ke ys a n d i n cl u de d c o m pre he nsi ve cli nical e val uati o ns a n d t he 
micr osc o pic assess me nt of a f ull list of tiss ues. T he m ulti ple - d ose c y n o m ol g us st u d y i n cl u de d 
electr ocar di o gr a p hic meas ure me nts. T o xic o ki netic data were als o c ollecte d i n t he m ulti ple - d ose 
rat a n d c y n o m ol g us st u dies. T he hi g hest d oses use d i n t he defi niti ve 1 3/ 2 0- wee k st u dies ( u p t o 
1 0 0 a n d 3 0 m g/ k g/ da y, i n rats a n d c y n o m ol g us m o n ke ys, res pecti vel y) re prese nte d H u m a n 
E q ui vale nt D oses ( H E D’s) of 1 5. 6 a n d 1 1. 1 m g/ k g/ da y, res pecti vel y.  
T h e eff ects of C C X 1 6 8 u p o n t he ce ntral n er v o us, res pirat or y, re nal, a n d car di o vasc ular s yste ms 
were als o assesse d i n si n gle - d os e sta n d-al o ne safet y p har m ac ol o g y e x peri me nts i n rats a n d 
c y n o m ol g us m o n ke ys. A n i n vitr o st u d y t o assess t he p ote ntial effects of C C X 1 6 8 u p o n h E R G 
c ha n nel i o nic c o n d u cta nce was als o c o n d u cte d. Wit h re gar d t o ge n ot o xicit y, i n vitr o bacterial 
(re verse m utati o n i n histi di ne-re q uiri n g strai ns of S. ty p hi m uri u m  a n d tr y pt o p ha n-re q uiri n g 
strai ns of E. c oli ) a n d ma m malia n ( m utati o n at t he t hy mi di ne ki nase l oc us of m o use l y m p h o ma 
L 5 1 7 8 Y cells) m uta ge nicit y tests wer e perf or me d. A d diti o nall y, a n i n viv o rat b o ne marr o w 
micr o n ucle us test was als o perf or me d.  
All t o xic ol o g y st u dies a n d safet y p har m ac ol o g y st u dies wit h t he e x ce pti o n of d ose a nal ys e s 
perf or me d i n s u p p ort of t he ac ute t o xic ol o g y a n d h E R G st u dies were c o n d ucte d i n acc or da n ce 
wit h G L P re g ul ati o ns. A s u m mar y of t he n o ncli nical t o xic ol o g y, ge n ot o xic ol o g y, a n d s afet y 
p har mac ol o g y st u dies perf or me d i n s u p p ort of C C X 1 6 8 are d escri be d i n Ta ble  1 a n d Ta ble  2. 
T a ble 1: 
O ver vie w of T o xic ol o g y St u dies Perf or me d wit h C C X 1 6 8  
St u d y T y pe  
Met h o d of 
A d mi nistr ati o n/ D osi n g 
Sc he d ule  
S pec ies 
D oses  
Ac ute  
Oral / Si n gle -D ose  
Rat  
0, 5, 2 5, 1 0 0 m g/ k g  
Re peat D ose  
Oral / 7 -Da y  
Rat  
0, 3 0, 1 0 0 m g/ k g  
 
Oral / 4 / ( 2) -d a y  
C y n o m ol g us m o n ke y  
3, 5 0, 6 5, ( 8 0), ( 1 2 0) 
m g/ k g  
 
Oral/ 2 8 -Da y   
Rat  
0, 5, 2 5, 1 0 0 m g/ k g  
 
Oral/ 2 8 -Da y  
C y n o m ol g us m o n ke y  
0, 5, 1 5 ,  5 0 m g/ k g 
 
Oral/ 1 3 -Wee k  
Rat  
0, 3, 1 5, 1 0 0 m g/ k g  
 
Oral/ 2 0 -Wee k  
C y n o m ol g us m o n ke y  
0, 5, 1 5, 3 0 m g/ k g  
Ge n ot o xicit y  
 
 
 
A mes  
N A  
S. ty p hi m uri u m  a n d 
tr y pt o p ha n-re q uiri n g 
strai ns of E. c oli  
U p t o 5 0 0 0 µ g/ plate  
M o use 
L y m p h o ma  
N A  
M o use l y m p h o ma 
cells 
U p t o 5 0 0 µ g/ m L  
Rat B o ne 
Marr o w 
Micr o n ucle us  
N A  
Rat  
0, 5 0 0, 1 0 0 0 a n d 2 0 0 0 
m g/ k g  


C he m o Ce ntr y x, I n c.                            C O N FI D E N TI A L                                Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8                                                                                                                    A me n d me nt 4. 0 
                                                                   Pa ge 3 7 of 1 1 3                               1 8 Se pte m ber 2 0 1 5 
St u d y T y pe  
Met h o d of 
A d mi nistr ati o n/ D osi n g 
Sc he d ule  
S pec ies 
D oses  
I m m u n ot o xicit y 
Oral/ 2 8 -Da y  
Rat  
0, 3, 1 5, 1 0 0 m g/ k g  
T a ble 2:  
O ver vie w of S af et y P h a r m ac ol o g y St u dies Perf or me d wit h C C X 1 6 8 
St u d y T y pe  
Met h o d of  
A d mi nistr ati o n/ D osi n g 
Sc he d ule  
S pecies  
D oses  
Ce ntral Ner v o us 
S yste m  
Oral/ Si n gle -D ose  
Rat  
0, 5, 2 5, 1 0 0 m g/ k g  
Car di o vasc ular –  
I n viv o 
Oral/ Si n gle -D ose  
C y n o m ol g us m o n ke y  
0, 5, 1 5, 5 0 m g/ k g  
Car di o vasc ular –  
I n vitr o ( hE R G)  
I n vitr o 
H u ma n cells 
tra nsfect ed wit h 
h u ma n K-c ha n nel 
ge n e  
0. 6, 1. 2, 2. 3 a n d 6. 9  µ M  
Res pirat or y  
Oral / Si n gle -D ose  
Rat  
0, 5, 2 5, 1 0 0 m g/ k g  
Re n al  
Or al/ Si n gle -D ose  
R at  
0, 5, 2 5, 1 0 0 m g/ k g  
Base d o n i n viv o saf et y p har mac ol o g y st u dies, w hic h i ncl u de d a ne ur o p har mac ol o g y st u d y i n 
rats, a pul m o nar y a n d re n al safet y st u d y i n rats, a n d a car di o v asc ular st u d y i n c o nsci o us 
tele metere d c y n o m ol g us m o n ke ys, t here w as n o e vi de nce of t o xicit y of C C X 1 6 8. I n a n i n vitr o 
car di o vas c ular safet y st u d y, t he I C 5 0 val ue f or h E R G i n hi biti o n was deter mi ne d t o b e > 2. 3 µ M 
(t he li mit of s ol u bilit y), f urt her i n dicati n g C C X 1 6 8 is u nli kel y t o ca use arr h yt h mias i n viv o. 
Pr otei n bi n di n g, re d bl o o d cell partiti o ni n g, he p at oc yt e meta b olis m, c yt o c hr o me P 4 5 0 i n hi biti o n 
a n d i n d ucti o n, Cac o- 2 per mea bilit y a n d ge n ot o xicit y st u dies, i ncl u di n g i n vitr o bacterial 
m uta ge ni cit y ( A mes test), i n vitr o ma m malia n cell m uta ge ni cit y ( m o us e l y m p h o ma) st u dies, a n d 
i n viv o rat b o ne marr o w micr o n ucle us test were als o c o n d ucte d a n d di d n ot i de ntif y a n y s afet y 
c o ncer ns or si g nifica nt p ote ntial  f or dr u g- dr u g i nt eracti o ns. I n a n ac ut e t o xic ol o g y st u d y, si n gl e 
d oses of C C X 1 6 8 u p t o 1 0 0 m g/ k g i n rats pr o d uce d n o re mar k a ble effects. I n a 2 8- da y 
i m m u n ot o xicit y st u d y i n rats, i n w hic h T-cell de pe n de nt a nti b o d y pr o d u cti o n was assesse d, d ail y 
C C X 1 6 8 d os es u p t o 1 0 0 m g/ k g were free of a n y effects.  
C C X 1 6 8 was well t olerat e d i n 2 8- da y st u dies u p t o d oses of 1 0 0 m g/ k g i n rats a n d 5 0 m g/ k g i n 
c y n o m ol g us m o n ke ys. T here w ere n o si g nifica nt t o xic ol o gical fi n di n gs of c o ncer n i n t hes e 
st u dies. At selecte d ti me p oi nts, mi n or b ut statisticall y si g nifica nt differe nces e xiste d i n selecte d 
cli nical pat h ol o g y p ara m eters bet w ee n c o ntr ol a n d C C X 1 6 8 treate d rats. T hese i ncl u de d a n 
i ncreas e i n retic ul oc ytes i n 1 0 0 m g/ k g/ da y rec o ver y- p h ase fe males, a n i n crease i n pr ot hr o m bi n 
ti me i n males gi ve n d oses > 2 5 m g/ k g/ da y a n d a mi ni mal i ncrease i n A L T le vels o n Da y 3 0 i n 
fe males a d mi nister e d 1 0 0 m g/ k g/ da y. T hes e differe nces were n ot clearl y test article-relate d a n d 
n ot c o nsi dere d t o be of t o xic ol o gical i m p orta nce b eca use of t heir s mall ma g nit u de, mea n a n d/ or 
i n di vi d ual val ues falli n g wit hi n t he ra n ge of n or m al varia bilit y a n d/ or re v ersi bilit y of t he fi n di n g 
f oll o wi n g a 2-wee k treat me nt -free peri o d. 


C he m o Ce ntr y x, I n c.                            C O N FI D E N TI A L                                Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8                                                                                                                    A me n d me nt 4. 0 
                                                                   Pa ge 3 8 of 1 1 3                               1 8 Se pte m ber 2 0 1 5 
C C X 1 6 8 was als o well t olerate d i n a 1 3- wee k st u d y i n rats u p t o d oses of 1 0 0 m g/ k g/ da y a n d i n 
a 2 0- wee k st u d y i n c y n o m ol g us m o n ke ys u p t o 3 0 m g/ k g/ da y. T here w ere n o si g nifica nt 
t o xic ol o gical fi n di n gs of c o ncer n i n t hes e st u dies. T he o nl y C C X 1 6 8-relate d cli nical o bser vati o n 
i n t he rat st u d y w as i nfre q ue nt sali vati o n, w hic h was n ote d i n t he mi d a n d hi g h d ose gr o u ps. 
T here w ere n o C C X 1 6 8 r elate d c h a n ges i n b o d y w ei g hts, f o o d c o ns u m pti o n, o p ht hal mic 
e x a mi nati o ns, he mat ol o g y, c o a g ulati o n, gr oss o bs er vati o ns, or ga n wei g hts, a n d micr osc o pic 
e val uati o n. T her e were n o C C X 1 6 8- relate d effects o n cli nical c he mistr y at ter mi nati o n or 
rec o ver y. Ser u m A S T, A L T, a n d s or bit ol de h y dr o ge n ase w ere mil dl y i ncrease d i n t w o fe mal es at 
1 0 0 m g/ k g/ da y at ter mi n ati o n. Gi ve n t he ma g nit u de a n d l o w i nci de n ce of t hese fi n di n gs, t he y 
were c o nsi dere d i n ci de nt al. I n t h e m o n ke y st u d y, C C X 1 6 8 was well t olerat e d at d oses u p t o 3 0 
m g/ k g/ da y.  
A meta b olite of C C X 1 6 8 ( C C X 1 6 8- M 1) has bee n detecte d i n h u ma n P hase 1 sa m ples i n 
si g nifica nt a m o u nts. T his c o m p o u n d, w hic h is als o pr o d uce d i n si g nifica nt a m o u nts i n t he 
precli nical t o xic ol o g y s p ecies, lac ks str o n g i n hi biti o n of t he h E R G p otassi u m c ha n nel 
(I C5 0 > 3 µ M).  
Gi ve n t he lac k of si g nifica nt safet y c o n cer ns i n t he t o xic ol o g y st u dies a n d t he P hase 1 st u d y i n 
healt h y v ol u nteers, a n d t he safet y r es ults fr o m t his cli nical trial s o far,  t he ris k f or seri o us or 
u na ntici pate d u nt o war d e ve nts t o occ ur i n t his cli nical trial is c o nsi dere d l o w. 
1. 2. 3.  
N o n -Cli nic al A D M E  
T he p har mac o ki netic be h a vi or of C C X 1 6 8 has bee n assesse d i n fe m ale C D- 1 mice, male 
S pra g u e -D a wle y r ats, male bea gle d o gs,  a n d m ale c y n o m ol g us m o n ke ys t hr o u g h i ntra v e n o us 
(i. v.) a n d/ or or al ( p. o.) d osi n g a n d t he d ata are s u m marize d i n Ta ble  3. F oll o wi n g i ntra v e n o us 
d osi n g, t he c o m p o u n d has a m o derate t o me di u m t otal b o d y cl eara nce i n mi ce, rats, a n d d o gs 
( 3 0 - 5 0 % of li ver bl o o d fl o w). T he ter mi nal eli mi nati o n half-life is m o derate t o l o n g, at ca. 2 h 
i n mice a n d rats, a n d 1 4. 2 h i n d o gs, w hile t he v ol u me of distri b uti o n is m o derate ( 1. 5, 2. 5, a n d 
4. 7 L/ k g f or mice, r ats, a n d d o gs, r es pecti vel y). F oll o wi n g oral d osi n g i n mice a n d rats, C C X 1 6 8 
is rea dil y a bs or be d, s h o wi n g m o der ate bi oa vaila bilit y f or t he a q ue o us h y dr o x y pr o p yl 
met h ylcell ul ose ( H P M C) s us pe nsi o n a n d hi g h bi o a vaila bilit y f or t he P E G- 4 0 0/ S ol ut ol H S- 1 5 
s ol uti o n.   


C he m o Ce ntr y x, I n c.                            C O N FI D E N TI A L                                Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8                                                                                                                    A me n d me nt 4. 0 
                                                                   Pa ge 3 9 of 1 1 3                               1 8 Se pte m ber 2 0 1 5 
T a ble 3:  
P h ar m a c o ki netic P ar a meters of C C X 1 6 8 i n C D- 1 Mice, S pr a g ue -D a wle y 
R ats, Be a gle D o gs, a n d C y n o m ol g us M o n ke ys after A d mi nistr ati o n of a 
Si n gle Or al G a v a ge or a n I ntr a ve n o us D ose of C C X 1 6 8 
a. I ntr a ve n o us d osi n g 
P ar a met er  
M o use  
R at  
D o g  
d o se ( m g/ k g) 
0. 5  
0. 5  
0. 5  
F or m ul ati o n 
Et ha n ol / N, N -
di met h ylaceta mi de / 
pr o p yle n e gl yc ol / 
0. 9 % sali ne 
( 1 0: 1 0: 3 0: 7 5)  
N, N -
di met h ylaceta mi de / 
et ha n ol / pr o p yl e ne 
gl yc ol ( 3 1. 6: 3 6. 8: 3 1. 6) 
pr o p yle n e gl yc ol / 
N, N -
di met h ylaceta mi de / 
water ( 3 1. 6/ 3 1. 6/ 3 6. 8) 
N =  
9  a 
2  
3  
C L [ m L/ mi n/ k g]  
2 6. 6  
2 1. 2  
1 1. 9  
t1/ 2 [ h] 
1. 8  
1. 9  
1 4. 2  
V d ss [ L/ k g] 
1. 5  
1. 8  
4. 7  
a N o n -serial bl o o d sa m pli n g w as use d a n d a c o m p osite P K pr ofile was o btai ne d usi n g t he mea n 
c o nce ntr ati o n at eac h ti me p oi nt 
b. Or al d osi n g 
P ar a met er  
M o use  
R at  
M o n ke y  
p . o. d ose ( m g/ k g) 
2  
3 0  
2  
3 0  
1 0 0  
F or m ul ati o n 
1 % H P M C 
(s us pe nsi o n) 
P E G -
4 0 0/s ol ut ol 
H S - 1 5 
( 7 0: 3 0) 
(s ol uti o n) 
1 % H P M C 
(s us pe nsi o n) 
P E G -
4 0 0/ S ol ut ol 
H S - 1 5 
( 7 0: 3 0) 
(s ol uti o n) 
P E G - 4 0 0 / 
s ol ut ol H S-
1 5 ( 7 0: 3 0) 
(s ol uti o n) 
N =  
9  a 
9  a 
2  
3  
3  
C m a x  [ n g/ m L] 
7 5  
4 6 3 0  
1 5 2  
2 5 3 0  
3 5 0 0  
A U C [ n g• h/ m L]  
2 4 0  
1 8 6 0 0  
4 6 4  
2 4 6 0 0  
3 3 3 0 0  
t1/ 2 [ h] 
  
5. 6  
2. 3  
4. 6  
6. 0  
tm a x  [ h] 
1. 0  
1. 0  
1. 0  
1. 5  
4. 0  
F [ %]  
1 7  
8 7  
2 7  
1 0 4  
- 
C C X 1 6 8 dis pla ys m o d er ate i n vitr o meta b olic t ur n o ver i n cr y o- pres er ve d m o use, rat, a n d d o g 
he pat oc ytes a n d l o w t o m o derate t ur n o v er i n h u ma n he pat oc ytes. T his res ult ge n erall y c orrel ates 
well wit h t he o bser ve d i n viv o cleara n ce i n mice, rats a n d  d o gs a n d pre dicts a l o w t o m o derate 
clear a nce i n h u ma ns. I n vitr o meta b olis m of C C X 1 6 8 is pri maril y t hr o u g h m o n o h y dr o x ylati o n i n 
m o n ke y a n d h u ma n he p at oc ytes a n d t hr o u g h m o n o h y dr o x yl ati o n, deal k yl ati o n a n d 
gl uc ur o ni d ati o n i n rat a n d d o g he p at oc ytes. A P hase I m o n o h y dr o x ylati o n meta b olite, C C X 1 6 8-
a N o n -serial bl o o d sa m pli n g was use d a n d a c o m p osite P K 
pr ofile was o btai ne d usi n g t he mea n c o nce ntrati o n at eac h 
ti me p oi nt. 
C L = t otal b o d y cleara nce  
t½  = ter mi nal half -life 
V d ss = v ol u me of distri b uti o n at stea d y state  
i. v. = i ntra ve n o us 
T ma x  = ti me of pea k c o nce ntrati o n  
p. o. = oral  
F = bi oa vaila bilit y  
C ma x  = ma xi m u m c o nce ntrati o n  
A U C = area u n der t he c o nce ntrati o n -vs. -ti me c ur ve 


C he m o Ce ntr y x, I n c.                            C O N FI D E N TI A L                                Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8                                                                                                                    A me n d me nt 4. 0 
                                                                   Pa ge 4 0 of 1 1 3                               1 8 Se pte m ber 2 0 1 5 
M 1, was f o u n d t o be a si g nifica nt circ ulati n g met a b olite i n h u ma n v ol u nteers i n t he P hase 1 
st u d y. Its e x p os ure was a de q uatel y c o v ere d i n rats a n d c y n o m ol g us m o n ke ys i n t he t o xic ol o g y 
st u dies t hat s u p p ort t he c urrent P hase 2 cli nical trial.    
T he e x creti o n pat h wa ys of C C X 1 6 8 i n a ni mals were i n vesti gate d i n bile- d uct ca n n ulate d 
S pra g u e - D a wle y r ats a n d i ntact c y n o m ol g us m o n k e ys. Neit her C C X 1 6 8 n or t he meta b olite 
C C X 1 6 8- M 1 is si g nifica ntl y e x crete d i nt o uri ne or bile i n rats or uri ne i n c y n o m ol g us m o n ke ys.  
T he c o m p o u n d is ver y hi g hl y pr otei n b o u n d i n m o use, rat, d o g, a n d h u ma n plas ma, at ca. 9 9 % or 
hi g her, wit h c orr es p o n di n g u n b o u n d fracti o ns at ca. 1 % or l o wer. C C X 1 6 8 has a l o w 
meta b olis m - me diate d dr u g-dr u g i nteracti o n p ote ntial as a per p etrat or, as its i n hi biti o n a gai nst 
maj or h u ma n c yt oc hr o m e P 4 5 0 is of or ms is mi ni mal ( ne gli gi ble f or C Y P 1 A 2, 2 C 9, 2 C 1 9, a n d 
2 D 6, a n d C Y P 3 A 4) a n d it s h o ws n o C Y P 3 A 4 a n d C Y P 2 B 6 i n d ucti o n p ote ntial at 1 0 μ M i n a 
h u ma n he pat oc yte c yt oc hr o me P 4 5 0 assa y. 
T he a bilit y of C C X 1 6 8 or C C X 1 6 8- M 1 t o i n hi bit t he ster oi d - meta b olizi n g e nz y mes 1 1 β-H S D 1 
a n d 1 1 β- H S D 2 was e val uate d usi n g s uita ble i n vitr o s yste ms a n d b ot h c o m p o u n ds were f o u n d t o 
be i nacti ve a gai nst t hese e nz y mes. C C X 1 6 8 was als o f o u n d t o ha ve n o i m pact o n t he u n b o u n d 
c o nce ntr ati o n of pre d nis o ne a n d pre d nis ol o ne i n h u ma n plas ma. F urt h er m ore, neit her C C X 1 6 8 
n or C C X 1 6 8- M 1 i nterfer e s wit h t he a nti pr oliferati ve effects of c ycl o p h os p ha mi de, w hic h is 
k n o w n t o re q uire C Y P- m e diate d c o n v ersi o n t o a n acti ve meta b olite. T heref ore, C C X 1 6 8 has l o w 
p ote ntial f or i nterf eri n g wit h t he bi ol o gical effects of eit her c ycl o p h os p h a mi de or c ortic oster oi ds.  
Preli mi n ar y res ults f r o m m ass b al a nce st u dies i n r ats a n d he alt h y h u m a n s u bjects 
( C L 0 0 4 _ 1 6 8) wit h or al a d mi nistr ati o n of [1 4C] C C X 1 6 8 s h o w e d t h at m ost of t he 
r a di o acti vit y w as rec o v ere d i n f eces, w hile re n al eli mi n ati o n pl a ye d a ver y mi n or r ole. 
He p atic met a b olis m of C C X 1 6 8 f oll o w e d b y bili ar y e xcreti o n of t he met a b olites a p pe are d 
t o be t he m aj o r r o ute of eli mi n ati o n f or C C X 1 6 8. Refer t o t he I n vesti g at or’s Br oc h ure f or 
m o re det ails.  
1. 3.  
Pri or H u m a n E x perie nce  
A P hase 1 st u d y i n 4 8 healt h y v ol u nteers has bee n c o m plete d. T his is a ra n d o mize d, d o u ble-
bli n d, place b o-c o ntr olle d, t w o- peri o d st u d y i n w hic h s u bjects recei ve d eit h er C C X 1 6 8 or place b o 
( 3: 1 rati o) as a si n gle d os e i n Peri o d 1 a n d as m ulti ple o nce dail y or t wice d ail y d oses i n Peri o d 
2. Si n gle d oses of 1, 3, 1 0, 3 0, a n d 1 0 0 m g C C X 1 6 8 were st u die d, 6 s u bjects i n eac h d ose c o h ort 
recei v e d CC X 1 6 8 a n d 2 r ecei ve d pl ace b o, e x ce pt i n c o h ort 1 i n w hic h 5 s u bjects recei ve d 
C C X 1 6 8 a n d 3 recei ve d place b o. I n Peri o d 2, C C X 1 6 8 d oses of 1, 3, a n d 1 0 m g o nce dail y f or 7 
da ys, a n d 3 0 a n d 5 0 m g t wice dail y f or 7 d a ys, were st u die d.  
C C X 1 6 8 a p pear e d t o be well t olerate d b y st u d y s u bjects i n t his st u d y. N o seri o us a d vers e e v e nts 
or wit h dra wals d u e t o a d verse e v e nts ha ve b ee n o bser ve d. A s u m mar y of all t he treat me nt -
e mer ge nt a d v erse e ve nts o bser ve d i n t he si n gle - d ose peri o d of t he st u d y is pr o vi de d i n Ta ble  4. 


C he m o Ce ntr y x, I n c.                            C O N FI D E N TI A L                                Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8                                                                                                                    A me n d me nt 4. 0 
                                                                   Pa ge 4 1 of 1 1 3                               1 8 Se pte m ber 2 0 1 5 
T a ble 4: 
S u m m ar y of Tre at me nt -e mer ge nt A d verse E ve nts O bser ve d i n t he S i n gle-
d ose Peri o d, C L 0 0 1 _ 1 6 8 
 
Peri o d 1 ( Si n gle D ose)  
S yste m Or g a n Cl ass  
Preferre d Ter m  
 
C C X 1 6 8 D ose  
 
Pl ace b o  
( N = 1 1) 
1 m g  
( N = 5) 
3 m g  
( N = 6) 
1 0 m g  
( N = 6) 
3 0 m g  
( N = 6) 
1 0 0 m g  
( N = 6) 
All 
C C X 1 6 8  
( N = 2 9) 
N u m ber ( %) of s u bj ects 
wit h A Es  
1 ( 9 %)  
3 ( 6 0 %)  
3 ( 5 0 %)  
1 ( 1 7 %)  
3 ( 5 0 %)  
2 
( 3 3 %) 
1 2 
( 4 1 %) 
Gastr oi ntesti nal 
Dis or ders  
0 ( 0 %)  
0  
1  
1  
1  
0  
3 ( 1 0 %)  
A b d o mi nal pai n  
0 ( 0 %)  
0  
0  
1  
1  
0  
2 ( 7 %)  
Diarr h oea  
0  ( 0 %) 
0  
1  
1  
0  
0  
2 ( 7 %)  
Ge neral Dis or ders a n d 
A d mi nistrati o n Site 
C o n diti o ns  
0 ( 0 %)  
1  
1  
0  
1  
0  
3 ( 1 0 %)  
Cat heter Site P ai n  
0 ( 0 %)  
0  
0  
0  
1  
0  
1 ( 3 %)  
Fati g ue  
0 ( 0 %)  
1  
0  
0  
0  
0  
1 ( 3 %)  
I nj ecti o n Site 
P hle bitis  
0 ( 0 %)  
0  
1  
0  
0  
0  
1 ( 3 %)  
Vessel p u nct ure site 
he mat o ma  
0 ( 0 %)  
0  
0  
0  
1  
0  
1 ( 3 %)  
I n vesti gati o ns 
0 ( 0 %)  
1  
0  
0  
2  
1  
4 ( 1 4 %)  
L y m p h oc yte 
perce nta ge decrease d  
0 ( 0 %)  
0  
0  
0  
1  
0  
1 ( 3 %)  
W hite bl o o d cell 
c o u nt decrease d  
0 ( 0 %)  
1  
0  
0  
1  
1  
3 ( 1 0 %)  
Me nstr uati o n a n d uteri ne 
blee di n g N E C  
0 ( 0 %)  
1  
0  
0  
0  
0  
1 ( 3 %)  
D ys me n o rr h oea 
0 ( 0 %)  
1  
0  
0  
0  
0  
1 ( 3 %)  
Ner v o us S yste m 
Dis or ders  
0 ( 0 %)  
0  
2  
0  
1  
1  
4 ( 1 4 %)  
Dizzi ness  
0 ( 0 %)  
0  
0  
0  
0  
1  
1 ( 3 %)  
Hea dac he  
0 ( 0 %)  
0  
2  
0  
1  
0  
3 ( 1 0 %)  
Res pirat or y, t h oracic a n d 
me diasti nal dis or ders  
1 ( 9 %)  
0  
0  
0  
2  
0  
2 ( 7 %)  
Nas o p har y n gitis  
0 ( 0 %)  
0  
0  
0  
1  
0  
1 ( 3 %)  
R hi nitis  
0 ( 0 %)  
0  
0  
0  
1  
0  
1 ( 3 %)  
T hr oat irritati o n  
1 ( 9 %)  
0  
0  
0  
0  
0  
0 ( 0 %)  
All a d verse e ve nts were mil d e x ce pt f or o ne A E of i njecti o n site p hle bitis i n t he 3 m g C C X 1 6 8 
gr o u p a n d o ne of W B C c o u nt decrease i n t he 1 m g gr o u p t h at were c o nsi d ere d  m o derate i n 
i nte nsit y. T he latter s u bject ha d a l o w W B C c o u nt at baseli ne, 3. 0 9 x 1 09/ L (l o wer li mit of n or mal 
[ L L N]: 4. 2 3 x 1 09/ L) a n d a me dical hist or y of l o w W B C c o u nt. T he na dir w as 2. 0 4 x 1 09/ L o n Da y 
4. T he ot her t w o s u bjects wit h l o w W B C ha d Gra de 1 a b n or malities; o ne s u bject, w h o ha d a l o w 
baseli ne W B C of 3. 5 8 x 1 0 9/ L ( L L N: 3. 9 8 x 1 09/ L), recei v e d 3 0 m g C C X 1 6 8 a n d ha d a n a dir of 
3. 2 6 x 1 0 9/ L o n D a y 2. T h e ot her s u bject, w h o als o ha d a baseli ne W B C of 4. 4 1 x 1 09/ L ( L L N: 
4. 2 3 x 1 0 9/ L), recei ve d 1 0 0 m g C C X 1 6 8 a n d reac h e d a na dir of 3. 8 3 x 1 09/ L o n Da y 4. T he ot her 
m ost c o m m o n A E was hea dac he, o bser ve d i n t hree s u bjects, t w o recei vi n g 3 m g a n d t he t hir d 
recei vi n g 3 0 m g C C X 1 6 8.  


C he m o Ce ntr y x, I n c.                            C O N FI D E N TI A L                                Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8                                                                                                                    A me n d me nt 4. 0 
                                                                   Pa ge 4 2 of 1 1 3                               1 8 Se pte m ber 2 0 1 5 
N o cli nicall y si g nifica nt c ha n ges i n la b orat or y para meters, vital si g ns, or E C G para meters wer e 
o bser ve d. K e y p har m ac o ki netic res ults ( mea n, S D) fr o m t he si n gle- d ose p eri o d are s u m marize d 
i n Ta ble 5.  
T a ble 5: 
S u m m ar y of Ke y P h ar m a c o ki netic Res ults fr o m t he Si n gle -d ose Peri o d, 
C L 0 0 1 _ 1 6 8 
 
C C X 1 6 8 D ose  
P ar a met er  
1 m g  
( N = 5) 
3 m g  
( N = 6) 
1 0 m g  
( N = 6) 
3 0 m g  
( N = 6) 
1 0 0 m g  
( N = 6) 
C ma x  ( n g/ m L) 1. 8 4 ( 0. 8 8 9)  
9. 1 7 ( 1. 9 8)  
2 5. 3 ( 5. 7 1)  
7 8. 7 ( 3 5. 6)  
1 9 7 ( 1 5 7)  
T ma x  ( hr) 
1. 1 ( 0. 2 2)  
1. 2 ( 0. 2 6)  
1. 7 ( 0. 2 6)  
1 . 7 ( 0. 4 1) 
2. 5 ( 1. 8)  
t1/ 2 ( hr) 
2. 0 3 ( 0. 7 2 1)  
1. 9 2 ( 1. 0 2)  
2 2. 9 ( 7. 3 3)  
7 1. 8 ( 3 2. 8)  
6 4. 0 ( 2 2. 1)  
C L/ F ( L/ hr)  
1 9 5 ( 7 9. 7)  
1 3 1 ( 3 8. 5)  
8 7. 0 ( 4 0. 8)  
5 1. 7 ( 1 4. 9)  
6 2. 4 ( 3 4. 0)  
A U C 0- i nf 
( n g• hr/ m L) 
6. 1 4 ( 3. 2 5)  
2 5. 2 ( 9. 5 8)  
1 3 0 ( 3 9. 4)  
6 2 8 ( 1 9 9)  
2 0 3 0 ( 1 0 7 0)  
I n ge ner al, C C X 1 6 8 a p pears t o ha ve a bi p hasic pr ofile, wit h a ra pi d distri b uti o n p hase f oll o we d 
b y a l o n g ter mi nal p hase, a n d l o w a p pare nt cleara nce i n h u ma ns. F oll o wi n g oral a d mi nistrati o n, 
C C X 1 6 8 was a bs or be d r a pi dl y a n d reac he d p ea k plas ma le vels after 1 t o 2 h o urs. F oll o wi n g a 
si n gle d ose of 1- 1 0 0 m g, mea n e x p os ures as meas ure d b y A U C 0- i nf ra nge d fr o m 6. 1 4 t o 2 0 3 0 
n g• hr/ m L w hile C ma x  ra n ge d fr o m 1. 8 4 t o 1 9 7 n g/ m L. T he a p pare nt mea n t er mi nal half-life 
ra n ge d fr o m 1. 9 2 t o 7 1. 8 hr, w hile cleara n ce ra n ge d fr o m 5 1. 7 t o 1 9 5 L/ hr. 
A s u m mar y of all t he treat me nt -e mer ge nt a d verse e ve nts o bser v e d i n t he m ulti ple - d ose peri o d of 
t he st u d y is pr o vi de d i n Ta ble 6 . 
T a ble 6: 
S u m m ar y of all t he tre at me nt -e mer ge nt a d vers e e ve nts o bser v e d i n t he 
m ulti ple -d ose peri o d of t he st u d y , C L 0 0 1 _ 1 6 8 
 
Peri o d 2 ( M ulti -D ose)  
S yste m Or g a n Cl ass  
Preferre d Ter m  
 
 
C C X 1 6 8 D ose Gr o u p  
 
Pl ace b o  
( N = 1 1) 
1 m g 
Q D  
X 7 d 
( N = 5) 
3 m g 
Q D  
X 7 d  
( N = 6) 
1 0 Q D  
X 7 d 
( N = 6) 
3 0 m g 
BI D  
X 7 d 
( N = 6) 
5 0 m g 
BI D  
X 7 d 
( N = 6) 
All 
C C X 1 6 8 
( N = 2 9) 
N u m ber ( %) of s u bj ects wit h A Es  
4  ( 3 6 %) 
3 ( 6 0 %)  
2 ( 3 3 %)  
5 
( 8 3 %) 
4 ( 6 7 %)  
3 ( 5 0 %)  
1 7 ( 5 9 %)  
E ye dis or ders  
1 ( 9 %)  
0  
0  
0  
0  
0  
0 ( 0 %)  
E yeli d oe de ma  
1 ( 9 %)  
0  
0  
0  
0  
0  
0 ( 0 %)  
Gastr oi ntesti nal dis or ders  
2 ( 1 8 %)  
0  
0  
2  
0  
2  
4 ( 1 4 %)  
A b d o mi nal pai n l o wer  
0 ( 0 %)  
0  
0  
1  
0  
1  
2 ( 7 %)  
A b d o mi nal pai n u p per  
2 ( 1 8 %)  
0  
0  
0  
0  
1  
1 ( 3 %)  
C o nsti pati o n  
0 ( 0 %)  
0  
0  
0  
0  
1  
1 ( 3 %)  
Diarr h oea  
1 ( 9 %)  
0  
0  
2  
0  
0  
2 ( 7 %)  
Na usea  
0 ( 0 %)  
0  
0  
1  
0  
1  
2 ( 7 %)  
V o miti n g  
0 ( 0 %)  
0  
0  
1  
0  
0  
1 ( 3 %)  
Ge neral dis or ders a n d 
a d mi nistrati o n site c o n diti o ns  
1 ( 9 %)  
1  
1  
0  
0  
0  
2 ( 7 %)  
Fati g ue  
1 ( 9 % ) 
0  
0  
0  
0  
0  
0 ( 0 %)  


C he m o Ce ntr y x, I n c.                            C O N FI D E N TI A L                                Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8                                                                                                                    A me n d me nt 4. 0 
                                                                   Pa ge 4 3 of 1 1 3                               1 8 Se pte m ber 2 0 1 5 
 
Peri o d 2 ( M ulti -D ose)  
S yste m Or g a n Cl ass  
Preferre d Ter m  
 
 
C C X 1 6 8 D ose Gr o u p  
 
Pl ace b o  
( N = 1 1) 
1 m g 
Q D  
X 7 d 
( N = 5) 
3 m g 
Q D  
X 7 d  
( N = 6) 
1 0 Q D  
X 7 d 
( N = 6) 
3 0 m g 
BI D  
X 7 d 
( N = 6) 
5 0 m g 
BI D  
X 7 d 
( N = 6) 
All 
C C X 1 6 8 
( N = 2 9) 
Feeli n g of b o d y te m perat ure 
c ha n ge  
0 ( 0 %)  
1  
0  
0  
0  
0  
1 ( 3 %)  
I nj ecti o n site s welli n g 
0 ( 0 %)  
0  
1  
0  
0  
0  
1 ( 3 %)  
I nfecti o ns a n d i nfestati o ns 
1 ( 9 %)  
0  
0  
1  
0  
0  
1 ( 3 %)  
C ystitis  
1 ( 9 %)  
0  
0  
0  
0  
0  
0 ( 0 %)  
Si n usitis  
0 ( 0 %)  
0  
0  
1  
0  
0  
1 ( 3 %)  
I nj ury, p ois o ni n g a n d pr oce d ural 
c o m plicati o ns  
0 ( 0 %)  
0  
0  
1  
0  
0  
1 ( 3 %)  
T o ot h i nj ur y  
0 ( 0 %)  
0  
0  
1  
0  
0  
1 ( 3 %)  
I n vesti gati o ns 
0 ( 0 %)  
1  
0  
1  
0  
0  
2 ( 7 %)  
As partate a mi n otra nsferase 
i ncrease d 
0 ( 0 %)  
0  
0  
1  
0  
0  
1 ( 3 %)  
Bl o o d creati ne p h os p h o ki nase 
i ncrease d 
0 ( 0 %)  
0  
0  
1  
0  
0  
1 ( 3 %)  
W hite cell c o u nt decrease d  
0 ( 0 %)  
1  
0  
0  
0  
0  
1 ( 3 %)  
M usc ul os keletal a n d c o n necti ve 
tiss ue dis or ders 
0 ( 0 %)  
0  
0  
1  
2  
0  
3 ( 1 0 %)  
B ac k pai n  
0 ( 0 %)  
0  
0  
1  
0  
0  
1 ( 3 %)  
M usc ular wea k ness  
0 ( 0 %)  
0  
0  
0  
1  
0  
1 ( 3 %)  
Nec k pai n  
0 ( 0 %)  
0  
0  
0  
1  
0  
1 ( 3 %)  
Ne r v o us s yste m dis or ders 
2 ( 1 8 %)  
2  
1  
0  
1  
3  
7 ( 2 4 %)  
Dizzi ness  
0 ( 0 %)  
0  
1  
0  
0  
1  
2 ( 7 %)  
Hea dac he  
2 ( 1 8 %)  
2  
0  
0  
1  
3  
6 ( 2 1 %)  
Res pirat or y, t h oracic a n d 
me diasti nal dis or ders  
1 ( 9 %)  
0  
0  
0  
2  
2  
4 ( 1 4 %)  
C hest pai n  
0 ( 0 %)  
0  
0  
0  
0  
1  
1 ( 3 %)  
Nas o p har y n gitis  
0 ( 0 %)  
0  
0  
0  
1  
0  
1 ( 3 %)  
Or o p har y n geal pai n  
1 ( 9 %)  
0  
0  
0  
1  
1  
2 ( 7 %)  
S ki n a n d s u bc uta ne o us tiss ue 
dis or ders  
0 ( 0 %)  
0  
0  
1  
1  
0  
2 ( 7 %)  
Se b orr h oeic der matitis  
0 ( 0 %)  
0  
0  
0  
1  
0  
1 ( 3 %)  
S ki n disc o mf ort  
0 ( 0 %)  
0  
0  
1  
0  
0  
1 ( 3 %)  
Vasc ular dis or ders  
0 ( 0 %)  
0  
0  
0  
1  
0  
1 ( 3 %)  
Hae mat o ma  
0 ( 0 %)  
0  
0  
0  
1  
0  
1 ( 3 %)  
T he m ost c o m m o n A E re p orte d i n s u bjects recei vi n g C C X 1 6 8 i n t he m ulti - d ose peri o d was 
hea dac he, r e p orte d i n 2 1 % of s u bjects recei vi n g C C X 1 6 8 c o m pare d t o 1 8 % i n t he place b o gr o u p. 
Diarr h oea ( 7 % vs. 9 % i n place b o), dizzi ness ( 7 % vs. 0 % i n place b o), l o w er a b d o mi nal pai n ( 7 % 
vs. 0 % i n place b o), n a usea ( 7 % vs. 0 % i n place b o), a n d or o p har y n geal pai n ( 7 % vs. 9 % i n 
place b o) w er e t he ot her m ore c o m m o nl y o bs er ve d A Es. T he o ne case of A S T a n d C P K ele vati o n 
was c o nsi der e d li kel y rel a te d t o stre n u o us e x ercis e a n d n ot c o nsi dere d rel ate d t o C C X 1 6 8. T he 
o ne s u bject wit h l o w W B C c o u nt i n t he 1 m g C C X 1 6 8 gr o u p was t he sa m e s u bject w h o ha d l o w 
W B C d uri n g t he si n gle- d ose peri o d of t he st u d y. T he W B C c o u nt na dir was 2. 2 2 x 1 0
9/ L o n 
Da y  4. N o clinicall y si g nifica nt c ha n ges i n la b orat or y p ara m eters, vital si g ns, or E C G para m eters 
were o bs er ve d. I n t he m ulti- d ose peri o d of t he st u d y, a sli g ht decrease i n mea n W B C a n d 


C he m o Ce ntr y x, I n c.                            C O N FI D E N TI A L                                Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8                                                                                                                    A me n d me nt 4. 0 
                                                                   Pa ge 4 4 of 1 1 3                               1 8 Se pte m ber 2 0 1 5 
ne utr o p hil c o u nt was o bs er ve d m or e fre q ue ntl y i n s u bjects recei vi n g C C X 1 6 8 c o m pare d t o 
place b o. T hes e W B C a n d ne utr o p hil c o u nts m ost ofte n re mai n e d wit hi n t he refere nce ra n ge, 
decreases w ere o bs er ve d wit hi n 1 t o 2 da ys aft er st art of d osi n g, a p peare d t o be m ost pr o n o u nce d 
i n s u bjects wit h baseli ne W B C a n d ne utr o p hil c o u nts at t he hi g her e n d of t h e n or mal ra n ge, a n d 
di d n ot a p pear t o pr o gr essi vel y w ors e n o ver t h e 7- da y d osi n g p eri o d. O nl y a fe w s u bjects ha d 
W B C or ne utr o p hil c o u nts bel o w t he l o wer li mit of n or mal o ver t he c o urse of t he st u d y, a n d 
t hese cas es wer e o bser v e d i n b ot h C C X 1 6 8 a n d place b o gr o u ps. T hese sli g ht c ha n ges i n W B C 
a n d ne utr o p hil c o u nts ma y be relate d t o t he p h ar m ac ol o g y of C C X 1 6 8 as a C 5a R bl oc ker. 
P har mac o ki netic r es ults ( mea n, S D) fr o m t he m ulti - d ose peri o d, o nce dail y d ose gr o u ps are 
s u m marize d i n Ta ble 7. C C X 1 6 8 was gi ve n as a d osi n g s ol uti o n t o s u bjects i n t hese t hree d ose 
gr o u ps. 
T a ble 7: 
S u m m ar y of P h ar m ac o ki netic Res ults ( me a n, S D) fr o m t he M ulti -d ose 
Peri o d, O nce D ail y D ose Gr o u ps, C L 0 0 1 _ 1 6 8 
P ar a meter  
1 m g o nce d ail y  
( N = 5) 
3  m g o nce d ail y  
( N = 6) 
1 0 m g o nce d ail y  
( N = 6) 
 
D a y 1  
D a y 7  
D a y 1  
D a y 7  
D a y 1  
D a y 7  
C ma x  ( n g/ m L) 
2. 5 9 ( 1. 1 2)  
2. 8 6 ( 1. 0 9)  
9. 9 4 ( 3. 6 9)  
1 3. 5 ( 4. 3 0)  
1 9. 2 ( 8. 4 6)  
3 1. 4 ( 9. 4 9)  
T ma x ( hr)  
1. 5 ( 0. 5 0)  
1. 0 ( 0. 0)  
1. 3 ( 0. 4 2)  
1. 2 ( 0. 2 6)  
1. 8 ( 0. 6 9)  
1. 9 ( 0. 5 9)  
t1/ 2 ( hr) 
2. 6 6 ( 0. 8 4 0)  
1 2. 2 ( 1 3. 7)  
6. 1 2 ( 3. 1 5)  
7 1. 8 ( 4 0. 2)  
6. 1 4 ( 1. 5 3)  
1 6 2 ( 7 4. 7)  
C L/ F ( L/ hr)  
1 3 4 ( 7 1. 0)  
8 6. 3 ( 5 1. 2)  
9 6. 9 ( 3 4. 1)  
3 4. 1 ( 2 1. 5)  
1 0 2 ( 3 6. 6)  
1 7. 3 ( 6. 7 3)  
A U C 0 -ta u ( n g• hr/ m L) 
8. 7 2 ( 4. 5 6)  
1 1. 2 ( 5. 2 4)  
3 4. 4 ( 1 1. 2)  
4 9. 1 ( 1 6. 8)  
1 0 4 ( 4 5. 3)  
1 7 8 ( 5 1. 2)  
P har mac o ki netic r es ults ( mea n, S D) fr o m t he m ulti - d ose peri o d, t wice d ail y d ose gr o u ps are 
s u m marize d i n Ta ble 8.  C C X 1 6 8 was gi ve n as ca ps ules, si milar t o t he ca ps ules teste d i n t he 
c urre nt st u d y, t o s u bjects i n t hese t w o d ose gr o u ps. 
T a ble 8: 
S u m m ar y of P h ar m ac o ki netic Res ults ( me a n, S D) fr o m t he M ulti -d ose 
Peri o d, T wice D ail y D ose Gr o u ps, C L 0 0 1 _ 1 6 8 
P ar a meter  
3 0 m g t wice d ail y, 
D a y 1  
( N = 6) 
3 0 m g t wice d ail y, 
D a y 7  
( N = 6) 
5 0 m g t wice d ail y, 
D a y 1  
( N = 6) 
5 0 m g t wice d ail y, 
D a y 7  
( N = 6) 
 
A M D ose  
P M D ose  
A M 
D ose  
P M D ose  
A M D ose  
P M D ose  
A M 
D ose  
P M D ose  
C ma x  ( n g/ m L) 
9 7. 2 
( 1 6. 4) 
2 7 4 
( 6 9. 9) 
1 6 1 
( 2 2. 9) 
1 9 1 ( 6 0. 2)  
2 0 2 ( 6 6. 1)  
4 2 3 ( 2 0 4)  
4 2 5 
( 1 5 6) 
3 5 9 ( 1 3 9)  
T ma x ( hr)  
1. 8 ( 0. 4 1)  
2. 0 ( 0. 0)  
2. 3 ( 0. 5 2)  
2. 2 ( 0. 9 8)  
2. 0 ( 0. 0)  
2. 3 ( 0. 5 2)  
2. 3 
( 0.5 2)  
2. 8 ( 0. 9 8)  
t1/ 2 ( hr) 
3. 0 8 
( 0. 7 6 1) 
4. 2 0 
( 1. 0 1) 
5. 0 5 
( 1. 3 3) 
1 2 9 ( 3 0. 7)  
2. 8 3 
( 0. 5 8 2) 
4. 0 9 
( 0. 6 6 6) 
4. 8 1 
( 1. 3 5) 
1 2 0 
( 1 9. 5) 
C L/ F ( L/ hr)  
7 8. 4 
( 2 3. 3) 
4 1. 4 
( 1 4. 4) 
2 8. 7 
( 1 0. 2) 
5. 8 6 
( 2. 0 9) 
6 2. 5 
( 2 0. 1) 
3 7. 3 
( 1 4. 7) 
1 9. 4 
( 7. 6 3) 
4. 0 5 
( 1. 0 2) 
A U C 0-ta u 
( n g• hr/ m L) 
3 8 0  
( 8 9. 3) 
6 9 5 ( 1 7 1)  
8 8 0 ( 2 3 0)  
9 6 6 ( 2 4 3)  
8 2 0  
( 2 7 4) 
1 4 0 0 
( 7 2 8) 
2 3 4 0 
( 8 8 5) 
2 1 8 0 
( 8 1 1) 
A U C 0- ta u is A U C0- 1 2 ( Da y 1) or A U C1 2 -2 4 ( Da y 7; s h o w n as A U C0- 1 2 i n Wi n N o nli n data file aft er 
a dj ust me nt of start ti me).  
F oll o wi n g a d mi nistrati o n at 1 t o 1 0 m g C C X 1 6 8 Q D f or 7 da ys, m ea n e x p os ures as meas ure d b y 
A U C 0- ta u ra n ge d fr o m 8. 7 2 t o 1 0 4 n g• hr/ m L o n Da y 1 a n d fr o m 1 1. 2 t o 1 7 8 n g• hr/ m L o n D a y 7, 
w hile C ma x  ra n ge d fr o m 2. 5 9 t o 1 9. 2 n g/ m L o n Da y 1 a n d 2. 8 6 t o 3 1. 4 n g/ m L o n D a y 7.    


C he m o Ce ntr y x, I n c.                            C O N FI D E N TI A L                                Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8                                                                                                                    A me n d me nt 4. 0 
                                                                   Pa ge 4 5 of 1 1 3                               1 8 Se pte m ber 2 0 1 5 
F oll o wi n g BI D a d mi nistrati o n at 3 0 t o 5 0 m g, mea n e x p os ures as meas ure d b y A U C 0- ta u ra n ge d 
fr o m 3 8 0 t o 1 4 0 0 n g• hr/ m L o n D a y 1 a n d fr o m 8 8 0 t o 2 3 4 0 n g• hr/ m L o n Da y 7, w hile Cma x 
ra n ge d fr o m 9 7. 2 t o 4 2 3 n g/ m L o n Da y 1 a n d 1 6 1 t o 4 2 5 n g/ m L o n Da y 7.  
C C X 1 6 8 le vels decli ne d i n a bi p hasic e x p o ne ntial ma n ner, wit h a ra pi d distri b uti o n p hase a n d a n 
a p pare nt mea n plas ma ter mi nal t 1/ 2 ra n gi n g fr o m 1 2. 2 t o 1 6 2 hr f oll o wi n g o nce dail y 
a d mi nistrati o n.  
Re peat a d mi nistrati o n f or se ve n da ys of C C X 1 6 8 res ulte d i n m o derate acc u m ulati o n (at 1 a n d 3 
m g Q D) or hi g h  acc u m ulati o n ( 1 0 m g Q D or 3 0 – 5 0 m g BI D) of C C X 1 6 8, c o nsiste nt wit h t he 
o bser ve d ter mi nal h alf -lif e i n t hese d osi n g gr o u ps. After Q D d osi n g, t he e x p os ure of C C X 1 6 8 as 
meas ure d b y C ma x  a n d A U C is a p pr o xi matel y d os e pr o p orti o nal i n t he 1 – 1 0 m g d ose ra n ge. 
After BI D d osi n g, t he e x p os ure i ncrease of C C X 1 6 8 statisticall y a p pears t o be li nearl y 
pr o p orti o nal f or 3 0 m g a n d 5 0 m g d oses. Base d o n t he meas ure d tr o u g h c o nce ntrati o ns, i n 
ge n eral, C C X 1 6 8 a p p ear e d t o reac h stea d y state after 3 - 4 da ys of d osi n g. 
F o o d e ffect w as als o i n vesti gate d b y c o m p ari n g t h e Da y 7 0- 1 2 h (faste d) a n d 1 2-2 4 h (fe d) 
para meters. N o si g nifica nt effects wer e see n i n eit her d ose gr o u p.  
C C X 1 6 8- M 1, a m o n o h y dr o x ylati o n meta b olite of C C X 1 6 8, was f o u n d t o be a maj or circ ulati n g 
meta b olite i n h u m a ns. F oll o wi n g 7 da ys of oral a d mi nistrati o n of C C X 1 6 8 at 3 0 m g a n d 5 0 m g 
BI D, t he A U C 0- 2 4 f or C C X 1 6 8- M 1 was 1 6 0 0 a n d 2 3 4 0 n g• hr/ m L, res pecti v el y. T h ese val u es 
re pres e nt 8 5 % a n d 5 2 %, res pecti vel y, of t h e le vels of C C X 1 6 8 meas ure d at t h ose d oses. Its 
e x p os ure was a de q u atel y c o vere d i n rats a n d c y n o m ol g us m o n ke ys i n t he t o xic ol o g y st u dies t hat 
s u p p ort t his P hase 2 cli nical trial. 
Ste ps 1 a n d 2 of t his st u d y ( C L 0 0 2 _ 1 6 8) h a ve b ee n c o m plete d. Res ults are s u m marize d i n t he 
I n v esti gat or’s Br oc h ure. T he t hir d ste p of t his cli nic al tri al C L 0 0 2 _ 1 6 8 is o n g oi n g. O ne 
s u bject i n Ste p 3 h a d a seri o us u ne x pecte d s us pecte d a d vers e re acti o n of i ncre ase d li ver 
a n d p a ncre atic e nz y mes. T he tre at me nt assi g n me nt re m ai ns bli n de d, si nce Ste p 3 is still 
o n g oi n g. A n 8 0- ye ar ol d m a n wit h a me dic al hist or y of alc o h ol a b use, s m o ki n g, a n d 
ele v ate d al k ali ne p h os p h at ase a n d G G T at b as eli ne, h a d a n i ncre ase i n he p atic 
tr a ns a mi n ases, G G T, al k ali ne p h os p h at ase, t ot al a n d direct bilir u bi n, a n d p a ncre atic 
a m yl ase st arti n g a p pr o xi m at el y 3 w ee ks af ter st arti n g st u d y me dic ati o n a n d I V 
c ycl o p h os p h a mi de, s ulf a met h o x az ole pl us tri met h o pri m, a n d p a nt o pr az ole. T he s u bject 
e x perie nce d v o miti n g, f ati g ue, re d uce d a p petite, a n d icter us si nce D a y 2 3. N o fe ver or 
e osi n o p hili a w as prese nt. A b d o mi n al ultr as o n o gr a p h y s h o w e d he p atic ste at osis a n d 
p ossi ble ve n o -occl usi ve dise ase. St u d y me dic ati o n a n d c ycl o p h os p h a mi de w ere st o p pe d o n 
D a y 2 6. T here w as n o e vi de nce of acti ve vir al he p atitis (i ncl u di n g C M V). A b d o mi n al C T 
d o ne o n D a y 3 1 s h o w e d si g ns of mi ni m al c h ol a n gitis, a n d n o si g n of c h olest asis or 
p a ncre atitis. Li ver bi o ps y o n D a y 3 4 s h o w e d a mi xe d p atter n of i nj ur y wit h wi del y res ol ve d 
he p atitis a n d persiste nt c h ol a n gitic/c h olest atic c o m p o ne nts, c o nsiste nt wit h dr u g -i n d uce d 
eti ol o g y. T he p atie nt’s c o n diti o n i m pr o ve d a n d he w as disc h ar ge d fr o m h os pit al o n D a y 4 1.  
1. 4.  
R ati o n ale f or t he St u d y  
A A V sta n dar d t h era p y i n cl u des c ycl o p h os p ha mi d e (I V or oral, alt h o u g h I V is preferre d b eca us e 
of a l o wer c u m ulati ve d ose  a n d l o wer t o xicit y), a n d oral c ortic oster oi ds, ta pere d o ver a p eri o d of 
ti me. Se vere diseas e warr a nts a d diti o n of I V c ortic oster oi ds a n d/ or plas ma e x c ha n ge. Res ults 


C he m o Ce ntr y x, I n c.                            C O N FI D E N TI A L                                Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8                                                                                                                    A me n d me nt 4. 0 
                                                                   Pa ge 4 6 of 1 1 3                               1 8 Se pte m ber 2 0 1 5 
fr o m rece nt cli nical trials (J o nes et al., 2 0 1 0 a n d St o ne et al. , 2 0 1 0) t hat ha v e atte m pte d t o 
eli mi nate c yc l o p h os p ha mi de use, i n dicate d t hat t he i nci de nce of a d v erse e ve nts a n d t he m ortalit y 
rate r e mai n hi g h i n s u bjects w h o di d n ot recei v e c ycl o p h os p h a mi de b ut rit u xi ma b . Hi g h d ose 
c ortic oster oi d use li kel y c o ntri b utes si g nifica ntl y t o t his hi g h m or bi dit y a n d m o rtalit y rates. 
T heref ore, t he m e dical n ee d f or alter nati ve t h era pies re mai ns hi g h a n d it is i m p orta nt t o fi n d 
wa ys t o re d uce or eli mi nate c ortic oster oi d use i n t he treat me nt of A A V.  
Base d o n e nc o ura gi n g res ults fr o m precli nical st u dies i n a h u ma n C 5a R k n o c k-i n m o use m o del 
of A A R V ( des cri be d i n s ecti o n 1. 2. 1. 2 , as well as t he I n v esti gat or’s Br oc h ure), C C X 1 6 8 has t he 
p ote ntial t o be a c ortic ost er oi d s pari n g or c ortic ost er oi d re place me nt t hera p y f or t his disease, 
wi t h p ote ntial safet y a n d t olera bilit y a d v a nta ges. He nce, t h e cli nical h y p ot hesis of t he trial is t o 
test t he feasi bilit y of usi n g C C X 1 6 8 as a c ortic oster oi d s pari n g or re place m e nt t hera p y d uri n g t he 
peri o d of i n d ucti o n of r e missi o n of A A V. Res ults fr o m a P h ase 1 cli nical trial c o n d ucte d i n 
h u ma n v ol u nteers i n dicat e d t hat C C X 1 6 8 was well t olerate d wit h a p har mac o ki netic pr ofile 
le n di n g it t o t wice dail y oral d osi n g, i n or d er t o pr o vi de s ufficie nt C 5a R plas ma c o vera ge. 
T heref ore, t he rati o nale f or t his P hase 2  st u d y is t o deter mi ne w het h er C C X 1 6 8 is safe a n d w ell 
t olerate d a n d s h o ws e vi de nce of effi cac y, b ase d o n its p ote ntial t o be c ortic oster oi d-s pari n g, after 
oral a d mi nistrati o n of C C X 1 6 8 f or 8 4 c o nsec uti ve da ys t o s u bjects wit h A A V.  
2.  
O B J E C TI V E S 
2. 1.  
Pri m ar y O bje cti ve 
T he pri mar y safet y o bjecti ve of t his st u d y is t o e v al uate t he safet y a n d t olera bilit y of C C X 1 6 8 i n 
s u bjects wit h A A V o n bac k gr o u n d c ycl o p h os p h a mi de or rit u xi ma b treat me nt.  
T he pri mar y efficac y o bj ecti ve is t o e val uate t h e efficac y of C C X 1 6 8 base d o n  t he Bir mi n g ha m 
Vasc ulitis Acti vit y Sc ore ( B V A S) . 
2. 2.  
Sec o n d ar y O bjecti ves  
T he sec o n d ar y o bjecti ves of t he st u d y i ncl u d e t he f oll o wi n g: 
1.  E val uati o n of t he efficac y of C C X 1 6 8 c o m p are d t o sta n dar d of care ( S O C) base d o n c ha n ges 
i n re nal disease acti vit y p ara meters: 
a. e G F R ( M D R D s er u m cr eati ni ne e q uati o n);  
b.  He mat uria ( ce ntr al la b or at or y micr osc o pic c o u nt of uri nar y R B Cs); a n d 
c. Al b u mi n uria (first m or ni n g uri nar y al b u mi n:creati ni ne rati o);  
2.  Assess me nt of c ha n ges i n re nal i nfla m mat or y acti vit y base d o n uri nar y m o n oc yt e 
c hem oattracta nt pr otei n - 1 ( M C P 1):creati ni ne rati o a n d ser u m C-reacti ve pr otei n 
c o nce ntr ati o n wit h C C X 1 6 8 c o m pare d t o S O C ; 
3.  Assess me nt of t he feasi bilit y of re d u ci n g or eli mi nati n g t he use of c ortic ost er oi ds i n t he 
treat me nt of s u bjects wit h A N C A -ass ociat e d vasc ulitis ( A AV) wit h o ut t he nee d f or resc u e 
c ortic oster oi d meas ures wit h C C X 1 6 8 c o m pare d t o S O C ; 
4.  Assess me nt of healt h -rel ate d q ualit y- of-life c ha n ges base d o n S h ort F or m- 3 6 versi o n 2 ( S F-
3 6 v 2) a n d E ur o Q O L- 5 D- 5 L ( E Q - 5 D- 5 L) wit h C C X 1 6 8 c o m pare d t o S O C ; 


C he m o Ce ntr y x, I n c.                            C O N FI D E N TI A L                                Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8                                                                                                                    A me n d me nt 4. 0 
                                                                   Pa ge 4 7 of 1 1 3                               1 8 Se pte m ber 2 0 1 5 
5.  Assess me nt of c ha n ges i n Vasc ulitis Da ma ge I n d e x ( V DI) wit h C C X 1 6 8 c o m pare d t o S O C ; 
6.  Assess me nt of c ha n ges i n A N C A (a nti - P R 3 a n d a nti- M P O) wit h C C X 1 6 8 c o m pare d t o S O C ; 
7.  Assess me nt of c ha n ges i n p har mac o d y n a mics mar kers i n plas ma a n d uri ne wit h C C X 1 6 8 
c o m pare d t o S O C ; 
8.  E val uati o n of t he p har m ac o ki netic pr ofile of C C X 1 6 8 i n s u bjects wit h A A V. 
3.  
S T U D Y D E SI G N  
A A V sta n dar d t h era p y i n cl u des c ycl o p h os p ha mi d e or rit u xi ma b  a n d oral c ortic oster oi ds, ta pere d 
o ver a peri o d of ti me. Se vere disease warra nts a d diti o n of I V c ortic oster oi ds a n d/ or plas ma 
e x c ha n ge. Base d o n n u m er o us st u dies i n dicati n g t he i n v ol ve me nt of C 5a i n A A V, as w ell as 
c o m pelli n g r es ults wit h C C X 1 6 8 i n precli nical st u dies i n a h u ma n C 5a R k n oc k-i n m o use m o del 
of A A R V, C C X 1 6 8 has t he p ote ntial t o be a c ortic oster oi d s pari n g or c ortic oster oi d re place me nt 
t hera p y f or t his diseas e. He nce, t h e cli nical h y p ot hesis of t he trial is t o test t he feasi bilit y of usi n g 
C C X 1 6 8 as a c ortic oster oi d s pari n g or re place me nt t hera p y d uri n g i n d ucti o n of re missi o n of 
A A V. T his h y p ot h esi s will be teste d i n a t hree-ste p ma n ner i n t his ra n d o mize d, d o u ble- bli n d, 
place b o - c o ntr olle d, P has e 2 cli nical trial i n u p t o a p pr o xi matel y 6 0 s u bjects wit h ne w or rel a pse d 
A A V. Refer t o t he St u d y Sc he ma f or t he st u d y fl o w. 
Ste ps 1 a n d 2 of t he st u d y ha v e bee n c o m plete d. 
S T E P 1  
I n Ste p 1 of t h e trial, u p t o a p pr o xi matel y 1 2 s u bjects will be stratifie d t o o ne of t w o strata, eit her 
ne wl y di a g n ose d A A R V or rela pse d A A R V, a n d t he n ra n d o mize d t o C C X 1 6 8 or place b o (i n a 
2: 1 rati o). A t w o-t hir ds r e d uce d d ose of oral c orti c oster oi ds will be gi v e n t o s u bjects ra n d o mize d 
t o C C X 1 6 8 a n d a f ull d ose of oral c ortic oster oi ds t o s u bjects ra n d o mize d t o place b o. All 1 2 
s u bjects will recei ve I V c ycl o p h os p h a mi de treat m e nt, w hic h is part of sta n dar d t hera p y f or 
A A R V. If necessa r y, res c ue I V met h yl pre d nis ol o ne or oral resc u e ster oi ds  after D a y 1 s h o ul d be 
gi ve n acc or di n g t o s ecti o n 5. 1 0  t o s u bjects wit h w orse ni n g dis ease.  
Pr o gr essi o n t o Ste p 2 is de pe n de nt u p o n t he safet y a n d effi cac y of C C X 1 6 8 o bser ve d i n Ste p 1.  
A n e xter nal data m o nit ori n g c o m mittee ( D M C) will re vie w safet y d ata, i ncl u di n g res c ue I V or 
oral c ortic oster oi d use o v er t he c o urse of t he st u d y a n d a d vise t he S p o ns or re gar di n g pr o gressi o n 
fr o m eac h ste p t o t he ne xt i n t he st u d y (see s ecti o n 7. 6 ). 
•  If t here ar e m ore t ha n o n e s us pecte d u n e x pecte d seri o us a d vers e reacti o n ( S U S A R) m ost 
li kel y rel ate d t o C C X 1 6 8, as assesse d b y t he D M C, i n s u bjects recei vi n g C C X 1 6 8 i n 
Ste p  1, f urt her r ecr uit me nt of s u bjects will be s us pe n de d.  
•  If A A R V diseas e acti vit y i n t he maj orit y of s u bjects ( > 5 0 %) o n C C X 1 6 8 i n Ste p 1 is n ot 
c o ntr olle d wit h o ut t he nee d f or I V or oral resc u e c ortic oster oi d t hera p y, f urt her 
recr uit me nt of s u bjects will be s us pe n de d.  
•  If a cli nicall y si g nifica nt t olera bilit y iss ue pre ve nts f urt her d osi n g of C C X 1 6 8 at 3 0 m g 
t wice dail y, c o nsi der ati o n will be gi ve n t o testi n g a l o wer d ose of C C X 1 6 8. I n t his e v e nt, 
t he pr ot oc ol will be a me n de d t o s pecif y t he d etails. 


C he m o Ce ntr y x, I n c.                            C O N FI D E N TI A L                                Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8                                                                                                                    A me n d me nt 4. 0 
                                                                   Pa ge 4 8 of 1 1 3                               1 8 Se pte m ber 2 0 1 5 
•  T he P K pr ofile of C C X 1 6 8 3 0 m g t wice d ail y will be assesse d after at least 3 s u bjects 
ha ve recei ve d at least o n e d ose of C C X 1 6 8 or place b o. T he P K pr ofile ( C m a x , A U C) o n 
Da y 1 will be c o m par e d t o t hat o bser ve d i n healt h y v ol u nteers. If o n a vera ge C C X 1 6 8 
plas ma e x p os ure is m ore t ha n t wice t hat o bser v e d i n healt h y v ol u nteers, c o nsi derati o n 
will be gi ve n t o testi n g a l o wer d ose of C C X 1 6 8. I n t his e ve nt, t he pr ot oc ol will be 
a me n de d t o s pecif y t he d etails.  
S T E P 2  
Ste p 2 will be o pe ne d f or e nr oll me nt if b ot h of t he f oll o wi n g criteria a re met: 
1.  N ot m ore t ha n 1 S U S A R m ost li kel y relate d t o C C X 1 6 8, as assesse d b y t h e D M C, is 
o bser ve d i n s u bjects r ecei vi n g C C X 1 6 8 i n Ste p 1; 
2.  A A R V disease acti vit y is c o ntr olle d i n t he maj orit y of s u bjects ( > 5 0 %) recei vi n g C C X 1 6 8 i n 
Ste p 1, wit h o ut t he nee d f or I V or oral resc ue c ortic oster oi d t hera p y, as ass esse d b y t he 
D M C.  
I n Ste p 2 of t h e trial, u p t o a p pr o xi matel y 1 2 s u bjects will be stratifie d t o o ne of t w o strata, eit her 
ne wl y di a g n ose d A A R V or rela pse d A A R V, a n d t he n ra n d o mize d t o C C X 1 6 8 or place b o (i n a 
2: 1 rati o). Oral c ortic oster oi ds will n ot be gi ve n t o s u bjects ra n d o mize d t o C C X 1 6 8, b ut a f ull 
d ose of oral c ortic oster oi ds will be gi ve n t o s u bjects ra n d o mize d t o place b o. All 1 2 s u bjects will 
recei v e I V c ycl o p h os p ha mi de treat me nt. If n ecess ar y, res c ue I V met h yl pre d nis ol o ne or oral 
resc ue ster oi ds s h o ul d be gi ve n acc or di n g t o s ecti o n 5. 1 0  t o s u bjects wit h w orse ni n g disease.  
S T E P 3  
I n Ste p 3, a p pr o xi matel y 3 6 s u bjects will be stratifie d pri or t o ra n d o mizati o n base d o n t he 
f oll o wi n g stratificati o n fact ors: 
1.  Eit her ne wl y dia g n ose d A A V or rela pse d A A V; 
2.  Eit her M P O or P R 3 A N C A p ositi vit y; 
3.  Will recei ve eit her c ycl o p h os p ha mi de or rit u xi ma b as part of sta n d ar d of care treat me nt.  
F oll o wi n g str atificati o n, s u bjects will be  ra n d o mize d 1: 1: 1 t o o ne of t hree gr o u ps: 
Gr o u p A: C C X 1 6 8 pl us c ycl o p h os p h a mi de/rit u xi ma b wit h n o oral c ortic oster oi ds; 
Gr o u p B: Place b o pl us c ycl o p h os p h a mi de/rit u xi ma b pl us a f ull starti n g d ose of oral 
c ortic oster oi ds; 
Gr o u p C: C C X 1 6 8 pl us c ycl o p h os p h a mi d e/rit u xi ma b pl us a t w o -t hir ds re d uce d starti n g d ose of 
oral c ortic oster oi ds.  
If necessar y, resc u e I V m et h yl pr e d nis ol o ne or oral resc ue ster oi ds s h o ul d be gi v e n t o s u bjects 
wit h w orse ni n g dis ease. 
Ste p 3  will be o pe ne d f or e nr oll me nt if b ot h of t he f oll o win g criteria are met:  
1.  N ot m ore t ha n 1 S U S A R m ost li kel y relate d t o C C X 1 6 8, as assesse d b y t h e D M C, is 
o bser ve d i n s u bjects r ecei vi n g C C X 1 6 8 i n Ste p 2; 
2.  A A V disease acti vit y is c o ntr olle d i n t he maj orit y of s u bjects ( > 5 0 %) recei vi n g C C X 1 6 8 i n 
Ste p 2, wit h o ut t he nee d f or I V or oral resc ue c ortic oster oi d t hera p y. 


C he m o Ce ntr y x, I n c.                            C O N FI D E N TI A L                                Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8                                                                                                                    A me n d me nt 4. 0 
                                                                   Pa ge 4 9 of 1 1 3                               1 8 Se pte m ber 2 0 1 5 
I n or der t o pr ot ect t he bli n di n g, a d o u ble- d u m m y desi g n will be utilize d. T he st u d y dr u g a n d 
ot her me dicati o n f or re n al vasc ulitis will be ta ke n as descri be d i n s ecti o ns 5. 3 , 5. 9 , 5. 1 0 , 1 1. 5  a n d 
1 1. 6 . 
T he d osi n g p eri o d is 8 4 da ys i n all t hr ee ste ps of t h e st u d y. F oll o wi n g t h e 8 4- da y d osi n g p eri o d, 
t here will be a n 8 4- da y f oll o w-u p peri o d. All s u bjects will visit t he st u d y ce nter d uri n g t he 
scree ni n g p eri o d, a n d o n Da ys 1, 2, 8, 1 5, 2 2, 2 9, 4 3, 5 7, 7 1, 8 5, 9 9, 1 1 3, 1 4 1, a n d 1 6 9. S u bjects 
will n ot ta ke st u d y me dicati o n i n t he m or ni n g of t he da ys of visits t o t he st u d y ce nter, a n d will be 
i nstr ucte d t o ta ke t he me dicati o n w hile at t he st u d y ce nter after all st u d y pr oce d ures f or t he d a y 
ha ve bee n c o m plete d. F or t he ot her st u d y da ys, st u d y me dicati o n will be ta ke n at h o me i n t he 
m or ni n g wit hi n 1 h o ur after brea kfast a n d i n t he e ve ni n g wit hi n 1 h o ur after di n ner.  
T he scree ni n g peri o d will be ke pt as s h ort as p ossi ble i n or der n ot t o dela y i nitiati o n of treat me nt. 
T he scree ni n g peri o d m ust n ot e x cee d 1 4 da ys. Scree ni n g pr o ce d ures will i ncl u de de m o gra p hi cs, 
me dical hist or y, m e dicati o n hist or y, p h ysical e x a mi nati o n a n d vital si g ns, ser u m c he mistr y, 
he mat ol o g y, P T, a n d a P T T, u ri nal ysis (i ncl u di n g h e mat uria a n d pr otei n ur ia [ A C R] assess me nt), 
E C G , c hest X ra ys, vir al scree ni n g, ser ol o g y a n d c o m ple me nt meas ure me nts (if n ot d o ne wit hi n 
t he pre vi o us 1 2 m o nt hs), e G F R ass ess me nt, A N C A meas ure m e nt (i n direct i m m u n ofl u oresce nce 
test f or P-A N C A a n d C -A N C A, as well as E LI S A tests f or P R 3 a n d M P O), re n al bi o ps y (if 
necessar y f or st u d y eli gi bilit y), a n d B V A S assess me nt. La b orat or y res ults fr o m t he l ocal 
la b orat ories o btai ne d wit hi n 7 2 h o urs of scree ni n g are acce pta ble i n or der t o a v oi d u n necess ar y 
bl o o d dra ws. Eli gi ble s u bjects m ust be A N C A- p ositi ve, m ust ha ve at least o ne “ maj or” ite m, or 
at least 3 n o n- maj or  ite ms, or at least t w o re n al ite ms o n t he B V A S versi o n 3 (see secti o n 1 1. 3 ).  
Eli gi ble s u bjects will visit t he st u d y ce nt er o n D a y 1, after a n o ver ni g ht fast of at least 8 h o urs, 
f or p h ysical e x a mi nati o n a n d vital si g ns, ser u m c h e mistr y, he m at ol o g y, uri nal ysis (i ncl u di n g 
he mat uria, pr otei n uri a [ A C R], M C P- 1:creati ni ne rati o assess me nt), e G F R, A N C A (a nti - P R 3 a n d 
a nti-M P O) meas ur e me nt, a B V A S a n d V DI assess me nt, S F - 3 6 v 2 a n d E Q-5 D -5 L assess me nt , 
baseli ne p har m ac o ki netics a n d p har mac o d y na mics ( P K/ P D) bl o o d sa m ple c ollecti o n, a n d 
ra n d o mizati o n. Me dicati o n will be a d mi nistere d (I V) a n d dis pe ns e d (f or oral me dicati o ns). T he 
s u bjects will ta ke t he first d ose of C C X 1 6 8 or place b o, a n d pre d nis o ne or place b o w hile at t he 
st u d y ce nt er. T he s u bj ects will sta y at t he cli nic f or at least 6 h o urs after t he first d ose o n Da y 1 
f or saf et y o bs er vati o n a n d P K sa m ple c ollecti o n. A s u bject c o ul d be ke pt o ver ni g ht i n t he 
h os pital o n Da y 1, if n ecessar y. T his h os pital sta y w o ul d n ot be c o nsi dere d a seri o us a d v erse 
e ve nt, u nless ot her S A E criteria are met. T wi ce d ail y d osi n g of C C X 1 6 8 or place b o will c o nti n ue 
f or 8 4 da ys. At p ost-D a y 1 st u d y visits, st u d y me di cati o n will be a d mi nistere d acc o r di n g t o t he 
pr ot oc ol sc he d ule, bl o o d a n d uri ne sa m ples will be c ollecte d f or safet y a n d efficac y a n d P K/ P D 
meas ure me nts. B V A S assess me nts will be ma de o n Da ys 1,  2 9, 8 5, 1 1 3, a n d 1 6 9. V DI 
assess me nts will be ma de o n Da ys 1, 8 5, a n d 1 6 9. S F - 3 6 v 2 a n d E Q- 5 D-5 L i nstr u me nts will be 
c o m plete d o n Da ys 1, 2 9, 8 5, a n d 1 6 9 i n Ste p 3. P h ysical e x a mi nati o ns, b o d y s yste m re vie ws, 
a n d vital si g n assess me nts will be perf or me d t hr o u g h o ut t he st u d y. C o nc o mita nt me dicati o n a n d 
a d verse e ve nt ass ess me nts will be ma de at e ver y  st u d y visit.  
S u bjects will be ter mi nate d fr o m t he st u d y w he n all t he St u d y D a y 1 6 9 visit pr oce d ures ha ve 
bee n c o m plete d.  
T o t he e xte nt p ossi ble, a n y a d v erse e ve nts t hat are dee me d st u d y dr u g -rel ate d a n d are o n g oi n g at 
disc har ge will be f oll o w e d - u p t o res ol uti o n or u ntil a deter mi nati o n is ma de t hat t he u nres ol ve d 
e ve nt is sta ble. T he s u bject’s c o n diti o n will be e val uate d b y t h e I n vesti gat or at t he e n d of t he 


C he m o Ce ntr y x, I n c.                            C O N FI D E N TI A L                                Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8                                                                                                                    A me n d me nt 4. 0 
                                                                   Pa ge 5 0 of 1 1 3                               1 8 Se pte m ber 2 0 1 5 
cli nical trial ( Da y 1 6 9) a n d a p pr o priate sta n d ar d of care me dical treat me nt will be pr o vi de d t o all 
s u bjects as nee d e d. 
4.  
S T U D Y P O P U L A TI O N  
4. 1.  
Size of t he P o p ul ati o n  
T he  ai m is t o e nr oll u p t o a p pr oxi matel y si xt y ( 6 0) s u bjects f or t he st u d y. S u bjects w h o dr o p o ut 
of t he st u d y pre mat ur el y will n ot be re place d. T hese s u bjects will be part of t he i nte nt- t o-treat 
p o p ulati o n. S u bjects will n ot be all o we d t o partici p ate i n m ore t ha n o ne Ste p of t he st u d y.  
4. 2.  
I ncl usi o n Criteri a 
S u bjects m ust meet all of t he f oll o wi n g i ncl usi o n criteria i n or der t o e nter t h e st u d y:  
1.  Cli nical dia g n osis of gra n ul o mat osis wit h p ol ya n giitis  ( We ge ner’s), micr osc o pic p ol ya n giitis 
or re nal li mite d vasc ulitis, c o nsiste nt wit h C ha pel- Hill c o nse ns us defi niti o ns ( J e n nette et al., 
2 0 1 3 ); 
2.  Male a n d p ost me n o pa usal ( l ac k of me nses f or at least 2 years  wit h o ut a n alter n ati ve 
e x pl a n ati o n ) or s ur gicall y sterile fe male s u bj ects, a ge d at least 1 8 years, wit h ne w ( wit hi n 4 
wee ks pri or t o scree ni n g) or rela pse d A A V w h ere treat me nt wit h c ycl o p h os p ha mi de or 
rit u xi ma b w o ul d be re q uire d; If f e m ale u n der 5 0 ye ars, t he p ost me n o p a us al st at us s h o ul d 
be c o nfir me d b y t he rele v a nt h or m o n al test.  M ale s u bjects wit h part ners of c hil d beari n g 
p ote ntial ma y p artici pate i n t he st u d y if t he y ha d a vasect o m y at least 6 m o nt hs pri or t o 
ra n d o mizati o n or if a de q uate c o ntrace pti o n is use d d uri n g, a n d f or at least t he t hree m o nt hs 
after st u d y c o m pleti o n; A de q uate c o ntrace pti o n is defi ne d as res ulti n g i n a fail ure rate of less 
t ha n 1 % per year; A cce pt a ble met h o ds i ncl u de c o m bi ne d estr o g e n a n d pr o gest o ge n ( or al, 
i ntr a v a gi n al, or tr a ns der m al), or pr o g est o ge n- o nl y h or m o n al c o ntr ace pti o n ( or al, 
i nject a ble, or i m pl a nt a ble), i ntr a-uteri ne de vice, i ntr a uteri ne h or m o ne rele asi n g s yste m, 
bil ater al t u b al occl usi o n, v asect o mize d p art ner, or se x u al a bsti ne nce;  
3.  P ositi ve i n direct i m m u n ofl u oresce n ce (II F) test f or P-A N C A or C - A N C A, or p ositi ve E LI S A 
test f or a nti- pr otei nas e- 3 ( P R 3) or a nti-m yel o p er o xi dase ( M P O) at scree ni n g; If o nl y t he II F 
assa y is p ositi ve at scree ni n g, a n d n o n e of t he E LI S A tests, t here m ust be d oc u me ntati o n i n 
t he st u d y r ec or ds of a p ositi ve E LI S A ass a y i n t he past; 
4.  Ha ve at least o ne “ maj or ” ite m, or at least 3 n o n- maj or  ite ms, or at least 2 re nal ite ms  o n t he 
B V A S  versi o n 3 (s ee secti o n 1 1. 3 ); 
5.  e G F R ≥2 0 m L per mi n ut e ( M D R D); 
6.  Willi n g a n d a ble t o gi ve writte n I nf or me d C o nse nt a n d t o c o m pl y wit h t he re q uire me nts of 
t he st u d y pr ot oc ol; a n d 
7.  J u d ge d t o be ot her wise h ealt h y b y t h e I n vesti gat or, base d o n me dical hist or y, p h ysical 
e x a mi nati o n (i ncl u di n g electr ocar di o gra m [ E C G]), a n d cli nical la b orat or y a ssess me nts. 
S u bjects wit h cli nical la b orat or y val ues t hat are o utsi de of n or mal li mits ( ot her t ha n t h ose 
s pecifie d i n t he E x cl usi o n Criteria) a n d/ or wit h ot her a b n or m al cli nical fi n di n gs t hat are 
j u d ge d b y t he I n vesti gat or n ot t o be of cli nical si g nifica nce, m a y be e nt ere d i nt o t he st u d y. 


C he m o Ce ntr y x, I n c.                            C O N FI D E N TI A L                                Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8                                                                                                                    A me n d me nt 4. 0 
                                                                   Pa ge 5 1 of 1 1 3                               1 8 Se pte m ber 2 0 1 5 
4. 3.  
E xcl usi o n Criteri a  
1.  Se vere disease as d eter mi ne d b y r a pi dl y pr o gressi ve gl o mer ul o ne p hritis s uc h t hat 
c o m me nce me nt of re n al re place me nt t hera p y c o ul d be a ntici pate d wit hi n 7 da ys, al ve olar 
he m orr ha ge lea di n g t o Gra de 3 or hi g h er h y p o xia (i.e., decreas e d o x y ge n s at urati o n at rest, 
e. g., p uls e o xi meter < 8 8 % or P aO 2 ≤5 5 m m H g), he m o pt ysis, ra pi d- o ns et m o n o ne uritis 
m ulti ple x ( Gra de 3 or hi g her, lea di n g t o se v ere s y m pt o ms t hat li mit self care acti vities of 
dail y li vi n g or re q uiri n g a n assisti ve de vice), or ce ntral ner v o us s yste m i n v ol ve me nt; 
2.  A n y ot her m ulti -s yste m a ut oi m m u ne disease i ncl u di n g e osi n o p hilic gra n ul o mat osis wit h 
p ol ya n giitis  ( C h ur g Stra uss), s yste mic l u p us er yt h e mat os us, I g A vasc ulitis (He n oc h -
Sc h ö nlei n p ur p ura), r he u mat oi d vasc ulitis, Sj ö gre n’s disease, a nti- gl o mer ular base me nt 
me m bra ne disease, or cr y o gl o b uli ne mia;  
3.  Me dical hist or y of c o a g ul o pat h y or blee di n g dis or der; 
4.  Recei ve d c ycl o p h os p ha mi de wit hi n 1 2 wee ks pri or t o scree ni n g; if o n azat hi o pri ne, 
m yc o p h e n olate m ofetil, or met h otre x ate at t he ti me of scree ni n g, t hese dr u gs m ust be 
wit h dra w n pri or t o r ecei vi n g t he c ycl o p h os p ha mi de d ose o n Da y 1; 
5.  Recei ve d i ntra v e n o us c ortic oster oi ds, > 3 0 0 0 m g m et h yl pre d nis ol o ne e q ui vale nt, wit hi n 1 2 
wee ks pri or t o scree ni n g; 
6.  Ha ve bee n ta ki n g a n or al dail y d ose of a c ortic oster oi d of m ore t ha n 1 0 m g pre d nis o ne-
e q ui vale nt f or m or e t ha n 6 wee ks c o nti n u o usl y pri or t o t he scree ni n g visit. If o n a n oral 
c ortic oster oi d at a d ail y d ose of m ore t ha n 1 0 m g pre d nis o ne e q ui val e nt at t he ti me of 
scree ni n g, t he oral d ose n ee ds t o be re d uce d t o a d ail y d ose n ot e x cee di n g 1 0 m g pre d nis o ne-
e qui vale nt pri or t o Da y 1; 
7.  Recei ve d rit u xi ma b or ot her B-cell a nti b o d y wit hi n 5 2 wee ks of s cree ni n g or 2 6 wee ks 
pr o vi de d B cell rec o nstit uti o n has occ urre d (i.e., C D 1 9 c o u nt > 0. 0 1 x 1 0 9/ L); recei ve d a nti-
T N F treat me nt, a bat ace pt, ale mt uz u ma b, I VI g, beli m u ma b, t ociliz u ma b, or plas ma e x c ha n ge 
wit hi n 1 2 wee ks pri or t o scree ni n g; 
8.  S y m pt o matic c o n gesti ve heart f ail ure re q uiri n g prescri pti o n me dicati o n, cli nicall y e vi de nt 
peri p her al e de ma of car diac ori gi n, p o orl y-c o ntr olle d h y p erte nsi o n (s yst olic bl o o d press ure 
> 1 6 0 or diast olic bl o o d press ure > 1 0 0), hist or y of u nsta ble a n gi na, m y ocar dial i nfarcti o n or 
str o ke wit hi n 6 m o nt hs pri or t o scree ni n g; 
9.  Hist or y or prese n ce of a n y f or m of ca n cer wit hi n t he 5 years pri or t o scree ni n g, wit h t he 
e x ce pti o n of e x cise d basal cell or  s q ua m o us cell carci n o ma of t h e s ki n, or cer vical carci n o ma 
i n sit u or breast car ci n o ma i n sit u t hat has bee n e x cise d or res ecte d c o m plet el y a n d is wit h o ut 
e vi de nce of l ocal r ec urre nce or metast asis;  
1 0.  Prese nce of t u ber c ul osis base d o n c hest X ra ys perf or me d d uri n g s cree ni n g as part of t he 
B V A S assess me nt.  
1 1.  P ositi ve H B V, H C V, or HI V viral scree ni n g test;  
1 2.  A n y i nf ecti o n re q uiri n g a nti bi otic treat me nt wit hi n 4 wee ks pri or t o scree ni n g (e x ce pt f or 
pr o p h ylactic treat me nt f or P ne u m ocystis c ari nii  p ne u m o nia [ P C P] or treat me nt f or s us pecte d 
i nfecti o n t hat i nstea d t ur ns o ut t o be a c o nse q ue n ce of A N C A vasc ulitis, e. g., p ne u m o nitis); 


C he m o Ce ntr y x, I n c.                            C O N FI D E N TI A L                                Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8                                                                                                                    A me n d me nt 4. 0 
                                                                   Pa ge 5 2 of 1 1 3                               1 8 Se pte m ber 2 0 1 5 
1 3.  Recei ve d a li ve v acci ne wit hi n 4 wee ks pri or t o s cree ni n g;  
1 4.  W B C c o u nt less t ha n 4 0 0 0/ μ L , or ne utr o p hil c o u nt less t ha n 2 0 0 0/ μ L, or l y m p h oc yte c o u nt 
less t ha n 1 0 0 0/ μ L;  
1 5.  He m o gl o bi n less t ha n 9 g/ d L ( or 5. 5 6 m m ol/ L) at s cree ni n g;  
1 6.  E vi de nce of h e patic disease; A S T, A L T, al kali ne p h os p hatase, or bilir u bi n > 3 x t he u p per 
li mit of n or mal; 
1 7.  Pr ot hr o m bi n ti me ( P T) or partial t hr o m b o plasti n ti me ( P T T) a b o ve t he n or mal refere nce 
li mit; 
1 8.  Cli nicall y si g nifica nt a b n or mal E C G d uri n g s cree ni n g, e. g., Q Tc F greater t ha n 4 5 0 msec; 
1 9.  Partici pate d i n a n y cli nical st u d y of a n i n vesti gati o nal pr o d uct wit hi n 3 0 da ys pri or t o 
scree ni n g or wit hi n 5 half-li ves after ta ki n g t he last d ose; a n d 
2 0.  Hist or y or prese n ce of a n y me dical c o n diti o n or disease w hic h, i n t he o pi ni o n of t he 
I n v esti gat or, ma y place t he s u bject at u nacce pt a ble ris k f or st u d y partici p ati o n. 
4. 4.  
Re m o v al of S u bjec ts fr o m T her a p y of Assess me nt 
S u bjects ma y be t er mi nat e d earl y fr o m t he st u d y f or a n y of t he f oll o wi n g reas o ns: 
1.  S u bject re q uest:  S u bjects ma y wit h dra w t heir c o nse nt t o partici pate i n t he st u d y at a n y ti me 
wit h o ut prej u dice.   
2.  I n v esti gat or re q u est:  T he I nvesti gat or ma y wit h dra w a s u bject if, i n his/ her cli nical 
j u d g me nt, it is i n t he best i nterest of t he s u bject or if t he s u bject ca n n ot c o m pl y wit h t he 
pr ot oc ol.   
3.  S p o ns or re q uest. 
I n t he e ve nt of wit h dra w al fr o m t he st u d y pri or t o t he Da y 8 5 visit, t he tests a n d e val uati o ns 
liste d f or St u d y Da y 8 5 s h o ul d be carrie d o ut as p art of t he Earl y Ter mi nati o n visit, w he ne ver 
p ossi ble. F or s u bjects w h o wit h dra w aft er D a y 8 5, t he Da y 1 6 9 st u d y pr oce d ures s h o ul d be 
perf or me d. T he S p o ns or s h o ul d be n otifie d of all st u d y wit h dra wals i n a ti mel y ma n n er.  
I n t he e ve nt of tr eat me nt fail ure w h ere resc u e c orti c oster oi d t hera p y is n ee d e d, t he st u d y dr u g 
( C C X 1 6 8 or place b o) a n d bli n de d pre d nis o ne/ place b o will be disc o nti n ue d, a n d a p pr o pri ate 
o pe n- la bel sta n d ar d of care meas ur es will be ta ke n. H o we ver, t he s u bject will be as ke d t o re mai n 
i n t he st u d y a n d c o m plet e all re mai ni n g st u d y visits. If t his is n ot p ossi ble, a n atte m pt will be 
ma de t o c o m plete all pr o ce d ures s c he d ule d f or t he Da y 8 5 visit (if t he resc u e e ve nt occ urre d 
pri or t o t he Da y 8 5 visit) a n d Da y 1 6 9 (if t h e res c u e e ve nt occ urre d aft er t he Da y 8 5 visit). 
5.  
S T U D Y M E DI C A TI O N/ T R E A T M E N T  
5. 1.  
Pr o d uct C h ar acteristics  
C C X 1 6 8 will be a d mi nistere d as h ar d gelati n ca ps ules c o ntai ni n g 1 0 m g C C X 1 6 8. T he ca ps ules 
are ma n ufact ur e d u n der c G M P. All d oses of st u d y me dicati o n will be a d mi nistere d orall y. T he 
C C X 1 6 8 ca ps ules will be s u p plie d t o t he st u d y ce nters i n plastic b ottles c o ntai ni n g 3 0 ca ps ules.  


C he m o Ce ntr y x, I n c.                            C O N FI D E N TI A L                                Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8                                                                                                                    A me n d me nt 4. 0 
                                                                   Pa ge 5 3 of 1 1 3                               1 8 Se pte m ber 2 0 1 5 
5. 2.  
R a n d o miz ati o n a n d Met h o d of Tre at me nt Assi g n me nt  
Eli gi ble s u bjects will be e nr olle d, strati fie d i n t w o strata: ( 1) ne wl y di a g n ose d ( wit hi n 4 wee ks of 
scree ni n g) or ( 2) rela ps e d A A V f or Ste ps 1 a n d 2, a n d t he t hree stratificati o n fact ors f or Ste p 3 
(i.e., ne wl y dia g n ose d or rela psi n g disease, P R 3 or M P O A N C A, a n d c ycl o p h os p ha mi de or 
rit u xi ma b use), a n d t he n ra n d o mize d t o o ne of t he t w o treat me nt gr o u ps i n a rati o of 2: 1, 
C C X 1 6 8: place b o f or Ste ps 1 a n d 2, a n d o ne of t hree treat me nt gr o u ps i n a rati o of 1: 1: 1, 
C C X 1 6 8 pl us n o pre d nis o ne: Place b o: C C X 1 6 8 pl us re d uce d pre d nis o ne i n Ste p 3. 
Ra n d o mizati o n will be perf or me d ce ntrall y via a n i nteracti ve v oice/ we b res p o nse s yste m 
(I V R S/I W R S). I n or der t o pr otect t he bli n di n g, t h e ra n d o mizati o n sc he d ule will n ot be accessi ble 
t o st u d y pers o n n el directl y i n v ol ve d i n t he st u d y.   
5. 3.  
D oses a n d Re gi me ns  
T here will be t w o gr o u ps i n eac h of t he first t w o ste ps of t he st u d y:   
•  Gr o u p A: C C X 1 6 8 3 0 m g t wice dail y  
•  Gr o u p B: Place b o t wice dail y 
T here will be t hr ee gr o u ps i n t he t hir d ste p of t he st u d y: 
•  Gr o u p A: C C X 1 6 8 3 0 m g t wice dail y pl us n o pre d nis o ne  
•  Gr o u p B: Place b o t wic e dail y 
•  Gr o u p C: C C X 1 6 8 3 0 m g t wice dail y pl us l o w d ose pre d nis o ne 
T he st u d y will be d o u ble- bli n d, d o u ble- d u m m y, i. e., place b o ca ps ules will be i de ntical i n 
a p pear a nce t o t he C C X 1 6 8 ca ps ules, a n d pre d nis o ne ca ps ules will als o ha ve matc hi n g place b o 
ca ps ules .  
S T E P 1  
I n Ste p 1, t he st u d y dr u g a n d ot her me dicati o n f or re nal vas c ulitis will be ta ke n as f oll o ws b y 
st u d y s u bjects: 
•  Gr o u p A ( 3 0 m g C C X 1 6 8 t wice dail y):  
− T hree 1 0 - m g C C X 1 6 8 ca ps ules i n t he m or ni n g a n d 3 ca ps ules i n t he e v e ni n g, 
a p pr o xi matel y 1 2 h o urs after t he m or ni n g d ose, d ail y f or 8 4 d a ys.  
− Pre d nis o ne 2 0 m g orall y per da y if t he s u bject’s b o d y w ei g ht is ≥5 5 k g or 1 5 m g per 
da y if t h e s u bject’s b o d y wei g ht is < 5 5 k g, starti n g o n Da y 1 wit h ta peri n g acc or di n g 
t o t he pr ot oc ol-s pecifi e d sc he d ule (see secti o n 1 1. 5 ). 
− Pre d nis o ne matc hi n g place b o ca ps ules e q ui vale nt t o 4 0 m g orall y per da y if t he 
s u bject’s b o d y wei g ht is ≥5 5 k g or 3 0 m g per da y if t he s u bject’s b o d y wei g ht is < 5 5 
k g, starti n g o n D a y 1 wit h ta peri n g acc or di n g t o t he pr ot oc ol- s pecifie d sc h e d ule (see 
secti o n 1 1. 5 ). 
− C ycl o p h os p ha mi de I V will be gi ve n o n D a y 1, a n d als o o n Da ys 1 5, 2 9, a n d 5 7; d oses 
o n Da ys 8 5, 1 1 3, 1 4 1, a n d 1 6 9 will be gi ve n at t he discreti o n of t he Pri nci p al 
I n v esti gat or (see secti o n 1 1. 6  f or i nstr ucti o ns).  


C he m o Ce ntr y x, I n c.                            C O N FI D E N TI A L                                Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8                                                                                                                    A me n d me nt 4. 0 
                                                                   Pa ge 5 4 of 1 1 3                               1 8 Se pte m ber 2 0 1 5 
− Resc ue I V met h yl pr e d nis ol o ne s h o ul d be gi ve n acc or di n g t o s ecti o n 5. 1 0  t o s u bjects 
wit h w orse ni n g dis ease. 
•  Gr o u p B ( Place b o t wice dail y):  
− T hree place b o C C X 1 6 8 ca ps ules i n t he m or ni n g a n d 3 ca ps ules i n t he e v e ni n g, 
a p pr o xi matel y 1 2 h o urs after t he m or ni n g d ose, d ail y f or 8 4 d a ys. 
− Pre d nis o ne 6 0 m g orall y per da y if t he s u bject’s b o d y w ei g ht is ≥5 5 k g or 4 5 m g per 
da y if t h e s u bject’s b o d y wei g ht is < 5 5 k g, starti n g o n Da y 1 wit h ta peri n g acc or di n g 
t o t he pr ot oc ol-s pecifi e d sc he d ule (see secti o n 1 1. 5 ). 
− C ycl o p h os p ha mi de I V will be gi ve n o n D a y 1 a n d als o o n Da ys 1 5, 2 9, a n d 5 7; d oses 
o n Da ys 8 5, 1 1 3, 1 4 1, a n d 1 6 9 will be gi ve n at t he discreti o n of t he Pri nci p al 
I n v esti gat or (see secti o n 1 1. 6  f or i nstr ucti o ns).  
− Resc ue I V met h yl pr e d nis ol o ne s h o ul d be gi ve n acc or di n g t o s ecti o n 5. 1 0  t o s u bjects 
wit h w orse ni n g dis ease.  
S T E P 2  
I n Ste p 2, st u d y dr u g a n d ot her me dicati o n f or v as c ulitis will be ta ke n as f oll o ws b y st u d y 
s u bjects: 
•  Gr o u p A ( 3 0 m g C C X 1 6 8 t wice dail y):  
− T hree 1 0 - m g C C X 1 6 8 ca ps ules i n t he m or ni n g a n d 3 i n t he e ve ni n g, a p pr o xi matel y 
1 2 h o urs after t h e m or ni n g d ose, d ail y f or 8 4 da ys.  
− Pre d nis o ne matc hi n g place b o ca ps ules e q ui vale nt t o 6 0 m g orall y per da y if t he 
s u bject’s b o d y wei g ht is ≥5 5 k g or 4 5 m g per da y if t he s u bject’s b o d y wei g ht is < 5 5 
k g, starti n g o n D a y 1 wit h ta peri n g acc or di n g t o t he pr ot oc ol- s pecifie d sc h e d ule (see 
secti on 1 1. 5 ). 
− C ycl o p h os p ha mi de I V will be gi ve n o n D a y 1 a n d als o o n Da ys 1 5, 2 9, a n d 5 7; d oses 
o n Da ys 8 5, 1 1 3, 1 4 1, a n d 1 6 9 will be gi ve n at t he discreti o n of t he Pri nci p al 
I n v esti gat or (see secti o n 1 1. 6  f or i nstr ucti o ns).  
− Resc ue I V met h yl pr e d nis ol o ne s h o ul d be gi ve n acc or di n g t o s ecti o n 5. 1 0  t o s u bjects 
wit h w orse ni n g dis ease.    
•  Gr o u p B ( Place b o t wice dail y):  
− T hree place b o C C X 1 6 8 ca ps ules i n t he m or ni n g a n d 3 i n t he e ve ni n g, a p pr o xi matel y 
1 2 h o urs after t h e m or ni n g d ose, d ail y f or 8 4 da ys. 
− Pre d nis o ne 6 0 m g orall y per da y if t he s u bject’s b o d y w ei g ht is ≥5 5 k g or 4 5 m g per 
da y if t h e s u bject’s b o d y wei g ht is < 5 5 k g, starti n g o n Da y 1 wit h ta peri n g acc or di n g 
t o t he pr ot oc ol-s pecifi e d sc he d ule (see secti o n 1 1. 5 ). 
− C ycl o p h os p ha mi de I V will be gi ve n o n D a y 1 a n d als o o n Da ys 1 5, 2 9, a n d 5 7; d oses 
o n Da ys 8 5, 1 1 3, 1 4 1, a n d 1 6 9 will be gi ve n a t t he discreti o n of t he Pri nci p al 
I n v esti gat or (see secti o n 1 1. 6  f or i nstr ucti o ns).  


C he m o Ce ntr y x, I n c.                            C O N FI D E N TI A L                                Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8                                                                                                                    A me n d me nt 4. 0 
                                                                   Pa ge 5 5 of 1 1 3                               1 8 Se pte m ber 2 0 1 5 
− Resc ue I V met h yl pr e d nis ol o ne s h o ul d be gi ve n acc or di n g t o s ecti o n 5. 1 0  t o s u bjects 
wit h w orse ni n g dis ease. 
S T E P 3  
I n Ste p 3, st u d y dr u g a n d ot her me dicati o n f or v as c ulitis will be ta ke n as f oll o ws b y st u d y 
s u bjects:  
•  Gr o u p A ( 3 0 m g C C X 1 6 8 t wice dail y wit h n o c ortic oster oi ds):  
− T hree 1 0 - m g C C X 1 6 8 ca ps ules i n t he m or ni n g a n d 3 i n t he e ve nin g, a p pr o xi matel y 
1 2 h o urs after t h e m or ni n g d ose, d ail y f or 8 4 da ys.  
− Pre d nis o ne matc hi n g place b o ca ps ules e q ui vale nt t o 6 0 m g orall y per da y if t he 
s u bject's b o d y w ei g ht is ≥5 5 k g or 4 5 m g per da y if t he s u bject's b o d y wei g ht is < 5 5 
k g, starti n g o n D a y 1 wit h ta peri n g acc or di n g t o a pr ot oc ol- s pecifi e d sc he d ule (see 
secti o n 1 1. 5 ). 
− If i n t he c ycl o p h os p ha mi de strat u m, c ycl o p h os p ha mi de I V will be gi v e n o n Da y 1 
a n d als o o n Da ys 1 5, 2 9, 5 7, a n d 8 5 ; starti n g o n D a y 9 9 t hr o u g h Da y 1 6 8, all s u bjects 
will recei ve oral azat hi o pri ne at a tar get d ose of 2 m g/ k g/ da y (see secti o n 1 1. 6  f or 
i nstr ucti o ns). 
− If i n t he rit u xi ma b strat u m, rit u xi ma b I V will be gi ve n o n Da ys 1, 8, 1 5, a n d 2 2 ( 3 7 5 
m g / m2 at eac h ti me p oi nt; see secti o n 1 1. 7 ).  
− Resc ue I V met h yl pr e d nis ol o ne or oral ster oi ds s h o ul d be gi ve n acc or di n g t o secti o n 
5. 1 0  t o s u bjects wit h w orse ni n g disease.  
•  Gr o u p B ( Place b o t wice dail y): 
− T hree place b o C C X 1 6 8 ca ps ules i n t he m or ni n g a n d 3 i n t he e ve ni n g, a p pr o xi matel y 
1 2 h o urs after t h e m or ni n g d ose, d ail y f or 8 4 da ys. 
− Pre d nis o ne 6 0 m g orall y per da y if t he s u bject's b o d y w ei g ht is ≥5 5 k g or 4 5 m g per 
da y if t h e s u bject's b o d y wei g ht is < 5 5 k g, starti n g o n Da y 1 wit h ta peri n g acc or di n g 
t o a pr ot oc ol-s pecifie d sc he d ule (see secti o n 1 1. 5 ). 
− If i n t he c ycl o p h os p ha mi de strat u m, c ycl o p h os p ha mi de I V will be gi v e n o n Da y 1 
a n d als o o n Da ys 1 5, 2 9, 5 7, a n d 8 5; starti n g o n D a y 9 9 t hr o u g h Da y 1 6 8, all s u bjects 
will recei ve oral azat hi o pri ne at a tar get d ose of 2 m g/ k g/ da y (see secti o n 1 1. 6  f or 
i nstr ucti o ns). 
− If i n t he rit u xi ma b strat u m, rit u xi ma b I V will be gi ve n o n Da ys 1, 8, 1 5, a n d 2 2 ( 3 7 5 
m g / m2 at eac h ti me p oi nt; see secti o n 1 1. 7 ). 
− Resc ue I V met h yl pr e d nis ol o ne or oral ster oi ds s h o ul d be gi ve n acc or di n g t o secti o n 
5. 1 0  t o s u bjects wit h w orse ni n g disease.  
•  Gr o u p C ( 3 0 m g C C X 1 6 8 t wice dail y wit h re d uce d c ortic oster oi ds):  
− T hree 1 0 - m g C C X 1 6 8 ca ps ules i n t he m or ni n g a n d 3 ca ps ules i n t he e v e ni n g, 
a p pr o xi matel y 1 2 h o urs after t he m or ni n g d ose, d ail y f or 8 4 d a ys.  


C he m o Ce ntr y x, I n c.                            C O N FI D E N TI A L                                Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8                                                                                                                    A me n d me nt 4. 0 
                                                                   Pa ge 5 6 of 1 1 3                               1 8 Se pte m ber 2 0 1 5 
− Pre d nis o ne 2 0 m g orall y per da y if t he s u bject's b o d y w ei g ht is ≥5 5 k g or 1 5 m g per 
da y if t h e s u bject's b o d y wei g ht is < 5 5 k g, starti n g o n Da y 1, wit h ta peri n g acc or di n g 
t o t he pr ot oc ol-s pecifi e d sc he d ule (see secti o n 1 1. 5 ). 
− Pre d nis o ne matc hi n g place b o ca ps ules e q ui vale nt t o 4 0 m g orall y per da y if t he 
s u bject's b o d y w ei g ht is ≥5 5 k g or 3 0 m g per da y if t he s u bject's b o d y wei g ht is < 5 5 
k g, starti n g o n D a y 1, wit h ta peri n g acc or di n g t o a pr ot oc ol- s pecifi e d sc he d ule. 
− If i n t he c ycl o p h os p ha mi de strat u m, c ycl o p h os p ha mi de I V will be gi v e n o n Da y 1 
a n d als o o n Da ys 1 5, 2 9, 5 7, a n d 8 5; starti n g o n D a y 9 9 t hr o u g h Da y 1 6 8, all s u bjects 
will recei ve oral azat hi o pri ne at a tar get d ose of 2 m g/ k g/ da y (see secti o n 1 1. 6  f or 
i nstr ucti o ns). 
− If i n t he rit u xi ma b strat u m, rit u xi ma b I V will be gi ve n o n Da ys 1, 8, 1 5, a n d 2 2 ( 3 7 5 
m g / m2 at eac h ti me p oi nt; see secti o n 1 1. 7 ).  
− Resc ue I V met h yl pr e d nis ol o ne or oral c ortic oster oi ds s h o ul d be gi ve n acc or di n g t o 
secti o n 5. 1 0  t o s u bjects wit h w orse ni n g dis ease.  
All s u bjects will ta ke st u d y me dicati o n as i nstr uct e d o n t he da ys of visits t o t he st u d y ce nter. F or 
t he ot her st u d y d a ys, st u d y me dicati o n will be ta ke n at h o me as i nstr ucte d. F oll o wi n g t he 8 4- da y 
d osi n g peri o d, t here will be a n 8 4- da y f oll o w - u p p eri o d.   
S u bjects i n Gr o u p A i n Ste ps 1 a n d 2, a n d Gr o u ps A a n d C i n Ste p 3 ( 3 0 m g C C X 1 6 8) will 
recei v e o ne kit co ntai ni n g t w o b ottles of C C X 1 6 8 ca ps ules o n Da ys 1, 8, 1 5, a n d 2 2. S u bjects i n 
Gr o u p A i n Ste ps 1 a n d 2, a n d Gr o u ps A a n d C i n Ste p 3 will recei ve t w o kits c o ntai ni n g 2  
b ottles eac h of C C X 1 6 8 ca ps ules o n Da ys 2 9, 4 3, 5 7, a n d 7 1. S u bjects will be as ke d t o t a ke 3 
ca ps ules e v er y m or ni n g a n d 3 ca ps ules e v er y e v e ni n g as i nstr ucte d. S u bjects will be as ke d t o 
bri n g all b ottles, w het h er e m pt y or n ot, t o t he st u d y ce nter at eac h st u d y visit. Ca ps ules will be 
ta ke n wit hi n 1 h o ur after brea kf ast or di n ner wit h water, prefera bl y wit h 5 0 m L, b ut n ot t o 
e x cee d 1 0 0 m L. S u bjects i n Gr o u p A will als o ta ke pre d nis o ne a n d/ or matc hi n g place b o ca ps ules 
o nce dail y o ver t h e c o urs e of t he st u d y.  
S u bjects i n Gr o u p B ( place b o) will recei ve o n e kit c o ntai ni n g t w o b ottles of place b o C C X 1 6 8 
ca ps ules o n Da ys 1, 8, 1 5, a n d 2 2. S u bjects i n Gr o u p B will recei ve t w o kits c o ntai ni n g 2 b ottles 
eac h of pl ace b o C C X 1 6 8 ca ps ules o n Da ys 2 9, 4 3, 5 7, a n d 7 1. S u bjects will be as ke d t o ta ke 3 
ca ps ules e v er y m or ni n g a n d 3 ca ps ules e v er y e v e ni n g as i nst r ucte d. S u bjects will be as ke d t o 
bri n g all b ottles, w het h er e m pt y or n ot, t o t he st u d y ce nter at eac h st u d y visit. Ca ps ules will be 
ta ke n wit hi n 1 h o ur after brea kf ast or di n ner wit h water, prefera bl y wit h 5 0 m L, b ut n ot t o 
e x cee d 1 0 0 m L. S u bjects i n Gr o u p B will als o ta k e pre d nis o ne a n d/ or matc hi n g place b o ca ps ules 
o nce dail y o ver t h e c o urs e of t he st u d y.  
Pre d nis o ne will be gi v e n as ta blets, o vere n ca ps ulat e d wit h har d gelati n ca ps ules i n or der t o 
mai ntai n t he bli n di n g. T w o d ose stre n gt hs of pre d nis o ne will be pr o vi de d, 2 0 m g a n d 5 m g. 
Place b o pre d nis o ne will be gi v e n as matc hi n g har d gel ati n ca ps ules wit h i n ert filler. Eac h s u bject 
will recei ve 1 kit wit h 3 b ottles c o ntai ni n g pre d nis o ne or place b o pre d nis o ne o n St u d y Da ys 1, 8, 
1 5, 2 9, 4 3, 5 7, 8 5, 9 9, a n d 1 1 3. T he 2 0 m g pre d nis o ne a n d matc hi n g place b o b ottles will c o ntai n 
1 0 ca ps ules per b ottle. T he 5 m g pre d nis o ne a n d matc hi n g place b o b ottles will c o ntai n 3 0 
ca ps ules per b ottle. S u bjects will be pr o vi de d wit h detaile d i nstr ucti o ns re gar di n g t he n u m ber of 


C he m o Ce ntr y x, I n c.                            C O N FI D E N TI A L                                Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8                                                                                                                    A me n d me nt 4. 0 
                                                                   Pa ge 5 7 of 1 1 3                               1 8 Se pte m ber 2 0 1 5 
ca ps ules t o ta ke eac h d a y fr o m eac h b ottle. T he d ose of c ortic oster oi ds will be ta pere d o ver t h e 
c o urse of t he st u d y (see s ecti o n 1 1. 5  f or t he ta p eri n g sc h e d ule). 
If a s u bject is alr ea d y o n a d ose of or al c ortic oster oi ds of ≤ 1 0 m g pre d nis o ne-e q ui vale nt at t he 
ti me of scree ni n g, a n atte m pt will be ma de t o ta per t his n o n-st u d y c ortic oster oi d use t o zer o o ver 
a peri o d n ot t o e x cee d 6 wee ks, starti n g o n D a y 1 of t he st u d y. If ta peri n g t o zer o is u ns uccessf ul, 
t he s u bject will be mai ntai ne d o n t he l o west p ossi ble n o n-st u d y mai nte n a n ce c ortic ost er oi d d ose, 
b ut n ot greater t ha n 1 0 m g pr e d nis o ne- e q ui vale nt. 
C ycl o p h os p ha mi de I V will be gi ve n o n D a y 1 a n d als o o n Da ys 1 5, 2 9, 5 7, a n d 8 5 ; starti n g o n 
Da y 9 9 t hr o u g h Da y 1 6 8, all s u bjects will recei ve oral azat hi o pri ne at a tar get d ose of 2 
m g/ k g/ da y (see secti o n 1 1. 6  f or i nstr ucti o ns). 
Rit u xi ma b I V will be gi v e n o n Da ys 1 , 8, 1 5, a n d 2 2 ( 3 7 5 m g/ m2 at eac h ti me p oi nt; see secti o n 
1 1. 7 ).   
I V met h yl pre d nis ol o ne or oral pre d nis o n e, o nl y as resc ue t h era p y, will be gi ve n acc or di n g t o 
secti o n 5. 1 0 . 
5. 4.  
R ati o n ale f or D ose Selecti o n  
Si n gle d oses of 1 m g u p t o 1 0 0 m g C C X 1 6 8 were st u die d i n a P hase 1 st u d y ( C L 0 0 1 _ 1 6 8) i n 4 8 
healt h y v ol u nteers, a n d m ulti ple o nce dail y d oses of 1, 3, a n d 1 0 m g C C X 1 6 8 a n d m ulti ple t wice 
dail y d oses of 3 0 m g a n d 5 0 m g f or u p t o 7 d a ys were st u die d i n t he m ulti ple d ose peri o d of t he 
st u d y. A d ose of 3 0 m g C C X 1 6 8 t wice dail y h as bee n selecte d f or t his P hase 2 st u d y i n s u bjects 
wit h A A V, w hic h is l o w er t ha n t he ma xi m u m d ose of 5 0 m g t wice dail y st u die d i n st u d y 
C L 0 0 1 _ 1 6 8. All d oses u p t o 1 0 0 m g (si n gle d ose) a n d 5 0 m g t wice dail y f or 7 d a ys were w ell 
t olerate d wit h n o si g nifi ca nt safet y c o n cer ns. It is a ntici pate d t hat d ose re gi me ns of ≥ 3 0 m g 
C C X 1 6 8 t wice dail y w o ul d pr o vi de at least 9 5 % C 5a R c o vera ge o n bl o o d n e utr o p hils 
c o nti n u o usl y t hr o u g h o ut t he da y. T his le vel of C 5 a R c o vera ge is dee m e d a p pr o priate t o ac hie ve 
o pti mal p har mac ol o g y base d o n bl oc ka de of C 5a-i n d uce d C D 1 1 b u pre g ul ati o n a n d C 5a-i n d uce d 
mi grati o n of n e utr o p hils base d o n i n vitr o assa ys c o n d ucte d i n w h ole bl o o d sa m ples o btai ne d 
fr o m t he P hase 1 cli nical trial.   
Base d o n t h e g o o d saf et y pr ofile o bser v e d i n t he t o xic ol o g y st u dies, a n d t h e safet y a n d 
t olera bilit y res ults fr o m t he P hase 1 cli nical trial a n d t his cli nical trial s o far , t he ris k f or seri o us 
or u na ntici pate d u nt o war d e ve nts ass ociate d wit h C C X 1 6 8 occ urri n g i n t his cli nical trial is 
c o nsi dere d l o w. 
5. 5.  
Dr u g S u p pl y  
5. 5. 1.  
P ac k a gi n g a n d L a beli n g 
C C X 1 6 8 ca ps ules c o ntai ni n g 1 0 m g C C X 1 6 8 a n d i de ntical a p peari n g place b o ca ps ules will be 
pac ka ge d i n hi g h d e nsit y p o l yet h yle n e ( H D P E) b ottles wit h c hil d-resista nt scre w ca ps a n d 
pr o vi de d t o t he st u d y sites i n kits ( b o x es) c o ntai ni n g t w o b ottles of C C X 1 6 8 or place b o ca ps ules. 
Eac h b ottle will c o ntai n 3 0 ca ps ules. All b ottles will be la bele d a p pr o priatel y t o i n dicate, at a 
mi ni m u m, pr ot oc ol n u m ber, t he b ottle n u m ber, t h e st u d y dr u g, t h e c o nte nts, st ora ge c o n diti o ns, 
ca uti o nar y state m e nt t o kee p o ut of r eac h of c hil dre n, a n d t he e x pir y d ate. O ne kit will be 
dis pe nse d t o a s u bject o n St u d y Da ys 1, 8, 1 5, a n d 2 2. T w o kits will be dis pe nse d t o a s u bject o n 


C he m o Ce ntr y x, I n c.                            C O N FI D E N TI A L                                Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8                                                                                                                    A me n d me nt 4. 0 
                                                                   Pa ge 5 8 of 1 1 3                               1 8 Se pte m ber 2 0 1 5 
St u d y Da ys 2 9, 4 3, 5 7, a n d 7 1. All kits will be la bele d a p pr o priatel y t o i n dicate, at a mi ni m u m, 
pr ot oc ol n u m ber, t he st u d y dr u g, t h e c o nte nts, st ora ge c o n diti o ns, ca uti o nar y stat e me nt t o kee p 
o ut of reac h of c hil dr e n, a n d t he e x pir y dat e. 
Pre d nis o ne ca ps ules c o ntai ni n g eit her 2 0 m g or 5 m g pre d nis o n e a n d i de nti cal a p peari n g place b o 
ca ps ules will be pac ka ge d i n hi g h de nsit y p ol yet h yl e n e ( H D P E) b ottles wit h c hil d- resista nt scre w 
ca ps a n d pr o vi d e d t o t he st u d y sites. Eac h b ottle of 2 0 m g or m atc hi n g place b o ca ps ules will 
c o ntai n 1 0 ca ps ules. E ac h b ottle of 5 m g or mat c hi n g place b o ca ps ules will c o ntai n 3 0 ca ps ules. 
All b ottles will be la bele d a p pr o priatel y t o i n dicat e, at a mi ni m u m, pr ot oc ol n u m ber, t he b ottle 
letter, t he st u d y dr u g, t he c o nte nts, st ora ge c o n diti o ns, ca uti o nar y state me nt t o kee p o ut of reac h 
of c hil dre n, a n d t h e e x pir y dat e. T he pre d nis o ne a n d matc hi n g place b o b ottles will be pac ka ge d 
i n kits ( b o x es) c o ntai ni n g t hree b ottles of pre d nis o ne a n d/ or place b o, de p e n di n g o n treat me nt 
gr o u p assi g n m e nt. O ne kit will be dis pe nse d t o a s u bject o n St u d y Da ys 1, 8, 1 5, 2 9, 4 3, 5 7, 8 5, 
9 9, a n d 1 1 3. Detaile d d osi n g i nstr ucti o ns will be pr o vi de d t o all s u bjects. All kits will be la bele d 
a p pr o priatel y t o i n dicate, at a mi ni m u m, pr ot oc ol n u m ber, t he st u d y dr u g, t he c o nte nts, st ora ge 
c o n diti o ns, ca uti o nar y state me nt t o kee p o ut of reac h of c hil dre n, a n d t he e x pir y dat e. 
5. 5. 2.  
St or a ge 
C C X 1 6 8 a n d C C X 1 6 8 place b o ca ps ules, as well as pre d nis o ne a n d pre d nis o ne place b o ca ps ules 
will be st ore d acc or di n g t o la bel i nstr ucti o ns. Access s h o ul d be restricte d t o p har ma c y staff or t o 
t he desi g n ate d r es p o nsi ble me m ber of t h e I n vesti gat or’s staff, a n d t o t he st u d y m o nit or. T he 
I n v esti gat or a gr ees t hat n eit her s/ he n or a n y of t he st u d y staff will s u p pl y st u d y me dicati o n t o 
a n y p ers o ns ot her t h a n t h ose e nr olle d i n t he st u d y.   
5. 6.  
B li n di n g 
T his st u d y is d o u ble- bli n d, d o u ble- d u m m y. Bli n di n g of t he st u d y will be ac hie ve d b y t he 
f oll o wi n g meas ures: 
1.  T he st u d y dr u g b ottles a n d ca ps ule a p p eara nce f or C C X 1 6 8 a n d its matc hi n g place b o, as well 
as pre d nis o ne a n d its matc hi n g place b o, wi ll be id e ntical;  
2.  Li mite d access t o t he r a n d o mizati o n c o de; st u d y site pers o n nel, st u d y s u bj ects, pers o n nel 
res p o nsi ble f or st u d y m o nit ori n g, a n d bi ostatisticia ns a n d data ma n a gers i n v ol ve d i n data 
a nal ysis of t he st u d y will re mai n bli n de d t o treat m e nt assi g n me nt f or t he d urati o n of t he 
st u d y; 
3.  W hile la b orat or y p ers o n n el c o n d ucti n g t he P K ass a ys will n ot be bli n de d t o treat me nt 
assi g n me nt, u n bli n de d C C X 1 6 8 plas ma c o nce ntrati o n res ults will n ot be s hare d wit h t he 
st u d y site pers o n n el or t he st u d y staff wit h direct c o ntact wit h st u d y sites d uri n g t he st u d y;  
4.  Efficac y data t hat w o ul d p ote ntiall y be u n bli n di n g, i.e, a nti- P R 3 a n d a nti- M P O a nti b o dies, 
uri nar y M C P- 1:creati ni ne rati o, uri nar y A C R, W B C a n d ne utr o p hil c o u nt data wit hi n t he 
n or mal ra n ge,  a n d hs C R P data, will n ot be ma de a vaila ble t o st u d y site p ers o n nel, st u d y 
s u bjects, pers o n nel r es p o nsi ble f or st u d y m o nit ori n g, a n d bi ostatisticia ns a n d data ma na gers 
d uri n g t he st u d y u nless f or safet y m o nit ori n g. 
Treat me nt assi g n me nts f or i n di vi d ual s u bjects will re mai n bli n de d t o t he st u d y tea m,  
i n vesti gat ors, a n d s u bj ects u ntil after t he st u d y d at a base has b ee n clea n e d a n d l oc ke d. Desi g nate d 
st u d y staff will be pr o vi de d wit h i nstr ucti o ns re gar di n g h o w t o u n bli n d i n di vi d ual s u bject 


C he m o Ce ntr y x, I n c.                            C O N FI D E N TI A L                                Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8                                                                                                                    A me n d me nt 4. 0 
                                                                   Pa ge 5 9 of 1 1 3                               1 8 Se pte m ber 2 0 1 5 
treat me nt assi g n me nt; i n di vi d ual s u bject treat me nt assi g n me nt ma y be u n bli n de d o nl y i n t he case 
of a n a d verse e v e nt t hat r e q uires k n o wle d ge of t he st u d y me dicati o n recei ve d b y t h e s u bject i n 
or der t o pr o vi de a p pr o pri ate treat me nt or m a na ge me nt of t he a d verse e v e nt. T he st u d y m o nit or 
s h o ul d be n otifie d as s o o n as p ossi ble i n t he e ve nt t hat u n bli n di n g of a n i n di vi d ual s u bject’s 
treat me nt assi g n me nt occ urs pri or t o st u d y c o m pleti o n. 
A n e xter nal data m o nit ori n g c o m mittee ( D M C) will be c o nstit ute d pri or t o start of t he st u d y (see 
secti o n 7. 6 ). T he D M C me m bers will re vie w d ata peri o dicall y o ver t he c o urse of t he st u d y i n a n 
u n bli n de d ma n ner. T he D M C will pr o vi de rec o m me n dati o ns t o t he S p o ns or re gar di n g f urt her 
c o n d uct of t he st u d y. T he D M C will o perate acc or di n g t o a c h arter de vel o p e d pri or t o st u d y 
i nitiati o n.   
5. 7.  
Dr u g Acc o u nt a bilit y  
T he st u d y p h ar macist a n d i n vesti gat or m ust mai ntai n acc urate rec or ds of d ates a n d q ua ntities of 
pr o d uct(s) r ecei v e d, t o w h o m dis pe nse d (s u bject- b y -s u bject acc o u nti n g), a n d acc o u nts of a n y 
pr o d uct acci de ntall y or d eli beratel y destr o ye d. T h e I n vesti gat or m ust retai n all u n use d a n d/ or 
e x pire d st u d y s u p plies u ntil t he st u d y m o nit or has c o nfir me d t he acc o u nt a bilit y d ata.  
5. 8.  
Tre at me nt C o m pli a nce  
T he C C X 1 6 8 a n d C C X 1 6 8 place b o ca ps ules, a n d pre d nis o ne a n d pre d nis o ne place b o ca ps ules, 
will be self -a d mi nistere d b y partici pati n g st u d y s u bjects. T h e m or ni n g d oses of st u d y dr u g o n 
Da y 1 will be ta ke n i n t h e prese n ce of st u d y site p ers o n nel. S u bjects will be pr o vi de d wit h d osi n g 
i nstr ucti o ns at t he start of t he st u d y, a n d will be e n c o ura ge d b y st u d y site pers o n nel t o ta ke t he 
st u d y me dicati o n acc or di ng t o t he i nstr ucti o ns f or t he d urati o n of t he st u d y. S u bjects will be 
i nstr ucte d t o bri n g t he assi g ne d b ottles of st u d y m e dicati o n t o t he site staff at eac h st u d y visit, 
w het her e m pt y or n ot. T h e st u d y dr u g dis p e nse d will be c hec ke d f or a n y u n use d st u d y dr u g, a n d 
a ca ps ule c o u nt will be d o ne of a n y r e mai ni n g C C X 1 6 8 a n d pre d nis o ne st u d y me dicati o n. T his 
i nf or mati o n will be rec or de d i n t he C R F.  
C C X 1 6 8 plas ma c o nce ntrati o n meas ure m e nts o ver t he c o urse of t he st u d y ma y als o be use d t o 
assess s u bject c o m plia nce. A n y e ve nts of n o n-c o m plia nce t o t he pr ot oc ol will be d oc u me nte d i n 
t he st u d y r ec or ds. 
5. 9.  
C o nc o mit a nt Me dic ati o ns a n d Restricti o ns  
Use of a n y dr u g ot her t ha n pr ot oc ol-s pecifi e d C C X 1 6 8 or C C X 1 6 8 place b o, oral pre d nis o ne or 
pre d nis o ne place b o, I V c ycl o p h os p h a m i de or rit u xi ma b , or resc ue I V or oral c ortic oster oi ds t o 
treat A A V is pr o hi bite d o ver t he c o urse of t he 8 4- da y treat me nt peri o d. T his i ncl u des use of oral 
c yclo p h os p ha mi de, azat hi o pri ne, m yc o p h e n olate m ofetil, met h otre x ate, a nti -T N F  treat me nt, 
a batace pt, ale mt uz u ma b, I VI g, beli m u ma b, t ociliz u ma b, or ot her i m m u n os u p pressi ve a ge nts. 
H o we ver, s u bjects i n t he c ycl o p h os p ha mi de strat u m will ta ke oral azat hi o pri ne at a tar get d ose of 
2 m g/ k g/ da y fr o m D a y 9 9  t hr o u g h D a y 1 6 8. 
Pr o p h ylactic treat me nt f or oste o p or osis, gastr o pr ot ecti o n, P ne u m ocystis c ari nii  p ne u m o nia 
( P C P), a n d a nti-na usea m e dicati o n (e. g., o n d a nsetr o n), acc or di n g t o l ocal practice, is all o we d.  
S u bjects will  als o be e nc o ura ge d t o r e mai n o n sta ble d oses of all ot her c o n c o mita nt me dicati o n 
o ver t he c o urse of t he st u d y. If a s u bject is o n a d ose of oral c ortic oster oi ds of ≤ 1 0 m g 


C he m o Ce ntr y x, I n c.                            C O N FI D E N TI A L                                Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8                                                                                                                    A me n d me nt 4. 0 
                                                                   Pa ge 6 0 of 1 1 3                               1 8 Se pte m ber 2 0 1 5 
pre d nis o ne- e q ui vale nt at t he ti me of scree ni n g, a n atte m pt will be ma de t o ta per t h is 
c ortic oster oi d use t o zer o o ver a peri o d n ot t o e x cee d 6 wee ks starti n g at Da y 1. If ta peri n g t o 
zer o is u ns uccessf ul, t he s u bject will be mai ntai ne d o n t he l o west p ossi ble mai nte na nce 
c ortic oster oi d d ose, b ut n ot greater t h a n 1 0 m g pre d nis o ne- e q ui vale nt. 
All c o nc o mita nt me dicati o ns ta ke n d uri n g t he c o urse of t he st u d y m ust be rec or de d o n t he 
c o nc o mita nt me dicati o n pa ges of t he C R F. 
S u bjects will be recei vi n g v ari o us b ac k gr o u n d t her a pies i n t his tri al, e. g., rit u xi m a b, 
c ycl o p h os p h a mi de, az at hi o pri ne, met h otre x ate, a n d/ or m yc o p he n ol ate m ofetil. T here a re 
prec a uti o ns liste d i n t he prescri bi n g i nf or m ati o n f or e ac h of t hese dr u gs. T he l oc al 
prescri bi n g i nf or m ati o n f or e ac h b ac k gr o u n d dr u g s h o ul d be c o ns ulte d pri or t o its use, s o 
t h at t hese prec a uti o ns c a n be t a ke n i nt o c o nsi der ati o n. 
5. 1 0.  
Criteri a f or Resc ue C ortic oster oi d Use  
Use of r esc ue I V or hi g h d ose  oral c ortic ost er oi ds is dee me d a treat me nt fail ure a n d s h o u l d o nl y 
be use d u n der t h e f oll o wi n g cir c u msta nces: 
•  S uccessi ve deteri orati o n of e G F R o ver 3 da ys s u g ges ti n g t hat, i n t he vie w of t he pri nci pal 
i n vesti gat or, if n o e xtra i nter ve nti o n is ta ke n, t he re nal f u ncti o n w o ul d c o nti n ue t o 
deteri orat e;  
•  W orse ni n g of r e nal f u n cti o n as j u d ge d b y e G F R decrease of > 1 0 m L/ mi n fr o m baseli ne 
d uri n g t he 8 4- da y treat me nt peri o d or d uri n g t he 8 4- da y f oll o w- u p p eri o d; 
•  Persiste nce or n e w occ urre nce of a maj or n o n -re n al ite m as per B V A S, s uc h as ga n gre n e, 
s u d de n visi o n l oss, reti nal c ha n ges, se ns ori n e ural heari n g l oss, massi ve 
he m o pt ysis/al ve olar h e m orr ha ge, res pirat or y fail ure, ca r di o m y o p at h y, isc h e mic 
a b d o mi nal pai n i n dicati ve of mese nteric isc he mia, or ner v o us s yste m diseas e; or 
•  If, base d o n t h e st u d y site p h ysicia n’s assess me nt, it w o ul d be i n t he best i nterest of t he 
s u bject.  
T he reas o n f or r esc ue c ortic oster oi d use will be ca pt ure d o n t he C R F. 
T he sta n dar d I V r esc ue tr eat me nt will c o nsist of 5 0 0 m g met h yl pre d nis ol o ne gi v e n o nce d ail y f or 
3 da ys. T his will be f oll o we d b y oral c ortic oster oi ds acc or di n g t o t he l ocal sta n dar d of care. Hi g h 
d ose oral pre d nis o ne res c ue me dicati o n, e. g., 2 0 0 m g pre d nis o n e, wit h a p pr o priate ta peri n g, m a y 
als o be use d at t he I n v esti gat or’s discreti o n. 
If res c ue c ortic oster oi d tr eat me nt is necess ar y, it w o ul d be c o nsi dere d a treat me nt fail ure a n d 
st u d y dr u g ( C C X 1 6 8 or place b o) a n d bli n de d pre d nis o ne/ place b o will be disc o nti n ue d. T he 
s u bject will recei v e a p pr o priate o pe n-l a bel sta n dar d of care. N e vert h eless, t he s u bject will 
c o nti n ue t o be f oll o we d i n t he st u d y ( s ee secti o n 4. 4 ).   


C he m o Ce ntr y x, I n c.                            C O N FI D E N TI A L                                Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8                                                                                                                    A me n d me nt 4. 0 
                                                                   Pa ge 6 1 of 1 1 3                               1 8 Se pte m ber 2 0 1 5 
6.  
S T U D Y P R O C E D U R E S  
6. 1.  
Scree ni n g a n d  E nr oll me nt  
I nf or me d C o nse nt m ust be o btai ne d pri or t o perf or ma nce of a n y st u d y-s pecific tests or 
e val uati o ns. It is i m p orta nt t o c o m plete t he scree ni n g pr oce d ures i n t he s h ortest ti me p ossi ble t o 
all o w s u bjects t o start treat me nt. Wit hi n a peri o d n ot t o e x cee d 1 4 da ys pri or t o ra n d o mizati o n, 
s u bjects will u n der g o t h e f oll o wi n g e v al uati o ns t o deter mi ne t heir eli gi bilit y f or st u d y 
partici pati o n:  
•  De m o gra p hics, me di cal hist or y, a n d pri or a n d c o nc o mita nt me dicati o n usa ge; 
•  I n or der t o e x pe dite t he s cree ni n g pr ocess, bl o o d will be c ollecte d f or testi n g at t he l o cal 
la b orat or y f or t he f oll o wi n g:  
− Fasti n g bl o o d c he mistr y, he mat ol o g y, P T T a n d a P T T;  
− A n esti mate d gl o mer ular filtrati o n rate will be calc ulate d base d o n t he f oll o wi n g 
M o dificati o n of Diet i n Re nal Diseas e ( M D R D) st u d y e q u ati o n: 
e G F R ( m L/ mi n/ 1. 7 3 m 2) = 1 7 5 x (ser u m creati ni ne i n m g/ d L)-1. 1 5 4 x ( A ge) -0. 2 0 3 x 
( 0. 7 4 2 if fe male) x ( 1. 2 1 2 if Africa n-A meri ca n/ Blac k)  
− A N C A meas ur e me nt (i n direct i m m u n ofl uresce nce test f or P -A N C A a n d C -A N C A, as 
well as E LI S A test f o r P R 3 a n d M P O); 
− Vir ol o g y assess me nts as detaile d i n secti o n 7. 2. 2 ; 
− Ser ol o g y a n d c o m ple me nt assess me nts as detaile d i n  secti o n 7. 2. 2 . T hese tests d o n ot 
nee d t o be perf o r me d if r es ults are a v aila ble fr o m tests d o ne wit hi n t he past 1 2 
m o nt hs pri or t o t he Scree ni n g visit. Res ults fr o m t he pri or tests m ust be rec or de d i n 
t he e C R Fs.  
•  B V A S assess me nt (s ee s ecti o n 1 1. 3 ); t o be eli gi ble f or e nr oll me nt, a s u bject m ust ha ve: 
− At least o ne “ maj or ” ite m (see ite ms mar ke d wit h “ #” i n secti o n  1 1. 3 ), or 
− At least t hree n o n- m aj or ite ms o n t he B V A S, or 
− A t least t w o re nal ite ms d ue t o vasc ulitis, i.e.: 
o  H y p erte nsi o n ( diast olic B P > 9 5 m m H g),  
o  Pr otei n uria > 1 + o n uri n al ysis or > 0. 2 g/ g creati ni ne,  
o  He mat uria  (‘ m o der ate” o n uri nal ysis or ≥1 0 R B C per hi g h p o wer fiel d, us u all y 
acc o m pa ni e d b y R B C casts ),  
o  Ser u m creati ni ne ≥1 2 5 μ m ol/ L, or  
o  > 3 0 % rise i n creati ni ne or  > 2 5 % fall i n creati ni ne cleara nce;  
•  S u bjects will be as ke d t o v oi d t heir bla d ders c o m pletel y, a n d a re pres e ntati ve clea n catc h, 
mi dstrea m uri ne sa m ple will be c ollecte d:  
− T he l ocal la b or at or y will perf or m a uri nal ysis f or h e mat uria  a n d pr otei n uria; 


C he m o Ce ntr y x, I n c.                            C O N FI D E N TI A L                                Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8                                                                                                                    A me n d me nt 4. 0 
                                                                   Pa ge 6 2 of 1 1 3                               1 8 Se pte m ber 2 0 1 5 
•  A p h ysi cal e x a mi nati o n (e x cl u di n g ge nit o uri nar y, o p ht hal mic, a n d fe male breast 
assess me nts) will be perf or me d; b o d y wei g ht, hei g ht, a n d b o d y mass i n de x will be 
deter mi ne d;  
•  Vital si g ns (te m perat ure, bl o o d press ure, heart rate) will be meas ure d s u pi n e after at least 
3 mi n utes of rest; 
•  A 1 2 -lea d E C G will be r ec or de d a n d assess e d f or a n y cli nicall y si g nifica nt a b n or malit y; 
•  C hest X ra ys will be ac q uire d f or B V A S assess m e nt a n d e x cl usi o n of T B;  
•  If a re nal bi o ps y has bee n perf or me d wit hi n 4 wee ks pri or t o scree ni n g, t he sta n dar d 
Hist ol o g y F or m will be c o m plete d;  a c o p y of t he bi o ps y res ults will be ke pt i n t he st u d y 
rec or ds; 
•  After all scree ni n g pr o ce d ures ha ve b ee n c o m plete d, a n d t he s u bject s atisfies all 
eli gi bilit y criteria, t he st u d y sc h e d ule will be disc usse d wit h t h e s u bject a n d t he sc he d ule 
will be pr o vi de d t o t he s u bject t o e ns ure c o m plia nce wit h t he st u d y visits; 
•  If t he s u bject has b ee n ta ki n g mai nte na n ce oral c ortic oster oi ds of 1 0 m g pre d nis o ne-
e q ui vale nt or less at t he ti me of scree ni n g, i. e., c ortic oster oi ds t hat are n ot c o nsi dere d part 
of t he st u d y me di cati o n, t he s u bject will be pr o vi d e d wit h d ose ta peri n g i nstr ucti o ns t o a 
d ose of zer o o ver a peri o d n ot t o e x cee d 6 wee ks fr o m Da y 1.  
6. 2.  
St u d y D a y 1  
If eli gi ble f or t he st u d y, t he s u bject will visit t he st u d y ce nter o n Da y 1, after a n o ver ni g ht fast of 
at least 8 h o urs, f or t he f oll o wi n g pr oce d ures: 
•  A p h ysical e x a mi nati o n (e x cl u di n g ge nit o uri nar y, o p ht hal mic, a n d fe male breast 
assess me nts); b o d y wei g ht assess me nt will be perf or me d;  
•  Vital si g ns (te m perat ure, bl o o d p ress ure, heart rate); 
•  Bl o o d will be c ollecte d f or s hi p me nt t o t he ce ntral la b orat or y f or ser u m c h e mistr y, 
he mat ol o g y, hs C R P, A N C A (a nti- P R 3 a n d a nti- M P O E LI S A) m eas ure me nt, a n d P K 
baseli ne meas ur e me nts; t he s u bjects ma y eat after t he ser u m c he mistr y sa m ple  has bee n 
c ollecte d;  
•  A bl o o d sa m ple will als o be ta ke n f or a n e x pe dite d W B C c o u nt at t he l ocal la b orat or y i n 
or der t o deter mi ne t he a p pr o priate c ycl o p h os p ha mi de d ose  f or s u bjects w h o are i n t he 
c ycl o p h os p ha mi de strat u m (see secti o n 1 1. 6 ); 
•  B V A S assess me nt (s ee  s ecti o n 1 1. 3 ); 
•  V DI assess me nt (s ee secti o n  1 1. 4 ); 
•  S u bjects will be as ke d t o c o m plete t he S F 3 6 v 2 a n d E Q- 5 D- 5 L q u esti o n nai res; 
•  A n y pre -tr eat me nt a d v ers e e ve nts (fr o m ti me of t he scree ni n g visit) will be rec or d e d; 
•  T he s u bject will be ra n d o mize d; 
•  St u d y m e dicati o n ( 1 kit of C C X 1 6 8 or C C X 1 6 8 place b o, a n d 1 kit of pre d nis o ne a n d/ or 
pre d nis o ne place b o) will be pr o vi de d t o t he s u bject wit h d osi n g i nstr ucti o ns; 


C he m o Ce ntr y x, I n c.                            C O N FI D E N TI A L                                Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8                                                                                                                    A me n d me nt 4. 0 
                                                                   Pa ge 6 3 of 1 1 3                               1 8 Se pte m ber 2 0 1 5 
•  S u bjects will be as ke d t o v oi d t heir bla d ders c o m pletel y, a n d a re pres e ntati ve clea n catc h, 
mi dstrea m uri ne sa m ple will be c ollecte d:  
− A uri ne sa m ple will be se nt t o t he ce ntral la b orat or y f or uri nal ysis i ncl u di n g p H, 
s pecific gr a vit y, gl uc os e, nitrite, ket o nes, bilir u bi n, ur o bili n o ge n, R B Cs, a n d W B Cs, 
as well as q ua ntitati ve m eas ure m e nt of al b u mi n a n d creati ni ne t o calc ulate t he A C R;  
− T w o a d diti o nal uri ne sa m ples will be fr oze n a n d se nt t o t he ce ntral la b orat or y f or 
q ua ntitati ve M C P -1 a n d creati ni ne meas ure m e nts t o calc ulate t he M C P -1:creati ni ne 
rati o, a n d f or e x pl orat or y uri nar y mar k ers; 
•  T he s u bject will be as ke d t o ta ke t he first d ose of st u d y me dicati o n w hile at t he st u d y 
ce nter; t he C C X 1 6 8 or C C X 1 6 8 place b o ca ps ules will be ta ke n at least 3 0 mi n utes bef ore 
t he pre d nis o ne a n d/ or pre d nis o ne ca ps ules are ta k e n; 
•  T he ti me of t he d osi n g of C C X 1 6 8 or C C X 1 6 8 place b o , as w ell as t he ti me of d osi n g of 
pre d nis o ne or pr e d nis o ne place b o, will be rec or d e d;  
•  Bl o o d sa m ples will be c ollecte d at 0. 5, 1, 2, 3, 4, a n d 6 h o urs f oll o wi n g C C X 1 6 8 or 
C C X 1 6 8 place b o d osi n g f or plas ma C C X 1 6 8 c o nce ntrati o n meas ure me nts; t he act ual 
ti me of eac h bl o o d sa m pl e c ollecti o n will be rec or de d; 
•  All uri ne will be c ollecte d f or a peri o d of 6 h o urs f oll o wi n g C C X 1 6 8 or C C X 1 6 8 place b o 
d osi n g; t he t otal uri ne v ol u me will be meas ure d a n d rec or d e d, a n d a re pres e ntati ve uri ne 
sa m ple will be o btai ne d f or C C X 1 6 8 meas ure me nt; 
•  A bl o o d sa m ple will b e c ollecte d f or p h ar mac o d y na mic  mar ker meas ure m e nts;  
•  A sali va sa m ple will be c ollect e d f or ge netic mar k er assess me nts;  
•  C ycl o p h os p ha mi de I V d ose will be gi ve n after recei pt of t he l ocal W B C c o u nt (see  
secti o n 1 1. 6 ) t o s u bjects i n t he c ycl o p h os p ha mi de strat u m; T he start a n d e n d ti me s of t he 
i nf usio n will be rec or d e d;  
•  Rit u xi ma b will be gi ve n t o s u bjects i n t he rit u xi ma b strat u m  (see secti o n 1 1. 7 ); A d ose of 
3 7 5 m g / m2 will be gi ve n;  T he start a n d e n d ti mes of t he i nf usi o n will be rec or de d; 
•  A n y c ha n ges i n c o nc o mita nt me dicati o n use will be rec or de d;  
•  A n y p ost- d osi n g a d v erse e ve nts will be rec or de d;  
•  A s u bject c o ul d be ke pt o ver ni g ht i n t he h os pital o n Da y 1, if necessar y. T his h os pital 
sta y w o ul d n ot be c o nsi dere d a s eri o us a d v erse e v e nt, u nless ot her S A E criteria are met;  
•  After all st u d y pr oce d ures ha ve bee n c o m plete d, t he s u bject will be re mi n d e d t o: 
− C o me t o t he st u d y ce nt er f or t he Da y 2 st u d y visit t he ne xt da y, if n ot ke pt o ver ni g ht; 
− St ore t he st u d y me dicati o ns i n a c o ol a n d dr y place acc or di n g t o la b el i nstr ucti o ns f or 
t he d urati o n of t he st u d y;  
− Ta ke t he C C X 1 6 8 or C C X 1 6 8 place b o me di cati o n i n t he e ve ni n g a p pr o xi matel y 1 2 
h o urs after t h e m or ni n g d ose;  


C he m o Ce ntr y x, I n c.                            C O N FI D E N TI A L                                Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8                                                                                                                    A me n d me nt 4. 0 
                                                                   Pa ge 6 4 of 1 1 3                               1 8 Se pte m ber 2 0 1 5 
− N ot t o ta ke C C X 1 6 8 a n d pre d nis o ne me dicati o n o n t he m or ni n g of Da y 2, u nless t he 
visit is sc he d ule d i n t h e after n o o n, i n w hic h case C C X 1 6 8 a n d pre d nis o ne me dicati o n 
s h o ul d be ta ke n t he m or ni n g of D a y 2; a n d 
− C o nti n ue ta ki n g all t heir ot her c o nc o mita nt me dicati o ns as us ual. 
6. 3.  
St u d y D a y 2  
T he St u d y D a y 2 visit m ust occ ur o n t he sc h e d ule d date. T he s u bjects m ust fast o ver ni g ht f or at 
least 8 h o urs bef ore t he st u d y visit. D uri n g t his visit, t he f oll o wi n g st u d y pr oce d ures will be 
perf or me d:  
•  B o d y s yste m re vie w t o deter mi ne if t here were a n y c h a n ges fr o m Da y 1;  
•  All treat me nt -e mer ge nt a d verse e v e nts will be rec or de d;  
•  Vi tal si g ns (te m perat ure, bl o o d press ure, heart rate); 
•  Bl o o d will be c ollecte d f or s hi p me nt t o t he ce ntral la b orat or y f or ser u m c h e mistr y a n d 
he mat ol o g y; t he s u bjects ma y eat aft er t he ser u m c he mistr y sa m ple c ollecti o n;  
•  S u bjects will be as ke d t o v oi d t heir b la d ders c o m pletel y, a n d a re pres e ntati ve clea n catc h, 
mi dstrea m uri ne sa m ple will be c ollecte d:  
− A uri ne sa m ple will be se nt t o t he ce ntral la b orat or y f or uri nal ysis i ncl u di n g p H, 
s pecific gr a vit y, gl uc os e, nitrite, ket o nes, bilir u bi n, ur o bili n o ge n, R B Cs, an d W B Cs;  
•  T he s u bject will be as ke d t o ta ke t he m or ni n g d os es of st u d y me dicati o n w hile at t he 
st u d y ce nt er, u nless alr ea d y ta k e n; t he C C X 1 6 8 or C C X 1 6 8 place b o ca ps ul es will be 
ta ke n at least 3 0 mi n utes bef ore t h e pre d nis o ne a n d/ or pre d nis o ne place b o ca ps ules are 
ta ke n; 
•  A n y c ha n ges i n c o nc o mita nt me dicati o n use will be rec or de d; a n d  
•  After all st u d y pr oce d ures ha ve bee n c o m plete d, t he s u bject will be re mi n d e d t o: 
− C o me t o t he st u d y ce nt er f or t he Da y 8 st u d y visit; 
− St ore t he st u d y me dicati o ns i n a c o ol a n d dr y place acc or di n g t o la b el i nstr ucti o ns f or 
t he d urati o n of t he st u d y;  
− Ta ke t he C C X 1 6 8 or C C X 1 6 8 place b o me di cati o n as i nstr ucte d;  
− Ta ke t he pr e d nis o ne or pre d nis o ne place b o d ose as i nstr ucte d;  
− N ot t o ta ke C C X 1 6 8 a n d pre d nis o ne me dicati o n o n t he m or ni n g of Da y 8, u nless t he 
visit is sc he d ule d i n t he after n o o n, i n w hic h case C C X 1 6 8 a n d pre d nis o ne me dicati o n 
s h o ul d be ta ke n t he m or ni n g of D a y 8; a n d 
− C o nti n ue ta ki n g all t heir ot her c o nc o mita nt me dicati o ns as us ual. 


C he m o Ce ntr y x, I n c.                            C O N FI D E N TI A L                                Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8                                                                                                                    A me n d me nt 4. 0 
                                                                   Pa ge 6 5 of 1 1 3                               1 8 Se pte m ber 2 0 1 5 
6. 4.  
St u d y D a y 8  
T he St u d y D a y 8 visit m ust occ ur o n t he sc h e d ule d date. T he s u bjects m ust fast o ver ni g ht f or 
at least 8 h o urs bef ore t h e st u d y visit. D uri n g t his visit, t he f oll o wi n g st u d y pr oce d ures will 
be perf or me d:  
•  B o d y s yste m re vie w t o deter mi ne if t here were a n y c h a n ges fr o m Da y 2;  
•  All treat me nt -e mer ge nt a d verse e v e nts will be rec or de d;  
•  Vital si g ns (te m perat ure, bl o o d press ure, heart rate); 
•  Bl o o d will be c ollecte d f or s hi p me nt t o t he ce ntral la b orat or y f or ser u m c h e mistr y, 
he mat ol o g y, a n d hs C R P meas ure me nts; t he s u bjects ma y eat after t he ser u m c he mistr y 
sa m ple c ollecti o n; 
•  T he date a n d ti me of t he last d ose of C C X 1 6 8 or C C X 1 6 8 place b o will be rec or d e d;  
•  A bl o o d sa m ple will be c ollecte d f or C C X 1 6 8 plas ma c o nce ntrati o n meas ure me nt, a n d 
t he date a n d ti me of t he s a m ple c ollecti o n will be rec or de d; 
•  S u bj ects will be as ke d t o v oi d t heir bla d ders c o m pletel y, a n d a re pres e ntati ve clea n catc h, 
mi dstrea m uri ne sa m ple will be c ollecte d:  
− A uri ne sa m ple will be se nt t o t he ce ntral la b orat or y f or uri nal ysis i ncl u di n g p H, 
s pecific gr a vit y, gl uc os e, nitrite, ket o nes, bilir u bi n, ur o bili n o ge n, R B Cs, a n d W B Cs, 
as well as q ua ntitati ve m eas ure m e nt of al b u mi n a n d creati ni ne t o calc ulate t he A C R;  
− T w o a d diti o nal uri ne sa m ples will be fr oze n a n d se nt t o t he ce ntral la b orat or y f or 
q ua ntitati ve M C P -1 a n d creati ni ne meas ure m e nts  t o calc ulate t he M C P-1:creati ni ne 
rati o, a n d f or e x pl orat or y uri nar y mar k ers; 
•  Dr u g acc o u nta bilit y of C C X 1 6 8 or C C X 1 6 8 place b o b ottles; 
•  Dr u g acc o u nta bilit y of pr e d nis o ne or pre d nis o ne place b o b ottles; 
•  St u d y m e dicati o n ( 1 kit of C C X 1 6 8 or C C X 1 6 8 place b o, a n d 1 kit of pre d nis o ne a n d/ or 
pre d nis o ne place b o) will be pr o vi de d t o t he s u bject wit h d osi n g i nstr ucti o ns; t he s u bject 
will ta ke t he m or ni n g d os es fr o m t hese n e w kits w hile at t he st u d y site, u nless t he 
m or ni n g d oses h a ve alr ea d y bee n ta ke n b y t h e s u bject; 
•  If a s u bject is o n n o n-st u d y or al mai nte na nce c ortic oster oi ds, d ose ta peri n g t o zer o nee ds 
t o be atte m pte d, if feasi ble; 
•  Rit u xi ma b will be gi ve n t o s u bjects i n t he rit u xi ma b strat u m (see secti o n 1 1. 7 ); A d ose of 
3 7 5 m g/ m 2 will be gi ve n;  T he start a n d e n d ti mes of t he i nf usi o n will be rec or de d; 
•  A n y c ha n ges i n c o nc o mita nt me dicati o n use will be rec or de d; a n d  
•  After all st u d y pr oce d ures ha ve bee n c o m plete d, t he s u bject will be re mi n d e d t o: 
− C o me t o t he st u d y ce nt er f o r t he Da y 1 5 st u d y visit; 
− St ore t he st u d y me dicati o ns i n a c o ol a n d dr y place acc or di n g t o la b el i nstr ucti o ns f or 
t he d urati o n of t he st u d y;  


C he m o Ce ntr y x, I n c.                            C O N FI D E N TI A L                                Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8                                                                                                                    A me n d me nt 4. 0 
                                                                   Pa ge 6 6 of 1 1 3                               1 8 Se pte m ber 2 0 1 5 
− Ta ke t he C C X 1 6 8 or C C X 1 6 8 place b o me di cati o n as i nstr ucte d;  
− Ta ke t he pr e d nis o ne or pre d nis o ne place b o d ose as i ns tr ucte d; 
− N ot t o ta ke C C X 1 6 8 a n d pre d nis o ne me dicati o n o n t he m or ni n g of Da y 1 5, u nless t he 
visit is sc he d ule d i n t he after n o o n, i n w hic h case C C X 1 6 8 a n d pre d nis o ne me dicati o n 
s h o ul d be ta ke n t he m or ni n g of D a y 1 5;  
− Bri n g all st u d y m e dicati o n c o ntai ners t o t he ne xt st u d y visit, w het her e m pt y or n ot, 
a n d 
− C o nti n ue ta ki n g all t heir ot her c o nc o mita nt me dicati o ns as us ual. 
6. 5.  
St u d y D a y 1 5  
T he St u d y D a y 1 5 visit m ust occ ur o n t he sc he d ule d date. T he s u bjects m ust fast o ver ni g ht f or at 
least 8 h o urs bef ore t he st u d y visit. D uri n g t his visit, t he f oll o wi n g st u d y pr oce d ures will be 
perf or me d:  
•  A p h ysical e x a mi nati o n (e x cl u di n g ge nit o uri nar y, o p ht hal mic, a n d fe male breast 
assess me nts) will be perf or me d; b o d y wei g ht will als o be meas ure d;  
•  All treat me nt -e mer ge nt a d verse e v e nts will be rec or de d; 
•  Vital si g ns (te m perat ure, bl o o d press ure, heart rate); 
•  Bl o o d will be c ollecte d f or s hi p me nt t o t he ce ntral la b orat or y f or ser u m c h e mistr y, 
he mat ol o g y, a n d hs C R P meas ure me nts; t he s u bjects ma y eat after t he ser u m c he mistr y 
sa m ple c ollecti o n; 
•  A bl o o d sa m ple will als o be ta ke n f or a n e x pe dite d W B C c o u nt at t he l ocal la b orat or y i n 
or der t o deter mi ne t he a p pr o priate c ycl o p h os p ha mi de d ose (see s ecti o n 1 1. 6 ) f or s u bjects 
w h o are i n t he c ycl o p h os p ha m i de strat u m; 
•  S u bjects will be as ke d t o v oi d t heir bla d ders c o m pletel y, a n d a re pres e ntati ve clea n catc h, 
mi dstrea m uri ne sa m ple will be c ollecte d:  
− A uri ne sa m ple will be se nt t o t he ce ntral la b orat or y f or uri nal ysis i ncl u di n g p H, 
s pecific gr a vit y, gl uc os e, nitrite, ket o nes, bilir u bi n, ur o bili n o ge n, R B Cs, a n d W B Cs, 
as well as q ua ntitati ve m eas ure m e nt of al b u mi n a n d creati ni ne t o calc ulate t he A C R;  
− T w o a d diti o nal uri ne sa m ples will be fr oze n a n d se nt t o t he ce ntral la b orat or y f or 
q ua ntitati ve M C P -1 a n d creati ni ne meas ure m e nts t o calc ulate t he M C P -1:creati ni ne 
rati o, a n d f or e x pl orat or y uri nar y mar k ers; 
•  Dr u g acc o u nta bilit y of C C X 1 6 8 or C C X 1 6 8 place b o b ottles; 
•  Dr u g acc o u nta bilit y of pr e d nis o ne or pre d nis o ne place b o b ottles; 
•  St u d y m e dicati o n ( 1 kit of C C X 1 6 8 or C C X 1 6 8 place b o, a n d 1 kit of pre d nis o ne a n d/ or 
pre d nis o ne place b o) will be pr o vi de d t o t he s u bject wit h d osi n g i nstr ucti o ns; t he s u bject 
will ta ke t he m or ni n g d os es fr o m t hese kits w hile at t he st u d y site, u nless t he m or ni n g 
d oses ha ve alr ea d y b ee n t a ke n b y  t he s u bject; 


C he m o Ce ntr y x, I n c.                            C O N FI D E N TI A L                                Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8                                                                                                                    A me n d me nt 4. 0 
                                                                   Pa ge 6 7 of 1 1 3                               1 8 Se pte m ber 2 0 1 5 
•  If a s u bject is still o n n o n-st u d y oral mai nte na nce c ortic oster oi ds, d ose ta p eri n g t o zer o 
nee ds t o be atte m pte d, if feasi ble;  
•  C ycl o p h os p ha mi de I V d ose will be gi ve n after recei pt of t he l ocal W B C c o u nt (see 
secti o n 1 1. 6 ) t o s u bjects i n t he c ycl o p h os p ha mi de strat u m; T he start a n d e n d ti mes of t he 
i nf usi o n will be rec or d e d; 
•  Rit u xi ma b will be gi ve n t o s u bjects i n t he rit u xi ma b strat u m (see secti o n 1 1. 7 ); A d ose of 
3 7 5 m g / m2 will be gi ve n;  T he start a n d e n d ti mes of t he i nf usi o n will be rec or de d; 
•  A bl o o d sa m ple will be c ollecte d f or C C X 1 6 8 plas ma c o nce ntrati o n meas ure me nt; t he 
date a n d ti me of t he last d ose pri or t o sa m ple c oll ecti o n will be rec or de d; 
•  A n y c ha n ges i n c o nc o mita nt me dicati o n use will be rec or de d; a n d  
•  After all st u d y pr oce d ures ha ve bee n c o m plete d, t he s u bject will be re mi n d e d t o: 
− C o me t o t he st u d y ce nt er f or t he Da y 2 2 st u d y visit; 
− St ore t he st u d y me dicati o ns i n a c o ol a n d dr y place acc or di n g t o la b el i nstr ucti o ns f or 
t he d urati o n of t he st u d y;  
− Ta ke t he C C X 1 6 8 or C C X 1 6 8 place b o me di cati o n as i nstr ucte d;  
− Ta ke t he pr e d nis o ne or pre d nis o ne place b o d ose as i nstr ucte d;  
− N ot t o ta ke C C X 1 6 8 a n d pre d nis o ne me dicati o n o n t he m or ni n g of Da y 2 2, u nless t he 
visit is sc he d ule d i n t he after n o o n, i n w hic h case C C X 1 6 8 a n d pre d nis o ne me dicati o n 
s h o ul d be ta ke n t he m or ni n g of D a y 2 2; 
− Bri n g all st u d y m e dicati o n c o ntai ners t o t he ne xt st u d y visit, w het her e m pt y or n ot, 
a n d 
− C o nti n ue ta ki n g all t heir ot her c o nc o mita nt me dicati o ns as us ual. 
6. 6.  
St u d y D a y 2 2  
T he St u d y D a y 2 2 visit m ust occ ur wit hi n a +/- 2- da y wi n d o w of t he s c he d ule d date. D uri n g t his 
visit, t he f oll o wi n g st u d y pr oce d ur es will be perf or me d: 
•  B o d y s yste m re vie w t o deter mi ne if t here were a n y c h a n ges fr o m Da y 1 5 ;  
•  All treat me nt -e mer ge nt a d verse e v e nts will be rec or de d;  
•  Vital si g ns (te m perat ure, bl o o d press ure, heart rate); 
•  S u bjects will be as ke d t o v oi d t heir bla d ders c o m pletel y, a n d a re pres e ntati ve clea n catc h, 
mi dstrea m uri ne sa m ple will be c ollecte d:  
− A uri ne sa m ple will be se nt t o t he ce ntral la b orat or y f or uri nal ysis i ncl u di n g p H, 
s pecific gr a vit y, gl uc os e, nitrite, ket o nes, bilir u bi n, ur o bili n o ge n, R B Cs, a n d W B Cs; 
•  Dr u g acc o u nta bilit y of C C X 1 6 8 or C C X 1 6 8 place b o b ottles; 


C he m o Ce ntr y x, I n c.                            C O N FI D E N TI A L                                Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8                                                                                                                    A me n d me nt 4. 0 
                                                                   Pa ge 6 8 of 1 1 3                               1 8 Se pte m ber 2 0 1 5 
•  St u d y m e dicati o n ( 1 kit of C C X 1 6 8 or C C X 1 6 8 place b o) will be pr o vi de d t o t he s u bject 
wit h d osi n g i nstr ucti o ns; t he s u bject will ta ke t he m or ni n g d ose fr o m t his kit w hile at t he 
st u d y site, u nless t he m or ni n g d ose h as alrea d y bee n ta ke n b y t he s u bject; 
•  If a s u bject is still o n n o n-st u d y oral mai nte na nce c ortic oster oi ds, d ose ta p eri n g t o zer o 
nee ds t o be atte m pte d, if feasi ble;  
•  A bl o o d sa m ple will be c ollecte d f or C C X 1 6 8 plas ma c o nce ntrati o n meas ure me nt; t he 
date a n d ti me of t he last d ose pri or t o sa m ple c oll ecti o n will be rec or de d; 
•  Rit u xi ma b will be gi ve n t o s u bjects i n t he rit u xi ma b strat u m (see secti o n 1 1. 7 ); A d ose of 
3 7 5 m g/ m 2 will be gi ve n;  T he start a n d e n d ti mes of t he i nf usi o n will be rec or de d; 
•  A n y c ha n ges i n c o nc o mita nt me dicati o n use will be r ec or de d; a n d  
•  After all st u d y pr oce d ures ha ve bee n c o m plete d, t he s u bject will be re mi n d e d t o: 
− C o me t o t he st u d y ce nt er f or t he Da y 2 9 st u d y visit; 
− St ore t he st u d y me dicati o ns i n a c o ol a n d dr y place acc or di n g t o la b el i nstr ucti o ns f or 
t he d urati o n of t he st u d y;  
− Ta ke t he C C X 1 6 8 or C C X 1 6 8 place b o me di cati o n as i nstr ucte d;  
− Ta ke t he pr e d nis o ne or pre d nis o ne place b o d ose as i nstr ucte d; 
− N ot t o ta ke C C X 1 6 8 a n d pre d nis o ne me dicati o n o n t he m or ni n g of Da y 2 9, u nless t he 
visit is sc he d ule d i n t he after n o o n, i n w hic h case C C X 1 6 8 a n d pre d nis o ne me dicati o n 
s h o ul d be ta ke n t he m or ni n g of D a y 2 9;  
− Bri n g all st u d y m e dicati o n c o ntai ners t o t he ne xt st u d y visit, w het her e m pt y or n ot, 
a n d 
− C o nti n ue ta ki n g all t heir ot her c o nc o mita nt me dicati o ns as us ual. 
6. 7.  
St u d y D a y 2 9  
T he St u d y D a y 2 9 visit m ust occ ur wit hi n a +/- 2- da y wi n d o w of t he s c he d ule d date. T he 
s u bjects m ust fast o ver ni g ht f or at least 8 h o urs bef ore t he st u d y visit. D uri n g t his visit, t he 
f oll o wi n g st u d y pr oce d ur es will be perf or me d: 
•  A p h ysical e x a mi nati o n (e x cl u di n g ge nit o uri nar y, o p ht hal mic, a n d fe male breast 
assess me nts) will be perf or me d; b o d y wei g ht will als o be meas ure d;  
•  All treat me nt -e mer ge nt a d verse e v e nts will be rec or de d;  
•  Vital si g ns (te m perat ure, bl o o d press ure, heart rate); 
•  A 1 2 -lea d E C G will be r ec or de d a n d assess e d f or a n y cli nicall y si g nifica nt a b n or malit y; 
•  Bl o o d will be c ollecte d f or s hi p me nt t o t he ce ntral la b orat or y f or ser u m c h e mistr y, 
he mat ol o g y, a n d hs C R P meas ure me nts; t he s u bjects ma y eat after t he ser u m c he mistr y 
sa m ple c ollecti o n; 


C he m o Ce ntr y x, I n c.                            C O N FI D E N TI A L                                Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8                                                                                                                    A me n d me nt 4. 0 
                                                                   Pa ge 6 9 of 1 1 3                               1 8 Se pte m ber 2 0 1 5 
•  A bl o o d sa m ple will als o be ta ke n f or a n e x pe dite d W B C c o u nt at t he l ocal la b orat or y i n 
or der t o deter mi ne t he a p pr o priate c ycl o p h os p ha mi de d ose (see s ecti o n 1 1. 6 ) f or s u bjects 
w h o are i n t he c ycl o p h os p ha mi de strat u m; 
•  A bl o o d sa m ple will be c ollecte d f or p h ar mac o d y na mics mar ker m eas ure me nts; 
•  S u bjects will be as ke d t o v oi d t heir bla d ders c o m pletel y, a n d a re pres e ntati ve clea n catc h, 
mi dstrea m uri ne sa m ple will be c ollecte d:  
− A uri ne sa m ple will be se nt t o t he ce ntral la b orat or y f or uri nal ysis i ncl u di n g p H, 
s pecific gr a vit y, gl uc os e, nitrite, ket o nes, bilir u bi n, ur o bili n o ge n, R B Cs, a n d W B Cs, 
as well as q ua ntitati ve m eas ure m e nt of al b u mi n a n d creati ni ne t o calc ulate t he A C R;  
− T w o a d diti o nal uri ne sa m ples will be fr oze n a n d se nt t o t h e ce ntral la b orat or y f or 
q ua ntitati ve M C P -1 a n d creati ni ne meas ure m e nts t o calc ulate t he M C P -1:creati ni ne 
rati o, a n d f or e x pl orat or y uri nar y mar k ers; 
•  A N C A (a nti - P R 3 a n d a nti-M P O E LI S A) m eas ure me nt;  
•  B V A S assess me nt; c h est X ra ys will o nl y b e ac q uire d if dee me d cli nicall y n ecessar y b y 
t he Pri nci pal I n vesti gat or (see secti o n 1 1. 3 ); 
•  S u bjects will be as ke d t o c o m plete t he S F 3 6 v 2 a n d E Q- 5 D- 5 L q u esti o n naires; 
•  Dr u g acc o u nta bilit y of C C X 1 6 8 or C C X 1 6 8 place b o b ottles; 
•  St u d y m e dicati o n ( 2 kits of C C X 1 6 8 or C C X 1 6 8 place b o, a n d 1 kit of pre d nis o ne a n d/ or 
pre d nis o ne place b o) will be pr o vi de d t o t he s u bject wit h d osi n g i nstr ucti o ns; t he s u bject 
will ta ke t he m or ni n g d os es fr o m t hese kits w hile at t he st u d y site, u nless t he m or ni n g 
d oses ha ve alr ea d y b ee n t a ke n b y t he s u bject;  
•  If a s u bject is still o n n o n-st u d y oral mai nte na nce c ortic oster oi ds, d ose ta p eri n g t o zer o 
nee ds t o be atte m pte d, if feasi ble.  
•  Dr u g acc o u nta bilit y of pr e d nis o ne or pre d nis o ne place b o b ottles; 
•  C ycl o p h os p ha mi de I V d ose will be gi ve n after recei pt of t he l ocal W B C c o u nt (see 
secti o n 1 1. 6 ) t o s u bjects i n t he c ycl o p h os p ha mi de strat u m; T he start a n d e n d ti mes of t he 
i nf usi o n will be rec or d e d; 
•  A bl o o d sa m ple will be c olle cte d f or C C X 1 6 8 plas ma c o nce ntrati o n meas ure me nt; t he 
date a n d ti me of t he last d ose pri or t o sa m ple c oll ecti o n will be rec or de d; 
•  A n y c ha n ges i n c o nc o mita nt me dicati o n use will be rec or de d; a n d  
•  After all st u d y pr oce d ures ha ve bee n c o m plete d, t he s u bject wi ll be re mi n d e d t o: 
− C o me t o t he st u d y ce nt er f or t he Da y 4 3 st u d y visit; 
− St ore t he st u d y me dicati o ns i n a c o ol a n d dr y place acc or di n g t o la b el i nstr ucti o ns f or 
t he d urati o n of t he st u d y; 
− Ta ke t he C C X 1 6 8 or C C X 1 6 8 place b o me di cati o n as i nstr ucte d;  
− Ta ke t he  pr e d nis o ne or pre d nis o ne place b o d ose as i nstr ucte d; 


C he m o Ce ntr y x, I n c.                            C O N FI D E N TI A L                                Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8                                                                                                                    A me n d me nt 4. 0 
                                                                   Pa ge 7 0 of 1 1 3                               1 8 Se pte m ber 2 0 1 5 
− N ot t o ta ke C C X 1 6 8 a n d pre d nis o ne me dicati o n o n t he m or ni n g of Da y 4 3, u nless t he 
visit is sc he d ule d i n t he after n o o n, i n w hic h case C C X 1 6 8 a n d pre d nis o ne me dicati o n 
s h o ul d be ta ke n t he m or ni n g of D a y 4 3;  
− Bri n g all st u d y m e dicati o n c o ntai ners t o t he ne xt st u d y visit, w het her e m pt y or n ot, 
a n d 
− C o nti n ue ta ki n g all t heir ot her c o nc o mita nt me dicati o ns as us ual. 
6. 8.  
St u d y D a y 4 3  
T he St u d y D a y 4 3 visit m ust occ ur wit hi n +/- 2 d a ys of t he sc h e d ule d date. T he s u bje cts m ust 
fast o ver ni g ht f or at least 8 h o urs bef ore t he st u d y visit. D uri n g t his visit, t he f oll o wi n g st u d y 
pr oce d ur es will be perf or me d: 
•  B o d y s yste m re vie w t o deter mi ne if t here were a n y c h a n ges fr o m Da y 2 9;  
•  All treat me nt -e mer ge nt a d verse e v e nts will be rec or de d; 
•  Vital si g ns (te m perat ure, bl o o d press ure, heart rate); 
•  Bl o o d will be c ollecte d f or s hi p me nt t o t he ce ntral la b orat or y f or ser u m c h e mistr y a n d 
he mat ol o g y meas ure m e nts; t he s u bjects ma y eat after t he ser u m c he mistr y sa m ple 
c ollecti o n;  
•  S u bjects will  be as ke d t o v oi d t heir bla d ders c o m pletel y, a n d a re pres e ntati ve clea n catc h, 
mi dstrea m uri ne sa m ple will be c ollecte d:  
− A uri ne sa m ple will be se nt t o t he ce ntral la b orat or y f or uri nal ysis i ncl u di n g p H, 
s pecific gr a vit y, gl uc os e, nitrite, ket o nes, bilir u bi n, ur o bili n o ge n, R B Cs, a n d W B Cs; 
•  Dr u g acc o u nta bilit y of C C X 1 6 8 or C C X 1 6 8 place b o b ottles; 
•  St u d y m e dicati o n ( 2 kits of C C X 1 6 8 or C C X 1 6 8 place b o, a n d 1 kit of pre d nis o ne a n d/ or 
pre d nis o ne place b o) will be pr o vi de d t o t he s u bject wit h d osi n g i nstr ucti o ns; t h e s u bject 
will ta ke t he m or ni n g d os es fr o m t hese kits w hile at t he st u d y site, u nless t he m or ni n g 
d oses ha ve alr ea d y b ee n t a ke n b y t he s u bject;  
•  Dr u g acc o u nta bilit y of pr e d nis o ne or pre d nis o ne place b o b ottles; 
•  If a s u bject h as bee n o n n o n-st u d y oral mai nte na nce c ortic oster oi ds, d ose t a peri n g t o zer o 
nee ds t o be c o m plete d b y t his visit, if feasi ble;  
•  A bl o o d sa m ple will be c ollecte d f or C C X 1 6 8 plas ma c o nce ntrati o n meas ure me nt; t he 
date a n d ti me of t he last d ose pri or t o sa m ple c oll ecti o n will be rec or de d; 
•  A n y c ha n ges i n c o nc o mita nt me dicati o n use will be rec or de d; a n d  
•  After all st u d y pr oce d ures ha ve bee n c o m plete d, t he s u bject will be re mi n d e d t o: 
− C o me t o t he st u d y ce nt er f or t he Da y 5 7 st u d y visit; 
− St ore t he st u d y me dicati o ns i n a c o ol a n d dr y place acc or di n g t o la b el i nstr ucti o ns f or 
t he d urati o n of t he st u d y; 
− Ta ke t he C C X 1 6 8 or C C X 1 6 8 place b o me di cati o n as i nstr ucte d;  


C he m o Ce ntr y x, I n c.                            C O N FI D E N TI A L                                Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8                                                                                                                    A me n d me nt 4. 0 
                                                                   Pa ge 7 1 of 1 1 3                               1 8 Se pte m ber 2 0 1 5 
− Ta ke t he pr e d nis o ne or pre d nis o ne place b o d ose as i nstr ucte d; 
− N ot t o ta ke C C X 1 6 8 a n d pre d nis o ne me dicati o n o n t he m or ni n g of Da y 5 7, u nless t he 
visit is sc he d ule d i n t he after n o o n, i n w hic h case C C X 1 6 8 a n d pre d nis o ne me dicati o n 
s h o ul d be ta ke n t he m or ni n g of D a y 5 7;  
− Bri n g all st u d y m e dicati o n c o ntai ners t o t he ne xt st u d y visit, w het her e m pt y or n ot, 
a n d 
− C o nti n ue ta ki n g all t heir ot her c o nc o mita nt me dicati o ns as us ual. 
6. 9.  
St u d y D a y 5 7  
T he St u d y D a y 5 7 visit m ust occ ur wit hi n +/- 2 d a ys of t he sc h e d ule d date. D uri n g t his visit, t he 
f oll o wi n g st u d y pr oce d ur es will be perf or me d: 
•  A p h ysic al e x a mi nati o n (e x cl u di n g ge nit o uri nar y, o p ht hal mic, a n d fe male breast 
assess me nts) will be perf or me d; b o d y wei g ht will als o be meas ure d;  
•  All treat me nt -e mer ge nt a d verse e v e nts will be rec or de d;  
•  Vital si g ns (te m perat ure, bl o o d press ure, heart rate); 
•  Bl o o d will be c ollecte d f or s hi p me nt t o t he ce ntral la b orat or y f or ser u m creati ni ne a n d 
hs C R P meas ure me nt;  
•  A bl o o d sa m ple will als o be ta ke n f or a n e x pe dite d W B C c o u nt at t he l ocal la b orat or y i n 
or der t o deter mi ne t he a p pr o priate c ycl o p h os p ha mi de d ose (see s ecti o n 1 1. 6 ) f or s u bjects 
w h o are i n t he c ycl o p h os p ha mi de strat u m;  
•  S u bjects will be as ke d t o v oi d t heir bla d ders c o m pletel y, a n d a re pres e ntati ve clea n catc h, 
mi dstrea m uri ne sa m ple will be c ollecte d:  
− A uri ne sa m ple will be se nt t o t he ce ntral la b orat or y f or uri nal ysis i ncl u di n g p H, 
s pecific gr a vit y, gl uc os e, nitrite, ket o nes, bilir u bi n, ur o bili n o ge n, R B Cs, a n d W B Cs, 
as well as q ua ntitati ve m eas ure m e nt of al b u mi n a n d creati ni ne t o calc ulate t he A C R;  
− T w o a d diti o nal uri ne sa m ples will be fr oze n a n d se nt t o t he ce ntral la b orat or y f or 
q ua ntitati ve M C P -1 a n d creati ni ne meas ure m e nts t o calc ulate t he M C P -1:creati ni ne 
rati o, a n d f or e x pl orat or y uri nar y mar k ers; 
•  Dr u g acc o u nta bilit y of C C X 1 6 8 or C C X 1 6 8 place b o b ottles; 
•  St u d y m e dicati o n ( 2 kits of C C X 1 6 8 or C C X 1 6 8 place b o, a n d 1 kit of pre d nis o ne a n d/ or 
pre d nis o ne place b o) will be pr o vi de d t o t he s u bject wit h d osi n g i nstr ucti o ns; t he s u bject 
will ta ke t he m or ni n g d os es fr o m t hese kits w hile at t he st u d y site, u nless t he m or ni n g 
d oses ha ve alr ea d y b ee n t a ke n b y t he s u bject;  
•  Dr u g acc o u nta bilit y of pr e d nis o ne or pre d nis o ne place b o b ottles; 
•  C ycl o p h os p ha mi de I V d ose will be gi ve n after recei pt of t he l ocal W B C c o u nt (see 
secti o n 1 1. 6 ) t o s u bjects i n t he c ycl o p h os p ha mi de strat u m; T he start a n d e n d ti mes of t he 
i nf usi o n will be rec or d e d; 


C he m o Ce ntr y x, I n c.                            C O N FI D E N TI A L                                Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8                                                                                                                    A me n d me nt 4. 0 
                                                                   Pa ge 7 2 of 1 1 3                               1 8 Se pte m ber 2 0 1 5 
•  A bl o o d sa m ple will be c ollecte d f or C C X 1 6 8 plas ma c o nce ntrati o n meas ure me nt; t he 
date a n d ti me of t he last d ose pri or t o sa m ple c oll ecti o n will be rec or de d;  
•  A n y c ha n ges i n c o nc o mita nt me dicati o n use will be rec or de d; a n d  
•  Aft er all st u d y pr oce d ures ha ve bee n c o m plete d, t he s u bject will be re mi n d e d t o: 
− C o me t o t he st u d y ce nt er f or t he Da y 7 1 st u d y visit; 
− St ore t he st u d y me dicati o ns i n a c o ol a n d dr y place acc or di n g t o la b el i nstr ucti o ns f or 
t he d urati o n of t he st u d y;  
− Ta ke t he C C X 1 6 8 or C C X 1 6 8 place b o me di cati o n as i nstr ucte d; 
− Ta ke t he pr e d nis o ne or pre d nis o ne place b o d ose as i nstr ucte d; 
− N ot t o ta ke C C X 1 6 8 a n d pre d nis o ne me dicati o n o n t he m or ni n g of Da y 7 1, u nless t he 
visit is sc he d ule d i n t he after n o o n, i n w hic h case C C X 1 6 8 a n d pre d nis o ne me dicati o n 
s h o ul d be ta ke n t he m or ni n g of D a y 7 1;  
− Bri n g all st u d y m e dicati o n c o ntai ners t o t he ne xt st u d y visit, w het her e m pt y or n ot, 
a n d 
− C o nti n ue ta ki n g all t heir ot her c o nc o mita nt me dicati o ns as us ual. 
6. 1 0.  
St u d y D a y 7 1  
T he St u d y D a y 7 1 visit m ust occ ur wit hi n +/- 2 d a ys of t he sc h e d ule d date. T he s u bjects m ust 
fast o ver ni g ht f or at least 8 h o urs bef ore t he st u d y visit. D uri n g t his visit, t he f oll o wi n g st u d y 
pr oce d ur es will be perf or me d: 
•  B o d y s yste m re vie w t o deter mi ne if t here were a n y c h a n ges f r o m Da y 5 7;  
•  All treat me nt -e mer ge nt a d verse e v e nts will be rec or de d;  
•  Vital si g ns (te m perat ure, bl o o d press ure, heart rate); 
•  Bl o o d will be c ollecte d f or s hi p me nt t o t he ce ntral la b orat or y f or ser u m c h e mistr y a n d 
he mat ol o g y meas ure m e nts; t he s u bjects ma y eat  after t he ser u m c he mistr y sa m ple 
c ollecti o n;  
•  S u bjects will be as ke d t o v oi d t heir bla d ders c o m pletel y, a n d a re pres e ntati ve clea n catc h, 
mi dstrea m uri ne sa m ple will be c ollecte d:  
− A uri ne sa m ple will be se nt t o t he ce ntral la b orat or y f or uri nal ysis i ncl u di n g p H, 
s pecific gr a vit y, gl uc os e, nitrite, ket o nes, bilir u bi n, ur o bili n o ge n, R B Cs, a n d W B Cs; 
•  Dr u g acc o u nta bilit y of C C X 1 6 8 or C C X 1 6 8 place b o b ottles; 
•  St u d y m e dicati o n ( 2 kits of C C X 1 6 8 or C C X 1 6 8 place b o) will be pr o vi de d t o t he s u bject 
wit h d osi n g i nstr ucti o ns; t he s u bject will ta ke t he m or ni n g d ose fr o m o ne of t hese kits 
w hile at t he st u d y site, u nless t he m or ni n g d ose h as alrea d y b ee n ta k e n b y t he s u bject;  
•  A bl o o d sa m ple will be c ollecte d f or C C X 1 6 8 plas ma c o nce ntrati o n meas ure me nt; t he 
date a n d ti me of t he last d ose pri or t o sa m ple c oll ecti o n will be rec or de d; 


C he m o Ce ntr y x, I n c.                            C O N FI D E N TI A L                                Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8                                                                                                                    A me n d me nt 4. 0 
                                                                   Pa ge 7 3 of 1 1 3                               1 8 Se pte m ber 2 0 1 5 
•  A n y c ha n ges i n c o nc o mita nt me dicati o n use will be rec or de d; a n d  
•  After all st u d y pr oce d ures ha ve bee n c o m plete d, t he s u bject will be re mi n d e d t o: 
− C o me t o t he st u d y ce nt er f or t he Da y 8 5 st u d y visit; 
− St ore t he st u d y me dicati o ns i n a c o ol a n d dr y place acc or di n g t o la b el i nstr ucti o ns f or 
t he d urati o n of t he st u d y; 
− Ta ke t he C C X 1 6 8 or C C X 1 6 8 place b o me di cati o n as i nstr ucte d;  
− Ta ke t he pr e d nis o ne or pre d nis o ne place b o d ose as i nstr ucte d; 
− N ot t o ta ke C C X 1 6 8 a n d pre d nis o ne me dicati o n o n t he m or ni n g of Da y 8 5, b ut t o 
ta ke t he pre d nis o n e me di cati o n at t he st u d y ce nter o n t he da y of t he visit;  
− Bri n g all st u d y m e dicati o n c o ntai ners t o t he ne xt st u d y visit, w het her e m pt y or n ot, 
a n d 
− C o nti n ue ta ki n g all t heir ot her c o nc o mita nt me dicati o ns as us ual. 
6. 1 1.  
St u d y D a y 8 5  
T he St u d y D a y 8 5 visit m ust occ ur wit hi n +/- 2 d a ys of t he sc h e d ule d date. T he s u bjects m ust 
fast o ver ni g ht f or at least 8 h o urs bef ore t he st u d y visit. D uri n g t his visit, t he f oll o wi n g st u d y 
pr oce d ur es will be perf or me d: 
•  A p h ysical e x a mi nati o n (e x cl u di n g ge nit o uri nar y, o p ht hal mic, a n d fe male breast 
assess me nts) will be perf or me d; b o d y wei g ht will als o be meas ure d;  
•  All treat me nt -e mer ge nt a d verse e v e nts will be rec or de d;  
•  Vital si g ns (te m perat ure, bl o o d press u re, heart rate); 
•  Bl o o d will be c ollecte d f or s hi p me nt t o t he ce ntral la b orat or y f or ser u m c h e mistr y, 
he mat ol o g y a n d hs C R P meas ure me nts; t he s u bjects ma y eat after t he ser u m c he mistr y 
sa m ple c ollecti o n; 
•  A bl o o d sa m ple will be c ollecte d f or p h ar mac o d y na mics mar ker m eas ure me nts;  
•  C ycl o p h os p ha mi de I V d ose will be gi ve n after recei pt of t he l ocal W B C c o u nt (see 
secti o n 1 1. 6 ) t o s u bjects i n t he c ycl o p h os p ha mi de strat u m; T he start a n d e n d ti mes of t he 
i nf usi o n will be recor d e d;  
•  S u bjects will be as ke d t o v oi d t heir bla d ders c o m pletel y, a n d a re pres e ntati ve clea n catc h, 
mi dstrea m uri ne sa m ple will be c ollecte d:  
− A uri ne sa m ple will be se nt t o t he ce ntral la b orat or y f or uri nal ysis i ncl u di n g p H, 
s pecific gr a vit y, gl uc os e, nitrite, ket o nes, bilir u bi n, ur o bili n o ge n, R B Cs, a n d W B Cs, 
as well as q ua ntitati ve m eas ure m e nt of al b u mi n a n d creati ni ne t o calc ulate t he A C R;  
− T w o a d diti o nal uri ne sa m ples will be fr oze n a n d se nt t o t he ce ntral la b orat or y f or 
q ua ntitati ve M C P -1 a n d creati ni ne meas ure m e nts t o calc ulate t he M C P -1:creati ni ne 
rati o, a n d f or e x pl orat or y uri nar y mar k ers; 
•  A N C A (a nti - P R 3 a n d a nti-M P O E LI S A) m eas ure me nt;  


C he m o Ce ntr y x, I n c.                            C O N FI D E N TI A L                                Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8                                                                                                                    A me n d me nt 4. 0 
                                                                   Pa ge 7 4 of 1 1 3                               1 8 Se pte m ber 2 0 1 5 
•  B V A S assess me nt; c h est X ra ys will o nl y b e ac q uire d if dee me d cli nicall y n ecessar y b y 
t he Pri nci pal I n vesti gat or (see secti o n 1 1. 3 ); 
•  V DI assess me nt (s ee secti o n  1 1. 4 ); 
•  S u bjects will be as ke d t o c o m plete t he S F 3 6 v 2 a n d E Q- 5 D- 5 L q u esti o n naires; 
•  Dr u g acc o u nta bilit y of C C X 1 6 8 or C C X 1 6 8 place b o b ottles; 
•  Dr u g acc o u nta bilit y of pr e d nis o ne or pre d nis o ne place b o b ottles; 
•  St u d y m e dicati o n ( 1 kit of pre d nis o ne a n d/ or pre d nis o ne place b o) will be pr o vi de d t o t he 
s u bject wit h d osi n g i nstr ucti o ns; t he s u bject will ta ke t he m or ni n g d ose fr o m t his kit 
w hile at t he st u d y site, u nless t he m or ni n g d ose h as alrea d y b ee n ta k e n b y t he s u bject;  
•  A bl o o d sa m ple will be c ollecte d f or C C X 1 6 8 plas ma c o nce ntrati o n meas ure me nt; t he 
date a n d ti me of t he last d ose pri or t o sa m ple c oll ecti o n will be rec or de d; 
•  If a r e nal bi o ps y has bee n perf or me d pri or t o Da y 1 f or re n al disease assess me nt, if 
p ossi ble, a re nal bi o ps y will be ta ke n at/ wit hi n  1 wee k after t he Da y 8 5 visit a n d t he 
Hist ol o g y F or m c o m plete d ; 
•  A n y c ha n ges i n c o nc o mita nt me dicati o n use will be rec or de d; a n d  
•  Afte r all st u d y pr oce d ures ha ve bee n c o m plete d, t he s u bject will be re mi n d e d t o: 
− C o me t o t he st u d y ce nt er f or t he Da y 9 9 st u d y visit; 
− St ore t he st u d y me dicati o ns i n a c o ol a n d dr y place acc or di n g t o la b el i nstr ucti o ns f or 
t he d urati o n of t he st u d y; 
− Ta ke t he pr e d nis o ne or pre d nis o ne place b o d ose as i nstr ucte d; 
− N ot t o ta ke t he pre d nis o n e me dicati o n o n t he m or ni n g of D a y 9 9, u nless t he visit is 
sc he d ule d i n t he after n o o n, i n w hic h case pre d nis o ne me dicati o n s h o ul d be ta ke n t he 
m or ni n g of D a y 9 9;   
− Bri n g all st u d y m e dicati o n c o ntai ners t o t he ne xt st u d y visit, w het her e m pt y or n ot, 
a n d 
− C o nti n ue ta ki n g all t heir ot her c o nc o mita nt me dicati o ns as us ual. 
6. 1 2.  
St u d y D a y 9 9  
T he St u d y D a y 9 9 visit m ust occ ur wit hi n +/- 4 d a ys of t he sc h e d ule d date. T he s u bjects m ust 
fast o ver ni g ht f or at least 8 h o urs bef ore t he st u d y visit. D uri n g t his visit, t he f oll o wi n g stu d y 
pr oce d ur es will be perf or me d: 
•  B o d y s yste m re vie w t o deter mi ne if t here were a n y c h a n ges fr o m Da y 8 5;  
•  All treat me nt -e mer ge nt a d verse e v e nts will be rec or de d;  
•  Vital si g n s (te m perat ure, bl o o d press ure, heart rate); 


C he m o Ce ntr y x, I n c.                            C O N FI D E N TI A L                                Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8                                                                                                                    A me n d me nt 4. 0 
                                                                   Pa ge 7 5 of 1 1 3                               1 8 Se pte m ber 2 0 1 5 
•  Bl o o d will be c ollecte d f or s hi p me nt t o t he ce ntral la b orat or y f or ser u m c h e mistr y a n d 
he mat ol o g y meas ure m e nts; t he s u bjects ma y eat after t he ser u m c he mistr y sa m ple 
c ollecti o n;  
•  S u bjects will be as ke d t o v oi d t h eir bla d ders c o m pletel y, a n d a re pres e ntati ve clea n catc h, 
mi dstrea m uri ne sa m ple will be c ollecte d:  
− A uri ne sa m ple will be se nt t o t he ce ntral la b orat or y f or uri nal ysis i ncl u di n g p H, 
s pecific gr a vit y, gl uc os e, nitrite, ket o nes, bilir u bi n, ur o bili n o ge n, R B Cs, a n d W B Cs; 
•  Dr u g acc o u nta bilit y of pr e d nis o ne or pre d nis o ne place b o b ottles; 
•  St u d y m e dicati o n ( 1 kit of pre d nis o ne a n d/ or pre d nis o ne place b o) will be pr o vi de d t o t he 
s u bject wit h d osi n g i nstr ucti o ns; t he s u bject will ta ke t he m or ni n g d ose fr o m t his kit 
w hile at t he st u d y site, u nless t he m or ni n g d ose h as alrea d y b ee n ta k e n b y t he s u bject;  
•  S u bjects i n t he c ycl o p h os p ha mi de strat u m will start oral azat hi o pri ne after t he W B C 
c o u nt fr o m t he l ocal la b orat or y has bee n recei v e d, a n d will c o nti n ue ta ki n g it t hr o u g h 
Da y 1 6 8 (see secti o n 1 1. 6  f or d osi n g i nstr u cti o ns); 
•  A n y c ha n ges i n c o nc o mita nt me dicati o n use will be rec or de d; a n d  
•  After all st u d y pr oce d ures ha ve bee n c o m plete d, t he s u bject will be re mi n d e d t o: 
− C o me t o t he st u d y ce nt er f or t he Da y 1 1 3 st u d y visit; 
− St ore t he st u d y me dicati o n i n a c o ol a n d dr y place acc or di n g t o la b el i nstr ucti o ns f or 
t he d urati o n of t he st u d y; 
− Ta ke t he pr e d nis o ne or pre d nis o ne place b o d ose as i nstr ucte d; 
− N ot t o ta ke t he pre d nis o n e me dicati o n o n t he m or ni n g of D a y 1 1 3, u nless t h e visit is 
sc he d ule d i n t he after n o o n, i n w hic h case pre d nis o ne me dicati o n s h o ul d be ta ke n t he 
m or ni n g of D a y 1 1 3;  
− Bri n g all st u d y m e dicati o n c o ntai ners t o t he ne xt st u d y visit, w het her e m pt y or n ot, 
a n d 
− C o nti n ue ta ki n g all t heir ot her c o nc o mita nt me dicati o ns as us ual.  
6. 1 3.  
St u d y D a y 1 1 3  
T he St u d y D a y 1 1 3 visit m ust occ ur wit hi n +/- 4 d a ys of t he sc h e d ule d date. D uri n g t his visit, t he 
f oll o wi n g st u d y pr oce d ur es will be perf or me d: 
•  A p h ysical e x a mi nati o n (e x cl u di n g ge nit o uri nar y , o p ht hal mic, a n d fe male breast 
assess me nts) will be perf or me d; b o d y wei g ht will als o be meas ure d;  
•  All treat me nt -e mer ge nt a d verse e v e nts will be rec or de d;  
•  Vital si g ns (te m perat ure, bl o o d press ure, heart rate); 
•  Bl o o d will be c ollecte d f or s hi p me nt t o t he ce ntral la b orat or y f or ser u m creati ni ne a n d 
hs C R P meas ure me nt;  


C he m o Ce ntr y x, I n c.                            C O N FI D E N TI A L                                Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8                                                                                                                    A me n d me nt 4. 0 
                                                                   Pa ge 7 6 of 1 1 3                               1 8 Se pte m ber 2 0 1 5 
•  B V A S assess me nt; c h est X ra ys will o nl y b e ac q uire d if dee me d cli nicall y n ecessar y b y 
t he Pri nci pal I n vesti gat or (see secti o n 1 1. 3 ); 
•  S u bjects will be as ke d t o v oi d t heir bla d ders c o m pletel y, a n d a re pres e ntati ve clea n catc h, 
mi dstrea m uri ne sa m ple will be c ollecte d:  
− A uri ne sa m ple will be se nt t o t he ce ntral la b orat or y f or uri nal ysis i ncl u di n g p H, 
s pecific gr a vit y, gl uc os e, nitrite, ket o nes, bilir u bi n, ur o bili n o ge n, R B Cs, a n d W B Cs, 
as well as q ua ntitati ve m eas ure m e nt of al b u mi n a n d creati ni ne t o calc ulate t he A C R;  
− T w o a d diti o nal uri ne sa m ples will be fr oze n a n d se nt t o t he ce ntral la b orat or y f or 
q ua ntitati ve M C P -1 a n d creati ni ne meas ure m e nts t o calc ulate  t he M C P-1:creati ni ne 
rati o, a n d f or e x pl orat or y uri nar y mar k ers; 
•  Dr u g acc o u nta bilit y of pr e d nis o ne or pre d nis o ne place b o b ottles; 
•  St u d y m e dicati o n ( 1 kit of pre d nis o ne a n d/ or pre d nis o ne place b o) will be pr o vi de d t o t he 
s u bject wit h d osi n g i nstr ucti o ns; t he s u bject will ta ke t he m or ni n g d ose fr o m t his kit 
w hile at t he st u d y site, u nless t he m or ni n g d ose h as alrea d y b ee n ta k e n b y t he s u bject;  
•  S u bjects i n t he c ycl o p h os p ha mi de strat u m will be gi ve n oral azat hi o pri ne at a tar get d ose 
of 2 m g/ k g/ da y (s ee secti o n  1 1. 6  f or d osi n g i nstr u cti o ns); 
•  A N C A (a nti - P R 3 a n d a nti-M P O E LI S A) m eas ure me nt;  
•  A n y c ha n ges i n c o nc o mita nt me dicati o n use will be rec or de d; a n d  
•  After all st u d y pr oce d ures ha ve bee n c o m plete d, t he s u bject will be re mi n d e d t o: 
− C o me t o t he st u d y ce nt er f or t he Da y 1 4 1 st u d y visit;  
− St ore t he st u d y me dicati o n i n a c o ol a n d dr y place acc or di n g t o la b el i nstr ucti o ns f or 
t he d urati o n of t he st u d y; 
− Ta ke t he pr e d nis o ne or pre d nis o ne place b o d ose as i nstr ucte d; 
− N ot t o ta ke t he pre d nis o n e me dicati o n o n t he m or ni n g of D a y 1 4 1; a n d 
− C o nti n ue ta ki n g all t heir ot her c o nc o mita nt me dicati o ns as us ual. 
6. 1 4.  
St u d y D a y 1 4 1  
T he St u d y D a y 1 4 1 visit m ust occ ur wit hi n +/- 4 d a ys of t he sc h e d ule d date. T he s u bjects m ust 
fast o ver ni g ht f or at least 8 h o urs bef ore t he st u d y visit. D uri n g t his visit, t he f oll o wi n g st u d y 
pr oce d ur es will be perf or me d: 
•  B o d y s yste m re vie w t o deter mi ne if t here were a n y c h a n ges fr o m Da y 1 1 3;  
•  All treat me nt -e mer ge nt a d verse e v e nts will be rec or de d;  
•  Vital si g ns (te m perat ur e, bl o o d press ure, heart rate); 
•  Bl o o d will be c ollecte d f or s hi p me nt t o t he ce ntral la b orat or y f or ser u m c h e mistr y a n d 
he mat ol o g y meas ure m e nts; t he s u bjects ma y eat after t he ser u m c he mistr y sa m ple 
c ollecti o n;  


C he m o Ce ntr y x, I n c.                            C O N FI D E N TI A L                                Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8                                                                                                                    A me n d me nt 4. 0 
                                                                   Pa ge 7 7 of 1 1 3                               1 8 Se pte m ber 2 0 1 5 
•  S u bjects will be as ke d t o v oi d t heir bla d ders c o m pletel y, a n d a re pres e ntati ve clea n catc h, 
mi dstrea m uri ne sa m ple will be c ollecte d:  
− A uri ne sa m ple will be se nt t o t he ce ntral la b orat or y f or uri nal ysis i ncl u di n g p H, 
s pecific gr a vit y, gl uc os e, nitrite, ket o nes, bilir u bi n, ur o bili n o ge n, R B Cs, a n d W B Cs; 
•  S u bjects i n t he c ycl o p h os p ha mi de strat u m will be gi ve n oral azat hi o pri ne at a tar get d ose 
of 2 m g/ k g/ da y (s ee secti o n 1 1. 6  f or d osi n g i nstr u cti o ns); a l oc al l a b or at or y W B C c o u nt 
will be d o ne i n t hese s u bjects;  
•  Dr u g acc o u nta bilit y of pr e d nis o ne or pre d nis o ne place b o b ottles; 
•  A n y c ha n ges i n c o nc o mita nt me dicati o n use will be rec or de d; a n d  
•  After all st u d y pr oce d ures ha ve bee n c o m plete d, t he s u bject will be re mi n d e d t o: 
− C o me t o t he st u d y ce nt er f or t he Da y 1 6 9 st u d y visit; a n d  
− C o nti n ue ta ki n g all t heir ot her c o nc o mita nt me dicati o ns as us ual. 
6. 1 5.  
St u d y D a y 1 6 9  
T he St u d y D a y 1 6 9 visit m ust occ ur wit hi n +/- 4 d a ys of t he sc h e d ule d date. T he s u bjects m ust 
fast o ver ni g ht f or at least 8 h o urs bef ore t he st u d y visit. D uri n g t his visit, t he f oll o wi n g st u d y 
pr oce d ur es will be perf or me d: 
•  A p h ysical e x a mi nati o n (e x cl u di n g ge nit o uri nar y, o p ht hal mic, a n d fe male breast 
assess me nts) will be perf or me d; b o d y wei g ht will als o be meas ure d;  
•  All treat me nt -e mer ge nt a d verse e v e nts will be rec or de d; 
•  Vital si g ns (te m perat ure, bl o o d press ure, heart rate); 
•  Bl o o d will be c ollecte d f or s hi p me nt t o t he ce ntral la b orat or y f or ser u m c h e mistr y, 
he mat ol o g y, a n d hs C R P meas ure me nts; t he s u bjects ma y eat after t he ser u m c he mistr y 
sa m ple c ollecti o n; 
•  A bl o o d sa m ple will be c ollecte d f or p h ar mac o d y na mics mar ker m eas ure me nts;  
•  S u bjects will be as ke d t o v oi d t heir bla d ders c o m pletel y, a n d a re pres e ntati ve clea n catc h, 
mi dstrea m uri ne sa m ple will be c ollecte d:  
− A uri ne sa m ple will be se nt t o t he ce ntral la b orat or y f or  uri nal ysis i ncl u di n g p H, 
s pecific gr a vit y, gl uc os e, nitrite, ket o nes, bilir u bi n, ur o bili n o ge n, R B Cs, a n d W B Cs, 
as well as q ua ntitati ve m eas ure m e nt of al b u mi n a n d creati ni ne t o calc ulate t he A C R;  
− T w o a d diti o nal uri ne sa m ples will be fr oze n a n d se nt t o t he ce ntral la b orat or y f or 
q ua ntitati ve M C P -1 a n d creati ni ne meas ure m e nts t o calc ulate t he M C P -1:creati ni ne 
rati o, a n d f or e x pl orat or y uri nar y mar k ers; 
•  A N C A (a nti - P R 3 a n d a nti-M P O E LI S A) m eas ure me nt;  
•  B V A S assess me nt; c h est X ra ys will o nl y b e ac q uire d if dee me d cli nicall y n ecessar y b y 
t he Pri nci pal I n vesti gat or (see secti o n 1 1. 3 ); 
•  V DI assess me nt (s ee  secti o n 1 1. 4 ); 


C he m o Ce ntr y x, I n c.                            C O N FI D E N TI A L                                Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8                                                                                                                    A me n d me nt 4. 0 
                                                                   Pa ge 7 8 of 1 1 3                               1 8 Se pte m ber 2 0 1 5 
•  S u bjects will be as ke d t o c o m plete t he S F 3 6 v 2 a n d E Q- 5 D- 5 L q u esti o n naires; 
•  Dr u g acc o u nta bilit y of pr e d nis o ne or pre d nis o ne place b o b ottles, o nl y if t his is a n earl y 
ter mi nati o n visit; 
•  A n y c ha n ges i n c o nc o mita nt me dicati o n use will be rec or de d; a n d  
•  After all st u d y pr oce d ures ha ve bee n c o m plete d, t he s u bject will be disc har ge d fr o m t he 
st u d y. T he s u bject’s c o n diti o n will be e val uate d b y t he I n v esti gat or at t he e n d of t he 
cli nical trial ( Da y 1 6 9) a n d a p pr o priate sta n d ar d of care me dical treat me nt will be 
pr o vi de d t o all s u bjects as nee de d. 
7.  
S T U D Y A S S E S S M E N T S  
7. 1.  
Effic ac y As sess me nts 
7. 1. 1.  
C ortic ost er oi d Resc ue Use  
If res c ue I V or hi g h d ose oral c ortic oster oi ds are n ee de d, it is dee me d a treat me n t fail ure f or 
p ur p oses of data a nal ysis (see secti o n 5. 1 0 ). Met h yl pre d nis ol o ne I V resc ue use (t otal d ose a n d 
d urati o n of d osi n g) a n d oral resc u e c ortic oster oi d use m ust be rec or de d f or all s u bjects a n d will 
be c o m par e d a m o n g  treat me nt gr o u ps. 
7. 1. 2.  
Esti m at e d Gl o mer ul ar Filtr ati o n R ate  
Esti mate d gl o mer ular filtrati o n rate ( e G F R) will be calc ul ate d at all a p plica ble st u d y visits usi n g 
t he f oll o wi n g M D R D e q uati o n: 
e G F R ( m L/ mi n/ 1. 7 3 m 2) = 1 7 5 x (ser u m creati ni ne i n m g/ d L)-1. 1 5 4 x ( A ge) -0. 2 0 3 x ( 0. 7 4 2 if 
fe male) x ( 1. 2 1 2 if Africa n-A meri ca n/ Bl ac k)  
C ystati n C meas ure me nts ma y b e use d t o cal c ulate e G F R usi n g t he  f oll o wi n g e q u ati o n: 
e G F R c ys ( m L/ mi n/ 1. 7 3 m2) = 1 2 7. 7 x ( c ystati n C  i n m g/ L)−1. 1 7 x ( A ge) −0. 1 3 x ( 0. 9 1 if fe male) 
x ( 1. 0 6 if Africa n -A m eri ca n/ Blac k)  
7. 1. 3.  
Uri n ar y Assess me nts  
Uri ne sa m ples c ollecte d at all st u d y visits will be a nal yze d f or he m at uria, R B C casts, a n d 
pr otei n uria ( A C R). A clea n catc h mi dstrea m uri ne sa m ple nee ds t o be c oll ecte d acc or di n g t o 
i nstr ucti o ns pr o vi de d se paratel y. T h e uri ne sa m ples will be se nt t o t he ce ntral la b orat or y f or 
a nal ysis. Micr osc o pic r e vie w is tri g gere d if a uri n ar y di pstic k test is p ositi ve f or W B C, R B C, 
nitrite or pr otei n. W he n micr osc o p y is perf or me d, he mat uria will be cate g orize d as f oll o ws:  
N o ne, Occasi o nal ( O cc), 1 - 2, 3 - 5, 6 - 9, 1 0 - 1 5, 1 6 - 2 9, 3 0 - 4 9, 5 0 - 7 5, a n d > 7 5 R B Cs per 
hi g h p o wer fiel d. F or t he p ur p ose of a n al yzi n g t he c ha n ge fr o m baseli ne i n uri nar y R B Cs, t he 
f oll o wi n g val ues will be assi g ne d f or eac h cate g or y: 
N o ne = 0. 1, O cc = 0. 5, 1 - 2 = 1, 3 - 5 = 3, 6 - 9 = 6, 1 0 - 1 5 = 1 0, 1 6 - 2 9  = 1 6, 3 0 - 4 9 = 3 0; 
5 0 - 7 5 = 5 0, > 7 5 = 7 5.   


C he m o Ce ntr y x, I n c.                            C O N FI D E N TI A L                                Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8                                                                                                                    A me n d me nt 4. 0 
                                                                   Pa ge 7 9 of 1 1 3                               1 8 Se pte m ber 2 0 1 5 
Pr otei n uria will be assess e d b y m eas uri n g t he al b u mi n a n d creati ni ne c o nce ntrati o ns a n d 
calc ulati n g t he uri nar y A C R. Res ults will be e x presse d as m g al b u mi n/ g creati ni ne.  T his will be 
d o ne b y t h e ce ntr al la b or at or y.  
7. 1. 4.  
Bir mi n g h a m V asc ulitis Acti vit y Sc ore ( B V A S)  
T he B V A S ( versi o n 3) will be calc ulate d bas e d o n res p o nses i n 1 0 d o mai ns: ge n eral, c ut a ne o us, 
m uc o us me m bra nes a n d e yes, ear/ n ose/t hr oat ( E N T), c hest, car di o vasc ul ar, a b d o mi nal, re nal, 
ner v o us s yst e m, a n d ot her. Secti o n 1 1. 3  pr o vi des a list of ite ms t hat nee d t o be assesse d. B V A S 
data will be a dj u dicate d  bef ore fi nalizati o n.  
7. 1. 5.  
V asc ulitis D a m a g e I n de x ( V DI)  
T he V DI (acc or di n g t o L u q ma ni a n d Hall, 2 0 0 4) will be calc ulate d b ase d o n res p o nses i n 1 1 
or ga n s yste ms as pr o vi de d i n secti o n 1 1. 4 . V DI data will be a dj u dicate d  bef ore fi nalizati o n. 
7. 1. 6.  
A nti -Ne utr o p hil C yt o pl as mic A nti b o d y ( A N C A) Assess me nts  
A N C A assess me nts will i ncl u de i n direct i m m u n ofl u oresce n ce test f or P -A N C A a n d C -A N C A as 
well as E LI S A tests f or a nti - P R 3 a n d a nti-M P O at Scree ni n g a n d o nl y E LI S A tests f or a nti - P R 3 
a n d a nti- M P O o n Da ys 1 ( pri or t o d osi n g), 2 9, 8 5, 1 1 3, a n d 1 6 9. E LI S A assa ys will be 
perf or me d b y a ce ntral la b orat or y.  
7. 1. 7.  
Ser u m C -Re a cti ve Pr ot ei n ( C R P)  
Ser u m C R P will be meas ure d b y t he ce ntral la b orat or y o n D a ys 1 ( pri or t o d osi n g), 8, 1 5, 2 9, 5 7, 
8 5, 1 1 3, a n d 1 6 9 b y hi g h se nsiti vit y C R P assa y.  
7. 1. 8.  
Uri n ar y M C P -1 Assess me nts  
Uri ne M C P -1 will be meas ure d b y t he ce ntral la b orat or y i n uri ne sa m ples c ollecte d o n St u d y 
Da ys 1 ( pri or t o d osi n g), 8, 1 5, 2 9, 5 7, 8 5, 1 1 3, a n d 1 6 9 b y s pecific E LI S A. Uri ne creati ni ne w ill 
be meas ur e d i n t he sa me uri ne sa m ples a n d M C P - 1 le vels will be sta n dar di ze d t o uri ne creati ni ne 
a n d e x presse d as p g M C P -1/ m g cr eati ni ne. A d diti o nal uri ne sa m ples will be st ore d at - 7 0° C a n d 
ma y als o be use d t o meas ure ot her c yt o ki n e, i nfla m mat or y a n d c o m ple me nt mar kers.  
7. 1. 9.  
He alt h -Rel ate d Q u alit y of Life Assess me nts  
T he S F - 3 6 v 2 a n d E Q- 5 D-5 L h ealt h -r elate d q ualit y of life q uesti o n naires will be c o m plete d b y 
st u d y s u bjects at t he D a y 1 ( pre- d os e), 2 9, 8 5, a n d 1 6 9 st u d y visits t o meas ure c ha n ges fr o m 
baseli ne i n healt h-r elate d q ualit y of life. Pr o v e n tra nslati o ns will be use d f or n o n-E n glis h 
s pea ki n g s u bj ects. A n a d mi nistrat or will facilitate c o m pleti o n of t he q uesti o n naires b y t he 
s u bjects. T he a d mi nistrat or will esta blis h a ra p p ort wit h t he s u bject, e m p hasize t he i m p orta nce of 
c o m pleti n g t he f or m, a n d ser ve t o a ns w er q uesti o ns a n d a d dress c o ncer ns. T he q uesti o n naires 
s h o ul d be c o m plete d b y s u bjects bef or e seei n g t he I n v esti gat or.  
7. 1. 1 0.  
Hist ol o g y 
A sta n dar d H ist ol o g y F or m will be pr o vi de d t o i n vesti gat ors t o d oc u m e n t hist ol o gic fi n di n gs i n a 
sta n dar d ma n ner f or all s u bjects fr o m w h o m re n al bi o psies are ta ke n. T his will be c o m plete d f or 
a vaila ble bas eli ne a n d D a y 8 5 re nal bi o psies. T o t he e xte nt p ossi ble, c ha n ges fr o m baseli ne i n 


C he m o Ce ntr y x, I n c.                            C O N FI D E N TI A L                                Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8                                                                                                                    A me n d me nt 4. 0 
                                                                   Pa ge 8 0 of 1 1 3                               1 8 Se pte m ber 2 0 1 5 
t y pical hist ol o gi c fi n di n gs, e. g., p erce nta ge of gl o mer uli wit h cresce nts or scler osis will be 
a nal yze d.  
7. 2.  
S afet y Assess me nts  
7. 2. 1.  
P h ysic al E x a mi n ati o ns a n d Vit al Si g ns 
A c o m plete p h ysical e x a mi nati o n (i ncl u di n g e val uati o n of ge neral a p peara nce/ me ntal stat us, 
H E E N T [ hea d, e yes, ears, n ose, t hr oat], a n d t he f oll o wi n g b o d y s yst e ms: der mat ol o gic, 
car di o vas c ular, r es pirat or y, gastr oi ntesti nal, m usc ul os keletal a n d ne ur ol o gic) f or safet y 
assess me nt will be perf or me d at Scree ni n g a n d St u d y D a ys 1, 1 5, 2 9, 5 7, 8 5, 1 1 3, a n d 1 6 9. 
P h ysical e x a mi nati o ns d uri n g scree ni n g a n d o n St u d y D a ys 1, 2 9, 8 5, 1 1 3, a n d 1 6 9 m ust be 
s ufficie ntl y c o m pre h e nsi ve t o i ncl u de A L L c o m p o ne nts of t he B V A S (see secti o n 1 1. 3 ). 
Fi n di n gs m ust be r ec or d e d i n t he s o urce d oc u m e nts. S yst e matic b o d y s y ste m re vie ws will be 
d o ne at St u d y D a ys 2, 8, 2 2, 4 3, 7 1, 9 9, a n d 1 4 1. 
A n y n e w or w orse ni n g fi n di n gs u p o n p h ysical e x a mi nati o n or b o d y s yste m re vie w nee d t o be 
rec or d e d as a d v erse e ve nts. 
B o d y wei g ht will be meas ure d at Scree ni n g a n d D a ys 1, 1 5, 2 9, 5 7, 8 5, 1 1 3, a n d 1 6 9.  
Vital si g ns will be meas ure d d uri n g Scree ni n g a n d o n eac h sc h e d ule d st u d y da y as i n dicate d i n 
t he Ti me a n d E ve nts T a ble. Bl o o d press ure, p ulse rate, a n d b o d y te m p erat ure will be meas ure d.  
All assess me nts will be per f or me d w hile t he s u bject is i n t he s u pi ne p ositi o n a n d after t he s u bject 
has reste d f or at least t hr ee mi n utes.  
T wel ve -lea d E C Gs will be ac q uir e d d uri n g Scree ni n g a n d o n D a y 2 9 a n d will be assesse d f or a n y 
cli nicall y si g nifica nt a b n or malities.  
7. 2. 2.  
Cli nic al S af et y L a b or at or y Assess me nts 
T he f oll o wi n g t es ts will be perf or me d at t he visits i de ntifie d i n t he Ti me a n d E ve nts Ta ble . 
•  He mat ol o g y:  h e m o gl o bi n, he mat ocrit, re d bl o o d cell ( R B C) c o u nt, w hite bl o o d cell 
( W B C) c o u nt wit h differ e ntial, platelet c o u nt, mea n cell he m o gl o bi n ( M C H), mea n cell 
he m o gl o bi n c o n ce ntrati o n ( M C H C), mea n c or p us c ular v ol u me. 
•  Ser u m C he mistr y:  li ver pa nel ( bilir u bi n, lactate d e h y dr o ge nas e [ L D H], S G O T/ A S T, 
S G P T/ A L T), r e nal pa n el ( B U N, creati ni ne), creati ne p h os p h o ki nase ( C P K), al b u mi n, 
s o di u m, p otassi u m, bicar b o nate, c hl ori de, calci u m, i n or ga ni c p h os p h or us, gl uc ose, t otal 
pr otei ns, al kali ne p h os p h atase, c h olest er ol, uric aci d. 
•  C oa g ulati o n ( meas ure d o nl y at Scree ni n g at t he l o cal la b orat or y): pr ot hr o m bi n ti me ( P T), 
a n d acti vate d p artial t hr o m b o plasti n ti me (a P T T)  
•  Uri nal ysis:  At t he ce ntral la b orat or y, p H, s pecific gra vit y, gl uc os e, nitrite, ket o nes, 
bilir u bi n, ur o bili n o ge n, R B C, a n d W B C tests, as w ell as q ua ntificati o n of al b u mi n a n d 
creati ni ne f or A C R calc ulati o n will be perf or me d. A sec o n d sa m ple will be a nal yze d 
q ua ntitati vel y f or M C P -1 a n d creati ni ne at visits s pecifie d i n t he Ti me a n d E ve nts T a ble. 
A nal ysis at t he l o cal la b orat or y will i ncl u de he mat uria a n d al b u mi n uria assess me nts at 
scree ni n g. 


C he m o Ce ntr y x, I n c.                            C O N FI D E N TI A L                                Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8                                                                                                                    A me n d me nt 4. 0 
                                                                   Pa ge 8 1 of 1 1 3                               1 8 Se pte m ber 2 0 1 5 
•  Vir ol o g y ( m eas ur e d o nl y at Scree ni n g at t he l o cal la b orat or y): he p atitis B s urface 
a nti ge n, h e patitis C a nti b o dies, HI V 1 a n d 2 a nti b o dies. 
•  Ser ol o g y a n d C o m ple me nt ( meas ure d o nl y at Scree ni n g at t he l ocal la b orat or y): a nti-
n uclear a n d a nti - G B M a nti b o d y le vels, C 3, C 4, I g G, I g M, a n d I g A. T h ese t ests d o n ot 
nee d t o be perf or me d if r es ults are a v aila ble fr o m tests d o ne wit hi n t he past 1 2 m o nt hs 
pri or t o t he Scree ni n g visit. Res ults fr o m t hese pri or tests m ust be rec or d e d i n t he e C R Fs.  
•  T B scr ee n: C hest X r a ys , d o ne as part of B V A S, will be perf or me d at scree ni n g t o r ule 
o ut T B. C hest X ra ys at s u bse q ue nt visits will o nl y be p erf or me d if dee me d cli nicall y 
necessar y b y t he Pri nci pal I n vesti gat or.  
7. 2. 3.  
Re p orti n g of A d verse E ve nts  
A n a d vers e e ve nt ( A E) is defi ne d as a n y u nt o war d me dical occ urre n ce i n a s u bject partici pati n g 
i n a cli nical trial w h o is a d mi nistere d a n i n vesti gati o nal pr o d uct, at a n y d os e; t he a d vers e e v e nt 
d oes n ot necess aril y h a ve t o ha ve a ca usal relati o ns hi p wit h t his pr o d uct. A n a d verse e ve nt c o ul d 
t heref or e be a n y u nfa v or a ble a n d/ or u ni nte n de d si g n (i ncl u di n g a b n or mal l a b orat or y fi n di n gs), 
s y m pt o m, or disease t e m p orall y ass ociate d wit h t he use of a n i n v esti gati o n al pr o d uct, w het her or 
n ot c o nsi dere d r elate d t o t he i n vesti gati o nal pr o d u ct. T his defi niti o n i ncl u des i nterc urre nt 
ill nesses or i nj uries, a n d e x acer bati o n of pre-e xisti n g c o n diti o ns. 
A n u ne x pecte d a d verse e ve nt is a n a d vers e e ve nt t hat is n ot i de ntifie d i n nat ure, se verit y, or 
fre q ue n c y i n t he c urre nt Cli nical I n vesti gat or’s Br oc h ure, or t h at is of great er se verit y t ha n 
e x pecte d base d o n t he i nf or mati o n i n t he Cli nical I n vesti gat or’s Br oc h ure. 
All a d verse e ve nts occ urri n g i n s u bjects w h o ha v e bee n ra n d o mize d t o treat me nt will be rec or d e d 
o n t he C R F a n d will be r e p orte d i n acc or d a nce wit h re g ul at or y re q uire me nts. A d verse e ve nts 
re p orte d pri or t o c o m me nce me nt of a d mi nistrati o n of st u d y me dicati o n will be c o nsi dere d pre-
treat me nt e ve nts. 
All a d verse e ve nts will be m o nit ore d u ntil res ol uti o n or, if t he A E is deter mi ne d t o be c hr o nic, 
u ntil a ca use i s i de ntifie d. If a n a d v erse e ve nt re mai ns u nres ol ve d at t he c o ncl usi o n of t he st u d y, 
a cli nical assess me nt will be ma de b y t he I n v esti gat or a n d t he S p o ns or’s M e dical M o nit or 
w het her c o nti n ue d f oll o w- u p of t he a d v erse e ve nt is warra nte d.  
T he se verit y of eac h a d v erse e v e nt will be deter mi ne d b y t he i n v esti gat or usi n g t he f oll o wi n g 
scale: 
•  Mil d ( Gra de 1):  n o li mitati o n of us ual acti vities.  
•  M o derate ( Gra d e 2):  s o me li mitati o n of us ual acti vities.  
•  Se vere ( Gra d e 3):  i na bilit y t o carr y o ut us ual acti vities.  
•  Lif e-t hreate ni n g ( Gra de 4):  a n i m me diate ris k of deat h. 
•  Deat h ( Gra de 5)  
T he relati o ns hi p of C C X 1 6 8/ place b o t o a n a d v ers e e ve nt will be det er mi ne d b y t he I n vesti gat or 
a n d S p o ns or base d o n t he f oll o wi n g defi niti o ns: 
•  Pr o ba bl y N ot Relat e d:  t he a d vers e e v e nt was  m ore li kel y e x plai ne d b y ca uses ot her t ha n 
C C X 1 6 8/ place b o.  


C he m o Ce ntr y x, I n c.                            C O N FI D E N TI A L                                Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8                                                                                                                    A me n d me nt 4. 0 
                                                                   Pa ge 8 2 of 1 1 3                               1 8 Se pte m ber 2 0 1 5 
•  P ossi bl y Relate d:  C C X 1 6 8/ place b o a d mi nistrati o n a n d t he a d verse e v e nt o cc urre nce 
were reas o n a bl y relat e d i n ti me, a n d t he A E was e x plai ne d e q uall y well b y ca uses ot her 
t ha n C C X 1 6 8/ place b o or was m ore li kel y e x plai ne d b y e x p os ure t o C C X 1 6 8/ place b o 
t ha n b y ot her ca us es. 
T he rel ati o ns hi p of c ortic oster oi d use, c ycl o p h os p ha mi de, rit u xi ma b, or azat hi o pri ne use  t o a n 
a d verse e ve nt will als o b e deter mi ne d b y t he I n vesti gat or a n d S p o ns or b as e d o n t he f oll o wi n g 
defi niti o ns: 
•  Pr o ba bl y N ot Relat e d:  t he a d vers e e v e nt was m ore li kel y e x plai ne d b y ca uses ot her t ha n 
c ortic oster oi d , c ycl o p h os p ha mi de, rit u xi ma b, or azat hi o pri ne use.   
•  P ossi bl y Relate d:  t he c ortic oster oi d , c ycl o p h os p ha mi de, rit u xi ma b, or azat hi o pri ne 
a d mi nistrati o n a n d t he a d verse e v e nt occ urre n ce w ere reas o na bl y relate d i n ti me, a n d t he 
A E was e x plai ne d e q uall y w ell b y ca uses ot her t ha n c ortic oster oi d , c ycl o p h os p ha mi de, 
rit u xi ma b, or azat hi o pri ne use or w as m ore li kel y e x plai ne d b y e x p os ure t o c ortic oster oi d, 
c ycl o p h os p ha mi de, rit u xi ma b, or azat hi o pri ne use t ha n b y ot her ca us es. 
A seri o us a d verse e v e nt ( S A E) is defi ne d as a n y u nt o war d me dical o cc urre nce t hat at a n y d os e:  
•  Res ults i n deat h. 
•  Is life-t hreate ni n g (i.e., t h e patie nt was, i n t he o pi ni o n of t he I n vesti gat or, at i m me diate 
ris k of deat h fr o m t he e v e nt as it occ urre d). 
•  Re q uires or pr ol o n gs h os pitalizati o n. 
•  Res ults i n persiste nt or si g nifica nt disa bilit y or i nca pacit y.  
•  Is a c o n ge nital a n o mal y or birt h defect. 
•  Is a n i m p orta nt a n d si g nifica nt me dical e ve nt t hat, base d o n a p pr o priate me dical 
j u d g me nt, ma y je o p ar dize t he patie nt a n d/ or ma y re q uire me di cal or s ur gical i nter ve nti o n 
t o pre ve nt o ne of t h e ot her o utc o mes defi ni n g s eri o us. Mali g na n cies a n d i nfecti o ns 
re q uiri n g a nti bi otics are c o nsi dere d seri o us. 
Electi ve s ur ger y, alrea d y k n o w n d uri n g s cree ni n g t o occ ur i n c o urse of t he st u d y a n d electi ve 
h os pitalizati o ns f or c o n ve nie nce of t he s u bject w hic h are clearl y u nrelate d t o a n y m e dical 
c o n diti o n, a n d a gree d bet wee n t he i n vesti gat or a n d t he s u bject p ri or t o ra n d o mizati o n, will n ot 
ha ve t o be r e p orte d as S A Es. 
A s us pecte d u ne x pecte d seri o us a d vers e reacti o n ( S U S A R) is defi ne d as a n S A E t hat is 
c o nsi dere d at least p ossi bl y rel ate d t o st u d y dr u g ( C C X 1 6 8/ place b o) a n d t h at is u ne x pecte d, i.e., 
n ot descri be d i n ter ms of nat ure, se v erit y, or fre q u e nc y i n t he c urre nt I n v esti gat or’s Br oc h ur e. 
S af et y l a b or at or y tests are perf or me d fre q ue ntl y o ver t he c o urs e of t he st u d y. L a b or at or y 
re p orts wit h a b n or m al fi n di n gs will be re vie w e d b y t he I n vesti g at or a n d t he Me dic al 
M o nit or a n d t he I n vesti g at or will be a d vise d t o f oll o w p atie nts wit h n ot a bl y hi g h li ver 
f u ncti o ns tests cl osel y a n d t o t a ke a p pr o pri ate ste ps, s uc h as p ote nti all y disc o nti n ui n g st u d y 
me dic ati o n, i n c ase t he a b n or m alities persist.  A d diti o n all y, if a s u bject de vel o ps gr a de t w o 
or w orse le u k o pe ni a ( W B C < 3 x 1 0 9/ L) O R a n a bs ol ute ne utr o p hil c o u nt < 1 x 1 09/ L, 
d osi n g wit h C C X 1 6 8 or pl ace b o m ust be ce ase d i n t his s u bject. St u d y dr u g m a y be res u me d 
o nl y if W B C a n d a bs ol ute ne utr o p hil c o u nt b ot h e xcee d t he l o w e r li mit of t he res pecti ve 




C he m o Ce ntr y x, I n c.                            C O N FI D E N TI A L                                Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8                                                                                                                    A me n d me nt 4. 0 
                                                                   Pa ge 8 4 of 1 1 3                               1 8 Se pte m ber 2 0 1 5 
7. 3.  
P h ar m ac o ki netic Assess me nts  
C o nce ntrati o ns of C C X 1 6 8 (a n d p ote ntial meta b olites) will be deter mi ne d i n plas ma fr o m 6- m L  
bl o o d sa m ples c ollecte d i n K 3E D T A t u bes o n Da ys 1, 8, 1 5, 2 2, 2 9, 4 3, 5 7, 7 1,  a n d 8 5. T he 
sa m ples o n Da y 1 will be c ollecte d j ust pri or t o t he m or ni n g d os e o n t hat d a y, a n d at 0. 5, 1, 2, 3, 
4, a n d 6 h o urs f oll o wi n g t he m or ni n g d ose. T h e date a n d ti me of t he last d ose pri or t o t he sa m ple 
c ollecti o ns o n Da ys 8, 1 5, 2 2, 2 9, 4 3, 5 7, 7 1,  a n d 8 5 will be rec or d e d i n t he e C R F. T he bl o o d 
sa m ples will be mi x e d ge ntl y a n d ke pt o n w et ice u ntil ce ntrif u ge d ( wit hi n 3 0 mi n utes after 
c ollecti o n) at a p pr o xi matel y 2 0 0 0 x g, f or a p pr o xi matel y 1 0 mi n utes. Res ulta nt plas ma nee ds t o 
be s plit i nt o t hree ~ 1 - m L ali q u ots a n d tra nsferre d t o t hree a p pr o priatel y la b ele d p ol y pr o p yl e ne 
t u bes a n d st ore d at a p pr o xi matel y - 7 0° C or b el o w u ntil a nal ysis. If t he site d oes n ot ha ve access 
t o a - 7 0° C freezer, t he sa m ples m ust be p ut o n dr y ice a n d s hi p pe d t o t he ce ntral la b orat or y as 
e x pe diti o usl y as p ossi ble, or st ore d at - 2 0° C a n d s hi p pe d t o t he ce ntral la b orat or y acc or di n g t o 
i nstr ucti o ns pr o vi de d.  
T otal plas ma c o nce ntrati o ns of C C X 1 6 8 (a n d p ote ntial meta b olites) will be deter mi ne d usi n g 
vali date d a nal ytical met h o ds. I n a d diti o n, plas ma c o nce ntrati o ns o f c o-me dicati o ns a n d rele v a nt 
acti ve meta b olites, s uc h as c ycl o p h os p ha mi de, 4- h y dr o x yc ycl o p h os p ha mi de or ot her 
meta b olites , rit u xi ma b, pre d nis o ne, a n d pre d nis ol o ne ma y be meas ure d i n t hese sa m ples. T hese 
plas ma sa m ples ma y als o be use d t o meas ure c yt o ki nes , c o m ple me nt fra g m e nts, or ot her mar k ers 
ass ociate d wit h A N C A v asc ulitis.  
A re pr ese ntati ve uri n e sa m ple fr o m a 6 - h o ur uri nar y c ollecti o n f oll o wi n g t he m or ni n g d os e o n 
Da y 1 will be use d t o det er mi ne t he C C X 1 6 8 uri n e c o nce ntrati o n. After t he 6- h o ur c ollecti o n, t he 
uri ne v ol u me will be meas ure d a n d r ec or d e d, a n d a re prese ntati ve uri ne sa m ple ( 2 0 m L) will als o 
be st ore d at - 7 0° C or b el o w u ntil a nal ysis. If t h e site d oes n ot ha ve access t o a - 7 0° C freezer, t he 
sa m ples m ust be p ut o n dr y ice a n d s hi p pe d t o t he ce ntral la b orat or y as e x pe diti o usl y as p ossi ble, 
or st ore d at - 2 0° C a n d s hi p pe d t o t he ce ntral la b orat or y acc or di n g t o i nstr ucti o ns pr o vi de d.  
7. 4.  
P h ar m ac o d y n a mic Assess me nts  
A plas ma sa m ple will be c ollecte d o n D a ys 1, 2 9, 8 5, a n d 1 6 9 f or p har mac o d y na mic m ar ke r 
meas ure me nts, i ncl u di n g, f or e x a m ple, c ystati n C, c o m ple me nt fra g m e nts, a n d i nfla m mat or y 
c yt o ki ne a n d c h e m o ki ne le vels. T he P K plas ma sa m ples ma y als o b e use d f or t hese 
p har mac o d y n a mic  mar k er meas ure m e nts.  
Uri ne sa m ples will als o b e c ollecte d o n Da ys 1,  8, 1 5, 2 9, 5 7, 8 5, 1 1 3, a n d 1 6 9 f or bi o mar ker 
assess me nts i ncl u di n g, f or e x a m ple, re nal i nj ur y a n d i nfla m mati o n mar kers (e. g., ki d ne y i nj ur y 
m olec ule - 1 [KI M - 1] a n d ne utr o p hil gel ati nase-ass ociate d li p ocali n [ N G A L] ), c o m ple me nt 
fra g me nts, i nfla m mat or y c he mo ki ne a n d c yt o ki n e le vels.  
A sali va sa m ple will be c ollecte d o n Da y 1 fr o m s u bjects w h o ha ve pr o vi de d i nf or me d c o ns e nt 
f or assess me nt of ge netic mar kers of A N C A disease as well as t he c o m ple me nt pat h wa y. 
P ote ntial mar kers i ncl u de H L A D P B 1 * 0 4 0 1 , S E R PI N A 1 , P R T N 3 , a n d H L A -D Q . C 5a R 
p ol y m or p his m ma y als o be i n vesti gat e d. 
7. 5.  
St u d y C o m pleti o n a n d Wit h dr a w al  
Da y 1 6 9 will be t he last St u d y Da y f or all s u bjects. Pr oce d ures f or t his da y will be c o m plete d per 
t he Ti me a n d E ve nts Ta ble . T he s u bject’s c o n diti o n will be e val uate d b y t h e I n vesti gat or at t he 


C he m o Ce ntr y x, I n c.                            C O N FI D E N TI A L                                Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8                                                                                                                    A me n d me nt 4. 0 
                                                                   Pa ge 8 5 of 1 1 3                               1 8 Se pte m ber 2 0 1 5 
e n d of t he cli nical trial a n d a p pr o priate sta n d ar d of care me dical treat me nt will be pr o vi de d t o all 
s u bjects as nee d e d. F or earl y wit h dra w als ( pri or t o Da y 8 5), t he pr o ce d ures sc he d ule d f or St u d y 
Da y 8 5 will be perf or me d a n d rec or de d as a n Earl y Ter mi nati o n visit. F or wit h dra wals after D a y 
8 5, pr oce d ur es f or Da y 1 6 9 will be perf or me d. 
7. 6.  
D at a M o nit ori n g C o m mittee  
I n a d diti o n t o o n g oi n g safet y m o nit ori n g b y t he M e dical M o nit or a n d Cli nical staff, a n e xter nal 
Data M o nit ori n g C o m mittee ( D M C) will m o nit or t he safet y of s u bj ects, i ncl u di n g res c ue 
c ortic oster oi d use, o v er t he c o urse of t he st u d y. T he D M C will c o nsist of t w o i n de pe n de nt 
p h ysicia ns a n d o ne bi ostatisticia n. A D M C c harter will be de vel o pe d b ef ore start of t he st u d y a n d 
t he D M C will f u ncti o n acc or di n g t o t he c h arter. T he f oll o wi n g will be tri g gers f or a f or mal D M C 
data re vie w:  
St u d y Ste p 1  
1.  M ore t ha n 1 s u bject ha v e a S U S A R, or 
2.  M ore t ha n 3 s u bjects r e q uire resc u e I V ster oi ds, or 
3.  1 0 s u bjects c o m plete Da y 8 5 wit h o ut a S U S A R a n d at least 7 of t hese s u bj ects di d n ot 
re q uire r esc u e c ortic oster oi d treat me nt, or 
4.  W he n 1 2 s u bjects i n Ste p 1 ha ve reac h e d at least t he Da y 2 9 visit. 
St u d y Ste p 2  
1.  M ore t ha n 1 s u bject ha v e a S U S A R, or 
2.  M ore t ha n 3 s u bjects r e q uire res c u e I V ster oi ds, or 
3.  1 0 s u bjects c o m plete Da y 8 5 wit h o ut a S U S A R a n d at least 7 of t hese s u bj ects di d n ot 
re q uire r esc u e c ortic oster oi d treat me nt, or 
4.  W he n 1 2 s u bjects i n Ste p 2 ha ve reac h e d at least t he Da y 2 9 visit. 
St u d y Ste p 3  
1.  O nce e v er y 3 t o 6 m o nt hs, de pe n di n g o n t he e nr oll me nt rate, or 
2.  If a n y u na ntici p ate d saf et y iss ues occ ur. 
It is a ntici pate d t hat t here will be at least 6 D M C  meeti n gs: a kic k - off m eeti n g a n d fi ve m eeti n gs 
o ver t he c o urse of t he st u d y. A d h o c meeti n gs ma y be s c he d ule d if u n a ntici pate d safet y e ve nts 
occ ur. After r e vie w of data at eac h meeti n g, t he D M C will ma ke rec o m me n dati o ns a b o ut f urt her 
c o n d uct of t he st u d y. 
T he D M C will re vie w all S U S A Rs a n d deter mi ne, base d o n a caref ul c o nsi derati o n of t he e ve nts 
w het her t he S U S A R is m ost li kel y relat e d t o C C X 1 6 8. T his re vie w will ta k e all as pects i nt o 
acc o u nt, i ncl u di n g o nset of t he S U S A R relati ve t o d osi n g wit h C C X 1 6 8, c o urse of a d v erse e ve nt 
i n relati o n t o de-c h alle n ge a n d p ossi ble re-c halle n ge wit h st u d y me dicati o n, act ual treat me nt (i.e. 
C C X 1 6 8 or place b o) recei ve d, ot her p ote ntial ca uses f or t he S U S A R s uc h as c o nc o mita nt 
me dicati o n i ncl u di n g c ortic oster oi d use, c ycl o p h os p ha mi de use, rit u xi ma b use, ot her 
c o m or bi dities a n d u n derl yi n g c o n diti o ns, a n d ot her pre vi o us a d v erse e ve nts o bser ve d o ver t he 
c o urse of t he st u d y.  


C he m o Ce ntr y x, I n c.                            C O N FI D E N TI A L                                Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8                                                                                                                    A me n d me nt 4. 0 
                                                                   Pa ge 8 6 of 1 1 3                               1 8 Se pte m ber 2 0 1 5 
7. 7.  
St a tistic al Met h o ds 
Statistical a nal ysis of effi cac y d ata will be perf or m e d usi n g S A S ® ( S A S I nstit ute, C ar y, N C) 
s oft ware, b ase d o n a pre d efi ne d Statistical A nal ysis Pla n. Data a nal ysis a n d writi n g of a n 
I nte grate d Cli nical a n d Statistical Re p ort (I C S R) f or all st u d y dat a will be p erf or me d b y t h e 
desi g nat e d C R O i n acc or da nce wit h its S O Ps. Data a nal ysis of p har mac o ki netic data, usi n g 
Wi n N o nli n s oft ware, a n d writi n g of a p har mac o ki netic a p pe n di x t o t he I C S R will be perf or me d 
b y a d esi g nate d p har mac o ki netic tea m i n acc or da nce wit h its S O Ps.  
U nless s pecifie d ot her wise, all statistical testi n g will be o ne -si de d a n d wit h t he t y pe I err or rate at 
α = 0. 0 5. I n t he c o nte xt of t his bei n g a P hase 2 tri al, n o a dj ust me nt f or m ulti plicit y will be ma de. 
N o missi n g data will be i m p ute d.  
7. 8.  
S u bject P o p ul ati o n s 
F or t he p ur p os es of dat a a nal ysis, t he I T T P o p ulati o n will i ncl u de all s u bjects w h o are 
ra n d o mize d, ha ve r ecei v ed at least o ne d os e of st u d y dr u g, a n d ha v e at least o ne p ost baseli ne 
B V A S assess me nt. T h e s afet y p o p ulati o n will i ncl u de all s u bjects w h o are ra n d o mize d a n d ha ve 
recei v e d at least o ne d ose of st u d y dr u g. A per pr ot oc ol ( P P) p o p ulati o n ma y als o be defi ne d if 
t here are maj or pr ot oc ol de viati o ns t hat c o ul d affect st u d y o utc o me. 
7. 9.  
S afet y E n d p oi nts  
T he pri mar y safet y e n d p oi nt is t he s u bject i nci de nce of a d v erse e ve nts.  
Ot her saf et y e n d p oi nts i ncl u de: 
1.  S u bject i nci de nce of e ve nts p ossi bl y ass ociate d wit h gl uc oc ortic oi d us e: seri o us i nfecti o ns, 
ne w -o nset dia b etes mellit us/ h y per gl yce mia, b o ne fract ure, pe ptic ulcer disease, cat aracts, 
ne w o nset/ w orse ni n g h y p erte nsi o n, wei g ht gai n m ore t ha n 1 0 k g, a n d ps yc hiatric dis or ders; 
2.  S u bject i nci de nce of i nfecti o ns, seri o us i nfecti o ns, se vere i nfecti o ns (i.e., Gra de 3), a n d 
i nfecti o ns lea di n g t o s u bject wit h dra w al fr o m t he st u d y; 
3.  C ha n ge fr o m bas eli ne i n all safet y la b orat or y para meters;  
4.  C ha n ge fr o m bas eli ne i n vital si g ns; 
5.  I n ci de nce of cli nicall y si g nifica nt E C G c ha n ges fr o m baseli ne. 
7. 1 0.  
Eff ic ac y E n d p oi nts 
T he pri mar y efficac y e n d p oi nt is t he pr o p orti o n of s u bjects ac hie vi n g disease res p o nse at Da y 8 5 
defi ne d as B V A S perce nt re d ucti o n fr o m bas eli ne of at least 5 0 % pl us n o w orse ni n g i n a n y b o d y 
s yste m c o m p o ne nt.  
Ot her efficac y e n d p oi nts i ncl u de: 
1.  I n p atie nts wit h he mat uri a a n d al b u mi n uria at b aseli ne, t he pr o p orti o n of s u bjects ac hie vi n g 
re nal r es p o nse at D a y 8 5; re nal r es p o nse is defi ne d as a n i m pr o ve me nt i n para meters of re n al 
vasc ulitis:  
a. a n i ncrease fr o m baseli ne t o Da y 8 5 i n e G F R ( M D R D ser u m creati ni ne e q u ati o n), pl us 


C he m o Ce ntr y x, I n c.                            C O N FI D E N TI A L                                Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8                                                                                                                    A me n d me nt 4. 0 
                                                                   Pa ge 8 7 of 1 1 3                               1 8 Se pte m ber 2 0 1 5 
b.  a decr eas e fr o m bas eli ne t o Da y 8 5 i n h e mat uria (ce ntral la b orat or y micr os c o pic c o u nt of 
uri nar y R B Cs), pl us 
c. a decr eas e fr o m bas eli ne t o Da y 8 5 i n al b u mi n uria (first m or ni n g uri nar y 
al b u mi n:creati ni ne rati o).  
2.  Pr o p orti o n of s u bjects ac hie vi n g diseas e re missi o n at Da y 8 5 defi ne d as B V A S of 0 or 1 pl us 
n o w orse ni n g i n e G F R a n d uri nar y R B C c o u nt < 1 0/ h pf; 
3.  Perce nt  c h a n ge fr o m bas eli ne t o Da y 8 5 i n B V A S; 
4.  C ha n ge a n d perce nt c ha n ge fr o m baseli ne t o D a y 8 5 i n e G F R; 
5.  I n s u bjects wit h baseli ne he mat uria > 5 R B Cs/ h pf, t he pr o p orti o n of s u bjects a n d ti me t o first 
ac hie vi n g uri nar y R B C c o u nt ≤ 5/ h pf at a n y ti me d uri n g t he 8 4- da y treat me nt peri o d; 
6.  I n s u bjects wit h baseli ne he mat uria ≥ 3 0 R B Cs/ h pf, t he pr o p orti o n of s u bjects a n d ti me t o 
first ac hie vi n g uri nar y R B C c o u nt < 3 0/ h pf at a n y ti me d uri n g t he 8 4- d a y treat me nt peri o d; 
7.  I n s u bjects wit h he mat uri a at baseli ne, t he p erce nt c ha n ge fr o m baseli ne t o Da y 8 5 i n uri nar y 
R B C c o u nt; 
8.  I n s u bjects wit h al b u mi n uria at baseli ne, t h e perce nt c ha n ge fr o m baseli ne t o Da y 8 5 i n 
uri nar y A C R;  
9.  Perce nt c h a n ge fr o m bas eli ne t o Da y 8 5 i n uri nar y M C P -1:creati ni ne rati o;  
1 0.  Pr o p orti o n of s u bjects re q u iri n g r esc ue I V or oral gl uc o c ortic oi d treat me nt; 
1 1.  C ha n ge fr o m bas eli ne t o Da y 8 5 i n t he Vas c ulitis Da ma ge I n d e x ( V DI); 
1 2.  C ha n ge fr o m bas eli ne t o Da y 8 5 i n healt h- relate d q ualit y- of-life as meas ure d b y t h e S h ort 
F or m - 3 6 versi o n 2. 0 ( S F- 3 6 v 2) a n d E ur o Q O L- 5 D-5 L  ( E Q- 5 D-5 L);  
Ot her e n d p oi nts i ncl u de: 
1.  T otal c u m ulati ve st u d y -s u p plie d pre d nis o ne d ose a n d d urati o n of d osi n g d uri n g t he 8 4-da y 
treat me nt peri o d; 
2.  T otal c u m ulati ve s yste mi c c ortic oster oi d d os e (a n y use) a n d d urati o n of d osi n g d uri n g t he 
8 4- da y d osi n g p eri o d; 
3.  T otal c u m ulati ve c ycl o p h os p ha mi de or rit u xi ma b d ose a n d d urati o n of d osi n g d uri n g t he 8 4-
da y d osi n g p eri o d;  
4.  Perce nt c h a n ge fr o m bas eli ne i n hs C R P ;  
5.  Perce nt c h a n ge fr o m bas eli ne i n A N C A (a nti - P R 3 a n d a nti-M P O) at D a y 8 5,  
6.  Pr o p orti o n of patie nts bec o mi n g A N C A n e gati ve at Da y 8 5; a n d  
7.  C ha n ge a n d perce nt c ha n ge fr o m baseli ne i n plas ma a n d uri ne bi o mar k ers. 
T he 8 4- da y f oll o w- u p peri o d res ults f or t he e n d p oi nts liste d a b o ve will als o be s u m marize d.  


C he m o Ce ntr y x, I n c.                            C O N FI D E N TI A L                                Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8                                                                                                                    A me n d me nt 4. 0 
                                                                   Pa ge 8 8 of 1 1 3                               1 8 Se pte m ber 2 0 1 5 
7. 1 1.  
P h ar m ac o ki netic E n d p oi nts  
Plas ma sa m ples will be c ollecte d o n Da ys 1, 8, 1 5, 2 2, 2 9, 4 3, 5 7, 7 1, a n d 8 5 t o deter mi ne t he P K 
pr ofile of C C X 1 6 8 a n d p ote ntial meta b olites . T he f oll o wi n g para meters will be deter mi ne d o n 
Da y 1, w here p ossi ble: 
C ma x    
Ma xi m u m plas ma c o nce ntrati o n  
T ma x     
 
Ti me of ma xi m u m plas ma c o nce ntrati o n  
A U C 0- 6 
Area u n d er t he plas ma c o nce ntrati o n -ti me c ur ve fr o m Ti me 0 t o H o ur 6 o n Da y 1 
C mi n     
Tr o u g h l e vel plas ma c o n ce ntrati o ns at p ost-D a y 1 visits  
7. 1 2.  
P h ar m ac o d y n a mic E n d p oi nts  
Plas ma sa m ples will be c ollecte d o n Da ys 1, 2 9, 8 5, a n d 1 6 9 t o meas ure p ote ntial c ha n ge a n d 
perce nt c ha n ge  fr o m bas eli ne i n bi o mar kers s uc h as c ystati n C, c o m ple me nt fra g me nts, 
i nfla m mat or y c he m o ki ne a n d c yt o ki ne le vels. T h e c ystati n C le vels ma y b e use d i n calc ulati n g 
e G F R c h a n ges fr o m baseli ne usi n g t he f oll o wi n g e q uati o n: 
e G F R c ys ( m L/ min/ 1. 7 3 m 2) = 1 2 7. 7 x (c ystati n C i n m g/ L)−1. 1 7  x ( A ge) −0. 1 3  x ( 0. 9 1 if fe male) 
x ( 1. 0 6 if Africa n -A m eri ca n/ Blac k)  
Uri ne sa m ples will be c ollecte d o n Da ys 1, 8, 1 5, 2 9, 5 7, 8 5, 1 1 3, a n d 1 6 9 t o meas ure p ote ntial 
c ha n ge a n d per ce nt c h a n ge  fr o m baseli ne i n bi om ar kers s u c h as re nal i nj ur y a n d i nfl a m mati o n 
mar kers ( e. g., KI M - 1 a n d N G A L ), c o m ple m e nt fra g me nts, i nfla m mat or y c h e m o ki ne a n d 
c yt o ki ne l e vels. 
A sali va sa m ple will be c ollecte d o n Da y 1 t o e v al uate t he effect of ge netic mar kers o n treat me nt 
res p o nse t o C C X 1 6 8 treat me nt. P ote ntial mar kers i ncl u de H L A D P B 1 * 0 4 0 1 , S E R PI N A 1 , 
P R T N 3 , a n d H L A -D Q . C 5a R p ol y m or p his m ma y als o be i n vesti gate d. 
7. 1 3.  
St atistic al A n al ysis Met h o d ol o g y  
A statistical a nal ysis pla n wit h s pecific details of all t he pla n ne d a nal yses will be ge n erat e d a n d 
a p pr o ve d bef ore u n bli n di n g t he d ata f or a nal ysis. 
7. 1 3. 1.  
B aseli ne C h ar acteristics  
All s u bject baseli ne c h ar acteristics a n d d e m o gra p hic data (a ge, s e x, race, et h nicit y, wei g ht, 
hei g ht, b o d y mass i n de x, s m o ki n g stat us, E C G, T B scree n res u lts, viral test res ults, A N C A, 
vasc ulitis disease d urati o n  (fr o m ti me of first i n d u cti o n treat me nt), B V A S, V DI, S F - 3 6 v 2, E Q-
5 D- 5 L,  hs C R P, e G F R, h e mat uria, uri ne R B C casts, pr otei n uria ( A C R), gl o mer ular 
hist o pat h ol o g y (if bi o ps y was ta ke n), uri nar y M C P- 1:creati ni ne rati o, p h ys ical e x a mi nati o n 
a b n or malities, me dical hist or y, pr e vi o us ( wit hi n 6 m o nt hs of scree ni n g) a n d c o nc o mita nt 
me dicati o ns (i ncl u di n g v asc ulitis me dicati o n use) at st u d y e ntr y will be liste d b y treat me nt 
gr o u p, st u d y ce nter, a n d s u bject n u m ber, a n d will als o be s u m marize d b y treat me nt gr o u p a n d 
ste p of t he st u d y. Data f or C C X 1 6 8 treate d s u bjects recei vi n g a re d uce d starti n g pre d nis o ne d os e 
fr o m Ste ps 1 a n d 3, a n d data f or C C X 1 6 8 treate d s u bjects recei vi n g a n o st u d y-s u p plie d 
pre d nis o ne fr o m Ste ps 2 a n d 3  will be c o m bi ne d f or s u m mar y a n d a n al ys es p ur p oses. Data f or 


C he m o Ce ntr y x, I n c.                            C O N FI D E N TI A L                                Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8                                                                                                                    A me n d me nt 4. 0 
                                                                   Pa ge 8 9 of 1 1 3                               1 8 Se pte m ber 2 0 1 5 
place b o treate d s u b jects fr o m Ste ps 1, 2, a n d 3 will be c o m bi ne d f or s u m mar y a n d a nal ys es 
p ur p oses. Data will als o be prese nt e d se paratel y f or t he c ycl o p h os p ha mi de a n d rit u xi ma b strata. 
T he n u m ber of p atie nts r a n d o mize d, c o m plete d, or disc o nti n ue d fr o m t he st u d y, al o n g wit h t he 
reas o n f or disc o nti n uati o n, will be prese nte d o v erall a n d b y treat me nt gr o u p. Patie nt c o u nt b y 
a nal ysis p o p ulati o n will als o be ta b ulate d.  
7. 1 3. 2.  
S af et y A n al yses 
Safet y a nal yses wi ll be perf or me d o n t h e Safet y P o p ulati o n. T he t w o C C X 1 6 8 treat me nt gr o u p s 
will be c o m pare d t o t he sta n dar d of care place b o gr o u p i n ter ms of t he s u bje ct i nci de nce of 
a d verse e ve nts.  
All safet y data will be s u m marize d descri pti vel y b y treat me nt gr o u p.  
A d verse e ve n ts will be c o de d usi n g Me d D R A a n d liste d, i ncl u di n g all a vaila ble i nf or mati o n of 
i nterest s uc h as o nset a n d res ol uti o n dates, st u d y d a y of o nset rel ati ve t o first d osi n g da y, 
se verit y, seri o us ness, ca usal relati o ns hi p t o st u d y me dicati o n, c ortic oster oi d , c ycl o p h os p ha mi de, 
or rit u xi ma b  use, acti o n ta ke n, a n d o utc o m e. A d verse e ve nts will be c o nsi d ere d as “ pre-
treat me nt” if t hes e occ ur pri or t o t he ti me of a d mi nistrati o n of t he first d ose of st u d y m e dicati o n. 
All ot her a d vers e e ve nts will be c o nsi dere d “treat me nt -e mer ge nt”. Pre-treat me nt a d verse e ve nts 
will be liste d se paratel y fr o m treat me nt -e m er ge nt a d verse e ve nts. S y m pt o ms or si g ns of 
vasc ulitis will be c o nsi dere d a d verse e v e nts if t hes e i ncrease i n se verit y or fre q ue nc y w hile a 
s u bject is o n-st u d y. A d v erse e ve nts will be liste d b y s u bject, a n d treat me nt gr o u p. A Es will be 
s u m marize d b y treat me nt gr o u p a n d ta b ulate d s e paratel y f or ma xi m u m se verit y, relati o ns hi p t o 
st u d y dr u g, a n d relati o ns hi p t o s yst e mic c ortic oster oi d, c ycl o p h os p ha mi de, or rit u xi ma b use. A Es 
lea di n g t o wit h dr a wal a n d S A Es will be ta b ulate d se parat el y. 
Safet y l a b orat or y d ata will be liste d b y treat me nt gr o u p a n d s u bject n u m ber, a n d will be 
s u m marize d b y treat me nt gr o u p. Act ual la b orat or y val ues a n d c h a n ge fr o m baseli ne i n la b orat or y 
val ues wil l be liste d a n d s u m marize d. La b orat or y val ues o utsi de t he refere nce ra n ges will be 
fla g ge d i n t he listi n gs. La b orat or y s hift ta bles fr o m baseli ne t o s u bse q ue nt st u d y visits will als o 
be ge ner ate d f or all saf et y la b or at or y para meters.  Vital si g ns data will be s u m marize d si milarl y. 
I n ci de nce of cli nicall y si g nifica nt E C G c ha n ges fr o m baseli ne will be s u m marize d b y treat me nt 
gr o u p. 
N o i nfere ntial statistical a nal ysis will be p erf or m e d o n a d verse e ve nt or ot h er safet y  d ata.   
7. 1 3. 3.  
Effic ac y A n al yses 
T he pri mar y effic ac y e n d p oi nt is t he pr o p orti o n of s u bjects ac hie vi n g disease res p o nse at Da y 8 5 
defi ne d as B V A S perce nt re d ucti o n fr o m bas eli ne of at least 5 0 % pl us n o w orse ni n g i n a n y b o d y 
s yste m c o m p o ne nt.  
T he pr o p orti o n of s u bjects ac hie vi n g dise ase res p o nse  will be c alc ulate d f or t he c o m paris o n 
bet wee n eac h C C X 1 6 8 gr o u p a gai nst t he S O C gr o u p. If t he l o wer b o u n d of t he 1-si de d 9 5 % 
c o nfi de nce i nter val f or t h e diff ere nce ( C C X 1 6 8 mi n us c o ntr ol gr o u p)  is greater t ha n - 0. 2 0, t he 
res pecti v e C C X 1 6 8 g r o u p will be c o nsi dere d n ot i nferi or t o t he S O C gr o u p. If t he l o w er 
b o u n d is gre ater t h a n 0 . 0, t he res pecti ve C C X 1 6 8 gr o u p will be c o nsi dere d s u peri or t o t he S O C 
gr o u p i n ac hie vi n g t he disease r es p o nse. C o nti n u o us varia bles will be a n al yze d usi n g mi xe d 
eff ects m o del f or re pe at e d me as ures ( M M R M) wit h treat me nt gr o u p , visit, tre at me nt-b y -visit 


C he m o Ce ntr y x, I n c.                            C O N FI D E N TI A L                                Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8                                                                                                                    A me n d me nt 4. 0 
                                                                   Pa ge 9 0 of 1 1 3                               1 8 Se pte m ber 2 0 1 5 
i nter acti o n, a n d ra n d o mizati o n strata ( ne wl y di a g n ose d A A V or rela pse d A A V, rit u xi ma b or 
c ycl o p h os p ha mi de, P R 3 or M P O A N C A) as f act o rs, a n d b aseli ne as c o variate. S u bjects will be 
c o nsi dere d as re pe at e d me as ure u nits o ver visits. T he M M R M m o del will be c o nstr ucte d 
f or t he tre at me nt peri o d as w ell as f or t he e ntire st u d y peri o d. P oi nt esti mates a n d 
c orres p o n di n g 9 5 % c o nfi de nce i nter v als will be c o m p ute d f or t he differe n ce bet wee n eac h 
C C X 1 6 8 gr o u p a n d t h e place b o gr o u p usi n g si m ple c o ntr ast fr o m t he m o del. A d diti o n all y, 
a n al ysis of c o v ari a nce ( A N C O V A) wit h t he s a me f act ors a n d c o v ari ates will be a p plie d f or 
t he bet w ee n gr o u p c o m p aris o n at e ac h visit. Data t hat are n ot n or m all y distri b ute d, e. g., 
ur i nar y A C R will be l o g- tra nsf or me d b ef ore a nal ysis. 
Cate g orical varia bles will be a nal yz e d usi n g t he C M H test. S u bjects recei vi n g resc ue ster oi ds 
after D a y 1 b ut bef ore D a y 8 5 + 7 da ys (i. e. Da y 9 2) will be c o nsi dere d as treat me nt fail ures. 
S u m mar y statisti cs will be calc ul ate d f or eac h of t he effi cac y e n d p oi nts. F or cate g orical 
e n d p oi nts, n u m bers a n d perce nt a ges will be calc ulate d. F or c o nti n u o us varia bles, n u m bers, 
mea ns, me dia ns, ra n ges, a n d sta n dar d d e viati o ns will be calc ulate d. Ge o m etric mea ns will be 
calc ulate d f or uri nar y A C R, uri nar y R B C c o u nt, uri nar y M C P- 1:creati ni ne, a n d hs C R P. S hift 
ta bles will be ge ner ate d f or uri nar y para met ers s uc h as he mat uria a n d al b u mi n uria. Res ults will 
be prese nt e d se paratel y f or eac h Ste p of t he st u d y, a n d als o c o m bi ne d f or t hree gr o u ps: ( 1) t he 
place b o s u bjects (st a n dar d of car e gr o u p) acr oss all t hree ste ps, ( 2) t he C C X 1 6 8 s u bjects 
recei vi n g a l o w d os e st u d y-s u p plie d pre d nis o ne d ose fr o m Ste ps 1 a n d 3, a n d ( 3) t he C C X 1 6 8 
s u bjects recei vi n g n o st u d y-s u p plie d pre d nis o ne fr o m Ste ps 2 a n d 3. Res ults will als o be 
prese nte d b y str at u m f or eac h of t h e t hree stratificati o n fact ors, ne wl y di a g n ose d vs. rela pse d 
patie nts, rit u xi ma b vs. c ycl o p h os p ha mi de use, a n d P R 3 vs. M P O p ositi ve A N C A. If t he t w o 
C C X 1 6 8 gr o u ps s h o we d a si milar res p o nse, t he t w o C C X 1 6 8 gr o u ps ma y b e c o m bi ne d f or 
testi n g a gai nst t he c o ntr ol gr o u p. 
T he mai n effi cac y a nal ysis will be i n t he I T T p o p ulati o n. Se nsiti vit y a nal ys es ma y  als o be d o ne 
i n t he P P p o p ulati o n 
Baseli ne is defi ne d as t he last val ue pri or t o start of d osi n g wit h st u d y me di cati o n (t y pi call y t he 
Da y 1 pre- d ose val u e).  
7. 1 3. 4.  
C o v ari ates 
T he eff ect of t h e f oll o wi n g bas eli ne para meters (i n a d diti o n t o t he defi ne d strata, i.e., ne wl y 
dia g n ose d A A V or rela ps e d A A V, rit u xi ma b or c ycl o p h os p ha mi de bac k gr o u n d treat me nt, a n d 
P R 3 vs. M P O A N C A ) o n st u d y o utc o me ma y b e assesse d: 
•  Ge n der  
•  B MI  
•  A ge at dia g n osis of A A V 
•  S m o ki n g 
•  S u bject’s a ge a n d et h nicit y (if pla usi ble) 
•  Baseli ne e G F R  
•  Baseli ne h e mat uria  


C he m o Ce ntr y x, I n c.                            C O N FI D E N TI A L                                Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8                                                                                                                    A me n d me nt 4. 0 
                                                                   Pa ge 9 1 of 1 1 3                               1 8 Se pte m ber 2 0 1 5 
•  Baseli ne uri nar y A C R  
•  Baseli ne B V A S  
•  Baseli ne V DI  
•  Baseli ne hs C R P  
•  Baseli ne uri n ar y M C P -1:creati ni ne rati o  
7. 1 3. 5.  
P h ar m a c o ki netic A n al ysis 
I n di vi d ual plas ma c o nce ntrati o ns of C C X 1 6 8 a n d rele va nt meta b olites, as well as c o-me di cati o ns 
a n d acti ve meta b olites s u c h as c ycl o p h os p h a mi de, 4- h y dr o x yc ycl o p h os p h a mi de, pre d nis o ne, a n d 
pre d nis ol o ne, a n d rit u xi ma b (if meas ure d) will b e liste d, pl otte d, a n d s u m marize d descri pti vel y 
a n d gra p hicall y. P har mac o ki netic para met ers will be calc ul ate d base d o n pl as ma C C X 1 6 8 
c o nce ntr ati o ns at t he ti me of sa m ple c ollecti o n i n relati o n t o ti me of a d mi nistrati o n of t he m ost 
rece nt d os e of st u d y me dicati o n. Plas ma le vels of si g nifica nt met a b olites ma y als o be deter mi ne d 
a n d P K para m eters calc ulate d.  
It is of i nterest t o e val uat e w het her t h e P K pr ofile of s u bjects wit h A N C A-ass ociate d vasc ulitis is 
si milar t o t he pr ofile i n healt h y v ol u nteers. T he rel ati o ns hi p bet wee n P K para meters a n d re n al 
f u ncti o n base d o n e G F R will als o be e val uate d. T he data ma y als o be use d t o e val uate t he P K/ P D 
relati o ns hi p of C C X 1 6 8 treat me nt. T o t his e n d, t he c ha n ge a n d/ or perce nt c ha n ge fr o m b aseli ne 
i n e G F R, B V A S, V DI, s er u m hs C R P, A N C A (a nti- P R 3 a n d a nti- M P O), A C R, he mat uria, 
uri nar y M C P- 1:creati ni ne rati o , or ot her bi o mar kers ma y b e use d as P D mar kers.  
7. 1 4.  
S a m ple Size J ustific ati o n  
Ste p 1 a n d 2  
Ste ps 1 a n d 2 are d esi g n e d t o pr o vi de a n i nitial e val uati o n o n t he safet y a n d feasi bilit y usi n g 
C C X 1 6 8 as a c ortic oster oi d s pari n g or re place me nt t hera p y d uri n g i n d ucti o n of re missi o n. T he 
st u d y will pr o gress t o t he ne xt ste p ( Ste p 3) if A A V disease acti vit y i n t he  maj orit y of s u bjects o n 
C C X 1 6 8 ( > 5 0 %) is mai ntai ne d wit h o ut t he nee d f or resc u e c ortic oster oi d t hera p y. A s a m ple size 
of 1 2 s u bjects ( 8 acti ve a n d 4 place b o) p er ste p f or t he first t w o ste ps of t he st u d y was c h ose n 
base d o n feasi bilit y. Ta bl e  9 pr o vi des t he pr o b a bilities of c o nti n ui n g t o t he ne xt ste p f or a ra n ge 
of tr ue per ce nta ges of patie nts re q uiri n g resc u e t hera p y.  
T a ble 9:  
Perce nt Pr o b a bilit y of C o nti n u ati o n t o Ne xt Ste p  
Tr ue Perce nt a ge of s u bjects 
re q uiri n g resc u e t her a p y  
Pr o b a bilit y of c o nti n ui n g t o 
ne xt ste p *  
1 0 %  
1 0 0 %  
2 0 %  
9 4 %  
3 0 %  
8 1 %  
4 0 %  
6 0 %  
5 0 %  
3 6 %  
6 0 %  
1 7 %  
7 0 %  
6 %  
8 0 %  
1 %  


C he m o Ce ntr y x, I n c.                            C O N FI D E N TI A L                                Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8                                                                                                                    A me n d me nt 4. 0 
                                                                   Pa ge 9 2 of 1 1 3                               1 8 Se pte m ber 2 0 1 5 
Tr ue Perce nt a ge of s u bjects 
re q uiri n g resc u e t her a p y  
Pr o b a bilit y of c o nti n ui n g t o 
ne xt ste p *  
9 0 %  
0 %  
* Pr o ba bilit y t h at at least 5 of t he 8 s u bjects o n C C X 1 6 8 di d n ot re q uire resc ue t hera p y. 
T he ta ble is rea d as f oll o ws: if t h e tr ue perce nta ge of s u bjects re q uiri n g res c ue t hera p y is 8 0 %, 
t he n t here is a 1 % c ha n ce t hat t he st u d y w o ul d c o nti n ue t o t he ne xt ste p. 
Ste p 3  
A sa m ple size of 3 6 s u bjects i n Ste p 3, 1 2 i n eac h of t he C C X 1 6 8 gr o u ps a n d 1 2 i n t he place b o, 
will pr o vi de a t o tal of a p pr o xi matel y 6 0 s u bjects acr oss all t hree ste ps, a n d ~ 2 0 s u bjects i n eac h 
of t he treat me nt gr o u ps. Ass u mi n g a c o ntr ol gr o u p B V A S res p o ns e of 4 4 % at Da y 8 5 a n d a 
C C X 1 6 8 gr o u p r es p o nse of 8 6 %, a sa m ple size of 2 0 i n eac h gr o u p will pr o vi de a p pr o xi mat el y 
9 0 % p o wer f or t he pri mar y efficac y a nal ysis . 
7. 1 5.  
I nteri m A n al ysis 
Efficac y a n d safet y data fr o m t he st u d y will be s u m marize d f or re vie w b y t he D M C at vari o us 
p oi nts o ver t he c o urse of t he st u d y (s ee secti o n 7. 6 ). T he D M C c harter will i ncl u de details of t he  
a nal ys es.  
Data fr o m Ste p 1 a n d 2 will be s u m marize d bef ore pr ocee di n g t o Ste p 3. N o i nfere ntial statistical 
a nal ysis will be p erf or m e d bef ore c o m pleti o n of Ste p 3. T heref ore, n o al p ha pe nalt y will be 
ta ke n. W hen all s u bjects ha ve c o m plete d t h e 8 4 -d a y treat me nt peri o d, data ma y b e a nal yze d t o 
ma ke decisi o ns re gar di n g f ut ure cli nical de vel o p me nt pla ns.     
7. 1 6.  
Pr ot oc ol De vi ati o ns  
Si g nifica nt pr ot oc ol d e viati o ns will be liste d a n d s u m m arize d b y cate g or y. T he effect of 
si g nifica nt pr ot o c ol de viati o ns o n t he safet y a n d efficac y o utc o m es will be assesse d b y 
c o n d ucti n g s e nsiti vit y a n al yses e x cl u di n g s u bjects a n d/ or st u d y visits wit h si g nifica nt pr ot o c ol 
de viati o ns. 
8.  
S T U D Y C O M P L E TI O N A N D  T E R MI N A TI O N  
8. 1.  
St u d y C o m pleti o n  
A s u bject  has c o m plete d t he st u d y w h e n s/ he has c o m plete d t he st u d y pr o ce d ures per pr ot oc ol. 
8. 2.  
St u d y Ter mi n ati o n  
T he e n d of st u d y is defi n e d as t he last st u d y visit of t he last cli nical trial s u bject.  


C he m o Ce ntr y x, I n c.                            C O N FI D E N TI A L                                Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8                                                                                                                    A me n d me nt 4. 0 
                                                                   Pa ge 9 3 of 1 1 3                               1 8 Se pte m ber 2 0 1 5 
9.  
R E G U L A T O R Y A N D A D MI N I S T R A TI V E R E Q UI R E M E NT S 
9. 1.  
I n vesti g at or Res p o nsi bilities 
Pri or t o trial i nitiati o n, t he I n vesti gat or will pr o vi d e t he S p o ns or wit h a f ull y e x ec ute d a n d si g n e d 
F D A F or m 1 5 7 2 a n d a Fi na ncial Discl os ure F or m. Fi na ncial Discl os ure F or ms als o will be 
c o m plete d f or all S u b- I n vesti gat ors liste d o n t he F or m 1 5 7 2 w h o will be i n v ol ve d directl y i n t he 
treat me nt or e v al uati o n of researc h s u bjects i n t his trial.  
T he st u d y will be c o n d ucte d i n acc or d a nce wit h t he Decl arati o n of H elsi n ki (a me n de d b y t he 
5 9t h W orl d Me dical Ass ociati o n Ge neral Asse m bl y, Oct o b er 2 0 0 8) a n d G o o d Cli nical Practice 
( G C P) acc or di n g t o I nter nati o nal C o nfere n ce o n Har m o nisati o n (I C H) g ui deli nes.  S pecificall y, 
t he st u d y is base d o n a de q uatel y p erf or me d la b orat or y a n d a ni mal e x peri me ntati o n; t he st u d y 
will be c o n d ucte d u n der a pr ot oc ol re vi e we d b y a pr o perl y c o nstit ute d I R B/ E C; t he st u d y will be 
c o n d ucte d b y scie ntifi call y a n d me di call y q ualifie d pers o ns; t he be nefits of t he st u d y are i n 
pr o p orti o n t o t he ris ks; t he ri g hts a n d welfare of t he s u bjects will be res p ect e d; a n d eac h s u bject 
will gi ve his / her i nf or m e d c o nse nt bef ore a n y pr ot oc ol-s pecific t ests or e val uati o ns are 
perf or me d.  
9. 2.  
I nstit uti o n al Re vie w B o ar d or Et hics C o m mittee 
Pri or t o i nitiati n g t he st u d y, t he I n vesti gat or will o btai n writte n c o nfir mati o n fr o m t he I R B/ E C 
t hat t he I R B/ E C was pr o perl y c o nstit ute d a n d met t he defi niti o n of all U nite d States C o de of 
Fe deral Re g ulati o ns Title 2 1, Secti o n 3 1 2. 3( b) a n d Part 5 6, a n d/ or t he a p pli ca ble l ocal, re gi o nal 
or nati o nal Re g ulat or y r e q u ire me nts. A c o p y of t h e c o n fir mati o n will be pr o vi de d t o t he S p o ns or.  
T he Pri nci pal I n vesti gat or will pr o vi de t he I R B/ E C wit h all a p pr o priate materials, i ncl u di n g t he 
pr ot oc ol a n d I nf or me d C o nse nt d oc u me nts. T he trial will n ot be i nitiate d u ntil I R B/ E C a p pr o v al 
of t he pr ot oc ol, t he I nf or me d C o nse nt d oc u me nt, a n d all recr uiti n g materials are o btai ne d i n 
writi n g b y t he I n v esti gat or a n d c o pies are recei ve d b y t he S p o ns or. A p pr o priate re p orts o n t he 
pr o gress of t he st u d y will be ma de t o t he I R B/ E C a n d t he S p o ns or b y t h e Pri nci pal I n vesti gat or 
i n acc or d a nce wit h a p plica ble g o ver n me ntal re g ulati o ns a n d i n a gree me nt wit h t he p olic y 
esta blis he d b y t h e S p o ns or. 
9. 3.  
I nf or me d C o nse nt 
A pr o perl y e x ec ute d, writte n, a n d a p pr o pri atel y e x plai ne d I nf or me d C o nse nt F or m, i n 
c o m plia nce wit h t he D eclarati o n of Helsi n ki, I C H G C P, a n d U S C o de of Fe deral Re g ul ati o ns f or 
Pr otecti o n of H u ma n S u bjects ( 2 1 C F R 5 0. 2 5[a, b], C F R 5 0. 2 7, a n d C F R Part 4 6, S u b part A), 
will be si g ne d b y eac h s u bject pri or t o e nteri n g t he  trial. Eit her t he i n vesti gat or or t he 
i n vesti gat or’s d esi g n ee will o btai n t he c o nse nt of t he st u d y s u bject. T he s u bject will be pr o vi de d 
as m uc h ti me as necessar y t o re vi e w t he d oc u m e nt, t o i n q uire a b o ut details of t he trial, a n d t o 
deci de w h et her or n ot t o partici pate i n t he st u d y. T he i nf or me d c o ns e nt will be si g ne d a n d d ate d 
b y t he st u d y s u bject a n d b y t he p ers o n w h o c o n d u cte d t he i nf or me d c o nse nt disc ussi o n. T he 
I n v esti gat or will pr o vi de a c o p y of t he si g ne d I nf or me d C o nse nt F or m t o eac h s u bject a n d will 
mai ntai n a c o p y i n t he s u bject’s rec or d file.  


C he m o Ce ntr y x, I n c.                            C O N FI D E N TI A L                                Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8                                                                                                                    A me n d me nt 4. 0 
                                                                   Pa ge 9 4 of 1 1 3                               1 8 Se pte m ber 2 0 1 5 
9. 4.  
Pr ot oc ol M o dific ati o ns  
O nl y t he S p o ns or ma y m o dif y t he pr ot oc ol. A me n d me nts t o t he pr ot oc ol will be ma de o nl y after 
c o ns ultati o n a n d a gree me nt bet wee n t he S p o ns or a n d t he Pri nci pal I n vesti gat or. T he o nl y 
e x ce pti o n is w he n t he I n vesti gat or c o nsi ders t hat a s u bject’s safet y w o ul d be c o m pr o m ise d 
wit h o ut i m me diate acti o n. I n t his cir c u msta nce, i m me diate a p pr o v al of t he c hair pers o n of t h e 
I R B/ E C m ust be s o u g ht, a n d t he I n vesti gat or s h o ul d i nf or m t he S p o ns or’s Me dical M o nit or a n d 
t he f ull I R B/ E C wit hi n fi ve w or kin g da ys  after t he e mer ge n c y occ urre d. All  ot her a me n d me nts 
t hat ha ve a n i m pact o n s u bject ris k or t he st u d y o bjecti ves, a n d/ or t hat re q uire re visi o n of t he 
I nf or me d C o nse nt F or m, m ust recei ve a p pr o val fr o m t he I R B/ E C pri or t o t heir i m ple me ntati o n, 
e x ce pt w he n t he c h a n ges i n v ol ve o nl y l o gistical o r a d mi nistrati ve as pects of t he trial. T he 
I R B/ E C m ust be n otifie d of c ha n ges t h at are m a de t o st u d y c o ntact p ers o n nel, b ut I R B/ E C 
re vie w or a p pr o val of t hese c ha n ges is n ot re q uire d. If pr ot oc ol a me n d m e nts are s u bsta ntial a n d 
are li kel y t o ha ve a n i m pact o n t he saf et y of t he tri al s u bjects or t o c ha n ge t he i nter pret ati o n of 
t he scie ntific d oc u me nts i n s u p p ort of t he c o n d uct of t he trial, or if t he y are ot her wise si g nifica nt, 
t he s p o ns or s hall n otif y t he c o m pete nt a ut h orities of t he Me m ber State or Me m ber States 
c o ncer n e d of t he reas o ns f or, a n d c o nt e nt of, t hese a me n d me nts acc or di n g t o t he E ur o pea n 
Directi ve “ Detaile d g ui d a nce o n t he re q uest t o t he c o m pete nt a ut h orities f or a ut h orizati o n of a 
cli nical trial o n a me dical pr o d ucts f or h u ma n us e, t he n otificati o n of s u bsta ntial a me n d me nts 
a n d t he decl arati o n of t he e n d of trial ( C T- 1)( 2 0 1 0/ C 8 2/ 0 1)”.  
9. 5.  
Re g ul at or y D oc u me nt ati o n  
All re g ul at or y d oc u me nt ati o n i ncl u di n g re g ul at or y s u b missi o ns, 1 5 7 2 f or ms, a n d c orres p o n d e nce 
re gar di n g t his st u d y will be ke pt b y t he S po ns or. T he C R O t hat will c o n d uct t he st u d y o n be h alf 
of t he S p o ns or will mai ntai n all st u d y d oc u me nt ati o n acc or di n g t o t heir S O Ps. Cli nical trial 
relate d d oc u m e nts will be arc hi v e d f or at least 1 0 years acc or di n g t o nati o n al S we dis h a n d E U 
re g ulati o ns ( L V F S 2 0 0 3: 3). 
9. 6.  
S u bject I de ntific ati o n Re gister  
T he I n vesti gat or a grees t o c o m plete a s u bject i de ntificati o n re gister, w hic h will be use d f or t he 
p ur p ose of l o n g- ter m f oll o w- u p, if nee d e d. T his f or m will be treate d as c o nfi de ntial, a n d will be 
file d b y t he I n vesti gat or i n a sec ur e l oc ke d pl ace. Ot her wise, all re p orts a n d c o m m u nicati o ns 
relati n g t o t he st u d y will i de ntif y partici pa nts b y i nitials a n d assi g ne d n u m ber o nl y. 
9. 7.  
Rec or d Rete nti o n  
T he I n vesti gat or m ust ret ai n all  st u d y rec or ds re q uire d b y t h e S p o ns or a n d b y t he a p plica ble 
re g ulati o ns i n a sec ure a n d safe f acilit y. T h e I n vesti gat or m ust c o ns ult a S p o ns or C R A bef ore 
dis p osal of a n y st u d y rec or ds, a n d m ust n otif y t he S p o ns or of a n y c ha n ge i n t he l ocati o n, 
dis p ositi o n, or c ust o d y of t he st u d y files. T h e F D A re q uires rete nti o n of rec or ds f or t w o years 
f oll o wi n g t he dat e a mar keti n g a p plicati o n is a p pr o ve d, or f or t w o years aft er t he F D A is n otifie d 
t hat t he I N D is disc o nti n ue d if t here is no mar keti n g a p plicati o n. Rec or ds m ust be retai ne d f or a 
peri o d at least a s l o n g as t hat s pecifie d b y F D A re g ulati o ns. Cli nical trial relate d d oc u me nts will 
be arc hi v e d f or at least 1 0 years acc or di n g t o nati o nal S we dis h a n d E U re g ulati o ns ( L V F S 
2 0 0 3: 3). 


C he m o Ce ntr y x, I n c.                            C O N FI D E N TI A L                                Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8                                                                                                                    A me n d me nt 4. 0 
                                                                   Pa ge 9 5 of 1 1 3                               1 8 Se pte m ber 2 0 1 5 
9. 8.  
C ase Re p ort F or m C o m pleti o n  
Electr o nic  Case Re p ort F or ms ( C R Fs) will be ge n erate d f or eac h s u bject. T he electr o nic s yste m 
m ust c o m pl y wit h C F R 2 1 Part 1 1. 
It is t he p olic y of t he S p o ns or t hat st u d y data m ust be verifia bl e t o t he s o urce data, w hic h 
necessitates access t o all ori gi nal r ec or di n gs, la b orat or y re p orts, a n d s u bjects’ rec or ds. T he 
I n v esti gat or m ust t heref ore a gr ee t o all o w access t o s u bjects’ rec or ds, a n d s o urce dat a m ust be 
ma de  a vaila ble f or all st u d y dat a. T he s u bjects ( or t heir le gal re prese ntati ves) m ust als o all o w 
access t o t he s u bjects’ m e dical rec or ds, a n d t h e y w ill be i nf or me d of t his re q uire me nt a n d will 
i n dicate t heir a gr ee me nt w he n gi vi n g I nf or me d C o nse nt. U p o n c o m pleti o n of t he st u d y, 
electr o nic c o pies of t he C R Fs will be pr o vi de d t o t he i n vesti gat ors a n d s h o ul d be i ncl u de d as part 
of his/ her st u d y files a n d retai ne d as p er F D A or l o cal re g ulati o ns. 
9. 9.  
M o nit ori n g  
At i nter vals d uri n g t h e st u d y, as well as after t he c o m pleti o n of s u bject e nr oll me nt, t he st u d y 
ce nter will be m o nit ore d b y a C R A f or c o m plia nce, w hic h will i ncl u de e ns uri n g t hat acc urate a n d 
c o m plete data are r ec or d e d o n C R Fs, a n d r e vi e wi n g s o urce d oc u m e ntati o n a n d dr u g 
acc o u nta bilit y rec or ds. T he st u d y will be c o n d u cte d acc or di n g t o t he pri nci ples of G C P as 
acce pte d i n t he U nite d States a n d acc or di n g t o C P M P/I C H/ 1 3 5/ 9 5. 
9. 1 0.  
O n- site A u dits 
T he S p o ns or’s re pr ese ntati ves will visit t he st u d y ce nter pri or t o i nitiati o n of t he st u d y t o re vie w 
wit h t he ce nter p ers o n nel i nf or mati o n re gar di n g t h e i n vesti gati o nal a ge nt, pr ot oc ol re q uire m e nts, 
m o nit ori n g re q uir e me nts, a n d re p orti n g of s eri o us a d verse e ve nts. 
I n cert ain circ u msta nces, a sec o n dar y a u dit ma y be c o n d ucte d b y me m bers of a Q ualit y 
Ass ura nce gr o u p desi g nate d b y t he S p o ns or. T h e I n vesti gat or will be i nf or me d if t his is t o ta ke 
place a n d a d vise d as t o t he nat ure of t he a u dit. Re prese ntati ves of t h e F o o d a n d Dr u g 
A d mi nistrati o n ( F D A) a n d/ or re pres e ntati ves of ot her re g ul at or y a ut h orities ma y als o c o n d uct a n 
a u dit of t he st u d y. If i nf or me d of s uc h a n a u dit, t he I n vesti gat or s h o ul d n otif y t he S p o ns or 
i m me diatel y. 
9. 1 1.  
Use of I nf or m ati o n a n d P u blic ati o n  
It is u n derst o o d b y t he I n vesti gat or t h at t he i nf or m ati o n ge n erat e d i n t his st u d y will be use d b y 
t he S p o ns or i n c o n necti o n wit h t he de vel o p me nt of t he pr o d uct a n d t h eref ore ma y be discl ose d t o 
g o ver n me nt a ge n cies i n vari o us c o u ntries. T o all o w f or t he use of i nf or mati o n deri ve d fr o m t he 
st u d y, it is u n derst o o d t hat t he I n vesti gat or is o bli ge d t o pr o vi de t he S p o ns or wit h c o m plete test 
res ults, all st u d y data, a n d access t o all st u d y rec or ds. 
T he S p o ns or rec o g nizes t he i m p orta nce of c o m m u nicati n g st u d y data a n d will discl ose or p u blis h 
t he res ults i n a s uita ble f or m re gar dl ess of o utc o me. T he S p o ns or ma y elect t o p u blis h s o me or 
all of t he res ults of t his st u d y i n scie ntific j o ur n als, at se mi nars or c o nfere nces, a n d/ or i n ot her 
ma n ner(s) it s o c h o os es. Res ults fr o m t his st u d y  s hall n ot be ma de a v aila ble t o a n y t hir d part y b y 
t he i n vesti gati n g tea m wit h o ut t he e x press per missi o n of t he S p o ns or. 


C he m o Ce ntr y x, I n c.                            C O N FI D E N TI A L                                Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8                                                                                                                    A me n d me nt 4. 0 
                                                                   Pa ge 9 6 of 1 1 3                               1 8 Se pte m ber 2 0 1 5 
1 0.  
R E F E R E N C E S  
A be K, Mi yaza ki M, K oji T, et al. 2 0 0 1 E n ha nce d es pressi o n of c o m ple me nt C 5a rece pt or 
m R N A i n h u ma n disease d ki d ne y assesse d b y i n sit u h y bri dizati o n. Ki d ne y I nt 6 0: 1 3 7- 1 4 6. 
J e n nette J C, Fal k RJ, Bac o n P A, et al. 2 0 1 3 2 0 1 2 Re vise d I nter nati o nal C h a pel Hill C o nse ns us 
C o nfere nce N o me n clat ur e of V as c uliti des. Art hritis R he u m 6 5 : 1– 1 1. 
J o nes J H, Ter vaert J W C, Ha user T, et al. 2 0 1 0 Rit u xi ma b vers us c ycl o p h os p ha mi de i n A N C A-
ass ociate d r e nal vas c ulitis. N E n gl J Me d 3 6 3: 2 1 1- 2 2 0. 
Sc hrei ber A, Xia o H, J e n nette J C, et al.  2 0 0 9. C 5a Rece pt or m e diates ne utr o p hil acti vati o n a n d 
A N C A -i n d uce d gl o mer ul o ne p hritis. J A m S oc Ne p hr ol 2 0: 2 8 9- 2 9 8. 
St o ne J H, Mer kel P A, S piera R, et al. 2 0 1 0 Rit u xi ma b vers us c ycl o p h os p h a mi de f or A N C A-
ass ociate d vas c ulitis. N E n gl J Me d 3 6 3: 2 2 1- 2 3 2. 


C he m o Ce ntr y x, I n c.                            C O N FI D E N TI A L                                Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8                                                                                                                    A me n d me nt 4. 0 
                                                                   Pa ge 9 7 of 1 1 3                               1 8 Se pte m ber 2 0 1 5 
1 1.  
A P P E N DI C E S  
1 1. 1.  
St ate me nt of O bli g ati o ns of S p o ns or, M o nit or, a n d Cli nic al 
I n vesti g at or 
S p o ns or a n d M o nit or 
If t he S p o ns or is n ot fa miliar wit h t he St u d y Site, t he S p o ns or or its desi g n ate d re prese ntati ve, 
will:  
A.   C o n d uct a prest u d y visit t o: 
1.  Esta blis h t he acce pt a bilit y of t he facilit y, t he recr uit me nt p ote ntial a n d t he sta n dar d of p atie nt 
care at t his site, a n d rec or d t his i n a writte n re p ort.  
2.  Disc uss t he pr o p ose d cli nical trial wit h t he I n vesti gat or, re vie w t h e C R F re q uire me nts, a n d 
s u p pl y t he I n v esti gat or Br oc h ure a n d t he draft pr ot oc ol f or re vi e w a n d a p pr o val. 
3.  Disc uss wit h t he I n v esti gat or F D A a n d ot her re g ul at or y re q uire me n ts wit h res pect t o 
I nf or me d C o nse nt, c o m p ete nt a ut h orit y ( C A) a n d et hics c o m mittee ( E C) a p pr o val of t he trial, 
t he pr ot oc ol, pr ot oc ol a m e n d me nts, a n d I nf or me d C o nse nt c ha n ges. 
B.   C o n d uct peri o dic site visits t o: 
1.  Ass ure a d h ere n ce t o t he pr ot oc ol.  
2.  Re vie w C R F s a n d me dical rec or ds f or acc urac y a n d c o m plete ness of i nf or mati o n. 
3.  E x a mi ne p har mac y rec or ds f or d oc u me ntati o n of q ua ntit y a n d d ate of recei pt of 
i n vesti gati o nal s u p plies, dis pe nsati o n a n d acc o u nta bilit y data f or a d mi nistrati o n t o eac h 
s u bject, l oss of materials, c o nta mi nati o n, a n d u n use d s u p plies. 
4.  Rec or d a n d r e p ort o bser v ati o ns o n t he pr o gress of t he trial a n d c o nti n ue d acce pta bilit y of t h e 
facilities i n a Site Visit Re p ort.   
5.  Re vie w I n vesti gat or files f or re q uire d d oc u me nts, e. g., pr ot oc ols, pr ot oc ol a me n d me nts, C A 
a n d E C a p pr o vals ( pr ot oc ols, a me n d me nts, I nf or me d C o nse nt, etc.), E C c h arter a n d 
me m bers hi p, a n d c o m m u nicati o ns bet wee n t he E C a n d t he I n vesti gat or.  
Cli nical I n vesti gat or  
A.  E C  
T he I n vesti gat or m ust ass ure t he m o nit or t hat t he E C: 
1.  Meets F D A re g u lati o ns as defi ne d i n 2 1 C F R Part 5 6 a n d ot her a p plica ble I C H a n d G C P 
re q uire me nts. 
2.  Has a ut h orit y dele gate d b y t he p are nt i nstit uti o n a n d f o u n d i n E C b y- la ws, o perati o n 
g ui deli nes, or c hart er t o a p pr o ve or disa p pr o v e cli nical trials a n d pr ot oc ols, i ncl u di n g 
I nf or me d C o nse nt F or ms a n d ot her d oc u me nts ( pr ot oc ol a me n d me nts, i nf or mati o n t o be 
s u p plie d t o s u bjects c o ncer ni n g I nf or m e d C o nse nt, etc.). 


C he m o Ce ntr y x, I n c.                            C O N FI D E N TI A L                                Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8                                                                                                                    A me n d me nt 4. 0 
                                                                   Pa ge 9 8 of 1 1 3                               1 8 Se pte m ber 2 0 1 5 
3.  C o m plies wit h pr o per pers o n nel ma ke u p of a n E C a n d mai ntai ns a n acti ve u p- t o-date r oster 
of all E C me m bers partici pati n g i n t he meeti n gs.  
4.  C o n ve nes meeti n gs usi n g acce pta ble r ules of or der f or ma ki n g decisi o ns, rec or di n g s u c h 
decisi o ns, a n d i m ple me nti n g t he m. 
5.  Files c o ntai n ( a) d oc u me ntati o n of its decisi o ns s uc h as are f o u n d i n E C mi n utes a n d 
c orres p o n d e nce, ( b) writte n g ui d eli nes or b y-la ws g o ver ni n g E C f u ncti o ns, (c) pr ot oc ols, ( d) 
pr ot oc ol i nf or mati o n t o be s u p plie d t o t he s u bject, (e) c orres p o n d e nce b et w ee n t he E C a n d 
t he I n v esti gat or (I nf or me d C o nse nt F or m c ha n ges, pr ot oc ol a me n d me nts, et c.). 
B.   I nf or me d C o nse nt of H u ma n S u bjects.  
T he Pri nci pal I n vesti gat or m ust ass ure t he m o nit or t hat t he I nf or me d C o nse nt F or m: 
1.  Meets F D A re g ulati o ns as defi ne d i n 2 1 C F R Part 5 0 I nf or me d C o nse nt, a n d ot her a p plica ble 
I C H a n d G C P re q uire m e nts. 
2.  Has bee n a p pr o ve d b y t h e E C, i ncl u di n g, w h e n re q uire d, i nf or mati o n t o be gi ve n t o t he 
s u bject re gar di n g t h e trial i n w hic h s/ he is e nr olle d. 
a. T he I nf or m e d C o nse nt F or m i ncl u des t he Basic Ele me nts a n d a n y A d diti o nal Ele me nts 
necessar y.  
b.  T he s u bject a n d a st u d y ce nter re pres e ntati ve si g n t he I nf or me d C o nse nt F or m a n d t he 
s u bject is gi ve n a c o p y. 
C.   St ora ge a n d Dis pe nsi n g of St u d y S u p plies. 
T he I n vesti gat or ( or p har macist or p har mac y tec h nicia n) m ust de m o nstrate t o t he m o nit or t hat: 
1.  A de q uate a n d acc urat e writte n rec or ds s h o w recei pt a n d dis p ositi o n of all st u d y s u p plies, 
i ncl u di n g dates, s erial or l ot n u m bers, q ua ntities recei ve d, a n d eac h q ua ntit y dis pe nse d, 
a d mi nistere d, or use d, wit h i de ntificati o n of eac h s u bject. 
2.  P ur p ose a n d reas o ns ar e gi ve n i n writte n rec or ds f or st u d y material dis p osal, e. g., t he a m o u nt 
c o nta mi nate d, br o ke n, or l ost, a n d t he q ua ntit y ret ur ne d t o t he S p o ns or. 
D.  Case Re p ort F or ms.  
T he I n vesti gat or m ust ass ure t he m o nit or t hat: 
1.  Case re p ort f or ms, w he n c o m plete d, acc urat el y reflect t he me dical rec or ds o n eac h s u bject.  
2.  Case re p ort f or ms a n d m e dical rec or ds will be accessi ble t o t he m o nit or or F D A a n d ot h er 
Re g ulat or y i ns pect ors d uri n g site visits.  
E.  Files a n d Rec or ds.  
T he I n vesti gat or m ust ass ure t he q ualit y, i nte grit y, a n d c o nte nt of his or her files t hat will be 
i ns pecte d b y t he mo nit or a n d re g ulat or y i ns pect ors.  T he files m ust c o ntai n, at a mi ni m u m: 
1.  C orres p o n de nce bet w ee n t he E C a n d t he I n vesti gat or. 
2.  T he f oll o wi n g d o c u me nts: 
a. E C -a p pr o ve d pr ot oc ols. 
b.  E C -a p pr o ve d pr ot oc ol a me n d me nts. 


C he m o Ce ntr y x, I n c.                            C O N FI D E N TI A L                                Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8                                                                                                                    A me n d me nt 4. 0 
                                                                   Pa ge 9 9 of 1 1 3                               1 8 Se pte m ber 2 0 1 5 
c. E C -a p pr o ve d I nf or me d C o nse nt F or m a n d i nf or mati o n s u p plie d t o t he s u bject. 
d.  E C c harter, m e m bers hi p, a n d q ualificati o ns. 
3. Cli nical s u p plies:  
a. Rec or d of r ecei pt, date a n d q ua ntit y, a n d b atc h or l ot n u m ber. 
b.  Dis p ositi o n dates a n d q ua ntit y a d mi nistere d t o eac h s u bject. 
c. I n v e nt or y rec or ds. 
T he F D A re q uir es ret e n ti o n of rec or ds f or t w o years f oll o wi n g t he d ate a m ar keti n g a p plicati o n 
is a p pr o ve d, or f or t w o years after t he F D A is n otifie d t hat t he I N D is disc o nti n ue d if t here is n o 
mar keti n g a p plicati o n. Rec or ds m ust be retai ne d f or a peri o d at least as l o n g as t hat s p ecifie d b y 
F D A r e g ulati o ns. Cli nical trial relate d d oc u me nts will be arc hi ve d f or at least 1 0 years acc or di n g 
t o nati o nal S we dis h a n d E U re g ulati o ns ( L V F S 2 0 0 3: 3). 


C he m o Ce ntr y x, I n c.                            C O N FI D E N TI A L                                Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8                                                                                                                    A me n d me nt 4. 0 
                                                                   Pa ge 1 0 0 of 1 1 3                               1 8 Se pte m ber 2 0 1 5 
1 1. 2.  
I nf or me d C o nse nt F or m  
I n s ee ki n g I nf or me d C o nse nt, t he f oll o wi n g i nf or mati o n s hall be pr o vi de d t o eac h s u bject: 
1.  A state me nt t hat t he st u d y i n v ol ves researc h, a n e x pla nati o n of t he p ur p oses of t he researc h 
a n d t he e x pecte d d ur ati o n of t he s u bject's partici p ati o n, a descri pti o n of t he pr oce d ures t o be 
f oll o we d, a n d i de ntificati o n of a n y pr oce d ures w hic h are e x peri me ntal.  
2.  A descri pti o n of a n y reas o na bl y f oreseea ble ris ks or disc o mf orts t o t he s u bject. 
3.  A descri pti o n of a n y b e n efits t o t he s u bject or t o ot hers t hat ma y reas o na bl y be e x pecte d fr o m 
t he researc h. 
4.  A discl os ure of a p pr o priate alter na ti ve pr o ce d ures or c o urses of treat me nt, if a n y, t hat mi g ht 
be a d va nta ge o us t o t he s u bject. 
5.  A state me nt descri bi n g t he e xte nt, if a n y, t o w hic h c o nfi de ntialit y of rec or ds i de ntif yi n g t he 
s u bject will be mai ntai ne d a n d t hat n otes t he p ossi bilit y t hat t he F oo d a n d Dr u g 
A d mi nistrati o n or ot her Re g ulat or y a ge n c y ma y i ns pect t he rec or ds. 
6.  F or r esearc h i n v ol vi n g m ore t ha n mi ni mal ris k, a n e x pla nati o n as t o w het her a n y 
c o m pe nsati o n a n d as t o w het her a n y m e dical treat me nts are a v aila ble if i nj ur y o cc urs a n d, if 
s o, w hat t he y c o nsist of, or w her e f urt her i nf or mati o n ma y be o bt ai ne d. 
7.  A n e x pla nati o n of w h o m t o c o ntact f or a ns wers t o perti ne nt q uesti o ns a b o ut t he researc h a n d 
researc h s u bjects' ri g hts, a n d w h o m t o c o ntact i n t he e ve nt of a res earc h rel ate d i nj ur y t o t he 
s u bject. 
8.  A state me nt t hat partici p ati o n is v ol u ntar y, t hat ref usal t o partici pate will i n v ol ve n o pe nalt y 
or l oss of be nefits t o w hi c h t he s u bject is ot her wis e e ntitle d, a n d t hat t he s u bject ma y 
disc o nti n ue partici pati o n at a n y ti me wit h o ut pe nalt y or l oss  of be n efits t o w hic h t he s u bject 
is ot her wise e ntitle d. 
A D DI TI O N A L E L E M E N T S O F I N F O R M E D C O N S E N T  
1.  A state me nt t hat t he parti c ular treat me nt or pr oce d ure ma y i n v ol ve ris ks t o t he s u bject ( or t o 
t he e m br y o or f et us if t he s u bject is or ma y b ec o m e pre g na nt) w hic h are c urre ntl y 
u nf oreseea ble.  
2.  A ntici pate d circ u msta n ces u n der w hic h t he s u bject’s partici pati o n ma y be ter mi nate d b y t h e 
I n v esti gat or wit h o ut re gar d t o t he s u bject’s c o nse nt. 
3.  A n y a d diti o nal c osts t o t he s u bject t hat ma y res ult fr o m partici pati o n i n t he researc h. 
4.  T he c o nse q u e nces of a s u bject’s decisi o n t o wit h dra w fr o m t he researc h a n d pr oce d ures f or 
or derl y ter mi nati o n of p artici pati o n b y t he s u bject.  
5.  A state me nt t hat si g nifica nt ne w fi n di n gs de vel o p e d d uri n g t he c o urs e of t he researc h w hic h 
ma y r elate t o t he s u bject’s willi n g ness t o c o nti n ue partici pati o n will be pr o vi de d t o t he 
s u bject. 
6.  T he a p pr o xi mate n u m ber of s u bjects i n v ol ve d i n t he st u d y. 






C he m o Ce ntr y x, I n c.                            C O N FI D E N TI A L                                Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8                                                                                                                    A me n d me nt 4. 0 
                                                                   Pa ge 1 0 3 of 1 1 3                               1 8 Se pte m ber 2 0 1 5 
1 1. 4.  
V asc ulitis D a m a ge I n de x  
T he Vasc ulitis Da ma ge I n de x ( V DI) is use d t o rec or d c u m ulati ve or ga n d a ma ge si nce t he o ns et 
of vasc ulitis, re gar dl ess of w het her it is attri b ute d t o vasc ulitis. It d oes n ot gi ve a n i n dicati o n of 
c urre nt disease acti vit y; t he B V A S is use d t o rec or d t he latter. T he V DI sc ore ca n o nl y re mai n 
t he sa me or get w orse o v er ti me. Ne wl y dia g n os e d patie nts (i.e., wit hi n t he pre vi o us 3 m o nt hs) 
t y picall y will ha ve a V DI of zer o. I n patie nts wit h vasc ulitis dia g n ose d m ore t ha n 3 m o nt hs a g o, 
t he sc ore c o ul d be n o n-zer o. 
T he V DI will be s c ore d acc or di n g t o t he g ui da nce pr o vi de d b y R. A. L u q ma ni a n d C. Hall f or 
T he L ot hia n U ni versit y H os pitals N H S Tr ust ( 2 0 0 4). T he V DI ca pt ures da ma ge i n t he f oll o wi n g 
or ga n s yste ms: M usc ul os keletal, S ki n/ M uc o us me m bra nes, Oc ul ar, E N T, P ul m o nar y, 
Car di o vasc ular, Peri p her al vasc ular disease, G astr oi ntesti nal, Re nal, Ne ur o ps yc hiatric, a n d 
Ot her. O ne p oi nt will be sc ore d f or eac h p ositi ve ite m a n d t he V DI is t he s u m of all sc ores. Ite ms 
sc ore d at a partic ular visit will be carrie d f or w ar d t o s u bse q ue nt visits. 


C he m o Ce ntr y x, I n c.                            C O N FI D E N TI A L                                Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8                                                                                                                    A me n d me nt 4. 0 
                                                                   Pa ge 1 0 4 of 1 1 3                               1 8 Se pte m ber 2 0 1 5 
1 1. 5.  
Pre d nis o ne D ose Sc he d ule  
Ste ps 1 a n d 3 (f or C C X 1 6 8 gr o u p wit h re d uce d pre d nis o ne)  
S u bjects wit h a b o d y w ei g ht of ≥5 5 k g, w h o are ra n d o mize d t o t he C C X 1 6 8 gr o u p, will start at 
a n oral pr e d nis o ne d ose of 2 0 m g p er da y o n D a y 1 a n d will ta per t he d ose d o w n t o zer o b y t he 
e n d of t he 1 6 8- d a y st u d y peri o d. S u bjects wit h a b o d y w ei g ht less t ha n 5 5 k g will start at a n oral 
pre d nis o ne d ose of 1 5 m g per da y a n d will ta per t he d ose d o w n t o zer o b y t he e n d of t he 1 6 8- d a y 
st u d y peri o d.  
S u bjects wit h a b o d y w ei g ht of ≥5 5 k g, w h o are ra n d o mize d t o t he place b o gr o u p, will start at a n 
oral pre d nis o ne d os e of 6 0 m g per da y o n D a y 1 a n d will ta per t he d ose d o w n t o zer o b y t he e n d 
of t he 1 6 8- da y st u d y peri o d. S u bjects wit h a b o d y wei g ht less t ha n 5 5 k g will start at a n oral 
pre d nis o ne d ose of 4 5 m g per da y a n d will ta per t he d ose d o w n t o zer o b y t he e n d of t he 1 6 8- d a y 
st u d y peri o d. 
T he ta peri n g sc h e d ule f or s u bjects wit h b o d y wei g ht of ≥5 5 k g is pr o vi de d i n t he ta ble bel o w: 
St u d y D a ys  
C C X 1 6 8 Gr o u p  
Pl ace b o Gr o u p  
 
D ail y Pre d nis o ne D ose  
1 t o 7  
2 0 m g  
6 0 m g  
8 t o 1 4  
1 5 m g  
4 5 m g  
1 5 t o 2 1  
1 0 m g  
3 0 m g  
2 2 t o 2 8  
1 0 m g  
2 5 m g  
2 9 t o 3 5  
1 0 m g  
2 5 m g  
3 6 t o 4 2  
1 0 m g  
2 5 m g  
4 3 t o 4 9  
5 m g  
2 0 m g  
5 0 t o 5 6  
5 m g  
2 0 m g  
5 7 t o 6 3  
5 m g  
1 5 m g  
6 4 t o 7 0  
5 m g  
1 5 m g  
7 1 t o 7 7  
5 m g  
1 0 m g  
7 8 t o 8 4  
5 m g  
1 0 m g  
8 5 t o 9 8  
5 m g  
1 0 m g  
9 9 t o 1 4 0  
0  
5 m g  
1 4 1 t o 1 6 8  
0  
0  
T he ta peri n g sc h e d ule f or s u bjects wit h b o d y wei g ht less t ha n 5 5 k g is pr o vi de d i n t he ta ble 
bel o w: 
St u d y D a ys  
C C X 1 6 8 Gr o u p  
Pl ace b o Gr o u p  
 
D ail y Pre d nis o ne D ose  
1 t o 7  
1 5 m g  
4 5 m g  
8 t o 1 4  
1 5 m g  
4 5 m g  
1 5 t o 2 1  
1 0 m g  
3 0 m g  
2 2 t o 2 8  
1 0 m g  
2 5 m g  
2 9 t o 3 5  
1 0 m g  
2 5 m g  
3 6 t o 4 2  
1 0 m g  
2 5 m g  
4 3 t o 4 9  
5 m g  
2 0 m g  
5 0 t o 5 6  
5 m g  
2 0 m g  
5 7 t o 6 3  
5 m g  
1 5 m g  
6 4 t o 7 0  
5 m g  
1 5 m g  
7 1 t o 7 7  
5 m g  
1 0 m g  
7 8 t o 8 4  
5 m g  
1 0 m g  
8 5 t o 9 8  
5 m g  
1 0 m g  


C he m o Ce ntr y x, I n c.                            C O N FI D E N TI A L                                Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8                                                                                                                    A me n d me nt 4. 0 
                                                                   Pa ge 1 0 5 of 1 1 3                               1 8 Se pte m ber 2 0 1 5 
St u d y D a ys  
C C X 1 6 8 Gr o u p  
Pl ace b o Gr o u p  
 
D ail y Pre d nis o ne D ose  
9 9 t o 1 4 0  
0  
5 m g  
1 4 1 t o 1 6 8  
0  
0  
Ste ps 2 a n d 3 (f or C C X 1 6 8 gr o u p wit h n o  pre d nis o ne)  
S u bjects w h o are ra n d o mize d t o t he C C X 1 6 8 gr o u p will n ot recei ve a n y oral pre d nis o ne o ver t he 
c o urse of t he st u d y.  
S u bjects wit h a b o d y w ei g ht of ≥5 5 k g, w h o are ra n d o mize d t o t he place b o gr o u p, will start at a n 
oral pre d nis o ne d os e of 6 0 m g per da y a n d will ta per t he d ose d o w n t o zer o b y t he e n d of t he 
1 6 8- da y st u d y p eri o d. S u bjects wit h a b o d y wei g ht less t ha n 5 5 k g will start at a n oral pre d nis o ne 
d ose of 4 5 m g p er d a y a n d will ta per t he d ose d o w n t o zer o b y t he e n d of t h e 1 6 8- da y st u d y 
peri o d. 
T he ta peri n g sc h e d ule f or s u bjects wit h b o d y wei g ht of ≥5 5 k g is pr o vi de d i n t he ta ble bel o w: 
St u d y D a ys  
C C X 1 6 8 Gr o u p  
Pl ace b o Gr o u p  
 
D ail y Pre d nis o ne D ose  
1 t o 7  
0  
6 0 m g  
8 t o 1 4  
0  
4 5 m g  
1 5 t o 2 1  
0  
3 0 m g  
2 2 t o 2 8  
0  
2 5 m g  
2 9 t o 3 5  
0  
2 5 m g  
3 6 t o 4 2  
0  
2 5 m g  
4 3 t o 4 9  
0  
2 0 m g  
5 0 t o 5 6  
0  
2 0 m g  
5 7 t o 6 3  
0  
1 5 m g  
6 4 t o 7 0  
0  
1 5 m g  
7 1 t o 7 7  
0  
1 0 m g  
7 8 t o 8 4  
0  
1 0 m g  
8 5 t o 9 8  
0  
1 0 m g  
9 9 t o 1 4 0  
0  
5 m g  
1 4 1 t o 1 6 8  
0  
0  
T he ta peri n g sc h e d ule f or s u bjects wit h b o d y wei g ht less t ha n 5 5 k g is pr o vi de d i n t he ta ble 
bel o w: 
St u d y D a ys  
C C X 1 6 8 Gr o u p  
Pl ace b o Gr o u p  
 
D ail y Pre d nis o ne D ose  
1 t o 7  
0  
4 5 m g  
8 t o 1 4  
0  
4 5 m g  
1 5 t o 2 1  
0  
3 0 m g  
2 2 t o 2 8  
0  
2 5 m g  
2 9 t o 3 5  
0  
2 5 m g  
3 6 t o 4 2  
0  
2 5 m g  
4 3 t o 4 9  
0  
2 0 m g  
5 0 t o 5 6  
0  
2 0 m g  
5 7 t o 6 3  
0  
1 5 m g  
6 4 t o 7 0  
0  
1 5 m g  
7 1 t o 7 7  
0  
1 0 m g  
7 8 t o 8 4  
0  
1 0 m g  
8 5 t o 9 8  
0  
1 0 m g  
9 9 t o 1 4 0  
0  
5 m g  


C he m o Ce ntr y x, I n c.                            C O N FI D E N TI A L                                Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8                                                                                                                    A me n d me nt 4. 0 
                                                                   Pa ge 1 0 6 of 1 1 3                               1 8 Se pte m ber 2 0 1 5 
St u d y D a ys  
C C X 1 6 8 Gr o u p  
Pl ace b o Gr o u p  
 
D ail y Pre d nis o ne D ose  
1 4 1 t o 1 6 8  
0  
0  
N ote t hat all s u bjects will ta ke pre d nis o n e or matc hi n g place b o ca ps ules i n or der t o mai ntai n t he 
st u d y bli n di n g. D etaile d d osi n g i nstr ucti o ns will be pr o vi de d t o all s u bjects. 


C he m o Ce ntr y x, I n c.                            C O N FI D E N TI A L                                Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8                                                                                                                    A me n d me nt 4. 0 
                                                                   Pa ge 1 0 7 of 1 1 3                               1 8 Se pte m ber 2 0 1 5 
1 1. 6.  
C ycl o p h os p h a mi de a n d Az at hi o pri ne D osi n g  
•  C ycl o p h os p ha mi de d oses gi ve n fr o m Da y 1 t hr o u g h 8 5 m ust be gi ve n acc or di n g t o 
directi o ns pr o vi de d bel o w. 
•  A d ose of 1 5 m g/ k g c ycl o p h os p ha mi de will be gi ve n I V t o all s u bjects, u nless a l o wer 
d ose is re q uire d p er i nstr ucti o ns bel o w. T he ma xi m u m per mitte d d ose is 1. 2 g.  
•  Mes na a n d a ntie m etic treat me nt nee d t o be gi ve n acc or di n g t o l ocal  practice. 
O n da nsetr o n 8 m g is rec o m me n de d. 
•  C ycl o p h os p ha mi de n ee ds t o be a d mi nistere d o ver a 1- h o ur peri o d.  
•  C ycl o p h os p ha mi de d ose will be deter mi ne d b y f o ur fact ors: s u bject a ge, e G F R, W B C at 
t he st u d y visit, a n d W B C na dir i n bet wee n d os e p ulses ( w here a pplica bl e).  
•  A ge:  
− If < 6 0 years, a f ull d ose will be gi ve n ( u nl ess i nfl ue nce d b y t he ot h er t hree fact ors); 
− If 6 0 t o 7 0 years, t he d os e will be re d uce d b y 2. 5 m g/ k g; 
− If > 7 0 years, t he d os e will be re d uce d b y 5 m g/ k g. 
•  e G F R:  
− If ≥3 0 m L/ mi n, a f ull d os e will be gi v e n ( u nless i nfl ue nce d b y t he ot h er t hree fact ors);  
− If < 3 0 m L/ mi n, t he d ose will be re d uce d b y 2. 5 m g/ k g. 
•  W B C c o u nt at t he ti me of c ycl o p h os p ha mi de d ose (l ocal la b W B C c o u nts):  
− If ≥4 x 1 09/ L, a f ull d ose will be gi ve n ( u nl ess i nfl ue nce d b y t he ot h er t hree fact ors);  
− If 2 t o 3. 9 x 1 09/ L, t he d ose will be re d uce d b y 2 5 %; 
− If < 2 x 1 09/ L, t he d os e will be wit h hel d u ntil t he W B C c o u nt i ncreas es t o a b o ve 3 x 
1 0 9/ L. 
•  W B C c o u nt na dir i n bet wee n c ycl o p h os p ha mi de d oses:  
− If > 3 x 1 09/ L, a f ull d ose will be gi ve n ( u nl ess i nfl ue nce d b y t he ot h er t hree fact ors); 
− If 2 t o 3 x 1 09/ L, t he d ose will be re d uce d b y 2 0 %; 
− If 1 t o 1. 9 x 1 09/ L, t he d ose will be re d uce d b y 4 0 %;  
− If < 1 x 1 09/ L, t he n e xt d ose will be wit h hel d a n d f urt her d osi n g w o ul d o nl y be gi v e n 
if t he W B C is > 3 x 1 09/ L. 
•  N ote t hat t he c ycl o p h os p ha mi de d ose a dj ust me nt is c u m ulati ve, e. g., a s u bj ect > 7 0 years, 
wit h a n e G F R < 3 0 m L/ mi n, a n d a W B C at ti me of d osi n g of 3 x 1 0 9/ L, will recei v e a 
d ose of 5. 6 m g/ k g ( 1 5 m g/ k g mi n us 5 m g/ k g f or a ge, mi n us 2. 5 m g/ k g f or e G F R, a n d 
re d uce d 2 5 % f or W B C). 
•  Oral azat hi o pri ne will be starte d o n Da y 9 9 a n d c o nti n ue t hr o u g h D a y 1 6 8. T y picall y t he 
d ose will be i ncrease d gr a d uall y u ntil t he tar get d ose of 2 m g/ k g/ da y is reac he d aft er 2 


C he m o Ce ntr y x, I n c.                            C O N FI D E N TI A L                                Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8                                                                                                                    A me n d me nt 4. 0 
                                                                   Pa ge 1 0 8 of 1 1 3                               1 8 Se pte m ber 2 0 1 5 
wee ks. D oses will be r o u n de d d o w n t o t he nearest 2 5 m g i ncre me nt, e. g., a s u bject 
wei g hi n g 6 0 k g will ha v e a tar get d ose of 1 0 0 m g azat hi o pri ne per da y. T esti n g f or 
t hi o p uri ne S- met h yltra nsferas e ( T P M T) p ol y m or p his m a n d d ose a dj ust me nt s h o ul d be 
i m ple me nte d acc or di n g t o t he l ocal i nstit uti o n p olic y. Azat hi o pri ne will n ot be i nitiate d if 
t he W B C c o u nt is < 2 x 1 09/ L. I n t his case treat me nt i nitiati o n will be dela ye d u ntil t he 
W B C has i ncreas e d t o a b o ve 2 x 1 0
9/ L. I n case az at hi o pri ne is n ot t olerate d, met h otre x ate 
or m yc o p he n olate m of etil c o ul d be use d i nstea d. 


C he m o Ce ntr y x, I n c.                            C O N FI D E N TI A L                                Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8                                                                                                                    A me n d me nt 4. 0 
                                                                   Pa ge 1 0 9 of 1 1 3                               1 8 Se pte m ber 2 0 1 5 
1 1. 7.  
R it u xi m a b D osi n g 
•  T he f oll o wi n g d osi n g r e gi me n of rit u xi ma b will be gi ve n : 
− 3 7 5 m g / m2 gi ve n as a n I V i nf usi o n o n St u d y Da ys 1, 8, 1 5, a n d 2 2. 
•  It is rec o m me n de d t o pr e- me dicate b ef ore eac h i nf usi o n wit h aceta mi n o p he n a n d a n a nti-
hista mi ne.  F or t he first rit u xi ma b i nf usi o n, 1 0 0 m g met h yl pre d nis ol o ne, or e q ui vale nt is 
rec o m me n de d.  
•  F or t he first I V i nf usi o n, i nitiate t he i nf usi o n at a rate of 5 0 m g/ hr. I n t he a bse nce of 
i nf usi o n t o xicit y, i n creas e i nf usi o n rate b y 5 0 m g/ hr i ncre me nts e v er y 3 0 mi n utes, t o a 
ma xi m u m of 4 0 0 m g/ hr. 
•  F or s u bse q u e nt i nf usi o ns, i nitiate t he i nf usi o n at a rate of 1 0 0 m g/ hr. I n t h e a bse nce of 
i nf usi o n t o xicit y, i n creas e rate b y 1 0 0 m g/ hr i ncre me nts at 3 0-mi n ute i nter vals, t o a 
ma xi m u m of 4 0 0 m g/ hr.  


C he m o Ce ntr y x, I n c.                            C O N FI D E N TI A L                                Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8                                                                                                                    A me n d me nt 4. 0 
                                                                   Pa ge 1 1 0 of 1 1 3                               1 8 Se pte m ber 2 0 1 5 
1 1. 8.  
A d verse E ve nts Re p orte d wit h C ycl o p h os p h a mi de  
Carci n o ge nesis  
I n crease d ris k of ca n cer; m ost fre q ue ntl y, t h ese ha ve bee n uri n ar y bla d d er, m yel o pr oliferati ve, or 
l y m p h o pr olifer ati ve mali g na n cies. 
Car diac S yste m  
C o n gesti ve heart f ail ure, he m orr ha gic m y o car ditis, he m o pericar di u m, m y o car dial necr osis, 
pericar ditis  
Di gesti ve S yste m  
Na usea, v o miti n g, a n or e xia, a b d o mi nal disc o mf ort, a b d o mi nal pai n, diarr h ea, he m orr ha gic 
c olitis, oral m uc osal ulcerati o n, ja u n dice 
He mat o p oietic S yste m  
Le u k o p e nia, ne utr o pe nia, fe ver i n n e utr o pe nic p atie nts, t hr o m b oc yt o p e nia, a ne mia  
I nfecti o ns 
Viral, bacterial, f u n gal, pr ot oz oa n, or hel mi nt hic i nfecti o ns 
Re pr o d ucti ve S yste m 
C ycl o p h os p ha mi de i nterf eres wit h o o ge n esis a n d s per mat o ge nesis. It ma y ca use sterilit y i n b ot h 
se x es. N ote t hat o nl y p ost- me n o pa usal w o me n will be eli gi ble f or t his st u d y. Me n treate d wit h 
c ycl o p h os p ha mi de ma y d e vel o p oli g os per mia, az o os per mia, i m paire d se x ual p ote nc y or li bi d o, 
testic ular atr o p h y  
Res pirat or y S yste m  
I nterstitial p ne u m o nitis, i nterstitial p ul m o nar y fi br osis 
S ki n a n d Its Str uct ures 
Al o pecia,  s ki n ras h, s ki n pi g me ntati o n, nail dis or d ers, Ste ve ns-J o h ns o n s y n dr o me, t o xic 
e pi der mal necr ol ysis  
Uri nar y S yste m  
C ystitis, uri nar y bl a d der fi br osis, he m orr ha gic ureteritis, re nal t u b ular necr osis 
Ot her  
A na p h yl actic r eacti o ns; deat h ass ociat e d wit h a na p h y lactic reacti o ns, SI A D H (s y n dr o me of 
i na p pr o priate A D H secreti o n), malaise, ast he nia 


C he m o Ce ntr y x, I n c.                            C O N FI D E N TI A L                                Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8                                                                                                                    A me n d me nt 4. 0 
                                                                   Pa ge 1 1 1 of 1 1 3                               1 8 Se pte m ber 2 0 1 5 
1 1. 9.  
A d verse E ve nts Re p orte d wit h C ortic oster oi d Use  
Aller gi c Reacti o ns  
A na p h yl act oi d or h y pers e nsiti vit y reacti o ns, a na p h yla xis, a n gi oe d e ma 
Car di o vasc ular S yste m  
Bra d ycar di a, car diac arr est, car diac arr h yt h mias, car diac e nlar ge me nt, circ ulat or y c olla pse, 
c o n gesti ve heart fail ur e, E C G c ha n ges ca use d b y p otassi u m deficie nc y, e d e ma, fat e m b olis m, 
h y perte nsi o n or a g gra vati o n of h y p erte nsi o n, m y ocar dial r u pt ure f oll o wi n g rece nt m y ocar dial 
i nfarcti o n, necr otizi n g a n giitis, p ul m o nar y e de ma, s y nc o p e, tac h ycar di a, t hr o m b oe m b olis m, 
t hr o m b o p hle bitis, vasc ulitis 
Der mat ol o gic  
Ac ne, ac neif or m er u pti o ns, aller gic der m atitis, al o pecia, a n gi o e de ma, a n gi o ne ur otic e de m a, 
atr o p h y a n d t hi n ni n g of s ki n, dr y scal y s ki n, ecc h y m oses a n d petec hi ae ( br uisi n g), er yt h e ma, 
facial e d e ma, hirs utis m, i m paire d w o u n d heali n g, i ncreas e d s weati n g, Kar p osi’s sarc o ma, l u p us 
er yt h e mat os us -li ke lesi o ns, peri neal irritati o n, p ur p ura, ras h, stri ae, s u bc uta ne o us fat atr o p h y, 
s u p pressi o n of reacti o ns t o s ki n tests, striae, tela n giectasis, t hi n fra gile s ki n, t hi n ni n g scal p hair, 
urticaria  
E n d ocri ne  
A dre nal i ns uffi cie nc y- gr eatest p ote ntial ca us e d b y hi g h p ote nc y gl u c oc ortic oi ds wit h l o n g 
d urati o n of acti o n ( ass oci ate d s y m pt o ms i ncl u de; art hral gias, b uffal o h u m p, dizzi ness, life-
t hreate ni n g h y p ote nsi o n, na usea, se v ere tire d ness or wea k n ess), a me n orr hea, p ost me n o pa usal 
blee di n g or ot her me nstr ual irre g ularities, d ecreas e d car b o h y drate a n d gl uc ose t olera nce, 
de vel o p me nt of c us hi n g oi d state, dia betes mellit us ( ne w o nset or m a nifestati o ns of late nt), 
gl yc os uri a, h y per gl yce mia, h y p ertric h osis, h y pert h yr oi dis m, h y p ot h yr oi dis m, i ncrease d 
re q uire me nts f or i ns uli n or oral h y p o gl yce mic a ge nts i n dia betics, li pi ds a b n or mal, m o o n face, 
ne gati ve nitr o ge n bal a nce ca use d b y pr otei n cata b olis m, sec o n dar y a dre n oc ortical a n d pit uitar y 
u nres p o nsi ve ness ( p artic ularl y i n ti mes of stress, as i n tra u ma, s ur ger y or ill ness) 
Fl ui d a n d Electr ol yte Dist ur ba nces  
C o n gesti ve heart f ail ure i n s usce pti ble p atie nts, fl ui d rete nti o n, h y p o k ale mia, h y p o k ale mic 
al kal osis, meta b olic al kal osis, h y p ote nsi o n or s h oc k-li ke reacti o n, p otassi u m l oss, s o di u m 
rete nti o n wit h res ulti n g e de ma 
Gastr oi ntesti nal  
A b d o mi nal diste nti o n, a b d o mi nal pai n, a n ore xia w hic h ma y res ult i n wei g ht l oss, c o nsti pati o n, 
diarr hea, ele v ati o n i n ser u m li ver e nz y me le v els ( us uall y re versi ble u p o n disc o nti n uati o n), 
gastri c irritati o n, he pat o me gal y, i n crease d a p p etite a n d wei g ht gai n, n a usea, or o p har y n geal 
ca n di diasis, pa ncr eatitis, pe ptic ulcer wit h p ossi ble perf orati o n a n d he m orr ha ge, perf orati o n of 
t he s mall a n d lar ge i ntesti ne ( partic ularl y i n p atie nts wit h i nfla m mat or y b o wel disease), 
ulcerati ve es o p ha gitis, v o miti n g  
He mat ol o gi c  
A ne mia, ne utr o pe ni a (i ncl u di n g fe brile ne utr o pe ni a) 


C he m o Ce ntr y x, I n c.                            C O N FI D E N TI A L                                Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8                                                                                                                    A me n d me nt 4. 0 
                                                                   Pa ge 1 1 2 of 1 1 3                               1 8 Se pte m ber 2 0 1 5 
Meta b olic  
Ne g ati ve nitr o ge n b ala nce d ue t o pr otei n cata b olis m 
M usc ul os keletal  
Art hral gias, as e ptic necr osis of fe m oral a n d h u meral hea ds, i ncreas e ris k of fract ure, l oss of 
m uscle mass, m uscle wea k ness, m yal gias, oste o p e nia, oste o p or osis, pat h ol o gic fract ure of l o n g 
b o nes, ster oi d m y o p at h y, te n d o n r u pt ure ( p artic ularl y of t h e Ac hilles te n d o n), verte bral 
c o m pressi o n fr act ures  
Ne ur ol o gical/ Ps yc hiatric  
A m nesia, a n xiet y, be ni g n i ntracra ni al h y p erte nsi o n, c o n v ulsi o ns, deliri u m, de me ntia 
(c har acterize d b y d eficits i n me m or y rete nti o n, atte nti o n, c o nce ntrati o n, me ntal s pee d a n d 
efficie n c y, a n d o cc u pati o nal perf or m a nce), de pressi o n, dizzi ness, E E G a b n or malities, e m oti o nal 
i nsta bilit y a n d irrita bilit y, e u p h oria, hall uci nati o ns, hea dac he, i m paire d c o g niti o n, i nci de nce of 
se vere ps yc hiatric s y m pt o ms, i ncrease d i ntracra nial press ure wit h pa pille de ma ( pse u d ot u m or 
cere bri) us uall y f oll o wi n g disc o nti n uati o n of treat me nt, i ncreas e d m ot or acti vit y, i ns o m nia, 
isc he mic ne ur o p at h y, l o n g-ter m me m or y l oss, ma nia, m o o d s wi n gs, ne uritis, ne ur o pat h y, 
parest hesia, p ers o nalit y c ha n ges, ps yc hiatric dis or ders i ncl u di n g ster oi d ps yc h os es or 
a g gra vati o n of pre- e xisti n g ps yc hi atric c o n diti o ns, restless ness, sc hiz o p hre nia, ver bal me m or y 
l oss, verti g o, wit h dra w n be ha vi or 
O p ht hal mic  
Bl urre d visi o n, catar acts (i ncl u di n g p osteri or s u bca ps ular cataracts), ce ntral ser o us 
c h ori oreti n o pat h y, esta blis h me nt of sec o n dar y bact erial, f u n gal a n d viral i nfecti o ns, 
e x o p ht hal m os, gla uc o ma, i ncreas e d i ntra oc ular press ure, o ptic ner ve da m a ge, p a pille de ma 
Ot her  
A b n or mal fat de p osits, a g gr a vati o n/ mas ki n g of i nfecti o ns, decreas e d resist a nce t o i nfecti o n, 
hicc u ps, i m m u n os u p presi o n, i ncreas e d or decrease d m otilit y a n d n u m ber of s per mat oz oa, 
malaise, i ns o m nia, m o o n face, p yr e xia 


C he m o Ce ntr y x, I n c.                            C O N FI D E N TI A L                                Pr ot oc ol C L 0 0 2 _ 1 6 8 
C C X 1 6 8                                                                                                                    A me n d me nt 4. 0 
                                                                   Pa ge 1 1 3 of 1 1 3                               1 8 Se pte m ber 2 0 1 5 
1 1. 1 0.  
A d verse E ve nts Re p orte d wit h Rit u xi m a b Use  
T he f oll o wi n g a d vers e e v e nts were re p ort e d at a n i nci de nce of ≥ 1 0 % i n s u bjects recei vi n g 
rit u xi ma b i n patie nts wit h G P A or M P A: na usea, diarr hea, hea d ac h e, m uscle s pas ms, a ne mia, 
peri p her al e de ma, i ns o m nia, art hral gia, c o u g h, fati g ue, i ncreas e d A L T, h y p erte nsi o n, e pista xis, 
d ys p nea, le u k o pe nia, a n d ras h. I nf usi o n reacti o ns i ncl u de d c yt o ki ne releas e s y n dr o me, fl us hi n g, 
t hr oat irritati o n, a n d tre m or. I nfecti o ns i ncl u di n g u p per res pirat or y tract i nfecti o ns, uri nar y tract 
i nfecti o ns, a n d her p es z oster ha ve b ee n re porte d. H y p o ga m ma gl o b uli ne mia was als o re p orte d.  
1 1. 1 1.  
A d verse E ve nts Re p orte d wit h Az at hi o pri ne Use  
Azat hi o pri ne use has bee n ass ociate d wit h a n i ncrease d ris k of cert ai n t y p es of ca ncers i ncl u di n g 
s ki n ca ncer a n d l y m p h o ma. Azat hi o pri ne ma y als o ca use seri o us (rarel y fatal) bl o o d dis or ders 
( decr ease d b o n e marr o w f u ncti o n lea di n g t o a ne mia, l o w W B C a n d platelet c o u nt). Its use is als o 
ass ociate d wit h i ncr ease d ris k of i nfecti o n.  

SAP_001.pdf: 
 
 
 
 
S T A TI S TI C A L  A N A L Y SI S P L A N  
 
 
A Ra n d o mize d, D o u ble- Bli n d, Place b o- C o ntr olle d, P hase 2 St u d y t o E val uate t he 
Safet y a n d Efficac y of C C X 1 6 8 i n S u bjects wit h A nti- Ne utr o p hil C yt o plas mic 
A nti b o d y ( A N C A)- Ass ociate d Vasc ulitis o n Bac k gr o u n d C ycl o p h os p ha mi de or 
Rit u xi ma b Treat me nt 
 
 
Pr ot oc ol N u m ber : C L 0 0 2 _ 1 6 8  
I n vesti g ati o n al Pro d uct : C o m ple me nt 5a Rece pt or A nta g o nist C C X 1 6 8  
 
 
 
 
S p o ns or: 
C he m o Ce ntr y x, I n c. 
 
 
 
 
 
 Versi o n N u m ber 2. 0 
D ate: N o ve m ber 3, 2 0 1 5 
 
 
 
 
 
C O N FI D E N TI A L 
T he i nf or mati o n c o ntai ne d her ei n is t he pr o pert y of t he S p o ns or a n d ma y n ot be re pr o d uce d, p u blis he d, or discl ose d 
t o ot hers wit h o ut t he writte n a ut h orizati o n fr o m t he S p o ns or. 
 
P P D




C he m oce ntr y x, I nc.  
Pr ot oc ol C L 0 0 2 1 6 8 
 
Statistical A nal ysis Pla n- Versi o n 2. 0 
 
P a ge 3 of 2 0 8  
R E VI SI O N HI S T O R Y 
Versi o n  
Date  
Descri pti o n of C ha n ges  
1. 0  
J ul y 1 7, 2 0 1 3 
Ori gi nal S A P  
2. 0  
N o ve m ber  
3 , 
2 0 1 5  
Re visi o ns f or a nal ysis of data f or Ste p 3 c orres p o n di n g 
t o Pr ot oc ol A me n d me nt 4 . 0 
 
 
 
 
 
 


C he m oce ntr y x, I nc.  
Pr ot oc ol C L 0 0 2 1 6 8 
 
Statistical A nal ysis Pla n- Versi o n 2. 0 
 
P a ge 4 of 2 0 8  
T A B L E O F C O N T E N T S 
1  
I ntr o d u cti o n ............................................................................................................................. 5 
2  
Trial O bjecti ves ....................................................................................................................... 6  
3  
Statistical Met h o d ol o g y ........................................................................................................... 7  
3. 1  
A nal ysis  P o p ulati o ns ........................................................................................................ 7 
3. 2  
A nal ysis O v er vie w ........................................................................................................... 7  
3. 3  
S u m mar y of De m o gra p hi c a n d Baseli ne C haract eristics ................................................. 7  
3. 4  
Safet y A nal yses ................................................................................................................ 7  
3. 5  
Efficac y A nal yses ............................................................................................................. 9  
3. 5. 1  
Pri mar y Efficac y A nal ysis ...................................................................................... 1 0  
3. 5. 2  
Ot her Effi c ac y A nal yses ......................................................................................... 1 1  
3. 6  
P har mac o ki netic A n al ys es ............................................................................................. 1 3  
4  
Sa m ple Siz e J ustificati o n ....................................................................................................... 1 3  
5  
I nteri m A nalysis .................................................................................................................... 1 4  
6  
Ge nera l I nf or m ati o n Re gar di n g Data A nal yses .................................................................... 1 4 
7  
Ta ble S hells ................................ ........................................................................................... 1 5 
8  
Listi n g S hells ....................................................................................................................... 1 8 6  


C he m oce ntr y x, I nc.  
Pr ot oc ol C L 0 0 2 1 6 8 
 
Statistical A nal ysis Pla n- Versi o n 2. 0 
P a ge 5 of 2 0 8  
1  
I ntr o d ucti o n 
T his d oc u me nt pr o vi des a descri pti o n of t he statistical 
met h o ds a n d pr oce d ures t o be 
i m ple me nte d f or t he a nal ysis of dat a fr o m all 3 ste ps of C he m o Ce ntr y x, I n c. Pr ot oc ol 
C L 0 0 2 _ 1 6 8.  A n a nal ysis was i nitiall y perf or me d after s u bjects e nr olle d i n Ste ps 1 a n d 2 of t he 
st u d y c o m plete d t he 1 6 8- da y st u d y p eri o d.  Base d o n t he res ults of t hat a nal ysis t he pr ot oc ol was 
a me n de d pri or t o e nr oll me nt of s u bjects i nt o Ste p 3.  A n o ver vie w of eac h of t h e 3 ste ps is 
pr o vi de d bel o w: 
S T E P 1 
T wel ve s u bjects wit h ne wl y dia g n ose d or rela ps e d A N C A- ass o ciate d re nal vasc ulitis ( A A R V) 
were tar gete d f or ra n d o miz ati o n t o C C X 1 6 8 or place b o i n a 2: 1 rati o.  Ra n d o mizati o n was 
stratifie d b y diseas e stat us ( ne wl y dia g n ose d or rela pse d).  A t w o-t hir ds re d uce d d ose of oral 
c ortic oster oi ds 
was gi v e n t o s u bjects ra n d o mize d t o C C X 1 6 8 a n d a f ull d ose of oral 
c ortic oster oi ds t o s u bjects ra n d o mize d t o place b o. All 1 2 s u bjects recei ve d I V c ycl o p h os p ha mi de 
treat me nt, 
w hich is 
part 
of sta n dar d t hera p y f or 
A A R V. If 
necessar y, res c ue I V 
met h yl pre d nis ol o ne or oral resc u e ster oi ds after Da y 1 were gi ve n t o s u bjects wit h w orse ni n g 
disease. 
S T E P 2 
T wel ve s u bjects wit h ne wl y dia g n ose d or rela ps e d A A R V were tar gete d f or ra n d o miz ati o n t o 
C C X 1 6 8 or place b o i n a 2: 1 rati o. Oral c ortic oster oi ds were n ot gi ve n t o s u bjects ra n d o mize d t o 
C C X 1 6 8 b ut a f ull d ose of oral c ortic oster oi ds were gi ve n t o s u bjects ra n d o mize d t o place b o. All 
s u bjects recei ve d I V c ycl o p h os p ha mi de tr eat me nt. If 
n ecessar y, resc u e I V 
or 
oral 
met h yl pre d nis ol o ne c o ul d be gi v e n t o s u bjects wit h w orse ni n g disease.  
S T E P 3 
A p pr o xi matel y 3 6 s u bjects wit h A N C A-ass ociate d vasc ulitis ( A A V), wit h or wit h o ut re nal 
disease i n v ol ve me nt, w ere pla n ne d t o be ra n d o mize d i n a 1: 1: 1 r ati o t o o ne of t he f oll o wi n g t hr ee 
gr o u ps: 
Gr o u p A: C C X 1 6 8 pl us c ycl o p h os p h a mi de/rit u xi ma b pl us n o oral c ortic oster oi ds; 
Gr o u p B : Place b o pl us c ycl o p h os p ha mi de/rit u xi ma b pl us a f ull starti n g d ose of oral 
c ortic oster oi ds; 
Gr o u p C : C C X 1 6 8 pl us c ycl o p h os p ha mi de/rit u xi ma b pl us a t w o -t hir ds re d uce d starti n g 
d ose of oral c ortic oster oi ds.  
Ra n d o mizati o n was stratifie d base d o n t he f oll o wi n g t hree varia bles: 
 Disease stat us ( n e wl y dia g n ose d A A V or rela pse d A A V); 
 A N C A p ositi vit y ( M P O or P R 3); a n d 
 Sta n dar d of care tr eat me nt (c ycl o p h os p ha mi de or rit u xi ma b ). 
If necessar y, resc u e I V met h yl pre d nis ol o ne or oral res c ue ster oi ds c o ul d be gi ve n t o s u bjects 
wit h w orse ni n g dis ease. 


C he m oce ntr y x, I nc.  
Pr ot oc ol C L 0 0 2 1 6 8 
 
Statistical A nal ysis Pla n- Versi o n 2. 0 
P a ge 6 of 2 0 8  
 
F or eac h ste p of t he st u d y, s u bjects partici pate d i n a n 8 4- da y d osi n g peri o d a n d a n 8 4- da y 
f oll o w- u p peri o d.  All s u bjects ha d sc h e d ule d st u d y visits at scree ni n g a n d o n Da ys 1, 2, 8, 1 5, 
2 2, 2 9, 4 3, 5 7, 7 1, 8 5, 9 9, 1 1 3, 1 4 1, a n d 1 6 9. 
F or a d diti o nal details r e ga r di n g t h e st u d y d esi g n, please refer t o t he pr ot oc ol. 
A A V sta n dar d t hera p y i ncl u des c ycl o p h os p h a mi de a n d oral c ortic oster oi ds, ta pere d o ver a peri o d 
of ti me. Se vere disease warra nts a d diti o n of I V c ortic oster oi ds a n d/ or pl as ma e x c ha n ge. Base d 
o n st u dies i n dicati n g t he i n v ol ve me nt of C 5a a n d C 5a R i n A A V, C C X 1 6 8 has t he p ote ntial t o be 
a c ortic oster oi d s pari n g or c ortic oster oi d re place me nt t hera p y f or t his disease. T h e ai m of t his 
trial is t o o pti mize t he treat me nt t o i n d uce re missi o n f or patie nts wit h A A V . T he i nte nt is t o 
re d uce t he t o xicit y of i n d ucti o n t hera p y b y re d uci n g t he o verall e x p os ure t o or eli mi nati n g 
e ntirel y t h e use of s yste mic c ortic oster oi ds d uri n g t he i n d ucti o n peri o d wit h a n i n hi bit or of t he 
c o m ple me nt C 5a rece pt or pl us c ycl o p h os p ha mi de or rit u xi ma b. 
T his a nal ysis  pla n was writte n after fi nalizati o n of pr ot oc ol a me n d me nt 4.  A n y de viati o ns fr o m 
t his a nal ysis pl a n will be s u bsta ntiate d b y s o u n d statistical rati o nale a n d d oc u me nte d i n t he 
I nte grate d Cli nical a n d Statistical Re p ort. 
2  
Tri al O bjecti ves 
As descri be d i n A me n d me nt 4, t he pri mar y safet y o bjecti ve of t his st u d y is t o e val uate t he safet y 
a n d t olera bilit y of C C X 1 6 8 i n s u bjects wit h A A V o n bac k gr o u n d c ycl o p h os p h a mi de or 
rit u xi ma b treat me nt.  T he pri mar y effi cac y o bjecti ve is t o e val uate t he efficac y of C C X 1 6 8 base d 
o n t he Bir mi n g ha m Va s c ulitis Acti vit y Sc ore ( B V A S) versi o n 3. 
 
T he sec o n d ar y o bjecti ves of t his st u d y i ncl u de: 
1.  E val uati o n of t he effic ac y of C C X 1 6 8 c o m pare d t o sta n dar d of care ( S O C) base d o n c h a n ges 
i n re nal disease acti vit y p ara meters: 
 
e G F R ( M D R D s er u m creati ni ne e q uati o n); 
 
He mat uria (c e ntr al la b orat or y micr osc o pic c o u nt of uri nar y R B Cs); a n d 
 
Al b u mi n uria (first m or ni n g uri nar y al b u mi n:creati ni ne rati o); 
2.  Assess me nt 
of c ha n ges i n re nal i nfla m mat or y acti vit y b ase d o n uri nar y 
m o n oc yte 
c he m oattracta nt pr otei n- 1 ( M C P- 1):creati ni ne rati o a n d ser u m C-reacti ve pr otei n ( C R P) 
c o nce ntr ati o n wit h C C X 1 6 8 c o m pare d t o S O C; 
3.  Assess me nt of t he feasi bilit y of re d u ci n g or eli mi nati n g t he use of c ortic oster oi ds i n t he 
treat me nt of s u bj ects wit h A N C A-ass ociate d vas c ulitis ( A A V) wit h o ut t he nee d f or resc u e 
c ortic oster oi d meas ures wit h C C X 1 6 8 c o m pare d t o S O C; 
4.  Assess me nt of h ealt h-relate d q ualit y- of-life c ha n ges base d o n S h ort F or m- 3 6 versi o n 2 ( S F-
3 6 v 2) a n d E ur o Q O L- 5 D- 5 L ( E Q- 5 D- 5 L) wit h C C X 1 6 8 c o m pare d t o S O C; 
5.  Assess me nt of c ha n ges i n Vasc ulitis Da ma ge I n d e x ( V DI) wit h C C X 1 6 8 c o m pare d t o S O C; 
6.  Assess me nt of c ha n ges i n A N C A (a nti- P R 3 a n d a nti- M P O) wit h C C X 1 6 8 c o m pare d t o S O C; 


C he m oce ntr y x, I nc.  
Pr ot oc ol C L 0 0 2 1 6 8 
 
Statistical A nal ysis Pla n- Versi o n 2. 0 
P a ge 7 of 2 0 8  
7.  Assess me nt of c ha n ges i n p har mac o d y na mics m ar kers i n plas ma a n d uri ne wit h C C X 1 6 8 
c o m pare d t o S O C;  a n d 
8.  E val uati o n of t he p har m ac o ki netic pr ofile of C C X 1 6 8 i n s u bjects wit h A A V. 
3  
St atistic al Me t h o d ol o g y 
3. 1  
A n al ysis P o p ul ati o ns  
F or t h e p ur p os es of data a nal ysis, t he i nte nt- t o-tr eat (I T T) P o p ulati o n will i ncl u de all s u bj ects 
w h o are ra n d o mize d, ha ve recei ve d at least o ne d ose of st u d y dr u g, a n d ha ve at least o ne p ost 
baseli ne o n treat me nt B V A S assess me nt. T he s afet y p o p ulati o n will i ncl u de all s u bjects w h o are 
ra n d o mize d a n d ha ve recei ve d at le ast o ne d ose of st u d y dr u g. A per pr ot oc ol ( P P) p o p ulati o n 
ma y als o be defi n e d if t here are maj or pr ot oc ol de viati o ns t hat c o ul d affect st u d y o utc o me.   
3. 2  
A n al ysis O ver vie w  
Data will  be s u m marize d descri pti vel y b y treat me nt gr o u p, st e p of t he st u d y, a n d o v erall.  F or 
c o nti n u o us varia bles, s u m mar y statistics will i ncl u de t he s a m ple siz e, m ea n, me di a n, sta n d ar d 
de viati o n ( S D), sta n dar d err or of t he mea n ( S E M), mi ni m u m, a n d ma xi m u m.  C o nti n u o us 
varia bles wit h s ke we d distri b uti o ns will be l o g-tr a nsf or me d f or a n al ysis i ncl u di n g uri n ar y A C R, 
uri nar y R B C c o u nt, uri nar y 
M C P- 1:creati ni ne rati o, a n d hs C R P. Fre q ue nc y c o u nts a n d 
perce nt a ges will be prese nte d f or cate g orical varia bles. All data will be dis pla ye d i n data listi n gs 
w hic h will be i ncl u de d as part of a n a p pe n di x t o t he Cli nical St u d y R e p ort. 
I n a nal ysis ta bles a n d listi n gs, t he t hr ee tr eat me nt gr o u ps will be referre d t o as ‘ Pla ce b o + F ull 
D ose Pre d nis o ne’, ‘ C C X 1 6 8 + L o w D ose Pre d nis o ne’, a n d ‘ C C X 1 6 8 + N o Pre d nis o ne’.  F or 
s u m maries a n d a nal yses f or w hic h all s u bjects ra n d o mize d t o C C X 1 6 8 treat me nt are p o ole d, t he 
treat me nt gr o u p will be referre d t o as ‘ All C C X 1 6 8’.  N ote t hat t he ‘ C C X 1 6 8 + L o w D ose 
Pre d nis o ne’ gr o u p i n cl u des all Ste p 1 s u bjects ra n d o mize d t o C C X 1 6 8 a n d all Ste p 3 s u bjects 
ra n d o mize d t o Gr o u p C.  T he ‘ C C X 1 6 8 + N o Pre d nis o ne’ gr o u p i ncl u d es all Ste p 2 s u bjects 
ra n d o mize d t o C C X 1 6 8 a n d all Ste p 3 s u bjects ra n d o mize d t o Gr o u p A. 
3. 3  
S u m m ar y of De m o g r a p hic a n d B aseli ne C h ar acteristi cs  
All s u bject baseli ne c h aract eristics a n d de m o gra p hic data (a ge, se x, race, et h nicit y, wei g ht, 
hei g ht, b o d y mass i n de x, s m o ki n g stat us, E C G, T B scree n res ults, vir al test res ults, A N C A,  
re nal vas c ulitis disease d urati o n (fr o m ti me of first i n d ucti o n of treat me nt), B V A S, V DI, 
S F 3 6 v 2, E Q- 5 D - 5 L, hs C R P, e G F R, h e mat uria, uri ne R B C casts, pr otei n uria ( A C R), gl o mer ular 
hist o pat h ol o g y (if bi o ps y was ta ke n), uri nar y 
M C P- 1:creati ni ne rati o, p h ysi cal e x a mi nati o n 
a b n or malities, 
me dical hist or y, pre vi o us ( wit hi n 6 
m o nt hs of scree ni n g) a n d c o n c o mita nt 
me dicati o ns (i ncl u di n g vasc ulitis me dicati o n use) at st u d y e ntr y will b e liste d b y treat me nt 
gr o u p, st u d y ce nter, a n d s u bject n u m ber, a n d will als o be s u m marize d b y treat me nt gr o u p, ste p 
of t he st u d y, a n d o verall.  
3. 4  
S afet y A n al yses 
Safet y assess me nts i ncl u de a d vers e e ve nts, p h ysical e x a mi nati o n a b n or malities, vital si g ns, 
cli nical la b orat or y tests (i ncl u di n g bl o o d c he mistr y, he mat ol o g y, a n d uri nal ysis), a n d E C Gs. 


C he m oce ntr y x, I nc.  
Pr ot oc ol C L 0 0 2 1 6 8 
 
Statistical A nal ysis Pla n- Versi o n 2. 0 
P a ge 8 of 2 0 8  
Safet y a nal yses will be perf or me d o n t he Safet y P o p ulati o n. Data will be s u m marize d f or eac h 
ste p a n d o verall (i ncl u di n g s u bjects fr o m all 3 ste ps).  F or t h e s u m maries wit hi n eac h ste p, data 
will be prese nte d f or eac h ra n d o mize d treat me nt gr o u p.  F or t he o verall s u m maries, data will 
prese nte d f or t he pla ce b o gr o u p, t h e C C X 1 6 8 + l o w d ose pre d nis o ne gr o u p, t he C C X 1 6 8 + n o 
pre d nis o ne gr o u p,  a n d a p o ole d s u m mar y t hat i ncl u des all C C X 1 6 8 s u bjects. I n ge neral, se p arate 
s u m maries will be pre p are d f or safet y e ve nts o cc urri n g d uri n g t h e 8 4- d a y treat me nt p eri o d a n d 
t he 1 6 8- da y st u d y peri o d.  N o i nfere ntial a nal yses will be perf or me d o n s afet y d ata. 
A n a d verse e v e nt will be c o nsi dere d as “ pre-treat me nt” if t he start date/ti me of t he e ve nt is pri or 
t o t he ti me of a d mi nistrati o n of t he first d ose of st u d y me dicati o n. All ot her a d verse e ve nts will 
be c o nsi dere d “treat me nt-e mer ge nt ” ( T E A E).  S y m pt o ms or si g ns of vasc ulitis 
will be 
c o nsi dere d a d vers e e ve nts if t hese i ncrease i n se v erit y or fre q u e nc y w hile a s u bject is o n st u d y. 
A n o ver vie w of tr eat me nt-e mer ge nt a d verse e v e nts will be pre pare d t hat prese nts all T E A Es, 
seri o us a d verse e v e nts ( S A Es), T E A Es lea di n g t o disc o nti n uati o n, e ve nts relate d t o st u d y 
me dicati o n, c ortic oster oi ds, c ycl o p h os p h a mi de, azat hi o pri ne, or rit u xi ma b, a n d T E A Es b y 
ma xi m u m se verit y. 
A d verse e v e nts will be c o de d usi n g Me d D R A a n d T E A Es will be s u m marize d b y s yste m or ga n 
class a n d preferre d ter m.  Si milar s u m maries will be pre pare d f or T E A Es relate d t o st u d y 
me dicati o n, c ortic oster oi ds, c ycl o p h os p ha mi de, azat hi o pri ne, or rit u xi ma b, T E A Es b y ma xi m u m 
se verit y, S A Es, a n d T E A Es lea di n g t o disc o nti n uati o n.  A d verse e ve nts will be liste d b y st e p a n d 
treat me nt gr o u p, i ncl u di n g all a vaila ble i nf or mati o n of i nterest s uc h as o nset a n d res ol uti o n 
dates, st u d y da y of o ns et relati ve t o first d osi n g d a y, s e verit y, seri o us ness, ca usal relati o ns hi p t o 
st u d y me dicati o n a n d c ortic oster oi d use, acti o n ta ke n, a n d o utc o me.   
La b orat or y para meter res u lts a n d c ha n ges fr o m baseli ne will s u m marize d b y visit. S hift ta bles 
fr o m baseli ne t o s u bs e q ue nt st u d y visits will als o be ge n erate d.  N ota ble a b n or malities will be 
liste d b y ste p, treat me nt gr o u p a n d s u bject n u m ber, a n d will be s u m marize d b y treat me nt gr o u p. 
La b orat or y v al ues o utsi de t he refere nce ra n ges will be fla g ge d i n t he listi n gs.  
Vital si g n res ults a n d c h a n ges fr o m b aseli ne will s u m marize d b y visit.  P h ysical e x a mi nati o n 
a b n or malities will be s u m mariz e d b y visit a n d b o d y s yste m.  E C G a b n or malities will be liste d. 
T he s u bject i nci de nce of effects p ossi bl y ass oci ate d wit h gl u c oc ortic oi d use i ncl u di n g seri o us 
i nfecti o ns, ne w- o nset dia betes 
mellit us/ h y p er gl yce mia, b o ne fract ure, pe ptic ulcer disease, 
cataracts, ne w o nset/ w orse ni n g h y pert e nsi o n, wei g ht gai n m ore t ha n 1 0 k g, a n d ps yc hiatric 
dis or ders will be s u m marize d b y tr eat me nt gr o u p f or t he 8 4- da y treat me nt peri o d a n d t he 1 6 8-
da y st u d y p eri o d. T hese effects will be i de ntifie d as f oll o ws: 
 Seri o us i nfecti o ns: All S A Es i n t he S yste m Or ga n Class I nfecti o ns a n d I nfestati o ns 
 Ne w- o nset dia b etes mellit us/ h y per gl yce mia: All T E A Es of h y per gl yce mia, dia betes, 
i ncreas e d bl o o d gl u c ose, pl us all patie nts wit h a fasti n g bl o o d gl uc os e le vel p ost baseli ne 
t hat is a b o ve t he u p per li mit of n or mal o n at least t w o c o nsec uti ve st u d y visits. 
 B o ne fract ure: All T E A Es i n dicati n g  l o n g b o ne or verte bral fract ures 
 Pe ptic ulcer disease: All T E A Es i n dicati n g u p per gastr oi ntesti nal ulcerati o n, er osi o n, or 
blee di n g 
 Cataracts: All T E A Es of cataract 


C he m oce ntr y x, I nc.  
Pr ot oc ol C L 0 0 2 1 6 8 
 
Statistical A nal ysis Pla n- Versi o n 2. 0 
P a ge 9 of 2 0 8  
 Ne w o nset/ w orse ni n g h y perte nsi o n: All T E A Es of h y perte nsi o n, w orse ni n g h y pert e nsi o n, 
or hi g h bl o o d pr ess ure, pl us all patie nts wit h a s yst olic bl o o d press ure i ncrease of at least 
2 0 m m H g fr o m b aseli ne, a n d > 1 4 0 m m H g ( s yst olic), or diast olic bl o o d press ure 
i ncreas e of at least 1 0 m m H g fr o m b aseli ne, a n d > 9 0 m m H g ( diast olic), t hat is prese nt 
o n at least t w o c o nsec uti ve st u d y visits. 
 Wei g ht gai n m ore t ha n 1 0 k g: C ha n ge fr o m baseli ne i n wei g ht of > 1 0 k g. 
 Ps yc hiatric dis or ders: All T E A Es of ps yc h osis, a n xiet y, a m nesia, c o n v ulsi o ns, deliri u m, 
de me ntia, de pressi o n, ma nia, e m oti o nal i nsta bilit y, irrita bilit y, e u p h ori a, hall uci nati o ns, 
i m paire d c o g niti o n, i ncrease d m ot or acti vit y, i ns o m nia, me m or y l oss, ma nia, m o o d 
s wi n gs, 
ne uritis, 
ne ur o pat h y, 
parest hesia, 
pers o nalit y 
c h a n ges, 
restless ness, 
sc hiz o p hre nia, verti g o, or wit h dra wal be h a vi or. 
T he s u bject i nci de nce of i nfecti o ns, seri o us i nfecti o ns, se vere i nfecti o ns (i.e., Gra d e 3), a n d 
i nfecti o ns lea di n g t o s u bject wit h dra wal fr o m t he st u d y will be s u m mariz e d b y treat me nt gr o u p 
f or t he 8 4- da y treat me nt peri o d a n d t he 1 6 8- d a y st u d y peri o d.  
3. 5  
Effic ac y A n al yses 
T he pri mar y  effi cac y e n d p oi nt is t he pr o p orti o n of s u bjects ac hie vi n g disease res p o nse at Da y 8 5 
defi ne d as B V A S perce nt re d ucti o n fr o m baseli ne of at least 5 0 % pl us n o w orse ni n g i n a n y b o d y 
s yste m c o m p o ne nt.  
Ot her efficac y e n d p oi nts i ncl u de: 
1.  I n p atie nts wit h he mat uri a a n d al b u mi n uria at b aseli ne, t he pr o p orti o n of s u bjects ac hie vi n g 
re nal r es p o nse at D a y 8 5; re nal r es p o nse is defi ne d as a n i m pr o ve me nt i n para meters of re n al 
vasc ulitis: 
a. a n i ncrease fr o m baseli ne t o Da y 8 5 i n e G F R ( M D R D ser u m creati ni ne e q u ati o n), pl us 
b.  a decreas e fr o m bas eli ne t o Da y 8 5 i n h e mat uria (ce ntral la b orat or y micr osc o pic c o u nt of 
uri nar y R B Cs), pl us 
c. a decreas e fr o m bas eli ne t o Da y 8 5 i n al b u mi n uria (first m or ni n g uri nar y 
al b u mi n:creati ni ne rati o). 
2.  Pr o p orti o n of s u bjects ac hie vi n g diseas e re missi o n at Da y 8 5 defi ne d as B V A S of 0 or 1 pl us 
n o w orse ni n g i n e G F R a n d uri nar y R B C c o u nt < 1 0/ h pf; 
3.  Perce nt c h a n ge fr o m bas eli ne t o Da y 8 5 i n B V A S; 
4.  C ha n ge  a n d perce nt c ha n ge fr o m baseli ne t o D a y 8 5 i n e G F R; 
5.  In s u bjects wit h baseli ne he mat uria > 5 R B Cs/ h pf, t he pr o p orti o n of s u bjects a n d ti me t o first 
ac hie vi n g uri nar y R B C c o u nt ≤ 5/ h pf at a n y ti me d uri n g t he 8 4- da y treat me nt peri o d; 
6.  I n s u bjects wit h baseli ne he mat uria ≥ 3 0 R B Cs/ h pf, t he pr o p orti o n of s u bjects a n d ti me t o 
first ac hie vi n g uri nar y R B C c o u nt < 3 0/ h pf at a n y ti me d uri n g t he 8 4- d a y treat me nt peri o d; 
7.  I n s u bjects wit h he mat uri a at baseli ne, t he perce nt c ha n ge fr o m baseli ne t o Da y 8 5 i n uri nar y 
R B C c o u nt;  
8.  I n s u bjects wit h al b u mi n uria at baseli ne, t h e p erce nt c ha n ge fr o m baseli ne t o Da y 8 5 i n 
uri nar y A C R;  


C he m oce ntr y x, I nc.  
Pr ot oc ol C L 0 0 2 1 6 8 
 
Statistical A nal ysis Pla n- Versi o n 2. 0 
P a ge 1 0  of 2 0 8 
9.  Perce nt c h a n ge fr o m bas eli ne t o Da y 8 5 i n uri nar y M C P- 1:creati ni ne rati o; 
1 0.  P r o p orti o n of s u bjects re q uiri n g resc ue I V or oral gl uc oc ortic oi d treat me nt; 
1 1.  C ha n ge fr o m bas eli ne t o Da y 8 5 i n t he Vas c ulitis D a ma ge I n d e x ( V DI); 
1 2.  C ha n ge  fr o m bas eli ne t o Da y 8 5 i n healt h-relate d q ualit y- of-life as me as ure d b y t h e S h ort 
F or m- 3 6 versi o n 2. 0 ( S F- 3 6 v 2) a n d E ur o Q O L- 5 D- 5 L ( E Q- 5 D- 5 L); 
Ot her e n d p oi nts i ncl u de:  
1.  T ota l c u m ulati ve st u d y-s u p plie d pre d nis o ne d ose a n d d urati o n of d osi n g d uri n g t he 8 4- da y 
treat me nt peri o d; 
2.  T otal c u m ulati ve s yste mi c c ortic oster oi d d os e (a n y use) a n d d urati o n of d osi n g d uri n g t he 
8 4- da y d osi n g  p eri o d; 
3.  T otal c u m ulati ve c ycl o p h os p ha mi de or rit u xi ma b d ose a n d d urati o n of d osi n g d uri n g t he 8 4-
da y d osi n g  p eri o d;  
4.  Perce nt c h a n ge fr o m bas eli ne i n hs C R P; 
5.  Perce nt c h a n ge fr o m bas eli ne i n A N C A (a n ti- P R 3 an d a nti- M P O) at D a y 8 5; 
6.  Pr o p orti o n of patie nts b ec o mi n g A N C A ne gati ve at Da y 8 5; a n d 
7.  C ha n ge  a n d perce nt c ha n ge fr o m baseli ne i n plas ma a n d uri ne bi o mar k ers. 
Ef ficac y anal ys es will be prese nte d i n t hree gr o u pi n gs:   
1.  Ste ps 1 t hr o u g h 3 c o m bi ne d 
2.  S te ps 1 a n d 2 c o m bi ne d a n d Ste p 3 
3.  Eac h ste p prese nte d se p aratel y 
N o f or mal testi n g will be use d t o c o m pare treat me nt gr o u ps wit hi n Ste p 1 a n d Ste p 2 d ue t o t he 
s mall sa m ple siz e. 
F or all efficac y e n d p oi nts, b aseli ne is defi ne d as t he last val ue pri or t o start of d osi n g wit h st u d y 
me dicati o n (t y pi call y t he Da y 1 pre- d ose val u e).  
3. 5. 1  
Pri m ar y Effic ac y  A n al ysis  
T he pr o p orti o n of s u bj ects ac hie vi n g disease res p o ns e ( defi ne d a b o v e) d u ri n g t he 8 4- d a y 
treat me nt peri o d will be calc ulate d t o c o m pare eac h C C X 1 6 8 gr o u p a gai nst t he place b o (sta n d ar d 
of care)  gr o u p. If t he D a y 8 5 res ult is missi n g, t he last p ost-ra n d o mizati o n res ult will be use d, 
u nless t he s u bject ha d w orse ni n g of A A V a n d re q uire d res c ue tr eat me nt. I n t he latter cas e t he 
s u bject w o ul d be c o nsi dere d a n o n-res p o n der. 
If t h e l o wer b o u n d of t h e 1-si de d 9 5 % c o nfi de n ce i nter val f or t he differe nce ( C C X 1 6 8 mi n us 
c o ntr ol gr o u p) is greater t ha n - 0. 2 0, t he res pecti ve C C X 1 6 8 gr o u p will be c o nsi dere d n ot i nferi or 
t o t he place b o gr o u p. If t he l o wer b o u n d is great er t ha n 0. 0, t h e res pecti ve C C X 1 6 8 gr o u p will be 
c o nsi dere d s u p eri or t o t h e place b o gr o u p i n ac hie vi n g t he dise ase res p o ns e.  F or t he p ur p os e of 
data prese ntati o n, t he 2-si de d 9 0 % c o nfi de nce i nt er vals will be dis pla ye d si nce t he l o wer b o u n d 
of t he 1-si de d 9 5 % c o nfi de nce i nter val is i de ntical t o t he l o wer b o u n d of t he 2-si de d 9 0 % 


C he m oce ntr y x, I nc.  
Pr ot oc ol C L 0 0 2 1 6 8 
 
Statistical A nal ysis Pla n- Versi o n 2. 0 
P a ge 1 1  of 2 0 8 
c o nfi de nce i nt er val. T he p- val ues fr o m t he h y p ot hesis tests of n o n-i nferi orit y ( H 1: p1- p2>- 0. 2) 
a n d s u peri orit y ( H 1: p1- p2> 0) will als o be dis pla ye d.  T he pri mar y a nal ysis will i ncl u de all 
s u bjects i n all 3 ste ps.  
Si milar a nal ys es will b e perf or me d t o c o m pare t h e All C C X 1 6 8 gr o u p t o t he place b o gr o u p.   I n 
a d diti o n, t he a nal yses will be re peate d f or all s u bjects i n Ste p 3, f or s u bjects i n Ste p 1 + Ste p 2 
c o m bi ne d, a n d f or Ste ps 1 a n d 2.  F or t hese a nal yses, c o nfi de nce i nter vals a n d p- val ues will n ot 
be dis pla ye d f or Ste p 1 a n d f or Ste p 2 d ue t o t he s mall sa m ple sizes. 
T hese a nal yses of disease res p o ns e will be r e p eate d f or t he 1 6 8- da y st u d y p eri o d.  F or t his 
a nal ysis, if t he D a y 1 6 9 res ult is missi n g, t he last res ult after Da y 8 5 will be use d, u nless t he 
s u bject ha d w orse ni n g of A A V a n d re q uir e d resc u e treat me nt. I n t he l atter case t he s u bject w o ul d 
be c o nsi dere d a n o n-res p o n der.   
S u b gr o u p a n a l ys es will als o be perf or m e d f or t he f oll o wi n g s u b gr o u ps: 
1.  S u bjects wit h re nal disease at baseli ne ( defi ne d as s u bjects wit h B V A S ite ms sc ore d 
i n t he re nal orga n s yst e m) 
2.  S u bjects wit h o ut re nal disease at baseli ne ( defi n e d as s u bjects wit h n o B V A S ite ms 
sc ore d i n t he re nal or ga n s yste m) 
3.  S u bjects recei vi n g c ycl o p h os p ha mi de bac k gr o u n d treat me nt 
4.  S u bjects rec ei vi n g rit u xi ma b bac k gr o u n d tr eat me nt 
5.  S u bjects wit h ne wl y dia g n ose d disease 
6.  S u bjects wit h re la pse d disease 
7.  S u bjects wit h M P O + disease 
8.  S u bjects wit h P R 3 + disease 
9.  S u bjects wit h gra n ul o mat osis wit h p ol ya n giitis ( We ge ner’s) 
1 0.  S u bjects wit h micr osc o pic p ol ya n giitis 
F or all s u b g r o u p a nal yses, c o nfi de nce i nter vals a n d p- val ues are c o nsi dere d descri pti ve. 
3. 5. 2  
Ot her Effic ac y A n al yses  
Cate g orical res p o nses will be a nal yze d usi n g t he sa me a p pr oac h as t he pri mar y effi cac y 
e n d p oi nt.  T his i ncl u des: 
 Re nal res p o nse i n s u bjects wit h he mat uria a n d al b u mi n uria at baseli ne at Da y 8 5 a n d at 
Da y 1 6 9; 
 B V A S diseas e re missi o n at Da y 8 5 a n d Da y 1 6 9.; 
Res ults f or s u bj ects w h o beca me A N C A ne gati ve ( P R 3 a n d M P O) at Da y 8 5 a n d Da y 1 6 9 will 
be s u m mariz e o nl y wit h n o i nfere ntial statistical a nal yses. 


C he m oce ntr y x, I nc.  
Pr ot oc ol C L 0 0 2 1 6 8 
 
Statistical A nal ysis Pla n- Versi o n 2. 0 
P a ge 1 2  of 2 0 8 
Q ua ntitati ve effic ac y varia bles will be s u m mariz e d at eac h visit as will c ha n ges a n d/ or perce nt 
c ha n ges fr o m b aseli ne.   Mi x e d m o dels f or re peat e d meas ures ( M M R M) will be use d t o c o m pare 
treat me nt gr o u ps d uri n g t he 8 4- da y treat me nt peri o d a n d t he 1 6 8- da y st u d y peri o d. F or a n al ys es 
of Ste ps 1 t hr o u g h 3 c o m bi ne d, t he m o dels will i ncl u de treat me nt gr o u p, visit, treat me nt- b y - visit 
i nteracti o n, A A V dise ase stat us ( ne w or rela pse d) a n d A N C A p ositi vit y ( M P O or P R 3) as fact ors 
a n d t he bas eli ne val ue as a c o variate.  F or a nal yses of data fr o m Ste p 3 o nl y, t he m o d els will 
i ncl u de fact ors f or treat me nt gr o u p, visit, treat me nt-b y - visit i nteracti o n, A A V disease stat us ( ne w 
or rela pse d), A N C A p ositi vit y ( M P O or P R 3), a n d sta n dar d of care treat me nt (rit u xi ma b or 
c ycl o p h os p ha mi de), a n d t he baseli ne val ue as a c o variate.  F or a nal yses of data fr o m Ste ps 1 a n d 
2 c o m bi ne d, t he  m o dels will i ncl u de fact ors f or treat me nt gr o u p, visit, treat me nt-b y - visit 
i nteracti o n, disease stat us, A N C A p ositi vit y, a n d t he baseli ne val ue as a c o variate. T h e o ut p ut 
fr o m t he M M R M a nal ysis will i ncl u de t he res ults at eac h visit as well as t he o verall res ults.  P-
val ues fr o m c o ntrasts c o m pari n g treat me nt gr o u ps will be prese nte d as will 9 5 % c o nfi de nce 
i nter vals f or treat me nt differe nces. 
A nal ysis of c o varia n ce ( A N C O V A) will als o be use d t o c o m pare treat me nt gr o u ps i n c ha n ge 
a n d/ or perce nt c h a n ge fr o m baseli ne at eac h visit, f or t he e n d of t he 8 4- da y treat me nt peri o d, a n d 
t he e n d of t he 8 4- da y st u d y peri o d.  F or a n al ys es of Ste ps 1 t hr o u g h 3 c o m bi ne d, t he m o dels will 
i ncl u de A A V disease stat us ( ne w or rela pse d) a n d A N C A p ositi vit y ( M P O or P R 3) as fact ors a n d 
t he baseli ne val ue as a c o variate.  F or a nal yses of data fr o m Ste p 3 o nl y, t he m o dels will i ncl u de 
fact ors f or A A V dise ase stat us ( ne w or rel a pse d), A N C A p ositi vit y ( M P O or P R 3), a n d sta n d ar d 
of care treat me nt (rit u xi ma b or c ycl o p h os p ha mi de) a n d t he baseli ne v al ue as a c o variate.  F or 
a nal ys es of data fr o m Ste ps 1 a n d 2 c o m bi ne d, t h e m o dels will i ncl u de fact ors f or dis ease stat us 
a n d A N C A p ositi vit y, a n d t he baseli ne val ue as a c o variate.  
T he varia bl es t o b e a nal yze d i n t his ma n ner i ncl u de: 
 B V A S perce nt c h a n ge fr o m baseli ne; 
 e G F R ( M D R D f or m ula) c ha n ge a n d perce nt c ha n ge fr o m baseli ne; 
 Uri nar y R B C c o u nt rati o a n d perce nt c ha n ge fr o m baseli ne i n s u bjects wit h he mat uria at 
baseli ne;  
 A C R rati o a n d perce nt c h a n ge fr o m b aseli ne i n s u bjects wit h al b u mi n uria at baseli ne; 
 Uri nar y  M C P- 1:creati ni ne rati o a n d perce nt c ha n ge fr o m baseli ne; 
 V DI c h a n ge a n d p erce nt c ha n ge fr o m baseli ne; 
 S F - 3 6 c han ge a n d perce nt c ha n ge fr o m baseli n e f or s u bjects i n Ste p 3 i ncl u di n g all 
d o mai ns a n d t he p h ysi c al c o m p o ne nt a n d me ntal h ealt h s u m maries; 
 E Q - 5 D- 5 L s c ores a n d V A S c ha n ge a n d p erce nt c ha n ge fr o m b aseli ne f or s u bjects i n Ste p 
3 i ncl u di n g  all d o mai ns a n d t he p h ysic al c o m p o ne nt a n d me ntal healt h s u m maries; 
 Ser u m hs C R P rati o a n d perce nt c ha n ge fr o m bas eli ne ;  
 A N C A (a nti- P R 3 a n d a nti- M P O) rati o a n d perce nt c ha n ge fr o m baseli ne; a n d 
 S er u m a n d uri ne bi o mar k ers c ha n ge a n d p erce nt c ha n ge fr o m b aseli ne. 
T he M M R M a nal yses will be re peate d f or t h e B V A S re nal s u bsc ore a n d n o n-re nal s u bsc ore f or 
t he I T T P o p ulati o n o nl y. 


C he m oce ntr y x, I nc.  
Pr ot oc ol C L 0 0 2 1 6 8 
 
Statistical A nal ysis Pla n- Versi o n 2. 0 
P a ge 1 3  of 2 0 8 
T he perce nta ge of s u bjects w h o ac hie ve uri nar y R B C < = 5 R B Cs/ h pf d uri n g t he 8 4- da y treat me nt 
peri o d will be s u m marize d.  T he p- val ues fr o m t he h y p ot hesis tests of n o differe n ce bet w ee n 
treat me nts ( H1: p1- p2≠0) will be dis pla ye d.   F or eac h treat me nt gr o u p, Ka pl a n- Meier esti mates of 
t he 2 5t h perce ntile, me di a n, a n d 7 5t h perce ntile d a ys t o first o cc urre nce will be cal c ulate d a n d 
treat me nt gr o u ps will be c o m pare d usi n g t he l o g-ra n k test.  T he a nal ysis will be re peate d f or t he 
1 6 8- da y st u d y p eri o d as well as f or eac h of t he f oll o wi n g s u b gr o u ps: 
1.  S u bjects wit h re nal disease at baseli ne ( defi ne d as s u bjects wit h B V A S ite ms sc ore d 
i n t he re nal orga n s yst e m) 
2.  S u bjects wit h o ut re nal disease at baseli ne ( defi n e d as s u bjects wit h n o B V A S ite ms 
sc ore d i n t he re nal or ga n s yste m) 
3.  S u bjects recei vi n g c ycl o p h os p ha mi de bac k gr o u n d treat me nt 
4.  S u bjects rec ei vi n g rit u xi ma b bac k gr o u n d tr eat me nt 
5.  S u bjects wit h ne wl y dia g n ose d disease 
6.  S u bjects wit h rela pse d disease 
7.  S u bjects wit h M P O + disea se 
8.  S u bjects wit h P R 3 + disease 
9.  S u bjects wit h gra n ul o mat osis wit h p ol ya n giitis ( We ge ner’s) 
1 0.  S u bjects wit h mi cr osc o pic p ol ya n giitis 
Si milar a nal ys es will b e prese nte d f or t he e n d p oi nt R B C < 3 0 R B C/ h pf a n d t he e n d p oi nt f or t he 
occ urre nce of res c ue I V or oral gl u c oc ortic oi d treat me nt.   F or t he a nal ysis or resc ue tr eat me nt, 
all s u bjects w h o recei ve resc ue bef ore Da y 9 2 ( Da y 8 5 + 7 d a y) will be c o nsi dere d a treat me nt 
fail ure. 
S u m maries of d ose a n d d urati o n of st u d y s u p plie d pre d nis o ne t a ke n d uri n g t he 8 4- da y treat me nt 
peri o d a n d t he 1 6 8- d a y st u d y p eri o d will be pr o vi d e d f or t he f ull st u d y p o p ulati o n a n d f or eac h of 
t he 1 0 s u b gr o u ps liste d a b o ve.  Si milar s u m maries 
will be pre pare d f or t otal s yste mic 
c ortic oster oi d tr eat me nt, c u m ulati ve c ycl o p h os p ha mi de treat me nt, a n d c u m ulati ve rit u xi ma b 
treat me nt ( Ste p 3 o nl y). 
T he mai n effic ac y a n al ysis will be i n t he I T T p o p ulati o n. Se nsiti vit y a nal ys es ma y als o be 
perf or me d e x cl u di n g s u bjects wit h maj or pr ot oc ol de viati o ns. 
3. 6  
P h ar m a c o ki netic A n al yses 
T he res ults fr o m p ha r mac o ki netic a nal yses will be re p ort e d i n a se parate re p ort. 
4  
S a m ple Size J ustific ati o n 
F or t he ori gi nal sa m ple si ze j ustificati o n f or t his st u d y, please refer t o t he pr ot oc ol. 


C he m oce ntr y x, I nc.  
Pr ot oc ol C L 0 0 2 1 6 8 
 
Statistical A nal ysis Pla n- Versi o n 2. 0 
P a ge 1 4  of 2 0 8 
5  
I nteri m A n al ysis 
Efficac y a n d safet y data fr o m t he st u d y were s u m marize d f or re vi e w b y t h e D M C at defi ne d 
p oi nts o ver t he c o urse of t he st u d y. T he D M C c harter i ncl u de d details of t he a nal ys es.  
As descri be d i n Secti o n 1, w he n t he last s u bject fr o m Ste p 2 c o m plete d t he st u d y, all a vaila bl e 
data were s u m marize d a n d e val uate d t o i nf or m d ecisi o ns re g ar di n g Ste p 3 of t his st u d y a n d 
f ut ure cli nical trials. 
6  
Ge ner al I nf or m ati o n R e g ar di n g D at a A n al yses 
 is res p o nsi ble f or pre p ari n g t he statistical a nal yses us e d t o s u p p ort t he fi n al st u d y 
re p ort f or Pr ot oc ol C L 0 0 2 _ 1 6 8.  All ta bl es a n d listi n gs will be ge n erat e d i n S A S®  versi o n 9. 3 or 
hi g her a n d all pr o gra ms use d t o ge nerate statistical a nal ys es will be vali date d acc or di n g t o 
 sta n dar d o perati n g pr oce d ures.  Ge nerall y, ta bles a n d listi n gs will be pri nte d usi n g 
C o urier Ne w 8 pt f o nt wit h all mar gi ns at least o ne i nc h.   T his c orres p o n ds t o setti n gs i n S A S of 
li nesiz e = 1 3 4 a n d pa gesiz e = 5 4.  T he f or mat of s o me dis pla ys ma y c h a n ge sli g htl y de p e n di n g o n 
t he act ual le n gt h of t he data dis pla ye d. 
P P D


C he m oce ntr y x, I nc.  
Pr ot oc ol C L 0 0 2 1 6 8 
 
Statistical A nal ysis Pla n- Versi o n 2. 0 
P a ge 1 5  of 2 0 8 
7  
T a ble S hells 
 
 


 
P a ge 1 6  of 2 0 8 
 
C h e m o C e n t r y x ,  I n c .  
P r o t o c o l  C L 0 0 2 _ 1 6 8                                                                                                          P a g e  1  o f  1  
T a b l e  1 4 . 1 . 1 . 1  
S u b j e c t  D i s p o s i t i o n  -  O v e r a l l  
A l l  E n r o l l e d  S u b j e c t s  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
                                               P l a c e b o  +            C C X 1 6 8  +             C C X 1 6 8  +  
  T o t a l                                   F u l l  D o s e  P r e d n i s o n e   L o w  D o s e  P r e d n i s o n e    N o  P r e d n i s o n e         A l l  C C X 1 6 8          T o t a l  
    C a t e g o r y                                     n    ( % )             n    ( % )             n    ( % )             n    ( % )             n    ( % )  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
  S c r e e n e d  
 
  F a i l e d  s c r e e n i n g  
    R e a s o n  1  
    R e a s o n  2  
 
  R a n d o m i z e d  
   
  S a f e t y  P o p u l a t i o n  
 
  I T T  P o p u l a t i o n  
 
  C o m p l e t e d  
 
  E a r l y  w i t h d r a w a l  o n / b e f o r e  D a y  8 5  
  E a r l y  w i t h d r a w a l  a f t e r  D a y  8 5  
 
  E a r l y  w i t h d r a w a l  ( t o t a l )  
    S u b j e c t  w i t h d r e w  c o n s e n t  
    S p o n s o r  d e c i s i o n  
    S u b j e c t  l o s t  t o  f o l l o w - u p  
    A d v e r s e  e v e n t  
    I n v e s t i g a t o r  d e c i s i o n  
    O t h e r  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
-  P e r c e n t a g e  o f  s c r e e n  f a i l u r e s  i s  b a s e d  o n  t h e  t o t a l  n u m b e r  o f  s u b j e c t s  s c r e e n e d .   P e r c e n t a g e s  o f  S a f e t y  p o p u l a t i o n  ( I n t e n t - t o - T r e a t )  
  p o p u l a t i o n ,  s u b j e c t s  c o m p l e t e d  a n d  e a r l y  w i t h d r a w a l s  a r e  b a s e d  o n  t h e  n u m b e r  o f  s u b j e c t s  r a n d o m i z e d .  
-  T h e  I T T  P o p u l a t i o n  i n c l u d e  a l l  s u b j e c t s  w h o  w e r e  r a n d o m i z e d  a n d  h a d  a t  l e a s t  o n e  p o s t  b a s e l i n e  o n  t r e a t m e n t  B V A S  m e a s u r e m e n t .   
-  T h e  S a f e t y  P o p u l a t i o n  i n c l u d e  a l l  s u b j e c t s  w h o  w e r e  r a n d o m i z e d  a n d  r e c e i v e d  a t  l e a s t  o n e  d o s e  o f  s t u d y  d r u g .  
 
P r o g r a m  N a m e :  X X X X X X X X . s a s                   R u n  D a t e :  D D M M M Y Y Y Y  H H : M M                                  D a t a b a s e  l a s t  m o d i f i e d :  D D M M Y Y Y Y  


 
P a ge 1 7  of 2 0 8 
 
C h e m o C e n t r y x ,  I n c .  
P r o t o c o l  C L 0 0 2 _ 1 6 8                                                                                                          P a g e  1  o f  1  
T a b l e  1 4 . 1 . 1 . 2  
S u b j e c t  D i s p o s i t i o n  b y  S t e p  
A l l  E n r o l l e d  S u b j e c t s  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
                                               P l a c e b o  +            C C X 1 6 8  +             C C X 1 6 8  +  
  S t e p                                    F u l l  D o s e  P r e d n i s o n e   L o w  D o s e  P r e d n i s o n e    N o  P r e d n i s o n e         A l l  C C X 1 6 8          T o t a l  
    C a t e g o r y                                     n    ( % )             n    ( % )             n    ( % )             n    ( % )             n    ( % )  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
  S t e p  1   
 
    S c r e e n e d  
 
    F a i l e d  s c r e e n i n g  
      R e a s o n  1  
      R e a s o n  2  
 
    R a n d o m i z e d  
 
    S a f e t y  P o p u l a t i o n  
 
    I T T  P o p u l a t i o n  
 
    C o m p l e t e d  
 
    E a r l y  w i t h d r a w a l  o n / b e f o r e  D a y  8 5  
    E a r l y  w i t h d r a w a l  a f t e r  D a y  8 5  
 
    E a r l y  w i t h d r a w a l  ( t o t a l )  
      S u b j e c t  w i t h d r e w  c o n s e n t  
      S p o n s o r  d e c i s i o n  
      S u b j e c t  l o s t  t o  f o l l o w - u p  
      A d v e r s e  e v e n t  
      I n v e s t i g a t o r  d e c i s i o n  
      O t h e r  
 
R e p e a t  f o r  S t e p  2  a n d  S t e p  3  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
-  P e r c e n t a g e  o f  s c r e e n  f a i l u r e s  i s  b a s e d  o n  t h e  t o t a l  n u m b e r  o f  s u b j e c t s  s c r e e n e d .   P e r c e n t a g e s  o f  S a f e t y  p o p u l a t i o n  ( I n t e n t - t o - T r e a t )  
  p o p u l a t i o n ,  s u b j e c t s  c o m p l e t e d  a n d  e a r l y  w i t h d r a w a l s  a r e  b a s e d  o n  t h e  n u m b e r  o f  s u b j e c t s  r a n d o m i z e d .  
-  T h e  I T T  P o p u l a t i o n  i n c l u d e  a l l  s u b j e c t s  w h o  w e r e  r a n d o m i z e d  a n d  h a d  a t  l e a s t  o n e  p o s t  b a s e l i n e  o n  t r e a t m e n t  B V A S  m e a s u r e m e n t .   
-  T h e  S a f e t y  P o p u l a t i o n  i n c l u d e  a l l  s u b j e c t s  w h o  w e r e  r a n d o m i z e d  a n d  r e c e i v e d  a t  l e a s t  o n e  d o s e  o f  s t u d y  d r u g .  
 
P r o g r a m  N a m e :  X X X X X X X X . s a s                   R u n  D a t e :  D D M M M Y Y Y Y  H H : M M                                  D a t a b a s e  l a s t  m o d i f i e d :  D D M M Y Y Y Y  
 


 
P a ge 1 8  of 2 0 8 
 
C h e m o C e n t r y x ,  I n c .  
P r o t o c o l  C L 0 0 2 _ 1 6 8                                                                                                          P a g e  1  o f  #  
T a b l e  1 4 . 1 . 2  
L i s t i n g  o f  S u b j e c t s  w h o  W i t h d r e w  P r e m a t u r e l y  
A l l  R a n d o m i z e d  S u b j e c t s  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
S t e p                                                                                   C u m u l a t i v e  
  T r e a t m e n t                         D a t e       F i r s t  D o s e     W i t h d r a w a l      L a s t  D o s e      C C X 1 6 8  
    S u b j e c t     A g e / S e x / R a c e      R a n d o m i z e d       D a t e         D a t e / D a y        D a t e / D a y     D o s e  ( m g )     R e a s o n  f o r  w i t h d r a w a l  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
S t e p  1  
 X X X X X X X X X X X X X  
    # # # - # # #   # # / M / X X X X X X X X X X     D D M M M Y Y Y Y     D D M M M Y Y Y Y     D D M M M Y Y Y Y / # #   D D M M M Y Y Y Y / # #     # # # #        X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X  
 
 
S t e p  2  
 X X X X X X X X X X X X X  
    # # # - # # #   # # / M / X X X X X X X X X X     D D M M M Y Y Y Y     D D M M M Y Y Y Y     D D M M M Y Y Y Y / # #   D D M M M Y Y Y Y / # #     # # # #        X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
N o t e :  S t u d y  d a y  i s  c a l c u l a t e d  b a s e d  o n  t h e  d a t e  o f  f i r s t  d o s e .  
 
P r o g r a m  N a m e :  X X X X X X X X . s a s                   R u n  D a t e :  D D M M M Y Y Y Y  H H : M M                                  D a t a b a s e  l a s t  m o d i f i e d :  D D M M Y Y Y Y  
 
 
 
N O T E 1: T his ta ble will be s orte d b y Ste p, treat m e nt gr o u p, a n d s u bject wit hi n treat me nt gr o u p.   
N O T E 2: C u m ulati ve d ose will be re p orte d b ase d o n t he n u m ber of ca ps ules ta ke n, w here eac h ca ps ule re prese nts 1 0 m g f or t he 
C C X 1 6 8 treat me nt gr o u p a n d 0 m g f or t he place b o gr o u p. 


 
P a ge 1 9  of 2 0 8 
 
C h e m o C e n t r y x ,  I n c .  
P r o t o c o l  C L 0 0 2 _ 1 6 8                                                                                                          P a g e  1  o f  #  
T a b l e  1 4 . 1 . 3 . 1  
S u m m a r y  o f  S u b j e c t  D e m o g r a p h i c s  -  O v e r a l l  
A l l  R a n d o m i z e d  S u b j e c t s  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
                                               P l a c e b o  +            C C X 1 6 8  +             C C X 1 6 8  +  
  D e m o g r a p h i c  c h a r a c t e r i s t i c              F u l l  D o s e  P r e d n i s o n e   L o w  D o s e  P r e d n i s o n e    N o  P r e d n i s o n e         A l l  C C X 1 6 8           T o t a l  
    S t a t i s t i c / C a t e g o r y                           ( N = # # )               ( N = # # )              ( N = # # )               ( N = # # )             ( N = # # )   
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
  A g e  ( Y e a r s )  
    n                                             # #                   # #                  # #                   # #                 # #  
    M e a n                                          # # . #                 # # . #                # # . #                 # # . #               # # . #  
    S D                                            # # . # #                # # . # #               # # . # #                # # . # #              # # . # #   
    M i n i m u m                                       # #                   # #                  # #                   # #                 # #    
    M e d i a n                                        # # . #                 # # . #                # # . #                 # # . #               # # . #  
    M a x i m u m                                       # #                   # #                  # #                   # #                 # #    
 
  G e n d e r ,  n  ( % )  
    M a l e                                       # #  ( # # # . # )           # #  ( # # # . # )          # #  ( # # # . # )           # #  ( # # # . # )         # #  ( # # # . # )  
    F e m a l e                                     # #  ( # # # . # )           # #  ( # # # . # )          # #  ( # # # . # )           # #  ( # # # . # )         # #  ( # # # . # )  
 
  E t h n i c i t y ,  n  ( % )  
    H i s p a n i c  o r  L a t i n o  
    N o t  H i s p a n i c  o r  L a t i n o  
 
  R a c e ,  n  ( % )  
    A s i a n  
    A m e r i c a n  I n d i a n  o r  A l a s k a  N a t i v e  
    B l a c k  o r  A f r i c a n  A m e r i c a n  
    N a t i v e  H a w a i i a n  o r  O t h e r  
     P a c i f i c  I s l a n d e r  
    W h i t e  
    O t h e r  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
P r o g r a m  N a m e :  X X X X X X X X . s a s                   R u n  D a t e :  D D M M M Y Y Y Y  H H : M M                                  D a t a b a s e  l a s t  m o d i f i e d :  D D M M Y Y Y Y  
 
 
 
N O T E:  O nl y n o n-zer o race cate g ori es will be i ncl u de d.  


 
P a ge 2 0  of 2 0 8 
 
C h e m o C e n t r y x ,  I n c .  
P r o t o c o l  C L 0 0 2 _ 1 6 8                                                                                                          P a g e  1  o f  #  
T a b l e  1 4 . 1 . 3 . 2  
S u m m a r y  o f  S u b j e c t  D e m o g r a p h i c s  b y  S t e p  
A l l  R a n d o m i z e d  S u b j e c t s  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
                                               S t e p  1                      S t e p  2                             S t e p  3  
                                    - - - - - - - - - - - - - - - - - - - - - - - - - -    - - - - - - - - - - - - - - - - - - - - - - - -    - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - -  
                                     P l a c e b o  +        C C X 1 6 8  +      P l a c e b o  +      C C X 1 6 8  +     P l a c e b o  +        C C X 1 6 8  +       C C X 1 6 8  +  
                                     F u l l  D o s e       L o w  D o s e       F u l l  D o s e         N o        F u l l  D o s e        L o w  D o s e          N o  
  D e m o g r a p h i c  c h a r a c t e r i s t i c          P r e d n i s o n e     P r e d n i s o n e      P r e d n i s o n e    P r e d n i s o n e    P r e d n i s o n e      P r e d n i s o n e      P r e d n i s o n e  
    S t a t i s t i c / C a t e g o r y                  ( N = # # )         ( N = # # )          ( N = # # )        ( N = # # )         ( N = # # )         ( N = # # )          ( N = # # )  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
  A g e  a t  s c r e e n i n g   
    n                                   # #             # #              # #            # #             # #             # #              # #  
    M e a n                                # # . #           # # . #            # # . #          # # . #           # # . #           # # . #            # # . #  
    S D                                  # # . # #          # # . # #           # # . # #         # # . # #          # # . # #          # # . # #           # # . # #  
    M i n i m u m                             # #             # #              # #            # #             # #             # #              # #  
    M e d i a n                              # # . #           # # . #            # # . #          # # . #           # # . #           # # . #            # # . #  
    M a x i m u m                             # #             # #              # #            # #             # #             # #              # #  
 
  G e n d e r ,  n  ( % )  
    M a l e                              # #  ( # # # . # )     # #  ( # # # . # )      # #  ( # # # . # )      # #  ( # # # . # )      # #  ( # # # . # )    # #  ( # # # . # )    # #  ( # # # . # )  
    F e m a l e                            # #  ( # # # . # )     # #  ( # # # . # )      # #  ( # # # . # )      # #  ( # # # . # )      # #  ( # # # . # )    # #  ( # # # . # )    # #  ( # # # . # )  
 
  E t h n i c i t y ,  n  ( % )  
    H i s p a n i c  o r  L a t i n o  
    N o t  H i s p a n i c  o r  L a t i n o  
 
  R a c e ,  n  ( % )  
    A s i a n  
    A m e r i c a n  I n d i a n  o r  A l a s k a  N a t i v e  
    B l a c k  o r  A f r i c a n  A m e r i c a n  
    N a t i v e  H a w a i i a n  o r  O t h e r  
     P a c i f i c  I s l a n d e r  
    W h i t e  
    O t h e r  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
P r o g r a m  N a m e :  X X X X X X X X . s a s                   R u n  D a t e :  D D M M M Y Y Y Y  H H : M M                                  D a t a b a s e  l a s t  m o d i f i e d :  D D M M Y Y Y Y  
 
 
 
N O T E:  O nl y n o n-zer o race cate g ori es will be i ncl u de d.  


 
P a ge 2 1  of 2 0 8 
 
C h e m o C e n t r y x ,  I n c .  
P r o t o c o l  C L 0 0 2 _ 1 6 8                                                                                                          P a g e  1  o f  #  
T a b l e  1 4 . 1 . 4 . 1  
S u m m a r y  o f  B a s e l i n e  C h a r a c t e r i s t i c s  -  O v e r a l l  
A l l  R a n d o m i z e d  S u b j e c t s  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
                                               P l a c e b o  +            C C X 1 6 8  +             C C X 1 6 8  +  
  B a s e l i n e  c h a r a c t e r i s t i c                F u l l  D o s e  P r e d n i s o n e   L o w  D o s e  P r e d n i s o n e    N o  P r e d n i s o n e         A l l  C C X 1 6 8           T o t a l  
    S t a t i s t i c / C a t e g o r y                           ( N = # # )               ( N = # # )              ( N = # # )               ( N = # # )             ( N = # # )   
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
  B o d y  w e i g h t  ( k g )  
    n                                            # #                   # #                  # #                   # #                 # #  
    M e a n                                         # # . #                 # # . #                # # . #                 # # . #               # # . #  
    S D                                           # # . # #                # # . # #               # # . # #                # # . # #              # # . # #  
    M i n i m u m                                      # #                   # #                  # #                   # #                 # #  
    M e d i a n                                       # # . #                 # # . #                # # . #                 # # . #               # # . #  
    M a x i m u m                                      # #                   # #                  # #                   # #                 # #  
 
  S m o k i n g  S t a t u s ,  n  ( % )  
    C u r r e n t  S m o k e r                           # #  ( # # # . # )           # #  ( # # # . # )          # #  ( # # # . # )      # #  ( # # # . # )              # #  ( # # # . # )  
    P a s t  S m o k e r  
    N e v e r  S m o k e d  
 
   T h i s  t a b l e  w i l l  a l s o  i n c l u d e  s u m m a r i e s  o f  b a s e l i n e  d a t a  f o r  t h e  f o l l o w i n g  p a r a m e t e r s :  
 
    H e i g h t  ( c m ) ,  B M I  ( k g / m ^ 2 ) ,  H e a r t  r a t e ,  S y s t o l i c  b l o o d  p r e s s u r e ,  D i a s t o l i c  b l o o d  p r e s s u r e ,  O r a l  t e m p e r a t u r e ,  T B  s c r e e n  r e s u l t s ,   
    v i r a l  t e s t  r e s u l t s ,  A N C A - n e w l y  d i a g n o s e d  v e r s u s  r e l a p s e d ,  A N C A  a s s o c i a t e d  v a s c u l i t i s  d i s e a s e  d u r a t i o n  [ 1 ] ,  s t a n d a r d  o f  c a r e   
    ( r i t u x i m a b  o r  c y c l o p h o s p h a m i d e ) ,  a n t i - M P O +  [ 2 ] ,  a n t i - P R 3  +  [ 2 ] ,  a n t i - M P O +  a n d  a n t i - P R 3 +  s t a t u s ,  t y p e  o f  A A V  ( G P A ,  M P O ,  o r  r e n a l   
    l i m i t e d  v a s c u l i t i s ) ,  B V A S ,  V D I ,  h s C R P ,  e G F R ,  h e m a t u r i a ,  u r i n e  R B C  c a s t s ,  p r o t e i n u r i a  ( A C R ) ,  u r i n a r y  M C P - 1 : c r e a t i n i n e   r a t i o ,  S F - 3 6   
    d o m a i n  s c o r e s ,  E Q - 5 D - 5 L  s c o r e  a n d  E Q - 5 D - 5 L  V A S .  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
[ 1 ]  I f  s u b j e c t  i s  r e l a p s e d ,  t h e  r e c o r d e d  d i a g n o s i s  d a t e  i s  u s e d  f o r  t h i s  s u m m a r y .  
[ 2 ]  F o r  s u m m a r i e s  o f  A n t i - M P O +  ( I U / m L )  a n d  A n t i - P R 3 +  ( I U / m L ) ,  o n l y  s u b j e c t s  w i t h  a  n o n - n e g a t i v e  b a s e l i n e  c a t e g o r i c a l  s t a t u s  a r e  
    i n c l u d e d .  R e s u l t s  r e p o r t e d  a s  ' > 1 5 6 . 0 '  a r e  t r e a t e d  a s  e q u a l  t o  1 5 6 . 0  f o r  c o m p u t i n g  s u m m a r y  s t a t i s t i c s .  
 
P r o g r a m  N a m e :  X X X X X X X X . s a s                   R u n  D a t e :  D D M M M Y Y Y Y  H H : M M                                  D a t a b a s e  l a s t  m o d i f i e d :  D D M M Y Y Y Y  


 
P a ge 2 2  of 2 0 8 
 
C h e m o C e n t r y x ,  I n c .  
P r o t o c o l  C L 0 0 2 _ 1 6 8                                                                                                          P a g e  1  o f  #  
T a b l e  1 4 . 1 . 4 . 2  
S u m m a r y  o f  B a s e l i n e  C h a r a c t e r i s t i c s  b y  S t e p  
A l l  R a n d o m i z e d  S u b j e c t s  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
                                               S t e p  1                      S t e p  2                             S t e p  3  
                                    - - - - - - - - - - - - - - - - - - - - - - - - - -    - - - - - - - - - - - - - - - - - - - - - - - -    - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - -  
                                     P l a c e b o  +        C C X 1 6 8  +      P l a c e b o  +      C C X 1 6 8  +     P l a c e b o  +        C C X 1 6 8  +       C C X 1 6 8  +  
                                     F u l l  D o s e       L o w  D o s e       F u l l  D o s e         N o        F u l l  D o s e        L o w  D o s e          N o  
  B a s e l i n e  c h a r a c t e r i s t i c             P r e d n i s o n e     P r e d n i s o n e      P r e d n i s o n e    P r e d n i s o n e    P r e d n i s o n e      P r e d n i s o n e      P r e d n i s o n e  
    S t a t i s t i c / C a t e g o r y                  ( N = # # )         ( N = # # )          ( N = # # )        ( N = # # )         ( N = # # )         ( N = # # )          ( N = # # )  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
  B o d y  w e i g h t  ( k g )  
    n                                   # #             # #              # #            # #             # #             # #              # #  
    M e a n                                # # . #           # # . #            # # . #          # # . #           # # . #           # # . #            # # . #  
    S D                                  # # . # #          # # . # #           # # . # #         # # . # #          # # . # #          # # . # #           # # . # #  
    M i n i m u m                             # #             # #              # #            # #             # #             # #              # #  
    M e d i a n                              # # . #           # # . #            # # . #          # # . #           # # . #           # # . #            # # . #  
    M a x i m u m                             # #             # #              # #            # #             # #             # #              # #  
 
  S m o k i n g  S t a t u s ,  n  ( % )  
    C u r r e n t  S m o k e r                   # #  ( # # # . # )     # #  ( # # # . # )      # #  ( # # # . # )      # #  ( # # # . # )      # #  ( # # # . # )    # #  ( # # # . # )    # #  ( # # # . # )  
    P a s t  S m o k e r  
    N e v e r  S m o k e d  
 
    H e i g h t  ( c m ) ,  B M I  ( k g / m ^ 2 ) ,  H e a r t  r a t e ,  S y s t o l i c  b l o o d  p r e s s u r e ,  D i a s t o l i c  b l o o d  p r e s s u r e ,  O r a l  t e m p e r a t u r e ,  T B  s c r e e n  r e s u l t s ,   
    v i r a l  t e s t  r e s u l t s ,  A N C A - n e w l y  d i a g n o s e d  v e r s u s  r e l a p s e d ,  A N C A  a s s o c i a t e d  v a s c u l i t i s  d i s e a s e  d u r a t i o n  [ 1 ] ,  s t a n d a r d  o f  c a r e   
    ( r i t u x i m a b  o r  c y c l o p h o s p h a m i d e ) ,  a n t i - M P O +  [ 2 ] ,  a n t i - P R 3  +  [ 2 ] ,  a n t i - M P O +  a n d  a n t i - P R 3 +  s t a t u s ,  t y p e  o f  A A V  ( G P A ,  M P O ,  o r  r e n a l   
    l i m i t e d  v a s c u l i t i s ) ,  B V A S ,  V D I ,  h s C R P ,  e G F R ,  h e m a t u r i a ,  u r i n e  R B C  c a s t s ,  p r o t e i n u r i a  ( A C R ) ,  u r i n a r y  M C P - 1 : c r e a t i n i n e   r a t i o ,  S F - 3 6   
    d o m a i n  s c o r e s ,  E Q - 5 D - 5 L  s c o r e  a n d  E Q - 5 D - 5 L  V A S .  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
[ 1 ]  I f  s u b j e c t  i s  r e l a p s e d ,  t h e  m o s t  r e c e n t  d i a g n o s i s  d a t e  i s  u s e d  f o r  t h i s  s u m m a r y .  
[ 2 ]  F o r  s u m m a r i e s  o f  A n t i - M P O +  ( I U / m L )  a n d  A n t i - P R 3 +  ( I U / m L ) ,  o n l y  s u b j e c t s  w i t h  a  n o n - n e g a t i v e  b a s e l i n e  c a t e g o r i c a l  s t a t u s  a r e  
    i n c l u d e d .  R e s u l t s  r e p o r t e d  a s  ' > 1 5 6 . 0 '  w e r e  t r e a t e d  a s  e q u a l  t o  1 5 6 . 0  f o r  c o m p u t i n g  s u m m a r y  s t a t i s t i c s .  
 
P r o g r a m  N a m e :  X X X X X X X X . s a s                   R u n  D a t e :  D D M M M Y Y Y Y  H H : M M                                  D a t a b a s e  l a s t  m o d i f i e d :  D D M M Y Y Y Y  


 
P a ge 2 3  of 2 0 8 
 
C h e m o C e n t r y x ,  I n c .  
P r o t o c o l  C L 0 0 2 _ 1 6 8                                                                                                          P a g e  1  o f  #  
T a b l e  1 4 . 1 . 5 . 1  
S u m m a r y  o f  P r i o r  M e d i c a t i o n s  -  O v e r a l l  
A l l  R a n d o m i z e d  S u b j e c t s  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
                                               P l a c e b o  +             C C X 1 6 8  +              C C X 1 6 8  +  
                                         F u l l  D o s e  P r e d n i s o n e   L o w  D o s e  P r e d n i s o n e    N o  P r e d n i s o n e        A l l  C C X 1 6 8           T o t a l  
  A n a t o m i c  T h e r a p e u t i c  C l a s s                     ( N = # # )               ( N = # # )               ( N = # # )              ( N = # # )             ( N = # # )  
    P r e f e r r e d  T e r m                               n    ( % )              n    ( % )              n    ( % )             n    ( % )            n    ( % )  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
  A n y  P r i o r  M e d i c a t i o n                          # #  ( # # # . # )           # #  ( # # # . # )           # #  ( # # # . # )          # #  ( # # # . # )         # #  ( # # # . # )  
 
  A n a t o m i c  T h e r a p e u t i c  C l a s s  1                  # #  ( # # # . # )           # #  ( # # # . # )           # #  ( # # # . # )          # #  ( # # # . # )         # #  ( # # # . # )  
     P r e f e r r e d  T e r m  1                           # #  ( # # # . # )           # #  ( # # # . # )           # #  ( # # # . # )          # #  ( # # # . # )         # #  ( # # # . # )  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
-  P r i o r  m e d i c a t i o n s  a r e  d e f i n e d  a s  a n y  m e d i c a t i o n  t a k e n  w i t h i n  6  m o n t h s  o f  s c r e e n i n g ,  d u r i n g  t h e  s c r e e n i n g  p e r i o d  b e t w e e n  t h e   
  s c r e e n i n g  v i s i t  a n d  t h e  d a y  o f  r a n d o m i z a t i o n .    
 
P r o g r a m  N a m e :  X X X X X X X X . s a s                   R u n  D a t e :  D D M M M Y Y Y Y  H H : M M                            D a t a b a s e  l a s t  m o d i f i e d :  D D M M Y Y Y Y  H H : M M  
 
 
 
N O T E: Ta ble w ill be s ort e d b y d esce n di n g fre q u e nc y i n t he t otal c ol u m n f or a nat o mic t hera pe utic class a n d preferre d ter m wit hi n 
a nat o mic t hera p e utic class.  


 
P a ge 2 4  of 2 0 8 
 
C h e m o C e n t r y x ,  I n c .  
P r o t o c o l  C L 0 0 2 _ 1 6 8                                                                                                          P a g e  1  o f  #  
T a b l e  1 4 . 1 . 5 . 2  
S u m m a r y  o f  P r i o r  M e d i c a t i o n s  b y  S t e p  
A l l  R a n d o m i z e d  S u b j e c t s  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
                                               S t e p  1                      S t e p  2                             S t e p  3  
                                    - - - - - - - - - - - - - - - - - - - - - - - - - -    - - - - - - - - - - - - - - - - - - - - - - - -    - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - -  
                                     P l a c e b o  +       C C X 1 6 8  +       P l a c e b o  +      C C X 1 6 8  +      P l a c e b o  +       C C X 1 6 8  +       C C X 1 6 8  +  
                                     F u l l  D o s e       L o w  D o s e       F u l l  D o s e         N o         F u l l  D o s e       L o w  D o s e          N o  
                                     P r e d n i s o n e     P r e d n i s o n e      P r e d n i s o n e     P r e d n i s o n e    P r e d n i s o n e     P r e d n i s o n e     P r e d n i s o n e  
  A n a t o m i c  T h e r a p e u t i c  C l a s s            ( N = # # )         ( N = # # )          ( N = # # )        ( N = # # )         ( N = # # )         ( N = # # )          ( N = # # )  
    P r e f e r r e d  T e r m                      n    ( % )        n    ( % )         n    ( % )       n    ( % )        n    ( % )        n    ( % )         n    ( % )  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
  A n y  P r i o r  M e d i c a t i o n                 # #  ( # # # . # )     # #  ( # # # . # )      # #  ( # # # . # )    # #  ( # # # . # )     # #  ( # # # . # )     # #  ( # # # . # )      # #  ( # # # . # )  
  
  A n a t o m i c  T h e r a p e u t i c  C l a s s  1         # #  ( # # # . # )     # #  ( # # # . # )      # #  ( # # # . # )    # #  ( # # # . # )     # #  ( # # # . # )     # #  ( # # # . # )      # #  ( # # # . # )  
     P r e f e r r e d  T e r m  1                  # #  ( # # # . # )     # #  ( # # # . # )      # #  ( # # # . # )    # #  ( # # # . # )     # #  ( # # # . # )     # #  ( # # # . # )      # #  ( # # # . # )  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
-  P r i o r  m e d i c a t i o n s  a r e  d e f i n e d  a s  a n y  m e d i c a t i o n  t a k e n  w i t h i n  6  m o n t h s  o f  s c r e e n i n g ,  d u r i n g  t h e  s c r e e n i n g  p e r i o d  b e t w e e n  t h e   
  s c r e e n i n g  v i s i t  a n d  t h e  d a y  o f  r a n d o m i z a t i o n .  
 
P r o g r a m  N a m e :  X X X X X X X X . s a s                   R u n  D a t e :  D D M M M Y Y Y Y  H H : M M                            D a t a b a s e  l a s t  m o d i f i e d :  D D M M Y Y Y Y  H H : M M  
 
 
 
N O T E: Ta ble will be s ort e d b y d esce n di n g fre q u e nc y i n t he t otal c ol u m n f or a nat o mic t hera pe utic class a n d preferre d ter m wit hi n 
a nat o mic t hera p e utic class. 


 
P a ge 2 5  of 2 0 8 
 
C h e m o C e n t r y x ,  I n c .  
P r o t o c o l  C L 0 0 2 _ 1 6 8                                                                                                          P a g e  1  o f  #  
T a b l e  1 4 . 1 . 6 . 1  
S u m m a r y  o f  C o n c o m i t a n t  M e d i c a t i o n s  -  O v e r a l l  
A l l  R a n d o m i z e d  S u b j e c t s  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
                                               P l a c e b o  +             C C X 1 6 8  +              C C X 1 6 8  +  
                                         F u l l  D o s e  P r e d n i s o n e   L o w  D o s e  P r e d n i s o n e    N o  P r e d n i s o n e        A l l  C C X 1 6 8           T o t a l  
  A n a t o m i c  T h e r a p e u t i c  C l a s s                     ( N = # # )               ( N = # # )               ( N = # # )              ( N = # # )             ( N = # # )  
    P r e f e r r e d  T e r m                               n    ( % )              n    ( % )              n    ( % )             n    ( % )            n    ( % )  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
  A n y  C o n c o m i t a n t  M e d i c a t i o n                    # #  ( # # # . # )           # #  ( # # # . # )           # #  ( # # # . # )          # #  ( # # # . # )         # #  ( # # # . # )  
 
  A n a t o m i c  T h e r a p e u t i c  C l a s s  1                  # #  ( # # # . # )           # #  ( # # # . # )           # #  ( # # # . # )          # #  ( # # # . # )         # #  ( # # # . # )  
     P r e f e r r e d  T e r m  1                           # #  ( # # # . # )           # #  ( # # # . # )           # #  ( # # # . # )          # #  ( # # # . # )         # #  ( # # # . # )  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
-  C o n c o m i t a n t  m e d i c a t i o n s  a r e  d e f i n e d  a s  a n y  m e d i c a t i o n  t a k e n  o n  o r  a f t e r  t h e  d a y  o f  r a n d o m i z a t i o n .  
 
P r o g r a m  N a m e :  X X X X X X X X . s a s                   R u n  D a t e :  D D M M M Y Y Y Y  H H : M M                            D a t a b a s e  l a s t  m o d i f i e d :  D D M M Y Y Y Y  H H : M M  
 
 
 
N O T E: Ta ble will be s ort e d b y d esce n di n g fre q u e nc y i n t he t otal c ol u m n f or a nat o mic t hera pe utic class a n d preferre d ter m wit hi n 
a nat o mic t hera p e utic class. 


 
P a ge 2 6  of 2 0 8 
 
C h e m o C e n t r y x ,  I n c .  
P r o t o c o l  C L 0 0 2 _ 1 6 8                                                                                                          P a g e  1  o f  #  
T a b l e  1 4 . 1 . 6 . 2  
S u m m a r y  o f  C o n c o m i t a n t  M e d i c a t i o n s  b y  S t e p  
A l l  R a n d o m i z e d  S u b j e c t s  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
                                               S t e p  1                      S t e p  2                             S t e p  3  
                                    - - - - - - - - - - - - - - - - - - - - - - - - - -    - - - - - - - - - - - - - - - - - - - - - - - -    - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - -  
                                     P l a c e b o  +       C C X 1 6 8  +       P l a c e b o  +      C C X 1 6 8  +      P l a c e b o  +       C C X 1 6 8  +       C C X 1 6 8  +  
                                     F u l l  D o s e       L o w  D o s e       F u l l  D o s e         N o         F u l l  D o s e       L o w  D o s e          N o  
                                     P r e d n i s o n e     P r e d n i s o n e      P r e d n i s o n e     P r e d n i s o n e    P r e d n i s o n e     P r e d n i s o n e     P r e d n i s o n e  
  A n a t o m i c  T h e r a p e u t i c  C l a s s            ( N = # # )         ( N = # # )          ( N = # # )        ( N = # # )         ( N = # # )         ( N = # # )          ( N = # # )  
    P r e f e r r e d  T e r m                      n    ( % )        n    ( % )         n    ( % )       n    ( % )        n    ( % )        n    ( % )         n    ( % )  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
  A n y  C o n c o m i t a n t  M e d i c a t i o n           # #  ( # # # . # )     # #  ( # # # . # )      # #  ( # # # . # )    # #  ( # # # . # )     # #  ( # # # . # )     # #  ( # # # . # )      # #  ( # # # . # )  
  
  A n a t o m i c  T h e r a p e u t i c  C l a s s  1         # #  ( # # # . # )     # #  ( # # # . # )      # #  ( # # # . # )    # #  ( # # # . # )     # #  ( # # # . # )     # #  ( # # # . # )      # #  ( # # # . # )  
     P r e f e r r e d  T e r m  1                  # #  ( # # # . # )     # #  ( # # # . # )      # #  ( # # # . # )    # #  ( # # # . # )     # #  ( # # # . # )     # #  ( # # # . # )      # #  ( # # # . # )  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
-  C o n c o m i t a n t  m e d i c a t i o n s  a r e  d e f i n e d  a s  a n y  m e d i c a t i o n  t a k e n  o n  o r  a f t e r  t h e  d a y  o f  r a n d o m i z a t i o n .  
 
P r o g r a m  N a m e :  X X X X X X X X . s a s                   R u n  D a t e :  D D M M M Y Y Y Y  H H : M M                            D a t a b a s e  l a s t  m o d i f i e d :  D D M M Y Y Y Y  H H : M M  
 
 
 
N O T E: Ta ble will be s ort e d b y d esce n di n g fre q u e nc y i n t he t ot al c ol u m n f or a nat o mic t hera pe utic class a n d preferre d ter m wit hi n 
a nat o mic t hera p e utic class. 


 
P a ge 2 7  of 2 0 8 
 
C h e m o C e n t r y x ,  I n c .  
P r o t o c o l  C L 0 0 2 _ 1 6 8                                                                                                          P a g e  #  o f  #  
T a b l e  1 4 . 2 . 1 . 1  
A n a l y s i s  o f  B V A S  R e s p o n s e  
I n t e n t - t o - T r e a t  P o p u l a t i o n  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
                                                                             D i f f e r e n c e                          
  D a y                                                                       i n  p e r c e n t a g e s    T w o - s i d e d  9 0 %  C I   N o n - i n f e r .   S u p e r i o r .  
   S t e p      T r e a t m e n t                                N          n     ( % )      v e r s u s  P l a c e b o     f o r  D i f f e r e n c e     P - v a l u e     P - v a l u e  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
  D a y  8 5  
    A l l      P l a c e b o  +  F u l l  D o s e  P r e d n i s o n e  ( N = # # )   # # #        # # #  ( # # # . # )  
            C C X 1 6 8  +  L o w  D o s e  P r e d n i s o n e  ( N = # # )     # # #        # # #  ( # # # . # )       # # . # #           ( # . # # ,  # . # # )       # . # # # #      # . # # # #  
            C C X 1 6 8  +  N o  P r e d n i s o n e  ( N = # # )           # # #        # # #  ( # # # . # )       # # . # #           ( # . # # ,  # . # # )       # . # # # #      # . # # # #  
            A l l  C C X 1 6 8  ( N = # # )                       # # #        # # #  ( # # # . # )       # # . # #           ( # . # # ,  # . # # )       # . # # # #      # . # # # #  
 
    3        P l a c e b o  +  F u l l  D o s e  P r e d n i s o n e  ( N = # # )   # # #        # # #  ( # # # . # )  
            C C X 1 6 8  +  L o w  D o s e  P r e d n i s o n e  ( N = # # )     # # #        # # #  ( # # # . # )       # # . # #           ( # . # # ,  # . # # )       # . # # # #      # . # # # #  
            C C X 1 6 8  +  N o  P r e d n i s o n e  ( N = # # )           # # #        # # #  ( # # # . # )       # # . # #           ( # . # # ,  # . # # )       # . # # # #      # . # # # #  
            A l l  C C X 1 6 8  ( N = # # )                       # # #        # # #  ( # # # . # )       # # . # #           ( # . # # ,  # . # # )       # . # # # #      # . # # # #  
 
    1 + 2      P l a c e b o  +  F u l l  D o s e  P r e d n i s o n e  ( N = # # )   # # #        # # #  ( # # # . # )  
            C C X 1 6 8  +  L o w  D o s e  P r e d n i s o n e  ( N = # # )     # # #        # # #  ( # # # . # )       # # . # #           ( # . # # ,  # . # # )       # . # # # #      # . # # # #  
            C C X 1 6 8  +  N o  P r e d n i s o n e  ( N = # # )           # # #        # # #  ( # # # . # )       # # . # #           ( # . # # ,  # . # # )       # . # # # #      # . # # # #  
            A l l  C C X 1 6 8  ( N = # # )                       # # #        # # #  ( # # # . # )       # # . # #           ( # . # # ,  # . # # )       # . # # # #      # . # # # #  
 
    2        P l a c e b o  +  F u l l  D o s e  P r e d n i s o n e  ( N = # # )   # # #        # # #  ( # # # . # )  
            C C X 1 6 8  +  N o  P r e d n i s o n e  ( N = # # )           # # #        # # #  ( # # # . # )  
 
    1        P l a c e b o  +  F u l l  D o s e  P r e d n i s o n e  ( N = # # )   # # #        # # #  ( # # # . # )  
            C C X 1 6 8  +  L o w  D o s e  P r e d n i s o n e  ( N = # # )     # # #        # # #  ( # # # . # )  
 
R e p e a t  f o r  D a y  1 6 9  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
-  B V A S  r e s p o n s e  i s  d e f i n e d  a s  a c h i e v i n g  a  5 0 %  r e d u c t i o n  f r o m  b a s e l i n e  i n  t h e  B V A S  p l u s  n o  w o r s e n i n g  i n  a n y  b o d y  s y s t e m  c o m p o n e n t .  
 
P r o g r a m  N a m e :  X X X X X X X X . s a s                   R u n  D a t e :  D D M M M Y Y Y Y  H H : M M                                  D a t a b a s e  l a s t  m o d i f i e d :  D D M M Y Y Y Y  


 
P a ge 2 8  of 2 0 8 
 
T he f oll o wi n g t a bles will ha ve t he sa me l a y o ut as Ta ble 1 4. 2. 1. 1: 
T a b l e  1 4 . 2 . 1 . 2  
A n a l y s i s  o f  B V A S  R e s p o n s e  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 1 . 3  
A n a l y s i s  o f  B V A S  R e s p o n s e  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N o n - R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 1 . 4  
A n a l y s i s  o f  B V A S  R e s p o n s e  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  C y c l o p h o s p h a m i d e  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 1 . 5  
A n a l y s i s  o f  B V A S  R e s p o n s e  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  R i t u x i m a b  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 1 . 6  
A n a l y s i s  o f  B V A S  R e s p o n s e  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N e w l y  D i a g n o s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 . 7  
A n a l y s i s  o f  B V A S  R e s p o n s e  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e l a p s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 . 8  
A n a l y s i s  o f  B V A S  R e s p o n s e  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M P O +  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 . 9  
A n a l y s i s  o f  B V A S  R e s p o n s e  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  P R 3 +  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 . 1 0  
A n a l y s i s  o f  B V A S  R e s p o n s e  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  G r a n u l o m a t o s i s  w i t h  P o l y a n g i i t i s  ( W e g e n e r ’ s )  
 
T a b l e  1 4 . 2 . 1 . 1 1  
A n a l y s i s  o f  B V A S  R e s p o n s e  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M i c r o s c o p i c  P o l y a n g i i t i s  


 
P a ge 2 9  of 2 0 8 
 
C h e m o C e n t r y x ,  I n c .  
P r o t o c o l  C L 0 0 2 _ 1 6 8                                                                                                          P a g e  #  o f  #  
T a b l e  1 4 . 2 . 2 . 1  
A n a l y s i s  o f  R e n a l  R e s p o n s e  f o r  P a t i e n t s  w i t h  H e m a t u r i a  a n d  A l b u m i n u r i a  a t  B a s e l i n e  
I n t e n t - t o - T r e a t  P o p u l a t i o n  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
                                                                             D i f f e r e n c e                          
  D a y                                                                       i n  p e r c e n t a g e s    T w o - s i d e d  9 0 %  C I   N o n - i n f e r .   S u p e r i o r .  
   S t e p      T r e a t m e n t                                N ’         n     ( % )      v e r s u s  P l a c e b o     f o r  D i f f e r e n c e     P - v a l u e     P - v a l u e  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
  
  D a y  8 5  
   
    A l l      P l a c e b o  +  F u l l  D o s e  P r e d n i s o n e  ( N = # # )   # # #        # # #  ( # # # . # )  
            C C X 1 6 8  +  L o w  D o s e  P r e d n i s o n e  ( N = # # )     # # #        # # #  ( # # # . # )       # # . # #           ( # . # # ,  # . # # )       # . # # # #      # . # # # #  
            C C X 1 6 8  +  N o  P r e d n i s o n e  ( N = # # )           # # #        # # #  ( # # # . # )       # # . # #           ( # . # # ,  # . # # )       # . # # # #      # . # # # #  
            A l l  C C X 1 6 8  ( N = # # )                       # # #        # # #  ( # # # . # )       # # . # #           ( # . # # ,  # . # # )       # . # # # #      # . # # # #  
 
    3        P l a c e b o  +  F u l l  D o s e  P r e d n i s o n e  ( N = # # )   # # #        # # #  ( # # # . # )  
            C C X 1 6 8  +  L o w  D o s e  P r e d n i s o n e  ( N = # # )     # # #        # # #  ( # # # . # )       # # . # #           ( # . # # ,  # . # # )       # . # # # #      # . # # # #  
            C C X 1 6 8  +  N o  P r e d n i s o n e  ( N = # # )           # # #        # # #  ( # # # . # )       # # . # #           ( # . # # ,  # . # # )       # . # # # #      # . # # # #  
            A l l  C C X 1 6 8  ( N = # # )                       # # #        # # #  ( # # # . # )       # # . # #           ( # . # # ,  # . # # )       # . # # # #      # . # # # #  
 
    1 + 2      P l a c e b o  +  F u l l  D o s e  P r e d n i s o n e  ( N = # # )   # # #        # # #  ( # # # . # )  
            C C X 1 6 8  +  L o w  D o s e  P r e d n i s o n e  ( N = # # )     # # #        # # #  ( # # # . # )       # # . # #           ( # . # # ,  # . # # )       # . # # # #      # . # # # #  
            C C X 1 6 8  +  N o  P r e d n i s o n e  ( N = # # )           # # #        # # #  ( # # # . # )       # # . # #           ( # . # # ,  # . # # )       # . # # # #      # . # # # #  
            A l l  C C X 1 6 8  ( N = # # )                       # # #        # # #  ( # # # . # )       # # . # #           ( # . # # ,  # . # # )       # . # # # #      # . # # # #  
 
    2        P l a c e b o  +  F u l l  D o s e  P r e d n i s o n e  ( N = # # )   # # #        # # #  ( # # # . # )  
            C C X 1 6 8  +  N o  P r e d n i s o n e  ( N = # # )           # # #        # # #  ( # # # . # )  
 
    1        P l a c e b o  +  F u l l  D o s e  P r e d n i s o n e  ( N = # # )   # # #        # # #  ( # # # . # )  
            C C X 1 6 8  +  L o w  D o s e  P r e d n i s o n e  ( N = # # )     # # #        # # #  ( # # # . # )  
 
R e p e a t  f o r  D a y  1 6 9  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
-  R e n a l  r e s p o n s e  a t  D a y  8 5  i s  d e f i n e d  a s  i m p r o v e m e n t  i n  t h e  f o l l o w i n g  p a r a m e t e r s  o f  r e n a l  v a s c u l i t i s :  ( 1 )  i n c r e a s e  f r o m   
  b a s e l i n e  t o  D a y  8 5  i n  e G F R  ( M D R D  e q u a t i o n ) ,  ( 2 )  d e c r e a s e  f r o m  b a s e l i n e  t o  D a y  8 5  i n  h e m a t u r i a  ( m i c r o s c o p i c  c o u n t  o f  u r i n a r y  R B C s ) ,  
  a n d  d e c r e a s e  f r o m  b a s e l i n e  t o  D a y  8 5  i n  a l b u m i n u r i a  ( f i r s t  m o r n i n g  u r i n a r y  a l b u m i n :  c r e a t i n i n e  r a t i o ) .  
 
P r o g r a m  N a m e :  X X X X X X X X . s a s                   R u n  D a t e :  D D M M M Y Y Y Y  H H : M M                                  D a t a b a s e  l a s t  m o d i f i e d :  D D M M Y Y Y Y  


 
P a ge 3 0  of 2 0 8 
 
T he f oll o wi n g t a bles w ill ha ve t he sa me l a y o ut as Ta ble 1 4. 2. 2. 1: 
T a b l e  1 4 . 2 . 2 . 2  
A n a l y s i s  o f  R e n a l  R e s p o n s e  f o r  P a t i e n t s  w i t h  H e m a t u r i a  a n d  A l b u m i n u r i a  a t  B a s e l i n e  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  C y c l o p h o s p h a m i d e  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 2 . 3  
A n a l y s i s  o f  R e n a l  R e s p o n s e  f o r  P a t i e n t s  w i t h  H e m a t u r i a  a n d  A l b u m i n u r i a  a t  B a s e l i n e  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  R i t u x i m a b  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 2 . 4  
A n a l y s i s  o f  R e n a l  R e s p o n s e  f o r  P a t i e n t s  w i t h  H e m a t u r i a  a n d  A l b u m i n u r i a  a t  B a s e l i n e  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N e w l y  D i a g n o s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 2 . 5  
A n a l y s i s  o f  R e n a l  R e s p o n s e  f o r  P a t i e n t s  w i t h  H e m a t u r i a  a n d  A l b u m i n u r i a  a t  B a s e l i n e  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e l a p s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 2 . 6  
A n a l y s i s  o f  R e n a l  R e s p o n s e  f o r  P a t i e n t s  w i t h  H e m a t u r i a  a n d  A l b u m i n u r i a  a t  B a s e l i n e  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M P O +  D i s e a s e  
 
T a b l e  1 4 . 2 . 2 . 7  
A n a l y s i s  o f  R e n a l  R e s p o n s e  f o r  P a t i e n t s  w i t h  H e m a t u r i a  a n d  A l b u m i n u r i a  a t  B a s e l i n e  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  P R 3 +  D i s e a s e  
 
T a b l e  1 4 . 2 . 2 . 8  
A n a l y s i s  o f  R e n a l  R e s p o n s e  f o r  P a t i e n t s  w i t h  H e m a t u r i a  a n d  A l b u m i n u r i a  a t  B a s e l i n e  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  G r a n u l o m a t o s i s  w i t h  P o l y a n g i i t i s  ( W e g e n e r ’ s )  
 
T a b l e  1 4 . 2 . 2 . 9  
A n a l y s i s  o f  R e n a l  R e s p o n s e  f o r  P a t i e n t s  w i t h  H e m a t u r i a  a n d  A l b u m i n u r i a  a t  B a s e l i n e  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M i c r o s c o p i c  P o l y a n g i i t i s  


 
P a ge 3 1  of 2 0 8 
 
 
C h e m o C e n t r y x ,  I n c .  
P r o t o c o l  C L 0 0 2 _ 1 6 8                                                                                                          P a g e  #  o f  #  
T a b l e  1 4 . 2 . 3 . 1  
A n a l y s i s  o f  B V A S  D i s e a s e  R e m i s s i o n  
I n t e n t - t o - T r e a t  P o p u l a t i o n  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
                                                                             D i f f e r e n c e                          
  D a y                                                                       i n  p e r c e n t a g e s    T w o - s i d e d  9 0 %  C I   N o n - i n f e r .   S u p e r i o r .  
   S t e p      T r e a t m e n t                                N          n     ( % )      v e r s u s  P l a c e b o     f o r  D i f f e r e n c e     P - v a l u e     P - v a l u e  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
  D a y  8 5  
   
    A l l      P l a c e b o  +  F u l l  D o s e  P r e d n i s o n e  ( N = # # )   # # #        # # #  ( # # # . # )  
            C C X 1 6 8  +  L o w  D o s e  P r e d n i s o n e  ( N = # # )     # # #        # # #  ( # # # . # )       # # . # #           ( # . # # ,  # . # # )       # . # # # #      # . # # # #  
            C C X 1 6 8  +  N o  P r e d n i s o n e  ( N = # # )           # # #        # # #  ( # # # . # )       # # . # #           ( # . # # ,  # . # # )       # . # # # #      # . # # # #  
            A l l  C C X 1 6 8  ( N = # # )                       # # #        # # #  ( # # # . # )       # # . # #           ( # . # # ,  # . # # )       # . # # # #      # . # # # #  
 
    3        P l a c e b o  +  F u l l  D o s e  P r e d n i s o n e  ( N = # # )   # # #        # # #  ( # # # . # )  
            C C X 1 6 8  +  L o w  D o s e  P r e d n i s o n e  ( N = # # )     # # #        # # #  ( # # # . # )       # # . # #           ( # . # # ,  # . # # )       # . # # # #      # . # # # #  
            C C X 1 6 8  +  N o  P r e d n i s o n e  ( N = # # )           # # #        # # #  ( # # # . # )       # # . # #           ( # . # # ,  # . # # )       # . # # # #      # . # # # #  
            A l l  C C X 1 6 8  ( N = # # )                       # # #        # # #  ( # # # . # )       # # . # #           ( # . # # ,  # . # # )       # . # # # #      # . # # # #  
 
    1 + 2      P l a c e b o  +  F u l l  D o s e  P r e d n i s o n e  ( N = # # )   # # #        # # #  ( # # # . # )  
            C C X 1 6 8  +  L o w  D o s e  P r e d n i s o n e  ( N = # # )     # # #        # # #  ( # # # . # )       # # . # #           ( # . # # ,  # . # # )       # . # # # #      # . # # # #  
            C C X 1 6 8  +  N o  P r e d n i s o n e  ( N = # # )           # # #        # # #  ( # # # . # )       # # . # #           ( # . # # ,  # . # # )       # . # # # #      # . # # # #  
            A l l  C C X 1 6 8  ( N = # # )                       # # #        # # #  ( # # # . # )       # # . # #           ( # . # # ,  # . # # )       # . # # # #      # . # # # #  
 
    2        P l a c e b o  +  F u l l  D o s e  P r e d n i s o n e  ( N = # # )   # # #        # # #  ( # # # . # )  
            C C X 1 6 8  +  N o  P r e d n i s o n e  ( N = # # )           # # #        # # #  ( # # # . # )  
 
    1        P l a c e b o  +  F u l l  D o s e  P r e d n i s o n e  ( N = # # )   # # #        # # #  ( # # # . # )  
            C C X 1 6 8  +  L o w  D o s e  P r e d n i s o n e  ( N = # # )     # # #        # # #  ( # # # . # )  
 
R e p e a t  f o r  D a y  1 6 9  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
-  D i s e a s e  r e m i s s i o n  i s  d e f i n e d  a s  a c h i e v i n g  a  B V A S  s c o r e  o f  0  o r  1  p l u s  n o  w o r s e n i n g  i n  e G F R  a n d  u r i n a r y  R B C  < 1 0 / h p f .  
 
P r o g r a m  N a m e :  X X X X X X X X . s a s                   R u n  D a t e :  D D M M M Y Y Y Y  H H : M M                                  D a t a b a s e  l a s t  m o d i f i e d :  D D M M Y Y Y Y  


 
P a ge 3 2  of 2 0 8 
 
T he f oll o wi n g t a bles w ill ha ve t he a m e la y o ut as Ta ble 1 4. 2. 3. 1: 
T a b l e  1 4 . 2 . 3 . 2  
A n a l y s i s  o f  B V A S  D i s e a s e  R e m i s s i o n  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 3 . 3  
A n a l y s i s  o f  B V A S  D i s e a s e  R e m i s s i o n  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N o n - R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 3 . 4  
A n a l y s i s  o f  B V A S  D i s e a s e  R e m i s s i o n  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  C y c l o p h o s p h a m i d e  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 3 . 5  
A n a l y s i s  o f  B V A S  D i s e a s e  R e m i s s i o n  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  R i t u x i m a b  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 3 . 6  
A n a l y s i s  o f  B V A S  D i s e a s e  R e m i s s i o n  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N e w l y  D i a g n o s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 3 . 7  
A n a l y s i s  o f  B V A S  D i s e a s e  R e m i s s i o n  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e l a p s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 3 . 8  
A n a l y s i s  o f  B V A S  D i s e a s e  R e m i s s i o n  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M P O +  D i s e a s e  
 
T a b l e  1 4 . 2 . 3 . 9  
A n a l y s i s  o f  B V A S  D i s e a s e  R e m i s s i o n  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  P R 3 +  D i s e a s e  
 
T a b l e  1 4 . 2 . 3 . 1 0  
A n a l y s i s  o f  B V A S  D i s e a s e  R e m i s s i o n  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  G r a n u l o m a t o s i s  w i t h  P o l y a n g i i t i s  ( W e g e n e r ’ s )  
 
T a b l e  1 4 . 2 . 3 . 1 1  
A n a l y s i s  o f  B V A S  D i s e a s e  R e m i s s i o n  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M i c r o s c o p i c  P o l y a n g i i t i s  


 
P a ge 3 3  of 2 0 8 
 
C h e m o C e n t r y x ,  I n c .  
P r o t o c o l  C L 0 0 2 _ 1 6 8                                                                                                          P a g e  1  o f  2  
T a b l e  1 4 . 2 . 4 . 1 . 1  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
                         P l a c e b o  +                       C C X 1 6 8  +                       C C X 1 6 8  +  
                    F u l l  D o s e  P r e d n i s o n e           L o w  D o s e  P r e d n i s o n e               N o  P r e d n i s o n e                    A l l  C C X 1 6 8  
                           ( N = # # )                         ( N = # # )                         ( N = # # )                          ( N = # # )  
   S t u d y  D a y       - - - - - - - - - - - - - - - - - - - - - - - -       - - - - - - - - - - - - - - - - - - - - - - - -     - - - - - - - - - - - - - - - - - - - - - - - -       - - - - - - - - - - - - - - - - - - - - - - - - -  
    S t a t i s t i c        V i s i t       %  C h a n g e            V i s i t       %  C h a n g e            V i s i t       %  C h a n g e             V i s i t       %  C h a n g e  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
   B a s e l i n e  
    N ’               # #  
    M e a n             # # . #  
    S D               # # . # #  
    M i n i m u m          # #  
    M e d i a n           # # . #  
    M a x i m u m          # #  
 
   D a y  2 9  
    N ’               # #             # #               # #             # #               # #             # #                # #             # #  
    M e a n             # # . #           # # . #             # # . #           # # . #             # # . #           # # . #              # # . #           # # . #  
    S D               # # . # #          # # . # #            # # . # #          # # . # #            # # . # #          # # . # #             # # . # #          # # . # #  
    M i n i m u m          # #             # #               # #             # #               # #             # #                # #             # #  
    M e d i a n           # # . #           # # . #             # # . #           # # . #             # # . #           # # . #              # # . #           # # . #  
    M a x i m u m          # #             # #               # #             # #               # #             # #                # #             # #   
    P - v a l u e *                                                   # . # # # #                         # . # # # #                          # . # # # #  
    9 5 %  C I *                                                ( - # # . # ,  # # . # )                  ( - # # . # ,  # # . # )                   ( - # # . # ,  # # . # )   
 
   D a y  8 5  
    N ’               # #             # #               # #             # #               # #             # #                # #             # #  
    M e a n             # # . #           # # . #             # # . #           # # . #             # # . #           # # . #              # # . #           # # . #  
    S D               # # . # #          # # . # #            # # . # #          # # . # #            # # . # #          # # . # #             # # . # #          # # . # #  
    M i n i m u m          # #             # #               # #             # #               # #             # #                # #             # #  
    M e d i a n           # # . #           # # . #             # # . #           # # . #             # # . #           # # . #              # # . #           # # . #  
    M a x i m u m          # #             # #               # #             # #               # #             # #                # #             # #   
    P - v a l u e *                                                   # . # # # #                         # . # # # #                          # . # # # #  
    9 5 %  C I *                                                ( - # # . # ,  # # . # )                  ( - # # . # ,  # # . # )                   ( - # # . # ,  # # . # )   
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
-  B a s e l i n e  i s  d e f i n e d  a s  t h e  l a s t  p r e - d o s e  v a l u e .  
-  N ' = n u m b e r  o f  s u b j e c t s  w i t h  d a t a  a t  b a s e l i n e  a n d  t h e  s p e c i f i e d  v i s i t .  
* P - v a l u e s  a n d  9 5 %  c o n f i d e n c e  i n t e r v a l s  f o r  d i f f e r e n c e s  b e t w e e n  s p e c i f i e d  t r e a t m e n t  g r o u p s  a n d  p l a c e b o  a r e  f r o m  a  M M R M  m o d e l  w i t h  
 t r e a t m e n t  g r o u p ,  v i s i t ,  t r e a t m e n t - b y  v i s i t  i n t e r a c t i o n ,  A A V  d i s e a s e  s t a t u s  ( n e w  o r  r e l a p s e d ) ,  a n d  A N C A  p o s i t i v i t y  ( M P O  o r  P R 3 )  a s    
 f a c t o r s  a n d  t h e  b a s e l i n e  v a l u e  a s  a  c o v a r i a t e .   
 
P r o g r a m  N a m e :  X X X X X X X X . s a s                   R u n  D a t e :  D D M M M Y Y Y Y  H H : M M                            D a t a b a s e  l a s t  m o d i f i e d :  D D M M Y Y Y Y  H H : M M  


 
P a ge 3 4  of 2 0 8 
 
 
C h e m o C e n t r y x ,  I n c .  
P r o t o c o l  C L 0 0 2 _ 1 6 8                                                                                                          P a g e  2  o f  2  
T a b l e  1 4 . 2 . 4 . 1 . 1  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
                         P l a c e b o  +                       C C X 1 6 8  +                       C C X 1 6 8  +  
                    F u l l  D o s e  P r e d n i s o n e           L o w  D o s e  P r e d n i s o n e               N o  P r e d n i s o n e                    A l l  C C X 1 6 8  
                           ( N = # # )                         ( N = # # )                         ( N = # # )                          ( N = # # )  
   S t u d y  D a y       - - - - - - - - - - - - - - - - - - - - - - - -       - - - - - - - - - - - - - - - - - - - - - - - -     - - - - - - - - - - - - - - - - - - - - - - - -       - - - - - - - - - - - - - - - - - - - - - - - - -  
    S t a t i s t i c        V i s i t       %  C h a n g e            V i s i t       %  C h a n g e            V i s i t       %  C h a n g e             V i s i t       %  C h a n g e  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
  
   O v e r a l l  
    N ’               # #             # #               # #             # #               # #             # #                # #             # #  
    M e a n             # # . #           # # . #             # # . #           # # . #             # # . #           # # . #              # # . #           # # . #  
    S D               # # . # #          # # . # #            # # . # #          # # . # #            # # . # #          # # . # #             # # . # #          # # . # #  
    M i n i m u m          # #             # #               # #             # #               # #             # #                # #             # #  
    M e d i a n           # # . #           # # . #             # # . #           # # . #             # # . #           # # . #              # # . #           # # . #  
    M a x i m u m          # #             # #               # #             # #               # #             # #                # #             # #   
    P - v a l u e *                                                   # . # # # #                         # . # # # #                          # . # # # #  
    9 5 %  C I *                                                ( - # # . # ,  # # . # )                  ( - # # . # ,  # # . # )                   ( - # # . # ,  # # . # )   
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
-  B a s e l i n e  i s  d e f i n e d  a s  t h e  l a s t  p r e - d o s e  v a l u e .  
-  N ' = n u m b e r  o f  s u b j e c t s  w i t h  d a t a  a t  b a s e l i n e  a n d  t h e  s p e c i f i e d  v i s i t .  
* P - v a l u e s  a n d  9 5 %  c o n f i d e n c e  i n t e r v a l s  f o r  d i f f e r e n c e s  b e t w e e n  s p e c i f i e d  t r e a t m e n t  g r o u p s  a n d  p l a c e b o  a r e  f r o m  a  M M R M  m o d e l  w i t h  
 t r e a t m e n t  g r o u p ,  v i s i t ,  t r e a t m e n t - b y  v i s i t  i n t e r a c t i o n ,  A A V  d i s e a s e  s t a t u s  ( n e w  o r  r e l a p s e d ) ,  a n d  A N C A  p o s i t i v i t y  ( M P O  o r  P R 3 )  a s    
 f a c t o r s  a n d  t h e  b a s e l i n e  v a l u e  a s  a  c o v a r i a t e .   
 
P r o g r a m  N a m e :  X X X X X X X X . s a s                   R u n  D a t e :  D D M M M Y Y Y Y  H H : M M                            D a t a b a s e  l a s t  m o d i f i e d :  D D M M Y Y Y Y  H H : M M  
 
 
N ote t o pr o gra m mer:  F or s u m mar y st atistics i n t he ‘ O verall’ cate g or y, first calc ul ate t he a vera ge val ue f or a s u bject t he n c o m bi ne 
s u bjects t o o btai n s u m mar y statistics. 


 
P a ge 3 5  of 2 0 8 
 
T he f oll o wi n g t a bles w ill ha ve t he a si milar la y o ut as Ta ble 1 4. 2. 4. 1. 1: 
T a b l e  1 4 . 2 . 4 . 1 . 2  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 4 . 1 . 3  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N o n - R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 4 . 1 . 4  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  C y c l o p h o s p h a m i d e  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 4 . 1 . 5  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  R i t u x i m a b  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 4 . 1 . 6  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N e w l y  D i a g n o s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 4 . 1 . 7  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e l a p s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 4 . 1 . 8  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M P O +  D i s e a s e  
 
T a b l e  1 4 . 2 . 4 . 1 . 9  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  P R 3 +  D i s e a s e  
 
T a b l e  1 4 . 2 . 4 . 1 . 1 0  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  G r a n u l o m a t o s i s  w i t h  P o l y a n g i i t i s  ( W e g e n e r ’ s )  
 
T a b l e  1 4 . 2 . 4 . 1 . 1 1  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M i c r o s c o p i c  P o l y a n g i i t i s  


 
P a ge 3 6  of 2 0 8 
 
T a b l e  1 4 . 2 . 4 . 1 . 1 2  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  
 
T a b l e  1 4 . 2 . 4 . 1 . 1 3  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 4 . 1 . 1 4  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N o n - R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 4 . 1 . 1 5  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  C y c l o p h o s p h a m i d e  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 4 . 1 . 1 6  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  R i t u x i m a b  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 4 . 1 . 1 7  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N e w l y  D i a g n o s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 4 . 1 . 1 8  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e l a p s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 4 . 1 . 1 9  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M P O +  D i s e a s e  
 
T a b l e  1 4 . 2 . 4 . 1 . 2 0  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  P R 3 +  D i s e a s e  
 
T a b l e  1 4 . 2 . 4 . 1 . 2 1  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  G r a n u l o m a t o s i s  w i t h  P o l y a n g i i t i s  ( W e g e n e r ’ s )  


 
P a ge 3 7  of 2 0 8 
 
T a b l e  1 4 . 2 . 4 . 1 . 2 2  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M i c r o s c o p i c  P o l y a n g i i t i s  
 
N ote t o pr o gra m mer:  T h e M M R M ta bles f or t h e 1 6 8- da y st u d y peri o d will i ncl u de s u m maries f or eac h p ost- b aseli ne visit a n d t he p-
val ue a n d 9 5 % c o nfi de n ce i nter val f or t he o verall differe n ces bet wee n eac h gr o u p a n d place b o.  T his c o m me nt a p plies t o all M M R M 
ta bles f or t he 1 6 8- d a y st u d y peri o d f or Ste ps 1 t hr o u g h 3 c o m bi ne d. 
 
T he ta bles bel o w will ha ve t he sa me la y o ut as Ta bles 1 4. 2. 4. 1. 1 a n d 1 4. 2. 4. 1. 1 2: 
T a b l e  1 4 . 2 . 4 . 1 . 2 3  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  B V A S  R e n a l  S u b - s c o r e  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  
 
T a b l e  1 4 . 2 . 4 . 1 . 2 4  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  B V A S  R e n a l  S u b - s c o r e  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  
 
T a b l e  1 4 . 2 . 4 . 1 . 2 5  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  B V A S  N o n - r e n a l  S u b - s c o r e  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  
 
T a b l e  1 4 . 2 . 4 . 1 . 2 6  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  B V A S  N o n - r e n a l  S u b - s c o r e  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  


 
P a ge 3 8  of 2 0 8 
 
C h e m o C e n t r y x ,  I n c .  
P r o t o c o l  C L 0 0 2 _ 1 6 8                                                                                                          P a g e  1  o f  #  
T a b l e  1 4 . 2 . 4 . 1 . 2 7  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
                        P l a c e b o  +                      C C X 1 6 8  +                      C C X 1 6 8  +  
                  F u l l  D o s e  P r e d n i s o n e             L o w  D o s e  P r e d n i s o n e            N o  P r e d n i s o n e                     A l l  C C X 1 6 8  
                         ( N = # # )                         ( N = # # )                        ( N = # # )                          ( N = # # )  
   S t u d y  D a y       - - - - - - - - - - - - - - - - - - - - - - - -       - - - - - - - - - - - - - - - - - - - - - - - -     - - - - - - - - - - - - - - - - - - - - - - - -       - - - - - - - - - - - - - - - - - - - - - - - - -  
    S t a t i s t i c        V i s i t       %  C h a n g e            V i s i t       %  C h a n g e            V i s i t       %  C h a n g e             V i s i t       %  C h a n g e  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
   B a s e l i n e  
    N ’               # #  
    M e a n             # # . #  
    S D               # # . # #  
    M i n i m u m          # #  
    M e d i a n           # # . #  
    M a x i m u m          # #  
 
   D a y  2 9  
    N ’               # #             # #               # #             # #               # #             # #                # #             # #  
    M e a n             # # . #           # # . #             # # . #           # # . #             # # . #           # # . #              # # . #           # # . #  
    S D               # # . # #          # # . # #            # # . # #          # # . # #            # # . # #          # # . # #             # # . # #          # # . # #  
    M i n i m u m          # #             # #               # #             # #               # #             # #                # #             # #  
    M e d i a n           # # . #           # # . #             # # . #           # # . #             # # . #           # # . #              # # . #           # # . #  
    M a x i m u m          # #             # #               # #             # #               # #             # #                # #             # #   
    P - v a l u e *                                                   # . # # # #                         # . # # # #                          # . # # # #  
    9 5 %  C I *                                                ( - # # . # ,  # # . # )                  ( - # # . # ,  # # . # )                   ( - # # . # ,  # # . # )   
 
  T h e  a d d i t i o n a l  v i s i t s  t o  b e  s u m m a r i z e d  i n c l u d e :  
     D a y  8 5 ,  E n d  o f  T r e a t m e n t ,  D a y  1 1 3 ,  D a y  1 6 9 ,  E n d  o f  F o l l o w - u p  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
-  B a s e l i n e  i s  d e f i n e d  a s  t h e  l a s t  p r e - d o s e  v a l u e .  
-  N ' = n u m b e r  o f  s u b j e c t s  w i t h  d a t a  a t  b a s e l i n e  a n d  t h e  s p e c i f i e d  v i s i t .  
-  T h e  E n d  o f  T r e a t m e n t  v a l u e  i s  t h e  l a s t  m e a s u r e m e n t  t h r o u g h  D a y  8 5 .  
-  T h e  E n d  o f  F o l l o w - u p  P e r i o d  i s  t h e  l a s t  m e a s u r e m e n t  a f t e r  D a y  8 5  t h r o u g h  D a y  1 6 9 .  
* P - v a l u e s  a n d  9 5 %  c o n f i d e n c e  i n t e r v a l s  f o r  d i f f e r e n c e s  b e t w e e n  s p e c i f i e d  t r e a t m e n t  g r o u p s  a n d  p l a c e b o  a r e  f r o m  A N C O V A  m o d e l s  w i t h  
 t r e a t m e n t  g r o u p ,  A A V  d i s e a s e  s t a t u s  ( n e w  o r  r e l a p s e d )  a n d  A N C A  p o s i t i v i t y  ( M P O  o r  P R 3 ) a s  f a c t o r s  a n d  t h e  b a s e l i n e  
 v a l u e  a s  a  c o v a r i a t e .   
 
P r o g r a m  N a m e :  X X X X X X X X . s a s                   R u n  D a t e :  D D M M M Y Y Y Y  H H : M M                            D a t a b a s e  l a s t  m o d i f i e d :  D D M M Y Y Y Y  H H : M M  


 
P a ge 3 9  of 2 0 8 
 
T he f oll o wi n g t a bles w ill ha ve t he sa me l a y o ut as Ta ble 1 4. 2. 4. 1. 2 7: 
T a b l e  1 4 . 2 . 4 . 1 . 2 8  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 4 . 1 . 2 9  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N o n - R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 4 . 1 . 3 0  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  C y c l o p h o s p h a m i d e  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 4 . 1 . 3 1  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  R i t u x i m a b  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 4 . 1 . 3 2  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N e w l y  D i a g n o s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 4 . 1 . 3 3  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e l a p s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 4 . 1 . 3 4  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M P O +  D i s e a s e  
 
T a b l e  1 4 . 2 . 4 . 1 . 3 5  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  P R 3 +  D i s e a s e  
 
T a b l e  1 4 . 2 . 4 . 1 . 3 6  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  G r a n u l o m a t o s i s  w i t h  P o l y a n g i i t i s  ( W e g e n e r ’ s )  
 
T a b l e  1 4 . 2 . 4 . 1 . 3 7  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M i c r o s c o p i c  P o l y a n g i i t i s  


 
P a ge 4 0  of 2 0 8 
 
C h e m o C e n t r y x ,  I n c .  
P r o t o c o l  C L 0 0 2 _ 1 6 8                                                                                                          P a g e  1  o f  #  
T a b l e  1 4 . 2 . 4 . 2 . 1  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
                  = = = = = = = = = = = = = = =  S t e p  1  a n d  2  C o m b i n e d  = = = = = = = = = = = = = = =        = = = = = = = = = = = = = = = = = = = = = = =  S t e p  3  = = = = = = = = = = = = = = = = = = = = = = =  
                     P l a c e b o  +            C C X 1 6 8  +                                  P l a c e b o  +          C C X 1 6 8  +  
                     F u l l  D o s e            L o w  D o s e             C C X 1 6 8  +              F u l l  D o s e          L o w  D o s e             C C X 1 6 8  +   
                     P r e d n i s o n e          P r e d n i s o n e         N o  P r e d n i s o n e            P r e d n i s o n e        P r e d n i s o n e         N o  P r e d n i s o n e  
                       ( N = # # )             ( N = # # )               ( N = # # )                 ( N = # # )             ( N = # # )               ( N = # # )  
   S t u d y  D a y       - - - - - - - - - - - - - - - -    - - - - - - - - - - - - - - - -    - - - - - - - - - - - - - - - -       - - - - - - - - - - - - - - - -    - - - - - - - - - - - - - - - -    - - - - - - - - - - - - - - - -  
    S t a t i s t i c      V i s i t    %  C h a n g e    V i s i t    %  C h a n g e    V i s i t    %  C h a n g e       V i s i t    %  C h a n g e    V i s i t    %  C h a n g e    V i s i t    %  C h a n g e  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
   B a s e l i n e  
    N ’              # #  
    M e a n            # # . #  
    S D              # # . # #  
    M i n i m u m         # #  
    M e d i a n          # # . #  
    M a x i m u m         # #  
 
   D a y  2 9  
    N ’              # #        # #        # #          # #        # #        # #             # #        # #        # #          # #        # #        # #  
    M e a n            # # . #      # # . #      # # . #        # # . #      # # . #      # # . #           # # . #      # # . #      # # . #        # # . #      # # . #      # # . #  
    S D              # # . # #     # # . # #     # # . # #       # # . # #     # # . # #     # # . # #          # # . # #     # # . # #     # # . # #       # # . # #     # # . # #     # # . # #  
    M i n i m u m         # #        # #        # #          # #        # #        # #             # #        # #        # #          # #        # #        # #  
    M e d i a n          # # . #      # # . #      # # . #        # # . #      # # . #      # # . #           # # . #      # # . #      # # . #        # # . #      # # . #      # # . #  
    M a x i m u m         # #        # #        # #          # #        # #        # #             # #        # #        # #          # #        # #        # #  
    P - v a l u e *                                   # . # # # #             # . # # # #                                      # . # # # #             # . # # # #  
    9 5 %  C I *                                ( - # # . # ,  # # . # )      ( - # # . # ,  # # . # )                               ( - # # . # ,  # # . # )      ( - # # . # ,  # # . # )  
 
 
  D a y  8 5  w i l l  a l s o  b e  s u m m a r i z e d .   I n  a d d i t i o n ,  t h e  p - v a l u e  a n d  9 5 %  c o n f i d e n c e  i n t e r v a l  f o r  t h e  o v e r a l l  d i f f e r e n c e s   
    b e t w e e n  e a c h  a c t i v e  g r o u p  a n d  p l a c e b o  w i l l  b e  d i s p l a y e d .  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
-  B a s e l i n e  i s  d e f i n e d  a s  t h e  l a s t  p r e - d o s e  v a l u e .  
-  N ' = n u m b e r  o f  s u b j e c t s  w i t h  d a t a  a t  b a s e l i n e  a n d  t h e  s p e c i f i e d  v i s i t .  
* P - v a l u e s  a n d  9 5 %  c o n f i d e n c e  i n t e r v a l s  f o r  d i f f e r e n c e s  b e t w e e n  s p e c i f i e d  t r e a t m e n t  g r o u p s  a n d  p l a c e b o  a r e  f r o m  M M R M  m o d e l s  w i t h  
  t r e a t m e n t  g r o u p ,  v i s i t ,  a n d  t r e a t m e n t - b y - v i s i t  a s  f a c t o r s  a n d  t h e  b a s e l i n e  v a l u e  a s  a  c o v a r i a t e .  T h e  m o d e l  f o r  a n a l y s i s  o f  S t e p  3   
  i n c l u d e d  f a c t o r s  f o r  A A V  d i s e a s e  s t a t u s  ( n e w  o r  r e l a p s e d ) ,  A N C A  p o s i t i v i t y  ( M P O  o r  P R 3 ) ,  a n d  s t a n d a r d  o f  c a r e  t r e a t m e n t  ( r i t u x i m a b   
  o r   c y c l o p h o s p h a m i d e ) .   T h e  m o d e l  f o r  S t e p  1  +  S t e p  2  i n c l u d e d  a  f a c t o r  f o r  d i s e a s e  s t a t u s  a n d  A N C A  p o s i t i v i t y .  
 
P r o g r a m  N a m e :  X X X X X X X X . s a s                   R u n  D a t e :  D D M M M Y Y Y Y  H H : M M                            D a t a b a s e  l a s t  m o d i f i e d :  D D M M Y Y Y Y  H H : M M  


 
P a ge 4 1  of 2 0 8 
 
T he f oll o wi n g t a bles w ill ha ve la y o ut si milar t o Ta ble 1 4. 2. 4. 2. 1: 
T a b l e  1 4 . 2 . 4 . 2 . 2  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d   
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 4 . 2 . 3  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d   
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N o n - R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 4 . 2 . 4  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d   
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  C y c l o p h o s p h a m i d e  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 4 . 2 . 5  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d   
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  R i t u x i m a b  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 4 . 2 . 6  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d   
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N e w l y  D i a g n o s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 4 . 2 . 7  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d   
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e l a p s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 4 . 2 . 8  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d   
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M P O +  D i s e a s e  
 
T a b l e  1 4 . 2 . 4 . 2 . 9  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d   
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  P R 3 +  D i s e a s e  
 
T a b l e  1 4 . 2 . 4 . 2 . 1 0  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d   
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  G r a n u l o m a t o s i s  w i t h  P o l y a n g i i t i s  ( W e g e n e r ’ s )  
 
T a b l e  1 4 . 2 . 4 . 2 . 1 1  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d   
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M i c r o s c o p i c  P o l y a n g i i t i s  


 
P a ge 4 2  of 2 0 8 
 
T a b l e  1 4 . 2 . 4 . 2 . 1 2  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d   
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  
 
T a b l e  1 4 . 2 . 4 . 2 . 1 3  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d   
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 4 . 2 . 1 4  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d   
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N o n - R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 4 . 2 . 1 5  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d   
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  C y c l o p h o s p h a m i d e  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 4 . 2 . 1 6  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d   
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  R i t u x i m a b  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 4 . 2 . 1 7  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d   
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N e w l y  D i a g n o s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 4 . 2 . 1 8  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d   
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e l a p s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 4 . 2 . 1 9  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d   
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M P O +  D i s e a s e  
 
T a b l e  1 4 . 2 . 4 . 2 . 2 0  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d   
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  P R 3 +  D i s e a s e  
 
T a b l e  1 4 . 2 . 4 . 2 . 2 1  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d   
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  G r a n u l o m a t o s i s  w i t h  P o l y a n g i i t i s  ( W e g e n e r ’ s )  


 
P a ge 4 3  of 2 0 8 
 
T a b l e  1 4 . 2 . 4 . 2 . 2 2  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d   
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M i c r o s c o p i c  P o l y a n g i i t i s  
 
N ote t o pr o gra m mer:  T h e M M R M ta bles f or t h e 1 6 8- da y st u d y peri o d will i ncl u de s u m maries f or eac h p ost- b aseli ne visit a n d t he p-
val ue a n d 9 5 % c o nfi de n ce i nter val f or t he o verall differe n ces bet wee n eac h gr o u p a n d place b o.  T his c o m me nt a p plies t o all M M R M 
ta bles f or t he 1 6 8- d a y st u d y peri o d f or Ste ps 1 a n d 2 c o m bi ne d a n d Ste p 3. 
 
T he ta bles bel o w will ha ve t he sa me la y o ut as Ta bles 1 4. 2. 4. 2. 1 a n d 1 4. 2. 4. 2. 1 2: 
T a b l e  1 4 . 2 . 4 . 2 . 2 3  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  B V A S  R e n a l  S u b - s c o r e  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  
 
T a b l e  1 4 . 2 . 4 . 2 . 2 4  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  B V A S  R e n a l  S u b - s c o r e  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  
 
T a b l e  1 4 . 2 . 4 . 2 . 2 5  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  B V A S  N o n - r e n a l  S u b - s c o r e  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  
 
T a b l e  1 4 . 2 . 4 . 2 . 2 6  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  B V A S  N o n - r e n a l  S u b - s c o r e  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  
 


 
P a ge 4 4  of 2 0 8 
 
C h e m o C e n t r y x ,  I n c .  
P r o t o c o l  C L 0 0 2 _ 1 6 8                                                                                                          P a g e  1  o f  #  
T a b l e  1 4 . 2 . 4 . 2 . 2 7  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
                  = = = = = = = = = = = = = = =  S t e p  1  a n d  2  C o m b i n e d  = = = = = = = = = = = = = = =        = = = = = = = = = = = = = = = = = = = = = = =  S t e p  3  = = = = = = = = = = = = = = = = = = = = = = =  
                     P l a c e b o  +            C C X 1 6 8  +                                  P l a c e b o  +          C C X 1 6 8  +  
                     F u l l  D o s e            L o w  D o s e             C C X 1 6 8  +              F u l l  D o s e          L o w  D o s e             C C X 1 6 8  +   
                     P r e d n i s o n e          P r e d n i s o n e         N o  P r e d n i s o n e            P r e d n i s o n e        P r e d n i s o n e         N o  P r e d n i s o n e  
                       ( N = # # )             ( N = # # )               ( N = # # )                 ( N = # # )             ( N = # # )               ( N = # # )  
   S t u d y  D a y       - - - - - - - - - - - - - - - -    - - - - - - - - - - - - - - - -    - - - - - - - - - - - - - - - -       - - - - - - - - - - - - - - - -    - - - - - - - - - - - - - - - -    - - - - - - - - - - - - - - - -  
    S t a t i s t i c      V i s i t    %  C h a n g e    V i s i t    %  C h a n g e    V i s i t    %  C h a n g e       V i s i t    %  C h a n g e    V i s i t    %  C h a n g e    V i s i t    %  C h a n g e  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
   B a s e l i n e  
    N ’              # #  
    M e a n            # # . #  
    S D              # # . # #  
    M i n i m u m         # #  
    M e d i a n          # # . #  
    M a x i m u m         # #  
 
   D a y  2 9  
    N ’              # #        # #        # #          # #        # #        # #             # #        # #        # #          # #        # #        # #  
    M e a n            # # . #      # # . #      # # . #        # # . #      # # . #      # # . #           # # . #      # # . #      # # . #        # # . #      # # . #      # # . #  
    S D              # # . # #     # # . # #     # # . # #       # # . # #     # # . # #     # # . # #          # # . # #     # # . # #     # # . # #       # # . # #     # # . # #     # # . # #  
    M i n i m u m         # #        # #        # #          # #        # #        # #             # #        # #        # #          # #        # #        # #  
    M e d i a n          # # . #      # # . #      # # . #        # # . #      # # . #      # # . #           # # . #      # # . #      # # . #        # # . #      # # . #      # # . #  
    M a x i m u m         # #        # #        # #          # #        # #        # #             # #        # #        # #          # #        # #        # #  
    P - v a l u e *                                   # . # # # #             # . # # # #                                      # . # # # #             # . # # # #  
    9 5 %  C I *                                ( - # # . # ,  # # . # )      ( - # # . # ,  # # . # )                               ( - # # . # ,  # # . # )      ( - # # . # ,  # # . # )  
 
 
  T h e  a d d i t i o n a l  v i s i t s  t o  b e  s u m m a r i z e d  i n c l u d e :  
     D a y  8 5 ,  E n d  o f  T r e a t m e n t ,  D a y  1 1 3 ,  D a y  1 6 9 ,  E n d  o f  F o l l o w - u p  P e r i o d .  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
-  B a s e l i n e  i s  d e f i n e d  a s  t h e  l a s t  p r e - d o s e  v a l u e .  
-  N ' = n u m b e r  o f  s u b j e c t s  w i t h  d a t a  a t  b a s e l i n e  a n d  t h e  s p e c i f i e d  v i s i t .  
-  T h e  E n d  o f  T r e a t m e n t  v a l u e  i s  t h e  l a s t  m e a s u r e m e n t  t h r o u g h  D a y  8 5 .  
-  T h e  E n d  o f  F o l l o w - u p  P e r i o d  i s  t h e  l a s t  m e a s u r e m e n t  a f t e r  D a y  8 5  t h r o u g h  D a y  1 6 9 .  
* P - v a l u e s  a n d  9 5 %  c o n f i d e n c e  i n t e r v a l s  f o r  d i f f e r e n c e s  b e t w e e n  s p e c i f i e d  t r e a t m e n t  g r o u p s  a n d  p l a c e b o  a r e  f r o m  A N C O V A  m o d e l s  w i t h  
  t r e a t m e n t  g r o u p  a s  a  f a c t o r  a n d  t h e  b a s e l i n e  v a l u e  a s  a  c o v a r i a t e .  T h e  m o d e l s  f o r  S t e p  3  i n c l u d e d  f a c t o r s  A A V  d i s e a s e  s t a t u s   
 ( n e w  o r  r e l a p s e d ) ,  A N C A  p o s i t i v i t y  ( M P O  o r  P R 3 ) ,  a n d  s t a n d a r d  o f  c a r e  t r e a t m e n t  ( r i t u x i m a b  o r   c y c l o p h o s p h a m i d e ) .   T h e  m o d e l s  f o r  
 S t e p  1  +  S t e p  2  i n c l u d e d  a  f a c t o r  f o r  d i s e a s e  s t a t u s  a n d  A N C A  p o s i t i v i t y .  
 
P r o g r a m  N a m e :  X X X X X X X X . s a s                   R u n  D a t e :  D D M M M Y Y Y Y  H H : M M                            D a t a b a s e  l a s t  m o d i f i e d :  D D M M Y Y Y Y  H H : M M  


 
P a ge 4 5  of 2 0 8 
 
T he f oll o wi n g t a bles w ill ha ve a si milar la y o ut as Ta ble 1 4. 2. 4. 2. 2 7: 
T a b l e  1 4 . 2 . 4 . 2 . 2 8  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 4 . 2 . 2 9  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N o n - R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 4 . 2 . 3 0  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  C y c l o p h o s p h a m i d e  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 4 . 2 . 3 1  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  R i t u x i m a b  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 4 . 2 . 3 2  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N e w l y  D i a g n o s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 4 . 2 . 3 3  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e l a p s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 4 . 2 . 3 4  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M P O +  D i s e a s e  
 
T a b l e  1 4 . 2 . 4 . 2 . 3 5  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  P R 3 +  D i s e a s e  
 
T a b l e  1 4 . 2 . 4 . 2 . 3 6  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  G r a n u l o m a t o s i s  w i t h  P o l y a n g i i t i s  ( W e g e n e r ’ s )  
 
T a b l e  1 4 . 2 . 4 . 2 . 3 7  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M i c r o s c o p i c  P o l y a n g i i t i s  


 
P a ge 4 6  of 2 0 8 
 
C h e m o C e n t r y x ,  I n c .  
P r o t o c o l  C L 0 0 2 _ 1 6 8                                                                                                          P a g e  1  o f  #  
T a b l e  1 4 . 2 . 4 . 3 . 1  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
  S t e p                    P l a c e b o  +                      C C X 1 6 8  +                        C C X 1 6 8  +  
   S t u d y         - -  F u l l  D o s e  P r e d n i s o n e  - -      - -  L o w  D o s e  P r e d n i s o n e  - -       - - - - -  N o  P r e d n i s o n e  - - - - -       - - - - - - -  A l l  C C X 1 6 8  - - - - - -  
    S t a t i s t i c        V i s i t       %  C h a n g e            V i s i t       %  C h a n g e            V i s i t       %  C h a n g e             V i s i t       %  C h a n g e  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
  S t e p  3                   N = # #                           N = # #                           N = # #                            N = # #  
 
   B a s e l i n e  
    N ’               # #  
    M e a n             # # . #  
    S D               # # . # #  
    M i n i m u m          # #  
    M e d i a n           # # . #  
    M a x i m u m          # #  
 
   D a y  2 9  
    N ’               # #             # #               # #             # #               # #             # #                # #             # #  
    M e a n             # # . #           # # . #             # # . #           # # . #             # # . #           # # . #              # # . #           # # . #  
    S D               # # . # #          # # . # #            # # . # #          # # . # #            # # . # #          # # . # #             # # . # #          # # . # #  
    M i n i m u m          # #             # #               # #             # #               # #             # #                # #             # #  
    M e d i a n           # # . #           # # . #             # # . #           # # . #             # # . #           # # . #              # # . #           # # . #  
    M a x i m u m          # #             # #               # #             # #               # #             # #                # #             # #   
    P - v a l u e *                                                   # . # # # #                         # . # # # #                          # . # # # #  
    9 5 %  C I *                                                ( - # # . # ,  # # . # )                  ( - # # . # ,  # # . # )                   ( - # # . # ,  # # . # )   
 
   
  D a y  8 5  w i l l  a l s o  b e  s u m m a r i z e d .   I n  a d d i t i o n ,  f o r  S t e p  3 ,  t h e  p - v a l u e  a n d  9 5 %  c o n f i d e n c e  i n t e r v a l  f o r  t h e  o v e r a l l  d i f f e r e n c e s   
    b e t w e e n  e a c h  a c t i v e  g r o u p  a n d  p l a c e b o  w i l l  b e  d i s p l a y e d .  
 
  R e p e a t  f o r  S t e p s  2  a n d  1  ( s u m m a r y  s t a t i s t i c s  o n l y ) .  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
-  B a s e l i n e  i s  d e f i n e d  a s  t h e  l a s t  p r e - d o s e  v a l u e .  
-  N ' = n u m b e r  o f  s u b j e c t s  w i t h  d a t a  a t  b a s e l i n e  a n d  t h e  s p e c i f i e d  v i s i t .  
* P - v a l u e s  a n d  9 5 %  c o n f i d e n c e  i n t e r v a l s  f o r  d i f f e r e n c e s  b e t w e e n  s p e c i f i e d  t r e a t m e n t  g r o u p s  a n d  p l a c e b o  a r e  f r o m  M M R M  m o d e l s  w i t h  
  t r e a t m e n t  g r o u p ,  v i s i t ,  a n d  t r e a t m e n t - b y - v i s i t ,  A A V  d i s e a s e  s t a t u s  ( n e w  o r  r e l a p s e d ) ,  A N C A  p o s i t i v i t y  ( M P O  o r  P R 3 ) ,  a n d  s t a n d a r d  o f   
  c a r e  t r e a t m e n t  ( r i t u x i m a b  o r   c y c l o p h o s p h a m i d e )  a s  f a c t o r s  a n d  t h e  b a s e l i n e  v a l u e  a s  a  c o v a r i a t e .   
 
P r o g r a m  N a m e :  X X X X X X X X . s a s                   R u n  D a t e :  D D M M M Y Y Y Y  H H : M M                            D a t a b a s e  l a s t  m o d i f i e d :  D D M M Y Y Y Y  H H : M M  


 
P a ge 4 7  of 2 0 8 
 
T he f oll o wi n g t a bles w ill ha ve a si milar la y o ut as Ta ble 1 4. 2. 4. 3. 1: 
T a b l e  1 4 . 2 . 4 . 3 . 2  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 4 . 3 . 3  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N o n - R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 4 . 3 . 4  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  C y c l o p h o s p h a m i d e  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 4 . 3 . 5  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  R i t u x i m a b  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 4 . 3 . 6  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N e w l y  D i a g n o s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 4 . 3 . 7  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e l a p s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 4 . 3 . 8  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M P O +  D i s e a s e  
 
T a b l e  1 4 . 2 . 4 . 3 . 9  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  P R 3 +  D i s e a s e  
 
T a b l e  1 4 . 2 . 4 . 3 . 1 0  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  G r a n u l o m a t o s i s  w i t h  P o l y a n g i i t i s  ( W e g e n e r ’ s )  
 
T a b l e  1 4 . 2 . 4 . 3 . 1 1  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M i c r o s c o p i c  P o l y a n g i i t i s  


 
P a ge 4 8  of 2 0 8 
 
T a b l e  1 4 . 2 . 4 . 3 . 1 2  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  
 
T a b l e  1 4 . 2 . 4 . 3 . 1 3  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 4 . 3 . 1 4  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N o n - R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 4 . 3 . 1 5  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  C y c l o p h o s p h a m i d e  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 4 . 3 . 1 6  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  R i t u x i m a b  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 4 . 3 . 1 7  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N e w l y  D i a g n o s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 4 . 3 . 1 8  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e l a p s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 4 . 3 . 1 9  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M P O +  D i s e a s e  
 
T a b l e  1 4 . 2 . 4 . 3 . 2 0  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  P R 3 +  D i s e a s e  
 
T a b l e  1 4 . 2 . 4 . 3 . 2 1  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  G r a n u l o m a t o s i s  w i t h  P o l y a n g i i t i s  ( W e g e n e r ’ s )  
 


 
P a ge 4 9  of 2 0 8 
 
T a b l e  1 4 . 2 . 4 . 3 . 2 2  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M i c r o s c o p i c  P o l y a n g i i t i s  
 
 
N ote t o pr o gra m mer:  T h e M M R M ta bles f or t h e 1 6 8- da y st u d y peri o d will i ncl u de s u m maries f or eac h p ost- b aseli ne visit a n d t he p-
val ue a n d 9 5 % c o nfi de n ce i nter val f or t he o verall differe n ces bet w ee n eac h gr o u p a n d place b o f or Ste p 3.  T his c o m me nt a p plies t o all 
M M R M ta bles f or t he 1 6 8- da y st u d y p eri o d b y Ste p. 
 
T he ta bles bel o w will ha ve t he sa me la y o ut as Ta bles 1 4. 2. 4. 3. 1 a n d 1 4. 2. 4. 3. 1 2: 
T a b l e  1 4 . 2 . 4 . 2 . 2 3  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  B V A S  R e n a l  S u b - s c o r e  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  
 
T a b l e  1 4 . 2 . 4 . 2 . 2 4  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  B V A S  R e n a l  S u b - s c o r e  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  
 
T a b l e  1 4 . 2 . 4 . 2 . 2 5  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  B V A S  N o n - r e n a l  S u b - s c o r e  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  
 
T a b l e  1 4 . 2 . 4 . 2 . 2 6  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  B V A S  N o n - r e n a l  S u b - s c o r e  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  


 
P a ge 5 0  of 2 0 8 
 
C h e m o C e n t r y x ,  I n c .  
P r o t o c o l  C L 0 0 2 _ 1 6 8                                                                                                          P a g e  1  o f  #  
T a b l e  1 4 . 2 . 4 . 3 . 2 7  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
  S t e p                    P l a c e b o  +                      C C X 1 6 8  +                        C C X 1 6 8  +  
   S t u d y         - -  F u l l  D o s e  P r e d n i s o n e  - -      - -  L o w  D o s e  P r e d n i s o n e  - -       - - - - -  N o  P r e d n i s o n e  - - - - -       - - - - - - -  A l l  C C X 1 6 8  - - - - - -  
    S t a t i s t i c        V i s i t       %  C h a n g e            V i s i t       %  C h a n g e             V i s i t       %  C h a n g e             V i s i t       %  C h a n g e  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
  S t e p  3                   N = # #                           N = # #                           N = # #                            N = # #  
 
   B a s e l i n e  
    N ’               # #  
    M e a n             # # . #  
    S D               # # . # #  
    M i n i m u m          # #  
    M e d i a n           # # . #  
    M a x i m u m          # #  
 
   D a y  2 9  
    N ’               # #             # #               # #             # #               # #             # #                # #             # #  
    M e a n             # # . #           # # . #             # # . #           # # . #             # # . #           # # . #              # # . #           # # . #  
    S D               # # . # #          # # . # #            # # . # #          # # . # #            # # . # #          # # . # #             # # . # #          # # . # #  
    M i n i m u m          # #             # #               # #             # #               # #             # #                # #             # #  
    M e d i a n           # # . #           # # . #             # # . #           # # . #             # # . #           # # . #              # # . #           # # . #  
    M a x i m u m          # #             # #               # #             # #               # #             # #                # #             # #   
    P - v a l u e *                                                   # . # # # #                         # . # # # #                          # . # # # #  
    9 5 %  C I *                                                ( - # # . # ,  # # . # )                  ( - # # . # ,  # # . # )                   ( - # # . # ,  # # . # )   
 
  R e p e a t  f o r  S t e p s  2  a n d  1 .   O n l y  d i s p l a y  p - v a l u e s  a n d  c o n f i d e n c e  i n t e r v a l s  f o r  S t e p  3 .  
 
  T h e  a d d i t i o n a l  v i s i t s  t o  b e  s u m m a r i z e d  i n c l u d e :  
     D a y  8 5 ,  E n d  o f  T r e a t m e n t ,  D a y  1 1 3 ,  D a y  1 6 9 ,  E n d  o f  F o l l o w - u p  P e r i o d .  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
-  B a s e l i n e  i s  d e f i n e d  a s  t h e  l a s t  p r e - d o s e  v a l u e .  
-  N ' = n u m b e r  o f  s u b j e c t s  w i t h  d a t a  a t  b a s e l i n e  a n d  t h e  s p e c i f i e d  v i s i t .  
-  T h e  E n d  o f  T r e a t m e n t  v a l u e  i s  t h e  l a s t  m e a s u r e m e n t  t h r o u g h  D a y  8 5 .  
-  T h e  E n d  o f  F o l l o w - u p  P e r i o d  i s  t h e  l a s t  m e a s u r e m e n t  a f t e r  D a y  8 5  t h r o u g h  D a y  1 6 9 .  
* P - v a l u e s  a n d  9 5 %  c o n f i d e n c e  i n t e r v a l s  f o r  d i f f e r e n c e s  b e t w e e n  s p e c i f i e d  t r e a t m e n t  g r o u p s  a n d  p l a c e b o  a r e  f r o m  A N C O V A  m o d e l s  w i t h  
 t r e a t m e n t  g r o u p  a n d  r a n d o m i z a t i o n  s t r a t a  ( A A V  s t a t u s ,  A N C A  p o s i t i v i t y ,  a n d  s t a n d a r d  o f  c a r e  t r e a t m e n t )  a s  f a c t o r s  a n d  t h e  b a s e l i n e   
 v a l u e  a s  a  c o v a r i a t e .  
 
P r o g r a m  N a m e :  X X X X X X X X . s a s                   R u n  D a t e :  D D M M M Y Y Y Y  H H : M M                            D a t a b a s e  l a s t  m o d i f i e d :  D D M M Y Y Y Y  H H : M M  


 
P a ge 5 1  of 2 0 8 
 
T he f oll o wi n g t a bles w ill ha ve t he sa me l a y o ut as Ta ble 1 4. 2. 4. 3. 2 7: 
T a b l e  1 4 . 2 . 4 . 3 . 2 8  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 4 . 3 . 2 9  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N o n - R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 4 . 3 . 3 0  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  C y c l o p h o s p h a m i d e  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 4 . 3 . 3 1  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  R i t u x i m a b  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 4 . 3 2  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N e w l y  D i a g n o s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 4 . 3 . 3 3  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e l a p s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 4 . 3 . 3 4  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M P O +  D i s e a s e  
 
T a b l e  1 4 . 2 . 4 . 3 . 3 5  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  P R 3 +  D i s e a s e  
 
T a b l e  1 4 . 2 . 4 . 3 . 3 6  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  G r a n u l o m a t o s i s  w i t h  P o l y a n g i i t i s  ( W e g e n e r ’ s )  
 
T a b l e  1 4 . 2 . 4 . 3 . 3 7  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  B V A S  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M i c r o s c o p i c  P o l y a n g i i t i s  


 
P a ge 5 2  of 2 0 8 
 
C h e m o C e n t r y x ,  I n c .  
P r o t o c o l  C L 0 0 2 _ 1 6 8                                                                                                          P a g e  1  o f  #  
T a b l e  1 4 . 2 . 5 . 1 . 1  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d   
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
                           P l a c e b o  +                       C C X 1 6 8  +                     C C X 1 6 8  +  
                      F u l l  D o s e  P r e d n i s o n e            L o w  D o s e  P r e d n i s o n e            N o  P r e d n i s o n e                     A l l  C C X 1 6 8  
                             ( N = # # )                         ( N = # # )                       ( N = # # )                          ( N = # # )  
   S t u d y  D a y         - - - - - - - - - - - - - - - - - - - - - - - -       - - - - - - - - - - - - - - - - - - - - - - - -     - - - - - - - - - - - - - - - - - - - - - - - -       - - - - - - - - - - - - - - - - - - - - - - - - -  
    S t a t i s t i c        V i s i t     C h a n g e   %  C h a n g e      V i s i t     C h a n g e   %  C h a n g e    V i s i t     C h a n g e   %  C h a n g e       V i s i t     C h a n g e   %  C h a n g e        
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
   B a s e l i n e   
    N ’               # #  
    M e a n             # # . # #  
    S D               # # . # # #  
    M i n i m u m          # # . #  
    M e d i a n           # # . # #  
    M a x i m u m          # # . #  
 
   D a y  2  
    N ’               # #        # #        # #           # #        # #        # #          # #        # #        # #           # #        # #        # #  
    M e a n             # # . # #     # # . # #     # # . # #        # # . # #     # # . # #     # # . # #       # # . # #     # # . # #     # # . # #        # # . # #     # # . # #     # # . # #  
    S D               # # . # # #    # # . # # #    # # . # # #       # # . # # #    # # . # # #    # # . # # #      # # . # # #    # # . # # #    # # . # # #       # # . # # #    # # . # # #    # # . # # #  
    M i n i m u m          # # . #      # # . #      # # . #         # # . #      # # . #      # # . #        # # . #      # # . #      # # . #         # # . #      # # . #      # # . #  
    M e d i a n           # # . # #     # # . # #     # # . # #        # # . # #     # # . # #     # # . # #       # # . # #     # # . # #     # # . # #        # # . # #     # # . # #     # # . # #  
    M a x i m u m          # # . #      # # . #      # # . #         # # . #      # # . #      # # . #        # # . #      # # . #      # # . #         # # . #      # # . #      # # . #  
    P - v a l u e *                                              # . # # # #    # . # # # #               # . # # # #    # . # # # #                # . # # # #    # . # # # #  
    9 5 %  C I  f o r  C h a n g e *                                     ( - # # . # ,  # # . # )                 ( - # # . # ,  # # . # )                  ( - # # . # ,  # # . # )  
    9 5 %  C I  f o r  %  C h a n g e *                                   ( - # # . # ,  # # . # )                 ( - # # . # ,  # # . # )                  ( - # # . # ,  # # . # )  
 
  T h e  a d d i t i o n a l  v i s i t s  t o  b e  s u m m a r i z e d  i n c l u d e :  
     D a y  8 ,  D a y  1 5 ,  D a y  2 9 ,  D a y  4 3 ,  D a y  5 7 ,  D a y  7 1 ,  D a y  8 5 ,  a n d  O v e r a l l  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
-  B a s e l i n e  i s  d e f i n e d  a s  t h e  l a s t  p r e - d o s e  v a l u e .  
-  N ' = n u m b e r  o f  s u b j e c t s  w i t h  d a t a  a t  b a s e l i n e  a n d  t h e  s p e c i f i e d  v i s i t .  
* P - v a l u e s  a n d  9 5 %  c o n f i d e n c e  i n t e r v a l s  f o r  d i f f e r e n c e s  b e t w e e n  s p e c i f i e d  t r e a t m e n t  g r o u p s  a n d  p l a c e b o  a r e  f r o m  a  M M R M  m o d e l  w i t h  
 t r e a t m e n t  g r o u p ,  v i s i t ,  t r e a t m e n t - b y  v i s i t  i n t e r a c t i o n ,  A A V  d i s e a s e  s t a t u s  ( n e w  o r  r e l a p s e d ) ,  a n d  A N C A  p o s i t i v i t y  ( M P O  o r  P R 3 )  a s    
 f a c t o r s  a n d  t h e  b a s e l i n e  v a l u e  a s  a  c o v a r i a t e .   
 
P r o g r a m  N a m e :  X X X X X X X X . s a s                   R u n  D a t e :  D D M M M Y Y Y Y  H H : M M                            D a t a b a s e  l a s t  m o d i f i e d :  D D M M Y Y Y Y  H H : M M  


 
P a ge 5 3  of 2 0 8 
 
T he f oll o wi n g t a bles w ill ha ve la y o ut si milar t o Ta ble 1 4. 2. 5. 1. 1: 
T a b l e  1 4 . 2 . 5 . 1 . 2  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 5 . 1 . 3  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N o n - R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 5 . 1 . 4  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  C y c l o p h o s p h a m i d e  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 5 . 1 . 5  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  R i t u x i m a b  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 5 . 1 . 6  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N e w l y  D i a g n o s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 5 . 1 . 7  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d   
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e l a p s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 5 . 1 . 8  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M P O +  D i s e a s e  
 
T a b l e  1 4 . 2 . 5 . 1 . 9  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  P R 3 +  D i s e a s e  
 
T a b l e  1 4 . 2 . 5 . 1 . 1 0  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  G r a n u l o m a t o s i s  w i t h  P o l y a n g i i t i s  ( W e g e n e r ’ s )  
 
T a b l e  1 4 . 2 . 5 . 1 . 1 1  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M i c r o s c o p i c  P o l y a n g i i t i s  


 
P a ge 5 4  of 2 0 8 
 
T a b l e  1 4 . 2 . 5 . 1 . 1 2  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  
 
T a b l e  1 4 . 2 . 5 . 1 . 1 3  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 5 . 1 . 1 4  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N o n - R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 5 . 1 . 1 5  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  C y c l o p h o s p h a m i d e  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 5 . 1 . 1 6  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  R i t u x i m a b  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 5 . 1 . 1 7  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N e w l y  D i a g n o s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 5 . 1 . 1 8  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e l a p s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 5 . 1 . 1 9  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M P O +  D i s e a s e  
 
T a b l e  1 4 . 2 . 5 . 1 . 2 0  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  P R 3 +  D i s e a s e  
 
T a b l e  1 4 . 2 . 5 . 1 . 2 1  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  G r a n u l o m a t o s i s  w i t h  P o l y a n g i i t i s  ( W e g e n e r ’ s )  
 


 
P a ge 5 5  of 2 0 8 
 
T a b l e  1 4 . 2 . 5 . 1 . 2 2  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M i c r o s c o p i c  P o l y a n g i i t i s  


 
P a ge 5 6  of 2 0 8 
 
C h e m o C e n t r y x ,  I n c .  
P r o t o c o l  C L 0 0 2 _ 1 6 8                                                                                                          P a g e  1  o f  #  
T a b l e  1 4 . 2 . 5 . 1 . 2 3  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  e G F R  ( M D R D )  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
                           P l a c e b o  +                       C C X 1 6 8  +                       C C X 1 6 8  +  
                      F u l l  D o s e  P r e d n i s o n e           L o w  D o s e  P r e d n i s o n e               N o  P r e d n i s o n e                    A l l  C C X 1 6 8  
                             ( N = # # )                         ( N = # # )                         ( N = # # )                          ( N = # # )  
   S t u d y  D a y         - - - - - - - - - - - - - - - - - - - - - - - -       - - - - - - - - - - - - - - - - - - - - - - - -     - - - - - - - - - - - - - - - - - - - - - - - -       - - - - - - - - - - - - - - - - - - - - - - - - -  
    S t a t i s t i c        V i s i t     C h a n g e   %  C h a n g e      V i s i t     C h a n g e   %  C h a n g e    V i s i t     C h a n g e   %  C h a n g e       V i s i t     C h a n g e   %  C h a n g e        
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
   B a s e l i n e   
    N ’               # #  
    M e a n             # # . # #  
    S D               # # . # # #  
    M i n i m u m          # # . #  
    M e d i a n           # # . # #  
    M a x i m u m          # # . #  
 
   D a y  2  
    N ’               # #        # #        # #           # #        # #        # #          # #        # #        # #           # #        # #        # #  
    M e a n             # # . # #     # # . # #     # # . # #        # # . # #     # # . # #     # # . # #       # # . # #     # # . # #     # # . # #        # # . # #     # # . # #     # # . # #  
    S D               # # . # # #    # # . # # #    # # . # # #       # # . # # #    # # . # # #    # # . # # #      # # . # # #    # # . # # #    # # . # # #       # # . # # #    # # . # # #    # # . # # #  
    M i n i m u m          # # . #      # # . #      # # . #         # # . #      # # . #      # # . #        # # . #      # # . #      # # . #         # # . #      # # . #      # # . #  
    M e d i a n           # # . # #     # # . # #     # # . # #        # # . # #     # # . # #     # # . # #       # # . # #     # # . # #     # # . # #        # # . # #     # # . # #     # # . # #  
    M a x i m u m          # # . #      # # . #      # # . #         # # . #      # # . #      # # . #        # # . #      # # . #      # # . #         # # . #      # # . #      # # . #  
    P - v a l u e *                                              # . # # # #    # . # # # #               # . # # # #    # . # # # #                # . # # # #    # . # # # #  
    9 5 %  C I  f o r  C h a n g e *                                     ( - # # . # ,  # # . # )                 ( - # # . # ,  # # . # )                  ( - # # . # ,  # # . # )  
    9 5 %  C I  f o r  %  C h a n g e *                                   ( - # # . # ,  # # . # )                 ( - # # . # ,  # # . # )                  ( - # # . # ,  # # . # )  
 
  T h e  a d d i t i o n a l  v i s i t s  t o  b e  s u m m a r i z e d  i n c l u d e :  
     D a y  8 ,  D a y  1 5 ,  D a y  2 9 ,  D a y  4 3 ,  D a y  5 7 ,  D a y  7 1 ,  D a y  8 5 ,  E n d  o f  T r e a t m e n t ,  D a y  9 9 ,  D a y  1 1 3 ,  D a y  1 4 1 ,  D a y  1 6 9 ,   
     a n d  E n d  o f  F o l l o w - u p  P e r i o d .  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
-  B a s e l i n e  i s  d e f i n e d  a s  t h e  l a s t  p r e - d o s e  v a l u e .  
-  N ' = n u m b e r  o f  s u b j e c t s  w i t h  d a t a  a t  b a s e l i n e  a n d  t h e  s p e c i f i e d  v i s i t .  
-  T h e  E n d  o f  T r e a t m e n t  v a l u e  i s  t h e  l a s t  m e a s u r e m e n t  t h r o u g h  D a y  8 5 .  
-  T h e  E n d  o f  F o l l o w - u p  P e r i o d  i s  t h e  l a s t  m e a s u r e m e n t  a f t e r  D a y  8 5  t h r o u g h  D a y  1 6 9 .  
* P - v a l u e s  a n d  9 5 %  c o n f i d e n c e  i n t e r v a l s  f o r  d i f f e r e n c e s  b e t w e e n  s p e c i f i e d  t r e a t m e n t  g r o u p s  a n d  p l a c e b o  a r e  f r o m  A N C O V A  m o d e l s  w i t h  
 t r e a t m e n t  g r o u p ,  A A V  d i s e a s e  s t a t u s  ( n e w  o r  r e l a p s e d )  a n d  A N C A  p o s i t i v i t y  ( M P O  o r  P R 3 ) a s  f a c t o r s  a n d  t h e  b a s e l i n e  v a l u e  a s  a  
 c o v a r i a t e .   
 
P r o g r a m  N a m e :  X X X X X X X X . s a s                   R u n  D a t e :  D D M M M Y Y Y Y  H H : M M                            D a t a b a s e  l a s t  m o d i f i e d :  D D M M Y Y Y Y  H H : M M  


 
P a ge 5 7  of 2 0 8 
 
T he f oll o wi n g t a bles w ill ha ve t he sa me l a y o ut as Ta ble 1 4. 2. 5. 1. 2 3: 
T a b l e  1 4 . 2 . 5 . 1 . 2 4  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  e G F R  ( M D R D )  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 5 . 1 . 2 5  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  e G F R  ( M D R D )  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N o n - R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 5 . 1 . 2 6  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  e G F R  ( M D R D )  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  C y c l o p h o s p h a m i d e  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 5 . 1 . 2 7  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  e G F R  ( M D R D )  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  R i t u x i m a b  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 5 . 1 . 2 8  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  e G F R  ( M D R D )  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N e w l y  D i a g n o s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 5 . 1 . 2 9  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  e G F R  ( M D R D )  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e l a p s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 5 . 1 . 3 0  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  e G F R  ( M D R D )  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M P O +  D i s e a s e  
 
T a b l e  1 4 . 2 . 5 . 1 . 3 1  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  e G F R  ( M D R D )  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  P R 3 +  D i s e a s e  
 
T a b l e  1 4 . 2 . 5 . 1 . 3 2  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  e G F R  ( M D R D )  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  G r a n u l o m a t o s i s  w i t h  P o l y a n g i i t i s  ( W e g e n e r ’ s )  
 
T a b l e  1 4 . 2 . 5 . 1 . 3 3  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  e G F R  ( M D R D )  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M i c r o s c o p i c  P o l y a n g i i t i s  


 
P a ge 5 8  of 2 0 8 
 
C h e m o C e n t r y x ,  I n c .  
P r o t o c o l  C L 0 0 2 _ 1 6 8                                                                                                          P a g e  1  o f  #  
T a b l e  1 4 . 2 . 5 . 2 . 1 . 1  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
                  = = = = = = = = = = = = = = =  S t e p  1  a n d  2  C o m b i n e d  = = = = = = = = = = = = = = =        = = = = = = = = = = = = = = = = = = = = = = =  S t e p  3  = = = = = = = = = = = = = = = = = = = = = = =  
                     P l a c e b o  +            C C X 1 6 8  +                                  P l a c e b o  +          C C X 1 6 8  +  
                     F u l l  D o s e            L o w  D o s e             C C X 1 6 8  +              F u l l  D o s e          L o w  D o s e             C C X 1 6 8  +   
                     P r e d n i s o n e          P r e d n i s o n e         N o  P r e d n i s o n e            P r e d n i s o n e        P r e d n i s o n e         N o  P r e d n i s o n e  
                       ( N = # # )             ( N = # # )               ( N = # # )                 ( N = # # )             ( N = # # )               ( N = # # )  
   S t u d y  D a y       - - - - - - - - - - - - - - - -    - - - - - - - - - - - - - - - -    - - - - - - - - - - - - - - - -       - - - - - - - - - - - - - - - -    - - - - - - - - - - - - - - - -    - - - - - - - - - - - - - - - -  
    S t a t i s t i c      V i s i t    %  C h a n g e    V i s i t    %  C h a n g e    V i s i t    %  C h a n g e       V i s i t    %  C h a n g e    V i s i t    %  C h a n g e    V i s i t    %  C h a n g e  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
   B a s e l i n e  
    N ’              # #  
    M e a n            # # . #  
    S D              # # . # #  
    M i n i m u m         # #  
    M e d i a n          # # . #  
    M a x i m u m         # #  
 
   D a y  2  
    N ’              # #        # #        # #          # #        # #        # #             # #        # #        # #          # #        # #        # #  
    M e a n            # # . #      # # . #      # # . #        # # . #      # # . #      # # . #           # # . #      # # . #      # # . #        # # . #      # # . #      # # . #  
    S D              # # . # #     # # . # #     # # . # #       # # . # #     # # . # #     # # . # #          # # . # #     # # . # #     # # . # #       # # . # #     # # . # #     # # . # #  
    M i n i m u m         # #        # #        # #          # #        # #        # #             # #        # #        # #          # #        # #        # #  
    M e d i a n          # # . #      # # . #      # # . #        # # . #      # # . #      # # . #           # # . #      # # . #      # # . #        # # . #      # # . #      # # . #  
    M a x i m u m         # #        # #        # #          # #        # #        # #             # #        # #        # #          # #        # #        # #  
    P - v a l u e *                                   # . # # # #             # . # # # #                                      # . # # # #             # . # # # #  
    9 5 %  C I *                                ( - # # . # ,  # # . # )      ( - # # . # ,  # # . # )                               ( - # # . # ,  # # . # )      ( - # # . # ,  # # . # )  
   
 T h e  a d d i t i o n a l  v i s i t s  t o  b e  s u m m a r i z e d  i n c l u d e :  
     D a y  8 ,  D a y  1 5 ,  D a y  2 9 ,  D a y  4 3 ,  D a y  5 7 ,  D a y  7 1 ,  D a y  8 5  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
-  B a s e l i n e  i s  d e f i n e d  a s  t h e  l a s t  p r e - d o s e  v a l u e .  
-  N ' = n u m b e r  o f  s u b j e c t s  w i t h  d a t a  a t  b a s e l i n e  a n d  t h e  s p e c i f i e d  v i s i t .  
* P - v a l u e s  a n d  9 5 %  c o n f i d e n c e  i n t e r v a l s  f o r  d i f f e r e n c e s  b e t w e e n  s p e c i f i e d  t r e a t m e n t  g r o u p s  a n d  p l a c e b o  a r e  f r o m  M M R M  m o d e l s  w i t h  
  t r e a t m e n t  g r o u p ,  v i s i t ,  a n d  t r e a t m e n t - b y - v i s i t  a s  f a c t o r s  a n d  t h e  b a s e l i n e  v a l u e  a s  a  c o v a r i a t e .  T h e  m o d e l  f o r  a n a l y s i s  o f  S t e p  3   
  i n c l u d e d  f a c t o r s  f o r  A A V  d i s e a s e  s t a t u s  ( n e w  o r  r e l a p s e d ) ,  A N C A  p o s i t i v i t y  ( M P O  o r  P R 3 ) ,  a n d  s t a n d a r d  o f  c a r e  t r e a t m e n t  ( r i t u x i m a b   
  o r  c y c l o p h o s p h a m i d e ) .   T h e  m o d e l  f o r  S t e p  1  +  S t e p  2  i n c l u d e d  a  f a c t o r  f o r   d i s e a s e  s t a t u s .  
 
P r o g r a m  N a m e :  X X X X X X X X . s a s                   R u n  D a t e :  D D M M M Y Y Y Y  H H : M M                            D a t a b a s e  l a s t  m o d i f i e d :  D D M M Y Y Y Y  H H : M M  


 
P a ge 5 9  of 2 0 8 
 
T he f oll o wi n g t a bles w ill ha ve la y o uts si milar t o Ta ble 1 4. 2. 5. 2. 1. 1: 
T a b l e  1 4 . 2 . 5 . 2 . 1 . 2  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 5 . 2 . 1 . 3  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N o n - R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 5 . 2 . 1 . 4  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  C y c l o p h o s p h a m i d e  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 5 . 2 . 1 . 5  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  R i t u x i m a b  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 5 . 2 . 1 . 6  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N e w l y  D i a g n o s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 5 . 2 . 1 . 7  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e l a p s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 5 . 2 . 1 . 8  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M P O +  D i s e a s e  
 
T a b l e  1 4 . 2 . 5 . 2 . 1 . 9  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  P R 3 +  D i s e a s e  
 
T a b l e  1 4 . 2 . 5 . 2 . 1 . 1 0  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  G r a n u l o m a t o s i s  w i t h  P o l y a n g i i t i s  ( W e g e n e r ’ s )  
 
T a b l e  1 4 . 2 . 5 . 2 . 1 . 1 1  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M i c r o s c o p i c  P o l y a n g i i t i s  


 
P a ge 6 0  of 2 0 8 
 
T a b l e  1 4 . 2 . 5 . 2 . 1 . 1 2  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  
 
T a b l e  1 4 . 2 . 5 . 2 . 1 . 1 3  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 5 . 2 . 1 . 1 4  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N o n - R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 5 . 2 . 1 . 1 5  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  C y c l o p h o s p h a m i d e  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 5 . 2 . 1 . 1 6  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  R i t u x i m a b  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 5 . 2 . 1 . 1 7  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N e w l y  D i a g n o s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 5 . 2 . 1 . 1 8  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e l a p s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 5 . 2 . 1 . 1 9  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M P O +  D i s e a s e  
 
T a b l e  1 4 . 2 . 5 . 2 . 1 . 2 0  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  P R 3 +  D i s e a s e  
 
T a b l e  1 4 . 2 . 5 . 2 . 1 . 2 1  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  G r a n u l o m a t o s i s  w i t h  P o l y a n g i i t i s  ( W e g e n e r ’ s )  


 
P a ge 6 1  of 2 0 8 
 
T a b l e  1 4 . 2 . 5 . 2 . 1 . 2 2  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M i c r o s c o p i c  P o l y a n g i i t i s  


 
P a ge 6 2  of 2 0 8 
 
T a b l e  1 4 . 2 . 5 . 2 . 2 . 1  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  
 
T a b l e  1 4 . 2 . 5 . 2 . 2 . 2  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 5 . 2 . 2 . 3  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N o n - R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 5 . 2 . 2 . 4  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  C y c l o p h o s p h a m i d e  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 5 . 2 . 2 . 5  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  R i t u x i m a b  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 5 . 2 . 2 . 6  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N e w l y  D i a g n o s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 5 . 2 . 2 . 7  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e l a p s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 5 . 2 . 2 . 8  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M P O +  D i s e a s e  
 
T a b l e  1 4 . 2 . 5 . 2 . 2 . 9  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  P R 3 +  D i s e a s e  
 
T a b l e  1 4 . 2 . 5 . 2 . 2 . 1 0  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  G r a n u l o m a t o s i s  w i t h  P o l y a n g i i t i s  ( W e g e n e r ’ s )  
 


 
P a ge 6 3  of 2 0 8 
 
T a b l e  1 4 . 2 . 5 . 2 . 2 . 1 1  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M i c r o s c o p i c  P o l y a n g i i t i s  


 
P a ge 6 4  of 2 0 8 
 
T a b l e  1 4 . 2 . 5 . 2 . 2 . 1 2  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  
 
T a b l e  1 4 . 2 . 5 . 2 . 2 . 1 3  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 5 . 2 . 2 . 1 4  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N o n - R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 5 . 2 . 2 . 1 5  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  C y c l o p h o s p h a m i d e  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 5 . 2 . 2 . 1 6  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  R i t u x i m a b  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 5 . 2 . 2 . 1 7  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N e w l y  D i a g n o s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 5 . 2 . 2 . 1 8  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e l a p s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 5 . 2 . 2 . 1 9  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M P O +  D i s e a s e  
 
T a b l e  1 4 . 2 . 5 . 2 . 2 . 2 0  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  P R 3 +  D i s e a s e  
 
T a b l e  1 4 . 2 . 5 . 2 . 2 . 2 1  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  G r a n u l o m a t o s i s  w i t h  P o l y a n g i i t i s  ( W e g e n e r ’ s )  


 
P a ge 6 5  of 2 0 8 
 
T a b l e  1 4 . 2 . 5 . 2 . 2 . 2 2  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M i c r o s c o p i c  P o l y a n g i i t i s  


 
P a ge 6 6  of 2 0 8 
 
C h e m o C e n t r y x ,  I n c .  
P r o t o c o l  C L 0 0 2 _ 1 6 8                                                                                                          P a g e  1  o f  #  
T a b l e  1 4 . 2 . 5 . 3 . 1  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  8 4 - d a y  T r e a t m e n t  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
  S t e p                        P l a c e b o  +                      C C X 1 6 8  +                     C C X 1 6 8  +  
   S t u d y            - -  F u l l  D o s e  P r e d n i s o n e  - -      - -  L o w  D o s e  P r e d n i s o n e  - -    - - - - -  N o  P r e d n i s o n e  - - - - -       - - - - - - -  A l l  C C X 1 6 8  - - - - - -  
    S t a t i s t i c        V i s i t     C h a n g e   %  C h a n g e      V i s i t     C h a n g e   %  C h a n g e    V i s i t     C h a n g e   %  C h a n g e       V i s i t     C h a n g e   %  C h a n g e  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
  S t e p  3                       N = # #                           N = # #                         N = # #                            N = # #  
 
   B a s e l i n e   
    N ’               # #  
    M e a n             # # . # #  
    S D               # # . # # #  
    M i n i m u m          # # . #  
    M e d i a n           # # . # #  
    M a x i m u m          # # . #  
 
   D a y  2  
    N ’               # #        # #        # #           # #        # #        # #          # #        # #        # #           # #        # #        # #  
    M e a n             # # . # #     # # . # #     # # . # #        # # . # #     # # . # #     # # . # #       # # . # #     # # . # #     # # . # #        # # . # #     # # . # #     # # . # #  
    S D               # # . # # #    # # . # # #    # # . # # #       # # . # # #    # # . # # #    # # . # # #      # # . # # #    # # . # # #    # # . # # #       # # . # # #    # # . # # #    # # . # # #  
    M i n i m u m          # # . #      # # . #      # # . #         # # . #      # # . #      # # . #        # # . #      # # . #      # # . #         # # . #      # # . #      # # . #  
    M e d i a n           # # . # #     # # . # #     # # . # #        # # . # #     # # . # #     # # . # #       # # . # #     # # . # #     # # . # #        # # . # #     # # . # #     # # . # #  
    M a x i m u m          # # . #      # # . #      # # . #         # # . #      # # . #      # # . #        # # . #      # # . #      # # . #         # # . #      # # . #      # # . #  
    P - v a l u e *                                              # . # # # #    # . # # # #               # . # # # #    # . # # # #                # . # # # #    # . # # # #  
    9 5 %  C I  f o r  C h a n g e *                                     ( - # # . # ,  # # . # )                 ( - # # . # ,  # # . # )                  ( - # # . # ,  # # . # )  
    9 5 %  C I  f o r  %  C h a n g e *                                   ( - # # . # ,  # # . # )                 ( - # # . # ,  # # . # )                  ( - # # . # ,  # # . # )  
 
  R e p e a t  f o r  S t e p s  2  a n d  1 .   O n l y  d i s p l a y  p - v a l u e s  a n d  c o n f i d e n c e  i n t e r v a l s  f o r  S t e p  3 .  
 
  T h e  a d d i t i o n a l  v i s i t s  t o  b e  s u m m a r i z e d  i n c l u d e :  
     D a y  8 ,  D a y  1 5 ,  D a y  2 9 ,  D a y  4 3 ,  D a y  5 7 ,  D a y  7 1 ,  D a y  8 5 ,  E n d  o f  T r e a t m e n t ,  D a y  9 9 ,  D a y  1 1 3 ,  D a y  1 4 1 ,  D a y  1 6 9 ,   
     a n d  E n d  o f  F o l l o w - u p  P e r i o d .  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
-  B a s e l i n e  i s  d e f i n e d  a s  t h e  l a s t  p r e - d o s e  v a l u e .  
-  N ' = n u m b e r  o f  s u b j e c t s  w i t h  d a t a  a t  b a s e l i n e  a n d  t h e  s p e c i f i e d  v i s i t .  
-  T h e  E n d  o f  T r e a t m e n t  v a l u e  i s  t h e  l a s t  m e a s u r e m e n t  t h r o u g h  D a y  8 5 .  
-  T h e  E n d  o f  F o l l o w - u p  P e r i o d  i s  t h e  l a s t  m e a s u r e m e n t  a f t e r  D a y  8 5  t h r o u g h  D a y  1 6 9 .  
* P - v a l u e s  a n d  9 5 %  c o n f i d e n c e  i n t e r v a l s  f o r  d i f f e r e n c e s  b e t w e e n  s p e c i f i e d  t r e a t m e n t  g r o u p s  a n d  p l a c e b o  a r e  f r o m  a  M M R M  m o d e l  w i t h  
 t r e a t m e n t  g r o u p  a n d  r a n d o m i z a t i o n  s t r a t a  ( A A V  s t a t u s ,  A N C A  p o s i t i v i t y ,  a n d  s t a n d a r d  o f  c a r e  t r e a t m e n t )  a s  f a c t o r s  a n d  t h e  b a s e l i n e   
 v a l u e  a s  a  c o v a r i a t e .  
 
P r o g r a m  N a m e :  X X X X X X X X . s a s                   R u n  D a t e :  D D M M M Y Y Y Y  H H : M M                            D a t a b a s e  l a s t  m o d i f i e d :  D D M M Y Y Y Y  H H : M M  


 
P a ge 6 7  of 2 0 8 
 
T he f oll o wi n g t a bles w ill ha ve a la y o ut si milar t o Ta ble 1 4. 2. 5. 3. 1: 
T a b l e  1 4 . 2 . 5 . 3 . 2  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  8 4 - d a y  T r e a t m e n t  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 5 . 3 . 3  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  8 4 - d a y  T r e a t m e n t  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N o n - R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 5 . 3 . 4  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  8 4 - d a y  T r e a t m e n t  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  C y c l o p h o s p h a m i d e  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 5 . 3 . 5  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  8 4 - d a y  T r e a t m e n t  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  R i t u x i m a b  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 5 . 3 . 6  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  8 4 - d a y  T r e a t m e n t  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N e w l y  D i a g n o s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 5 . 3 . 7  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  8 4 - d a y  T r e a t m e n t  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e l a p s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 5 . 3 . 8  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  8 4 - d a y  T r e a t m e n t  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M P O +  D i s e a s e  
 
T a b l e  1 4 . 2 . 5 . 3 . 9  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  8 4 - d a y  T r e a t m e n t  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  P R 3 +  D i s e a s e  
 
T a b l e  1 4 . 2 . 5 . 3 . 1 0  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  8 4 - d a y  T r e a t m e n t  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  G r a n u l o m a t o s i s  w i t h  P o l y a n g i i t i s  ( W e g e n e r ’ s )  
 
T a b l e  1 4 . 2 . 5 . 3 . 1 1  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  8 4 - d a y  T r e a t m e n t  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M i c r o s c o p i c  P o l y a n g i i t i s  


 
P a ge 6 8  of 2 0 8 
 
T a b l e  1 4 . 2 . 5 . 3 . 1 2  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  
 
T a b l e  1 4 . 2 . 5 . 3 . 1 3  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 5 . 3 . 1 4  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N o n - R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 5 . 3 . 1 5  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  C y c l o p h o s p h a m i d e  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 5 . 3 . 1 6  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  R i t u x i m a b  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 5 . 3 . 1 7  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N e w l y  D i a g n o s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 5 . 3 . 1 8  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e l a p s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 5 . 3 . 1 9  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M P O +  D i s e a s e  
 
T a b l e  1 4 . 2 . 5 . 3 . 2 0  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  P R 3 +  D i s e a s e  
 
T a b l e  1 4 . 2 . 5 . 3 . 2 1  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  G r a n u l o m a t o s i s  w i t h  P o l y a n g i i t i s  ( W e g e n e r ’ s )  


 
P a ge 6 9  of 2 0 8 
 
T a b l e  1 4 . 2 . 5 . 3 . 2 2  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  e G F R  ( M D R D )  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M i c r o s c o p i c  P o l y a n g i i t i s  
 
N ote t o pr o gra m mer:  T he M M R M ta bles f or t he 1 6 8- da y st u d y peri o d will i ncl u de s u m maries f or eac h p ost- bas eli ne visit.  I n 
a d diti o n, f or Ste p 3 t he p- val ue a n d 9 5 % c o nfi d e nce i nter val f or t he o verall differe nces bet wee n eac h gr o u p a n d place b o will be 
dis pla ye d. 


 
P a ge 7 0  of 2 0 8 
 
C h e m o C e n t r y x ,  I n c .  
P r o t o c o l  C L 0 0 2 _ 1 6 8                                                                                                          P a g e  1  o f  2  
T a b l e  1 4 . 2 . 6 . 1  
I n c i d e n c e  a n d  T i m e  t o  t h e  F i r s t  O c c u r r e n c e  o f  U r i n a r y - R B C  < = 5  R B C s / h p f  i n  t h e  U r i n e  
U r i n a r y - R B C  A s s e s s e d  b y  C e n t r a l  L a b o r a t o r y  
I n t e n t - t o - T r e a t  P o p u l a t i o n  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
P e r i o d                                                                               D a y s  t o  F i r s t  O c c u r r e n c e  o f  U r i n a r y  R B C  < = 5 / H P F    
     S t e p      T r e a t m e n t                          N ’       n     ( % )       P - v a l u e    2 5 t h  p e r c e n t i l e   M e d i a n   7 5 t h  p e r c e n t i l e    P - v a l u e  [ 1 ]  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
 8 4 - d a y  d o s i n g  p e r i o d  
 
    A l l   P l a c e b o  +  F u l l  D o s e  P r e d n i s o n e  ( N = # # )   # # #     # # #   ( # # # . # )                     # #          # # . #          # #  
         C C X 1 6 8  +  L o w  D o s e  P r e d n i s o n e  ( N = # # )     # # #     # # #   ( # # # . # )     # . # # # #           # #          # # . #          # #           # . # # # #  
         C C X 1 6 8  +  N o  P r e d n i s o n e  ( N = # # )           # # #     # # #   ( # # # . # )     # . # # # #           # #          # # . #          # #           # . # # # #  
         A l l  C C X 1 6 8  ( N = # # )                       # # #     # # #   ( # # # . # )     # . # # # #           # #          # # . #          # #           # . # # # #  
 
    3     P l a c e b o  +  F u l l  D o s e  P r e d n i s o n e  ( N = # # )   # # #     # # #   ( # # # . # )                     # #          # # . #          # #  
         C C X 1 6 8  +  L o w  D o s e  P r e d n i s o n e  ( N = # # )     # # #     # # #   ( # # # . # )     # . # # # #           # #          # # . #          # #           # . # # # #  
         C C X 1 6 8  +  N o  P r e d n i s o n e  ( N = # # )           # # #     # # #   ( # # # . # )     # . # # # #           # #          # # . #          # #           # . # # # #  
         A l l  C C X 1 6 8  ( N = # # )                       # # #     # # #   ( # # # . # )     # . # # # #           # #          # # . #          # #           # . # # # #  
 
    1 + 2   P l a c e b o  +  F u l l  D o s e  P r e d n i s o n e  ( N = # # )   # # #     # # #   ( # # # . # )                     # #          # # . #          # #  
         C C X 1 6 8  +  L o w  D o s e  P r e d n i s o n e  ( N = # # )     # # #     # # #   ( # # # . # )     # . # # # #           # #          # # . #          # #           # . # # # #  
         C C X 1 6 8  +  N o  P r e d n i s o n e  ( N = # # )           # # #     # # #   ( # # # . # )     # . # # # #           # #          # # . #          # #           # . # # # #  
         A l l  C C X 1 6 8  ( N = # # )                       # # #     # # #   ( # # # . # )     # . # # # #           # #          # # . #          # #           # . # # # #  
 
    2     P l a c e b o  +  F u l l  D o s e  P r e d n i s o n e  ( N = # # )   # # #     # # #   ( # # # . # )                     # #          # # . #          # #  
         C C X 1 6 8  +  N o  P r e d n i s o n e  ( N = # # )           # # #     # # #   ( # # # . # )     # . # # # #           # #          # # . #          # #           # . # # # #  
 
    1     P l a c e b o  +  F u l l  D o s e  P r e d n i s o n e  ( N = # # )   # # #     # # #   ( # # # . # )                     # #          # # . #          # #  
         C C X 1 6 8  +  L o w  D o s e  P r e d n i s o n e  ( N = # # )     # # #     # # #   ( # # # . # )     # . # # # #           # #          # # . #          # #           # . # # # #  
 
R e p e a t  f o r  1 6 8 - D a y  S t u d y  P e r i o d  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
-  N ’  =  n u m b e r  o f  s u b j e c t s  w i t h  b a s e l i n e  u r i n a r y - R B C  o f  g r e a t e r  t h a n  5  R B C / h p f   a n d  a t  l e a s t  o n e  p o s t - b a s e l i n e  u r i n a r y - R B C  a s s e s s m e n t   
     d u r i n g  t h e  s p e c i f i e d  p e r i o d  
-  n  =  n u m b e r  o f  s u b j e c t s  w i t h  u r i n a r y - R B C  o f  < = 5  R B C / h p f  d u r i n g  t h e  s p e c i f i e d  p e r i o d  
[ 1 ]  E s t i m a t e s  a r e  K a p l a n - M e i e r  e s t i m a t e s  a n d  p - v a l u e s  a r e  f r o m  t h e  l o g - r a n k  t e s t  t o  c o m p a r e  t h e  s p e c i f i e d  t r e a t m e n t  g r o u p  t o  p l a c e b o .  
 
P r o g r a m  N a m e :  X X X X X X X X . s a s                   R u n  D a t e :  D D M M M Y Y Y Y  H H : M M                            D a t a b a s e  l a s t  m o d i f i e d :  D D M M Y Y Y Y  H H : M M  


 
P a ge 7 1  of 2 0 8 
 
T he f oll o wi n g t a bles w ill ha ve t he sa me l a y o ut as Ta ble 1 4. 2. 6. 1: 
T a b l e  1 4 . 2 . 6 . 2  
I n c i d e n c e  a n d  T i m e  t o  t h e  F i r s t  O c c u r r e n c e  o f  U r i n a r y - R B C  < = 5  R B C s / h p f  i n  t h e  U r i n e  
U r i n a r y - R B C  A s s e s s e d  b y  C e n t r a l  L a b o r a t o r y  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  C y c l o p h o s p h a m i d e  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 6 . 3  
I n c i d e n c e  a n d  T i m e  t o  t h e  F i r s t  O c c u r r e n c e  o f  U r i n a r y - R B C  < = 5  R B C s / h p f  i n  t h e  U r i n e  
U r i n a r y - R B C  A s s e s s e d  b y  C e n t r a l  L a b o r a t o r y  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  R i t u x i m a b  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 6 . 4  
I n c i d e n c e  a n d  T i m e  t o  t h e  F i r s t  O c c u r r e n c e  o f  U r i n a r y - R B C  < = 5  R B C s / h p f  i n  t h e  U r i n e  
U r i n a r y - R B C  A s s e s s e d  b y  C e n t r a l  L a b o r a t o r y  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N e w l y  D i a g n o s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 6 . 5  
I n c i d e n c e  a n d  T i m e  t o  t h e  F i r s t  O c c u r r e n c e  o f  U r i n a r y - R B C  < = 5  R B C s / h p f  i n  t h e  U r i n e  
U r i n a r y - R B C  A s s e s s e d  b y  C e n t r a l  L a b o r a t o r y  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e l a p s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 6 . 6  
I n c i d e n c e  a n d  T i m e  t o  t h e  F i r s t  O c c u r r e n c e  o f  U r i n a r y - R B C  < = 5  R B C s / h p f  i n  t h e  U r i n e  
U r i n a r y - R B C  A s s e s s e d  b y  C e n t r a l  L a b o r a t o r y  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M P O +  D i s e a s e  
 
T a b l e  1 4 . 2 . 6 . 7  
I n c i d e n c e  a n d  T i m e  t o  t h e  F i r s t  O c c u r r e n c e  o f  U r i n a r y - R B C  < = 5  R B C s / h p f  i n  t h e  U r i n e  
U r i n a r y - R B C  A s s e s s e d  b y  C e n t r a l  L a b o r a t o r y  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  P R 3 +  D i s e a s e  
 
T a b l e  1 4 . 2 . 6 . 8  
I n c i d e n c e  a n d  T i m e  t o  t h e  F i r s t  O c c u r r e n c e  o f  U r i n a r y - R B C  < = 5  R B C s / h p f  i n  t h e  U r i n e  
U r i n a r y - R B C  A s s e s s e d  b y  C e n t r a l  L a b o r a t o r y  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  G r a n u l o m a t o s i s  w i t h  P o l y a n g i i t i s  ( W e g e n e r ’ s )  
 
T a b l e  1 4 . 2 . 6 . 9  
I n c i d e n c e  a n d  T i m e  t o  t h e  F i r s t  O c c u r r e n c e  o f  U r i n a r y - R B C  < = 5  R B C s / h p f  i n  t h e  U r i n e  
U r i n a r y - R B C  A s s e s s e d  b y  C e n t r a l  L a b o r a t o r y  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M i c r o s c o p i c  P o l y a n g i i t i s  


 
P a ge 7 2  of 2 0 8 
 
C h e m o C e n t r y x ,  I n c .  
P r o t o c o l  C L 0 0 2 _ 1 6 8                                                                                                          P a g e  1  o f  2  
T a b l e  1 4 . 2 . 7 . 1  
I n c i d e n c e  a n d  T i m e  t o  t h e  F i r s t  O c c u r r e n c e  o f  U r i n a r y - R B C  < 3 0  R B C s / h p f  i n  t h e  U r i n e  
U r i n a r y - R B C  A s s e s s e d  b y  C e n t r a l  L a b o r a t o r y  
I n t e n t - t o - T r e a t  P o p u l a t i o n  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
 P e r i o d                                                                              D a y s  t o  F i r s t  O c c u r r e n c e  o f  U r i n a r y  R B C  < 3 0 / H P F    
     S t e p      T r e a t m e n t                          N ’       n     ( % )       P - v a l u e    2 5 t h  p e r c e n t i l e   M e d i a n   7 5 t h  p e r c e n t i l e    P - v a l u e  [ 1 ]  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
 8 4 - d a y  d o s i n g  p e r i o d  
    A l l   P l a c e b o  +  F u l l  D o s e  P r e d n i s o n e  ( N = # # )   # # #     # # #   ( # # # . # )                     # #          # # . #          # #  
         C C X 1 6 8  +  L o w  D o s e  P r e d n i s o n e  ( N = # # )     # # #     # # #   ( # # # . # )     # . # # # #           # #          # # . #          # #           # . # # # #  
         C C X 1 6 8  +  N o  P r e d n i s o n e  ( N = # # )           # # #     # # #   ( # # # . # )     # . # # # #           # #          # # . #          # #           # . # # # #  
         A l l  C C X 1 6 8  ( N = # # )                       # # #     # # #   ( # # # . # )     # . # # # #           # #          # # . #          # #           # . # # # #  
 
    3     P l a c e b o  +  F u l l  D o s e  P r e d n i s o n e  ( N = # # )   # # #     # # #   ( # # # . # )                     # #          # # . #          # #  
         C C X 1 6 8  +  L o w  D o s e  P r e d n i s o n e  ( N = # # )     # # #     # # #   ( # # # . # )     # . # # # #           # #          # # . #          # #           # . # # # #  
         C C X 1 6 8  +  N o  P r e d n i s o n e  ( N = # # )           # # #     # # #   ( # # # . # )     # . # # # #           # #          # # . #          # #           # . # # # #  
         A l l  C C X 1 6 8  ( N = # # )                       # # #     # # #   ( # # # . # )     # . # # # #           # #          # # . #          # #           # . # # # #  
 
    1 + 2   P l a c e b o  +  F u l l  D o s e  P r e d n i s o n e  ( N = # # )   # # #     # # #   ( # # # . # )                     # #          # # . #          # #  
         C C X 1 6 8  +  L o w  D o s e  P r e d n i s o n e  ( N = # # )     # # #     # # #   ( # # # . # )     # . # # # #           # #          # # . #          # #           # . # # # #  
         C C X 1 6 8  +  N o  P r e d n i s o n e  ( N = # # )           # # #     # # #   ( # # # . # )     # . # # # #           # #          # # . #          # #           # . # # # #  
         A l l  C C X 1 6 8  ( N = # # )                       # # #     # # #   ( # # # . # )     # . # # # #           # #          # # . #          # #           # . # # # #  
 
    2     P l a c e b o  +  F u l l  D o s e  P r e d n i s o n e  ( N = # # )   # # #     # # #   ( # # # . # )                     # #          # # . #          # #  
         C C X 1 6 8  +  N o  P r e d n i s o n e  ( N = # # )           # # #     # # #   ( # # # . # )     # . # # # #           # #          # # . #          # #           # . # # # #  
 
    1     P l a c e b o  +  F u l l  D o s e  P r e d n i s o n e  ( N = # # )   # # #     # # #   ( # # # . # )                     # #          # # . #          # #  
         C C X 1 6 8  +  L o w  D o s e  P r e d n i s o n e  ( N = # # )     # # #     # # #   ( # # # . # )     # . # # # #           # #          # # . #          # #           # . # # # #  
 
R e p e a t  f o r  1 6 8 - d a y  s t u d y  p e r i o d  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
-  N ’  =  n u m b e r  o f  s u b j e c t s  w i t h  b a s e l i n e  u r i n a r y - R B C  o f  > = 3 0  R B C / h p f   a n d  a t  l e a s t  o n e  p o s t - b a s e l i n e  u r i n a r y - R B C  a s s e s s m e n t  d u r i n g  
     t h e  s p e c i f i e d  p e r i o d  
-  n  =  n u m b e r  o f  s u b j e c t s  w i t h  u r i n a r y - R B C  o f  < 3 0  R B C / h p f  d u r i n g  t h e  s p e c i f i e d  p e r i o d  
[ 1 ]  E s t i m a t e s  a r e  K a p l a n - M e i e r  e s t i m a t e s  a n d  p - v a l u e s  a r e  f r o m  t h e  l o g - r a n k  t e s t  t o  c o m p a r e  t h e  s p e c i f i e d  t r e a t m e n t  g r o u p  t o  p l a c e b o .  
 
P r o g r a m  N a m e :  X X X X X X X X . s a s                   R u n  D a t e :  D D M M M Y Y Y Y  H H : M M                            D a t a b a s e  l a s t  m o d i f i e d :  D D M M Y Y Y Y  H H : M M  


 
P a ge 7 3  of 2 0 8 
 
 T he f oll o wi n g t a bles will ha ve t he sa me la y o ut as Ta ble 1 4. 2. 7. 1: 
T a b l e  1 4 . 2 . 7 . 2  
I n c i d e n c e  a n d  T i m e  t o  t h e  F i r s t  O c c u r r e n c e  o f  U r i n a r y - R B C  < 3 0  R B C s / h p f  i n  t h e  U r i n e  
U r i n a r y - R B C  A s s e s s e d  b y  C e n t r a l  L a b o r a t o r y  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  C y c l o p h o s p h a m i d e  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 7 . 3  
I n c i d e n c e  a n d  T i m e  t o  t h e  F i r s t  O c c u r r e n c e  o f  U r i n a r y - R B C  < 3 0  R B C s / h p f  i n  t h e  U r i n e  
U r i n a r y - R B C  A s s e s s e d  b y  C e n t r a l  L a b o r a t o r y  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  R i t u x i m a b  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 7 . 4  
I n c i d e n c e  a n d  T i m e  t o  t h e  F i r s t  O c c u r r e n c e  o f  U r i n a r y - R B C  < 3 0  R B C s / h p f  i n  t h e  U r i n e  
U r i n a r y - R B C  A s s e s s e d  b y  C e n t r a l  L a b o r a t o r y  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N e w l y  D i a g n o s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 7 . 5  
I n c i d e n c e  a n d  T i m e  t o  t h e  F i r s t  O c c u r r e n c e  o f  U r i n a r y - R B C  < 3 0  R B C s / h p f  i n  t h e  U r i n e  
U r i n a r y - R B C  A s s e s s e d  b y  C e n t r a l  L a b o r a t o r y  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e l a p s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 7 . 6  
I n c i d e n c e  a n d  T i m e  t o  t h e  F i r s t  O c c u r r e n c e  o f  U r i n a r y - R B C  < 3 0  R B C s / h p f  i n  t h e  U r i n e  
U r i n a r y - R B C  A s s e s s e d  b y  C e n t r a l  L a b o r a t o r y  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M P O +  D i s e a s e  
 
T a b l e  1 4 . 2 . 7 . 7  
I n c i d e n c e  a n d  T i m e  t o  t h e  F i r s t  O c c u r r e n c e  o f  U r i n a r y - R B C  < 3 0  R B C s / h p f  i n  t h e  U r i n e  
U r i n a r y - R B C  A s s e s s e d  b y  C e n t r a l  L a b o r a t o r y  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  P R 3 +  D i s e a s e  
 
T a b l e  1 4 . 2 . 7 . 8  
I n c i d e n c e  a n d  T i m e  t o  t h e  F i r s t  O c c u r r e n c e  o f  U r i n a r y - R B C  < 3 0  R B C s / h p f  i n  t h e  U r i n e  
U r i n a r y - R B C  A s s e s s e d  b y  C e n t r a l  L a b o r a t o r y  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  G r a n u l o m a t o s i s  w i t h  P o l y a n g i i t i s  ( W e g e n e r ’ s )  
 
T a b l e  1 4 . 2 . 7 . 9  
I n c i d e n c e  a n d  T i m e  t o  t h e  F i r s t  O c c u r r e n c e  o f  U r i n a r y - R B C  < 3 0  R B C s / h p f  i n  t h e  U r i n e  
U r i n a r y - R B C  A s s e s s e d  b y  C e n t r a l  L a b o r a t o r y  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M i c r o s c o p i c  P o l y a n g i i t i s  


 
P a ge 7 4  of 2 0 8 
 
C h e m o C e n t r y x ,  I n c .  
P r o t o c o l  C L 0 0 2 _ 1 6 8                                                                                                          P a g e  1  o f  #  
T a b l e  1 4 . 2 . 8 . 1 . 1  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  R B C *  C o u n t :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  H e m a t u r i a  a t  B a s e l i n e  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
                           P l a c e b o  +                       C C X 1 6 8  +                     C C X 1 6 8  +  
                      F u l l  D o s e  P r e d n i s o n e            L o w  D o s e  P r e d n i s o n e             N o  P r e d n i s o n e                    A l l  C C X 1 6 8  
                             ( N = # # )                         ( N = # # )                       ( N = # # )                          ( N = # # )  
   S t u d y  D a y         - - - - - - - - - - - - - - - - - - - - - - - -       - - - - - - - - - - - - - - - - - - - - - - - -     - - - - - - - - - - - - - - - - - - - - - - - -       - - - - - - - - - - - - - - - - - - - - - - - - -  
    S t a t i s t i c        V i s i t     R a t i o   %  C h a n g e       V i s i t     R a t i o   %  C h a n g e     V i s i t     R a t i o   %  C h a n g e        V i s i t     R a t i o   %  C h a n g e  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
   B a s e l i n e   
    N ’               # #  
    M e a n             # # . # #  
    G e o .  M e a n  ( G M )   # # . # #  
    M i n i m u m          # # . #  
    M e d i a n           # # . # #  
    M a x i m u m          # # . #  
 
  D a y  2  
    N ’               # #        # #        # #           # #        # #        # #          # #        # #        # #           # #        # #        # #  
    M e a n             # # . # #     # # . # #     # # . # #        # # . # #     # # . # #     # # . # #       # # . # #     # # . # #     # # . # #        # # . # #     # # . # #     # # . # #  
    G e o .  M e a n  ( G M )   # # . # # #    # # . # # #                # # . # # #    # # . # # #               # # . # # #    # # . # # #                # # . # # #    # # . # # #  
    M i n i m u m          # # . #      # # . #      # # . #         # # . #      # # . #      # # . #        # # . #      # # . #      # # . #         # # . #      # # . #      # # . #  
    M e d i a n           # # . # #     # # . # #     # # . # #        # # . # #     # # . # #     # # . # #       # # . # #     # # . # #     # # . # #        # # . # #     # # . # #     # # . # #  
    M a x i m u m          # # . #      # # . #      # # . #         # # . #      # # . #      # # . #        # # . #      # # . #      # # . #         # # . #      # # . #      # # . #      
    P - v a l u e ^                                               # . # # # #                       # . # # # #                         # . # # # #  
    9 5 %  C I ^  f o r  R a t i o                                  ( - # . # # ,  # . # # )                ( - # . # # ,  # . # # )                  ( - # . # # ,  # . # # )        
        
  A d d i t i o n a l  v i s i t s  t o  b e  s u m m a r i z e d  i n c l u d e :  
     D a y  8 ,  D a y  1 5 ,  D a y  2 9 ,  D a y  4 3 ,  D a y  5 7 ,  D a y  7 1 ,  D a y  8 5 ,  a n d  O v e r a l l  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
-  B a s e l i n e  i s  d e f i n e d  a s  t h e  l a s t  p r e - d o s e  v a l u e .  
-  N ' = n u m b e r  o f  s u b j e c t s  w i t h  d a t a  a t  b a s e l i n e  a n d  t h e  s p e c i f i e d  v i s i t .  
-  R a t i o  i s  d e f i n e d  f o r  e a c h  s u b j e c t  a s  t h e  v i s i t  v a l u e  d i v i d e d  b y  t h e  b a s e l i n e  v a l u e .  
*  S i n c e  u r i n a r y - R B C  v a l u e s  a r e  r e p o r t e d  a s  r a n g e s ,  q u a n t i t a t i v e  v a l u e s  a r e  d e f i n e d  a s :  7 5  f o r  r e s u l t  o f  ‘ > 7 5 ’ ,  5 0  f o r  ’ 5 0 - 7 5 ’ ,  
  3 0  f o r  ’ 3 0 - 4 9 ’ ,  1 6  f o r  ’ 1 6 - 2 9 ’ ,  1 0  f o r  ’ 1 0 - 1 5 ’ ,  6  f o r  ‘ 6 - 9 ’ ,  3  f o r  ‘ 3 - 5 ’ ,  1  f o r  ‘ 1 - 2 ’ ,  0 . 5  f o r  ‘ O c c ’ ,  a n d  0 . 1  f o r  ‘ N o n e ’ .  
^ P - v a l u e s  a n d  9 5 %  c o n f i d e n c e  i n t e r v a l s  f o r  d i f f e r e n c e s  b e t w e e n  s p e c i f i e d  t r e a t m e n t  g r o u p s  a n d  p l a c e b o  a r e  f r o m  a  M M R M  m o d e l  w i t h  
 t r e a t m e n t  g r o u p ,  v i s i t ,  t r e a t m e n t - b y  v i s i t  i n t e r a c t i o n ,  A A V  d i s e a s e  s t a t u s  ( n e w  o r  r e l a p s e d ) ,  a n d  A N C A  p o s i t i v i t y  ( M P O  o r  P R 3 )  a s    
 f a c t o r s  a n d  t h e  b a s e l i n e  v a l u e  a s  a  c o v a r i a t e .   L o g a r i t h m i c  t r a n s f o r m a t i o n s  w e r e  a p p l i e d  t o  t h e  d a t a  b e f o r e  f i t t i n g  t h e  A N C O V A  m o d e l .   
 T h e  9 5 %  c o n f i d e n c e  i n t e r v a l  w a s  t r a n s f o r m e d  b a c k  t o  t h e  o r i g i n a l  s c a l e .  
 
P r o g r a m  N a m e :  X X X X X X X X . s a s                   R u n  D a t e :  D D M M M Y Y Y Y  H H : M M                            D a t a b a s e  l a s t  m o d i f i e d :  D D M M Y Y Y Y  H H : M M  


 
P a ge 7 5  of 2 0 8 
 
T he f oll o wi n g t a bles w ill a la y o ut si milar Ta bl e 1 4. 2. 8. 1. 1: 
T a b l e  1 4 . 2 . 8 . 1 . 2  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  R B C *  C o u n t :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  H e m a t u r i a  a t  B a s e l i n e  a n d  R e c e i v i n g  C y c l o p h o s p h a m i d e  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 8 . 1 . 3  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  R B C *  C o u n t :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  H e m a t u r i a  a t  B a s e l i n e  a n d  R e c e i v i n g  R i t u x i m a b  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 8 . 1 . 4  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  R B C *  C o u n t :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  H e m a t u r i a  a t  B a s e l i n e  a n d  N e w l y  D i a g n o s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 8 . 1 . 5  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  R B C *  C o u n t :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  H e m a t u r i a  a t  B a s e l i n e  a n d  R e l a p s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 8 . 1 . 6  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  R B C *  C o u n t :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  H e m a t u r i a  a t  B a s e l i n e  a n d  M P O +  D i s e a s e  
 
T a b l e  1 4 . 2 . 8 . 1 . 7  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  R B C *  C o u n t :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  H e m a t u r i a  a t  B a s e l i n e  a n d  P R 3 +  D i s e a s e  
 
T a b l e  1 4 . 2 . 8 . 1 . 8  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  R B C *  C o u n t :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  H e m a t u r i a  a t  B a s e l i n e  a n d  G r a n u l o m a t o s i s  P o l y a n g i i t i s  ( W e g e n e r ’ s )  
 
T a b l e  1 4 . 2 . 8 . 1 . 9  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  R B C *  C o u n t :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  H e m a t u r i a  a t  B a s e l i n e  a n d  M i c r o s c o p i c  P o l y a n g i i t i s  


 
P a ge 7 6  of 2 0 8 
 
T a b l e  1 4 . 2 . 8 . 1 . 1 0  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  R B C *  C o u n t :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  H e m a t u r i a  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 8 . 1 . 1 1  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  R B C *  C o u n t :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  H e m a t u r i a  a t  B a s e l i n e  a n d  R e c e i v i n g  C y c l o p h o s p h a m i d e  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 8 . 1 . 1 2  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  R B C *  C o u n t :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  H e m a t u r i a  a t  B a s e l i n e  a n d  R e c e i v i n g  R i t u x i m a b  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 8 . 1 . 1 3  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  R B C *  C o u n t :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  H e m a t u r i a  a t  B a s e l i n e  a n d  N e w l y  D i a g n o s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 8 . 1 . 1 4  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  R B C *  C o u n t :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  H e m a t u r i a  a t  B a s e l i n e  a n d  R e l a p s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 8 . 1 . 1 5  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  R B C *  C o u n t :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  H e m a t u r i a  a t  B a s e l i n e  a n d  M P O +  D i s e a s e  
 
T a b l e  1 4 . 2 . 8 . 1 . 1 6  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  R B C *  C o u n t :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  H e m a t u r i a  a t  B a s e l i n e  a n d  P R 3 +  D i s e a s e  
 
T a b l e  1 4 . 2 . 8 . 1 . 1 7  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  R B C *  C o u n t :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  H e m a t u r i a  a t  B a s e l i n e  a n d  P o l y a n g i i t i s  ( W e g e n e r ’ s )  
 
T a b l e  1 4 . 2 . 8 . 1 . 1 8  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  R B C *  C o u n t :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  H e m a t u r i a  a t  B a s e l i n e  a n d  M i c r o s c o p i c  P o l y a n g i i t i s  


 
P a ge 7 7  of 2 0 8 
 
C h e m o C e n t r y x ,  I n c .  
P r o t o c o l  C L 0 0 2 _ 1 6 8                                                                                                          P a g e  1  o f  #  
T a b l e  1 4 . 2 . 8 . 1 . 1 9  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  U r i n a r y  R B C *  C o u n t :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e   
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  H e m a t u r i a  a t  B a s e l i n e  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
                           P l a c e b o  +                       C C X 1 6 8  +                     C C X 1 6 8  +  
                      F u l l  D o s e  P r e d n i s o n e            L o w  D o s e  P r e d n i s o n e             N o  P r e d n i s o n e                    A l l  C C X 1 6 8  
                             ( N = # # )                         ( N = # # )                       ( N = # # )                          ( N = # # )  
   S t u d y  D a y         - - - - - - - - - - - - - - - - - - - - - - - -       - - - - - - - - - - - - - - - - - - - - - - - -     - - - - - - - - - - - - - - - - - - - - - - - -       - - - - - - - - - - - - - - - - - - - - - - - - -  
    S t a t i s t i c        V i s i t     R a t i o   %  C h a n g e       V i s i t     R a t i o   %  C h a n g e     V i s i t     R a t i o   %  C h a n g e        V i s i t     R a t i o   %  C h a n g e  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
   B a s e l i n e   
    N ’               # #   
    M e a n             # # . # #  
    G e o .  M e a n  ( G M )   # # . # #  
    M i n i m u m          # # . #   
    M e d i a n           # # . # #  
    M a x i m u m          # # . #  
 
   D a y  2  
    N ’               # #        # #        # #           # #        # #        # #          # #        # #        # #           # #        # #        # #  
    M e a n             # # . # #     # # . # #     # # . # #        # # . # #     # # . # #     # # . # #       # # . # #     # # . # #     # # . # #        # # . # #     # # . # #     # # . # #  
    G e o .  M e a n  ( G M )   # # . # # #    # # . # # #                # # . # # #    # # . # # #               # # . # # #    # # . # # #                # # . # # #    # # . # # #  
    M i n i m u m          # # . #      # # . #      # # . #         # # . #      # # . #      # # . #        # # . #      # # . #      # # . #         # # . #      # # . #      # # . #  
    M e d i a n           # # . # #     # # . # #     # # . # #        # # . # #     # # . # #     # # . # #       # # . # #     # # . # #     # # . # #        # # . # #     # # . # #     # # . # #  
    M a x i m u m          # # . #      # # . #      # # . #         # # . #      # # . #      # # . #        # # . #      # # . #      # # . #         # # . #      # # . #      # # . #      
    P - v a l u e ^                                               # . # # # #                       # . # # # #                         # . # # # #  
    9 5 %  C I ^  f o r  R a t i o                                  ( - # . # # ,  # . # # )                ( - # . # # ,  # . # # )                  ( - # . # # ,  # . # # )        
 
  A d d i t i o n a l  v i s i t s  t o  b e  s u m m a r i z e d  i n c l u d e :  
     D a y  8 ,  D a y  1 5 ,  D a y  2 9 ,  D a y  4 3 ,  D a y  5 7 ,  D a y  7 1 ,  D a y  8 5 ,  E n d  o f  T r e a t m e n t ,  D a y  9 9 ,  D a y  1 1 3 ,  D a y  1 4 1 ,  D a y  1 6 9 ,   
     a n d  E n d  o f  F o l l o w - u p  P e r i o d .  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
-  B a s e l i n e  i s  d e f i n e d  a s  t h e  l a s t  p r e - d o s e  v a l u e .  
-  N ' = n u m b e r  o f  s u b j e c t s  w i t h  d a t a  a t  b a s e l i n e  a n d  t h e  s p e c i f i e d  v i s i t .  
-  T h e  E n d  o f  T r e a t m e n t  v a l u e  i s  t h e  l a s t  m e a s u r e m e n t  t h r o u g h  D a y  8 5 .  
-  T h e  E n d  o f  F o l l o w - u p  P e r i o d  i s  t h e  l a s t  m e a s u r e m e n t  a f t e r  D a y  8 5  t h r o u g h  D a y  1 6 9 .  
-  R a t i o  i s  d e f i n e d  f o r  e a c h  s u b j e c t  a s  t h e  v i s i t  v a l u e  d i v i d e d  b y  t h e  b a s e l i n e  v a l u e .  
*  S i n c e  u r i n a r y - R B C  v a l u e s  a r e  r e p o r t e d  a s  r a n g e s ,  q u a n t i t a t i v e  v a l u e s  a r e  d e f i n e d  a s :  7 5  f o r  r e s u l t  o f  ‘ > 7 5 ’ ,  5 0  f o r  ’ 5 0 - 7 5 ’ ,  
  3 0  f o r  ’ 3 0 - 4 9 ’ ,  1 6  f o r  ’ 1 6 - 2 9 ’ ,  1 0  f o r  ’ 1 0 - 1 5 ’ ,  6  f o r  ‘ 6 - 9 ’ ,  3  f o r  ‘ 3 - 5 ’ ,  1  f o r  ‘ 1 - 2 ’ ,  0 . 5  f o r  ‘ O c c ’ ,  a n d  0 . 1  f o r  ‘ N o n e ’ .  
^ P - v a l u e s  a n d  9 5 %  c o n f i d e n c e  i n t e r v a l s  f o r  d i f f e r e n c e s  b e t w e e n  s p e c i f i e d  t r e a t m e n t  g r o u p s  a n d  p l a c e b o  a r e  f r o m  A N C O V A  m o d e l s  w i t h  
  t r e a t m e n t  g r o u p ,  A A V  d i s e a s e  s t a t u s  ( n e w  o r  r e l a p s e d ) ,  a n d  A N C A  p o s i t i v i t y  ( M P O  o r  P R 3 ) a s  f a c t o r s  a n d  t h e  b a s e l i n e  v a l u e  a s  a   
  c o v a r i a t e .  L o g a r i t h m i c  t r a n s f o r m a t i o n s  w e r e  a p p l i e d  t o  t h e  d a t a  b e f o r e  f i t t i n g  t h e  A N C O V A  m o d e l .  T h e  9 5 %  c o n f i d e n c e  i n t e r v a l  w a s  
  t r a n s f o r m e d  b a c k  t o  t h e  o r i g i n a l  s c a l e .  
 
P r o g r a m  N a m e :  X X X X X X X X . s a s                   R u n  D a t e :  D D M M M Y Y Y Y  H H : M M                            D a t a b a s e  l a s t  m o d i f i e d :  D D M M Y Y Y Y  H H : M M  


 
P a ge 7 8  of 2 0 8 
 
 T he f oll o wi n g t a bles will ha ve t he sa me la y o ut as Ta ble 1 4. 2. 8. 1. 1 9: 
T a b l e  1 4 . 2 . 8 . 1 . 2 0  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  U r i n a r y  R B C *  C o u n t :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  –  P a t i e n t s  w i t h  H e m a t u r i a  a t  B a s e l i n e  a n d  R e c e i v i n g  C y c l o p h o s p h a m i d e  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 8 . 1 . 2 1  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  U r i n a r y  R B C *  C o u n t :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  H e m a t u r i a  a t  B a s e l i n e  a n d  R e c e i v i n g  R i t u x i m a b  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 8 . 1 . 2 2  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  U r i n a r y  R B C *  C o u n t :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  H e m a t u r i a  a t  B a s e l i n e  a n d  N e w l y  D i a g n o s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 8 . 1 . 2 3  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  U r i n a r y  R B C *  C o u n t :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  H e m a t u r i a  a t  B a s e l i n e  a n d  R e l a p s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 8 . 1 . 2 4  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  U r i n a r y  R B C *  C o u n t :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  H e m a t u r i a  a t  B a s e l i n e  a n d  a n d  M P O +  D i s e a s e  
 
T a b l e  1 4 . 2 . 8 . 1 . 2 5  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  U r i n a r y  R B C *  C o u n t :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  H e m a t u r i a  a t  B a s e l i n e  a n d  P R 3 +  D i s e a s e  
 
T a b l e  1 4 . 2 . 8 . 1 . 2 6  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  U r i n a r y  R B C *  C o u n t :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  H e m a t u r i a  a t  B a s e l i n e  a n d  G r a n u l o m a t o s i s  w i t h  P o l y a n g i i t i s  ( W e g e n e r ’ s )  
 
T a b l e  1 4 . 2 . 8 . 1 . 2 7  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  U r i n a r y  R B C *  C o u n t :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  H e m a t u r i a  a t  B a s e l i n e  a n d  M i c r o s c o p i c  P o l y a n g i i t i s  


 
P a ge 7 9  of 2 0 8 
 
C h e m o C e n t r y x ,  I n c .  
P r o t o c o l  C L 0 0 2 _ 1 6 8                                                                                                          P a g e  1  o f  #  
T a b l e  1 4 . 2 . 8 . 2 . 1  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  R B C *  C o u n t :  R a t i o  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  H e m a t u r i a  a t  B a s e l i n e  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
                  = = = = = = = = = = = = = = =  S t e p  1  a n d  2  C o m b i n e d  = = = = = = = = = = = = = = =        = = = = = = = = = = = = = = = = = = = = = = =  S t e p  3  = = = = = = = = = = = = = = = = = = = = = = =  
                     P l a c e b o  +            C C X 1 6 8  +                                  P l a c e b o  +          C C X 1 6 8  +  
                     F u l l  D o s e            L o w  D o s e             C C X 1 6 8  +              F u l l  D o s e          L o w  D o s e             C C X 1 6 8  +   
                     P r e d n i s o n e          P r e d n i s o n e         N o  P r e d n i s o n e            P r e d n i s o n e        P r e d n i s o n e         N o  P r e d n i s o n e  
                       ( N = # # )             ( N = # # )               ( N = # # )                 ( N = # # )             ( N = # # )               ( N = # # )  
   S t u d y  D a y       - - - - - - - - - - - - - - - -    - - - - - - - - - - - - - - - -    - - - - - - - - - - - - - - - -       - - - - - - - - - - - - - - - -    - - - - - - - - - - - - - - - -    - - - - - - - - - - - - - - - -  
    S t a t i s t i c       V i s i t     R a t i o      V i s i t     R a t i o      V i s i t     R a t i o         V i s i t     R a t i o      V i s i t     R a t i o      V i s i t     R a t i o  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
   B a s e l i n e   
    N ’               # #   
    M e a n             # # . # #  
    G e o .  M e a n  ( G M )   # # . # #  
    M i n i m u m          # # . #   
    M e d i a n           # # . # #  
    M a x i m u m          # # . #  
 
   D a y  2  
    N ’              # #         # #        # #          # #        # #        # #           # #        # #         # #        # #         # #        # #  
    M e a n            # # . #       # # . #      # # . #        # # . #      # # . #      # # . #         # # . #      # # . #       # # . #      # # . #       # # . #      # # . #  
    G e o .  M e a n  ( G M )  # # . # #      # # . # #     # # . # #       # # . # #     # # . # #     # # . # #        # # . # #     # # . # #      # # . # #     # # . # #      # # . # #     # # . # #  
    M i n i m u m         # #         # #        # #          # #        # #        # #           # #        # #         # #        # #         # #        # #  
    M e d i a n          # # . #       # # . #      # # . #        # # . #      # # . #      # # . #         # # . #      # # . #       # # . #      # # . #       # # . #      # # . #  
    M a x i m u m         # #         # #        # #          # #        # #        # #           # #        # #         # #        # #         # #        # #  
    P - v a l u e ^                                    # . # # # #             # . # # # #                                   # . # # # #              # . # # # #  
    9 5 %  C I ^  f o r  R a t i o                       ( - # . # # ,  # . # # )      ( - # . # # ,  # . # # )                            ( - # . # # ,  # . # # )       ( - # . # # ,  # . # # )  
    
 T h e  a d d i t i o n a l  v i s i t s  t o  b e  s u m m a r i z e d  i n c l u d e :  
     D a y  8 ,  D a y  1 5 ,  D a y  2 9 ,  D a y  4 3 ,  D a y  5 7 ,  D a y  7 1 ,  D a y  8 5  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
-  B a s e l i n e  i s  d e f i n e d  a s  t h e  l a s t  p r e - d o s e  v a l u e .  
-  N ' = n u m b e r  o f  s u b j e c t s  w i t h  d a t a  a t  b a s e l i n e  a n d  t h e  s p e c i f i e d  v i s i t .  
*  S i n c e  u r i n a r y - R B C  v a l u e s  a r e  r e p o r t e d  a s  r a n g e s ,  q u a n t i t a t i v e  v a l u e s  a r e  d e f i n e d  a s :  7 5  f o r  r e s u l t  o f  ‘ > 7 5 ’ ,  5 0  f o r  ’ 5 0 - 7 5 ’ ,  
  3 0  f o r  ’ 3 0 - 4 9 ’ ,  1 6  f o r  ’ 1 6 - 2 9 ’ ,  1 0  f o r  ’ 1 0 - 1 5 ’ ,  6  f o r  ‘ 6 - 9 ’ ,  3  f o r  ‘ 3 - 5 ’ ,  1  f o r  ‘ 1 - 2 ’ ,  0 . 5  f o r  ‘ O c c ’ ,  a n d  0 . 1  f o r  ‘ N o n e ’ .  
^ P - v a l u e s  a n d  9 5 %  c o n f i d e n c e  i n t e r v a l s  f o r  d i f f e r e n c e s  b e t w e e n  s p e c i f i e d  t r e a t m e n t  g r o u p s  a n d  p l a c e b o  a r e  f r o m  M M R M  m o d e l s  w i t h  
  t r e a t m e n t  g r o u p ,  v i s i t ,  a n d  t r e a t m e n t - b y - v i s i t  a s  f a c t o r s  a n d  t h e  b a s e l i n e  v a l u e  a s  a  c o v a r i a t e .   T h e  m o d e l  f o r  a n a l y s i s  o f  S t e p  3  
  i n c l u d e d  f a c t o r s  f o r  A A V  d i s e a s e  s t a t u s  ( n e w  o r  r e l a p s e d ) ,  A N C A  p o s i t i v i t y  ( M P O  o r  P R 3 ) ,  a n d  s t a n d a r d  o f  c a r e  t r e a t m e n t  ( r i t u x i m a b   
  o r  c y c l o p h o s p h a m i d e .   T h e  m o d e l  f o r  S t e p  1  +  S t e p  2  i n c l u d e d  a  f a c t o r  f o r  d i s e a s e  s t a t u s . a s  f a c t o r s  a n d  t h e  b a s e l i n e  v a l u e  a s  a   
  c o v a r i a t e .  L o g a r i t h m i c  t r a n s f o r m a t i o n s  w e r e  a p p l i e d  t o  t h e  d a t a  b e f o r e  f i t t i n g  t h e  A N C O V A  m o d e l .  T h e  9 5 %  c o n f i d e n c e  i n t e r v a l  w a s  
  t r a n s f o r m e d  b a c k  t o  t h e  o r i g i n a l  s c a l e .  
 
P r o g r a m  N a m e :  X X X X X X X X . s a s                   R u n  D a t e :  D D M M M Y Y Y Y  H H : M M                            D a t a b a s e  l a s t  m o d i f i e d :  D D M M Y Y Y Y  H H : M M  


 
P a ge 8 0  of 2 0 8 
 
T he f oll o wi n g t a bles w ill ha ve a la y o ut si milar t o Ta ble 1 4. 2. 8. 2. 1: 
T a b l e  1 4 . 2 . 8 . 2 . 2  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  R B C *  C o u n t :  R a t i o  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  H e m a t u r i a  a t  B a s e l i n e  a n d  R e c e i v i n g  C y c l o p h o s p h a m i d e  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 8 . 2 . 3  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  R B C *  C o u n t :  R a t i o  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  H e m a t u r i a  a t  B a s e l i n e  a n d  R e c e i v i n g  R i t u x i m a b  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 8 . 2 . 4  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  R B C *  C o u n t :  R a t i o  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  H e m a t u r i a  a t  B a s e l i n e  a n d  N e w l y  D i a g n o s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 8 . 2 . 5  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  R B C *  C o u n t :  R a t i o  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  H e m a t u r i a  a t  B a s e l i n e  a n d  R e l a p s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 8 . 2 . 6  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  R B C *  C o u n t :  R a t i o  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  H e m a t u r i a  a t  B a s e l i n e  a n d  M P O +  D i s e a s e  
 
T a b l e  1 4 . 2 . 8 . 2 . 7  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  R B C *  C o u n t :  R a t i o  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  H e m a t u r i a  a t  B a s e l i n e  a n d  P R 3 +  D i s e a s e  
 
T a b l e  1 4 . 2 . 8 . 2 . 8  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  R B C *  C o u n t :  R a t i o  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  H e m a t u r i a  a t  B a s e l i n e  a n d  G r a n u l o m a t o s i s  w i t h  P o l y a n g i i t i s  ( W e g e n e r ’ s )  
 
T a b l e  1 4 . 2 . 8 . 2 . 9  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  R B C *  C o u n t :  R a t i o  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  H e m a t u r i a  a t  B a s e l i n e  a n d  M i c r o s c o p i c  P o l y a n g i i t i s  


 
P a ge 8 1  of 2 0 8 
 
T a b l e  1 4 . 2 . 8 . 2 . 1 0  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  R B C *  C o u n t :  R a t i o  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  H e m a t u r i a  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 8 . 2 . 1 1  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  R B C *  C o u n t :  R a t i o  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  H e m a t u r i a  a t  B a s e l i n e  a n d  R e c e i v i n g  C y c l o p h o s p h a m i d e  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 8 . 2 . 1 2  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  R B C *  C o u n t :  R a t i o  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  H e m a t u r i a  a t  B a s e l i n e  a n d  R e c e i v i n g  R i t u x i m a b  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 8 . 2 . 1 3  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  R B C *  C o u n t :  R a t i o  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  H e m a t u r i a  a t  B a s e l i n e  a n d  N e w l y  D i a g n o s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 8 . 2 . 1 4  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  R B C *  C o u n t :  R a t i o  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  H e m a t u r i a  a t  B a s e l i n e  a n d  R e l a p s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 8 . 2 . 1 5  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  R B C *  C o u n t :  R a t i o  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  H e m a t u r i a  a t  B a s e l i n e  a n d  M P O +  D i s e a s e  
 
T a b l e  1 4 . 2 . 8 . 2 . 1 6  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  R B C *  C o u n t :  R a t i o  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  H e m a t u r i a  a t  B a s e l i n e  a n d  P R 3 +  D i s e a s e  
 
T a b l e  1 4 . 2 . 8 . 2 . 1 7  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  R B C *  C o u n t :  R a t i o  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  H e m a t u r i a  a t  B a s e l i n e  a n d  G r a n u l o m a t o s i s  w i t h  P o l y a n g i i t i s  ( W e g e n e r ’ s )  
 
T a b l e  1 4 . 2 . 8 . 2 . 1 8  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  R B C *  C o u n t :  R a t i o  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  H e m a t u r i a  a t  B a s e l i n e  a n d  M i c r o s c o p i c  P o l y a n g i i t i s  


 
P a ge 8 2  of 2 0 8 
 
C h e m o C e n t r y x ,  I n c .  
P r o t o c o l  C L 0 0 2 _ 1 6 8                                                                                                          P a g e  1  o f  #  
T a b l e  1 4 . 2 . 8 . 3 . 1  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  R B C *  C o u n t :  R a t i o  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  H e m a t u r i a  a t  B a s e l i n e  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
  S t e p                       P l a c e b o  +                       C C X 1 6 8  +                     C C X 1 6 8  +  
   S t u d y            - -  F u l l  D o s e  P r e d n i s o n e  - -      - -  L o w  D o s e  P r e d n i s o n e  - -    - - - - -  N o  P r e d n i s o n e  - - - - -       - - - - - - -  A l l  C C X 1 6 8  - - - - - -  
    S t a t i s t i c        V i s i t     R a t i o   %  C h a n g e       V i s i t     R a t i o   %  C h a n g e     V i s i t     R a t i o   %  C h a n g e        V i s i t     R a t i o   %  C h a n g e  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
  S t e p  3                       N = # #                           N = # #                         N = # #                            N = # #  
 
   B a s e l i n e   
    N ’               # #   
    M e a n             # # . # #  
    G e o .  M e a n  ( G M )   # # . # #  
    M i n i m u m          # # . #   
    M e d i a n           # # . # #  
    M a x i m u m          # # . #  
 
   D a y  2  
    N ’               # #        # #        # #           # #        # #        # #          # #        # #        # #           # #        # #        # #  
    M e a n             # # . # #     # # . # #     # # . # #        # # . # #     # # . # #     # # . # #       # # . # #     # # . # #     # # . # #        # # . # #     # # . # #     # # . # #  
    G e o .  M e a n  ( G M )   # # . # # #    # # . # # #                # # . # # #    # # . # # #               # # . # # #    # # . # # #                # # . # # #    # # . # # #  
    M i n i m u m          # # . #      # # . #      # # . #         # # . #      # # . #      # # . #        # # . #      # # . #      # # . #         # # . #      # # . #      # # . #  
    M e d i a n           # # . # #     # # . # #     # # . # #        # # . # #     # # . # #     # # . # #       # # . # #     # # . # #     # # . # #        # # . # #     # # . # #     # # . # #  
    M a x i m u m          # # . #      # # . #      # # . #         # # . #      # # . #      # # . #        # # . #      # # . #      # # . #         # # . #      # # . #      # # . #  
    P - v a l u e ^                                               # . # # # #                       # . # # # #                         # . # # # #  
    9 5 %  C I ^  f o r  R a t i o                                  ( - # . # # ,  # . # # )                ( - # . # # ,  # . # # )                  ( - # . # # ,  # . # # )  
 
R e p e a t  f o r  S t e p s  2  a n d  1 .   O n l y  d i s p l a y  p - v a l u e s  a n d  c o n f i d e n c e  i n t e r v a l s  f o r  S t e p  3 .  
 
  A d d i t i o n a l  v i s i t s  i n c l u d e  D a y  2  8 ,  1 5 ,  2 9 ,  4 3 ,  5 7 ,  7 1 ,  a n d  8 5 .  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
-  B a s e l i n e  i s  d e f i n e d  a s  t h e  l a s t  p r e - d o s e  v a l u e .  
-  N ' = n u m b e r  o f  s u b j e c t s  w i t h  d a t a  a t  b a s e l i n e  a n d  t h e  s p e c i f i e d  v i s i t .  
-  R a t i o  i s  d e f i n e d  f o r  e a c h  s u b j e c t  a s  t h e  v i s i t  v a l u e  d i v i d e d  b y  t h e  b a s e l i n e  v a l u e .  
*  S i n c e  u r i n a r y - R B C  v a l u e s  a r e  r e p o r t e d  a s  r a n g e s ,  q u a n t i t a t i v e  v a l u e s  a r e  d e f i n e d  a s :  7 5  f o r  r e s u l t  o f  ‘ > 7 5 ’ ,  5 0  f o r  ’ 5 0 - 7 5 ’ ,  
  3 0  f o r  ’ 3 0 - 4 9 ’ ,  1 6  f o r  ’ 1 6 - 2 9 ’ ,  1 0  f o r  ’ 1 0 - 1 5 ’ ,  6  f o r  ‘ 6 - 9 ’ ,  3  f o r  ‘ 3 - 5 ’ ,  1  f o r  ‘ 1 - 2 ’ ,  0 . 5  f o r  ‘ O c c ’ ,  a n d  0 . 1  f o r  ‘ N o n e ’ .  
^ P - v a l u e s  a n d  9 5 %  c o n f i d e n c e  i n t e r v a l s  f o r  d i f f e r e n c e s  b e t w e e n  s p e c i f i e d  t r e a t m e n t  g r o u p s  a n d  p l a c e b o  a r e  f r o m  a  M M R M  m o d e l s  w i t h  
  t r e a t m e n t  g r o u p  a n d  r a n d o m i z a t i o n  s t r a t a  ( A A V  s t a t u s ,  A N C A  p o s i t i v i t y ,  a n d  s t a n d a r d  o f  c a r e  t r e a t m e n t )  a s  f a c t o r s  a n d  t h e  b a s e l i n e   
  v a l u e  a s  a  c o v a r i a t e .  L o g a r i t h m i c  t r a n s f o r m a t i o n s  w e r e  a p p l i e d  t o  t h e  d a t a  b e f o r e  f i t t i n g  t h e  A N C O V A  m o d e l .  T h e  9 5 %  c o n f i d e n c e  
  i n t e r v a l  w a s  t r a n s f o r m e d  b a c k  t o  t h e  o r i g i n a l  s c a l e .  
 
P r o g r a m  N a m e :  X X X X X X X X . s a s                   R u n  D a t e :  D D M M M Y Y Y Y  H H : M M                            D a t a b a s e  l a s t  m o d i f i e d :  D D M M Y Y Y Y  H H : M M  


 
P a ge 8 3  of 2 0 8 
 
T he f oll o wi n g t a bles w ill ha ve la y o uts si milar t o Ta ble 1 4. 2. 8. 3. 1: 
T a b l e  1 4 . 2 . 8 . 3 . 2  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  R B C *  C o u n t :  R a t i o  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  H e m a t u r i a  a t  B a s e l i n e  a n d  R e c e i v i n g  C y c l o p h o s p h a m i d e  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 8 . 3 . 3  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  R B C *  C o u n t :  R a t i o  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  H e m a t u r i a  a t  B a s e l i n e  a n d  R e c e i v i n g  R i t u x i m a b  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 8 . 3 . 4  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  R B C *  C o u n t :  R a t i o  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  P a t i e n t s  w i t h  H e m a t u r i a  a t  B a s e l i n e  a n d  N e w l y  D i a g n o s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 8 . 3 . 5  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  R B C *  C o u n t :  R a t i o  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  H e m a t u r i a  a t  B a s e l i n e  a n d  R e l a p s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 8 . 3 . 6  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  R B C *  C o u n t :  R a t i o  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  H e m a t u r i a  a t  B a s e l i n e  a n d  M P O +  D i s e a s e  
 
T a b l e  1 4 . 2 . 8 . 3 . 7  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  R B C *  C o u n t :  R a t i o  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  H e m a t u r i a  a t  B a s e l i n e  a n d  P R 3 +  D i s e a s e  
 
T a b l e  1 4 . 2 . 8 . 3 . 8  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  R B C *  C o u n t :  R a t i o  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  H e m a t u r i a  a t  B a s e l i n e  a n d  P o l y a n g i i t i s  ( W e g e n e r ’ s )  
 
T a b l e  1 4 . 2 . 8 . 3 . 9  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  R B C *  C o u n t :  R a t i o  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  H e m a t u r i a  a t  B a s e l i n e  a n d  M i c r o s c o p i c  P o l y a n g i i t i s  


 
P a ge 8 4  of 2 0 8 
 
T a b l e  1 4 . 2 . 8 . 3 . 1 0  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  R B C *  C o u n t :  R a t i o  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  H e m a t u r i a  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 8 . 3 . 1 1  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  R B C *  C o u n t :  R a t i o  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  H e m a t u r i a  a t  B a s e l i n e  a n d  R e c e i v i n g  C y c l o p h o s p h a m i d e  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 8 . 3 . 1 2  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  R B C *  C o u n t :  R a t i o  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  H e m a t u r i a  a t  B a s e l i n e  a n d  R e c e i v i n g  R i t u x i m a b  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 8 . 3 . 1 3  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  R B C *  C o u n t :  R a t i o  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  P a t i e n t s  w i t h  H e m a t u r i a  a t  B a s e l i n e  a n d  N e w l y  D i a g n o s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 8 . 3 . 1 4  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  R B C *  C o u n t :  R a t i o  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  H e m a t u r i a  a t  B a s e l i n e  a n d  R e l a p s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 8 . 3 . 1 5  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  R B C *  C o u n t :  R a t i o  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  H e m a t u r i a  a t  B a s e l i n e  a n d  M P O +  D i s e a s e  
 
T a b l e  1 4 . 2 . 8 . 3 . 1 6  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  R B C *  C o u n t :  R a t i o  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  H e m a t u r i a  a t  B a s e l i n e  a n d  P R 3 +  D i s e a s e  
 
T a b l e  1 4 . 2 . 8 . 3 . 1 7  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  R B C *  C o u n t :  R a t i o  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  H e m a t u r i a  a t  B a s e l i n e  a n d  P o l y a n g i i t i s  ( W e g e n e r ’ s )  
 
T a b l e  1 4 . 2 . 8 . 3 . 1 8  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  R B C *  C o u n t :  R a t i o  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  H e m a t u r i a  a t  B a s e l i n e  a n d  M i c r o s c o p i c  P o l y a n g i i t i s  


 
P a ge 8 5  of 2 0 8 
 
T he f oll o wi n g t a bles w ill ha ve la y o uts si milar t o t o Ta bles 1 4. 2. 8. x 
T a b l e  1 4 . 2 . 9 . 1 . 1  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A C R :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  A l b u m i n u r i a  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 9 . 1 . 2  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A C R :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  A l b u m i n u r i a  a t  B a s e l i n e  a n d  R e c e i v i n g  C y c l o p h o s p h a m i d e  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 9 . 1 . 3  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A C R :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  A l b u m i n u r i a  a t  B a s e l i n e  a n d  R e c e i v i n g  R i t u x i m a b  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 9 . 1 . 4  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A C R :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  A l b u m i n u r i a  a t  B a s e l i n e  a n d  N e w l y  D i a g n o s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 9 . 1 . 5  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A C R :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  A l b u m i n u r i a  a t  B a s e l i n e  a n d  R e l a p s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 9 . 1 . 6  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A C R :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  A l b u m i n u r i a  a t  B a s e l i n e  a n d  M P O +  D i s e a s e  
 
T a b l e  1 4 . 2 . 9 . 1 . 7  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A C R :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  A l b u m i n u r i a  a t  B a s e l i n e  a n d  P R 3 +  D i s e a s e  
 
T a b l e  1 4 . 2 . 9 . 1 . 8  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A C R :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  A l b u m i n u r i a  a t  B a s e l i n e  a n d  G r a n u l o m a t o s i s  P o l y a n g i i t i s  ( W e g e n e r ’ s )  
 
T a b l e  1 4 . 2 . 9 . 1 . 9  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A C R :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  A l b u m i n u r i a  a t  B a s e l i n e  a n d  M i c r o s c o p i c  P o l y a n g i i t i s  


 
P a ge 8 6  of 2 0 8 
 
T a b l e  1 4 . 2 . 9 . 1 . 1 0  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A C R :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  A l b u m i n u r i a  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 9 . 1 . 1 1  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A C R :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  A l b u m i n u r i a  a t  B a s e l i n e  a n d  R e c e i v i n g  C y c l o p h o s p h a m i d e  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 9 . 1 . 1 2  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A C R :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  A l b u m i n u r i a  a t  B a s e l i n e  a n d  R e c e i v i n g  R i t u x i m a b  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 9 . 1 . 1 3  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A C R :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  A l b u m i n u r i a  a t  B a s e l i n e  a n d  N e w l y  D i a g n o s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 9 . 1 . 1 4  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A C R :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  A l b u m i n u r i a  a t  B a s e l i n e  a n d  R e l a p s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 9 . 1 . 1 5  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A C R :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  A l b u m i n u r i a  a t  B a s e l i n e  a n d  M P O +  D i s e a s e  
 
T a b l e  1 4 . 2 . 9 . 1 . 1 6  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A C R :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  A l b u m i n u r i a  a t  B a s e l i n e  a n d  P R 3 +  D i s e a s e  
 
T a b l e  1 4 . 2 . 9 . 1 . 1 7  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A C R :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  A l b u m i n u r i a  a t  B a s e l i n e  a n d  P o l y a n g i i t i s  ( W e g e n e r ’ s )  
 
T a b l e  1 4 . 2 . 9 . 1 . 1 8  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A C R :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  A l b u m i n u r i a  a t  B a s e l i n e  a n d  M i c r o s c o p i c  P o l y a n g i i t i s  


 
P a ge 8 7  of 2 0 8 
 
T a b l e  1 4 . 2 . 9 . 1 . 1 9  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  A C R :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  A l b u m i n u r i a  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 9 . 1 . 2 0  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  A C R :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  A l b u m i n u r i a  a t  B a s e l i n e  a n d  R e c e i v i n g  C y c l o p h o s p h a m i d e  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 9 . 1 . 2 1  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  A C R :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  A l b u m i n u r i a  a t  B a s e l i n e  a n d  R e c e i v i n g  R i t u x i m a b  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 9 . 1 . 2 2  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  A C R :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  A l b u m i n u r i a  a t  B a s e l i n e  a n d  N e w l y  D i a g n o s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 9 . 1 . 2 3  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  A C R :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  A l b u m i n u r i a  a t  B a s e l i n e  a n d  R e l a p s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 9 . 1 . 2 4  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  A C R :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  A l b u m i n u r i a  a t  B a s e l i n e  a n d  M P O +  D i s e a s e  
 
T a b l e  1 4 . 2 . 9 . 1 . 2 5  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  A C R :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  A l b u m i n u r i a  a t  B a s e l i n e  a n d  P R 3 +  D i s e a s e  
 
T a b l e  1 4 . 2 . 9 . 1 . 2 6  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  A C R :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  A l b u m i n u r i a  a t  B a s e l i n e  a n d  w i t h  G r a n u l o m a t o s i s  P o l y a n g i i t i s  ( W e g e n e r ’ s )  
 
T a b l e  1 4 . 2 . 9 . 1 . 2 7  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  A C R :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  A l b u m i n u r i a  a t  B a s e l i n e  a n d  M i c r o s c o p i c  P o l y a n g i i t i s  


 
P a ge 8 8  of 2 0 8 
 
T a b l e  1 4 . 2 . 9 . 2 . 1  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A C R :  R a t i o  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  A l b u m i n u r i a  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 9 . 2 . 2  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A C R :  R a t i o  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  A l b u m i n u r i a  a t  B a s e l i n e  a n d  R e c e i v i n g  C y c l o p h o s p h a m i d e  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 9 . 2 . 3  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A C R :  R a t i o  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  A l b u m i n u r i a  a t  B a s e l i n e  a n d  R e c e i v i n g  R i t u x i m a b  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 9 . 2 . 4  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A C R :  R a t i o  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  A l b u m i n u r i a  a t  B a s e l i n e  a n d  N e w l y  D i a g n o s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 9 . 2 . 5  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A C R :  R a t i o  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  A l b u m i n u r i a  a t  B a s e l i n e  a n d  R e l a p s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 9 . 2 . 6  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A C R :  R a t i o  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  A l b u m i n u r i a  a t  B a s e l i n e  a n d  M P O +  D i s e a s e  
 
T a b l e  1 4 . 2 . 9 . 2 . 7  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A C R :  R a t i o  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  A l b u m i n u r i a  a t  B a s e l i n e  a n d  P R 3 +  D i s e a s e  
 
T a b l e  1 4 . 2 . 9 . 2 . 8  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A C R :  R a t i o  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  A l b u m i n u r i a  a t  B a s e l i n e  a n d  G r a n u l o m a t o s i s  w i t h  P o l y a n g i i t i s  ( W e g e n e r ’ s )  
 
T a b l e  1 4 . 2 . 9 . 2 . 9  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A C R :  R a t i o  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  A l b u m i n u r i a  a t  B a s e l i n e  a n d  M i c r o s c o p i c  P o l y a n g i i t i s  


 
P a ge 8 9  of 2 0 8 
 
T a b l e  1 4 . 2 . 9 . 2 . 1 0  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A C R :  R a t i o  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  A l b u m i n u r i a  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 9 . 2 . 1 1  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A C R :  R a t i o  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  A l b u m i n u r i a  a t  B a s e l i n e  a n d  R e c e i v i n g  C y c l o p h o s p h a m i d e  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 9 . 2 . 1 2  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A C R :  R a t i o  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  A l b u m i n u r i a  a t  B a s e l i n e  a n d  R e c e i v i n g  R i t u x i m a b  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 9 . 2 . 1 3  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A C R :  R a t i o  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  A l b u m i n u r i a  a t  B a s e l i n e  a n d  N e w l y  D i a g n o s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 9 . 2 . 1 4  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A C R :  R a t i o  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  A l b u m i n u r i a  a t  B a s e l i n e  a n d  R e l a p s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 9 . 2 . 1 5  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A C R :  R a t i o  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  A l b u m i n u r i a  a t  B a s e l i n e  a n d  M P O +  D i s e a s e  
 
T a b l e  1 4 . 2 . 9 . 2 . 1 6  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A C R :  R a t i o  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  A l b u m i n u r i a  a t  B a s e l i n e  a n d  P R 3 +  D i s e a s e  
 
T a b l e  1 4 . 2 . 9 . 2 . 1 7  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A C R :  R a t i o  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  A l b u m i n u r i a  a t  B a s e l i n e  a n d  G r a n u l o m a t o s i s  w i t h  P o l y a n g i i t i s  ( W e g e n e r ’ s )  
 
T a b l e  1 4 . 2 . 9 . 2 . 1 8  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A C R :  R a t i o  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  A l b u m i n u r i a  a t  B a s e l i n e  a n d  M i c r o s c o p i c  P o l y a n g i i t i s  


 
P a ge 9 0  of 2 0 8 
 
T a b l e  1 4 . 2 . 9 . 3 . 1  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A C R :  R a t i o  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  A l b u m i n u r i a  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 9 . 3 . 2  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A C R :  R a t i o  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  A l b u m i n u r i a  a t  B a s e l i n e  a n d  R e c e i v i n g  C y c l o p h o s p h a m i d e  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 9 . 3 . 3  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A C R :  R a t i o  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  A l b u m i n u r i a  a t  B a s e l i n e  a n d  R e c e i v i n g  R i t u x i m a b  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 9 . 3 . 4  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A C R :  R a t i o  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  P a t i e n t s  w i t h  A l b u m i n u r i a  a t  B a s e l i n e  a n d  N e w l y  D i a g n o s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 9 . 3 . 5  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A C R :  R a t i o  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  A l b u m i n u r i a  a t  B a s e l i n e  a n d  R e l a p s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 9 . 3 . 6  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A C R :  R a t i o  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  A l b u m i n u r i a  a t  B a s e l i n e  a n d  M P O +  D i s e a s e  
 
T a b l e  1 4 . 2 . 9 . 3 . 7  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A C R :  R a t i o  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  A l b u m i n u r i a  a t  B a s e l i n e  a n d  P R 3 +  D i s e a s e  
 
T a b l e  1 4 . 2 . 9 . 3 . 8  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A C R :  R a t i o  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  A l b u m i n u r i a  a t  B a s e l i n e  a n d  P o l y a n g i i t i s  ( W e g e n e r ’ s )  
 
T a b l e  1 4 . 2 . 9 . 3 . 9  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A C R :  R a t i o  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  A l b u m i n u r i a  a t  B a s e l i n e  a n d  M i c r o s c o p i c  P o l y a n g i i t i s  


 
P a ge 9 1  of 2 0 8 
 
T a b l e  1 4 . 2 . 9 . 3 . 1 0  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A C R :  R a t i o  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  A l b u m i n u r i a  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 9 . 3 . 1 1  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A C R :  R a t i o  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  A l b u m i n u r i a  a t  B a s e l i n e  a n d  R e c e i v i n g  C y c l o p h o s p h a m i d e  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 9 . 3 . 1 2  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A C R :  R a t i o  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  A l b u m i n u r i a  a t  B a s e l i n e  a n d  R e c e i v i n g  R i t u x i m a b  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 9 . 3 . 1 3  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A C R :  R a t i o  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  P a t i e n t s  w i t h  A l b u m i n u r i a  a t  B a s e l i n e  a n d  N e w l y  D i a g n o s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 9 . 3 . 1 4  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A C R :  R a t i o  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  A l b u m i n u r i a  a t  B a s e l i n e  a n d  R e l a p s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 9 . 3 . 1 5  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A C R :  R a t i o  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  A l b u m i n u r i a  a t  B a s e l i n e  a n d  M P O +  D i s e a s e  
 
T a b l e  1 4 . 2 . 9 . 3 . 1 6  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A C R :  R a t i o  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  A l b u m i n u r i a  a t  B a s e l i n e  a n d  P R 3 +  D i s e a s e  
 
T a b l e  1 4 . 2 . 9 . 3 . 1 7  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A C R :  R a t i o  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  A l b u m i n u r i a  a t  B a s e l i n e  a n d  P o l y a n g i i t i s  ( W e g e n e r ’ s )  
 
T a b l e  1 4 . 2 . 9 . 3 . 1 8  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A C R :  R a t i o  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  P a t i e n t s  w i t h  A l b u m i n u r i a  a t  B a s e l i n e  a n d  M i c r o s c o p i c  P o l y a n g i i t i s  


 
P a ge 9 2  of 2 0 8 
 
T he f oll o wi n g t a bles w ill ha ve la y o uts si milar t o t o Ta bles 1 4. 2. 8. x 
T a b l e  1 4 . 2 . 1 0 . 1 . 1  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  M C P - 1 : C r e a t i n i n e  R a t i o :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  
 
T a b l e  1 4 . 2 . 1 0 . 1 . 2  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  M C P - 1 : C r e a t i n i n e  R a t i o :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  –  A l l  P a t i e n t s  w i t h  R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 1 0 . 1 . 3  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  M C P - 1 : C r e a t i n i n e  R a t i o :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  –  A l l  P a t i e n t s  w i t h  N o n - R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 1 0 . 1 . 4  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  M C P - 1 : C r e a t i n i n e  R a t i o :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  –  A l l  P a t i e n t s  R e c e i v i n g  C y c l o p h o s p h a m i d e  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 1 0 . 1 . 5  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  M C P - 1 : C r e a t i n i n e  R a t i o :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  –  A l l  P a t i e n t s  R e c e i v i n g  R i t u x i m a b  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 1 0 . 1 . 6  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  M C P - 1 : C r e a t i n i n e  R a t i o :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  –  A l l  P a t i e n t s  w i t h  N e w l y  D i a g n o s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 0 . 1 . 7  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  M C P - 1 : C r e a t i n i n e  R a t i o :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  –  A l l  P a t i e n t s  w i t h  R e l a p s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 0 . 1 . 8  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  M C P - 1 : C r e a t i n i n e  R a t i o :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  –  A l l  P a t i e n t s  w i t h  M P O +  D i s e a s e  


 
P a ge 9 3  of 2 0 8 
 
T a b l e  1 4 . 2 . 1 0 . 1 . 9  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  M C P - 1 : C r e a t i n i n e  R a t i o :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  –  A l l  P a t i e n t s  w i t h  P R 3 +  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 0 . 1 . 1 0  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  M C P - 1 : C r e a t i n i n e  R a t i o :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  –  A l l  P a t i e n t s  w i t h  G r a n u l o m a t o s i s  P o l y a n g i i t i s  ( W e g e n e r ’ s )  
 
T a b l e  1 4 . 2 . 1 0 . 1 . 1 1  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  M C P - 1 : C r e a t i n i n e  R a t i o :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  –  A l l  P a t i e n t s  w i t h  M i c r o s c o p i c  P o l y a n g i i t i s  


 
P a ge 9 4  of 2 0 8 
 
T a b l e  1 4 . 2 . 1 0 . 1 . 1 2  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  M C P - 1 : C r e a t i n i n e  R a t i o :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  
 
T a b l e  1 4 . 2 . 1 0 . 1 . 1 3  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  M C P - 1 : C r e a t i n i n e  R a t i o :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  –  A l l  P a t i e n t s  w i t h  R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 1 0 . 1 . 1 4  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  M C P - 1 : C r e a t i n i n e  R a t i o :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  –  A l l  P a t i e n t s  w i t h  N o n - R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 1 0 . 1 . 1 5  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  M C P - 1 : C r e a t i n i n e  R a t i o :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  –  A l l  P a t i e n t s  R e c e i v i n g  C y c l o p h o s p h a m i d e  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 1 0 . 1 . 1 6  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  M C P - 1 : C r e a t i n i n e  R a t i o :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  –  A l l  P a t i e n t s  R e c e i v i n g  R i t u x i m a b  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 1 0 . 1 . 1 7  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  M C P - 1 : C r e a t i n i n e  R a t i o :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  –  A l l  P a t i e n t s  w i t h  N e w l y  D i a g n o s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 0 . 1 . 1 8  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  M C P - 1 : C r e a t i n i n e  R a t i o :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  –  A l l  P a t i e n t s  w i t h  R e l a p s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 0 . 1 . 1 9  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  M C P - 1 : C r e a t i n i n e  R a t i o :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  –  A l l  P a t i e n t s  w i t h  M P O +  D i s e a s e  


 
P a ge 9 5  of 2 0 8 
 
T a b l e  1 4 . 2 . 1 0 . 1 . 2 0  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  M C P - 1 : C r e a t i n i n e  R a t i o :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  –  A l l  P a t i e n t s  w i t h  P R 3 +  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 0 . 1 . 2 1  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  M C P - 1 : C r e a t i n i n e  R a t i o :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  –  A l l  P a t i e n t s  w i t h  G r a n u l o m a t o s i s  P o l y a n g i i t i s  ( W e g e n e r ’ s )  
 
T a b l e  1 4 . 2 . 1 0 . 1 . 2 2  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  M C P - 1 : C r e a t i n i n e  R a t i o :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  –  A l l  P a t i e n t s  w i t h  M i c r o s c o p i c  P o l y a n g i i t i s  


 
P a ge 9 6  of 2 0 8 
 
T a b l e  1 4 . 2 . 1 0 . 1 . 2 3  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  U r i n a r y  M C P - 1 : C r e a t i n i n e  R a t i o :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  
 
T a b l e  1 4 . 2 . 1 0 . 1 . 2 4  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  U r i n a r y  M C P - 1 : C r e a t i n i n e  R a t i o :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  –  A l l  P a t i e n t s  w i t h  R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 1 0 . 1 . 2 5  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  U r i n a r y  M C P - 1 : C r e a t i n i n e  R a t i o :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  –  A l l  P a t i e n t s  w i t h  N o n - R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 1 0 . 1 . 2 6  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  U r i n a r y  M C P - 1 : C r e a t i n i n e  R a t i o :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  –  A l l  P a t i e n t s  R e c e i v i n g  C y c l o p h o s p h a m i d e  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 1 0 . 1 . 2 7  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  U r i n a r y  M C P - 1 : C r e a t i n i n e  R a t i o :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  –  A l l  P a t i e n t s  R e c e i v i n g  R i t u x i m a b  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 1 0 . 1 . 2 8  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  U r i n a r y  M C P - 1 : C r e a t i n i n e  R a t i o :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  –  A l l  P a t i e n t s  w i t h  N e w l y  D i a g n o s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 0 . 1 . 2 9  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  U r i n a r y  M C P - 1 : C r e a t i n i n e  R a t i o :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  –  A l l  P a t i e n t s  w i t h  R e l a p s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 0 . 1 . 3 0  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  U r i n a r y  M C P - 1 : C r e a t i n i n e  R a t i o :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  –  A l l  P a t i e n t s  w i t h  M P O +  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 0 . 1 . 3 1  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  U r i n a r y  M C P - 1 : C r e a t i n i n e  R a t i o :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  –  A l l  P a t i e n t s  w i t h  P R 3 +  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 0 . 1 . 3 2  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  U r i n a r y  M C P - 1 : C r e a t i n i n e  R a t i o :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  –  A l l  P a t i e n t s  w i t h  G r a n u l o m a t o s i s  P o l y a n g i i t i s  ( W e g e n e r ’ s )  
 


 
P a ge 9 7  of 2 0 8 
 
T a b l e  1 4 . 2 . 1 0 . 1 . 3 3  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  U r i n a r y  M C P - 1 : C r e a t i n i n e  R a t i o :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  –  A l l  P a t i e n t s  w i t h  M i c r o s c o p i c  P o l y a n g i i t i s  


 
P a ge 9 8  of 2 0 8 
 
T a b l e  1 4 . 2 . 1 0 . 2 . 1  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  M C P - 1 : C r e a t i n i n e  R a t i o :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  
 
T a b l e  1 4 . 2 . 1 0 . 2 . 2  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  M C P - 1 : C r e a t i n i n e  R a t i o :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  –  A l l  P a t i e n t s  w i t h  R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 1 0 . 2 . 3  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  M C P - 1 : C r e a t i n i n e  R a t i o :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  –  A l l  P a t i e n t s  w i t h  N o n - R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 1 0 . 2 . 4  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  M C P - 1 : C r e a t i n i n e  R a t i o :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  –  A l l  P a t i e n t s  R e c e i v i n g  C y c l o p h o s p h a m i d e  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 1 0 . 2 . 5  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  M C P - 1 : C r e a t i n i n e  R a t i o :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  –  A l l  P a t i e n t s  R e c e i v i n g  R i t u x i m a b  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 1 0 . 2 . 6  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  M C P - 1 : C r e a t i n i n e  R a t i o :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  –  A l l  P a t i e n t s  w i t h  N e w l y  D i a g n o s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 0 . 2 . 7  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  M C P - 1 : C r e a t i n i n e  R a t i o :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  –  A l l  P a t i e n t s  w i t h  R e l a p s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 0 . 2 . 8  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  M C P - 1 : C r e a t i n i n e  R a t i o :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  –  A l l  P a t i e n t s  w i t h  M P O +  D i s e a s e  


 
P a ge 9 9  of 2 0 8 
 
T a b l e  1 4 . 2 . 1 0 . 2 . 9  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  M C P - 1 : C r e a t i n i n e  R a t i o :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  –  A l l  P a t i e n t s  w i t h  P R 3 +  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 0 . 2 . 1 0  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  M C P - 1 : C r e a t i n i n e  R a t i o :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  –  A l l  P a t i e n t s  w i t h  G r a n u l o m a t o s i s  P o l y a n g i i t i s  ( W e g e n e r ’ s )  
 
T a b l e  1 4 . 2 . 1 0 . 2 . 1 1  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  M C P - 1 : C r e a t i n i n e  R a t i o :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  –  A l l  P a t i e n t s  w i t h  M i c r o s c o p i c  P o l y a n g i i t i s  


 
P a ge 1 0 0  of 2 0 8  
 
T a b l e  1 4 . 2 . 1 0 . 2 . 1 2  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  M C P - 1 : C r e a t i n i n e  R a t i o :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  
 
T a b l e  1 4 . 2 . 1 0 . 2 . 1 3  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  M C P - 1 : C r e a t i n i n e  R a t i o :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  –  A l l  P a t i e n t s  w i t h  R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 1 0 . 2 . 1 4  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  M C P - 1 : C r e a t i n i n e  R a t i o :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  –  A l l  P a t i e n t s  w i t h  N o n - R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 1 0 . 2 . 1 5  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  M C P - 1 : C r e a t i n i n e  R a t i o :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  –  A l l  P a t i e n t s  R e c e i v i n g  C y c l o p h o s p h a m i d e  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 1 0 . 2 . 1 6  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  M C P - 1 : C r e a t i n i n e  R a t i o :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  –  A l l  P a t i e n t s  R e c e i v i n g  R i t u x i m a b  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 1 0 . 2 . 1 7  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  M C P - 1 : C r e a t i n i n e  R a t i o :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  –  A l l  P a t i e n t s  w i t h  N e w l y  D i a g n o s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 0 . 2 . 1 8  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  M C P - 1 : C r e a t i n i n e  R a t i o :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  –  A l l  P a t i e n t s  w i t h  R e l a p s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 0 . 2 . 1 9  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  M C P - 1 : C r e a t i n i n e  R a t i o :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  –  A l l  P a t i e n t s  w i t h  M P O +  D i s e a s e  


 
P a ge 1 0 1  of 2 0 8  
 
T a b l e  1 4 . 2 . 1 0 . 2 . 2 0  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  M C P - 1 : C r e a t i n i n e  R a t i o :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  –  A l l  P a t i e n t s  w i t h  P R 3 +  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 0 . 2 . 2 1  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  M C P - 1 : C r e a t i n i n e  R a t i o :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  –  A l l  P a t i e n t s  w i t h  G r a n u l o m a t o s i s  P o l y a n g i i t i s  ( W e g e n e r ’ s )  
 
T a b l e  1 4 . 2 . 1 0 . 2 . 2 2  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  M C P - 1 : C r e a t i n i n e  R a t i o :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  –  A l l  P a t i e n t s  w i t h  M i c r o s c o p i c  P o l y a n g i i t i s  


 
P a ge 1 0 2  of 2 0 8  
 
T a b l e  1 4 . 2 . 1 0 . 3 . 1  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  M C P - 1 : C r e a t i n i n e  R a t i o :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  
 
T a b l e  1 4 . 2 . 1 0 . 3 . 2  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  M C P - 1 : C r e a t i n i n e  R a t i o :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  –  A l l  P a t i e n t s  w i t h  R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 1 0 . 3 . 3  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  M C P - 1 : C r e a t i n i n e  R a t i o :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  –  A l l  P a t i e n t s  w i t h  N o n - R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 1 0 . 3 . 4  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  M C P - 1 : C r e a t i n i n e  R a t i o :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  –  A l l  P a t i e n t s  R e c e i v i n g  C y c l o p h o s p h a m i d e  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 1 0 . 3 . 5  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  M C P - 1 : C r e a t i n i n e  R a t i o :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  –  A l l  P a t i e n t s  R e c e i v i n g  R i t u x i m a b  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 1 0 . 3 . 6  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  M C P - 1 : C r e a t i n i n e  R a t i o :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  –  A l l  P a t i e n t s  w i t h  N e w l y  D i a g n o s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 0 . 3 . 7  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  M C P - 1 : C r e a t i n i n e  R a t i o :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  –  A l l  P a t i e n t s  w i t h  R e l a p s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 0 . 3 . 8  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  M C P - 1 : C r e a t i n i n e  R a t i o :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  –  A l l  P a t i e n t s  w i t h  M P O +  D i s e a s e  


 
P a ge 1 0 3  of 2 0 8  
 
T a b l e  1 4 . 2 . 1 0 . 3 . 9  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  M C P - 1 : C r e a t i n i n e  R a t i o :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  –  A l l  P a t i e n t s  w i t h  P R 3 +  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 0 . 3 . 1 0  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  M C P - 1 : C r e a t i n i n e  R a t i o :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  –  A l l  P a t i e n t s  w i t h  G r a n u l o m a t o s i s  P o l y a n g i i t i s  ( W e g e n e r ’ s )  
 
T a b l e  1 4 . 2 . 1 0 . 3 . 1 1  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  M C P - 1 : C r e a t i n i n e  R a t i o :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  –  A l l  P a t i e n t s  w i t h  M i c r o s c o p i c  P o l y a n g i i t i s  


 
P a ge 1 0 4  of 2 0 8  
 
T a b l e  1 4 . 2 . 1 0 . 3 . 1 2  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  M C P - 1 : C r e a t i n i n e  R a t i o :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  
 
T a b l e  1 4 . 2 . 1 0 . 3 . 1 3  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  M C P - 1 : C r e a t i n i n e  R a t i o :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  –  A l l  P a t i e n t s  w i t h  R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 1 0 . 3 . 1 4  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  M C P - 1 : C r e a t i n i n e  R a t i o :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  –  A l l  P a t i e n t s  w i t h  N o n - R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 1 0 . 3 . 1 5  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  M C P - 1 : C r e a t i n i n e  R a t i o :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  –  A l l  P a t i e n t s  R e c e i v i n g  C y c l o p h o s p h a m i d e  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 1 0 . 3 . 1 6  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  M C P - 1 : C r e a t i n i n e  R a t i o :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  –  A l l  P a t i e n t s  R e c e i v i n g  R i t u x i m a b  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 1 0 . 3 . 1 7  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  M C P - 1 : C r e a t i n i n e  R a t i o :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  –  A l l  P a t i e n t s  w i t h  N e w l y  D i a g n o s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 0 . 3 . 1 8  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  M C P - 1 : C r e a t i n i n e  R a t i o :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  –  A l l  P a t i e n t s  w i t h  R e l a p s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 0 . 3 . 1 9  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  M C P - 1 : C r e a t i n i n e  R a t i o :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  –  A l l  P a t i e n t s  w i t h  M P O +  D i s e a s e  


 
P a ge 1 0 5  of 2 0 8  
 
T a b l e  1 4 . 2 . 1 0 . 3 . 2 0  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  M C P - 1 : C r e a t i n i n e  R a t i o :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  –  A l l  P a t i e n t s  w i t h  P R 3 +  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 0 . 3 . 2 1  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  M C P - 1 : C r e a t i n i n e  R a t i o :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  –  A l l  P a t i e n t s  w i t h  G r a n u l o m a t o s i s  P o l y a n g i i t i s  ( W e g e n e r ’ s )  
 
T a b l e  1 4 . 2 . 1 0 . 3 . 2 2  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  U r i n a r y  M C P - 1 : C r e a t i n i n e  R a t i o :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  –  A l l  P a t i e n t s  w i t h  M i c r o s c o p i c  P o l y a n g i i t i s   


 
P a ge 1 0 6  of 2 0 8  
 
C h e m o C e n t r y x ,  I n c .  
P r o t o c o l  C L 0 0 2 _ 1 6 8                                                                                                          P a g e  1  o f  #  
T a b l e  1 4 . 2 . 1 1  
L i s t i n g  o f  R e s c u e  I V  o r  O r a l  G l u c o c o r t i c o i d  T r e a t m e n t  
I n t e n t - t o - T r e a t  P o p u l a t i o n  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
S t e p             D a t e  o f      R T :  R e p o r t e d  T e r m  
  T r e a t m e n t    F i r s t  D o s e    A T C :  A n a t o m i c  T h e r a p e u t i c  C l a s s            S t a r t  D a t e  ( D a y ) /    D u r a t i o n         
    S u b j e c t   o f  S t u d y  M e d .   P T :  P r e f e r r e d  T e r m                        E n d  D a t e  ( D a y )        ( D a y s )     D o s e / U n i t            R o u t e  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
 S t e p  1  
  X X X X X X X X X X X X X X X X X X X X X X X X X X X X X   
    # # # - # # #     D D M M M Y Y Y Y     R T : X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X     D D M M M Y Y Y Y  ( # # # ) /          # #    X X X X X X X X X X X X X X X X X X    X X X X X X X X X X X X  
                           A T C : X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X     D D M M M Y Y Y Y  ( # # # )  
                            P T : X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
P r o g r a m  N a m e :  X X X X X X X X . s a s                   R u n  D a t e :  D D M M M Y Y Y Y  H H : M M                            D a t a b a s e  l a s t  m o d i f i e d :  D D M M Y Y Y Y  H H : M M  


 
P a ge 1 0 7  of 2 0 8  
 
T he f oll o wi n g t a bles w ill ha ve si milar la y o uts t o Ta bles 1 4. 2. 5. x: 
T a b l e  1 4 . 2 . 1 2 . 1 . 1  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  V D I  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d   
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  
 
T a b l e  1 4 . 2 . 1 2 . 1 . 2  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  V D I  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d   
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 1 2 . 1 . 3  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  V D I  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d   
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N o n - R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 1 2 . 1 . 4  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  V D I  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d   
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  C y c l o p h o s p h a m i d e  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 1 2 . 1 . 5  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  V D I  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d   
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  R i t u x i m a b  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 1 2 . 1 . 6  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  V D I  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d   
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N e w l y  D i a g n o s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 2 . 1 . 7  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  V D I  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d   
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e l a p s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 2 . 1 . 8  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  V D I  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d   
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M P O +  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 2 . 1 . 9  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  V D I  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d   
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  P R 3 +  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 2 . 1 . 1 0  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  V D I  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d   
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  G r a n u l o m a t o s i s  w i t h  P o l y a n g i i t i s  ( W e g e n e r ’ s )  


 
P a ge 1 0 8  of 2 0 8  
 
T a b l e  1 4 . 2 . 1 2 . 1 . 1 1  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  V D I  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d   
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M i c r o s c o p i c  P o l y a n g i i t i s  


 
P a ge 1 0 9  of 2 0 8  
 
T a b l e  1 4 . 2 . 1 2 . 2 . 1 . 1 2  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  V D I  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  
 
T a b l e  1 4 . 2 . 1 2 . 2 . 1 . 1 3  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  V D I  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 1 2 . 2 . 1 . 1 4  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  V D I  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N o n - R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 1 2 . 2 . 1 . 1 5  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  V D I  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  C y c l o p h o s p h a m i d e  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 1 2 . 2 . 1 . 1 6  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  V D I  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  R i t u x i m a b  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 1 2 . 2 . 1 . 1 7  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  V D I  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N e w l y  D i a g n o s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 2 . 2 . 1 . 1 8  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  V D I  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e l a p s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 2 . 2 . 1 . 1 9  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  V D I  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M P O +  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 2 . 2 . 1 . 2 0  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  V D I  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  P R 3 +  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 2 . 2 . 1 . 2 1  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  V D I  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  G r a n u l o m a t o s i s  w i t h  P o l y a n g i i t i s  ( W e g e n e r ’ s )  


 
P a ge 1 1 0  of 2 0 8  
 
T a b l e  1 4 . 2 . 1 2 . 2 . 1 . 2 2  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  V D I  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M i c r o s c o p i c  P o l y a n g i i t i s  


 
P a ge 1 1 1  of 2 0 8  
 
T a b l e  1 4 . 2 . 1 2 . 1 . 2 3  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  V D I  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  
 
T a b l e  1 4 . 2 . 1 2 . 1 . 2 4  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  V D I  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 1 2 . 1 . 2 5  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  V D I  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N o n - R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 1 2 . 1 . 2 6  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  V D I  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  C y c l o p h o s p h a m i d e  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 1 2 . 1 . 2 7  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  V D I  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  R i t u x i m a b  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 1 2 . 1 . 2 8  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  V D I  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N e w l y  D i a g n o s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 2 . 1 . 2 9  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  V D I  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e l a p s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 2 . 1 . 3 0  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  V D I  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M P O +  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 2 . 1 . 3 1  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  V D I  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  P R 3 +  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 2 . 1 . 3 2  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  V D I  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  G r a n u l o m a t o s i s  w i t h  P o l y a n g i i t i s  ( W e g e n e r ’ s )  
 


 
P a ge 1 1 2  of 2 0 8  
 
T a b l e  1 4 . 2 . 1 2 . 1 . 3 3  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  V D I  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M i c r o s c o p i c  P o l y a n g i i t i s  


 
P a ge 1 1 3  of 2 0 8  
 
T a b l e  1 4 . 2 . 1 2 . 2 . 2 . 1  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  V D I  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  
 
T a b l e  1 4 . 2 . 1 2 . 2 . 2 . 3  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  V D I  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 1 2 . 2 . 2 . 4  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  V D I  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N o n - R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 1 2 . 2 . 2 . 4  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  V D I  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  C y c l o p h o s p h a m i d e  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 1 2 . 2 . 2 . 5  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  V D I  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  R i t u x i m a b  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 1 2 . 2 . 2 . 6  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  V D I  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N e w l y  D i a g n o s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 2 . 2 . 2 . 7  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  V D I  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e l a p s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 2 . 2 . 2 . 8  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  V D I  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M P O +  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 2 . 2 . 2 . 9  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  V D I  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  P R 3 +  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 2 . 2 . 2 . 1 0  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  V D I  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  G r a n u l o m a t o s i s  w i t h  P o l y a n g i i t i s  ( W e g e n e r ’ s )  


 
P a ge 1 1 4  of 2 0 8  
 
T a b l e  1 4 . 2 . 1 2 . 2 . 2 . 1 1  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  V D I  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M i c r o s c o p i c  P o l y a n g i i t i s  


 
P a ge 1 1 5  of 2 0 8  
 
T a b l e  1 4 . 2 . 1 2 . 3 . 1  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  V D I  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  
 
T a b l e  1 4 . 2 . 1 2 . 3 . 2  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  V D I  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 1 2 . 3 . 3  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  V D I  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N o n - R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 1 2 . 3 . 4  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  V D I  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  C y c l o p h o s p h a m i d e  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 1 2 . 3 . 5  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  V D I  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  R i t u x i m a b  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 1 2 . 3 . 6  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  V D I  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N e w l y  D i a g n o s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 2 . 3 . 7  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  V D I  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e l a p s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 2 . 3 . 8  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  V D I  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M P O +  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 2 . 3 . 9  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  V D I  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  P R 3 +  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 2 . 3 . 1 0  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  V D I  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  G r a n u l o m a t o s i s  w i t h  P o l y a n g i i t i s  ( W e g e n e r ’ s )  


 
P a ge 1 1 6  of 2 0 8  
 
T a b l e  1 4 . 2 . 1 2 . 3 . 1 1  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  V D I  C h a n g e  f r o m  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M i c r o s c o p i c  P o l y a n g i i t i s  


 
P a ge 1 1 7  of 2 0 8  
 
C h e m o C e n t r y x ,  I n c .  
P r o t o c o l  C L 0 0 2 _ 1 6 8                                                                                                          P a g e  1  o f  #  
T a b l e  1 4 . 2 . 1 3 . 1  
S u m m a r y  o f  S F - 3 6  v 2 . 0 :  A c t u a l ,  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  b y  V i s i t  
S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
                           P l a c e b o  +                       C C X 1 6 8  +                     C C X 1 6 8  +  
                      F u l l  D o s e  P r e d n i s o n e            L o w  D o s e  P r e d n i s o n e             N o  P r e d n i s o n e                    A l l  C C X 1 6 8  
  D o m a i n                      ( N = # # )                         ( N = # # )                       ( N = # # )                          ( N = # # )  
   S t u d y  D a y         - - - - - - - - - - - - - - - - - - - - - - - -       - - - - - - - - - - - - - - - - - - - - - - - -     - - - - - - - - - - - - - - - - - - - - - - - -       - - - - - - - - - - - - - - - - - - - - - - - - -  
    S t a t i s t i c        V i s i t     C h a n g e   %  C h a n g e      V i s i t     C h a n g e   %  C h a n g e    V i s i t     C h a n g e   %  C h a n g e       V i s i t     C h a n g e   %  C h a n g e  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
  P h y s i c a l  F u n c t i o n i n g  
   B a s e l i n e  
    N ’               # #  
    M e a n             # # . # #  
    S D               # # . # # #  
    M i n i m u m          # # . #  
    M e d i a n           # # . # #  
    M a x i m u m          # # . #  
 
   D a y  2 9  
    N ’               # #        # #        # #           # #        # #        # #          # #        # #        # #           # #        # #        # #  
    M e a n             # # . # #     # # . # #     # # . # #        # # . # #     # # . # #     # # . # #       # # . # #     # # . # #     # # . # #        # # . # #     # # . # #     # # . # #  
    S D               # # . # # #    # # . # # #    # # . # # #       # # . # # #    # # . # # #    # # . # # #      # # . # # #    # # . # # #    # # . # # #       # # . # # #    # # . # # #    # # . # # #  
    M i n i m u m          # # . #      # # . #      # # . #         # # . #      # # . #      # # . #        # # . #      # # . #      # # . #         # # . #      # # . #      # # . #  
    M e d i a n           # # . # #     # # . # #     # # . # #        # # . # #     # # . # #     # # . # #       # # . # #     # # . # #     # # . # #        # # . # #     # # . # #     # # . # #  
    M a x i m u m          # # . #      # # . #      # # . #         # # . #      # # . #      # # . #        # # . #      # # . #      # # . #         # # . #      # # . #      # # . #   
    P - v a l u e *                                               # . # # # #    # . # # # #               # . # # # #    # . # # # #                # . # # # #    # . # # # #  
    9 5 %  C I  f o r  C h a n g e *                                      ( - # # . # ,  # # . # )                 ( - # # . # ,  # # . # )                  ( - # # . # ,  # # . # )  
    9 5 %  C I  f o r  % C h a n g e *                                     ( - # # . # ,  # # . # )                 ( - # # . # ,  # # . # )                  ( - # # . # ,  # # . # )  
 
  T h e  d o m a i n s  t o  b e  s u m m a r i z e d  i n c l u d e :  
     R o l e - P h y s i c a l ,  R o l e - E m o t i o n a l ,  S o c i a l  F u n c t i o n i n g ,  B o d i l y  P a i n ,  M e n t a l  H e a l t h ,  V i t a l i t y ,  G e n e r a l  H e a l t h  P e r c e p t i o n s ,  
     C h a n g e  i n  H e a l t h ,  P h y s i c a l  C o m p o n e n t  S u m m a r y ,  M e n t a l  H e a l t h  S u m m a r y .  
 
  T h e  a d d i t i o n a l  v i s i t s  t o  b e  s u m m a r i z e d  i n c l u d e :  
     D a y  2 9 ,  D a y  8 5 ,  E n d  o f  T r e a t m e n t ,  D a y  1 6 9 ,  a n d  E n d  o f  F o l l o w - u p  P e r i o d .  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
-  B a s e l i n e  i s  d e f i n e d  a s  t h e  l a s t  p r e - d o s e  v a l u e .  
-  N ' = n u m b e r  o f  s u b j e c t s  w i t h  d a t a  a t  b a s e l i n e  a n d  t h e  s p e c i f i e d  v i s i t .  
-  T h e  E n d  o f  T r e a t m e n t  v a l u e  i s  t h e  l a s t  m e a s u r e m e n t  t h r o u g h  D a y  8 5 .  
-  T h e  E n d  o f  F o l l o w - u p  P e r i o d  i s  t h e  l a s t  m e a s u r e m e n t  a f t e r  D a y  8 5  t h r o u g h  D a y  1 6 9 .  
* P - v a l u e s  a n d  9 5 %  c o n f i d e n c e  i n t e r v a l s  f o r  d i f f e r e n c e s  b e t w e e n  s p e c i f i e d  t r e a t m e n t  g r o u p s  a n d  p l a c e b o  a r e  f r o m  a  M M R M  m o d e l  w i t h  
 t r e a t m e n t  g r o u p  a s  a  f a c t o r  a n d  r a n d o m i z a t i o n  s t r a t a  ( A A V  s t a t u s ,  A N C A  p o s i t i v i t y ,  a n d  s t a n d a r d  o f  c a r e  t r e a t m e n t )  a s  c o v a r i a t e s .  
 
P r o g r a m  N a m e :  X X X X X X X X . s a s                   R u n  D a t e :  D D M M M Y Y Y Y  H H : M M                            D a t a b a s e  l a s t  m o d i f i e d :  D D M M Y Y Y Y  H H : M M  


 
P a ge 1 1 8  of 2 0 8  
 
N O T E: T he S F - 3 6 was o nl y c ollecte d f or patie nts partici pati n g i n Ste p 3.  


 
P a ge 1 1 9  of 2 0 8  
 
T he f oll o wi n g t a ble w ill ha ve si milar la y o uts t o Ta bles 1 4. 2. 1 3. 1: 
T a b l e  1 4 . 2 . 1 3 . 2  
S u m m a r y  o f  S F - 3 6  v 2 . 0 :  A c t u a l ,  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  b y  V i s i t  
S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 1 3 . 3  
S u m m a r y  o f  S F - 3 6  v 2 . 0 :  A c t u a l ,  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  b y  V i s i t  
S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N o n - R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 1 3 . 4  
S u m m a r y  o f  S F - 3 6  v 2 . 0 :  A c t u a l ,  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  b y  V i s i t  
S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  C y c l o p h o s p h a m i d e  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 1 3 . 5  
S u m m a r y  o f  S F - 3 6  v 2 . 0 :  A c t u a l ,  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  b y  V i s i t  
S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  R i t u x i m a b  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 1 3 . 6  
S u m m a r y  o f  S F - 3 6  v 2 . 0 :  A c t u a l ,  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  b y  V i s i t  
S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N e w l y  D i a g n o s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 3 . 7  
S u m m a r y  o f  S F - 3 6  v 2 . 0 :  A c t u a l ,  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  b y  V i s i t  
S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e l a p s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 3 . 8  
S u m m a r y  o f  S F - 3 6  v 2 . 0 :  A c t u a l ,  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  b y  V i s i t  
S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M P O +  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 3 . 9  
S u m m a r y  o f  S F - 3 6  v 2 . 0 :  A c t u a l ,  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  b y  V i s i t  
S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  P R 3 +  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 3 . 1 0  
S u m m a r y  o f  S F - 3 6  v 2 . 0 :  A c t u a l ,  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  b y  V i s i t  
S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  G r a n u l o m a t o s i s  w i t h  P o l y a n g i i t i s  ( W e g e n e r ’ s )  
 
T a b l e  1 4 . 2 . 1 3 . 1 1  
S u m m a r y  o f  S F - 3 6  v 2 . 0 :  A c t u a l ,  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  b y  V i s i t  
S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M i c r o s c o p i c  P o l y a n g i i t i s  


 
P a ge 1 2 0  of 2 0 8  
 
T he f oll o wi n g t a ble w ill ha ve si milar la y o uts t o Ta bles 1 4. 2. 1 3. 1: 
T a b l e  1 4 . 2 . 1 4 . 1  
S u m m a r y  o f  E Q - 5 D - 5 L  H e a l t h  S c a l e  S c o r e  a n d  V A S :  A c t u a l ,  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  b y  V i s i t  
S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  
 
T a b l e  1 4 . 2 . 1 4 . 2  
S u m m a r y  o f  E Q - 5 D - 5 L  H e a l t h  S c a l e  S c o r e  a n d  V A S :  A c t u a l ,  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  b y  V i s i t  
S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 1 4 . 3  
S u m m a r y  o f  E Q - 5 D - 5 L  H e a l t h  S c a l e  S c o r e  a n d  V A S :  A c t u a l ,  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  b y  V i s i t  
S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N o n - R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 1 4 . 4  
S u m m a r y  o f  E Q - 5 D - 5 L  H e a l t h  S c a l e  S c o r e  a n d  V A S :  A c t u a l ,  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  b y  V i s i t  
S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  C y c l o p h o s p h a m i d e  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 1 4 . 5  
S u m m a r y  o f  E Q - 5 D - 5 L  H e a l t h  S c a l e  S c o r e  a n d  V A S :  A c t u a l ,  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  b y  V i s i t  
S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  R i t u x i m a b  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 1 4 . 6  
S u m m a r y  o f  E Q - 5 D - 5 L  H e a l t h  S c a l e  S c o r e  a n d  V A S :  A c t u a l ,  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  b y  V i s i t  
S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N e w l y  D i a g n o s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 4 . 7  
S u m m a r y  o f  E Q - 5 D - 5 L  H e a l t h  S c a l e  S c o r e  a n d  V A S :  A c t u a l ,  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  b y  V i s i t  
S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e l a p s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 4 . 8  
S u m m a r y  o f  E Q - 5 D - 5 L  H e a l t h  S c a l e  S c o r e  a n d  V A S :  A c t u a l ,  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  b y  V i s i t  
S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M P O +  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 4 . 9  
S u m m a r y  o f  E Q - 5 D - 5 L  H e a l t h  S c a l e  S c o r e  a n d  V A S :  A c t u a l ,  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  b y  V i s i t  
S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  P R 3 +  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 4 . 1 0  
S u m m a r y  o f  E Q - 5 D - 5 L  H e a l t h  S c a l e  S c o r e  a n d  V A S :  A c t u a l ,  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  b y  V i s i t  
S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  G r a n u l o m a t o s i s  w i t h  P o l y a n g i i t i s  ( W e g e n e r ’ s )  


 
P a ge 1 2 1  of 2 0 8  
 
T a b l e  1 4 . 2 . 1 4 . 1 1  
S u m m a r y  o f  E Q - 5 D - 5 L  H e a l t h  S c a l e  S c o r e  a n d  V A S :  A c t u a l ,  C h a n g e  a n d  P e r c e n t  C h a n g e  f r o m  B a s e l i n e  b y  V i s i t  
S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M i c r o s c o p i c  P o l y a n g i i t i s  


 
P a ge 1 2 2  of 2 0 8  
 
C h e m o C e n t r y x ,  I n c .  
P r o t o c o l  C L 0 0 2 _ 1 6 8                                                                                                          P a g e  1  o f  1  
T a b l e  1 4 . 2 . 1 5  
S u m m a r y  o f  D o s e  a n d  D u r a t i o n  o f  S t u d y  S u p p l i e d  P r e d n i s o n e  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
 S t e p  
   P e r i o d                             C C X 1 6 8  +                         C C X 1 6 8  +                   C C X 1 6 8  +      
     V a r i a b l e                    F u l l  D o s e  P r e d n i s o n e            L o w  D o s e  P r e d n i s o n e           N o  P r e d n i s o n e               A l l  C C X 1 6 8   
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
 A l l  S t e p s                               N = # #                           N = # #                       N = # #                       N = # #  
 
   8 4 - d a y  d o s i n g  p e r i o d ,  N ’  ( % )  
 
     T o t a l  s t u d y - s u p p l i e d  p r e d n i s o n e / p l a c e b o  d o s e  ( m g )  
       N ’  
       M e a n  
       S D  
       M i n i m u m  
       M e d i a n  
       M a x i m u m  
 
     T o t a l  d u r a t i o n  o f  s t u d y - s u p p l i e d  p r e d n i s o n e / p l a c e b o  u s e  ( d a y s )  
       N ’  
       M e a n  
       S D  
       M i n i m u m  
       M e d i a n  
       M a x i m u m  
 
R e p e a t  f o r  1 6 8 - d a y  s t u d y  p e r i o d  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
-  N ’  =  n u m b e r  o f  s u b j e c t s  w h o  t o o k  s t u d y  s u p p l i e d  p r e d n i s o n e  d u r i n g  t h e  d e s i g n a t e d  s t u d y  p e r i o d .  
 
P r o g r a m  N a m e :  X X X X X X X X . s a s                   R u n  D a t e :  D D M M M Y Y Y Y  H H : M M                                  D a t a b a s e  l a s t  m o d i f i e d :  D D M M Y Y Y Y  
 
N O T E: Re peat f or Ste p 3, Ste ps 1 a n d 2 C o m bi ne d, Ste p 2, a n d Ste p 1.  


 
P a ge 1 2 3  of 2 0 8  
 
C h e m o C e n t r y x ,  I n c .  
P r o t o c o l  C L 0 0 2 _ 1 6 8                                                                                                          P a g e  1  o f  1  
T a b l e  1 4 . 2 . 1 6  
S u m m a r y  o f   D o s e  a n d  D u r a t i o n  o f  T o t a l  S y s t e m i c  C o r t i c o s t e r o i d  T r e a t m e n t *  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
 S t e p  
   P e r i o d                             C C X 1 6 8  +                         C C X 1 6 8  +                   C C X 1 6 8  +      
     V a r i a b l e                    F u l l  D o s e  P r e d n i s o n e            L o w  D o s e  P r e d n i s o n e           N o  P r e d n i s o n e               A l l  C C X 1 6 8   
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
 A l l  S t e p s                              N = # #                       N = # #                       N = # #                       N = # #  
 
  8 4 - d a y  d o s i n g  p e r i o d ,  N ’  ( % )  
 
      T o t a l  s y s t e m i c  c o r t i c o s t e r o i d  d o s e  ( m g )  
        N ’  
        M e a n  
        S D  
        M i n i m u m  
        M e d i a n  
        M a x i m u m  
 
      T o t a l  d u r a t i o n  o f  s y s t e m i c  c o r t i c o s t e r o i d  u s e  ( d a y s )  
        N ’  
        M e a n  
        S D  
        M i n i m u m  
        M e d i a n  
        M a x i m u m  
 
R e p e a t  f o r  1 6 8 - d a y  s t u d y  p e r i o d  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
-  N ’  =  n u m b e r  o f  s u b j e c t s  w h o  t o o k  c o r t i c o s t e r o i d  d u r i n g  t h e  d e s i g n a t e d  s t u d y  p e r i o d .  
* T o t a l  s y s t e m i c  ( I V  a n d  o r a l )  c o r t i c o s t e r o i d  t r e a t m e n t  i n c l u d e s  ( 1 )  s t u d y - s u p p l i e d  p r e d n i s o n e  u s e ,  ( 2 )  n e w  c o r t i c o s t e r o i d  u s e ,  a n d  
 ( 3 )  m a i n t e n a n c e  c o r t i c o s t e r o i d  u s e  o v e r  t h e  c o u r s e  o f  t h e  t r i a l .  
 
P r o g r a m  N a m e :  X X X X X X X X . s a s                   R u n  D a t e :  D D M M M Y Y Y Y  H H : M M                                  D a t a b a s e  l a s t  m o d i f i e d :  D D M M Y Y Y Y  
 
N O T E: Re peat f or Ste p 3, Ste ps 1 a n d 2 C o m bi ne d, Ste p 2, a n d Ste p 1.  


 
P a ge 1 2 4  of 2 0 8  
 
C h e m o C e n t r y x ,  I n c .  
P r o t o c o l  C L 0 0 2 _ 1 6 8                                                                                                          P a g e  1  o f  1  
T a b l e  1 4 . 2 . 1 7 . 1  
S u m m a r y  o f  T o t a l  C u m u l a t i v e  C y c l o p h o s p h a m i d e  D o s e  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
 S t e p  
   P e r i o d                             C C X 1 6 8  +                         C C X 1 6 8  +                   C C X 1 6 8  +      
     V a r i a b l e                    F u l l  D o s e  P r e d n i s o n e            L o w  D o s e  P r e d n i s o n e           N o  P r e d n i s o n e               A l l  C C X 1 6 8   
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
A l l  S t e p s                               N = # #                             N = # #                       N = # #                       N = # #  
 
  8 4 - d a y  d o s i n g  p e r i o d ,  n  ( % )  
 
      T o t a l  c u m u l a t i v e  d o s e  ( m g )  
        N ’  
        M e a n  
        S D  
        M i n i m u m  
        M e d i a n  
        M a x i m u m  
 
      T o t a l  d u r a t i o n  o f  d o s i n g  ( d a y s )  
        N ’  
        M e a n  
        S D  
        M i n i m u m  
        M e d i a n  
        M a x i m u m  
 
 
R e p e a t  f o r  1 6 8 - d a y  s t u d y  p e r i o d  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
-  N ’  =  n u m b e r  o f  s u b j e c t s  w h o  t o o k  a t  l e a s t  o n e  d o s e  o f  c y c l o p h o s p h a m i d e  d u r i n g  t h e  d e s i g n a t e d  s t u d y  p e r i o d .  
-  C y c l o p h o s p h a m i d e  d o s e  w a s  c a l c u l a t e d  a s  r e c o r d e d  a m o u n t ( m g / k g ) * w e i g h t  a t  t h a t  v i s i t .  I f  w e i g h t  w a s  m i s s i n g ,  t h e  l a s t  w e i g h t  f o r  
  t h a t  s u b j e c t  w o u l d  b e  u s e d .  
 
P r o g r a m  N a m e :  X X X X X X X X . s a s                   R u n  D a t e :  D D M M M Y Y Y Y  H H : M M                                  D a t a b a s e  l a s t  m o d i f i e d :  D D M M Y Y Y Y  
 
N O T E: Re peat f or Ste p 3, Ste ps 1 a n d 2 C o m bi ne d, Ste p 2, a n d Ste p 1.  


 
P a ge 1 2 5  of 2 0 8  
 
C h e m o C e n t r y x ,  I n c .  
P r o t o c o l  C L 0 0 2 _ 1 6 8                                                                                                          P a g e  1  o f  1  
T a b l e  1 4 . 2 . 1 7 . 2  
S u m m a r y  o f  T o t a l  C u m u l a t i v e  R i t u x i m a b  D o s e  
S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
 S t e p  
   P e r i o d                             C C X 1 6 8  +                         C C X 1 6 8  +                   C C X 1 6 8  +      
     V a r i a b l e                    F u l l  D o s e  P r e d n i s o n e            L o w  D o s e  P r e d n i s o n e           N o  P r e d n i s o n e               A l l  C C X 1 6 8   
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
A l l  S t e p s                               N = # #                             N = # #                       N = # #                       N = # #  
 
  8 4 - d a y  d o s i n g  p e r i o d ,  n  ( % )  
 
      T o t a l  c u m u l a t i v e  d o s e  ( m g )  
        N ’  
        M e a n  
        S D  
        M i n i m u m  
        M e d i a n  
        M a x i m u m  
 
      T o t a l  d u r a t i o n  o f  d o s i n g  ( d a y s )  
        N ’  
        M e a n  
        S D  
        M i n i m u m  
        M e d i a n  
        M a x i m u m  
 
 
R e p e a t  f o r  1 6 8 - d a y  s t u d y  p e r i o d  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
-  N ’  =  n u m b e r  o f  s u b j e c t s  w h o  t o o k  a t  l e a s t  o n e  d o s e  o f  c y c l o p h o s p h a m i d e  d u r i n g  t h e  d e s i g n a t e d  s t u d y  p e r i o d .  
 
P r o g r a m  N a m e :  X X X X X X X X . s a s                   R u n  D a t e :  D D M M M Y Y Y Y  H H : M M                                  D a t a b a s e  l a s t  m o d i f i e d :  D D M M Y Y Y Y  
 
 
 


 
P a ge 1 2 6  of 2 0 8  
 
T he f oll o wi n g t a bles w ill ha ve si milar la y o uts t o Ta bles 1 4. 2. 8. x: 
( F or t hes e ta bles, t he last f o ot n ote will be ‘- Res ults of ' < 0. 2' were i m p ute d t o ha ve a val u e of 0. 2 f or a nal ysis.’) 
T a b l e  1 4 . 2 . 1 8 . 1 . 1  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  S e r u m  h s C R P :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n   
 
T a b l e  1 4 . 2 . 1 8 . 1 . 2  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  S e r u m  h s C R P :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 1 8 . 1 . 3  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  S e r u m  h s C R P :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N o n - R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 1 8 . 1 . 4  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  S e r u m  h s C R P :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  C y c l o p h o s p h a m i d e  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 1 8 . 1 . 5  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  S e r u m  h s C R P :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  R i t u x i m a b  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 1 8 . 1 . 6  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  S e r u m  h s C R P :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N e w l y  D i a g n o s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 8 . 1 . 7  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  S e r u m  h s C R P :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e l a p s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 8 . 1 . 8  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  S e r u m  h s C R P :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M P O +  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 8 . 1 . 9  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  S e r u m  h s C R P :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  P R 3 +  D i s e a s e  
 


 
P a ge 1 2 7  of 2 0 8  
 
T a b l e  1 4 . 2 . 1 8 . 1 . 1 0  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  S e r u m  h s C R P :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  G r a n u l o m a t o s i s  w i t h  P o l y a n g i i t i s  ( W e g e n e r ’ s )  
 
T a b l e  1 4 . 2 . 1 8 . 1 . 1 1  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  S e r u m  h s C R P :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M i c r o s c o p i c  P o l y a n g i i t i s  


 
P a ge 1 2 8  of 2 0 8  
 
T a b l e  1 4 . 2 . 1 8 . 1 . 1 2  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  S e r u m  h s C R P :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n   
 
T a b l e  1 4 . 2 . 1 8 . 1 . 1 3  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  S e r u m  h s C R P :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 1 8 . 1 . 1 4  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  S e r u m  h s C R P :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N o n - R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 1 8 . 1 . 1 5  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  S e r u m  h s C R P :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  C y c l o p h o s p h a m i d e  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 1 8 . 1 . 1 6  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  S e r u m  h s C R P :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  R i t u x i m a b  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 1 8 . 1 . 1 7  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  S e r u m  h s C R P :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N e w l y  D i a g n o s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 8 . 1 . 1 8  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  S e r u m  h s C R P :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e l a p s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 8 . 1 . 1 9  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  S e r u m  h s C R P :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M P O +  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 8 . 1 . 2 0  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  S e r u m  h s C R P :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  P R 3 +  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 8 . 1 . 2 1  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  S e r u m  h s C R P :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  G r a n u l o m a t o s i s  w i t h  P o l y a n g i i t i s  ( W e g e n e r ’ s )  


 
P a ge 1 2 9  of 2 0 8  
 
T a b l e  1 4 . 2 . 1 8 . 1 . 2 2  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  S e r u m  h s C R P :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M i c r o s c o p i c  P o l y a n g i i t i s  


 
P a ge 1 3 0  of 2 0 8  
 
T a b l e  1 4 . 2 . 1 8 . 1 . 2 3  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  S e r u m  h s C R P :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e   
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n   
 
T a b l e  1 4 . 2 . 1 8 . 1 . 2 4  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  S e r u m  h s C R P :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e   
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 1 8 . 1 . 2 5  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  S e r u m  h s C R P :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e   
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N o n - R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 1 8 . 1 . 2 6  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  S e r u m  h s C R P :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e   
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  C y c l o p h o s p h a m i d e  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 1 8 . 1 . 2 7  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  S e r u m  h s C R P :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e   
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  R i t u x i m a b  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 1 8 . 1 . 2 8  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  S e r u m  h s C R P :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e   
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N e w l y  D i a g n o s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 8 . 1 . 2 9  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  S e r u m  h s C R P :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e   
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e l a p s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 8 . 1 . 3 0  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  S e r u m  h s C R P :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e   
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M P O +  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 8 . 1 . 3 1  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  S e r u m  h s C R P :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e   
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  P R 3 +  D i s e a s e  


 
P a ge 1 3 1  of 2 0 8  
 
 
T a b l e  1 4 . 2 . 1 8 . 1 . 3 2  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  S e r u m  h s C R P :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e   
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  G r a n u l o m a t o s i s  w i t h  P o l y a n g i i t i s  ( W e g e n e r ’ s )  
 
T a b l e  1 4 . 2 . 1 8 . 1 . 3 3  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  S e r u m  h s C R P :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e   
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M i c r o s c o p i c  P o l y a n g i i t i s  


 
P a ge 1 3 2  of 2 0 8  
 
T a b l e  1 4 . 2 . 1 8 . 2 . 1  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  S e r u m  h s C R P :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n   
 
T a b l e  1 4 . 2 . 1 8 . 2 . 2  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  S e r u m  h s C R P :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 1 8 . 2 . 3  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  S e r u m  h s C R P :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N o n - R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 1 8 . 2 . 4  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  S e r u m  h s C R P :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  C y c l o p h o s p h a m i d e  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 1 8 . 2 . 5  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  S e r u m  h s C R P :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  R i t u x i m a b  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 1 8 . 2 . 6  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  S e r u m  h s C R P :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N e w l y  D i a g n o s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 8 . 2 . 7  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  S e r u m  h s C R P :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e l a p s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 8 . 2 . 8  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  S e r u m  h s C R P :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M P O +  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 8 . 2 . 9  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  S e r u m  h s C R P :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  P R 3 +  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 8 . 2 . 1 0  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  S e r u m  h s C R P :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  G r a n u l o m a t o s i s  w i t h  P o l y a n g i i t i s  ( W e g e n e r ’ s )  


 
P a ge 1 3 3  of 2 0 8  
 
T a b l e  1 4 . 2 . 1 8 . 2 . 1 1  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  S e r u m  h s C R P :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M i c r o s c o p i c  P o l y a n g i i t i s  


 
P a ge 1 3 4  of 2 0 8  
 
T a b l e  1 4 . 2 . 1 8 . 2 . 1 2  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  S e r u m  h s C R P :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n   
 
T a b l e  1 4 . 2 . 1 8 . 2 . 1 3  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  S e r u m  h s C R P :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 1 8 . 2 . 1 4  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  S e r u m  h s C R P :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N o n - R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 1 8 . 2 . 1 5  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  S e r u m  h s C R P :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  C y c l o p h o s p h a m i d e  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 1 8 . 2 . 1 6  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  S e r u m  h s C R P :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  R i t u x i m a b  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 1 8 . 2 . 1 7  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  S e r u m  h s C R P :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N e w l y  D i a g n o s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 8 . 2 . 1 8  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  S e r u m  h s C R P :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e l a p s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 8 . 2 . 1 9  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  S e r u m  h s C R P :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M P O +  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 8 . 2 . 2 0  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  S e r u m  h s C R P :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  P R 3 +  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 8 . 2 . 2 1  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  S e r u m  h s C R P :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  G r a n u l o m a t o s i s  w i t h  P o l y a n g i i t i s  ( W e g e n e r ’ s )  


 
P a ge 1 3 5  of 2 0 8  
 
T a b l e  1 4 . 2 . 1 8 . 2 . 2 2  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  S e r u m  h s C R P :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M i c r o s c o p i c  P o l y a n g i i t i s  


 
P a ge 1 3 6  of 2 0 8  
 
T a b l e  1 4 . 2 . 1 8 . 3 . 1  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  S e r u m  h s C R P :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n   
 
T a b l e  1 4 . 2 . 1 8 . 3 . 2  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  S e r u m  h s C R P :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 1 8 . 3 . 3  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  S e r u m  h s C R P :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N o n - R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 1 8 . 3 . 4  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  S e r u m  h s C R P :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  C y c l o p h o s p h a m i d e  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 1 8 . 3 . 5  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  S e r u m  h s C R P :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  R i t u x i m a b  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 1 8 . 3 . 6  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  S e r u m  h s C R P :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N e w l y  D i a g n o s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 8 . 3 . 7  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  S e r u m  h s C R P :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e l a p s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 8 . 3 . 8  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  S e r u m  h s C R P :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M P O +  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 8 . 3 . 9  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  S e r u m  h s C R P :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  P R 3 +  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 8 . 3 . 1 0  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  S e r u m  h s C R P :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  G r a n u l o m a t o s i s  w i t h  P o l y a n g i i t i s  ( W e g e n e r ’ s )  


 
P a ge 1 3 7  of 2 0 8  
 
T a b l e  1 4 . 2 . 1 8 . 3 . 1 1  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  S e r u m  h s C R P :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M i c r o s c o p i c  P o l y a n g i i t i s  


 
P a ge 1 3 8  of 2 0 8  
 
T a b l e  1 4 . 2 . 1 8 . 3 . 1 2  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  S e r u m  h s C R P :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n   
 
T a b l e  1 4 . 2 . 1 8 . 3 . 1 3  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  S e r u m  h s C R P :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 1 8 . 3 . 1 4  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  S e r u m  h s C R P :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N o n - R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 1 8 . 3 . 1 5  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  S e r u m  h s C R P :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  C y c l o p h o s p h a m i d e  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 1 8 . 3 . 1 6  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  S e r u m  h s C R P :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  R i t u x i m a b  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 1 8 . 3 . 1 7  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  S e r u m  h s C R P :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N e w l y  D i a g n o s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 8 . 3 . 1 8  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  S e r u m  h s C R P :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e l a p s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 8 . 3 . 1 9  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  S e r u m  h s C R P :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M P O +  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 8 . 3 . 2 0  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  S e r u m  h s C R P :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  P R 3 +  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 8 . 3 . 2 1  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  S e r u m  h s C R P :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  G r a n u l o m a t o s i s  w i t h  P o l y a n g i i t i s  ( W e g e n e r ’ s )  


 
P a ge 1 3 9  of 2 0 8  
 
T a b l e  1 4 . 2 . 1 8 . 3 . 2 2  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  S e r u m  h s C R P :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M i c r o s c o p i c  P o l y a n g i i t i s  


 
P a ge 1 4 0  of 2 0 8  
 
T he f oll o wi n g t a bles w ill ha ve si milar la y o uts t o Ta bles 1 4. 2. 8. x: 
 
T a b l e  1 4 . 2 . 1 9 . 1 . 1  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A N C A  ( a n t i - P R 3  a n d  a n t i - M P O ) :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n   
 
T a b l e  1 4 . 2 . 1 9 . 1 . 2  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A N C A  ( a n t i - P R 3  a n d  a n t i - M P O ) :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 1 9 . 1 . 3  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A N C A  ( a n t i - P R 3  a n d  a n t i - M P O ) :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N o n - R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 1 9 . 1 . 4  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A N C A  ( a n t i - P R 3  a n d  a n t i - M P O ) :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  C y c l o p h o s p h a m i d e  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 1 9 . 1 . 5  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A N C A  ( a n t i - P R 3  a n d  a n t i - M P O ) :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  R i t u x i m a b  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 1 9 . 1 . 6  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A N C A  ( a n t i - P R 3  a n d  a n t i - M P O ) :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N e w l y  D i a g n o s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 9 . 1 . 7  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A N C A  ( a n t i - P R 3  a n d  a n t i - M P O ) :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e l a p s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 9 . 1 . 8  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A N C A  ( a n t i - P R 3  a n d  a n t i - M P O ) :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M P O +  D i s e a s e  


 
P a ge 1 4 1  of 2 0 8  
 
T a b l e  1 4 . 2 . 1 9 . 1 . 9  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A N C A  ( a n t i - P R 3  a n d  a n t i - M P O ) :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  P R 3 +  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 9 . 1 . 1 0  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A N C A  ( a n t i - P R 3  a n d  a n t i - M P O ) :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  G r a n u l o m a t o s i s  w i t h  P o l y a n g i i t i s  ( W e g e n e r ’ s )  
 
T a b l e  1 4 . 2 . 1 9 . 1 . 1 1  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A N C A  ( a n t i - P R 3  a n d  a n t i - M P O ) :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M i c r o s c o p i c  P o l y a n g i i t i s  


 
P a ge 1 4 2  of 2 0 8  
 
T a b l e  1 4 . 2 . 1 9 . 1 . 1 2  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A N C A  ( a n t i - P R 3  a n d  a n t i - M P O ) :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e   
d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n   
 
T a b l e  1 4 . 2 . 1 9 . 1 . 1 3  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A N C A  ( a n t i - P R 3  a n d  a n t i - M P O ) :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e   
d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 1 9 . 1 . 1 4  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A N C A  ( a n t i - P R 3  a n d  a n t i - M P O ) :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e   
d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N o n - R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 1 9 . 1 . 1 5  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A N C A  ( a n t i - P R 3  a n d  a n t i - M P O ) :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e   
d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  C y c l o p h o s p h a m i d e  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 1 9 . 1 . 1 6  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A N C A  ( a n t i - P R 3  a n d  a n t i - M P O ) :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e   
d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  R i t u x i m a b  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 1 9 . 1 . 1 7  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A N C A  ( a n t i - P R 3  a n d  a n t i - M P O ) :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e   
d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N e w l y  D i a g n o s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 9 . 1 . 1 8  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A N C A  ( a n t i - P R 3  a n d  a n t i - M P O ) :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e   
d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e l a p s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 9 . 1 . 1 9  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A N C A  ( a n t i - P R 3  a n d  a n t i - M P O ) :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e   
d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M P O +  D i s e a s e  


 
P a ge 1 4 3  of 2 0 8  
 
T a b l e  1 4 . 2 . 1 9 . 1 . 2 0  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A N C A  ( a n t i - P R 3  a n d  a n t i - M P O ) :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e   
d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  P R 3 +  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 9 . 1 . 2 1  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A N C A  ( a n t i - P R 3  a n d  a n t i - M P O ) :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e   
d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  G r a n u l o m a t o s i s  w i t h  P o l y a n g i i t i s  ( W e g e n e r ’ s )  
 
T a b l e  1 4 . 2 . 1 9 . 1 . 2 2  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A N C A  ( a n t i - P R 3  a n d  a n t i - M P O ) :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e   
d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M i c r o s c o p i c  P o l y a n g i i t i s  


 
P a ge 1 4 4  of 2 0 8  
 
T a b l e  1 4 . 2 . 1 9 . 1 . 2 3  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  A N C A  ( a n t i - P R 3  a n d  a n t i - M P O ) :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  
 
T a b l e  1 4 . 2 . 1 9 . 1 . 2 4  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  A N C A  ( a n t i - P R 3  a n d  a n t i - M P O ) :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 1 9 . 1 . 2 5  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  A N C A  ( a n t i - P R 3  a n d  a n t i - M P O ) :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N o n - R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 1 9 . 1 . 2 6  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  A N C A  ( a n t i - P R 3  a n d  a n t i - M P O ) :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  C y c l o p h o s p h a m i d e  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 1 9 . 1 . 2 7  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  A N C A  ( a n t i - P R 3  a n d  a n t i - M P O ) :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e   
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  R i t u x i m a b  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 1 9 . 1 . 2 8  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  A N C A  ( a n t i - P R 3  a n d  a n t i - M P O ) :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N e w l y  D i a g n o s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 9 . 1 . 2 9  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  A N C A  ( a n t i - P R 3  a n d  a n t i - M P O ) :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e l a p s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 9 . 1 . 3 0  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  A N C A  ( a n t i - P R 3  a n d  a n t i - M P O ) :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M P O +  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 9 . 1 . 3 1  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  A N C A  ( a n t i - P R 3  a n d  a n t i - M P O ) :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  P R 3 +  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 9 . 1 . 3 2  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  A N C A  ( a n t i - P R 3  a n d  a n t i - M P O ) :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  G r a n u l o m a t o s i s  w i t h  P o l y a n g i i t i s  ( W e g e n e r ’ s )  


 
P a ge 1 4 5  of 2 0 8  
 
T a b l e  1 4 . 2 . 1 9 . 1 . 3 3  
S u m m a r y  a n d  A n a l y s i s  ( A N C O V A )  o f  A N C A  ( a n t i - P R 3  a n d  a n t i - M P O ) :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e  
S t e p s  1  t h r o u g h  3  C o m b i n e d  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M i c r o s c o p i c  P o l y a n g i i t i s  


 
P a ge 1 4 6  of 2 0 8  
 
T a b l e  1 4 . 2 . 1 9 . 2 . 1  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A N C A  ( a n t i - P R 3  a n d  a n t i - M P O ) :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e   
d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n   
 
T a b l e  1 4 . 2 . 1 9 . 2 . 2  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A N C A  ( a n t i - P R 3  a n d  a n t i - M P O ) :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e   
d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 1 9 . 2 . 3  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A N C A  ( a n t i - P R 3  a n d  a n t i - M P O ) :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e   
d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N o n - R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 1 9 . 2 . 4  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A N C A  ( a n t i - P R 3  a n d  a n t i - M P O ) :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e   
d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  C y c l o p h o s p h a m i d e  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 1 9 . 2 . 5  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A N C A  ( a n t i - P R 3  a n d  a n t i - M P O ) :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e   
d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  R i t u x i m a b  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 1 9 . 2 . 6  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A N C A  ( a n t i - P R 3  a n d  a n t i - M P O ) :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e   
d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N e w l y  D i a g n o s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 9 . 2 . 7  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A N C A  ( a n t i - P R 3  a n d  a n t i - M P O ) :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e   
d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e l a p s e d  D i s e a s e  


 
P a ge 1 4 7  of 2 0 8  
 
T a b l e  1 4 . 2 . 1 9 . 2 . 8  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A N C A  ( a n t i - P R 3  a n d  a n t i - M P O ) :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e   
d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M P O +  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 9 . 2 . 9  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A N C A  ( a n t i - P R 3  a n d  a n t i - M P O ) :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e   
d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  P R 3 +  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 9 . 2 . 1 0  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A N C A  ( a n t i - P R 3  a n d  a n t i - M P O ) :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e   
d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  G r a n u l o m a t o s i s  w i t h  P o l y a n g i i t i s  ( W e g e n e r ’ s )  
 
T a b l e  1 4 . 2 . 1 9 . 2 . 1 1  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A N C A  ( a n t i - P R 3  a n d  a n t i - M P O ) :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e   
d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M i c r o s c o p i c  P o l y a n g i i t i s  


 
P a ge 1 4 8  of 2 0 8  
 
T a b l e  1 4 . 2 . 1 9 . 2 . 1 2  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A N C A  ( a n t i - P R 3  a n d  a n t i - M P O ) :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e   
d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n   
 
T a b l e  1 4 . 2 . 1 9 . 2 . 1 3  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A N C A  ( a n t i - P R 3  a n d  a n t i - M P O ) :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e   
d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 1 9 . 2 . 1 4  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A N C A  ( a n t i - P R 3  a n d  a n t i - M P O ) :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e   
d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N o n - R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 1 9 . 2 . 1 5  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A N C A  ( a n t i - P R 3  a n d  a n t i - M P O ) :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e   
d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  C y c l o p h o s p h a m i d e  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 1 9 . 2 . 1 6  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A N C A  ( a n t i - P R 3  a n d  a n t i - M P O ) :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e   
d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  R i t u x i m a b  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 1 9 . 2 . 1 7  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A N C A  ( a n t i - P R 3  a n d  a n t i - M P O ) :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e   
d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N e w l y  D i a g n o s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 9 . 2 . 1 8  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A N C A  ( a n t i - P R 3  a n d  a n t i - M P O ) :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e   
d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e l a p s e d  D i s e a s e  


 
P a ge 1 4 9  of 2 0 8  
 
T a b l e  1 4 . 2 . 1 9 . 2 . 1 9  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A N C A  ( a n t i - P R 3  a n d  a n t i - M P O ) :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e   
d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M P O +  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 9 . 2 . 2 0  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A N C A  ( a n t i - P R 3  a n d  a n t i - M P O ) :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e   
d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  P R 3 +  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 9 . 2 . 2 1  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A N C A  ( a n t i - P R 3  a n d  a n t i - M P O ) :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e   
d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  G r a n u l o m a t o s i s  w i t h  P o l y a n g i i t i s  ( W e g e n e r ’ s )  
 
T a b l e  1 4 . 2 . 1 9 . 2 . 2 2  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A N C A  ( a n t i - P R 3  a n d  a n t i - M P O ) :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e   
d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S t e p s  1  a n d  2  C o m b i n e d  a n d  S t e p  3  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  G r a n u l o m a t o s i s  w i t h  P o l y a n g i i t i s  ( W e g e n e r ’ s )  


 
P a ge 1 5 0  of 2 0 8  
 
T a b l e  1 4 . 2 . 1 9 . 3 . 1  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A N C A  ( a n t i - P R 3  a n d  a n t i - M P O ) :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e   
d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n   
 
T a b l e  1 4 . 2 . 1 9 . 3 . 2  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A N C A  ( a n t i - P R 3  a n d  a n t i - M P O ) :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e   
d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 1 9 . 3 . 3  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A N C A  ( a n t i - P R 3  a n d  a n t i - M P O ) :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e   
d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N o n - R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 1 9 . 3 . 4  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A N C A  ( a n t i - P R 3  a n d  a n t i - M P O ) :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e   
d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  C y c l o p h o s p h a m i d e  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 1 9 . 3 . 5  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A N C A  ( a n t i - P R 3  a n d  a n t i - M P O ) :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e   
d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  R i t u x i m a b  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 1 9 . 3 . 6  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A N C A  ( a n t i - P R 3  a n d  a n t i - M P O ) :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e   
d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N e w l y  D i a g n o s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 9 . 3 . 7  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A N C A  ( a n t i - P R 3  a n d  a n t i - M P O ) :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e   
d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e l a p s e d  D i s e a s e  


 
P a ge 1 5 1  of 2 0 8  
 
T a b l e  1 4 . 2 . 1 9 . 3 . 8  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A N C A  ( a n t i - P R 3  a n d  a n t i - M P O ) :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e   
d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M P O +  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 9 . 3 . 9  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A N C A  ( a n t i - P R 3  a n d  a n t i - M P O ) :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e   
d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  P R 3 +  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 9 . 3 . 1 0  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A N C A  ( a n t i - P R 3  a n d  a n t i - M P O ) :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e   
d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  G r a n u l o m a t o s i s  w i t h  P o l y a n g i i t i s  ( W e g e n e r ’ s )  
 
T a b l e  1 4 . 2 . 1 9 . 3 . 1 1  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A N C A  ( a n t i - P R 3  a n d  a n t i - M P O ) :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e   
d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M i c r o s c o p i c  P o l y a n g i i t i s  


 
P a ge 1 5 2  of 2 0 8  
 
T a b l e  1 4 . 2 . 1 9 . 3 . 1 2  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A N C A  ( a n t i - P R 3  a n d  a n t i - M P O ) :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e   
d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n   
 
T a b l e  1 4 . 2 . 1 9 . 3 . 1 3  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A N C A  ( a n t i - P R 3  a n d  a n t i - M P O ) :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e   
d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 1 9 . 3 . 1 4  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A N C A  ( a n t i - P R 3  a n d  a n t i - M P O ) :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e   
d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N o n - R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 1 9 . 3 . 1 5  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A N C A  ( a n t i - P R 3  a n d  a n t i - M P O ) :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e   
d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  C y c l o p h o s p h a m i d e  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 1 9 . 3 . 1 6  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A N C A  ( a n t i - P R 3  a n d  a n t i - M P O ) :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e   
d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  R i t u x i m a b  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 1 9 . 3 . 1 7  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A N C A  ( a n t i - P R 3  a n d  a n t i - M P O ) :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e   
d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N e w l y  D i a g n o s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 9 . 3 . 1 8  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A N C A  ( a n t i - P R 3  a n d  a n t i - M P O ) :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e   
d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e l a p s e d  D i s e a s e  


 
P a ge 1 5 3  of 2 0 8  
 
T a b l e  1 4 . 2 . 1 9 . 3 . 1 9  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A N C A  ( a n t i - P R 3  a n d  a n t i - M P O ) :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e   
d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M P O +  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 9 . 3 . 2 0  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A N C A  ( a n t i - P R 3  a n d  a n t i - M P O ) :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e   
d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  P R 3 +  D i s e a s e  
 
T a b l e  1 4 . 2 . 1 9 . 3 . 2 1  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A N C A  ( a n t i - P R 3  a n d  a n t i - M P O ) :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e   
d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  G r a n u l o m a t o s i s  w i t h  P o l y a n g i i t i s  ( W e g e n e r ’ s )  
 
T a b l e  1 4 . 2 . 1 9 . 3 . 2 2  
S u m m a r y  a n d  A n a l y s i s  ( M M R M )  o f  A N C A  ( a n t i - P R 3  a n d  a n t i - M P O ) :  R a t i o  a n d  P e r c e n t  C h a n g e  C o m p a r e d  t o  B a s e l i n e   
d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
B y  S t e p  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M i c r o s c o p i c  P o l y a n g i i t i s  


 
P a ge 1 5 4  of 2 0 8  
 
C h e m o C e n t r y x ,  I n c .  
P r o t o c o l  C L 0 0 2 _ 1 6 8                                                                                                          P a g e  #  o f  #  
T a b l e  1 4 . 2 . 2 0 . 1  
A n a l y s i s  o f  S u b j e c t s  w h o  B e c a m e  A N C A  N e g a t i v e  
I n t e n t - t o - T r e a t  P o p u l a t i o n  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
 P a r a m e t e r                                                                       D i f f e r e n c e  
  D a y                                                                          i n  p e r c e n t a g e s  
   S t e p      T r e a t m e n t                                   N ’         n     ( % )      v e r s u s  P l a c e b o  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
P R 3  ( c A N C A )  a n t i b o d y  b y  E L I S A  
 
  D a y  8 5  
   
    A l l      P l a c e b o  +  F u l l  D o s e  P r e d n i s o n e  ( N = # # )      # # #        # # #  ( # # # . # )  
            C C X 1 6 8  +  L o w  D o s e  P r e d n i s o n e  ( N = # # )        # # #        # # #  ( # # # . # )       # # # . #  
            C C X 1 6 8  +  N o  P r e d n i s o n e  ( N = # # )              # # #        # # #  ( # # # . # )       # # # . #  
            A l l  C C X 1 6 8  ( N = # # )                          # # #        # # #  ( # # # . # )       # # # . #  
 
    3        P l a c e b o  +  F u l l  D o s e  P r e d n i s o n e  ( N = # # )      # # #        # # #  ( # # # . # )  
            C C X 1 6 8  +  L o w  D o s e  P r e d n i s o n e  ( N = # # )        # # #        # # #  ( # # # . # )       # # # . #  
            C C X 1 6 8  +  N o  P r e d n i s o n e  ( N = # # )              # # #        # # #  ( # # # . # )       # # # . #  
            A l l  C C X 1 6 8  ( N = # # )                          # # #        # # #  ( # # # . # )       # # # . #  
 
    1 + 2      P l a c e b o  +  F u l l  D o s e  P r e d n i s o n e  ( N = # # )      # # #        # # #  ( # # # . # )  
            C C X 1 6 8  +  L o w  D o s e  P r e d n i s o n e  ( N = # # )        # # #        # # #  ( # # # . # )       # # # . #  
            C C X 1 6 8  +  N o  P r e d n i s o n e  ( N = # # )              # # #        # # #  ( # # # . # )       # # # . #  
            A l l  C C X 1 6 8  ( N = # # )                          # # #        # # #  ( # # # . # )       # # # . #  
 
    2        P l a c e b o  +  F u l l  D o s e  P r e d n i s o n e  ( N = # # )      # # #        # # #  ( # # # . # )  
            C C X 1 6 8  +  N o  P r e d n i s o n e  ( N = # # )              # # #        # # #  ( # # # . # )       # # # . #  
 
    1        P l a c e b o  +  F u l l  D o s e  P r e d n i s o n e  ( N = # # )      # # #        # # #  ( # # # . # )  
            C C X 1 6 8  +  L o w  D o s e  P r e d n i s o n e  ( N = # # )        # # #        # # #  ( # # # . # )       # # # . #  
 
R e p e a t  f o r  D a y  1 6 9  
 
R e p e a t  f o r  p a r a m e t e r :  M P O  ( p A N C A )  a n t i b o d y  b y  E L I S A  ( I U / m L )  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
-  N ' = n u m b e r  o f  s u b j e c t s  w h o  w e r e  P R 3  p o s i t i v e  ( f o r  P R 3  a n a l y s i s )  a n d  M P O  p o s i t i v e  ( f o r  M P O  a n a l y s i s )  a t  b a s e l i n e  w i t h  p o s t - b a s e l i n e   
  A N C A  d a t a  d u r i n g  t h e  s p e c i f i e d  d o s i n g  p e r i o d .  
 
P r o g r a m  N a m e :  X X X X X X X X . s a s                   R u n  D a t e :  D D M M M Y Y Y Y  H H : M M                                  D a t a b a s e  l a s t  m o d i f i e d :  D D M M Y Y Y Y  


 
P a ge 1 5 5  of 2 0 8  
 
T he f oll o wi n g t a bles w ill ha ve t he a m e la y o ut as Ta ble 1 4. 2. 2 0. 1: 
T a b l e  1 4 . 2 . 2 0 . 2  
A n a l y s i s  o f  S u b j e c t s  w h o  B e c a m e  A N C A  N e g a t i v e  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 2 0 . 3  
A n a l y s i s  o f  S u b j e c t s  w h o  B e c a m e  A N C A  N e g a t i v e  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N o n - R e n a l  D i s e a s e  a t  B a s e l i n e  
 
T a b l e  1 4 . 2 . 2 0 . 4  
A n a l y s i s  o f  S u b j e c t s  w h o  B e c a m e  A N C A  N e g a t i v e  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  C y c l o p h o s p h a m i d e  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 2 0 . 5  
A n a l y s i s  o f  S u b j e c t s  w h o  B e c a m e  A N C A  N e g a t i v e  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  R e c e i v i n g  R i t u x i m a b  B a c k g r o u n d  T r e a t m e n t  
 
T a b l e  1 4 . 2 . 2 0 . 6  
A n a l y s i s  o f  S u b j e c t s  w h o  B e c a m e  A N C A  N e g a t i v e  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  N e w l y  D i a g n o s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 2 0 . 7  
A n a l y s i s  o f  S u b j e c t s  w h o  B e c a m e  A N C A  N e g a t i v e  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  R e l a p s e d  D i s e a s e  
 
T a b l e  1 4 . 2 . 2 0 . 8  
A n a l y s i s  o f  S u b j e c t s  w h o  B e c a m e  A N C A  N e g a t i v e  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  G r a n u l o m a t o s i s  w i t h  P o l y a n g i i t i s  ( W e g e n e r ’ s )  
 
T a b l e  1 4 . 2 . 2 0 . 9  
A n a l y s i s  o f  S u b j e c t s  w h o  B e c a m e  A N C A  N e g a t i v e  
I n t e n t - t o - T r e a t  P o p u l a t i o n  -  A l l  P a t i e n t s  w i t h  M i c r o s c o p i c  P o l y a n g i i t i s  


 
P a ge 1 5 6  of 2 0 8  
 
C h e m o C e n t r y x ,  I n c .  
P r o t o c o l  C L 0 0 2 _ 1 6 8                                                                                                          P a g e  1  o f  #  
T a b l e  1 4 . 3 . 1 . 1  
O v e r v i e w  o f  T r e a t m e n t - e m e r g e n t  A d v e r s e  E v e n t s  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
S a f e t y  P o p u l a t i o n  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
                                                                   P l a c e b o  +             C C X 1 6 8  +            C C X 1 6 8  +  
 S t e p                                                        F u l l  D o s e  P r e d n i s o n e   L o w  D o s e  P r e d n i s o n e    N o  P r e d n i s o n e     A l l  C C X 1 6 8  
   C a t e g o r y                                                          n    ( % )               n    ( % )             n    ( % )          n    ( % )   
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
 O v e r a l l                                                              N = # #                  N = # #                N = # #             N = # #  
   T r e a t m e n t - e m e r g e n t  a d v e r s e  e v e n t  ( T E A E )                          # #  ( # # # . # )            # #  ( # # # . # )          # #  ( # # # . # )       # #  ( # # # . # )  
   P o s s i b l y  S t u d y  M e d i c a t i o n  R e l a t e d  T E A E  
   P o s s i b l y  C o r t i c o s t e r o i d  U s e  R e l a t e d  T E A E  
   P o s s i b l y  C y c l o p h o s p h a m i d e  R e l a t e d  T E A E  
   P o s s i b l y  A z a t h i o p r i n e  R e l a t e d  T E A E  
   P o s s i b l y  R i t u x i m a b  R e l a t e d  T E A E  
 
   M a x i m u m  s e v e r i t y  o f  T E A E  
     M i l d  
     M o d e r a t e  
     S e v e r e  
     L i f e - t h r e a t e n i n g  
     D e a t h  
 
   S e r i o u s  T E A E  
   P o s s i b l y - S t u d y  M e d i c a t i o n  R e l a t e d  S e r i o u s  T E A E  
   P o s s i b l y - C o r t i c o s t e r o i d  U s e  R e l a t e d  S e r i o u s  T E A E  
   P o s s i b l y - C y c l o p h o s p h a m i d e  R e l a t e d  S e r i o u s  T E A E  
   P o s s i b l y  A z a t h i o p r i n e  R e l a t e d  S e r i o u s  T E A E  
   P o s s i b l y  R i t u x i m a b  R e l a t e d  S e r i o u s  T E A E  
 
   D i s c o n t i n u e d  s t u d y  m e d i c a t i o n  d u e  t o  T E A E  
     D u e  t o  P o s s i b l y - S t u d y  M e d i c a t i o n  R e l a t e d  S e r i o u s  T E A E  
     D u e  t o  P o s s i b l y - C o r t i c o s t e r o i d  U s e  R e l a t e d  T E A E  
     D u e  t o  P o s s i b l y - C o r t i c o s t e r o i d  U s e  R e l a t e d  S e r i o u s  T E A E  
     D u e  t o  P o s s i b l y - C y c l o p h o s p h a m i d e  R e l a t e d  T E A E  
     D u e  t o  P o s s i b l y - C y c l o p h o s p h a m i d e  R e l a t e d  S e r i o u s  T E A E  
     D u e  t o  P o s s i b l y - R i t u x i m a b  R e l a t e d  T E A E  
     D u e  t o  P o s s i b l y -  R i t u x i m a b  R e l a t e d  S e r i o u s  T E A E  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
-  A n  a d v e r s e  e v e n t  i s  c o n s i d e r e d  t r e a t m e n t - e m e r g e n t  i f  t h e  s t a r t  d a t e  o f  t h e  e v e n t  i s  o n  o r  a f t e r  a d m i n i s t r a t i o n  o f  t h e  f i r s t  d o s e  
  o f  s t u d y  m e d i c a t i o n .    
 
P r o g r a m  N a m e :  X X X X X X X X . s a s                   R u n  D a t e :  D D M M M Y Y Y Y  H H : M M                                  D a t a b a s e  l a s t  m o d i f i e d :  D D M M Y Y Y Y  
 
N O T E: Re peat f or Ste p 3, 2, a n d 1. 


 
P a ge 1 5 7  of 2 0 8  
 
T he f oll o wi n g t a bles w ill ha ve t he sa me l a y o ut as Ta ble 1 4. 3. 1. 1: 
T a b l e  1 4 . 3 . 1 . 2  
O v e r v i e w  o f  T r e a t m e n t - e m e r g e n t  A d v e r s e  E v e n t s  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
S a f e t y  P o p u l a t i o n  


 
P a ge 1 5 8  of 2 0 8  
 
C h e m o C e n t r y x ,  I n c .  
P r o t o c o l  C L 0 0 2 _ 1 6 8                                                                                                          P a g e  1  o f  #  
T a b l e  1 4 . 3 . 1 . 3  
S u m m a r y  o f  T r e a t m e n t - e m e r g e n t  A d v e r s e  E v e n t s  D u r i n g  t h e  8 4 - D a y  T r e a t m e n t  P e r i o d  
b y  S y s t e m  O r g a n  C l a s s  a n d  P r e f e r r e d  T e r m  
S a f e t y  P o p u l a t i o n  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
 S t e p                                                                  P l a c e b o  +             C C X 1 6 8  +            C C X 1 6 8  +  
  S y s t e m  O r g a n  C l a s s                                             F u l l  D o s e  P r e d n i s o n e   L o w  D o s e  P r e d n i s o n e   N o  P r e d n i s o n e    A l l  C C X 1 6 8  
   P r e f e r r e d  T e r m                                                     n    ( % )                n    ( % )             n    ( % )         n    ( % )   
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
O v e r a l l                                                                N = # #                   N = # #                N = # #            N = # #  
  A n y  T r e a t m e n t - e m e r g e n t  A d v e r s e  E v e n t                               # #  ( # # # . # )             # #  ( # # # . # )          # #  ( # # # . # )      # #  ( # # # . # )  
 
  S y s t e m  O r g a n  C l a s s  1                                               # #  ( # # # . # )             # #  ( # # # . # )          # #  ( # # # . # )      # #  ( # # # . # )  
    P r e f e r r e d  T e r m  1                                                 # #  ( # # # . # )             # #  ( # # # . # )          # #  ( # # # . # )      # #  ( # # # . # )  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
-  A n  a d v e r s e  e v e n t  i s  c o n s i d e r e d  t r e a t m e n t - e m e r g e n t  i f  t h e  s t a r t  d a t e  o f  t h e  e v e n t  i s  o n  o r  a f t e r  a d m i n i s t r a t i o n  o f  t h e  f i r s t  d o s e  
  o f  s t u d y  m e d i c a t i o n .    
 
P r o g r a m  N a m e :  X X X X X X X X . s a s                   R u n  D a t e :  D D M M M Y Y Y Y  H H : M M                            D a t a b a s e  l a s t  m o d i f i e d :  D D M M Y Y Y Y  H H : M M  
 
 
 
N O T E: Ta ble will be s ort e d b y Ste p of t he st u d y, d esce n di n g fre q ue n c y i n t h e t otal c ol u m n f or s yste m or ga n class a n d preferre d ter m 
wit hi n s yst e m or ga n cl ass.  
N O T E: Re peat f or Ste p 3, 2, a n d 1. 


 
P a ge 1 5 9  of 2 0 8  
 
T he f oll o wi n g t a bles w ill ha ve t he sa me l a y o ut as Ta ble 1 4. 3. 1. 3: 
T a b l e  1 4 . 3 . 1 . 4  
S u m m a r y  o f  T r e a t m e n t - e m e r g e n t  A d v e r s e  E v e n t s  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
b y  S y s t e m  O r g a n  C l a s s  a n d  P r e f e r r e d  T e r m  
S a f e t y  P o p u l a t i o n  


 
P a ge 1 6 0  of 2 0 8  
 
C h e m o C e n t r y x ,  I n c .  
P r o t o c o l  C L 0 0 2 _ 1 6 8                                                                                                          P a g e  1  o f  #  
T a b l e  1 4 . 3 . 1 . 5  
S u m m a r y  o f  T r e a t m e n t - e m e r g e n t  A d v e r s e  E v e n t s  d u r i n g  t h e  1 6 8 - d a y  T r e a m e n t  P e r i o d  
b y  S y s t e m  O r g a n  C l a s s ,  P r e f e r r e d  T e r m  a n d  R e l a t i o n s h i p  t o  S t u d y  D r u g  
S a f e t y  P o p u l a t i o n  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
S t e p                                                                   P l a c e b o  +           C C X 1 6 8  +            C C X 1 6 8  +  
  S y s t e m  O r g a n  C l a s s                                                   F u l l  D o s e           L o w  D o s e              N o  
    P r e f e r r e d  T e r m                                                     P r e d n i s o n e         P r e d n i s o n e          P r e d n i s o n e    A l l  C C X 1 6 8  
      R e l a t i o n s h i p  t o  S t u d y  D r u g                                        n    ( % )            n    ( % )             n    ( % )         n    ( % )  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
O v e r a l l                                                                  N = # #               N = # #               N = # #            N = # #  
  A n y  T r e a t m e n t - e m e r g e n t  A d v e r s e  E v e n t                                 # #  ( # # # . # )         # #  ( # # # . # )         # #  ( # # # . # )       # #  ( # # # . # )  
      P o s s i b l y  r e l a t e d  t o  s t u d y  d r u g                                   # #  ( # # # . # )         # #  ( # # # . # )         # #  ( # # # . # )       # #  ( # # # . # )  
      P r o b a b l y  n o t  r e l a t e d  t o  s t u d y  d r u g                               # #  ( # # # . # )         # #  ( # # # . # )         # #  ( # # # . # )       # #  ( # # # . # )  
 
  S y s t e m  O r g a n  C l a s s  1                                                 # #  ( # # # . # )         # #  ( # # # . # )         # #  ( # # # . # )       # #  ( # # # . # )  
      P o s s i b l y  r e l a t e d  t o  s t u d y  d r u g                                   # #  ( # # # . # )         # #  ( # # # . # )         # #  ( # # # . # )       # #  ( # # # . # )  
      P r o b a b l y  n o t  r e l a t e d  t o  s t u d y  d r u g                               # #  ( # # # . # )         # #  ( # # # . # )         # #  ( # # # . # )       # #  ( # # # . # )  
 
    P r e f e r r e d  T e r m  1                                                   # #  ( # # # . # )         # #  ( # # # . # )         # #  ( # # # . # )       # #  ( # # # . # )  
      P o s s i b l y  r e l a t e d  t o  s t u d y  d r u g                                   # #  ( # # # . # )         # #  ( # # # . # )         # #  ( # # # . # )       # #  ( # # # . # )  
      P r o b a b l y  n o t  r e l a t e d  t o  s t u d y  d r u g                               # #  ( # # # . # )         # #  ( # # # . # )         # #  ( # # # . # )       # #  ( # # # . # )  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
-  A n  a d v e r s e  e v e n t  i s  c o n s i d e r e d  t r e a t m e n t - e m e r g e n t  i f  t h e  s t a r t  d a t e  o f  t h e  e v e n t  i s  o n  o r  a f t e r  a d m i n i s t r a t i o n  o f  t h e  f i r s t  d o s e  
  o f  s t u d y  m e d i c a t i o n .    
 
P r o g r a m  N a m e :  X X X X X X X X . s a s                   R u n  D a t e :  D D M M M Y Y Y Y  H H : M M                            D a t a b a s e  l a s t  m o d i f i e d :  D D M M Y Y Y Y  H H : M M  
 
 
 
N O T E: Ta ble will be s orte d b y ste p of t he st u d y, desce n di n g fre q ue n c y i n t he t otal c ol u m n f or s yst e m or ga n class a n d preferre d ter m 
wit hi n s yst e m or ga n cl ass.  
N O T E: Re peat f or Ste p 3, 2, a n d 1. 


 
P a ge 1 6 1  of 2 0 8  
 
C h e m o C e n t r y x ,  I n c .  
P r o t o c o l  C L 0 0 2 _ 1 6 8                                                                                                          P a g e  1  o f  #  
T a b l e  1 4 . 3 . 1 . 6  
S u m m a r y  o f  T r e a t m e n t - e m e r g e n t  A d v e r s e  E v e n t s  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
b y  S y s t e m  O r g a n  C l a s s ,  P r e f e r r e d  T e r m  a n d  R e l a t i o n s h i p  t o  C o r t i c o s t e r o i d  U s e  
S a f e t y  P o p u l a t i o n  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
S t e p                                                                   P l a c e b o  +           C C X 1 6 8  +            C C X 1 6 8  +  
  S y s t e m  O r g a n  C l a s s                                                   F u l l  D o s e           L o w  D o s e              N o  
    P r e f e r r e d  T e r m                                                     P r e d n i s o n e         P r e d n i s o n e          P r e d n i s o n e      A l l  C C X 1 6 8  
      R e l a t i o n s h i p  t o  C o r t i c o s t e r o i d  U s e                                n    ( % )            n    ( % )             n    ( % )         n    ( % )   
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
O v e r a l l                                                                  N = # #               N = # #                N = # #            N = # #  
  A n y  T r e a t m e n t - e m e r g e n t  A d v e r s e  E v e n t                                 # #  ( # # # . # )         # #  ( # # # . # )          # #  ( # # # . # )      # #  ( # # # . # )  
      P o s s i b l y  r e l a t e d  t o  c o r t i c o s t e r o i d  u s e                           # #  ( # # # . # )         # #  ( # # # . # )          # #  ( # # # . # )      # #  ( # # # . # )  
      P r o b a b l y  n o t  r e l a t e d  t o  c o r t i c o s t e r o i d  u s e                       # #  ( # # # . # )         # #  ( # # # . # )          # #  ( # # # . # )      # #  ( # # # . # )  
 
  S y s t e m  O r g a n  C l a s s  1                                                 # #  ( # # # . # )         # #  ( # # # . # )          # #  ( # # # . # )      # #  ( # # # . # )  
      P o s s i b l y  r e l a t e d  t o  c o r t i c o s t e r o i d  u s e                           # #  ( # # # . # )         # #  ( # # # . # )          # #  ( # # # . # )      # #  ( # # # . # )  
      P r o b a b l y  n o t  r e l a t e d  t o  c o r t i c o s t e r o i d  u s e                       # #  ( # # # . # )         # #  ( # # # . # )          # #  ( # # # . # )      # #  ( # # # . # )  
 
    P r e f e r r e d  T e r m  1                                                   # #  ( # # # . # )         # #  ( # # # . # )          # #  ( # # # . # )      # #  ( # # # . # )  
      P o s s i b l y  r e l a t e d  t o  c o r t i c o s t e r o i d  u s e                           # #  ( # # # . # )         # #  ( # # # . # )          # #  ( # # # . # )      # #  ( # # # . # )  
      P r o b a b l y  n o t  r e l a t e d  t o  c o r t i c o s t e r o i d  u s e                       # #  ( # # # . # )         # #  ( # # # . # )          # #  ( # # # . # )      # #  ( # # # . # )  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
-  A n  a d v e r s e  e v e n t  i s  c o n s i d e r e d  t r e a t m e n t - e m e r g e n t  i f  t h e  s t a r t  d a t e  o f  t h e  e v e n t  i s  o n  o r  a f t e r  a d m i n i s t r a t i o n  o f  t h e  f i r s t  d o s e  
  o f  s t u d y  m e d i c a t i o n .    
 
P r o g r a m  N a m e :  X X X X X X X X . s a s                   R u n  D a t e :  D D M M M Y Y Y Y  H H : M M                            D a t a b a s e  l a s t  m o d i f i e d :  D D M M Y Y Y Y  H H : M M  
 
 
N O T E: Ta ble will be s orte d b y ste p of t he st u d y, desce n di n g fre q ue n c y i n t he t otal c ol u m n f or s yst e m or ga n class a n d preferre d ter m 
wit hi n s yst e m or ga n cl ass.  
N O T E: Re pea t f or Ste p 3, 2, a n d 1. 


 
P a ge 1 6 2  of 2 0 8  
 
T he f oll o wi n g t a ble w ill ha ve t he sa me l a y o ut as Ta ble 1 4. 3. 1. 6: 
T a b l e  1 4 . 3 . 1 . 7  
S u m m a r y  o f  T r e a t m e n t - e m e r g e n t  A d v e r s e  E v e n t s  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
b y  S y s t e m  O r g a n  C l a s s ,  P r e f e r r e d  T e r m  a n d  R e l a t i o n s h i p  t o  C o r t i c o s t e r o i d  U s e  
S a f e t y  P o p u l a t i o n  


 
P a ge 1 6 3  of 20 8  
 
C h e m o C e n t r y x ,  I n c .  
P r o t o c o l  C L 0 0 2 _ 1 6 8                                                                                                          P a g e  1  o f  #  
T a b l e  1 4 . 3 . 1 . 8  
S u m m a r y  o f  T r e a t m e n t - e m e r g e n t  A d v e r s e  E v e n t s  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
b y  S y s t e m  O r g a n  C l a s s ,  P r e f e r r e d  T e r m  a n d  R e l a t i o n s h i p  t o  C y c l o p h o s p h a m i d e  U s e  
S a f e t y  P o p u l a t i o n  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
S t e p                                                                 P l a c e b o  +           C C X 1 6 8  +            C C X 1 6 8  +  
  S y s t e m  O r g a n  C l a s s                                                 F u l l  D o s e           L o w  D o s e              N o  
    P r e f e r r e d  T e r m                                                   P r e d n i s o n e         P r e d n i s o n e          P r e d n i s o n e     A l l  C C X 1 6 8  
      R e l a t i o n s h i p  t o  C y c l o p h o s p h a m i d e  U s e                            n    ( % )            n    ( % )             n    ( % )         n    ( % )   
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
O v e r a l l                                                                  N = # #               N = # #                N = # #            N = # #  
  A n y  T r e a t m e n t - e m e r g e n t  A d v e r s e  E v e n t                               # #  ( # # # . # )         # #  ( # # # . # )          # #  ( # # # . # )      # #  ( # # # . # )  
      P o s s i b l y  r e l a t e d  t o  c y c l o p h o s p h a m i d e  u s e                       # #  ( # # # . # )         # #  ( # # # . # )          # #  ( # # # . # )      # #  ( # # # . # )  
      P r o b a b l y  n o t  r e l a t e d  t o  c y c l o p h o s p h a m i d e  u s e                   # #  ( # # # . # )         # #  ( # # # . # )          # #  ( # # # . # )      # #  ( # # # . # )  
 
  S y s t e m  O r g a n  C l a s s  1                                               # #  ( # # # . # )         # #  ( # # # . # )          # #  ( # # # . # )      # #  ( # # # . # )  
      P o s s i b l y  r e l a t e d  t o  c y c l o p h o s p h a m i d e  u s e                       # #  ( # # # . # )         # #  ( # # # . # )          # #  ( # # # . # )      # #  ( # # # . # )  
      P r o b a b l y  n o t  r e l a t e d  t o  c y c l o p h o s p h a m i d e  u s e                   # #  ( # # # . # )         # #  ( # # # . # )          # #  ( # # # . # )      # #  ( # # # . # )  
 
    P r e f e r r e d  T e r m  1                                                 # #  ( # # # . # )         # #  ( # # # . # )          # #  ( # # # . # )      # #  ( # # # . # )  
      P o s s i b l y  r e l a t e d  t o  c y c l o p h o s p h a m i d e  u s e                       # #  ( # # # . # )         # #  ( # # # . # )          # #  ( # # # . # )      # #  ( # # # . # )  
      P r o b a b l y  n o t  r e l a t e d  t o  c y c l o p h o s p h a m i d e  u s e                   # #  ( # # # . # )         # #  ( # # # . # )          # #  ( # # # . # )      # #  ( # # # . # )  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
-  A n  a d v e r s e  e v e n t  i s  c o n s i d e r e d  t r e a t m e n t - e m e r g e n t  i f  t h e  s t a r t  d a t e  o f  t h e  e v e n t  i s  o n  o r  a f t e r  a d m i n i s t r a t i o n  o f  t h e  f i r s t  d o s e  
  o f  s t u d y  m e d i c a t i o n .    
 
P r o g r a m  N a m e :  X X X X X X X X . s a s                   R u n  D a t e :  D D M M M Y Y Y Y  H H : M M                            D a t a b a s e  l a s t  m o d i f i e d :  D D M M Y Y Y Y  H H : M M  
 
 
N O T E: Ta ble will be s orte d b y ste p of t he st u d y, desce n di n g fre q ue n c y i n t he t otal c ol u m n f or s yst e m or ga n class a n d preferre d ter m 
wit hi n s yst e m or ga n cl ass.  
N O T E: Re peat f or Ste p 3, 2, a n d 1. 


 
P a ge 1 6 4  of 2 0 8  
 
T he f oll o wi n g t a ble w ill ha ve t he sa me l a y o ut as Ta ble 1 4. 3. 1. 8: 
T a b l e  1 4 . 3 . 1 . 9  
S u m m a r y  o f  T r e a t m e n t - e m e r g e n t  A d v e r s e  E v e n t s  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
b y  S y s t e m  O r g a n  C l a s s ,  P r e f e r r e d  T e r m  a n d  R e l a t i o n s h i p  t o  C y c l o p h o s p h a m i d e  U s e  
S a f e t y  P o p u l a t i o n  


 
P a ge 1 6 5  of 2 0 8  
 
C h e m o C e n t r y x ,  I n c .  
P r o t o c o l  C L 0 0 2 _ 1 6 8                                                                                                          P a g e  1  o f  #  
T a b l e  1 4 . 3 . 1 . 1 0  
S u m m a r y  o f  T r e a t m e n t - e m e r g e n t  A d v e r s e  E v e n t s  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d   
b y  S y s t e m  O r g a n  C l a s s ,  P r e f e r r e d  T e r m  a n d  R e l a t i o n s h i p  t o  R i t u x i m a b  U s e  
S a f e t y  P o p u l a t i o n  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
S t e p                                                                 P l a c e b o  +           C C X 1 6 8  +            C C X 1 6 8  +  
  S y s t e m  O r g a n  C l a s s                                                 F u l l  D o s e           L o w  D o s e              N o  
    P r e f e r r e d  T e r m                                                   P r e d n i s o n e         P r e d n i s o n e          P r e d n i s o n e     A l l  C C X 1 6 8  
      R e l a t i o n s h i p  t o  R i t u x i m a b  U s e                                   n    ( % )            n    ( % )             n    ( % )         n    ( % )   
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
S t e p  3                                                                N = # #               N = # #                N = # #            N = # #  
  A n y  T r e a t m e n t - e m e r g e n t  A d v e r s e  E v e n t                               # #  ( # # # . # )         # #  ( # # # . # )          # #  ( # # # . # )      # #  ( # # # . # )  
      P o s s i b l y  r e l a t e d  t o  r i t u x i m a b  u s e                              # #  ( # # # . # )         # #  ( # # # . # )          # #  ( # # # . # )      # #  ( # # # . # )  
      P r o b a b l y  n o t  r e l a t e d  t o  r i t u x i m a b  u s e                          # #  ( # # # . # )         # #  ( # # # . # )          # #  ( # # # . # )      # #  ( # # # . # )  
 
  S y s t e m  O r g a n  C l a s s  1                                               # #  ( # # # . # )         # #  ( # # # . # )          # #  ( # # # . # )      # #  ( # # # . # )  
      P o s s i b l y  r e l a t e d  t o  r i t u x i m a b  u s e                              # #  ( # # # . # )         # #  ( # # # . # )          # #  ( # # # . # )      # #  ( # # # . # )  
      P r o b a b l y  n o t  r e l a t e d  t o  r i t u x i m a b  u s e                          # #  ( # # # . # )         # #  ( # # # . # )          # #  ( # # # . # )      # #  ( # # # . # )  
 
    P r e f e r r e d  T e r m  1                                                 # #  ( # # # . # )         # #  ( # # # . # )          # #  ( # # # . # )      # #  ( # # # . # )  
      P o s s i b l y  r e l a t e d  t o  r i t u x i m a b  u s e                              # #  ( # # # . # )         # #  ( # # # . # )          # #  ( # # # . # )      # #  ( # # # . # )  
      P r o b a b l y  n o t  r e l a t e d  t o  r i t u x i m a b  u s e                          # #  ( # # # . # )         # #  ( # # # . # )          # #  ( # # # . # )      # #  ( # # # . # )  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
-  A n  a d v e r s e  e v e n t  i s  c o n s i d e r e d  t r e a t m e n t - e m e r g e n t  i f  t h e  s t a r t  d a t e  o f  t h e  e v e n t  i s  o n  o r  a f t e r  a d m i n i s t r a t i o n  o f  t h e  f i r s t  d o s e  
  o f  s t u d y  m e d i c a t i o n .    
 
P r o g r a m  N a m e :  X X X X X X X X . s a s                   R u n  D a t e :  D D M M M Y Y Y Y  H H : M M                            D a t a b a s e  l a s t  m o d i f i e d :  D D M M Y Y Y Y  H H : M M  
 
 
N O T E: Ta ble will be s orte d b y ste p of t he st u d y, desce n di n g fre q ue n c y i n t he t otal c ol u m n f or s yst e m or ga n class a n d preferre d ter m 
wit hi n s yst e m or ga n cl ass. Als o n ote t hat rit u xi ma b was o nl y all o we d i n Ste p 3. 


 
P a ge 1 6 6  of 2 0 8  
 
T he f oll o wi n g t a ble will ha ve t he sa me l a y o ut as Ta ble 1 4. 3. 1. 1 0: 
T a b l e  1 4 . 3 . 1 . 1 1  
S u m m a r y  o f  T r e a t m e n t - e m e r g e n t  A d v e r s e  E v e n t s  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
b y  S y s t e m  O r g a n  C l a s s ,  P r e f e r r e d  T e r m  a n d  R e l a t i o n s h i p  t o  R i t u x i m a b  U s e  
S a f e t y  P o p u l a t i o n  


 
P a ge 1 6 7  of 2 0 8  
 
C h e m o C e n t r y x ,  I n c .  
P r o t o c o l  C L 0 0 2 _ 1 6 8                                                                                                          P a g e  1  o f  #  
T a b l e  1 4 . 3 . 1 . 1 2  
S u m m a r y  o f  T r e a t m e n t - e m e r g e n t  A d v e r s e  E v e n t s  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
b y  S y s t e m  O r g a n  C l a s s ,  P r e f e r r e d  T e r m  a n d  M a x i m u m  S e v e r i t y  
S a f e t y  P o p u l a t i o n  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
S t e p                                                                 P l a c e b o  +           C C X 1 6 8  +            C C X 1 6 8  +  
  S y s t e m  O r g a n  C l a s s                                                 F u l l  D o s e           L o w  D o s e              N o  
    P r e f e r r e d  T e r m                                                   P r e d n i s o n e         P r e d n i s o n e          P r e d n i s o n e     A l l  C C X 1 6 8  
      M a x i m u m  S e v e r i t y                                                n    ( % )            n    ( % )             n    ( % )         n    ( % )   
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
O v e r a l l                                                                N = # #               N = # #                N = # #  
  A n y  T r e a t m e n t - e m e r g e n t  A d v e r s e  E v e n t                               # #  ( # # # . # )         # #  ( # # # . # )          # #  ( # # # . # )      # #  ( # # # . # )  
      M i l d                                                           # #  ( # # # . # )         # #  ( # # # . # )          # #  ( # # # . # )      # #  ( # # # . # )  
      M o d e r a t e                                                       # #  ( # # # . # )         # #  ( # # # . # )          # #  ( # # # . # )      # #  ( # # # . # )  
      S e v e r e                                                         # #  ( # # # . # )         # #  ( # # # . # )          # #  ( # # # . # )      # #  ( # # # . # )  
      L i f e - t h r e a t e n i n g                                               # #  ( # # # . # )         # #  ( # # # . # )          # #  ( # # # . # )      # #  ( # # # . # )  
      D e a t h                                                          # #  ( # # # . # )         # #  ( # # # . # )          # #  ( # # # . # )      # #  ( # # # . # )  
 
  S y s t e m  O r g a n  C l a s s  1                                               # #  ( # # # . # )         # #  ( # # # . # )          # #  ( # # # . # )  
      M i l d                                                           # #  ( # # # . # )         # #  ( # # # . # )          # #  ( # # # . # )      # #  ( # # # . # )  
      M o d e r a t e                                                       # #  ( # # # . # )         # #  ( # # # . # )          # #  ( # # # . # )      # #  ( # # # . # )  
      S e v e r e                                                         # #  ( # # # . # )         # #  ( # # # . # )          # #  ( # # # . # )      # #  ( # # # . # )  
      L i f e - t h r e a t e n i n g                                               # #  ( # # # . # )         # #  ( # # # . # )          # #  ( # # # . # )      # #  ( # # # . # )  
      D e a t h                                                          # #  ( # # # . # )         # #  ( # # # . # )          # #  ( # # # . # )      # #  ( # # # . # )  
 
    P r e f e r r e d  T e r m  1                                                 # #  ( # # # . # )         # #  ( # # # . # )          # #  ( # # # . # )  
      M i l d                                                           # #  ( # # # . # )         # #  ( # # # . # )          # #  ( # # # . # )      # #  ( # # # . # )  
      M o d e r a t e                                                       # #  ( # # # . # )         # #  ( # # # . # )          # #  ( # # # . # )      # #  ( # # # . # )  
      S e v e r e                                                         # #  ( # # # . # )         # #  ( # # # . # )          # #  ( # # # . # )      # #  ( # # # . # )  
      L i f e - t h r e a t e n i n g                                               # #  ( # # # . # )         # #  ( # # # . # )          # #  ( # # # . # )      # #  ( # # # . # )  
      D e a t h                                                          # #  ( # # # . # )         # #  ( # # # . # )          # #  ( # # # . # )      # #  ( # # # . # )  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
-  A n  a d v e r s e  e v e n t  i s  c o n s i d e r e d  t r e a t m e n t - e m e r g e n t  i f  t h e  s t a r t  d a t e  o f  t h e  e v e n t  i s  o n  o r  a f t e r  a d m i n i s t r a t i o n  o f  t h e  f i r s t  d o s e  
  o f  s t u d y  m e d i c a t i o n .    
 
P r o g r a m  N a m e :  X X X X X X X X . s a s                   R u n  D a t e :  D D M M M Y Y Y Y  H H : M M                            D a t a b a s e  l a s t  m o d i f i e d :  D D M M Y Y Y Y  H H : M M  
 
 
N O T E: Ta ble will be s orte d b y Ste p of t he st u d y, desce n di n g fre q u e nc y i n t he t otal c ol u m n f or s yst e m or ga n class a n d preferre d ter m 
wit hi n s yst e m or ga n cl ass.  
N O T E: Re peat f or Ste p 3, 2, a n d 1. 
N O T E: If t here are n o e v e nts i n a cate g or y, e. g. Deat h, t he r o w will n ot a p pear i n t he s u m mar y ta bl e. 


 
P a ge 1 6 8  of 2 0 8  
 
T he f oll o wi n g t a ble w ill ha ve t he sa me l a y o ut as Ta ble 1 4. 3. 1. 1 2: 
T a b l e  1 4 . 3 . 1 . 1 3  
S u m m a r y  o f  T r e a t m e n t - e m e r g e n t  A d v e r s e  E v e n t s  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
b y  S y s t e m  O r g a n  C l a s s ,  P r e f e r r e d  T e r m  a n d  M a x i m u m  S e v e r i t y  
S a f e t y  P o p u l a t i o n  


 
P a ge 1 6 9  of 2 0 8  
 
C h e m o C e n t r y x ,  I n c .  
P r o t o c o l  C L 0 0 2 _ 1 6 8                                                                                                          P a g e  1  o f  #  
T a b l e  1 4 . 3 . 1 . 1 4  
S u m m a r y  o f  T r e a t m e n t - e m e r g e n t  A d v e r s e  E f f e c t s  P o s s i b l y  A s s o c i a t e d  w i t h  G l u c o c o r t i c o i d  U s e  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
b y  S y s t e m  O r g a n  C l a s s  a n d  P r e f e r r e d  T e r m  
S a f e t y  P o p u l a t i o n  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
 S t e p                                                                  P l a c e b o  +             C C X 1 6 8  +            C C X 1 6 8  +  
  S y s t e m  O r g a n  C l a s s                                             F u l l  D o s e  P r e d n i s o n e   L o w  D o s e  P r e d n i s o n e   N o  P r e d n i s o n e    A l l  C C X 1 6 8  
   P r e f e r r e d  T e r m                                                     n    ( % )                n    ( % )             n    ( % )         n    ( % )   
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
O v e r a l l                                                                N = # #                   N = # #                N = # #            N = # #  
  A n y  T r e a t m e n t - e m e r g e n t  A d v e r s e  E f f e c t                            # #  ( # # # . # )             # #  ( # # # . # )          # #  ( # # # . # )      # #  ( # # # . # )  
 
  S e r i o u s  I n f e c t i o n s                                               # #  ( # # # . # )             # #  ( # # # . # )          # #  ( # # # . # )      # #  ( # # # . # )  
  N e w  o n s e t  d i a b e t e s / h y p e r g l y c e m i a                                 # #  ( # # # . # )             # #  ( # # # . # )          # #  ( # # # . # )      # #  ( # # # . # )  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
   
 
P r o g r a m  N a m e :  X X X X X X X X . s a s                   R u n  D a t e :  D D M M M Y Y Y Y  H H : M M                            D a t a b a s e  l a s t  m o d i f i e d :  D D M M Y Y Y Y  H H : M M  
 
N ote t o Pr o gra m mer:  T h ese a d verse effects will be i de ntifie d as f oll o ws: 
 
Seri o us i nfecti o ns: All S A Es i n t he S yste m Or ga n Class I nfecti o ns a n d I nfestati o ns 
 
Ne w- o nset dia betes mellit us/ h y per gl yce mia: All T E A Es of h y per gl yce mia, dia betes, i ncrease d bl o o d gl uc ose, pl us all patie nts wit h a 
fasti n g bl o o d gl uc ose le vel p ost baseli ne t hat is a b o ve t he u p p er li mit of n or mal o n at least t w o c o nsec uti ve st u d y visits. 
 
B o ne fract ure: All T E A Es i n dicati n g  l o n g b o ne or verte bral fract ures 
 
Pe ptic ulcer disease: All T E A Es i n dicati n g u p per gastr oi ntesti nal ulcerati o n, er osi o n, or blee di n g 
 
Cataracts: All T E A Es of cataract 
 
Ne w o nset/ w orse ni n g h y perte nsi o n: All T E A Es of h y perte nsi o n, w orse ni n g h y perte nsi o n, or hi g h bl o o d press ure, pl us all patie nts wit h a 
s yst olic bl o o d press ure i ncrease of at least 2 0 m m H g fr o m baseli ne, a n d > 1 4 0 m m H g (s yst olic), or diast olic bl o o d press ure i ncrease of at 
least 1 0 m m H g fr o m baseli ne, a n d > 9 0 m m H g ( diast olic), t hat is prese nt o n at least t w o c o nsec uti ve st u d y visits. 
 
Wei g ht gai n m ore t ha n 1 0 k g: C ha n ge fr o m baseli ne i n wei g ht of > 1 0 k g. 
 
Ps yc hiatric dis or ders: All T E A Es of ps yc h osis, a n xiet y, a m nesia, c o n v ulsi o ns, deliri u m, de me ntia, de pressi o n, 
ma nia, e m oti o nal 
i nsta bilit y, irrita bilit y, e u p h oria, hall uci nati o ns, i m paire d c o g niti o n, i ncrease d m ot or acti vit y, i ns o m nia, me m or y l oss, ma nia, m o o d 
s wi n gs, ne uritis, ne ur o pat h y, parest hesi a, pers o nalit y c ha n ges, restless ness, sc hiz o p hre nia, verti g o, or wit h dra wal be ha vi or. 


 
P a ge 1 7 0  of 2 0 8  
 
T he f oll o wi n g t a ble w ill ha ve t he sa me l a y o ut as Ta ble 1 4. 3. 1. 1 4: 
T a b l e  1 4 . 3 . 1 . 1 5  
S u m m a r y  o f  T r e a t m e n t - e m e r g e n t  A d v e r s e  E f f e c t s  P o s s i b l y  A s s o c i a t e d  w i t h  G l u c o c o r t i c o i d  U s e  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
b y  S y s t e m  O r g a n  C l a s s  a n d  P r e f e r r e d  T e r m  
S a f e t y  P o p u l a t i o n  


 
P a ge 1 7 1  of 2 0 8  
 
C h e m o C e n t r y x ,  I n c .  
P r o t o c o l  C L 0 0 2 _ 1 6 8                                                                                                          P a g e  1  o f  #  
T a b l e  1 4 . 3 . 1 . 1 6  
S u m m a r y  o f  T r e a t m e n t - e m e r g e n t  I n f e c t i o n s *  d u r i n g  t h e  8 4 - d a y  T r e a t m e n t  P e r i o d  
b y  S y s t e m  O r g a n  C l a s s  a n d  P r e f e r r e d  T e r m  
S a f e t y  P o p u l a t i o n  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
 S t e p                                                                  P l a c e b o  +             C C X 1 6 8  +            C C X 1 6 8  +  
  S y s t e m  O r g a n  C l a s s                                             F u l l  D o s e  P r e d n i s o n e   L o w  D o s e  P r e d n i s o n e   N o  P r e d n i s o n e    A l l  C C X 1 6 8  
   P r e f e r r e d  T e r m                                                     n    ( % )                n    ( % )             n    ( % )         n    ( % )   
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
O v e r a l l                                                                N = # #                   N = # #                N = # #            N = # #  
  A n y  T r e a t m e n t - e m e r g e n t  I n f e c t i o n                                   # #  ( # # # . # )             # #  ( # # # . # )          # #  ( # # # . # )      # #  ( # # # . # )  
  A n y  S e r i o u s  T r e a t m e n t - e m e r g e n t  I n f e c t i o n                           # #  ( # # # . # )             # #  ( # # # . # )          # #  ( # # # . # )      # #  ( # # # . # )  
  A n y  S e v e r e  T r e a t m e n t - e m e r g e n t  I n f e c t i o n                            # #  ( # # # . # )             # #  ( # # # . # )          # #  ( # # # . # )      # #  ( # # # . # )  
  A n y  T r e a t m e n t - e m e r g e n t  I n f e c t i o n  L e a d i n g  t o  W i t h d r a w a l             # #  ( # # # . # )             # #  ( # # # . # )          # #  ( # # # . # )      # #  ( # # # . # )  
 
  S y s t e m  O r g a n  C l a s s  1                                               # #  ( # # # . # )             # #  ( # # # . # )          # #  ( # # # . # )      # #  ( # # # . # )  
    P r e f e r r e d  T e r m  1                                                 # #  ( # # # . # )             # #  ( # # # . # )          # #  ( # # # . # )      # #  ( # # # . # )  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
-  A n  a d v e r s e  e v e n t  i s  c o n s i d e r e d  t r e a t m e n t - e m e r g e n t  i f  t h e  s t a r t  d a t e  o f  t h e  e v e n t  i s  o n  o r  a f t e r  a d m i n i s t r a t i o n  o f  t h e  f i r s t  d o s e  
  o f  s t u d y  m e d i c a t i o n .    
* S u m m a r y  t a b l e  i n c l u d e s  t r e a t m e n t - e m e r g e n t  i n f e c t i o n s  i n c l u d i n g  s e r i o u s  i n f e c t i o n s ,  s e v e r e  i n f e c t i o n s  ( i . e . ,  G r a d e  3 ) ,  a n d  i n f e c t i o n s  
 L e a d i n g  t o  w i t h d r a w a l  f r o m  t h e  s t u d y .  
 
P r o g r a m  N a m e :  X X X X X X X X . s a s                   R u n  D a t e :  D D M M M Y Y Y Y  H H : M M                            D a t a b a s e  l a s t  m o d i f i e d :  D D M M Y Y Y Y  H H : M M  


 
P a ge 1 7 2  of 2 0 8  
 
T he f oll o wi n g t a ble w ill ha ve t he sa me l a y o ut as Ta ble 1 4. 3. 1. 1 6: 
T a b l e  1 4 . 3 . 1 . 1 7  
S u m m a r y  o f  T r e a t m e n t - e m e r g e n t  I n f e c t i o n s *  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
b y  S y s t e m  O r g a n  C l a s s  a n d  P r e f e r r e d  T e r m  
S a f e t y  P o p u l a t i o n  


 
P a ge 1 7 3  of 2 0 8  
 
T he f oll o wi n g t a bles w ill ha ve t he sa me l a y o uts as Ta bles i n Secti o n 1 4. 3. 1: 
T a b l e  1 4 . 3 . 1 . 1 8  
S u m m a r y  o f  S e r i o u s  T r e a t m e n t - e m e r g e n t  A d v e r s e  E v e n t s  d u r i n g  t h e  8 4 - D a y  T r e a t m e n t  P e r i o d  
b y  S y s t e m  O r g a n  C l a s s  a n d  P r e f e r r e d  T e r m  
S a f e t y  P o p u l a t i o n  
 
T a b l e  1 4 . 3 . 1 . 1 9  
S u m m a r y  o f  S e r i o u s  T r e a t m e n t - e m e r g e n t  A d v e r s e  E v e n t s  d u r i n g  t h e  1 6 8 - d a y  S t u d y  P e r i o d  
b y  S y s t e m  O r g a n  C l a s s  a n d  P r e f e r r e d  T e r m  
S a f e t y  P o p u l a t i o n  
 
T a b l e  1 4 . 3 . 1 . 2 0  
S u m m a r y  o f  T r e a t m e n t - e m e r g e n t  A d v e r s e  E v e n t s  L e a d i n g  t o  D i s c o n t i n u a t i o n  o f  S t u d y  M e d i c a t i o n   
S a f e t y  P o p u l a t i o n  


 
P a ge 1 7 4  of 2 0 8  
 
C h e m o C e n t r y x ,  I n c .  
P r o t o c o l  C L 0 0 2 _ 1 6 8                                                                                                          P a g e  1  o f  #  
T a b l e  1 4 . 3 . 2 . 1  
L i s t i n g  o f  S e r i o u s  A d v e r s e  E v e n t s  
S a f e t y  P o p u l a t i o n  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
 S t e p                 R T :  R e p o r t e d  T e r m                                                                           - - - - - - - -  S A E  - - - - - - - -  
  T r e a t m e n t           O C :  P r i m a r y  S y s t e m  O r g a n  C l a s s       S t a r t  D a t e  ( D a y ) /           R e l a t e d -  A c t i o n    O u t -       I m p r o v e /      R e a p p e a r /  
    S u b j e c t    T E A E ?   P T :  P r e f e r r e d  T e r m                   E n d  D a t e  ( D a y )    S e v e r i t y   n e s s [ 1 ]   T a k e n [ 2 ]  c o m e [ 3 , 4 ]  D i s a p p e a r [ 5 ]  W o r s e n [ 6 ]  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
 S t e p  3  
  X X X X X X X X X X X X X X X X X X X X X X X X X X X X X   
    # # # - # # #     X X X    R T : X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X    D D M M M Y Y Y Y  ( # # # ) /   X X X X X X X X   # / # / #          #        # / #      X X           X X  
                     O C : X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X    D D M M M Y Y Y Y  ( # # # )  
                     P T : X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X  
 
    L a s t  D o s e  D a t e  ( D a y ) :   D D M M M Y Y Y Y  ( # # # )      C u m u l a t i v e  D o s e  ( m g )  [ 7 ] : # # #   
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
-  A n  a d v e r s e  e v e n t  i s  c o n s i d e r e d  t r e a t m e n t - e m e r g e n t  i f  t h e  s t a r t  d a t e  o f  t h e  e v e n t  i s  o n  o r  a f t e r  a d m i n i s t r a t i o n  o f  t h e  f i r s t  d o s e  
  o f  s t u d y  m e d i c a t i o n .  ‘ D a y ’  i s  t h e  n u m b e r  o f  d a y s  f r o m  t h e  d a t e  o f  r a n d o m i z a t i o n .   
[ 1 ]  R e l a t e d n e s s : ( S t u d y  M e d i c a t i o n / C o r t i c o s t e r o i d / C y c l o p h o s p h a m i d e )  0 = P r o b a b l y  N o t  R e l a t e d ,  1 = P o s s i b l y  R e l a t e d  
[ 2 ]  A c t i o n s  T a k e n :  1 = N o n e ,  2 = S t u d y  m e d i c a t i o n  d i s c o n t i n u e d ,  3 = S t u d y  m e d i c a t i o n  i n t e r r u p t e d  
[ 3 ]  O u t c o m e :  1 = R e s o l v e d ,  2 = R e s o l v e d  w i t h  s e q u e l a e ,  3 = O n g o i n g ,  4 = D e a t h ,  5 = U n k n o w n   
[ 4 ]  S e r i o u s  O u t c o m e :  1 = R e s u l t s  i n  d e a t h ,  2 = L i f e  t h r e a t e n i n g ,  3 = I n p a t i e n t  h o s p i t a l i z a t i o n  o r  p r o l o n g a t i o n  o f  e x i s t i n g  h o s p i t a l i z a t i o n ,  
                     4 = P e r s i s t e n t  o r  s i g n i f i c a n t  d i s a b i l i t y / i n c a p a c i t y ,  5 = C o n g e n i t a l  a b n o r m a l i t y  o r  b i r t h  d e f e c t ,  
                     6 = I m p o r t a n t  m e d i c a l  e v e n t   
[ 5 ]  D i d  t h e  e v e n t  i m p r o v e  o r  d i s a p p e a r  a f t e r  s t o p p i n g  s t u d y  m e d i c a t i o n  ( d e c h a l l e n g e ) ?  
[ 6 ]  D i d  t h e  e v e n t  r e a p p e a r  o r  w o r s e n  a f t e r  r e s t a r t i n g  s t u d y  m e d i c a t i o n  ( r e c h a l l e n g e ) ?  
[ 7 ]  R e p r e s e n t s  t h e  c u m u l a t i v e  d o s e  ( m g )  o f  C C X 1 6 8  t a k e n  p r i o r  t o  o n s e t  o f  e v e n t .  
 
P r o g r a m  N a m e :  X X X X X X X X . s a s                   R u n  D a t e :  D D M M M Y Y Y Y  H H : M M                            D a t a b a s e  l a s t  m o d i f i e d :  D D M M Y Y Y Y  H H : M M  
 
 
N O T E: T his ta ble will be s orte d b y Ste p of t he st u d y, treat me nt gr o u p a n d s u bject wit hi n treat me nt gr o u p. 
N O T E: Re peat f or Ste ps 2 a n d 1.  
N O T E: C u m ulati ve d ose f or place b o s u bjects will be 0 m g. 


 
P a ge 1 7 5  of 2 0 8  
 
T h e f oll o wi n g t a ble w ill ha ve t he sa me l a y o ut as Ta ble 1 4. 3. 2. 1: 
T a b l e  1 4 . 3 . 2 . 2  
L i s t i n g  o f  A d v e r s e  E v e n t s  L e a d i n g  t o  D i s c o n t i n u a t i o n  o f  S t u d y  M e d i c a t i o n  
S a f e t y  P o p u l a t i o n  
 
T a b l e  1 4 . 3 . 2 . 3  
L i s t i n g  o f  T r e a t m e n t - e m e r g e n t  I n f e c t i o n s  
S a f e t y  P o p u l a t i o n  


 
P a ge 1 7 6  of 2 0 8  
 
C h e m o C e n t r y x ,  I n c .  
P r o t o c o l  C L 0 0 2 _ 1 6 8                                                                                                          P a g e  1  o f  #  
T a b l e  1 4 . 3 . 4 . 1  
S u m m a r y  o f  C h a n g e s  i n  H e m a t o l o g y  L a b o r a t o r y  P a r a m e t e r s  b y  V i s i t  
S a f e t y  P o p u l a t i o n  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
 S t e p  
  P a r a m e t e r               P l a c e b o  +                       C C X 1 6 8  +                        C C X 1 6 8  +   
   S t u d y  D a y     - -  F u l l  D o s e  P r e d n i s o n e  - -      - -  L o w  D o s e  P r e d n i s o n e  - -       - - - - -  N o  P r e d n i s o n e  - - - - -       - - - - - - -  A l l  C C X 1 6 8  - - - - - -  
    S t a t i s t i c        V i s i t         C h a n g e            V i s i t         C h a n g e            V i s i t         C h a n g e             V i s i t         C h a n g e  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
 O v e r a l l                    N = # #                           N = # #                           N = # #                            N = # #  
  P a r a m e t e r  1  ( U n i t )   
   B a s e l i n e  
     N ’              # #                             # #                             # #                              # #  
     M e a n            # # . #                           # # . #                           # # . #                            # # . #  
     S D              # # . # #                          # # . # #                          # # . # #                           # # . # #  
     S E M             # # . # #                          # # . # #                          # # . # #                           # # . # #  
     M i n i m u m         # #                             # #                             # #                              # #  
     M e d i a n          # # . #                           # # . #                           # # . #                            # # . #  
     M a x i m u m         # #                             # #                             # #                              # #   
 
   D a y  2  
     N ’              # #             # #               # #             # #               # #             # #                # #             # #  
     M e a n            # # . #           # # . #             # # . #           # # . #             # # . #           # # . #              # # . #           # # . #  
     S D              # # . # #          # # . # #            # # . # #          # # . # #            # # . # #          # # . # #             # # . # #          # # . # #  
     S E M             # # . # #          # # . # #            # # . # #          # # . # #            # # . # #          # # . # #             # # . # #          # # . # #  
     M i n i m u m         # #             # #               # #             # #               # #             # #                # #             # #  
     M e d i a n          # # . #           # # . #             # # . #           # # . #             # # . #           # # . #              # # . #           # # . #  
     M a x i m u m         # #             # #               # #             # #               # #             # #                # #             # #   
 
  T h e  l a b o r a t o r y  p a r a m e t e r s  t o  b e  s u m m a r i z e d  i n c l u d e :      
    H e m a t o l o g y :  H e m o g l o b i n ,  H e m a t o c r i t ,  R B C  C o u n t ,  W B C  C o u n t  ( w i t h  b o t h  a b s o l u t e  a n d  %  d i f f e r e n t i a l ) ,  P l a t e l e t  c o u n t ,  M C H ,  M C H C  a n d   
                M C V .  
 
  T h e  v i s i t s  t o  b e  s u m m a r i z e d  i n c l u d e :  
     D a y  2 ,  D a y  8 ,  D a y  1 5 ,  D a y  2 9 ,  D a y  4 3 ,  D a y  7 1 ,  D a y  8 5 ,  E n d  o f  T r e a t m e n t ,  D a y  9 9 ,  D a y  1 4 1 ,  D a y  1 6 9 ,  E n d  o f  F o l l o w - u p  P e r i o d .  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
-  B a s e l i n e  i s  d e f i n e d  a s  t h e  l a s t  p r e - d o s e  v a l u e .  
-  N ' = n u m b e r  o f  s u b j e c t s  w i t h  d a t a  a t  b a s e l i n e  a n d  t h e  s p e c i f i e d  v i s i t .  
-  T h e  E n d  o f  T r e a t m e n t  v a l u e  i s  t h e  l a s t  m e a s u r e m e n t  t h r o u g h  D a y  8 5 .  
-  T h e  E n d  o f  F o l l o w - u p  P e r i o d  i s  t h e  l a s t  m e a s u r e m e n t  a f t e r  D a y  8 5  t h r o u g h  D a y  1 6 9 .  
 
P r o g r a m  N a m e :  X X X X X X X X . s a s                   R u n  D a t e :  D D M M M Y Y Y Y  H H : M M                            D a t a b a s e  l a s t  m o d i f i e d :  D D M M Y Y Y Y  H H : M M  
 
N O T E: Re peat f or Ste ps 3, 2 a n d 1.  


 
P a ge 1 7 7  of 2 0 8  
 
C h e m o C e n t r y x ,  I n c .  
P r o t o c o l  C L 0 0 2 _ 1 6 8                                                                                                          P a g e  1  o f  #  
T a b l e  1 4 . 3 . 4 . 2  
S u m m a r y  o f  C h a n g e s  i n  C h e m i s t r y  L a b o r a t o r y  P a r a m e t e r s  b y  V i s i t  
S a f e t y  P o p u l a t i o n  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
S t e p  
  P a r a m e t e r               P l a c e b o  +                       C C X 1 6 8  +                        C C X 1 6 8  +   
   S t u d y  D a y     - -  F u l l  D o s e  P r e d n i s o n e  - -      - -  L o w  D o s e  P r e d n i s o n e  - -       - - - - -  N o  P r e d n i s o n e  - - - - -       - - - - - - -  A l l  C C X 1 6 8  - - - - - -  
    S t a t i s t i c        V i s i t         C h a n g e            V i s i t         C h a n g e            V i s i t         C h a n g e             V i s i t         C h a n g e  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
O v e r a l l                    N = # #                           N = # #                           N = # #                            N = # #  
  P a r a m e t e r  1  ( U n i t )   
   B a s e l i n e  
     N ’              # #                             # #                             # #                              # #  
     M e a n            # # . #                           # # . #                           # # . #                            # # . #  
     S D              # # . # #                          # # . # #                          # # . # #                           # # . # #  
     S E M             # # . # #                          # # . # #                          # # . # #                           # # . # #  
     M i n i m u m         # #                             # #                             # #                              # #  
     M e d i a n          # # . #                           # # . #                           # # . #                            # # . #  
     M a x i m u m         # #                             # #                             # #                              # #   
 
   D a y  2  
     N ’              # #             # #               # #             # #               # #             # #                # #             # #  
     M e a n            # # . #           # # . #             # # . #           # # . #             # # . #           # # . #              # # . #           # # . #  
     S D              # # . # #          # # . # #            # # . # #          # # . # #            # # . # #          # # . # #             # # . # #          # # . # #  
     S E M             # # . # #          # # . # #            # # . # #          # # . # #            # # . # #          # # . # #             # # . # #          # # . # #  
     M i n i m u m         # #             # #               # #             # #               # #             # #                # #             # #  
     M e d i a n          # # . #           # # . #             # # . #           # # . #             # # . #           # # . #              # # . #           # # . #  
     M a x i m u m         # #             # #               # #             # #               # #             # #                # #             # #   
 
  T h e  l a b o r a t o r y  p a r a m e t e r s  t o  b e  s u m m a r i z e d  i n c l u d e :      
    T o t a l  B i l i r u b i n ,  L D H ,  S G O T / A S T ,  S G P T / A L T ,  B U N ,  C r e a t i n i n e ,  C P K ,  A l b u m i n ,  S o d i u m ,  P o t a s s i u m ,  B i c a r b o n a t e ,  C h l o r i d e ,   
    C a l c i u m ,  I n o r g a n i c  p h o s p h o r u s ,  G l u c o s e ,  T o t a l  p r o t e i n ,  A l k a l i n e  p h o s p h a t a s e ,  C h o l e s t e r o l  a n d  U r i c  a c i d .  
 
  T h e  v i s i t s  t o  b e  s u m m a r i z e d  i n c l u d e :  
     D a y  2 ,  D a y  8 ,  D a y  1 5 ,  D a y  2 9 ,  D a y  4 3 ,  D a y  7 1 ,  D a y  8 5 ,  E n d  o f  T r e a t m e n t ,  D a y  9 9 ,  D a y  1 4 1 ,  D a y  1 6 9 ,  E n d  o f  F o l l o w - u p  P e r i o d .  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
-  B a s e l i n e  i s  d e f i n e d  a s  t h e  l a s t  p r e - d o s e  v a l u e .  
-  N ' = n u m b e r  o f  s u b j e c t s  w i t h  d a t a  a t  b a s e l i n e  a n d  t h e  s p e c i f i e d  v i s i t .  
-  T h e  E n d  o f  T r e a t m e n t  v a l u e  i s  t h e  l a s t  m e a s u r e m e n t  t h r o u g h  D a y  8 5 .  
-  T h e  E n d  o f  F o l l o w - u p  P e r i o d  i s  t h e  l a s t  m e a s u r e m e n t  a f t e r  D a y  8 5  t h r o u g h  D a y  1 6 9 .  
 
P r o g r a m  N a m e :  X X X X X X X X . s a s                   R u n  D a t e :  D D M M M Y Y Y Y  H H : M M                            D a t a b a s e  l a s t  m o d i f i e d :  D D M M Y Y Y Y  H H : M M  
 
N O T E: Re peat f or Ste ps 3, 2 a n d 1.  


 
P a ge 1 7 8  of 2 0 8  
 
C h e m o C e n t r y x ,  I n c .  
P r o t o c o l  C L 0 0 2 _ 1 6 8                                                                                                          P a g e  1  o f  #  
T a b l e  1 4 . 3 . 4 . 3  
S u m m a r y  o f  C h a n g e s  i n  U r i n a l y s i s  L a b o r a t o r y  P a r a m e t e r s  b y  V i s i t  
S a f e t y  P o p u l a t i o n  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
S t e p  
  P a r a m e t e r               P l a c e b o  +                       C C X 1 6 8  +                        C C X 1 6 8  +   
   S t u d y  D a y     - -  F u l l  D o s e  P r e d n i s o n e  - -      - -  L o w  D o s e  P r e d n i s o n e  - -       - - - - -  N o  P r e d n i s o n e  - - - - -       - - - - - - -  A l l  C C X 1 6 8  - - - - - -  
    S t a t i s t i c        V i s i t         C h a n g e            V i s i t         C h a n g e            V i s i t         C h a n g e             V i s i t         C h a n g e  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
O v e r a l l                    N = # #                           N = # #                           N = # #                            N = # #  
  P a r a m e t e r  1  ( U n i t )   
   B a s e l i n e  
     N ’              # #                             # #                             # #                              # #  
     M e a n            # # . #                           # # . #                           # # . #                            # # . #  
     S D              # # . # #                          # # . # #                          # # . # #                           # # . # #  
     S E M             # # . # #                          # # . # #                          # # . # #                           # # . # #  
     M i n i m u m         # #                             # #                             # #                              # #  
     M e d i a n          # # . #                           # # . #                           # # . #                            # # . #  
     M a x i m u m         # #                             # #                             # #                              # #   
 
   D a y  2  
     N ’              # #             # #               # #             # #               # #             # #                # #             # #  
     M e a n            # # . #           # # . #             # # . #           # # . #             # # . #           # # . #              # # . #           # # . #  
     S D              # # . # #          # # . # #            # # . # #          # # . # #            # # . # #          # # . # #             # # . # #          # # . # #  
     S E M             # # . # #          # # . # #            # # . # #          # # . # #            # # . # #          # # . # #             # # . # #          # # . # #  
     M i n i m u m         # #             # #               # #             # #               # #             # #                # #             # #  
     M e d i a n          # # . #           # # . #             # # . #           # # . #             # # . #           # # . #              # # . #           # # . #  
     M a x i m u m         # #             # #               # #             # #               # #             # #                # #             # #   
 
  T h e  l a b o r a t o r y  p a r a m e t e r s  t o  b e  s u m m a r i z e d  i n c l u d e :  
    p H  a n d  s p e c i f i c  g r a v i t y .  
 
  T h e  v i s i t s  t o  b e  s u m m a r i z e d  i n c l u d e :  
     D a y  2 ,  D a y  8 ,  D a y  1 5 ,  D a y  2 9 ,  D a y  4 3 ,  D a y  7 1 ,  D a y  8 5 ,  E n d  o f  T r e a t m e n t ,  D a y  9 9 ,  D a y  1 4 1 ,  D a y  1 6 9 ,  E n d  o f  F o l l o w - u p  P e r i o d .  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
-  B a s e l i n e  i s  d e f i n e d  a s  t h e  l a s t  p r e - d o s e  v a l u e .  
-  N ' = n u m b e r  o f  s u b j e c t s  w i t h  d a t a  a t  b a s e l i n e  a n d  t h e  s p e c i f i e d  v i s i t .  
-  T h e  E n d  o f  T r e a t m e n t  v a l u e  i s  t h e  l a s t  m e a s u r e m e n t  t h r o u g h  D a y  8 5 .  
-  T h e  E n d  o f  F o l l o w - u p  P e r i o d  i s  t h e  l a s t  m e a s u r e m e n t  a f t e r  D a y  8 5  t h r o u g h  D a y  1 6 9 .  
 
P r o g r a m  N a m e :  X X X X X X X X . s a s                   R u n  D a t e :  D D M M M Y Y Y Y  H H : M M                            D a t a b a s e  l a s t  m o d i f i e d :  D D M M Y Y Y Y  H H : M M  
 
 
N O T E: Re peat f or Ste ps 3, 2 a n d 1.  


 
P a ge 1 7 9  of 2 0 8  
 
C h e m o C e n t r y x ,  I n c .  
P r o t o c o l  C L 0 0 2 _ 1 6 8                                                                                                          P a g e  1  o f  #  
T a b l e  1 4 . 3 . 4 . 4  
S h i f t  T a b l e  f o r  C a t e g o r i c a l  C h a n g e s  i n  H e m a t o l o g y  P a r a m e t e r s  b y  V i s i t  
S a f e t y  P o p u l a t i o n  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
S t e p /                                                                                B a s e l i n e          C a t e g o r y  a t  S p e c i f i e d  V i s i t   
  P a r a m e t e r ( N o r m a l  R a n g e )         V i s i t         T r e a t m e n t                              C a t e g o r y        < L L N          N o r m a l        > U L N  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
O v e r a l l         
  P a r a m e t e r  1  ( # # # - # # # )           D a y  2          P l a c e b o + F u l l  D o s e  P r e d n i s o n e  ( N ’ = # #  )   <  L L N       # #  ( # # # . # )    # #  ( # # # . # )    # #  ( # # # . # )  
                                                                                      N o r m a l      # #  ( # # # . # )    # #  ( # # # . # )    # #  ( # # # . # )  
                                                                                      >  U L N       # #  ( # # # . # )    # #  ( # # # . # )    # #  ( # # # . # )  
 
                                               C C X 1 6 8 + L o w  D o s e  P r e d n i s o n e  ( N ’ = # # )  
 
                                               C C X 1 6 8 + N o  P r e d n i s o n e  ( N ’ = # # )  
 
                                               A l l  C C X 1 6 8  ( N ’ = # # )  
 
 
  T h e  l a b o r a t o r y  p a r a m e t e r s  t o  b e  s u m m a r i z e d  w i l l  i n c l u d e :      
    H e m o g l o b i n ,  H e m a t o c r i t ,  R e d  b l o o d  c e l l  c o u n t ,  W h i t e  b l o o d  c e l l  c o u n t  ( w i t h  d i f f e r e n t i a l ) ,  P l a t e l e t  c o u n t ,  M C H ,  M C H C   
    a n d  M C V .  
 
    C h e m i s t r y :  T o t a l  B i l i r u b i n ,  L D H ,  S G O T / A S T ,  S G P T / A L T ,  B U N ,  C r e a t i n i n e ,  C P K ,  A l b u m i n ,  S o d i u m ,  P o t a s s i u m ,  B i c a r b o n a t e ,  C h l o r i d e ,   
               C a l c i u m ,  I n o r g a n i c  p h o s p h o r o u s ,  G l u c o s e ,  T o t a l  p r o t e i n ,  A l k a l i n e  p h o s p h a t a s e ,  C h o l e s t e r o l ,  a n d  U r i c  a c i d .  
 
    U r i n a l y s i s :  p H  a n d  s p e c i f i c  g r a v i t y .  
 
  T h e  v i s i t s  t o  b e  s u m m a r i z e d  i n c l u d e :  
     D a y  2 ,  D a y  8 ,  D a y  1 5 ,  D a y  2 9 ,  D a y  4 3 ,  D a y  7 1 ,  D a y  8 5 ,  E n d  o f  T r e a t m e n t ,  D a y  9 9 ,  D a y  1 4 1 ,  D a y  1 6 9 ,  E n d  o f  F o l l o w - u p  P e r i o d .  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
-  N ’ =  n u m b e r  o f  s u b j e c t s  i n  g r o u p  w i t h  a s s e s s m e n t  o f  p a r a m e t e r  a t  b a s e l i n e  a n d  s p e c i f i e d  v i s i t  
-  n  =  n u m b e r  o f  s u b j e c t s  i n  g r o u p  w i t h  p o s t - b a s e l i n e  m e a s u r e m e n t  o u t s i d e  s p e c i f i e d  l i m i t  
-  %  =  1 0 0 * n / N ’  
-  T h e  E n d  o f  T r e a t m e n t  v a l u e  i s  t h e  l a s t  m e a s u r e m e n t  t h r o u g h  D a y  8 5 .  
-  T h e  E n d  o f  F o l l o w - u p  P e r i o d  i s  t h e  l a s t  m e a s u r e m e n t  a f t e r  D a y  8 5  t h r o u g h  D a y  1 6 9 .  
 
P r o g r a m  N a m e :  X X X X X X X X . s a s                   R u n  D a t e :  D D M M M Y Y Y Y  H H : M M                            D a t a b a s e  l a s t  m o d i f i e d :  D D M M Y Y Y Y  H H : M M  
 
N O T E: Re peat f or Ste ps 3, 2, a n d 1. 


 
P a ge 1 8 0  of 2 0 8  
 
T he f oll o wi n g t a ble w ill ha ve t he sa me l a y o ut as Ta ble 1 4. 3. 4. 4: 
 
T a b l e  1 4 . 3 . 4 . 5  
S h i f t  T a b l e  f o r  C a t e g o r i c a l  C h a n g e s  i n  C h e m i s t r y  P a r a m e t e r s  b y  V i s i t  
S a f e t y  P o p u l a t i o n  
 
T he para meters t o be s u m marize d i n Ta ble 1 4. 3. 4. 5 i ncl u de: T otal Bilir u bi n, L D H, S G O T/ A S T, S G P T/ A L T, B U N, Creati ni ne, C P K, 
Al b u mi n, S o di u m, P otassi u m, Bicar b o nate, C hl ori de, Calci u m, I n or ga nic p h os p h or o us, Gl uc ose, T otal pr otei n, Al k ali ne p h os p hatase, 
C h olester ol, a n d Uric aci d. 


 
P a ge 1 8 1  of 2 0 8  
 
C h e m o C e n t r y x ,  I n c .  
P r o t o c o l  C L 0 0 2 _ 1 6 8                                                                                                          P a g e  1  o f  #  
T a b l e  1 4 . 3 . 4 . 7  
S h i f t  T a b l e  f o r  C a t e g o r i c a l  U r i n a l y s i s  P a r a m e t e r s  b y  V i s i t  
S a f e t y  P o p u l a t i o n  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
S t e p                                      P o s t -                  P l a c e b o  +                C C X 1 6 8 +             C C X 1 6 8 +   
  P a r a m e t e r             B a s e l i n e           B a s e l i n e           F u l l  D o s e  P r e d n i s o n e    L o w  D o s e  P r e d n i s o n e    N o  P r e d n i s o n e        A l l  C C X 1 6 8  
   V i s i t                V a l u e              V a l u e                    n   ( % )                 n   ( % )              n   ( % )              n   ( % )  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
O v e r a l l                                                           N = # #                   N = # #                N = # #                N = # #  
 
  P a r a m e t e r  1                                                     N ’ = # #               N ’ = # #               N ’ = # #               N ’ = # #  
   D a y  2               C a t e g o r y  1         C a t e g o r y  1   
                                        C a t e g o r y  2  
                                        C a t e g o r y  n  
 
 
 T h e  u r i n a l y s i s  p a r a m e t e r s  i n c l u d e :  
 G l u c o s e ,  N i t r i t e ,  K e t o n e s ,  B i l i r u b i n ,  B l o o d  U r o b i l i n o g e n ,  R B C ,  a n d  W B C   
 
T h e  v i s i t s  t o  b e  s u m m a r i z e d  i n c l u d e :  
     D a y  2 ,  D a y  8 ,  D a y  1 5 ,  D a y  2 2 ,  D a y  2 9 ,  D a y  4 3 ,  D a y  5 7 ,  D a y  7 1 ,  D a y  8 5 ,  E n d  o f  T r e a t m e n t ,  D a y  9 9 ,  D a y  1 1 3 ,  D a y  1 4 1 ,  D a y  1 6 9 ,  E n d  o f   
     F o l l o w - u p  P e r i o d .  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
-  N ’ =  n u m b e r  o f  s u b j e c t s  i n  g r o u p  w i t h  b a s e l i n e  a n d  p o s t - b a s e l i n e  m e a s u r e m e n t s  o f  s p e c i f i e d  p a r a m e t e r  
-  n  =  n u m b e r  o f  s u b j e c t s  i n  g r o u p  w i t h  p o s t - b a s e l i n e  m e a s u r e m e n t  o u t s i d e  s p e c i f i e d  l i m i t  
-  %  =  1 0 0 * n / N ’  
-  T h e  E n d  o f  T r e a t m e n t  v a l u e  i s  t h e  l a s t  m e a s u r e m e n t  t h r o u g h  D a y  8 5 .  
-  T h e  E n d  o f  F o l l o w - u p  P e r i o d  i s  t h e  l a s t  m e a s u r e m e n t  a f t e r  D a y  8 5  t h r o u g h  D a y  1 6 9 .  
 
P r o g r a m  N a m e :  X X X X X X X X . s a s                   R u n  D a t e :  D D M M M Y Y Y Y  H H : M M                            D a t a b a s e  l a s t  m o d i f i e d :  D D M M Y Y Y Y  H H : M M  
 
 
 
N O T E: R e peat f or Ste ps 3, 2 a n d 1.  


 
P a ge 1 8 2  of 2 0 8  
 
C h e m o C e n t r y x ,  I n c .  
P r o t o c o l  C L 0 0 2 _ 1 6 8                                                                                                          P a g e  1  o f  #  
T a b l e  1 4 . 3 . 4 . 8  
L i s t i n g  o f  L a b o r a t o r y  D a t a  f o r  S u b j e c t s  w i t h  N o t a b l e  L a b o r a t o r y  A b n o r m a l i t i e s  
S a f e t y  P o p u l a t i o n  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
S t e p  
  T r e a t m e n t                                        N o r m a l                                                                C l i n i c a l  
     S u b j e c t     P a r a m e t e r  ( U n i t )                   R a n g e      V i s i t  ( S t u d y  D a y )           V a l u e                          S i g n i f i c a n c e  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
S t e p  1  
  X X X X X X X X X X X X X X X X X X X X X X  
    # # # # - # # # #   X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X   X X X X - X X X X   X X X X X X X X X X X X X X  ( # # )        # # # # # # # # # # # #                     N O T A B L E  H I G H  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
P r o g r a m  N a m e :  X X X X X X X X . s a s                   R u n  D a t e :  D D M M M Y Y Y Y  H H : M M                                  D a t a b a s e  l a s t  m o d i f i e d :  D D M M Y Y Y Y  
 
 
 
N O T E: T he ta ble will i ncl u de all rec or ds f or eac h para meter t hat has at least o ne occ urre nce of a N O T A B L E or C RI TI C A L 
a b n or malit y as i de ntifie d b y t he ce ntral la b orat or y.  T his ta ble will be s orte d b y Ste p, treat me nt gr o u p a n d s u bject wit hi n treat me nt 
gr o u p.   


 
P a ge 1 8 3  of 2 0 8  
 
C h e m o C e n t r y x ,  I n c .  
P r o t o c o l  C L 0 0 2 _ 1 6 8                                                                                                          P a g e  1  o f  #  
T a b l e  1 4 . 3 . 5 . 1  
S u m m a r y  o f  C h a n g e s  i n  V i t a l  S i g n s ,  W e i g h t ,  a n d  B M I  b y  V i s i t  
S a f e t y  P o p u l a t i o n  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
S t e p  
  P a r a m e t e r               P l a c e b o  +                       C C X 1 6 8  +                        C C X 1 6 8  +   
   S t u d y  D a y     - -  F u l l  D o s e  P r e d n i s o n e  - -      - -  L o w  D o s e  P r e d n i s o n e  - -       - - - - -  N o  P r e d n i s o n e  - - - - -       - - - - - - -  A l l  C C X 1 6 8  - - - - - -  
    S t a t i s t i c        V i s i t         C h a n g e            V i s i t         C h a n g e            V i s i t         C h a n g e             V i s i t         C h a n g e  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
O v e r a l l                    N = # #                           N = # #                           N = # #                            N = # #  
  P a r a m e t e r  1  ( U n i t )   
   B a s e l i n e  
     N ’              # #                             # #                             # #                              # #  
     M e a n            # # . #                           # # . #                           # # . #                            # # . #  
     S D              # # . # #                          # # . # #                          # # . # #                           # # . # #  
     M i n i m u m         # #                             # #                             # #                              # #  
     M e d i a n          # # . #                           # # . #                           # # . #                            # # . #  
     M a x i m u m         # #                             # #                             # #                              # #   
 
   D a y  2  
     N ’              # #             # #               # #             # #               # #             # #                # #             # #  
     M e a n            # # . #           # # . #             # # . #           # # . #             # # . #           # # . #              # # . #           # # . #  
     S D              # # . # #          # # . # #            # # . # #          # # . # #            # # . # #          # # . # #             # # . # #          # # . # #  
     M i n i m u m         # #             # #               # #             # #               # #             # #                # #             # #  
     M e d i a n          # # . #           # # . #             # # . #           # # . #             # # . #           # # . #              # # . #           # # . #  
     M a x i m u m         # #             # #               # #             # #               # #             # #                # #             # #   
 
 
  T h e  v i t a l  s i g n  p a r a m e t e r s  t o  b e  s u m m a r i z e d  i n c l u d e :      
    S y s t o l i c  b l o o d  p r e s s u r e ,  D i a s t o l i c  b l o o d  p r e s s u r e ,  H e a r t  r a t e ,  O r a l  t e m p e r a t u r e ,  W e i g h t ,  B M I .  
 
 
  T h e  v i s i t s  t o  b e  s u m m a r i z e d  i n c l u d e :  
     D a y  2 ,  D a y  8 ,  D a y  1 5 ,  D a y  2 2 ,  D a y  2 9 ,  D a y  4 3 ,  D a y  5 7 ,  D a y  7 1 ,  D a y  8 5 ,  E n d  o f  T r e a t m e n t ,  D a y  9 9 ,  D a y  1 1 3 ,  D a y  1 4 1 ,  D a y  1 6 9 ,  
     E n d  o f  F o l l o w - u p  P e r i o d  f o r  s y s t o l i c  b l o o d  p r e s s u r e ,  d i a s t o l i c  b l o o d  p r e s s u r e ,  h e a r t  r a t e ,  a n d  o r a l  t e m p e r a t u r e ,  a n d  D a y  1 5 ,   
     D a y  2 9 ,  D a y  5 7 ,  D a y  8 5 ,  E n d  o f  T r e a t m e n t ,  D a y  1 1 3 ,  D a y  1 6 9 ,  a n d  E n d  o f  F o l l o w - u p  P e r i o d  f o r  W e i g h t  a n d  B M I .  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
-  B a s e l i n e  i s  d e f i n e d  a s  t h e  l a s t  p r e - d o s e  v a l u e .  
-  N ' = n u m b e r  o f  s u b j e c t s  w i t h  d a t a  a t  b a s e l i n e  a n d  t h e  s p e c i f i e d  v i s i t .  
-  T h e  E n d  o f  T r e a t m e n t  v a l u e  i s  t h e  l a s t  m e a s u r e m e n t  t h r o u g h  D a y  8 5 .  
-  T h e  E n d  o f  F o l l o w - u p  P e r i o d  i s  t h e  l a s t  m e a s u r e m e n t  a f t e r  D a y  8 5  t h r o u g h  D a y  1 6 9 .  
 
P r o g r a m  N a m e :  X X X X X X X X . s a s                   R u n  D a t e :  D D M M M Y Y Y Y  H H : M M                            D a t a b a s e  l a s t  m o d i f i e d :  D D M M Y Y Y Y  H H : M M  
 
N O T E: Re peat f or Ste ps 3, 2 , a n d 1.  


 
P a ge 1 8 4  of 2 0 8  
 
C h e m o C e n t r y x ,  I n c .  
P r o t o c o l  C L 0 0 2 _ 1 6 8                                                                                                          P a g e  1  o f  #  
T a b l e  1 4 . 3 . 5 . 2  
S u m m a r y  o f  P h y s i c a l  E x a m i n a t i o n  a n d  B o d y  S y s t e m  R e v i e w s  b y  V i s i t  
S a f e t y  P o p u l a t i o n  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
                                                                  P l a c e b o +            C C X 1 6 8 +  
 S t e p                                                             F u l l  D o s e           L o w  D o s e            C C X 1 6 8 +      
   D a y                                                           P r e d n i s o n e          P r e d n i s o n e        N o  P r e d n i s o n e       A l l  C C X 1 6 8  
    B o d y  S y s t e m              R e s u l t                              N ’    n      ( % )      N ’     n    ( % )      N ’     n    ( % )      N ’    n      ( % )  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
O v e r a l l                                                            N = # #                N = # #                N = # #               N = # #  
   D a y  1         
     G e n e r a l  A p p e a r a n c e       N o r m a l / s a m e  a s  p r e v i o u s  P E         # #   # #   ( # # # . # )     # #   # #   ( # # # . # )    # #    # #   ( # # # . # )   # #    # #   ( # # # . # )  
                             A b n o r m a l ,  n e w  s i n c e  p r e v i o u s  P E    # #   # #   ( # # # . # )     # #   # #   ( # # # . # )    # #    # #   ( # # # . # )   # #    # #   ( # # # . # )  
 
     H E E N T                    N o r m a l / s a m e  a s  p r e v i o u s  P E         # #   # #   ( # # # . # )     # #   # #   ( # # # . # )    # #    # #   ( # # # . # )   # #    # #   ( # # # . # )  
                             A b n o r m a l ,  n e w  s i n c e  p r e v i o u s  P E    # #   # #   ( # # # . # )     # #   # #   ( # # # . # )    # #    # #   ( # # # . # )   # #    # #   ( # # # . # )  
 
 
 
  T h e  b o d y  s y s t e m s  t o  b e  s u m m a r i z e d  i n c l u d e :      
    G e n e r a l  A p p e a r a n c e / M e n t a l  S t a t u s ,  H E E N T ,  D e r m a t o l o g i c ,  C a r d i o v a s c u l a r ,  R e s p i r a t o r y ,  G a s t r o i n t e s t i n a l ,  M u s c u l o s k e l a t a l ,  a n d   
    D e r m a t o l o g i c .  
 
  T h e  v i s i t s  t o  b e  s u m m a r i z e d  i n c l u d e :  
     D a y  1 ,  D a y  2 ,  D a y  8 ,  D a y  1 5 ,  D a y  2 2 ,  D a y  2 9 ,  D a y  4 3 ,  D a y  5 7 ,  D a y  7 1 ,  D a y  8 5 ,  D a y  9 9 ,  D a y  1 1 3 ,  D a y  1 4 1 ,  a n d  D a y  1 6 9 .  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
-  N ' = n u m b e r  o f  s u b j e c t s  w i t h  b o d y  s y s t e m  r e v i e w e d  a t  t h e  s p e c i f i e d  v i s i t .  
 
P r o g r a m  N a m e :  X X X X X X X X . s a s                   R u n  D a t e :  D D M M M Y Y Y Y  H H : M M                            D a t a b a s e  l a s t  m o d i f i e d :  D D M M Y Y Y Y  H H : M M  
 
 
 
N O T E: Re peat f or Ste ps 3, 2, a n d 1.  


 
P a ge 1 8 5  of 2 0 8  
 
C h e m o C e n t r y x ,  I n c .  
P r o t o c o l  C L 0 0 2 _ 1 6 8                                                                                                          P a g e  1  o f  #  
T a b l e  1 4 . 3 . 5 . 3  
L i s t i n g  o f  E C G  A b n o r m a l i t i e s  
S a f e t y  P o p u l a t i o n  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
S t e p    
  T r e a t m e n t                                                                                                                C l i n i c a l l y  
    S u b j e c t     A g e / S e x / R a c e                       D a t e / D a y          A b n o r m a l i t y                                             S i g n i f i c a n t ?  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
  X X X X X X X X X X X X X X X X X X X X X X  
    # # # # - # # # #   # # / M / X X X X X X X X X X X X X X X X X X X X X X X X      D D M M M Y Y Y Y / X X      X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X            Y e s   
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
P r o g r a m  N a m e :  X X X X X X X X . s a s                   R u n  D a t e :  D D M M M Y Y Y Y  H H : M M                                  D a t a b a s e  l a s t  m o d i f i e d :  D D M M Y Y Y Y  
 
 
 
N O T E: T his ta ble will be s orte d b y Ste p, treat me nt gr o u p a n d s u bject wit hi n treat me nt gr o u p.   
 
 


C he m oce ntr y x, I nc.  
Pr ot oc ol C L 0 0 2 1 6 8 
 
Statistical A nal ysis Pla n- Versi o n 2. 0 
 
P a ge 1 8 6  of 2 0 8  
 
8  
Listi n g S hells 
Listi n g D 1  ................................................................................................................................... 1 8 8 
S u bject Treat me nt I nf or mati o n, De m o gra p hics, a n d Baseli ne C haracteristics  
All Ra n d o mize d S u bjects  
Listi n g D 2 ................................................................................................................................... 1 8 9  
St u d y M e dica ti o n I nf or m ati o n 
All Ra n d o mize d S u bjects  
Listi n g D 3. 1 ................................................................................................................................ 1 9 0  
C ycl o p h os p ha mi de I V D osi n g  
All Ra n d o mize d S u bjects  
Listi n g D 3. 2 ................................................................................................................................ 1 9 1  
Rit u xi ma b D osi n g ( Ste p 3)  
All Ra n d o mize d S u bjects  
Listi n g D 4 ................................................................................................................................... 1 9 2  
C o nc o mita nt Me dicati o ns  
All Ra n d o mize d S u bjects  
Listi n g D 5 ................................................................................................................................... 1 9 3  
A d verse E v e nts  
All Ra n d o mize d S u bjects  
Listi n g D 6 ................................................................................................................................... 1 9 4  
Ce ntral La b orat or y Data - C he mistr y  
All Ra n d o mize d S u bjects  
Listi n g D 7 ................................................................................................................................... 1 9 5  
Ce ntral La b orat or y Data - He mat ol o g y  
All Ra n d o mize d S u bjects  
Listi n g D 8 ................................................................................................................................... 1 9 5  
C e ntral La b orat or y Data - Uri nal ys is 
All Ra n d o mize d S u bjects  
Listi n g D 9 ................................................................................................................................... 1 9 5  
Ce ntral La b orat or y Data – Uri ne C he mistr y  
All Ra n d o mize d S u bjects  
Listi n g D 1 0. 1 .............................................................................................................................. 1 9 5  
L ocal La b orat or y D ata – Ser u m C he mistr y, He m at ol o g y, a n d C oa g ulati o n  
All Ra n d o mize d S u bjects  
Listi n g D 1 0. 2 .............................................................................................................................. 1 9 5  
L ocal La b orat or y D ata – Vir ol o g y, I m m u n ol o g y, a n d C hest X-ra y Res ults  
All Ra n d o mize d S u bjects  
Listi n g D 1 1 ................................................................................................................................. 1 9 6  
L ocal La b orat or y D ata - Uri nal ysis  
All Ra n d o mize d S u bjects  
Listi n g D 1 2 ................................................................................................................................. 1 9 7  
Bir mi n g h a m Va sc ulitis Acti vit y Sc ore ( B V A S) 
All Ra n d o mize d S u bjects  
 
 


C he m oce ntr y x, I nc.  
Pr ot oc ol C L 0 0 2 1 6 8 
 
Statistical A nal ysis Pla n- Versi o n 2. 0 
 
P a ge 1 8 7  of 2 0 8  
 
Listi n g D 1 3 ................................................................................................................................. 1 9 8 
Vasc ulitis Da ma ge I n d e x  
All Ra n d o mize d S u bjects  
Listi n g D 1 4 ................................................................................................................................. 1 9 9  
A N C A Res ults  
All Ra n d o mize d S u bjects  
Listi n g D 1 5 ................................................................................................................................. 2 0 0  
Ser u m hs C R P R es ults ( m g/ L)  
All Ra n d o mize d S u bjects  
Listi n g D 1 6 ................................................................................................................................. 2 0 1  
Ser u m Uri nar y  M C P- 1: Creati ni ne Rati o Res ults ( p g/ m g Creati ni ne) 
All Ra n d o mize d S u bjects  
Listi n g D 1 7 ................................................................................................................................. 2 0 2  
Esti mate d Gl o mer ular F iltrati o n Rate ( M D R D) Res ults ( m L/ mi n/ 1. 7 3 m ^ 2) 
All Ra n d o mize d S u bjects  
C he m o Ce ntr y x, I n c.  
Listi n g D 1 8 ................................................................................................................................. 2 0 3  
A C R Res ults ( m g/ g ) 
All Ra n d o mize d S u bjects  
Listi n g D 1 9 ................................................................................................................................. 2 0 4  
Vital Si g ns, Wei g ht, a n d B MI  
All Ra n d o mize d S u bjects  
Listi n g D 2 0 ................................................................................................................................. 2 0 5  
P h ysical E x a m a n d B o d y S yste m Re vie ws  
All Ra n d o mize d S u bjects  
Listi n g D 2 1 ................................................................................................................................. 2 0 6  
S F - 3 6 v 2 Healt h S ur vey  
All Ra n d o mize d S u bjects i n Ste p 3  
Listi n g D 2 2 ................................................................................................................................. 2 0 8  
E Q - 5 D- 5 L Q uesti o n naire a n d V A S  
All Ra n d o mize d S u bjects i n Ste p 3  
 
 


 
P a ge 1 8 8  of 2 0 8  
 
C h e m o C e n t r y x ,  I n c .  
P r o t o c o l  C L 0 0 2 _ 1 6 8                                                                                                          P a g e  1  o f  #  
L i s t i n g  D 1  
S u b j e c t  T r e a t m e n t  I n f o r m a t i o n ,  D e m o g r a p h i c s ,  a n d  B a s e l i n e  C h a r a c t e r i s t i c s  
A l l  R a n d o m i z e d  S u b j e c t s  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
  S U B J E C T :  # # # - # # #  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
  T R E A T M E N T  I N F O R M A T I O N  
 
    T r e a t m e n t :  C C X 1 6 8             
    D a t e  o f  F i r s t  D o s e :  D D M M M Y Y Y Y          
    D a t e  o f  L a s t  D o s e  ( D a y ) :  O n g o i n g     
    D a t e  o f  C o m p l e t i o n / E a r l y  T e r m i n a t i o n  ( D a y ) :  O n g o i n g  
    D i d  t h e  S u b j e c t  C o m p l e t e  t h e  S t u d y ? :  N o  
    P r i m a r y  R e a s o n  f o r  W i t h d r a w a l :  X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X  
- - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - -  
  B A S E L I N E  I N F O R M A T I O N  
   
    A g e / S e x / E t h n i c i t y / R a c e :     # # / F E M A L E / N o t  H i s p a n i c  o r  L a t i n o / X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X   
    P r o t o c o l  v e r s i o n :  A m e n d m e n t  #  
    B o d y  w e i g h t  ( k g ) :  # # . #  
    B M I  ( k g / m ^ 2 ) :  # # . #  
    S m o k i n g  s t a t u s :  X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X  
    A A V  S t a t u s :  ( S p e c i f y  a s  n e w l y  d i a g n o s e d  o r  r e l a p s e d . )  
    D a t e  o f  A A V  D i a g n o s i s :  D D M M M Y Y Y Y  
    D u r a t i o n  o f  A A V  d i s e a s e :  # # . #  
    T y p e  o f  A A V :  ( S p e c i f y  a s  G r a n u l o m a t o s i s  w i t h  p o l y a n g i i t i s  ( W e g e n e r ' s ) ,  M i c r o s c o p i c  p o l y a n g i i t i s ,  o r  R e n a l  l i m i t e d  v a s c u l i t i s )  
    I I F  T e s t  f o r  P - A N C A :  ( S p e c i f y  a s  p o s i t i v e  o r  n e g a t i v e . )  
    I I F  T e s t  f o r  C - A N C A :  ( S p e c i f y  a s  p o s i t i v e  o r  n e g a t i v e . )  
    E L I S A  T e s t  f o r  A n t i - p r o t e i n a s e - 3 :  ( S p e c i f y  a s  p o s i t i v e  o r  n e g a t i v e . )  
    E L I S A  T e s t  f o r  A n t i - m y e l o p e r o x i d a s e :  ( S p e c i f y  a s  p o s i t i v e  o r  n e g a t i v e . )  
    B a s e l i n e  B i r m i n g h a m  V a s c u l i t i s  A c t i v i t y  S c o r e  ( B V A S ) :  # #  
    B a s e l i n e  V a s c u l i t i s  D a m a g e  I n d e x  S c o r e  ( V D I ) :  # #  
    W a s  r e n a l  b i o p s y  d o n e  t o  c o n f i r m  d i a g n o s i s  o f  r e n a l  v a s c u l i t i s ?  X X X  [ I f  y e s ,  d e s c r i b e  a b n o r m a l i t i e s . ]  
    B a s e l i n e  E s t i m a t e d  G l o m e r u l a r  F i l t r a t i o n  R a t e  ( M D R D )  R e s u l t s  ( m L / m i n / 1 . 7 3  m ^ 2 ) :  # # . #  
    B a s e l i n e  A C R  ( m g / g ) :  # # . #  
    B a s e l i n e  U r i n e  R e d  B l o o d  C e l l  C o u n t  ( / h p f ) :  # #  
    B a s e l i n e  M C P - 1 : C r e a t i n i n e  R a t i o ( p g / m g  C r e a t i n i n e ) :  # #  
- - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - -  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
P r o g r a m  N a m e :  X X X X X X X X . s a s                   R u n  D a t e :  D D M M M Y Y Y Y  H H : M M                            D a t a b a s e  l a s t  m o d i f i e d :  D D M M Y Y Y Y  H H : M M  


 
P a ge 1 8 9  of 2 0 8  
 
C h e m o C e n t r y x ,  I n c .  
P r o t o c o l  C L 0 0 2 _ 1 6 8                                                                                                          P a g e  1  o f  #  
L i s t i n g  D 2  
S t u d y  M e d i c a t i o n  I n f o r m a t i o n  
A l l  R a n d o m i z e d  S u b j e c t s  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
 S t e p  
   T r e a t m e n t                                   - - - - - - - - - - -  C C X 1 6 8 / P l a c e b o  - - - - - - - - - -         - - - - - - - -  P r e d n i s o n e / P l a c e b o  - - - - - - - - -  
     S u b j e c t       V i s i t        D a t e              C a p s u l e s  D i s p e n s e d   C a p s u l e s  R e t u r n e d         C a p s u l e s  D i s p e n s e d   C a p s u l e s  R e t u r n e d  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
S t e p  1  
  X X X X X X X X X X X X X X X X X X X X X X X X X X X X X   
      # # # - # # #     D a y  1      D D M M M Y Y Y Y                  # #                   # #                        # #                   # #  
                 D a y  8      D D M M M Y Y Y Y                  # #                   # #                        # #                   # #  
                 D a y  1 5     D D M M M Y Y Y Y                  # #                   # #                        # #                   # #  
 
                 D u r a t i o n  o f  D o s i n g  ( d a y s ) :                   # # #                                          # # #  
                 O v e r a l l  P e r c e n t  C o m p l i a n c e :                   # # # . #                                        # # # . #  
                 T o t a l  C C X 1 6 8  o r  P r e d n i s o n e  D o s e :             # # #                                          # # #  
- - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - -  
S t e p  2  
  X X X X X X X X X X X X X X X X X X X X X X X X X X X X X   
      # # # - # # #     D a y  1      D D M M M Y Y Y Y                  # #                   # #                        # #                   # #  
                 D a y  8      D D M M M Y Y Y Y                  # #                   # #                        # #                   # #  
                 D a y  1 5     D D M M M Y Y Y Y                  # #                   # #                        # #                   # #  
 
                 D u r a t i o n  o f  D o s i n g  ( d a y s ) :                   # # #                                          # # #  
                 O v e r a l l  P e r c e n t  C o m p l i a n c e :                   # # # . #                                        # # # . #  
                 T o t a l  C C X 1 6 8  o r  P r e d n i s o n e  D o s e :             # # #                                          # # #  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
P r o g r a m  N a m e :  X X X X X X X X . s a s                   R u n  D a t e :  D D M M M Y Y Y Y  H H : M M                            D a t a b a s e  l a s t  m o d i f i e d :  D D M M Y Y Y Y  H H : M M  
 
 
 
N ote t o pr o gra m mer:  F or t he calc ul ati o n of pre d nis o ne d ose, m ost s u bjects use d b ottles c o ntai ni n g 2 0 m g pre d nis o ne f or t he first 
wee k a n d t h e n b ottles c o ntai ni n g 5 m g pre d nis o ne after wee k 1 (refer t o pr ot oc ol secti o n 1 1. 6 f or details). 


 
P a ge 1 9 0  of 2 0 8  
 
C h e m o C e n t r y x ,  I n c .  
P r o t o c o l  C L 0 0 2 _ 1 6 8                                                                                                          P a g e  1  o f  #  
L i s t i n g  D 3 . 1  
C y c l o p h o s p h a m i d e  I V  D o s i n g  
A l l  R a n d o m i z e d  S u b j e c t s  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
 S t e p                                   W a s  C y c l o p h o s p h a m i d e  
   T r e a t m e n t                                A d m i n i s t e r e d                   D o s e  A m i n i s t e r e d   
T o t a l  D o s e  A m i n i s t e r e d  
     S u b j e c t       V i s i t        D a t e         A t  T h i s  V i s i t ?                      ( m g / k g )   
  
 
( m g )  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
S t e p  1  
  X X X X X X X X X X X X X X X X X X X X X X X X X X X X X   
      # # # - # # #     D a y  1      D D M M M Y Y Y Y             X X X                             # # #  
                 D a y  1 5     D D M M M Y Y Y Y             X X X                             # # #                        # # #  
                 D a y  2 9     D D M M M Y Y Y Y             X X X                             # # #                        # # #  
                 D a y  5 7     D D M M M Y Y Y Y             X X X                             # # #                        # # #  
                 D a y  8 5     D D M M M Y Y Y Y             X X X                             # # #                        # # #  
 
                 T o t a l  D a y s  o f  D o s i n g  D u r i n g  8 4 - D a y  T r e a t m e n t  P e r i o d :                                     # # #  
                 C u m u l a t i v e  D o s e  8 4  D a y  T r e a t m e n t  P e r i o d :                                                 # # #  
 
                 D a y  1 1 3    D D M M M Y Y Y Y             X X X                             # # #                        # # #  
                 D a y  1 4 1    D D M M M Y Y Y Y             X X X                             # # #                        # # #  
                 D a y  1 6 9    D D M M M Y Y Y Y             X X X                             # # #                        # # #  
            
                 T o t a l  D a y s  o f  D o s i n g  D u r i n g  1 6 8 - D a y  T r e a t m e n t  P e r i o d :                                    # # #  
                 C u m u l a t i v e  D o s e  1 6 8  D a y  T r e a t m e n t  P e r i o d :                                                # # #  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
P r o g r a m  N a m e :  X X X X X X X X . s a s                   R u n  D a t e :  D D M M M Y Y Y Y  H H : M M                            D a t a b a s e  l a s t  m o d i f i e d :  D D M M Y Y Y Y  H H : M M  


 
P a ge 1 9 1  of 2 0 8  
 
C h e m o C e n t r y x ,  I n c .  
P r o t o c o l  C L 0 0 2 _ 1 6 8                                                                                                          P a g e  1  o f  #  
L i s t i n g  D 3 . 2  
R i t u x i m a b  D o s i n g  ( S t e p  3 )  
A l l  R a n d o m i z e d  S u b j e c t s  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
                                          W a s  R i t u x i m a b                                            T o t a l  D o s e  
   T r e a t m e n t                                A d m i n i s t e r e d                                           A m i n i s t e r e d     R e a s o n  
     S u b j e c t       V i s i t        D a t e         A t  T h i s  V i s i t ?     S t a r t  D a t e / T i m e     S t o p  D a t e / T i m e         ( m g )        N o t  D o n e  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
  X X X X X X X X X X X X X X X X X X X X X X X X X X X X X   
      # # # - # # #     D a y  1      D D M M M Y Y Y Y             X X X          D D M M M Y Y Y Y / H H : M M    D D M M M Y Y Y Y / H H : M M          # # #        X X X X X X X X X X X X X X X X X X X X    
                 D a y  1 5     D D M M M Y Y Y Y             X X X          D D M M M Y Y Y Y / H H : M M    D D M M M Y Y Y Y / H H : M M          # # #        X X X X X X X X X X X X X X X X X X X X  
                 D a y  2 2     D D M M M Y Y Y Y             X X X          D D M M M Y Y Y Y / H H : M M    D D M M M Y Y Y Y / H H : M M          # # #        X X X X X X X X X X X X X X X X X X X X  
                 U N S        D D M M M Y Y Y Y             X X X          D D M M M Y Y Y Y / H H : M M    D D M M M Y Y Y Y / H H : M M          # # #        X X X X X X X X X X X X X X X X X X X X  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
P r o g r a m  N a m e :  X X X X X X X X . s a s                   R u n  D a t e :  D D M M M Y Y Y Y  H H : M M                            D a t a b a s e  l a s t  m o d i f i e d :  D D M M Y Y Y Y  H H : M M  


 
P a ge 1 9 2  of 2 0 8  
 
C h e m o C e n t r y x ,  I n c .  
P r o t o c o l  C L 0 0 2 _ 1 6 8                                                                                                          P a g e  1  o f  #  
L i s t i n g  D 4  
C o n c o m i t a n t  M e d i c a t i o n s  
A l l  R a n d o m i z e d  S u b j e c t s  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
S t e p             R T :  R e p o r t e d  T e r m  
  T r e a t m e n t     A T C :  A n a t o m i c  T h e r a p e u t i c  C l a s s            S t a r t  D a t e  ( D a y ) /            
    S u b j e c t      P T :  P r e f e r r e d  T e r m                        E n d  D a t e  ( D a y )           D o s e / U n i t            R o u t e            F r e q u e n c y  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
 S t e p  1  
  X X X X X X X X X X X X X X X X X X X X X X X X X X X X X   
    # # # - # # #      R T : X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X    D D M M M Y Y Y Y  ( # # # ) /     X X X X X X X X X X X X X X X X X X    X X X X X X X X X X X X    X X X X X X X X X X X X X X X X X  
                O C : X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X    D D M M M Y Y Y Y  ( # # # )  
                P T : X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
P r o g r a m  N a m e :  X X X X X X X X . s a s                   R u n  D a t e :  D D M M M Y Y Y Y  H H : M M                            D a t a b a s e  l a s t  m o d i f i e d :  D D M M Y Y Y Y  H H : M M  


 
P a ge 1 9 3  of 2 0 8  
 
C h e m o C e n t r y x ,  I n c .  
P r o t o c o l  C L 0 0 2 _ 1 6 8                                                                                                          P a g e  1  o f  #  
L i s t i n g  D 5  
A d v e r s e  E v e n t s  
A l l  R a n d o m i z e d  S u b j e c t s  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
                                                                                          
 S t e p                 R T :  R e p o r t e d  T e r m                                                                           - - - - - - - -  S A E  - - - - - - - -  
  T r e a t m e n t           O C :  P r i m a r y  S y s t e m  O r g a n  C l a s s       S t a r t  D a t e  ( D a y ) /           R e l a t e d -  A c t i o n    O u t -       I m p r o v e /      R e a p p e a r /  
    S u b j e c t    T E A E ?   P T :  P r e f e r r e d  T e r m                   E n d  D a t e  ( D a y )    S e v e r i t y   n e s s [ 1 ]   T a k e n [ 2 ]  c o m e [ 3 , 4 ]  D i s a p p e a r [ 5 ]  W o r s e n [ 6 ]  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
 S t e p  1  
  X X X X X X X X X X X X X X X X X X X X X X X X X X X X X   
    # # # - # # #     X X X    R T : X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X    D D M M M Y Y Y Y  ( # # # ) /   X X X X X X X X   # / # / #          #        # / #      X X           X X  
                     O C : X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X    D D M M M Y Y Y Y  ( # # # )  
                     P T : X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
-  A n  a d v e r s e  e v e n t  i s  c o n s i d e r e d  t r e a t m e n t - e m e r g e n t  i f  t h e  s t a r t  d a t e  o f  t h e  e v e n t  i s  o n  o r  a f t e r  a d m i n i s t r a t i o n  o f  t h e  f i r s t  d o s e  
  o f  s t u d y  m e d i c a t i o n .  ‘ D a y ’  i s  t h e  n u m b e r  o f  d a y s  f r o m  t h e  d a t e  o f  r a n d o m i z a t i o n .   
[ 1 ]  R e l a t e d n e s s : ( S t u d y  M e d i c a t i o n / C o r t i c o s t e r o i d / C y c l o p h o s p h a m i d e )  0 = P r o b a b l y  N o t  R e l a t e d ,  1 = P o s s i b l y  R e l a t e d  
[ 2 ]  A c t i o n s  T a k e n :  1 = N o n e ,  2 = S t u d y  m e d i c a t i o n  d i s c o n t i n u e d ,  3 = S t u d y  m e d i c a t i o n  i n t e r r u p t e d  
[ 3 ]  O u t c o m e :  1 = R e s o l v e d ,  2 = R e s o l v e d  w i t h  s e q u e l a e ,  3 = O n g o i n g ,  4 = D e a t h ,  5 = U n k n o w n   
[ 4 ]  S e r i o u s  O u t c o m e :  1 = R e s u l t s  i n  d e a t h ,  2 = L i f e  t h r e a t e n i n g ,  3 = I n p a t i e n t  h o s p i t a l i z a t i o n  o r  p r o l o n g a t i o n  o f  e x i s t i n g  h o s p i t a l i z a t i o n ,  
                     4 = P e r s i s t e n t  o r  s i g n i f i c a n t  d i s a b i l i t y / i n c a p a c i t y ,  5 = C o n g e n i t a l  a b n o r m a l i t y  o r  b i r t h  d e f e c t ,  
                     6 = I m p o r t a n t  m e d i c a l  e v e n t   
[ 5 ]  D i d  t h e  e v e n t  i m p r o v e  o r  d i s a p p e a r  a f t e r  s t o p p i n g  s t u d y  m e d i c a t i o n  ( d e c h a l l e n g e ) ?  
[ 6 ]  D i d  t h e  e v e n t  r e a p p e a r  o r  w o r s e n  a f t e r  r e s t a r t i n g  s t u d y  m e d i c a t i o n  ( r e c h a l l e n g e ) ?  
 
P r o g r a m  N a m e :  X X X X X X X X . s a s                   R u n  D a t e :  D D M M M Y Y Y Y  H H : M M                            D a t a b a s e  l a s t  m o d i f i e d :  D D M M Y Y Y Y  H H : M M  


 
P a ge 1 9 4  of 2 0 8  
 
C h e m o C e n t r y x ,  I n c .  
P r o t o c o l  C L 0 0 2 _ 1 6 8                                                                                                          P a g e  1  o f  #  
L i s t i n g  D 6  
C e n t r a l  L a b o r a t o r y  D a t a  -  C h e m i s t r y  
A l l  R a n d o m i z e d  S u b j e c t s  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
S t e p    
  T r e a t m e n t                                        N o r m a l                                                                A b n o r m a l  
     S u b j e c t     P a r a m e t e r  ( U n i t )                   R a n g e      V i s i t                       V a l u e                             F l a g  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
S t e p  1    
  X X X X X X X X X X X X X X X X X X X X X X  
    # # # # - # # # #   X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X   X X X X - X X X X   X X X X X X X X X X X X X X           # # # # # # # # # # # #                     N O T A B L E  H I G H  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
P r o g r a m  N a m e :  X X X X X X X X . s a s                   R u n  D a t e :  D D M M M Y Y Y Y  H H : M M                                  D a t a b a s e  l a s t  m o d i f i e d :  D D M M Y Y Y Y  


 
P a ge 1 9 5  of 2 0 8  
 
T he f oll o wi n g listi n gs will ha ve t he sa me la y o ut as Listi n g D 6: 
L i s t i n g  D 7  
C e n t r a l  L a b o r a t o r y  D a t a  -  H e m a t o l o g y  
A l l  R a n d o m i z e d  S u b j e c t s  
 
L i s t i n g  D 8  
C e n t r a l  L a b o r a t o r y  D a t a  -  U r i n a l y s i s  
A l l  R a n d o m i z e d  S u b j e c t s  
 
L i s t i n g  D 9  
C e n t r a l  L a b o r a t o r y  D a t a  –  U r i n e  C h e m i s t r y  
A l l  R a n d o m i z e d  S u b j e c t s  
 
L i s t i n g  D 1 0 . 1  
L o c a l  L a b o r a t o r y  D a t a  –  S e r u m  C h e m i s t r y ,  H e m a t o l o g y ,  a n d  C o a g u l a t i o n  
A l l  R a n d o m i z e d  S u b j e c t s  
 
L i s t i n g  D 1 0 . 2  
L o c a l  L a b o r a t o r y  D a t a  –  V i r o l o g y ,  I m m u n o l o g y ,  a n d  C h e s t  X - r a y  R e s u l t s  
A l l  R a n d o m i z e d  S u b j e c t s  
 
N ote t o pr o gra m mer: Verif y t he c o nte nts of t his ta ble wit h data ma na ge me nt. 


 
P a ge 1 9 6  of 2 0 8  
 
C h e m o C e n t r y x ,  I n c .  
P r o t o c o l  C L 0 0 2 _ 1 6 8                                                                                                          P a g e  1  o f  #  
L i s t i n g  D 1 1  
L o c a l  L a b o r a t o r y  D a t a  -  U r i n a l y s i s  
A l l  R a n d o m i z e d  S u b j e c t s  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
                                                          R B C                           A n y  
  S t e p                                     S a m p l e          C o u n t            R B C           C l o t s          W a s  T r a n s f u s i o n  R e q u i r e d /  
     S u b j e c t          T r e a t m e n t            C o l l e c t e d ?       ( / h p f )          C a s t s         P r e s e n t ?        A n y  U r i n a r y  T r a c t  O b s t r u c t i o n ?  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
S t e p  1  
    # # # # - # # # #    X X X X X X X X X X X X X X X X X X X X X X       X X X             # # #           X X X X X X X X        X X X                      X X X  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
P r o g r a m  N a m e :  X X X X X X X X . s a s                   R u n  D a t e :  D D M M M Y Y Y Y  H H : M M                                  D a t a b a s e  l a s t  m o d i f i e d :  D D M M Y Y Y Y  


 
P a ge 1 9 7  of 2 0 8  
 
C h e m o C e n t r y x ,  I n c .  
P r o t o c o l  C L 0 0 2 _ 1 6 8                                                                                                          P a g e  1  o f  #  
L i s t i n g  D 1 2  
B i r m i n g h a m  V a s c u l i t i s  A c t i v i t y  S c o r e  ( B V A S )  
A l l  R a n d o m i z e d  S u b j e c t s  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
                                                   W a s                                                         C h a n g e        %  C h a n g e  
  S t e p                                              B V A S                              B a s e l i n e       V i s i t         f r o m           f r o m  
   S u b j e c t   T r e a t m e n t     V i s i t           D a t e      A s s e s s e d ?   C a t e g o r y                   S c o r e        S c o r e        B a s e l i n e      B a s e l i n e  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
  S t e p  1  
 
   # # # - # # #   X X X X X X X X X   S c r e e n i n g      D D M M M Y Y Y Y       Y e s      G e n e r a l                     # #            # #           # #            # # . #  
                                                            C u t a n e o u s  
                                                            M u c o u s  m e m b r a n e / e y e s  
                                                            E a r ,  n o s e ,  t h r o a t  
                                                            C h e s t  
                                                            C a r d i o v a s c u l a r  
                                                            A b d o m i n a l  
                                                            R e n a l  
                                                            N e r v o u s  s y s t e m  
                                                            O t h e r  
                                                            B V A S  T o t a l  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
P r o g r a m  N a m e :  X X X X X X X X . s a s                   R u n  D a t e :  D D M M M Y Y Y Y  H H : M M                            D a t a b a s e  l a s t  m o d i f i e d :  D D M M Y Y Y Y  H H : M M  


 
P a ge 1 9 8  of 2 0 8  
 
C h e m o C e n t r y x ,  I n c .  
P r o t o c o l  C L 0 0 2 _ 1 6 8                                                                                                          P a g e  1  o f  #  
L i s t i n g  D 1 3  
V a s c u l i t i s  D a m a g e  I n d e x  
A l l  R a n d o m i z e d  S u b j e c t s  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
                                                   W a s                                                     C h a n g e        %  C h a n g e  
  S t e p                                              V D I                            B a s e l i n e      V i s i t        f r o m           f r o m  
   S u b j e c t   T r e a t m e n t     V i s i t           D a t e      A s s e s s e d ?    C a t e g o r y             S c o r e         S c o r e        B a s e l i n e      B a s e l i n e  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
  S t e p  1  
     
   # # # - # # #   X X X X X X X X X   S c r e e n i n g      D D M M M Y Y Y Y       Y e s       M u s c u l o s k e l a t a l        # #            # #           # #            # # . #  
                                                             S k i n / M u c o u s  m e m b r a n e s  
                                                             O c u l a r  
                                                             E N T  
                                                             P u l m o n a r y  
                                                             C a r d i o v a s c u l a r  
                                                             P V D  
                                                             G a s t r o i n t e s t i n a l  
                                                             R e n a l  
                                                             N e u r o p s y c h i a t r i c  
                                                             O t h e r  
                                                             O v e r a l l  V D I  S c o r e  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
P r o g r a m  N a m e :  X X X X X X X X . s a s                   R u n  D a t e :  D D M M M Y Y Y Y  H H : M M                            D a t a b a s e  l a s t  m o d i f i e d :  D D M M Y Y Y Y  H H : M M  


 
P a ge 1 9 9  of 2 0 8  
 
C h e m o C e n t r y x ,  I n c .  
P r o t o c o l  C L 0 0 2 _ 1 6 8                                                                                                          P a g e  1  o f  #  
L i s t i n g  D 1 4  
A N C A  R e s u l t s  
A l l  R a n d o m i z e d  S u b j e c t s  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
                                                                                                      C h a n g e        %  C h a n g e  
  S t e p                                                                        B a s e l i n e       V i s i t        f r o m           f r o m  
   S u b j e c t   T r e a t m e n t     V i s i t           D a t e        P a r a m e t e r                  R e s u l t        R e s u l t       B a s e l i n e      B a s e l i n e  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
  S t e p  1  
 
   # # # - # # #   X X X X X X X X X   S c r e e n i n g      D D M M M Y Y Y Y      A n t i - P R 3  ( I U / m L )             # #            # #           # #            # # . #  
                                                   A n t i - M P O  ( I U / m L )  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
-  B a s e l i n e  i s  d e f i n e d  a s  t h e  l a s t  p r e - d o s e  v a l u e .  
 
P r o g r a m  N a m e :  X X X X X X X X . s a s                   R u n  D a t e :  D D M M M Y Y Y Y  H H : M M                            D a t a b a s e  l a s t  m o d i f i e d :  D D M M Y Y Y Y  H H : M M  


 
P a ge 2 0 0  of 2 0 8  
 
C h e m o C e n t r y x ,  I n c .  
P r o t o c o l  C L 0 0 2 _ 1 6 8                                                                                                          P a g e  1  o f  #  
L i s t i n g  D 1 5  
S e r u m  h s C R P  R e s u l t s  ( m g / L )  
A l l  R a n d o m i z e d  S u b j e c t s  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
                                                                                      C h a n g e        %  C h a n g e  
  S t e p                                                        B a s e l i n e      V i s i t         f r o m           f r o m  
   S u b j e c t       T r e a t m e n t       V i s i t             D a t e           R e s u l t       R e s u l t      B a s e l i n e       B a s e l i n e  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
  S t e p  1  
      
   # # # - # # #       X X X X X X X X X     S c r e e n i n g        D D M M M Y Y Y Y            # #          # #           # #            # # . #                                              
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
-  B a s e l i n e  i s  d e f i n e d  a s  t h e  l a s t  p r e - d o s e  v a l u e .  
 
P r o g r a m  N a m e :  X X X X X X X X . s a s                   R u n  D a t e :  D D M M M Y Y Y Y  H H : M M                            D a t a b a s e  l a s t  m o d i f i e d :  D D M M Y Y Y Y  H H : M M  


 
P a ge 2 0 1  of 2 0 8  
 
C h e m o C e n t r y x ,  I n c .  
P r o t o c o l  C L 0 0 2 _ 1 6 8                                                                                                          P a g e  1  o f  #  
L i s t i n g  D 1 6  
S e r u m  U r i n a r y  M C P - 1 : C r e a t i n i n e  R a t i o  R e s u l t s  ( p g / m g  C r e a t i n i n e )  
A l l  R a n d o m i z e d  S u b j e c t s  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
                                                                                      C h a n g e        %  C h a n g e  
  S t e p                                                        B a s e l i n e      V i s i t         f r o m           f r o m  
   S u b j e c t       T r e a t m e n t       V i s i t             D a t e           R e s u l t       R e s u l t      B a s e l i n e       B a s e l i n e  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
  S t e p  1  
 
   # # # - # # #       X X X X X X X X X     S c r e e n i n g        D D M M M Y Y Y Y            # #          # #           # #            # # . #  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
-  B a s e l i n e  i s  d e f i n e d  a s  t h e  l a s t  p r e - d o s e  v a l u e .  
 
P r o g r a m  N a m e :  X X X X X X X X . s a s                   R u n  D a t e :  D D M M M Y Y Y Y  H H : M M                            D a t a b a s e  l a s t  m o d i f i e d :  D D M M Y Y Y Y  H H : M M  


 
P a ge 2 0 2  of 2 0 8  
 
C h e m o C e n t r y x ,  I n c .  
P r o t o c o l  C L 0 0 2 _ 1 6 8                                                                                                          P a g e  1  o f  #  
L i s t i n g  D 1 7  
E s t i m a t e d  G l o m e r u l a r  F i l t r a t i o n  R a t e  ( M D R D )  R e s u l t s  ( m L / m i n / 1 . 7 3  m ^ 2 )  
A l l  R a n d o m i z e d  S u b j e c t s  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
                                                                                      C h a n g e        %  C h a n g e  
  S t e p                                                        B a s e l i n e      V i s i t         f r o m           f r o m  
   S u b j e c t       T r e a t m e n t       V i s i t             D a t e           R e s u l t       R e s u l t      B a s e l i n e       B a s e l i n e  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
  S t e p  1  
      
   # # # - # # #       X X X X X X X X X     S c r e e n i n g        D D M M M Y Y Y Y            # #          # #           # #            # # . #  
                                                    
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
-  B a s e l i n e  i s  d e f i n e d  a s  t h e  l a s t  p r e - d o s e  v a l u e .  
 
P r o g r a m  N a m e :  X X X X X X X X . s a s                   R u n  D a t e :  D D M M M Y Y Y Y  H H : M M                            D a t a b a s e  l a s t  m o d i f i e d :  D D M M Y Y Y Y  H H : M M  


 
P a ge 2 0 3  of 2 0 8  
 
C h e m o C e n t r y x ,  I n c .  
P r o t o c o l  C L 0 0 2 _ 1 6 8                                                                                                          P a g e  1  o f  #  
L i s t i n g  D 1 8  
A C R  R e s u l t s  ( m g / g )  
A l l  R a n d o m i z e d  S u b j e c t s  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
                                                                                      C h a n g e        %  C h a n g e  
  S t e p                                                        B a s e l i n e      V i s i t         f r o m           f r o m  
   S u b j e c t       T r e a t m e n t       V i s i t             D a t e           R e s u l t       R e s u l t      B a s e l i n e       B a s e l i n e  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
  S t e p  1  
      
   # # # - # # #       X X X X X X X X X     S c r e e n i n g        D D M M M Y Y Y Y            # #          # #           # #            # # . #  
                                                    
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
-  B a s e l i n e  i s  d e f i n e d  a s  t h e  l a s t  p r e - d o s e  v a l u e .  
 
P r o g r a m  N a m e :  X X X X X X X X . s a s                   R u n  D a t e :  D D M M M Y Y Y Y  H H : M M                            D a t a b a s e  l a s t  m o d i f i e d :  D D M M Y Y Y Y  H H : M M  


 
P a ge 2 0 4  of 2 0 8  
 
C h e m o C e n t r y x ,  I n c .  
P r o t o c o l  C L 0 0 2 _ 1 6 8                                                                                                          P a g e  1  o f  #  
L i s t i n g  D 1 9  
V i t a l  S i g n s ,  W e i g h t ,  a n d  B M I   
A l l  R a n d o m i z e d  S u b j e c t s  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
                                                                                                                    C h a n g e  
  S t e p                                                                                    B a s e l i n e       V i s i t          f r o m  
   S u b j e c t   T r e a t m e n t     V i s i t           D a t e                  P a r a m e t e r                     R e s u l t        R e s u l t        B a s e l i n e  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
  S t e p  1  
 
   # # # - # # #   X X X X X X X X X   S c r e e n i n g      D D M M M Y Y Y Y     S y s t o l i c  B l o o d  P r e s s u r e  ( m m H g )              # #           # #            # #  
                                                  D i a s t o l i c  B l o o d  P r e s s u r e  ( m m H g )  
                                                  H e a r t  R a t e  ( b p m )  
                                                  O r a l  T e m p e r a t u r e  ( F )  
                                                  H e i g h t  ( c m )  
                                                  W e i g h t  ( k g )  
                                                  B o d y  M a s s  I n d e x  ( k g / m ^ 2 )  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
P r o g r a m  N a m e :  X X X X X X X X . s a s                   R u n  D a t e :  D D M M M Y Y Y Y  H H : M M                            D a t a b a s e  l a s t  m o d i f i e d :  D D M M Y Y Y Y  H H : M M  


 
P a ge 2 0 5  of 2 0 8  
 
C h e m o C e n t r y x ,  I n c .  
P r o t o c o l  C L 0 0 2 _ 1 6 8                                                                                                          P a g e  1  o f  #  
L i s t i n g  D 2 0  
P h y s i c a l  E x a m  a n d  B o d y  S y s t e m  R e v i e w s   
A l l  R a n d o m i z e d  S u b j e c t s  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
  S t e p  
   S u b j e c t       T r e a t m e n t       V i s i t         D a t e      B o d y  S y s t e m                          R e s u l t         A b n o r m a l  F i n d i n g ( s )  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
  S t e p  1  
 
   # # # - # # #       X X X X X X X X X     S c r e e n i n g    D D M M M Y Y Y Y    G e n e r a l  A p p e r a n c e / M e n t a l  S t a t u s      N o t  d o n e  
                                                     H E E N T                                N o r m a l  
                                                     D e r m a t o l o g i c                         N o r m a l  
                                                     C a r d i o v a s c u l a r                       N o r m a l  
                                                     R e s p i r a t o r y                          N o r m a l  
                                                     G a s t r o i n t e s t i n a l                     N o r m a l  
                                                     M u s c u l o s k e l e t a l                      N o r m a l  
                                                     N e u r o l o g i c                           A b n o r m a l      x x x x x x x x x x x x x x x x x x x x x x x x x x x x x x  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
P r o g r a m  N a m e :  X X X X X X X X . s a s                   R u n  D a t e :  D D M M M Y Y Y Y  H H : M M                            D a t a b a s e  l a s t  m o d i f i e d :  D D M M Y Y Y Y  H H : M M  


 
P a ge 2 0 6 of 2 0 8  
 
C h e m o C e n t r y x ,  I n c .  
P r o t o c o l  C L 0 0 2 _ 1 6 8                                                                                                          P a g e  1  o f  #  
L i s t i n g  D 2 1  
S F - 3 6 v 2  H e a l t h  S u r v e y  
A l l  R a n d o m i z e d  S u b j e c t s  i n  S t e p  3  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
  S U B J E C T :  # # # - # # #    S T E P :  X  T R E A T M E N T :  X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X  
- - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - -  
    V I S I T :  D a y  1    D A T E :  M M D D Y Y Y Y  
   - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - -  
 
  I n  g e n e r e r a l ,  w o u l d  y o u  s a y  y o u r  h e a l t h  i s :                                                               X X X X X X X X X X X X X X X X X X X X  
  C o m p a r e d  t o  o n e  y e a r  a g o ,  h o w  w o u l d  y o u  r a t e  y o u r  h e a l t h  i n  g e n e r a l  n o w :                                  X X X X X X X X X X X X X X X X X X X X  
 
  H o w  m u c h  d o e s  y o u r  h e a l t h  n o w  l i m i t  y o u  i n  t h e  f o l l o w i n g  a c t i v i t i e s :  
    V i g o r o u s  a c t i v i t i e s :                                                                                    X X X X X X X X X X X X X X X X X X X X X  
    M o d e r a t e  a c t i v i t e s :  
    L i f t i n g  o f  c a r r y i n g  g r o c i e r i e s :  
    C l i m b i n g  s e v e r a l  f l i g h t s  o f  s t a i r s :  
    C l i m b i n g  o n e  f l i g h t  o f  s t a i r s :  
    B e n d i n g ,  k n e e l i n g ,  o r  s t o o p i n g :  
    W a l k i n g  m o r e  t h a n  a  m i l e :  
    W a l k i n g  s e v e r a l  h u n d r e d  y a r d s :  
    W a l k i n g  o n e  h u n d r e d  y a r d s :  
    B a t h i n g  o r  d r e s s i n g  y o u r s e l f :  
 
  D u r i n g  t h e  p a s t  4  w e e k s ,  h o w  m u c h  o f  t h e  t i m e  h a v e  y o u  h a d  p r o b l e m s  w i t h  t h e  f o l l o w i n g  a s  a  r e s u l t  o f  y o u r  p h y s c i a l  h e a l t h :  
    C u t  d o w n  o n  t h e  a m o u n t  o f  t i m e  y o u  s p e n t  o n  w o r k  o r  o t h e r  a c t i v i t i e s :  
    A c c o m p l i s h e d  l e s s  t h a n  y o u  w o u l d  l i k e :  
    W e r e  l i m i t e d  i n  t h e  k i n d  o f  w o r k  o r  o t h e r  a c t i v i t i e s :  
    H a d  d i f f i c u l t y  p e r f o r m i n g  t h e  w o r k  o r  o t h e r  a c t i v i t i e s :  
 
  D u r i n g  t h e  p a s t  4  w e e k s ,  h o w  m u c h  o f  t h e  t i m e  h a v e  y o u  h a d  p r o b l e m s  w i t h  t h e  f o l l o w i n g  a s  a  r e s u l t  o f  y o u r  e m o t i o n a l  h e a l t h :  
    C u t  d o w n  o n  t h e  a m o u n t  o f  t i m e  y o u  s p e n t  o n  w o r k  o r  o t h e r  a c t i v i t i e s :  
    A c c o m p l i s h e d  l e s s  t h a n  y o u  w o u l d  l i k e :  
    D i d  w o r k  o r  o t h e r  a c t i v i t i e s  l e s s  c a r e f u l l y  t h a n  u s u a l :  
 
  D u r i n g  t h e  p a s t  4  w e e k s ,  t o  w h a t  e x t e n t  h a s  p h y s i c a l  o r  e m o t i o n a l  h e a l t h  i n t e r f e r e d  w i t h  s o c i a l  a c t i v i t e s :   
  H o w  m u c h  b o d i l y  p a i n  h a v e  y o u  h a d  i n  t h e  l a s t  4  w e e k s :  
  D u r i n g  t h e  p a s t  4  w e e k s ,  h o w  m u c h  d i d  p a i n  i n t e r f e r e  w i t h  y o u r  n o r m a l  w o r k :  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
P r o g r a m  N a m e :  X X X X X X X X . s a s                   R u n  D a t e :  D D M M M Y Y Y Y  H H : M M                            D a t a b a s e  l a s t  m o d i f i e d :  D D M M Y Y Y Y  H H : M M  
 


 
P a ge 2 0 7  of 2 0 8  
 
C h e m o C e n t r y x ,  I n c .  
P r o t o c o l  C L 0 0 2 _ 1 6 8                                                                                                          P a g e  2  o f  #  
L i s t i n g  D 2 1  
S F - 3 6 v 2  H e a l t h  S u r v e y  
A l l  R a n d o m i z e d  S u b j e c t s  i n  S t e p  3  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
  S U B J E C T :  # # # - # # #    S T E P :  X  T R E A T M E N T :  X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X  
- - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - -  
    V I S I T :  D a y  1    D A T E :  M M D D Y Y Y Y  
  - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - -  
 
  D u r i n g  t h e  p a s t  4  w e e k s :  
    D i d  y o u  f e e l  f u l l  o f  l i f e :  
    H a v e  y o u  b e e n  v e r y  n e r v o u s :  
    H a v e  y o u  f e l t  s o  d o w n  i n  t h e  d u m p s  t h a t  n o t h i n g  c o u l d  c h e e r  y o u  u p :  
    H a v e  y o u  f e l t  c a l m  a n d  p e a c e f u l :  
    D i d  y o u  h a v e  a  l o t  o f  e n e r g y :  
    H a v e  y o u  f e l t  d o w n h e a r t e d  a n d  l o w :  
    D i d  y o u  f e e l  w o r n  o u t :  
    H a v e  y o u  b e e n  h a p p y :  
    D i d  y o u  f e e l  t i r e d :  
 
  D u r i n g  t h e  p a s t  4  w e e k s ,  h o w  m u c h  o f  t h e  t i m e  h a s  y o u r  p h y s i c a l  o r  e m o t i o n a l  p r o b l e m s  i n t e r f e r e d  w i t h  y o u r  s o c i a l  a c t i v i t i e s :  
      X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X X  
 
  H o w  T R U E  o r  F A L S E  i s  e a c h  o f  t h e  f o l l o w i n g  s t a t e m e n t s  f o r  y o u :  
    I  s e e m  t o  g e t  i l l  m o r e  e a s i l y  t h a n  o t h e r  p e o p l e  
    I  a m  a s  h e a l t h y  a s  a n y b o d y  I  k n o w :  
    I  e x p e c t  m y  h e a l t h  t o  g e t  w o r s e :  
    M y  h e a l t h  i s  e x c e l l e n t :  
 
  - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - - -  
  S u m m a r y  S c o r e s :  
    R o l e - P h y s i c a l :  
    R o l e - E m o t i o n a l :  
    S o c i a l  F u n c t i o n i n g :  
    B o d i l y  P a i n :  
    M e n t a l  H e a l t h :  
    G e n e r a l  H e a l t h  P e r c e p t i o n s :  
    C h a n g e  i n  H e a l t h :  
    P h y s c i a l  C o m p o n e n t  S u m m a r y :  
    M e n t a l  H e a l t h  S u m m a r y :  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
P r o g r a m  N a m e :  X X X X X X X X . s a s                   R u n  D a t e :  D D M M M Y Y Y Y  H H : M M                            D a t a b a s e  l a s t  m o d i f i e d :  D D M M Y Y Y Y  H H : M M  


 
P a ge 2 0 8  of 2 0 8  
 
C h e m o C e n t r y x ,  I n c .  
P r o t o c o l  C L 0 0 2 _ 1 6 8                                                                                                          P a g e  1  o f  #  
L i s t i n g  D 2 2  
E Q - 5 D - 5 L  Q u e s t i o n n a i r e  a n d  V A S  
A l l  R a n d o m i z e d  S u b j e c t s  i n  S t e p  3  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
 S t e p  
   S u b j e c t   T r e a t m e n t        V i s i t        D a t e        Q u e s t i o n                              R e s u l t  
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
  S t e p  1  
 
   # # # - # # #   X X X X X X X X X X X X     D a y  1      D D M M M Y Y Y Y     M o b i l i t y                              X X X X X X X X X X X X X X X  
                                                   S e l f - c a r e  
                                                   U s u a l  a c t i v i t i e s  
                                                   P a i n / D i s c o m f o r t  
                                                   A n x i e t y  D e p r e s s i o n  
                                                   H e a l t h  S c a l e  S c o r e  
                                                   V A S         
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _  
 
P r o g r a m  N a m e :  X X X X X X X X . s a s                   R u n  D a t e :  D D M M M Y Y Y Y  H H : M M                            D a t a b a s e  l a s t  m o d i f i e d :  D D M M Y Y Y Y  H H : M M