Phase II Study of MGTA-145 in Combination With Plerixafor in the Mobilization of Hematopoietic Stem Cells for Autologous Transplantation in Patients With Multiple Myeloma
Phase: PHASE2
Conditions: Multiple Myeloma
Sponsor: Surbhi Sidana, MD

 
1  
 
St a nf o r d C a n c e r I n stit ut e  
 
TI T L E: P h a s e II St u d y of M G T A -1 4 5 i n c o m bi n ati o n wit h pl e ri x af o r i n t h e m o bili z ati o n of 
h e m at o p oi eti c st e m c ell s f o r a ut ol o g o u s t r a n s pl a nt ati o n i n p ati e nt s wit h m ulti pl e m y el o m a     
 
C o or di n ati n g C e nt er  
St a nf or d C a nc er I nstit ut e  
8 7 5 Bl a k e Wil b ur Dri v e  
St a nf or d, C A 9 4 3 0 5  
 
Pr ot oc ol Dir e ct or  
N a m e : S ur b hi Si d a n a, M D 
A d dr ess :  3 0 0 P ast e ur Dri v e H 0 1 0 1c, St a nf or d, C A, 9 4 3 0 5 
T el e p h o n e : 6 5 0-7 2 3 -0 8 2 2  
F a x : 6 5 0-7 2 5 -9 5 9 0  
e -m ail a d dr ess : sur b hi.si d a n a @st a nf or d. e d u  
 
C o -I n v esti g at ors 
J u dit h S hiz ur u, M D, P h D 
E v er ett M e y er, M D, P h D  
L ori M uffl y, M D  
D a vi d Mi kl os M D, P h D  
P ar v e e n S hir a z, M D  
M att h e w Fr a n k, M D, P h D  
R o b ert L o ws k y, M D  
A n dr e w R e z v a ni, M D  
W e n -K ai W e n g , M D, P h D 
L a ur a J o h nst o n, M D  
S all y  Ar ai, M D  
 
Bi ost atistici a n s 
N a m e s: J o h n T a m ar esis, P h D a n d Yi n g L u, P h D  
A d dr ess : 
 
e -m ail a d dr ess : 
 

Pr ot oc ol: M G T A -1 4 5 + pl eri x af or m o bili z ati o n  
 
Pr ot oc ol V ersi o n 1. 4 , 2 1 A pril  2 0 2 1  
2  
I R B-5 7 0 5 6  
 
 
St u d y C o or di n at or  
N a m e : K h a n h N g u y e n 
A d dr ess  
 
T el e p h o n e : ( 6 5 0) 7 2 1-2 3 7 2  
 
e -m ail a d dr ess : k h a n h p n @st a nf or d. e d u 
 
P h ar m a c e utic al P art n er:  M a g e nt a T h er a p e utics  
 
V e rsi o n 1. 4  / V e rsi o n D at e: 2 1 A p ril  2 0 2 1  
 
 
 
 

Pr ot oc ol: M G T A -1 4 5 + pl eri x af or m o bili z ati o n  
 
Pr ot oc ol V ersi o n 1. 4 , 2 1 A pril  2 0 2 1  
3  
I R B-5 7 0 5 6  
 
 
T A B L E O F C O N T E N T S  
P R O T O C O L DI R E C T O R  ........................................................................................................................................... 1  
C O -I N V E S TI G A T O R S ............................................................................................................................................... 1  
BI O S T A TI S TI CI A N S  ................................................................................................................................................. 1  
S T U D Y C O O R DI N A T O R  .......................................................................................................................................... 2  
T A B L E O F C O N T E N T S  ............................................................................................................................................. 3  
T A B L E O F T A B L E S  ................................................................................................................................................... 6  
P R O T O C O L S Y N O P SI S ............................................................................................................................................. 7  
1. 1  
E X P L O R A T O R Y O BJ E C TI V E S  ...................................................................................................................................... 8  
1. 2  
E X P L O R A T O R Y E N D P OI N T S  ...................................................................................................................................... 8  
S T U D Y S C H E M A  ..................................................................................................................................................... 1 4  
T A B L E O F A B B R E VI A TI O N S  .............................................................................................................................. 1 5  
1  
O BJ E C TI V E S / E N D P OI N T S ......................................................................................................................... 1 7  
1. 1  
P RI M A R Y O BJ E C TI V E  .............................................................................................................................................. 1 7  
1. 2  
P RI M A R Y E N D P OI N T :.............................................................................................................................................. 1 7  
1. 3  
S E C O N D A R Y O BJ E C TI V E S  ....................................................................................................................................... 1 7  
1. 4  
S E C O N D A R Y E N D P OI N T S  ........................................................................................................................................ 1 7  
1. 5  
E X P L O R A T O R Y O BJ E C TI V E S  ................................................................................................................................... 1 8  
1. 6  
E X P L O R A T O R Y E N D P OI N T S  ................................................................................................................................... 1 8  
2  
B A C K G R O U N D  .............................................................................................................................................. 1 9  
2. 1  
A U T O L O G O U S T R A N S P L A N T A N D S T E M C E L L M O BI LI Z A TI O N I N M U L TI P L E M Y E L O M A  .......................... 1 9  
2. 1. 1  
M ulti pl e M y el o m a a n d R ol e of Tr a ns pl a nt: .......................................................................................... 1 9  
2. 1. 2  
St e m C ell M o biliz ati o n f or A S C T:  ............................................................................................................. 1 9  
2. 1. 3  
R ati o n al e f or pr o p os e d st u d y:  ................................................................................................................... 2 0  
2. 2  
M G T A -1 4 5 .............................................................................................................................................................. 2 1  
2. 3  
R A TI O N A L E  ............................................................................................................................................................... 2 4  
2. 4  
S T U D Y D E SI G N  ......................................................................................................................................................... 2 5  
2. 5  
C O R R E L A TI V E S T U DI E S B A C K G R O U N D  ............................................................................................................... 2 5  
2. 6  
P A TI E N T R E P O R T E D O U T C O M E M E A S U R E S : B A C K G R O U N D A N D E V A L U A TI O N  ......................................... 3 0  
3  
P A R TI CI P A N T S E L E C TI O N A N D E N R O L L M E N T P R O C E D U R E S  ................................................... 3 1  
3. 1  
IN C L U SI O N C RI T E RI A  .............................................................................................................................................. 3 1  
3. 2  
E X C L U SI O N C RI T E RI A  ............................................................................................................................................. 3 1  

Pr ot oc ol: M G T A -1 4 5 + pl eri x af or m o bili z ati o n  
 
Pr ot oc ol V ersi o n 1. 4 , 2 1 A pril  2 0 2 1  
4  
I R B-5 7 0 5 6  
 
3. 3  
IN F O R M E D C O N S E N T P R O C E S S  ............................................................................................................................. 3 2  
3. 4  
S T U D Y T I M E LI N E ..................................................................................................................................................... 3 2  
4  
T R E A T M E N T P L A N  ..................................................................................................................................... 3 2  
4. 1  
S T U D Y T R E A T M E N T A N D F O L L O W -U P  ................................................................................................................ 3 2  
4. 1. 1  
Pr e -m o biliz ati o n p h as e:  .............................................................................................................................. 3 2  
4. 1. 2  
St u d y Tr e at m e nt: st e m c ell m o biliz ati o n / a p her esis:  ....................................................................... 3 2  
4. 1. 3.      E v e nt M o nit ori n g / F oll o w -u p : ................................................................................................................. 3 5  
4. 2  
C RI T E RI A F O R P A TI E N T W I T H D R A W A L .............................................................................................................. 3 5  
4. 3  
S T U D Y D I S C O N TI N U A TI O N .................................................................................................................................... 3 6  
4. 4  
A L T E R N A TI V E S  ........................................................................................................................................................ 3 7  
5  
I N V E S TI G A TI O N A L A G E N T I N F O R M A TI O N ....................................................................................... 3 7  
5. 1  
IN V E S TI G A TI O N A L A G E N T M G T A -1 4 5  .............................................................................................................. 3 7  
5. 2  
A V AI L A BI LI T Y  .......................................................................................................................................................... 3 7  
5. 3  
A G E N T O R D E RI N G  ................................................................................................................................................... 3 7  
5. 4  
A G E N T A C C O U N T A BI LI T Y / P R E P A R A TI O N / A D MI NI S T R A TI O N ....................................................................... 3 7  
6  
D O S E M O DI FI C A TI O N S  .............................................................................................................................. 3 8  
7  
A D V E R S E E V E N T S A N D R E P O R TI N G P R O C E D U R E S ........................................................................ 3 8  
7. 1  
P O T E N TI A L A D V E R S E E V E N T S .............................................................................................................................. 3 8  
7. 1. 1  
M G T A -1 4 5  ......................................................................................................................................................... 3 8  
7. 1. 2  
Pl erix af or:  ......................................................................................................................................................... 4 1  
7. 2  
A D V E R S E E V E N T A N D S E RI O U S A D V E R S E E V E N T R E P O R TI N G  ...................................................................... 4 3  
7. 2. 1  
Defi niti o ns  ......................................................................................................................................................... 4 3  
7. 2. 2  
S eri o us A d v ers e E v e nt R e p orti n g  ............................................................................................................. 4 4  
7. 2. 3  
B as eli n e a n d F oll o w -u p A d v ers e E v e nt E v al u ati o ns:  ....................................................................... 4 5  
8  
L A B O R A T O R Y C O R R E L A TI V E / S P E CI A L S T U DI E S  ........................................................................... 4 7  
8. 1  
( AP H E R E SI S P R O D U C T C O M P O SI TI O N –  L A B O R A T O R Y C O R R E L A TI V E S T U D Y # 1 A A N D # 1 B ) ............... 4 7  
8. 2  
( C F U A S S A Y –  L A B O R A T O R Y C O R R E L A TI V E S T U D Y # 2)  ................................................................................. 4 8  
8. 3  
(IM M U N E R E C O N S TI T U TI O N A T D A Y 2 8  A N D 1 0 0  B Y F L O W C Y T O M E T R Y A N D A T D A Y 1 0 0  B Y C Y T O F  –  
L A B O R A T O R Y C O R R E L A TI V E S T U D Y # 3 A A N D 3 B ) .......................................................................................................... 4 8  
9  
S T U D Y C A L E N D A R  ...................................................................................................................................... 5 0  
1 0  M E A S U R E M E N T S  ......................................................................................................................................... 5 6  
1 0. 1  
H S C  YI E L D I N A P H E R E SI S P R O D U C T ( PRI M A R Y A N D S E C O N D A R Y O U T C O M E M E A S U R E S ) ....................... 5 6  
1 0. 2  
O T H E R S E C O N D A R Y O U T C O M E M E A S U R E S ........................................................................................................ 5 6  
1 0. 2. 1  
I nf usi o n r el at e d t oxiciti es wit h M G T A-1 4 5; A d v ers e e ve nts wit h m o biliz ati o n of H S Cs 

Pr ot oc ol: M G T A -1 4 5 + pl eri x af or m o bili z ati o n  
 
Pr ot oc ol V ersi o n 1. 4 , 2 1 A pril  2 0 2 1  
5  
I R B-5 7 0 5 6  
 
wit h M G T A -1 4 5 a n d pl erix af or.  ................................................................................................................................ 5 6  
1 0. 2. 2  
N e utr o p hil a n d pl at el et e n gr aft m e nt  ............................................................................................... 5 7  
1 0. 2. 3  
Tr a ns pl a nt r el at e d m ort alit y  ............................................................................................................... 5 7  
1 0. 2. 4  
N o n -r el a ps e r el at e d m ort alit y ............................................................................................................. 5 8  
1 0. 2. 5  
Pr o gr essi o n Fr e e S ur vi v al ( P F S):  ........................................................................................................ 5 8  
1 0. 2. 6  
O v er all S ur vi v al ( O S):  .............................................................................................................................. 5 8  
1 0. 2. 7  
R es p o ns e Ass ess m e nt t o Deter mi n e Pr o gr essi o n: ......................................................................... 5 8  
1 1  R E G U L A T O R Y C O N SI D E R A TI O N S  .......................................................................................................... 5 9  
1 1. 1  
IN S TI T U TI O N A L R E VI E W O F P R O T O C O L  ............................................................................................................. 5 9  
1 1. 2  
D A T A A N D S A F E T Y M O NI T O RI N G P L A N .............................................................................................................. 5 9  
1 1. 2. 1  
Pri nci p al I n v esti g at or a n d St u d y R es e arc h Te a m ........................................................................ 5 9  
1 1. 2. 2  
St a nf or d C a nc er I nstit ut e D at a a n d S af et y M o nit ori n g C o m mitte e ( D S M C)  ..................... 5 9  
1 1. 2. 3  
D at a M a n a g e m e nt Pl a n  ......................................................................................................................... 5 9  
1 2  C O L L A B O R A TI V E A G R E E M E N T S  ........................................................................................................... 6 0  
1 3  S T A TI S TI C A L C O N SI D E R A TI O N S  ........................................................................................................... 6 0  
1 3. 1  
S T A TI S TI C A L D E SI G N  .............................................................................................................................................. 6 0  
1 3. 2  
S A F E T Y R U N -IN / F U TI LI T Y R U L E  ......................................................................................................................... 6 0  
1 3. 3  
D E S C RI P TI V E S T A TI S TI C S A N D E X P L O R A T O R Y D A T A A N A L Y SI S .................................................................... 6 1  
1 3. 4  
P RI M A R Y A N A L Y SI S  ................................................................................................................................................ 6 1  
1 3. 5  
S E C O N D A R Y A N A L Y SI S  ........................................................................................................................................... 6 1  
1 3. 5. 1  
S ec o n d ar y E n d p oi nt # 1:  ......................................................................................................................... 6 1  
1 3. 5. 2  
S ec o n d ar y E n d p oi nt # 2:  ......................................................................................................................... 6 2  
1 3. 5 . 3 
S ec o n d ar y E n d p oi nt # 3:  ......................................................................................................................... 6 2  
1 3. 5. 4  
S ec o n d ar y E n d p oi nt # 4:  ......................................................................................................................... 6 2  
1 3. 5. 5  
S ec o n d ar y E n d p oi nt # 5:  ......................................................................................................................... 6 3  
1 3. 6  
S A M P L E S I Z E ............................................................................................................................................................ 6 3  
1 3. 6. 1  
Accr u al esti m at es ...................................................................................................................................... 6 3  
1 3. 6. 2  
S a m pl e siz e j ustific ati o n  ........................................................................................................................ 6 3  
1 3. 6. 3  
Eff ect siz e j ustific ati o n  ............................................................................................................................ 6 4  
1 3. 7  
C RI T E RI A F O R F U T U R E S T U DI E S  ........................................................................................................................... 6 5  
1 4  R E F E R E N C E S  ................................................................................................................................................. 6 6  
1 5  A P P E N DI C E S  ................................................................................................................................................. 7 0  
1 5. 1  
A P P E N DI X  A:  P A R TI CI P A N T E LI GI BI LI T Y C H E C K LI S T  ................................................................................... 7 0  

Pr ot oc ol: M G T A -1 4 5 + pl eri x af or m o bili z ati o n  
 
Pr ot oc ol V ersi o n 1. 4 , 2 1 A pril  2 0 2 1  
6  
I R B-5 7 0 5 6  
 
1 5. 2  
7 2  
1 5. 3  
7 2  
1 5. 4  
7 2  
A P P E N DI X B  ............................................................................................................................................................ 7 3  
 
 
T a bl e of T a bl es  
T a bl e 1: A d v ers e eff e cts wit h M G T A a n d pl eri x af or i n a p h as e 1 st u d y of h e alt h y v ol u nt e ers  ............. 2 3  
T a bl e 2: Fl o w C yt o m etr y P a n el wit h A nti b o di es f or H S C a n d pr o g e nit or c ell st ai ni n g  ............................ 2 6  
T a bl e 3: C y T O F P a n el wit h A nti b o di es f or p eri p h er al bl o o d m o n o n ucl e ar c ell i m m u n o p h e n ot y pi n g
.......................................................................................................................................................................................................... 2 6  
T a bl e 4: Fl o w C yt o m etr y P a n el wit h A nti b o di es f or T a n d N K c ell st ai ni n g  .................................................. 2 9  
T a bl e 5: Fl o w C yt o m etr y P a n el wit h A nti b o di es f or B c ell st ai ni n g  .................................................................. 2 9  
T a bl e 6: Tr e at m e nt Pl a n  ....................................................................................................................................................... 3 3  
T a bl e 7: S u m m ar y of Ac ut e B a c k P ai n  ( T a bl e s o urc e: I n v esti g at or’s Br oc h ur e) ......................................... 3 9  
T a bl e 8: A d v ers e R e a cti o ns i n ≥5 % of N o n -H o d g ki n's L y m p h o m a a n d M ulti pl e M y el o m a P ati e nts 
R e c ei vi n g Pl eri x af or a n d M or e Fr e q u e nt t h a n Pl a c e b o d uri n g H S C M o biliz ati o n a n d A p h er esis ........ 4 3  
T a bl e 9: A d v ers e E v e nt Ass ess m e nt  ................................................................................................................................ 4 6  
T a bl e 1 0 A: Sc h e d ul e of St u d y Ass ess m e nts  ................................................................................................................. 5 0  
T a bl e 1 1: St e m c ell m o biliz ati o n d at a fr o m pri or st u di es of G -C S F a n d G C S F + / -pl eri x af or i n 
m y el o m a  ...................................................................................................................................................................................... 6 4  
 
 

Pr ot oc ol: M G T A -1 4 5 + pl eri x af or m o bili z ati o n  
 
Pr ot oc ol V ersi o n 1. 4 , 2 1 A pril  2 0 2 1  
7  
I R B-5 7 0 5 6  
 
P R O T O C O L S Y N O P SI S  
TI T L E:  P h as e II St u d y of M G T A -1 4 5 i n c o m bi n ati o n wit h pl eri x af or i n t h e m o biliz ati o n of 
h e m at o p oi etic st e m c ells ( H S Cs) f or a ut ol o g o us tr a ns pl a nt ati o n i n p ati e nts wit h m ulti pl e m y el o m a   
S T U D Y P H A S E:  P h as e 2, o p e n l a b el , pr o of of c o nc e pt st u d y 
S T U D Y C E N T E R:  Si n gl e C e nt er, St a nf or d C a nc er I nstit ut e  
I N V E S TI G A TI O N A L P R O D U C T: M G T A -1 4 5 (i ntr a v e n o us dr u g)  
I N DI C A TI O N: H e m at o p oi etic s t e m c ell ( H S C) c oll e cti o n f or a ut ol o g o us st e m c ell tr a ns pl a nt ati o n i n 
m ulti pl e m y el o m a  
P RI M A R Y O BJ E C TI V E( S)  
1.  T o ass ess t h e effic a c y of M G T A -1 4 5 i n c o m bi n ati o n wit h pl eri x af or i n m o bilizi n g  a d e q u at e 
n u m b er of  h e m at o p oi etic st e m c ells  (>  2 x 1 0 6  C D 3 4 + c ells / k g) i n p ati e nts wit h m ulti pl e 
m y el o m a ( M M ) i n pr e p ar ati o n f or a ut ol o g o us st e m c ell tr a ns pl a nt ati o n ( A S C T). 
S E C O N D A R Y O BJ E C TI V E( S)  
1.  T o ass ess t h e effic a c y of M G T A -1 4 5 a n d  pl eri x af or i n m o bilizi n g diff er e nt H S Cs t ar g et g o als 
i n p ati e nts wit h M M i n pr e p ar ati o n f or A S C T. 
2.  T o ass ess t h e s af et y a n d t ol er a bilit y of M G T A -1 4 5 a n d pl eri x af or f or m o bilizi n g H S Cs i n 
p ati e nts wit h M M.  
3.  T o ass ess t h e e n gr aft m e nt r at e a n d ti m e t o e n gr aft m e nt f oll o wi n g A S C T aft er H S C 
m o biliz ati o n wit h M G T A -1 4 5 a n d  pl eri x af or  i n p ati e nts wit h M M u n d er g oi n g u pfr o nt A S C T. 
4.  T o ass ess r at e of o n g oi n g e n gr aft m e nt at d a y 3 0 a n d 1 0 0 aft er st e m c ell i nf usi o n i n p ati e nts 
wit h M M w h o ar e m o biliz e d wit h M G T A -1 4 5  a n d pl eri x af or  u n d er g oi n g u pfr o nt A S C T.  
5.  T o ass ess tr a ns pl a nt a n d dis e as e -r el at e d o utc o m es aft er m o biliz ati o n of H S Cs wit h M G T A -
1 4 5 a n d pl eri x af or  i n p ati e nts wit h M M u n d er g oi n g u pfr o nt A S C T. 
P RI M A R Y E N D P OI N T( S)  
1.  T o e v al u at e t h e r at e of s ucc essf ul c oll e cti o n i n p ati e nts wit h M M of ≥2 . 0 x  1 0 6  C D 3 4 +  
c ells / k g i n u p t o t w o d a ys of a p h er esis aft er M G T A-1 4 5  + pl eri x af or d osi n g . 
S E C O N D A R Y E N D P OI N T( S)  
1.  T o ass ess t h e r at e of s ucc essf ul c oll e cti o n of ( a) ≥4. 0  x  1 0 6  C D 3 4 +  c ells / k g i n u p t o t w o 
a p h er esis s essi o ns aft er 
M G T A -1 4 5 a n d pl eri x af or d osi n g i n p ati e nts 
wit h 
M M  ( K e y 
S e c o n d a r y E n d p oi nt)  ( b) ≥6. 0  x  1 0 6  C D 3 4 +  c ells / k g wit h u p t o t w o a p h er esis s essi o ns a ft er 
M G T A -1 4 5 
a n d 
pl eri x af or 
d osi n g i n 
p ati e nts 
wit h 
M M  ( c) ≥2. 0  x  1 0 6  C D 3 4 +  a n d 
≥4. 0  x  1 0 6  C D 3 4 +  c ells / k g wit h o n e a p h er esis s essi o n aft er M G T A -1 4 5 a n d pl eri x af or d osi n g .   
2.  T o ass ess i nf usi o n r el at e d t o xiciti es wit h M G T A -1 4 5 a n d a d v ers e e v e nts wit h m o bili z ati o n of 
H S Cs wit h M G T A -1 4 5 a n d pl eri x af or.  

Pr ot oc ol: M G T A -1 4 5 + pl eri x af or m o bili z ati o n  
 
Pr ot oc ol V ersi o n 1. 4 , 2 1 A pril  2 0 2 1  
8  
I R B-5 7 0 5 6  
 
3.  T o ass ess ti m e t o n e utr o p hil ( d efi n e d as t h e first d a y of A N C  ≥0. 5  x  1 0 9 / L f or 3  d a ys f oll o wi n g 
st e m c ell i nf usi o n) a n d pl at el et e n gr aft m e nt ( d efi n e d as first d a y of pl at el et c o u nt m or e t h a n 
or e q u al t o 2 0  x  1 0 9 / L wit h o ut tr a nsf usi o n i n t h e l ast 7 d a ys a n d wit h pl at el et c o u nt 
≥2 0  x  1 0 9 / L o n 2  s e p ar at e, s u bs e q u e nt d a ys ), a n d t o als o d et er mi n e ti m e t o first d a y of 
pl at el et c o u nt >  5 0  x  1 0 9 / L, wit h o ut n e e d f or pl at el et tr a nsf usi o n i n 4 8 h o urs  i n p ati e nts 
u n d er g oi n g u pfr o nt A S C T.  
4.  T o ass ess r at es of o n g oi n g s ucc essf ul e n gr aft m e nt at 3 0 - a n d 1 0 0 -d a ys p ost -tr a ns pl a nt aft er 
s ucc essf ul pri m ar y e n gr aft m e nt i n p ati e nts u n d er g oi n g u pfr o nt A S C T.  
5.  T o ass ess r at e of tr a ns pl a nt r el at e d 
m ort alit y, n o n -r el a ps e 
m ort alit y, pr ogr essi o n fr e e 
s ur vi v al a n d o v er all s ur vi v al at 1 0 0 d a ys i n p ati e nts wit h u n d er g oi n g u pfr o nt A S C T.  
 
E x pl o r at o r y  O bj e cti v es  
1.  Ass ess t h e c o m p ositi o n of t h e a p h er esis pr o d uct wit h m o biliz ati o n wit h M G T A -1 4 5 a n d 
pl eri x af or i n p ati e nts wit h M M.  
2.  Ass ess pr olif er ati o n a n d diff er e nti ati o n p ot e nti al of H S Cs c oll e ct e d wit h M G T A -1 4 5 a n d 
pl eri x af or vi a c ol o n y -f or mi n g u nit ass a ys. 
3.  T o ass ess i m m u n ol o gic al r e c o v er y i n p ati e nts wit h M M u n d er g oi n g u pfr o nt tr a ns pl a nt ati o n 
aft er i nf usi o n of H S Cs c oll e ct e d wit h M G T A -1 4 5 a n d pl eri x af or m o biliz ati o n.  
4.  T o ass ess p ati e nt r e p ort e d s y m pt o ms wit h st e m c ell m o biliz ati o n wit h M G T A -1 4 5 a n d 
pl eri x af or i n p ati e nts wit h M M.  
 
E x pl o r at o r y  E n d p oi nt s  
1.  T o ass ess  t h e c o m p ositi o n of t h e a p h er esis pr o d uct o bt ai n e d aft er M G T A-1 4 5 a n d pl eri x af or 
m o biliz ati o n t hr o u g h 
e n u m er ati o n 
C D 3 4
+ C D 9 0 + C D 4 5 R A - c ells i n t h e 
pr o d uct 
a n d 
i m m u n ol o gic al c h ar a ct eriz ati o n of t h e T c ell s u bs ets, B c ell s u bs ets, N K c ells, n e utr o p hils, a n d 
m y el oi d / m o n oc ytic c ells i n t h e pr o d uct usi n g fl o w c yt o m etr y a n d C y T O F ( C yt o m etr y b y 
Ti m e of Fli g ht)  a n d mi ni m al r esi d u al dis e as e ( M R D) ass ess m e nt usi n g n e xt g e n er ati o n fl o w 
c yt o m etr y ( N G F) .  
 
2.  Ass ess pr olif er ati o n a n d diff er e nti ati o n p ot e nti al of H S Cs c oll e ct e d wit h M G T A-1 4 5 a n d 
pl eri x af or vi a c ol o n y -f or mi n g u nit ass a ys fr o m t h e a p h er esis a ut o gr aft. 
 
3.  T o ass ess i m m u n ol o gic al c h ar a ct eriz ati o n of w hit e c ell p o p ul ati o n s u bs ets ( T c ell s u bs ets, B 
c ell s u bs ets, N K c ells, n e utr o p hils, a n d m y el oi d / m o n oc ytic c ells)  i n p ati e nt’s p eri p h er al 
bl o o d at d a y 2 8 usi n g fl o w c yt o m etr y a n d at d a y 1 0 0 usi n g fl o w c yt o m etr y a n d m ass 
c yt o m etr y / C y T O F a n d ass ess i m m u n ol o gic al c h ar act eriz ati o n of 
w hit e c ell p o p ul ati o n 
s u bs ets i n t h e b o n e m arr o w micr o e n vir o n m e nt at d a y 1 0 0 f oll o wi n g t r a ns pl a nt. 
 
4.  T o ass ess p ati e nt r e p ort e d s y m pt o ms, usi n g it e ms f or i nf usi o n r e a cti o n a n d p ai n a d a pt e d 
fr o m t h e p ati e nt r e p ort e d o utc o m es c o m m o n t er mi n ol o g y crit eri a f or a d v ers e e v e nts 
( P R O C T C A E) as w ell as t h e bri ef p ai n i n v e nt or y t o ol f oll o wi n g pl eri x af or a n d M G T A -1 4 5 
i nf usi o n. 
B A C K G R O U N D A N D R A TI O N A L E : 
A ut ol o g o us st e m c ell tr a ns pl a nt is a n eff e cti v e t h er a p y f or m ulti pl e m y el o m a a n d r e m ai ns a st a n d ar d 
of c ar e f or eli gi bl e p ati e nts. 1  W hil e m ost p ati e nts u n d er g o u pfr o nt tr a ns pl a nt s o o n aft er st e m c ell 
c oll e cti o n, s o m e p ati e nts 
m a y o pt t o d el a y tr a ns pl a nt u ntil r el a ps e. C oll e cti o n of C D 3 4 + 
h e m at o p oi etic st e m c ells ( H S Cs) t o r esc u e t h e b o n e m arr o w fr o m eff e cts of hi g h -d os e c h e m ot h er a p y 

Pr ot oc ol: M G T A -1 4 5 + pl eri x af or m o bili z ati o n  
 
Pr ot oc ol V ersi o n 1. 4 , 2 1 A pril  2 0 2 1  
9  
I R B-5 7 0 5 6  
 
i n A S C T is m ost c o m m o nl y d o n e t hr o u g h m o biliz ati o n a n d c oll e cti o n of H S Cs  fr o m t h e p eri p h er al 
bl o o d. T h e t w o c o m m o n str at e gi es t o a c hi e v e t his ar e  c h e m ot h er a p y -b as e d m o biliz ati o n al o n g wit h 
gr a n ul oc yt e c ol o n y sti m ul ati n g f a ct or  ( G-C S F ) or G-C S F  m o biliz ati o n  wit h or wit h o ut pl eri x af or.  
W hil e t h es e m et h o ds ar e  effic a ci o us, t h e pr oc ess c a n b e l o n g a n d is ass oci at e d wit h s o m e a d v ers e 
eff e cts. F or e x a m pl e, c h e m ot h er a p y -b as e d m o biliz ati o n c a n t a k e t w o t o t hr e e w e e ks a n d r es ult i n 
c o m plic ati o ns li k e n e utr o p e nic f e v er a n d si g nific a nt c yt o p e ni as. 2 -4  G -C S F i nj e cti o ns c a n r es ult i n 
si g nific a nt b o n e p ai n. R ar e s eri o us a d v ers e eff e cts wit h G -C S F i ncl u d e s pl e nic r u pt ur e a n d p ul m o n ar y 
t o xicit y. E v e n wit h G -C S F a n d pl eri x af or us e, at l e ast 4 d a ys of G C S F i nj e cti o ns ar e n e e d e d e v e n b ef or e 
m o bili z ati o n c a n b e gi n , r e q uiri n g 5-8 d a ys f or c oll e cti o n .5   
T h e i d e al st e m c ell m o biliz ati o n a n d c oll e cti o n m et h o d  s h o ul d r es ult i n  r a pi d a n d r eli a bl e c oll e cti o n 
of a d e q u at e C D 3 4 + H S Cs , wit h mi ni m al a d v ers e eff e cts.  A mi ni m u m of 2. 0  x  1 0 6  C D 3 4 +  c ells / k g ar e 
r e c o m m e n d e d t o pr oc e e d wit h tr a ns pl a nt p er St a nf or d B M T Di visi o n i nstit uti o n al pr ot oc ol a n d 
I nt er n ati o n al 
M y el o m a 
W or ki n g Gr o u p g ui d eli n es. C oll e cti o n of a n a d diti o n al 2. 0 x  1 0 6  C D 3 4 +  
c ells / k g is pr ef err e d as a b a c k -u p st e m c ell i nf usi o n p er i nstit uti o n al pr ot oc ol.  
 I n n o v ati o n i n t h e st e m c ell m o biliz ati o n m et h o d t o r e d uc e t h e ti m e t a k e n t o c oll e ct st e m c ells a n d 
d e cr e as e  a d v ers e eff e cts  will b e b e n efici al fr o m b ot h p ati e nt s a n d h e alt h c ar e r es o urc e utiliz ati o n . 
M G T A -1 4 5  is a C X C R 2 a g o nist , whic h h as s h o w n pr o misi n g a cti vit y f or r a pi d m o biliz ati o n of C D 3 4 + 
H S Cs i n pr e-cli nic al m o d els .6 Its a cti vit y w as s y n er gistic wit h pl eri x af or, a C X C R 4 a nt a g o nist, i n 
a ni m al m o d els  a n d i n a p h as e I st u d y of h e alt h y v ol u nt e ers, wit h r a pi d m o biliz ati o n of st e m c ells. 7   
Gi v e n t h e pr o misi n g r es ults s e e n  i n pr e-cli nic al m o d els a n d a p h as e 1 st u d y of  h e alt h y v ol u nt e ers , w e 
e x p e ct t his a g e nt t o r es ult i n f ast, eff e cti v e a n d s af e st e m c ell m o biliz ati o n i n p ati e nts wit h m ulti pl e 
m y el o m a, i n c o m bi n ati o n wit h pl eri x af or. T his p h a s e II p r o of of c o n c e pt st u d y  will e v al u at e  st e m 
c ell m o biliz ati o n i n p ati e nts wit h m y el o m a wit h a c o m bi n ati o n of M G T A -1 4 5 a n d pl eri x af or. T h e d os e 
of M G T A -1 4 5 ( 0. 0 3 m g / k g) i n c o m bi n ati o n wit h pl eri x af or h as b e e n i d e ntifi e d i n a p h as e I st u d y of 
h e alt h y v ol u nt e ers. 7  T h er e w er e mi ni m al a d diti o n al si d e eff e cts o bs er v e d wit h M G T A -1 4 5 i n t h at 
st u d y, wit h m ost p ati e nts r e p orti n g tr a nsi e nt b ac k p ai n l asti n g l ess t h a n 2 0 mi n ut es i n t h e M G T A -
1 4 5 vs.  pl a c e b o ar m. A d diti o n al e x p e ct e d si d e eff e cts, i ncl u di n g di arr h e a , w er e n ot e d i n t h e M G T A -
1 4 5 + pl eri x af or ar m. 7  Wit h t his r e gi m e n,  p ati e nts will u n d er g o s a m e d a y 
m o biliz ati o n aft er 
a d mi nistr ati o n of M G T A -1 4 5 a n d pl eri x af or f or a m a xi m u m of 2 a p h er esis s essi o ns.  
T R E A T M E N T S U M M A R Y : 
T his is a p h as e 2 st u d y t o e v al u at e s af et y a n d effic a c y of st e m c ell m o biliz ati o n (f or hi g h -d os e t h er a p y 
a n d a ut ol o g o us st e m c ell r esc u e) wit h a c o m bi n ati o n of M G T A -1 4 5 a n d pl eri x af or, f oll o w e d b y s a m e 
d a y a p h er esis i n p ati e nts wit h m ulti pl e m y el o m a.  T h e d os e of M G T A -1 4 5 i n c o m bi n ati o n wit h 
pl eri x af or h as b e e n d et er mi n e d i n a p h as e 1 st u d y o f h e alt h y v ol u nt e ers. P ati e nts will u n d er g o u p t o 
2 d a ys of m o biliz ati o n f oll o w e d b y s a m e d a y a p h er esis s essi o ns. 
T h e pri m ar y e n d p oi nt is s ucc essf ul c oll e cti o n of at l e ast ≥2 . 0 x  1 0 6  C D 3 4 +  c ells / k g wit h u p t o t w o d a ys 
o f a p h er esis s essi o n s aft er M G T A -1 4 5  a n d pl eri x af or d osi n g.  K e y s e c o n d ar y e n d p oi nt is t h e  c oll e cti o n 
of ≥4. 0  x  1 0 6  C D 3 4 +  c ells / k g i n u p t o t w o d a ys of a p h er esis . Ot h er se c o n d ar y e n d p oi nts i ncl u d e 
c oll e cti o n of ≥2. 0  x  1 0 6  C D 3 4 +  c ells / k g a n d ≥4. 0  x  1 0 6  C D 3 4 +  c ells / k g o n d a y o n e of a p h er esis a n d of 
≥4. 0  x  1 0 6  C D 3 4 +  c ells / k g a n d ≥6. 0  x  1 0 6  C D 3 4 +  c ells / k g i n u p t o t w o d a ys of a p h er esis aft er 
M G T A -1 4 5  a n d pl eri x af or d osi n g, ass ess m e nt of a d v ers e e v e nts a n d s ucc essf ul e n gr aft m e nt as 
d escri b e d a b o v e.  
T h e st u d y i s di vi d e d i nt o t h r e e p h a s es : 
1.  Pr e -m o biliz ati o n p h as e :  

Pr ot oc ol: M G T A -1 4 5 + pl eri x af or m o bili z ati o n  
 
Pr ot oc ol V ersi o n 1. 4 , 2 1 A pril  2 0 2 1  
1 0  
I R B-5 7 0 5 6  
 
P ati e nts will u n d er g o scr e e ni n g pr oc e d ur es aft er si g ni n g i nf or m e d c o ns e nt. T h e y will u n d er g o 
b as eli n e e v al u ati o n  d uri n g t h e pr e -m o biliz ati o n p h as e u p t o 3 0 d a ys b ef or e m o biliz ati o n  a n d b e 
e nr oll e d if t h e y m e et eli gi bilit y crit eri a . 
2.  St e m c ell m o biliz ati o n / a p h er esis p h as e :  
P ati e nts will u n d er g o s e q u e nti al a d mi nistr ati o n of pl eri x af or 0. 2 4 m g / k g  ( d os e 0. 1 6 m g / k g f or 
cr e ati ni n e cl e ar a nc e <  5 0 ml / mi n ut e)  s u bc ut a n e o usl y f oll o w e d 2 h o urs l at er b y M G T A -1 4 5 at  a 
d os e of  0. 0 3 m g / k g i ntr a v e n o usl y. T his will b e f oll o w e d b y a p h er esis . A p h er esis s h o ul d b e gi n as 
s o o n as f e asi bl e aft er M G T A -1 4 5 i nf usi o n is c o m pl et e.  
E a c h s essi o n of a p h er esis will b e st a n d ar diz e d t o  p r oc es s  3 bl o o d v ol u m es, wit h a m a r gi n of + / -
1 0 %  o r 4. 5 h o u r s, w hi c h e v e r i s l o n g e r.  
 
A s e c o n d d a y of m o biliz ati o n a n d a p h er esis will b e p urs u e d i n p ati e nts w h o h a v e n ot c oll e ct e d 
6. 0  x  1 0 6  C D 3 4 +  c ells / k g i n o n e s essi o n.  
 
A mi ni m u m of 2 . 0 x  1 0 6  C D 3 4 +  c ells / k g ar e r e c o m m e n d e d t o pr oc e e d wit h tr a ns pl a nt p er St a nf or d 
B M T Di visi o n  i nstit uti o n al pr ot oc ol a n d I nt er n ati o n al M y el o m a W or ki n g Gr o u p g ui d eli n es. C oll e cti o n 
of a n a d diti o n al 2. 0  x  1 0 6  C D 3 4 + c ells / k g  is pr ef err e d as a b ac k -u p st e m c ell i nf usi o n p er i nstit uti o n al 
pr ot oc ol.  
 
If t h e mi ni m u m g o al of 2. 0 x  1 0 6  C D 3 4 +  c ells / k g is n ot m et  aft er 2 d a ys of c oll e cti o n , p ati e nts m a y 
pr oc e e d wit h w as h o ut pr ot o c ol a n d alt er n ati v e st e m c ell m o biliz ati o n str at e g y  at t h e discr eti o n of 
t h e tr e ati n g p h ysici a n . 
 
F or p ati e nts c oll e cti n g b et w e e n 2. 0 a n d 4. 0 x 1 0 6  C D 3 4 +  c ells / k g , p ati e nts m a y pr oc e e d t o tr a ns pl a nt 
or c oll e ct a d diti o n al st e m c ells wit h alt er n ati v e str at e gi es t o m e et t h e t ot al g o al of 4. 0  x  1 0 6  C D 3 4 +  
c ells / k g  p er tr e ati n g p h ysici a n discr eti o n . If a n a d diti o n al c oll e cti o n is s o u g ht at t h e discr eti o n of t h e 
tr e ati n g ph ysici a n, it is r e c o m m e n d e d n ot t o c o m bi n e t h e st a n d ar d of c ar e m o biliz e d gr aft a n d t h e 
M G T A -1 4 5 + pl eri x af or m o biliz e d gr aft f or o n e tr a ns pl a nt.  
3.  E v e nt M o nit ori n g / F oll o w -u p:  
 
a.  A p ati e nt will b e c o nsi d er e d t o h a v e f ail e d m o biliz ati o n a n d will disc o nti n u e t h er a p y  
o n tri al  a n d g o t o E v e nt M o nit ori n g if t h e y f ail t o c oll ect t h e mi ni m u m of 2. 0 milli o n 
C D 3 4 c ells / k g aft er t w o a p h er esis s essi o ns.   
 
b.  P ati e nts w h o ar e n ot pr oc e e di n g wit h u pfr o nt A S C T (i. e p ati e nts w h o c oll e ct a n d st or e 
st e m c ells a n d o p t f or d el a y e d tr a ns pl a nt) will b e f oll o w e d i n E v e nt M o nit ori n g u ntil 
3 0 d a ys aft er c o m pl eti o n of tr e at m e nt. Aft er t h at n o, f urt h er f oll o w -u p is r e q uir e d.  
 
c. If p ati e nts ar e pr oc e e di n g wit h u pfr o nt tr a ns pl a nt, t h e y will b e f oll o w e d u ntil d a y 1 0 0 
f or s e c o n d ar y o utc o m es of e n gr aft m e nt, dis e as e pr o gr essi o n a n d s ur vi v al. Aft er t h at, 
n o f urt h er f oll o w -u p is r e q uir e d. P ati e nts 
will b e off er e d p artici p ati o n i n a n 
i nstit uti o n al tr a ns pl a nt r e gistr y st u d y f or l o n g er t er m f oll o w u p.  
 

Pr ot oc ol: M G T A -1 4 5 + pl eri x af or m o bili z ati o n  
 
Pr ot oc ol V ersi o n 1. 4 , 2 1 A pril  2 0 2 1  
1 1  
I R B-5 7 0 5 6  
 
d.  A d v ers e E v e nts occ urri n g aft er t h e p ati e nt c o m pl et es st u d y i nt er v e nti o n ( M G T A-1 4 5 
a n d pl eri x af or) will b e c oll e ct e d o n t h e E v e nt M o nit ori n g F or m if t h e y ar e > / = gr a d e 
3, at l e ast p ossi bl y r el at e d t o st u d y tr e at m e nt a n d h a v e n ot b e e n pr e vi o usl y r e p ort e d.  
 
e.  F or p ati e nts pr oc e e di n g wit h u pfr o nt  tr a ns pl a nt ( d efi n e d u p t o 6 0 d a ys aft er H S C 
m o biliz ati o n), m y el o a bl ati v e m el p h al a n c o n diti o ni n g ( m el p h al a n 1 4 0 - 2 0 0 m g / m 2 ) 
o n d a y -2 f oll o w e d b y i nf usi o n of c oll e ct e d H S Cs c ells o n d a y 0 p er i nstit uti o n al 
st a n d ar d of pr a ctic e pr ot oc ol. T h e m a xi m u m i nt er v a l b et w e e n st e m c ell c oll e cti o n 
a n d tr a ns pl a nt o n t his st u d y is 6 0 d a ys. Wit hi n 6 0 d a ys, t h e ti mi n g of tr a ns pl a nt is at 
t h e discr eti o n of t h e tr e ati n g p h ysici a n.  
 
f. 
If t h es e p ati e nts u n d er g o r esc u e m o biliz ati o n f or a n y r e as o n (i ncl u di n g p h ysici a n 
discr eti o n t o c oll e ct m or e t h a n 2 milli o n C D 3 4 + c ell s / k g) a n d pr oc e e d t o u pfr o nt 
tr a ns pl a nt wit h M G T A-1 4 5 a n d pl eri x af or m o biliz e d gr aft, t h e y will b e f oll o w e d f or 
s e c o n d ar y tr a ns pl a nt r el at e d o utc o m es. O n t h e ot h er h a n d, if t h e y u n d er g o u pfr o nt 
tr a ns pl a nt wit h t h e st e m c ell gr aft o bt ai n e d fr o m r esc u e m o biliz ati o n o nl y, t h e y will 
n ot b e f oll o w e d f or s e c o n d ar y o utc o m es. It is r e c o m m e n d e d n ot t o c o m bi n e gr afts 
fr o m diff er e nt m o biliz ati o n pr ot oc ols 
Eli gi bilit y C rit eri a:  
I n cl u si o n C rit eri a 
1.  Di a g n osis of m ulti pl e m y el o m a p er t h e I nt er n ati o n al M y el o m a 
W or ki n g Gr o u p (I M W G) 
crit eri a  
2.  A g e: 1 8 t o 7 0 y e ars  
3.  Eli gi bl e f or A S C T p er i nstit uti o n al g ui d eli n es  
4.  Wit hi n o n e y e ar of st art of m y el o m a t h er a p y  
5.  C ar di a c a n d p ul m o n ar y st at us s uffici e nt t o u n d er g o a p h er esis a n d tr a ns pl a nt ati o n p er 
i nstit uti o n al tr a ns pl a nt g ui d eli n es. 
6.  C alc ul at e d cr e ati ni n e cl e ar a nc e > 3 0 m L / mi n a cc or di n g t o t h e M o dific ati o n of Di et i n R e n al 
Dis e as e ( M D R D) f or m ul a.  
7.  A bs ol ut e n e utr o p hil c o u nt >  1 5 0 0 x 1 0 6 / L  a n d pl at el ets >  1 0 0, 0 0 0 x 1 0 6 / L  
8.  A bilit y t o u n d erst a n d a n d t h e willi n g n ess t o si g n a writt e n i nf or m e d c o ns e nt d oc u m e nt.  
9.  A gr e e m e nt t o us e a n a p pr o v e d f or m of c o ntr ac e pti o n f or m al e p ati e nts or f e m al e p ati e nts of 
c hil d b e ari n g p ot e nti al. 
E x cl u si o n C rit eri a  
1.  Hist or y of pri or st e m c ell tr a ns pl a nt f or m ulti pl e m y el o m a or ot h er i n dic ati o ns  
2.  Pl a n n e d t a n d e m st e m c ell tr a ns pl a nt  
3.  Pri or hist or y of f ail ur e t o c oll e ct H S Cs.  
4.  Li v er f u ncti o n t ests: T ot al bilir u bi n > 1. 5 x u p p er li mit of n or m al ( U L N)  i n t h e a bs e nc e of a 
d oc u m e nt e d hist or y of Gil b ert’s s y n dr o m e  a n d / or A S T / A L T > 3 x U L N.  
5.  K n o w n all er g y t o M G T A -1 4 5 or pl eri x af or . 
6.  Lif eti m e e x p os ur e t o l e n ali d o mi d e  or a n ot h er i m m u n o m o d ul at or y dr u g  gr e at er t h a n 6 
c u m ul ati v e m o nt hs of tr e at m e nt i. e m or e t h a n si x 2 8 -d a y c ycl es or m or e t h a n ei g ht 2 1 -d a y 
c ycl es. 

Pr ot oc ol: M G T A -1 4 5 + pl eri x af or m o bili z ati o n  
 
Pr ot oc ol V ersi o n 1. 4 , 2 1 A pril  2 0 2 1  
1 2  
I R B-5 7 0 5 6  
 
7.  Pr e g n a nt or l a ct ati n g w o m e n.   
 
S af et y M o nit o ri n g of t h e R es e a r c h : T h e PI will r e vi e w t h e st u d y p eri o dic all y t o r e vi e w t h e pr o gr ess 
of t his pr ot o c ol a n d b e k e pt a w ar e of effic a c y a n d t o xicit y iss u es. T his st u d y will b e m o nit or e d 
a cc or di n g t o t h e St a nf or d C a nc er I nstit ut e  D at a S af et y M o nit ori n g  Pl a n  t h at is c urr e ntl y i n pla c e  f or 
i n v esti g at or-i niti at e d cli nic al tri als a n d will r e p ort t o t h e St a nf or d C a nc er I nstit u t e D at a S af et y 
M o nit ori n g C o m mitt e e ( D S M C).   
T o xicit y will b e c a pt ur e d a s p er N CI C T C A E versi o n 5. 0, w h er ei n t h e t er m t o xicit y is d efi n e d as 
a d v ers e e v e nts t h at ar e cl assifi e d as eit h er p ossi bl y, pr o b a bl y, or d efi nit el y r el at e d t o st u d y 
tr e at m e nt.  N o n-h e m at ol o gic t o xiciti es will b e e v al u at e d vi a t h e or di n al C T C st a n d ar d t o xicit y 
gr a di n g. D ur ati o n of t o xicit y e v e nts c a n b e i nc or p or at e d i nt o ass ess m e nts of t o xicit y gr a di n g. 
H e m at ol o gic t o xicit y m e as ur es of t hr o m b oc yt o p e ni a, n e utr o p e ni a, a n d l e u k o p e ni a will b e ass ess e d 
usi n g c o nti n u o us v ari a bl es as t h e o utc o m e m e as ur es as w ell as c at e g oriz ati o n vi a C T C st a n d ar d 
t o xicit y gr a di n g. 
O v er all t o xicit y i nci d e nc e 
will b e e x pl or e d a n d s u m m ariz e d. Fr e q u e nc y 
distri b uti o ns, gr a p hic al t e c h ni q u es a n d ot h er d escri pti v e m e as ur es will f or m t h e b asis of t h es e 
a n al ys es . 
D R U G A V AI L A BI LI T Y  
C O M M E R CI A L A G E N T: Pl eri x af or  
P H A R M A C E U TI C A L  C O M P A N Y S U P P LI E D: M G T A -1 4 5  
S A M P L E SI Z E : 2 5 p ati e nts   
S T A TI S TI C A L C O N SI D E R A TI O N S  
S a m pl e Si z e:  F or t his si n gl e -ar m tri al wit h a bi n o mi al pri m ar y e n d p oi nt, t h e m a xi m u m s a m pl e si z e 
is 2 5 e v al u a bl e p ati e nts.  
B as e d o n hist oric al d at a wit h H S C m o biliz ati o n wit h pl eri x af or a n d G -C S F 5, 8 -1 0 , w e h y p ot h esi z e t h at 
at l e ast 9 5 % of s u bj e cts w h o 
u n d er g o m o biliz ati o n wit h  t his n o v el r e gi m e n of M G T A -1 4 5 a n d 
pl eri x af or  will m e et t h e pri m ar y e n d -p oi nt of c oll e cti n g ≥2 . 0 x  1 0 6  C D 3 4 +  c ells / k g i n t w o d a ys of 
a p h er esi s. W e m o d el t h e pri m ar y e n d p oi nt as a bi n o mi al r a n d o m v ari a bl e. 
T h e tri al will b e c o n d uct e d i n t w o s e q u e nti al s e g m e nts : t h e first f or e v al u ati o n of s af et y a n d 
f e asi bilit y, a n d t h e n if s ucc essf ul, t h e n u m b er will b e e x p a n d e d t o f urt h er e v al u at e s af et y a n d 
f e asi bilit y. I n t h e first s e g m e nt, 1 5 p ati e nts will b e e v al u at e d f or t h e pri m ar y e n d p oi nt. I n t h e s e c o n d 
s e g m e nt, w hic h will b e c o nti n g e nt u p o n a c hi e vi n g s ucc ess i n t h e first s e g m e nt, 1 0 p ati e nts will b e 
e nr oll e d t o g ai n a d diti o n al s af et y a n d f e asi bilit y e x p eri e nc e wit h t his r e gi m e n a n d t o e v al u at e t h e k e y 
s e c o n d ar y e n d p oi nt. Acc o u nti n g f or b ot h s e g m e nts, t h e t ot al s a m pl e si z e is 2 5 p ati e nts. 
A r es p o n d er is d efi n e d as a p ati e nt m e eti n g t h e pri m ar y e n d p oi nt. T h e st atistic al d esi g n f or t h e 
pri m ar y e n d p oi nt is b as e d o n a bi n o mi al e x a ct t est f or t h e n ull h y p ot h esis of a r es p o n d er r at e b ei n g 
7 0 % or l ess a n d t h e alt er n ati v e h y p ot h esis of a r es p o n d er of 9 5 % or m or e. T h e crit eri o n f or s uc c ess 
of t h e tri al is at l e ast 1 3 o ut of 1 5 p ati e nts ar e r es p o n d ers. T h e t y p e I err or r at e is 5 % a n d st atistic al 
p o w er is 8 5 %.  

Pr ot oc ol: M G T A -1 4 5 + pl eri x af or m o bili z ati o n  
 
Pr ot oc ol V ersi o n 1. 4 , 2 1 A pril  2 0 2 1  
1 3  
I R B-5 7 0 5 6  
 
T his d esi g n f e at ur es a n i m plicit f utilit y st o p pi n g r ul e. P ati e nts will b e e nr oll e d c o nti n u o usl y t o t h e 
first s e g m e nt aft er i niti al s af et y r u n-i n. T h e crit eri o n f or s uc c ess is t h at at l e ast 1 3 o ut of 1 5 p ati e nts 
ar e r es p o n d ers. T hi s i m pli es t h at if 3 o r m o r e p ati e nt s a r e n o n -r es p o n d e r s at a n y p oi nt, t h e n 
t h e t ri al will b e st o pp e d f o r f utilit y .  
T h e c oll e cti o n of ≥4. 0 x 1 0 ^ 6 C D 3 4 + wit hi n 2 d a ys a k e y s e c o n d a r y e n d p oi nt  of t his tri al.  W e 
h y p ot h esi z e t h at at l e ast 7 5 % of t h e 2 5 p ati e nts will m e et t his s e c o n d ar y e n d p oi nt . T h e  st atistic al 
d esi g n f or t h e s e c o n d ar y e n d p oi nt is b as e d o n e nr olli n g e n o u g h p ati e nts i n t h e s e c o n d s e g m e nt s uc h 
t h at t h e t ot al s a m pl e si z e will pr o d uc e a pr e cisi o n esti m at e wit h a l o w er bi n o mi al c o nfi d e nc e li mit 
t h at e xcl u d es t h e r es p o ns e r at e of t h e hist oric al c o ntr ols (i. e., 5 0 %). F or t his s e c o n d ar y e n d p oi nt, w e 
c o m p ut e a pr e cisi o n esti m at e of o ur h y p ot h esi z e d r es p o ns e r at e f or t h e sc e n ari o t h at b ot h s e g m e nts 
ar e c o m pl et e d. W e m o d el t h e s e c o n d ar y e n d p oi nt as a bi n o mi al r a n d o m v ari a bl e. W e d efi n e pr e cisi o n 
t o b e t h e l e n gt h of t h e bi n o mi al c o nfi d e nc e i nt erv al f or t h e h y p ot h esi z e d r es p o ns e r at e  of 7 5 %. W e 
c o m p ut e pr e cisi o n usi n g t h e m et h o d of Wils o n at t h e si g nific a nc e l e v el of 9 0 % w hic h is a p pr o pri at e 
f or a n e arl y-p h as e tri al. B as e d o n t h es e s p e cific ati o ns, t h e pr e cisi o n is 2 6. 8 %.  
E a rl y S af et y R u n -i n: T h e first si x p ati e nts a ccr u e d t o t his st u d y will b e p ati e nts w h o ar e pl a n n e d f or 
u pfr o nt tr a ns pl a nt f oll o wi n g c oll e cti o n. A d diti o n al p ati e nts will n ot b e gi n m o biliz ati o n u ntil t h es e si x 
p ati e nts h a v e c o m pl et e d st e m c ell m o biliz ati o n a n d ar e at l e a st 2 1 d a ys f oll o wi n g i nf usi o n of st e m 
c ells aft er m y el o a bl ati v e m el p h al a n c o n diti o ni n g. S af et y r e vi e w will b e d o n e t o e v al u at e st u d y dr u g -
r el at e d a d v ers e e v e nts, i n p artic ul ar a n y u n e x p e ct e d gr a d e 3 or hi g h er tr e at m e nt r el at e d a d v ers e 
e v e nts d uri n g t his p e ri o d. E n gr aft m e nt d at a will als o b e r e vi e w e d. If a n y u n e x p e ct e d gr a d e 3 or 
hi g h er a d v ers e e v e nts p ossi bl y, pr o b a bl y or d efi nit el y r el at e d t o t h e st u d y dr u g ar e n ot e d i n t h e first 
t hr e e p ati e nts, e nr oll m e nt will p a us e w hil e t h e PI r e vi e ws t h e st u d y wit h t h e F D A t o d et er mi n e 
w h et h er st u d y c a n c o nti n u e.    
I N D N u m b e r: 1 5 1 8 7 4  
 
 

Pr ot oc ol: M G T A -1 4 5 + pl eri x af or m o bili z ati o n  
 
Pr ot oc ol V ersi o n 1. 4 , 2 1 A pril  2 0 2 1  
1 5  
I R B-5 7 0 5 6  
 
T A B L E O F A B B R E VI A TI O N S  
 
 
 
 
A E  
A d v ers e e v e nt  
A L T  
Al a ni n e a mi n otr a nsf er as e  
A S C T  
A ut ol o g o us st e m c ell tr a ns pl a nt  
A S T  
As p art at e a mi n otr a nsf er as e  
C X C R 2  
C h e m o ki n e r e c e pt or 2  
C X C R 4  
C h e m o ki n e r e c e pt or  4  
C X C L 2  
C h e m o ki n e ( C -X -C m otif) li g a n d 2  
C B C  
C o m pl et e bl o o d c o u nt  
C D 3 4  
Cl ust er of diff er e nti ati o n 3 4  
C F U  
C ol o n y f or mi n g u nit  
C R F  
C as e r e p ort f or m  
C R  
C o m pl et e r es p o ns e  
C T C A E  
C o m m o n t er mi n ol o g y crit eri a f or a d v ers e e v e nts  
C y T O F  
C yt o m etr y b y ti m e of fli g ht  
D L T  
D os e li miti n g t o xicit y  
D S M B  
D at a s af et y m o nit ori n g b o ar d  
E C G  
El e ctr oc ar di o gr a m  
E C O G  
E ast er n c o o p er ati v e o nc ol o g y gr o u p  
E D C  
El e ctr o nic d at a c a pt ur e  
E O S  
E n d of St u d y  
G -C S F  
Gr a n ul oc yt e – c ol o n y-sti m ul ati n g f a ct or 
G C P  
G o o d cli nic al pr a ctic e  
H S Cs  
H e m at o p oi etic st e m c ells  
I B 
I n v esti g at or’s br oc h ur e 
I C F 
I nf or m e d c o ns e nt f or m 
I R B 
I nstit uti o n al r e vi e w b o ar d 
I V 
I ntr a v e n o us 
M D R D  
M o difi c ati o n of di et i n r e n al dis e as e  
M M  
M ulti pl e m y el o m a  
M R D  
Mi ni m al r esi d u al dis e as e  
N H L  
N o n -H o d g ki n l y m p h o m a  

Pr ot oc ol: M G T A -1 4 5 + pl eri x af or m o bili z ati o n  
 
Pr ot oc ol V ersi o n 1. 4 , 2 1 A pril  2 0 2 1  
1 6  
I R B-5 7 0 5 6  
 
 
 
 
 
 
 
N K c ells  
N at ur al kill er c ells 
O S  
O v er all s ur vi v al  
P B  
P eri p h er al bl o o d  
P F S  
Pr o gr essi o n fr e e s ur vi v al  
P K  
p h ar m a c o ki n etics  
Q o L  
Q u alit y of lif e  
S A E  
S eri o us a d v ers e e v e nt  
S C  
S u bc ut a n e o us  
U L N  
U p p er li mit of t h e n or m al r a n g e  

Pr ot oc ol: M G T A -1 4 5 + pl eri x af or m o bili z ati o n  
 
Pr ot oc ol V ersi o n 1. 4 , 2 1 A pril  2 0 2 1  
1 7  
I R B-5 7 0 5 6  
 
1  
O BJ E C TI V E S / E N D P OI N T S  
1. 1  
P ri m a r y O bj e cti v e  
 
1.  T o ass ess t h e effic a c y of M G T A -1 4 5 i n c o m bi n ati o n wit h pl eri x af or i n m o bilizi n g a d e q u at e 
n u m b er of h e m at o p oi etic st e m c ells ( H S Cs) i n p ati e nts wit h m ulti pl e m y el o m a ( M M) i n 
pr e p ar ati o n f or a ut ol o g o us st e m c ell tr a ns pl a nt ati o n  ( A S C T). 
1. 2  
P ri m a r y E n d p oi nt :  
1.  T o ass ess t h e r at e of s ucc essf ul c oll e cti o n of ≥2 . 0 x  1 0 6  C D 3 4 +  c ells / k g i n u p t o t w o d a ys of 
a p h er esis aft er M G T A -1 4 5 + pl eri x af or d osi n g  i n p ati e nts wit h M M. 
1. 3  
S e c o n d a r y O bj e cti v es   
1.  T o ass ess t h e effic a c y of M G T A -1 4 5 i n c o m bi n ati o n wit h pl eri x af or i n m o bil izi n g diff er e nt 
H S Cs t ar g et g o als i n p ati e nts wit h M M i n pr e p ar ati o n f or A S C T . 
 
2.  T o ass ess t h e s af et y a n d t ol er a bilit y of M G T A -1 4 5 a n d  pl eri x af or f or m o bilizi n g H S Cs  i n 
p ati e nts wit h M M . 
 
3.  T o ass ess t h e r at e a n d ki n etics of e n gr aft m e nt f oll o wi n g A S C T aft er H S C m o biliz ati o n wit h 
M G T A -1 4 5 + pl eri x af or  i n p ati e nts wit h M M  u n d er g oi n g u pfr o nt A S C T.  
 
4.  T o ass ess r at e of o n g oi n g e n gr aft m e nt at d a y  2 8  a n d  1 0 0  aft er A S C T wit h  H S Cs wit h M G T A -
1 4 5 a n d pl e ri x af or i n p ati e nts wit h M M u n d er g oi n g u pfr o nt A S C T.  
 
5.  T o ass ess tr a ns pl a nt a n d dis e as e -r el at e d o utc o m es aft er m o biliz ati o n of H S Cs wit h M G T A -
1 4 5 a n d  pl eri x af or  i n p ati e nts wit h M M u n d er g oi n g u pfr o nt A S C T.  
1. 4  
S e c o n d a r y E n d p oi nt s   
1.  T o ass ess t h e r at e of s ucc essf ul c oll e cti o n of  ( a) ≥4. 0  x  1 0 6  C D 3 4 +  c ells / k g i n u p t o t w o 
a p h er esis s essi o ns aft er M G T A -1 4 5 a n d pl eri x af or d osi n g i n p ati e nts wit h M M.  ( K e y 
S e c o n d a r y E n d p oi nt)  ( b) ≥6. 0  x  1 0 6  C D 3 4 +  c ells / k g wit h u p t o t w o a p h er esis s essi o ns aft er 
M G T A -1 4 5 a n d pl e ri x af or d osi n g i n p ati e nts wit h M M.  ( c) ≥2. 0  x  1 0 6  C D 3 4 +  a n d 
≥4. 0  x  1 0 6  C D 3 4 +  c ells / k g wit h o n e a p h er esis s essi o n aft er M G T A -1 4 5 a n d pl eri x af or d osi n g . 
 
2.  T o ass ess i nf usi o n r el at e d t o xiciti es wit h M G T A -1 4 5 a n d a d v ers e e v e nts wit h m o biliz ati o n 
of H S Cs wit h M G T A -1 4 5 a n d pl eri x af or.  
 
3.  T o ass ess ti m e t o n e utr o p hil ( d efi n e d as t h e first d a y of A N C  ≥0. 5  x  1 0 9 / L f or 3  d a ys 
f oll o wi n g st e m c ell i nf usi o n) a n d pl at el et e n gr aft m e nt ( d efi n e d as first d a y of pl at el et c o u nt 
m or e t h a n or e q u al t o 2 0  x  1 0 9 / L wit h o ut tr a nsf usi o n i n t h e l ast 7 d a ys a n d wit h pl at el et 

Pr ot oc ol: M G T A -1 4 5 + pl eri x af or m o bili z ati o n  
 
Pr ot oc ol V ersi o n 1. 4 , 2 1 A pril  2 0 2 1  
1 8  
I R B-5 7 0 5 6  
 
c o u nt ≥2 0 x  1 0 9 / L o n 2  s e p ar at e, s u bs e q u e nt d a ys a n d t o als o d et er mi n e ti m e t o  first d a y of 
pl at el et c o u nt >  5 0  x  1 0 9 / L  (wit h o ut tr a nsf usi o n i n 4 8 h o urs ) i n p ati e nts u n d er g oi n g 
u pfr o nt A S C T.  
 
4.  T o ass ess r at es of  o n g oi n g s ucc essf ul e n gr aft m e nt at 3 0 - a n d 1 0 0 -d a ys p ost -tr a ns pl a nt aft er 
s ucc essf ul pri m ar y e n gr aft m e nt i n p ati e nts u n d er g oi n g u pfr o nt A S C T.  
 
5.  T o ass ess r at e of tr a ns pl a nt r el at e d m ort alit y, n o n -r el a ps e m ort alit y, pr o gr essi o n fr e e 
s ur vi v al a n d o v er all s ur v i v al at 1 0 0 d a ys i n p ati e nts u n d er g oi n g u pfr o nt A S C T. 
1. 5  
E x pl o r at o r y O bj e cti v es  
1.  Ass ess t h e c o m p ositi o n of t h e a p h er esis pr o d uct wit h m o biliz ati o n wit h M G T A -1 4 5 a n d 
pl eri x af or i n p ati e nts wit h M M . 
 
2.  Ass ess pr olif er ati o n a n d diff er e nti ati o n p ot e nti al of H S Cs c oll e ct e d wit h M G T A -1 4 5 a n d 
pl eri x af or vi a c ol o n y -f or mi n g u nit ass a ys. 
 
3.  T o ass ess i m m u n ol o gic al r e c o v er y i n p ati e nts wit h M M u n d er g oi n g u pfr o nt tr a ns pl a nt ati o n 
aft er i nf usi o n of H S Cs c oll e ct e d wit h M G T A -1 4 5 a n d pl eri x af or m o biliz ati o n.  
 
4.  T o ass ess p ati e nt r e p ort e d s y m pt o ms wit h st e m c ell m o biliz ati o n  wit h M G T A -1 4 5 a n d 
pl eri x af or i n p ati e nts wit h M M.  
1. 6  
E x pl o r at o r y E n d p oi nt s  
1.   T o a ss ess t h e c o m p ositi o n of t h e a p h er esis pr o d uct o bt ai n e d aft er M G T A -1 4 5 a n d pl eri x af or 
m o biliz ati o n 
t hr o u g h 
e n u m er ati o n 
C D 3 4+ C D 9 0 + C D 4 5 R A - c ells i n t h e 
pr o d uct 
a n d 
i m m u n ol o gic al c h ar a ct eriz ati o n of t h e T c ell s u bs ets, B c ell s u bs ets, N K c ells, n e utr o p hils, a n d 
m y el oi d / m o n oc ytic c ells i n t h e pr o d uct usi n g fl o w c yt o m etr y a n d C y T O F ( C yt o m etr y b y 
Ti m e of Fli g ht) .  
 
2.  Ass ess pr olif er ati o n a n d diff er e nti ati o n p ot e nti al of H S Cs c oll e ct e d wit h M G T A -1 4 5 a n d 
pl eri x af or vi a c ol o n y -f or mi n g u nit ass a ys fr o m t h e a p h er esis a ut o gr aft. 
 
3.  T o a ss ess i m m u n ol o gic al c h ar a ct eriz ati o n of w hit e c ell p o p ul ati o n s u bs ets (T c ell s u bs ets, B 
c ell s u bs ets, N K c ells, n e utr o p hils, a n d m y el oi d / m o n oc ytic c ells)  i n p ati e nt’s p eri p h er al 
bl o o d at d a y 2 8 usi n g fl o w c yt o m etr y a n d at d a y 1 0 0 usi n g fl o w c yt o m etr y a n d m ass 
c yt o m etr y / C y T O F  a n d ass ess i m m u n ol o gic al c h ar act eriz ati o n of 
w hit e c ell p o p ul ati o n 
s u bs ets i n t h e b o n e m arr o w micr o e n vir o n m e nt at d a y 1 0 0 f oll o wi n g tr a ns pl a nt.  
 
4.  T o ass ess p ati e nt r e p ort e d s y m pt o ms, usi n g it e ms f or i nf usi o n r e a cti o n a n d p ai n a d a pt e d 
fr o m t h e p ati e nt r e p ort e d o utc o m es c o m m o n t er mi n ol o g y crit eri a f or a d v ers e e v e nts 
( P R O C T C A E) as w ell as t h e bri ef p ai n i n v e nt or y t o ol f oll o wi n g pl eri x af or a n d M G T A -1 4 5 
i nf usi o n. ( Q u esti o n n air e i n A p p e n di x B ) 

Pr ot oc ol: M G T A -1 4 5 + pl eri x af or m o bili z ati o n  
 
Pr ot oc ol V ersi o n 1. 4 , 2 1 A pril  2 0 2 1  
1 9  
I R B-5 7 0 5 6  
 
2  
B A C K G R O U N D  
2. 1  
A ut ol o g o u s  T r a n s pl a nt a n d St e m C ell M o bili z ati o n i n M ulti pl e M y el o m a 
 
2. 1. 1  M ulti pl e M y el o m a a n d R ol e of T r a n s pl a nt :  
 
A n n u all y, o v er 3 0, 0 0 0 p ati e nts ar e di a g n os e d wit h m ulti pl e m y el o m a i n t h e U nit e d St at es  ( N CI S E E R  
pr o gr a m) .1 1  A ut ol o g o us st e m c ell tr a ns pl a nt  ( A S C T) f or t h e tr e at m e nt of m y el o m a w as first d escri b e d 
i n 1 9 8 0s.1 2  S u bs e q u e nt st u di es, i ncl u di n g l ar g e r a n d o miz e d cli nic al tri als d e m o nstr at e d cl e ar 
e vi d e nc e of s ur vi v al b e n efit of tr a ns pl a nt a n d it b e c a m e a st a n d ar d of c ar e tr e at m e nt f or p ati e nts wit h 
m y el o m a.  A S C T is t y pic all y p urs u e d aft er a f e w c ycl es of i n d ucti o n t h er a p y. It i n v ol v es a d mi nistr ati o n 
of hi g h -d os e m el p h al a n c h e m ot h er a p y f oll o w e d b y r esc u e of t h e b o n e m arr o w wit h H S Cs  c oll e ct e d 
fr o m t h e p ati e nt pri or t o c h e m ot h er a p y a d mi nistr ati o n.1  O v er t h e p ast t w o d e c a d es, s e v er al n e w 
dr u gs h a v e b e e n a p pr o v e d f or t h e tr e at m e nt of m ulti pl e m y el o m a  a n d h a v e r es ult e d i n dr a m atic 
i m pr o v e m e nt of  p ati e nt  o utc o m es. 1 3, 1 4  E v e n t h o u g h tr e at m e nt of m y el o m a h as e v ol v e d si g nific a ntl y 
wit h t h e a v ail a bilit y of n o v el a g e nts  i n t h e p ast t w o d e c a d es, A S C T still r e m ai ns a c or n erst o n e of 
tr e at m e nt i n eli gi bl e p ati e nts.1 5   
 
T h e I F M -2 0 0 9 r a n d o miz e d cli ni c al tri al e v al u at e d o utc o m es of p ati e nts wit h e arl y vs. d el a y e d 
tr a ns pl a nt f oll o wi n g i n d ucti o n c h e m ot h er a p y wit h b ort e z o mi b, l e n ali d o mi d e a n d d e x a m et h as o n e. 
E arl y tr a ns pl a nt w as ass oci at e d wit h i m pr o v e d pr o gr essi o n fr e e s ur vi v a l ( P F S) a n d hi g h er mi ni m al 
r esi d u al dis e as e n e g ati vit y c o m p ar e d wit h  d el a y e d A S C T  at t h e ti m e of first r el a ps e . 1, 1 6  O v er all 
s ur vi v al o utc o m es w er e si mil ar i n p ati e nts pr oc e e di n g wit h e arl y tr a ns pl a nt vs. p ati e nts d el a y i n g 
tr a ns pl a nt u ntil t h e  ti m e of first r el a ps e. Si mil arl y, t h e  F O R T E tri al c o m p are d  p ati e nts pr oc e e di n g  
wit h e arl y A S C T vs. n ot aft er  in d ucti o n wit h c arfil z o mi b, l e n ali d o mi d e a n d d e x a m et h as o n e . I nt eri m 
r es ults s h o w t h at p ati e nts wit h hi g h -ris k m y el o m a u n d er g oi n g tr a ns pl a nt  h a d hi g h er p ersist e nt M R D 
n e g ati vit y r at es a n d l o w er ris k of r el a ps e. 1 7  F oll o w -u p f or s ur vi v al o utc o m es is still o n g oi n g.  A m et a -
a n al ysis of r a n d o miz e d cli nic al tri als fr o m 2 0 0 0 -2 0 1 7 s h o w e d t h at hi g h -d os e t h er a p y / A S C T w as 
ass oci at e d wit h s u p eri or P F S a n d mi ni m al t o xic eff e cts c o m p ar e d t o st a n d ar d d os e t h er a p y. 1 8  
T h er ef or e, all eli gi bl e p ati e nts wit h m ulti pl e m y el o m a s h o ul d b e c o nsi d er e d f or tr a ns pl a nt. E arl y 
tr a ns pl a nt is pr ef err e d, es p e ci all y i n i nt er m e di at e or hi g h-ris k p ati e nts. St a n d ar d ris k p ati e nts m a y 
c h o os e  t o d el a y tr a ns pl a nt u ntil first-r el a ps e. 1 5  I n s uc h p ati e nts, st e m c ell c oll e cti o n a n d st or a g e is 
r e c o m m e n d e d aft er a f e w c ycl es of i n d ucti o n t h er a p y , as l o n g-t er m l e n ali d o mi d e m ai nt e n a nc e 
t h er a p y c a n i m p air f ut ur e st e m c ell c oll e cti o n. 1 5, 1 9   
 
2. 1. 2  St e m C ell M o bili z ati o n  f o r A S C T:  
 
C oll e cti o n of  C D 3 4 + H S Cs t o r esc u e t h e b o n e m arr o w fr o m eff e cts of hi g h -d os e c h e m ot h er a p y i n A S C T 
is m ost c o m m o nl y d o n e t hr o u g h m o bili z ati o n a n d c oll e cti o n of st e m c ells fr o m t h e p eri p h er al bl o o d. 
A mi ni m u m of 2  x  1 0 6  C D 3 4 +  c ells / k g ar e r e c o m m e n d e d f or tr a ns pl a nt, a n d ma n y i nstit uti o n al 
pr ot oc ols c oll e ct u p t o 4  x  1 0 6  C D 3 4 +  c ells / k g. 2 0  At b as eli n e, o nl y a s m all n u m b er of H S Cs, i d e ntifi e d 
b y pr es e nc e of C D 3 4 p ositi vit y  o n s urf a c e, ar e circ ul ati n g i n p eri p h er al bl o o d. B ef or e st e m c ells c a n 
b e c oll e ct e d, sti m ul ati o n  a n d m o biliz ati o n of st e m c ells fr o m t h e b o n e m arr o w is r e q uir e d. It w as 
i niti all y a c hi e v e d wit h 
m y el os u p pr essi v e c h e m ot h er a p y li k e c ycl o p h os p h a mi d e a n d et o p osi d e, 
w h er e  r e b o u n d of t h e m arr o w fr o m t h e m y el os u p pr essi v e  eff e cts of c h e m ot h er a p y w as ass oci at e d  
wit h i ncr e as e d H S Cs i n t h e p eri p h er al  bl o o d. 2 0  T h e i ntr o d u cti o n of G -C S F f urt h er i ncr e as e d t h e H S Cs 
i n t h e circ ul ati o n  aft er m y el o s u p pr essi v e t h er a p y  a n d s h ort e n e d t h e ti m e n e e d e d f or a c hi e vi n g 
a d e q u at e st e m c ell c o u nts i n t h e bl o o d. G -C S F w as als o e x pl or e d as a si n gl e a g e nt f or H S C c oll e cti o n. 

Pr ot oc ol: M G T A -1 4 5 + pl eri x af or m o bili z ati o n  
 
Pr ot oc ol V ersi o n 1. 4 , 2 1 A pril  2 0 2 1  
2 0  
I R B-5 7 0 5 6  
 
H o w e v er, f ail ur e r at es wit h G -C S F al o n e w er e as hi g h as 4 0 -5 0 %. 2 1  Pl eri x af or, a  C X C R -4 i n hi bit or w as 
st udi e d i n a d diti o n t o G -C S F , wit h e xc ell e nt o utc o m es  f or m o biliz ati o n.5  It h as n o w b e c o m e a st an d ar d 
of c ar e r e gi m e n. 3, 5, 2 0  Pr ot oc ols wit h G -C S F a n d pl eri x af or m a y i ncl u d e us e of pl eri x af or o n d a y 4 of G-
C S F i n all p ati e nts or us e of pl eri x af or i n a ris k-a d a pt e d m a n n er b as e d o n p eri p h er al bl o o d C D 3 4 
c o u nt aft er 4 d a ys of G -C S F. 2 2, 2 3  A li mit ati o n i n t h e us e of pl eri x af or is t h e n e e d t o c o m bi n e it wit h 
s e v er al d a ys of G -C S F i nj e cti o ns. Pl eri x af or al o n e h as b e e n st u di e d f or st e m c ell m o biliz ati o n i n 
h e alt h y v ol u nt e ers a n d b y its elf h as li mit e d f e asi bilit y f or a r a pi d c oll e cti o n.2 4  Gi v e n  t h e l a c k of r o b ust 
r es p o ns e wit h si n gl e a g e nt pl eri x af or i n h e alt h y d o n ors, it h as n ot b e e n e v al u at e d i n l ar g e st u di es f or 
st e m c ell m o biliz ati o n i n p ati e nts wit h h e m at ol o gic m ali g n a nci es u n d er g oi n g A S C T.  
T h er ef or e, t he t w o c o m m o n str at e gi es t o a c hi e v e H S C c oll e cti o n  i n t h e c urr e nt er a  ar e  c h e m ot h er a p y -
b as e d m o biliz ati o n  (t ypi c all y c ycl o p h os p h a mi d e)  al o n g wit h G -C S F or G -C S F m o biliz ati o n wit h 
pl eri x af or, i n all p ati e nts or i n a ris k-a d a pti v e al g orit h m .2 0  W hil e t h es e m et h o ds ar e effic a ci o us, t h e 
pr oc ess c a n b e l o n g as s h o w n i n 
Fi g u r e 1  a n d c a n b e ass oci at e d 
wit h a d v ers e eff e cts. 
C ycl o p h os p h a mi d e m o biliz ati o n c a n t a k e t w o t o t hr e e w e e ks a n d r es ult i n c o m plic ati o ns li k e 
n e utr o p e nic f e v er a n d si g nific a nt c yt o p e ni as. 2 -4  G -C S F a n d pl eri x af or m o biliz ati o n r e q uir es u p t o 4 
d a ys of G -C S F i nj e cti o ns b ef or e m o biliz ati o n c a n b e gi n ( Fi gu r e 1) . I nj e cti o ns ar e c o nti n u e d t hr o u g h 
c oll e cti o n, w hi c h c a n b e as l o n g as 4 s essi o ns, t h er ef or e r e q uiri n g 5 -8 d a ys f or c oll e ct i o n.5  G -C S F m a y 
r es ult i n si g nific a nt b o n e p ai n , f ati g u e, h e a d a c h e, a n d f e v er. R ar e s eri o us a d v ers e eff e cts wit h G-C S F 
i ncl ud e s pl e nic r u pt ur e a n d p ul m o n ar y t o xicit y.  
 
2. 1. 3  R ati o n al e f o r p r o p o s e d st u d y:  
 
T h e o pti m al st e m c ell 
m o biliz ati o n a n d c oll e cti o n m et h o d s h o ul d r es ult i n r a pi d a n d r eli a bl e 
c oll e cti o n of a d e q u at e C D 3 4 + H S Cs wit h mi ni m al a d v ers e eff e cts. T h er e is a n e e d f or i n n o v ati o n t o 
r e d uc e t h e ti m e t a k e n f or st e m c ell m o biliz ati o n a n d f urt h er d e cr e as e t h e a d v ers e eff e cts ass oci at e d 
wit h st e m c ell c oll e cti o n i n p ati e nts wit h m ulti pl e m y el o m a. T his will b e b e n efici al t o p ati e nts a n d 
als o i m pr o v e h e alt hc ar e r es o urc e utiliz ati o n . W e a r e p r o p o si n g a st u d y of  a n o v el a g e nt f o r st e m 
c ell m o bili z ati o n, M G T A -1 4 5 i n c o m bi n ati o n wit h pl e ri x af o r f o r r a pi d, s a m e d a y st e m c ell 
m o bili z ati o n i n p ati e nt s wit h m ulti pl e m y el o m a ( Fi g u r e 1).  
 
 

Pr ot oc ol: M G T A -1 4 5 + pl eri x af or m o bili z ati o n  
 
Pr ot oc ol V ersi o n 1. 4 , 2 1 A pril  2 0 2 1  
2 1  
I R B-5 7 0 5 6  
 
2. 2  
M G T A -1 4 5  
 
M G T A -1 4 5 ( als o k n o w n as Gr o -b et a tr u nc at e; G R O β T) is a s y nt h eticall y m a n uf a ct ur e d n at ur all y 
occ urri n g  f o ur a mi n o a ci d tr u nc at e d pr ot ei n v ari a nt of C X C L 2, a n d h as a 6 9 a mi n o a ci d pr ot ei n 
pri m ar y str uct ur e  as list e d b el o w . 
T E L R C Q C L Q T L Q GI H L K NI Q S V K V K S P G P H C A Q T E VI A T L K N G Q K A C L N P A S P M V K KII E K M L K N G K S N  
 
M G T A -1 4 5 h as t w o dis ulfi d e bri d g es: C 5 t o C 3 1 a n d C 7 t o C 4 7, a n d is c o m pris e d of a n al p h a h eli x a n d 
a b et a -s h e et.  
 
C X C L 2 pr ef er e nti all y bi n d s t o th e c ell -s urf a c e c h e m o ki n e r e c e pt or C X C R 2 , w hi c h is e x pr ess e d 
pr e d o mi n a ntl y o n n e utr o p hils a n d e n d ot h eli al c ells.  G R O β T bi n ds t o t h e C X C R 2 r e c e pt or a n d wit h 
gr e at er p ot e nc y t h a n f ull -l e n gt h G R O β.2 5   
 
P h a r m a c e uti c al P r o p e rti e s:  M G T A -1 4 5 bi n ds t o t h e c ell -s urf a c e c h e m o ki n e r e c e pt or C X C R 2, w hic h 
is e x pr ess e d pr e d o mi n a ntl y o n n e utr o p hils a n d e n d ot h eli al c ells t o i n d uc e m o biliz ati o n of H S Cs i nt o 
t h e p eri p h er al bl o o d. H S C m o biliz ati o n is d e p e n d e nt o n t h e e q uili bri u m of M M P-9 a n d tiss u e 
i n hi bit or of m et all o pr ot ei n as e-1 ( TI M P -1) s hifti n g t o w ar ds M M P -9 f oll o wi n g M M P -9 r el e as e b y 
n e utr o p hils u p o n C X C R 2 bi n di n g. St u di es s h o w e d t h at t h e a d diti o n of A M D 3 1 0 0 ( pl eri x af or) i n hi bits 
cr oss -c o m m u nic ati o n b et w e e n C X C R 4 a n d C X C R 2 r e c e pt ors w hic h n or m all y f u ncti o n t o di mi nis h t h e 
C X C R 2 r es p o ns e. T h e a d diti o n of pl eri x af or h el ps e n h a nc e t h e r el e as e of M M P -9, l e a d i n g t o i ncr e as e d 
m o bili z ati o n of h e m at o p oi etic c ells.  
 
M G T A -1 4 5 P h ysi c al  a n d C h e mi c al P r o p e rti e s  
 
M ol e c ul ar F or m ul a ( n et)  
C 3 2 5 H 5 5 7 N 9 7 O 9 5 S 6  
M ol e c ul ar M ass ( a v er a g e)  
7 5 3 6. 0  
A p p e ar a nc e of S ol uti o n  
Cl e ar, c ol orl ess s ol uti o n, ess e nti all y fr e e of visi bl e p articl es  
S ol u bilit y  
> 1 0 0 m g / m L  
 
F o r m ul ati o n:  M G T A -1 4 5 is f or m ul at e d at 2 0 m g / m L i n 2 0 m M s o di u m a c et at e, 1 5 0 m M s o di u m 
c hl ori d e, 0. 0 2 % ( w / v) P ol ys or b at e 8 0 at p H 4. 0. M G T A -1 4 5 is s u p pli e d i n a si n gl e -d os e 2 m L T y p e I 
a m b er gl ass vi al. E a c h vi al c o nt ai ns n ot l ess t h a n 1 m L.  
 
St o r a g e C o n diti o n s  
Vi als  of M G T A -1 4 5 s h o ul d b e st or e d at -2 0° C ± 5° C i n t h e c art o n t o pr ot e ct fr o m li g ht.  
I n st r u cti o n s f o r U s e a n d H a n dli n g  
M G T A -1 4 5 (i nj e cti o n) is a st eril e s ol uti o n f or I V us e s u p pli e d as a si n gl e -d os e vi al p a c k a g e d i n a 
c art o n.  
Vi als of M G T A -1 4 5 s h o ul d b e t h a w e d o v er ni g ht at 4° C pri or t o us e a n d ar e st a bl e at 4° C f or u p t o 3 0 
d a ys. O nc e t h a w e d, M G T A -1 4 5 s h o ul d n ot b e r efr oz e n.  
 
M G T A -1 4 5 s h o ul d b e dil ut e d i n 0. 9 % s ali n e i n a cc or d a nc e wit h t h e st u d y pr ot oc ol a n d p h ar m a c y 
m a n u al. S ol uti o ns of M G T A -1 4 5 ( 0. 0 3 6 t o 1. 4 4 m g / m L) pr e p ar e d i n 0. 9 % s ali n e m a y b e st or e d f or n o 
m or e t h a n 8 h o urs at r o o m t e m p er at ur e pri or t o a d mi nistr ati o n.  
 

Pr ot oc ol: M G T A -1 4 5 + pl eri x af or m o bili z ati o n  
 
Pr ot oc ol V ersi o n 1. 4 , 2 1 A pril  2 0 2 1  
2 2  
I R B-5 7 0 5 6  
 
M G T A -1 4 5 s h o ul d b e a d mi nist er e d as a n I V i nf usi o n o v er 3 -1 0 mi n ut es , as cli nic all y i n dic at e d. 
 
P r e cli ni c al d at a:  Pr e cli nic al st u di es d e m o nstr at e d t h at G R O β T  ( M G T A-1 4 5) , w h e n a d mi nist er e d 
si m ult a n e o usl y wit h pl eri x af or, l e d t o a s y n er gistic i ncr e as e i n m o biliz ati o n of hi g hl y e n gr aft a bl e 
H S Cs i n mic e. M o biliz ati o n w as r a pi d, occ urri n g wit hi n mi n ut es of a d mi nistr ati o n of G R O β T, a n d 
r o b ust, m o bilizi n g si g nific a ntl y m or e H S Cs t h a n eit h er G R O β T or pl eri x af or al o n e. 6  I n a d diti o n t o t h e 
e v al u ati o n of mic e i n t his st u d y, s af et y a n d p h ar m a c o ki n etic st u di es of M G T A -1 4 5 h a v e b e e n d o n e i n 
r ats a n d n o n-h u m a n pri m at es as d escri b e d i n t h e a cc o m p a n yi n g I n v esti g at o r’s Br o c h u r e .  
 
Cli ni c al d at a : I n a p h as e 1 st u d y6 , a d ult h e alt h y m al es r e c ei v e d esc al ati n g d os es of pl a c e b o i nj e cti o n 
f oll o w e d b y M G T A -1 4 5 f o ur w e e ks l at er.  M ost c o m m o n si d e eff e ct w as a c ut e l u m b ar b a c k p ai n, w hic h 
l ast e d 1 0 mi n ut es or l ess a n d di d n ot r e q uir e tr e at m e nt. B a c k p ai n w as s ee n is s u bj e cts w h o r e c ei v e d 
i nf usi o ns of 0. 0 9 m g / k g or hi g h er a n d w as s e e n i n 2 9. 7 % of s u bj e cts r e c ei vi n g M G T A -1 4 5  vs. 5. 3 % 
r e c ei vi n g pl a c e b o. Si n gl e a g e nt M G T A -1 4 5  r es ult e d i n C D 3 4 m o biliz ati o n at hi g h er d os es, b ut it w as 
n ot f elt t o b e s uffici e nt f or a d e q u at e st e m c ell c oll e cti o n wit h M G T A -1 4 5  us e d as a si n gl e a g e nt.  
A p h as e 1 st u d y of M G T A -1 4 5, wit h or wit h o ut pl eri x af or w as c o n d uct e d i n h e alt h y v ol u nt e ers t o 
d et er mi n e t h e s af et y a n d d os e of M G T A -1 4 5. 7  M G T A -1 4 5 w as a d mi nist er e d t o 7 9  h e alt h y v ol u nt e ers.  
T h e st u d y h a d f o ur p arts. I n P art A, s u bj e cts r e c ei v e d si n gl e d os e of M G T A -1 4 5 al o n e ( 0. 0 0 7 5 -0. 3 
m g / k g). I n P art B , s u bj e cts r e c ei v e d si n gl e d os e of M G T A-1 4 5 ( 0. 0 1 5 -0. 1 5 m g / k g) wit h pl eri x af or. I n 
P art C, s u bj e cts r e c ei v e d 2 d ail y d os es of M G T A -1 4 5 ( 0. 0 3 a n d 0. 0 7 m g / k g) a n d pl eri x af or. I n P art D, 
s u bj e cts r e c ei v e d si n gl e d os e of M G T A -1 4 5 ( 0. 0 1 5 a n d 0. 0 3 m g / k g) a n d pl eri x af or , f oll o w e d b y s a m e 
d a y a p h er esis.   M G T A -1 4 5 w as f o u n d t o h a v e a g o o d s af et y pr ofil e, wit h m ost c o m m o n si d e eff e ct  of 
tr a nsi e nt b ac k p ai n l asti n g l ess t h a n 2 0 mi n ut es i n m ost p ati e nts. I n P art  A, b a c k p ai n w as s e e n i n 
7 9 % ( 1 9 / 2 4) of v ol u nt e ers r e c ei vi n g si n gl e a g e nt M G T A -1 4 5 vs. 0 % ( 0 / 1 2) of v ol u nt e ers r e c ei vi n g 
pl eri x af or. T h er e w er e n o ot h er tr e at m e nt e m er g e nt a d v er s e eff e cts ( T E A E) i n s u bj e cts i n  P art  A 
r e c ei vi n g M G TA -1 4 5 ( 0. 0 0 7 5 t o 0. 3 m g / k g).  W h e n M G T A -1 4 5 ( 0. 0 1 5  t o 0. 1 5 m g / k g) w as c o m bi n e d 
wit h pl eri x af or i n P art -B , T E A E w er e s e e n i n 8 1 % ( 2 6 / 3 2) of s u bj e cts c o m p ar e d t o 5 8 % ( 7 / 1 2) of 
p ati e nts  r e c ei vi n g pl eri x af or al o n e. T E A E i n t h e M G T A -1 4 5 a n d pl eri x af or ar m i ncl u d e d b ac k 
p ai n / m usc ul os k el et al p ai n i n 6 2. 5 % ( n = 2 0), di arr h e a, n a us e a a n d diz zi n ess i n 1 5. 6 %  s u bj e cts e a c h 
( n = 5 e a c h), r es p e cti v el y, a b d o mi n al p ai n i n 1 2. 5 % ( n = 4), v o miti n g a n d  h e a d a c h e i n 9. 4 % ( n = 3 e a c h)  
of s u bj e cts e a c h, r es p e cti v el y a n d p ar est h esi a i n 6. 3 % of s u bj e cts . T his d at a is s h o w n i n t h e T a bl e 1  
b el o w ( s o urc e: Di Persi o et al T C T 2 0 1 9 or al pr es e nt ati o n )  

Pr ot oc ol: M G T A -1 4 5 + pl eri x af or m o bili z ati o n  
 
Pr ot oc ol V ersi o n 1. 4 , 2 1 A pril  2 0 2 1  
2 3  
I R B-5 7 0 5 6  
 
T a bl e 1 : A d v e r s e eff e ct s wit h M G T A a n d pl e ri x af o r i n a p h a s e 1 st u d y of h e alt h y v ol u nt ee r s  
 
  S o urc e: Di p ersi o et al. T C T 2 0 2 0  or al pr es e nt ati o n  
At t h e 0. 0 3 m g / k g d os e t h e m e di a n C D 3 4 + c ell c o u nt i n p eri p h er al f oll o wi n g M G T A -1 4 5 a n d 
pl eri x af or a d mi nistr ati o n w as 4 0 c ells / micr o L (r a n g e 1 8 -6 3), w hic h w as hi g h er t h a n t h at o bs er v e d 
wit h pl eri x af or al o n e ( m e di a n 2 4, r a n g e: 1 3 -7 8). T h e d os e of 0. 0 3 m g / k g w as d et er mi n e d t o b e s a f e 
a n d eff e cti v e, wit h pr e dict a bl e, d os e li n e ar p h ar m ac o ki n etics  t h at ar e n ot aff e ct e d b y t h e c o-
a d mi nistr ati o n of pl eri x af or . A d mi nistr ati o n of pl eri x af or f oll o w e d b y M G T A-1 4 5 aft er a 2 -h o ur  
i nt er v al w as ass oci at e d wit h t h e b est st e m c ell m o biliz ati o n o utc o m es, as d et er mi n e d b y p eri p h er al 
bl o o d C D 3 4 c o u nts a n d will b e t h e r e gi m e n us e d i n t his tri al.   
A st u d y of M G T A -1 4 5 i n s u bj e cts wit h r e n al i m p air m e nt is o n g oi n g. T h e pri m ar y o bj e cti v e  of t h at 
st u d y is t o est a blis h t h e eff e ct of r e n al i m p air m e nt o n M G T A-1 4 5 p h ar m a c o ki n etics. A si n gl e, 
m o n ot h er a p y d os e 0. 0 7 m g / k g w as us e d f or t his st u d y.  T w e nt y -t hr e e a d ult s u bj e cts w er e e nr oll e d 
i nt o 1 of t h e f oll o wi n g 3 gr o u ps b as e d o n e G F R, d et er mi n e d b y t h e M o dific ati o n of Di et i n R e n al 
Dis e as e St u d y ( M D R D) e q u ati o n : 1. n or m al ≥ 9 0; 2. mil d 6 0 t o 8 9; a n d 3. m o d er at e 3 0 t o 5 9 
m L / mi n / 1. 7 3 m 2 ). O v er all, t h e r es ults d e m o nstr at e t h at M G T A -1 4 5 w as w ell t ol er at e d i n s u bj e cts 
wit h n or m al ki d n e y f u ncti o n a n d i n s u bj e cts wit h r e n al i m p air m e nt. R e n al i m p air m e nt h a d n o i m p a ct 
o n  t h e P K of M G T A-1 4 5. M G T A -1 4 5 is n ot e xcr et e d i n uri n e . 
 
N o n -cli nic al 
p h a r m a c o ki n eti c s : P l e as e r ef er t o a cc o m p a n yi n g I n v esti g at or’s Br oc h ur e  f or 
p h ar m a c o ki n etic st u di es i n mic e, r ats  a n d n o n -h u m a n pri m at es.  
 
Cli ni c al p h a r m a c o ki n eti c s : I n a p h as e 1 st u d y6 , a d ult h e alt h y m al es r e c ei v e d esc al ati n g d os es of 
pl a c e b o i nj e cti o n f oll o w e d b y M G T A -1 4 5  w e e ks l at er. T h e m a xi m u m t ol er at e d d os e w as d et er mi n e d 
t o b e 0. 2 m g / k g.  
I n t h e M a g e nt a T h er a p e utics p h as e 1 st u d y of h e alt h y v ol u nt e ers, p h ar m a c o ki n etic st u di es of 
diff e r e nt d os es of M G T A-1 4 5, wit h or wit h o ut pl eri x af or w er e d o n e. M G T A -1 4 5 pl as m a dr u g 
c o nc e ntr ati o n i ncr e as e d pr o p orti o n all y wit h t h e i nf us e d d os e a n d w as n ot aff e ct e d b y pl eri x af or . T h e 
C m a x of M G T A -1 4 5 i ncr e as e d al o n g wit h d os e li n e arl y a n d A U C i ncr e as e d i n  a s u pr a pr o p orti o n al 
m a n n er i n t h e d osi n g r a n g e of 0. 0 0 7 5 m g / k g t o 0. 3 m g / k g. I n P art A, t h e m e a n C m a x of M G T A -1 4 5 
r a n g e d fr o m 3 5. 1 2 μ g / L t o 1 4 6 1. 5 5 μ g / L, a n d t h e m e a n ar e a u n d er t h e c o nc e ntr ati o n -ti m e c ur v e fr o m 
ti m e z er o t o ti m e of l ast m e as ur a bl e c o nc e ntr ati o n ( A U Cl ast) of M G T A-1 4 5 r a n g e d fr o m 2 0. 1 h* μ g / L 

Pr ot oc ol: M G T A -1 4 5 + pl eri x af or m o bili z ati o n  
 
Pr ot oc ol V ersi o n 1. 4 , 2 1 A pril  2 0 2 1  
2 4  
I R B-5 7 0 5 6  
 
t o 2 1 4 9. 9 h* μ g / L. M e a n h alf-lif e (t 1 / 2) r a n g e d fr o m 0. 5 1 t o 1. 9 7 h o urs. C o-a d mi nistr ati o n of 
pl eri x af or of 1 0 mi n ut es or 2 h o urs h a d n o i m p a ct o n t h e P K pr ofil e of M G T A -1 4 5. F oll o wi n g t h e 
a d mi nistr ati o n  of a s e c o n d d os e o n D a y 2 i n P art C, 
mi ni m al a cc u m ul ati o n 
w as o bs er v e d. 
Acc u m ul ati o n w as hi g h er wit h t h e 0. 0 7 m g / k g d os e t h a n wit h t h e 0. 0 3 m g / k g d os e. Uri n e e xcr eti o n 
of M G T A -1 4 5 w as ass ess e d fr o m 4 h o urs t o 1 6 d a ys aft er d osi n g, a n d M G T A -1 4 5 is n ot e xcr et e d i n 
uri n e.  
 
T h e i nf usi o n ti m e i n t h e st u d y of h e alt h y v ol u nt e ers 
w as s et at 1 0 
mi n ut es. S u bs e q u e nt 
p h ar m a c o ki n etic m o d eli n g d o n e b y M a g e nt a T h er a p e utics s u p p orts a n i nf usi o n ti m e of 1 t o 1 0 
mi n ut es as d e e m e d a cc e pt a bl e i n f ut ur e st u di es.  
Pl e as e r ef er t o t h e 
a cc o m p a n yi n g 
I n v esti g at o r’s 
B r o c h u r e a n d 
a d diti o n al 
P o p ul ati o n 
P h a r m ac o ki n etic A n al ysis of M G T A -1 4 5  ( D R-2 0 -0 0 2) V e rsi o n 0 0 d at e d 1 4 J ul y 2 0 2 0  f or a d diti o n al  
d et ails.  
R e g ul at o r y C o m pli a n c e:  Pl eri x af or is F D A a p pr o v e d f or st e m c ell m o biliz ati o n i n  c o m bi n ati o n wit h 
G -C S F  i n p ati e nts 
wit h m ulti pl e 
m y el o m a. 
M G T A -1 4 5 is n ot F D A a p pr o v e d. T h er ef or e, a n 
I n v esti g ati o n al N e w Dr u g a p plic ati o n (I N D) is r e q uir e d a n d will b e s u b mitt e d.  
2. 3  
R ati o n al e  
M G T A -1 4 5 ( G R O β T) a cti v at es t h e C X C R 2 p at h w a y i n n e utr o p hils. It h as s h o w n pr o misi n g a cti vit y f or 
r a pi d m o biliz ati o n of C D 3 4 + H S Cs i n pr e-cli nic al e v al u ati o n i n mic e. 6  W h e n us e d i n c o m bi n ati o n wit h 
pl eri x af or, a C X C R 4 i n hi bit or, M G T A -1 4 5 h as t h e p ot e nti al t o r a pi dl y a n d r eli a bl y m o biliz e a d e q u at e 
n u m b ers of H S Cs f or st e m c ell tr a ns pl a nt ati o n. It h as b e e n e v al u at e d wit h pl eri x af or i n a p h as e I st u d y 
of h e alt h y v ol u nt e ers , w h er e it w as f o u n d t o b e s af e a n d r es ult e d i n r a pi d m o biliz ati o n of st e m c ells 
o n t h e s a m e d a y. 7  T h es e H S Cs w er e tr a ns pl a nt e d i nt o s u bl et h all y irr a di at e d ( 2 0 0 c G y) N S G mic e at 
li mit dil uti o n ( 3 c ell d os es). E n gr aft m e nt of h u m a n C D 4 5+  ( h C D 4 5+ ) i n peri p h er al bl o o d w as 
m e as ur e d at w e e k 4 w e e ks p ost -tr a ns pl a nt b y fl o w c yt o m etr y. E n gr aft m e nt w as m or e r o b ust i n mic e 
i nj e ct e d h u m a n H S Cs o bt ai n e d fr o m M G T A-1 4 5 a n d pl eri x af or vs. t h os e o bt ai n e d wit h G -C S F al o n e. 7  
Gi v e n t h e pr o misi n g r es ults s e e n i n p r e-cli nic al m o d els a n d p h as e 1 d at a , w e e x p e ct t h at t h e 
c o m bi n ati o n of M G T A -1 4 5 a n d pl eri x af or will r es ult i n f ast, eff e cti v e a n d s af e st e m c ell m o biliz ati o n 
i n p ati e nts wit h m ulti pl e m y el o m a. M G T A -1 4 5 is a p ot e nti al alt er n ati v e t o G -C S F w h e n us e d i n 
c o m bin ati o n 
wit h pl eri x af or t o a c hi e v e s a m e d a y d osi n g a n d a p h er esis, r es ulti n g i n p ati e nt 
c o n v e ni e nc e, i m pr o v e d h e alt h c ar e r es o urc e utiliz ati o n a n d p ot e nti all y f e w er a d v ers e eff e cts t h a n 
ass oci at e d wit h m ulti -d a y G -C S F  b as e d r e gi m e ns .  
T h er ef or e, t o  e v al u at e t h e effic a c y a n d s af et y of t his n o v el m o biliz ati o n r e gi m e n, w e ar e p r o p osi n g a 
p h a s e II , p r o of of c o n c e pt st u d y of M G T A -1 4 5 a n d pl e ri x af o r i n p ati e nt s wit h m y el o m a . P ati e nts 
will r e c ei v e pl eri x af or at 0. 2 4 m g / k g  ( 0. 1 6 m g / k g f or cr e ati ni n e cl e ar a nc e <  5 0 ml / mi n)  S C f oll o w e d 
b y M G T A -1 4 5 at 0. 0 3 m g / k g I V aft er a t w o-h o ur  i nt er v al. T his will b e f oll o w e d b y s a m e d a y a p h er esis. 
D osi n g m a y b e r e p e at e d o n d a y 2 b as e d o n C D 3 4 + yi el d of a p h er esis pr o d uct. T his st u d y will als o 
i ncl u d e c orrel ati v e st u di es t o u n d erst a n d t h e gr aft c o m p ositi o n, pr olif er ati o n of H S Cs i n vitr o wit h  
c ol o n y f or mi n g u nit  (C F U ) ass a ys, e n gr aft m e nt ki n etics a n d i m m u n e r e c o nstit uti o n f oll o wi n g 
i nf usi o n of st e m c ells m o biliz e d wit h t his n o v el r e gi m e n.   
 

Pr ot oc ol: M G T A -1 4 5 + pl eri x af or m o bili z ati o n  
 
Pr ot oc ol V ersi o n 1. 4 , 2 1 A pril  2 0 2 1  
2 5  
I R B-5 7 0 5 6  
 
2. 4  
St u d y D esi g n  
•  
T h is is a p h as e II,  o p e n l a b el , pr o of of c o nc e pt st u d y t o e v al u at e t h e s af et y a n d effic a c y 
of H S C m o biliz ati o n wit h a n o v el dr u g, M G T A -1 4 5 at a fi x e d d os e l e v el i n c o m bi n ati o n 
wit h pl eri x af or i n p ati e nts wit h M M f or st e m c ell m o biliz ati o n i n pr e p ar ati o n of 
a ut ol o g o us st e m c ell tr a ns pl a nt.  
•  
P ri m ar y p ur p os e of  t h e st u d y i nt er v e nti o n : I nt er v e nti o n al ( H S C m o biliz ati o n) 
•  
Si n gl e c o h ort, si n gl e c e nt er, o p e n l a b el st u d y wit h o n e i nt er v e nti o n ar m  
•  
R a n d o miz ati o n: N o n -r a n d o miz e d 
•  
P ri m ar y o utc o m e: Effic a c y  
•  
S e c o n d ar y O utc o m es: Effic a c y, s af et y, e n gr aft m e nt, s ur vi v al  
 
2. 5  
C o r r el ati v e St u di es B a c k g r o u n d  
 
C o r r el ati v e St u d y # 1: A s s es s m e nt of g r aft c o m p o siti o n of st e m c ell p o p ul ati o n s a n d ot h e r 
w hit e bl o o d c ell s u b s et s b y fl o w c yt o m et r y  a n d C y T O F . 
 
Ai m:  T o ass ess  t h e c o m p ositi o n of t h e a p h er esis pr o d uct o bt ai n e d  aft er M G T A -1 4 5 a n d pl eri x af or 
m o bili z ati o n t hr o u g h e n u m er ati o n C D 3 4
+ C D 9 0 + C D 4 5 R A - c ells i n t h e pr o d uct a n d i m m u n ol o gic al 
c h ar a ct eriz ati o n of t h e T c ell s u bs ets, B c ell s u bs ets, N K c ells, n e utr o p hils, a n d m y el oi d / m o n oc ytic 
c ells i n t h e pr o d uct usi n g  a c o m bi n ati o n of  fl o w c yt o m etr y a n d C y T O F .  
 
C D 9 0 + C D 3 4 + st e m c ells h a v e b e e n s h o w n t o b e tr u e s elf -r e n e wi n g pl uri p ot e nt H S Cs a m o n g t h e 
C D 3 4 + c ells, 
w h er e as C D 9 0( -) C D 3 4 + c ells h a v e b e e n s h o w n t o b e diff er e nti at e d c o m mitt e d 
pr o g e nit or c ells .2 6  W hil e d at a is li mit e d, t h er e is e vi d e nc e t h at C D 9 0 + C D 3 4 + c ells i n t h e gr aft dri v e 
t h e e n gr aft m e nt effici e nc y of st e m c ell gr afts. 
M G T A-1 4 5 
w as ass oci at e d 
wit h pr ef er e nti al 
m o bili z ati o n of C D 9 0 + C D 3 4 + st e m c ells i n pr e -cli nic al m o d els vs. gr afts m o biliz e d b y G -C S F.  6, 7   I n 
t h e p h as e 1 st u d y of M G T A-1 4 5 wit h pl eri x af or, t h e m e di a n C D 9 0 + C D 3 4 + st e m c ell pr o p orti o n w as 
3 3 %, hi g h er t h a n t h at o bs er v e d i n a s m all s u bs et of p ati e nts ( n = 2) m o biliz e d wit h G -C S F. T his 
c orr el ati v e st u d y will pr o vi d e  d at a o n t h e C D 9 0 + C D 3 4 + c ells fr a cti o n fr o m a p h er esis pr o d ucts 
c oll e ct e d wit h t his n o v el m o biliz ati o n r e gi m e n  (T a bl e 2 ). 
 
S e c o n dl y, w e will e v al u at e gr aft c o m p ositi o n of T, B a n d N K c ells a n d t h eir s u bs ets usi n g v ali d at e d 
fl o w c yt o m etr y/ C y T O F  p a n els  (T a bl e 3 ). It h as b e e n s h o w n t h at hi g h er l y m p h oc yt e s u bs et 
pr o p orti o ns i n t h e st e m c ell gr aft ar e ass oci at e d  wit h b ett er p ost -tr a ns pl a nt i m m u n e r e c o nstit uti o n 
a n d s ur vi v al.  
Ass ess m e nt of gr aft c o m p ositi o n  will b e d o n e b y fl o w c yt o m etr y i n t h e l a b of Dr. J u dit h S hiz ur u  at 
St a nf or d U ni v ersit y  a n d b y C y T O F i n t h e H u m a n Im m u n e M o nit ori n g C e nt er at St a nf or d U ni v ersit y . 
C orr el ati o n of fr a cti o n of C D 9 0 + C D 3 4 + c ells wit h e n gr aft m e nt ki n etics will b e e x pl or e d.  T his d at a will 
b e c o m p ar e d wit h d at a fr o m  a c o nt e m p or a n e o us c o h ort of  p ati e nts  wit h 
m ulti pl e 
m y el o m a  
u n d er g oi n g H S C c oll e cti o n wit h c urr e ntl y a p pr o v e d r e gi m e ns . S a m pl es will b e o bt ai n e d u n d er a 
s e p ar at e I R B a p pr o v e d bi or e p osit or y pr ot oc ol.  
T hir dl y, w e will e v al u at e gr aft c o nt a mi n ati o n usi n g n e xt g e n er ati o n fl o w c yt o m etr y ass a y mi ni m al 
r esi d u al dis e as e n e g a ti vit y t esti n g. M R D N G F will b e b as e d o n t h e E ur o -Fl o w st a n d ar diz e d al g orit h m 
a n d will b e d o n e at M a y o Cli nic. 2 7 -2 9  T h e s e nsiti vit y of t h e ass a y is 1 i n 1 x 1 0 5  t o 1 x 1 06  c ells. It is a 2 
t u b e, 8 c ol or ass a y a n d t h e f oll o wi n g p a n els ar e us e d:  

Pr ot oc ol: M G T A -1 4 5 + pl eri x af or m o bili z ati o n  
 
Pr ot oc ol V ersi o n 1. 4 , 2 1 A pril  2 0 2 1  
2 6  
I R B-5 7 0 5 6  
 
 
T u b e # 1: C D 3 8 / C D 5 6 / C D 4 5 / C D 1 9 / C D 1 1 7 / C D 8 1 / C D 1 3 8 /  C D 2 7   
T u b e # 2: C D 3 8 / C D 5 6 / C D 4 5 / C D 1 9 / K a p p a / L a m b d a / C D 1 3 8 / C D 2 7   
 
 
T a bl e 2 : Fl o w C yt o m et r y P a n el wit h A nti b o di es f o r H S C a n d p r o g e nit o r c ell st ai ni n g  
Fl u or oc hr o m e  
T ar g et  
Cl o n e  
V e n d or  
C at al o g N o.  
Fi n al 
Dil uti o n  
 
µ L A b / 
1 0 0 µ L 
c oc kt ail  
FI T C  
h C D 4 5 R A  
HI 1 0 0  
e Bi osci e nc es  
1 1 -0 4 5 8 -4 2  
1: 1 0 0  
1  
P E  
h C D 9 0  
( 2 0 g / m L)  
P R 1 3  
P a cific G M P  
l ot # 1 0 2-
1 4 P / N 7 0 -
0 0 3 1  
1: 1 0  
1 0  
A P C -C y 7  
h C D 1 2 3  
6 H 6  
e Bi osci e nc es  
4 7 -1 2 3 9 -4 2  
1: 1 0 0  
1  
A P C  
h C D 3 4  
5 6 1  
Bi o L e g e n d  
3 4 3 6 0 8  
1: 1 0 0  
1  
P E -C y 7  
h C D 3 8  
HI T 2  
e Bi osci e nc es  
2 5 -0 3 8 9 -4 2  
1: 1 0 0  
1  
B V 5 1 0  
h C D 1 0  
HI 1 0 a  
Bi o L e g e n d  
3 1 2 2 1 9  
1: 1 0 0  
1  
P a c Bl u e  
h C D 1 1 7  
1 0 4 D 2  
e Bi osci e nc es  
4 8 -1 1 7 8 -4 2  
1: 1 0 0  
1  
P er C P -C y 5. 5  
h C D 1 9  
HI B 1 9  
B D Bi osci e nc es  
5 6 1 2 9 5  
1: 1 0 0  
1  
 
T a bl e 3 : C y T O F P a n el wit h A nti b o di es f o r p e ri p h e r al bl o o d m o n o n u cl e a r c ell 
i m m u n o p h e n ot y pi n g  
P B M C p h e n ot y pi n g  
M et al l a b el  
S p e cifi cit y  
Cl o n e  
C o nj u g at e d b y  
1 0 2 -1 1 0 P d  
  
  
  
1 1 3I n  
C D 5 7  
H C D 5 7, Bi o L e g e n d  
HI M C  
1 1 5I n  
li v e / d e a d 
  
  
1 3 9 L a  
  
  
  
1 4 1 P r  
  
  
  

Pr ot oc ol: M G T A -1 4 5 + pl eri x af or m o bili z ati o n  
 
Pr ot oc ol V ersi o n 1. 4 , 2 1 A pril  2 0 2 1  
2 7  
I R B-5 7 0 5 6  
 
1 4 2 N d  
C D 1 9  
SJ 2 5 C 1, S o ut h er n Bi o T ec h  
HI M C  
1 4 3 N d  
C D 4  
S K 3, Bi o L e g e n d  
HI M C  
1 4 4 N d  
C D 8  
S K 1, Bi o L e g e n d  
HI M C  
1 4 5 N d  
C D 3 9 B  
Bi o L e g e n d  
HI M C  
1 4 6 N d  
I g D 
I A 6-2, Bi o L e g e n d  
HI M C  
1 4 7 S m  
C D 8 5j  
2 9 2 3 1 9, R & D S yst e ms  
HI M C  
1 4 8 N d  
C D 1 1c  
B u 1 5, Bi o L e g e n d  
HI M C  
1 4 9 S m  
C D 1 6  
3 G 8, Bi o L e g e n d  
HI M C  
1 5 0 N d  
C D 3  
U C H T 1, B D  
HI M C  
1 5 1 E u  
C D 3 8  
H B -7, B D  
HI M C  
1 5 2 S m  
C D 2 7  
L 1 2 8, B D  
HI M C  
1 5 3 E u  
C D 1 1 b  
I C R F 4 4, Bi o L e g e n d 
HI M C  
1 5 4 S m  
C D 1 4  
M 5 E 2, Bi o L e g e n d  
HI M C  
1 5 5 G d  
C C R 6  
1 1 A 9, B D or G 0 3 4 E 3, Bi o L e g e n d  
HI M C  
1 5 6 G d  
C D 9 4  
H P -3 D 9, B D  
HI M C  
1 5 7 G d  
C D 8 6  
I T 2. 2, Bi o L e g e n d 
HI M C  
1 5 8 G d  
C X C R 5  
R F 8 B 2, B D  
HI M C  
1 5 9 T b  
C X C R 3  
G 0 2 5 H 7, Bi ol e g e n d  
HI M C  
1 6 0 G d  
C C R 7  
1 5 0 5 0 3, R & D S yst e ms  
HI M C  
1 6 1 D y  
  
  
  
1 6 2 D y  
C D 4 5 R A  
HI 1 0 0, Bi o L e g e n d  
HI M C  
1 6 3 D y  
  
  
  
1 6 4 D y  
C D 2 0  
2 H 7, Bi o L e g e n d  
HI M C  
1 6 5 H o  
C D 1 2 7  
A 0 1 9 D 5, Bi o L e g e n d  
HI M C  
1 6 6 E r  
C D 3 3  
P 6 7. 8, B D  
HI M C  

Pr ot oc ol: M G T A -1 4 5 + pl eri x af or m o bili z ati o n  
 
Pr ot oc ol V ersi o n 1. 4 , 2 1 A pril  2 0 2 1  
2 8  
I R B-5 7 0 5 6  
 
1 6 7 E r  
C D 2 8  
L 2 9 3, B D  
HI M C  
1 6 8 E r  
C D 2 4  
M L 5, Bi o L e g e n d  
HI M C  
1 6 9 T m  
I C O S 
D X 2 9, B D  
HI M C  
1 7 0 E r  
C D 1 6 1  
D X 1 2, B D  
HI M C  
1 7 1 Y b  
T C R g d  
B 1, Bi o L e g e n d  
HI M C  
1 7 2 Y b  
P D -1  
E H 1 2. 1, B D  
HI M C  
1 7 3 Y b  
C D 1 2 3  
9 F 5, B D  
HI M C  
1 7 4 Y b  
C D 5 6  
N C A M 1 6. 2, B D  
HI M C  
1 7 5 L u  
H L A -D R  
G 4 6 -6, B D  
HI M C  
1 7 6 Y b  
C D 2 5  
M -A 2 5 1, B D  
HI M C  
 
C o r r el ati v e St u d y # 2: A s s es s p r olif e r ati o n a n d diff e r e nti ati o n p ot e nti al of H S C s c oll e ct e d wit h 
M G T A -1 4 5 a n d pl e ri x af o r vi a c ol o n y -f o r mi n g u nit ( C F U) a s s a ys f r o m t h e a p h e r esi s a ut o g r aft.  
C F U ass a ys ar e d o n e  t o e v al u at e t h e pr olif er ati o n a n d diff er e nti ati o n p ot e nti al of H S Cs. 3 0 -3 2  T h e y 
pr o vi d e i n-vitr o  f u ncti o n al d at a of c oll e cte d H S Cs, w hic h c o m pl e m e nts t h e d at a o bt ai n e d fr o m fl o w 
c yt o m etric e v al u ati o n of t h e a p h er esis pr o d uct . T h e a p h er esis pr o d uct c o nt ai ni n g t h e H S Cs is pl at e d  
o n a c ult ur e m e di u m. C ult ur es ar e gr o w n f or a fi x e d d ur ati o n of ti m e ( 1 0 -1 4 d a ys) a n d pr o g e nit or 
c ol oni es gr o wi n g i n t h e c ult ur e m e di a ar e t h e n q u a ntifi e d. T h e y i ncl u d e: c ol o n y f or mi n g u nit 
er yt hr oc yti c ( C F U -E), bl ast f or mi n g u nit er yt hr oc ytic ( B F U -E), c ol o n y f or mi n g u nit gr a n ul oc ytic a n d 
m o n oc ytic ( C F U -G M), c ol o n y f or mi n g u nit gr a n ul oc ytic, er yt hr oc yti c,  m o n oc yti c a n d m e g a k ar y oc ytic 
( C F U-G E M M), a n d hi g hl y pr olif er ati v e pr o g e nit ors ( H P P).   
 
C F U ass a ys will b e d o n e p er St a nf or d B M T C ell ul ar T h er a p y F a cilit y  st a n d ar d pr ot oc ol, w hi c h is 
s u m m ariz e d b el o w: 
 
o  D esir e d pl ati n g c o nc e ntr ati o n f or u n m a ni p ul at e d fr es h or t h a w e d p eri p h er all y c oll e ct e d 
a ut ol o g o us H S Cs:  St arti n g Pr o d uct: 1 -5 x 1 0 5  c ells 
o  R e q uir e d v ol u m e of fr es h or t h a w e d c ell s us p e nsi o n a n d Isc o v e is a d d e d t o C F U m e di a t u b e 
o  F oll o wi n g t h e a d diti o n c ells a n d Isc o v e ar e a d d e d t o m et h y ell ul os e tiss u e c ult ur e m e di u m, 
t h e c ells ar e v ort e x e d  
o  O nc e t h e c ell - m et h y ell ul os e tiss u e c ult ur e s ol uti o n s ettl es, 2 w ells of a 2 4 -w ell pl at e ar e e a c h 
s e e d e d wit h 0. 2 5 m L of t h e s ol uti o n  
o  T h e r e m ai ni n g w ells ar e fill e d wit h ~ 0. 5 m L P B S t o pr e v e nt e v a p or ati o n  
o  Pl at e  is i nc u b at e d f or 1 4 d a ys, t h e n r e a d f or C F Us. 
 
 

Pr ot oc ol: M G T A -1 4 5 + pl eri x af or m o bili z ati o n  
 
Pr ot oc ol V ersi o n 1. 4 , 2 1 A pril  2 0 2 1  
2 9  
I R B-5 7 0 5 6  
 
C o r r el ati v e St u d y # 3: T o a s s es s i m m u n ol o gi c al c h a r a ct eri z ati o n of w hit e c ell p o p ul ati o n 
s u b s et s ( T c ell s u b s et s, B c ell s u b s et s, N K c ell s, n e ut r o p hil s, a n d m y el oi d / m o n o c yti c c ell s) i n 
p ati e nt’s p e ri p h e r al bl o o d at d a y 2 8 u si n g fl o w c yt o m et r y a n d at d a y 1 0 0 u si n g fl o w c yt o m et r y 
a n d C y T O F  
 
H S Cs i nf us e d aft er hi g h -d os e c h e m ot h er a p y r e p o p ul at e t h e h ost h e m at o p oi esis a n d l e a d t o gr a d u al 
i m m u n e r e c o nstit uti o n. T h e ti m e t a k e n f or r e c o v er y of diff er e nt i m m u n e c ell s u bs ets c a n v ar y.  
I m m u n e r e c o nstit uti o n n ot o nl y i m p a cts i nf e cti o us dis e as e o utc o m es, b ut als o h as b e e n s h o w n t o 
h a v e a n i m p a ct o n s ur vi v al o utc o m es. E arl y l y m p h oc yt e c o u nt r e c o v er y is ass oci at e d wit h b ett er 
s ur vi v al aft er A S C T i n p ati e nts wit h m ulti pl e m y el o m a a n d n o n -H o d g ki n’s l y m p h o m a. 3 3, 3 4  It h as b e e n 
s h o w n t h at hi g h er l y m p h oc yt e s u bs et pr o p orti o ns i n t h e st e m c ell gr aft ar e ass oci at e d wit h p ost -
tr a ns pl a nt l y m p h oc yt e c o u nt, w hic h i n t ur n h as a str o n g c orr el ati o n wit h s ur vi v al o utc o m es. 3 3, 3 5    
 
W hit e c ell s u bs ets will b e e v al u at e d b y fl o w c y t o m etr y a n d ( C y T O F  (C yt o m etr y b y Ti m e -Of -Fli g ht) at 
d a y 2 8 a n d  d a y 1 0 0  (T a bl e 4  a n d  T a bl e 5 ). M ass c yt o m etr y all o ws si m ult a n e o us t esti n g of s e v er al 
a nti g e ns a n d is a d v a nt a g e o us t o pr o vi d e a d et ail e d o v er vi e w of diff er e nt c ell p o p ul ati o ns a n d w h e n 
st u d yi n g s m all tr a nsiti o n al i m m u n e p o p ul ati o ns. 3 6, 3 7  T h e pr oc ess is w ell -est a blis h e d at t h e  h u m a n 
i m m u n e m o nit ori n g c or e l a b or at or y f a ciliti es at St anf or d U ni v ersit y. 3 8 -4 3    
 
T a bl e 4 : Fl o w C yt o m et r y P a n el wit h A nti b o di es f o r T a n d N K c ell st ai ni n g  
Fl u or oc hr o m e  
T ar g et  
Cl o n e  
V e n d or  
C at al o g 
N o.  
Fi n al 
Dil uti o n  
µ L A b / 
1 0 0 µ L 
c oc kt ail  
FI T C  
h C D 1 0 3  
B er -A C T 8  
B D Bi osci e nc es  
5 5 0 2 5 9  
1: 1 0 0  
1  
P E  
h C D 6 2 L  
D R E G -5 6  
B D Bi osci e nc es  
5 5 5 5 4 4  
1: 1 0 0  
1  
P er C P -C y 5. 5  
h C D 4  
R P A -T 4  
B D Bi osci e nc es  
5 6 0 6 5 0  
1: 1 0 0  
1  
A P C -H 7  
h C D 8  
S K 1  
B D Bi osci e nc es  
5 6 0 1 7 9  
1: 1 0 0  
1  
A P C  
h C D 5 6  
B 1 5 9  
B D Bi osci e nc es  
5 5 5 5 1 8  
1: 5 0  
2  
P E -C y 7  
h C D 4 5 R A  
HI 1 0 0  
B D Bi osci e nc es  
5 6 0 6 7 5  
1: 1 0 0  
1  
B V 5 1 0  
h C D 3 1  
W M 5 9  
B D Bi osci e nc es  
5 6 3 4 5 4  
1: 1 0 0  
1  
A P C -R 7 0 0  
h C C R 7  
3 D 1 2  
B D Bi osci e nc es  
5 6 5 8 6 7  
1: 1 0 0  
1  
B V 4 2 1  
h C D 3  
S K 7  
B D Bi osci e nc es  
5 6 3 7 9 8  
1: 1 0 0  
1  
 
T a bl e 5 : Fl o w C yt o m et r y P a n el wit h A nti b o di es f o r B c ell st ai ni n g  
Fl u or oc hr o m e  
T ar g et  
Cl o n e  
V e n d or  
C at al o g 
N o.  
Fi n al 
Dil uti o n  
µ L A b / 
1 0 0 µ L 
c oc kt ail  
FI T C  
hI g M  
G 2 0 -1 2 7  
B D Bi osci e nc es  
5 5 5 7 8 2  
1: 1 0 0  
1  
P E  
h C D 2 4  
M L 5  
B D Bi osci e nc es  
5 5 5 4 2 8  
1: 1 0 0  
1  
P er C P -C y 5. 5  
h C D 1 9  
HI B 1 8  
B D Bi osci e nc es  
5 6 1 2 9 5  
1: 1 0 0  
1  
A P C -H 7  
h C D 2 0  
2 H 7  
e Bi osci e nc es  
5 6 1 1 7 2  
1: 1 0 0  
1  
A P C  
h C D 3 8  
HI T 2  
B D Bi osci e nc es  
5 5 5 4 6 2  
1: 1 0 0  
1  
P E -C y 7  
h C D 2 7  
M -T 2 7 1  
B D Bi osci e nc es  
5 6 0 6 0 9  
1: 1 0 0  
1  
B V 5 1 0  
hI g D  
I A 6-2  
B D Bi osci e nc es  
5 6 3 0 3 4  
1: 1 0 0  
1  
B V 4 2 1  
h C D 3  
S K 7  
B D Bi osci e nc es  
5 6 3 7 9 8  
1: 1 0 0  
1  

Pr ot oc ol: M G T A -1 4 5 + pl eri x af or m o bili z ati o n  
 
Pr ot oc ol V ersi o n 1. 4 , 2 1 A pril  2 0 2 1  
3 0  
I R B-5 7 0 5 6  
 
2. 6  
P ati e nt R e p o rt e d O ut c o m e M e a s u r es: B a c k g r o u n d a n d E v al u ati o n  
A c o m bi n e d q u esti o n n air e i nc or p or ati n g it e ms a d a pt e d f or i nf usi o n sit e r e a cti o n fr o m t h e P ati e nt 
R e p ort e d O utc o m es C o m m o n  T er mi n ol o g y Crit eri a f or A d v ers e E v e nts ( P R O -C T C A E) as w ell as t h e 
Bri ef P ai n I n v e nt or y will b e us e d  t o m e as ur e p ati e nt r e p ort e d o utc o m es. P ati e nts will b e as k e d t o 
c o m pl et e q u esti o n n air es o n e a c h d a y of c oll e cti o n a n d wit hi n o n e w e e k f oll o wi n g st e m c oll e cti o n. 
 
P R O -C T C A E  is a s elf-r e p orti n g t o ol, w hic h h as b e e n pr e vi o usl y v ali d at e d i n p ati e nts u n d er g oi n g 
tr e at m e nt f or h e m at ol o gic m ali g n a nci es.4 4, 4 5  It c o v ers v ari o us s y m pt o ms a n d ass ess es t h e pr es e nc e, 
fr e q u e nc y a n d s e v erit y of t h es e s y m pt o ms. W e will ass ess t h e f oll o wi n g s y m pt o m ati c A Es : ‘p ai n 
a n d s w elli n g at i nj e cti o n sit e ’ 
 
T h e B ri ef P ai n I n v e nt o r y ( B PI)  c o nsists of 2 d o m ai ns: p ai n s e v erit y a n d p ai n i nt erf er e nc e  wit h a 
r e c all of 2 4 h o urs. 4 6  W e will us e t h e s h ort -f or m B PI wit h 9 it e ms, w hic h r e q uir es a b o ut 5 mi n ut es t o 
c o m pl et e, o n a v er a g e. P ati e nts ar e as k e d if t h e y h a v e p ai n a n d t h e l oc ati o n of p ai n, P ai n s e v erit y is 
sc or e d o n a sc al e of 0 -1 0 wit h 0 = n o p ai n a n d 1 0 = p ai n as b a d as y o u c a n i m a gi n e.  T h e B PI p ai n 
s e v erit y q u esti o ns ass ess p ai n at its “ w orst,” “l e ast,” “ a v er a g e,” a n d “ n o w” (c urr e n t p ai n). A c o m p osit e 
m e a n s e v erit y sc or e of t h es e f o ur it e ms m a y b e c alc ul at e d. P ai n i nt erf er e nc e is esti m at e d b y 7 
q u esti o ns t h at as k p ati e nts h o w p ai n h as i nt erf er e d wit h s e v e n d ail y a cti viti es: w al ki n g, w or k, m o o d, 
e nj o y m e nt of lif e, r el ati o ns wit h ot h e rs, a n d sl e e p. P ai n i nt erf er e nc e is sc or e d as a m e a n of t h e 7 
i nt erf er e nc e it e ms. I m p ut ati o n f or missi n g d at a m a y b e us e d if at l e ast 5 0 % ( 4 of 7 it e ms h a v e b e e n 
c o m pl et e d at a gi v e n ti m e p oi nt). Q u esti o ns a b o ut p ai n m e dic ati o ns a n d t h eir eff e cti v e n ess ar e  als o 
i ncl u d e d.  
 
A c o p y of t h e i nstr u m e nt is pr o vi d e d i n t h e A P P E N DI X B : P R O Q u esti o n n air e  .  It is als o a v ail a bl e i n 
ot h er l a n g u a g es i ncl u di n g S p a nis h. It is v ali d at e d f or b ot h a c ut e a n d c hr o nic p ai n. It c a n b e s elf -
r e p ort e d or as k e d i n a n i nt er vi e w f or m at. As d escri b e d a b o v e, t h er e is n o sc ori n g al g orit h m, b ut 
" w orst p ai n" or t h e arit h m etic m e a n  of t h e f o ur s e v erit y it e ms  c a n b e us e d as m e as ur es of p ai n 
s e v erit y; t h e arit h m etic m e a n of t h e s e v e n i nt erf er e nc e it e ms c a n b e us e d as a m e as ur e of p ai n 
i nt erf er e nc e.  
 
I nt er n al c o nsist e nc y f or B PI r a n g es fr o m 0. 8 0 t o 0. 8 7 f or 4 it e ms f or p ai n s e v erit y as d e m o nstr at e d 
i n n u m er o us st u di es, i ncl u di n g c a nc er p ati e nts 4 7, 4 8 . T est-r et est ( 1 d a y t o 1 w e e k) r eli a bilit y f or 
r ati n gs of p ai n as w orst ( 0. 9 3) a n d u s u al or a v er a g e p ai n ( 0. 7 8) i n p ati e nts wit h m et ast atic c a nc er 
w er e a cc e pt a bl e 
4 6 . F a ct or str uct ur e a n d t h e vali dit y of B PI h as b e e n s ucc essf ull y t est e d 4 9 . T h e 
v ali dit y of B PI w as ass ess e d usi n g k n o w n -gr o u p m et h o ds wit h m e a n sc or es c o m p ar e d a cr oss c a nc er 
p ati e nts t a ki n g diff er e nt t y p es of p ai n m e dic ati o n ( n arc otic v ers us n o n -n arc otic p ai n r eli e v ers). T h e 
B PI m e a n sc or es si g nific a ntl y dif f er e d b et w e e n t h e 2 gr o u ps, d e m o nstr ati n g str o n g e vi d e nc e of 
v ali dit y 
4 7 . F or c o n diti o ns s uc h as b o n e 
m et ast asis a n d fi br o m y al gi a, t h e mi ni m u m cli nic all y 
i m p ort a nt diff er e nc e f or B PI is a p pr o xi m at el y a 2-p oi nt c h a n g e fr o m b as eli n e 5 0, 5 1 . A n ot h er st u d y o n 
b u pr e n or p hi n e us e d i n l o w er b a c k p ai n us e d a m i ni m al cli nic all y im p ort a nt d iff er e nc e of 2 p oi nts f or 
B PI 5 2 . 
 
P R O q u esti o n n air es will b e d esi g n e d i n a p a p er f or m at t o b e fill e d b y t h e p ati e nt or st u d y t e a m 
m e m b er i nt er vi e wi n g t h e p ati e nt. D at a will b e u pl o a d e d i n t h e E D C t o ol.  

Pr ot oc ol: M G T A -1 4 5 + pl eri x af or m o bili z ati o n  
 
Pr ot oc ol V ersi o n 1. 4 , 2 1 A pril  2 0 2 1  
3 1  
I R B-5 7 0 5 6  
 
3  
P A R TI CI P A N T S E L E C TI O N A N D E N R O L L M E N T P R O C E D U R E S  
P ati e nt eli gi bilit y will b e r e vi e w e d. I ncl usi o n a n d e xcl usi o n crit eri a ar e list e d b el o w. Eli gi bl e 
p ati e nts will b e i n vit e d t o e nr oll o n t o t h e st u d y a n d will si g n i nf or m e d  c o ns e nt as d escri b e d i n 
S e cti o n 3. 3 . R ef er t o t h e P artici p a nt Eli gi bilit y C h e c klist i n A P P E N DI X B : P R O Q u esti o n n air e  .  
3. 1  
I n clu si o n C rit eri a  
 
3. 1. 1  
Di a g n osis of m ulti pl e m y el o m a p er t h e I nt er n ati o n al M y el o m a 
W or ki n g Gr o u p (I M W G) 
crit eri a .  
 
3. 1. 2      A g e: 1 8 t o 7 0 y e ars  
 
3. 1. 3  
Eli gi bl e f or A S C T p er i nstit uti o n al g ui d eli n es  
 
3. 1. 4  
Wit hi n o n e y e ar of st art of m y el o m a t h er a p y 
 
 
3. 1. 5  
C ar di a c a n d p ul m o n ar y st at us s uffici e nt t o u n d er g o a p h er esis a n d tr a ns pl a nt ati o n p er 
i nstit uti o n al tr a ns pl a nt g ui d eli n es. 
  
 
3. 1. 6  
C alc ul at e d  cr e ati ni n e cl e ar a nc e > 3 0 m L / mi n a cc or di n g t o t h e M o dific ati o n of Di et i n R e n al 
Dis e as e ( M D R D) f or m ul a 
 
 
3. 1. 7  
A bs ol ut e n e utr o p hil c o u nt >  1 5 0 0 x 1 0 6 / L  a n d pl at el ets >  1 0 0, 0 0 0 x 1 0 6 / L  
 
3. 1. 8  
A bilit y t o u n d erst a n d a n d t h e willi n g n ess t o si g n a writt e n i nf or m e d c o ns e nt d oc u m e nt.  
 
3. 1. 9  
A gr e e m e nt t o us e a n a p pr o v e d f or m of c o ntr ac e pti o n f or m al e p ati e nts or f e m al e p ati e nts of 
c hil d b e ari n g  p ot e nti al.  
 
3. 2  
E x cl u si o n C rit eri a  
 
3. 2. 1  
Hist or y of pri or st e m c ell tr a ns pl a nt f or m ulti pl e m y el o m a or ot h er i n dic ati o ns  
 
3. 2. 2  
Pl a n n e d t a n d e m st e m c ell tr a ns pl a nt 
 
3. 2. 3  
Pri or hist or y of f ail ur e t o c oll e ct H S Cs . 
 
3. 2. 4  
Li v er f u ncti o n t ests: T ot al b ilir u bi n > 1. 5 x u p p er li mit of n or m al ( U L N) i n t h e a bs e nc e of a 
d oc u m e nt e d hist or y of Gil b ert’s s y n dr o m e a n d / or A S T / A L T > 3 x U L N  
 
3. 2. 5  
K n o w n all er g y t o M G T A -1 4 5 or pl eri x af or  
 
3. 2. 6  
Lif eti m e e x p os ur e t o l e n ali d o mi d e gr e at er t h a n 6 c u m ul ati v e m o nt hs of tr e at m e nt , i. e. m or e 
t h a n si x 2 8 -d a y c ycl es or m or e t h a n ei g ht 2 1 -d a y c ycl es  
 
3. 2. 7  
Pr e g n a nt or l a ct ati n g w o m e n .   
 

Pr ot oc ol: M G T A -1 4 5 + pl eri x af or m o bili z ati o n  
 
Pr ot oc ol V ersi o n 1. 4 , 2 1 A pril  2 0 2 1  
3 2  
I R B-5 7 0 5 6  
 
3. 3  
I nf o r m e d C o n s e nt P r o c es s 
All p artici p a nts will b e  pr o vi d e d a c o ns e nt f or m d escri bi n g t h e st u d y wit h s uffici e nt i nf or m ati o n f or 
p artici p a nts t o m a k e a n i nf or m e d d e cisi o n r e g ar di n g t h eir p artici p ati o n.  P artici p a nts will  si g n t h e 
I R B a p pr o v e d i nf or m e d c o ns e nt pri or t o p artici p ati o n i n a n y st u d y s p e cific pr oc e d ur e. T h e 
p artici p a nt will  r e c ei v e a c o p y of t h e si g n e d a n d d at e d c o ns e nt d oc u m e nt.  T h e ori gi n al si g n e d c o p y 
of t h e c o ns e nt d oc u m e nt will  b e r et ai n e d i n t h e m e dic al r e c or d or r es e arc h fil e.  
 
 
3. 4  
St u d y Ti m eli n e  
P ri m a r y C o m pl eti o n:  T h e st u d y is a ntici p at e d t o r e a c h pri m ar y c o m pl eti o n 1 5  m o nt hs fr o m t h e 
ti m e t h e st u d y o p e ns t o a ccr u al. W e a ntici p at e a ccr u al t o b e c o m pl et e d i n 9 -1 2  m o nt hs a n d t h e 
r e m ai n d er of t h e ti m e is r e q uir e d t o c o m pl et e i nt er v e nti o n a n d f oll o w -u p f or pri m ar y e n d p oi nt.  
 
St u d y C o m pl eti o n:  T h e st u d y is a ntici p at e d t o r e a c h st u d y c o m pl eti o n 1 8  m o nt hs fr o m t h e ti m e t h e 
st u d y o p e ns t o a ccr u al.  
 
4  
T R E A T M E N T P L A N  
T his is a n o p e n l a b el, pr o of of c o nc e pt,  p h as e 2 st u d y t o e v al u at e s af et y a n d effic a c y of st e m c ell 
m o bili z ati o n (f or hi g h -d os e t h er a p y a n d a ut ol o g o us st e m c ell r esc u e) wit h a c o m bi n ati o n of M G T A -
1 4 5 a n d pl eri x af or, f oll o w e d b y s a m e d a y a p h er esis i n p ati e nts wit h m ulti pl e m y el o m a.  
 
T h e pri m ar y e n d p oi nt is s ucc essf ul c oll e cti o n ≥2 . 0 x  1 0 6  C D 3 4 +  c ells / k g wit h u p t o t w o  a p h er esis 
s essi o ns aft er 
M G T A -1 4 5 / pl eri x af or 
d osi n g. 
S e c o n d ar y 
e n d p oi nts i ncl u d e 
c oll e cti o n 
of 
≥2. 0  x  1 0 6  C D 3 4 +  c ells / k g a n d ≥4. 0  x  1 0 6  C D 3 4 +  c ells / k g o n d a y o n e of a p h er esis a n d ≥4. 0  x  1 0 6  C D 3 4 +  
c ells / k g a n d ≥6. 0  x  1 0 6  C D 3 4 +  c ells / k g i n u p t o t w o d a ys of a p h er esis aft er M G T A-1 4 5 / pl eri x af or 
d osi n g, ass ess m e nt of a d v ers e e v e nts a n d s ucc essf ul e n gr aft m e nt as d escri b e d a b o v e.  
 
4. 1  
St u d y T r e at m e nt  a n d F oll o w -u p  
 
T h e st u d y i s di vi d e d i nt o t h r e e p h a s es: 
4. 1. 1  P r e -m o bili z ati o n  p h a s e :  
a.  P ati e nts w h o ar e eli gi bl e  will b e  i n vit e d t o p artici p at e i n  t h e st u d y. P ati e nts will 
u n d er g o scr e e ni n g a n d b as eli n e e v al u ati o n .  
b.  If p ati e nts d o n ot m e et eli gi bilit y crit eri a aft er si g ni n g c o ns e nt  o n t h e st u d y, t h e y will 
b e d e e m e d scr e e n f ail ur es  a n d m a y b e r e pl a c e d t o m e et a ccr u al g o al.  P ati e nts m a y b e 
r escr e e n e d at t h e discr eti o n of t h e pri nci p al i n v esti g at or.  P ati e nts will b e e nr oll e d 
aft er t h e y m e et eli gi bilit y crit eri a .  
c. C h e m ot h er a p y, i ncl u di n g i m m u n o m o d ul at or y dr u gs (I Mi Ds) t o b e st o p p e d 1 4 d a ys 
pri or t o st art of m o biliz ati o n.   
 
4. 1. 2  S tu d y T r e at m e nt: s t e m c ell m o bili z ati o n / a p h e r esi s: 
 
All p ati e nts will r e c ei v e M G T A -1 4 5 a n d pl eri x af or at si n gl e, w ei g ht-b as e d d os es. D os e of M G T A -1 4 5 
h as b e e n d et er mi n e d fr o m a p h as e 1 st u d y.  P ati e nts will u n d er g o s e q u e nti al a d mi nistr ati o n of 
pl eri x af or 0. 2 4  m g / k g s u bc ut a n e o usl y f oll o w e d 2 h o urs l at er b y M G T A -1 4 5 at 0. 0 3 m g / k g 
i ntr a v e n o usl y (3 t o 1 0 mi n ut e i nf usi o n)  as s h o w n i n T a bl e 6 . F or p ati e nts wit h cr e ati ni n e cl e ar a nc e 
< 5 0 ml / mi n ut e, pl eri x af or d os e will b e r e d uc e d t o 0. 1 6 m g / k g p er p a c k a g e i ns ert.  T his will b e 
f oll o w e d b y a p h er esis. 

Pr ot oc ol: M G T A -1 4 5 + pl eri x af or m o bili z ati o n  
 
Pr ot oc ol V ersi o n 1. 4 , 2 1 A pril  2 0 2 1  
3 3  
I R B-5 7 0 5 6  
 
T a bl e 6 : T r e at m e nt Pl a n  
M G T A -1 4 5  
Pl e ri x af o r  
D o s e 
( m g / k g) 
T ot al D o s es 
A d mi ni st er e d  
R o ut e  
D o s e 
( m g / k g) 
T ot al D o s es 
A d mi ni st er e d  
R o ut e  
0. 0 3  
1 p er d a y f or 1 t o 
2  d a ys  
I V 
0. 2 4 0  or 
0. 1 6*  
1 p er d a y f or 1 
t o 2 d a ys  
S C  
  I V =i ntr a v e n o us; S C =s u bc ut a n e o us. * Pl eri x af or d os e r e d uc e d t o 0. 1 6 m g / k g p er 
p a c k a g e i ns ert f or cr e ati ni n e cl e ar a nc e <  5 0 ml / mi n ut e  
 
•  
D r u g di s c o nti n u ati o n / d o s e m o difi c ati o n s:   
T h er e ar e n o d os e m o dific ati o ns f or t h e i n v esti g ati o n al a g e nt ( M G T A-1 4 5). If a n y p ati e nt e x p eri e nc e 
gr a d e 3 or hi g h er n o n -h e m at ol o gic al t o xicit y, n ot a p pr o pri at el y c o ntr oll e d b y a p pr o pri at e 
i nt er v e nti o ns, a n d c o nsi d er e d p ossi bl y, pr o b a bl y or d efi nit el y r elat e d t o M G T A -1 4 5, t h e st u d y dr u g 
will b e disc o nti n u e d a n d p ati e nt will g o t o e v e nt m o nit ori n g.   
 
•  
I nf u si o n r e a cti o n:  
P ati e nts s h o ul d b e c ar ef ull y m o nit or e d d uri n g M G T A -1 4 5 i nf usi o n. If a n i nf usi o n-r el at e d r e a cti o n 
d e v el o ps, t h e n t h e i nf usi o n s h o ul d b e t e m p or aril y i nt err u pt e d or sl o w e d d o w n. S u bj e cts w h o 
e x p eri e nc e a d v ers e e v e nts d uri n g t h e i nf usi o n m ust b e tr e at e d a cc or d i n g t o t h e i n v esti g at or’s 
j u d g m e nt a n d b est cli nic al pr a ctic e. As d escri b e d a b o v e, if p ati e nt e x p eri e nc es a gr a d e 3 or hi g h er 
i nf usi o n r e a cti o n wit h M G T A-1 4 5 t h at is n ot a d e q u at el y c o ntr ol l e d b y a p pr o pri at e i nt er v e nti o ns, 
tr e at m e nt wit h M G T A-1 4 5 will b e disc o nti n u e d.  
 
•  
A p h e r e si s:   
 
A p h er esis c oll e cti o n will b e gi n as s o o n as f e asi bl e aft er M G T A -1 4 5 i nf usi o n. A p h er esis s h o ul d 
c o m m e nc e (i. e.  i nl et fl o w i niti at e d) n o l at er t h a n 3 0 mi n ut es aft er t h e st art of M G T A-1 4 5 i nf usi o n. 
A p h er esis pr oc e d ur e , i ncl u di n g t h e m a c hi n e, c oll e cti o n pr oc e d ur e a n d s u p p orti v e c ar e will b e i n 
a cc or d a nc e wit h  st a n d ar diz e d pr oc e d ur es of  St a nf or d B l o o d a n d m arr o w tr a nspl a nt ati o n 
( B M T) /A p h er esis  pr o gr a m. E a c h s essi o n of a p h er esis will b e st a n d ar diz e d  t o p r o c es s at l e a st 3  
bl o o d v ol u m es, wit h a m a r gi n of + / -1 0 %  o r 4. 5 h o u r s, w hi c h e v e r i s l o n g e r . T h e pr oc essi n g a n d 
cr y o pr es er v ati o n of t h e a p h er esis pr o d uct will b e i n a cc or d a nc e wit h St a nf or d B M T / C ell T h er a p y 
F a cilit y ( C T F) st a n d ar d iz e d pr oc e d ur es.  
A s e c o n d d a y of m o biliz ati o n a n d a p h er esis will b e p urs u e d i n p ati e nts w h o h a v e n ot c oll e ct e d 
6. 0  x  1 0 6  C D 3 4 +  c ells / k g i n o n e s essi o n.  
 
A mi ni m u m  of 2 . 0 x  1 0 6  C D 3 4 +  c ells / kg s h o ul d b e c oll ect e d i n o n e t o t w o a p h er esis s essi o ns. If t his 
g o al is n ot m et, p ati e nt m a y pr oc e e d wit h w as h o ut pr ot oc ol a n d alt er n ati v e st e m c ell m o biliz ati o n 
str at e g y as r o uti n e cli nic al c ar e  at discr eti o n of tr e ati n g p h ysici a n .  
 
P ati e nts w h o h a v e c oll e ct e d b et w e e n 2. 0 t o 3. 9 x 1 0 6 C D 3 4 +  c ells / k g m a y pr oc e e d t o tr a ns pl a nt at 
t h e discr eti o n of t h e tr e ati n g p h ysici a n p er c urr e nt i nstit uti o n al g ui d eli n es. Alt er n ati v el y, t h e y c a n  
pr oc e e d wit h w as h o ut pr ot oc ol a n d alt er n ati v e st e m c ell m o biliz ati o n str at e g y  as r o uti n e cli nic al 
c ar e . T his d e cisi o n will b e m a d e b y t h e tr e ati n g p h ysici a n i n c o ns ult ati o n wit h t h e PI. A mi ni m u m of 
2. 0  x  1 0 6  C D 3 4 +  c ells / k g ar e r e c o m m e n d e d f or s ucc essf ul e n gr aft m e nt f or tr a ns pl a nt a n d w hil e a 
b ac k -u p is pr ef err e d, it is n ot m a n d at or y. T his is i n a cc or d a nc e wit h c urr e nt i nstit uti o n al st a n d ar d 
of pr a ctic e.  

Pr ot oc ol: M G T A -1 4 5 + pl eri x af or m o bili z ati o n  
 
Pr ot oc ol V ersi o n 1. 4 , 2 1 A pril  2 0 2 1  
3 4  
I R B-5 7 0 5 6  
 
 
If a n a d diti o n al c oll e cti o n is s o u g ht at t h e discr eti o n of t h e tr e ati n g p h ysici a n, it is r e c o m m e n d e d 
n ot t o c o m bi n e t h e st a n d ar d of c ar e m o biliz e d gr aft a n d t h e M G T A -1 4 5 + pl eri x af or m o biliz e d gr aft 
f or o n e tr a ns pl a nt. 
 
•  
P ati e nt R e p o rt e d O ut c o m es A s s es s m e nt  
 
P ati e nt r e p ort e d o utc o m es will b e ass ess e d as d escri b e d i n S e cti o n 2. 6  a b o v e usi n g a p ati e nt 
r e p ort e d q u esti o n n air e i nc or p or ati n g it e ms fr o m P R O -C T C A E, as w ell as t h e Bri ef P ai n I n v e nt or y.  
 
El e ctr o nic q u esti o n n air es will b e d esi g n e d t h at will b e a d mi nist er e d usi n g a t a bl et. P a p er c o pi es will 
b e a v ail a bl e as b ac k -u p a n d m a y b e us e d if u n a bl e t o c o m pl et e t h e el e ctr o nic q u esti o n n air es. 
El e ctr o nic q u esti o n n air es will b e dir e ctl y u pl o a d e d i nt o t h e  M e d ri o d at a b as e. If p a p er c o pi es ar e 
us e d, t h e y will b e h a n d e d t o t h e st u d y c o or di n at or a n d st or e d i n a l oc k e d r o o m at St a nf or d H e alt h 
C ar e. P a p er a ns w ers will b e e nt er e d i nt o el e ctr o nic f or m at b y t h e st u d y st aff.  
 
P R O q u esti o n n air e is d esi g n e d t o b e s elf-a d mi nist er e d b y t h e p ati e nt ( assist a nc e c a n b e pr o vi d e d b y 
st u d y /cli nic al st aff, as n e e d e d) o n e a c h d a y of a p h er esis a n d at t h e p ost -a p h er esis cli nic visit ( wit hi n 
7 d a ys of a p h er esis). O n t h e d a y of a p h er esis, t h e q u esti o n n air e s h o ul d b e gi v e n t o t h e p at i e nt aft er 
at l e ast 3 0 mi n ut es f oll o wi n g M G T A -1 4 5 i nf usi o n. It is r e c o m m e n d e d t o gi v e it t o t h e p ati e nt t o w ar ds 
t h e e n d of t h e a p h er esis s essi o n.  
 
G e n e r al C o n c o mit a nt M e di c ati o n a n d S u p p o rti v e C a r e G ui d eli n es D u ri n g St u d y 
T r e at m e nt  
- 
Ot h er gr o wt h f a ct or m e d ic ati o ns (e. g. , G-C S F, gr a n ul oc yt e -m a cr o p h a g e c ol o n y -
sti m ul ati n g f a ct or) ar e pr o hi bit e d u p t o 7 d a ys pri or t o a n d d uri n g t h e 
m o biliz ati o n a n d a p h er esis p h as e of t h e st u d y.  
- 
P ati e nts e nr oll e d o n t his st u d y will r efr ai n fr o m p artici p ati o n i n a n y ot h er 
i n v esti g ati o n al st u d y dr u g i n w hic h t h e y will r e c ei v e a n i n v esti g ati o n al st u d y dr u g 
f or at l e ast f o ur w e e ks b ef or e d osi n g of M G T A -1 4 5 a n d pl eri x af or.  
- 
I m m u n o m o d ul at or y dr u gs (I Mi Ds) t o b e st o p p e d 1 4 d a ys pri or t o st art of 
m o biliz ati o n. Ot h er c a nc er dir e ct e d t h er a p y t o b e st o p p e d at l e ast 1 0 d a ys pri or 
t o st art of m o biliz ati o n. 
- 
P ati e nts s h o ul d r e c ei v e f ull s u p p orti v e c ar e w hil e o n t his st u d y. T his i ncl u d es 
bl o o d pr o d u ct s u p p ort, a nti bi otic tr e at m e nt, a n d tr e at m e nt of ot h er n e wl y 
di a g n os e d or c o nc urr e n t m e dic al c o n diti o ns. All bl o o d pr o d ucts a n d c o nc o mit a nt 
m e dic ati o ns s uc h as a nti di arr h e als, a n al g esics, a n d / or a nti e m etics r e c ei v e d fr o m 
t h e first d a y of st u d y tr e at m e nt a d mi nistr ati o n u ntil 3 0 d a ys aft er t h e fi n al d os e 
of M G T A -1 4 5 or u ntil st art of tr a ns pl a nt c o n diti o ni n g ( w hic h e v er c o m es first) will 
b e r e c or d e d i n t h e m e dic al r e c or ds.  
 

Pr ot oc ol: M G T A -1 4 5 + pl eri x af or m o bili z ati o n  
 
Pr ot oc ol V ersi o n 1. 4 , 2 1 A pril  2 0 2 1  
3 5  
I R B-5 7 0 5 6  
 
4. 1. 3.  E v e nt M o nit o ri n g / F oll o w -u p :  
a.  A p ati e nt will b e c o nsi d er e d t o h a v e f ail e d m o biliz ati o n a n d will disc o nti n u e st u d y 
dr u g a n d g o t o E v e nt M o nit ori n g if t h e y f ail t o c oll e ct t h e mi ni m u m of 2. 0 milli o n C D 3 4 
c ells / k g aft er t w o a p h er esis s essi o ns . 
 
b.  If p ati e nts u n d er g o r esc u e 
m o biliz ati o n f or a n y r e as o n (i ncl u di n g p h ysici a n 
discr eti o n t o c oll e ct m or e t h a n 2 milli o n C D 3 4 + c ells / k g) a n d pr oc e e d t o u pfr o nt 
tr a ns pl a nt wit h M G T A-1 4 5 a n d pl eri x af or m o biliz e d gr aft, t h e y will b e f oll o w e d f or 
s e c o n d ar y tr a ns pl a nt r el at e d o utc o m es. O n t h e ot h er h a n d, if  t h e y u n d er g o u pfr o nt 
tr a ns pl a nt wit h t h e  st e m c ell gr aft o bt ai n e d fr o m r esc u e m o biliz ati o n  o nl y , t h e y will 
n ot b e f oll o w e d f or s e c o n d ar y o utc o m es.  It is r e c o m m e n d e d n ot t o c o m bi n e gr afts 
fr o m diff er e nt m o biliz ati o n pr ot oc ols 
 
c. P ati e nts w h o ar e n ot pr oc e e di n g wit h u pfr o nt A S C T will b e f oll o w e d i n E v e nt 
M o nit ori n g u ntil 3 0 d a ys  aft er c o m pl eti o n of st u d y dr u g. Aft er t h at n o, f urt h er f oll o w -
u p is r e q uir e d  i n p ers o n or vi a p h o n e visits. P ati e nts ’ el e ctr o nic m e dic al r e c or ds m a y 
b e p eri o dic all y r e vi e w e d t o f oll o w -u p o n 
m y el o m a tr e at m e nt, r es p o ns e a n d 
l a b or at or y v al u es f or u p t o 5 y e ars or w h e n t h e y u n d er g o A S C T l at er i n dis e as e c o urs e, 
w hic h e v er is l at er.  
 
d.  If p ati e nts ar e pr oc e e di n g wit h u pfr o nt tr a ns pl a nt, t h e y will b e f oll o w e d u ntil d a y 1 0 0 
f or s e c o n d ar y o ut c o m es of e n gr aft m e nt, dis e as e pr o gr essi o n a n d s ur vi v al. Aft er  t h at, 
n o f urt h er f oll o w -u p is r e q uir e d  i n p ers o n or vi a p h o n e visits. P ati e nts el e ctr o nic 
m e dic al r e c or ds m a y b e p eri o dic all y r e vi e w e d t o f oll o w -u p o n m y el o m a tr e at m e nt, 
r es p o ns e a n d l a b or at or y v al u es f or u p t o 5 y e ars    
 
e.  A d v ers e E v e nts occ urri n g aft er t h e p ati e nt c o m pl et es st u d y dr u g ( M G T A-1 4 5 a n d 
pl eri x af or)  will b e c oll e ct e d o n t h e E v e nt M o nit ori n g F or m if t h e y ar e > / = gr a d e 3, at 
l e ast p ossi bl y r el at e d t o st u d y dr u gs a n d h a v e n ot b e e n pr e v i o usl y r e p ort e d.  
 
f. 
F or p ati e nts pr oc e e di n g wit h u pfr o nt tr a ns pl a nt ( d efi n e d u p  t o 6 0 d a ys aft er H S C 
m o biliz ati o n), m y el o a bl ati v e m el p h al a n c o n diti o ni n g  ( m el p h al a n 1 4 0- 2 0 0 m g / m 2 ) 
o n d a y -2 f oll o w e d will b y i nf usi o n of c oll e ct e d H S Cs c ells o n d a y 0  p er i nst it uti o n al 
st a n d ar d of pr a ctic e pr ot oc ol.  T h e m a xi m u m i nt er v al b et w e e n st art of st e m c ell 
c oll e cti o n  o n st u d y  a n d tr a ns pl a nt o n t his st u d y is 6 0 d a ys. Wit hi n 6 0 d a ys, t h e ti mi n g 
of tr a ns pl a nt is at t h e discr eti o n of t h e tr e ati n g p h ysici a n.  If tr a ns pl a nt is n ot pl a n n e d 
or is pl a n n e d aft er 6 0 d a ys of c oll e cti o n, t h es e p ati e nts will b e c o nsi d er e d i n t h e 
d el a y e d tr a ns pl a nt gr o u p a n d tr a ns pl a nt r el at e d o utc o m es will n ot b e f oll o w e d.  
 
4. 2  
C rit e ri a f o r P ati e nt Wit h d r a w al  
 
P ati e nts m a y b e wit h dr a w n fr o m t h e st u d y f or t h e f oll o wi n g r e as o ns: 
 

Pr ot oc ol: M G T A -1 4 5 + pl eri x af or m o bili z ati o n  
 
Pr ot oc ol V ersi o n 1. 4 , 2 1 A pril  2 0 2 1  
3 6  
I R B-5 7 0 5 6  
 
a.  If a p ati e nt si g ns t h e c o ns e nt f or m, b ut h as n ot r e c ei v e d t h e st u d y dr u g, t h e p ati e nt will b e 
r e g ar d e d as n o n -e v al u a bl e a n d will b e r e pl a c e d . T his i ncl u d es p ati e nts u n a bl e t o pr oc e e d wit h 
pl a n n e d st e m c ell c oll e cti o n f or a n y r e as o n, i ncl u di n g m e dic al iss u es  t h at pr e cl u d e f urt h er 
p artici p ati o n or r e q uir e a pr o hi bit e d c o nc o mit a nt tr e at m e nt , or a c h a n g e of pl a n . S uc h 
p ati e nts  will b e wit h dr a w n  fr o m t h e st u d y. W h e n  s uc h a n e v e nt h a p p e ns b ef or e t h e st u d y 
dr u g is gi v e n, t h e n p ati e nt will n ot b e f oll o w e d f or  e v e nts / o utc o m es.  T his p ati e nt will b e 
d e e m e d a ‘c a nc el’ a n d n o f urt h er d at a s u b missi o n is n e c ess ar y.  P ati e nts m a y b e r e pl a c e d t o 
m e et a ccr u al g o al.  
b.  P ati e nts w h o r e q u est t o wit h dr a w fr o m t h e st u d y  at a n y p oi nt  will b e r e m o v e d.   
 
c. P ati e nts w h o ar e l ost t o f oll o w -u p b ef or e e n d of st u d y ( d efi n e d as t h e i n a bilit y t o c o nt a ct t h e 
p ati e nt o n 3 s e p ar at e d oc u m e nt e d occ asi o ns) will b e wit h dr a w n fr o m t h e st u d y.  
 
d.  P ati e nts w h o pr oc e e d t o r esc u e m o biliz ati o n a n d u n d er g o tr a ns pl a nt d uri n g t h e st u d y wit h a 
st a n d ar d of c ar e m o biliz e d H S C gr aft.  
 
4. 3  
St u d y St o p pi n g R ul es  
T h e f oll o wi n g ar e s p e cific st u d y st o p pi n g r ul es.  E nr oll m e nt will b e p a us e d p e n di n g disc ussi o ns wit h 
t h e F D A, a g e nt m a n uf a ct ur er a n d St a nf or d I R B s h o ul d a n y of t h e f oll o wi n g st u d y st o p pi n g r ul es occ ur:  
 
 ( 1) A n y p ati e nt wit h a st u d y a g e nt r el at e d Gr a d e 4 or Gr a d e 5 e v e nt  
 
( 2) M or e t h a n  1 ( o n e) p ati e nt e x p eri e nc es f ail e d / d el a y e d e n gr aft m e nt d u e t o n e utr o p hil r e c o v er y 
b e y o n d D a y 4 2  
 
( 3) M or e t h a n  1 ( o n e) p ati e nt d e v el o ps s y m pt o m atic h y p erl e u k oc yt osis .  
S y m pt o m ati c h y p e rl e u k o c yt o si s  is d efi n e d as a w hit e bl o o d c ell ( W B C) c o u nt of o v er 1 0 0 x 1 0 9 / L 
( 1 0 0, 0 0 0 / micr o L) ass oci at e d wit h  s y m pt o ms t h o u g ht t o b e d u e t o tiss u e h y p o xi a . m ost c o m m o n  
s y m pt o ms i ncl u d e  r es pir at or y (s uc h as d ys p n e a a n d h y p o xi a wit h or wit h o ut diff us e i nt erstiti al or 
al v e ol ar i nfiltr at es o n i m a gi n g st u di es ) or n e ur ol o gic al distr ess  (s uc h as vis u al c h a n g es, h e a d a c h e, 
diz zi n ess, ti n nit us, g ait i nst a bilit y, c o nf usi o n, s o m n ol e nc e, a n d  c o m a ). L ess c o m m o n si g ns or 
s y m pt o ms i ncl ud e el e ctr oc ar di o gr a p hic si g ns of m y oc ar di al isc h e mi a or ri g ht v e ntric ul ar o v erl o a d, 
w ors e ni n g r e n al i ns uffici e nc y, pri a pis m, a c ut e li m b isc h e mi a, or b o w el i nf arcti o n . 
I n a d diti o n t o t h e a b o v e r ul es, th e st u d y m a y b e t e m p or aril y or p er m a n e ntl y disc o nti n u e d at a n y 
ti m e. R e as o ns f or st u d y disc o nti n u ati o n m a y i ncl u d e, b ut ar e n ot li mit e d t o, t h e f oll o wi n g: 
 
a.  S af et y c o nc er ns  
b.  P o or e nr oll m e nt  
c. R e q u est t o disc o nti n u e t h e tri al b y a r e g ul at or y or h e alt h a ut h orit y or a n I R B  
d.  M a n uf a ct uri n g diffic ulti es / c o nc er ns  
 

Pr ot oc ol: M G T A -1 4 5 + pl eri x af or m o bili z ati o n  
 
Pr ot oc ol V ersi o n 1. 4 , 2 1 A pril  2 0 2 1  
3 7  
I R B-5 7 0 5 6  
 
4. 4  
 Alt e r n ati v es  
If p ati e nts d o n ot p artici p at e i n t his st u d y, t h e y m a y u n d er g o H S C m o biliz ati o n wit h t h e t w o c o m m o n 
st a n d ar d of c ar e str at e gi es t o a c hi e v e H S C c oll e cti o n  as d escri b e d i n S e cti o n 2. 1. 2.  T h es e i ncl u d e 
c h e m ot h er a p y -b as e d 
m o biliz ati o n (t y pic all y c ycl o p h os p h a m i d e) al o n g 
wit h G-C S F or G -C S F 
m o bili z ati o n wit h pl eri x af or, i n all p ati e nts or i n a ris k -a d a pti v e al g orit h m. 2 0  C ycl o p h os p h a mi d e 
m o bili z ati o n c a n t a k e t w o t o t hr e e w e e ks a n d r es ult i n c o m plic ati o ns li k e n e utr o p e nic f e v er a n d 
si g nific a nt c yt o p e ni as. 2 -4  G -C S F a n d pl eri x af or m o biliz ati o n r e q uir es u p t o 4 d a ys of G -C S F i nj e cti o ns 
b ef or e m o biliz ati o n c a n b e gi n ( Fi g u r e 1). I nj e cti o ns ar e c o nti n u e d t hr o u g h c oll e cti o n, w hic h c a n b e 
as l o n g as 4 s essi o ns, t h er ef or e r e q uiri n g 5 -8 d a ys f or c oll e cti o n. 5   
5  
I N V E S TI G A TI O N A L A G E N T I N F O R M A TI O N 
5. 1  
I n v e sti g ati o n al A g e nt M G T A -1 4 5  
C o m pl et e i nf or m a ti o n o n i n v esti g ati o n al st u d y a g e nt is pr o vi d e d i n t h e a cc o m p a n yi n g 
I n v e sti g at o r’s B r o c h u r e. T his i ncl u des  t h e m e c h a nis m of a cti o n, s u m m ari es of a ni m al a n d cli nic al 
st u di es, n o n -cli nic al a n d cli nic al p h ar m a c o ki n etics, m aj or r o ut e of eli mi n ati o n, s af et y pr o fil e, a n d 
t h e r ati o n al e f or t h e st arti n g d os e a n d r e gi m e n c h os e n. T his als o i ncl u d es i nf or m ati o n o n t h e 
m et a b olis m of M G T A -1 4 5  i n h u m a ns. 
5. 2  
A v ail a bilit y  
M G T A -1 4 5 i n v esti g ati o n al a g e nt will b e pr o vi d e d b y M a g e nt a T h er a p e utics. Pl eri x af or will b e 
a c q uir e d c o m m erci all y b as e d o n st a n d ar d i nstit uti o n al pr oc ur e m e nt f or p ati e nts u n d er g oi n g st e m 
c ell m o bili z ati o n. 
5. 3  
A g e nt O r d e ri n g  
M G T A -1 4 5 will b e d eli v er e d t o St a nf or d H e alt h C ar e I n v esti g ati o n al Dr u g P h ar m a c y b y M a g e nt a 
T h er a p e utics. T w o d os es of M G T A -1 4 5 m ust b e a v ail a bl e b ef or e att e m pt e d m o biliz ati o n f or a gi v e n 
p ati e nt o n t his pr ot oc ol. Pl eri x af or will b e o bt ai n e d c o m m erci all y p er i nstit uti o n al st a n d ar d 
pr oc e d ur es.  
 
S hi p pi n g a d dr ess of t h e i n v esti g ati o n al p h ar m a c y:  
St a nf or d H e alt h C ar e  
Att e nti o n: I n v esti g ati o n al P h ar m a c y  
 
St a nf or d, C A 9 4 3 0 5  
 
 
5. 4  
A g e nt A c c o u nt a bilit y / P r e p a r ati o n / A d mi ni st r ati o n  
T h e i n v esti g ati o n al a g e nt will b e s e c ur e d b y t h e St a nf or d H e alt h C ar e i n v esti g ati o n al p h ar m a c y p er 
st a n d ar d pr oc e d ur es.  
 

Pr ot oc ol: M G T A -1 4 5 + pl eri x af or m o bili z ati o n  
 
Pr ot oc ol V ersi o n 1. 4 , 2 1 A pril  2 0 2 1  
3 8  
I R B-5 7 0 5 6  
 
a.  F o r m ul ati o n:  M G T A -1 4 5 is f or m ul at e d at 2 0 m g / m L i n 2 0 m M s o di u m a c et at e, 1 5 0 m M 
s o di u m c hl ori d e, 0. 0 2 % ( w / v) P ol ys or b at e 8 0 at p H 4. 0. M G T A -1 4 5 is s u p pli e d i n a si n gl e -
d os e 2 m L T y p e I a m b er gl ass vi al. E a c h vi al c o nt ai ns n ot l ess t h a n 1 m L. M G T A -1 4 5 
(i nj e cti o n) is a st eril e s ol uti o n f or I V us e s u p pli e d as a si n gl e -d os e vi al p a c k a g e d i n a c art o n.  
 
b.  St o r a g e:  Vi als of M G T A -1 4 5 s h o ul d b e st or e d at -2 0° C ± 5° C i n t h e c art o n t o pr ot e ct fr o m 
li g ht.  Vi als of M G T A-1 4 5 s h o ul d b e t h a w e d o v er ni g ht at 4° C pri or t o us e a n d ar e st a bl e at 4° C 
f or u p t o 3 0 d a ys. O nc e t h a w e d, M G T A-1 4 5 s h o ul d n ot b e r efr oz e n.  
 
c. T h a wi n g:  T h e t h a wi n g will b e c o n d uct e d p er i n v esti g ati o n al p h ar m a c y st a n d ar d pr oc e d ur es 
a n d dr u g h a n d off t o St a nf or d H e alt h C ar e a p h er esis u nit at 8 7 5 Bl a k e Wil b ur Dri v e will occ ur 
i n t h e m or ni n g b ef or e a d mi nistr ati o n p er i nstit uti o n al pr oc e d ur es.   
 
d.  P r e p a r ati o n:  M G T A -1 4 5 will b e dil ut e d i n 0. 9 % s ali n e i n a cc or d a nc e wit h t h e p h ar m a c y 
m a n u al. S ol uti o ns of M G T A -1 4 5 ( 0. 0 3 6 t o 1. 4 4 m g / m L) pr e p ar e d i n 0. 9 % s ali n e m a y b e 
st or e d f or n o m or e t h a n 8 h o urs at r o o m t e m p er at ur e pri or t o a d mi nistr ati o n.  
 
e.  A d mi ni st r ati o n:  M G T A -1 4 5 will b e a d mi nist er e d as a n I V i nf usi o n o v er 3 t o 1 0 mi n ut es , as 
cli nic all y i n dic at e d.  
 
6  
D O S E M O DI FI C A TI O N S  
T h er e ar e n o pr o p os e d d os e m o dific ati o ns of M G T A -1 4 5 or pl eri x af or as d escri b e d i n s e cti o n 4 u n d er 
tr e at m e nt pl a n. If a n y p ati e nt e x p eri e nc es gr a d e 3 or hi g h er n o n -h e m at ol o gic al t o xicit y, n ot 
r e v ersi bl e t o gr a d e 1 or b as eli n e usi n g st a n d ar d of c ar e  i nt er v e nti o ns wit hi n 2 4  h o urs , a n d is 
c o nsi d er e d p ossi bl y, pr o b a bl y or d efi nit el y r el at e d t o M G T A -1 4 5, t h e st u d y dr u g will b e disc o nti n u e d 
a n d p ati e nt will g o t o e v e nt m o nit ori n g. T h e p ati e nt c a n c o nti n u e t o r e c ei v e  pl eri x af or if t h at is f elt 
a p pr o pri at e b y t h e tr e ati n g p h ysici a n. T h e p ati e nts will b e f oll o w e d f or 3 0 d a ys fr o m l ast d os e of 
M G T A -1 4 5 or till st art of tr a ns pl a nt c o n diti o ni n g, w hic h e v er c o m es first.  
 
7  
A D V E R S E E V E N T S A N D R E P O R TI N G P R O C E D U R E S  
7. 1  
P ot e nti al A d v e r s e E v e nt s   
7. 1. 1  M G T A -1 4 5   
 
St u d y 1 4 5 -H V -1 0 1, P h a s e 1 st u d y 
wit h 
M G T A -1 4 5 a n d pl e ri x af o r i n h e alt h y 
v ol u nt e e r s:  O v er all, 7 7 o ut of 1 0 7 s u bj e cts ( 6 5 w h o r e c ei v e d M G T A -1 4 5 a n d 1 2 w h o 
r e c ei v e d pl a c e b o) e x p eri e nc e d at l e ast 1 A E d uri n g t h e st u d y. T h e 
m ost fr e q u e nt 
tr e at m e nt-e m er g e nt a d v ers e e v e nts ( T E A Es) ( occ urri n g i n > 5 s u bj e cts) a m o n g s u bj e cts 
r a n d o miz e d t o r e c ei v e M G T A-1 4 5 w er e b ac k p ai n ( 4 8 s u bj e cts), n a us e a ( 1 3 s u bj e cts), 
diz zi n ess ( 1 3 s u bj e cts), di arr h e a ( 8 s u bj e cts), a n d h e a d a c h e ( 8 s u bj e cts). A t ot al of 6 3 
s u bj e cts e x p eri e nc e d a T E A E c o nsi d er e d b y t h e I n v esti g at or t o b e r el at e d t o M G T A -1 4 5. 

Pr ot oc ol: M G T A -1 4 5 + pl eri x af or m o bili z ati o n  
 
Pr ot oc ol V ersi o n 1. 4 , 2 1 A pril  2 0 2 1  
3 9  
I R B-5 7 0 5 6  
 
T h e m ost fr e q u e nt T E A Es ( occ urri n g i n > 1 s u bj e ct) w er e b ac k p ai n, di arr h e a, n a us e a, 
v o miti n g, f ati g u e, a b d o mi n al disc o mf ort, a b d o mi n al p ai n, i nj e cti o n sit e pr urit us,  
h e a d a c h e, p ar est h esi a, m usc ul os k el et al c h est p ai n, a n d diz zi n ess. T h e m aj orit y of all A Es 
r e p ort e d w er e d escri b e d as mil d ( gr a d e 1) i n N CI C T C A E s e v erit y, wit h o ut a n a p p ar e nt 
tr e n d a cr oss d os e l e v els or st u d y p art. T h e c o nsist e nt o bs er v ati o n ass oci at e d wit h M G T A-
1 4 5 i nf usi o n w as gr a d e 1 b o n e p ai n i n t h e b ac k t h at b e gi ns d uri n g t h e 1 0 -mi n ut e i nf usi o n 
a n d g e n er all y a p pr o xi m at el y 1 0 mi n ut es l at er (T a bl e 7 : S u m m ar y of Ac ut e B a c k P ai n ). 
All of t h e r e p ort e d A Es of b ac k p ai n w er e c o nsi d er e d b y t h e I n v esti g at or t o b e r el at e d t o 
st u d y tr e at m e nt.  
 
T a bl e 7 : S u m m a r y of A c ut e B a c k P ai n  (T a bl e s o u r c e : I n v esti g at o r’s B r o c h u r e) 
 
 
T h e k n o w n t o xiciti es of pl eri x af or w er e o bs er v e d a n d g e n er all y ass ess e d as mil d. F or t his 
st u d y, a d os e-li miti n g t o xicit y ( D L T) w as d efi n e d as a gr a d e 2 or hi g h er T E A E b as e d o n 
t h e N CI C T C A E v 5. 0 t h at is n ot a c o m m o nl y e x p e ct e d T E A E ass oci at e d wit h pl eri x af or 
(> 5 % of pl eri x af or a d mi nistr ati o ns) (s e e b el o w i n S e cti o n 7. 1. 2 ). 
 
T h er e w er e n o gr a d e 3 or gr a d e 4 T E A Es i n t h e st u d y. A D L T of 1 e v e nt of gr a d e 2 b ac k 
p ai n w as o bs er v e d at 0. 0 7 5 
m g / k g 
M G T A -1 4 5 
m g / k g, a d mi nist er e d 2 h o urs aft er 
pl eri x af or ( P art B). Aft er d os e r e d ucti o n t o 0. 0 7 0 m g / k g, n o D L Ts w er e o bs er v e d at t h at 
d os e or at s u bs e q u e nt d os es of 0. 0 3 0 m g / k g or 0. 0 1 5 m g / k g w h e n M G T A -1 4 5 w as 
a d mi nist er e d 2 h o urs aft e r pl eri x af or. N ot a bl y, gr a d e 2 b ac k p ai n w as n ot r e p ort e d f or 
s u bj e cts a d mi nist er e d M G T A -1 4 5 at 0. 0 7 5 a n d 0. 1 5 0 
m g / k g i m m e di at el y f oll o wi n g 
pl eri x af or i n P art B, or f or s u bj e cts a d mi nist er e d M G T A -1 4 5 at 0. 0 7 0 m g / k g o n 2 d a ys i n 
P art C.  
 
S e v e nt y -s e v e n s u bj e cts ( 6 5 i n a M G T A -1 4 5 gr o u p a n d 1 2 i n t h e pl a c e b o gr o u p) r e p ort e d 
at l e ast 1 gr a d e 1 T E A E; 4 s u bj e cts e x p eri e nc e d a m o d er at e ( gr a d e 2) e v e nt, a n d n o ≥ 
_ gr a d e 3 e v e nts w er e o bs er v e d. O n e s u bj e ct ( S u bj e ct 1 0 1-3 9 3) e x p eri e nc e d a n A E t h at l e d 

Pr ot oc ol: M G T A -1 4 5 + pl eri x af or m o bili z ati o n  
 
Pr ot oc ol V ersi o n 1. 4 , 2 1 A pril  2 0 2 1  
4 0  
I R B-5 7 0 5 6  
 
t o st u d y tr e at m e nt wit h dr a w al. T his gr a d e 2 e v e nt of b ac k p ai n occ urr e d f oll o wi n g 
a d mi nistr ati o n of 0. 0 7 5 m g / k g M G T A -1 4 5 w hi c h w as gi v e n 2 h o urs aft er pl eri x af or a n d 
m et t h e crit eri a f or a D L T p er t h e pr ot oc ol. As s uc h, a m a xi m u m d os e of 0. 0 7 m g / k g 
M G T A -1 4 5 T a bl e i n c o m bi n ati o n wit h pl eri x af or w as us e d as t h e hi g h est d os e f or t h e 
r e m ai n d er of t h e st u d y d osi n g. N o s u bj e ct i n t h e st u d y w as disc o nti n u e d fr o m a n M G T A -
1 4 5 st u d y d u e t o a n A E, a n d n o s eri o us a d v ers e e v e nts ( S A Es) or d e at hs w er e r e p ort e d.  
 
T h er e w er e n o c h a n g es i n a n y l a b or at or y i n v esti g ati o n t h at w er e ass ess e d as cli nic all y 
si g nific a nt. Is ol at e d el e v ati o ns i n bilir u bi n fr o m 1. 4 t o 3. 2 m g / d L ( n or m al 0 t o 1. 2 m g / d L) 
w er e s e e n o n D a ys 1 t o 3 i n 8 s u bj e cts ( 6 r e c ei v e d M G T A -1 4 5 + pl eri x af or, 1 r e c ei v e d 
pl a c e b o + pl eri x af or, 1 r e c ei v e d M G T A -1 4 5 al o n e). El e v ati o ns i n bilir u bi n ar e k n o w n t o 
b e ass oci at e d wit h pl eri x af or d osi n g. N o el e v ati o n w as ass ess e d as cli nic all y si g nific a nt, 
a n d all v al u es r et ur n e d t o n or m al b ef or e st u d y c o m pl eti o n. T h e 1 s u bj e ct w h o r e c ei v e d 
M G T A -1 4 5 al o n e i n P art A w h o h a d a n el e v at e d bilir u bi n t o 1. 4 m g / d L o n D a y 2 als o h a d 
a n el e v at e d l e v el of 1. 3 m g / d L at b as eli n e. T h us, t h e el e v ati o n o n D a y 2 w as n ot attri b ut e d 
t o st u d y dr u g. El e v ati o ns i n W B Cs a n d W B C s u bs ets ar e e x p e ct e d as p art of t h e P D a cti o n 
of M G T A -1 4 5 + pl eri x af or. Pr ot hr o m bi n ti m e / a cti v at e d p arti al t hr o m b o pl asti n ti m e 
( a P T T), fi bri n o g e n, a n d d-di m er t ests w er e a d d e d t o t h e l a b or at or y i n v esti g ati o ns f or t h e 
fi n al s u bj e cts i n t h e st u d y (s u bj e cts r e c ei vi n g M G T A-1 4 5 0. 0 1 5 m g / k g 2 h o urs aft er 
pl eri x af or i n P art B a n d P art D) aft er a s u bj e ct at t h e 0. 0 3 m g / k g d os e l e v el i n P art D f ail e d 
t o est a blis h a d e q u at e v e n o us fl o w f or a p h er esis aft er m o biliz ati o n. F or t h es e s u bj e cts 
r e c ei vi n g t h e M G T A-1 4 5 0. 0 1 5 m g / k g d os e 2 h o urs aft er pl eri x af or, tr a nsi e nt el e v ati o ns 
w er e s e e n i n d -di m er t o 0. 5 9 t o 1. 4 4 m g / L i n 5 of 9 s u bj e cts ( n or m al r a n g e 0 t o 0. 4 9 m g / L) 
6 h o urs aft er M G T A -1 4 5 i nf usi o n. D -di m er r et ur n e d t o n or m al i n all s u bj e cts at t h e n e xt 
o bs er v ati o n at 4 8 h o urs. Tr a nsi e nt c h a n g es i n d -di m er ar e c o nsist e nt wit h w h at is 
o bs er v e d d uri n g ot h er m o biliz ati o n r e gi m e ns. T h er e w as n o cli nic all y si g nific a nt c h a n g e 
i n vit al si g ns wit h M G T A-1 4 5 d osi n g. Tr a nsi e nt el e v ati o ns i n s yst olic bl o o d pr ess ur e ≥ 
1 4 0 m m H g w er e n ot e d i n 1 0 s u bj e cts w h o e x p eri e nc e d b ac k p ai n.  
 
C h a n g es i n el e ctr oc ar di o gr a m ( E C G) 
m e as ur e m e nts w er e c o nsi d er e d n ot cli nic all y 
si g nific a nt, a n d n o cli nic all y i m p ort a nt i ncr e as e i n t h e fr e q u e nc y of Q Tc a b n or m aliti es 
aft er i niti ati o n of tr e at m e nt w as o bs er v e d.  
 
O v er all, t h e i nci d e nc e of A Es w as si mil ar b et w e e n t h e tr e at m e nt c o h orts. N o i ncr e as e i n 
s e v erit y or fr e q u e nc y of a d v ers e eff e cts ass oci at e d wit h pl eri x af or w as o bs er v e d w h e n it 
w as gi v e n i n c o m bi n ati o n wit h M G T A -1 4 5.  
 
Pl e as e r ef er t o a cc o m p a n yi n g I n v esti g at or’s Br oc h ur e f or a d et ail e d d escripti o n of a d v ers e 
e v e nts i n e a c h of t h e 4 ar ms of t h e st u d y as w ell as A Es s e e n i n t h e o n g oi n g st u d y of M G T A -
1 4 5 i n p ati e nts wit h r e n al i m p air m e nt. 
 
 

Pr ot oc ol: M G T A -1 4 5 + pl eri x af or m o bili z ati o n  
 
Pr ot oc ol V ersi o n 1. 4 , 2 1 A pril  2 0 2 1  
4 1  
I R B-5 7 0 5 6  
 
7. 1. 2  Pl e ri x af o r :  
T h e p ot e nti al a d v ers e e v e nt ass oci at e d wit h pl eri x af or as o bt ai n e d fr o m t h e F D A a p pr o v e d 
p a c k a g e i n s e rt  ar e as f oll o ws:  
 
Cli ni c al T ri al s E x p e ri e n c e: T h e m ost c o m m o n a d v ers e r e a cti o ns ( ≥1 0 %) r e p ort e d i n 
p ati e nts w h o r e c ei v e d pl eri x af or i n c o nj u ncti o n wit h G -C S F r e g ar dl ess of c a us alit y a n d 
m or e fr e q u e nt wit h pl eri x af or t h a n pl a c e b o d uri n g H S C m o biliz ati o n a n d a p h er esis w er e 
di arr h e a, n a us e a, f ati g u e, i nj e cti o n sit e r e a cti o ns, h e a d a c h e, art hr al gi a, diz zi n ess, a n d 
v o miti n g.  
 
S af et y d at a f or pl eri x af or i n c o m bi n ati o n wit h G -C S F w er e o bt ai n e d fr o m t w o r a n d o miz e d 
pl a c e b o -c o ntr oll e d st u di es ( 3 0 1 p ati e nts) a n d 1 0 u nc o ntr oll e d st u di es ( 2 4 2 p ati e nts). 
P ati e nts w er e pri m aril y tr e at e d wit h Pl eri x af or at d ail y d os es of 0. 2 4 m g / k g S C. M e di a n 
e x p os ur e t o Pl eri x af or i n t h es e st u di es w as 2 d a ys (r a n g e 1 t o 7 d a ys).  
 
I n t h e t w o r a n d o miz e d st u di es i n p ati e nts with N H L a n d M M, a t ot al of 3 0 1 p ati e nts w er e 
tr e at e d i n t h e Pl eri x af or a n d G-C S F gr o u p a n d 2 9 2 p ati e nts w er e tr e at e d i n t h e pl a c e b o 
a n d G -C S F gr o u p. P ati e nts r e c ei v e d d ail y m or ni n g d os es of G -C S F 1 0 micr o gr a ms / k g f or 
4 d a ys pri or t o t h e first d os e of Pl eri x a f or 0. 2 4 m g / k g S C or pl a c e b o a n d o n e a c h m or ni n g 
pri or t o a p h er esis. T h e a d v ers e r e a cti o ns t h at occ urr e d i n ≥5 % of t h e p ati e nts w h o 
r e c ei v e d Pl eri x af or r e g ar dl ess of c a us alit y a n d w er e m or e fr e q u e nt wit h Pl eri x af or t h a n 
pl a c e b o d uri n g H S C m o biliz ati o n a n d a p h er esis ar e s h o w n i n T a bl e 8 .  
 
I n t h e r a n d o miz e d st u di es, 3 4 % of p ati e nts wit h N H L or M M h a d mil d t o m o d er at e 
i nj e cti o n sit e r e a cti o n s at t h e sit e of s u bc ut a n e o us a d mi nistr ati o n of pl eri x af or. T h es e 
i ncl u d e d er yt h e m a, h e m at o m a, h e m orr h a g e, i n d ur ati o n, i nfl a m m ati o n, irrit ati o n, p ai n, 
p ar est h esi a, pr urit us, r as h, s w elli n g, a n d urtic ari a.  
 
Mil d t o m o d e r at e all e r gi c r e a cti o n s  w er e o bs er v e d i n l ess t h a n 1 % of p ati e nts wit hi n 
a p pr o xi m at el y 3 0 mi n aft er Pl eri x af or a d mi nistr ati o n, i ncl u di n g o n e or m or e of t h e 
f oll o wi n g: urtic ari a ( n = 2), p eri or bit al s w elli n g ( n = 2), d ys p n e a ( n = 1) or h y p o xi a ( n = 1). 
S y m pt o ms g e n er all y r es p o n d e d t o tr e at m e nts ( e. g., a nti hist a mi n es, c ortic ost er oi ds, 
h y dr ati o n or s u p pl e m e nt al o x y g e n) or r es ol v e d s p o nt a n e o usl y.  
 
V a s o v a g al r e a cti o n s, o rt h o st ati c h y p ot e n si o n, a n d / o r s y n c o p e  c a n occ ur f oll o wi n g 
s u bc ut a n e o us i nj e cti o ns. I n pl eri x af or o nc ol o g y a n d h e alt h y v ol u nt e er cli nic al st u di es, 
l ess t h a n 1 % of s u bj e cts e x p eri e nc e d v as o v a g al r e a cti o ns f oll o wi n g s u bc ut a n e o us 
a d mi nistr ati o n of Pl eri x af or d os es ≤0. 2 4 m g / k g. T h e m aj orit y  of t h es e e v e nts occ urr e d 
wit hi n 1 h o ur of Pl eri x af or a d mi nistr ati o n. B e c a us e of t h e p ot e nti al f or t h es e r e a cti o ns, 
a p pr o pri at e pr e c a uti o ns s h o ul d b e t a k e n.  
 
Ot h er a d v ers e r e a cti o ns i n t h e r a n d o miz e d st u di es t h at occ urr e d i n < 5 % of p ati e nts b ut 
w er e r e p ort e d as r el at e d t o pl eri x af or d uri n g H S C m o biliz ati o n a n d a p h er esis i ncl u d e d 
a b d o mi n al p ai n, h y p er hi dr osis, a b d o mi n al dist e nti o n, dr y m o ut h, er yt h e m a, st o m a c h 

Pr ot oc ol: M G T A -1 4 5 + pl eri x af or m o bili z ati o n  
 
Pr ot oc ol V ersi o n 1. 4 , 2 1 A pril  2 0 2 1  
4 2  
I R B-5 7 0 5 6  
 
disc o mf ort, m al ais e, h y p o est h esi a or al, c o nsti p ati o n, d ys p e psi a, a n d m usc ul os k el et al 
p ai n.  
 
H y p e rl e u k o c yt o si s: I n cli ni c al tri als, w hit e bl o o d c ell c o u nts of 1 0 0, 0 0 0 / mc L or gr e at er 
w er e o bs er v e d, o n t h e d a y pri or t o or a n y d a y of a p h er esis, i n 7 % of p ati e nts r e c ei vi n g 
pl eri x af or a n d i n 1 % of p ati e nts r e c ei vi n g pl a c e b o. N o c o m plic ati o ns or cli nic al s y m pt o ms 
of l e u k ost asis w er e o bs er v e d.  
 
P o st -m a r k eti n g E x p e ri e n c e: I n a d diti o n t o a d v ers e r e a cti o ns r e p ort e d fr o m cli nic al 
tri als, t h e f oll o wi n g a d v ers e r e a cti o ns h a v e b e e n r e p ort e d fr o m p ost m ar k eti n g 
e x p eri e nc e wit h Pl eri x af or. B e c a us e t h es e r e a cti o ns ar e r e p o rt e d v ol u nt aril y fr o m a 
p o p ul ati o n of u nc ert ai n si z e, it is n ot al w a ys p ossi bl e t o r eli a bl y esti m at e t h eir fr e q u e nc y 
or est a blis h a c a us al r el ati o ns hi p t o dr u g e x p os ur e.  
•  
Bl o o d a n d L y m p h atic S yst e m: S pl e n o m e g al y a n d s pl e nic r u pt ur e  
•  
I m m u n e S yst e m Dis or d ers: A n a p h yl a ctic r e a cti o ns, i ncl u di n g a n a p h yl a ctic s h oc k  
•  
Ps yc hi atric Dis or d ers: A b n or m al dr e a ms a n d ni g ht m ar es  
 

Pr ot oc ol: M G T A -1 4 5 + pl eri x af or m o bili z ati o n  
 
Pr ot oc ol V ersi o n 1. 4 , 2 1 A pril  2 0 2 1  
4 3  
I R B-5 7 0 5 6  
 
T a bl e 8 : A d v e r s e R e a cti o n s i n ≥5 % of N o n-H o d g ki n' s L y m p h o m a a n d M ulti pl e M y el o m a 
P ati e nt s R e c ei vi n g Pl e ri x af o r a n d M o r e F r e q u e nt t h a n Pl a c e b o d u ri n g H S C 
M o bili z ati o n a n d A p h e r esi s  
 
 
7. 2  
A d v e r s e E v e nt a n d S e ri o u s A d v e r s e E v e nt R e p o rti n g  
7. 2. 1  D efi niti o n s  
A d v e r s e E v e nt:  
A n a d v ers e e v e nt ( A E) is d efi n e d as a n y r e a cti o n, si d e eff e ct, or u nt o w ar d e v e nt t h at occ urs d uri n g 
t h e c o urs e of th e cli nic al tri al ass oci at e d wit h t h e us e of a dr u g i n h u m a ns, w h et h er or n ot t h e e v e nt 
is c o nsi d er e d r el at e d t o t h e tr e at m e nt or cli nic all y si g nific a nt.  F or t his st u d y, A Es will i ncl u d e e v e nts 
r e p ort e d b y t h e s u bj e ct, as w ell as cli nic all y si g nific a nt a b n or m al fi n di n gs o n p h ysic al e x a mi n ati o n or 
l a b or at or y e v al u ati o n.  A n e w ill n ess, s y m pt o m, si g n or cli nic all y si g nific a nt l a b or at or y a b n or m alit y 
or w ors e ni n g of a pr e -e xisti n g c o n diti o n or a b n or m alit y is c o nsi d er e d a n A E.  All a d v ers e e v e nts will 
b e gr a d e d  a cc or di n g t o N CI C T C A E v 5. 0.  
T o xiciti es pr es e nt at t h e i niti ati o n of st u d y dr u g will b e c o nsi d er e d b as eli n e c o n diti o ns.  

Pr ot oc ol: M G T A -1 4 5 + pl eri x af or m o bili z ati o n  
 
Pr ot oc ol V ersi o n 1. 4 , 2 1 A pril  2 0 2 1  
4 4  
I R B-5 7 0 5 6  
 
S e ri o u s A d v e r s e E v e nt  
A n A E or s us p e ct e d a d v ers e r e a cti o n is c o nsi d er e d s eri o us if i n t h e vi e w of t h e i n v esti g at or or t h e 
s p o ns or, it r es ults i n a n y of t h e f oll o wi n g:  
•  
D e at h,  
•  
A lif e -t hr e at e ni n g a d v ers e dr u g e x p eri e nc e 
•  
I n-p ati e nt h os pit aliz ati o n or pr ol o n g ati o n of e xisti n g h os pit aliz ati o n [ N ot e: H os pit aliz ati o ns 
r el at e d t o sc h e d uli n g iss u es ( e. g.  e v e ni n g or w e e k e n d e v al u ati o ns) a n d c o n v e ni e nc e 
c o nsi d er ati o ns d o n ot c o nstit ut e a s eri o us a d v ers e e v e nt.]  
•  
P ersist e nt or si g nific a nt i nc a p a cit y or s u bst a nti al disr u pti o n of t h e a bilit y t o c o n d uct n or m al 
lif e f u ncti o ns 
•  
A c o n g e nit al a n o m al y / birt h d ef e ct.  
•  
I mp ort a nt m e dic al e v e nts t h at m a y n ot r es ult i n d e at h, b e lif e -t hr e at e ni n g, or r e q uir e 
h os pit aliz ati o n m a y b e c o nsi d er e d a s eri o us a d v ers e dr u g e x p eri e nc e w h e n, b as e d u p o n 
a p pr o pri at e m e dic al j u d g m e nt, t h e y m a y j e o p ar diz e t h e s u bj e ct or s u bj e ct a n d m a y r e q ui r e 
m e dic al or s ur gic al i nt er v e nti o n t o pr e v e nt o n e of t h e o utc o m es list e d i n t his d efi niti o n.  
 
7. 2. 2  S e ri o u s A d v e r s e E v e nt R e p o rti n g  
A d v ers e e v e nts a n d S eri o us A d v ers e E v e nts i n t his st u d y will b e gr a d e d f or s e v erit y a cc or di n g t o 
N ati o n al C a nc er I nstit ut e C o m m o n  T er mi n ol o g y Crit eri a f or A d v ers e E v e nts v ersi o n 5. 0 ( C T C A E 
v 5. 0 ).   
 
B ot h S eri o us a n d N o n -S eri o us A d v ers e E v e nts will b e cl e arl y n ot e d i n s o urc e d oc u m e nt ati o n a n d 
list e d o n st u d y s p e cific C as e R e p ort F or ms ( C R Fs).  T h e Pr ot oc ol Dir e ct or ( P D) or d esi g n e e will ass ess 
e a c h S eri o us A d v ers e E v e nt ( S A E) t o d et er mi n e w h et h er it is u n e x p e ct e d a cc or di n g t o t h e R ef er e nc e 
S af et y I nf or m ati o n s e cti o n of t h e I n v esti g at or’s Br oc h ur e f or MG T A -1 4 5 or t h e p a c k a g e i ns ert f or 
pl eri x af or .  
 
All S eri o us A d v ers e E v e nts ( S A Es) will b e tr ac k e d fr o m pr e-m o biliz ati o n e v al u ati o n ( b as eli n e A E 
ass ess m e nt, c a n occ ur u p t o 7  d a ys b ef or e m o biliz ati o n as d escri b e d i n sc h e d ul e of e v e nts)  u ntil 
r es ol uti o n, or u ntil 3 0 d a ys aft er t h e l ast d os e of t h e st u d y i nt er v e nti o n i. e. m o biliz ati o n a g e nts . St u d y 
i nt er v e nti o n r el at e d A Es / S A Es s h o ul d c o nti n u e t o b e f oll o w e d u ntil r es ol uti o n or st a biliz ati o n.  A n y 
S A E occ urri n g aft er t h e r e p orti n g p eri o d m ust b e pr o m ptl y r e p ort e d if a c a us al r el ati o ns hi p t o t h e 
i n v esti g ati o n al dr u g is s us p e ct e d.   
 
W h e n ass essi n g w h et h er a n a d v ers e e v e nt is r el at e d t o t h e i n v esti g ati o n al i nt er v e nti o n, t h e f oll o wi n g 
attri b uti o n c at e g ori es ar e utiliz e d:  
 
D efi nit e - T h e a d v ers e e v e nt is cl e arl y r el at e d t o t h e a g e nt(s).  
Pr o b a bl e - T h e a d v ers e e v e nt is li k el y r el at e d t o t h e a g e nt(s).  
P ossi bl e - T h e a d v ers e e v e nt m a y b e r el at e d t o t h e a g e nt(s).  

Pr ot oc ol: M G T A -1 4 5 + pl eri x af or m o bili z ati o n  
 
Pr ot oc ol V ersi o n 1. 4 , 2 1 A pril  2 0 2 1  
4 5  
I R B-5 7 0 5 6  
 
U nli k el y - T h e a d v ers e e v e nt is d o u btf ull y r el at e d t o t h e a g e nt(s).  
U nr el at e d - T h e a d v ers e e v e nt is cl e arl y N O T r el at e d t o t h e a g e nt(s).  
 
F or t h e p ur p os e of r e g ul at or y r e p orti n g r e q uir e m e nts, c a us al r el ati o ns hi ps of d efi nit e, pr o b a bl e, a n d 
p ossi bl e will b e c o nsi d er e d tr e at m e nt r el at e d, w hil e u nli k el y a n d u nr el at e d will b e c o nsi d er e d n ot 
tr e at m e nt r el at e d.  
 
S A Es C T C A E Gr a d e 3 a n d a b o v e, a n d all s u bs e q u e nt f oll o w -u p r e p orts will b e r e p ort e d t o t h e St a nf or d 
C a nc er I nstit ut e D at a a n d S af et y M o nit ori n g C o m mitt e e ( D S M C) usi n g t h e st u d y s p e cific C R F 
r e g ar dl ess of t h e e v e nt’s r el at e d n ess t o t h e i n v esti g ati o n al pr o d uct . F oll o wi n g r e vi e w b y  t h e D S M C, 
e v e nts m e eti n g t h e I R B d efi niti o n of ‘ U n a ntici p at e d Pr o bl e m’ will b e r e p ort e d t o t h e I R B usi n g 
e Pr ot oc ol wit hi n 1 0 w or ki n g d a ys of D S M C r e vi e w, or wit hi n 5 w or ki n g d a ys f or d e at hs or lif e -
t hr e at e ni n g e x p eri e nc es.  
 
All S A Es  occ urri n g d uri n g st e m  c ell m o biliz ati o n a n d u p t o p ost -m o biliz ati o n  visit , r e g ar dl ess of 
r el ati o ns hi p t o st u d y tr e at m e nt, m ust b e r e p ort e d i m m e di at el y t o M a g e nt a  T h er a p e utics  wit h t h e 
ti m efr a m e n ot t o e xc e e d 2 4 h o urs of t h e I n v esti g at or b ec o mi n g a w ar e of t h e e v e nt.  I niti al S A E 
n otific ati o n s h o ul d b e m a d e b y e m aili n g or f a xi n g t h e S A E r e p ort f or m t o M a g e nt a T h er a p e utics at 
t h e e m ail or f a x n u m b er pr o vi d e d b y M a g e nt a T h er a p e utics. A n y pr e g n a nc y i n a f e m al e p ati e nt or 
p art n er of a m al e p ati e nt m ust als o b e r e p ort e d t o M a g e nt a  T h er a p e utics , wit hi n 2 4 h o urs of t h e 
i n v esti g at or b e c o mi n g a w ar e of t h e e v e nt usi n g a Pr e g n a nc y R e p ort F or m.   
 
S A Es occ urri n g aft er t h e p ati e nt disc o nti n u es M G T A -1 4 5, i. e. b e y o n d t h e p ost -m o biliz ati o n visit, will 
b e c oll e ct e d a n d r e p ort e d a cc or di n g t o a b o v e pr ot oc ol if t h e y ar e  at l e ast p ossi bl y r el at e d t o st u d y 
a g e nt, a n d h a v e n ot b e e n pr e vi o usl y r e p ort e d ( u n e x p ect e d).  
 
As st u d y s p o ns or, th e I n v esti g at or is r es p o nsi bl e f or all c o m m u nic ati o ns wit h t h e F D A. T h e 
I n v esti g at or will r e p ort t o t h e F D A, a n y s eri o us a d v ers e e v e nt t h at m e ets t h e F D A’s crit eri a f or 
e x p e dit e d r e p orti n g f oll o wi n g r e p orti n g r e q uir e m e nts a n d ti m eli n es s et b y t h e F D A.  
 
M a g e nt a will n otif y I n v esti g at ors of all s us p e ct e d u n e x p e ct e d s eri o us a d v ers e r e a cti o ns ( S U S A Rs 
7 / 1 5 D a y S af et y R e p orts) t h at occ ur d uri n g a n y cli nic al st u di es t h at ar e usi n g t h e i n v esti g ati v e 
c o m p o u n d. E a c h sit e is r es p o nsi bl e f or n otif yi n g t h eir I R B / E C of t h es e a d diti o n al S U S A Rs i n 
a cc or d a nc e wit h l oc al r e g ul ati o ns.  
7. 2. 3  B a s eli n e a n d F oll o w -u p A d v e r s e E v e nt E v al u ati o n s : 
 
a.  A d v ers e e v e nts t o b e gr a d e d at b as eli n e, aft er e a c h d os e of M G T A -1 4 5, i n a ti m el y m a n n er of 
st u d y i nt er v e nti o n f or st e m c ell m o biliz ati o n  a n d 3 0 d a ys aft er t h e l ast d os e of st u d y 
i nt er v e nti o n, or u ntil i niti ati o n of tr a ns pl a nt pr oc e d ur es, w hic h e v er is first. If t h e A E occ urs 
aft er i niti ati o n of tr a ns pl a nt pr oc e d ur es b ut b ef or e t h e c o ncl usi o n of t h e 3 0 d a ys, A Es will b e 
c oll e ct e d if t h e y ar e s eri o us a n d  p ossi bl y, pr o b a bl y or d efi nit el y r el at e d t o t h e st u d y a g e nt. 

Pr ot oc ol: M G T A -1 4 5 + pl eri x af or m o bili z ati o n  
 
Pr ot oc ol V ersi o n 1. 4 , 2 1 A pril  2 0 2 1  
4 6  
I R B-5 7 0 5 6  
 
Tr a ns pl a nt r el at e d A Es will N O T b e c oll e ct e d o n t his st u d y.  
 
b.  D e at h wit hi n 3 0 d a ys of l ast i n v esti g ati o n al a g e nt a n d d e at h at a n y ti m e  at l e ast p ossi bl y 
r el at e d t o st u d y a g e nt will als o b e r e p ort e d o n t h e E v e nt M o nit ori n g F or m  a n d r e p ort e d t o 
D S M C, M a g e nt a  T h er a p e utics  usi n g a n S A E F or m  a n d t h e F D A .  
 
c. A d v ers e e v e nts will b e gr a d e d a cc or di n g t o C o m m o n T er mi n ol o g y Crit eri a f or A d v ers e E v e n ts 
( C T C A E) v ersi o n 5. 0 gr a di n g u nl ess ot h er wis e st at e d.  
 
d.  T h e f oll o wi n g pr e -tr e at m e nt s y m pt o ms / c o n diti o ns (T a bl e 9 ) ar e t o b e gr a d e d at b as eli n e 
a n d a d v ers e e v e nts ar e t o b e gr a d e d at e a c h e v al u ati o n usi n g C T C A E v 5. 0 gr a di n g  ( d oc u m e nt 
0 if a bs e nt) .  
 
T a bl e 9 : A d v e r s e E v e nt A s s es s m e nt   
C T C A E  S Y S T E M /  
O R G A N / C L A S S  
A d v ers e e v e nt / S y m pt o ms  
B as eli n e  
M o biliz ati o n 
D a ys  
P ost -
m o biliz ati o n  
I n v esti g ati o ns 
Cr e ati ni n e i ncr e as e d  
X  
X  
X  
N e utr o p hil c o u nt 
 
X  
X  
X  
N e utr o p hil c o u nt 
 
X  
X  
X  
Pl at el et c o u nt d e cr e as e d  
X  
X  
X  
A L T i ncr e as e d  
X  
X  
X  
A S T i ncr e as e d  
X  
X  
X  
Bilir u bi n i ncr e as e d  
X  
X  
X  
G e n er al dis or d ers a n d  F ati g u e  
X  
X  
X  
Fl u li k e s y m pt o ms  
X  
X  
X  
P ai n  
X  
X  
X  
I nj e cti o n sit e r e a cti o n 
X  
X  
X  
Pr oc e d ur al 
 
I nf usi o n r el at e d r e a cti o n 
X  
X  
X  
G astr oi nt esti n al 
Dis or d ers  
N a us e a  
X  
X  
X  
V o miti n g  
X  
X  
X  
A b d o mi n al P ai n  
X  
X  
X  
B as eli n e n u m b er of st o ols  
X  
 
 

Pr ot oc ol: M G T A -1 4 5 + pl eri x af or m o bili z ati o n  
 
Pr ot oc ol V ersi o n 1. 4 , 2 1 A pril  2 0 2 1  
4 7  
I R B-5 7 0 5 6  
 
 
e.  T h e f oll o wi n g A Es e x p eri e nc e d b y a p ati e nt a n d n ot s p e cifi e d i n S e cti o n 7. 2. 2 will b e 
r e c or d e d i n t h e st u d y d at a b as e usi n g C as e R e p ort F or ms . 
o  Gr a d e 2 A Es d e e m e d p ossi bl y, pr o b a bl y, or d efi nit el y r el at e d t o t h e st u d y tr e at m e nt or 
pr oc e d ur e  at a b o v e d efi n e d ti m e p oi nts.  
o  Gr a d e 3 a n d 4 A Es r e g ar dl ess of attri b uti o n t o t h e st u d y tr e at m e nt or pr oc e d ur e  at 
a b o v e d efi n e d ti m e p oi nts.  
o  Gr a d e 5 A Es ( D e at hs). A n y d e at h wit hi n 3 0 d a ys of t h e p ati e nt’s l ast st u d y i nt er v e nti o n 
r e g ar dl ess of attri b uti o n t o t h e st u d y a g e nt . A n y d e at h m or e t h a n 3 0 d a ys aft er t h e 
p ati e nt’s l ast st u d y a g e nt  t h at is f elt t o b e at l e ast p ossi bl y r el at e d t o t h e st u d y a g e nt  
m ust als o b e r e p ort e d as a Gr a d e 5 A E, wit h t h e r e as o n f or d e at h, a n d attri b uti o n 
assi g n e d.  
8  
L A B O R A T O R Y C O R R E L A TI V E / S P E CI A L S T U DI E S  
8. 1  
 ( A p h er esis Pr o d uct C o m p ositi o n –  L a b or at or y C orr el ati v e St u d y # 1 a , # 1 b , # 1c) 
8. 1. 1  
C oll e cti o n of S p e ci m e n(s) : T w o 2 -4 m L s a m pl e s of cr y o pr es er v e d p eri p h er all y m o bili z e d 
a p h er esis pr o d uct will t a k e n fr o m t h e m o biliz e d fr es h pr o d uct  fr o m t h e first d a y of 
a p h er esis d eli v er e d t o t h e St a nf or d B M T C ell ul ar T h er a p y F a cilit y ( C T F) . A t hir d fr es h 
s a m pl e ( 2-3 m L) will b e c oll e ct e d f or mi ni m al r esi d u al dis e as e a n al ysis . 
8. 1. 2  
H a n dli n g of S p e ci m e ns(s) : S a m pl es will b e pr oc ess e d , ali q u ot e d a n d cr y o pr es er v e d i n t h e 
C ell ul ar T h er a p y F a cilit y at St a nf or d a n d st or e d i n li q ui d nitr o g e n u ntil r e a d y f or b atc h e d 
a n al ysis . F or M R D t esti n g (st u d y # 1c), fr es h s a m pl e will b e ali q u ot e d i n a E D T A ( p ur pl e 
t o p) t u b e t o b e s hi p p e d t o M a y o Cli nic f or pr oc essi n g a n d a n al ysis. 
8. 1. 3  
S hi p pi n g of S p e ci m e n(s) : C r y o pr es er v e d s a m pl es will b e h a n d d eli v er e d  Dr. S hiz ur u’s  l a b 
f or a n al ysis f or fl o w c yt o m etr y a n d H u m a n I m m u n e M o nit ori n g Ce nt er at St a nf or d f or 
C y T O F a n al ysis.  S a m pl es will b e t h a w e d p er l a b or at or y pr ot oc ol pri or t o b atc h e d t esti n g.  
Fr es h s a m pl e ( d e -i d e ntifi e d) f or M R D t esti n g will b e s hi p p e d o v er ni g ht i n a st yr of o a m 
c o nt ai n er wit h c hill e d or a m bi e nt p a c ks.  
Di arr h e a  
X  
X  
X  
Bl o o d a n d l y m p h atic 
 
 
A n e mi a  
X  
X  
X  
M usc ul os k el et al  
B o n e P ai n  
X  
X  
X  
B a c k P ai n  
X  
X  
X  
Art hr al gi a  
X  
X  
X  
M uscl e Cr a m p  
X  
X  
X  
N er v o us S yst e m 
Dis or d ers  
P ar est h esi as  
X  
X  
X  
H e a d a c h e  
X  
X  
X  
Diz zi n ess  
X  
X  
X  

Pr ot oc ol: M G T A -1 4 5 + pl eri x af or m o bili z ati o n  
 
Pr ot oc ol V ersi o n 1. 4 , 2 1 A pril  2 0 2 1  
4 8  
I R B-5 7 0 5 6  
 
8. 1. 4   
Sit e(s) P erf or mi n g C orr el ati v e St u d y : St a nf or d U ni v ersit y ( S hiz ur u l a b a n d H u m a n 
I m m n u e M o nit ori n g Ce nt er - HI M C ), M a y o Cli nic ( S h aji K u m ar l a b) f or M R D t esti n g. 
8. 1. 5  
C o di n g of s p e ci m e ns f or pri v a c y pr ot e cti o n : F or s p e ci m e ns b ei n g t est e d i nt er n all y at 
St a nf or d U ni v ersit y: s p e ci m e ns m a y  b e l a b el e d wit h p ati e nt’s n a m e, m e dic al r e c or d 
n u m b er a n d st u d y I D.  F or s a m pl es t o b e  t o b e s e nt o utsi d e of St a nf or d U ni v ersit y, P HI 
i nf or m ati o n will b e r e m o v e d a n d s a m pl es will b e l a b el e d wit h p ati e nts i niti als a n d St u d y 
I D o nl y. T h e l att er c o n v e nti o n (st u d y I D a n d i niti als) m a y b e a d o pt e d f or all i nt er n al 
s a m pl es as w ell. 
 
8. 2  
 ( C F U a s s a y–  L a b o r at o r y C o r r el ati v e St u d y # 2)  
8. 2. 1  
C oll e cti o n of S p e ci m e n(s) : S a m pl es of p eri p h er all y m o biliz e d a p h er esis pr o d uct will t a k e n 
fr o m t h e m o biliz e d pr o d uct d eli v er e d t o t h e C T F fr o m a p h er esis u nit. C F U c a n b e d o n e o n 
b ot h fr es h a n d t h a w e d s a m pl es. Fr es h s a m pl es will b e us e d. C F U t esti n g p er St a nf or d B M T 
C T F F a cilit y.  
8. 2. 2  
H a n dli n g of S p e ci m e ns(s) : Pr oc essi n g a n d t esti n g will b e d o n e i n t h e St a nf or d B M T C T F 
l a b p er i nstit uti o n al S O P.  
8. 2. 3  
S hi p pi n g of S p e ci m e n(s) : N o n e 
8. 2. 4  
Sit e(s) P erf or mi n g C orr el ati v e St u d y : St a nf or d B M T C ell ul ar T h er a p y F a cilit y  
8. 2. 5  
C o di n g of s pe ci m e ns f or pri v a c y pr ot e cti o n: S p e ci m e ns will b e l a b el e d wit h p ati e nt’s n a m e, 
m e dic al r e c or d n u m b er a n d st u d y I D. S a m pl es will b e a n al y z e d wit hi n St a nf or d U ni v ersit y. 
I n c as e s a m pl es n e e d t o b e s e nt o utsi d e of St a nf or d U ni v ersit y, P HI i nf or m ati o n will be 
r e m o v e d a n d s a m pl es will b e l a b el e d wit h p ati e nts i niti als a n d St u d y I D o nl y. T h e l att er 
c o n v e nti o n (st u d y I D a n d i niti als) m a y b e a d o pt e d f or all i nt er n al s a m pl es as w ell.  
 
8. 3  
 (I m m u n e R e c o n stit uti o n at d a y 2 8 a n d 1 0 0  b y fl o w c yt o m et r y  a n d at d a y 1 0 0  b y C y T O F 
–  L a b o r at o r y C o r r el ati v e St u d y # 3 a a n d 3 b ) 
 
8. 3 . 1 
C oll e cti o n of S p e ci m e n(s) : P eri p h er al bl o o d s a m pl es, t w o ali q u ots of 3-4 m L e a c h  will b e 
c oll e ct e d i n c o nj u ncti o n wit h t h e cli nic al l a b a n d / or cli nic al tri als r es e arc h u nit ( C T R U ). 
S a m pl es will b e d eli v er e d t o t h e C T F . 
8. 3 . 2 
H a n dli n g of S p e ci m e ns(s) : S a m pl es will b e pr oc ess e d at t h e  C ell ul ar T h er a p y F a cilit y, 
w h er e t h e y will b e st or e d u ntil r e a d y f or b atc h e d a n al ysis b y  t h e S hiz ur u l a b  a n d HI M C . 
8. 3 . 3 
S hi p pi n g of S p e ci m e n(s) : N o n e, s a m pl es will b e h a n d d eli v er e d t o Dr. S hiz ur u’s l a b f or 
a n al ysis f or fl o w c yt o m etr y a n d H u m a n I m m u n e M o nit ori n g C e nt er at St a nf or d f or C y T O F 
a n al ysis.  
8. 3 . 4  
Sit e(s) P erf or mi n g C orr el ati v e St u d y : St a nf or d u ni v ersit y S hiz ur u L a b, St a nf or d HI M C8. 1.  
8. 3. 5  
C o di n g of s p e ci m e ns f or pri v a c y pr ot e cti o n: S p e ci m e ns will b e l a b el e d wit h p ati e nt’s n a m e, 
m e dic al r e c or d  n u m b er a n d st u d y I D. S a m pl es will b e a n al y z e d wit hi n St a nf or d U ni v ersit y. 
I n c as e s a m pl es n e e d t o b e s e nt o utsi d e of St a nf or d U ni v ersit y, P HI i nf or m ati o n will b e 

Pr ot oc ol: M G T A -1 4 5 + pl eri x af or m o bili z ati o n  
 
Pr ot oc ol V ersi o n 1. 4 , 2 1 A pril  2 0 2 1  
4 9  
I R B-5 7 0 5 6  
 
r e m o v e d a n d s a m pl es will b e l a b el e d wit h p ati e nts i niti als a n d St u d y I D o nl y. T h e l att er 
c o n ve nti o n (st u d y I D a n d i niti als) m a y b e a d o pt e d f or all i nt er n al s a m pl es as w ell.  
 

Pr ot oc ol: M G T A -1 4 5 + pl eri x af or m o bili z ati o n  
 
Pr ot oc ol V ersi o n 1. 4 , 2 1 A pril  2 0 2 1  
5 3  
I R B-5 7 0 5 6  
 
f  H e m at ol o g y is c o m pl et e bl o o d c o u nt wit h pl at el ets a n d diff er e nti al.  
g C D 3 4 +  c ell c o u nt i n a bs ol ut e n u m b ers a n d p er k g.  
h  P r oc e d ur e st art / e n d ti m es, bl o o d v ol u m e pr oc ess e d, v ol u m e of pr o d uct c oll e ct e d, pr oc e d ur e -r el at e d A Es 
i F oll o w -u p p h o n e c all b y st u d y t e a m at d a y 3 0 t o disc uss a n y o n g oi n g c o nc er ns, i ncl u di n g r es ol uti o n o f a n y i n v esti g ati o n al tr e at m e nt r el at e d t o xiciti es 
j C or el ati v e r es e arc h b o n e m arr o w bi o ps y as pir at e s a m pl e t o b e d o n e o nl y i n p ati e nts u n d er g oi n g a B M bi o ps y b y d a y 1 0 0 f or r o ut i n e cli nic al c ar e aft er 
tr a ns pl a nt at St a nf or d.  
k  C h e mistr y a n d H e m at ol o g y m a y b e d o n e wit hi n 2 4 h o urs of st arti n g a p h er esis, i ncl u di n g o n t h e d a y pri or  
l P ost -M o bili z ati o n a d v ers e e v e nt ass ess m e nt will b e c o n d uct e d i n a ti m el y m a n n er (r ef er e nc e S ecti o n 7. 2. 3 f or c o m pl et e i nf or m ati o n) 
All t e st s a n d p r o c e d u r e s a r e st a n d a r d of c a r e u nl e ss m a r k e d R,  R = r e s e a r c h  
 
 
 
 
 

Pr ot oc ol: M G T A -1 4 5 + pl eri x af or m o bili z ati o n  
 
Pr ot oc ol V ersi o n 1. 4 , 2 1 A pril  2 0 2 1  
5 4  
I R B-5 7 0 5 6  
 
T a bl e 1 0 B : C o r r el ati v e L a b St u d y S c h e d ul e   
 
C o rr el ati v e St u di es 
Ti mi n g  
S o urc e  
A m o u nt  
C oll e cti n g T u b e  
A p h e r e si s p r o d u ct  
Fl o w C yt o m etr y  
C y T O F  
C F U ass a y  
M R D ass a y  
A d diti o n al s a m pl e f or 
b a n ki n g  
A p h er esis d a y 1  
A p h er esis pr o d uct  
1 5 -2 0  m L  
S a m pl e o bt ai n e d fr o m 
a p h er esis c oll e cti o n b a g . 
 
At l e ast 5  ali q u ots ( 2-4 m L) . 
T h es e will b e us e d f or fl o w 
c yt o m etr y, C y T O F , M R D 
t esti n g a n d C F U a n al ysis.  At 
l e ast 1 ali q u ot t o b e b a n k e d 
(If l ar g e a m o u nt of s a m pl e 
r e m ai ni n g f or b a n ki n g, c a n 
di vi d e i n m or e t h a n 1 
cr y o vi al)  
 
C F U ass a y a n d M R D t esti n g o n 
fr es h s a m pl e. R est will b e o n 
cr y o pr es er v e d s a m pl es. 
P e ri p h e r al Bl o o d  
Fl o w C yt o m etr y  
C y T O F  
A d diti o n al sa m pl e f or 
b a n ki n g  
B a s eli n e s a m pl e: all  
U pf r o nt T r a n s pl a nt 
Gr o u p:  P ost -
tr a ns pl a nt d a y 2 8 
visit a n d d a y 1 0 0 
visit  
P eri p h er al Bl o o d  
1 0 -2 0  m L  
 
 
B a s eli n e s a m pl e:  1 0 m L 
(fr es h) i n a gr e e n t o p t u b e 
 
D a y 2 8 s a m pl e :  
1 0 m L (fr es h) i n a gr e e n t o p 
t u b e f or fl o w c yt o m etr y 
A d diti o n al 5 m L pr oc ess e d 
a n d cr y o pr es er v e d i n 1 
ali q u ot, w hic h will b e b a n k e d  

Pr ot oc ol: M G T A -1 4 5 + pl eri x af or m o bili z ati o n  
 
Pr ot oc ol V ersi o n 1. 4 , 2 1 A pril  2 0 2 1  
5 5  
I R B-5 7 0 5 6  
 
D a y 1 0 0 s a m pl e:   
1 0 m L (fr es h) i n a gr e e n t o p 
t u b e. 
 R est of t h e s a m pl e pr oc ess e d 
a n d st or e d i n at l e ast tw o  
cr y o pr es er v e d ali q u ots  of 5 
m L e a c h . 1 ali q u ot  will b e 
us e d f or C y T O F a n d a n  
a d diti o n al  ali q u ot will b e 
b a n k e d.  
B o n e M a r r o w Bi o p s y  
C y T O F  
U pf r o nt T r a n s pl a nt 
Gr o u p ( D o n e o nl y i n 
p ati e nts u n d er g oi n g 
B M bi o ps y b y d a y 
1 0 0 ti m e p oi nt f or 
cli nic al c ar e f oll o wi n g 
tr a ns pl a nt), Ti mi n g: 
B y d a y 1 0 0. 
Ti m e p oi nt. Us u all y 
d o n e d a y 6 0 -1 0 0 p ost 
tr a ns pl a nt 
B o n e m arr o w 
as pir at e  
5 m L  
S a m pl e will b e pr oc ess e d a n d 
cr y o pr es er v e d i n o n e ali q u ot 
of 5 m L.  
 

 
5 6  
 
1 0  M E A S U R E M E N T S   
 
P ri m a r y O ut c o m e M e a s u r e D efi niti o n:  C D 3 4 yi el d  of c oll e ct e d H S Cs e x pr ess e d as C D 3 4 + c ells / k g 
of p ati e nt b o d y w ei g ht.  
•  Ti m e F r a m e : D a y o n e a n d t w o of st e m c ell m o biliz ati o n a n d a p h er esis  
•  S af et y Is s u e : Is t his o utc o m e m e as ur e ass essi n g a s af et y iss u e?  N o 
 
1 0. 1  
H S C yi el d i n a p h e r esi s p r o d u ct  (P ri m a r y a n d S e c o n d a r y O ut c o m e m e a s u r es ) 
T h e pri m ar y o utc o m e is c oll e cti o n of 2 . 0 x 1 06 C D 3 4 c ells / k g i n o n e or t w o d a ys of a p h er esis aft er 
m o bili z ati o n wit h M G T A -1 4 5 a n d pl eri x af or. S e c o n d ar y o utc o m es als o i ncl u d e d a y o n e yi el d of 2. 0 x 
1 0 6 C D 3 4 c ells / k g a n d 4. 0 x 1 0 6 C D 3 4 c ells / k g a n d t ot al yi el d of 4. 0 x 1 0 6 C D 3 4 c ells / k g a n d 6. 0 x 1 0 6 
C D 3 4 c ells / k g i n u p t o  t w o d a ys of a p h er esis 
1 0. 1. 1 R el e v a nt S u b s et : All p ati e nts e nr oll e d i n t h e st u d y w h o u n d er g o st e m c ell m o biliz ati o n, N = 2 5  
1 0. 1. 2 M e a s u r e m e nt d efi niti o n  a n d ti m e p oi nt s: C D 3 4 yi el d of a p h er esis pr o d uct o n d a y 1 a n d 2 
of a p h er esis will esti m at e d t hr o u g h fl o w c yt o m etric e v al u ati o n of t h e a p h er esis pr o d u ct. T h e 
a p h er esis pr o d uct v ol u m e a n d t h e a bs ol ut e n u m b er of C D 3 4 + c ells p er u nit v ol u m e ar e t o b e 
m e as ur e d t o c alc ul at e t h e t ot al yi el d of C D 3 4 + c ells. 
T his 
will b e d o n e f oll o wi n g St a nf or d 
B M T / A p h er esis a n d C T F st a n d ar d o p er ati n g pr oc e d ur es.  
T ot al yi el d re p orti n g i ncl u d es t h e  c o m bi n e d yi el d fr o m d a y 1 a n d 2 (if p ati e nt u n d er g o es a s e c o n d 
d a y of c oll e cti o n).  
1 0. 2  
Ot h e r S e c o n d a r y  O ut c o m e  M e a s u r es  
 
1 0. 2. 1  Inf u si o n r el at e d t o xi citi e s wit h M G T A -1 4 5 ; Ad v e r s e e v e nt s wit h m o bili z ati o n of H S C s 
wit h M G T A -1 4 5 a n d pl e ri x af o r.  
 
R el e v a nt S u b s et:  All p ati e nts e nr oll e d i n t h e st u d y w h o u n d er g o st e m c ell m o biliz ati o n, N = 2 5 .  
M e a s u r e m e nt d efi niti o n a n d ti m e p oi nt s: T his will b e ass ess e d b y N CI C T C A E v ersi o n 5. 0 as 
d escri b e d u n d er s e cti o n 7 of t h e pr ot oc ol. F or a gi v e n A E , t h e m a xi m al gr a d e A E o n t h at d a y will b e 
r e p ort e d.  
 
A E ass ess m e nt will occ ur at  t h e f oll o wi n g ti m e p oi nts, ( e xc e pt i nf usi o n r el at e d A Es, w hic h will o nl y 
ass ess e d o n e a c h d a y of m o biliz ati o n ): 
 
•  
B as eli n e ( wit hi n 3 d a ys of m o biliz ati o n)  
•  
O n e a c h d a y of m o biliz ati o n  

Pr ot oc ol: M G T A -1 4 5 + pl eri x af or m o bili z ati o n  
 
Pr ot oc ol V ersi o n 1. 4 , 2 1 A pril  2 0 2 1  
5 7  
I R B-5 7 0 5 6  
 
•  
Wit hi n 7 d a ys aft er m o biliz ati o n  
 
1 0. 2. 2  N e ut r o p hil  a n d pl at el et e n g r aft m e nt  
 
R el e v a nt S u b s et:  O nl y p ati e nts pr oc e e di n g wit h u pfr o nt tr a ns pl a nt will b e ass ess e d.  
 
M e a s u r e m e nt d efi niti o n a n d ti m e p oi nt s:  
 
Ti m e t o pri m ar y e n gr aft m e nt is d efi n e d as ti m e i n d a ys fr o m t h e d a y of st e m c ell i nf usi o n t o t h e first 
d a y of e n gr aft m e nt.  
 
O n g oi n g s ucc essf ul e n gr aft m e nt at 3 0 - a n d 1 0 0 -d a ys p ost -tr a ns pl a nt aft er s ucc essf ul pri m ar y 
e n gr aft m e nt will als o b e ass ess e d b y C B C  d o n e as p art of r o uti n e cli nic al c ar e of p a ti e nts u n d er g oi n g 
tr a ns pl a nt. 
 
E n gr aft m e nt d efi niti o ns ar e as f oll o ws:  
 
•  
N e utr o p hil e n gr aft m e nt is d efi n e d as t h e first d a y of A N C  ≥0. 5  x  1 0 9 / L f or 3  d a ys f oll o wi n g 
st e m c ell i nf usi o n . T his is ass ess e d b y a bs ol ut e n e utr o p hil c o u nts o bt ai n e d as p art of C B C wit h 
diff er e nti al. It is st a n d ar d 
pr a ctic e t o f oll o w 
p ati e nts r e g ul arl y i n t h e i n p ati e nt 
u nit / o ut p ati e nt tr a ns pl a nt u nit u ntil t h e y a c hi e v e e n gr aft m e nt.   
•  
P l at el et e n gr aft m e nt is d efi n e d as t h e first d a y of pl at el et c o u nt m or e t h a n or e q u al t o 
2 0  x  1 0 9 / L wit h o ut tr a nsf usi o n i n t h e l ast 7 d a ys a n d wit h pl at el et c o u nt ≥2 0  x  1 0 9 / L o n 
2  s e p ar at e, s u bs e q u e nt d a ys . T his is ass ess e d b y pl at el ets c o u nts o bt ai n e d as p art of C B C wit h 
diff er e nti a l. It is st a n d ar d pr a ctic e t o f oll o w p ati e nts r e g ul arl y i n t h e tr a ns pl a nt i n p ati e nt 
u nit / o ut p ati e nt tr a ns pl a nt u ntil t h e y a c hi e v e e n gr aft m e nt.  
•  
W e will als o  d et er mi n e ti m e t o first d a y of pl at el et c o u nt m or e t h a n or e q u al t o 5 0  x  1 0 9 / L, 
wit h o ut tr a nsf usi o n wit hi n 4 8 h o urs.   
 
1 0. 2. 3  T r a n s pl a nt r el at e d m o rt alit y  
 
R el e v a nt S u b s et:  O nl y p ati e nts pr oc e e di n g wit h u pfr o nt tr a ns pl a nt will b e ass ess e d.  
 
M e a s u r e m e nt d efi niti o n a n d ti m e p oi nt s: Tr a ns pl a nt r el at e d m ort alit y is d escri b e d as d e at h fr o m 
a n y c a us e wit hi n t h e first 1 0 0 d a ys of H S C i nf usi o n. 
 

Pr ot oc ol: M G T A -1 4 5 + pl eri x af or m o bili z ati o n  
 
Pr ot oc ol V ersi o n 1. 4 , 2 1 A pril  2 0 2 1  
5 8  
I R B-5 7 0 5 6  
 
1 0. 2. 4  N o n -r el a p s e r el at e d m o rt alit y  
 
R el e v a nt S u b s et:  O nl y p ati e nts pr oc e e di n g wit h u pfr o nt tr a ns pl a nt will b e ass ess e d.  
 
M e a s u r e m e nt d efi niti o n a n d ti m e p oi nt s: Tr a ns pl a nt r el at e d n o n -r el a ps e m ort alit y is d escri b e d 
as d e at h fr o m a n y c a us e e xc e pt dis e as e r el a ps e / pr o gr essi o n wit hi n t h e first 1 0 0 d a ys of H S C i nf usi o n.  
 
1 0. 2. 5  P r o g r es si o n F r e e S u r vi v al  ( P F S):  
 
R el e v a nt S u b s et:  O nl y p ati e nts pr oc e e di n g wit h u pfr o nt tr a ns pl a nt will b e ass ess e d.  
 
M e a s u r e m e nt d efi niti o n a n d ti m e p oi nt s: D ur ati o n fr o m st art of t h e A S C T t o dis e as e pr o gr essi o n 
or d e at h (r e g ar dl ess of c a us e of d e at h), w hic h e v er c o m es first. Pr o gr essi o n  will b e ass ess e d  usi n g d a y 
1 0 0 p os t-tr a ns pl a nt r es p o ns e a n d P F S r at es will b e r e p ort e d at d a y 1 0 0 i n p ati e nts u n d er g oi n g  
u pfr o nt tr a ns pl a nt.  Aft er d a y 1 0 0, t h es e p ati e nts will b e f oll o w e d f or P F S off st u d y u n d er t h e B M T 
r es e arc h d at a b as e u n d er a s e p ar at e I R B a p pr o v e d pr ot oc ol.  
 
1 0. 2. 6  O v e r all S u r vi v al  ( O S):  
 
R el e v a nt S u b s et:  O nl y p ati e nts pr oc e e di n g wit h u pfr o nt tr a ns pl a nt will b e ass ess e d.  
 
M e a s u r e m e nt d efi niti o n a n d ti m e p oi nt s: D ur ati o n  fr o m st art of t h e A S C T t o d e at h (r e g ar dl ess of 
c a us e of d e at h). T his will b e ass ess e d fr o m st art of tr a ns pl a nt a n d O S r at es will b e r e p ort e d at d a y 
1 0 0 f oll o wi n g tr a ns pl a nt i n p ati e nts u n d er g oi n g u pfr o nt tr a ns pl a nt. Aft er d a y 1 0 0, t h es e p ati e nts will 
b e f oll o w e d f or P F S off st u d y u n d er t h e B M T r es e arc h d at a b as e u n d er a s e p ar at e I R B a p pr o v e d 
pr ot oc ol.  
 
1 0. 2. 7  R es p o n s e A s s es s m e nt  t o D et er mi n e P r o g r es si o n:  
 
R el e v a nt 
S u b s et:  R es p o ns e 
b ef or e 
m o biliz ati o n 
will 
b e 
ass ess e d 
i n 
all 
p ati e nts. 
R es p o ns e / pr o gr essi o n  f oll o wi n g tr a ns pl a nt 
will b e ass ess e d i n p ati e nts u n d er g oi n g u pfr o nt 
tr a ns pl a nt. 
 
M e a s u r e m e nt d efi niti o n a n d ti m e p oi nt s  M y el o m a r es p o ns e b ef or e st e m c ell m o biliz ati o n  a n d at 
d a y 1 0 0 will b e ass ess e d p er t h e I nt er n ati o n al M y el o m a W or ki n g Gr o u p  U nif or m R es p o ns e Crit eri a. 
2 7  
 

Pr ot oc ol: M G T A -1 4 5 + pl eri x af or m o bili z ati o n  
 
Pr ot oc ol V ersi o n 1. 4 , 2 1 A pril  2 0 2 1  
5 9  
I R B-5 7 0 5 6  
 
1 1  R E G U L A T O R Y C O N SI D E R A TI O N S  
1 1. 1  
I n stit uti o n al R e vi e w of P r ot o c ol 
T h e pr ot oc ol, t h e pr o p os e d i nf or m e d c o ns e nt a n d all f or ms of p artici p a nt i nf or m ati o n r el at e d t o t h e 
st u d y ( e. g. a d v ertis e m e nts us e d t o r e cr uit p artici p a nts) will b e r e vi e w e d a n d a p pr o v e d b y t h e 
St a nf or d I R B a n d St a nf or d C a nc er I nstit ut e Sci e ntific R e vi e w C o m mitt e e ( S R C).  n y c h a n g es m a d e t o 
t h e pr ot oc ol will b e s u b mitt e d as a 
m o dific ati o n a n d will b e a p pr o v e d b y t h e I R B pri or t o 
i m pl e m e nt ati o n. T h e Pr ot oc ol Dir e ct or will diss e mi n at e t h e pr ot oc ol a m e n d m e nt i nf or m ati o n t o all 
p artici p ati n g i n v esti g at ors.  
 
1 1. 2  D at a a n d S af et y M o nit o ri n g Pl a n  
1 1. 2. 1  P ri n ci p al I n v esti g at o r a n d St u d y  R es e a r c h T e a m  
T h e cli nic al r es e arc h t e a m a n d i n v esti g at or s p o ns or will m e et o n a r e g ul ar b asis d uri n g p ati e nt 
scr e e ni n g a n d w hil e p ati e nts ar e a cti v el y r e c ei vi n g t h e i n v esti g ati o n al a g e nt o n t his tri al t o disc uss 
eli gi bilit y q u esti o ns, tri al a ccr u al a n d t o xiciti es.  
 
All d at a will b e c oll e ct e d i n a ti m el y m a n n er a n d r e vi e w e d b y t h e i n v esti g at or -s p o ns or or a l e a d 
ass oci at e i n v esti g at or. A d v ers e e v e nts will b e r e p ort e d as r e q uir e d a b o v e. A n y s af et y c o nc er ns, n e w 
i nf or m ati o n t h at mi g ht aff e ct eit h er t h e et hical a n d or sci e ntific c o n d uct of t h e tri al, or pr ot oc ol 
d e vi ati o ns will b e i m m e di at el y r e p ort e d t o t h e D S M C , s p o ns or-i n v esti g at or a n d F D A as n e e d e d.   
T h e pri nci p al i n v esti g at or will r e vi e w a d v ers e e v e nt a n d o utc o m e  d at a o n e a c h p ati e nt  t o e ns ur e 
s af et y a n d d at a a cc ur a c y. T h e pri nci p al i n v esti g at or will p ers o n all y c o n d uct or s u p er vis e t h e 
i n v esti g ati o n a n d pr o vi d e a p pr o pri at e d el e g ati o n of r es p o nsi biliti es t o ot h er 
m e m b ers of t h e 
r es e arc h st aff.   
 
1 1. 2. 2  St a nf o r d C a n c e r I n stit ut e D at a a n d S af et y M o nit o ri n g C o m mitt e e ( D S M C)  
T h e St a nf or d C a nc er I nstit ut e D at a a n d S af et y M o nit ori n g C o m mitt e e ( D S M C) will b e t h e m o nit ori n g 
e ntit y f or t his st u d y. T h e D S M C will a u dit st u d y -r el at e d a cti viti es t o d et er mi n e w h et h er t h e st u d y h as 
b e e n c o n d uct e d i n a cc or d a nc e 
wit h t h e pr ot oc ol , l oc al st a n d ar d o p er ati n g pr oc e d ur es, F D A 
r e g ul ati o ns, a n d G o o d Cli nic al Pr a ctic e ( G C P).  T his m a y i ncl u d e r e vi e w of t h e f oll o wi n g t y p es of 
d oc u m e nts p artici p ati n g i n t h e st u d y: r e g ul at or y bi n d ers, c as e r e p ort f or ms, eli gi bilit y c h e c klists, a n d 
s o urc e d o cu m e nts. I n a d diti o n, t h e D S M C will r e g ul arl y r e vi e w s eri o us a d v ers e e v e nts a n d pr ot oc ol 
d e vi ati o ns ass oci at e d wit h t h e r es e arc h t o e ns ur e t h e pr ot e cti o n of h u m a n s u bj e cts. R es ults of t h e 
D S M C a u dit will b e c o m m u nic at e d t o t h e I R B a n d t h e a p pr o pri at e r e g ul a t or y a ut h oriti es at t h e ti m e 
of c o nti n ui n g r e vi e w, or i n a n e x p e dit e d f as hi o n, as n e e d e d.  
 
1 1. 2. 3  D at a M a n a g e m e nt Pl a n  
T h e Pr ot oc ol Dir e ct or, or his / h er d esi g n e e, will pr e p ar e a n d m ai nt ai n a d e q u at e a n d a cc ur at e 
p artici p a nt c as e hist ori es wit h o bs er v ati o ns a n d d at a p erti n e nt t o t h e st u d y , i n a cc or d a nc e wit h t h e 
est a blis h e d pr a ctic es of t h e St a nf or d Di visi o n of Bl o o d a n d M arr o w Tr a ns pl a nt ati o n . St u d y s p e cific 
el e ctr o nic C as e R e p ort F or ms ( C R Fs) will d oc u m e nt tr e at m e nt o utc o m es f or d at a a n al ysis. D at a will 

Pr ot oc ol: M G T A -1 4 5 + pl eri x af or m o bili z ati o n  
 
Pr ot oc ol V ersi o n 1. 4 , 2 1 A pril  2 0 2 1  
6 0  
I R B-5 7 0 5 6  
 
b e tr a nscri b e d i nt o e C R Fs usi n g M e d ri o s oft w ar e. M e d ri o s oft w ar e is c o m pli a nt wit h 2 1 C o d e of 
F e d er al R e g ul ati o ns P art 1 1 F D A r e q uir e m e nts . 
 
D at a will b e e nt er e d f or t his st u d y utilizi n g o n e or a c o m bi n ati o n of t h e f oll o wi n g m et h o ds:  
 
o  D at a m a y b e  c a pt ur e d el e ctr o nic all y, wit h o ut us e of p a p er .  
o  D at a m a y b e tr a nscri b e d fr o m t h e El e ctr o nic M e dic al R e c or d s o urc e d oc u m e nts i nt o t h e E D C 
s yst e m wit h o ut t h e us e of p a p er 
o  D at a m a y b e c a pt ur e d o n p a p er (s o urc e d oc u m e nts) a n d tr a nscri b e d i nt o t h e E D C s yst e m. I n 
t his c as e p a p er d oc u m e nt ati o n will b e r et ai n e d a n d will b e a v ail a bl e f or r e vi e w.  
D S M C of t h e St a nf or d C a nc er C e nt er will o v ers e e t his st u d y a n d will r e c ei v e r e g ul ar r e p orti n g of S A Es 
a n d m o nit ori n g. T his C o m mitt e e i n v ol v es k n o wl e d g e a bl e p h ysici a ns, sci e ntists a n d st atistici a ns w h o 
o v ers e e all p h as e I /II tri als c o n d uct e d u n d er t h e a us pic es of t h e St a nf or d C a nc er C e nt er. I n di vi d u als 
w h o s er v e o n t his C o m mitt e e w h o ar e ass oci at e d wit h t his tri al will r e c us e t h e ms el v es fr o m 
disc ussi o ns of st u d y r es ults, a n d  d e cisi o ns a b o ut r e visi o n or cl os ur e. A u dits will b e c o n d u ct e d as 
r e q u est e d. D at a will als o b e s u b mitt e d t o t h e F D A as r e q uir e d b y o ur I N D a n d f oll o w t h e a p pr o pri at e 
r e p orti n g r ul es a n d r e g ul ati o ns i ncl u di n g a n a n n u al r e p ort . D at a will b e s h ar e d wit h M a g e n t a 
T h er a p e utics a cc or di n g t o  a d at a s h ari n g a n d us e a gr e e m e nt.  
 
1 2  C O L L A B O R A TI V E A G R E E M E N T S  
A c oll a b or ati v e r es e arc h a gr e e m e nt e xists f or r e c ei pt of M G T A -1 4 5 a n d fi n a nci al s u p p ort fr o m 
M a g e nt a T h er a p e utics. T his c oll a b or ati v e a gr e e m e nt is discl os e d i n t h e i nf or m e d c o ns e nt 
d oc u m e nt. I n t his a gr e e m e nt, M a g e nt a T h er a p e utics h as a cc ess t o st u d y d at a a n d st u d y i nf or m ati o n 
a n d  m a y r e c ei v e st u d y s a m pl es f or a n al ysis.  
 
1 3  S T A TI S TI C A L C O N SI D E R A TI O N S  
1 3. 1  St ati sti c al D esi g n  
T his is a si n gl e c e nt er, si n gl e ar m p h as e II pr o of of c o n c e pt  st u d y d esi g n e d t o ass ess t h e s ucc ess r at e 
of st e m c ell m o biliz ati o n wit h a n o v el r e gi m e n of a n i n v esti g ati o n al dr u g ( M G T A -1 4 5) wit h pl eri x af or 
i n p ati e nts wit h m ulti pl e m y el o m a. T h e s a m pl e si z e of t his p h as e II pr o of of c o nc e pt st u d y is 2 5 
p ati e nts as d escri b e d b el o w. A d diti o n al p ati e nts m a y b e scr e e n e d t o a cc o u nt f or p ati e nts w h o scr e e n 
f ail or wit h dr a w pri or t o st art of st u d y i nt er v e nti o n i. e a d mi nistr ati o n of  M G T A -1 4 5.  
1 3. 2  S af et y R u n -I n / F utilit y Rul e  
E a rl y S af et y R u n -i n: T h e first si x p ati e nts a ccr u e d t o t his st u d y will b e p ati e nts w h o ar e pl a n n e d f or 
u pfr o nt tr a ns pl a nt f oll o wi n g c oll e cti o n. A d diti o n al p ati e nts will n ot b e gi n m o biliz ati o n u ntil t h es e 
t hr e e p ati e nts h a v e c o m pl et e d st e m c ell m o biliz ati o n a n d ar e at l e ast 2 1 d a ys f oll o wi n g i nf usi o n of 
st e m c ells aft er m y el o a bl ati v e m el p h al a n c o n diti o ni n g.  S af et y r e vi e w will b e d o n e t o e v al u at e st u d y 
dr u g -r el at e d a d v ers e e v e nts, i n p artic ul ar f or a n y u n e x p e ct e d gr a d e 3 or hi g h er a d v ers e e v e nts dr u g 
d uri n g t his p eri o d. E n gr aft m e nt d at a will als o b e r e vi e w e d. If a n y u n e x p e ct e d gr a d e 3 or hi g h er 

Pr ot oc ol: M G T A -1 4 5 + pl eri x af or m o bili z ati o n  
 
Pr ot oc ol V ersi o n 1. 4 , 2 1 A pril  2 0 2 1  
6 1  
I R B-5 7 0 5 6  
 
a d v ers e e v e nts pr o b a bl y or d efi nit el y r el at e d t o t h e st u d y dr u g ar e n ot e d i n t h e first t hr e e p ati e nts, 
t h e PI will r e vi e w t h e st u d y wit h t h e F D A t o d et er mi n e w h et h er st u d y c a n c o nti n u e.    
1 3. 3  D es c ri pti v e St ati sti c s a n d E x pl o r at o r y D at a A n al ysi s  
D escri pti v e st atistics will b e  us e d t o d escri b e st u d y p ati e nt p o p ul ati o n a n d e x pl or at or y d at a 
a n al ysis. C at e g oric al v ari a bl es will b e s u m m ariz e d i n fr e q u e nci es . C o nti n u o us v ari a bl es will b e 
s u m m ariz e d i n m e a n a n d m e di a n, or m e di a n a n d i nt er q u artil e r a n g e w h e n t h e y ar e a p pr o pri at e. 
Ni n e t y-fi v e c o nfi d e nc e i nt er v als will b e pr es e nt e d.  
 
1 3. 4  Pri m ar y A n al ysis   
T h e p ri m a r y e n d p oi nt of t his st u d y is t o ass ess t h e r at e of s ucc essf ul c oll e cti o n of ≥2 . 0 x  1 0 6  C D 3 4 +  
c ells / k g i n u p t o t w o d a ys of a p h er esis aft er M G T A -1 4 5 a n d  pl eri x af or d osi n g i n p ati e nts wit h M M.  
( as d escri b e d i n s e cti o n 1. 1  a n d  1 0. 1 )   
 
a.  A n al ysi s P o p ul ati o n : P ati e nts pr oc e e di n g wit h st e m c ell m o biliz ati o n aft er r e c ei vi n g t h e 
n o v el r e gi m e n of M G T A -1 4 5 a n d pl eri x af or. T his i ncl u d e s 1 5 p ati e nts  i n s e g m e nt o n e. If t h e 
pri m ar y e n d p oi nt is m et i n t h e first s e g m e nt of t h e st u d y, t his e n d p oi nt will als o b e e v al u at e d 
i n t h e t ot al s a m pl e si z e of 2 5 p ati e nts (s e g m e nt o n e a n d t w o). 
 
b.  A n al ysi s Pl a n : A p h er es is pr o d uct c ell c o u nts ar e r e p ort e d as a st a n d ar d of c ar e i n all p ati e nts 
u n d er g oi n g st e m c ell c oll e cti o n usi n g fl o w c yt o m etr y p er i nstit uti o n al g ui d eli n es. T h e 
s ucc essf ul a c hi e v e m e nt of t h e pri m ar y o bj e cti v e of effic a c y of t his r e gi m e n will b e ass ess e d 
b y  c alc ul ati n g t h e pr o p orti o n of p ati e nts m e eti n g t his e n d p oi nt aft er u p t o t w o d a ys of 
i n v esti g ati o n al dr u g r e gi m e n f oll o w e d b y a p h er esis.  
 
H S Cs c o u nts will b e a d d e d t o g et t h e fi n al H S C c o u nt p er k g of p ati e nt b o d y w ei g ht fr o m H S Cs  
c oll e ct e d i n diff er e nt b a gs a n d o n diff er e nt d a ys as is st a n d ar d  o p er ati n g pr oc e d ur e . As 
d escri b e d u n d er s a m pl e si z e, w e h y p ot h esi z e t h at at l e ast 9 5 % of p ati e nts will m e et t h e 
pri m ar y e n d p oi nt.  T h us, w e e x p e ct t h at at l e ast 1 3  o ut of 1 5 s u bj e cts  i n t h e first s e g m e nt of 
t h e st u d y will a c hi e v e t his H S C c oll e cti o n t ar g et.  
 
1 3. 5  S e c o n d a r y A n al ysi s  
1 3. 5. 1  S e c o n d a r y  E n d p oi nt  # 1:  
T o ass ess t h e r at e of s ucc essf ul c oll e cti o n of  ( a) ≥4. 0  x  1 0 6  C D 3 4 +  c ells / k g i n u p t o t w o a p h er esis 
s essi o ns aft er M G T A-1 4 5 a n d pl eri x a f or d osi n g i n p ati e nts wit h M M. ( K e y S e c o n d a r y E n d p oi nt) ( b) 
≥6. 0  x  1 0 6  C D 3 4 +  c ells / k g wit h u p t o t w o a p h er esis s essi o ns aft er M G T A -1 4 5 a n d pl eri x af or d osi n g i n 
p ati e nts wit h M M.  ( c) ≥2. 0  x  1 0 6  C D 3 4 +  a n d ≥4. 0  x  1 0 6  C D 3 4 +  c ells / k g wit h o n e a p h er esis s essi o n 
aft er M G T A -1 4 5 a n d pl eri x af or d osi n g . 
 
A n al y si s P o p ul ati o n: All p ati e nts pr oc e e di n g wit h st e m c ell m o biliz ati o n aft er r e c ei vi n g t h e n o v el 
r e gi m e n of M G T A-1 4 5 a n d pl eri x af or. T his i ncl u d es 2 5 p ati e nts i ncl u di n g s e g m e nt o n e a n d t w o. 

Pr ot oc ol: M G T A -1 4 5 + pl eri x af or m o bili z ati o n  
 
Pr ot oc ol V ersi o n 1. 4 , 2 1 A pril  2 0 2 1  
6 2  
I R B-5 7 0 5 6  
 
A n al y si s Pl a n: A p h er esis pr o d uct c ell c o u nts ar e r e p ort e d as a st a n d ar d of c ar e i n al l p ati e nts 
u n d er g oi n g st e m c ell c oll e cti o n usi n g fl o w c yt o m etr y p er i nstit uti o n al g ui d eli n es. T h e s ucc essf ul 
a c hi e v e m e nt of t h e s e c o n d ar y e n d p oi nt 1 a a n d 1 b will b e ass ess e d b y c alc ul ati n g t h e pr o p orti o n of 
p ati e nts m e eti n g t h e t ar g ets list e d a b o v e aft er r e c ei vi n g i n v esti g ati o n al dr u g r e gi m e n f oll o w e d b y 
a p h er esis.  H S Cs c o u nts will b e a d d e d t o g et t h e fi n al H S C c o u nt p er k g of p ati e nt b o d y w ei g ht fr o m 
H S Cs c oll e ct e d i n diff er e nt b a gs a n d o n diff er e nt d a ys as is st a n d ar d o p er ati n g pr oc e d ur e. Pr o p orti o n 
of p ati e nts m e eti n g t h e e n d p oi nts will b e r e p ort e d.  
 
1 3. 5. 2  S e c o n d a r y E n d p oi nt # 2:  
T o ass ess i nf usi o n r el at e d t o xiciti es wit h M G T A -1 4 5 a n d a d v ers e e v e nts wit h m o biliz ati o n of H S Cs 
wit h M G T A -1 4 5 a n d pl eri x af or.  
 
A n al y si s P o p ul ati o n: All p ati e nts pr oc e e di n g wit h st e m c ell m o biliz ati o n aft er r e c ei vi n g t h e n o v el 
r e gi m e n of M G T A-1 4 5 a n d pl eri x af or. T his i ncl u d es 2 5 p ati e nts i ncl u di n g s e g m e nt o n e a n d t w o. 
 
A n al y si s Pl a n:  A d v ers e e v e nts will b e r e p ort e d usi n g N CI C T C A E v ersi o n 5. 0 as d escri b e d i n  s e cti o n 
7 . Pr o p orti o n of p ati e nts e x p eri e nci n g e a c h si d e eff e ct a n d gr a d e of si d e eff e ct will b e a n al y z e d a n d 
r e p ort e d.  
 
1 3. 5. 3  S e c o n d a r y E n d p oi nt # 3:  
T o ass ess ti m e t o n e utr o p hil ( d efi n e d as t h e first d a y of A N C  ≥0. 5  x  1 0 9 / L f or 3  d a ys f oll o wi n g st e m 
c ell i nf usi o n) a n d pl at el et e n gr aft m e nt ( d efi n e d as first d a y of pl at el et c o u nt m or e t h a n or e q u al t o 
2 0  x  1 0 9 / L wit h o ut tr a nsf usi o n i n t h e l ast 7 d a ys a n d wit h pl at el et c o u nt ≥2 0  x  1 0 9 / L o n 2  s e p ar at e, 
s u bs e q u e nt d a ys a n d t o als o d et er mi n e ti m e t o first d a y of pl at el et c o u nt m or e t h a n or e q u al t o 
5 0  x  1 0 9 / L, wit h o ut tr a nsf usi o n wit hi n 4 8 h o urs i n p ati e nts u n d er g oi n g u pfr o nt A S C T. 
 
A n al y si s P o p ul ati o n: All p ati e nts pr oc e e di n g wit h u pfr o nt tr a ns pl a nt ( d efi n e d as wit hi n 6 0 d a ys of 
H S C m o bili z ati o n a n d c oll e cti o n) will b e e v al u at e d f or t his e n d p oi nt.   
 
A n al y si s Pl a n: N u m b er a n d p erc e nt a g e of  p ati e nts a c hi e vi n g e n gr aft m e nt b y a b o v e p ar a m et er s.  
Ti m e t o e n gr aft m e nt will b e e v al u at e d f or e a c h p ati e nt a n d s u m m ariz e d as m e di a n, i nt er -q u artil e 
r a n g e, r a n g e a n d pr o p orti o n of p ati e nts a c hi e vi n g e n gr aft m e nt b y a gi v e n ti m e p oi nt.  
 
1 3. 5. 4  S e c o n d a r y E n d p oi nt # 4 :  
T o ass ess r at es of o n g oi n g s ucc essf ul e n gr aft m e nt at 3 0 - a n d 1 0 0 -d a ys p ost -tr a ns pl a nt aft er 
s ucc essf ul pri m ar y e n gr aft m e nt i n p ati e nts u n d er g oi n g u pfr o nt A S C T.  
 
A n al y si s P o p ul ati o n: All p ati e nts pr oc e e di n g wit h u pfr o nt tr a ns pl a nt ( d efi n e d as wit hi n 6 0 d a ys of 
H S C m o bili z ati o n a n d c oll e cti o n) will b e e v al u at e d f or t his e n d p oi nt.   
 
A n al y si s Pl a n: Pr o p orti o n of p ati e nts a c hi e vi n g pri m ar y e n gr aft m e nt b y a b o v e p ar a m et e rs will als o 
b e ass ess e d f or o n g oi n g s e c o n d ar y e n gr aft m e nt b y C B C wit h diff er e nti al o n d a y 3 0 a n d 1 0 0 p ost -
tr a ns pl a nt 
 

Pr ot oc ol: M G T A -1 4 5 + pl eri x af or m o bili z ati o n  
 
Pr ot oc ol V ersi o n 1. 4 , 2 1 A pril  2 0 2 1  
6 3  
I R B-5 7 0 5 6  
 
1 3. 5. 5  S e c o n d a r y E n d p oi nt # 5 :  
T o ass ess r at e of tr a ns pl a nt r el at e d m ort alit y, n o n -r el a ps e m ort alit y, pr o gr essi o n fr e e s ur vi v al a n d 
o v er all s ur v i v al at 1 0 0 d a ys i n p ati e nts wit h u n d er g oi n g u pfr o nt A S C T. 
 
A n al y si s P o p ul ati o n: All p ati e nts pr oc e e di n g wit h u pfr o nt tr a ns pl a nt ( d efi n e d as wit hi n 6 0 d a ys of 
H S C m o bili z ati o n a n d c oll e cti o n) will b e e v al u at e d f or t his e n d p oi nt.   
 
A n al y si s Pl a n: N u m b er a n d  p erc e nt a g e of p ati e nts e x p eri e nci n g d e at h r el at e d t o a n y c a us e 
( Tr a ns pl a nt r el at e d m ort alit y), d e at h r el at e d t o a n y c a us e, e xc e pt dis e as e pr o gr essi o n ( N o n -r el a ps e 
m ort alit y) a n d p ati e nts r e m ai ni n g pr o gr essi o n fr e e a n d ali v e ( D a y 1 0 0 pr o gr essi o n fr e e s ur vi v al), 
a n d p ati e nts ali v e at d a y 1 0 0 ( d a y 1 0 0 O S) will b e r e p ort e d.  K a pl a n -M ei er s ur vi v al c ur v es will b e 
g e n er at e d. 
W h e n e v er p ossi bl e, m e di a n ti m es t o t h e a b o v e e v e nts will b e esti m at e d wit h 9 5 % 
c o nfi d e nc e i nt er v als. 
1 3. 6  S a m pl e Si z e  
1 3. 6. 1  A c c r u al esti m at es  
 
T h e s a m pl e si z e f or t his si n gl e c e nt er, si n gl e ar m st u d y is 2 5 p ati e nts w h o u n d er g o m o biliz ati o n as 
d escri b e d b el o w. A d diti o n al p ati e nts m a y b e scr e e n e d t o d et er mi n e eli gi bilit y f or t h e tri al.  
W e e x p e ct t o c o m pl et e st u d y a ccr u al i n 9 -1 2 m o nt hs as 1 0 0 -1 2 0 p ati e nts wit h m ulti pl e m y el o m a 
u n d er g o A S C T a n n u all y i n o ur di visi o n.  
If a ccr u al f alls s h ort of e x p e ct ati o ns, t h e n w e will c o nti n u e t o e nr oll p ati e nts f or a d diti o n al 6-9 m o nt hs 
t o c o m pl et e a ccr u al. 
 
1 3. 6. 2  S a m pl e si z e j u stifi c ati o n  
F or t his si n gl e -ar m tri al wit h a bi n o mi al pri m ar y e n d p oi nt, t h e m a xi m u m s a m pl e si z e is 2 5 e v al u a bl e 
p ati e nts.  B as e d o n hist oric al d at a wit h H S C m o biliz ati o n wit h G -C S F 5, 8 -1 0 , w e h y p ot h esi z e t h at at l e ast 
9 5 % of s u bj e cts w h o u n d er g o m o biliz ati o n wit h t his n o v el r e gi m e n of M G T A -1 4 5 a n d pl eri x af or will 
m e et t h e pri m ar y e n d -p oi nt of c oll e cti n g ≥2 . 0 x  1 0 6  C D 3 4 +  c ells / k g i n t w o d a ys of a p h er esis . W e 
m o d el t h e pri m ar y e n d p oi nt as a bi n o mi al r a n d o m v ari a bl e. T h e st atistic al d esi g n is b as e d o n t h e 
f oll o wi n g o n e-si d e d h y p ot h esis t est. T h e n ull h y p ot h esis is t h at t h e pri m ar y e n d p oi nt  is l ess t h a n or 
e q u al t o 7 0 %. T h e alt er n ati v e h y p ot h esis is t h at t h e pri m ar y e n d p oi nt is e q u al t o or gr e at er t h a n 9 5 %.  
T h e tri al will b e c o n d uct e d i n t w o s e q u e nti al s e g m e nts: t h e first f or e v al u ati o n of s af et y a n d 
f e asi bilit y, a n d t h e n if s ucc essf ul, t h e n u m b er will b e e x p a n d e d t o f urt h er e v al u at e s af et y a n d 
f e asi bilit y. I n t h e first s e g m e nt, 1 5 p ati e nts will b e e v al u at e d f or t h e pri m ar y e n d p oi nt. I n t h e s e c o n d 
s e g m e nt, w hic h will b e c o nti n g e nt u p o n a c hi e vi n g s ucc ess i n t h e first s e g m e nt, 1 0 p ati e nts will b e 
e nr oll e d t o g ai n a d diti o n al s af et y a n d f e asi bilit y e x p eri e nc e wit h t his r e gi m e n a n d t o e v al u at e t h e k e y 
s e c o n d ar y e n d p oi nt. Acc o u nti n g f or b ot h s e g m e nts, t h e t ot al s a m pl e si z e is 2 5 p ati e nts.  
W e m o d el t h e pri m ar y e n d p oi nt as a bi n o mi al r a n d o m v ari a bl e.  A r es p o n d er is d efi n e d as a p ati e nt 
m e eti n g t h e pri m ar y e n d p oi nt. T h e st atistic al d esi g n f or t h e pri m ar y e n d p oi nt is b as e d o n a bi n o mi al 
e x a ct t est f or t h e n ull h y p ot h esis of a r es p o n d er r at e (i. e m e eti n g t h e pri m ar y e n d p oi nt) b ei n g 7 0 % 
or l ess a n d t h e alt er n ati v e h y p ot h esis of a r es p o n d er of 9 5 % or m or e. T h e crit eri o n f or s u cc ess of t h e 

Pr ot oc ol: M G T A -1 4 5 + pl eri x af or m o bili z ati o n  
 
Pr ot oc ol V ersi o n 1. 4 , 2 1 A pril  2 0 2 1  
6 4  
I R B-5 7 0 5 6  
 
tri al is at l e ast 1 3 o ut of 1 5 p ati e nts ar e r es p o n d ers. T h e t y p e I err or r at e is 5 % a n d st atistic al p o w er 
is 8 5 %.  
T his d esi g n f e at ur es a n i m plicit f utilit y st o p pi n g r ul e . P ati e nts will b e e nr oll e d c o nti n u o usl y t o t h e 
first s e g m e nt aft er i niti al s af et y r u n-i n. T h e crit eri o n f or s uc c ess is t h at at l e ast 1 3 o ut of 1 5 p ati e nts 
ar e r es p o n d ers. T his i m pli es t h at if 3 or m or e p ati e nts ar e n o n -r es p o n d ers at a n y p oi n t, t h e n t h e tri al 
will b e st o p p e d f or f utilit y.  
T h e c oll e cti o n of ≥4. 0 x 1 0 ^ 6 C D 3 4 + wit hi n 2 d a ys a k e y s e c o n d a r y e n d p oi nt  of t his tri al.  W e 
h y p ot h esi z e t h at at l e ast 7 5 % of t h e 2 5 p ati e nts will m e et t his s e c o n d ar y e n d p oi nt  T h e  st atistic al 
d esi g n f or t h e s e c o n d ar y e n d p oi nt is b as e d o n e nr olli n g e n o u g h p ati e nts i n t h e s e c o n d s e g m e nt s uc h 
t h at t h e t ot al s a m pl e si z e will pr o d uc e a pr e cisi o n esti m at e wit h a l o w er bi n o mi al c o nfi d e nc e li mit 
t h at e xcl u d es t h e r es p o ns e r at e of t h e hist oric al c o ntr ols (i. e., 5 0 %). F or t his s e c o n d ar y e n d p oi nt, w e 
c o m p ut e a pr e cisi o n esti m at e of o ur h y p ot h esi z e d r es p o ns e r at e f or t h e sc e n ari o t h at b ot h s e g m e nts 
ar e c o m pl et e d. W e m o d el t h e s e c o n d ar y e n d p oi nt as a bi n o mi al r a n d o m v ari a bl e. W e d efi n e pr e cisi o n 
t o b e t h e l e n gt h of t h e bi no mi al c o nfi d e nc e i nt er v al f or t h e h y p ot h esi z e d r es p o ns e r at e  of 7 5 %. W e 
c o m p ut e pr e cisi o n usi n g t h e m et h o d of Wils o n at t h e si g nific a nc e l e v el of 9 0 % w hic h is a p pr o pri at e 
f or a n e arl y-p h as e tri al. B as e d o n t h es e s p e cific ati o ns, t h e pr e cisi o n is 2 6. 8 %.  
E nr o ll e d p ati e nts m a y b e r e pl a c e d if t h e y wit h dr a w c o ns e nt or if t h er e is a c h a n g e i n tr e at m e nt pl a n 
f or a n y r e as o n b ef or e st art of st u d y tr e at m e nt t o all o w f or 2 5 e v al u a bl e p ati e nts. 
1 3. 6. 3  Eff e ct si z e j u stifi c ati o n  
Hist oric al d at a fr o m cli nic al st u di es of m o biliz ati o n wit h diff er e nt r e gi m e ns  p artic ul arl y G -C S F al o n e  
i n p ati e nts wit h m ulti pl e m y el o m a (T a bl e 1 1 ) w as e v al u at e d t o d et er mi n e eff e ct si z e. S ucc e ss r at e of 
c oll e cti n g ≥2 . 0 x  1 0 6  C D 3 4 +  c ells / k g i n u p t o t w o d a ys of a p h er esis aft er m o biliz ati o n wit h M G T A -1 4 5 
a n d pl eri x af or w as esti m at e d t o b e at l e ast 9 5 % b as e d o n d at a b el o w a n d fr o m r es ults of t h e p h as e 1 
st u d y wit h M G T A-1 4 5 a n d pl eri x af or i n h e alt h y v ol u nt e ers 5, 7, 9, 1 0, 2 2, 5 3   Diff er e nc es i n r e gi m e ns a n d 
v ari a bl e  pri m ar y a n d s e c o n d ar y e n d p oi nt t ar g ets w er e t a k e n i nt o a cc o u nt . 
T a bl e 1 1 : St e m c ell m o bili z ati o n d at a f r o m p ri o r st u di es of G-C S F a n d G C S F + / -pl e ri x af o r i n 
m y el o m a  
St u d y  
M o bili z ati o n R e gi m e n  
R es ult s  
Fl o m er b er g et al., 
2 0 0 5  
G -C S F + Pl eri x af or  a n d G -C S F al o n e  
C o m p aris o n of H S C s m o biliz e d wit h G -C S F 
+ Pl eri x af or vs. G -C S F al o n e , 9 of 2 5 
p ati e nts ( 3 6 %) di d n ot m e et 2 milli o n 
C D 3 4 + c ells / k g c oll e cti o n g o al i n t h e G -
C S F al o n e att e m pt.  N ot e: M o bili z ati o n 
w as all o w e d b e y o n d 2 a p h er esis s essi o ns  
Di P ersi o et al., 2 0 0 9  
G -C S F + Pl eri x af or vs. G -C S F al o n e  
Pri m ar y e n d p oi nt w as c oll e cti o n  of 6 
milli o n C D 3 4 c ells / k g i n 2 a p h er esis d a ys.  
Pri m ar y e n d p oi nt m et b y 7 1 % of p ati e nts 
i n G-C S F + Pl eri x af or ar m vs. 3 4 % of 
p ati e nts  i n G-C S F o nl y ar m . 

Pr ot oc ol: M G T A -1 4 5 + pl eri x af or m o bili z ati o n  
 
Pr ot oc ol V ersi o n 1. 4 , 2 1 A pril  2 0 2 1  
6 5  
I R B-5 7 0 5 6  
 
Mic all ef et al  2 0 1 2  
C o m p aris o n of diff er e nt m o biliz ati o n 
r e gi m e ns 
I n p ati e nts r e c ei vi n g m o biliz ati o n wit h o ut 
pl eri x af or, (i. e G -C S F al o n e or G -C S F wit h  
c h e m ot h er a p y), f ail ur e t o c oll e ct at l e ast 2 
milli o n C D 3 4 + c ells w as s e e n i n 1 9 % of 
p ati e nts.  N ot e: M o biliz ati o n w as all o w e d 
b e y o n d 2 a p h er esis s essi o ns  
Si n h a et al 2 0 1 2  
Diff er e nt m o biliz ati o n r e gi m e ns i n 
p ati e nts r e c ei vi n g l e n ali d o mi d e b as e d 
i n d ucti o n 
1 1 % of p ati e nts i n t h e G C S F ar m di d n ot 
m e et c oll e cti o n g o al of 2 milli o n C D -3 4 + 
c ells / k g. N ot e: M o biliz ati o n w as all o w e d 
b e y o n d 2 a p h er esis s essi o ns  
S h a u g h n ess y et al., 
2 0 1 3  
G -C S F + S C Pl eri x af or  
2 milli o n C D 3 4 + c ells / k g i n u p t o 3 d a ys of 
a p h er esis w as a c hi e v e d b y 9 8 % of 
p ati e nts wit h M M.  
N ot e: M o biliz ati o n w as all o w e d b e y o n d 2 
a p h er esis s essi o ns  
I nstit uti o n al D at a ( u n p u blis he d): A m o n gst p ati e nts u n d er g oi n g A S C T f or 
m y el o m a  at St a nf or d 
U ni v ersit y M e dic al C e nt er fr o m J a n 2 0 1 7 -M arc h 2 0 2 0, w e a n al y z e d 6 9 c o ns e c uti v e p ati e nts w h o 
u n d er w e nt c oll e cti o n wit h G C S F 1 0 mc g / k g a n d as n e e d e d pl eri x af or ( w hic h w as gi v e n w h e n C D 3 4 
c o u nt o n d a y 4 w as < 1 0). Pl eri x af or w as n e e d e d i n 5 5 % ( n = 3 8) p ati e nts.  M e di a n n u m b er of 
a p h er esis w as 2, wit h r a n g e of 1 t o 5. 9 8. 5 % p ati e nts ( 6 8 of 6 9) c oll e ct e d ≥2. 0 x 1 0 ^ 6 C D 3 4 + c ells / k g. 
7 8 % of p ati e nts ( 5 5 of 6 9) c oll e ct e d ≥4. 0 x 1 0 ^ 6 C D 3 4 + c ells / k g. 6 4 % ( 4 4 o ut of 6 9) p ati e nts c oll e ct e d 
≥4. 0 x 1 0 ^ 6 C D 3 4 + wit hi n 2 d a ys . 
1 3. 7  C rit e ri a f o r f ut u r e st u di es  
If t h e st u d y m e ets its pri m ar y e n d p oi nt, f ut ur e st u di es will b e pl a n n e d wit h t his c o m bi n ati o n r e gi m e n 
f or st e m c ell m o biliz ati o n pri or t o A S C T i n p ati e nts wit h m y el o m a. 

Pr ot oc ol: M G T A -1 4 5 + pl eri x af or m o bili z ati o n  
 
Pr ot oc ol V ersi o n 1. 4 , 2 1 A pril  2 0 2 1  
6 6  
I R B-5 7 0 5 6  
 
1 4  R E F E R E N C E S  
 
1.  
Attal M, La u wers -Ca n ces V, H uli n C, et al. Le n ali d o mi de, B ort ez o mi b, a n d 
De xa met h as o ne wit h Tra ns pla ntati o n f or M yel o ma. N E n gl J Me d. 2 0 1 7; 3 7 6( 1 4): 1 3 1 1 -
1 3 2 0.  
2.  
Afifi S, A del N G, De vli n S, et al. U pfr o nt pleri xaf or pl us G -C S F vers us 
cycl o p h os p ha mi de pl us G -C S F f or ste m cell m o bili z ati o n i n m ulti ple m yel o ma: effica c y 
a n d c ost a nal ysis st u d y. B o ne m arr o w tr a ns pl a nt ati o n. 2 0 1 6; 5 1( 4): 5 4 6 -5 5 2.  
3.  
A ntar A, Otr oc k Z K, K harfa n -Da baja M A, et al. G -C S F pl us pree m pti ve pleri x af or vs 
h y perfracti o n ate d C Y pl us G-C S F f or a ut ol o g o us ste m cell m o biliz ati o n i n m ulti pl e 
m yel o m a: eff ecti ve ness, safet y a n d c ost a nal ysis. B o ne m arr o w tr a ns pl a nt ati o n. 
2 0 1 5; 5 0( 6): 8 1 3 -8 1 7.  
4.  
Ja gasia M H, Sa va ni B N, Neff A, Di x o n S, C he n H, Pic kar d A S. O utc o me, t o xicit y 
pr ofile  a n d c ost a nal ysis of a ut ol o g o us ste m cell m o biliz ati o n. B o ne m arr o w 
tr a ns pl a nt ati o n. 2 0 1 1; 4 6( 8): 1 0 8 4 -1 0 8 8.  
5.  
Di Persi o J F, Sta dt m a uer E A, Na de ma nee A, et al. Pl eri xaf or a n d G -C S F v ers us place b o 
a n d G-C S F t o m o biliz e he m at o p oi eti c ste m cells f or a ut ol o g o us  ste m cell tra ns pl a ntati o n 
i n pati e nts wit h m ulti ple m y el o m a. Bl o o d. 2 0 0 9; 1 1 3( 2 3): 5 7 2 0 -5 7 2 6.  
6.  
H o g gatt J, Si n g h P, Tate T A, et al. Ra pi d M o biliz ati o n Re veals a Hi g hl y E n grafta ble 
He mat o p oi eti c Ste m C ell. C ell. 2 0 1 8; 1 7 2( 1 -2): 1 9 1 -2 0 4.e 1 1 0.  
7.  
Di Persi o J F, H o g gatt J, De vi ne S, et al. R a pi d a n d R o b ust M o bilizati o n of C D 3 4 + H S Cs 
wit h o ut G -C S F F oll o wi n g A d mi nistrati o n of M gta -1 4 5 Al o ne or i n C o m bi n ati o n wit h 
Pleri xaf or. Bl o o d. 2 0 1 9; 1 3 4( S u p ple me nt _ 1): 1 9 6 1 -1 9 6 1.  
8.  
K u mar S K, Mi k hael J, La pl a nt B, et al. P hase 2 tr ial of i ntra ve n o usl y a d mi nistere d 
pleri xaf or f or st e m cell m o bili zati o n i n patie nts wit h m ulti ple m y el o m a f oll o wi n g 
le nali d o mi d e-base d i niti al t her a p y. B o ne m arr o w tr a ns pl a nt ati o n. 2 0 1 4; 4 9( 2): 2 0 1 -2 0 5.  
9.  
Fl o m e n ber g N, De vi ne S M, Di persi o J F, et al. T he use  of A M D 3 1 0 0 pl us G -C S F f or 
a ut ol o g o us he m at o p oi eti c pr o ge nit or cell m o biliz ati o n is s u peri or t o G -C S F al o n e. Bl o o d. 
2 0 0 5; 1 0 6( 5): 1 8 6 7 -1 8 7 4.  
1 0.  
S ha u g h ness y P, U berti J, De vi ne S, et al. Pl eri xaf or a n d G -C S F f or a ut ol o g o us ste m cell 
m o bili zati o n i n patie nts wit h N H L, H o d g ki n's l y m p h o ma a n d m ulti ple m y el o m a: res ults 
fr o m t he e x p a n de d ac cess pr o gra m. B o n e m arr o w tr a ns pl a nt ati o n. 2 0 1 3; 4 8( 6): 7 7 7 -7 8 1.  
1 1.  
N CI S ur veilla nc e Ea E R S p. Ca ncer Stat Facts: M yel o ma. 2 0 2 0; 
htt ps://se er.ca n cer. g o v/statfacts/ ht ml/ m ul m y. ht ml . A ccesse d A pril, 2 0 2 0. 
1 2.  
Mc El wai n TJ, P o wles R L. Hi g h -d ose i ntra ve n o us m el p hal a n f or plas m a -cell le u k ae mia 
a n d m yel o ma. L a ncet ( L o n d o n, E n gl a n d). 1 9 8 3; 2( 8 3 5 4): 8 2 2 -8 2 4.  
1 3.  
K u mar S K, Raj k u mar S V, Dis pe nzieri A, et al. I m pr o ve d s ur vi val i n m ulti ple m yel o ma 
a n d t he i m pact of n o vel t hera pi es. Bl o o d. 2 0 0 8; 1 1 1( 5): 2 5 1 6 -2 5 2 0.  
1 4.  
T h orstei ns d ottir S, Dic k ma n P W, La n d gre n O, et al. Dra mati call y i m pr o v e d s ur vi val i n 

Pr ot oc ol: M G T A -1 4 5 + pl eri x af or m o bili z ati o n  
 
Pr ot oc ol V ersi o n 1. 4 , 2 1 A pril  2 0 2 1  
6 7  
I R B-5 7 0 5 6  
 
m ulti ple m y el o m a patie nts i n t he rec e nt de ca de: res ults fr o m a S we dis h p o p ulati o n -base d 
st u d y. H ae m at ol o gic a. 2 0 1 8; 1 0 3( 9):e 4 1 2 -e 4 1 5.  
1 5.  
G o nsal ves WI, B u a di F K, Aila wa d hi S, et al. U tili zati o n of he mat o p oietic st e m cell 
tra ns pla nt ati o n f or t he tre at m e nt of m ulti ple m y el o m a: a Ma y o Stratifi cati o n of M yel o ma 
a n d Ris k -A da pte d T h era p y ( m S M A R T) c o nse ns us state m e nt. B o n e m arr o w 
tr a ns pl a nt ati o n. 2 0 1 8.  
1 6.  
Perr ot A, La u wers -Ca nces V, C orre J,  et al. Mi ni m al resi d ual dise ase ne gati vit y usi n g 
dee p se q ue nci n g is a m aj or pr o g n ostic f act or i n m ulti ple m yel o ma. Bl o o d. 
2 0 1 8; 1 3 2( 2 3): 2 4 5 6 -2 4 6 4.  
1 7.  
Ga y F, Cerrat o C, Petr uc ci M T, et al. Effica c y of carfilz o mi b le n ali d o mi de 
de xa met has o ne ( K R d) wit h or wi t h o ut tra ns pla ntati o n i n ne wl y dia g n ose d m yel o ma 
acc or di n g t o ris k stat us: R es ults fr o m t h e F O R T E tri al. J o ur n al of Cli ni c al O nc ol o g y. 
2 0 1 9; 3 7( 1 5 _s u p pl): 8 0 0 2 -8 0 0 2.  
1 8.  
D ha kal B, Sza b o A, C h ha bra S, et al. A ut ol o g o us Tr a ns pla ntati o n f or Ne wl y Dia g n ose d 
M ult i ple M yel o ma i n t he Era of N o vel A ge nt I n d u cti o n: A S yste mati c Re vi e w a n d Meta-
a nal ysis. J A M A o nc ol o g y. 2 0 1 8; 4( 3): 3 4 3 -3 5 0.  
1 9.  
K u mar S, Dis pe nzieri A, L ac y M Q, et al. I m pact of l e nali d o mi de t hera p y o n st e m cell 
m o bili zati o n a n d e n gr aft me nt p ost -peri p h eral bl o o d ste m c ell tra ns pla ntati o n i n patie nts 
wit h ne wl y dia g n ose d m yel o ma. Le uke mi a. 2 0 0 7; 2 1( 9): 2 0 3 5 -2 0 4 2.  
2 0.  
Giralt S, C osta L, Sc hri ber J, et al. O pti mizi n g A ut ol o g o us Ste m Cell M o biliz ati o n 
Strate gi es t o I m pr o ve Patie nt O utc o mes: C o nse ns us G ui deli nes a n d R ec o m me n dati o ns. 
Bi ol o gy of Bl o o d a n d M arr o w Tr a ns pl a nt ati o n. 2 0 1 4; 2 0( 3): 2 9 5 -3 0 8.  
2 1.  
Gert z M A, K u mar S K, Lac y M Q, et al. C o m p aris o n of hi g h -d ose C Y a n d gr o wt h fact or 
wit h gr o wt h fact or al o ne f or m o bili zati o n of ste m cells f or tra ns pla nt ati o n i n patie nts  wit h 
m ulti ple m y el o m a. B o ne m arr o w tr a ns pl a nt ati o n. 2 0 0 9; 4 3( 8): 6 1 9 -6 2 5.  
2 2.  
Micall ef I N, Si n ha S, Gasti nea u D A, et al. C ost -effe cti ve n ess a nal ysis of a ris k -a da pte d 
al g orit h m of pleri xaf or use f or a ut ol o g o us peri p her al bl o o d ste m c ell m o bilizati o n. 
Bi ol o g y of bl o o d a n d m arr o w tr a ns pl a nt ati o n : j o ur n al of t he A meric a n S ociet y f or Bl o o d 
a n d M arr o w Tr a ns pl a nt ati o n. 2 0 1 3; 1 9( 1): 8 7 -9 3.  
2 3.  
C osta LJ, Miller A N, Ale xa n d er E T, et al. Gr o wt h fa ct or a n d patie nt -a da pt e d use of 
pleri xaf or is s u peri or t o C Y a n d gr o wt h f act or f or a ut ol o g o us he mat o p oietic ste m cells 
m o bili zati o n. B o ne m arr o w tr a ns pl a nt ati o n. 2 0 1 1; 4 6( 4): 5 2 3 -5 2 8.  
2 4.  
C he n Y B, Le -Ra de mac her J, Bra za us kas R, et al. Pleri xaf or al o n e f or t he m o bili zati o n 
a n d tra ns pla ntati o n of H L A -matc h e d si bli n g d o n or h e mat o p oi etic ste m cells. Bl o o d 
a dv a nces. 2 0 1 9; 3( 6): 8 7 5 -8 8 3.  
2 5.  
Ki n g A G, J o ha ns o n K, Fre y C L, et al. I d e ntificati o n of u ni q ue tr u nc ate d K C/ G R O bet a 
c he m o ki n es wit h p ote nt he m at o p oi etic a n d a nti -i nfe cti ve a cti viti es. J o ur n al of 
i m m u n ol o gy ( B alti m ore, M d : 1 9 5 0). 2 0 0 0; 1 6 4( 7): 3 7 7 4 -3 7 8 2.  
2 6.  
Ra dt ke S, A dair J E, Giese M A, et al. A disti n ct he m at o p oietic st e m c ell p o p ulati o n f or 
ra pi d m ultili nea g e e n graft me nt i n n o n h u ma n pri mates. Scie nce tr a nsl ati o n al me dici n e. 

Pr ot oc ol: M G T A -1 4 5 + pl eri x af or m o bili z ati o n  
 
Pr ot oc ol V ersi o n 1. 4 , 2 1 A pril  2 0 2 1  
6 8  
I R B-5 7 0 5 6  
 
2 0 1 7; 9( 4 1 4).  
2 7.  
K u mar S, Pai va B, A n ders o n K C, et al. I nte r nati o nal M yel o ma W or ki n g Gr o u p 
c o nse ns us criteri a f or res p o nse a n d mi ni mal resi d ual disease assess me nt i n m ulti ple 
m yel o m a. L a ncet O nc ol. 2 0 1 6; 1 7( 8): e 3 2 8 -e 3 4 6.  
2 8.  
Stetler -St e ve ns o n M, Pai va B, St o ol ma n L, et al. C o nse ns us g ui deli nes f or m yel o ma 
mi ni m al r esi d u al dise ase sa m pl e stai ni n g a n d dat a ac q uisiti o n. Cyt o metry P art B, Cli nic al 
cyt o metry. 2 0 1 6; 9 0( 1): 2 6 -3 0.  
2 9.  
Ra wstr o n A C, Orfa o A, Be ksac M, et al. Re p ort of t he E ur o pea n M yel o ma Net w or k o n 
m ulti p ara m etri c fl o w c yt o metr y i n m ulti ple m yel o m a a n d rel ate d dis or ders. 
H ae m at ol o gic a. 2 0 0 8; 9 3( 3): 4 3 1 -4 3 8.  
3 0.  
Frisc h BJ, Cal vi L M. He mat o p oietic st e m c ell c ult ur es a n d assa ys. Met h o ds i n m olec ul ar 
bi ol o gy ( Clift o n, N J). 2 0 1 4; 1 1 3 0: 3 1 5 -3 2 4.  
3 1.  
Pa nti n J, P ure v E, Ti a n X, et al. Effect of hi g h -d ose pleri xaf or o n C D 3 4( +) c ell 
m o bili zati o n i n he alt h y st e m c ell d o n ors: res ults of a ra n d o miz e d cr oss o ver trial. 
H ae m at ol o gic a. 2 0 1 7; 1 0 2( 3): 6 0 0 -6 0 9.  
3 2.  
Le mer y SJ, Hsie h M M, S mit h A, et al. A pil ot st u d y e val uati n g t h e safet y a n d C D 3 4 + 
cell m o bilizi n g acti vit y of escalati n g d oses of pleri xaf or i n healt h y v ol u nt eers. Britis h 
j o ur n al of h ae m at ol o g y. 2 0 1 1; 1 5 3( 1): 6 6 -7 5.  
3 3.  
P orrata L F, Gertz M A, Ge yer S M, et al. T he d ose of i nf use d l y m p h oc ytes i n t h e a ut o gr aft 
directl y c orrelates wit h cli nical o utc o me after a ut ol o g o us peri p heral bl o o d he m at o p oietic 
ste m cell tr a ns pla nt ati o n i n m ulti pl e m yel o ma. Le uk e mi a. 2 0 0 4; 1 8( 6): 1 0 8 5 -1 0 9 2.  
3 4.  
P orrata L F. A ut o graft i m m u n e effect or cells a n d s ur vi val i n a ut ol o g o us peri p heral b l o o d 
he mat o p oietic ste m cell tra ns pl a ntati o n. J Cli n A p h er. 2 0 1 8; 3 3( 3): 3 2 4 -3 3 0.  
3 5.  
Var ma v u o V, Ma nt y ma a P, Sil ve n n oi n e n R, N o usiai ne n T, K uitti ne n T, Ja nt u ne n E. 
C D 3 4 + cell s u bcl asses a n d l y m p h oc yte s u bsets i n bl o o d grafts c oll ect e d after v ari o us 
m o bili za ti o n met h o ds i n m yel o ma p ati e nts. Tr a nsf usi o n. 2 0 1 3; 5 3( 5): 1 0 2 4 -1 0 3 2.  
3 6.  
G o o d Z, B or ges L, Vi v a nc o G o nzalez N, et al. Pr olif erati o n traci n g wit h si n gl e -cell mass 
c yt o metr y o pti miz es ge nerati o n of ste m cell me m or y -li ke T cells. N at Bi otec h n ol. 
2 0 1 9; 3 7( 3): 2 5 9 -2 6 6.  
3 7.  
Ster n L, Mc G uire H, A v dic S, et al. Mass C yt o metr y f or t he Assess me nt of I m m u ne 
Rec o nstit uti o n After He mat o p oietic Ste m Cell Tra ns pla ntati o n. Fr o ntiers i n i m m u n ol o gy. 
2 0 1 8; 9: 1 6 7 2.  
3 8.  
Lei p ol d M D, Maec k er H T. P he n ot y pi n g of Li ve H u ma n P B M C usi n g C y T O F T M Mass 
C yt o metr y. Bi o -pr ot oc ol. 2 0 1 5; 5( 2): e 1 3 8 2.  
3 9.  
Hart ma n n FJ, Ba b d or J, G her ar di ni P F, et al. C o m pr e he nsi ve I m m u ne M o nit ori n g of 
Cli nic al Trials t o A d va n ce H u ma n I m m u n ot her a p y. Cell R e p. 2 0 1 9; 2 8( 3): 8 1 9 -8 3 1. e 8 1 4.  
4 0.  
Sa haf B, Ra h ma n A, Mae c ker H T,  Be n dall S C. Hi g h -Para met er I m m u n e Pr ofili n g wit h 
C y T O F. Met h o ds i n m ol ec ul ar bi ol o gy ( Clift o n, N J). 2 0 2 0; 2 0 5 5: 3 5 1 -3 6 8.  
4 1.  
Perez -A n dres M, Pai va B, Niet o W G, et al. H u m a n p eri p heral bl o o d B -c ell 

Pr ot oc ol: M G T A -1 4 5 + pl eri x af or m o bili z ati o n  
 
Pr ot oc ol V ersi o n 1. 4 , 2 1 A pril  2 0 2 1  
6 9  
I R B-5 7 0 5 6  
 
c o m part me nts: a cr ossr oa d i n B -cell tr affic. C yt o metry P art B, Cli nic al cyt o metry. 
2 0 1 0; 7 8 S u p pl 1: S 4 7 -6 0.  
4 2.  
T u n g J W, He y dari K, Tir o u v a nzia m R, et al. M o der n fl o w c yt o metr y: a pr acti cal 
a p pr oac h. Cli n L a b Me d. 2 0 0 7; 2 7( 3): 4 5 3 -4 6 8, v.  
4 3.  
va n D o n ge n JJ, L her mitte L, B ottc h er S, et al. E ur o Fl o w a nti b o d y pa n els f or sta n dar dize d 
n -di me nsi o nal fl o w c yt o metric i m m u n o p he n ot y pi n g of n or mal, re acti ve a n d m ali g n a nt 
le u k oc yt es. Le uke mi a. 2 0 1 2; 2 6( 9): 1 9 0 8 -1 9 7 5.  
4 4.  
Effica ce F, Gai da n o G, L o -C oc o F. Patie nt -r e p orte d o utc o mes i n h e mat ol o g y: is it ti me t o 
f oc us m ore o n t he m i n cli ni cal trials a n d h e mat ol o g y practi ce? Bl o o d. 2 0 1 7; 1 3 0( 7): 8 5 9 -
8 6 6.  
4 5.  
Mc Carter SJ, Vija y var gi ya P, Si da na S, et al. A case of i br uti ni b -ass ociat e d as per gill osis 
prese nti n g wit h ce ntr al ner v o us s yste m, m y o car di al, p ul m o nar y, i ntra m usc ular, a n d 
s u bc uta ne o us a bscesses. Le uk Ly m p h o m a. 2 0 1 9; 6 0( 2): 5 5 9 -5 6 1.  
4 6.  
Cleel a n d C S, R ya n K. T he bri ef pai n i n ve nt or y user g ui de. [I nter n et]. 1 9 9 1; 
htt p:// w w w. m da n ders o n. or g/e d uc ati o n -a n d-r esearc h/ de part m e nts-pr o gra ms -a n d-
la bs/ de part me nts-a n d-di visi o ns/s y m pt o m -resear c h/s y m pt o m-assess me nt -
t o ols/ B PI _ User G ui de. p df. Ac cesse d Mar 2, 2 0 1 7. 
4 7.  
Da ut R L, Cle ela n d C S, Fla n er y R C. De vel o p me nt of t he Wisc o nsi n Brief Pai n 
Q uesti o n naire t o assess pai n i n c a ncer a n d ot her dise ases. P ai n. 1 9 8 3; 1 7( 2): 1 9 7 -2 1 0.  
4 8.  
Me n d oza T, Ma y ne T, R u blee D, Cleel a n d C. Relia bilit y a n d vali dit y of a m o difie d Bri ef 
Pai n I n ve nt or y s h o rt f or m i n patie nts wit h ost e oart hritis. E ur o pe a n j o ur n al of p ai n 
( L o n d o n, E n gl a n d). 2 0 0 6; 1 0( 4): 3 5 3 -3 6 1.  
4 9.  
Zal o n M L. Usi n g a n d u n derst a n di n g fact or a nal ysis: t he Brief Pai n i n ve nt or y. N urse 
rese arc her. 2 0 0 6; 1 4( 1): 7 1 -8 4.  
5 0.  
Mat hias S D, Cr os b y R D, Qia n Y,  Jia n g Q, Da nse y R, C h u n g K. Esti mati n g mi ni mall y 
i m p orta nt differ e nces f or t he w orst pai n rati n g of t he Brief Pai n I n ve nt or y-S h ort F or m. 
T he j o ur n al of s u p p ortive o n c ol o gy. 2 0 1 1; 9( 2): 7 2 -7 8.  
5 1.  
Mease PJ, S paet h M, Cla u w DJ, et al. Esti mati o n of mi ni m u m cli nic all y i m p ort a nt 
differe nc e f or pai n i n fi br o m yal gia. Art hritis c are & rese arc h. 2 0 1 1; 6 3( 6): 8 2 1 -8 2 6.  
5 2.  
Yarlas A, Miller K, We n W, et al. B u pre n or p hi n e tra ns der mal s yste m c o m par e d wit h 
place b o  r e d uces i nterfere n ce i n f u n cti o ni n g f or c hr o nic l o w bac k p ai n. P ost gr a d Me d 
2 0 1 5; 1 2 7( 1): 3 8 -4 5.  
5 3.  
Si n ha S, Gert z M A, Lac y M Q, et al. Maj orit y of pati e nts recei vi n g i niti al t h era p y wit h 
le nali d o mi d e-base d re gi me ns ca n b e s uccessf ull y m o biliz e d wit h a p pr o p riat e 
m o bili zati o n strat e gies. Le uk e mi a. 2 0 1 2; 2 6( 5): 1 1 1 9 -1 1 2 2.  
 
 

Pr ot oc ol: M G T A -1 4 5 + pl eri x af or m o bili z ati o n  
 
Pr ot oc ol V ersi o n 1. 4 , 2 1 A pril  2 0 2 1  
7 0  
I R B-5 7 0 5 6  
 
1 5  A P P E N DI C E S  
1 5. 1  A P P E N DI X A : P a rti ci p a nt Eli gi bilit y C h e c kli st  
A P artici p a nt Eli gi bilit y C h e c klist m ust b e c o m pl et e d i n its e ntir et y f or e a c h s u bj e ct pri or t o 
r e gistr ati o n. T h e c o m pl et e d, si g n e d, a n d d at e d c h e c klist m ust b e r et ai n e d i n t h e p ati e nt’s st u d y fil e 
a n d t h e st u d y’s R e g ul at or y Bi n d er.  
 
T h e st u d y c o or di n at or , tr e ati n g p h ysici a n a n d a n i n d e p e n d e nt r e vi e w er m ust v erif y t h at t h e 
p artici p a nt’s eli gi bilit y  is a cc ur at e, c o m pl et e, a n d l e gi bl e i n s o urc e r e c or ds. A d escri pti o n of t h e 
eli gi bilit y v erific ati o n pr oc ess s h o ul d b e i ncl u d e d i n t h e E PI C  or ot h er El e ctr o nic M e dic al R e c or d  
pr o gr ess n ot e .  
 
T h e f oll o wi n g is t h e P artici p a nt Eli gi bilit y c h e c klist t e m pl at e  t h at will b e us e d f or t his pr ot oc ol 
   
B M T 3 6 2 : P h as e II St u d y of M G T A -1 4 5 i n c o m bi n ati o n wit h pl eri x af or i n t h e m o biliz ati o n of 
h e m at o p oi etic st e m c ells f or a ut ol o g o us tr a ns pl a nt ati o n i n p ati e nts wit h m ulti pl e m y el o m a  
S u r b hi Si d a n a, M D  
S u bj e ct N a m e / M R N:  
 
G e n d er:    
 M al e     
 F e m al e  
 
I ncl u si o n C rit eri a  
 
_____   Di a g n osis of m ulti pl e m y el o m a p er t h e I nt er n ati o n al M y el o m a W or ki n g Gr o u p (I M W G) crit eri a.  
 
_____   A g e: 1 8 t o 7 0 y e ars  
 
_____   Eli gi bl e f or A S C T p er i nstit uti o n al g ui d eli n es  
 
_____   Wit hi n o n e y e ar of st art of m y el o m a t h er a p y 
 
 
_____   C ar di a c a n d p ul m o n ar y st at us s uffici e nt t o u n d er g o a p h er esis a n d tr a ns pl a nt ati o n p er 
i nstit uti o n al tr a ns pl a nt g ui d eli n es. 
 
 
_____   C alc ul at e d cr e ati ni n e cl e ar a nc e > 3 0 m L / mi n acc or di n g t o t h e M o dific ati o n of Di et i n R e n al 
Dis e as e ( M D R D) f or m ul a  

Pr ot oc ol: M G T A -1 4 5 + pl eri x af or m o bili z ati o n  
 
Pr ot oc ol V ersi o n 1. 4 , 2 1 A pril  2 0 2 1  
7 1  
I R B-5 7 0 5 6  
 
 
 
_____   A bs ol ut e n e utr o p hil c o u nt >  1 5 0 0 x 1 0 6 / L  a n d pl at el ets >  1 0 0, 0 0 0 x 1 0 6 / L  
 
_____   A bilit y t o u n d erst a n d a n d t h e willi n g n ess t o si g n a writt e n i nf or m e d c o ns e nt d oc u m e nt.  
 
_____   A gr e e m e nt t o us e a n a p pr o v e d f or m of c o ntr ac e pti o n if of c hil d b e ari n g p ot e nti al 
 
 
E x cl u si o n C rit e ri a  
 
_____   Hist or y of pri or st e m c ell tr a ns pl a nt f or m ulti pl e m y el o m a or ot h er i n dic ati o ns  
 
_____   Pl a n n e d t a n d e m st e m c ell tr a ns pl a nt 
 
_____   Pri or hist or y of f ail ur e t o c oll e ct H S Cs. 
 
_____  Li v er f u ncti o n t ests: T ot al bilir u bi n > 1. 5 x u p p er li mit of n or m al ( U L N) i n t h e a bs e nc e of a 
d oc u m e nt e d hist or y of Gil b ert’s s y n dr o m e a n d / or A S T / A L T > 3 x U L N  
 
_____   K n o w n all er g y t o M G T A -1 4 5 or pl eri x af or  
 
_____   Lif eti m e e x p os ur e t o l e n ali d o mi d e gr e at er t h a n 6 c u m ul ati v e m o nt hs of tr e at m e nt i. e m or e 
t h a n si x 2 8-d a y c ycl es or m or e t h a n ei g ht 2 1 -d a y c ycl es  
 
_____   Pr e g n a nt or l a ct ati n g w o m e n 
 
Tr e ati n g P h ysici a n Si g n at ur e:  
D at e:  
Pri nt e d N a m e:  
 
 

Pr ot oc ol: M G T A -1 4 5 + pl eri x af or m o bili z ati o n  
 
Pr ot oc ol V ersi o n 1. 4 , 2 1 A pril  2 0 2 1  
7 2  
I R B-5 7 0 5 6  
 
 
S e c o n d ar y R e vi e w er Si g n at ur e:  
D at e:  
Pri nt e d N a m e:  
 
 
St u d y C o or di n at or Si g n at ur e:  
D at e:  
Pri nt e d N a m e:  
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 

Pr ot oc ol: M G T A -1 4 5 + pl eri x af or m o bili z ati o n  
 
Pr ot oc ol V ersi o n 1. 4 , 2 1 A pril  2 0 2 1  
7 3  
I R B-5 7 0 5 6  
 
1 5. 2   A P P E N DI X B : P R O Q u esti o n n ai r e   
 
1.  H a v e y o u n otic e d a n y p ai n, s w elli n g or r e d n ess at a sit e of dr u g i nj e cti o n or I V  t o d a y? 
( A d a pt e d fr o m P R O -C T C A E)  
 
o  Y es  
o  N o  
 
2.  If y es, w h at w as t h e s e v erit y of t h e s y m pt o ms at w ors e? ( A d a pt e d fr o m P R O-C T C A E)  
 
o  N o n e  
o  Mil d  
o  M o d er at e  
o  S e v er e  
o  V er y S e v er e  
 
 
 
If y o u a n s w er e d y es t o e x p e ri e n ci n g p ai n t o d a y, pl e a s e a n s w er t h e f oll o wi n g q u esti o n s 
 
3.  H o w l o n g di d t h e p ai n l ast? 
 
o  < 2 0 mi ns  
o  2 0 mi ns - 1 h o ur  
o  M or e t h a n 1 h o ur  
 
 
B ri ef p ai n i n v e nt o r y t o ol  ( S h o rt F o r m, S a m pl e):  

Pr ot oc ol: M G T A -1 4 5 + pl eri x af or m o bili z ati o n  
 
Pr ot oc ol V ersi o n 1. 4 , 2 1 A pril  2 0 2 1  
7 4  
I R B-5 7 0 5 6  
 
 
 
 
 

Pr ot oc ol: M G T A -1 4 5 + pl eri x af or m o bili z ati o n  
 
Pr ot oc ol V ersi o n 1. 4 , 2 1 A pril  2 0 2 1  
7 5  
I R B-5 7 0 5 6