Clinical trial NCT04552743
Conditions: Multiple Myeloma
Sponsor: Surbhi Sidana, MD
Start date: 2020-10-05; Completion date: 2022-06-30
Enrollment: 25 participants; Allocation: NA; Intervention model: SINGLE_GROUP

Brief summary:
This study evaluates a new drug MGTA-145 in combination with plerixafor (Mozobil) to mobilize stem cells into the peripheral blood for collection by apheresis. The stem cells will be used for autologous stem cell transplant for treatment of multiple myeloma.

Detailed description:
PRIMARY OBJECTIVE

1\. To assess the efficacy of MGTA-145 in combination with plerixafor in mobilizing adequate number of hematopoietic stem cells (\> 2 x 10e6 CD34+ cells/kg) in patients with multiple myeloma (MM) in preparation for autologous stem cell transplantation (ASCT).

SECONDARY OBJECTIVES

1. To assess the efficacy of MGTA-145 and plerixafor in mobilizing different Hematopoietic stem cells (HSCs) target goals in patients with MM in preparation for ASCT.
2. To assess the safety and tolerability of MGTA-145 and plerixafor for mobilizing HSCs in patients with MM.
3. To assess the engraftment rate and time to engraftment following ASCT after HSC mobilization with MGTA-145 and plerixafor in patients with MM undergoing upfront ASCT.
4. To assess rate of ongoing engraftment at Day 30 and 100 after stem cell infusion in patients with MM who are mobilized with MGTA-145 and plerixafor undergoing upfront ASCT.
5. To assess transplant and disease-related outcomes after mobilization of HSCs with MGTA-145 and plerixafor in patients with MM undergoing upfront ASCT.

Arms: arm 1: Patients after screening will undergo baseline evaluation during the premobilization phase up to 30 days before mobilization. Patients will undergo sequential administration of plerixafor 0.24 mg/kg subcutaneously followed 2 hours later by MGTA-145 at 0.03 mg/kg intravenously (3 to10 minute infusion). This will be followed by apheresis. A second day of mobilization and apheresis will be pursued in patients who have not collected 6.0 x 106 CD34+ cells/kg in one session.
Interventions: intervention 1: MGTA-145 intervention 2: Plerixafor
Intervention descriptions: intervention 1: A chemokine receptor type 2 (CXCR2) agonist protein, administered via intravenous (IV) infusion over 3 to 10 minutes. intervention 2: An azamacrocycle CXCR4 chemokine receptor antagonist, administered at 0.24 mg/kg subcutaneously, reduced to 0.16 mg/kg in patients with renal dysfunction (per package insert).

Protocol documents:
Prot_SAP_000.pdf: 
 
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  


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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. 
 


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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  
 


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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 


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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 


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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  
 
 
 

ICF_001.pdf: 
 
 
 
STANFORD UNIVERSITY   Research Consent Form 
Protocol Director: 
Surbhi Sidana, MD 
Protocol Title:   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    
 
 
Page 1 of 23 
 
NCT : NCT04552743 
IRB Use Only 
Approval Date:   August 17, 2021 
Expiration Date: August 17, 2022 
Participant ID: 
 
Are you participating in any other research studies? _____ Yes _____No 
 
Concise Summary: This research study tests a new medicine for mobilizing stem 
cells (helping push stem cells out of the the bone marrow into the blood) so they 
can be collected and used for autologous stem cell transplant for treatment of your 
multiple myeloma. This investigational medicine (MGTA-145) will be given with 
plerixafor. Plerixafor is approved by the United States Food and Drug 
Administration (FDA) for mobilizing stem cells and is currently given with another 
medicine called G-CSF (granulocye colonly stimulating factor). The combination of 
MGTA-145 and plerixafor in this study is investigational. We think giving you the 
MGTA-145 with plerixafor will allow us to collect the necessary number of stem 
cells for your future transplant in less time and with less side effects than the 
standard mobilization procedure of plerixafor and G-CSF. Usually the plerixafor and 
G-CSF must be given over 4 or 5 days before the stem cell collection (called 
‘apheresis’). Chemotherapy may also be given to help ‘mobilize’ stem cells and it 
also requires several days to mobilize the stem cells. Both of these standard 
methods can require several days of blood collection (apheresis).  
 
In this study plerixafor and MGTA-145 will be followed by the stem cell collection 
on the same day, and both may be repeated the next day, for a maximum of two 
days. If successful, the benefit of this approach is it will also allow us to shorten 
the number of apheresis collections to only two days. This approach could limit 
side effects that may occur with medicines like G-CSF.  
 
This approach is experimental as MGTA-145 is not approved by the FDA. MGTA-
145 has been studied in healthy volunteers, when given alone and when given 
with with plerixafor. It has shown promising results for stem cell mobilization. 
Overall, it was found to be safe. In the group of healthy volunteers who were given 
MGTA-145 alone, 80% reported back pain, which was short lived and went away 
within 20 minutes in the majority of people. Amongst healthy volunteers who 
received MGTA-145 and plerixafor, people also reported brief periods of back pain. 
Another side effect reported with this combination was diarrhea, which is 
commonly observed with plerixafor. Side effects of MGTA-145 and plerixafor are 
listed in detail later in this document. No new or unexpected side effects were seen 
with the combination of plerixafor and MGTA-15 in the study of healthy volunteers. 
 
If the number of stem cells needed for your transplant cannot be collected with 
this investigational combination of medicines, your doctor may ask you to have 
another approach for collecting stem cells, which may require that you take a break 


 
 
 
STANFORD UNIVERSITY   Research Consent Form 
Protocol Director: 
Surbhi Sidana, MD 
Protocol Title:   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    
 
 
Page 2 of 23 
 
NCT : NCT04552743 
IRB Use Only 
Approval Date:   August 17, 2021 
Expiration Date: August 17, 2022 
Participant ID: 
to let your bone marrow recover before having another stem cell mobilization and 
apheresis procedure. 
 
After stem cell collection, we will ask you to come back for a clinic visit within one 
week. If you are not planning a stem cell transplant within two months after the 
cell collection, we will call you one month after collection. If you don’t have any 
side effects from the collection at that time, no further follow-up is needed on this 
study. 
 
If you and your doctors are planning a stem cell transplant within two months of 
stem cell collection, we will follow you in clinic and with phone calls for up to four 
months after the transplant, as described below.  
 
Participation in this study is voluntary. If you chose not to participate, your doctor 
will consider alternative approaches for mobilizing your stem cells before apheresis 
including G-CSF and plerixafor or chemotherapy and G-CSF, with plerixafor if 
needed. These are described in detail in this consent. 
  
 
PURPOSE OF RESEARCH 
 
You were selected as a possible participant in this study because you have multiple 
myeloma and are being considered for an autologous stem cell transplant, either in 
the immediate future or later in your disease course. To prepare for the transplant 
we need to collect special cells called ‘hematopoetic stem cells’. 
 
All of the cells of the immune system including red cells, white cells and platelets 
are made in the bone marrow. All of these cells develop from a type of cell found in 
the bone marrow called a "hematopoietic stem cell." This is the type of cell collected 
for blood and marrow transplantation. In order to collect enough hematopoietic 
stem cells from the bone marrow to make a successful transplant, we have to move 
the cells from the bone marrow to the blood. Once they are in the blood, they can 
be collected during a procedure called ‘apheresis’. Currently this requires several 
days of different medicines (4-5 days) and several days of apheresis. In this study 
you will be given an investigational medicine (MGTA-145) in combination with an 
FDA approved medicine called plerixafor. Currently plerixafor given with granulocyte 
colony stimulating factor or G-CSF is the procedure approved by the FDA for stem 
cell collection. In this study you will not receive G-CSF. 
 


 
 
 
STANFORD UNIVERSITY   Research Consent Form 
Protocol Director: 
Surbhi Sidana, MD 
Protocol Title:   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    
 
 
Page 3 of 23 
 
NCT : NCT04552743 
IRB Use Only 
Approval Date:   August 17, 2021 
Expiration Date: August 17, 2022 
Participant ID: 
The MGTA-145 is being provided by Magenta Therapeutics, the study medicine’s 
manufacturing company and will be provided to you at no charge. 
 
We hope to learn whether the combination of MGTA-145 and plerixafor, given on 
the same morning as stem cell collection for up to two days, allows us to collect 
enough stem cells to do an autologous stem cell transplant. We will also evaluate 
the side effects of this new medicine combination and whether there is any effect 
on the transplant, including engraftment (the repopulation of normal blood cells 
with the infused stem cells) and myeloma response, for up to three months after 
your transplant. We will also ask you questions about your pain and how you are 
feeling after receiving the MGTA-145 and plerixafor. 
 
If you consent to participate in this study, the study team will review your 
treatment history and results of laboratoty tests and other tests ordered by your 
transplant doctor as part of your transplant evaluation. This is to determine 
whether you meet the criteria to receive MGTA-145.  
 
If you meet the criteria, you will first be given plerixafor as a shot or injection 
under the skin, followed 2 hours later by the investigational study drug (MGTA-
145), which is given as an intravenous (IV) infusion (into a vein in your arm). This 
infusion will take up to ten minutes. Following MGTA-145, you will start the stem 
cell collection procedure (apheresis). This procedure will last at least 4.5 hours and 
up to 6 to 7 hours, depending on your height and weight. Your blood will be drawn 
through a needle in your arm or through a central venous catheter (CVC) and will 
then pass through an apheresis machine, which identifies stem cells by targeting 
a marker on them called CD34. The medicines and apheresis may be repeated the 
next day depending on the number of stem cells collected. The goal is to collect 
at least 2 million CD34+ stem cells/kg of your body weight in two days.  
 
If enough cells are not collected on these two days, your physician may ask you 
to use another method for mobilizing stem cells after after you have a rest.  
 
You can decide to stop participation at any time during this study. If you decide to 
stop participating in this study, you should notify   
 
Surbhi Sidana, MD 
300 Pastuer Drive H0101c 
Stanford, CA 94305 
Phone Number: 650-723-0822 
Email: surbhi.sidana@stanford.edu 


 
 
 
STANFORD UNIVERSITY   Research Consent Form 
Protocol Director: 
Surbhi Sidana, MD 
Protocol Title:   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    
 
 
Page 4 of 23 
 
NCT : NCT04552743 
IRB Use Only 
Approval Date:   August 17, 2021 
Expiration Date: August 17, 2022 
Participant ID: 
 
This research study is looking for 25 people with multiple myeloma who need stem 
cells collected for autologous stem cell transplantation. At this time, this study is 
only open at Stanford University. 
 
After one to two days of MGTA and plerixafor followed by stem cell collection, you 
will not receive any further investigational agent. The remainder of the procedures, 
including transplant will be done according to Stanford University Blood and 
Marrow Transplantation (BMT) Division’s standard institutional practice. 
 
VOLUNTARY PARTICIPATION  
 
Your participation in this study is entirely voluntary.  Your decision not to 
participate will not have any negative effect on you or your medical care.  You can 
decide to participate now, but withdraw your consent later and stop being in the 
study without any loss of benefits or medical care to which you are entitled. 
 
DURATION OF STUDY INVOLVEMENT  
 
This research study is expected to take approximately 24 months to recruit all 
participants and report results.  
 
Your active participation in the study including the medicines for cell mobilization, 
apheresis, clinic visits or phone calls is for a duration of two to six months.  
 
If you are scheduled for transplant within two months of stem cell collection, we 
will see you in clinic and call you on the phone for up to four months after your 
transplant. If you are NOT planning a transplant within two months of collecting 
your stem cells, we will follow you for up to one month after stem cell collection, 
including clinic visits/phone calls.  
 
After the active follow-up period, which includes clinic visits/phone calls, we may 
continue to collect information on your health status, myeloma treatment and 
transplant history from your electronic medical record periodically, whichever is 
later. If these stored stem cells are shipped to another medical center for a stem 
cell transplant, we will request that center send us medical records about your 
stem cell transplant after getting your authorization to do so. 
 
 
 


 
 
 
STANFORD UNIVERSITY   Research Consent Form 
Protocol Director: 
Surbhi Sidana, MD 
Protocol Title:   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    
 
 
Page 5 of 23 
 
NCT : NCT04552743 
IRB Use Only 
Approval Date:   August 17, 2021 
Expiration Date: August 17, 2022 
Participant ID: 
 
PROCEDURES 
 
If you choose to participate, Surbhi Sidana, MD, the Protocol Director and her 
research study staff will consent you to the study.  
 
The study is divided into three phases: 
 
1. 
Pre-mobilization phase: 
 
After you sign informed consent, you will have a physical examination, blood 
tests and other tests that are routinely done before stem cell collection and 
transplant per Stanford University BMT Division’s guidelines and standard 
practices. The study team will review the results of these tests, as well as 
review your medical history and myeloma treatment history to determine 
whether you are eligibile to participate in the study and proceed with stem 
cell mobilization.  
 
2. Mobilzation phase: This phase of the study is the only phase which 
involves the investigational/experimental medicine. A summary is provided 
in Table 1. 
 
On your scheduled day of stem cell mobilization, you will receive plerixafor 
injection under the skin (subcutaneously), followed two hours later by 
MGTA-145, which is given by vein (IV - intravenously) over 3 to 10 minutes. 
A few minutes after that you will start the stem cell collection procedure/ 
apheresis, which will last at least 4.5 hours and may last up to 6-7 hours 
depending on your height and weight. Our goal is for the machine to 
circulate and process your entire blood volume at least three times to collect 
enough stem cells. 
 
Apheresis or stem cell collection is done through a peripheral intravenous 
catheter (small plastic tube in your arm) or a central line, which is a 
intravenous line placed in your neck. The decision regarding the type of 
approach used for stem cell collection is made by your treating physician 
and the apheresis team. Participation in this study will not impact the choice 
of catheter used for apheresis. 
 
You will have at least three blood samples drawn on each day of 
mobilization/stem cell collection. The first sample will be collected in the 


 
 
 
STANFORD UNIVERSITY   Research Consent Form 
Protocol Director: 
Surbhi Sidana, MD 
Protocol Title:   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    
 
 
Page 6 of 23 
 
NCT : NCT04552743 
IRB Use Only 
Approval Date:   August 17, 2021 
Expiration Date: August 17, 2022 
Participant ID: 
morning, the second one will be collected approximately 15-30 minutes 
after MGTA-145 infusion and the third at the end of apheresis. Additional 
samples may be collected if anything is not normal.  This is according to 
Stanford BMT standard guidelines and is not specific to this research. 
 
We will also ask you questions about side effects you have and ask you to 
fill out questionnaires on each day of stem cell collection and in the follow-
up clinic visit after stem cell collection. These questionnaires pertain to side 
effects, pain and quality of life during collection. 
 
The stem cells collected will be processed per Stanford BMT/Cell Therapy 
Facilty standard procedures for evaluating the stem cell count and for 
cryopreservation i.e storage under very cold temperatures. This is done for 
all stem cell collections in patients with myeloma.  
 
A part of the collected stem cell product, about 15 mL will be stored for 
research purposes. The goal of these research tests is to understand the 
nature and effectiveness of the stem cells collected with this new technique 
and other cells or components that are present in the collected stem cell 
product. 
 
 
 
Table 1: Study Medicines and Stem Cell Collection Procedures 
 
Medication/Procedure Day 1 
Day 2 (If 
needed) 
Plerixafor 
One dose given subcutaneously  
Same as day 1 
MGTA-145 
(Investigational 
medicine) 
One dose given as an intravenous 
infusion over 3 to 10 minutes. This will 
be given 2 hours after plerixafor. 
Same as day 1 
Apheresis procedure to 
collect stem cells 
At least 4.5 hours and can last upto 6-
7 hours depending on your height and 
weight. Remaining details of this 
procedure will be per Stanford 
University BMT division’s standard 
practices. 
Same as day 1 
Questionnaire asking 
about symptoms 
After receiving MGTA-145 
Same as day 1 


 
 
 
STANFORD UNIVERSITY   Research Consent Form 
Protocol Director: 
Surbhi Sidana, MD 
Protocol Title:   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    
 
 
Page 7 of 23 
 
NCT : NCT04552743 
IRB Use Only 
Approval Date:   August 17, 2021 
Expiration Date: August 17, 2022 
Participant ID: 
Blood Samples 
At least three blood samples: 
 
Before receiving plerixafor for routine 
blood work (standard practice) 
 
After receiving MGTA-145 to check 
CD34 count (research) 
 
After stem cell collection to check 
CD34 count (research) 
 
Additional samples as clinically needed 
for your care 
Same as day 1 
 
3. Follow-up phase 
 
The follow up procedures will be slightly different depending upon when you and 
your transplant doctor plan for you to receive the stem cell transplant: 
 
Patients not planning a stem cell transplant within two months of stem 
cell collection: You will be asked to follow-up in clinic within one week after stem 
cell collection. This is routinely done as part of follow-up after collection. You will 
receive a follow-up phone call one month after stem cell collection. Additional 
follow-up may be needed if you experience any side effects related to collection 
that are still present at that time. Beyond this point, we may periodically review 
your medical records to get information about your myeloma treatment and overall 
health or when you come back for a transplant using the stored stem cells. 
 
Patients planning to proceed with stem cell transplantation within two 
months of stem cell collection: All procedures, treatment and follow-up 
related to stem cell transplant will be done per standard practices of the Stanford 
BMT Division. 
 
In addition we will look at the following data to see if the investigational 
medicine for mobilizing stem cells affects your transplant course: 
 
We will check your blood counts to see how long it takes them to recover after 
transplant. We will also collect other details pertaining to transplant and 
myeloma response. 
 


 
 
 
STANFORD UNIVERSITY   Research Consent Form 
Protocol Director: 
Surbhi Sidana, MD 
Protocol Title:   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    
 
 
Page 8 of 23 
 
NCT : NCT04552743 
IRB Use Only 
Approval Date:   August 17, 2021 
Expiration Date: August 17, 2022 
Participant ID: 
You will be asked to come to clinic around one month and three months after 
transplant as part of study follow-up. This is routinely done as part of follow-up 
for transplant patients. A research blood sample will be taken along with your usual 
tests. This research sample involves collection of 15 mL of blood, which is 
approximately one tablespoon.  
 
A bone marrow biopsy is typically done as part of routine care two to three months 
after stem cell transplant. If you are undergoing a bone marrow aspirtate and 
biopsy after transplant at Stanford University, we will also collect an extra research 
sample from the bone marrow aspirate. This sample will be 5 mL or one teaspoon. 
 
Additional follow-up may be needed if you are still experiencing side effects that 
are thought to be related to the investigational drug. Beyond this point, we may 
periodically evaluate your medical records to monitor your myeloma treatment 
and general health. 
 
This research will not include whole genome sequencing. Any of your specimens 
which are used in research may result in new products, tests or discoveries. In 
some instances, these may have potential commercial value and may be developed 
and owned by the Investigators, Stanford University and/or others.  However, 
donors of specimens do not retain any property rights to the materials.  Therefore, 
you would not share in any financial benefits from these products, tests or 
discoveries. 
 
Future Use of Private Information and/or Specimens  
 
Research using private information and/or specimens is an important way to try 
to understand human disease.  You are being given this information because the 
investigators want to save private information and/or specimens for future 
research. Magnenta Pharmaceuticals, the company manufacturing the MGTA-145 
may receive your study information and some of your study samples as part of 
this research.  
 
A part of your specimens will be stored under very cold temperatures in the labs 
at Stanford BMT Division and Cell Therapy Facility. Your specimens will be stored 
frozen with your study identification number, not your name or other information 
that can identify you personally. Only the investigator and study team have access 
to the information linking you to your study identification number, which is kept 
secure in a locked file or password protected secure server. 
 


 
 
 
STANFORD UNIVERSITY   Research Consent Form 
Protocol Director: 
Surbhi Sidana, MD 
Protocol Title:   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    
 
 
Page 9 of 23 
 
NCT : NCT04552743 
IRB Use Only 
Approval Date:   August 17, 2021 
Expiration Date: August 17, 2022 
Participant ID: 
 
Identifiers might be removed from identifiable private information and/or 
identifiable specimens and, after such removal, the information and/or specimens 
could be used for future research studies or distributed to another investigator for 
future research studies without additional informed consent from you.  
 
PARTICIPANT RESPONSIBILITIES           
 
As a participant, your responsibilities include:  
 
• Follow the instructions of the Protocol Director and study staff. 
• Keep your study appointments. If it is necessary to miss an appointment, 
please contact the Protocol Director or research study staff to reschedule 
as soon as you know you will miss the appointment. 
• Tell the Protocol Director or research study staff about any side effects, 
doctor visits, or hospitalizations that you may have. 
• Tell the Protocol Director or research staff if you believe you might be 
pregnant or gotten your partner pregnant. 
• Complete your questionnaires as instructed. 
• Ask questions as you think of them. 
• Tell the Protocol Director or research staff if you change your mind about 
staying in the study. 
 
WITHDRAWAL FROM STUDY 
 
If you first agree to participate and then you change your mind, you are free to 
withdraw your consent and discontinue your participation at any time. Your 
decision will not affect your ability to receive medical care for your disease and 
you will not lose any benefits to which you would otherwise be entitled.  
 
If you decide to withdraw your consent to participate in this study, you should 
notify Surbhi Sidana, MD at 650-723-0822 with a verbal request to withdraw from 
the study or submit a verbal or written request to the study team or your Stanford 
BMT doctor.  
 
If you withdraw from the study before stem cell collection, no treatment with 
MGTA-145 will be given. Your team will continue your routine clinical care 
 


 
 
 
STANFORD UNIVERSITY   Research Consent Form 
Protocol Director: 
Surbhi Sidana, MD 
Protocol Title:   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    
 
 
Page 10 of 23 
 
NCT : NCT04552743 
IRB Use Only 
Approval Date:   August 17, 2021 
Expiration Date: August 17, 2022 
Participant ID: 
The Protocol Director may also withdraw you from the study and the study 
medication may be stopped, without your consent for one or more of the following 
reasons:   
o Failure to follow the instructions of the Protocol Director and 
study staff. 
o The Protocol Director decides that continuing your 
participation could be harmful to you. 
o You need treatment not allowed in the study, include need for 
stem cell mobilization with other treatments. 
o The study is cancelled.  
o Other administrative reasons. 
o Unanticipated circumstances. 
 
POSSIBLE RISKS, DISCOMFORTS, AND INCONVENIENCES  
 
There are risks, discomforts, and inconveniences associated with any research 
study. These deserve careful thought. You should talk with the Protocol Director if 
you have any questions. 
 
MGTA-145: 
 
In healthy volunteers receving MGTA-145, the most common side effect with 
MGTA-145 was lower back/muscle pain seen in the majority of (60-80%) of 
volunteers. Back pain was usually short lived and improved without any medication 
within 20 minutes in the majority of patients. When MGTA-145 was used in 
combination with plerixafor in healthy volunteers, other side effects were reported 
as listed below. Overall the drug combination was considered safe, with mild side 
effects. 
 
Frequent/most common side effects (seen in more than 10% of 
volunteers) after treatment with MGTA-145 and plerixafor 
 
- 
Back pain/musculoskeletal pain  
- 
Diarrhea  
- 
Nausea  
- 
Dizziness  
- 
Abdominal pain  
- 
Headache 
 
Less common side effects (seen in less than 10% of volunteers) 


 
 
 
STANFORD UNIVERSITY   Research Consent Form 
Protocol Director: 
Surbhi Sidana, MD 
Protocol Title:   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    
 
 
Page 11 of 23 
 
NCT : NCT04552743 
IRB Use Only 
Approval Date:   August 17, 2021 
Expiration Date: August 17, 2022 
Participant ID: 
 
- 
Vomiting  
- 
Abnormal tingling sensation  
- 
Fatigue 
- 
Abdominal discomfort 
- 
Chest pain 
- 
Muscle/bone pain 
Allergic and infusion related reaction to MGTA-145 are also possible, though not 
observed in humans yet.  It is also possible that you develop an antibody to MGTA-
145, though it has also not been observed in humans yet. 
  
As we are testing a new drug, it may involve risks which are currently 
unforeseeable.  
 
Plerixafor: 
 
The most common side effects (≥10%) in patients who received 
plerixafor with G-CSF in clinical studies were: 
 
- 
Diarrhea 
- 
Nausea 
- 
Fatigue 
- 
Injection site reactions 
- 
Headache 
- 
Joint Pain 
- 
Dizziness 
- 
Vomiting 
 
Less common (<10%) 
- 
High white cell count 
- 
Difficulty sleeping 
- 
Flatulence 
 
Rare: 
- 
Spleen enlargement/ spleen rupture 
- 
Passing out/nearly passing out 
- 
Allergic reactions with plerixafor are seen in less than 1% of patients. 
Women of Childbearing Potential 


 
 
 
STANFORD UNIVERSITY   Research Consent Form 
Protocol Director: 
Surbhi Sidana, MD 
Protocol Title:   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    
 
 
Page 12 of 23 
 
NCT : NCT04552743 
IRB Use Only 
Approval Date:   August 17, 2021 
Expiration Date: August 17, 2022 
Participant ID: 
If you are a woman who is able to become pregnant, it is expected that you 
will use an effective method of birth control to prevent exposing a fetus to a 
potentially dangerous agent with unknown risk.  If you are pregnant or 
currently breast feeding, you may not participate in this study.  You 
understand that if you are pregnant, if you become pregnant, or if you are 
breast-feeding during this study, you or your child may be exposed to an 
unknown risk. 
To confirm to the extent medically possible that you are not pregnant, you 
agree to have a pregnancy test done before beginning this research study.   
You must agree to avoid sexual intercourse or use a birth control method 
judged to be effective by the investigator and which will not interfere with the 
proposed investigation. You must accept the risk that pregnancy could still 
result despite the responsible use of reliable method of birth control.  You 
agree to notify the investigator as soon as possible of any failure of proper 
use of your birth control method, or if you become pregnant, either of which 
may result in your being withdrawn from the study. 
If you are a man participating in this study and your partner is able to 
become pregnant, you and your partner must use adequate (at least two 
methods of highly effective) contraception while you are participating in the 
study and for at least 90 days after administration of study medication.  
You should also agree to not donate sperm for at least 90 days after 
administration of study medication. Your doctor will discuss with you what 
methods of birth control are considered adequate. You should inform your 
study doctor if your partner becomes pregnant. 
POTENTIAL BENEFITS                              
 
A potential benefit of this study is that stem cell mobilization and collection will be 
completed in two days without any significant additional side effects. Tranditional 
methods can take several additional days to mobilize and collect stem cells.  
 
However, we cannot and do not guarantee or promise that you will receive any 
benefits from this study. 
 
ALTERNATIVES 
 


 
 
 
STANFORD UNIVERSITY   Research Consent Form 
Protocol Director: 
Surbhi Sidana, MD 
Protocol Title:   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    
 
 
Page 13 of 23 
 
NCT : NCT04552743 
IRB Use Only 
Approval Date:   August 17, 2021 
Expiration Date: August 17, 2022 
Participant ID: 
You may chose to undergo stem cell mobilization and collection with alternative 
strategies that are available. Two common strategies include: 
 
1) Chemotherapy and GCSF (granulocyte colony simulating factor) 
based mobilization with as needed use of plerixafor: In this method, 
you are given intravenous chemotherapy, typically cyclophosphamide over 
one day. You have to come to the treatment center for tests and IV fluids 
one day before and one day after the chemotherapy. After chemotherapy 
is given, you are given daily G-CSF and asked to do daily blood count 
checks. Your blood counts drop over the first few days and then gradually 
start coming up. Once your blood counts come up beyond a certain number 
(typically two weeks after chemotherapy), we ask you to come in to get a 
CD34 count (stem cell count) checked. If CD34 count is adequate per 
institutional requirements, you may start stem cell collection the next day. 
If it is not adequate, your doctor may decide to treat you with plerixafor 
before stem cell collection. You will  continue GCSF and plerixafor (if it has 
been added) on each day of stem cell collection. If CD34 counts are not 
high enough either before colletion starts or with collection, your doctor 
may make a decision to abandon the proedure and proceed with alternative 
methods. 
 
The benefit of stem cell collection with this procedure is that it has been 
used for a long time for stem cell collection before transplant in myeloma 
and is a well established method. 
 
Risks with this method include the risks that come with chemotherapy, 
including risk for fever and infectious complications when your blood counts 
are low, potential need of blood product transfusion, as well small risks for 
kidney and other organ damage. Other side effects include bone pain and 
night sweats from G-CSF. If plerixafor is used, additional side effects related 
to plerixafor may occur as described above, including diarrhea. 
 
2. GCSF with on demand plerixafor: In this method you are given four days 
of injections with G-CSF, which is given subcutaneously i.e under the skin. On the 
fourth day, we check stem cell CD34 counts. If they are below the a certain 
number, you are given plerixafor subcutaneously and start stem cell collection 
procedure the next day. You continue both G-CSF and plerixafor on each day of 
collection. About half the patients will receive plerixafor based on our experience 
and published literature. 
 


 
 
 
STANFORD UNIVERSITY   Research Consent Form 
Protocol Director: 
Surbhi Sidana, MD 
Protocol Title:   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    
 
 
Page 14 of 23 
 
NCT : NCT04552743 
IRB Use Only 
Approval Date:   August 17, 2021 
Expiration Date: August 17, 2022 
Participant ID: 
Side effects with GCSF include bone pain, low grade fever and night sweats and 
the rare chance of serious organ damage like spleen rupture. Side effects with 
plerixafor are described above including diarrhea, which is the most common side 
effect. These side effects typically resolve within a few days of stopping these 
medications.  
 
The benefit of stem cell collection with this procedure is that it has been used for 
a long time for stem cell collection before transplant in myeloma and is a well 
established method. The disadvantage is that you need four days of GCSF 
injections before collection. After four days of injection, the collection period can 
vary from one day to up to four or five days of collection. 
 
PARTICIPANT’S RIGHTS 
 
You should not feel obligated to agree to participate.  Your questions should be 
answered clearly and to your satisfaction.  If you decide not to participate, tell the 
Protocol Director.   
 
You will be told of any important new information that is learned during the course 
of this research study, which might affect your condition or your willingness to 
continue participation in this study. 
 
ClinicalTrials.gov 
 
A description of this clinical trial will be available on 
http://www.ClinicalTrials.gov, as required by U.S. Law.  This Web site will not 
include information that can identify you.  At most, the Web site will include a 
summary of the results.  You can search this Web site at any time. 
 
CONFIDENTIALITY 
 
The results of this research study may be presented at scientific or medical 
meetings or published in scientific journals. Your identity and/or your personal 
health information will not be disclosed except as authorized by you or as required 
by law.  However, there is always some risk that even de-identified information 
might be re-identified.  
 


 
 
 
STANFORD UNIVERSITY   Research Consent Form 
Protocol Director: 
Surbhi Sidana, MD 
Protocol Title:   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    
 
 
Page 15 of 23 
 
NCT : NCT04552743 
IRB Use Only 
Approval Date:   August 17, 2021 
Expiration Date: August 17, 2022 
Participant ID: 
Patient information may be provided to Federal and other regulatory agencies as 
required.  The Food and Drug Administration (FDA), for example, may inspect 
research records and learn your identity if this study falls within its jurisdiction. 
 
The purpose of this research study is to obtain information on the safety and 
effectiveness of MGTA-145. The data and information regarding your participation 
in this study will be provided to Magenta Therapeutics, the company who is 
providing the drug. In addition information may be provided to the Food and Drug 
Administration and other federal and regulatory agencies as required. 
This research is covered by a Certificate of Confidentiality from the National 
Institutes of Health. The researchers with this Certificate may not disclose or use 
information, documents, or biospecimens that may identify you in any federal, 
state, or local civil, criminal, administrative, legislative, or other action, suit, or 
proceeding, or be used as evidence, for example, if there is a court subpoena, 
unless you have consented for this use. Information, documents, or 
biospecimens protected by this Certificate cannot be disclosed to anyone else 
who is not connected with the research except, if there is a federal, state, or 
local law that requires disclosure (such as to report child abuse or communicable 
diseases but not for federal, state, or local civil, criminal, administrative, 
legislative, or other proceedings, see below); if you have consented to the 
disclosure, including for your medical treatment; or if it is used for other scientific 
research, as allowed by federal regulations protecting research subjects.  
The Certificate cannot be used to refuse a request for information from 
personnel of the United States federal or state government agency sponsoring 
the project that is needed for auditing or program evaluation by the National 
Center for Advancing Translational Sciences which is funding this project or for 
information that must be disclosed in order to meet the requirements of the 
federal Food and Drug Administration (FDA).  You should understand that a 
Certificate of Confidentiality does not prevent you from voluntarily releasing 
information about yourself or your involvement in this research. If you want your 
research information released to an insurer, medical care provider, or any other 
person not connected with the research, you must provide consent to allow the 
researchers to release it. 
The Certificate of Confidentiality will not be used to prevent disclosure as 
required by federal, state, or local law of such as child abuse and neglect, or 
harm to self or others.  


 
 
 
STANFORD UNIVERSITY   Research Consent Form 
Protocol Director: 
Surbhi Sidana, MD 
Protocol Title:   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    
 
 
Page 16 of 23 
 
NCT : NCT04552743 
IRB Use Only 
Approval Date:   August 17, 2021 
Expiration Date: August 17, 2022 
Participant ID: 
The Certificate of Confidentiality will not be used to prevent disclosure for any 
purpose you have consented to in this informed consent document. Please refer 
to the section entitled “What Personal Information Will Be Obtained, Used 
or Disclosed?”. 
 


 
 
 
STANFORD UNIVERSITY   Research Consent Form 
Protocol Director: 
Surbhi Sidana, MD 
Protocol Title:   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    
 
 
Page 17 of 23 
 
NCT : NCT04552743 
IRB Use Only 
Approval Date:   August 17, 2021 
Expiration Date: August 17, 2022 
Participant ID: 
Authorization To Use Your Health Information 
For Research Purposes  
 
Because information about you and your health is personal and private, 
it generally cannot be used in this research study without your written 
authorization.  If you sign this form, it will provide that authorization.  
The form is intended to inform you about how your health information 
will be used or disclosed in the study.  Your information will only be 
used in accordance with this authorization form and the informed 
consent form and as required or allowed by law.  Please read it 
carefully before signing it.   
 
What is the purpose of this research study and how will my 
health information be utilized in the study? 
 
This is a clinical trial studying a new drug called MGTA-145 in 
combination with plerixafor to collect stem cells in prepeparation for 
autologous stem cell transplant in multiple myeloma. Results of this 
study, which includes de-identified patient information about how well 
this combination worked, side effects, treatment history and outcomes 
with transplant will be reported in scientific presentations and 
publications. The information will also be submitted to Magenta 
Therapeutics, which is providing the drug, and the FDA.  
 
Do I have to sign this authorization form? 
You do not have to sign this authorization form.  But if you do not, you 
will not be able to participate in this research study including receiving 
any research-related treatment. Signing the form is not a condition for 
receiving any medical care outside the study. 
 
If I sign, can I revoke it or withdraw from the research later? 
If you decide to participate, you are free to withdraw your 
authorization regarding the use and disclosure of your health 
information (and to discontinue any other participation in the study) 


 
 
 
STANFORD UNIVERSITY   Research Consent Form 
Protocol Director: 
Surbhi Sidana, MD 
Protocol Title:   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    
 
 
Page 18 of 23 
 
NCT : NCT04552743 
IRB Use Only 
Approval Date:   August 17, 2021 
Expiration Date: August 17, 2022 
Participant ID: 
at any time.  After any revocation, your health information will no 
longer be used or disclosed in the study, except to the extent that 
the law allows us to continue using your information (e.g., necessary 
to maintain integrity of research).  If you wish to revoke your 
authorization for the research use or disclosure of your health 
information in this study, you must write to:  
 
Surbhi Sidana, MD 
300 Pastuer Drive H0101c 
Stanford, CA 94305 
Phone Number: 650-723-0822 
 
What Personal Information Will Be Obtained, Used or 
Disclosed? 
Your health information related to this study, may be used or disclosed 
in connection with this research study, including, but not limited to 
demographics, medical history, physical examination findings, 
myeloma diagnosis, treatment and response, laboratory reports 
include blood/urine tests and pathology reports, imaging reports, 
reports of procedures including echocardiogram and lung function 
tests and medical history.  
 
Who May Use or Disclose the Information? 
The following parties are authorized to use and/or disclose your health 
information in connection with this research study: 
• The Protocol Director (Surbhi Sidana, MD) 
• The Stanford University Administrative Panel on Human Subjects 
in Medical Research and any other unit of Stanford University as 
necessary 
• Research Staff 
  
Who May Receive or Use the Information? 
The parties listed in the preceding paragraph may disclose your health 
information to the following persons and organizations for their use in 
connection with this research study:  


 
 
 
STANFORD UNIVERSITY   Research Consent Form 
Protocol Director: 
Surbhi Sidana, MD 
Protocol Title:   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    
 
 
Page 19 of 23 
 
NCT : NCT04552743 
IRB Use Only 
Approval Date:   August 17, 2021 
Expiration Date: August 17, 2022 
Participant ID: 
• The Office for Human Research Protections in the U.S. 
Department of Health and Human Services 
• Magenta Therapeutics 
• The United States Food and Drug Administration 
• National Institutes of Health  
 
Your information may be re-disclosed by the recipients described 
above, if they are not required by law to protect the privacy of the 
information. 
 
When will my authorization expire? 
Your authorization for the use and/or disclosure of your health 
information will end on December 31, 2045 or when the research 
project ends, whichever is earlier.  
 
 
________________________________             
  ______________ 
Signature of Adult Participant                                        Date 
 
 
________________________________             
 
Print Name of Adult Participant                                         
 
_______________________________                       ______________ 
Signature of Legally Authorized Representative (LAR)          Date 
(e.g., parent, guardian or conservator) 
 
 
________________________________             
 
Print Name of LAR                                         
 
________________________________________________________ 
LAR’s Authority to Act for Participant 
(e.g., parent, guardian or conservator) 
 


 
 
 
STANFORD UNIVERSITY   Research Consent Form 
Protocol Director: 
Surbhi Sidana, MD 
Protocol Title:   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    
 
 
Page 20 of 23 
 
NCT : NCT04552743 
IRB Use Only 
Approval Date:   August 17, 2021 
Expiration Date: August 17, 2022 
Participant ID: 
FINANCIAL CONSIDERATIONS 
 
Payment/Reimbursement 
You will not be paid to participate in this research study. 
 
 
Costs 
If you participate in this study, the study will pay for those services, supplies, 
procedures, and care associated with the study that are not a part of your routine 
medical care.  However, there may be additional costs to you.  These include basic 
expenses like transportation and the personal time it will take to come to the study 
visits.  You and/or your health insurance must pay for services, supplies, 
procedures, and care that are required during this study for routine medical care. 
You will not be charged for the MGTA-145. You will also be responsible for 
any co-payments and/or deductibles as required by your insurance.  
Participation in this study is not a substitute for health insurance.   
 
Magenta Therapeutics is providing financial support and/or material for this study.  
 
Consultative or Financial Relationships 
 
Dr. Surbhi Sidana is a paid advisor to Magenta Therapeutics, Inc. the company 
sponsoring this research study. 
  
COMPENSATION for Research-Related Injury 
 
All forms of medical diagnosis and treatment – whether routine or experimental – 
involve some risk of injury.  In spite of all precautions, you might develop medical 
complications from participating in this study.  If such complications arise, the 
Protocol Director and the research study staff will assist you in obtaining 
appropriate medical treatment.  In the event that you have an injury or illness that 
is directly caused by your participation in this study, care will be provided to you.  
You will not be responsible for any of these costs.  
 
If you receive Medicare benefits, and if the sponsor of this study pays for any 
study-related treatment, complications or injuries, personal information about you, 
your treatment, and your participation in this study will be provided to the sponsor, 
who is required by law to provide it to Medicare. 
 
You do not waive any liability rights for personal injury by signing this form. 
 


 
 
 
STANFORD UNIVERSITY   Research Consent Form 
Protocol Director: 
Surbhi Sidana, MD 
Protocol Title:   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    
 
 
Page 21 of 23 
 
NCT : NCT04552743 
IRB Use Only 
Approval Date:   August 17, 2021 
Expiration Date: August 17, 2022 
Participant ID: 
CONTACT INFORMATION  
 
Questions, Concerns, or Complaints:  If you have any questions, concerns or 
complaints about this research study, its procedures, risks and benefits, or 
alternative courses of treatment, you should ask the Protocol Director, Surbhi 
Sidana, MD at 650-723-0822.  You should also contact him/her at any time if you 
feel you have been hurt by being a part of this study. 
 
Independent Contact:  If you are not satisfied with how this study is being 
conducted, or if you have any concerns, complaints, or general questions about 
the research or your rights as a participant, please contact the Stanford 
Institutional Review Board (IRB) to speak to someone independent of the research 
team at (650)-723-5244 or toll free at 1-866-680-2906.  You can also write to the 
Stanford IRB, Stanford University, 1705 El Camino Real, Palo Alto, CA 94306. 
 
EXPERIMENTAL SUBJECT’S BILL OF RIGHTS 
 
As a research participant you have the following rights.  These rights include but 
are not limited to the participant's right to: 
• 
be informed of the nature and purpose of the experiment;  
• 
be given an explanation of the procedures to be followed in the medical 
experiment, and any drug or device to be utilized;  
• 
be given a description of any attendant discomforts and risks reasonably to 
be expected;  
• 
be given an explanation of any benefits to the subject reasonably to be 
expected, if applicable;  
• 
be given a disclosure of any appropriate alternatives, drugs or devices that 
might be advantageous to the subject, their relative risks and benefits; 
• 
be informed of the avenues of medical treatment, if any available to the 
subject after the experiment if complications should arise;  
• 
be given an opportunity to ask questions concerning the experiment or the 
procedures involved;  
• 
be instructed that consent to participate in the medical experiment may be 
withdrawn at any time and the subject may discontinue participation 
without prejudice;  
• 
be given a copy of the signed and dated consent form; and  
• 
be given the opportunity to decide to consent or not to consent to a medical 
experiment without the intervention of any element of force, fraud, deceit, 
duress, coercion or undue influence on the subject's decision. 
 


 
 
 
STANFORD UNIVERSITY   Research Consent Form 
Protocol Director: 
Surbhi Sidana, MD 
Protocol Title:   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    
 
 
Page 22 of 23 
 
NCT : NCT04552743 
IRB Use Only 
Approval Date:   August 17, 2021 
Expiration Date: August 17, 2022 
Participant ID: 
Signing your name means you agree to be in this study and that you will receive 
a copy of this signed and dated consent form.  
 
 
 
_____________________________________                ____________ 
Signature of Adult Participant 
 
 
 
   
   Date 
 
 
_____________________________________             
Print Name of Adult Participant                                         
 
 
_____________________________________                ____________         
Signature of Legally Authorized Representative (LAR)  
  Date  
(e.g., parent, guardian or conservator)    
 
_____________________________________             
Print Name of LAR                                         
                               
_____________________________________ 
LAR’s Authority to Act for Participant  
 
 
(e.g., parent, guardian or conservator) 
 
 
 
 
 
 
 
_____________________________________                ____________  
Signature of Person Obtaining Consent  
 
   
  Date 
 
 
_____________________________________             
Print Name of Person Obtaining Consent 
The following witness line is to be signed only if the consent is provided as a summary 
form and accompanied by a short form foreign language consent. 
 
 
 
_____________________________________                     ____________ 


 
 
 
STANFORD UNIVERSITY   Research Consent Form 
Protocol Director: 
Surbhi Sidana, MD 
Protocol Title:   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    
 
 
Page 23 of 23 
 
NCT : NCT04552743 
IRB Use Only 
Approval Date:   August 17, 2021 
Expiration Date: August 17, 2022 
Participant ID: 
Signature of Witness                                                              Date 
 
__________________________________              
Print Name of Witness                                         
 
(e.g., staff, translator/interpreter, family member) 
 
• Translated short form must be signed and dated by both the participant (or their 
LAR) AND the witness. 
• The English consent form (referred to as the "Summary Form" in the regulations): 
− Must be signed by the witness AND the Person Obtaining Consent (POC). 
− The non-English speaking participant/LAR does not sign the English consent. 
− The non-English speaking participant/LAR should not sign the HIPAA participant line 
− If the participant or the LAR is non-English speaking, the Person Obtaining Consent 
(POC) must ensure that 1) the LAR's Description of Authority is completed and 2) 
that any questions or options presented by the consent form are documented and 
initialed by the POC on the Summary Form, per the participant's wishes, as they are 
understood during the consent process.