untitled h1
8. 3 (i m m

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
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.  
| Table 10A: Schedule of Study Assessments   | All patients   | No transplant     | Undergoing Upfront Transplant   |               |                        |                 |
|--------------------------------------------|----------------|-------------------|---------------------------------|---------------|------------------------|-----------------|
| Study period                               | Pre-           | Mobilization      | Post-Mobilization               | 30 days post- | Day -2    Day 28 (± 5) | Day +100 (± 10) |
| procedures                                 | mobilization   | mobilization (±5) | to 30'                          |               |                        |                 |
| a                                          |                |                   |                                 |               |                        |                 |
| 1 30                                       | ≤ 7            | Daily             | Within 7 days of                |               |                        |                 |
| dys                                        | dys            | mobilization      |                                 |               |                        |                 |
| Complete medical history                   | x              |                   |                                 |               |                        |                 |
| Inclusion/exclusion                        | X              |                   |                                 |               |                        |                 |
| criteria review                            |                |                   |                                 |               |                        |                 |
| Prior antineoplastic                       | x              |                   |                                 |               |                        |                 |
| therapy review                             |                |                   |                                 |               |                        |                 |
| Bone marrow biopsy and                     | x              | (X) a             |                                 |               |                        |                 |
| aspirate results review for                |                |                   |                                 |               |                        |                 |
| response assessment and                    |                |                   |                                 |               |                        |                 |
| cllularity c                               |                |                   |                                 |               |                        |                 |
| SPEP, FLC and                              | x              | (X) d             |                                 |               |                        |                 |
| immunofixation review                      |                |                   |                                 |               |                        |                 |
| for response assessment                    |                |                   |                                 |               |                        |                 |
| from chart review c                        |                |                   |                                 |               |                        |                 |
| UPEP and immunofixation                    | x              | (X) a             |                                 |               |                        |                 |
| review for response                        |                |                   |                                 |               |                        |                 |
| assessment (as clinically                  |                |                   |                                 |               |                        |                 |
| indicated)c from chart                     |                |                   |                                 |               |                        |                 |
| review                                     |                |                   |                                 |               |                        |                 |
| Height                                     | X              |                   |                                 |               |                        |                 |
| Weight, BSA                                | X              | X                 |                                 |               |                        |                 |
| Physical examination -                     | X              | x                 | x                               |               |                        |                 |
| Vital signs                                | X              | X                 | X                               |               |                        |                 |
untitled h1
9 study calendar

| All patients              | No transplant   | Undergoing Upfront Transplant   |                   |               |                        |                 |
|---------------------------|-----------------|---------------------------------|-------------------|---------------|------------------------|-----------------|
| Study period              | Pre-            | Mobilization                    | Post-Mobilization | 30 days post- | Day -2    Day 28 (± 5) | Day +100 (± 10) |
| procedures                | mobilization    | mobilization (±5)               | to 30°            |               |                        |                 |
| < 30                      | 7 ≤             | Daily                           | Within 7 days of  |               |                        |                 |
| days                      | dys             | mobilization                    |                   |               |                        |                 |
| ECOG score                | X               |                                 |                   |               |                        |                 |
| Serum/urine βHCG          | X               |                                 |                   |               |                        |                 |
| (women of childbearing    |                 |                                 |                   |               |                        |                 |
| potential)                |                 |                                 |                   |               |                        |                 |
| 12-Lead ECG               | X               |                                 |                   |               |                        |                 |
| Urinalysis                | X               | x                               |                   |               |                        |                 |
| Chemistry f               | X               | X                               | X                 | X             |                        |                 |
| x                         |                 |                                 |                   |               |                        |                 |
| Hematology'               | X               | X                               | X                 | X             |                        |                 |
| Peripheral blood CD34+    | X (30 mins ±    |                                 |                   |               |                        |                 |
| counts                    | 15 mins after   |                                 |                   |               |                        |                 |
| MGTA-145                  |                 |                                 |                   |               |                        |                 |
| infusion start            |                 |                                 |                   |               |                        |                 |
| and at end of             |                 |                                 |                   |               |                        |                 |
| apheresis)                |                 |                                 |                   |               |                        |                 |
| Apheresis parameters &h   | X               |                                 |                   |               |                        |                 |
| MGTA-145                  | X               |                                 |                   |               |                        |                 |
| administration R          |                 |                                 |                   |               |                        |                 |
| Plerixafor administration | x               |                                 |                   |               |                        |                 |
| X                         |                 |                                 |                   |               |                        |                 |
| QoL/Pain measuresR        | x               | x                               |                   |               |                        |                 |
| Adverse event             | X               | x                               | x                 |               |                        |                 |
| assessment®               |                 |                                 |                   |               |                        |                 |
| Concomitant medications®  | x               | x                               | x                 |               |                        |                 |  
| All patients            | No transplant   | Undergoing Upfront Transplant   |                   |               |                        |                 |
|-------------------------|-----------------|---------------------------------|-------------------|---------------|------------------------|-----------------|
| Study period            | Pre-            | Mobilization                    | Post-Mobilization | 30 days post- | Day -2    Day 28 (± 5) | Day +100 (± 10) |
| procedures              | mobilization    | mobilization (±5)               | to 30 b           |               |                        |                 |
| ≤ 30                    | 1 ≤             | Daily                           | Within 7 days of  |               |                        |                 |
| days                    | days            | mobilization                    |                   |               |                        |                 |
| Correlative study       | X               | X                               | X                 | Xi            |                        |                 |
| samplesR                |                 |                                 |                   |               |                        |                 |
| Follow-up Phone CalliR  | X               |                                 |                   |               |                        |                 |
| Data collection and     | X               | X                               | X                 |               |                        |                 |
| reporting for secondary |                 |                                 |                   |               |                        |                 |
| outcomes (engraftment,  |                 |                                 |                   |               |                        |                 |
| progression, death) R   |                 |                                 |                   |               |                        |                 |  
Abbreviations: ECOG: Eastern Cooperative Oncology Group, EOS: end of study; FLC: serum free light chains, HIV: Human immunodeficiency virus, QoL: Quality of life, SPEP: serum protein electrophoresis, UPEP: urine protein electrophoresis ª Mobilization with apheresis collection will occur over 1 or 2 days, depending on Day 1 CD34* cell yield.  Timing of mobilization and relative to HSCT is at the Investigator discretion. b Timing of transplant will vary and can be up to 60 days following start of collection per treating physician discretion. Management during transplant, including labs and other clinical evaluation will be per Stanford BMT institutional standardized clinical practice. Data on outcomes will be collected by study staff from electronic medical record or labs done locally on case report forms at periodic intervals up to day +30. In case patients have not engrafted by day 30, data will continue to be collected until engraftment. After Day 30, data on these outcomes from day 30 to 100 will be collected on dy 100 by study staff.  
e Bone marrow biopsy, SPEP, UPEP and immunofixation are standard of care procedures and may be done at non-Stanford local labs. Results of these tests should be reviewed per medical records and documented at the clinic visit d Restaging studies may be done as standard of care between day 60 and 100 visit and may be done locally. Results of the restaging should also be collected at the day 100 Visit.  
* A complete physical examination will be conducted at Pre and Post-Mobilization Visit.  An abbreviated physical examination will be conducted at all other time points. f Clinical chemistry: Albumin, alkaline phosphatase, total bilirubin, bicarbonate, BUN, calcium, chloride, creatinine, glucose, phosphorus, potassium, total protein, SGOT[AST], SGPT[ALT], sodium.  
```
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.

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.

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

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

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.

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

```  
| 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  G r 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                                                                                                                                                                                                                                |  
| 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  G r 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. |
untitled h1
1 0 m e a

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.  
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- 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 bili z 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 e si 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 e s )

T h e pri m ar y o utc o m e is c oll e cti o n of 2 . 0 x 1 0

6
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 bili z 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).
untitled h1
1 0. 2 ot h

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 bili z 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 bili z ati o n ):  
- B as eli n e ( wit hi n 3 d a ys of m o bili z ati o n)  
- O n e a c h d a y of m o bili z ati o n - Wit hi n 7 d a ys aft er m o bili z ati o n
untitled h1
1 0. 2. 2 n

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.
untitled h1
m e a s u

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:
untitled h1
1 0. 2. 3 t

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.  
- 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 / 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 / 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 / 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 / L,  
wit h o ut tr a nsf usi o n wit hi n 4 8 h o urs.
untitled h1
1 0. 2. 4 n

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.
untitled h1
1 0. 2. 5 p

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.
untitled h1
1 0. 2. 6 o

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.
untitled h1
1 0. 2. 7 r

R el e v a nt S u b s et: R es p o ns e b ef or e m o bili z 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 bili z 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
untitled h1
1 1 r e g

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.
untitled h1
1 1. 2. 1 p

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.
untitled h1
1 1. 2. 2 st

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.
untitled h1
1 1. 2. 3 d

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 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.
untitled h1
1 2 c o l

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.
untitled h1
1 3 s t a

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 bili z 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.
untitled h1
1 3. 2 s af

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 bili z 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 bili z 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 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.
untitled h1
1 3. 3 d es

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.
untitled h1
1 3. 4 pri m

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 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 y si 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 bili z 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 y si 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.
untitled h1
1 3. 5. 1 s

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 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 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 C D 3 4 a n d ≥ 4. 0 x 1 0 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 bili z 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 V ersi o n 1. 4 , 2 1 A pril 2 0 2 1 6 1 I R B-5 7 0 5 6 A 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.
untitled h1
1 3. 5. 2 s

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.  
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 bili z 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.
untitled h1
1 3. 5. 3 s

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 / 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 / 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 / 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 / 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.
untitled h1
1 3. 5. 4 s

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
untitled h1
1 3. 5. 5 s

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.
untitled h1
1 3. 6. 1 a

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 bili z 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.
untitled h1
1 3. 6. 2 s

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 bili z 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 bili z 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 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 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.
untitled h1
1 3. 6. 3 eff

Hist oric al d at a fr o m cli nic al st u di es of m o bili z 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 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 bili z 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 e s 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 bili z 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 .                                                                                |              |  
| Mic all ef et al 2 0 1 2                                                                           | C o m p aris o n of diff er e nt m o bili z ati o n  r e gi m e ns                                                           | I n p ati e nts r e c ei vi n g m o bili z 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 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   |                                                   |
|----------------------------------------------------------------------------------------------------|------------------------------------------------------------------------------------------------------------------------------|-----------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------|---------------------------------------------------|
| Si n h a et al 2 0 1 2                                                                             | Diff er e nt m o bili z 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 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 |                                                                                                                              |                                                                                                                                                                                                                                                                                                                                                     |                                                   |
| 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 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                                                                                                                            |                                                   |
| 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 .
untitled h1
1 3. 7 c rit

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 bili z 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.
untitled h1
1 4 r e f

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

```  
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 N e 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 Metaa 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.  
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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 V ersi o n 1. 4 , 2 1 A pril 2 0 2 1 6 8 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 dla 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.
untitled h1
1 5 a p p

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 bili z 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   |
|---------------|----------|--------------|
untitled h1
i ncl u si o

_____ 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  
_____ A bs ol ut e n e utr o p hil c o u nt > 1 5 0 0 x 1 0 / L a n d pl at el ets > 1 0 0, 0 0 0 x 1 0 / 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
untitled h1
e x cl u si

_____ 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:                          |           |  
| 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:                          |           |
untitled h1
1 5. 2 a p

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 e s 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 e sti 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