Comparative Effectiveness of Elranatamab (PF-06863135) in Clinical Study C1071003 Versus Standard of Care (SOC) in Real-World (RW) External Control Arms in Patients With Triple-Class Refractory (TCR) Multiple Myeloma (MM)
Conditions: Multiple Myeloma
Sponsor: Pfizer

P F -0 6 8 6 3 1 3 5 ( Elra nata ma b)
N O N -I N T E R V E N TI O N A L S T U D Y P R O T O C O L
C 1 0 7 1 0 3 1 Versi o n 1. 0, 0 1 Ma y 2 0 2 3
P FI Z E R C O N FI D E N TI A L 
C T 2 4 -WI -G L 0 2 -R F 0 2 4. 0 N o n -I nter ve nti o nal St u d y Pr ot oc ol Te m plate F or Sec o n dar y Data C ollecti o n St u d y 
0 1 -J u n-2 0 2 2 
Pa ge 1 of 7 6
N O N- I N T E R V E N TI O N A L ( NI) S T U D Y P R O T O C O L
St u d y Inf or m ati o n
Title
C o m parati ve Effecti ve ness o f E lra nata ma b
(P F- 0 6 8 6 3 1 3 5) i n Cli nical St u d y C 1 0 7 1 0 0 3 Vers us 
Sta n dar d o f Care ( S O C) i n Real -W orl d
( R W)
E xter nal C o ntr ol Ar m s in Patie nts w it h Tri ple-Class 
Refract o r y ( T C R) M ulti ple M y el o ma ( M M)
Pr ot oc ol n u m ber
C 1 0 7 1 0 3 1
Pr ot oc ol versi o n i de ntifier
1. 0
D ate
0 1 Ma y 2 0 2 3
Acti ve s u bst a nce
Elra nata ma b ( P F -0 6 8 6 3 1 3 5)
Rese arc h q uesti o n a n d o bjecti ves
Pri m ar y o bjecti ve s:

T o c o m pare P F S a m o n g T C R M M patie nts 
treate d wit h elra nata ma b i n St u d y C 1 0 7 1 0 0 3 
wit h a c o m para ble c o h ort of T C R M M 
patie nts recei vi n g S O C t hera py fr o m t he 
C O T A data base.

T o c o m pare P F S a m o n g T C R M M patie nts 
treate d wit h elra nata ma b i n St u d y C 1 0 7 1 0 0 3 
wit h a c o m para ble c o h ort of T C R M M 
patie nts recei vi n g S O C t hera py fr o m t he 
Flatir o n Healt h data base.
Sec o n d ar y o bjecti ve:

T o c o m pare O S i n T C R M M patie nts treate d 
wit h elra nata ma b i n St u d y C 1 0 7 1 0 0 3 wit h a 
c o m para ble c o h ort of T C R M M patie nts 
recei vi n g S O C t hera py fr o m t he C O T A 
data base .

T o c o m pare O S i n T C R M M
patie nts treate d 
wit h elra nata ma b i n St u d y  C 1 0 7 1 0 0 3 wit h a 
c o m para ble c o h ort of T C R M M patie nts 
0 9 0 1 7 7 e 1 9 d 6 a 1 0 1 2\ A p pr o v e d\ A p pr o v e d O n: 1 5- M a y- 2 0 2 3 0 2: 3 3 ( G M T)

P F -0 6 8 6 3 1 3 5 ( Elra nata ma b)
N O N -I N T E R V E N TI O N A L S T U D Y P R O T O C O L
C 1 0 7 1 0 3 1 Versi o n 1. 0, 0 1 Ma y 2 0 2 3
P FI Z E R C O N FI D E N TI A L 
C T 2 4 -WI -G L 0 2 -R F 0 2 4. 0 N o n -I nter ve nti o nal St u d y Pr ot oc ol Te m plate F or Sec o n dar y Data C ollecti o n St u d y 
0 1 -J u n-2 0 2 2 
Pa ge 2 of 7 6
recei vi n g S O C t hera py fr o m t he Flat ir o n 
Healt h data base.
E x pl or at or y o bjecti ve:

T o c o m pare t he c ha n ge of patie nt -re p orte d 
o utc o mes i n T C R M M patie nts treate d w it h 
elra nata ma b i n St u d y C 1 0 7 1 0 0 3 wit h a 
c o m para ble c o h ort of T C R M M patie nts 
recei vi n g S O C t hera py fr o m t he pr os pecti ve 
o bser vati o nal st u dies C 1 0 7 1 0 1 3 a n d 
C 1 0 7 1 0 1 4 .
A ut h or
, P h D
 
Pfizer , I nc
6 6 H u ds o n B o ule var d , Ne w Y or k, N Y 1 0 0 0 1
T his d oc u me nt c o ntai ns c o nfi de ntial i nf or mati o n bel o n gi n g t o Pfiz er. E xce pt as ot her wise a gree d t o i n writi n g, 
b y acce pti n g or re vie wi n g t his d oc u me nt, y o u a gree t o h ol d t his i nf or mati o n i n c o nfi de nce a n d n ot c o p y or 
discl ose it t o ot hers (e xce pt w here re q uire d b y a p plica ble la w) or use it f or u na ut h orize d p ur p oses. I n t he e ve nt 
of a n y act ual or s us pecte d breac h of t his o bli gati o n, Pfizer m ust be pr o m ptl y n o tifie d.
0 9 0 1 7 7 e 1 9 d 6 a 1 0 1 2\ A p pr o v e d\ A p pr o v e d O n: 1 5- M a y- 2 0 2 3 0 2: 3 3 ( G M T)
P P D
P P D
P P D

P F -0 6 8 6 3 1 3 5 ( Elra nata ma b)
N O N -I N T E R V E N TI O N A L S T U D Y P R O T O C O L
C 1 0 7 1 0 3 1 Versi o n 1. 0, 0 1 Ma y 2 0 2 3
P FI Z E R C O N FI D E N TI A L 
C T 2 4 -WI -G L 0 2 -R F 0 2 4. 0 N o n -I nter ve nti o nal St u d y Pr ot oc ol Te m plate F or Sec o n dar y Data C ollecti o n St u d y 
0 1 -J u n-2 0 2 2 
Pa ge 3 of 7 6
1. T A B L E O F C O N T E N T S
1. T A B L E O F C O N T E N T S .......................................................................................................3
2. LI S T O F A B B R E VI A T I O N S................................................................................................ 5
3. R E S P O N SI B L E P A R TI E S .................................................................................................... 8
4. A B S T R A C T ........................................................................................................................... 9
5. A M E N D M E N T S A N D U P D A T E S ..................................................................................... 1 0
6. MI L E S T O N E S ..................................................................................................................... 1 1
7. R A TI O N A L E A N D B A C K G R O U N D ................................................................................ 1 2
8. R E S E A R C H Q U E S TI O N A N D O BJ E C TI V E S ................................................................. 1 4
9. R E S E A R C H M E T H O D S .................................................................................................... 1 5
9. 1. St u d y  Desi g n........................................................................................................... 1 5
9. 2. Setti n g ...................................................................................................................... 1 8
9. 2. 1. St u d y  C 1 0 7 1 0 0 3 ......................................................................................... 1 8
9. 2. 2. Real -W orl d Data S o urces
........................................................................... 1 8
9. 2. 3. I ncl usi o n a n d E x cl usi o n Criteria f or E xter nal C o ntr ol Ar ms ..................... 2 0
9. 3. Varia bles .................................................................................................................. 2 4
9. 4. Data S o urces ............................................................................................................ 2 8
9. 5. St u d y  Size................................................................................................................ 2 9
9. 6. Data Ma na ge me nt ................................................................................................... 2 9
9. 7. Data A nal ys es .......................................................................................................... 3 0
9. 7. 1. Descri pti ve Statistics .................................................................................. 3 4
9. 7. 2. Mai n A nal ys es ............................................................................................ 3 5
9. 7. 3. Se nsiti vit y  A nal yses.................................................................................... 3 5
9. 7. 4. A d diti o nal A nal y s es....................................................................................3 6
9. 8. Q ualit y  C o ntr ol........................................................................................................ 3 8
9. 9. L i mitati o ns of t he Researc h Met h o ds...................................................................... 3 8
9. 1 0. Ot her As pects ........................................................................................................ 3 9
1 0. P R O T E C TI O N O F H U M A N S U BJ E C T S ........................................................................3 9
1 0. 1. Patie nt I nf or mati o n ................................................................................................ 3 9
1 0. 2. Patie nt C o nse nt ...................................................................................................... 3 9
1 0. 3. Patie nt Wit h dra wal ................................................................................................ 3 9
0 9 0 1 7 7 e 1 9 d 6 a 1 0 1 2\ A p pr o v e d\ A p pr o v e d O n: 1 5- M a y- 2 0 2 3 0 2: 3 3 ( G M T)

P F -0 6 8 6 3 1 3 5 ( Elra nata ma b)
N O N -I N T E R V E N TI O N A L S T U D Y P R O T O C O L
C 1 0 7 1 0 3 1 Versi o n 1. 0, 0 1 Ma y 2 0 2 3
P FI Z E R C O N FI D E N TI A L 
C T 2 4 -WI -G L 0 2 -R F 0 2 4. 0 N o n -I nter ve nti o nal St u d y Pr ot oc ol Te m plate F or Sec o n dar y Data C ollecti o n St u d y 
0 1 -J u n-2 0 2 2 
Pa ge 4 of 7 6
1 0. 4. I nstit uti o nal Re vie w B o ar d (I R B)/I n de pe n de nt Et hics C o m mittee (I E C) ............ 4 0
1 0. 5. Et hical C o n d uct of t he St u d y ................................................................................ 4 0
1 1. M A N A G E M E N T A N D R E P O R TI N G O F A D V E R S E E V E N T S/ A D V E R S E 
R E A C TI O N S ...................................................................................................................... 4 1
1 2. P L A N S F O R DI S S E M I NA TI N G A N D C O M M U NI C A TI N G S T U D Y R E S U L T S ........ 4 1
1 3. R E F E R E N C E S .................................................................................................................. 4 2
1 4. LI S T O F T A B L E S ............................................................................................................. 4 8
1 5. LI S T O F FI G U R E S ........................................................................................................... 4 9
A N N E X 1. LI S T O F S T A N D A L O N E D O C U M E N T S......................................................... 5 0
A N N E X 2. M A P PI N G O F E LI GI BI LI T Y C RI T E RI A B E T W E E N S T U D Y C 1 0 7 1 0 0 3 
A N D R W D S O U R C E S. ...................................................................................................... 5 1
A N N E X 3. LI S T O F S U B G R O U P S A N D C O M P RI SI N G T R E A T M E N T S ......................... 7 4
0 9 0 1 7 7 e 1 9 d 6 a 1 0 1 2\ A p pr o v e d\ A p pr o v e d O n: 1 5- M a y- 2 0 2 3 0 2: 3 3 ( G M T)

P F -0 6 8 6 3 1 3 5 ( Elra nata ma b)
N O N -I N T E R V E N TI O N A L S T U D Y P R O T O C O L
C 1 0 7 1 0 3 1 Versi o n 1. 0, 0 1 Ma y 2 0 2 3
P FI Z E R C O N FI D E N TI A L 
C T 2 4 -WI -G L 0 2 -R F 0 2 4. 0 N o n -I nter ve nti o nal St u d y Pr ot oc ol Te m plate F or Sec o n dar y Data C ollecti o n St u d y 
0 1 -J u n-2 0 2 2 
Pa ge 5 of 7 6
2. LI S T O F A B B R E VI A TI O N S
A b bre vi ati o n
Ter m
A D C
A nti b o d y -D r u g C o nj u gate
A E
A d verse E ve nt
A L T
A la ni ne A mi n otra nsferase
A MI
Ac ute M y ocar dial Infarcti o n
A nti -C D 3 8 m A b
A nti -C D 3 8 M o n ocl o nal A nti b o d y
A S T
A s partate A mi n otra nsferase
A T E
A vera ge Treat me nt Effect
A T T
A vera ge Treat me nt Effect A m o n gst Treate d
B C M A
B -Cell Mat urati o n A nti ge n
B M
B o ne Marr o w
C A R
C hi meric A nti ge n Rece pt or
C A T E
C o n diti o nal A vera ge T reat me nt E ffect
C CI
C harls o n C o m or bi dit y  In de x
CI
C o nfi de nce I nter val
C V D
Car di o vasc ular Disease
D O R
D urati o n of Res p o nse
E C O G
Easter n C o o perati ve O nc ol o g y  Gr o u p
e C R F
Electr o nic Case Re p ort F or m
E H R
Electr o nic Healt h Rec or d
E M A
E ur o pea n Me dici nes A ge nc y
E M D
E xtra me d ullar y  Disease
E O R T C Q L Q- C 3 0
E ur o pea n Or ga nizati o n f or Re searc h a n d Treat me nt of Ca ncer Q ualit y  
of L ife Q uesti o n naire Versi o n 3. 0
E O R T C Q L Q-
M Y 2 0
E ur o pea n Or ga nizati o n f o r Researc h a n d Treat me nt of Ca ncer M ulti ple 
M y el o ma Q uesti o n naire
E Q -5 D
E ur o p ea n Q ualit y of Li fe 5 Di me nsi o n 
E Q -V A S
E ur o Q o L – Vis ual A nal o g Scale
F C S
F ull y  C o n diti o nal S pecificati o n
F D A
F o o d a n d Dr u g A d mi nistrati o n
F L C
Free Li g ht C hai n
G B S
G uillai n -Barre S y n dr o me
G V H D
Graft Vers us H ost Disease
H B V
He patitis B Vir us
H C V
H e patitis C Vir us
H C P
Healt hcare Pr o vi der
HI V
H u ma n I m m u n o deficie nc y  Vir us
I Mi D
I m m u n o m o d ulat or y Dr u g
I M W G
I nter nati o nal Mye l o ma W or ki n g Gr o u p
I P T W
I n verse Pr o ba bilit y of Treat me nt Wei g hts
0 9 0 1 7 7 e 1 9 d 6 a 1 0 1 2\ A p pr o v e d\ A p pr o v e d O n: 1 5- M a y- 2 0 2 3 0 2: 3 3 ( G M T)

P F -0 6 8 6 3 1 3 5 ( Elra nata ma b)
N O N -I N T E R V E N TI O N A L S T U D Y P R O T O C O L
C 1 0 7 1 0 3 1 Versi o n 1. 0, 0 1 Ma y 2 0 2 3
P FI Z E R C O N FI D E N TI A L 
C T 2 4 -WI -G L 0 2 -R F 0 2 4. 0 N o n -I nter ve nti o nal St u d y Pr ot oc ol Te m plate F or Sec o n dar y Data C ollecti o n St u d y 
0 1 -J u n-2 0 2 2 
Pa ge 6 of 7 6
A b bre vi ati o n
Ter m
Kar M Ma -R W
C o m paris o n o f I deca bta ge ne Vicle ucel wit h Real- W orl d
O utc o mes i n 
Rela pse d an d Refract or y  M ulti ple M yel o ma
L V E F
Left Ve ntric ular Ejecti o n Fracti o n
L O T
Li ne of T hera p y
M A M M O T H
M o n ocl o nal A nti b o dies i n M ulti ple M y el o ma: O utc o mes after T hera p y 
Fail ure
M A R
Missi n g ness at Ra n d o m
MI
M ulti ple I m p utati o n
MI C E
M u lti ple Im p utati o n s b y C hai ne d E q uati o ns
M M
M ulti ple M y el o ma
M M R M
Mi xe d -E ffects M o del wit h R e peate d M eas ures
M -pr otei n
M o n ocl o nal Im m u n o gl o b uli n P r otei n
M U G A
M ulti gate d Ac q uisiti o n S ca n
NI  
N o n -I nter ve nti o nal
NI C E
Nati o nal I nstit ute f or Healt h a n d Care E xcelle nce
O R R
O bjecti ve Res p o nse Rate 
O S
O verall S ur vi val
P D
Pr o gressi ve Disease
P GI C
Patie nt Gl o bal I m pressi o n of C ha n ge
PI
Pr oteas o me I n hi bit or
P F S
Pr o gressi o n -Free S ur vi val
P O E M S
P ol y ne ur o pat h y , Or ga n o me gal y , E n d ocri n o pat h y, M o n ocl o nal
Ga m m o p at h y, a n d S ki n C ha n ges
P S
Pr o pe nsit y  Sc ores
P R O
Patie nt -Re p orte d O utc o mes
Q o L
Q ualit y  of Life
Q T
Ti me fr o m the be gi n ni n g of t he Q R S C o m ple x t o t he E n d of t he T 
Wa ve
Q Tc F
C orrecte d Q T ( Fri dericia Met h o d)
R C T
Ra n d o mize d C o ntr olle d Trial
R W
Real -W orl d
R W D
Real -W orl d Data
R W E
Real -W orl d E vi d e nce
R R M M
Rela pse d or Refract or y  M M
S A P
Statistical a nal y sis pla n
S A R S -C o v 2
Se vere Ac ute Res pirat or y S y n dr o me - C or o na vir us 2
S L A M F 7
Si g nali n g L y m p h oc y tic Acti vati o n M olec ule Fa mil y Me m ber 7
S M D
Sta n dar di ze d Mean Differe nce
S O C
Sta n dar d of Care
S P E P
S er u m P r otei n E lectr o p h oresis
T C R
Tr i ple-Class Refract or y
0 9 0 1 7 7 e 1 9 d 6 a 1 0 1 2\ A p pr o v e d\ A p pr o v e d O n: 1 5- M a y- 2 0 2 3 0 2: 3 3 ( G M T)

P F -0 6 8 6 3 1 3 5 ( Elra nata ma b)
N O N -I N T E R V E N TI O N A L S T U D Y P R O T O C O L
C 1 0 7 1 0 3 1 Versi o n 1. 0, 0 1 Ma y 2 0 2 3
P FI Z E R C O N FI D E N TI A L 
C T 2 4 -WI -G L 0 2 -R F 0 2 4. 0 N o n -I nter ve nti o nal St u d y Pr ot oc ol Te m plate F or Sec o n dar y Data C ollecti o n St u d y 
0 1 -J u n-2 0 2 2 
Pa ge 7 of 7 6
A b bre vi ati o n
Ter m
U L N
U p per Li mit of N or mal
U P E P
Uri ne Pr otei n Electr o p h oresis 
U S
U nite d States
V A S
Vis ual A nal o g Scale
0 9 0 1 7 7 e 1 9 d 6 a 1 0 1 2\ A p pr o v e d\ A p pr o v e d O n: 1 5- M a y- 2 0 2 3 0 2: 3 3 ( G M T)

P F -0 6 8 6 3 1 3 5 ( Elra nata ma b)
N O N -I N T E R V E N TI O N A L S T U D Y P R O T O C O L
C 1 0 7 1 0 3 1 Versi o n 1. 0, 0 1 Ma y 2 0 2 3
P FI Z E R C O N FI D E N TI A L 
C T 2 4 -WI -G L 0 2 -R F 0 2 4. 0 N o n -I nter ve nti o nal St u d y Pr ot oc ol Te m plate F or Sec o n dar y Data C ollecti o n St u d y 
0 1 -J u n-2 0 2 2 
Pa ge 8 of 7 6
3. R E S P O N SI B L E P A R TI E S
P ri nci p al I n vesti g a t or(s) of t he Pr ot oc ol
N a m e, de gree(s)
J o b Title
Affili ati o n
A d dress
, P h D
Pfizer , I nc
6 6 H u ds o n B o ule var d , 
Ne w  Y or k, N Y 1 0 0 0 1
, M P H
Pfizer , I nc
6 6 H u ds o n B o ule var d, 
Ne w  Y or k, N Y 1 0 0 0 1
, M D
Pfizer, I nc
Walt o n Oa ks, D or ki n g 
R oa d, Ta d w ort h, U K 
S urre y K T 2 0 7 N S
, P har m D, M B A
Pfiz er, I nc
6 6 H u ds o n B o ule var d, 
Ne w  Y or k, N Y 1 0 0 0 1
, P h D
Pfizer, I nc
5 0 0 Arc ola R oa d, 
C olle ge ville, P A 1 9 4 2 6
, M P H
Pfizer, I nc
6 6 H u ds o n B o ule var d, 
Ne w  Y or k, N Y 1 0 0 0 1
, P h D
S T A T L O G, 
I nc
4 6 7 3 Clara Br o ussea u, 
Q ué bec, Q C G 1 Y 3 M 9
, M P H
S T A T L O G, 
I nc
4 6 7 3 Clara Br o ussea u, 
Q ué bec, Q C G 1 Y 3 M 9
0 9 0 1 7 7 e 1 9 d 6 a 1 0 1 2\ A p pr o v e d\ A p pr o v e d O n: 1 5- M a y- 2 0 2 3 0 2: 3 3 ( G M T)
P P D
P P D
P P D
P P D
P P D
P P D
P P D
P P D
P P D

P F -0 6 8 6 3 1 3 5 ( Elra nata ma b)
N O N -I N T E R V E N TI O N A L S T U D Y P R O T O C O L
C 1 0 7 1 0 3 1 Versi o n 1. 0, 0 1 Ma y 2 0 2 3
P FI Z E R C O N FI D E N TI A L 
C T 2 4 -WI -G L 0 2 -R F 0 2 4. 0 N o n -I nter ve nti o nal St u d y Pr ot oc ol Te m plate F or Sec o n dar y Data C ollecti o n St u d y 
0 1 -J u n-2 0 2 2 
Pa ge 9 of 7 6
4. A B S T R A C T
N ot a p plica ble .
0 9 0 1 7 7 e 1 9 d 6 a 1 0 1 2\ A p pr o v e d\ A p pr o v e d O n: 1 5- M a y- 2 0 2 3 0 2: 3 3 ( G M T)

P F -0 6 8 6 3 1 3 5 ( Elra nata ma b)
N O N -I N T E R V E N TI O N A L S T U D Y P R O T O C O L
C 1 0 7 1 0 3 1 Versi o n 1. 0, 0 1 Ma y 2 0 2 3
P FI Z E R C O N FI D E N TI A L 
C T 2 4 -WI -G L 0 2 -R F 0 2 4. 0 N o n -I nter ve nti o nal St u d y Pr ot oc ol Te m plate F or Sec o n dar y Data C ollecti o n St u d y 
0 1 -J u n-2 0 2 2 
Pa ge 1 0 of 7 6
5. A M E N D M E N T S A N D U P D A T E S
N o ne.
0 9 0 1 7 7 e 1 9 d 6 a 1 0 1 2\ A p pr o v e d\ A p pr o v e d O n: 1 5- M a y- 2 0 2 3 0 2: 3 3 ( G M T)

P F -0 6 8 6 3 1 3 5 ( Elra nata ma b)
N O N -I N T E R V E N TI O N A L S T U D Y P R O T O C O L
C 1 0 7 1 0 3 1 Versi o n 1. 0, 0 1 Ma y 2 0 2 3
P FI Z E R C O N FI D E N TI A L 
C T 2 4 -WI -G L 0 2 -R F 0 2 4. 0 N o n -I nter ve nti o nal St u d y Pr ot oc ol Te m plate F or Sec o n dar y Data C ollecti o n St u d y 
0 1 -J u n-2 0 2 2 
Pa ge 1 1 of 7 6
6. MI L E S T O N E S
M ilest o ne
Pl a n ne d d ate
Start of data c ollect i o n 
1 5 Ma y 2 0 2 3
E n d of data c ollecti o n
2 2 Ma y 2 0 2 3
Fi nal st u d y re p ort
2 6 J ul y 2 0 2 3
0 9 0 1 7 7 e 1 9 d 6 a 1 0 1 2\ A p pr o v e d\ A p pr o v e d O n: 1 5- M a y- 2 0 2 3 0 2: 3 3 ( G M T)

P F -0 6 8 6 3 1 3 5 ( Elra nata ma b)
N O N -I N T E R V E N TI O N A L S T U D Y P R O T O C O L
C 1 0 7 1 0 3 1 Versi o n 1. 0, 0 1 Ma y 2 0 2 3
P FI Z E R C O N FI D E N TI A L 
C T 2 4 -WI -G L 0 2 -R F 0 2 4. 0 N o n -I nter ve nti o nal St u d y Pr ot oc ol Te m plate F or Sec o n dar y Data C ollecti o n St u d y 
0 1 -J u n-2 0 2 2 
Pa ge 1 2 of 7 6
7. R A T I O N A L E A N D B A C K G R O U N D
M M is a rare a n d pr o gressi ve t y pe of ca ncer t hat is c haracterize d b y  t he ra pi d acc u m ulati o n 
of m o n ocl o nal plas ma cells i n t he b o n e marr ow ( B M). T his res ults i n t he pr o d ucti o n of 
m o n ocl o nal i m m u n o gl o b uli n pr otei n ( M -pr otei n) a n d e ve nt u all y lea ds t o e n d -or ga n da ma ge. 
T he gl o bal i nci de nce of M M is risi n g a n d is esti mate d at 1 6 0, 0 0 0 i n 2 0 1 8, wit h 
1 0 6, 0 0 0 deat hs attri b ute d t o M M a n n uall y . I n t he past 3 deca des, fr o m 1 9 9 0 t o 2 0 1 6, t he 
gl o bal i nci de nce of M M has i ncrease d b y 1 2 6 %, wit h t he hi g hest i nci de nce rates re p orte d i n 
A ustralia, Wester n E ur o pe, a n d t he U nite d States ( U S). 1
O ver t he ye ars, a d va nce me nts h a ve bee n ma de i n t he disc o ver y  a n d utilizati o n of M M 
t hera pies, wit h t he c urre nt S O C i ncl u di n g pr oteas o me i n hi bit ors ( PIs) ( b ortez o mi b, 
carfilz o mi b, i xaz o mi b), i m m u n o m o d ulat or y  dr u gs (I Mi Ds) (t hali d o mi de, le nali d o mi de, 
p o mali d o mi de), a n d a nti -C D 3 8 m o n ocl o n al a nti b o dies (a nti-C D 3 8 m A b : darat u m u ma b a n d 
isat u xi ma b).2
Des pite t hese treat me nt a d va nces, a maj or pr o p orti o n of M M patie nts will fail all 3 classes of 
t he c urre nt S O C re gi me ns (i ncl u di n g PI, I MI Ds, a nd a nti -C D 3 8) a n d bec o me tri ple class 
refract or y ( T C R). T he treat me nt of T C R M M prese nts a t hera pe utic c halle n ge beca use of t he 
i n here nt cl o nal heter o ge neit y a n d ge netic i nsta bilit y of M M t u m or cells, i nfl ue nci n g t he 
e ve nt ual de vel o p me nt of t hera pe ut ic resista nce.3 T heref ore, as T C R patie nts pr o gress a n d are 
e x p ose d t o a n i ncreasi n g n u m ber of t hera pies, t he d urati o n of res p o nse ( D O R) decreases 
al o n g wit h P F S a n d O S u ntil t he disease is ulti matel y  fatal.4 -6
Bela nta ma b maf o d oti n -bl mf was t he first B -cell mat urati o n a nti ge n ( B C M A) t hera p y 
a p pr o ve d b y t he F D A i n A u g ust 2 0 2 0 ,7 a n d i n N o ve m ber 20 2 2, t he U S mar keti n g 
a ut h orizati o n was wit h dra w n .8 Si nce 2 0 2 0, t he F D A has a p pr o ve d 3 ot her B C M A t hera pies , 
i ncl u di n g 2 C A R T -cell t hera pies (i deca bta ge ne vicle ucel a n d ciltaca bta ge ne a ut ole ucel) a n d 
m os t rece ntl y t he first-i n-class B C M A bis pecific a nti b o d y  t hera py t eclista ma b -c q y v .9 -1 1
Pfizer has de vel o pe d elra nata ma b ( P F -0 6 8 6 3 1 3 5), a n i n vesti gati o nal B -cell mat urat i o n 
a nti ge n ( B C M A) C D 3-tar gete d bis pecific a nti b o d y, f or t he treat me nt of patie nts wit h T C R 
M M. T h e preli mi nar y res ults of t he P hase 1 st u d y ( N C T 0 3 2 6 9 1 3 6) de m o nstrate d ma na gea ble 
safety of elra nata ma b a n d a res p o nse rate of 6 9 % (ie, 9/ 1 3 patie nts) a t t he rec o m me nde d d ose 
of 1 0 0 0 µ g/ k g .1 2 A d diti o nall y , elra nata ma b has bee n gra nte d Fast Trac k Desi g nati o n b y t he 
U S F o o d a n d Dr u g A d mi nistrati o n ( F D A) a n d E ur o pea n Me dici nes A ge nc y ( E M A) t o ai d t he 
ra pi d de vel o pme nt a n d re vie w of t his n o vel a ge nt a n d t here b y a d dress t he u n met me dical 
nee ds of T C R M M patie nts .1 3 Pfizer is c urre ntl y c o n d ucti n g a P hase 2 st u d y t o e val uate t he 
cli nical be nefit of elra nata ma b i n T C R M M pati e nts ( S t u d y C 10 7 1 0 0 3). T he u p date d res ults 
fr o m t he Ma g netis M M-3 st u d y  s h o we d a n o bjecti ve res p o nse rate ( O R R ) of 6 1. 0 % after a 
me dia n f oll o w -u p of 1 0. 4 m o nt hs .1 4 Base d o n t hese res ults, i n N o ve m ber 2 0 2 2, elra na ta ma b 
recei ve d F D A Brea kt hr o u g h T hera p y  Desi g nati o n f or t he treat me nt of patie nts wit h rela pse d 
or refrac t or y M M ( R R M M).1 5 C o nsi deri n g t he et hical a n d practical c halle n ges ass ociate d 
wit h c o n d ucti n g a ra n d o mize d cli nical trial wit hi n a partic ularl y diffic ult-t o-treat p o p ulati o n 
of M M patie nts wit h u n met nee ds a n d n o clear si n gle S O C, a si n gle ar m desi g n was a d o pte d 
f or t he P hase 2 st u dy. 1 6 T h us, t here is a nee d t o ge ne rate e vi de nce o n t he cli nical o utc o mes of 
0 9 0 1 7 7 e 1 9 d 6 a 1 0 1 2\ A p pr o v e d\ A p pr o v e d O n: 1 5- M a y- 2 0 2 3 0 2: 3 3 ( G M T)

P F -0 6 8 6 3 1 3 5 ( Elra nata ma b)
N O N -I N T E R V E N TI O N A L S T U D Y P R O T O C O L
C 1 0 7 1 0 3 1 Versi o n 1. 0, 0 1 Ma y 2 0 2 3
P FI Z E R C O N FI D E N TI A L 
C T 2 4 -WI -G L 0 2 -R F 0 2 4. 0 N o n -I nter ve nti o nal St u d y Pr ot oc ol Te m plate F or Sec o n dar y Data C ollecti o n St u d y 
0 1 -J u n-2 0 2 2 
Pa ge 1 3 of 7 6
T C R patie nts w h o recei ve S O C t o hel p c o nte xt ualize t he res ults fr o m St u d y C 1 0 7 1 0 0 3. T his 
has bee n acc o m plis he d b y  creati n g a n e xter nal c o ntr ol ar m usi n g real- worl d d ata ( R W D) t o 
s u p p ort t he o n g oi n g e val uati o n of elra nata ma b.
Ot h er st u dies use d t he R W data t o a nal yze cli nical o utc o mes i n patie nts wit h 
rela pse d/refract or y M M ( R R M M). I n t he M A M M O T H st u d y,1 7 2 4 9 M M patie nts starti n g a 
ne w treat me nt after bec o mi n g refract or y to a n a nti-C D 3 8 m A b (darat u m u ma b or isat u xi ma b ) 
were i de ntifie d fr o m 1 4 U S aca de mic i nstit uti o ns. I n t hese patie nts, t he O R R f or t he first 
re gi me n after bec o mi n g refract or y t o a nti- C D 3 8 m A b was 3 1 %, a n d me dia n P F S a n d O S 
fr o m t he ti me w he n patie nts beca me refract or y t o an a nti-C D 3 8 m A b were 3. 4 m o nt hs 
( 9 5 % c o nfi de nce i nter val, CI: 2. 8- 4. 0) a n d 9. 3 m o nt hs ( 9 5 % CI : 8. 1- 1 0. 6) , res pecti vely. 1 7
T he Kar M Ma -R W st u d y  was a gl o bal NI, retr os pecti ve st u d y t hat assesse d treat me nt patter n s 
a n d o utc o mes i n R W R R M M patie nts tre ate d wit h c urre ntl y a vaila ble t hera pies, a n d w h ose 
c haracteristics were si milar t o t he Kar M Ma st u d y, a P hase 2 si n gle -ar m st u d y of 
i deca bta ge ne vicle ucel i n hea vil y pretreate d R R M M patie nts w h o were tri ple-class e x p osed 
a n d refract or y t o t he last re gi me n.1 8 A p pr o x i matel y 4 3 % of t he Kar M Ma-R W p o p ulati o n 
were T C R. T he O R R of t he 1 9 0 eli gi ble R R M M patie nts i n t he R W setti n g was 3 2 % 
( 9 5 % CI : 2 4. 4 - 4 2. 3), a n d 2 9. 4 % ( 9 5 % CI: 2 0. 2-4 2. 8) i n t he matc he d eli gi ble R R M M s u bset. 
I n t he eli gi ble R R M M c o h orts, t he me dia n P F S was 3. 5 m o nt hs, a n d t he me dia n O S was 
1 4. 7 m o nt hs.
Re g ulat or y  A ge ncies, i ncl u di n g F D A a n d E M A, ha ve rec o g nize d t he utilit y of e xter nal 
c o ntr ol ar ms deri ved fr o m R W D i n decisi o n -ma ki n g, a n d ha ve esta blis he d g ui da nce o n 
c o n diti o ns t hat s h o ul d be satisfie d bef ore a n e xter nal c o ntr ol a p pr oac h is dee me d a p pr o priate, 
as well as ke y met h o d ol o gical c o nsi derati o ns t o be a d dresse d.1 9 -2 1 Acc or di n g t o t hese 
g ui deli nes, a n e xter nal c o ntr ol a p pr oa c h is c o nsi dere d a p pr o priate u n der certai n c o n diti o ns, 
i ncl u di n g (a) t here is a hi g h u n met nee d (e g, seri o us rare diseases), ( b) t here is a well -
d oc u me nt e d, hi g hl y pre dicta ble disease c o urse t hat ca n be o bjecti vel y meas ure d a n d verifie d, 
a n d (c) t here is an e x pecte d dr u g effect t hat is lar ge, self -e vi de nt, a n d te m p orall y cl osely 
ass ociate d wit h t he i nter ve nti o n. 2 2 ,2 3 A rece ntl y p u blis he d re vie w of t he Nati o nal I nstit ute f or 
Healt h a n d Care E xcel le nce ( NIC E) a p praisals s h o we d t hat 2 2 tec h n ol o gies ( 1 2 i n a n 
o nc ol o g y  i n dicati o n) were a p praise d b y NI C E fr o m 20 0 0 t o 2 0 1 6 ba se d o n n o n-ra n d o mize d 
c o ntr olle d trial ( R C T) data; 2 7 % of t h ose use d o bser vati o nal data t o esta blis h c o m parati ve 
effecti ve ness ;2 4 o ver half of t he NI C E a p plicati o ns were ma de i n t he last 2 y ears alo ne.
T o c o nte x t ualize t he cli nical pr ofile of elra nata ma b, a retr os pecti ve c o h ort st u d y (St u d y  
C 1 0 7 1 0 2 4; N C T 0 5 5 6 5 3 9 1), was c o n d ucte d t o c o m pare t he efficac y  o utc o mes o bser ve d i n 
t he partici pa nts of St u d y  C 1 0 7 1 0 0 3 ( wit h at least 9 m o nt hs of f oll o w- u p ) a n d R W patie nts 
selecte d fr o m 2 U S -base d o nc ol o g y  electr o nic healt h rec or d ( E H R) data bases, Flatir o n 
Healt h a n d C O T A. T he res ults of St u d y  C 1 0 7 1 0 2 4 s h o we d t hat a m o n g T C R M M patie nts, 
t h ose treate d wit h elra nata ma b ha d si g nifica ntly hi g her O R R t ha n t h ose treat e d wit h SO C. 
T h u s, i n t he IP T W a nal ysis c o m pari n g partici pa nts fr o m St u d y  C 1 0 7 1 0 0 3 wit h R W patie nts 
fr o m CO T A or Flatir o n Healt h, t he esti mate d relati ve ris k ( R R) was 2. 2 2 ( 9 5 % CI : 1. 6 9- 2. 9 0, 
p <. 0 0 0 1) a n d 1. 7 9 ( 9 5 % CI : 1. 0 1- 3. 1 5, p =. 0 4 4 7), res pecti vely. Di ffere nces i n t he ti me t o 
0 9 0 1 7 7 e 1 9 d 6 a 1 0 1 2\ A p pr o v e d\ A p pr o v e d O n: 1 5- M a y- 2 0 2 3 0 2: 3 3 ( G M T)

P F -0 6 8 6 3 1 3 5 ( Elra nata ma b)
N O N -I N T E R V E N TI O N A L S T U D Y P R O T O C O L
C 1 0 7 1 0 3 1 Versi o n 1. 0, 0 1 Ma y 2 0 2 3
P FI Z E R C O N FI D E N TI A L 
C T 2 4 -WI -G L 0 2 -R F 0 2 4. 0 N o n -I nter ve nti o nal St u d y Pr ot oc ol Te m plate F or Sec o n dar y Data C ollecti o n St u d y 
0 1 -J u n-2 0 2 2 
Pa ge 1 4 of 7 6
res po nse ( T T R) bet wee n t h ose treate d wit h elra nata ma b a n d S O C were n ot c o nsiste nt l y 
o bser ve d, h o we ver, D O R was si g nifica ntl y i m pr o ve d f or elra nata ma b c o m pare d t o S O C 
(IP T W hazar d rati o [ H R] 0. 1 1, 9 5 % CI : 0. 0 6 - 0. 2 2, p <. 0 0 0 1 a n d 0. 2 1, 9 5 % C I: 0. 1 0 - 0. 4 5, 
p <. 0 0 0 1 w he n c o m pari n g partici pa nts fr o m St u d y C 1 0 7 1 0 0 3 wit h R W patie nts fr o m C O T A 
or Flatir o n Healt h, res pecti vel y ). T he c urre nt st u d y is t he c o nti n uati o n of t he C 1 0 7 1 0 2 4 wit h 
a l o n ger a vaila ble f oll o w-u p of t he St u d y C 1 0 7 1 0 0 3 par tici pa nts a n d a lar ger set of
o utc o mes . 
T his st u d y  ai ms t o c o m pare t he P F S a n d O S i n partici pa nts of St u d y C 1 0 7 1 0 0 3 treate d wit h 
elra nata ma b vers us R W T C R M M patie nts treate d wit h S O C t hera pies. T o re d uce t he 
p ote ntial f or bias, e xter nal c o ntr ol ar ms will be c o nst r ucte d b y  selecti n g fit-f or-p ur p ose R W D 
s o urces (ie, relia ble a n d rele va nt),2 5 a n d a p pr o priate c o m parati ve effecti ve ness met h o ds,
i ncl u di n g statistical tec h ni q ues (e g, I P T W) 2 6 -2 8 A d diti o nall y , a n e x pl orat or y a nal ys is of 
patie nt re p orte d o utc o mes ( P R Os ) will be c o n d ucte d.
8. R E S E A R C H Q U E S TI O N A N D O B J E C TI V E S
T hi s st u d y ai ms t o assess t he c o m parati ve effecti ve ness of elranata ma b v ers us S O C treat me nt 
i n T C R M M patie nts usi n g e xter nal c o ntr ol ar ms f or t he o pe n-la bel, m ultice nter, n o n-
ra n d o mize d si n gle-ar m P hase 2 St u d y  C 1 0 7 1 0 0 3 .
Pri m ar y o bjecti ve s:

T o c o m pare P F S a m o n g T C R M M patie nts treate d wit h elra nata ma b i n St u d y  
C 1 0 7 1 0 0 3 wit h a c o m par a ble c o h ort of T C R M M patie nts recei vi n g S O C t hera py 
fr o m t he C O T A data base.

T o c o m pare P F S a m o n g T C R M M patie nts treate d wit h elra nata ma b i n St u d y  
C 1 0 7 1 0 0 3 wit h a c o m para ble c o h ort of T C R M M patie nts recei vi n g S O C t hera py 
fr o m t he Flatiro n Healt h d ata base.
Sec o n d ar y o bjecti v es:

T o c o m pare O S i n T C R M M patie nts treate d wit h elra nata ma b i n St u dy C 1 0 7 1 0 0 3 
wit h a c o m para ble c o h ort of T C R M M patie nts recei vi n g S O C t hera py fr o m t he 
C O T A data base.

T o c o m pare O S i n T C R M M patie nts treate d wit h elra nata ma b in St u d y C 1 0 7 1 0 0 3 
wit h a c o m para ble c o h ort of T C R M M patie nts recei vi n g S O C t hera py fr o m t he 
Flat ir o n Healt h data base.
E x pl or at or y o bjecti ve :

T o c o m pare t he c ha n ge of patie nt -re p orte d o utc o mes i n T C R M M patie nts treate d 
wit h elra nata ma b i n St u d y  C 1 0 7 1 0 0 3 wit h a c om para ble c o h ort of T C R M M patie nts 
recei vi n g S O C t hera py fr o m t he pr os pecti ve o bser vati o nal st u dies C 1 0 7 1 0 1 3 a n d 
C 1 0 7 1 0 1 4 .
0 9 0 1 7 7 e 1 9 d 6 a 1 0 1 2\ A p pr o v e d\ A p pr o v e d O n: 1 5- M a y- 2 0 2 3 0 2: 3 3 ( G M T)

P F -0 6 8 6 3 1 3 5 ( Elra nata ma b)
N O N -I N T E R V E N TI O N A L S T U D Y P R O T O C O L
C 1 0 7 1 0 3 1 Versi o n 1. 0, 0 1 Ma y 2 0 2 3
P FI Z E R C O N FI D E N TI A L 
C T 2 4 -WI -G L 0 2 -R F 0 2 4. 0 N o n -I nter ve nti o nal St u d y Pr ot oc ol Te m plate F or Sec o n dar y Data C ollecti o n St u d y 
0 1 -J u n-2 0 2 2 
Pa ge 1 5 of 7 6
9. R E S E A R C H M E T H O D S
9. 1. St u d y D esi g n
T his retr os pecti ve c o h ort st u d y  will use patie nt -le vel data fr o m si n gle ar m cli nical St u d y  
C 1 0 7 1 0 0 3 a n d e xter nal c o ntr ol ar ms i de ntifie d pre vi o usl y fr o m R W D s o urces. T he relati n g 
st u d y pr ot ocols are i n A n ne x 1 . T o ma xi mize c o m para bilit y , t he eli gi bilit y criteria f or t he
partici pa nts fr o m St u d y C 1 0 7 1 0 0 3 ha ve bee n a pplie d t o patie nt s fr o m t he R W D so urces (see 
Secti o n 9. 2. 3 f or m ore detail).
M M patie nts eli gi ble f or selecti o n i nt o e xter nal c o ntr ol ar ms are t h ose patie nts w h o are 
refract or y t o at least 1 PI, 1 IMi D, a n d 1 anti -C D 3 8 m A b a n d ha ve starte d at least 1 ne w 
treat me nt si nce t he d oc u me ntati o n of T C R stat us. Refract or y  is defi ne d as ha vi n g disease 
pr o gressi o n, acc or di n g t o I nter nati o nal M yel o ma W or ki n g Gr o u p (I M W G) criteria or cli nical 
assess me nt, w hile o n t her a p y or wit hi n 6 0 da y s of t he last d ose i n a n y line of t hera p y ( L O T), 
re gar dless of res p o nse. I n Flatir o n Healt h a n d C O T A, if a s u bse q ue nt L O T was i nitiate d a n d 
a pr o gressi o n was o bser ve d after at least 3 0 da y s of t he L O T start, t he patie nt is c o nsi dere d 
refract ory t o t he s u bse q ue nt L O T e ve n if t he pr o gr essi o n occ urre d wit hi n 6 0 da y s after t he 
last d ose of t he prece di n g LO T. Details o n t he o perati o nal defi niti o ns of I M W G criteria f or 
pr o gressi o n use d i n Flatir o n Healt h , C O T A, C 1 0 7 1 0 1 3, a n d C 1 0 7 1 0 1 4 a n d a si d e-b y -side 
c o m paris o n wit h t he criteria use d i n S t u d y C 1 0 7 1 0 0 3 are pr o vi de d i n t he relate d st u d y 
pr ot oc ols i n A n ne x 1 .
I n t he R W setti n g, n o si n gle S O C c urre ntly e xists f or T C R M M patie nts, a n d c o m bi nati o ns of 
treat me nts are fre q ue ntl y use d i n lie u of m o n ot hera p y. 2 9. F or si m plicit y, i n t his pr ot oc ol, t he 
ter m “ S O C” refers t o all sta n dar d treat me nt o pti o ns a vaila ble f or T C R M M patie nts. 
Selecti o n of T C R M M patie nts i nitiat i n g a ne w treat me nt i n t he e xter nal c o ntr ol ar m e na bles 
c o m para bilit y of patie nts at a si milar sta ge i n disease pro gressi o n f oll o wi n g T C R 
d oc u me ntati o n.
T he date of i nitiati o n of t he first re gi me n after T C R M M eli gi bilit y  will be defi ne d as t he 
i n de x date. F or t he c o m p arati ve a nal ys is of P F S a n d O S , onl y  patie nts wit h a n i n de x date 
occ urri n g bet wee n 1 6 N o ve m ber 2 0 1 5, a n d 30 J u ne 2 0 2 2 will be selecte d (t he first a nti-
C D 3 8 m A b t hera p y was a p pr o ve d by t he F D A o n 1 6 N o ve m ber 2 0 1 5) fr o m C O T A a n d 
Flatir o n Healt h .
 
 
T he st u d y  peri o d will be c o m prise d of t he baseli ne peri o d (ti me prece di n g t he 
i n de x date) a n d t he obser vat i o nal peri o d (ti me f oll o wi n g t he i n de x date). T he baseli ne peri o d 
(scree ni n g peri o d) f or t he partici pa nts of C 1 0 7 1 0 0 3 is 2 8 da ys bef ore t he i nitiati o n of 
elra nata ma b. D ue t o t he o bser vati o nal nat ure of data , t he baseli ne peri o d f or R W p atie nts f or
the c oll ecti o n of patie nts’ hist or y  a n d la b meas ure me nts e xte n ds fr o m t he ti me of t he first 
M M dia g n os is t o t he i n de x date. T he s pecific ti me wi n d o ws f or eac h assess me nt are detaile d 
i n t he res pecti ve R W pr ot oc ols (A n n e x 1) a n d i n t he list of c o variates pr o vi de d i n 
Secti o n 9. 3 . T he o bser vati o nal peri o d will s pa n fr o m t he i n de x date t o t he earliest of deat h, or 
t he latest a vaila ble patie nt rec or d, w hic he ver c o mes first. T he pr i mar y cli nical o utc o me of 
i nterest will be P F S. T he sec o n dar y  cli nical o utc o me of i nterest will be O S . It is e x pecte d t hat 
0 9 0 1 7 7 e 1 9 d 6 a 1 0 1 2\ A p pr o v e d\ A p pr o v e d O n: 1 5- M a y- 2 0 2 3 0 2: 3 3 ( G M T)
C CI

P F -0 6 8 6 3 1 3 5 ( Elra nata ma b)
N O N -I N T E R V E N TI O N A L S T U D Y P R O T O C O L
C 1 0 7 1 0 3 1 Versi o n 1. 0, 0 1 Ma y 2 0 2 3
P FI Z E R C O N FI D E N TI A L 
C T 2 4 -WI -G L 0 2 -R F 0 2 4. 0 N o n -I nter ve nti o nal St u d y Pr ot oc ol Te m plate F or Sec o n dar y Data C ollecti o n St u d y 
0 1 -J u n-2 0 2 2 
Pa ge 1 6 of 7 6
t he me dia n O S will n ot be reac he d i n St u d y C 1 0 7 1 0 0 beca use of i ns ufficie nt f oll o w- u p d ata 
use d i n t his a nal y sis. For t he e x pl orat or y a nal ys is, P R Os meas ure d usi n g self-a d mi nistere d 
q uesti o n naires will be assesse d .
I n t he mai n a nalys is c o m pari n g P F S a n d O S, differe nces i n baseli ne c haracteristics 
( de m o gra p hic c haracteristics, treat me nt hist or y, a n d disease-relate d c haracteristics ) bet wee n 
patie nts i n St u d y  C 1 0 7 1 0 0 3 a n d eac h e xter nal c o ntr ol ar m will be bala nce d usi n g I P T W (see 
Secti o n 9. 7. 2 ). Se nsiti vit y  a n al yses of P F S a n d O S will be c o n d ucte d t o e val uate t he effect of 
alter nati ve met h o ds t o a d dress c o nf o u n di n g a n d selecti o n bias, i ncl u di n g t he d o u bl y r o b ust 
met h o d, a n d u si n g alter nati ve i ncl usi o n/e xcl usi o n criteria. A q ua ntitati ve bias a nal ys is 
( n ullificati o n a nalys is) will be perf or me d t o e val uate t he r o b ust ness of results i n t he prese nce 
of p ote ntial t hreats t o i nter nal vali dit y  (see Secti o n 9. 7. 3 ).
A sc he matic fi g ure of St u d y  C 1 0 7 1 0 0 3 a n d e xter nal c o ntr ol ar ms is pr o vi de d i n Fi g ure 1 a n d 
Fi g ure 2 , res pecti vel y.
0 9 0 1 7 7 e 1 9 d 6 a 1 0 1 2\ A p pr o v e d\ A p pr o v e d O n: 1 5- M a y- 2 0 2 3 0 2: 3 3 ( G M T)

P F -0 6 8 6 3 1 3 5 ( Elra nata ma b)
N O N -I N T E R V E N TI O N A L S T U D Y P R O T O C O L
C 1 0 7 1 0 3 1 Versi o n 1. 0, 0 1 Ma y 2 0 2 3
P FI Z E R C O N FI D E N TI A L 
C T 2 4 -WI -G L 0 2 -R F 0 2 4. 0 N o n -I nter ve nti o nal St u d y Pr ot oc ol Te m plate F or Sec o n dar y Data C ollecti o n St u d y 
0 1 -J u n-2 0 2 2 
Pa ge 1 7 of 7 6
Fi g ure 1. B aseli ne a n d O bser v ati o n P eri o ds i n St u d y C 1 0 7 1 0 0 3
Fi g ure 2 . B aseli ne a n d O bser v ati o n P eri o ds i n E xter n al C o nt r ol A r ms
0 9 0 1 7 7 e 1 9 d 6 a 1 0 1 2\ A p pr o v e d\ A p pr o v e d O n: 1 5- M a y- 2 0 2 3 0 2: 3 3 ( G M T)

P F -0 6 8 6 3 1 3 5 ( Elra nata ma b)
N O N -I N T E R V E N TI O N A L S T U D Y P R O T O C O L
C 1 0 7 1 0 3 1 Versi o n 1. 0, 0 1 Ma y 2 0 2 3
P FI Z E R C O N FI D E N TI A L 
C T 2 4 -WI -G L 0 2 -R F 0 2 4. 0 N o n -I nter ve nti o nal St u d y Pr ot oc ol Te m plate F or Sec o n dar y Data C ollecti o n St u d y 
0 1 -J u n-2 0 2 2 
Pa ge 1 8 of 7 6
9. 2. Setti n g
9. 2. 1. St u d y C 1 0 7 1 0 0 3 
St u d y  C 1 0 7 1 0 0 3 is a n o pe n-la bel, m ulti-ce nter, n o n-ra n d o mize d P hase 2 st u d y of 
elra nata ma b (P F -0 6 8 6 3 1 3 5 ) m o n ot hera p y , w hic h was i nitiate d i n Fe br uar y 2 0 2 1.3 0 T o 
deter mi ne t he effects of pri or B C M A - directe d t hera p y o n t he res p o nse t o elra nata ma b
m o n ot hera p y, St u d y C 1 0 7 1 0 0 3 e nr oll e d 2 i n de pe n de nt a n d parallel c o h orts, 1 wit h patie nts 
w h o are naï ve t o B C M A -directe d t hera pies ( C o h ort A; 1 2 3 patie nts) a n d t he ot her wit h 
patie nts pre vi o usl y e x pose d t o B C M A - directe d t hera p y ( C o h ort B; 6 4 patie nts ). Beca use fe w 
patie nts were e x p ose d t o B C M A- directe d t hera p y  i n our R W data base s o urces, t he f oc us of 
t he c o m paris o ns will be o n C o h ort A i n St u d y  C 1 0 7 1 0 0 3, t h o u g h a d diti o nal a nalys es will 
i ncl u de b ot h C o h orts A a n d B.
9. 2. 2. Re al -W orl d D at a S o urces
T he c o h orts of R W T C R M M patie nts f or t he e xter nal c o ntr ol ar m will be i de ntifie d fr o m 
E H R data bases Flatir o n Healt h a n d C O T A a n d p r os pecti ve o bser vati o nal st u dies C 1 0 7 1 0 1 3 
a n d C 1 0 7 1 0 1 4 . T hese data bases ha ve bee n selecte d accor di n g t o dat a a vaila bilit y a n d as 
fit-f or-p ur p ose f or f ulfilli n g st u d y  o bjecti ves ( 2 3). T he eli gi ble peri o d f or t he i n de x dates of 
patie nts fr o m eac h R W data base h a s bee n selecte d t o ali g n as cl osel y  as p ossi ble t o eac h 
ot her a n d St u d y  C 1 0 7 1 0 0 3 .
Fl atir o n He a lt h
T he F latir o n Healt h data base is a l o n git u di nal, de m o gra p hically, a n d ge o gra p hicall y di verse 
data base deri ve d f r o m E H R data. Flatir o n i ncl u des data fr o m o ver 2 8 0 c o m m u nit y ca ncer 
ce nters a n d aca de mic i nstit uti o ns ( ~ 8 0 0 sites of care) re prese nti n g m o re tha n 2. 4 m illi o n U S 
acti ve ca ncer patie nts a vaila ble f or a nal ys is. T he s o urce p o p ulati o n is t he o verall p o p ulati o n 
re p orte d i n t he E H R a n d i ncl u des patie nts ma na ge d i n at least 1 of t he U S o nc ol o g y ce nters 
ta ki n g part i n t he Flatir o n Healt h net w or k fr o m 0 1 Ja n uar y 20 1 1 o n war ds.
Flatir o n Healt h data bases c o nsist of l o n git u di nal data o n M M patie nts, i ncl u di n g n or malize d 
data fr o m str uct ure d E H R, e n ha nce d data o n patie nts’ c haracteristics a bstracte d fr o m 
u nstr uct ure d E H R, a n d deri ve d data t hat is create d ba se d o n Flati ro n-s pecific b usi ness 
r ules.3 1
Str uct ure d data i ncl u de i nf or m ati o n o n patie nts’ de m o gra p hics, visit dates, dia g n oses, vitals, 
me dicati o ns, t he Easte r n C o o perati ve O nc ol o g y Gr o u p ( E C O G) perf or ma nce stat us, a n d 
la borat or y  tests. U nstr uct ure d data c o ntai n a d diti o nal i nf or mati o n o n c o m or bi dities, 
bi o mar ker re p orts, a n d details of tra ns pla nts. Flatir o n Healt h als o pr o vi des deri ve d data s uc h 
as pr o gressi o n a n d res p o nse varia bles w here al g orit h ms are de vel o p e d foll o wi n g a da pte d 
I M W G criteria t o i de ntif y t hese e ve nts t hr o u g h o ut t he c o urse of a patie nt’s j o ur ne y.
Details o n t he i de ntificati o n of t he e xter nal c o ntr ol ar m usi n g Flatir o n Healt h data ca n be 
f o u n d in t he relate d pr ot oc ol as D oc u me nt 0 0 1 i n A n ne x 1 .
0 9 0 1 7 7 e 1 9 d 6 a 1 0 1 2\ A p pr o v e d\ A p pr o v e d O n: 1 5- M a y- 2 0 2 3 0 2: 3 3 ( G M T)

P F -0 6 8 6 3 1 3 5 ( Elra nata ma b)
N O N -I N T E R V E N TI O N A L S T U D Y P R O T O C O L
C 1 0 7 1 0 3 1 Versi o n 1. 0, 0 1 Ma y 2 0 2 3
P FI Z E R C O N FI D E N TI A L 
C T 2 4 -WI -G L 0 2 -R F 0 2 4. 0 N o n -I nter ve nti o nal St u d y Pr ot oc ol Te m plate F or Sec o n dar y Data C ollecti o n St u d y 
0 1 -J u n-2 0 2 2 
Pa ge 1 9 of 7 6
C O T A
C O T A mai ntai ns a m ulti disci pli nar y data c urati o n a p pr oac h. T he C O T A data base is a 
l o n git u di nal data base deri ve d fr o m t he E H R of healt hcare pr o vi der ( H C P) sites i ncl u di n g 
aca de mic i nstit uti o ns, c o m m u nit y ce nters, a n d h os pital s yste ms re prese nti n g 5 0 0, 0 0 0 patie nts 
fr o m o ver 2 0 0 sites of care i n t he U S. Data ele me nts are sta n dar dize d acr oss s o u rces t o create 
a si n gle, str uct ure d dataset t o c o ver t he f ull l o n git u di nal hist or y  of a patie nt’s cli nical care. 
F or eac h patie nt, t he da tasets c o ntain i nf or mati o n fr o m t he ti me of i nitial M M dia g n osis t o 
t he m ost rece nt d oc u me ntati o n i n t he H E R. 3 2
As wit h Flatir o n Healt h, C O T A c ollects i nf or mati o n fr o m str uct ure d a n d u nstr uct ure d data 
s o urces. Deri ve d data base d o n a da pte d I M W G criteria are als o pr o vi de d (e g, disease 
pr o gressi o n a n d treat me nt res p o nse). C O T A dif fers fr o m Flatir o n Healt h i n t he pr o visi o n of 
s pecific data bases t hat are de dicate d t o re p orti n g fact ors rele va nt t o t he e x perie nce of a 
pati e nt wit h M M a n d i ncl u des datasets c o ntai ni n g a bstracte d i nf or mati o n o n b o ne lesi o ns, 
plas mac y t o mas, ra diati o n t herap y , a n d a d verse e ve nts ( A Es).
Details o n t he i de ntificati o n of t he e xter nal c o ntr ol ar m usi n g C O T A data ca n be f o u n d i n t he 
relate d pr ot oc ol as D oc u me nt 0 0 2 i n A n ne x 1 . 
0 9 0 1 7 7 e 1 9 d 6 a 1 0 1 2\ A p pr o v e d\ A p pr o v e d O n: 1 5- M a y- 2 0 2 3 0 2: 3 3 ( G M T)
C CI

P F -0 6 8 6 3 1 3 5 ( Elra nata ma b)
N O N -I N T E R V E N TI O N A L S T U D Y P R O T O C O L
C 1 0 7 1 0 3 1 Versi o n 1. 0, 0 1 Ma y 2 0 2 3
P FI Z E R C O N FI D E N TI A L 
C T 2 4 -WI -G L 0 2 -R F 0 2 4. 0 N o n -I nter ve nti o nal St u d y Pr ot oc ol Te m plate F or Sec o n dar y Data C ollecti o n St u d y 
0 1 -J u n-2 0 2 2 
Pa ge 2 0 of 7 6
9. 2. 3. I ncl usi o n a n d E xcl usi o n C riteri a f or E xter n al C o ntr ol A r ms
C o m para bilit y  of patie nts bet wee n St u d y C 1 0 7 1 0 0 3 a n d t he e xter nal co ntr ol ar ms is o ne of 
t he ke y c o nsi derati o ns t o mi ni mize bias. Care will be ta ke n t o e ns ure t hat patie nts i de ntifie d 
fr o m eac h R W T C R M M c o h ort are si milar t o t he St u d y  C 1 0 7 1 0 0 3 patie nts b y satisf yi n g 
pre -s pecifie d i ncl usi o n a n d e xcl usi o n criteria. I ncl usio n a n d e xcl usi o n criteria fr o m St u d y 
C 1 0 7 1 0 0 3 are pr o vi de d i n Ta ble 1 a n d will be m o difie d f or eac h c o h ort of R W T C R patie nts 
base d o n data a vaila bilit y. O perati o nal defi niti o ns of i ncl usi o n a n d e xcl usi o n criteria f or eac h 
R W D s o u rce are s u m marize d i n A n ne x 2 .
0 9 0 1 7 7 e 1 9 d 6 a 1 0 1 2\ A p pr o v e d\ A p pr o v e d O n: 1 5- M a y- 2 0 2 3 0 2: 3 3 ( G M T)
C CI

P F -0 6 8 6 3 1 3 5 ( Elra nata ma b)
N O N -I N T E R V E N TI O N A L S T U D Y P R O T O C O L
C 1 0 7 1 0 3 1 Versi o n 1. 0, 0 1 Ma y 2 0 2 3
P FI Z E R C O N FI D E N TI A L 
C T 2 4 -WI -G L 0 2 -R F 0 2 4. 0 N o n -I nter ve nti o nal St u d y Pr ot oc ol Te m plate F or Sec o n dar y Data C ollecti o n St u d y 
0 1 -J u n-2 0 2 2 
Pa ge 2 1 of 7 6
D ue t o t he missi n g ness prese nt wit hi n R W D s o urces, t here is a n i nhere nt tra de off bet wee n 
a nal yz a ble sa m ple size a n d t he de gree of c o m para bilit y of patie nts fr o m St u d y C 1 07 1 0 0 3 
wit h t he e xter nal c o ntr ol ar m sa m ple. I n ot her w or ds, t he m ore cl osel y t he 
i ncl usi o n/e xcl usi o n criteria f or t he e xter nal c o ntr ol ar ms are ali g ne d t o St u d y C 1 0 7 1 0 0 3, t he 
s maller t he a vaila ble sa m ple size. T heref ore, f or t he c o m parati ve a nal ys is of P F S a n d O S , 2 
sa m ples of e xter nal c o ntr ol ar ms (t he critical eli gi bilit y criteria sa m ple a n d t he e x pa n de d 
eli gi bilit y criteria sa mple) will be selecte d fro m eac h c o h ort of R W T C R M M patie nts
i de ntifie d i n C O T A or Flatir o n Healt h (see Ta ble 1 ). B ot h sa m ples will i nc or p orate c ore
i ncl usi o n/e xcl usi o n criteria fr o m St u d y C 1 0 7 0 1 0 0 3 w hic h re prese nt ke y cli nical varia bles t o 
defi ne t he c o h ort. F urt her m ore, t he e x pa n de d eli gi bilit y criteria sa m ple will i nc or p orate 
a d diti o nal i ncl usi o n/e xcl usi o n criteria fr o m St u d y C 1 0 7 1 0 0 3 (e g, la b val ues) at t he e x pe nse 
of a s maller sa m ple size.
F or t he c o m parati ve a nal y ses of P FS a n d O S , the critical eli gi bilit y  criteria sa m ple will 
c o nstit ute t he mai n a nal ysis set. Se nsiti vit y  a nalys es will be c o n d ucte d usin g t he e x pa n de d 
eli gi bilit y criteria t o assess t he p ote ntial i nfl ue nce of t he selecti o n pr ocess o n t he o bser ve d 
effects (ie, ma i n a nal yses will be re peate d usi n g t his sa m ple). T he n u m ber of patie nts 
i ncl u de d/e xcl u de d at eac h ste p of t he st u d y will be re p ort ed.
Ta ble 1 pr o vi des a ta b ular view s u m marizi n g t he i ncl usi o n a n d e xcl usi o n criteria of St u d y 
C 1 0 7 1 0 0 3 w hic h are a vaila ble a n d will be a p plie d f or t he selecti on of critical a n d e x pa n de d 
eli gi bilit y criteria sa m ples of R W patie nts (si g nifie d wit h a c hec k mar k s y m b ol w here t he 
critical a n d/ or e x pa n de d selecti o n criteria defi niti o ns are a p plica ble).
0 9 0 1 7 7 e 1 9 d 6 a 1 0 1 2\ A p pr o v e d\ A p pr o v e d O n: 1 5- M a y- 2 0 2 3 0 2: 3 3 ( G M T)
C CI

P F -0 6 8 6 3 1 3 5 ( Elra nata ma b)
N O N -I N T E R V E N TI O N A L S T U D Y P R O T O C O L
C 1 0 7 1 0 3 1 Versi o n 1. 0, 0 1 Ma y 2 0 2 3
P FI Z E R C O N FI D E N TI A L 
C T 2 4 -WI -G L 0 2 -R F 0 2 4. 0 N o n -I nter ve nti o nal St u d y Pr ot oc ol Te m plate F or Sec o n dar y Data C ollecti o n St u d y 
0 1 -J u n-2 0 2 2 
Pa ge 2 2 of 7 6
T a ble 1. I ncl usi o n a n d E xcl usi o n C riteri a f or St u d y C 1 0 7 1 0 0 3 a n d R W D at a S o urces.
P atie nt criteri a per St u d y C 1 0 7 1 0 0 3
I mple me nt ati o n i n 
Fl atir o n He alt h
I mple me nt ati o n i n 
C O T A
I mple me nt ati o n 
i n C 1 0 7 1 0 1 3 a n d
C 1 0 7 1 0 1 4
Critic al
eli gi bilit y 
criteri a
E x p a n de d
eli gi bilit y 
criteri a
Critic al
eli gi bilit y 
criteri a
E x p a n de d
eli gi bilit y 
criteri a
Critic al
eli gi bilit y 
criteri a
I ncl usi o n Criteri a
Male or fe male patie nts a ge d ≥1 8 y ears
ü 
ü 
ü 
ü
ü
Willi n g a n d a ble
t o c o m pl y wit h all sc he d ule d visits, 
treat me nt pla ns, la b orat or y tests, lifest yle 
c o nsi derati o ns, a n d ot her st u d y pr oce d ures
N/ A  
N/ A  
N/ A  
N/ A
N/ A
Pri or dia g n osis of M M as defi ne d acc or di n g t o 
I M W G criteria*
ü 
ü 
ü 
ü
ü
Meas ura ble disease, base d o n I M W G criteria as 
defi ne d b y at least 1 of t he f oll o wi n g
a) Ser u m M -pr otei n ≥0. 5 g/ d L b y S P E P
b) Uri nar y M -pr otei n e xcreti o n ≥2 0 0 m g/ 2 4 h o urs 
b y Uri ne Pr otei n Electr o p h oresis ( U P E P)
c) Ser u m i m m u n o gl o b uli n Free Li g ht C hai n 
( F L C)≥1 0 m g/ d L ( ≥1 0 0 m g/ L) a n d a b n or mal ser u m 
i m m u n o gl o b uli n ka p pa t o la m b da F L C rati o ( < 0. 2 6 
or > 1. 6 5)
ü 
ü 
ü 
ü
ü
Patie nts are T C R defi ne d as bei n g refract or y t o all 
3 of t he f oll o wi n g:
a) Refract or y t o at lea st 1 I Mi D
b) Refract or y t o a t least 1 PI
c) Refract or y t o at least 1 a nti -C D 3 8 m A b
Rela pse d/refract or y t o last a nti -M M re gi me n
ü 
ü 
ü 
ü
ü
I nitiate d at least 1 a nti-M M s yste mic t hera p y after 
bec o mi n g T C R eli gi ble. T he first s yste mic treat me nt 
i nitiati on after bec o mi n g T C R eli gi ble m us t n ot 
c o m prise a n y st u d y or i n vesti gati o nal a ge nt. * *
ü 
ü 
ü 
ü
ü
E C O G perf or ma nce sta t us ≤2
ü 
ü 
ü 
ü
ü
A de q uate he patic f u ncti o n c haracterize d b y all of t he 
f oll o wi n g:
a) T otal bilir u bi n ≤2 x U p per Li mit of N or mal 
( U L N) (≤3 x U L N if d oc u me nte d Gil bert’s 
s yn dr o me)
b) as partate a mi n otra nsferase ( A S T) ≤2. 5 x U L N
c) ala ni ne a mi n otra nsferase ( A L T) ≤2. 5 x U L N
X 
ü  
X 
ü
X
A de q uate re nal f u ncti o n, defi ne d b y a n 
esti mate d creati ni ne cleara nce ≥3 0 m L/ mi n 
(acc or di n g t o t he C oc kcr oft Ga ult f or m ula, b y 
2 4 h o ur uri ne c ollecti o n f or creati ni ne cleara nce, or 
acc or di n g t o t he l ocal i nstit uti o nal sta n dar d met h od)
X 
ü  
X 
ü
X
A de q uate B M f u ncti o n c haracterize d b y all of t he 
f oll o wi n g
a) A bs ol ute ne utr o p hil c o u nt ≥1. 0 × 1 0 9/ L ( use of 
gra n ul oc yte -c ol o n y sti m ulati n g fact ors is 
per mitte d if c o m plete d at least 7 da ys bef ore 
pla n ne d start of d osi n g)
b) Platelets ≥2 5 × 1 0 9/ L (tra nsf us i o n s u p p ort is 
per mitte d if c o m plete d at least 7 da ys bef ore t he 
pla n ne d start of d osi n g)
X 
ü  
X 
ü
X
0 9 0 1 7 7 e 1 9 d 6 a 1 0 1 2\ A p pr o v e d\ A p pr o v e d O n: 1 5- M a y- 2 0 2 3 0 2: 3 3 ( G M T)

P F -0 6 8 6 3 1 3 5 ( Elra nata ma b)
N O N -I N T E R V E N TI O N A L S T U D Y P R O T O C O L
C 1 0 7 1 0 3 1 Versi o n 1. 0, 0 1 Ma y 2 0 2 3
P FI Z E R C O N FI D E N TI A L 
C T 2 4 -WI -G L 0 2 -R F 0 2 4. 0 N o n -I nter ve nti o nal St u d y Pr ot oc ol Te m plate F or Sec o n dar y Data C ollecti o n St u d y 
0 1 -J u n-2 0 2 2 
Pa ge 2 3 of 7 6
P atie nt criteri a per St u d y C 1 0 7 1 0 0 3
I mple me nt ati o n i n 
Fl atir o n He alt h
I mple me nt ati o n i n 
C O T A
I mple me nt ati o n 
i n C 1 0 7 1 0 1 3 a n d
C 1 0 7 1 0 1 4
Critic al
eli gi bilit y 
criteri a
E x p a n de d
eli gi bilit y 
criteri a
Critic al
eli gi bilit y 
criteri a
E x p a n de d
eli gi bilit y 
criteri a
Critic al
eli gi bilit y 
criteri a
c) He m o gl o bi n ≥8 g/ d L (tra nsf usi o n s u p p ort is 
per mitte d if c o m plete d at least 7 da ys bef ore t he 
pla n ne d start of d osi n g)
Left ve ntric ular eje cti o n fracti o n ≥4 0 % as 
deter mi ne d b y a m ulti gate d ac q uisiti o n sca n 
( M U G A) or ec h ocar di o gra m
X  
X  
X  
X
X
Res ol ve d ac ute effects of a n y pri or t hera p y t o 
baseli ne se verit y or C o m m o n Ter mi n ol o g y Criteria 
f or A E Gra de ≤1
X  
X  
X  
X
X
E xcl usi o n Criteri a
Acti v e plas ma cell le u ke mia
ü 
ü 
ü 
ü
ü
A m yl oi d osis
ü 
ü 
ü 
ü
ü
Pre vi o us treat me nt wit h a n a nti -B C M A bis pecific 
a nti b o d y.
X  
X  
X  
X
X
Pre vi o us a d mi nistrati o n wit h a n i n vesti gati o nal dr u g 
wit hi n 3 0 da ys ( or as deter mi ne d b y t he l ocal 
req uire me nt) or 5 half -li ves prece di n g t he first d ose 
of st u d y i nter ve nti o n use d i n t his st u d y ( w hic he ver is 
l o n ger).
ü 
ü 
ü 
ü
ü
S m ol deri n g M M
ü 
ü 
ü 
ü
ü
Ste m cell tra ns pla nt wit hi n 1 2 wee ks bef ore 
e nr ol me nt or acti ve Graft Vers us H ost Disease 
( G V H D).
ü 
ü 
ü 
ü
ü
A n y ot her acti ve mali g na n c y  wit hi n 3 years bef ore 
e nr ol me nt, e xce pt f or a de q uatel y treate d basal cell or 
s q ua m o us cell s ki n ca ncer, or carci n o ma i n sit u.
ü 
ü 
ü 
ü
ü
P O E M S s y n dr o me
X  
X  
X  
X
ü
I m paire d car di o vasc ular f u ncti o n or cli nicall y 
si g nifica nt car di o vasc ular diseases, defi n e d base d o n 
t he hist or y of a n y of t he f oll o wi n g c o n diti o ns wit hi n 
6 m o nt hs bef ore e nr ol me nt:
a) Ac ute m y ocar dial i nfarcti o n or ac ute c or o nar y 
s yn dr o mes (e g, u nsta ble a n gi na, c or o nar y arter y 
b y pass graft, c or o nar y a n gi o plast y or ste nti n g, 
s y m pt o matic pericar dial eff usi o n)
b) Cli nicall y si g nifica nt car diac arr h yt h mias 
(e g, u nc o ntr olle d atrial fi brillati o n or u nc o ntr olle d 
par o x ys mal s u pra ve ntric ular tac h ycar dia)
c) T hr o m b oe m b olic or cere br o vasc ular e ve nts 
(e g, tra nsie nt isc he mic attac k, cere br o vasc ular 
acci de n t, dee p vei n t hr o m b osis, or p ul m o nar y 
e mb olis m)
d) Pr ol o n ge d Ti me fr o m t he be gi n ni n g of t he Q R S 
C o m ple x t o t he E n d of t he T Wa ve ( Q T) s y n dr o me 
( or tri plicate a vera ge C orrecte d Q T ( Fri dericia 
Met h o d) ( Q Tc F) > 4 7 0 mse c).
X 
ü  
X 
ü
X
Acti ve He patitis B Vir us ( H B V), He patitis C Vir us 
( H C V), Se vere Ac ute Res pirat or y S y n dr o me-
C or o na vir us 2 ( S A R S -C o V 2), k n o w n H u ma n 
ü 
ü 
ü 
ü
ü
0 9 0 1 7 7 e 1 9 d 6 a 1 0 1 2\ A p pr o v e d\ A p pr o v e d O n: 1 5- M a y- 2 0 2 3 0 2: 3 3 ( G M T)

P F -0 6 8 6 3 1 3 5 ( Elra nata ma b)
N O N -I N T E R V E N TI O N A L S T U D Y P R O T O C O L
C 1 0 7 1 0 3 1 Versi o n 1. 0, 0 1 Ma y 2 0 2 3
P FI Z E R C O N FI D E N TI A L 
C T 2 4 -WI -G L 0 2 -R F 0 2 4. 0 N o n -I nter ve nti o nal St u d y Pr ot oc ol Te m plate F or Sec o n dar y Data C ollecti o n St u d y 
0 1 -J u n-2 0 2 2 
Pa ge 2 4 of 7 6
P atie nt criteri a per St u d y C 1 0 7 1 0 0 3
I mple me nt ati o n i n 
Fl atir o n He alt h
I mple me nt ati o n i n 
C O T A
I mple me nt ati o n 
i n C 1 0 7 1 0 1 3 a n d
C 1 0 7 1 0 1 4
Critic al
eli gi bilit y 
criteri a
E x p a n de d
eli gi bilit y 
criteri a
Critic al
eli gi bilit y 
criteri a
E x p a n de d
eli gi bilit y 
criteri a
Critic al
eli gi bilit y 
criteri a
I m m u n o deficie nc y Vir us ( HI V), or a n y acti ve, 
u nc o ntr olle d bacterial, f u n gal, or viral i nfecti o n. 
Acti ve i nfecti o ns m ust be res ol ve d at least 1 4 da ys 
bef ore e nr ol me nt.
Ot her s ur gical (i ncl u di n g maj or s ur ger y wit hi n 
1 4 da ys bef ore e nr ol me nt), me dical, or ps yc hiatric 
c o n diti o ns i ncl u di n g rece nt ( wit hi n t he past year) or 
acti ve s uici dal i deati o n/ be ha vi or or la b orat or y 
ab n or malit y t hat ma y i ncrease t he ris k of st u d y 
partici pati o n or, i n t he i n vesti gat or’s j u d g me nt, ma ke 
t he patie nt i na p pr o priate f or t he st u d y.
X  
X  
X  
X
X
O n g oi n g Gra de ≥2 peri p heral se ns or y or m ot or 
ne ur o pat h y.
X 
ü  
X  
X
X
Hist or y of a n y gra de peri p heral se ns or y or m ot or 
ne ur o pat h y wit h pri or B C M A -directe d t hera p y 
( C o h ort B).
X 
ü  
X 
ü
X
Hist or y of G uillai n - Barre S y n dr o me ( G B S) or G B S 
varia nts, or hist or y of a n y Gra de ≥3 peri p heral m ot or 
ne ur o pat h y
X 
ü  
X  
X
X
I n vesti gat or site staff or Pfizer e m pl o yees directl y 
i n v ol ve d i n t he c o n d uct of t he st u d y, site staff 
ot her wise s u per vise d b y t he i n vesti gat or, a n d t heir 
res pecti ve fa mil y me m bers.
X  
X  
X  
X
X
K n o w n or s us pecte d h y perse nsiti vit y t o t he st u d y 
i nter ve nti o n or a n y of its e xci pie nts.
X  
X  
X  
X
X
Li ve atte n uate d vacci ne m ust n ot be a d mi nist ere d 
wit hi n 4 wee ks of t he first d ose of t he st u d y 
i nter ve nti o n.
X  
X  
X  
X
X
A b bre vi ati o ns : A S T =as partate a mi n otra nsferase; A L T =ala ni ne a mi n otra nsferase; B C M A = B-cell mat urati o n a nti ge n; 
B M = b o ne marr o w; E C O G = Easter n C o o perati ve O nc ol o g y Gr o u p; H C V = he patitis C vir us; F L C =free li g ht c hai n; G B S = 
G uillai n -Barre s y n dr o me; G V H D = graft vers us h ost disease; H B V = he patitis B vir us; HI V = h u ma n i m m u n o deficie nc y vir us; 
I mi D =i m m u n o m o d ulat or y dr u g; I M W G =I nter nati o nal M yel o ma W or ki n g Gr o u p; M M = m ulti ple m yel o ma; PI = pr oteas o me 
i n hi bit or; P O E M S = p ol y ne ur o pat h y, or ga n o me gal y, e n d ocri n o pat h y, m o n ocl o nal ga m m o pat h y, a n d s ki n c ha n ges; 
Q Tc F =c orrecte d Q T ( Fri dericia met h o d); S A R S -C o V 2 =se vere ac ute res pirat or y s y n dr o me c or o na vir us 2; S P E P =ser u m 
pr otei n electr o p h oresis; T C R =tri ple -class refract or y; U L N = u p per li mit of n or mal; U P E P = ur i ne pr otei n electr o p h oresis.
N ote:
* Acc or di n g t o Raj k u mar S V, Di m o p o ul os M A, Pal u m b o A, et al. I nter nati o nal M yel o ma W or ki n g Gr o u p u p date d criteria f or 
t he dia g n osis of m ulti ple m yel o ma. La ncet O nc ol. 2 0 1 4; 1 5( 1 2):e 5 3 8 -e 5 4 8. d oi: 1 0. 1 0 1 6/ S 1 4 7 0 - 2 0 4 5( 1 4) 7 0 4 4 2- 5.
* * T his criteri o n is n ot a m o n g t he i ncl usi o n/e xcl usi o n criteria of St u d y C 1 0 7 1 0 0 0 3 a n d is a p plie d o nl y t o R W patie nts.
Le ge n d : ü : t his criteri o n ca n be a p plie d usi n g t his R W dat aset;
X: t his criteri o n ca n n ot be a p plie d/assesse d d ue t o a lac k of i nf or mati o n i n t his R W dataset.
9. 3. V ari a bles
E x p os ure defi niti o n
I n eac h c o m paris o n, patie nts will be classifie d i nt o 1 of t he 2 treat me nt gr o u ps acc or di n g t o 
t he t hera py recei ve d after T C R eli gi bilit y, as t h ose treate d wit h elra nata ma b a n d t h ose treate d 
wit h a S O C re gi me n (a n y sta n dar d treat me nt o pti o n a vaila ble f or R W T C R M M patie nts).
0 9 0 1 7 7 e 1 9 d 6 a 1 0 1 2\ A p pr o v e d\ A p pr o v e d O n: 1 5- M a y- 2 0 2 3 0 2: 3 3 ( G M T)

P F -0 6 8 6 3 1 3 5 ( Elra nata ma b)
N O N -I N T E R V E N TI O N A L S T U D Y P R O T O C O L
C 1 0 7 1 0 3 1 Versi o n 1. 0, 0 1 Ma y 2 0 2 3
P FI Z E R C O N FI D E N TI A L 
C T 2 4 -WI -G L 0 2 -R F 0 2 4. 0 N o n -I nter ve nti o nal St u d y Pr ot oc ol Te m plate F or Sec o n dar y Data C ollecti o n St u d y 
0 1 -J u n-2 0 2 2 
Pa ge 2 5 of 7 6
St u d y o utc o mes
C o nsiste nc y  bet ween o utc o me defi niti o ns acr oss data s o urces is a n ot her ke y  c o nsi derati o n t o
mi ni mize bias. C o m par ati ve effecti ve ness e n d p oi nts P F S a n d O S will be e val uate d base d o n 
a vaila bilit y wit hi n eac h R W D s o urce. Defi niti o ns of t he o utc o mes will be ali g ne d, w here 
p ossi ble, wit h St u d y  C 1 0 7 1 0 0 3 (Ta ble 2 ).
T a ble 2. Defi niti o ns of C o m p ar ati ve E ffecti ve ness O utc o mes i n St u d y C 1 0 7 1 0 0 3, 
Fl atir o n He alt h, a n d C O T A
O utc o me
St u d y C 1 0 7 1 0 0 3
Fl atir o n He alt h
C O T A
1 Pr o gr essi o n
-free
s ur vi val 
( P F S)
Ti me fr o m t he date of 
t he first d ose u ntil 
c o nfir me d P D per 
I M W G criteria or 
deat h d ue t o a n y ca use, 
w hic he ver occ urs first.
T i me fr o m i nitiati o n of 
t he first li ne after T C R t o 
eit her t he date of 
pr o gressi o n * or deat h 
d ue t o a n y ca use, 
w hic he ver occ urs first .
T i me fr o m i nitiati o n of 
t he first li ne after T C R t o 
eit her t he date of 
pr o gressi o n * or deat h 
d ue t o a n y ca use, 
w hic he ver occ urs first.
2 O verall 
s ur vi val 
( O S)
Ti me fr o m t he date of 
t he first d ose u ntil 
deat h d ue t o a n y ca use
Ti me fr o m i nitiati o n of 
t he first li ne after T C R 
u ntil t he date of deat h 
d ue t o a n y ca use.
Ti me fr o m i nitiati o n of 
t he first li ne after T C R 
u ntil t he date of deat h 
d ue t o a n y ca use.
N ote: *
C O T A uses a t hir d-part y o bit uar y data s o urce t o ca pt ure m ortali t y data. C O T A defi nes pr o gres si o n base d o n 
I M W G criteria. C O T A defi nes a pr o gressi o n as a n increase of ≥2 5 % fr o m t he l o west res p o nse val ue i n a n y o ne 
or m ore of t he f oll o wi n g criteria: S P E P w it h a n a bs ol ute i ncrease > 0. 5 g/ d L ; 2 4 -h o ur U P E P wit h a n a bs ol ute 
i ncrease > 2 0 0 m g/ 2 4 h ; i n patie nts wit h o ut meas ura ble ser u m a n d uri ne M-pr otei n, t he a bs olute i ncrease of
> 1 0 m g/ d L i n t he differe nce bet wee n i n v ol ve d a n d u ni n v ol ve d F L C le vels ; or a n a bs ol ute b o ne marr o w  plas ma 
cell perce nta ge > 1 0 %.
Flatir o n Healt h’s m ortalit y varia ble is create d t hr o u g h a n a mal ga mati o n of str uct ure d data el e me nts, 
u nstr uct ure d d oc u me nts, a n d li n ki n g t o e xter nal m ortalit y s o urces a n d t he S ocial Sec urit y Deat h I n de x ( S S DI). 
Flatir o n Healt h defi nes pr o gressi o n base d o n I M W G criteria. Flatir o n Healt h defi nes a pr o gressi o n as a n 
increase of ≥2 5 % fr o m baseli ne/ na d ir val ue i n a n y o ne or m ore of t he f oll o wi n g: a n a bs ol ute i ncrease i n ser u m 
M- pr otei n b y S P E P b y ≥0. 5 g/ d L ; ser u m M -pr otei n ≥1 g/ d L if t he l o west M c o m p o ne nt was ≥5 g/ d L ; a n 
a bs ol ute i ncrease i n u ri ne M-pr otei n b y U P E P b y ≥2 0 0 m g/ 2 4 h ; i n patie nts wit h o ut meas ura ble ser u m a n d 
uri ne M -pr otei n le vels , a n a bs ol ute i ncrease i n t he differe nce bet wee n i n v ol ve d a n d u ni n v ol ve d F L C le vels of
> 1 0 m g/ d L .
A b bre viati o ns: I M W G =I nter nati o nal M yel o ma W or ki n g Gr o u p; P D = pr o gressi ve disease; T C R =tri ple -class 
refract or y.
0 9 0 1 7 7 e 1 9 d 6 a 1 0 1 2\ A p pr o v e d\ A p pr o v e d O n: 1 5- M a y- 2 0 2 3 0 2: 3 3 ( G M T)
C CI

P F -0 6 8 6 3 1 3 5 ( Elra nata ma b)
N O N -I N T E R V E N TI O N A L S T U D Y P R O T O C O L
C 1 0 7 1 0 3 1 Versi o n 1. 0, 0 1 Ma y 2 0 2 3
P FI Z E R C O N FI D E N TI A L 
C T 2 4 -WI -G L 0 2 -R F 0 2 4. 0 N o n -I nter ve nti o nal St u d y Pr ot oc ol Te m plate F or Sec o n dar y Data C ollecti o n St u d y 
0 1 -J u n-2 0 2 2 
Pa ge 2 6 of 7 6
Ot her v ari a b les
E ns uri n g t hat baseli ne fact ors relate d t o treat me nt assi g n me nt a n d o utc o mes are ca pt ure d is 
of ut m os t i m p orta nce f or t he vali dit y of e xter nal c o ntr ol ar ms. T he a vaila bilit y of t hese 
varia bles i n selecte d R W D s o urces deter mi nes t he feasi bilit y  of t he e xter nal c o ntr ol ar m. 
Baseli ne c o variates will be selecte d t o c o m pare patie nts fr o m elra nata ma b a n d e xter nal 
c o ntr ol ar ms a n d t o perf or m f urt her statistical a dj ust me nts t o c o ntr ol f or t he baseli ne 
c o nf o u n di n g (e g, via I PT W).
0 9 0 1 7 7 e 1 9 d 6 a 1 0 1 2\ A p pr o v e d\ A p pr o v e d O n: 1 5- M a y- 2 0 2 3 0 2: 3 3 ( G M T)
C CI

P F -0 6 8 6 3 1 3 5 ( Elra nata ma b)
N O N -I N T E R V E N TI O N A L S T U D Y P R O T O C O L
C 1 0 7 1 0 3 1 Versi o n 1. 0, 0 1 Ma y 2 0 2 3
P FI Z E R C O N FI D E N TI A L 
C T 2 4 -WI -G L 0 2 -R F 0 2 4. 0 N o n -I nter ve nti o nal St u d y Pr ot oc ol Te m plate F or Sec o n dar y Data C ollecti o n St u d y 
0 1 -J u n-2 0 2 2 
Pa ge 2 7 of 7 6
Baseli ne c o variates t o be ca pt ure d w ill i ncl u de t h ose relate d t o patie nt de m o gra p hic 
c haracteristics, disease c haracteristics, c o m or bi di t y pr ofile, la b orat or y meas ure me nts, a n d 
M M treat me nt patter ns, i ncl u di n g L O T. Eac h varia ble will be ta ke n o n or bef ore t he i n de x 
date; if bef ore, t he m ost rece nt meas ure me nt will be use d. All varia bles liste d bel o w will be 
use d t o descri be t he st u d y c o h or ts a n d t o a dj ust f or t he baseli ne c o nf o u n di n g. Si nce a s mall 
sa m ple size li mits t he n u m ber of c o variates t hat ca n be reas o na bly acc o u nte d f or i n t he 
statistical a dj ust me nt (e g, I P T W), a sys te matic literat ure re vie w was c o n d ucte d t o i de ntif y t he 
varia bles m ost str o n gl y a n d c o nsiste ntl y c orrelate d wit h o utc o mes i n R W D st u dies c o n d ucte d
a m o n g R R M M patie nts. 
 
 
 
 
 
A d diti o nal c o nf o u n ders i de ntifie d i n t he pre vi o us p hase of t he st u d y will be i ncl u de d i n t he 
a nal ys is t o o pti mize t he bala nce i n cli nical c haracteristics bet wee n partici pa nts fr o m St u d y 
C 1 0 7 1 0 0 3 a n d eac h R W D s o u rce. T hese c o nf o u n ders ha ve bee n selecte d base d o n t heir 
cli nical i m p orta nce a nd rele va nce t o t he disease pr o g n osis a n d disease c o m plicati o ns (le vel 
of ser u m al b u mi n, ser u m calci u m le vel, prese nce of b o ne lesi o ns, e xtra me d ullar y  disease 
[ E M D], he m o gl o bi n, a n d ser u m creati ni ne), li ver d y sf u ncti o n (le vels of bilir u bi n, as partate 
a mi n otra nsferase [ A S T], a n d ala ni ne a mi n otra nsferase [ A L T]), a n d b ur de n of c o m or bi d 
c o n diti o ns ( C harls o n c o m or bi dit y i n de x [ C CI]). Of n ote, t he prese nce of E M D is o nl y 
a vaila ble f or the a nal ys es usi n g c o m bi ne d datasets of St u d y C 1 0 7 1 0 0 3 a n d C O T A patie nts.
M ulti p le i m p utati o ns will be use d t o a d dress t he prese nce of missi n g val ues f or c o variates i n 
R W D .
List of c o v ari ates

A ge at i n de x date

Se x ( male; fe male) 

I S S (I nter nati o nal Sta gi n g S y ste m) sta ge (I; II; III) wit hi n 9 0 da ys bef ore or o n t he 
i n de x date, if feasi ble

E C O G perf or ma nce stat us wit hi n 9 0 da y s bef ore or o n t he i n de x date, if feasi ble 

Ti me si nce i nitial M M dia g n osis ( y ears)

N u m ber of pre -i n de x treat me nt li nes

Pe nta -refract ory stat us at i n de x date

H i g h cyt o g e nic ris k ( yes ; n o) at i n de x date, if feasi ble

E M D i de ntifie d wit hi n 1 2 m o nt hs bef ore or o n t he i n de x date
0 9 0 1 7 7 e 1 9 d 6 a 1 0 1 2\ A p pr o v e d\ A p pr o v e d O n: 1 5- M a y- 2 0 2 3 0 2: 3 3 ( G M T)
C CI

P F -0 6 8 6 3 1 3 5 ( Elra nata ma b)
N O N -I N T E R V E N TI O N A L S T U D Y P R O T O C O L
C 1 0 7 1 0 3 1 Versi o n 1. 0, 0 1 Ma y 2 0 2 3
P FI Z E R C O N FI D E N TI A L 
C T 2 4 -WI -G L 0 2 -R F 0 2 4. 0 N o n -I nter ve nti o nal St u d y Pr ot oc ol Te m plate F or Sec o n dar y Data C ollecti o n St u d y 
0 1 -J u n-2 0 2 2 
Pa ge 2 8 of 7 6

Prese nce of b o ne lesi o ns i n patie nts wit h n o e xtra me d ullar y disease wit hi n 1 2 m o nt hs 
bef ore or o n t he i n de x date

C ha rls o n C o m or bi dit y In de x wit hi n 1 2 m o nt hs bef ore or o n t he i n de x date 

As partate a mi n otra nsferase ( micr o kat/ L ) wit hi n 9 0 da y s bef ore or o n t he i n de x date, 
if feasi ble

Ala ni ne a mi n otra nsferase ( micr o kat/ L ) wit hi n 9 0 da y s bef ore or o n t he i n de x date, if 
feasible

Creati ni ne cleara nce ( m L/ mi n) wit hi n 9 0 da y s before or o n t he i n de x date, if feasi ble

He m o gl o bi n ( g/ L) wit hi n 9 0 da y s bef ore or o n t he i n de x date, if feasi ble

Bilir u bi n ( m g/ d L) Bilir u bi n ( m g/ d L) wit hi n 9 0 da y s bef ore or o n t he i n de x date

Calci u m i n ser u m or plas ma ( m m ol/ L ) re p orte d wit hi n 9 0 da ys bef ore or o n t he i n de x 
date

Ser u m al b u mi n ( g/ d L) re p orte d wit hi n 9 0 da y s bef ore or o n t he i n de x date.
9. 4. D at a S o urces
D at a i nte gr ati o n
U p o n c o nstr ucti n g t he a nal y tical file f or t he c o m paris o n bet wee n St u d y C 1 0 7 1 0 03 a n d 
Flatir o n Healt h, St u d y C 1 0 7 1 0 0 3 a n d C O T A, St u d y  C 1 0 7 1 0 0 3, a n d pr os pecti ve 
o bser vati o nal st u dies , a c o m m o n data m o del f or St u d y C 1 0 7 1 0 0 3 a n d eac h of t he e xter nal 
c o ntr ol ar m s will be create d wit h sta n dar dize d data ele me nts. T he varia ble tra nsf or mati o n
will i ncl u de t he creati o n of t he c o m m o n varia ble t y pe, f or mat, a n d ta x o n o m y . C o nsiste nt 
defi niti o ns will be a p plie d t o create deri ve d varia bles f or treat me nts, i n de x date, o utc o mes, 
a n d c o m or bi dities. All data tra nsf or mati o n will be d oc u me nte d f or tra ns pa re nc y.
C o m bi ne d st u d y d at asets
C o m bi ne d st u d y  datasets will be create d fr o m t he e ntiret y of t he St u d y  C 1 0 7 1 0 0 3 ar m pl us 
t he selecte d e xter nal c o ntr ol ar m base d o n t he data s o urce a n d eli gi bilit y  criteria.
A t otal of 7 c o m bi ne d st u d y datasets will be created. Eac h will use t he sa me patie nts fr o m 
St u d y  C 1 0 7 1 0 0 3 al o n g wit h var y i n g R W patie nts, de pe n di n g u p o n t he R W D s o urce a n d 
i ncl usi o n/e xcl usi o n criteria a p plie d t o i de ntif y t hem, as i n dicate d bel o w.
F or t he mai n a nalys is a n d se nsiti vit y a nalys es t o a d dress t he pri mar y  a n d sec o n dar y 
o bjecti ves :

S t u d y C 1 0 7 1 0 0 3 C o h ort A ar m pl us t he e xter nal c o ntr ol ar m selecte d usi n g critical
eli gi bilit y criteria fr o m C O T A
0 9 0 1 7 7 e 1 9 d 6 a 1 0 1 2\ A p pr o v e d\ A p pr o v e d O n: 1 5- M a y- 2 0 2 3 0 2: 3 3 ( G M T)

P F -0 6 8 6 3 1 3 5 ( Elra nata ma b)
N O N -I N T E R V E N TI O N A L S T U D Y P R O T O C O L
C 1 0 7 1 0 3 1 Versi o n 1. 0, 0 1 Ma y 2 0 2 3
P FI Z E R C O N FI D E N TI A L 
C T 2 4 -WI -G L 0 2 -R F 0 2 4. 0 N o n -I nter ve nti o nal St u d y Pr ot oc ol Te m plate F or Sec o n dar y Data C ollecti o n St u d y 
0 1 -J u n-2 0 2 2 
Pa ge 2 9 of 7 6

S t u d y C 1 0 7 1 0 0 3 C o h ort A ar m pl us t he e xter nal c o ntr ol ar m selecte d usi n g critical
eli gi bilit y criteria fr o m Flatir o n Healt h
F or a se nsiti vit y a nalys is base d o n alter nati ve i ncl usi o n/e xcl usi o n criteria :

S t u d y C 1 0 7 1 0 0 3 C o h ort A ar m pl us t he e xter nal c o ntr ol ar m selecte d usi n g e x pa n de d
eli gi bilit y criteria fr o m C O T A

S t u d y C 1 0 7 1 0 0 3 C o h ort A ar m pl us t he e xter nal c ontr ol ar m selecte d usi n g e x pa n de d
eli gi bilit y criteria fr o m Flatir o n Healt h
F or t he a d diti o nal a nal ys es:

St u d y  C 1 0 7 1 0 0 3 C o h ort A a n d C o h ort B ar ms pl us e xter nal c o ntr ol ar m selecte d 
usi n g critical eli gi bilit y criteria fr o m C O T A

St u d y  C 1 0 7 1 0 0 3 C o h ort A a n d C o h ort B ar ms pl us e xter nal c o ntr ol ar m selecte d 
usi n g critical eli gi bilit y criteria fr o m Flatir o n Healt h

St u d y  C 1 0 7 1 0 0 3 C o h ort A pl us e xter nal c o ntr ol ar m selecte d usi n g critical eli gi bilit y 
criteria fr o m pr os pecti ve o bser vati o nal st u d y C 1 0 7 1 0 1 3 a n d st u d y C 1 0 7 1 0 1 4.
9. 5. St u d y S ize
T he st u d y  sa m ple will be i de ntifie d fr o m t he a nal ysis of sec o n dar y data t hat has alrea d y bee n 
c ollecte d. Acc or di n gl y, t he sa m ple size will be li mite d b y  t he d urati o n of t he o bser vati o n 
wi n d o w. All patie nts w h o meet t he i ncl usi o n/e xcl u si o n criteria defi ne d i n Secti o n 9. 2. 3 will 
be i ncl u de d i n t he a nal y ses. R etr os pecti ve c o h ort St u d y  C 1 0 7 1 0 2 4 , refere nce d i n Secti o n 7 ,
i de ntifie d 3 4 2 a n d 4 7 7 T C R M M patie nts i n Flatir o n Healt h a n d C O T A, res pecti vel y bef ore 
a p pl yi n g i ncl usi o ns a n d e xcl usi o n criteria.
 
N o f or mal sa m ple size esti mati o ns ha ve bee n 
perf or me d f or t his o bser vati o nal st u d y .
9. 6. D at a M a n a ge me nt
T his st u d y  will use str uct ure d data bases fr o m St u d y  C 1 0 7 1 0 0 3, a n o pe n -la bel, m ulti-ce nter, 
n o n -ra n d omi ze d P hase 2 st u d y of elra nata ma b (P F - 0 6 8 6 3 1 3 5) m o n ot hera p y, 3 0 Flatir o n 
Healt h, a n d C O T A (t he latter 2 data bases c ollect i nf or mati o n o n M M patie nts treate d acr oss 
t he U S), a n d pr os pecti ve o bser vati o nal st u dies C 1 0 7 1 0 1 3 a n d C 1 0 7 1 0 1 4. T he R W data 
s o urces h a ve bee n selecte d acc or di n g t o data a vaila bilit y a n d as fit -f or-p ur p ose f or f ulfilli n g 
st u d y o bjecti ves (t he U S F D A, 2 0 1 8 Fra me w or k). T he peri o d f or i n de x dates f or eac h R W 
data base has bee n selecte d t o ali g n as closel y  as p ossi ble t o eac h ot her a n d t o St ud y  
C 1 0 7 1 0 0 3 w hic h will i ncl u de a p pr o xi matel y 1 5 m o nt hs of f oll o w - u p.
0 9 0 1 7 7 e 1 9 d 6 a 1 0 1 2\ A p pr o v e d\ A p pr o v e d O n: 1 5- M a y- 2 0 2 3 0 2: 3 3 ( G M T)
C CI

P F -0 6 8 6 3 1 3 5 ( Elra nata ma b)
N O N -I N T E R V E N TI O N A L S T U D Y P R O T O C O L
C 1 0 7 1 0 3 1 Versi o n 1. 0, 0 1 Ma y 2 0 2 3
P FI Z E R C O N FI D E N TI A L 
C T 2 4 -WI -G L 0 2 -R F 0 2 4. 0 N o n -I nter ve nti o nal St u d y Pr ot oc ol Te m plate F or Sec o n dar y Data C ollecti o n St u d y 
0 1 -J u n-2 0 2 2 
Pa ge 3 0 of 7 6
9. 7. D at a A n al yses
Detaile d met h o d ol o g y  f or s u m mar y a n d statistical a nalys es of t he data c ollecte d i n t his st u d y  
will be d oc u me nte d i n t he statistica l a nalys is pla n ( S A P), w hic h will be date d, file d, a n d 
mai ntai ne d b y t he s p o ns or. 
T he S A P ma y  m o dif y t he pla ns o utli ne d i n t he pr ot oc ol; a n y maj or m o dificati o ns of pri mar y 
e n d p oi nt defi niti o ns or t heir a nal ys es w o ul d be reflecte d i n a pr ot oc ol a me n d me nt.
Ta ble 3 pr o vi des t h e s u m mar y of statistical a nalys es f or t he c o m bi ne d st u d y c o h orts .
0 9 0 1 7 7 e 1 9 d 6 a 1 0 1 2\ A p pr o v e d\ A p pr o v e d O n: 1 5- M a y- 2 0 2 3 0 2: 3 3 ( G M T)

P F -0 6 8 6 3 1 3 5 ( Elra nata ma b)
N O N -I N T E R V E N TI O N A L S T U D Y P R O T O C O L
C 1 0 7 1 0 3 1 Versi o n 1. 0, 0 1 Ma y 2 0 2 3
P FI Z E R C O N FI D E N TI A L 
C T 2 4 -WI -G L 0 2 -R F 0 2 4. 0 N o n -I nter ve nti o nal St u d y Pr ot oc ol Te m plate F or Sec o n dar y Data C ollecti o n St u d y 
0 1 -J u n-2 0 2 2 
Pa ge 3 1 of 7 6
T a ble 3 . S u m m ar y of S t atistic al A n al ys es f or Co m bi ne d S t u d y C o h orts.
Critic al eli gi bilit y criteri a 
s a m ples selecte d fr om 
C O T A or Fl atir o n He alt h 
E x p a n de d 
eli gi bilit y 
criteri a 
s a m ples 
selecte d fr o m 
C O T A or
Fl atir o n 
He alt h
Critic al eli gi bilit y criteri a 
s a m ple selecte d fr o m 
C 1 0 7 1 0 1 3 a n d C 1 0 7 1 0 1 4
Mai n a nal ys es

I P T W c o m paris o n of P F S i n 
elra nata ma b ( C o h ort A) vers us 
S O C -treate d patie nts selecte d 
fr o m C O T A

I P T W c o m paris o n of  P F S i n 
elra nata ma b ( C o h ort A) vers us 
S O C -treate d patie nts selecte d 
fr o m Flatir o n Healt h

I P T W c o m paris o n of O S i n 
elra nata ma b ( C o h ort A) vers u s 
S O C -treate d patie nts selecte d 
fr o m C O T A

I P T W c o m paris o n of  O S in 
elra nata ma b ( C o h ort A) vers us 
S O C -treate d patie nts selecte d 
fr o m Flatir o n Healt h
Se nsiti vit y  
a nal ys es

D o u bl y r o b ust c o m paris o ns of 
P F S i n elra nata ma b ( C o h ort A) 
vers us S O C -treate d patie nts
selecte d fr o m C O T A

D o u bl y r o b ust c o m paris o ns of 
P F S i n elra nata ma b ( C o h ort A) 
vers us S O C -treate d patie nts
selecte d fr o m Flatir o n Healt h

D o u bl y r o b ust c o m paris o ns of 
O S i n elra nata ma b ( C o h ort A) 
vers us S O C -treate d patie nts
selecte d fr o m C O T A

D o u bl y r o b ust c o m paris o ns of 
O S i n elra nata ma b ( C o h ort A) 
vers us S O C -treate d patie nts
selecte d fr o m Flatir o n Healt h

Q ua ntitati ve bias assess me nts 
( usi n g I P T W esti mates) w he n 
c o m pari n g P F S i n elra nata ma b 
( C o h ort A) vers us S O C -treate d 
patie nts selecte d fr o m C O T A

Q ua ntitati ve bias assess me nts 
( usi n g I P T W esti mates) w he n 
c o m pari n g P F S i n elra nata ma b 
( C o h ort A) vers us S O C -treate d 

I P T W 
c o m paris o n of 
P F S i n 
elra nata ma b 
( C o h ort A) 
vers us S O C -
treate d patie nts 
selecte d fr o m 
C O T A

I P T W 
c o m paris o n of 
P F S i n
elra nata ma b 
( C o h ort A) 
vers us S O C -
treate d patie nts 
selecte d fr o m 
Flatir o n Healt h

I P T W 
c o m paris o n of 
O S i n 
elra nata ma b 
( C o h ort A) 
vers u s S O C-
treate d patie nts 
0 9 0 1 7 7 e 1 9 d 6 a 1 0 1 2\ A p pr o v e d\ A p pr o v e d O n: 1 5- M a y- 2 0 2 3 0 2: 3 3 ( G M T)

P F -0 6 8 6 3 1 3 5 ( Elra nata ma b)
N O N -I N T E R V E N TI O N A L S T U D Y P R O T O C O L
C 1 0 7 1 0 3 1 Versi o n 1. 0, 0 1 Ma y 2 0 2 3
P FI Z E R C O N FI D E N TI A L 
C T 2 4 -WI -G L 0 2 -R F 0 2 4. 0 N o n -I nter ve nti o nal St u d y Pr ot oc ol Te m plate F or Sec o n dar y Data C ollecti o n St u d y 
0 1 -J u n-2 0 2 2 
Pa ge 3 2 of 7 6
Critic al eli gi bilit y criteri a 
s a m ples selecte d fr om 
C O T A or Fl atir o n He alt h 
E x p a n de d 
eli gi bilit y 
criteri a 
s a m ples 
selecte d fr o m 
C O T A or
Fl atir o n 
He alt h
Critic al eli gi bilit y criteri a 
s a m ple selecte d fr o m 
C 1 0 7 1 0 1 3 a n d C 1 0 7 1 0 1 4
patie nts selecte d fr o m Flatir o n 
Healt h

Q ua ntitati ve bias assess me nts 
( usi n g I P T W esti mates) w he n 
c o m pari n g O S i n elra nata ma b 
( C o h ort A) vers us S O C -treate d 
patie nts selecte d fr o m C O T A

Q ua ntitati ve bias assess me nt s 
( usi n g I P T W esti mates) w he n 
c o m pari n g O S i n elra nata ma b 
( C o h ort A) vers us S O C -treate d 
patie nts selecte d fr o m Flatir o n 
Healt h
selecte d fr o m 
C O T A

I P T W
c o m paris o n of 
O S i n 
elra nata ma b 
( C o h ort A) 
vers us S O C -
treate d patie nts 
selected fr o m  
Flatir o n Healt h
A d diti o nal 
a nal ys es

C o m paris o ns of P F S i n P S 
matc he d elra nata ma b ( C o h ort A) 
a n d S O C -treate d patie nts
selecte d fr o m C O T A

C o m paris o ns of P F S i n P S 
matc he d elra nata ma b ( C o h ort A) 
a n d S O C -treate d patie nts
selecte d fr o m Flatir o n Healt h

C o m p aris o ns of O S i n P S 
matc he d elra nata ma b ( C o h ort A) 
a n d S O C -treate d patie nts
selecte d fr o m C O T A

C o m paris o ns of O S i n P S 
matc he d elra nata ma b ( C o h ort A) 
a n d S O C -treate d patients
selecte d fr o m Flatir o n Healt h

I P T W c o m paris o n of P F S i n 
elra nata ma b ( C o h ort A+ C o h ort 
B ) vers us S O C- treate d patie nts 
selecte d fr o m C O T A

I P T W c o m paris o n of P F S i n 
elra nata ma b ( C o h ort A + C o h ort 
B ) vers us S O C-treate d patie nts 
selecte d fr o m Flatir o n Healt h

I P T W c o m paris o n of O S i n 
elra nata ma b ( C o h ort A + C o h ort 
B ) vers us S O C-treate d patie nts 
selecte d fr o m C O T A

I P T W c o m paris o n of O S i n 
elra nata ma b ( C o h ort A + C o h ort 

N u m ber a n d perce nta ge of 
partici pa nts w h o c o m plete d 
Q L Q -C 3 0 d o m ai ns, Q L Q-
M Y 2 0
d o m ai ns, E Q-5 D i n de x, 
E Q -5 D -V A S, a n d P GI C at 
baseli ne a n d eac h f oll o w - u p 
visit .

Descri pti ve sta tistics of 
Q L Q -C 3 0 d o m ai ns, Q L Q-
M Y 2 0
d o m ai ns, E Q-5 D i n de x, 
E Q -5 D -V A S, a n d P GI C i n 
elra nata ma b ( C o h ort A) a n d 
S O C -treate d patie nts, i ncl u di n g 
mea n a n d a bs ol ute c ha n ge i n 
P R O sc ores (fi nal assess me nt 
sc ore mi n us baseli ne sc ore)
Base d o n t he res ults of t he 
d escri pti ve a nal ysis of 
c o m plete ness, a n d t he ti mi n g of 
assess me nts i n t he t w o treatme nt 
ar ms, t he f oll o wi n g a nal yses ma y 
be c o n d ucte d:

C o m paris o n of c ha n ge fr o m 
baseli ne o n t he Q L Q -C 3 0
d o m ai ns, Q L Q-M Y 2 0
d o m ai ns, 
E Q - 5D i n de x, a n d E Q -5 D -V A S
usi n g naï ve a n d a dj uste d mi xe d -
effect m o del s
0 9 0 1 7 7 e 1 9 d 6 a 1 0 1 2\ A p pr o v e d\ A p pr o v e d O n: 1 5- M a y- 2 0 2 3 0 2: 3 3 ( G M T)

P F -0 6 8 6 3 1 3 5 ( Elra nata ma b)
N O N -I N T E R V E N TI O N A L S T U D Y P R O T O C O L
C 1 0 7 1 0 3 1 Versi o n 1. 0, 0 1 Ma y 2 0 2 3
P FI Z E R C O N FI D E N TI A L 
C T 2 4 -WI -G L 0 2 -R F 0 2 4. 0 N o n -I nter ve nti o nal St u d y Pr ot oc ol Te m plate F or Sec o n dar y Data C ollecti o n St u d y 
0 1 -J u n-2 0 2 2 
Pa ge 3 3 of 7 6
Critic al eli gi bilit y criteri a 
s a m ples selecte d fr om 
C O T A or Fl atir o n He alt h 
E x p a n de d 
eli gi bilit y 
criteri a 
s a m ples 
selecte d fr o m 
C O T A or
Fl atir o n 
He alt h
Critic al eli gi bilit y criteri a 
s a m ple selecte d fr o m 
C 1 0 7 1 0 1 3 a n d C 1 0 7 1 0 1 4
B) vers us S O C -treate d patie nts 
selecte d fr o m Flatir o n Healt h

S u b gr o u p a nal ys es t o 
c o m pare P F S a n d O S i n 
partici pa nts of St u d y C 1 0 7 1 0 0 3 
a n d patie nts fr o m Flatir o n 
Healt h a n d C O T A 
I n t his st u d y, meas ures ta ke n t o re d uce p ote ntial bias i ncl u de a pre-s pecifie d a n d detaile d 
S A P. A d diti o nall y , t he pr o pe nsit y sc ore ( P S) bala nci n g ste ps will be c o n d ucte d 
i n de pe n de ntly a n d bef ore a vaila bilit y/ k n o wle d ge of t he o utc o m es (ie, bala nci n g will be 
c o n d ucte d usi n g o nl y baseli ne c haracteristic data fr o m a preli mi nar y data c ut rat her t ha n t he 
fi nal data c ut c o ntai ni n g St u d y C 1 0 7 1 0 0 3 efficac y o utc o me data).
M ulti ple i m p ut ati o ns
MI  will be perf or m e d t o a d dress missin g val ues o nl y i n baseli ne c o variates u n der t he 
ass u m pti o n of missi n g ness at ra n d o m ( M A R) .
MA R ass u mes t hat t here mi g ht be s ys te matic 
differe nces bet wee n t he missi n g a n d o bser ve d val ues of pr o g n ostic c haracteristics , b ut t hese 
ca n be e ntirel y e x plai ne d b y ot her o bser ve d pr o g n ostic c haracteristics, t he treat me nt varia ble,
a n d t he o bser ve d overa ll s ur vi val. M ulti ple i m p utati o ns b y  c hai ne d e q uati o ns ( MIC E) will be 
perf or me d usi n g t he f ull y c o n diti o nal s pecificati o n ( F C S) met h o d .3 4 T his a p pr oac h i m p utes 
m ulti variate missi n g data o n a varia ble -b y -vari a ble basis, b y  s pecif yi n g a n i m p utati o n m o del 
f or eac h varia ble. T he tec h ni q ue is iterati ve a n d pr ocee ds via Gi b bs sa m pli n g if t he i nitial 
j oi nt distri b uti o n defi ne d b y t he s pecifie d c o n diti o nal distri b uti o ns e xists.3 5
As wit h ot her iterati ve pr oce d ures, dia g n ostics are esse ntial t o assess a n d e val uate t he 
res ulti n g i m p utati o n m o dels o btai ne d via MI C E a n d t o deter mi ne w het her c o n ver ge nce has 
bee n ac hie ve d ( 3 6). Vis ual assess me nt will be perf or me d usi n g w or m pl ots , stri p pl ots, a n d 
de nsit y  pl ots t o e xa mi ne t he o bser ve d a n d i m p ute d data a n d c o m pare t heir distri b uti o ns. 
C o n ver ge nce will be e val uate d b y  pl otti n g t he mea n a n d varia nce of each i m p utati o n r u n 
acr oss iterati o ns, t o c o nfir m t hat t here are n o a p pare nt tre n d s. If c o n ver ge nce has n ot bee n 
ac hie ve d, t he n u m ber of iterati o ns will be i ncrease d u ntil mea ns a n d varia nces sta bilize.
F or eac h i m p ute d dataset, t he c o m parati ve a nalys is of P FS a n d O S will be c o n d ucte d, t he 
i n di vi d ual esti mates will be c o m bi ne d usi n g R u b i n’s r ules2 4 (the deta ils are pr o vi de d i n t he 
S A P ).
0 9 0 1 7 7 e 1 9 d 6 a 1 0 1 2\ A p pr o v e d\ A p pr o v e d O n: 1 5- M a y- 2 0 2 3 0 2: 3 3 ( G M T)

P F -0 6 8 6 3 1 3 5 ( Elra nata ma b)
N O N -I N T E R V E N TI O N A L S T U D Y P R O T O C O L
C 1 0 7 1 0 3 1 Versi o n 1. 0, 0 1 Ma y 2 0 2 3
P FI Z E R C O N FI D E N TI A L 
C T 2 4 -WI -G L 0 2 -R F 0 2 4. 0 N o n -I nter ve nti o nal St u d y Pr ot oc ol Te m plate F or Sec o n dar y Data C ollecti o n St u d y 
0 1 -J u n-2 0 2 2 
Pa ge 3 4 of 7 6
Pr o pe nsit y sc ores a n d i n verse pr o b a bilit y of tre at me nt wei g hti n g
I n a nalys es of R W D, a n i m p orta nt c o nsi derati o n i n t he i de ntificati o n of p ote ntiall y  ca usal 
effects is a c o ntr ol f or c o nf o u n di n g. I P T W is a well-esta blis he d met h o d f or ca usal i nfere nce 
i n n o nra n d o mize d st u dies, w hic h ai ms t o eli mi nate t he effect of c o nf o u n di n g b y o bser ve d
baseli ne patie nt c haracteristics, i m pr o ve c o variate bala nce, a n d t here b y o btai n u n biase d 
esti mates of treat me nt effects . Esti mati n g t he P S, o n w hic h I P T W relies, is a f or m of 
di me nsi o nalit y  re d ucti o n, i n w hic h se veral i n di vi d ual c haracteristics rele va nt to treat me nt 
assi g n me nt a n d t he o utc o me, or t h ose relate d o nl y  t o o utc o me de vel op me n t, are use d t o 
esti mate t he c o n diti o nal pr o ba bilit y t hat t he patie nt is assi g ne d t o a gi ve n treat me nt. I P T W is 
a n a p pr oac h ofte n use d i n n o n-ra n d o mize d st u dies t o create a pse u d o-p o p ulati o n i n w hic h t he 
c o variates are i n de pe n de nt of t he treat me nt assi g n me n t, t here by per mitti n g a n u n biase d 
esti mate of t he A T E , pr o vi de d t hat f u n da me ntal ass u m pti o ns of ca usal i nfere nce are satisfie d .
T he P S will be esti mate d usi n g l o gistic re gre ssi o n m o dels, w here t he de pe n de nt varia ble is a 
bi nar y  i n dicat or of t he treat me nt ar m (elra nata ma b vers us S O C). C o variates descri be d i n
Secti o n 9. 3 will be e ntere d i nt o t he l o gistic re gressi o n m o del . Dia g n ostics of t he esti mate d P S 
will be a p plie d b y  e xa mi ni n g t he distrib u tio n of t he P S i n eac h treat me nt gr o u p t o i de ntif y t he 
de gree of o verla p a n d re gi o n of c o m m o n s u p p ort .3 7 T he esti mate d P S will be u se d t o ge nerate 
I P T Ws, w hic h ma y  be sta bilize d t o re d uce varia nce a n d t he effe ct of ver y lar ge wei g hts. A 
sta n dar dize d mea n differe nce ( S M D) of ≥2 0 % will be use d as a n i n dicat or of c o variate 
i m bala nce bet wee n t he treat me nt ar ms re q uiri n g f urt her i n vesti gati o n a n d refi ni n g t he l o gistic 
m o del f or deri vi n g t he wei g hts .3 8 ,3 9
9. 7. 1. Descri pti ve S t atistics
Ta b ular s u m maries of baseli ne patie nt de m o gra p hics a n d cli nical c haracteristics b y  treat me nt 
ar m will be prese nte d. S u m mar y statistics will i ncl u de fre q ue ncie s a n d perce nta ges 
(cate g orical varia bles) a n d mea n/ me dia n, mi ni m u m/ ma xi m u m wit h sta n dar d 
de viati o n/i nter q uartile ra n ge (c o nti n u o us varia bles).
P F S a n d O S will be descri be d usi n g Ka pla n -Meier ( K M) met h o ds .4 0 T he esti mates of t he 
ti me-t o-e ve nt c ur ves will be pl otte d a n d t he me dia n P F S a n d O S wit h 9 5 % CI  will be 
re p orte d. T he 9 5 % CI f or t he me dia n will be esti mate d usi n g t he Br o o k me yer a n d Cr o wle y 
met h o d .4 1 T he P F S a n d O S e ve nt rates (t he e ve nts of P D a n d deat h, res pecti vel y) will be 
esti mate d wit h c orres p o n di n g t w o -si de d 9 5 % CIs. T he CIs f or t he s ur vi val f u ncti o n esti mates 
will be deri ve d usi n g t he l o g( -l o g) met h o d acc or di n g t o Kal bfleisc h a n d Pre ntice.4 2 T he 
esti mate of t he sta n dar d er r or will be c o m p ute d usi n g Gree n w o o d’s f or m ula.4 3 F or P F S, t he 
fre q ue nc y ( n u m ber a n d perce nta ge) of patie nts wit h eac h e ve nt ty pe ( P D or deat h) a n d
ce ns ori n g reas o ns f or PF S will be prese nte d al o n g wit h t he o verall e ve nt a n d ce ns or rates.
T he t otal n u m ber of deat hs a n d ce ns ori n g reas o ns f or O S will be prese nte d al o n g wit h t he 
o verall e ve nt a n d ce ns or rates.
0 9 0 1 7 7 e 1 9 d 6 a 1 0 1 2\ A p pr o v e d\ A p pr o v e d O n: 1 5- M a y- 2 0 2 3 0 2: 3 3 ( G M T)

P F -0 6 8 6 3 1 3 5 ( Elra nata ma b)
N O N -I N T E R V E N TI O N A L S T U D Y P R O T O C O L
C 1 0 7 1 0 3 1 Versi o n 1. 0, 0 1 Ma y 2 0 2 3
P FI Z E R C O N FI D E N TI A L 
C T 2 4 -WI -G L 0 2 -R F 0 2 4. 0 N o n -I nter ve nti o nal St u d y Pr ot oc ol Te m plate F or Sec o n dar y Data C ollecti o n St u d y 
0 1 -J u n-2 0 2 2 
Pa ge 3 5 of 7 6
9. 7. 2. M ai n A n al yses
F or c o m parati ve effecti ve ness , P FS a n d O S will be ass esse d usi n g I P T W c o m paris o ns 
esti mati n g t he a vera ge treat me nt effect ( A T E).
P F S a n d O S will be c o m pare d bet wee n treat me nt ar ms usi n g hazar d rati os esti mate d fr o m 
C o x pr o p orti o nal hazar ds m o dels. T he pr o p orti o nal hazar ds ass u m pti o n w ill be c hec ke d, a n d 
i n case of de viati o ns, a restricte d mea n s ur vi val ti me m o del will be a p plie d i nstea d of t he 
C o x pr o p orti o nal hazar ds re gressi o n m o del .4 4 Sta n dar d err ors a n d CI f or t he I P T W esti mat or 
will be o bta i ne d via a r o b ust san d wic h -t ype esti mat or of varia nce. 4 5
Beca use m e dia n O S li kel y will n ot be reac he d i n St u d y C 1 0 7 1 0 0 3, o nl y  f or t he c o m bi ne d 
st u d y c o h orts selecte d usi n g critical eli gi bilit y criteria, I P T wei g hte d s ur vi val pr o ba b ilities 
will be descri be d usi n g K M esti mat or a n d c o m pare d bet wee n t w o treat me nt gr o u ps usi n g t he 
l o g-ra n k test 4 6 ,4 7 at 1 2 a n d 1 5 m o nt hs of f o ll o w- u p.
9. 7. 3. Se nsiti vit y A n a l yses
T o e val uate t he r o b ust ness of res ults fr o m t he pri mar y a nalys es, se nsiti vit y a nal yses will be 
c o n d ucte d.
D o u bl y r o b ust c o m p aris o n
A c o n diti o nal a vera ge treat me nt effect ( C A T E) a n d its ass ociate d hazar d rati o will be 
esti mate d usi n g a se mi -para metric a p pr oac h descri be d b y  Ya dl o ws k y et al.4 8 ,4 9 T his esti mat or 
will pr o vi de a d o u bl y  r o b ust c o m paris o n f or P F S a n d O S bet wee n treat me nt ar ms. Sta n dar d
err ors a n d 9 5 % c o nfi de nce i nter vals are o btai ne d f or t his esti mat or usi n g t he n o n-para metric 
b o otstra p. T he met h o d is i m ple me nte d i n R i n t he prec me d pac ka ge, usi n g t he a tefits ur v 
f u ncti o n.
A nal y ses e m pl o yi n g a d o u bl y r o b ust esti mat or re q uire fitti n g 2 m odels: a m o del f or treat me nt 
or e x p os ure stat us, a n d a sec o n d m o del f or t he o utc o me of i nterest. As wit h ot her ca usal 
i nfere nce met h o ds, vali d a n d u n biase d esti mates re q uire ass u m pti o ns of n o u n meas ure d 
c o nf o u n di n g (e xc ha n gea bilit y), p ositi vit y (t he e x peri m e ntal treat me nt ass u m pti o n), n o 
i nterfere nce, a n d c o nsiste nc y.5 0 Ass u mi n g t hese ass u m pti o n s are u p hel d, if at least 1 of t hese 
2 m o dels is c orrectl y s pecifie d, res ulti n g i n a d o u bl y  r o b ust esti mate t hat ma y  re mai n 
c o nsiste nt a n d u n biase d .5 1
Dia g n ostics will be a p plie d t o assess w het her t he d o u bl y  r o b ust m o del a p pears t o be 
well -s pecifie d.5 2
0 9 0 1 7 7 e 1 9 d 6 a 1 0 1 2\ A p pr o v e d\ A p pr o v e d O n: 1 5- M a y- 2 0 2 3 0 2: 3 3 ( G M T)

P F -0 6 8 6 3 1 3 5 ( Elra nata ma b)
N O N -I N T E R V E N TI O N A L S T U D Y P R O T O C O L
C 1 0 7 1 0 3 1 Versi o n 1. 0, 0 1 Ma y 2 0 2 3
P FI Z E R C O N FI D E N TI A L 
C T 2 4 -WI -G L 0 2 -R F 0 2 4. 0 N o n -I nter ve nti o nal St u d y Pr ot oc ol Te m plate F or Sec o n dar y Data C ollecti o n St u d y 
0 1 -J u n-2 0 2 2 
Pa ge 3 6 of 7 6
Q u a ntit ati ve bi as assess me nt
Q ua nt itati ve bias assess me nt will be d o ne usi n g nullificati o n a nal ysis t o assess t he p ote ntial 
i nfl ue nce of u n meas ure d c o nf o u n di n g o n t he H R esti mates fr o m t he mai n a nal y sis. A 
s h ortlist of s us pecte d u n meas ure d c o nf o u n ders will be i de ntifie d; t hese are varia ble s t hat 
were o bser v e d i n St u d y  C 1 0 7 1 0 0 3 b ut n ot a vaila ble ( or o nl y wit h s u bsta ntial missi n g ness) i n 
t he R W D s o urces. T he E- val ue will be c o m p ute d a n d reflects t he mi ni m um stre n gt h of 
ass ociati o n t he c o nf o u n der w o ul d nee d t o ha ve wit h t he e x p os ure a n d o utc o m e, c o n diti o nal 
o n t he meas ure d c o variates, t o f ull y e x plai n a wa y t he o bser ve d treat me nt effect .5 3
E -val ues are relati vel y i nt uiti ve a n d offer a c o m pre he nsi ble, easil y c o m m u nicate d s u m mar y 
of t he fi n di n gs . T he l owest p ossi ble E - val ue is 1, a n d as t he val ue i ncreases, s o d oes t he 
i m plie d de gree of bias re q uire d t o ex plai n t he res ults .5 4
Se nsiti vit y a n al yses b ase d o n t he R W s a m ple i de ntifie d usi n g e x p a n de d eli gi bi lit y 
criteri a
T o assess t he p ote ntial i nfl ue nce of p o p ulati o n selecti o n o n t he o bser ve d effect esti mates, 
P F S a n d O S will be c o m pare d i n elra nata ma b ( C o h ort A) vers us S O C-treate d patie nts i n
c o m bi ne d st u d y c o h orts selecte d usi n g t he e x pa n de d eli gi bilit y criteria. I PT Ws wi ll be 
estimate d as descri be d i n Secti o n 9. 7 . Descri pti ve statistics will be re p orte d as descri be d i n 
Secti o n 9. 7. 1 . P F S a nd O S will be c o m pare d bet wee n treat me nt ar ms usi n g I PT- wei g hte d 
C o x pr o p orti o nal hazar ds m o dels . T he pr o p orti o nal hazar ds ass u m pti o n will be c hec ke d, a n d 
i n case of de viati o ns, a restricte d mea n s ur vi val ti me m o del will be a p plie d i nstea d of t he 
C o x pr o p or ti o nal hazar ds re gressi o n m o del.4 4
9. 7. 4. A d diti o n al A n al yses
A n al ysis of p atie nts m atc he d o n P S f or t he esti m ati o n of t he A T T effect
T his a nal y sis will be c o n d ucte d i n t he c o m bi ne d st u d y c o h orts selecte d wit h critical
eli gi bilit y criteria. T o esti mate t he A T T effect, eac h pat ie nt fr o m t he C 1 0 7 1 0 0 3 trial will be 
matc he d t o patie nts fr o m a n o bser vati o nal c o h ort usi n g gree d y nearest nei g h b or matc hi n g o n 
0. 2 sta n dar d de viati o n of t he l o git of t he P S .5 5 Pre vi o us st u dies s h o we d t hat t his cali per 
all o we d t he eli mi nati o n of a b o ut 9 9 % of t he bias d ue t o t he meas ure d c o nf o u n ders. Patie nts 
wit h n o matc h will be e xcl u de d fr o m t he a nal ys is. P S will be esti mate d as descri be d a b o ve i n 
Secti o n 9. 7 . Descri pti ve statistics will be re p orte d as descri be d i n Secti o n 9. 7. 1 . P F S a n d O S 
will be c o m pare d bet wee n treat me nt ar ms usi n g hazar d rati os es ti mate d fr o m C o x 
pr o p orti o nal hazar ds m o dels. T he pr o p orti o nal hazar ds ass u m pti o n will be c hec k e d, a n d i n 
case of de viati o ns, a restricte d mea n s ur vi val ti me m o del will be a p plie d i nstea d of t he C o x 
pr o p orti o nal hazar ds re gres si o n m o del.4 4
0 9 0 1 7 7 e 1 9 d 6 a 1 0 1 2\ A p pr o v e d\ A p pr o v e d O n: 1 5- M a y- 2 0 2 3 0 2: 3 3 ( G M T)

P F -0 6 8 6 3 1 3 5 ( Elra nata ma b)
N O N -I N T E R V E N TI O N A L S T U D Y P R O T O C O L
C 1 0 7 1 0 3 1 Versi o n 1. 0, 0 1 Ma y 2 0 2 3
P FI Z E R C O N FI D E N TI A L 
C T 2 4 -WI -G L 0 2 -R F 0 2 4. 0 N o n -I nter ve nti o nal St u d y Pr ot oc ol Te m plate F or Sec o n dar y Data C ollecti o n St u d y 
0 1 -J u n-2 0 2 2 
Pa ge 3 7 of 7 6
C o m p ar ati ve a n al yses of P F S a n d O S i ncl u di n g p artici p a nts fr o m St u d y C 1 0 7 1 0 0 3 
pre vi o usl y e x p ose d t o B C M A -directe d t her a pies
T he g oal of t his a nal ys is is t o e x pl ore if pre vi o us treat me nt wit h B C M A-directe d t hera pies 
ma y  i m pact t he efficac y o utc o mes o bser ve d i n St u d y  C 1 0 7 1 0 0 3. T his a nal ysis will be 
perf or me d usi n g all partici pa nts of St u d y  1 0 7 1 0 0 3 (ie, C o h ort A a n d C o h ort B) a n d R W 
patie nts selecte d wit h critical eli gi bilit y criteria. I PT Ws will be e sti mate d as descri be d i n 
Secti o n 9. 7 . Descri pti ve statistics will be re p orte d as descri be d i n Secti o n 9. 7. 1 . P F S a n d O S 
will be c o m pare d b et wee n treat me nt ar ms us i n g I P T-wei g hte d C o x pr o p orti o nal hazar ds 
m o dels. T he pr o p orti o nal hazar ds ass u m pti o n will be c hec ke d, a n d i n case of de viati o ns, a 
restricte d mea n s ur vi val ti me m o del will be a p plie d i nstea d of t he C o x pr o p orti o nal hazar ds 
re gressio n m o del .4 4
A n al ysis of P R O s
F or eac h treat me nt gr o u p (elra nata ma b or S O C) , t he n u m ber a n d perce nta ge of partici pa nts 
w h o c o m plete d t he i nstr u me nts at eac h visit , will be s u m marize d . T he ti mi n g of P R O 
c om pleti o n b y  treat me nt gr o u p will be descri be d. A n i nstr u me nt will be c o nsi dere d 
c o m plete d if at least o ne ite m was a ns were d b y t he partici pa nt.
Descri pti ve statistics of P R Os i n elra nata ma b a n d S O C -treate d patie nts
S u m mar y  statistics will be rep orte d f or eac h of t he P R Os liste d i n Secti o n 9. 3 b y  visit (t he 
baseli ne visit a n d t he f oll o w- u p visit s) as well as c ha n ges i n sc ores fr o m t he baseli ne t o t he
f oll o w- u p visit s.  
 
 
 
 
If t he sa m ple size per mits, t he c o m parati ve a nal y ses of P R Os descri be d bel o w will be 
c o n d ucte d.
 
 
O nl y  patie nts w h o c o m plete d t he P R O 
i nstr u me nts at baseli ne will be i ncl u de d i n t he a nal ysis.
C o m paris o n of c ha n ge fr o m baseli ne i n elra nata ma b an d S O C -treate d patie nts
A m o n g patie nts f or w h o m a baseli ne a n d at least t w o f oll o w - u p meas ures f or t he sa me P R O 
o utc o me are a vaila ble, a re peate d meas ures mi xe d -effects m o del will be fit t o t he data t o 
e xa mi ne t he effects o f ti me ( visit) a m o n g patie nts treate d wit h elra nata ma b vers us S O C.
Mi xe d -effect s m o del wit h re peate d meas ures ( M M R M) will be carrie d o ut f or t he d o mai ns of 
0 9 0 1 7 7 e 1 9 d 6 a 1 0 1 2\ A p pr o v e d\ A p pr o v e d O n: 1 5- M a y- 2 0 2 3 0 2: 3 3 ( G M T)
C CI
C CI

P F -0 6 8 6 3 1 3 5 ( Elra nata ma b)
N O N -I N T E R V E N TI O N A L S T U D Y P R O T O C O L
C 1 0 7 1 0 3 1 Versi o n 1. 0, 0 1 Ma y 2 0 2 3
P FI Z E R C O N FI D E N TI A L 
C T 2 4 -WI -G L 0 2 -R F 0 2 4. 0 N o n -I nter ve nti o nal St u d y Pr ot oc ol Te m plate F or Sec o n dar y Data C ollecti o n St u d y 
0 1 -J u n-2 0 2 2 
Pa ge 3 8 of 7 6
Q L Q -C 3 0 a n d Q L Q -M Y 2 0, E Q -5 D i n de x , a n d E Q-V A S t o e xa mi ne t he effects of ti me 
( visit) b y c o h ort a n d ove rall. In t he m o del, o utc o mes ar e p ost -baseli ne sc ores (a n d c ha n ge 
sc ores se parately) , t he pre dict or is t he treat me nt gr o u p (elra nata ma b vers us S O C), a n d t he 
c o ntr olli n g c o variates are t he c orres p o n di n g baseli ne P R O sc ore, 
 
 
S u b gr o u p a n al ysis
Beca use t he treat me nt patter ns descri be d i n C O T A a n d Fla tir o n ma y n ot ge neralize t o all 
n o n- U S c o u ntries , s u b gr o u p a nal ys es will be perf or me d t o o nl y  i ncl u de treat me nts fre q ue ntly 
a vaila ble f or t his patie nt p o p ulati o n i n n o n- U S c o u ntries.   T he list of treat me nts f or 
s u b-gr o u p a nal ys es is pr o vi de d i n A n ne x 3 .
S u b gr o u p a nal ys es will be c o n d ucte d t o c o m pare P F S a n d O S i n partici pa nts of St u d y  
C 1 0 7 1 0 0 3 C o h ort A a n d patie nts fr o m Flatir o n Healt h a n d C O T A selecte d wit h critical 
eli gi bilit y criteria.
F or eac h treat me nt s u b gr o u p, I PT Ws will b e esti mate d as descri be d i n Secti o n 9. 7 . If t he 
sa m ple size is s ufficie nt, P F S a n d O S will be c o m pare d bet wee n partici pa nts of St u d y  
C 1 0 7 1 0 0 3 a n d patie nts fr o m Flatir o n Healt h a n d C O T A usi n g I PT wei g hte d C o x’s 
pr o p orti o nal hazar d m o del .
I n case of i ns ufficie nt sa m ple size, o nl y descri pti ve a nalys is will be pr o vi de d (Secti o n 9. 7. 1 ).
9. 8. Q u alit y C o ntr ol
T his is a retr os pecti ve st u d y, s o iss ues of q ualit y c o ntr ol at st u d y sites, e g, data q ueries, d o 
n ot a p pl y . A nal y ses ar e pr o gra m me d acc or di n g t o t he s pecificati o ns i n t he pr ot oc ol, a n d if 
a p plica ble, t he statistical a nal ys is pla n a n d d ocu me nte d i n a pr o gra m mi n g pla n. Fi nal 
deli vera bles are r e vie we d a n d verifie d b y a sec o n d, i n de pe n de nt pr o gra m mer w h o ma y  also 
perf or m d o u b le pr o gra m mi n g. All q ualit y  c hec ks are d oc u me nte d i n t he pr o gra m mi n g pla n.
9. 9. Li mit ati o ns of t he R ese arc h M et h o d s
U nli ke cli nical trial setti n gs wit h s pecific defi niti o ns of st u d y  o utc o mes a n d sc he d ule d 
assess me nts descri be d i n t he pr ot oc ol, t he assess me nt of t reat me nt res p o nse i ncl u di n g 
pr o gressi o ns i n R W cli nical practice setti n gs ma y  n ot be ma de c o nsiste ntly acr oss patie nts 
a n d p h ysicia ns. Specificall y , i n real-w orl d o bser vati o nal st u dies, es peciall y  t h ose perf or me d 
retr os pecti vely, it is n ot p ossi ble t o i m pl e me nt c o nsiste nt m o nit ori n g a n d a p plicati o n of 
h o m o ge n o us e val uati o n criteria (e g, I M W G) t hat are i n here nt t o cli nical trial desi g n. As s uc h, 
i n R W patie nts, de pe n di n g o n ho w ofte n cli nical assess me nts are ma de, t he date of disease 
pr o gressi o n is m ore li ke l y t o be dia g n ose d later t ha n it w o ul d be dia g n ose d if patie nts ha d 
sc he d ule d assess me nts si milar t o cli nical st u d y setti n gs.5 6 ,5 7 T his ma y  res ult i n l o n ger P F S 
a n d bias t he c o m parati ve effecti ve ness esti mates i n fa v or of t he R W ar ms.
0 9 0 1 7 7 e 1 9 d 6 a 1 0 1 2\ A p pr o v e d\ A p pr o v e d O n: 1 5- M a y- 2 0 2 3 0 2: 3 3 ( G M T)
C CI

P F -0 6 8 6 3 1 3 5 ( Elra nata ma b)
N O N -I N T E R V E N TI O N A L S T U D Y P R O T O C O L
C 1 0 7 1 0 3 1 Versi o n 1. 0, 0 1 Ma y 2 0 2 3
P FI Z E R C O N FI D E N TI A L 
C T 2 4 -WI -G L 0 2 -R F 0 2 4. 0 N o n -I nter ve nti o nal St u d y Pr ot oc ol Te m plate F or Sec o n dar y Data C ollecti o n St u d y 
0 1 -J u n-2 0 2 2 
Pa ge 3 9 of 7 6
T he use of R W D s o urces has f urt her li mitati o ns. First, missi n g data a n d t he acc urac y  of 
rec or de d data on disease c haracteristics, la b res ults, a n d c o m o r bi dities i n R W D ma y 
i ntr o d uce a n i nf or mati o n bias a n d resi d ual c o nf o u n di n g. Sec o n d, patie nts wit h o ut s ufficie nt 
i nf or mati o n i n t heir E H Rs were e xcl u de d fr o m t he datasets by t he data pr o vi ders t o a v oi d 
i nf or mati o n bias. H o we ver, t his ma y i ntr o d uce a selecti o n bias. T hir d, t he varia bilit y i n 
p o p ulati o ns a n d differe nces i n t he pr o p orti o ns of a vaila ble val ues of c o variates ( n o n -missi n g 
val ues) ca n res ult i n s o me discre pa ncies i n t he esti mates usi n g differe nt R W s o urces.
T o a d dress a n d/ or re d uce t he i m pact of p ote ntial bias a n d i m pr o ve e xc ha n ge a bilit y bet wee n 
t he trial a n d t he e xter nal c o ntr ol ar ms2 7 ,5 8 ,5 9 meas ures will be ta ke n t o ali g n c o m para ble 
p o p ulat i o ns, ad v a nce d a dj ust me nt met h o ds will be e m pl o y e d, a n d a series of se nsiti vit y 
a nal ys es will be c o n d ucte d t o e val uate t he i m pact of ke y  ass u m pti o ns a n d selecti o n criteria. 
H o we ver, t his a nalys is is still s u bject t o u n meas ure d c o nf o u n di n g. Q ua ntitati ve bias a naly sis 
will atte m pt t o esti mate t he p ote ntial bias fr o m u n meas ure d c o nf o u n ders, b ut t hese esti mates 
are li kel y t o be c o nser vati ve a n d ma y n ullif y a tr ue treat me nt effect.
Partic i pati o n i n t he C 1 0 7 1 0 1 3 a n d C 1 0 7 1 0 1 4 pr os pecti ve o bser vati o nal st u dies was 
de pe n de nt o n p h y sicia ns’ a n d patie nts’ a bilit y a n d willi n g ness t o partici pate. He nce, t here is 
a p ossi bilit y t hat i ncl u de d p h ysicia ns are n ot f ully re prese ntati ve of t he c o m plete ta r gete d 
p h y sicia n p o p ulati o n, w hic h c o ul d i m pact patie nt re presentati ve ness. Si mila rly, res ults ma y 
n ot be tra nslata ble t o ot her ge o gra p hies wit h vastl y  differe nt healt hcare sys te ms a n d 
treat me nt a p pr oac hes. I n a d diti o n, t he availa bilit y  of i nf or mati o n i n rec or ds ma y var y b y 
p h y sicia n practice a n d ma y reflect differe nces i n practice patt erns, rec or di n g practices, a n d 
me dical n or ms. T hese areas pr o vi de o p p ort u nities f or f ut ure researc h.
9. 1 0. Ot her A s pects
N ot a p plica ble.
1 0. P R O T E C TI O N O F H U M A N S U B J E C T S
1 0. 1. P atie nt Inf or m ati o n
T his st u d y  i n v ol ves data t hat e xist i n a n a n o n ymize d str u ct ure d f or mat a n d c o ntai n n o patie nt 
pers o nal i nf or mati o n.
1 0. 2. P atie nt C o nse nt
As t his st u d y  i n v ol ves a n o n ymize d str uct ure d data, w hic h acc or di n g t o a p plica ble le gal 
re q uire me nts d o n ot c o ntai n data s u bject t o pri vac y la ws, o btai ni n g i nf or me d c o nse nt fr o m 
pat ie nts b y Pfizer is n ot re q uire d.
1 0. 3. P atie nt W it h dr a w al
N ot a p plica ble .
0 9 0 1 7 7 e 1 9 d 6 a 1 0 1 2\ A p pr o v e d\ A p pr o v e d O n: 1 5- M a y- 2 0 2 3 0 2: 3 3 ( G M T)

P F -0 6 8 6 3 1 3 5 ( Elra nata ma b)
N O N -I N T E R V E N TI O N A L S T U D Y P R O T O C O L
C 1 0 7 1 0 3 1 Versi o n 1. 0, 0 1 Ma y 2 0 2 3
P FI Z E R C O N FI D E N TI A L 
C T 2 4 -WI -G L 0 2 -R F 0 2 4. 0 N o n -I nter ve nti o nal St u d y Pr ot oc ol Te m plate F or Sec o n dar y Data C ollecti o n St u d y 
0 1 -J u n-2 0 2 2 
Pa ge 4 0 of 7 6
1 0. 4. I nstit uti o n al R e vie w B o ar d (I R B)/I n de pe n de nt Et hics C o m mittee (I E C)
A p pr o val fr o m a n i nstit uti o nal re vie w b oar d /in de pe n de nt et hics c o m mittee is n ot re q uire d f or 
t his st u d y as o nl y de-i de ntifie d sec o n dar y data s o urces a n d a n o n ymize d me dical rec or d data 
fr o m E H R will be use d. T heref ore, t his st u d y is c o nsi dere d e xe m pt fr o m t he re q uire me nts f or 
“ h u ma n s u bjects researc h”.
1 0. 5. Et hic al C o n d uct of t he S t ud y
T he st u d y  will be c o n d ucte d i n acc or da nce wit h le gal a n d re g ulator y  re q uire me nts, as well as 
wit h scie ntific p ur p ose, val ue, a n d ri g or , a n d f oll o w ge nerall y  acce pte d researc h practices 
descri be d i n t he G ui deli nes of G o o d P har mac oe pi de mi o l o g y Practices (G P P) iss ue d b y  t he
P u blic P olic y  C o m mittee, I nter nati o nal S ociety for P har mac oe pi de mi ol o g y (I S P E), G o o d 
Practices f or O utc o mes Researc h iss ue d b y t he I nter nati o nal S ociet y f or P har mac oec o n o mics 
a n d O utc o mes Researc h (IS P O R), I nter nati o nal E t hical G ui deli nes f or E pi de mi ol o gical 
st u dies iss ue d b y t he C o u ncil f or I nter nati o nal Or ga nizati o ns of Me dical Scie nces ( CI O M S). 
0 9 0 1 7 7 e 1 9 d 6 a 1 0 1 2\ A p pr o v e d\ A p pr o v e d O n: 1 5- M a y- 2 0 2 3 0 2: 3 3 ( G M T)

P F -0 6 8 6 3 1 3 5 ( Elra nata ma b)
N O N -I N T E R V E N TI O N A L S T U D Y P R O T O C O L
C 1 0 7 1 0 3 1 Versi o n 1. 0, 0 1 Ma y 2 0 2 3
P FI Z E R C O N FI D E N TI A L 
C T 2 4 -WI -G L 0 2 -R F 0 2 4. 0 N o n -I nter ve nti o nal St u d y Pr ot oc ol Te m plate F or Sec o n dar y Data C ollecti o n St u d y 
0 1 -J u n-2 0 2 2 
Pa ge 4 1 of 7 6
1 1. M A N A G E M E N T A N D R E P O R TI N G O F A D V E R S E E V E N T S/ A D V E R S E 
R E A C TI O N S
T his st u d y  is retr os pecti ve, it i n v ol ves data t hat e xist as str uct ure d data by t h e ti me of st u d y 
start.
I n t hese data s o urces, i n di vi d ual patient data are n ot retrie ve d or vali date d, a n d it is n ot 
p ossi ble t o li n k (ie, i de ntif y a p ote ntial ass ociati o n bet wee n) a partic ular pr o d uct a n d me dical 
e ve nt f or a n y i n di vi d ual. T h us, t he mi ni m u m criteria f or re p orti n g a n A E (ie, i de ntifia ble 
patie nt, i de nt ifia ble re p orter, a s us pect pr o d uct, a n d e ve nt) ca n n ot be met.
1 2. P L A N S F O R DI S S E MI N A TI N G A N D C O M M U NI C A TI N G S T U D Y R E S U L T S
F or all p u blicati o ns relati n g t o t he st u d y, Pfizer will c o m pl y  wit h rec o g nize d et hical 
sta n dar ds c o ncer ni n g p u blicati o ns a n d a ut h ors hi p, i ncl u di n g Secti o n II - “ Et hical 
C o nsi derati o ns i n t he C o n d uct a n d Re p orti n g of Researc h” of t he U nif or m Re q uire me nts f or 
Ma n uscri pts S u b mitte d t o Bi o me dical J o ur nals, htt p:// w w w.ic mje. or g/i n de x. ht ml #a ut h ors hi p, 
esta blis he d b y t he I nter nati o nal C o m mittee of Me dical J o ur nal E dit ors.
I n t he e ve nt of a ny pr o hi biti o n or restricti o n i m p ose d (e g, cli nical h ol d) b y  a n a p plica ble 
c o m pete nt aut h orit y i n a n y area of t he w orl d, or if t he i n vesti gat or part y res p o nsi ble f or 
c ollecti n g data fr o m t he partici pa nt is a ware of a n y ne w i nfor mati o n w hi c h mi g ht i nfl ue nce 
t he e val uati o n of t he be nefits a n d ris ks of a Pfizer pr o d uct , Pfizer s h o ul d be i nf or me d 
i m me diatel y.
0 9 0 1 7 7 e 1 9 d 6 a 1 0 1 2\ A p pr o v e d\ A p pr o v e d O n: 1 5- M a y- 2 0 2 3 0 2: 3 3 ( G M T)

P F -0 6 8 6 3 1 3 5 ( Elra nata ma b)
N O N -I N T E R V E N TI O N A L S T U D Y P R O T O C O L
C 1 0 7 1 0 3 1 Versi o n 1. 0, 0 1 Ma y 2 0 2 3
P FI Z E R C O N FI D E N TI A L 
C T 2 4 -WI -G L 0 2 -R F 0 2 4. 0 N o n -I nter ve nti o nal St u d y Pr ot oc ol Te m plate F or Sec o n dar y Data C ollecti o n St u d y 
0 1 -J u n-2 0 2 2 
Pa ge 4 2 of 7 6
1 3. R E F E R E N C E S
1.
Pa dala S A, Bars o u k A, Bars o u k A, Ra wla P, Va kiti A, K ol he R, et al. 
E pi de mi ol o g y , Sta gi n g, a n d Ma nage me nt of M ul ti ple M yel o ma. Me d Sci ( Basel) 
[Inter net]. 2 0 2 1 Ja n 2 0 [cite d 2 0 2 2 Ma y  2 4]; 9( 1): 3. A vaila ble fr o m: 
htt ps:// p u b me d. nc bi. nl m. ni h. g o v/ 3 3 4 9 8 3 5 6/.
2.
Walli n gt o n -Be d d oe C T, P its o n S M. N o vel t hera pies f or m ulti ple m yel o ma. 
A gi n g [I nter net]. 2 0 1 7 [cite d 2 0 2 2 Ma y  2 4];9( 8): 1 8 5 7 – 8. A vaila ble fr o m: 
htt ps:// p u b me d. nc bi. nl m. ni h. g o v/ 2 8 8 5 4 1 4 7/ .
3.
N o o ka A K, Kastritis E, Di m o p o ul os M A, L o nial S. Treat me nt o pti o ns f or 
rela pse d a n d refract or y m ulti ple m y el o ma. Bl o o d [Inter net]. 2 0 1 5 Ma y  1 4 [cite d 
2 0 2 2 A pr 2 4]; 1 2 5( 2 0): 3 0 8 5 – 9 9. A vail a ble fr o m: 
htt ps:// p u b me d. nc bi. nl m. ni h. g o v/ 2 5 8 3 8 3 4 2/ .
4.
Y o n g K, Delf or ge M, Driesse n C, Fi n k L, Fli n ois A, G o nzalez - Mc Q uire S, et al. 
M ulti ple m y el o ma: patie nt o utc o me s i n real-w orl d practice. Br J Hae mat ol 
[Inter net]. 2 0 1 6 Oct 1 [cite d 2 0 2 2 A pr 2 4]; 1 7 5( 2): 2 5 2 – 6 4. A vaila ble fr o m: 
htt ps:// p u b me d. nc bi. nl m. ni h. g o v/ 2 7 4 1 1 0 2 2/.
5.
C osta L J, C or nell R F, Calla n der N S, C h ha bra S, Lie dt ke M, Ka nsa gra A, et al. 
Efficac y  of Treat me nts f or Patie nts wit h Tri ple-Class Refract or y  ( T C R) M ulti ple 
M y el o ma ( M M): Be nc h m ar k f or Ne w A ge nts Utilizi n g Real- W orl d Data ( R W D). 
Bl o o d [I nter net]. 2 0 2 1 N o v 2 3 [cite d 2 0 2 3 Ja n 3 0]; 1 3 8( S u p ple me nt 1): 3 7 8 6 –
3 7 8 6. A vaila ble fr o m: htt ps://as h p u blicati o ns. or g/ bl o o d/article/ 1 3 8/ S u p ple me nt 
1/ 3 7 8 6/ 4 8 2 5 8 2/ Efficac y - of-Treat me nts -f or-Patie nts -wi t h-Tri ple .
6.
Wa n g F, G ors h B, Der Sar kissia n M, Pa ka P, B ha k R, B o yts o v N, et al. Treat me nt 
Patter ns a n d O utc o mes of Patie nts wit h D o u ble- Class Refract or y  or Tri ple-Class 
Refract or y
M ulti ple M y el o ma: A Retr os pecti ve U S Electr o nic Healt h Rec or d 
Data base St u d y. Bl o o d. 2 0 2 1 N o v 5; 1 3 8( S u p ple me nt 1): 2 7 0 5 – 2 7 0 5. 
7.
F D A gra nte d accelerate d a p pr o val t o bela nta ma b maf o d oti n -bl mf f or m ulti ple 
m y el o ma | F D A [I nter net]. [cite d 2 0 2 2 N o v 2 8]. A vaila b le fr o m: 
htt ps:// w w w.f da. g o v/ dr u gs/res o urces -i nf or mati o n-a p pr o ve d -dr u gs/f da -gr a nte d-
accelerate d-a p pr o val -bela nta ma b -maf o d oti n -bl mf -m ulti ple -m y el o ma.
8.  
G S K pr o vi des a n u p date o n Ble nre p ( bela nta ma b maf o d oti n- bl mf) U S mar keti n g 
a ut h orisati o n | G S K [I nter net]. [cite d 2 0 2 3 Mar 2 7]. A vaila ble fr o m: 
htt ps:// w w w. gs k.c o m/e n -g b/ me dia/ press -releases/ gs k -pr o vi des -u p date - o n-
ble nre p -us- mar keti n g -a ut h orisatio n/.
0 9 0 1 7 7 e 1 9 d 6 a 1 0 1 2\ A p pr o v e d\ A p pr o v e d O n: 1 5- M a y- 2 0 2 3 0 2: 3 3 ( G M T)

P F -0 6 8 6 3 1 3 5 ( Elra nata ma b)
N O N -I N T E R V E N TI O N A L S T U D Y P R O T O C O L
C 1 0 7 1 0 3 1 Versi o n 1. 0, 0 1 Ma y 2 0 2 3
P FI Z E R C O N FI D E N TI A L 
C T 2 4 -WI -G L 0 2 -R F 0 2 4. 0 N o n -I nter ve nti o nal St u d y Pr ot oc ol Te m plate F or Sec o n dar y Data C ollecti o n St u d y 
0 1 -J u n-2 0 2 2 
Pa ge 4 3 of 7 6
9.
F D A a p pr o ves teclista ma b -c q y v f or rela pse d or refract or y m ulti ple m yel o ma |  
F D A [I nter net]. [cite d 2 0 2 2 N o v 2 8]. A vaila ble fr o m: 
htt ps:// w w w.f da. g o v/ dr u gs/res o urces -i nf or mati o n-a p pr o ve d -dr u g s/f da-a p pr o ves-
teclista ma b-c q y v -rela pse d- or-refract or y-m ulti p le-m y el o ma.
1 0.
F D A a p pr o ves ciltaca bta ge ne a ut ole ucel f or rela pse d or refract or y  m ulti ple 
m y el o ma | F D A [I nter net]. [cit e d 2 0 2 2 N o v 2 8]. A vaila ble fr o m: 
htt ps:// w w w.f da. g o v/ dr u gs/res o urces -i nf or matio n- a p pr o ve d - dr u gs/f da-a p pr o ves -
ciltaca bta ge ne-a ut ole ucel -rela pse d-or- refract or y-m ulti ple -m y el o ma.
1 1.
F D A a p pr o ves i deca bta ge ne vicle ucel f or m ulti ple m y el o ma | F D A [Inter net]. 
[cited 2 0 2 2 N o v 2 8]. A vaila ble fr o m: h tt ps:// w w w.f da. g o v/ dr u gs/res o urces-
i nf or mati on- a p pr o ve d - dru gs/f da -a p pr o ves -i deca bta ge ne-vicle ucel -m ulti ple -
m y el o ma.
1 2.
Se ba g M, Raje N S, Ba hlis NJ, C ostell o C, D h olaria B, S ol h M, et al. 
Elra nata ma b ( P F -0 6 8 6 3 1 3 5), a B - Cell Mat urati o n A nti ge n ( B C M A) Tar gete d 
C D 3 - E n ga gi n g Bis pecific M olec ule, f or Patie nts wit h Rela psed or Refract or y  
M ulti ple M y el o ma: Res ults fr o m Ma g netis m m -1 [I nter net]. 2 0 2 2 [cite d 2 0 2 2 A pr 
2 4]. A vaila ble fr o m: 
htt ps://as h p u blicati o ns. or g/ bl o o d/article/ 1 3 8/ S u p pl e me nt % 2 0 1/ 8 9 5/ 4 8 2 6 2 7/ Elra n
ata ma b-P F - 0 6 8 6 3 1 3 5-a- B-Cell -Mat urati o n .
1 3.
Pfizer I niti ates Pi v otal P hase 2 Ma g netis M M - 3 Trial of B C M A-C D 3 Bis pecific 
A nti b o d y  Elra nata ma b ( P F-0 6 8 6 3 1 3 5) i n M ulti ple M y el o ma | Pfizer [I nter net]. 
2 0 2 2 [cite d 2 0 2 2 Ma y  2 4]. A vaila ble fr om: htt ps:// w w w. pfizer.c o m/ ne ws/ pr ess-
release/ press-release-detail/ pfizer -i nitiates- pi v otal-p hase - 2-ma g netis m m - 3-trial-
bc ma .
1 4.
Pfizer Prese nts U p date d Fa v ora ble Elra nata ma b Data fr o m Pi v otal P hase 2 
Ma g netis M M -3 Trial | Pfizer [I nter net]. [cite d 2 0 2 3 Mar 2 3]. A vaila ble fr o m: 
htt ps:// w w w. pfiz er.c o m/ ne ws/ press -release/ press-release-detai l/ pfizer-pres e nts-
u p date d -fa v ora ble-elra nata ma b -data - pi v otal.
1 5.
F D A Gra nts Brea kt hr o u g h T hera p y Desi g nati o n t o Elra nata ma b f or 
Rela pse d/ Refract or y  M ulti ple M yel o ma [I nter net]. [cit e d 2 0 2 2 N o v 1 5]. 
A vaila ble fr o m: htt ps:// w w w. o ncli ve.c o m/ vie w/f da -gra nts -brea k t hr o u g h-t herap y -
desi g nati o n -t o-elra nata ma b -f or-rela pse d-refract or y-m ulti ple -m y el o ma.
1 6.
Pfizer Prese nts First Data fr o m Pla n ne d I nteri m A nal y sis of Pi v otal P hase 2 
Ma g netis M M -3 Tria l of B C M A-C D 3 Bis pecific A nti b o d y Elra nata ma b U n der 
I n vesti gati o n f or Rela pse d/ Refract or y Multi ple M y el o ma | Pfizer [I nter net]. 2 0 2 2 
[cite d 2 0 2 2 J u n 2 6]. A vaila ble fr o m: htt ps:// w w w. pfizer.c o m/ ne ws/ press-
release/ press-release-detail/ pfizer -prese nts -first- data-pla n ne d -i nteri m-a nal ysis -
pi v o tal.
0 9 0 1 7 7 e 1 9 d 6 a 1 0 1 2\ A p pr o v e d\ A p pr o v e d O n: 1 5- M a y- 2 0 2 3 0 2: 3 3 ( G M T)

P F -0 6 8 6 3 1 3 5 ( Elra nata ma b)
N O N -I N T E R V E N TI O N A L S T U D Y P R O T O C O L
C 1 0 7 1 0 3 1 Versi o n 1. 0, 0 1 Ma y 2 0 2 3
P FI Z E R C O N FI D E N TI A L 
C T 2 4 -WI -G L 0 2 -R F 0 2 4. 0 N o n -I nter ve nti o nal St u d y Pr ot oc ol Te m plate F or Sec o n dar y Data C ollecti o n St u d y 
0 1 -J u n-2 0 2 2 
Pa ge 4 4 of 7 6
1 7.
Ga n d hi U H, C or nell R F, La ks h ma n A, Ga h va ri ZJ, Mc Ge hee E, Ja g os k y M H, et 
al. O utc o mes of patie nts wit h m ulti ple m y el o ma refract or y t o C D 3 8-tar gete d 
m o n ocl o nal a nti b o d y  t hera p y. Le u ke mia. 2 0 1 9 Se p 1; 3 3( 9): 2 2 6 6– 7 5. 
1 8.
Ja gan nat h S, L i n Y, G ol dsc h mi dt H, Reece D E, N o o ka A K, R o drí g uez Oter o P, 
et al. Kar M Ma-R W: A s t u d y of real-w orl d treat me nt patter ns i n hea vil y 
pretreate d patie nts wit h rela pse d a n d refract or y  m ulti ple m yel o ma ( R R M M) a n d 
c o m paris o n of o utc o mes t o Kar M Ma. J o ur n al of Cli nical O nc ol o g y. 2 0 2 0 Ma y
2 0; 3 8( 1 5 _s u p pl): 8 5 2 5 – 8 5 2 5. 
1 9.
I C H E 1 0 C h oice of c o ntr ol gr ou p i n cli nical trials | E ur o pea n Me dici nes A ge nc y  
[Inter net]. 2 0 0 1 [cite d 2 0 2 2 Ma y  2 4]. A vaila ble fr o m: 
htt ps:// w w w.e ma.e ur o pa.e u/e n/ic h -e 1 0 -c h oice -c o ntr ol-gr o u p -clinical -trials.
2 0.
Rare Diseases: Nat u ral Hist or y St u dies f or Dr u g De vel o p me nt | F DA [I nter net].
[cite d 2 0 2 2 Ma y 2 4]. A vaila ble fr o m: htt ps:// w w w.f da. g o v/re g ulat or y-
i nf or mati o n/searc h-f da-g ui da nce -d oc u me nts/rare -diseases -nat ural -hist or y -
st u dies-dr u g -de vel o p me nt .
2 1.
Fra me w or k f or F D A’s Real -W orl d E vi de nce Pr o gra m. 2 0 1 8 [cite d 2 0 2 2 Ma y  
2 4]; A vaila ble fr o m: w w w.f da. g o v .
2 2.
Ja ha ns ha hi M, Gre g g K, Da vis G, N d u A, Miller V, V oc kle y J, et al. T he Use of 
E xter nal C o ntr ols i n F D A Re g ulat or y  Decisi o n Ma ki n g. T her I n n o v Re g ul Sci 
[Inter net]. 2 0 2 1 Se p 1 [cite d 2 0 2 2 A pr 2 4]; 5 5( 5): 1 0 1 9 – 3 5. A vaila ble fr o m: 
ht t ps:// p u b me d.nc bi. nl m. ni h. g o v/ 3 4 0 1 4 4 3 9/ .
2 3.
C o m mittee f or Me dici nal Pr o d ucts f or H u ma n Use ( C H M P) g ui deli ne o n cli nical 
trials i n s mall p o p ulati o ns draft a gree d by efficac y w or ki n g part y/ a d h oc gr o u p 
o n cli nical tr ials i n s mall p o p ulati o ns a d o pti o n b y C H M P f or release f or
c o ns ultati o n. 2 0 0 2 [cite d 2 0 2 2 A pr 2 4]; A vaila ble fr o m: htt p:// w w w.e mea.e u.i nt .
2 4.
A n ders o n M, Naci H, M orris o n D, Osi pe n k o L, M ossial os E. A re vie w of NI C E 
a p praisals of p har mace uticals 2 0 0 0 -2 0 1 6 f o u n d variati o n i n esta blis hi n g 
c o m parati ve cli n ical effecti ve ness. J Cli n E pi de mi ol [Inter net]. 2 0 1 9 Ja n 1 [cite d 
2 0 2 3 Fe b 5]; 1 0 5: 5 0 – 9. A vaila ble fr o m: 
htt ps:// p u b me d. nc bi. nl m. ni h. g o v/ 3 0 2 3 6 4 8 4/ .
2 5.
Fra me w or k f or F D A’s Real -W orl d
E vi de nce Pr o gra m. 2 0 1 8 [cite d 2 0 2 2 J u n 2 9]; 
A vaila ble fr o m: w w w.f da. g o v .
2 6.
Carr oll N. A p plic ati o n of Pr o pe nsit y Sc ore M o dels i n O bser vati o nal St u dies. 
S A S Gl o b F or u m. 2 0 1 8; Pa per 2 5 2 2 - 2 0 1 8. 
0 9 0 1 7 7 e 1 9 d 6 a 1 0 1 2\ A p pr o v e d\ A p pr o v e d O n: 1 5- M a y- 2 0 2 3 0 2: 3 3 ( G M T)

P F -0 6 8 6 3 1 3 5 ( Elra nata ma b)
N O N -I N T E R V E N TI O N A L S T U D Y P R O T O C O L
C 1 0 7 1 0 3 1 Versi o n 1. 0, 0 1 Ma y 2 0 2 3
P FI Z E R C O N FI D E N TI A L 
C T 2 4 -WI -G L 0 2 -R F 0 2 4. 0 N o n -I nter ve nti o nal St u d y Pr ot oc ol Te m plate F or Sec o n dar y Data C ollecti o n St u d y 
0 1 -J u n-2 0 2 2 
Pa ge 4 5 of 7 6
2 7.
G ha dessi M, Ta n g R, Z h o u J, L i u R, Wa n g C, T o yoiz u mi K, et al. A r o a d ma p t o 
usi n g hist orica l c o ntr ols i n cli nical trials - B y  Dr u g I nf or mati o n Ass ociati o n 
A da pti ve Desi g n Scie ntific W or ki n g Gr o u p ( DI A- A D S W G). Or p ha net J Rare 
Dis. 2 0 2 0 Mar 1 2; 1 5( 1). 
2 8.
C hes na y e N C, Stel V S, Tri pe pi G, De k ker F W, F u E L , Z occali C, et al. A n 
intr o d ucti o n t o i n verse p r o ba bilit y of treat me nt wei g hti n g i n o bser vati o nal 
researc h. Cli n K i d ne y J. 2 0 2 2 Ja n 1; 1 5( 1): 1 4– 2 0. 
2 9.
Mi k hael J. Treat me nt O pti o ns f or Tri ple -class Refract or y M ulti ple Myel o ma. 
Cli n L y m p h o ma M yel o ma Le u k [I nter net]. 2 0 2 0 Ja n 1 [cite d 2 0 2 2 Ma y  
2 4]; 2 0( 1): 1 – 7. A vaila ble fr o m: htt ps:// p u b me d. nc bi. nl m. ni h. g o v/ 3 1 7 6 7 5 2 9/.
3 0.
Pfizer. Ma g netis M M -3, a n o pe n -la bel, m ultice nter, n o n-ra n d o mize d p hase 2 
st u d y of elra nata ma b ( pf-0 6 8 6 3 1 3 5) m o n ot hera p y i n partici pa nts wit h M M w h o 
are refract or y t o at least o ne pr oteas o me i n hi bit or ( PI), o ne i m m u n o m o d ulat or y 
dr u g, a n d o ne a nti -C D 3 8 a nti b o d y. Final Pr ot oc ol A me n d me nt 7, 1 1 N o ve m ber 
2 0 2 1. 2 0 2 1. 
3 1.
Flatir o n Healt h I nc. A nal ytic g ui de f or Flatir o n Healt h Data. Tri ple -class 
refract or y m ulti ple m yel o ma. Versi o n 1. 2 0 2 1. 
3 2.
C O T A. M ulti ple m yel o ma real -w orl d e vi de nce. Data ma n ual. 2 0 2 2. 
3 3.
D ola n P. M o deli n g v al uati o ns f or E ur o Q ol healt h states. Me d Care [I nter net]. 
1 9 9 7 [cite d 2 0 2 3 Mar 6]; 3 5( 1 1): 1 0 9 5 – 1 0 8. A vaila ble fr o m: 
htt ps:// p u b me d. nc bi. nl m. ni h. g o v/ 9 3 6 6 8 8 9/ .
3 4.
B u ure n S va n, Gr o ot h uis -O u ds h o or n K. M ulti variate i m p utati o n b y  c hai ne d 
e q uati o ns i n R . J Stat S oftw. 2 0 1 1; 4 5( 3): 1 – 6 7. 
3 5.
va n B u ure n S. M ulti ple i m p utati o n of discrete a n d c o nti n u o us data b y  f ull y 
c o n diti o nal s pecificati o n. Stat Met h o ds Me d Res. 2 0 0 7 J u n 2; 1 6( 3): 2 1 9 – 4 2. 
3 6.
va n B u ure n S. Fle x i ble i m p utati o n of missi n g data. B oca Rat o n, Fl ori da: 
C ha p ma n & Hal l/ C R C; 2 0 1 2. 
3 7.
Peterse n M L, P orter K E, Gr u ber S, Wa n g Y, va n der Laa n MJ. Dia g n osi n g a n d 
res p o n di n g t o vi olati o ns i n t he p ositi vit y ass u m pti o n. 
3 8.
R u bi n D B. Usi n g Pr o pe nsit y  Sc ores t o Hel p Desi g n O bser vati o nal St u dies: 
A p plicati o n t o t he T o bacc o Li ti gati o n. Healt h Ser vices a n d O utc o mes Researc h 
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0 9 0 1 7 7 e 1 9 d 6 a 1 0 1 2\ A p pr o v e d\ A p pr o v e d O n: 1 5- M a y- 2 0 2 3 0 2: 3 3 ( G M T)

P F -0 6 8 6 3 1 3 5 ( Elra nata ma b)
N O N -I N T E R V E N TI O N A L S T U D Y P R O T O C O L
C 1 0 7 1 0 3 1 Versi o n 1. 0, 0 1 Ma y 2 0 2 3
P FI Z E R C O N FI D E N TI A L 
C T 2 4 -WI -G L 0 2 -R F 0 2 4. 0 N o n -I nter ve nti o nal St u d y Pr ot oc ol Te m plate F or Sec o n dar y Data C ollecti o n St u d y 
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4 3.
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Oct 3 0 [cite d 2 0 2 3 A pr 1 7]; 2 4( 2 0): 3 0 8 9 – 1 1 0. A vaila ble fr o m: 
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Vali dati o n of Rati o- base d C o n diti o nal A vera ge Tr eat me nt Effects Usi n g 
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5 0.
F u n k MJ, Westreic h D, Wiese n C, St ür mer T, Br o o k hart M A, Da vi dia n M. 
D o u bl y  R o b ust Esti mati o n of Ca usal Effects. A m J E pi de mi ol. 2 0 1 1 Apr 
1 ; 1 7 3( 7): 7 6 1– 7. 
0 9 0 1 7 7 e 1 9 d 6 a 1 0 1 2\ A p pr o v e d\ A p pr o v e d O n: 1 5- M a y- 2 0 2 3 0 2: 3 3 ( G M T)

P F -0 6 8 6 3 1 3 5 ( Elra nata ma b)
N O N -I N T E R V E N TI O N A L S T U D Y P R O T O C O L
C 1 0 7 1 0 3 1 Versi o n 1. 0, 0 1 Ma y 2 0 2 3
P FI Z E R C O N FI D E N TI A L 
C T 2 4 -WI -G L 0 2 -R F 0 2 4. 0 N o n -I nter ve nti o nal St u d y Pr ot oc ol Te m plate F or Sec o n dar y Data C ollecti o n St u d y 
0 1 -J u n-2 0 2 2 
Pa ge 4 7 of 7 6
5 1.
Ba n g H, R o bi ns J M. D o u bl y  R o b ust Esti mati o n i n Missi n g Data a n d Ca usal 
I nfere nce M o dels. Bi o metrics. 2 0 0 5 Dec; 6 1( 4): 9 6 2 –7 3. 
5 2.
A usti n P C, St uart E A. M o vi n g t o war ds best practice w he n usi n g i n verse 
pr o ba bilit y  of treat me nt wei g hti n g (IP T W) usi n g t he pr o pe nsit y  sc ore t o esti mate 
ca usal treat me nt effects i n o bser vati o nal st u dies. Stat Me d. 2 0 1 5 Dec 
1 0; 3 4( 2 8): 3 6 6 1 – 7 9. 
5 3.
va n der Weele TJ, Di n g P. Se nsiti vit y  A nalys is i n O bser vati o nal Researc h: 
I ntr o d uci n g t he E-Val ue. A n n I nter n Me d [I nter net]. 2 0 1 7 A u g 1 5 [cite d 2 0 2 2 
Ma y  2 5]; 1 6 7( 4): 2 6 8– 7 4. A vaila ble fr o m: 
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5 4.
va n der Weele TJ, Di n g P. Se nsiti vit y  A nalys is i n O bser vati o nal Researc h: 
I ntr o d uci n g t he E-Val ue. A n n I nter n Me d [I nter net]. 2 0 1 7 A u g 1 5 [cite d 2 0 2 2 
Ma y  3 0]; 1 6 7( 4): 2 6 8 – 7 4. A vaila ble fr o m: 
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5 6.
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Pr o gressi o n wit h cli nical feat ures is ass ociate d w it h w orse s u bse q ue nt s ur vi val i n 
m ulti ple m y el o ma. A m J He mat ol [Inter net]. 2 0 1 9 A pr 1 [cit e d 2 0 2 3 Mar 
2 0]; 9 4( 4): 4 3 9 – 4 5. A vaila ble fr o m: htt ps:// p u b me d. nc bi. nl m. ni h. g o v/ 3 0 6 6 3 8 0 5/ .
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al. O utc o mes after bi oc he mical or cli nical pr o gressi o n i n patie nts wit h m ulti ple
m y el o ma. Bl oo d A d v [I nter net]. 2 0 2 3 Mar 2 8 [cite d 2 0 2 3 Mar 2 0]; 7( 6): 9 0 9 – 1 7. 
A vaila ble fr o m: 
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after-bi oc h e mical-or -cli nical-pr o gressi o n .
5 8.
P oc oc k SJ. T he c o m bi nati o n of ra n d o mize d a n d h ist orical c o ntr ols i n cli nical 
trials. J C hr o nic Dis [Inter net]. 1 9 7 6 [cite d 2 0 2 2 Ma y 2 4]; 2 9( 3): 1 7 5 – 8 8. 
A vaila ble fr o m: htt ps:// p u b me d. nc bi. nl m. ni h. g o v/ 7 7 0 4 9 3/ .
5 9.
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salie nt i nf or mati o n o n hist orical c o ntr ols: A str uct ure d assess me nt of vali dit y  a n d 
c o m para bilit y acr oss st u dies. Cli nical Trials. 2 0 2 0 Dec 1; 1 7( 6): 6 0 7– 1 6.
0 9 0 1 7 7 e 1 9 d 6 a 1 0 1 2\ A p pr o v e d\ A p pr o v e d O n: 1 5- M a y- 2 0 2 3 0 2: 3 3 ( G M T)

P F -0 6 8 6 3 1 3 5 ( Elra nata ma b)
N O N -I N T E R V E N TI O N A L S T U D Y P R O T O C O L
C 1 0 7 1 0 3 1 Versi o n 1. 0, 0 1 Ma y 2 0 2 3
P FI Z E R C O N FI D E N TI A L 
C T 2 4 -WI -G L 0 2 -R F 0 2 4. 0 N o n -I nter ve nti o nal St u d y Pr ot oc ol Te m plate F or Sec o n dar y Data C ollecti o n St u d y 
0 1 -J u n-2 0 2 2 
Pa ge 4 8 of 7 6
1 4. LI S T O F T A B L E S
Ta ble 1. I ncl usi o n a n d E xcl usi o n Criteria f or St u d y C 1 0 7 1 0 0 3 a n d R W Data S o urces. ........ 2 2
Ta ble 2. Defi niti o ns of C o m parati ve Effecti ve ness O utc o mes i n St u d y  C 1 0 7 1 00 3, 
Flatir o n Healt h, a n d C O T A ...................................................................... 2 5
Ta ble 3. S u m mar y  of Statistical A nal yses f or C o m bi ne d St u d y C o h orts................................ 3 1
0 9 0 1 7 7 e 1 9 d 6 a 1 0 1 2\ A p pr o v e d\ A p pr o v e d O n: 1 5- M a y- 2 0 2 3 0 2: 3 3 ( G M T)

P F -0 6 8 6 3 1 3 5 ( Elra nata ma b)
N O N -I N T E R V E N TI O N A L S T U D Y P R O T O C O L
C 1 0 7 1 0 3 1 Versi o n 1. 0, 0 1 Ma y 2 0 2 3
P FI Z E R C O N FI D E N TI A L 
C T 2 4 -WI -G L 0 2 -R F 0 2 4. 0 N o n -I nter ve nti o nal St u d y Pr ot oc ol Te m plate F or Sec o n dar y Data C ollecti o n St u d y 
0 1 -J u n-2 0 2 2 
Pa ge 4 9 of 7 6
1 5. L I S T O F FI G U R E S
Fi g ure 1. Baseli ne a n d O bser vati o n Peri o ds i n St u d y  C 1 0 7 1 0 0 3............................................ 1 7
Fi g ure 2. Baseli ne a n d O bser vati o n Peri o ds i n E xter nal C o ntr ol Ar ms .................................. 1 7
0 9 0 1 7 7 e 1 9 d 6 a 1 0 1 2\ A p pr o v e d\ A p pr o v e d O n: 1 5- M a y- 2 0 2 3 0 2: 3 3 ( G M T)

P F -0 6 8 6 3 1 3 5 ( Elra nata ma b)
N O N -I N T E R V E N TI O N A L S T U D Y P R O T O C O L
C 1 0 7 1 0 3 1 Versi o n 1. 0, 0 1 Ma y 2 0 2 3
P FI Z E R C O N FI D E N TI A L 
C T 2 4 -WI -G L 0 2 -R F 0 2 4. 0 N o n -I nter ve nti o nal St u d y Pr ot oc ol Te m plate F or Sec o n dar y Data C ollecti o n St u d y 
0 1 -J u n-2 0 2 2 
Pa ge 5 0 of 7 6
A N N E X 1. L I S T O F S T A N D A L O N E DO C U M E N T S
N u m ber
D oc u me nt 
Refere nce 
N u m ber
D ate
Ti tle
0 0 1 
0 0 1 
1 1 Mar c h 2 0 1 9
0 0 2 
0 0 2 
1 7 Ma y
2 0 2 2
0 0 3 
0 0 3 
2 5 Oct o ber 2 0 2 1
0 0 4 
0 0 4 
1 8 Oct o b er 2 0 2 1
0 9 0 1 7 7 e 1 9 d 6 a 1 0 1 2\ A p pr o v e d\ A p pr o v e d O n: 1 5- M a y- 2 0 2 3 0 2: 3 3 ( G M T)
C CI

P F -0 6 8 6 3 1 3 5 ( Elra nata ma b)
N O N -I N T E R V E N TI O N A L S T U D Y P R O T O C O L
C 1 0 7 1 0 3 1 Versi o n 1. 0, 0 1 Ma y 2 0 2 3
P FI Z E R C O N FI D E N TI A L 
C T 2 4 -WI -G L 0 2 -R F 0 2 4. 0 N o n -I nter ve nti o nal St u d y Pr ot oc ol Te m plate F or Sec o n dar y Data C ollecti o n St u d y 
0 1 -J u n-2 0 2 2 
Pa ge 5 1 of 7 6
A N N E X 2 . M A P PI N G O F E LI GI BILI T Y C RI T E RI A B E T W E E N S T U D Y C 1 0 7 1 0 0 3
A N D R W D S O U R C E S
0 9 0 1 7 7 e 1 9 d 6 a 1 0 1 2\ A p pr o v e d\ A p pr o v e d O n: 1 5- M a y- 2 0 2 3 0 2: 3 3 ( G M T)

P F -0 6 8 6 3 1 3 5 ( Elra nata ma b)
N O N -I N T E R V E N TI O N A L S T U D Y P R O T O C O L
C 1 0 7 1 0 3 1 Versi o n 1. 0, 0 1 Ma y 2 0 2 3
P FI Z E R C O N FI D E N TI A L 
C T 2 4 -WI -G L 0 2 -R F 0 2 4. 0 N o n -I nter ve nti o nal St u d y Pr ot oc ol Te m plate F or Sec o n dar y Data C ollecti o n St u d y 
0 1 -J u n-2 0 2 2 
Pa ge 5 2 of 7 6
S u p ple me nt ar y T a ble 1 5. 1. I ncl usi o n a n d E xcl usi o n Criteri a i n St u d y C 1 0 7 1 0 0 3, Fl atir o n He alt h a n d C O T A.
P atie nt i ncl us i o n criteri a per 
C 1 0 7 1 0 0 3 St u d y  
Fl atir o n S p otli g ht R W al ter n ati ve 
a p pr o ac h t o be use d i n t he e xter n al 
c o ntr ol ar m
I m ple me nt ati o n
C O T A R W alter n ati ve
a p pr o ac h t o be use d i n t he 
e xter n al c o ntr ol ar m
I m ple me nt ati o n
A ge a n d Se x:
Male or fe male partici pa nts a ge ≥1 8 
years.
Male or fe male partici pa nts a ge ≥1 8 
years.
Critical a n d 
e x pa n de d 
criteria
Male or fe male partici pa nts a ge 
≥1 8 years.
Critical a n d 
e x pa n de d 
criteria
A fe male partici pa nt is eli gi ble t o 
partici pate if s he is n ot pre g na nt or 
breas tfee di n g. 
N ot a vaila ble .
N/ A
N ot a vaila bl e.
N/ A
T y pe of P artici p a nt a n d Dise ase C h ar acteristics:
Partici pa nts w h o are willi n g a n d a ble 
t o c o m pl y wit h all sc he d ule d visits, 
treat me nt pla n, la b orat or y tests, 
lifest yle c o nsi derati o ns, a n d ot her 
st ud y  pr oce d ures.
N ot a p plica ble .
N/ A
N ot a p plica ble .
N/ A
Pri or dia g n osis of M M as defi ne d 
acc or di n g t o I M W G criteria .
Perfect or cl ose matc h .
Patie nts were e xtracte d b y Flatir o n base d 
o n t he prese nce of a dia g n osis c o de f or 
M M (I C D -9 2 0 3. 0 x or I C D -1 0 C 9 0. 0 x) 
a n y ti me pri or t o t he i n de x date. Acti ve 
M M dia g n osis was c o nfir me d b y 
Flatir o n via a bstracti o n of u n str uct ure d 
data .
Critical a n d 
e x pa n de d 
criteria
Perfect or cl ose matc h .
M M dia g n osis c o nfir me d 
t hr o u g h pat h ol o g y re p orts or 
t hr o u g h cli nical dia g n osis a n d 
s u p p orti n g d oc u me ntati o n s uc h 
as la b tests.
Critical a n d 
e x pa n de d 
criteria
0 9 0 1 7 7 e 1 9 d 6 a 1 0 1 2\ A p pr o v e d\ A p pr o v e d O n: 1 5- M a y- 2 0 2 3 0 2: 3 3 ( G M T)

P F -0 6 8 6 3 1 3 5 ( Elra nata ma b)
N O N -I N T E R V E N TI O N A L S T U D Y P R O T O C O L
C 1 0 7 1 0 3 1 Versi o n 1. 0, 0 1 Ma y 2 0 2 3
P FI Z E R C O N FI D E N TI A L 
C T 2 4 -WI -G L 0 2 -R F 0 2 4. 0 N o n -I nter ve nti o nal St u d y Pr ot oc ol Te m plate F or Sec o n dar y Data C ollecti o n St u d y 
0 1 -J u n-2 0 2 2 
Pa ge 5 3 of 7 6
P atie nt i ncl us i o n criteri a per 
C 1 0 7 1 0 0 3 St u d y  
Fl atir o n S p otli g ht R W al ter n ati ve 
a p pr o ac h t o be use d i n t he e xter n al 
c o ntr ol ar m
I m ple me nt ati o n
C O T A R W alter n ati ve
a p pr o ac h t o be use d i n t he 
e xter n al c o ntr ol ar m
I m ple me nt ati o n
Meas ura ble disease base d o n I M W G 
criteria as defi ne d b y at least 1 of t he 
f oll o wi n g:
a. Ser u m M -pr otei n ≥0. 5 g/ d L b y 
S P E P
b. Uri nar y M -pr otei n e xcreti o n ≥2 0 0 
m g/ 2 4 h o urs b y U P E P
c. Ser u m i m m u n o gl o b uli n F L C ≥1 0 
m g/ d L ( ≥1 0 0 m g/ L) A N D a b n o r mal 
ser u m i m m u n o gl o b uli n ka p pa t o 
l a mb da F L C rati o ( < 0. 2 6 or > 1. 6 5)
Perfect or cl ose matc h.
Meas ura ble disease, defi ne d
wit hi n 9 0 
da ys pri or t o or o n t he i n de x date.
Critical a n d 
e x pa n de d 
criteria
Perfect or cl ose matc h.
Meas ura ble disease, defi ne d 
wit hi n 9 0 da ys bef ore or o n t he 
i n de x date.
Cr itical a n d 
e x pa n de d 
criteria
Refract or y t o at least o ne I Mi D.
Perfect or cl ose matc h. 
Refract or y t o at least o ne I Mi D defi ne d 
as disease pr o gressi o n, base d o n I M W G 
criteria or H C P assess me nt, w hile a 
patie nt is o n t hera p y or wit hi n 6 0 da ys of 
t he last thera p y d ose i n a n y li ne, 
re gar dless of res p o nse. 
Critical a n d 
e x pa n de d 
criteria
Perfect or cl ose matc h. 
Refract or y t o at least o ne I Mi D 
defi ne d as disease pr o gressi o n, 
base d o n I M W G criteria or H C P 
assess me nt, w hile a patie nt is o n 
t hera p y or wit hi n 6 0 da ys of t he 
last t hera p y d ose i n a n y li ne, 
re gar dless of res p o nse.
Critical a n d 
e x pa n de d 
criteria
Refract or y t o at least o ne PI.
Perfect or cl ose matc h. 
Refract or y t o at least o ne PI defi ne d as 
disease pr o gressi o n, base d o n I M W G 
criteria or H C P assess me nt, w hile a 
patie nt is o n t hera p y or wit hi n 6 0 da ys of 
t he last t hera p y d ose i n a n y li ne, 
re gar dless of res p o nse.
Critical a n d 
e x pa n de d 
criteria
Perfect or cl ose matc h. 
Refract or y t o at least o ne PI 
defi ne d as disease pr o gressi o n, 
base d o n I M W G criteria or H C P 
assess me nt, w hile a patie nt is o n 
t hera p y or wit hi n 6 0 da ys of t he 
last t hera p y d ose i n a n y li ne, 
re gar dless of res p o nse. 
Critical a n d 
e x pa n de d 
criteria
0 9 0 1 7 7 e 1 9 d 6 a 1 0 1 2\ A p pr o v e d\ A p pr o v e d O n: 1 5- M a y- 2 0 2 3 0 2: 3 3 ( G M T)

P F -0 6 8 6 3 1 3 5 ( Elra nata ma b)
N O N -I N T E R V E N TI O N A L S T U D Y P R O T O C O L
C 1 0 7 1 0 3 1 Versi o n 1. 0, 0 1 Ma y 2 0 2 3
P FI Z E R C O N FI D E N TI A L 
C T 2 4 -WI -G L 0 2 -R F 0 2 4. 0 N o n -I nter ve nti o nal St u d y Pr ot oc ol Te m plate F or Sec o n dar y Data C ollecti o n St u d y 
0 1 -J u n-2 0 2 2 
Pa ge 5 4 of 7 6
P atie nt i ncl us i o n criteri a per 
C 1 0 7 1 0 0 3 St u d y  
Fl atir o n S p otli g ht R W al ter n ati ve 
a p pr o ac h t o be use d i n t he e xter n al 
c o ntr ol ar m
I m ple me nt ati o n
C O T A R W alter n ati ve
a p pr o ac h t o be use d i n t he 
e xter n al c o ntr ol ar m
I m ple me nt ati o n
Refract or y t o at least o ne a nti -C D 3 8 
m A b .
Perfect or cl ose matc h. 
Refract or y t o at lea st o ne a nti- C D 3 8 
m A b defi ne d as disease pr o gressi o n, 
base d o n I M W G criteria or H C P 
assess me nt, w hile a patie nt is o n t hera p y 
or wit hi n 6 0 da ys of t he last t hera p y d ose 
i n a n y li ne, re gar dless of res p o nse. 
Critical a n d 
e x pa n de d 
criteria
Perfect or cl ose matc h.
Refract or y t o at least o ne a nti -
C D 3 8 m A b defi ne d as disease 
pr o gressi o n, base d o n I M W G 
criteria or H C P assess me nt, 
w hile a patie nt is o n t hera p y or 
wit hi n 6 0 da ys of t he last 
t hera p y d ose i n a n y li ne, 
re gar dless of res p o nse. 
Critical a n d 
e x pa n de d 
criter ia
Rela pse d/refract or y t o last a nti -M M 
re gi me n.
N ote: Refract or y is defi ne d as ha vi n g 
pr o gressi ve disease w hile o n t hera p y 
or wit hi n 6 0 da ys of t he last d ose i n 
a n y li ne, re gar dless of res p o nse.
Perfect or cl ose matc h .
B y  desi g n as the i n de x date is t he first 
li ne after t he T C R eli gi bilit y date, all 
patie nts will be refract or y t o t he last a nti -
m yel o ma re gi me n.
Critical a n d 
e x pa n de d 
criteria
Perfect or cl ose matc h .
B y  desi g n as t he i n de x date is 
t he first li ne after t he T C R 
eli gi bilit y date, all patie nts wi ll 
be refract or y t o t he last a nti -
m yel o ma re gi me n.
Critical a n d 
e x pa n de d 
criteria
C o h ort A: Has n ot recei ve d pri or 
B C M A -directe d t hera p y.
I nf or mati o n is c ollecte d as baseli ne 
c o variates.
Baseli ne 
c o variate
I nf or mati o n is c ollecte d as 
baseli ne c o variat es.
Baseli ne 
c o variate
C o h ort B: Has recei ve d pri or B C M A -
directe d A D C or B C M A -directe d 
C A R T -cell t hera p y, eit her a p pr o ve d 
or i n vesti gati o nal.
I nf or mati o n is c ollecte d as baseli ne 
c o variates.
Baseli ne 
c o variate
I nf or mati o n is c ollecte d as 
baseli ne c o v ariates.
Baseli ne 
c o variate
0 9 0 1 7 7 e 1 9 d 6 a 1 0 1 2\ A p pr o v e d\ A p pr o v e d O n: 1 5- M a y- 2 0 2 3 0 2: 3 3 ( G M T)

P F -0 6 8 6 3 1 3 5 ( Elra nata ma b)
N O N -I N T E R V E N TI O N A L S T U D Y P R O T O C O L
C 1 0 7 1 0 3 1 Versi o n 1. 0, 0 1 Ma y 2 0 2 3
P FI Z E R C O N FI D E N TI A L 
C T 2 4 -WI -G L 0 2 -R F 0 2 4. 0 N o n -I nter ve nti o nal St u d y Pr ot oc ol Te m plate F or Sec o n dar y Data C ollecti o n St u d y 
0 1 -J u n-2 0 2 2 
Pa ge 5 5 of 7 6
P atie nt i ncl us i o n criteri a per 
C 1 0 7 1 0 0 3 St u d y  
Fl atir o n S p otli g ht R W al ter n ati ve 
a p pr o ac h t o be use d i n t he e xter n al 
c o ntr ol ar m
I m ple me nt ati o n
C O T A R W alter n ati ve
a p pr o ac h t o be use d i n t he 
e xter n al c o ntr ol ar m
I m ple me nt ati o n
E C O G perf or ma nce stat us ≤2. 
Perfect or cl ose matc h. 
E C O G perf or ma nce stat us ≤2 wit hi n 
9 0 da ys pri or t o or o n t he i n de x date
Critical a n d 
e x pa n de d 
criteria
Perfect or cl ose matc h. 
E C O G perf or ma nce stat us ≤2 
wit hi n 9 0 da ys pri or t o or o n t he 
i n de x date
K P S sc ore t o be c o n verte d t o 
E C O G if patie nt 's perf or ma nce 
stat us test cl osest t o t he i n de x 
date is K P S
Critical a n d 
e x pa n de d 
criteria
L V E F ≥4 0 % as deter mi ne d b y a 
M U G A sca n or E C H O.
N ot a vaila ble. 
N/ A
N ot a vaila ble. 
N/ A
A de q uate he patic f u ncti o n 
c haracterize d b y t he f oll o wi n g:
a. T otal bilir u bi n ≤2 x U L N ( ≤3 x 
U L N if d oc u me nte d Gil bert’s 
s y n dr o me);
b. A S T ≤2. 5 x U L N; a n d
c. A L T ≤2. 5 x U L N
Perfect or cl ose matc h .
A de q uate he patic bef ore st u d y i n de x 
date.
Baseli ne 
c o variate, 
e x pa n de d 
criteria
Perfect or cl ose matc h .
A de q uate he patic f u ncti o n 
bef ore st u d y i n de x date.
Baseli ne 
c o variate, 
e x pa n de d 
criteria
A de q uate re nal f u ncti o n defi ne d b y 
a n esti mate d creati ni ne cleara nce ≥3 0 
m L/ mi n (acc or di n g t o t he C oc kcr oft 
Ga ult f or m ul a, b y  2 4-h o ur s uri ne 
c ollecti o n f or creati ni ne cleara nce, or 
acc or di n g t o l ocal i nstit uti o nal 
sta n dar d met h o d).
Perfect or cl ose matc h .
A de q uate re nal f u ncti o n bef ore st u d y 
i n de x date.
Baseli ne 
c o variate, 
e x pa n de d 
criteria
Perfect or cl ose matc h .
A de q uat e r e nal f u ncti o n bef ore 
st u d y i n de x date.
Baseli ne 
c o variate, 
e x pa n de d 
criteria
0 9 0 1 7 7 e 1 9 d 6 a 1 0 1 2\ A p pr o v e d\ A p pr o v e d O n: 1 5- M a y- 2 0 2 3 0 2: 3 3 ( G M T)

P F -0 6 8 6 3 1 3 5 ( Elra nata ma b)
N O N -I N T E R V E N TI O N A L S T U D Y P R O T O C O L
C 1 0 7 1 0 3 1 Versi o n 1. 0, 0 1 Ma y 2 0 2 3
P FI Z E R C O N FI D E N TI A L 
C T 2 4 -WI -G L 0 2 -R F 0 2 4. 0 N o n -I nter ve nti o nal St u d y Pr ot oc ol Te m plate F or Sec o n dar y Data C ollecti o n St u d y 
0 1 -J u n-2 0 2 2 
Pa ge 5 6 of 7 6
P atie nt i ncl us i o n criteri a per 
C 1 0 7 1 0 0 3 St u d y  
Fl atir o n S p otli g ht R W al ter n ati ve 
a p pr o ac h t o be use d i n t he e xter n al 
c o ntr ol ar m
I m ple me nt ati o n
C O T A R W alter n ati ve
a p pr o ac h t o be use d i n t he 
e xter n al c o ntr ol ar m
I m ple me nt ati o n
A de q uate B M f u ncti o n c haracterize d 
b y t he f oll o wi n g:
a. A N C ≥1. 0 × 1 0 9
/ L ( use of gra n ul oc yte-c ol o n y 
sti m ulati n g fact ors is per mitte d if 
c o m plete d at least 7 da ys pri or t o 
p la nne d start of d osi n g);
b. Platelets ≥2 5 × 1 0 9
/ L (tra nsf usi o n s u p p ort is per mitte d if 
c o m plete d at least 
7 da ys pri or t o pla n ne d start of 
d osi n g); a n d
c. He m o gl o bi n ≥8 g/ d L (tra nsf usi o n 
s u p p ort is per mitte d if c o m plete d at 
least 
7 da ys pri or t o pla n ne d start of 
d osi n g).
Perfect or cl ose matc h .
A de q uate B M f u ncti o n bef ore st u d y 
i n de x date.
Baseli ne 
c o variate, 
e x pa n de d 
criteria
Perfect or cl ose matc h.
A de q uate B M f u ncti o n bef ore 
st u d y i n de x date.
Baseli ne 
c o variate, 
e x pa n de d 
criteria
Res ol ve d ac ute eff ect s of a n y pri or 
t hera p y t o baseli ne se verit y or 
C T C A E Gra de ≤1.
N ot a vaila ble.
N/ A
N ot a vaila ble. 
N/ A
I nf or me d C o nse nt:
Ca pa ble of gi vi n g si g ne d i nf or me d 
c o nse nt, w hic h i ncl u des c o m plia nce 
wit h t he re q uire me nts a n d restricti o ns 
liste d i n t he IC D a n d i n t his pr ot oc ol.
N ot a p plica ble
N/ A
N/ A
N/ A
0 9 0 1 7 7 e 1 9 d 6 a 1 0 1 2\ A p pr o v e d\ A p pr o v e d O n: 1 5- M a y- 2 0 2 3 0 2: 3 3 ( G M T)

P F -0 6 8 6 3 1 3 5 ( Elra nata ma b)
N O N -I N T E R V E N TI O N A L S T U D Y P R O T O C O L
C 1 0 7 1 0 3 1 Versi o n 1. 0, 0 1 Ma y 2 0 2 3
P FI Z E R C O N FI D E N TI A L 
C T 2 4 -WI -G L 0 2 -R F 0 2 4. 0 N o n -I nter ve nti o nal St u d y Pr ot oc ol Te m plate F or Sec o n dar y Data C ollecti o n St u d y 
0 1 -J u n-2 0 2 2 
Pa ge 5 7 of 7 6
P atie nt e xcl usi o n criteri a per 
C 1 0 7 1 0 0 3 St u d y  
Fl atir o n S p otli g ht R W alter n ati ve 
a p pr o ac h t o be use d i n t he e xter n al 
c o ntr ol ar m
I m ple me nt ati o n
C O T A R W alter n ati ve a p pr o ac h t o 
be use d i n t he e xter n al c o ntr ol ar m
I m ple me nt ati o n
Me dic al C o n diti o ns:
S m ol deri n g M M.
N ot A vai la ble.
N ot i de ntifia ble directl y i n Flatir o n 
S p otli g ht data, h o w e ver patie nts are 
o nl y i ncl u de d i n t he data if t he y ha ve 
e vi de nce of acti ve M M. Si nce 
s mol deri n g M M ( S M M) ca n b e a 
prec urs or t o acti ve M M, patie nts ma y 
ha ve ha d S M M bef ore pr o gressi n g t o 
active M M.
N ot i m ple me nte d 
directl y
Perfect or cl ose matc h .
Patie nt will be e xcl u de d if S M M is 
t he t y pe of M M liste d f or t he patie nt 
cl oset t o ( b ut n ot after) t he i n de x date
Crit ical a n d 
e x pa n de d 
criteria
Acti ve plas ma cell le u ke mia.
Perfect or cl ose matc h.
Dia g n osis of plas ma cell le u ke mia is 
i de ntifie d base d o n t he prese nce of ≥1 
dia g n osis c o de w it hi n t he 3 6 5 da ys pri or 
t o or o n t he i n de x date or base d o n 
i de ntificati o n of plas ma cell le u ke mia i n 
e n ha nce d data a bstracte d fr o m 
u nstr uct ure d s o urces.
Critical a n d 
e x pa n de d criteria
Perfect or cl ose matc h .
Acti ve pl as ma cell le u ke mia wit hi n 
t he 3 6 5 da ys bef ore or o n t he i n de x 
date ca n be i de ntifie d i n t he sec o n dar y 
disease dia g n osis dataset.
Patie nts are ce ns ore d b y C O T A a n d 
n o l o n ger f oll o w e d i n t he data after a 
deter mi nati o n of acti ve plas ma cell 
le u ke mia.
Critical a n d 
e x pa n de d 
criteria
A m yl oi d osis. 
Perfect or cl ose matc h.
Dia g n osis of a m yl oi d osis is i de ntifie d 
base d o n t he prese nce of ≥1 dia g n osis 
c o de wit hi n t he 3 6 5 da ys pri or t o or o n 
t he i n de x date or base d o n i de ntificati o n 
of a m yl oi d osis i n e n ha nce d data 
Critical a n d 
e x pa n de d crite ria
Perfect or cl ose matc h.
A m yl oi d osis wit hi n 3 6 5 da ys bef ore 
or o n t he i n de x date ca n be i de ntifie d
i n t he sec o n dar y disease dia g n osis 
dataset.
Patie nts are ce ns ore d b y C O T A a n d 
Critical a n d 
e x pa n de d 
criteria
0 9 0 1 7 7 e 1 9 d 6 a 1 0 1 2\ A p pr o v e d\ A p pr o v e d O n: 1 5- M a y- 2 0 2 3 0 2: 3 3 ( G M T)

P F -0 6 8 6 3 1 3 5 ( Elra nata ma b)
N O N -I N T E R V E N TI O N A L S T U D Y P R O T O C O L
C 1 0 7 1 0 3 1 Versi o n 1. 0, 0 1 Ma y 2 0 2 3
P FI Z E R C O N FI D E N TI A L 
C T 2 4 -WI -G L 0 2 -R F 0 2 4. 0 N o n -I nter ve nti o nal St u d y Pr ot oc ol Te m plate F or Sec o n dar y Data C ollecti o n St u d y 
0 1 -J u n-2 0 2 2 
Pa ge 5 8 of 7 6
a bstracte d fr o m u nstr uct ure d s o urces.
n o l o n ger f oll o we d i n t he data after a 
dia g n osis of a m yl oi d osis.
P O E M S s y n dr o me
N ot a vaila ble
N/ A
N ot a vaila ble
N/ A
Ste m cell tra ns pla nt wit hi n 1 2 wee ks 
pri or t o e nr oll me nt or acti ve G V H D.
Perfect or cl ose matc h .
Dia g n osis of G V H D is i de ntifie d base d 
o n t he prese nce of ≥1 dia g n osis c o de 
wit hi n t h e 1 2 wee ks pri or t o or o n t he 
i n de x date, b ut t here are n o s pecific 
c o des f or “acti ve”. 
T he i n dicat or of ste m cell tra ns pla nt a n d 
t he date t he patie nt recei ve d t he ste m
cell tra ns pla nt is pr o vi de d. 
Critical a n d 
e x pa n de d criteria
Partial matc h.
Ste m cell tra ns pla nts wit hi n 1 2 w ee ks 
pri or t o i n de x date.
Critical a n d 
e x pa n de d 
criteria
I mpaire d car di o vasc ular f u ncti o n or 
cli nicall y si g nifica nt car di o vasc ular 
diseases, def i ne d as a n y of t he 
f oll o wi n g wit hi n 6 m o nt hs pri or t o 
e nr oll me nt:
a. Ac ute m y ocar dial i nfarcti o n or 
ac ute c or o nar y s y n dr o mes (e g, 
u nsta ble a n gi na, c or o nar y arter y 
b y pass graft, c or o nar y a n gi o plast y 
or ste nti n g, s y m pt o matic pericar dial 
eff usi o n);
b. Cli nica ll y si g nifica nt car diac 
arr h yt h mias (e g, u nc o ntr olle d atrial 
fi brillati o n or u nc o ntr olle d 
par o x ys mal s u pra ve ntric ular 
tac h ycar dia);
c. T hr o m b oe m b olic or 
Parti al matc h.
I de ntifia ble base d o n dia g n osis c o des 
(e xce pt f or Q T s y n dr o me); e n ha nce d 
dat a a bstracte d fr o m u nstr uct ure d 
s o urces ca n i de ntif y t he f oll o wi n g C V 
c o n diti o ns:  a n gi na, 
atrial arr h yt h mia, cere br o vasc ular 
acci de nt, 
c o n gesti ve heart fail ure, 
D V T, MI, a n ot her arr h yt h mia, P E, TI A, 
ve ntric ular arr h yt h mia.
H o w e ver, f or arr h yt h mias t here is n o 
i nf or mati o n as t o t heir cli nical
si g nifica nce (e g, if t he arr h yt h mia is 
u nc o ntr olle d).
Baseli ne c o variate, 
e x pa n de d criteria
Partial matc h .
I de ntifia ble base d o n c o m or bi d 
c o n diti o ns t hat are i ncl u de d i n t he 
C CI.
H o w e ver, c o m or bi d c o n diti o ns 
i ncl u ded i n t he C CI are o nl y ca pt ure d 
o nce per patie nt.
Hist or y of car diac arr h yt h mia, Q T 
s y n dr o me, a n d ot her f or ms of ac ute 
c or o nar y s y n dr o me are n ot 
i de ntifia ble.
Baseli ne 
c o variate, 
e x pa n de d 
criteria
0 9 0 1 7 7 e 1 9 d 6 a 1 0 1 2\ A p pr o v e d\ A p pr o v e d O n: 1 5- M a y- 2 0 2 3 0 2: 3 3 ( G M T)

P F -0 6 8 6 3 1 3 5 ( Elra nata ma b)
N O N -I N T E R V E N TI O N A L S T U D Y P R O T O C O L
C 1 0 7 1 0 3 1 Versi o n 1. 0, 0 1 Ma y 2 0 2 3
P FI Z E R C O N FI D E N TI A L 
C T 2 4 -WI -G L 0 2 -R F 0 2 4. 0 N o n -I nter ve nti o nal St u d y Pr ot oc ol Te m plate F or Sec o n dar y Data C ollecti o n St u d y 
0 1 -J u n-2 0 2 2 
Pa ge 5 9 of 7 6
cere br o vasc ular e ve nts (e g, tra nsie nt 
isc he mic attac k, cere br o vasc ular 
acci de nt, dee p vei n t hr o m b osis 
[u nless ass ociate d wit h a ce ntral 
ve n o us access c o m plicati o n] or 
p ul m o nar y e m b olis m);
d. Pr ol o n ge d Q T s y n dr o me ( or 
tri plicate a vera ge Q Tc F > 4 7 0 msec 
at scree ni n g). 
O n g oi n g Gra de ≥2 peri p heral 
se ns or y or m ot or ne ur o pat h y. 
P artial matc h.
O n g oi n g Gra de ≥2 peri p heral se ns or y or 
m o t or ne ur o pat h y ca n n ot be i de ntifie d 
i n t he data as t here is n o i nf or mati o n 
a b o ut Gra de. 
H o w e ver, treat me nts use d t o ma na ge 
ne ur o pat hic pai n will be use d as a pr o x y 
t o i de ntif y prese nce of m ore se vere
ne ur o pat h y cas es t o descri be patie nts 
Baseli ne c o variate, 
e x pa n de d criteria
N ot a vaila ble .
N/ A
Hist or y of a n y gra de peri p heral 
se ns or y or m ot or ne ur o pat h y wit h 
pri or B C M A -directe d t hera p y 
( C o h ort B).
Perfect or cl ose matc h .
Se ns or y a n d m ot or ne ur o pat h y ma y be 
i de ntifie d base d o n t he prese nce of ≥1 
dia g n osis c o de a n y ti me pri or t o or o n 
t he i n de x date.
A m o n g patie nts wit h pri or B C M A -
directe d t hera p y o nl y.
P ossi ble e xcl usi o n 
criteria 
Perfect or cl ose matc h .
M a y  be i de ntifie d base d o n A Es t hat 
res ulte d in B C M A t reat me nt 
disc o nti n uati o n
P ossi ble 
e xcl usi o n 
criteria 
0 9 0 1 7 7 e 1 9 d 6 a 1 0 1 2\ A p pr o v e d\ A p pr o v e d O n: 1 5- M a y- 2 0 2 3 0 2: 3 3 ( G M T)

P F -0 6 8 6 3 1 3 5 ( Elra nata ma b)
N O N -I N T E R V E N TI O N A L S T U D Y P R O T O C O L
C 1 0 7 1 0 3 1 Versi o n 1. 0, 0 1 Ma y 2 0 2 3
P FI Z E R C O N FI D E N TI A L 
C T 2 4 -WI -G L 0 2 -R F 0 2 4. 0 N o n -I nter ve nti o nal St u d y Pr ot oc ol Te m plate F or Sec o n dar y Data C ollecti o n St u d y 
0 1 -J u n-2 0 2 2 
Pa ge 6 0 of 7 6
Hist or y of G B S or G B S varia nts, or 
hist or y of a n y Gra de ≥3 peri p heral 
m o t or p ol y ne ur o pat h y.
Partial matc h .
G B S c o ul d be i de ntifie d base d o n 
dia g n osis c o des. T here is n o i nf or mati o n 
a b o ut t he gra de i n t he data t hat all o ws 
us t o i de ntif y Gra de ≥3 peri p heral m ot or 
p ol y ne ur o pat h y. Treat me nts use d t o 
ma na ge ne ur o pat hic pai n will be use d as 
a pr o x y t o i de ntif y m ore s e vere 
ne ur o pat h y cases. 
Baseli ne c o variate, 
e x pa n de d criteria
N ot a vaila ble .
N/ A
Ac ti ve H B V, H C V, S A R S-C o V 2, 
HI V, or a n y acti ve, u nc o ntr olle d 
bacterial, f u n gal, or viral 
i nfecti o n. Acti ve i nfecti o ns m ust be 
res ol ve d at least 1 4 da ys pri or t o 
e nr oll me nt. 
Partial matc h .
I de ntifia ble base d o n la b tests as well as 
dia g n osis c o des. T here are n o s pecific 
c o des f or “acti ve” or “ u nc o ntr olle d”, b ut 
“acti ve” i nfecti o ns will be cl osel y 
a p pr o xi mate d usi n g cli nicall y pla usi ble 
c o variate assess me nt peri o ds. H B V or 
HI V a n y ti me bef ore i n de x. Ot her 
i nfecti o ns wit hi n 3 0 da ys bef ore i n de x.
Critical a n d
ex pa n de d criteria
Partial matc h .
H B V, H C V, a n d HI V at a n y ti me 
bef ore i n de x (c o m or bi dities i ncl u di n g 
t hese i nfecti o ns are o nl y ca pt ure d 
o nce per patie nt).
N o i nf or mati o n f or S A R S -C o V 2.
I nf or mati o n o n i nfecti o ns is n ot 
a vaila ble t hr o u g h la b data.
Critical a n d 
e x pa nde d 
criteria
A n y ot her acti ve mali g na nc y wit hi n 
3 years pri or t o e nr oll me nt, e xce pt 
f or a de q uatel y treate d basal cell or 
s q ua m o us cell s ki n ca ncer, or 
carci n o ma i n sit u.
Perfect or cl ose matc h.
Will be i de ntifie d base d o n t he prese nce 
of ≥1 dia g n osis c o de of a n y ot her 
mali g na nc y, e xce pt f or a de q uatel y 
treate d basal cell or s q ua m o us cell s ki n 
ca ncer, or carci n o ma i n sit u wit hi n t he 
3 years pri or t o or o n t he i n de x date. 
" Ade q uatel y treate d" is n o t i de ntifia ble 
t heref ore all basal or s q ua m o us cell s ki n 
ca ncer or carci n o ma i n sit u will be 
c o nsi dere d a de q uatel y treate d a n d n ot 
reas o n f or e xcl usi o n.
Critical a n d 
e x pa n de d criteria
Perfect or cl ose matc h.
A n y sec o n dar y mali g na nc y de vel o pe d 
u p t o 3 year s bef ore i n de x treat me nt. 
O nl y sec o n dar y mali g na ncies 
dia g n ose d bet wee n i nitial M M 
dia g n osis a n d i n de x date are 
i de ntifia ble. C O T A will ce ns or 
patie nts w h o de vel o p sec o n dar y
ca ncer after i nitial M M dia g n osis.
Critical a n d 
e x pa n de d 
criteria
0 9 0 1 7 7 e 1 9 d 6 a 1 0 1 2\ A p pr o v e d\ A p pr o v e d O n: 1 5- M a y- 2 0 2 3 0 2: 3 3 ( G M T)

P F -0 6 8 6 3 1 3 5 ( Elra nata ma b)
N O N -I N T E R V E N TI O N A L S T U D Y P R O T O C O L
C 1 0 7 1 0 3 1 Versi o n 1. 0, 0 1 Ma y 2 0 2 3
P FI Z E R C O N FI D E N TI A L 
C T 2 4 -WI -G L 0 2 -R F 0 2 4. 0 N o n -I nter ve nti o nal St u d y Pr ot oc ol Te m plate F or Sec o n dar y Data C ollecti o n St u d y 
0 1 -J u n-2 0 2 2 
Pa ge 6 1 of 7 6
Ot her s ur gical (i ncl u di n g maj or 
s ur ger y wit hi n 1 4 da ys pri or t o 
e nr oll me nt), me dical or ps yc hiatric 
c o n diti o ns i ncl u di n g rece nt ( wit hi n 
t he past year) or acti ve s uici dal 
i deati o n/ be ha vi or or la b orat or y 
a b n or malit y t hat ma y i ncrease t he 
ris k of st u d y partici pati on or, i n t he 
i n vesti gat or’s j u d g me nt, ma ke t he 
partici pa nt i na p pr o priate f or t he 
st u d y.
N ot a vaila ble .
N/ A
N/ A
N/ A
Pri or/ C o nc o m it a nt T her a p y:
Pre vi o us treat me nt wit h a n 
a nti -B C M A bis pecific a nti b o d y.
Partial matc h .
O nl y n o n -i n vesti gati o nal a ge nts are 
ide ntifia ble, t heref ore t o e ns ure n o 
e x p os ure t o a nti -B C M A bis pecific 
a nti b o d y treat me nt, all patie nts treate d 
wit h i n vesti gati o nal a ge nts w o ul d nee d 
t o be e xcl u de d
N/ A
Partial matc h.
O nl y n o n -i n vesti gati o nal a ge nts are 
i de ntifia ble, t heref ore t o e ns ure n o
e x p os ure t o a nti -B C M A bis pecific 
a nti b o d y treat me nt, all patie nts treate d 
wit h i n vesti gati o nal a ge nts w o ul d 
nee d t o be e xcl u de d
N/ A
Pri or/ C o nc urre nt Cli nic al St u d y E x perie nce:
Pre vi o us a d mi nistrati o n wit h a n 
i n vesti gati o nal dr u g wit hi n 3 0 da ys 
( or as deter mi ne d b y t he l ocal 
re q uire me nt) or 5 half -li ves 
prece di n g t he first d ose of st u d y 
i nter ve nti o n use d i n t his st u d y 
( whic he ver is l o n ger). 
Perfect or cl ose matc h.
Pre vi o us a d mi nistrati o n wit h a n 
i n vesti gati o nal dr u g wit hi n 3 0 da ys 
bef ore t he i n de x date or o n i n de x date.
Critical a n d 
e x pa n de d criteria
Perfect or cl ose matc h.
Pre vi o us a d mi nistrati o n wit h a n 
i n vesti gati o nal dr u g wit hi n 3 0 da ys 
bef ore t he i n de x date or o n i n de x dat e.
Critical a n d 
e x pa n de d 
criteria
0 9 0 1 7 7 e 1 9 d 6 a 1 0 1 2\ A p pr o v e d\ A p pr o v e d O n: 1 5- M a y- 2 0 2 3 0 2: 3 3 ( G M T)

P F -0 6 8 6 3 1 3 5 ( Elra nata ma b)
N O N -I N T E R V E N TI O N A L S T U D Y P R O T O C O L
C 1 0 7 1 0 3 1 Versi o n 1. 0, 0 1 Ma y 2 0 2 3
P FI Z E R C O N FI D E N TI A L 
C T 2 4 -WI -G L 0 2 -R F 0 2 4. 0 N o n -I nter ve nti o nal St u d y Pr ot oc ol Te m plate F or Sec o n dar y Data C ollecti o n St u d y 
0 1 -J u n-2 0 2 2 
Pa ge 6 2 of 7 6
Ot her E xcl usi o ns:
I n vestigat or site staff or Pfizer 
e mpl o y ees directl y i n v ol ve d i n t he 
c o n d uct of t he st u d y, site staff 
ot her wise s u per vise d b y t he 
i n vesti gat or, a n d t heir res pecti ve 
fa mil y me m bers. 
N ot a p plica ble .
N/ A
N/ A
N/ A
K n o w n or s us pecte d h y perse nsiti vit y 
t o t he st u d y i nter ve nti o n or a n y of its 
e xci pie nts.
N ot a p plica ble .
N/ A
N/ A
N/ A
Li ve atte n uate d vacci ne m ust n ot be 
a d mi nistere d wit hi n 4 wee ks of t he 
first d ose of st u d y i nter ve nti o n. 
N ot a vaila ble. 
N/ A
N/ A
N/ A
0 9 0 1 7 7 e 1 9 d 6 a 1 0 1 2\ A p pr o v e d\ A p pr o v e d O n: 1 5- M a y- 2 0 2 3 0 2: 3 3 ( G M T)

P F -0 6 8 6 3 1 3 5 ( Elra nata ma b)
N O N -I N T E R V E N TI O N A L S T U D Y P R O T O C O L
C 1 0 7 1 0 3 1 Versi o n 1. 0, 0 1 Ma y 2 0 2 3
P FI Z E R C O N FI D E N TI A L 
C T 2 4 -WI -G L 0 2 -R F 0 2 4. 0 N o n -I nter ve nti o nal St u d y Pr ot oc ol Te m plate F or Sec o n dar y Data C ollecti o n St u d y 
0 1 -J u n-2 0 2 2 
Pa ge 6 3 of 7 6
S u p ple me nt ar y T a ble 1 5. 2. I ncl usi o n a n d E xcl usi o n C riteri a i n St u d y C 1 0 7 1 0 0 3, C 1 0 7 1 0 1 3 a n d C 1 0 7 1 0 1 4.
P atie nt i ncl usi o n criteri a 
per C 1 0 7 1 0 0 3 St u d y  
C 1 0 7 1 0 1 3 R W alter n ati ve a p pr o ac h t o be 
use d i n t he e xter n al c o ntr ol ar m
I m ple me nt ati o n
C 1 0 7 1 0 1 4 R W a lter n ati ve a p pr o ac h 
t o be use d i n t he e xter n al c o ntr ol 
a r m
I m ple me nt ati o n
A ge a n d Se x:
Male or fe male partici pa nts 
a ge ≥1 8 years.
Perfect or cl ose matc h.
Male or fe male partici pa nts a ge ≥1 8 years.
Critical criteria
Perfect or cl ose matc h.
Male or fe male partici pa nts a ge 
≥1 8 years.
Critical criteria
A fe male partici pa nt is 
eli gi ble t o partic i pate if s he 
is n ot pre g na nt or 
breastfee di n g. 
Perfect or cl ose matc h.
A fe male partici pa nt is eli gi ble t o partici pate if 
s he is n ot pre g na nt or breastfee di n g.
Critical criteria
Perfect or cl ose matc h.
A fe male partici pa nt is eli gi ble t o 
partici pate if s h e is n ot pre g na nt or 
breastfee di n g.
Critical criteria
T y pe of P artici p a nt a n d Dise ase C h ar acteristics:
Partici pa nts w h o are willi n g 
a n d a ble t o c o m pl y wit h all 
sc he d ule d visits, treat me nt 
pla n, la b orat or y tests, 
lifest yle c o nsi derati o ns, a n d 
ot her st u d y pr oce d ures.
N ot a p plica ble.
N/ A
N ot a p plica ble.
N/ A
Pri or dia g n osis of M M as 
defi ne d acc or di n g t o I M W G 
criteria . 
Perfect or cl ose matc h.
Pri or dia g n osis of M M as defi ne d acc or di n g t o 
H C P j u d g me n t or c o nfir me d usi n g I M W G 
criteria
Critical criteria
Perfect or cl ose matc h.
If a p plicati o n of I nter nati o nal 
M y el o ma W or ki n g Gr o u p (I M W G) 
criteria f or dia g n osis of M M or 
assess me nt of res p o nse is n ot 
a vaila ble as part of re g ular cli nical 
practice, cli nici an assess me nt ma y be 
use d.
Critical criteria
0 9 0 1 7 7 e 1 9 d 6 a 1 0 1 2\ A p pr o v e d\ A p pr o v e d O n: 1 5- M a y- 2 0 2 3 0 2: 3 3 ( G M T)

P F -0 6 8 6 3 1 3 5 ( Elra nata ma b)
N O N -I N T E R V E N TI O N A L S T U D Y P R O T O C O L
C 1 0 7 1 0 3 1 Versi o n 1. 0, 0 1 Ma y 2 0 2 3
P FI Z E R C O N FI D E N TI A L 
C T 2 4 -WI -G L 0 2 -R F 0 2 4. 0 N o n -I nter ve nti o nal St u d y Pr ot oc ol Te m plate F or Sec o n dar y Data C ollecti o n St u d y 
0 1 -J u n-2 0 2 2 
Pa ge 6 4 of 7 6
P atie nt i ncl usi o n criteri a 
per C 1 0 7 1 0 0 3 St u d y  
C 1 0 7 1 0 1 3 R W alter n ati ve a p pr o ac h t o be 
use d i n t he e xter n al c o ntr ol ar m
I m ple me nt ati o n
C 1 0 7 1 0 1 4 R W a lter n ati ve a p pr o ac h 
t o be use d i n t he e xter n al c o ntr ol 
a r m
I m ple me nt ati o n
Meas ur a ble disease base d 
o n I M W G criteria as 
defi ne d b y at least 1 of t he 
f oll o wi n g:
a. Ser u m M -pr otei n ≥0. 5 
g/ d L b y S P E P
b. Uri nar y M- pr otei n 
e xcreti o n ≥2 0 0 m g/ 2 4 h o urs 
b y U P E P
c. Ser u m i m m u n o gl o b uli n 
F L C ≥1 0 m g/ d L ( ≥1 0 0 
m g/ L) A N D a b n or mal 
ser u m i m m u n o gl o b uli n
ka p pa t o la m b da F L C rati o
( < 0. 2 6 or > 1. 6 5)
Perfect or cl ose matc h.
I M W G criteria are n ot al wa ys use d or 
d oc u me nte d i n R W. W he n I M W G is n ot 
a vaila ble, H C P j u d g me nt is use d.
Critical criteria
Perfect or c lose matc h.
I ncrease of 2 5 % fr o m t he l o west 
c o nfir me d res p o nse val ue i n o ne or 
m o re of t he I M W G criteria.
Critical criteria
Refract or y t o at least o ne 
I Mi D.
Perfect or cl ose matc h.
I M W G criteria are n ot al wa ys use d or 
d oc u me nte d i n R W. W he n I M W G is n ot 
availa ble, H C P j u d g me nt is use d.
Critical criteria
P erfect or cl ose matc h. 
Refract or y t o at least o ne I Mi D 
defi ne d as disease pr o gressi o n, base d 
o n I M W G criteria w hile a pati e nt is 
o n t hera p y or wit hi n 6 0 da ys of t he 
last t hera p y d ose i n a n y li ne, 
re gar dless of res p o nse.
Critical criteria
0 9 0 1 7 7 e 1 9 d 6 a 1 0 1 2\ A p pr o v e d\ A p pr o v e d O n: 1 5- M a y- 2 0 2 3 0 2: 3 3 ( G M T)

P F -0 6 8 6 3 1 3 5 ( Elra nata ma b)
N O N -I N T E R V E N TI O N A L S T U D Y P R O T O C O L
C 1 0 7 1 0 3 1 Versi o n 1. 0, 0 1 Ma y 2 0 2 3
P FI Z E R C O N FI D E N TI A L 
C T 2 4 -WI -G L 0 2 -R F 0 2 4. 0 N o n -I nter ve nti o nal St u d y Pr ot oc ol Te m plate F or Sec o n dar y Data C ollecti o n St u d y 
0 1 -J u n-2 0 2 2 
Pa ge 6 5 of 7 6
P atie nt i ncl usi o n criteri a 
per C 1 0 7 1 0 0 3 St u d y  
C 1 0 7 1 0 1 3 R W alter n ati ve a p pr o ac h t o be 
use d i n t he e xter n al c o ntr ol ar m
I m ple me nt ati o n
C 1 0 7 1 0 1 4 R W a lter n ati ve a p pr o ac h 
t o be use d i n t he e xter n al c o ntr ol 
a r m
I m ple me nt ati o n
Refract or y t o a t least o ne PI.
Perfect or cl ose matc h.
I M W G criteria are n ot al wa ys use d or 
d oc u me nte d i n R W. W he n I M W G is n ot 
a vaila ble, H C P j u d g me nt is use d.
Critical criteria
Perfect or cl ose matc h. 
Refract or y t o at least o ne PI defi ne d 
as disease pr o gressi o n, base d o n 
I M W G criteria, w hile a patie nt is o n 
t hera p y or wit hi n 6 0 da ys of t he last 
t hera p y d ose i n a n y li ne, re gar dless of 
res p o nse. 
Critical criteria
Refract or y t o at least o ne 
a nti -C D 3 8 m A b a nti b o d y.
Per fect or cl ose matc h.
I M W G criteria are n ot al wa ys use d or 
d oc u me nte d i n R W. W he n I M W G is n ot 
a vaila ble, H C P j u d g me nt is use d.
Critical criteria
Perfect or cl ose matc h. 
Refract or y t o at least o ne a nti -C D 3 8 
m A b defi ne d as disease pr o gressi o n, 
base d o n I M W G criteri a, w hile a 
patie nt is o n t hera p y or wit hi n 6 0 da ys 
of t he last t hera p y d ose i n a n y li ne, 
re gar dless of res p o nse. 
Critical criteria
Rela pse d/refract or y t o last 
a nti -M M re gi me n.
N ote: Refract or y is defi ne d 
as ha vi n g pr o gressi ve 
disease w hile o n t hera p y or
wit hi n 6 0 da ys of t he last 
d ose i n a n y li n e, re gar dless 
of res p o nse.
Perfect or cl ose matc h.
I M W G criteria are n ot al wa ys use d or 
d oc u me nte d i n R W. W he n I M W G is n ot 
a vaila ble, H C P j u d g me nt is use d.
Critical criteria
Perfect or cl ose matc h.
B y  desi g n as t he i n de x date is t he first 
li ne after t he T C R eli gi bilit y date, all 
patie nts will be refract or y t o t he last 
a nti -m yel o ma re gi me n.
C ritical c
0 9 0 1 7 7 e 1 9 d 6 a 1 0 1 2\ A p pr o v e d\ A p pr o v e d O n: 1 5- M a y- 2 0 2 3 0 2: 3 3 ( G M T)

P F -0 6 8 6 3 1 3 5 ( Elra nata ma b)
N O N -I N T E R V E N TI O N A L S T U D Y P R O T O C O L
C 1 0 7 1 0 3 1 Versi o n 1. 0, 0 1 Ma y 2 0 2 3
P FI Z E R C O N FI D E N TI A L 
C T 2 4 -WI -G L 0 2 -R F 0 2 4. 0 N o n -I nter ve nti o nal St u d y Pr ot oc ol Te m plate F or Sec o n dar y Data C ollecti o n St u d y 
0 1 -J u n-2 0 2 2 
Pa ge 6 6 of 7 6
P atie nt i ncl usi o n criteri a 
per C 1 0 7 1 0 0 3 St u d y  
C 1 0 7 1 0 1 3 R W alter n ati ve a p pr o ac h t o be 
use d i n t he e xter n al c o ntr ol ar m
I m ple me nt ati o n
C 1 0 7 1 0 1 4 R W a lter n ati ve a p pr o ac h 
t o be use d i n t he e xter n al c o ntr ol 
a r m
I m ple me nt ati o n
C o h ort A: Has n ot recei ve d 
pri or B C M A -directe d 
t hera p y.
Perfect or cl ose matc h.
I nf or mati o n is c ollected at t he ti me of 
e nr oll me nt w he n a vaila ble.
P ossi ble
i ncl usi o n criteria
Perfect or cl ose matc h.
I nf or mati o n is c ollecte d at t he ti me of 
e nr oll me nt w he n a vaila ble.
P ossi ble 
i ncl usi o n criteria
C o h ort B: Has recei ve d 
pri or B C M A -directe d A D C 
or B C M A -directe d C A R T -
cell t hera p y, eit her a p pr o ve d 
or i n vesti gati o nal.
Perfect or cl ose matc h.
I nf or mati o n is c ollecte d at t he ti me of 
e nr oll me nt w he n a vaila ble.
P ossi ble 
i ncl usi o n criteria 
Perfect or cl ose matc h.
I nf or mati o n is c ollecte d at t he ti me of 
e nr oll me nt w h e n a vailable.
P ossi ble 
i ncl usi o n criteria 
E C O G per f or ma nce stat us 
≤2. 
Perfect or cl ose matc h.
E C O G perf or ma nce stat us ≤2.
Critical criteria
Perfect or cl ose matc h.
E C O G perf or ma nce stat us ≤2.
Critical criteria
L V E F ≥4 0 % as deter mi ne d 
b y a M U G A sca n o r E C H O.
P erfect or cl ose matc h.
L V E F ≥4 0 % as deter m i ne d b y a M U G A sca n or 
E C H O.
N/ A
Perfect or cl ose matc h.
L V E F ≥4 0 % as deter mi ne d b y a 
M U G A sca n or E C H O.
N/ A
A de q uate he patic f u ncti o n 
c haracterize d b y t he 
f oll o wi n g:
a. T otal bilir u bi n ≤2 x U L N 
(≤3 x U L N if d o c u me nte d 
Gil bert’s s y n dr o me);
b. A S T ≤2. 5 x U L N; a n d
c. A L T ≤2. 5 x U L N
Perfect or cl ose matc h.
A de q uate he patic f u ncti o n c haracterize d b y t he 
f oll o wi n g:
a. T otal bilir u bi n ≤2 x U L N ( ≤3 x U L N if 
d oc u me nte d Gil bert’s s y n dr o me);
b. A S T ≤2. 5 x U L N; a n d
c. AL T ≤2. 5 x U L N
N/ A
Perfect or cl ose matc h
a. T otal bilir u bi n ≤2 x U L N ( ≤3 x 
U L N if d oc u me nte d Gil bert’s 
s y n dr o me);
b. A S T ≤2. 5 x U L N; a n d
c. A L T ≤2. 5 x U L N
N/ A
0 9 0 1 7 7 e 1 9 d 6 a 1 0 1 2\ A p pr o v e d\ A p pr o v e d O n: 1 5- M a y- 2 0 2 3 0 2: 3 3 ( G M T)

P F -0 6 8 6 3 1 3 5 ( Elra nata ma b)
N O N -I N T E R V E N TI O N A L S T U D Y P R O T O C O L
C 1 0 7 1 0 3 1 Versi o n 1. 0, 0 1 Ma y 2 0 2 3
P FI Z E R C O N FI D E N TI A L 
C T 2 4 -WI -G L 0 2 -R F 0 2 4. 0 N o n -I nter ve nti o nal St u d y Pr ot oc ol Te m plate F or Sec o n dar y Data C ollecti o n St u d y 
0 1 -J u n-2 0 2 2 
Pa ge 6 7 of 7 6
P atie nt i ncl usi o n criteri a 
per C 1 0 7 1 0 0 3 St u d y  
C 1 0 7 1 0 1 3 R W alter n ati ve a p pr o ac h t o be 
use d i n t he e xter n al c o ntr ol ar m
I m ple me nt ati o n
C 1 0 7 1 0 1 4 R W a lter n ati ve a p pr o ac h 
t o be use d i n t he e xter n al c o ntr ol 
a r m
I m ple me nt ati o n
A de q uate re nal f u ncti o n 
defi ne d b y a n esti mate d 
creati ni ne cleara nce ≥3 0 
m L/ mi n (acc or di n g t o t he 
C oc kcr oft Ga ult f or m ula, b y 
2 4 -h o urs uri ne c ollecti o n f or 
creati ni ne cleara nc e, or 
acc or di n g t o l ocal 
i nstit uti o nal sta n dar d 
met h o d).
Perfect or cl ose matc h.
A de q uate re nal f u ncti o n defi ne d b y a n esti mate d 
creati ni ne cleara nce ≥3 0 m L/ mi n
N/ A
P erfect or cl ose matc h.
A de q uate re nal f u ncti o n defi ne d b y a n 
esti mate d creati ni ne cleara nce ≥3 0 
m L/ mi n
N/ A
A de q uate B M f u ncti o n 
c haracterize d b y t he 
f oll o wi n g:
a. A N C ≥1. 0 × 1 0 9
/ L ( use of gra n ul oc yte-
c ol o n y sti m ulati n g fact ors is 
per mitte d if 
c o m plete d at leas t 7 da ys 
pri or t o pla n ne d start of 
d osi n g);
b. Platelets ≥2 5 × 1 0 9
/ L (tra nsf usi o n s u p p ort is 
per mitte d if c o m plete d at 
least 
7 da ys pri or t o pla n ne d start 
of d osi n g); a n d
c. He m o gl o bi n ≥8 g/ d L 
(tra nsf usi o n s u p p ort is 
per mitte d if c o m plete d at 
least 
7 da ys pri or t o pla n ne d start 
of d osi n g).
Perfe ct or cl ose matc h.
a. A N C ≥1. 0 × 1 0 9
/ L;
b. Platelets ≥2 5 × 1 0 9
/L; a n d
c. He m o gl o bi n ≥8 g/ d L
N/ A
Perfect or cl ose matc h.
a. A N C ≥1. 0 × 1 0 9
/ L;
b. Platelets ≥2 5 × 1 0 9
/L; a n d
c. He m o gl o bi n ≥8 g/ d L 
N/ A
0 9 0 1 7 7 e 1 9 d 6 a 1 0 1 2\ A p pr o v e d\ A p pr o v e d O n: 1 5- M a y- 2 0 2 3 0 2: 3 3 ( G M T)

P F -0 6 8 6 3 1 3 5 ( Elra nata ma b)
N O N -I N T E R V E N TI O N A L S T U D Y P R O T O C O L
C 1 0 7 1 0 3 1 Versi o n 1. 0, 0 1 Ma y 2 0 2 3
P FI Z E R C O N FI D E N TI A L 
C T 2 4 -WI -G L 0 2 -R F 0 2 4. 0 N o n -I nter ve nti o nal St u d y Pr ot oc ol Te m plate F or Sec o n dar y Data C ollecti o n St u d y 
0 1 -J u n-2 0 2 2 
Pa ge 6 8 of 7 6
P atie nt i ncl usi o n criteri a 
per C 1 0 7 1 0 0 3 St u d y  
C 1 0 7 1 0 1 3 R W alter n ati ve a p pr o ac h t o be 
use d i n t he e xter n al c o ntr ol ar m
I m ple me nt ati o n
C 1 0 7 1 0 1 4 R W a lter n ati ve a p pr o ac h 
t o be use d i n t he e xter n al c o ntr ol 
a r m
I m ple me nt ati o n
Res ol ve d ac ute effects of 
a n y pri or t hera p y t o base li ne 
se verit y or C T C A E Gra de 
≤1.
Perfect or cl ose matc h.
Res ol ve d ac ute effects of a n y pri or t hera p y t o 
baseli ne se verit y or C T C A E Gra de ≤1.
N/ A
Perfect or cl ose matc h.
Res ol ve d ac ute effects of a n y pri or 
t hera py t o baseli ne se verit y or 
C T C A E Gra de ≤1.
N / A
I nf or me d C o nse nt:
Ca pa ble of gi vi n g si g ne d 
i nf or me d c o nse nt, w hic h 
i ncl u des c o m plia nce wit h 
t he re q uire me nts a n d 
restricti o ns liste d i n t he I C D 
a n d i n t his pr ot oc ol.
Perfect or cl ose matc h.
E vi de nce o f a pers o nall y si g ne d a n d date d 
i nf or me d c onse nt d oc u me nt i n dicati n g t hat t he 
patie nt ( or a le gall y acce pta ble re prese ntati ve) 
has bee n i nf or me d of all perti ne nt as pects of t he 
st u d y.
St u d y eli gi bilit y 
criteria
Perfect or cl ose matc h.
E vi de nce o f a pers onall y si g ne d a n d 
date d i nf or me d c o nse nt d oc u me nt 
i n dicati n g t hat t he patie nt ( or a le gall y 
acce pta ble re prese ntati ve) has bee n 
i nf or me d of all perti ne nt as pects of 
t he st u d y.
St u d y eli gi bilit y 
criteria
Me dic al C o n diti o ns:
S m ol deri n g M M.
Perfect or cl ose matc h .
Patie nt s are e xcl u de d if S M M is t he t y pe of M M 
liste d f or t he patie nt cl oset t o ( b ut n ot after) t he 
i n de x date
Critical criteria
Perfect or cl ose matc h .
Patie nt s are e xcl u de d if S M M is t he 
t ype of M M liste d f or t he patie nt 
cl oset t o ( b ut n ot after) t he i n de x date
Cri tical criteria
Acti ve plas ma cell 
le u ke mia.
Perfect or cl ose matc h .
Patie nt s wit h acti ve plas ma cell le u ke mia are
e xcl u de d 
Critical criteria
Perfect or cl ose matc h .
Patie nt s wit h acti ve plas ma cell 
le u ke mia are e xclu de d
Critical criteria
A m yl oi d osis. 
Perfect or cl o se matc h.
Patie nt s wit h a m yl oi d osis are e xcl u de d.
Critical criteria
Perfect or cl ose matc h .
Patie nt s wit h a m yl oi d osis are
e xcl u de d
Critical criteria
0 9 0 1 7 7 e 1 9 d 6 a 1 0 1 2\ A p pr o v e d\ A p pr o v e d O n: 1 5- M a y- 2 0 2 3 0 2: 3 3 ( G M T)

P F -0 6 8 6 3 1 3 5 ( Elra nata ma b)
N O N -I N T E R V E N TI O N A L S T U D Y P R O T O C O L
C 1 0 7 1 0 3 1 Versi o n 1. 0, 0 1 Ma y 2 0 2 3
P FI Z E R C O N FI D E N TI A L 
C T 2 4 -WI -G L 0 2 -R F 0 2 4. 0 N o n -I nter ve nti o nal St u d y Pr ot oc ol Te m plate F or Sec o n dar y Data C ollecti o n St u d y 
0 1 -J u n-2 0 2 2 
Pa ge 6 9 of 7 6
P atie nt i ncl usi o n criteri a 
per C 1 0 7 1 0 0 3 St u d y  
C 1 0 7 1 0 1 3 R W alter n ati ve a p pr o ac h t o be 
use d i n t he e xter n al c o ntr ol ar m
I m ple me nt ati o n
C 1 0 7 1 0 1 4 R W a lter n ati ve a p pr o ac h 
t o be use d i n t he e xter n al c o ntr ol 
a r m
I m ple me nt ati o n
P O E M S s y n dr o me
Perfect or cl ose matc h .
Patie nt s wit h P O E M S s y n dr o me are e xcl u de d.
St u d y eli gi bilit y 
criteria
Perfect or cl ose matc h.
Patie nt s wit h P O E M S s y n dr o me are
e xcl u de d.
St u d y eli gi bilit y 
criteria
Ste m cell tra ns pla nt wit hi n 
1 2 w ee ks pri or t o 
e nr oll me nt or acti ve G V H D.
Perfect or cl ose matc h .
Pri o r ste m cell tra ns pla nt wit hi n 1 2 wee ks pri or 
t o st u dy e nr oll me nt or acti ve graft -vers us -h ost -
disease ( G V H D)
Critical criteria
Perfect or cl ose matc h .
Pri or ste m cell tra ns pla nt wit hi n 1 2 
wee ks pri or t o st u d y e nr oll me nt or 
acti ve graft -vers us -h ost -disease
( G V H D)
Critical criteria
I mpaire d car di o vasc ular 
f u ncti o n or cli nicall y 
si g nifica nt car di o vasc ular 
diseases, defi ne d as a n y of 
t he f oll o wi n g wit hi n 6 
m o nt hs pri or t o e nr oll me nt:
a. Ac ute m y ocar dial 
i nfarcti o n or ac ute c or o nar y 
s y n dr o mes (e g, u nsta ble 
a ngi na, c or o nar y arter y 
b y pass graft, c or o nar y 
a n gi o plast y or ste nti n g, 
s y m pt o matic pericar dial 
eff usi o n);
b. Cli nicall y si g nifica nt 
car diac arr h yt h mias (e g, 
u nc o ntr olle d atrial 
fi brillati o n or u nc o ntr olle d 
par o x ys mal s u pra ve ntric ular 
tac h ycar dia);
c. T hr o m b o e m b olic or 
Perfect or cl ose matc h .
Hist or y  of i m paire d car di o vasc ular f u ncti o n or 
cli nicall y si g nifica nt car di o vasc ular diseases 
(C V D s), defi ne d as a n y of t he f oll o wi n g wit hi n 
6 m o nt hs pri or t o st u d y e nr oll me nt:
a. Ac ute m y ocar dial i nfarcti o n ( A MI) or ac ute 
c or o nar y s y n dr o mes (e g , u nsta ble a n gi na, 
c or o nar y arter y b y pass graft, c or o nar y 
a n gi o plast y or ste nti n g, s y m pt o matic pericar dial 
eff usi o n);
b. Cli nicall y si g nifica nt car diac arr h yt h mias (e g , 
u nc o ntr olle d atrial fi brillati o n or u nc o ntr olle d 
par o x ys mal s u pra ve ntric ular tac h ycar dia);
c.T hr o m b oe m b o lic or cere br o vasc ular e ve nts 
(eg , tra nsie nt isc he mic attac k, cere br o vasc ular 
acci de nt, dee p vei n t hr o m b osis [ u nless 
ass ociate d wit h a ce ntral ve n o us access 
c o m plicati o n] or p ul m o nar y e m b olis m)
d. Pr ol o n ge d Q T s y n dr o me ( or tri plicate a vera ge 
Q Tc c orrecte d u si n g Fri dericia’s f or m ula 
[ Q Tc F] > 4 7 0 msec at scree ni n g)
St u d y eli gi bilit y 
criteria
Perfect or cl ose matc h .
Hist or y of i m paire d car di o vasc ular 
f u ncti o n or cli nicall y si g nifica nt 
car di o vasc ular diseases ( C V D), 
defi ne d as a n y of t he f oll o wi n g wit hi n 
6 m o nt h s pri or t o st u d y e nr oll me nt:
a.Ac ute m y ocar dial i nfarcti o n ( A MI) 
or ac ute c or o nar y s y n dr o mes (e. g., 
u nsta ble a n gi na, c or o nar y arter y 
b y pass graft, c or o nar y a n gi o plast y or 
ste nti n g, s y m pt o matic pericar dial
eff usi o n);
b. Cli nicall y si g nifica nt car diac 
arr h yt h mias (e g , u nc o ntr olle d atrial 
fi brillati o n or u nc o ntr olle d 
par o x ys mal s u pra ve ntric ular 
tac h ycar dia);
c.T hr o m b oe m b olic or cere br o vasc ular 
e ve nts (e g , tra nsie nt isc he mic attac k, 
cere br o vasc ular acci de nt, de e p vei n 
t hr o m b osis [ u nless ass ociate d wit h a 
St u d y eli gi bilit y 
criteria
0 9 0 1 7 7 e 1 9 d 6 a 1 0 1 2\ A p pr o v e d\ A p pr o v e d O n: 1 5- M a y- 2 0 2 3 0 2: 3 3 ( G M T)

P F -0 6 8 6 3 1 3 5 ( Elra nata ma b)
N O N -I N T E R V E N TI O N A L S T U D Y P R O T O C O L
C 1 0 7 1 0 3 1 Versi o n 1. 0, 0 1 Ma y 2 0 2 3
P FI Z E R C O N FI D E N TI A L 
C T 2 4 -WI -G L 0 2 -R F 0 2 4. 0 N o n -I nter ve nti o nal St u d y Pr ot oc ol Te m plate F or Sec o n dar y Data C ollecti o n St u d y 
0 1 -J u n-2 0 2 2 
Pa ge 7 0 of 7 6
P atie nt i ncl usi o n criteri a 
per C 1 0 7 1 0 0 3 St u d y  
C 1 0 7 1 0 1 3 R W alter n ati ve a p pr o ac h t o be 
use d i n t he e xter n al c o ntr ol ar m
I m ple me nt ati o n
C 1 0 7 1 0 1 4 R W a lter n ati ve a p pr o ac h 
t o be use d i n t he e xter n al c o ntr ol 
a r m
I m ple me nt ati o n
cere br o vasc ular e ve nts (e g, 
tra nsie nt isc he mic attac k, 
cere br o vasc ular acci de nt, 
dee p vei n t hr o m b osis 
[ u nless ass ociate d wit h a 
ce ntral ve n o us access 
c o m plicati o n] or p ul m o nar y 
e mb olis m);
d. Pr ol o n ge d Q T s y n dr o me 
( or tri plicate a vera ge Q Tc F 
> 4 7 0 msec at scree ni n g). 
ce ntral ve n o us access c o m plicati o n] 
or p ul m o nar y e m b olis m)
d. Pr ol o n ge d Q T s y n dr o me ( or 
tri plicate a vera ge Q Tc c orrecte d usi n g 
Fri dericia’s f or m ula [ Q Tc F] > 4 7 0 
m sec at scree ni n g)
O n g oi n g Gra de ≥2 
peri p heral se ns or y or m ot or 
ne ur o pat h y. 
Perfect or cl ose matc h .
.
Prese nce of o n g oi n g Gra de > 2 peri p heral 
se ns or y or m ot or ne ur o pat h y
St u d y eli gi bilit y 
criteria
Perfect or cl ose matc h .
.
Prese nce of o n g oi n g Gra de > 2 
peri p heral se ns or y or m ot or 
ne ur o pat h y
St u d y eli gi bilit y 
criteria
Hist or y of a n y gra de 
peri p heral se ns or y or m ot or 
ne ur o pat h y wit h pri or 
B C M A -directe d t hera p y 
( C o h ort B).
Perfect or cl ose matc h .
Hist or y of a n y gra de peri p her al se ns or y or 
m o t or ne ur o pat h y, c ollecte d a m o n g patie nts 
wit h pri or B C M A -directe d t hera p y
A m o n g patie nts wit h pri or B C M A -directe d 
t hera p y o nl y.
N/ A
Perfect or cl ose matc h .
Hist or y of a n y gra de peri p heral 
se ns or y or m ot or ne ur o pat h y, 
c ollecte d a m o n g patie nts wit h pri or 
B C M A -directe d t hera p y
N/ A
Hist or y of G B S or G B S 
varia nts, or hist or y of a n y 
Gra de ≥3 peri p heral m ot or 
p ol y ne ur o pat h y.
Perfect or cl ose matc h.
Hist or y of G B S or G B S varia nts, or hist or y of 
a n y Gra de > 3 peri p heral m ot or p ol y ne ur o pat h y .
N/ A
Perfect or cl ose matc h.
Hist or y of G B S or G B S varia nts, or 
hist or y of a n y Gra de > 3 peri p heral 
m o t or p ol y ne ur o pat h y.
N/ A
Acti ve H B V, H C V, S A R S -
C o V 2, HI V, or a n y acti ve, 
u nc o ntr olle d bacterial, 
f u n gal, or viral 
i nfecti o n. Acti ve i nfecti o ns 
Perfect or cl ose matc h.
Prese nce of acti ve he patitis B vir us ( H B V), 
he patitis C v ir us ( H C V), S A R S-C o V -2, h u ma n 
i m m u n o deficie nc y vir us ( HI V), or u nc o ntr olle d 
N/ A
Perfect or cl ose matc h.
Prese nce of acti ve he patitis B vir us 
( H B V), he patitis C vir us ( H C V), 
S A R S -C o V -2, h u ma n 
N/ A
0 9 0 1 7 7 e 1 9 d 6 a 1 0 1 2\ A p pr o v e d\ A p pr o v e d O n: 1 5- M a y- 2 0 2 3 0 2: 3 3 ( G M T)

P F -0 6 8 6 3 1 3 5 ( Elra nata ma b)
N O N -I N T E R V E N TI O N A L S T U D Y P R O T O C O L
C 1 0 7 1 0 3 1 Versi o n 1. 0, 0 1 Ma y 2 0 2 3
P FI Z E R C O N FI D E N TI A L 
C T 2 4 -WI -G L 0 2 -R F 0 2 4. 0 N o n -I nter ve nti o nal St u d y Pr ot oc ol Te m plate F or Sec o n dar y Data C ollecti o n St u d y 
0 1 -J u n-2 0 2 2 
Pa ge 7 1 of 7 6
P atie nt i ncl usi o n criteri a 
per C 1 0 7 1 0 0 3 St u d y  
C 1 0 7 1 0 1 3 R W alter n ati ve a p pr o ac h t o be 
use d i n t he e xter n al c o ntr ol ar m
I m ple me nt ati o n
C 1 0 7 1 0 1 4 R W a lter n ati ve a p pr o ac h 
t o be use d i n t he e xter n al c o ntr ol 
a r m
I m ple me nt ati o n
m ust be res ol ve d at least 1 4 
da ys pri or t o e nr oll me nt. 
i nfecti o n. Acti ve i nfecti o ns m ust be res ol ve d at 
least 1 4 da ys pri or t o st u d y e nr oll me nt.
i m m u n o deficie nc y vir us ( HI V), or 
u nc o ntr olle d i nfecti o n. Acti ve 
i nfecti o ns m ust be res ol ve d at least 1 4 
da ys pri or t o st u d y e nr oll me nt .
A n y ot her acti ve 
mali g na nc y wit hi n 3 years 
pri or t o e nr oll me nt, e xce pt 
f or a de q uatel y treate d basal 
cell or s q ua m o us cell s ki n 
ca ncer, or carci n o ma i n sit u.
Perfect or cl ose matc h.
A n y ot her acti ve mali g na nc y wit hi n 3 years 
pri or t o st u d y e nr oll me nt, e xce pt f or a de q uatel y 
treated basal cell or s q ua m o us cell s ki n ca ncer, 
or carci n o ma i n sit u .
Critical criteria
Perfect or cl ose matc h.
A n y ot her acti ve mali g na nc y wit hi n 3 
years pri or t o st u d y e nr oll me nt, 
e xce pt f or a de q uatel y treate d basal 
cell or s q ua m o us cell s ki n ca ncer, or 
carci n o ma i n sit u.
Critical criteria
Ot her s ur gical (i ncl u di n g 
maj o r s ur ger y wit hi n 1 4 
da ys pri or t o e nr oll me nt), 
me dical or ps yc hiatric 
c o n diti o ns i ncl u di n g rece nt 
( wit hi n t he past year) or 
acti ve s uici dal 
i deati o n/ be ha vi or or 
la b orat or y a b n or malit y t hat 
ma y i ncrease t he ris k of 
st u d y partici pati o n or, i n t he 
i n vesti gat or’s j u d g me nt, 
ma ke t he partici pa nt 
i na p pr o priate f or t he st u d y.
Perfect or cl ose matc h.
Prese nce of s ur gical (i ncl u di n g maj or s ur ger y 
wit hi n 1 4 da ys pri or t o st u d y e nr oll me nt), 
me dical or ps yc hiatric c o n diti o ns i ncl u di n g 
rece nt ( wit hi n t he past year) or acti ve s uici dal 
i deati o n/ be ha vi or or la b orat or y a b n or malit y t hat 
ma y i ncrease t he ris k of st u d y partici pati o n or, 
i n t he i n vesti gat or’s j u d g me nt, ma ke t he 
i n di vi d ual i na p pr o priate f or t he st u dy.
St u d y eli gi bilit y 
criteria
Perfect or cl ose matc h.
Prese nce of s ur gical (i ncl u di n g maj or 
s ur ger y wit hi n 1 4 da ys pri or t o st u d y 
e nr oll me nt), me dical or ps yc hiatric 
c o n diti o ns i ncl u di n g rece nt ( wit hi n 
t he past year) or acti ve s uici dal 
i deati o n/ be ha vi or or la b orat or y 
a b n or malit y t hat ma y i ncrease t he ris k 
of st u d y par tici pati o n or, i n t he 
i n vesti gat or’s j u d g me nt, ma ke t he 
i n di vi d ual i na p pr o priate f or t he st u d y.
St u d y eli gi bilit y 
criteria
0 9 0 1 7 7 e 1 9 d 6 a 1 0 1 2\ A p pr o v e d\ A p pr o v e d O n: 1 5- M a y- 2 0 2 3 0 2: 3 3 ( G M T)

P F -0 6 8 6 3 1 3 5 ( Elra nata ma b)
N O N -I N T E R V E N TI O N A L S T U D Y P R O T O C O L
C 1 0 7 1 0 3 1 Versi o n 1. 0, 0 1 Ma y 2 0 2 3
P FI Z E R C O N FI D E N TI A L 
C T 2 4 -WI -G L 0 2 -R F 0 2 4. 0 N o n -I nter ve nti o nal St u d y Pr ot oc ol Te m plate F or Sec o n dar y Data C ollecti o n St u d y 
0 1 -J u n-2 0 2 2 
Pa ge 7 2 of 7 6
P atie nt i ncl usi o n criteri a 
per C 1 0 7 1 0 0 3 St u d y  
C 1 0 7 1 0 1 3 R W alter n ati ve a p pr o ac h t o be 
use d i n t he e xter n al c o ntr ol ar m
I m ple me nt ati o n
C 1 0 7 1 0 1 4 R W a lter n ati ve a p pr o ac h 
t o be use d i n t he e xter n al c o ntr ol 
a r m
I m ple me nt ati o n
Pri or/ C o nc o m it a nt 
T her a p y:
Pre vi o us treat me nt wit h a n 
a nti -B C M A bis pecific 
a nti b o d y.
Perfect or cl ose matc h.
Pri or or c o nc o mita nt treat me nt wit h a n a nti-
B C M A bis pecific a nti b o d y, i ncl u di n g P F -
0 6 8 6 3 1 3 5 .
St u d y eli gi bilit y 
criteria
Perfect or cl ose matc h.
Pri or or c o nc o mita nt treat me nt w it h 
a n a nti -B C M A bis pecif ic a nti b o d y, 
i ncl u di n g P F-0 6 8 6 3 1 3 5 .
St u d y eli gi bilit y 
criteria
Pri or/ C o nc urre nt Cli nic al 
St u d y E x perie nce:
Pre vi o us a d mi nistrati o n 
wit h a n i n vesti gati o nal dr u g 
wit hi n 3 0 da ys ( or as 
deter mi ne d b y t he l ocal 
re q uire me nt) or 5 half -li ves 
prece di n g t he f irst d ose of 
st u d y i nter ve nti o n use d i n 
t his st u d y ( w hic he ver is 
l o n ger). 
Perfect or cl ose matc h.
Pri or or c o n c o mita nt treat me nt wit h a n a nti-
B C M A bis pecific a nti b o d y, i ncl u di n g P F -
0 6 8 6 3 1 3 5 .
St u d y eli gi bilit y 
criteria
Perfect or cl ose matc h.
Pri or or c o nc o mita nt treat me nt wit h 
a n a nti -B C M A bis pecific a nti b o d y, 
i ncl u di n g P F-0 6 8 6 3 1 3 5 .
St u d y eli gi bilit y 
criteria
Ot her E xcl usi o ns:
I n vesti gat or site staff or 
Pfizer e m pl o yees directl y 
i n v ol ve d i n t he c o n d uct of 
t he st u d y, site staff 
N/ A
N/ A
N/ A
N/ A
0 9 0 1 7 7 e 1 9 d 6 a 1 0 1 2\ A p pr o v e d\ A p pr o v e d O n: 1 5- M a y- 2 0 2 3 0 2: 3 3 ( G M T)

P F -0 6 8 6 3 1 3 5 ( Elra nata ma b)
N O N -I N T E R V E N TI O N A L S T U D Y P R O T O C O L
C 1 0 7 1 0 3 1 Versi o n 1. 0, 0 1 Ma y 2 0 2 3
P FI Z E R C O N FI D E N TI A L 
C T 2 4 -WI -G L 0 2 -R F 0 2 4. 0 N o n -I nter ve nti o nal St u d y Pr ot oc ol Te m plate F or Sec o n dar y Data C ollecti o n St u d y 
0 1 -J u n-2 0 2 2 
Pa ge 7 3 of 7 6
P atie nt i ncl usi o n criteri a 
per C 1 0 7 1 0 0 3 St u d y  
C 1 0 7 1 0 1 3 R W alter n ati ve a p pr o ac h t o be 
use d i n t he e xter n al c o ntr ol ar m
I m ple me nt ati o n
C 1 0 7 1 0 1 4 R W a lter n ati ve a p pr o ac h 
t o be use d i n t he e xter n al c o ntr ol 
a r m
I m ple me nt ati o n
ot her wise s u per vise d b y t he 
i n vesti gat or, a n d t heir 
res pecti ve fa mil y me m bers. 
K n o w n or s us pecte d 
h y perse nsiti vit y t o t he st u d y 
i nter ve nti o n or a n y of its 
e xci pie nts.
N/ A
N/ A
N/ A
N/ A
Li ve atte n uate d vacci ne 
m ust n ot be a d mi nistere d 
wit hi n 4 wee ks of t h e first 
d ose of st u d y i nter ve nti o n. 
N/ A
N/ A
N/ A
N/ A
0 9 0 1 7 7 e 1 9 d 6 a 1 0 1 2\ A p pr o v e d\ A p pr o v e d O n: 1 5- M a y- 2 0 2 3 0 2: 3 3 ( G M T)

P F -0 6 8 6 3 1 3 5 ( Elra nata ma b)
N O N -IN T E R V E N TI O N A L S T U D Y P R O T O C O L
C 1 0 7 1 0 3 1 Versi o n 1. 0, 1 Ma y 2 0 2 3
P FI Z E R C O N FI D E N TI A L 
C T 2 4 -WI -G L 0 2 -R F 0 2 4. 0 N o n -I nter ve nti o nal St u d y Pr ot oc ol Te m plate F or Sec o n dar y Data C ollecti o n St u d y 
0 1 -J u n-2 0 2 2 
Pa ge 7 4 of 7 6
A N N E X 3 . LI S T O F S U B G R O U P S A N D C O M P RI SI N G T R E A T M E N T S
S u b gr o u p
Tre at me nts t o be i ncl u de d i n S u b gr o u p A n al ysis
S u b gr o u p 1
All re gi me ns e xce pt t h ose w hic h i ncl u de t he f oll o wi n g t hera pies:
-Seli ne x or
-Bela nta ma b
-C A R - T
S u b gr o u p 2
O nl y i ncl u di n g t he f oll o wi n g re gi me ns:
-Carfilz o mi b + D e xa met has o ne
-Pa na bi n ostat + B ortez o mi b + D e xa met has o ne
-Carfilz o mi b + L e nali d o mi de+ De xa met has o ne
-Carfil z o mi b + C ycl o p h os p ha mi de + D e x a met has o ne
-P o mali d o mi de + D e x a met has o ne
-P o mali d o mi de + C ycl o p h os p ha mi de + D e x a met has o ne
S u b gr o u p 3
All re gi me ns e xce pt t h ose w hic h i ncl u de t he f oll o wi n g t hera pies:
-Seli ne x or
-Bela nta ma b
-Pa n o bi n ostat
-Be n da m usti ne
S u b gr o u p 4
All re gi me ns e xce pt t h ose w hic h i ncl u de t he f oll o wi n g t hera pies:
-Pa n o bi n ostat
-Ve net ocla x
-Seli ne x or
-H y al ur o ni dase
-A birater o ne
- Trast uz u ma b
-Da brafe ni b
-A ut ol o g o us ste m cell tra ns pla nt
S u b gr o u p 5
O nl y i ncl u di n g t he f oll o wi n g re gi me ns:
-De xa met has o ne/ Pre d nis ol o ne + B ortez o mi b 
-C y cl o p h os p ha mi de + De xa met has o ne/ Pre d nis ol o ne + B ortez o mi b
-Be n da m usti ne 
-P o mali d o mi de 
0 9 0 1 7 7 e 1 9 d 6 a 1 0 1 2\ A p pr o v e d\ A p pr o v e d O n: 1 5- M a y- 2 0 2 3 0 2: 3 3 ( G M T)

P F -0 6 8 6 3 1 3 5 ( Elra nata ma b)
N O N -IN T E R V E N TI O N A L S T U D Y P R O T O C O L
C 1 0 7 1 0 3 1 Versi o n 1. 0, 1 Ma y 2 0 2 3
P FI Z E R C O N FI D E N TI A L 
C T 2 4 -WI -G L 0 2 -R F 0 2 4. 0 N o n -I nter ve nti o nal St u d y Pr ot oc ol Te m plate F or Sec o n dar y Data C ollecti o n St u d y 
0 1 -J u n-2 0 2 2 
Pa ge 7 5 of 7 6
S u b gr o u p
Tre at me nts t o be i ncl u de d i n S u b gr o u p A n al ysis
-De xa met has o ne/ Pre d nis ol o ne + P o mali d o mi de
- De xa met has o ne + P o mali d o mi de + Darat u m u ma b 
-De xa met has o ne/ Pre d nis ol o ne + P o mali d o mi de +Isat u xi ma b
-C y cl o p h os p ha mi de + De xa met has o ne/ Pre d nis ol o ne + P o mali d o mi de
-De xa met has o ne/ Pre d nis ol o n e+ Carfilz o mi b
-De xa met has o ne/ Pre d nis ol o ne + Carfilz o mi b + Darat u m u ma b
-C y cl o p h os p ha mi de + De xa met has o ne + Carfilz o mi b
-De xa met has o ne + P o mali d o mi de + Carfilz o mi b
- De xa met has o ne/ Pre d nis ol o ne + Pa n o bi n ostat
-De xa met has o ne/ Pre d nis ol o ne + B ortez o mi b + Pa n o bi n ostat
-Darat u m u ma b
- De xa met has o ne/ Pre d nis ol o ne + Darat u m u ma b
-De xa met has o ne/ Pre d ni s ol o ne + Le nali d o mide + Darat u m u ma b
-De xa met has o ne/ Pre d nis ol o ne + B ortez o mi b + Darat u m u ma b
-Darat u m u ma b + Carfilz o mi b + L e nali d o mi de + De xa met has o ne/
Pre d nis ol o ne
-Darat u m u ma b + B ortez o mi b + C y cl o p h os p h a mi de + De xa met has o ne/
Pre d nis ol o ne
-De xa met has o ne/ Pre d nis ol o ne + El ot uz u ma b
-De xa met has o ne/ Pre d nis ol o ne +I xaz o mi b
-Isat u xi ma b
-De xa met has o ne/ Pre d nis ol o ne + Carfilz o mi b +I sat u xi ma b
-Isat u xi ma b + De xa met has o ne + P o mali d o mi de + Carfilz o mi b
-A n y ot her re gi me n c o ntai n in g Isat u xi ma b
-Bela nta ma b
-Seli ne x or
0 9 0 1 7 7 e 1 9 d 6 a 1 0 1 2\ A p pr o v e d\ A p pr o v e d O n: 1 5- M a y- 2 0 2 3 0 2: 3 3 ( G M T)

P F -0 6 8 6 3 1 3 5 ( Elra nata ma b)
N O N -IN T E R V E N TI O N A L S T U D Y P R O T O C O L
C 1 0 7 1 0 3 1 Versi o n 1. 0, 1 Ma y 2 0 2 3
P FI Z E R C O N FI D E N TI A L 
C T 2 4 -WI -G L 0 2 -R F 0 2 4. 0 N o n -I nter ve nti o nal St u d y Pr ot oc ol Te m plate F or Sec o n dar y Data C ollecti o n St u d y 
0 1 -J u n-2 0 2 2 
Pa ge 7 6 of 7 6
S u b gr o u p
Tre at me nts t o be i ncl u de d i n S u b gr o u p A n al ysis
- Seli ne x or + De xa met has o ne/ Pre d nis ol o ne
-Ve net ocla x + B ortez o mi b + De xa met has o ne/ Pre d nis ol o ne
-C y cl o p h os p ha mi de
-Pe g y late d li p os o mal d o x or u bici n
-Mel p hala n + De xa met has o ne/ Pre d nis ol o ne
-A dria m yci ne + C ycl o p h os p ha mi de + De xa met has o ne/ Pre d nis ol o ne
-C y cl o p h os p ha mi de + A dria ymici ne/ Pe g y late d li p os o mal 
d o x or u bici n + De xa met has o ne
-Vi ncristi ne + A dria m yci ne + C ycl o p h os p ha mi de + De xa met has o ne
0 9 0 1 7 7 e 1 9 d 6 a 1 0 1 2\ A p pr o v e d\ A p pr o v e d O n: 1 5- M a y- 2 0 2 3 0 2: 3 3 ( G M T)

D o c u m e nt A p pr o v al R e c or d
D o c u m e nt N a m e:
D o c u m e nt Titl e:
Si g n e d B y:
D at e( G M T)
Si g ni n g C a p a cit y
0 9 0 1 7 7 e 1 9 d 6 a 1 0 1 2\ A p pr o v e d\ A p pr o v e d O n: 1 5- M a y- 2 0 2 3 0 2: 3 3 ( G M T)
P P D