BESPONSA® INJECTION 1 MG SPECIAL INVESTIGATION
Conditions: Acute Lymphocytic Leukemia
Sponsor: Pfizer

I n ot uz u ma b
B 1 9 3 1 0 2 4 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
A me n de d 8 , 12 Dece m ber 2 0 2 3
P FI Z E R C O N FI D E N TI A L
C T 2 4 -WI -J P N-G P S P 0 1 -R F 0 9 8 . 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 -N o v -2 0 2 3
P a ge 1 of 3 3
B E S P O N S A ® I njecti o n 1 m g S peci al I n vesti g ati o n
F U L L  P R O T O C O L
Pfizer Ja pa n I nc.
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 Pfizer. E xce pt as ot her w ise 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 otifie d.
0 9 0 1 7 7 e 1 a 2 4 e 5 1 b a\ A p pr o v e d\ A p pr o v e d O n: 0 3- D e c- 2 0 2 4 0 6: 3 3 ( G M T)

I n ot uz u ma b
B 1 9 3 1 0 2 4 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
A me n de d 8 , 12 Dece m ber 2 0 2 3
P FI Z E R C O N FI D E N TI A L
C T 2 4 -WI -J P N-G P S P 0 1 -R F 0 9 8 . 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 -N o v -2 0 2 3
P a ge 2 of 3 3
S T U D Y I N F O R M A TI O N
Title
B E S P O N S A ® I njecti o n 1 m g S pecial I n vesti gati o n
Pr ot oc ol n u m ber
B 1 9 3 1 0 2 4
Pr ot oc ol versi o n i de ntifier
A me n de d 8
D ate 
1 2 Dece m ber 2 0 2 3
Acti ve s u bst a nce
I n ot uz u ma b oz o ga mici n ( ge netical rec o m bi nati o n)
Me dici n al pr o d uct
B E S P O N S A ® I njecti o n 1 m g 
Rese arc h q uesti o n a n d o bjecti ves
T he f oll o wi n g matters will be i n vesti gate d i n 
patie nts recei vi n g B E S P O N S A u n der act ual 
c o n diti o ns of use after mar keti n g:
①
Occ urre nce of a d verse e ve nts ( A Es) (i ncl u di n g 
t he f oll o wi n g e val uati o ns): Bac k gr o u n d 
c haracteristics p ossi bl y affecti n g t he o nset of 
liver dis or der i ncl u di n g ve n o occl usi ve li ver 
disease ( V O D)/si n us oi dal o bstr ucti o n 
s yn dr o me ( S O S), m ye l os u p pressi o n, 
i nfecti o ns a n d hae m orr ha ge, a n d gra de ≥3 
e ve nts base d o n t he C o m m o n Ter mi n ol o g y 
Criteria f or A d verse E ve nts ( C T C A E) v 4. 0 as 
well as ris k fact ors f or earl y deat h of 
he mat o p oietic ste m cell tra ns pla nt ( H S C T) 
patie nts after B E S P O N S A treat me nt
Efficac y ( he mat ol o gic re missi o n rate a n d s ur vi val)
A ut h or
0 9 0 1 7 7 e 1 a 2 4 e 5 1 b a\ A p pr o v e d\ A p pr o v e d O n: 0 3- D e c- 2 0 2 4 0 6: 3 3 ( G M T)
P P D
P P D

I n ot uz u ma b
B 1 9 3 1 0 2 4 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
A me n de d 8 , 12 Dece m ber 2 0 2 3
P FI Z E R C O N FI D E N TI A L
C T 2 4 -WI -J P N-G P S P 0 1 -R F 0 9 8 . 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 -N o v -2 0 2 3
P a ge 3 of 3 3
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 TI O N S .................................................................................................................. 6
3.
R E S P O N SI B L E P A R TI E S ...................................................................................................................... 7
4.
A M E N D M E N T S A N D U P D A T E S ......................................................................................................... 8
5.
M I L E S T O N E S....................................................................................................................................... 1 1
6.
R A TI O N A L E A N D B A C K G R O U N D ..................................................................................................1 1
7.
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 2
7. 1.
Safet y  S pecificati o ns................................................................................................................ 1 2
7. 1. 1.
I m p orta nt Ide ntifie d R is ks.......................................................................................... 1 2
7. 1. 2.
I m p orta nt P ote ntial R is ks........................................................................................... 1 3
7. 2.
Researc h O bjecti ves ................................................................................................................. 1 3
8.
R E S E A R C H M E T H O D S ...................................................................................................................... 1 3
8. 1.
St u d y Desi g n ............................................................................................................................ 1 3
8. 2.
Setti n g ...................................................................................................................................... 1 3
8. 3.
St u d y Sites ............................................................................................................................... 1 4
8. 4.
Pla n ne d S t u d y P eri o d ............................................................................................................... 1 4
8. 5.
St u d y M et h o d ........................................................................................................................... 1 4
8. 6.
C o ntract ....................................................................................................................................1 4
8. 7.
O bser vati o n P eri o d................................................................................................................... 1 5
8. 8.
Varia bles ..................................................................................................................................1 6
8. 8. 1.
Patie nt C haracteristics ................................................................................................1 7
8. 8. 2.
Hist or y of T reat me nt f or t he T ar get D isease at B aseli ne ........................................... 1 8
8. 8. 3.
Tar gete d D r u g U se R ec or d (A ct ual T reat me nt R ec or d) ............................................. 1 8
8. 8. 4.
Rec or ds of B E S P O N S A T reat me nt E n d/ D isc o nti n uati o n.......................................... 1 8
8. 8. 5.
Treat me nt f or t he T ar get D isease after t he S tart of B E S P O N S A T reat me nt..............1 8
8. 8. 6.
Pr o p h ylactic Treat me nt f or R e d uci n g Inf usi o n R eacti o ns (P rese nce or A bse nce) .....1 8
8. 8. 7.
Pr o p h ylactic T reat me nt f or P re ve nti n g a n d R e d uci n g T u m or L ysis S y n dr o me 
( Prese nce or A bse nce) ................................................................................................1 8
8. 8. 8.
Cli nical L a b orat or y T ests ........................................................................................... 1 8
8. 8. 9.
Rec or ds of i n vesti gati o n disc o nti n uati o n ...................................................................1 9
8. 8. 1 0. E C O G P S ( Easter n C o o perati ve O nc ol o g y Perf or ma nce Stat us) ...............................1 9
8. 8. 1 1. Cli nical E val uati o n .....................................................................................................1 9
8. 8. 1 1. 1. He mat ol o gic R e missi o n .......................................................................... 1 9
0 9 0 1 7 7 e 1 a 2 4 e 5 1 b a\ A p pr o v e d\ A p pr o v e d O n: 0 3- D e c- 2 0 2 4 0 6: 3 3 ( G M T)

I n ot uz u ma b
B 1 9 3 1 0 2 4 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
A me n de d 8 , 12 Dece m ber 2 0 2 3
P FI Z E R C O N FI D E N TI A L
C T 2 4 -WI -J P N-G P S P 0 1 -R F 0 9 8 . 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 -N o v -2 0 2 3
P a ge 4 of 3 3
8. 8. 1 1. 2. Mi ni mal R esi d ual D isease ( M R D) ......................................................... 2 0
8. 8. 1 2. C o nfir mati o n of S ur vi val ............................................................................................ 2 0
8. 8. 1 3. Pre g na nc y S tat us (fe male o nl y).................................................................................. 2 0
8. 8. 1 4. A d verse E ve nts ........................................................................................................... 2 0
8. 9.
Data S o urces ............................................................................................................................ 2 1
8. 1 0. St u d y Size ................................................................................................................................2 1
8. 1 0. 1. Pla n ne d Sa m ple S ize ..................................................................................................2 1
8. 1 0. 2. Rati o nale ..................................................................................................................... 2 1
8. 1 1. Data M a na ge me nt .................................................................................................................... 2 1
8. 1 1. 1. Case R e p ort F or ms ( C R Fs) / Electr o nic D ata R ec or ds ............................................... 2 1
8. 1 1. 2. Rec or d R ete nti o n ........................................................................................................ 2 2
8. 1 1. 3. Data C ollecti o n M et h o d ............................................................................................. 2 2
8. 1 1. 4. Patie nt R e gistrati o n ....................................................................................................2 2
8. 1 1. 5. P oi nts t o C o nsi der f or C o m pleti o n, R e visi o n, a n d S u b missi o n of C ase R e p ort F or m 2 2
8. 1 1. 5. 1. Data E ntr y ............................................................................................... 2 2
8. 1 1. 5. 2 . Re visi o n ..................................................................................................2 2
8. 1 1. 5. 3. S u b missi o n ............................................................................................. 2 2
8. 1 2. Data A nal ysis ........................................................................................................................... 2 2
8. 1 2. 1. Defi niti o n of A nal ysis S ets ......................................................................................... 2 3
8. 1 2. 2. A nal ysis M et h o ds ....................................................................................................... 2 3
8. 1 2. 2. 1. A nal ysis of S afet y En d p oi nts .................................................................2 3
8. 1 2. 2. 2. A nal ysis of E fficac y E n d p oi nts .............................................................. 2 3
8. 1 3. Q ualit y C o ntr ol ........................................................................................................................ 2 3
8. 1 4. Li mitati o ns of t he R esearc h M et h o ds ...................................................................................... 2 3
8. 1 5. Ot her As pects ........................................................................................................................... 2 3
9.
P R O T E C TI O N O F H U M A N P A R TI CI P A N T S ................................................................................... 2 4
9. 1.
Patie nt I nf or mati o n .................................................................................................................. 2 4
9. 2.
Patie nt C o nse nt ........................................................................................................................ 2 4
9. 3.
I nstit uti o nal Re vie w B oar d (I R B)/ Et hics C o m mittee ( E C)...................................................... 2 5
9. 4.
Et hical C o n d uct of t he St u d y ...................................................................................................2 5
1 0. 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 D R U G R E A C TI O N S .2 5
1 0. 1. Re q uire me nts ........................................................................................................................... 2 5
1 0. 2. Re p orti n g Peri o d ...................................................................................................................... 2 6
0 9 0 1 7 7 e 1 a 2 4 e 5 1 b a\ A p pr o v e d\ A p pr o v e d O n: 0 3- D e c- 2 0 2 4 0 6: 3 3 ( G M T)

I n ot uz u ma b
B 1 9 3 1 0 2 4 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
A me n de d 8 , 12 Dece m ber 2 0 2 3
P FI Z E R C O N FI D E N TI A L
C T 2 4 -WI -J P N-G P S P 0 1 -R F 0 9 8 . 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 -N o v -2 0 2 3
P a ge 5 of 3 3
1 0. 3. C a usalit y A ssess me nt............................................................................................................... 2 6
1 0 . 4. Defi niti o ns of S afet y E ve nts ....................................................................................................2 7
1 0. 4. 1. A d verse E ve nts ........................................................................................................... 2 7
1 0. 4. 2 .Seri o us A d verse E ve nts .............................................................................................. 2 7
1 0. 4. 3. Sce nari os N ecessitati n g R e p orti n g t o Pfizer Safet y wit hi n 2 4 H o urs ........................2 9
1 1. P L A N S F O R DI S S E MI N A T I N G A N D C O M M U NI C A TI NG S T U D Y R E S U L T S ............................3 1
1 2. N A M E, A N D A D D R E S S O F C O N T R A C T O R A S W E L L A S S C O P E O F W O R K C O N T R A C T E D 3 2
1 3. A D DI TI O N A L M E A S U R E S T H A T M A Y 
B E I M PL E M E N T E D B A S E D O N
T H E S U R V E Y 
R E S U L T S A N D C RI T E RI A F O R D E T E R MI N A TI O N O F T H E I NI TI A TI O N .................................3 2
1 4. S C H E D U L E D TI MI N G O F MI L E S T O N E S A N D T H EI R R A TI O N A L E S F O R E V A L U A TI O N O F 
S T U D Y I M P L E M E N T A TI O N S T A T U S A N D R E S U L T S A N D R E P O R TI N G T O T H E P M D A .....3 2
1 5. O T H E R N E C E S S A R Y M A T T E R S ...................................................................................................... 3 3
1 6. C O N T A C T I N F O R M A TI O N ................................................................................................................ 3 3
1 6. 1. C o ntact I nf or mati o n f or t he C o nte nts of t he St u d y ..................................................................3 3
1 6. 2. C o ntact I nf or mati o n f or t he E D C S yste m ................................................................................ 3 3
0 9 0 1 7 7 e 1 a 2 4 e 5 1 b a\ A p pr o v e d\ A p pr o v e d O n: 0 3- D e c- 2 0 2 4 0 6: 3 3 ( G M T)

I n ot uz u ma b
B 1 9 3 1 0 2 4 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
A me n de d 8 , 12 Dece m ber 2 0 2 3
P FI Z E R C O N FI D E N TI A L
C T 2 4 -WI -J P N-G P S P 0 1 -R F 0 9 8 . 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 -N o v -2 0 2 3
P a ge 6 of 3 3
2.
LI S T O F A B B R E VI A TI O N S
A b bre vi ati o n
Defi niti o n
A D R
a d verse dr u g reacti o n
A E
a d verse e ve nt
A L L
ac ute l y m p h oc ytic le u ke mia
A L T
ala ni ne a mi n otra nsferase
A L - P
al kali ne p h os p hatase
A N C
a bs ol ute ne utr o p hil c o u nt
A S T
as partate a mi n otra nsferase
B U N
bl o o d urea nitr o ge n
C N S
ce ntral ner v o us s yste m
C R
co m plete re missi o n
C R F
case re p ort f or m
C Ri
co m plete re missi o n wit h i nc o m plete c o u nt rec o ver y
C T C A E
C o m m o n Ter mi n ol o g y Criteria f or A d verse E ve nts
E C
et hics c o m mittee
E C O G
Easter n C o o perati ve O nc ol o g y Gr o u p
E D C
electr o nic data ca pt ure
E D P
e x p os ure d uri n g pre g na nc y
γ-G T P
ga m ma gl uta m yl tra ns pe pti dase
G O T
gl uta mic o xal oacetic tra nsa mi nase
G P T
gl uta mic p yr u vic tra nsa mi nase
G V H D
graft vers us h ost disease
H S C T
h e mat o p oietic ste m cell tra ns pla nt
I R B
instit uti o nal re vie w b oar d
M H L W
Mi nistr y of Healt h, La b o ur a n d Welfare
M R D
mi ni mal resi d ual disease
NI S
n o n -i nter ve nti o nal st u d y
P M D A
P har mace uticals a n d Me dical De vices A ge nc y
S A E
seri o us a d verse e ve nt
S A P
statistical a nal ysis pla n
S O S
si n us oi dal o bstr ucti o n s y n dr o me 
U L N
u p per li mit of n or mal
V O D
ve n o occl usi ve disease
W H O
W orl d Healt h Or ga nizati o n
0 9 0 1 7 7 e 1 a 2 4 e 5 1 b a\ A p pr o v e d\ A p pr o v e d O n: 0 3- D e c- 2 0 2 4 0 6: 3 3 ( G M T)

I n ot uz u ma b
B 1 9 3 1 0 2 4 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
A me n de d 8 , 12 Dece m ber 2 0 2 3
P FI Z E R C O N FI D E N TI A L
C T 2 4 -WI -J P N-G P S P 0 1 -R F 0 9 8 . 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 -N o v -2 0 2 3
P a ge 7 of 3 3
3.
R E S P O N SI B L E P A R TI E S
T he Ja pa n G o o d P ost ₋ mar keti n g St u d y Practice officer
0 9 0 1 7 7 e 1 a 2 4 e 5 1 b a\ A p pr o v e d\ A p pr o v e d O n: 0 3- D e c- 2 0 2 4 0 6: 3 3 ( G M T)

I n ot uz u ma b
B 1 9 3 1 0 2 4 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
A me n de d 8 , 12 Dece m ber 2 0 2 3
P FI Z E R C O N FI D E N TI A L
C T 2 4 -WI -J P N-G P S P 0 1 -R F 0 9 8 . 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 -N o v -2 0 2 3
P a ge 8 of 3 3
4.
A M E N D M E N T S A N D U P D A T E S
Versi o n 
I de ntifier
D ate
A me n d me nt 
T y pe
(su bst a nti al or 
a d mi nistr ati ve)
Pr ot oc ol 
S ecti o n(s) 
C h a n ge d
S u m m ar y of 
A me n d me nt(s)
Re as o n
A me n de d 8
1 2 
Dece m ber 
2 0 2 3
A d mi nistrati ve 
re visi o n
A M E N D M E N T S 
A N D U P D A T E S
A d diti o n of “ T y pe of 
re visi o n”
C ha n ge ass ociate d wit h 
t he re visi o n of t he 
i nter nal f or mats,
a me n d me nt of t he 
pr ot oc ol
LI S T O F 
A B B R E VI A TI O N S
A dj ust me nt of 
descri pti o n
C ha n ge ass ociate d wit h 
t he re visi o n of t he 
i nter nal f or mats
S u bsta ntial 
re visi o n
Pl a n ne d St u d y Peri o d
C ha n ge of t he 
re gistrati o n peri o d
B eca use re gistrati o n n ot 
necessitati n g creati o n of 
C R Fs is ter mi nate d base d 
o n Q & A 5 of 
A d mi nistrati ve N otice 
date d 1 0 A u g ust 2 0 2 3 .
St u d y Met h o d
C orre cti o n of 
descri pti o n a b o ut 
“re gistrati o n -o nl y 
i n vesti gati o n”
Patie nt R e gistrati o n
Deleti o n of 
descri pti o n a b o ut 
“re gistrati o n-o nl y 
i n vesti gati o n”
A d mi nistrati ve 
re visi o n
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
A dj ust me nt of 
descri pti o n
C ha n ge ass ociate d wit h 
t he re visi o n of t he 
i nter nal f or mats
N A M E, A D D R E S S, 
A N D 
O U T S O U R C E D 
O P E R A TI O N S O F 
T H E P E R S O N W H O 
W A S 
C O N T R A C T E D 
WI T H T H E 
O P E R A TI O N S
A d diti o n of A 2 
Healt hcare 
C or p orati o n
C ha n ge of t he o uts o urce 
of E D C acc o u nt 
ma na ge me nt acti vities
A me n de d 7
1 1 
N o ve m ber 
2 0 2 2
A d mi nistrati ve 
re visi o n
St u d y i nf or mati o n
C ha n ge fr o m “ Date of 
last versi o n of 
pr ot oc ol” t o “ Date of 
creati o n”
C ha n ge of t he 
di visi o n na me of t he 
a ut h or
C ha n ge ass ociate d wit h 
t he re visi o n of t he 
i nter nal f or mats, c ha n ge 
of t he or ga nizati o n
LI S T O F 
A B B R E VI A TI O N S
A d diti o n of ite ms, 
a dj ust me nt of 
descri pti o n
C ha n ge ass ociate d wit h 
t he re visi o n of t he 
i nter nal f or mats
R E S P O N SI B L E 
P A R TI E S
Deleti o n of “ Cli nical 
Lea d ( gr o u p hea d)”
C ha n ge ass ociate d wit h 
t he re visi o n of t he 
i nter nal f or mats, 
a me n d me nt of t he 
pr ot oc ol
A M E N D M E N T S 
A N D U P D A T E S
Deleti o n of 
“ A me n d me nt of t he 
C ha n ge ass ociate d wit h 
t he re visi o n of t he 
i nter nal f or mats, 
0 9 0 1 7 7 e 1 a 2 4 e 5 1 b a\ A p pr o v e d\ A p pr o v e d O n: 0 3- D e c- 2 0 2 4 0 6: 3 3 ( G M T)

I n ot uz u ma b
B 1 9 3 1 0 2 4 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
A me n de d 8 , 12 Dece m ber 2 0 2 3
P FI Z E R C O N FI D E N TI A L
C T 2 4 -WI -J P N-G P S P 0 1 -R F 0 9 8 . 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 -N o v -2 0 2 3
P a ge 9 of 3 3
Pl a n/ Ot hers,” a d diti o n 
of t he a me n d me nt
a me n d me nt of t he 
pr ot oc ol
MI L E S T O N E S
A d diti o n of ite ms, 
p ost p o ne me nt of t he 
date of fi nal re p ort 
pre parati o n
C ha n ge ass ociate d wit h 
t he re visi o n of t he 
i nter nal f or mats, dela y of 
t he i n vesti gati o n
Data a nal ysis
Li mitati o ns of t he 
researc h met h o ds
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
A dj ust me nt of 
descri pti o n
C ha n ge ass ociate d wit h 
t he re visi o n of t he 
i nter nal f or mats
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 
D R U G R E A C TI O N S
C ha n ge of ter ms, 
a dj ust me nt of 
descri pti o n
C ha n ge ass ociate d wit h 
t he re visi o n of t he 
i nter nal f or mats
C O M M U NI C A TI O N 
O F I S S U E S
O R G A NI Z A TI O N A L 
S Y S T E M F O R 
S T U D Y 
I M P L E M E N T A TI O N
Deleti o n
C ha n ge ass ociate d wit h 
t he re visi o n of t he 
i nter nal f or mats
N A M E, A D D R E S S 
A N D 
O U T S O U R C E D 
O P E R A TI O N S O F 
T H E P E R S O N W H O 
W A S 
C O N T R A C T E D 
WI T H T H E 
O P E R A TI O N S
A d diti o n of 
“ M o nit ori n g” t o 
Pf izer R & D Ja pa n 
G. K.
E x pa nsi o n of t he sc o pe of
o uts o urci n g
C O N T A C T 
I N F O R M A TI O N
C ha n ges of t he 
or ga nizati o n na me of 
Pf izer R & D Ja pa n 
G. K. a n d t he na me, 
tele p h o ne n u m ber, 
a n d e-mail a d dress of 
t he c o ntact 
i nf or mati o n f or t he 
E D C s yste m
C ha n ge of t he 
or ga nizati o n , c ha n ge of 
t he c o ntact i nf or mati o n 
f or t he E D C s yste m
A me n de d 6. 1
8 J u ne 
2 0 2 2
A d mi nistrati ve 
re visi o n
C o ver pa ge
C ha n ge of t he 
c o m pa n y l o g o
C ha n ge of t he l o g o
A me n de d 6
2 2 A pril 
2 0 2 0
S u bsta ntial 
re visi o n
D urati o n of st u d y
C ha n ge of t he 
re gistrati o n peri o d
M o vi n g o n t o a patie nt 
re gistrati o n-o nl y 
i n vesti gati o n
St u d y pr oce d ures
Descri pti o n of t he 
m o vi n g o n t o a patie nt 
re gistrati o n-o nl y 
i n vesti gati o n
O bser vati o n peri o d
Descri pti o n of a 
patie nt fr o m w h o m 
data s h o ul d b e
0 9 0 1 7 7 e 1 a 2 4 e 5 1 b a\ A p pr o v e d\ A p pr o v e d O n: 0 3- D e c- 2 0 2 4 0 6: 3 3 ( G M T)

I n ot uz u ma b
B 1 9 3 1 0 2 4 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
A me n de d 8 , 12 Dece m ber 2 0 2 3
P FI Z E R C O N FI D E N TI A L
C T 2 4 -WI -J P N-G P S P 0 1 -R F 0 9 8 . 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 -N o v -2 0 2 3
P a ge 1 0 of 3 3
c ollecte d t o t he e xte nt 
p ossi ble at t he 
me dical i nstit uti o n t o 
w hic h t he patie nt has 
bee n tra nsferre d
Patie nt re gistrati o n
A d diti o n of t he 
descri pti o n of t he 
m o vi n g o n t o a patie nt 
re gistrati o n-o nl y 
i n vesti gati o n
A me n de d 5
2 2 Ma y 
2 0 1 9
A d mi nistrati ve 
re visi o n
Data ma na ge me nt
A d diti o n of t he “ Case 
re p ort 
f or ms/ Electr o nic data 
rec or ds” a n d “ Rec or d 
rete nti o n” secti o ns
C ha n ge ass ociate d wit h 
t he re visi o n of t he 
i nter nal f or mats
P R O T E C TI O N O F 
H U M A N S U BJ E C T S
C ha n ges t o t he 
“ Patie nt i nf or mati o n,” 
“ Patie nt c o nse nt,” a n d
“ Et hical c o n d uct of 
t he st u d y” secti o ns
A d verse e ve nts
C orrecti o n of 
descri pti o n
Si n gle refere nce 
safet y d oc u me nt
Deleti o n
C O N T A C T 
I N F O R M A TI O N
C ha n ge of t he e -mail 
a d dress i n “t he 
c o ntact i nf or mati o n 
f or t he E D C s yste m”
C ha n ge of t he e -mail
a d dress
O verall
U nificati o n of “ Pfizer 
Ja pa n I nc.” t o 
“ Pfizer”
U nificati o n of t he ter m
A me n de d 4
1 
Dece m ber 
2 0 1 8
A d mi nistrati ve 
re visi o n
N A M E, A D D R E S S, 
A N D 
O U T S O U R C E D 
O P E R A TI O N S O F 
T H E P E R S O N W H O 
W A S 
C O N T R A C T E D 
WI T H T H E 
O P E R A TI O N S
A d diti o n of Pfizer 
R & D Ja pa n G. K.
Esta blis h me nt of Pfizer 
R & D Ja pa n G. K.
C O N T A C T 
I N F O R M A TI O N
C ha n ge of t he na me 
of t he c o ntact 
i nf or mati o n f or t he 
c o nte nts of t he st u d y 
t o “Pf izer R & D Ja pa n 
G. K.”
A me n de d 3
2 5 Ma y 
2 0 1 8
A d mi nistrati ve 
re visi o n
Varia bles
C orrecti o n of err ors i n 
writi n g, a dj ust me nt of 
descri pti o n
Err ors i n writi n g
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 
D R U G R E A C TI O N S
A dj ust me nt of 
descri pti o n
Acc urate defi niti o n of t he 
re p orti n g peri o d, 
reflecti o n of t he i nter nal 
f or mats
0 9 0 1 7 7 e 1 a 2 4 e 5 1 b a\ A p pr o v e d\ A p pr o v e d O n: 0 3- D e c- 2 0 2 4 0 6: 3 3 ( G M T)

I n ot uz u ma b
B 1 9 3 1 0 2 4 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
A me n de d 8 , 12 Dece m ber 2 0 2 3
P FI Z E R C O N FI D E N TI A L
C T 2 4 -WI -J P N-G P S P 0 1 -R F 0 9 8 . 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 -N o v -2 0 2 3
P a ge 1 1 of 3 3
A me n de d 2
2 9 Marc h 
2 0 1 8
A d mi nistrati ve 
re visi o n
N A M E, A D D R E S S, 
A N D 
O U T S O U R C E D 
O P E R A TI O N S O F 
T H E P E R S O N W H O 
W A S 
C O N T R A C T E D 
WI T H T H E 
O P E R A TI O N S
A d diti o n of E P S 
C or p orati o n a n d 
Me di data S ol uti o ns 
K. K.
Deter mi nati o n of t he 
c o ntract ors
C O N T A C T 
I N F O R M A TI O N
A d diti o n of t he 
c o ntact i nf or mati o n 
f or t he c o nte nts of t he 
st u d y a n d t he c o ntact 
i nf or mati o n f or t he 
E D C s yste m
Deter mi nati o n of t he 
c o ntact i nf or mati o n
O verall
A dj ust me nt of 
descri pti o n
A dj ust me nt of 
descri pti o n
A me n de d 1
1 4 Marc h 
2 0 1 8
S u bsta ntial 
re visi o n
Varia bles
( 1) Bac k gr o u n d: 
A d diti o n of “ Date of 
c o nfir mi n g t he i nitial 
o nset” as a n ite m n ot 
t o be c ollecte d f or 
tra nsferre d patie nts.
( 2) Cli nical la b orat or y 
tests: A d diti o n of 
peri p heral blast cells 
a n d m yel o blast cells
( 1) O missi o n of 
descri pti o n
( 2) A d diti o n of t he 
para meters t o i n vesti gate 
ris k fact ors
First versi o n
7 Marc h 
2 0 1 8
N / A
N/ A
N/ A
N/ A
5.
MI L E S T O N E S
Milest o ne
Pl a n ne d d ate
Start of i n vesti gati o n peri o d
Start date of releasi n g B E S P O N S A t o 
t he mar ket
Start of data c ollecti o n (re gistrati o n date of t he first patie nt)
2 J ul y 2 0 1 8
E n d of i n vesti gati o n peri o d
5 years after t he start date of releasi n g 
B E S P O N S A t o t he mar ket *
E n d of data c ollecti o n (release date of t he data base)
Ma y 2 0 2 4
Fi nal st u d y re p ort
Oct o ber 2 0 2 4
*: See Secti o n 8. 4. “ Pla n ne d St u d y Peri o d .”
6.
R A TI O N A L E A N D B A C K G R O U N D
B E S P O N S A ® I njecti o n 1 m g ( n o n pr o prietar y na me, i n ot uz u ma b oz o ga mici n [ ge netical rec o m bi nati o n]) 
( herei nafter referre d t o as B E S P O N S A) is a c he m ot hera pe utic tar geti n g t he C D 2 2 a nti ge n t hat is a n a nti b o d y-
dr u g c o nj u gate c o m p ose d of a C D 2 2 -s pecific a nti b o d y li n ke d t o calic hea mici n, a c yt ot o xic a ntit u m or 
a nti bi otic. I n Ja pa n, t he dr u g was a p pr o ve d i n Ja n uar y 2 0 1 8 f or t he i n dicati o n, “ Rela pse d or refract or y C D 2 2 -
p ositi ve ac ute l y m p h o blastic le u ke mia ( A L L) .”
0 9 0 1 7 7 e 1 a 2 4 e 5 1 b a\ A p pr o v e d\ A p pr o v e d O n: 0 3- D e c- 2 0 2 4 0 6: 3 3 ( G M T)

I n ot uz u ma b
B 1 9 3 1 0 2 4 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
A me n de d 8 , 12 Dece m ber 2 0 2 3
P FI Z E R C O N FI D E N TI A L
C T 2 4 -WI -J P N-G P S P 0 1 -R F 0 9 8 . 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 -N o v -2 0 2 3
P a ge 1 2 of 3 3
T he “ B E S P O N S A ® I njecti o n 1 m g S pecial I n vesti gati o n” will be c o n d ucte d t o u n dersta n d t he safet y of 
B E S P O N S A w he n a d mi nistere d u n der act ual c o n diti o ns of use i n A L L patie nts.
7.
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
7. 1.
S afet y S pecific ati o ns
7. 1. 1.
I m p ort a nt Ide ntifie d R is ks
( 1)
Li ver dis or der i ncl u di n g ve n o occl usi ve li ver disease ( V O D)/si n us oi dal o bstr ucti o n s y n dr o me ( S O S) *
Rati o nale: I n gl o bal P hase 3 ( B 1 9 3 1 0 2 2) a n d n o n -Ja pa nese P hase 1/ 2 ( B 1 9 3 1 0 1 0) st u dies i n patie nts 
wit h rela pse d or refract or y A L L, V O D/ S O S fre q ue ntl y occ urre d, a n d gra de ≥3 he pat ot o xicit y was 
als o re p orte d i n t he i n ot uz u ma b oz o ga mici n gr o u p. He nce, t he occ urre nce of a d verse dr u g reacti o ns 
( A D Rs) a n d ot her rele va nt matters after mar keti n g will be e xa mi ne d i n t he s pecial i n vesti gati o n.
( 2)
M yel os u p pressi o n
Rati o nale: I n St u d y B 1 9 3 1 0 2 2, m yel os u p pressi o n occ urre d i n t he i n ot uz u ma b oz o ga mici n gr o u p, 
a n d ma n y of t hese e ve nts were gra de ≥3. He nce, t he occ urre nce of A D Rs a n d ot her rele va nt matters 
after mar keti n g will be e val uate d i n t he s pecial i n vesti gati o n.
( 3)
I nfecti o ns
Rati o nale: I n St u d y B 1 9 3 1 0 2 2, i nfecti o ns occ urre d i n ma n y patie nts i n t he i n ot uz u ma b oz o ga mici n 
gr o u p, a n d i nfecti o ns attri b uta ble t o m ye l os u p pressi o n w o ul d be li kel y t o fre q ue ntl y de vel o p. He nce, 
t he occ urre nce of A D Rs a n d ot her rele va nt matters after mar keti n g will be assesse d i n t he s pecial 
i n vesti gati o n.
( 4)
He m orr ha ge
Rati o nale: I n St u d y B 1 9 3 1 0 2 2, he m orr ha ge occ urre d i n t he i n ot uz u ma b oz o ga mici n gr o u p, a n d 
he m orr ha ge attri b uta ble t o m yel os u p pressi o n w o ul d be li kel y t o fre q ue ntl y de vel o p. He nce, t he 
occ urre nce of A D Rs a n d ot her rele va nt matters after mar keti n g will be e xa mi ne d i n t he s pecial 
i n vesti gati o n.
( 5)
T u m or l ysis s yn dr o me
Rati o nale: I n St u d y B 1 9 3 1 0 2 2, t u m or l ysis s y n dr o me occ urre d i n t he i n ot uz u ma b oz o ga mici n gr o u p, 
a n d ma n y of t hese e ve nts were gra de ≥3. He nce, t he occ urre nce of A D Rs a n d ot her rele va nt matters 
after mar keti n g will be e val uate d i n t he s pecial i n vesti gati o n.
( 6)
I nf usi o n reacti o n
R ati o nale: I n St u d y B 1 9 3 1 0 2 2, i nf usi o n reacti o n relate d t o i n ot uz u ma b oz o ga mici n occ urre d i n t he 
i n ot uz u ma b oz o ga mici n gr o u p. He nce, t he occ urre nce of A D Rs a n d ot her rele va nt matters after 
mar keti n g will be assesse d i n t he s pecial i n vesti gati o n.
( 7)
P a ncreatiti s
Rati o nale: I n St u dies B 1 9 3 1 0 2 2 a n d B 1 9 3 1 0 1 0, a n d St u dies B 1 9 3 1 0 0 6 a n d B 1 9 3 1 0 0 8 i n v ol vi n g 
s u bjects ot her t ha n patie nts wit h rela pse d or refract or y A L L, pa ncreatitis occ urre d i n t he i n ot uz u ma b 
oz o ga mici n gr o u p. Pa ncreatitis ma y affect t he be nefit -ris k balance of i n ot uz u ma b oz o ga mici n. 
0 9 0 1 7 7 e 1 a 2 4 e 5 1 b a\ A p pr o v e d\ A p pr o v e d O n: 0 3- D e c- 2 0 2 4 0 6: 3 3 ( G M T)

I n ot uz u ma b
B 1 9 3 1 0 2 4 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
A me n de d 8 , 12 Dece m ber 2 0 2 3
P FI Z E R C O N FI D E N TI A L
C T 2 4 -WI -J P N-G P S P 0 1 -R F 0 9 8 . 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 -N o v -2 0 2 3
P a ge 1 3 of 3 3
He nce, t he occ urre nce of A D Rs a n d ot her rele va nt matters after mar keti n g will be e xa mi ne d i n t he 
s pecial i n vesti gati o n.
7. 1. 2.
I m p ort a nt P ote nti al R is ks
( 1)
I nterstitial l u n g disease
Rati o nale: I n St u d y B 1 9 3 1 0 2 2 a n d St u d y B 1 9 3 1 0 0 6 i n v ol vi n g s u bjects ot her t ha n patie nts wit h rela pse d 
or refract or y A L L, i nterstitial l u n g disease occ urre d i n t he i n ot uz u ma b oz o ga mici n gr o u p. I nterstitial l u n g 
disease ma y affect t he be nefit -ris k bala nce of i n ot uz u ma b oz o ga mici n. T h us, if it is re p orte d i n t he s pecial 
i n vesti gati o n, a p plica ble patie nts will be e xtracte d a n d e val uate d.
( 2)
I nfla m mat or y gastr oi ntesti nal e ve nts
Rati o nale: I n St u dies B 1 9 3 1 0 2 2 a n d B 1 9 3 1 0 1 0, a n d St u dies B 1 9 3 1 0 0 3, B 1 9 3 1 0 0 4 a n d B 1 9 3 1 0 0 8 
i n v ol vi n g s u bjects ot her t ha n patie nts wit h rela pse d or refract or y A L L, i nfla m mat or y gastr oi ntesti nal 
e ve nts occ urre d i n t he i n ot uz u ma b oz o ga mici n gr o u p. Gastr oi ntesti nal m uc osal i nfla m mati o n a n d ulcers 
ma y affect t he be nefit -ris k bala nce of i n ot uz u ma b oz o ga mici n. T h us, if t he y are re p orte d i n t he s pecial 
i n vesti gati o n, a p plica ble patie nts will be e xtracte d a n d assesse d.
( 3)
Q Tc i nter val pr ol o n ge d
Rati o nale: I n St u d y B 1 9 3 1 0 2 2, Q Tc i nter val pr ol o n ge d relate d t o i n ot uz u ma b oz o ga mici n occ urre d i n t he 
i n ot uz u ma b oz o ga mici n gr o u p. T h us, if it is re p orte d i n t he s pecial i n vesti gati o n, a p plica ble patie nts will 
be e xtracte d a n d e xa mi ne d.
( 4)
Re pr o d ucti ve t o xicit y
Rati o nale: W hile n o e x p os ure d uri n g pre g na nc y or lactati o n was re p orte d i n cli nical st u dies, fi n di n gs i n 
t he re pr o d ucti ve or ga ns, a n d e m br y os a n d fet uses were o btai ne d i n n o ncli nical st u dies. T h us, if it is 
re p orte d i n t he s pecial i n vesti gati o n, a p plica ble patie nts will be e xtracte d a n d e val uate d.
7. 2.
Rese arc h O bjecti ves
T he f oll o wi n g matters will be i n vesti gate d i n patie nts recei vi n g B E S P O N S A u n der act ual c o n diti o n s of 
use after mar keti n g:
( 1) Occ urre nce of a d verse e ve nts ( A Es) (i ncl u di n g t he f oll o wi n g e val uati o ns):
Bac k gr o u n d c haracteristics p ossi bl y affecti n g t he o nset of li ver dis or der i ncl u di n g V O D/ S O S, 
m yel os u p pressi o n , i nfecti o ns a n d he m orr ha ge, a n d gra de ≥3 e ve nts base d o n t he C T C A E v 4. 0 as well 
as ris k fact ors f or earl y deat h of H S C T patie nts after B E S P O N S A treat me nt
( 2) Efficac y ( he mat ol o gic re missi o n rate a n d s ur vi val)
8.
R E S E A R C H M E T H O D S
8. 1.
St u d y Desi g n
T his is a m ultice nter, c o h ort st u d y i n patie nts recei vi n g B E S P O N S A. T he i n vesti gat ors will c o m plete 
case re p ort f or ms ( C R Fs) base d o n t he i nf or mati o n re q uire d f or t he st u d y t hat will be e xtracte d fr o m 
me dical rec or ds create d i n dail y me dical practice.
8. 2.
Setti n g
All patie nts recei vi n g B E S P O N S A will be i nc l u de d i n t his i n vesti gati o n.
0 9 0 1 7 7 e 1 a 2 4 e 5 1 b a\ A p pr o v e d\ A p pr o v e d O n: 0 3- D e c- 2 0 2 4 0 6: 3 3 ( G M T)

I n ot uz u ma b
B 1 9 3 1 0 2 4 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
A me n de d 8 , 12 Dece m ber 2 0 2 3
P FI Z E R C O N FI D E N TI A L
C T 2 4 -WI -J P N-G P S P 0 1 -R F 0 9 8 . 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 -N o v -2 0 2 3
P a ge 1 4 of 3 3
[I N DI C A TI O N]
Rela pse d or refract or y C D 2 2 -p ositi ve A L L
[ D O S A G E A N D A D MI NI S T R A TI O N]
T he us ual d osa ge f or a d ults is 0. 8 m g/ m 2 ( b o d y s urface area) of i n ot uz u ma b oz o ga mici n ( ge netical 
rec o m bi nati o n) o n Da y 1 a n d 0. 5 m g/ m2 ( b o d y s urface area) o n Da ys 8 a n d 1 5 o nce dail y b y 
i ntra ve n o us i nf usi o n o ver at least 1 h o ur f oll o we d b y a treat me nt-free i nter val. T he le n gt h of c ycle is 2 1 
t o 2 8 da ys f or C ycle 1 a n d 2 8 da ys f or C ycles 2 a n d s u bse q ue nt c ycles. T he treat me nt s h o ul d be 
repeate d. T he n u m ber of c ycles s h o ul d be deci de d w hile ta ki n g acc o u nt of t he sc he d ule of H S C T. T he 
d ose s h o ul d be decrease d as necessar y acc or di n g t o t he patie nt’s c o n diti o n.
8. 3.
St u d y Sites
T he i n vesti gati o n will be c o n d ucte d at a p pr o xi matel y 2 0 0 sites mai nl y t he de part me nts of he mat ol o g y 
a n d i nter nal me dici ne.
8. 4.
Pl a n ne d S t u d y P eri o d
T he pla n ne d peri o d of t his i n vesti gati o n is as f oll o ws:
S t u d y peri o d : For 5 years fr o m t he start date of releasi n g B E S P O N S A t o t he mar ket *
Re gistrati o n peri o d: Fr o m t he start date of releasi n g B E S P O N S A t o t he mar ket t o 3 0 A pril 2 0 2 0 *
*: P atie nts starti n g t o recei ve B E S P O N S A o n or bef ore 3 0 A pril 2 0 2 0 will be re gistere d i n t his 
i n vesti gati o n re gar dless of t he re gistrati o n peri o d a n d C R Fs will be c ollecte d.
8. 5.
St u d y M et h o d
A ce ntral re gistrati o n all -patie nt s ur veilla nce s yste m will be use d f or t his i n vesti gati o n. Patie nts 
a p plica ble t o t he i n vesti gati o n will be re gistere d u ntil meeti n g t he tar get sa m ple 
size. Re gistrati o n t o t he i n vesti gati o n will be ma de b y e nteri n g i nf or mati o n i n t he electr o nic data 
ca pt ure ( E D C) s yste m f or p ost -mar keti n g dr u g i n vesti gati o n o n t he I nter net w hic h will be pr o vi de d b y 
t he s p o ns or wit hi n a p pr o xi matel y 2 wee ks after t he start of B E S P O N S A treat me nt ( or after c o ncl u di n g 
a c o ntract).
T he s p o ns or will c hec k t hat patie nts w h o ha ve starte d treat me nt wit h B E S P O N S A b y  3 0 A pril 2 0 2 0
ha ve bee n re gistere d usi n g t he E D C s yste m or pa per f or m.
After it is c o nfir me d t hat t he tar get i nf or mati o n ca n be c ollecte d, patie nt re gistrati o n a n d re q uest f or 
ne w C R F c o m pleti o n will be e n de d. H o we ver, t he s yste m f or ta ki n g pr o m pt meas ures s h o ul d be 
mai ntai ne d i n pre parati o n f or t he cases i n w hic h a d diti o nal i n vesti gati o n, etc. i s j u d ge d t o be necessar y 
fr o m t he vie w p oi nt of safet y meas ures, etc., a n d is i nstr ucte d b y t he P har mace uticals a n d Me dical 
De vices A ge nc y or t he Mi nistr y of Healt h, La b o ur a n d Welfare ( M H L W).
8. 6.
C o ntr act
A writte n a gree me nt o n t he i m ple me ntati o n of t his i n ves ti gati o n s h o ul d be, i n pri nci ple, c o ncl u de d wit h 
t he hea d of t he site bef ore deli veri n g B E S P O N S A, a n d t he n B E S P O N S A s h o ul d be deli vere d. 
H o we ver, e ve n if B E S P O N S A is gi ve n t o a patie nt bef ore c o ncl u di n g t he a gree me nt d ue t o t hera pe utic 
pri orit y, t he patie n t s h o ul d be re gistere d i n t he i n vesti gati o n. If s pecifie d a gree me nt d oc u me nts are 
a vaila ble at t he sites, t he y s h o ul d be res pecte d.
0 9 0 1 7 7 e 1 a 2 4 e 5 1 b a\ A p pr o v e d\ A p pr o v e d O n: 0 3- D e c- 2 0 2 4 0 6: 3 3 ( G M T)

I n ot uz u ma b
B 1 9 3 1 0 2 4 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
A me n de d 8 , 12 Dece m ber 2 0 2 3
P FI Z E R C O N FI D E N TI A L
C T 2 4 -WI -J P N-G P S P 0 1 -R F 0 9 8 . 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 -N o v -2 0 2 3
P a ge 1 5 of 3 3
8. 7.
O bser v ati o n P eri o d
O bser vati o n peri o d: T he o bser vati o n peri o d will be 5 2 wee ks f or patie nts n ot u n der g oi n g H S C T wit hi n 
5 2 wee ks after t he start of B E S P O N S A treat me nt or u ntil 5 2 wee ks f oll o wi n g H S C T f or patie nts 
u n der g oi n g it wit hi n 5 2 wee ks after t he start of B E S P O N S A treat me nt. F or a patie nt starti n g 
B E S P O N S A treat me nt bef ore 3 0 A pril 2 0 2 0 w h o is tra nsferre d t o a n ot her h os pital d uri n g t he 
o bser vati o n peri o d, t he c o ncl usi o n of t he a gree me nt o n t he i m ple me ntati o n of t his i n vesti gati o n s h o ul d 
be re q ueste d t o t he h os pital t o w hic h t he patie nt has bee n tra nsferre d, a n d data fr o m t here s h o ul d be 
c ollecte d t o t he e xte nt p ossi ble.
( 1) Treat me nt p hase
Fr o m treat me nt i nitiati o n t o Da y 2 8 p ost -treat me nt: Safet y (e. g., A Es) a n d efficac y ( he mat ol o gic 
re missi o n a n d s ur vi val)
( 2) F oll o w -u p p hase
Fr o m Da y 2 9 p ost -treat me nt t o Wee k 5 2 (patie nts w h o di d n ot u n der g o H S C T bef ore Wee k 5 2 ) or 5 2 
wee ks after H S C T ( patie nts w h o u n der we nt H S C T bef ore Wee k 5 2 ): Safet y ( V O D/ S O S) a n d efficac y 
(s ur vi val)
0 9 0 1 7 7 e 1 a 2 4 e 5 1 b a\ A p pr o v e d\ A p pr o v e d O n: 0 3- D e c- 2 0 2 4 0 6: 3 3 ( G M T)

I n ot uz u ma b
B 1 9 3 1 0 2 4 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
A me n de d 8 , 12 Dece m ber 2 0 2 3
P FI Z E R C O N FI D E N TI A L
C T 2 4 -WI -J P N-G P S P 0 1 -R F 0 9 8 . 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 -N o v -2 0 2 3
P a ge 1 6 of 3 3
8. 8.
V ari a bles
T he f oll o wi n g i nf or mati o n o btai ne d d uri n g t he o bser vati o n peri o d i n t he i n vesti gati o n is defi ne d as s ur ve y 
ite ms.
Ta ble 1. Sc he d ule of O bser vati o n
Varia bles
Re gistrati o n 
f or m
B o o klet 1
B o o klet 2
Baseli ne
Treat me nt 
p hase *
F oll o w -u p 
p hase * 2
Patie nt 
bac k gr o u n d
I D n u m ber
●
◯
Ge n der
●
◯
Birt h m o nt h a n d year (a ge at t he start of t he 
i n vesti gati o n if it is n ot pr o vi de d)
●
◯
E x perie nce of partici pati o n i n t his 
i n vesti gati o n
●
◯
N o
( Pla n ne d) start date of B E S P O N S A 
treat me nt
●
Tar get disease of t he i n vesti gati o n
●
I nf or me d c o nse nt fr o m t he patie nt 
( B E S P O N S A treat me nt)* 3
●
I nf or me d c o nse nt fr o m t he patie nt 
(p u blicati o n of
t he i n v esti g ati o n 
r es ults)* 4
●
◯
Y es
Patie nt re gistrati o n n u m ber at t he 
site fr om 
w hic h 
t he patie nt 
is 
tra nsferre d
●
◯
Na me of t he site fr om w hic h t he 
patie nt is tra nsferre d
●
◯
Hei g ht a n d wei g ht
●
Tar get disease
●
Date of c o nfir mi n g t he i nitial o nset
●
C hr o m os o mal kar y ot y pe
●
Me dical hist or y
●
Treat me nt hist or y at baseli ne (re missi o n i n d ucti o n 
c he m ot hera pies a n d H S C T fr o m t he i nitial o nset)
●
B E S P O N S A treat me nt rec or d
Pre g na nc y stat us
Cli nical la b orat or y tests
( he mat ol o g y, he patic f u ncti o n test, re nal f u ncti o n test, 
electr ocar di o gra p h y, pa ncreatic f u ncti o n test, b o ne 
marr o w test)
●
▲
▲
E C O G P S
●
▲
▲
Treat me nt of t he tar get disease after t he start of 
B E S P O N S A treat me nt ( H S C T, H S C T d o n or i nf or mati o n, 
dr u g t hera p y, n o n -dr u g t hera p y)
Pr o p h ylactic treat me nt f or re d uci n g i nf usi o n reacti o ns
Pr o p h ylactic treat me nt f or pre ve nti n g a n d re d uci n g 
t u m o ur l ysis s y n dr o me
Cli nical e val uati o n ( he mat ol o gic re missi o n a n d M R D)
0 9 0 1 7 7 e 1 a 2 4 e 5 1 b a\ A p pr o v e d\ A p pr o v e d O n: 0 3- D e c- 2 0 2 4 0 6: 3 3 ( G M T)

I n ot uz u ma b
B 1 9 3 1 0 2 4 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
A me n de d 8 , 12 Dece m ber 2 0 2 3
P FI Z E R C O N FI D E N TI A L
C T 2 4 -WI -J P N-G P S P 0 1 -R F 0 9 8 . 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 -N o v -2 0 2 3
P a ge 1 7 of 3 3
A d verse e ve nts
A n y A Es
V O D/ S O S
Rec or ds of treat me nt e n d/ disc o nti n uati o n
●
Rec or ds of i n vesti gati o n c o nti n uati o n/ disc o nti n uati o n
●
●
C o nfir mati o n of s ur vi val (i ncl u di n g reas o ns f or deat h)
*: 
Fr o m treat me nt i nitiati o n t o Da y 2 8 p ost -treat me nt
* 2: Fr o m Da y 2 9 p ost -treat me nt t o Wee k 5 2 ( patie nts w h o di d n ot u n der g o H S C T bef ore Wee k 5 2) or 5 2 wee ks after H S C T ( patie nts 
w h o u n der we nt H S C T bef ore Wee k 5 2)
* 3: I nf or me d c o nse nt f or B E S P O N S A treat me nt s h o ul d be o btai ne d after a de q uatel y e x plai ni n g t he efficac y of a n d ris ks fr o m 
B E S P O N S A t o t he patie nt.
* 4: I nf or me d c o nse nt s h o ul d be o btai ne d w he ne ver p ossi ble f or discl osi n g t he res ults fr o m t his i n vesti gati o n at aca de mic c o nfere nces, 
i n pa pers a n d/ or t hr o u g h ot her rele va nt mea ns.
: Data e ntr y or ite ms t o be e ntere d; , If t here is n o c ha n ge i n data, data i n t he re gistrati o n f or m s h o ul d be use d as t h ose i n t he C R F.
▲: H S C T pretreat me nt data s h o ul d be e ntere d f or patie nts w h o u n der we nt H S C T after t he i nitiati o n of B E S P O N S A treat me nt o nl y.
8. 8. 1.
P atie nt C h ar acteristics
T he f oll o wi n g i nf or mati o n at t he start of B E S P O N S A treat me nt will be e ntere d or rec or de d:
( 1) I D n u m ber
( 2) Ge n der
( 3) Birt h m o nt h a n d year (a ge at t he start of t he i n vesti gati o n if it is n ot pr o vi de d)
( 4) Hei g ht a n d wei g ht
( 5) Tar get disease ( na me of disease, W H O Classificati o n 4t h e diti o n, rela pse d or refract or y)
( 6) Date of c o nfir mi n g t he i nitial o nset
( 7) C hr o m os o mal kar y ot y pe
( 8) Me dical hist or y ( V O D/ S O S, graft vers us h ost disease [ G V H D], he patitis, li ver disease, ot her diseases)
( 9) E x perie nce of partici pati o n i n t his i n vesti gati o n
( 1 0) Patie nt re gistrati o n n u m ber at t he site fr o m w hic h t he patie nt is tra nsferre d (f or tra nsferre d patie nts)
( 1 1) Na me of t he site fr o m w hic h t he patie nt is tra nsferre d (f or tra nsferre d pati e nts)
( 1 2) I nf or me d c o nse nt fr o m t he patie nt ( B E S P O N S A treat me nt)*
( 1 3) I nf or me d c o nse nt fr o m t he patie nt (p u blicati o n of t he i n v esti g ati o n r es ults)
I nf or me d c o nse nt s h o ul d be o btai ne d w he ne ver p ossi ble f or discl osi n g t he res ults fr o m t his i n vesti gati o n 
at aca de mic c o nfere nces, i n pa pers a n d/ or t hr o u g h ot her rele va nt mea ns.
( 1 4) Tar get disease of t he i n vesti gati o n *
( 1 5) ( Pla n ne d) start date of B E S P O N S A treat me nt*
*: Ite ms t o be e ntere d i n t he re gistrati o n f or m o nl y
T he f oll o wi n g ite ms will n ot be c ollecte d fr o m tra nsferre d patie nts:
Inf or me d c o nse nt fr o m t he patie nt ( B E S P O N S A treat me nt)
Inf or me d c o nse nt fr o m t he patie nt ( p u blicati o n of t he i n v esti g ati o n r es ults)
Hei g ht a n d wei g ht
0 9 0 1 7 7 e 1 a 2 4 e 5 1 b a\ A p pr o v e d\ A p pr o v e d O n: 0 3- D e c- 2 0 2 4 0 6: 3 3 ( G M T)

I n ot uz u ma b
B 1 9 3 1 0 2 4 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
A me n de d 8 , 12 Dece m ber 2 0 2 3
P FI Z E R C O N FI D E N TI A L
C T 2 4 -WI -J P N-G P S P 0 1 -R F 0 9 8 . 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 -N o v -2 0 2 3
P a ge 1 8 of 3 3
Tar get disease ( na me of disease, W H O Classificati o n 4t h e diti o n, rela pse d or refract or y)
Date of c o nfir mi n g t he i nitial o nset
C hr o m os o mal kar y ot y pe
E C O G P S ( baseli ne)
Me dical hist or y ( V O D/ S O S, G V H D, he patitis, li ver disease, ot her diseases)
Tar get disease of t he i n vesti gati o n
( Pla n ne d) start date of B E S P O N S A treat me nt
8. 8. 2. Hist or y of T re at me nt f or t he T ar get D ise ase at B aseli ne
( 1) Re missi o n i n d ucti o n c he m ot hera p y (re missi o n i n d ucti o n c he m ot hera pe utics, n u m ber of treat me nts, 
a n d res p o nse)
( 2) H S C T (t y pe of tra ns pla ntati o n, date of tra ns pla ntati o n, a n d t y pe of pretreat me nt dr u gs)
T hese ite ms wil l n ot be c ollecte d fr o m tra nsferre d patie nts.
8. 8. 3.
T ar gete d D r u g U se R ec or d (A ct u al T re at me nt R ec or d)
( 1) Date of d osi n g
( 2) Si n gle d ose
( 3) N u m ber of c ycles a d mi nistere d
8. 8. 4.
Rec or ds of B E S P O N S A T re at me nt E n d/ D isc o nti n u ati o n
( 1) W het her or n ot t he treat me nt is c o m plete d or disc o nti n ue d
( 2) Date of c o m pleti o n or disc o nti n uati o n
( 3) Reas o n f or c o m pleti o n or disc o nti n uati o n
( disease pr o gressi o n or w orse ni n g of ge neral c o n diti o n, A Es, n ot ma ki n g a n ot her visit, re missi o n, 
tra nsfer t o a n ot her h os pital or de part me nt, or ot her)
8. 8. 5.
Tre at me nt f or t he T ar get D ise ase after t he S t art of B E S P O N S A T re at me nt
( 1) H S C T (t y pe of tra ns pla ntati o n, date of tra ns pla ntati o n, a n d t y pe of pretreat me nt dr u gs)
( 2) H S C T d o n or i nf or mati o n (alter nati ve d o n or or matc he d relate d d o n or)
( 3) Dr u g t hera p y ( na me of dr u g, start date, a n d r o ute of a d mi nistrati o n)
( 4) N o n -dr u g t hera p y (ra di ot hera p y, plas ma tra nsf usi o n i ncl u de d: na me of t hera p y a n d start date)
8. 8. 6.
Pr o p h yl actic Tre at me nt f or R e d uci n g Inf usi o n R e acti o ns (P rese nce or A bse nce)
8. 8. 7.
Pr o p h yl actic T re at me nt f or P re ve nti n g a n d R e duci n g T u m or L ysis S y n dr o me ( P rese nce or 
A bse nce)
8. 8. 8.
Cli nic al L a b or at or y T ests
( 1) He mat ol o g y (re d bl o o d cell c o u nt, he m o gl o bi n, he mat ocrit, w hite bl o o d cell c o u nt, differe ntial w hite 
bl o o d c o u nt, peri p heral blast cells a n d platelet c o u nt)
( 2) He patic f u ncti o n test ( A S T [ G O T] *, A L T [ G P T] *, A L-P, γ -G T P, t otal bilir u bi n *)
*: Data o n t he u p per li mit of n or mal ( U L N) at t he site will be e ntere d.
( 3) Re nal f u ncti o n test ( B U N, creati ni ne)
( 4) Electr ocar di o gra p h y ( Q T, Q Tc)
0 9 0 1 7 7 e 1 a 2 4 e 5 1 b a\ A p pr o v e d\ A p pr o v e d O n: 0 3- D e c- 2 0 2 4 0 6: 3 3 ( G M T)

I n ot uz u ma b
B 1 9 3 1 0 2 4 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
A me n de d 8 , 12 Dece m ber 2 0 2 3
P FI Z E R C O N FI D E N TI A L
C T 2 4 -WI -J P N-G P S P 0 1 -R F 0 9 8 . 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 -N o v -2 0 2 3
P a ge 1 9 of 3 3
( 5) P a ncreatic f u ncti o n test (a m ylase, li pase)
( 6) B o ne marr o w test ( m yel o blast)
8. 8. 9.
Rec or ds of i n vesti g ati o n disc o nti n u ati o n
F o r patie nts w h o wit h dra w fr o m t he i n vesti gati o n d uri n g t he o bser vati o n peri o d, e nter t he date of 
disc o nti n uati o n. F or t he mai n reas o n f or disc o nti n uati o n, i.e., w h y t he i n vesti gati o n ca n n ot be c o nti n ue d, 
select o ne of t he f oll o wi n g o pti o ns .
( N ot ma ki n g a n ot her visit, deat h, tra nsfer t o a n ot her h os pital or de part me nt, or ot her)
8. 8. 1 0. E C O G P S ( E aster n C o o per ati ve O nc ol o g y Perf or m a nce St at us)
( 1) Date of assess me nt
( 2) Assess me nt res ults
8. 8. 1 1. Cli nic al E v al u ati o n
8. 8. 1 1. 1. He m at ol o gic R e missi o n
Res p o nse will be assesse d acc or di n g t o t he f oll o wi n g res p o nse criteria, a n d t he best o verall res p o nse d uri n g 
t he o bser vati o n peri o d will be e ntere d. H o we ver, if s u bse q ue nt treat me nt f or t he tar get disease is starte d or 
H S C T is perf or me d, t he best o verall r es p o nse bef ore t he start of t he s u bse q ue nt treat me nt or H S C T, 
w hic he ver is earlier, will be e ntere d.
Res p o nse criteria
1.
C R 

N o peri p heral blasts or e xtra me d ullar y disease ( n o l y m p ha de n o pat h y, s ple n o me gal y, or 
s ki n/ g u m i nfiltrati o n/testic ular mass/ C N S i n v ol ve me nt)

Trili nea ge he mat o p oiesis ( T L H) a n d < 5 % blasts

A bs ol ute ne utr o p hil c o u nt ( A N C) > 1, 0 0 0/ L

Platelets > 1 0 0, 0 0 0/ L

N o rec urre nce f or 4 wee ks
2.
C Ri ( C R wit h i nc o m plete c o u nt rec o ver y )

Rec o ver y of platelets b ut < 1 0 0, 0 0 0/ L or A N C is < 1, 0 0 0/L
3.
Pr o gressi ve disease ( P D)

I ncrease of at least 2 5 % i n t he a bs ol ute n u m ber of peri p heral bl o o d or b o ne marr o w blasts 
or de vel o p me nt of e xtra me d ullar y disease
4.
Rela pse d disease

Rea p peara nce of blasts i n t he bl o o d or b o ne marr o w ( > 5 %) or i n a n y e xtra me d ullar y site 
after C R
5.
I ne val ua ble
0 9 0 1 7 7 e 1 a 2 4 e 5 1 b a\ A p pr o v e d\ A p pr o v e d O n: 0 3- D e c- 2 0 2 4 0 6: 3 3 ( G M T)

I n ot uz u ma b
B 1 9 3 1 0 2 4 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
A me n de d 8 , 12 Dece m ber 2 0 2 3
P FI Z E R C O N FI D E N TI A L
C T 2 4 -WI -J P N-G P S P 0 1 -R F 0 9 8 . 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 -N o v -2 0 2 3
P a ge 2 0 of 3 3
8. 8. 1 1. 2. Mi ni m al R esi d u al D ise ase ( M R D)
If a M R D test gi ves a ne gati ve res ult d uri n g t he o bser vati o n peri o d, t he earliest date will be e ntere d. 
H o we ver, if s u bse q ue nt treat me nt f or t he tar get disease is starte d or H S C T is perf or me d, M R D will be 
assesse d bef ore t he start of t he s u bse q ue nt treat me nt or H S C T, w hic he ver is earlier.
8. 8. 1 2. C o nfir m ati o n of S ur vi v al
T he f oll o wi n g i nf or mati o n will be e ntere d t o assess t he o verall s ur vi val d uri n g a peri o d fr o m t he start 
date of B E S P O N S A treat me nt t o t he e n d date of t he o bser vati o n peri o d:
Res ult of c o nfir mati o n of s ur vi val at t he e n d of t he o bser vati o n peri o d, date of c o nfir mi n g t he s ur vi val 
stat us if t he patie nt is ali ve, date of deat h if t he patie nt die d ( w he n t he date of deat h is u n k n o w n, t he last 
dat e of c o nfir mi n g s ur vi val), reas o n f or deat h
8. 8. 1 3. Pre g n a nc y S t at us (fe m ale o nl y)
T he pre g na nc y stat us d uri n g t he peri o d fr o m t he start date of B E S P O N S A treat me nt t o t he e n d date of 
t he o bser vati o n peri o d i n t he treat me nt p hase ( Da y 2 8 p ost-treat me nt) will be e ntere d.
8. 8. 1 4. A d verse E ve nts
Perti ne nt t o safet y assess me nt, t he stat us of A Es d uri n g t he peri o d fr o m t he start of B E S P O N S A 
treat me nt t o t he e n d date of t he o bser vati o n peri o d i n t he treat me nt p hase ( Da y 2 8 p ost-treat me nt) will be 
c o nfir me d, a n d t he f oll o wi n g i nf o r mati o n will be e ntere d. F or V O D/ S O S, e ve nts d uri n g a peri o d fr o m 
t he start of B E S P O N S A treat me nt t o t he e n d date of t he o bser vati o n peri o d i n t he f oll o w-u p p hase will 
be e ntere d. W he n a n A E is o bser ve d, t he i n vesti gat or s h o ul d ta ke a n a p pr o priate t hera pe u tic acti o n a n d 
pr o m ptl y n otif y it t o t he s p o ns or. W he n t he A E is relate d t o B E S P O N S A, its c o urse s h o ul d be f oll o we d 
u p u ntil t he A E or its se q uelae res ol ve or bec o me sta ble t o t he e xte nt t hat is acce pta ble f or t he 
i n vesti gat or a n d s p o ns or.
Als o, f urt her i n vesti gati o n s h o ul d be se paratel y c o n d ucte d, if dee me d necessar y b y sp o ns or f or patie nts 
w h o e x perie nce d a seri o us a d verse reacti o n, a n u ne x pecte d a d verse reacti o n or ot her a d verse reacti o ns 
n ot liste d i n t he pac ka ge i nsert.
( 1) Prese nce/a bse nce of A E
( 2) Na me of A E
( 3) Date of occ urre nce
( 4) C T C A E v 4. 0 Gra de ( w orst gra de)
( 5) I nter ve nti o n
( 6) Seri o us ness
( 7) O utc o me at t he prese nt
( 8) Ca usal relati o ns hi p
N ote: A n A E is a n y a n d all u nfa v ora ble e ve nt (i ncl u di n g cli nicall y si g nifica nt a b n or mal la b orat or y 
c ha n ge) occ urri n g i n a patie nt after starti n g B E S P O N S A treat me nt re gar dless of ca usal relati o ns hi p. A 
seri o us a d verse e ve nt ( S A E) is a n y u nfa v ora ble me dical occ urre nce t hat res ults i n deat h, is life-
t hreate ni n g, re q uires ( or pr ol o n gs) h os pitalizati o n, ca uses persiste nt or si g nifica nt disa bilit y/i nca pacit y, 
res ults i n c o n ge nital a n o malies or birt h defects, or is ot her c o n diti o ns w hic h re prese nt si g nifica nt healt h 
0 9 0 1 7 7 e 1 a 2 4 e 5 1 b a\ A p pr o v e d\ A p pr o v e d O n: 0 3- D e c- 2 0 2 4 0 6: 3 3 ( G M T)

I n ot uz u ma b
B 1 9 3 1 0 2 4 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
A me n de d 8 , 12 Dece m ber 2 0 2 3
P FI Z E R C O N FI D E N TI A L
C T 2 4 -WI -J P N-G P S P 0 1 -R F 0 9 8 . 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 -N o v -2 0 2 3
P a ge 2 1 of 3 3
hazar ds.
8. 9.
D at a S o urces 
I n t his i n vesti gati o n, t he i n vesti gat ors will e xtract necessar y i nf or mati o n fr o m t he me dical rec or ds i n 
acc or da nce wit h t he pr ot oc ol.
8. 1 0. St u d y Size
8. 1 0. 1. Pl a n ne d S a m ple S ize
T he s u bjects are patie nts w h o recei ve d B E S P O N S A. T he pla n ne d n u m ber of patie nts is 1 7 6 patie nts wit h 
rela pse d or refract or y C D 2 2-p ositi ve A L L t o be a nal yze d f or safet y. C o nsi deri n g patie nts t o be e xcl u de d 
a n d dr o p o uts, 2 0 0 e nr olle d patie nts will be c ollecte d.
8. 1 0. 2. R ati o n ale 
A t otal of 1 7 6 patie nts was c h ose n as t he n u m ber of patie nts e val ua ble f or ris k fact ors relate d t o t he o nset 
of V O D/ S O S. T he i nci de nce of V O D/ S O S was 1 2. 3 % ( 2 6/ 2 1 2 patie nts ) i n clinical st u dies ( St u dies 
B 1 9 3 1 0 2 2 a n d B 1 9 3 1 0 1 0).
A sa m ple size e na bli n g t o e val uate t he effect of ris k fact ors relate d t o t he o nset of V O D/ S O S, w hic h 
were i de ntifie d i n cli nical st u dies, u n der act ual c o n diti o ns of use, a n d t o searc h ot her ris k fact ors was 
selecte d. Ass u mi n g t he tr ue i nci de nce of 1 2. 3 % i n all patie nts, a tr ue relati ve ris k ass ociate d wit h t he 
o nset of e ve nts i n s u b gr o u ps of 3. 0 (e q ui vale nt t o t he i nci de nce i n t he hi g h a n d l o w ris k gr o u ps bei n g 
ass u me d t o be 1 8. 5 % a n d 6. 2 %, res pecti vel y), a n d a rati o of t he patie nts i n eac h gr o u p bei n g 1: 1, w he n 
defi ni n g a pr o ba bilit y of detecti n g a statisticall y si g nifica nt relati ve ris k as p o wer, a sa m ple size of 1 7 6 
patie nts w o ul d pr o vi de p o wer of 8 0. 4 % i n a (t w o -si de d) c hi s q uare test wit h a si g nifica nce le vel of 0. 1.
8. 1 1. D at a M a n a ge me nt
8. 1 1. 1. C ase R e p ort F or ms ( C R Fs) / Electr o nic D at a R ec or ds
T he C R F i n t his pr ot oc ol refers t o eit her a pa per f or m or a n electr o nic data rec or d, or b ot h, de pe n di n g o n t he 
data c ollecti o n met h o d d uri n g t his i n vesti gati o n.
A C R F will be c o m plete d f or eac h patie nt re gistere d i n t his i n vesti gati o n. All ori gi nal c o m plete d C R Fs are t he 
s ole pr o pert y of Pfizer a n d s h o ul d n ot be ma de a vaila ble i n a n y f or m t o t hir d parties, e xce pt f or a ut h orize d 
re prese ntati ves of Pfizer or a p pr o priate re g ulat or y a ut h orities, wit h o ut writte n per missi o n fr o m Pfizer. T he 
i n vesti gat or s h o ul d e ns ure t hat t he C R Fs will be sec urel y st ore d at t he site i n pa per or e ncr y pte d electr o nic 
f or ms a n d pr otecte d b y pass w or ds or st ore d i n a l oc ke d r o o m t o pre ve nt access b y  u na ut h orize d t hir d parties.
T he i n vesti gat or will be res p o nsi ble f or c ollecti n g a n d re p orti n g all cli nical, safet y, a n d la b orat or y data e ntere d 
i n t he C R Fs a n d a n y ot her data c ollecti o n f or ms (s o urce d oc u me nts) a n d e ns uri n g t hat t he y are acc urate, 
a ut hentic/ ori gi nal, attri b uta ble, c o m plete, c o nsiste nt, le gi ble, ti mel y (c o nte m p ora ne o us), e n d uri n g, a n d 
a vaila ble w he n re q uire d. T he C R Fs m ust be si g ne d b y t he i n vesti gat or or b y a n a ut h orize d pers o n. T hese 
si g nat ures certif y t hat t he i nf or mati o n rec or de d i n the C R Fs is tr ue. A n y c orrecti o n ma de t o t he e ntries i n t he 
C R F s or s o urce d oc u me nts m ust be date d, i nitiale d or sta m pe d, a n d e x plai ne d (if necessar y) a n d s h o ul d n ot 
o bsc ure t he ori gi nal e ntr y.
A s o urce d oc u me nt is a patie nt’s me dical rec or d t hat is st ore d at t he site or o w ne d b y a p h ysicia n. I n t his case, 
t he i nf or mati o n rec or de d i n t he C R F m ust be c o nsiste nt wit h t he i nf or mati o n i n t hese me dical rec or ds.
0 9 0 1 7 7 e 1 a 2 4 e 5 1 b a\ A p pr o v e d\ A p pr o v e d O n: 0 3- D e c- 2 0 2 4 0 6: 3 3 ( G M T)

I n ot uz u ma b
B 1 9 3 1 0 2 4 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
A me n de d 8 , 12 Dece m ber 2 0 2 3
P FI Z E R C O N FI D E N TI A L
C T 2 4 -WI -J P N-G P S P 0 1 -R F 0 9 8 . 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 -N o v -2 0 2 3
P a ge 2 2 of 3 3
8. 1 1. 2. Rec or d R ete nti o n
T he site will retai n t h e rec or ds relate d t o t his i n vesti gati o n u ntil t he c o m pleti o n of t his i n vesti gati o n is re p orte d 
b y  Pfizer or u ntil t he dea dli ne s pecifie d b y t he site, w hic he ver is l o n ger.
8. 1 1. 3. D at a C ollecti o n M et h o d
I n t his i n vesti gati o n, data will be e ntere d i n C R Fs a n d e val uate d usi n g t he E D C s yste m f or c ollecti n g p ost-
mar keti n g dr u g i n vesti gati o n data ( herei nafter referre d t o as t he s yste m) o n t he I nter net s u p plie d b y t he 
s p o ns or. H o we ver, o nl y w he n t he E D C s yste m ca n n ot be use d at t he sites d ue t o c o m pelli n g reas o ns, data wi ll 
be c ollecte d usi n g pa per C R Fs. T he i n vesti gat or will c o m plete C R Fs wit hi n t he i n vesti gati o n peri o d after t he 
e n d of t he o bser vati o n peri o d f or eac h b o o klet a n d s u b mit t he m t o t he s p o ns or.
8. 1 1. 4. P atie nt R e gistr ati o n
Pr oce d ure f or patie nt re gistrati o n: T he i n vesti gat or will e nter re gistrati o n ite ms o n t he patie nt re gistrati o n 
scree n of t he s yste m a n d se n d t he data after di gital si g ni n g. H o we ver, if pa per C R Fs are t o be use d, t he 
i n vesti gat or will c o m plete t he re gistrati o n f or m pr o vi de d b y t he s p o ns or, a n d s u b mit t he f or m via F A X usi n g 
t he t oll-free n u m ber pr o vi de d i n t he re gistrati o n f or m.
8. 1 1. 5. P oi nts t o C o nsi der f or C o m pleti o n, R e visi o n, a n d S u b missi o n of C ase R e p ort F or m
8. 1 1. 5. 1. D at a E ntr y
T he i n vesti gat or will, u p o n c o nfir mi n g t h e s ur ve y ite ms, c o m plete t he C R F base d o n me dical c harts a n d ot her 
me dical rec or ds, electr o nicall y si g n t he f or m a n d se n d it. H o we ver, if pa per C R Fs are t o be use d, after all t he 
s ur ve y ite ms are e ntere d a n d c orrecte d, t he i n vesti gat or s h o ul d re vie w t he c o m plete d C R Fs a n d t he data 
e ntere d i n t he q uer y f or ms a gai n, rec or d his/ her na me a n d affi x a seal or si g n t he C R Fs. T he C R Fs a n d q uer y 
f or ms s h o ul d be s u b mitte d i n acc or da nce wit h met h o ds s pecifie d b y t he s p o ns or.
8. 1 1. 5. 2. R e visi o n
U p o n recei vi n g t he s p o ns or’s i n q uir y o n t he c o nte nts of t he C R F ( q uer y f or ms), t he i n vesti gat or will a gai n 
c o nfir m t he c o nte nts of me dical rec or ds descri be d earlier, a n d as re q uire d, c orrect rele va nt secti o ns a n d 
res u b mit t he f or m.
8. 1 1. 5. 3. S u b m issi o n
T he C R Fs s h o ul d be s u b mitte d pr o m ptl y u p o n c o m pleti o n i n acc or da nce wit h t he pr oce d ures set o ut b y t he 
s p o ns or. H o we ver, if pa per C R Fs are t o be use d, after all t he s ur ve y ite ms are e ntere d a n d c orrecte d, t he 
i n vesti gat or s h o ul d re vie w t he c o m plete d C R Fs a n d t he data e ntere d i n t he q uer y f or ms a gai n, rec or d his/ her 
na me a n d affi x a seal or si g n t he C R Fs. T he C R Fs a n d q uer y f or ms s h o ul d be s u b mitte d i n acc or da nce wit h 
met h o ds s pecifie d b y t he s p o ns or.
8. 1 2. D at a A n al ysis
S u m maries of data t o be c ollecte d i n t his i n vesti gati o n a n d detaile d statistical a nal ysis met h o ds will be 
descri be d i n t he Statistical A nal ysis Pla n ( S A P) f or t his i n vesti gati o n, w hic h will be date d a n d retai ne d b y 
Pfizer. 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; h o w e ver, a n y maj or m o dificati o ns of t he 
pri mar y e n d p oi nt defi niti o n or its a nal ysis will als o be reflecte d i n a pr ot oc ol a me n d me nt.
0 9 0 1 7 7 e 1 a 2 4 e 5 1 b a\ A p pr o v e d\ A p pr o v e d O n: 0 3- D e c- 2 0 2 4 0 6: 3 3 ( G M T)

I n ot uz u ma b
B 1 9 3 1 0 2 4 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
A me n de d 8 , 12 Dece m ber 2 0 2 3
P FI Z E R C O N FI D E N TI A L
C T 2 4 -WI -J P N-G P S P 0 1 -R F 0 9 8 . 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 -N o v -2 0 2 3
P a ge 2 3 of 3 3
8. 1 2. 1. Defi niti o n of A n al ysis S ets
( 1)
A safet y set will be c o m p ose d of patie nts re gistere d i n t his i n vesti gati o n w h o are c o nfir me d t o ha ve 
recei ve d at least o ne d ose of B E S P O N S A.
( 2)
A n efficac y set will be se paratel y s pecifie d i n t he S A P.
8. 1 2. 2. A n al ysis M et h o ds
8. 1 2. 2. 1. A n al ysis of S afet y E n d p oi nts
T he occ urre nce of A D Rs will be ta b ulate d. I n a d diti o n t o a n a nal ysis usi n g t he e ntire safet y set, a nal yses i n 
s u b gr o u ps stratifie d acc or di n g t o patie nt bac k gr o u n d c haracteristics will be carrie d o ut. T he occ urre nce of li ver 
dis or der i ncl u di n g V O D/ S O S, m yel os u p pressi o n , i nfecti o ns, he m orr ha ge , t u m or l ysis s yn dr o me, i nf u si o n 
reacti o n, pa ncreatitis, i nterstitial l u n g disease, Q Tc i nter val pr ol o n ge d, i nfla m mat or y gastr oi ntesti nal e ve nts 
a n d re pr o d ucti ve t o xicit y will be ta b ulate d. F or patie nts wit h s pecial bac k gr o u n d c haracteristics s uc h as 
c hil dre n, el derl y patie nts, pre g n a nt w o me n, patie nts wit h re nal i m pair me nt a n d patie nts wit h he patic 
d y sf u ncti o n, t he occ urre nce will be ta b ulate d. Ris k fact ors relate d t o t he de vel o p me nt of V O D/ S O S a n d ris k 
fact ors relate d t o earl y deat h of H S C T patie nts after B E S P O N S A treat me nt will be assesse d.
T he details of t he a nal ysis pla n are s pecifie d i n t he S A P.
8. 1 2. 2. 2. A n al ysis of E ffic ac y E n d p oi nts
T he mai n e n d p oi nts will be he mat ol o gic c o m plete re missi o n a n d o verall s ur vi val. E x pl orat or y a nal yses s uc h 
as s u b gr o u p a nal ysis b y patie nt bac k gr o u n d c haracteristic will be, as necessar y, perf or me d.
T he details of t he a nal ysis pla n are s pecifie d i n t he S A P.
8. 1 3. Q u alit y C o ntr ol
T he staff res p o nsi ble f or me dical i nstit uti o ns will e x plai n t he c o nte nts of t he pr ot oc ol, etc. t o t he i n vesti gat or 
pri or t o t he i m pl e me ntati o n of t his i n vesti gati o n a n d as k t he i n vesti gat or t o pre pare C R Fs base d o n me dical 
rec or ds.
8. 1 4. Li mit ati o ns of t he R ese arc h M et h o ds
T he f oll o wi n g matters are c o nsi dere d f or t his i n vesti gati o n:
1) Si nce n o c o ntr ol gr o u p is set i n t he i n vesti gati o n, t here is a li mit t o t he j u d g me nt o n w het her or n ot a 
ris k of de vel o pi n g A Es a n d A D Rs i ncreases d ue t o t he a d mi nistrati o n of B E S P O N S A.
2) T he c o nsi derati o n f or c o nf o u n di n g fact ors ma y n ot be a de q uate be ca use t he bac k gr o u n d i nf or mati o n 
ma y n ot be s ufficie ntl y o btai ne d.
3) Si nce t his is a n i n vesti gati o n t hat c ollects t he i nf or mati o n descri be d i n me dical c harts, t he set data ma y 
n ot be c ollecte d or t here ma y be missi n g i nf or mati o n.
8. 1 5.
Ot her As pects
N ot a p plica ble
0 9 0 1 7 7 e 1 a 2 4 e 5 1 b a\ A p pr o v e d\ A p pr o v e d O n: 0 3- D e c- 2 0 2 4 0 6: 3 3 ( G M T)

I n ot uz u ma b
B 1 9 3 1 0 2 4 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
A me n de d 8 , 12 Dece m ber 2 0 2 3
P FI Z E R C O N FI D E N TI A L
C T 2 4 -WI -J P N-G P S P 0 1 -R F 0 9 8 . 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 -N o v -2 0 2 3
P a ge 2 4 of 3 3
9.
P R O T E C TI O N O F H U M A N P A R TI CI P A N T S
9. 1.
P atie nt I nf or m ati o n
All parties will c o m pl y wit h all a p plica ble la ws, i ncl u di n g la ws re gar di n g t he i m ple me ntati o n of or ga nizati o nal 
a n d tec h nical meas ures t o e ns ure pr otecti o n of patie nt pers o nal data. S uc h meas u res will i ncl u de o mitti n g 
patie nt na mes or ot her directl y i de ntifia ble data i n a n y re p orts, p u blicati o ns, or ot her discl os ures, e xce pt w here 
re q uire d b y a p plica ble la ws. T he pers o nal data will be st ore d at t he st u d y site i n e ncr y pte d electr o nic a n d/ or 
pa pe r f or m a n d will be pass w or d pr otecte d or sec ure d i n a l oc ke d r o o m t o e ns ure t hat o nl y a ut h orize d st u d y 
staff ha ve access. T he st u d y site will i m ple me nt a p pr o priate tec h nical a n d or ga nizati o nal meas ures t o e ns ure 
t hat t he pers o nal data ca n be rec o vere d i n t he e ve nt of disaster. I n t he e ve nt of a p ote ntial pers o nal data breac h, 
t he st u d y site s hall be res p o nsi ble f or deter mi ni n g w het her a pers o nal data breac h has i n fact occ urre d a n d, if 
s o, pr o vi di n g breac h n otificati o ns as re q uire d b y la w.
T o pr otect t he ri g hts a n d free d o ms of nat ural pers o ns wit h re gar d t o t he pr ocessi n g of pers o nal data, w he n 
st u d y data are c o m pile d f or tra nsfer t o Pfizer a n d ot her a ut h orize d parties, patie nt na mes will be re m o ve d a n d 
will be re place d b y a si n gle, s pecific, n u merical c o de, base d o n a n u m beri n g s yste m defi ne d b y Pfizer. All 
ot her i de ntifia ble data tra nsferre d t o Pfizer or ot her a ut h orize d parties will be i de ntifie d b y t his si n gle, patie nt -
s pecific c o de. T he i n vesti gat or site will mai ntai n a c o nfi de ntial list of patie nts w h o partici pate d i n t he st u d y, 
li n ki n g eac h patie nt’s n u merical c o de t o his or her act ual i de ntit y. I n case of data tra nsfer, Pfizer will mai ntai n 
hi g h sta n dar ds of c o nfi de ntialit y a n d pr otecti o n of patie nts’ pers o nal data c o nsiste nt wit h t he cli nical st u d y 
a gree me nt a n d a p plica ble pri vac y la ws.
9. 2.
P atie nt C o nse nt
As t his st u d y d oes n ot i n v ol ve data s u bject t o pri vac y la ws acc or di n g t o a p plica ble le gal re q uire me nts, or it is 
a n i nf or mati o n pr o visi o n base d o n t he la w (e ve n t h o u g h t hat i n v ol ve s data s u bject t o pri va c y la ws acc or di n g t o 
a p plica ble le gal re q uire me nts), o btai ni n g i nf or me d c o nse nt fr o m patie nts b y Pfizer is n ot re q uire d wit h re gar d 
t o t he pers o nal i nf or mati o n pr o visi o n fr o m t he st u d y site t o Pfizer. Als o, beca use t he re p ort of i nf or mati o n or 
res ults c ollecte d i n t his st u d y t o t he l ocal re g ulat or y a ut h orit y or healt hcare pr o vi ders b y Pfizer as nee de d is a n 
i nf or mati o n pr o visi o n base d o n t he la w, o btai ni n g i nf or me d c o nse nt fr o m patie nts b y Pfizer is n ot re q uire d.
I n t his st u d y, Pfizer will c ollect i nf or matio n t hat ca n n ot i de ntif y s pecific patie nts fr o m t he i nstit uti o ns. T he 
res ults of t his st u d y, w hic h are pre pare d n ot t o i de ntif y s pecific patie nts, ma y be re p orte d t o Pfizer I nc., w hic h 
is t he c or p orate pare nt of t he s p o ns or of t his st u d y, or gr o u p c o m pa nies, or re g ulat or y a ut h orities i n ot her 
c o u ntries, as nee de d, or p u blis he d it as a prese ntati o n at aca de mic c o nfere nces or ma n uscri pt f or t he p ur p ose of 
pr o vi di n g pr o per use i nf or mati o n f or t his dr u g. If t his i nf or mati o n falls u n der pers o nal i nf or mati o n of t h e 
Pers o nal I nf or mati o n Pr otecti o n Act, t hese acti o ns ma y n ot be base d o n t he la ws or re g ulati o ns, a n d t heref ore, 
ma y c orres p o n d t o pr o visi o n t o t he t hir d part y a n d usi n g t he i nf or mati o n f or p ur p oses ot her t ha n b usi ness t hat 
re q uires c o nse nt fr o m t he patie nt. T heref ore, t he st u d y i nstit uti o ns will o btai n writte n or ver bal c o nse nt fr o m 
t he patie nts t o be i ncl u de d i n t his st u d y s o t hat Pfizer ca n use t he res ults of t his st u d y t o re p ort t o Pfizer I nc., 
gr o u p c o m pa nies or re g ulat or y a ut h o rities i n ot her c o u ntries, or t o prese nt it at aca de mic c o nfere nces or 
p u blis h ma n uscri pt, etc. W het her c o nse nt is o btai ne d fr o m patie nts or n ot is descri be d i n t he C R F. T he ori gi nal 
of t he writte n i nf or me d c o nse nt f or m s h o ul d be retai ne d b y t he st u d y i n ve sti gat or.
I n ge neral, t he i n vesti gat or m ust o btai n c o nse nt fr o m a patie nt pers o nall y. H o we ver, if t he i n vesti gat or 
deter mi nes t hat a patie nt’s decisi o nal ca pacit y is s o li mite d t hat he or s he ca n n ot reas o na bl y be c o ns ulte d or he 
or s he is a mi n or, c o nse nt is o btai ne d fr o m le gall y acce pta ble re prese ntati ve or pare nt(s).
0 9 0 1 7 7 e 1 a 2 4 e 5 1 b a\ A p pr o v e d\ A p pr o v e d O n: 0 3- D e c- 2 0 2 4 0 6: 3 3 ( G M T)

I n ot uz u ma b
B 1 9 3 1 0 2 4 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
A me n de d 8 , 12 Dece m ber 2 0 2 3
P FI Z E R C O N FI D E N TI A L
C T 2 4 -WI -J P N-G P S P 0 1 -R F 0 9 8 . 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 -N o v -2 0 2 3
P a ge 2 5 of 3 3
9. 3.
I nstit uti o n al Re vie w B o ar d (I R B)/ Et hics C o m mittee ( E C)
I n t his i n vesti gati o n, t he re vie w b y t he I nstit uti o nal Re vie w B oar d (I R B)/ Et hics C o m mittee ( E C) is n ot 
esse ntial.
9. 4.
Et hic al C o n d uct of t h e St u d y
T his i n vesti gati o n is wit hi n t he sc o pe of t he “ Mi nisterial Or di na nce o n G o o d P ost -ma r keti n g St u d y Practice f or 
Dr u gs” ( M H L W Or di na nce N o. 1 7 1 date d Dece m ber 2 0, 2 0 0 4) a n d will be c o n d ucte d i n acc or da nce wit h le gal 
a n d re g ulat or y re q uire me nts, as we ll as scie ntific o bjecti ves, val ues, a n d ri g or.
1 0. 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 D R U G R E A C TI O N S
1 0. 1. Re q uire me nts
T he ta ble bel o w s u m marizes t he re q uire me nts f or rec or di n g safet y e ve nts o n t he C R F  a n d f or re p orti n g safet y 
e ve nts o n t he n o n -i nter ve nti o nal st u d y ( NI S) a d verse e ve nt m o nit ori n g ( A E M) Re p ort F or m t o Pfizer Safet y.  
T hese re q uire me nts are deli neate d f or t hree t y pes of e ve nts: ( 1) seri o us a d verse e ve nts ( S A Es); ( 2) n o n -seri o us 
A Es (as a p plica ble); a n d ( 3) sce nari os i n v ol vi n g e x p os ure t o a Pfizer pr o d uct, i ncl u di n g e x p os ure d uri n g 
pre g na nc y, e x p os ure d uri n g breastfee di n g , me dicati o n err or, o ver d ose, mis use, e xtra vasati o n, lac k of efficac y, 
a n d occ u pati o nal e x p os ure.  T hese e ve nts are defi ne d i n t he secti o n “ Defi niti o ns of S afet y E ve nts”.
S afet y e ve nt
Rec or de d o n t he C R Fs
Re p orte d o n t he NI S A E M Re p ort 
F or m t o Pfizer S afet y wit hi n 2 4 h o urs 
of a w are ness
S A E
All 
All
N o n -seri o us A E
All
N o ne
Sce nari os i n v ol vi n g e x p os ure 
t o a Pfizer pr o d uct, i ncl u di n g 
e x p os ure d uri n g pre g na nc y, 
e x p os ure d uri n g 
breastfee di n g , me dicati o n 
err or, o ver d ose, mis use, 
e xtra vasati o n, lac k of 
efficac y, a n d occ u pati o nal 
e x p os ure
All (re gar dless of w het her 
ass ociate d wit h a n A E), e xce pt 
occ u p ati o n al e x p os ure
All (re gar dless of w het her ass ociate d wit h 
a n A E) i n v ol vi n g e x p os ure t o a Pfizer 
pr o d uct
N ote: A n y ass ociate d A E is re p orte d 
t o get her wit h t he e x p os ure sce nari o.
F o r eac h A E, t he i n vesti gat or m ust p urs ue a n d o btai n i nf or mati o n a de q uate b ot h t o deter mi ne t he o utc o me of 
t he A E a n d t o assess w het her it meets t he criteria f or classificati o n as a S A E (see secti o n " Seri o us A d verse 
E ve nts” bel o w).
Safet y  e ve nts liste d i n t he ta ble a b o ve m ust be re p orte d t o Pfizer wit hi n 2 4 h o urs of a ware ness of t he e ve nt b y 
t he i n vesti gat or re g ar dless of w het her t he e ve nt is deter mi ne d b y t he i n vesti g at or t o be rel ate d t o t his 
dr u g .   I n partic ular, if t he S A E is fatal or life-t hreate ni n g, n otificati o n t o Pfizer m ust be ma de i m me diatel y, 
0 9 0 1 7 7 e 1 a 2 4 e 5 1 b a\ A p pr o v e d\ A p pr o v e d O n: 0 3- D e c- 2 0 2 4 0 6: 3 3 ( G M T)

I n ot uz u ma b
B 1 9 3 1 0 2 4 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
A me n de d 8 , 12 Dece m ber 2 0 2 3
P FI Z E R C O N FI D E N TI A L
C T 2 4 -WI -J P N-G P S P 0 1 -R F 0 9 8 . 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 -N o v -2 0 2 3
P a ge 2 6 of 3 3
irres pecti ve of t he e xte nt of a vaila ble e ve nt i nf or mati o n.  T his ti mefra me als o a p plies t o a d diti o nal ne w 
(f oll o w-u p) i nf or mati o n o n pre vi o usl y f or war de d safet y e ve nt re p orts.  I n t he rare sit uati o n t hat t he i n vesti gat or 
d oes n ot bec o me i m me diatel y a ware of t he occ urre nce of a safet y e ve nt, t he i n ves ti gat or m ust re p ort t he e ve nt 
wit hi n 2 4 h o urs after lear ni n g of it a n d d oc u me nt t he ti me of his/ her first a ware ness of t he e ve nts.
F o r t h ose safet y e ve nts t hat are c o nsi dere d seri o us or t hat are i de ntifie d i n t he far ri g ht c ol u m n of t he ta ble 
a b o ve t hat are re p orta ble t o Pfizer wit hi n 2 4 h o urs of a ware ness, t he i n vesti gat or is o bli gate d t o p urs ue a n d t o 
pr o vi de a n y a d diti o nal i nf or mati o n t o Pfizer i n acc or da nce wit h t his 2 4 -h o ur ti mefra me.  I n a d diti o n, a n 
i n vesti gat or ma y be re q ueste d b y Pfizer t o o btai n specific f oll o w -u p i nf or mati o n i n a n e x pe dite d fas hi o n.  T his 
i nf or mati o n is m ore detaile d t ha n t hat rec or de d o n t he C R F .  I n ge neral, t his will i ncl u de a descri pti o n of t he 
A E i n s ufficie nt detail t o all o w f or a c o m plete me dical assess me nt of t he case a n d i n de pe n de nt deter mi nati o n 
of p ossi ble ca usalit y.  I nf or mati o n rele va nt t o t he e ve nt, s uc h as c o nc o mita nt me dicati o ns a n d ill nesses m ust be 
pr o vi de d.  I n t he case of a patie nt deat h, a s u m mar y of a vaila ble a ut o ps y fi n di n gs m ust be s u b mitte d as s o o n as 
p ossi ble t o Pfizer or its desi g nate d re prese ntati ve.
1 0. 2. Re p orti n g Peri o d
F o r eac h patie nt, t he peri o d of safet y e ve nt re p orti n g i n C R F (see t he ta ble a b o ve) be gi ns at t he ti me of t he 
patie nt’s first d ose of B E S P O N S A, a n d lasts t hr o u g h t he e n d of t he o bser vati o n peri o d ( t his refers t o t he e n d 
date of o bser vati o n peri o d i n t he treat me nt p hase ) of t he st u d y, b ut t he re p orti n g peri o d t o Pfizer Safet y m ust 
i ncl u de at least 2 8 cale n dar da ys f oll o wi n g t he last d ose of B E S P O N S A ; a re p ort m ust be s u b mitte d t o Pfizer 
Safe t y ( or its desi g nate d re prese ntati ve) f or a n y of t he t y pes of safet y e ve nts liste d i n t he ta ble a b o ve occ urri n g 
d uri n g t his peri o d. If a patie nt was recei vi n g B E S P O N S A o n t he last da y of t he o bser vati o n peri o d, t he n t he 
re p orti n g peri o d s h o ul d be e xte n de d f or 2 8 cale n dar da ys f oll o wi n g t he e n d of o bser vati o n peri o d .
D uri n g t he V O D/ S O S re p orti n g peri o d, all V O D/ S O S occ urre d bef ore t he e n d date of t he o bser vati o n peri o d i n 
t he f oll o w-u p p hase s hall be re p orte d as S A Es re gar dless of t he ca usal relati o ns hi p a n d se verit y.
If t he i n vesti gat or bec o mes a ware of a S A E occ urri n g at a n y ti me after c o m pleti o n of t he st u d y (t his refers t o 
t he e n d date of t he o bser vati o n peri o d i n t he treat me nt p hase) a n d s/ he c o nsi ders t he S A E t o be relate d t o 
B E S P O N S A, t he S A E als o m ust be re p orte d t o Pfizer Safet y.
1 0. 3.
C a us alit y A ssess me nt
T he i n vesti gat or is re q uire d t o assess a n d rec or d t he ca usal relati o ns hi p.  F or all A Es, s ufficie nt i nf or mati o n 
s h o ul d be o btai ne d b y t he i n vesti gat or t o deter mi ne t he ca usalit y of eac h A E.  F or A Es wit h a ca usal 
relati o ns hi p t o B E S P O N S A , f oll o w-u p b y t he i n vesti gat or is re q uire d u ntil t he e ve nt a n d/ or its se q uelae res ol ve 
or sta bilize at a le vel acce pta ble t o t he i n vesti gat or, a n d Pfizer c o nc urs wit h t hat assess me nt.  
A n i n vesti gat or’s ca usalit y assess me nt is t he deter mi nati o n of w het her t here e xists a reas o na ble p ossi bilit y t hat 
B E S P O N S A ca use d or c o ntri b ute d t o a n A E.  If t he i n vesti gat or’s fi nal deter mi nati o n of ca usalit y is 
“ u n k n o w n” a n d s/ he ca n n ot deter mi ne w het her B E S P O N S A ca use d t he e ve nt, t he safet y e ve nt m ust be 
re p orte d wit hi n 2 4 h o urs. 
If t he i n vesti gat or ca n n ot deter mi ne t he eti ol o g y of t he e ve nt b ut s/ he deter mi nes t hat B E S P O N S A di d n ot 
ca use t he e ve nt, t his s h o ul d be clearl y d oc u me nte d o n t he  C R F a n d t he NI S A E M Re p ort F or m.
0 9 0 1 7 7 e 1 a 2 4 e 5 1 b a\ A p pr o v e d\ A p pr o v e d O n: 0 3- D e c- 2 0 2 4 0 6: 3 3 ( G M T)

I n ot uz u ma b
B 1 9 3 1 0 2 4 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
A me n de d 8 , 12 Dece m ber 2 0 2 3
P FI Z E R C O N FI D E N TI A L
C T 2 4 -WI -J P N-G P S P 0 1 -R F 0 9 8 . 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 -N o v -2 0 2 3
P a ge 2 7 of 3 3
1 0. 4. Defi niti o ns of S afet y E ve nts
1 0. 4. 1. A d verse E ve nts
A n A E is a n y u nt o war d me dical occ urre nce i n a patie nt a d mi nistere d a me dici nal pr o d uct.  T he e ve nt nee d n ot 
necessaril y ha ve a ca usal relati o ns hi p wit h t he pr o d uct treat me nt or usa ge . E xa m ples of A Es i ncl u de b ut are 
n ot li mite d t o: 

A b n or mal test fi n di n gs (see bel o w f or circ u msta nces i n w hic h a n a b n or mal test fi n di n g c o nstit utes a n A E);

Cli nicall y si g nifica nt si g ns a n d s y m pt o ms;

C ha n ges i n p h ysical e xa mi nati o n fi n di n gs;

H y perse nsiti vi t y;

Lac k of efficac y; 

Dr u g a b use; 

Dr u g de pe n de nc y.
A d diti o nall y, f or me dici nal pr o d ucts, t he y ma y i ncl u de t he si g ns or s y m pt o ms res ulti n g fr o m:

Dr u g o ver d ose;

Dr u g wit h dra wal;

Dr u g mis use; 

Off -la bel use; 

Dr u g i nteracti o ns;

E xtra vasati o n;

E x p os ure d uri n g pre g na nc y;

E x p os ure d uri n g breastfee di n g ;

Me dicati o n err or;

Occ u pati o nal e x p os ure.
A b n or mal test fi n di n gs
T he criteria f or deter mi ni n g w het her a n a b n or mal test fi n di n g s h o ul d be re p orte d as a n A E are as f oll o ws: 

Test res ult is ass ociate d wit h acc o m pa n yi n g s y m pt o ms, a n d/ or

Test res ult re q uires a d diti o nal dia g n ostic testi n g or me dical/s ur gical i nter ve nti o n, a n d/ or

Test res ult lea ds t o a c ha n ge i n B E S P O N S A d osi n g or disc o nti n uati o n fr o m t he st u d y, si g nifica nt 
a d diti o nal c o nc o mita nt dr u g treat me nt, or ot her t hera p y, a n d/ or

Test res ult is c o nsi dere d t o be a n A E b y t he i n vesti gat or or s p o ns or.
Merel y re peati n g a n a b n or mal test, i n t he a bse nce of a n y of t he a b o ve c o n diti o ns, d oes n ot c o nstit ute a n A E.  
A n y  a b n or mal test res ult t hat is deter mi ne d t o be a n err or d oes n ot re q uire re p orti n g as a n A E .
1 0. 4. 2. Seri o us A d verse E ve nts
A S A E is a n y u nt o war d me dical occ urre nce i n a patie nt a d mi nistere d a me dici nal or n utriti o nal pr o d uct 
(i ncl u di n g pe diatric f or m ulas) at a n y d ose t hat:

R es ults i n deat h;

Is life-t hreate ni n g;

Re q uires i n patie nt h os pitalizati o n or pr ol o n gati o n of h os pitalizati o n (see bel o w f or circ u msta nces t hat d o 
n ot c o nstit ute A Es);
0 9 0 1 7 7 e 1 a 2 4 e 5 1 b a\ A p pr o v e d\ A p pr o v e d O n: 0 3- D e c- 2 0 2 4 0 6: 3 3 ( G M T)

I n ot uz u ma b
B 1 9 3 1 0 2 4 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
A me n de d 8 , 12 Dece m ber 2 0 2 3
P FI Z E R C O N FI D E N TI A L
C T 2 4 -WI -J P N-G P S P 0 1 -R F 0 9 8 . 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 -N o v -2 0 2 3
P a ge 2 8 of 3 3

Res ults i n persiste nt or si g nifica nt disa bilit y/i nca pacit y (s u bsta ntial disr u pti o n of t he a bilit y t o c o n d uct
n or mal life f u ncti o ns);

Res ults i n c o n ge nital a n o mal y/ birt h defect .
Pr o gressi o n of t he mali g na nc y u n der st u d y (i ncl u di n g si g ns a n d s y m pt o ms of pr o gressi o n) s h o ul d n ot be 
re p orte d as a S A E u nless t he o utc o me is fatal wit hi n t he safet y re p orti n g peri o d.  H os pitalizati o n d ue t o si g ns 
a n d s y m pt o ms of disease pr o gressi o n s h o ul d n ot be re p orte d as a S A E.  If t he mali g na nc y has a fatal o utc o me 
d uri n g t he st u d y or wit hi n t he safet y re p orti n g peri o d, t he n t he e ve nt lea di n g t o deat h m ust be rec or de d as a n 
A E a n d a s a S A E wit h se verit y Gra de 5.
Me dical a n d scie ntific j u d g me nt is e xercise d i n deter mi ni n g w het her a n e ve nt is a n i m p orta nt me dical e ve nt.  
A n i m p orta nt me dical e ve nt ma y n ot be i m me diatel y life -t hreate ni n g a n d/ or res ult i n deat h or h os pitalizati o n.  
H o we v er, if it is deter mi ne d t hat t he e ve nt ma y je o par dize t he patie nt or ma y re q uire i nter ve nti o n t o pre ve nt 
o ne of t he ot her o utc o mes liste d i n t he defi niti o n a b o ve, t he i m p orta nt me dical e ve nt s h o ul d be re p orte d as 
seri o us.
E xa m ples of s uc h e ve nts are i nte ns i ve treat me nt i n a n e mer ge nc y r o o m or at h o me f or aller gic br o nc h os pas m; 
bl o o d d yscrasias or c o n v ulsi o ns t hat d o n ot res ult i n h os pitalizati o n; or de vel o p me nt of dr u g de pe n de nc y or 
dr u g a b use.
A d diti o nall y, a n y s us pecte d tra ns missi o n via a Pfizer pr o d uct of a n i nfecti o us a ge nt, pat h o ge nic or n o n -
pat h o ge nic, is c o nsi dere d seri o us.  T he e ve nt ma y be s us pecte d fr o m cli nical s y m pt o ms or la b orat or y fi n di n gs 
i n dicati n g a n i nfecti o n i n a patie nt e x p ose d t o a Pfizer pr o d uct.  T he ter ms “s us pecte d tra ns missi o n” a n d 
“tra ns missi o n” are c o nsi dere d s y n o n y m o us.  T hese cases are c o nsi dere d u ne x pecte d a n d ha n dle d as seri o us 
e x pe dite d cases b y p har mac o vi gila nce ( P V) pers o n nel.  S uc h cases are als o c o nsi dere d f or re p orti n g as pr o d uct 
defects, if a p pr o priate.
H os pitalizati o n
H os pitalizati o n is defi ne d as a n y i nitial a d missi o n (e ve n if less t ha n 2 4 h o urs) t o a h os pital or e q ui vale nt 
healt hcare facilit y or a n y pr ol o n gati o n t o a n e xisti n g a d missi o n.  A d missi o n als o i ncl u des tra nsfer wit hi n t he 
h os pital t o a n ac ute/i nte nsi ve care u nit (e. g., fr o m t he ps yc hiatric wi n g t o a me dical fl o or, me dical fl o or t o a 
c or o nar y care u nit, ne ur ol o gical fl o or t o a t u berc ul osis u nit). A n e mer ge nc y r o o m visit d oes n ot necessaril y 
c o nstit ute a h os pitalizati o n; h o we ver, a n e ve nt lea di n g t o a n e mer ge nc y r o o m visit s h o ul d be assesse d f or 
me dical i m p orta nce.
H os pitalizati o n i n t he a bse nce of a me dical A E is n ot i n itself a n A E a n d is n ot re p orta ble.  F or e xa m ple, t he 
f oll o wi n g re p orts of h os pitalizati o n wit h o ut a me dical A E are n ot t o be re p orte d.

S ocial a d missi o n (e. g., patie nt has n o place t o slee p)

A d mi nistrati ve a d missi o n (e. g., f or yearl y e xa m)

O pti o nal a d missi o n n ot ass ociate d wit h a preci pitati n g me dical A E (e. g., f or electi ve c os metic s ur ger y)

H os pitalizati o n f or o bser vati o n wit h o ut a me dical A E

A d m issi o n f or treat me nt of a pre-e xisti n g c o n diti o n n ot ass ociate d wit h t he de vel o p me nt of a ne w A E or 
wit h a w orse ni n g of t he pre -e xisti n g c o n diti o n (e. g., f or w or k -u p of persiste nt pre -treat me nt la b 
a b n or malit y)
0 9 0 1 7 7 e 1 a 2 4 e 5 1 b a\ A p pr o v e d\ A p pr o v e d O n: 0 3- D e c- 2 0 2 4 0 6: 3 3 ( G M T)

I n ot uz u ma b
B 1 9 3 1 0 2 4 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
A me n de d 8 , 12 Dece m ber 2 0 2 3
P FI Z E R C O N FI D E N TI A L
C T 2 4 -WI -J P N-G P S P 0 1 -R F 0 9 8 . 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 -N o v -2 0 2 3
P a ge 2 9 of 3 3
1 0. 4. 3. Sce n ari os N ecessit ati n g R e p orti n g t o Pfizer Safet y wit hi n 2 4 H o urs
Sce nari os i n v ol vi n g e x p os ure d uri n g pre g na nc y, e x p os ure d uri n g breastfee di n g, me dicati o n err or, o ver d ose, 
mis use, e xtra vasati o n, lac k of efficac y, a n d occ u pati o nal e x p os ure are descri be d bel o w.
E x p os ure d uri n g pre g na nc y
A n e x p os ure d uri n g pre g na nc y ( E D P) occ urs if:
1.
A fe male bec o mes, or is f o u n d t o be, pre g na nt eit her w hile recei vi n g or ha vi n g bee n e x p ose d t o 
B E S P O N S A (e. g., e n vir o n me ntal e x p os ure), or t he fe male bec o mes, or is f o u n d t o be, pre g na nt after 
disc o nti n ui n g a n d/ or bei n g e x p ose d t o B E S P O N S A ( mater nal e x p os ure).
A n e xa m ple of e n vir o n me ntal e x p os ure w o ul d be a case i n v ol vi n g direct c o ntact wit h a Pfizer pr o d uct i n a 
pre g na nt w o ma n (e. g., a n urse re p orts t hat s he is pre g na nt a n d has bee n e x p ose d t o c he m ot hera pe utic 
pr o d ucts).
2.
A male has bee n e x p ose d, eit her d ue t o treat me nt or e n vir o n me ntal e x p os ure t o B E S P O N S A pri or t o or 
ar o u n d t he ti me of c o nce pti o n a n d/ or is e x p ose d d uri n g t he part ner pre g na nc y ( pater nal e x p os ure).
F o r e x p os ure d uri n g pre g na nc y i n st u dies of pre g na nt w o me n, data o n t he e x p os ure t o B E S P O N S A d uri n g 
pre g na nc y, are n ot re p orta ble u nless ass ociate d wit h S A Es or n o n -seri o us A Es.
As a ge neral r ule, pr os pecti ve a n d retr os pecti ve e x p os ure d uri n g pre g na nc y re p orts fr o m a n y s o urce are 
re p orta ble irres pecti ve of t he prese nce of a n ass ociate d A E a n d t he pr oce d ures f or S A E re p orti n g s h o ul d be 
f oll o we d , wit h t he e xce pti o n of t h ose st u dies c o n d ucte d i n pre g na nt w o me n (as descri be d a b o ve), f or w hic h 
data o n t he e x p os ure are n ot re p orta ble u nless ass ociate d wit h S A Es or n o n- seri o us A Es. 
If a st u d y partici pa nt or st u d y partici pa nt’s part ner bec o mes, or is f o u n d t o be, pre g na nt d uri n g t he st u d y 
partici pa nt’s treat me nt wit h B E S P O N S A , t his i nf or mati o n m ust be s u b mitte d t o Pfizer, irres pecti ve of w het her 
a n A E has occ urre d, usi n g t he NI S A E M Re p ort F or m a n d t he E D P S u p ple me ntal F or m. 
I n a d diti o n, t he i nf or mati o n re gar di n g e n vir o n me ntal e x p os ure t o B E S P O N S A , i n a pre g na nt w o ma n (e. g., a 
patie nt re p orts t hat s he is pre g na nt a n d has bee n e x p ose d t o a c yt ot o xic pr o d uct b y i n halati o n or s pilla ge) m ust 
be s u b mitte d usi n g t he NI S A E M Re p ort F or m a n d t he E D P S u p ple me ntal F or m.  T his m ust be d o ne 
irres pecti ve of w het her a n A E has occ urre d.  
I nf or mati o n s u b mitte d s h o ul d i ncl u de t he a ntici pate d date of deli ver y (see bel o w f or i nf or mati o n relate d t o 
ter mi nati o n of pre g na nc y).
F o ll o w-u p is c o n d ucte d t o o btai n ge neral i nf or mati o n o n t he pre g na nc y; i n a d diti o n, f oll o w -u p is c o n d ucte d t o 
o btai n i nf or mati o n o n E D P o utc o me f or all E D P re p orts wit h pre g na nc y o utc o me u n k n o w n.  A pre g na nc y is 
f oll o we d u ntil c o m pleti o n or u ntil pre g na nc y ter mi nati o n (e. g., i n d uce d a b orti o n) a n d Pfizer is n otifie d of t he 
o utc o me.  T his i nf or mati o n is pr o vi de d as a f oll o w u p t o t he i nitial E D P re p ort.  I n t he case of a li ve birt h, t he 
str uct ural i nte grit y of t he ne o nate ca n be assesse d at t he ti me of birt h.  I n t he e ve nt of a ter mi nati o n, t he 
reas o n(s) f or ter mi nati o n s h o ul d be s pecifie d a n d, if clinicall y p ossi ble, t he str uct ural i nte grit y of t he 
ter mi nate d fet us s h o ul d be assesse d b y gr oss vis ual i ns pecti o n ( u nless pre-pr oce d ure test fi n di n gs are 
c o ncl usi ve f or a c o n ge nital a n o mal y a n d t he fi n di n gs are re p orte d).
0 9 0 1 7 7 e 1 a 2 4 e 5 1 b a\ A p pr o v e d\ A p pr o v e d O n: 0 3- D e c- 2 0 2 4 0 6: 3 3 ( G M T)

I n ot uz u ma b
B 1 9 3 1 0 2 4 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
A me n de d 8 , 12 Dece m ber 2 0 2 3
P FI Z E R C O N FI D E N TI A L
C T 2 4 -WI -J P N-G P S P 0 1 -R F 0 9 8 . 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 -N o v -2 0 2 3
P a ge 3 0 of 3 3
If t he o utc o me of t he pre g na nc y meets t he criteria f or a n S A E (e. g., ect o pic pre g na nc y, s p o nta ne o us a b orti o n, 
i ntra uteri ne fetal de mise, ne o natal deat h, or c o n ge nital a n o mal y [i n a li ve b or n, a ter mi nate d fet us, a n 
i ntra uteri ne fetal de mise, or a ne o natal deat h]), t he pr oce d ures f or re p ortin g S A Es s h o ul d be f oll o we d.
A d diti o nal i nf or mati o n a b o ut pre g na nc y o utc o mes t hat are re p orte d as S A Es f oll o ws: 

S p o nta ne o us a b orti o n i ncl u des miscarria ge a n d misse d a b orti o n;

Ne o natal deat hs t hat occ ur wit hi n 1 m o nt h of birt h s h o ul d be re p orte d, wit h o ut re gar d t o ca usalit y, as 
S A Es.  I n a d diti o n, i nfa nt deat hs after 1 m o nt h s h o ul d be re p orte d as S A Es w he n t he i n vesti gat or assesses 
t he i nfa nt deat h as relate d or p ossi bl y relate d t o e x p os ure t o i n vesti gati o nal pr o d uct.
A d diti o nal i nf or mati o n re gar di n g t he e x p os ure d uri n g pre g na nc y ma y be re q ueste d.  F urt her f oll o w -u p of birt h 
o utc o mes will be ha n dle d o n a case -b y -case basis (e. g., f oll o w -u p o n preter m i nfa nts t o i de ntif y de vel o p me ntal 
dela ys).  
I n t he case of pater nal e x p os ure, t he st u d y partici pa nt will be pr o vi de d wit h t he Pre g na nt Part ner Release of 
I nf or mati o n F or m t o deli ver t o his part ner.  It m ust be d oc u me nte d t hat t he st u d y partici pa nt was gi ve n t his 
letter t o pr o vi de t o his part ner.
E x p os ure d uri n g breastfee di n g
Sce nari os of e x p os ure d uri n g breastfe e di n g m ust be re p orte d, irres pecti ve of t he prese nce of a n ass ociate d A E.  
A n e x p os ure d uri n g breastfee di n g re p ort is n ot create d w he n a Pfizer dr u g s pecificall y a p pr o ve d f or use i n 
breastfee di n g w o me n (e. g., vita mi ns) is a d mi nistere d i n acc or d wit h a ut h or ize d use.  H o we ver, if t he i nfa nt 
e x perie nces a n A E ass ociate d wit h s uc h a dr u g’s a d mi nistrati o n, t he A E is re p orte d t o get her wit h t he e x p os ure 
d uri n g breastfee di n g.
Me dicati o n err or
A me dicati o n err or is a n y u ni nte nti o nal err or i n t he prescri bi n g, dis pe ns i n g or a d mi nistrati o n of a me dici nal 
pr o d uct t hat ma y ca use or lea d t o i na p pr o priate me dicati o n use or patie nt har m w hile i n t he c o ntr ol of t he 
healt h care pr ofessi o nal, patie nt, or c o ns u mer.  S uc h e ve nts ma y be relate d t o pr ofessi o nal practice, healt h car e 
pr o d ucts, pr oce d ures, a n d s yste ms i ncl u di n g: prescri bi n g; or der c o m m u nicati o n; pr o d uct la beli n g, pac ka gi n g, 
a n d n o me nclat ure; c o m p o u n di n g; dis pe nsi n g; distri b uti o n; a d mi nistrati o n; e d ucati o n; m o nit ori n g; a n d use. 
Me dicati o n err ors i ncl u de:

Near misses, i n v ol vi n g or n ot i n v ol vi n g a patie nt directl y (e. g., i na d verte nt/err o ne o us a d mi nistrati o n, 
w hic h is t he acci de ntal use of a pr o d uct o utsi de of la beli n g or prescri pti o n o n t he part of t he healt hcare 
pr o vi der or t he patie nt/c o ns u mer);

C o nf usi o n wit h re gar d t o i n ve nte d na me (e. g., tra de na me, bra n d na me).
T he i n vesti gat or m ust s u b mit t he f oll o wi n g me dicati o n err ors t o Pfizer, irres pecti ve of t he prese nce of a n 
ass ociate d A E/ S A E:

Me dicati o n err ors i n v ol vi n g patie nt e x p os ure t o t he pr o d uct, w het her or n ot t he me dicati o n err or is 
acc o m pa nie d b y a n A E.
0 9 0 1 7 7 e 1 a 2 4 e 5 1 b a\ A p pr o v e d\ A p pr o v e d O n: 0 3- D e c- 2 0 2 4 0 6: 3 3 ( G M T)

I n ot uz u ma b
B 1 9 3 1 0 2 4 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
A me n de d 8 , 12 Dece m ber 2 0 2 3
P FI Z E R C O N FI D E N TI A L
C T 2 4 -WI -J P N-G P S P 0 1 -R F 0 9 8 . 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 -N o v -2 0 2 3
P a ge 3 1 of 3 3

Me dicati o n err ors t hat d o n ot i n v ol ve a patie nt directl y (e. g., p ote ntial me dicati o n err ors or near misses).  
W he n a me dicati o n err or d oes n ot i n v ol ve patie nt e x p os ure t o t he pr o d uct , t he f oll o wi n g mi ni m u m criteria 
co nstit ute a me dicati o n err or re p ort:

A n i de ntifia ble re p orter;

A s us pect pr o d uct;

T he e ve nt me dicati o n err or.
O ver d ose, Mis use, E xtra vasati o n
Re p orts of o ver d ose, mis use, a n d e xtra vasati o n ass ociate d wit h t he use of a Pfizer pr o d uct are re p orte d t o 
Pfizer b y t he i n vesti gat or, irres pecti ve of t he prese nce of a n ass ociate d A E/ S A E. 
Lac k of Efficac y
Re p orts of lac k of efficac y t o a Pfizer pr o d uct a re re p orte d t o Pfizer b y t he i n vesti gat or, irres pecti ve of t he 
prese nce of a n ass ociate d A E/ S A E or t he i n dicati o n f or use of t he Pfizer pr o d uct.
Occ u pati o nal E x p os ure
Re p orts of occ u pati o nal e x p os ure t o a Pfizer pr o d uct are re p orte d t o Pfizer b y t he i n vesti gat or, irres pecti ve of 
t he prese nce of a n ass ociate d A E/ S A E.
1 1. P L A N S F O R DI S S E MI N A T I N G A N D C O M M U NI C A TI NG S T U D Y R E S U L T S
T h e i nf or m ati o n c oll e ct e d i n t his i n v esti g ati o n will b e us e d f or r e p orti n g t o t h e M H L W , t h e 
P h ar m a c e uti c als a n d M e di c al D e vi c es A g e n c y ( P M D A), a n d t h e p ar e nt c o m p a n y (Pfi z er i n c. i n t h e U nit e d 
St at es ), or its gr o u p c o m p a ni es or o v ers e as r e g ul at or y a ut h oriti es, a n d r e e x a mi n ati o n a p pli c ati o n 
(i n cl u di n g P eri o di c S af et y U p d at e R e p ort ) f or B E S P O N S A, r e e v al u ati o n a p pli c ati o n, cr e ati o n of d at a f or 
pr o vi di n g i nf or m ati o n o n t h e pr o p er us e of B E S P O N S A, pr e p ar ati o n of arti cl es, a n d a cti viti es t o pr o vi d e 
i nf or m ati o n, et c. I n a d diti o n, Pfi z er m a y p u blis h t h e i n v esti g ati o n r es ults at w w w. cli ni c altri als. g o v 
(Cli ni c al Tri als. g o v) or at a c a d e mi c c o nf er e n c es or i n p a p ers, et c. f or t h e p ur p os e of pr o vi di n g i nf or m ati o n 
o n t h e pr o p er us e of B E S P O N S A as n e c ess ar y.
T h e d at a o n p ati e nts c oll e ct e d i n t his i n v esti g ati o n will b e r e p ort e d t o t h e M H L W
b as e d o n t h e A ct o n 
S e c uri n g Q u alit y, Effi c a c y a n d S af et y of Pr o d u cts I n cl u di n g P h ar m a c e uti c als a n d M e di c al D e vi c es. I n 
s u c h c as es, i nf or m ati o n i n cl u di n g t h e dr u g n a m e, a d v ers e r e a cti o n n a m e, g e n d er, a g e ( a g e gr o u p), et c. f or 
t h e d at a o n t h e r el e v a nt p ati e nts m a y b e dis cl os e d i n a p ati e nt list i n “ P h ar m a c e uti c als a n d M e di c al 
D e vi c es S af et y I nf or m ati o n ” b y t h e M H L W , a n d “ W e bsit e f or Pr o vi di n g P h ar m a c e uti c als a n d M e di c al 
D e vi c es I nf or m ati o n ( htt p:// w w w.i nf o. p m d a. g o.j p). ” F urt h er m or e, t h e c oll e ct e d p ati e nt d at a will b e 
dis cl os e d w h e n dis cl os ur e is r e q u est e d t o t h e M H L W
b as e d o n t h e “ A ct o n A c c ess t o I nf or m ati o n H el d b y 
A d mi nistr ati v e Or g a ns ” ( A ct N o. 4 2 of M a y 1 4, 1 9 9 9). I n all of t h es e c as es, h o w e v er, i nf or m ati o n s u c h as 
t h e n a m es of t h e i n v esti g at ors a n d t h e n a m es of t h e i nstit uti o ns will n ot b e r e p ort e d, a n d t h er ef or e, will 
n ot b e p u blis h e d or dis cl os e d.
0 9 0 1 7 7 e 1 a 2 4 e 5 1 b a\ A p pr o v e d\ A p pr o v e d O n: 0 3- D e c- 2 0 2 4 0 6: 3 3 ( G M T)

I n ot uz u ma b
B 1 9 3 1 0 2 4 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
A me n de d 8 , 12 Dece m ber 2 0 2 3
P FI Z E R C O N FI D E N TI A L
C T 2 4 -WI -J P N-G P S P 0 1 -R F 0 9 8 . 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 -N o v -2 0 2 3
P a ge 3 2 of 3 3
Pfizer will be n otifie d of a n y pr o hi biti o n or restricti o n i m p ose d (e. g., cl i nical st u d y ter mi nati o n) b y t he 
a p plica ble re g ulat or y a ut h orit y i n eac h area of t he w orl d. W he n bec o mi n g a ware of ne w i nf or mati o n t hat ma y 
i nfl ue nce t he e val uati o n of be nefits a n d ris ks of a Pfizer pr o d uct, t he i n vesti gat or s hall pr o m ptl y n otif y t he 
Pfize r of t he i nf or mati o n.
1 2. N A M E, A N D A D D R E S S O F C O N T R A C T O R A S W E L L A S S C O P E O F W O R K C O N T R A C T E D
Na me: Pfizer R & D Ja pa n G. K.
A d dress: 3 -2 2 -7 Y o y o gi, S hi b u ya -k u, T o k y o
Sc o pe of w or k c o ntracte d : O perati o ns relate d t o t he pla n ni n g of t his i n vesti gati o n, t he drafti n g of t he pr ot oc ol, 
t he o perati o n of t his i n vesti gati o n, m o nit ori n g, etc.
Na me: E P S C or p orati o n
A d dress: 2 -2 3 S hi m o mi ya bic h o, S hi nj u k u -k u, T o k y o
Sc o pe of w or k c o ntracte d : O perati o ns f or t his i n vesti gati o n s uc h as re gistrati o n 
rece pti o n, esta blis h me nt of t he p ost-mar keti n g s ur veilla nce data c ollecti o n s yste m ( E D C), 
data ma na ge me nt acti vities, a n d ta b ulati o n a n d a nal ysis o perati o ns, e xcl u di n g o perati o ns t o 
ma na ge t he p ost -mar keti n g st u d y
Na me: Me di data S ol uti o ns
A d dress: 2 - 7-2 Mar u n o uc hi, C hi y o da -k u, T o k y o
Sc o pe of w or k c o ntracte d : Acti vities relate d t o t he esta blis h me nt a n d o perati o ns of t he p ost-mar keti n g 
s ur veilla nce data c ollecti o n s yste m ( E D C)
Na me: A 2 Healt hcare C or p orati o n
A d dress: 1 - 4- 1- K ois hi ka wa, B u n k y o -k u, T o k y o
Sc o pe of w or k c o ntracte d : Acti vities relate d t o E D C acc o u nt ma na ge me nt, etc.
1 3. A D DI TI O N A L M E A S U R E S T H A T M A Y B E I M P L E M E N T E D B A S E D O N T H E S U R V E Y 
R E S U L T S A N D C RI T E RI A F O R D E T E R MI N A TI O N O F T H E I NI TI A TI O N
At milest o nes, t he ris k ma na ge me nt pla n ( R M P) i ncl u di n g t h e f oll o wi n g ite ms s h o ul d be re vie we d:
1) T o e val uate w het her or n ot t here are ne w safet y s pecificati o ns;
2)
T o e xa mi ne w het her or n ot it is necessar y t o pla n a d diti o nal p har mac o vi gila nce acti vities a n d t o esta blis h 
a ris k mi ni mizati o n pla n f or a ne w safet y s pec ificati o n; a n d
3)
T o c o nsi der w het her or n ot it is necessar y t o a me n d t he a d diti o nal p har mac o vi gila nce acti vit y pla n a n d 
t he details of ris k mi ni mizati o n acti vities f or t he c urre nt safet y s pecificati o ns.
1 4. S C H E D U L E D TI MI N G O F MI L E S T O N E S A N D T H EI R
R A TI O N A L E S F O R E V A L U A TI O N O F 
S T U D Y I M P L E M E N T A TI O N S T A T U S A N D R E S U L T S A N D R E P O R TI N G T O T H E P M D A
Sc he d ule d ti mi n g is at t he ti me of s u b mitti n g Peri o dic Safet y U p date Re p ort , t he ti me of ter mi nati n g 
re gistrati o n (at t he ti me of pre pari n g t he 5t h or 6t h Peri o dic Safet y U p date Re p ort ), t he ti me of e n di n g t he 
i n vesti gati o n (at t he ti me of pre pari n g t he 9t h or 1 0t h Peri o dic Safet y U p date Re p ort ) a n d t he ti me of pre pari n g 
ree xa mi nati o n a p plicati o n data.
0 9 0 1 7 7 e 1 a 2 4 e 5 1 b a\ A p pr o v e d\ A p pr o v e d O n: 0 3- D e c- 2 0 2 4 0 6: 3 3 ( G M T)

I n ot uz u ma b
B 1 9 3 1 0 2 4 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
A me n de d 8 , 12 Dece m ber 2 0 2 3
P FI Z E R C O N FI D E N TI A L
C T 2 4 -WI -J P N-G P S P 0 1 -R F 0 9 8 . 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 -N o v -2 0 2 3
P a ge 3 3 of 3 3
1 5. O T H E R N E C E S S A R Y M A T T E R S
1)
A me n d me nt of t he f ull pr ot oc ol
Base d o n t he ne w k n o wle d ge t o be o btai ne d acc or di n g t o t he pr o gress of t his i n vesti gati o n, t he nee d f or 
a me n d me nt of t he pr ot oc ol will be e xa mi ne d a n d t he pr ot oc ol will be a me n de d if necessar y. Als o, t he nee d f or 
a me n d me nt of t he pr ot oc ol will be e xa mi ne d a n d t he pr ot oc ol will be a me n de d e ve n if t he partial c ha n ge i n t he 
d osa ge a n d a d mi nistrati o n or i n dicati o n is a p pr o ve d d uri n g t he ree xa mi nati o n peri o d (e xce pt t he case w he n t he 
ree xa mi nati o n peri o d is ne wl y desi g nate d), etc.
2)
Acti o ns t o be ta ke n f or a n y pr o ble m or iss ue
I n t he cases w here t he o nset of a n y seri o us a n d u n k n o w n A D Rs is s u g geste d, a si g nifica nt i ncrease i n t he 
fre q ue nc y of A D Rs is o bser ve d, a n y pr o ble m is f o u n d i n t he efficac y a n d safet y of t he dr u g c o m pare d t o t h ose 
pri or t o t he a p pr o val, t he o nset of a differe nt ki n d of A D R is s u g geste d, etc., t he a me n d me nt of t he pac ka ge 
i nsert a n d i m ple me ntati o n of ne w p ost-mar keti n g s ur veilla nce s h o ul d be c o nsi dere d.
1 6. C O N T A C T I N F O R M A TI O N
1 6. 1. C o nt act I nf or m ati o n f or t he C o nte nts of t he St u d y
Na me
P M S Affairs , Pfizer R & D Ja pa n G. K.
A d dress
S hi nj u k u B u n ka Q ui nt B uil di n g, 3 -2 2 -7 Y o y o gi, S hi b u y a-k u, T o k y o 1 5 1 -8 5 8 9
Fa x
0 3 -5 3 0 9 -9 1 8 6
E- mail a d dress
B E S _ P M S @ pfizer.c o m
1 6. 2. C o nt act I nf or m ati o n f or t he E D C S yste m
Na me
Me di data, Hel p Des k
O pe n h o urs
Wee k da ys: 9: 0 0 t o 2 0: 0 0 (e xcl u di n g Sat ur da ys, S u n da ys, nati o nal h oli da ys, a n d year -
e n d a n d Ne w Year h oli da ys)
Tel
 ( de dicate d Pfizer dial)
E- mail a d dress
ja pa n hel p des k @ m ds ol.c o m
0 9 0 1 7 7 e 1 a 2 4 e 5 1 b a\ A p pr o v e d\ A p pr o v e d O n: 0 3- D e c- 2 0 2 4 0 6: 3 3 ( G M T)
P P D