Clinical trial NCT04927247
Conditions: Sickle Cell Disease Vaso-occlusive Crisis Vaso-occlusive Pain Episode in Sickle Cell Disease
Keywords: Re-admission Acute blood disorders hemoglobin red blood cells RBCs sickle-like shape mutation in hemoglobin gene sickle-cell trait sickle-cell crisis Sickle Cell Disease SCD Vaso-occlusive Crisis VOC SCA
Sponsor: Pfizer
Start date: 2021-12-09; Completion date: 2023-11-24
Enrollment: 72 participants; Allocation: RANDOMIZED; Intervention model: PARALLEL

Brief summary:
This Phase 3 study will assess the safety and efficacy of a single dose of inclacumab, a P-selectin inhibitor, for a vaso-occlusive crisis (VOC) after an index VOC in participants with sickle cell disease (SCD). Participants will be randomized to receive either inclacumab or placebo.

Detailed description:
The study will include approximately 280 adult and adolescent participants (≥ 12 years of age) with SCD.

Eligible participants will be administered inclacumab or placebo intravenous (IV) as a single dose.

Participants that complete the study through Day 90 will be provided the opportunity to enroll in an open-label extension (OLE) study.

Arms: arm 1: Inclacumab 30 mg/kg administered intravenously (IV) arm 2: Placebo administered IV
Interventions: intervention 1: Inclacumab intervention 2: Placebo
Intervention descriptions: intervention 1: Inclacumab will be supplied in single use 10 mL vials at a concentration of 50 mg/mL. One vial contains 500 mg of inclacumab. This is a liquid concentrate for IV infusion. intervention 2: Placebo will be supplied in single use 10 mL vials containing the same ingredients without the active drug. Placebo will be prepared as a liquid concentrate for IV infusion and administered in the same manner as active study drug.

Protocol documents:
Prot_000.pdf: 



















































































































































SAP_001.pdf: 
 
           
 
 
 
STATISTICAL ANALYSIS PLAN  
 
 
Study Title: 
A RANDOMIZED, DOUBLE-BLIND, PLACEBO-
CONTROLLED, MULTICENTER STUDY OF A 
SINGLE DOSE OF INCLACUMAB TO REDUCE RE-
ADMISSION IN PARTICIPANTS WITH SICKLE CELL 
DISEASE AND RECURRENT VASO-OCCLUSIVE 
CRISES 
 
 
Development Phase: 
Phase 3 
 
 
Protocol Number: 
GBT2104-132 (C5361002) 
 
 
Protocol Date and Version: 
17 February 2022 (Amendment 2), Version 3.0 
 
 
Investigational Product: 
Inclacumab (GBT2104 / PF-07940370) 
 
 
Sponsor: 
Global Blood Therapeutics, Inc., a wholly owned 
subsidiary of Pfizer Inc. 
181 Oyster Point Boulevard 
South San Francisco, CA  94080 
United States of America 
 
 
Analysis Plan Version: 
Version 1.0 
 
 
Analysis Plan Date: 
10 August 2023 
 
 
 
 
 
 
 
 
 
 
CONFIDENTIAL AND PROPRIETARY INFORMATION 
 
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G B T, a w h oll y o w ne d s u bsi diar y of Pfizer
                                    I nclac u ma b
Statistical A nal ysis Pla n
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Gl o bal Bl o o d T h era pe utics, I nc., a w h oll y o w ne d s u bsi diar y of 
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                                    Inclacumab 
Statistical Analysis Plan 
           GBT2104-132 (C5361002) 
 
 
PFIZER CONFIDENTIAL 
Page 3 
10 August 2023 
 
TABLE OF CONTENTS 
STATISTICAL ANALYSIS PLAN REVIEW AND APPROVAL ................................................2 
TABLE OF CONTENTS .................................................................................................................3 
LIST OF TABLES ...........................................................................................................................4 
LIST OF FIGURES .........................................................................................................................4 
1. 
GLOSSARY OF ABBREVIATIONS ..........................................................................5 
2. 
INTRODUCTION ........................................................................................................6 
2.1. 
Study Design .................................................................................................................6 
2.2. 
Study Endpoints ............................................................................................................7 
2.2.1. 
Primary Efficacy Endpoint ...........................................................................................7 
2.2.2. 
Secondary Efficacy Endpoints ......................................................................................8 
2.2.3. 
Exploratory Endpoints ..................................................................................................8 
2.2.4. 
Safety Endpoints ...........................................................................................................9 
2.2.5. 
Exploratory Pharmacology Endpoints ..........................................................................9 
2.3. 
Determination of Sample Size ......................................................................................9 
2.3.1. 
Basis for Original Sample Size Assumptions .............................................................10 
2.4. 
Randomization ............................................................................................................10 
2.5. 
Analysis Timing ..........................................................................................................11 
3. 
GENERAL CONSIDERATIONS ..............................................................................12 
3.1. 
Definitions and Terminology ......................................................................................12 
3.2. 
Visit Windows ............................................................................................................13 
4. 
ANALYSIS POPULATIONS ....................................................................................15 
4.1. 
Intent-to-Treat Population ..........................................................................................15 
4.2. 
Safety Population ........................................................................................................15 
5. 
STATISTICAL METHODS .......................................................................................16 
5.1. 
Summaries of Study Conduct .....................................................................................16 
5.2. 
Summaries of Demographic, Baseline Characteristics, and Concomitant 
Medications .................................................................................................................16 
5.3. 
Efficacy Analyses .......................................................................................................16 
5.3.1. 
Primary Efficacy Endpoint .........................................................................................17 
5.3.2. 
Secondary Efficacy Endpoints ....................................................................................18 
5.3.2.1. 
Time to First VOC ......................................................................................................18 
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                                    Inclacumab 
Statistical Analysis Plan 
           GBT2104-132 (C5361002) 
 
 
PFIZER CONFIDENTIAL 
Page 4 
10 August 2023 
 
5.3.2.2. 
Participant Status with at Least 1 VOC Requiring Re-Admission with 30 
Days ............................................................................................................................18 
5.3.2.3. 
Rate of VOCs Leading to Healthcare Visit and Requiring Parenteral Pain 
Medication ..................................................................................................................18 
5.3.3. 
Exploratory Efficacy Endpoints..................................................................................19 
5.4. 
Safety Analyses ..........................................................................................................19 
5.4.1. 
Exposure to Study Drug and Compliance ..................................................................19 
5.4.2. 
Adverse Events ...........................................................................................................20 
5.4.3. 
Clinical Laboratory Assessments ...............................................................................20 
5.4.4. 
Vital Signs and Weight ...............................................................................................21 
5.5. 
Exploratory PK, ADA, and PD Analyses ...................................................................21 
5.5.1. 
Pharmacokinetic Analyses ..........................................................................................21 
5.5.2. 
Anti-drug Antibody Analyses .....................................................................................21 
5.5.3. 
Pharmacodynamic Analyses .......................................................................................22 
6. 
TABLES, LISTINGS, AND FIGURES .....................................................................23 
7. 
REFERENCES ...........................................................................................................24 
 
LIST OF TABLES 
Table 1: 
Analysis Windows for Assessments Other than ASCQ-Me.......................................13 
Table 2: 
Analysis Windows for Assessment of ASCQ-Me ......................................................14 
Table 3: 
ASCQ-Me Pain Impact (Short Form) Response Mapping .........................................19 
 
LIST OF FIGURES 
Figure 1: 
GBT2104-132 Study Design ........................................................................................7 
 
 
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G B T, a w h oll y o w ne d s u bsi diar y of Pfizer 
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P FI Z E R C O N FI D E N TI A L 
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1.  
G L O S S A R Y O F A B B R E VI A TI O N S 
A C S
 
  ac ute
 
c hest
 
s y n dr o me
  
A D A
 
  a nti- dr u g
 
a nti b o di es
 
A E SI  
  
a d verse e ve nt of s pecial i nt erest 
A S C Q- M e   
A d ult Sic kle C ell Q ualit y of Life Me as ure me nt 
C GI- C
 
  
Cli ni cia n’s  Gl o bal I m pressi o n of C ha n ge 
CI    
  
c o nfi de nc e  i nt er val  
C M H
 
  C oc hra n- Ma ntel- Hae nszel
 
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 
D M C  
  
Data M o nit ori n g C o m mitte e 
E O S  
  
e n d of st u d y 
H U  
  
h y dr o x y urea 
I R T
 
  i ntera cti v e
 
res p o nse
 
tec h n ol o g y
 
I T T
 
 i nte nt
 
t o
 
tre at
 
Me d D R A    
Me dical Di cti o n ar y f or Re g ulat or y Acti viti es 
N S AI D
 
  
n o nster oi dal
 
a nti-i nfla m mat or y
 
dr u g
 
O L E
 
  o pe n-la bel
 
e xt e nsi o n
 
P D   
  
p har mac o d y n a mi cs(s) 
P GI- C
 
 
Patie nt’s Gl o bal I m pressi o n of C ha n g e  
P K   
  
p har mac o ki netic 
S A E  
  
seri o us a d verse e ve nt 
S A P
 
  statisti cal
 
a nal ysis
 
pl a n
 
S C D  
  
sic kle c ell disease 
S O C  
  
S yste m Or ga n Class 
T E A E
 
  
treat me nt- e mer ge nt
 
a d verse
 
e ve nt
 
V O C
 
  vas o- occl usi ve
 
crisis
 
W H O  
  
W orl d Healt h Or ga nizati o n  
 
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                                    Inclacumab 
Statistical Analysis Plan 
           GBT2104-132 (C5361002) 
 
 
PFIZER CONFIDENTIAL 
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10 August 2023 
 
2. 
INTRODUCTION 
The primary objective of Study GBT2104-132 (C5361002) is to evaluate the safety and efficacy 
of a single dose of inclacumab compared with placebo to reduce the incidence of re-admission to 
a healthcare facility for a vaso-occlusive crisis (VOCs) after an admission for an index VOC in 
participants with sickle cell disease (SCD). However, as of June 2023, the Sponsor decided to 
discontinue Study GBT2104-132 early, prior to full enrollment. The decision is based on 
business considerations and not due to any specific safety reason or request from any regulatory 
authority. 
This statistical analysis plan (SAP) provides details of the planned analyses and statistical 
methods for Study GBT2104-132. In many instances, analyses have been limited and/or are 
descriptive in nature, due to the early discontinuation of the study. 
Where this document differs from the high-level analysis plan described in the study protocol, 
the methodology described in this SAP is considered the latest and supersedes the corresponding 
section(s) in the protocol. 
Population pharmacokinetic (PK), exploratory pharmacodynamic (PD), and exploratory 
biomarker analyses will be described in a separate document. 
2.1. 
Study Design 
Study GBT2104-132 is a Phase 3, randomized, placebo-controlled, double-blind, multicenter, 
parallel-group study to assess the safety and efficacy of a single dose of inclacumab in reducing 
the incidence of re-admission to a healthcare facility for a VOC after an index VOC. As 
originally planned, the study was to include up to approximately 280 adult and adolescent 
participants (≥ 12 years of age) with SCD. However, as noted, the Sponsor has decided to 
discontinue the study prior to full enrollment (with approximately 26% of the total planned 
participants randomized). All randomized participants are to complete the study per the protocol 
Schedule of Assessments. 
Initial enrollment was to include participants ≥ 16 years of age until the independent Data 
Monitoring Committee (DMC) recommends to the Sponsor that adequate safety and PK data 
support the enrollment of participants 12 to 15 years of age. 
Eligible participants will be randomized with a 1:1 ratio into one of two treatment arms (up to 
approximately 140 participants per arm) as follows: 
x Inclacumab 30 mg/kg administered IV; or  
x Placebo administered IV.  
All participants will undergo study assessments (safety, efficacy, PK, PD, and biomarkers, as 
applicable) at Screening, Baseline (Day 1), and through Day 91, as detailed in the Schedules of 
Assessments (see study protocol, Appendices 1 to 3). 
Following completion of the Day 91 Visit, eligible participants will be given the option to enroll 
in an open-label extension (OLE) study under a separate protocol. For participants enrolling in 
the OLE study, the Day 91 Visit will be the end of study (EOS) visit.  For participants not 
enrolling in the OLE study, an additional required Follow-up Visit at Day 161 will be the EOS 
visit. 
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Statistical Analysis Plan
           GBT2104-132 (C5361002)
PFIZER CONFIDENTIAL
Page 7
10 August 2023
A diagram of the study design is provided in Figure 1. Further study design details are provided 
in the study protocol.
Figure 1:
GBT2104-132 Study Design 
Abbreviations: IV=intravenous, OLE=open-label extension.
2.2.
Study Endpoints
2.2.1.
Primary Efficacy Endpoint
The primary endpoint for the study is, following an index VOC, the proportion of participants 
with at least 1 VOC that required admission to a healthcare facility and treatment with parenteral 
pain medication within 90 days of randomization. 
An admission for a VOC includes:
x
A hospital admission, or
x
An admission to an emergency room, observation unit, or infusion center for ≥ 12 hours, 
or
x
2 visits to an emergency room, observation unit, or infusion center over a 72-hour period,
for an acute episode of pain with no other cause other than a vaso-occlusive event that includes 
the following:
x
Uncomplicated VOC, or 
x
Acute chest syndrome, or
x
Hepatic sequestration, or
x
Splenic sequestration. or
x
Priapism.
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Statistical Analysis Plan 
           GBT2104-132 (C5361002) 
 
 
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10 August 2023 
 
The definition of the index VOC requiring admission is the same as the primary endpoint VOC 
requiring admission. 
To ensure consistency across study sites, all on-study VOCs reported by the study investigators 
will be adjudicated by an independent, blinded VOC Adjudication Committee comprised of 
experts in SCD. Unless otherwise noted, efficacy analyses will be performed on adjudicated data. 
2.2.2. 
Secondary Efficacy Endpoints 
The secondary efficacy endpoints for the study are the following: 
x Time to first VOC that required admission to a healthcare facility and treatment with 
parenteral pain medication within 90 days of randomization. 
x Proportion of participants with at least 1 VOC that required admission to a healthcare 
facility and treatment with parenteral pain medication within 30 days of randomization. 
x Rate of VOCs leading to a healthcare visit (hospital, emergency room, clinic visit, or 
remote contact with a healthcare provider) that requires parenteral pain medication (e.g., 
parenteral narcotic agents or parenteral nonsteroidal anti-inflammatory drugs [NSAIDs]), 
or an increase in treatment with oral narcotics within 90 days following randomization.  
2.2.3. 
Exploratory Endpoints 
The exploratory endpoints for the study are the following: 
x Time to second VOC that required admission to a healthcare facility and treatment with 
parenteral pain medication within 90 days of randomization.  
x Rate of complicated VOCs (i.e., acute chest syndrome, hepatic sequestration, splenic 
sequestration, and priapism) during the 90 days following randomization. 
x Rate of inpatient hospital admissions for any reason during the 90 days following 
randomization. 
x Number of days of inpatient hospitalization for any reason during the 90 days following 
randomization. 
x Proportion of participants rated as “very much improved” or “much improved” based on 
the Patient’s Global Impression of Change (PGI-C) at Day 46 and Day 91. 
x Proportion of participants rated as “very much improved” or “much improved” based on 
the Clinician’s Global Impression of Change (CGI-C) at Day 46 and Day 91. 
x Change from Baseline in the cumulative score for the Adult Sickle Cell Quality of Life 
Measurement (ASCQ-Me) Pain Impact – Short Form over time to Day 91. 
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G B T, a w h oll y o w ne d s u bsi diar y of Pfizer 
                                    I nclac u ma b 
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P FI Z E R C O N FI D E N TI A L 
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2. 2. 4.  
S afet y E n d p oi nts 
T he safet y e n d p oi nts f or t h e st u d y are t h e f oll o wi n g: 
 I nci de nc e of treat me nt-e m er ge nt a d verse e v e nts ( T E A Es). 
 C ha n ge fr o m Baseli ne i n la b orat or y assess me nts (c o m plet e bl o o d c o u nt, c he mistr y, a n d 
c oa g ulati o n). 
2. 2. 5.  
E x pl or at or y P h ar m ac ol o g y E n d p oi nts 
T he e x pl or at or y p har mac ol o g y e n d p oi nts of t h e st u d y are t he f oll o wi n g: 
 Plas ma P K of i ncla c u ma b as assesse d b y p o p u lati o n P K a nal ysis usin g n o nli near mi xe d-
effects m o d eli n g. 
 I nci de nc e of a nti- dr u g a nti b o dies ( A D A) t o i ncla c u m a b. 
 P har mac o d y na mics i n cl u di n g 
 
 
 
. 
 Bi o mar kers i n cl u di n g 
 
Relati o ns hi ps bet wee n P K, P D, bi o mar kers, cli ni cal l a bs, safet y, a n d efficac y will be e x pl ore d. 
2. 3.  
Det er mi n ati o n of S a m ple Si ze 
T he ori gi n al s a m ple siz e deter mi nati o n was base d o n statisti cal p o wer c o nsi derati o ns f or t he 
pri mar y efficac y e n d p oi nt , t he pr o p orti o n of partici p a nts wit h at least 1 V O C t hat re q uire d 
a d missi o n t o a h ealt hcare f acilit y a n d tre at m e nt wit h pare nter al pai n me di cati o n wit hi n 9 0 d a ys 
of ra n d o miz ati o n.  
As ori gi nall y pl a n ne d, t h e st u d y was t o use a gr o u p s e q ue nti al desi g n t o e val uate t he pri mar y 
effica c y e n d p oi nt base d o n 2 se q ue ntial a nal yses:  (i) o ne i nteri m a n al ysis aft er a p pr o xi m atel y 7 5 
partici pa nts per ar m ha ve c o m p l ete d t he st u d y t hr o u g h 9 0 da ys, an d (ii) if r e q uire d, a fi nal st u d y 
a nal ysis base d o n a p pr o xi matel y 1 4 0 partici pa nts per ar m. 
F or t he pri mar y e n d p oi nt, t he pl a n ne d sa m pl e size of u p t o 2 8 0  partici pa nts ( 1 4 0 partici p a nts per 
treat me nt gr o u p) was t o pr o vi de a p pr o xi mat el y 9 0 % p o wer t o dete ct a tar g et e d 5 0 % rel ati v e 
re d ucti o n i n t he pr o p orti o n of partici p a nts wit h at le ast 1 V O C t hat req uire d a d missi o n t o a 
healt h care fa cilit y a n d treat me nt w it h par e nter al pai n c o ntr ol wit hi n 9 0 da ys, fr o m a r ate of 3 5 % 
f or place b o t o 1 7. 5 % f or i ncl ac u m a b, usi n g a 2-side d test of t he differe nc e i n 2 bi n o mial 
pr o p orti o ns ( N or mal a p pr o xi mati o n). T o mai nt ai n a n o verall T y pe I err or rate of 0. 0 5, 
calc ulati o ns ass u me d a n O’ Brie n - Fle mi n g b o u n dar y of t he La n- De Mets al p ha-s pe n di n g f u n cti o n 
( East®  versi o n 6. 5). F or t h e sa m pl e size c alc ulati o n, a 5 % dr o p- o ut rat e d uri n g t he 9 0- d a y st u d y 
peri o d was use d.  
D o c U UI D : 4f 7 9 1 7 6 4- 8 3 e b- 4 3 c 6- 8 7 2 7- 5 5 2f 3 8 2 9 cf 6 e
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                                    Inclacumab 
Statistical Analysis Plan 
           GBT2104-132 (C5361002) 
 
 
PFIZER CONFIDENTIAL 
Page 10 
10 August 2023 
 
With this original sample size, the smallest observed treatment difference expected to be 
statistically significant at the final analysis was approximately a 32% relative reduction 
(i.e., reduction in the proportion of participants with at least 1 VOC that required admission to a 
healthcare facility and treatment with parenteral pain medication within 90 days from 35% for 
placebo to 23.8% for inclacumab). 
As of June 2023, the Sponsor has decided to discontinue the study prior to full enrollment, with 
approximately 26% of the total number of planned participants randomized. The ultimate study 
sample size is based on screening and enrollment at the time of the decision to discontinue, 
rather than any statistical considerations.  
2.3.1. 
Basis for Original Sample Size Assumptions 
Based on 2016 nationwide re-admission data, a 30-day all cause re-admission rate of 26.9% was 
reported among patients admitted with a principal diagnosis of sickle cell crisis (Kumar, 2020). 
The majority (86%) of such re-admissions were due to sickle cell crisis or related diagnosis. 
Similarly, an analysis of patient claims data (Source Healthcare Analytics Patient Transaction 
Dataset; August 2017 to July 2018 cohort) showed that among patients with a history of VOC, 
approximately 50% of patients hospitalized for an index VOC are expected to be re-hospitalized 
for a VOC within 90 days (data on file). However, overall hospital admissions have decreased 
with the COVID-19 pandemic (Birkmeyer, 2020) and patients with SCD may try to avoid 
hospitalizations (McFarling, 2020; Powell, 2020).  
In this study, the primary endpoint evaluates, following an index VOC, the occurrence of at least 
1 VOC that required admission to a healthcare facility (hospital admission; admission to an 
emergency room, observation unit, or infusion center for ≥ 12 hours; or 2 visits to an emergency 
room, observation unit, or infusion center over a 72-hour period) and treatment with parenteral 
pain medication within 90 days of randomization. For purposes of sample size calculation, it was 
assumed that approximately 35% of participants in the placebo group experience such a 
protocol-defined VOC within 90 days of randomization.   
The assumption of 5% drop-out rate during the 90-day study period was based on data from the 
Phase 3 clinical trial of voxelotor, Study GBT440-031, in patients with SCD (data on file). 
2.4. 
Randomization 
Eligible participants will be randomized on Day 1 through a central interactive response 
technology (IRT) system. Randomization may occur up to 5 days following investigator-assessed 
resolution of index VOC (for example, hospital discharge, completion of parenteral analgesia, or 
transition to oral analgesics). 
Participants will be randomized with a 1:1 ratio to receive treatment with inclacumab or placebo. 
A stratified permuted block design will be used, with randomization stratified by baseline 
hydroxyurea (HU) use (yes; no), number of VOCs in the preceding 12 months (2 to 4 episodes; 
5 to 10 episodes), and geographic region (North America; rest of world). 
On the day of randomization (Day 1), participants will receive a single dose of study drug. 
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Statistical Analysis Plan 
           GBT2104-132 (C5361002) 
 
 
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2.5. 
Analysis Timing 
Due to the early termination of the study, the protocol-specified interim efficacy analysis will not 
occur.  
Final study data will be used for all the analyses to be performed for the CSR after all 
randomized participants have completed the study or discontinued early (i.e., last participant’s 
last visit has occurred), and all corresponding data have been entered into the database, reviewed, 
and verified and the database is locked. 
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3. 
GENERAL CONSIDERATIONS  
3.1. 
Definitions and Terminology 
Study Drug 
The term study drug refers to either inclacumab or placebo.  
Baseline Value 
Baseline is defined as the last available pre-treatment value taken on or before the day of 
randomization, and will be used for summary of baseline characteristics and change-from-
baseline analyses, as appropriate. For ASCQ-Me, baseline is defined as the assessment taken on 
the day of randomization. 
Day 1  
Day 1 is the date of randomization.  
Study Day  
Study Day is defined relative to the date of randomization.  
For study assessments or events that occur on or after the date of randomization, study day is 
calculated as: 
Study Day = Event Date – Randomization Date + 1. 
For study assessments or events that occur before the date of randomization, study day is 
calculated as: 
Study Day = Event Date – Randomization Date.  
Study Visit  
Study Visit is the protocol visit as shown in Table 1.  
Treatment Day 
Treatment Day is defined relative to the date of first dose of study drug.  
For study assessments or events that occur on or after the date of first dose, treatment day is 
calculated as: 
Treatment Day = Event Date – First Dose Date + 1. 
For study assessments or events that occur before the date of first dose, treatment day is 
calculated as: 
Treatment Day = Event Date – First Dose Date. 
Treatment-Emergent Adverse Event 
Treatment-emergent adverse events (TEAEs) are defined as adverse events that occur on or after 
Day 1 of study treatment or the worsening of a preexisting condition on or after Day 1 of study 
treatment. Note: Given the long half-life of inclacumab, events occurring through end of study 
will be considered treatment emergent. 
 
 
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Study Completion 
Completion of study is specified on the End of Study (EOS) CRF. Participants are considered to 
have completed the study if (1) the subject completed the study through the Day 91 visit and 
elected to enroll in the open-label extension study, or (2) the subject did not enroll in the open-
label extension study and completed the study through the Day 161 visit. 
Treatment Completion 
Completion of treatment is specified on the End of Treatment (EOT) CRF and includes all 
participants who did not permanently discontinue study drug early. 
3.2. 
Visit Windows 
For summaries by timepoint (e.g., CGI-C, PGI-C, vital signs, laboratory values), analysis visit 
windows will be used to classify assessments based on the actual study day of the measurement 
regardless of the original nominal visit label. This includes assessments collected at 
Unscheduled, Early Termination, or EOS visits. Target study days, the protocol-specified study 
day windows, and the analysis visit windows are shown in Table 1.  
Table 1: 
Analysis Windows for Assessments Other than ASCQ-Me 
Study Visit 
Target Study 
Day 
Visit Window  
per Protocol  
(study days) 
Window for  
Statistical Analysis 
(study days) 
Screening 
- 
[-10, -1] 
[-10, -1] 
Day 1 
1 
1 
1 
Day 46 
46 
[39, 53] 
[32, 60] 
Day 91 
91 
[84, 98] 
[77, 105] 
Day 161 
161 
[147, 175] 
[140, 182] 
ASCQ-Me=Adult Sickle Cell Quality of Life Measurement, 
Note: Baseline is defined as the last available pre-treatment value taken on or before the day of randomization.  
Note: If multiple measurements fall within the statistical analysis window, the measurement closest to the target 
study day will be used. If two measurements are equally close, the earlier value will be used. 
 
 
 
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For the ASCQ-Me questionnaire (completed weekly at home), the analysis windows shown in 
Table 2 will apply for data summary.  
Table 2: 
Analysis Windows for Assessment of ASCQ-Me 
Analysis Timepoint 
Target Study Day 
Window for Statistical Analysis 
(study days) 
Baseline 
1 
1 
Week 1 
8 
[2, 11] 
Week 2 
15 
[12, 18] 
Week 3 
22 
[19, 25] 
Week 4 
29 
[26, 32] 
Week 5 
36 
[33, 39] 
Week 6 
43 
[40, 46] 
Week 7 
50 
[47, 53] 
Week 8 
57 
[54, 60] 
Week 9 
64 
[61, 67] 
Week 10 
71 
[68, 74] 
Week 11 
78 
[75, 81] 
Week 12 
85 
[82, 88] 
Week 13 
92 
[89, 95] 
ASCQ-Me=Adult Sickle Cell Quality of Life Measurement  
Note: Baseline is defined as the assessment taken on the day of randomization. If multiple measurements fall within 
the statistical analysis window, the measurement closest to the target study day will be used. If two measurements 
are equally close, the earlier value will be used. 
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4. 
ANALYSIS POPULATIONS 
Two main analysis populations are defined for this study: the intent-to-treat (ITT) population and 
the safety population. 
4.1. 
Intent-to-Treat Population 
The ITT population includes all randomized participants. For analyses based on this population, 
participants will be grouped according to treatment assigned at randomization.  
The ITT population will be the main analysis population for efficacy analyses and summaries of 
demographic and baseline characteristics.  
4.2. 
Safety Population 
The safety population includes randomized participants who received treatment with study drug. 
For analyses based on this population, participants will be grouped according to the actual study 
treatment received. Any participant who receives treatment with inclacumab will be classified in 
the inclacumab treatment arm. Participants who receive treatment with placebo only will be 
classified in the placebo treatment arm.  
The safety population will be the primary analysis population for safety and exposure data. 
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5. 
STATISTICAL METHODS 
5.1. 
Summaries of Study Conduct 
The number of participants randomized will be tabulated by region, country, study site, and 
treatment group. Participant disposition (the number of participants randomized, treated, and 
completing the study) will be tabulated by treatment group. Reasons for study drug interruption 
and discontinuation, as well as study discontinuation, will be summarized.  
Any eligibility criteria deviations, dosing errors, and other significant protocol deviations will 
also be tabulated by treatment group and evaluated for potential impact on the interpretation of 
study results. 
5.2. 
Summaries of Demographic, Baseline Characteristics, and 
Concomitant Medications 
Demographic and baseline characteristics (such as age, sex, race, body weight in kg, estimated 
glomerular filtration rate [eGFR], sickle cell genotype, number of VOCs in the 12 months prior 
to the screening visit, baseline HU use, baseline voxelotor use, prior crizanlizumab use, and 
geographic region) will be summarized for the ITT population by treatment group. 
Medical history will be coded using Medical Dictionary for Regulatory Activities (MedDRA; the 
most current version at the time of the analysis will be used) and summarized for each treatment 
group. Prior and concomitant medications will be coded using the World Health Organization 
(WHO) Drug Dictionary and summarized.  
Time on study, defined as time from randomization (Day 1) to the participant’s end of study 
date, will also be tabulated. 
5.3. 
Efficacy Analyses 
Efficacy analyses will be based on the ITT patient population (Section 4.1), with participants 
grouped according to the treatment assigned at randomization. Data from all randomized 
participants, regardless of treatment with study drug or adherence to the protocol will be 
included in the efficacy analyses. 
Covariate Adjustment 
Unless otherwise noted, analyses of primary and secondary efficacy endpoints will be adjusted 
for the following randomization stratification variables: 
x Baseline HU use (yes; no) 
x Number of VOCs in the 12 months prior to study entry (2 to 4 episodes; 5 to 10 episodes) 
If convergence or model issues arise, then analyses will be simplified by excluding one of the 
above stratification variables.  
Note: The additional randomization stratification variable of geographic region (North America; 
rest of world) will not be included in the analyses due to the early study discontinuation and the 
low number of subjects enrolled from North America.   
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For stratification variables, the value recorded in the clinical database (i.e., per case report form) 
will be compared to value captured in the IRT. If differences are observed, the values from the 
clinical database will be used in the analysis.  
Statistical Tests 
No adjustment for multiple comparisons will be performed. 
5.3.1. 
Primary Efficacy Endpoint 
For the primary efficacy endpoint, VOCs that meet the protocol-specified definition as assessed 
by the VOC Adjudication Committee with an onset date from randomization (Day 1) through the 
90-day study period (Day 91), inclusive, will be used in the analysis.  
The proportion of participants with at least 1 VOC that required admission to a healthcare 
facility and treatment with parenteral pain medication within 90 days (primary endpoint) from 
randomization will be compared between treatment groups using the exact Cochran-Mantel-
Haenszel (CMH) general association test, stratified by the randomization stratification factors 
(baseline HU use [yes; no] and number of VOCs in the 12 months prior to study entry [2 to 4 
episodes; 5 to 10 episodes]). 
Every effort will be made to obtain the 90-day re-admission status for a VOC for each 
participant. For purposes of the primary analysis, participants with an unknown 90-day re-
admission status (e.g., participants who discontinue the study prior to 90 days and without a re-
admission for a VOC) will be classified as having experienced at least 1 protocol-defined VOC 
within 90 days (i.e., counted as “failure”). A sensitivity analyses based on observed data (i.e., 
without imputation of re-admission for early study discontinuation) will be performed to assess 
the robustness of the primary analysis results to assumptions regarding drop-outs. 
SAS code for the exact CMH test similar to the following will be used for the primary analysis 
(where trtgrp is the randomized treatment group and response is an indicator variable for 
occurrence of at least one protocol-defined VOCs during the 90 days post randomization): 
PROC LOGISTIC; 
 CLASS trtgrp /PARAM=REF; 
 MODEL response (event=’Yes’) = trtgrp;  
 STRATA = <categorical variables>; 
 EXACT trtgrp; 
RUN; 
Similarly, the proportion for each treatment group and the difference in proportions between 
treatment groups (inclacumab versus placebo) will be estimated by the weighted average of the 
observed proportions and the differences in observed proportions over the strata using the CMH 
weights and 95% confidence intervals (CIs) provided (Mehrotra and Railkar 2000). 
  
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5.3.2. 
Secondary Efficacy Endpoints 
5.3.2.1. 
Time to First VOC 
Time to first VOC that required admission to a healthcare facility and treatment with parenteral 
pain medication within 90 days will be measured from randomization (Day 1) to onset date of 
the first protocol-defined VOC event. For participants who do not experience a protocol-defined 
VOC within 90 days of randomization, time to first VOC will be censored at the end of their 
time at risk (participant’s end of study date or Study Day 91, whichever is earlier).  
Summary statistics will include number of subjects with event, number of subjects censored, 
median time to event along with 95% CI for the median and quartiles. The CIs for the median 
will be calculated according to Brookmeyer and Crowley (1982) and the CIs for the survival 
function estimates at fixed time points will be derived using the log-log transformation according 
to Kalbfleisch and Prentice (2002) (conftype=loglog default option in SAS Proc LIFETEST) 
with back transformation to a CI on the untransformed scale. The estimate of the standard error 
will be computed using Greenwood’s formula. 
Treatment comparison between inclacumab and placebo will be performed based on log-rank test 
stratified by the randomization factors of baseline HU use and number of VOCs in the 12 months 
prior to study entry. Kaplan-Meier plots will be generated. A Cox regression model will be used 
to estimate the hazard ratio between the inclacumab and placebo groups, as appropriate.  
5.3.2.2. 
Participant Status with at Least 1 VOC Requiring Re-Admission with 30 Days 
For the proportion of participants with at least 1 VOC that required admission to a healthcare 
facility and treatment with parenteral pain medication within 30 days, the same statistical 
methods used for the primary efficacy endpoint will be used. 
5.3.2.3. 
Rate of VOCs Leading to Healthcare Visit and Requiring Parenteral Pain 
Medication 
For the rate of VOCs leading to a healthcare visit within 90 days, the total number of VOCs in 
the 90-day period will be compared between the treatment groups using a negative 
binomial regression model stratified by the randomization factors of baseline HU use and 
number of VOCs in the 12 months prior to study entry. For each participant, the time period at 
risk for evaluation of VOCs is from date of randomization (Day 1) to the participant’s end of 
study date or Study Day 91, whichever is earlier. 
The regression model will include covariates for treatment group (inclacumab, placebo) and the 
randomization stratification factors. The logarithm of observed patient-time at risk will be used 
as an offset term in the model to account for different lengths of follow-up across participants. 
The rate of VOCs adjusted for the specified baseline covariates will be estimated for each 
treatment arm based on the regression model. Similarly, the ratio of the VOC rate (inclacumab 
versus placebo) along with the associated 95% confidence interval (CI) and p-value will be 
estimated from the regression model. 
A plot of the mean cumulative function of VOC episodes will be presented using recurrent 
events analysis methods.  
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5.3.3. 
Exploratory Efficacy Endpoints 
Exploratory endpoints will be summarized by descriptive statistics. For comparison between 
inclacumab and placebo groups, point estimates, CIs, and p-values may be presented as 
appropriate, without adjustment for multiplicity. 
Time to second VOC that required admission to a healthcare facility and treatment with 
parenteral pain medication within 90 days will be measured from randomization (Day 1) to onset 
date of the second protocol-defined VOC event and summarized using the same methodology as 
for time to first VOC (secondary endpoint). 
The rate of complicated VOCs, rate of inpatient hospital admissions, and number of days of 
inpatient hospitalization for any reason during the 90 days following randomization will be 
summarized descriptively, using similar patient-time methodology as the rate of VOCs leading to 
a healthcare visit (secondary endpoint). 
The proportion of participants rated as “very much improved” or “much improved” based on the 
PGI-C at Day 46 and Day 91 will be summarized descriptively by timepoint and treatment 
group. A similar analysis will be performed for the proportion of participants rated as “very 
much improved” or “much improved” based on the CGI-C. 
A cumulative score for the ASCQ-Me Pain Impact (Short Form) will be calculated for each 
participant and assessment timepoint. For each assessment a cumulative score will only be 
calculated if all five questions on the form were answered. The cumulative score is the sum of 
the individual raw scores according to the response mapping in Table 3. 
Table 3: 
ASCQ-Me Pain Impact (Short Form) Response Mapping 
Response to Each Question 
Raw Score 
Always 
1 
Often 
2 
Sometimes 
3 
Rarely 
4 
Never 
5 
Changes from baseline over time to Day 91 will be calculated and summarized descriptively by 
timepoint (weekly) and treatment group. 
5.4. 
Safety Analyses 
Safety analyses will be based on the safety patient population (Section 4.2), with participants 
grouped according to the actual study treatment received. Safety will be assessed through 
descriptive summaries of adverse events, clinical laboratory test results, and vital signs. 
5.4.1. 
Exposure to Study Drug and Compliance 
Exposure to study drug (amount of study drug administered) will be summarized by treatment 
group. 
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For Day 1, the actual dose administered (in mg) and the difference between the planned dose and 
actual dose administered (in mg/kg) will be summarized descriptively by treatment group. For 
each participant, the percent compliance with study drug will be calculated based on the ratio of 
the actual dose administered (mg/kg) and the planned dose (i.e., 30 mg/kg). The percent 
compliance will be summarized by treatment group. 
In addition, the number and percentage of participants with an infusion interruption will be 
summarized by treatment group. 
5.4.2. 
Adverse Events 
Adverse events will be classified according to MedDRA; the most current version at the time of 
analysis will be used. Severity of adverse events will be graded using the National Cancer 
Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) version 5.0, when 
possible, or based on the protocol-specified grading for adverse events not covered in the NCI 
CTCAE. 
Summaries of treatment emergent adverse events (TEAEs), defined as adverse events that occur 
on or after Day 1 of study treatment or the worsening of a preexisting condition on or after Day 1 
of study treatment, will be tabulated by system organ class (SOC) and preferred term, as 
appropriate.  
Summaries of TEAEs by treatment group will be provided for the following categories: 
x All TEAEs 
x All TEAEs by maximum severity 
x TEAEs assessed as related to study drug by the investigator 
x TEAEs leading to study drug interruption 
x TEAEs leading to study drug discontinuation 
x Adverse events of special interest (AESIs) (i.e., infusion-related reaction TEAEs) 
x Treatment-emergent serious adverse events (SAEs) 
Listings for all adverse events, TEAEs leading to study drug discontinuation, AESIs, SAEs, and 
deaths (if any) will be provided. 
VOC events will be collected and summarized separately (uncomplicated VOC, acute chest 
syndrome [ACS], hepatic sequestration, splenic sequestration, and priapism). 
5.4.3. 
Clinical Laboratory Assessments 
Laboratory abnormalities assessed by the Investigator as clinically significant will be recorded as 
adverse events. 
Descriptive summaries of laboratory parameters (hematology, serum chemistry, and coagulation) 
at baseline and each evaluation post baseline, as well as changes from baseline over time, will be 
provided by treatment group. If any of the results are below the limit of quantitation or above the 
limit of quantitation, then the numerical limit will be used in the descriptive summaries. 
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G B T, a w h oll y o w ne d s u bsi diar y of Pfizer 
                                    I nclac u ma b 
Statistical A nal ysis Pla n 
           G B T 2 1 0 4- 1 3 2 ( C 5 3 6 1 0 0 2) 
 
 
P FI Z E R C O N FI D E N TI A L 
Pa ge 2 1 
1 0 A u g ust 2 0 2 3 
 
La b orat or y a b n or malities will be  gr a de d vi a t he C o m m o n Ter mi n ol o g y Criteri a f or A d verse 
E ve nt ( C T C A E). A tr eat me nt-e mer ge nt la b or at or y a b n or malit y is a n y p ost- baseli ne la b orat or y 
assess me nt perf or me d o n or after i niti ati o n of st u d y dr u g w hic h de m o nstrates a n i n crease of 1 
gra de or m ore fr o m t he baseli ne t o xicit y val ue. If t h e baseli ne v al ue is missi n g, a n y gra de d 
a b n or malit y ( gra d e 1 or hi g her) t h at oc c urs f oll o wi n g i niti ati o n of st u d y dr u g will be d ee m e d 
treat me nt e mer ge nt. T he n u m ber a n d p erce nta ge of p artici pa nts e x p erie nci n g tre at m e nt-e mer ge nt 
la b orat or y a b n or malities will be s u m mari ze d b y tr eat me nt gr o u p. L a b orat or y a b n or malit y s hifts 
fr o m baseli n e t hr o u g h e ac h e val uati o n p ost baseli ne will be s u m m ariz e d b y treat me nt gr o u p. 
5. 4. 4.  
Vit al Si g ns a n d Wei g ht 
Descri pti ve s u m mari es of vital si g ns (e. g., s yst olic a n d diast oli c bl o o d press ure, he art rat e, a n d 
b o d y te m per at ure) at baseli ne a n d eac h e val u ati o n p ost baseli ne, as well as c h a n ges fr o m 
baseli ne o v er ti me, will be ge ner ate d b y trea t me nt gr o u p. I n a d diti o n, d escri pti ve s u m m aries of 
wei g ht at b aseli n e will b e ge nerate d b y tr eat me nt gr o u p. 
5. 5.  
E x pl or at or y P K, A D A, a n d P D A n al yses 
5. 5. 1.  
P h ar m ac o ki netic A n al yses 
5. 5. 2.  
A nti- dr u g A nti b o d y A n al yses  
D o c U UI D : 4f 7 9 1 7 6 4- 8 3 e b- 4 3 c 6- 8 7 2 7- 5 5 2f 3 8 2 9 cf 6 e
0 9 0 1 7 7 e 1 9 e 5 5 0f 9 c\ A p p r o v e d\ Ap p r o v e d O n: 1 7- A ug - 2 0 2 3 1 5: 3 6 (G M T )
C CI
C CI


G B T, a w h oll y o w ne d s u bsi diar y of Pfizer 
                                    I nclac u ma b 
Statistical A nal ysis Pla n 
           G B T 2 1 0 4- 1 3 2 ( C 5 3 6 1 0 0 2) 
 
 
P FI Z E R C O N FI D E N TI A L 
Pa ge 2 2 
1 0 A u g ust 2 0 2 3 
 
5. 5. 3.  
P h ar m ac o d y n a mic A n al yses 
 
D o c U UI D : 4f 7 9 1 7 6 4- 8 3 e b- 4 3 c 6- 8 7 2 7- 5 5 2f 3 8 2 9 cf 6 e
0 9 0 1 7 7 e 1 9 e 5 5 0f 9 c\ A p p r o v e d\ Ap p r o v e d O n: 1 7- A ug - 2 0 2 3 1 5: 3 6 (G M T )
C CI
C CI


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6. 
TABLES, LISTINGS, AND FIGURES 
A separate document will provide mockups of the tables, listings, and figures that support the 
analyses proposed in this SAP. 
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7. 
REFERENCES 
1. Birkmeyer JD et al. The impact of the COVID-19 pandemic on hospital admissions in the 
United States. Health Affairs 2020;39(11):2010–17. 
2. Brookmeyer, R, and Crowley, JJ. (1982). A Confidence Interval for the Median Survival 
Time. Biometrics, 38: 29–41. 
3. Kalbfleisch, JD, and Prentice,, RL. (2002). Statistical Analysis of Failure Time Data. 2nd 
Edition. Hoboken, Wiley Interscience. 
4. Kumar V, Chaudhary N, and Achebe MM. Epidemiology and Predictors of all-cause 30-Day 
readmission in patients with sickle cell crisis. Scientific Reports 2020;10:2082. 
5. McFarling UL. ‘They are really afraid’: Fears about Covid-19 are complicating care for 
patients with sickle cell.  STATNews SEPTEMBER 17, 2020 
https://www.statnews.com/2020/09/17/sickle-cell-treatment-covid19-pandemic/ 
6. Mehrotra DV, Railkar R. Minimum risk weights for comparing treatments in stratified 
binomial trials. Stat Med 2000;19:811–25. 
7. Powell L. Sickle Cell Disease Brings Higher Risk of Coronavirus Death, but Inconsistent 
Treatment. The City, Jul 14, 2020. 
https://www.thecity.nyc/coronavirus/2020/7/14/21324875/sickle-cell-disease-coronavirus-
death-new-york-city 
 
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