Clinical trial NCT03704298
Conditions: Relapsed/Refractory Large B-cell Lymphoma
Sponsor: Kite, A Gilead Company
Start date: 2018-11-20; Completion date: 2022-12-15
Enrollment: 15 participants; Allocation: NON_RANDOMIZED; Intervention model: SEQUENTIAL

Brief summary:
The primary objectives of this study are:

Phase 1: To evaluate the safety of axicabtagene ciloleucel in combination with utomilumab and to identify the most appropriate dose and timing of utomilumab to carry forward into Phase 2

Phase 2: To evaluate the efficacy of axicabtagene ciloleucel and utomilumab as measured by complete response rate in participants with refractory large B-cell lymphoma

Detailed description:
This study was intended to be a Phase 1/2. Enrollment was stopped prior to completion of Phase 1 portion of the study based on the sponsor's decision to end the program. No participants were enrolled in Phase 1 Cohort 5 and Phase 2.

After the study ends, participants who received an infusion of axicabtagene ciloleucel and utomilumab will complete the remainder of the 15-year follow-up assessments in a separate Long-term Follow-up study, KT-US-982-5968 (NCT05041309).

Arms: arm 1: Participants will receive cyclophosphamide 500 mg/m\^2/day intravenously (IV) and fludarabine 30 mg/m\^2/day IV conditioning chemotherapy for 3 days followed by axicabtagene ciloleucel administered as a single IV infusion at a target dose of 2 x 10\^6 anti-cluster of differentiation (CD) 19 chimeric antigen receptor (CAR) transduced autologous T cells/kg on Day 0. For participants weighing ≥ 100 kg, a maximum flat dose of axicabtagene ciloleucel at 2 x 10\^8 anti-CD19 CAR T cells will be administered. Participants also will receive utomilumab 10 mg on Day 1, IV infusion, once every 4 weeks (Q4W) for 6 months or until progressive disease, whichever came first. arm 2: Participants will receive cyclophosphamide 500 mg/m\^2/day IV and fludarabine 30 mg/m\^2/day IV conditioning chemotherapy for 3 days followed by axicabtagene ciloleucel administered as a single IV infusion at a target dose of 2 x 10\^6 anti-CD19 CAR transduced autologous T cells/kg on Day 0. For participants weighing ≥ 100 kg, a maximum flat dose of axicabtagene ciloleucel at 2 x 10\^8 anti-CD19 CAR T cells will be administered. Participants also will receive utomilumab 30 mg on Day 1, IV infusion, Q4W for 6 months or until progressive disease, whichever came first. arm 3: Participants will receive cyclophosphamide 500 mg/m\^2/day IV and fludarabine 30 mg/m\^2/day IV conditioning chemotherapy for 3 days followed by axicabtagene ciloleucel administered as a single IV infusion at a target dose of 2 x 10\^6 anti-CD19 CAR transduced autologous T cells/kg on Day 0. For participants weighing ≥ 100 kg, a maximum flat dose of axicabtagene ciloleucel at 2 x 10\^8 anti-CD19 CAR T cells will be administered. Participants also will receive utomilumab 100 mg on Day 1, IV infusion, Q4W for 6 months or until progressive disease, whichever came first. arm 4: Participants will receive cyclophosphamide 500 mg/m\^2/day IV and fludarabine 30 mg/m\^2/day IV conditioning chemotherapy for 3 days followed by axicabtagene ciloleucel administered as a single IV infusion at a target dose of 2 x 10\^6 anti-CD19 CAR transduced autologous T cells/kg on Day 0. For participants weighing ≥ 100 kg, a maximum flat dose of axicabtagene ciloleucel at 2 x 10\^8 anti-CD19 CAR T cells will be administered. Participants also will receive utomilumab 200 mg on Day 1, IV infusion, Q4W for 6 months or until progressive disease, whichever came first. arm 5: Participants will receive cyclophosphamide 500 mg/m\^2/day IV and fludarabine 30 mg/m\^2/day IV conditioning chemotherapy for 3 days followed by axicabtagene ciloleucel administered as a single IV infusion at a target dose of 2 x 10\^6 anti-CD19 CAR transduced autologous T cells/kg on Day 0. For participants weighing ≥ 100 kg, a maximum flat dose of axicabtagene ciloleucel at 2 x 10\^8 anti-CD19 CAR T cells will be administered. Participants also will receive utomilumab 400 mg on Day 1, IV infusion, Q4W for 6 months or until progressive disease, whichever came first.

However, as the study was early terminated, no participants were enrolled in Cohort 5 of the study. arm 6: Participants will receive cyclophosphamide 500 mg/m\^2/day IV and fludarabine 30 mg/m\^2/day IV conditioning chemotherapy for 3 days followed by axicabtagene ciloleucel and utomilumab based on the dose and schedule selected to move forward from the Phase 1 portion of the study as recommended by the internal safety review team.

However, as the study was early terminated, no participants were enrolled in Phase 2 of the study.
Interventions: intervention 1: Cyclophosphamide intervention 2: Fludarabine intervention 3: Axicabtagene Ciloleucel intervention 4: Utomilumab
Intervention descriptions: intervention 1: Administered according to package insert intervention 2: Administered according to package insert intervention 3: A single infusion of chimeric antigen receptor (CAR)-transduced autologous T cells administered intravenously intervention 4: Administered as an IV infusion

Protocol documents:
SAP_002.pdf: 
 
TRANSLATIONAL STATISTICAL ANALYSIS PLAN  
KTE-C19-111 END OF PHASE I ANALYSIS 
 
 
 
 
CONFIDENTIALITY NOTICE 
This document contains confidential information of Kite Pharma Inc., a wholly owned 
subsidiary of Gilead Sciences Inc.  This document must not be disclosed to anyone other than 
the site research staff and members of the Institutional Review Board/Independent Ethics 
Committee, a scientific review board or an equivalent.  The information in this document 
cannot be used for any purpose other than the conduct of the clinical investigation without the 
prior written consent of Kite Pharma Inc.  Questions regarding how this document should be 
used or the conduct of the clinical trial should be directed to the key sponsor contacts. 
Sponsor: 
Kite Pharma, Inc. 
2400 Broadway 
Santa Monica, CA 90404 
United States of America 
 
 
Product Name: 
KTE-C19 
 
 
Protocol: 
A Phase 1/2 MulticenterStudy Evaluating the Safety and 
Efficacy of Axicabtagene Ciloleucel in Combination with 
Utomilumab in Subjects with Refractory Large B-Cell 
Lymphoma (ZUMA-11) 
 
 
Protocol Version Number 
and Date: 
Amendment 4.0; 09 August 2021 
 
 
TSAP Release Date: 
13 September 2021 Final Version 1.0 
 
 
Replaces Previous 
Version(s): 
 


Axicabtagene ciloleucel KTE-C19 
Translational Statistical Analysis Plan 
Kite Pharma, Inc. 
KTE-C19-111 (ZUMA-11) 
Draft 
CONFIDENTIAL 
Page 2 
25 March 2021 
TABLE OF CONTENTS 
 
TABLE OF CONTENTS .............................................................................................................................................. 2 
LIST OF TABLES ......................................................................................................................................................... 2 
LIST OF ABBREVIATIONS ........................................................................................................................................ 3 
1. 
INTRODUCTION ................................................................................................................................................ 4 
2. 
OBJECTIVES ....................................................................................................................................................... 5 
2.1. 
Objectives ................................................................................................................................................. 5 
2.2. 
Hypothesis ................................................................................................................................................ 5 
3. 
ENDPOINTS, SUBGROUPS AND COVARIATES ........................................................................................... 6 
3.1. 
Biomarker datasets ................................................................................................................................... 6 
3.2. 
Endpoints ................................................................................................................................................. 8 
4. 
DEFINITIONS ...................................................................................................................................................... 9 
4.1. 
General ..................................................................................................................................................... 9 
4.2. 
Key Measurements of PK: Anti-CD19 CAR+ T-Cell .............................................................................. 9 
4.3. 
Key Measurements of PD: Serum Analytes ............................................................................................. 9 
4.4. 
Key Measurements of Product Characteristics ....................................................................................... 10 
 
 
4.6. 
Key Measurements of CSF analytes ...................................................................................................... 10 
4.7. 
Key Measurements of Utomilumab ....................................................................................................... 11 
5. 
ANALYSIS SETS............................................................................................................................................... 12 
6. 
STATISTICAL ANALYSIS ............................................................................................................................... 13 
6.1. 
General Methods .................................................................................................................................... 13 
6.2. 
Analysis .................................................................................................................................................. 13 
6.2.1. 
Characterize the anti-CD19 CAR T cell expansion (Pharmacokinetic) and 
serum analyte (pharmacodynamic) profile ........................................................................... 13 
6.2.2. 
Characterize the product attributes ....................................................................................... 13 
6.2.3. 
Characterize Utomilumab Pharmacokinetic and Immunogenicity ....................................... 13 
7. 
REFERENCES ................................................................................................................................................... 14 
8. 
APPENDIX ......................................................................................................................................................... 15 
 
Appendix 1. 
Analytic Visit Windows ......................................................................................................... 16 
Appendix 2. 
CSF T-cell Analytes ............................................................................................................... 18 
Appendix 3. 
Using Trapezoidal Rule to Approximate the AUC ................................................................. 19 
 
 
LIST OF TABLES 
 
Table 1. 
Data overview on assay methods and biomarker lists for all cohorts ....................................... 6 
Table 2. 
Analysis Visit Windows for Phase 1 Cohort 1, 2, 3 and 4 ...................................................... 16 
Table 3. 
CSF T-cell analytes ................................................................................................................ 18 
 
 
CCI


Axicabtagene ciloleucel KTE-C19 
Translational Statistical Analysis Plan 
Kite Pharma, Inc. 
KTE-C19-111 (ZUMA-11) 
Draft 
CONFIDENTIAL 
Page 3 
25 March 2021 
LIST OF ABBREVIATIONS  
ADA 
Anti-drug antibodies 
AUC 
Area under the curve 
Axicabtagene ciloleucel / KTE-C19 
autologous T cells transduced with retroviral vector containing anti-CD19 
CD28/CD3 zeta chimeric antigen receptor 
CAR 
Chimeric antigen receptor 
CI 
Confidence Interval 
CRP 
C-reactive protein 
CSF 
Cerebrospinal fluid 
mITT 
Modified intent-to-treat 
Nab 
Neutralizing antibody 
ORR 
Objective response rate 
PBMC 
Peripheral blood mononuclear cells 
PK/PD 
Pharmacokinetic/pharmacodynamic 
SAP 
Statistical analysis plan 
Study day 0 
The first day that axicabtagene ciloleucel is administered to the subject 
TSAP 
Translational tatistical analysis plan 
 
 
 


Axicabtagene ciloleucel KTE-C19 
Translational Statistical Analysis Plan 
Kite Pharma, Inc. 
KTE-C19-111 (ZUMA-11) 
Draft 
CONFIDENTIAL 
Page 4 
25 March 2021 
1. 
INTRODUCTION  
This translational statistical analysis plan (TSAP) outlines the end of Phase I analyses to be 
conducted for Pharmacokinetic (levels of anti-CD19 CAR T cells in blood), pharmacodynamics 
(serum analytes), product characteristics, and other biomarkers in support of end of Phase I 
analysis for KTE-C19 in combination with Utomilumab in Subjects with Refractory Large B-
Cell Lymphoma. The analysis will be conducted for biomarker data within protocol KTE-C19-
111 (ZUMA-11), amendment 3 dated 14 July 2020. 




Axicabtagene ciloleucel KTE-C19 
Translational Statistical Analysis Plan 
Kite Pharma, Inc. 
KTE-C19-111 (ZUMA-11) 
Draft 
CONFIDENTIAL 
Page 6 
25 March 2021 
3. 
ENDPOINTS, SUBGROUPS AND COVARIATES 
3.1. 
Biomarker datasets  
Table 1. 
Data overview on assay methods and biomarker lists for all cohorts 
Data type 
Assay method/ 
Sample type 
Assessment time points 
Biomarker set 
Pharmacokinetic 
data (Anti- CD19 
CAR T cell levels 
in blood) 
qPCR/PBMC 
samples 
Baseline, Day 1, Day 3, Day 7, 
Day 10, Week 2, Week 3, 
Week 4, Day 31, Week 8, Day 
59, Week 12, Week 16, Week 
20, Month 6, Month 9, Month 
12, Month 15, 
Month 18, Month 24, Month 
36, Month 48, Month 60 
number of CAR T cells (/uL),  
Pharmacodynamic 
data (levels of 
serum analytes) 
soluble analyte 
assays/serum 
samples 
Baseline, Day 0, Day 1, Day 3, 
Day 7, Week 2, Week 3, Week 
4, Day 31, Week 8, Day 59, 
Week 12  
• 
Serum levels of following 
key cytokines, chemokines, 
and immune effector 
molecules will be reported- 
• 
CRP, CXCL10, Ferritin, 
SAA, Granzyme B, 
ICAM-1, IFN-gamma, 
IL-1RA, IL‑2, IL-2Ralpha, 
IL-5, IL-6, IL-7, IL-8, IL-4, 
IL-10, IL-15, Perforin, 
TNF-alpha, VCAM-1 and 
GM-CSF 
Pharmacokinetic 
and 
immunogenicity 
data 
(Utomilumab) 
Immunoassays/serum 
samples 
• 
Utomilumab 
concentrations: Day 1, 
Day 3, Day 7, Week 2, 
Week 3, Week 4, Day 31, 
Week 12, Month 6 
• 
Anti-Drug Antibody: 
Day 1, Week 4, Month 6 
• 
Anti-Drug Antibody  
• 
Utomilumab concentrations 
(ng/mL) 


Axicabtagene ciloleucel KTE-C19 
Translational Statistical Analysis Plan 
Kite Pharma, Inc. 
KTE-C19-111 (ZUMA-11) 
Draft 
CONFIDENTIAL 
Page 7 
25 March 2021 
Data type 
Assay method/ 
Sample type 
Assessment time points 
Biomarker set 
Product data 
Product 
characteristics, 
several assays/ 
product samples 
 
Total Number of T Cells,  
Total Number of CAR T Cells,  
Total number of Tnaive cells 
Transduction Rate (%),  
CD3 (% and number), CD4/CD8 
Ratio, T naïve/CM/EM/EFF 
cells (% and number), 
(% Tnaive + % T central 
memory) / (% T effector 
memory + % T effector cells), 
IFN-gamma level (pg/ml), 
Vector Copy Number, % 
viability, CD4 Cells (% and 
number ), CD8 Cells (% and 
number),  CCR7+ (Tnaive + 
Tcm) % and number,  CCR7- 
(Tem + Teff) %, Total number 
of CCR7+ T cells infused 
CSF analytes 
soluble analyte 
assays/CSF samples 
Baseline, Day 5, Week 4 
Biomarker list are the same with 
those in the pharmacodynamic 
serum analytes 
CSF cell subsets 
Flow cytometry/CSF 
samples 
Baseline, Day 5, Week 4 
Analytes are listed in 
Appendix 2  
 
Zuma-11 end of Phase I analysis will include the following data. The analysis will be outlined in 
the following sections. 
• Axi-cel Pharmacokinetic (Anti-CD19 CAR T cell levels in blood) 
• Pharmacodynamics (levels of serum analytes) 
• Utomilumab pharmacokinetic and immunogenicity (ADA) 
• Product attributes 


Axicabtagene ciloleucel KTE-C19 
Translational Statistical Analysis Plan 
Kite Pharma, Inc. 
KTE-C19-111 (ZUMA-11) 
Draft 
CONFIDENTIAL 
Page 8 
25 March 2021 
3.2. 
Endpoints 
All definitions can be generally applied to anti-CD19 CAR T pharmacokinetic and/or 
pharmacodynamic endpoints. Detailed definitions can be found in Section 4. 
• Change from baseline at Day X 
• Fold change  
1) Fold change from baseline at Day X 
2) Fold change from Day 0 
• Peak 
• Time to peak 
• Area-Under-Curve (AUC)  
All measurable values at each visit will be used as main endpoints for all biomarkers listed in 
Table 1 as following 
• Levels of anti-CD19 CAR T cells in blood samples measured as anti-CD19 CAR+ cells/µL 
by visit, peak, Day 0-28 AUC, and time to peak (details for the derivations are included in 
the definition section) 
• Levels of serum analytes by visit, fold change from baseline, Day 0, peak, Day 0-28 AUC, 
and time to peak 
• Product attributes measurements after product manufacturing and prior to dosing. 


Axicabtagene ciloleucel KTE-C19 
Translational Statistical Analysis Plan 
Kite Pharma, Inc. 
KTE-C19-111 (ZUMA-11) 
Draft 
CONFIDENTIAL 
Page 9 
25 March 2021 
4. 
DEFINITIONS 
4.1. 
General 
• Study day 0 is defined as the day the subject received the first KTE-C19 infusion.  The day 
prior to study day 0 will be study day -1.  Any days after enrollment and prior to study day -1 
will be sequential and negative integer-valued. 
• Baseline is defined as the last non-missing value measured on or prior to conditioning 
chemotherapy. 
4.2. 
Key Measurements of PK: Anti-CD19 CAR+ T-Cell  
• The expansion and persistence of anti-CD19 CAR T cells in peripheral blood will be 
measured by qPCR analysis.  
• Scheduled blood draw for anti-CD19 CAR T cell  
This TSAP will focus on the anti-CD19 CAR T cell data collected as per planned assessment. 
The schedule of assessments and the analytic visit windows are defined in Appendix 1. 
• Number of anti-CD19 CAR T (cells/ uL blood) is defined as: 
White Blood Cell counts (per uL) x (Monocytes (%) + Lymphocytes (%)) x CAR+ PBMCs 
(%) 
• Baseline number of anti-CD19 CAR T (cells/ µL) is defined as 0 since the axicabtagene 
ciloleucel infused in day 0, and number of CAR T (cell/µL) will not be derived. 
• Peak of anti-CD19 CAR T cell (cells/ uL blood) is defined as the maximum absolute 
number of anti-CD19 CAR T cells in serum attained after Day 0. 
• Time-to-Peak of anti-CD19 CAR T cell (days) is defined as “Peak Date – KTE-C19 
Dosing Date + 1”. 
• Area-Under-Curve (AUC) of level of anti-CD19 CAR T cell (cells/ uL ▪ days) is defined 
as the area under the curve in a plot of levels of anti-CD19 CAR T cells against scheduled 
visits from day 0 to day 28. This AUC measures the total levels of anti-CD19 CAR T cells 
overtime. Given the anti-CD19 CAR T cell is measured at certain discrete time points, the 
trapezoidal rule will be used to estimate the AUCs. 
4.3. 
Key Measurements of PD: Serum Analytes 
• Scheduled blood draw for serum analytes: Within approximately 5 days of eligibility 
confirmation (Enrollment/Leukapheresis) as baseline, Day 0, Day 1, Day 3, Day 7, Week 2 
(Day 14) (± 2 days),  Week 3 (± 2 days), Week 4 (Day 29), Day 31, Week 8 ,  and Week 12. 






Axicabtagene ciloleucel KTE-C19 
Translational Statistical Analysis Plan 
Kite Pharma, Inc. 
KTE-C19-111 (ZUMA-11) 
Draft 
CONFIDENTIAL 
Page 12 
25 March 2021 
5. 
ANALYSIS SETS  
• Safety analysis set (per clinical SAP) will be used if not specified.  
• The ‘Modified intent-to-treat (mITT)’ analysis set will be used to be consistent with Phase 1 
efficacy analysis in CSR.  
Generally, this TSAP will utilize the same efficacy analysis strategy in CSR, such as analysis 
sets and related outcome definitions. 




Axicabtagene ciloleucel KTE-C19 
Translational Statistical Analysis Plan 
Kite Pharma, Inc. 
KTE-C19-111 (ZUMA-11) 
Draft 
CONFIDENTIAL 
Page 14 
25 March 2021 
7. 
REFERENCES 
Brown LD, Cai TT, DasGupta A. Confidence Intervals for a Binomial Proportion and 
Asymptotic Expansions. The Annals of Statistics 2002;30 (1):160-201. 
Dunn OJ. Multiple Comparisons Using Rank Sums. Technometrics 1964;6 (3):241-52. 
Gelman A, Hill J. Linear regression. In: Gelman A, Hill J, eds.  Data Analysis Using Regression 
and Multilevel/Hierarchical Models. 5th ed. New York: Cambridge University 
Press; 2006: 31-77.  
Holm S. A Simple Sequentially Rejective Multiple Test Procedure. Scandinavian Journal of 
Statistics 1979;6 (2):65-70. 
Hommel G. A Stagewise Rejective Multiple Test Procedure Based on a Modified Bonferroni 
Test. Biometrika 1988;75 (2):383-6. 
Kruskal WH, Wallis WA. Use of Ranks in One-Criterion Variance Analysis. Journal of the 
American Statistical Association 1952;47 (260):583-621. 
Siegel S. The One-Sample Runs Test. In: Morgan CT, ed.  Nonparametric Statistics: For the 
Behavioral Sciences. New York: McGraw-Hill Book Company, Inc.; 1956: 75-
83.  
Wilcoxon F. Individual Comparisons by Ranking Methods. Biometrics Bulletin 1945;1 (6):80-3. 
 
 


Axicabtagene ciloleucel KTE-C19 
Translational Statistical Analysis Plan 
Kite Pharma, Inc. 
KTE-C19-111 (ZUMA-11) 
Draft 
CONFIDENTIAL 
Page 15 
25 March 2021 
8. 
APPENDIX 
Appendix 1. 
Analytic Visit Windows for Phase 1 Cohort 1, 2, 3 and 4 
Appendix 2. 
CSF T-cell Analytes 
Appendix 3. 
Using Trapezoidal Rule to Approximate the AUC 
 
 


Axicabtagene ciloleucel KTE-C19 
Translational Statistical Analysis Plan 
 
Kite Pharma, Inc. 
KTE-C19-111 (ZUMA-11) 
 
Draft 
CONFIDENTIAL 
Page 16 
25 March 2021 
Appendix 1. 
Analytic Visit Windows  
Table 2. 
Analysis Visit Windows for Phase 1 Cohort 1, 2, 3 and 4 
Timeline 
nCAR T 
Infusion 
UTO IV 
UTO Pharmacokinetic 
Visit Window 
UTO ADA Visit 
Window 
CART Pharmacokinetic 
Visit Window 
Pharmacodynamic Visit 
Window 
Baseline 
  
  
  
  
  
APDY < -5 
Day 0 
Day 0 
  
  
  
DAY 0 
DAY 0 
Day 1 
  
D1 (#1) 
D1 1 APDY, preUTO1 and 
posUTO1 (both 
measurements are taken in 
same day, use the lab time 
to identify pre/post UTO 
measurements) 
D1 1 APDY, preUTO1 
and posUTO1 (both 
measurements are taken 
in same day, use the lab 
time to identify pre/post 
UTO measurements) 
DAY 1 1 APDY= 
ADY_uto_dt1, preUTO1 
DAY 1 1 APDY= 
ADY_uto_dt1, preUTO1 
Day 3 
  
  
D3 3 APDY in [2, 5] 
D3 3 APDY in [2, 5] 
DAY 3 3 APDY in 
[ADY_uto_dt1+1, 5], 
posUTO1 
DAY 3 3 APDY in 
[ADY_uto_dt1+1, 5], 
posUTO1 
Day 7 
  
  
D7 7 APDY in [6, 10] 
D7 7 APDY in [6, 10] 
DAY 7 7 APDY in [6, 8] 
DAY 7 7 APDY in [6, 10] 
Day 10 
  
  
  
  
DAY 10 10 APDY in [9, 
12] 
  
Week 2 
  
  
Week 2 14 APDY in [11, 
17] 
Week 2 14 APDY in [11, 
17] 
Week 2 14 APDY in [13, 
17] 
Week 2 14 APDY in [11, 17] 
Week 3 
  
  
Week 3 21 APDY in [18, 
25] 
Week 3 21 APDY in [18, 
25] 
Week 3 21 APDY in [18, 
25] 
Week 3 21 APDY in [18, 25] 
Week 4 
  
D29 (#2) 
Week 4 29 APDY in 
[26,57], preUTO2 and 
posUTO2 (both 
measurements are taken in 
same day, use the lab time 
to identify pre/post UTO 
measurements) 
Week 4 29 APDY in 
[26,57], preUTO2 and 
posUTO2 (both 
measurements are taken 
in same day, use the lab 
time to identify pre/post 
UTO measurements) 
Week 4 29 APDY in [26, 
ADY_uto_dt2], 
D29preUTO2 
Week 4 29 APDY in [26, 
ADY_uto_dt2], 
D29preUTO2 


Axicabtagene ciloleucel KTE-C19 
Translational Statistical Analysis Plan 
 
Kite Pharma, Inc. 
KTE-C19-111 (ZUMA-11) 
 
Draft 
CONFIDENTIAL 
Page 17 
25 March 2021 
Timeline 
nCAR T 
Infusion 
UTO IV 
UTO Pharmacokinetic 
Visit Window 
UTO ADA Visit 
Window 
CART Pharmacokinetic 
Visit Window 
Pharmacodynamic Visit 
Window 
Week 4 
  
  
 Week 4 31 APDY in 
[ADY_uto_dt2+1,  
ADY_uto_dt3-1], 
D31posUTO2 ----
Cohort 4 only 
Week 4 31 APDY in 
[ADY_uto_dt2+1, 44], 
D31posUTO2 
Week 4 31 APDY in 
[ADY_uto_dt2+1, 44], 
D31posUTO2 
Week 8 
  
D57 (#3) 
  
  
Week 8 57 APDY in [45, 
ADY_uto_dt3], 
D57preUTO3 
Week 8 57 APDY in [45, 
ADY_uto_dt3], 
D57preUTO3 
Week 8 
  
  
  
 Week 8 59 APDY in 
[ADY_uto_dt3+1, 72], 
D59posUTO3 ----Cohort 
4 only 
Week 8 59 APDY in 
[ADY_uto_dt3+1, 72], 
D59posUTO3 
Week 8 59 APDY >= 
ADY_uto_dt3+1, 
D59posUTO3 
Week 12 
(Month 3) 
  
D85 (#4) 
Week 12 85 APDY in [58, 
127], preUTO4 and 
posUTO4 (both 
measurements are taken in 
same day, use the lab time 
to identify pre/post UTO 
measurements) 
Week 12 85 APDY in 
[73, 127], preUTO4 and 
posUTO4 (both 
measurements are taken 
in same day, use the lab 
time to identify pre/post 
UTO measurements) 
Week 12 85 APDY in [73, 
ADY_uto_dt4], 
D85preUTO4 
  
Week 16 
(Month 4) 
  
D113 
(#5) 
  
  
Week 16 113 APDY in 
[ADY_uto_dt4+1, 
ADY_uto_dt5], 
D113preUTO5 
  
Week 20 
(Month 5) 
  
D141 
(#6) 
  
  
Week 20 141 APDY in 
[ADY_uto_dt5+1, 
ADY_uto_dt6], 
D141preUTO6 
  
Week 24 
(Month 6)  
  
  
30 days follow last-UTO 
170 APDY >=128 
30 days follow last-UTO 
170 APDY >=128 
30 days follow last-UTO 
170 APDY >= 
ADY_uto_dt6+1 
  
 


Axicabtagene ciloleucel KTE-C19 
Translational Statistical Analysis Plan 
Kite Pharma, Inc. 
KTE-C19-111 (ZUMA-11) 
Draft 
CONFIDENTIAL 
Page 18 
25 March 2021 
Appendix 2. 
CSF T-cell Analytes 
Visits: Screening CSF, Post-CSF, Baseline CSF, Unscheduled CSF 
Table 3. 
CSF T-cell analytes 
Number 
Name of the analyte (cells/Volume unit) 
1 
CD14 (#, gated on CD45+) 
2 
CD19 (#, gated on CD45+/66b/14-) 
3 
CD3 (#, gated on CD45+/66b/14-) 
4 
CD3 CAR+ (#, gated on CD 45+/66b/14-/3+) 
5 
CD4 (#, gated of CD45+/66b/14-/3+) 
6 
CD4 CAR+ (#, gated on CD45+/66b/14-/3+/4+) 
7 
CD45 (#, gated on viable singlets cells) 
8 
CD56+CD3- (#, gated on CD45+/66b/14-) 
9 
CD56+CD3+(#, gated onCD45+/66b/14-) 
10 
CD66b (#, gated on CD45+) 
11 
CD8 (#, gated on CD45+/66b/14-/3+) 
12 
CD8 CAR+ (#, gated on CD45+/66b/14-/3+/8+) 
13 
CSF Volume 
14 
Viability (# Total Cells, Gated on Singlets) 
15 
Viability (# Viable Cells, Gated on Singlets) 
 


Axicabtagene ciloleucel KTE-C19 
Translational Statistical Analysis Plan 
Kite Pharma, Inc. 
KTE-C19-111 (ZUMA-11) 
Draft 
CONFIDENTIAL 
Page 19 
25 March 2021 
Appendix 3. 
Using Trapezoidal Rule to Approximate the AUC 
AUC ≈1
2 (𝑦𝑦0 + 𝑦𝑦1) ∙Δ𝑥𝑥+ 1
2 (𝑦𝑦1 + 𝑦𝑦2) ∙Δ𝑥𝑥+ 1
2 (𝑦𝑦2 + 𝑦𝑦3) ∙Δ𝑥𝑥+ ⋯ 
 
 
 
 
 
 
  
 

Prot_000.pdf: 


A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 2
0 7 J u ne 2 0 2 2
P R O T O C O L S Y N O P SI S
Title:
A P hase 1/ 2 M ultice nter St u d y E val uati n g t he Safet y a n d Efficac y of 
A xica bta ge ne Cil ole ucel i n C o m bi nati o n wit h Ut o mil u ma b i n 
S u bjects wit h Rela pse d/ Refract or y Lar ge B-cell L y m p h o ma
I n dic ati o n:
F or t he treat me nt of a d ult s u bjects wit h rela pse d/refract or y lar ge 
B-cell l y m p h o ma
St u d y Desi g n: 
T his is a P hase 1/ 2, o pe n-la bel, m ultice nter st u d y e val uati n g t he 
safet y a n d efficac y of a xica bta ge ne cil ole ucel i n c o m bi nati o n wit h 
ut o mil u ma b a d mi nistrati o n i n s u bjects wit h rela pse d/refract or y lar ge 
B-cell l y m p h o ma. T he trial will be se parate d i nt o 2 disti nct p hases 
desi g nate d as P hase 1 a n d P hase 2.
D uri n g P hase 1, u p t o 3 6 s u bjects wit h refract or y lar ge B-cell 
l y m p h o ma will be e nr olle d i n a 3 + 3 desi g n i n u p t o 6 of 1 0 p ossi ble 
c o h orts t o e val uate t he safet y of a xica bta ge ne cil ole ucel a n d 
ut o mil u ma b c o m bi nati o n re gi me ns. A xica bta ge ne cil ole ucel will be 
a d mi nistere d as a si n gle d ose, a n d ut o mil u ma b will be a d mi nistere d 
at escalati n g d oses.  S u bjects will be e nr olle d a n d treate d o ne at a 
ti me d uri n g t he P hase 1 p orti o n of t he st u d y. S u bject treat me nt wit h 
a xica bta ge ne cil ole ucel will be sta g gere d b y at least 2 wee ks. T he 
P hase 1 c o h orts a n d ut o mil u ma b re gi me ns a d mi nistere d are o utli ne d 
i n t he f oll o wi n g ta ble.
D ose Le vel *
C o h ort
First Ut o mil u m a b 
A d mi nistr ati o n
1
Da y 1
1 0 m g
1 A
Da y 2 1
2
Da y 1
3 0 m g
2 A
Da y 2 1
3
Da y 1
1 0 0 m g
3 A
Da y 2 1
4
Da y 1
2 0 0 m g
4 A
Da y 2 1
5
Da y 1
4 0 0 m g
5 A
Da y 2 1
* Ut o mil u ma b d oses re prese nt t otal d oses ( 1 0 m g, 3 0 m g, 1 0 0 m g, 2 0 0 m g, a n d 4 0 0 m g) 
a n d will be a d mi nistere d Q 4 W f or 6 m o nt hs or u ntil pr o gressi ve disease ( P D), w hic he ver 
c o mes first.


A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 3
0 7 J u ne 2 0 2 2
A safet y re vie w tea m ( S R T) t hat is i nter nal t o t he st u d y s p o ns or a n d 
at least o ne P hase 1 i n vesti gat or will re vie w safet y data after all 
s u bjects i n eac h P hase 1 c o h ort ha ve ha d t he o p p ort u nit y t o 
c o m plete t he d ose-li miti n g t o xicities ( D L T) wi n d o w. T he S R T will 
ma ke rec o m me n dati o ns o n f urt her st u d y c o n d uct of P hase 1 a n d 
pr o gressi o n t o P hase 2 as de picte d i n Fi g ure 5  a n d o utli ne d i n 
Secti o n 9. 1 0 .
I n P hase 2, a p pr o xi matel y 2 4 s u bjects will be e nr olle d t o recei ve 
treat me nt wit h a xica bta ge ne cil ole ucel a n d ut o mil u ma b base d o n t he 
d ose/re gi me n selecte d t o m o ve f or war d fr o m t he P hase 1 p orti o n of 
t he st u d y as rec o m me n de d b y t he S R T. 
I n de pe n de nt of t he c o h ort or p hase of t he st u d y, eac h s u bject will 
pr ocee d t hr o u g h t he f oll o wi n g st u d y peri o ds: 

Scree ni n g

E nr oll me nt/ Le u ka p heresis 

Bri d gi n g t hera p y; if a p plica ble 

C o n diti o ni n g c he m ot hera p y 

I n vesti gati o nal pr o d uct treat me nt (a xica bta ge ne cil ole ucel a n d 
ut o mil u ma b)

P ost-treat me nt assess me nt

L o n g-ter m f oll o w- u p ( L T F U)
F or st u d y re q uire me nt details assi g ne d t o eac h st u d y peri o d, refer t o 
t he sc he d ule of assess me nts ( S O As) a n d Secti o n 7 .
A st u d y sc he ma is pr o vi de d i n Fi g ure 1 .
St u d y O bjecti ves: 
Pri mar y o bjecti ve:

T he pri mar y o bjecti ve of P hase 1 is t o e val uate t he safet y of 
a xica bta ge ne cil ole ucel i n c o m bi nati o n wit h ut o mil u ma b a n d t o 
i de ntif y t he m ost a p pr o priate d ose a n d ti mi n g of ut o mil u ma b t o 
carr y f or war d i nt o P hase 2.

T he pri mar y o bjecti ve of P hase 2 is t o e val uate t he efficac y of 
a xica bta ge ne cil ole ucel a n d ut o mil u ma b, as meas ure d b y 
c o m plete res p o nse ( C R) rate, i n s u bjects wit h rela pse d/refract or y 
lar ge B-cell l y m p h o ma.
Sec o n dar y o bjecti ves:

Sec o n dar y o bjecti ves f or P hase 1 a n d P hase 2 will i ncl u de 
assessi n g t he safet y a n d t olera bilit y of a xica bta ge ne cil ole ucel 
a n d ut o mil u ma b, a d diti o nal efficac y e n d p oi nts, a n d le vels of 
a xica bta ge ne cil ole ucel i n bl o o d ( p har mac o ki netics [ P K]) a n d 
le vels of c yt o ki nes i n ser u m.


A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 4
0 7 J u ne 2 0 2 2
H y p ot hesis:
N o f or mal h y p ot hesis will be teste d i n t he st u d y. T he P hase 2 
p orti o n of t he st u d y is desi g ne d t o esti mate t he tr ue C R rate i n 
s u bjects wit h rela pse d/refract or y lar ge B-cell l y m p h o ma treate d wit h 
a xica bta ge ne cil ole ucel f oll o we d b y ut o mil u ma b.
Pri m ar y E n d p oi nts:

P hase 1: I nci de nce of a d verse e ve nts defi ne d as D L Ts

P hase 2: C o m plete res p o nse rate per t he L u ga n o Classificati o n 
{ C hes o n 2 0 1 4 }, (refer t o A p pe n di x 2 ), as deter mi ne d b y st u d y 
i n vesti gat ors
Sec o n d ar y 
E n d p oi nt(s):
P hase 1 a n d P hase 2:

O bjecti ve res p o nse rate ( C R + partial res p o nse [ P R]) per t he 
L u ga n o Classificati o n { C hes o n 2 0 1 4 }, as deter m i ne d b y st u d y 
i n vesti gat ors

D urati o n of res p o nse 

Pr o gressi o n-free S ur vi val per t he L u ga n o Classificati o n { C hes o n 
2 0 1 4 }, as deter m i ne d b y st u d y i n vesti gat ors 

O verall s ur vi val ( O S)

I nci de nce of a d verse e ve nts a n d cli nicall y si g nifica nt c ha n ges i n 
safet y la b val ues 

P K: Le vels of a xica bta ge ne cil ole ucel i n bl o o d  

P har mac o d y na mics: Le vels of c yt o ki nes i n ser u m  
S a m ple Size: 
A p pr o xi matel y 3 t o 6 0 s u bjects
P hase 1: a p pr o xi matel y 3 t o 3 6 s u bjects 
P hase 2: a p pr o xi matel y 2 4 s u bjects 
St u d y Eli gi bilit y: 
Refer t o Secti o n 5  f or a c o m plete a n d detaile d list of i ncl usi o n a n d 
e xcl usi o n criteria. 
Tre at me nt:
C o n diti o ni n g c he m ot hera p y treat me nt: 

A c o n diti o ni n g c he m ot hera p y re gi me n c o nsisti n g of 
c ycl o p h os p ha mi de 5 0 0 m g/ m 2/ da y a n d fl u dara bi ne 
3 0 m g/ m 2/ da y will be a d mi nistere d x 3 da ys pri or t o 
a xica bta ge ne cil ole ucel i nf usi o n. Refer t o Secti o n 6  f or 
c he m ot hera p y treat me nt details.


A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 5
0 7 J u ne 2 0 2 2
Bri d gi n g t hera p y peri o d, if a p plica ble:

N o n-c he m ot hera p y bri d gi n g t hera p y will be a d mi nistere d at t he 
discreti o n of t he i n vesti gat or.  F or s u bjects recei vi n g bri d gi n g 
t hera p y, refer t o Secti o n 6  f or bri d gi n g t hera p y details.
I n vesti gati o nal pr o d uct(s):

A xica bta ge ne cil ole ucel treat me nt c o nsists of a si n gle i nf usi o n of 
c hi meric a nti ge n rece pt or (C A R ) tra ns d uce d a ut ol o g o us T cells 
a d mi nistere d i ntra ve n o usl y at a tar get d ose of 2 x 1 0 6 a nti- C D 1 9 
C A R T cells/ k g.  Refer t o Secti o n 6  a n d Sectio n 7. 8  f or 
treat me nt details. 

Ut o mil u ma b treat me nt c o nsists of a n i ntra ve n o us i nf usi o n 
starte d o n eit her st u d y Da y 1 or st u d y Da y 2 1 a n d c o nti n ui n g 
Q 4 W f or 6 m o nt hs or u ntil P D, w hic he ver c o mes first. I n t he 
P hase 1 p orti o n of t he st u d y, t he d oses of ut o mil u ma b 
a d mi nistere d will be 1 0 m g, 3 0 m g, 1 0 0 m g, 2 0 0 m g, a n d 
4 0 0 m g. 
A d diti o nal a xica bta ge ne cil ole ucel a n d ut o mil u ma b re gi me ns ma y 
be e x pl ore d i n P hase 1 at t he rec o m me n dati o n of t he S R T. 
T he a xica bta ge ne cil ole ucel a n d ut o mil u ma b treat me nt i n P hase 2 
will f oll o w t he d osi n g re gi me n wit h t he best o verall be nefit/ris k 
pr ofile teste d i n P hase 1 as deter mi ne d b y t he safet y re vie w tea m 
(see Fi g ure 5 ). 
S u bjects w h o recei ve a xica bta ge ne cil ole ucel treat me nt, w hic h 
c o nsists of a si n gle i nf usi o n of C A R tra ns d uce d a ut ol o g o us T cells 
a d mi nistere d i ntra ve n o usl y o n Da y 0 at a tar get d ose of 2 x 1 0 6 
a nti- C D 1 9 C A R T cells/ k g ( ma xi m u m 2 x 1 0 8 a nti- C D 1 9 C A R T 
cells) a n d at least o ne d ose of ut o mil u ma b, will be c o nsi dere d 
e val ua ble i n t he efficac y set.
Pr oce d ures:
As o utli ne d i n t he sc he d ule of assess me nts ( S O As), s u bjects will 
u n der g o t he f oll o wi n g pr oce d ures:  c ollecti o n of i nf or me d c o nse nt 
a n d me dical hist or y, i ncl u di n g Easter n C o o perati ve O nc ol o g y Gr o u p 
( E C O G) perf or ma nce stat us.
S u bjects will als o u n der g o bl o o d dra ws f or t he f oll o wi n g:

L ocal e val uati o n: c o m plete bl o o d c o u nt ( C B C), c he mistr y 
pa nels, C-reacti ve pr otei n, a n d pre g na nc y test (if a p plica ble).

Ce ntral e val uati o n: C B C wit h differe ntial, P K assess me nts 
( ut o mil u ma b a n d a xica bta ge ne cil ole ucel), p har mac o d y na mic 
assess me nt (c yt o ki nes), ut o mil u ma b a nti- dr u g a nti b o dies 
( A D A), re plicati o n-c o m pete nt retr o vir us ( R C R), a n d l y m p h oc yte 
s u bsets ( B-cell rec o ver y).  




A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 7
0 7 J u ne 2 0 2 2
Fi g ure 1.
St u d y Sc he m a ( P h ase 1 a n d P h ase 2)
1   
N o n-c he m ot hera p y bri d gi n g t hera p y will be a d mi nistere d at t he discreti o n of t he i n vesti gat or.  F or s u bjects recei vi n g bri d gi n g t hera p y, refer t o Secti o n 6. 1. 2  f or 
bri d gi n g t hera p y details.
2   
A xica bta ge ne cil ole ucel is a d mi nistere d after a c o n diti o ni n g c he m ot hera p y re gi me n c o nsisti n g of c ycl o p h os p ha mi de 5 0 0 m g/ m 2/ da y a n d fl u dara bi ne 3 0 m g/ m2/ da y, 
a d mi nistere d x 3 da ys.  Refer t o Secti o n 6  f or c he m othera p y treat me nt details.
3   
A xica bta ge ne cil ole ucel treat me nt c o nsists of a si n gle i nf usi o n of C A R tra ns d uce d a ut ol o g o us T cells a d mi nistere d i ntra ve n o usl y o n Da y 0 at a tar get d ose of 2 x 1 0 6 
a nti- C D 1 9 C A R T cells/ k g ( ma xi m u m 2 x 1 0 8 a nti- C D 1 9 C A R T cells). 
4   
Ut o mil u ma b treat me nt c o nsists of a n i ntra ve n o us i nf usi o n starte d o n Da y 1 or Da y 2 1 a n d c o nti n ui n g Q 4 wee ks f or 6 m o nt hs or u ntil P D, w hic he ver c o mes first.   T he 
S R T will ma ke rec o m me n dati o ns o n f urt her st u d y c o n d uct of P hase 1.  A d diti o nal a xica bta ge ne cil ole ucel a n d ut o mil u ma b re gi me ns ma y be e x pl ore d i n P hase 1. 
5   
After t he e n d of K T E- C 1 9- 1 1 1, s u bjects w h o recei ve d a n i nf usi o n of a xica bta ge ne cil ole ucel a n d ut o mil u ma b will c o m plete t he re mai n der of t he 1 5- year f oll o w- u p 
assess me nts i n a se parate L o n g-ter m F oll o w- u p st u d y, K T- U S- 9 8 2- 5 9 6 8.
Ut o mil u ma b treat me nt i n P hase 2 will f oll o w t he d osi n g re gi me n fr o m P hase 1 a gree d u p o n b y t he st u d y s p o ns ors a n d t he S R T.
A b bre viati o ns: I P, i n vesti gati o nal pr o d uct.
Scree ni n g
E nr oll me nt/ Le u ka p heresis
C o n diti o ni n g C he m ot hera p y 2
I P A d mi nistrati o n Peri o d 
I P Treat me nt Peri o d 
A xica bta ge ne Cil ole ucel 
Treat me nt 3
( Da y 0)
S u bjects will recei ve 
a xica bta ge ne cil ole ucel i n 
a healt hcare facilit y 
f oll o we d b y a mi ni m u m 
7- da y o bser vati o n peri o d. 
I P Treat me nt Peri o d 
Ut o mil u ma b Treat me nt 4
( Da y 1 or Da y 2 1, Q 4 Wee ks)
P hase 1: Ut o mil u ma b will be 
a d mi nistere d i n u p t o 6 of 1 0 
c o h orts t o i de ntif y best d ose 
f or P hase 2
P hase 2: Ut o mil u ma b will be 
a d mi nistere d acc or di n g t o 
res ults of P hase 1 
P ost-treat me nt Assess me nt 
L o n g-ter m F oll o w- u p Peri o d 5
Bri d gi n g T hera p y (if a p plica ble) 1
 
 
 
 
 
 
 


A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 8
0 7 J u ne 2 0 2 2
T A B L E O F C O N T E N T S
P R O T O C O L S Y N O P SI S................................................................................................................................................ 2
T A B L E O F C O N T E N T S................................................................................................................................................ 8
LI S T O F T A B L E S........................................................................................................................................................ 1 0
LI S T O F FI G U R E S ...................................................................................................................................................... 1 0
LI S T O F A B B R E VI A TI O N S....................................................................................................................................... 1 1
1.   
O BJ E C TI V E S....................................................................................................................................................... 1 4
1. 1.  
Pri mar y O bjecti ve.................................................................................................................................... 1 4
1. 2.  
Sec o n dar y O bjecti ve(s) ........................................................................................................................... 1 4
2.   
DI S E A S E B A C K G R O U N D A N D R A TI O N A L E............................................................................................... 1 5
2. 1.   
Disease Bac k gr o u n d................................................................................................................................ 1 5
2. 2.   
A xica bta ge ne Cil ole ucel.......................................................................................................................... 1 5
2. 3.  
Pri or A nti- C D 1 9 C A R T-cell St u d y Desi g n............................................................................................ 1 6
2. 4.   
Ut o mil u ma b............................................................................................................................................. 1 6
2. 4. 1.  
S u m mar y of Cli nical St u dies f or Ut o mil u ma b...................................................................... 1 7
2. 5.  
Rati o nale f or C o m bi nati o n T hera p y........................................................................................................ 1 8
3.  
S T U D Y D E SI G N................................................................................................................................................. 2 2
3. 1.   
Ge neral St u d y Desi g n.............................................................................................................................. 2 2
3. 2.   
D osi n g Rati o nale..................................................................................................................................... 2 4
3. 2. 1.  
Rati o nale f or C o n diti o ni n g C he m ot hera p y ........................................................................... 2 4
3. 2. 2.  
Rati o nale f or A xica bta ge ne Cil ole ucel D ose ........................................................................ 2 5
3. 2. 3.  
Rati o nale f or Ut o mil u ma b D ose............................................................................................ 2 5
3. 3.  
Partici pati n g Sites.................................................................................................................................... 2 6
3. 4.   
N u m ber of S u bjects................................................................................................................................. 2 6
3. 5.  
Re place me nt of S u bjects ......................................................................................................................... 2 7
3. 6.  
St u d y D urati o n......................................................................................................................................... 2 7
3. 6. 1.  
St u d y D urati o n f or I n di vi d ual S u bjects................................................................................. 2 7
3. 6. 2.  
C o m pleti o n of St u d y.............................................................................................................. 2 7
3. 6. 3.  
L o n g-ter m F oll o w- u p ............................................................................................................ 2 7
4.  
S U BJ E C T I D E N TI FI C A TI O N A S SI G N M E N T ................................................................................................. 2 8
5.  
S U BJ E C T E LI GI BI LI T Y..................................................................................................................................... 2 9
5. 1.  
I ncl usi o n Criteria..................................................................................................................................... 2 9
5. 2.  
E xcl usi o n Criteria.................................................................................................................................... 3 1
6.  
P R O T O C O L T R E A T M E N T................................................................................................................................ 3 4
6. 1.  
St u d y Treat me nt ...................................................................................................................................... 3 4
6. 1. 1.  
Le u ka p heresis........................................................................................................................ 3 4
6. 1. 2.  
Bri d gi n g T hera p y................................................................................................................... 3 4
6. 1. 3.  
C o n diti o ni n g C he m ot hera p y ................................................................................................. 3 5
6. 1. 4.   
A xica bta ge ne Cil ole ucel........................................................................................................ 3 5
6. 1. 5.   
Ut o mil u ma b........................................................................................................................... 3 6
6. 1. 6.  
C o nc o mita nt T hera p y............................................................................................................ 3 7
6. 1. 7.  
E xcl u de d Me dicati o ns........................................................................................................... 3 8
6. 1. 8.  
S u bse q ue nt T hera p y .............................................................................................................. 3 9
6. 1. 9.   
A xica bta ge ne Cil ole ucel T o xicit y Ma na ge me nt................................................................... 3 9
C CI


A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 9
0 7 J u ne 2 0 2 2
6. 1. 1 0.   
Ut o mil u ma b T o xicit y Ma na ge me nt ...................................................................................... 3 9
7.  
S T U D Y P R O C E D U R E S...................................................................................................................................... 4 0
7. 1.  
I nf or me d C o nse nt.................................................................................................................................... 4 0
7. 2.  
Scree ni n g................................................................................................................................................. 4 0
7. 2. 1.  
Rescree ni n g ........................................................................................................................... 4 1
7. 3.   
De m o gra p hic Data................................................................................................................................... 4 1
7. 4.   
Me dical a n d Treat me nt Hist or y............................................................................................................... 4 1
7. 5.  
P h ysical E xa m, Vital Si g ns, a n d Perf or ma nce Stat us ............................................................................. 4 1
7. 5. 1.  
Car diac F u ncti o n ................................................................................................................... 4 2
7. 6.   
Ne ur ol o gical E xa mi nati o n....................................................................................................................... 4 2
7. 7.   
Disease Assess me nt................................................................................................................................. 4 2
7. 7. 1.  
I ma gi n g.................................................................................................................................. 4 3
7. 7. 2.   
Deter mi nati o n of B o ne Marr o w I n v ol ve me nt....................................................................... 4 3
7. 8.  
Cell C ollecti o n a n d A xica bta ge ne Cil ole ucel St u d y Treat me nt Sc he d ule a n d A d mi nistrati o n.............. 4 4
7. 8. 1.  
Le u ka p heresis........................................................................................................................ 4 4
7. 8. 2.  
C o n diti o ni n g C he m ot hera p y Peri o d...................................................................................... 4 4
7. 8. 3.  
I n vesti gati o nal Pr o d uct Treat me nt Peri o d............................................................................. 4 7
7. 9.  
La b orat or y ............................................................................................................................................... 5 1
7. 9. 1.  
L ocal La b A nal ysis................................................................................................................ 5 1
7. 9. 2.  
Ce ntral La b orat or y A nal yses................................................................................................. 5 2
7. 1 0.  
P ost-treat me nt Assess me nt Peri o d .......................................................................................................... 5 6
7. 1 1.  
L o n g-ter m F oll o w- u p Peri o d................................................................................................................... 5 6
8.  
S U BJ E C T WI T H D R A W A L................................................................................................................................ 6 6
8. 1.  
Reas o ns f or Re m o val fr o m Treat me nt .................................................................................................... 6 6
8. 2.  
Reas o ns f or Re m o val fr o m St u d y............................................................................................................ 6 6
8. 3.  
I nstr ucti o ns f or F oll o w- u p a n d Data t o Be C ollecte d f or S u bjects Wit h dra w n Fr o m 
Treat me nt/ St u d y...................................................................................................................................... 6 7
9.  
S A F E T Y R E P O R TI N G........................................................................................................................................ 6 8
9. 1.   
A d verse E ve nts........................................................................................................................................ 6 8
9. 2.  
Re p orti n g of A Es..................................................................................................................................... 6 8
9. 2. 1.  
Re p orti n g A b n or mal La b orat or y Fi n di n gs............................................................................ 7 0
9. 2. 2.   
A b n or mal Li ver F u ncti o n Tests ( H y’s La w): Dr u g-i n d uce d Li ver I nj ur y............................ 7 0
9. 2. 3.   
A Es of S pecial I nterest (I m me diatel y Re p orta ble t o t he S p o ns or)........................................ 7 0
9. 3.   
Defi niti o n of Seri o us A d verse E ve nts..................................................................................................... 7 1
9. 4.  
Re p orti n g of S A Es................................................................................................................................... 7 1
9. 5.  
Re p orti n g Deat hs..................................................................................................................................... 7 3
9. 6.   
Dia g n osis vers us Si g ns a n d S y m pt o ms................................................................................................... 7 3
9. 7.  
Pre g na nc y a n d Lactati o n ......................................................................................................................... 7 3
9. 8.   
H os pitalizati o n a n d Pr ol o n ge d H os pitalizati o n....................................................................................... 7 5
9. 9.   
A b n or mal Vital Si g n Val ues ................................................................................................................... 7 5
9. 1 0.  
Safet y Re vie w Tea m a n d D ose-li miti n g T o xicit y................................................................................... 7 5
1 0. 
S T A TI S TI C A L C O N SI D E R A TI O N S................................................................................................................. 7 8
1 0. 1.   
H y p ot hesis............................................................................................................................................... 7 8
1 0. 2.  
St u d y E n d p oi nts....................................................................................................................................... 7 8
1 0. 2. 1.  
Pri mar y E n d p oi nts................................................................................................................. 7 8
1 0. 2. 2.  
Sec o n dar y E n d p oi nts............................................................................................................. 7 8
1 0. 3.  
Sa m ple Size C o nsi derati o ns.................................................................................................................... 7 9
1 0. 4.  I nteri m A nal ysis a n d Earl y St o p pi n g G ui deli nes.................................................................................... 8 0
1 0. 4. 1.  
Safet y I nteri m A nal ysis......................................................................................................... 8 0
C CI


A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 1 0
0 7 J u ne 2 0 2 2
1 0. 5.   
A nal ysis S u bsets...................................................................................................................................... 8 0
1 0. 6.  
Pla n ne d Met h o d of A nal ysis ................................................................................................................... 8 1
1 0. 6. 1.  
C o m plete Res p o nse Rate....................................................................................................... 8 1
1 0. 6. 2.   
O bjecti ve Res p o nse Rate....................................................................................................... 8 1
1 0. 6. 3.   
D urati o n of Res p o nse............................................................................................................ 8 1
1 0. 6. 4.  
Pr o gressi o n-free S ur vi val...................................................................................................... 8 1
1 0. 6. 5.   
O verall S ur vi val..................................................................................................................... 8 1
1 0. 6. 6.  
Safet y..................................................................................................................................... 8 2
1 0. 6. 7.  
L o n g-ter m Data A nal ysis ...................................................................................................... 8 2
1 1. 
R E G U L A T O R Y O B LI G A TI O N S....................................................................................................................... 8 3
1 1. 1.  I n de pe n de nt Re vie w B oar d/I n de pe n de nt Et hics C o m mittee................................................................... 8 3
1 1. 2.  
S u bject C o nfi de ntialit y............................................................................................................................ 8 3
1 1. 3.  I n vesti gat or Si g nat or y O bli gati o ns.......................................................................................................... 8 4
1 2. 
P R O T O C O L A M E N D M E N T S A N D T E R MI N A TI O N..................................................................................... 8 5
1 3. 
S T U D Y D O C U M E N T A TI O N A N D A R C HI VI N G............................................................................................ 8 6
1 4. 
S T U D Y M O NI T O RI N G A N D D A T A C O L L E C TI O N...................................................................................... 8 7
1 5. 
P U B LI C A TI O N ................................................................................................................................................... 8 8
1 6. 
R E F E R E N C E S..................................................................................................................................................... 8 9
A D DI N E N. R E F LI S T.......................................................................................................................................... 8 9
1 7. 
A P P E N DI C E S...................................................................................................................................................... 9 2
A p pe n di x 1.  
S p o ns or a n d I n vesti gat or Si g nat ure Pa ge................................................................................ 9 3
A p pe n di x 2.  
L u ga n o Classificati o n { C hes o n 2 0 1 4 }.................................................................................... 9 4
A p pe n di x 3.  
C hil d beari n g P ote ntial a n d Birt h C o ntr ol................................................................................ 9 8
LI S T O F T A B L E S
Ta ble 1.  
Cli nical E x perie nce wit h Ut o mil u ma b.................................................................................... 1 8
Ta ble 2.   
Ut o mil u ma b Re gi me ns............................................................................................................ 2 3
Ta ble 3.  
Bri d gi n g T hera p y Re gi me ns.................................................................................................... 3 4
Ta ble 4.  
L ocal La b orat or y Para meters................................................................................................... 5 2
Ta ble 5.  
Sc he d ule of Assess me nts C o h ort 1, 2, 3, 4, 5 a n d P hase 2 (as a p plica ble) 
Ut o mil u ma b A d mi nistrati o n Starti n g o n Da y 1 ...................................................................... 5 8
Ta ble 6.  
Sc he d ule of Assess me nts C o h ort 1 A, 2 A, 3 A, 4 A, 5 A a n d P hase 2 (as a p plica ble) 
Ut o mil u ma b A d mi nistrati o n Starti n g o n Da y 2 1 .................................................................... 6 1
Ta ble 7.  
L o n g-ter m F oll o w- u p Assess me nts......................................................................................... 6 4
Ta ble 8.  
E xact C o nfi de nce I nter vals ( 9 5 % a n d 8 0 %) C orres p o n di n g t o O bser ve d C R Rate 
F oll o wi n g Treat me nt of 2 7 S u bjects wit h A xica bta ge ne Cil ole ucel a n d Ut o mil u ma b........... 8 0
LI S T O F FI G U R E S
Fi g ure 1.  
St u d y Sc he ma ( P hase 1 a n d P hase 2)........................................................................................ 7
Fi g ure 2.  
E x pa nsi o n of C A R T Cells was Ass ociate d wit h O bjecti ve Res p o nse a n d 
Ne ur ol o gic E ve nts ................................................................................................................... 1 9
Fi g ure 3.  
P ost-i nf usi o n Ki netics of C A R T Cells i n t he Bl o o d............................................................... 2 0
Fi g ure 4.   
U pre g ulati o n of 4- 1 B B o n C A R T Cells After I nf usi o n ......................................................... 2 1
Fi g ure 5.  
St u d y Desi g n O ver vie w........................................................................................................... 2 3


A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 1 1
0 7 J u ne 2 0 2 2
LI S T O F A B B R E VI A TI O N S
A B C
Acti vate d B cell
A D A
A nti- dr u g a nti b o d y
A E
A d verse e ve nt
AI C D
Acti vati o n i n d uce d cell deat h
A L T
Ala ni ne a mi n otra nsferase
A N C
A bs ol ute ne utr o p hil c o u nt
A S C T
A ut ol o g o us ste m cell tra ns pla nt
A S T
As partate a mi n otra nsferase 
A U C
Area u n der t he c ur ve
B P
Bl o o d press ure
C A R
C hi meric a nti ge n rece pt or 
C B C
C o m plete bl o o d c o u nt
C eff
Efficaci o us c o nce ntrati o n
CI
C o nfi de nce I nter val
C L L
C hr o nic l y m p h oc ytic le u ke mia
C M V
C yt o me gal o vir us
C N S
Ce ntral ner v o us s yste m
C R
C o m plete res p o nse
C R F
Case re p ort f or m
C R O
C o ntract Researc h Or ga nizati o n
C R P
C-reacti ve pr otei n
C R S
C yt o ki ne release s y n dr o me
C S F
Cere br os pi nal fl ui d
C T
C o m p ute d t o m o gra p h 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
DI LI
Dr u g-i n d uce d li ver i nj ur y
D L B C L
Diff use lar ge B-cell l y m p h o ma
D L T
D ose-li miti n g t o xicit y
D O R
D urati o n of res p o nse
D O R R
D urati o n retreat me nt res p o nse
E B V
E pstei n- Barr vir us
E C G
Electr ocar di o gra m
E C H O
Ec h ocar di o gra m
E C O G
Easter n C o o perati ve O nc ol o g y Gr o u p
F A S
F ull a nal ysis set
F D A
F o o d a n d Dr u g A d mi nistrati o n
F L
F ollic ular l y m p h o ma
G C B
Ger mi nal ce nter B cell


A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 1 2
0 7 J u ne 2 0 2 2
G C P
G o o d Cli nical Practice
G M- C S F
Gra n ul oc yte macr o p ha ge-c ol o n y sti m ulati n g fact or
H E E N T
Hea d, e ye, ear, n ose, a n d t hr oat
H G B C L
Hi g h- gra de B-cell l y m p h o ma
HI V
H u ma n i m m u n o deficie nc y vir us
H L H
He m o p ha g oc ytic l y m p h o histi oc yt osis
I B
I n vesti gat or’s Br oc h ure 
I C F
I nf or me d c o nse nt f or m
I C H
I nter nati o nal C o u ncil f or Har m o nisati o n of Tec h nical 
Re q uire me nts f or P har mace uticals f or H u ma n Use
I C U
I nte nsi ve care u nit
I D
I de ntificati o n
I F
I nterfer o n
I H C
I m m u n o hist oc he mistr y
I L
I nterle u ki n
I P
I n vesti gati o nal pr o d uct
I P M
I n vesti gati o nal Pr o d uct Ma n ual
I R B/I E C
I nstit uti o nal Re vie w B oar d/I n de pe n de nt Et hics 
C o m mittee
I V
I ntra ve n o us
L T F U
L o n g-ter m f oll o w- u p
L V E F
Left ve ntric ular ejecti o n fracti o n
m A b
M o n ocl o nal a nti b o d y
M A P
Mit o ge n acti vate d pr otei n 
M C P
M o n oc yte c he m oattracta nt pr otei n
MI P
Macr o p ha ge i nfla m mat or y pr otei n
mI T T
M o difie d i nte nt-t o-treat
M RI
Ma g netic res o na nce i ma gi n g
Na b
Ne utralizi n g a nti b o d y
N CI
Nati o nal Ca ncer I nstit ute
N E
Ne ur ol o gic e ve nt
N F- k B
N uclear fact or ka p pa-li g ht-c hai n-e n ha ncer of acti vate d 
B cells
N H L
N o n- H o d g ki n l y m p h o ma
N K
Nat ural killer
O R R
O bjecti ve res p o nse rate
O S
O verall s ur vi val
P B M C
Peri p heral bl o o d m o n o n uclear cell
P C R
P ol y merase c hai n reacti o n
P D
Pr o gressi ve disease
P E T- C T
P ositr o n e missi o n t o m o gra p h y –c o m p ute d t o m o gra p h y


A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 1 3
0 7 J u ne 2 0 2 2
P F S
Pr o gressi o n-free s ur vi val
P K
P har mac o ki netics
P R
Partial res p o nse
q P C R
Q ua ntitati ve p ol y merase c hai n reacti o n
R C R 
Re plicati o n-c o m pete nt retr o vir us
S A E
Seri o us a d verse e ve nt
sc F v
Si n gle-c hai n varia ble fra g me nt
S C T
Ste m cell tra ns pla nt
S D
Sta ble disease
S O A
Sc he d ule of assess me nt
S O C
Sta n dar d of care
S R T
Safet y re vie w tea m
T E A E
Treat me nt-e mer ge nt a d verse e ve nt
T F L
Tra nsf or me d f ollic ular l y m p h o ma
T GI
T u m or gr o wt h i n hi biti o n
T L S
T u m or l ysis s y n dr o me
T N F
T u m or necr osis fact or 
U L N
U p per li mit of n or mal
W B C
W hite bl o o d cell




A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 1 5
0 7 J u ne 2 0 2 2
2.   
DI S E A S E B A C K G R O U N D A N D R A TI O N A L E
2. 1.   
Dise ase B ac k gr o u n d
N o n- H o d g ki n l y m p h o ma ( N H L) is a heter o ge ne o us gr o u p of ca ncers ori gi nati n g i n B 
l y m p h oc ytes, T l y m p h oc ytes, or nat ural killer ( N K) cells.  I n t he U nite d States, B-cell 
l y m p h o mas re prese nt 8 0 % t o 8 5 % of cases re p orte d. I n 2 0 1 8, it is esti mate d t hat t here will be 
a p pr o xi matel y 7 4, 6 8 0 ne w cases of N H L a n d o ver 1 9, 0 0 0 deat hs relate d t o t he disease.  N H L is 
t he m ost pre vale nt he mat ol o gical mali g na nc y a n d is t he se ve nt h m ost c o m m o n t y pe of ca ncer 
a m o n g me n a n d w o me n a n d acc o u nts f or 4. 3 % of all ne w ca ncer cases a n d 3 % of deat hs relate d 
t o ca ncer {N o o ne 2 0 1 7 }.
Diff use lar ge B-cell l y m p h o ma ( D L B C L) is t he m ost c o m m o n s u bt y pe of N H L, acc o u nti n g f or 
a p pr o xi matel y 3 0 % of N H L cases.  T here are a p pr o xi matel y 2 2, 0 0 0 ne w dia g n oses of D L B C L i n 
t he U nite d States eac h year.  I n t he past 2 deca des, pr o gress has bee n ma de i n u n dersta n di n g t he 
bi ol o gical heter o ge neit y of D L B C L a n d i n i m pr o vi n g s ur vi val wit h c o m bi nati o ns of 
c he m ot hera p y a n d i m m u n ot hera p y.  T he a d diti o n of rit u xi ma b i nt o c o m bi nati o n t hera pies f or 
D L B C L has greatl y i m pr o ve d patie nt o utc o mes.  H o we ver, patie nts wit h c he m ot hera p y-
refract or y D L B C L still ha ve a partic ularl y dire pr o g n osis {Fl o wers 2 0 1 0 }.
S C H O L A R- 1, a lar ge m ultice nter, patie nt-le vel retr os pecti ve st u d y, e xa mi ne d o utc o mes of 
refract or y D L B C L usi n g p o ole d data fr o m 2 ra n d o mize d P hase 3 cli nical trials ( L y m p h o ma 
Aca de mic Researc h Or ga nizati o n- C O R A L a n d Ca na dia n Ca ncer Trials Gr o u p L Y. 1 2) a n d 
2 o bser vati o nal c o h orts ( M D A n ders o n Ca ncer Ce nter a n d U ni versit y of I o wa/ Ma y o Cli nic 
L y m p h o ma S pecialize d Pr o gra m of Researc h E xcelle nce).  F or t he st u d y, refract or y D L B C L was 
defi ne d as pr o gressi ve disease or sta ble disease as t he best res p o nse at a n y p oi nt d uri n g 
c he m ot hera p y ( > 4 c ycles of first-li ne or 2 c ycles of later-li ne t hera p y) or rela pse d ≤ 1 2 m o nt hs 
of a ut ol o g o us ste m cell tra ns pla ntati o n ( A S C T).  T he st u d y hi g hli g hte d t he p o or pr o g n osis of 
patie nts affecte d wit h refract or y D L B C L, fi n di n g a n o bjecti ve res p o nse rate ( O R R) t o t he ne xt 
li ne of t hera p y of 2 6 % (c o m plete res p o nse [ C R], 7 %) a n d a me dia n o verall s ur vi val ( O S) of o nl y 
6. 3 m o nt hs { Cr u m p 2 0 1 7 }.
2. 2.   
A xic a bt a ge ne Cil ole ucel
A xica bta ge ne cil ole ucel is a C D 1 9- directe d ge neticall y m o difie d a ut ol o g o us T-cell 
i m m u n ot hera p y.  F or t his t hera p y, a patie nt’s T cells are ge neticall y m o difie d t o pr o d uce a 
c hi meric a nti ge n rece pt or ( C A R) pr otei n, all o wi n g t he T cells t o i de ntif y a n d eli mi nate C D 1 9-
e x pressi n g n or mal a n d mali g na nt cells. C D 1 9 is e x presse d b y m ost B-cell mali g na ncies 
{ Le o nar d 2 0 0 1 , Olej nicza k 2 0 0 6 , R o dri g uez 1 9 9 4 , Uc k u n 1 9 8 8 } as well as n or ma l B 
l y m p h oc ytes i n peri p heral bl o o d a n d s plee n, b ut n ot b y gra n ul oc ytes, m o n oc ytes, platelets, 
er yt hr oc ytes, a n d T l y m p h oc ytes { Uc k u n 1 9 8 8 }. Briefl y, t he a nti- C D 1 9 C A R tra ns ge ne 
c o m prises t he f oll o wi n g ke y d o mai ns: 1) a n e xtracell ular a nti- h u ma n C D 1 9 si n gle-c hai n varia ble 
re gi o n fra g me nt (sc F v) deri ve d fr o m t he m o n ocl o nal a nti b o d y F M C 6 3; 2) t he tra ns me m bra ne c o-
sti m ulat or y d o mai n C D 2 8; a n d 3) t he c yt o plas mic p orti o n of h u ma n C D 3ζ t hat i ncl u des t he 
si g nali n g d o mai ns {Nic h ols o n 1 9 9 7 }. F oll o wi n g C A R e n ga ge me nt wit h C D 1 9 +  tar get cells, t he 


A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 1 6
0 7 J u ne 2 0 2 2
c o-sti m ulat or y d o mai ns acti vate a d o w nstrea m si g nali n g casca de t hat lea ds t o T-cell acti vati o n, 
pr oliferati o n, a n d ac q uisiti o n of effect or f u ncti o n.
O n 1 8 Oct o ber 2 0 1 7, t he F o o d a n d Dr u g A d mi nistrati o n ( F D A) a p pr o ve d a xica bta ge ne 
cil ole ucel f or t he treat me nt of a d ult patie nts wit h rela pse d or refract or y lar ge B-cell l y m p h o ma 
after 2 or m ore li nes of s yste mic t hera p y, i ncl u di n g D L B C L n ot ot her wise s pecifie d, pri mar y 
me diasti nal lar ge B-cell l y m p h o ma, hi g h- gra de B-cell l y m p h o ma ( H G B C L), a n d D L B C L arisi n g 
fr o m f ollic ular l y m p h o ma ( F L) {Y E S C A R T A 2 0 1 7 }.
F D A a p pr o val was base d o n Z U M A- 1, a si n gle-ar m m ultice nter trial of 1 0 8 a d ult s u bjects wit h 
rela pse d or refract or y a g gressi ve lar ge B-cell N H L.  Of t he 1 0 1 s u bjects e val uate d f or efficac y, 
t he O R R was 7 2 %, wit h a C R rate of 5 1 %.  T he me dia n d urati o n of res p o nse ( D O R) was l o n ger 
i n s u bjects wit h a best o verall res p o nse of C R c o m pare d wit h t h ose w h o ac hie ve d a best o verall 
res p o nse of partial res p o nse ( P R) ( n ot esti ma ble vs 2. 1 m o nt hs, res pecti vel y) {Y E S C A R T A 
2 0 1 7 } . T he m ost c o m m o n Gra de 3 or hi g her a d verse e ve nts ( A Es; i nci de nce of ≥ 1 0 %) were 
fe ver, c yt o ki ne release s y n dr o me ( C R S), e nce p hal o pat h y, i nfecti o ns ( pat h o ge n u ns pecifie d), 
h y p ote nsi o n, a n d h y p o xia.  Seri o us a d verse reacti o ns occ urre d i n 5 2 % of s u bjects a n d i ncl u de d, 
b ut were n ot li mite d t o, e nce p hal o pat h y, fe ver, fe brile ne utr o pe nia, a n d seri o us i nfecti o ns.  Fatal 
cases of C R S a n d ne ur ol o gic t o xicit y occ urre d. F D A a p pr o ve d a xica bta ge ne cil ole ucel wit h a 
Ris k E val uati o n a n d Miti gati o n Strate g y { Y E S C A R T A 2 0 1 7 }.
T he d ose of a xica bta ge ne cil ole ucel is a si n gle i ntra ve n o us (I V) i nf usi o n wit h a tar get of 
2 x 1 0 6 C A R- p ositi ve via ble T cells per k g b o d y wei g ht ( ma xi m u m 2 x 1 08 cells), prece de d b y 
c ycl o p h os p ha mi de a n d fl u dara bi ne l y m p h o de pleti n g c he m ot hera p y { Y E S C A R T A 2 0 1 7 }.  
A d diti o nal details re gar di n g t he mec ha nis m of acti o n a n d cli nical res ults of a xica bta ge ne 
cil ole ucel ca n be f o u n d i n t he m ost c urre nt versi o n of t he a xica bta ge ne cil ole ucel I n vesti gat or’s 
Br oc h ure (I B).
2. 3.  
Pri or A nti- C D 1 9 C A R T-cell St u d y Desi g n
T he desi g n a n d rati o nale of t his st u d y is i n part deri ve d fr o m pri or e x perie nce wit h a xica bta ge ne 
cil ole ucel o n ot her Kite P har ma, I nc., ( hereafter referre d t o as Kite or Kite P har ma) s p o ns ore d 
st u dies, i ncl u di n g Z U M A- 1, a n d o n a si n gle-ce nter st u d y c o n d ucte d at t he Nati o nal Ca ncer 
I nstit ute ( N CI). See t he m ost c urre nt versi o n of t he a xica bta ge ne cil ole ucel I B f or details.
2. 4.   
Ut o mil u m a b
T he i n d uci ble c o-sti m ulat or y rece pt or 4- 1 B B ( C D 1 3 7 or t u m or necr osis fact or [ T N F] 
s u perfa mil y 9) is a critical me diat or of i m m u ne res p o nses, a n d it is e x presse d o n s u bsets of 
acti vate d i m m u ne cells, i ncl u di n g T cells { Wa n g 2 0 0 9 }.  Its l i ga n d, 4- 1 B B L, is e x presse d o n 
acti vate d a nti ge n- prese nti n g cells, a n d t hr o u g h 4- 1 B B e n ga ge me nt, it tri g gers d o w nstrea m 
i ntracell ular si g nali n g t hr o u g h t he n uclear fact or ka p pa-li g ht-c hai n-e n ha ncer of acti vate d B cells 
( N F- k B) a n d mit o ge n acti vate d pr otei n ( M A P) ki nase pat h wa ys t hat pr o m ote cell ular 
pr oliferati o n, s ur vi val, a n d c yt o ki ne pr o d ucti o n { Cr oft 2 0 0 9 }.  F or t hese rea s o ns, 4- 1 B B is a n 
attracti ve i m m u n ot hera p y tar get.


A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 1 7
0 7 J u ne 2 0 2 2
Ut o mil u ma b (c o m pa n y c o de: P F- 0 5 0 8 2 5 6 6) is a hi g hl y selecti ve, f ull y h u ma n 4- 1 B B I g G 2 
m o n ocl o nal a nti b o d y ( m A b) t hat bi n ds as a n a g o nist t o t he h u ma n 4- 1 B B wit h na n o m olar 
affi nit y.  I n precli nical st u dies, ut o mil u ma b acti vate d t he N F- k B pat h wa y a n d i n d uce d 
d o w nstrea m c yt o ki ne pr o d ucti o n i n e n gi neere d cell li nes a n d pri mar y l y m p h oc ytes.  I n a d diti o n, 
ut o mil u ma b pr o m ote d h u ma n le u k oc yte pr oliferati o n i n a m o del of h u ma n peri p heral bl o o d 
m o n o n uclear cell ( P B M C) e n graft me nt a n d i n hi bite d t u m or gr o wt h i n xe n o graft t u m or m o dels i n 
w hic h h u ma n P B M C ha d bee n c o-e n grafte d.  Ut o mil u ma b ma y, t heref ore, e n ha nce t he i m m u ne 
res p o nse a gai nst t u m ors {Fis her 2 0 1 2 } (refer t o P F 0 5 0 8 2 5 6 6 I B).
2. 4. 1.  
S u m m ar y of Cli nic al St u dies f or Ut o mil u m a b 
Cli nical e x perie nce t o date wit h ut o mil u ma b is s u m marize d i n Ta ble 1 .  As of J ul y 2 0 1 7, 
P F- 0 5 0 8 2 5 6 6 has bee n a d mi nistere d t o a t otal of 3 9 0 s u bjects. St u d y B 1 6 4 1 0 0 1 is a P hase 1 
o pe n-la bel, m ultice nter, d ose escalati o n st u d y of P F- 0 5 0 8 2 5 6 6 as a si n gle a ge nt ( P orti o n A) i n 
s u bjects wit h rela pse d s oli d t u m ors or rela pse d or refract or y B-cell l y m p h o ma or i n c o m bi nati o n 
wit h rit u xi ma b ( P orti o n B) i n s u bjects wit h rela pse d or refract or y C D 2 0 p ositi ve N H L (refer t o 
P F- 0 5 0 8 2 5 6 6 I B) { G o pal 2 0 1 5 , Se gal 2 0 1 8 }.  P F- 5 0 8 2 5 6 6 d oses ra n ge d fr o m 0. 0 0 6 m g/ k g t o 
1 0 m g/ k g a d mi nistere d i ntra ve n o usl y (I V) o nce e ver y 2 8 da ys.  T he re gi me n was well t olerate d 
wit h t he m ost fre q ue ntl y o bser ve d treat me nt-relate d, treat me nt-e mer ge nt a d verse e ve nt ( T E A E) 
i n P orti o n A bei n g fati g ue ( 1 4. 0 %).  Treat me nt-relate d T E A Es were m ostl y Gra de 1 or Gra de 2 
wit h o nl y 4 Gra de 3/ 4 A Es re p orte d (c olitis, diarr hea, fati g ue, h y per bilir u bi ne mia, a n d 
h y p o natre mia).  T he m ost fre q ue ntl y o bser ve d treat me nt-relate d T E A Es i n P orti o n B wit h 
6 6 s u bjects were i nf usi o n-relate d reacti o n ( 2 1. 2 %) a n d fati g ue ( 1 9. 7 %).  All of t he i nf usi o n 
reacti o ns were ass ociate d wit h rit u xi ma b i nf usi o n. P F- 0 5 0 8 2 5 6 6-relate d T E A Es were eit her 
Gra de 1 or Gra de 2 i n se verit y.  Gra de 3/ 4 A Es were re p orte d i n 6 s u bjects i n c o m bi nati o n wit h 
rit u xi ma b o nl y: i nf usi o n-relate d reacti o n a n d ne utr o pe nia ( 2 s u bjects eac h) a n d decrease d 
l y m p h oc yte a n d ne utr o p hil c o u nts ( 1 s u bject eac h).  T here were n o d ose-li miti n g t o xicities 
(D L Ts ). P F- 0 5 0 8 2 5 6 6 is n o w c o nsi dere d t o be safe u p t o d oses of 1 0 m g/ k g, e q ui vale nt t o 
a p pr o xi matel y 7 0 0 m g i n a 7 0- k g s u bject, as a m o n ot hera p y or i n c o m bi nati o n wit h rit u xi ma b 
( 3 7 5 m g/ m2).  T h us, t he pr o p ose d starti n g d ose of 1 0 m g t otal d ose f or t his pr ot oc ol re n ders a 
mar gi n of safet y of at least 7 0-f ol d relati ve t o hist orical safet y data.  
St u d y B 1 6 4 1 0 0 3 is a P hase 1 o pe n-la bel, m ultice nter, d ose escalati o n st u d y of P F- 0 5 0 8 2 5 6 6 
a d mi nistere d i n c o m bi nati o n wit h pe m br oliz u ma b (a n a nti P D- 1 m A b) i n a d ult s u bjects wit h 
a d va nce d s oli d mali g na ncies.  E nr oll me nt of s u bjects i nt o t he d ose escalati o n p hase of t he st u d y 
is c o m plete, wit h 2 3 s u bjects e nr olle d at P F- 0 5 0 8 2 5 6 6 d oses ra n gi n g fr o m 0. 4 5 m g/ k g t o 
5. 0 m g/ k g a d mi nistere d I V c o nc urre ntl y wit h pe m br oliz u ma b at 2 m g/ k g o nce e ver y 2 1 da ys. 
Treat me nt-relate d T E A Es were m ostl y Gra de 1 or Gra de 2.  T here were o nl y 2 treat me nt-relate d 
( P F- 0 5 0 8 2 5 6 6 a n d pe m br oliz u ma b) Gra de 3 A Es re p orte d i n 2 s u bjects: a dre nal i ns ufficie nc y 
( n o e vi de nce of ass ociate d h y p o p h ysitis) a n d h y p o kale mia ( 1 s u bject eac h).  N o Gra de 4 or 
Gra de 5 treat me nt-relate d A Es were o bser ve d.  T his c o m bi nati o n was well t olerate d w he n t he 
ma xi m u m a d mi nistere d d ose of P F- 0 5 0 8 2 5 6 6 was 5 m g/ k g f or t his c o m bi nati o n, r o u g hl y 3 5 0 m g 
i n a 7 0- k g s u bject, a n d was dee me d well t olerate d wit h n o D L Ts o bser ve d i n t he st u d y.  Ot her 
c o m bi nati o n strate gies are als o u n der i n vesti gati o n as s h o w n i n Ta ble 1 .


A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 1 8
0 7 J u ne 2 0 2 2
T a ble 1.
Cli nic al E x perie nce wit h Ut o mil u m a b
S u bjects
Pr ot oc ol 
Title
Pl a n ne d 
Tre ate d 
St at us
B 1 6 4 1 0 0 1
A P hase 1 St u d y of P F 0 5 0 8 2 5 6 6 as a Si n gle A ge nt 
i n Patie nts wit h A d va nce d Ca ncer a n d i n 
C o m bi nati o n wit h Rit u xi ma b i n Patie nts wit h N o n-
H o d g ki n's L y m p h o ma ( N H L)
7 2- 1 6 1 
1 8 0 
O n g oi n g
B 1 6 4 1 0 0 3
A P hase 1 St u d y of t he 4- 1 B B A g o nist P F- 0 5 0 8 2 5 6 6 
i n C o m bi nati o n Wit h t he P D- 1 I n hi bit or M K- 3 4 7 5 i n 
Patie nts wit h A d va nce d S oli d T u m ors
4 5- 7 5 
2 3 
C o m plete d
B 1 6 4 1 0 0 4
A P hase 1 b St u d y of P F- 0 5 0 8 2 5 6 6 i n C o m bi nati o n 
Wit h M o ga m uliz u ma b ( K W- 0 7 6 1) i n Patie nts Wit h 
A d va nce d S oli d T u m ors
7 0 
2 4 
Ter mi nate d
B 9 9 9 1 0 0 4
A P hase 1 b/ 2 O pe n- La bel St u d y t o E val uate Safet y, 
Cli nical Acti vit y, P har mac o ki netics a n d 
P har mac o d y na mics of A vel u ma b ( M S B 0 0 1 0 7 1 8 C) 
i n C o m bi nati o n wit h Ot her Ca ncer I m m u n ot hera pies 
i n Patie nts wit h A d va nce d Mali g na ncies ( A vel u ma b 
I B versi o n 7, 3 1 Marc h 2 0 1 7)
3 1 7 
1 5 3 
O n g oi n g
B 0 6 0 1 0 0 2
A P hase 1 O pe n La bel, D ose Escalati o n St u d y of P F-
0 4 5 1 8 6 0 0 as a Si n gle A ge nt a n d i n C o m bi nati o n wit h 
P F- 0 5 0 8 2 5 6 6 i n Patie nts wit h Selecte d L ocall y 
A d va nce d or Metastatic Carci n o mas ( P F- 0 4 5 1 8 6 0 0 
I B Dece m ber 2 0 1 6)
1 9 0 
1 0 
O n g oi n g
A b bre viati o ns: I B, I n vesti gat or’s Br oc h ure.
2. 5.   
R ati o n ale f or C o m bi n ati o n T her a p y 
Pre vi o us cli nical trial e x perie nce wit h a xica bta ge ne cil ole ucel i n t he sa me patie nt p o p ulati o n 
( Z U M A- 1) de m o nstrate d a n O R R of 8 2 % a n d a C R rate of 5 8 % at t he u p date d a nal ysis after 
me dia n f oll o w- u p of 1 5. 4 m o nt hs { Neela p u 2 0 1 7 b }.  O n g oi n g re s p o nses were see n i n 4 2 % of 
s u bjects, of w hic h 4 0 % were i n C R. A m o n g t he 5 8 % of s u bjects w h o were n ot i n o n g oi n g 
res p o nse at t he 1 2- m o nt h f oll o w- u p, 1 8 % of s u bjects de m o nstrate d pri mar y resista nce t o t hera p y 
( di d n ot ac hie ve P R or C R), a n d 4 0 % of s u bjects ac hie ve d a n o bjecti ve res p o nse a n d later 
pr o gresse d or die d { Neela p u 2 0 1 7a }.  P ossi ble mec ha nis ms of resista nce t o a xica bta ge ne 
cil ole ucel are h y p ot hesize d t o be s u b o pti mal C A R T-cell e x pa nsi o n { Neela p u 2 0 1 7 b }, a n 
e xcl usi o nar y t u m or micr oe n vir o n me nt { R ossi 2 0 1 8 }, a n d C D 1 9 tar get a nti ge n l oss { Neela p u 
2 0 1 7a }.  
F urt her, it was als o o bser ve d i n Z U M A- 1 t hat 2 3/ 6 0 s u bjects wit h eit her a P R ( 1 1/ 3 5) or sta ble 
disease ( S D) ( 1 2/ 2 5) at t he first t u m or assess me nt ( 1 m o nt h after a xica bta ge ne cil ole ucel 
a d mi nistrati o n) s u bse q ue ntl y ac hie ve d C R u p t o 1 5 m o nt hs after i nf usi o n wit h o ut a d diti o nal 
t hera p y {Neela p u 2 0 1 7a }.  I n a d diti o n, pea k e x pa nsi o n a n d area u n der t he c ur ve 0- 2 8 d (A U C 0- 2 8 d) 
are ass ociate d wit h res p o nse { Neela p u 2 0 1 7 b } ( Fi g ure 2 ).  T heref ore, c o m bi nati o n strate gies t hat 
eit her i ncrease pr oliferati o n, e x pa nsi o n, a n d persiste nce of C A R T cells or pre ve nt acti vati o n-
i n d uce d cell deat h ( AI C D) of C A R T cells ma y i m pr o ve cli nical o utc o mes see n wit h a nti- C D 1 9 
C A R T-cell t hera p y.  


A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 1 9
0 7 J u ne 2 0 2 2
Fi g ure 2.
E x p a nsi o n of C A R T Cells w as Ass oci ate d wit h O bjecti ve Res p o nse 
a n d Ne ur ol o gic E ve nts
A b bre viati o ns: O R R, o bjecti ve res p o nse rate; N E, ne ur ol o gic e ve nt; C R S, c yt o ki ne release s y n dr o me; A U C, area u n der t he 
c ur ve; C A R, c hi meric a nti ge n rece pt or.
C A R A U C defi ne d as c u m ulati ve le vels of C A R + cells/ µ L of bl o o d o ver t he first 2 8 da ys after  a xica bta ge ne cil ole ucel 
a d mi nistrati o n.   
P val ues are calc ulate d b y Wilc o x o n ra n k s u m test. 
Strate gies t hat ma y e n ha nce t he e n graft me nt of C A R T cells are c urre ntl y bei n g e x pl ore d i n t he 
cli nical setti n g.  Z U M A- 6 is a P hase 1/ 2 st u d y e xa mi ni n g a xica bta ge ne cil ole ucel i n c o m bi nati o n 
wit h atez oliz u ma b (a n a nti P D- L 1 m A b) i n refract or y D L B C L ( N C T 0 2 9 2 6 8 3 3).  Pr o gra m me d 
cell deat h pr otei n 1 ( P D- 1) is a n acti vati o n/e x ha usti o n mar ker of c yt ol ytic T cells a n d is 
u pre g ulate d o n t he s urface of C A R T cells d uri n g i n vitr o e x p os ure t o C D 1 9-e x pressi n g tar get 
cells {B ot 2 0 1 5 }.  F urt her, i n s u bje cts treate d wit h a nti- C D 1 9 C A R T cells, P D- 1 is u pre g ulate d 
o n C A R T cells after i nf usi o n { Perez 2 0 1 5 }.  Base d o n t he k n o w n bi ol o g y of P D- 1, u pre g ulati o n 
c o ul d p ossi bl y lea d t o i n hi biti o n of cell di visi o n, c yt ol ytic acti vit y, a n d e x ha usti o n of i nf use d 
C A R T cells { Keir 2 0 0 8 }.  T here f ore, it was h y p ot hesize d t hat disr u pti o n of t he P D- 1/ P D- L 1 
T-cell re g ulat or y a xis c o ul d e n ha nce t he c yt ol ytic ca pacit y of t he i nf use d C A R T cells a n d 
a u g me nt a nti- C D 1 9 C A R T-cell e n graft me nt. 
T he P hase 1 p orti o n of Z U M A- 6 wit h a xica bta ge ne cil ole ucel i n c o m bi nati o n wit h atez oliz u ma b 
has bee n c o m plete d wit h a ma na gea ble safet y pr ofile. T here were n o Gra de 5 e ve nts, a n d t he 
m ost c o m m o n Gra de 3/ 4 T E A Es were a ne mia 7 ( 7 8 %), e nce p hal o pat h y 5 ( 5 6 %), ne utr o pe nia 4 
( 4 4 %), h y p o natre mia 3 ( 3 3 %), p yre xia 3 ( 3 3 %), a n d t hr o m b oc yt o pe nia 3 ( 3 3 %).  O verall, t here 
was n o e vi de nce of w orse ni n g or rec urre nt A Es c o nsiste nt wit h C R S, ne ur ol o gic e ve nts ( N E), or 
ot her C A R T-cell-relate d t o xicities f oll o wi n g atez oliz u ma b a d mi nistrati o n { L oc ke 2 0 1 7 }.
After  atez oliz u ma b i nf usi o n, ki netics of C A R T cells s u g gest t hat atez oliz u ma b ma y i ncrease 
o verall C A R T-cell A U C wit h o ut i ncreasi n g t o xicit y or c ha n gi n g t he safet y pr ofile ( Fi g ure 3 ) 
{ L oc ke 2 0 1 7 }.  Ate z oliz u mab a d mi nistrati o n o n Da y 1 at 1, 2 0 0 m g is c urre ntl y bei n g utilize d i n 
t he P hase 2 c o h ort e x pa nsi o n p orti o n of t he st u d y. 
 
 
   
 
 
 
   
 
 
 
   
 
 
 
 
  
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
  
 
 
  
 
 
  
 
 
 
 
 
 
  
 
  
 
  
 
  
 
  
 
  
 
  
 
  
 
  
 


A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 2 0
0 7 J u ne 2 0 2 2
Fi g ure 3.
P ost-i nf usi o n Ki netics of C A R T Cells i n t he Bl o o d
B L, baseli ne; C A R, c hi meric a nti ge n rece pt or; D, da y; D L B C L, diff use lar ge B cell l y m p h o ma; M o n, m o nt h; W k, wee k.
S oli d li nes dis pla y me dia n val ues. Das he d li nes dis pla y t he i nter q uartile ( Q 1, Q 3) ra n ge. A nal ysis was n ot a vaila ble f or 1 patie nt.
Si milar t o P D- 1, 4- 1 B B e x pressi o n is u pre g ulate d o n a nti- C D 1 9 C A R T cells t hat are acti vate d 
(Fi g ure 4 ).  I ntracell ular si g nali n g d o w nstrea m of 4- 1 B B e n ga ge me nt is k n o w n t o e n ha nce T-
-cell pr oliferati o n a n d effect or f u ncti o n {S h uf or d 1 9 9 7 }, u pre g ulate  a nti-a p o pt otic pr otei ns 
{ H urta d o 1 9 9 7 }, a n d pr o m ot e s ur vi val a n d ce ll c ycle pr o gressi o n {Lee 2 0 0 3 } a n d pla ys a criti cal 
r ole i n t he f or mati o n of i m m u n ol o gical me m or y {Lee 2 0 0 2 }.  T h us, a g o nis m of 4- 1 B B o n 
e n gi neere d a nti- C D 1 9 C A R T cells mi g ht e n ha nce a nti-t u m or acti vit y of a xica bta ge ne cil ole ucel 
via t he f oll o wi n g mec ha nis ms:  ( 1) i ncreasi n g t he via bilit y of a nti- C D 1 9 C A R T cells t hr o u g h 
u pre g ulati o n of a nti-a p o pt otic pr otei ns, ( 2) e n ha nci n g a nti- C D 1 9 C A R T-cell e x pa nsi o n a n d 
pr oliferati o n, a n d 3) c o ntri b uti n g t o t he T-cell i m m u ne res p o nse.  It is, t heref ore, p ossi ble t hat 
c o nc o mita nt 4- 1 B B e n ga ge me nt c o ul d pre ve nt pri mar y a n d sec o n dar y resista nce, s u p p ort a n d 
s ustai n c o n versi o n of P R/ S D t o C R, a n d p ossi bl y pre ve nt earl y rela pse.
O ne p ossi ble c o ncer n is t hat e n ha nce d C A R T-cell e n graft me nt a n d acti vit y ma y lea d t o 
i ncrease d t o xicit y.  T o miti gate t his ris k, a starti n g ut o mil u ma b t otal d ose of 1 0 m g per d osi n g 
c ycle has bee n c h ose n. T his d ose has bee n dee me d t o be safe as a b ot h a si n gle a ge nt a n d i n 
c o m bi nati o n wit h rit u xi ma b.  F urt her details o n ut o mil u ma b d osi n g rati o nale are gi ve n i n 
Secti o n 3. 2. 3 .
 
 
  
 
 
 
 
    
  
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 




A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 2 2
0 7 J u ne 2 0 2 2
3.  
S T U D Y D E SI G N
3. 1.   
Ge ner al St u d y Desi g n
T his is a P hase 1/ 2, o pe n-la bel, m ultice nter st u d y e val uati n g t he safet y a n d efficac y of 
a xica bta ge ne cil ole ucel i n c o m bi nati o n wit h ut o mil u ma b a d mi nistrati o n i n s u bjects wit h 
rela pse d/refract or y lar ge B-cell l y m p h o ma.  T he trial will be se parate d i nt o 2 disti nct p hases 
desi g nate d as P hase 1 a n d P hase 2.
D uri n g P hase 1, u p t o 3 6 s u bjects wit h rela pse d/refract or y lar ge B-cell l y m p h o ma will be 
e nr olle d i n a 3 + 3 desi g n i n u p t o 6 of 1 0 c o h orts t o e val uate t he safet y of a xica bta ge ne cil ole ucel 
a n d ut o mil u ma b treat me nt re gi me ns.  A xica bta ge ne cil ole ucel will be a d mi nistere d as a si n gle 
d ose, a n d ut o mil u ma b will be a d mi nistere d at escalati n g d oses.  S u bjects will be e nr olle d a n d 
treate d o ne at a ti me d uri n g t he P hase 1 p orti o n of t he st u d y.  S u bject treat me nt wit h 
a xica bta ge ne cil ole ucel will be sta g gere d b y at least 2 wee ks. 
A xica bta ge ne cil ole ucel will be a d mi nistere d at a tar get d ose of 2 x 1 0 6 a nti- C D 1 9 C A R 
T cells/ k g o n Da y 0. Ut o mil u ma b will be gi n at a fi xe d d ose of 1 0 m g o n Da y 1 i n C o h ort 1 
(Fi g ure 5 ).  A n i nter nal safet y re view tea m ( S R T), c o m prisi n g t he st u d y s p o ns or a n d at least o ne 
P hase 1 i n vesti gat or, will re vie w safet y data after all s u bjects i n C o h ort 1 c o m plete t he D L T 
wi n d o w ( 2 8 da ys after t he last s u bject i n a c o h ort recei ve d t he first ut o mil u ma b i nf usi o n).  If 
C o h ort 1 passes D L T criteria (see Secti o n 9. 1 0 ), t he n t he st u d y will pr ocee d t o C o h ort 2.  If 
C o h ort 1 d oes n ot pass D L T criteria (see Secti o n 9. 1 0 ), t he n t he st u d y will pr ocee d t o C o h ort 1 A, 
w hic h will e x pl ore t he sa me fi xe d d ose of ut o mil u ma b ( 1 0 m g) a d mi nistere d o n Da y 2 1.  T he 
S R T will ma ke rec o m me n dati o ns o n f urt her st u d y c o n d uct of P hase 1, i ncl u di n g d ose escalati o n 
t o C o h ort 2 A ( 3 0 m g), or e x pl ori n g a d diti o nal d osi n g a n d/ or ti mi n g of ut o mil u ma b 
a d mi nistrati o n as nee de d.  T he sa me r ules will a p pl y t o C o h ort 2, C o h ort 3, C o h ort 4, a n d 
C o h ort 5.  Refer t o Fi g ure 5  a n d Secti o n 9. 1 0 .  
At t he c o ncl usi o n of eac h S R T safet y re vie w meeti n g, t he S R T will ma ke rec o m me n dati o ns o n 
f urt her st u d y c o n d ucts o utli ne d i n Secti o n 9. 1 0 .


A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 2 3
0 7 J u ne 2 0 2 2
Fi g ure 5.
St u d y Desi g n O ver vie w
*   
Ut o mil u ma b will be a d mi nistere d at t otal d oses of 1 0 m g, 3 0 m g, 1 0 0 m g, 2 0 0 m g, a n d 4 0 0 m g Q 4 W f or 6 m o nt hs u nless 
t here is disease pr o gressi o n.
A b bre viati o ns: D L T, d ose-li miti n g t o xicit y.
T he P hase 1 C o h orts a n d ut o mil u ma b re gi me ns a d mi nistere d are o utli ne d i n Ta ble 2 .
T a ble 2.
Ut o mil u m a b Re gi me ns
D ose Le vel *
C o h ort
First Ut o mil u m a b A d mi nistr ati o n
1
Da y 1
1 0 m g
1 A
Da y 2 1
2
Da y 1
3 0 m g
2 A
Da y 2 1
3
Da y 1
1 0 0 m g
3 A
Da y 2 1
4
Da y 1
2 0 0 m g
4 A
Da y 2 1
5
Da y 1
4 0 0 m g
5 A
Da y 2 1
*   
Ut o mil u ma b t otal d oses of 1 0 m g, 3 0 m g, 1 0 0 m g, 2 0 0 m g, a n d 4 0 0 m g will be a d mi nistere d Q 4 W f or 6 m o nt hs or u ntil 
pr o gressi ve disease ( P D), w hic he ver c o mes first.  T he S R T will ma ke rec o m me n dati o ns o n f urt her st u d y c o n d uct of P hase 1.  
 
 
 
 
 
   
 
 
 
 
 
    
 
 
 
  
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
   
 
 
 
  
 
  
 
  
 
   
      
 
 
 
 
 
 
 
  
 


A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 2 4
0 7 J u ne 2 0 2 2
I n P hase 2, u p t o 2 4 s u bjects will be e nr olle d t o recei ve a xica bta ge ne cil ole ucel a n d ut o mil u ma b 
base d o n t he d ose a n d sc he d ule selecte d t o m o ve f or war d fr o m t he P hase 1 p orti o n of t he st u d y 
as rec o m me n de d b y t he S R T.
I n de pe n de nt of t he c o h ort or p hase of t he st u d y, eac h s u bject will pr ocee d t hr o u g h t he f oll o wi n g 
st u d y peri o ds:

Scree ni n g 

E nr oll me nt/ Le u ka p heresis 

Bri d gi n g t hera p y, if a p plica ble 

C o n diti o ni n g c he m ot hera p y 

I n vesti gati o nal pr o d uct (I P) treat me nt (a xica bta ge ne cil ole ucel a n d ut o mil u ma b)

P ost-treat me nt assess me nt

L o n g-ter m F oll o w- u p ( L T F U)
T he S R T will a d diti o nall y meet o n at least 1 occasi o n d uri n g t he P hase 2 p orti o n of t he st u d y 
after 6 s u bjects ha ve c o m plete d t heir 1- m o nt h disease assess me nt.  T he S R T will re vie w safet y 
a n d efficac y data a n d will be c hartere d t o ma ke trial c o n d uct rec o m me n dati o ns base d o n a n 
a nal ysis of be nefit/ris k rati o.  Refer t o Secti o n 9. 1 0 .
F or st u d y re q uire me nt  details assi g ne d t o eac h st u d y peri o d, refer t o t he sc he d ule of assess me nts 
( S O As) i n Secti o n 7 .
A st u d y sc he ma is descri be d i n Fi g ure 1 .
3. 2.   
D osi n g R ati o n ale
3. 2. 1.   
R ati o n ale f or C o n diti o ni n g C he m ot her a p y 
I ncreasi n g le vels of c o n diti o ni n g c he m ot hera p y c orrelate wit h cli nical res p o nses t o a d o pti ve cell 
t hera p y {D u dle y 2 0 0 8 }.  S pecifi call y, t here a p pears t o be a li n k bet wee n a de q uate 
l y m p h o de pleti o n a n d a d o pti vel y tra nsferre d T-cell e x pa nsi o n a n d f u ncti o n i n precli nical m o dels, 
w hic h de m o nstrate t hat t he de pt h a n d d urati o n of l y m p h o de pleti o n c orrelates wit h a nti-t u m or 
acti vit y of t he a d o pti vel y tra nsferre d t u m or-s pecific C D 8 +  T cells {Gatti n o ni 2 0 0 5 }.  
L y m p h o de pleti o n ma y f u ncti o n b y era dicati n g c yt o ki ne si n ks f or t he tra nsferre d cells, 
eli mi nati n g T re g ulat or y cells, or e n ha nci n g a nti ge n prese nti n g cell acti vati o n { Kle ba n off 2 0 0 5 }.  
C ycl o p h os p ha mi de a n d fl u dara bi ne c o m bi nati o n is a p ote nt c o n diti o ni n g re gi me n.  
C ycl o p h os p ha mi de ( 5 0 0 m g/ m 2/ da y) a n d fl u dara bi ne ( 3 0 m g/ m2/ da y) are b ot h gi ve n f or 
3 c o nsec uti ve da ys.  T his c o m bi nati o n has bee n st u die d i n s u bjects wit h B-cell mali g na ncies a n d 
was t olerate d b y t his p o p ulati o n { O' Brie n 2 0 0 1 }.  C ycl o p h os p ha mi de a n d fl u dara bi ne 
c o m bi nati o n treat me nt was als o use d i n t he Z U M A- 1 trial { Neela p u 2 0 1 7 b }. 


A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 2 5
0 7 J u ne 2 0 2 2
3. 2. 2.   
R ati o n ale f or A xic a bt a ge ne Cil ole ucel D ose
T he rati o nale f or t he a xica bta ge ne cil ole ucel d ose i n t his st u d y is base d o n t he a g gre gate safet y 
a n d efficac y data c o m pile d fr o m Z U M A- 1 as o utli ne d i n t he a xica bta ge ne cil ole ucel I B.  Base d 
o n t he fa v ora ble be nefit/ris k rati o see n i n Z U M A- 1, a xica bta ge ne cil ole ucel will be a d mi nistere d 
at a tar get d ose of 2 x 1 0 6  a nti- C D 1 9 C A R T cells/ k g, b ut ma y be d ose d at a mi ni m u m of 
1 x 1 0 6  a nti- C D 1 9 C A R T cells/ k g.  F or s u bjects wei g hi n g > 1 0 0 k g, a ma xi m u m flat d ose of 
a xica bta ge ne cil ole ucel at 2 x 1 0 8  a nti- C D 1 9 C A R T cells will be a d mi nistere d. 
3. 2. 3.   
R ati o n ale f or Ut o mil u m a b D ose
Ut o mil u ma b ( P F- 0 5 0 8 2 5 6 6) s h o we d a fa v ora ble t o xicit y pr ofile i n all cli nical testi n g c o n d ucte d 
t h us far (Ta ble 1 ; refer t o Secti o n 2. 4. 1 ).  Si n gle-a gent ut o mil u ma b or i n c o m bi nati o n wit h 
rit u xi ma b was f o u n d t o be safe wit h n o D L Ts u p t o 1 0 m g/ k g Q 4 W i n st u d y B 1 6 4 1 0 0 1, a d ose 
e q ui vale nt t o 7 0 0 m g i n a 7 0- k g s u bject, a n d t he M T D was esti mate d t o be at least 1 0 m g/ k g 
Q 4 W.  Treat me nt-relate d, treat me nt-e mer ge nt a d verse e ve nts were m ostl y Gra de 1 or - 2 wit h 
o nl y f o ur Gra de 3/ 4 A Es re p orte d (c olitis, diarr hea, fati g ue, h y per bilir u bi ne mia, a n d 
h y p o natre mia), Gra de 3/ 4 c olitis a n d diarr hea were o bser ve d i n t he sa me patie nt.  N o Gra de 5 
treat me nt-relate d A Es were o bser ve d.
S u bse q ue ntl y, preli mi nar y p o p ulati o n P K a nal ysis was c o n d ucte d usi n g data fr o m 6 8 s u bjects 
( n = 4 1 i n P orti o n A a n d n = 2 7 fr o m P orti o n B) fr o m st u d y B 1 6 4 1 0 0 1, w hic h i n dicate d t hat t he 
e x p os ure at d ose le vels hi g her t ha n 0. 1 2 m g/ k g ( ~ 8 m g i n 7 0- k g s u bject) was a b o ve t he ass u me d 
efficaci o us c o nce ntrati o n ( Ceff, 1 7 3 0 n g/ m L) wit h b ot h o nce e ver y 3 wee ks ( Q 3 W) a n d Q 4 W 
sc he d ule.  Briefl y, efficaci o us c o nce ntrati o n ( Ceff) was esti mate d usi n g t he mea n dr u g ser u m 
c o nce ntrati o n of 1 1. 5 n M ( 1 7 3 0 n g/ m L) fr o m t he bi o mar ker, s ol u ble C D 1 3 7 (s C D 1 3 7), a n d 
t u m or gr o wt h i n hi biti o n ( T GI) st u dies usi n g a s urr o gate a nti b o d y ( Ma B 9 3 7 1), w hic h has a 
si milar affi nit y t o m o use 4- 1 B B ( K D = 1 2 n M) c o m pare d wit h t hat of P F- 0 5 0 8 2 5 6 6 t o h u ma n 4-
1 B B.  F or t he bi o mar ker s C D 1 3 7, E C 5 0 was esti mate d t o be 1 1 n M ( 1 6 5 0 n g/ m L) usi n g t he 
i n direct -res p o nse E ma x m o del. F or T GI, a c o m plete res p o nse was ac hie ve d at 0. 2 m g/ k g 
( 1 2 n M), w hic h was c o nsiste nt wit h t he E C 5 0 of 1 1 n M esti mate d fr o m t he bi o mar ker data.  
He nce, a mea n dr u g ser u m c o nce ntrati o n of 1 1. 5 n M ( 1 7 3 0 n g/ m L) fr o m t he bi o mar ker a n d T GI 
st u dies was use d f or h u ma n si n gle-a ge nt efficaci o us c o nce ntrati o n/ d ose pr ojecti o n.  Lastl y, b o d y 
wei g ht acc o u nte d f or o nl y a s mall perce nta ge ( ~ < 7 %) of t he i nter-i n di vi d ual varia bilit y i n ser u m 
P F- 0 5 0 8 2 5 6 6 e x p os ure, a n d als o si m ulati o ns i n dicate d t hat P F- 0 5 0 8 2 5 6 6 e x p os ure was si milar 
bet wee n b o d y wei g ht- base d a n d fi xe d d osi n g re gi me ns.  
 
F or t his st u d y, ut o mil u ma b was a d mi nistere d o n Da y 1 f oll o wi n g a xica bta ge ne cil ole ucel 
a d mi nistrati o n i n li g ht of pre-e xisti n g a n d i nter nal data, w hic h s h o ws u pre g ulati o n of 4- 1 B B at 
1 8 h o urs after C A R T-cell tar get e n ga ge me nt/acti vati o n ( Fi g ure 4  a n d data o n file).  I n t he e ve nt 
t hat a n y of t he d ose le vel/c o h ort wit h Da y 1 ut o mil u ma b a d mi nistrati o n fails t o pass t he D L T 
criteria, Da y 2 1 a d mi nistrati o n of ut o mil u ma b will be e x pl ore d.  Da y 2 1 a d mi nistrati o n of 
ut o mil u ma b was c h ose n beca use b y t his ti me, t he maj orit y of s u bjects will ha ve passe d t he 
wi n d o w f or ac ute a xica bta ge ne cil ole ucel-relate d t o xicities a n d pea k C A R T-cell e x pa nsi o n.  
C CI


A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 2 6
0 7 J u ne 2 0 2 2
T he starti n g d ose of 1 0 m g (t otal d ose) is 7 0-f ol d less t ha n w hat has bee n s h o w n t o be safe as 
m o n ot hera p y or i n c o m bi nati o n wit h rit u xi ma b ( 1 0 m g/ k g = 7 0 0 m g i n a 7 0- k g s u bject).  T he 
1 0- m g t otal d ose was c h ose n t o e ns ure safet y of s u bjects o n t his st u d y beca use t he effect of 
ut o mil u ma b acti vit y o n a xica bta ge ne cil ole ucel is u n k n o w n.  
At t he d ose escalati o n S R T re vie w of t he Z U M A- 1 1, data f or c o h orts 1- 3, n o D L Ts were 
o bser ve d.  W orst gra de C R S a n d N E were gra de 2.  N o ut o mil u ma b d ose dela ys d ue t o t o xicit y 
were see n i n all 3 c o h orts.  Gra de 3/ 4 treat me nt relate d, treat me nt-e mer ge nt a d verse e ve nts 
( T E A Es) were m ostl y c yt o pe nias: a ne mia ( 4 4 %), ne utr o pe nia ( 4 4 %), l y m p h o pe nia ( 3 3 %) a n d 
w hite bl o o d cell c o u nt decrease d ( 3 3 %).  T here was n o tre n d of i ncreasi n g gra de a n d/ or rate of 
c yt o pe nia wit h i ncreasi n g d ose of ut o mil u ma b.  N o treat me nt-relate d Gr 5 A Es were o bser ve d.  
F urt her, a tre n d was o bser ve d bet wee n pea k a n d c u m ulati ve le vels ( A U C 0- 2 8 d) of a nti- C D 1 9 C A R 
T-cells a n d ut o mil u ma b e x p os ure.  Me dia n C A R T-cell pea k le vels were f o u n d t o be 2. 2 ti mes 
hi g her ( 3 0 m g ut o mil u ma b c o h ort) a n d 4. 7 ti mes hi g her ( 1 0 0 m g ut o mil u ma b c o h ort) relati ve t o 
Z U M A- 1 P hase 2 C o h ort 1.  
I n t his sa me c o h ort of patie nts, pea k le vels of I L- 2 a n d I F N-γ i n ser u m i ncrease i n a d ose-
de pe n de nt ma n ner wit h ut o mil u ma b e x p os ure, p ote ntiall y de m o nstrati n g e n ha nce d T 1 T cell 
acti vit y.  M yel oi d ass ociate d c yt o ki nes a n d c he m o ki nes, pre vi o usl y ass ociate d wit h C R S a n d 
ne ur ol o gic t o xicit y ( Neela p u, N EJ M 2 0 1 7) s h o we d n o e vi de nce of ut o mil u ma b d ose- de pe n de nt 
i ncreases c o m me ns urate wit h t he lac k of i ncrease d t o xicit y relati ve t o Z U M A- 1 P hase 2 
C o h ort 1.
T he p har mac o ki netic a n d p har mac o d y na mic res ults o bser ve d i n t he first t hree c o h orts of 
Z U M A- 1 1, c o m bi ne d wit h t he cli nical o utc o me res ults s u g gest t hat a n i ncrease d d ose of 
ut oli m u ma b c o ul d f urt her e n ha nce C A R T-cell pr oliferati o n a n d acti vit y lea di n g t o a n e n ha nce d 
C R rate a n d d ura bilit y of res p o nse wit h acce pta ble safet y pr ofile.  It is ac k n o wle d ge d t hat t hese 
le vels of ut o mil u ma b are s u bsta ntiall y hi g her t ha n t he d ose ra n ge deter mi ne d f or a de q uate 
e x p os ure a n d tar get m o d ulati o n i n pre vi o us st u dies of ut o mil u ma b { Se gal 2 0 1 8 }.  H o we ver, t he  
s u per- p h ysi ol o gic le vels of T cells i n d uce d b y C A R- T cell e x pa nsi o n i n t ur n s u g gest hi g her 
le vels of i n d uce d 4- 1 B B rece pt or c o nce ntrati o n i n setti n g of a xi-cel c o m pare d t o t he p h ysi ol o gic 
le vels see n i n pre vi o us ut o mil u ma b st u dies.  T heref ore, t he P K, P D data, a n d ut o mil u ma b d ose 
le vels deter mi ne d fr o m pre vi o us st u dies li kel y are n ot a p plica ble i n t he setti n g of a xi-cel a n d 
hi g her d oses of ut o mil u ma b ma y be nee de d t o ac hie ve ma xi m u m 4- 1 B B rece pt or occ u pa nc y f or 
ma xi m u m a g o nist acti vit y. 
3. 3.  
P artici p ati n g Sites
A p pr o xi matel y 8 ce nters l ocate d i n N ort h A merica will partici pate i n t his st u d y.  D uri n g t he 
c o n d uct of t he st u d y, a d diti o nal re gi o ns, c o u ntries, or sites ma y be a d de d as necessar y .
3. 4.   
N u m ber of S u bjects
Partici pa nts i n t his trial will be referre d t o as “s u bjects.”  It is a ntici pate d t hat a p pr o xi matel y u p 
t o 6 0 s u bjects will be e nr olle d i nt o t his st u d y as defi ne d bel o w:


A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 2 7
0 7 J u ne 2 0 2 2

P hase 1: a p pr o xi matel y 3 t o 3 6 s u bjects

P hase 2: a p pr o xi matel y 2 4 s u bjects 
3. 5.   
Re pl ace me nt of S u bjects
S u bjects will be re place d a n d c o nti n ue t o be e nr olle d u ntil t he s pecifie d n u m ber of s u bjects are 
attai ne d i n t he D L T e val ua ble ( P hase 1) a n d m o difie d i nte nt-t o-treat (mI T T ) sets ( P hase 2).  See 
Secti o n 1 0  f or a d ditio nal i nf or mati o n. S u bjects w h o recei ve bet wee n 1. 0 a n d 2. 4 x 1 0 6 a nti- C D 1 9 
C A R T cells/ k g or a flat d ose of 2. 0 x 1 0 8 a nti- C D 1 9 C A R T cells f or s u bjects > 1 0 0 k g a n d w h o 
recei ve at least 1 d ose of ut o mil u ma b will be c o nsi dere d e val ua ble i n t he efficac y a nal ysis. 
S u bjects w h o ha ve n ot recei ve d a n a xica bta ge ne cil ole ucel cell d ose i n t his ra n ge or ha ve n ot 
recei ve d at least 1 d ose of ut o mil u ma b will be retai ne d i n t he a nal yses of dis p ositi o n a n d safet y, 
w here a p pr o priate ( Secti o n 1 0. 5 ).
3. 6.  
St u d y D ur ati o n
3. 6. 1.  
St u d y D ur ati o n f or I n di vi d u al S u bjects 
T he d urati o n of t he st u d y f or i n di vi d ual s u bjects will var y, de pe n di n g o n a s u bject’s scree ni n g 
re q uire me nts, res p o nse t o treat me nt a n d s ur vi val, a n d if a p plica ble, ti mi n g of tra nsiti o n t o t he 
se parate L T F U st u d y, K T- U S- 9 8 2- 5 9 6 8 ( disc usse d i n Secti o n 3. 6. 3 ).  
3. 6. 2.   
C o m pleti o n of St u d y
C o m pleti o n of t he st u d y is defi ne d as t he ti me at w hic h t he last s u bject c o m pletes 6 m o nt hs of 
assess me nts. T he e n d- of-st u d y f or eac h s u bject is defi ne d as t he last visit o n t his st u d y, or w he n a 
s u bject is c o nsi dere d l ost t o f oll o w- u p, wit h dra ws c o nse nt, or dies.  
3. 6. 3.  
L o n g-ter m F oll o w- u p
T he s u bjects w h o recei ve d a n i nf usi o n of a xica bta ge ne cil ole ucel will be pr o vi de d t he 
o p p ort u nit y t o tra nsiti o n t o a se parate L T F U st u d y, K T- U S- 9 8 2- 5 9 6 8, w here t he y will be 
m o nit ore d f or occ urre nce of late- o nset tar gete d A Es/ S A Es s us pecte d t o be p ossi bl y relate d t o 
a xica bta ge ne cil ole ucel as defi ne d i n K T- U S- 9 8 2- 5 9 6 8, prese nce of re plicati o n-c o m pete nt 
retr o vir us ( R C R) a n d/ or i nserti o nal m uta ge nesis f or u p t o 1 5 years fr o m t he ti me of a xica bta ge ne 
cil ole ucel i nf usi o n (als o refer t o Secti o n 7. 1 1 ).
F or eac h s u bject, t he fi nal visit o n t his st u d y ma y be c o m bi ne d wit h t he s u bject’s first visit o n t he 
L T F U st u d y.  T he ti mi n g of t he s u bject’s fi nal visit/first L T F U st u d y visit will de pe n d u p o n t he 
ti mi n g of t he c ollecti o n of all of t he s u bject’s data t hat are re q uire d f or t he pla n ne d a nal ysis f or 
t his st u d y.  I n K T- U S- 9 8 2- 5 9 6 8, s u bjects will c o nti n ue assess me nts at ti me p oi nts c o nti g u o us 
wit h t he L T F U ti me p oi nts i n t his st u d y.


A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 2 8
0 7 J u ne 2 0 2 2
4.  
S U B J E C T I D E N TI FI C A TI O N A S SI G N M E N T
Eac h s u bject w h o e nters t he scree ni n g peri o d, w hic h starts w he n t he s u bject si g ns t he i nf or me d 
c o nse nt f or m  (I C F), will recei ve a u ni q ue s u bject i de ntificati o n (I D) n u m ber.  T his n u m ber will 
be use d t o i de ntif y t he s u bject t hr o u g h o ut t he st u d y a n d m ust be use d o n all st u d y d oc u me ntati o n 
relate d t o t he s u bject.  T he s u bject I D n u m ber will ne ver be c ha n ge d e ve n if t he s u bject is 
rescree ne d.


A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 2 9
0 7 J u ne 2 0 2 2
5.  
S U B J E C T E LI GI BI LI T Y
5. 1.  
I ncl usi o n Criteri a
1 0 1) 
Hist ol o gicall y pr o ve n lar ge B-cell l y m p h o ma, i ncl u di n g t he f oll o wi n g t y pes defi ne d b y 
{ S wer dl o w 2 0 1 6 }:
a)
D L B C L n ot ot her wise s pecifie d (acti vate d B cell/ ger mi nal ce nter B cell [A B C/ G C B ]) 
b)
H G B C L wit h or wit h o ut M Y C a n d B C L 2 a n d/ or B C L 6 rearra n ge me nt
c)
D L B C L arisi n g fr o m F L
d)
T-cell/ histi oc yte ric h lar ge B-cell l y m p h o ma
e)
D L B C L ass ociate d wit h c hr o nic i nfla m mati o n
f)
Pri mar y c uta ne o us D L B C L, le g t y pe
g)
E pstei n- Barr vir us ( E B V) + D L B C L 
1 0 2) 
C he m ot hera p y-refract or y disease, defi ne d as o ne or m ore of t he f oll o wi n g:
a)
N o res p o nse t o first-li ne t hera p y ( pri mar y refract or y disease); s u bjects w h o are 
i nt olera nt t o first-li ne s yste mic c he m ot hera p y are e xcl u de d
i)
Pr o gressi ve disease ( P D) as best res p o nse t o first-li ne t hera p y
ii)
S D as best res p o nse after at least 4 c ycles of first-li ne t hera p y (e g, 4 c ycles of R-
C H O P) wit h S D d urati o n n o l o n ger t ha n 6 m o nt hs fr o m last d ose of t hera p y
b)
N o res p o nse t o sec o n d- or greater-li nes of t hera p y
i)
P D as best res p o nse t o m ost rece nt t hera p y re gi me n 
ii)
S D as best res p o nse after at least 2 c ycles of last li ne of t hera p y wit h S D d urati o n 
n o l o n ger t ha n 6 m o nt hs fr o m last d ose of t hera p y 
O R
c)
Refract or y p ost- A S C T
i)
Disease pr o gressi o n or rela pse d ≤ 1 2 m o nt hs after A S C T ( m ust ha ve bi o ps y 
pr o ve n rec urre nce i n rela pse d s u bjects)
ii) If sal va ge t hera p y is gi ve n p ost- A S C T, t he s u bject m ust ha ve ha d n o res p o nse t o 
or rela pse d after t he last li ne of t hera p y 
O R


A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 3 0
0 7 J u ne 2 0 2 2
d)
Rela pse d or refract or y L B C L i ncl u di n g D L B C L, T F L, a n d H G B C L after 2 li nes of 
s yste mic t hera p y t hat is defi ne d b y a n d ali g ns wit h c urre ntl y a p pr o ve d i n dicati o n:
i) 
Rela pse d disease after 2 li nes of s yste mic t hera p y
O R
ii)
Best res p o nse t hat is less t ha n a C R t o sec o n d li ne s yste mic t hera p y
1 0 3) 
At least 1 meas ura ble lesi o n acc or di n g t o t he L u ga n o Classificati o n { C hes o n 2 0 1 4 }.  
Lesi o ns t hat ha ve bee n pre vi o usl y irra diate d will be c o nsi dere d meas ura ble o nl y if 
pr o gressi o n has bee n d oc u me nte d f oll o wi n g c o m pleti o n of ra diati o n t hera p y.
1 0 4) 
S u bjects m ust ha ve recei ve d a de q uate pri or t hera p y, i ncl u di n g at a mi ni m u m: 
a)
A nti- C D 2 0 m A b u nless i n vesti gat or deter mi nes t hat t u m or is C D 2 0 - ne gati ve, a n d
b)
A n a nt hrac ycli ne c o ntai ni n g c he m ot hera p y re gi me n
1 0 5) 
N o ra di o gra p hic  e vi de nce, s us pici o n, a n d/ or hist or y of ce ntral ner v o us s yste m ( C N S) 
i n v ol ve me nt of l y m p h o ma
1 0 6) 
At least 2 wee ks or 5 half-li ves, w hic he ver is s h orter, m ust ha ve ela pse d si nce a n y pri or 
s yste mic t hera p y at t he ti me t he s u bject is pla n ne d f or le u ka p heresis. 
1 0 7) 
T o xicities d ue t o pri or t hera p y m ust be sta ble a n d rec o vere d t o ≤ Gra de 1 (e xce pt f or 
cli nicall y n o n-si g nifica nt t o xicities, s uc h as al o pecia).
1 0 8) 
A ge 1 8 or ol der
1 0 9) 
Easter n C o o perati ve O nc ol o g y Gr o u p ( E C O G) perf or ma nce stat us of 0 or 1
1 1 0) 
A bs ol ute ne utr o p hil c o u nt ( A N C) ≥ 1, 0 0 0/ μ L
1 1 1) 
Platelet c o u nt ≥ 7 5, 0 0 0/ μ L
1 1 2) 
A bs ol ute l y m p h oc yte c o u nt ≥ 1 0 0/ μ L
1 1 3) 
A de q uate re nal, he patic, p ul m o nar y, a n d car diac f u ncti o n defi ne d as:
a)
Creati ni ne cleara nce (as esti mate d b y C oc kcr oft Ga ult) ≥ 6 0 m L/ mi n
b)
Ser u m ala ni ne a mi n otra nsferase/as partate a mi n otra nsferase ( A L T/ A S T) ≤ 2. 5 u p per 
li mit of n or mal ( U L N) 
c)
T otal bilir u bi n ≤ 1. 5 m g/ d L, e xce pt i n s u bjects wit h Gil bert’s s y n dr o me
d)
Car diac ejecti o n fracti o n ≥ 5 0 % a n d n o e vi de nce of pericar dial eff usi o n wit hi n 
1 8 0 da ys pr o vi de d t he s u bject di d n ot recei ve a n a nt hrac ycli ne- base d treat me nt or 
e x perie nce a car diac e ve nt or c ha n ge i n perf or ma nce stat us 


A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 3 1
0 7 J u ne 2 0 2 2
e)
N o cli nicall y si g nifica nt ple ural eff usi o n
f)
Baseli ne o x y ge n sat urati o n > 9 2 % o n r o o m air 
1 1 4) 
Fe males of c hil d beari n g p ote ntial m ust ha ve a ne gati ve ser u m or uri ne pre g na nc y test 
(fe males w h o ha ve u n der g o ne s ur gical sterilizati o n or w h o ha ve bee n p ost- me n o pa usal 
f or at least 2 years are n ot c o nsi dere d t o be of c hil d beari n g p ote ntial)
5. 2.  
E xcl usi o n Criteri a
2 0 1) 
Hist ol o gicall y pr o ve n pri mar y me diasti nal B-cell l y m p h o ma (P M B C L )  
2 0 2) 
Hist or y of Ric hter’s tra nsf or mati o n of c hr o nic l y m p h oc ytic le u ke mia (C L L ) 
2 0 3) 
Pri or C A R t hera p y or ot her ge neticall y m o difie d T-cell t hera p y
2 0 4) 
Hist or y of se vere, i m me diate h y perse nsiti vit y reacti o n attri b ute d t o a mi n o gl yc osi des 
2 0 5) 
Prese nce or s us pici o n of f u n gal, bacterial, viral, or ot her i nfecti o n t hat is u nc o ntr olle d or 
re q uiri n g I V a nti micr o bials f or ma na ge me nt.  Si m ple uri nar y tract i nfecti o n (U TI ) a n d 
u nc o m plicate d bacterial p har y n gitis are per mitte d if res p o n di n g t o acti ve treat me nt a n d 
after c o ns ultati o n wit h t he s p o ns or’s me dical m o nit or  
2 0 6) 
Hist or y of h u ma n i m m u n o deficie nc y vir us  (HI V ) i nfecti o n or ac ute or c hr o nic acti ve 
he patitis B or C i nfecti o n.  S u bjects wit h hist or y of he patitis i nfecti o n m ust ha ve cleare d 
t heir i nfecti o n as deter mi ne d b y sta n dar d ser ol o gical a n d ge netic testi n g per c urre nt 
I nfecti o us Diseases S ociet y of A merica (I D S A) g ui deli nes or a p plica ble c o u ntr y g ui deli nes.
2 0 7) 
Prese nce of a n y i n d welli n g li ne or drai n (e g, perc uta ne o us ne p hr ost o m y t u be, i n d welli n g 
F ole y cat heter, biliar y drai n, or ple ural/ perit o neal/ pericar dial cat heter).  De dicate d ce ntral 
ve n o us access cat heters, s uc h as a P ort-a- Cat h or Hic k ma n cat heter, are per mitte d. 
2 0 8) 
S u bjects wit h detecta ble cere br os pi nal fl ui d ( C S F)  mali g na nt cells, brai n metastases, or a 
hist or y of C N S l y m p h o ma
2 0 9) 
Hist or y or prese nce of C N S dis or der, s uc h as seiz ure dis or der, cere br o vasc ular 
isc he mia/ he m orr ha ge, de me ntia, cere bellar disease, or a n y a ut oi m m u ne disease wit h C N S 
i n v ol ve me nt 
2 1 0) 
S u bjects wit h car diac atrial or car diac ve ntric ular l y m p h o ma i n v ol ve me nt
2 1 1) 
Hist or y of m y ocar dial i nfarcti o n, car diac a n gi o plast y or ste nti n g, u nsta ble a n gi na, or 
ot her cli nicall y si g nifica nt car diac disease wit hi n 1 2 m o nt hs of e nr oll me nt
2 1 2) 
Re q uire me nt f or ur ge nt t hera p y d ue t o t u m or mass effects (e g, bl o o d vessel c o m pressi o n, 
b o wel o bstr ucti o n, or tra ns m ural gastric i n v ol ve me nt)
2 1 3) 
Pri mar y i m m u n o deficie nc y 


A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 3 2
0 7 J u ne 2 0 2 2
2 1 4) 
Hist or y of a ut oi m m u ne disease (e g, Cr o h n’s, r he u mat oi d art hritis, s yste mic l u p us) 
res ulti n g i n e n d or ga n i nj ur y or re q uiri n g s yste mic i m m u n os u p pressi o n/s yste mic disease 
m o dif yi n g a ge nts wit hi n t he last 2 years.  S u bjects wit h a hist or y of a ut oi m m u ne-relate d 
h y p ot h yr oi dis m o n a sta ble d ose of t h yr oi d re place me nt h or m o ne a n d s u bjects wit h 
c o ntr olle d t y pe 1 dia betes mellit us o n a sta ble i ns uli n re gi me n ma y be eli gi ble f or t his 
st u d y
2 1 5) 
Hist or y of s y m pt o matic dee p vei n t hr o m b osis or p ul m o nar y e m b olis m wit hi n 6 m o nt hs of 
e nr oll me nt
2 1 6) 
A n y me dical c o n diti o n li kel y t o i nterfere wit h assess me nt of safet y or efficac y of st u d y 
treat me nt 
2 1 7) 
Hist or y of se vere i m me diate h y perse nsiti vit y reacti o n t o a n y of t he a ge nts use d i n t his 
st u d y 
2 1 8) 
Li ve vacci ne ≤ 6 wee ks pri or t o pla n ne d start of c o n diti o ni n g c he m ot hera p y
2 1 9) 
W o me n of c hil d beari n g p ote ntial w h o are pre g na nt or breastfee di n g beca use of t he 
p ote ntiall y da n ger o us effects of t he pre parati ve c he m ot hera p y o n t he fet us or i nfa nt.  
Fe males w h o ha ve u n der g o ne s ur gical sterilizati o n or w h o ha ve bee n p ost- me n o pa usal 
f or at least 2 years are n ot c o nsi dere d t o be of c hil d beari n g p ote ntial.
2 2 0) 
S u bjects of b ot h ge n ders w h o are n ot willi n g t o practice birt h c o ntr ol fr o m t he ti me of 
c o nse nt t hr o u g h 9 0 da ys after t he last d ose of ut o mil u ma b a n d at least 6 m o nt hs after t he 
c o m pleti o n of c o n diti o ni n g c he m ot hera p y
2 2 1) 
Hist or y of mali g na nc y ot her t ha n n o n- mela n o ma s ki n ca ncer i n sit u (e g, cer vi x, bla d der, 
breast) or l o w- gra de ( Gleas o n ≤ 6) pr ostate ca ncer or s ur veilla nce wit h o ut a n y pla ns f or 
treat me nt, u nless disease-free f or a least 3 years 
2 2 2) 
A S C T wit hi n 6 wee ks of pla n ne d e nr oll me nt 
2 2 3) 
Pri or or ga n tra ns pla ntati o n i ncl u di n g pri or all o ge neic ste m cell tra ns pla nt  ( S C T) 
2 2 4) 
Pri or C D 1 9 tar gete d t hera p y wit h t he e xce pti o n of s u bjects w h o recei ve d a xica bta ge ne 
cil ole ucel i n t his st u d y a n d are eli gi ble f or retreat me nt 
2 2 5) 
Use of a n y sta n dar d or e x peri me ntal a nti-ca ncer t hera p y wit hi n 2 wee ks pri or t o 
e nr oll me nt, i ncl u di n g c yt ore d ucti ve t hera p y a n d ra di ot hera p y, i m m u n ot hera p y, or 
c yt o ki ne t hera p y (e xce pt f or er yt hr o p oieti n) 
2 2 6) 
Pri or treat me nt wit h P D- L 1 i n hi bit or, P D- 1 i n hi bit or, a nti- C T L A 4, a nti- C D 1 3 7 ( 4- 1 B B), 
a nti- O X 4 0 or ot her i m m u ne c hec k p oi nt bl oc ka de or acti vat or t hera p y


A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 3 3
0 7 J u ne 2 0 2 2
2 2 7) 
Treat me nt wit h s yste mic i m m u n osti m ulat or y a ge nts (i ncl u di n g, b ut n ot li mite d t o, 
i nterfer o n [I F] a n d i nterle u ki n- 2 [I L- 2]) wit hi n 6 wee ks or 5 half-li ves of t he dr u g, 
w hic he ver is s h orter, pri or t o t he first ut o mil u ma b d ose
2 2 8) 
Hist or y of i di o pat hic p ul m o nar y fi br osis, or ga nizi n g p ne u m o nia (e g, br o nc hi olitis 
o blitera ns), dr u g-i n d uce d p ne u m o nitis, i di o pat hic p ne u m o nitis, or e vi de nce of acti ve 
p ne u m o nitis per c hest c o m p ute d t o m o gra p h y  ( C T) sca n at scree ni n g. Hist or y of ra diati o n 
p ne u m o nitis i n t he ra diati o n fiel d (fi br osis) is all o we d. 
2 2 9)
I n t he i n vesti gat or’s j u d g me nt, t he s u bject is u nli kel y t o c o m plete all pr ot oc ol-re q uire d 
st u d y visits or pr oce d ures, i ncl u di n g f oll o w- u p visits, or c o m pl y wit h t he st u d y 
re q uire me nts f or partici pati o n. 


A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 3 4
0 7 J u ne 2 0 2 2
6.  
P R O T O C O L T R E A T M E N T
6. 1.  
St u d y Tre at me nt
6. 1. 1.  
Le u k a p heresis
Le u ka p heresis refers t o pr oce d ure f or c ollecti n g P B M Cs t hat are use d t o ma n ufact ure t he 
s u bject-s pecific a xica bta ge ne cil ole ucel treat me nt.
S u bjects will u n der g o le u ka p heresis t o o btai n T cells f or t he ma n ufact uri n g of a xica bta ge ne 
cil ole ucel.  Le u ka p herese d cells o btai ne d at partici pati n g ce nters will be s hi p pe d t o t he s p o ns or’s 
ma n ufact uri n g facilit y as descri be d i n t he I n vesti gati o nal Pr o d uct Ma n ual (I P M). 
Le u ka p heresis ma y be re peate d i n case of ma n ufact uri n g fail ure(s).
6. 1. 2.  
Bri d gi n g T her a p y
At t he discreti o n of t he i n vesti gat or, n o n-c he m ot hera p y bri d gi n g t hera p y ma y be c o nsi dere d f or 
s u bjects wit h hi g h disease b ur de n at scree ni n g or baseli ne assess me nts (e g, b ul k y disease or 
ra pi dl y pr o gressi n g disease).  Bri d gi n g t hera p y re gi me ns are o utli ne d i n Ta ble 3 .  Bri d gi n g 
t hera p y will be s u p plie d b y t he i n vesti gati ve site u nless ot her wise n ote d. Sites s h o ul d refer t o t he 
c urre nt pr o d uct la bel f or g ui da nce o n pac ka gi n g, st ora ge, pre parati o n, a d mi nistrati o n, a n d 
t o xicit y ma na ge me nt of bri d gi n g t hera p y. 
T a ble 3.
Bri d gi n g T her a p y Re gi me ns
T y pe 
T her a p y Re gi me ns a 
Ti mi n g a n d W as h o ut Re q uire me nts 
C ortic oster oi d
De xa met has o ne at a d ose of 2 0 
m g t o 4 0 m g or e q ui vale nt, 
eit her P O or I V dail y f or 1 t o 4 
da ys.
C h oice of c ortic oster oi d a n d 
d ose ca n be a dj uste d f or 
a ge/c o m or bi dities or per l ocal or 
i nstit uti o nal g ui deli nes
Ma y be a d mi nistere d after e nr oll me nt/le u ka p heresis 
a n d m ust be c o m plete d pri or t o t he start of 
c o n diti o ni n g c he m ot hera p y
N ote:  P E T- C T,  c he mistr y pa nel, a n d C B C wit h 
differe ntial m ust be re peate d pri or t o start of 
c o n diti o ni n g c he m ot hera p y t o c o nfir m eli gi bilit y t o 
pr ocee d 
H D M P +
Rit u xi ma b
{ Castr o 2 0 0 9 }
1 gra m/ m 2 of hi g h d ose 
met h yl pre d nis ol o ne ( H D M P) 
f or 3 da ys i n c o m bi nati o n wit h 
rit u xi ma b at 3 7 5 m g/ m2 wee kl y 
f or 3 wee ks
Ma y be a d mi nistere d after e nr oll me nt/le u ka p heresis 
a n d c o m plete d at least 7 da ys pri or t o t he start of 
c o n diti o ni n g c he m ot hera p y
N ote : P E T- C T,  c he mistr y pa nel, a n d C B C wit h 
differe ntial m ust be re peate d pri or t o start of 
c o n diti o ni n g c he m ot hera p y t o c o nfir m eli gi bilit y t o 
pr ocee d
A b bre viati o ns: P E T- C T, p ositr o n e missi o n t o m o gra p h y –c o m p ute d t o m o gra p h y; C B C, c o m plete bl o o d c o u nt.
a
Bri d gi n g t hera p y re gi me n ma y be c h ose n at t he discreti o n of t he i n vesti gat or.


A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 3 5
0 7 J u ne 2 0 2 2
6. 1. 3.   
C o n diti o ni n g C he m ot her a p y
C o n diti o ni n g c he m ot hera p y refers t o c ycl o p h os p ha mi de a n d fl u dara bi ne use d f or 
l y m p h o de pleti o n pri or t o a d mi nistrati o n of a xica bta ge ne cil ole ucel.
C o n diti o ni n g c he m ot hera p y will be s u p plie d b y t he i n vesti gati ve site u nless ot her wise n ote d.  
Refer t o t he c urre nt pr o d uct la bel f or g ui da nce o n pac ka gi n g, st ora ge, pre parati o n, 
a d mi nistrati o n, a n d t o xicit y ma na ge me nt ass ociate d wit h t he a d mi nistrati o n of c he m ot hera p y 
a ge nts.
6. 1. 3. 1.  
C ycl o p h os p ha mi de
C ycl o p h os p ha mi de is a nitr o ge n m ustar d- deri vati ve t hat acts as a n al k ylati n g a ge nt f oll o wi n g 
c o n versi o n t o acti ve meta b olites i n t he li ver a n d has p ote nt i m m u n os u p pressi ve acti vit y.  T he 
ser u m half-life after I V a d mi nistrati o n ra n ges fr o m 3 t o 1 2 h o urs; t he dr u g a n d/ or its meta b olites 
ca n be detecte d i n t he ser u m f or u p t o 7 2 h o urs after a d mi nistrati o n.  
Refer t o t he m ost rece nt versi o n of t he pac ka ge i nsert f or s pecific details s urr o u n di n g t he 
a d mi nistrati o n of c ycl o p h os p ha mi de.
6. 1. 3. 2.  
Fl u dara bi ne
Fl u dara bi ne p h os p hate ( hereafter, fl u dara bi ne) is a s y nt hetic p uri ne n ucle osi de t hat differs fr o m 
p h ysi ol o gic n ucle osi des i n t hat t he s u gar m oiet y is ara bi n ose i nstea d of ri b ose or de o x yri b ose.  
Fl u dara bi ne is a p uri ne a nta g o nist a nti meta b olite.
Refer t o t he m ost rece nt versi o n of t he pac ka ge i nsert f or s pecific details s urr o u n di n g t he 
a d mi nistrati o n of fl u dara bi ne.
6. 1. 3. 3.   
Mes na
Mes na is a det o xif yi n g a ge nt use d t o i n hi bit t he he m orr ha gic c ystitis i n d uce d b y c he m ot hera p y.  
T he acti ve i n gre die nt i n mes na is a s y nt hetic s ulf h y dr yl c o m p o u n d desi g nate d as 
s o di u m- 2- merca pt oet ha ne s ulf o nate wit h a m olec ular f or m ula of C 2H 5Na O 3S 2. 
Mes na s h o ul d be a d mi nistere d per i nstit uti o nal g ui deli nes.  Refer t o t he m ost rece nt versi o n of 
t he pac ka ge i nsert f or s pecific details s urr o u n di n g t he a d mi nistrati o n of mes na.
6. 1. 4.   
A xic a bt a ge ne Cil ole ucel 
A xica bta ge ne cil ole ucel is a n I P f or t his st u d y. 
A xica bta ge ne cil ole ucel is s u p plie d cr y o preser ve d i n cr y ost ora ge ba gs.  T he pr o d uct i n t he ba g is 
sli g htl y cl o u d y a n d crea m t o yell o w c ol or.  T he cr y ost ora ge ba g c o ntai ni n g a xica bta ge ne 
cil ole ucel arri ves fr oze n i n a li q ui d nitr o ge n dr y s hi p per.  T he ba g m ust be st ore d i n va p or p hase 
of li q ui d nitr o ge n a n d re mai n fr oze n u ntil t he s u bject is rea d y f or treat me nt t o ass ure t hat via ble 


A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 3 6
0 7 J u ne 2 0 2 2
li ve a ut ol o g o us cells are a d mi nistere d t o t he s u bject.  Se veral i nacti ve i n gre die nts are a d de d t o 
t he pr o d uct t o ass ure via bilit y a n d sta bilit y of t he li ve cells t hr o u g h t he freezi n g, t ha wi n g, a n d 
i nf usi o n pr ocess.
A xica bta ge ne cil ole ucel is a s u bject-s pecific pr o d uct.  T he pr o d uct is la belle d per l ocal 
re g ulati o ns wit h t he s u bject’s u ni q ue s u bject I D n u m ber assi g ne d at t he ti me of scree ni n g.  U p o n 
recei pt, verificati o n t hat t he pr o d uct a n d s u bject-s pecific la bels matc h t he s u bject’s i nf or mati o n 
(e g, s u bject I D n u m ber) is esse ntial.  D o n ot i nf use t he pr o d uct if t he i nf or mati o n o n t he s u bject 
-s pecific la bel d oes n ot matc h t he i nte n de d s u bject.  T he v ol u me of a xica bta ge ne cil ole ucel 
i nf use d, t he t ha w start/st o p ti me, a n d a xica bta ge ne cil ole ucel a d mi nistrati o n start/st o p ti me will 
all be n ote d i n t he s u bject me dical rec or d.  T he pr o d uct m ust n ot be t ha we d u ntil t he s u bject is 
rea d y f or t he i nf usi o n.  Refer t o t he I P M f or details a n d i nstr ucti o n o n st ora ge, t ha wi n g, a n d 
a d mi nistrati o n of a xica bta ge ne cil ole ucel.  
T here ha ve bee n n o i nsta nces of acci de ntal o ver d ose of s u bjects i n t his pr o gra m t o date.  I n case 
of acci de ntal o ver d ose, treat me nt s h o ul d be s u p p orti ve.  C ortic oster oi d t hera p y ma y be 
c o nsi dere d if a n y d ose is ass ociate d wit h se vere t o xicit y.
If a n y pr o ble ms relate d t o t he use of a xica bta ge ne cil ole ucel or a n y pr o d ucts t hat s u p p ort t he 
ma na ge me nt of a xica bta ge ne cil ole ucel (e g, cr y ost ora ge ba gs, s u bject I D la bels) are i de ntifie d, 
researc h staff s h o ul d re p ort t he pr o ble m per t he i nstr ucti o ns i n t he I P M. 
6. 1. 5.   
Ut o mil u m a b
6. 1. 5. 1.  
F or m ulati o n, Pac ka gi n g, a n d Ha n dli n g 
Ut o mil u ma b dr u g pr o d uct will be s u p plie d i n clear glass vials at a 2 5 m g/ m L ( 1 2 5 m g/ vial) 
c o nce ntrati o n as sterile s ol uti o n f or I V a d mi nistrati o n a n d la bele d as o pe n s u p plies.  See t he I P M 
f or a d diti o nal i nf or mati o n.
6. 1. 5. 2.   
D osa ge, A d mi nistrati o n, a n d C o m plia nce 
Ut o mil u ma b will be a d mi nistere d as a n I V i nf usi o n o nce e ver y f o ur wee ks ( ± 2  da ys) starti n g o n 
eit her Da y 1 or Da y 2 1 ( de pe n di n g o n c o h ort).  T he starti n g d ose of ut o mil u ma b will be 1 0 m g.
As wit h a n y a nti b o d y, aller gic reacti o ns t o d ose a d mi nistrati o n are p ossi ble.  T heref ore, 
a p pr o priate dr u gs a n d me dical e q ui p me nt t o treat aller gic or a na p h ylactic reacti o ns m ust be 
i m me diatel y a vaila ble, a n d st u d y pers o n nel m ust be trai ne d t o rec o g nize a n d treat aller gic a n d 
a na p h ylactic reacti o ns.  S u bjects s h o ul d be i nstr ucte d t o re p ort a n y dela ye d reacti o ns t o t he 
i n vesti gat or i m me diatel y.  Fr o m cli nical trial e x perie nce, o nl y occasi o nal mil d, i nf usi o n-relate d 
reacti o ns ha ve bee n o bser ve d, w hic h di d n ot re q uire t he usa ge of a n y c o nc o mita nt me dicati o ns or 
pre me dicati o ns.  
T he e xact d urati o n of i nf usi o n s h o ul d be rec or de d i n b ot h s o urces d oc u me nt a n d case re p ort 
f or ms ( C R Fs).  T he i nf usi o n rate s h o ul d be re d uce d or i nterr u pte d i n t he case of s y m pt o ms of 
i nf usi o n reacti o n, a n d s y m pt o matic treat me nt s h o ul d be a d mi nistere d.  T he i nf usi o n ma y be 


A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 3 7
0 7 J u ne 2 0 2 2
c o nti n ue d at o ne- half t he pre vi o us rate u p o n i m pr o ve me nt of s y m pt o ms.  If s y m pt o ms persist or 
w orse n, t he i nf usi o n s h o ul d be disc o nti n ue d.  T he st u d y dr u g d ose escalati o n le vels are s h o w n i n 
Ta ble 2 . 
6. 1. 5. 3.   
D ose M o dificati o n
N o re d ucti o n or m o dificati o n of ut o mil u ma b d ose will be all o we d wit hi n eac h c o h ort.  A n y 
t o xicit y ass ociate d or p ossi bl y ass ociate d wit h ut o mil u ma b treat me nt s h o ul d be ma na ge d 
acc or di n g t o ut o mil u ma b I B.  A d diti o nal la b orat or y tests or bi o psies ma y be use d t o deter mi ne a 
p ossi ble i m m u n o ge nic eti ol o g y. 
Ut o mil u ma b ma y be wit h hel d f or u p t o 5 6 da ys.  If ut o mil u ma b has t o be wit h hel d f or > 5 6 da ys 
d ue t o dr u g-relate d e ve nts wit h o ut a p pr o priate res ol uti o n, des pite a p pr o priate ma na ge me nt, t he n 
ut o mil u ma b s h o ul d be disc o nti n ue d.  H o we ver, if, i n t he i n vesti gat or’s j u d g me nt, t he s u bject is 
li kel y t o deri ve be nefit fr o m res u mi n g ut o mil u ma b after a 5 6- da y dela y, ut o mil u ma b ma y be 
restarte d o nl y wit h t he a p pr o val of t he me dical m o nit or.
Ut o mil u ma b d osi n g will be dela ye d f or o n g oi n g Gra de 2 or hi g her C R S or Gra de 2 or hi g her 
N Es.  T he dela ye d d ose will be gi ve n u p o n res ol uti o n of C R S a n d ne ur ol o gic e ve nts t o Gra de 1 
or l o wer. If a d ose is hel d > 1 0 da ys be y o n d t he sc he d ule d a d mi nistrati o n, it will n ot be gi ve n. 
S h o ul d a n ut o mil u ma b d ose be dela ye d ≤ 1 0 da ys, t he re mai ni n g d oses s h o ul d be a d mi nistere d 
e ver y 2 8 da ys ( ± 2 da ys) fr o m t he o nset date of first d ose.  Li ke wise, all pr ot oc ol-re q uire d 
pr oce d ures s h o ul d f oll o w t he sa me sc he d ule, as o utli ne d i n t he S O As be gi n ni n g o n t he o nset date 
of t he first d ose.
6. 1. 6.   
C o nc o mit a nt T her a p y
C o nc o mita nt t hera p y refers t o treat me nt t hat s u bjects recei ve d uri n g t he c o n d uct of t he st u d y.
D uri n g t he c o urse of t he st u d y, i n vesti gat ors ma y prescri be a n y c o nc o mita nt t hera pies dee me d 
necessar y t o pr o vi de a de q uate s u p p orti ve care e xce pt t h ose me dicati o ns liste d i n Secti o n 6. 1. 7 .
All c o nc o mita nt t hera pies, i ncl u di n g me dicati o ns, o ver-t he-c o u nter pr o d ucts, i nt u bati o n, dial ysis, 
o x y ge n, a n d bl o o d pr o d ucts, will be rec or de d.
F or s u bjects w h o recei ve a xica bta ge ne cil ole ucel treat me nt:  

C o nc o mita nt t hera pies will be rec or de d fr o m t he date of t he i nf or me d c o nse nt u ntil 3 0 da ys 
after t he fi nal ut o mil u ma b i nf usi o n or f or 1 2 wee ks after c o m pleti n g treat me nt wit h 
a xica bta ge ne cil ole ucel ( w hic he ver is l o n ger).  

After t he p ost-treat me nt f oll o w- u p, tar gete d c o nc o mita nt t hera pies will be rec or de d f or eit her 
2 4 m o nt hs after a xica bta ge ne cil ole ucel i nf usi o n or u ntil disease pr o gressi o n, w hic he ver 
occ urs first.  Tar gete d c o nc o mita nt t hera pies i ncl u de ga m ma gl o b uli n, i m m u n os u p pressi ve 
dr u gs, a nti-i nfecti ve dr u gs, a n d vacci nati o ns.  


A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 3 8
0 7 J u ne 2 0 2 2
F or s u bjects w h o are e nr olle d, b ut n ot d ose d wit h a xica bta ge ne cil ole ucel, c o nc o mita nt t hera pies 
will be rec or de d fr o m t he date of t he i nf or me d c o nse nt u ntil 3 0 da ys after t he last st u d y-s pecific 
pr oce d ure has occ urre d (e g, le u ka p heresis, c o n diti o ni n g c he m ot hera p y) or u ntil t he i nitiati o n of 
ne w a nti-ca ncer t hera p y, w hic he ver occ urs first.
F or s u bjects w h o are n ot e nr olle d (e g, scree n fail ure), c o nc o mita nt t hera pies relate d t o a n y 
seri o us a d verse e ve nt(s) ( S A Es) will be rec or de d.
S pecific c o nc o mita nt t hera p y c ollecti o n re q uire me nts a n d i nstr ucti o ns are i ncl u de d i n t he C R F 
c o m pleti o n g ui deli nes.
6. 1. 7.  
E xcl u de d Me dic ati o ns
E xcl u de d me dicati o ns refer t o treat me nt t hat is n ot t o be a d mi nistere d, u nless ot her wise 
s pecifie d, d uri n g t he c o n d uct of t he st u d y.
C ortic oster oi d t hera p y at a p har mac ol o gic d ose ( ≥ 5 m g/ da y of pre d nis o ne or e q ui vale nt d oses of 
ot her c ortic oster oi ds) a n d ot her i m m u n os u p pressi ve dr u gs m ust be a v oi de d f or 7 da ys pri or t o 
le u ka p heresis a n d 5 da ys pri or t o a xica bta ge ne cil ole ucel a d mi nistrati o n. 
S yste mic c ortic oster oi ds ma y n ot be a d mi nistere d as pre- me dicati o n t o s u bjects f or w h o m C T 
sca ns wit h c o ntrast are c o ntrai n dicate d (ie, s u bjects wit h c o ntrast aller g y or i m paire d re nal 
cleara nce).  S uc h s u bjects s h o ul d u n der g o n o n-c o ntrast C T sca ns i nstea d. 
C ortic oster oi ds a n d ot her i m m u n os u p pressi ve dr u gs s h o ul d als o be a v oi de d f or 3 m o nt hs after 
a xica bta ge ne cil ole ucel a d mi nistrati o n u nless use d t o ma na ge a xica bta ge ne cil ole ucel-relate d 
t o xicities.  Ot her me dicati o ns, s uc h as n o nster oi dal a nti-i nfla m mat or y a ge nts, t hat mi g ht i nterfere 
wit h t he e val uati o n of a xica bta ge ne cil ole ucel s h o ul d als o be a v oi de d f or t he sa me peri o d u nless 
me dicall y necessar y.  
T hera pe utic d oses of s yste mic a ntic oa g ula nts, s uc h as u nfracti o nate d he pari n a n d l o w- m olec ular 
wei g ht he pari n, s h o ul d be a v oi de d a n yti me s u bjects are at ris k of blee di n g d ue t o 
t hr o m b oc yt o pe nia.
Treat me nts f or l y m p h o ma, s uc h as c he m ot hera p y, i m m u n ot hera p y, tar gete d a ge nts, ra diati o n, 
a n d hi g h- d ose c ortic oster oi ds ( ot her t ha n t h ose defi ne d/all o we d i n t his pr ot oc ol) a n d ot her 
i n vesti gati o nal a ge nts, are pr o hi bite d, e xce pt as nee de d f or treat me nt of disease pr o gressi o n after 
a xica bta ge ne cil ole ucel.
If per missi bilit y of a s pecific me dicati o n/treat me nt is i n q uesti o n, c o ntact t he s p o ns or’s me dical 
m o nit or.


A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 3 9
0 7 J u ne 2 0 2 2
6. 1. 8.  
S u bse q ue nt T her a p y
S u bse q ue nt t hera p y refers t o treat me nt a d mi nistere d after i n vesti gati o nal treat me nt t hat is 
necessar y t o treat a s u bject’s disease.
S u bse q ue nt t hera p y a d mi nistere d after i n vesti gati o nal treat me nt t hat is necessar y t o treat a 
s u bject’s L B C L, s uc h as n o n-st u d y s pecifie d c he m ot hera p y, i m m u n ot hera p y, tar gete d a ge nts, 
ste m cell tra ns pla nt, or ra diati o n t hera p y, will be rec or de d f or all s u bjects d ose d u ntil o ne of t he 
f oll o wi n g ha p pe ns: t he s u bject tra nsiti o ns t o t he K T- U S- 9 8 2- 5 9 6 8 L T F U st u d y, is c o nsi dere d 
l ost t o f oll o w- u p, wit h dra ws c o nse nt, or dies.  
F or s u bjects w h o are e nr olle d, b ut d o n ot recei ve i n vesti gati o nal treat me nt i nf usi o n, a n y 
a d diti o nal a nti-ca ncer t hera p y will als o be c ollecte d u ntil t he s u bject c o m pletes t heir partici pati o n 
i n t he c urre nt st u d y, is c o nsi dere d l ost t o f oll o w- u p, wit h dra ws c o nse nt, or dies, w hic he ver 
occ urs first. 
6. 1. 9.   
A xic a bt a ge ne Cil ole ucel T o xicit y M a n a ge me nt 
T o date, t he f oll o wi n g ris ks ha ve bee n i de ntifie d wit h a xica bta ge ne cil ole ucel: C R S, N Es, 
i nfecti o ns, c yt o pe nias, a n d h y p o ga m ma gl o b uli ne mia.  Refer t o Secti o n 6  of t he c urrent 
a xica bta ge ne cil ole ucel I B f or details re gar di n g t hese e ve nts a n d ma na ge me nt g ui da nce.  
As t he safet y e x perie nce wit h a xica bta ge ne cil ole ucel i ncreases, t he ma na ge me nt g ui da nce ma y 
be u p date d.  T heref ore, it is i m p orta nt t o al wa ys refer t o t he m ost c urre nt versi o n of t he 
a xica bta ge ne cil ole ucel I B f or g ui da nce re gar di n g ma na gi n g a xica bta ge ne cil ole ucel-relate d 
t o xicities.  A d diti o nal i nf or mati o n a n d ma na ge me nt rec o m me n dati o ns ca n als o be f o u n d i n t he 
I B re gar di n g i m p orta nt p ote ntial ris ks ass ociate d wit h a xica bta ge ne cil ole ucel, as well as 
p ossi ble c o m plicati o ns ass ociate d wit h mali g na nc y a n d ca ncer treat me nt. 
6. 1. 1 0.   
Ut o mil u m a b T o xicit y M a n a ge me nt
Refer t o t he c urre nt ut o mil u ma b I B f or details re gar di n g t o xicit y ma na ge me nt g ui da nce.  


A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 4 0
0 7 J u ne 2 0 2 2
7.  
S T U D Y P R O C E D U R E S
Researc h staff s h o ul d refer t o S O A Ta ble 5 , Ta ble 6 , a n d Ta ble 7  f or a n o utli ne of the pr oce d ures 
re q uire d.  
T he visit sc he d ule is calc ulate d fr o m a xica bta ge ne cil ole ucel i nf usi o n o n Da y 0. 
A n o ver vie w of st u d y assess me nts/ pr oce d ures is o utli ne d bel o w.  Refer t o t he C R F c o m pleti o n 
g ui deli nes f or data c ollecti o n re q uire me nts a n d best practices f or d oc u me ntati o n of st u d y 
pr oce d ures.
7. 1.  
I nf or me d C o nse nt
Bef ore a s u bject partici pates i n t he cli nical st u d y, t he i n vesti gat or is res p o nsi ble f or o btai ni n g 
writte n i nf or me d c o nse nt fr o m t he s u bject after a de q uatel y e x plai ni n g t he st u d y desi g n, 
a ntici pate d be nefits, a n d p ote ntial ris ks.  S u bjects s h o ul d si g n t he m ost c urre nt I nstit uti o nal 
Re vie w B oar d/I n de pe n de nt Et hics C o m mittee (I R B/I E C) a p pr o ve d I C F bef ore a n y st u d y-
s pecific acti vit y or pr oce d ure is perf or me d.
T he c o nse nt pr ocess a n d t he s u bject’s a gree me nt or ref usal t o partici pate i n t he st u d y m ust be 
d oc u me nte d i n t he s u bject’s me dical rec or ds.  If t he s u bject a grees t o partici pate, t he I C F m ust be 
si g ne d a n d date d b y b ot h t he s u bject a n d t he pers o n w h o c o n d ucte d t he i nf or me d c o nse nt 
disc ussi o n.  T he ori gi nal si g ne d I C F will be retai ne d i n acc or da nce wit h i nstit uti o n p olic y a n d 
I R B/I E C re q uire me nts, a n d a c o p y of t he I C F will be pr o vi de d t o t he s u bject.
All s u bjects w h o are e nr olle d i nt o t he st u d y s h o ul d be re-c o nse nte d wit h a n y u p date d versi o n of 
t he I R B/I E C-a p pr o ve d I C F if t he ne w versi o n is rele va nt t o t heir partici pati o n.
7. 2.  
Scree ni n g
I n vesti gati ve sites will mai ntai n a l o g of all scree ne d s u bjects w h o were re vie we d a n d e val uate d 
f or st u d y partici pati o n.  I nf or mati o n c ollecte d i n t he scree ni n g l o g s h o ul d i ncl u de li mite d 
i nf or mati o n, s uc h as t he date of scree ni n g, date t he s u bject was e nr olle d, or t he reas o n f or w h y 
t he s u bject faile d scree ni n g.
T he scree ni n g peri o d be gi ns o n t he date t he s u bject si g ns t he I R B/I E C-a p pr o ve d I C F a n d 
c o nti n ues t hr o u g h c o nfir mati o n of eli gi bilit y i nt o t he st u d y.  I nf or me d c o nse nt m ust be o btai ne d 
bef ore c o m pleti o n of a n y n o n-sta n dar d of care st u d y-s pecific pr oce d ures.  Pr oce d ures t hat are 
part of sta n dar d of care ( S O C) are n ot c o nsi dere d st u d y-s pecific a n d, t heref ore, ma y be 
perf or me d pri or t o o btai ni n g c o nse nt a n d use d t o c o nfir m eli gi bilit y pr o vi de d t he y occ ur wit hi n 
t he ti me all o wa nce o utli ne d bel o w a n d i n t he S O A.
After writte n i nf or me d c o nse nt has bee n o btai ne d, Kite P har ma, I nc., will assi g n a scree ni n g 
n u m ber t o t he s u bject, as descri be d i n Secti o n 4 .  


A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 4 1
0 7 J u ne 2 0 2 2
See Secti o n 7. 2. 1  f or t he st u d y pr oced ures f or s u bjects w h o rescree n i nt o t he st u d y.  O nl y 
s u bjects w h o meet t he eli gi bilit y criteria liste d i n Secti o n 5  a n d w h o c o m mence le u ka p heresis 
will be e nr olle d i nt o t he st u d y.  If at a n y ti me pri or t o e nr oll me nt t he s u bject fails t o meet t he 
eli gi bilit y criteria, t he s u bject s h o ul d be desi g nate d as a scree n fail ure o n t he s u bject scree ni n g 
l o g, a n d t he reas o ns f or faili n g scree ni n g s h o ul d als o be rec or de d.  T he scree ni n g wi n d o w is 
2 8 da ys pri or t o e nr oll me nt.
Refer t o t he S O A f or a listi n g of st u d y pr oce d ures t o be c o m plete d d uri n g t he scree ni n g peri o d. 
7. 2. 1.   
Rescree ni n g
S u bjects w h o are u na ble t o c o m plete or meet t he eli gi bilit y criteria d uri n g t he 2 8- da y scree ni n g 
peri o d will be per mitte d t o rescree n o ne ti me.  S u bjects will retai n t he sa me s u bject I D  n u m ber 
assi g ne d at t he ori gi nal scree ni n g.  If rescree ni n g occ urs wit hi n 2 8 da ys of t he si g ni n g of t he 
ori gi nal i nf or me d c o nse nt, it is o nl y necessar y t o perf or m t he pr oce d ure(s)/assess me nt(s) t hat di d 
n ot ori gi nall y meet t he eli gi bilit y criteria; all ot her i nitial scree ni n g pr oce d ures/assess me nts d o 
n ot nee d t o be re peate d.  If rescree ni n g occ urs or le u ka p heresis is dela ye d m ore t ha n 2 8 da ys 
fr o m t he si g ni n g of t he ori gi nal i nf or me d c o nse nt, s u bjects m ust be re-c o nse nte d a n d re peat all 
scree ni n g pr oce d ures/assess me nts.
7. 3.   
De m o gr a p hic D at a
De m o gra p hic data will be c ollecte d as per c o u ntr y a n d l ocal re g ulati o ns a n d g ui deli nes.  W here 
a p plica ble, de m o gra p hic data will i ncl u de se x, year of birt h, race, et h nicit y, a n d c o u ntr y of 
e nr oll me nt t o st u d y a p ossi ble ass ociati o n bet wee n t hese varia bles a n d s u bject safet y a n d 
treat me nt effecti ve ness.
7. 4.   
Me dic al a n d Tre at me nt Hist or y
Rele va nt me dical hist or y pri or t o t he start of A E re p orti n g will be c ollecte d.  Rele va nt me dical 
hist or y is defi ne d as data o n t he s u bject’s c urre nt me dical c o n diti o n t hat w o ul d be t y picall y 
s hare d i n a referral letter. I n a d diti o n t o t he me dical hist or y, all hist or y relate d t o t he s u bject’s 
disease, treat me nt, a n d res p o nse t o treat me nt will be c ollecte d a n d m ust date bac k t o t he ori gi nal 
dia g n osis.  All fi n di n gs will be rec or de d i n t he C R Fs.
F or s u bjects w h o are bei n g referre d fr o m a n ot her cli nic or i nstit uti o n t o t he partici pati n g researc h 
ce nter, c o pies fr o m t he s u bject’s c hart s h o ul d be o btai ne d.
7. 5.  
P h ysic al E x a m, Vit al Si g ns, a n d Perf or m a nce St at us 
P h ysical e xa ms will be perf or me d d uri n g scree ni n g a n d at ti mes n ote d i n t he S O A.  All p h ysical 
e xa m c ha n ges n ote d i n s u bse q ue nt e xa ms w he n c o m pare d t o t he baseli ne e xa m will be re p orte d 
as A Es.  
Vital si g ns, i ncl u di n g bl o o d press ure, heart rate, res pirat or y rate, o x y ge n sat urati o n, a n d 
te m perat ure, will be m o nit ore d a n d rec or de d at scree ni n g a n d at ti mes o utli ne d i n t he S O A.  I n 
a d diti o n t o t he ti me p oi nts o utli ne d i n t he S O A, it is rec o m me n de d t hat vital si g ns are m o nit ore d 
d uri n g a n d after st u d y treat me nt a n d as cli nicall y i n dicate d.  


A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 4 2
0 7 J u ne 2 0 2 2
F or t he first i nf usi o n of ut o mil u ma b, t he s u bject’s vital si g ns ( bl o o d press ure, heart rate, 
res pirat or y rate, o x y ge n sat urati o n, a n d te m perat ure) s h o ul d be deter mi ne d wit hi n 6 0 mi n utes 
bef ore t he start of i nf usi o n, d uri n g t he i nf usi o n (e ver y 1 5 [ ± 5] mi n utes), a n d 3 0 ( ± 1 0) mi n utes 
a n d 2 h o urs ( ± 1 5 mi n utes) after t he start of i nf usi o n. F or s u bse q ue nt ut o mil u ma b i nf usi o ns, vital 
si g ns will be c ollecte d wit hi n 6 0 mi n utes bef ore t he start of i nf usi o n, d uri n g t he i nf usi o n if 
cli nicall y i n dicate d or if s y m pt o ms occ urre d i n t he pri or i nf usi o n, a n d 1 h o ur ( ± 1 0 mi n utes) after 
t he start of i nf usi o n.
Perf or ma nce stat us as meas ure d b y t he E C O G scale will be perf or me d t o q ua ntif y t he s u bject’s 
ge neral well- bei n g a n d a bilit y t o perf or m acti vities of dail y life.
7. 5. 1.   
C ar di ac F u ncti o n
Eac h s u bject’s car diac f u ncti o n, as meas ure d b y left ve ntric ular ejecti o n fracti o n ( L V E F), will be 
assesse d d uri n g t he scree ni n g peri o d t o c o nfir m st u d y eli gi bilit y.  E vi de nce of n o pericar dial 
eff usi o n will als o be c o nfir me d per st u d y eli gi bilit y criteria.  B ot h L V E F a n d prese nce of 
pericar dial eff usi o n will be assesse d b y ec h ocar di o gra m ( E C H O) pri or t o e nr oll me nt.  A n E C H O 
t hat was perf or me d after t he s u bject’s last c he m ot hera p y treat me nt ma y als o be use d t o c o nfir m 
eli gi bilit y, pr o vi de d t hat it occ urre d ≤ 2 8 da ys pri or t o si g ni n g t he c o nse nt.
T o esta blis h a baseli ne, a 1 2-lea d electr ocar di o gra m ( E C G) will als o be perf or me d d uri n g t he 
scree ni n g peri o d.
7. 6.   
Ne ur ol o gic al E x a mi n ati o n
A ne ur ol o gical e xa mi nati o n will be perf or me d, a n d a n y of t he f oll o wi n g a b n or malities will be 
rec or de d:  le vel of c o nsci o us ness, orie ntati o n, visi o n, cra nial ner ves a n d brai n ste m f u ncti o ns, 
p yra mi dal a n d e xtra p yra mi dal m ot or s yste m, refle xes, m uscle t o ne a n d tr o p hic fi n di n gs, 
c o or di nati o n, se ns or y s yste m, a n d ne ur o ps yc h ol o gical fi n di n gs (e g, s peec h, c o g niti o n, a n d 
e m oti o n). 
A ne ur ol o gical assess me nt s h o ul d be d o ne pri or t o a xica bta ge ne cil ole ucel i nf usi o n o n treat me nt 
Da y 0, t he n o n treat me nt Da y 1, a n d o n e ver y ot her da y d uri n g t he o bser vati o n peri o d, w hic h 
m ust last a mi ni m u m of 7 da ys.  
S u bjects will be s pecificall y as ke d a b o ut c ha n ges i n ne ur ol o gical stat us si nce t he pre vi o us 
ne ur ol o gical e xa mi nati o n, as n ote d i n t he S O A (see Ta ble 5 , Ta ble 6 , a n d Ta ble 7 ).  
7. 7.   
Dise ase Assess me nt 
S u bjects will be e val uate d f or disease res p o nse b y t he site i n vesti gat or at ti mes i n dicate d i n t he 
S O A.  Disease assess me nts will be e val uate d per t he L u ga n o Classificati o n { C hes o n 2 0 1 4 }; refer 
t o A p pe n di x 2 .  Fl o w c yt o metric, m olec ular, or c yt o ge netic st u dies will n ot be use d t o deter mi ne 
res p o nse.


A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 4 3
0 7 J u ne 2 0 2 2
7. 7. 1.  
I m a gi n g
7. 7. 1. 1.  
I ma gi n g at Baseli ne
Eac h s u bject will u n der g o a scree ni n g brai n ma g netic res o na nce i ma gi n g ( M RI) t o r ule o ut C N S 
metastasis d uri n g t he scree ni n g peri o d of t he st u d y.
T o c o nfir m eli gi bilit y a n d/ or t o esta blis h a baseli ne, p ositr o n e missi o n t o m o gra p h y –c o m p ute d 
t o m o gra p h y ( P E T- C T) sca ns of t he nec k, c hest, a b d o me n, a n d pel vis, al o n g wit h t he a p pr o priate 
i ma gi n g of all ot her sites of disease, are re q uire d at scree ni n g.  P E T- C T perf or me d f oll o wi n g t he 
s u bjects last li ne of t hera p y a n d pri or t o si g ni n g t he c o nse nt ma y be use d f or c o nfir mati o n of 
eli gi bilit y if wit hi n 2 8 da ys pri or t o e nr oll me nt, a n d n o ot her a nti-ca ncer treat me nt has bee n 
a d mi nistere d.  If P E T- C T is perf or me d > 2 8 da ys pri or t o e nr oll me nt or if s u bject recei ves a n y 
a nti-ca ncer t hera p y bet wee n scree ni n g a n d c o n diti o ni n g c he m ot hera p y, t he P E T- C T sca n m ust 
be re peate d t o esta blis h a ne w baseli ne. P E T- C T s h o ul d be perf or me d as cl ose t o e nr oll me nt as 
p ossi ble.
7. 7. 1. 2.  
P ost-treat me nt Res p o nse Assess me nt
T he first pla n ne d p ost-treat me nt P E T- C T t u m or assess me nt will occ ur at Wee k 4.
P E T- C Ts will c o nti n ue at ti me p oi nts o utli ne d i n t he S O A t hr o u g h M o nt h 2 4 or u ntil disease 
pr o gressi o n, w hic he ver c o mes first.  If t he s u bject’s disease has n ot pr o gresse d b y M o nt h 2 4, 
disease assess me nts will c o nti n ue t o be perf or me d per S O C.  S u bjects wit h s y m pt o ms s u g gesti ve 
of disease pr o gressi o n s h o ul d be e val uate d f or pr o gressi o n at t he ti me s y m pt o ms occ ur e ve n if 
t his re q uires a n u nsc he d ule d visit.
7. 7. 1. 3.  
Res p o nse E val uati o n t o Retreat me nt
F or t he p ur p ose of deter mi ni n g res p o nse t o retreat me nt wit h a xica bta ge ne cil ole ucel, t he last 
sca n pri or t o retreat me nt will be c o nsi dere d t he baseli ne.
7. 7. 2.   
Deter mi n ati o n of B o ne M arr o w I n v ol ve me nt
A s u bject’s b o ne marr o w i n v ol ve me nt s h o ul d be c o nfir me d b y P E T- C T or b o ne marr o w bi o ps y 
a n d as pirate pri or t o t he start of c o n diti o ni n g c he m ot hera p y.  C o nfir mati o n of marr o w 
i n v ol ve me nt ca n be deter mi ne d u p o n i nitial dia g n osis of disease or, if ne gati ve or u n k n o w n at 
i nitial dia g n osis, at t he ti me of rela pse fr o m first-li ne t hera p y.  
If t here is e vi de nce of baseli ne b o ne marr o w i n v ol ve me nt a n d P E T- C T is n ot a vaila ble or if t here 
are u ne x plai ne d c yt o pe nias or s us pici o n of b o ne marr o w i n v ol ve me nt, a b o ne marr o w as pirate 
a n d bi o ps y will be perf or me d i n s u bjects w h o are bei n g assesse d f or C R i n or der t o c o nfir m 
c o m plete res p o nse.  T he b o ne marr o w as pirate a n d bi o ps y m ust s h o w n o e vi de nce of disease b y 
m or p h ol o g y, or, if i n deter mi nate b y m or p h ol o g y, it m ust be ne gati ve b y i m m u n o hist oc he mistr y  
(I H C).  Refer t o A p pe n di x 2  f or treat me nt res p o nse assess me nt re q uire me nts per t he L u ga n o 
Classificati o n { C hes o n 2 0 1 4 }. 


A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 4 4
0 7 J u ne 2 0 2 2
B o ne marr o w as pirate/ bi o ps y s h o ul d als o be c o nsi dere d t o e val uate he m o p ha g oc ytic 
l y m p h o histi oc yt osis ( H L H) as i n dicate d.  Refer t o t he a xica bta ge ne cil ole ucel I B f or a d diti o nal 
i nf or mati o n.
7. 8.   
Cell C ollecti o n a n d A xic a bt a ge ne Cil ole ucel St u d y Tre at me nt Sc he d ule a n d 
A d mi nistr ati o n
7. 8. 1.  
Le u k a p heresis 
S u bjects m ust re mai n eli gi ble per t he eli gi bilit y criteria o utli ne d i n Secti o n 5  pri or t o t he start of 
le u ka p heresis. 
If a n y scree ni n g assess me nts or pr oce d ures are re peate d bet wee n c o nfir mati o n of eli gi bilit y a n d 
t he start of le u ka p heresis a n d res ults are o utsi de t he eli gi bilit y criteria liste d i n Secti o n 5 , c o ntact 
t he s p o ns or’s me dical m o nit or pri or t o pr ocee di n g wit h le u ka p heresis.
Bef ore le u ka p heresis c o m me nces, t he bel o w criteria m ust be met:

N o e vi de nce or s us pici o n of a n i nfecti o n

C ortic oster oi d t hera p y at a p har mac ol o gic d ose ( ≥ 5 m g/ da y of pre d nis o ne or e q ui vale nt 
d oses of ot her c ortic oster oi ds) a n d ot her i m m u n os u p pressi ve dr u gs m ust be a v oi de d f or 
7 da ys pri or t o le u ka p heresis.
If criteria are n ot met, le u ka p heresis m ust be dela ye d u ntil t he e ve nt res ol ves.  If le u ka p heresis is 
dela ye d m ore t ha n 5 da ys after eli gi bilit y c o nfir mati o n, baseli ne c o m plete bl o o d c o u nt (C B C) 
wit h differe ntial a n d c he mistr y pa nel m ust be re peate d.  If res ults are o utsi de t he eli gi bilit y 
criteria liste d i n Secti o n 5 , c o ntact t he me dical m o nit or pri or t o pr ocee di n g wit h le u ka p heresis. 
T he le u ka p heresis visit s h o ul d occ ur wit hi n a p pr o xi matel y 5 da ys of eli gi bilit y c o nfir mati o n.  
After a s u bject c o m me nces le u ka p heresis, t he s u bject will be c o nsi dere d e nr olle d i nt o t he st u d y.  
After t he a b o ve criteria are met, m o n o n uclear cells will be o btai ne d b y le u ka p heresis 
( 1 2 t o 1 5 L) wit h a g oal t o tar get a p pr o xi matel y 5 t o 1 0 x 1 0 9 m o n o n uclear cells.  T he 
le u ka p herese d cells are t he n pac ka ge d f or e x pe dite d s hi p me nt t o t he ma n ufact uri n g facilit y as 
descri be d i n t he I P M.
Refer t o t he S O A Ta ble 5  a n d Ta ble 6  f or a listi n g of st u d y pr oced ures t o be c o m plete d o n t he 
le u ka p heresis c ollecti o n da y (pri or t o t he start of le u ka p heresis).  L ocal a n d ce ntral la bs ca n be 
c ollecte d o n t he da y pri or t o le u ka p heresis.
7. 8. 2.   
C o n diti o ni n g C he m ot her a p y Peri o d
A d mi nistrati o n of C A R T cells t o s u bjects wit h o n g oi n g i nfecti o n or i nfla m mati o n, e ve n if s uc h 
pr ocesses are as y m pt o matic, i ncreases t he ris k of hi g h gra de a n d fatal t o xicit y.  All eff orts s h o ul d 
be ma de t o r ule o ut s uc h c o n diti o ns pri or t o cell i nf usi o n.  


A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 4 5
0 7 J u ne 2 0 2 2
Si g ns, s y m pt o ms, or a b n or mal la b orat or y res ults attri b ute d t o t he mali g na nc y (e g, “t u m or fe ver,” 
ele vate d C-reacti ve pr otei n [C R P ]) are c o nsi dere d t o be dia g n oses of e xcl usi o n a n d, t h us, re q uire 
a d oc u me nte d w or k u p t o de m o nstrate t hat all ot her p ossi ble dia g n oses are r ule d o ut. 
C o n diti o ni n g c he m ot hera p y a n d a xica bta ge ne cil ole ucel i nf usi o n s h o ul d be i nitiate d o nl y w he n it 
is reas o na bl y ass ure d t hat cell i nf usi o n ca n safel y pr ocee d.
Refer t o Secti o n 7. 8. 2. 1 f or re q uire me nts t o w or k u p p ote ntial i nfecti o us a n d/ or i nfla m mat or y 
states.
7. 8. 2. 1.  
Criteria Pri or t o C o n diti o ni n g C he m ot hera p y
If a n y of t he f oll o wi n g criteria are met pri or t o t he i nitiati o n of c o n diti o ni n g c he m ot hera p y, t he n 
t he w or k u p liste d i n Secti o n 7. 8. 3. 1. 1 . m ust be perf or me d t o deter mi ne t he p ote ntial ca use if 
t here is n o i de ntifie d s o urce of i nfecti o n.  

Te m perat ure > 3 8° C wit hi n 7 2 h o urs of c o n diti o ni n g c he m ot hera p y

C R P > 1 0 0 m g/ L a n yti me bet wee n e nr oll me nt t o start of c o n diti o ni n g c he m ot hera p y

W hite bl o o d c o u nt ( W B C) c o u nt or W B C differe ntial c o ncer ni n g f or i nfecti o us pr ocess 
bet wee n e nr oll me nt t o start of c o n diti o ni n g c he m ot hera p y (e g, W B C > 2 0, 0 0 0/ µ L, ra pi dl y 
i ncreasi n g W B C, or differe ntial wit h hi g h perce nta ge of se g me nts/ ba n ds)
A d diti o nall y:

If a n y scree ni n g assess me nts or pr oce d ures are re peate d bet wee n c o nfir mati o n of eli gi bilit y 
a n d t he start of c o n diti o ni n g c he m ot hera p y a n d res ults are o utsi de t he eli gi bilit y criteria liste d 
i n Secti o n 5 , t he n t he c o n diti o n m ust res ol ve pri or t o pr ocee di n g wit h c o n diti o ni n g 
c he m ot hera p y.

C o m plete hist or y a n d p h ysical e xa m, i ncl u di n g hea d, e ye, ear, n ose, a n d t hr oat ( H E E N T) a n d 
car diac, vasc ular, res pirat or y, gastr oi ntesti nal, i nte g u me ntar y, a n d ne ur ol o gical s yste ms, m ust 
n ot re veal e vi de nce of i nfecti o n/i nfla m mati o n. 

T he s u bject m ust n ot ha ve recei ve d s yste mic a nti micr o bials f or t he treat me nt of a k n o w n or 
s us pecte d i nfecti o n wit hi n 4 8 h o urs bef ore c o n diti o ni n g c he m ot hera p y ( pr o p h ylactic use of 
a nti micr o bials is all o we d).  

Treat me nt c o urse of a n y a nti micr o bials gi ve n f or k n o w n or s us pecte d a ntece de nt i nfecti o n 
s h o ul d be c o m plete as per i nfecti o us disease c o ns ult (if a p plica ble) rec o m me n dati o n bef ore 
st o p pi n g or s witc hi n g t o pr o p h ylactic a nti micr o bials.  

If a s u bject is c o nfir me d t o ha ve a n i nfecti o us pr ocess f or w hic h a nti micr o bials are n ot 
a vaila ble (e g, viral p ne u m o nia), t he i nfecti o n m ust be cli nicall y res ol ve d as deter mi ne d b y 
t he i n vesti gat or i n c o ns ultati o n wit h i nfecti o us disease ser vice (if a p plica ble).


A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 4 6
0 7 J u ne 2 0 2 2

M ost rece ntl y c ollecte d bl o o d, uri ne, or ot her b o d y fl ui d c ult ures m ust s h o w n o gr o wt h f or at 
least 4 8 h o urs, a n d a n y ot her i nfecti o us w or k u p perf or me d (e g, bacterial, viral ser ol o gies, 
p ol y merase c hai n reacti o n [P C R ], st o ol st u dies, i ma gi n g st u dies) m ust be ne gati ve.  If 
cli nical s us pici o n is f or a n i nfecti o n f or w hic h c ult ures are u nli kel y t o be p ositi ve wit hi n 
4 8 h o urs (e g, f u n gal i nfecti o n), a de q uate ti me m ust be all o we d f or c ult ures t o bec o me 
p ositi ve.
After t he a b o ve criteria are met, t he n t he s u bject ca n pr ocee d wit h c o n diti o ni n g c he m ot hera p y.
7. 8. 2. 2.  
C o n diti o ni n g C he m ot hera p y A d mi nistrati o n ( Da y – 5 t hr o u g h Da y – 3 Pri or t o 
A xica bta ge ne Cil ole ucel I nf usi o n)
S u bjects will recei ve a c o n diti o ni n g c he m ot hera p y re gi me n c o nsisti n g of c ycl o p h os p ha mi de a n d 
fl u dara bi ne.  T he first d ose of c o n diti o ni n g c he m ot hera p y will be desi g nate d as Da y – 5.  
S u bjects will i nitiate c o n diti o ni n g c he m ot hera p y wit h c ycl o p h os p ha mi de a n d fl u dara bi ne 
be gi n ni n g o n Da y – 5 a n d t hr o u g h Da y – 3, wit h 2 rest da ys ( Da y – 2 a n d Da y – 1) bef ore recei vi n g 
a xica bta ge ne cil ole ucel.  T he 3- da y c o n diti o ni n g c he m ot hera p y re gi me n will be a d mi nistere d i n 
a n o ut patie nt setti n g. 
T he 3- da y c o n diti o ni n g re gi me n of c ycl o p h os p ha mi de a n d fl u dara bi ne will be a d mi nistere d i n 
acc or da nce wit h t he f oll o wi n g dail y d osi n g i nstr ucti o ns.

I V h y drati o n wit h a bala nce d cr ystall oi d acc or di n g t o i nstit uti o nal g ui deli nes pri or t o 
a d mi nistrati o n of c ycl o p h os p ha mi de o n t he da y of i nf usi o n

C ycl o p h os p ha mi de 5 0 0 m g/ m 2 I V o ver 6 0 mi n utes ( ± 1 5 mi n utes) f oll o we d b y

Fl u dara bi ne 3 0 m g/ m 2 I V o ver 3 0 mi n utes ( ± 1 5 mi n utes) f oll o we d b y

A d diti o nal I V h y drati o n wit h a bala nce d cr ystall oi d acc or di n g t o i nstit uti o nal g ui deli nes t o be 
a d mi nistere d u p o n c o m pleti o n of i nf usi o n

Mes na t o be a d mi nistere d per i nstit uti o nal g ui deli nes
S u bjects s h o ul d be i nstr ucte d t o dri n k ple nt y of li q ui ds d uri n g c he m ot hera p y a n d t hr o u g h o ut t he 
2 4- h o ur peri o d f oll o wi n g c he m ot hera p y (a p pr o xi matel y 2 L/ 2 4 h o urs).  I n ge neral, s u bjects 
s h o ul d be ke pt well- h y drate d b ut cl osel y m o nit ore d t o pre ve nt fl ui d o verl oa d. 
Refer t o t he S O A Ta ble 5  a n d Ta ble 6  f or a listi n g of st u d y pr oced ures t o be c o m plete d d uri n g 
t he c o n diti o ni n g c he m ot hera p y peri o d. 


A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 4 7
0 7 J u ne 2 0 2 2
7. 8. 3.  
I n vesti g ati o n al Pr o d uct Tre at me nt Peri o d
7. 8. 3. 1.   
A xica bta ge ne Cil ole ucel Pretreat me nt Criteria 
If a n y of t he f oll o wi n g criteria are met pri or t o t he i nitiati o n of a xica bta ge ne cil ole ucel i nf usi o n, 
t he n t he w or k u p liste d i n Secti o n 7. 8. 3. 1. 1 . m ust be perf or me d t o deter mi ne t he p ote ntial ca use if 
t here is n o i de ntifie d s o urce of i nfecti o n.  

Te m perat ure > 3 8° C wit hi n 7 2 h o urs of a xica bta ge ne cil ole ucel i nf usi o n

C R P > 1 0 0 m g/ L a n yti me bet wee n e nr oll me nt t o start of a xica bta ge ne cil ole ucel i nf usi o n 

W B C c o u nt or W B C differe ntial c o ncer ni n g f or i nfecti o us pr ocess bet wee n e nr oll me nt t o 
start of a xica bta ge ne cil ole ucel i nf usi o n (e g, W B C > 2 0, 0 0 0/ µ L, ra pi dl y i ncreasi n g W B C, or 
differe ntial wit h hi g h perce nta ge of se g me nts/ ba n ds)
A d diti o nall y:

If a n y scree ni n g assess me nts or pr oce d ures are re peate d bet wee n c o nfir mati o n of eli gi bilit y 
a n d t he start of a xica bta ge ne cil ole ucel i nf usi o n a n d res ults are o utsi de t he eli gi bilit y criteria 
liste d i n Secti o n 5 , t he n t he c o n diti o n m ust res ol ve pri or t o pr ocee di n g wit h a xica bta ge ne 
cil ole ucel i nf usi o n (e xce pt f or peri p heral bl o o d cell c o u nts t hat ha ve bee n i m pacte d b y 
c o n diti o ni n g c he m ot hera p y).

C o m plete hist or y a n d p h ysical e xa m, i ncl u di n g H E E N T a n d car diac, vasc ular, res pirat or y, 
gastr oi ntesti nal, i nte g u me ntar y, a n d ne ur ol o gical s yste ms, m ust n ot re veal e vi de nce of 
i nfecti o n/i nfla m mati o n. 

T he s u bject m ust n ot ha ve recei ve d s yste mic a nti micr o bials f or t he treat me nt of a k n o w n or 
s us pecte d i nfecti o n wit hi n 4 8 h o urs bef ore a xica bta ge ne cil ole ucel i nf usi o n ( pr o p h ylactic use 
of a nti micr o bials is all o we d).  

Treat me nt c o urse of a n y a nti micr o bials gi ve n f or k n o w n or s us pecte d a ntece de nt i nfecti o n 
s h o ul d be c o m plete as per i nfecti o us disease c o ns ult (if a p plica ble) rec o m me n dati o n bef ore 
st o p pi n g or s witc hi n g t o pr o p h ylactic a nti micr o bials.  

If a s u bject is c o nfir me d t o ha ve a n i nfecti o us pr ocess f or w hic h a nti micr o bials are n ot 
a vaila ble (e g, viral p ne u m o nia), t he i nfecti o n m ust be cli nicall y res ol ve d as deter mi ne d b y 
t he i n vesti gat or i n c o ns ultati o n wit h i nfecti o us disease ser vice (if a p plica ble).

M ost rece ntl y c ollecte d bl o o d, uri ne, or ot her b o d y fl ui d c ult ures m ust s h o w n o gr o wt h f or at 
least 4 8 h o urs, a n d a n y ot her i nfecti o us w or k u p perf or me d (e g, bacterial, viral ser ol o gies, 
P C R, st o ol st u dies, i ma gi n g st u dies) m ust be ne gati ve.  If cli nical s us pici o n is f or a n i nfecti o n 
f or w hic h c ult ures are u nli kel y t o be p ositi ve wit hi n 4 8 h o urs (e g, f u n gal i nfecti o n), a de q uate 
ti me m ust be all o we d f or c ult ures t o bec o me p ositi ve.


A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 4 8
0 7 J u ne 2 0 2 2
After t he a b o ve criteria are met, t he n t he s u bject ca n pr ocee d wit h a d mi nistrati o n of a xica bta ge ne 
cil ole ucel.
If t he a xica bta ge ne cil ole ucel i nf usi o n is dela ye d > 2 wee ks, pr ot oc ol g ui deli nes s h o ul d be 
f oll o we d re gar di n g t he nee d t o re peat c o n diti o ni n g c he m ot hera p y. 
7. 8. 3. 1. 1.  
Re q uire me nts t o W or k u p P ote ntial I nfecti o us a n d/ or I nfla m mat or y States
I n t he a bse nce of a n i de ntifie d s o urce of i nfecti o n (e g, li ne i nfecti o n, p ne u m o nia o n c hest X-ra y), 
t he mi ni m u m w or k u p t o be perf or me d pri or t o a d mi nistrati o n of c o n diti o ni n g c he m ot hera p y 
a n d/ or a xica bta ge ne cil ole ucel c o nsists of:

Call Kite me dical m o nit or

I nfecti o us disease ser vice c o ns ult (if a vaila ble)

C T i ma gi n g of t he c hest, a b d o me n, a n d pel vis wit h I V c o ntrast.  If t here is a me dical 
c o ntrai n dicati o n t o c o ntrast, t he n n o n-c o ntrast C T is all o we d.  

T he f oll o wi n g m ust be perf or me d ( pri or t o t he i nitiati o n of a nti micr o bials if cli nicall y 
feasi ble): 

Bl o o d c ult ures (aer o bic a n d a naer o bic x 2 b ottles eac h) a n d uri nar y a nal ysis a n d uri ne 
c ult ure; dee p/i n d uce d s p ut u m c ult ure if cli nicall y i n dicate d

All i n d welli n g li nes, s uc h as ce ntral ve n o us cat heters, s h o ul d be e xa mi ne d f or a n y si g ns 
of i nfecti o n, a n d a d diti o nal c ult ures s h o ul d be dra w n fr o m t he li ne.

Nas o p har y n geal-t hr oat ( N P T) s wa b or e q ui vale nt assa y f or viral i nfecti o n, s uc h as 
i nfl ue nza A/ B (i ncl u di n g H 1 N 1), parai nfl ue nza 1/ 2/ 3, a de n o vir us, res pirat or y s y nc ytial 
vir us, c or o na vir us, meta p ne u m o vir us

C ollecti o n of f u n gal c ult ures a n d mar kers as a p pr o priate (e g, galact o ma n na n, F u n gitell ® )

C ollecti o n of a p pr o priate ser u m viral st u dies (e g, c yt o me gal o vir us [C M V ])

If a ce ntral ner v o us s yste m pr ocess is s us pecte d, a p pr o priate brai n i ma gi n g a n d s u bse q ue nt 
l u m bar p u nct ure wit h c yt ol o g y, c ult ure, Gra m stai n, a n d viral P C R s h o ul d be perf or me d.  

A n y a d diti o nal si g n or s y m pt o m- directe d i n vesti gati o n s h o ul d be perf or me d as cli nicall y 
i n dicate d.
Pri or t o pr ocee di n g wit h c o n diti o ni n g c he m ot hera p y a n d/ or a xica bta ge ne cil ole ucel i nf usi o n, t he 
a b o ve w or k u p m ust n ot s u g gest t he prese nce of a n acti ve i nfecti o n, a n d all re q uire me nts f or 
c o n diti o ni n g c he m ot hera p y a n d/ or a xica bta ge ne cil ole ucel i nf usi o n m ust be satisfie d.  


A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 4 9
0 7 J u ne 2 0 2 2
If t he a xica bta ge ne cil ole ucel i nf usi o n is dela ye d > 2 wee ks f oll o wi n g c o n diti o ni n g 
c he m ot hera p y, pr ot oc ol g ui deli nes s h o ul d be f oll o we d re gar di n g t he nee d t o re peat c o n diti o ni n g 
c he m ot hera p y. 
If t he a b o ve w or k u p was tri g gere d d ue t o C R P > 1 0 0 m g/ L, C R P s h o ul d be re peate d, a n d if C R P 
c o nti n ues t o i ncrease si g nifica ntl y, e val uati o n s h o ul d be perf or me d f or a n y ot her p ote ntial 
i nfecti o us or i nfla m mat or y c o n diti o n n ot pre vi o usl y e val uate d.  
7. 8. 3. 2.   
A xica bta ge ne Cil ole ucel Pre me dicati o n D osi n g
T he f oll o wi n g pre a xica bta ge ne cil ole ucel i nf usi o n me dicati o n s h o ul d be a d mi nistere d 
a p pr o xi matel y 1 h o ur pri or t o i nf usi o n.  Alter nati ves t o t he rec o m me n dati o ns bel o w s h o ul d be 
disc usse d wit h t he me dical m o nit or.

Aceta mi n o p he n 6 5 0 m g P O or e q ui vale nt 

Di p he n h y dra mi ne 1 2. 5 m g a d mi nistere d eit her orall y or via I V or e q ui vale nt
7. 8. 3. 3.   
A xica bta ge ne Cil ole ucel A d mi nistrati o n Da y 0
All s u bjects will recei ve a xica bta ge ne cil ole ucel i nf usi o n at a healt hcare facilit y, f oll o we d b y 
dail y m o nit ori n g at a healt hcare facilit y f or at least 7 da ys t o m o nit or f or si g ns a n d s y m pt o ms of 
C R S a n d N Es.  Alter nati vel y, s u bjects ma y be h os pitalize d t o recei ve t heir a xica bta ge ne 
cil ole ucel i nf usi o n a n d be o bser ve d f or C R S a n d N Es i n t he h os pital setti n g, if dee me d 
a p pr o priate b y t he i n vesti gat or.
If s u bjects are h os pitalize d, s u bjects s h o ul d n ot be disc har ge d fr o m t he h os pital u ntil all 
a xica bta ge ne cil ole ucel-relate d n o n- he mat ol o gical t o xicities res ol ve t o ≤ Gra de 1 or ret ur n t o 
baseli ne.  S u bjects ma y be disc har ge d wit h n o n-critical a n d cli nicall y sta ble or i m pr o vi n g 
t o xicities (e g, re nal i ns ufficie nc y), e ve n if > Gra de 1, if dee me d a p pr o priate b y t he i n vesti gat or.  
S u bjects s h o ul d re mai n i n a h os pital f or o n g oi n g a xica bta ge ne cil ole ucel-relate d fe ver, 
h y p ote nsi o n, h y p o xia, or o n g oi n g N Es > Gra de 1 or if dee me d necessar y b y t he i n vesti gat or.
S u bjects s h o ul d be i nstr ucte d t o re mai n wit hi n pr o xi mit y of t he cli nical st u d y site f or at least 
4 wee ks f oll o wi n g a xica bta ge ne cil ole ucel i nf usi o n.  S u bjects a n d t heir fa mil y 
me m bers/care gi vers s h o ul d be e d ucate d o n p ote ntial C R S a n d ne ur ol o gic s y m pt o ms, s uc h as 
fe ver, d ys p nea, c o nf usi o n, a p hasia, d ys p hasia, s o m n ole nce, e nce p hal o pat h y, ata xia, or tre m or.  
S u bjects or t heir fa mil y me m bers/care gi vers s h o ul d be i nstr ucte d t o i m me diatel y c o ntact t he 
treati n g i n vesti gat or or see k i m me diate me dical atte nti o n if a n y of t hese s y m pt o ms de vel o p.
Refer t o t he S O A f or a listi n g of st u d y pr oce d ures t o be c o m plete d d uri n g t he a xica bta ge ne 
cil ole ucel treat me nt peri o d.


A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 5 0
0 7 J u ne 2 0 2 2
Ce ntral ve n o us access, s uc h as a p ort or a peri p herall y i nserte d ce ntral cat heter, is re q uire d f or 
t he a d mi nistrati o n of a xica bta ge ne cil ole ucel.  Cat heter care, per i nstit uti o nal g ui deli nes, s h o ul d 
be f oll o we d.  Materials a n d i nstr ucti o ns f or t he t ha wi n g, ti mi n g, a n d a d mi nisteri n g of 
a xica bta ge ne cil ole ucel are o utli ne d i n t he I P M.  Vital si g ns s h o ul d be meas ure d d uri n g a n d after 
a xica bta ge ne cil ole ucel treat me nt (see Secti o n 7. 5 ).  T he I P M m ust be re vie we d pri or t o 
a d mi nistrati o n of a xica bta ge ne cil ole ucel.
Researc h sites s h o ul d f oll o w i nstit uti o nal g ui deli nes f or t he i nf usi o n of cell pr o d ucts.  
7. 8. 3. 4.   
Ut o mil u ma b A d mi nistrati o n
Ut o mil u ma b treat me nt c o nsists of a n I V i nf usi o n gi ve n e ver y 4 wee ks f or 6 m o nt hs or u ntil 
disease pr o gressi o n, w hic he ver c o mes first. I n t he P hase 1 p orti o n of t he st u d y, t he d oses of 
ut o mil u ma b a d mi nistere d will be 1 0 m g, 3 0 m g, 1 0 0 m g, 2 0 0 m g, a n d 4 0 0 m g be gi n ni n g at 
eit her Da y 1 or Da y 2 1 after a xica bta ge ne cil ole ucel i nf usi o n.  T he S R T will ma ke 
rec o m me n dati o ns o n f urt her st u d y c o n d uct of P hase 1 a n d pr o gressi o n t o P hase 2 as de picte d i n 
Fi g ure 5  a n d o utli ne d i n Secti o n 9. 1 0 .
S u bjects will re mai n i n cli nic f or p ost- ut o mil u ma b o bser vati o n f or at least 2 h o urs after t he e n d 
of t he first i nf usi o n of ut o mil u ma b a n d f or at least 1 h o ur after t he e n d of s u bse q ue nt i nf usi o ns.
F or m ore detaile d i nf or mati o n re gar di n g a d mi nistrati o n, refer t o t he I P M a n d I B.
7. 8. 3. 5.   
A xica bta ge ne Cil ole ucel Retreat me nt 
S u bjects w h o ac hie ve a P R or C R a n d s u bse q ue ntl y e x perie nce disease pr o gressi o n ma y ha ve a n 
o pti o n t o recei ve a sec o n d c o urse of c o n diti o ni n g c he m ot hera p y a n d a xica bta ge ne cil ole ucel 
w hile e nr olle d i n t his st u d y. T he sec o n d c o urse of treat me nt m ust be gi ve n wit hi n 2 4 m o nt hs of 
t he i nitial a xica bta ge ne cil ole ucel i nf usi o n.
I n eit her case, t he f oll o wi n g criteria m ust be met i n or der t o be c o nsi dere d f or a re peat c o urse of 
t hera p y:

S u bject ha d a P R or C R at t he first disease assess me nt at Wee k 4 b ut s u bse q ue ntl y 
e x perie nce d pr o gressi o n of disease at a later ti me. 

C D 1 9 t u m or e x pressi o n c o nfir me d l ocall y b y bi o ps y after disease pr o gressi o n a n d pri or t o 
retreat me nt.

S u bject c o nti n ues t o meet t he ori gi nal st u d y eli gi bilit y criteria wit h t he e xce pti o n of pri or 
a xica bta ge ne cil ole ucel use i n t his st u d y; scree ni n g assess me nts a n d pr oce d ures s h o ul d be 
re peate d if cli nicall y i n dicate d (e g, M RI, E C H O)

S u bject has n ot recei ve d s u bse q ue nt t hera p y f or t he treat me nt of l y m p h o ma.

S u bject di d n ot e x perie nce a life-t hreate ni n g ( 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 ] Gra de ≥ 4) t o xicit y relate d t o a xica bta ge ne cil ole ucel d uri n g t he ori gi nal 
c o urse of treat me nt.


A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 5 1
0 7 J u ne 2 0 2 2

T o xicities relate d t o c o n diti o ni n g c he m ot hera p y (c ycl o p h os p ha mi de a n d fl u dara bi ne), wit h 
t he e xce pti o n of al o pecia, ha ve res ol ve d t o ≤ Gra de 1 or ret ur ne d t o baseli ne pri or t o 
retreat me nt.

S u bject d oes n ot ha ve k n o w n a xica bta ge ne cil ole ucel ne utralizi n g a nti b o dies (e xce pti o n: if a 
n o n- ne utralizi n g a xica bta ge ne cil ole ucel a nti b o d y de vel o ps, s u bject ma y be retreate d if t he 
ori gi nal st u d y eli gi bilit y criteria are met).

S u bject meets a xica bta ge ne cil ole ucel pretreat me nt criteria. 
Sites are str o n gl y e nc o ura ge d t o c ollect a bi o ps y c o nfir mi n g disease pr o gressi o n a n d C D 1 9 
e x pressi o n a n d s u b mit t he bi o psie d tiss ue t o t he ce ntral la b orat or y bef ore i nitiati n g retreat me nt.
T he decisi o n t o retreat s h o ul d be ma de i n c o ns ultati o n wit h t he s p o ns or’s me dical m o nit or.  I n 
a d diti o n, bef ore perf or mi n g a n y st u d y-relate d pr oce d ures or treat me nt, it is necessar y t o 
1) disc uss t he ris ks a n d be nefits of retreat me nt wit h t he s u bject, a n d 2) c o nfir m wit h t he s u bject 
h o w t he sec o n d d ose will be ma n ufact ure d.  T he sec o n d d ose c o ul d be ma n ufact ure d usi n g 
e xisti n g cr y o preser ve d P B M Cs.  Alter nati vel y, t he s u bject ma y nee d t o u n der g o a sec o n d 
le u ka p heresis a n d s h o ul d be i nf or me d of t his p ossi bilit y.  T hese c o n versati o ns s h o ul d als o be 
rec or de d i n t he s u bject’s s o urce d oc u me nt.  
A ma xi m u m of 1 retreat me nt c o urse ma y occ ur per s u bject.  
All o wa nce f or retreat me nt is base d o n cli nical e x perie nce i n Z U M A- 1, w here a t otal of 
1 0 s u bjects were retreate d wit h a xica bta ge ne cil ole ucel ( 1 s u bject i n P hase 1 a n d 9 s u bjects i n 
P hase 2).  O verall, 6 of 1 0 retreate d s u bjects ha d a P R or C R t o t he retreat me nt.  T he s u bject i n 
P hase 1 ac hie ve d a P R at M o nt h 1 after retreat me nt.  A m o n g t he 9 s u bjects retreate d i n P hase 2, 
fi ve s u bjects res p o n de d ( 2 C R a n d 3 P R) at M o nt h 1.  A nal ysis of d urati o n of retreat me nt 
res p o nse ( D O R R) a m o n g t he retreate d s u bjects i n P hase 2 s h o we d a me dia n D O R R of 
3. 5 m o nt hs as of t he data c ut off date.  Res ults were i de ntical i n a se nsiti vit y a nal ysis t hat 
e xcl u de d res p o nses i n 4 s u bjects w h o u n der we nt A S C T f oll o wi n g retreat me nt.    
After a s u bject is dee me d eli gi ble f or retreat me nt a n d t he mea ns b y w hic h t he sec o n d d ose of 
a xica bta ge ne cil ole ucel has bee n c o nfir me d ( w hic h will i ncl u de deter mi ni n g w het her a sec o n d 
le u ka p heresis is re q uire d), t he s u bject will f oll o w t he sa me st u d y pr oce d ure re q uire me nts t hat 
were f oll o we d d uri n g his or her i nitial c o urse of treat me nt wit h t he e xce pti o n of ut o mil u ma b 
i nf usi o ns.
7. 9.  
L a b or at or y
7. 9. 1.  
L oc al L a b A n al ysis
Assess me nts liste d i n Ta ble 4  will be perf or med at t he l ocal la b orat or y at t he ti me p oi nts 
i n dicate d i n t he S O A.  A d diti o nal sa m ples (e g, bl o o d, uri ne, C S F, tiss ue) ma y be c ollecte d as 
nee de d f or f urt her safet y testi n g.


A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 5 2
0 7 J u ne 2 0 2 2
T a ble 4.
L oc al L a b or at or y P ar a meters
Ser u m C he mistries
He m at ol o g y
Ot her
Al b u mi n
A L T/ G P T
A L P
A S T/ G O P
Bicar b o nate t otal
Bilir u bi n direct
Bilir u bi n t otal
B U N or urea a 
Calci u m t otal
C hl ori de
Creati ni ne
Creati ni ne cleara nce
Gl uc ose
I n or ga nic P h os p h or us
L D H
Ma g nesi u m t otal
P otassi u m
S o di u m
Uric aci d b 
C B C wit h differe ntial b 
C R P
Ferriti n
Pre g na nc y test c
A b bre viati o ns:  A L P, al kali ne p h os p hatase; A L T, ala ni ne a mi n otra nsferase; A S T, as partate a mi n otra nsferase; B U N, bl o o d urea 
nitr o ge n; C B C, c o m plete bl o o d c o u nt; C R P, C-reacti ve pr otei n; G O P, ser u m gl uta mic- o xal oacetic tra nsa mi nase; G P T, ser u m 
gl uta mic- p yr u vic tra nsa mi nase; L D H, lactate de h y dr o ge nase.
a  
If B U N test ca n n ot be a nal yze d b y t he l ocal la b, urea s h o ul d be a nal yze d. 
b  
Per i nstit uti o nal g ui deli nes.
c   
W o me n of c hil d beari n g p ote ntial o nl y.
7. 9. 2.   
Ce ntr al L a b or at or y A n al yses
Bi o mar ker a nal ysis will be perf or me d o n bl o o d, C S F, a n d t u m or sa m ples t o e val uate P K a n d 
p har mac o d y na mic mar kers f or a xica bta ge ne cil ole ucel i n c o nj u ncti o n wit h ut o mil u ma b. 
Pr o g n ostic mar kers f or a g gressi ve N H L a n d relate d t o t he t u m or i m m u ne e n vir o n me nt ma y als o 
be e val uate d.
Cli nical bi os peci me ns (e g, t u m or tiss ue, b o ne marr o w, c o m plete bl o o d, C S F, or ot her b o dil y 
fl ui ds) will be se nt fr o m cli nical st u d y ce nters t o t he ce ntral la b orat or y f or sa m ple pr ocessi n g, 
accessi o ni n g, a n d distri b uti o n t o s pecialt y la b orat ories or Kite. Sa m ples will be o btai ne d at t he 
ti mes i n dicate d i n t he S O A.  A d diti o nal sa m ples (e g, c o m plete bl o o d, uri ne, C S F, tiss ue) ma y be 
c ollecte d as nee de d f or f urt her safet y testi n g.
A p heresis a n d pr o d uct retai ns are pr o vi de d t o t he ce ntral la b or are st ore d at a Kite s p o ns ore d 
ce ntral la b orat or y.  C o m plete i nstr ucti o ns re gar di n g sa m ple s u b missi o n t o ce ntral la b orat ories are 
pr o vi de d i n t he Ce ntral La b orat or y Ma n ual.






A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 5 5
0 7 J u ne 2 0 2 2

Ser u m sa m ples will be assa ye d f or ut o mil u ma b c o nce ntrati o ns wit h t he use of a vali date d 
a nal ytical met h o d.  P K sa m ples are t o be c ollecte d at t he f oll o wi n g ti me p oi nts (i n relati o n t o 
ut o mil u ma b i nf usi o n): 

I nf usi o n 1: pri or t o i nf usi o n; e n d of i nf usi o n; a n d o n Da ys 3, 7, 1 4 ( Wee k 2), a n d 
2 1 ( Wee k 3) f oll o wi n g ut o mil u ma b i nf usi o n

I nf usi o ns 2 a n d 4: pri or t o i nf usi o n; e n d of i nf usi o n; a n d 2 da ys f oll o wi n g ut o mil u ma b 
i nf usi o n 2

At t he p ost-treat me nt f oll o w- u p visit 
7. 9. 2. 3. 2.  
Sa m ple C ollecti o ns f or S u bjects wit h Ut o mil u ma b A d mi nistere d Starti n g o n 
Da y 2 1

A D A sa m ples are t o be c ollecte d at t he f oll o wi n g ti me p oi nts (i n relati o n t o ut o mil u ma b 
i nf usi o n):

I nf usi o n 1 a n d 2: pri or t o i nf usi o n 

At t he p ost-treat me nt f oll o w- u p visit

P K sa m ples are t o be c ollecte d at t he f oll o wi n g ti me p oi nts (i n relati o n t o ut o mil u ma b 
i nf usi o n): 

I nf usi o n 1: pri or t o i nf usi o n, e n d of i nf usi o n, a n d 2 a n d 8 ( Wee k 4) da ys f oll o wi n g t he 
ut o mil u ma b i nf usi o n

I nf usi o ns 2 a n d 4: pri or t o i nf usi o n; e n d of i nf usi o n; a n d 2 da ys f oll o wi n g ut o mil u ma b 
i nf usi o n 2

At t he p ost-treat me nt f oll o w- u p visit 
7. 9. 2. 3. 3.  
Sa m ple C ollecti o ns f or S u bjects i n P hase 2

Ser u m sa m ples will be assa ye d f or t he prese nce of A D As t o ut o mil u ma b wit h t he use of 
vali date d i m m u n oassa ys.  A D A sa m ples are t o be c ollecte d at t he f oll o wi n g ti me p oi nts 
(i n relati o n t o ut o mil u ma b i nf usi o n):

I nf usi o n 1 a n d 2: pri or t o i nf usi o n

At t he p ost-treat me nt f oll o w- u p visit

Ser u m sa m ples will be assa ye d f or ut o mil u ma b c o nce ntrati o ns wit h t he use of a vali date d 
a nal ytical met h o d.  P K sa m ples are t o be c ollecte d at t he f oll o wi n g ti me p oi nts (i n relati o n t o 
ut o mil u ma b i nf usi o n): 

I nf usi o n 1, 2, a n d 4: pri or t o a n d f oll o wi n g t he e n d of i nf usi o n


A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 5 6
0 7 J u ne 2 0 2 2
7. 9. 2. 4.  
Pr o d uct C haracteristics
I n a d diti o n, baseli ne le u ka p heresis a n d fi nal a xica bta ge ne cil ole ucel sa m ples will be ba n ke d a n d 
ma y be a nal yze d b y i m m u n o p he n ot y pi n g, q ua ntitati ve p ol y merase c hai n reacti o n  (q P C R), a n d/ or 
ge ne e x pressi o n pr ofili n g.  
 
Sa m ples of a p heresis material or fi nal pr o d uct will be retai ne d a n d teste d b y t he s p o ns or or 
s pecialt y la b orat or y f or t he p ur p ose of u n dersta n di n g t he mec ha nis m of acti o n a n d safet y pr ofile 
of a xica bta ge ne cil ole ucel. 
7. 9. 2. 5.  
R C R Testi n g
A xica bta ge ne cil ole ucel c o m prises T cells tra ns d uce d wit h a γ-retr o viral vect or; he nce, t here is a 
t he oretical ris k f or R C R de vel o pi n g i n e x p ose d s u bjects.  R C R testi n g will occ ur at t he f oll o wi n g 
ti me p oi nts: baseli ne, Wee k 1 2, Wee k 2 0/ 2 3 ( de pe n di n g o n c o h ort), a n d M o nt h 1 2.  T hereafter, 
sa m ples will be c ollecte d yearl y a n d hel d f or u p t o 1 5 years.  If a s u bject tests p ositi ve f or R C R 
at a n y ti me p oi nt wit hi n t he first year, sa m ples will c o nti n ue t o be c ollecte d a n d teste d yearl y f or 
u p t o 1 5 years or as cli nicall y i n dicate d.  A d diti o nal i nf or mati o n is pr o vi de d i n t he a xica bta ge ne 
cil ole ucel I B. 
7. 1 0.  
P ost-tre at me nt Assess me nt Peri o d
After c o m pleti n g st u d y treat me nt, all s u bjects will ret ur n t o t he cli nic f or a p ost-treat me nt 
f oll o w- u p visit. 
If a s u bject d oes n ot res p o n d t o treat me nt at a n y ti me d uri n g t he st u d y, t he n t he s u bject will 
c o nti n ue t o u n der g o f oll o w- u p pr oce d ures a n d t he n be f oll o we d f or s ur vi val a n d disease 
o utc o mes i n t he L T F U p orti o n of t he st u d y.  
Refer t o t he S O As f or a listi n g of st u d y pr oce d ures a n d disease assess me nts t o be c o m plete d 
d uri n g t he p ost-treat me nt f oll o w- u p peri o d.
7. 1 1.  
L o n g-ter m F oll o w- u p Peri o d
All e nr olle d s u bjects will be f oll o we d i n t he L T F U peri o d f or safet y a nal ysis a n d s ur vi val a n d 
disease stat us, if a p plica ble.  S u bjects will be gi n t he L T F U peri o d after c o m pleti o n of t he 
p ost-treat me nt f oll o w- u p visit ( w het her t he y ha ve res p o n de d t o treat me nt or we nt strai g ht t o t he 
p ost-treat me nt f oll o w- u p visit d ue t o disease pr o gressi o n).  S u bjects w h o recei ve d a n i nf usi o n of 
a xica bta ge ne cil ole ucel will be gi ve n t he o p p ort u nit y t o tra nsiti o n t o a se parate L T F U st u d y, K T-
U S- 9 8 2- 5 9 6 8, after pr o vi di n g si g ne d i nf or me d c o nse nt. S u bjects w h o di d n ot res p o n d t o 
treat me nt will c o nti n ue t o be f oll o we d f or disease assess me nts (if pr o gressi o n was n ot 
d oc u me nte d), s u bse q ue nt a nti-ca ncer t hera p y, a n d s ur vi val. 
S u bjects ma y als o be c o ntacte d b y tele p h o ne t o c o nfir m s ur vi val stat us a n d s u bse q ue nt a nti-
ca ncer t hera p y use.  If t he s u bject fails t o ret ur n t o t he cli nic f or a sc he d ule d pr ot oc ol-s pecific 
visit, sites will nee d t o ma ke 2 atte m pts, usi n g b ot h t he tele p h o ne a n d eit her mail or e mail t o 
C CI


A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 5 7
0 7 J u ne 2 0 2 2
c o ntact t he s u bject.  Sites m ust d oc u me nt b ot h atte m pts t o c o ntact t he s u bject.  If a s u bject d oes 
n ot res p o n d wit hi n 1 m o nt h after t he sec o n d c o ntact, t he n t he s u bject will be c o nsi dere d l ost t o 
f oll o w- u p, a n d n o a d diti o nal c o ntact will be re q uire d. 
Refer t o t he S O A f or a listi n g of st u d y pr oce d ures a n d disease assess me nts t o be c o m plete d 
d uri n g t he L T F U peri o d.
S u bjects w h o are e nr olle d, b ut di d n ot recei ve t he I P(s) will be f oll o we d o nl y u ntil t he e n d of t his 
st u d y a n d will u n der g o t he f oll o wi n g assess me nts at t he ti me p oi nts o utli ne d i n t he S O A: 

S u bse q ue nt t hera p y f or t he treat me nt of N H L 

S ur vi val stat us 

Disease assess me nt per pr ot oc ol

A E/ S A Es per Secti o n 9


A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 5 8
0 7 J u ne 2 0 2 2
T a ble 5.
Sc he d ule of Assess me nts C o h ort 1, 2, 3, 4, 5 a n d P h ase 2 ( as a p plic a ble) Ut o mil u m a b A d mi nistr ati o n 
St arti n g o n D a y 1
Pr oce d ures
Scree ni n g
E nr oll me nt/
Le u k a p heresis
C o n diti o ni n g 
C he m ot her a p y
I P A d mi nistr ati o n Peri o d
P ost-
tre at me nt 
F oll o w- u p
D a y
Wit hi n 
2 8 d a ys of 
e nr oll me nt 
  
- 5 
- 4 
- 3 
0 
1 
2- 7
Wee k 
2
( 1 4 ± 2 
d a ys)
Wee k 
3
( 2 1 ± 2 
d a ys)
Wee k 
4
( 2 8 ± 3 
d a ys)
Wee k 
8
( ± 1 
wee k)
Wee k 
1 2
( ± 1 
wee k)
Wee k 
1 6
( ± 1 
wee k)
Wee k 
2 0
( ± 1 
wee k)
3 0 d a ys
( ± 1 wee k) 
f oll o wi n g l ast 
ut o mil u m a b 
i nf usi o n 
Ut o mil u m a b I nf usi o n #
# 1
# 2 
# 3 
# 4 
# 5 
# 6
Me dical Hist or y 
X
E C O G Perf or ma nce Stat us
X
1 2-lea d E C G
X
E C H O
X
Brai n M RI
X
Ne ur ol o gical Assess me nt 
X
X 
Q O D 1
P E T- C T/ Disease Assess me nt 2
X
X
X
X
Arc hi val/ Fres h T u m or 3
X
D 5
P ost- Pr o gressi o n Bi o ps y 4
X
P h ysical E xa m
X
X
X 
X 
X 
X
X 
X 
X 
X 
X
X
Vital Si g ns ( B P, H R, O 2 
sat urati o n, R R, Te m perat ure) 5
X
X 
X 
X 
X 
X 
X 1 5
X 
X 
X 
X 1 5
X 1 5
X 1 5
X 1 5
X 1 5
X
Wei g ht (pl us Hei g ht at 
scree ni n g)
X
X
Pre g na nc y Test 6 (ser u m or 
uri ne)
X
X
X
L u m bar P u nct ure 7
X
D 5
X
Bl o o d Dra w f or C he mistr y 
Pa nel
X
X 
X 
X 
X 
X 
X 
X 
X
X 
X 
X 
X 
X
X


A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 5 9
0 7 J u ne 2 0 2 2
Pr oce d ures
Scree ni n g
E nr oll me nt/
Le u k a p heresis
C o n diti o ni n g 
C he m ot her a p y
I P A d mi nistr ati o n Peri o d
P ost-
tre at me nt 
F oll o w- u p
D a y
Wit hi n 
2 8 d a ys of 
e nr oll ment 
  
- 5 
- 4 
- 3 
0 
1 
2- 7
Wee k 
2
( 1 4 ± 2 
d a ys)
Wee k 
3
( 2 1 ± 2 
d a ys)
Wee k 
4
( 2 8 ± 3 
d a ys)
Wee k 
8
( ± 1 
wee k)
Wee k 
1 2
( ± 1 
wee k)
Wee k 
1 6
( ± 1 
wee k)
Wee k 
2 0
( ± 1 
wee k)
3 0  d a ys
( ± 1 wee k) 
f oll o wi n g l ast 
ut o mil u m a b 
i nf usi o n 
Ut o mil u m a b I nf u si o n #
# 1
# 2 
# 3 
# 4 
# 5 
# 6
Bl o o d Dra w f or C B C 
w/ Differe ntial
X
X 
X 
X 
X 
X 
X 
X 
X
X 
X 
X 
X 
X
X
Bl o o d Dra w f or C R P
X
Bl o o d Dra w f or C yt o ki nes 8, 9
X
X 
X 
D 3, D 7, D 1 4 
X 
X 9
X 9
X 9
Ce ntral C B C w/ Differe ntial 
a n d P B M C 8, 1 0
X
X 
X
D 3, D 7, D 1 0, 
D 1 4
X 
X 1 0
X 1 0
X 
X 
X
X
Bl o o d Dra w f or Ut o mil u ma b 
P K 1 1  
X
D 3, 
D 7
X 
X 
X
X
X
Bl o o d Dra w f or Ut o mil u ma b 
A D A 1 2
X
X
X
Le u ka p heresis 
X
C ycl o p h os p ha mi de/ Fl u dara bi ne
X 
X 
X
A xica bta ge ne Cil ole ucel 
I nf usi o n I V
X
Ut o mil u ma b I nf usi o n I V 1 3
D 1
D 2 9 
D 5 7 
D 8 5 
D 1 1 3 
D 1 4 1
A d verse E ve nts/ C o nc o mita nt 
Me dicati o n
1 4
X
A b bre viati o ns:  C B C, c o m plete bl o o d c o u nt; P B M C, peri p heral bl o o d m o n o n uclear cell; P E T- C T, p ositr o n e missi o n t o m o gra p h y-c o m p ute d t o m o gra p h y; S A E, seri o us a d verse 
e ve nt; I P, i n vesti gati o nal pr o d uct; D, da y; I V, i ntra ve n o us; A D A, a nti- dr u g a nti b o d y; C R P, C-reacti ve pr otei n; P K, p har mac o ki netics; E C O G, Easter n C o o perati ve O nc ol o g y 
Gr o u p; E C G, electr ocar di o gra m; E C H O, ec h ocar di o gra m; M RI, ma g netic res o na nce i ma gi n g; B P, bl o o d press ure; H R, heart rate; R R, res pirati o n rate; Q O D, < defi ne acr o n y m >. 
1   
Assess me nts o n Da y 1, e ver y ot her da y u ntil Da y 7, a n d as cli nicall y i n dicate d t hereafter. 
2  
P E T- C T ( Nec k- C hest- A b d o me n- Pel vis)/ Disease Assess me nt: P E T- C T perf or me d f oll o wi n g t he s u bject’s last li ne of t hera p y a n d pri or t o si g ni n g t he i nf or me d c o nse nt ma y be 
use d f or c o nfir mati o n of eli gi bilit y if wit hi n 2 8 da ys pri or t o e nr oll me nt a n d n o ot her a nti-ca ncer treat me nt has bee n a d mi nistere d.  If P E T- C T is perf or me d > 2 8 da ys pri or t o 
e nr oll me nt or if s u bject recei ves a n y a nti-ca ncer t hera p y bet wee n scree ni n g a n d c o n diti o ni n g c he m ot hera p y, t he P E T- C T sca n m ust be re peate d t o esta blis h a ne w baseli ne. 
P E T- C T will als o be perf or me d at Wee k 4, Wee k 1 2, a n d at t he p ost-treat me nt f oll o w- u p visit.




A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 6 1
0 7 J u ne 2 0 2 2
T a ble 6.
Sc he d ule of Assess me nts C o h ort 1 A, 2 A, 3 A, 4 A, 5 A a n d P h ase 2 ( as a p plic a ble)
Ut o mil u m a b A d mi nistr ati o n St arti n g o n D a y 2 1
Pr oce d ures
Scree ni n g
E nr oll me nt/
Le u k a p heresis
C o n diti o ni n g 
C he m ot her a p y
I P A d mi nistr ati o n Peri o d
P ost-
tre at me nt 
F oll o w- u p
D a y
Wit hi n 2 8 
d a ys of 
e nr oll me nt 
  
- 5 
- 4 
- 3 
0 
1- 7
Week 
2
(14 ± 2 
days)
Week 
3
(21 ± 
2 
days)
Week 
4
(28 ± 
3 
days)
Week 
7
( ±1 
wee k)
Week 
11
( ± 1 
wee k)
Week 
1214
( ± 1 
wee k)
Week 
15
( ± 1 
wee k)
Week 
19 
( ± 1 
wee k)
Week 
23 
( ± 1 
wee k)
3 0 d a ys 
( ± 1 wee k) 
f oll o wi n g l ast 
ut o mil u m a b 
i nf usi o n 
Ut o mil u m a b I nf usi o n #
# 1
# 2 
# 3
# 4 
# 5 
# 6
Me dical Hist or y 
X
E C O G Perf or ma nce Stat us
X
1 2-lea d E C G
X
E C H O
X
Brai n M RI
X
Ne ur ol o gical Assess me nt 
X
X 
Q O D 1
P E T- C T/ Disease Assess me nt 2
X
X
X
X
Arc hi val/ Fres h T u m or 3
X
D 5
P ost- Pr o gressi o n Bi o ps y 4
X
P h ysical E xa m
X
X
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X
X
Vital Si g ns ( B P, H R, O 2 
sat urati o n, R R, Te m perat ure) 5
X
X 
X 
X 
X 
X 
X 
X 
X 1 6
X 
X 1 6
X 1 6
X 
X 1 6
X 1 6
X 1 6
X
Wei g ht (pl us Hei g ht at 
scree ni n g)
X
X
Pre g na nc y Test 6 (ser u m or 
uri ne)
X
X
X
L u m bar P u nct ure 7
X
D 5
X
Bl o o d Dra w f or C he mistr y Pa nel 
X
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X
X
Bl o o d Dra w f or C B C 
w/ Differe ntial
X
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X
X


A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 6 2
0 7 J u ne 2 0 2 2
Pr oce d ures
Scree ni n g
E nr oll me nt/
Le u k a p heresis
C o n diti o ni n g 
C he m ot her a p y
I P A d mi nistr ati o n Peri o d
P ost-
tre at me nt 
F oll o w- u p
D a y
W it hi n 2 8 
d a ys of 
e nr oll me nt 
  
- 5 
- 4 
- 3 
0 
1- 7
W eek 
2
(14 ± 2 
days)
W eek 
3
(21 ± 
2 
days)
W eek 
4
(28 ± 
3 
days)
W eek 
7
( ±1 
wee k)
W eek 
11
( ± 1 
wee k)
W eek 
1214
( ± 1 
wee k)
W eek 
15
( ± 1 
wee k)
W eek 
19 
( ± 1 
wee k)
W eek 
23 
( ± 1 
wee k)
3 0 d a ys 
( ± 1 wee k) 
f oll o wi n g l ast 
ut o mil u m a b 
i nf usi o n 
Ut o mil u m a b I nf u si o n #
# 1
# 2 
# 3
# 4 
# 5 
# 6
Bl o o d Dra w f or C R P
X
Bl o o d Dra w f or C yt o ki nes 8, 9
X
X
D 1, D 3, D 7, 
D 1 4
X 9
X 
X 9
X 9
X 9
Ce ntral C B C w/ Differe ntial a n d 
P B M C 8, 1 0
X
X
D 1, D 3, D 7, 
D 1 0, D 1 4
X 1 0
X 
X 1 0
X 1 0
X 
X 
X 
X
X
Bl o o d Dra w f or Ut o mil u ma b 
P K 1 1  
X 1 1
X 
X
X
X
Bl o o d Dra w f or Ut o mil u ma b 
A D A 1 2
X
X
X
Le u ka p heresis 
X
C ycl o p h os p ha mi de/ Fl u dara bi ne
X 
X 
X
A xica bta ge ne Cil ole ucel 
I nf usi o n I V
X
Ut o mil u ma b I nf usi o n I V 1 3
D 2 1
D 4 9 
D 7 7
D 1 0 5 
D 1 3 3 
D 1 6 1
A d verse E ve nts/ C o nc o mita nt 
Me dicati o n
1 5
X
A b bre viati o ns:  C B C, c o m plete bl o o d c o u nt; P B M C, peri p heral bl o o d m o n o n uclear cell; P E T- C T, p ositr o n e missi o n t o m o gra p h y c o m p ute d t o m o gra p h y; S A E, seri o us a d verse 
e ve nt; I P, i n vesti gati o nal pr o d uct; D, da y; I V, i ntra ve n o us; A D A, a nti- dr u g a nti b o d y; C R P, C-reacti ve pr otei n; P K, p har mac o ki netics; E C O G, Easter n C o o perati ve O nc ol o g y 
Gr o u p; E C G, electr ocar di o gra m; E C H O, ec h ocar di o gra m; M RI, ma g netic res o na nce i ma gi n g; B P, bl o o d press ure; H R, heart rate; R R, res pirati o n rate; Q O D, < defi ne acr o n y m >.
1   
Assess me nts o n Da y 1, e ver y ot her da y u ntil Da y 7, a n d as cli nicall y i n dicate d t hereafter. 
2  
P E T- C T ( Nec k- C hest- A b d o me n- Pel vis)/ Disease Assess me nt: P E T- C T perf or me d f oll o wi n g t he s u bject’s last li ne of t hera p y a n d pri or t o si g ni n g t he i nf or me d c o nse nt ma y be 
use d f or c o nfir mati o n of eli gi bilit y if wit hi n 2 8 da ys pri or t o e nr oll me nt a n d n o ot her a nti-ca ncer treat me nt has bee n a d mi nistere d.  If P E T- C T is perf or me d > 2 8 da ys pri or t o 
e nr oll me nt or if s u bject recei ves a n y a nti-ca ncer t hera p y bet wee n scree ni n g a n d c o n diti o ni n g c he m ot hera p y, t he P E T- C T sca n m ust be re peate d t o esta blis h a ne w baseli ne. 
P E T- C T will als o be perf or me d at Wee k 4, Wee k 1 2, a n d at t he p ost-treat me nt f oll o w- u p visit.
3  
Sites are str o n gl y e nc o ura ge d t o s u b mit arc hi val/fres h t u m or sa m ple: Eit her f or mali n-fi xe d paraffi n e m be d de d ( F F P E) t u m or bl oc k or u p t o 2 0 u nstai ne d sli des.  Arc hi ve d a n d 
fres h t u m or sa m ples will be s u b mitte d t o ce ntral la b orat or y after eli gi bilit y has bee n c o nfir me d a n d pri or t o start of c o n diti o ni n g c he m ot hera p y. 
 
. 
C CI






A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 6 5
0 7 J u ne 2 0 2 2
8  
I n t he e ve nt t his st u d y is ter mi nate d pri or t o t he c o m pleti o n of t he 1 5- year f oll o w u p f or all s u bjects, s u bjects w h o recei ve d a n i nf usi o n of a xica bta ge ne cil ole ucel will be pr o vi de d t he 
o p p ort u nit y t o tra nsiti o n t o a L T F U st u d y ( K T- U S- 9 8 2- 5 9 6 8) after pr o vi di n g si g ne d i nf or me d c o nse nt.  T he s u bject’s fi nal o n-st u d y visit f or t his st u d y ma y be c o m bi ne d wit h t he first 
visit o n t he L T F U st u d y.  T he ti mi n g of t he fi nal o n-st u d y visit/first L T F U st u d y visit will de pe n d o n t he ti mi n g of t he c ollecti o n of all t he s u bject’s data t hat are re q uire d f or t he pla n ne d 
a nal ysis f or t his st u d y.


A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 6 6
0 7 J u ne 2 0 2 2
8.  
S U B J E C T WI T H D R A W A L
S u bjects ha ve t he ri g ht t o wit h dra w fr o m t he st u d y at a n y ti me a n d f or a n y reas o n wit h o ut 
prej u dice t o t heir f ut ure me dical care b y t he p h ysicia n or at t he i nstit uti o n.
S u bjects ca n decli ne t o c o nti n ue t o recei ve st u d y-re q uire d treat me nt a n d/ or ot her pr ot oc ol 
re q uire d pr oce d ures at a n y ti me d uri n g t he st u d y w hile c o nti n ui n g t o partici pate i n t he st u d y.  
T his is referre d t o as partial wit h dra wal of c o nse nt.  Refer t o Secti o n 8. 3  f or i nstr uctio ns o n 
f oll o w- u p a n d data t o be c ollecte d.
Wit h dra wal of f ull c o nse nt fr o m a st u d y mea ns t hat t he s u bject d oes n ot wis h t o recei ve f urt her 
pr ot oc ol-re q uire d t hera p y, u n der g o pr oce d ures, a n d c o nti n ue partici pati n g i n st u d y f oll o w- u p.  
T he i n vesti gat or is t o disc uss wit h t he s u bject a p pr o priate pr oce d ures f or wit h dra wal fr o m t he 
st u d y (refer t o Secti o n 8. 3 ). 
T he i n vesti gat or a n d/ or s p o ns or ca n als o deci de t o wit h dra w a s u bject fr o m t he I P a n d/ or ot her 
pr ot oc ol-re q uire d t hera pies, pr ot oc ol pr oce d ures, or t he st u d y as a w h ole at a n y ti me pri or t o 
st u d y c o m pleti o n.
8. 1.   
Re as o ns f or Re m o v al fr o m Tre at me nt
Reas o ns f or re m o val fr o m pr ot oc ol-re q uire d I Ps or pr oce d ures i ncl u de a n y of t he f oll o wi n g:

A Es

S u bject re q uest

Pr o d uct n ot a vaila ble

L ost t o f oll o w- u p

Deat h

Decisi o n b y s p o ns or
8. 2.   
Re as o ns f or Re m o v al fr o m St u d y
Reas o ns f or re m o vi n g a s u bject fr o m t he st u d y are as f oll o ws:

S u bject wit h dra wal of c o nse nt fr o m f urt her f oll o w- u p

I n vesti gat or decisi o n

L ost t o f oll o w- u p 

Deat h


A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 6 7
0 7 J u ne 2 0 2 2
8. 3.  
I nstr ucti o ns f or F oll o w- u p a n d D at a t o Be C ollecte d f or S u bjects Wit h dr a w n 
Fr o m Tre at me nt/ St u d y
If partial wit h dra wal of c o nse nt occ urs ( defi ne d i n Secti o n 8 ), t he i n vesti gat or m ust disc uss wit h 
t he s u bject t he a p pr o priate pr ocess f or disc o nti n uati o n fr o m I P or ot her pr ot oc ol-re q uire d 
t hera pies a n d m ust disc uss o pti o ns f or c o nti n ue d partici pati o n, c o m pleti o n of pr oce d ures, a n d t he 
ass ociate d data c ollecti o n as o utli ne d i n t he S O A ( Ta ble 5 , Ta ble 6 , a n d Ta ble 7 ).  T he le vel of 
f oll o w- u p a n d met h o d of c o m m u nicati o n s h o ul d als o be disc usse d bet wee n t he researc h staff a n d 
t he s u bject a n d d oc u me nte d i n t he s o urce d oc u me nts.
If wit h dra wal of f ull c o nse nt occ urs ( defi ne d i n Secti o n 8 ), t he i n vesti gat or is t o disc uss wit h t he 
s u bject t he a p pr o priate pr oce d ures f or wit h dra wal fr o m t he st u d y.  If a s u bject wit h dra ws f ull 
c o nse nt, s u bject data c ollecte d u p t o wit h dra wal of c o nse nt will be retai ne d a n d i ncl u de d i n t he 
a nal ysis of t he st u d y, a n d w here per mitte d b y l ocal re g ulati o ns, p u blicl y a vaila ble data (e g, deat h 
rec or ds).
As part of t he st u d y, sites ma y be as ke d t o c o n d uct searc hes of p u blic rec or ds s uc h as t h ose 
esta blis hi n g s ur vi val stat us (e g, f or s u bjects l ost t o f oll o w- u p), if a vaila ble, t o o btai n s ur vi val 
data f or a n y s u bject f or w h o m t he s ur vi val stat us is n ot k n o w n, per t he a p plica ble l ocal la ws.  
Sites ma y be als o as ke d t o retrie ve a ut o ps y re p orts t o c o nfir m stat us of disease at t he ti me of 
deat h, if p ossi ble, per t he l ocal la ws.


A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 6 8
0 7 J u ne 2 0 2 2
9.  
S A F E T Y R E P O R TI N G
9. 1.   
A d verse E ve nts
A n A E is defi ne d as a n y u nt o war d me dical occ urre nce i n a cli nical trial s u bject.  T he e ve nt d oes 
n ot necessaril y ha ve a relati o ns hi p wit h st u d y treat me nt.  T he i n vesti gat or is res p o nsi ble f or 
e ns uri n g t hat a n y A Es o bser ve d b y t he i n vesti gat or or re p orte d b y t he s u bject are rec or de d i n t he 
s u bject’s me dical rec or d.  T he defi niti o n of A Es i ncl u des w orse ni n g of a pre-e xisti n g me dical 
c o n diti o n.  W orse ni n g i n dicates t hat t he pre-e xisti n g me dical c o n diti o n has i ncrease d i n se verit y, 
fre q ue nc y, a n d/ or d urati o n or has a n ass ociati o n wit h a w orse o utc o me.  W he n rec or di n g s uc h 
e ve nts, descri pti o ns t hat t he pre-e xisti n g c o n diti o n has c ha n ge d (e g, m ore fre q ue nt hea dac hes f or 
a s u bject wit h pre-e xisti n g hea dac hes or bl o o d press ure [ B P]) i n a s u bject wit h pre-e xisti n g 
h y perte nsi o n) m ust be pr o vi de d.
A n A E d oes n ot i ncl u de t he f oll o wi n g:

A pre-e xisti n g c o n diti o n t hat has n ot w orse ne d d uri n g t he st u d y or i n v ol ves a n i nter ve nti o n 
(s uc h as electi ve c os metic s ur ger y or a me dical pr oce d ure) w hile o n st u d y  

I nter ve nti o ns f or pretreat me nt c o n diti o ns (s uc h as electi ve c os metic s ur ger y) or me dical 
pr oce d ures t hat were pla n ne d bef ore st u d y partici pati o n 

H os pitalizati o n f or st u d y treat me nt i nf usi o ns or st u d y- ma n date d pr oce d ures or h os pitalizati o n 
as a preca uti o nar y meas ure per i nstit uti o nal p olic y  

T he ter m “ disease pr o gressi o n,” as assesse d b y meas ure me nt of mali g na nt lesi o ns o n 
ra di o gra p hs or ot her met h o ds is n ot c o nsi dere d t o be a n A E.  Deat h d ue t o disease 
pr o gressi o n i n t he a bse nce of si g ns a n d s y m pt o ms s h o ul d be re p orte d as t he pri mar y t u m or 
t y pe (e g, B-cell l y m p h o ma). 
W he n a n A E or S A E is d ue t o t he disease u n der i n vesti gati o n, it is necessar y t o re p ort t he si g ns 
a n d s y m pt o ms.  W orse ni n g of si g ns a n d s y m pt o ms of t he mali g na nc y u n der st u d y s h o ul d als o be 
re p orte d as A Es i n t he a p pr o priate secti o n of t he C R F. 
T he i n vesti gat or’s cli nical j u d g me nt is use d t o deter mi ne w het her a s u bject is t o be re m o ve d 
fr o m treat me nt d ue t o a n A E.  If a s u bject re q uests t o wit h dra w fr o m pr ot oc ol-re q uire d t hera pies 
or t he st u d y beca use of a n A E, t he n t he s u bject s h o ul d u n der g o t he pr oce d ures o utli ne d i n t he 
p ost-treat me nt f oll o w- u p visit of t he S O A. 
9. 2.   
Re p orti n g of A Es
T he i n vesti gat or is res p o nsi ble f or re p orti n g all A Es o bser ve d b y t he i n vesti gat or or re p orte d b y 
t he s u bject t hat occ ur after e nr oll me nt t hr o u g h 3 0 da ys after c o m pleti n g t he fi nal d ose of 
ut o mil u ma b or 1 2 wee ks after t he a xica bta ge ne cil ole ucel i nf usi o n, w hic he ver is l o n ger.  F or 
s u bjects w h o are e nr olle d, b ut d o n ot recei ve a xica bta ge ne cil ole ucel, t he A E re p orti n g peri o d 
e n ds 3 0 da ys after t he last st u d y-s pecific pr oce d ure (e g, le u ka p heresis, c o n diti o ni n g 
c he m ot hera p y).  After t hat, o nl y tar gete d A Es will be re p orte d t hr o u g h 2 4 m o nt hs after 


A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 6 9
0 7 J u ne 2 0 2 2
a xica bta ge ne cil ole ucel i nf usi o n or disease pr o gressi o n, w hic he ver occ urs first, a n d will be 
rec or de d i n t he e C R F 
Tar gete d A Es i ncl u de ne ur ol o gical e ve nts, he mat ol o gical e ve nts, i nfecti o ns, G V H D, 
a ut oi m m u ne dis or ders, a n d sec o n dar y mali g na ncies.  Sec o n dar y mali g na ncies are defi ne d as t he 
de vel o p me nt of a n y ne w mali g na ncies, wit h t he e xce pti o n of a rela pse of t he pri mar y 
mali g na nc y, occ urri n g after t he a d mi nistrati o n of st u d y treat me nt.
All A Es dee me d relate d t o a xica bta ge ne cil ole ucel i nf usi o n or ut o mil u ma b s h o ul d be rec or de d i n 
t he e C R F a n d re p orte d re gar dless of st u d y peri o d
T he i n vesti gat or m ust pr o vi de t he i nf or mati o n liste d bel o w re gar di n g t he A Es bei n g re p orte d: 

A E dia g n osis or s y n dr o me (if n ot k n o w n, si g ns or s y m pt o ms)

Dates of o nset a n d res ol uti o n

Se verit y

Assess me nt of relate d ness t o I P, c o n diti o ni n g c he m ot hera p y, or st u d y pr oce d ures

Acti o n ta ke n
T he A E gra di n g scale use d will be t he N CI C T C A E.  A c o p y of t he gra di n g scale ca n be 
d o w nl oa de d fr o m t he Ca ncer T hera p y E val uati o n Pr o gra m ( C T E P) h o me pa ge 
( htt p://cte p.ca ncer. g o v).  C R S e ve nts will be re p orte d usi n g t he gra di n g scale o utli ne d i n t he 
a xica bta ge ne cil ole ucel I B (as m o difie d fr o m Lee, et al, 2 0 1 4).  T he se verit y of ne ur ol o gic 
t o xicities will be gra de d usi n g t he N CI C T C A E.  T he se verit y of i n di vi d ual si g ns/s y m pt o ms of 
C R S a n d ne ur ol o gic t o xicities will be gra de d acc or di n g t o t he N CI C T C A E f or t h ose 
si g ns/s y m pt o ms t hat are n ot part of t he gra di n g scale.  
I n re vie wi n g A Es, i n vesti gat ors m ust assess w het her t he A E is p ossi bl y relate d t o 
1) a xica bta ge ne cil ole ucel, 2) ut o mil u ma b, 3) c o n diti o ni n g c he m ot hera p y, 4) a n y 
pr ot oc ol-re q uire d st u d y pr oce d ure or treat me nt, 5) disease pr o gressi o n, 6) c o nc urre nt disease, 
7) c o nc o mita nt me dicati o n, or 8) ot her.  
T he relati o ns hi p is i n dicate d b y a “relate d” or “ n ot relate d” res p o nse a n d e ntere d i n t he e C R F.  I n 
assessi n g ca usalit y, t he i n vesti gat or or q ualifie d s u bi n vesti gat or will use cli nical j u d g me nt a n d 
t he f oll o wi n g c o nsi derati o ns:

N ot Relate d: E vi de nce e xists t hat t he A E has a n eti ol o g y ot her t ha n t he I P or st u d y 
pr oce d ure.  F or S A Es, a n alter nati ve ca usalit y m ust be pr o vi de d (e g, disease pr o gressi o n, 
c o nc urre nt disease[s], c o nc o mita nt me dicati o ns, or ot her).

Relate d: T here is reas o na ble p ossi bilit y t hat t he e ve nt ma y ha ve bee n ca use d b y t he I P or as a 
res ult of a st u d y pr oce d ure.


A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 7 0
0 7 J u ne 2 0 2 2
A d diti o nal rele va nt data wit h res pect t o descri bi n g t he A E will be c ollecte d i n t he C R Fs.  F or 
A Es/ S A Es, a dia g n osis (if k n o w n) rat her t ha n i n di vi d ual si g ns a n d s y m pt o ms s h o ul d be rec or de d 
o n t he e C R F A E f or m.  T he e xce pti o n is f or C R S w here b ot h t he dia g n osis a n d si g nifica nt si g ns 
a n d s y m pt o ms will be ca pt ure d i n t he e C R F.
T he i n vesti gat or is e x pecte d t o f oll o w re p orte d A Es u ntil sta bilizati o n or res ol uti o n.  If a s u bject 
be gi ns a ne w a nti-ca ncer t hera p y, t he A E re p orti n g peri o d f or n o n- S A Es e n ds at t he ti me t he ne w 
treat me nt is starte d.
9. 2. 1.   
Re p orti n g A b n or m al L a b or at or y Fi n di n gs
T he i n vesti gat or is res p o nsi ble f or re vie wi n g la b orat or y test res ults a n d deter mi ni n g w het her a n 
a b n or mal val ue i n a n i n di vi d ual st u d y s u bject re prese nts a cli nicall y si g nifica nt c ha n ge fr o m t he 
s u bject’s baseli ne val ues.  
I n ge neral, a b n or mal la b orat or y fi n di n gs wit h o ut cli nical si g nifica nce ( base d o n t he 
i n vesti gat or’s j u d g me nt) are n ot t o be rec or de d as A Es.  H o we ver, a b n or mal la b orat or y fi n di n gs 
t hat res ult i n ne w or w orse ni n g cli nical se q uelae or t hat re q uire t hera p y or a dj ust me nt i n c urre nt 
t hera p y, are c o nsi dere d A Es.  W here a p plica ble, cli nical se q uelae ( n ot t he la b orat or y 
a b n or malit y) are t o be rec or de d as t he A E. 
A n a b n or mal la b orat or y test res ult m ust be re p orte d as a n A E if it is a c ha n ge fr o m baseli ne a n d 
meets a n y of t he f oll o wi n g criteria:

Ass ociate d wit h cli nical s y m pt o ms

Res ults i n a me dical i nter ve nti o n (e g, p otassi u m s u p ple me ntati o n f or h y p o kale mia or ir o n 
re place me nt t hera p y f or a ne mia) or a c ha n ge i n c o nc o mita nt t hera p y

Cli nicall y si g nifica nt i n t he i n vesti gat or’s j u d g me nt 
9. 2. 2.   
A b n or m al Li ver F u ncti o n Tests ( H y’s L a w): Dr u g-i n d uce d Li ver I nj ur y
Ele vate d A L T or A S T ( > 3 x U L N), i n c o m bi nati o n wit h eit her a n ele vate d t otal bilir u bi n ( > 2 x 
U L N) or cli nical ja u n dice i n t he a bse nce of c h olestasis or ot her ca uses of h y per bilir u bi ne mia, is 
c o nsi dere d t o be a n i n dicat or of se vere li ver i nj ur y (ie, dr u g-i n d uce d li ver i nj ur y [ DI LI]). 
T heref ore, i n vesti gat ors m ust re p ort t he f oll o wi n g e ve nts as S A Es:

A L T or A S T > 3 x U L N i n c o m bi nati o n wit h t otal bilir u bi n > 2 x U L N ( of w hic h 3 5 % is 
direct bilir u bi n)

A L T or A S T > 3 x U L N i n c o m bi nati o n wit h cli nical ja u n dice
9. 2. 3.   
A Es of S peci al I nterest (I m me di atel y Re p ort a ble t o t he S p o ns or)
I n vesti gat ors m ust re p ort t he f oll o wi n g A Es of s pecial i nterest t o t he s p o ns or n o m ore t ha n 
2 4 h o urs after lear ni n g of t he e ve nt:

DI LI is defi ne d i n Secti o n 9. 2. 2 . A ris k ma na ge me nt pla n ass ociate d wit h ut o mil u ma b 
re q uires t hat a n y DI LI is re p orte d as a n A E u n der e x pe dite d ti meli nes. 


A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 7 1
0 7 J u ne 2 0 2 2

A n y DI LI occ urri n g bet wee n t he first i nf usi o n of ut o mil u ma b a n d 3 0 da ys after c o m pleti n g 
t he fi nal d ose of ut o mil u ma b m ust be re p orte d wit hi n 2 4 h o urs. 

C R S e ve nts Gra de 3 or hi g her ( by m o difie d Lee criteri a) 

Ne ur ol o gic e ve nts Gra de 3 or hi g her ( by C T C A E)

All e ve nts of cere bral e de ma 

All e ve nts of he m o p ha g oc ytic l y m p h o histi oc yt osis/ macr o p ha ge acti vati o n s y n dr o me
9. 3.   
Defi niti o n of Seri o us A d verse E ve nts
A n S A E is defi ne d as a n A E t hat meets at least 1 of t he f oll o wi n g seri o us criteria:

Fatal

Life-t hreate ni n g (ie, a n e ve nt t hat places t he s u bject at i m me diate ris k of deat h); it d oes n ot 
refer t o a n e ve nt t hat h y p ot heticall y mi g ht ha ve ca use d deat h if it were m ore se vere

Re q uires i n- patie nt h os pitalizati o n or pr ol o n gati o n of e xisti n g h os pitalizati o n

Res ults i n persiste nt or si g nifica nt disa bilit y/i nca pacit y

C o n ge nital a n o mal y/ birt h defect

Ot her me dicall y i m p orta nt seri o us e ve nt.  If a n i n vesti gat or c o nsi ders a n e ve nt t o be 
cli nicall y i m p orta nt, b ut it d oes n ot meet a n y of t he seri o us criteria, t he e ve nt c o ul d be 
classifie d as a n S A E wit h t he criteri o n of “ ot her me dicall y i m p orta nt seri o us e ve nt.”
A n A E w o ul d meet t he criteri o n of “re q uires h os pitalizati o n” if t he e ve nt necessitate d a n 
a d missi o n t o a healt hcare facilit y (e g, o ver ni g ht sta y). 
E ve nts t hat re q uire a n escalati o n of care w he n t he s u bject is alrea d y h os pitalize d s h o ul d be 
rec or de d as a n S A E.  E xa m ples of s uc h e ve nts i ncl u de m o ve me nt fr o m r o uti ne care i n t he 
h os pital t o t he i nte nsi ve care u nit (I C U) or if t hat e ve nt res ulte d i n a pr ol o n gati o n of t he e xisti n g 
pla n ne d h os pitalizati o n.  T he ter ms “se vere” a n d “seri o us” are n ot s y n o n y m o us.  Se verit y refers 
t o t he i nte nsit y of a n A E acc or di n g t o N CI C T C A E criteria; t he e ve nt itself ma y be of relati vel y 
mi n or me dical si g nifica nce a n d, t heref ore, ma y n ot meet t he seri o us ness criteria.  Se verit y a n d 
seri o us ness nee d t o be i n de pe n de ntl y assesse d f or eac h A E rec or de d o n t he electr o nic C R F 
(e C R F).
9. 4.   
Re p orti n g of S A Es
T he i n vesti gat or is res p o nsi ble f or re p orti n g all S A Es o bser ve d b y t he i n vesti gat or or re p orte d b y 
t he s u bject t hat occ ur after si g ni n g of t he i nf or me d c o nse nt t hr o u g h t he 3 0 da ys after c o m pleti n g 


A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 7 2
0 7 J u ne 2 0 2 2
t he fi nal d ose of ut o mil u ma b or 1 2 wee ks after t he a xica bta ge ne cil ole ucel i nf usi o n, w hic he ver is 
l o n ger.  After t his f oll o w- u p peri o d has bee n c o m plete d, o nl y tar gete d S A Es will be re p orte d.  
Tar gete d S A Es are defi ne d as a n d i ncl u de ne ur ol o gical, he mat ol o gical, i nfecti o ns, G V H D a n d 
a ut oi m m u ne dis or ders, a n d sec o n dar y mali g na ncies t hat occ ur u p t o 2 4 m o nt hs or u ntil disease 
pr o gressi o n.  After 2 4 m o nt hs, tar gete d S A Es of sec o n dar y mali g na ncies will c o nti n ue t o be 
re p orte d t hr o u g h 1 5 years after a xica bta ge ne cil ole ucel i nf usi o n a n d will be rec or de d i n t he 
e C R F.
S A Es, w hic h t he i n vesti gat or assesses as relate d t o a xica bta ge ne cil ole ucel or ut o mil u ma b, 
i ncl u di n g all deat hs t hat occ ur fr o m si g ni n g of t he scree ni n g I C F s h o ul d be re p orte d re gar dless of 
t he ti me peri o d.  Refer t o Secti o n 9. 5  f or i nstr uctio ns o n re p orti n g deat hs.
F or s u bjects w h o scree n fail or are e nr olle d, b ut d o n ot recei ve a xica bta ge ne cil ole ucel, t he 
re p orti n g peri o d f or S A Es e n ds 3 0 da ys after t he last st u d y-s pecific pr oce d ure (e g, scree n 
pr oce d ure, le u ka p heresis, c o n diti o ni n g c he m ot hera p y).  
T he f oll o wi n g m ust be s u b mitte d t o Kite via t he e S A E s yste m wit hi n 2 4 h o urs of t he 
i n vesti gat or’s k n o wle d ge of t he e ve nt:

All S A Es

Pre g na nc y or lactati o n e x p os ure as defi ne d i n Secti o n 9. 7

A Es of s pecial i nterest as defi ne d i n Secti o n 9. 2. 3
If t he e S A E s yste m is u na vaila ble (e g, s yste m o uta ge), t he n t he S A E m ust be s u b mitte d usi n g t he 
S A E Re p ort F or m a n d se nt via e mail t o t he S A E Re p orti n g mail b o x: 
 
S u bse q ue ntl y, S A Es will be e ntere d i nt o t he e S A E s yste m o nce it bec o mes a vaila ble. 
All S A Es will be re p orte d t o t he healt h a ut h orities per l ocal re p orti n g g ui deli nes.
Disease pr o gressi o n of t he mali g na nc y is n ot c o nsi dere d a n A E.  H o we ver, si g ns a n d s y m pt o ms 
of disease pr o gressi o n ma y be rec or de d o n t he C R F as A Es or S A Es a n d i n dicate d as bei n g d ue 
t o disease pr o gressi o n.  If t he mali g na nc y has a fatal o utc o me bef ore 2 4 m o nt hs, t he n t he e ve nt 
“ B-cell l y m p h o ma” m ust be rec or de d as a n S A E wit h t he o utc o me bei n g fatal. 
Deat h m ust be re p orte d if it occ urs d uri n g t he S A E re p orti n g peri o d, irres pecti ve of a n y 
i nter ve ni n g treat me nt.  Refer t o Secti o n 9. 5  f or i nstr uctio ns o n re p orti n g deat hs ass ociate d wit h 
t he u n derl yi n g mali g na nc y/ disease pr o gressi o n.
A n y deat h occ urri n g after e nr oll me nt  a n d pri or t o t he p ost-treat me nt f oll o w- u p peri o d , re gar dless 
of attri b uti o n t o treat me nt, re q uires e x pe dite d re p orti n g wit hi n 2 4 h o urs.  A n y deat h occ urri n g 
after t he p ost-treat me nt f oll o w- u p peri o d re q uires e x pe dite d re p orti n g wit hi n 2 4 h o urs o nl y if it is 
c o nsi dere d relate d t o treat me nt, a xica bta ge ne cil ole ucel, ut o mil u ma b, a n d/ or st u d y-re q uire d 
treat me nts (e g, l y m p h o de pleti n g c he m ot hera p y).
P P D


A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 7 3
0 7 J u ne 2 0 2 2
F oll o wi n g c o m pleti o n of K T E- C 1 9- 1 1 1, a n y rele va nt i nf or mati o n o n o n g oi n g S A Es m ust be 
s u b mitte d t o Kite P har ma wit hi n 2 4 h o urs of t he i n vesti gat or’s k n o wle d ge of t he e ve nt usi n g t he 
pa per S A E Re p ort F or m a n d se nt via e- mail t o t he S A E Re p orti n g mail b o x: 
9. 5.   
Re p orti n g De at hs
Deat hs t hat occ ur d uri n g t he pr ot oc ol-s pecifie d A E re p orti n g peri o d ( wit hi n t he 3 0 da ys after 
c o m pleti n g t he fi nal d ose of ut o mil u ma b or 1 2 wee ks after t he a xica bta ge ne cil ole ucel i nf usi o n, 
w hic he ver is l o n ger) t hat are attri b ute d b y t he i n vesti gat or s olel y t o pr o gressi o n of u n derl yi n g 
l y m p h o ma s h o ul d be rec or de d as S A Es wit h t he preferre d ter m “ B-cell l y m p h o ma” a n d m ust be 
re p orte d i m me diatel y t o t he s p o ns or.  A n y deat h occ urri n g after t he si g ni n g of t he mai n st u d y 
I C F wit hi n t he A E re p orti n g peri o d a n d re gar dless of attri b uti o n t o treat me nt, re q uires e x pe dite d 
re p orti n g wit hi n 2 4 h o urs after t he i n vesti gat or’s k n o wle d ge of t he e ve nt.  A n y deat h occ urri n g 
after t he A E re p orti n g peri o d re q uires e x pe dite d re p orti n g wit hi n 2 4 h o urs o nl y if it is c o nsi dere d 
relate d t o treat me nt, a xica bta ge ne cil ole ucel i nf usi o n, ut o mil u ma b, a n d/ or st u d y-re q uire d 
treat me nts (e g, l y m p h o de pleti n g c he m ot hera p y).
Deat h is a n o utc o me a n d n ot a disti nct e ve nt.  T he e ve nt or c o n diti o n t hat ca use d or c o ntri b ute d 
t o t he fatal o utc o me s h o ul d be rec or de d o n t he A E f or m.  H o we ver, e ver y eff ort s h o ul d be ma de 
t o ca pt ure t he esta blis he d ca use of deat h, w hic h ma y bec o me a vaila ble later o n (e g, after 
a ut o ps y).  
9. 6.   
Di a g n osis vers us Si g ns a n d S y m pt o ms
F or A Es, a dia g n osis (if k n o w n) rat her t ha n i n di vi d ual si g ns a n d s y m pt o ms s h o ul d be rec or de d 
o n t he A E f or m.  T he e xce pti o n is f or C R S w here b ot h t he dia g n osis a n d t he si g ns a n d s y m pt o ms 
c o m prisi n g C R S will be ca pt ure d o n t he C R F A E f or m.  F or sit uati o ns w he n a n A E or S A E is 
d ue t o t he disease u n der i n vesti gati o n, re p ort t he si g ns a n d s y m pt o ms of t he A E or S A E. 
W orse ni n g of si g ns a n d s y m pt o ms of t he mali g na nc y u n der st u d y s h o ul d als o be re p orte d as A Es 
i n t he a p pr o priate secti o n of t he C R F.
9. 7.  
Pre g n a nc y a n d L act ati o n
T here is n o rele va nt cli nical e x perie nce wit h a xica bta ge ne cil ole ucel i n pre g na nt or lactati n g 
w o me n, a n d a ni mal re pr o d ucti ve st u dies ha ve n ot bee n perf or me d.  W o me n of c hil d beari n g 
p ote ntial m ust ha ve a ne gati ve pre g na nc y test pri or t o e nr oll me nt beca use of t he p ote ntiall y 
da n ger o us effects of t he pre parati ve c he m ot hera p y o n t he fet us.  W o me n of c hil d beari n g 
p ote ntial s h o ul d be m o nit ore d acc or di n g t o l ocal a n d c o u ntr y-s pecific re g ulati o ns.  T his 
e x peri me ntal t hera p y s h o ul d n ot be a d mi nistere d t o pre g na nt w o me n or w o me n w h o are 
breastfee di n g. 
Fe male s u bjects a n d fe male part ners of male s u bjects are rec o m me n de d t o use hi g hl y effecti ve 
c o ntrace pti o n ( met h o d m ust ac hie ve a n a n n ual fail ure rate of < 1 %) t hr o u g h at least 9 0 da ys after 
t he last d ose of ut o mil u ma b or f or at least 6 m o nt hs after a xica bta ge ne cil ole ucel i nf usi o n or 
c o n diti o ni n g c he m ot hera p y d osi n g ( w hic he ver occ urs later).  Male s u bjects are rec o m me n de d t o 
P P D


A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 7 4
0 7 J u ne 2 0 2 2
n ot fat her a c hil d f or at least 9 0 da ys after t he last d ose of ut o mil u ma b or f or at least 6 m o nt hs 
after c o m pleti o n of a xica bta ge ne cil ole ucel i nf usi o n or c o n diti o ni n g c he m ot hera p y d osi n g 
( w hic he ver occ urs later).  Refer t o A p pe n di x 3  f or a c o mplete list of hi g hl y effecti ve 
c o ntrace pti o n met h o ds.
If a pre g na nc y occ urs i n eit her a fe male s u bject e nr olle d i nt o t he st u d y a n y ti me after 
a xica bta ge ne cil ole ucel i nf usi o n, wit hi n 6 m o nt hs after c o n diti o ni n g c he m ot hera p y, or wit hi n 
9 0 da ys after t he last d ose of ut o mil u ma b, w hic he ver is l o n ger, or i n a fe male part ner of a male 
s u bject wit hi n 6 m o nt hs of a xica bta ge ne cil ole ucel i nf usi o n or c o m pleti n g t he c o n diti o ni n g 
c he m ot hera p y or wit hi n 9 0 da ys after t he last d ose of ut o mil u ma b, w hic he ver is l o n ger, t he 
pre g na nc y m ust be re p orte d usi n g t he Pre g na nc y Re p orti n g F or m wit hi n 2 4 h o urs of t he 
i n vesti gat or’s k n o wle d ge of t he pre g na nc y e ve nt.
T he pre g na nc y itself is n ot c o nsi dere d a n A E n or is a n i n d uce d electi ve a b orti o n t o ter mi nate a 
pre g na nc y wit h o ut me dical reas o ns.  A n y pre mat ure ter mi nati o n of pre g na nc y (e g, a s p o nta ne o us 
a b orti o n or a n i n d uce d t hera pe utic a b orti o n d ue t o c o m plicati o ns or ot her me dical reas o ns) m ust 
be re p orte d wit hi n 2 4 h o urs as a n S A E.  T he u n derl yi n g me dical reas o n f or t his pr oce d ure s h o ul d 
be rec or de d as t he A E ter m.  A n y S A E occ urri n g as a n a d verse pre g na nc y o utc o me after t he 
st u d y has bee n c o m plete d m ust be re p orte d t o Kite Patie nt Safet y a n d P har mac o vi gila nce.
T he pre g na nt s u bject or s u bject part ner s h o ul d recei ve a p pr o priate m o nit ori n g a n d care u ntil 
c o ncl usi o n of t he pre g na nc y.  T he o utc o me s h o ul d be re p orte d t o Kite Patie nt Safet y a n d 
P har mac o vi gila nce usi n g t he Pre g na nc y O utc o me Re p ort F or m.  If t he e n d of t he pre g na nc y 
occ urs after t he st u d y has bee n c o m plete d, t he o utc o me s h o ul d be re p orte d directl y t o Kite 
Patie nt Safet y a n d P har mac o vi gila nce.
Pre g na ncies of fe male part ners of male st u d y s u bjects e x p ose d t o pr ot oc ol-re q uire d t hera pies 
m ust als o be re p orte d a n d rele va nt i nf or mati o n s h o ul d be s u b mitte d t o Kite Patie nt Safet y a n d 
P har mac o vi gila nce usi n g t he pre g na nc y a n d pre g na nc y o utc o me f or ms wit hi n 2 4 h o urs.  If t he 
e n d of t he pre g na nc y occ urs after t he st u d y has bee n c o m plete d, t he o utc o me s h o ul d be re p orte d 
directl y t o Kite Patie nt Safet y a n d P har mac o vi gila nce.
All s uc h pre g na ncies m ust be re p orte d t o Kite Patie nt Safet y a n d P har mac o vi gila nce usi n g t he 
Pre g na nc y Re p ort F or m wit hi n 2 4 h o urs after bec o mi n g a ware of t he pre g na nc y.  I nf or mati o n 
re gar di n g t he pre g na nc y a n d/ or o utc o me will be re q ueste d b y t he s p o ns or.  Pre g na nc y Re p ort 
F or ms s h o ul d be re p orte d t o Kite Patie nt Safet y a n d P har mac o vi gila nce at 
 or fa x: 
. 
If a lactati o n case occ urs w hile t he fe male s u bject is ta ki n g pr ot oc ol-re q uire d t hera pies, t he 
lactati o n case s h o ul d be re p orte d t o t he ke y s p o ns or c o ntact wit hi n 2 4 h o urs of t he i n vesti gat or’s 
k n o wle d ge of t he e ve nt usi n g t he S pecial Sit uati o ns Re p orti n g F or m.
I n a d diti o n t o re p orti n g a lactati o n case d uri n g t he st u d y, i n vesti gat ors s h o ul d m o nit or f or 
lactati o n cases t hat occ ur after t he last d ose of pr ot oc ol-re q uire d t hera pies t hr o u g h 6 m o nt hs.  
Re p ort t he lactati o n case a n d S pecial Sit uati o ns Re p orti n g F or ms t o Kite Patie nt Safet y a n d 
P har mac o vi gila nce at 
 or fa x: 
.
P P D
P P D
P P D
P P D


A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 7 5
0 7 J u ne 2 0 2 2
9. 8.   
H os pit aliz ati o n a n d Pr ol o n ge d H os pit aliz ati o n
A n y A E t hat res ults i n h os pitalizati o n or pr ol o n ge d h os pitalizati o n s h o ul d be d oc u me nte d a n d 
re p orte d as a n S A E as descri be d i n Secti o n 9. 4 .  
T he f oll o wi n g h os pitalizati o n sce nari os are n ot c o nsi dere d t o be S A Es:

H os pitalizati o n f or palliati ve care or h os pice care

Pla n ne d h os pitalizati o n re q uire d b y t he pr ot oc ol (e g, f or m o nit ori n g of t he s u bject or t o 
perf or m a n efficac y meas ure me nt f or t he st u d y)

Pla n ne d h os pitalizati o n f or a pre-e xisti n g c o n diti o n

H os pitalizati o n d ue t o pr o gressi o n of t he u n derl yi n g ca ncer
9. 9.   
A b n or m al Vit al Si g n V al ues
N ot all vital si g n a b n or malities q ualif y as a n A E.  A vital si g n res ult m ust be re p orte d as a n A E if 
it is a c ha n ge fr o m baseli ne a n d meets a n y of t he f oll o wi n g criteria:

Acc o m pa nie d b y cli nical s y m pt o ms

Res ults i n a me dical i nter ve nti o n or a c ha n ge i n c o nc o mita nt t hera p y

Cli nicall y si g nifica nt i n t he i n vesti gat or’s j u d g me nt
It is t he i n vesti gat or’s res p o nsi bilit y t o re vie w all vital si g n fi n di n gs.  Me dical a n d scie ntific 
j u d g me nt s h o ul d be e xercise d i n deci di n g w het her a n is olate d vital si g n a b n or malit y s h o ul d be 
classifie d as a n A E.  H o we ver, if a cli nicall y si g nifica nt vital si g n a b n or malit y is a si g n of a 
disease or s y n dr o me (e g, hi g h bl o o d press ure), o nl y t he dia g n osis (ie, h y perte nsi o n) s h o ul d be 
rec or de d o n t he C R F.
9. 1 0.  
S afet y Re vie w Te a m a n d D ose-li miti n g T o xicit y 
T he S R T will be s pecificall y c hartere d t o re vie w safet y data d uri n g P hase 1 of t he st u d y a n d 
ma ke rec o m me n dati o ns o n f urt her st u d y c o n d uct i n P hase 1 a n d pr o gressi o n t o P hase 2 base d o n 
t he i nci de nce of D L Ts a n d re vie w of S A Es.
D ose-li miti n g t o xicit y is defi ne d as t he f oll o wi n g a xica bta ge ne cil ole ucel- or ut o mil u ma b-relate d 
e ve nts wit h a n o nset fr o m i m me diatel y after a n d t hr o u g h 2 8 da ys f oll o wi n g t he first ut o mil u ma b 
i nf usi o n: 

Gra de 4 he mat ol o gic t o xicit y lasti n g m ore t ha n 3 0 da ys (e xce pt l y m p h o pe nia or B-cell 
a plasia)


A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 7 6
0 7 J u ne 2 0 2 2

All a xica bta ge ne cil ole ucel- or ut o mil u ma b-relate d Gra de 3 n o n- he mat ol o gic t o xicities 
lasti n g f or > 7 da ys a n d all a xica bta ge ne cil ole ucel- or ut o mil u ma b-relate d Gra de 4 
n o n- he mat ol o gic t o xicities re gar dless of d urati o n are c o nsi dere d D L Ts, wit h t he e xce pti o n of 
t he f oll o wi n g, w hic h are n ot c o nsi dere d D L Ts:

A p hasia/ d ys p hasia or c o nf usi o n/c o g niti ve dist ur ba nce, w hic h res ol ves t o at least Gra de 1 
wit hi n 2 wee ks a n d t o at least baseli ne wit hi n 4 wee ks 

Fe ver of a n y gra de

I m me diate I P-relate d h y perse nsiti vit y reacti o ns occ urri n g wit hi n 2 h o urs of cell or 
ut o mil u ma b i nf usi o n t hat are re versi ble t o a Gra de 2 or less wit hi n 2 4 h o urs or 
a d mi nistrati o n wit h sta n dar d t hera p y 

Re nal t o xicit y, w hic h re q uires dial ysis f or ≤ 7 da ys

I nt u bati o n f or air wa y pr otecti o n if ≤ 7 da ys 

T u m or l ysis s y n dr o me ( T L S), i ncl u di n g ass ociate d ma nifestati o ns attri b uta ble t o T L S 
(e g, electr ol yte a b n or malities, re nal f u ncti o n, h y per urice mia)

Gra de 3 tra nsa mi nase, al kali ne p h os p hatase, bilir u bi n or ot her li ver f u ncti o n test 
ele vati o n, pr o vi de d t here is res ol uti o n t o ≤ Gra de 2 wit hi n 1 4 da ys 

Gra de 4 tra nsie nt ser u m he patic e nz y me a b n or malities pr o vi de d t here is res ol uti o n 
t o ≤ Gra de 3 wit hi n < 7 2 h o urs 

Gra de 3 na usea a n d/ or a n ore xia 
C R S will be gra de d acc or di n g t o a m o difie d gra di n g s yste m { Lee 2 0 1 4 } as  descri be d i n t he 
c urre nt a xica bta ge ne cil ole ucel I B.  A Es attri b ute d t o C R S will be ma p pe d t o t he o verall C R S 
gra di n g assess me nt f or t he deter mi nati o n of D L T.  C o nsiste nt wit h n o n- C R S t o xicities, all 
occ urre nces of Gra de 3 C R S of d urati o n > 7 da ys a n d all occ urre nces of Gra de 4 C R S are 
c o nsi dere d D L Ts, ot her t ha n occ urre nces of C R S d ue t o t he e xce pti o ns liste d a b o ve.  
D uri n g P hase 1, a p pr o xi matel y 3 t o 3 6 s u bjects wit h lar ge B-cell l y m p h o ma will be e nr olle d t o 
e val uate t he safet y of a xica bta ge ne cil ole ucel a n d ut o mil u ma b re gi me ns. 
S u bjects i n eac h c o h ort will be e val uate d f or D L Ts wit hi n t he first 2 8 da ys f oll o wi n g t he 
c o m pleti o n of t heir first d ose of ut o mil u ma b.  T he a nal ysis of D L Ts will be base d o n t he 
i nci de nce of D L T a n d o verall safet y pr ofile of t he a xica bta ge ne cil ole ucel a n d ut o mil u ma b 
re gi me n.  If t he s u bject i nci de nce of D L T is 0 of 3 s u bjects, t he st u d y will pr ocee d t o t he ne xt 
c o h ort or if s pecific t o C o h ort 5, t he st u d y ma y pr ocee d t o P hase 2 of t he trial.  T his decisi o n will 
be base d o n o verall be nefit/ris k a n d a vaila ble bi o mar ker data.


A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 7 7
0 7 J u ne 2 0 2 2
H o we ver, if 1 of t he 3 e nr olle d s u bjects wit hi n a n y c o h ort prese nt wit h a pr ot oc ol- defi ne d D L T 
d uri n g P hase 1, t he S R T ma y rec o m me n d e nr olli n g a n a d diti o nal set of 3 s u bjects ( u p t o 
6 s u bjects i n t otal) at t he sa me d ose t hat was a d mi nistere d i n t he first 3 s u bjects i n t hat c o h ort.  I n 
t his sce nari o, pr o gressi o n t o t he ne xt c o h ort or t o P hase 2 of t he st u d y will pr ocee d if 1 of t he 
first 6 s u bjects prese nts wit h a D L T.
If t he s u bject i nci de nce of D L T is ≥ 2/ 6, ot her a xica bta ge ne cil ole ucel a n d ut o mil u ma b re gi me ns, 
i ncl u di n g differe nt d oses of ut o mil u ma b, ma y be e x pl ore d i n c olla b orati o n wit h t he S R T i n a n 
a d diti o nal 3 t o 6 s u bjects ( Fi g ure 5 ).  T he sa me D L T r ules a p pl y as pre vi o usl y descri be d. 
At t he c o ncl usi o n of eac h S R T safet y re vie w meeti n g, t he S R T will ma ke rec o m me n dati o ns o n 
f urt her st u d y c o n d uct.  T he S R T will ma ke a rec o m me n dati o n fr o m a m o n g t he f oll o wi n g: 

E nr oll a d diti o nal s u bjects f or t hat d osi n g c o h ort 

C o nti n ue e val uati o n of a d diti o nal re gi me ns as s pecifie d i n t he st u d y pr ot oc ol 

C ha n ge i n st u d y c o n d uct 

Pr ocee d t o P hase 2 

Ter mi nate st u d y 
A d diti o nal a xica bta ge ne cil ole ucel a n d ut o mil u ma b re gi me ns n ot c urre ntl y s pecifie d i n t he 
pr ot oc ol ma y als o be e x pl ore d i n P hase 1 at t he rec o m me n dati o n of t he S R T, f oll o wi n g a 
pr ot oc ol a me n d me nt.  Lastl y, t he S R T ma y deci de t o halt escalati o n of t he ut o mil u ma b d ose 
le vels at t he ti me of eac h S R T meeti n g after re vie w of t he data, e ve n i n t he a bse nce of D L T, if it 
dee ms n o f urt her be nefit w o ul d be deri ve d wit h hi g her d oses of ut o mil u ma b.
As part of its o versi g ht of t he st u d y, t he S R T will als o meet 1 ti me after 6 s u bjects ha ve bee n 
treate d wit h a xica bta ge ne cil ole ucel a n d ut o mil u ma b i n t he P hase 2 p orti o n of t he st u d y a n d ha ve 
c o m plete d t he Wee k 4 disease assess me nt.  T he S R T will re vie w safet y a n d efficac y data a n d 
will be c hartere d t o ma ke trial c o n d uct rec o m me n dati o ns base d o n a n a nal ysis of be nefit/ris k 
rati o.  T he S R T ma y meet m ore ofte n as nee de d.  
F or b ot h P hase 1 a n d P hase 2 p orti o ns of t he st u d y, i n cases w here a c o nse ns us rec o m me n dati o n 
is n ot reac he d b y t he S R T, t he S p o ns or will ser ve as t he fi nal decisi o n- ma ki n g b o d y f or f urt her 
st u d y c o n d uct.


A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 7 8
0 7 J u ne 2 0 2 2
1 0.  
S T A TI S TI C A L C O N SI D E R A TI O N S
1 0. 1.   
H y p ot hesis
N o f or mal h y p ot hesis will be teste d i n t his st u d y.  T he P hase 2 p orti o n of t he st u d y is desi g ne d t o 
esti mate t he tr ue C R rate i n s u bjects wit h rela pse d/refract or y lar ge B-cell l y m p h o ma treate d wit h 
t he a xica bta ge ne cil ole ucel f oll o we d b y ut o mil u ma b.
1 0. 2.  
St u d y E n d p oi nts
1 0. 2. 1.  
Pri m ar y E n d p oi nts

P hase 1: I nci de nce of A Es defi ne d as D L Ts.

P hase 2: C o m plete res p o nse rate ( C R per t he L u ga n o Classificati o n { C hes o n 2 0 1 4 }), as 
deter mi ne d b y t he st u d y i n vesti gat ors.
1 0. 2. 2.  
Sec o n d ar y E n d p oi nts
Sec o n dar y e n d p oi nts are a p plica ble t o b ot h P hase 1 a n d P hase 2:

O bjecti ve res p o nse rate: O bjecti ve res p o nse rate is defi ne d as t he i nci de nce of eit her a C R or 
a P R per L u ga n o Classificati o n { C hes o n 2 0 1 4 } as deter m i ne d b y st u d y i n vesti gat ors.  All 
s u bjects w h o d o n ot meet t he criteria f or a n o bjecti ve res p o nse b y t he a nal ysis data c ut off 
date will be c o nsi dere d n o n-res p o n ders.

D urati o n of res p o nse: A m o n g s u bjects w h o e x perie nce a n o bjecti ve res p o nse, D O R is 
defi ne d as t he date of t heir first o bjecti ve res p o nse t o disease pr o gressi o n per L u ga n o 
Classificati o n { C hes o n 2 0 1 4 }, as deter m i ne d b y st u d y i n vesti gat ors, or deat h fr o m a n y ca use. 
Data fr o m t he  retreat me nt peri o d will n ot be i ncl u de d f or a nal ysis.  S u bjects n ot meeti n g t he 
criteria f or pr o gressi o n or deat h b y t he a nal ysis data c ut off date will be ce ns ore d at t heir last 
e val ua ble disease assess me nt date, a n d t heir res p o nse will be n ote d as o n g oi n g.  S u bjects 
w h o recei ve a d diti o nal a nti-ca ncer t hera p y i n t he a bse nce of d oc u me nte d pr o gressi o n will be 
ce ns ore d at t he last e val ua ble disease assess me nt pri or t o t he a d diti o nal t hera p y.  S u bjects 
w h o recei ve S C T i n t he a bse nce of d oc u me nte d pr o gressi o n will be ce ns ore d at t he last 
e val ua ble disease assess me nt pri or t o t he date of t he S C T.  A se nsiti vit y a nal ysis will be 
c o n d ucte d i n w hic h disease assess me nts o btai ne d after S C T are i ncl u de d i n t he deri vati o n of 
D O R. 

Pr o gressi o n-free s ur vi val: Pr o gressi o n-free s ur vi val ( P F S) is defi ne d as t he ti me fr o m t he 
a xica bta ge ne cil ole ucel i nf usi o n date t o t he date of disease pr o gressi o n per L u ga n o 
Classificati o n { C hes o n 2 0 1 4 }, as deter m i ne d b y st u d y i n vesti gat ors, or deat h fr o m a n y ca use.  
Data fr o m t he retreat me nt peri o d will n ot be i ncl u de d f or a nal ysis.  S u bjects n ot meeti n g t he 
criteria f or pr o gressi o n or deat h b y t he a nal ysis data c ut off date will be ce ns ore d at t heir last 
e val ua ble disease assess me nt date.  T he P F S f or s u bjects w h o  u n der g o S C T w hile i n 




A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 8 0
0 7 J u ne 2 0 2 2
A d diti o nal ass u m pti o ns a n d c orres p o n di n g t w o-si de d 9 5 % a n d 8 0 % e xact CIs are pr o vi de d  i n 
Ta ble 8 .
T a ble 8.
E x act C o nfi de nce I nter v als ( 9 5 % a n d 8 0 %) C orres p o n di n g t o 
O bser ve d C R R ate F oll o wi n g Tre at me nt of 2 7 S u bjects wit h 
A xic a bt a ge ne Cil ole ucel a n d Ut o mil u m a b
S u bjects wit h C R 
O bser ve d C R R ate 
9 5 % C o nfi de nce I nter v al 
8 0 % C o nfi de nce I nter v al
1 5
5 6 %
[ 3 5 %, 7 5 %]
[ 4 2 %, 6 9 %]
1 7
6 3 %
[ 4 2 %, 8 1 %]
[ 4 9 %, 7 6 %]
1 9
7 0 %
[ 5 0 %, 8 6 %]
[ 5 6 %, 8 2 %]
2 1
7 8 %
[ 5 8 %, 9 1 %]
[ 6 4 %, 8 8 %]
2 3
8 5 %
[ 6 6 %, 9 6 %]
[ 7 3 %, 9 3 %]
A b bre viati o ns: C R, c o m plete res p o nse. 
1 0. 4.  
I nteri m A n al ysis a n d E arl y St o p pi n g G ui deli nes
F or mal i nteri m a nal yses of efficac y are n ot pla n ne d f or t he earl y trial st o p pi n g p ur p ose.
1 0. 4. 1.  
S afet y I nteri m A n al ysis
A n S R T will be c hartere d t o re vie w safet y d uri n g P hase 1 of t he st u d y o nl y a n d t o ma ke 
rec o m me n dati o ns o n f urt her st u d y c o n d uct i n P hase 1 a n d pr o gressi o n t o P hase 2.
T he S R T will a d diti o nall y meet o n at least o ne occasi o n d uri n g t he P hase 2 p orti o n of t he st u d y 
after 6 s u bjects ha ve c o m plete d t heir 1- m o nt h disease assess me nt.  T he S R T will re vie w safet y 
a n d efficac y data a n d will be c hartere d t o ma ke trial c o n d uct rec o m me n dati o ns base d o n a n 
a nal ysis of be nefit vs ris k.
1 0. 5.   
A n al ysis S u bsets  
F ull a nal ysis set ( F A S):  T he F A S will c o nsist of all e nr olle d s u bjects a n d will be use d f or 
s u m maries of s u bject dis p ositi o n.
M o difie d i nte nt-t o-treat set ( mI T T): T he mI T T will c o nsist of all s u bjects e nr olle d a n d treate d 
wit h t he tar get d ose of a xica bta ge ne cil ole ucel, 2 x 1 0 6 C A R T cells/ k g (ra n ge 1 x 1 06 t o 2. 4 x 
1 0 6 C A R T cells/ k g), a n d at least 1 d ose of ut o mil u ma b as deter mi ne d u p o n c o m pleti o n of t he 
P hase 1 a n d P hase 2 p orti o ns of t he st u d y.  T his a nal ysis set will be use d f or all efficac y 
a nal yses.
Safet y a nal ysis set: T he safet y a nal ysis set will c o nsist of all s u bjects treate d wit h a n y d ose of 
a xica bta ge ne cil ole ucel.


A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 8 1
0 7 J u ne 2 0 2 2
T he D L T e val ua ble set: T he D L T e val ua ble set will c o nsist of all s u bjects i n eac h P hase 1 c o h ort 
w h o are treate d wit h a xica bta ge ne cil ole ucel a n d at least 1 d ose of ut o mil u ma b w h o eit her:

Recei ve d t he tar get a xica bta ge ne cil ole ucel d ose a n d were f oll o we d f or at least 2 8 da ys after 
t he first ut o mil u ma b i nf usi o n

Recei ve d a d ose of a nti- C D 1 9 C A R T cells l o wer t ha n t he tar get f or t hat c o h ort a n d a 
s u bse q ue nt ut o mil u ma b i nf usi o n a n d e x perie nce d a D L T d uri n g t he 2 8- da y, p ost- ut o mil u ma b 
i nf usi o n peri o d
1 0. 6.  
Pl a n ne d Met h o d of A n al ysis
T he pri mar y a nal ysis will be perf or me d w he n t he last treate d s u bject i n t he mI T T set has ha d t he 
o p p ort u nit y t o be e val uate d f or res p o nse 6 m o nt hs after t he Wee k 4 disease assess me nt.  T he 
fi nal a nal ysis will occ ur w he n all s u bjects ha ve c o m plete d t he st u d y.
1 0. 6. 1.   
C o m plete Res p o nse R ate 
T he i nci de nce of c o m plete res p o nse a n d e xact 2-si de d 9 5 % CIs will be ge nerate d.
1 0. 6. 2.   
O bjecti ve Res p o nse R ate
T he i nci de nce of o bjecti ve res p o nse a n d e xact 2-si de d 9 5 % CIs will be ge nerate d.
1 0. 6. 3.   
D ur ati o n of Res p o nse
Ka pla n- Meier esti mates a n d 2-si de d 9 5 % CIs will be ge nerate d f or D O R.  Esti mates of t he 
pr o p orti o n of s u bjects ali ve a n d i n res p o nse at 3- m o nt h i nter vals will be pr o vi de d.
1 0. 6. 4.  
Pr o gressi o n-free S ur vi v al
Ka pla n- Meier esti mates a n d 2-si de d 9 5 % CIs will be ge nerate d f or P F S.  Esti mates of t he 
pr o p orti o n of s u bjects ali ve a n d pr o gressi o n-free at 3- m o nt h i nter vals will be pr o vi de d.
1 0. 6. 5.   
O ver all S ur vi v al
Ka pla n- Meier esti mates a n d 2-si de d 9 5 % CIs will be ge nerate d f or O S.  Esti mates of t he 
pr o p orti o n of s u bjects ali ve at 3- m o nt h i nter vals will be pr o vi de d.


A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 8 2
0 7 J u ne 2 0 2 2
1 0. 6. 6.  
S afet y
S u bject i nci de nce rates of A Es, i ncl u di n g all, seri o us, fatal, C T C A E Gra de 3 or hi g her, a n d 
treat me nt-relate d A Es re p orte d t hr o u g h o ut t he c o n d uct of t he st u d y will be ta b ulate d b y preferre d 
ter m a n d s yste m or ga n class.  C ha n ges i n la b orat or y val ues a n d vital si g ns will be s u m marize d 
wit h descri pti ve statistics.  T he i nci de nce of c o nc o mita nt me dicati o ns will be s u m marize d.
Ta bles a n d/ or narrati ves of deat hs t hr o u g h t he L T F U peri o d a n d treat me nt relate d S A Es will be 
pr o vi de d.
1 0. 6. 7.  
L o n g-ter m D at a A n al ysis
All s u bjects will be f oll o we d f or s ur vi val stat us f or u p t o a p pr o xi matel y 1 5 years after recei vi n g 
a xica bta ge ne cil ole ucel. L T F U data a nal ysis will be perf or me d o n s u bjects i n t his st u d y a n d after 
tra nsiti o n t o t he K T- U S- 9 8 2- 5 9 6 8 L T F U st u d y. N o f or mal h y p ot hesis testi n g will be perf or me d 
base d o n data o btai ne d after t he c ut off f or t he pri mar y a nal ysis.  Descri pti ve esti mates of ke y 
efficac y a n d safet y a nal yses ma y be u p date d t o assess t he o verall treat me nt pr ofile.


A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 8 3
0 7 J u ne 2 0 2 2
1 1.   
R E G U L A T O R Y O B LI G A TI O N S 
T his st u d y will be c o n d ucte d i n acc or da nce wit h t he pr ot oc ol a n d wit h t he f oll o wi n g:

C o nse ns us et hical pri nci ples deri ve d fr o m i nter nati o nal g ui deli nes i ncl u di n g t he Declarati o n 
of Helsi n ki

A p plica ble I nter nati o nal C o nfere nce o n Har m o nisati o n (I C H) G o o d Cli nical Practice ( G C P) 
G ui deli nes

A p plica ble la ws a n d re g ulati o ns
1 1. 1.  
I n de pe n de nt Re vie w B o ar d/I n de pe n de nt Et hics C o m mittee 
A c o p y of t he pr ot oc ol, I C F, a n d a n y a d diti o nal s u bject or trial i nf or mati o n, s uc h as s u bject 
recr uit me nt materials, m ust be s u b mitte d t o eac h site’s res pecti ve I R B/I E C f or a p pr o val.  After 
a p pr o val is o btai ne d fr o m t he I R B/I E C, all d oc u me nts m ust be pr o vi de d t o t he ke y s p o ns or 
c o ntact bef ore s u bject recr uit me nt ca n be gi n.
T he i n vesti gat or m ust als o recei ve I R B/I E C a p pr o val f or all pr ot oc ol a n d I C F c ha n ges or 
a me n d me nts.  I n vesti gat ors m ust e ns ure t hat o n g oi n g/c o nti n u o us I R B/I E C a p pr o val (ie, a n n ual 
a p pr o val) is pr o vi de d t hr o u g h o ut t he c o n d uct of t he st u d y.  C o pies of I R B/I E C a p pr o val are t o be 
f or war de d t o t he ke y s p o ns or c o ntact f or arc hi vi n g.
D uri n g t he c o urse of t he st u d y, i n vesti gat ors are t o s u b mit site-s pecific a n d st u d y S A Es 
( pr o vi de d t o t he site b y t he ke y s p o ns or c o ntact), al o n g wit h a n y pr ot oc ol de viati o ns, t o t heir 
I R B/I E C i n acc or da nce wit h t heir res pecti ve I R B/I E C p olicies. 
1 1. 2.  
S u bject C o nfi de nti alit y
S u bject c o nfi de ntialit y m ust be mai ntai ne d wit hi n all material t hat is s u b mitte d t o t he ke y 
s p o ns or c o ntact.  T he f oll o wi n g r ules are t o be a p plie d.

S u bjects will be i de ntifie d b y a u ni q ue I D n u m ber.

Year of birt h/a ge at ti me of e nr oll me nt will be re p orte d acc or di n g t o l ocal la ws a n d 
re g ulati o ns.
F or re p orti n g of S A Es, s u bjects will be i de ntifie d b y t heir res pecti ve s u bject I D n u m ber, i nitials, 
a n d year of birt h (as per t heir l ocal re p orti n g re q uire me nts f or b ot h i nitials a n d year of birt h).
Per c o u ntr y-s pecific re g ulati o ns a n d I C H/ G C P g ui deli nes, i n vesti gat ors a n d i nstit uti o ns are 
re q uire d t o per mit a ut h orizati o n t o t he s p o ns or, C o ntract Researc h Or ga nizati o n ( C R O), 
I R B/I E C, a n d re g ulat or y a ge ncies t o s u bject’s ori gi nal s o urce d oc u me nts f or verificati o n of st u d y 
data.  T he i n vesti gat or is res p o nsi ble f or i nf or mi n g p ote ntial s u bjects t hat s uc h i n di vi d uals will 
ha ve access t o t heir me dical rec or ds, w hic h i ncl u des pers o nal i nf or mati o n.


A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 8 4
0 7 J u ne 2 0 2 2
1 1. 3.  
I n vesti g at or Si g n at or y O bli g ati o ns
Eac h cli nical st u d y re p ort will be si g ne d b y t he c o or di nati n g i n vesti gat or.  T he c o or di nati n g 
i n vesti gat or will be i de ntifie d b y Kite P har ma, I nc., base d o n t he f oll o wi n g criteria:

Is a rec o g nize d e x pert i n t he disease setti n g

Pr o vi de d si g nifica nt c o ntri b uti o ns t o t he desi g n or a nal ysis of st u d y data

Partici pate d i n t he st u d y a n d e nr olle d a hi g h n u m ber of eli gi ble s u bjects


A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 8 5
0 7 J u ne 2 0 2 2
1 2.  
P R O T O C O L A M E N D M E N T S A N D T E R MI N A TI O N
If t he pr ot oc ol is a me n de d, t he i n vesti gat or’s a gree me nt wit h t he a me n d me nt a n d t he I R B/I E C 
a p pr o val of t he a me n d me nt m ust be o btai ne d.  D oc u me ntati o n ac k n o wle d gi n g a p pr o val fr o m 
b ot h parties are t o be s u b mitte d t o t he ke y s p o ns or c o ntact.
B ot h Kite P har ma, I nc., a n d t he i n vesti gat or reser ve t he ri g ht t o ter mi nate t he i n vesti gat or’s 
partici pati o n i n t he st u d y as per t he ter ms of t he a gree me nt i n t he st u d y c o ntract.  T he 
i n vesti gat or is t o pr o vi de writte n c o m m u nicati o n t o t he I R B/I E C re gar di n g eit her t he trial 
c o m pleti o n or earl y ter mi nati o n a n d pr o vi de t he C R O wit h a c o p y of t he c orres p o n de nce.
Kite P har ma, I nc., reser ves t he u nilateral ri g ht, at its s ole discreti o n, t o deter mi ne w het her t o 
ma n ufact ure a xica bta ge ne cil ole ucel a n d pr o vi de it t o sites a n d s u bjects after t he c o m pleti o n of 
t he st u d y.


A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 8 6
0 7 J u ne 2 0 2 2
1 3.  
S T U D Y D O C U M E N T A TI O N A N D A R C HI VI N G
T he i n vesti gat or will mai ntai n a list of q ualifie d staff t o w h o m st u d y res p o nsi bilities ha ve bee n 
dele gate d.  T he i n di vi d uals a ut h orize d t o f ulfill t hese res p o nsi bilities s h o ul d be o utli ne d a n d 
i ncl u de d i n t he Dele gati o n of A ut h orit y F or m.
S o urce d oc u me nts are ori gi nal d oc u me nts, data, a n d rec or ds f or w hic h t he st u d y data are 
c ollecte d a n d verifie d.  E xa m ples of s uc h s o urce d oc u me nts ma y i ncl u de, b ut are n ot li mite d t o, 
h os pital rec or ds a n d patie nt c harts; la b orat or y, p har mac y, ra di ol o g y rec or ds; s u bject diaries; 
micr ofic hes; c orres p o n de nce; a n d deat h re gistries.  C R F e ntries ma y be c o nsi dere d as s o urce data 
if t he site of t he ori gi nal data c ollecti o n is n ot a vaila ble.  H o we ver, t he use of t he C R Fs as s o urce 
d oc u me ntati o n is n ot rec o m me n de d as a r o uti ne practice.
T he i n vesti gat or a n d st u d y staff are res p o nsi ble f or mai ntai ni n g a c o m pre he nsi ve a n d ce ntralize d 
fili n g s yste m of all s u bject rec or ds t hat are rea dil y retrie ve d t o be m o nit ore d a n d or a u dite d at 
a n y ti me b y t he ke y s p o ns or c o ntact, healt h a ut h orities, a n d I R B/I E Cs.  T he fili n g s yste m will 
i ncl u de at mi ni m u m:

S u bject c o nte nt i ncl u di n g I C Fs a n d s u bject i de ntificati o n lists

Pr ot oc ols a n d pr ot oc ol a me n d me nts, I B, c o pies of pre-st u d y d oc u me ntati o n, a n d all I R B/I E C 
a n d s p o ns or c o m m u nicati o n

Pr o of of recei pt, e x peri me ntal treat me nt fl o w rec or ds, a n d e x peri me ntal pr o d uct-relate d 
c orres p o n de nce.
Ori gi nal s o urce d oc u me nts s u p p orti n g e ntries i nt o C R Fs m ust be mai ntai ne d at t he site a n d 
rea dil y a vaila ble u p o n re q uest.  N o st u d y d oc u me nts s h o ul d be discar de d wit h o ut pri or writte n 
a gree me nt bet wee n Kite P har ma, I nc., a n d t he i n vesti gat or.  If st ora ge is n o l o n ger a vaila ble t o 
arc hi ve s o urce d oc u me nts or if s o urce d oc u me nts m ust be m o ve d t o a n alter nati ve l ocati o n, t he 
researc h staff s h o ul d n otif y t he ke y s p o ns or c o ntact pri or t o s hi p pi n g t he d oc u me nts.


A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 8 7
0 7 J u ne 2 0 2 2
1 4.  
S T U D Y M O NI T O RI N G A N D D A T A C O L L E C TI O N
T he ke y s p o ns or c o ntact, m o nit ors, a u dit ors, or re g ulat or y i ns pect ors are res p o nsi ble f or 
c o ntacti n g a n d visiti n g t he i n vesti gat or f or t he p ur p ose of i ns pecti n g t he facilities a n d verif yi n g 
s o urce d oc u me nts a n d rec or ds m ust als o ass ure t hat s u bject c o nfi de ntiall y is res pecte d.
T he m o nit or is res p o nsi ble f or s o urce d oc u me nt verificati o n of C R F data at re g ular i nter vals 
d uri n g t he st u d y.  Pr ot oc ol a d here nce a n d acc urac y a n d c o nsiste nc y of st u d y c o n d uct a n d data 
c ollecti o n wit h res pect t o l ocal re g ulati o ns will be c o nfir me d.  M o nit ors will ha ve access t o 
s u bject rec or ds as i de ntifie d i n Secti o n 1 3 .
B y si g ni n g t he i n vesti gat or’s a gree me nt, t he i n vesti gat or a grees t o c o o perate wit h t he m o nit or t o 
a d dress a n d res ol ve iss ues i de ntifie d d uri n g m o nit ori n g visits.
I n acc or da nce wit h I C H G C P a n d t he a u dit pla n, a site ma y be c h ose n f or a site a u dit.  A site 
a u dit w o ul d i ncl u de, b ut is n ot li mite d t o, a n i ns pecti o n of t he facilit y(ies), re vie w of s u bject- a n d 
st u d y-relate d rec or ds, a n d c o m plia nce wit h pr ot oc ol re q uire me nts as well as I C H G C P a n d 
a p plica ble re g ulat or y p olicies.
All data will be c ollecte d i n a n electr o nic C R F s yste m.  All e ntries m ust be c o m plete d i n E n glis h, 
a n d c o nc o mita nt t hera pies s h o ul d be i de ntifie d b y tra de na mes.  F or f urt her details s urr o u n di n g 
t he c o m pleti o n of C R Fs, refer t o t he C R F c o m pleti o n g ui deli nes.


A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 8 8
0 7 J u ne 2 0 2 2
1 5.  
P U B LI C A TI O N
A ut h ors hi p of p u blicati o ns fr o m data ge nerate d i n K T E- C 1 9- 1 1 1 will be deter mi ne d base d o n 
t he u nif or m re q uire me nts f or ma n uscri pts s u b mitte d t o bi o me dical j o ur nals (as o utli ne d i n t he 
I nter nati o nal C o m mittee of Me dical J o ur nal E dit ors Dece m ber 2 0 1 3), w hic h states t hat 
a ut h ors hi p s h o ul d be base d o n: 

S u bsta ntial c o ntri b uti o ns t o t he c o nce pti o n or desi g n of t he w or k, ac q uisiti o n of data, 
a nal ysis, or i nter pretati o n of data f or t he w or k; a n d

Drafti n g t he article or re visi n g it criticall y f or i m p orta nt i ntellect ual c o nte nt; a n d

Fi nal a p pr o val of t he versi o n t o be p u blis he d; a n d

A gree me nt t o be acc o u nta ble f or all as pects of t he w or k i n e ns uri n g t hat q uesti o ns relate d t o 
t he acc urac y or i nte grit y of a n y part of t he w or k be a p pr o priatel y i n vesti gate d or res ol ve d
W he n a lar ge, m ultice nter gr o u p has c o n d ucte d t he w or k, t he gr o u p s h o ul d i de ntif y t he 
i n di vi d uals w h o acce pt direct res p o nsi bilit y f or t he ma n uscri pt.  T his i n di vi d ual s h o ul d f ull y 
meet t he criteria f or a ut h ors hi p defi ne d a b o ve.
F u n di n g, c ollecti o n of data, or ge neral s u per visi o n of t he researc h al o ne or i n c o m bi nati o n d oes 
n ot q ualif y a n i n di vi d ual f or a ut h ors hi p.
A n y p u blicati o n, i n a n y f or m, t hat is deri ve d fr o m t his st u d y m ust be s u b mitte d t o Kite P har ma, 
I nc., f or re vie w a n d a p pr o val.  T he st u d y c o ntract a m o n g t he i nstit uti o n, pri nci pal i n vesti gat or, 
a n d Kite P har ma, I nc., or its dele gate will o utli ne t he re q uire me nts f or p u blicati o n re vie w.


A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 8 9
0 7 J u ne 2 0 2 2
1 6.   
R E F E R E N C E S
Barri n gt o n S F, Mi k haeel N G, K osta k o gl u L, Mei g na n M, H utc hi n gs M, M ueller S P, et al. R ole 
of i ma gi n g i n t he sta gi n g a n d res p o nse assess me nt of l y m p h o ma: c o nse ns us of t he 
I nter nati o nal C o nfere nce o n Mali g na nt L y m p h o mas I ma gi n g W or ki n g Gr o u p. J 
Cli n O nc ol 2 0 1 4; 3 2 ( 2 7): 3 0 4 8- 5 8.
B ot A, K oc he n derfer J, Mar dir os A, Perez A, Na vale L, C ha n g R, et al. Bi o mar ker a nal ysis of 
patie nts treate d wit h a nti- C D 1 9 c hi meric a nti ge n rece pt or ( C A R) T cells [ A bstract 
3 0 2 8]. A merica n S ociet y of Cli nical O nc ol o g y ( A S C O) A n n ual Meeti n g; 2 0 1 5 2 9 
Ma y- 0 2 J u ne; C hica g o, I L. 
Castr o J E, Ja mes D F, Sa n d o val- S us J D, Jai n S, B ole J, Rasse nti L, et al. Rit u xi ma b i n 
c o m bi nati o n wit h hi g h- d ose met h yl pre d nis ol o ne f or t he treat me nt of c hr o nic 
l y m p h oc ytic le u ke mia. Le u ke mia 2 0 0 9; 2 3 ( 1 0): 1 7 7 9- 8 9.
C hes o n B D, Fis her RI, Barri n gt o n S F, Ca valli F, Sc h wartz L H, Z ucca E, et al. 
Rec o m me n dati o ns f or i nitial e val uati o n, sta gi n g, a n d res p o nse assess me nt of 
H o d g ki n a n d n o n- H o d g ki n l y m p h o ma: t he L u ga n o classificati o n. J Cli n O nc ol 
2 0 1 4; 3 2 ( 2 7): 3 0 5 9- 6 8.
Cli nical Trials Facilitati o n Gr o u p ( C T F G). Rec o m me n dati o ns Relate d t o C o ntrace pti o n a n d 
Pre g na nc y Testi n g i n Cli nical Trials. Fi nal Versi o n. 1 5 Se pte m ber.  2 0 1 4.
Cr oft M. T he r ole of T N F s u perfa mil y me m bers i n T-cell f u ncti o n a n d diseases. Nat Re v 
I m m u n ol 2 0 0 9; 9 ( 4): 2 7 1- 8 5.
Cr u m p M, Neela p u S S, Far o o q U, Va n De n Neste E, K ur u villa J, Westi n J, et al. O utc o mes i n 
refract or y diff use lar ge B-cell l y m p h o ma: res ults fr o m t he i nter nati o nal 
S C H O L A R- 1 st u d y. Bl o o d 2 0 1 7; 1 3 0 ( 1 6): 1 8 0 0- 8.
D u dle y M E, Ya n g J C, S herr y R, H u g hes M S, R o yal R, Ka m m ula U, et al. A d o pti ve cell t hera p y 
f or patie nts wit h metastatic mela n o ma: e val uati o n of i nte nsi ve m yel oa blati ve 
c he m ora diati o n pre parati ve re gi me ns. J Cli n O nc ol 2 0 0 8; 2 6 ( 3 2): 5 2 3 3- 9.
Fis her T S, Ka m persc hr oer C, Oli p ha nt T, L o ve V A, Lira P D, D o y o n nas R, et al. Tar geti n g of 4-
1 B B b y m o n ocl o nal a nti b o d y P F- 0 5 0 8 2 5 6 6 e n ha nces T-cell f u ncti o n a n d 
pr o m otes a nti-t u m or acti vit y. Ca ncer I m m u n ol I m m u n ot her 2 0 1 2; 6 1 ( 1 0): 1 7 2 1-
3 3.
Fl o wers C R, Si n ha R, V ose J M. I m pr o vi n g o utc o mes f or patie nts wit h diff use lar ge B-cell 
l y m p h o ma. C A Ca ncer J Cli n 2 0 1 0; 6 0 ( 6): 3 9 3- 4 0 8.
Gatti n o ni L, Fi n kelstei n S E, Kle ba n off C A, A nt o n y P A, Pal mer D C, S piess PJ, et al. Re m o val of 
h o me ostatic c yt o ki ne si n ks b y l y m p h o de pleti o n e n ha nces t he efficac y of 
a d o pti vel y tra nsferre d t u m or-s pecific C D 8 + T cells. J E x p Me d 2 0 0 5; 2 0 2 ( 7): 9 0 7-
1 2.


A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 9 0
0 7 J u ne 2 0 2 2
G o pal A K, Bartlett N L, Le v y R, H o u ot R, S mit h S D, Se gal N H, et al. A p hase I st u d y of P F-
0 5 0 8 2 5 6 6 (a nti- 4- 1 B B) + rit u xi ma b i n patie nts wit h C D 2 0 + N H L. J Cli n O nc ol 
2 0 1 5; 3 3 ( 1 5 _s u p pl): 3 0 0 4-.
H urta d o J C, Ki m YJ, K w o n B S. Si g nals t hr o u g h 4- 1 B B are c osti m ulat or y t o pre vi o usl y acti vate d 
s ple nic T cells a n d i n hi bit acti vati o n-i n d uce d cell deat h. J I m m u n ol 1 9 9 7; 1 5 8 
( 6): 2 6 0 0- 9.
Keir M E, B utte MJ, Free ma n GJ, S har pe A H. P D- 1 a n d its li ga n ds i n t olera nce a n d i m m u nit y. 
A n n u Re v I m m u n ol 2 0 0 8; 2 6: 6 7 7- 7 0 4.
Kle ba n off C A, K h o n g H T, A nt o n y P A, Pal mer D C, Restif o N P. Si n ks, s u p press ors a n d a nti ge n 
prese nters: h o w l y m p h o de pleti o n e n ha nces T cell- me diate d t u m or 
i m m u n ot hera p y. Tre n ds I m m u n ol 2 0 0 5; 2 6 ( 2): 1 1 1- 7.
Lee D W, Gar d ner R, P orter D L, L o uis C U, A h me d N, Je nse n M, et al. C urre nt c o nce pts i n t he 
dia g n osis a n d ma na ge me nt of c yt o ki ne release s y n dr o me. Bl o o d 2 0 1 4; 1 2 4 
( 2): 1 8 8- 9 5.
Lee H W, Na m K O, Se o S K, Ki m Y H, Ka n g H, K w o n B S. 4- 1 B B cr oss-li n ki n g e n ha nces t he 
s ur vi val a n d cell c ycle pr o gressi o n of C D 4 T l y m p h oc ytes. Cell I m m u n ol 
2 0 0 3; 2 2 3 ( 2): 1 4 3- 5 0.
Lee H W, Par k SJ, C h oi B K, Ki m H H, Na m K O, K w o n B S. 4- 1 B B pr o m otes t he s ur vi val of 
C D 8 + T l y m p h oc ytes b y i ncreasi n g e x pressi o n of Bcl- x L a n d Bfl- 1. J I m m u n ol 
2 0 0 2; 1 6 9 ( 9): 4 8 8 2- 8.
Le o nar d J P, Sc hatt ner EJ, C ole ma n M. Bi ol o g y a n d ma na ge me nt of ma ntle cell l y m p h o ma. C urr 
O pi n O nc ol 2 0 0 1; 1 3 ( 5): 3 4 2- 7.
L oc ke F L, Westi n J R, Mi kl os D B, Herrara A F, Jac o bs o n C A, Lee J, et al. P hase 1 Res ults fr o m 
Z U M A- 6: A xica bta ge ne Cil ole ucel (a xi-cel; K T E- C 1 9) i n C o m bi nati o n wit h 
Atez oliz u ma b f or t he Treat me nt of Patie nts wit h Refract or y Diff use Lar ge B Cell 
L y m p h o ma ( D L B C L). Bl o o d 2 0 1 7; 1 3 0 ( S u p pl 1): 2 8 2 6-.
Neela p u S S, L oc ke F L, Bartlett N L, Le ka kis LJ, Mi kl os D B, Jac o bse n E D, et al. L o n g- Ter m 
F oll o w- u p Z U M A- 1: A Pi v otal Trial of A xica bta ge ne Cil ole ucel ( A xi- Cel; K T E-
C 1 9) i n Patie nts wit h Refract or y A g gressi ve N o n- H o d g ki n L y m p h o ma ( N H L). 
Bl o o d 2 0 1 7a; 1 3 0 ( S u p pl 1): 5 7 8-.
Neela p u S S, L oc ke F L, Bartlett N L, Le ka kis LJ, Mi kl os D B, Jac o bs o n C A, et al. A xica bta ge ne 
Cil ole ucel C A R T- Cell T hera p y i n Refract or y Lar ge B- Cell L y m p h o ma. N E n gl J 
Me d 2 0 1 7 b; 3 7 7 ( 2 6): 2 5 3 1- 4 4.
Nic h ols o n I C, Le nt o n K A, Little DJ, Dec ors o T, Lee F T, Sc ott A M, et al. C o nstr ucti o n a n d 
c haracterisati o n of a f u ncti o nal C D 1 9 s pecific si n gle c hai n F v fra g me nt f or 
i m m u n ot hera p y of B li nea ge le u kae mia a n d l y m p h o ma. M ol I m m u n ol 1 9 9 7; 3 4 
( 1 6- 1 7): 1 1 5 7- 6 5.


A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 9 1
0 7 J u ne 2 0 2 2
N o o ne A, H o wla der N, Kra pc h o M, Miller D, Brest A, Y u M, et al. S E E R Ca ncer Statistics 
Re vie w, 1 9 7 5- 2 0 1 5. Nati o nal Ca ncer I nstit ute. Bet hes da, M D, 
htt ps://seer.ca ncer. g o v/csr/ 1 9 7 5 _ 2 0 1 5/, base d o n N o ve m ber 2 0 1 7 S E E R data 
s u b missi o n, p oste d t o t he S E E R we b site, A pril 2 0 1 8. 2 0 1 7.
O' Brie n S M, Ka ntarjia n H M, C ortes J, Bera n M, K oller C A, Giles FJ, et al. Res ults of t he 
fl u dara bi ne a n d c ycl o p h os p ha mi de c o m bi nati o n re gi me n i n c hr o nic l y m p h oc ytic 
le u ke mia. J Cli n O nc ol 2 0 0 1; 1 9 ( 5): 1 4 1 4- 2 0.
Olej nicza k S H, Ste wart C C, D o n o h ue K, Cz ucz ma n M S. A q ua ntitati ve e x pl orati o n of s urface 
a nti ge n e x pressi o n i n c o m m o n B-cell mali g na ncies usi n g fl o w c yt o metr y. 
I m m u n ol I n vest 2 0 0 6; 3 5 ( 1): 9 3- 1 1 4.
Perez A, Na vale L, R ossi J M, S he n Y- w, Jia n g Y, S her ma n M, et al. P har mac o d y na mic Pr ofile 
a n d Cli nical Res p o nse i n Patie nts wit h B- Cell Mali g na ncies of A nti- C D 1 9 C A R 
T- Cell T hera p y. Bl o o d 2 0 1 5; 1 2 6 ( 2 3): 2 0 4 2.
R o dri g uez J, P u g h W C, R o ma g uera J E, Ca ba nillas F. Pri mar y me diasti nal lar ge cell l y m p h o ma. 
He mat ol o gical O nc ol o g y 1 9 9 4; 1 2 ( 4): 1 7 5- 8 4.
R ossi J M, Gal o n J, T urca n S, Da na n C, L oc ke F L, Neela p u S S, et al. C haracteristics of t he 
pretreat me nt t u m or micr oe n vir o n me nt ma y i nfl ue nce cli nical res p o nse i n patie nts 
wit h refract or y lar ge B cell l y m p h o ma treate d wit h a xica bta ge ne cil ole ucel (a xi-
cel) i n t he pi v otal Z U M A- 1 [ A bstract L B- 0 1 6/ 1 6]. A merica n Ass ociati o n f or 
C nacer Researc h ( A A C R); 2 0 1 8 1 4- 1 8 A pril; C hica g o, Illi n ois. 
Se gal N H, He A R, D oi T, Le v y R, B hatia S, Pis h vaia n MJ, et al. P hase I St u d y of Si n gle- A ge nt 
Ut o mil u ma b ( P F- 0 5 0 8 2 5 6 6), a 4- 1 B B/ C D 1 3 7 A g o nist, i n Patie nts wit h A d va nce d 
Ca ncer. Cli n Ca ncer Res 2 0 1 8; 2 4 ( 8): 1 8 1 6- 2 3.
S h uf or d W W, Kl uss ma n K, Tritc hler D D, L o o D T, C hal u p n y J, Sia da k A W, et al. 4- 1 B B 
c osti m ulat or y si g nals prefere ntiall y i n d uce C D 8 + T cell pr oliferati o n a n d lea d t o 
t he a m plificati o n i n vi v o of c yt ot o xic T cell res p o nses. J E x p Me d 1 9 9 7; 1 8 6 
( 1): 4 7- 5 5.
S wer dl o w S H, Ca m p o E, Pileri S A, Harris N L, Stei n H, Sie bert R, et al. T he 2 0 1 6 re visi o n of t he 
W orl d Healt h Or ga nizati o n classificati o n of l y m p h oi d ne o plas ms. Bl o o d 
2 0 1 6; 1 2 7 ( 2 0): 2 3 7 5- 9 0.
Uc k u n F M, Jaszcz W, A m br us J L, Fa uci A S, Gajl- Peczals ka K, S o n g C W, et al. Detaile d St u dies 
o n E x pressi o n a n d F u ncti o n of C D 1 9 S urface Deter mi na nt b y Usi n g B 4 3 
M o n ocl o nal A nti b o d y a n d t he Cli nical P ote ntial of A nti- C D 1 9 I m m u n ot o xi ns. 
Bl o o d 1 9 8 8; 7 1 ( 1): 1 3- 2 9.
Wa n g C, Li n G H, Mc P hers o n AJ, Watts T H. I m m u ne re g ulati o n b y 4- 1 B B a n d 4- 1 B B L: 
c o m ple xities a n d c halle n ges. I m m u n ol Re v 2 0 0 9; 2 2 9 ( 1): 1 9 2- 2 1 5.
Y E S C A R T A, Kite P har ma I nc. Y E S C A R T A ® (a xica bta ge ne cil ole ucel) S us pe nsi o n f or 
I ntra ve n o us I nf usi o n. U. S. Prescri bi n g I nf or mati o n. Sa nta M o nica, C A.  2 0 1 7:


A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 9 2
0 7 J u ne 2 0 2 2
1 7.   
A P P E N DI C E S
A p pe n di x 1.  
S p o ns or a n d I n vesti gat or Si g nat ure Pa ge
A p pe n di x 2.  
L u ga n o Classificati o n { C hes o n 2 0 1 4 }
A p pe n di x 3.  
C hil d beari n g P ote ntial a n d Birt h C o ntr ol




A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 9 4
0 7 J u ne 2 0 2 2
A p pe n di x 2.  
L u g a n o Cl assific ati o n { C hes o n 2 0 1 4 }
5- P oi nt Sc ale ( 5 P S) { B arri n gt o n 2 0 1 4 }
Sc ore 
Descri pti o n 
1 
N o u pta ke a b o ve bac k gr o u n d 
2 
U pta ke ≤ me diasti n u m 
3 
U pta ke > me diasti n u m b ut ≤ li ver 
4 
U pta ke m o deratel y hi g her t ha n li ver 
5 
U pta ke mar ke dl y hi g her t ha n li ver a n d/ or ne w lesi o ns
X 
Ne w areas of u pta ke u nli kel y t o be relate d t o l y m p h o ma  
C o m plete Re missi o n:
C o m plete Meta b olic Res p o nse ( C M R) f or  P ositr o n E missi o n T o m o gra p h y- C o m p ute d 
T o m o gra p h y ( P E T- C T)- Base d Res p o nse
T he desi g nati o n of C M R re q uires all of t he f oll o wi n g:

A 5 P S ( 5- p oi nt scale) sc ore of 1, 2, or 3 wit h or wit h o ut a resi d ual mass 

I n Wal de yer’s ri n g or e xtra n o dal sites wit h hi g h p h ysi ol o gic u pta ke or wit h acti vati o n 
wit hi n s plee n or marr o w, u pta ke ma y be greater t ha n n or mal me diasti n u m a n d/ or li ver. I n 
t his circ u msta nce, C M R ma y be i nferre d if u pta ke at sites of i nitial i n v ol ve me nt is n o 
greater t ha n s urr o u n di n g n or mal tiss ue e ve n if t he tiss ue has hi g h p h ysi ol o gic u pta ke.

N o ne w sites of disease s h o ul d be o bser ve d

N o e vi de nce of fl u or o de o x y gl uc ose ( F D G)-a vi d disease i n b o ne marr o w
C o m plete Ra di ol o gic Res p o nse ( C R R) f or C o m p ute d T o m o gra p h y ( C T )- Base d Res p o nse 
T he desi g nati o n of C R R re q uires all of t he f oll o wi n g:

Tar get n o des/ n o dal masses m ust re gress t o ≤ 1. 5 c m i n l o n gest tra ns verse dia meter of a lesi o n 
( L Di)

N o e xtral y m p hatic sites of disease

A bse nt n o n- meas ure d lesi o n

Or ga n e nlar ge me nt re gress t o n or mal

N o ne w sites of disease s h o ul d be o bser ve d

B o ne marr o w n or mal b y m or p h ol o g y; if i n deter mi nate, i m m u n o hist oc he mistr y ne gati ve


A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 9 5
0 7 J u ne 2 0 2 2
P arti al Re missi o n: 
Partial Meta b olic Res p o nse ( P M R) f or P E T- C T- Base d Res p o nse
T he desi g nati o n of P M R re q uires all of t he f oll o wi n g:

A 5 P S sc ore of 4 or 5 wit h re d uce d u pta ke c o m pare d t o baseli ne (scree ni n g) a n d resi d ual 
mass(es) of a n y size 
N ote: 

At i nteri m, t hese fi n di n gs s u g gest res p o n di n g disease

At e n d of treat me nt, t hese fi n di n gs s u g gest resi d ual disease

N o ne w sites of disease s h o ul d be o bser ve d

Resi d ual u pta ke hi g her t ha n u pta ke i n n or mal b o ne marr o w b ut re d uce d c o m pare d wit h 
baseli ne ( diff use u pta ke c o m pati ble wit h reacti ve c ha n ges fr o m c he m ot hera p y all o we d)
If t here are persiste nt f ocal c ha n ges i n t he marr o w i n t he c o nte xt of a n o dal res p o nse, 
c o nsi derati o n s h o ul d be gi ve n t o f urt her e val uati o n wit h ma g netic res o na nce i ma gi n g bi o ps y, or 
a n i nter val sca n.
Partial Ra di ol o gic Res p o nse ( P R R) f or C T- Base d Res p o nse 
T he desi g nati o n of P R R re q uires all of t he f oll o wi n g:

≥ 5 0 % decrease i n s u m of t he pr o d uct of t he per pe n dic ular dia meters ( S P D) of u p t o 6 tar get 
meas ura ble n o des a n d e xtra n o dal sites

W he n a lesi o n is t o o s mall t o meas ure o n C T, assi g n 5 x 5 m m as t he defa ult val ue

W he n n o l o n ger visi ble, 0 x 0 m m

F or a n o de > 5 x 5 m m, b ut s maller t ha n n or mal, use t he act ual meas ure me nt f or calc ulati o n 

A bse nt/ n or mal, re gresse d, b ut n o i ncrease of n o n- meas ure d lesi o ns

S plee n m ust ha ve re gresse d b y > 5 0 % i n le n gt h be y o n d n or mal

N o ne w sites of disease s h o ul d be o bser ve d. 


A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 9 6
0 7 J u ne 2 0 2 2
St a ble Dise ase:
N o Meta b olic Res p o nse ( N M R) f or P E T- C T- Base d Res p o nse 
T he desi g nati o n of N M R re q uires all of t he f oll o wi n g:

A 5 P S sc ore of 4 or 5 wit h n o si g nifica nt c ha n ge i n F D G u pta ke c o m pare d t o baseli ne 
(scree ni n g) at a n i nteri m ti me p oi nt or at t he e n d of treat me nt 

N o ne w sites of disease s h o ul d be o bser ve d

N o c ha n ge fr o m baseli ne i n b o ne marr o w
Sta ble Ra di ol o gic Disease ( S R D) f or C T- Base d Res p o nse
T he desi g nati o n of S R D re q uires all of t he f oll o wi n g:

< 5 0 % decrease fr o m baseli ne i n t he S P D of u p t o 6 d o mi na nt meas ura ble n o des a n d 
e xtra n o dal sites; n o criteria f or pr o gressi ve disease are met

N o i ncrease c o nsiste nt wit h pr o gressi o n i n n o n meas ure d lesi o n a n d or ga n e nlar ge me nt 

N o ne w sites of disease s h o ul d be o bser ve d
Pr o gressi ve Dise ase:
Pr o gressi ve Meta b olic Disease ( P M D) f or P E T- C T- Base d Res p o nse 
T he desi g nati o n of P M D re q uires at least 1 of t he f oll o wi n g:

A 5 P S sc ore of  4 or 5 wit h a n i ncrease i n i nte nsit y of u pta ke fr o m baseli ne

Ne w F D G-a vi d f oci c o nsiste nt wit h l y m p h o ma at i nteri m or e n d of treat me nt assess me nt

Ne w F D G-a vi d f oci c o nsiste nt wit h l y m p h o ma rat her t ha n a n ot her eti ol o g y (e g, i nfecti o n or 
i nfla m mati o n). If u ncertai n re gar di n g eti ol o g y of ne w lesi o ns, bi o ps y or i nter val sca n ma y be 
c o nsi dere d.

Ne w or rec urre nt F D G-a vi d f oci i n b o ne marr o w 


A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 9 7
0 7 J u ne 2 0 2 2
Pr o gressi ve Ra di ol o gic Disease ( P R D) f or C T- Base d Res p o nse 
T he desi g nati o n of P R D re q uires at least 1 of t he f oll o wi n g:

A n i n di vi d ual n o de/lesi o n m ust be a b n or mal wit h all of t he f oll o wi n g:

L Di > 1. 5 c m 

I ncrease b y ≥ 5 0 % fr o m cr oss pr o d uct of L Di a n d per pe n dic ular dia meter na dir

A n i ncrease i n L Di or s h ortest tra ns verse dia meter ( S Di), s h ortest a xis per pe n dic ular t o 
t he L Di, S Di fr o m na dir 
■
0. 5 c m f or lesi o ns ≤ 2 c m 
■
1. 0 c m f or lesi o ns > 2 c m

I n t he setti n g of s ple n o me gal y, t he s ple nic le n gt h m ust i ncrease b y > 5 0 % of t he e xte nt of its 
pri or i ncrease be y o n d baseli ne (e g, a 1 5-c m s plee n m ust i ncrease t o > 1 6 c m). If t here is n o 
pri or s ple n o me gal y, t he s ple nic le n gt h m ust i ncrease b y at least 2 c m fr o m baseli ne 

Ne w or rec urre nt s ple n o me gal y

Ne w or clear pr o gressi o n of pre-e xisti n g n o n meas ure d lesi o ns

Ne w lesi o ns, defi ne d b y a n y of t he f oll o wi n g:

Re gr o wt h of pre vi o usl y res ol ve d lesi o ns

A ne w n o de > 1. 5 c m i n a n y a xis

A ne w e xtra n o dal site > 1. 0 c m i n a n y a xis; if < 1. 0 c m i n a n y a xis, its prese nce m ust be 
u ne q ui v ocal a n d m ust be attri b uta ble t o l y m p h o ma

Assessa ble disease of a n y size u ne q ui v ocall y attri b uta ble t o l y m p h o ma

Ne w or rec urre nt b o ne marr o w i n v ol ve me nt


A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 9 8
0 7 J u ne 2 0 2 2
A p pe n di x 3.   
C hil d be ari n g P ote nti al a n d Birt h C o ntr ol 
T his st u d y will f oll o w t he rec o m me n dati o ns fr o m t he Cli nical Trial Facilitati o n Gr o u p ( C T F G) 
{ Cli nical Trials Facilitati o n Gr o u p ( C T F G) 2 0 1 4 }, a s descri be d bel o w. 
A.
Defi niti o n of C hil d be ari n g P ote nti al
A fe male is c o nsi dere d of c hil d beari n g p ote ntial (ie, fertile) f oll o wi n g me narc he a n d u ntil 
bec o mi n g p ost me n o pa usal u nless per ma ne ntl y sterile. Per ma ne nt sterilizati o n met h o ds i ncl u de 
h ysterect o m y, bilateral sal pi n gect o m y, a n d bilateral o o p h orect o m y. 
A p ost me n o pa usal state is defi ne d as n o me nses f or 1 2 m o nt hs wit h o ut a n alter nati ve me dical 
ca use. A hi g h f ollicle sti m ulati n g h or m o ne ( F S H) le vel i n t he p ost me n o pa usal ra n ge ma y be use d 
t o c o nfir m a p ost me n o pa usal state i n w o me n n ot usi n g h or m o nal c o ntrace pti o n or h or m o nal 
re place me nt t hera p y. H o we ver, i n t he a bse nce of 1 2 m o nt hs of a me n orr hea, a si n gle F S H 
meas ure me nt is i ns ufficie nt.
F or t he p ur p ose of t his st u d y, a male is c o nsi dere d fertile after p u bert y u nless per ma ne ntl y sterile 
b y bilateral orc hi dect o m y.
B.
Birt h C o ntr ol Met h o ds T h at M a y Be C o nsi dere d as Hi g hl y Effecti ve
Met h o ds t hat ca n ac hie ve a fail ure rate of less t ha n 1 % per year w he n use d c o nsiste ntl y a n d 
c orrectl y are c o nsi dere d as hi g hl y effecti ve birt h c o ntr ol met h o ds. S uc h met h o ds i ncl u de: 

C o m bi ne d (estr o ge n- a n d pr o gester o ne-c o ntai ni n g) h or m o nal c o ntrace pti o n ass ociate d wit h 
i n hi biti o n of o v ulati o n1 via t he f oll o wi n g f or m of a d mi nistrati o n:

Oral

I ntra va gi nal

Tra ns der mal

Pr o gest o ge n- o nl y h or m o nal c o ntrace pti o n ass ociate d wit h i n hi biti o n of o v ulati o n 1:

Oral

I njecta ble

I m pla nta ble2
1  H or m o nal c o ntrace pti o n ma y be s usce pti ble t o i nteracti o n wit h t he i n vesti gati o nal pr o d uct, w hic h ma y re d uce t he 
efficac y of t he c o ntrace pti o n met h o d.
2  C o ntracepti o n met h o ds t hat i n t he c o nte xt of t his g ui da nce are c o nsi dere d t o ha ve l o w user de pe n de nc y.


A xica bta ge ne Cil ole ucel
Kite P har ma, I nc
Cli nical Pr ot oc ol
K T E- C 1 9- 1 1 1 ( Z U M A- 1 1)
A me n d me nt 5
C O N FI D E N TI A L
Pa ge 9 9
0 7 J u ne 2 0 2 2

I ntra uteri ne de vice)2

I ntra uteri ne h or m o ne-releasi n g s yste m2

Bilateral t u bal occl usi o n 2

Vasect o mize d part ner 2 ,3

Se x ual a bsti ne nce 4
C.
U n acce pt a ble Birt h C o ntr ol Met h o ds
Birt h c o ntr ol met h o ds t hat are u nacce pta ble i ncl u de peri o dic a bsti ne nce (cale n dar, 
s y m pt ot her mal, or p ost o v ulati o n met h o ds), wit h dra wal (c oit us i nterr u pt us), s per mici des o nl y, 
a n d lactati o nal a me n orr hea met h o d L A M. A fe male c o n d o m a n d a male c o n d o m s h o ul d n ot be 
use d t o get her.
3  Vasect o mize d part ner is a hi g hl y effecti ve birt h c o ntr ol met h o d pr o vi de d t hat part ner is t he s ole se x ual part ner of 
t he w o ma n of c hil d beari n g p ote ntial trial partici pa nt a n d t hat t he vasect o mize d part ner has recei ve d me dical 
assess me nt of t he s ur gical s uccess.
4  I n t he c o nte xt of t his g ui da nce se x ual a bsti ne nce is c o nsi dere d a hi g hl y effecti ve met h o d o nl y if defi ne d as 
refrai ni n g fr o m heter ose x ual i nterc o urse d uri n g t he e ntire peri o d of ris k ass ociate d wit h t he st u d y treat me nts. T he 
relia bilit y of se x ual a bsti ne nce nee ds t o be e val uate d i n relati o n t o t he d urati o n of t he cli nical trial a n d t he 
preferre d a n d us ual lifest yle of t he s u bject.

SAP_001.pdf: 
 
 
 
S T A TI S TI C A L  A N A L Y SI S  P L A N  
 
S p o ns o r:  
Kit e  P h ar m a, I n c.  
2 4 0 0  Br o a d w a y 
S a nt a  M o ni c a,  C A  9 0 4 0 4 
U nit e d  St at es  of  A m eri c a  
 
 
C o m p a n y  C o d e:  
K T E -C 1 9  
P F -0 5 0 8 2 5 6 6  
 
 
U S A N /I N N  : 
A xi c a bt a g e n e  cil ol e u c el  
Ut o mil u m a b  
 
 
P r ot o c ol  Titl e  
A  P h as e  1/ 2  M ulti c e nt er  St u d y  E v al u ati n g t h e  S af et y  a n d 
Effi c a c y  of  A xi c a bt a g e n e  Cil ol e u c el i n  C o m bi n ati o n  wit h 
Ut o mil u m a b  i n  S u bj e cts  wit h  R efr a ctor y  L ar g e  B- C ell 
L y m p h o m a   
 
 
P r ot o c ol  N u m b e r  
K T E -C 1 9 -1 1 1  
 
 
V e rsi o n  N u m b e r:  
2. 0  
 
 
R el e as e  D at e:  
2 9  M ar c h  2 0 2 1  
 
 
R e pl a c es  P r e vi o us  V e rsi o n(s):   
V 1. 0,   1 9 J ul y  2 0 1 9  
 
 
C O N FI D E N TI A LI T Y  N O TI C E  
T his  d o c u m e nt  c o nt ai ns  c o nfi d e nti al i nf or m ati o n  of  Kit e  P h ar m a I n c.,  a  w h oll y  o w n e d 
s u bsi di ar y  of  Gil e a d  S ci e n c es, I n c.   T his  d o c u m e nt  m ust  n ot  b e  dis cl os e d t o  a n y o n e  ot h er t h a n 
t h e sit e r es e ar c h st aff  a n d  m e m b ers  of t h e i nstit uti o n al r e vi e w  b o ar d/i n d e p e n d e nt  et hi cs 
c o m mitt e e,  a s ci e ntifi c r e vi e w  b o ar d,  or  a n  e q ui v al e nt.   T h e i nf or m ati o n i n t his  d o c u m e nt 
c a n n ot  b e  us e d f or  a n y  p ur p os e  ot h er t h a n t h e  c o n d u ct  of t h e  cli ni c al i n v esti g ati o n  wit h o ut t h e 
pri or  writt e n  c o ns e nt  of  Kit e  P h ar m a, I n c.   Q u esti o ns r e g ar di n g  h o w t his  d o c u m e nt s h o ul d  b e 
us e d  or t h e  c o n d u ct  of t h e  cli ni c al tri al s h o ul d  b e  dir e ct e d t o t h e  k e y s p o ns or  c o nt a cts.  


K T E- C 1 9 -1 1 1  
Kit e  P h ar m a, I n c.  
St atisti c al  A n al ysis  Pl a n  
 
C O N FI D E N TI A L  
P a g e  2 
2 9  M ar c h  2 0 2 1  
T A B L E  O F  C O N T E N T S  
S T A TI S TI C A L  A N A L Y SI S  P L A N  .............................................................................................................................. 1 
T A B L E  O F  C O N T E N T S  .............................................................................................................................................. 2 
LI S T  O F  T A B L E S  ......................................................................................................................................................... 3 
LI S T  O F  FI G U R E S  ....................................................................................................................................................... 3 
LI S T  O F  A B B R E VI A TI O N S  A N D  D E FI NI TI O N S  O F  T E R M S  ................................................................................ 4 
1.  
I N T R O D U C TI O N ................................................................................................................................................ 5 
2.  
O BJ E C TI V E S  ....................................................................................................................................................... 6 
3.  
S T U D Y  D E SI G N  .................................................................................................................................................. 7 
3. 1.  
O v er vi e w  .................................................................................................................................................. 7 
3. 2.  
H y p ot h esis  ................................................................................................................................................ 9 
3. 3.  
S a m pl e  Si z e  C o nsi d er ati o n  ....................................................................................................................... 9 
4.  
S T U D Y  E N D P OI N T S  A N D  C O V A RI A T E S  .................................................................................................... 1 1  
4. 1.  
E n d p oi nts  ............................................................................................................................................... 1 1  
4. 1. 1.  
Pri m ar y  E n d p oi nt  ................................................................................................................. 1 1  
4. 1. 2.  
S e c o n d ar y  E n d p oi nts  ............................................................................................................ 1 1  
4. 2.  
S u b gr o u ps  a n d  C o v ari at es  ...................................................................................................................... 1 1  
5.  
D E FI NI TI O N S  .................................................................................................................................................... 1 3  
5. 1.  
G e n er al  ................................................................................................................................................... 1 3  
5. 2.  
S af et y  ..................................................................................................................................................... 1 4  
5. 3.  
Effi c a c y  .................................................................................................................................................. 1 6  
6.  
A N A L Y SI S  S E T S ............................................................................................................................................... 1 8  
6. 1.  
F ull  A n al ysis  S et  .................................................................................................................................... 1 8  
6. 2.  
M o difi e d I nt e nt -t o-tr e at  A n al ysis  S et ..................................................................................................... 1 8  
6. 3.  
S af et y  A n al ysis  S et  ................................................................................................................................ 1 8  
6. 4.  
D L T  A n al ysis  S et  ................................................................................................................................... 1 8  
6. 5.  
S af et y r etr e at m e nt  a n al ysis s et  ............................................................................................................... 1 8  
6. 6.  
mI T T  R etr e at m e nt  A n al ysis  S et  ............................................................................................................. 1 8  
7.  
I N T E RI M  A N A L Y SI S  A N D  E A R L Y  S T O P PI N G  G UI D E LI N E S ................................................................... 1 9  
7. 1.  
S af et y I nt eri m  A n al ysis  .......................................................................................................................... 1 9  
8.  
D A T A  S C R E E NI N G  A N D  A C C E P T A N C E ...................................................................................................... 2 0  
8. 1.  
G e n er al  Pri n ci pl es  .................................................................................................................................. 2 0  
8. 2.  
El e ctr o ni c  Tr a nsf er  a n d  Ar c hi vi n g  of  D at a  ............................................................................................ 2 0  
8. 3.  
H a n dli n g  of  Missi n g  a n d I n c o m pl et e  D at a  ............................................................................................. 2 0  
8. 3. 1.  
Effi c a c y  ................................................................................................................................ 2 0  
8. 3. 2.  
S af et y .................................................................................................................................... 2 0  
8. 4.  
D et e cti o n  of  Bi as  .................................................................................................................................... 2 0  
8. 5.  
O utli ers  ................................................................................................................................................... 2 0  
8. 6.  
Distri b uti o n al  C h ar a ct eristi cs  ................................................................................................................. 2 1  
8. 7.  
V ali d ati o n  a n d  C o nfi g ur ati o n  M a n a g e m e nt  ........................................................................................... 2 1  
9.  
S T A TI S TI C A L  M E T H O D S  O F  A N A L Y SI S  .................................................................................................... 2 2  
9. 1.  
G e n er al  Pri n ci pl es  .................................................................................................................................. 2 2  
9. 2.  
S u bj e ct  A c c o u nt a bilit y  ........................................................................................................................... 2 2  


K T E- C 1 9 -1 1 1  
Kit e  P h ar m a, I n c.  
St atisti c al  A n al ysis  Pl a n  
 
C O N FI D E N TI A L  
P a g e  3 
2 9  M ar c h  2 0 2 1  
9. 3.  
I m p ort a nt  Pr ot o c ol  D e vi ati o ns ............................................................................................................... 2 2  
9. 4.  
D e m o gr a p hi c  a n d  B as eli n e  C h ar a ct eristi cs  ............................................................................................ 2 3  
9. 5.  
M e di c al  a n d  S ur gi c al  Hist or y  ................................................................................................................. 2 4  
9. 6.  
Pri or  T h er a p y f or  Pri m ar y  St u d y  Dis e as e  a n d  Pri or  R a di ot h er a p y  ........................................................ 2 4  
9. 7.  
Effi c a c y  A n al ys es  ................................................................................................................................... 2 4  
9. 7. 1.  
C o m pl et e  R es p o ns e  a n d  O bj e cti v e  R es p o ns e  ....................................................................... 2 4  
9. 7. 2.  
D ur ati o n  of  R es p o ns e  ........................................................................................................... 2 5  
9. 7. 3.  
Pr o gr essi o n  Fr e e  S ur vi v al  .................................................................................................... 2 5  
9. 7. 4.  
O v er all  S ur vi v al  ................................................................................................................... 2 5  
9. 7. 5.  
T u m or  B ur d e n  ...................................................................................................................... 2 5  
9. 8.  
S af et y  A n al ys es  ...................................................................................................................................... 2 5  
9. 8. 1.  
A d v ers e  E v e nts  ..................................................................................................................... 2 6  
9. 8. 2.  
L a b or at or y  T est  R es ults ........................................................................................................ 2 7  
9. 8. 3.  
A nti -dr u g  A nti b o d y  .............................................................................................................. 2 7  
9. 8. 4.  
R e pli c ati o n  C o m p et e nt  R etr o vir us  ....................................................................................... 2 7  
9. 9.  
E x p os ur e t o  St u d y  Tr e at m e nts  a n d  Pr o d u ct  C h ar a ct eristi cs  ................................................................... 2 7  
9. 1 0.  
E x p os ur e t o  C o n c o mit a nt  M e di c ati o ns  a n d  Pr o c e d ur es  ......................................................................... 2 7  
9. 1 1.  
S u bs e q u e nt  A nti -C a n c er  T h er a p y  .......................................................................................................... 2 8  
9. 1 2.  
D ur ati o n  M etri cs  .................................................................................................................................... 2 8  
9. 1 3.  
A xi c a bt a g e n e  Cil ol e u c el  D eli v er y  Ti m e  ................................................................................................ 2 8  
9. 1 4.  
P h ar m a c o ki n eti cs  ................................................................................................................................... 2 8  
9. 1 5.  
P h ar m a c o d y n a mi cs  ................................................................................................................................ 2 8  
1 0.  C H A N G E S  F R O M  P R O T O C O L  S P E CI FI E D  A N A L Y S E S  ............................................................................. 2 9  
1 1.  R E F E R E N C E S  ................................................................................................................................................... 3 0  
1 2.  A P P E N DI C E S  .................................................................................................................................................... 3 1  
 
A p p e n di x  1.  
C o n v e nti o ns f or  Cli ni c al  D at a  T h at  R e q uir e I m p ut ati o n f or  P arti al  or  Missi n g  D at es  ........... 3 2  
A p p e n di x  2.  
D eri v ati o n  of  Ti m e t o  E v e nt  E n d p oi nts  .................................................................................. 3 4  
A p p e n di x  3.  
D eri v ati o n  of  L ast  d at e  k n o w n t o  b e  ali v e  .............................................................................. 3 7  
 
LI S T  O F  T A B L E S  
T a bl e  1.  
E x a ct  C o nfi d e n c e I nt er v als ( 9 5 %  a n d  8 0 %)  C orr es p o n di n g t o  O bs er v e d  C R  R at e 
b as e d  o n  2 7  S u bj e cts  .............................................................................................................. 1 0  
T a bl e  2.  
I m p ut ati o n  R ul es f or  P arti al  or  Missi n g  St art  D at es ............................................................... 3 2  
T a bl e  3.  
Pri m ar y  A n al ysis  of  D O R  ...................................................................................................... 3 4  
T a bl e  4.  
S e nsiti vit y  A n al ysis  of  D O R  .................................................................................................. 3 5  
T a bl e  5.  
Pri m ar y  A n al ysis  of  P F S  ........................................................................................................ 3 5  
T a bl e  6.  
S e nsiti vit y  A n al ysis  of  P F S  .................................................................................................... 3 6  
T a bl e  7.  
I m p ut ati o n  R ul e of  O S  E v e nt/ C e ns ori n g  D at e  ....................................................................... 3 6  
 
LI S T  O F  FI G U R E S  
Fi g ur e  1.  
St u d y  S c h e m a  of  3 + 3  D esi g n  ................................................................................................... 8 
Fi g ur e  2.  
St u d y  S c h e m a i n  P h as e  1  a n d  P h as e  2  ...................................................................................... 9 
 


K T E- C 1 9 -1 1 1  
Kit e  P h ar m a, I n c.  
St atisti c al  A n al ysis  Pl a n  
 
C O N FI D E N TI A L  
P a g e  4 
2 9  M ar c h  2 0 2 1  
LI S T  O F  A B B R E VI A TI O N S  A N D  D E FI NI TI O N S  O F  T E R M S  
A D A  
A nti -Dr u g  A nti b o d y  
A D a M  
A n al ysis  d at a  m o d el  
A E  
A d v ers e  e v e nt  
A S C T  
A ut ol o g o us st e m  c ell tr a ns pl a nt  
B S A  
B o d y s urf a c e  ar e a  
C A R  
C hi m eri c  a nti g e n r e c e pt or  
C R  
C o m pl et e r es p o ns e   
C R F  
C as e r e p ort f or m  
C R S  
C yt o ki n e r el e as e s y n dr o m e  
C T C A E  
C o m m o n  t er mi n ol o g y  crit eri a f or  a d v ers e  e v e nt 
D L T 
D os e- li miti n g t o xi cit y 
D O R  
D ur ati o n  of r es p o ns e   
E C O G  
E ast er n  C o o p er ati v e  O n c ol o g y  Gr o u p  
H L G T  
Hi g h -l e v el  gr o u p t er m 
I P D 
I m p ort a nt  pr ot o c ol  d e vi ati o ns 
M e d D R A  
M e di c al  Di cti o n ar y f or  R e g ul at or y  A cti viti es  
mI T T 
M o difi e d i nt e nt -t o-tr e at  
N CI  
N ati o n al  C a n c er I nstit ut e  
N H L  
N o n -H o d g ki n l y m p h o m a  
O R R  
O bj e cti v e r es p o ns e r at e   
P D  
Pr o gr essi v e  dis e as e   
P F S  
Pr o gr essi o n- fr e e s ur vi v al  
P R  
P arti al r es p o ns e  
P T  
Pr ef er t er m  
R C R  
R e pli c ati o n -c o m p et e nt r etr o vir us  
O S  
O v er all s ur vi v al   
S A E  
S eri o us  a d v ers e  e v e nt  
S A P  
St atisti c al  a n al ysis  pl a n  
S C T  
St e m  c ell tr a ns pl a nt  
S D  
St a bl e  dis e as e  
S D T M  
St u d y  d at a t a b ul ati o n  m o d el   
S M Q  
St a n d ar di z e d  M e d D R A  q u er y   
S O C  
S yst e m  or g a n  cl ass  
S O P  
St a n d ar d  o p er ati n g  pr o c e d ur e  
S P D  
S u m  of t h e  pr o d u ct  of t h e  di a m et ers   
S R T  
S af et y r e vi e w t e a m  
T E A E 
Tr e at m e nt -e m er g e nt  a d v ers e  e v e nt  
W H O  
W orl d  H e alt h  Or g a ni z ati o n  
 


K T E- C 1 9 -1 1 1  
Kit e  P h ar m a, I n c.  
St atisti c al  A n al ysis  Pl a n  
 
C O N FI D E N TI A L  
P a g e  5 
2 9  M ar c h  2 0 2 1  
1.   
I N T R O D U C TI O N  
T his st atisti c al  a n al ysis  pl a n ( S A P) s ets f ort h  pr os p e cti v el y t h e  d et ails  of st atisti c al  a n al ys es t h at 
ar e  o utli n e d i n  pr ot o c ol  K T E -C 1 9 - 1 1 1 e ntitl e d  “ A  P h as e  1/ 2  M ulti c e nt er  St u d y  E v al u ati n g t h e 
S af et y  a n d  Effi c a c y  of  A xi c a bt a g e n e  Cil ol e u c el i n  C o m bi n ati o n  wit h  Ut o mil u m a b  i n  S u bj e cts 
wit h  R efr a ct or y  L ar g e  B -C ell  L y m p h o m a ( Z U M A - 1 1) ”. 


K T E- C 1 9 -1 1 1  
Kit e  P h ar m a, I n c.  
St atisti c al  A n al ysis  Pl a n  
 
C O N FI D E N TI A L  
P a g e  6 
2 9  M ar c h  2 0 2 1  
2.   
O B J E C TI V E S  
T h e  pri m ar y  o bj e cti v e  of  P h as e  1  is t o e v al u at e t h e s af et y  of  a xi c a bt a g e n e  cil ol e u c el i n 
c o m bi n ati o n  wit h  ut o mil u m a b  a n d t o i d e ntif y t h e  m ost  a p pr o pri at e  d os e  a n d ti mi n g  of 
ut o mil u m a b t o  c arr y f or w ar d i nt o  P h as e  2 .   
T h e  pri m ar y  o bj e cti v e  of  P h as e  2 is t o  e v al u at e t h e  effi c a c y  of  a xi c a bt a g e n e  cil ol e u c el  a n d 
ut o mil u m a b,  as  m e as ur e d  b y  c o m pl et e r es p o ns e  ( C R) r at e, i n s u bj e cts  wit h r efr a ct or y l ar g e 
B- c ell l y m p h o m a.  
T h e s e c o n d ar y  o bj e cti v es  of  P h as e  1  a n d  P h as e  2  ar e t o  ass ess t h e s af et y  a n d t ol er a bilit y  of 
a xi c a bt a g e n e  cil ol e u c el  a n d  ut o mil u m a b,  a d diti o n al  effi c a c y  e n d p oi nts,  a n d l e v els  of 
a xi c a bt a g e n e  cil ol e u c el i n  bl o o d ( p h ar m a c o ki n eti cs [ P K])  a n d l e v els  of  c yt o ki n es i n s er u m 
( p h ar m a c o d y n a mi cs [ P D]). 






K T E- C 1 9 -1 1 1  
Kit e  P h ar m a, I n c.  
St atisti c al  A n al ysis  Pl a n  
 
C O N FI D E N TI A L  
P a g e  9 
2 9  M ar c h  2 0 2 1  
Fi g u r e  2. 
St u d y  S c h e m a  i n  P h as e  1 a n d  P h as e  2  
 
 
3. 2.   
H y p ot h esis  
N o f or m al  h y p ot h esis  will  b e t est e d i n t his st u d y.  T h e  P h as e  2  p orti o n  of t h e st u d y is  d esi g n e d t o 
esti m at e t h e tr u e  C R  r at e i n s u bj e cts  wit h r efr a ct or y l ar g e  B-c ell l y m p h o m a tr e at e d  wit h 
a xi c a bt a g e n e  cil ol e u c el f oll o w e d  b y  ut o mil u m a b.  
3. 3.   
S a m pl e  Si z e  C o nsi d e r ati o n  
T h e  a nti ci p at e d  e nr oll m e nt i n t his st u d y is  a p pr o xi m at el y  3 t o  6 0 s u bj e cts.  P h as e  1  will  e nr oll 
a p pr o xi m at el y  3 t o  3 6 s u bj e cts. If t h e st u d y  pr o c e e ds t o  P h as e  2,  a t ot al  of  u p t o  2 4  a d diti o n al 
s u bj e cts  will  b e  e nr oll e d.  
T his st u d y  us es  a si n gl e -ar m  d esi g n t o  esti m at e t h e tr u e  C R r at e i n s u bj e cts  wit h r efr a ct or y l ar g e 
B- c ell l y m p h o m a tr e at e d  wit h  a xi c a bt a g e n e  cil ol e u c el  a n d  ut o mil u m a b  at t h e  d osi n g s c h e d ul e 
t est e d i n  P h as e  1  a n d  d e e m e d s af e  b y t h e  S R T.  Wit h  a t ot al s a m pl e si z e  of   a p pr o xi m at el y  2 7 
s u bj e cts  at  a  gi v e n  d osi n g s c h e d ul e,  of  w hi c h  at l e ast  3  will  h a v e  b e e n tr e at e d i n t h e  P h as e  1 
p orti o n,  a n  o bs er v e d  C R r at e  of  7 0 %  will  yi el d  a n  e x a ct  9 5 %  c o nfi d e n c e i nt er v al ( CI)  of ( 5 0 %, 
8 6 %).  T his t ar g et  C R r at e,  a n d t h e l o w er li mit  of t h e  7 0 %  CI f or t h e  C R r at e, is  m e a ni n gf ul 
b e c a us e it  w o ul d r e pr es e nt  a si g nifi c a nt i m pr o v e m e nt i n t h e r es p o ns e r at e f or t h e s u bj e cts  wit h 
r el a ps e d/r efr a ct or y l ar g e  B-c ell l y m p h o m a  o v er  e xisti n g t h er a pi es. .  A d diti o n al  ass u m pti o ns  a n d 
c orr es p o n di n g t w o-si d e d  9 5 %  a n d  8 0 %  e x a ct  c o nfi d e n c e i nt er v als  ar e  pr o vi d e d i n  T a bl e  1 .    
 
  
 
  
 
 
 
 
 
 
  
  
 
  
  
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
     
    
 
 
 
 
 
 
 
 
 
   
 
  
 
 
 
 
  
         
 
 
 
  
  
 
 
 
 
 
 
 
 
 
 
 
 
 
  
  
   
 
 
 
 
 
 
 
 
   
  
 
 
 
  
 
  
 
  
 
 
 
 
 
 
 
 
  
 
 
 
 
 
 
   
  
 
 
 
 
 
 
 
 
 
 
  
  
   
     
   
 
 
 
 
 
 
 
  
  
  
  
  
  
 
 
 
  
 
 
 
   
 
   
 
 
 
 
 
 
 
 
 
  
 
    
 
  
     
  
 
 
 
  
  
 
 
  
 
 
 
 
 
   
 
  
 
 
           
  
 
    
 
     
   
 
     
 
 
  
 
  
 
 
  
 
 
 
 
  
 
 
 
 
 
  
  
 
 
 
     
 
 
 
 
 
 
   
 
 
 
  
  
 
 
 
 
 
 
 
 
 
 
    
  
  
  
 
 
 
 
  
  
  
 
  
    
 
 
  
  
 
 
  
 
 
 
 
 
 
 
 
  
  
 
 
 


K T E- C 1 9 -1 1 1  
Kit e  P h ar m a, I n c.  
St atisti c al  A n al ysis  Pl a n  
 
C O N FI D E N TI A L  
P a g e  1 0  
2 9  M ar c h  2 0 2 1  
T a bl e  1. 
E x a ct  C o nfi d e n c e I nt e r v als  ( 9 5 %  a n d  8 0 %) C o r r es p o n di n g t o 
O bs e r v e d  C R  R at e  b as e d  o n  2 7  S u bj e cts  
S u bj e cts  wit h  C R  
O bs e r v e d  C R  R at e  
9 5 %  C o nfi d e n c e 
I nt e r v al 
8 0 %  C o nfi d e n c e 
I nt e r v al 
1 5    
5 6 %  
[ 3 5 %,  7 5 %] 
[ 4 2 %,  6 9 %] 
1 7    
6 3 %  
[ 4 2 %,  8 1 %] 
[ 4 9 %,  7 6 %] 
1 9    
7 0 %  
[ 5 0 %,  8 6 %] 
[ 5 6 %,  8 2 %] 
2 1    
7 8 %  
[ 5 8 %,  9 1 %] 
[ 6 4 %,  8 8 %] 
2 3    
8 5 %  
[ 6 6 %,  9 6 %] 
[ 7 3 %,  9 3 %] 
A b br e vi ati o ns:  C R,  c o m pl et e r es p o ns e  


K T E- C 1 9 -1 1 1  
Kit e  P h ar m a, I n c.  
St atisti c al  A n al ysis  Pl a n  
 
C O N FI D E N TI A L  
P a g e  1 1  
2 9  M ar c h  2 0 2 1  
4.   
S T U D Y  E N D P OI N T S  A N D  C O V A RI A T E S  
4. 1.   
E n d p oi nts  
4. 1. 1.   
P ri m a r y  E n d p oi nt  
•   P h as e  1: I n ci d e n c e  of  A Es  d efi n e d  as  D L Ts  
•   P h as e  2:  C o m pl et e r es p o ns e ( C R) r at e  p er t h e  L u g a n o  Cl assifi c ati o n ( C h es o n  et  al,  2 0 1 4),  as 
d et er mi n e d  b y t h e st u d y i n v esti g at ors 
4. 1. 2.   
S e c o n d a r y  E n d p oi nts  
S e c o n d ar y  e n d p oi nts  ar e  a p pli c a bl e t o  b ot h  P h as e  1  a n d  P h as e  2: 
•   O bj e cti v e  R es p o ns e  R at e ( C R  +  p arti al r es p o ns e [ P R] )  p er t h e L u g a n o  Cl assifi c ati o n 
{ C h es o n  2 0 1 4 },  as  d et er mi n e d  b y st u d y i n v esti g at ors 
•   D ur ati o n  of  R es p o ns e  ( D O R) 
•   Pr o gr essi o n  Fr e e  S ur vi v al  ( P F S) p er t h e  L u g a n o  Cl assifi c ati o n  { C h es o n  2 0 1 4 } ,  as 
d et er mi n e d  b y st u d y i n v esti g at ors  
•   O v er all  S ur vi v al  ( O S) 
•   I n ci d e n c e  of  A Es  a n d  cli ni c all y si g nifi c a nt  c h a n g es i n s af et y l a b  v al u es 
•   P h ar m a c o ki n eti cs ( P K):  L e v els  of  a xi c a bt a g e n e  cil ol e u c el i n  bl o o d  
•   P h ar m a c o d y n a mi cs ( P D):  L e v els  of  c yt o ki n es i n s er u m  
4. 2.   
S u b g r o u ps  a n d  C o v a ri at es 
T h e f oll o wi n g  v ari a bl es  m a y  b e  us e d t o  e x a mi n e  effi c a c y r es ults i n s u b gr o u ps  or  c o v ari at e 
a n al ys es,  as  w ell  as s af et y  a n al ys es.  A n al ys es  mi g ht  b e s u bj e ct t o r estri cti o ns  d u e t o s m all 
s a m pl e si z e. 
•   E ast er n  C o o p er ati v e  O n c ol o g y  G r o u p ( E C O G) p erf or m a n c e st at us  ( 0,  1)  
•   A g e  at  b as eli n e  ( < 6 5  y e ars,  ≥ 6 5  y e ars)  
•   R a c e  
•   G e n d er 
•   Hist ol o gi c all y  pr o v e n  D L B C L t y p e   


K T E- C 1 9 -1 1 1  
Kit e  P h ar m a, I n c.  
St atisti c al  A n al ysis  Pl a n  
 
C O N FI D E N TI A L  
P a g e  1 2  
2 9  M ar c h  2 0 2 1  
•   C ell  of  ori gi n  (G C B ,  AB C,   or Ot h er )  
•   D o u bl e/ Tri pl e  hit st at us  
•   Dis e as e  st a g e  at st u d y  e ntr y   
•   I nt er n ati o n al  Pr o g n osti c I n d e x (I PI) t ot al s c or e  at  b as eli n e   
•   R efr a ct or y s u b gr o u p  ( pri m ar y r efr a ct or y, r efr a ct or y t o s e c o n d li n e  or  gr e at er, r el a ps e  p ost 
A S C T)   
•   N u m b er  of  pri or  c h e m ot h er a p y r e gi m e ns   
•   Pri or  tr a ns pl a nt ( a ut ol o g o us st e m  c ell tr a ns pl a nt [ A S C T]) ( Y/ N) 
•   B ul k y  dis e as e  ( at l e ast  o n e l esi o n  1 0  c m i n  di a m et er))   
•   T u m or  b ur d e n  at  b as eli n e,  as  m e as ur e d  b y t h e s u m  of t h e  pr o d u ct  of t h e  di a m et ers ( S P D)  of 
t ar g et l esi o ns  at  b as eli n e ( ≤  m e di a n  vs.  >  m e di a n  v al u e) 
•   R e c ei v e d  bri d gi n g  c h e m ot h er a p y 


K T E- C 1 9 -1 1 1  
Kit e  P h ar m a, I n c.  
St atisti c al  A n al ysis  Pl a n  
 
C O N FI D E N TI A L  
P a g e  1 3  
2 9  M ar c h  2 0 2 1  
5.   
D E FI NI TI O N S  
5. 1.   
G e n e r al  
St u d y  e n r oll m e nt : St u d y  e nr oll m e nt  o c c urs  w h e n  a s u bj e ct is  c o nfir m e d t o  b e  eli gi bl e f or t h e 
st u d y  a n d  c o m m e n c es l e u k a p h er esis.  
St u d y  D a y  0 :  D efi n ed  as t h e  d a y t h e s u bj e ct r e c ei v e s t h e  a xi c a bt a g e n e  cil ol e u c el  i nf usi o n. T h e 
d a y  pri or t o  D a y  0  will  b e st u d y  d a y - 1, t h e  d a y  aft er   D a y  0  will  b e st u d y  d a y  1.  A n y  d a ys  pri or 
D a y   0  will  b e s e q u e nti al  a n d  n e g ati v e i nt e g er-v al u e d ,  a n y  d a ys aft er   D a y  0  will  b e s e q u e nti al  a n d 
p ositi v e  i nt e g er- v al u e d.  
B as eli n e : D efi n e d  as t h e l ast  n o n- missi n g  v al u e  m e as ur e d  o n  or  pri or t o  c o n diti o ni n g 
c h e m ot h er a p y,  u nl ess s p e cifi e d  ot h er wis e.   
B as eli n e  of  r et r e at m e nt : If t h e s u bj e ct is  eli gi bl e f or r etr e at m e nt  wit h  a xi c a bt a g e n e  cil ol e u c el, 
t h e l ast r e c or ds  o n  or  pri or t o  c o n diti o ni n g  c h e m ot h er a p y r etr e at m e nt  will  b e  c o nsi d er e d t h e 
b as eli n e  of r etr e at m e nt,  u nl ess  ot h er wis e s p e cifi e d.  
R ef r a ct o r y  S u b g r o u p  at  B as eli n e :  
C h e m ot h er a p y- r efr a ct or y  dis e as e is  d efi n e d  as  o n e  or  m or e  of t h e f oll o wi n g: 
•   Pri m ar y r efr a ct or y  dis e as e  
   P D  as  b est r es p o ns e t o first -li n e t h er a p y,  or  
   S D  as  b est r es p o ns e  aft er  at l e ast  4  c y cl es  of first -li n e t h er a p y ( e. g.,  4  c y cl es  of  R-C H O P) 
wit h  S D  d ur ati o n  n o l o n g er t h a n  6  m o nt hs fr o m l ast  d os e  of  t h e t h er a p y  
•   R efr a ct or y  t o s e c o n d  or gr e at er li n e  of t h er a p y  
   P D  as  b est r es p o ns e t o  t h e m ost r e c e nt   t her a p y ,  or 
   S D  as  b est r es p o ns e  aft er  at l e ast  2  c y cl es  of l ast li n e  of t h er a p y  wit h  S D  d ur ati o n  n o 
l o n g er t h a n  6  m o nt hs fr o m l ast  d os e  of t h e t h er a p y  
•   R efr a ct or y  p ost -A S C T  
   Dis e as e  pr o gr essi o n  or r el a ps e d  ≤  1 2  m o nt hs  aft er  A S C T ( m ust  h a v e  bi o ps y  pr o v e n 
r e c urr e n c e i n r el a ps e d s u bj e cts) 
   If s al v a g e t h er a p y is  gi v e n  p ost- A S C T, t h e s u bj e ct  m ust  h a v e  h a d  n o r es p o ns e t o  or 
r el a ps e d  aft er t h e l ast li n e  of t h er a p y 


K T E- C 1 9 -1 1 1  
Kit e  P h ar m a, I n c.  
St atisti c al  A n al ysis  Pl a n  
 
C O N FI D E N TI A L  
P a g e  1 4  
2 9  M ar c h  2 0 2 1  
I n  c as e  a s u bj e ct  m a y  m e et t h e  crit eri a f or  m ulti pl e r efr a ct or y s u b gr o u ps, t his s u bj e ct  will  b e 
assi g n e d  t o t h e s u b gr o u p  b as e d  o n t h e  hi er ar c h y  of  pri orit y  of R efr a ct or y  p ost  A S C T  > 
R efr a ct or y t o  s e c o n d  or gr e at er li n e  of t h er a p y  >  Pri m ar y r efr a ct or y  dis e as e .   
5. 2.   
S af et y  
T r e at m e nt -e m e r g e nt  a d v e rs e  e v e nt ( T E A E) :   A n y  w ors e ni n g  of  a  pr e-e xisti n g  m e di c al 
c o n diti o n t h at  o c c urs  o n  or  aft er  a xi c a bt a g e n e  cil ol e u c el i nf usi o n   or  a n y  a d v ers e  e v e nt  wit h  o ns et 
o n  or  aft er  a xi c a bt a g e n e  cil ol e u c el i nf usi o n .  
D e at hs :  A n y  d e at h  o c c urri n g  aft er t h e l e u k a p h er esis  u p t hr o u g h t h e  e n d  of st u d y.   
A d v e rs e  e v e nts  of i nt e r est :  T h e f oll o wi n g  A Es  ar e of i nt er est  f or t h e tr e at m e nts  of a xi c a bt a g e n e 
cil ol e u c el i n  c o m bi n ati o n  wit h  ut o mil u m a b:   
I d e ntifi e d ris ks:  
•   C yt o ki n e- r el e as e s y n dr o m e ( C R S)  
•   N e ur ol o gi c  E v e nt 
•   C yt o p e ni as  
•   I nf e cti o ns 
•   H y p o g a m m a gl o b uli n e mi a 
P ot e nti al ris ks:   
•   S e c o n d ar y  m ali g n a n c y 
•   R e pli c ati o n  c o m p et e nt r etr o vir us ( R C R)  
•   I m m u n o g e ni cit y ( a nti-a xi c a bt a g e n e  cil ol e u c el  a n d  a nti- ut o mil u m a b  a nti b o di es)  
•   A ut oi m m u n e  dis or d ers  
•   B o n e  m arr o w f ail ur e  
C R S :  C R S is i d e ntifi e d  vi a  c oll e cti o n  of t h e s y n dr o m e  o n  a  c as e r e p ort fr o m ( C R F) s p e cifi c all y 
d esi g n e d t o  c oll e ct  C R S.  S p e cifi c i n di vi d u al s y m pt o ms  of  C R S ( e g, f e v er)  c oll e ct e d  o n t h e  A E 
l o g  ar e  c o d e d  usi n g  M e d D R A  a n d li n k e d t o t h e  c orr es p o n di n g  C R S  e pis o d e. I n di vi d u al 
s y m pt o ms  of  C R S  ar e  gr a d e d  p er  t h e l at est  v ersi o n  of  C o m m o n  T er mi n ol o g y  Crit eri a f or 
A d v ers e  E v e nts ( C T C A E ), a n d  C R S  as  a s y n dr o m e is  gr a d e d  p er  m o difi e d  L e e  crit eri a  { L e e 
2 0 1 4 }. I n t h e  m o difi e d  gr a di n g s c al e,  n e ur ol o gi c  A Es  ar e  n ot r e p ort e d  as  p art  of t h e  C R S 
s y n dr o m e  as t h e y  will  b e r e p ort e d s e p ar at el y  wit h t h e  n e ur ol o gi c  e v e nts  c at e g or y  a n d  gr a d e d  p er 
t h e l at est  v ersi o n  of C T C A E.  


K T E- C 1 9 -1 1 1  
Kit e  P h ar m a, I n c.  
St atisti c al  A n al ysis  Pl a n  
 
C O N FI D E N TI A L  
P a g e  1 5  
2 9  M ar c h  2 0 2 1  
N e u r ol o gi c  E v e nt :  N e ur ol o gi c A E s  ar e i d e ntifi e d  wit h  a s e ar c h str at e g y  b as e d  o n  k n o w n 
n e ur ol o gi c t o xi citi es  ass o ci at e d  wit h  a nti - C D 1 9 i m m u n ot h er a p y  {T o p p  2 0 1 5 } .   T h e s e ar c h 
str at e g y f o c us es  o n  c e ntr al  n er v o us s yst e m t o xi cit y,  wit h o ut r e g ar d t o t e m p or al r el ati o ns hi p  or 
c o n c o mit a nt  c o n diti o ns ( e. g.  C R S).    A d diti o n all y, t h e  M e d D R A s yst e m  or g a n  cl ass es ( S O Cs)  of 
Ps y c hi atri c  Dis or d ers  a n d  N er v o us  S yst e m  Dis or d ers  will  b e r e vi e w e d f or  a d diti o n al  e v e nts; 
t h es e  e v e nts  will t h e n  b e  e v al u at e d f or  p ot e nti al i n cl usi o n  as  n e ur ol o gi c  A Es. 
C yt o p e ni as :   C yt o p e ni as ( n e utr o p e ni a,  a n e mi a,  a n d t hr o m b o c yt o p e ni a)  ar e i d e ntifi e d  as:  
•   N e utr o p e ni a is i d e ntifi e d  usi n g  t h e l at est  v ersi o n  of M e d D R A s e ar c h t er ms ( M S T) 
d o c u m e nt e d  pri or t o  e a c h  a n al ysis. 
•   A n e mi a  (i n cl u di n g  a pl asti c  a n e mi a) is i d e ntifi e d  usi n g t h e st a n d ar di z e d  M e d D R A  q u er y 
( S M Q)  h a e m at o p oi eti c  er yt hr o p e ni a ( br o a d s e ar c h). 
•   T hr o m b o c yt o p e ni a is i d e ntifi e d  usi n g t h e  S M Q  h a e m at o p oi eti c t hr o m b o c yt o p e ni a ( n arr o w 
s e ar c h).  
I nf e cti o ns:  I nf e cti o ns  ar e i d e ntifi e d  as  A Es  wit hi n t h e  M e d D R A  S O C  of I nf e cti o ns  a n d 
I nf est ati o ns t h at  o c c ur aft er tr e at m e nt  wit h  a nti - C D 1 9  C A R  T  c ells.  S u bt y p es  of i nf e cti o ns  ar e 
i d e ntifi e d  usi n g  M e d D R A  hi g h l e v el  gr o u p t er ms ( H L G T) t h at  c a pt ur e  e v e nts  of:  
•   B a ct eri al i nf e cti o n,  e n c o m p assi n g  t h e  M e d D R A  H L G Ts  of  
   B a ct eri al  i nf e cti o us  dis or d ers  
   C hl a m y di al  i nf e ctio us  dis or d ers 
•   V ir al i nf e cti o n,  e n c o m p assi n g t h e  M e d D R A  H L G T  of  vir al i nf e cti o us  dis or d ers 
•   O p p ort u nisti c i nf e cti o ns,  e n c o m p assi n g t h e  M e d D R A  H L G Ts  of  
   F u n g al i nf e cti o us  dis or d ers 
   M y c o b a ct eri al  i nf e cti o us  dis or d ers 
•   Ot h er i nf e cti o ns,  e n c o m p assi n g t h e  M e d D R A  H L G T  of I nf e cti o ns  –  p at h o g e n  u ns p e cifi e d  
H y p o g a m m a gl o b uli n e mi a : H y p o g a m m a gl o b uli n e mi a  will  b e i d e ntifi e d  usi n g  a  M S T s e ar c h 
str at e g y  d efi n e d  b y  Kit e.   
S e c o n d a r y  m ali g n a n c y :  S e c o n d ar y  m ali g n a n ci es  ar e i d e ntifi e d  vi a  c oll e cti o n  o n  a C R F  i n  w hi c h 
t h e i n v esti g at or  cl assifi es t h e  e v e nt  as  a s e c o n d ar y  m ali g n a n c y.   A d diti o n all y, A E s t h at  ar e  c o d ed 
i nt o t h e  S O C  of  N e o pl as ms  b e ni g n,  m ali g n a nt,  a n d  u ns p e cifi e d (i n cl u di n g  c ysts  a n d  p ol y ps)  wit h 
t h e  e x c e pti o n  of  pr ef err e d t er ms  c o nt ai ni n g  “ B-c ell ”  or  “ B  c ell ”  a n d  “ L y m p h o m a ”  will  b e 
r e vi e w e d t o i d entif y  ot h er  p ot e nti al  e v e nts.    


K T E- C 1 9 -1 1 1  
Kit e  P h ar m a, I n c.  
St atisti c al  A n al ysis  Pl a n  
 
C O N FI D E N TI A L  
P a g e  1 6  
2 9  M ar c h  2 0 2 1  
I m m u n o g e ni cit y ( a nti- a xic a bt a g e n e  cil ol e u c el  a n d  a nti- ut o mil u m a b  a nti b o di es) : 
I m m u n o g e ni cit y  will  b e i d e ntifi e d f or s u bj e cts  w h o  h a v e tr e at m e nt  e m er g e nt  a nti-a xi c a bt a g e n e 
cil ol e u c el  or  a nti- ut o mil u m a b  a nti b o d y , r es p e cti v el y,  a n d  h a v e  d e v el o p e d  a n y  A E  b el o n gi n g t o 
t h e  S M Q  of  a n a p h yl a cti c r e a cti o n  a n d t h e  S M Q  of  h y p ers e nsiti vit y.   T h e  n arr o w  v ersi o n  of t h es e 
2  S M Qs  will  b e  us e d.   
A ut oi m m u n e  dis o r d e rs :  A ut oi m m u n e  dis or d ers  ar e i d e ntifi e d  vi a  c oll e cti o n  o n  a C R F  i n  w hi c h 
t h e i n v esti g at or  cl assifi es t h e  e v e nt  as  a n  a ut oi m m u n e  dis or d er.  A d diti o n all y,  a d v ers e  e v e nts t h at 
ar e  c o d e d i nt o t h e  M e d D R A  H L G T  of  a ut o -i m m u n e  dis or d ers  wit hi n t h e i m m u n e s yst e m 
dis or d ers  S O C  will  b e r e vi e w e d t o i d e ntif y  ot h er  p ot e nti al  e v e nts.  
B o n e  m a r r o w f ail u r e:   B o n e  m arr o w f ail ur e  will  b e i d e ntifi e d  usi n g t h e  n arr o w  S M Q  of 
h a e m at o p oi eti c  c yt o p e ni as  aff e cti n g  m or e t h a n  o n e t y p e  of  bl o o d t est. 
St u d y  d a y  of  o ns et  of  e v e nt/s y n d r o m e :  St u d y  d a y  of  o ns et  of  a n  e v e nt/s y n dr o m e is  d efi n e d  as 
t h e st u d y  d a y  of t h e first  o c c urr e n c e  of t h e  e v e nt/s y n dr o m e.  St u d y  d a y  of  ons et  o f  Gr a d e  3  or 
hi g h er  e v e nts/ s y n dr o m es  ar e  d efi n e d i n t h e s a m e  w a y,  b ut r estri ct e d t o  Gr a d e  3  or  hi g h er 
e v e nts/s y n dr o m es. 
St u d y  d a y  of  r es ol uti o n  of  a n  e v e nt/s y n d r o m e :  St u d y  d a y  of r es ol uti o n  of  a n  e v e nt/s y n dr o m e 
is t h e l ast st u d y  d a y t h e  e v e nt is  pr es e nt. If  m ulti pl e  e v e nts  o c c ur  aft er a xi c a bt a g e n e  cil ol e u c el 
i nf usi o n, t h e st u d y  d a y  of r es ol uti o n is t h e l ast  d a y  of t h e  m ulti pl e  e v e nts  pr es e nt e d.  St u d y  d a y  of 
r es ol uti o n  will  n ot  b e  c al c ul at e d f or  e v e nts t h at  ar e  o n g oi n g  at t h e ti m e  of t h e  d at a  c ut off  d at e  or 
d e at h.   
D u r ati o n  of  a n  A E  of i nt e r est :  T h e  d ur ati o n  of  a n  A E  of i nt er est  m a y  b e  d eri v e d  o nl y  a m o n g 
s u bj e cts f or  w h o m  all  e v e nts  of t h e  cl ass  h a v e r es ol v e d  b y t h e  a n al ysis  d at a  c ut off  d at e.  T h e 
d ur ati o n is  d efi n e d  as t h e  ti m e fr o m t h e  e arli est  o ns et  d at e  of t h e  A Es i n t h e  e v e nt  cl ass  of 
i nt er est t hr o u g h t h e r es ol uti o n  d at e  of t h e l ast  A Es i n t h e  e v e nt  cl ass, r e g ar dl ess  of t h e  g a ps  of t h e 
d a ys  b et w e e n  m ulti pl e  e v e nts,  i e, t h e r es ol uti o n  d at e  of t h e l ast  A E i n t h e  e v e nt  cl ass –  t h e st art 
d at e  of t h e first  A E i n t h e  e v e nt  cl ass  +  1 .   
T h e  c u m ul ati v e  d os e  of  ut o mil u m a b  is  d efi n e d  as t h e s u m  of  all  d os es (i n  m g)  a d mi nist er e d 
a cr oss t h e tr e at m e nt  p eri o d.  
5. 3.   
Effi c a c y  
C o m pl et e  R es p o ns e  R at e ( C R R) :   T h e  pr o p orti o n  of s u bj e cts  wit h  a  C R w hil e  aft er tr e at m e nt 
wit h  a xi c a bt a g e n e  cil ol e u c el  a n d  ut o mil u m a b   a n d  pri or t o  a n y s u bs e q u e nt a nti -c a n c er  t h er a p y. 
S u bj e cts  w h o  d o  n ot  m e et t h e  crit eri a f or  C R  b y t h e  a n al ysis  c ut off  d at e  will  b e  c o nsi d er e d  n o n -
C R.  T h e  d eri v ati o n  of t his  e n d p oi nt  will  o nl y i n cl u d e r es p o ns e  ass ess m e nts  o bt ai n e d  aft er  at l e ast 
o n e  d os e  of  ut o mil u m a b  a d mi nistr ati o n  aft er  a xi c a bt a g e n e  cil ol e u c el i nf usi o n,  a n d  pri or t o  a n y 
s u bs e q u e nt  a nti- c a n c er  t h er a pi es (in cl u di n g  st e m  c ell tr a ns pl a nt [ S C T ]).   R es p o ns es  will  b e 
ass ess e d  p er  t h e  L u g a n o  Cl assifi c ati o n { C h es o n  2 0 1 4 },  as  d et er mi n e d  b y t h e st u d y i n v esti g at ors. 
O bj e cti v e  R es p o ns e  R at e ( O R R) :   T h e  pr o p orti o n  of s u bj e cts  wit h  a C R  or  P R  w hil e  aft er 
tr e at m e nt  wit h  a xi c a bt a g e n e  cil ol e u c el  a n d ut o mil u m a b   a n d  pri or t o  a n y s u bs e q u e nt a nti -c a n c er 


K T E- C 1 9 -1 1 1  
Kit e  P h ar m a, I n c.  
St atisti c al  A n al ysis  Pl a n  
 
C O N FI D E N TI A L  
P a g e  1 7  
2 9  M ar c h  2 0 2 1  
t h er a p y.  Su bj e cts  w h o  d o  n ot  m e et t h e  crit eri a  b y t h e  a n al ysis  c ut off  d at e  will  b e  c o nsi d er e d 
n o n-r es p o n d ers.  T h e  d eri v ati o n  of t his  e n d p oi nt  will  o nl y i n cl u d e r es p o ns e  ass ess m e nts  o bt ai n e d 
aft er  at l e ast  o n e  d os e  of  ut o mil u m a b  a d mi nistr ati o n  aft er  a xi c a bt a g e n e  cil ol e u c el i nf usi o n,  a n d 
pri or t o  a n y s u bs e q u e nt   a nti-c a n c er  t h er a pi es (i n cl u di n g S C T) .  R es p o ns es  will  b e  ass ess e d  p er 
t h e  L u g a n o  Cl assifi c ati o n { C h es o n  2 0 1 4 },  as  d et er mi n e d  b y t h e st u d y i n v esti g at ors.  
D u r ati o n  of  r es p o ns e ( D O R) :  D O R is  d efi n e d  o nl y f or s u bj e cts  w h o  e x p eri e n c e  a n  o bj e cti v e 
r es p o ns e ( C R  or  P R)  a n d is t h e ti m e fr o m t h e first  o bj e cti v e r es p o ns e t o  dis e as e  pr o gr essi o n  p er 
t h e  L u g a n o  Cl assifi cati o n  { C h es o n  2 0 1 4 }  as  d et er mi n e d  b y st u d y i n v esti g at ors  or  d e at h  d u e t o 
a n y r e as o n.  S u bj e cts  n ot  m e eti n g t h e  crit eri a f or  pr o gr essi o n  or  d e at h  b y t h e  a n al ysis  d at a  c ut off 
d at e  will  b e  c e ns or e d  at t h eir l ast  e v al u a bl e  dis e as e  ass ess m e nt  d at e  a n d t h eir r es p o ns e  will  b e 
n ot e d  as  o n g oi n g.  S u bj e cts  w h o r e c ei v e  a d diti o n al  a nti- c a n c er t h er a p y i n t h e  a bs e n c e  of 
d o c u m e nt e d  pr o gr essi o n  will  b e  c e ns or e d  at t h e l ast  e v al u a bl e  dis e as e  ass ess m e nt  pri or t o t h e 
a d diti o n al t h er a p y.  S u bj e cts  w h o r e c ei v e  a n  S C T i n t h e  a bs e n c e  of  d o c u m e nt e d  pr o g r essi o n  will 
b e  c e ns or e d  at t h e l ast  e v al u a bl e  dis e as e  ass ess m e nt  pri or t o t h e  d at e  of t h e  S C T.  A s e nsiti vit y 
a n al ysis  will  b e  c o n d u ct e d i n  w hi c h  dis e as e  ass ess m e nts  o bt ai n e d  aft er  S C T  w hil e i n 
a xi c a bt a g e n e  cil ol e u c el i n d u c e d r e missi o n  ar e i n cl u d e d i n t h e  d eri v ati o n  of  D O R.  A d diti o n al 
d et ails  o n t h e  d eri v ati o n  of  D O R  ar e  pr o vi d e d i n  A p p e n di x  2 .  
P r o g r essi o n -f r e e  S u r vi v al ( P F S):  P F S is  d efi n e d  as t h e ti m e fr o m t h e a xi c a bt a g e n e  cil ol e u c el 
i nf usi o n d at e t o t h e  d at e  of  dis e as e  pr o gr essi o n  p er t h e  L u g a n o  Cl assifi c ati o n   {C h es o n  2 0 1 4 }  as 
d et er mi n e d  b y st u d y i n v esti g at ors  or  d e at h fr o m  a n y  c a us e.  S u bj e cts  n ot  m e eti n g t h e  c rit eri a f or 
pr o gr essi o n  or  d e at h  b y t h e  a n al ysis  d at a  c ut off  d at e  will  b e  c e ns or e d  at t h eir l ast  e v al u a bl e 
dis e as e  ass ess m e nt  d at e.  T h e  P F S f or s u bj e cts  w h o  u n d er g o  S C T  w hil e i n r e missi o n  will  b e 
c e ns or e d  at t h e l ast  e v al u a bl e  dis e as e  ass ess m e nt  pri or t o t h e  d at e  of  S C T; t h e  P F S f or s u bj e cts 
w h o  u n d er g o  ot h er  n e w  a nti- c a n c er t h er a pi es i n t h e  a bs e n c e  of  d o c u m e nt e d r el a ps e  will  b e 
c e ns or e d  at t h e l ast  e v al u a bl e  dis e as e  ass ess m e nt  pri or t o t h e  n e w  a nti -c a n c er t h er a pi es.  A 
s e nsiti vit y  a n al ysis  will  b e  c o n d u ct e d  i n  w hi c h  dis e as e  ass ess m e nts  o bt ai n e d  aft er  S C T  w hil e i n 
a xi c a bt a g e n e  cil ol e u c el i n d u c e d r e missi o n  ar e i n cl u d e d i n t h e  d eri v ati o n  of  P F S.  A d diti o n al 
d et ails  o n t h e  d eri v ati o n  of  P F S  ar e  pr o vi d e d i n  A p p e n di x  2 . 
O v e r all  S u r vi v al ( O S) :  O S is  d efi n e d  as t h e ti m e fr o m t h e a xi c a bt a g e n e  cil ol e u c el i nf usi o n  d at e 
t o t h e  d at e  of  d e at h.  S u bj e cts  w h o  h a v e  n ot  di e d  b y t h e  a n al ysis  d at a  c ut off  d at e  will  b e  c e ns or e d 
at t h eir l ast  d at e  k n o w n t o  b e  ali v e  or t h e  d at a  c ut off  d at e,  w hi c h e v er is  e arli er.   F urt h er  d et ails  o n 
t h e  d eri v ati o n  of  O S  a n d t h e s p e cifi c  d at a  m o d ul es t h at  will  b e  us e d t o  d eri v e t h e l ast  d at e  k n o w n 
t o  b e  ali v e  ar e  pr o vi d e d i n A p p e n di x  2 .    


K T E- C 1 9 -1 1 1  
Kit e  P h ar m a, I n c.  
St atisti c al  A n al ysis  Pl a n  
 
C O N FI D E N TI A L  
P a g e  1 8  
2 9  M ar c h  2 0 2 1  
6.   
A N A L Y SI S  S E T S  
T h e f oll o wi n g  a n al ys es s ets  ar e  d efi n e d f or t his st u d y. 
6. 1.   
F ull  A n al ysis  S et  
T h e f ull  a n al ysis s et  ( F A S) c o nsists  of  all  e nr oll e d s u bj e cts (i. e.  c o m m e n c es l e u k a p h er esis)  a n d 
will  b e  us e d f or s u m m ari es  of s u bj e ct  dis p ositi o n. 
6. 2.   
M o difi e d I nt e nt -t o-t r e at A n al ysis  S et  
T h e  m o difi e d i nt e nt- t o-tr e at ( mI T T)  a n al ysis s et c o nsists  of  all s u bj e cts  e nr oll e d  a n d  tr e at e d  wit h 
t h e t ar g et  d os e of  a xi c a bt a g e n e  cil ol e u c el  at   2  x  1 06 a nti  C D 1 9  C A R  T  c ells/ k g ( r a n g e  1. 0  x  1 06 
t o  2. 4  x  1 06) a n d  at l e ast  o n e  d os e  of  ut o mil u m a b  aft er  a xi c a bt a g e n e  cil ol e u c el i nf usi o n , as 
d et er mi n e d  u p o n  c o m pl eti o n  of t h e  P h as e  1  a n d  P h as e  2  p orti o ns  of t h e st u d y .  T his  a n al ysis s et 
will  b e  us e d f or  all  effi c a c y  a n al ys es.   
6. 3.   
S af et y  A n al ysis  S et 
T h e s af et y  a n al ysis s et  ( S A S) c o nsists  of  all s u bj e cts tr e at e d  wit h  a n y  d os e  of  a xi c a bt a g e n e 
cil ol e u c el.  
6. 4.   
D L T  A n al ysis  S et  
T h e  D L T  e v al u a bl e s et  c o nsists  of  all s u bj e cts i n  e a c h  P h as e  1  c o h ort  w h o  ar e tr e at e d  wit h 
a xi c a bt a g e n e  cil ol e u c el  a n d  at l e ast  o n e  d os e  of  ut o mil u m a b  w h o  eit h er:   
•   R e c ei v e d  t h e t ar g et  a xi c a bt a g e n e  cil ol e u c el  d os e  a n d  w er e f oll o w e d f or  at l e ast  2 8  d a ys  aft er 
t h e first  ut o mil u m a b i nf usi o n,  or 
•   R e c ei v e d  a  d os e  of  a xi c a bt a g e n e  cil ol e u c el  l o w er t h a n t h e t ar g et f or t h at  c o h ort  a n d  a 
s u bs e q u e nt  ut o mil u m a b i nf usi o n  a n d  e x p eri e n c e d  a  D L T  d uri n g t h e  2 8- d a y,  p ost- ut o mil u m a b 
i nf usi o n  p eri o d 
6. 5.   
S af et y  r et r e at m e nt  a n al ysis s et   
T h e s af et y r etr e at m e nt  a n al ysis s et  will i n cl u d e  all s u bj e cts  w h o  u n d er g o r etr e at m e nt  wit h  a n y 
d os e  of  a xi c a bt a g e n e  cil ol e u c el.  T his s et  will  b e  us e d f or  all r etr e at m e n t s af et y  a n al ys es. 
6. 6.   
mI T T  R et r e at m e nt  A n al ysis  S et   
T h e  mI T T r etr e at m e nt  a n al ysis s et  will  c o nsist  of  all s u bj e cts  w h o  u n d er g o r etr e at m e nt  wit h 
t ar g et  d os e  of  a xi c a bt a g e n e  cil ol e u c el.  T his s et  will  b e  us e d f or  all r etr e at m e nt  effi c a c y  a n al ys es. 


K T E- C 1 9 -1 1 1  
Kit e  P h ar m a, I n c.  
St atisti c al  A n al ysis  Pl a n  
 
C O N FI D E N TI A L  
P a g e  1 9  
2 9  M ar c h  2 0 2 1  
7.   
I N T E RI M  A N A L Y SI S  A N D  E A R L Y  S T O P PI N G  G UI D E LI N E S  
F or m al i nt eri m  a n al ysi s  of  effi c a c y  is  n ot  pl a n n e d f or t h e  e arl y tri al st o p pi n g  p ur p os e.  
7. 1.   
S af et y I nt e ri m  A n al ysis  
A n i nt er n al  S R T,  c o m prisi n g t h e st u d y s p o ns or  m e di c al  m o nit or,  dr u g s af et y  p h ysi ci a n, st u d y 
st atisti ci a n,  a n d  at l e ast o n e  a cti v e i n v esti g at or,  will  b e  c h art er e d t o r e vi e w s af et y  d uri n g  P h as e  1 
of t h e st u d y  a n d t o  m a k e r e c o m m e n d ati o ns  o n f urt h er st u d y  c o n d u ct i n  P h as e  1  a n d  pr o gr essi o n 
t o  P h as e  2  b as e d  o n t h e i n ci d e n c e of  i n v esti g ati o n al  pr o d u ct r el at e d  d os e-li miti n g t o xi citi es 
(D L Ts)   a n d r e vi e w  of s eri o us  a d v ers e  e v e nts ( S A Es).  
T h e  S R T  will  m e et  t o r evi e w t h e s af et y  d at a f oll o wi n g t h e  a c cr u al  a n d  c o m pl eti o n  of t h e  D L T 
ass ess m e nt  p eri o d  of  e a c h  P h as e  1  c o h ort, i. e.  f or  e a c h P h as e  1  c o h ort  aft er  all s u bj e cts i n  t h at 
c o h ort  h a v e  h a d t h e  o p p ort u nit y t o  c o m pl et e t h e  D L T  wi n d o w  ( 2 8  d a ys  aft er t h e l ast s u bj e ct i n  a 
c o h ort r e c ei v e d t h e first  ut o mil u m a b i nf usi o n).  
T h e  S R T  will  a d diti o n all y  m e et  o n  at l e ast  o n e  o c c asi o n  d uri n g t h e  P h as e  2  p orti o n  of t h e st u d y 
aft er  6 s u bj e cts  h a v e  c o m pl et e d t h eir  1 -m o nt h  dis e as e  ass ess m e nt.  T h e  S R T  will r e vi e w s af et y 
a n d  effi c a c y  d at a  a n d  will  m a k e tri al  c o n d u ct r e c o m m e n d ati o ns  b as e d  o n  a n  a n al ysis  of  b e n efit 
vs ris k. 
T h e  S R T  m a y  m e et  m or e  oft e n  as  n e e d e d.  


K T E- C 1 9 -1 1 1  
Kit e  P h ar m a, I n c.  
St atisti c al  A n al ysis  Pl a n  
 
C O N FI D E N TI A L  
P a g e  2 0  
2 9  M ar c h  2 0 2 1  
8.   
D A T A  S C R E E NI N G  A N D  A C C E P T A N C E  
8. 1.   
G e n e r al  P ri n ci pl es  
T h e  d at a b as e  will  b e s u bj e ct t o t h e  e dit  c h e c ks  o utli n e d i n t h e  D at a  M a n a g e m e nt  Pl a n  a n d 
a d diti o n al  m a n u al  d at a r e vi e ws  d efi n e d  b y t h e st u d y t e a m.  D at a i n c o nsist e n ci es  will  b e r e vi e w e d 
a n d r es ol v e d  b ef or e t h e  d at a b as e s n a ps h ot f or t h e  pri m ar y  a n al ysis  a n d t h e fi n al  d at a b as e l o c k.  
8. 2.   
El e ct r o ni c  T r a nsf e r  a n d  A r c hi vi n g   of  D at a 
T h e  M e di d at a  R a v e s yst e m  will  b e  us e d t o  c oll e ct t h e  d at a i n t his st u d y.  R a w  d at a  e xtr a ct e d fr o m 
M e di d at a  R a v e  will  b e  ar c hi v e d  pri or t o f urt h er  d at as et  cr e ati o n,  m ai nt e n a n c e,  a n d  a n al ysis. 
D at as ets (r a w  d at a, st u d y  d at a  t a b ul ati o n  m o d el [ S D T M]  d at a,  a n d/ or  a n al ysis  d at a  m o d el 
[ A D a M]  d at a) f or  pl a n n e d  a n al ys es  will  b e  ar c hi v e d.  A n y  a d diti o n al  u n pl a n n e d  a n al ys es t h at 
o c c ur  aft er t h e  pri m ar y  a n al ysis  a n d  pri or t o t h e fi n al  a n al ysis  will  als o  b e  ar c hi v e d.  K e y  d at a 
e xt er n al t o  t h e  cli ni c al st u d y  d at a b as e  will  b e i n cl u d e d i n t h e r el e v a nt  S D T M  a n d  A D a M 
m o d ul es  w h e n t h e  e xt er n al  d at a  ar e  a v ail a bl e.  
8. 3.   
H a n dli n g  of  Missi n g  a n d I n c o m pl et e  D at a  
8. 3. 1.   
Effi c a c y  
T h e  m et h o d f or  h a n dli n g  missi n g  d at a is  d es cri b e d i n t h e  d efi niti o n f or  e a c h  effi c a c y  e n d p oi nt. 
E v er y  eff ort  will  b e  m a d e t o  o bt ai n  c o m pl et e  d at es f or  d e at hs. I n t h e  e v e nt  of  a  p arti al  or  missi n g 
d e at h  d at e  a n d t h e  c orr es p o n di n g  c e ns ori n g  d at e f or s ur vi v al, t h e  al g orit h m i n  A p p e n di x  1  
( S e cti o n  1 2) will  b e  us e d.  
8. 3. 2.   
S af et y  
P arti al  A E st art  d at es  will  b e i m p ut e d. If  d at es  ar e  missi n g  or i n c o m pl et e f or  A E  st art  d at es, t h e 
al g orit h m  d efi n e d i n  A p p e n di x  1  ( S e cti o n  1 2)  will  b e  us e d.  C o m pl et el y  missi n g  d e at h  d at es  or 
d e at h  d at es  wit h  o nl y  a  y e ar r e p ort e d  will  n ot  b e i m p ut e d. 
8. 4.   
D et e cti o n  of  Bi as  
A listi n g  of s u bj e cts  wit h i m p ort a nt  pr ot o c ol  d e vi ati o ns  (I P D) will  b e  g e n er at e d.  T h e  d e vi ati o ns 
i n cl u d e d i n t his list  will i n cl u d e,  b ut  n ot  b e li mit e d t o,  vi ol ati o ns  of  eli gi bilit y  crit eri a  a n d  us e  of 
e x cl usi o n ar y  m e di c ati o n  d uri n g t h e st u d y.  L a c k  of  pr ot o c ol  c o m pli a n c e  will  b e  e v al u at e d  b y 
s u m m ari zi n g t h e  s u bj e ct i n ci d e n c e  of  I P D.  Hi g h r at es  of I P D m a y i n di c at e  bi as.  
8. 5.   
O utli e rs  
D es cri pti v e st atisti cs  m a y  b e  us e d t o i d e ntif y  p ot e nti al  o utli ers i n  a n y  k e y  v ari a bl es  a n al y z e d. 
S us p e ct e d  o utli ers  will  b e i n cl u d e d i n  all  a n al ys es  u nl ess t h er e is s uffi ci e nt s ci e ntif i c j ustifi c ati o n 
t o  e x cl u d e t h e m. 


K T E- C 1 9 -1 1 1  
Kit e  P h ar m a, I n c.  
St atisti c al  A n al ysis  Pl a n  
 
C O N FI D E N TI A L  
P a g e  2 1  
2 9  M ar c h  2 0 2 1  
8. 6.   
Dist ri b uti o n al  C h a r a ct e risti cs  
T h e  Cl o p p er- P e ars o n  ( a n  e x a ct i nt er v al) m et h o d is  us e d t o  g e n er at e  9 5 %  CI f or t h e  C R r at e.  T his 
m et h o d  ass u m es  t h at i n di vi d u al s u bj e ct r es p o ns es  ar e i n d e p e n d e nt  wit h  bi n o mi al  distri b uti o n. 
W h il e t h e  Cl o p p er- P e ars o n i nt er v al  pr o vi d es  a d e q u at e  c o v er a g e  pr o b a bilit y, it is  c o m m o nl y 
wi d er t h a n  n e c ess ar y  { Br o w n  2 0 0 2 } , l e a di n g t o  o v erl y  c o ns er v ati v e  esti m at es  of t h e l ow er  b o u n d 
of r es p o ns e r at e.  
8. 7.   
V ali d ati o n  a n d  C o nfi g u r ati o n  M a n a g e m e nt  
Pr o gr a ms f or t h e  d e v el o p m e nt  of t h e  S D T M  a n d  A D a M  d at as ets  a n d t h e  g e n er ati o n  of t h e t a bl es, 
fi g ur es,  a n d listi n gs  will  b e  d e v el o p e d  a n d  m ai nt ai n e d  a c c or di n g t o Kit e  P h ar m a’s  St a n d ar d 
O p er ati n g  Pr o c e d ur es ( S O Ps) if  a p pli c a bl e.  T h e s oft w ar e  a n d  v ersi o n  us e d t o  g e n er at e  a n al ys es 
will  b e i n di c at e d i n t h e  ar c hi v e d  d o c u m e nt ati o n. 


K T E- C 1 9 -1 1 1  
Kit e  P h ar m a, I n c.  
St atisti c al  A n al ysis  Pl a n  
 
C O N FI D E N TI A L  
P a g e  2 2  
2 9  M ar c h  2 0 2 1  
9.   
S T A TI S TI C A L  M E T H O D S  O F  A N A L Y SI S  
9. 1.   
G e n e r al  P ri n ci pl es  
A n al ys es  of t h e  P h as e  1  a n d  P h as e  2  of  t h e st u d y  will  b e pr es e nt e d s e p ar at el y ,  u nl ess  ot h er wis e 
s p e cifi e d. Wit hi n t h e  P h as e  1 s u m m ari es,  e a c h  c o h ort  will  b e  pr es e nt e d s e p ar at el y.  
T h e  pri m ar y  effi c a c y  a n al ysis  will  b e  p erf or m e d  w h e n t h e l ast tr e at e d s u bj e ct i n t h e  mI T T s et  h as 
h a d t h e   o p p ort u nit y t o  b e  e v al u at e d f or r es p o ns e  6  m o nt hs  aft er t h e  W e e k  4  dis e as e  ass ess m e nt.  
All s u bj e cts  will  b e f oll o w e d f or s ur vi v al f or  u p t o  a p pr o xi m at el y  1 5  y e ars  aft er t h e l ast s u bj e ct 
r e c ei v es  a xi c a bt a g e n e  cil ol e u c el.  N o f or m al  h y p ot h esis t esti n g  will  b e  p erf or m e d  b as e d  o n  d at a 
o bt ai n e d  aft er t h e  c ut off f or t h e  pri m ar y  a n al ysis.  D es cri pti v e  esti m at es  of  k e y  effi c a c y  a n d 
s af et y  a n al ys es  m a y  b e  u p d at e d t o  ass ess t h e  o v er all tr e at m e nt  pr ofil e.  
T h e fi n al  a n al ysis  will  o c c ur  w h e n  all s u bj e cts  h a v e  c o m pl et e d t h e st u d y.   A d diti o n al  a n al ys es 
f or i nt er n al r e vi e w  or  p u bli c ati o n  m a y  o c c ur  aft er t h e  pri m ar y  a n al ysis. 
9. 2.   
S u bj e ct  A c c o u nt a bilit y  
T h e  n u m b er  of s u bj e cts s cr e e n e d,  e nr oll e d  (i. e. l e u k a p h er es e d), tr e at e d  wit h  c o n diti o ni n g 
c h e m ot h er a p y, tr e at e d   a n d r e-tr e at e d wit h i n v esti g ati o n al tr e at m e nt s  will  b e s u m m ari z e d.  T h e 
r e as o ns f or  dis c o nti n ui n g tr e at m e nt  a n d  dis c o nti n ui n g st u d y  will  b e s u m m ari z e d. 
S u m m ari es  of  a ct u al  a n d  p ot e nti al f oll o w -u p ti m e  fr o m  a xi c a bt a g e n e  cil ol e u c el i nf usi o n will  b e 
pr o vi d e d.  A ct u al f oll o w -u p ti m e is  d efi n e d  as ti m e fr o m t h e first  d os e  of  a xi c a bt a g e n e  cil ol e u c el 
t o  d at e  of  d e at h  or t h e l ast  d at e  k n o w n  ali v e,  a n d  c al c ul at e d  as  d at e  of  d e at h  or t h e l ast  d at e 
k n o w n  ali v e -  a xi c a bt a g e n e  cil ol e u c el i nf usi o n  d at e  +  1.  P ot e nti al f oll o w -u p ti m e is  d efi n e d  as 
ti m e fr o m t h e first  d os e  of  a xi c a bt a g e n e  cil ol e u c el t o d at a  c ut off  d at e,  w hi c h  c o ul d  b e l at er t h a n 
d e at h  or t h e l ast  d at e  k n o w n  ali v e.  
T h e  n u m b er  of s u bj e cts  e nr oll e d  b y sit e  will  b e s u m m ari z e d. 
T h e  n u m b er  of s u bj e cts i n  e a c h  a n al ysis s et  al o n g  wit h r e as o ns f or  e x cl usi o n   will  b e  pr o vi d e d if 
d at a  ar e  a v ail a bl e . 
9. 3.   
I m p o rt a nt  P r ot o c ol  D e vi ati o ns  
T h e  cli ni c al st u d y t e a m  will  d efi n e  I P D c at e g ori es  a n d r e vi e w  all  p ot e nti al  I P D at  mi ni m u m, 
pri or t o t h e  d at a b as e s n a ps h ot f or t h e  pri m ar y  effi c a c y  a n al ysis.  I P Ds  will  b e  c at e g ori z e d  b y  
d e vi ati o n t y p e ( e g,  e ntr y  eli gi bilit y,  us e  of  e x cl u d e d  m e di c ati o n).  T h e s u bj e ct i n ci d e n c e  of  I P Ds 
will  b e s u m m ari z e d  o v er all  a n d  b y  d e vi ati o n  c at e g or y. 


K T E- C 1 9 -1 1 1  
Kit e  P h ar m a, I n c.  
St atisti c al  A n al ysis  Pl a n  
 
C O N FI D E N TI A L  
P a g e  2 3  
2 9  M ar c h  2 0 2 1  
9. 4.   
D e m o g r a p hi c  a n d  B as eli n e  C h a r a ct e risti cs  
St atisti cs  a n d fr e q u e n ci es f or t h e f oll o wi n g  d e m o gr a p hi c  a n d  b as eli n e  c h ar a ct eristi cs  will  b e 
t a b ul at e d: 
•   A g e (i n  y e ars)  at  b as eli n e  a n d  b y  c at e g or y ( <  6 5,  ≥  6 5)  
•   S e x  
•   Et h ni cit y  a n d r a c e 
•      W ei g ht at l e u k a p h er esis  
•   E C O G  p erf or m a n c e st at us  at  b as eli n e  
•   N u m b er  of  pri or  c h e m ot h er a p y r e gi m e ns  a n d  b est  o v er all r es p o ns e  t o t h e l ast  pri or r e gi m e n 
•   Pri or  A S C T  a n d  b est  o v er all r es p o ns e  c orr es p o n d i n g t o t h e  A S C T 
•   R efr a ct or y  s u b gr o u p ( pri m ar y r efr a ct or y, s e c o n d li n e  or  gr e at er, r el a ps e  p ost -A S C T)  
•   T u m or  b ur d e n,  as  m e as ur e d  b y t h e  S P D  of s el e ct e d  n o d es  or l esi o ns  at  b as eli n e  
•   D is e as e st a g e  at st u d y  e ntr y (I, II, III, I V) 
•   C ell  of  ori gi n  
•   Hist ol o gi c all y  pr o v e n  D L B C L t y p e 
•   Dis e as e  e xt e nt ( pr es e n c e  of  B s y m pt o ms,  S [s pl e ni c i n v ol v e m e nt],  E [ e xtr a n o d al  dis e as e],  X 
[ b ul k y  dis e as e],  b o n e  m arr o w i n v ol v e m e nt)  as  d et er mi n e d  b y t h e i n v esti g at or  at s cr e e ni n g 
•   D o u bl e/ Tri pl e  Hit  St at us  
•   I nt er n ati o n al  Pr o g n osti c I n d e x T ot al  S c or e  at  b as eli n e    
•   F or s u bj e cts  wit h   c yt o g e n eti cs t esti n g  p erf or m e d:  
   B C L - 2  alt er n ati o ns/ o v er- e x pr essi o ns ( Y/ N)  
   B C L - 6  alt er n ati o ns/ o v er- e x pr essi o ns ( Y/ N)  
   C- M Y C  alt er n ati o ns/ o v er- e x pr essi o ns ( Y/ N)   


K T E- C 1 9 -1 1 1  
Kit e  P h ar m a, I n c.  
St atisti c al  A n al ysis  Pl a n  
 
C O N FI D E N TI A L  
P a g e  2 4  
2 9  M ar c h  2 0 2 1  
9. 5.   
M e di c al  a n d  S u r gi c al  Hist o r y  
M e di c al   a n d s ur gi c al  hist or y  will  b e  c o d e d  usi n g t h e l at est  v ersi o n  of  M e d D R A.  T h e  n u m b ers 
a n d  p er c e nt a g es  of s u bj e cts  wit h  m e di c al  hist or y  will  b e r e p ort e d  b y  S O C  a n d  pr ef err e d t er m 
( P T).  
9. 6.   
P ri o r   T h e r a p y f o r  P ri m a r y  St u d y  Dis e as e a n d  P ri o r  R a di ot h e r a p y  
Pri or t h er a p y f or  D L B C L   will  b e  c o d e d  b y a n at o mi c al  t h er a p e uti c c h e mi c al  ( A T C)  c o d e  a n d  P T 
usi n g t h e l at est  v ersi o n  of  W orl d  H e alt h  Or g a ni z ati o n  Dr u g  Di cti o n ar y ( W H O Dr u g)  a n d  will  b e 
s u m m ari z e d i n fr e q u e n c y t a b ul ati o ns (s u bj e ct  c o u nts  a n d  p er c e nt a g es)  b y  P T.     
I nt e nt  of  pri or r a di ot h er a p y  a n d  b o d y sit e t o r e c ei v e t h e r a di ot h er a p y  will  b e s u m m ari z e d 
i n fr e q u e n c y t a b ul ati o ns (s u bj e ct  c ou nts  a n d  p er c e nt a g es).  
9. 7.   
Effi c a c y  A n al ys es 
Effi c a c y  a n al ys es  will  b e  c o n d u ct e d  o n t h e  mI T T  a n al ysis s et ,  a n d t h e i n v esti g at or  ass ess m e nt  of 
dis e as e st at us  p er  t h e  L u g a n o  Cl assifi c ati o n { C h es o n  2 0 1 4 }  will  b e  us e d  f or  dis e as e r es p o ns e 
r el at e d  a n al ys es.  
F or s u bj e cts r etr e at e d  wit h  a xi c a bt a g e n e  cil ol e u c el,  dis e as e  ass ess m e nts  o bt ai n e d  pri or t o 
r etr e at m e nt  will  b e i n cl u d e d i n t h e  pri m ar y s u m m ari es  of  o bj e cti v e  a n d  b est r es p o ns e,  D O R, 
P F S,  a n d s u m m ari es  of  c h a n g e i n t u m or  b ur d e n.  F or s u c h s u bj e cts,  dis e as e  ass ess m e nts  o bt ai n e d 
aft er r etr e at m e nt  will  b e i n cl u d e d i n t h e  s u m m ari es  of  o bj e cti v e  a n d  b est r es p o ns e t o r etr e at m e nt 
wit h  a xi c a bt a g e n e  cil ol e u c el  a n d  D O R  aft er r etr e at m e nt  wit h  a xi c a bt a g e n e  cil ol e u c el .  T h e 
s u bj e ct’s  O S ti m e  will  b e  d eri v e d fr o m t h e l ast  d at e  k n o w n  ali v e r e g ar dl ess  of r etr e at m e nt ti m e.  
9. 7. 1.   
C o m pl et e  R es p o ns e  a n d  O bj e cti v e  R es p o ns e  
9. 7. 1. 1.   
A n al ys es  of  R es p o ns e  R at e  
T h e s u bj e ct i n ci d e n c e  of  C R  a n d  o bj e cti v e r es p o ns e ( C R + P R)  will  b e  c al c ul at e d.  T w o -si d e d 9 5 % 
C o nfi d e n c e I nt er v als  will  b e  g e n er at e d  usi n g t h e  Cl o p p er- P e ars o n ( a n  e x a ct i nt er v al)  m et h o d.  
T h e  n u m b er  a n d  p er c e nt a g e   of s u bj e cts w h o  i niti all y  d o  n ot  att ai n  C R  a n d  w h o s u bs e q u e ntl y 
att ai n  a  C R  will  b e s u m m ari z e d .  
9. 7. 1. 2.   
S u b gr o u p  A n al ys es  
T h e r es p o ns e r at es  a n d  e x a ct  2 -si d e d  9 5 %  c o nfi d e n c e i nt er v als  will  b e  g e n er at e d f or s u b gr o u ps 
of t h e  mI T T  a n al ysis s et  b as e d  o n  b u t  n ot li mit e d t o t h e  c o v ari at es  d efi n e d i n S e cti o n  4. 2 .  A 
f or est  pl ot  of t h e  pr o p orti o n  of r es p o n d ers f or  e a c h  of t h es e s u bgr o u ps  will  b e  g e n er at e d. 


K T E- C 1 9 -1 1 1  
Kit e  P h ar m a, I n c.  
St atisti c al  A n al ysis  Pl a n  
 
C O N FI D E N TI A L  
P a g e  2 5  
2 9  M ar c h  2 0 2 1  
9. 7. 2.   
D u r ati o n  of  R es p o ns e  
T h e  n u m b er  of s u bj e cts  c e ns or e d  or  h a vi n g  e v e nts,  a n d t h e r e as o ns f or  c e ns ori n g  or t y p e  of 
e v e nts ( P D  or  d e at h)  will  b e s u m m ari z e d.  T h e  K a pl a n -M ei er  a p pr o a c h  will  b e  us e d t o  esti m at e 
D O R   a n d its q u artil es’   2-si d e d  9 5 %  c o nfi d e n c e i nt er val ,  as  w ell  as esti m at es  of t h e  pr o p orti o n  of 
s u bj e cts  ali v e  a n d i n r es p o ns e  at  3 - m o nt h i nt er v als. T h e r e v ers e  K a pl a n -M ei er  a p pr o a c h 
{ S c h e m p er  1 9 9 6 }  will  b e  us e d t o  esti m at e t h e f oll o w - u p ti m e f or  D O R.     
A s e nsiti vit y  a n al ysis  of  D O R  will  b e  c o n d u ct e d i n  w hi c h  dis e as e  ass ess m e nts  o bt ai n e d  aft er 
S C T (f or s u bj e cts  w h o  u n d er g o  S C T  w hil e i n  a n  a xi c a bt a g e n e  cil ol e u c el -i n d u c e d r es p o ns e)  ar e 
us e d i n t h e  d eri v ati o n  of  D O R.   
D O R  m a y  b e s u m m ari z e d i n s u b gr o u ps  d efi n e d  b y t h e  b est r es p o ns e  att ai n e d  o n st u d y. 
9. 7. 3.   
P r o g r essi o n  F r e e  S u r vi v al   
T h e  n u m b er  of s u bj e cts  c e ns or e d  or  h a vi n g  e v e nts,  a n d t h e r e as o ns f or  c e ns ori n g  or t y p e  of 
e v e nts ( P D  or  d e at h)  will  b e s u m m ari z e d.  K a pl a n -M ei er  pl ots,  q u artil e  esti m at es  a n d  2 -si d e d 
9 5 %  c o nfi d e n c e i nt er v als  will  b e  g e n er at e d f or  P F S   usi n g t h e  K a pl a n-M ei er  a p pr o a c h.  Esti m at es 
of t h e  pr o p orti o n  of s u bj e cts  ali v e  a n d  pr o gr essi o n- fr e e  at  3- m o nt h i nt er v als  will  b e  pr o vi d e d 
usi n g t h e s a m e  a p pr o a c h.  T h e r e v ers e  K a pl a n -M ei er  a p pr o a c h  will  b e  us e d  t o  esti m at e t h e 
f oll o w-u p ti m e f or  P F S . 
A s e nsiti vit y  a n al ysis  of  P F S  will  b e  c o n d u ct e d i n  w hi c h t h e  dis e as e  ass ess m e nts  o bt ai n e d  aft er 
S C T  will  b e i n cl u d e d i n t h e  d eri v ati o n  of  P F S .   S u b gr o u p  a n al ys es  of t h e  P F S r at e  at 6  m o nt hs 
m a y  b e  g e n er at e d i n s u b gr o u ps  d efi n e d  b y t h e  c o v ari at es i n  S e cti o n  4. 2 .  
P F S  m a y  b e s u m m ari z e d i n s u b gr o u ps  d efi n e d  b y t h e  b est r es p o ns e  att ai n e d  o n st u d y. 
9. 7. 4.   
O v e r all  S u r vi v al 
T h e  a n al ysis  of  O S  will  us e t h e s a m e  m et h o ds  as t h e  a n al ysis  of  P F S.   
O S  m a y  b e s u m m ari z e d i n s u b gr o u ps  d efi n e d  b y t h e  b est r es p o ns e  att ai n e d  o n st u d y. 
9. 7. 5.   
T u m o r  B u r d e n  
T h e  c h a n g e i n t u m or  b ur d e n,  as  m e as ur e d  b y t h e  S P D  of t h e s el e ct e d l esi o ns, fr o m  b as eli n e t o 
p ost- b as eli n e  n a dir  will  b e s u m m ari z e d i n  a bs ol ut e  n u m b ers ( m m 2)  a n d  p er c e nt a g e  c h a n g e.  
S u m m ar y st atisti cs  will  b e  pr o vi d e d f or t his  c h a n g e.  D at a  c oll e ct e d  aft er  n e w  a nti -c a n c er t h er a p y  
(i n cl u di n g  S C T)  will  n ot  be i n cl u d e d f or t h e  a n al ys es . 
9. 8.   
S af et y  A n al ys es 
S af et y  a n al ys es  will  b e  c o n d u ct e d  o n t h e s af et y  a n al ysis s et ,  wit h t h e  e x c e pti o n t h at  P h as e  1  D L T 
will  b e s u m m ari z e d i n t h e  D L T  a n al ysis s et .  T h e  pri m ar y  a n al ysis  of s af et y  d at a  will s u m m ari z e 
all  T E A Es  a n d l a b o r at or y  v al u es.  


K T E- C 1 9 -1 1 1  
Kit e  P h ar m a, I n c.  
St atisti c al  A n al ysis  Pl a n  
 
C O N FI D E N TI A L  
P a g e  2 6  
2 9  M ar c h  2 0 2 1  
A Es   will  b e  c o d e d  wit h t h e l at est  v ersi o n  of  M e d D R A.  T h e s e v erit y  of A E s  will  b e  gr a d e d  usi n g 
t h e l at est  v ersi o n  of  N ati o n al  C a n c er I nstit ut e ( N CI)  C o m m o n  T er mi n ol o g y  Criteri a f or  A d v ers e 
E v e nts ( C T C A E) .  T h e i n ci d e n c e  a n d s e v erit y  of  C yt o kin e r el e as e s y n dr o m e ( C R S)  will  b e 
gr a d e d  usi n g  a r e vis e d  C R S  gr a di n g s c al e  d e v el o p e d  b y  L e e  a n d  c oll e a g u es  { L e e  2 0 1 4 }. 
I n di vi d u al s y m pt o ms  ass o ci at e d  wit h  C R S  will  b e  gr a d e d  p er t h e l at est  v ersi o n  of  C T C A E. 
S u bj e cts  e nr oll e d , b ut  n ot  r e c ei v e d i n v esti g ati o n al tr e at m e nts, will  b e f oll o w e d f or  A Es f or 
3 0  d a ys  aft er t h e l ast st u d y  pr o c e d ur e.  A Es r e p ort e d i n t h es e s u bj e cts  will  b e  ar c hi v e d i n t h e 
st u d y  d at a b as e  a n d  a v ail a bl e i n  S D T M  a n d  A D a M  d at as ets,  b ut  will  n ot  b e t a b ul at e d i n  A E  
s u m m ari es. 
9. 8. 1.   
A d v e rs e  E v e nts   
D L Ts  o bs er v e d i n t h e  P h as e  1 s u bj e ct’s  D L T  wi n d o w  will  b e list e d  a n d t a b ul at e d  b y  S O C  a n d 
P T i n t h e  D L T  a n al ysis s et.  
T h e s u bj e ct i n ci d e n c e  of t h e f oll o wi n g  T E A Es  will  b e  t a b ul at e d i n t h e s af et y  a n al ysis s et:  
•   S u m m ar y  of  A E s ( a n y,  w orst s e v erit y, s eri o us, r el at e d , f at al)  
•   All  A Es   b y  S O C  a n d  P T 
•   All  S A Es   b y  S O C  a n d  P T 
•   All  a xi c a bt a g e n e  cil ol e u c el  a n d / or  ut o mil u m a b r el at e d A Es / S A Es  
•   All  Gr a d e  3  or  hi g h er  A Es   
•   All  G r a d e  3  or  hi g h er  a xi c a bt a g e n e cil ol e u c el  a n d / or  ut o mil u m a b r el at e d A Es   
•   A Es  of i nt er est, i n cl u di n g i d e ntifi e d ris ks  a n d  p ot e nti al ris ks   
•   A Es l e a di n g t o st u d y tr e at m e nt  dis c o nti n u ati o n  
•   Ot h er  cli ni c all y i m p ort a nt  a d v ers e r e a cti o ns   
•   F at al  A E  a n d  d e at h (t hr o u g h t h e l o n g t er m f oll o w- u p  p eri o d) 
S u bj e cts  wit h  pr ol o n g e d  c yt o p e ni as ( n e utr o p e ni a  or t hr o m b o c yt o p e ni a  or  a n e mi a)  will  b e 
i d e ntifi e d  wit h  c yt o p e ni as ( n e utr o p e ni a  or t hr o m b o c yt o p e ni a  or  a n e mi a)  pr es e nt  o n  or  aft er 
D a y   3 0  p ost  a xi c abt a g e n e  cil ol e u c el i nf usi o n   
S u m m ar y st atisti cs f or t h e st u d y  d a y  of  o ns et, t h e st u d y  d a y  of r es ol uti o n,  a n d t h e  d ur ati o n  of 
A Es  of i nt er est  will  b e  pr o vi d e d.  A s u bj e ct listi n g  of  d e at hs  a n d  S A Es  will  b e  pr o vi d e d  b y 
o v er all  a n d  b y tr e at m e nt  p eri o d.  
S u b gr o u p  a n al ys es  of  A Es  m a y  b e  g e n er at e d  usi n g t h e  c o v ari at es list e d i n  S e cti o n  4. 2  if 
a p pli c a bl e. 


K T E- C 1 9 -1 1 1  
Kit e  P h ar m a, I n c.  
St atisti c al  A n al ysis  Pl a n  
 
C O N FI D E N TI A L  
P a g e  2 7  
2 9  M ar c h  2 0 2 1  
9. 8. 2.   
L a b o r at o r y  T est  R es ults    
S el e ct e d  a n al yt es  of l a b or at or y r es ults  will  b e  gr a d e d  a c c or di n g t o  t h e l at est  v ersi o n  of  C T C A E.   
T h e  i n ci d e n c e  of  w orst  gr a d e  C T C A E  at  p ost-b as eli n e f or s el e ct e d  a n al yt es  will  b e  s u m m ari z e d. 
9. 8. 3.   
A nti -d r u g  A nti b o d y   
T h e  s u bj e ct i n ci d e n c e  of  a n y  a nti- a xi c a bt a g e n e  cil ol e u c el , a nti -ut o mil u m a b   a n d  n e utr ali zi n g 
a nti b o di es  will  b e t a b ul at e d.  F or s u bj e cts t esti n g  p ositi v e f or  a nti b o di es, t h e  p ersist e n c e  (i n 
m a g nit u d e [tit er]  o v er ti m e , ti m e  of  o ns et,  a n d  d ur ati o n) of t h e  a nti b o d y  will  b e s u m m ari z e d . 
9. 8. 4.   
R e pli c ati o n  C o m p et e nt  R et r o vi r us     
T h e s u bj e ct i n ci d e n c e  of  R C R  d et e ct e d i n  bl o o d s a m pl es  will  b e t a b ul at e d  o v er all  a n d  b y 
ass ess m e nt ti m e.  T h e  p ersist e n c e  of  R C R  o v er ti m e  will  b e s u m m ari z e d . 
9. 9.   
E x p os u r e t o  St u d y  T r e at m e nts  a n d  P r o d u ct  C h a r a ct e risti cs  
S u m m ar y st atisti cs  a n d s u bj e ct listi n gs  will  b e  pr o vi d e d f or t h e f oll o wi n g:  
•   T ot al  b o d y s urf a c e  ar e a ( B S A)- a dj ust e d  d os e  of  c y cl o p h os p h a mi d e  
•   T ot al  B S A - a dj ust e d  d os e  of fl u d ar a bi n e  
•   W ei g ht- a dj ust e d  d os e  of  a xi c a bt a g e n e  cil ol e u c el      
•   T ot al  C A R  T  c ells  of  t h e  a xi c a bt a g e n e cil ol e u c el i nf usi o n   
•   T ot al  T  c ells  of  t h e  a xi c a bt a g e n e cil ol e u c el i nf usi o n   
•   Tr a ns d u cti o n  p er c e nt a g e   
•   R ati o  of  C D 4  a n d  C D 8  T  c ells  
•   P er c e nt a g es  of  T  c ell  m e m or y  p h e n ot y p es   
•   I nt erf er o n  g a m m a (I F N-γ)  pr o d u cti o n i n  c o -c ult ur es  of  a xi c a bt a g e n e  cil ol e u c el  pr o d u ct  
•   N u m b er  of I nf usi o n  a n d   c u m ul ati v e  d os e    of ut o mil u m a b  
9. 1 0.   
E x p os u r e t o  C o n c o mit a nt  M e di c ati o ns  a n d  P r o c e d u r es  
T h e s u bj e ct i n ci d e n c e  of  c o n c o mit a nt  m e di c ati o ns  will  b e  pr o vi d e d  a n d s u m m ari z e d  b y 
m e di c ati o n  c at e g or y  ( g e n er al, i m m u n os u p pr essi v e,  a nti-i nf e cti v e,  v as o pr ess or,  c orti c ost er oi d, 
a n d t o cili z u m a b)  a n d  W H O Dr u g  c o d e d t er m.  T h e s u bj e ct i n ci d e n c e  of  pr o c e d ur es  will  b e 
t a b ul at e d. 


K T E- C 1 9 -1 1 1  
Kit e  P h ar m a, I n c.  
St atisti c al  A n al ysis  Pl a n  
 
C O N FI D E N TI A L  
P a g e  2 8  
2 9  M ar c h  2 0 2 1  
9. 1 1.   
S u bs e q u e nt  A nti -C a n c e r  T h e r a p y  
T h e i n ci d e n c e  a n d t y p e ( b y  W H O  Dr u g  c o d e d t er m  a n d  c at e g ori es)  of s u bs e q u e nt  a nti- c a n c er 
t h er a p y  a n d st e m  c ell tr a ns pl a nt ( a ut ol o g o us,  all o g e n ei c)  will  b e s u m m ari z e d.  
9. 1 2.   
D u r ati o n  M et ri cs   
S u m m ar y st atisti cs  will  b e  pr o vi d e d f or t h e f oll o wi n g  d ur ati o ns:  
•   D a ys fr o m l e u k a p h er esis t o  c o m m e n c e m e nt  of  c o n diti o ni n g  c h e m ot h er a p y 
•   D a ys fr o m l e u k a p h er esis t o  a d mi nistr ati o n  of  a xi c a bt a g e n e  cil ol e u c el   
•   D a ys fr o m  c o m m e n c e m e nt  of  c o n diti o ni n g  c h e m ot h er a p y t o  a d mi nistr ati o n  of  a xi c a bt a g e n e 
cil ol e u c el   
•   D ur ati o n  of  h os pit ali z ati o n f or  t h e  a xi c a bt a g e n e cil ol e u c el i nf usi o n  
9. 1 3.   
A xi c a bt a g e n e  Cil ol e u c el  D eli v e r y  T i m e 
S u m m ar y st atisti cs  will  b e  pr o vi d e d f or t h e f oll o wi n g  d eli v er y ti m e:  
•   D a ys fr o m l e u k a p h er esis t o  a xi c a bt a g e n e  cil ol e u c el r el e as e   
•   D a ys fr o m l e u k a p h er esis t o  d eli v er y  of  a xi c a bt a g e n e  cil ol e u c el  at st u d y sit e  a m o n g  d os e d 
s u bj e cts 
9. 1 4.   
P h a r m a c o ki n eti cs  
R ef er t o s e p ar at e  P K /P D  S A P.  
9. 1 5.   
P h a r m a c o d y n a mi cs 
R ef er t o s e p ar at e  P K /P D  S A P.  


K T E- C 1 9 -1 1 1  
Kit e  P h ar m a, I n c.  
St atisti c al  A n al ysis  Pl a n  
 
C O N FI D E N TI A L  
P a g e  2 9  
2 9  M ar c h  2 0 2 1  
1 0.   
C H A N G E S  F R O M  P R O T O C O L  S P E CI FI E D  A N A L Y S E S  
N ot  a p pli c a bl e. 


K T E- C 1 9 -1 1 1  
Kit e  P h ar m a, I n c.  
St atisti c al  A n al ysis  Pl a n  
 
C O N FI D E N TI A L  
P a g e  3 0  
2 9  M ar c h  2 0 2 1  
1 1.   
R E F E R E N C E S  
Br o w n  L D,  C ai  T T,  D as G u pt a  A.  C o nfi d e n c e I nt er v als f or  a  Bi n o mi al  Pr o p orti o n  a n d 
As y m pt oti c  E x p a nsi o ns.  T h e  A n n als  of  St atisti cs  2 0 0 2; 3 0 ( 1): 1 6 0- 2 0 1. 
C h es o n  B D,  Fis h er  RI,  B arri n gt o n  S F,  C a v alli  F,  S c h w art z  L H,  Z u c c a  E,  et  al. 
R e c o m m e n d ati o ns f or i niti al  e v al u ati o n, st a gi n g,  a n d r es p o ns e  ass ess m e nt  of 
H o d g ki n  a n d  n o n- H o d g ki n l y m p h o m a: t h e  L u g a n o  cl assifi c ati o n. J  Cli n  O n c ol 
2 0 1 4; 3 2 ( 2 7): 3 0 5 9- 6 8. 
L e e  D W,  G ar d n er  R,  P ort er  D L,  L o uis  C U,  A h m e d  N, J e ns e n  M,  et  al.  C urr e nt  c o n c e pts i n t h e 
di a g n osis  a n d  m a n a g e m e nt  of  c yt o ki n e r el e as e s y n dr o m e.  Bl o o d  2 0 1 4; 1 2 4 
( 2): 1 8 8- 9 5. 
S c h e m p er  M,  S mit h  T L.  A  n ot e  o n  q u a ntif yi n g f oll o w- u p i n st u di es  of f ail ur e ti m e.  C o ntr ol  Cli n 
Tri als  1 9 9 6; 1 7 ( 4): 3 4 3- 6.  
T o p p  M S,  G o k b u g et  N,  St ei n  A S,  Z u g m ai er  G,  O' Bri e n  S,  B ar g o u  R C,  et  al.  S af et y  a n d  a cti vit y 
of  bli n at u m o m a b f or  a d ult  p ati e nts  wit h r el a ps e d  or r efr a ct or y  B -pr e c urs or  a c ut e 
l y m p h o bl asti c l e u k a e mi a:  a  m ulti c e ntr e, si n gl e- ar m,  p h as e  2 st u d y.  L a n c et  O n c ol 
2 0 1 5; 1 6 ( 1): 5 7- 6 6. 
 


K T E- C 1 9 -1 1 1  
Kit e  P h ar m a, I n c.  
St atisti c al  A n al ysis  Pl a n  
 
C O N FI D E N TI A L  
P a g e  3 1  
2 9  M ar c h  2 0 2 1  
1 2.   
A P P E N DI C E S  
A p p e n di x  1.  
C o n v e nti o ns f or  Cli ni c al  D at a  T h at  R e q uir e I m p ut ati o n f or  P arti al  or  Missi n g  D at es  
A p p e n di x  2.  
D eri v ati o n  of  Ti m e t o  E v e nt  E n d p oi nts  
A p p e n di x  3.  
D eri v ati o n  of  L ast  d at e  k n o w n t o  b e  ali v e  


K T E- C 1 9 -1 1 1  
Kit e  P h ar m a, I n c.  
St atisti c al  A n al ysis  Pl a n  
 
C O N FI D E N TI A L  
P a g e  3 2  
2 9  M ar c h  2 0 2 1  
A p p e n di x  1.  
C o n v e nti o ns f o r  Cli ni c al  D at a  T h at  R e q ui r e I m p ut ati o n f o r  P a rti al  o r 
Missi n g  D at es  
T h e f oll o wi n g  d at a  will  b e i m p ut e d  usi n g t h e  al g orit h m s h o w n i n  T a bl e  2   b el o w: 
•   A d v ers e  e v e nt st art  d at es   
•   D e at hs (s e e  e x c e pti o ns  b el o w)   
•   C o n c o mit a nt  m e di c ati o n st art  d at es  
T a bl e  2. 
I m p ut ati o n  R ul es f o r  P a rti al  o r  Missi n g  St a rt  D at es  
St art  D at e  
St o p  D at e  
C o m pl et e:  
y y y y m m d d  
P arti al:   y y y y m m   
P arti al:   y y y y   
Missi n g  
<  d a y  0   
≥  d a y  0   
<  d a y  0 
y y y y m m  
≥  d a y  0  
y y y y m m  
<  d a y  0 
y y y y  
≥  d a y  0  
y y y y  
P arti al  
y y y y m m  
=  d a y  0  
y y y y m m  
2 
1 
2 
1  
n/ a   
1  
1 
≠  d a y  0  
y y y y m m  
2 
2  
2  
2  
2 
 
P arti al  
y y y y  
=  d a y  0  
y y y y  
3 
1 
3 
1  
n/ a   
1  
1 
≠  d a y  0   
y y y y  
3 
3  
3  
3  
3 
 
Missi n g  
4   
1   
4   
1   
4   
1   
1  
1  = i m p ut e t h e  d at e  of  d a y  0  
2  = i m p ut e t h e first  of t h e  m o nt h  
3  = i m p ut e J a n u ar y  1  of t h e  y e ar  
4  = i m p ut e J a n u ar y  1  of t h e st o p  y e ar  
N ot e:  if t h e st art  d at e i m p ut ati o n l e a ds t o  a st art  d at e t h at is  aft er t h e st o p  d at e, t h e n  d o  n ot  i m p ut e t h e st art  d at e. 
I m p ut ati o n  r ul es f o r  p a rti al  o r  missi n g  d e at h  d at es:  
1)   If  d e at h  y e ar  a n d  m o nt h  ar e  a v ail a bl e  b ut  d a y is  missi n g: 
•   If  m m y y y y f or t h e l ast  c o nt a ct  d at e  =  m m y y y y f or  d e at h  d at e, s et  d e at h  d at e t o t h e  d a y  aft er 
t h e l ast  d at e  k n o w n t o  b e ali v e.  
•   If  m m y y y y f or t h e l ast  d at e  k n o w n t o  b e  ali v e  <  m m y y y y f or  d e at h  d at e, s et  d e at h  d at e t o t h e 
first  d a y  of t h e  d e at h  m o nt h. 
•   If  m m y y y y f or l ast  d at e  k n o w n t o  b e  ali v e  >  m m y y y y f or  d e at h  d at e,  d at a  err or  a n d  d o  n ot 
i m p ut e. 
2)   If  b ot h  m o nt h  a n d  d a y  ar e  missi n g f or  d e at h  d at e  or  a  d e at h  d at e is  c o m pl et el y  missi n g,  d o 
n ot i m p ut e  a n d  c e ns or t h e s u bj e ct s ur vi v al ti m e  at t h e l ast  d at e  k n o w n t o  b e  ali v e. 


K T E- C 1 9 -1 1 1  
Kit e  P h ar m a, I n c.  
St atisti c al  A n al ysis  Pl a n  
 
C O N FI D E N TI A L  
P a g e  3 3  
2 9  M ar c h  2 0 2 1  
I m p ut ati o n  r ul es f o r  o ri gi n al  d at e  of  di a g n osis:   
1)   If  y e ar  a n d  m o nt h  ar e  a v ail a bl e  b ut  d a y is  missi n g, t h e n i m p ut e t h e first  d a y  of t h e  m o nt h.   
2)   If  y e ar is  a v ail a bl e  b ut  m o nt h  a n d  d a y  ar e  missi n g, t h e n i m p ut e J a n u ar y  1  of t h e  y e ar. 








K T E- C 1 9 -1 1 1  
Kit e  P h ar m a, I n c.  
St atisti c al  A n al ysis  Pl a n  
 
C O N FI D E N TI A L  
P a g e  3 7  
2 9  M ar c h  2 0 2 1  
A p p e n di x  3.    
D e ri v ati o n  of  L ast  d at e  k n o w n t o  b e  ali v e 
T h e l ast  d at e  k n o w n t o  b e  ali v e  will  b e  d eri v e d  b y  o bt ai ni n g t h e  m a xi m u m  c o m pl et e  d at e  a m o n g 
t h e f oll o wi n g  d at a  m o d ul es: 
•   St art  d at e  of  A E (i n cl u di n g t ar g et e d  A E)  
•   L e u k a p h er esis  d at e  
•   C o n diti o ni n g  c h e m o t h er a p y a d mi n  d at e  
•   A xi c a bt a g e n e  cil ol e u c el  i nf usi o n  d at e 
•   Ut o mil u m a b  i nf usi o n  d at e 
•   C T s c a n  d at e  
•   P E T s c a n  d at e  
•   T ar g et l esi o n  ass ess m e nt  d at e  
•   N o n -t ar g et l esi o n  ass ess m e nt  d at e 
•   N e w l esi o n  ass ess m e nt  d at e  
•   Dis e as e r es p o ns e  ass ess m e nt  d at e  
•   L o n g t er m f oll o w -u p s u bj e ct st at us  d at e  w h er e st at us  = ‘ ali v e’  
•   E n d  of tr e at m e nt  dis p ositi o n  w h er e st at us is  n ot  e q u al t o  d e at h  or l ost t o f oll o w  u p 
•   E n d  of  p ost- tr e at m e nt  ass ess m e nt  p eri o d  w h er e st at us is  n ot  e q u al t o  d e at h  or l ost t o f oll o w 
u p 
•   E n d  of st u d y  d at a  w h er e  e n d  of st u d y r e as o n is  n ot  e q u al t o  d e at h  or l ost t o f oll o w  u p