A Phase I Study of Stem Cell Gene Therapy for HIV Mediated by Lentivector Transduced, Pre-Selected CD34+ Cells
Phase: PHASE1
Conditions: HIV Infection, Mature T-Cell and NK-Cell Non-Hodgkin Lymphoma, Plasmablastic Lymphoma, Recurrent Adult Hodgkin Lymphoma, Recurrent Adult Non-Hodgkin Lymphoma, Recurrent Burkitt Lymphoma, Recurrent Follicular Lymphoma, Stage III Follicular Lymphoma, Stage III Mantle Cell Lymphoma, Stage IV Follicular Lymphoma, Stage IV Mantle Cell Lymphoma
Sponsor: AIDS Malignancy Consortium

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SUMMARY OF CHANGES 
A Phase I Study of Stem Cell Gene Therapy for HIV Mediated by Lentivector Transduced, 
Pre-Selected CD34+ Cells 
Version 9.0 
 
NCI Protocol #: AMC-097 
Local Protocol #: AMC-097 
 
NCI Version Date: 07FEB2022 
Protocol Date: 07FEB2022 
 
I. Recommendations from Approval with Recommendations Memorandum for Protocol 
Version 8.0 (dated 10SEP2021) from R. Little, dated 24SEP2020: 
# 
Section 
Comments 
1.  
Appendix VII 
2.5.4.1 
What is being measured by ELISA for Monitoring for immune response 
to TRIM5α? 
PI Response: This procedure was designed to be used if any immune 
response was being developed against the gene modified cells. We 
hypothesized that we would initially infer that an immune response 
toward the cells was occurring if the levels of in vivo gene marking 
decreased and eventually went to zero over time post-infusion. To 
date, none of the infused patients have displayed gene marking which 
would suggest an immune response toward the cells is occurring.  
Therefore, we have not needed to perform this assay. 
2.  
Appendix VII 
2.5.4.3, B. 
Please expand on as to how the team plan to Monitor of changes in the 
HIV reservoir and viral diversity in PBMCs?  
PI Response: As described in Section 2.5.4.3, B in order to 
characterize changes in the HIV reservoir and viral diversity blood 
samples will be collected at baseline, 2 and 10 weeks and then 6, 12- 
and 24-months post-transplant for HIV reservoir characterization. 
These studies will include quantitative PCR analysis of PBMC-
associated HIV DNA, RNA (including unspliced and multiply spliced 
forms) and 2-LTR circles. In addition, single genome sequencing of 
HIV gp120 and HIV pol will be performed to understand sequence 
evolution following transplantation and detection of minority 
resistance variants. Single-copy HIV from plasma will also be 
determined at each of these time points. Samples will be sent to the 
Timothy Henrich Laboratory at University of California, San 
Francisco. 

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II. 
Scientific and Substantive Changes: 
# 
Section 
Description of Change 
3.  
Protocol 
Schema 
2.4 
5.1 
The ART withdrawal period has been extended from 12 weeks to until at 
one of the ART resumption criteria are met. This will allow sufficient 
time for the viral load to spike and then decrease and provided more data 
for evaluating effects of the transformed cells. 
4.  
2.4 
2.4.2 
7.5 
App – 1C 
Appendix-XI 
Appendix XII 
Appendix XIII 
 
Monitoring before and during the ART withdrawal period has been 
updated to include required COVID-19 testing prior to ATI (and as 
needed) and weekly testing for CD4/CD8 counts, viral load, and clinical 
assessments (review of medical and medication history, height, weight, 
BSA, neurological exam, and toxicity assessment) to closely monitor for 
acute retroviral syndrome and opportunistic infections (OI). Clinical 
assessments may be performed remotely to minimize the study burden on 
participants.  
In addition, several appendices have been added including an ATI 
disclosure form for participants, ATI risk mitigation counseling forms, 
and PrEP information sheets for participants. The ATI disclosure form 
must be reviewed with participants prior to ATI. ATI risk mitigation 
counseling must be performed prior to-ATI and weekly during ATI, and 
may be performed remotely. PrEP information sheets should be given to 
participants as a resource prior to ATI.  
5.  
2.4.1 
2.4.2 
App – 1C 
 
ART resumption criteria have been revised to expand on the clinical and 
viral load safety parameters. The viral load criterion has been revised to 
require ART to be reinstated if there are persistent increases in HIV viral 
load over a period of time rather than relying on an extremely high single 
value. Participants with an initial viral load greater than 400 copies/ml 
during weekly testing must be retested within 3 business days or as soon 
as possible. Clinical criteria have been expanded to require ART 
resumption in the event of non-infectious symptoms suggestive of severe 
acute retroviral syndrome for consistency with clinical care standards and 
other HIV cure-intent protocols. 
6.  
2.4.3 
App – 1C 
 
Post ART withdrawal monitoring has been added. Participants will have 
CD4/CD8+T cell counts, HIV viral load, and clinical assessments at 4, 8, 
and 24 weeks after restarting ART to ensure that viral load and CD4 
counts remain at a safe level. If viral load is not below the level of 
quantification by Week 8, participants will be evaluated every four weeks 
until the viral load is confirmed to be less than the level of quantification. 
In addition, Plasma and PBMCs will be collected in four 10mL EDTA 

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Description of Change 
tubes and cryopreserved at the time of ART resumption and at weeks 4, 
8 and 24 after restarting ART. 
7.  
3.1.2.3 
Inclusion criterion was added for HIV viral load requirements, which 
include options for patients with higher viral loads if it can be determined 
which antiretroviral regimen can suppress the virus, as this is more 
consistent with clinical practice. 
8.  
4.2.1 
Dosing recommendations for BEAM and R-BEAM therapy have been 
eliminated to permit the use of biosimilars for rituximab, as sites are 
permitted to administer therapy per standard of care and their local 
institutional guidelines. In addition, rituximab biosimilars inclusion 
enables an alternative option in the event there is a supply shortage of 
rituximab at their institution.  
9.  
4.7 
Criteria for withdrawing from the study was revised. “Progression of 
Lymphoma” was removed as a criterion and participants will instead be 
followed for correlative studies as outlined in Section 7.5.8. 
10. 
7.5.6 
7.5.8.7 
7.6.1.6 
7.6.2.6 
App – 1B 
App – 1D 
Appendix VII 
The protocol has been updated to include optional bone marrow biopsy 
collections at 3 months, 12 months, and at study discontinuation for 
participants that do not have bone marrow involvement at baseline and 
that consent to optional studies. In addition, clarifications were added to 
distinguish requirements / purpose of required (restaging for those with 
bone marrow involvement) vs optional bone marrow biopsies (for 
research). 
11. 
7.5.8.2 
The University of California San Francisco (UCSF) is conducting the 
SCOPE study which includes an optional GI biopsy at the same 
timepoints as this study. The SCOPE study is an observational, 
prospective study of HIV-1 infected volunteers designed to provide a 
specimen bank of samples with carefully characterized clinical data. 
Specimens banked as part of the SCOPE study will be used to examine 
questions involving virologic, immunologic, and host factors involved in 
HIV-1 infection, progression, non-progression, response to treatment, 
control of HIV-1 virus, and evolution of drug resistance. Participants that 
agree to enroll into both the SCOPE study and AMC-097 at the same time 
(co-enroll) and agree to optional tissue collections will have their biopsy 
results shared for both studies to reduce the number of biopsies performed 
and facilitate enrollment to these optional studies. 

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12. 
7.7 
App – 1B 
Follow-up requirements have been revised to remove UC Davis as the 
responsible party for contacting participants and/or their providers during 
study required follow-up. Each site will contact the participants they 
enrolled. 
13. 
App – 1B 
App – 1D 
4.6 
7.7 
Added optional blood sample collections every 12 months for years 3-15 
for HIV viral load, and DNA and immunologic correlative studies to 
monitor participant’s HIV and vector parameters in the body. 
 
III. 
Administrative and Editorial Changes: 
# 
Section 
Description of Change 
14. 
Global 
The protocol version was changed from 8.0 to 9.0 and the date from 
10SEP2020 to 07FEB2022. 
15. 
Global 
Editorial and administrative changes were applied to spelling, grammar, 
punctuation, and table/figure numbering. 
16. 
Global 
Replaced “subject” with “participant” throughout. 
17. 
Protocol 
Synopsis 
1.3.2.5 
1.3.2.6 
2.3 
9.1 
The definition of ANC and Platelet engraftment has been clarified to 
include the underscore line with the greater than symbol.  The “>” 
(greater than) is changed to ‘>/=’ (greater than or equal) to be consistent 
with other sections of the protocol which state ‘absolute neutrophil count 
of at least 500 cells/mm3 and platelet count of at least 20,000 cells/mm3 
 
18. 
2.4.1 
2.4.2 
2.4.3 
Subsections for ART resumption criteria, ART monitoring, and post-
ART monitoring have been added for clarity. 
19. 
3.5 
Appendix 
VII 
Corrected the EDC participant ID obtained after registration from nine 
digit to eleven digits. 
20. 
5.1 
Removed definition of CD4+T cells for brevity. 

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21. 
7.4.2 
Updated conditioning therapy to include R-BEAM as an option for 
consistency with Section 4.2.1. 
22. 
Appendix I- 
App – 1D 
Sample collections for CCR5-Tropism were added for consistency with 
the screening evaluations in Section 7.1. Collections for immunology 
studies were updated to indicate specimens are to be collected in four 
10mL EDTA tubes and one 5 mL redtop tube at 3, 6, and 12 months post-
transplant and treatment discontinuation for consistency with Appendix 
VII. In addition, footnotes were added for GI biopsy collections to reflect 
that an specimen collections are optional and that GI biopsies may be 
delayed until the participant’s CD4 count has reached 300 cells/mm3 and 
the biopsy repeated 2 months after the first collection for consistency with 
Section 7.5.8.2 and Appendix I.   
23. 
Appendix VI AMC Data and Safety Monitoring Plan was updated to version 9.0. Key 
revisions include revision to state that the IRB review plan is identified 
in the protocol, and will continue to reference situations that apply for 
both domestic and international trials, as the AMC is preparing to open 
international trials where the requirements for US IRB review and 
international IRB review will be specific to the region. 

AMC-097 (Version 9.0) 07FEB2022 
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AMC PROTOCOL #097: 
A Phase I Study of Stem Cell Gene Therapy for HIV Mediated by 
Lentivector Transduced, Pre-Selected CD34+ Cells 
A Trial of the AIDS Malignancy Consortium (AMC) 
 
Sponsored by: 
National Cancer Institute 
Office of HIV and AIDS Malignancy (OHAM) 
 
NCT Registration 
Number: 
NCT02797470 
 
Investigational Agent: 
Autologous CD34+ cells transduced with a pre-
selective marker and triple combination of anti-
HIV genes (NSC 784725) 
 
IND #: 
16129 
Sponsor-Investigator: Mehrdad Abedi, MD 
 
Protocol Chair: 
Mehrdad Abedi, MD 
 
Protocol Co-Chair: 
Joseph Anderson, PhD 
Ariela Noy, MD 
Version 9.0, 07FEB2022  
NCI Version Date 07FEB2022  

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AMC PROTOCOL SIGNATURE PAGE 
I, ____________________, Principal Investigator at site ________, agree to conduct and follow 
this protocol: AMC Protocol # 097 – A Phase I Study of Stem Cell Gene Therapy for HIV 
Mediated by Lentivector Transduced, Pre-Selected CD34+ Cells (Version 9.0, 07FEB2022), 
as written according to AMC, NCI, International Conference on Harmonization guideline for Good 
Clinical Practice E6, the Declaration of Helsinki, and FDA guidelines. I understand that no 
deviations from the protocol eligibility criteria or waivers for protocol deviations will be permitted. 
 
 
_________________________________ 
 
______________________ 
Signature 
Date (DDMMMYYYY) 
 
 

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TABLE OF CONTENTS 
SUMMARY OF CHANGES ................................................................................................................ i 
I. 
Recommendations from Approval with Recommendations Memorandum 
for Protocol Version 8.0 (dated 10SEP2021) from R. Little, dated 
24SEP2020:.............................................................................................................. i 
II. 
Scientific and Substantive Changes: ....................................................................... ii 
III. 
Administrative and Editorial Changes: .................................................................. iv 
AMC PROTOCOL SIGNATURE PAGE ......................................................................................... 2 
TABLE OF CONTENTS .................................................................................................................... 3 
LIST OF TABLES AND FIGURES ................................................................................................... 6 
PROTOCOL ROSTER ....................................................................................................................... 8 
PROTOCOL SYNOPSIS .................................................................................................................... 9 
PROTOCOL SCHEMA .................................................................................................................... 12 
LIST OF ABBREVIATIONS ........................................................................................................... 13 
1.0 
OBJECTIVES ...................................................................................................................... 18 
1.1 
Hypothesis..............................................................................................................18 
1.2 
Primary Objective ..................................................................................................18 
1.3 
Secondary Objectives.............................................................................................18 
1.4 
Exploratory Objectives ..........................................................................................19 
2.0 
BACKGROUND .................................................................................................................. 20 
2.1 
Study Disease .........................................................................................................20 
2.2 
Study Agent: Autologous CD34+ Cells Transduced with a Pre-Selective 
Marker and a Triple Combination of Anti-HIV Genes ..........................................22 
2.3 
Study Design and Rationale ...................................................................................23 
2.4 
ART Withdrawal / Analytic Treatment Interruption (ATI) ...................................26 
2.5 
Correlative Studies .................................................................................................30 
3.0 
PARTICIPANT SELECTION ........................................................................................... 49 
3.1 
Eligibility Criteria ..................................................................................................49 
3.2 
Exclusion Criteria ..................................................................................................52 
3.3 
Number of Participants to be Enrolled...................................................................53 
3.4 
Recruitment Methods .............................................................................................54 
3.5 
Participant Enrollment Procedures ........................................................................55 
4.0 
TREATMENT PLAN .......................................................................................................... 56 
4.1 
HSC Mobilization and Peripheral Stem Cell Collection .......................................56 

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4.2 
Agent Administration.............................................................................................57 
4.3 
Definition of Dose-Limiting Toxicity ....................................................................59 
4.4 
General Concomitant Medication and Supportive Care Guidelines ......................60 
4.5 
Duration of Therapy ...............................................................................................60 
4.6 
Duration of Follow Up ...........................................................................................61 
4.7 
Withdrawal of Participants ....................................................................................61 
5.0 
ADVERSE EVENTS: LIST AND REPORTING REQUIREMENTS ........................... 62 
5.1 
Comprehensive Adverse Events and Potential Risks Lists....................................62 
5.2 
Classification of AEs by Severity and Relationship to Study Drug 
Administration .......................................................................................................64 
5.3 
Expedited Adverse Event Reporting ......................................................................64 
5.4 
Routine Adverse Event Reporting .........................................................................68 
5.5 
Clinical Laboratory Results ...................................................................................68 
5.6 
Secondary Malignancy...........................................................................................68 
5.7 
Second Malignancy ................................................................................................69 
6.0 
PHARMACEUTICAL INFORMATION .......................................................................... 70 
6.1 
Autologous CD34+ Cells Transduced with a Pre-selective Marker and 
Triple Combination of Anti-HIV Genes, IND Agent (NSC # 784725) .................70 
7.0 
CLINICAL AND LABORATORY EVALUATIONS ...................................................... 77 
7.1 
Screening Evaluations Required Within 8 Weeks Prior to Stem Cell 
Collection (unless otherwise noted) .......................................................................77 
7.2 
Screening Evaluations required within 1 week of admission for stem cell 
transplant ................................................................................................................78 
7.3 
Required on the day of admission for transplant prior to start of 
chemotherapy .........................................................................................................79 
7.4 
Evaluations During Treatment ...............................................................................79 
7.5 
Post-Treatment Follow-up Evaluations .................................................................79 
7.6 
Discontinuation Evaluations ..................................................................................82 
7.7 
Post-Study Follow-up ............................................................................................83 
8.0 
MEASUREMENT OF EFFECT ........................................................................................ 84 
8.1 
Response Parameters .............................................................................................84 
8.2 
Frequency of Response Evaluation ........................................................................84 
8.3 
Safety Measures .....................................................................................................84 
9.0 
STATISTICAL CONSIDERATIONS ............................................................................... 85 

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9.1 
Study Design/Endpoints.........................................................................................85 
9.2 
Sample Size/Accrual Rate ......................................................................................86 
9.3 
Early Stopping Rules .............................................................................................86 
9.4 
Cohort Escalation Design ......................................................................................86 
9.5 
Analysis of Secondary Endpoints ..........................................................................86 
9.6 
AMC Policy for Monitoring of Phase II Trials ......................................................87 
10.0 
ROLE OF DATA MANAGEMENT .................................................................................. 89 
10.1 CRF Instructions ....................................................................................................89 
10.2 Data Quality ...........................................................................................................89 
10.3 Data Monitoring .....................................................................................................89 
11.0 
ETHICAL AND REGULATORY CONSIDERATIONS ................................................ 90 
11.1 IRB Approval and Informed Consent ....................................................................90 
11.2 Multi-Site Research ...............................................................................................90 
11.3 Data Management and Confidentiality ..................................................................91 
11.4 Changes to the Protocol .........................................................................................92 
11.5 Vulnerable Populations ..........................................................................................92 
11.6 Non-English-Speaking Participants .......................................................................92 
11.7 Women and Minorities ..........................................................................................92 
11.8 Compensation for Research-Related Injury ...........................................................93 
11.9 Economic Burden to Participants...........................................................................93 
12.0 
REFERENCES ..................................................................................................................... 94 
APPENDIX I: SCHEDULE OF EVALUATIONS ......................................................................... 97 
App – 1A: Pre-Transplant Screening; Mobilization Period .............................................97 
App – 1B: Post-Transplant Follow Up .............................................................................99 
App – 1C: Analytic Treatment Interruption ...................................................................101 
App – 1D: Sample Collection guidance .........................................................................103 
APPENDIX II: PERFORMANCE STATUS SCALES ................................................................ 105 
APPENDIX III: AIDS AND CANCER SPECIMEN RESOURCE (ACSR) SPECIMEN 
PREPARATION AND SHIPPING INSTRUCTIONS ................................................... 106 
APPENDIX IV: ACSR INFORMED CONSENT ......................................................................... 109 
APPENDIX V: CONSENT FORM FOR GI BIOPSIES .............................................................. 114 
APPENDIX VI: AMC DATA AND SAFETY MONITORING PLAN ...................................... 120 
APPENDIX VII: CORRELATIVE STUDY SPECIMEN PREPARATION AND SHIPPING 

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INSTRUCTIONS ............................................................................................................... 125 
APPENDIX VIII: INTERNATIONAL PROGNOSTIC INDEX (AGE-ADJUSTED) ............. 137 
APPENDIX IX: CCR5-TROPISM ASSAY .................................................................................. 138 
APPENDIX X: RISK LIST FOR BEAM ...................................................................................... 139 
APPENDIX XI: ATI RISK MITIGATION COUSENLING FORMS: ...................................... 141 
APPENDIX XII: HIV TREATMENT INTERRUPTION PARTICIPANT DISCLOSURE 
FORM: ................................................................................................................................ 143 
APPENDIX XIII: PREP INFORMATION SHEET FOR PARTICIPANTS ............................ 144 
 
 
LIST OF TABLES AND FIGURES  
Table 2-A: Study Cohorts ................................................................................................................. 24 
Figure 2-B: Enrichment of 1TAX vector transduced cells: Human CD34+ HPC were 
transduced with the 1TAX vector, purified by CD25 immunomagnetic beads, and analyzed by 
flow cytometry for CD25 expression. Non‐ transduced cells (NT), flow through (ft) .................. 32 
Figure 2-C: Down regulation of CCR5 expression on 1TAX cells: Ghost‐R5/X4/R3 cells 
transduced with the 1TAX vector were analyzed by flow cytometry for CCR5 cell surface 
expression. A representative histogram is displayed with NT cells (unshaded) and 1TAX vector 
transduced cells (shaded). Experiments were performed in at least triplicate. *** p<0.001 ...... 33 
Figure 2-D: Phenotypic profile of 1TAX vector transduced peripheral blood T cells: Human 
CD34+ HSC were transduced with the 1TAX vector and transplanted into NRG mice. The 
peripheral blood of engrafted mice was analyzed by multi-parametric flow cytometry for T cell 
phenotypes .......................................................................................................................................... 34 
Figure 2-E: HIV‐1 challenge of 1TAX HSC derived macrophages: A) Nontransduced (NT) (♦) 
and 1TAX vector transduced (■) HSC derived macrophages were challenged with a CCR5-
tropic BaL-1 strain of HIV-1. On multiple days post‐infection, cell culture supernatants were 
sampled and assessed for HIV-1 using a p24 antigen ELISA kit. Experiments were performed 
in triplicate. * p<0.05, ** p<0.01, *** p<0.001 ................................................................................ 35 
Figure 2-F: Maintenance of human CD4+ T cells in vivo in 1TAX cell engrafted NRG mice 
infected with HIV‐1: NRG mice engrafted with either untransduced (NT) or 1TAX vector 
transduced cells were infected intraperitoneally with a (A) CCR5‐tropic BaL‐1 or a (B) 
CXCR4‐tropic NL43 strain of HIV‐1. Various weeks post‐infection, mice were bled via tail vein 
and analyzed by flow cytometry for total human CD3+/CD4+ cell percent. Bar graphs display 
averages and standard deviations from seven mice per cohort for the BaL‐1 infections and four 
mice per cohort for the NL43 infections for CD3+/CD4+ cell levels pre-infection and 15 weeks 
post‐infection. * p<0.05 and ** p<0.002 ........................................................................................... 37 
Figure 2-G: Reduction in HIV-1 plasma viremia in NRG mice engrafted with 1TAX vector 
transduced HSC: NRG mice engrafted with either untransduced (NT) or 1TAX vector 
transduced cells were infected intraperitoneally with a (A) CCR5‐tropic BaL‐1 or a (B) 
CXCR4-tropic NL43 strain of HIV‐1. Various weeks post-infection, mice were bled via tail vein 
and analyzed by qPCR for plasma viremia using a primer/probe set specific for the HIV LTR. 

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Log RNA copies/mL for NT (♦) (solid grey lines), 1TAX vector transduced (■) (solid black 
lines), and background levels from uninfected (UI) mice (▲) (dashed grey lines). Line graphs 
display averages and standard deviations from seven mice per cohort for the BaL‐1 infections 
and four mice per cohort for the NL43 infections. * p<0.05 and ** p<0.005. .............................. 38 
Table 4-A: Dose of Transduced Versus Untransduced Cells ......................................................... 57 
Table 4-B: Criteria for dose escalation ............................................................................................ 59 
Table 7A: Follow-Up Schedule ......................................................................................................... 79 
Table 11-A: Accrual Targets ............................................................................................................. 93 
The schedule during the ATI period will be defined by clinical course and the characteristics of 
any virus rebound. ART will be initiated for clinical events, sustained drop in CD4+T cell 
counts or sustained increased in viremia as described in Section 2.4.1. Prior to these events, 
participants will be followed. If collection timepoints for CD4/CD8 and HIV VL overlap with 
collections as part of the Follow-up Evaluations, then they do not need to be repeated. Clinical 
laboratory testing, assessments, and ATI counseling may be performed remotely to minimize 
in-person visits during the ATI.  ..................................................................................................... 101 
 
 

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PROTOCOL ROSTER 
AMC Protocol # 097 
A Phase I Study of Stem Cell Gene Therapy for HIV Mediated by Lentivector Transduced, 
Pre-Selected CD34+ Cells 
Protocol Chair: 
Mehrdad Abedi, MD 
University of California Davis 
Comprehensive Cancer Center 
4501 X Street 
Sacramento, CA 95817 
Tel: (916) 734-3772 
Fax: (916) 734-7946 
Email: mabedi@ucdavis.edu 
Protocol Statistician: 
Jeannette Lee, PhD 
University of Arkansas for Medical Sciences 
4301 West Markham Street 
Tel: 501-526-6712 
Fax: 501-526-6729 
Email: jylee@uams.edu 
Protocol Co-Chair: 
Joseph Anderson, PhD 
University of California Davis 
2921 Stockton Blvd. Room 1300 
Sacramento, CA 95817 
Tel: 916-703-9300 
Fax: 916-703-9310 
Email: jsanderson@ucdavis.edu 
Data Management/Operations: 
AMC Operations and Data Management 
Center 
The Emmes Company, LLC 
401 N. Washington Street, Suite 700 
Rockville, MD 20850 
Tel: (301) 251-1161 
Fax: (240) 238-2842 
Email: amcpm@emmes.com 
Protocol Co-Chair: 
Ariela Noy, MD 
Memorial Sloan-Kettering Cancer Center 
Lymphoma Service 
1275 York Avenue 
New York, NY 10065 
Tel: (212) 639-7423 
Fax: (646) 422-2284 
Email: noya@mskcc.org 
AMC Hematologic Malignancy Working 
Group Chair: 
Richard Ambinder, MD, PhD 
Oncology Center 
Bunting-Blaustein Cancer Research Building 
1650 Orleans Street, Room 389 
Baltimore, MD 21231Tel: (410) 955-8839 
Fax: (410) 955-0960 
Email: ambinri@jhmi.edu 
AMC Biorepository Director: 
Sylvia Silver, DA 
George Washington University Medical 
Center 
AMC Biorepository 
Ross Hall, Room 118 
2300 I St, NW 
Washington, DC 20037 
Tel: (202) 994-2945 
Fax: (202) 994-5056 
Email: ssilver@gwu.edu 
 

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PROTOCOL SYNOPSIS 
Title: 
A Phase I Study of Stem Cell Gene Therapy for HIV Mediated by 
Lentivector Transduced, Pre-Selected CD34+ Cells 
Phase of Study: 
Phase I 
Methodology 
Open label 
Participating 
Institutions: 
This protocol will be open initially to UC Davis, UC San Diego, and UC 
San Francisco Medical Centers. Upon completion of the first cohorts 
additional AMC sites will be added dependent on the recruitment rate. 
A decision on any additional participating trial sites will be made after 
consultation with the AMC Hematologic Malignancy Working Group. 
Memorial Sloan Kettering Cancer Center was added as a site in AUG 
2019. 
Accrual Target: 
This study will enroll a minimum of 2 participants and a maximum of 
18 participants. 
Population: 
Male or female participants greater than 18 years of age, with HIV-1 
related lymphomas that are refractory/resistant to first line of therapy or 
relapse after achieving a remission and are eligible for an autologous 
HSCT as a part of their routine therapy are eligible for this study. 
Participants must be on a multi-drug anti-HIV regimen (excluding 
zidovudine [AZT, ZDV, Retrovir®, or agents containing zidovudine 
(e.g., Combivir® and Trizivir®)] and efavirenz [EFV, Sustiva®, or agents 
containing efazirenz (e.g., Atripla®)]) and have an HIV-1 viral load < 50 
copies/mL by RT-PCR at the time of study enrollment. 
Regimen: 
For the first 2 cohorts, both transduced (autologous CD34+ cells 
transduced with a pre-selective marker and a triple combination of anti-
HIV genes) and un-manipulated product will be infused within 4 to 24 
hours of each other. After DSMB approval, only transduced product will 
be administered to the rest of the participants. In case of non-
engraftment, the back-up product stored at the respective site will be 
infused. The number of cells infused to each participant depends on the 
cohort in which the participant is enrolled. The number of cells in the 
transduced products is based on CD34 counts after transduction of the 
stem cells and before freezing. 
Study 
Cohorts 
Ratio of 
Transduced vs 
Un-transduced 
Stem Cells 
Minimum Number of 
Transduced/Un-
transduced Cells 
Acceptable. CD34 
cells/kg 
Range for Number of 
Transduced 
Cells/Unmanipulated. 
CD34 cells/kg 
1 
1:1 
1 X 106:1 X 106 
1 to 5 X 106: 1 to 5 X 
106 
2 
5:1 
2 X 106:1 X 106 
2 to 5 X 106:1 X 106 

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3 
1:0 
2 X 106: 0 
2 to 10 X 106: 0 
3+ 
1:0 
3.0 X 106: 0 
3.0 to 10 X 106: 0 
Duration: 
The initial follow-up period for this clinical trial will be two years; 
however, all participants will be followed for 15 years for safety, as 
mandated by FDA for all gene therapy study participants. 
Primary Objective: To evaluate the clinical safety of our approach using our development 
candidate (i.e., anti-HIV gene modified hematopoietic stem cells). See 
Section 1.2 for endpoint definitions. 
Secondary 
Objectives: 
To evaluate immune reconstitution with HIV-1 resistant immune cells, 
viral load and HIV-1 status, integration sites of vector sequences in 
circulating cells, presence of any in vivo clonal expansion of transduced 
cells, and any immune reaction to transduced cells. 
Major Secondary Objectives 
• To determine the efficacy of our approach using our development 
candidate. 
• To determine the presence, quantity, and duration of gene modified 
HIV-1 resistant peripheral blood cells and gut mucosal immune cells. 
• To study the integration sites of vector sequences in circulating cells. 
Minor Secondary Objectives 
• To study progression-free survival. 
• To study overall survival. 
• To study complete response rate and duration. 
• To study partial response rate and duration. 
• To study time to neutrophil engraftment (first measurement of 3 
consecutive laboratory values obtained on different days) of ANC > 
/= 500 cells/mm3. 
• To study time to platelet engraftment (first measurement of 3 
consecutive laboratory values obtained on different days) of platelets  
> /= 20,000 cells/mm3 without platelet transfusions 7 days prior). 
• To study hematologic function at Day 100 (ANC > 1500, Hb > 
10g/dl without transfusion and platelets > 100,000) 
• To study CD4 recovery at the conclusion of the trial. 
• To study safety in terms of toxicities, infections, transfusions, and 
infusion-related reactions. 
• To study HIV-1 viral load over time. 

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• To study persistence of vector-transduced cells over time. 
Exploratory 
Objectives: 
To explore the role of anti-retroviral therapy (ART) interruption on 
expansion of HIV-1 resistant HIV-1 target cells, including immune cells, 
both in the peripheral blood and the gut mucosa. 
Statistical 
Methodology 
Three participants will be enrolled in each cohort with potential 
expansion of the cohort to 6 with the maximum of 18 participants. 
The primary endpoint of the study is safety. 
Safety is defined as timely engraftment (the collective establishment of 
a persistent absolute neutrophil count of at least 500 cells/mm3 and 
platelet count of at least 20,000 cells/mm3 for 3 consecutive 
measurements of laboratory values obtained on different days) within 
one month post-transplant, in the absence of any study candidate-
specific grade 3 and 4 non-hematopoietic organ toxicity, excluding 
alopecia, or any clonal expansion. 

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PROTOCOL SCHEMA 
 
 
 
*ART reinstitution will occur if / when participants satisfy one of the ART resumption criteria in 
section 2.4.1. 
 
 

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LIST OF ABBREVIATIONS 
ACSR ................................................................................... AIDS and Cancer Specimen Resource 
AdvantageEDCSM .......................................................................... AMC internet data entry system 
AE ................................................................................................................................ adverse event 
AIDS .................................................................................... acquired immunodeficiency syndrome 
AMC ................................................................................................ AIDS Malignancy Consortium 
AML ........................................................................................................ acute myelocytic leukemia 
ANC .......................................................................................................... absolute neutrophil count 
ART.................................................................................................................. antiretroviral therapy 
ATI .................................................................................................... analytic treatment interruption 
AZT .................................................................................................................................. zidovudine 
BCNU .............................................................................................................................. carmustine 
BEAM ................................................................. carmustine, etoposide, cytarabine, and melphalan 
R-BEAM ........................................... rituximab, carmustine, etoposide, cytarabine, and melphalan 
BID .................................................................................................................bis in die (twice daily) 
BSA ....................................................................................................................... body surface area 
BMT .............................................................................................................bone marrow transplant 
CBC................................................................................................................. complete blood count 
CCR5................................................................................................. c-c chemokine receptor type 5 
CDUS .................................................................................................. Clinical Data Update System 
CEF ........................................................................................................ CMV-EBV-influenza virus 
CFR ...................................................................................................... Code of Federal Regulations 
CFSE ..................................................................................... carboxyfluorescein succinimidyl ester 
CFU ....................................................................................................................colony forming unit 
CI......................................................................................................................... confidence interval 
cm ...................................................................................................................................... centimeter 
CMP ................................................................................................ comprehensive metabolic panel 
CMV ....................................................................................................................... cytomegalovirus 
CNS ............................................................................................................... central nervous system 
COA ................................................................................................................ certificate of analysis 
CR ........................................................................................................................ complete response 
CRF ......................................................................................................................... case report form 
CT ......................................................................................................... computed tomography scan 
CTA............................................................................................................... clinical trial agreement 
CTCAE ............................................ Common Terminology Criteria for Adverse Event Reporting 
CTEP ....................................................................................... Cancer Therapy Evaluation Program 
CTEP-AERS ..................................................................... CTEP Adverse Event Reporting System 
CTL ............................................................................................................. cytotoxic T lymphocyte 
CTMS ......................................................................................... Clinical Trials Monitoring Service 

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CXCR4 .................................................................................................... chemokine receptor type 4 
CXR ................................................................................................................................ chest X-ray 
DARF .............................................................................................. drug accountability record form 
DC ............................................................................................................................... dendritic cells 
DHHS ............................................................................ Department of Health and Human Services 
DLCO ............................................................... diffusing capacity of the lung for carbon monoxide 
DLT ................................................................................................................. dose-limiting toxicity 
DMSO ................................................................................................................. dimethyl sulfoxide 
DNA ............................................................................................................... deoxyribonucleic acid 
DSMB ........................................................................................... data and safety monitoring board 
EBMT ......................................................................................... European bone marrow transplant 
EBV..................................................................................................................... Epstein-Barr Virus 
ECOG .................................................................................... Eastern Cooperative Oncology Group 
EDTA .............................................................................................. ethylenediaminetetraacetic acid 
EFV ..................................................................................................................................... efavirenz 
EGFP ......................................................................................... enhanced green fluorescent protein 
ELISA .................................................................................... enzyme linked immunosorbent assay 
EU/mL.................................................................................................. endotoxin units per milliliter 
FACS........................................................................................... fluorescence activated cell sorting 
FACT ........................................................... Foundation for the Accreditation of Cellular Therapy 
FBS ...................................................................................................................... fetal bovine serum 
FDA.................................................................................................. Food and Drug Administration 
FDG-PET ......................................................... fluorodeoxyglucose-positron emission tomography 
FEV-1 ........................................................................................................ forced expiratory volume 
ft .................................................................................................................................... flow through 
FVC ................................................................................................................... forced vital capacity 
g/dl .......................................................................................................................grams per deciliter 
GALT ................................................................................................gut associated lymphoid tissue 
GAPDH ................................................................................................ glyceraldehyde-6-phosphate 
GCP ................................................................................................................. good clinical practice 
GI .............................................................................................................................. gastrointestinal 
GMP ..................................................................................................... good manufacturing practice 
HAV ................................................................................................................................. hepatitis A 
Hb ................................................................................................................................... hemoglobin 
HIV ................................................................................................. human immunodeficiency virus 
HCT..................................................................................................... hematopoietic cell transplant 
HIPAA ........................................................... Health Insurance Portability and Accountability Act 
HLA .......................................................................................................... human leukocyte antigen 
HPC ................................................................................................... hematopoietic progenitor cells 
HSC ............................................................................................................ hematopoietic stem cells 

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HSCT .................................................................................. hematopoietic stem cell transplantation 
HSV.................................................................................................................. herpes simplex virus 
HTLV ............................................................................................ human T cell lymphotropic virus 
ICH ............................................................................... International Conference on Harmonization 
IDB ....................................................................................................... Investigational Drug Branch 
IFN ..................................................................................................................................... interferon 
IHC ................................................................................................................ immunohistochemistry 
IL ...................................................................................................................................... interleukin 
IND ......................................................................................... investigational new drug application 
INR ..................................................................................................... international normalized ratio 
IPI ...................................................................................................... International Prognostic Index 
IRB ........................................................................................................... institutional review board 
kg......................................................................................................................................... kilogram 
LAL ........................................................................................................... limulus amebocyte lysate 
LN .............................................................................................................................. liquid nitrogen 
LAM-PCR ..................................................................................linear amplification-mediated PCR 
LDH ............................................................................................................... lactate dehydrogenase 
LTR ................................................................................................................... long terminal repeat 
LVEF............................................................................................... left ventricular ejection fraction 
MCP .................................................................................................monocyte chemotactic proteins 
mg ...................................................................................................................................... milligram 
MIP ............................................................................................. macrophage inflammatory protein 
µL ....................................................................................................................................... microliter 
MDS ........................................................................................................ myelodysplastic syndrome 
mL ........................................................................................................................................milliliter 
mm3 ......................................................................................................................... cubic millimeter 
MOI ............................................................................................................ multiplicity of infections 
MOP ................................................................................................................ manual of procedures 
mRNA ................................................................................................ messenger ribose nucleic acid 
MUGA ............................................................................................. multiple gated acquisition scan 
NCI ............................................................................................................ National Cancer Institute 
NCT.............................................................................................National Clinical Trials [Registry] 
ng........................................................................................................................................nanogram 
NIH………………………………………………………………. ..... National Institutes of Health 
NHL………………………………………………………………… ..... Non-Hodgkin Lymphoma 
NK cells ................................................................................................................ natural killer cells 
NRG mouse ...................................................... NOD-Rag1null IL2rgnull, NOD rag gamma mouse 
NSC ............................................................................................................. National Service Center 
NT .....................................................................................................................non‐transduced cells 
ODMC............................................................................. Operations and Data Management Center 

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OHAM ................................................................................... Office of HIV and AIDS Malignancy 
OI ................................................................................................................. opportunistic infections 
OS .............................................................................................................................overall survival 
PB ............................................................................................................................ peripheral blood 
PBMC ....................................................................................... peripheral blood mononuclear cells 
PCP ............................................................................................... Pneumocystis carinii pneumonia 
PCR ..........................................................................................................polymerase chain reaction 
PDL .............................................................................................................................. poly-d-lysine 
PET ................................................................................................... positron emission tomography 
PFS ............................................................................................................ progression-free survival 
PFT .............................................................................................................. pulmonary function test 
PHI ....................................................................................................... protected health information 
PI ..................................................................................................................... principal investigator 
PIO ........................................................................................................ Protocol Information Office 
PT .......................................................................................................................... prothrombin time 
PTT ........................................................................................................ partial thromboplastin time 
QC .............................................................................................................................. quality control 
QA .......................................................................................................................... quality assurance 
qPCR .................................................................................... quantitative polymerase chain reaction 
RAC ................................................................................. Recombinant DNA Advisory Committee 
RCL ................................................................................................. replication competent lentivirus 
RNA .....................................................................................................................ribose nucleic acid 
RPR .................................................................................................................... rapid plasma reagin 
RT-PCR.................................................................. reverse transcription polymerase chain reaction 
SAE ................................................................................................................. serious adverse event 
SOC ......................................................................................................................system organ class 
SD ........................................................................................................................ standard deviation 
SEB .....................................................................................................staphylococcal enterotoxin-B 
SGOT ............................................................................... serum glutamic oxaloacetic transaminase 
SGPT ..................................................................................... serum glutamic-pyruvic transaminase 
SMART ......................................................... Strategies for Management of Antiretroviral Therapy 
SOP ..................................................................................................... standard operating procedure 
shRNA............................................................................................ short hairpin ribose nucleic acid 
STI............................................................................ structured (or strategic) treatment interruption 
TAR.....................................................................................................tat activation response region 
TAX (1TAX) ................................................................................................. transactivating protein 
Tat ..................................................................................................... trans-activator of transcription 
TNFα ...................................................................................................... tumor necrosis factor-alpha 
TRIM5α .......................................................................... tripartite motif containing protein 5 alpha 
TTR ............................................................................................................... time to tumor response 

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Treg ....................................................................................................................... regulatory T cells 
VDRL .................................................................................. Venereal Disease Research Laboratory 
VZV ................................................................................................................. varicella zoster virus 
ULN ................................................................................................................ upper limit of normal 
USP ...................................................................................................... United States Pharmacopeia 
ZDV ................................................................................................................................. zidovudine 

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1.0 
OBJECTIVES 
1.1 
Hypothesis 
Our hypothesis states that anti-HIV gene expressing stem cells can safely reconstitute the 
hematopoietic system of the recipient and can produce a pool of mature myeloid and 
lymphoid cells that harbor anti-HIV genes capable of resisting HIV-1. As a secondary 
objective, we will interrogate two physical compartments of immune system, (i.e., 
peripheral blood cells and gut mucosa) for the presence, quantity, and duration of gene 
modified HIV-1-resistant immune cells. In addition, we will determine the integration sites 
of vector sequences in circulating cells and monitor for any clonal expansion of 
hematopoietic cells. 
We also hypothesize that transduced HIV-resistant immune cells will have a selective 
survival advantage as compared to non-transduced cells in the face of a viral load, as 
demonstrated by our preclinical in vivo experiments, and suggested by previous clinical 
trials using comparable stem cell gene therapy approaches. As an exploratory endpoint, we 
therefore want to test the efficacy of our stem cell gene therapy for HIV-1 in the context of 
structured HIV-1 treatment interruptions. 
1.2 
Primary Objective 
The primary endpoint of the study is safety, defined as timely engraftment (the collective 
establishment of a persistent absolute neutrophil count of at least 500 cells/mm3 and platelet 
count of 20,000 cells/mm3 without transfusion for 3 consecutive measurements of 
laboratory values obtained on different days) by one month post-transplant, in the absence 
of any grade 3 and 4 non-hematopoietic organ toxicity that can be attributed (possibly, 
probably, or definitely) to lentiviral transduced stem cell transplant, excluding alopecia, or 
any clonal expansion and excluding expected toxicities that are associated with the pre-
transplant conditioning regimen (see Appendix X for full list). 
1.3 
Secondary Objectives 
1.3.1 Major secondary objectives 
1.3.1.1 To determine efficacy of the candidate product, defined as establishment 
of > 5% mononuclear blood cells expressing anti-HIV genes in the 
peripheral blood at 3 months post-transplant. 
1.3.1.2. To determine the presence, quantity, and duration of gene modified HIV-
1 resistant peripheral blood cells and gut mucosal immune cells. 
1.3.1.3 To study the integration sites of vector sequences in circulating cells. 
1.3.2 Minor secondary objectives 
1.3.2.1 To study progression-free survival. 
1.3.2.2 To study overall survival. 
1.3.2.3 To study complete response rate and duration. 
1.3.2.4 To study partial response rate and duration. 

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1.3.2.5 To study time to neutrophil engraftment (first measurement of 3 
consecutive laboratory values on different days) of ANC >/= 500 
cells/mm3). 
1.3.2.6 To study time to platelet engraftment (first measurement of 3 consecutive 
measurements laboratory values obtained on different days) of platelets > 
/=20,000 cells/mm3 without platelet transfusions 7 days prior). 
1.3.2.7 To study hematologic function at Day 100 (ANC > 1500, Hb > 10g/dl 
without transfusion and platelets > 100,000) 
1.3.2.8 To study CD4 recovery at the conclusion of the trial. 
1.3.2.9 To study safety in terms of toxicities, infections, transfusions, and 
infusion-related reactions. 
1.3.2.10 To study HIV-1 viral load over time. 
1.3.2.11 To study persistence of vector-transduced cells over time. 
1.4 
Exploratory Objectives 
1.4.1 To evaluate the presence and the magnitude of expansion of HIV-1 resistant 
immune cells in the peripheral blood and gut mucosa of transplanted participants, 
subsequent to withholding ART. 

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2.0 
BACKGROUND 
2.1 
Study Disease 
HIV-1/AIDS continues to be a major public health problem worldwide. Based on recent 
estimations, there are approximately 33.3 million people living with HIV-1/AIDS with 2.6 
million new HIV-1 infections per year, and 1.8 million annual deaths due to AIDS. It is 
estimated that more than one million people are living with HIV-1 in the USA and that 
more than half a million have died after developing AIDS. Despite the advances in anti-
retroviral therapy (ART), eradication of HIV-1 from infected patients is still an elusive 
goal. Lifelong therapy is associated with potential long-term toxicity, adherence problems, 
and development of drug resistance (1-6). Interruption of ART therapy at any stage will 
result in a rebound in HIV-1 replication and rapid deterioration of a patient’s immune 
system. Even new drug classes such as integrase inhibitors and CCR5 antagonists, despite 
their promise, have failed to eradicate HIV-1 (5-6, 9-10). Patients who are successfully 
treated with ART often do not achieve full recovery of immune responses, may develop 
HIV-1-related cancers, and exhibit increased levels of immune activation (7-8). Such a 
chronic inflammatory status can result in immune exhaustion (and subsequent infections) 
and may precipitate multiple clinical complications such as cardiovascular and 
cerebrovascular disease, diabetes, chronic kidney disease, osteoporosis, and cancer, and 
collectively, accelerated aging. While the cause of this chronic inflammatory status is not 
completely clear, evidence suggests the role of innate immunity, ART interruption, non-
compliance, effect of HIV-1 on endothelial cells, interruptions in the coagulation system, 
incomplete immune recovery, microbial translocation, co-infections, residual viremia, etc. 
2.1.1 Gene therapy for HIV-1 patients with lymphoma 
Therefore, the idea of a cell-based gene therapy approach to cure HIV-1 would be 
especially compelling (11). Gene therapy has the potential to not only control viral 
replication and prevent CD4+ T cell depletion, but also provide a pool of immune 
cells with sustained intrinsic protection. HIV-1 gene therapy is not a new idea, and 
currently, gene therapy clinical trials are ongoing (12-14). However, there are major 
differences in the types of genes utilized as well as the cells targeted, which may 
be either peripheral blood T cells or hematopoietic stem cells (HSC). 
Gene therapy targets can be classified according to the same concept as applied to 
other anti-HIV-1 therapeutic strategies: Class I therapeutics inhibit HIV-1 entry and 
replication; Class II therapeutics inhibit viral regulatory and structural protein 
expression; and Class III therapeutics inhibit viral assembly and release. Most of 
the current anti-HIV gene therapy approaches (including our proposed protocol) 
focus on Class II and especially Class I gene targets that have been shown to 
perform as best available antivirals by far. Recently, a similar comparison has 
resulted in a similar conclusion in in vivo studies (15). 
While T cells are easy to obtain, their long-term engraftment is limited despite 
current lympho-depletion approaches. In addition, questions have recently been 
raised on the expansion of T cells in response to a high viral load (16-17). 
Furthermore, T cell gene therapy, despite its relative ease, cannot alleviate the main 
concerns about HIV-1 therapy such as the presence of low-level viral replication 
caused by dormant HIV-1 reservoirs. Therefore, the benefits of current T cell gene 

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therapy protocols with the need for multiple, life long, continued cell infusions will 
not exceed the advantages of current ART therapy. Targeting human stem cells, as 
described here, on the other hand, can address many of these concerns. Such a stem 
cell therapy would require only a single treatment that would replace, in the long 
run; the reservoirs of active and latent HIV-1 with anti-HIV gene modified cells 
resistant to infection, thus alleviating the need for long-term ART and would 
ultimately cure these patients. In addition, unlike challenges in in vivo expansions 
of gene therapy modified T cells (as described above), anti-HIV gene modified stem 
cell derived HIV-1 target cells have shown that they do respond to an HIV-1 viral 
load with significant expansion of T cells (15,18). The best evidence supporting the 
feasibility of this comes from a case report of an HIV-1 patient transplanted with 
stem cells from a host homozygous for the CCR5 delta-32 mutation. The transplant 
resulted in reduction of HIV-1 viral load over time and the replacement of 
microglial and Kupffer cells, common reservoirs of latent HIV-1, by donor-derived 
HIV-1 resistant cells (18). This strongly suggests that the patient is now cured of 
HIV-1. Previous trials of HIV-1 gene therapy into HSCs have been plagued by low 
transduction efficiencies and loss/silencing of the anti-HIV genes over time (14, 
18). Furthermore, single anti-HIV genes harbor the risk of developing resistant 
HIV-1 mutants (12). Finally, in some of these trials, the use of retroviral vectors 
(versus lentiviral vectors in our proposal) increases the risk of oncogenesis (13). 
Lessons learned from two recent computational models of HIV-1 replication and 
development of resistance to gene therapy regimens support our notion that the 
combinatorial anti-HIV gene therapy protocols and higher numbers of anti-HIV 
gene modified immune cells are critical factors in the success of gene therapy 
protocols for HIV-1 (19-20). Furthermore, they showed that when 1-2 anti-HIV 
targets are used, the success will be significantly dependent on the potency of the 
approach, while multiple combinatorial gene therapy approaches can eliminate 
such a requirement. 
2.1.2 Bone marrow transplant for HIV-1 related lymphomas 
The prognosis for patients with refractory and relapsed non-Hodgkin’s lymphoma 
(NHL) is poor with overall survival rates of less than 20% for patients treated with 
non-transplant salvage therapies. Based on these randomized trials, high-dose 
therapy with hematopoietic stem cell transplantation (HSCT) has been established 
as the standard of care for patients with relapsed but chemotherapy-sensitive NHL 
(21-23). 
This initial transplant experience was marked by what appeared to be an increased 
risk of opportunistic infection (OI). Since the availability of ART, however, the 
transplant experience has evolved considerably, and several groups have now 
demonstrated the feasibility of such an approach. 
In a trial published by City of Hope, patients with induction-failure NHL, 
chemotherapy-sensitive disease, and International Prognostic Index (IPI) high-risk 
disease were eligible for treatment. Twenty participants were enrolled in the trial. 
All of the participants successfully achieved adequate autologous stem cell 
collections. Participants were treated with either high-dose chemotherapy or 
intensive chemo-radiotherapy. At a median follow-up of 31.8 months, 17 of 20 

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participants were alive and free of evidence of disease. Treatment-related 
complications were comparable to those seen in the non-HIV-1 infected patient 
population (24). 
In a similar trial by the AIDS Malignancy Consortium (AMC), 27 participants with 
either HIV-1-related NHL or Hodgkin’s lymphoma were enrolled and 20 of them 
subsequently underwent autologous hematopoietic cell transplantation (HCT) with 
a dose-reduced busulfan and cyclophosphamide regimen. At a median follow-up 
time of 23 weeks, 10 participants were alive and free of disease (25). In a recent 
trial by the AMC, 43 participants with HIV-1 CCSR aggressive NHL or Hodgkin’s 
lymphoma were enrolled and 40 underwent autologous HCT using BEAM regimen 
(35). In this trial, 39 participants were alive 100 days post-HCT and 36 of the 39 of 
these participants were free of disease; and 24 of 32 participants achieved recovery 
of hematological function by 24 months post-HCT. Other groups have also 
demonstrated the feasibility of performing autologous HCT in this patient 
population (26-28). 
More recently, the European Bone Marrow Transplant (EBMT) Lymphoma 
Working Party, in a much bigger trial, published treatment outcomes for 68 
participants with HIV-1-related lymphoma (including 18 participants with Hodgkin 
lymphoma) that underwent autologous HCT at 20 different institutions. Sixty-five 
participants were treated with the carmustine, etoposide, cytarabine, and melphalan 
(BEAM) regimen and the remainder were treated with radiation therapy-based 
regimens. At a median follow-up of 32 months, the 3-year estimated progression-
free and overall survivals for the group were 56% and 61% respectively, with low 
overall cumulative incidence of non-relapse mortality of 4.4% and 7.5% at three 
and 12 months respectively. Relapse or progression was identified in 20 
participants after a median time of 4.1 months from autologous HCT with the 
predicted cumulative incidence of relapse of 30% at 24 months (29). 
A subsequent EBMT study for patients with Hodgkin's disease and non-Hodgkin's 
lymphoma used a comparison matched control design stratified by HIV-1 status. 
The results of this comparative study revealed that HIV-1 patients undergoing 
autologous HCT for lymphoma showed similar relapse rates, progression free 
survival, and overall survival (30). 
Based on these data, the practice of autologous HSCT is routinely used in many 
centers in the country as part of the treatment of relapsed/refractory (but still 
chemo-sensitive) NHLs. 
2.2 
Study Agent: Autologous CD34+ Cells Transduced with a Pre-Selective Marker and 
a Triple Combination of Anti-HIV Genes 
Our proposed novel anti-HIV therapeutic candidate combines multiple anti-HIV genes into 
a single lentiviral vector that is designed to block HIV-1 infection at different stages of the 
HIV-1 life cycle (entry, uncoating, and viral transcription). This vector also contains a pre-
selective 
molecule, 
which 
marks 
transduced 
cells 
and 
allows 
for 
the 
purification/enrichment of the transduced population away from untransduced cells. This 
approach not only provides strong pre-integration inhibition of HIV-1 infection, but also 
decreases the generation of viral escape mutants. The major anti-HIV components of our 

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vector are a CCR5 shRNA, a chimeric TRIM5α, and a TAR decoy. Due to the natural HIV-
1-resistant phenotype conferred by the delta-32 bp CCR5 mutation found in a small percent 
of humans, CCR5 knockdown utilizing shRNAs is an excellent approach for HIV-1 gene 
therapy. However, utilizing only a single CCR5 shRNA will not afford protection against 
CXCR4 or other tropisms of HIV-1. Therefore, we have incorporated two other highly 
potent anti-HIV genes into our lentiviral vector, which protect against all tropisms of HIV-
1. The second molecule, a human/rhesus macaque chimeric TRIM5α molecule, acts at the 
post-entry/pre-integration stage of the HIV-1 life cycle. Old world monkey TRIM5α 
isoforms, which include the rhesus macaque isoform, are strong inhibitors of HIV-1 
infection, however, human TRIM5α does not inhibit HIV-1. Therefore, we have utilized a 
chimeric isoform of human TRIM5α where we have incorporated the critical amino acids 
of the rhesus macaque isoform necessary to inhibit HIV-1 infection. This chimeric human 
TRIM5α now only contains 13 amino acids from the rhesus macaque isoform; however, it 
is now able to potently inhibit HIV-1 infection. The third molecule, a TAR decoy, acts at 
the pre-transcription stage of the HIV-1 life cycle and scavenges the HIV-1 Tat protein 
away from its normal function in HIV-1 proviral transcription. This removal of Tat from 
the transcriptional complex disrupts HIV-1 proviral transcription and does not allow new 
viral mRNA, and therefore, new virus particles to form. The pre-selective molecule, a 
truncated and mutated form of human CD25, is used to mark the cell surface of transduced 
cells, so they can be purified away from untransduced cells with CD25 immunomagnetic 
separation. This modified human CD25 molecule has been fully truncated of its 
intracellular amino acids to avoid any possible intracellular signaling. Two-point mutations 
have also been made to inhibit any IL2 binding, and also to inhibit the incorporation of this 
molecule into the mature IL2 receptor complex. 
The three anti-HIV molecules expressed together in a single lentiviral vector have 
demonstrated complete protection from productive HIV-1 infection and prevented 
integration of multiple strains of HIV-1-1 (including macrophage and T cell tropic HIV-1 
strains) in both transduced cultured cells and transduced CD34+ HSCs derived into 
macrophages in vitro (17). In vivo safety and efficacy studies in a humanized mouse model 
for HIV-1 showed excellent results including maintenance of normal total human CD4+ 
cell levels with a pre-integration block of HIV-1 infection (18). Our preclinical in-vitro and 
in-vivo safety and efficacy data are mature enough to start the regulatory approval process 
for a stem cell gene therapy clinical trial. The successful development of a multi-gene pre-
selective anti-HIV vector, excellent safety and efficacy results in both in vitro and in vivo 
testing of the vector transduced cells even for resistant strains of HIV-1, production of high 
titer GMP-grade lentiviral vector, and superior transduction efficiencies into HSCs are 
highlights of our proposed therapeutic candidate, which make it unique and more 
promising than other currently available gene therapy for HIV-1 approaches. 
Extensive preclinical data supporting this study is included in our IND document and the 
investigator’s brochure. 
2.3 
Study Design and Rationale 
This is an open-label, single-arm, multi institution, Phase I study of maximum of 18 
participants. The study is designed to fulfill the mission of a Phase I study as “a trial to 
study the safety, and determine the dose limiting toxicity (DLT) to a new treatment.” All 
participants will be treated with anti-HIV gene transduced CD34+ cells. 

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Table 2-A: Study Cohorts 
Study Cohorts 
Ratio of Transduced versus Untransduced Stem 
Cells 
1 
1:1 
2 
5:1 
3 
1:0 
3+* 
1:0 but with a higher transduced cell count only in 
case of DLT due to insufficient engraftment in 
cohort 3 
 
*. See section 2.3.1 
Three participants will be treated at each cohort level, with the option of 3-6 additional 
participants in the highest dose level cohort. Participants will be recruited to the groups in 
order that they were screened. Participants will be monitored on a weekly basis for the first 
4 weeks and then every other week thereafter. A cohort will be expanded from 3 to 6 
participants if at least one participant in the cohort experiences engraftment failure or grade 
3 or 4 non-hematopoietic toxicity, excluding alopecia. The enrollment of the participants 
within the cohorts will be staggered so each participant needs to show at least early signs 
of engraftment for neutrophils (ANC) >/= 500 cells/mm3 and platelets >/= 20,000 
cells/mm3 before the next participant is enrolled. The enrollment of participants between 
the cohorts will be staggered, so every participant in the same cohort needs to have at least 
30 days of follow up from the time of stem cell transplant for the AMC Data and Safety 
Monitoring Board (DSMB) to review the side effects and before we can proceed to the next 
cohort. 
Achievement of high levels of engraftment with anti-HIV genes will be needed to secure a 
reasonable anti-HIV response. Therefore, in the third cohort, the participants will receive 
only the transduced product. The un-manipulated portion of product will remain frozen as 
a backup. In this cohort, the backup product will only be given to the participant if delayed 
engraftment occurs, especially in the setting of ongoing complications such as bleeding or 
infection. 
For safety reasons and based on FDA request, participants enrolled in the first cohort of 
the study will receive both transduced and un-transduced product at equal number. This is 
to prevent a prolonged cytopenia in case the transduced cells fail to engraft and recover the 
peripheral blood in a timely manner. In the second cohort, once the safety of the product is 
established, we will increase the ratio of the transduced cells to untransduced cells, with 
the goal to give 5 times (or higher) more transduced cells. 
Participants with relapsed/refractory HIV-1 related lymphoma in need of autologous bone 
marrow transplantation will be enrolled. Upon documentation of disease response, their 
peripheral stem cells will be collected using standard mobilization approaches. Because of 
the unpredictability of the number of cells collected for each patient and the significant 

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variability of the number of cells obtained after transduction and selection process, a set of 
criteria has been created to explain the minimum and the range of cells infused to each 
patient (Table 4-A).  
• A target cell number of 10 x 106 CD34+ cells/kg will be collected, with a minimum of 
5 x 106 CD34+ cells/kg only if we cannot collect enough cells after multiple attempts.  
• A minimum of 1.5 x 106 of unmanipulated CD34+ HSC/kg will be collected upfront 
and will be frozen in a minimum of 3 cryo bags and stored at each institution’s local 
stem / progenitor cell facility per site SOPs. Even though the minimum amount of 
unmanipulated cells infused in the first two cohorts are 1.0 x 106 CD34+ cells/kg, 
additional 0.5 x 106 CD34+ cells/kg of recipient weight will be collected to ensure a 
safe transplant in the unlikely situation that the transduced product is not usable. 
• The minimum total number of CD34+ cells (manipulated + unmanipulated) infused to 
the patient is 2 x 106 CD34+ cells/kg of recipient weight independent of the cohort to 
assure the safety of transplant. 
• In cohorts 1 and 2, a minimum of 1 x 106 of unmanipulated CD34+ HSC/kg will be 
infused, and the rest of the cells will be stored as back-up. Additional CD34 cells from 
the transduced product given based on each cohort is expected. In cohort 3, all of the 
untransduced cells will be stored and used only in the case of delayed engraftment. 
• Additional cells can be collected and stored after the target number for CD34 (10 x 106 
CD34+ cells/kg) is achieved based on the discretion of participant’s transplant 
physician in consultation with the study principal investigator. These cells can be given 
to the patient, but the ratio of total manipulated to unmanipulated cells needs to follow 
the guidelines in Table 4-A. 
• In case of any failure in the transduction process, all of the unmanipulated cells will be 
infused to the patient. 
Apheresis product, from the first day of collection, will be split into two parts: One part 
(1.5 x 106 of CD34+ HSC/kg) will be cryopreserved (unmanipulated), and the second part, 
if additional cells are available, will be sent immediately to the GMP facility for gene 
transduction. We expect that we will need several days of collection for each participant. 
Once 1.5 x 106 CD34+ cells/kg is collected, then no more products will be divided for back 
up, and for the rest of the collections, the entire product from subsequent collections will 
be used for gene transduction. After transduction, this product will be frozen. Release tests 
will be performed on aliquots (see Section 6.1.11). 
2.3.1 Three+ (3+) expansion cohort (protocol version 8) 
The ultimate goal of the study is to replace the full hematopoietic system of the 
recipient with a gene modified anti-HIV hematopoietic system. The design of this 
clinical trial is different from other dose escalating clinical trials as we are not 
testing an increasing dose of our product and therefore routine phase I, 3+3 design 
is not applicable in this trial. The purified manipulated cells may not have the same 
robust engrafting capabilities as unmanipulated HSCs and, therefore, if in the third 
cohort 2 patients develop DLT due to insufficient engraftment, an additional 3 
patients will be treated with a higher dose of gene-modified CD34 cells (3.0 x106 

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CD34 cells/kg of recipient body weight) to assess whether a higher dose of cells 
will improve engraftment. Any additional engraftment issue on this expanded 
cohort deemed to be related to product will result in stopping of the trial.  
2.4 
ART Withdrawal / Analytic Treatment Interruption (ATI) 
The presence of sizable untransduced CD34 cells in the infused product will result in a 
population of hematopoietic cells in the participant after transplant that will remain 
sensitive to HIV-1 and still can drive a significant viral load with its associated 
complications. In the City of Hope clinical trial, significant expansion of HIV-1 resistant 
hematopoietic clone was observed when the participant was off his ART. Based on the 
above rationale and supporting observation, we are proposing to withdraw ART after 6 
months post-transplant and when the CD4 counts reaches ≥ 300 cells/mm3. 
At six months post-transplant, or later, ART will be voluntarily withheld for participants 
with a CD4 count of 300 or higher with no detectable viral load (by single copy PCR)   until 
one or more of the criterion in Section 2.4.1 prompting resumption of treatment. We do 
understand that the time to achieve a T-cell count of 300 may take longer than six months 
for HIV-1 infected participants post-transplant. Therefore, for participants in which the 
CD4 T-cell count has not risen to ≥ 300 cells/mm3 at the time of the planned ART 
interruption, ART will continue until the T-cell count has risen to ≥ 300 cells/mm3. 
In addition, participants will be tested for the virus that causes COVID-19 prior to 
ART interruption and if positive by PCR, will remain on ART until they recover, and the 
study team approves them to proceed with ATI. Participants on ATI will be offered 
COVID testing if symptoms associated with COVID-19 develop and will restart ART 
if they test positive by PCR. 
The duration for ART withdrawal has been selected as it is an appropriate duration for 
assessment of any selective survival advantage of anti-HIV gene expressing immune cells, 
and as this is an accepted and safe duration for possible viremia rebound in previously ART 
controlled HIV-1 participants. Interruption of ART in previous clinical trials has led to a 
consistent re-emergence of viremia in approximately 2 weeks, with re-establishing a set 
point in 4-6 weeks (33). This approach has been proven to be safe for participants. 
Designed as an exploratory endpoint in our study, this duration of treatment interruption 
will allow determining whether participants’ viral loads return to their pre-ART set points. 
Multiple clinical trials testing therapeutic vaccination for HIV-1 as a strategy for modifying 
the role of ART for controlling HIV-1 disease have used a similar interruption (formerly 
known as Structured, or Strategic, Treatment Interruption (STI)) for 12 weeks due to its 
safety profile. 
2.4.1 ART Resumption Criteria 
Several safety parameters will be monitored and will be used to restart therapy 
earlier, if needed. These include: 
1. CD 4+ T cell criteria: A decline of CD4+ count to less than 200 cells/mm3 in 2 
consecutive measurements laboratory values obtained on different days, 2 
weeks apart, or more than 50% drop in absolute CD4 count. 
2. Viral load criteria: ART will be initiated for any one of the following outcomes:  

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• viremia > 50,000 copies RNA/mL for four weeks 
• viremia > 10,000 copies RNA/mL for 6 weeks 
• viremia > 2000 copies RNA/ml for 12 weeks 
• viremia > 400 copies/RNA for 24 weeks  
Operationally, this will allow for an acute viremic state that will need to resolve 
within 12 weeks, with high-level viremia only being allowed for a brief period. 
After the spike, the viral load should fall as the numbers of susceptible target 
cells are deleted, leaving just the transformed cells. Post-treatment control 
(defined as viremia <400 copies RNA/mL) should be achieved prior to Week 
24 post-virus rebound.  
For any initial viral load greater than 400 copies/mL during weekly monitoring, 
participants must return within 3 business days for a repeat test. 
3. Clinical criteria: ART will also be re-initiated for any symptoms suggestive of 
a severe acute retroviral syndrome, particularly signs of altered mental status or 
viral meningitis (severe headache, neck stiffness), but including unexplained 
rash, lymphadenopathy, fevers, and occurrence of additional opportunistic 
infection (OI). ART will also be re-initiated if a participant tests positive for 
COVID-19 by PCR. 
4. If, in the opinion of the subject or the primary care physician, restart is desired 
for other reasons. It will be requested, but not required, to discuss this decision 
with the PI and research team beforehand, if feasible. 
2.4.2 ART Withdrawal Monitoring 
Participants will be closely monitored weekly when they are off ART, and 
treatment can be restarted at the discretion of the participant, their HIV-1 physician, 
or the site PI. All patients on ART withdrawal will receive anti-OI prophylactic 
antibiotics. 
Despite a long history with the application of ATI for testing the therapeutic impact 
of candidate immunomodulatory treatment strategies, some studies have indicated 
that treatment interruptions, when devised with a medication sparing intent and 
without close clinical observation, may carry certain risks and are not always 
successful long-term (31). The host of published data on ATIs has recently been 
summarized in a Cochrane report (32). An important conclusion of this report is 
that ATIs continue to have applicability to the testing of new therapeutic concepts 
or drugs in well-characterized participant populations in the context of well-
controlled clinical settings. However, ATIs without the addition of any other 
treatment modality, such as immunotherapy or with an ART sparing interval, are 
no longer supported. 
Based on recent trial results, the U.S. Department of Health and Human Services 
(DHHS) HIV-1 Treatment Guidelines (2015) state that, apart from unplanned ATI 
or short-term ATI due to drug toxicities, long-term ATIs are not recommended (34). 
The guidelines provide important guidance, advising that if therapy has to be 
discontinued, participants should be counseled about the need for close clinical and 
laboratory monitoring. They should also be aware of the risks of viral rebound, 

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acute retroviral syndrome, increased risk for HIV-1 transmission, decline of CD4+ 
T-cell count, HIV-1 disease progression or death, development of minor HIV-1-
associated manifestations such as oral thrush, development of serious non-AIDS 
complications, development of drug resistance, and the need for chemoprophylaxis 
against opportunistic infections depending on the CD4+ T-cell count. Treatment 
interruptions possibly result in rapid reductions in CD4+ T-cell counts. The risks 
of repeated treatment interruptions are not clearly defined in the guidelines; 
however, data from the NIH’s Strategies for Management of Antiretroviral Therapy 
(SMART) study suggest that cycles of treatment interruption at low CD4+ T cell 
counts could result in increased risk of death or disease progression. These risks 
continue to exist for short, prolonged, and repeated cycles of ATI. 
Participants in the current study will be monitored on a weekly basis during the 12-
week ATI and beyond. Participants will also be counseled with regard to the risks 
(outlined in the HIV-1 Treatment Guidelines) prior to ATI and weekly during ATI. 
During ATI, participants will be tested for COVID-19 if they experience symptoms 
consistent with COVID-19 and will restart ART COVID-19 positive by PCR. In 
addition, drug resistance data will be carefully reviewed by the investigator prior to 
allowing a participant to enter a second treatment interruption. Complications, such 
as acute retroviral syndrome when suspending ART or immune reconstitution 
syndrome when restarting ART, are more common in the setting of their de novo 
occurrence (i.e., when HIV-1 is first contracted and when ART is first initiated in 
the ART naïve patient). Even in these circumstances, their occurrence is rarely 
clinically significant with the potential exception of cryptococcal meningitis and 
reactivation mycobacterium tuberculosis disease in the case of immune 
reconstitution inflammatory syndrome. Irrespective, participants will be alerted to 
the potential for each of these respective events and instructed on alerting the study 
team of any sentinel symptoms so that clinical observation can be intensified. 
Additionally, participants with a history of mycobacterium tuberculosis latent or 
remote infection, cryptococcal meningitis, or clinically significant cardiovascular 
disease will be excluded from participating in the study without careful review by 
the protocol leadership and a more intensive discussion with the participant about 
the potential risks of reactivation or clinical manifestations of these respective 
conditions. 
Based on this background, treatment interruption following engraftment will be 
delayed until CD4+ T-cell counts recover to ≥ 300 cells/mm3 to provide a buffer 
above the 200 cell/mm3 threshold for restarting ART prior to the end of the pre-
specified 12-week period. Any clinically significant deterioration observed or 
reported during the treatment interruption including, but not limited to, signs of 
altered mental status or viral meningitis (severe headache, neck stiffness), the 
emergence of fever to greater than 38.5oC, severe lethargy, or signs/symptoms of a 
possible OI, any OI including esophageal thrush or pneumonia, or any deterioration 
that in the opinion of the study team or the participant’s primary provider would 
place the participant at risk for significant morbidity will be monitored and will be 
used to restart therapy earlier, if needed. While understandably non-specific, the 
safety of the participant will be the primary consideration attending any clinical 
management criteria during the course of this clinical trial. Consideration for 

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reinstating an ATI once interrupted due to the side effects will only be considered 
after consultation with the FDA. 
The rationale for establishing a threshold for restarting ART prematurely if the 
CD4+ T-cell count goes below 200 cells/mm3 is based on the expectation that 
selective expansion of transformed cells is likely to occur in the setting of loss of 
non-transformed cells and an overall decline in total CD4 count. The anticipated 
risk of this strategy is expected to be mitigated by the limited duration of a low CD4 
count accompanying high viremia. Clinical experience has demonstrated that the 
risk of OI occurrence increases over time. As an example, in the experience of the 
clinical investigators, unintentional interruption of Pneumocystis carinii 
pneumonia (PCP) prophylaxis is rarely associated with reactivation PCP sooner 
than several months. Additionally, while risk of OI occurrence is tightly associated 
with decline in CD4+ T-cell count, the level of viremia interacts with the rate of 
risk as well. Reinstitution of potent ART, which rapidly suppresses viremia within 
days to weeks in the setting of close clinical monitoring, will minimize the risk to 
the participant of significant clinical events. While an independent association of 
the level of viremia and risk of OI or other morbidities has not been established, the 
team will err on the side of caution and use the escalating viral load criteria 
described above as a threshold. This will be implemented more to be confident of 
the ability to control attendant inflammation and potential consequences, rather 
than suggesting that HIV-1 viremia is associated independently with increased risk 
of OI. 
Please refer to Appendix 1: App – 1C for the schedule of assessment for the ATI 
portion of the study, Appendix XII for the ATI disclosure form for participants, 
Appendix XI for ATI risk mitigation and counseling forms, and Appendix XIII for 
PrEP information sheets for participants. 
2.4.3 Post ART Withdrawal Monitoring 
Once ART is reinitiated, participants will be evaluated at Weeks 4 and 8 on ART. 
If viral load is not below the level of quantification by Week 8, participants will be 
evaluated every four weeks until the viral load is confirmed to be less than the level 
of quantification. A follow up visit will occur 24 weeks after resumption of ART.  
Routine safety studies, CD4+/CD8+ T cell counts, and viral load will be performed 
at Weeks 4, 8, and 24, and as needed between Week 8 and Week 24 to monitor 
safety and evaluate decrease of viral load to below level of quantification as 
described above. Plasma and PBMCs will be collected and cryopreserved at the 
time of ART resumption and at Weeks 4, 8 and 24 on ART. Please refer to 
Appendix 1: App – 1C for the schedule of assessment for the ATI portion of the 
study. 
2.4.4 Change in ART withdrawal criteria (protocol version 8) 
The previous goal of ART withdrawal when T-cell count has risen to ≥ 500 
cells/mm3 was not achievable for most participants. Some patients achieved a CD4 
count of ≥ 300 cells/mm3. Resuming ART when CD4 is < 200 cells/mm3 is 
appropriate for safety reasons. Below 200, we expect participants to be prone to 

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OIs, hence most providers use this as a threshold to begin prophylactic antibiotic. 
This change in the CD4 criteria will allow participants to begin controlled ART 
withdrawal earlier around 6 months to a year and not wait for two years to do so. 
ART withdrawal in these participants will provide critical information about the kinetics of 
the selection of HIV-resistant transplanted clones of CD4 T-cells and the potential 
kinetics of HIV reservoir decay in that setting. This will inform the design of future 
studies when models of the threshold for a potential requirement for a proportion for 
transformed cells required for cure might be identified. In other words, the 
information collected during a structured STI may provide sufficient information to 
model what fraction of the transplanted product should be HIV resistant stem cells 
in order to achieve an extinction of the HIV reservoir. Although it is not anticipated 
that any of the participants from earlier cohorts will achieve control of their infection 
following an STI, we have observed an expansion of the transformed CD4 clone in 
participants who became viremic due to ART non-compliance. Ultimately, the goal of 
our treatment intervention is that the proportion of transformed CD4/HIV-resistance 
cells in recipients is sufficient such that they no longer need ART to control HIV 
infection. STI is the most certain way to establish that desired endpoint. Including 
participants from the earlier cohorts will assist in guiding the design of future clinical 
trials to achieve that goal.2.5 
Correlative Studies 
2.5.1 Biologic rationale and proposed mechanism of action for the study agent 
Survival in patients infected with HIV has improved dramatically with the advent 
of ART. However, neither a cure nor a vaccine exists. Infected individuals develop 
AIDS related malignancies such as lymphoma that are in general more aggressive 
and present with higher disease stages. Even after successful curative therapy of 
their malignancy, their survival depends on high compliance with ART therapy to 
prevent the relapse of lymphoma. Lack of immunological response despite 
virological suppression, issues with non-compliance or drug resistance, side effects 
of medications, and development of a well characterized but poorly understood 
chronic inflammatory status associated with different chronic illnesses are just a 
few of the complications that continue to negatively affect the quality of life and 
survival in patients with HIV related lymphomas. Hematopoietic stem cell (HSC) 
gene therapy for HIV may offer an alternative one-time treatment, with the 
possibility of controlling both the lymphoma as well as HIV itself by eliminating 
reservoirs of HIV that are responsible for persistence of the disease. Our proposed 
novel anti-HIV therapeutic candidate, autologous HSCs transduced with a triple 
combination of anti-HIV genes transferred by a single lentiviral vector is aimed at 
blocking HIV infection at different stages of the HIV life cycle. This approach 
provided strong pre-integration inhibition of HIV-1 infection, eliminated the 
generation of viral escape mutants, and prevented the integration of multiple, 
including resistant strains of HIV in various cell types in vitro. In vivo 
safety/efficacy studies in a humanized mouse model displayed excellent results 
including a selective survival advantage of anti-HIV gene modified cells and 
maintenance of normal human CD4+ cell levels. We also use a novel method of 
transduction that improves our transduction efficiency to a more clinically relevant 
level. Furthermore, for the first time in any HIV gene therapy approach, we are 

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proposing to use a selectable surface marker on transduced cells to purify and 
transplant only anti-HIV vector transduced HSCs. Significantly, this improvement 
in transduction efficiency and selection for anti-HIV transduced HSC, resulted in 
decreased viremia in our model. We have successfully filed an application to the 
NIH Recombinant DNA Advisory Committee (RAC) and have been awarded an 
FDA IND approval and have started to manufacture the clinical GMP reagents for 
our clinical trial. We will be targeting HIV related lymphoma patients that require 
transplant as a part of their standard of care. This provides us an ethically acceptable 
setting to test the safety and efficacy of our product. By using a highly effective 
combinatorial anti-HIV gene therapy approach, developing a novel and 
significantly improved transduction process, revising transplant procedures to 
transduce HSCs before freezing and, most importantly, selecting and transplanting 
only the HIV resistant transduced HSCs, we expect to replace the patient’s immune 
system with HIV resistant cells, thus depleting viral load in the absence of ART. 
2.5.2 Specific hypothesis 
Anti-HIV gene expressing stem cells can safely reconstitute the hematopoietic 
system of the recipient and can produce a pool of mature myeloid and lymphoid 
cells that harbor anti-HIV genes capable of resisting HIV-1. As a secondary 
objective, we will interrogate two physical compartments of immune system, (i.e., 
peripheral blood (PB) cells and gut mucosa) for the presence, quantity and duration 
of gene modified HIV-1-resistant immune cells. In addition, we will determine the 
integration sites of vector sequences in circulating cells and monitor for any clonal 
expansion of hematopoietic cells. 
2.5.3 Relevant preclinical data 
We have obtained strong preclinical data in both our in vitro and in vivo 
experiments demonstrating safety and efficacy of our tCD25 pre-selective 
combination anti-HIV lentiviral vector. This vector encodes a chimeric 
human/rhesus macaque TRIM5α isoform, a CCR5 shRNA, and a TAR decoy each 
of which inhibit HIV in a different stage of its life cycle. It also expresses a 
truncated/mutated form of human CD25 which is used as a selectable marker to 
purify transduced HSC away from untransduced HSC so an enriched population of 
HIV-resistant HSC can be transplanted into patients. 
SAFETY/FEASIBILITY/TOXICITY STUDIES: To evaluate the levels of 
enrichment of 1TAX vector transduced HPC, flow cytometry was performed on 
CD25 immunomagnetic bead purified cells. As displayed in Figure 2-B, enrichment 
levels ranged from 89.6% to 98.3% with an average of 94.2% (Post‐enrich). This 
was compared to initial transduction efficiencies, which ranged from 19.8% to 
60.4% with an average of 33.0% (Pre‐enrich). Human HPC do not normally express 
CD25 and this is displayed as background expression, ~1.33% (Non-transduced, or 
NT). Flow through (ft) levels from the Stem Cell Technologies magnetic columns, 
~2.07%, demonstrated that the majority of 1TAX vector transduced cells were 
collected during the enrichment procedure. 
 
 

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Figure 2-B: Enrichment of 1TAX vector transduced cells: Human CD34+ HPC were 
transduced with the 1TAX vector, purified by CD25 immunomagnetic beads, and analyzed 
by flow cytometry for CD25 expression. Non‐ transduced cells (NT), flow through (ft) 
Enrichment of 1TAX vector transduced cells: Human CD34+ HPC were 
transduced with the 1TAX vector, purified by CD25 immunomagnetic beads, 
and analyzed by flow cytometry for CD25 expression. Non‐ transduced cells 
(NT), flow through (ft) 
Safety was observed in 1TAX vector transduction of HPC using colony forming 
unit assays and differentiation of these colonies into phenotypically normal 
macrophages. No significant differences in colony formation or expression of 
normal macrophage cell surface markers was observed in 1TAX vector transduced 
HPC compared to NT cells. Genetic stability of integrated 1TAX vector including 
the lack of deletions and rearrangements was also observed. The correct sequence 
of the integrated 1TAX vector was observed in transduced HPC. Expression of all 
three anti-HIV genes was detected in 1TAX vector transduced HPC. Potent down 
regulation of CCR5 expression was observed in 1TAX vector transduced cells 
(>94%, p<0.001) (Figure 2-C). 
 
 

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Figure 1-C: Down regulation of CCR5 expression on 1TAX cells: Ghost‐R5/X4/R3 cells 
transduced with the 1TAX vector were analyzed by flow cytometry for CCR5 cell surface 
expression. A representative histogram is displayed with NT cells (unshaded) and 1TAX 
vector transduced cells (shaded). Experiments were performed in at least triplicate. *** 
p<0.001 
 
With the transduction of lentiviral vectors, there is the possibility for integration to 
occur in an area of the genome which may immortalize that specific transduced 
cell. Therefore, to determine if any immortalization occurred with 1TAX‐
transduced HPC, a long-term culture assay was performed similar to previously 
described (Modlich et al., 2009). No visual immortalization had occurred in either 
the 1TAX transduced or NT HPC cultures upon scoring 192 individual wells. To 
further evaluate the tumorigenicity of 1TAX vector transduced HSC, a NOD-
RAG1-/-IL2rgamma-/- (NRG) knockout mouse model was used. Human CD34+ 
HSC were transduced with the 1TAX vector, enriched by CD25 immunomagnetic 
bead separation and transplanted into NRG pups (N=8). Engrafted mice were then 
left in the UC Davis vivarium for 6 months and transferred to the UC Davis 
Comparative Pathology Laboratory where they were euthanized and evaluated for 
any tumors. Gross necropsy and histologic examination of all major organs was 
performed. The anatomic pathologist and director of the comparative pathology lab 
confirmed that there were no tumors noted grossly or histologically. 
To evaluate the in vivo safety and toxicity of 1TAX vector transduced HSC, human 
CD34+ cells transduced with the 1TAX vector were transplanted into NRG pups 
(N=10). Twelve weeks post-transplantation, mice were screened for engraftment in 
the peripheral blood by flow cytometry. 1TAX transplanted HSC (55.5%) (N=10) 
displayed similar CD3+/CD4+ human T cell engraftment as NT HSC (51.6%) 
(N=7) transplanted mice. The phenotypic profile of peripheral blood 1TAX T cells 
was analyzed by multi-color flow cytometry. No significant differences in the 
percentage of the CD3+ T cell markers, human CD4+ (p=0.50), CD8+ (p=0.32), 
CD4+/CD8+ (p=0.34), CXCR4 (p=0.08), CD28 (p=0.17), and CD45RA+/CCR7+ 
(p=0.84), were observed when comparing NT and 1TAX vector transduced cells 
(Figure 2-D). 
 
 

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Figure 2-D: Phenotypic profile of 1TAX vector transduced peripheral blood T cells: Human 
CD34+ HSC were transduced with the 1TAX vector and transplanted into NRG mice. The 
peripheral blood of engrafted mice was analyzed by multi-parametric flow cytometry for T 
cell phenotypes 
NRG mice engrafted with 1TAX transduced and enriched cells were then evaluated 
for human immune cells in other hematopoietic sites such as the spleen, thymus, 
and bone marrow. 1TAX transplanted HSC (54.4%) (N=10) displayed similar 
CD3+/CD4+ human T cell engraftment levels as NT HSC (59.7%) (N=7) 
transplanted mice. No significant differences in the percentage of the CD3+ T cell 
markers, human CD4+ (p=0.36), CD8+ (p=0.64), CD4+/CD8+ (p=0.48), CXCR4 
(p=0.20), CD28 (p=0.08), and CD45RA+/CCR7+ (p=0.62), were observed when 
comparing NT and 1TAX vector transduced cells. 1TAX vector transplanted HSC 
(41.7%) (N=10) displayed similar CD3+/CD4+ human T cell engraftment levels as 
NT HSC (42.9%) (N=7) transplanted mice. No significant differences in the 
percentage of the CD3+ T cell markers, human CD4+ (p=0.79), CD8+ (p=0.18), 
CD4+/CD8+ (p=0.13), CD3-/CD8+ (p=0.0.58), CXCR4 (p=0.09), CD28 (p=0.43), 
and CD45RA+/CCR7+ (p=0.34), were observed when comparing NT and 1TAX 
vector transduced cells. 
The levels of 1TAX B cells in the spleen and bone marrow and 1TAX macrophages 
in the bone marrow of engrafted NRG mice were also analyzed by flow cytometry. 
No significant differences in the levels of spleen B cells (p=0.47), bone marrow B 
cells (p=0.27), or bone marrow macrophages (p=0.22) were observed in 1TAX 
compared to NT cell engrafted mice. 

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These results demonstrate that 1TAX vector transduced and enriched HSC are safe, 
are not toxic, and are able to successfully engraft in NRG and undergo normal 
multi-lineage hematopoiesis in the peripheral blood and lymphoid organs all while 
not causing tumorigenicity. 
EFFICACY STUDIES: The ultimate goal of HIV gene therapy is to transplant a 
HIV‐resistant immune system into HIV infected patients. To evaluate the efficacy 
of 1TAX vector transduced HSC in inhibiting HIV infection, both in vitro and in 
vivo challenge experiments were performed. 
First, an in vitro HIV-1 model system incorporating human CD34+ HPC derived 
macrophages was used. To evaluate the anti‐HIV efficacy of enriched 1TAX vector 
transduced cells, in vitro HPC derived macrophages were challenged with a CCR5‐
tropic BaL‐1 strain of HIV‐1. As displayed in Figure 2-E, significant resistance 
(over 2 logs on days 14 and 21 post-infection) to HIV‐1 infection was observed in 
1TAX vector transduced cells compared to NT cells (day 3 p=0.033, day 7 p<0.001, 
day 14 p<0.005, day 21 p<0.001, and day 28 p<0.001) as determined by p24 antigen 
Enzyme Linked Immunosorbent Assay (ELISA). 
Figure 2-E: HIV‐1 challenge of 1TAX HSC derived macrophages: A) Nontransduced (NT) 
(♦) and 1TAX vector transduced (■) HSC derived macrophages were challenged with a 
CCR5-tropic BaL-1 strain of HIV-1. On multiple days post‐infection, cell culture 
supernatants were sampled and assessed for HIV-1 using a p24 antigen ELISA kit. 
Experiments were performed in triplicate. * p<0.05, ** p<0.01, *** p<0.001 
An in vitro selective survival experiment was performed to determine if anti-HIV 
gene modified cells were capable of surviving during the course of an HIV-1 
infection. In cultures containing a 1:1 and 2:1 ratio of combination vector 
transduced cells to untransduced cells, the pre‐infection Enhanced Green 
Fluorescent Protein (EGFP) percentage being, ~46% and ~73% respectively, 
increased to >84% EGFP positive cells by day 21 post‐infection for both BaL‐1 and 
NL4‐3. This increase was statistically significant (BaL‐ 1 50% p value=0.001, BaL‐

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1 75% p value=0.05) (NL4‐3 50% p value=0.001, NL4‐3 75% p value=0.001). 
These results are in contrast to cultures containing EGFP‐alone control vector 
transduced cells where the EGFP percent positive cell population remained 
relatively constant on day 21 post‐infection (~42% and ~72% for ratios of 1:1 and 
2:1, respectively) compared to the pre‐infection EGFP percentages of 46% and 70% 
(BaL‐1 50% p value=0.23, BaL‐1 75% p value=0.69) (NL4‐3 50% p value=0.62, 
NL4‐3 75% p value=0.95). 
To determine if viral inhibition was occurring at the stages of pre-entry (due to the 
CCR5 shRNA) and post-entry/pre-integration (due to TRIM5α), cells challenged 
with both BaL-1 and NL4-3 were further analyzed by quantitative polymerase chain 
reaction (qPCR) for genomic HIV-1 provirus. A highly potent block of HIV-1 
provirus formation was demonstrated. Anti-HIV vector transduced cells contained 
undetectable levels, similar to background levels in uninfected cells, of HIV-1 
provirus as compared to control untransduced and EGFP-alone vector transduced 
infected cells at multiplicity of infections (MOIs) of 0.01 and 0.05 for BaL-1 and 
NL4-3 strains of HIV-1. These data confirm that the anti-HIV vector conferred a 
strong block to HIV-1 integration, therefore preventing HIV integration. 
The hallmark characteristics of progressive HIV infection include a decline in 
CD4+ cells and an increase in plasma viremia. To evaluate the in vivo anti‐HIV 
efficacy of 1TAX transduced cells, a humanized NRG mouse model was used. 
NRG mice engrafted with 1TAX HSC were infected with either a CCR5‐ tropic 
BaL‐1 or CXCR4‐tropic NL43 strain of HIV‐1. On specific weeks post-infection, 
the peripheral blood of infected mice was analyzed by flow cytometry for CD4+ T 
cells and qPCR for plasma viremia. Mice engrafted with NT HSC (N=7) and 
infected with BaL‐1 displayed a depletion of CD4+ T cells from pre‐infection levels 
(68.1%) to 15 weeks post‐infection (21.6%). This was in comparison and 
significantly different (p=0.0019) than mice engrafted with 1TAX HSC (N=7) and 
infected with BaL‐1 which did not display CD4+ T cell depletion with pre‐infection 
levels of 68.8% and 15 weeks post‐infection of 77.9% (Figure 2-F A). Similar 
results were observed in NT and 1TAX engrafted mice infected with NL43. NT 
mice (N=4) displayed a decrease in CD4+ T cell levels from pre‐infection (78.1%) 
to 15 weeks post‐infection (14.8%) and 1TAX mice (N=4) displayed a decrease in 
CD4+ T cell levels from pre‐infection (74.0%) to post‐infection (56.3%) (Figure 2-
F B). A significant (p=0.028) depletion of CD4+ T cells was observed on week 15 
post‐infection when comparing NT and 1TAX mice. Even though a decrease in 
CD4+ T cells (pre‐infection to post‐infection) was observed in 1TAX mice infected 
with NL43, this decrease was not significant (p=0.23). These results demonstrate 
that maintenance of CD4+ T cell levels could be achieved when an enriched 
population of 1TAX‐transduced HSC is transplanted into NRG mice. 
 
 

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Figure 2-F: Maintenance of human CD4+ T cells in vivo in 1TAX cell engrafted NRG mice 
infected with HIV‐1: NRG mice engrafted with either untransduced (NT) or 1TAX vector 
transduced cells were infected intraperitoneally with a (A) CCR5‐tropic BaL‐1 or a (B) 
CXCR4‐tropic NL43 strain of HIV‐1. Various weeks post‐infection, mice were bled via tail 
vein and analyzed by flow cytometry for total human CD3+/CD4+ cell percent. Bar graphs 
display averages and standard deviations from seven mice per cohort for the BaL‐1 
infections and four mice per cohort for the NL43 infections for CD3+/CD4+ cell levels pre-
infection and 15 weeks post‐infection. * p<0.05 and ** p<0.002 
 
 
 
To evaluate the levels of HIV‐1 viremia in BaL‐1 and NL43 infected NT and 1TAX 
mice, qPCR was performed on peripheral blood plasma on various weeks post- 
infection. As displayed in Figure 2-GA, inhibition of HIV‐1 infection (~1.5 logs) 
was observed in 1TAX cell engrafted mice (solid black line) compared to NT cell 
engrafted mice (solid grey line). A significant difference in plasma viremia was 
observed on week 9 (p=0.0092) and week 15 (p=0.0043) post‐infection. Levels of 
plasma viremia in 1TAX mice infected with BaL‐1 were similar to those of 
background-uninfected mice (dashed grey line). As for the NL43 infections, 
inhibition of HIV‐1 infection was observed (~2 logs) in 1TAX cell engrafted mice 
(solid black line) compared to NT cell engrafted mice (solid grey line) (Figure 2-
GB). A significant difference (p=0.0073) was observed on week 15 post-infection. 
Again, levels of plasma viremia in 1TAX mice infected with NL43 were similar to 
those of background-uninfected mice (dashed grey lines). These results highlight 
the ability of 1TAX vector transduced HSC to generate a HIV‐resistant immune 
system when transplanted into NRG mice. 
 
 

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Figure 2-G: Reduction in HIV-1 plasma viremia in NRG mice engrafted with 1TAX vector 
transduced HSC: NRG mice engrafted with either untransduced (NT) or 1TAX vector 
transduced cells were infected intraperitoneally with a (A) CCR5‐tropic BaL‐1 or a (B) 
CXCR4-tropic NL43 strain of HIV‐1. Various weeks post-infection, mice were bled via tail 
vein and analyzed by qPCR for plasma viremia using a primer/probe set specific for the HIV 
LTR. Log RNA copies/mL for NT (♦) (solid grey lines), 1TAX vector transduced (■) (solid 
black lines), and background levels from uninfected (UI) mice (▲) (dashed grey lines). Line 
graphs display averages and standard deviations from seven mice per cohort for the BaL‐1 
infections and four mice per cohort for the NL43 infections. * p<0.05 and ** p<0.005. 
 
Here, as we were able to initially enrich for 1TAX vector transduced cells prior to 
transplantation, we were able to generate a human immune system in the NRG mice 
which was resistant to HIV infection. This allowed for only a small percentage of 
NT and unprotected cells being transplanted with the majority of the transplanted 
cells protected. Combined, these results and preclinical data highlight the potential 
of our therapeutic candidate to succeed in our Phase I human clinical trial. 
2.5.4 Reason for selection of the assay methodology 
To evaluate the differentiation and persistence of anti-HIV HSC in recipient 
participants including functional and phenotypic changes in immune cell 
populations following HSC transplant, and to measure viral load and HIV response. 
Correlative studies, especially for gene therapy protocols, are critical to evaluate 
the feasibility, safety, and efficacy of the therapeutic intervention. 
The correlative studies can be divided into immunological and HIV studies, DNA 
monitoring studies, and monitoring for oncogenesis and hematopoietic clonality. 
We will evaluate various aspects of the anti-HIV lentiviral therapy including DNA 
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expression of anti-HIV genes and stability of the integrated vector will also be 
evaluated. The primary objectives of the immunology studies in this protocol are to 
monitor the reconstitution of the immune system, especially the homeostasis and 
function of T cell immunity in HIV-infected participants transplanted with anti-
HIV gene transduced stem cells. A large number of clinical trials have shown that 
chronic HIV infection results in depletion of key T cell subsets. 
2.5.4.1 DNA monitoring studies 
The DNA monitoring core will evaluate engraftment (critical for efficacy) 
and safety of our approach in peripheral blood and gut tissue of transplanted 
participants. We will evaluate sites of vector integration, immune responses 
to foreign genes, potential silencing of anti-HIV genes (CCR5 shRNA, 
chimeric TRIM5α, and TAR decoy), and toxicity of the cells including 
tumorigenicity. Pre-transplant samples will be collected from the UC Davis 
GMP facility and also from leftover infusion bags. Post-transplant, both 
peripheral blood and biopsies will be obtained according to the timeline in 
Appendix I. 
Approximately 20 mL of ethylenediaminetetraacetic acid (EDTA) anti-
coagulated blood specimens will be collected for DNA monitoring studies 
(see Appendix VII for additional details of DNA monitoring studies). 
Objective 1 
Determine the levels of in vivo gene marking and vector 
copy number in 1TAX vector transduced cells. 
Total DNA will be extracted from the peripheral blood of 
participants transplanted with the 1TAX vector transduced 
cells. qPCR will be performed with a vector-specific 
primer/probe set specific for the chimeric TRIM5α 
transgene. The levels of glyceraldehyde-6-phosphate 
(GAPDH) expression will also be evaluated as an internal 
control. 
Objective 2 
Determine integrated 1TAX vector sequence and stability. 
To evaluate integrated 1TAX vector sequence and stability, 
sequencing will be performed. Total DNA will be extracted 
from the participant’s peripheral blood. PCR amplification 
and sequencing (with vector specific primers) of integrated 
1TAX vector will be performed. Sequences obtained will be 
evaluated for rearrangements, deletions, and additions. 
Objective 3 
Evaluate expression of the anti-HIV genes: 
To determine if all three anti-HIV genes (CCR5 shRNA, 
chimeric TRIM5α, and TAR decoy) are being expressed 
long-term, qPCR will be performed. Total RNA will be 
extracted from the participant’s peripheral blood. qPCR with 
gene specific primers will be performed to determine 
expression levels. GAPDH expression levels will be used as 
an internal control. 

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Objective 4 
Evaluate clonality of 1TAX transduced cells in duodenal 
tissue: 
In any gene therapy protocol, there is a risk of insertional 
oncogenesis due to random insertion of the vector. We will 
routinely monitor participants for any evidence of clonality. 
This is important for stem cell transplantations in lymphoma 
patients since there is an inherent risk of secondary 
malignancies (about 5-8%) due to the chemotherapy both as 
a treatment for controlling the malignancy and as a 
transplant preparative regimen. We will perform LAM-PCR 
every 6 months for 1TAX vector integrant sites and look for 
evidence of increasing oligoclonality or monoclonality, 
defined as 2-fold increase over two successive timepoints to 
> 20% of total transduced cells. 
Objective 5 
Determine if an immune response develops in participants 
due to the expression of the chimeric TRIM5α transgene. 
The chimeric TRIM5α anti-HIV gene contains a 13-amino 
acid patch which is foreign to native human TRIM5α. 
Therefore, it is possible for an antibody based immune 
reaction to occur against cells expressing this region. 
However, this is highly unlikely in a myeloablative setting 
when immune tolerance can be induced against more 
prominent antigens such as human leukocyte antigen (HLA). 
Also, in our preliminary data, no stimulation was observed 
in cells pulsed with this amino acid region. Still and in 
accordance with the FDA, we will monitor participant serum 
for any antibody production by ELISA. 
2.5.4.2 Immunological studies 
The immunological studies will be done in 2 compartments: peripheral 
blood and gut mucosa. 
A large number of clinical studies have demonstrated that chronic HIV 
infection results in preferential depletion of key T cell subsets, namely naïve 
and various T memory cells. An increased level of T cell activation has also 
been demonstrated in association with disease pathogenesis in AIDS 
patients as measured by HLA-DR and CD38 co-expression on CD8+ T 
cells. More recently, other cell surface markers have also been tested 
including CD69 and CD71. In addition to the imbalanced Th1/Th2 subsets, 
HIV infection also induces impairment in Th17 and regulatory T cells 
(Treg), an imbalance that reflects a systemic immunologic defect more 
pronounced in advanced HIV disease and after effective viral suppression 
with ART. 
In the current proposal, homeostasis and differentiation of major T cell 
populations, and HIV-specific T cell responses will evaluate the adaptive 

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antiviral T cell functions. Furthermore, Type-1 and Type-2 T cell responses 
to common pathogens including CMV and EBV antigens will be assessed. 
Additional components of the innate immune system, including NK, NK-T 
cells, and dendritic cells will be examined in order to characterize functional 
immunologic changes following transplant. Furthermore, antigen-specific 
T cell responses will evaluate the adaptive antiviral T cell functions. 
Approximately 40 mL EDTA anti-coagulated blood specimens and 5 mL of 
non-anticoagulated blood (serum) will be collected for immunological 
studies. 
Out of the 40mL blood, about 2-mL whole blood will be used as fresh for 
phenotyping assays to determine the absolute counts of T cells, B cells and 
NK cells. The rest of blood will be: 
• Isolate plasma and peripheral blood mononuclear cells (PBMC) (38 
mL): 
o 10 mL plasma will be collected and stored in 1 mL aliquots for 
cytokine assays in later days (Year 3 and Year 4). 
o 10-20x106 cells will be used in assays immediately as fresh (listed 
as Functional assay in Section 2.5). Out of these cells, a minimum 
of 5x106 PBMC will be used for phenotyping assays of immune cell 
populations. A minimum of 10x106 PBMC will be needed for T cell 
function assays (T cell responses to pooled HIV peptides 
stimulations, to CEF (CMV-EBV-influenza virus), as well as culture 
median only (negative control) and to one of the positive mitogenic 
stimulations (positive controls, PMA or SEB (Staphylococcal 
enterotoxin-B)). 
o The rest of PBMC will be cryopreserved in 10% DMSO-FBS and 
stored in liquid nitrogen for testing in a later time, tentatively 
scheduled in Year 3 and Year 4. The expected number of PBMC in 
cryopreservation is 20-30x106, but could be less, especially for 
earlier time points post-transplant. In this case, we will make sure at 
least 5x106 cells will be preserved for any possible need of retesting. 
From the plasma (10 mL) and serum samples (3 mL), aliquot as stored 
samples till batched assays, tentatively scheduled for Year 3 and Year 4. 
Cytokines studies will focus on the increased inflammatory status seen in 
patients with controlled HIV associated with immune exhaustion. They 
include but not limited to TNF-alpha, interferon gamma, IL2, IL10 and also 
other markers of chronic immune activation/exhaustion such as soluble 
PDL1/2. 
 
 

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Reconstitution of immune system in peripheral blood 
Our laboratory will use established polychromatic flow cytometry panels to 
enumerate lymphocyte subsets and antigen specific cytokine expression in 
CD4 and CD8 T cells. Isolated PBMC from 40 mL EDTA anti-coagulated 
blood specimens will be collected from each participant at baseline, and at 
3, 6, 12, and 24 months post HSCT for the following studies. The processing 
for participants enrolled at UC Davis will be done at UC Davis Medical 
Center. Critical assays will be performed with fresh whole blood and freshly 
isolated PBMC. Additional PBMCs will be stored in liquid nitrogen for later 
time assays (and necessary re-test). 
• Chimerism of engrafted transformed cells:  
A portion of whole blood sample (~5mL) and a minimum of 10 x 106 
PBMC will be tested in the flow cytometric assays to:  
a. Determine the proportion of circulating chimeric CD34+ cells 
expressing CD25. 
b. Determine the proportion of circulating chimeric non-lymphoid 
immune cells expressing CD25 will be explored employing a variety 
of antibody subsets to identify the most robust subset believed to 
reflect engrafted and persistent transformed populations. 
c. Consideration will be given to sorting these populations for PCR 
verification of TRIM5ɑ genotype. 
• Mixed lymphocyte reactions: 
In patients who are losing engraftment of the transduced cells, mixed 
lymphocyte reactions will be monitored in order to determine whether 
unmodified, post-SCT PBMC recognize and kill engrafted, modified 
cells. A mixed lymphocyte reaction will be performed using post-SCT 
PBMCs obtained when more than 50% drop in transduced cell 
chimerism is determined by PCR. PBMC obtained from 20 cc of 
peripheral blood will be mixed with pre-SCT modified stem cells 
(generated from remaining CD34+ depleted CD4+ T cells from pre-
SCT leukapheresis). This will be performed in the immunology core. 
Gene modified cell killing and markers of T and NK cell activation and 
function will be determined in the ex vivo experiments using flow 
cytometric analysis. 
To study the homeostasis of T Lymphocytes and their subsets, a small 
portion of whole blood sample (~2-mL) and a minimum of 5x106 PBMC 
will be tested in the flow cytometric assays to: 
a. Determine memory and naïve CD4 and CD8 T cells and T cell 
activation: 
A multi-parameter test will be performed to detect viable CD3, CD4, 
CD8, CCR7, CD45RA, CD38, HLA-DR, CD27, CD57, Ki67 and 
viable cell stain. Naïve, central memory and effector T cells will be 

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analyzed by expression of CCR7/CD45RA on T cells that are gated 
on viable-cell/CD3 and CD4 or CD8 staining. T cell activation will 
be measured using combinations of CD38/HLA-DR as well as 
CD27, CD57 and/orKi67. 
b. Determine the Treg, NK, NK-T cells, and DC subsets: 
These will be examined with antibodies to CD45, CD11c, CD123, 
CD3, CD4, CD8, CD25, CD16, CD56, IL-7R, FoxP3, and viable 
cell stain. Expression of CD25+/ FoxP3+/IL-7R- will be used to 
identify Treg in viable CD3/CD4 cells. NK and NK-T cells will be 
identified by markers of CD3, CD16/CD56, and CD8. Expression 
of homing molecules (integrin α4β7, CD62L, CCR9, CD18, CD31 
and CD54) and inhibitory and activating receptors on NK cells will 
also be included in this panel pending on specimen availability. 
c. Pending specimen availability, homeostasis of B cell classes and 
expression of T cell homing molecules (integrin α4β7, CD62L, 
CCR9, CD18, CD31, and CD54) will also be accessed (at baseline 
and month 12). 
• Functional responses upon reconstitution of T Cell Immunity 
Isolated PBMC will be cultured with mitogen or HIV-specific antigens 
and used in the following studies. For participants enrolled at UC Davis, 
the processing starts as soon as possible after blood collection, usually 
within 1-hour. For participants enrolled at other sites: EDTA blood 
samples will be sent to UC Davis with chilled packaging via FedEx 
Priority Overnight shipping: 
a. Total Th1/Th2/Th17 subsets upon mitogen stimulation: 
The detection panel will be: viable stain, CD3, CD4, CD8, and intra-
cellular Granzyme-B, IFNγ, TNFα, IL2, IL4, IL10, and IL17a. 
Th1/Th2/Th17 subsets will be identified as IFNγ/TNFα/IL2, 
IL4/IL10, or IL17a, respectively, on viable CD4 T cells. 
b. HIV antigen specific T cell responses: 
PBMC stimulated with HIV-gag peptides will be examined with a 
panel of viability stain, CD3, CD4, CD8, and intra-cellular IFNγ, 
TNFα, IL2, IL4 and IL17a. The profile of cytokine(s) produced in 
viable CD4 or CD8 T cells will be analyzed and correlated to 
Granzyme- positive T cells. T cell responses to Staphylococcal 
enterotoxin-B (SEB) will be used as positive controls. Another 
control antigen, CMV-EBV-influenza virus (CEF) peptides antigens 
will be used as an indicator of T cell responses to pathogens 
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c. T cell proliferation responses to HIV gag stimulations: 
If additional PBMC are available (15x106 cells more), proliferation 
responses will also be tested using CFSE (Carboxyfluorescein 
succinimidyl ester) assays. Furthermore, at one-year post transplant, 
based on the American Society of Blood and Marrow 
Transplantation (ASBMT) guidelines, participants will be 
immunized with inactivated vaccines. Acquisition of immunity 
against a neoantigen can therefore be assessed in these patients by 
their response to immunization with a replication incompetent 
antigen such as tetanus toxoid. 
Analysis of the extra-vascular compartment of the immune system (gut 
mucosa: 
Immune restoration in the duodenum is profoundly blunted with absolute 
numbers of CD4+ T cells in the lamina propria changing little after 
prolonged viral suppression. We believe that a thorough evaluation of 
immune effects of our product will be enhanced by demonstration of 
transduced cells in duodenal tissue. It is anticipated that transduced cells 
will home to the lamina propria and will be particularly enriched during 
HIV treatment interruption. Presence of transduced immune cells in gut 
associated lymphoid tissue (GALT) will likely be a hallmark of the success 
for our gene therapy protocol. Our HIV team members are leaders in the 
area of gut immunity and reconstitution in response to HIV infection, with 
a long track record of performing upper endoscopy safely in HIV research 
participants, having performed over 100 procedures on 60 research 
participants over the last 4 years. 
To demonstrate homing/persistence of transduced lymphocytes in this 
compartment, the distal duodenum will be sampled at 6 months post-
transplant (or when CD4 count reaches above 300 cell/mm3) and will be 
repeated two months after the first GI biopsy. This will facilitate addressing 
two important questions, namely the uptake of the transduced population of 
lymphocytes into the extravascular compartment and the degree of 
enrichment that occurs following selective survival pressure on these 
lymphocytes during ART withholding. The categorical residence of 
transduced cells and both their functional integrity and that of the local 
immune system in general will be assessed for both IHC and digestion into 
single cell suspension for flow cytometry. 
The distal duodenum will be sampled by a designated gastrointestinal (GI) 
physician at each center at 6 months post-transplant (or when CD4 count 
reaches above 300 cell/mm3) and will be repeated two months after the first 
GI biopsy. This will be under separate consent form for the study and the 
participation therefore is not mandatory for enrollment in the main study 
(see Appendix V). 
• Tissue biopsies will be immediately processed at the time of endoscopy 
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single cell suspension for flow cytometry. Cryopreserved samples can 
be used for HIV quantitation and sequencing, RNA microarray, and 
quantitation of transduced cells by PCR of the inserted vector. 
• IHC will be performed to enumerate critical populations of the mucosal 
immune system, specifically CD4+ and CD8+ T cells and T-regulatory 
cells. 
• Flow cytometry will be performed in unstimulated CD4+ and CD8+ T 
cells and T-regulatory cells. 
• Functional assays of immune function will be performed by incubating 
duodenal cells with mock challenge; SEB, CEF, and HIV gag peptides 
for intracellular cytokine measurement by flow cytometry of TNFα, 
IFN-γ, IL-2, IL-17a, and CD103. 
These experiments are expected to demonstrate both the persistence of the 
transduced lymphocyte population, as well as improved immunologic 
function in this compartment, which can be compared to the time series of 
parallel immune function assays performed on PBMCs over the same time 
period. 
2.5.4.3 Virologic studies 
A. ART resistance and co-receptor usage: These studies will be performed 
at baseline, 2 and 10 weeks and then 6, 12, and 24 months post-
transplant.  
B. To characterize changes in the HIV reservoir and viral diversity: 
Additional blood samples will be collected at baseline, 2 and 10 weeks 
and then 6, 12- and 24-months post-transplant for HIV reservoir 
characterization. These studies will include quantitative PCR analysis 
of PBMC-associated HIV DNA, RNA (including unspliced and 
multiply spliced forms) and 2-LTR circles. In addition, single genome 
sequencing of HIV gp120 and HIV pol will be performed to understand 
sequence evolution following transplantation and detection of minority 
resistance variants. Single-copy HIV from plasma will also be 
determined at each of these time points. Samples will be sent to the 
Timothy Henrich Laboratory at University of California, San Francisco. 
2.5.5 1TAX lentiviral vector stability 
The marker is a stably integrated lentiviral vector harboring a combination of anti-
HIV genes and a pre-selective marker gene. As the 1TAX vector is stably 
integrated, it will remain permanently in the transduced cells. 
2.5.6 Technical performance characteristics, positive and negative controls, and methods 
of scoring for the assays 
Study T lymphocytes and their subsets 
The flow cytometry data is highly reproducible and accurate. Variability will be 
observed from participant to participant; however, samples will be evaluated and 

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compared from the same participant to decrease variability. 
Flow cytometry positive controls will be obtained from normal healthy donors. 
Negative controls are isotype stained cells. 
The flow cytometry data will be scored by percent + cell populations staining for 
the specific immune cell surface markers. 
Functional responses upon reconstitution of T cell immunity 
The functional cytokine secretion data is highly reproducible and accurate. 
Variability will be observed from participant to participant; however, samples will 
be evaluated and compared from the same participant to decrease variability. 
The functional cytokine secretion positive controls will be obtained from normal 
healthy donors. Negative controls are unstimulated cells from the same participant. 
The functional cytokine secretion data will be scored based on the levels of 
cytokines as measured in ng/mL. 
Monitoring for insertional oncogenesis and clonality of hematopoiesis after gene 
therapy 
We will use LAM-PCR (Linear Amplification Mediated PCR), a highly accurate 
method that is able to specifically identify the sites of integration of the 1TAX 
vectors. Variability will be observed even in the same participant samples as the 
vector is capable of integrating in 1000s of places in the targeted genome. 
There are no positive or negative controls for this assay. This is specific for 1TAX 
vector integration sites. It is not possible to specifically integrate a 1TAX lentiviral 
vector into the chromosome of a host genome. 
Insertional oncogenesis and clonality will be scored based on the sites of integration 
of the 1TAX vector and the number of events correlating to each specific 
integration site. 
DNA monitoring studies for in vivo gene marking, vector stability, and anti-HIV 
gene expression 
These assays are highly accurate and specific due to the use of primers specific for 
the 1TAX vector and expressed genes. Variability will be observed between 
participant to participant due to the different levels of engraftment. This will affect 
the levels of in vivo gene marking and anti-HIV gene expression. Variability will 
be minimized by comparing the results of these assays within each sample from the 
same participant. 1TAX vector stability should be consistent as we have not 
observed any deletions or rearrangements in our preclinical data. 
The positive control for in vivo gene marking is the transfer plasmid used for vector 
production, which includes the psi sequence. The negative control is a plasmid that 
does not contain the vector specific psi sequence. 1TAX vector stability positive 
control is again the transfer plasmid used for vector production. The negative 
control for 1TAX vector stability is genomic DNA from untransduced cells. The 
positive control for anti-HIV gene expression is total RNA from 1TAX vector 

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transduced HEK-293T cells. The negative control for anti-HIV gene expression is 
total RNA from untransduced cells. 
In vivo gene marking will be scored by 1TAX vector copies per cell. Vector stability 
will be scored based on the correct size of the PCR product in comparison to the 
positive control transfer plasmid. Anti-HIV gene expression will be scored by 
relative gene expression as compared to GAPDH expression (housekeeping gene). 
Monitoring for immune response to TRIM5α 
The reproducibility of this assay will vary from participant to participant. This assay 
is designed to detect any immune response which may occur with the expression of 
the chimeric TRIM5α transgene and so we may never detect any immune response 
in participants. Accuracy will be assessed by baseline plasma levels obtained from 
participants prior to transplantation. 
The positive control for the TRIM5α ELISA is an anti-chimeric TRIM5α antibody 
generated in rabbits. The negative control is plasma from a normal healthy donor. 
The immune response to the chimeric TRIM5α will be scored based on the levels 
of anti-chimeric TRIM5α antibody in ng/mL. 
Monitoring of transduction efficiency and stem cell capabilities of transduced 
hematopoietic stem cells in immunocompromised mouse models 
Transduction efficiencies have been consistently reproduced to exceed 50% of the 
cell population. Variability is expected from participant to participant sample as we 
have observed in our preclinical data. 
The positive control for transduction efficiency is transduced HEK-293T cells. The 
negative control is an isotype control for flow cytometry. 
Transduction efficiency will be scored based on CD25+ cell expression by flow 
cytometry. 
2.5.7 The site performing the correlative studies 
UC Davis will perform the correlative studies. Dr. Pollard’s lab will perform the 
immunological core studies and Dr. Anderson’s lab will perform the DNA 
monitoring core studies. Specialized immunologic and virologic assays will also be 
performed in the laboratory of Dr. Timothy Henrich at University of California, 
San Francisco. Basic immunology studies will be performed according to the 
standard of care by CLIA-certified local laboratories at each participating 
institution. 
2.5.8 Additional sample for safety investigation 
In the unusual circumstance where an unexpected or sudden change in the subject’s 
clinical status occurs where product safety concerns raise a relevant question 
requiring additional blood samples outside the specified time points, participants 
may be asked for an additional blood sample (maximum of 30 mL of EDTA whole 
blood in a purple top tube), at the local PI’s discretion in consultation with the 
protocol chair. The samples will be shipped to Dr. Pollard’s Laboratory at UC Davis 
(see Appendix VII), and will be analyzed per the protocol correlative studies 

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relevant for the safety concern, such as mixed lymphocyte reactions. It is not 
anticipated that this will occur with every subject or on more than one occasion 
outside of the current schedule of events. 

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3.0 
PARTICIPANT SELECTION 
A rostered AMC investigator must document that each protocol participant meets meet all 
stated eligibility criteria. Participating sites must have documentation that each eligibility 
requirement is satisfied prior to participant enrollment. In compliance with CTEP policy, 
no exceptions to eligibility criteria will be granted under any circumstance. 
General participant population eligible for this trial: Male or female participants greater 
than 18 years of age with HIV-1 related lymphoma that are refractory/resistant to first line 
of therapy, or have relapsed after achieving a remission, and are eligible for an autologous 
HSCT as a part of their routine therapy are eligible for this study. 
The type of the chemotherapy and the number of cycles prior to the study, as well as the 
timing of stem cell collection in relation to the last chemotherapy, will be determined based 
on the transplant physician discretion and each individual institution guidelines. There 
should be a minimum of 2 weeks from the last day of any chemotherapy (except the 
one used for stem cell mobilization) before a participant can be enrolled on this trial. 
While screening studies may be performed within 8 weeks of the stem cell collection, 
enrollment in the screening segment should occur before stem cell collection and no more 
than 1 week prior to stem cell collection. 
NOTE: Institutions may use this section of the protocol as an eligibility checklist for source 
documentation if it has been reviewed, signed, and dated before registration by the study 
investigator. If used as source documentation, this checklist must be printed, the 
investigator must check each item to document their assessment that the participant meets 
each eligibility criterion, and the completed checklist must be maintained in the 
participant’s chart. All questions regarding eligibility should be directed to the study chair. 
Participant ID Number: 097 - ___ ___ ___ - ___ ___ ___ 
Patient’s Initials (L, F, M): ___ ___ ___ 
3.1 
Eligibility Criteria 
3.1.1 Inclusion criteria associated with type and status of lymphoma, one of the following 
must be applicable: 
____ 3.1.1.1 Biopsy-proven intermediate or high-grade non-Hodgkin’s lymphoma, 
meeting one of the following criteria (timeline 8 months prior to 
enrollment in the screening segment*): 
• In partial remission, 
• Relapsed after initial complete remission, 
• Failed induction therapy, but responds to salvage therapy (i.e., 
chemosensitive disease), 
• In complete remission with high-risk features as specified by the 
International Prognostic Index (See Appendix VIII). 
____ 3.1.1.2 Biopsy-proven advanced stage follicular lymphoma, that have failed at 
least two lines of therapy multi-agent chemotherapy, but responds to 

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salvage therapy (i.e., chemosensitive disease) (timeline 8 months prior to 
enrollment in the screening segment*). 
____ 3.1.1.3 Biopsy-proven advanced stage Mantle cell lymphoma with Ki-67 > 10% 
in first complete remission (timeline 8 months prior to enrollment in the 
screening segment*). 
____ 3.1.1.4 Biopsy-proven Hodgkin’s lymphoma, meeting one of the following 
criteria (timeline 8 months prior to enrollment*). 
• In first, or greater relapse after initial complete remission, 
• In partial remission, 
• Failed induction therapy, but responds to salvage therapy (i.e., 
chemosensitive disease). 
____ 3.1.1.5 Biopsy-proven Burkitt’s lymphoma, meeting one of the following criteria 
(timeline 8 months prior to enrollment*): 
• In second complete remission after relapse following initial complete 
remission, 
• Failed induction therapy, but responds (very good partial remission, 
complete remission, or near complete remission) to salvage therapy 
(i.e., chemosensitive disease). 
____ 3.1.1.6 Biopsy proven plasmablastic lymphomas, or peripheral T cell lymphoma 
(with the exception of ALK+ type in first or second complete remission) 
(timeline 8 months prior to enrollment*). 
*NOTE: Patients meeting the following criteria are exempt from the 8-
month timeline and do not require additional biopsy: 
• Patients who have never achieved a complete remission on the last 
biopsy-proven site of disease and went on to the next therapy then 
achieved a complete remission. 
• Patients who relapsed quickly (within 3 months of their last 
chemotherapy) and now have achieved a complete remission with 
salvage therapy. 
3.1.2 Inclusion criteria associated with HIV-1 status 
____ 3.1.2.1 HIV-1 infection, as documented by any federally approved, licensed HIV 
rapid test performed in conjunction with screening (or ELISA, test kit, 
and confirmed by Western blot or other approved test). Alternatively, this 
documentation may include a record demonstrating that another physician 
has documented the participant's HIV status based on either: 1) approved 
diagnostic tests, or 2) the referring physician's written record that HIV 
infection was documented, with supporting information on the 
participant's relevant medical history and/or current management of HIV 
infection. 
____ 3.1.2.2 Must be on a multi-drug anti-HIV regimen (excluding zidovudine [AZT, 

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ZDV, Retrovir®, or agents containing zidovudine (e.g., Combivir® and 
Trizivir®)], and efavirenz [Sustiva®, or agents containing efavirenz (e.g., 
Atripla®)]). 
 
 
Participants on zidovudine [AZT, ZDV, Retrovir®; including Combivir® 
and Trizivir®] and efazirenz [Sustiva®; including Atripla®] must switch to 
an alternative regimen without anticipated drug-drug interactions or 
myelosuppressive properties based on known viral resistance patterns 
and/or ART history, such as raltegravir and Truvada (emtricitabine and 
tenofovir) at least two weeks prior to the transplant. 
_____ 3.1.2.3 Participant taking ARTs must satisfy one of the following: 
• Undetectable HIV viral load (< 50 copies/mL). For patients who have 
had negative viral loads in the past 6 months and no known HIV viral 
load >500 copies/mL within the last 6 months, minor fluctuations of 
viral load (isolated escalations up to 500 copies/mL) are acceptable. 
The participant’s history of negative viral loads may be documented 
with recent laboratory results and/or a record from the participant’s 
HIV care provider. 
• If viral load is detectable at < 2000 copies/mL a review of previous 
antiretroviral regimens or previous genotypic or phenotypic testing 
which indicate the ability to fully suppress virus by addition of 
sensitive drugs must be performed. This review will be carried out by 
the protocol ID team or the ID specialist caring for the patient. 
• If viral load is detectable at ≥ 2000 copies/mL, a current HIV genotype 
and/or phenotype must be obtained. If a HAART regimen to which 
the patient’s virus is sensitive can be determined based on genotype 
and previous antiretroviral experience, then the patient will be 
considered eligible in this regard. This review will be carried out by 
the protocol ID team or the ID specialist caring for the patient. 
3.1.3 General Inclusion Criteria (timeline: within 8 weeks prior to enrollment in the 
screening segment, unless otherwise specified) 
____ 3.1.3.1 Karnofsky performance status of 70-100%. ECOG performance status < 
[2] (see Appendix II) 
____ 3.1.3.2 SGOT and SGPT ≤ 2.5 times upper limit of normal (ULN). Serum 
bilirubin ≤ 2.5 times ULN except for participants who are on atazanavir 
or indinavir, or with elevated indirect bilirubin related to bilirubin 
conjugation issues such as Gilbert’s disease, provided that the 
participant’s direct bilirubin is within normal institutional limits. 
____ 3.1.3.3 Participants who are hepatitis C virus antibody positive, or hepatitis B 
virus surface antigen positive must be free of clinical evidence of cirrhosis 
as determined by the principal investigator in consultation with the 
institutional Gastroenterology Service. Timeline: within 3 weeks prior to 
enrollment. 

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____ 3.1.3.4 Participants with Hepatitis B should be on appropriate anti-viral therapy 
at the time of the transplant, and their viral load should be negative. 
Timeline: within 3 weeks prior to enrollment. 
____ 3.1.3.5 Serum creatinine ≤ 2 times ULN. Timeline: within 3 weeks prior to 
enrollment. 
____ 3.1.3.6 Creatinine clearance ≥ 60 mL/min by the modified Cockcroft-Gault 
Formula. Timeline: within 3 weeks prior to enrollment. 
____ 3.1.3.7 PT/PTT ≤ 2 times upper limit of normal (ULN), or international 
normalized ratio (INR)/PTT ≤ 2 times the ULN. Timeline: within 3 weeks 
prior to enrollment. 
____ 3.1.3.8 FEV-1 or DLCO (corrected for hemoglobin) ≥ 50% predicted. Timeline: 
within 4 weeks prior to enrollment. 
____ 3.1.3.9 LVEF ≥ 50% by 2D ECHO or MUGA scan. Timeline: within 4 weeks 
prior to enrollment. 
____ 3.1.3.10 Not pregnant or nursing, with negative serum pregnancy test. Pregnant 
women are excluded from this study because the conditioning regimen 
has the potential for teratogenic or abortifacient effects. Because there is 
an unknown but potential risk for adverse events in nursing infants 
secondary to treatment of the mother with BEAM, breastfeeding should 
be discontinued. These potential risks may also apply to other agents used 
in this study. Timeline: within 3 weeks prior to enrollment. 
____ 3.1.3.11 Participants should agree to practice effective contraceptive precautions 
and to use at least one method of contraception for the duration of the 
study and for 3 months post-transplant. 
____ 3.1.3.12 Age ≥18 years. Because only adult transplant centers are participating as 
study sites. 
____ 3.1.3.13 Life expectancy of greater than 3 months. 
____ 3.1.3.14 Ability to understand and the willingness to sign a written informed 
consent document. 
____ 3.1.3.15 Receipt of a stable ART regimen for at least 3 weeks prior to enrollment. 
3.2 
Exclusion Criteria 
Participants who do not fulfill the criteria as listed in Section 3.1 above, are ineligible. 
Additionally, the presence of any of the following conditions will exclude a participant 
from study enrollment (timeline for all the exclusion criteria is within 8 weeks prior to 
enrollment in the screening segment): 
____ 3.2.1 Participants with > 5% involvement of bone marrow by malignant cells (either by 
manual count or flow cytometry) prior to stem cell collection. 
____ 3.2.2 Participants with any abnormal cytogenetics in the bone marrow not related to the 
lymphoma, and not deemed to be constitutional. 
____ 3.2.3 Participants with unexplained anemia and/or thrombocytopenia. 

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____ 3.2.4 Participants 
with 
clear 
evidence 
of 
myeloproliferative 
disorders, 
or 
myelodysplastic disorders in the marrow. 
____ 3.2.5 Presence of any active CNS disease at the time of evaluation (parenchymal or 
leptomeningeal). 
____ 3.2.6 Any history of HIV-1 associated encephalopathy. 
____ 3.2.7 Participants with persistently low CD4 counts less than 200 and a history of any 
AIDS-defining infection in the last 6 months before screening are excluded from 
the study. 
____ 3.2.8 Symptomatic/active bacterial, or fungal, or any other opportunistic infection. 
____ 3.2.9 Active CMV retinitis, or other active CMV-related organ dysfunction. 
____ 3.2.10 Relapse of pneumocystis carinii pneumonia within the past year before enrollment. 
____ 3.2.11 Intractable, severe diarrhea, defined as > 1.500 cc diarrheal fluid per day, or 
diarrhea causing persistent severe electrolyte abnormalities, or hypoalbuminemia. 
____ 3.2.12 History of active myocardial ischemia, cardiomyopathy, uncontrolled dysrhythmia, 
or congestive heart failure within the last 6 months before enrollment. 
____ 3.2.13 Dementia of any kind. 
____ 3.2.14 Seizures within the past 12 months before enrollment. 
____ 3.2.15 History of Grade III hemorrhagic cystitis due to prior cyclophosphamide 
chemotherapy. 
____ 3.2.16 History of other prior malignancy, except squamous cell carcinoma of the cervix or 
anus, superficial basal cell or squamous cell skin cancer, or other malignancy 
curatively treated more than 5 years ago before enrollment. 
____ 3.2.17 Active psychosocial condition that would hinder study compliance and follow-up. 
____ 3.2.18 Any perceived inability to directly (and without the means of a legal guardian) 
provide informed consent. 
____ 3.2.19 Any medical or physical contraindication, or other inability to undergo HPC 
collection. 
____ 3.2.20 Participants who are receiving any other investigational agents. 
____ 3.2.21 Uncontrolled intercurrent illness including, but not limited to, ongoing or active 
infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac 
arrhythmia, or psychiatric illness/social situations that would limit compliance with 
study requirements. 
Physician Signature: ______________________________ Date: ___________________ 
(Optional unless this section is used as an eligibility checklist) 
3.3 
Number of Participants to be Enrolled 
3.3.1 Proposed sample size 
This study will enroll a minimum of 2 participants and a maximum of 18 

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participants. 
3.3.2 Accrual rate 
Approximately 1 participant per month. 
3.4 
Recruitment Methods 
Participants will be recruited from UC Davis, AIDS Malignancy Consortium (AMC) sites 
as well as from other referring clinics (throughout California and beyond) and will be 
enrolled and treated on protocol at either UC Davis or AMC sites. Recruitment is open to 
all minorities and both genders. Although distributions may vary by disease type, our 
recruitment procedures have been developed to enroll participants who are representative 
of the respective target population. Screening of participants will be done by PIs as well as 
other members of the transplant team at either institution. 
All procedures will be performed at the UC Davis Cancer Center, UCSF Adult Hematology 
and BMT Clinic, the UCSD Moores Cancer Center, and Memorial Sloan Kettering Cancer 
Center. The UC Davis IRB, UCSF Committee on Human Research, the UCSD Human 
Research Protections Program, and the Memorial Sloan Kettering Cancer Center 
Institutional Review Board/Privacy Board will oversee study conduct at their respective 
sites. After the first 3 participants are recruited to the protocol and dependent on the pace 
of participant recruitment to the protocol, a decision will be made by principal investigator 
and in consultation with AMC Steering Committee to extend the recruitment to other AMC 
sites. 
Since it would not be possible to contact all of the participants associated with the study to 
obtain consent, the lead site (UC Davis) is requesting a waiver of HIPAA Authorization 
for recruitment purposes only. The PIs/research team will identify potential participants 
while at clinical appointments and access medical records to verify eligibility prior to the 
treating physician and research coordinator approaching the potential participant. Consent 
to participate and HIPAA Authorization to access additional information in the medical 
records will be obtained prior to enrollment into the study. For a description of the Cancer 
Center’s plan to safeguard the Protected Health Information (PHI) from improper use and 
disclosure as well as assurance that the PHI will not be inappropriately reused or disclosed 
to any other person or entity (See Section 11.3). 
All the information disseminated to participants (handouts, brochures, etc.) and any 
advertisements will be first approved by the IRB for the site. Screening requirements 
outlined in Section 7.1 and Appendix I will be used to meet inclusion or exclusion criteria 
that are defined for the study (see Section 3.1 and 3.2). Participants will be informed of 
their eligibility only by their treating physicians. Only participants who have signed all 
appropriate documentation and who meet participant eligibility requirements will be 
enrolled. We are completely aware of the difficulties in recruiting participants with this 
rather rare condition (i.e., HIV-1 related relapsed lymphoma). Therefore, we have proposed 
an ambitious effort to enroll participants not only from our catchment area of Northern 
California, but also from other areas of California, as well as from other states. Accrual 
will be facilitated by a dedicated community advocate, who will initiate and retain 
interactions with the target community and provide education about the trial. This advocate 
will interact with clinical research centers as well as other referring clinics across 

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California. He/she will use all available communication tools and specifically the internet 
to identify and reach out to the target populations for this study through support groups or 
specific websites, as community outreach materials and methods are specifically approved 
by the reviewing IRB. All information intended for distribution on the internet or any other 
media for the purpose of advertising for the study will be approved by the IRB in advance. 
We have already established a network of collaborators throughout the country and have 
received verbal and written commitment from some of these centers to propose the study 
to eligible participants and to facilitate the transfer of their care to our study centers. Entry 
criteria for cancer studies will be disease, not gender- or ethnicity-specific. Although, 
participants under 18 and pregnant or nursing women will not be eligible. Information will 
be disseminated to participants and physicians (e.g., handouts, brochures, etc.), and any 
advertisement or instruction will be approved by the IRB for the site. 
3.5 
Participant Enrollment Procedures 
Sites must have this protocol approved by their Institutional Review Boards (IRB) and 
Institutional BioSafety Committee (IBC) and be registered for study participation with the 
AMC Operations and Data Management Center (ODMC) before they may enroll 
participants. 
3.5.1 Registration for screening 
After an informed consent form has been signed by the participant, the participant 
must be registered for screening (AMC-097, Segment A) on-line via 
AdvantageEDCSM. After successful registration into screening, the participant will 
receive an eleven-digit participant ID and will then enter the screening process 
(Screening and Pre-entry visits). 
3.5.2 Enrollment 
After the screening evaluations have been obtained and the participant is 
determined to be eligible, the participating site will complete the protocol-specific 
eligibility checklist. Sites should email the completed (signed by site PI) 
eligibility 
checklist 
to 
mabedi@ucdavis.edu 
and 
hs-
CellularTherapyResearch@ucdavis.edu for approval by the protocol chair (or 
a designated sub-investigator at UC Davis) before enrolling the participant 
into AMC-097 Segment B (on-line via AdvantageEDC). Enrollment should 
occur no more than 1 week prior to, but before stem cell collection (enrollment 1 
day prior to or on the day of stem cell collection is strongly encouraged). There 
should also be a minimum of 2 weeks from the last day of any chemotherapy 
(except the one used for stem cell mobilization) before a participant can be enrolled 
on this trial. Once the eligibility checklist is submitted, a system generated 
confirmation email will be sent to the enroller upon successful completion of the 
participant enrollment. If the on-line system is inaccessible, the site should notify 
the AMC ODMC (via email at amcpm@emmes.com or via phone at 301-251-1161) 
for further instructions. 
Participants must be enrolled into AMC-097 Segment B prior to receiving 
HCT. 

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4.0 
TREATMENT PLAN 
Protocol agent will be administered on an inpatient basis. Reported adverse events and 
potential risks are described in Section 5.0 and Section 6.1.16. No investigational or 
commercial agents or therapies other than those described below may be administered with 
the intent to treat the participant's malignancy. 
The final product is autologous CD34+ cells transduced with a triple combination of anti-
HIV genes that will be re-suspended in participants’ serum, cryopreserved and then thawed 
and infused directly into central vein of the participants though a central catheter, 1 day 
after finishing their conditioning chemotherapy. Different number of non-transduced, un-
manipulated product will be co-infused similarly according to the study cohort. 
Autologous CD34+ cells isolated via clinical grade magnetic cell separation from 
mobilized peripheral blood apheresis products of HIV-1 lymphoma participants will be 
transduced with a 1TAX lentiviral vector carrying a triple combination of anti-HIV genes, 
tested and cryopreserved. CD34+ cell isolation, transduction, and qualification of the final 
product will be carried out in the UC Davis GMP facility following established Standard 
Operating Procedures (SOPs) and appropriate Quality Control (QC) and Quality Assurance 
(QA). The final product will be tested for identity, viability, purity, sterility, absence of 
mycoplasma and endotoxin, and for integrated transgene copy numbers per genome. A 
Certificate of Analysis (COA) will be generated (see Section 6.1). After completed testing, 
the autologous, cryopreserved product will be thawed and used for infusion into the HIV-
1 lymphoma participant who has undergone a conditioning regimen. 
4.1 
HSC Mobilization and Peripheral Stem Cell Collection 
See Section 2.3 for additional details of collection. 
Prior to stem cell mobilization, eligibility and suitability of the autologous donor will be 
confirmed based on the screening data and the individual institution guidelines. The 
infectious disease profile and the rest of the blood donor test will be performed with FDA 
licensed blood donor tests. 
In all patients, ART will be withheld 3 days prior and during stem cell collection. This is 
to decrease the possibility of ART therapy to interfere with the transduction of stem cells 
before transplant. 
The stem cell mobilization regimen will be based on the individual participant’s need, 
decided by the transplant physician. Both chemo mobilization and/or growth factor 
mobilization is allowed. HSC/progenitor cells will be collected by participating 
institutions, according to the institutional standards. 
The quality, as well as the quantity, of stem/progenitor cells may be different in each 
collection. While it is difficult to assess the quality of stem cells, their quantity can be easily 
measured by the number of CD34 cells at the end of each day collection.  
We aim to collect a minimum of 5 x 106 CD34 cells/kg of body weight of the recipient 
(ideally 10 x 106 CD34 cells/kg). 
Assignment of the mobilized stem cells for the transduced group and the non-transduced 
group will be done according to the guidelines below: 

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1. Minimum number of required unmanipulated cells will be collected first (1.5 x 106 
CD34 cells/kg of recipient) and will be immediately frozen and stored in harvesting 
center Progenitor Laboratories as per site SOP. 
2. Additional cells available will be assigned to transduced portion of stem cell collection, 
which will be transferred to the UC Davis GMP facility by courier in temperature 
controlled transport containers, undergo CD34+ cell selection and transduction culture, 
followed by freezing and storage in the GMP facility. We prefer to receive the samples 
on the same day but they can be held for up to 24 hours and shipped with the next day 
collection based on the communications of the transplant physicians in the collection 
facility and the PI or Co-PI of the study. 
Stem cell collection goals: Ideally, we aim to collect 10 x 106 CD34+ cells/kg of body 
weight of the recipient (for both manipulated and unmanipulated product). A minimum of 
5 x 106 CD34+ cells/kg of body weight of the recipient is needed to proceed with the study.  
1.5 x 106 CD34 cells/kg of body weight of the recipient will be used as the unmanipulated 
product and will be frozen. The rest of the product will be used for stem cell transduction. 
Additional cells can be collected and stored after the target number for CD34 (10 x 106 
CD34+ cells/kg) is achieved based on the discretion of the subject’s transplant physician 
and in consultation with the study principal investigator. These cells can be given to the 
patient, but the ratio of total manipulated to unmanipulated cells needs to follow the 
guidelines in Table 4-A. 
The collection will continue until we achieve our collection targets. Table 4-Abelow 
provides the dose of transduced versus untransduced cells in each cohort of the study. 
 
Table 4-A: Dose of Transduced Versus Untransduced Cells  
Cohort Level 
Ratio of Transduced 
Versus Untransduced 
Stem Cells. 
Minimum Number of 
Transduced/ 
Untransduced Cells 
Acceptable. CD34 
cells/kg 
Range for Number of 
Transduced Cells / 
Unmanipulated Cells. 
CD34 cells/kg 
1 
1:1 
1 X 106:1 X 106 
1 to 5 X 106: 1 to 5 X 106 
2 
5:1 
2 X 106:1 X 106 
2 to 5 X 106:1 X 106 
3 
1:0 
2 X 106: 0 
2 to 10 X 106: 0 
3+ expansion 
1.0 
3 x106 : 0 
3 to 10 X 106: 0 
 
4.2 
Agent Administration 
4.2.1 Preparative regimen for transplant 
All participants enrolled in this protocol will be hospitalized, in all clinical centers, 
in accordance with the procedures for recipients of HSCT as defined by the treating 
institutions. They will receive BEAM or R-BEAM regimen as a standard of care 
preparative regimen administered per institutional guidelines. Physicians may use 

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their discretion and institutional guidelines in determining the appropriate body 
weight (actual, adjusted, or ideal) for the conditioning regimen dose calculation.  
The BEAM and R-BEAM regimens for this study are comprised of the following 
intravenous medications: 
• BCNU  
• Cytarabine (Ara-C); 
• VP-16 
• Melphalan 
• Participants with B cell Lymphoma may be administered rituximab prior to 
chemotherapy and post-transplant. Biosimilar agents are permitted at 
the investigator’s discretion. 
4.2.2 Stem cell infusion 
For the first 2 cohorts, both transduced and un-manipulated product will be infused 
within 4 to 24 hours of each other at day 0, intravenously: 
• The transduced product will be transported from the GMP facility of UC Davis, 
stored at the BMT unit of participating facilities and thawed in the BMT unit 
and immediately infused into the participants. 
o The stem cell lab at participating facilities will be receiving the frozen 
product via overnight courier, in a temperature-controlled transport 
container, prior to the start of the conditioning regimen. 
• The un-transduced product will be stored at the participating facilities stem cell 
laboratories, thawed in the BMT unit and immediately infused into the 
participants, 4 to 24 hours after the infusion of transduced product is concluded. 
Again, both procedures, including the route and duration of infusion, will be in 
accordance with the procedures for recipients of HCT as defined by the treating 
institution. 
After DSMB approval, Participants in the third cohort will be administered only 
transduced product. 
All the sites will follow site SOPs for thawing and infusing the transduced product, 
which will be coordinated before the start of the trial. 
In case of non-engraftment, the back-up product stored at the respective site will be 
infused. 
The number of cells infused to each participant depends on the cohort that the 
participant is enrolled in and is mentioned in Table 4-A above. The number of cells 
in the transduced products is based on CD34 counts after transduction of the stem 
cells and before freezing. 
4.2.3 Antibiotic prophylaxis 
All participants during transplant will receive antiviral (acyclovir or valacyclovir), 
antifungal (fluconazole) and anti-Pneumocystis jiroveci prophylaxis based on each 

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institution guidelines/preference. Antiviral and anti-Pneumocystis jiroveci 
prophylaxis will be continued for a minimum of one year and 6 months, 
respectively, after transplant. Antifungal prophylaxis will be continued for 30 days 
post-transplant. All prophylactic therapy will be restarted during ART withdrawal. 
4.2.4 Antiretroviral therapy 
Antiretroviral therapy will be stopped during transplant only for participants with 
excessive nausea or vomiting and for participants who are not able to take pills due 
to mucositis, etc. The therapy will be resumed once the transplant team feels 
confident that the toxicities are resolved, and participant has adequate oral intake. 
Potential interaction of ART with some of the chemotherapy agents and potential 
negative effects on stem cell engraftment and marrow recovery will be avoided by 
using ART that do not have those interactions or side effects. Participants on 
zidovudine [AZT, ZDV, Retrovir®, or agents containing zidovudine (e.g., 
Combivir® and Trizivir®], and efavirenz [Sustiva©, or agents containing efavirenz 
(e.g., Atripla®)], prior to transplant will be switched at least two weeks before the 
transplant to an alternative regimen without anticipated drug-drug interactions or 
myelosuppressive properties based on known viral resistance patterns and/or ART 
history, such as raltegravir and Truvada (emtricitabine and tenofovir). 
Antiretroviral drugs with long half-lives, such as efavirenz, will be avoided post-
transplant in participants’ combination therapy; if a drug regimen containing a long 
half-life drug is stopped, only a single drug will remain in the system for a 
prolonged period of time, and drug-resistant variants of HIV-1 can emerge very 
quickly. 
4.3 
Definition of Dose-Limiting Toxicity 
Dose limiting toxicity in the trial is defined as lack of timely engraftment (the collective 
establishment of a persistent absolute neutrophil count of at least 500/mm3 and platelet 
count of at least 20,000/mm3 for 3 consecutive laboratory values obtained on different 
days) within one month post-transplant, or any grade 3 and 4 non-hematopoietic organ 
toxicity that can be attributed (possibly, probably, or definitely) to lentiviral transduced 
stem cell transplant, excluding alopecia, or any evidence of clonal expansion and excluding 
expected toxicities that are associated with the pre-transplant conditioning regimen, 
regardless of grade (see Appendix X for full list). 
Dose escalation will proceed within each cohort according to the following scheme. Dose-
limiting toxicity (DLT) is defined above. 
Table 4-B: Criteria for dose escalation 
Number of Participants with 
DLT at a Given Dose Level 
Escalation Decision Rule 
0 out of 3 
Enter 3 participants at the next dose level. 
≥ 2 
If this threshold is exceeded in cohorts 1 or 2, dose 
escalation will be stopped. This dose level will be 
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Number of Participants with 
DLT at a Given Dose Level 
Escalation Decision Rule 
(highest dose administered).   
If 2 participants in the 3rd cohort develop DLT due 
to insufficient engraftment, an additional 3 
patients will be enrolled in the cohort 3+ with an 
increase in minimum number of transduced CD34 
cells from 2 to 3.0 X 106 cells/kg of recipient 
weight. 
If 2 or more DLTs occur in cohort 3+, the trial 
will be stopped.  
1 out of 3 
Enter at least 3 more participants at this dose 
level. 
If 0 of these 3 participants experience DLT, 
proceed to the next dose level. 
If 1 or more of this expansion group suffer DLT, 
then dose escalation is stopped, and this dose is 
declared the maximally administered dose. Three 
(3) additional participants will be entered at the 
next lowest dose level if only 3 participants were 
treated previously at that dose. 
≤ 1 out of 6 at highest dose 
level at or below the maximally 
administered dose 
This is generally the recommended phase 2 dose. 
At least 6 participants must be entered at the 
recommended phase 2 dose. 
4.4 
General Concomitant Medication and Supportive Care Guidelines 
All concomitant medication, including blood products or general supportive guidelines, 
will be addressed by treating physicians and based on the institutional guidelines. 
Section 4.2.4 explains the details of anti-retroviral therapy (ART) during the study period 
and in the follow up period. 
All medications must be reported in the site’s source documents from the time of study 
enrollment in Segment A through the treatment discontinuation visit. All medications will 
be reviewed and updated in the site’s source documents at all visits where indicated in the 
schedule of evaluations. Only a subset of medications will be reported in the Concomitant 
Medications CRF as listed below: 
• Anti-retroviral 
• Antiviral 
• Growth colony stimulation factors (GCSF) 
4.5 
Duration of Therapy 
The investigational part of this clinical trial (i.e., infusion of gene modified hematopoietic 

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stem cells) will occur only once after conditioning regimen administration is concluded. 
4.6 
Duration of Follow Up 
The initial follow-up period for this clinical trial will be two years post-transplant. For 
practical reasons, close and complete follow up of the participants needs to be limited. We 
have decided to use the 2-year limit for the close clinical follow-up since majority of the 
participants post an autologous transplant have recovered their immune system, and they 
are in a less risk of opportunistic infections as well as disease relapse after 2 years. 
However, all participants will be followed for 15 years albeit, in a less frequent and 
intensive form (every 12 months for years 3-15) for safety, as mandated by FDA for all 
gene therapy study participants. Additionally, every 12 months for years 3-15 we will 
request optional blood collections to monitor the patient’s HIV and vector parameters in 
the body for participants that consent to optional studies. 
4.7 
Withdrawal of Participants 
Participants will complete protocol treatment with HCT. Following HCT, participants may 
be withdrawn from the study prior to expected completion of that participant voluntarily 
or by their physician(s) for safety reasons, failure of the participant to adhere to protocol 
requirements, participant consent withdrawal. Patients who have progression of lymphoma 
within 24 months of gene therapy infusion will be followed for correlative studies as 
outlined in section 7.5.8.1. The goal is to monitor the patient’s HIV and vector parameters 
in the body.  
If early termination of the study should occur for any of the above-stated reasons, the study 
doctor(s) will have a discussion with the participant on how to stop safely, such as 
evaluation for risks from the study drugs, follow-up care, and any other procedures that 
might be recommended. 
Even though participants may be withdrawn prematurely from the study, it is imperative 
to collect at least survival data on such participants throughout the protocol defined follow-
up period for that participant (though careful thought should be given to the full data set 
that should to be collected on such participants to fully support the analysis). Such data is 
important to the integrity of the final study analysis since early withdrawal could be related 
to the safety profile of the study drug. If a participant withdraws consent to participate in 
the study, attempts should be made to obtain permission to record at least survival data up 
to the protocol-described end of the participant follow-up period. 

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5.0 
ADVERSE EVENTS: LIST AND REPORTING REQUIREMENTS 
Adverse event (AE) monitoring and reporting is a routine part of every clinical trial. The 
following list of AEs (Section 5.1) and the characteristics of an observed AE (Section 5.2) 
will determine whether the event requires expedited reporting via the CTEP Adverse Event 
Reporting System (CTEP-AERS) in addition to routine reporting (via AdvantageEDCSM). 
CTCAE term (AE description) and grade: The descriptions and grading scales found in the 
revised NCI Common Terminology Criteria for Adverse Events (CTCAE) version 5.0 will 
be utilized for AE reporting beginning April 1, 2018. All appropriate treatment areas should 
have access to a copy of the CTCAE version 5.0. A copy of the CTCAE version 5.0 can be 
downloaded from the CTEP web site  
http://ctep.cancer.gov/protocolDevelopment/electronic_applications/ctc.htm.  
This study will be monitored by the Clinical Data Update System (CDUS). Cumulative 
CDUS data will be submitted quarterly to CTEP by electronic means. Reports are due 
January 31, April 30, July 31, and October 31. 
5.1 
Comprehensive Adverse Events and Potential Risks Lists 
5.1.1 Adverse event list for genetically modified hematopoietic stem cells 
Autologous stem cell transplant is a potentially toxic procedure and can result 
in multiple side effects. Autologous stem cell transplant is clinically indicated 
in all the participant of this study and is a part of standard of care for all these 
patients. Therefore, in this protocol we will focus on only side effects 
attributable (possibly, probably or definitely) to lentivector transduced stem 
cell transplant, excluding expected toxicities that are associated with the pre-
transplant conditioning regimen (see Appendix X for full list). The complete 
product information of commercial chemotherapeutic agents to be 
administered as part of the protocol for stem cell transplant with BEAM or R-
BEAM regimen can be found in FDA approved package insert for each agent. 
Side Effects specific to participation in this protocol include but not limited to 
(see Section 6.1.19 for additional details): 
Blood draw risks 
Bruising, infection, and minor pain or discomfort comparable to a needle prick. 
IV inserted in the vein risks 
Inflammation, pain, bruising, bleeding, or infection. 
Stem cell infusion 
Problems that may occur during stem cell infusion are usually associated with 
infusion of DMSO and include low blood pressure, lightheadedness, fainting, fever, 
chills, tachycardia, bronchospasm, and hypoxia. These are temporary effects that 
are likely to subside after the infusion. 
Allergic reactions 
Proteins made from large amounts of non-human (protein) can sometimes lead to 
future allergic reactions. There is a small amount of a modified protein in the 

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modified cells for which there is a small chance of allergic reaction, but it is unlikely 
to be harmful to you even if it occurs. If an allergic reaction occurs, it is more likely 
that the modified cells will be destroyed. After the cell infusion, blood will be tested 
for any reaction that could affect the use of these agents in the future, but this 
reaction would not be directly harmful. This reaction is estimated at about a 4% 
chance of happening. 
Possibility of another cancer or leukemia 
The lentivirus used to modify the cells does not have the genes needed to reproduce 
itself, but it is possible in the future that it could combine with other genes in the 
body, or that it could activate an unknown virus to result in a known or unknown 
medical problem or disease, including the possibility of another cancer. 
Reproductive risks 
Since the effects of the proposed treatments on a fetus are unknown, any woman 
who should become pregnant while on this study will be removed from the study, 
and another method of treatment will be suggested. If male, the participant must 
practice effective birth control and must advise their partner to contact their 
physician should they become pregnant. We request the participants use at least one 
method of contraception for 3 months post-transplant. 
Risk of antiviral therapy withdrawal 
Participants may be asked to hold on taking their anti-HIV medications on at least 
three occasions. First, for 3 days before and during stem cell collection (to avoid 
the interaction of anti-HIV medications on stem cell transduction). The second 
time, during and/or immediately after chemotherapy for transplant in participants 
who cannot tolerate oral intake due to severe nausea/vomiting or mucositis. The 
third time (only in the third cohort), sometime after the first 6 months after the 
transplant when their immune system has recovered at least to some extent 
measured by CD4 T cells, participants in the third cohort will be asked if they would 
agree to voluntarily hold their anti-HIV medications until the onset of the criteria 
that will prompt resumption of treatment (see Section 2.4.1). The rationale for the 
third withdrawal is to expand the anti-HIV-1 immune system. Each time the 
participant stops anti-HIV medications, there is a potential risk that HIV-1 infection 
can become reactivated, and suppress their immune system, and make them 
susceptible to severe and potentially life-threatening infections. Clinicians, 
including an HIV doctor, will closely monitor the participant’s immune system, and 
will restart their anti-HIV medications as soon as level of viral reactivation and 
suppression of the immune system reaches certain thresholds accepted by experts 
in the field. However, this may not completely eliminate the risk of complications. 
Unknown risks 
The experimental treatments may have side effects that no one knows about yet. 
The researchers will tell participants of new information that might make 
participants change their mind about participating in the study. 

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5.2 
Classification of AEs by Severity and Relationship to Study Drug Administration 
5.2.1 Adverse Event: Any unfavorable and unintended sign (including an abnormal 
laboratory finding), symptom or disease temporally associated with the use of a 
medical treatment or procedure regardless of whether it is considered related to the 
medical treatment or procedure (attribution of unrelated, unlikely, possible, 
probable, or definite). 
5.2.2 Life-threatening Adverse Event: Any AE that places the participant or participant, 
in view of the Investigator, at immediate risk of death from the reaction. 
5.2.3 Serious Adverse Event (SAE): Any AE occurring at any dose that results in any of 
the following outcomes: Death, a life-threatening AE, inpatient hospitalization or 
prolongation of existing hospitalization, a persistent or significant disability/ 
incapacity, or a congenital anomaly/birth defect. 
5.2.4 Please note for hospitalization – All hospitalizations (or prolongation of existing 
hospitalization) for medical events equivalent to CTCAE Grade 3, 4, 5 must be 
reported regardless of the requirements for Phase of study, expected or unexpected, 
and attribution. For example, do not report an admission for pharmacokinetic 
sampling, but do report an admission for a myocardial infarction.  
5.2.5 Toxicity: Toxicity is a term NOT clearly defined by regulatory organizations. 
Toxicity has been described as an AE that has an attribution of possibly, probably 
or definitely related to investigational treatment. To minimize confusion the NCI 
would recommend that the term toxicity NOT be utilized for AE reporting 
purposes. The CTCAE continues to use the term ‘toxicity’ because of familiarity. 
5.2.6 Unexpected Adverse Event: Any AE that is not listed in available sources including 
the package insert, the Investigator’s Brochure, or the protocol. 
5.2.7 CTEP Adverse Event Reporting System (CTEP-AERS): An electronic system for 
expedited submission of AE reports. 
5.2.8 Attribution: The determination of whether an AE is related to a medical treatment 
or procedure. Attribution categories: 
Definite – The AE is clearly related to the investigational agent. 
Probable – The AE is likely related to the investigational agent. 
Possible – The AE may be related to the investigational agent. 
Unlikely – The AE is doubtfully related to the investigational agent. 
Unrelated – The AE is clearly NOT related to the investigational agent. 
5.3 
Expedited Adverse Event Reporting 
5.3.1 Expedited AE reporting for this study must use CTEP-AERS (CTEP Adverse Event 
Reporting System), accessed via the CTEP home page (http://ctep.cancer.gov). The 
reporting procedures to be followed are presented in the “CTEP, NCI Guidelines: 
Adverse Event Reporting Requirements” which can be downloaded from the CTEP 
home page (http://ctep.cancer.gov). These requirements are briefly outlined in the 
table below (Section 5.3.3). 

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A 24-hour notification is to be made to AMC ODMC by telephone at 301-251-
1161, only when Internet connectivity is disrupted. Once Internet connectivity is 
restored, a 24-hour notification phoned in must be entered electronically into 
CTEP-AERS by the original submitter at the site. 
5.3.2 CTEP-AERS is programmed for automatic electronic distribution of reports to the 
following individuals: Study Coordinator of the Lead Organization, Principal 
Investigator, and the local treating physician. CTEP-AERS provides a copy feature 
for other e-mail recipients. 
5.3.3 Expedited Reporting Guidelines 
Use the NCI protocol number and the protocol-specific participant ID assigned 
during trial registration on all reports. 
Note: A death on study requires both routine and expedited reporting regardless of 
causality, unless as noted below. Attribution to treatment or other cause must be 
provided. 
Death due to progressive disease should be reported as Grade 5 “General 
disorders and administration site conditions - Disease Progression” under the 
system organ class (SOC) of the same name. Evidence that the death was a 
manifestation of underlying disease (e.g., radiological changes suggesting tumor 
growth or progression: clinical deterioration associated with a disease process) 
should be submitted. 
 
 

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Table 5-A: Phase 1 and Early Phase 2 Studies: Expedited Reporting Requirements for 
Adverse Events that Occur on Studies under an IND/IDE within 30 Days of the Last 
Administration of the Investigational Agent/Intervention 1, 2 
FDA REPORTING REQUIREMENTS FOR SERIOUS ADVERSE EVENTS (21 CFR Part 312) 
NOTE: Investigators MUST immediately report to the sponsor (NCI) ANY Serious Adverse Events, whether or 
not they are considered related to the investigational agent(s)/intervention (21 CFR 312.64) 
An adverse event is considered serious if it results in ANY of the following outcomes: 
• 
Death 
• 
A life-threatening adverse event  
• 
An adverse event that results in inpatient hospitalization or prolongation of existing hospitalization for ≥ 24 
hours  
• 
A persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions  
• 
A congenital anomaly/birth defect.  
Important Medical Events (IME) that may not result in death, be life threatening, or require hospitalization may be 
considered serious when, based upon medical judgment, they may jeopardize the participant or participant and may 
require medical or surgical intervention to prevent one of the outcomes listed in this definition. (FDA, 21 CFR 
312.32; ICH E2A and ICH E6). 
ALL SERIOUS adverse events that meet the above criteria MUST be immediately reported to the NCI via CTEP-
AERS within the timeframes detailed in the table below. 
Hospitalization 
Grade 1 and Grade 2 Timeframes 
Grade 3-5 Timeframes 
Resulting in Hospitalization 
≥ 24 hrs 
10 Calendar Days 
24-Hour 5 Calendar Days 
Not resulting in 
Hospitalization ≥ 24 hrs 
Not required 
Expedited AE reporting timelines are defined as: 
• 
“24-Hour; 5 Calendar Days” - The AE must initially be reported via CTEP-AERS within 24 hours of learning 
of the AE, followed by a complete expedited report within 5 calendar days of the initial 24-hour report. 
• 
“10 Calendar Days” - A complete expedited report on the AE must be submitted within 10 calendar days of 
learning of the AE. 
1Serious adverse events that occur more than 30 days after the last administration of investigational 
agent/intervention and have an attribution of possible, probable, or definite require reporting as follows: 
Expedited 24-hour notification followed by complete report within 5 calendar days for: 
All Grade 3, 4, and Grade 5 AEs 
Expedited 10 calendar day reports for: 
Grade 2 AEs resulting in hospitalization or prolongation of hospitalization 
2 For studies using PET or SPECT IND agents, the AE reporting period is limited to 10 radioactive half-lives, 
rounded UP to the nearest whole day, after the agent/intervention was last administered. Footnote “1” above applies 
after this reporting period. 
Effective Date: May 5, 2011 

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5.3.4 SAE reporting to FDA and IND sponsor-investigator 
All SAEs that occur in trial participants following treatment initiation require a 
report to IND sponsor-investigator within one working day of awareness of the 
event, or immediately if the events are life-threatening or fatal. Participating 
institutions will make this report by submitting the Adverse Event Form in 
AdvantageEDCSM. The AMC ODMC will manage all SAE report submissions to 
the IND sponsor-investigator in accordance with its agreement with the IND 
sponsor-investigator, using the applicable forms designated for this purpose. 
The sponsor-investigator will be responsible for reporting all serious and 
unexpected suspected adverse drug experiences to the FDA for the IND for this 
trial. 
Participating study sites should NOT report SAEs to the FDA. Rather, participating 
sites should report SAEs to the AMC ODMC, and the sponsor-investigator or his 
designee will be responsible for reporting to FDA. 
5.3.4.1 The study sponsor is required to report certain study events in an 
expedited fashion to the FDA. These written notifications of adverse 
events are referred to as IND safety reports. 
The following describes the sponsor safety reporting requirements by 
timeline for reporting and associated type of event: 
Within 7 calendar days 
Any study event that is: 
• associated with the use of the study drug 
• unexpected, 
• fatal or life-threatening 
Within 15 calendar days 
Any study event that is: 
• associated with the use of the study drug, 
• unexpected, and 
• serious, but not fatal or life-threatening 
-or- 
• A previous adverse event that was not initially deemed 
reportable but is later found to fit the criteria for reporting 
(reporting within 15 calendar days from when event was deemed 
reportable). 
Any finding from tests in laboratory animals that: 
• suggests a significant risk for human participants including 
reports of mutagenicity, teratogenicity, or carcinogenicity. 
5.3.4.2 Additional reporting requirements 

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Sponsors are also required to identify in IND safety reports all previous 
reports concerning similar adverse events and to analyze the significance 
of the current event in light of the previous reports. 
5.3.4.3 Reporting process 
Adverse events may be submitted on FDA Form 3500A, or in a narrative 
format. If supplied in a narrative format, the minimum information to be 
supplied is noted above. 
5.4 
Routine Adverse Event Reporting 
All AEs that require expedited reporting via CTEP-AERS must also be reported in routine 
study data submissions (Adverse Event CRF). Routine reporting of all AEs attributed 
(possible, probable or definite) to lentivector transduced stem cell transplant, regardless of 
grade, must be reported on the Adverse Event CRF. Expected toxicities that are associated 
with the pre-transplant conditioning regimen (see Appendix X for full list) do not require 
routine reporting. All grade 3 or greater AEs, whether or not attributable to lentivector 
transduced stem cell transplant, will be recorded on the Adverse Event CRF.  
Reporting of AEs must begin at the time of stem cell collection. 
5.5 
Clinical Laboratory Results 
Clinical laboratory results that are outside of the institution’s reference ranges must be 
reported as adverse events if they are deemed clinically significant by the investigator(s). 
5.5.1 A clinical laboratory abnormality should be deemed clinically significant if any one 
of the following conditions is met: 
• Is not an erroneous result as proven by repeat testing, if performed, within 48 
hours. 
• The laboratory abnormality is directly attributable to the lentivector transduced 
stem cell transplant, excluding expected toxicities that are associated with the 
pre-transplant conditioning regimen (see Appendix X for full list). 
• The abnormality suggests a disease and/or organ toxicity that is new or has 
worsened from baseline. 
• The abnormality is of a degree that requires additional active management, e.g., 
change of dose, discontinuation of the drug(s), close observation, more frequent 
follow-up assessments, or further diagnostic investigation. 
• Is associated with a serious adverse event or is otherwise judged by the 
Investigator to be of significant clinical impact. 
In general, a laboratory abnormality that is not clinically significant will be 
consistent with CTCAE grade 1 (mild) or 2 (moderate) severity, as categorized by 
the relevant severity description in the Investigations System Organ Class (SOC) 
or Metabolism and Nutrition Disorders SOC. Investigators must designate 
laboratory abnormalities that are consistent with grade 3 or greater severity as 
clinically significant. 
5.6 
Secondary Malignancy 

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A secondary malignancy is a cancer caused by treatment for a previous malignancy (e.g., 
treatment with investigational agent/intervention, radiation or chemotherapy). A secondary 
malignancy is not considered a metastasis of the initial neoplasm. 
CTEP requires all secondary malignancies that occur following treatment with an agent 
under an NCI IND/IDE be reported expeditiously via CTEP-AERS. Three options are 
available to describe the event: 
• Leukemia secondary to oncology chemotherapy (e.g., acute myelocytic leukemia 
[AML]) 
• Myelodysplastic syndrome (MDS) 
• Treatment-related secondary malignancy 
Any malignancy possibly related to cancer treatment (including AML/MDS) should also 
be reported via the routine reporting mechanisms outlined in each protocol. 
5.7 
Second Malignancy 
A second malignancy is one unrelated to the treatment of a prior malignancy (and is NOT 
a metastasis from the initial malignancy). Second malignancies require ONLY routine 
adverse event reporting via CDUS unless otherwise specified. 

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6.0 
PHARMACEUTICAL INFORMATION 
A list of the adverse events and potential risks associated with the investigational or 
commercial agents administered in this study can be found in Section 5.1. The complete 
product information of commercial chemotherapeutic agents to be administered as part of 
the protocol for stem cell transplant with BEAM or R-BEAM regimen can be found in 
FDA approved package insert for each agent. 
6.1 
Autologous CD34+ Cells Transduced with a Pre-selective Marker and Triple 
Combination of Anti-HIV Genes, IND Agent (NSC # 784725) 
Autologous CD34+ cells transduced with a pre-selective marker and triple combination of 
anti-HIV genes is an investigational agent supplied to AMC investigators by GMP facility 
at UC Davis Medical Center. 
6.1.1 Description 
The final product is autologous CD34+ cells transduced with a triple combination 
of anti-HIV genes that will be re-suspended in participants’ serum, cryopreserved 
and then thawed and infused directly into central vein of the participant though a 
central catheter, 1 day after finishing their conditioning chemotherapy. Different 
number of non-transduced, unmanipulated product will be co-infused similarly 
according to the study cohort. 
Autologous CD34+ cells isolated via clinical grade magnetic cell separation from 
mobilized peripheral blood apheresis products of HIV-1 lymphoma will be 
transduced with a 1TAX lentiviral vector carrying a triple combination of anti-HIV 
genes, tested and cryopreserved. CD34+ cell isolation, transduction and 
qualification of the final product will be carried out in the UC Davis GMP facility 
following established Standard Operating Procedures (SOPs) and appropriate 
Quality Control (QC) and Quality Assurance (QA). The final product will be tested 
for identity, viability, purity, sterility, absence of mycoplasma and endotoxin, and 
for integrated transgene copy numbers per genome. A Certificate of Analysis 
(COA) will be generated. After completed testing, the autologous, cryopreserved 
product will be thawed and used for infusion into the HIV-1 lymphoma participant 
who has undergone a conditioning regimen. 
6.1.2 GMP manufacturing, transduction, cryostorage and qualification of autologous 
CD34+ cells 
All steps are controlled by Standard Operating Procedures (SOPs), overseen by QC 
and QA and are performed in a state of the art GMP facility at UC Davis Medical 
Center by qualified personnel. 
6.1.3 The GMP manufacturing process of the cellular product consists of 7 phases 
1. Receipt of donor apheresis product. 
2. CD34+ cell selection. 
3. In process testing (cell number, CD34+ analysis, viability). 
4. Overnight transduction of the isolated CD34+ cells on a fibronectin matrix in 
the presence of cytokines (SCF, Flt-3 and TPO). 

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5. Harvesting of the transduced cells. 
6. Freezing and storage of the transplantable, final product. 
7. Qualification of the final product and generation of a COA (cell number, 
viability, sterility, endotoxin, mycoplasma, number of vector integrants per 
genome). 
6.1.4 Transfer from the manufacturing facility to the clinical site 
The fresh product will be transported to the AMC clinical sites. A validated 
transport container (Liquid Nitrogen, LN2 dry vapor shipper) will be used that can 
maintain the LN2 vapor phase temperature for at least 48 hours. Transport to the 
UC Davis Medical Center clinical site only takes 15 minutes. Transfer from the 
other sites throughout California will be done by a courier and usually within the 
same day. The cells will be thawed according to standard bone marrow transplant 
procedure SOPs and will be infused into the participant. Samples will be tested for 
viability. A final COA will be generated. 
6.1.5 Plasmid production 
Plasmid release tests: Identity (restriction enzyme digest), purity (Limulus 
amebocyte lysate (LAL) endotoxin assay), sequence (DNA sequencing). 
Acceptance criteria: Confirmed identity (restriction enzyme digest with readout on 
gel, bands at appropriate positions), confirmed purity (endotoxin level below 
acceptable limit), and DNA base pair sequence of plasmid identical to required 
sequence. 
6.1.6 Vector manufacturing 
Vector release tests: Sterility (USP 14-day sterility according to 21CFR 610.12), 
LAL endotoxin assay, mycoplasma PCR, RCL (susceptible cell / cell line 
transductions), sequence of transferred genes (DNA sequencing), contaminating 
DNA (VSV-g DNA PCR). 
Acceptance criteria: Sterility (USP 14 day sterility according to 21CFR 610.12) – 
no organisms seen, LAL endotoxin level below acceptable limit (less than 0.5 
EU/mL), mycoplasma not detected by PCR, RCL not detected by HIV-1 p24 from 
transduction culture of susceptible cell lines and primary target cells, sequence of 
transferred gene identical to expected sequence, contaminating DNA within 
acceptable limits. In addition, stability testing (transducing titer) will be performed 
annually. 
6.1.7 CD34+ cell isolation, culture, and transduction 
CD34+ cell isolation: In process tests: Cell count, viability (dye exclusion) and 
CD34+ analysis using FACS. 
Acceptance criteria: Viability greater than 70%, CD34+ cell content greater than 
70%. 

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6.1.8 Labeling, receiving, storage, dispensing and return 
Procedure for Labeling and Tracking the Product: The product is tracked and 
labeled according to FACT approved SOPs available in the clinical stem cell labs. 
The labels used for the product conform to current FACT standards. 
Receipt of Drug Supplies: Shipping of the product is carried out in accordance to 
current FACT standards. The shipping container allows for controlled temperature 
(2-8 degrees C) to be maintained within the shipping period, the temperature is 
tracked using data loggers. The apheresis product slated for transduction is received 
in the UC Davis GMP facility with appropriate chain of custody documentation and 
is properly logged. The shipment is acceptable if the temperature was maintained 
within 2-8 deg C during shipping, and no damage to the container or its contents is 
visible. Chain of custody documentation is filled in and a copy of it is sent to the 
center that shipped the product. Transduction of the product begins immediately 
after receipt. 
6.1.9 Stem cell transduction 
The Collection of CD34+ cell may take multiple days. The product needed for the 
un-transduced portion of this protocol will be collected first and will be frozen and 
stored according to each institution’s guidelines. Once the minimum required 
number of CD34+ cells in the product is collected, the collection for products aimed 
for transduction will start. The cells will be immediately transduced within 24 hours 
of collection each day and harvested 48 hours later. After harvest, the transduced 
cell population will be isolated using clinical grade CD25 antibodies coupled to 
magnetic beads and then cryopreserved. Multiple apheresis products may need to 
be collected and transduced until the required number of selected cells has been 
obtained. An aliquot of transduced, selected cells from each final product will be 
tested for viability, sterility, endotoxin, mycoplasma, vector copy number, and 
transduction efficiency post selection. Right before the stem cell infusion, the 
transduced product will be thawed, and an aliquot will be used from each bag for 
stability testing. 
6.1.10 Storage 
The cryopreserved, transduced, and selected products are stored at the 
cryopreservation facilities of each stem cell lab. The same FACT approved SOPs 
as used for cryostorage, labeling and tracking of other stem cell products apply and 
are modified accordingly for these research products. Storage of the products is 
segregated from other products designated for routine clinical use. 
6.1.11 Dispensing 
Criteria for final cellular product release tests 
• Gram stain 
• Sterility (USP 14-day sterility according to 21CFR 610.12) 
• LAL endotoxin level 
• Mycoplasma by PCR 

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• Viability (dye exclusion) 
• Identity (described below) 
• Potency (described below) 
• Transduction efficiency (CD25 expression by flow cytometry) 
• Number of vector copies (qPCR). 
Acceptance criteria: Gram stain and sterility – no organisms seen, endotoxin below 
acceptable limits (less than 0.5 EU/mL), mycoplasma not detectable, viability 
greater than 70%, identity and purity-confirmed CD34+ purity greater than 70%, 
transduction efficiency: 1-99%, number of vector copies: less than 3 per genome. 
6.1.12 Return 
The cryopreserved products are shipped to the centers in an LN2 container. The 
shipping container allows for controlled temperature (-70 to -80 degrees C) to be 
maintained within the shipping period, the temperature is tracked using data 
loggers. Appropriate chain of custody documentation accompanies the shipment. 
The receiving center fills in the required portion of the chain of custody 
documentation, downloads the temperature data from the data logger and 
immediately returns a copy of the filled in document and temperature log to the UC 
Davis GMP facility. The shipment is acceptable if the temperature was maintained 
less than -140 degrees C during shipping, and no damage to the container or its 
contents is visible. 
6.1.13 Criteria for final infused product release tests: (performed by transplant centers 
after thawing the product). 
• Stat viability, 14-day sterility. 
• Acceptance criteria: Stat viability greater than 70%.14-day sterility - no 
organisms seen. 14-day sterility is only available after 14 days; a positive 
culture action plan is followed in case of a positive culture result. 
6.1.14 Stability 
Vector stability will be assessed every year through the following tests: sequencing 
of transduced cells, transgene rearrangement/deletion PCR, levels of transgene 
expression, and vector titer. 
The final product is stable for > 5 years. 
6.1.15 Solution preparation 
Final formulation will consist of a CD25+ cell enriched fraction in 70 mL bags 
containing 40ml total volume containing Normosol supplemented with 15% 
clinical grade human serum albumin and 10% clinical grade DMSO. 
The product must be thawed according to institutional cellular product thawing 
protocols. 

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6.1.16 Route of administration 
Intravenous infusion according to standard transplant protocols. 
6.1.17 Method of administration 
Intravenous infusion according to standard transplant protocols. 
6.1.18 Special handling 
The final CD25+/CD34+ cell product will be shipped frozen in LN2 to the 
respective AMC site for proper institutional cellular product thawing. 
6.1.19 Side effects 
No known side effects from 1TAX vector transduced cells according to the 
preclinical data. 
6.1.19.1 Toxicities associated with conditioning regimen 
Participants will receive BEAM or R-BEAM chemotherapy as a standard 
of care conditioning regimen for transplant. Therefore, these toxicities 
will not be discussed as a part of this protocol. 
6.1.19.2 Gene therapy associated toxicities 
Monitoring for insertional oncogenesis and clonality of hematopoiesis 
after gene therapy 
In any gene therapy protocol using integrating lentiviruses there is a 
theoretical risk of insertional oncogenesis due to Replication Competent 
Lentivirus (RCL) or random or pseudo-random insertional mutagenesis. 
The latter could activate proto-oncogenes, inactivate tumor suppressor 
genes, or activate genes that could lead to cellular dysregulation (e.g., 
generation of an autocrine signaling loop). Based on our pre-clinical 
experience (in collectively more than 600 mice) and experience with 
vectors with very similar backbone in other clinical trials, we do not 
expect to observe gene therapy induced malignancies. However, we will 
routinely monitor participants for any evidence of transduced cell 
clonality. This is particularly important for HSCT based studies for cancer 
since there is an inherent increased risk of secondary malignancies in 
these participants (about 5-8%) due to the risk of chemotherapy, both as 
a treatment for controlling the malignancy and as preparative regimen for 
the transplant. We will routinely perform LAM-PCR every 3 months for 
the first 2 years of the study for vector integrants and look for evidence of 
increasing oligoclonality or monoclonality, defined as a 2-fold increase 
over two successive time points to >20% of total transduced cells. In the 
case that we identify oligo or monoclonality, we will initiate clinical and 
laboratory evaluations, including history and physical exam, blood 
counts, chemistries and appropriate imaging and bone marrow biopsy (if 
clinically indicated), sequencing of LAM-PCR product, flow cytometry 
with immunophenotyping of the cells and cytogenetic analyses. If none 
detected, then LAM-PCRs monitoring will be extended Q3 months for all 

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participants for additional 2 years with the prospective for lifetime routine 
monitoring. 
Incidental production of replication-competent lentivirus (RCL) during 
vector production 
This is highly unlikely during the vector production phase due to the 
design of the vector system; however, the vector product will undergo 
rigorous testing for RCL before release to prevent such a theoretical 
possibility and the incidental production of replication competent 
lentivirus after introduction of vector-transduced cells into the participant. 
RCL testing will also be done for the follow up of participants based on 
the FDA nonbinding recommendations: 
http://www.fda.gov/downloads/BiologicsBloodVaccines/GuidanceComp
lianceRegulatoryInformation/Guidances/CellularandGeneTherapy/ucm0
78723.pdf 
Immune response to foreign Trim V alpha amino acid sequence 
In the chimeric TRIM5α anti-HIV gene, there is a 13-amino acid patch, 
which is foreign to native human TRIM5α. Due to this amino acid 
sequence, it is possible for an antibody based immune reaction to occur 
against cells expressing this region. This is highly unlikely in the setting 
of a myeloablative bone marrow transplant when immune tolerance can 
be induced even against more prominent antigens such as major or minor 
HLA antigens. Furthermore, in our preliminary data, no immune 
stimulation was observed in cells pulsed with this amino acid region. Still, 
and in accordance with FDA guidelines, we will monitor participant 
serum for any chimeric TRIM5α antibody production using an ELISA 
test. Additional testing for cytotoxic T lymphocyte (CTL) reactivity will 
be performed if the ELISA test is positive. If it occurs, we do not expect 
a major negative effect in the participants since the immune reaction will 
most likely delete only transplanted cells that harbor the sequence. In the 
unlikely situation that the reaction eliminates other transplanted stem 
cells, the back-up product can be given to the participants. 
6.1.20 Availability 
The 1TAX vector will be available in sufficient quantity for transduction of all 
CD34+ cells from all 18 participants enrolled in the clinical trial. 
6.1.21 Agent accountability 
The product is tracked and labeled according to FACT approved SOPs available in 
the clinical stem cell labs. The labels used for the product conform to current FACT 
standards. 
Shipping of the product is carried out in accordance to current FACT standards. 
The shipping container allows for controlled temperature (2-8 deg C) to be 
maintained within the shipping period, the temperature is tracked using data 
loggers. The apheresis product slated for transduction is received in the UC Davis 

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GMP facility with appropriate chain of custody documentation and is properly 
logged. The shipment is acceptable if the temperature was maintained within 2-8 
deg C during shipping, and no damage to the container or its contents is visible. 
Chain of custody documentation is filled in and a copy of it is sent to the center that 
shipped the product. Transduction of the product begins immediately after receipt. 

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7.0 
CLINICAL AND LABORATORY EVALUATIONS 
Schedules shown in the Study Calendar below are provided in Appendix I. 
7.1 
Screening Evaluations Required Within 8 Weeks Prior to Stem Cell Collection (unless 
otherwise noted) 
7.1.1 Medical history, history of drug allergies, CDC HIV risk categories, history of 
current and past anti-retroviral regimens, if available; and history of any prior 
AIDS-defining conditions. Date of initial lymphoma diagnosis is required, with a 
copy of the pathology report in the medical record. Current concomitant medication 
list, including all anti-retroviral, anti-viral, antibiotics, and opportunistic 
prophylaxis should be obtained (must be on stable ART regimen for at least 3 weeks 
prior to enrollment in the screening segment). 
7.1.2 Physical examination, including performance status (see Appendix II), vital signs 
(height, weight, body surface area), neurological exam, and physical staging of the 
disease as appropriate for the participant’s lymphoma. 
7.1.3 Chest X-ray (CXR) 
7.1.4 Staging by a CT or a combined PET + CT is required for this study 
7.1.5 EKG 
7.1.6 PFTs; DLCO, FEVI, FVC (within 4 weeks prior to study enrollment) 
7.1.7 Cardiac ejection fraction (by 2D ECHO or MUGA scan) (within 4 weeks prior to 
study enrollment) 
7.1.8 Bone marrow biopsy and aspirate with flow cytometry for pathology 
7.1.9 Laboratory tests, including: 
7.1.9.1 
Assessment for Cytomegalovirus immunoglobulin (CMV IgG) and 
CMV quantitative PCR. 
7.1.9.2 
Assessment for Hepatitis A antibody, Hepatitis B core antibody, 
Hepatitis B surface antigen (HBsAg), Hepatitis B surface antibody, and 
Hepatitis C antibody (within 3 weeks prior to study enrollment). 
If Hepatitis B core antibody or Hepatitis C antibody is positive, 
complete Hepatitis B DNA PCR and Hepatitis C DNA PCR 
7.1.9.3 
Assessment for Herpes simplex virus (HSV) 1 & 2 IgG and PCR 
7.1.9.4 
Assessment for Toxoplasma immunoglobulin G (IgG) 
7.1.9.5 
Assessment for Varicella zoster virus immunoglobulin G (VZV IgG) 
7.1.9.6 
Assessment for Syphilis, Rapid Plasma Reagin (RPR) or Venereal 
Disease Research Laboratory (VDRL) 
7.1.9.7 
Assessment of HIV-1 and Human T-lymphotropic virus antibodies 
7.1.9.8 
Assessment of Creatinine Clearance (within 3 weeks prior to study 
enrollment) 

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7.1.9.9 
Complete blood count (CBC) with differential, Platelet Count, 
comprehensive metabolic panel (CMP), Lactic Acid Dehydrogenase 
(LDH), and Uric Acid 
7.1.9.10 
Assessment of CD4 count 
7.1.9.11 
Assessment of Quantitative immunoglobulins (IgM, IgG, and IgA) 
7.1.9.12 
Serum pregnancy test for women of childbearing potential 
7.1.9.13 
Assessment of PT/PTT or INR/PTT (within 3 weeks prior to study 
enrollment) 
7.1.9.14 
HIV-1 RNA viral load to be assessed by any FDA-approved viral load 
assay (evaluated at the local laboratory). (The HIV viral load assay must 
detect at minimum viremia < 50 copies/mL by RT-PCR) (within 6 
months prior to study enrollment; see Section 3.1.2.2). 
7.1.10 Optional donation to the AIDS and Cancer Specimen Resource (ACSR). (See 
Appendix IV for ACSR Informed Consent Form and Appendix III for ACSR 
Specimen Preparation and Shipping Instructions). 
7.1.11 CCR5-tropism assay (We will use both commercially available assays (i.e., 
phenotypic assay performed by Monogram Biosciences and the genotypic assay 
performed by Quest Diagnostics) (See Appendix IX). 
7.1.12 Immunology assays (correlative study, see Appendix I and Appendix VII). 
7.1.13 DNA monitoring assays (correlative study, see Appendix I and Appendix VII). 
7.1.14 Virology assays (correlative study, see Appendix I and Appendix VII). 
7.2 
Screening Evaluations required within 1 week of admission for stem cell transplant 
7.2.1 Medical history, history of drug allergies, history of current and past anti-retroviral 
regimens, if available; and history of any prior AIDS-defining conditions. Date of 
initial lymphoma diagnosis is required, with a copy of the pathology report in the 
medical record. Current concomitant medication list, including all anti-retroviral, 
anti-viral, antibiotics, and opportunistic prophylaxis should be obtained. 
7.2.2 Physical examination, including performance status (see Appendix II), vital signs 
(height, weight, body surface area), neurological exam, and physical staging of the 
disease. 
7.2.3 CXR 
7.2.4 Complete blood count (CBC) with differential, Platelet Count, comprehensive 
metabolic panel (CMP), Lactic Acid Dehydrogenase (LDH), and Uric Acid 
7.2.5 Assessment of CD4 count 
7.2.6 HIV-1 RNA viral load to be assessed by any FDA-approved viral load assay 
(evaluated at the local laboratory). (The HIV viral load assay must detect at 
minimum viremia < 50 copies/mL by RT-PCR). 

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7.3 
Required on the day of admission for transplant prior to start of chemotherapy 
7.3.1 Medical history, history of drug allergies, history of current and past anti-retroviral 
regimens, if available; and history of any prior AIDS-defining conditions. Date of 
initial lymphoma diagnosis is required, with a copy of the pathology report in the 
medical record. Current concomitant medication list, including all anti-retroviral, 
anti-viral, antibiotics, and opportunistic prophylaxis should be obtained. 
7.3.2 Serum pregnancy test for women of childbearing potential. 
7.3.3 Basic immune assessment (i.e., CD4, CD8, and IgG) will be collected at the 
participating site. 
7.4 
Evaluations During Treatment 
All the procedures in association with the transplant that are mentioned below will be 
performed based on each institute clinical protocols and SOPs. See Section 4.0 for the 
Treatment Plan. 
7.4.1 Performance of apheresis procedure. 
7.4.2 Conditioning (BEAM or R-BEAM) regimen to treat HIV-1-related lymphoma. 
7.4.3 Treatment of conditioning regimen associated side effects such as nausea, diarrhea, 
mucositis, neutropenia fever, etc., and, 
7.4.4 Prophylactic antibiotic choice and duration for fungal, viral, bacterial, and parasitic 
infections. Any deviation from the standard of care needs to be reported to the study 
PI in AdvantageEDCSM and will be reviewed by the AMC DSMB when reviewing 
all participants on the cohort. 
7.5 
Post-Treatment Follow-up Evaluations 
The schedule of follow up is shown in detail in Appendix I and Table 4-A and is 
summarized in the table below. During each follow up, in addition to regular history and 
physical exam, participants will be evaluated for study related side effects, status of their 
lymphoma and the status of HIV-1 using appropriate tests. Assessments during strategic 
ART withdrawal will be on a weekly basis and may be performed remotely, as described 
in Appendix XI. If required clinical and laboratory assessments (physical exam, medical 
history and medical review, toxicity assessments, CD4/CD8 counts and HIV VL) during 
ART withdrawal overlap with required post-treatment follow-up evaluations then these 
assessments do not need to be repeated. 
Table 7A: Follow-Up Schedule 
Study Visit 
Target Day Post-
Transplant 
1 week 
7 ± 3 days 
2 week 
14 ± 3 days 

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Study Visit 
Target Day Post-
Transplant 
3 week 
21 ± 3 days 
4 week 
28 ± 3 days 
6 week 
42 ± 3 days 
2 months 
60 ± 3 days 
3 months 
90 ± 3 days 
4 months 
120 ± 3 days 
6 months 
180 ± 10 days 
8 months 
240 ± 10 days 
10 months 
300 ± 10 days 
12 months 
360 ± 10 days 
14 months 
420 ± 20 days 
16 months 
480 ± 20 days 
18 months 
540 ± 20 days 
20 months 
600± 20 days 
22 months 
660 ± 20 days 
24 months 
720 ± 20 days 
7.5.1 Update medical history. History to include concomitant medication changes, and 
any signs and symptoms. Adverse event assessment (adverse events must be 
recorded in AdvantageEDCSM as per Section 5.4). 
7.5.2 Physical examination, including performance status (see Appendix II), vital signs 
(height, weight, body surface area), neurological exam at every follow-up visit. 
7.5.3 Toxicity assessment 
7.5.4 Assessment of engraftment. 

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Note: this assessment will stop after 2 months. However, assessment of anti-HIV-
1 transduced clonal cell expansion will continue as a part of correlative studies for 
the entire period of follow-up. 
7.5.5 Restaging by a CT or a combined PET + CT, at 3, 6, 12, and 24 months’ time points. 
See Section 8.1 and Section 8.2 for response parameters and frequency of response 
evaluations. 
7.5.6 For participants with bone marrow involvement by the lymphoma: Bone marrow 
biopsy and aspirate with flow cytometry in participants to assess marrow as a part 
of restaging  at 3 months and 12 months timepoints. 
7.5.7 Laboratory tests, including: 
7.5.7.1 Assessment for Cytomegalovirus by CMV quantitative PCR weekly for the 
first month post-transplant, and then at 2, 4, and 6 months post-transplant. 
7.5.7.2 Complete blood count (CBC), comprehensive metabolic panel (CMP), 
Lactic Acid Dehydrogenase (LDH), and Uric Acid 
7.5.7.3 Basic immune reconstitution assays, CD4 and CD8, and IgG levels. 
Performed at 4 weeks post-transplant; and at 2, 3, 4, 6, 8, 10, 12, 18, and 24 
months post-transplant. 
7.5.7.4 HIV-1 RNA viral load to be assessed by any FDA-approved viral load assay 
(evaluated at the local laboratory). The HIV viral load assay must detect at 
minimum viremia < 50 copies/mL by RT-PCRHIV viral load will be 
performed at 4 weeks, 3, 6, 8, 10, 12, 14, 16, 20, and 24 months post-
transplant. 
7.5.8 Correlative studies 
For a complete list of correlative studies and their timeline see the study calendar 
(Appendix I) and also the tables on correlative section (Appendix VII). 
7.5.8.1 Virology studies: 
Blood samples will be collected at 2 and 10 weeks and then 6, 12- and 24-
months post-transplant for HIV reservoir characterization. These studies 
will include quantitative PCR analysis of PBMC-associated HIV DNA, 
RNA (including unspliced and multiply spliced forms) and 2-LTR circles. 
In addition, single genome sequencing of HIV gp120 and HIV pol will be 
performed to understand sequence evolution following transplantation and 
detection of minority resistance variants. Single-copy HIV from plasma will 
also be determined at each of these time points.  
7.5.8.2 GI biopsy (optional) will be performed voluntarily at 6 months post-
transplant (or when CD4 count reaches above 300 cell/mm3) and will be 
repeated two months after the first GI biopsy. The University of California 
San Francisco is conducting a cohort study called SCOPE that also includes 
an optional intestinal biopsy. The SCOPE study is an observational, 
prospective study of HIV-1 infected volunteers designed to provide a 
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Specimens banked as part of the SCOPE study will be used to examine 
questions involving virologic, immunologic, and host factors involved in 
HIV-1 infection, progression, non-progression, response to treatment, 
control of HIV-1 virus, and evolution of drug resistance. If participants 
choose to co-enroll in the SCOPE study at the same time as this study and 
agree to optional GI biopsies, then we will use their intestinal biopsy results 
for both studies. SCOPE is a separate study and requires a separate consent 
form. Participation in SCOPE is not a requirement of this study. 
7.5.8.3 DNA and flow cytometry-based monitoring studies and assessment of anti-
HIV-1 transduced clonal cell expansion and insertional mutagenesis and 
flow cytometric based assays for CD25 expression on CD34+ lymphoid and 
non-lymphoid immune cell populations. These studies will be performed at 
2 weeks and then, 1, 2, 4, 6, 8, 10, 12, 18, and 24 months post-transplant. 
7.5.8.4 Monitoring for immune response to TRIM5α. These studies will be 
performed at 1, 2, 6, 12, 18, and 24 months post-transplant. 
7.5.8.5 Monitoring for mixed lymphocyte reactions: to determine whether 
unmodified, post-SCT PBMC recognize and kill engrafted, modified cells, 
a mixed lymphocyte reaction will be performed using post-SCT PBMCs 
obtained when more than 50% drop in chimerism of transduced cells in PB 
is determined. PBMC will be mixed with pre-SCT modified stem cells 
(generated from remaining CD34+ depleted CD4+ T cells from pre-SCT 
leukapheresis). Gene modified cell killing and markers or T and NK cell 
activation and function will be determined in the ex vivo experiments using 
flow cytometric analysis. 
7.5.8.6 Immunological studies. These studies will be performed at 3, 6, 12, and 24 
months post-transplant. 
7.5.8.7 Optional bone marrow biopsy and aspirate at 3 and 12 months will be 
performed for research in participants without bone marrow involvement 
and that consent to optional studies. 
7.6 
Discontinuation Evaluations 
7.6.1 Early discontinuation. Participants who discontinue protocol treatment early for 
any reason should have the following evaluations and laboratory tests within 30 
days of discontinuation: 
7.6.1.1 History and physical exam and assessment of adverse effect. 
7.6.1.2 Laboratory evaluation for CBC, LDH, uric acid, and CMP. 
7.6.1.3 CT or PET CT and bone marrow biopsy and aspirate with flow cytometry 
for participants with bone marrow involvement for documentation of 
disease staging. See Section 8.1 for response parameters.  
7.6.1.4 Basic immune reconstitution assay (i.e., CD4, CD8 counts and IgG levels). 
7.6.1.5 Correlative studies: immune reconstitution, virologic, and specialized 
immunologic and DNA monitoring studies. 

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7.6.1.6 Optional bone marrow biopsy and aspirate for participants without bone 
marrow involvement that consent to optional studies. 
7.6.2 Off-study evaluations. Participants who complete all treatment per-protocol should 
have the following evaluations and laboratory tests within 30 days of 
discontinuation: 
7.6.2.1 History and physical exam and assessment of adverse effect. 
7.6.2.2 Laboratory evaluation for CBC, LDH, uric acid, and CMP. 
7.6.2.3 CT or PET CT and bone marrow biopsy and aspirate with flow cytometry 
for participants with bone marrow involvement for documentation of 
disease staging. See Section 8.1 for response parameters. 
7.6.2.4 Basic immune reconstitution assay (i.e., CD4 and CD8 counts, and IgG 
levels). 
7.6.2.5 Correlative studies: immune reconstitution, virologic, and specialized 
immunologic and DNA monitoring studies. 
7.6.2.6 Optional bone marrow biopsy and aspirate for participants without bone 
marrow involvement that consent to optional studies. 
7.6.2.7 Follow for survival if stop during 2-year post-treatment (Section 4.7) 
7.7 
Post-Study Follow-up 
At the completion of all follow-up evaluations described in Section 7.5 or Section 7.6, the 
Off-Study Summary Form should be completed in AdvantageEDC. 
Follow for survival if stop during 2-year post-treatment (Section 4.7) 
Based on the FDA requirements for gene therapy protocols, for all participants on this 
study who received the gene-modified stem cells, whether they completed the study or 
came off the study early, all participants will be monitored for 15 years post-
transplantation. The study team at each respective institution will make yearly phone calls 
to the participants’ primary care physician, their oncologist, or their HIV physicians to fill 
out a simple questionnaire about participant health, HIV status, and development of any 
other cancer or clonal abnormality. 
For years 3-15, with participant consent, optional blood samples will be collected for HIV 
viral load, DNA, and immunological studies to monitor HIV and for research on vector 
parameters. 

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8.0 
MEASUREMENT OF EFFECT 
8.1 
Response Parameters 
The Lugano Classification by Cheson DB, et al., 2014 (36) will be used for response 
assessments. 
• Time to Tumor Response (TTR): time from the first study treatment until 
documentation of first objective response. 
• Response Duration: time from first documentation of response to documentation of 
first relapse or progression. 
• Duration of CR: time from the first documentation of CR until first date that relapsed 
or progressive disease is objectively documented. 
• Progression-Free Survival (PFS): time from start of study treatment to relapse, 
progression, or death from any cause, whichever occurs first. For this endpoint, 
persistent disease (not meeting criteria for progression) is not considered a failure. 
Participants who receive anti-cancer therapy other than the study drug (including 
consolidative radiation therapy or BMT) will be censored for all endpoints other than 
overall survival, including toxicity, response, and PFS. 
• Overall Survival (OS): time from start of study treatment to death. 
8.2 
Frequency of Response Evaluation 
• Physical examination for any evidence of disease relapse should occur at the time of 
each history and physical exam. 
• In the absence of evident disease progression, response will be assessed formally at 3, 
6, 12- and 24-months post-transplant per study calendar. 
• F-FDG-PET/CT or CT scan is required for restaging of lymphoma. 
• one marrow biopsy and aspirate with flow cytometry are required at 3 and 12-months 
post-transplant for all participants with bone marrow involvement at baseline. 
Additional bone marrow biopsy maybe required based on the discretion of participants’ 
physicians. 
8.3 
Safety Measures 
All AEs will be monitored by ongoing review of reported adverse events by the protocol 
chair and the AMC Operations and Data Management Center, and will be discussed during 
the monthly investigators’ Lymphoma Working Group conference calls (see Section 9.3 
for rules for early study termination). 

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9.0 
STATISTICAL CONSIDERATIONS 
9.1 
Study Design/Endpoints 
Each cohort will enroll 3 participants but can be extended to 6 participants if recommended 
by DSMB (this will occur only if there is a treatment failure either from failure to engraft 
or from Grade 3 or 4 toxicities, excluding alopecia). 
The primary endpoint of the study is: 
• Safety defined as timely engraftment (the collective establishment of a persistent 
absolute neutrophil count of at least 500 cells/mm3 and platelet count of at least 20,000 
cells/mm3 for 3 consecutive days) at one-month post-transplant, in the absence of any 
study candidate-specific grade 3 and 4 non-hematopoietic organ toxicity, excluding 
alopecia, or any clonal expansion. 
The major secondary endpoints of the study are: 
• The efficacy of the candidate product, defined as establishment of > 5% mononuclear 
blood cells expressing anti-HIV genes in the peripheral blood at 3 months post-
transplant. 
The 5% threshold of the study has been set by our external advisory committee based 
on the outcome of previously performed clinical trials with similar vectors. However, 
we expect to have much higher levels of engraftment of our product based on high 
vector titer levels, new selection method for harvesting only transduced stem cells, 
improved and novel method of transduction, and the new sequence of transduction 
followed by freezing that has provided a significant improvement in our transduction 
efficiency. 
• To determine the presence, quantity, and duration of gene modified HIV-1 resistant 
peripheral blood cells and gut mucosal immune cells; and 
• To study the integration sites of vector sequences in circulating cells. 
In addition, the following minor endpoints will be studied as secondary endpoints: 
• Progression-free survival 
• Overall survival 
• Complete response rate and duration 
• Partial response rate and duration 
• Time to neutrophil engraftment (first measurement of 3 consecutive laboratory values 
obtained on different days) of ANC > /= 500 cells/mm3) 
• Time to platelet engraftment (first measurement of 3 consecutive laboratory values on 
obtained different days) of platelets > /= 20,000 cells/mm3 without platelet transfusions 
7 days prior) 
• Hematologic function at Day 100 (ANC > 1500, Hb > 10g/dl without transfusion, and 
platelets > 100,000. 
• CD4 recovery at the conclusion of the study 

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• Safety in terms of toxicities, infections, transfusions, and infusion-related reactions 
• HIV-1 viral load over time 
• Persistence of vector-transduced cells over time 
The exploratory endpoint of this proposal is to evaluate the presence and the magnitude 
of expansion of HIV-1 resistant immune cells in the peripheral blood and gut mucosa of 
transplanted participants, subsequent to withholding ART. 
9.2 
Sample Size/Accrual Rate 
A minimum of 2 and a maximum of 18 participants will be enrolled in the study, with more 
than 7 participants being enrolled only if one or more participants in a cohort experience 
failure of engraftment or Grade 3 or 4 toxicity, excluding alopecia. The accrual rate is 
anticipated to be 1 participant per month. 
With a sample size of 7, any toxicity occurring in as much as 20% of this patient population 
has a 79% chance of being seen in at least one of the participants in the study, and a toxicity 
occurring in as much as 10% of the population has a 52% chance of being observed in at 
least one participant. 
Toxicity will be summarized as the proportion experiencing a given toxicity or group of 
toxicities, at or above a specified level of severity, with exact 95% CIs. The analysis set 
for toxicity will be participants who enrolled and received at least one active treatment with 
gene therapy. 
9.3 
Early Stopping Rules 
The AMC DSMB is responsible for assessing trial data for dose escalation/cohort 
expansion and evaluating the stopping rules for this study. The composition and role of the 
AMC DSMB in this clinical trial is defined in the AMC DSMB charter. Based on pre-IND 
discussions with FDA, the following short term (day 30) and long term (day 90) stopping 
rules will be evaluated: 
• Lack of timely hematopoietic engraftment in two out of 6 participants, within the first 
30 days after stem cell infusion (as defined in Section 1.2). If this rule is met in cohort 
3 (2.0 x 106 cells/µL), the DSMB will evaluate whether it is safe for the trial to proceed 
on cohort 3+ (3.0 x 106 cells/µL). 
• Grade 3/4 non-hematopoietic toxicity, excluding alopecia, directly associated with the 
gene therapy product in two out of 6 participants in each cohort within the first 90 days 
of the trial. 
• Death of one participant directly related to the gene therapy product (e.g., death due to 
lack of engraftment after day 20, but before day 30). 
9.4 
Cohort Escalation Design 
The dose escalation in this trial is based on the increasing ratio of the administrated 
transduced product to untransduced mobilized peripheral stem cells. Please see the clinical 
trial design section for the details of dose escalation in each cohort. 
9.5 
Analysis of Secondary Endpoints 

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Secondary and exploratory endpoints will be summarized descriptively. Efficacy rates will 
be summarized by the proportion of participants who meet the criteria for efficacy (defined 
in Section 9.1), with 95% exact binomial confidence intervals (CIs). For dichotomous 
endpoints (e.g., the presence of gene modified HIV resistant peripheral blood cells and gut 
mucosal immune cells, complete response) the frequency, proportion, and exact 95% 
confidence interval for proportion will be calculated. Continuous measures (e.g., quantity 
and duration of gene modified HIV resistant peripheral blood cells and gut mucosal 
immune cells) will be summarized by mean (SD) and median (range), with log 
transformation if necessary, for skewed measures, as would be typical for cell counts. 
Time-to-event data (e.g., overall survival, progression free survival, duration of response) 
will be presented graphically by Kaplan-Meier plots and summarized by estimated median 
time to event (if that is estimable from the data) with 95% confidence interval. 
Separate inferences for the three cohorts will not be possible, given the limited sample size, 
but descriptive summaries will be prepared for key variables to inform decisions about the 
next stage of clinical trials. 
9.6 
AMC Policy for Monitoring of Phase II Trials 
This protocol will follow the AMC’s policy for data monitoring (See Appendix VI), which 
serves to assure that: 
• Only participants who meet the study eligibility criteria are enrolled. 
• The informed consent process is conducted appropriately. 
• Data is collected and analyzed as specified in the protocol. 
• Adverse events are reviewed promptly and reported as required. 
• Privacy and confidentiality of study participants are maintained. 
Independent Data and Safety Monitoring Board 
All gene therapy protocols according to NIH and FDA guidelines are included in the “high 
risk” category and therefore require a Data and Safety Monitoring Board (DSMB) for 
assessing the risk to the patient population. The AMC DSMB will be used as an 
independent group to monitor the potential efficacy and/or toxicities of the protocol. The 
members of the DSMB (at least 6 members) will be chosen with appropriate broad 
spectrum of expertise, including biostatisticians, HIV experts, gene therapy experts, 
bioethics experts, HIV patient advocates, and lay members. They will set the guideline to 
assess the study risk by description of anticipated adverse events, adverse event grading 
and plans for reporting adverse events. They will also institute a safety-monitoring plan  
The AMC DSMB will be given a report by the PI(s) indicating the number of participants 
who have enrolled, a list of all adverse events, and the results of clinical measures. The 
AMC DSMB will review the safety data for the participants in each cohort after the 30 
days post-transplant follow up period to identify whether DLT occurred, and if a decision 
should be made to proceed with the study for the subsequent participants according to the 
DLT definition and dose escalation rules in Section 4.3. At the time of each review, the 
AMC DSMB will be asked to recommend continuation or discontinuation of the trial. If, 
in the opinion of the AMC DSMB there is any indication of compromise of the safety of 

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the participants, the AMC DSMB will contact the PI(s) and the study will be halted. 

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10.0 
ROLE OF DATA MANAGEMENT 
10.1 
CRF Instructions 
Access to the internet data entry system for this study, AdvantageEDCSM, and instructions 
for recording of study data on CRFs will be provided by the AMC ODMC at 
www.amcoperations.com. Participating institutions are responsible for submitting data 
and/or data forms via AdvantageEDCSM in accordance with the AMC Data Entry Guide 
and specific form instructions and in compliance with 21 CFR 11, within the timelines 
specified by the AMC’s Standards of Procedure for Site Performance Measures. 
10.2 
Data Quality 
It is the responsibility of the AMC ODMC to assure the quality of data for the study (See 
Appendix VI, AMC Data and Safety Monitoring Plan). This role extends from protocol 
development to generation of the final study database. 
10.3 
Data Monitoring 
This study will be monitored in compliance with AMC policies and by the Clinical Data 
Update System (CDUS) Version 3.0. Cumulative protocol- and participant-specific CDUS 
data will be submitted electronically to CTEP on a quarterly basis. Reports are due January 
31, April 30, July 31, and October 31. Instructions for submitting data using the CDUS can 
be found on the CTEP Web site (http://ctep.cancer.gov/reporting/cdus.html). 
The AMC ODMC is responsible for compiling and submitting CDUS data to CTEP for all 
participants and for providing the data to the Principal Investigator for review. 

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11.0 
ETHICAL AND REGULATORY CONSIDERATIONS 
11.1 
IRB Approval and Informed Consent 
The principles of Institutional Review Board (IRB) approval and informed consent 
described in the Food and Drug Administration (FDA) regulations (21 CFR Part 50 and 
56) and Department of Health and Human Services (DHHS) regulations for the Protection 
of Human Subjects regulations (45 CFR Part 46), as well as the International Conference 
on Harmonization guideline for Good Clinical Practice E6 (R1) and Declaration of 
Helsinki must be followed. IRB approval of the protocol and the informed consent form 
must be given in writing. 
The sponsor’s designee (AMC ODMC) must receive a copy of the letter of approval from 
the IRB, which specifically approves the protocol and informed consent, before participant 
enrollment. The IRB must also approve any significant changes to the protocol and 
documentation of this approval must be sent to the AMC ODMC. The IRB must review 
the research project at least once every 365 days during the duration of the project. 
Continuing approval of the project must also be given in writing and provided to the AMC 
ODMC. 
Records of all study review and approval documents must be kept on file by the 
Investigator and are participant to inspection during or after completion of the study. AEs 
must be reported to the IRB according to local procedures. The IRB should receive 
notification of completion of the study and final report within 3 months of study completion 
and termination. The Investigator will maintain an accurate and complete record of all 
submissions made to the IRB, including a list of all reports and documents submitted. 
Written informed consent will be obtained from the participant. The nature and significance 
of the risks associated with the study must be explained to the participant. The informed 
consent will describe the purpose of the study, the procedures to be followed, the risks and 
benefits of participation, all risks of the investigational agent(s) and/or study participation 
as listed in the model informed consent form, and all other elements of informed consent 
as required by regulation. A copy of the consent form will be given to the participant to 
keep. 
In addition, any institution(s) conducting research according to the guidelines of this 
protocol is required to adhere to local and national laws and regulations governing the 
confidentiality and disclosure of health information. 
11.2 
Multi-Site Research 
• All sites have the most current version of the protocol, consent document, and HIPAA 
authorization. 
• All required approvals will be obtained at each site (including approval by the site’s 
IRB of record and Institutional Biosafety Committee as applicable). 
• All modifications will be approved by FDA first and they will be communicated to 
sites and approved (including approval by the site’s IRB of record) before the 
modification is implemented. 

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• All engaged participating sites will safeguard data as required by local information 
security policies. 
• All local site investigators conduct the study appropriately. 
• All non-compliance with the study protocol or applicable requirements will be reported 
in accordance with local policy. 
The DSMB will communicate the information below to all the engaged participating sites: 
• Problems. 
• Interim results. 
• The closure of a study. 
11.3 
Data Management and Confidentiality 
Provisions to protect the privacy interests of participants 
Prior to study enrollment, participants will be contacted by a member of the research team 
in a private setting such as the clinic exam room. The research team will explain the study 
treatment, procedures, and the timing of each to the participants. The participants will be 
offered the opportunity to ask questions. After study enrollment, participants’ privacy 
interests will be respected at all times, and requests for specific privacy requirements will 
be met if at all possible, taking into consideration the study protocol requirements and the 
participants’ welfare. 
HIPAA 
A HIPAA authorization will be obtained for every participant (or Legally Authorized 
Representative, if applicable) enrolled to the study, and it will be done at the time the 
participant is consented to the study. The HIPAA Authorization approved by the UC Davis 
Compliance Office or the participating site’s compliance office will be used. 
After a study participant has signed a study-specific HIPAA authorization and an IRB-
approved study consent form, the research team is permitted to access all sources of their 
medical information and protected health information (PHI). This includes the participant’s 
electronic medical record (EMR) as well as any scans or medical/PHI documentation in 
paper form. 
Privacy risks 
The Sponsor-Investigator will maintain all study records according to ICH-GCP and 
applicable regulatory requirement(s). 
Data and/or specimens will be labeled with a code that the research team can link to 
personal identifying information when acquired. The code sheet will be secured and kept 
separate from the dataset. 
The researchers will store study records and other information in a secure location and will 
grant access only to those with a need to know. However, just like with other personal 
information kept by health care providers, banks, and others, even these safeguards cannot 
guarantee absolute protection of the data. If private information gets into the wrong hands, 

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it can cause harm. Although rare, there are reported cases of breaches that have resulted in 
discrimination in insurance or employment. 
11.4 
Changes to the Protocol 
Any change or addition to this protocol requires a written protocol amendment that must 
be approved by CTEP and the Investigator before implementation. All amendments require 
approval by the IRB of the treating institution. A copy of the written approval of the IRB 
must be sent to the ODMC. 
11.5 
Vulnerable Populations 
This study is not designed to enroll any participants from the following vulnerable 
populations: pregnant women, neonates, prisoners, persons who have not attained the legal 
age for consent to treatments or procedures involved in the research, or cognitively 
impaired adults. 
11.6 
Non-English-Speaking Participants 
Non-English-speaking participants are not excluded from participation in this trial. For 
potential participants who do not speak English, local IRB policy for translation of 
informed consent forms and/or documentation of a short form consent must be followed. 
11.7 
Women and Minorities 
This study is being conducted by the NCI-sponsored AIDS Malignancy Consortium 
(AMC). As part of their contractual obligations, each participating site within the AMC 
and the AMC as a whole is required to assure that the participation of women and minority 
participants reflects the percentage representation of these populations in their geographic 
region and, for the AMC, the United States as a whole. As such, it is expected that the 
representation of participants on this trial will reflect the constitution of the respective 
populations. 
 
 

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Table 11-A: Accrual Targets 
Ethnic Category 
Sex/Gender 
Females 
 
Males 
 
Total 
Hispanic or Latino 
1 
+ 
3 
= 
4 
Not Hispanic or Latino 
1 
+ 
13 
= 
14 
Ethnic Category: Total of all participants 
2 
+ 
16 
= 
18 
Racial Category 
 
American Indian or Alaskan Native 
0 
+ 
0 
= 
0 
Asian 
0 
+ 
1 
= 
1 
Black or African American 
1 
+ 
3 
= 
4 
Native Hawaiian or other Pacific Islander 
0 
+ 
1 
= 
1 
White 
1 
+ 
11 
= 
12 
Racial Category: Total of all participants 
2 
+ 
16 
= 
18 
11.8 
Compensation for Research-Related Injury 
Participants are instructed that it is important they promptly tell the person in charge if they 
believe that they have been injured because of taking part in this study. For participants 
who are injured as a result of being in this study, individual institutions will provide 
necessary medical treatment based on their own policies. Depending on the circumstances, 
the costs of the treatment may be covered by the University or the study sponsor or may be 
billed to the participant’s insurance company just like other medical costs. The University 
and the study sponsor do not normally provide any other form of compensation for injury. 
11.9 
Economic Burden to Participants 
Standard of care and other routine costs involved with this study will be billed to the 
participant or the participant’s insurance carrier, Medicare, or Medi-Cal, where 
appropriate. These costs are consistent with the routine care participants would receive 
outside of the study and may include standard medical tests and examinations (such as 
blood work and physical exams, pharmacy charges, scans, etc.). Whenever possible, 
preauthorization will be obtained. If the costs are not covered, these costs will be discussed 
with the participant prior to proceeding with the study. The participant will be responsible 
for any costs not covered by his/her insurance. 

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12.0 
REFERENCES 
1. Martinez‐Picado J, et al., 2000. Antiretroviral resistance during successful therapy of HIV‐1 
type 1 infection. Proc Natl Acad Sci USA; 97:10948–10953. 
2. Winters MA, et al., 2000. Frequency of antiretroviral drug resistance mutations in HIV-1 
strains from patients failing triple drug regimens. The Terry Beirn Community Programs 
for Clinical Research on AIDS. Antivir Ther; 5:57–63. 
3. Bacheler LT, et al., 2000. Human immunodeficiency virus type 1 mutations selected in patients 
failing efavirenz combination therapy. Antimicrob Agents Chemother; 44:2475–2484. 
4. Lafeuillade A, et al., 2001. Phenotypic and genotypic resistance to nucleoside reverse 
transcriptase inhibitors in HIV-1 clinical isolates. HIV-1 Med; 2:231-235. 
5. Henrich TJ, et al., 2010. Evolution of CCR5 antagonist resistance in an HIV-1 subtype C 
clinical isolate. J Acquir Immune Defic Syndr. 55:420-427. 
6. Tilton JC, et al., 2010. A maraviroc resistant HIV-1 with narrow cross-resistance to other 
CCR5 antagonists depends on both N-terminal and extracellular loop domains of drug-
bound CCR5. J Virol. 84:10863-10876. 
7. Aiuti F and Mezzaroma I., 2006. Failure to reconstitute CD4+ T cells despite suppression of 
HIV-1 replication under HAART. AIDS Rev. 8:88-97. 
8. Ianello A, et al., 2010. Potential role of IL-18 in the immunopathogenesis of AIDS, HIV-1-
associated lipodystrophy and related clinical conditions. Curr HIV-1 Res. 8:147-164. 
9. Baldanti F, et al., 2010. Early emergence of raltegravir resistance mutations in patients 
receiving HAART salvage regimens. J Med Virol. 82:116-122. 
10. Mascolini M, et al., 2008. Clinical implications of resistance to antiretrovirals: new resistance 
technologies and interpretations. Antivir Ther. 13:319-34. 
11. Strayer DS, et al., 2005. Current Status of Gene Therapy Strategies to Treat HIV- 1/AIDS. 
Mol. Ther. 11:823-841. 
12. Humeau LM, et al., 2004. Efficient lentiviral vector-mediated control of HIV-1 replication in 
CD4 lymphocytes from diverse HIV-1+ infected patients grouped according to CD4 count 
and viral load. Mol. Ther. 9:902-913. 
13. Mitsuyasu RT, et al., 2009. Phase 2 gene therapy trial of an anti-HIV ribozyme in autologous 
CD34+ cells. Nat Med. 15:285-292. 
14. DiGiusto DL, et al., 2010. RNA-based gene therapy for HIV-1 with lentiviral vector-modified 
CD34(+) cells in patients undergoing transplantation for AIDS- related lymphoma. Sci 
Transl Med. 2:36ra43. 
15. Kimpel, J., Braun, S.E., Qiu, G., Wong, F.E., Conolle, M., Schmitz, J.E., Brendel, C., Humeau, 
L.M., Dropulic, B., Rossi, J.J. et al., 2010. Survival of the fittest: positive selection of CD4+ 
T cells expressing a membrane-bound fusion inhibitor following HIV-1 infection. PLoS 
One, 5, e12357. 

AMC-097 (Version 9.0) 07FEB2022 
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16. Van Lunzen, et al., 2007. Transfer of autologous gene-modified T cells in HIV-1- infected 
patients with advanced immunodeficiency and drug-resistant virus. Mol Ther 15: 1024-
1033. 
17. Von Laer, D, Hasselmann, S, Hasselmann, K, 2006. Gene therapy for HIV-1 infection: what 
does it need to make it work? J Gene Med 8: 658-667. 
18. Podsakoff GM, Engel BC, Carbonaro DA, Choi C, Smogorzewska EM, Bauer G, Selander D, 
Csik S, Wilson K, Betts MR, Koup RA, Nabel GJ, Bishop K, King S, Schmidt M, von 
Kalle C, Church JA, Kohn DB., 2005. Selective survival of peripheral blood lymphocytes 
in children with HIV-1 following delivery of an anti- HIV gene to bone marrow CD34(+) 
cells. Mol Ther. 2005 12:77-86. 
19. Aviran, S., Shah, P.S., Schaffer, D.V. and Arkin, A.P., 2010. Computational models of HIV-1 
resistance to gene therapy elucidate therapy design principles. PLoS Comput. Biol., 6, 
e1000883. 
20. Applegate, T.L., Birkett, D.J., McIntyre, G.J., Jaramillo, A.B., Symonds, G. and Murray, J.M., 
2010. In silico modeling indicates the development of HIV-1 resistance to multiple shRNA 
gene therapy differs to standard antiretroviral therapy. Retrovirology, 7, 83. 
21. Kessinger A, Nademanee A, Forman SJ, Armitage JO., 1990. Autologous bone marrow 
Transplantation for Hodgkin’s and non-Hodgkin’s lymphoma. Hematol Oncol Clin North 
Am. 1990; 4:577. 
22. Philip T, Guglielmi C, Hagenbeek A, Somers R, van der Lelie H, Bron D, Sonnefeld P, 
Gisselbrecht C, Cahn J-Y, Harousseau J-L, Coiffier B, Biron P, Mandelli F, Chauvin F., 
1995. Autologous bone marrow transplantation as compared with salvage chemotherapy 
in relapses of chemotherapy sensitive non-Hodgkin's lymphoma. N Engl J Med 
1995;333:1540. 
23. Vose JM, Anderson JR, Kessinger A, Bierman Ph, Coccia P, Reed EC, Gordon B, Armitage 
JO., 1993. High-dose chemotherapy and autologous hematopoietic stem-cell 
transplantation for aggressive non-Hodgkin's lymphoma. J Clin Oncol 1993;11:1846. 
24. Krishnan A, Molina A, Zaia, J, et al., 2005. Durable remission with autologous stem cell 
transplantation for high-risk HIV-1-associated lymphomas. Blood. 2005;105:874. 
25. Spitzer TR, Ambinder RF, Lee JY, et al., 2008. Dose-reduced busulfan, cyclophosphamide 
and autologous stem cell transplantation for human immunodeficiency virus-associated 
lymphoma: AIDS Malignancy Consortium study. Biol Blood Marrow Transplant. 
2008;14:59. 
26. Gabarre J, LeBlond V, Sutton L, et al., 1996. Autologous bone marrow transplant in relapsed 
HIV-1-related non-Hodgkin’s lymphoma. Bone Marrow Transplant. 1996;18:1195. 
27. Re A, Cattaneo C, Michieli M, et al., 2003. High-dose therapy and autologous peripheral blood 
stem-cell transplantation as salvage treatment for HIV-1-associated lymphoma in patients 
receiving highly active antiretroviral therapy. J Clin Oncol. 2003;23:4423. 
28. Serrano D, Carrion R, Balsolobre P, et al., 2005. HIV-1-associated lymphoma successfully 
treated with peripheral blood stem cell transplantation. Exp Hematol. 2005;33:487. 

AMC-097 (Version 9.0) 07FEB2022 
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29. Balsalobre P, Diez-Martin JL, Re A et al., 2009. Autologous stem-cell transplantation in 
patients with HIV-1-related lymphoma. J Clin Oncol. 2009: 27:2192. 
30. Diez-Martin JL, Balsalobre P, Re A, et al., 2009. Comparable survival between HIV-1+ and 
HIV-1- non-Hodgkin and Hodgkin lymphoma patients undergoing autologous peripheral 
blood stem cell transplantation. Blood 2009: 113:6011. 
31. Lundgren JD, Babiker A, El-Sadr W, et al., 2008. An inferior clinical outcome of the CD4+ 
cell count-guided antiretroviral treatment interruption strategy in the SMART study: role 
of CD4+ cell counts and HIV-1 RNA levels during follow-up. J Infect Dis 2008; 197: 1145-
55. 
32. Pai NP, Tulsky JP, Lawrence J, et al., Structured treatment interruptions (STI) in chronic 
suppressed HIV-1 infection in adults (review). Cochrane Library 2008; 1:1-37. 
33. G. Carcelain et al., 2001. Transient mobilization of human immunodeficiency virus (HIV-1)-
specific CD4 T helper cells fails to control virus rebounds during intermittent antiretroviral 
therapy in chronic HIV-1 type 1 infection. J. Virol., 75, 2001; 234–241 
34. Panel on Antiretroviral Guidelines for Adults and Adolescents. Guidelines for the use of 
antiretroviral agents in HIV-1-1-infected adults and adolescents. Department of Health and 
Human 
Services. 
08 
April 
2015; 
1-288. 
Available 
at, 
http://aidsinfo.nih.gov/contentfiles/AdultandAdolescentGL.pdf 
35. Alvarnas J, Le Rademacher J, et al., 2014. Autologous Hematopoietic Stem Cell Transplant 
(AHCT) in Patients with Chemotherapy-Sensitive, Relapsed/Refractor (CSRR) Human 
Immunodeficiency Virus (HIV)-Associated Lymphoma (HAL): Results from the Blood 
and marrow Transplant Clinical Trials network (BMT CTN 0803)/AIDS Malignancy 
Consortium (AMC-071) Trial. ASH Oral and Poster Abstract 674, 08 December 2014; 
Available at, https://ash.confex.com/ash/2014/webprogram/Paper70947.html 
36. Cheson DB, Fisher RI, et. al, 2014. Recommendations for initial evaluation, staging, and 
response assessment of Hodgkin and non-Hodgkin lymphoma: The Lugano Classification. 
J Clin Oncol. 2014; 32(27): 3059-68. 
 
 

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APPENDIX I: SCHEDULE OF EVALUATIONS 
The schedule of evaluations below applies to all participants on study. 
App – 1A: Pre-Transplant Screening; Mobilization Period 
Required Studies/Testing 
Within 8 
Weeks Prior 
to Enrollment 
(Stem Cell 
Collection) 
Within 1 
Week Prior 
to 
Admission 
for 
Transplant 
On the Day of 
Admission for 
Transplant Before 
Start of 
Chemotherapy 
History & Physical with assessment of physical staging of the 
disease, Height and Weight, BSA, neurologic examination, 
review of current and prior anti-retroviral regimens, concomitant 
medications, and history of any AIDS defining conditions 
X 
X 
X 
Hepatitis Panel5 (HAV Ab, HBsAg, HBcAb HBsAb, Hepatitis C 
Ab), HSV 1 & 2 IgG and PCR, Toxoplasma IgG, VZV IgG, 
Syphilis (RPR or VDRL), HIV-1 and HTLV antibody 
X 
 
 
CXR 
X 
X 
 
EKG 
X 
 
 
PFTs; DLCO, FEVI, FVC4] 
X 
 
 
Creatinine Clearance5 
X 
 
 
CMV Immunoglobulin level and CMV quantitative PCR 
X 
 
 
Bone marrow biopsy and aspirate with flow cytometry for 
pathology 
X 
 
 
HIV-1 RNA10, CD4 Count 
X 
X 
 
HepB DNA PCR and HepC DNA PCR1 
X 
 
 
Cardiac Ejection Fraction (by 2D ECHO or MUGA scan)4 
X 
 
 
B –HCG Test for Females of Childbearing Potential 
X 
 
X 
Quantitative immunoglobulins (IgM, IgG, and IgA) 
X 
 
 
Staging CT or PET CT 
X 
 
 
Karnofsky Performance Score/ECOG Performance Status 
X 
X 
 
CBC with differential, Platelet Count, CMP, LDH and Uric acid 
X 
X 
 
CCR5-tropism assay2 
X 
 
 
Immune reconstitution assays, basic3 
 
 
X 

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Required Studies/Testing 
Within 8 
Weeks Prior 
to Enrollment 
(Stem Cell 
Collection) 
Within 1 
Week Prior 
to 
Admission 
for 
Transplant 
On the Day of 
Admission for 
Transplant Before 
Start of 
Chemotherapy 
PT/PTT assessment5 
X 
 
 
Immunology assays, correlatives8 
X 
 
 
DNA monitoring assays, correlatives9 
X 
 
 
Virology assays, correlatives8 
X 
 
 
Banking of CD34+ depleted PBMC from apheresis samples6 
X 
 
 
Optional donation to the ACSR7 
X 
 
 
1- Only if the core Ab is positive for HepB or the HepC Ab is positive, respectively. 
2- We will use both commercially available assays (i.e., phenotypic assay performed by Monogram Biosciences and the genotypic assay performed 
by Quest Diagnostics) (See Appendix IX). 
3- CD4, CD8, and IgG will be collected at the participating site as standard of care. 
4- To be collected within 4 weeks prior to study enrollment. 
5- To be collected within 3 weeks prior to study enrollment. 
6– CD34+ depleted cells obtained from GMP Facility. 
7– See Appendix IV for ACSR Informed Consent and Appendix III for ACSR Specimen Preparation and Shipping Instructions. 
8- On all visits where immunological and viral correlatives are to be completed, 40 mL of blood (total) will be drawn in lavender top (EDTA) tubes 
and 5 mL of blood will be drawn in red top tubes.  
9- On all visits where DNA monitoring correlatives are to be completed, 20 mL of blood will be drawn in lavender top (EDTA) tubes. 
10- Within 6 months before study enrollment. See Section 3.1.2.2. 
 

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App – 1B: Post-Transplant Follow Up 
Study Assessments/Testing Post 
Transplant 
Weeks Post-HCT 
Months Post-HCT 
Discontinuation 
Early Disc., and Off-
Study 
Years Post-HCT6 
1 
2  
3 
4 
6 
2 
3 
4 
6 
8 
10 
12 14 
16 
18 20 22 
24 
Within 30 days of 
discontinuation 
3-15, once/yr 
History & Physical, including 
performance status, height, weight and 
BSA, neurological exam, concomitant 
medication review, and Toxicity 
Assessment 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
 
Assessment of engraftment 
X 
X 
X 
X 
X 
X4 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
CBC, LDH, Uric Acid, CMP 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
 
CT or PET-CT restaging 
 
 
 
 
 
 
X 
 
X 
 
 
X 
 
 
 
 
 
X 
X 
 
Required bone marrow biopsy and 
aspirate with flow cytometry (for 
restaging), if bone marrow involvement 
at baseline2 
 
 
 
 
 
 
X 
 
 
 
 
X 
 
 
 
 
 
 
X 
 
Optional bone marrow biopsy and 
aspirate with flow cytometry (for 
research) for those without bone marrow 
involvement at baseline2 
 
 
 
 
 
 
X 
 
 
 
 
X 
 
 
 
 
 
 
X 
 
GI biopsy 
 
 
 
 
 
 
 
 
X5 
X5 
 
 
 
 
 
 
 
 
 
 
Immune Reconstitution Assays, basic1 
 
 
 
X 
 
X 
X 
X 
X 
X 
X 
X 
 
 
X 
 
 
X 
X 
X10 
Immune Reconstitution Assays, 
correlative studies 
 
 
 
 
 
 
X 
 
X 
 
 
X 
 
 
 
 
 
X 
X 
 
CMV PCR  
X 
X 
X 
X 
 
X 
 
X 
X 
 
 
 
 
 
 
 
 
 
 
 
HIV-1 viral load 
 
 
 
X 
 
 
X 
 
X 
X 
X 
X 
X 
X 
 
X 
 
X 
X 
X10 
Virologic and specialized immunologic 
assays, correlative studies3,8 
 
X 
 
 
 
X7 
 
 
X 
 
 
X 
 
 
 
 
 
X 
X 
X10 
DNA monitoring, correlative studies4,9 
 
X 
 
X 
 
X 
 
X 
X 
X 
X 
X 
 
 
X 
 
 
X 
X 
X10 

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Study Assessments/Testing Post 
Transplant 
Weeks Post-HCT 
Months Post-HCT 
Discontinuation 
Early Disc., and Off-
Study 
Years Post-HCT6 
1 
2  
3 
4 
6 
2 
3 
4 
6 
8 
10 
12 14 
16 
18 20 22 
24 
Within 30 days of 
discontinuation 
3-15, once/yr 
Adverse event assessment 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
 
 
Telephone follow-up for HIV status and 
development of secondary malignancies 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
X 
X6 
1. 
At these time points basic immunological tests (i.e., CD4, CD8, and IgG will be done). If collection timepoints overlap with collections as part of ATI / ART resumption, then labs will not need to be repeated. 
2. 
Bone marrow biopsy and aspirate with flow cytometry will be performed as a part of restaging for participants with bone marrow involvement by the lymphoma. Optional bone marrow biopsy and aspirate with 
flow cytometry will be performed for participants without bone marrow involvement that consent to optional studies. 
3. 
Samples will be collected and stored for possible HIV-1 nucleotide sequence analyses for ART drug resistance; Samples will also be collected and stored to explore the impact of anti-HIV transduced stem cells 
on genetic makeup of HIV-1 populations (e.g., co-receptor usage, genetic changes in tat or TRIM5α targets). If needed, we will use both commercially available assays (i.e., phenotypic assay performed by 
Monogram Biosciences and the genotypic assay performed by Quest Diagnostics). 
4. 
Assessment for hematological engraftment will stop at 2 months. However, assessment of anti-HIV-1 transduced clonal cell expansion will continue as a part of correlative studies for the entire period of follow 
up. 
5. 
GI biopsies (upper endoscopy) will be offered to all the participants of the study as a voluntary option at 6 months post-transplant (or when CD4 count reaches above 300 cell/mm3) and will be repeated in two 
months after the first GI biopsy. 
6. 
The follow-up in years 3-15 will be performed by phone calls to participants or their health care provider. The telephone contact for follow-up is not required for participants who discontinue protocol treatment 
early for any reason (early discontinuation) but is required for participants who complete all treatment per-protocol. 
7. 
To be collected at 10 weeks post-transplant. 
8. 
 On all visits where immunological and viral correlatives are to be completed, 40 mL of blood (total) will be drawn in lavender top (EDTA) tubes and 5 mL of blood will be drawn in red top tubes for 
immunological studies and 40 mL of blood (total) will be drawn in lavender top (ETA) tubes for viral studies. 
9. 
On all visits where DNA monitoring correlatives are to be completed, 20 mL of blood will be drawn in lavender top (EDTA) tubes. 
10. Participants that consent to optional studies will have blood samples (30 mL) collected to monitor the participant’s HIV and vector parameters. 
 
 
 
 

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App – 1C: Analytic Treatment Interruption 
The schedule during the ATI period will be defined by clinical course and the characteristics of any virus rebound. ART will be initiated 
for clinical events, sustained drop in CD4+T cell counts or sustained increased in viremia as described in Section 2.4.1. Prior to these 
events, participants will be followed. If required clinical and laboratory assessments (physical exam, medical history and medical review, 
toxicity assessments, CD4/CD8 counts and HIV VL) during ART withdrawal overlap with required post-treatment follow-up evaluations 
then these assessments do not need to be repeated. Clinical assessments, and ATI counseling may be performed remotely to minimize 
in-person visits during the ATI.  
Assessments 
Within One Week 
Prior to ATI 
Weeks 1-12 
Extended ATI 
(Weeks 13+) 
Post ATI1 
 
 
Weekly 
Weekly 
ART 
Resumption 
W4 
W8 
W24 
COVID Testing2 
X 
 
 
 
 
 
 
ATI 
 
X 
X 
 
 
 
 
Clinical Assessments3 
 
X 
X 
X 
X 
X 
X 
T cell counts (CD4 and CD8) 
 
X 
X 
X 
X 
X 
X 
HIV Viral Load4 
 
X 
X 
X 
X 
X 
X 
Plasma and PBMCs5 
 
 
 
X 
X 
X 
X 
Pre-ATI 
counseling 
and 
disclosure6 
X 
 
 
 
 
 
 
Ongoing ATI counseling6 
 
X 
X 
 
 
 
 
 
1.  Once ART is restarted, participants will be followed at the time of ART restart, and at 4, 8, and 24 weeks after restart.  
2.  Participants will be tested for the virus that causes COVID-19. If the test is positive, participants will remain on ART until they recover, and the study team approves them to 
proceed with ATI. Participants on ATI will be offered COVID testing periodically or if symptoms develop. ART will be restarted if participants test positive for COVID-19 by 
PCR. 
3. 
Clinical assessments include: review of medical history, review of concomitant medications, performance status, height, weight, body surface area, neurological exam, and 
toxicity assessment. 
4. 
During ATI participants with an initial viral result greater than 400 copies/mL must have repeat testing within 3 business days. Participants will be evaluated every four weeks 
between weeks 8 and 24 until the viral load is confirmed to be less than the level of quantification. 

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5.  Plasma and PBMCs will be collected in four (4) 10 mL EDTA tubes and cryopreserved at the time of ART resumption and at Weeks 4, 8 and 24 on ART. 
6. 
Participants must be provided the participant HIV interruption participation disclosure sheet (Appendix XII) and have pre-ATI counseling performed (Appendix XI) prior to 
ATI. ATI risk mitigation counseling must be performed weekly during ATI.  
 
 

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App – 1D: Sample Collection guidance 
 
Correlative 
Laboratory 
Baseline 
2 
Weeks 
4 
Weeks 
2 
Months 
10 
Weeks 
3 
Months 
4 
Months 
6 
Months 
8 
Months 
10 
Months 
12 
Months 
18 
Months 
24 
Months 
Discont.n Follow-up 
Years 3-5 
Immunology 
(Pollard 
Lab) 
 
 
 
 
 
 
 
 
 
DNA 
(Anders
on Lab) 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Virology 
(Henrich 
Lab) 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Optional 
GI 
Biopsy1, 2 
(Pollard 
Lab) 
X 
X 

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CCR5-
Tropism 
assays 
(Monogram 
Bioscience) 
   
 
 
Optional 
Bone 
Marrow 
Biopsies3 
 
 
 
 
 
1 Participants must sign the separate consent form to participate in these optional studies 
2 If the participant’s CD4 count is not >300 cells/mm3 at the 6-month timepoint, GI biopsies may be delayed until the participant’s CD4 count has reached 300 cells/mm3, and 
biopsies will be repeated 2 months after the first collection. 
3 For participants with bone marrow involvement at baseline that consent to optional collections. 
KEY:  
 
5mL redtop tube 
 
4mL purple top (EDTA) tube 
 
10mL purple top (EDTA) tube 
 
 

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APPENDIX II: PERFORMANCE STATUS SCALES 
Karnofsky Performance Scale 
ECOG Performance Status Scale 
Percent 
Description 
Grade 
Description 
100 
Normal, no complaints, no 
evidence of disease. 
0 
Normal activity. Fully active, able to 
carry on all pre-disease performance 
without restriction. 
90 
Able to carry on normal activity; 
minor signs or symptoms of 
disease. 
80 
Normal activity with effort; some 
signs or symptoms of disease. 
1 
Symptoms, but ambulatory. 
Restricted in physically strenuous 
activity, but ambulatory and able to 
carry out work of a light or 
sedentary nature (e.g., light 
housework, office work). 
70 
Cares for self, unable to carry on 
normal activity or to do active 
work. 
60 
Requires occasional assistance but 
is able to care for most of his/her 
needs. 
2 
In bed <50% of the time. 
Ambulatory and capable of all self-
care, but unable to carry out any 
work activities. Up and about more 
than 50% of waking hours. 
50 
Requires considerable assistance 
and frequent medical care. 
40 
Disabled, requires special care and 
assistance. 
3 
In bed >50% of the time. Capable of 
only limited self-care, confined to 
bed or chair more than 50% of 
waking hours. 
30 
Severely disabled, hospitalization 
indicated. Death not imminent. 
20 
Very sick, hospitalization 
indicated. Death not imminent. 
4 
100% bedridden. Completely 
disabled. Cannot carry on any self-
care. Totally confined to bed or 
chair. 
10 
Moribund, fatal processes 
progressing rapidly. 
0 
Dead. 
5 
Dead. 

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APPENDIX III: AIDS AND CANCER SPECIMEN RESOURCE (ACSR) SPECIMEN 
PREPARATION AND SHIPPING INSTRUCTIONS 
A. 
GENERAL 
To ship blood specimens, use a diagnostic shipper approved for a volume of at least 30 cc. 
The use of the SAF-T-PAK STP 210 diagnostic cardboard shipper is recommended. These 
shippers may be ordered at the SAF-T-PAK website: www.saftpak.com. The following 
instructions below are for use with the recommended STP-210 shipper. If using another 
federally approved diagnostic shipper, please follow instructions provided for that specific 
shipper. 
NOTE: Specimens MUST BE SHIPPED Mondays through Wednesday as an 
OVERNIGHT PRIORITY shipment. Specimens are NOT ACCEPTED ON FRIDAYS 
OR SATURDAYS in the ACSR. 
B. 
SPECIMEN PREPARATION, PACKAGING, AND SHIPMENT 
Blood specimens 
Draw two 8.5 cc (mL) yellow top [acid citrate dextrose (ACD)] tubes from study 
participant. With a black, water resistant, sharpie pen, label each specimen with the 
following information: 
• AMC Protocol # 097 
• AMC Participant ID# 
• Date and time of collection 
• Specimen type, (i.e., WB=Whole Blood, P=Plasma, S=Serum, or Tissue) 
• Specimen purpose: Donation 
Specimen shipment 
• Seal the tops of the two 8.5 cc yellow tops with parafilm. 
• Place the two sealed tubes into bubble wrap (provided in STP-210 kit). 
• Tape around the bubble wrap so that the roll stays together and the tubes cannot fall 
out or break. 
• Place absorbent material sheet around the bubble wrapped tubes and slip into a 
biohazard poly-bag and “self-seal.” 
• Place poly-bag containing tubes into the white TYVEK bag and seal. 
• Place the TYVEK bag into the STP-210 diagnostic cardboard shipper. Seal the 
cardboard shipper with clear packing/shipping tape. 
• Affix the FedEx airbill on blank side of the shipper making sure that it is marked 
“FedEx PRIORITY OVERNIGHT.” 
• Mark “OTHER” in the airbill under “Packaging.” Please use the FedEx # available on 
the AMC member’s only website. 

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• Under airbill section “Special Handling” indicate, “YES-SHIPPERS DECLARATION 
NOT REQUIRED.” 
• Place “From/To” information onto areas provided on the shipper. 
Blood specimens should be shipped by overnight express at room temperature to: 
Sylvia Silver, DA 
GW Biorepository 
George Washington Medical Center 
Ross Hall, Room 118 
2300 I St, NW 
Washington, DC 20037 
Phone: (202) 994-2945 
Fax: (202) 994-5056 
Email: ssilver@gwu.edu 
• Make certain that shipper is already either pre-labeled with ‘UN#3373’ stamp or make 
a paper label with ‘UN#3373” and affix it to the shipper. 
• Make certain that the net volume of the specimen being shipped is written in the space 
provided on the shipper or make a separate label with the volume in mL and affix to 
the shipper. 
• Affix airbill to shipper so that the ‘UN’ and ‘VOLUME’ labels are visible. 
• RETAIN THE TOP COPY OF THE AIRWAY BILL FOR YOUR RECORDS. 
• Place the box in the FedEx pickup area at your site or call to request a package pickup. 
Please Note: The shippers will be mailed back to each AMC site. 
INSTRUCTIONS FOR BLOOD SPECIMENS COLLECTED ON THURSDAY OR 
FRIDAY 
Preparation of plasma and mononuclear cells 
Refer to the ACSR’s SOP on Separation of Plasma and Mononuclear Cells on the AMC 
Operations web site for instructions on preparing plasma and PBMC aliquots. It is 
preferable that separation occurs as soon as possible. If necessary, whole blood in ACD 
(yellow top tubes) can be held at room temperature for no more than 24 hours. 
Freeze the cell suspension in 0.5 mL aliquots in sterile NUNC vials by placing the NUNC 
tubes in a room temperature, alcohol saturated, control rate freezer container and store in 
the -80°C freezer overnight. Transfer the cell suspension into the liquid nitrogen 
temperature freezer for long-term storage the next working day. 
***PLEASE DOUBLE CHECK PACKAGING OF SHIPPER AND DO NOT 
DEVIATE FROM REQUESTED LABELING. Shipping frozen aliquots requires the 
use of packaging acceptable for dry ice and Class 9 label with weight of dry ice written 

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on package. 
Preparation of tissue samples 
Tissue specimens to be fresh frozen should be placed in OCT and then on dry ice 
immediately. The specimens may stay on dry ice until being transferred to a -80ºC freezer. 
Tissue specimens for donation may be batched for shipping after storage in -80ºC freezer. 
*NOTE: Specimens can only be accepted Monday through Thursday. Therefore, 
specimens can only be shipped Sunday-Wednesday for delivery the next day. Shipping 
frozen tissue requires the use of packaging acceptable for dry ice and Class 9 label with 
weight of dry ice written on package. 
TISSUE specimens should be shipped by overnight express to: 
Sylvia Silver, DA 
GW Biorepository 
George Washington Medical Center 
Ross Hall, Room 118 
2300 I St, NW 
Washington, DC 20037 
Tel: (202) 994-2945 
Fax: (202) 994-5056 
Email: ssilver@gwu.edu 
C. 
RECORD OF SPECIMENS 
This study will track specimens via GlobalTraceSM, a component of the AMC 
AdvantageEDCSM system. The GlobalTraceSM shipment manifest must accompany all 
specimen shipments. 

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APPENDIX IV: ACSR INFORMED CONSENT 
Study Title for Study Participants: 
Collecting Blood and Tissue Sample Donations for 
Research for HIV/AIDS-Related Cancers 
Official Study Title: 
Biospecimen Collection and Donation to the AIDS  
and Cancer Specimen Resource (ACSR) 
What is the usual approach to donate blood and/or tissue to the ACSR? 
You are being asked to donate blood and/or tissue for future research. You are being asked to 
donate your blood and/or tissue samples to the ACSR because you have HIV infection and are 
being considered for participation in an AIDS Malignancy Consortium (AMC) clinical trial. The 
AMC works with the ACSR to collect donated samples from persons with HIV infection for 
research studies. People who do not take part in an AMC clinical trial can also donate samples to 
the ACSR. 
What are my other choices if I do not take part in this study? 
It is your choice to donate or not donate your blood and/or tissue samples. You may still take part 
in the AMC clinical study if you choose not to donate blood or biopsy samples to the ACSR.  
You may also choose to donate: 
• Blood but not tissue, or 
• Tissue but not blood. 
What is the AIDS and Cancer Specimen Resource (ACSR)? 
The ACSR is a biorepository (biobank) that collects human biological specimens (samples) from 
persons who have HIV or cancers related to HIV/AIDS. The ACSR stores the samples and some 
of the donor’s medical information for use by researchers in future research studies. The National 
Cancer Institute (NCI) has set up the ACSR to assist researchers locate samples needed for their 
studies. 
The ACSR has an independent research panel that approves researchers’ requests to use the 
ACSR’s stored samples for research studies. The ACSR only gives samples and medical 
information to researchers after their projects have been approved. Researchers may use the 
samples to study cancers and other diseases associated with HIV disease. This information may 
help us learn more about the causes of HIV-related diseases and cancers and to develop better 
ways to screen, diagnose, and treat them. 
Why is this study being done? 
The purpose of this study is to collect samples for the ACSR for future research studies. 
Researchers may study samples from the ACSR in combination with hundreds or thousands of 
other samples to explore how biologic or genetic factors may be related to HIV-related diseases 
and cancer. The information might help doctors in the future to identify who will or will not benefit 
from treatment. The samples may be used to learn more about how HIV-related diseases and 
cancers develop. The samples may also lead to new tests or discoveries. Finally, researchers may 
use the samples to study the genetic material from your cancer tissue and compare it to the material 
from your normal tissue (blood) to try to find the differences that exist. These studies could make 
it possible to identify many of the changes that are associated with diseases such as cancers. It may 

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also help us tailor treatments to a patient's unique genetic make-up and/or to the genetic markers 
of the tumors. 
What extra tests and procedures will I have if I take part in this study? 
1) If you agree to donate blood, the medical team will draw about 2 tablespoons of blood to give 
to the ACSR. This takes about 10 minutes. 
2) If you agree to donate tissue, your leftover tissue biopsy material will be donated to and stored 
by the ACSR. 
3) Some of your clinical information will be released to the ACSR and entered into their database. 
The information given to the ACSR will not include your name or any information that could 
personally identify you. 
We will only give the ACSR tissue that is left over after making decisions about your treatment or 
diagnosis. The study doctor will not take any extra biopsies just for the ACSR. 
We cannot tell you right now what future research these samples would be used for. Instead, we 
are asking that you give approval to give your samples for future testing without contacting you 
again. The results of whatever research is done on your samples will not be told to you or your 
doctor. The results of the tests will not be placed in your study records. 
How long will ACSR keep my samples? 
Your blood and/or tissue sample will be stored until it is used for research. The samples may be 
stored indefinitely. 
What possible risks can I expect from taking part in this study? 
• Blood Draw: The risks of drawing blood include temporary discomfort from the needle stick, 
bruising, and, rarely, infection. 
• Confidentiality: The ACSR will receive study samples with code numbers. There will be no 
personal identifiers on the samples. Then the samples will be re-labeled with a barcode and 
stored for future testing. While the ACSR and researchers who study ACSR samples will have 
no information that could identify you, there is a risk that someone could use information from 
genetic studies to trace your samples back to you. The researchers believe the chance that 
someone will identify you is very small, but the risk may change in the future as people come 
up with new ways of tracing information. In some cases, this information could be used to 
make it harder for you to get or keep a job. There are laws against misuse of genetic 
information, but they may not give full protection. The researchers believe the chance these 
things will happen is very small but cannot promise that they will not occur. 
Let your study doctor know of any questions you have about these possible risks. You can ask the 
study doctor questions about side effects at any time. 
What possible benefits can I expect from taking part in this study? 
This study is unlikely to help you. This study may help us learn things that may help people in the 
future. 
The information may help to identify those who are at increased risk and those who may benefit 
from targeted treatment and screening. In turn, these studies could help find ways to prevent or 
improve treatments for HIV-related diseases and AIDS-related cancers. 

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Can I stop taking part in this study? 
Yes, you may withdraw your samples from the ACSR at any time. You may contact your AMC 
study coordinator if you would like to withdraw your samples. The coordinator can ask in writing 
that your sample be removed from research use and that any identifiable sample and information 
still in their possession be destroyed. However, if any research has already been done using some 
of your samples, the data will be kept and analyzed as part of those studies. 
What are my rights in this study? 
Taking part in this study is your choice. No matter what decision you make, and even if your 
decision changes, there will be no penalty to you. You will not lose medical care or any legal 
rights. 
For questions about your rights while in this study, call the ________________________ (insert 
name of center) Institutional Review Board at __________________ (insert telephone number). 
(Note to Local Investigator: Contact information for patient representatives or other individuals 
at a local institution who are not on the IRB or research team but take calls regarding clinical 
trial questions can also be listed here.) 
What are the costs of taking part in this study? 
There will be no cost to you for donating your samples to the ACSR. You will not be paid for 
taking part in this study. 
What happens if I am injured or hurt because I took part in this study? 
If you are injured or hurt as a result of taking part in this study and need medical treatment, please 
tell your study doctor. The AMC will not offer to pay for medical treatment for injury. Your 
insurance company may not be willing to pay for study-related injury. If you have no insurance, 
you would be responsible for any costs. 
If you feel this injury was a result of medical error, you keep all your legal rights to seek payment 
for injury even though you are in a study. 
Who will see my medical information? 
Your privacy is very important to us and the researchers will make every effort to protect it. Your 
information may be given out if required by law. For example, certain states require doctors to 
report to health boards if they find a disease like tuberculosis. However, the researchers will do 
their best to make sure that any information that is released will not identify you. Some of your 
health information, and/or information about your specimen, from this study will be kept in a 
central database for research. Your name or contact information will not be put in the database. 
There are organizations that may inspect your records. These organizations are required to make 
sure your information is kept private, unless required by law to provide information. Some of these 
organizations are: 
• The AIDS Malignancy Consortium (AMC) 
• The Institutional Review Board, IRB, is a group of people who review the research with the 
goal of protecting the people who take part in the study. 
• The Office for Human Research Protections and the National Cancer Institute in the U.S. 

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To protect your privacy, the AMC does not keep identifying information that links study 
participants to specific samples. As a result, the AMC and ACSR will not be able to link the results 
from studies that use your samples back to you. Thus, information, including genetic information, 
that researchers may obtain in studies that use your samples may not be directly linked to you and 
will not be placed in your medical record. However, some clinical and basic information obtained 
confidentially from the AMC will be attached with these data. It is possible that findings may one 
day help, for example, people of the same race or sex as you. It also is possible that genetic factors 
might come to be associated with people who have HIV and cancer through these kinds of studies. 
Where can I get more information? 
You may visit the NCI Web site at http://cancer.gov/ for more information about studies or 
general information about cancer. You may also call the NCI Cancer Information Service to get 
the same information at 1-800-4-CANCER (1-800-422-6237). 
Who can answer my questions about this study? 
You can talk to the study doctor about any questions or concerns you have about this study or to 
report side effects or injuries. Contact the study doctor __________________ (insert name of study 
doctor[s]) at __________________ (insert telephone number). 
Please circle your answer to show whether or not you would like to take part in each option: 
1) I agree to donate my blood to the ACSR for future research that may be used to learn about, 
prevent, diagnose, or treat HIV-related diseases and cancer. 
YES 
 
NO 
2) I agree to donate my blood to the ACSR for future research that may include genetic testing to 
learn about, prevent, diagnose, or treat HIV-related diseases and cancer. 
YES 
 
NO 
3) I agree to donate some of my tissue biopsy material that is not required for my treatment or 
diagnosis to the ACSR for future research that may be used to learn about, prevent, diagnose, 
or treat HIV-related diseases and cancer. 
YES 
 
NO 
4) I agree to donate some of my tissue biopsy material to the ACSR for future research that may 
include genetic testing to learn about, prevent, diagnose, or treat HIV-related diseases and 
cancer. 
YES 
NO 
 
 

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My Signature Agreeing to Take Part in the Study 
I have read this consent form or had it read to me. I have discussed it with the study doctor and my 
questions have been answered. I will be given a signed copy of this form. I agree to take part in 
the optional study.  
Participant’s signature________________________________ 
 
Date of signature____________________________________ 
 
Signature of person(s) conducting the informed consent discussion________________________ 
 
Date of signature_____________________________________ 
 
 

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APPENDIX V: CONSENT FORM FOR GI BIOPSIES 
CONSENT TO PARTICIPATE IN A RESEARCH STUDY 
STUDY TITLE: A Pilot Project of Virologic, and Immunologic Correlates of Gastrointestinal- 
Associated Lymphoid Tissue Immune Reconstitution as a Part of Bone Marrow Transplant 
with Anti-HIV Gene Modified Stem Cells 
INTRODUCTION 
You are being asked if you want to take part in a research study. The main goal of a research 
study is to learn things to help patients in the future. No one can guarantee that a research study 
will help you. 
Participating in research is voluntary. You have the right to know about the procedures, risks, 
and benefits of the research study. If you decide to take part, you can change your mind later 
and leave the study. To participate in this study, you will need to give your written consent 
by signing this form. Please take your time to make your decision and discuss it with your 
family, friends, and caregivers. 
A minimum of seven people and a maximum of 18 people will take part in this study at 
University of California Davis and other AIDS Malignancy Consortium ( AMC) sites. 
WHY IS THIS STUDY BEING DONE? 
We are asking that you take part in a research study of how the immune system in the small 
intestines improves after your bone marrow transplant with anti-Human Immunodeficiency 
Virus (HIV-1) gene treated stem cells. We do this by taking biopsies from the part of the 
intestines just below the stomach. A biopsy is a sampling of small snips of tissue. The samples 
will be studied in the laboratory. 
The main purpose of this study is to measure any increase in the number of immune cells in 
the intestines to see if this number is related to the development of an immune system that is 
resistant to HIV-1 because of gene therapy. 
We are asking you to take part in this study because you participated in the Stem Cell Gene 
Therapy for HIV-1 in AIDS Lymphoma Patients protocol. 
You must be between the ages of 18 and 60. In order to participate in this study, it will be 
necessary to give your written consent. 
Up to 18 people who have participated in the Stem Cell Gene Therapy for HIV-1 in AIDS 
Lymphoma Patients protocol will take part in this study. Ten volunteers will be HIV-1 
negative and will only undergo a single set of procedures called upper endoscopy. This is 
when the doctor takes an endoscope to look into the intestines and take biopsies that can be 
taken to the laboratory for testing. Two sets of endoscopies (at approximately 6 months and 8 
months after transplant) will be performed. 
BEFORE YOU BEGIN THE STUDY 
The only additional test you need for this study (above and beyond the tests and exams that you 
have done) is a short medical history and physical exam by the doctor that performs the study. 

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A blood draw (approximately 1/3 of tablespoon of blood) will also be done by inserting a 
needle into a vein in your arm to make sure that it is safe to do the endoscopy. We will 
measure the clotting factors in your blood. 
If you decide to take part in this study, you will be asked to have the screening exams, tests 
or procedures mentioned above to show that you can continue to be in the study.  
WHAT WILL HAPPEN IF I TAKE PART IN THIS RESEARCH STUDY? 
If you decide to take part in this study, you will be asked to have a procedure called endoscopy. 
If you are an HIV-1 positive participant, you will be asked to have two sets of endoscopies: 
at 6 months after the transplant and at 8 months post-transplant. 
You will also have blood drawn at the time of endoscopy. We will also review your medical 
records with respect to the HIV-1 infection you have and your treatment for it. 
Endoscopy uses a small flexible rubber tube (about as big around as a pencil) connected to a 
video camera to examine the inside of your esophagus, stomach and small intestines. It is 
commonly used to diagnose and treat many conditions of the upper intestinal tract and we 
will use this tube to take biopsy samples by inserting a small device into the tube that can 
take a tiny piece of your gut. 
All participants will undergo the procedure in the same manner as any other patient having 
endoscopy. You will receive an IV injection of a pain medication and/or sedative (Demerol 
or Versed) and be placed on your side. The back of your throat will be numbed using 2% 
lidocaine gel (like numbing medication used by dentists). 
An experienced gastroenterologist (specialist in intestinal disease) will pass the flexible 
endoscope tube into your stomach and intestines. He/She will examine them by looking at the 
video camera image. He/She will collect biopsy samples (small pieces of tissue about the 
size of a grain of rice) from your small intestines in several places (up to 24 biopsies total). 
This tissue will be examined under a microscope and cells will be examined for certain 
chemicals that are thought to be important in HIV-1 infection. In addition, cells will be examined 
and counted for T-helper and other types of lymphocytes (the cells affected by HIV-1). Finally, 
the functions of the cells in your biopsy specimens will be measured and the amount of HIV-1 
virus and HIV-1 medication in the specimen will be determined. 
You must be fasting for 12 hours before the endoscopy procedures. You will be asked about your 
medications before the endoscopy, check with the study doctor or nurse to see if you may take 
your routine medications during the fasting period. Do not take aspirin, products, containing 
aspirin or anti-inflammatory agents like Motrin, Advil, or similar non-steroidal anti-inflammatory 
drugs (NSAID) medications (Tylenol/acetaminophen is permitted) for 7 days before and after the 
endoscopy procedures. Ask the study doctor what alternatives that you may take. The endoscopy 
procedure lasts about 30 minutes. 
You are sedated during the procedure, so it is not uncomfortable. 
You will be observed for any problems after the procedure and will be given an emergency 
phone number to call if you experience problems after you leave the endoscopy area. You 
will need to arrange for transportation to and from the endoscopy procedure so that you do not 
drive home after the procedure. Any medical observations of clinical importance will be 
discussed with you and forwarded, in writing, to your treating physician. 

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HOW LONG WILL I BE IN THE STUDY? 
The study is limited to the 2 time points for endoscopy mentioned above. There are no additional 
studies scheduled after that point. 
WHAT SIDE EFFECTS OR RISKS CAN I EXPECT FROM BEING IN THE STUDY? 
You may have side effects while on this study. Everyone taking part in the study will be watched 
carefully for any side effects. However, the researcher may not know all the side effects or 
risks. Side effects may be mild or very serious. The researchers may give you medicines to 
help lessen side effects. Many side effects go away. In some cases, side effects can be serious, 
long lasting, or may never go away. You should talk to the researchers about any side effects 
that you have while taking part in the study. 
Risks and side effects related to the procedures we are studying are listed below: 
Blood draw risks: You will feel some pain when we first insert the tube into your vein. You 
may have some redness, swelling, or bruising where the tube goes under your skin. In some 
cases, this type of tube can cause an infection where it goes under the skin. In rare cases, 
it can cause a blood clot in the vein. 
Endoscopy risks: The endoscopy is being performed as a part of a research project. The risks 
of endoscopy are remote, however, there can be pain, gagging, bleeding, infection, perforation 
(a hole through the bowel wall can occur) and rarely, death can occur. 
Sedation risks: The risks of intravenous sedation (putting someone to sleep) include bleeding, 
bruising, pain, infection from intravenous catheter, low blood pressure, decreased breathing, 
and allergic reaction to the medicine. 
Allergic reaction risks: Symptoms of allergic reaction may vary from individual to individual 
but often consists of generalized rash (red skin), itchiness, and a feeling of being excited or 
anxious. 
Very rarely swelling of the throat and/or difficulty breathing can accompany these symptoms. 
Occasionally (rare), midazolam (also called versed, a drug used for sedation) may cause a state 
of over excitement. The risks for small bowl biopsy are perforation and bleeding. These events 
have occurred in some patients though not with these physicians. These risks will be explained 
to you again by in detail prior to the endoscopy procedure by the gastroenterologist. 
Reproductive risks: You should not become pregnant or father a baby while on this study because 
the drugs in this study can affect an unborn baby. Women should not breastfeed a baby 
while on this study. It is important to understand that you need to use birth control while on 
this study. Check with your study doctor about what kind of birth control methods to use and 
how long to use them. Some methods might not be approved for use in this study. 
There may also be risks to your privacy. The researchers will store study records and other 
information about you in a secure location and will grant access only to those with a need 
to know. However, just like with other personal information kept by your health care 
providers, your banks, and others, even these safeguards cannot guarantee absolute protection of 
the data. If private information gets into the wrong hands, it can cause harm. Although rare, 
there are reported cases of breaches that have resulted in discrimination in insurance or 
employment. 

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For more information about risks and side effects, ask the researcher. 
ARE THERE BENEFITS TO TAKING PART IN THE STUDY? 
We do not expect that you personally benefit from participating in this study. However, we 
may learn more about how immune cells coming from stem cells treated with anti-HIV genes 
recover after transplant and how it reacts when challenged by HIV-1. 
WHAT ARE THE COSTS OF TAKING PART IN THIS STUDY? 
There is no charge for you to participate in this study. Neither you nor your insurance carrier 
will be charged for your taking part in the research. All costs associated with the study 
including medications and procedures will be paid by the research. 
WILL MY INFORMATION BE KEPT CONFIDENTIAL? 
We will do our best to make sure that the personal information in your medical record will 
be kept private. However, we cannot guarantee total privacy. Your personal information may 
be given out if required by law. 
There are organizations that may inspect your records. These organizations are required to make 
sure your information is kept private, unless required by law to provide information. Some of these 
organizations are: 
• The AIDS Malignancy Consortium (AMC) 
• The Institutional Review Board, IRB, is a group of people who review the research with the 
goal of protecting the people who take part in the study. 
• The Food and Drug Administration and the National Cancer Institute in the U.S. 
If we access protected health information (for example your medical record), you will be asked 
to sign a separate form to give your permission. Your medical records may become part of 
the research record. If that happens, your medical records may be looked at and/or copied by 
the sponsor of this study and government agencies or other groups associated with the study. 
Participation in research may involve a loss of privacy, but information about you will be 
handled as confidentially as possible. A research record will be created because of your 
participation in this study. Your consent form and some of your research test results will be 
included in this record. Therefore, your other doctors may become aware of your participation. 
Hospital regulations require that all health care providers treat information in medical records 
confidentially. 
Designated University officials, including the Institutional Review Board, and the Food & 
Drug Administration have the authority to review research records. 
If information from the study is published or presented at scientific meetings, your name 
and other personal information will not be used. 
DO I HAVE TO PARTICIPATE AND CAN I STOP BEING IN THE STUDY? 
Taking part in this study is your choice and completely voluntary. Participation or a decline 
in participation in this part of the study is independent of your participation in the stem cell 
gene therapy study. 

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If you decide to take part in this study, you can decide to stop at any time. Leaving the study 
will not affect your medical. Tell the researcher if you are thinking about stopping or decide 
to stop so any risks from the can be managed safely. 
The researcher may withdraw you from this research if circumstances arise which warrant doing 
so even if you would like to continue. 
We will tell you about new information or changes in the study that may affect your health 
or willingness to continue in the study. 
WILL I BE COMPENSATED FOR BEING IN THIS STUDY? 
For the optional intestinal biopsy (upper endoscopy), a fee of $300 for each procedure completed 
will be paid to you. 
WHO CAN ANSWER MY QUESTIONS ABOUT THE STUDY? 
If you have questions, please ask us. You can talk to the study doctor about any questions or 
concerns you have about this study. 
Contact the study doctor __________________ (insert name of study doctor[s]) at 
__________________ (insert telephone number). 
For questions about your rights while in this study, call the ________________________ (insert 
name of center) Institutional Review Board at __________________ (insert telephone number). 
(Note to Local Investigator: Contact information for patient representatives or other individuals 
at a local institution who are not on the IRB or research team but take calls regarding clinical 
trial questions can also be listed here.) 
WHAT HAPPENS IF I AM INJURED BECAUSE I TOOK PART IN THIS STUDY? 
If you are injured or hurt as a result of taking part in this study and need medical treatment, please 
tell your study doctor. The study sponsors will not offer to pay for medical treatment for injury. 
Your insurance company may not be willing to pay for study-related injury. If you have no 
insurance, you would be responsible for any costs. 
If you feel this injury was a result of medical error, you keep all your legal rights to receive 
payment for this even though you are in a study. 
It is important that you promptly tell the person in charge of the research if you believe that you 
have been injured because of taking part in this study. If you are injured as a result of being in this 
study, ________________________ (insert name of center) will provide necessary medical 
treatment. Depending on the circumstances, the costs of the treatment may be covered by 
________________________ (insert name of center), or may be billed to your insurance company 
just like other medical costs. The University and the study sponsor do not normally provide any 
other form of compensation for injury. For more information about compensation, you may call 
the ________________________ (insert name of center) Institutional Review Board at 
__________________ (insert telephone number). 
 
 
 

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My Signature Agreeing to Take Part in the Study 
I have read the consent form or had it read to me and understand the information above. I have 
discussed it with the study doctor and my questions have been answered I understand that I will 
be given a signed and dated copy of this consent form. I agree to take part in the study. My 
signature below indicates that I understand my rights and want to take part in this study as a 
research participant. 
 
Signature of Participant ______________________________________ 
Date_____________ 
 
Print Name of Participant _____________________________________ 
 
Signature of person(s) obtaining consent________________________ 
Date______________ 
 
Print Name of Person Obtaining Consent_________________________________ 
 
 

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APPENDIX VI: AMC DATA AND SAFETY MONITORING PLAN 
(Version 9.0  06OCT2020) 
 
Introduction 
The AIDS Malignancy Consortium (AMC) Data and Safety Monitoring Plan (DSMP) outlines the 
measures employed by the group to monitor the safety of participants and ensure the data validity 
and integrity for all clinical trials it conducts. This includes methods to: 1) monitor the progress of 
trials and the safety of participants; 2) comply with regulatory requirements for adverse event (AE) 
reporting; 3) processes for trial termination or temporary suspension and major modifications; and 
4) plans for ensuring data accuracy and protocol compliance. As the AMC conducts protocols of 
varying research phase, region of conduct (which may include trials conducted in the U.S., 
international sites, or both), IND sponsor (AMC investigator, CTEP, or industry-sponsored) and 
clinical data entry system use, this plan addresses broad processes applying to the range of trial 
designs and requirements. Refer to the individual AMC protocol to identify the applicable study 
characteristics for the relevant requirements described in this plan. 
Monitoring the Progress of Trials and the Safety of Participants 
Routine and expedited AE reporting 
All AMC protocols that collect safety data adhere to the National Cancer Institute (NCI), Cancer 
Therapy Evaluation Program (CTEP) Guidelines: Adverse Event Reporting Requirements 
(https://ctep.cancer.gov/protocolDevelopment/adverse_effects.htm), as applicable to the clinical 
protocol. AEs are to be recorded in the source documents, assessed by a clinical investigator for 
the AE reporting criteria, and promptly reported in the clinical data entry system as required by 
each protocol. For AMC trials conducted under a CTEP IND and AMC trials conducted within the 
U.S., all AEs that meet the NCI’s expedited reporting requirements are reported to the NCI via the 
CTEP Adverse Event Reporting System (CTEP-AERS) web application, either directly or through 
integration with Medidata Rave where this system is employed for AMC protocols. Use of this 
system ensures notification to the protocol chair and Investigational Drug Branch (IDB) at CTEP, 
as required for trials conducted under a CTEP IND, and a uniform expedited reporting and safety 
review process for AMC domestic trials. The system may also be programmed to include sponsor 
notification as required for trials with industry support. Alternate process for expedited AE 
reporting to the AMC protocol chairs and AMC Operations and Data Management Center 
(ODMC) within the clinical data entry system (AdvantageEDC or Advantage eClinical only) may 
be defined in the protocol for select trials (international studies and The ANCHOR Study). 
All serious adverse events (SAEs) received by the AMC ODMC will be reviewed by the AMC 
medical monitor at the AMC ODMC for consideration of individual participant safety, safe trial 
conduct, data reporting quality for AE term selection, and appropriate application of the regulatory 
criteria for seriousness, expectedness, and relatedness to the investigational therapy. If alternate 
procedures are followed for SAE review, the process for adequate medical monitoring will be 
defined in the AMC protocol and the Transfer of Regulatory Obligations (TORO) with the sponsor. 
AMC medical monitor review includes review of the CTEP-AERS report before CTEP submission 
for IDB review (if applicable), or review of the SAE report in the data entry system for trials not 
using CTEP-AERS for expedited reporting. The IND sponsor or its designee will issue the 
determination as to whether the AE requires IND safety reporting to FDA as a serious and 

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unexpected suspected adverse drug reaction (SUSAR). For protocols not conducted under an IND, 
in the event of disagreement between the reporting physician and the AMC medical monitor 
regarding the relationship of the AE to the investigational agent(s) (i.e., determination of whether 
the attribution is unrelated or unlikely, or possible, probable, or definite), the AMC medical 
monitor will provide the final determination of the relationship. IND safety reporting to FDA is 
performed by CTEP for trials conducted under a CTEP IND; IND safety reporting is performed 
by the sponsor or sponsor’s designee (AMC ODMC or other party defined in the study agreement 
or TORO) for IND studies sponsored by AMC investigators or industry sponsors. 
Expedited reporting to the Institutional Review Board (IRB) 
The requirements for IRB review will be identified in the protocol section on ethical and regulatory 
obligations. All AMC trials initiated before September 1, 2020 and all international sites for all 
AMC studies are subject to local IRB review; only U.S. sites are subject to the NCI requirement 
to use a single IRB for protocols initiated on or after September 1, 2020. For trials subject to local 
IRB review, the site principal investigator is responsible for ensuring that expedited AE reports 
for its trial participants and any unanticipated problems that affect the local institution only are 
submitted to the local IRB of the reporting institution, per the local IRB’s requirements for such 
reporting. For studies reviewed by the single IRB, the protocol chair will render a determination 
as to whether a SAE or other problem constitutes a trial-wide unanticipated problem that requires 
reporting to that IRB, in accordance with its standards of procedure.  
To comply with investigator notification requirements for IND studies under 21 CFR 312.32 and 
312.55, IND safety reports from all trials the AMC conducts and reports from external sponsors 
investigating the same agents are made available to all investigators upon receipt from the sponsor 
or its designee, either via the password-protected section of the AMC Operations web site (AMC 
trials subject to local IRB review only) or the CTSU website (U.S. trials subject to single IRB 
review/CTEP IND agents). The site clinical investigator responsible for the applicable AMC 
protocol(s) is responsible for reviewing any IND safety reports received and documenting 
submission to the IRB of record (if required by local policy) within the timeline defined by the 
Clinical Trials Monitoring Branch (CTMB) audit guidelines. 
Procedures for monitoring trial progress and pharmacovigilance 
For trials using AdvantageEDC or Advantage eClinical for clinical data entry, the AMC ODMC 
provides on demand tabular listings of all reported AEs and SAEs on a participant level to the 
protocol chair and co-chair(s) for review via the password-protected section of the AMC 
Operations web site, www.AIDScancer.org. For trials using OPEN and Medidata Rave for clinical 
data collection, data listing will be made available using that system. Summary reports of AEs by 
frequency and relationship to the investigational agent(s) are provided to all AMC investigators 
and their staff It is the responsibility of each site to provide trial-specific AE listings to their 
respective IRB, if required by its policies. For blinded studies, the AE and SAE listings are 
reviewed and tabulated without treatment assignment. 
Accrual summaries for each AMC trial are updated nightly on the password-protected section of 
the AMC web site. The progress of each AMC trial is reviewed regularly by the protocol chair and 
also by the appropriate Scientific Working Group (SWG) during scheduled conference calls 
(monthly SWG calls and as required, protocol-specific monitoring conference calls). Summary 
accrual, summary AE, and individual SAE reports are provided to SWG leadership and protocol 
chairs to monitor participant safety during these monthly calls. 

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The AMC medical monitor reviews listings of all reported AEs on a quarterly basis for assuring 
compliance with the protocol requirements for AE reporting and the identification of any safety 
concerns (individual AE or increased frequency/severity of expected AEs) for the agents under 
investigation. Findings from these reviews are communicated to the protocol chairs and all AMC 
investigators, and posted to the AMC Operations web site. 
Data and Safety Monitoring Board (DSMB) review 
The AMC has formed an independent Data and Safety Monitoring Board (DSMB) for AMC trials 
and for the ANCHOR Study. As required by NCI policy, the AMC requires DSMB review for all 
phase III randomized trials. All other clinical trials that the AMC initiates will be reviewed by the 
AMC ODMC and AMC Statistical Center during protocol development to issue a recommendation 
as to whether the study requires DSMB oversight, which will require the approval of the AMC 
Executive Committee. This determination  will be based on the phase of the study, experimental 
design, risk posed by the investigational approach, extent of data available on the safety of an 
investigational agent, risk posed by the natural course of the health condition under research, and 
the categories of vulnerable populations involved. The involvement of a DSMB in reviewing an 
AMC protocol will be identified in each clinical protocol as approved by CTEP and, as applicable, 
required by the IRB of record. 
Regarding the composition of the AMC DSMB, voting members usually include physicians, 
statisticians, an ethicist, and a patient advocate. All voting members have no other affiliation to 
the AMC and are appointed by the AMC Executive Committee with the approval of the OHAM 
Director. Nonvoting members are the AMC group statistician, the protocol statistician, an AMC 
ODMC staff member, two representatives (normally a clinician or statistician) from CTEP, and 
the grant program directors from the NCI Office of HIV and AIDS Malignancy (OHAM). 
The DSMB reviews all applicable AMC studies in accordance with the National Cancer Institute’s 
Policy for Data and Safety Monitoring. Confidential reports of all trials under review are prepared 
by the AMC group statistician with support from the AMC ODMC. A written report containing 
the current status of each trial monitored, and when appropriate, any toxicity and outcome data, 
are sent to DSMB members by the AMC ODMC within the timelines specified by the DSMB 
charter. This report addresses specific toxicity issues and any other concerns about the conduct of 
the trial, as defined by the protocol plan for DSMB review. The report may contain information 
for the DSMB to render determinations for participant safety, early trial termination, results 
reporting, or continuing accrual or follow-up. 
The results of each DSMB meeting are summarized in a formal report sent by the DSMB chair to 
the AMC group chair and AMC ODMC. The DSMB report contains recommendations on whether 
to close each study reviewed, whether to report the results, and whether to continue accrual or 
follow-up. A primary recommendation (e.g., continue with no change; recommended or required 
modification; stop) must be included in the document. The group chair or designee is then 
responsible for notifying the protocol chair and relevant SWG chair before the recommendations 
of the DSMB are carried out. In the unlikely event that the protocol chair does not concur with the 
DSMB, then the OHAM program directors and the NCI division director or designee must be 
informed of the reason for the disagreement. The protocol chair, relevant SWG chair, group chair, 
DSMB chair, and NCI division director or designee will be responsible for reaching a mutually 
acceptable decision about the study. CTEP approval of a protocol amendment will be required 
prior to any implementation of a change to the study. 

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Following a DSMB meeting, the DSMB’s recommendations are provided to all AMC investigators 
and staff. It is each site principal investigator’s responsibility for conveying this information to its 
local IRB as relevant for its protocol participation. For trials reviewed by a single IRB, the AMC 
ODMC will support notification to the IRB as required per its procedures. 
Cohort trial reviews not subject to DSMB review 
For phase I dose escalation trials, dose escalation (or dose de-escalation) is based on the rules in 
the protocol and the protocol chair, AMC medical monitor, and protocol statistician determine 
whether these criteria have been met based on a review of all safety data for the protocol-defined 
evaluation period. If applicable for phase II trials, stopping the trial for toxicity or efficacy, or 
suspending enrollment pending observation of responses in a multi-stage phase II trial, is based on 
meeting criteria stated in the protocol, and the protocol chair, AMC medical monitor, and protocol 
statistician determine whether these criteria have been met. 
Plans for Assuring Compliance with Requirements Regarding AE Reporting 
The protocol chair, AMC group chair, and the AMC ODMC share responsibility in assuring that 
participating investigators comply with applicable regulatory and protocol requirements for AE 
reporting. The AMC site principal investigator certifies compliance with NCI and FDA 
requirements for trial conduct by signing the site subaward agreement for the grant and the AMC 
Adherence Statement for site membership; clinical investigators also certify compliance in 
completing the protocol signature page for each protocol active at the site, and Form FDA-1572 
for CTEP investigator registration, and also for AMC IND studies sponsored by AMC 
investigators or industry sponsors. Protocol compliance with AE identification, assessment and 
reporting requirements is assessed by the AMC ODMC using several methods: 1) programmed 
system checks and messages to instruct the site to complete routine and/or expedited reporting 
when certain criteria are reported in the clinical data entry system; 2) programmed data reports 
provided to the protocol chairs that identify reports requiring expedited AE reporting; 3) remote 
review of data entry or data reports to ensure compliance with protocol and NCI AE reporting 
requirements; 4) AMC medical monitor review described in the section above; and, 5) routine site 
audits by reviewing the site’s source documentation. 
The clinical data entry systems used for AMC studies include the Oncology Patient Enrollment 
Network, OPEN for enrollment, and Medidata Rave for clinical data entry for enrolled 
participants; trials activated before September 1, 2020 or that involve only AMC international sites 
may be reported in AdvantageEDC/Advantage eClinical, a web-based data entry and enrollment 
system. These data entry systems are programmed to notify the site investigator, protocol chair, 
AMC medical monitor, and AMC ODMC via email in the event that a site reports an AE that 
meets expedited reporting criteria to NCI and/or FDA. Additional reporting conditions may be 
programmed depending on the sponsor reporting requirements of a given protocol (e.g., adverse 
events of special interest [AESI]). If the site does not follow with an expedited report, the AMC 
ODMC contacts sites to request compliance with reporting requirements. Additionally, the 
protocol chair, AMC ODMC, and the AMC medical monitor review reported AEs on a routine 
basis to identify AEs reported by sites that require expedited reporting. The protocol chair, AMC 
SWG chairs, AMC group chair, and IND sponsors have general oversight for assuring that routine 
and expedited adverse reporting requirements are met by the responsible parties. 
For studies monitored by CTEP using the Data Mapping Utility (DMU), cumulative protocol- and 
patient-specific data will be submitted weekly to CTEP electronically via the DMU. For trials 

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monitored by the NCI’s Clinical Data Update System (CDUS), AE information is transmitted 
electronically to NCI on a quarterly basis. For trials monitored by NCI’s Clinical Trials Monitoring 
Service (CTMS), AE information is transmitted electronically to NCI every two weeks. 
Plans for Assuring that any Action Resulting in a Temporary or Permanent Suspension of 
an NCI-Funded Clinical Trial is Reported to the NCI Grant Program Director Responsible 
for the Grant 
In the event that temporary or permanent suspension of a trial, or major modification to the 
protocol is under consideration, the protocol chair will convene the AMC ODMC, AMC Statistical 
Center, and SWG chair by conference call to discuss the options. Suspension actions will also be 
reviewed by the AMC Executive Committee for program oversight and direct communication of 
the action with the OHAM program directors. For phase III trials, closure decisions are typically 
rendered by the AMC DSMB; if the trial in question is under AMC DSMB oversight but rendered 
by the AMC investigators, the AMC DSMB will be notified of the suspension and the reason. For 
phase I and II trials, the protocol chair also has the option of asking the DSMB to review the study. 
The AMC ODMC will inform the CTEP Protocol Information Office (PIO), with copy to OHAM 
Directors, when studies are temporarily or permanently closed. In the event of major trial 
modification, CTEP must approve all protocol amendments prior to distributing to the AMC sites. 
Plans for Assuring Data Accuracy and Protocol Compliance 
All study data for AMC clinical trials are entered directly by AMC clinical site staff into the 
applicable clinical data entry system for the trial. During data entry, the system performs validation 
checks on many fields and performs consistency checks between select fields. Range checks are 
placed on each field to eliminate entry of out-of-range values. Edit check programs are run on the 
database on a set schedule to identify and resolve inconsistencies between forms or data collected 
at different points in time. Submitted data entry forms are reviewed for compliance with the 
protocol and data entry instructions according to the AMC ODMC’s standards for data quality 
processes. AMC ODMC staff routinely interacts with site staff to resolve any data submission 
problems. 
In accordance with NCI guidelines, the AMC ODMC conducts audits at the AMC sites to 
evaluate compliance with regulatory issues, and to review data for specific cases by checking 
source documents. These reports are sent to the site principal investigator and to the NCI. In the 
event that major violations are identified, sites are asked to provide a written corrective and 
preventative action plan to correct deficiencies. If needed, a repeat site audit is conducted. In the 
event that a site does not correct deficiencies in a pre-determined time frame, the AMC 
Executive Committee has the option to implement remedial action(s) for the site. Possible 
actions include, but are not limited to, suspending enrollment of new patients to AMC trials until 
deficiencies are corrected; recommending a decrease in funding to the site; and requiring specific 
training for site investigators or staff members. 
 
 

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APPENDIX VII: CORRELATIVE STUDY SPECIMEN PREPARATION AND 
SHIPPING INSTRUCTIONS 
DNA and Immunological Monitoring studies for Stem Cell Gene Therapy for HIV Mediated by 
Lentivector Transduced, Pre-selected CD34+ Cells in AIDS-lymphoma participants 
Biomarker 
Name, 
Laboratory 
Conducting 
Assay and 
Laboratory 
Director 
Assay 
(CLIA: 
Y/N) 
Use and  
 Purpose 
Tissue/Body 
Fluid Tested 
and Timing of 
Assay 
Tube Type, 
Blood 
Component 
Needed, Whole 
Blood 
Collection 
Volume, and 
Aliquot Volume 
Timing of Analysis, 
(conducted during 
the trial or after the 
trial) 
Funding 
Source(s) 
Study T 
Lymphocytes and 
their subsets  
Pollard, UC Davis 
Flow 
Cytometry 
CLIA: N 
Exploratory 
To evaluate the 
development of 
normal T cell 
subsets 
Blood 
Baseline, 3, 6, 
12, 24 months 
post-transplant, 
Early Disc., and 
Off-Study Disc.  
White blood cell 
component  
EDTA whole 
blood, 2-mL, 
purple top, and 
(PBMCs), from 
18-mL EDTA, 
purple top 
Analyses will be 
performed during the 
trial. Samples should 
be shipped to the 
Pollard lab upon 
collection. 
CIRM 
Cytokine studies 
using stored 
plasma and serum, 
Pollard, UC Davis 
ELISA 
CLIA: N 
Exploratory to 
evaluate the level 
of cytokines 
related to a 
chronic 
inflammatory 
status 
Blood 
Baseline, 3, 6, 
12, 24 months 
post-transplant, 
Early Disc., and 
Off-Study Disc. 
Serum from 5 
mL Red top, 
stored as 0.5 mL 
aliquots 
10 mL Plasma 
(from EDTA 
blood), stored as 
1 mL aliquots 
Plasma and Serum 
samples will be 
aliquoted and stored 
for delayed analysis 
CIRM 
Functional 
Responses Upon 
Reconstitution of 
T Cell Immunity 
Pollard, UC Davis 
Flow 
cytometry, 
assessment 
of response 
to antigen 
CLIA: N 
Exploratory 
To evaluate the 
development of 
functional 
immune cells 
Blood 
Baseline, 3, 6, 
12, 24 months 
post-transplant, 
Early Disc., and 
Off-Study Disc. 
White blood cell 
component 
(PBMCs), 
EDTA, purple 
top, from 20 mL 
aliquot 
Analyses will be 
performed during the 
trial. Samples should 
be shipped to the 
Pollard lab upon 
collection. 
CIRM 
Monitoring for 
Insertional 
Oncogenesis and 
Clonality of 
Hematopoiesis 
after Gene 
Therapy 
Anderson, UC 
Davis 
LAM-PCR 
CLIA: N 
Exploratory 
To determine if 
clones of 
transduced cells 
have developed 
Blood 
1, 2, 4, 6, 8, 10, 
12, 18, 24 
months post-
transplant, 
Early Disc., and 
Off-Study Disc. 
White blood cell 
component 
(PBMCs), 
EDTA, purple 
top, 10 mL 
aliquot 
Analyses will be 
performed during the 
trial. Samples should 
be shipped to the 
Pollard lab upon 
collection and will be 
transferred to the 
Anderson lab for 
analysis.  
CIRM 

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Biomarker 
Name, 
Laboratory 
Conducting 
Assay and 
Laboratory 
Director 
Assay 
(CLIA: 
Y/N) 
Use and  
 Purpose 
Tissue/Body 
Fluid Tested 
and Timing of 
Assay 
Tube Type, 
Blood 
Component 
Needed, Whole 
Blood 
Collection 
Volume, and 
Aliquot Volume 
Timing of Analysis, 
(conducted during 
the trial or after the 
trial) 
Funding 
Source(s) 
DNA monitoring 
studies 
Anderson, UC 
Davis 
PCR, qPCR, 
sequencing 
CLIA: N 
Exploratory 
To evaluate in 
vivo gene 
marking, anti-HIV 
gene expression, 
sequence analysis, 
vector stability 
analysis 
Blood 
Baseline, 1, 2, 
4, 6, 8, 10, 12, 
18, 24 months 
post-transplant, 
Early Disc., and 
Off-Study Disc. 
White blood cell 
component 
(PBMCs), 
EDTA, purple 
top, 5 mL aliquot 
Analyses will be 
performed during the 
trial. Samples should 
be shipped to the 
Pollard lab upon 
collection and will be 
transferred to the 
Anderson lab for 
analysis. 
CIRM 
Monitoring for 
immune response 
to TRIM5α 
Anderson, UC 
Davis 
ELISA 
CLIA: N 
Exploratory 
To evaluate 
whether any 
immune response 
is occurring with 
the expression of 
the chimeric 
TRIM5α protein 
Blood 
1, 2, 6, 12, 18, 
24 months post-
transplant, 
Early Disc., and 
Off-Study Disc. 
White blood cell 
component 
(PBMCs), 
EDTA, purple 
top, 10 mL 
aliquot 
Analyses will be 
performed during the 
trial. Samples should 
be shipped to the 
Pollard lab upon 
collection and will be 
transferred to the 
Anderson lab for 
analysis. 
CIRM 
Monitoring of 
changes in the 
HIV reservoir and 
viral diversity 
Henrich, UC San 
Francisco 
Peripheral 
blood and 
leukapher-
esis 
cells/plasma 
CLIA: N 
Exploratory 
To characterize 
changes in HIV 
reservoir/diversity 
Blood; 
baseline, 2 and 
10 weeks then 
6, 12- and 24-
months post-
transplant 
Double spun 
plasma and 
EDTA PBMC  
(2 x 107) 
 
Analysis during 
study, shipped to the 
Henrich Lab at UC 
San Francisco 
Dr. Henrich 
Lab  
Internal 
Funding 
Monitoring of 
mixed lymphocyte 
reactions 
Henrich, UC San 
Francisco 
Peripheral 
blood and 
Leukapher-
esis CD34+ 
depleted 
cells; PBMC 
CLIA: N 
Exploratory 
To monitor for 
reaction to 
transformed cells 
Blood 
baseline, 2 and 
10 weeks then 
6, 12- and 24-
months post-
transplant 
CD34+ depleted 
leukapheresis 
cells 
EDTA PBMC  
(2 x 107) 
Analysis during 
study, shipped to the 
Henrich Lab at UC 
San Francisco 
Dr. Henrich 
Lab 
Internal 
Funding 

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Biomarker 
Name, 
Laboratory 
Conducting 
Assay and 
Laboratory 
Director 
Assay 
(CLIA: 
Y/N) 
Use and  
 Purpose 
Tissue/Body 
Fluid Tested 
and Timing of 
Assay 
Tube Type, 
Blood 
Component 
Needed, Whole 
Blood 
Collection 
Volume, and 
Aliquot Volume 
Timing of Analysis, 
(conducted during 
the trial or after the 
trial) 
Funding 
Source(s) 
GI Biopsy 
IFA, PCR 
and RT-PCR 
and FACS, 
IHC 
Exploratory to 
evaluate the 
response of 
immune system 
following 
reconstitution of 
new anti-HIV 
hematopoietic 
cells. To evaluate 
the persistence of 
HIV in HIV 
dormant 
reservoirs 
following 
transplant 
Endoscopy 
biopsy samples 
will be obtained 
at 6 months 
post-transplant 
for the group 
that will not 
undergo ART 
withdrawal or 
any time before 
ART 
withdrawal and 
at 8 weeks later.  
Separate samples 
will be obtained 
for IHC, snap 
freezing or to put 
on RNA later for 
DNA and RNA 
analysis 
Fresh samples will be 
run immediately after 
receiving the samples 
by courier. Frozen 
samples will be 
stored at UC Davis 
for future tests.  
CIRM 
Bone Marrow 
Aspirate 
CFU assay, 
DNA 
extraction, 
quantitative 
PCR 
CLIA:N 
 
To evaluate the in 
vivo gene marking 
of human CD34+ 
cells 
 
Residual 
Samples of 
Bone marrow 
aspirate 
Two 10.0 ml 
EDTA / (Purple 
top tubes) 
 
3- and 12-months 
post-transplant, 
treatment 
discontinuation 
CIRM 
 
 

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SPECIMEN COLLECTION AND PROCESSING INSTRUCTIONS 
The correlative studies can be divided into immunological and HIV studies, DNA monitoring 
studies, and monitoring for oncogenesis and hematopoietic clonality. The immunological studies 
will be done in 2 compartments: peripheral blood samples and endoscopy (gut mucosa) samples. 
1. 
Blood Samples 
Samples will be collected via blood draws from participants (Maximum of 65 mL of blood 
collected). 
Blood samples for immunological studies 
A total of 45 mL of blood will be collected for immunological studies: 
• 40 mL EDTA anti-coagulated whole blood specimens and divided into four 10 mL 
tubes. Whole blood (for PBMCs) is required from each participant at each time point 
described in the clinical protocol. 
• 5mL of non-anticoagulated blood (processed for ~ 3 mL of serum) in one 5 mL tube 
red top tube 
Processing procedures red top tube serum 
• Collect 5 mLs of whole blood in a 5 mL red top tube. 
• After blood is drawn, invert collection tube 5 times and allow the blood to clot 60 
minutes, leaving the tube in an upright position during the clotting process for 
immediate processing. If multiple samples are collected in one day, and the site would 
like to hold the sample for processing later the same day, store the sample between 2-
8oc until ready to invert the tube and clot the sample. 
Once clotted, the collection tube should not be allowed to sit at room temperature for 
more than 1 hour prior to centrifugation and separation of the serum from the clot. 
Centrifuge the collection tube for 10 minutes at 1000 g. After separation, the serum 
should be clear and free from all blood cells. Aliquot the serum in the 3 mL cryovial. 
• Separated sera aliquots must be stored refrigerated until ready for shipment to UC 
Davis on next available business day (specimens should be shipped MONDAY 
through THURSDAY). 
Each sample tube should be labeled with GlobalTraceSM specimen labels using a Sharpie 
pen to include the following information: 
• 11-digit Participant # 
• Date and time of collection 
• Specimen type: (i.e., WB=Whole Blood, S=Serum) 
• Specimen purpose: (i.e., Immunological Studies) 
Blood samples for DNA monitoring studies 
A total of 20 mL of blood will be collected for DNA monitoring studies (see Section 2.5.4 
for analyses to be performed): 

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• 20 mL of EDTA anti-coagulated whole blood specimens in two 10 mL tubes will be 
collected. 
Each sample tube should be labeled with GlobalTraceSM specimen labels using a Sharpie 
pen to include the following information: 
• 11-digit Participant # 
• Date and time of collection 
• Specimen type: (i.e., WB=Whole Blood) 
• Specimen purpose: (i.e., DNA Monitoring Studies) 
Blood samples for safety monitoring studies 
A total of 30 mL of blood may be collected for safety monitoring studies, as determined 
with the protocol chair per Section 2.5.8. If determined to be required, 30 mL of EDTA 
anti-coagulated whole blood specimens in three 10 mL tubes will be collected and shipped 
to the Pollard lab as noted below for blood samples. 
Each sample tube should be labeled with GlobalTraceSM specimen labels using a Sharpie 
pen to include the following information: 
• 11-digit Participant # 
• Date and time of collection 
• Specimen type: (i.e., WB=Whole Blood) 
• Specimen purpose: (i.e., Safety Monitoring Studies) 
Blood specimen shipment 
All Blood Samples should be shipped by priority overnight express on MONDAY through 
Thursday to the UC Davis correlative study laboratory. 
Refrigerated Blood Specimen Protocol 
• Apply a strip of Parafilm, around all vials, where the cap meets the tube. 
• Wrap all blood tubes individually with bubble wrap. 
• Place all specimens in the clear plastic biohazard bag with an absorbent pad and seal. 
• Place the clear plastic biohazard bag in the white Tyvek biohazard bag and seal. 
• Place the sealed white Tyvek biohazard bag in the inner cardboard box of the shipper. 
• Place the inner cardboard box in the Styrofoam cutout, put the notched flap down first, 
and fold across. Remove your gloves. 
• Place four frozen cold packs in the Styrofoam box, one on each side of the inner 
cardboard box. Thermal protective gloves are recommended. 
• Place the lid on the Styrofoam box and put the Styrofoam box in the outer cardboard 
shipper. 
• Place the shipping manifest on the top of the closed Styrofoam box. 

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• Close and tape the box along the center break and on the sides. Secure the shipping 
label to the top of the box. 
NOTE: FedEx’s “Priority Overnight” is required shipping method. 
• It is the responsibility of the Primary Investigator to ensure that all staff personnel who 
will be handling, packaging, and/or shipping clinical specimens act in conformance 
with International Air Transport Association (IATA) regulations (IATA Packing 
Instruction 650) relating to the handling and shipping of hazardous goods. (IATA 
Packing Instruction 650 is located on the AMC web site). 
• Use a federally approved shipper for biological substance shipment (Category B). 
Label the shipper with the "Infectious substance" diamond shaped label. On one side, 
in black marker write “Biological Substance, Category B, UN 3373”, your name or 
name of responsible person, date of collection and phone number of the person 
responsible for the package. Package and label the shipment in accordance with the 
instructions provided for that specific shipper. 
• A Shipper’s Declaration for Dangerous Goods is not required. 
Please Note: The shipper will be mailed back to the AMC site. The STP-100 SAF-T-PAK 
shipper (VWR Cat # 11217-082) is a complete kit (bubble wrap, absorbent paper, labels, 
etc.) for specimen shipment. To reuse the shipper, you will need new labels, wrap, etc. 
There is a refurbishment kit with extra bubble wrap and absorbent material (Fisher 
Scientific Cat # 19-038-301E). 
Sample receipt and processing are performed Tuesday through Friday, unless other 
arrangements have been made for with the lab. Avoid collecting specimens for shipment 
on Fridays, and on days before an observed UC Davis holiday. All specimens should be 
shipped to: 
Dr. Joseph Anderson 
University of California Davis  
2921 Stockton Blvd., Room 1300 
Sacramento, CA 95817 
Tel: (916) 703-9300 
Email: jsanderson@ucdavis.edu 
 
2. 
Blood, plasma and leukapheresis for mixed-lymphocyte reaction analysis and HIV 
reservoir characterization. 
Samples will be sent same day by special courier by arrangement of Dr. Henrich and sent 
to:  
Henrich Laboratory 
University of California, San Francisco 
1001Potrero Ave., Bldg. 3, Rm 603 

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San Francisco, CA 94110  
Email: Timothy.Henrich@ucsf.edu 
 
 

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3. 
Endoscopy Samples 
Participants will have upper endoscopy performed in Gastrointestinal Suites 
according to standard clinical procedures: 
Participants are sedated with IV Benadryl, versed, and Demerol to achieve conscious 
sedation while being monitored for vital signs and comfort levels (heart rate and rhythm, 
blood pressure, O2 saturation). When participants are adequately sedated, an Olympus 
upper endoscope is passed to the small intestines where 24 biopsies are obtained using 
Boston Scientific biopsy forceps (large 2.8 mm). This typically requires 12 passes to 
acquire adequate samples for subsequent studies. 
Sample disposition during the procedure is as follows: 
A total of 24 endoscopy (gut mucosa) samples will be collected. 
Endoscopy samples for RNA & DNA analysis 
• A total of 8 biopsy specimens should be collected and snap frozen: 
o Six of the eight biopsy specimen should be transferred overnight on dry ice to UC 
Davis. 
o Two of the eight biopsy specimen should be placed in 1 mL of RNAlater Qiagen 
and sent over night to UC Davis with chilled packaging. 
Endoscopy samples for IHC and in situ staining 
• A total of 3 biopsies should be collected and placed in formalin per clinical practice. 
• These are transported to the Pathology laboratory for paraffin embedding per clinical 
practice. 
Endoscopy samples for vector frequency, immunologic flow cytometry analysis, and 
cellular HIV DNA measurement 
• A total of 13 biopsies are collected and placed in chilled 30 mL of RPMI media and 
transported overnight with chilled packaging to UC Davis. 
• These are transported to the UC Davis immunology laboratory for collagenase 
digestion and single cell suspension. 
Each sample tube should be labeled with GlobalTraceSM specimen labels using a Sharpie pen to 
include the following information: 
• 9-digit Participant # 
• Date and time of collection 
• Specimen type: (i.e., WB=Whole Blood) 
• Specimen purpose: (i.e., DNA Monitoring Studies) 
Participants are monitored in the Endoscopy Suites recovery area and are released to a companion 
for transport home with specific instructions about who to contact from the study team should any 
concerns or side effects arise. 
 
 

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Endoscopy Samples obtained at UC Davis Medical Center will be transported directly from the 
endoscopy suite to the laboratory. Samples from UC San Francisco will be transported by special 
courier as has been performed by previous clinical protocols. This is achieved by car from San 
Francisco and air from San Diego. 
ENDOSCOPY SPECIMEN SHIPMENT 
Frozen endoscopy specimen protocol 
• Apply a strip of Parafilm, around all vials, where the cap meets the tube. 
• Place all specimens in the inner plastic biohazard bag with an absorbent pad and seal. 
• Place the clear plastic inner biohazard bag in the white Tyvek biohazard bag and seal. 
• Place the sealed white Tyvek biohazard bag in the inner cardboard box of the shipper. 
• Place the inner cardboard box in the Styrofoam cutout, put the notched flap down first, and 
fold across. Remove your gloves. 
• Using thermal protective gloves place the dry ice in the Styrofoam box and spread it evenly 
around each side of the inner cardboard box. 
• Place the lid on the Styrofoam box and put the Styrofoam box in the outer cardboard shipper. 
• Place the shipping manifest on the top of the closed Styrofoam box. 
Close and tape the box along the center break and on the sides. Secure the shipping label to the top 
of the box. 
NOTE: FedEx’s “Priority Overnight” is required shipping method 
• It is the responsibility of the Primary Investigator to ensure that all staff personnel who will be 
handling, packaging, and/or shipping clinical specimens act in conformance with International 
Air Transport Association (IATA) regulations (IATA Packing Instruction 650) relating to the 
handling and shipping of hazardous goods. (IATA Packing Instruction 650 is located on the 
AMC web site). 
• Use a federally approved shipper for biological substance shipment (Category B). Label the 
shipper with the “Infectious substance” diamond shaped label. On one side, in black marker 
write “Biological Substance, Category B, UN 3373,” your name or name of responsible person, 
date of collection and phone number of the person responsible for the package. Package and 
label the shipment in accordance with the instructions provided for that specific shipper. 
• A Shipper’s Declaration for Dangerous Goods is not required. However, for all dry ice 
shipments, the following information must be shown in sequence on the airway bill in the 
“Nature and Quality of Goods” box: Dry Ice, 9, UN1845, number of boxes being shipped, net 
weight of dry ice per box. 
Please Note: The shipper will be mailed back to the AMC site. The STP-100 SAF-T-PAK 
shipper (VWR Cat # 11217-082) is a complete kit (bubble wrap, absorbent paper, labels, etc.) 
for specimen shipment. To reuse the shipper, you will need new labels, wrap, etc. There is a 
refurbishment kit with extra bubble wrap and absorbent material (Fisher Scientific Cat # 19-
038-301E). 

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Sample receipt and processing are performed Tuesday through Friday, unless other 
arrangements have been made for with the lab. Avoid collecting specimens for shipment on 
Fridays, and on days before an observed UC Davis holiday. All specimens should be shipped 
to: 
Dr. Joseph Anderson 
University of California Davis  
2921 Stockton Blvd., Room 1300 
Sacramento, CA 95817 
Tel: (916) 703-9300 
Email: jsanderson@ucdavis.edu 
 
Billing 
Bill the FedEx # available on the AMC member’s only website. This number is supplied by the 
AMC Operations and Data Management Center. It is only to be used for billing shipment of 
specimens to the lab where the sample is processed and/or stored. 
Record of Blood and Endoscopy Specimens 
This study will track specimens via GlobalTraceSM, a component of the AMC AdvantageEDCSM 
system. The GlobalTraceSM shipment manifest must accompany all specimen shipments. 
Technical Questions 
Study Team 
University of California, Davis 
Comprehensive Cancer Center 
4501 X Street, Suite 3016 
Sacramento, CA 95817 
Tel: 916-703-9118 
Fax: 916-734-1895 
Email: HS-CELLULARTHERAPYRESEARCH@UCDAVIS.EDU 
 
4.  
Bone Marrow Aspirate samples for RNA & DNA analysis 
Processing procedures 
• Two 10.0 ml EDTA / Purple top tubes 
• Collect specimen per site’s institutional SOP 
• Immediately transfer and if possible, completely fill two 10 ml EDTA (purple top 
tubes) with the bone marrow aspirate. 

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• Gently invert tubes 8-10 times. 
 
Each sample tube should be labeled with GlobalTraceSM specimen labels using a Sharpie 
pen to include the following information: 
• 9-digit Participant # 
• Date and time of collection 
• Specimen type: (i.e., BMA “Bone Marrow Aspirate”) 
• Specimen purpose: (i.e., DNA Studies) 
 
All Bone Marrow Aspirate Samples should be shipped by priority overnight express on 
MONDAY through Thursday to the UC Davis correlative study laboratory. 
 
Cool Bone Marrow Aspirate Specimen Process and Shipping 
• Apply a strip of Parafilm, around all vials, where the cap meets the tube. 
• Wrap all tubes individually with bubble wrap. 
• Place all specimens in the clear plastic biohazard bag with an absorbent pad and seal. 
• Place the clear plastic biohazard bag in the white Tyvek biohazard bag and seal. 
• Place the sealed white Tyvek biohazard bag in the inner cardboard box of the shipper. 
• Place the inner cardboard box in the Styrofoam cutout, put the notched flap down first, 
and fold across. Remove your gloves. 
• Place four cool gel packs in the Styrofoam box, one on each side of the inner cardboard 
box.  
• Place the lid on the Styrofoam box and put the Styrofoam box in the outer cardboard 
shipper. 
• Place the shipping manifest on the top of the closed Styrofoam box. 
• Close and tape the box along the center break and on the sides. Secure the shipping 
label to the top of the box. 
 
NOTE: FedEx’s “Priority Overnight” is required shipping method. 
• It is the responsibility of the Primary Investigator to ensure that all staff personnel who 
will be handling, packaging, and/or shipping clinical specimens act in conformance 
with International Air Transport Association (IATA) regulations (IATA Packing 
Instruction 650) relating to the handling and shipping of hazardous goods. (IATA 
Packing Instruction 650 is located on the AMC web site). 
• Use a federally approved shipper for biological substance shipment (Category B). 

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Label the shipper with the "Infectious substance" diamond shaped label. On one side, 
in black marker write “Biological Substance, Category B, UN 3373”, your name or 
name of responsible person, date of collection and phone number of the person 
responsible for the package. Package and label the shipment in accordance with the 
instructions provided for that specific shipper. 
• A Shipper’s Declaration for Dangerous Goods is not required. 
Please Note: The shipper will be mailed back to the AMC site. The STP-100 SAF-T-PAK 
shipper (VWR Cat # 11217-082) is a complete kit (bubble wrap, absorbent paper, labels, 
etc.) for specimen shipment. To reuse the shipper, you will need new labels, wrap, etc. 
There is a refurbishment kit with extra bubble wrap and absorbent material (Fisher 
Scientific Cat # 19-038-301E). 
Sample receipt and processing are performed Tuesday through Friday, unless other 
arrangements have been made for with the lab. Avoid collecting specimens for shipment 
on Fridays, and on days before an observed UC Davis holiday. All specimens should be 
shipped to: 
Dr. Joseph Anderson 
University of California Davis  
2921 Stockton Blvd., Room 1300 
Sacramento, CA 95817 
Tel: (916) 703-9300 
 

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APPENDIX VIII: INTERNATIONAL PROGNOSTIC INDEX (AGE-ADJUSTED) 
Characteristic 
Prognostic Factor 
(1 point for each) 
Ann Arbor Stage 
Stage III or IV 
Serum LDH level 
Elevated 
ECOG performance status 
2, 3 or 4 (patients with PS 3 or 4 are not 
eligible)  
 
 

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APPENDIX IX: CCR5-TROPISM ASSAY 
1. Monogram Biosciences Assays: 
GenoSure ArchiveSM DNA Sequencing – Test Code: R6000 
Trofile® DNA – Test Code: E3600 
2. This sample is to be collected within 8 weeks prior to study enrollment. 
3. Draw 4mL of whole blood in 1 lavender top EDTA tube. 
4. DO NOT CENTRIFUGE. 
5. Freeze immediately at -20°C. Sample needs to be FULLY frozen before shipping. 
6. Call Monogram Biosciences Client Services to schedule specimen pick-up at 800-777-
0171 by 1:00 p.m. to ensure next day delivery. 
7. Upon arrival of courier, please place frozen specimen tube into the secondary container 
offered by the courier. 
8. Ensure there is enough bubble wrap and absorbency sheets inside to contain any spills. 
9. Close container and give back to the courier for shipment to Monogram Biosciences. 
 
 

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APPENDIX X: RISK LIST FOR BEAM 
COMMON 
CTCAE Term 
Grade 
BEAM 
Neutrophil count decreased 
1-2 
Platelet count decreased 
1-2 
Lymphocyte count decreased 
1-2 
Febrile Neutropenia 
3 
Nausea 
1-2 
Vomiting 
1-2 
Mucositis oral, 
1-2 
Esophageal ulcer, esophagitis 
1-2 
Abdominal pain 
1-2 
Diarrhea 
1-2 
Anorexia, dysphagia 
1-2 
Alopecia 
1-2 
Fatigue 
1-2 
Infusion of Unmodified Cells 
Presyncope, dizziness 
1-2 
Syncope 
3 
Hypoxia 
2 
Fever 
1-2 
OCCASIONAL  
BEAM 
Blood Bilirubin increased 
1-2 
Alanine aminotransferase (ALT) increased 
1-2 
Aspartate aminotransferase (AST) increased 
1-2 
Pneumonitis 
1-2 
Hypotension 
1-2 
Hyperuricemia 
1-2 
Maculopapular Rash 
1-2 
Chills 
1-2 
Infusion of Unmodified Cells 

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Sinus Tachycardia 
1-2 
 
 
 

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APPENDIX XI: ATI RISK MITIGATION COUSENLING FORMS: 
 
 
 

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APPENDIX XII: HIV TREATMENT INTERRUPTION PARTICIPANT DISCLOSURE 
FORM: 
 
 
 

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APPENDIX XIII: PREP INFORMATION SHEET FOR PARTICIPANTS 
 
 
 

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AMC-097 (Version 9.0) 07FEB2022 
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SUMMARY OF CHANGES 
A Phase I Study of Stem Cell Gene Therapy for HIV Mediated by Lentivector Transduced, 
Pre-Selected CD34+ Cells 
Version 9.0 
 
NCI Protocol #: AMC-097 
Local Protocol #: AMC-097 
 
NCI Version Date: 07FEB2022 
Protocol Date: 07FEB2022 
 
I. 
Scientific and Substantive Changes 
# 
Section 
Description of Change 
1. 
How long will 
I be in this 
study 
During years 3-15, participants will be contacted by a person from the 
clinic rather than a person from the protocol team to collect information 
about their health. 
2. 
During the 
Study 
The conditioning regimen prior to transplant has been revised to 
indicate that agents proven that have the same effects as rituximab 
(biosimilars) may be used instead of ritixumab for participants with B 
cell lymphoma. Participants will be informed of whether they will 
receive rituximab or a similar agent. In addition, the duration that each 
chemotherapy agent will be given has been removed. Both of these 
changes are because instiutions are permitted to administer 
conditioning regimens in accordance to their local guidelines and for 
consistency with the protocol. In addition, allowing administration of 
agents similar to ritiumab will give participating centers an alternative 
option for treatment if there is a supply shortage of rituximab.  
3. 
Use of HIV-1 
Medications 
During the 
Study 
Risk 
Considerations 
related to 
COVID-19 
Attachment 2 
Additional information on the effects and risks of stopping HIV 
medications have been added. In addition recommendations and 
requirements to help reduce these risks have been added. Participants 
will be informed that HIV levels will increase while immune function 
may decrease and that it may take over 4-6 weeks for these levels to 
return to baseline, increasing the risk of developing symptoms. 
Additional risks include: 
• Symptoms similar to those experienced upon initial HIV 
infection 
• Inflammation / heart problems 
• Resistence to HIV medications, increased risk of spreading HIV 
• Increased risk of infection of another strain of HIV 

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# 
Section 
Description of Change 
• Risks related to COVID-19 have been added. 
Participants will be counseled on the risks of stopping HIV 
medications, and if sexually active, advised to use condoms and to refer 
their partners for HIV testing and post-exposure treatment. Participants 
will now be required to be tested for COVID-19 before stopping HIV 
medications. In addition, participants will restart their HIV medications 
if they development symptoms believed to be related to increased levels 
of HIV, have been exposed to HIV infection, or become pregnant. A 
calendar was added in Attachment 2 that outlines required assessment 
required after stopping and restarting HIV medications. 
4. 
Use of HIV-1 
Medications 
During the 
Study 
Attachment 2 
The duration that HIV medications may be stopped post transplant has 
been updated from 12 weeks to until the onset of criterion for 
resumption to allow for additional assessment on the survival of the 
anti-HIV gene. This is considered a safe duration as participants will be 
closely monitored and HIV medications will be restarted if symptoms 
develop.  
5. 
Use of HIV-1 
Medications 
During the 
Study 
Requirements during the conditioning regimen were revised to indicate 
that HIV medication may be held and that participants will be informed 
by their study doctor whether holding HIV medications is necessary. 
This change is consistent with the protocol requirements. 
6. 
Optional 
Sample 
Collections 
Attachment 2 
The consent has been revised to include information on the SCOPE 
study being conducted by University of Califnornia San Franscico. The 
SCOPE study includes optional GI biopsies at the same timepoints as 
the optional GI biopsies in this study. Participants that join this study 
and the SCOPE study at the same time and consent to optional studies 
may have biopsy results from the SCOPE study used for this study. This 
will minimize the number of biopsies taken for participants planning to 
participate in both studies and that consent to optional GI biopsies. GI 
biopsies were added to the study calendar to clarify timepoints and 
collection. 
7. 
How long will 
I be in this 
study 
Optional Bone 
Marrow 
Biospies 
Optional 
Sample 
Optional bone marrow collections have been added for research to 
study the effects of the vector on participants’ genetics and immune 
system. Participants that consent to optional bone marrow biopsies will 
have about 2 tablespoons (20mL) of bone marrow collected at 3, 
months, 12 months, and at study discontinuation. Risks of participating 
in optional bone marrow collection include discomfort, pain, bleeding, 
scarring or infection at the site of the aspirate/biopsy and chance of 
allergic reaction to the numbing medicine. 

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# 
Section 
Description of Change 
Collections 
What is 
Involved? 
Risks of bone 
marrow 
biopsies 
Samples for 
Future 
Research 
Studies 
Attachment 2 
8. 
How long will 
I be in this 
study 
Optional 
Blood Sample 
Collections 
Optional 
Sample 
Collections 
What is 
Involved? 
Risk of blood 
collections 
Samples for 
Future 
Research 
Studies 
Attachment 2 
Optional blood sample collections have been added to monitor HIV 
levels and the vector. Participants that consent to optional blood 
collections will have about 3 tablespoons (30mL) of blood collected 
every 12 months for years 3-15 on study. Risks of bloow draws include 
pain, a bruise or lump at the point where the blood is taken, redness and 
swelling of the vein and infection, and a rare risk of fainting. 
9. 
Attachment 2 
Required bone marrow biopsies to assess disease for staging were 
added for consistency with the protocol 
 
 
 
 

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II. 
Administrative and Editorial Changes  
# 
Section 
Description of Change 
10. 
Global 
The protocol version was changed to 9.0 and the date to 07FEB2022 
11. Attachment 2 Optional GI biopsy collections were added to attachment 2 for 
consistency with the optional study collections sections of the consent 
form.  
 
 

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AMC-097 MODEL INFORMED CONSENT FORM 
 
Study Title for Study Participants: Stem Cell Gene Therapy for HIV-1 in AIDS Lymphoma 
Participants 
Official Study Title for Internet Search on http://www.ClinicalTrials.gov: A Phase I Study of 
Stem Cell Gene Therapy for HIV Mediated by Lentivector Transduced, Pre-selected CD34+ 
Cells 
A Clinical Trial of the AIDS Malignancy Consortium (AMC) 
INTRODUCTION 
You are being asked if you want to take part in a research study. The main goal of a research study 
is to learn things to help participants in the future. No one can guarantee that a research study will 
help you. 
Participating in research is voluntary. You have the right to know about the procedures, risks, and 
benefits of the research study. To participate in this study, you will need to give your written 
consent by signing this form. Please take your time to make your decision and discuss it with your 
family, friends, and caregivers. 
This study is being led by Mehrdad Abedi, MD from the Department of Hematology/Oncology at 
the University of California Davis, with AIDS Malignancy Consortium (AMC) sites at medical 
centers across the U.S. 
WHAT IS THE USUAL APPROACH TO TREAT MY LYMPHOMA? 
You are being asked to take part in this study because you have human immunodeficiency virus 
(HIV) infection and a type of blood cancer called lymphoma. You have already been treated with 
chemotherapy and your disease is now growing. People who are not in a study are usually treated 
with an autologous peripheral blood stem cell transplant with high dose chemotherapy (for 
example BEAM, described below). An autologous peripheral blood stem cell transplant is when 
your own stem cells are collected from your blood, frozen, and then given back to you after you 
receive high dose chemotherapy. This transplant with high dose chemotherapy is intended to 
prevent your disease from coming back in the future. 
WHAT ARE MY OTHER CHOICES IF I DO NOT TAKE PART IN THIS STUDY? 
If you decide not to take part in this study, you have other choices. For example: 
• You may choose to have the usual approach described above. 
• You may choose to take part in a different study, if one is available. 
• You may choose not to be treated for cancer, but you may want to receive comfort care to 
relieve symptoms. Comfort care, or palliative care, helps reduce pain, tiredness, appetite 
problems, and other issues caused by your disease. It does not treat the disease directly, but 
instead tries to improve how you feel. Comfort care tries to keep you as active and comfortable 
as possible. 
WHY IS THIS STUDY BEING DONE? 
The purpose of this study is to test the safety of combining the autologous stem cell therapy with 
gene therapy to treat your lymphoma. For gene therapy, researchers in a laboratory will add small 

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stretches of DNA called “anti-HIV genes” into your stem cells to make the gene therapy product 
used in this study. The type of anti-HIV genes and therapy in this study might make your immune 
cells more resistant to HIV. 
The goal of the study is to develop an immune system that can actively prevent new immune cells 
from getting infected with HIV, while the older cells die due to HIV. This new immune system 
might be able to fight the spread of HIV even without HIV medications. 
The gene therapy product in combination with your clinically indicated stem cell transplant in this 
study is considered experimental, because it has not been used in human clinical trials in the setting 
of an autologous transplant, and has not been approved by the Food and Drug Administration 
(FDA) for treatment of HIV. A similar gene therapy product has been used in other human clinical 
trials with stem cell therapy. Up to 18 people will take part in this study. 
WHAT ARE THE STUDY GROUPS? 
Study participants will get increasing amounts of stem cells with anti-HIV genes with autologous 
stem cell transplant. 
• The first group of study participants will receive equal amounts of the stem cells with anti-HIV 
genes and stem cells without anti-HIV genes. 
• If gene therapy does not cause serious side effects, it will be given to the second group of study 
participants. The second group will receive more stem cells with anti-HIV genes and fewer 
stem cells without anti-HIV genes. 
• If the second group does not have serious side effects, the third group of participants will 
receive only stem cells with anti-HIV genes. 
• If there are issues with cells engrafting in the third group of participants, a fourth group will 
receive only stem cells with anti-HIV genes, but at a higher amount than the third group 
received. 
If you are among the participants that receive only stem cells with anti-HIV-1 genes, some of the 
stem cells without anti-HIV genes will be stored and will only be used as a safety back up if you 
need them. This means that the backup cells would be used if the stem cells with the anti-HIV 
genes do not allow for recovery of your blood cells quickly enough. 
HOW LONG WILL I BE IN THIS STUDY? 
You will receive a one-time infusion of stem cells with anti-HIV genes. The infusion goes into 
your vein like a blood transfusion over less than an hour. This is called an autologous transplant. 
You will have clinic visits to follow up on your transplant on this clinical trial for two years after 
your stem cell transplant. However, we would like to keep track of your medical condition for at 
least 15 years and maybe for the rest of your life. Additionally, every 12 months for years 3-15 we 
will request optional lab studies to monitor your HIV levels and to see how the vector is working 
in your body. A member of the research team at the clinic  will contact you by phone or mail once 
a year for a short conversation and to ask if any new changes have occurred in your health. 
Keeping in touch with you and checking on your condition every year helps us look at the long-
term effects of the study and the process of transplantation in general. Many transplant centers 
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WHAT EXTRA TESTS AND PROCEDURES WILL I HAVE IF I TAKE PART IN THIS 
STUDY? 
Most of the exams, tests, and procedures you will have are part of the usual approach for your 
cancer. However, there are some extra exams, tests, and/or procedures that you will need to have 
if you take part in this study. 
Before You Begin the Study: 
Your study doctor will review your past medical history to determine if you are eligible to 
participate. If you are eligible, you will have an opportunity to ask questions and review the study 
completely. If you agree to participate, you will sign the consent form. You will also be required 
to have certain tests performed prior to the start of the study to make sure you are eligible for this 
study. Most of these tests are standard of care for autologous transplant. 
You will have: 
• A review of your HIV status, which includes asking questions about behaviors related 
to risk factors for HIV, 
• A physical examination, 
• A chest x-ray, 
• Pregnancy test for females who could become pregnant, 
• An EKG (basic heart test), 
• An echocardiogram (heart ultrasound) that assesses the heart function and heart valves, 
• A pulmonary function test to determine how well your lungs are working and how well 
you are breathing, 
• Blood tests (several tablespoons will be taken from a vein in your arm). 
If you recently had any of these tests, your doctor may decide it is not necessary to repeat 
them. 
You may be required to have: 
• A cardiac stress test that assess heart function when you are exercising for about 20 
minutes, 
• An imaging scan of your body called CT scan, to look at the location and size of your 
cancer and any possible source for infection, 
• A bone scan if clinically indicated, and/or a bone marrow biopsy. 
During the Study: 
If the exams, tests, and procedures show that you can take part in the study, and you choose to take 
part, you will be admitted to the hospital as an inpatient only to give you the experimental stem 
cell therapy. If you experience sudden side effects or your lymphoma worsens during the study, 
an additional blood sample (about two tablespoons) may be drawn to study the causes. 
The three parts of the experimental stem cell therapy include: 
Part 1: Taking Blood for Stem Cells 
To collect your stem cells, you will get standard drugs to move stem cells from your bone 
marrow into your blood. This process is called stem cell mobilization. The treatment required 
for stem cell mobilization is called the mobilization regimen. This is a standard of care 

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procedure for all patients undergoing autologous transplant. Your transplant doctor will discuss 
the procedure with you. 
After the stem cells are in your blood, you will have a procedure called leukopheresis. This is 
where your blood is run through a machine to collect the stem cells. Leukopheresis will take 
about 4 hours for most people. You will have this procedure to collect stem cells each day until 
there are enough stem cells for a safe transplant. 
The stem cells that are collected will be split into two parts: 
• The first part will be used for stem cells with the anti-HIV genes. In a very clean laboratory, 
the anti-HIV genes will be put into your stem cells. Once the anti-HIV genes are in the 
stem cells, they will be frozen and stored until you are ready to receive them. 
• The second part of the stem cells will just be frozen and stored without the anti-HIV genes 
in them. 
Part 2: Conditioning Regimen 
Starting six days before your stem cell transplant, you will get a high dose chemotherapy 
conditioning regimen called BEAM or R-BEAM through a vein in your arm to kill the 
lymphoma cells in your body. BEAM and R-BEAM are a mixture of several chemotherapy 
drugs that stop the growth of cancer cells. These chemotherapy drugs are widely used to treat 
lymphoma and for stem cell transplants. The name of the chemotherapy drugs in BEAM and 
R-BEAM regimens are: 
• BCNU (also called carmustine) 
• Etoposide (also called VP-16) 
• Ara-C (also called cytarabine) 
• Melphalan 
• If you have a type of lymphoma called B cell lymphoma, you may receive rituximab or a 
drug proven to have the same effect. Rituximab or similar drug will be given before the 
transplant, and again after the transplant on day 21 and day 28. The study doctor will let 
you know if you will be given rituximab or a biological similar agent. 
Part 3: Reinfusion of Stem Cells (Transplantation) 
One day after BEAM conditioning regimen is complete, the frozen stem cells that were 
previously collected from you will be thawed and re-infused into your veins through a catheter 
in your arm. 
As mentioned before, depending on the study group you are in, you may receive only anti-HIV 
gene treated stem cells, or a combination of anti-HIV gene treated stem cells and stem cells 
without anti-HIV genes. The stem cells with or without anti-HIV genes are supposed to travel 
to your bone marrow where they will begin making healthy, new blood cells. This step is 
necessary because the high doses of chemotherapy given to you during the conditioning 
regimen will not only destroy lymphoma cells, but also healthy cells in your bone marrow. 
Until the new stem cells begin producing healthy blood cells, you will be at an increased risk 
of excessive bleeding or developing an infection. 
You will stay in the hospital for about 3 to 4 weeks until your blood counts recover. The 
duration of hospital stay can be longer if any complication occurs or if your blood counts do 
not recover on time and you require additional stem cell infusion. During your transplant and 

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for about 6 months after transplant you will receive antibiotics to prevent some specific 
infections that can occur after transplant. Your transplant doctor will prescribe those 
medications for you based on the hospital’s guidelines. 
Use of HIV-1 Medications During the Study 
You are expected to take your anti-HIV medications during this study except for two occasions: 
1. The anti-HIV medications you are currently taking may be stopped during BEAM 
chemotherapy, and may be held immediately after that if you cannot tolerate taking oral 
medicines because of the side effects of BEAM chemotherapy. The anti-HIV medications 
will be resumed as soon as you are able to tolerate taking pills. The study doctor will let 
you know if any of your anti-HIV medications need to be stopped. In addition, some 
antiretroviral therapies may have effects on the bone marrow during its recovery (e.g., 
zidovudine) or may interact with BEAM chemotherapy causing side effects. The study 
doctor will let you know if any of your anti-HIV medications need to be changed. 
2. At a minimum of 6 months after transplant and only when we document that your immune 
system has recovered from the transplant, you will be asked if you would agree to stop all 
anti-HIV medications until you meet criteria for resuming your medications. This is to give 
an opportunity for the immune cells that have anti-HIV genes in them to increase in 
number; the cells that do not have anti-HIV genes will be eliminated. We will check your 
level of HIV-1 in the blood and its impact on your blood cells and your general health 
weekly.Your doctor will restart your anti-HIV medications if you or your doctor decides 
this is necessary, or you feel uncomfortable at any time. The decision to stop the anti-HIV 
medications during that time is voluntary and will not affect your follow up in this study. 
Even if the approach works, your virus is expected to come back (rebound) for several 
weeks before it comes under control. If the regimen works, the viral rebound will be 
temporary, meaning that it should go up and then down, but this is not guaranteed. Different 
participants might have different “patterns” of rebound – quick versus slow, high levels 
versus low levels, short versus prolonged. During this time, the study doctor will monitor 
you very carefully. Your capacity to transmit HIV will increase during this time, 
particularly when the virus rebounds, so we will ask you to protect your sexual partner(s). 
The study doctor will give you information on HIV testing sites and HIV prevention 
programs and will counsel you about risks related to stopping your HIV medications. 
If you choose to pause your HIV medications, you will be asked to get tested for the virus that 
causes COVID-19. If the test is positive, we will ask you to stay on your HIV medicines until you 
recover. During the period when you pause taking your HIV medications, if you experience 
COVID-19 symptoms you will be asked to get tested for the virus that causes COVID-19. If you 
test positive, you will need to resume your HIV medications. 
 
The follow-up study visits will be in an outpatient clinic. Your study doctor or a nurse will see you 
in the clinic to assess your health status. You will be requested to have blood tests on a periodic 
basis after you finish receiving the experimental treatment. These blood tests include: 
• About three and a half tablespoons of blood will be taken from a vein in your arm for the blood 
tests to test the status of your health, and 
• Approximately 4 large tablespoons of blood (60 mL) will be taken from a vein in your arm at 

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several time points for studies of your immune system, genetic studies of the effect of 
introduced gene in your blood cells and HIV-1 studies. 
Optional Intestinal Biopsies 
With your consent, we will collect optional biopsies from your upper intestine to see if combining 
the autologous stem cell therapy with gene therapy works to treat your lymphoma. More 
information about this optional study will be explained to you in the Additional Studies Section of 
this consent. 
Optional Blood Tests: 
With your consent, we will ask you have to have blood tests to monitor the levels of HIV in your 
blood. More information about this optional study will be explained to you in the Additional 
Studies Section of this consent. 
Optional Bone Marrow Biopises 
With your consent, we will collect optional bone marrow biospies to study the effects of the 
introduced gene on your genetics and immune systems. You can find more information about this 
optional study  in the Additional Studies Section of this consent. 
A study calendar that shows how often these exams, tests, and procedures will be done is 
attached. 
WHAT POSSIBLE RISKS CAN I EXPECT FROM TAKING PART IN THIS STUDY? 
You may have side effects while on this study. Everyone taking part in the study will be watched 
carefully for any side effects. However, the study doctor may not know or be able to predict all the 
side effects or risks. Side effects may be mild or very serious. The study doctors may give you 
medicines to help lessen side effects. In some cases, side effects can be serious, long lasting, or 
may never go away. There also is a risk of death. You should talk to the study doctor about any 
side effects that you have while taking part in the study. 
If you choose to take part in this study, there is a risk that: 
• You may lose time at work or home and spend more time in the hospital or doctor’s office than 
usual 
• You may be asked sensitive or private questions which you normally do not discuss. 
• The study approach may not be better, and could possibly be worse, than the usual approach 
for your cancer. 
Here are important points about side effects: 
• The study doctors do not know who will or will not have side effects. 
• Some side effects may go away soon, some may last a long time, or some may never go away. 
• Some side effects may interfere with your ability to have children. 
• Some side effects may be serious and may even result in death. 
Here are important points about how you and the study doctor can make side effects less of a 
problem: 

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• Tell the study doctor if you notice or feel anything different so they can see if you are having 
a side effect. 
• The study doctor may be able to treat some side effects. 
The study doctor may adjust the study drugs to try to reduce side effects. The tables below show 
the most common and the most serious side effects that researchers know about. There might be 
other side effects that researchers do not yet know about. If important new side effects are found, 
the study doctor will discuss these with you. 
The side effects associated with this study can be divided into side effects associated with 
autologous transplant that is the standard of care for the treatment of your lymphoma, and the side 
effect associated with gene therapy that is specific to your participation in this clinical trial. 
Potential Side Effects with Autologous Transplant with BEAM Chemotherapy Include 
(But Not Limited To): 
COMMON, SOME MAY BE SERIOUS 
In 100 people receiving BEAM, more than 20 and up to 100 may have: 
• Low blood counts 
• Nausea/vomiting 
• Mouth sores 
• Sores in esophagus 
• Abdominal pain/diarrhea 
• Difficulty eating 
• Hair loss 
• Fatigue 
 
OCCASIONAL, SOME MAY BE SERIOUS 
In 100 people receiving BEAM, from 4 to 20 may have: 
• Liver problems 
• Lung problems 
• Low blood pressure 
• High levels of uric acid 
• Skin rash 
• Chills 
 

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RARE, AND SERIOUS 
In 100 people receiving BEAM, 3 or fewer may have: 
• Liver failure 
• Severe lung problems 
• Severe allergic reactions 
• Second cancers, including Myelodysplastic Syndromes (MDS) and leukemia 
• Life-threatening infection 
• Disease of the peripheral nervous system 
• Sterility 
 
Potential Side Effects with Gene Therapy Include (But Not Limited To): 
As with any procedure, new adverse effects resulting in injury may be discovered. However, a 
similar gene therapy clinical trial for HIV-1 with a similar product given to five previous 
participants in a different institution was well tolerated. In the laboratory, no side effects have been 
seen. However, the potential side effects are: 
• Failure of the stem cells to “take” and thrive in the bone marrow after infusion (this is known 
as engraftment). Back up stem cells will be saved if you are in the third group, and can be given 
to you if this happens. 
• Gene therapy could theoretically cause leukemia. This has not been seen with this gene therapy 
model in the laboratory in extensive testing and in limited human studies with a similar vector. 
Four instances of leukemia were reported in children who participated in an experimental gene 
therapy study conducted in France, not under the jurisdiction of the U.S. Food and Drug 
Administration (FDA). In that study, children were treated by gene therapy for a serious 
genetic disease.  
• Allergic Reactions: Proteins made from large amounts of non-human (protein) can sometimes 
lead to future allergic reactions. There is a small amount of a modified protein in the modified 
cells for which there is a small chance of allergic reaction, but it is unlikely to be harmful to 
you even if it occurs. If an allergic reaction occurs, it is more likely that the modified cells will 
be destroyed. After you receive the stem cells, your blood will be tested for any reaction that 
could affect the use of these agents in the future, but this reaction would not be directly harmful 
to you. This reaction is estimated to happen in about 4 out of 100 people. 
• Reproductive Risks: You should not get pregnant, breastfeed, or father a baby during stem cell 
transplant and for the first 3 months after the day of stem cell transplant. The stem cells with 
anti-HIV genes used in this study could be very damaging to an unborn baby. 
If you are a woman of childbearing age, you can only be admitted to the study if: 

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a) you are not breast feeding; 
b) you are not pregnant (as determined by a pre-study blood test for pregnancy); 
c) you have been surgically sterilized or are using effective birth control. 
Since the effects of the proposed treatments on a fetus are unknown, any woman who 
becomes pregnant while on this study must immediately let the study doctor know and will 
be removed from the study. Another method of treatment will be suggested. 
If you are a male participant, you must practice effective birth control. You must 
immediately let the study doctor know if your partner becomes pregnant, and advise your 
partner to contact her physician. 
Check with the study doctor about what types of birth control, or pregnancy prevention, to 
use while in this study. 
• Unknown Risks: The experimental treatments may have side effects that no one knows 
about yet. In addition, there may be problems of gene therapy that are unexpected but which 
may occur. The special virus (“retroviral vector”) that is used to modify your cells does not 
have the genes needed to reproduce itself, but it is possible in the future that it could 
combine with other genes in your body, or that it could activate an unknown virus in your 
body, to result in a known or unknown medical problem or disease, including the 
possibility of another cancer. 
For more information about risks and side effects of the gene therapy transplant, ask your study 
doctor. 
Potential Side Effects with Stem Cell Infusion Unrelated to the Gene Therapy 
COMMON, SOME MAY BE SERIOUS 
In 100 people receiving Gene Therapy, more than 20 and up to 100 may have: 
• Low blood pressure 
• Lightheadedness 
• Fainting 
• Fever 
• Decrease in oxygen in the blood 
 
OCCASIONAL, SOME MAY BE SERIOUS 
In 100 people receiving Gene Therapy, from 4 to 20 may have: 
• Rapid heart rate 
• Chills 
• Allergic reactions 
 

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RARE, AND SERIOUS 
In 100 people receiving Gene Therapy, 3 or fewer may have: 
• Bronchospasm (asthma like symptoms) 
• Second cancers, including leukemia 
Additionally, while the cells are infused you may experience a garlic-like taste and a scratchy 
throat sensation. This is caused by the chemical used to preserve the cells and is usually a mild and 
temporary discomfort. 
Risk of Antiviral Therapy Withdrawal: Your doctor will ask if you would agree to stop taking your 
anti-HIV medications on at least two occasions as discussed above. 
Each time you stop your anti-HIV medications, there is a potential risk that HIV-1 infection 
become reactivated and suppress your immune system and make you susceptible to severe and 
potentially life threatening infections. The study doctors, including your HIV-1 doctor, will closely 
watch your immune system and will restart your anti-HIV medications if HIV viral reactivation 
and suppression of immune system reaches certain levels. However, restarting the anti-HIV 
medications may not completely stop the risk of complications. 
Your HIV viral load will increase several days to weeks after you stop your HIV medications. The 
viral load might increase to very high levels. If this happens, your CD4+ T cell counts will likely 
drop. It is important to realize that, in this study, the doctors will monitor you very closely and will 
restart your HIV medications as soon as it is clear that the viral load or T cell count puts you at 
high risk of developing any new clinical problems. Your viral load and immune function might 
take over 4-6 weeks to return back to levels comparable to where they were when you entered the 
study. 
When your HIV medications are stopped, you might have symptoms similar to when you first 
were infected with HIV, such as fever, rash, swollen glands, headache, sore throat, nausea, or 
vomiting. Other serious inflammation related symptoms, like increased buildup of plaque on artery 
walls which could possibly result in future heart attacks could happen. If you develop these 
symptoms, the study team will examine you and may restart your HIV medications if they believe 
the symptoms are due to increased HIV levels.  
Resistance to your HIV medications might occur once viral loads become detectable. However, 
because the study doctor will stop all of your HIV medications the same time, the risk of 
developing resistance to your HIV medications is lower.  
While you are off your HIV medications, you are at increased risk of transmitting HIV to sexual 
partners. You may also be at increased risk of becoming infected with a different HIV strain (called 
“superinfection”). Therefore, you should use male or female condoms at all times after HIV 
medications are stopped. If you feel you may have exposed someone to HIV, you should 
immediately refer them for testing and post-exposure prophylaxis (PEP). You should also seriously 
consider referring your HIV- negative sexual partner(s) for pre-exposure prophylaxis (PrEP) if you 
will be sexually active during the treatment interruption period. If you feel you have been exposed 
to HIV while off your HIV medications you may restart your medication this is recommended by 
your primary care provider.  

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If you are a woman and become pregnant while not taking your HIV medications, the study team 
will ask you to restart your medications immediately because there is a chance that you could 
transmit HIV to your developing baby. 
Considerations related to COVID-19  
There are additional risks of participation in this study due to the SARS-CoV-2 pandemic. SARS-
CoV-2 is the virus that causes COVID-19, which is an illness that affects each person differently. 
Some people have no symptoms at all, and some people have very severe symptoms that require 
hospitalization, oxygen, or support with a breathing machine. COVID-19 is a potentially deadly 
illness.  
The effects of COVID-19 in people with HIV are not fully known. It is currently thought that 
people with HIV on HIV medications are at similar risk of getting severe COVID-19 as people 
without HIV. The risk in people with HIV who are not taking HIV medications is unknown, but 
may very well be higher. This is important during the portion of the study when you will be asked 
to pause your HIV medications. If you choose to pause your HIV medications, you will be asked 
to get tested for the virus that causes COVID-19. If the test is positive, we will ask you to stay on 
your HIV medicines until you recover. Once you pause your HIV medications, you will be offered 
testing periodically or if you develop symptoms. If a test for the virus that causes COVID-19 
returns positive, you may be asked to restart HIV medications.  
 
WHAT POSSIBLE BENEFITS CAN I EXPECT FROM TAKING PART IN THIS STUDY? 
You may not personally benefit from participating in this study. This study may help us learn 
things that may help people in the future. 
CAN I STOP TAKING PART IN THIS STUDY? 
Yes. You can decide to stop at any time. If you decide to stop for any reason, it is important to let 
the study doctor know as soon as possible so you can stop safely. If you stop, you can decide 
whether or not to let the study doctor continue to provide your samples or your medical information 
to the organization running the study. If you decide you no longer want your samples to be used, 
any sample that remains will be destroyed and related health information will no longer be 
collected. Samples or related information that have already been given to or used by researchers 
will not be returned. 
The study doctor will tell you about new information or changes in the study that may affect your 
health or your willingness to continue in the study. 
The study doctor may take you out of the study: 
• If your health changes and the study is no longer in your best interest 
• If new information becomes available 
• If you do not follow the study rules 
• If the study is stopped by the sponsor, IRB or FDA. 
WHAT ARE MY RIGHTS IN THIS STUDY? 
Taking part in this study is your choice. No matter what decision you make, and even if your 

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decision changes, there will be no penalty to you. You will not lose medical care or any legal 
rights. 
For questions about your rights while in this study, call the ________________________ (insert 
name of center) Institutional Review Board at __________________ (insert telephone number). 
(Note to Local Investigator: Contact information for patient representatives or other individuals 
at a local institution who are not on the IRB or research team but take calls regarding clinical 
trial questions can also be listed here.) 
WHAT ARE THE COSTS OF TAKING PART IN THIS STUDY? 
The gene modified anti-HIV stem cells will be supplied at no charge while you take part in this 
study. The cost of getting a regular stem cell transplant as a standard of care for your disease is not 
paid by the study sponsor, so you or your insurance company may have to pay for this usually 
before transplant. 
You and/or your health plan/insurance company will need to pay for all the other costs of taking 
care of your cancer while in this study, including the cost of tests, procedures, or medicines to 
manage any side effects, unless you are told that certain tests are supplied at no charge. Before you 
decide to be in the study, you should check with your health plan or insurance company to find out 
exactly what they will pay for. 
You will not be paid for taking part in the mandatory part of this study. For the optional intestinal 
biopsy (upper endoscopy), a fee of $300 for each procedure completed will be paid to you. 
WHAT HAPPENS IF I AM INJURED OR HURT BECAUSE I TOOK PART IN THIS 
STUDY? 
If you are injured or hurt as a result of taking part in this study and need medical treatment, please 
tell your study doctor. The study sponsors will not offer to pay for medical treatment for injury. 
Your insurance company may not be willing to pay for study-related injury. If you have no 
insurance, you would be responsible for any costs. 
If you feel this injury was a result of medical error, you keep all your legal rights to receive 
payment for this even though you are in a study. 
WHO WILL SEE MY MEDICAL INFORMATION? 
Your privacy is very important to us and the researchers will make every effort to protect it. Your 
information may be given out if required by law. For example, certain states require doctors to 
report to health boards if they find a disease like tuberculosis. However, the researchers will do 
their best to make sure that any information that is released will not identify you. Some of your 
health information, and/or information about your specimen, from this study will be kept in a 
central database for research. Your name or contact information will not be put in the database. 
There are organizations that may inspect your records. These organizations are required to make 
sure your information is kept private, unless required by law to provide information. Some of these 
organizations are: 
• The AIDS Malignancy Consortium (AMC) 
• The Institutional Review Board, IRB, is a group of people who review the research with the 
goal of protecting the people who take part in the study. 

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• The Food and Drug Administration and the National Cancer Institute in the U.S. 
• The drug manufacturers supporting the study (University of California Davis and University 
of Indiana). 
WHERE CAN I GET MORE INFORMATION? 
You may visit the NCI Web site at http://cancer.gov/ for more information about studies or general 
information about cancer. You may also call the NCI Cancer Information Service to get the same 
information at: 1-800-4-CANCER (1-800-422-6237). 
A description of this clinical trial will be available on http://www.ClinicalTrials.gov, as required 
by U.S. Law. This web site will not include information that can identify you. At most, the web 
site will include a summary of the results. You can search this web site at any time. 
WHO CAN ANSWER MY QUESTIONS ABOUT THIS STUDY? 
You can talk to the study doctor about any questions or concerns you have about this study or to 
report side effects or injuries. Contact the study doctor __________________ (insert name of study 
doctor[s]) at __________________ (insert telephone number). 
ADDITIONAL STUDIES SECTION 
This part of the consent form is about optional studies that you can choose to take part in. You will 
not get health benefits from any of these studies. The researchers leading this optional study hope 
the results will help other people with cancer in the future. 
The results will not be added to your medical records, but you or your study doctor will know the 
results if requested. 
You will not be billed for these optional studies. You can still take part in the main study even if 
you say ‘no’ to any or all of these studies. If you sign up for but cannot complete any of the studies 
for any reason, you can still take part in the main study. 
Circle your choice of “yes” or “no” for each of the following studies. 
Optional Sample Collections for Laboratory Studies and Donation of Leftover Tissue 
Samples to the Aids and Cancer Specimen Resource (ACSR) 
Researchers are trying to learn more about cancer, HIV/AIDS, and other health problems. Much 
of this research is done using samples from your tissue, blood, urine, or other fluids. Through these 
studies, researchers hope to find new ways to prevent, detect, treat, or cure health problems. 
Some of these studies may be about genes. Genes carry information about features that are found 
in you and in people who are related to you. Researchers are interested in the way that genes affect 
how your body responds to treatment. 
If you choose to take part in this study, the study doctor for the main study would like to collect 
these specimen types: 

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• Optional intestinal biopsy: To obtain important information about the efficacy of this study, 
we would like to ask you to provide a biopsy sample from your upper intestine. Your doctor 
will discuss this with you 6 months after the transplant, or when your CD4 count is greater 
than 300 cells/ mm3. The endoscopy would be repeated 2 months after the first biopsy. If you 
agree to the intestinal biopsy 6 months and 8 months after the transplant, you will be 
asked to sign a separate consent at that time before those procedures. Our study partner at 
The University of California San Francisco is conducting a cohort study called SCOPE. There 
is also optional intestinal biopsies in this study. If you chose to coenroll in the SCOPE study, 
we will use their intestinal biopsy results for both studies. SCOPE is a separate study and 
requires a separate consent form. Participation in SCOPE is not a requirement of this study. If 
you agree to participate, you will be asked to sign a separate SCOPE consent form. We will 
give you the contact information for the SCOPE team if you are interested in learning more. 
• Optional bone marrow biopsies (for participants that do not have bone marrow 
involvement at baseline): If you agree to participate in the optional portion of the study, we 
will request a research bone marrow biopsy or aspirate performed for research studies at 3 
months, 12 months after your stem cell infusion, and when you discontinue the study. These 
studies would normally be standard of care if there is lymphoma in your bone marrow. These 
samples will be used to monitor the effects of the introduced gene on your genetic and immune 
systems. 
• Optional blood collections: If you choose to take part in this study we will request optional 
lab studies every 12 months for years 3-15 to monitor your HIV levels and to see how the 
vector is working in your body.  
• Donation of left over study specimens to the ACSR for all study participants: If you 
choose to take part in this clinical trial, the researchers would like to collect unused blood and 
biopsy tissue left over after the study is done. The researchers ask your permission to store and 
use your samples and related health information (for example, your response to cancer 
treatment, results of study tests and medicines you are given) for medical research. The 
research that may be done is unknown at this time. Storing samples for future studies is called 
“biobanking.” The Biobank is being run by the AIDS and Cancer Specimen Resource and is 
supported by the National Cancer Institute. 
What is Involved? 
If you agree to take part, here is what will happen next: 
1) Optional intestinal biopsy: An endoscopy will be performed to obtain small GI tissue 
samples. An endoscope (a small flexible tube) will be inserted into your stomach and a small 
tissue sample will be collected from your upper intestine using forceps. We will attempt to 
collect 24 very small tissue samples during each endoscopy session from you. 
2) Optional bone marrow biopsies: About 2 tablespoons (20mL) of your bone marrow will be 
collected. A bone marrow biopsy is performed using a hollow needle to remove a small sample 
of bone marrow. Before the biopsy, a local anesthetic is used to numb the area where the needle 
will be inserted. 
3) Optional blood sample collections: We will ask you to have about 3 tablespoons (30 mL) of 
blood collected. You will be seated in the lab or clinic and blood will be drawn by putting a 
needle into a vein in your arm. 
4) Donation of left over study specimens to the ACSR for all study participants: Your sample 
and some related health information will be stored in the ACSR Biobank, along with samples 

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and information from other people who take part. The samples will be kept until they are used 
up. Information from your medical record may be updated after the study is over. 
Qualified researchers can submit a request to use the materials stored in the ACSR. A science 
committee at the ACSR will review each request. There will also be an ethics review to ensure 
that the request is necessary and proper. Researchers will not be given your name or any other 
information that could directly identify you. 
Neither you nor your study doctor will be notified when research will be conducted or given 
reports or other information about any research that is done using your samples.  
Some of your genetic and health information may be placed in central databases that may be 
public, along with information from many other people. Information that could directly 
identify you will not be included. 
Optional bone marrow biopsies, intestinal biopsy, and blood samples collected may be stored for 
up to 15 years to address research questions related to the treatment or the disease under study.  
What are the Possible Risks? 
Risks of Participating in the Optional Intestinal Biopsy (Upper Endoscopy) 
1) Endoscopy risks: The upper endoscopy is being performed as a part of a research project. The 
risks of endoscopy are remote, however, there can be pain, gagging, bleeding, infection, 
perforation (a hole through the bowel wall can occur) and rarely, death can occur. 
2) Sedation risks: The risks of intravenous sedation (putting someone to sleep) include bleeding, 
bruising, pain, infection from intravenous catheter, low blood pressure, decreased breathing, 
and allergic reaction to the medicine. 
3) Allergic reaction risks: Symptoms of allergic reaction may vary from individual to individual 
but often consists of generalized rash (red skin), itchiness, and a feeling of being excited or 
anxious. 
4) Very rarely swelling of the throat and/or difficulty breathing can accompany these symptoms. 
Occasionally (rare), midazolam (also called versed, a drug used for sedation) may cause a state 
of over excitement. The risks for small bowl biopsy are perforation and bleeding. These events 
have occurred in some patients though not with these physicians. These risks will be explained 
to you again by in detail prior to the endoscopy procedure by the gastroenterologist. 
5) Reproductive risks: You should not become pregnant or father a baby if having an upper 
endoscopy because the drugs used during the procedure can affect an unborn baby. Women 
should not breastfeed a baby if having an upper endoscopy. It is important to understand that 
you need to use birth control. Check with your study doctor about what kind of birth control 
methods to use and how long to use them. Some methods might not be approved for use in this 
study. 
Risks of Participating in the Optional Bone Marrow Biopsies: 
The procedure may cause discomfort, pain, bleeding, scarring or infection at the site of the 
aspirate/biopsy. There is also a small chance that you could have an allergic reaction to the 
numbing medicine. After your skin heals, you may have a small scar where we took the sample. 
Risks of Participating in Optional Blood Sample Collections 
The risks of taking blood include pain, a bruise or lump at the point where the blood is taken, 
redness and swelling of the vein and infection, and a rare risk of fainting. 

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Risks of Donation of Left Over Study Specimens to the ACSR 
1) There is a risk that someone could get access to the personal information in your medical 
records or other information researchers have stored about you. 
2) There is a risk that someone could trace the information in a central database back to you. Even 
without your name or other identifiers, your genetic information is unique to you. The 
researchers believe the chance that someone will identify you is very small, but the risk may 
change in the future as people come up with new ways of tracing information. 
3) In some cases, this information could be used to make it harder for you to get or keep a job or 
insurance. There are laws against the misuse of genetic information, but they may not give full 
protection. There can also be a risk in knowing genetic information. New health information 
about inherited traits that might affect you or your blood relatives could be found during a 
study. The researchers believe the chance these things will happen is very small, but cannot 
promise that they will not occur. 
How Will Information About Me be Kept Private? 
Your privacy is very important to the researchers and they will make every effort to protect it. 
Here are just a few of the steps they will take: 
1) When your sample(s) is sent to the researchers, no information identifying you (such as your 
name) will be sent. Samples will be identified by a unique code only. 
2) The list that links the unique code to your name will be kept separate from your sample and 
health information. The ACSR and AMC staff with access to the list must sign an agreement 
to keep your identity confidential. 
3) Researchers to whom the ACSR and the AMC send your sample and information will not know 
who you are. They must also sign an agreement that they will not try to find out who you are. 
4) Information that identifies you will not be given to anyone, unless required by law. 
5) If research results are published, your name and other personal information will not be used. 
What are the Possible Benefits? 
You will not benefit from taking part. The researchers, using the samples from you and others, 
might make discoveries that could help people in the future. 
Are There any Costs or Payments? 
There are no additional costs to you or your insurance for these optional studies. You will not be 
paid for taking part in the mandatory part of this study. For the optional intestinal biopsy (upper 
endoscopy), a fee of $300 for each procedure completed will be paid to you. If any of the research 
leads to new tests, drugs, or other commercial products, you will not share in any profits. 
What if I Change My Mind? 
If you decide you no longer want your samples to be used, you can call the study doctor, 
_________________, (insert name of study doctor for main trial) at ________________________ 
(insert telephone number of study doctor for main trial) who will let the researchers know. Then, 
any sample that remains in the bank will no longer be used and related health information will no 
longer be collected. Samples or related information that have already been given to or used by 
researchers will not be returned. 

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What if I Have More Questions? 
If you have questions about the use of your samples for research, contact the study doctor, 
________________, (insert name of study doctor for main trial), at _____________________ 
(insert telephone number of study doctor for main trial). 
Please circle your answer to show whether or not you would like to take part in each option: 
 
 

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Samples for Future Research Studies: 
I agree to have an optional intestinal biopsy and/or have biopsy results from the SCOPE 
study used for this study as described above. 
 
YES 
 
 
NO 
 
 
Participant Initials: ____ 
I agree to have to have bone marrow biopsies as described above 
 
YES 
 
 
NO 
 
 
Participant Initials: ____ 
I agree to have blood samples collected as described above. 
 
YES 
 
 
NO 
 
 
Participant Initials: ____ 
My samples and related information may be donated to ACSR Biobank for use in future 
health research. 
 
YES 
 
 
NO 
 
 
Participant Initials: ____ 
I agree to have my samples undergo genetic testing to learn about, prevent, diagnose, or treat 
HIV-related diseases and cancer. 
 
YES 
 
 
NO 
 
 
Participant Initials: ____   
This is the end of the section about optional studies__________________________________ 
 
 

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MY SIGNATURE AGREEING TO TAKE PART IN THE MAIN STUDY 
I have read the consent form or had it read to me and understand the information above. I have 
discussed it with the study doctor and my questions have been answered I understand that I will 
be given a signed and dated copy of this consent form. I agree to take part in the main study and 
any additional studies where I circled ‘yes.’ My signature below indicates that I understand my 
rights and want to take part in this study as a research participant. 
 
Participant’s signature: ________________________________ 
 
Date of signature: _____________________________________ 
 
Signature of person(s) conducting the informed consent discussion: _______________________ 
 
Date of signature: _____________________________________ 
 
 

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ATTACHMENT 1: AMC CERTIFICATE OF CONFIDENTIALITY STATEMENT 
The NIH has given the AMC a Certificate of Confidentiality. The Certificate does not mean that 
the NIH or the U.S. Government recommend that you take part in this study. This Certificate helps 
us keep your health information private. 
Your records for this study will have information that may identify you. This Certificate lets us 
turn down legal demands for your study records. We can use the Certificate to turn down demands 
for records from a U.S. court. The Certificate can be used in any federal, state, or local legal 
matters. We will use the Certificate to turn down any demands for your study records. The cases 
where we cannot use the Certificate are explained below. 
We cannot use the Certificate to turn down a demand from the U.S. Government for study records. 
This applies to audits or reviews of the AMC. This also applies to study records that we have to 
report to the FDA. 
The Certificate does not stop you or your family members from sharing your health information. 
It does not stop you from talking about taking part in this study. You may give written permission 
for an insurer, employer, or other person to get copies of your study records. If you give 
permission, we cannot use the Certificate to say no to a request for your study records. 
 
 

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ATTACHMENT 2: STUDY CALENDAR 
Prior to study enrollment and treatment, the following tests will be performed: 
Required Studies/Testing 
Within 8 weeks 
prior to enrollment 
(stem cell collection) 
Within 1 week prior to 
admission for transplant 
On the day of admission 
for transplant before start 
of chemotherapy 
Medical history and physical 
examination  
X 
X 
X 
Infectious disease tests  
X 
 
 
Chest X ray 
X 
X 
 
Heart strip 
X 
 
 
Imaging for staging of your disease 
X 
 
 
Lung function test 
X 
 
 
Kidney function blood tests 
X 
 
 
Bone marrow biopsy  
X 
 
 
Tests for HIV-1 
X 
X 
 
Heart tests 
X 
 
 
Review of your diagnosis slides 
X 
 
 
Tests of your immune systems  
X 
X 
X 
Future research blood sample 
 
X 
 
Pregnancy test for females of 
childbearing potential 
X 
 
X 
After you receive your stem cell transplant, you will have the following tests and follow up 
visits: 
Study 
Assessments/
Testing Post 
Transplant 
Weeks after-HCT 
Months After-HCT 
Years 
After-
HCT 
1 
2 
3 
4 
6 
2 
3 
4 
6 
8 
10 
12 
14 
16, 
18 
20 
22 
24 
3-15, 
once a 
year 
Blood count 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
 
History and 
physical exam 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
 
Imaging for 
staging of 
your disease 
 
 
 
 
 
 
X 
 
X 
 
 
X 
 
 
 
 
 
X 
 
Required bone 
marrow if you 
have bone 
 
 
 
 
 
 
X 
 
 
 
 
X 
 
 
 
 
 
 
X 

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Study 
Assessments/
Testing Post 
Transplant 
Weeks after-HCT 
Months After-HCT 
Years 
After-
HCT 
1 
2 
3 
4 
6 
2 
3 
4 
6 
8 
10 
12 
14 
16, 
18 
20 
22 
24 
3-15, 
once a 
year 
marrow 
involvement 
(for staging 
your disease) 
Optional bone 
marrow biopsy 
(for research) 
 
 
 
 
 
 
X 
 
 
 
 
X 
 
 
 
 
 
 
X 
Optional GI 
biospy 
 
 
 
 
 
 
 
 
X 
X 
 
 
 
 
 
 
 
 
 
Tests of your 
immune 
system 
 
 
 
X 
 
X 
X 
X 
X 
X 
X 
X 
 
 
X 
 
 
X 
X* 
Tests for HIV-
1 activity 
 
 
 
 
 
 
X 
 
X 
X 
X 
X 
 
X 
 
X 
 
X 
X* 
Genetic testing  
 
 
 
X 
 
X 
 
X 
X 
X 
X 
X 
 
 
X 
 
 
X 
X* 
Telephone contact with you or your health care provider for the follow up of HIV status and development of 
any other cancer 
X 
Most of the tests above involve standard medical care. Standard care is what is normally done to 
prevent, diagnose, or treat a certain condition or illness. Other parts of this study, namely 
administration of stem cells with anti-HIV gene in them is investigational. Furthermore, the follow 
up visits and test for assessment of the side effects and the evaluation of your immune system will 
be a part of the study, and out of the standard of care boundaries, and the follow up blood work 
may involve that are being tested for a certain condition or illness. 
Assessments if you stop taking your HIV medicaitons: 
Assessments 
Within 1 Week 
prior to stopping 
HIV medications 
After 
stopping 
your 
HIV 
medications  
(for about 12 
weeks or more) 
After you resume taking your HIV 
medications 
 
 
Weekly 
When you start 
W4 
W8 
W24 

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your HIV 
medications 
COVID Testing 
X 
 
 
 
 
 
Stop 
taking 
ART 
medication 
 
X 
 
 
 
 
Clinical Assessment 
 
X 
X 
X 
X 
X 
Tests on your immune 
system 
 
X 
X 
X 
X 
X 
HIV studies 
 
X 
X 
X 
X 
X 
Blood samples collected 
for research 
 
 
X 
X 
X 
X 
Couseling 
about 
not 
taking 
your 
HIV 
medications 
X 
X