Trial Outcomes & Findings for Gene Therapy in Treating Patients With Human Immunodeficiency Virus-Related Lymphoma Receiving Stem Cell Transplant (NCT NCT02797470)

NCT ID: NCT02797470

Last Updated: 2025-04-20

Results Overview

Timely engraftment is defined as a persistent an absolute neutrophil count (ANC) of at least 500 cells/mm3 and a platelet count of at least 20,000 cells/mm3 for at least 3 days

• Recruitment status: ACTIVE_NOT_RECRUITING
• Study phase: PHASE1/PHASE2
• Target enrollment: 11 participants
• Primary outcome timeframe: 1 month post-transplant
• Results posted on: 2025-04-20

Participant Flow

Participant milestones

Measure	Treatment (Anti-HIV Gene Transduced CD34+ Cells): 1:0 Ratio Patients receive BEAM regimen administered as standard of care comprising carmustine on day -6, cytarabine BID on days -5 to -2, etoposide BID on days -5 to -2, and melphalan on day -1. Patients undergo infusion of lentivirus vector CCR5 shRNA/TRIM5alpha/TAR decoy-transduced autologous CD34-positive hematopoietic progenitor cells over 1 hour. Autologous Hematopoietic Stem Cell Transplantation: Undergo infusion of lentivirus vector CCR5 shRNA/TRIM5alpha/TAR decoy-transduced autologous CD34-positive hematopoietic progenitor cells Carmustine: 300 mg/m2 on Day -6, as part of BEAM and R-BEAM regimens. Cytarabine: 100 mg/m2 BID on Days -5 through -2, as part of BEAM and R-BEAM regimens. Etoposide: VP-16: 100 mg/m2 BID on Days -5 through -2, as part of BEAM and R-BEAM regimens. Laboratory Biomarker Analysis: Correlative studies Lentivirus Vector CCR5 shRNA/TRIM5alpha/TAR Decoy-transduced Autologous CD34-positive Hematopoietic Progenitor Cells: Undergo infusion of lentivirus vector CCR5 shRNA/TRIM5alpha/TAR decoy-transduced autologous CD34-positive hematopoietic progenitor cells Melphalan: 140 mg/m2 on Day -1, as part of BEAM and R-BEAM regimens. Peripheral Blood Stem Cell Transplantation: Undergo infusion of lentivirus vector CCR5 shRNA/TRIM5alpha/TAR decoy-transduced autologous CD34-positive hematopoietic progenitor cells	Treatment (Anti-HIV Gene Transduced CD34+ Cells): 1:1 Ratio Patients receive BEAM regimen administered as standard of care comprising carmustine on day -6, cytarabine BID on days -5 to -2, etoposide BID on days -5 to -2, and melphalan on day -1. Patients undergo infusion of lentivirus vector CCR5 shRNA/TRIM5alpha/TAR decoy-transduced autologous CD34-positive hematopoietic progenitor cells over 1 hour. Autologous Hematopoietic Stem Cell Transplantation: Undergo infusion of lentivirus vector CCR5 shRNA/TRIM5alpha/TAR decoy-transduced autologous CD34-positive hematopoietic progenitor cells	Treatment (Anti-HIV Gene Transduced CD34+ Cells): 5:1 Ratio Patients receive BEAM regimen administered as standard of care comprising carmustine on day -6, cytarabine BID on days -5 to -2, etoposide BID on days -5 to -2, and melphalan on day -1. Patients undergo infusion of lentivirus vector CCR5 shRNA/TRIM5alpha/TAR decoy-transduced autologous CD34-positive hematopoietic progenitor cells over 1 hour. Autologous Hematopoietic Stem Cell Transplantation: Undergo infusion of lentivirus vector CCR5 shRNA/TRIM5alpha/TAR decoy-transduced autologous CD34-positive hematopoietic progenitor cells
Overall Study STARTED	5	4	2
Overall Study COMPLETED	5	4	2
Overall Study NOT COMPLETED	0	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Patients per arm were displayed.

Baseline characteristics by cohort

Measure	Treatment (Anti-HIV Gene Transduced CD34+ Cells) n=11 Participants Patients receive BEAM regimen administered as standard of care comprising carmustine on day -6, cytarabine BID on days -5 to -2, etoposide BID on days -5 to -2, and melphalan on day -1. Patients undergo infusion of lentivirus vector CCR5 shRNA/TRIM5alpha/TAR decoy-transduced autologous CD34-positive hematopoietic progenitor cells over 1 hour. Autologous Hematopoietic Stem Cell Transplantation: Undergo infusion of lentivirus vector CCR5 shRNA/TRIM5alpha/TAR decoy-transduced autologous CD34-positive hematopoietic progenitor cells Carmustine: 300 mg/m2 on Day -6, as part of BEAM and R-BEAM regimens. Cytarabine: 100 mg/m2 BID on Days -5 through -2, as part of BEAM and R-BEAM regimens. Etoposide: VP-16: 100 mg/m2 BID on Days -5 through -2, as part of BEAM and R-BEAM regimens. Laboratory Biomarker Analysis: Correlative studies Lentivirus Vector CCR5 shRNA/TRIM5alpha/TAR Decoy-transduced Autologous CD34-positive Hematopoietic Progenitor Cells: Undergo infusion of lentivirus vector CCR5 shRNA/TRIM5alpha/TAR decoy-transduced autologous CD34-positive hematopoietic progenitor cells Melphalan: 140 mg/m2 on Day -1, as part of BEAM and R-BEAM regimens. Peripheral Blood Stem Cell Transplantation: Undergo infusion of lentivirus vector CCR5 shRNA/TRIM5alpha/TAR decoy-transduced autologous CD34-positive hematopoietic progenitor cells
Age, Continuous Treatment (Anti-HIV Gene Transduced CD34+ Cells): 1:0 Ratio	61 years STANDARD_DEVIATION 6.4 • n=5 Participants • Patients per arm were displayed.
Age, Continuous Treatment (Anti-HIV Gene Transduced CD34+ Cells): 1:1 Ratio	51 years STANDARD_DEVIATION 4.7 • n=4 Participants • Patients per arm were displayed.
Age, Continuous Treatment (Anti-HIV Gene Transduced CD34+ Cells): 5:1 Ratio	61 years STANDARD_DEVIATION 9.9 • n=2 Participants • Patients per arm were displayed.
Sex: Female, Male Treatment (Anti-HIV Gene Transduced CD34+ Cells): 1:0 Ratio · Female	0 Participants n=5 Participants • Patients per arm were displayed.
Sex: Female, Male Treatment (Anti-HIV Gene Transduced CD34+ Cells): 1:0 Ratio · Male	5 Participants n=5 Participants • Patients per arm were displayed.
Sex: Female, Male Treatment (Anti-HIV Gene Transduced CD34+ Cells): 1:1 Ratio · Female	1 Participants n=4 Participants • Patients per arm were displayed.
Sex: Female, Male Treatment (Anti-HIV Gene Transduced CD34+ Cells): 1:1 Ratio · Male	3 Participants n=4 Participants • Patients per arm were displayed.
Sex: Female, Male Treatment (Anti-HIV Gene Transduced CD34+ Cells): 5:1 Ratio · Female	0 Participants n=2 Participants • Patients per arm were displayed.
Sex: Female, Male Treatment (Anti-HIV Gene Transduced CD34+ Cells): 5:1 Ratio · Male	2 Participants n=2 Participants • Patients per arm were displayed.
Ethnicity (NIH/OMB) Treatment (Anti-HIV Gene Transduced CD34+ Cells): 1:0 Ratio · Hispanic or Latino	1 Participants n=5 Participants • Patients per arm were displayed.
Ethnicity (NIH/OMB) Treatment (Anti-HIV Gene Transduced CD34+ Cells): 1:0 Ratio · Not Hispanic or Latino	4 Participants n=5 Participants • Patients per arm were displayed.
Ethnicity (NIH/OMB) Treatment (Anti-HIV Gene Transduced CD34+ Cells): 1:0 Ratio · Unknown or Not Reported	0 Participants n=5 Participants • Patients per arm were displayed.
Ethnicity (NIH/OMB) Treatment (Anti-HIV Gene Transduced CD34+ Cells): 1:1 Ratio · Hispanic or Latino	1 Participants n=4 Participants • Patients per arm were displayed.
Ethnicity (NIH/OMB) Treatment (Anti-HIV Gene Transduced CD34+ Cells): 1:1 Ratio · Not Hispanic or Latino	3 Participants n=4 Participants • Patients per arm were displayed.
Ethnicity (NIH/OMB) Treatment (Anti-HIV Gene Transduced CD34+ Cells): 1:1 Ratio · Unknown or Not Reported	0 Participants n=4 Participants • Patients per arm were displayed.
Ethnicity (NIH/OMB) Treatment (Anti-HIV Gene Transduced CD34+ Cells): 5:1 Ratio · Hispanic or Latino	0 Participants n=2 Participants • Patients per arm were displayed.
Ethnicity (NIH/OMB) Treatment (Anti-HIV Gene Transduced CD34+ Cells): 5:1 Ratio · Not Hispanic or Latino	2 Participants n=2 Participants • Patients per arm were displayed.
Ethnicity (NIH/OMB) Treatment (Anti-HIV Gene Transduced CD34+ Cells): 5:1 Ratio · Unknown or Not Reported	0 Participants n=2 Participants • Patients per arm were displayed.
Race (NIH/OMB) Treatment (Anti-HIV Gene Transduced CD34+ Cells): 1:0 Ratio · American Indian or Alaska Native	0 Participants n=5 Participants • Patients per arm were displayed.
Race (NIH/OMB) Treatment (Anti-HIV Gene Transduced CD34+ Cells): 1:0 Ratio · Asian	0 Participants n=5 Participants • Patients per arm were displayed.
Race (NIH/OMB) Treatment (Anti-HIV Gene Transduced CD34+ Cells): 1:0 Ratio · Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants • Patients per arm were displayed.
Race (NIH/OMB) Treatment (Anti-HIV Gene Transduced CD34+ Cells): 1:0 Ratio · Black or African American	1 Participants n=5 Participants • Patients per arm were displayed.
Race (NIH/OMB) Treatment (Anti-HIV Gene Transduced CD34+ Cells): 1:0 Ratio · White	3 Participants n=5 Participants • Patients per arm were displayed.
Race (NIH/OMB) Treatment (Anti-HIV Gene Transduced CD34+ Cells): 1:0 Ratio · More than one race	0 Participants n=5 Participants • Patients per arm were displayed.
Race (NIH/OMB) Treatment (Anti-HIV Gene Transduced CD34+ Cells): 1:0 Ratio · Unknown or Not Reported	1 Participants n=5 Participants • Patients per arm were displayed.
Race (NIH/OMB) Treatment (Anti-HIV Gene Transduced CD34+ Cells): 1:1 Ratio · American Indian or Alaska Native	0 Participants n=4 Participants • Patients per arm were displayed.
Race (NIH/OMB) Treatment (Anti-HIV Gene Transduced CD34+ Cells): 1:1 Ratio · Asian	0 Participants n=4 Participants • Patients per arm were displayed.
Race (NIH/OMB) Treatment (Anti-HIV Gene Transduced CD34+ Cells): 1:1 Ratio · Native Hawaiian or Other Pacific Islander	0 Participants n=4 Participants • Patients per arm were displayed.
Race (NIH/OMB) Treatment (Anti-HIV Gene Transduced CD34+ Cells): 1:1 Ratio · Black or African American	0 Participants n=4 Participants • Patients per arm were displayed.
Race (NIH/OMB) Treatment (Anti-HIV Gene Transduced CD34+ Cells): 1:1 Ratio · White	3 Participants n=4 Participants • Patients per arm were displayed.
Race (NIH/OMB) Treatment (Anti-HIV Gene Transduced CD34+ Cells): 1:1 Ratio · More than one race	0 Participants n=4 Participants • Patients per arm were displayed.
Race (NIH/OMB) Treatment (Anti-HIV Gene Transduced CD34+ Cells): 1:1 Ratio · Unknown or Not Reported	1 Participants n=4 Participants • Patients per arm were displayed.
Race (NIH/OMB) Treatment (Anti-HIV Gene Transduced CD34+ Cells): 5:1 Ratio · American Indian or Alaska Native	0 Participants n=2 Participants • Patients per arm were displayed.
Race (NIH/OMB) Treatment (Anti-HIV Gene Transduced CD34+ Cells): 5:1 Ratio · Asian	0 Participants n=2 Participants • Patients per arm were displayed.
Race (NIH/OMB) Treatment (Anti-HIV Gene Transduced CD34+ Cells): 5:1 Ratio · Native Hawaiian or Other Pacific Islander	0 Participants n=2 Participants • Patients per arm were displayed.
Race (NIH/OMB) Treatment (Anti-HIV Gene Transduced CD34+ Cells): 5:1 Ratio · Black or African American	0 Participants n=2 Participants • Patients per arm were displayed.
Race (NIH/OMB) Treatment (Anti-HIV Gene Transduced CD34+ Cells): 5:1 Ratio · White	2 Participants n=2 Participants • Patients per arm were displayed.
Race (NIH/OMB) Treatment (Anti-HIV Gene Transduced CD34+ Cells): 5:1 Ratio · More than one race	0 Participants n=2 Participants • Patients per arm were displayed.
Race (NIH/OMB) Treatment (Anti-HIV Gene Transduced CD34+ Cells): 5:1 Ratio · Unknown or Not Reported	0 Participants n=2 Participants • Patients per arm were displayed.
Bone Marrow Biopsy status Treatment (Anti-HIV Gene Transduced CD34+ Cells): 1:0 Ratio	4 Participants n=5 Participants • Patients per arm were displayed.
Bone Marrow Biopsy status Treatment (Anti-HIV Gene Transduced CD34+ Cells): 1:1 Ratio	4 Participants n=4 Participants • Patients per arm were displayed.
Bone Marrow Biopsy status Treatment (Anti-HIV Gene Transduced CD34+ Cells): 5:1 Ratio	2 Participants n=2 Participants • Patients per arm were displayed.

PRIMARY outcome

Timeframe: 1 month post-transplant

Timely engraftment is defined as a persistent an absolute neutrophil count (ANC) of at least 500 cells/mm3 and a platelet count of at least 20,000 cells/mm3 for at least 3 days

Outcome measures

Measure	Treatment (Anti-HIV Gene Transduced CD34+ Cells): 1:0 Ratio n=5 Participants Patients receive BEAM regimen administered as standard of care comprising carmustine on day -6, cytarabine BID on days -5 to -2, etoposide BID on days -5 to -2, and melphalan on day -1. Patients undergo infusion of lentivirus vector CCR5 shRNA/TRIM5alpha/TAR decoy-transduced autologous CD34-positive hematopoietic progenitor cells over 1 hour. Autologous Hematopoietic Stem Cell Transplantation: Undergo infusion of lentivirus vector CCR5 shRNA/TRIM5alpha/TAR decoy-transduced autologous CD34-positive hematopoietic progenitor cells Carmustine: 300 mg/m2 on Day -6, as part of BEAM and R-BEAM regimens. Cytarabine: 100 mg/m2 BID on Days -5 through -2, as part of BEAM and R-BEAM regimens. Etoposide: VP-16: 100 mg/m2 BID on Days -5 through -2, as part of BEAM and R-BEAM regimens. Laboratory Biomarker Analysis: Correlative studies Lentivirus Vector CCR5 shRNA/TRIM5alpha/TAR Decoy-transduced Autologous CD34-positive Hematopoietic Progenitor Cells: Undergo infusion of lentivirus vector CCR5 shRNA/TRIM5alpha/TAR decoy-transduced autologous CD34-positive hematopoietic progenitor cells Melphalan: 140 mg/m2 on Day -1, as part of BEAM and R-BEAM regimens. Peripheral Blood Stem Cell Transplantation: Undergo infusion of lentivirus vector CCR5 shRNA/TRIM5alpha/TAR decoy-transduced autologous CD34-positive hematopoietic progenitor cells	Treatment (Anti-HIV Gene Transduced CD34+ Cells): 1:1 Ratio n=4 Participants Patients receive BEAM regimen administered as standard of care comprising carmustine on day -6, cytarabine BID on days -5 to -2, etoposide BID on days -5 to -2, and melphalan on day -1. Patients undergo infusion of lentivirus vector CCR5 shRNA/TRIM5alpha/TAR decoy-transduced autologous CD34-positive hematopoietic progenitor cells over 1 hour. Autologous Hematopoietic Stem Cell Transplantation: Undergo infusion of lentivirus vector CCR5 shRNA/TRIM5alpha/TAR decoy-transduced autologous CD34-positive hematopoietic progenitor cells	Treatment (Anti-HIV Gene Transduced CD34+ Cells): 5:1 Ratio n=2 Participants Patients receive BEAM regimen administered as standard of care comprising carmustine on day -6, cytarabine BID on days -5 to -2, etoposide BID on days -5 to -2, and melphalan on day -1. Patients undergo infusion of lentivirus vector CCR5 shRNA/TRIM5alpha/TAR decoy-transduced autologous CD34-positive hematopoietic progenitor cells over 1 hour. Autologous Hematopoietic Stem Cell Transplantation: Undergo infusion of lentivirus vector CCR5 shRNA/TRIM5alpha/TAR decoy-transduced autologous CD34-positive hematopoietic progenitor cells
Percentage of Participants Who Achieve a Timely Engraftment	60 percentage of participants who achieve a Interval 14.7 to 94.7	75 percentage of participants who achieve a Interval 19.4 to 99.4	100 percentage of participants who achieve a Interval 15.8 to 100.0

SECONDARY outcome

Timeframe: 3 months post-transplant

To determine efficacy of the candidate product, defined as establishment of > 5% mononuclear blood cells expressing anti-HIV genes

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Up to 24 months post-transplant

To determine the presence, quantity, and duration of gene modified HIV-1 resistant peripheral blood cells and gut mucosal immune cells

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Up to 24 months post-transplant

Summarized descriptively. Continuous measures will be summarized by mean (standard deviation [SD]) and median (range), with log transformation if necessary for skewed measures, as would be typical for cell counts.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Up to 24 months post-transplant

Single genome sequencing of HIV gp120 and HIV pol will be performed to understand sequence evolution following transplantation and detection of minority resistance variants.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Time from start of study treatment to relapse, progression, or death from any cause

Time-to-event data 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.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Up to 15 years

The length of time from the start of treatment until death

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 24 months

A complete response is the complete disappearance of any disease, as determined by imaging

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Time from the first documentation of CR until first date that relapsed or progressive disease is objectively documented, assessed up to 15 years

Complete response is defined as the absence of any disease on imaging or by exam; progressive disease is defined as new lesions or new evidence of disease

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Up to 15 years

Will be assessed following the Lugano Classification. In the absence of evident disease progression, response will be assessed formally at 3, 6, 12 and 24 months post transplant per study calendar.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Up to 15 years

First measurement of 3 consecutive laboratory values obtained on different days) of ANC > 500 cells/mm3

.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Up to 15 years

First measurement of 3 consecutive laboratory values obtained on different days) of platelets > 20,000 cells/mm3 without platelet transfusions 7 days prior

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 100 days

To study hematologic function at Day 100

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Up to 24 months post-treatment

At six months post-transplant, or later, ART will be voluntarily withheld for a 12 week period only for participants who have a CD4 count of 300 or higher with no detectable viral load. 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.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Up to 15 years

To study safety in terms of the frequency of toxicities, infections, transfusions, and infusion-related reactions

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: At week 4, months 3, 6, 8, 10, 12, 14, 16, 20, and 24 post-transplant.

To study HIV-1 viral load over time

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Up to 15 years

Vector stability analysis will be performed via qPRC sequencing.

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: Up to 24 months post-transplant

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

Outcome measures

Outcome data not reported

Adverse Events

Treatment (Anti-HIV Gene Transduced CD34+ Cells): 1:0 Ratio

Serious events: 1 serious events

Other events: 2 other events

Deaths: 2 deaths

Treatment (Anti-HIV Gene Transduced CD34+ Cells): 1:1 Ratio

Serious events: 1 serious events

Other events: 4 other events

Deaths: 2 deaths

Treatment (Anti-HIV Gene Transduced CD34+ Cells): 5:1 Ratio

Serious events: 0 serious events

Other events: 0 other events

Deaths: 1 deaths

Serious adverse events

Measure	Treatment (Anti-HIV Gene Transduced CD34+ Cells): 1:0 Ratio n=5 participants at risk Patients receive BEAM regimen administered as standard of care comprising carmustine on day -6, cytarabine BID on days -5 to -2, etoposide BID on days -5 to -2, and melphalan on day -1. Patients undergo infusion of lentivirus vector CCR5 shRNA/TRIM5alpha/TAR decoy-transduced autologous CD34-positive hematopoietic progenitor cells over 1 hour. Autologous Hematopoietic Stem Cell Transplantation: Undergo infusion of lentivirus vector CCR5 shRNA/TRIM5alpha/TAR decoy-transduced autologous CD34-positive hematopoietic progenitor cells Carmustine: 300 mg/m2 on Day -6, as part of BEAM and R-BEAM regimens. Cytarabine: 100 mg/m2 BID on Days -5 through -2, as part of BEAM and R-BEAM regimens. Etoposide: VP-16: 100 mg/m2 BID on Days -5 through -2, as part of BEAM and R-BEAM regimens. Laboratory Biomarker Analysis: Correlative studies Lentivirus Vector CCR5 shRNA/TRIM5alpha/TAR Decoy-transduced Autologous CD34-positive Hematopoietic Progenitor Cells: Undergo infusion of lentivirus vector CCR5 shRNA/TRIM5alpha/TAR decoy-transduced autologous CD34-positive hematopoietic progenitor cells Melphalan: 140 mg/m2 on Day -1, as part of BEAM and R-BEAM regimens. Peripheral Blood Stem Cell Transplantation: Undergo infusion of lentivirus vector CCR5 shRNA/TRIM5alpha/TAR decoy-transduced autologous CD34-positive hematopoietic progenitor cells	Treatment (Anti-HIV Gene Transduced CD34+ Cells): 1:1 Ratio n=4 participants at risk Patients receive BEAM regimen administered as standard of care comprising carmustine on day -6, cytarabine BID on days -5 to -2, etoposide BID on days -5 to -2, and melphalan on day -1. Patients undergo infusion of lentivirus vector CCR5 shRNA/TRIM5alpha/TAR decoy-transduced autologous CD34-positive hematopoietic progenitor cells over 1 hour. Autologous Hematopoietic Stem Cell Transplantation: Undergo infusion of lentivirus vector CCR5 shRNA/TRIM5alpha/TAR decoy-transduced autologous CD34-positive hematopoietic progenitor cells	Treatment (Anti-HIV Gene Transduced CD34+ Cells): 5:1 Ratio n=2 participants at risk Patients receive BEAM regimen administered as standard of care comprising carmustine on day -6, cytarabine BID on days -5 to -2, etoposide BID on days -5 to -2, and melphalan on day -1. Patients undergo infusion of lentivirus vector CCR5 shRNA/TRIM5alpha/TAR decoy-transduced autologous CD34-positive hematopoietic progenitor cells over 1 hour. Autologous Hematopoietic Stem Cell Transplantation: Undergo infusion of lentivirus vector CCR5 shRNA/TRIM5alpha/TAR decoy-transduced autologous CD34-positive hematopoietic progenitor cells
Investigations PLATELET COUNT DECREASED	20.0% 1/5 • Number of events 1 • 24 months after transplant	0.00% 0/4 • 24 months after transplant	0.00% 0/2 • 24 months after transplant
Investigations BLOOD BILIRUBIN INCREASED	0.00% 0/5 • 24 months after transplant	25.0% 1/4 • Number of events 1 • 24 months after transplant	0.00% 0/2 • 24 months after transplant

Other adverse events

Measure	Treatment (Anti-HIV Gene Transduced CD34+ Cells): 1:0 Ratio n=5 participants at risk Patients receive BEAM regimen administered as standard of care comprising carmustine on day -6, cytarabine BID on days -5 to -2, etoposide BID on days -5 to -2, and melphalan on day -1. Patients undergo infusion of lentivirus vector CCR5 shRNA/TRIM5alpha/TAR decoy-transduced autologous CD34-positive hematopoietic progenitor cells over 1 hour. Autologous Hematopoietic Stem Cell Transplantation: Undergo infusion of lentivirus vector CCR5 shRNA/TRIM5alpha/TAR decoy-transduced autologous CD34-positive hematopoietic progenitor cells Carmustine: 300 mg/m2 on Day -6, as part of BEAM and R-BEAM regimens. Cytarabine: 100 mg/m2 BID on Days -5 through -2, as part of BEAM and R-BEAM regimens. Etoposide: VP-16: 100 mg/m2 BID on Days -5 through -2, as part of BEAM and R-BEAM regimens. Laboratory Biomarker Analysis: Correlative studies Lentivirus Vector CCR5 shRNA/TRIM5alpha/TAR Decoy-transduced Autologous CD34-positive Hematopoietic Progenitor Cells: Undergo infusion of lentivirus vector CCR5 shRNA/TRIM5alpha/TAR decoy-transduced autologous CD34-positive hematopoietic progenitor cells Melphalan: 140 mg/m2 on Day -1, as part of BEAM and R-BEAM regimens. Peripheral Blood Stem Cell Transplantation: Undergo infusion of lentivirus vector CCR5 shRNA/TRIM5alpha/TAR decoy-transduced autologous CD34-positive hematopoietic progenitor cells	Treatment (Anti-HIV Gene Transduced CD34+ Cells): 1:1 Ratio n=4 participants at risk Patients receive BEAM regimen administered as standard of care comprising carmustine on day -6, cytarabine BID on days -5 to -2, etoposide BID on days -5 to -2, and melphalan on day -1. Patients undergo infusion of lentivirus vector CCR5 shRNA/TRIM5alpha/TAR decoy-transduced autologous CD34-positive hematopoietic progenitor cells over 1 hour. Autologous Hematopoietic Stem Cell Transplantation: Undergo infusion of lentivirus vector CCR5 shRNA/TRIM5alpha/TAR decoy-transduced autologous CD34-positive hematopoietic progenitor cells	Treatment (Anti-HIV Gene Transduced CD34+ Cells): 5:1 Ratio n=2 participants at risk Patients receive BEAM regimen administered as standard of care comprising carmustine on day -6, cytarabine BID on days -5 to -2, etoposide BID on days -5 to -2, and melphalan on day -1. Patients undergo infusion of lentivirus vector CCR5 shRNA/TRIM5alpha/TAR decoy-transduced autologous CD34-positive hematopoietic progenitor cells over 1 hour. Autologous Hematopoietic Stem Cell Transplantation: Undergo infusion of lentivirus vector CCR5 shRNA/TRIM5alpha/TAR decoy-transduced autologous CD34-positive hematopoietic progenitor cells
Investigations BLOOD BILIRUBIN INCREASED	0.00% 0/5 • 24 months after transplant	25.0% 1/4 • Number of events 1 • 24 months after transplant	0.00% 0/2 • 24 months after transplant
Metabolism and nutrition disorders HYPOALBUMINEMIA	0.00% 0/5 • 24 months after transplant	50.0% 2/4 • Number of events 2 • 24 months after transplant	0.00% 0/2 • 24 months after transplant
Metabolism and nutrition disorders HYPOPHOSPHATEMIA	0.00% 0/5 • 24 months after transplant	25.0% 1/4 • Number of events 1 • 24 months after transplant	0.00% 0/2 • 24 months after transplant
Investigations LYMPHOCYTE COUNT DECREASED	0.00% 0/5 • 24 months after transplant	25.0% 1/4 • Number of events 1 • 24 months after transplant	0.00% 0/2 • 24 months after transplant
Investigations NEUTROPHIL COUNT DECREASED	40.0% 2/5 • Number of events 2 • 24 months after transplant	50.0% 2/4 • Number of events 2 • 24 months after transplant	0.00% 0/2 • 24 months after transplant
Investigations PLATELET COUNT DECREASED	20.0% 1/5 • Number of events 1 • 24 months after transplant	50.0% 2/4 • Number of events 2 • 24 months after transplant	0.00% 0/2 • 24 months after transplant
Investigations WHITE BLOOD CELL DECREASED	0.00% 0/5 • 24 months after transplant	25.0% 1/4 • Number of events 2 • 24 months after transplant	0.00% 0/2 • 24 months after transplant

Additional Information

Deukwoo Kwon

Statistical and Data Analysis Center, AIDS Malignancy Consortium

Phone: (713) 500-7964

Email: deukwoo.kwon@mountsinai.org

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place