A Phase 1/2 interventional study of Autologous Hematopoietic Stem Cell Transplantation and Carmustine in HIV Infection, Mature T-Cell and NK-Cell Non-Hodgkin Lymphoma and Plasmablastic Lymphoma, sponsored by AIDS Malignancy Consortium. Active, not recruiting at 4 sites in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-05-18.
Sponsored by AIDS Malignancy Consortium · Phase 1/2, Interventional, and Treatment
This phase I/II trial studies the side effects and best dose of gene therapy in treating patients with human immunodeficiency virus (HIV)-related lymphoma that did not respond to therapy or came back after an original response receiving stem cell transplant. In gene therapy, small stretches of deoxyribonucleic acid (DNA) called "anti-HIV genes" are introduced into the stem cells in the laboratory to make the gene therapy product used in this study. The type of anti-HIV genes and therapy in this study may make the patient's immune cells more resistant to HIV-1 and prevent new immune cells from getting infected with HIV-1.
PRIMARY OBJECTIVE:
I. Safety, defined as timely engraftment (the collective establishment of a persistent absolute neutrophil count of at least 500 cells/mm\^3 and platelet count of 20,000 cells/mm\^3 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.
SECONDARY OBJECTIVES:
I. 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.
II. To determine the presence, quantity, and duration of gene modified HIV-1 resistant peripheral blood cells and gut mucosal immune cells.
III. To study the integration sites of vector sequences in circulating cells. IV. To study progression-free survival. V. To study overall survival. VI. To study complete response rate and duration. VII. To study partial response rate and duration. VIII. To study time to neutrophil engraftment (first measurement of 3 consecutive laboratory values on different days) of absolute neutrophil count [ANC] >= 500 cells/mm\^3).
IX. To study time to platelet engraftment (first measurement of 3 consecutive measurements laboratory values obtained on different days) of platelets >= 20,000 cells/mm\^3 without platelet transfusions 7 days prior).
X. To study hematologic function at day 100 (ANC > 1500, hemoglobin [Hb] > 10 g/dl without transfusion and platelets > 100,000) XI. To study CD4 recovery at the conclusion of the trial. XII. To study safety in terms of toxicities, infections, transfusions, and infusion-related reactions.
XIII. To study HIV-1 viral load over time. XIV. To study persistence of vector-transduced cells over time.
EXPLORATORY OBJECTIVE:
I. 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 anti-retroviral therapy (ART).
OUTLINE: This is a dose-escalation study of lentivirus vector CCR5 shRNA/TRIM5alpha/TAR decoy-transduced autologous CD34-positive hematopoietic progenitor cells.
Patients receive BEAM or BEAM-R regimen administered as standard of care comprising carmustine on day -6, cytarabine twice daily (BID) on days -5 to -2, etoposide BID on days -5 to -2, and melphalan on day -1. Patients with B-cell lymphoma also receive rituximab on day -6 before chemotherapy and on days 21 and 28 post-transplant as standard of care. Patients undergo intravenous (IV) infusion of lentivirus vector CCR5 shRNA/TRIM5alpha/TAR decoy-transduced autologous CD34-positive hematopoietic progenitor cells over 1 hour.
After completion of study treatment, patients are followed up at days 7, 14, 21, 28, 42, 60, 90, 120, 180, 240, 300, 360, 420, 480, 520, 600, 660, and 720, and then yearly for at least 15 years.
4,734 studies on the registry are indexed under HIV Infections; 244 are open to participants now.
This study's enrollment of 11 is below the median of 80 across 3,489 interventional studies indexed under HIV Infections.
Browse HIV Infections studies →AIDS Malignancy Consortium is the lead sponsor of 61 studies on the registry; 9 are open to participants now.
Of its 10 completed or terminated interventional studies of FDA-regulated products, 9 (90%) have results posted.
Counted across the registry records on this site, refreshed daily.
Inclusion Criteria
Inclusion criteria associated with type and status of lymphoma, one of the following must be applicable:
-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,
- 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.
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).
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).
Biopsy proven plasmablastic lymphomas, or peripheral T cell lymphoma (with the exception of ALK+ type in first or second complete remission) *NOTE: Patients meeting the following criteria are exempt from the 8-month timeline and do not require additional biopsy:
Inclusion criteria associated with -1 status
Participants on zidovudine [AZT, ZDV, Retrovir®; including Combivir® and Trizivir®] and efavirenz [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.
o Participant taking ARTs must satisfy one of the following:
General Inclusion Criteria (timeline: within 8 weeks prior to enrollment in the screening segment, unless otherwise specified)
Age ≥18 years. Because only adult transplant centers are participating as study sites.
Exclusion Criteria
Participants who do not fulfill the criteria as listed 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):
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Relapsed: the illness came back after treatment had worked. Refractory: it did not respond to treatment, or stopped responding while still on it.
A line of therapy is one course of treatment, which may combine several drugs. First-line is the first course given; second-line follows when that stops working or is not tolerated.
“Two prior lines” means two courses that count separately under the study’s rules — not simply two drugs. Ask the study team if you are unsure how yours are counted.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
A 0–100 scale of how well someone can carry out everyday activities. 100 is no complaints and no signs of illness; 70 is able to look after yourself but not to work or keep up normal activity; 50 is needing considerable help and frequent medical care.
Studies often ask for 60 or 70 and above. Your doctor gives the score.
A scale of how much an illness limits everyday activity. For people being assessed to join a study, scores run from 0 (fully active) to 4; lower is more active.
Studies most often ask for 0–1 or 0–2. Your doctor gives the score; the WHO scale uses the same steps. The full scale also has 5, which records death.
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
How quickly the kidneys clear creatinine, a waste product, from the blood. A study may estimate it from a blood test with age, weight and sex (often by the Cockcroft–Gault formula), or measure it using blood and urine collected over a set time. Given in mL/min; higher is better.
Blood tests of how long blood takes to clot. The INR is the prothrombin time (PT) put on a common scale; about 1 is usual for someone not taking a blood thinner.
Criteria often make an exception for people on a blood thinner such as warfarin, whose INR is raised on purpose.
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
The share of blood the heart’s main pumping chamber (the left ventricle) pushes out with each beat, measured by an echocardiogram (heart ultrasound) or a MUGA scan. About 50–70% is normal.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
The study doctor’s estimate of how long someone is likely to live. Criteria use it so that people can stay in the study long enough for it to answer its question.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Cancer that has spread to the leptomeninges, the thin layers covering the brain and spinal cord, and the fluid between them. It is not the same as brain metastases.
Criteria often treat it separately, and many studies leave it out even where treated brain metastases are allowed.
The number of CD4 cells, a type of white blood cell, in a given amount of blood. It is used to follow how well the immune system is doing in HIV.
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Biopsy and archival tissue
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Relapsed and refractory
Relapsed: the illness came back after treatment had worked. Refractory: it did not respond to treatment, or stopped responding while still on it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Line of therapy
A line of therapy is one course of treatment, which may combine several drugs. First-line is the first course given; second-line follows when that stops working or is not tolerated.
“Two prior lines” means two courses that count separately under the study’s rules — not simply two drugs. Ask the study team if you are unsure how yours are counted.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Karnofsky performance status (KPS)
A 0–100 scale of how well someone can carry out everyday activities. 100 is no complaints and no signs of illness; 70 is able to look after yourself but not to work or keep up normal activity; 50 is needing considerable help and frequent medical care.
Studies often ask for 60 or 70 and above. Your doctor gives the score.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ECOG performance status
A scale of how much an illness limits everyday activity. For people being assessed to join a study, scores run from 0 (fully active) to 4; lower is more active.
Studies most often ask for 0–1 or 0–2. Your doctor gives the score; the WHO scale uses the same steps. The full scale also has 5, which records death.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ALT and AST (liver enzymes)
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Bilirubin
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis C tests
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis B tests
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Creatinine clearance (CrCl)
How quickly the kidneys clear creatinine, a waste product, from the blood. A study may estimate it from a blood test with age, weight and sex (often by the Cockcroft–Gault formula), or measure it using blood and urine collected over a set time. Given in mL/min; higher is better.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
INR, PT and aPTT (clotting tests)
Blood tests of how long blood takes to clot. The INR is the prothrombin time (PT) put on a common scale; about 1 is usual for someone not taking a blood thinner.
Criteria often make an exception for people on a blood thinner such as warfarin, whose INR is raised on purpose.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hemoglobin (Hb)
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
LVEF (left ventricular ejection fraction)
The share of blood the heart’s main pumping chamber (the left ventricle) pushes out with each beat, measured by an echocardiogram (heart ultrasound) or a MUGA scan. About 50–70% is normal.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Life expectancy
The study doctor’s estimate of how long someone is likely to live. Criteria use it so that people can stay in the study long enough for it to answer its question.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Leptomeningeal disease
Cancer that has spread to the leptomeninges, the thin layers covering the brain and spinal cord, and the fluid between them. It is not the same as brain metastases.
Criteria often treat it separately, and many studies leave it out even where treated brain metastases are allowed.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
CD4 count
The number of CD4 cells, a type of white blood cell, in a given amount of blood. It is used to follow how well the immune system is doing in HIV.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cancer (malignancy)
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraindication
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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.
Procedure: Autologous Hematopoietic Stem Cell Transplantation · Drug: Carmustine · Drug: Cytarabine · Drug: Etoposide · Other: Laboratory Biomarker Analysis · Biological: Lentivirus Vector CCR5 shRNA/TRIM5alpha/TAR Decoy-transduced Autologous CD34-positive Hematopoietic Progenitor Cells · Drug: Melphalan · Procedure: Peripheral Blood Stem Cell Transplantation
Undergo infusion of lentivirus vector CCR5 shRNA/TRIM5alpha/TAR decoy-transduced autologous CD34-positive hematopoietic progenitor cells
Also known as: Autologous Stem Cell Transplantation
300 mg/m2 on Day -6, as part of BEAM and R-BEAM regimens.
Also known as: BCNU, Becenum, Becenun, BiCNU, Bis(chloroethyl) Nitrosourea, Bis-Chloronitrosourea, Carmubris, Carmustin, Carmustinum, FDA 0345, Gliadel, N,N'-Bis(2-chloroethyl)-N-nitrosourea, Nitrourean, Nitrumon, SK 27702, SRI 1720, WR-139021
100 mg/m2 BID on Days -5 through -2, as part of BEAM and R-BEAM regimens.
Also known as: .beta.-Cytosine arabinoside, 1-.beta.-D-Arabinofuranosyl-4-amino-2(1H)pyrimidinone, 1-.beta.-D-Arabinofuranosylcytosine, 1-Beta-D-arabinofuranosyl-4-amino-2(1H)pyrimidinone, 1-Beta-D-arabinofuranosylcytosine, 1.beta.-D-Arabinofuranosylcytosine, 2(1H)-Pyrimidinone, 4-Amino-1-beta-D-arabinofuranosyl-, 2(1H)-Pyrimidinone, 4-amino-1.beta.-D-arabinofuranosyl-, Alexan, Ara-C, ARA-cell, Arabine, Arabinofuranosylcytosine, Arabinosylcytosine, Aracytidine, Aracytin, Aracytine, Beta-Cytosine Arabinoside, CHX-3311, Cytarabinum, Cytarbel, Cytosar, Cytosar-U, Cytosine Arabinoside, Cytosine-.beta.-arabinoside, Cytosine-beta-arabinoside, Erpalfa, Starasid, Tarabine PFS, U 19920, U-19920, Udicil, WR-28453
VP-16: 100 mg/m2 BID on Days -5 through -2, as part of BEAM and R-BEAM regimens.
Also known as: Demethyl Epipodophyllotoxin Ethylidine Glucoside, EPEG, Lastet, Toposar, Vepesid, VP 16-213, VP-16, VP-16-213
Correlative studies
Undergo infusion of lentivirus vector CCR5 shRNA/TRIM5alpha/TAR decoy-transduced autologous CD34-positive hematopoietic progenitor cells
Also known as: Lentivirus Vector CCR5 shRNA/TRIM5alpha/TAR Decoy-transduced Autologous CD34-positive HPCs
140 mg/m2 on Day -1, as part of BEAM and R-BEAM regimens.
Also known as: Alanine Nitrogen Mustard, CB-3025, L-PAM, L-Phenylalanine Mustard, L-Sarcolysin, L-Sarcolysin Phenylalanine mustard, L-Sarcolysine, Melphalanum, Phenylalanine Mustard, Phenylalanine Nitrogen Mustard, Sarcoclorin, Sarkolysin, WR-19813
Undergo infusion of lentivirus vector CCR5 shRNA/TRIM5alpha/TAR decoy-transduced autologous CD34-positive hematopoietic progenitor cells
Also known as: PBPC transplantation, Peripheral Blood Progenitor Cell Transplantation, Peripheral Stem Cell Support, Peripheral Stem Cell Transplantation
Percentage of Participants Who Achieve a Timely Engraftment
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
Time frame: 1 month post-transplant
Proportion of Study Participants Who Achieve Greater Than 5% Mononuclear Blood Cells Expressing Anti-HIV Genes in Peripheral Blood
To determine efficacy of the candidate product, defined as establishment of \> 5% mononuclear blood cells expressing anti-HIV genes
Time frame: 3 months post-transplant
Proportion of Study Participants With Gene Modified HIV-1 Resistant Peripheral Blood Cells and Gut Mucosal Immune Cells
To determine the presence, quantity, and duration of gene modified HIV-1 resistant peripheral blood cells and gut mucosal immune cells
Time frame: Up to 24 months post-transplant
Quantity of Gene Modified HIV-1 Resistant Peripheral Blood Cells and Gut Mucosal Immune Cells
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.
Time frame: Up to 24 months post-transplant
Integration Sites of Vector Sequences in Circulating Cells
Single genome sequencing of HIV gp120 and HIV pol will be performed to understand sequence evolution following transplantation and detection of minority resistance variants.
Time frame: Up to 24 months post-transplant
Progression-free Survival
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.
Time frame: Time from start of study treatment to relapse, progression, or death from any cause
Overall Survival
The length of time from the start of treatment until death
Time frame: Up to 15 years
Number of Participants With a Complete Response
A complete response is the complete disappearance of any disease, as determined by imaging
Time frame: 24 months
Number of Days From the First Documentation of a Complete Response Until the First Day of Relapse
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
Time frame: Time from the first documentation of CR until first date that relapsed or progressive disease is objectively documented, assessed up to 15 years
Partial Response Rate and Duration
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.
Time frame: Up to 15 years
Time to Neutrophil Engraftment
First measurement of 3 consecutive laboratory values obtained on different days) of ANC \> 500 cells/mm3 .
Time frame: Up to 15 years
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
Time frame: Up to 15 years
Number of Participants With an Absolute Neutrophil Count of at Least 1500 Cells/mm3, Hemoglobin of at Least 10 g/dL, and Platelets Greater Than 100,000.
To study hematologic function at Day 100
Time frame: 100 days
CD4 Count Recovery
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.
Time frame: Up to 24 months post-treatment
Number of Participants With Adverse Events as Assessed by the CTCAE
To study safety in terms of the frequency of toxicities, infections, transfusions, and infusion-related reactions
Time frame: Up to 15 years
HIV-1 Viral Load
To study HIV-1 viral load over time
Time frame: At week 4, months 3, 6, 8, 10, 12, 14, 16, 20, and 24 post-transplant.
Persistence of Vector-transduced Cells Over Time
Vector stability analysis will be performed via qPRC sequencing.
Time frame: Up to 15 years
Expansion of HIV-1 Resistant Immune Cells
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
Time frame: Up to 24 months post-transplant
| Milestone | Treatment (Anti-HIV Gene Transduced CD34+ Cells): 1:0 Ratio | Treatment (Anti-HIV Gene Transduced CD34+ Cells): 1:1 Ratio | Treatment (Anti-HIV Gene Transduced CD34+ Cells): 5:1 Ratio |
|---|---|---|---|
| Started | 5 | 4 | 2 |
| Completed | 5 | 4 | 2 |
| Not completed | 0 | 0 | 0 |
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
| percentage of participants who achieve a | Treatment (Anti-HIV Gene Transduced CD34+ Cells): 1:0 Ratio | Treatment (Anti-HIV Gene Transduced CD34+ Cells): 1:1 Ratio | Treatment (Anti-HIV Gene Transduced CD34+ Cells): 5:1 Ratio |
|---|---|---|---|
| Percentage of Participants Who Achieve a Timely Engraftment | 60 (14.7 to 94.7) | 75 (19.4 to 99.4) | 100 (15.8 to 100) |
To determine efficacy of the candidate product, defined as establishment of \> 5% mononuclear blood cells expressing anti-HIV genes
Results for this outcome have not been posted.
To determine the presence, quantity, and duration of gene modified HIV-1 resistant peripheral blood cells and gut mucosal immune cells
Results for this outcome have not been posted.
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.
Results for this outcome have not been posted.
Single genome sequencing of HIV gp120 and HIV pol will be performed to understand sequence evolution following transplantation and detection of minority resistance variants.
Results for this outcome have not been posted.
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.
Results for this outcome have not been posted.
The length of time from the start of treatment until death
Results for this outcome have not been posted.
A complete response is the complete disappearance of any disease, as determined by imaging
Results for this outcome have not been posted.
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
Results for this outcome have not been posted.
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.
Results for this outcome have not been posted.
First measurement of 3 consecutive laboratory values obtained on different days) of ANC \> 500 cells/mm3 .
Results for this outcome have not been posted.
First measurement of 3 consecutive laboratory values obtained on different days) of platelets \> 20,000 cells/mm3 without platelet transfusions 7 days prior
Results for this outcome have not been posted.
To study hematologic function at Day 100
Results for this outcome have not been posted.
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.
Results for this outcome have not been posted.
To study safety in terms of the frequency of toxicities, infections, transfusions, and infusion-related reactions
Results for this outcome have not been posted.
To study HIV-1 viral load over time
Results for this outcome have not been posted.
Vector stability analysis will be performed via qPRC sequencing.
Results for this outcome have not been posted.
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
Results for this outcome have not been posted.
Collected over 24 months after transplant. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Treatment (Anti-HIV Gene Transduced CD34+ Cells): 1:0 Ratio | 2/5 (40%) | 1/5 (20%) | 2/5 (40%) |
| Treatment (Anti-HIV Gene Transduced CD34+ Cells): 1:1 Ratio | 2/4 (50%) | 1/4 (25%) | 4/4 (100%) |
| Treatment (Anti-HIV Gene Transduced CD34+ Cells): 5:1 Ratio | 1/2 (50%) | 0/2 (0%) | 0/2 (0%) |
| Event | Treatment (Anti-HIV Gene Transduced CD34+ Cells): 1:0 Ratio | Treatment (Anti-HIV Gene Transduced CD34+ Cells): 1:1 Ratio | Treatment (Anti-HIV Gene Transduced CD34+ Cells): 5:1 Ratio |
|---|---|---|---|
| BLOOD BILIRUBIN INCREASEDInvestigations | 0/5 | 1/4 | 0/2 |
| PLATELET COUNT DECREASEDInvestigations | 1/5 | 0/4 | 0/2 |
| Event | Treatment (Anti-HIV Gene Transduced CD34+ Cells): 1:0 Ratio | Treatment (Anti-HIV Gene Transduced CD34+ Cells): 1:1 Ratio | Treatment (Anti-HIV Gene Transduced CD34+ Cells): 5:1 Ratio |
|---|---|---|---|
| HYPOALBUMINEMIAMetabolism and nutrition disorders | 0/5 | 2/4 | 0/2 |
| NEUTROPHIL COUNT DECREASEDInvestigations | 2/5 | 2/4 | 0/2 |
| PLATELET COUNT DECREASEDInvestigations | 1/5 | 2/4 | 0/2 |
| BLOOD BILIRUBIN INCREASEDInvestigations | 0/5 | 1/4 | 0/2 |
| HYPOPHOSPHATEMIAMetabolism and nutrition disorders | 0/5 | 1/4 | 0/2 |
| LYMPHOCYTE COUNT DECREASEDInvestigations | 0/5 | 1/4 | 0/2 |
| WHITE BLOOD CELL DECREASEDInvestigations | 0/5 | 1/4 | 0/2 |
Participants received Stem Cell Gene Therapy
| Age, Continuous(years) | Treatment (Anti-HIV Gene Transduced CD34+ Cells) |
|---|---|
| Treatment (Anti-HIV Gene Transduced CD34+ Cells): 1:0 Ratio | 61 ± 6.4 |
| Treatment (Anti-HIV Gene Transduced CD34+ Cells): 1:1 Ratio | 51 ± 4.7 |
| Treatment (Anti-HIV Gene Transduced CD34+ Cells): 5:1 Ratio | 61 ± 9.9 |
| Sex: Female, Male(Participants) | Treatment (Anti-HIV Gene Transduced CD34+ Cells) |
|---|---|
| Treatment (Anti-HIV Gene Transduced CD34+ Cells): 1:0 Ratio — Female | 0 |
| Treatment (Anti-HIV Gene Transduced CD34+ Cells): 1:0 Ratio — Male | 5 |
| Treatment (Anti-HIV Gene Transduced CD34+ Cells): 1:1 Ratio — Female | 1 |
| Treatment (Anti-HIV Gene Transduced CD34+ Cells): 1:1 Ratio — Male | 3 |
| Treatment (Anti-HIV Gene Transduced CD34+ Cells): 5:1 Ratio — Female | 0 |
| Treatment (Anti-HIV Gene Transduced CD34+ Cells): 5:1 Ratio — Male | 2 |
| Ethnicity (NIH/OMB)(Participants) | Treatment (Anti-HIV Gene Transduced CD34+ Cells) |
|---|---|
| Treatment (Anti-HIV Gene Transduced CD34+ Cells): 1:0 Ratio — Hispanic or Latino | 1 |
| Treatment (Anti-HIV Gene Transduced CD34+ Cells): 1:0 Ratio — Not Hispanic or Latino | 4 |
| Treatment (Anti-HIV Gene Transduced CD34+ Cells): 1:0 Ratio — Unknown or Not Reported | 0 |
| Treatment (Anti-HIV Gene Transduced CD34+ Cells): 1:1 Ratio — Hispanic or Latino | 1 |
| Treatment (Anti-HIV Gene Transduced CD34+ Cells): 1:1 Ratio — Not Hispanic or Latino | 3 |
| Treatment (Anti-HIV Gene Transduced CD34+ Cells): 1:1 Ratio — Unknown or Not Reported | 0 |
| Treatment (Anti-HIV Gene Transduced CD34+ Cells): 5:1 Ratio — Hispanic or Latino | 0 |
| Treatment (Anti-HIV Gene Transduced CD34+ Cells): 5:1 Ratio — Not Hispanic or Latino | 2 |
| Treatment (Anti-HIV Gene Transduced CD34+ Cells): 5:1 Ratio — Unknown or Not Reported | 0 |
| Race (NIH/OMB)(Participants) | Treatment (Anti-HIV Gene Transduced CD34+ Cells) |
|---|---|
| Treatment (Anti-HIV Gene Transduced CD34+ Cells): 1:0 Ratio — American Indian or Alaska Native | 0 |
| Treatment (Anti-HIV Gene Transduced CD34+ Cells): 1:0 Ratio — Asian | 0 |
| Treatment (Anti-HIV Gene Transduced CD34+ Cells): 1:0 Ratio — Native Hawaiian or Other Pacific Islander | 0 |
| Treatment (Anti-HIV Gene Transduced CD34+ Cells): 1:0 Ratio — Black or African American | 1 |
| Treatment (Anti-HIV Gene Transduced CD34+ Cells): 1:0 Ratio — White | 3 |
| Treatment (Anti-HIV Gene Transduced CD34+ Cells): 1:0 Ratio — More than one race | 0 |
| Treatment (Anti-HIV Gene Transduced CD34+ Cells): 1:0 Ratio — Unknown or Not Reported | 1 |
| Treatment (Anti-HIV Gene Transduced CD34+ Cells): 1:1 Ratio — American Indian or Alaska Native | 0 |
| Treatment (Anti-HIV Gene Transduced CD34+ Cells): 1:1 Ratio — Asian | 0 |
| Treatment (Anti-HIV Gene Transduced CD34+ Cells): 1:1 Ratio — Native Hawaiian or Other Pacific Islander | 0 |
| Treatment (Anti-HIV Gene Transduced CD34+ Cells): 1:1 Ratio — Black or African American | 0 |
| Treatment (Anti-HIV Gene Transduced CD34+ Cells): 1:1 Ratio — White | 3 |
| Treatment (Anti-HIV Gene Transduced CD34+ Cells): 1:1 Ratio — More than one race | 0 |
| Treatment (Anti-HIV Gene Transduced CD34+ Cells): 1:1 Ratio — Unknown or Not Reported | 1 |
| Treatment (Anti-HIV Gene Transduced CD34+ Cells): 5:1 Ratio — American Indian or Alaska Native | 0 |
| Treatment (Anti-HIV Gene Transduced CD34+ Cells): 5:1 Ratio — Asian | 0 |
| Treatment (Anti-HIV Gene Transduced CD34+ Cells): 5:1 Ratio — Native Hawaiian or Other Pacific Islander | 0 |
| Treatment (Anti-HIV Gene Transduced CD34+ Cells): 5:1 Ratio — Black or African American | 0 |
| Treatment (Anti-HIV Gene Transduced CD34+ Cells): 5:1 Ratio — White | 2 |
| Treatment (Anti-HIV Gene Transduced CD34+ Cells): 5:1 Ratio — More than one race | 0 |
| Treatment (Anti-HIV Gene Transduced CD34+ Cells): 5:1 Ratio — Unknown or Not Reported | 0 |
| Bone Marrow Biopsy status(Participants) | Treatment (Anti-HIV Gene Transduced CD34+ Cells) |
|---|---|
| Treatment (Anti-HIV Gene Transduced CD34+ Cells): 1:0 Ratio | 4 |
| Treatment (Anti-HIV Gene Transduced CD34+ Cells): 1:1 Ratio | 4 |
| Treatment (Anti-HIV Gene Transduced CD34+ Cells): 5:1 Ratio | 2 |
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: Undecided
Ran from Jun 2016 to primary completion in May 2021, 58 months.
Results were first posted in Jul 2023, 2 years 2 months after primary completion.
Of 35 non-industry phase 1/2 interventional studies under HIV Infections, half reached primary completion within 55 months of starting (the median), counting those still running; a quarter within 29 and three quarters within 73. This study's 58 months is close to that.
Counting every sponsor, half of 42 studies reached it within 47 months; a quarter within 22 and three quarters within 73.
How these figures are worked out, and which studies are left out of them.
This study is active, not recruiting, as verified in May 2026. You cannot join it, but the record below documents what was studied.
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Studies of the drugs tested here: Carmustine · Cytarabine · Etoposide · Melphalan
AIDS Malignancy Consortium