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ClinicalTrials.gov public records Last synced Oct 1, 2026, 10:38 PM EDT

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Showing 1–24 of 36 matching trials from the live ClinicalTrials.gov search.
Filters: Intervention: Gene-Transduced Autologous CD34+ Stem Cells Clear all
Active, not recruiting Phase 3 Interventional

A Study Evaluating Gene Therapy With BB305 Lentiviral Vector in Sickle Cell Disease

NCT04293185
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
bb1111
Genetic
Lead sponsor
Genetix Biotherapeutics Inc.
Industry
Eligibility
2 Years to 50 Years
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2027
U.S. locations
9
States / cities
Birmingham, Alabama • Washington D.C., District of Columbia • Boston, Massachusetts + 6 more
Source: ClinicalTrials.gov public record
Updated Dec 10, 2024 · Synced Oct 1, 2026, 10:38 PM EDT
Terminated Phase 1 Interventional

Gene Therapy and Chemotherapy in Treating Patients With Advanced Solid Tumors or Non-Hodgkin's Lymphoma

NCT00003567
View directory record Official record
Conditions
Brain and Central Nervous System Tumors, Lymphoma, Unspecified Adult Solid Tumor, Protocol Specific
Interventions
filgrastim, sargramostim, therapeutic autologous lymphocytes, O6-benzylguanine, carmustine, temozolomide, in vitro-treated peripheral blood stem cell transplantation
Biological · Drug · Procedure
Lead sponsor
Case Comprehensive Cancer Center
Other
Eligibility
18 Years to 70 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1999 – 2007
U.S. locations
1
States / cities
Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated Jun 11, 2010 · Synced Oct 1, 2026, 10:38 PM EDT
Active, not recruiting Phase 3 Interventional

A Study to Investigate the Efficacy and Safety of OTL-203 in Subjects With MPS-IH Compared With Standard of Care With Allogeneic HSCT

NCT06149403
View directory record Official record
Conditions
MPS-IH (Hurler Syndrome)
Interventions
Experimental: OTL-203, Active Comparator: Allo-HSCT
Genetic
Lead sponsor
Orchard Therapeutics
Industry
Eligibility
28 Days to 30 Months
Enrollment
41 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2031
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Dec 22, 2025 · Synced Oct 1, 2026, 10:38 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional Results available

Gene Therapy in Treating Patients With Human Immunodeficiency Virus-Related Lymphoma Receiving Stem Cell Transplant

NCT02797470
View directory record Official record
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
Interventions
Autologous Hematopoietic Stem Cell Transplantation, Carmustine, Cytarabine, Etoposide, Laboratory Biomarker Analysis, Lentivirus Vector CCR5 shRNA/TRIM5alpha/TAR Decoy-transduced Autologous CD34-positive Hematopoietic Progenitor Cells, Melphalan, Peripheral Blood Stem Cell Transplantation
Procedure · Drug · Other + 1 more
Lead sponsor
AIDS Malignancy Consortium
Network
Eligibility
18 Years and older
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2036
U.S. locations
4
States / cities
La Jolla, California • Sacramento, California • San Francisco, California + 1 more
Source: ClinicalTrials.gov public record
Updated May 18, 2026 · Synced Oct 1, 2026, 10:38 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCID

NCT02999984
View directory record Official record
Conditions
Severe Combined Immunodeficiency Due to ADA Deficiency
Interventions
Infusion of autologous cryopreserved EFS-ADA LV CD34+ cells (OTL-101), busulfan, PEG-ADA ERT
Genetic · Drug
Lead sponsor
University of California, Los Angeles
Other
Eligibility
30 Days to 17 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Aug 3, 2022 · Synced Oct 1, 2026, 10:38 PM EDT
Terminated Phase 1Phase 2 Interventional

Gene Transfer for Patients With Sickle Cell Disease

NCT02186418
View directory record Official record
Conditions
Anemia, Sickle Cell
Interventions
ARU-1801
Genetic
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
18 Years to 45 Years
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2022
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Apr 12, 2024 · Synced Oct 1, 2026, 10:38 PM EDT
Completed Phase 1 Interventional

Stem Cell Gene Therapy to Treat X-Linked Severe Combined Immunodeficiency (XSCID)

NCT00028236
View directory record Official record
Conditions
Severe Combined Immunodeficiency
Interventions
Gene-Transduced Autologous CD34+ Stem Cells
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
18 Months to 20 Years
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2011
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 2, 2017 · Synced Oct 1, 2026, 10:38 PM EDT
Terminated Phase 1 Interventional

A Trial to Evaluate Safety and Efficacy of RP-L401-0120 in Subjects With Infantile Malignant Osteopetrosis

NCT04525352
View directory record Official record
Conditions
Infantile Malignant Osteopetrosis
Interventions
RP-L401
Biological
Lead sponsor
Rocket Pharmaceuticals Inc.
Industry
Eligibility
1 Month and older
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2021
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Jul 13, 2022 · Synced Oct 1, 2026, 10:38 PM EDT
Completed Phase 3 Interventional Results available

A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-Thalassemia

NCT03207009
View directory record Official record
Conditions
Beta-Thalassemia
Interventions
LentiGlobin BB305 Drug Product
Genetic
Lead sponsor
Genetix Biotherapeutics Inc.
Industry
Eligibility
0 Years to 50 Years
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2022
U.S. locations
3
States / cities
Oakland, California • Chicago, Illinois • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 7, 2024 · Synced Oct 1, 2026, 10:38 PM EDT
Completed Phase 1Phase 2 Interventional

A Clinical Trial to Evaluate the Safety and Efficacy of RP-L201 in Subjects With Leukocyte Adhesion Deficiency-I

NCT03812263
View directory record Official record
Conditions
Leukocyte Adhesion Defect - Type I
Interventions
RP-L201
Biological
Lead sponsor
Rocket Pharmaceuticals Inc.
Industry
Eligibility
3 Months and older
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2023
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Nov 15, 2023 · Synced Oct 1, 2026, 10:38 PM EDT
Completed Phase 1 Interventional

Gene Therapy for Pyruvate Kinase Deficiency (PKD)

NCT04105166
View directory record Official record
Conditions
Pyruvate Kinase Deficiency
Interventions
RP-L301
Biological
Lead sponsor
Rocket Pharmaceuticals Inc.
Industry
Eligibility
8 Years to 50 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2025
U.S. locations
1
States / cities
Stanford, California
Source: ClinicalTrials.gov public record
Updated Sep 5, 2025 · Synced Oct 1, 2026, 10:38 PM EDT
Completed Phase 2 Interventional Results available

Long Term Follow-Up Study of Human Immunodeficiency Virus Type 1 (HIV-1) Positive Patients Who Have Received OZ1 Gene Therapy as Part of a Clinical Trial

NCT01177059
View directory record Official record
Conditions
HIV-1
Interventions
OZ1 transduced cells
Genetic
Lead sponsor
Janssen-Cilag Pty Ltd
Industry
Eligibility
Not listed
Enrollment
68 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2017
U.S. locations
3
States / cities
Los Angeles, California • San Francisco, California • New York, New York
Source: ClinicalTrials.gov public record
Updated Dec 12, 2018 · Synced Oct 1, 2026, 10:38 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Stem Cell Gene Therapy for Cystinosis

NCT03897361
View directory record Official record
Conditions
Lysosomal Storage Diseases, Cystinosis
Interventions
CTNS-RD-04 (including CTNS-RD-04-LB manufactured with LentiBOOST)
Genetic
Lead sponsor
University of California, San Diego
Other
Eligibility
18 Years and older
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2024
U.S. locations
1
States / cities
La Jolla, California
Source: ClinicalTrials.gov public record
Updated Aug 6, 2026 · Synced Oct 1, 2026, 10:38 PM EDT
Recruiting Phase 1Phase 2 Interventional

Autologous Gene Therapy for Artemis-Deficient SCID

NCT03538899
View directory record Official record
Conditions
Severe Combined Immunodeficiency
Interventions
AProArt-CD34, CliniMACS® CD34 Reagent System cell sorter device, Busulfan
Drug · Device
Lead sponsor
University of California, San Francisco
Other
Eligibility
2 Months and older
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2038
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Feb 13, 2026 · Synced Oct 1, 2026, 10:38 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Autologous CD34+ Hematopoietic Stem Cells Transduced ex Vivo With Elongation Factor 1 Alpha Shortened (EFS) Lentiviral Vector Encoding for the Human ADA Gene

NCT01852071
View directory record Official record
Conditions
ADA-SCID
Interventions
Infusion of autologous EFS-ADA LV CD34+ (OTL-101), busulfan, PEG-ADA ERT
Genetic · Drug
Lead sponsor
University of California, Los Angeles
Other
Eligibility
1 Month to 17 Years
Enrollment
46 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2018
U.S. locations
2
States / cities
Los Angeles, California • Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 3, 2022 · Synced Oct 1, 2026, 10:38 PM EDT
Active, not recruiting Phase 1 Interventional

Gene Transfer for Sickle Cell Disease

NCT03282656
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
single infusion of autologous bone marrow derived CD34+ HSC cells transduced with the lentiviral vector containing a short-hairpin RNA targeting BCL11a
Biological
Lead sponsor
David Williams
Other
Eligibility
3 Years to 40 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2026
U.S. locations
2
States / cities
Los Angeles, California • Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jan 9, 2026 · Synced Oct 1, 2026, 10:38 PM EDT
Recruiting Phase 1Phase 2 Interventional

Lentiviral Gene Transfer for Treatment of Children Older Than Two Years of Age With X-Linked Severe Combined Immunodeficiency (XSCID)

NCT01306019
View directory record Official record
Conditions
X-linked Severe Combined Immunodeficiency (XSCID)
Interventions
Ex vivo culture and transduction of the patient's autologous CD34+ HSC with lentivirus vector VSV-G pseudotyped CL20- 4i-EF1alpha-hgammac-OPT vector, Busulfan, Palifermin
Biological · Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
2 Years to 50 Years · Male only
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2032
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 28, 2026 · Synced Oct 1, 2026, 10:38 PM EDT
Completed Phase 1 Interventional

Gene Transfer Therapy for Severe Combined Immunodeficieny Disease (SCID) Due to Adenosine Deaminase (ADA) Deficiency

NCT00018018
View directory record Official record
Conditions
Severe Combined Immunodeficiency Syndrome
Interventions
CD34+ cells transduced with ADA retrovir
Drug
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
1 Month and older
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2014
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Dec 12, 2019 · Synced Oct 1, 2026, 10:38 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Gene Therapy for Adenosine Deaminase Severe Combined Immune Deficiency Using Peripheral Blood and EFS ADA Vector

NCT05432310
View directory record Official record
Conditions
Adenosine Deaminase Severe Combined Immune Deficiency
Interventions
A cryopreserved formulation of autologous mPB CD34+ hematopoietic stem and progenitor cells transduced ex vivo with the EFS-ADA lentiviral vector encoding the human ADA enzyme
Combination Product
Lead sponsor
University of California, Los Angeles
Other
Eligibility
1 Month and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2027
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Aug 20, 2026 · Synced Oct 1, 2026, 10:38 PM EDT
Completed Phase 1 Interventional

Gene Therapy-Treated Stem Cells in Treating Patients Undergoing Stem Cell Transplant for Intermediate-Grade or High-Grade AIDS-Related Lymphoma

NCT00569985
View directory record Official record
Conditions
Lymphoma
Interventions
lentivirus vector rHIV7-shI-TAR-CCR5RZ-transduced hematopoietic progenitor cells, carmustine, cyclophosphamide, etoposide, autologous hematopoietic stem cell transplantation
Biological · Drug · Procedure
Lead sponsor
City of Hope Medical Center
Other
Eligibility
18 Years to 60 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2019
U.S. locations
1
States / cities
Duarte, California
Source: ClinicalTrials.gov public record
Updated Nov 15, 2019 · Synced Oct 1, 2026, 10:38 PM EDT
Terminated Early Phase 1 Interventional

Gene Therapy for Chronic Granulomatous Disease

NCT00394316
View directory record Official record
Conditions
Chronic Granulomatous Disease
Interventions
Phagocyte Oxidase Subunit Transduced CD34 Hematopoietic Stem Cells
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
3 Years to 55 Years · Male only
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2014
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 5, 2018 · Synced Oct 1, 2026, 10:38 PM EDT
Completed Phase 1Phase 2 Interventional

Stem Cell Gene Therapy for Sickle Cell Disease

NCT02247843
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
βAS3-FB vector transduced peripheral blood CD34+ cells
Biological
Lead sponsor
Donald B. Kohn, M.D.
Other
Eligibility
18 Years and older
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2025
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Dec 17, 2025 · Synced Oct 1, 2026, 10:38 PM EDT
Suspended Phase 2 Interventional

Clinical Trial to Evaluate the Efficacy of Gene Therapy for Pyruvate Kinase Deficiency

NCT06422351
View directory record Official record
Conditions
Pyruvate Kinase Deficiency
Interventions
RP-L301
Biological
Lead sponsor
Rocket Pharmaceuticals Inc.
Industry
Eligibility
8 Years to 55 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2029
U.S. locations
1
States / cities
Palo Alto, California
Source: ClinicalTrials.gov public record
Updated Apr 16, 2026 · Synced Oct 1, 2026, 10:38 PM EDT
Completed Phase 3 Interventional Results available

A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-Thalassemia, Who do Not Have a β0/β0 Genotype

NCT02906202
View directory record Official record
Conditions
Beta-Thalassemia
Interventions
LentiGlobin BB305 Drug Product
Genetic
Lead sponsor
Genetix Biotherapeutics Inc.
Industry
Eligibility
0 Years to 50 Years
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2022
U.S. locations
3
States / cities
Oakland, California • Chicago, Illinois • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jun 18, 2023 · Synced Oct 1, 2026, 10:38 PM EDT