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Showing 1–24 of 57 matching trials from the live ClinicalTrials.gov search.
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Filters: Intervention: Transduced CD34+ Cells Clear all
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 12, 2026 · Synced Sep 23, 2026, 2:52 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 21, 2025 · Synced Sep 23, 2026, 2:52 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 14, 2019 · Synced Sep 23, 2026, 2:52 PM EDT
Enrolling by invitation Phase 1Phase 2 Interventional

Part B- G1X-CGD (Lentiviral Vector Transduced CD34+ Cells) in Patients With X-Linked Chronic Granulomatous Disease

NCT07113743
View directory record Official record
Conditions
Chronic Granulomatous Disease (CGD)
Interventions
Busulfan, Tocilizumab, Eltrombopag, Sirolimus, pCCLChimGp91lentiviral vector containing the human gp91 phox (CYBB) gene
Drug · Other
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
3 Years to 60 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2029
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 7, 2026 · Synced Sep 23, 2026, 2:52 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 14, 2023 · Synced Sep 23, 2026, 2:52 PM EDT
Completed Phase 1 Interventional

Gene Therapy for the Treatment of Fanconi's Anemia Type C

NCT00001399
View directory record Official record
Conditions
Fanconi's Anemia, Pancytopenia
Interventions
Transduced CD34+ Cells
Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
5 Years and older
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1993 – 2009
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 1, 2017 · Synced Sep 23, 2026, 2:52 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 5, 2026 · Synced Sep 23, 2026, 2:52 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 11, 2024 · Synced Sep 23, 2026, 2:52 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 10, 2010 · Synced Sep 23, 2026, 2:52 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 8, 2026 · Synced Sep 23, 2026, 2:52 PM EDT
Terminated Phase 1 Interventional

CSL200 Gene Therapy in Adults With Severe Sickle Cell Disease

NCT04091737
View directory record Official record
Conditions
Anemia, Sickle Cell
Interventions
Autologous enriched CD34+ cell fraction that contains CD34+ cells transduced with lentiviral vector encoding human γ-globinG16D and short-hairpin RNA734
Biological
Lead sponsor
CSL Behring
Industry
Eligibility
18 Years to 45 Years
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
1
States / cities
Duarte, California
Source: ClinicalTrials.gov public record
Updated Jun 17, 2021 · Synced Sep 23, 2026, 2:52 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 11, 2018 · Synced Sep 23, 2026, 2:52 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 4, 2025 · Synced Sep 23, 2026, 2:52 PM EDT
Temporarily Not Available No phase listed Expanded access

Expanded Access Protocol for Gene Therapy Utilizing shmiR Lentivirus Vector to Induce Fetal Hemoglobin in Sickle Cell Disease

NCT07640815
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Autologous CD34+ HSC cells transduced with the lentiviral vector containing a shRNA targeting BCL11a
Biological
Lead sponsor
David Williams
Other
Eligibility
13 Years to 55 Years
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jun 10, 2026 · Synced Sep 23, 2026, 2:52 PM EDT
Withdrawn Phase 1 Interventional

Gene-Modified HIV-Protected Stem Cell Transplant in Treating Patients With HIV-Associated Lymphoma

NCT02378922
View directory record Official record
Conditions
AIDS-Related Hodgkin Lymphoma, AIDS-Related Non-Hodgkin Lymphoma, HIV Infection, Recurrent Hodgkin Lymphoma, Recurrent Non-Hodgkin Lymphoma
Interventions
Gene-Modified HIV-Protected Hematopoietic Stem Cells, Laboratory Biomarker Analysis, Transplant Conditioning
Biological · Other
Lead sponsor
Fred Hutchinson Cancer Center
Other
Eligibility
18 Years to 66 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
1
States / cities
Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Dec 26, 2018 · Synced Sep 23, 2026, 2:52 PM EDT
Terminated Phase 1 Interventional

Treatment of Newly Diagnosed Brain Tumors With Chemotherapy and Radiation Using Cells Modified for Chemoprotection and an Experimental Drug to Decrease the Tumor Cell Resistance to Chemotherapy

NCT00272870
View directory record Official record
Conditions
Glioblastoma Multiforme (WHO Grade IV), Anaplastic Astrocytoma (WHO Grade III)
Interventions
06Benzylguanine, MGMT P140K, Meltenyi CliniMacs
Drug · Genetic · Device
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
5 Years to 55 Years
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2012
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Aug 28, 2012 · Synced Sep 23, 2026, 2:52 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 15, 2026 · Synced Sep 23, 2026, 2:52 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 17, 2023 · Synced Sep 23, 2026, 2:52 PM EDT
Recruiting Phase 1Phase 2 Interventional

Safety and Efficacy of PMT Therapy of hPAP

NCT05761899
View directory record Official record
Conditions
Hereditary Pulmonary Alveolar Proteinosis
Interventions
Gene-Corrected Macrophages administered by bronchoscopic instillation
Combination Product
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
18 Years and older
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2038
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Dec 18, 2025 · Synced Sep 23, 2026, 2:52 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 1, 2017 · Synced Sep 23, 2026, 2:52 PM EDT
Withdrawn Phase 2Phase 3 Interventional

A Clinical Study to Enable Process Validation of Commercial Grade OTL-101

NCT04140539
View directory record Official record
Conditions
Severe Combined Immunodeficiency Due to ADA Deficiency
Interventions
OTL-101
Biological
Lead sponsor
University of California, Los Angeles
Other
Eligibility
30 Days to 17 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Aug 2, 2022 · Synced Sep 23, 2026, 2:52 PM EDT
Suspended Phase 1Phase 2 Interventional

Gene Transfer for X-Linked Severe Combined Immunodeficiency in Newly Diagnosed Infants

NCT01512888
View directory record Official record
Conditions
Severe Combined Immunodeficiency Disease, X-linked
Interventions
CL20-i4-EF1α-hγc-OPT, Busulfan, CliniMacs
Genetic · Drug · Device
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
Up to 24 Months · Male only
Enrollment
28 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2034
U.S. locations
3
States / cities
San Francisco, California • Memphis, Tennessee • Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Nov 9, 2025 · Synced Sep 23, 2026, 2:52 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 11, 2019 · Synced Sep 23, 2026, 2:52 PM EDT
Recruiting Phase 1 Interventional

Assessing the Safety, Tolerability, and Efficacy of APR-2020 in Pediatric and Adolescent Subjects With RPS19 Deficient Diamond-Blackfan Anemia

NCT07476183
View directory record Official record
Conditions
RPS19 Deficient Diamond-Blackfan Anemia
Interventions
APR-2020
Biological
Lead sponsor
Apriligen, Inc.
Industry
Eligibility
2 Years to 25 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
2
States / cities
Stanford, California • Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jul 20, 2026 · Synced Sep 23, 2026, 2:52 PM EDT