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ClinicalTrials.gov public records Last synced Sep 24, 2026, 4:42 PM EDT

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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Filters: Intervention: Transduced CD34+ Cells Clear all
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 24, 2026, 4:42 PM EDT
Active, not recruiting Phase 1 Interventional Results available

HERV-E TCR Transduced Autologous T Cells in People With Metastatic Clear Cell Renal Cell Carcinoma

NCT03354390
View directory record Official record
Conditions
Kidney Cancer
Interventions
cell infusion
Biological
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
18 Years to 75 Years
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2029
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Apr 27, 2026 · Synced Sep 24, 2026, 4:42 PM EDT
Completed No phase listed Observational

Gene Transfer Therapy for Severe Combined Immunodeficieny Disease (SCID) Due to Adenosine Deaminase (ADA) Deficiency: A Natural History Study

NCT00001255
View directory record Official record
Conditions
Severe Combined Immunodeficiency
Interventions
ADA PBSC, ADA Umbilical Cord Blood Cells, Transduced Lymphocytes
Drug
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
Not listed
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1990 – 2002
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 3, 2008 · Synced Sep 24, 2026, 4:42 PM EDT
Completed Phase 2Phase 3 Interventional Results available

A Study of the Efficacy and Safety of Hematopoietic Stem Cells Transduced With Lenti-D Lentiviral Vector for the Treatment of Cerebral Adrenoleukodystrophy (CALD)

NCT01896102
View directory record Official record
Conditions
Cerebral Adrenoleukodystrophy (CALD)
Interventions
Lenti-D Drug Product (eli-cel)
Genetic
Lead sponsor
Genetix Biotherapeutics Inc.
Industry
Eligibility
Up to 17 Years · Male only
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2021
U.S. locations
3
States / cities
Los Angeles, California • Boston, Massachusetts • Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Apr 24, 2022 · Synced Sep 24, 2026, 4:42 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 24, 2026, 4:42 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 2, 2022 · Synced Sep 24, 2026, 4:42 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 24, 2026, 4:42 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 24, 2026, 4:42 PM EDT
Recruiting Phase 1Phase 2 Interventional

Phase I/II Trial of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan Conditioning

NCT03311503
View directory record Official record
Conditions
Severe Combined Immunodeficiency, X Linked, Gene Therapy
Interventions
autologous CD34+ cell transduced with G2SCID vector
Biological
Lead sponsor
David Williams
Other
Eligibility
0 Years to 5 Years · Male only
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2028
U.S. locations
4
States / cities
Los Angeles, California • Atlanta, Georgia • Boston, Massachusetts + 1 more
Source: ClinicalTrials.gov public record
Updated Dec 10, 2025 · Synced Sep 24, 2026, 4:42 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 24, 2026, 4:42 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 24, 2026, 4:42 PM EDT
Active, not recruiting Phase 3 Interventional

A Clinical Study to Evaluate the Use of a Cryopreserved Formulation of OTL-103 in Subjects With Wiskott-Aldrich Syndrome

NCT03837483
View directory record Official record
Conditions
Wiskott-Aldrich Syndrome
Interventions
OTL-103
Genetic
Lead sponsor
Fondazione Telethon
Other
Eligibility
Up to 65 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2027
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Sep 28, 2025 · Synced Sep 24, 2026, 4:42 PM EDT
Completed Phase 2 Interventional Results available

MND-ADA Transduction of CD34+ Cells From Children With ADA-SCID

NCT00794508
View directory record Official record
Conditions
Severe Combined Immunodeficiency
Interventions
ADA gene transfer
Biological
Lead sponsor
Donald B. Kohn, M.D.
Other
Eligibility
1 Month to 18 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2015
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Apr 22, 2021 · Synced Sep 24, 2026, 4:42 PM EDT
Completed Phase 1Phase 2 Interventional

Study of Gene Therapy Using a Lentiviral Vector to Treat X-linked Chronic Granulomatous Disease

NCT02234934
View directory record Official record
Conditions
Granulomatous Disease, Chronic, X-linked
Interventions
Lentiviral G1XCGD Gene Therapy
Biological
Lead sponsor
University of California, Los Angeles
Other
Eligibility
23 Months and older · Male only
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2024
U.S. locations
3
States / cities
Los Angeles, California • Bethesda, Maryland • Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated May 1, 2025 · Synced Sep 24, 2026, 4:42 PM EDT
Completed Phase 1 Interventional

Infusion of Genetically Modified T Cell for Post Transplant Patients With Relapsed Disease

NCT00871702
View directory record Official record
Conditions
Leukemia, Lymphoma, Non-Hodgkin, Hodgkin Disease, Myelodysplastic Syndromes, Multiple Myeloma
Interventions
CD34-TK75 transduced donor lymphocytes, Sub Study - 18 FHBG PET/CT Scans
Genetic · Radiation
Lead sponsor
Washington University School of Medicine
Other
Eligibility
18 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2012
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Jul 10, 2013 · Synced Sep 24, 2026, 4:42 PM EDT
Enrolling by invitation No phase listed Observational

Long-Term Follow-up: Phase I/II Clinical Study to Evaluate the Safety and Efficacy of the Infusion of RP-L102

NCT07527975
View directory record Official record
Conditions
Fanconi Anemia
Interventions
RP-L102
Biological
Lead sponsor
Rocket Pharmaceuticals Inc.
Industry
Eligibility
Not listed
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2038
U.S. locations
1
States / cities
Palo Alto, California
Source: ClinicalTrials.gov public record
Updated Apr 13, 2026 · Synced Sep 24, 2026, 4:42 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 24, 2026, 4:42 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 24, 2026, 4:42 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 12, 2022 · Synced Sep 24, 2026, 4:42 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 24, 2026, 4:42 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 2, 2022 · Synced Sep 24, 2026, 4:42 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 24, 2026, 4:42 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 4, 2018 · Synced Sep 24, 2026, 4:42 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 24, 2026, 4:42 PM EDT