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ClinicalTrials.gov public records Last synced Oct 19, 2026, 1:41 PM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Showing 49–72 of 838 matching trials from the live ClinicalTrials.gov search.
Filters: Intervention: Gene Transfer Clear all
Suspended Phase 1 Interventional

MT2022-01: MSCs for ALD

NCT06030648
View directory record Official record
Conditions
Cerebral Adrenoleukodystrophy
Interventions
Mesenchymal stem cells (IV-MSC)
Biological
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
3 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2029
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jun 18, 2026 · Synced Oct 19, 2026, 1:41 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 19, 2026, 1:41 PM EDT
Terminated Phase 1 Interventional

Phase 1/2 Study of Metastatic Renal Cancer Using T-Cells Transduced With a T-Cell Receptor Which Recognizes TRAIL Bound to the DR4 Receptor

NCT00923390
View directory record Official record
Conditions
Renal Cancer, Kidney Cancer
Interventions
2G-1 TCR Retroviral Vector-Transduced lyn, Aldesleukin, Cyclophosphamide, Fludarabine, (PG13-A(F/K-F-SGSG-T2a-B (opt) (2G-1 TCR) retroviral vector-transduced lymphocyte
Genetic · Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years to 99 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2012
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Apr 9, 2019 · Synced Oct 19, 2026, 1:41 PM EDT
Terminated Phase 1Phase 2 Interventional Results available

Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-318 in Subjects With MPS I

NCT02702115
View directory record Official record
Conditions
MPS I
Interventions
SB-318
Biological
Lead sponsor
Sangamo Therapeutics
Industry
Eligibility
5 Years and older
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2021
U.S. locations
1
States / cities
Oakland, California
Source: ClinicalTrials.gov public record
Updated Jan 26, 2023 · Synced Oct 19, 2026, 1:41 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Lessons Learned From the Family Gene Toolkit

NCT02154633
View directory record Official record
Conditions
Women With BRCA 1 or BRCA 2 Mutation, Non-tested Female Family Members
Interventions
Family Gene Toolkit, Delayed Family Gene Toolkit
Behavioral
Lead sponsor
University of Michigan
Other
Eligibility
18 Years and older · Female only
Enrollment
13 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2010 – 2017
U.S. locations
1
States / cities
Ann Arbor, Michigan
Source: ClinicalTrials.gov public record
Updated Feb 25, 2020 · Synced Oct 19, 2026, 1:41 PM EDT
Completed Phase 1Phase 2 Interventional Results available

AAV9 U7snRNA Gene Therapy to Treat Boys With DMD Exon 2 Duplications.

NCT04240314
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
scAAV9.U7.ACCA
Biological
Lead sponsor
Megan Waldrop
Other
Eligibility
6 Months to 13 Years · Male only
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2025
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Sep 11, 2025 · Synced Oct 19, 2026, 1:41 PM EDT
Completed Phase 1 Interventional Results available

Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1

NCT02122952
View directory record Official record
Conditions
Spinal Muscular Atrophy 1
Interventions
AVXS-101
Biological
Lead sponsor
Novartis Gene Therapies
Industry
Eligibility
Up to 6 Months
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2017
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Sep 15, 2022 · Synced Oct 19, 2026, 1:41 PM EDT
Recruiting No phase listed Observational

CIBMTR Research Database

NCT01166009
View directory record Official record
Conditions
Autologous Stem Cell Transplantation, Allogeneic Stem Cell Transplantation, Solid Tumors, Blood Cancers, CAR-T, Gene Therapy, Non-malignant Disease
Interventions
Not listed
Lead sponsor
Center for International Blood and Marrow Transplant Research
Network
Eligibility
Not listed
Enrollment
99,999,999 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2050
U.S. locations
2
States / cities
Minneapolis, Minnesota • Milwaukee, Wisconsin
Source: ClinicalTrials.gov public record
Updated Aug 12, 2025 · Synced Oct 19, 2026, 1:41 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

UCSF Center for Genome Surgery Biobank and Registry

NCT07396285
View directory record Official record
Conditions
Any Conditions That Are Genetic or Suspected to be Genetic in Origin
Interventions
Not listed
Lead sponsor
University of California, San Francisco
Other
Eligibility
Not listed
Enrollment
10,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2026 – 2050
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Sep 1, 2026 · Synced Oct 19, 2026, 1:41 PM EDT
Terminated Phase 1 Interventional Results available

A Study to Evaluate the Safety and Tolerability of PF-06939926 Gene Therapy in Duchenne Muscular Dystrophy

NCT03362502
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
PF-06939926
Genetic
Lead sponsor
Pfizer
Industry
Eligibility
4 Years and older · Male only
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2025
U.S. locations
22
States / cities
Los Angeles, California • Durham, North Carolina • Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated Sep 15, 2025 · Synced Oct 19, 2026, 1:41 PM EDT
Completed No phase listed Observational

Needs Assessment of Knowledge, Beliefs, and Attitudes of Patients With Hemophilia B About Gene Therapy

NCT05044845
View directory record Official record
Conditions
Gene Therapy, Hemophilia B
Interventions
Interview
Behavioral
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
12 Years and older
Enrollment
150 participants
Timeline
2022 – 2025
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Nov 14, 2025 · Synced Oct 19, 2026, 1:41 PM EDT
Completed Phase 1 Interventional

Gene Modified Allogeneic Neuroblastoma Cells For Treatment of Relapsed/Refractory Neuroblastoma

NCT00186862
View directory record Official record
Conditions
Neuroblastoma
Interventions
Interleukin-2
Drug
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
Up to 21 Years
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1998 – 2007
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Jun 3, 2008 · Synced Oct 19, 2026, 1:41 PM EDT
Terminated Phase 2 Interventional

Study of AAV-GAD Gene Transfer Into the Subthalamic Nucleus for Parkinson's Disease

NCT00643890
View directory record Official record
Conditions
Parkinson's Disease
Interventions
Bilateral surgical infusion of AAV-GAD into the subthalamic nucleus
Genetic
Lead sponsor
Neurologix, Inc.
Industry
Eligibility
30 Years to 75 Years
Enrollment
44 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2008
U.S. locations
7
States / cities
Stanford, California • Aurora, Colorado • Boston, Massachusetts + 4 more
Source: ClinicalTrials.gov public record
Updated Feb 22, 2012 · Synced Oct 19, 2026, 1:41 PM EDT
Completed Phase 1 Interventional

HPV-16/18 E6/E7-Specific T Lymphocytes, Relapsed HPV-Associated Cancers, HESTIA

NCT02379520
View directory record Official record
Conditions
Human Papillomavirus-Related Carcinoma, Human Papillomavirus Positive Oropharyngeal Carcinoma, Human Papillomavirus Positive Cervical Carcinoma, Human Papillomavirus Positive Anal Carcinoma, Human Papillomavirus Positive Vulvar Carcinoma, Human Papillomavirus Positive Penile Carcinoma
Interventions
HPV Specific T Cells, Cytoxan, Fludarabine, Nivolumab
Genetic · Drug · Biological
Lead sponsor
Baylor College of Medicine
Other
Eligibility
18 Years and older
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2026
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Oct 2, 2026 · Synced Oct 19, 2026, 1:41 PM EDT
Withdrawn Phase 1 Interventional

Gene Therapy for Post-Operative Atrial Fibrillation

NCT05223725
View directory record Official record
Conditions
Atrial Fibrillation, Post-operative Atrial Fibrillation
Interventions
AdKCNH2-G628S
Biological
Lead sponsor
University of Massachusetts, Worcester
Other
Eligibility
18 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2026
U.S. locations
1
States / cities
Worcester, Massachusetts
Source: ClinicalTrials.gov public record
Updated Apr 1, 2025 · Synced Oct 19, 2026, 1:41 PM EDT
Recruiting Phase 2 Interventional

Investigational Trial to Evaluate XC001 Delivered Via an Cardiac Catheter in Subjects With Chronic Angina.

NCT07048808
View directory record Official record
Conditions
Refractory Angina, Coronary Artery Disease
Interventions
XC001, Sham (No Treatment)
Combination Product
Lead sponsor
XyloCor Therapeutics, Inc.
Industry
Eligibility
18 Years to 85 Years
Enrollment
106 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
14
States / cities
Birmingham, Alabama • Phoenix, Arizona • Atlanta, Georgia + 11 more
Source: ClinicalTrials.gov public record
Updated Sep 11, 2026 · Synced Oct 19, 2026, 1:41 PM EDT
Terminated Phase 1 Interventional

Gene Therapy in Treating Patients With Cancer of The Liver

NCT00003147
View directory record Official record
Conditions
Liver Cancer
Interventions
Ad5CMV-p53 gene
Biological
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1998
U.S. locations
2
States / cities
The Bronx, New York • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Feb 5, 2013 · Synced Oct 19, 2026, 1:41 PM EDT
Completed Phase 2 Interventional

Trial of VLTS-589 in Subjects With Intermittent Claudication

NCT00068133
View directory record Official record
Conditions
Intermittent Claudication, Peripheral Vascular Disease
Interventions
Plasmid based Gene Transfer product-VLTS-589
Genetic
Lead sponsor
Valentis
Industry
Eligibility
40 Years to 80 Years
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2005
U.S. locations
20
States / cities
Birmingham, Alabama • Phoenix, Arizona • Beverly Hills, California + 17 more
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 19, 2026, 1:41 PM EDT
Completed Phase 3 Interventional Results available

A Study to Evaluate Prospective Efficacy and Safety Data of Current FIX Prophylaxis Replacement Therapy in Adult Hemophilia B Subjects (FIX:C≤2%) or Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A Subjects (FVIII:C≤1%)

NCT03587116
View directory record Official record
Conditions
Hemophilia B, Hemophilia A
Interventions
Standard of Care FIX Replacement therapy, Standard of Care FVIII Replacement therapy
Drug
Lead sponsor
Pfizer
Industry
Eligibility
18 Years to 64 Years · Male only
Enrollment
212 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2024
U.S. locations
13
States / cities
Palo Alto, California • San Francisco, California • Stanford, California + 7 more
Source: ClinicalTrials.gov public record
Updated Nov 6, 2025 · Synced Oct 19, 2026, 1:41 PM EDT
Completed No phase listed Observational

Immunoscore in Rectal Cancer

NCT02017509
View directory record Official record
Conditions
Cancer of the Rectum, Neoplasms, Rectal, Rectal Cancer, Rectal Tumors, Rectal Adenocarcinoma
Interventions
Diagnostic Biopsy and Surgical Procedure, Intravoxel incoherent motion MRI (IVIM), Dynamic Contrast Enhanced MRI (DCE-MRI), RNA gene expression analysis
Procedure · Genetic
Lead sponsor
Providence Health & Services
Other
Eligibility
18 Years and older
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2018
U.S. locations
2
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Aug 29, 2018 · Synced Oct 19, 2026, 1:41 PM EDT
Completed Not applicable Interventional

Phase I Trial of Intratumoral pIL-12 Electroporation in Malignant Melanoma

NCT00323206
View directory record Official record
Conditions
Malignant Melanoma
Interventions
IL-12p DNA, Intratumoral Electroporation
Biological · Procedure
Lead sponsor
H. Lee Moffitt Cancer Center and Research Institute
Other
Eligibility
18 Years and older
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2008
U.S. locations
1
States / cities
Tampa, Florida
Source: ClinicalTrials.gov public record
Updated Feb 23, 2017 · Synced Oct 19, 2026, 1:41 PM EDT
Terminated Phase 3 Interventional

A Study to Understand the Long-term Safety and Effects of an Experimental Gene Therapy for Duchenne Muscular Dystrophy.

NCT05689164
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
fordadistrogene movaparvovec
Biological
Lead sponsor
Pfizer
Industry
Eligibility
0 Years and older · Male only
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
12
States / cities
Los Angeles, California • Durham, North Carolina • Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated Oct 21, 2025 · Synced Oct 19, 2026, 1:41 PM EDT
Not listed Phase 2 Interventional

Safety and Efficacy Study of INGN 241 Gene Therapy in Patients With In Transit Melanoma

NCT00116363
View directory record Official record
Conditions
Malignant Melanoma, Neoplasm Metastasis
Interventions
investigational drug INGN 241
Genetic
Lead sponsor
Introgen Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2006
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Apr 1, 2008 · Synced Oct 19, 2026, 1:41 PM EDT
Completed Phase 2 Interventional Results available

Dose Confirmation Trial of AAV5-hFIXco-Padua

NCT03489291
View directory record Official record
Conditions
Hemophilia B
Interventions
AAV5-hFIXco-Padua (AMT-061)
Genetic
Lead sponsor
CSL Behring
Industry
Eligibility
18 Years and older · Male only
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2023
U.S. locations
4
States / cities
Phoenix, Arizona • Sacramento, California • San Diego, California + 1 more
Source: ClinicalTrials.gov public record
Updated Jun 28, 2024 · Synced Oct 19, 2026, 1:41 PM EDT