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ClinicalTrials.gov public records Last synced Oct 22, 2026, 5:10 AM EDT

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Showing 1–24 of 838 matching trials from the live ClinicalTrials.gov search.
Filters: Intervention: Gene transfer Clear all
Completed Phase 1 Interventional

A Phase I Study of AdV-tk + Prodrug Therapy in Combination With Radiation Therapy for Pediatric Brain Tumors

NCT00634231
View directory record Official record
Conditions
Malignant Glioma, Recurrent Ependymoma
Interventions
AdV-tk, valacyclovir, Radiation
Biological · Drug · Radiation
Lead sponsor
Candel Therapeutics, Inc.
Industry
Eligibility
3 Years to 21 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2021
U.S. locations
2
States / cities
Chicago, Illinois • Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Aug 25, 2023 · Synced Oct 22, 2026, 5:10 AM EDT
Completed Phase 1 Interventional

A Phase I Study of Systemic Gene Therapy With SGT-94 in Patients With Solid Tumors

NCT01517464
View directory record Official record
Conditions
Neoplasm
Interventions
SGT-94
Genetic
Lead sponsor
SynerGene Therapeutics, Inc.
Industry
Eligibility
18 Years and older
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2015
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Apr 26, 2017 · Synced Oct 22, 2026, 5:10 AM EDT
Recruiting Phase 1Phase 2 Interventional

Study to Evaluate ACDN-01 in ABCA4-related Stargardt Retinopathy (STELLAR)

NCT06467344
View directory record Official record
Conditions
Stargardt Disease, Cone Rod Dystrophy, Juvenile Macular Degeneration, Stargardt Disease 1
Interventions
ACDN-01
Drug
Lead sponsor
Ascidian Therapeutics, Inc
Industry
Eligibility
12 Years and older
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2030
U.S. locations
11
States / cities
San Francisco, California • Gainesville, Florida • Miami, Florida + 8 more
Source: ClinicalTrials.gov public record
Updated Oct 7, 2026 · Synced Oct 22, 2026, 5:10 AM EDT
Recruiting Phase 1Phase 2 Interventional

Quaratusugene Ozeplasmid (Reqorsa) and Atezolizumab Maintenance Therapy in ES-SCLC Patients

NCT05703971
View directory record Official record
Conditions
Small Cell Lung Cancer Extensive Stage
Interventions
quaratusugene ozeplasmid, atezolizumab
Biological
Lead sponsor
Genprex, Inc.
Industry
Eligibility
18 Years and older
Enrollment
62 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2028
U.S. locations
17
States / cities
Lone Tree, Colorado • St Louis, Missouri • Canton, Ohio + 9 more
Source: ClinicalTrials.gov public record
Updated Sep 11, 2026 · Synced Oct 22, 2026, 5:10 AM EDT
Recruiting Phase 1 Interventional

C7R-GD2.CAR T Cells for Patients With GD2-expressing Brain Tumors (GAIL-B)

NCT04099797
View directory record Official record
Conditions
Diffuse Intrinsic Pontine Glioma, High Grade Glioma, Embryonal Tumor, Ependymal Tumor
Interventions
C7R-GD2.CART cells (IV and ICV infusion)
Genetic
Lead sponsor
Baylor College of Medicine
Other
Eligibility
12 Months to 25 Years
Enrollment
56 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2041
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Aug 3, 2026 · Synced Oct 22, 2026, 5:10 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Gene Therapy for Cardiomyopathy Associated With Friedreich's Ataxia

NCT05445323
View directory record Official record
Conditions
Friedreich Ataxia, Cardiomyopathy, Secondary
Interventions
Low dose LX2006, Mid Dose LX2006, High Dose LX2006
Genetic
Lead sponsor
Lexeo Therapeutics
Industry
Eligibility
18 Years to 50 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2029
U.S. locations
3
States / cities
Los Angeles, California • Tampa, Florida • Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Dec 23, 2025 · Synced Oct 22, 2026, 5:10 AM EDT
Completed Phase 2 Interventional Results available

Choroideremia Gene Therapy Clinical Trial

NCT02553135
View directory record Official record
Conditions
Choroideremia
Interventions
Injection of AAV2-REP1 (10e11 vg)
Biological
Lead sponsor
Byron Lam
Other
Eligibility
18 Years and older · Male only
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2018
U.S. locations
1
States / cities
Miami, Florida
Source: ClinicalTrials.gov public record
Updated Jul 29, 2019 · Synced Oct 22, 2026, 5:10 AM EDT
Recruiting Phase 3 Interventional

Single Intravitreal (IVT) Injection of 4D-150 in Patients With Macular Neovascularization Secondary to Age-Related Macular Degeneration

NCT07064759
View directory record Official record
Conditions
Neovascular Age-Related Macular Degeneration (nAMD)
Interventions
4D-150 IVT (3E10 vg/eye), EYLEA® (aflibercept) Injection 2 mg (0.05mL)
Biological
Lead sponsor
4D Molecular Therapeutics
Industry
Eligibility
50 Years and older
Enrollment
480 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2029
U.S. locations
60
States / cities
Scottsdale, Arizona • Sun City, Arizona • Tucson, Arizona + 55 more
Source: ClinicalTrials.gov public record
Updated Oct 19, 2026 · Synced Oct 22, 2026, 5:10 AM EDT
Recruiting Phase 4 Interventional

ASsessing The REAl-world Safety & Effectiveness of Spinal Muscular Atrophy Participants Treated With Intrathecal Onasemnogene Abeparvovec-brve (OAV101B) (ITVISMA®): A U.S. Pragmatic Multicenter Study (STREAM)

NCT07448610
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
Onasemnogene Abeparvovec-brve
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
2 Years to 100 Years
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2032
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Sep 18, 2026 · Synced Oct 22, 2026, 5:10 AM EDT
Completed Phase 3 Interventional Results available

Phase IIIb, Open-label, Multi-center Study to Evaluate Safety, Tolerability and Efficacy of OAV101 Administered Intrathecally to Participants With SMA Who Discontinued Treatment With Nusinersen or Risdiplam

NCT05386680
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
OAV101
Genetic
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
2 Years to 17 Years
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
3
States / cities
Boston, Massachusetts • Norfolk, Virginia • Madison, Wisconsin
Source: ClinicalTrials.gov public record
Updated Jan 13, 2026 · Synced Oct 22, 2026, 5:10 AM EDT
Enrolling by invitation No phase listed Observational

Long-term Follow-up of Subjects with Sickle Cell Disease Treated with Ex Vivo Gene Therapy

NCT04628585
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Safety and efficacy assessments
Other
Lead sponsor
Genetix Biotherapeutics Inc.
Industry
Eligibility
2 Years to 53 Years
Enrollment
85 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2038
U.S. locations
15
States / cities
Birmingham, Alabama • Oakland, California • Atlanta, Georgia + 12 more
Source: ClinicalTrials.gov public record
Updated Mar 20, 2025 · Synced Oct 22, 2026, 5:10 AM EDT
Completed Phase 1Phase 2 Interventional Results available

CAR T Cell Receptor Immunotherapy Targeting EGFRvIII for Patients With Malignant Gliomas Expressing EGFRvIII

NCT01454596
View directory record Official record
Conditions
Malignant Glioma, Glioblastoma, Brain Cancer, Gliosarcoma
Interventions
Epidermal growth factor receptor(EGFRv)III Chimeric antigen receptor (CAR) transduced PBL, Aldesleukin, Fludarabine, Cyclophosphamide
Biological · Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years to 70 Years
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2019
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 21, 2019 · Synced Oct 22, 2026, 5:10 AM EDT
Recruiting Phase 1 Interventional

Modulation of SERCA2a of Intra-Myocytic Calcium Trafficking in Cardiomyopathy Secondary to Duchenne Muscular Dystrophy

NCT06224660
View directory record Official record
Conditions
DMD-Associated Dilated Cardiomyopathy
Interventions
SRD-001
Genetic
Lead sponsor
Sardocor Corp.
Industry
Eligibility
18 Years and older · Male only
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2030
U.S. locations
3
States / cities
Kansas City, Kansas • Cincinnati, Ohio • Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Feb 27, 2025 · Synced Oct 22, 2026, 5:10 AM EDT
Active, not recruiting No phase listed Observational

Long - Term Follow Up of Sickle Cell Disease and Beta-thalassemia Subjects Previously Exposed to BIVV003 or ST-400.

NCT05145062
View directory record Official record
Conditions
Blood and Lymphatic Diseases
Interventions
BIVV003, ST-400
Drug
Lead sponsor
Sangamo Therapeutics
Industry
Eligibility
18 Years to 45 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2038
U.S. locations
7
States / cities
Oakland, California • Sacramento, California • Atlanta, Georgia + 3 more
Source: ClinicalTrials.gov public record
Updated Nov 12, 2025 · Synced Oct 22, 2026, 5:10 AM EDT
Completed Phase 2 Interventional Results available

Redirected Auto T Cells for Advanced Myeloma

NCT01352286
View directory record Official record
Conditions
Multiple Myeloma
Interventions
Autologous Genetically modified T cells
Genetic
Lead sponsor
GlaxoSmithKline
Industry
Eligibility
18 Years to 80 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2019
U.S. locations
2
States / cities
Baltimore, Maryland • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Dec 3, 2019 · Synced Oct 22, 2026, 5:10 AM EDT
Not listed Phase 2 Interventional

Safety and Efficacy of p53 Gene Therapy Combined With Immune Checkpoint Inhibitors in Solid Tumors.

NCT03544723
View directory record Official record
Conditions
Solid Tumor, Lymphoma
Interventions
Ad-p53
Drug
Lead sponsor
MultiVir, Inc.
Industry
Eligibility
18 Years and older
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2022
U.S. locations
3
States / cities
Chicago, Illinois • Morristown, New Jersey
Source: ClinicalTrials.gov public record
Updated Jun 9, 2020 · Synced Oct 22, 2026, 5:10 AM EDT
Terminated Phase 1 Interventional

Phase 1 Study of GEN2 in Patients With Advanced Solid Tumors

NCT06391918
View directory record Official record
Conditions
Solid Tumor, Adult
Interventions
GEN2 + Valganciclovir
Biological
Lead sponsor
GenVivo, Inc.
Industry
Eligibility
18 Years and older
Enrollment
37 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
7
States / cities
Duarte, California • Los Angeles, California • Indianapolis, Indiana + 3 more
Source: ClinicalTrials.gov public record
Updated Mar 13, 2026 · Synced Oct 22, 2026, 5:10 AM EDT
Recruiting No phase listed Observational

Non-interventional Study of Seroprevalence of Pre-existing Antibodies Against Adenovirus-associated Virus Vector (AAV9) and the Progression of Disease in Patients With Plakophilin 2 (PKP2)-Associated Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC)

NCT06311708
View directory record Official record
Conditions
Arrhythmogenic Right Ventricular Cardiomyopathy
Interventions
Not listed
Lead sponsor
Tenaya Therapeutics
Industry
Eligibility
14 Years to 65 Years
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2030
U.S. locations
8
States / cities
San Francisco, California • Aurora, Colorado • Baltimore, Maryland + 5 more
Source: ClinicalTrials.gov public record
Updated Nov 1, 2024 · Synced Oct 22, 2026, 5:10 AM EDT
Active, not recruiting Phase 1 Interventional

Gene Therapy and Combination Chemotherapy in Treating Patients With AIDS-Related Non-Hodgkin Lymphoma

NCT02337985
View directory record Official record
Conditions
AIDS-Related Burkitt Lymphoma, AIDS-Related Diffuse Large B-cell Lymphoma, AIDS-Related Plasmablastic Lymphoma, AIDS-Related Primary Effusion Lymphoma, HIV Infection, AIDS Related Non-Hodgkin Lymphoma
Interventions
Prednisone, Rituximab, Etoposide, Doxorubicin Hydrochloride, Vincristine Sulfate, Cyclophosphamide, Filgrastim, Lentivirus Vector rHIV7-shI-TAR-CCR5RZ-transduced Hematopoietic Stem/Progenitor Cells
Drug · Biological
Lead sponsor
City of Hope Medical Center
Other
Eligibility
18 Years to 65 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2027
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 1, 2026 · Synced Oct 22, 2026, 5:10 AM EDT
Active, not recruiting Phase 1 Interventional

Safety Study of Zinc Finger Nuclease CCR5-modified Hematopoietic Stem/Progenitor Cells in HIV-1 Infected Patients

NCT02500849
View directory record Official record
Conditions
HIV
Interventions
SB-728mR-HSPC Infusion 3 days following busulfan conditioning
Genetic
Lead sponsor
City of Hope Medical Center
Other
Eligibility
18 Years to 75 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2027
U.S. locations
5
States / cities
Duarte, California • Los Angeles, California • San Francisco, California + 1 more
Source: ClinicalTrials.gov public record
Updated Oct 2, 2026 · Synced Oct 22, 2026, 5:10 AM EDT
Completed Phase 1 Interventional

Gene Transfer for Cancer Pain

NCT00804076
View directory record Official record
Conditions
Cancer Pain
Interventions
NP2
Biological
Lead sponsor
Diamyd Inc
Industry
Eligibility
18 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2013
U.S. locations
5
States / cities
Miami, Florida • New Orleans, Louisiana • Ann Arbor, Michigan + 2 more
Source: ClinicalTrials.gov public record
Updated Feb 19, 2014 · Synced Oct 22, 2026, 5:10 AM EDT
Active, not recruiting Phase 1 Interventional

Dose Escalation Study of Intravitreal 4D-110 in Patients With Choroideremia

NCT04483440
View directory record Official record
Conditions
Choroideremia
Interventions
4D-110
Biological
Lead sponsor
4D Molecular Therapeutics
Industry
Eligibility
18 Years and older · Male only
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2027
U.S. locations
2
States / cities
Dallas, Texas • Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated May 22, 2025 · Synced Oct 22, 2026, 5:10 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Therapeutic Vaccination in Treated HIV Disease

NCT03606213
View directory record Official record
Conditions
HIV-1-infection
Interventions
PENNVAX-GP, INO-6145, INO-9012, CELLECTRA® 2000
Biological · Device
Lead sponsor
Steven Deeks
Other
Eligibility
18 Years to 65 Years
Enrollment
56 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
2
States / cities
Los Angeles, California • San Francisco, California
Source: ClinicalTrials.gov public record
Updated May 31, 2023 · Synced Oct 22, 2026, 5:10 AM EDT
Completed Phase 1 Interventional

Gene Therapy in Treating Patients With Advanced Recurrent or Persistent Ovarian Cancer or Primary Peritoneal Cancer

NCT00003450
View directory record Official record
Conditions
Ovarian Cancer, Peritoneal Cavity Cancer
Interventions
Ad5CMV-p53 gene
Biological
Lead sponsor
University of Texas Southwestern Medical Center
Other
Eligibility
18 Years to 120 Years · Female only
Healthy volunteers
Healthy volunteers not accepted
Timeline
1998 – 1999
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Feb 2, 2021 · Synced Oct 22, 2026, 5:10 AM EDT