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ClinicalTrials.gov public records Last synced Oct 21, 2026, 10:08 AM EDT

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Showing 1–24 of 1,664 matching trials from the live ClinicalTrials.gov search.
Filters: Intervention: gene therapy Clear all
Terminated Phase 1 Interventional

The Seven Trial: Exploiting the Unfolded Protein Response

NCT06076837
View directory record Official record
Conditions
Pancreatic Cancer Metastatic, Pancreatic Adenocarcinoma Metastatic
Interventions
Botensilimab, Balstilimab, Chloroquine Phosphate, Celecoxib, Nab paclitaxel, Gemcitabine, Cisplatin
Drug
Lead sponsor
HonorHealth Research Institute
Other
Eligibility
18 Years and older
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025
U.S. locations
1
States / cities
Scottsdale, Arizona
Source: ClinicalTrials.gov public record
Updated Jun 23, 2026 · Synced Oct 21, 2026, 10:08 AM EDT
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 21, 2026, 10:08 AM EDT
Completed Phase 2 Interventional Results available

Docetaxel and Carboplatin in Treating Patients With Metastatic, Castration Resistant Prostate Cancer Containing Inactivated Genes in the BRCA 1/2 Pathway

NCT02598895
View directory record Official record
Conditions
Castration Levels of Testosterone, Castration-Resistant Prostate Carcinoma, Metastatic Prostate Carcinoma, Prostate Carcinoma Metastatic in the Bone, PSA Progression, Stage IV Prostate Cancer
Interventions
Carboplatin, Docetaxel, Laboratory Biomarker Analysis
Drug · Other
Lead sponsor
University of Washington
Other
Eligibility
18 Years and older · Male only
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2021
U.S. locations
1
States / cities
Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Nov 17, 2022 · Synced Oct 21, 2026, 10:08 AM EDT
Completed Phase 4 Interventional Results available

Liraglutide Effects on Epicardial Fat Inflammatory Genes

NCT03260881
View directory record Official record
Conditions
Type2 Diabetes, Coronary Artery Disease
Interventions
Liraglutide Pen Injector [Victoza], matching liraglutide-placebo pre-filled pens
Drug
Lead sponsor
University of Miami
Other
Eligibility
18 Years and older
Enrollment
38 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2024
U.S. locations
1
States / cities
Miami, Florida
Source: ClinicalTrials.gov public record
Updated Jul 8, 2025 · Synced Oct 21, 2026, 10:08 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 21, 2026, 10:08 AM EDT
Completed Phase 2 Interventional

Study of Crenolanib in Recurrent/Refractory Glioblastoma With PDGFRA Gene Amplification

NCT02626364
View directory record Official record
Conditions
Recurrent/Refractory Glioblastoma
Interventions
crenolanib
Drug
Lead sponsor
Arog Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2020
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jul 20, 2020 · Synced Oct 21, 2026, 10:08 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 21, 2026, 10:08 AM EDT
Completed Phase 2 Interventional Results available

Melphalan, Peripheral Stem Cell Transplantation, and Interleukin-2 Followed by Interferon Alfa in Treating Patients With Advanced Multiple Myeloma

NCT00006244
View directory record Official record
Conditions
Refractory Multiple Myeloma, Stage I Multiple Myeloma, Stage II Multiple Myeloma, Stage III Multiple Myeloma
Interventions
melphalan, recombinant interferon alfa, aldesleukin, in vitro-treated peripheral blood stem cell transplantation
Drug · Biological · Procedure
Lead sponsor
Fred Hutchinson Cancer Center
Other
Eligibility
18 Years to 69 Years
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2016
U.S. locations
1
States / cities
Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Jul 12, 2017 · Synced Oct 21, 2026, 10:08 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 21, 2026, 10:08 AM EDT
Recruiting Phase 1 Interventional

Mitoxantrone for Venetoclax Resistant Acute Myeloid Leukemia

NCT06429449
View directory record Official record
Conditions
Leukemia, Myeloid Leukemia, Monocytic Leukemia
Interventions
Venetoclax, Azacitidine, Mitoxantrone
Drug
Lead sponsor
University of Colorado, Denver
Other
Eligibility
18 Years to 80 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
1
States / cities
Aurora, Colorado
Source: ClinicalTrials.gov public record
Updated Jan 15, 2026 · Synced Oct 21, 2026, 10:08 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 21, 2026, 10:08 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 21, 2026, 10:08 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 21, 2026, 10:08 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 21, 2026, 10:08 AM EDT
Completed Not applicable Interventional

Safety Study of Preservon-Treated Bone Implants for Cervical Fusion in the Treatment of Cervical Radiculopathy or Myelopathy

NCT00344890
View directory record Official record
Conditions
Cervical Radiculopathy, Myelopathy
Interventions
Anterior Cervical Discectomy and Fusion
Procedure
Lead sponsor
LifeNet Health
Industry
Eligibility
18 Years to 65 Years
Enrollment
106 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2012
U.S. locations
1
States / cities
Richmond, Virginia
Source: ClinicalTrials.gov public record
Updated Apr 7, 2015 · Synced Oct 21, 2026, 10:08 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 21, 2026, 10:08 AM EDT
Terminated Phase 2 Interventional Results available

Study of Seribantumab in Adult Patients With NRG1 Gene Fusion Positive Advanced Solid Tumors

NCT04383210
View directory record Official record
Conditions
Locally Advanced Solid Tumor, Metastatic Solid Tumor, Pancreatic Cancer, Lung Cancer, Head and Neck Cancer, Breast Cancer, Kidney Cancer, Colorectal Cancer, Bladder Cancer, Ovarian Cancer, Sarcoma, Gallbladder Cancer, Bile Duct Cancer, Esophageal Cancer, Uterine Cancer, Cholangiocarcinoma, Prostate Cancer
Interventions
Seribantumab
Drug
Lead sponsor
Elevation Oncology
Industry
Eligibility
18 Years and older
Enrollment
54 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2024
U.S. locations
35
States / cities
Birmingham, Alabama • Scottsdale, Arizona • Fountain Valley, California + 32 more
Source: ClinicalTrials.gov public record
Updated Apr 3, 2025 · Synced Oct 21, 2026, 10:08 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 21, 2026, 10:08 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 21, 2026, 10:08 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 21, 2026, 10:08 AM EDT
Completed No phase listed Observational

Gene Mutations in Patients With Advanced Prostate Cancer That Is Not Responsive to Hormone Therapy

NCT00002924
View directory record Official record
Conditions
Prostate Cancer
Interventions
mutation analysis
Other
Lead sponsor
Alliance for Clinical Trials in Oncology
Other
Eligibility
Male only
Enrollment
184 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1997 – 2006
U.S. locations
49
States / cities
Birmingham, Alabama • La Jolla, California • San Francisco, California + 30 more
Source: ClinicalTrials.gov public record
Updated Jul 14, 2016 · Synced Oct 21, 2026, 10:08 AM EDT
Completed No phase listed Observational

Genetic Markers in Patients With Colorectal Cancer

NCT00014079
View directory record Official record
Conditions
Colorectal Cancer
Interventions
DNA stability analysis, loss of heterozygosity analysis, microsatellite instability analysis
Genetic
Lead sponsor
Alliance for Clinical Trials in Oncology
Other
Eligibility
Not listed
Enrollment
675 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1997 – 2005
U.S. locations
3
States / cities
Scottsdale, Arizona • Jacksonville, Florida • Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Jul 13, 2016 · Synced Oct 21, 2026, 10:08 AM EDT
Completed Phase 1 Interventional

Allopregnanolone as a Regenerative Treatment for Parkinson's Disease

NCT06263010
View directory record Official record
Conditions
Parkinson Disease
Interventions
Allopregnanolone
Drug
Lead sponsor
Roberta Brinton
Other
Eligibility
40 Years to 85 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2025
U.S. locations
1
States / cities
Tucson, Arizona
Source: ClinicalTrials.gov public record
Updated Mar 13, 2026 · Synced Oct 21, 2026, 10:08 AM EDT
Active, not recruiting Phase 2 Interventional Results available

Testing Trastuzumab and Pertuzumab in Patients With Higher Than Normal Copies of the HER2 Gene Found in Their Tumors (MATCH - Subprotocol J)

NCT06136897
View directory record Official record
Conditions
Malignant Solid Neoplasm
Interventions
Biopsy Procedure, Biospecimen Collection, Echocardiography Test, Pertuzumab, Radiologic Examination, Trastuzumab
Procedure · Biological
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years and older
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2027
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jul 30, 2026 · Synced Oct 21, 2026, 10:08 AM EDT