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ClinicalTrials.gov public records Last synced Oct 19, 2026, 4:54 PM EDT

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Showing 121–144 of 838 matching trials from the live ClinicalTrials.gov search.
Filters: Intervention: Gene Transfer Clear all
Recruiting Phase 1Phase 2 Interventional

PS-002 for the Treatment of IgA Nephropathy in Adults

NCT07182227
View directory record Official record
Conditions
Immunoglobulin A (IgA) Nephropathy
Interventions
PS-002
Genetic
Lead sponsor
Purespring Therapeutics Limited
Industry
Eligibility
18 Years and older
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2029
U.S. locations
2
States / cities
Miami, Florida • Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 9, 2026 · Synced Oct 19, 2026, 4:54 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Safety and Efficacy of TSHA-102 in Pediatric Females With Rett Syndrome (REVEAL Pediatric Study)

NCT06152237
View directory record Official record
Conditions
Rett Syndrome
Interventions
TSHA-102
Genetic
Lead sponsor
Taysha Gene Therapies, Inc.
Industry
Eligibility
5 Years to 8 Years · Female only
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2031
U.S. locations
3
States / cities
Chicago, Illinois • Saint Paul, Minnesota • St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Oct 15, 2025 · Synced Oct 19, 2026, 4:54 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Microdystrophin Gene Transfer Study in Adolescents and Children With DMD

NCT03368742
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
SGT-001
Genetic
Lead sponsor
Solid Biosciences Inc.
Industry
Eligibility
4 Years to 17 Years · Male only
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2026
U.S. locations
2
States / cities
Los Angeles, California • Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Oct 15, 2026 · Synced Oct 19, 2026, 4:54 PM EDT
Active, not recruiting Phase 1 Interventional

Safety and Pharmacodynamics of GNSC-001 Intra-articular Injection for Knee Osteoarthritis

NCT05835895
View directory record Official record
Conditions
Osteoarthritis, Knee, Osteo Arthritis Knee, Knee Osteoarthritis
Interventions
GNSC-001, transient immune-modulation, Placebo
Genetic · Drug
Lead sponsor
Genascence Corporation
Industry
Eligibility
40 Years to 75 Years
Enrollment
67 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2029
U.S. locations
9
States / cities
Mobile, Alabama • La Mesa, California • San Diego, California + 6 more
Source: ClinicalTrials.gov public record
Updated Aug 1, 2025 · Synced Oct 19, 2026, 4:54 PM EDT
Completed No phase listed Observational

CART-meso Long-term Follow-up

NCT02388828
View directory record Official record
Conditions
Subjects Who Have Received Lentiviral-based CART-meso Therapy
Interventions
lentiviral-based CART meso therapy
Biological
Lead sponsor
University of Pennsylvania
Other
Eligibility
18 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2019
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jun 29, 2025 · Synced Oct 19, 2026, 4:54 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

A Phase 1/Phase 2 Open-label Study to Evaluate the Safety, Tolerability, and Efficacy of a Single Intravenous Administration of SAR444836 in Adult Participants With Phenylketonuria

NCT05972629
View directory record Official record
Conditions
Phenylketonuria
Interventions
SAR444836
Drug
Lead sponsor
Sanofi
Industry
Eligibility
18 Years to 65 Years
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2030
U.S. locations
7
States / cities
San Francisco, California • Aurora, Colorado • Gainesville, Florida + 4 more
Source: ClinicalTrials.gov public record
Updated Sep 23, 2025 · Synced Oct 19, 2026, 4:54 PM EDT
Terminated Phase 1 Interventional Results available

T Cells Expressing Fully-human Anti-CD19 and Anti-CD20 Chimeric Antigen Receptors for Treating B-cell Malignancies and Hodgkin Lymphoma

NCT04160195
View directory record Official record
Conditions
Lymphoma, B-Cell, Lymphoma, Non-hodgkins, Chronic Lymphocytic Leukemia, B-Cell Chronic Lymphocytic Leukemia
Interventions
Anti-cluster of differentiation 19 (CD19)-Chimeric Antigen Receptors (CAR) and Anti-cluster of differentiation 20 (CD20)-CAR T cells, Cyclophosphamide, Fludarabine
Biological · Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years and older
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Feb 15, 2022 · Synced Oct 19, 2026, 4:54 PM EDT
Completed Phase 2 Interventional Results available

ADVM-022 Intravitreal Gene Therapy for DME

NCT04418427
View directory record Official record
Conditions
Diabetic Macular Edema, Diabetic Retinopathy
Interventions
6E11 vg/eye of ADVM-022, 2E11 vg/eye of ADVM-022, Aflibercept
Biological
Lead sponsor
Adverum Biotechnologies, Inc.
Industry
Eligibility
18 Years and older
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2023
U.S. locations
13
States / cities
Phoenix, Arizona • Bakersfield, California • Beverly Hills, California + 10 more
Source: ClinicalTrials.gov public record
Updated Jul 10, 2025 · Synced Oct 19, 2026, 4:54 PM EDT
Recruiting Phase 1Phase 2 Interventional

Melpida: Recombinant Adeno-associated Virus (Serotype 9) Encoding a Codon Optimized Human AP4M1 Transgene (hAP4M1opt)

NCT05518188
View directory record Official record
Conditions
Spasticity, Muscle, Microcephaly, Intellectual Deficiency, Growth Retardation, SPG50, Spastic Paraplegia
Interventions
MELPIDA
Biological
Lead sponsor
Elpida Therapeutics SPC
Industry
Eligibility
4 Months to 10 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2030
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Jul 8, 2026 · Synced Oct 19, 2026, 4:54 PM EDT
Completed Phase 2 Interventional Results available

Enzalutamide With or Without Vaccine Therapy for Advanced Prostate Cancer

NCT01867333
View directory record Official record
Conditions
Prostate Cancer
Interventions
PROSTVAC (rilimogene galvacirepvec/rilimogene glafolivec) -F/TRICOM, PROSTVAC (rilimogene galvacirepvec/rilimogene glafolivec) -V/TRICOM, Enzalutamide
Biological
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years and older · Male only
Enrollment
57 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2022
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 26, 2023 · Synced Oct 19, 2026, 4:54 PM EDT
Recruiting Phase 1Phase 2 Interventional

A Phase 1/2, First-in-Human Dose Escalation/Expansion Study to Evaluate the Safety, Tolerability and Preliminary Efficacy of a Subretinal Injection of SB-007 in Subjects With Stargardt Disease (STGD1)

NCT06942572
View directory record Official record
Conditions
Stargardt Disease, Stargardt Macular Degeneration, Stargardt Macular Dystrophy
Interventions
SB-007
Genetic
Lead sponsor
Splice Bio
Industry
Eligibility
12 Years to 65 Years
Enrollment
57 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
5
States / cities
Aurora, Colorado • Miami, Florida • Boston, Massachusetts + 2 more
Source: ClinicalTrials.gov public record
Updated Apr 29, 2025 · Synced Oct 19, 2026, 4:54 PM EDT
Terminated Phase 1 Interventional

Gene Transfer Clinical Study in Crigler-Najjar Syndrome

NCT03223194
View directory record Official record
Conditions
Crigler-Najjar Syndrome
Interventions
AT342
Genetic
Lead sponsor
Audentes Therapeutics
Industry
Eligibility
1 Year and older
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2021
U.S. locations
2
States / cities
The Bronx, New York • Strasburg, Pennsylvania
Source: ClinicalTrials.gov public record
Updated May 18, 2022 · Synced Oct 19, 2026, 4:54 PM EDT
Not yet recruiting Phase 1Phase 2 Interventional

Safety and Efficacy of AAV9/AP4B1 (BFB-101) For Patients With AP4B1-related Hereditary Spastic Paraplegia Type 47 (SPG47)

NCT06948019
View directory record Official record
Conditions
HSP, Hereditary Spastic Paraplegia, Hereditary Spastic Paraparesis, Hereditary Spastic Paraplegia Type 50, Hereditary Spastic Paraplegia Type 47, Hereditary Spastic Paraplegia Type 51, Hereditary Spastic Paraplegia Type 52, SPG47, AP4B1, Neurogenetic Disorders, Neurodevelopmental Conditions, Movement Disorders, Gene Therapy
Interventions
BFB-101 (AAV9-CBh-AP4B1)
Biological
Lead sponsor
BlackfinBio Ltd
Industry
Eligibility
12 Months to 60 Months
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2032
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Apr 28, 2025 · Synced Oct 19, 2026, 4:54 PM EDT
Enrolling by invitation Phase 2 Interventional

Long-term Follow-Up Study of RGX-314 and Fellow Eye Substudy

NCT03999801
View directory record Official record
Conditions
Neovascular Age-related Macular Degeneration, Wet Macular Degeneration
Interventions
RGX-314
Genetic
Lead sponsor
AbbVie
Industry
Eligibility
50 Years and older
Enrollment
865 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2028
U.S. locations
9
States / cities
Phoenix, Arizona • Santa Barbara, California • Baltimore, Maryland + 5 more
Source: ClinicalTrials.gov public record
Updated Jun 17, 2024 · Synced Oct 19, 2026, 4:54 PM EDT
Enrolling by invitation Phase 1Phase 2 Interventional

rAAV-Olig001-ASPA Gene Therapy for Treatment of Children With Typical Canavan Disease

NCT04833907
View directory record Official record
Conditions
Canavan Disease
Interventions
rAAV-Olig001-ASPA, Levetiracetam, Prednisone
Drug
Lead sponsor
Myrtelle Inc.
Industry
Eligibility
3 Months to 60 Months
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2027
U.S. locations
1
States / cities
Dayton, Ohio
Source: ClinicalTrials.gov public record
Updated Nov 14, 2025 · Synced Oct 19, 2026, 4:54 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Long-Term Follow-Up of Recipient of Gene Transfer Research

NCT01492036
View directory record Official record
Conditions
Retroviridae Infections, Cancer
Interventions
Blood Tests, Questionnaires
Other · Behavioral
Lead sponsor
M.D. Anderson Cancer Center
Other
Eligibility
Not listed
Enrollment
130 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2011 – 2027
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jul 15, 2026 · Synced Oct 19, 2026, 4:54 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 2, 2017 · Synced Oct 19, 2026, 4:54 PM EDT
Completed No phase listed Observational

Identifying and Genotyping Homozygous Familial Hypercholesterolemia (HoFH) Patients

NCT04148001
View directory record Official record
Conditions
Homozygous Familial Hypercholesterolemia (HoFH)
Interventions
Not listed
Lead sponsor
REGENXBIO Inc.
Industry
Eligibility
18 Years and older
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2020
U.S. locations
1
States / cities
Boca Raton, Florida
Source: ClinicalTrials.gov public record
Updated Feb 2, 2021 · Synced Oct 19, 2026, 4:54 PM EDT
Recruiting No phase listed Observational

ATHN Transcends: A Natural History Study of Non-Neoplastic Hematologic Disorders

NCT04398628
View directory record Official record
Conditions
Hematologic Disorder, Bleeding Disorder, Connective Tissue Disorder, Hemophilia, Thrombosis, Von Willebrand Diseases, Thrombophilia, Rare Bleeding Disorder, Platelet Disorder, Factor IX Deficiency, Factor VIII Deficiency, Thalassemia, Sickle Cell Disease
Interventions
Not listed
Lead sponsor
American Thrombosis and Hemostasis Network
Network
Eligibility
Not listed
Enrollment
3,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2035
U.S. locations
76
States / cities
Phoenix, Arizona • Little Rock, Arkansas • Los Angeles, California + 60 more
Source: ClinicalTrials.gov public record
Updated Aug 17, 2026 · Synced Oct 19, 2026, 4:54 PM EDT
Recruiting Phase 3 Interventional

Trial of Nadofaragene Firadenovec vs. Observation in Participants With Intermediate Risk Non-Muscle Invasive Bladder Cancer

NCT06510374
View directory record Official record
Conditions
Intermediate Risk Non-Muscle Invasive Bladder Cancer
Interventions
Nadofaragene Firadenovec
Drug
Lead sponsor
Ferring Pharmaceuticals
Industry
Eligibility
18 Years and older
Enrollment
454 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2031
U.S. locations
54
States / cities
Mobile, Alabama • Phoenix, Arizona • Tucson, Arizona + 48 more
Source: ClinicalTrials.gov public record
Updated Oct 16, 2026 · Synced Oct 19, 2026, 4:54 PM 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 19, 2026, 4:54 PM EDT
Not listed No phase listed Observational

Patients Treated for SCID (1968-Present)

NCT01346150
View directory record Official record
Conditions
SCID, ADA-SCID, XSCID, Leaky SCID, Omenn Syndrome, Reticular Dysgenesis
Interventions
Not listed
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
Not listed
Enrollment
1,007 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2023
U.S. locations
31
States / cities
Birmingham, Alabama • Los Angeles, California • Palo Alto, California + 26 more
Source: ClinicalTrials.gov public record
Updated Nov 12, 2020 · Synced Oct 19, 2026, 4:54 PM 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 19, 2026, 4:54 PM EDT
Completed Phase 1Phase 2 Interventional

AAVRh.10 Administered to Children With Late Infantile Neuronal Ceroid Lipofuscinosis

NCT01414985
View directory record Official record
Conditions
Late Infantile Neuronal Ceroid Lipofuscinosis, Batten Disease
Interventions
AAVrh.10CUCLN2
Biological
Lead sponsor
Weill Medical College of Cornell University
Other
Eligibility
3 Years to 18 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2017
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Aug 31, 2020 · Synced Oct 19, 2026, 4:54 PM EDT