Independent directory Public ClinicalTrials.gov records United States
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ClinicalTrials.gov public records Last synced Sep 7, 2026, 11:40 AM EDT

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Showing 1–24 of 720 matching trials from the live ClinicalTrials.gov search.
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Conditions
LCA5
Interventions
AAV8.hLCA5
Biological
Lead sponsor
Opus Genetics, Inc
Industry
Eligibility
4 Years and older
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2028
U.S. locations
2
States / cities
Philadelphia, Pennsylvania • Dallas, Texas
Conditions
Cutaneous Squamous Cell Carcinoma, Basal Cell Carcinoma
Interventions
IFx-Hu2.0
Biological
Lead sponsor
TuHURA Biosciences, Inc.
Industry
Eligibility
18 Years and older
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2022
U.S. locations
1
States / cities
Brandon, Florida
Conditions
SHANK3 Haploinsufficiency, Phelan-McDermid Syndrome
Interventions
JAG201
Genetic
Lead sponsor
Jaguar Gene Therapy, LLC
Industry
Eligibility
2 Years to 9 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2031
U.S. locations
3
States / cities
Chicago, Illinois • Boston, Massachusetts • New York, New York
Conditions
Gastric Cancer, Breast Cancer, Cervical Cancer, Lung Cancer
Interventions
KK-LC-1 TCR-T cells, Aldesleukin
Biological · Drug
Lead sponsor
Christian Hinrichs
Other
Eligibility
18 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2030
U.S. locations
2
States / cities
New Brunswick, New Jersey
Conditions
AMD, nAMD, Wet Age-related Macular Degeneration, wAMD, Wet AMD, CNV, Neovascular AMD, Neovascular Age-related Macular Degeneration, Choroidal Neovascularization
Interventions
ABBV-RGX-314, Ranibizumab (LUCENTIS®)
Genetic · Biological
Lead sponsor
AbbVie
Industry
Eligibility
50 Years to 89 Years
Enrollment
671 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2027
U.S. locations
89
States / cities
Phoenix, Arizona • Sun City, Arizona • Little Rock, Arkansas + 77 more
Conditions
Hemophilia B
Interventions
CSL222 (AAV5-hFIXco-Padua)
Genetic
Lead sponsor
CSL Behring
Industry
Eligibility
18 Years and older
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2032
U.S. locations
4
States / cities
San Diego, California • Ann Arbor, Michigan • Pittsburgh, Pennsylvania + 1 more
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
Conditions
Mesial Temporal Lobe Epilepsy
Interventions
AAV9-hSyn1-miGRIK2
Genetic
Lead sponsor
UniQure Biopharma B.V.
Industry
Eligibility
18 Years to 75 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2031
U.S. locations
18
States / cities
Birmingham, Alabama • Phoenix, Arizona • Palo Alto, California + 15 more
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
Conditions
Advanced Cancers, Leukemia
Interventions
Leukapheresis, Fludarabine, Cyclophosphamide, T-cell Infusion
Procedure · Drug
Lead sponsor
M.D. Anderson Cancer Center
Other
Eligibility
18 Years to 80 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2017
U.S. locations
1
States / cities
Houston, Texas
Conditions
Friedreich's Ataxia (FA)
Interventions
SGT-212
Drug
Lead sponsor
Solid Biosciences Inc.
Industry
Eligibility
18 Years to 40 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2032
U.S. locations
3
States / cities
Los Angeles, California • Columbus, Ohio • Philadelphia, Pennsylvania
Conditions
Macular Neovascularization Secondary to Age-Related Macular Degeneration
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 – 2028
U.S. locations
91
States / cities
Phoenix, Arizona • Scottsdale, Arizona • Springdale, Arkansas + 79 more
Conditions
Pleural Mesothelioma, Metastatic Pleural Effusions
Interventions
Adenoviral-mediated Interferon-beta, SCH 721015
Biological
Lead sponsor
University of Pennsylvania
Other
Eligibility
18 Years and older
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2009
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Conditions
Leber Congenital Amaurosis (LCA), Eye Diseases, Eye Diseases, Hereditary, Retinal Diseases
Interventions
AAV OPTIRPE65
Biological
Lead sponsor
MeiraGTx UK II Ltd
Industry
Eligibility
3 Years to 100 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2023
U.S. locations
1
States / cities
Ann Arbor, Michigan
Completed Phase 1Phase 2 Interventional Results available

Gene Therapy for Achromatopsia (CNGB3)

NCT03001310
Conditions
Achromatopsia
Interventions
AAV - CNGB3
Biological
Lead sponsor
MeiraGTx UK II Ltd
Industry
Eligibility
3 Years and older
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2019
U.S. locations
1
States / cities
Ann Arbor, Michigan
Conditions
Healthy Volunteers
Interventions
Fast release BRB confection, Intermed release BRB confection, Prolong release BRB confection
Other
Lead sponsor
Ohio State University Comprehensive Cancer Center
Other
Eligibility
18 Years to 65 Years
Enrollment
67 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2014
U.S. locations
1
States / cities
Columbus, Ohio
Conditions
Cystic Fibrosis
Interventions
SP-101 and doxorubicin Cohort 1, SP-101 and doxorubicin Cohort 2
Combination Product
Lead sponsor
Spirovant Sciences, Inc.
Industry
Eligibility
18 Years to 65 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
4
States / cities
Kansas City, Kansas • Boston, Massachusetts • New York, New York + 1 more
Conditions
Neoplasms
Interventions
Telephonic interview
Other
Lead sponsor
GlaxoSmithKline
Industry
Eligibility
18 Years and older
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2018
U.S. locations
1
States / cities
Collegeville, Pennsylvania
Conditions
Cellular Immunotherapy, Adoptive, Genetic Therapy, Tissue Donors, Hematopoietic Stem Cell Transplantation
Interventions
Not listed
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
4 Years to 120 Years
Enrollment
2,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2017 – 2050
U.S. locations
1
States / cities
Bethesda, Maryland
Conditions
Sarcoma
Interventions
Autologous HER2-specific T cells, Fludarabine, Cyclophosphamide, Autologous CAR Positive T cells
Genetic · Drug
Lead sponsor
Baylor College of Medicine
Other
Eligibility
Not listed
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2032
U.S. locations
2
States / cities
Houston, Texas
Conditions
Choroideremia
Interventions
BIIB111
Genetic
Lead sponsor
Biogen
Industry
Eligibility
18 Years and older · Male only
Enrollment
169 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2020
U.S. locations
8
States / cities
Los Angeles, California • Miami, Florida • Baltimore, Maryland + 5 more
Conditions
Choroideremia, CHM (Choroideremia) Gene Mutations
Interventions
AAV2-hCHM
Biological
Lead sponsor
Spark Therapeutics, Inc.
Industry
Eligibility
18 Years and older · Male only
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2022
U.S. locations
3
States / cities
Boston, Massachusetts • Philadelphia, Pennsylvania
Conditions
Angina Pectoris
Interventions
pVGI.1(VEGF2)
Genetic
Lead sponsor
Corautus Genetics
Industry
Eligibility
18 Years and older
Enrollment
404 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2004
U.S. locations
35
States / cities
Birmingham, Alabama • Phoenix, Arizona • La Jolla, California + 27 more
Conditions
Brain Neoplasm, Neoplasm Metastasis
Interventions
Cytovene (Ganciclovir Sodium), G1TKSVNa.53 Producer Cell Line
Drug · Device
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
19 Years and older
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1992 – 2010
U.S. locations
1
States / cities
Bethesda, Maryland