- Conditions
- Achromatopsia
- Interventions
- rAAV2tYF-PR1.7-hCNGB3
- Biological
- Lead sponsor
- Beacon Therapeutics
- Industry
- Eligibility
- 4 Years and older
- Enrollment
- 32 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2026
- U.S. locations
- 8
- States / cities
- Gainesville, Florida • Miami, Florida • Chicago, Illinois + 4 more
Search Results
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Search by query text, NCT ID, condition, intervention, sponsor, city, state, recruitment status, phase, study type, healthy volunteer eligibility, sex, or age. Results are retrieved from ClinicalTrials.gov and synchronized into the directory. Search pages remain noindex by default.
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Showing 25–48
of 24
matching trials from the live ClinicalTrials.gov search.
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- Conditions
- Dilated Cardiomyopathy (DCM)
- Interventions
- RP-A701 is a recombinant viral vector composed of an AAV serotype rh.74 (AAVrh.74) capsid encapsulating the transgene, BCL2-associated Athanogene 3 (BAG3)
- Genetic
- Lead sponsor
- Rocket Pharmaceuticals Inc.
- Industry
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 8 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2029
- U.S. locations
- 3
- States / cities
- San Diego, California • Rochester, Minnesota • Charleston, South Carolina
- Conditions
- Hemophilia B
- Interventions
- Not listed
- Lead sponsor
- Spark Therapeutics, Inc.
- Industry
- Eligibility
- 18 Years and older · Male only
- Enrollment
- 4 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2007 – 2017
- U.S. locations
- 2
- States / cities
- Philadelphia, Pennsylvania • Pittsburgh, Pennsylvania
- Conditions
- Hepatitis C
- Interventions
- Not listed
- Lead sponsor
- Tacere Therapeutics, Inc.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 9 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2018
- U.S. locations
- 1
- States / cities
- Durham, North Carolina
- Conditions
- Canavan Disease
- Interventions
- AAV9 BBP-812
- Biological
- Lead sponsor
- Aspa Therapeutics
- Industry
- Eligibility
- Up to 30 Months
- Enrollment
- 26 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2032
- U.S. locations
- 4
- States / cities
- Oakland, California • Chicago, Illinois • Boston, Massachusetts + 1 more
- Conditions
- Congenital Adrenal Hyperplasia
- Interventions
- AAV BBP-631
- Biological
- Lead sponsor
- Adrenas Therapeutics Inc
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 8 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2029
- U.S. locations
- 5
- States / cities
- Chicago, Illinois • Bethesda, Maryland • Minneapolis, Minnesota + 2 more
- Conditions
- Phenylketonuria (PKU)
- Interventions
- BMN 307
- Drug
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- 15 Years and older
- Enrollment
- 100 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2027
- U.S. locations
- 2
- States / cities
- Tampa, Florida • Morristown, New Jersey
- Conditions
- Ornithine Transcarbamylase (OTC) Deficiency
- Interventions
- No Intervention
- Other
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 11 participants
- Timeline
- 2018 – 2029
- U.S. locations
- 4
- States / cities
- Aurora, Colorado • Boston, Massachusetts • New York, New York + 1 more
- Conditions
- Hemophilia B
- Interventions
- DTX101
- Genetic
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Industry
- Eligibility
- 18 Years and older · Male only
- Enrollment
- 6 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2017
- U.S. locations
- 6
- States / cities
- Little Rock, Arkansas • Los Angeles, California • Gainesville, Florida + 3 more
- Conditions
- Duchenne Muscular Dystrophy
- Interventions
- rAAV2.5-CMV-minidystrophin (d3990)
- Biological
- Lead sponsor
- Nationwide Children's Hospital
- Other
- Eligibility
- 5 Years to 15 Years · Male only
- Enrollment
- 6 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2006 – 2010
- U.S. locations
- 1
- States / cities
- Columbus, Ohio
- Conditions
- Parkinson's Disease
- Interventions
- Surgical infusion of AAV-GAD into the subthalamic nucleus
- Genetic
- Lead sponsor
- Neurologix, Inc.
- Industry
- Eligibility
- 25 Years to 75 Years
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2003 – 2005
- U.S. locations
- 1
- States / cities
- Manhasset, New York
- Conditions
- Achromatopsia
- Interventions
- AGTC-402
- Biological
- Lead sponsor
- Beacon Therapeutics
- Industry
- Eligibility
- 4 Years and older
- Enrollment
- 24 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2026
- U.S. locations
- 5
- States / cities
- Gainesville, Florida • Miami, Florida • Boston, Massachusetts + 2 more
- Conditions
- Alpha 1-Antitrypsin Deficiency
- Interventions
- rAAV2-CB-hAAT Gene Vector
- Genetic
- Lead sponsor
- University of Massachusetts, Worcester
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2004 – 2020
- U.S. locations
- 2
- States / cities
- Gainesville, Florida • Worcester, Massachusetts
- 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
- Hemophilia B
- Interventions
- Not listed
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Industry
- Eligibility
- 18 Years and older · Male only
- Enrollment
- 6 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2021
- U.S. locations
- 3
- States / cities
- Little Rock, Arkansas • Gainesville, Florida • Ann Arbor, Michigan
- Conditions
- Hepatitis C
- Interventions
- TT-034
- Drug
- Lead sponsor
- Tacere Therapeutics, Inc.
- Industry
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 9 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2016
- U.S. locations
- 4
- States / cities
- San Diego, California • Durham, North Carolina • Dallas, Texas + 1 more
- Conditions
- Leber Congenital Amaurosis
- Interventions
- voretigene neparvovec-rzyl
- Biological
- Lead sponsor
- Spark Therapeutics, Inc.
- Industry
- Eligibility
- 8 Years and older
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2007 – 2018
- U.S. locations
- 1
- States / cities
- Philadelphia, Pennsylvania
- Conditions
- Hypertrophic Cardiomyopathy
- Interventions
- TN-201
- Genetic
- Lead sponsor
- Tenaya Therapeutics
- Industry
- Eligibility
- 18 Years to 75 Years
- Enrollment
- 30 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2032
- U.S. locations
- 10
- States / cities
- La Jolla, California • San Francisco, California • Atlanta, Georgia + 7 more
- Conditions
- Pompe Disease (Late-onset), Pompe Disease Late-Onset, LOPD
- Interventions
- AB-1009 (GAA Gene)
- Genetic
- Lead sponsor
- AskBio Inc
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2032
- U.S. locations
- 9
- States / cities
- Phoenix, Arizona • Irvine, California • Palo Alto, California + 6 more
- Conditions
- Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (OTOF)
- Interventions
- DB-OTO
- Genetic
- Lead sponsor
- Regeneron Pharmaceuticals
- Industry
- Eligibility
- Up to 17 Years
- Enrollment
- 30 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2032
- U.S. locations
- 9
- States / cities
- Los Angeles, California • San Diego, California • Jacksonville, Florida + 6 more
- Conditions
- Neovascular Age-Related Macular Degeneration (nAMD), Wet AMD
- Interventions
- Ixo-vec, Aflibercept
- Genetic · Drug
- Lead sponsor
- Adverum Biotechnologies, Inc.
- Industry
- Eligibility
- 50 Years and older
- Enrollment
- 311 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2030
- U.S. locations
- 75
- States / cities
- Gilbert, Arizona • Phoenix, Arizona • Scottsdale, Arizona + 67 more
- Conditions
- Huntington's Disease
- Interventions
- intra-striatal rAAV5-miHTT, Imitation (sham) surgery
- Genetic · Other
- Lead sponsor
- UniQure Biopharma B.V.
- Industry
- Eligibility
- 25 Years to 65 Years
- Enrollment
- 43 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2029
- U.S. locations
- 12
- States / cities
- Birmingham, Alabama • Tucson, Arizona • San Francisco, California + 9 more
- Conditions
- OTC Deficiency
- Interventions
- DTX301, Placebo, Oral Corticosteroids, Placebo for oral corticosteroids, Sodium Acetate
- Genetic · Other · Drug
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Industry
- Eligibility
- 12 Years and older
- Enrollment
- 37 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2031
- U.S. locations
- 4
- States / cities
- Los Angeles, California • Aurora, Colorado • Chicago, Illinois + 1 more
- Conditions
- Glycogen Storage Disease Type IA, Von Gierke's Disease (GSD Type Ia)
- Interventions
- No intervention
- Other
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2025
- U.S. locations
- 3
- States / cities
- Farmington, Connecticut • Ann Arbor, Michigan • Houston, Texas