- Conditions
- Congenital Adrenal Hyperplasia
- Interventions
- Pre-Screening
- Other
- Lead sponsor
- Adrenas Therapeutics Inc
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 65 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2024
- U.S. locations
- 1
- States / cities
- Los Angeles, California
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 1–24
of 59
matching trials from the live ClinicalTrials.gov search.
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- Conditions
- Parkinson's Disease
- Interventions
- Bilateral surgical infusion of AAV-GAD into the subthalamic nucleus
- Genetic
- Lead sponsor
- Neurologix, Inc.
- Industry
- Eligibility
- 30 Years to 75 Years
- Enrollment
- 44 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Started 2008
- U.S. locations
- 7
- States / cities
- Stanford, California • Aurora, Colorado • Boston, Massachusetts + 4 more
- Conditions
- Alpha 1-Antitrypsin Deficiency
- Interventions
- rAAV1-CB-hAAT
- Biological
- Lead sponsor
- University of Massachusetts, Worcester
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 9 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2006 – 2015
- U.S. locations
- 2
- States / cities
- Gainesville, Florida • Worcester, Massachusetts
- Conditions
- Hemophilia B
- Interventions
- AAV8-hFIX19
- Biological
- Lead sponsor
- Spark Therapeutics, Inc.
- Industry
- Eligibility
- 18 Years and older · Male only
- Enrollment
- 4 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2016
- U.S. locations
- 2
- States / cities
- Philadelphia, Pennsylvania • Pittsburgh, Pennsylvania
- Conditions
- X-Linked Retinitis Pigmentosa
- Interventions
- AGTC-501 (high dose and standard corticosteroid regimen), AGTC-501 (low dose and standard corticosteroid regimen), AGTC-501 (high dose and modified corticosteroid regimen)
- Biological
- Lead sponsor
- Beacon Therapeutics
- Industry
- Eligibility
- 12 Years and older · Male only
- Enrollment
- 24 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2029
- U.S. locations
- 7
- States / cities
- Jacksonville, Florida • Miami, Florida • Boston, Massachusetts + 4 more
- 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
- X-Linked Retinitis Pigmentosa (XLRP)
- Interventions
- Adeno-associated virus vector expressing a human RPGR gene
- Biological
- Lead sponsor
- Beacon Therapeutics
- Industry
- Eligibility
- 12 Years to 50 Years · Male only
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2030
- U.S. locations
- 6
- States / cities
- Jacksonville, Florida • Miami, Florida • Durham, North Carolina + 3 more
- Conditions
- Ornithine Transcarbamylase Deficiency, Wilson Disease, Glycogen Storage Disease Type IA
- Interventions
- Not listed
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 51 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2022
- U.S. locations
- 1
- States / cities
- Akron, Ohio
- 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
- X-linked Retinoschisis
- Interventions
- rAAV2tYF-CB-hRS1
- Biological
- Lead sponsor
- Beacon Therapeutics
- Industry
- Eligibility
- 6 Years and older · Male only
- Enrollment
- 27 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2023
- U.S. locations
- 9
- States / cities
- San Francisco, California • Miami, Florida • Baltimore, Maryland + 6 more
- Conditions
- Muscular Dystrophies
- Interventions
- rAAV1.tMCK.human-alpha-sarcoglycan- First cohort, Genetic: rAAV1.tMCK.human-alpha-sarcoglycan- Second cohort
- Genetic
- Lead sponsor
- Nationwide Children's Hospital
- Other
- Eligibility
- 5 Years and older
- Enrollment
- 6 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2011
- U.S. locations
- 1
- States / cities
- Columbus, Ohio
- Conditions
- Pompe Disease (Late-onset), Pompe Disease Late-Onset, LOPD
- Interventions
- AB-1009 (GAA Gene)
- Genetic
- Lead sponsor
- AskBio Inc
- Industry
- Eligibility
- 18 Years to 65 Years
- 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
- Duchenne Muscular Dystrophy
- Interventions
- SGT-003, Placebo
- Drug
- Lead sponsor
- Solid Biosciences Inc.
- Industry
- Eligibility
- 7 Years to 11 Years · Male only
- Enrollment
- 80 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2034
- U.S. locations
- 3
- States / cities
- Little Rock, Arkansas • Flower Mound, Texas • Norfolk, Virginia
- 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
- 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
- Hemophilia A
- Interventions
- SPK-8011
- Genetic
- Lead sponsor
- Spark Therapeutics, Inc.
- Industry
- Eligibility
- 18 Years and older · Male only
- Enrollment
- 25 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2023
- U.S. locations
- 11
- States / cities
- Sacramento, California • Gainesville, Florida • Boston, Massachusetts + 7 more
- Conditions
- Glycogen Storage Disease Type IA
- Interventions
- DTX401, Placebo, Oral prednisolone, Placebo for oral prednisolone
- Genetic · Other · Drug
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Industry
- Eligibility
- 8 Years and older
- Enrollment
- 49 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2026
- U.S. locations
- 9
- States / cities
- Orange, California • Aurora, Colorado • Farmington, Connecticut + 6 more
- Conditions
- Parkinson's Disease
- Interventions
- AAV-hAADC-2
- Genetic
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- 40 Years to 75 Years
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2004 – 2013
- U.S. locations
- 1
- States / cities
- San Francisco, California
- 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
- 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
- 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
- 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
- Arrhythmogenic Right Ventricular Cardiomyopathy
- Interventions
- TN-401
- Genetic
- Lead sponsor
- Tenaya Therapeutics
- Industry
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 15 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2029
- U.S. locations
- 7
- States / cities
- San Francisco, California • Aurora, Colorado • Baltimore, Maryland + 4 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