- 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
Search Results
Search by objective public record fields.
Search by query text, NCT ID, condition, intervention, sponsor, city, state, recruitment status, phase, study type, healthy volunteer eligibility, sex, or age. Results come from ClinicalTrials.gov. When the live registry is unavailable, a clearly labeled stored copy may be shown.
These results were retrieved from the live ClinicalTrials.gov registry. Filters and sort order apply to this result list.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Showing 1–24
of 59
matching trials from the live ClinicalTrials.gov search.
- 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
- 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
- Adeno-Associated Virus (AAV), Blood Coagulation Disorder, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, Factor VIII (FVIII), Factor VIII (FVIII) Deficiency, Factor VIII (FVIII) Gene, Factor VIII (FVIII) Protein, Genetic Diseases, Inborn, Genetic Diseases, X-Linked, Gene Therapy, Gene Transfer, Hematologic Diseases, Hemorrhagic Disorders, Recombinant, Vector, Inhibitors
- Interventions
- SPK-8016
- Genetic
- Lead sponsor
- Spark Therapeutics, Inc.
- Industry
- Eligibility
- 18 Years and older · Male only
- Enrollment
- 4 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2023
- U.S. locations
- 11
- States / cities
- Los Angeles, California • Peoria, Illinois • Ann Arbor, Michigan + 7 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
- AAV5-hFIXco-Padua, Factor IX (FIX)
- Genetic · Biological
- Lead sponsor
- CSL Behring
- Industry
- Eligibility
- 18 Years and older · Male only
- Enrollment
- 67 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2025
- U.S. locations
- 19
- States / cities
- Phoenix, Arizona • Little Rock, Arkansas • Los Angeles, California + 14 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
- Wet Age-related Macular Degeneration, Neovascular Age-related Macular Degeneration
- Interventions
- ADVM-022
- Biological
- Lead sponsor
- Adverum Biotechnologies, Inc.
- Industry
- Eligibility
- 50 Years and older
- Enrollment
- 23 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2030
- U.S. locations
- 9
- States / cities
- Bakersfield, California • Beverly Hills, California • Lakewood, Colorado + 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
- 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
- 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
- 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
- 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
- Hemophilia A
- Interventions
- BAY2599023 (DTX201)
- Drug
- Lead sponsor
- Bayer
- Industry
- Eligibility
- 18 Years and older · Male only
- Enrollment
- 11 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2026
- U.S. locations
- 3
- States / cities
- Little Rock, Arkansas • Ann Arbor, Michigan • Madison, Wisconsin
- 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
- X-Linked Retinitis Pigmentosa
- Interventions
- rAAV2tYF-GRK1-RPGR
- Biological
- Lead sponsor
- Beacon Therapeutics
- Industry
- Eligibility
- 6 Years to 50 Years · Male only
- Enrollment
- 29 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2025
- U.S. locations
- 4
- States / cities
- Durham, North Carolina • Cincinnati, Ohio • Portland, Oregon + 1 more
- 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
- GSD1
- Interventions
- DTX401, steroid regimen
- Genetic · Drug
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2021
- U.S. locations
- 3
- States / cities
- Farmington, Connecticut • Ann Arbor, Michigan • Houston, Texas
- 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
- 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
- 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
- Hemophilia A
- Interventions
- Blood sample collection
- Procedure
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 186 participants
- Timeline
- 2020 – 2023
- U.S. locations
- 1
- States / cities
- Madison, Wisconsin
- 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
- 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