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ClinicalTrials.gov public records Last synced May 21, 2026, 11:10 PM EDT

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 57 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
Filters: Condition: Adeno-Associated Virus (AAV) Clear all
Active, not recruiting Phase 2 Interventional
View directory record Official record
Conditions
X-Linked Retinitis Pigmentosa
Interventions
rAAV2tYF-GRK1-RPGR
Biological
Lead sponsor
Beacon Therapeutics
Industry
Eligibility
8 Years to 50 Years · Male only
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2027
U.S. locations
6
States / cities
Jacksonville, Florida • Boston, Massachusetts • Cincinnati, Ohio + 3 more
Source: ClinicalTrials.gov public record
Updated Aug 11, 2024 · Synced May 21, 2026, 11:10 PM EDT
Completed Early Phase 1 Interventional
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Apr 9, 2020 · Synced May 21, 2026, 11:10 PM EDT
Completed Phase 1 Interventional Results available
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Dec 14, 2016 · Synced May 21, 2026, 11:10 PM EDT
Active, not recruiting Phase 3 Interventional
View directory record Official record
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
32 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
Source: ClinicalTrials.gov public record
Updated Feb 16, 2026 · Synced May 21, 2026, 11:10 PM EDT
Completed Phase 1 Interventional
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Nov 1, 2020 · Synced May 21, 2026, 11:10 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated May 17, 2026 · Synced May 21, 2026, 11:10 PM EDT
Completed Phase 1 Interventional
View directory record Official record
Conditions
Alzheimer's Disease
Interventions
CERE-110: Adeno-Associated Virus Delivery of NGF
Genetic
Lead sponsor
Sangamo Therapeutics
Industry
Eligibility
50 Years to 80 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2010
U.S. locations
2
States / cities
San Diego, California • Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Nov 9, 2022 · Synced May 21, 2026, 11:10 PM EDT
Completed Phase 1 Interventional
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Feb 4, 2013 · Synced May 21, 2026, 11:10 PM EDT
Completed Phase 1Phase 2 Interventional Results available
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jun 11, 2023 · Synced May 21, 2026, 11:10 PM EDT
Completed Phase 1 Interventional
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Feb 4, 2013 · Synced May 21, 2026, 11:10 PM EDT
Recruiting Phase 3 Interventional
View directory record Official record
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
284 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2031
U.S. locations
46
States / cities
Phoenix, Arizona • Scottsdale, Arizona • Springdale, Arkansas + 38 more
Source: ClinicalTrials.gov public record
Updated May 19, 2026 · Synced May 21, 2026, 11:10 PM EDT
Recruiting Phase 1 Interventional
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated May 6, 2026 · Synced May 21, 2026, 11:10 PM EDT
Recruiting Phase 1 Interventional
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Feb 5, 2025 · Synced May 21, 2026, 11:10 PM EDT
Active, not recruiting Phase 2Phase 3 Interventional
View directory record Official record
Conditions
X-Linked Retinitis Pigmentosa
Interventions
rAAV2tYF-GRK1-hRPGRco, Control
Biological · Drug
Lead sponsor
Beacon Therapeutics
Industry
Eligibility
12 Years to 50 Years · Male only
Enrollment
85 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2031
U.S. locations
19
States / cities
Scottsdale, Arizona • Los Angeles, California • Jacksonville, Florida + 15 more
Source: ClinicalTrials.gov public record
Updated Apr 6, 2026 · Synced May 21, 2026, 11:10 PM EDT
Completed No phase listed Observational Results available
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Mar 17, 2026 · Synced May 21, 2026, 11:10 PM EDT
Completed Phase 3 Interventional Results available
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Mar 26, 2026 · Synced May 21, 2026, 11:10 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Oct 20, 2025 · Synced May 21, 2026, 11:10 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jul 21, 2022 · Synced May 21, 2026, 11:10 PM EDT
Active, not recruiting No phase listed Observational
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Feb 9, 2026 · Synced May 21, 2026, 11:10 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated May 19, 2024 · Synced May 21, 2026, 11:10 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis
Interventions
AMT-162
Drug
Lead sponsor
UniQure Biopharma B.V.
Industry
Eligibility
18 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2031
U.S. locations
11
States / cities
Phoenix, Arizona • Irvine, California • San Francisco, California + 8 more
Source: ClinicalTrials.gov public record
Updated Oct 21, 2025 · Synced May 21, 2026, 11:10 PM EDT
Recruiting No phase listed Observational
View directory record Official record
Conditions
Arrhythmogenic Right Ventricular Cardiomyopathy
Interventions
Not listed
Lead sponsor
Tenaya Therapeutics
Industry
Eligibility
14 Years to 65 Years
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2030
U.S. locations
8
States / cities
San Francisco, California • Aurora, Colorado • Baltimore, Maryland + 5 more
Source: ClinicalTrials.gov public record
Updated Oct 31, 2024 · Synced May 21, 2026, 11:10 PM EDT
Completed Phase 1Phase 2 Interventional Results available
View directory record Official record
Conditions
Ornithine Transcarbamylase (OTC) Deficiency
Interventions
scAAV8OTC, Reactive Corticosteroid Taper Regimen, Prophylactic Corticosteroid Taper Regimen
Genetic · Drug
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
18 Years and older
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2021
U.S. locations
4
States / cities
Aurora, Colorado • Boston, Massachusetts • New York, New York + 1 more
Source: ClinicalTrials.gov public record
Updated Jan 25, 2023 · Synced May 21, 2026, 11:10 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Dec 11, 2024 · Synced May 21, 2026, 11:10 PM EDT