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ClinicalTrials.gov public records Last synced Oct 7, 2026, 5:43 PM EDT

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Showing 25–43 of 43 matching trials from the live ClinicalTrials.gov search.
Filters: Sponsor: Sarepta Therapeutics, Inc. Clear all
Active, not recruiting Phase 3 Interventional

A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Non-Ambulatory and Ambulatory Participants With Duchenne Muscular Dystrophy (DMD)

NCT05881408
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
delandistrogene moxeparvovec, placebo
Genetic
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
Male only
Enrollment
148 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2028
U.S. locations
14
States / cities
Little Rock, Arkansas • Palo Alto, California • Sacramento, California + 11 more
Source: ClinicalTrials.gov public record
Updated May 22, 2026 · Synced Oct 7, 2026, 5:43 PM EDT
Terminated Phase 2 Interventional

Two-Part Study for Dose Determination of Vesleteplirsen (SRP-5051) (Part A), Then Dose Expansion (Part B) in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment

NCT04004065
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Vesleteplirsen
Drug
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
7 Years to 21 Years · Male only
Enrollment
62 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2025
U.S. locations
11
States / cities
Sacramento, California • Farmington, Connecticut • Gulf Breeze, Florida + 8 more
Source: ClinicalTrials.gov public record
Updated Mar 10, 2025 · Synced Oct 7, 2026, 5:43 PM EDT
Terminated Phase 1Phase 2 Interventional Results available

An Open-Label Extension Study for Patients With Duchenne Muscular Dystrophy Who Participated in Studies of SRP-5051 (Vesleteplirsen)

NCT03675126
View directory record Official record
Conditions
Muscular Dystrophy, Duchenne
Interventions
SRP-5051
Drug
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
4 Years and older · Male only
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
7
States / cities
Hartford, Connecticut • Gulf Breeze, Florida • Atlanta, Georgia + 4 more
Source: ClinicalTrials.gov public record
Updated Sep 19, 2024 · Synced Oct 7, 2026, 5:43 PM EDT
Terminated Phase 4 Interventional

An Open-Label Study of Golodirsen in Non-Ambulant Patients With Duchenne Muscular Dystrophy

NCT04708314
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Golodirsen 50 MG/1 ML Intravenous Solution [VYONDYS 53]
Drug
Lead sponsor
Rare Disease Research, LLC
Other
Eligibility
7 Years and older · Male only
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2021
U.S. locations
2
States / cities
Atlanta, Georgia • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 3, 2026 · Synced Oct 7, 2026, 5:43 PM EDT
Recruiting Phase 1 Interventional

A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD) - Non-Ambulatory Cohort

NCT04626674
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
delandistrogene moxeparvovec
Genetic
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
2 Years and older · Male only
Enrollment
83 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2028
U.S. locations
12
States / cities
Little Rock, Arkansas • La Jolla, California • Los Angeles, California + 9 more
Source: ClinicalTrials.gov public record
Updated Sep 11, 2026 · Synced Oct 7, 2026, 5:43 PM EDT
Withdrawn Phase 3 Interventional

A Study of SRP-9005 in Limb Girdle Muscular Dystrophy Type 2C/R5 Pediatric and Adult Participants

NCT06952686
View directory record Official record
Conditions
Muscular Dystrophies, Limb-Girdle
Interventions
SRP-9005, Corticosteroid
Biological · Drug
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
4 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2032
U.S. locations
1
States / cities
Norfolk, Virginia
Source: ClinicalTrials.gov public record
Updated Aug 6, 2025 · Synced Oct 7, 2026, 5:43 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A Study to Assess the Safety, Tolerability and Pharmacokinetics of AVI-7288 in Healthy Adult Volunteers

NCT01566877
View directory record Official record
Conditions
Marburg Hemorrhagic Fever
Interventions
AVI-7288, Placebo
Drug · Other
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
18 Years to 50 Years
Enrollment
40 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2014
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated May 7, 2014 · Synced Oct 7, 2026, 5:43 PM EDT
Completed Phase 1 Interventional

rAAVrh74.MHCK7.DYSF.DV for Treatment of Dysferlinopathies

NCT02710500
View directory record Official record
Conditions
Dysferlinopathy
Interventions
rAAVrh74.MHCK7.DYSF.DV
Drug
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
18 Years and older
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated May 13, 2021 · Synced Oct 7, 2026, 5:43 PM EDT
Active, not recruiting Phase 3 Interventional

A Study to Compare Safety and Efficacy of High Doses of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) (MIS51ON)

NCT03992430
View directory record Official record
Conditions
Muscular Dystrophy, Duchenne
Interventions
Eteplirsen
Drug
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
4 Years to 13 Years · Male only
Enrollment
160 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2026
U.S. locations
3
States / cities
Birmingham, Alabama • Gainesville, Florida • Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Feb 27, 2026 · Synced Oct 7, 2026, 5:43 PM EDT
Terminated Phase 1 Interventional

A Gene Transfer Therapy to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) Following Therapeutic Plasma Exchange (Plasmapheresis) in Participants With Duchenne Muscular Dystrophy (DMD) and Pre-existing Antibodies to AAVrh74

NCT06597656
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
delandistrogene moxeparvovec, Plasmapheresis
Genetic · Procedure
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
4 Years to 8 Years · Male only
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2025
U.S. locations
3
States / cities
Gainesville, Florida • St Louis, Missouri • Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Sep 4, 2025 · Synced Oct 7, 2026, 5:43 PM EDT
Recruiting Phase 4 Interventional

Study to Evaluate the Safety and Effectiveness of ELEVIDYS in Participants With Duchenne Muscular Dystrophy Treated in a Post-Marketing Setting

NCT07542314
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
ELEVIDYS, Sirolimus, Glucocorticoids, Antibiotics
Drug
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
4 Years and older · Male only
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2027
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Oct 6, 2026 · Synced Oct 7, 2026, 5:43 PM EDT
Completed Phase 1Phase 2 Interventional Results available

A Gene Transfer Therapy Study to Evaluate the Safety of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)

NCT03375164
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
delandistrogene moxeparvovec
Genetic
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
3 Months to 7 Years · Male only
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2023
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Nov 14, 2024 · Synced Oct 7, 2026, 5:43 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

Safety Study of Single Administration Post-Exposure Prophylaxis Treatment for Ebola Virus

NCT01353027
View directory record Official record
Conditions
Ebola Hemorrhagic Fever
Interventions
Placebo, AVI-6002
Drug
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
18 Years to 50 Years
Enrollment
30 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2010 – 2011
U.S. locations
1
States / cities
Knoxville, Tennessee
Source: ClinicalTrials.gov public record
Updated Mar 28, 2012 · Synced Oct 7, 2026, 5:43 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

Pharmacokinetic Study in Cerebral Spinal Fluid After a Single Dose of AVI-4020

NCT00387283
View directory record Official record
Conditions
West Nile Virus
Interventions
AVI-4020 Injection
Drug
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
18 Years to 64 Years · Male only
Enrollment
14 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2006 – 2009
U.S. locations
1
States / cities
Tacoma, Washington
Source: ClinicalTrials.gov public record
Updated Jul 8, 2009 · Synced Oct 7, 2026, 5:43 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Your Voice; Impact of Duchenne Muscular Dystrophy (DMD) on the Lives of Families

NCT03680365
View directory record Official record
Conditions
Duchenne Muscular Dystrophy, Burden, Dependency, Disability Physical, Disease Management, Impairment, Rare Diseases
Interventions
Not listed
Lead sponsor
Jett Foundation, Inc.
Other
Eligibility
11 Years and older
Enrollment
60 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2019
U.S. locations
1
States / cities
Eagan, Minnesota
Source: ClinicalTrials.gov public record
Updated Apr 1, 2019 · Synced Oct 7, 2026, 5:43 PM EDT
Enrolling by invitation Phase 3 Interventional

A Long-term Follow-up Study of Participants Who Received Delandistrogene Moxeparvovec (SRP-9001) in a Previous Clinical Study

NCT05967351
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
delandistrogene moxeparvovec
Genetic
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
Male only
Enrollment
400 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2033
U.S. locations
22
States / cities
Little Rock, Arkansas • Los Angeles, California • Palo Alto, California + 19 more
Source: ClinicalTrials.gov public record
Updated Feb 6, 2026 · Synced Oct 7, 2026, 5:43 PM EDT
Terminated Phase 1Phase 2 Interventional

Gene Delivery Clinical Trial of SRP-9003 (Bidridistrogene Xeboparvovec) for Participants With Limb-Girdle Muscular Dystrophy, Type 2E (LGMD2E) (Beta-Sarcoglycan Deficiency)

NCT03652259
View directory record Official record
Conditions
Limb-Girdle Muscular Dystrophy, Type 2E
Interventions
SRP-9003
Genetic
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
4 Years to 15 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2025
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Jan 20, 2026 · Synced Oct 7, 2026, 5:43 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

Pharmacokinetic Study of a Single Dose of AVI-4126 (RESTEN-NG®) in Cerebral Spinal Fluid

NCT00343148
View directory record Official record
Conditions
Neoplasms
Interventions
AVI-4126 Injection (RESTEN-NG®)
Drug
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
18 Years to 64 Years · Male only
Enrollment
6 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2006 – 2009
U.S. locations
1
States / cities
Tacoma, Washington
Source: ClinicalTrials.gov public record
Updated Jul 8, 2009 · Synced Oct 7, 2026, 5:43 PM EDT
Completed Phase 1Phase 2 Interventional Results available

A Randomized, Double-blind, Placebo-controlled Study of Delandistrogene Moxeparvovec (SRP-9001) for Duchenne Muscular Dystrophy (DMD)

NCT03769116
View directory record Official record
Conditions
Muscular Dystrophy, Duchenne
Interventions
delandistrogene moxeparvovec, placebo
Genetic
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
4 Years to 7 Years · Male only
Enrollment
41 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2023
U.S. locations
2
States / cities
Los Angeles, California • Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Nov 14, 2024 · Synced Oct 7, 2026, 5:43 PM EDT