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)

Description

The study will evaluate the safety and efficacy of delandistrogene moxeparvovec gene transfer therapy in non-ambulatory and ambulatory males with DMD. This is a randomized, double-blind, placebo-controlled 2-part study. Participants will be in the study for approximately 128 weeks. All participants will have the opportunity to receive intravenous (IV) delandistrogene moxeparvovec in either Part 1 or Part 2.

Conditions

Duchenne Muscular Dystrophy

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Study Overview

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Study Details

Study overview

The study will evaluate the safety and efficacy of delandistrogene moxeparvovec gene transfer therapy in non-ambulatory and ambulatory males with DMD. This is a randomized, double-blind, placebo-controlled 2-part study. Participants will be in the study for approximately 128 weeks. All participants will have the opportunity to receive intravenous (IV) delandistrogene moxeparvovec in either Part 1 or Part 2.

A Phase 3, Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of SRP- 9001 in Non-Ambulatory and Ambulatory Subjects With Duchenne Muscular Dystrophy (ENVISION)

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)

Condition
Duchenne Muscular Dystrophy
Intervention / Treatment

-

Contacts and Locations

Little Rock

Arkansas Children's Hospital, Little Rock, Arkansas, United States, 72202

Palo Alto

Lucile Packard Children's Hospital Stanford, Palo Alto, California, United States, 94304

Sacramento

University of California at Davis Medical Center, Sacramento, California, United States, 95817

San Diego

Rady Children's Hospital-San Diego, San Diego, California, United States, 92123

Gainesville

University of Florida, UF Health Center for Pediatric Neuromuscular and Rare Diseases, Gainesville, Florida, United States, 32608

Chicago

Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, United States, 60611

Iowa City

University of Iowa Hospitals and Clinics, Dept of Pediatrics, Iowa City, Iowa, United States, 52242

Baltimore

The Johns Hopkins Hospital, Charlotte R. Bloomberg Children's Center, Pediatric Clinical Research Unit, Baltimore, Maryland, United States, 21287

Boston

Boston Children's Hospital, Boston, Massachusetts, United States, 02115

Saint Louis

Washington University of St. Louis, St. Louis Children's Hospital, Saint Louis, Missouri, United States, 63110

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility Criteria

  • * Definitive diagnosis of DMD based on documented clinical findings and prior genetic testing.
  • * Cohort 1 only: Non-ambulatory per protocol specified criteria.
  • * Cohort 2 only: Ambulatory per protocol specified criteria and ≥8 to \<18 years of age at the time of Screening.
  • * Ability to cooperate with motor assessment testing.
  • * Stable daily dose of oral corticosteroids for at least 12 weeks prior to Screening, and the dose is expected to remain constant throughout the study (except for modifications to accommodate changes in weight).
  • * Recombinant Adeno-Associated Virus Serotype rh74 (rAAVrh74) antibody titers are not elevated as per protocol-specified requirements.
  • * A pathogenic frameshift mutation or premature stop codon contained between exons 18 and 79 (inclusive).
  • * Exposure to gene therapy, investigational medication, or any treatment designed to increase dystrophin expression within protocol specified time limits.
  • * Abnormality in protocol-specified diagnostic evaluations or laboratory tests.
  • * Presence of any other clinically significant illness, medical condition, or requirement for chronic drug treatment that in the opinion of the Investigator creates unnecessary risk for gene transfer.

Ages Eligible for Study

to

Sexes Eligible for Study

MALE

Accepts Healthy Volunteers

No

Collaborators and Investigators

Sarepta Therapeutics, Inc.,

Medical Director, STUDY_DIRECTOR, Sarepta Therapeutics, Inc.

Study Record Dates

2028-06-30