ACTIVE_NOT_RECRUITING

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)

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Conditions

Duchenne Muscular DystrophyDMDGene-DeliveryPediatricNorth Star Ambulatory Assessment (NSAA)Performance of Upper Limb (PUL)Duchenne

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

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.

Official Title

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)

Quick Facts

Study Start:2023-05-31
Study Completion:2028-06-30
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:ACTIVE_NOT_RECRUITING

Study ID

NCT05881408

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.

Ages Eligible for Study:Not specified
Sexes Eligible for Study:MALE
Accepts Healthy Volunteers:No
Standard Ages:CHILD, ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. * Definitive diagnosis of DMD based on documented clinical findings and prior genetic testing.
  2. * Cohort 1 only: Non-ambulatory per protocol-specified criteria.
  3. * Cohort 2 only: Ambulatory per protocol-specified criteria and ≥8 to \<18 years of age at the time of Screening.
  4. * Ability to cooperate with motor assessment testing.
  5. * 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).
  6. * Recombinant Adeno-Associated Virus Serotype rh74 (rAAVrh74) antibody titers are not elevated as per protocol-specified requirements.
  7. * A pathogenic frameshift mutation or premature stop codon in the DMD gene, except for any deletion mutations in exon 8 and/or 9.
  1. * Exposure to gene therapy, investigational medication, or any treatment designed to increase dystrophin expression within protocol specified time limits.
  2. * Abnormality in protocol-specified diagnostic evaluations or laboratory tests.
  3. * 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.

Contacts and Locations

Principal Investigator

Medical Director
STUDY_DIRECTOR
Sarepta Therapeutics, Inc.

Study Locations (Sites)

Arkansas Children's Hospital
Little Rock, Arkansas, 72202
United States
Lucile Packard Children's Hospital Stanford
Palo Alto, California, 94304
United States
University of California at Davis Medical Center
Sacramento, California, 95817
United States
Rady Children's Hospital-San Diego
San Diego, California, 92123
United States
University of Florida, UF Health Center for Pediatric Neuromuscular and Rare Diseases
Gainesville, Florida, 32608
United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
United States
The Johns Hopkins Hospital, Charlotte R. Bloomberg Children's Center, Pediatric Clinical Research Unit
Baltimore, Maryland, 21287
United States
Boston Children's Hospital
Boston, Massachusetts, 02115
United States
Washington University of St. Louis, St. Louis Children's Hospital
Saint Louis, Missouri, 63110
United States
University of Rochester, Department of Neurology
Rochester, New York, 14642
United States
Lenox Baker Children's Hospital (Duke University)
Durham, North Carolina, 27705
United States
Nationwide Children's Hospital
Columbus, Ohio, 43205
United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
United States
Children's Hospital of the King's Daughters
Norfolk, Virginia, 23510
United States

Collaborators and Investigators

Sponsor: Sarepta Therapeutics, Inc.

  • Medical Director, STUDY_DIRECTOR, Sarepta Therapeutics, Inc.

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2023-05-31
Study Completion Date2028-06-30

Study Record Updates

Study Start Date2023-05-31
Study Completion Date2028-06-30

Terms related to this study

Keywords Provided by Researchers

  • DMD
  • Gene-Delivery
  • Pediatric
  • North Star Ambulatory Assessment (NSAA)
  • Performance of Upper Limb (PUL)
  • Duchenne

Additional Relevant MeSH Terms

  • Duchenne Muscular Dystrophy