TERMINATED

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

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Conditions

Duchenne Muscular DystrophyGene-DeliveryDMDAmbulatoryPediatricDuchenneSeropositivePre-existing antibodiesAAVrh74 antibodyPlasmapheresisTiterSeropositivity

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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

This is a gene transfer therapy study evaluating the safety of and delandistrogene moxeparvovec dystrophin protein expression from delandistrogene moxeparvovec following therapeutic plasma exchange (plasmapheresis) in ambulatory male participants with DMD and pre-existing antibodies to AAVrh74 over a period of 59 weeks.

Official Title

An Open-Label, Systemic Gene Delivery Study to Evaluate the Safety, Tolerability and Expression of Delandistrogene Moxeparvovec Following Plasmapheresis in Subjects With Duchenne Muscular Dystrophy and Pre-existing Antibodies to AAVrh74

Quick Facts

Study Start:2024-09-18
Study Completion:2025-08-05
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:TERMINATED

Study ID

NCT06597656

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:4 Years to 8 Years
Sexes Eligible for Study:MALE
Accepts Healthy Volunteers:No
Standard Ages:CHILD
Inclusion CriteriaExclusion Criteria
  1. * Ambulatory per protocol specified criteria.
  2. * Has a definitive diagnosis of DMD prior to Screening based on documentation of clinical findings and prior confirmatory genetic testing.
  3. * Ability to cooperate with motor assessment testing.
  4. * Has elevated AAVrh74 antibody titers per protocol-specified requirements.
  5. * A pathogenic frameshift mutation, nonsense mutation or premature stop codon or pathogenic variant in the DMD gene that is expected to lead to absence of dystrophin protein with exception of a mutation in exon 8 and/or 9.
  6. * 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).
  1. * Has reduced left ventricular ejection fraction on the screening ECHO or clinical signs and/or symptoms of cardiomyopathy.
  2. * Presence of any other clinically significant illness, including cardiac, pulmonary, hepatic, renal, hematologic, immunologic, or behavioral disease, or infection or malignancy or concomitant illness or requirement for chronic drug treatment that in the opinion of the Investigator creates unnecessary risks for gene transfer or a medical condition or extenuating circumstance that, in the opinion of the Investigator, might compromise the participant's ability to comply with the protocol required testing or procedures or compromise the participant's wellbeing, safety, or clinical interpretability.
  3. * Exposure to gene therapy, investigational medication, or other protocol-specified treatment within the protocol specified time limits.
  4. * Abnormality in protocol-specified diagnostic evaluations or laboratory tests. .

Contacts and Locations

Principal Investigator

Medical Director
STUDY_DIRECTOR
Sarepta Therapeutics, Inc.

Study Locations (Sites)

University of Florida, College of Medicine
Gainesville, Florida, 32610
United States
Washington University School of Medicine in St. Louis
St Louis, Missouri, 63110
United States
Nationwide Children's Hospital
Columbus, Ohio, 43205
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 Date2024-09-18
Study Completion Date2025-08-05

Study Record Updates

Study Start Date2024-09-18
Study Completion Date2025-08-05

Terms related to this study

Keywords Provided by Researchers

  • Gene-Delivery
  • DMD
  • Ambulatory
  • Pediatric
  • Duchenne
  • Seropositive
  • Pre-existing antibodies
  • AAVrh74 antibody
  • Plasmapheresis
  • Titer
  • Seropositivity

Additional Relevant MeSH Terms

  • Duchenne Muscular Dystrophy