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

NCT ID: NCT06597656

Last Updated: 2025-09-04

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE1
• Total Enrollment: 3 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2024-09-18
• Study Completion Date: 2025-08-05

Brief Summary

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.

Conditions

Duchenne Muscular Dystrophy

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Delandistrogene Moxeparvovec After Plasmapheresis Procedure

Participants will receive a single intravenous (IV) infusion of delandistrogene moxeparvovec on Day 1 after plasmapheresis procedure if AAVrh74 antibodies are sufficiently low.

Group Type: EXPERIMENTAL

delandistrogene moxeparvovec

Intervention Type: GENETIC

Single IV infusion of delandistrogene moxeparvovec

Plasmapheresis

Intervention Type: PROCEDURE

Therapeutic plasma exchange procedure

Interventions

delandistrogene moxeparvovec

Single IV infusion of delandistrogene moxeparvovec

Intervention Type: GENETIC

Plasmapheresis

Therapeutic plasma exchange procedure

Intervention Type: PROCEDURE

Other Intervention Names

SRP-9001; delandistrogene moxeparvovec-rokl; ELEVIDYS

Eligibility Criteria

Inclusion Criteria

• Ambulatory per protocol specified criteria.
• Has a definitive diagnosis of DMD prior to Screening based on documentation of clinical findings and prior confirmatory genetic testing.
• Ability to cooperate with motor assessment testing.
• Has elevated AAVrh74 antibody titers per protocol-specified requirements.
• 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.
• 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).

Exclusion Criteria

• Has reduced left ventricular ejection fraction on the screening ECHO or clinical signs and/or symptoms of cardiomyopathy.
• 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.
• Exposure to gene therapy, investigational medication, or other protocol-specified treatment within the protocol specified time limits.
• Abnormality in protocol-specified diagnostic evaluations or laboratory tests. .

• Minimum Eligible Age: 4 Years
• Maximum Eligible Age: 8 Years
• Eligible Sex: MALE
• Accepts Healthy Volunteers: No

Sponsors

Sarepta Therapeutics, Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Medical Director

Role: STUDY_DIRECTOR

Sarepta Therapeutics, Inc.

Locations

University of Florida, College of Medicine

Gainesville, Florida, United States

Washington University School of Medicine in St. Louis

St Louis, Missouri, United States

Nationwide Children's Hospital

Columbus, Ohio, United States

Countries

United States

Other Identifiers

SRP-9001-105

Identifier Type: -

Identifier Source: org_study_id