Gene therapy for Duchenne Muscular Dystrophy using plasmapheresis
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
This study is testing a new gene therapy combined with a blood treatment to see if it can help boys with Duchenne Muscular Dystrophy who have certain antibodies.
Quick facts
| Phase | Phase 1 |
|---|---|
| Study type | Interventional |
| Enrollment | 16 (estimated) |
| Ages | 4 Years to 8 Years |
| Sex | Male |
| Sponsor | Sarepta Therapeutics, Inc. Industry-sponsored |
| Locations | 2 sites (Saint Louis, Missouri and 1 other locations) |
| Trial ID | NCT06597656 on ClinicalTrials.gov |
What this trial studies
This clinical trial evaluates the safety and efficacy of delandistrogene moxeparvovec, a gene transfer therapy, in ambulatory male participants with Duchenne Muscular Dystrophy (DMD) who have pre-existing antibodies to AAVrh74. The study involves therapeutic plasma exchange (plasmapheresis) to enhance the treatment's effectiveness. Participants will be monitored over a period of 58 weeks to assess dystrophin protein expression and overall safety. The trial aims to provide insights into the potential of gene therapy for this condition.
Who should consider this trial
Good fit: Ideal candidates are ambulatory male participants diagnosed with DMD who have elevated AAVrh74 antibody titers.
Not a fit: Patients with significant cardiac issues or other clinically significant illnesses may not benefit from this study.
Why it matters
Potential benefit: If successful, this therapy could significantly improve muscle function and quality of life for patients with Duchenne Muscular Dystrophy.
How similar studies have performed: Other studies have shown promise with gene therapy approaches for DMD, indicating potential for success in this novel application.
Eligibility criteria
Show full inclusion / exclusion 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. . Note: Other inclusion or exclusion criteria could apply.
Where this trial is running
Saint Louis, Missouri and 1 other locations
- Washington University School of Medicine in St. Louis — Saint Louis, Missouri, United States (Recruiting)
- Nationwide Children's Hospital — Columbus, Ohio, United States (Recruiting)
Study contacts
- Study coordinator: Sarepta Therapeutics Inc., For Clinical Trial Information, Select Option 4
- Email: SareptAlly@sarepta.com
- Phone: 1-888-SAREPTA (1-888-727-3782)
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.