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

Launched by SAREPTA THERAPEUTICS, INC. · Sep 12, 2024

Keywords

ClinConnect Summary

This clinical trial is testing a new gene therapy called delandistrogene moxeparvovec (also known as SRP-9001) for boys with Duchenne Muscular Dystrophy (DMD), a serious condition that affects muscle strength. The study aims to see if this therapy is safe and effective for participants who already have antibodies against a certain virus used in the treatment. Participants will be boys aged 14 to 21 who can walk and have a confirmed diagnosis of DMD. They must also have been stable on a daily dose of corticosteroids for at least 12 weeks before the study.

If eligible and chosen to participate, boys will undergo a procedure called plasmapheresis to help prepare their bodies for the gene therapy. The study will last for 58 weeks, during which participants will receive the therapy and be monitored closely for any side effects or improvements in their condition. It's important to note that not everyone with DMD will qualify for the study, as those with certain heart issues or other serious health concerns may be excluded to ensure their safety. This trial is currently recruiting participants, so families interested in this potential treatment should consider discussing it with their healthcare provider.

Gender

MALE

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. .
• • Note: Other inclusion or exclusion criteria could apply.