A Gene Delivery Study to Evaluate the Safety and Expression of Delandistrogene Moxeparvovec in Participants Under the Age of Four With Duchenne Muscular Dystrophy (DMD)

Launched by HOFFMANN-LA ROCHE · Nov 8, 2023

Keywords

ClinConnect Summary

This clinical trial is investigating a new gene therapy called delandistrogene moxeparvovec for young boys with Duchenne Muscular Dystrophy (DMD), a condition that affects muscle strength and function. The study aims to determine how safe this treatment is and how well it can help produce a protein that is missing in DMD. Boys aged from a few months up to 4 years old may be eligible to participate, provided they have a confirmed diagnosis of DMD and specific genetic changes related to the disease.

Participants in the study will be monitored for about 264 weeks, or roughly five years, to see how they respond to the treatment. It’s important to know that boys who have previously received certain gene therapies, are on specific medications like corticosteroids, or have other significant health issues might not be able to join. This study is currently recruiting participants, and caregivers can expect ongoing assessments to ensure the child's safety and gather important information about the therapy's effects.

Gender

MALE

Eligibility criteria

• Inclusion Criteria:
• • Cohort A: \>=3 years of age to \<4 years of age
• • Cohort B: \>=2 years of age to \<3 years of age
• • Cohort C: \>6 months to \<2 years of age
• • Cohort D: \<=6 months of age
• • Has a definitive diagnosis of DMD prior to screening based on documentation of clinical findings and prior confirmatory genetic testing using a clinical diagnostic genetic test
• • Able to cooperate with age-appropriate motor assessment testing
• • A pathogenic frameshift mutation or premature stop codon contained between exons 18 and 79 (inclusive)
• Exclusion Criteria:
• • Exposure to gene therapy, investigational medication, or any treatment designed to increase dystrophin expression, within protocol-specified time limits
• • Recombinant Adeno-Associated Virus Serotype rh74 (rAArh74) antibody titers are elevated, as per protocol-specified criteria
• • Receiving regular oral corticosteroids as a treatment for DMD or planning to receive oral corticosteroids as a treatment for DMD within 1 year of baseline
• • 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
• • 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 well-being or safety, or clinical interpretability
• • Other inclusion or exclusion criteria could apply