A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy

Launched by BIOMARIN PHARMACEUTICAL · Feb 20, 2024

Keywords

ClinConnect Summary

This clinical trial is testing a new treatment called BMN 351 for boys aged 4 to 10 who have Duchenne Muscular Dystrophy (DMD), a genetic condition that affects muscle strength. The main goal of the study is to find out if BMN 351 is safe and well-tolerated in these participants, especially those with a specific genetic change that could benefit from the treatment. To be eligible, boys must be able to walk, have been on a stable dose of steroid medication for the last three months, and not require help with breathing from a ventilator.

Participants in the trial will go through some initial tests to check their heart and lung function before starting the treatment. During the study, they will receive BMN 351 and be monitored closely to see how their bodies respond. It’s important to note that boys who have received certain types of previous therapies, like gene therapy or other exon skipping treatments, won't be able to participate. This trial is currently recruiting participants, and it's an exciting opportunity for those who qualify to help advance research in DMD.

Gender

MALE

Eligibility criteria

• Inclusion Criteria:
• • Age 4 to 10
• • Diagnosis of Duchenne muscular dystrophy with a specific genetic change amenable to exon 51 skipping
• • Able to walk
• • Not requiring assistance from a ventilator to breathe
• • Currently on consistent doses of steroid treatment for the last 12 weeks
• Exclusion Criteria:
• • The participant will have some initial clinical labs and studies to assess baseline level of heart and lung function.
• • Treatment with an exon skipping therapy within 12 weeks prior to the first visit.
• • Any history of treatment with gene therapy