Clinical Trial

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

Active, Not Recruiting Phase 1/2
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Summary
The purpose of this study is to test the safety and tolerability of BMN 351 in participants with Duchenne Muscular Dystrophy (DMD) with a genetic mutation amenable to exon 51 skipping.
Protocol Amendment History 3 changes
critical Trial status changed: Recruiting → Active, Not Recruiting 2026-06-26
notable Primary completion pushed: 2026-09-30 -> 2027-04-14 2026-06-26
minor Completion pushed: 2026-09-30 -> 2027-04-14 2026-06-26
Trial Details
NCT Number NCT06280209
Lead Sponsor BioMarin Pharmaceutical
Conditions Duchenne Muscular Dystrophy
Enrollment 18 participants
Start Date 2024-01-03
Primary Completion 2027-04-14 (estimated)
Study Completion 2027-04-14 (estimated)
Updated on ClinicalTrials.gov 2026-06-25