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

StatusActive, Not Recruiting
PhasePhase 1/2
Started
View on ClinicalTrials.gov ↗

Record History

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  1. Primary and study completion pushed from Sep 30, 2026 to Apr 14, 2027
    • Trial status changed: Recruiting → Active, Not Recruiting
    • Study sites: 8 to 7
    v9
  2. Site contacts updated at 1 of 8 sitesv8
  3. Study sites: 7 to 8v7
  4. Re-verified, no change to tracked fieldsv6
  5. Primary and study completion dates: Dec 2025 to Sep 30, 2026
    • Secondary endpoint revised
    • Site contacts updated at 7 of 7 sites
    v5
Show 3 earlier versionsHide 3 earlier versions
  1. Study sites: 6 to 7v4
  2. Study sites: 3 to 6v3
  3. Study sites: 1 to 3v2
  1. Study First Submitted
  • Stopped: an ongoing trial was terminated, suspended or withdrawn.
  • Notable: the trial’s plan changed such as a big move in its timeline or size, a new primary endpoint, sponsor or phase, or recruiting opened or resumed.
  • Minor: routine progress and housekeeping, such as sites added or enrollment closed.
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.
Trial Details
NCT Number NCT06280209
Lead Sponsor BioMarin Pharmaceutical
Conditions
(from sponsor)		 Duchenne Muscular Dystrophy
Related terms Muscular Dystrophy, Duchenne
Enrollment 18 participants
Start Date
Primary Completion (estimated)
Study Completion (estimated)
Updated on ClinicalTrials.gov