Active, Not Recruiting
Phase 1
Phase 2
Age: 4Years - 10Years
MALE
ID06280209
A Phase 12, Open-Label, Dose Escalation Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Multiple Intravenous Doses of BMN 351 in Participants With Duchenne Muscular Dystrophy
Led by BioMarin Pharmaceutical · Updated on 2026-06-25
18
Participants Needed
7
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Brief Title
A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy
Who Can Participate
Age: 4Years - 10Years
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
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