A Phase 1/2, 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

Trial ID: NCT06280209

Interventional - Phase 1/2 - Active, not recruiting

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

• Duchenne Muscular Dystrophy

Interventions

• BMN 351

Eligibility

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

Sponsor

BioMarin Pharmaceutical

Source: ClinicalTrials.gov

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