RecruitingPhase 1Phase 2NCT06280209

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

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

Sponsor

BioMarin Pharmaceutical

Enrollment

18 participants

Start Date

Jan 3, 2024

Study Type

INTERVENTIONAL

Conditions

Duchenne Muscular Dystrophy

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.

Eligibility

Sex: MALEMin Age: 4 YearsMax Age: 10 Years

Inclusion Criteria5

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 Criteria3

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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