Duchenne muscular dystrophy (DMD)

BMN 351

(351-201)

Trial Status Recruiting

Condition

Duchenne Muscular Dystrophy

Technology

Antisense Oligonucleotide

Title

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

Study Description

This Phase 1/2 clinical study will evaluate the safety and tolerability of BMN 351 in patients with Duchenne Muscular Dystrophy (DMD) with a genetic mutation amenable to exon 51 skipping.

Primary Outcome Measure*

Incidence of adverse and serious adverse events with BMN 351

Select Inclusion Criteria*

Ambulatory patients 4-10 years of age
Diagnosis of DMD with a mutation amenable to exon 51 skipping

Secondary Outcome Measure*

Pharmacokinetic (PK) concentrations of BMN 351 in plasma, urine and muscle

Select Exclusion Criteria*

History of gene therapy treatment
Use of exon skipping therapy within 12 weeks prior to first visit

* Additional measures/criteria may apply.
DMD, duchenne muscular dystrophy.

For reference and comprehensive trial information, visit ClinicalTrials.gov NCT06280209

Pre-IND

Phase 1

Phase 2

Phase 3

Phase 4

Qualitative interviews with caregivers of children aged 4–7 years with Duchenne muscular dystrophy to assess content validity of the proxy-completed DMD-QOL

Quantitative measurement of near full-length dystrophin and muscle content normalizer proteins in human muscle by IA‑UPLC-MS/MS

A phase 1/2 clinical trial of the antisense oligonucleotide BMN 351 now actively recruiting boys with exon 51–skip-amenable Duchenne muscular dystrophy

Development of a next-generation multiplex ddPCR assay for measurement of in-frame dystrophin mRNA in subjects with Duchenne muscular dystrophy treated with BMN 351