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

Phase 1/2 Active not recruiting 18 enrolled BioMarin Pharmaceutical

Non-randomizedSequentialOpen-labelTreatment

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.

Timeline

Start: 2024-01-03
Primary completion: 2027-04-14
Completion: 2027-04-14

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	BMN 351	Antisense oligonucleotide	—	Intravenous

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

Summary

Timeline

Drugs

Indications