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

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying a drug called BMN 351 for people with duchenne muscular dystrophy. The study is currently recruiting participants at 8 locations. People eligible for this study include men aged 4 Years to 10 Years.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUGBMN 351

Anti-sense Oligonucleotide BMN 351 will be administered intravenously.

Locations(8)

Children's Hospital LHSC

London, Ontario, Canada

Fondazione Serena ETS - Centro Clinico NeMO Milano

Milan, Italy

UOC Fase I - Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro Cuore

Rome, Italy

Leids Universitair Medisch Centrum

Leiden, Netherlands

Hospital Sant Joan de Deu

Barcelona, Spain

Hospital Viamed Santa Angela De la Cruz

Seville, Spain

Yeditepe University Kosuyolu Hospital

Istanbul, Turkey (Türkiye)

Great Ormond Street Hospital NHS Foundation Trust

London, United Kingdom

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NCT06280209

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