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A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy
Led by BioMarin Pharmaceutical · Updated on 2026-04-08
18
Participants Needed
8
Research Sites
143 weeks
Total Duration
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AI-Summary
What this Trial Is About
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
Official Title
A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age between 4 and 10 years
- Diagnosis of Duchenne muscular dystrophy with a genetic mutation amenable to exon 51 skipping
- Able to walk without assistance
- Not requiring ventilator support to breathe
- On consistent steroid treatment doses for at least 12 weeks
You will not qualify if you...
- Undergoing initial clinical labs and tests to assess baseline heart and lung function
- Received exon skipping therapy within 12 weeks before the first visit
- History of treatment with gene therapy
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 8 locations
1
Children's Hospital LHSC
London, Ontario, Canada, N6A 5W9
Actively Recruiting
2
Fondazione Serena ETS - Centro Clinico NeMO Milano
Milan, Italy
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3
UOC Fase I - Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro Cuore
Rome, Italy
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4
Leids Universitair Medisch Centrum
Leiden, Netherlands, 2333 ZA
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5
Hospital Sant Joan de Deu
Barcelona, Spain, 08950
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6
Hospital Viamed Santa Angela De la Cruz
Seville, Spain, 41013
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7
Yeditepe University Kosuyolu Hospital
Istanbul, Turkey (Türkiye)
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8
Great Ormond Street Hospital NHS Foundation Trust
London, United Kingdom, WC1N 3JH
Actively Recruiting
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
4
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