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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
Led by BioMarin Pharmaceutical · Updated on 2026-04-08
18
Participants Needed
8
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are studying the safety and tolerability of BMN 351, an anti-sense oligonucleotide drug, in boys aged 4 to 10 with Duchenne Muscular Dystrophy (DMD) who have a genetic mutation suitable for exon 51 skipping. This Phase 1/2, open-label, multi-center trial aims to evaluate increasing doses of BMN 351 and monitor its effects and safety in this specific group. Participants will be assigned to one of four groups called cohorts. Cohort 1A participants will first receive escalating doses every two weeks for up to 8 weeks, then move to weekly dosing for up to 89 weeks. Cohort 1B, 2, and 3 will receive low, medium, or high doses once weekly for up to 97, 73, or 48 weeks respectively. BMN 351 is given intravenously during these treatment periods. Throughout the study, participants will have safety assessments for up to 97 weeks, including monitoring for adverse effects and laboratory tests. Pharmacokinetic samples of BMN 351 will be collected from plasma, urine, and muscle about every 8 weeks. The study includes evaluations of heart and lung function at baseline and ongoing safety follow-up. The total estimated participation time varies by cohort and dosing schedule.
CONDITIONS
Brief 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...
- Male children aged 4 to 10 years
- Diagnosed with Duchenne Muscular Dystrophy with a genetic mutation amenable to exon 51 skipping
- Able to walk without assistance
- Not requiring ventilator support for breathing
- On a stable dose of steroid treatment for at least 12 weeks
You will not qualify if you...
- History of treatment with exon skipping therapy within 12 weeks before study start
- Any previous gene therapy treatment
- Baseline heart and lung function assessments indicating unsuitability for participation
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
Duration - Up to 97 weeks depending on cohort
Participants receive intravenous BMN 351 at escalating doses according to cohort assignment. Cohort 1A receives dosing once every 2 weeks for up to 8 weeks then once weekly for up to 89 weeks. Cohorts 1B, 2, and 3 receive low, medium, or high doses once weekly for up to 97, 73, or 48 weeks respectively.
Weekly visits during dosing periods with additional visits the week after dosing in Cohort 1A during dose escalation
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
Actively Recruiting
3
UOC Fase I - Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro Cuore
Rome, Italy
Actively Recruiting
4
Leids Universitair Medisch Centrum
Leiden, Netherlands, 2333 ZA
Actively Recruiting
5
Hospital Sant Joan de Deu
Barcelona, Spain, 08950
Actively Recruiting
6
Hospital Viamed Santa Angela De la Cruz
Seville, Spain, 41013
Actively Recruiting
7
Yeditepe University Kosuyolu Hospital
Istanbul, Turkey (Türkiye)
Actively Recruiting
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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