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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
Sponsor: BioMarin Pharmaceutical
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.
Official title: 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
Key Details
Gender
MALE
Age Range
4 Years - 10 Years
Study Type
INTERVENTIONAL
Enrollment
18
Start Date
2024-01-03
Completion Date
2026-09-30
Last Updated
2026-04-08
Healthy Volunteers
No
Conditions
Interventions
BMN 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