An Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of BMN 351 in Participants With Duchenne Muscular Dystrophy
Condition(s) studied
Investigational drug(s) / intervention(s)
BMN 351: Anti-sense Oligonucleotide BMN 351 will be administered intravenously
Study summary
This open-label extension study aims to evaluate the long-term safety and tolerability of weekly BMN 351 infusions, as well as to assess the effect of BMN 351 on physical function, in participants with DMD who participated in the 351-201 study.
Eligibility
Primary outcome measure(s)
- To assess the long-term safety and tolerability of BMN 351 in participants with DMD — Through study completion, at least 1 year
The safety and tolerability of BMN 351 will be assessed based on the incidence of adverse and serious adverse events.
Trial sites (6)
| Facility | City | Region | Status |
|---|---|---|---|
| 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 Viamed Santa Angela De la Cruz | Seville | Spain | |
| Yeditepe University Kosuyolu Hospital | Istanbul | Turkey | |
| Great Ormond Street Hospital NHS Foundation Trust | London | United Kingdom |
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This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT07573631 on ClinicalTrials.gov ↗ ← All trials in the UK