A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy
BMN 351: Anti-sense Oligonucleotide BMN 351 will be administered intravenously.
Study 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
MALE
Min age
4 Years
Max age
10 Years
Healthy volunteers
No
Inclusion Criteria:
* Age 4 to 10
* Diagnosis of Duchenne muscular dystrophy with a specific genetic change amenable to exon 51 skipping
* Able to walk
* Not requiring assistance from a ventilator to breathe
* Currently on consistent doses of steroid treatment for the last 12 weeks
Exclusion Criteria:
* The participant will have some initial clinical labs and studies to assess baseline level of heart and lung function.
* Treatment with an exon skipping therapy within 12 weeks prior to the first visit.
* Any history of treatment with gene therapy
Primary outcome measure(s)
To evaluate and safety and tolerability of single and multiple doses of BMN 351 (incidence, severity, and dose-relationship of adverse effects and changes in laboratory parameters). — Up to 97 weeks. The safety and tolerability of BMN 351 will be assessed based on the incidence of adverse and serious adverse events.
Trial sites (7)
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
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.
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