BMN 351 Exon 51 Skipping Therapy for Duchenne Muscular Dystrophy: Phase 1/2 Clinical Trial
Active, Not RecruitingPhase 1/2Exon Skipping
Study Overview
- Age
- 4–10 years
- Phase
- Phase 1/2
- Sponsor
- BioMarin Pharmaceutical
- Therapeutic Approach
- Exon Skipping
- Variant Requirement
- Diagnosis of Duchenne muscular dystrophy with a specific genetic change amenable to exon 51 skipping
- Eligible Sex
- Male
- Ambulation
- Ambulatory
- Study Start (Actual)
- 2024-01-03
- Primary Completion (Estimated)
- 2027-04-14
- Study Completion (Estimated)
- 2027-04-14
- Enrollment (Estimated)
- 18
- Countries
- ItalyNetherlandsSpainTurkeyUnited Kingdom
Study Requirements and Criteria
Steroid Use
Currently on consistent doses of steroid treatment for the last 12 weeks.
Inclusion Criteria
Not requiring assistance from a ventilator to breathe
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
Clinical Trial Registry
NCT ID
NCT06280209This information is provided for educational purposes only. Always consult the study investigators before making medical decisions.