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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
- ItalyNetherlandsSpainTurkiyeUnited 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
Contact Information
This section provides contact details for people who can answer questions about joining this study
Not yet provided
Clinical Trial Registry
NCT ID
NCT06280209This information is provided for educational purposes only. Always consult the study investigators before making medical decisions.