BMN 351 Exon 51 Skipping Therapy for Duchenne Muscular Dystrophy: Phase 1/2 Clinical Trial
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
Contacts and Locations
Contacts and Locations
Locations
This study has 7 locations
Italy
Milan, Italy
Fondazione Serena ETS - Centro Clinico NeMO Milano
Rome, Italy
UOC Fase I - Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro Cuore
Netherlands
Leiden, Netherlands, 2333 ZA
Leids Universitair Medisch Centrum
Spain
Barcelona, Spain, 08950
Hospital Sant Joan de Deu
Seville, Spain, 41013
Hospital Viamed Santa Angela De la Cruz
Turkey (Türkiye)
Istanbul, Turkey (Türkiye)
Yeditepe University Kosuyolu Hospital
United Kingdom
London, United Kingdom, WC1N 3JH
Great Ormond Street Hospital NHS Foundation Trust
Clinical Trial Registry
NCT ID
NCT06280209This information is provided for educational purposes only. Always consult the study investigators before making medical decisions.