Effect, Safety and Pharmacokinetics Study of PRO051 to Treat DMD
Recruiting
- Conditions
- Duchenne Muscular Dystrophy
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- Not specified
- Target Recruitment
- 18
Inclusion Criteria
1. Boys aged between 5 and 16 years inclusive
2. Duchenne muscular dystrophy resulting from a mutation correctable by treatment with PRO051
Exclusion Criteria
1. Aberrant RNA splicing and/or aberrant response to PRO051, detected by in vitro PRO051 assay during screening
2. Known presence of dystrophin in > 5% of fibres in a pre-study diagnostic muscle biopsy
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Effect of PRO051 on dystrophin expression in muscle and on muscle strength and function <br /><br><br /><br>Safety and tolerability of PRO051<br /><br><br>Pharmacokinetics of PRO051
- Secondary Outcome Measures
Name Time Method