Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional
Official Title: Congenital Muscular Dystrophy Ascending Multiple Dose Cohort Study Analyzing Pharmacokinetics at Three Dose Levels In Children and Adolescents With Assessment of Safety an
Brief Summary:
The purpose of the study is to establish the pharmacokinetic profile of omigapil in paediatric and adolescent patients with CMD and to evaluate the safety and tolerability of omigapil.
Funding source - FDA OOPD