Congenital Muscular Dystrophy Ascending Multiple Dose Cohort Study Analyzing Pharmacokinetics at Three Dose Levels In Children and Adolescents With Assessment of Safety and Tolerability of Omigapil (CALLISTO)

PHASE1CompletedINTERVENTIONAL

Enrollment

Participants

Timeline

Start Date

December 31, 2014

Primary Completion Date

December 5, 2017

Study Completion Date

January 29, 2018

Conditions

Congenital Muscular Dystrophy

Interventions

DRUG

Omigapil

Cohort 1 0.02 mg/kg/day Cohort 2 0.08 mg/kg/day Cohort 3a 0.04 mg/kg/day Cohort 3b 0.06 mg/kg/day

Trial Locations (1)

NINDS, Bethesda