Clinical Trial: 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)

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

Detailed Summary:
Sponsor: Santhera Pharmaceuticals

Current Primary Outcome: Primary objective: to establish the pharmacokinetic profile of omigapil at a range of doses in paediatric and adolescent patients with CMD. [ Time Frame: Week 4 and Week 8 ]

Original Primary Outcome: Same as current

Current Secondary Outcome: Secondary objective: to evaluate the safety and tolerability of omigapil at a range of doses in paediatric and adolescent patients. [ Time Frame: Baseline, Week 4, 8 and 12 ]

Original Secondary Outcome: Same as current

Information By: Santhera Pharmaceuticals

Dates:
Date Received: March 4, 2013
Date Started: December 2014
Date Completion:
Last Updated: February 13, 2017
Last Verified: February 2017