A Long-Term Open-Label Extension Study of RP103-MITO-001 to Assess the Safety, Tolerability and Efficacy of Cysteamine Bitartrate Delayed-release Capsules (RP103) for Treatment of Children With Inherited Mitochondrial Disease
Phase of Trial: Phase II
Latest Information Update: 17 Apr 2017
At a glance
- Drugs Mercaptamine bitartrate (Primary)
- Indications Mitochondrial disorders
- Focus Therapeutic Use
- Sponsors Horizon Pharma; Raptor Pharmaceutical Inc
- 11 Apr 2017 Status changed from active, no longer recruiting to completed.
- 16 Feb 2017 Time frame of primary endpoint has been changed from Baseline vs. Month 24 to Baseline, Every 3 months and Study Exit (up to 24 Months). and Age group also changed from 2-17years to 6-17years.
- 16 Feb 2017 Planned End Date changed from 1 May 2018 to 1 Mar 2017.