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

Trial Profile

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

Completed
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
  • Most Recent Events

    • 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.
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