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An Open-Label, Dose-Escalating Study to Assess the Safety, Tolerability, Efficacy, Pharmacokinetics and Pharmacodynamics of Cysteamine Bitartrate Delayed-release Capsules (RP103) for Treatment of Children With Inherited Mitochondrial Disease

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Trial Profile

An Open-Label, Dose-Escalating Study to Assess the Safety, Tolerability, Efficacy, Pharmacokinetics and Pharmacodynamics of Cysteamine Bitartrate Delayed-release Capsules (RP103) for Treatment of Children With Inherited Mitochondrial Disease

Status: Completed
Phase of Trial: Phase II

Latest Information Update: 07 Nov 2021

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At a glance

  • Drugs Mercaptamine bitartrate (Primary)
  • Indications Leigh disease; Mitochondrial disorders
  • Focus Therapeutic Use
  • Acronyms MITO-001
  • Sponsors Horizon Pharma USA; Raptor Pharmaceutical Corp
  • Most Recent Events

    • 15 Feb 2017 Status changed from recruiting to completed.
    • 25 Oct 2016 This trial was planned by Raptor Pharmaceutical Corp. The company has now been acquired by Horizon Pharma plc, according to a Horizon Pharmaceuticals media release.
    • 06 May 2016 According to a Raptor Pharmaceuticals media release, the second interim analysis occurred after 12 patients completed 24 weeks of treatment and indicated the trial should continue as planned.
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