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
Phase of Trial: Phase II
Latest Information Update: 16 Nov 2017
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
- 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.