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: 31 Dec 2024

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At a glance

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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