CI

At a glance

ClinicalIndex Comparison Record
Phase 2Completed· 36 enrolled
Drug / intervention
Cysteamine Bitartratedrug
Likely dose
Not stated in record
Structured eligibility isn't available for this trial yet — see the full criteria in the Eligibility tab below.

Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.

Search/NCT02023866
NCT02023866Phase 2Completed
CompareCT.gov

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

Amgen·interventional·Posted Dec 30, 2013·Updated Dec 27, 2024

In Brief

A Phase 2 clinical trial evaluating Cysteamine Bitartrate for Inherited Mitochondrial Disease, Including Leigh Syndrome. Completed, enrolled 36 participants across 5 sites.

Detailed Summary

To evaluate safety, tolerability and efficacy of cysteamine bitartrate delayed-release capsules (RP103) administered at a target maintenance dose of 1.3 g/m²/day in two divided doses, every 12 hours, for up to 6 months in patients with inherited mitochondrial disease.

Study Details

Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsInherited Mitochondrial Disease, Including Leigh Syndrome
CountriesUnited States
Collaborators--

Timeline

Phase 2CompletedFinished
2014201520162017201820192020202120222023202420252026
First PostedDec 30, 2013
Enrollment StartMay 1, 2014
Primary CompletionOct 1, 2016
TodayJul 2, 2026
Enrollment to primary: 2.4 yearsPosted 12.5 years ago

Interventions

Cysteamine Bitartratedrug

Cysteamine Bitartrate Delayed-release capsules