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Open-Label, Dose-Escalating Study Assessing Safety, Tolerability, Efficacy, of RP103 in Mitochondrial Disease

NCT02023866 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 36

Last updated 2024-12-27

Study results available
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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.

Conditions

  • Inherited Mitochondrial Disease, Including Leigh Syndrome

Interventions

DRUG

Cysteamine Bitartrate

Cysteamine Bitartrate Delayed-release capsules

Sponsors & Collaborators

Principal Investigators

  • MD · Amgen

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
6 Years
Max Age
17 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2014-05-31
Primary Completion
2016-10-31
Completion
2016-10-31

Countries

  • United States

Study Locations

More Related Trials

Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02023866 on ClinicalTrials.gov