Inherited Mitochondrial Disease, Including Leigh Syndrome Clinical Trial
Official title:
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
To evaluate safety, tolerability and efficacy of cysteamine bitartrate delayed-release capsules (RP103) administered up to 1.3 g/m²/day in two divided doses, every 12 hours, for up to 6 months in patients with inherited mitochondrial disease.
This is an open-label, dose-escalation 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.
Prior to treatment, patients will undergo a Screening Visit. If eligible, each participant
will return for the Day 1 study visit and begin dosing. Every 2 weeks over the subsequent 8
weeks, participants will alternate between returning to the clinic for detailed assessments
(Weeks 4 and 8) and receiving a telephone call from the Investigator team to assess safety
and RP103 dose (Weeks 2 and 6) and the potential need for an immediate unscheduled study
visit. Thereafter, participants will continue to return to the clinic every 4 weeks for
detailed assessments at Weeks 12, 16, 20, and 24 (the Study Exit visit).
The Study Exit visit will occur at Week 24, and participants will be offered the opportunity
to continue on to an extension study (RP103-MITO-002 [NCT02473445]) until results of the
present study are known.
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