Long-Term Safety & Efficacy Evaluation of EPI-743 in Children With Leigh

Phase 1: Researchers test a new drug or treatment in a small group of people for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
Phase 2: The drug or treatment is given to a larger group of people to see if it is effective and to further evaluate its safety.
Phase 3: The drug or treatment is given to large groups of people to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
Phase 4: Studies are done after the drug or treatment has been marketed to gather information on the drug's effect in various populations and any side effects associated with long-term use.

Status Completed

Clinical Trial Summary

EPI-743 in Leigh syndrome participants that participated in previous EPI743-12-002 (NCT01721733) study.

To monitor the long-term safety and neurodevelopmental effects of EPI-743 on children who complete the EPI743-12-002 placebo-controlled trial. ;

See also
Status	Clinical Trial	Phase
Completed	`NCT04378075` - A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy	Phase 2/Phase 3
Completed	`NCT01721733` - Safety and Efficacy Study of EPI-743 in Children With Leigh Syndrome	Phase 2
Withdrawn	`NCT05277363` - A Study of the Natural Course of SURF1 Deficiency
Completed	`NCT02544217` - A Dose-escalating Clinical Trial With KH176	Phase 1
Recruiting	`NCT03137355` - The International Registry for Leigh Syndrome
Recruiting	`NCT01793168` - Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Recruiting	`NCT05554835` - Global Registry and Natural History Study for Mitochondrial Disorders
Withdrawn	`NCT03747328` - ABI-009 (Nab-sirolimus) in Patients With Genetically-confirmed Leigh or Leigh-like Syndrome	Phase 2