Leigh Syndrome
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CLINICAL_TRIALS trial_result · 2026-03-25Clinical Trial Update: A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy [Phase 2, Phase 3]
A Phase 2/3 study evaluating Vatiquinone for mitochondrial disease in participants with refractory epilepsy has been terminated, and results…
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CLINICAL_TRIALS trial_result · 2025-01-10Clinical Trial Update: The KHENERGYC Study [Phase 2]
Phase 2 KHENERGYC study for mitochondrial diseases is currently suspended.
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CLINICAL_TRIALS trial_result · 2024-10-23Clinical Trial Update: Long-Term Safety and Efficacy Evaluation of EPI-743 in Children With Leigh Syndrome [Phase 2]
This document summarizes the Phase 2 long-term safety and efficacy results of EPI-743 in children with Leigh syndrome.
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CLINICAL_TRIALS trial_result · 2020-11-12Clinical Trial Update: A Dose-escalating Clinical Trial With KH176 [Phase 1]
Phase 1 dose-escalation trial of KH176 in mitochondrial diseases completed with results posted.
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CLINICAL_TRIALS trial_result · 2020-08-31Clinical Trial Update: Safety and Efficacy Study of EPI-743 in Children With Leigh Syndrome [Phase 2]
Phase 2 clinical trial assessing safety and efficacy of EPI-743 in children with Leigh Syndrome has been completed.
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CLINICAL_TRIALS trial_result · 2017-11-13Clinical Trial Update: Open-Label, Dose-Escalating Study Assessing Safety, Tolerability, Efficacy, of RP103 in Mitochondrial Disease [Phase 2]
Phase 2 open-label, dose-escalation study of RP103 to assess safety, tolerability, and efficacy in inherited mitochondrial diseases, includi…