BENITIONSClinical Trial Intelligence

A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy

Phase 2/3 Terminated NCT04378075

ConditionMitochondrial Diseases, Drug Resistant Epilepsy, Leigh Disease, Leigh Syndrome
PhasePhase 2/3
SponsorPTC Therapeutics
StatusTerminated
Enrollment68 participants
InterventionsVatiquinone
Start dateSep 28, 2020
Completion dateMar 18, 2023
Results postedMar 25, 2026

Summary

This is a parallel-arm, double-blind, placebo-controlled study with a screening phase that includes a 28-day run-in phase to establish baseline seizure frequency, followed by a 24-week, randomized, placebo-controlled phase. After completion of the randomized, placebo-controlled phase, participants may enter a 48-week, long-term, extension phase during which they will receive open-label treatment with vatiquinone.

Percent Change From Baseline to Week 24 in the Number of Observable Motor Seizures Per 28 Days During the Double-blind Period

Baseline to Week 24 · percent change

Double-blind Period: Vatiquinone-12.74 ± -28.67 percent change
Double-blind Period: Placebo-0.33 ± -28.97 percent change
p-value= 0.173
Median Difference (Final Values)-8.31 (95% CI -31.3–16.4)

Open in BENITIONS View on ClinicalTrials.gov

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