A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy
Phase 2/3
Terminated
NCT04378075
| Condition | Mitochondrial Diseases, Drug Resistant Epilepsy, Leigh Disease, Leigh Syndrome |
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| Phase | Phase 2/3 |
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| Sponsor | PTC Therapeutics |
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| Status | Terminated |
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| Enrollment | 68 participants |
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| Interventions | Vatiquinone |
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| Start date | Sep 28, 2020 |
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| Completion date | Mar 18, 2023 |
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| Results posted | Mar 25, 2026 |
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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 |
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| Double-blind Period: Placebo | -0.33 ± -28.97 percent change |
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| 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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