A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy
2/3期
提前终止
NCT04378075
| 适应症 | Mitochondrial Diseases, Drug Resistant Epilepsy, Leigh Disease, Leigh Syndrome |
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| 分期 | 2/3期 |
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| 申办方 | PTC Therapeutics |
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| 状态 | 提前终止 |
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| 受试者人数 | 68 人 |
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| 干预(药物) | Vatiquinone |
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| 开始日期 | 2020年9月28日 |
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| 完成日期 | 2023年3月18日 |
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| 结果公布日 | 2026年3月25日 |
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摘要
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) |
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本页整理自 ClinicalTrials.gov 的公开数据,不构成医疗建议,也不构成投资建议。请务必咨询专业医师。