BENITIONSClinical Trial Intelligence

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
分期2/3期
申办方PTC Therapeutics
状态提前终止
受试者人数68 人
干预(药物)Vatiquinone
开始日期2020年9月28日
完成日期2023年3月18日
结果公布日2026年3月25日

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
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)

在 BENITIONS 中查看 查看 ClinicalTrials.gov 原文

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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。