BENITIONSClinical Trial Intelligence

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

第2/3相 早期終了 NCT04378075

対象疾患Mitochondrial Diseases, Drug Resistant Epilepsy, Leigh Disease, Leigh Syndrome
第2/3相
スポンサーPTC Therapeutics
ステータス早期終了
参加者数68 名
介入(薬剤)Vatiquinone
開始日2020年9月28日
完了日2023年3月18日
結果公開日2026年3月25日

概要

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)

BENITIONS で見る ClinicalTrials.gov の原文を見る

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このページは ClinicalTrials.gov の公開データをまとめたものです。医学的助言ではなく、投資助言でもありません。必ず専門医にご相談ください。