難治性てんかん患者におけるミトコンドリア病治療薬バチキノンの有効性および安全性を評価する研究

基本情報

NCT ID: NCT04378075
ステータス: 中止
試験のフェーズ: 第2/第3相
試験タイプ: 介入
目標被験者数: 68
治験依頼者名: PTC Therapeutics

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.

対象疾患

Mitochondrial DiseasesDrug Resistant EpilepsyLeigh DiseaseLeigh SyndromeMitochondrial Encephalopathy (MELAS)Pontocerebellar Hypoplasia Type 6 (PCH6)Alpers DiseaseAlpers Syndrome

介入

Vatiquinone(DRUG)

Placebo(OTHER)

依頼者（Sponsor）

Ptc Therapeutics(INDUSTRY)