Expanded Access Protocol for Vatiquinone in Previously Treated Patients With Inherited Mitochondrial Disease

Medical University of South Carolina0 个研究点开始时间: 2025年9月8日最近更新: 2026年4月9日

适应症

试验速览

阶段: 不适用
状态: No Longer Available
发起方: Medical University of South Carolina

研究概览入排标准研究者记录与标识符相似试验

研究概览

简要总结

The purpose of this study is to provide participants who successfully completed the study titled "An Open-label, Safety Study for Previously Treated Vatiquinone (PTC743) Subjects With Inherited Mitochondrial Disease" with liquid Vatiquinone through an intermediate patient population expanded access protocol.

详细描述

This expanded access protocol serves to make Vatiquinone accessible to subjects with inherited mitochondrial disease who previously participated in a Vatiquinone clinical trial at the Medical University of South Carolina (MUSC) who in the opinion of the investigator would benefit from continued access to liquid Vatiquinone.

研究设计

研究类型: Expanded Access

入排标准

年龄范围: 1 Year 至 —（Child, Adult, Older Adult）
性别: All
接受健康志愿者: 否

入选标准

•Subjects with molecular genetic mitochondrial disease including Leigh syndrome, and Alpers syndrome.
•Completed participation in the study titled "An Open-label, Safety Study for Previously Treated Vatiquinone (PTC743) Subjects With Inherited Mitochondrial Disease"
•In the opinion of the clinical investigator, the continuation to receive Vatiquinone will be of benefit to the participants.
•Enrollment into a clinical trial to receive Vatiquinone is not possible

排除标准

•Current participation in any other interventional study
•Pregnant or currently breast feeding.

研究者

发起方

Medical University of South Carolina

申办方类型

Other

责任方

Principal Investigator

主要研究者

Thomas Koch

Professor-Faculty

Medical University of South Carolina

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