drugset / Trial / NCT01370447

EPI-743 for Mitochondrial Respiratory Chain Diseases

NCT01370447 ↗

Expanded access No longer available PTC Therapeutics

Summary

This study will evaluate the safety and efficacy of EPI-743 in participants with severe mitochondrial respiratory chain diseases who are considered to be within 90 days of end-of-life care.

Timeline

Start
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Primary completion
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Completion
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Drugs

EvaluationDrugModalityDoseRoute
Subject Vatiquinone Small molecule — Oral

Indications