Astellas Clinical Trial for ASP0367



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We are recruiting patients with genetically confirmed primary mitochondrial myopathy who are at least 18 years old for a randomized, double-blind, placebo-controlled study of a new drug to treat this disorder. This study is designed to assess whether experimental drug ASP0367 is safe and effective for patients with mitochondrial myopathy. Participants will take the study drug orally once per day. This is a placebo-controlled study followed by an open-label extension, which means that during the treatment period some patients will receive ASP0367 and some will receive a placebo, which does not contain active study medication. At the end of the treatment period all participants will be offered ASP0367 for a 6-month period.

The study will last roughly 1 year and 8 months and involves at least 11 visits to CHOP, remote interviews, video assessments, and home visits for additional blood draws. If you are interested in learning more about this study, please visit or contact the study team for additional information.

Who Do I Contact?

If you are interested in participating in the study or want to learn more please contact our study team at dennisl [at] or 267-426-0229.

Eligibility & Criteria

IRB #:
Official Title:
A Randomized, Double-blind, Placebo-controlled Adaptive Phase 2/3 Study with Open-label Extension to Assess the Efficacy, Safety and Tolerability of ASP0367 in Participants with Primary Mitochondrial Myopathy (MOUNTAINSIDE)
Study Phase:
Phase II
Phase III
Eligible Age Range:
18 - 80 Years
Study Categories:

Visit Criteria

  • 11 to 12 visits to CHOP (travel and lodging provided by the study)
  • 14 to 15 Phone Calls
  • 11-12 Home Visits