REN001 for Adults with Primary Mitochondrial Myopathy

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The purpose of this new clinical trial is to investigate study drug β€œREN001” in adult patients with Primary Mitochondrial Myopathy. REN001 is currently not approved by the U.S. Food and Drug Administration nor any other regulatory agencies for this disease; it is considered an experimental drug. The study period will last for approximately 9 months and will include at least 4 outpatient visits at CHOP, where participants will be provided the study drug or placebo and monitored for safety. The study also involves blood and urine tests, cardiac tests, physical exercise tests, and other study procedures. For more information: https://www.clinicaltrials.gov/ct2/show/NCT04535609 

Participants will be able to receive travel support and reimbursement for related expenses.

Eligibility & Criteria

IRB #:
21-018417
Official Title:
A Double-Blind, Placebo-Controlled, Study to Evaluate the Efficacy and Safety of 24 Weeks Treatment with REN001 in Patients with Primary Mitochondrial Myopathy (PMM)
Study Phase:
Phase II
Eligible Age Range:
18 - 99 Years
Gender:
All
Study Categories:

Visit Criteria

The study will require at least 4 outpatient visits at CHOP; procedures include:

  • Medical history review; physical examinations
  • Cardiac tests; eye examinations; wrist x-ray (if you are younger than 25 years)
  • Physical exercise tests
  • Blood and urine tests
  • Study assessments; review of eDiary (including pedometer)
  • Randomized to take daily study drug (REN001) or placebo
Leader
Marni J. Falk

Marni J. Falk, MD