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REN001 for Adults with Primary Mitochondrial Myopathy

REN001 for Adults with Primary Mitochondrial Myopathy

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If you are interested in participating in the study or want to learn more, please contact: Contact us
This study is no longer recruiting.

Description

The purpose of this new clinical trial is to investigate study drug “REN001” 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 and criteria


IRB Number:
21-018417
Eligible age range:
18 years - 99 years
Clinical trial phase:
Phase II
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)

What to expect


Smiling CHOP patient holding shopping bag

We need families like you

Your time and participation make a difference in supporting the work of the Research Institute and children in our community. Learn more about opportunities to participate in our research.

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