Mitochondrial Medicine Program Clinical Studies

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Astellas Clinical Trial for ASP0367

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 ClinicalTrials.gov or contact the study team for additional information.

DCA Trial in PDC Deficiency

We are studying an investigational drug called sodium dichloroacetate (DCA). Investigational means that the study drug has not been approved by the US Food and Drug Administration (FDA) and that its use in this study is experimental. This a purpose of this research study is to determine whether DCA is a helpful treatment for patient with pyruvate dehydrogenase complex (PDC) deficiency (PDCD).

Mitochondrial Myopathy Rating Scale

The purpose of this research study is to 1) validate the use of surveys such as fatigue scales, Mitochondrial Disease scales, and quality of life scales, 2) validate clinical exams, such as muscle strength tests, and the 6 minute walk test, and 3) to define the natural history of the disease in the Mitochondrial Disease patient population.

MELAS Study

The purpose of this research is to investigate study drug IW-6463 in patients with Mitochondrial Encephalomyopathy, Lactic Acidosis, and Stroke-Like Episodes (MELAS). IW-6463 is not approved by the U.S. Food and Drug Administration for any use.

The study period will last for approximately 75 days and will include at least 3 to 6 on-site visits at the Children’s Hospital of Philadelphia (CHOP), where participants will be provided the study drug and monitored for safety.

For more information, please contact Sara Nguyen at nguyens2@email.chop.edu

REN001 for Adults with Primary Mitochondrial Myopathy

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.