Mitochondrial Medicine Clinical Studies

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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 fatiguescales, Mitochondrial Disease scales, and quality of life scales, 2) validate clinicalexams, such as muscle strength tests, and the 6 minute walk test, and 3) to define thenatural history of the disease in the Mitochondrial Disease patient population.

Cyclerion 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 4 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