Investigational agent in Mitochondrial Respiratory Chain Disease End-of-Life Care

Mitochondria are found in nearly every cell in the human body. The main function of mitochondria is to convert the energy of food sources into energy that the body’s cells can use. A problem or defect with the mitochondria may lead to a wide variety of diseases. This study is looking at the use of an investigational agent (EPI-743) in patients with diseases caused by mitochondrial dysfunction known as mitochondrial respiratory chain diseases.

Medication Under Study:
EPI-743 (investigational drug)

Full study title:
Emergency Use Protocol for EPI-743 in Acutely Ill Patients with Inherited Mitochondrial Respiratory Chain Disease Within 90 Days of End-of-Life Care

IRB Study ID:

Who may qualify:

Patients with mitochondiral respiratory chain diseases within 90 days end-of-life care

Who does not qualify:

Patients with an allergy to sesame oil or certain medical conditions such as abnormal liver or kidney function

If you are interested in this study or have questions about your child's eligibility, please contact:

Rebecca D. Considine Research Institute, 330-543-5012

For urgent patient care matters involving a current study patient, please call: 330-920-5774

Lead Principal Investigator:

Bruce H. Cohen, MD, FAAN
Director, Pediatric Neurology

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