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Children's Hospital is ranked One of America's Best Children's Hospitals.
The purpose of this multi-center study is to evaluate the effects of RTA 408 (omaveloxolone), taken in pill form, for treatment of mitochondrial myopathy. Mitochondrial diseases are rare and occur when one or more genetic mutations result in mitochondria failing to create enough energy to support the body. Mitochondrial myopathy is a consequence of those mitochondrial diseases that cause muscular problems.
Subject to certain exclusion criteria, the study accepts male and female patients, ages 18 to 75, who have a genetically confirmed mitochondrial myopathy. Genetic testing can be performed as part of the screening visit, if not already completed.
Males and Females: Ages 18 to 75
Following an initial screening visit, which will include a physical exam and various physical tests, participants enrolled in the study will be randomly chosen to receive either the study drug or a placebo to be taken for 12 weeks. Study drug dosage levels for participant groups are pre-determined by study parameters. Over the course of their enrollment, participants will have a total of 12 visits, most of which will include physical exams and blood and urine tests. Two visits will be conducted via telephone to discuss health, and questions about side effects and the medication.
Duration: about 20 weeks
Study Description at National Institutes of Health
Medical Genetics Research
Gerard Vockley, MD, PhD
For more information about the study or enrollment, please contact:
Michele Graham, RN
Children's Hospital's main campus is located in the Lawrenceville neighborhood. Our main hospital address is:
Children’s Hospital of Pittsburgh of UPMC
One Children’s Hospital Way
4401 Penn Ave.
Pittsburgh, PA 15224
In addition to the main hospital, Children's has many convenient locations in other neighborhoods throughout the greater Pittsburgh region.
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Interested in giving to Children's Hospital? Visit Children's Hospital of Pittsburgh Foundation's website to make a donation online.