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The purpose of this multi-center study is to determine if the drug REN001 can have a beneficial effect for patients with primary mitochondrial myopathy (PMM). PMMs are a group of rare, often life-threatening diseases caused by genetic mutations in the mitochondria. PMM can cause muscles to work less well and to be weaker, because mitochondria within cells are unable to produce the necessary energy needed by the body. Researchers believe that REN001 may be effective in increasing the number of healthy mitochondria and improving energy to help muscles work.
The study is accepting participants, ages 18 and older, who have been diagnosed through genetic testing with a mitochondrial disease with muscle weakness (myopathy) attributed to the m.3243A>G-related mitochondrial mutation or other mitochondrial DNA mutations.
Males and Females: Age 18 and older
Participants in the study will be randomly divided into groups that will take REN001 in a 100 mg dose or a placebo, once daily for 24 weeks. During study visits, participants will receive physical examinations, which will include blood and urine sampling, various physical movement and strength tests, eye tests, and questionnaires.
Visits: 8, of which 4 may be done at the patient’s home by a study nurse.
Duration: 36 weeks
Reneo Pharma Ltd.
Study Description at National Institutes of Health
Medical Genetics Research
Gerard Vockley, MD, PhD
For more information about the study or enrollment, please contact:
Clinical Research Coordinator Michele Graham, RN
Children's Hospital's main campus is located in the Lawrenceville neighborhood. Our main hospital address is:
UPMC Children’s Hospital of Pittsburgh
One Children’s Hospital Way
4401 Penn Ave.
Pittsburgh, PA 15224
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