An Observational Study of Patients With Primary Mitochondrial Disease (SPIMM-300) (RePOWER)
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This is an observational study of patients with Primary Mitochondrial Disease with either signs or symptoms suggestive of myopathy. The Investigator will identify potential patients through existing medical records and one on-site visit.
Condition or disease
Primary Mitochondrial Disease
An observational study of patients with presumed Primary Mitochondrial Disease designed to better characterize and correlate symptoms and signs of myopathy and genetic test results and the use of commonly prescribed treatments. The study will help define and identify a subject population for a future trial of an investigational product to treat primary mitochondrial disease associated with signs and symptoms of myopathy.
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
Ages Eligible for Study:
16 Years to 80 Years (Child, Adult, Older Adult)
Sexes Eligible for Study:
Accepts Healthy Volunteers:
Patients with clinical presentation of PMD with either signs or symptoms suggestive of myopathy
Willing and able to provide a signed informed consent form (ICF) prior to participation in any-trial related procedures
Patient has clinical presentation of PMD with either signs or symptoms suggestive of myopathy
Patient is ambulatory and able to attempt 6MWT
Patient has symptoms of PMD due to secondary mitochondrial dysfunction
Patient has had prior exposure to elamipretide
Patient does not have the cognitive capacity to understand and complete all study assessments
Patient has a medical history of severe renal impairment
History of active alcoholism or drug addiction during the year before enrollment