Study Design and Participants
myopathy, end-organ damage, fatigue, 7–28
MITOCHONDRIAL DISEASE, MYOPATHY, MUSCLE WEAKNESS
MMPOWER-3 was a 24-week , randomized, double-blind, parallel-group, placebo-controlled clinical trial for adults with PMM conducted at 27 clinical research centers in 7 countries (Canada, Denmark, England, Germany, Hungary, Italy, and the United States).MMPOWER-3 trial participants were primarily identified by the RePOWER registry, a global, prospective, noninterventional registry enrolling 413 ambulatory individuals 16–80 years of age with signs and/or symptoms of PMM.After screening (7–28 days), eligible participants were randomized in a 1:1 ratio to receive either 24-weeks of once-daily SC dosing of 40 mg of elamipretide or placebo. Study drug or placebo was self-administered subcutaneously by trained participants or their caregivers, at rotating sites around 4 quadrants of the abdomen or the thighs. Treatment began at the baseline visit with assessments at weeks 4, 12, and 24.Eligible participants were those between 16 years or older and 80 years or younger (18 years or older in Germany), diagnosed with PMM with a confirmed sequence alteration affecting mitochondrial function, and those with symptoms (i.e., exercise intolerance, fatigue, muscle weakness) and/or physical examination findings consistent with a myopathy as the predominant manifestation of their mitochondrial disease. In addition, participants had to be willing and able to provide consent and adhere to trial requirements for inclusion. An acceptable form of birth control was required of participants of childbearing potential during the study.Participants walking <100 meters or >450 meters during the 6MWT at screening/baseline were also excluded. Participants were not allowed to have had a recent (within 30 days) or planned hospitalization/procedure and were excluded if they had a clinically significant end-organ damage in the opinion of the investigator.