Efficacy Assessments
tiredness, fatigue
SECONDARY, MITOCHONDRIAL MYOPATHY
Coprimary endpoints evaluated the effect of elamipretide for 24 weeks including the distance walked (in meters) on the 6MWT and the total fatigue score on the Primary Mitochondrial Myopathy Symptom Assessment (PMMSA), previously described in detail.Most bothersome symptom score on the PMMSA and the NeuroQoL Short-Form fatigue scores were secondary endpoints. The Neuro-QoL evaluates and monitors sensations ranging from tiredness to an overwhelming, debilitating, and sustained sense of exhaustion that decreases capacity for physical, functional, social, and mental activities, based on a 5-point scale (1 = never, 2 = rarely, 3 = sometimes, 4 = often, and 5 = always).Other secondary endpoints included the patient global impression (PGI) and clinician global impression (CGI) of PMM symptoms. PGI and CGI assess patient and clinician overall assessment of the severity of patients' symptoms related to PMM on a 5-point scaled question scored 0 to 4 (0 = none, 1 = mild, 2 = moderate, 3 = severe, and 4 = very Severe) and changes to their symptoms on a 7-point scale scored −3 to 3 (−3 = very much worse, −2 = moderately worse, −1 = a little worse, 0 = no change, 1 = a little better, 2 = moderately better, and 3 = very much better).The PMMSA was performed during screening, baseline, and daily throughout the 24-week study period. Other efficacy endpoints were performed at screening, baseline, and at weeks 4, 12, and 24 of randomized treatment.