Outcomes
shoulder abductors
GNE MYOPATHY, SECONDARY, ADVERSE EVENTS
Participants were subjected to the following efficacy assessments every 12 weeks, over 48 weeks. The primary endpoint was change in UEC score from baseline to Week 48. UEC score was calculated as the sum of bilateral average of strength values (in kilograms) for grip, shoulder abductors, elbow flexors, and elbow extensors. The strength of each muscle group was measured with a hand-held dynamometer.The key secondary endpoint was efficacy rate based on a comprehensive assessment made by the investigator. First, the investigator assessed the following four items for one of the following: improvement, no change, deterioration, or undecidable (not applicable). This was for: (1) MMT for upper extremity, or grip strength, (2) UEC score, (3) change in UEC score compared with the placebo-administered GNE myopathy patients in previous clinical studies, and (4) other secondary endpoints. Subsequently, the comprehensive efficacy in each patient was assessed as effective (improvement judged by evaluator), ineffective (no improvement judged by evaluator), or unable to determine (The evaluator could not determine whether the drug was effective or ineffective) on integrating the assessments (1)–(4). If any one of the four assessment methods was “effective”, the subject was judged effective. The proportion of subjects judged as effective was defined as the effective rate.Other secondary endpoints included the following changes from baseline to Week 48: GNEM-FAS upper extremity, mobility, and self-care scores; GNEM-FAS total score; bilateral average of individual muscle strength for grip, shoulder abductors, elbow flexors, and elbow extensors comprising UEC score; bilateral average of muscle strength for knee extensors.The safety assessments included the incidence and severity of adverse events, vital signs and weight, electrocardiogram, and clinical laboratory tests.