Endpoints and assessments
BBS
The primary endpoint was the proportion of patients aged ≥ 12 years who reached ≥ 10% reduction in body weight compared with baseline after 52 weeks of setmelanotide treatment [Self-reported HRQOL was measured in pediatric and adolescent patients (aged ≥ 6 to < 18 years) by patients or caregivers using age-specific Pediatric Quality of Life Inventory (PedsQL) assessments (child age range, 5–12 years; teen age range, 13–18 years) [This analysis focuses on the cohort of patients with BBS. Descriptive analyses were conducted on outcomes reported for patients with either PedsQL or IWQOL-Lite data both at active treatment baseline, defined as the last available measure before the first active setmelanotide dose, and after 52 weeks of setmelanotide treatment. Spearman correlations were used to evaluate associations between percent change in PedsQL or IWQOL-Lite and changes in body weight, BMI or BMI Z score, and hunger scores.