Open-label Phase 3b metreleptin study in children <6 years with generalised lipodystrophy

Phase 3, open-label, single-arm trial of metreleptin in generalized lipoatrophy, type 2 diabetes mellitus (T2DM) and hypertriglyceridemia, sponsored by Amryt Pharma. Primary endpoints: Percent change from baseline in fasting serum triglyceride levels at Month 12 for subjects with fasting triglycerides ≥2.3 mmol/L at baseline and Absolute change from baseline in HbA1c at Month 12 for subjects with HbA1c ≥6.5% at baseline. Started Feb 2024; status: completed.