NCT06502990

Open-label Study to Evaluate Metreleptin in Children Under 6 Years of Age With Generalised Lipodystrophy

Open-label, Phase 3b Study to Evaluate Effectiveness, Safety and Pharmacokinetic Parameters of Metreleptin in Patients Under 6 Years of Age With Generalised Lipodystrophy and Associated Diabetes Mellitus and/or Hypertriglyceridaemia

• Status: Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 15 (estimated)
• Sponsor: Amryt Pharma · Industry
• Sex: All
• Age: 5 Years
• Healthy volunteers: Not accepted

Summary

This is an open-label, Phase 3b study to evaluate effectiveness, safety and pharmacokinetic parameters of metreleptin in patients under 6 years of age with generalised lipodystrophy and associated diabetes mellitus and/or hypertriglyceridaemia

Conditions

• Generalized Lipodystrophy

Interventions

Type	Name	Description
DRUG	Metreleptin	Metreleptin is a recombinant human leptin analog that is indicated as an adjunct to diet as replacement therapy to treat the complications of leptin deficiency

Timeline

• Start date: 2025-05-23
• Primary completion: 2026-09-30
• Completion: 2026-09-30
• First posted: 2024-07-16
• Last updated: 2026-01-20

Locations

12 sites across 4 countries: Belgium, France, Germany, Italy