Open-label Study to Evaluate Metreleptin in Children Under 6 Years of Age With Generalised Lipodystrophy
NCT Number: NCT06502990
Status: RECRUITING
Study Type: INTERVENTIONAL
Phases: PHASE3
Conditions: Generalized Lipodystrophy
Interventions: DRUG: Metreleptin
Sponsor: Amryt Pharma
Sex: ALL
Age: CHILD
Enrollment: 15
Study Design: Allocation: NA|Intervention Model: SINGLE_GROUP|Masking: NONE|Primary Purpose: TREATMENT
Start Date: 2024-07-31
Completion Date: 2026-09-30
Locations: UZ Leuven, Leuven, 3000, Belgium|Hôpital Necker - Enfants Malades, Paris, 75015, France|Universitätsklinikum Hamburg-Eppendorf, Hamburg, 20251, Germany|Universitaetsklinikum Ulm - Klinik fuer Kinder- und Jugendmedizin, Ulm, 89075, Germany|IRCCS Ospedale Pediatrico Bambino Gesù, Chieti, 66100, Italy|Azienda Ospedaliera Universitaria "Federico II", Naples, 80131, Italy|Azienda Ospedaliero Universitaria Maggiore della Carità di Novara, Novara, 28100, Italy|Azienda Ospedaliero-Universitaria di Parma, Parma, 43126, Italy|Azienda Ospedaliero Universitaria Pisana, Pisa, 56124, Italy|IRCCS Ospedale Pediatrico Bambino Gesù, Rome, 00165, Italy|Ospedale Filippo Del Ponte Varese - ASST Sette Laghi, Varese, 21100, Italy

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

Primary Outcome Measures: Percent change from baseline in fasting serum TG levels at Month 12 for subjects with fasting TG levels ≥2.3 mmol/L (200 mg/dL) at baseline, To evaluate the effectiveness of metreleptin in patients under 6 years of age with GL and associated diabetes mellitus and/or hypertriglyceridaemia, 12 months|Absolute change from baseline in glycated haemoglobin (HbA1c) at Month 12 for subjects with HbA1c ≥6.5% at baseline, To evaluate the effectiveness of metreleptin in patients under 6 years of age with GL and associated diabetes mellitus and/or hypertriglyceridaemia, 12 months
Secondary Outcome Measures: Proportion of subjects of those with Baseline HbA1c ≥6.5% achieving target actual decreases of at least 0.5%, 1%, 1.5%, 2% absolute decrease in HbA1c or HbA1c <6.5% or HbA1c <5.7% at Month 12, To evaluate the efficacy of metreleptin treatment in patients with GL, 12 months|Proportion of subjects of those with Baseline HbA1c ≥5.7% achieving target actual decreases of at least 0.5%, 1%, 1.5%, 2% absolute decrease in HbA1c or HbA1c <5.7% at Month 12, To evaluate the efficacy of metreleptin treatment in patients with GL, 12 months|Proportion of subjects of those with fasting serum TG ≥ 1.7 mmol/L (150 mg/dL) achieving target actual decreases from baseline of at least 15%, 20%, 25%, 30%, 35%, 40% at Month 12, To evaluate the efficacy of metreleptin treatment in patients with GL, 12 months|Proportion of subjects of those with fasting serum TG ≥ 2.3 mmol/L (200 mg/dL) achieving target actual decreases from baseline of at least 15%, 20%, 25%, 30%, 35%, 40% at Month 12, To evaluate the efficacy of metreleptin treatment in patients with GL, 12 months|Change from baseline in liver volume and liver span as assessed by ultrasound at each post-baseline visit through Month 12, To evaluate the efficacy of metreleptin treatment in patients with GL, 12 months|Incidence of, treatment emergent adverse events (TEAEs), serious adverse events (SAEs), treatment related adverse events (AEs), AEs of special interest (AESIs) and AEs leading to study drug discontinuation, To assess safety and tolerability of metreleptin, 12 months