Open-label Study to Evaluate Metreleptin in Children Under 6 Years of Age With Generalised Lipodystrophy
- Registration Number
- NCT06502990
- Lead Sponsor
- Amryt Pharma
- Brief 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
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 15
- Confirmed diagnosis of Generalised Lipodystrophy
- Metreleptin treatment naive
- Weight <9 kg at Screening (Visit 1)
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Metreleptin Metreleptin Metreleptin \[Recombinant-methionyl human Leptin; rmetHuLeptin\] for daily injection is a sterile, white, solid lyophilised cake
- Primary Outcome Measures
Name Time Method 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 12 months To evaluate the effectiveness of metreleptin in patients under 6 years of age with GL and associated diabetes mellitus and/or hypertriglyceridaemia
Absolute change from baseline in glycated haemoglobin (HbA1c) at Month 12 for subjects with HbA1c ≥6.5% at baseline 12 months To evaluate the effectiveness of metreleptin in patients under 6 years of age with GL and associated diabetes mellitus and/or hypertriglyceridaemia
- Secondary Outcome Measures
Name Time Method 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 12 months To evaluate the efficacy of metreleptin treatment in patients with GL
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 12 months To evaluate the efficacy of metreleptin treatment in patients with GL
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 12 months To evaluate the efficacy of metreleptin treatment in patients with GL
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 12 months To evaluate the efficacy of metreleptin treatment in patients with GL
Change from baseline in liver volume and liver span as assessed by ultrasound at each post-baseline visit through Month 12 12 months To evaluate the efficacy of metreleptin treatment in patients with GL
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 12 months To assess safety and tolerability of metreleptin
Trial Locations
- Locations (10)
Azienda Ospedaliera Universitaria "Federico II"
🇮🇹Naples, Italy
Hôpital Necker - Enfants Malades
🇫🇷Paris, France
Universitätsklinikum Hamburg-Eppendorf
🇩🇪Hamburg, Germany
Universitaetsklinikum Ulm - Klinik fuer Kinder- und Jugendmedizin
🇩🇪Ulm, Germany
IRCCS Ospedale Pediatrico Bambino Gesù
🇮🇹Rome, Italy
Azienda Ospedaliero-Universitaria di Parma
🇮🇹Parma, Italy
Ospedale Filippo Del Ponte Varese - ASST Sette Laghi
🇮🇹Varese, Italy
Azienda Ospedaliero Universitaria Maggiore della Carità di Novara
🇮🇹Novara, Italy
UZ Leuven
🇧🇪Leuven, Belgium
Azienda Ospedaliero Universitaria Pisana
🇮🇹Pisa, Italy