Open-label Study to Evaluate Metreleptin in Patients With Partial Lipodystrophy

Phase 4

Recruiting

• Conditions: Partial Lipodystrophy
• Interventions: Drug: Metreleptin

• Registration Number: NCT06484868
• Lead Sponsor: Amryt Pharma

Brief Summary

This is an Open Label, Phase IV, Post Authorisation Study to Evaluate the Efficacy, Safety and Immunogenicity of Daily Subcutaneous Metreleptin Treatment in people with Partial Lipodystrophy

Detailed Description

Not available

Study Timeline

• Study Start: 2024-02-08
• Study First Submitted: 2024-06-26
• Study First Submitted QC: 2024-06-26
• Study First Posted: 2024-07-03
• Status Verified: 2024-12
• Last Update Submitted: 2024-12-23
• Last Update Posted: 2024-12-27
• Primary Completion: 2027-09
• Study Completion: 2027-09

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 12

Inclusion Criteria

• Confirmed diagnosis of familial or acquired partial lipodystrophy

Exclusion Criteria

• Treatment with any Investigational Medicinal Product (IMP) within 6 months or 5 times the terminal half-life of the corresponding IMP, whichever is longer, before the screening visit.

Other protocol defined inclusion/exclusion criteria apply

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Metreleptin	Metreleptin	Metreleptin \[Recombinant-methionyl human Leptin; r-metHuLeptin\] for daily injection is a sterile, white, solid lyophilised cake

Primary Outcome Measures

Name	Time	Method
Number of patients with decrease of at least 0.5% in glycated haemoglobin (HbA1c) at Month 12 compared to Baseline or HbA1c <6.5 % at Month 12, in patients with baseline HbA1c ≥6.5%.	12 months	To evaluate the efficacy (HbA1c) of metreleptin treatment in patients with PL
Number of patients with decrease of at least 30% in triglycerides (TG) at Month 12 compared to Baseline, in patients with baseline TG levels ≥500 mg/dL (5.65 mmol/L)	12 months	To evaluate the efficacy (TG) of metreleptin treatment in patients with PL

Secondary Outcome Measures

Name	Time	Method
Number of patients with decrease of at least 0.5% in HbA1c at Month 24 compared to Baseline or HbA1c <6.5 % at Month 24, in patients with baseline HbA1c ≥6.5%.	24 months	To evaluate the long-term efficacy of metreleptin treatment in patients with PL
Number of patients with decrease of at least 30% in TG levels at Month 24 compared to Baseline, in patients with baseline TG levels ≥500 mg/dL (5.65 mmol/L).	24 months	To evaluate the long-term efficacy of metreleptin treatment in patients with PL
Change from baseline in liver volume at Month 12 and Month 24	12 months and 24 months	To assess changes in liver volume.
Incidence of, Treatment emergent adverse events (TEAEs), Deaths and other serious adverse events (SAEs), Treatment related adverse events (AEs), AEs of special interest (AESIs) and AEs leading to study drug discontinuation	24 months	To evaluate the safety of metreleptin treatment in patients with PL

Trial Locations

Locations (12)

Azienda Ospedaliero Universitaria Pisana - Stabilimento di Cisanello; 🇮🇹 Pisa, Italy

Hopital Pitie-Salpetriere; 🇫🇷 Paris, France

Azienda Ospedaliero Universitaria Maggiore della Carità di Novara; 🇮🇹 Novara, Italy

Hôpital Saint-Antoine; 🇫🇷 Paris, France

A.S.U Integrata di Udine - Presidio Ospedaliero Santa Maria della Misericordia; 🇮🇹 Udine, Italy

Charite-Universitaetsmedizin Berlin - Campus Charite Mitte (CCM); 🇩🇪 Berlin, Germany

Azienda Ospedaliero-Universitaria di Bologna Policlinico S. Orsola-Malpighi; 🇮🇹 Bologna, Italy

Centre Hospitalier Lyon-Sud; 🇫🇷 Pierre-Bénite, France

Cambridge University Hospitals NHS Foundation Trust - Addenbrookes Hospital; 🇬🇧 Cambridge, United Kingdom

Universitá degli Studi "Magna Graecia" di Catanzaro; 🇮🇹 Catanzaro, Italy

Universitaetsklinikum Ulm - Klinik fuer Kinder- und Jugendmedizin; 🇩🇪 Ulm, Germany

Centre Hospitalier Régional Universitaire de Lille (CHRU) - Hôpital Claude Huriez; 🇫🇷 Lille, France