Clinical trial NCT06484868: Open-label Study to Evaluate Metreleptin in Patients With Partial Lipodystrophy
Official title: A 24-Month, Multi-Centre, Open Label Phase IV Post Authorisation Efficacy Study to Evaluate the Efficacy, Safety and Immunogenicity of Daily Subcutaneous Metreleptin Treatment in Patients With Partial Lipodystrophy
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE4
Conditions: Partial Lipodystrophy
Keywords: Metreleptin
Lead sponsor: Amryt Pharma
Sponsor class: INDUSTRY
First posted: 2024-07-03
Enrollment: 12
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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


Eligibility criteria: 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

Primary outcomes:
measure: 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%.
timeFrame: 12 months
measure: 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)
timeFrame: 12 months
Secondary outcomes:
measure: 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%.
timeFrame: 24 months
measure: 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).
timeFrame: 24 months
measure: Change from baseline in liver volume at Month 12 and Month 24
timeFrame: 12 months and 24 months
measure: 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
timeFrame: 24 months
Other outcomes:
Locations:
city: Lille
country: France
facility: Centre Hospitalier Régional Universitaire de Lille (CHRU) - Hôpital Claude Huriez
city: Paris
country: France
facility: Hopital Pitie-Salpetriere
city: Paris
country: France
facility: Hôpital Saint-Antoine
city: Pierre-Bénite
country: France
facility: Centre Hospitalier Lyon-Sud
city: Berlin
country: Germany
facility: Charite-Universitaetsmedizin Berlin - Campus Charite Mitte (CCM)
city: Ulm
country: Germany
facility: Universitaetsklinikum Ulm - Klinik fuer Kinder- und Jugendmedizin
city: Bologna
country: Italy
facility: Azienda Ospedaliero-Universitaria di Bologna Policlinico S. Orsola-Malpighi
city: Catanzaro
country: Italy
facility: Universitá degli Studi "Magna Graecia" di Catanzaro
city: Novara
country: Italy
facility: Azienda Ospedaliero Universitaria Maggiore della Carità di Novara
city: Pisa
country: Italy
facility: Azienda Ospedaliero Universitaria Pisana - Stabilimento di Cisanello
city: Udine
country: Italy
facility: A.S.U Integrata di Udine - Presidio Ospedaliero Santa Maria della Misericordia
city: Cambridge
country: United Kingdom
facility: Cambridge University Hospitals NHS Foundation Trust - Addenbrookes Hospital
Study officials:
References: