Open-label Study to Evaluate Metreleptin in Patients With Partial Lipodystrophy
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Metreleptin.
- Who it may be relevant to
- Registry conditions: Partial Lipodystrophy. Basic parameters: from 12 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- France, Germany, Italy, United Kingdom
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
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
Overview
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
Interventions
- 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
Primary outcome measures
- 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%. [Time frame: 12 months]
- 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) [Time frame: 12 months]
Secondary outcome measures (4)
- 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%. [Time frame: 24 months]
- 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). [Time frame: 24 months]
- Change from baseline in liver volume at Month 12 and Month 24 [Time frame: 12 months and 24 months]
- 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 [Time frame: 24 months]
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
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- N/A
- Model
- Single group
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
Italy · 5 centers
- Azienda Ospedaliero-Universitaria di Bologna Policlinico S. Orsola-Malpighi — Bologna
- Universitá degli Studi "Magna Graecia" di Catanzaro — Catanzaro
- Azienda Ospedaliero Universitaria Maggiore della Carità di Novara — Novara
- Azienda Ospedaliero Universitaria Pisana - Stabilimento di Cisanello — Pisa
- A.S.U Integrata di Udine - Presidio Ospedaliero Santa Maria della Misericordia — Udine
France · 4 centers
- Centre Hospitalier Régional Universitaire de Lille (CHRU) - Hôpital Claude Huriez — Lille
- Hopital Pitie-Salpetriere — Paris
- Hôpital Saint-Antoine — Paris
- Centre Hospitalier Lyon-Sud — Pierre-Bénite
Germany · 2 centers
- Charite-Universitaetsmedizin Berlin - Campus Charite Mitte (CCM) — Berlin
- Universitaetsklinikum Ulm - Klinik fuer Kinder- und Jugendmedizin — Ulm
United Kingdom · 1 center
- Cambridge University Hospitals NHS Foundation Trust - Addenbrookes Hospital — Cambridge
Identifiers
NCT: NCT06484868 · APL-22 · 2022-502950-14-00