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
Open-label Study to Evaluate Metreleptin in Patients With Partial Lipodystrophy
Status
Recruiting
Phase
Phase 4
Enrollment
12
Locations
12
Results
Not posted
Publications
0
Study summary
What the protocol is testing.
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
Treatment arms and agents.
Timeline
From registration to results.
First posted
Jul 3, 2024
Study start
Feb 8, 2024
Primary completion
Feb 2028
Study completion
Feb 2028
Results posted
Not reported
Registry updated
Jul 3, 2025
Outcomes
What the study measures.
Primary outcomes
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
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)
Time frame · 12 months
To evaluate the efficacy (TG) of metreleptin treatment in patients with PL
Secondary outcomes
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
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).
Time frame · 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
Time frame · 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
Time frame · 24 months
To evaluate the safety of metreleptin treatment in patients with PL
Eligibility
Who can take part.
- Minimum age
- 12 Years
- Maximum age
- Not reported
- Sex
- ALL
- Healthy volunteers
- No
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 locations
12 registered sites.
France · Germany · Italy · United Kingdom. Showing up to 24 locations stored in the fast local snapshot.
Centre Hospitalier Régional Universitaire de Lille (CHRU) - Hôpital Claude Huriez
Lille, France
Hopital Pitie-Salpetriere
Paris, France
Hôpital Saint-Antoine
Paris, France
Centre Hospitalier Lyon-Sud
Pierre-Bénite, France
Charite-Universitaetsmedizin Berlin - Campus Charite Mitte (CCM)
Berlin, Germany
Universitaetsklinikum Ulm - Klinik fuer Kinder- und Jugendmedizin
Ulm, Germany
Azienda Ospedaliero-Universitaria di Bologna Policlinico S. Orsola-Malpighi
Bologna, Italy
Universitá degli Studi "Magna Graecia" di Catanzaro
Catanzaro, Italy
Azienda Ospedaliero Universitaria Maggiore della Carità di Novara
Novara, Italy
Azienda Ospedaliero Universitaria Pisana - Stabilimento di Cisanello
Pisa, Italy
A.S.U Integrata di Udine - Presidio Ospedaliero Santa Maria della Misericordia
Udine, Italy
Cambridge University Hospitals NHS Foundation Trust - Addenbrookes Hospital
Cambridge, United Kingdom
Publications
Results and literature.
No PMID-linked publications were present in this registry snapshot.
Related trials
More studies on Metreleptin.
NCT IDStudyPhase / statusEnrollmentUpdated
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50
2026-07
A Study of the Safety of Mibavademab in Pediatric and Adult Participants Switching From Metreleptin to Mibavademab for the Treatment of Generalized Lipodystrophy (GLD)
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Phase 3
Completed
9
2026-07
Compassionate Use of Metreleptin in Previously Treated People With Partial Lipodystrophy
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) · Diabetes · Lipodystrophy
Phase 2
Active, not recruiting
29
2026-07
Open-label Study to Evaluate Metreleptin in Children Under 6 Years of Age With Generalised Lipodystrophy
Amryt Pharma · Generalized Lipodystrophy
Phase 3
Terminated
1
2026-07
Compassionate Use of Metreleptin in Previously Treated People With Generalized Lipodystrophy
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) · Lipodystrophy · Diabetes
Phase 3
Active, not recruiting
24
2026-07
MEASuRE: Metreleptin Effectiveness And Safety Registry
Chiesi Farmaceutici S.p.A. · Generalised Lipodystrophy · Partial Lipodystrophy
Not applicable
Recruiting
100
2026-07
Open-label Extension Study to Evaluate Metreleptin in Patients With Partial Lipodystrophy
Chiesi Farmaceutici S.p.A. · Familial Partial Lipodystrophy
Phase 3
Recruiting
24
2026-07
Study to Evaluate the Safety and Efficacy of Daily Subcutaneous Metreleptin Treatment in Subjects With PL
Chiesi Farmaceutici S.p.A. · Partial Lipodystrophy
Phase 3
Active, not recruiting
69
2026-06
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Put the record in context.
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