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

July 2, 2025 updated by: Amryt Pharma

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

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

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

12

Phase

  • Phase 4

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • France
      • Lille, France
        • Recruiting
        • Centre Hospitalier Régional Universitaire de Lille (CHRU) - Hôpital Claude Huriez
        • Contact:
          • Marie-Christine Vantyghem
      • Paris, France
        • Recruiting
        • Hôpital Saint-Antoine
        • Contact:
          • Corinne Vigouroux
      • Paris, France
        • Recruiting
        • Hôpital Pitié-Salpêtrière
        • Contact:
          • Fabrizio Andreelli
      • Pierre-Bénite, France
        • Recruiting
        • Centre Hospitalier Lyon-Sud
        • Contact:
          • Emmanuel Disse
  • Germany
      • Berlin, Germany
        • Recruiting
        • Charite-Universitaetsmedizin Berlin - Campus Charite Mitte (CCM)
        • Contact:
          • Thomas Bobbert
      • Ulm, Germany
        • Not yet recruiting
        • Universitaetsklinikum Ulm - Klinik fuer Kinder- und Jugendmedizin
        • Contact:
          • Martin Wabitsch
  • Italy
      • Bologna, Italy
        • Recruiting
        • Azienda Ospedaliero-Universitaria di Bologna Policlinico S. Orsola-Malpighi
        • Contact:
          • Alessandra Gambineri
      • Catanzaro, Italy
        • Not yet recruiting
        • Universitá degli Studi "Magna Graecia" di Catanzaro
        • Contact:
          • Antonio Aversa
      • Novara, Italy
        • Recruiting
        • Azienda Ospedaliero Universitaria Maggiore della Carità di Novara
        • Contact:
          • Flavia Prodam
      • Pisa, Italy
        • Recruiting
        • Azienda Ospedaliero Universitaria Pisana - Stabilimento di Cisanello
        • Contact:
          • Giovanni Ceccarini
      • Udine, Italy
        • Recruiting
        • A.S.U Integrata di Udine - Presidio Ospedaliero Santa Maria della Misericordia
        • Contact:
          • Annalisa Sechi
  • United Kingdom
      • Cambridge, United Kingdom
        • Not yet recruiting
        • Cambridge University Hospitals NHS Foundation Trust - Addenbrookes Hospital
        • Contact:
          • Anna Stears

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Metreleptin
Metreleptin [Recombinant-methionyl human Leptin; r-metHuLeptin] for daily injection is a sterile, white, solid lyophilised cake
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

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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
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
To evaluate the efficacy (TG) of metreleptin treatment in patients with PL
12 months

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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
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
To evaluate the long-term efficacy of metreleptin treatment in patients with PL
24 months
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.
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
To evaluate the safety of metreleptin treatment in patients with PL
24 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Amryt Pharma

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

February 8, 2024

Primary Completion (Estimated)

February 1, 2028

Study Completion (Estimated)

February 1, 2028

Study Registration Dates

First Submitted

June 26, 2024

First Submitted That Met QC Criteria

June 26, 2024

First Posted (Actual)

July 3, 2024

Study Record Updates

Last Update Posted (Actual)

July 3, 2025

Last Update Submitted That Met QC Criteria

July 2, 2025

Last Verified

July 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • APL-22
  • 2022-502950-14-00 (Ctis)

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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