Open-label Study to Evaluate Metreleptin in Children Under 6 Years of Age With Generalised Lipodystrophy

January 16, 2026 updated by: Amryt Pharma

Open-label, Phase 3b Study to Evaluate Effectiveness, Safety and Pharmacokinetic Parameters of Metreleptin in Patients Under 6 Years of Age With Generalised Lipodystrophy and Associated Diabetes Mellitus and/or Hypertriglyceridaemia

This is an open-label, Phase 3b study to evaluate effectiveness, safety and pharmacokinetic parameters of metreleptin in patients under 6 years of age with generalised lipodystrophy and associated diabetes mellitus and/or hypertriglyceridaemia

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

15

Phase

  • Phase 3

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

  • Belgium
      • Leuven, Belgium, 3000
        • Recruiting
        • UZ Leuven
        • Contact:
          • Kristina Casteels
  • France
      • Paris, France, 75015
        • Recruiting
        • Hôpital Necker - Enfants Malades
        • Contact:
          • Jacques BELTRAND
      • Paris, France
        • Recruiting
        • Assistance Publique-Hopitaux de Paris (AP-HP) - Hopital Universitaire Robert-Debre
        • Contact:
          • Elise Bismuth
  • Germany
      • Hamburg, Germany, 20251
        • Not yet recruiting
        • Universitatsklinikum Hamburg-Eppendorf
        • Contact:
          • Konstantinos Tsiakas
      • Ulm, Germany, 89075
        • Recruiting
        • Universitaetsklinikum Ulm - Klinik fuer Kinder- und Jugendmedizin
        • Contact:
          • Martin Wabitsch
  • Italy
      • Chieti, Italy, 66100
        • Recruiting
        • IRCCS Ospedale Pediatrico Bambino Gesù
        • Contact:
          • Stefano Tumini
      • Naples, Italy, 80131
        • Recruiting
        • Azienda Ospedaliera Universitaria "Federico II"
        • Contact:
          • Enza Mozzillo
      • Novara, Italy, 28100
        • Recruiting
        • Azienda Ospedaliero Universitaria Maggiore della Carità di Novara
        • Contact:
          • Flavia Prodam
      • Parma, Italy, 43126
        • Recruiting
        • Azienda Ospedaliero-Universitaria di Parma
        • Contact:
          • Maria Street
      • Pisa, Italy, 56124
        • Recruiting
        • Azienda Ospedaliero Universitaria Pisana
        • Contact:
          • Giovanni Ceccarini
      • Rome, Italy, 00165
        • Recruiting
        • IRCCS Ospedale Pediatrico Bambino Gesù
        • Contact:
          • Marco Cappa
      • Varese, Italy, 21100
        • Recruiting
        • Ospedale Filippo Del Ponte Varese - ASST Sette Laghi
        • Contact:
          • Gianluca Musolino

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

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • Confirmed diagnosis of Generalised Lipodystrophy
  • Metreleptin treatment naive

Exclusion Criteria:

  • Weight <9 kg at Screening (Visit 1)

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; rmetHuLeptin] 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
Percent change from baseline in fasting serum TG levels at Month 12 for subjects with fasting TG levels ≥2.3 mmol/L (200 mg/dL) at baseline
Time Frame: 12 months
To evaluate the effectiveness of metreleptin in patients under 6 years of age with GL and associated diabetes mellitus and/or hypertriglyceridaemia
12 months
Absolute change from baseline in glycated haemoglobin (HbA1c) at Month 12 for subjects with HbA1c ≥6.5% at baseline
Time Frame: 12 months
To evaluate the effectiveness of metreleptin in patients under 6 years of age with GL and associated diabetes mellitus and/or hypertriglyceridaemia
12 months

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Proportion of subjects of those with Baseline HbA1c ≥6.5% achieving target actual decreases of at least 0.5%, 1%, 1.5%, 2% absolute decrease in HbA1c or HbA1c <6.5% or HbA1c <5.7% at Month 12
Time Frame: 12 months
To evaluate the efficacy of metreleptin treatment in patients with GL
12 months
Proportion of subjects of those with Baseline HbA1c ≥5.7% achieving target actual decreases of at least 0.5%, 1%, 1.5%, 2% absolute decrease in HbA1c or HbA1c <5.7% at Month 12
Time Frame: 12 months
To evaluate the efficacy of metreleptin treatment in patients with GL
12 months
Proportion of subjects of those with fasting serum TG ≥ 1.7 mmol/L (150 mg/dL) achieving target actual decreases from baseline of at least 15%, 20%, 25%, 30%, 35%, 40% at Month 12
Time Frame: 12 months
To evaluate the efficacy of metreleptin treatment in patients with GL
12 months
Proportion of subjects of those with fasting serum TG ≥ 2.3 mmol/L (200 mg/dL) achieving target actual decreases from baseline of at least 15%, 20%, 25%, 30%, 35%, 40% at Month 12
Time Frame: 12 months
To evaluate the efficacy of metreleptin treatment in patients with GL
12 months
Change from baseline in liver volume and liver span as assessed by ultrasound at each post-baseline visit through Month 12
Time Frame: 12 months
To evaluate the efficacy of metreleptin treatment in patients with GL
12 months
Incidence of, treatment emergent adverse events (TEAEs), serious adverse events (SAEs), treatment related adverse events (AEs), AEs of special interest (AESIs) and AEs leading to study drug discontinuation
Time Frame: 12 months
To assess safety and tolerability of metreleptin
12 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)

May 23, 2025

Primary Completion (Estimated)

September 30, 2026

Study Completion (Estimated)

September 30, 2026

Study Registration Dates

First Submitted

July 9, 2024

First Submitted That Met QC Criteria

July 9, 2024

First Posted (Actual)

July 16, 2024

Study Record Updates

Last Update Posted (Actual)

January 20, 2026

Last Update Submitted That Met QC Criteria

January 16, 2026

Last Verified

January 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • APL-20
  • 2022-501781-22-00 (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

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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