Study to Evaluate the Safety and Efficacy of Daily Subcutaneous Metreleptin Treatment in Subjects With PL (METRE-PL)

August 25, 2025 updated by: Amryt Pharma

A 12-Month Randomized, Multicenter, Double-Blind, Placebo-Controlled Phase 3 Study to Evaluate the Safety and Efficacy of Daily Subcutaneous Metreleptin Treatment in Subjects With Partial Lipodystrophy

This is a Phase III, double-blind, placebo-controlled, safety and efficacy study of daily SC metreleptin in subjects with Partial Lipodystrophy.

Study Overview

Status

Active, not recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

69

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 Locations

  • Belgium
      • Leuven, Belgium
        • UZ Leuven
  • Brazil
      • Fortaleza, Brazil
        • Universitário Walter Cantídio
      • Porto Alegre, Brazil
        • Insight Centro de Pesquisas
      • Ribeirão Preto, Brazil
        • Hospital Das Clínicas Fmrp-Usp
      • Rio de Janeiro, Brazil
        • Instituto Estadual de Diabetes e Endocrinologia (IEDE-RJ)
  • Canada
    • Nova Scotia
      • Halifax, Nova Scotia, Canada, B3H 2Y9
        • Nova Scotia Health
      • Sydney, Nova Scotia, Canada, B1M 0A1
        • Care Access Clinic
    • Ontario
      • Hamilton, Ontario, Canada, L8N 3Z5
        • Hamilton General Hospital- McMaster University
      • London, Ontario, Canada, N6A 5B7
        • London Health Science Centre
    • Quebec
      • Chicoutimi, Quebec, Canada, G7H 7K9
        • Ecogene-21
      • Québec, Quebec, Canada, G1V4W2
        • Faculty of Medicine, Universite Laval
  • Chile
      • Santiago, Chile
        • Universidad Catolica
  • Israel
      • Jerusalem, Israel
        • Hadassah University Hospital - Ein Kerem
  • Netherlands
      • Leiden, Netherlands
        • Leids Universitair Medisch Centrum
      • Nijmegen, Netherlands
        • Radboud University Medical Centre Interne Geneeskund
  • Poland
      • Katowice, Poland
        • Uniwersyteckie Centrum Kliniczne
      • Krakow, Poland
        • SPZOZ Szpital Uniwersytecki w Krakowie
  • Spain
      • Santiago de Compostela, Spain
        • University Clinical Hospital of Santiago de Compostela/Hospital Universitario
  • United States
    • Alabama
      • Birmingham, Alabama, United States, 35233
        • University of Alabama
    • California
      • Sacramento, California, United States, 95817
        • UC Davis
    • Florida
      • Boca Raton, Florida, United States, 33431
        • Flourish Boca Raton
    • Georgia
      • Atlanta, Georgia, United States, 30329
        • Emory University
    • Massachusetts
      • Boston, Massachusetts, United States, 02114
        • Massachusetts General Hospital
    • Michigan
      • Ann Arbor, Michigan, United States, 48109
        • University of Michigan
    • Minnesota
      • Rochester, Minnesota, United States, 55902
        • Mayo Clinic
    • Missouri
      • Columbia, Missouri, United States, 65212
        • University of Missouri
    • Ohio
      • Columbus, Ohio, United States, 43210
        • The Ohio State University Wexner Medical Center
      • Columbus, Ohio, United States, 43201
        • Amryt Research Site, Endocrinology Research Associates Inc
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • The Children's Hospital of Philadelphia
    • Texas
      • Dallas, Texas, United States, 75390
        • UT southwestern Medical Center
    • Wisconsin
      • Milwaukee, Wisconsin, United States, 53226
        • Medical College of Wisconsin

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

8 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • Diagnosis of Familial Partial Lipodystrophy (FPLD)
  • Subjects with poor metabolic control defined as:

HbA1c ≥7% (at Visit 1 and Visit 3) and/or Fasting TGs ≥500 mg/dL (5.65 mmol/L, at Visit 1 and Visit 3)

  • Patients should be receiving optimized stable therapy

Exclusion Criteria:

  • Previous treatment with metreleptin
  • Leptin levels >20.0 ng/mL
  • Acquired or radiation induced partial lipodystrophy (APL)

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: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Active Comparator: 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
Placebo Comparator: placebo
Placebo for daily injection is a sterile, white, solid lyophilised cake
Drug: Placebo
Placebo

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change from baseline to month 6 in HbA1c in subjects with partial lipodystrophy (PL)
Time Frame: 6 months
To evaluate the efficacy (HbAa1c) of daily SC metreleptin treatment
6 months
Percent change from Baseline to month 6 in fasting TGs in subjects with partial lipodystrophy (PL)
Time Frame: 6 months
To evaluate the efficacy (TGs) of daily SC metreleptin treatment
6 months

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Safety analysis of AEs, AESIs, SAEs by treatment arm
Time Frame: 12 months
To evaluate the safety of daily SC metreleptin treatment in subjects with PL
12 months
Change from baseline to months 9 and 12 in HbA1c in subjects with partial lipodystrophy (PL)
Time Frame: 12 months
To assess the effect of metreleptin on HbA1c
12 months
Percent change from baseline to months 9 and 12 in fasting TGs in subjects with partial lipodystrophy (PL)
Time Frame: 12 months
To assess the effect of metreleptin on TGs
12 months
Change from Baseline to each assessment time point in quality of life (QoL) in all subjects
Time Frame: 12 months
To assess the effect of metreleptin on quality of life (QoL) in all subjects
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)

December 17, 2021

Primary Completion (Estimated)

July 21, 2026

Study Completion (Estimated)

October 31, 2026

Study Registration Dates

First Submitted

November 11, 2021

First Submitted That Met QC Criteria

December 6, 2021

First Posted (Actual)

December 20, 2021

Study Record Updates

Last Update Posted (Estimated)

August 29, 2025

Last Update Submitted That Met QC Criteria

August 25, 2025

Last Verified

August 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • APG-20

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

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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