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

November 4, 2026 updated by: Chiesi Farmaceutici S.p.A.

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

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Detailed Description

This Phase 3, randomised, multicentre, double-blind, placebo-controlled study is designed to evaluate the safety and efficacy of once-daily subcutaneous metreleptin in participants with partial lipodystrophy and associated metabolic complications. The study compares metreleptin with placebo over a 12-month treatment period, followed by an extension phase. The primary objective is to assess the effect of metreleptin on key metabolic parameters, including glycaemic control and hypertriglyceridemia.

Secondary objectives include evaluation of safety, tolerability, additional metabolic outcomes, liver-related parameters, and patient-reported outcomes in this rare disease population.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
69

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • 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 Clinicas 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

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

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

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

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

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
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

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
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

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
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

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Chiesi Farmaceutici S.p.A.

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Principal Investigator: Elif Oral, University of Michigan

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)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
December 17, 2021

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
July 14, 2026

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
October 6, 2026

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
November 11, 2021

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
December 6, 2021

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
December 20, 2021

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
November 9, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
November 4, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
November 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant 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

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.

Cookie Settings

We use cookies to ensure that we give you the best experience on our website. For more details carefully read our Privacy and Cookies Policy. ...>>>You can change your preferences or withdraw your consent at any time by deleting the cookies from your website or computer as described in the policy. Please accept it and choose your preferences by ticking the corresponding boxes in the "Manage Settings" section below. Please carefully read our Terms of Service before you proceed with using any part of this website.
«Manage Settings