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

March 11, 2024 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

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

65

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

  • Canada
    • Nova Scotia
      • Halifax, Nova Scotia, Canada, B3H 2Y9
        • Recruiting
        • Amryt Research Site, Nova Scotia Health
    • Ontario
      • Hamilton, Ontario, Canada, L8N 3Z5
        • Recruiting
        • Amryt Research Site, Hamilton General Hospital- McMaster University
      • London, Ontario, Canada, N6A 5B7
        • Recruiting
        • Amryt Research Site, London Health Science Centre
    • Quebec
      • Chicoutimi, Quebec, Canada, G7H 7K9
        • Recruiting
        • Amryt Research Site, ECOGENE-21
      • Québec, Quebec, Canada, G1V4W2
        • Recruiting
        • Amryt Research Site, Faculty of Medicine, Universite Laval
  • United States
    • Alabama
      • Birmingham, Alabama, United States, 35233
        • Recruiting
        • Amryt Research Site, University of Alabama
    • Massachusetts
      • Boston, Massachusetts, United States, 02114
        • Recruiting
        • Massachusetts General Hospital
    • Michigan
      • Ann Arbor, Michigan, United States, 48109
        • Recruiting
        • Amryt Research Site, University of Michigan
    • Minnesota
      • Rochester, Minnesota, United States, 55902
        • Recruiting
        • Amryt Research Site, Mayo Clinic,
    • Missouri
      • Columbia, Missouri, United States, 65212
        • Recruiting
        • Amryt Research Site, University of Missouri
    • Ohio
      • Columbus, Ohio, United States, 43210
        • Recruiting
        • Amryt Research Site, The Ohio State University Wexner Medical Center
      • Columbus, Ohio, United States, 43201
        • Suspended
        • Amryt Research Site, Endocrinology Research Associates Inc
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • Recruiting
        • Amryt Research Site, University of Pennsylvania
    • Texas
      • Dallas, Texas, United States, 75390
        • Recruiting
        • Amryt Research Site, UT Southwestern Medical Center
    • Wisconsin
      • Milwaukee, Wisconsin, United States, 53226
        • Recruiting
        • Amryt Research Site, 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

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

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • Diagnosis of Familial Partial Lipodystrophy (FPLD)

Exclusion Criteria:

  • Previous treatment with metreleptin

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)

June 30, 2025

Study Completion (Estimated)

January 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 (Actual)

March 12, 2024

Last Update Submitted That Met QC Criteria

March 11, 2024

Last Verified

March 1, 2024

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

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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