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

NCT ID: NCT05164341

Last Updated: 2025-08-29

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE3

Total Enrollment

69 participants

Study Classification

INTERVENTIONAL

Study Start Date

2021-12-17

Study Completion Date

2026-10-31

Brief Summary

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

Detailed Description

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Conditions

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Partial Lipodystrophy

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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metreleptin

Metreleptin \[Recombinant-methionyl human Leptin; r-metHuLeptin\] for daily injection is a sterile, white, solid lyophilised cake

Group Type ACTIVE_COMPARATOR

metreleptin

Intervention Type DRUG

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

Placebo for daily injection is a sterile, white, solid lyophilised cake

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Placebo

Interventions

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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

Intervention Type DRUG

Placebo

Placebo

Intervention Type DRUG

Eligibility Criteria

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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)
Minimum Eligible Age

12 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Amryt Pharma

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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University of Alabama

Birmingham, Alabama, United States

Site Status

UC Davis

Sacramento, California, United States

Site Status

Flourish Boca Raton

Boca Raton, Florida, United States

Site Status

Emory University

Atlanta, Georgia, United States

Site Status

Massachusetts General Hospital

Boston, Massachusetts, United States

Site Status

University of Michigan

Ann Arbor, Michigan, United States

Site Status

Mayo Clinic

Rochester, Minnesota, United States

Site Status

University of Missouri

Columbia, Missouri, United States

Site Status

Amryt Research Site, Endocrinology Research Associates Inc

Columbus, Ohio, United States

Site Status

The Ohio State University Wexner Medical Center

Columbus, Ohio, United States

Site Status

The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Site Status

UT Southwestern Medical Center

Dallas, Texas, United States

Site Status

Medical College of Wisconsin

Milwaukee, Wisconsin, United States

Site Status

UZ Leuven

Leuven, , Belgium

Site Status

Universitário Walter Cantídio

Fortaleza, , Brazil

Site Status

Insight Centro de Pesquisas

Porto Alegre, , Brazil

Site Status

Hospital das Clinicas FMRP-USP

Ribeirão Preto, , Brazil

Site Status

Instituto Estadual de Diabetes e Endocrinologia (IEDE-RJ)

Rio de Janeiro, , Brazil

Site Status

Nova Scotia Health

Halifax, Nova Scotia, Canada

Site Status

Care Access Clinic

Sydney, Nova Scotia, Canada

Site Status

Hamilton General Hospital- McMaster University

Hamilton, Ontario, Canada

Site Status

London Health Science Centre

London, Ontario, Canada

Site Status

Ecogene-21

Chicoutimi, Quebec, Canada

Site Status

Faculty of Medicine, Universite Laval

Québec, Quebec, Canada

Site Status

Universidad Catolica

Santiago, , Chile

Site Status

Hadassah University Hospital - Ein Kerem

Jerusalem, , Israel

Site Status

Leids Universitair Medisch Centrum

Leiden, , Netherlands

Site Status

Radboud University Medical Centre Interne Geneeskund

Nijmegen, , Netherlands

Site Status

Uniwersyteckie Centrum Kliniczne

Katowice, , Poland

Site Status

SPZOZ Szpital Uniwersytecki w Krakowie

Krakow, , Poland

Site Status

University Clinical Hospital of Santiago de Compostela/Hospital Universitario

Santiago de Compostela, , Spain

Site Status

Countries

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United States Belgium Brazil Canada Chile Israel Netherlands Poland Spain

Other Identifiers

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APG-20

Identifier Type: -

Identifier Source: org_study_id

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