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

NCT05164341 | Active Not Recruiting Phase 3 DMC FDA Drug

• 1 other identifier: APG-20
• Study Type: interventional
• Target: 69
• Locations: 9 countries
• Sites: 31

Brief Summary

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

Conditions

Partial Lipodystrophy

Keywords

metreleptin

Outcome Measures

Primary Outcomes (2)

• Change from baseline to month 6 in HbA1c in subjects with partial lipodystrophy (PL)

  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)

  To evaluate the efficacy (TGs) of daily SC metreleptin treatment

  6 months

Secondary Outcomes (4)

• Safety analysis of AEs, AESIs, SAEs by treatment arm

  12 months

• Change from baseline to months 9 and 12 in HbA1c in subjects with partial lipodystrophy (PL)

  12 months

• Percent change from baseline to months 9 and 12 in fasting TGs in subjects with partial lipodystrophy (PL)

  12 months

• Change from Baseline to each assessment time point in quality of life (QoL) in all subjects

  12 months

Study Arms (2)

metreleptin

ACTIVE COMPARATOR

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

Drug: metreleptin

placebo

PLACEBO COMPARATOR

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

Drug: Placebo

Interventions

metreleptin 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

metreleptin

Placebo DRUG

Placebo

placebo

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

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

Sponsors & Collaborators

• Amryt Pharma: lead

Study Sites (31)

University of Alabama

Birmingham, Alabama, 35233, United States

Location

UC Davis

Sacramento, California, 95817, United States

Location

Flourish Boca Raton

Boca Raton, Florida, 33431, United States

Location

Emory University

Atlanta, Georgia, 30329, United States

Location

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

University of Michigan

Ann Arbor, Michigan, 48109, United States

Location

Mayo Clinic

Rochester, Minnesota, 55902, United States

Location

University of Missouri

Columbia, Missouri, 65212, United States

Location

Amryt Research Site, Endocrinology Research Associates Inc

Columbus, Ohio, 43201, United States

Location

The Ohio State University Wexner Medical Center

Columbus, Ohio, 43210, United States

Location

The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

UT Southwestern Medical Center

Dallas, Texas, 75390, United States

Location

Medical College of Wisconsin

Milwaukee, Wisconsin, 53226, United States

Location

UZ Leuven

Leuven, Belgium

Location

Universitário Walter Cantídio

Fortaleza, Brazil

Location

Insight Centro de Pesquisas

Porto Alegre, Brazil

Location

Hospital das Clinicas FMRP-USP

Ribeirão Preto, Brazil

Location

Instituto Estadual de Diabetes e Endocrinologia (IEDE-RJ)

Rio de Janeiro, Brazil

Location

Nova Scotia Health

Halifax, Nova Scotia, B3H 2Y9, Canada

Location

Care Access Clinic

Sydney, Nova Scotia, B1M 0A1, Canada

Location

Hamilton General Hospital- McMaster University

Hamilton, Ontario, L8N 3Z5, Canada

Location

London Health Science Centre

London, Ontario, N6A 5B7, Canada

Location

Ecogene-21

Chicoutimi, Quebec, G7H 7K9, Canada

Location

Faculty of Medicine, Universite Laval

Québec, Quebec, G1V4W2, Canada

Location

Universidad Catolica

Santiago, Chile

Location

Hadassah University Hospital - Ein Kerem

Jerusalem, Israel

Location

Leids Universitair Medisch Centrum

Leiden, Netherlands

Location

Radboud University Medical Centre Interne Geneeskund

Nijmegen, Netherlands

Location

Uniwersyteckie Centrum Kliniczne

Katowice, Poland

Location

SPZOZ Szpital Uniwersytecki w Krakowie

Krakow, Poland

Location

University Clinical Hospital of Santiago de Compostela/Hospital Universitario

Santiago de Compostela, Spain

Location

MeSH Terms

Conditions

Lipodystrophy, Partial, Acquired

Interventions

metreleptin

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 11, 2021
• First Posted: December 20, 2021
• Study Start: December 17, 2021
• Primary Completion (Estimated): July 21, 2026
• Study Completion (Estimated): October 31, 2026
• Last Updated: August 29, 2025

Record last verified: 2025-08

Locations

BE (1); BR (4); CL (1); IL (1); US (13); PL (2); CA (6); ES (1); NL (2)