Open-label Extension Study to Evaluate Metreleptin in Patients With Partial Lipodystrophy

NCT06679270 | Recruiting Phase 3 FDA Drug

• 2 other identifiers: APG-20-OLEAPG-20 OLE
• Study Type: interventional
• Target: 24
• Locations: 1 country
• Sites: 7

Brief Summary

This Phase 3 study is an Open Label Extension of the APG-20 Study To Evaluate the Long-term Safety and Efficacy of Daily Subcutaneous Metreleptin Treatment in Subjects with Partial Lipodystrophy

Conditions

Familial Partial Lipodystrophy

Keywords

Metreleptin; familial partial lipodystrophy

Outcome Measures

Primary Outcomes (1)

• Evaluate the Incidence and Frequency of Treatment-Emergent Adverse Events (Safety and Tolerability)

  Incidence and frequency of treatment-emergent adverse events (TEAEs) including serious adverse events (SAEs), treatment-related TEAEs, adverse events of special interest (AESI), and adverse events leading to study treatment discontinuation/withdrawal from the study

  From enrollment to the end of treatment (until the last participant completes 24 months)

Secondary Outcomes (3)

• To evaluate the long-term efficacy (HbA1c) of daily SC metreleptin treatment in participants with familial partial lipodystrophy (FPLD)

  From enrollment to the end of treatment (until the last participant completes 24 months)

• To evaluate the long-term efficacy (TGs) of daily SC metreleptin treatment in participants with FPLD

  From enrollment to the end of treatment (until the last participant completes 24 months)

• To evaluate the long-term efficacy (FBG) of daily SC metreleptin treatment in participants with FPLD

  From enrollment to the end of treatment (until the last participant completes 24 months)

Study Arms (1)

Metreleptin

EXPERIMENTAL

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

Drug: Metreleptin

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

Eligibility Criteria

• Age: 13 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Age ≥13 years of age, inclusive, at the time of signing the informed consent form (ICF).
• Subjects must have completed the Parent study APG-20 and, in the opinion of the Investigator and Sponsor, have been compliant with study procedures through Parent study Month 12 visit.
• Negative pregnancy test (urine or serum) for female subjects of childbearing potential
• Female subjects must be postmenopausal (defined as cessation of menses for at least 1 year), surgically sterile (hysterectomy, bilateral oophorectomy, or tubal ligation), or willing to use a highly effective method of contraception (such methods include combined \[estrogen and progestogen containing\] hormonal contraception associated with inhibition of ovulation: oral/intravaginal; transdermal/progestogen-only hormonal contraception associated with inhibition of ovulation: oral/injectable; implantable/intrauterine device \[IUD\]/intrauterine hormone-releasing system \[IUS\]/bilateral tubal occlusion/vasectomized partner/sexual abstinence) for the duration of the study (from the time they sign an ICF, until 4 weeks after the last dose of study treatment). Hormonal contraception alone (including oral, injectable, transdermal, and implantable) is not acceptable; an additional barrier method must be used. Intravaginal hormonal contraception or IUS alone are allowed per Investigator's discretion. Subjects on oral contraceptives will not be required to discontinue medication. Subjects will not be permitted to commence oral contraceptives while taking study treatment during the study.
• Subjects who are blood/egg/sperm donors should be willing to halt donations during the study and for 4 weeks following their last dose of study treatment.
• Subjects who are willing to provide informed consent/assent prior to any study-specific procedures. If a minor, the subject has a parent or legal guardian able to read, understand, and sign the ICF and/or a Child Assent Form (if applicable), communicate with the Investigator, and understand and comply with the protocol requirements. Adolescent subjects must also read and understand the Child Assent Form.
• Subjects who are willing to follow the dietary restrictions recommended by the Investigator.

You may not qualify if:

• Severe hypersensitivity reactions to the study treatment of the Parent study APG-20.
• Known to have tested positive for human immunodeficiency virus (HIV) or known to be diagnosed with HIV-related LD. Positive HIV test in countries requiring HIV testing.
• Are immunocompromised or receiving immunomodulatory drugs.
• Estimated glomerular filtration rate (eGFR) \<30 mL/min/1.73m2 calculated by Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) for subjects ≥18 years of age and by Bedside Schwartz for subjects \<18 years of age.
• Diagnosis of clinically significant hematological abnormalities (including but not limited to clinically significant leukopenia, neutropenia, bone marrow abnormalities, leukemia or lymphoma, or clinically significant pathological lymphadenopathy).
• Malignancy that is ongoing/not in remission or that currently requires or has required active treatment within the past year (with the exception of basal cell carcinoma of the skin, squamous cell carcinoma of the skin, or carcinoma in situ \[e.g., breast carcinoma, cervical cancer in situ\] that have undergone potentially curative therapy).
• For females only: currently pregnant (confirmed with a positive pregnancy test) or breastfeeding.
• Any condition where, in the opinion of the Investigator, participation in this study may pose a significant risk to the subject.

Sponsors & Collaborators

• Amryt Pharma: lead

Study Sites (7)

University of Alabama

Birmingham, Alabama, 35233, United States

RECRUITING

Flourish Research

Boca Raton, Florida, 33434, United States

RECRUITING

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

RECRUITING

University of Michigan

Ann Arbour, Michigan, 48109, United States

RECRUITING

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

NOT YET RECRUITING

UT Southwestern Medical Center

Dallas, Texas, 75390, United States

RECRUITING

The Medical College of Wisconsin

Milwaukee, Wisconsin, 53226, United States

RECRUITING

MeSH Terms

Conditions

Lipodystrophy, Familial Partial

Interventions

metreleptin

Condition Hierarchy (Ancestors)

Laminopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lipodystrophy; Skin Diseases, Metabolic; Skin Diseases; Skin and Connective Tissue Diseases; Lipid Metabolism, Inborn Errors; Lipid Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases

Central Study Contacts

Lori Hartnett, PhD

CONTACT

+3905212791; clinicaltrials_info@chiesi.com

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 5, 2024
• First Posted: November 7, 2024
• Study Start: October 14, 2024
• Primary Completion (Estimated): March 1, 2028
• Study Completion (Estimated): December 1, 2028
• Last Updated: February 2, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will not share

Locations

US (7)