Immunogenicity of Metreleptin in Patients With Generalized Lipodystrophy

NCT04026178 | Completed Phase 4 Results FDA Drug

• 1 other identifier: AEGR-734-401
• Study Type: interventional
• Target: 11
• Locations: 1 country
• Sites: 8

Brief Summary

MYALEPT™ (metreleptin) has been approved as an adjunct to diet as replacement therapy to treat the complications of leptin deficiency in patients with congenital or acquired generalized lipodystrophy (MYALEPT Prescribing Information). This study is a multicenter, open-label, Phase 4 trial to provide an assessment of the immunogenicity associated with metreleptin and of any major potential risks due to development of antibodies to metreleptin. The study is being conducted to comply with a postmarketing requirement.

Conditions

Generalized Lipodystrophy

Keywords

Immunogenicity; Antibodies; Lipodystrophy

Outcome Measures

Primary Outcomes (1)

• Evaluate the Immunogenicity Associated With Daily Subcutaneous (SC) Metreleptin Treatment in Patients With Congenital Generalized Lipodystrophy (CGL) or Acquired Generalized Lipodystrophy (AGL).

  Anti-metreleptin, anti-human leptin (HuL) binding antidrug antibody (ADA) titers over time. Category of in vitro cell-based neutralizing antibody (NAb) activity to metreleptin, and titer in the receptor blocking (RB) NAb assay in ADA positive samples over time.

  36 months

Secondary Outcomes (2)

• Assess 2 Methods of Measuring in Vitro NAb Activity to Metreleptin.

  36 months

• Evaluate the Safety and Tolerability in Relation to the Development of or Absence of Anti-metreleptin and Anti-HuL Binding ADAs, and/or in Vitro NAb Activity to Metreleptin in Patients With CGL or AGL

  36 months

Other Outcomes (2)

• Evaluate the Efficacy With Daily Metreleptin in Patients With GL

  36 months

• Evaluate the Efficacy With Daily Metreleptin in Patients With GL

  36 months

Study Arms (1)

Metreleptin

EXPERIMENTAL

Subjects will receive prescribed dosage of metreleptin as indicated in the USPI Patients (males and females) ≤ 40 kg: 0.06mg/kg Male patients \> 40 kg: 2.5mg Female patients \> 40 kg: 5mg

Drug: Metreleptin

Interventions

Metreleptin DRUG

Subjects will receive prescribed dosage of metreleptin as indicated in the USPI

Metreleptin

Eligibility Criteria

• Age: 1 Year+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Provision of informed consent prior to any study specific procedures. If \<18 years of age, has a parent or guardian able to read, understand, and sign the Informed Consent Form (ICF) and a Child Assent form, communicate with the Investigator, and understand and comply with protocol requirements. Adolescent patients must also read and understand the Child Assent Form. If the child is too young or unable to read, then the Child Assent form must be explained to the child.
• Female and/or male patients ≥1 years of age.
• Physician-confirmed diagnosis of congenital or acquired generalized lipodystrophy and will begin treatment with MYALEPT for the first time.
• Negative pregnancy test (urine or serum) for female patients of childbearing potential.
• Female patients of childbearing potential must be 1 year postmenopausal, surgically sterile, or be willing to use an acceptable method of contraception (an acceptable method of contraception is defined as a barrier method in conjunction with a spermicide) for the duration of the study (from the time they sign consent). In addition, oral contraceptives, approved contraceptive implant, long-term injectable contraception, intrauterine device, or tubal ligation are allowed. Oral contraception alone is not acceptable; additional barrier methods in conjunction with spermicide must be used.
• Male patients must be surgically sterile or be willing to use an acceptable method of contraception (defined as barrier methods in conjunction with spermicides) for the duration of the study (from the time they sign consent).
• Patients who are blood donors should not donate blood during the study and for 3 months following their last dose of metreleptin.

You may not qualify if:

• Involvement in the planning and/or conduct of the study (applies to both Aegerion staff and/or staff at the study site.)
• Previous treatment with metreleptin.
• Participation in another clinical study with an investigational product during the last 6 months.
• Patients with prior severe hypersensitivity reactions to metreleptin or to any of the product components.
• Known to have tested positive for human immunodeficiency virus, are immunocompromised, or are receiving immunomodulatory drugs.
• Known history of drug or alcohol abuse within 1 year of screening.
• Creatinine clearance \<30 mL/min using institutional standards:
• e.g., calculated using Cockcroft-Gault formula for patients ≥18 years of age; calculated using Schwartz equation for patients \<18 years of age.
• For women only - currently pregnant (confirmed with positive pregnancy test) or breast-feeding.
• Any condition where, in the opinion of the Investigator, participation in this study may pose a significant risk to the patient or could render the patient unable to successfully complete the study.

Sponsors & Collaborators

• Aegerion Pharmaceuticals, Inc.: lead

Study Sites (8)

Univ. Alabama-Birmingham

Birmingham, Alabama, 35294, United States

Location

Ochsner Clinic

New Orleans, Louisiana, 70121, United States

Location

University of Michigan

Ann Arbor, Michigan, 48109, United States

Location

Endocrinology Research Associates

Columbus, Ohio, 43201, United States

Location

Ohio State University

Columbus, Ohio, 43203, United States

Location

Childrens Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

University Texas Southwestern INT

Dallas, Texas, 75390, United States

Location

Seattle Children's Hospital

Seattle, Washington, 98105, United States

Location

MeSH Terms

Conditions

Lipodystrophy, Congenital Generalized; Lipodystrophy

Interventions

metreleptin

Condition Hierarchy (Ancestors)

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

Limitations and Caveats

Following limitations may have occurred: 1. Exposure misclassification 2. Selection bias 3. Attrition 4. Confounders (e.g., concomitant use of antidiabetic medications, metreleptin dose and compliance, pregnancy, puberty) 5. Small sample size

Results Point of Contact

• Title: Metreleptin Clinical Program Lead
• Organization: Chiesi

clinicaltrials_info@chiesi.com

+3905212791

Study Officials

• Janet Boylan

  Aegerion Pharmaceuticals, Inc.

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 30, 2018
• First Posted: July 19, 2019
• Study Start: November 14, 2018
• Primary Completion: October 31, 2024
• Study Completion: October 31, 2024
• Last Updated: November 26, 2025
• Results First Posted: November 26, 2025

Record last verified: 2025-11

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, ICF
• Time Frame: A copy of data generated at the study site will be provided to individual sites after the overall publication by the sponsor is completed and submitted

Locations

US (8)