Current partner codePEPTIDESDE
NCT04026178·Phase 4·INTERVENTIONAL

Immunogenicity of Metreleptin in Patients With Generalized Lipodystrophy

Status

Completed

Phase

Phase 4

Enrollment

11

Locations

8

Results

Posted

Publications

0

Study summary

What the protocol is testing.

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.

Interventions

Treatment arms and agents.

DRUG

Metreleptin

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

Timeline

From registration to results.

  1. First posted

    Jul 19, 2019

  2. Study start

    Nov 14, 2018

  3. Primary completion

    Oct 31, 2024

  4. Study completion

    Oct 31, 2024

  5. Results posted

    Nov 26, 2025

  6. Registry updated

    Nov 26, 2025

Outcomes

What the study measures.

Primary outcomes

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

Time frame · 36 months

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.

Secondary outcomes

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

Time frame · 36 months

The percent (standard error) of patients with a positive result was compared for the RB NAb assay and the cell-based NAb assay over time.

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

Time frame · 36 months

Serious adverse events (SAEs), AEs leading to discontinuation, loss of response (as assessed by glycated hemoglobin (HbA1c) and serum triglycerides), severe infections and/or sepsis, standard laboratory tests, and vital signs over time.

Eligibility

Who can take part.

Minimum age
1 Year
Maximum age
Not reported
Sex
ALL
Healthy volunteers
No

Inclusion Criteria: 1. 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. 2. Female and/or male patients ≥1 years of age. 3. Physician-confirmed diagnosis of congenital or acquired generalized lipodystrophy and will begin treatment with MYALEPT for the first time. 4. Negative pregnancy test (urine or serum) for female patients of childbearing potential. 5. 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. 6. 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). 7. Patients who are blood donors should not donate blood during the study and for 3 months following their last dose of metreleptin. Exclusion Criteria: 1. Involvement in the planning and/or conduct of the study (applies to both Aegerion staff and/or staff at the study site.) 2. Previous treatment with metreleptin. 3. Participation in another clinical study with an investigational product during the last 6 months. 4. Patients with prior severe hypersensitivity reactions to metreleptin or to any of the product components. 5. Known to have tested positive for human immunodeficiency virus, are immunocompromised, or are receiving immunomodulatory drugs. 6. Known history of drug or alcohol abuse within 1 year of screening. 7. 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. 8. For women only - currently pregnant (confirmed with positive pregnancy test) or breast-feeding. 9. 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.

Study locations

8 registered sites.

United States. Showing up to 24 locations stored in the fast local snapshot.

Univ. Alabama-Birmingham

Birmingham, Alabama, United States

Ochsner Clinic

New Orleans, Louisiana, United States

University of Michigan

Ann Arbor, Michigan, United States

Endocrinology Research Associates

Columbus, Ohio, United States

Ohio State University

Columbus, Ohio, United States

Childrens Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

University Texas Southwestern INT

Dallas, Texas, United States

Seattle Children's Hospital

Seattle, Washington, United States

Publications

Results and literature.

No PMID-linked publications were present in this registry snapshot.

Primary links

Continue at the source.

Related trials

More studies on Metreleptin.

Related PeptideStat pages

Put the record in context.

Research pages describe evidence. Vendor pages, where available, describe independently tracked research-product listings and are not clinical recommendations.