Safety of Mibavademab for Patients with Generalized Lipodystrophy Switching from Metreleptin

A Single-Arm, Open-Label, Safety Study in Patients With Generalized Lipodystrophy Switching From Metreleptin to Mibavademab, A Leptin Receptor Agonist Antibody

This study is testing if switching to a new drug called mibavademab is safe for people with generalized lipodystrophy who have been using metreleptin for a while.

Quick facts

• Phase: PHASE3
• Study type: Interventional
• Enrollment: 8 (estimated)
• Ages: 2 Years and up
• Sex: All
• Sponsor: Regeneron Pharmaceuticals (industry)
• Drugs / interventions: mibavademab
• Locations: 2 sites (Bethesda, Maryland and 1 other locations)
• Trial ID: NCT06548100 on ClinicalTrials.gov

What this trial studies

This study investigates the safety and tolerability of an experimental drug called mibavademab in participants with generalized lipodystrophy (GLD) who have been on a stable dose of metreleptin for at least six months. Participants will be monitored for side effects, blood levels of mibavademab, and the body's immune response to the drug. The goal is to determine if switching to mibavademab is a safe alternative for these patients. The study involves both pediatric and adult participants.

Who should consider this trial

Good fit: Ideal candidates include individuals diagnosed with congenital or acquired generalized lipodystrophy who have been stable on metreleptin for at least six months.

Not a fit: Patients who have recently used weight loss medications or those not currently on metreleptin may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could provide a new treatment option for patients with generalized lipodystrophy who are not adequately managed on metreleptin.

How similar studies have performed: While this approach is novel in the context of switching from metreleptin to mibavademab, similar studies have shown promise in exploring new treatments for metabolic disorders.

Eligibility criteria

Key Inclusion Criteria:

1. Diagnosis of congenital or acquired GLD as defined by Multi-Society Practice Guidelines
2. Treatment with metreleptin for ≥6 months at time of screening at a stable dose, defined as no change in dose within the last 3 months prior to screening
3. Generally stable diet (based on participant's recall) and stable medication regimen for diabetes and/or dyslipidemia (in addition to metreleptin), for the last 3 months prior to screening
4. Willing and able to comply with clinic visits and study-related procedures. Participants who are unable/unwilling to self-inject, but are willing to have a capable caregiver inject, are considered eligible
5. Willing and able to provide, or have the treating physician provide, values of HbA1c and fasting triglycerides from at least 6 months prior to screening, as defined in the protocol

Key Exclusion Criteria:

1. Treatment with over-the-counter or prescription medications for weight loss within 3 months prior to the screening visit
2. Current chronic treatment with high-dose corticosteroids, as defined in the protocol
3. Any malignancy, eg, lymphoma, within the past 1 year, prior to screening visit except for fully treated basal cell or squamous epithelial cell carcinomas of the skin or carcinoma in situ of the cervix or anus
4. Estimated glomerular filtration rate (GFR) of \<30 mL/min/1.73 m\^2 based on chronic kidney disease epidemiology collaboration (CKD-EPI)/Schwartz equation at screening. Assessment can be repeated once
5. History of heart failure hospitalization, diagnosis of a myocardial infarction, stroke, clinically significant arrhythmia, transient ischemic attack, unstable angina, percutaneous or surgical revascularization procedure, or intracardiac device placement within 3 months before the screening visit, as defined in the protocol
6. Any physical examination findings and/or history of any illness that, in the opinion of the study investigator, might confound the results of the study or pose an additional risk to the participant by their participation in the study, as defined in the protocol

NOTE: Other protocol-defined inclusion / exclusion criteria apply

Where this trial is running

Bethesda, Maryland and 1 other locations

• National Institutes of Health — Bethesda, Maryland, United States (RECRUITING)
• University of Michigan — Ann Arbor, Michigan, United States (RECRUITING)

Study contacts

• Study coordinator: Clinical Trials Administrator

Conditions: Generalized Lipodystrophy, Congenital or acquired generalized lipodystrophy, Leptin deficiency, Loss of subcutaneous tissue, Nutritional deprivation, Severe metabolic derangements, Severe diabetes mellitus, Hypertriglyceridemia