A Study of the Safety of Mibavademab in Pediatric and Adult Participants Switching From Metreleptin to Mibavademab for the Treatment of Generalized Lipodystrophy (GLD)

NCT06548100 | Active Not Recruiting Phase 3 FDA Drug

• 3 other identifiers: R4461-GLD-22842024-513202-54-002026-000121-16
• Study Type: interventional
• Target: 9
• Locations: 1 country
• Sites: 2

Brief Summary

This study is researching an experimental drug called mibavademab. The study is focused on participants with GLD who have been on metreleptin treatment for at least 6 months with no change in dose for the last 3 months. The aim of the study is to see how safe and tolerable mibavademab is when switching from treatment with metreleptin. The study is looking at several other research questions, including:

• What side effects may happen from taking mibavademab
• How much mibavademab is in the blood at different times
• Whether the body makes antibodies against mibavademab (which could make mibavademab less effective or could lead to side effects)

Conditions

Generalized Lipodystrophy

Keywords

Congenital or acquired generalized lipodystrophy; Leptin deficiency; Loss of subcutaneous tissue; Nutritional deprivation; Severe metabolic derangements; Severe diabetes mellitus; Hypertriglyceridemia; Berardinelli-Seip Syndrome; Lawrence Syndrome; Severe Insulin Resistance

Outcome Measures

Primary Outcomes (2)

• Incidence of treatment-emergent adverse events (TEAEs)

  Up to week 68

• Severity of TEAEs

  Up to week 68

Secondary Outcomes (14)

• Change in Hemoglobin A1c (HbA1c)

  Baseline, week 20 and week 52

• Occurrence of HbA1c <7%

  Week 20 and week 52

• Occurrence of HbA1c <6.5%

  Week 20 and week 52

• Occurrence of requiring therapy with insulin in participants treated with mibavademab

  Week 20 and week 52

• Change in total insulin dose

  Baseline, week 20 and week 52

Study Arms (1)

mibavademab

EXPERIMENTAL

Patients switching from at least 6 months of therapy with metreleptin to mibavademab.

Drug: mibavademab

Interventions

mibavademab DRUG

Administered by intravenous (IV) infusion followed by subcutaneous (SC) injection

Also known as: REGN4461

mibavademab

Eligibility Criteria

• Age: 2 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Diagnosis of congenital or acquired GLD as defined by Multi-Society Practice Guidelines
• 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
• 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
• 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
• 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

You may not qualify if:

• Treatment with over-the-counter or prescription medications for weight loss within 3 months prior to the screening visit
• Current chronic treatment with high-dose corticosteroids, as defined in the protocol
• 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
• 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
• 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
• 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

Sponsors & Collaborators

• Regeneron Pharmaceuticals: lead

Study Sites (2)

National Institutes of Health

Bethesda, Maryland, 20892, United States

Location

University of Michigan

Ann Arbor, Michigan, 48109, United States

Location

MeSH Terms

Conditions

Lipodystrophy, Congenital Generalized; Obesity, Morbid; Diabetes Mellitus; Hypertriglyceridemia; Lipodystrophy, Familial Partial; Insulin Resistance

Condition Hierarchy (Ancestors)

Lipid Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lipodystrophy; Skin Diseases, Metabolic; Skin Diseases; Skin and Connective Tissue Diseases; Lipid Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases; Obesity; Overweight; Overnutrition; Nutrition Disorders; Body Weight; Signs and Symptoms; Pathological Conditions, Signs and Symptoms; Glucose Metabolism Disorders; Endocrine System Diseases; Hyperlipidemias; Dyslipidemias; Laminopathies; Hyperinsulinism

Study Officials

• Clinical Trial Management

  Regeneron Pharmaceuticals

  STUDY DIRECTOR

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: August 7, 2024
• First Posted: August 12, 2024
• Study Start: December 16, 2024
• Primary Completion (Estimated): July 14, 2026
• Study Completion (Estimated): November 3, 2026
• Last Updated: March 30, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR, ANALYTIC CODE
• Time Frame: When Regeneron has : * received marketing authorization from major health authorities (e.g., FDA, European Medicines Agency (EMA), Pharmaceuticals and Medical Devices Agency (PMDA), etc.) for the product and indication or has globally discontinued development of the product for all indications on or after April 2020 and has no plans for future development * made results publicly available (e.g., scientific publication, scientific conference, clinical trial registry) * the legal authority to share the data, and * ensured the ability to protect participant privacy
• Access Criteria: Qualified researchers can submit a proposal for access to individual patient or aggregate level data from a Regeneron-sponsored clinical trial through Vivli. Regeneron's Independent Research Request Evaluation Criteria can be found at: https://www.regeneron.com/sites/default/files/Regeneron-External-Data-Sharing-Policy-and-Independent-Research-Request-Evaluation-Criteria.pdf

Locations

US (2)