Mibavademab for adults and children with generalized lipodystrophy

A Two-Part, Randomized, Placebo-Controlled Phase 3 Study of the Efficacy, Safety, and Pharmacokinetics of Mibavademab in Patients With Generalized Lipodystrophy (LAGO)

This trial will test whether mibavademab helps adults and children with generalized lipodystrophy by improving blood sugar, lowering triglycerides, and reducing liver fat while checking safety and drug levels.

Quick facts

• Phase: Phase 3
• Study type: Interventional
• Enrollment: 28 (estimated)
• Ages: 2 Years and up
• Sex: All
• Sponsor: Regeneron Pharmaceuticals (Industry-sponsored)
• Drugs / interventions: mibavademab
• Locations: 2 sites (Ann Arbor, Michigan and 1 other locations)
• Trial ID: NCT07220785 on ClinicalTrials.gov

What this trial studies

This is a Phase 3, two-part trial enrolling both adults and pediatric patients with generalized lipodystrophy to test the effects and safety of mibavademab compared with placebo. Part A evaluates efficacy in participants aged 2 years and older who meet metabolic criteria (for example elevated HbA1c or high fasting triglycerides), while Part B focuses on safety and pharmacokinetics in younger pediatric participants under 12. Participants will receive either mibavademab or placebo and undergo regular blood tests for glucose, triglycerides, and drug levels, plus assessments of liver fat and antibody development. The study is conducted at specialized centers with serial visits to monitor metabolic outcomes and adverse events.

Who should consider this trial

Good fit: Ideal candidates are people with a confirmed diagnosis of congenital or acquired generalized lipodystrophy who meet the age and metabolic criteria (Part A: age ≥2 years with elevated HbA1c or high fasting triglycerides; Part B: children <12 years for safety/PK), and who can meet weight and screening test requirements.

Not a fit: People without generalized lipodystrophy, those who do not meet the metabolic entry criteria for the efficacy group, or those with medical conditions that exclude them from the protocol are unlikely to benefit from participation.

Why it matters

Potential benefit: If successful, mibavademab could improve metabolic control (lower blood sugar and triglycerides) and reduce liver fat in people with generalized lipodystrophy, potentially improving long-term health.

How similar studies have performed: Leptin replacement therapies (for example metreleptin) have previously improved metabolic outcomes in lipodystrophy, but mibavademab is a newer approach with limited published Phase 3 data to date.

Eligibility criteria

Key Inclusion Criteria:

1\. Diagnosis of congenital or acquired GLD as defined by Multi-Society Practice Guidelines

For Part A only:

1. Participants ≥2 years of age at screening
2. At least one of the below criteria are fulfilled during screening (measurements can be repeated once during screening period)

   * HbA1c ≥7%
   * Fasting TG ≥500 mg/dL
   * Fasting TG value of ≥300 mg/dL and the presence of another complication of GLD consistent with leptin deficiency (history of diabetes mellitus, hyperphagia, Metabolic Associated Fatty Liver Disease (MAFLD), polycystic ovary syndrome, etc)
3. Weight ≥15 kg at screening
4. Willing and able to provide, or have the treating physician provide, values of HbA1c and fasting TG from at least 6 months prior to screening, as described in the protocol

For Part B only:

1. Participants \<12 years of age at screening
2. Weighing ≥7 kg at screening
3. No metabolic criteria for study entry is required, as described in the protocol

Key Exclusion Criteria:

1. Has a current diagnosis of familial or acquired partial lipodystrophy or autoimmune (Type 1) diabetes mellitus
2. Any malignancy, eg, lymphoma, within the past 1 year, prior to screening visit, as described in the protocol
3. eGFR of \<30 mL/min/1.73 m2 based on Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) creatinine or Schwartz equation, as applicable, at screening. Assessment can be repeated once
4. History of heart failure hospitalization, diagnosis of a myocardial infarction, stroke, clinically significant arrhythmia, as described in the protocol
5. Treatment with over-the-counter or prescription medications with the intention of weight loss within 3 months prior to the screening visit

For Part A only:

1. Treatment with metreleptin within 3 months of the screening visit
2. Addition or discontinuation of prescription medications or over-the-counter supplements for diabetes and/or dyslipidemia within 3 months prior to the start of the screening period, or changes in the use of these medications, as described in the protocol
3. Significant changes to lifestyle and diet, as described in the protocol
4. Current chronic treatment with high-dose corticosteroids, defined as use of higher than physiologic doses, as described in the protocol

NOTE: Other protocol defined inclusion/exclusion criteria apply.

Where this trial is running

Ann Arbor, Michigan and 1 other locations

• University of Michigan — Ann Arbor, Michigan, United States (Recruiting)
• UT Southwestern Medical Center — Dallas, Texas, United States (Recruiting)

Study contacts

• Study coordinator: Clinical Trials Administrator

Conditions Generalized Lipodystrophy; Subcutaneous adipose tissue; GLD; Berardinelli-Seip Syndrome; Congenital Generalized Lipodystrophy; CGL; Lawrence Syndrome; Acquired Generalized Lipodystrophy