Efficacy and Safety of Mibavademab in Adult and Pediatric Patients With Generalized Lipodystrophy

Regeneron Pharmaceuticals

Status and phase: Begins enrollment this month; Phase 3

Conditions: Generalized Lipodystrophy

Treatments: Drug: Placebo; Drug: Mibavademab

Study type: Interventional

Funder types: Industry

Identifiers

NCT07220785

R4461-GLD-2285

2024-519879-25-00 (Registry Identifier)

Details and patient eligibility

About

This study is researching a new drug called mibavademab (called "study drug"). The study involves participants with a condition called Generalized Lipodystrophy (GLD). The aim of the study is to see how well mibavademab works and what side effects it has. Researchers will also look at how much mibavademab is in the body at different times.

This is a 2-part study: Part A is an efficacy study in pediatric and adult participants, Part B is a safety and pharmacokinetic study in pediatric participants.

The study is researching several other questions, including:

• How mibavademab affects the amount of sugar in the blood
• How mibavademab affects the amount of fat (triglycerides) in the blood
• How mibavademab affects the amount of fat that has built up in the liver
• Whether the body makes antibodies against the study drug (which could make the drug less effective or could lead to side effects)

Enrollment: 28 estimated patients

Sex: All

Ages: 2+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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 ≥2 and <12 years of age at screening
2. No metabolic criteria or minimum weight for study entry is required

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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28 participants in 5 patient groups, including a placebo group

Part A: Arm A

Experimental group

Treatment:

Drug: Mibavademab

Part A: Arm B

Experimental group

Treatment:

Drug: Mibavademab

Drug: Placebo

Part A: Arm C

Experimental group

Treatment:

Drug: Mibavademab

Part A: Arm D

Placebo Comparator group

Treatment:

Drug: Placebo

Part B

Experimental group

Treatment:

Drug: Mibavademab