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

NCT07220785 | Recruiting Phase 3 DMC FDA Drug

• 2 other identifiers: R4461-GLD-22852024-519879-25-00
• Study Type: interventional
• Target: 28
• Locations: 1 country
• Sites: 2

Brief Summary

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)

Conditions

Generalized Lipodystrophy

Keywords

Subcutaneous (SC) adipose tissue; GLD; Berardinelli-Seip Syndrome; Congenital Generalized Lipodystrophy; CGL; Lawrence Syndrome; Acquired Generalized Lipodystrophy; AGL

Outcome Measures

Primary Outcomes (5)

• Change in Hemoglobin A1c (HbA1c)

  Part A

  Through 36 weeks of exposure to mibavademab

• Percent change in fasting Triglycerides (TG)

  Part A

  Through 36 weeks of exposure to mibavademab

• Occurrence of Treatment Emergent Adverse Events (TEAEs)

  Part B

  Up to 15 months

• Severity of TEAEs

  Part B

  Up to 15 months

• Concentrations of total mibavademab in serum

  Part B

  Up to 15 months

Secondary Outcomes (34)

• Change in HbA1c compared to placebo

  From baseline to week 20

• Change in HbA1c compared to placebo

  From week 56 to week 64

• Change in HbA1c

  From baseline to week 52

• Percent change in fasting TG compared to placebo

  From baseline to week 20

• Percent change in fasting TG compared to placebo

  From week 56 to week 64

Study Arms (5)

Part A: Arm A

EXPERIMENTAL

Drug: Mibavademab

Part A: Arm B

EXPERIMENTAL

Drug: Mibavademab; Drug: Placebo

Part A: Arm C

EXPERIMENTAL

Drug: Mibavademab

Part A: Arm D

PLACEBO COMPARATOR

Drug: Placebo

Part B

EXPERIMENTAL

Drug: Mibavademab

Interventions

Placebo DRUG

Administered as per the protocol

Part A: Arm B; Part A: Arm D

Mibavademab DRUG

Administered as per the protocol

Also known as: REGN4461

Part A: Arm A; Part A: Arm B; Part A: Arm C; Part B

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
• For Part A only:
• Participants ≥2 years of age at screening
• 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)
• Weight ≥15 kg at screening
• 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:
• Participants \<12 years of age at screening
• Weighing ≥7 kg at screening
• No metabolic criteria for study entry is required, as described in the protocol

You may not qualify if:

• Has a current diagnosis of familial or acquired partial lipodystrophy or autoimmune (Type 1) diabetes mellitus
• Any malignancy, eg, lymphoma, within the past 1 year, prior to screening visit, as described in the protocol
• 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
• History of heart failure hospitalization, diagnosis of a myocardial infarction, stroke, clinically significant arrhythmia, as described in the protocol
• 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:
• Treatment with metreleptin within 3 months of the screening visit
• 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
• Significant changes to lifestyle and diet, as described in the protocol
• Current chronic treatment with high-dose corticosteroids, defined as use of higher than physiologic doses, as described in the protocol

Sponsors & Collaborators

• Regeneron Pharmaceuticals: lead

Study Sites (2)

University of Michigan

Ann Arbor, Michigan, 48109, United States

RECRUITING

UT Southwestern Medical Center

Dallas, Texas, 75390, United States

RECRUITING

MeSH Terms

Conditions

Lipodystrophy, Congenital Generalized; Lipodystrophy, Familial Partial

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; Laminopathies

Study Officials

• Clinical Trial Management

  Regeneron Pharmaceuticals

  STUDY DIRECTOR

Central Study Contacts

Clinical Trials Administrator

CONTACT

844-734-6643; clinicaltrials@regeneron.com

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR
• Expanded Access: Yes

Model Details: Part A will be double blinded; Part B will be open label

Study Record Dates

• First Submitted: October 22, 2025
• First Posted: October 24, 2025
• Study Start: March 13, 2026
• Primary Completion (Estimated): May 19, 2028
• Study Completion (Estimated): September 8, 2028
• Last Updated: May 6, 2026

Record last verified: 2026-05

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 the study 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)