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    Summary
    EudraCT Number:2026-000121-16
    Sponsor's Protocol Code Number:R4461-GLD-2284
    Clinical Trial Type:Outside EU/EEA
    Date on which this record was first entered in the EudraCT database:2026-08-28
    Trial results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    H.4 THIRD COUNTRY IN WHICH THE TRIAL WAS FIRST AUTHORISED
    Expand All   Collapse All
    A. Protocol Information
    A.2EudraCT number2026-000121-16
    A.3Full title of the trial
    A Single-Arm, Open-Label, Safety Study in Patients with Generalized Lipodystrophy Switching from Metreleptin to Mibavademab, A Leptin Receptor Agonist Antibody
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Study of the Safety of Mibavademab in Pediatric and Adult Participants Switching from Metreleptin to Mibavademab for the Treatment of Generalized Lipodystrophy (GLD)
    A.4.1Sponsor's protocol code numberR4461-GLD-2284
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT06548100
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorRegeneron Pharmaceuticals Inc.
    B.1.3.4CountryUnited States
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportRegeneron Pharmaceuticals Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationRegeneron Pharmaceuticals Inc.
    B.5.2Functional name of contact pointMedical Affairs
    B.5.3 Address:
    B.5.3.1Street AddressOne Warrington Place
    B.5.3.2Town/ cityDublin
    B.5.3.3Post codeD02 HH27
    B.5.3.4CountryIreland
    B.5.4Telephone number+35 3818882469
    B.5.6E-mailmedical.information_global@regeneron.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/24/2903
    D.3 Description of the IMP
    D.3.1Product nameMIBAVADEMAB
    D.3.2Product code PRD/0000018152
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    Subcutaneous use
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product Yes
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Generalized lipodystrophy
    E.1.1.1Medical condition in easily understood language
    Extremely Low Body Fat
    E.1.1.2Therapeutic area Diseases [C] - Nutritional and Metabolic Diseases [C18]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level HLT
    E.1.2Classification code 10059674
    E.1.2Term Inborn errors of lipid metabolism
    E.1.2System Organ Class 100000004850
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To assess the safety and tolerability of treatment with mibavademab in individuals switching from treatment with metreleptin
    E.2.2Secondary objectives of the trial
    1. To describe glycemic control during 1 year of treatment with mibavademab in individuals switching from treatment with metreleptin
    2. To describe control of hypertriglyceridemia during 1 year of treatment with mibavademab in individuals switching from
    treatment with metreleptin
    3. To characterize the PK profile of mibavademab
    4. To evaluate the immunogenicity of mibavademab
    E.2.3Trial contains a sub-study No
    E.3Principal 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

    NOTE: Other protocol-defined inclusion criteria apply
    E.4Principal 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 (CKDEPI)/ 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 exclusion criteria apply
    E.5 End points
    E.5.1Primary end point(s)
    1. Incidence of treatment-emergent adverse events (TEAEs)
    2. Severity of TEAEs
    E.5.1.1Timepoint(s) of evaluation of this end point
    1-2. Up to week 68
    E.5.2Secondary end point(s)
    1. Change in Hemoglobin A1c (HbA1c)
    2. Occurrence of HbA1c <7%
    3. Occurrence of HbA1c <6.5%
    4. Occurrence of requiring therapy with insulin in participants treated with mibavademab
    5. Change in total insulin dose
    6. Change in fasting plasma glucose
    7. Percent change in fasting triglycerides
    8. Occurrence of fasting triglycerides <500 mg/dL in participants treated with mibavademab
    9. Occurrence of fasting triglycerides <200 mg/dL in participants treated with mibavademab
    10. Occurrence of fasting triglycerides <150 mg/dL in participants treated with mibavademab
    11. Concentrations of total mibavademab in serum
    12. Incidence of anti-drug antibodies (ADAs) to mibavademab
    13. Titer of ADAs to mibavademab
    14. Incidence of neutralizing antibodies (Nabs) to mibavademab
    E.5.2.1Timepoint(s) of evaluation of this end point
    1, 5-10. Baseline, week 20 and week 52
    2-4. Week 20 and week 52
    11-14. Up to week 68
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others Yes
    E.6.13.1Other scope of the trial description
    Tolerability, Immunogenicity
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other Yes
    E.8.1.7.1Other trial design description
    Single-arm
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial1
    E.8.3 Will this trial be conducted at a single site globally? No
    E.8.4 Will this trial be conducted at multiple sites globally? Yes
    E.8.6 Trial involving sites outside the EEA
    E.8.6.2Trial being conducted completely outside of the EEA Yes
    E.8.6.3Specify the countries outside of the EEA in which trial sites are planned
    United States
    E.8.7Trial has a data monitoring committee No
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months6
    E.8.9.2In all countries concerned by the trial days23
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 1
    F.1.1.1In Utero No
    F.1.1.1.1Number of subjects for this age range: 0
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.2.1Number of subjects for this age range: 0
    F.1.1.3Newborns (0-27 days) No
    F.1.1.3.1Number of subjects for this age range: 0
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.4.1Number of subjects for this age range: 0
    F.1.1.5Children (2-11years) No
    F.1.1.5.1Number of subjects for this age range: 0
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 1
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 7
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 1
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception No
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    under age
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.2 For a multinational trial
    F.4.2.2In the whole clinical trial 9
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    None
    G. Investigator Networks to be involved in the Trial
    H.4 Third Country in which the Trial was first authorised
    H.4.1Third Country in which the trial was first authorised: United States
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