Mibavademab for adults and children with generalized lipodystrophy

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

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.

Why it matters

Who can join: the full rules

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 it's running

• University of Michigan
  Ann Arbor, Michigan, United States Recruiting
• UT Southwestern Medical Center
  Dallas, Texas, United States Recruiting
• Walter Cantidio University Hospital, Federal University of Ceara - Ebserh
  Fortaleza, Ceará, Brazil Recruiting
• Centro de Pesquisas Clinicas de Natal (Cepclin)
  Natal, Rio Grande do Norte, Brazil Recruiting
• UPCO - HCPA - Universidade Federal do Rio Grande do Sul - UFRGS
  Porto Alegre, Rio Grande do Sul, Brazil Recruiting
• Hospital Das Clinicas - School of Medicine Ribeirao Preto, University of Sao Paulo
  Ribeirão Preto, São Paulo, Brazil Recruiting

Study contacts

• Study coordinator: Clinical Trials Administrator
• Email: clinicaltrials@regeneron.com
• Phone: 844-734-6643

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Mibavademab for adult women with functional hypothalamic amenorrhea

  This trial tests whether the experimental drug mibavademab helps adult women with functional hypothalamic amenorrhea make the hormones needed to resume ovulation and menstrual cycles.

  Recruiting at 10 sites Phase 2 Regeneron Pharmaceuticals · Phoenix, Arizona and 10 other locations · NCT07235917

• Mibavademab for obesity caused by leptin (LEP) gene mutations in children, adolescents, and adults

  This trial will try mibavademab in children, adolescents, and adults with leptin gene–related obesity to help control body weight and check safety.

  Recruiting at 3 sites Phase 3 Regeneron Pharmaceuticals · Ulm and 2 other locations · NCT07220772

• ALN-CIDEB in adults with MASLD to see if it helps the liver

  This study tests whether ALN-CIDEB can help adults with MASLD and its liver damage compared to placebo.

  Recruiting at 4 sites Phase 3 Regeneron Pharmaceuticals · Miami, Florida and 3 other locations · NCT07854782

• Olatorepatide vs placebo for adults with moderate-to-severe hidradenitis suppurativa who are overweight

  This study tests whether olatorepatide can help people with hidradenitis suppurativa who are overweight or obese.

  Recruiting Phase 2 Regeneron Pharmaceuticals · The Bronx, New York · NCT07830329

• Three antibodies plus heparin in adults with end-stage kidney disease on dialysis

  This study tests three investigational antibodies (REGN9933, REGN7508, REGN9533) in adults with end-stage kidney disease on hemodialysis who are already using heparin to prevent clots.

  Recruiting Phase 1 Regeneron Pharmaceuticals · Ghent, Oost-Vlaanderen · NCT07759167

• REGN17235 for CCUS and LR-MDS with SF3B1 mutation

  This study tests REGN17235 in adults with CCUS or LR-MDS carrying an SF3B1 mutation to see if it is safe and tolerable and to get early signals of efficacy.

  Recruiting at 2 sites Phase 1 Regeneron Pharmaceuticals · Ghent and 1 other locations · NCT07753148

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.