Efficacy and Safety of Mibavademab in Adult and Pediatric Patients With Generalized Lipodystrophy
- Conditions
- Generalized Lipodystrophy
- Interventions
- Drug: Placebo
- Registration Number
- NCT07220785
- Lead Sponsor
- Regeneron Pharmaceuticals
- 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)
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- NOT_YET_RECRUITING
- Sex
- All
- Target Recruitment
- 28
1. 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 ≥2 and <12 years of age at screening
- No metabolic criteria or minimum weight for study entry is required
Key
- 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
NOTE: Other protocol defined inclusion/exclusion criteria apply.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Part A: Arm A Mibavademab - Part A: Arm B Mibavademab - Part A: Arm B Placebo - Part A: Arm C Mibavademab - Part A: Arm D Placebo - Part B Mibavademab -
- Primary Outcome Measures
Name Time Method Change in Hemoglobin A1c (HbA1c) Through 36 weeks of exposure to mibavademab Part A
Percent change in fasting Triglycerides (TG) Through 36 weeks of exposure to mibavademab Part A
Occurrence of Treatment Emergent Adverse Events (TEAEs) Up to 15 months Part B
Severity of TEAEs Up to 15 months Part B
Concentrations of total mibavademab in serum Up to 15 months Part B
- Secondary Outcome Measures
Name Time Method Change in HbA1c compared to placebo From week 56 to week 64 Part A
Change in HbA1c From baseline to week 52 Part B
Percent change in fasting TG compared to placebo From week 56 to week 64 Part A
Percent change in fasting TG From baseline to week 52 Part B
Occurrence of HbA1c <7% Through 36 weeks of exposure to mibavademab Part A
Occurrence of HbA1c <6.5% Through 36 weeks of exposure to mibavademab Part A
Occurrence of fasting TG <500 mg/dL Through 36 weeks of exposure to mibavademab Part A
Occurrence of TEAEs Through Week 72 Part A
Occurrence of fasting TG <200 mg/dL Through 36 weeks of exposure to mibavademab Part A
Occurrence of fasting TG <150 mg/dL Through 36 weeks of exposure to mibavademab Part A
Percent change in Liver Fat Content (LFC) From pre-mibavademab exposure to week 56 Part A
Change in fasting glucose From week 56 to week 64 Part A
Change in total daily insulin dose From baseline to week 52 Part B
Change in liver volume From pre-mibavademab exposure to week 56 Part A
Concentrations of total mibavademab in serum Through Week 72 Part A
Occurrence of anti-drug antibodies (ADA) to mibavademab Through Week 72 Part A
Occurrence of ADA to mibavademab Through Week 60 Part B
Titer of ADA to mibavademab Through Week 60 Part B
Severity of TEAEs Through Week 72 Part A