A Two-Part, Randomized, Placebo-Controlled Phase 3 Study of the Efficacy, Safety, and Pharmacokinetics of Mibavademab in Patients With Generalized Lipodystrophy (LAGO)
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)
∙ 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 \<12 years of age at screening
• Weighing ≥7 kg at screening
• No metabolic criteria for study entry is required, as described in the protocol