Effect of Metreleptin Therapy in the Treatment of Severe Insulin Resistance

Study Description: Patients with mutations of the insulin receptor have diabetes that is challenging to control with conventional therapies, leading to early morbidity and mortality. We hypothesize that recombinant leptin (metreleptin) in these patients will improve glycemia control. Objectives: Primary Objective: To determine if 1 year of metreleptin will improve glycemia control in patients with genetic defects of the insulin receptor. Secondary Objectives: To determine mechanisms by which metreleptin improves glycemia. Endpoints: Primary Endpoint: Hemoglobin A1c. Secondary Endpoints: fasting plasma glucose, fasting insulin/C-peptide, glucose/insulin/C-peptide area under the curve during oral glucose tolerance test. Study Population: 20 male or female patients with mutations of the insulin receptor, age (Bullet)5 years, at the NIH Clinical Center. Description of Sites/Facilities Enrolling Participants: Description of Study Intervention: NIH Clinical Center Open label study of metreleptin, 0.2 mg/kg/day (max dose 0.24 mg/kg/day).

NCT Number	NCT00085982
Lead Sponsor	National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Conditions	Severe Insulin Resistance
Enrollment	11 participants
Start Date	2003-08-21
Primary Completion	2019-10-23 (estimated)
Study Completion	2030-01-01 (estimated)
Updated on ClinicalTrials.gov	2025-01-30