Effect of Metreleptin Therapy in the Treatment of Severe Insulin Resistance
Status:
Recruiting
Trial end date:
2030-01-01
Target enrollment:
Participant gender:
Summary
Study Description:
Patients with mutations of the insulin receptor have diabetes that is challenging to control
withconventional therapies, leading to early morbidity and mortality. We hypothesize that
recombinant leptin (metrel eptin) 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).
Phase:
Phase 2
Details
Lead Sponsor:
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)