Partial Lipodystrophy Clinical Trial
Official title:
Phase II Study of Growth Hormone Inhibition Using Pegvisomant In Severe Insulin Resistance
Background: Lipodystrophy (LD) syndromes are a group of rare disorders that affect how a person s body can store and use fat tissue. Many people with LDs become severely insulin resistant. Some people are insulin resistant because of a variant in the insulin receptor gene. Insulin resistance causes many health problems. Objective: To learn if blocking the effects of growth hormone in the body will help people with severe insulin resistance. Eligibility: Adults aged 18 to 65 years with either a known variant in the insulin receptor gene or with a diagnosis of partial LD. Design: Participants will have 2 hospital stays, about 1 month apart. Each stay will be 3 or 4 nights. During each hospital stay, participants will have many tests. They will have a physical exam with blood tests. They will have all of their urine collected for a 24-hour period. They will have scans to measure their muscle, bone, and fat tissues. They will have tests to measure metabolism and insulin sensitivity. They may have an optional biopsy of fat tissue. During the first hospital visit, participants will learn how to give themselves shots of a drug (pegvisomant) that blocks growth hormone. The drug is injected under the skin. Participants will continue to give themselves these shots once a day at home. After the first hospital visit, participants will talk on the phone with members of the study team once each week. After 2 weeks they will have blood drawn for tests. Participants will stop the shots after the second hospital visit.
STUDY DESCRIPTION The role of growth hormone (GH) in mediating pathological consequences of inadequate lipid storage will be studied in rare patient populations with high lipolysis and severe metabolic syndrome. Specifically, patients with partial lipodystrophy and pathogenic variants in the insulin receptor gene (INSR) will be studied before and after 1 month of administration of pegvisomant (a GH receptor blocker). OBJECTIVES Primary Objective: Establish proof of concept that GH blockade reduces adipose tissue lipolysis in humans with severe insulin resistance. Secondary Objectives: Determine the effects of pegvisomant on lipolytic products and IGF-1. ENDPOINTS Primary Endpoint: Adipose tissue lipolysis measured by glycerol and palmitate rates of appearance using stable isotope tracers. Secondary Endpoints: Free fatty acids (FFA), glycerol, IGF-1. ;
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