Clinical Research Directory

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.

MEASuRE: Metreleptin Effectiveness And Safety Registry

The study is a post-authorization, prospective, voluntary registry of patients treated with commercial metreleptin including, but not limited to, patients in the US and EEA.

Study to Evaluate the Safety and Efficacy of Daily Subcutaneous Metreleptin Treatment in Subjects With PL

This is a Phase III, double-blind, placebo-controlled, safety and efficacy study of daily SC metreleptin in subjects with Partial Lipodystrophy.

Open-label Study to Evaluate Metreleptin in Patients With Partial Lipodystrophy

This is an Open Label, Phase IV, Post Authorisation Study to Evaluate the Efficacy, Safety and Immunogenicity of Daily Subcutaneous Metreleptin Treatment in people with Partial Lipodystrophy