Phase II Trial of Effect of Metreleptin Therapy in Severe Insulin Resistance
Metreleptin
Hyperinsulinism+1
+ Insulin Resistance
+ Metabolic Diseases
Treatment Study
Summary
Study start date: August 21, 2003
Actual date on which the first participant was enrolled.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).
Protocol
This section provides details of the study plan, including how the study is designed and what the study is measuring.11 patients to be enrolled
Total number of participants that the clinical trial aims to recruit.Treatment Study
Eligibility
Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.Any sex
Biological sex of participants that are eligible to enroll.Over 5 Years
Range of ages for which participants are eligible to join.Healthy volunteers not allowed
If individuals who are healthy and do not have the condition being studied can participate.Conditions
Pathology
Criteria
* INCLUSION CRITERIA: * Provision of signed and dated informed consent form * Male or female, aged \> 5 years * Clinically significant, severe insulin resistance caused by a known or suspected defect in the insulin receptor * Presence of at least one of the following metabolic abnormalities: * Fasting insulin \>30 micro U/ml, or * Presence of diabetes as defined by the 2006 American Diabetes Association (ADA) criteria: * Fasting plasma glucose \>= 126 mg/dL * 2 hour plasma glucose \>= 200 mg/dL following a 75 gram (1.75g/kg if less than 40kg) oral glucose load, or * Diabetic symptoms with a random plasma glucose \>= 200 mg/dL EXCLUSION CRITERIA: * Pregnant at time of enrollment, women in their reproductive years who do not use an effective method of birth control, and women currently nursing or lactating within 6 weeks of having completed nursing. * Known infectious liver disease * Known HIV infection * Current alcohol or substance abuse * Active tuberculosis * Use of anorexigenic drugs * Other conditions which in the opinion of the clinical investigators would impede completion of the study. * Subjects who have a known hypersensitivity to E. Coli derived proteins.
Study Plan
Find out more about all the medication administered in this study, their detailed description and what they involve.One single intervention group is designated in this study
This study does not include a placebo group
Treatment Groups
Group I
ExperimentalStudy Objectives
Primary Objectives
Secondary Objectives
Study Centers
These are the hospitals, clinics, or research facilities where the trial is being conducted. You can find the location closest to you and its status.This study has 1 location
National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, United StatesSee the location