Effect of Metreleptin Therapy in the Treatment of Severe Insulin Resistance (NCT00085982) | Clinical Trial Compass
Active — Not RecruitingPhase 2
Effect of Metreleptin Therapy in the Treatment of Severe Insulin Resistance
United States11 participantsStarted 2003-08-21
Plain-language summary
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).
Who can participate
Age range
5 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
* 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.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Change in HbA1C
Timeframe: Change at month 12 from baseline
Trial details
NCT IDNCT00085982
SponsorNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)