Effect of Leptin Therapy in the Treatment of Severe Insulin Resistance
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|ClinicalTrials.gov Identifier: NCT00085982|
Recruitment Status : Recruiting
First Posted : June 21, 2004
Last Update Posted : December 14, 2020
Leptin is a hormone produced by the fat cells that researchers have shown to play a role in controlling appetite. Certain people with severe insulin resistance have little or no leptin.
The purpose of this study is to investigate people whose leptin levels have been found to be lower than 85 percent of the general population. Researchers will determine whether insulin levels in these participants improve when they are treated with genetically engineered leptin.
Study participants must be age 8 years or older and must have severe insulin resistance syndrome and leptin deficiency. During an initial 7-day visit to the Clinical Center, researchers will evaluate participants' metabolic parameters, such as insulin responsiveness, lipid levels, appetite, and hormone levels. After taking these tests, participants will self-inject doses of leptin once or twice a day and will be monitored for treatment outcomes as well as side effects via follow-up visits. These inpatient follow-up visits will involve both blood tests and imaging studies at the Clinical Center at 4, 8, and 12 months after the initial visit, and every 6 months thereafter.
|Condition or disease||Intervention/treatment||Phase|
|Severe Insulin Resistance||Drug: Metreleptin||Phase 2|
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.
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.
Primary Endpoint: Hemoglobin A1c
Secondary Endpoints: fasting plasma glucose, fasting insulin, glucose/insulin area under the curve during oral glucose tolerance test.
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).
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||20 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Pase II Trial of Effect of Leptin Therapy in Severe Insulin Resistance|
|Actual Study Start Date :||August 21, 2003|
|Estimated Primary Completion Date :||January 1, 2030|
|Estimated Study Completion Date :||January 1, 2030|
Experimental: Leptin Treatment
300 mg of study drug administered via SC injections.
Administered SQ twice/day to achieve physiological concentrations that will be effective in improving the severe state of insulin resistance seen in patients with genetic defects on their insulin rece
- Improvements in HbA1c, fasting blood glucose, and fasting insulin levels [ Time Frame: Every 6-12 months ]Lower HbA1C, lower blood glucose values, & lower insulin levels.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00085982
|Contact: Megan S Startzell, R.N.||(301) firstname.lastname@example.org|
|Contact: Rebecca J Brown, M.D.||(301) email@example.com|
|United States, Maryland|
|National Institutes of Health Clinical Center, 9000 Rockville Pike||Recruiting|
|Bethesda, Maryland, United States, 20892|
|Contact: For more information at the NIH Clinical Center contact Office of Patient Recruitment (OPR) 800-411-1222 ext TTY8664111010 firstname.lastname@example.org|
|Principal Investigator:||Rebecca J Brown, M.D.||National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)|