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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
Sponsor:
Information provided by (Responsible Party):
National Institutes of Health Clinical Center (CC) ( National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) )

Study Description
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Brief Summary:

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

Detailed Description:

Study Description:

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.

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, glucose/insulin 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).

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 20 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: Leptin Treatment
300 mg of study drug administered via SC injections.
 Drug: Metreleptin
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


Outcome Measures
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Primary Outcome Measures :
  1. 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.


Eligibility Criteria
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Ages Eligible for Study:   5 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria
  • INCLUSION CRITERIA:
  • Provision of signed and dated informed consent form
  • Stated willingness to comply with all study procedures and availability for the duration of the study
  • 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 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
  • Psychiatric disorder impeding competence or compliance
  • Active tuberculosis
  • Use of anorexiogenic 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.
Contacts and Locations
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Information from the National Library of Medicine

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


Contacts
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Contact: Megan S Startzell, R.N. (301) 402-6371 megan.startzell@nih.gov
Contact: Rebecca J Brown, M.D. (301) 594-0609 brownrebecca@mail.nih.gov

Locations
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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    prpl@cc.nih.gov   
Sponsors and Collaborators
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Investigators
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Principal Investigator: Rebecca J Brown, M.D. National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
More Information
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Additional Information:

Publications:

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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Responsible Party: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
ClinicalTrials.gov Identifier: NCT00085982    
Other Study ID Numbers: 030257
03-DK-0257
First Posted: June 21, 2004    Key Record Dates
Last Update Posted: December 14, 2020
Last Verified: October 27, 2020

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by National Institutes of Health Clinical Center (CC) ( National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) ):
Rabson Mendenhall
Type B Insulin Resistance
Type A Insulin Resistance
Additional relevant MeSH terms:
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Insulin Resistance
Hyperinsulinism
Glucose Metabolism Disorders
Metabolic Diseases