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Effect of Metreleptin Therapy in the Treatment of Severe Insulin Resistance

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT00085982
Recruitment Status : Active, not recruiting
First Posted : June 21, 2004
Results First Posted : December 15, 2021
Last Update Posted : May 3, 2022
Sponsor:
Information provided by (Responsible Party):
National Institutes of Health Clinical Center (CC) ( National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) )

Brief Summary:

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/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).


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/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).

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 11 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Phase II Trial of Effect of Metreleptin Therapy in Severe Insulin Resistance
Actual Study Start Date : August 21, 2003
Actual Primary Completion Date : October 23, 2019
Estimated Study Completion Date : January 1, 2030

Resource links provided by the National Library of Medicine


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 receptor mutation




Primary Outcome Measures :
  1. Change in HbA1C [ Time Frame: Change at month 12 from baseline ]
    Change in HbA1C at month 12 from baseline.


Secondary Outcome Measures :
  1. Change in Fasting Insulin Level [ Time Frame: Change at month 12 from baseline ]
    Change in fasting insulin level at month 12 from baseline

  2. Change in Fasting Blood Glucose [ Time Frame: Change at month 12 from baseline ]
    Change in fasting blood glucose at month 12 from baseline.



Information from the National Library of Medicine

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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
  • 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
  • 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.

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


Locations
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United States, Maryland
National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States, 20892
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)
  Study Documents (Full-Text)

Documents provided by National Institutes of Health Clinical Center (CC) ( National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) ):
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
Results First Posted: December 15, 2021
Last Update Posted: May 3, 2022
Last Verified: July 2021

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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