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Protocol Details

Phase II Trial of Effect of Metreleptin Therapy in Severe Insulin Resistance

This study is currently recruiting participants.

Summary | Eligibility | Citations | Contacts

Summary

Number

03-DK-0257

Sponsoring Institute

National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

Recruitment Detail

Type: Participants currently recruited/enrolled
Gender: Male & Female
Min Age: 5
Max Age: N/A

Referral Letter Required

Yes

Population Exclusion(s)

None

Special Instructions

Currently Not Provided

Keywords

Rabson Mendenhall;
Type B Insulin Resistance;
Type A Insulin Resistance

Recruitment Keyword(s)

None

Condition(s)

Severe Insulin Resistance

Investigational Drug(s)

Metreleptin

Investigational Device(s)

None

Intervention(s)

Drug: Metreleptin

Supporting Site

National Institute of Diabetes and Digestive and Kidney Diseases

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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Eligibility

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.


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Citations:

Sekizkardes H, Chung ST, Chacko S, Haymond MW, Startzell M, Walter M, Walter PJ, Lightbourne M, Brown RJ. Free fatty acid processing diverges in human pathologic insulin resistance conditions. J Clin Invest. 2020 Jul 1;130(7):3592-3602. doi: 10.1172/JCI135431. PMID: 32191645; PMCID: PMC7324196.

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Contacts:

Principal Investigator

Referral Contact

For more information:

Rebecca J. Brown, M.D.
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
BG 10-CRC RM 6-5942
10 CENTER DR
BETHESDA MD 20814
(301) 594-0609
brownrebecca@mail.nih.gov

Megan S. Startzell, R.N.
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
National Institutes of Health
Building 10
Room 5-5750
10 Center Drive
Bethesda, Maryland 20892
(301) 402-6371
megan.startzell@nih.gov

Office of Patient Recruitment
National Institutes of Health Clinical Center (CC)
Building 61, 10 Cloister Court
Bethesda, Maryland 20892
Toll Free: 1-800-411-1222
Local Phone: 301-451-4383
TTY: 1-866-411-1010
PRPL@cc.nih.gov

Clinical Trials Number:

NCT00085982

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