Metreleptin for Insulin Resistance

Recruiting in Palo Alto (17 mi)

Overseen by Rebecca J Brown, M.D.

Age: Any Age

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Waitlist Available

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

No Placebo Group

Prior Safety Data

Approved in 2 Jurisdictions

Trial Summary

What is the purpose of this trial?

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).

Eligibility Criteria

This trial is for males and females over the age of 5 with severe insulin resistance due to insulin receptor mutations. Participants must have diabetes as defined by ADA criteria, with fasting insulin >30 micro U/ml or glucose levels indicating diabetes. Pregnant women, those not using birth control, or with certain infections like HIV are excluded.

Inclusion Criteria

Fasting insulin >30 micro U/ml

I have diabetes symptoms and my blood sugar level is 200 mg/dL or higher.

I have severe insulin resistance due to issues with my insulin receptor.

Exclusion Criteria

I have a liver infection.

Subjects who have a known hypersensitivity to E. Coli derived proteins

I am HIV positive.

Participant Groups

The study tests if metreleptin therapy (0.2 mg/kg/day) can improve blood sugar control in patients with genetic defects in the insulin receptor over a year. It's an open-label study at NIH Clinical Center focusing on changes in Hemoglobin A1c and other blood sugar markers.

Group I: Leptin Treatment Experimental Treatment 1 Intervention

300 mg of study drug administered via subcutaneous (SC) injections.

Metreleptin is already approved in United States, Canada for the following indications:

🇺🇸 Approved in United States as Myalept for:

• Complications of leptin deficiency in patients with congenital or acquired generalized lipodystrophy

🇨🇦 Approved in Canada as Myalept for:

• Complications of leptin deficiency in patients with congenital or acquired generalized lipodystrophy
• Patients with partial lipodystrophy

Find a Clinic Near You

Research Locations Nearby Select from list below to view details:

National Institutes of Health Clinical Center, 9000 Rockville Pike Bethesda, MD

Who Is Running the Clinical Trial?

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

References