Compassionate Use of Metreleptin in Previously Treated People With Partial Lipodystrophy

• Status: Active, not recruiting
• Trial end date: 2025-07-31
• Target enrollment: 0
• Participant gender: All

Summary

Background:

• Partial lipodystrophy can cause high blood fat levels and resistance to insulin. This can lead to health problems including diabetes. Researchers have found that the drug metreleptin improves health in people with this disease.

Objective:

• To test the safety and effectiveness of metreleptin.

Eligibility:

• People age 6 months and older with partial lipodystrophy who

• have received metreleptin through NIH studies and shown improvement AND

• cannot get metreleptin other ways.

Design:

• Participants will come to NIH approximately every 6 months during year one, then every 1 2 years. Financial assistance may be available for travel within the U.S.

• At visits, participants will get a supply of metreleptin to take home for daily injections, or it can be shipped to them inside the U.S. They will have:

• plastic catheter placed in an arm vein.

• blood tests, urine collection, and physical exam.

• oral glucose tolerance test, drinking a sweet liquid.

• ultrasound of the heart, liver, uterus, and ovaries. A gel and a probe are placed on the skin and pictures are taken of the organs.

• echocardiogram, which takes pictures of the heart with sound waves.

• Resting Metabolic Rate taken. A plastic hood is worn over the head while the oxygen they breathe is measured.

• Participants will have up to 3 DEXA scan x-rays per year.

• Participants may have:

• annual bone x-rays.

• liver biopsies every few years. A needle will be inserted into the liver to obtain a small piece. Participants will sign a separate consent for this.

• Participants must be seen regularly by their local doctors and have blood tests at least every 3-6 months at home.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

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

Criteria

• INCLUSION CRITERIA

• Age greater than or equal to 6 months

• Partial lipodystrophy (either genetic or acquired)

• Previously or currently treated with metreleptin under NIH study 02-DK-0022 and/or NIH study 13-DK-0057.

• Documented metabolic benefit from prior or current metreleptin treatment, defined as one or more of the following:

• TG reduction greater than or equal to 30% OR

• HbA1c reduction greater than or equal to 1% OR

• Decrease in insulin requirements greater than or equal to 40% OR

• Decrease in episodes of pancreatitis OR

• Improvement in steatohepatitis OR

• Withdrawal of metreleptin led to marked worsening of metabolic parameters

EXCLUSION CRITERIA

• Availability of metreleptin to the patient either as an approved drug, or through local compassionate use or expanded access programs.

• Known HIV infection or HIV-associated lipodystrophy.

• Psychiatric disorder impeding competence or compliance.

• Any medical condition or medication that will increase risk to the subject.

• Current alcohol or substance abuse.

• Subjects who have a known hypersensitivity to E. coli derived proteins.