Compassionate Use of Metreleptin in Previously Treated People With Generalized Lipodystrophy

Launched by NATIONAL INSTITUTE OF DIABETES AND DIGESTIVE AND KIDNEY DISEASES (NIDDK) · Oct 10, 2014

Keywords

ClinConnect Summary

This clinical trial is studying a medication called metreleptin to see how safe and effective it is for people with generalized lipodystrophy, a condition that can lead to high blood fat levels and diabetes. The goal is to help those who have already tried metreleptin in earlier studies but cannot access it in their home countries. Eligible participants are anyone aged 6 months and older who have this condition and cannot get the medication through other means.

If you or a family member decides to participate, you would visit the National Institutes of Health (NIH) about every six months in the first year and then once or twice a year after that. During these visits, you will receive the medicine to take home, along with several tests, including blood tests and ultrasounds, to monitor your health. There may also be financial assistance for travel within the U.S. It’s important to note that participants must continue seeing their local doctors regularly and have blood tests every three to six months. Overall, this trial aims to provide valuable information about how metreleptin can improve health for individuals with generalized lipodystrophy.

Gender

ALL

Eligibility criteria

• * INCLUSION CRITERIA:
• • 1. Age greater than or equal to 6 months.
• • 2. Generalized lipodystrophy (either congenital or acquired).
• • 3. Those who cannot obtain metreleptin through approved or compassionate use mechanisms in their home country.
• EXCLUSION CRITERIA:
• • 1. Availability of metreleptin to the patient either as an approved drug, or through local compassionate use or expanded access programs.
• • 2. Known HIV infection or HIV-associated lipodystrophy.
• • 3. Any medical condition or medication that will increase risk to the subject.
• • 4. Current alcohol or substance abuse.
• • 5. Subjects who have a known hypersensitivity to E. coli derived proteins (as leptin is derived from such proteins).