This study is currently recruiting participants.
Number
15-DK-0003
Sponsoring Institute
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Recruitment Detail
Type: Participants currently recruited/enrolled Gender: Male & Female Min Age: 6 mo Max Age: 98 Years
Referral Letter Required
Yes
Population Exclusion(s)
None
Keywords
Diabetes; Leptin; Lipodystrophy; Hypertriglyceridemia
Recruitment Keyword(s)
Condition(s)
Lipodystrophy; Diabetes; Hyperlipidemia
Investigational Drug(s)
Metreleptin
Investigational Device(s)
Intervention(s)
Drug: Metreleptin
Supporting Site
National Institute of Diabetes and Digestive and Kidney Diseases
- Generalized 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 ages 6 months and older with generalized lipodystrophy who:
- have received metreleptin through NIH studies AND
- cannot get it through approved or compassionate use mechanisms in their home country.
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. 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.
--Back to Top--
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).
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).
Principal Investigator
Referral Contact
For more information: