Protocol Details
Compassionate Use of Metreleptin in Previously-Treated Patients with Partial Lipodystrophy
This study is NOT currently recruiting participants.
Summary
Number | 15-DK-0002 |
Sponsoring Institute | National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) |
Recruitment Detail | Type: No longer recruiting/follow-up only
Gender: Male & Female
Min Age: 6 Mo
Max Age: 98 Years |
Referral Letter Required | Yes |
Population Exclusion(s) | None |
Keywords | Lipodystrophy;
Leptin;
Hypertriglyceridemia;
Diabetes |
Recruitment Keyword(s) | None |
Condition(s) | Diabetes;
Lipodystrophy;
Hyperlipidemia |
Investigational Drug(s) | Metreleptin
|
Investigational Device(s) | None |
Intervention(s) | Drug: Metreleptin
|
Supporting Site | National Institute of Diabetes and Digestive and Kidney Diseases |
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.
Eligibility
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.
Citations:
Akinci B, Oral EA, Neidert A, Rus D, Cheng WY, Thompson-Leduc P, Cheung HC, Bradt P, Foss de Freitas MC, Montenegro RM, Fernandes VO, Cochran E, Brown RJ. Comorbidities and Survival in Patients With Lipodystrophy: An International Chart Review Study. J Clin Endocrinol Metab. 2019 Nov 1;104(11):5120-5135. doi: 10.1210/jc.2018-02730. PMID: 31314093; PMCID: PMC6760298.
Contacts:
Clinical Trials Number:
NCT02262806