Compassionate Use of Metreleptin in Previously Treated People With Partial Lipodystrophy (NCT02262806) | Clinical Trial Compass
Active — Not RecruitingPhase 2
Compassionate Use of Metreleptin in Previously Treated People With Partial Lipodystrophy
United States29 participantsStarted 2014-10-14
Plain-language 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.
Who can participate
Age range
6 Months – 98 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
* 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.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Serum hemoglobin A1C
Timeframe: every 6-12 months
2
Serum triglycerides
Timeframe: every 6-12 months
Trial details
NCT IDNCT02262806
SponsorNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)