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Compassionate Use of Metreleptin in Previously Treated People With Generalized Lipodystrophy

NCT02262832 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 24

Last updated 2026-05-12

No results posted yet for this study

Summary

Background:

\- 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.

Conditions

Interventions

DRUG

Metreleptin

administered subcutaneously 1-2 times/day

Sponsors & Collaborators

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

    lead NIH

Principal Investigators

  • Rebecca J Brown, M.D. · National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
6 Months
Max Age
98 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2014-10-09
Primary Completion
2027-07-31
Completion
2027-07-31
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02262832 on ClinicalTrials.gov