Efficacy of Leptin Replacement in Treatment of Genetic and Acquired Lipodystrophies

Study ID
Study00000088

Cancer Related
No

Healthy Volunteers
No

Study Sites

Contact
Claudia Quittner
214-648-9296
claudia.quittner@utsouthwestern.edu

Principal Investigator
Abhimanyu Garg

Summary

The study will be conducted as an open-label observational study to test the safety and potential efficacy of long term leptin replacement in patients with partial lipodystrophy.Following a screening evaluation, patients who qualify will be continued on leptin. eligible patients will be treated with metreleptin initially for a period of 12 months, during which they will be evaluated at yearly intervals. if the patient maintains improvements in his/her metabolic parameters while on metreleptin, the patient will be invited to continue taking the study medication as long as astra Zeneca is willing to continue providing drug. During the long term extension, metabolic parameters will be monitored every 12 months.

Participant Eligibility

Inclusion criteria

1. Age > 2 years

2. Partial ipodystrophy, previously treated with metreleptin..

3. Evidence of benefit with metreleptin from baseline pre-treatment within the past year ( Subject needs to meet only one of the following):

a. Serum triglyceride reduction >= 30%.
b. Reduction in HgbA1c >= 1 %
c. Decrease in daily insulin requirements of >= 40 %
d. Decrease in episodes of acute pancreatitis
e. Improvement in steatohepatitis
f. The withdrawal of metreleptin therapy leads to (or led to) marked worsening of metabolic parameters.