Efficacy of Leptin Replacement in Treatment of Genetic and Acquired Lipodystrophies

Study ID

Cancer Related

Healthy Volunteers

Study Sites

Claudia Quittner

Principal Investigator
Abhimanyu Garg


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 different types of lipodystrophy.Following a screening evaluation, patients will be followed for a 4-week pre-baseline period, if feasible, without changing hypoglycemic and lipid lowering drugs in order to establish a baseline state. eligible patients will be treated with metreleptin initially for a period of 12 months, during which they will be evaluated at 3 month intervals. if the patient shows improvements in his/her metabolic parameters while on metreleptin, the patient will be invited to continue taking the study medication.During the long term extension, metabolic parameters will be monitored every 6 months.
in subjects who have not been treated with metreleptin previously, metreleptin therapy will be started at a dose to attain 100% of the normal leptin concentration and after 1 month, the dose will be increased to achieve 200% of the normal dose. Patients who have previously participated in leptin-replacement trials will continue metreleptin therapy at 200% of estimated replacement dose.

Participant Eligibility

1. Age> 2 years
2.Partial and generalized lipodystrophy,either genetic or acquired
3.Serum leptin levels less than 7.0ng/ml in females and less than 4.0ng/ml in males.
4. Presenceof atleast one of the following metabolic abnormalities:
a. Type 2 Diabetes mellitus- HbA1C.8%
b. Fasting serum insulin >30uU/ml
c. Fasting serum triglycerides> 300 mg/dl