Efficacy of Leptin Replacement in Treatment of Genetic and Acquired Lipodystrophies

Study ID

Cancer Related

Healthy Volunteers

Study Sites

  • UT Southwestern-Clinical Translational Research Center (CTRC)
  • UT Southwestern University Hospital—St. Paul
  • Parkland Health & Hospital System

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