Dettagli per singolo studio

DEC-NET Serial number FR296

Published online	22/07/2004 15.16.00
Last updated	01/09/2005 15.28.06

Other protocol ID number	20010222
Current trial status	Complete(closed to recruitment of participants: follow-up complete)
Major Disease (ICD9 class)	LIPODYSTROPHY
Experimental drug
R-metHuLeptine Treatment regimen (dosage and duration) R-metHuLeptin is administered once a day by subcutaneous injection at doses predicted to achieve 50%, 100% and 200% of normal leptin levels.The dose of leptin needed to achieve a normal leptin concentration (100%) will be 0.03mg/kg of lean body weight.
Gender	Both
Age (range)	18 month-15 year
Eligibility criteria
Inclusion criteria
- children with congenital lipoatrophy - fasting serum leptin level below 3ng/ml before entry - hypertriglyceridemia (> 2mmol/l)and/or hyperinsulinemia (> 10mU/l) - the patients should follow their usual diet for at least two weeks before inclusion - informed consent signed by both parents of each child
Exclusion criteria
- Patients unable to follow the treatment (one injection per day) - Family unable to provide informed consent - Patients presenting an underlying disease that could affect the metabolism - Patients receiving anti-diabetic treatment of any kind - Patients with renal failure, as defined by a serum creatinine level > 120 ľmol/l - Patients with lipodystrophic syndrome under HIV antiretroviral treatment - Patients with an history of anaphylaxis or anaphylactoid-like reactions or a known hypersensitivity to E. Coli derived proteins - Women in their reproductive years undergoing pregnancy, or lactating, or not using an effective method of birth control - Known HIV positive patients - Known HCV positive patients (or other infectious liver disease) - Any other condition that, in the opinion of the investigator would impede completion of the study
Trial design/methodology
Phase	2
Kind of study	Efficacy
Design
Purpose of study
To measure the effects of leptin replacement therapy in non-diabetic children with congenital lipoatrophy




Summary of study design, objectives, and ongoing research findings
Congenital lipodystrophy is a very rare autosomal recessive disease with very low leptine levels. In the present trial, R-metHuLeptin replacement therapy is administered once a day by subcutaneous injection for 4 months to 10 affected - but not diabetic - children to improve insulin sensitivity and hepatic steatosis.