This trial is active, not recruiting.

Conditions obesity, metabolic syndrome, diabetes
Treatment recombinant methionyl human leptin
Phase phase 2
Sponsor University of Miami
Collaborator National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Start date June 2005
End date January 2010
Trial size 1 participant
Trial identifier NCT00659828, 20060295, 2R01DK058851-03


We will assess the endocrine and immune effects of leptin replacement in leptin-deficient children, from a consanguineous Turkish family. We hypothesize that leptin replacement will have significant effects on immune and endocrine function.

United States No locations recruiting
Other countries No locations recruiting

Study Design

Endpoint classification safety/efficacy study
Intervention model single group assignment
Masking open label
Primary purpose treatment
1 leptin-deficient male born in 2000
recombinant methionyl human leptin Metreleptin
recombinant methionyl human leptin, subcutaneous, once a day, 0.02 to 0.04 mg/kg (adjusted according to weight loss), indeterminate duration.

Primary Outcomes

time frame: Before and every 6 months after treatment is initiated, during 5 years

Secondary Outcomes

Endocrine parameters
time frame: Before and every 6 months after treatment is initiated, during 5 years
Bone mineral density
time frame: Before and every 6 months after treatment is initiated, during 5 years
Immune function
time frame: Before and every 6 months after treatment is initiated, during 5 years

Eligibility Criteria

Male participants from 5 years up to 18 years old.

Inclusion Criteria: - Children with a functional leptin gene mutation from a consanguineous Turkish family. Only one leptin-naïve child from this family is alive and eligible. Exclusion Criteria: - N/A

Additional Information

Official title Effects of Leptin Replacement in Children
Principal investigator Julio Licinio, MD
Description The proposed study of the treatment of a child with congenital leptin deficiency will permit to elucidate key aspects of human endocrine and immune function, and will give new insights on the role of leptin in human endocrine regulation. Leptin administration in leptin-deficient children will possibly increase energy and fat metabolism by increasing sympathetic nervous system activity. To test this hypothesis, we will measure food intake, energy expenditure, body composition and sympathetic nervous system activity in patients homozygous due to a leptin gene mutation, before and throughout the leptin replacement therapy. Leptin modulates T-cell function and affects the phagocytic activity of macrophages. Immune function will be assessed during the course of this study. Specifically, tests for antibody, complement and phagocytic function, tests for T-cell immunity, flow cytometry, TREC PCR, thymus imaging studies will be performed. Antibody levels for pathogen organisms will be checked and the child will be vaccinated if needed. Leptin also has important roles on thyroid, adrenal and gonadal functions. Morevover, leptin is correlated with levels of lipids, glucose and insulin. To test the effects of leptin replacement in leptin-deficient humans, endocrine and metabolic parameters will be measured, before and during treatment. Leptin determines changes on bone mineral density. To evaluate these changes, bone function and densitometry will also be assessed in this leptin deficient child. Finally, leptin may have a role in brain growth and development. We will conduct volumetric brain imaging studies in this patient during the course of leptin replacement, ensuring safe exposure to radiation. Neuropsychological evaluation will also be undertaken.
Trial information was received from ClinicalTrials.gov and was last updated in April 2008.
Information provided to ClinicalTrials.gov by University of Miami.