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 2001
End date January 2010
Trial size 3 participants
Trial identifier NCT00657605, 1ROIDK58851-01, 20060282


This study will test the hypothesis that leptin contributes to the regulation of the dynamics of human 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
Three adult patients with congenital leptin deficiency.
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

Weight loss
time frame: Before and every 6 months after treatment is initiated, from 2001 to 2010

Secondary Outcomes

Improvement of metabolic parameters
time frame: Before and every 6 months after treatment is initiated, from 2001 to 2010

Eligibility Criteria

Male or female participants from 18 years up to 80 years old.

Inclusion Criteria: - Congenital leptin deficiency (these are only 3 adult individuals in the world that have been identified as leptin-naïve thus far). Exclusion Criteria: - Pregnant, trying to become pregnant, breast-feeding an infant or sexually active women, not using contraception. - Subjects with hemoglobin levels below 12 g/dl. - Subjects whose body contains a ferromagnetic implanted device that might produce a safety hazard during fMRI.

Additional Information

Official title Effects of Human Leptin Replacement
Principal investigator Julio Licinio, MD
Description Three separate, but complementary, specific aims, will elucidate the role of leptin in the regulation of (A) the human pituitary-adrenal axis, (B) the human pituitary-gonadal axis, and (C) the human pituitary-thyroid axis. These aims will be approached by a carefully designed, prospective clinical study of the rapidly-sampled dynamics of endocrine rhythms during the course of leptin-replacement treatment in the only three adult individuals identified in the world so far who are leptin-naive due to a functional leptin gene mutation. A study of leptin-naive subjects avoids all confounding factors and pitfalls, because the only bioactive leptin to which they will be exposed is the exogenously administered recombinant protein. Thus, this proposal will permit us to ascertain the endocrine effects of human leptin. The proposed studies will elucidate key aspects of human endocrine function and will give new insights on the role of leptin in human endocrine regulation. We also propose to perform structural MRI scans (on and off leptin replacement) to test the association of the changes in brain tissue composition with leptin replacement, and to test how leptin influences regional brain function during the presentation of food-related stimuli (pictures of food), which we will present to the patients along with neutral pictures (e.g., landscapes). We will test how this possible effect on brain function will be related to food craving or hunger. In yet another part of the study we will assess the kinetics of human recombinant leptin.
Trial information was received from ClinicalTrials.gov and was last updated in April 2008.
Information provided to ClinicalTrials.gov by University of Miami.