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Effects of Human Leptin Replacement

The recruitment status of this study is unknown. The completion date has passed and the status has not been verified in more than two years.
Verified April 2008 by University of Miami.
Recruitment status was:  Active, not recruiting
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Information provided by:
University of Miami Identifier:
First received: April 9, 2008
Last updated: April 14, 2008
Last verified: April 2008
This study will test the hypothesis that leptin contributes to the regulation of the dynamics of human endocrine function.

Condition Intervention Phase
Obesity Metabolic Syndrome Diabetes Drug: Recombinant methionyl human leptin Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Effects of Human Leptin Replacement

Resource links provided by NLM:

Further study details as provided by University of Miami:

Primary Outcome Measures:
  • Weight loss [ Time Frame: Before and every 6 months after treatment is initiated, from 2001 to 2010 ]

Secondary Outcome Measures:
  • Improvement of metabolic parameters [ Time Frame: Before and every 6 months after treatment is initiated, from 2001 to 2010 ]

Enrollment: 3
Study Start Date: June 2001
Estimated Study Completion Date: December 2010
Estimated Primary Completion Date: January 2010 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: 1
Three adult patients with congenital leptin deficiency.
Drug: Recombinant methionyl human leptin
Recombinant methionyl human leptin, subcutaneous, once a day, 0.02 to 0.04 mg/kg (adjusted according to weight loss), indeterminate duration.
Other Names:
  • Metreleptin
  • r-metHuLeptin

Detailed 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.


Ages Eligible for Study:   18 Years to 80 Years   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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.
  Contacts and Locations
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Please refer to this study by its identifier: NCT00657605

United States, Florida
University of Miami Miller School of Medicine, Center on Pharmacogenomics
Miami, Florida, United States, 33136
Sponsors and Collaborators
University of Miami
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Principal Investigator: Julio Licinio, MD University of Miami
  More Information


Publications automatically indexed to this study by Identifier (NCT Number):
Responsible Party: Julio Licinio. MD, University of Miami Identifier: NCT00657605     History of Changes
Other Study ID Numbers: 20060282
Study First Received: April 9, 2008
Last Updated: April 14, 2008

Keywords provided by University of Miami:
Congenital leptin deficiency
Metabolic syndrome

Additional relevant MeSH terms:
Metabolic Syndrome X
Insulin Resistance
Glucose Metabolism Disorders
Metabolic Diseases processed this record on August 18, 2017