Effects of Leptin Replacement in Children

The recruitment status of this study is unknown because the information has not been verified recently.
Verified April 2008 by University of Miami.
Recruitment status was  Active, not recruiting
Information provided by:
University of Miami
ClinicalTrials.gov Identifier:
First received: April 9, 2008
Last updated: April 14, 2008
Last verified: April 2008

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.

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

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Effects of Leptin Replacement in Children

Resource links provided by NLM:

Further study details as provided by University of Miami:

Primary Outcome Measures:
  • Weight [ Time Frame: Before and every 6 months after treatment is initiated, during 5 years ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Endocrine parameters [ Time Frame: Before and every 6 months after treatment is initiated, during 5 years ] [ Designated as safety issue: Yes ]
  • Bone mineral density [ Time Frame: Before and every 6 months after treatment is initiated, during 5 years ] [ Designated as safety issue: Yes ]
  • Immune function [ Time Frame: Before and every 6 months after treatment is initiated, during 5 years ] [ Designated as safety issue: Yes ]

Enrollment: 1
Study Start Date: June 2005
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
1 leptin-deficient male born in 2000
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:

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.


Ages Eligible for Study:   5 Years to 18 Years
Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No

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
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Please refer to this study by its ClinicalTrials.gov identifier: NCT00659828

United States, Florida
University of Miami, Center on Pharmacogenomics
Miami, Florida, United States, 33132
Sponsors and Collaborators
University of Miami
Principal Investigator: Julio Licinio, MD University of Miami
  More Information


Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Julio Licinio, University of Miami
ClinicalTrials.gov Identifier: NCT00659828     History of Changes
Other Study ID Numbers: 20060295, 2R01DK058851-03
Study First Received: April 9, 2008
Last Updated: April 14, 2008
Health Authority: United States: Food and Drug Administration

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

Additional relevant MeSH terms:
Metabolic Syndrome X
Glucose Metabolism Disorders
Insulin Resistance
Metabolic Diseases

ClinicalTrials.gov processed this record on August 27, 2015