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
Sponsor:
Collaborator:
Information provided by:
University of Miami
ClinicalTrials.gov Identifier:
NCT00659828
First received: April 9, 2008
Last updated: April 14, 2008
Last verified: April 2008

April 9, 2008
April 14, 2008
June 2005
January 2010   (final data collection date for primary outcome measure)
Weight [ Time Frame: Before and every 6 months after treatment is initiated, during 5 years ] [ Designated as safety issue: Yes ]
Same as current
Complete list of historical versions of study NCT00659828 on ClinicalTrials.gov Archive Site
  • 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 ]
Same as current
Not Provided
Not Provided
 
Effects of Leptin Replacement in Children
Effects of Leptin Replacement in Children

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.

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.

Interventional
Phase 2
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
  • Obesity
  • Metabolic Syndrome
  • Diabetes
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
Experimental: 1
1 leptin-deficient male born in 2000
Intervention: Drug: Recombinant methionyl human leptin

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Active, not recruiting
1
December 2010
January 2010   (final data collection date for primary outcome measure)

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
Male
5 Years to 18 Years
No
Contact information is only displayed when the study is recruiting subjects
United States
 
NCT00659828
20060295, 2R01DK058851-03
Yes
Julio Licinio, University of Miami
University of Miami
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Principal Investigator: Julio Licinio, MD University of Miami
University of Miami
April 2008

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP