Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu
Help guide our efforts to modernize ClinicalTrials.gov.
Send us your comments by March 14, 2020.

Effects of Leptin Replacement in Children

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT00659828
Recruitment Status : Completed
First Posted : April 16, 2008
Last Update Posted : January 10, 2020
Sponsor:
Collaborator:
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Information provided by (Responsible Party):
Richard Bookman, University of Miami

Brief Summary:
To assess the endocrine and immune effects of leptin replacement in leptin-deficient children, from a consanguineous Turkish family. The hypothesis is that leptin replacement will have significant effects on immune and endocrine function.

Condition or disease Intervention/treatment Phase
Obesity Metabolic Syndrome Diabetes Drug: Recombinant methionyl human leptin Phase 2

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.

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 1 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Effects of Leptin Replacement in Children
Actual Study Start Date : June 2005
Actual Primary Completion Date : April 16, 2010
Actual Study Completion Date : April 16, 2010

Resource links provided by the National Library of Medicine

Drug Information available for: Metreleptin

Arm Intervention/treatment
Experimental: Treatment Group
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




Primary Outcome Measures :
  1. Weight [ Time Frame: Before and every 6 months after treatment is initiated, during 5 years ]

Secondary Outcome Measures :
  1. Endocrine parameters [ Time Frame: Before and every 6 months after treatment is initiated, during 5 years ]
  2. Bone mineral density [ Time Frame: Before and every 6 months after treatment is initiated, during 5 years ]
  3. Immune function [ Time Frame: Before and every 6 months after treatment is initiated, during 5 years ]


Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   5 Years to 18 Years   (Child, Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00659828


Locations
Layout table for location information
United States, Florida
University of Miami, Center on Pharmacogenomics
Miami, Florida, United States, 33132
Sponsors and Collaborators
University of Miami
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Investigators
Layout table for investigator information
Principal Investigator: Julio Licinio, MD University of Miami

Publications:

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Layout table for additonal information
Responsible Party: Richard Bookman, Associate Professor, University of Miami
ClinicalTrials.gov Identifier: NCT00659828    
Other Study ID Numbers: 20060295
2R01DK058851-03 ( U.S. NIH Grant/Contract )
First Posted: April 16, 2008    Key Record Dates
Last Update Posted: January 10, 2020
Last Verified: January 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Richard Bookman, University of Miami:
Congenital leptin deficiency
Obesity
Metabolic Syndrome
Diabetes
Additional relevant MeSH terms:
Layout table for MeSH terms
Metabolic Syndrome
Insulin Resistance
Hyperinsulinism
Glucose Metabolism Disorders
Metabolic Diseases