Effects of Leptin Replacement in Children

University of Miami1 enrolled

Overview

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

The proposed study of the treatment of a child with congenital leptin deficiency will permit to elucidate key aspects of human endocrine function, and will give new insights on the role of leptin in human endocrine regulation.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Obesity Metabolic Syndrome Diabetes

Interventions

Recombinant methionyl human leptinDRUG

Recombinant methionyl human leptin, subcutaneous, once a day, 0.02 to 0.04 mg/kg (adjusted according to weight loss), indeterminate duration.

Eligibility

Sex: MALEMin age: 5 YearsMax age: 18 Years

Medical Language ↔ Plain English

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

Locations (1)

University of Miami, Center on Pharmacogenomics

Miami, Florida, United States

Outcomes

Primary Outcomes

Weight

Time frame: Baseline, 58 months

Secondary Outcomes

Glucose Levels

Time frame: Baseline, 58 months

Bone Mineral Density

Time frame: Baseline, 58 months

Data from ClinicalTrials.gov

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