Effects of Leptin Replacement in Children

University of Miami

Status and phase: Completed; Phase 2

Conditions: Diabetes; Obesity; Metabolic Syndrome

Treatments: Drug: Recombinant methionyl human leptin

Study type: Interventional

Funder types: Other; NIH

Identifiers

NCT00659828

20060295

2R01DK058851-03 (U.S. NIH Grant/Contract)

Details and patient eligibility

About

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.

Full description

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.

Enrollment: 1 patient

Sex: Male

Ages: 5 to 18 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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1 participants in 1 patient group

Recombinant Methionyl Human Leptin

Experimental group

Description:

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

Treatment:

Drug: Recombinant methionyl human leptin