Effects of Human Leptin Replacement

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

NCT00657605

1R0IDK58851-01 (Other Identifier)

20060282

Details and patient eligibility

About

This study will test the hypothesis that leptin contributes to the regulation of the dynamics of human endocrine function.

Full description

The study aims to elucidate the role of leptin in the regulation of human endocrine function approached by a carefully designed, prospective clinical study of the rapidly-sampled dynamics of endocrine rhythms during the course of leptin-replacement treatment in the only three adult individuals identified in the world so far who are leptin-naive due to a functional leptin gene mutation. A study of leptin-naive subjects avoids all confounding factors and pitfalls, because the only bioactive leptin to which they will be exposed is the exogenously administered recombinant protein. Thus, this proposal will permit us to ascertain the endocrine effects of human leptin.

Enrollment: 3 patients

Sex: All

Ages: 18 to 80 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Congenital leptin deficiency (these are only 3 adult individuals in the world that have been identified as leptin-naïve thus far).

Exclusion criteria

• Pregnant, trying to become pregnant, breast-feeding an infant or sexually active women, not using contraception.
• Subjects with hemoglobin levels below 12 g/dl.
• Subjects whose body contains a ferromagnetic implanted device that might produce a safety hazard during fMRI.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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3 participants in 1 patient group

Recombinant methionyl human leptin

Experimental group

Description:

Participants with congenital leptin deficiency will receive the Recombinant methionyl human leptin intervention subcutaneously, once a day with a dose of 0.02 to 0.04 mg/kg (adjusted according to weight loss).

Treatment:

Drug: Recombinant methionyl human leptin