Effects of Human Leptin Replacement

Phase 2

Completed

• Conditions: Diabetes; Metabolic Syndrome; Obesity
• Interventions: Drug: Recombinant methionyl human leptin

• Registration Number: NCT00657605
• Lead Sponsor: University of Miami

Brief Summary

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

Detailed 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.

Study Timeline

• Study Start: 2001-06
• Study First Submitted: 2008-04-09
• Study First Submitted QC: 2008-04-09
• Study First Posted: 2008-04-14
• Primary Completion: 2010-05-04
• Study Completion: 2010-05-04
• Status Verified: 2020-02
• Results First Submitted: 2020-02-11
• Results First Submitted QC: 2020-02-11
• Last Update Submitted: 2020-02-11
• Results First Posted: 2020-02-13
• Last Update Posted: 2020-02-13

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 3

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Recombinant methionyl human leptin	Recombinant methionyl human leptin	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).

Primary Outcome Measures

Name	Time	Method
Change in Weight	Baseline, 107 months

Secondary Outcome Measures

Name	Time	Method
Change in Glucose Levels	Baseline, 107 months

Trial Locations

Locations (1)

University of Miami Miller School of Medicine, Center on Pharmacogenomics; 🇺🇸 Miami, Florida, United States