Effects of Human Leptin Replacement

• Sponsor: University of Miami (Other)
• Overall Status: Completed
• CT.gov ID: NCT00657605
• Collaborator: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) (NIH)
• Enrollment: 3
• Location: 1
• Arm: 1
• Actual Duration (Months): 107.1
• Patients Per Site Per Month: 0

Study Details

Study Description

Brief Summary

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

Condition or Disease	Intervention/Treatment	Phase
Obesity; Metabolic Syndrome; Diabetes	Drug: Recombinant methionyl human leptin	Phase 2

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 Design

• Study Type: Interventional
• Actual Enrollment: 3 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: Effects of Human Leptin Replacement
• Actual Study Start Date: Jun 1, 2001
• Actual Primary Completion Date: May 4, 2010
• Actual Study Completion Date: May 4, 2010

Arms and Interventions

Arm	Intervention/Treatment
Experimental: 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).	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

Outcome Measures

Primary Outcome Measures

1. Change in Weight [Baseline, 107 months]

Secondary Outcome Measures

1. Change in Glucose Levels [Baseline, 107 months]

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years to 80 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	University of Miami Miller School of Medicine, Center on Pharmacogenomics	Miami	Florida	United States	33136

Sponsors and Collaborators

• University of Miami
• National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

Investigators

• Principal Investigator: Julio Licinio, MD, University of Miami

Study Documents (Full-Text)

None provided.

More Information

Publications

• Strobel A, Issad T, Camoin L, Ozata M, Strosberg AD. A leptin missense mutation associated with hypogonadism and morbid obesity. Nat Genet. 1998 Mar;18(3):213-5.

• Responsible Party: Richard Bookman, Associate Professor, University of Miami
• ClinicalTrials.gov Identifier: NCT00657605
• Other Study ID Numbers: 20060282; 1R0IDK58851-01
• First Posted: Apr 14, 2008
• Last Update Posted: Feb 13, 2020
• Last Verified: Feb 1, 2020

• Individual Participant Data (IPD) Sharing Statement: No
• Plan to Share IPD: No
• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No
• Keywords provided by Richard Bookman, Associate Professor, University of Miami: Congenital leptin deficiency; Obesity; Metabolic syndrome; Diabetes
• Additional relevant MeSH terms: Metabolic Syndrome

Study Results

Participant Flow

Arm/Group Title	Recombinant Methionyl Human Leptin
Arm/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).
Period Title: Overall Study
STARTED	3
COMPLETED	3
NOT COMPLETED	0

Baseline Characteristics

Arm/Group Title	Recombinant Methionyl Human Leptin
Arm/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).
Overall Participants	3
Age (Count of Participants)
<=18 years	0 0%
Between 18 and 65 years	3 100%
>=65 years	0 0%
Sex: Female, Male (Count of Participants)
Female	2 66.7%
Male	1 33.3%
Ethnicity (NIH/OMB) (Count of Participants)
Hispanic or Latino	0 0%
Not Hispanic or Latino	3 100%
Unknown or Not Reported	0 0%

Outcome Measures

1. Primary Outcome

Title	Change in Weight
Time Frame	Baseline, 107 months

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Recombinant Methionyl Human Leptin
Arm/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).
Measure Participants	3
Number [lbs]	NA

2. Secondary Outcome

Title	Change in Glucose Levels
Time Frame	Baseline, 107 months

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Recombinant Methionyl Human Leptin
Arm/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).
Measure Participants	3
Number [mg/dL]	NA

Adverse Events

Time Frame	107 months
Adverse Event Reporting Description	Adverse Events not reported due to participant confidentiality.
Arm/Group Title	Recombinant Methionyl Human Leptin
Arm/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).
All Cause Mortality
	Recombinant Methionyl Human Leptin
	Affected / at Risk (%)	# Events
Total	0/3 (0%)
Serious Adverse Events
	Recombinant Methionyl Human Leptin
	Affected / at Risk (%)	# Events
Total	0/0 (NaN)
Other (Not Including Serious) Adverse Events
	Recombinant Methionyl Human Leptin
	Affected / at Risk (%)	# Events
Total	0/0 (NaN)

Limitations/Caveats

This is a rare disease population study. To protect participant privacy and maintain confidentiality, results will not be reported.

More Information

Certain Agreements

All Principal Investigators ARE employed by the organization sponsoring the study.

There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.