Diabetes, Metabolic Syndrome, Obesity
Conditions
Keywords
Congenital leptin deficiency, Obesity, Metabolic syndrome, Diabetes
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.
Interventions
Recombinant methionyl human leptin, subcutaneous, once a day, 0.02 to 0.04 mg/kg (adjusted according to weight loss), indeterminate duration.
Sponsors
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
University of Miami
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 80 Years
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Change in Weight | Baseline, 107 months |
Secondary
| Measure | Time frame |
|---|---|
| Change in Glucose Levels | Baseline, 107 months |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| 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). | 3 |
| Total | 3 |
Baseline characteristics
| Characteristic | Recombinant Methionyl Human Leptin |
|---|---|
| Age, Categorical <=18 years | 0 Participants |
| Age, Categorical >=65 years | 0 Participants |
| Age, Categorical Between 18 and 65 years | 3 Participants |
| Ethnicity (NIH/OMB) Hispanic or Latino | 0 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 3 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Sex: Female, Male Female | 2 Participants |
| Sex: Female, Male Male | 1 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 3 |
| other Total, other adverse events | 0 / 0 |
| serious Total, serious adverse events | 0 / 0 |
Outcome results
Primary
Change in Weight
Time frame: Baseline, 107 months
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Recombinant Methionyl Human Leptin | Change in Weight | NA lbs |
Secondary
Change in Glucose Levels
Time frame: Baseline, 107 months
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Recombinant Methionyl Human Leptin | Change in Glucose Levels | NA mg/dL |