Diabetes, Metabolic Syndrome, Obesity
Conditions
Keywords
Congenital leptin deficiency, Obesity, Metabolic Syndrome, Diabetes
Brief summary
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.
Detailed 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.
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
MALE
Age
5 Years to 18 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Weight | Baseline, 58 months |
Secondary
| Measure | Time frame |
|---|---|
| Glucose Levels | Baseline, 58 months |
| Bone Mineral Density | Baseline, 58 months |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Recombinant Methionyl Human Leptin Recombinant methionyl human leptin: Recombinant methionyl human leptin, subcutaneous, once a day, 0.02 to 0.04 mg/kg (adjusted according to weight loss). | 1 |
| Total | 1 |
Baseline characteristics
| Characteristic | Recombinant Methionyl Human Leptin |
|---|---|
| Age, Categorical <=18 years | 1 Participants |
| Age, Categorical >=65 years | 0 Participants |
| Age, Categorical Between 18 and 65 years | 0 Participants |
| Ethnicity (NIH/OMB) Hispanic or Latino | 0 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 1 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Sex: Female, Male Female | 0 Participants |
| Sex: Female, Male Male | 1 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 1 |
| other Total, other adverse events | 0 / 0 |
| serious Total, serious adverse events | 0 / 0 |
Outcome results
Primary
Weight
Time frame: Baseline, 58 months
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Recombinant Methionyl Human Leptin | Weight | NA lbs |
Secondary
Bone Mineral Density
Time frame: Baseline, 58 months
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Recombinant Methionyl Human Leptin | Bone Mineral Density | NA g/cm^2 |
Secondary
Glucose Levels
Time frame: Baseline, 58 months
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Recombinant Methionyl Human Leptin | Glucose Levels | NA mg/dL |