Prader-willi Syndrome
Conditions
Keywords
Body weight, fat mass, growth hormone, ghrelin
Brief summary
The purpose of this study is to evaluate the effect of rimonabant, a cannabinoid receptor-1 blocking drug, on the appetite, body weight, body fat and growth hormone level of subjects with Prader-Willi Syndrome (PWS). This will be a double blind placebo controlled clinical trial involving a total of 18 young adults aged 18 to 35 years with PWS. Patients will be divided in to the two groups of control and intervention, and treated with either placebo (inactive drug), or rimonabant 20 mg once a day for a total duration of 6 months. Body weight, fat distribution, objective and subjective assessment of the hunger, fasting blood sample for measurement of ghrelin and leptin (two hormones regulating appetite), serum lipids , IGF-1(growth hormone related protein), insulin and glucose concentrations will be measured upon enrollment, at 3 months, and at the end of the study. The proportion of body fat to muscle will be determined using a radiological technique, whole body dual-energy x-ray absorptiometry (DEXA) scan, and also by measurement of skin fold thickness, waist and hip circumference at the enrollment prior to the intervention, and at the end of the study.
Interventions
DRUGrimonabant
rimonabant capsule 20 mg per day PO for 6 months
DRUGplacebo
Subjects take 20 mg placebo capsule one a day PO for 6 months
Sponsors
National Institutes of Health (NIH)
PWSAUSA
Weill Medical College of Cornell University
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to 35 Years
Healthy volunteers
No
Inclusion criteria
1. Subjects will be selected if they have Prader Willi Syndrome previously confirmed by standard genetic testing (the DNA methylation test) or meet the clinical diagnostic criteria as follows :the presence of at least four of the six principal characteristics of PWS syndrome including 1) infantile hypotonia, 2) abnormal pubertal development, 3) obesity after early infancy, 4) dysfunctional central nervous system performance, 5) dysmorphic facial features, and 6) short stature. In addition, they must have one or more of the following characteristics commonly associated with PWS: 1) small hands and feet, 2) skin problems, 3) behavioral problems related to food, and 4) decreased pain sensitivity. 2. Subjects must be 18 to 35 years of age and fairly cooperative with the study protocol. 3. Subjects must have a BMI of at least 30 or more.
Exclusion criteria
1. Presence of pulmonary disease. 2. Presence of any other abnormal endocrine findings, including abnormal thyroid function. 3. Presence of significant behavioral problems or psychiatric illness including anxiety disorder and depression, interfering with the follow up of protocol. Any degree of depression and moderate to severe anxiety will be
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Body Weight and Body fat mass | 6 months |
Secondary
| Measure | Time frame |
|---|---|
| IGF-1, Leptin, Ghrelin, Serum Lipids and insulin sensitivity | 6 months |
Countries
United States
Outcome results
None listed