Effect of Rimonabant on Weight Gain and Body Composition in Adults With Prader Willi Syndrome

Study Overview
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Brief Summary
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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.

Official Title
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Effect of Rimonabant, a Cannabinoid Receptor 1 Antagonist on Weight Gain and Body Composition in Adults With Prader Willi Syndrome.

Conditions
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Prader-willi Syndrome

Intervention / Treatment
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* Drug: rimonabant
* Drug: placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: 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. Subjects must be 18 to 35 years of age and fairly cooperative with the study protocol. Subjects must have a BMI of at least 30 or more. Exclusion Criteria: Presence of pulmonary disease. Presence of any other abnormal endocrine findings, including abnormal thyroid function. 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 exclusion criteria for this study. Subjects with Prader Willi Syndrome who are on other medications including growth hormone therapy, anti epileptic medications, or antipsychotic medications. The presence of moderate to severe renal or liver disease. Mild elevations of liver enzymes are not exclusive. Subjects who are on any other research or weight loss medication

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 35 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: I<br>Subjects receive rimonabant 20 mg per day PO | Drug: rimonabant<br>* rimonabant capsule 20 mg per day PO for 6 months<br>* Other names: Acomplia;|
| Placebo Comparator: II<br>Subjects take placebo capsule one a day PO | Drug: placebo<br>* Subjects take 20 mg placebo capsule one a day PO for 6 months<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Body Weight and Body fat mass |  | 6 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| IGF-1, Leptin, Ghrelin, Serum Lipids and insulin sensitivity |  | 6 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Body weight, fat mass, growth hormone, ghrelin