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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00603109
Recruitment Status : Unknown
Verified January 2011 by Weill Medical College of Cornell University.
Recruitment status was:  Recruiting
First Posted : January 28, 2008
Last Update Posted : January 4, 2011
National Institutes of Health (NIH)
Information provided by:
Weill Medical College of Cornell University

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.

Condition or disease Intervention/treatment Phase
Prader-willi Syndrome Drug: rimonabant Drug: placebo Phase 3

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 18 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Effect of Rimonabant, a Cannabinoid Receptor 1 Antagonist on Weight Gain and Body Composition in Adults With Prader Willi Syndrome.
Study Start Date : August 2007
Estimated Primary Completion Date : June 2008
Estimated Study Completion Date : August 2008

Arm Intervention/treatment
Experimental: I
Subjects receive rimonabant 20 mg per day PO
Drug: rimonabant
rimonabant capsule 20 mg per day PO for 6 months
Other Name: Acomplia

Placebo Comparator: II
Subjects take placebo capsule one a day PO
Drug: placebo
Subjects take 20 mg placebo capsule one a day PO for 6 months

Primary Outcome Measures :
  1. Body Weight and Body fat mass [ Time Frame: 6 months ]

Secondary Outcome Measures :
  1. IGF-1, Leptin, Ghrelin, Serum Lipids and insulin sensitivity [ Time Frame: 6 months ]

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Ages Eligible for Study:   18 Years to 35 Years   (Adult)
Sexes Eligible for Study:   All
Accepts 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 exclusion criteria for this study.
  4. Subjects with Prader Willi Syndrome who are on other medications including growth hormone therapy, anti epileptic medications, or antipsychotic medications.
  5. The presence of moderate to severe renal or liver disease. Mild elevations of liver enzymes are not exclusive.
  6. Subjects who are on any other research or weight loss medication

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00603109

Contact: Roja Motaghedi, MD 212-746-3462
Contact: Moris Angulo, MD 516-663-3090

United States, New York
New York Presbyterian Hospital-Weill Cornell Medical College Recruiting
New york, New York, United States, 10065
Contact: Roja Motaghedi, MD    212-746-3462   
Contact: Elizabeth G Diaz, CPNP    212-746-3462   
Principal Investigator: Roja Motaghedi, MD         
Sub-Investigator: Elizabeth G Diaz, CPNP         
Sponsors and Collaborators
Weill Medical College of Cornell University
National Institutes of Health (NIH)
Principal Investigator: Roja Motaghedi, MD NYPH-Weill Cornell Medical College
Principal Investigator: Moris Angulo, MD Winthrop University Hospital

Responsible Party: Roja Motaghedi, MD, Weill Cornell Medical Center Identifier: NCT00603109     History of Changes
Other Study ID Numbers: 0611008841
First Posted: January 28, 2008    Key Record Dates
Last Update Posted: January 4, 2011
Last Verified: January 2011

Keywords provided by Weill Medical College of Cornell University:
Body weight, fat mass, growth hormone, ghrelin

Additional relevant MeSH terms:
Weight Gain
Prader-Willi Syndrome
Pathologic Processes
Body Weight Changes
Body Weight
Signs and Symptoms
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
Abnormalities, Multiple
Congenital Abnormalities
Chromosome Disorders
Genetic Diseases, Inborn
Nutrition Disorders
Cannabinoid Receptor Antagonists
Cannabinoid Receptor Modulators
Neurotransmitter Agents
Molecular Mechanisms of Pharmacological Action
Hormones, Hormone Substitutes, and Hormone Antagonists
Physiological Effects of Drugs