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

Sponsor

Weill Medical College of Cornell University (Other)

Overall Status

Unknown status

CT.gov ID

NCT00603109

Collaborator

National Institutes of Health (NIH) (NIH), PWSAUSA (Other)

Enrollment

Location

Arms

Duration (Months)

1.5

Patients Per Site Per Month

Study Details

Study Description

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 Design

Study Type:

Interventional

Anticipated 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 :

Aug 1, 2007

Anticipated Primary Completion Date :

Jun 1, 2008

Anticipated Study Completion Date :

Aug 1, 2008

Arms and Interventions

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 Names: 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

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 Names:

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

Outcome Measures

Primary Outcome Measures

Body Weight and Body fat mass [6 months]

Secondary Outcome Measures

IGF-1, Leptin, Ghrelin, Serum Lipids and insulin sensitivity [6 months]

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years to 35 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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:

small hands and feet, 2) skin problems, 3) behavioral problems related to food, and
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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	New York Presbyterian Hospital-Weill Cornell Medical College	New york	New York	United States	10065

Sponsors and Collaborators

Weill Medical College of Cornell University
National Institutes of Health (NIH)
PWSAUSA

Investigators

Principal Investigator: Roja Motaghedi, MD, NYPH-Weill Cornell Medical College
Principal Investigator: Moris Angulo, MD, Winthrop University Hospital

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

, ,

ClinicalTrials.gov Identifier:

NCT00603109

Other Study ID Numbers:

0611008841

First Posted:

Jan 28, 2008

Last Update Posted:

Jan 4, 2011

Last Verified:

Jan 1, 2011

Keywords provided by , ,

Body weight, fat mass, growth hormone, ghrelin

Additional relevant MeSH terms:

Prader-Willi Syndrome

Syndrome

Weight Gain

Rimonabant

Study Results

No Results Posted as of Jan 4, 2011