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

Primary Purpose

Prader-willi Syndrome

Status

Terminated

Phase

Phase 3

Locations

United States

Study Type

Interventional

Intervention

rimonabant

placebo

About this trial

This is an interventional treatment trial for Prader-willi Syndrome focused on measuring Body weight, fat mass, growth hormone, ghrelin

Eligibility Criteria

18 Years - 35 Years (Adult)All SexesDoes not accept 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: 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

Sites / Locations

New York Presbyterian Hospital-Weill Cornell Medical College

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

Arm Description

Subjects receive rimonabant 20 mg per day PO

Subjects take placebo capsule one a day PO

Outcomes

Primary Outcome Measures

Body Weight and Body fat mass

Secondary Outcome Measures

IGF-1, Leptin, Ghrelin, Serum Lipids and insulin sensitivity

Full Information

NCT ID

NCT00603109

First Posted

January 15, 2008

Last Updated

March 16, 2023

Sponsor

Weill Medical College of Cornell University

Collaborators

National Institutes of Health (NIH), PWSAUSA

1. Study Identification

Unique Protocol Identification Number

NCT00603109

Brief Title

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

Acronym

PWS

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

March 2016

Overall Recruitment Status

Terminated

Why Stopped

Drug side effects leading to premature stop

Study Start Date

August 2007 (undefined)

Primary Completion Date

January 2009 (Actual)

Study Completion Date

January 2009 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Weill Medical College of Cornell University

Collaborators

National Institutes of Health (NIH), PWSAUSA

4. Oversight

Data Monitoring Committee

Yes

5. 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Prader-willi Syndrome

Keywords

Body weight, fat mass, growth hormone, ghrelin

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 3

Interventional Study Model

Parallel Assignment

Masking

ParticipantCare ProviderInvestigatorOutcomes Assessor

Allocation

Randomized

Enrollment

10 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Arm Type

Experimental

Arm Description

Subjects receive rimonabant 20 mg per day PO

Arm Title

Arm Type

Placebo Comparator

Arm Description

Subjects take placebo capsule one a day PO

Intervention Type

Drug

Intervention Name(s)

rimonabant

Other Intervention Name(s)

Acomplia

Intervention Description

rimonabant capsule 20 mg per day PO for 6 months

Intervention Type

Drug

Intervention Name(s)

placebo

Intervention Description

Subjects take 20 mg placebo capsule one a day PO for 6 months

Primary Outcome Measure Information:

Title

Body Weight and Body fat mass

Time Frame

6 months

Secondary Outcome Measure Information:

Title

IGF-1, Leptin, Ghrelin, Serum Lipids and insulin sensitivity

Time Frame

6 months

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Maximum Age & Unit of Time

35 Years

Accepts Healthy Volunteers

Eligibility Criteria

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

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Roja Motaghedi, MD

Organizational Affiliation

NYPH-Weill Cornell Medical College

Official's Role

Principal Investigator

First Name & Middle Initial & Last Name & Degree

Moris Angulo, MD

Organizational Affiliation

Winthrop University Hospital

Official's Role

Principal Investigator

Facility Information:

Facility Name

New York Presbyterian Hospital-Weill Cornell Medical College

City

New York

State/Province

New York

ZIP/Postal Code

10065

Country

United States

12. IPD Sharing Statement

Plan to Share IPD

Citations:

PubMed Identifier

23543664

Citation

Deal CL, Tony M, Hoybye C, Allen DB, Tauber M, Christiansen JS; 2011 Growth Hormone in Prader-Willi Syndrome Clinical Care Guidelines Workshop Participants. GrowthHormone Research Society workshop summary: consensus guidelines for recombinant human growth hormone therapy in Prader-Willi syndrome. J Clin Endocrinol Metab. 2013 Jun;98(6):E1072-87. doi: 10.1210/jc.2012-3888. Epub 2013 Mar 29.

Results Reference

background

PubMed Identifier

20965292

Citation

Motaghedi R, Lipman EG, Hogg JE, Christos PJ, Vogiatzi MG, Angulo MA. Psychiatric adverse effects of rimonobant in adults with Prader Willi syndrome. Eur J Med Genet. 2011 Jan-Feb;54(1):14-8. doi: 10.1016/j.ejmg.2010.09.015. Epub 2010 Oct 20.

Results Reference

result

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

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