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

NCT00603109 | Terminated Phase 3 DMC

• 1 other identifier: 0611008841
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

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.

Conditions

Prader-willi Syndrome

Keywords

Body weight, fat mass, growth hormone, ghrelin

Outcome Measures

Primary Outcomes (1)

• Body Weight and Body fat mass

  6 months

Secondary Outcomes (1)

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

  6 months

Study Arms (2)

I

EXPERIMENTAL

Subjects receive rimonabant 20 mg per day PO

Drug: rimonabant

II

PLACEBO COMPARATOR

Subjects take placebo capsule one a day PO

Drug: placebo

Interventions

rimonabant DRUG

rimonabant capsule 20 mg per day PO for 6 months

Also known as: Acomplia

I

placebo DRUG

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

II

Eligibility Criteria

• Age: 18 Years - 35 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• Presence of pulmonary disease.
• Presence of any other abnormal endocrine findings, including abnormal thyroid function.
• 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

Sponsors & Collaborators

• Weill Medical College of Cornell University: lead
• National Institutes of Health (NIH): collaborator
• PWSAUSA: collaborator

Study Sites (1)

New York Presbyterian Hospital-Weill Cornell Medical College

New York, New York, 10065, United States

Location

Related Publications (2)

• 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.

  PMID: 23543664 BACKGROUND

• 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.

  PMID: 20965292 RESULT

MeSH Terms

Conditions

Prader-Willi Syndrome; Body Weight

Interventions

Rimonabant

Condition Hierarchy (Ancestors)

Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Nervous System Diseases; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Chromosome Disorders; Genetic Diseases, Inborn; Imprinting Disorders; Obesity; Overweight; Overnutrition; Nutrition Disorders; Nutritional and Metabolic Diseases; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Pyrazoles; Azoles; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Piperidines

Study Officials

• Roja Motaghedi, MD

  NYPH-Weill Cornell Medical College

  PRINCIPAL INVESTIGATOR

• Moris Angulo, MD

  Winthrop University Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 15, 2008
• First Posted: January 28, 2008
• Study Start: August 1, 2007
• Primary Completion: January 1, 2009
• Study Completion: January 1, 2009
• Last Updated: March 20, 2023

Record last verified: 2016-03

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)