Prader-willi SyndromePhase 3PWSNCT00603109
Effect of Rimonabant on Weight Gain and Body Composition in Adults With Prader Willi Syndrome
A Phase 3 study testing rimonabant in people with Prader-willi Syndrome, run by Weill Medical College of Cornell University at 1 site in United States.
Trial 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). Read more about this study
- Who is it for?
- Ages 18 to 35 years
- Conditions the study is for
- Prader-willi Syndrome
- What is being tested?
- rimonabant
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 10 participants
- Where
- 1 siteUnited States
- Study dates
- Aug 2007to Jan 2009
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 35 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- 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.
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
United States1 site
New York Presbyterian Hospital-Weill Cornell Medical College
New York, New York, 10065
About this study
Official title: Effect of Rimonabant, a Cannabinoid Receptor 1 Antagonist on Weight Gain and Body Composition in Adults With Prader Willi Syndrome.
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
- Being tested
- rimonabant, placebo
- Keywords
- Body weight, fat mass, growth hormone, ghrelin
- Medical subject headings
- Prader-Willi Syndrome, Body Weight
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
rimonabantDrug
Also called Acomplia
rimonabant capsule 20 mg per day PO for 6 months
placeboDrug
Subjects take 20 mg placebo capsule one a day PO for 6 months
| Study group | Role | What they receive |
|---|---|---|
| I | Experimental | Subjects receive rimonabant 20 mg per day PO Receives: Drug: rimonabant |
| II | Placebo comparator | Subjects take placebo capsule one a day PO Receives: Drug: placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Body Weight and Body fat mass
Measured: 6 months
Secondary outcomes (1)
IGF-1, Leptin, Ghrelin, Serum Lipids and insulin sensitivity
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Jan 2008 (estimated)
- Study startAug 2007
- Primary completionJan 2009
- Study completionJan 2009
Why it stopped: Drug side effects leading to premature stop
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Prader-willi SyndromeStudies of the same condition, open and under way.
- More trials run by Weill Medical College of Cornell UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- Weill Medical College of Cornell University
- Sponsor type
- University, hospital or other
- Collaborators
- National Institutes of Health (NIH), PWSAUSA
- Responsible party
- Sponsor
- Investigators
- Roja Motaghedi, MD, Principal investigator, NYPH-Weill Cornell Medical CollegeMoris Angulo, MD, Principal investigator, Winthrop University Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00603109
- Sponsor's protocol ID
- 0611008841
- Acronym
- PWS
- Status last verified
- Mar 2016
- First submitted
- 15 Jan 2008
- Last updated
- 20 Mar 2023
- Results posted
- No
- Data monitoring committee
- Yes
- Individual data sharing
- No
Publications
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.
PubMed 23543664Motaghedi 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.
PubMed 20965292
Study at a glance
From the registry record, last updated by the sponsor on 20 Mar 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Effect of Rimonabant, a Cannabinoid Receptor 1 Antagonist on Weight Gain and Body Composition in Adults With Prader Willi Syndrome.
- ClinicalTrials.gov ID
- NCT00603109
- Acronym / sponsor ID
- PWS / 0611008841
- Conditions
- Prader-willi Syndrome
- Keywords
- Body weight, fat mass, growth hormone, ghrelin
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Drug side effects leading to premature stop
- Enrolment
- 10 participants (actual)
- Lead sponsor
- Weill Medical College of Cornell UniversityUniversity, hospital or other
- Collaborators
- National Institutes of Health (NIH), PWSAUSA
- Study start
- Aug 2007
- Primary completion
- Jan 2009 (actual)
- Study completion
- Jan 2009 (actual)
- First posted
- 28 Jan 2008 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| I | Experimental | Drug: rimonabantSubjects receive rimonabant 20 mg per day PO |
| II | Placebo comparator | Drug: placeboSubjects take placebo capsule one a day PO |
Who may qualify
- Ages
- 18 to 35 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (3 listed)
- 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.
Key exclusion criteria (6 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| rimonabantAlso: Acompliarimonabant capsule 20 mg per day PO for 6 months | Drug | I |
| placeboSubjects take 20 mg placebo capsule one a day PO for 6 months | Drug | II |
Primary outcome
Body Weight and Body fat mass
Time frame: 6 months
1 secondary outcome
IGF-1, Leptin, Ghrelin, Serum Lipids and insulin sensitivity
Time frame: 6 months
Results and safety
The study was stopped early (reason given: Drug side effects leading to premature stop). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Mar 2023.
- Background: PubMed 23543664Deal 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 publication: PubMed 20965292Motaghedi 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.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Roja Motaghedi, MD principal investigator
NYPH-Weill Cornell Medical College
Moris Angulo, MD principal investigator
Winthrop University Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing rimonabant in people with Prader-willi Syndrome, run by Weill Medical College of Cornell University at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 20 Mar 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: Drug side effects leading to premature stop). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Body Weight and Body fat mass (time frame: 6 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00603109, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Mar 2023; AzTrials last refreshed its copy on 7 Nov 2026.