Prader Willi SyndromePhase 2NCT01038570
Comparative Study Between Prader-Willi Patients Who Take Oxytocin Versus Placebo
A Phase 2 study testing Syntocinon®/- Spray and Physiological serum (Sodium chloride) in people with Prader Willi Syndrome, run by University Hospital, Toulouse at 1 site in France.
Trial summary
The role of oxytocin (OT) is already known in the regulation of satiety but some clinical studies demonstrated that OT participates also in the regulation of social behavior by its implication on a better comprehension of emotion which plays a role on theory of mind and empathy. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Prader Willi Syndrome
- What is being tested?
- Syntocinon®/- Spray and Physiological serum (Sodium chloride)
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 24 participants
- Where
- 1 siteFrance
- Study dates
- Jun 2009to Sep 2009
- Phase
- Phase 2
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- PWS genetically confirmed,
- Age 18-year-old superior,
- Negative pregnancy test.
Who cannot join
- Severe psychiatric troubles
- Problem administration staff,
- Saving of justice,
- Abnormalities of the heart rhythm,
- Hepatic or renal insufficiency,
- Pregnancy,
- Oxytocin hypersensibility,
- Treatment causing rhythm disorders.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Evaluation of the Effect of the Oxytocin Administered in Nasal Pulverizing on the Social Skills, the Stress, the Anxiety and the Eating Habits at Grown-up Patients Presenting a Syndrome of Prader-Willi: Pilot Study
The role of oxytocin (OT) is already known in the regulation of satiety but some clinical studies demonstrated that OT participates also in the regulation of social behavior by its implication on a better comprehension of emotion which plays a role on theory of mind and empathy. By the way, these 2 behaviors are deviants for patients with Prader-Willi Syndrome (PWS). Actually, no study was led on the effect of OT on PWS patients but Swaab and al in 1995 showed a significant reduction in number and in volume of neurons expressing OT in the paraventricular nucleus of PWS patients. Recent data were obtained studying OT in patients with autism which showed a link between the deregulation of OT and the autistic pathology. Clinical and imaging studies obtained with PWS and autistic populations make us believe that some mechanisms are common between these two pathologies.
The objectives of this project are:
- to look for an influence on the understanding of the social codes of the PWS patients,
- to look for an influence on the behavior of stress and anxiety and on the regulation of eating habits when patients receive a nasal pulverizing of OT.
Read the detailed description
Recent clinical studies showed that the oxytocin (OT), next to its long-time known actions (lactation, satiety), participates in the regulation of the social behavior. Swaab and al in 1995 showed a significant reduction in number and in volume of neurons expressing OT in the paraventricular nucleus of PWS patients. Recent data suggest a link between OT and the autistic pathology: the existence of a lower plasmatic rate on OT and the association with a polymorphism of the OT's receptor for autistics patients. Because OT has a role on the pro-social behavior and on the nervous control of the stress, the hypothesis on which a deficit in OT would play a role in the abnormalities of the social behavior was advanced. Moreover, certain features of the autistic spectrum disorder can be found in PWS patients. Moreover, a recent study has shown that OT administered by intra-nasal spray reduces psychosocial stress and increases the confidence in each other in healthy volunteers. The PWS is a genetic pathology and some clinical features are very similar to the autistic pathology. The PWS is the most common cause of syndromic autism. Data from the literature and a collaborative study conducted between our team and the Prof. B. Rogé's team suggests that these two pathologies share common pathophysiological mechanisms. To our knowledge, no study with the OT was conducted in people with PWS. We want to conduct a pilot study in adult patients with PWS. Given the severity of this disease, related to socialization disorders, significant anxiety and overeating, and with no effective therapy, this justifies the pilot study. Moreover, side effects of OT are almost nonexistent when used by intra-nasal.
Objectives: The objectives of this project are to investigate whether the administration by intra-nasal spray of OT plays a role on the understanding of social codes of PWS adult patients. We also analyze its effect on the level of stress, anxiety and eating behavior.
Methodology: This study is a double blind control study. We will include 24 PWS patients, aged over 18 years recruited at the Hendaye Marine Hospital (associated with the PWS reference center whose coordination is in Toulouse). The hospital routinely receives PWS adults for a 4 weeks stay which gives optimal conditions for the establishment of this study. Patients are separated into 2 groups matched one by one on the sex and IQ. One group receives the OT, the other a placebo. Forty five minutes after inhalation, all of the patients are evaluated on their social comprehension thanks to psychometric tests. Furthermore, an analysis based on observation sheets allows us to score daily stress, anxiety and eating behavior. These sheets serve for the comparison between 2 days before and 2 days after the nasal spray on the above described scoring components.
Results: Differences between the two groups will be statistically analyzed by non parametric statistical tests such as Mann-Whitney's test.
- Conditions
- Prader Willi Syndrome
- Being tested
- Syntocinon®/- Spray, Physiological serum (Sodium chloride)
- Keywords
- Prader Willi syndrome, adults, social behaviour, stress, anxiety, psychological tests
- Medical subject headings
- Prader-Willi Syndrome, Social Behavior, Anxiety Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Syntocinon®/- SprayDrug
3 sprays corresponding to 24 IU in each nostril
Physiological serum (Sodium chloride)Drug
Sodium chloride 0.9 % (0.3 ml) in each nostril
| Study group | Role | What they receive |
|---|---|---|
| Oxytocin | Experimental | Not described Receives: Drug: Syntocinon®/- Spray |
| Physiological serum | Placebo comparator | Not described Receives: Drug: Physiological serum (Sodium chloride) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Result scores at Psychological tests : "Reading the mind in the eyes" (RMET), Tests for evaluation of the "theory of mind" : Sally and Ann, Cartoons,"L'esprit des autres", "Social Attribution Task" (SAT).
Measured: fourth day
Secondary outcomes (1)
Result scores at daily stress, anxiety and eating behavior tests
Measured: Five days (first day to fifth day)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Dec 2009 (estimated)
- Study startJun 2009
- Primary completionSep 2009
- Study completionSep 2009
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Prader Willi SyndromeStudies of the same condition, open and under way.
- 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 24 participants (actual)
Sponsor and investigators
- Lead sponsor
- University Hospital, Toulouse
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Maïthé TAUBER, MD, Principal investigator, Centre de référence du syndrome de Prader-Willi - Equipe d'Endocrinologie
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01038570
- Sponsor's protocol ID
- 09 071 03
- Status last verified
- May 2017
- First submitted
- 16 Nov 2009
- Last updated
- 12 May 2017
- Results posted
- No
- Data monitoring committee
- Yes
- Individual data sharing
- No
Publications
Tauber M, Mantoulan C, Copet P, Jauregui J, Demeer G, Diene G, Roge B, Laurier V, Ehlinger V, Arnaud C, Molinas C, Thuilleaux D. Oxytocin may be useful to increase trust in others and decrease disruptive behaviours in patients with Prader-Willi syndrome: a randomised placebo-controlled trial in 24 patients. Orphanet J Rare Dis. 2011 Jun 24;6:47. doi: 10.1186/1750-1172-6-47.
PubMed 21702900
Study at a glance
From the registry record, last updated by the sponsor on 12 May 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Evaluation of the Effect of the Oxytocin Administered in Nasal Pulverizing on the Social Skills, the Stress, the Anxiety and the Eating Habits at Grown-up Patients Presenting a Syndrome of Prader-Willi: Pilot Study
- ClinicalTrials.gov ID
- NCT01038570
- Acronym / sponsor ID
- 09 071 03
- Conditions
- Prader Willi Syndrome
- Keywords
- Prader Willi syndrome, adults, social behaviour, stress, anxiety, psychological tests
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 24 participants (actual)
- Lead sponsor
- University Hospital, ToulouseUniversity, hospital or other
- Study start
- Jun 2009
- Primary completion
- Sep 2009 (actual)
- Study completion
- Sep 2009 (actual)
- First posted
- 24 Dec 2009 (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 role of oxytocin (OT) is already known in the regulation of satiety but some clinical studies demonstrated that OT participates also in the regulation of social behavior by its implication on a better comprehension of emotion which plays a role on theory of mind and empathy. By the way, these 2 behaviors are deviants for patients with Prader-Willi Syndrome (PWS). Actually, no study was led on the effect of OT on PWS patients but Swaab and al in 1995 showed a significant reduction in number and in volume of neurons expressing OT in the paraventricular nucleus of PWS patients. Recent data were obtained studying OT in patients with autism which showed a link between the deregulation of OT and the autistic pathology. Clinical and imaging studies obtained with PWS and autistic populations make us believe that some mechanisms are common between these two pathologies.
The objectives of this project are:
- to look for an influence on the understanding of the social codes of the PWS patients,
- to look for an influence on the behavior of stress and anxiety and on the regulation of eating habits when patients receive a nasal pulverizing of OT.
Detailed description
Recent clinical studies showed that the oxytocin (OT), next to its long-time known actions (lactation, satiety), participates in the regulation of the social behavior. Swaab and al in 1995 showed a significant reduction in number and in volume of neurons expressing OT in the paraventricular nucleus of PWS patients. Recent data suggest a link between OT and the autistic pathology: the existence of a lower plasmatic rate on OT and the association with a polymorphism of the OT's receptor for autistics patients. Because OT has a role on the pro-social behavior and on the nervous control of the stress, the hypothesis on which a deficit in OT would play a role in the abnormalities of the social behavior was advanced. Moreover, certain features of the autistic spectrum disorder can be found in PWS patients. Moreover, a recent study has shown that OT administered by intra-nasal spray reduces psychosocial stress and increases the confidence in each other in healthy volunteers. The PWS is a genetic pathology and some clinical features are very similar to the autistic pathology. The PWS is the most common cause of syndromic autism. Data from the literature and a collaborative study conducted between our team and the Prof. B. Rogé's team suggests that these two pathologies share common pathophysiological mechanisms. To our knowledge, no study with the OT was conducted in people with PWS. We want to conduct a pilot study in adult patients with PWS. Given the severity of this disease, related to socialization disorders, significant anxiety and overeating, and with no effective therapy, this justifies the pilot study. Moreover, side effects of OT are almost nonexistent when used by intra-nasal.
Objectives: The objectives of this project are to investigate whether the administration by intra-nasal spray of OT plays a role on the understanding of social codes of PWS adult patients. We also analyze its effect on the level of stress, anxiety and eating behavior.
Methodology: This study is a double blind control study. We will include 24 PWS patients, aged over 18 years recruited at the Hendaye Marine Hospital (associated with the PWS reference center whose coordination is in Toulouse). The hospital routinely receives PWS adults for a 4 weeks stay which gives optimal conditions for the establishment of this study. Patients are separated into 2 groups matched one by one on the sex and IQ. One group receives the OT, the other a placebo. Forty five minutes after inhalation, all of the patients are evaluated on their social comprehension thanks to psychometric tests. Furthermore, an analysis based on observation sheets allows us to score daily stress, anxiety and eating behavior. These sheets serve for the comparison between 2 days before and 2 days after the nasal spray on the above described scoring components.
Results: Differences between the two groups will be statistically analyzed by non parametric statistical tests such as Mann-Whitney's test.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Oxytocin | Experimental | Drug: Syntocinon®/- Spray |
| Physiological serum | Placebo comparator | Drug: Physiological serum (Sodium chloride) |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- PWS genetically confirmed,
- Age 18-year-old superior,
- Negative pregnancy test.
Key exclusion criteria (8 listed)
- Severe psychiatric troubles
- Problem administration staff,
- Saving of justice,
- Abnormalities of the heart rhythm,
- Hepatic or renal insufficiency,
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 |
|---|---|---|
| Syntocinon®/- Spray3 sprays corresponding to 24 IU in each nostril | Drug | Oxytocin |
| Physiological serum (Sodium chloride)Sodium chloride 0.9 % (0.3 ml) in each nostril | Drug | Physiological serum |
Primary outcome
Result scores at Psychological tests : "Reading the mind in the eyes" (RMET), Tests for evaluation of the "theory of mind" : Sally and Ann, Cartoons,"L'esprit des autres", "Social Attribution Task" (SAT).
Time frame: fourth day
1 secondary outcome
Result scores at daily stress, anxiety and eating behavior tests
Time frame: Five days (first day to fifth day)
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 May 2017.
- Results publication: PubMed 21702900Tauber M, Mantoulan C, Copet P, Jauregui J, Demeer G, Diene G, Roge B, Laurier V, Ehlinger V, Arnaud C, Molinas C, Thuilleaux D. Oxytocin may be useful to increase trust in others and decrease disruptive behaviours in patients with Prader-Willi syndrome: a randomised placebo-controlled trial in 24 patients. Orphanet J Rare Dis. 2011 Jun 24;6:47. doi: 10.1186/1750-1172-6-47.
- 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.
Maïthé TAUBER, MD principal investigator
Centre de référence du syndrome de Prader-Willi - Equipe d'Endocrinologie
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Syntocinon®/- Spray and Physiological serum (Sodium chloride) in people with Prader Willi Syndrome, run by University Hospital, Toulouse at 1 site in France.
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 8 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: finished, as of the sponsor's last update on 12 May 2017. 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 France. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Result scores at Psychological tests : "Reading the mind in the eyes" (RMET), Tests for evaluation of the "theory of mind" : Sally and Ann, Cartoons,"L'esprit des autres", "Social Attribution Task" (SAT). (time frame: fourth day). 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 NCT01038570, which also lists 1 linked publication.
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 12 May 2017.