Prader-Willi SyndromePhase 2/3DYSMOTNCT05298085
Effect of Intranasal Oxytocin on Dysphagia in Children and Adolescents With Prader-Willi Syndrome
A Phase 2/3 study testing Oxytocin nasal spray in people with Prader-Willi Syndrome, run by University Hospital, Toulouse at 4 sites in France.
Trial summary
This phase 2B is designed to test the effectiveness of intranasal Oxytocin on Prader Willi Syndrome (PWS). This is a prospective, multicentre, randomised, double-blind, Phase 2B clinical study planned to include around 24 PWS patients aged 2-17 years and 5 months. Read more about this study
- Who is it for?
- Ages 2 to 17 years
- Conditions the study is for
- Prader-Willi Syndrome
- What is being tested?
- Oxytocin nasal spray
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 25 participants
- Where
- 4 sitesFrance
- Study dates
- 5 Apr 2022to 11 Apr 2023
- Phase
- Phase 2/3
- 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 2 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Male or female patients between 2 years and 17 years and 5 months at inclusion.
- Genetically confirmed diagnosis of PWS.
- Parents (or legal representative) have signed the informed consent form and are willing to comply with all study procedures.
Who cannot join
- A history of hypersensitivity to the study drug or drugs with similar chemical structures, to excipients of the product, or to latex;
- Intolerance of intranasal administrations (including when due to a major behavioural problem);
- Hyponatremia (clinically relevant at the discretion of the investigator);
- Hypokalaemia (clinically relevant at the discretion of the investigator);
- Prolongation of the QT interval and/or family history of prolongation of the QT interval;
- Concomitant treatment prolonging the QT interval;
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- Start of growth hormone (GH) treatment within the last 4 weeks before inclusion;
- History of abnormal electrocardiogram (ECG) (validated by a cardiologist);
- Pregnant girls; (for girls with childbearing potential who do not have contraception and are sexually active, a negative pregnancy test will be required)
- Patient with clinical signs in the context of contact with COVID-19 infected person.
- Patient included in another study protocol on a medicinal product within the last 6 months;
- Administrative problems:
- Inability to give parents (or legal representatives) expert medical information;
- No coverage by a social security regime.
Trial locations and contacts
4 sites in 1 country.
France4 sites
Hopital Jeanne de Flandre
Lille
Hôpital Femme-Mère-enfant Groupement hospitalier Est
Lyon
Chu Rouen
Rouen
CHU de Toulouse
Toulouse
About this study
Official title: Effect of Intranasal Oxytocin on Dysphagia Related to Oropharyngo-oesophageal Dysmotility in Children and Adolescents With Prader-Willi Syndrome: a Phase 2B Study
This phase 2B is designed to test the effectiveness of intranasal Oxytocin on Prader Willi Syndrome (PWS).
This is a prospective, multicentre, randomised, double-blind, Phase 2B clinical study planned to include around 24 PWS patients aged 2-17 years and 5 months.
- Conditions
- Prader-Willi Syndrome
- Being tested
- Oxytocin nasal spray, Placebo
- Keywords
- Prader-Willi syndrome, Oxytocin
- Medical subject headings
- Prader-Willi Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Oxytocin nasal sprayDrug
Also called oxytocin
Study treatment will be administered intranasally daily for 12 weeks. The daily dose of OT will be adapted according to age at the beginning of the study
PlaceboDrug
Also called placebo nasal spray
Study treatment will be administered intranasally daily for 12 weeks.
| Study group | Role | What they receive |
|---|---|---|
| OXYTOCIN nasal spray | Experimental | intranasal administration of Oxytocin Receives: Drug: Oxytocin nasal spray |
| PLACEBO | Placebo comparator | intranasal administration of placebo Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
videofluoroscopic swallowing study (VFSS) score change
Percentage of patient with at least one VFSS subscore's change from baseline
Measured: after 12 weeks oxytocin (OT) / placebo (at V2)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Mar 2022
- Study start5 Apr 2022
- Primary completion11 Apr 2023
- Study completion11 Apr 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Prader-Willi SyndromeStudies of the same condition, open and under way.
- More trials run by University Hospital, ToulouseThe 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 2/3
- Primary purpose
- Other
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 25 participants (actual)
Sponsor and investigators
- Lead sponsor
- University Hospital, Toulouse
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- TAUBER Maithé, MD, Principal investigator, University Hospital, Toulouse
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05298085
- Sponsor's protocol ID
- RC31/20/0518
- Acronym
- DYSMOT
- Status last verified
- May 2023
- First submitted
- 17 Mar 2022
- Last updated
- 1 Jun 2023
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 1 Jun 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Effect of Intranasal Oxytocin on Dysphagia Related to Oropharyngo-oesophageal Dysmotility in Children and Adolescents With Prader-Willi Syndrome: a Phase 2B Study
- ClinicalTrials.gov ID
- NCT05298085
- Acronym / sponsor ID
- DYSMOT / RC31/20/0518
- Conditions
- Prader-Willi Syndrome
- Keywords
- Prader-Willi syndrome, Oxytocin
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 25 participants (actual)
- Lead sponsor
- University Hospital, ToulouseUniversity, hospital or other
- Study start
- 5 Apr 2022 (actual)
- Primary completion
- 11 Apr 2023 (actual)
- Study completion
- 11 Apr 2023 (actual)
- First posted
- 28 Mar 2022 (actual)
- 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.
This phase 2B is designed to test the effectiveness of intranasal Oxytocin on Prader Willi Syndrome (PWS).
This is a prospective, multicentre, randomised, double-blind, Phase 2B clinical study planned to include around 24 PWS patients aged 2-17 years and 5 months.
Study design
- Primary purpose
- Other
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| OXYTOCIN nasal spray | Experimental | Drug: Oxytocin nasal sprayintranasal administration of Oxytocin |
| PLACEBO | Placebo comparator | Drug: Placebointranasal administration of placebo |
Who may qualify
- Ages
- 2 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (3 listed)
- Male or female patients between 2 years and 17 years and 5 months at inclusion.
- Genetically confirmed diagnosis of PWS.
- Parents (or legal representative) have signed the informed consent form and are willing to comply with all study procedures.
Key exclusion criteria (14 listed)
- A history of hypersensitivity to the study drug or drugs with similar chemical structures, to excipients of the product, or to latex;
- Intolerance of intranasal administrations (including when due to a major behavioural problem);
- Hyponatremia (clinically relevant at the discretion of the investigator);
- Hypokalaemia (clinically relevant at the discretion of the investigator);
- Prolongation of the QT interval and/or family history of prolongation of the QT interval;
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 |
|---|---|---|
| Oxytocin nasal sprayAlso: oxytocinStudy treatment will be administered intranasally daily for 12 weeks. The daily dose of OT will be adapted according to age at the beginning of the study | Drug | OXYTOCIN nasal spray |
| PlaceboAlso: placebo nasal sprayStudy treatment will be administered intranasally daily for 12 weeks. | Drug | PLACEBO |
Primary outcome
videofluoroscopic swallowing study (VFSS) score change
Time frame: after 12 weeks oxytocin (OT) / placebo (at V2)
Percentage of patient with at least one VFSS subscore's change from baseline
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 1 Jun 2023.
- 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.
TAUBER Maithé, MD principal investigator
University Hospital, Toulouse
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Oxytocin nasal spray in people with Prader-Willi Syndrome, run by University Hospital, Toulouse at 4 sites 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 14 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 1 Jun 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites 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 combined 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.
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 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?
videofluoroscopic swallowing study (VFSS) score change (time frame: after 12 weeks oxytocin (OT) / placebo (at V2)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 1 Jun 2023.