Prader-Willi Syndrome
Conditions
Keywords
Prader-Willi syndrome, Oxytocin
Brief 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.
Interventions
DRUGOxytocin nasal spray
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
DRUGPlacebo
Study treatment will be administered intranasally daily for 12 weeks.
Sponsors
University Hospital, Toulouse
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
OTHER
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
2 Years to 17 Years
Healthy volunteers
No
Inclusion criteria
1. Male or female patients between 2 years and 17 years and 5 months at inclusion. 2. Genetically confirmed diagnosis of PWS. 3. Parents (or legal representative) have signed the informed consent form and are willing to comply with all study procedures.
Exclusion criteria
1. A history of hypersensitivity to the study drug or drugs with similar chemical structures, to excipients of the product, or to latex; 2. Intolerance of intranasal administrations (including when due to a major behavioural problem); 3. Hyponatremia (clinically relevant at the discretion of the investigator); 4. Hypokalaemia (clinically relevant at the discretion of the investigator); 5. Prolongation of the QT interval and/or family history of prolongation of the QT interval; 6. Concomitant treatment prolonging the QT interval; 7. Start of growth hormone (GH) treatment within the last 4 weeks before inclusion; 8. History of abnormal electrocardiogram (ECG) (validated by a cardiologist); 9. Pregnant girls; (for girls with childbearing potential who do not have contraception and are sexually active, a negative pregnancy test will be required) 10. Patient with clinical signs in the context of contact with COVID-19 infected person. 11. Patient included in another study protocol on a medicinal product within the last 6 months; 12. Administrative problems: 1. Inability to give parents (or legal representatives) expert medical information; 2. No coverage by a social security regime.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| videofluoroscopic swallowing study (VFSS) score change | after 12 weeks oxytocin (OT) / placebo (at V2) | Percentage of patient with at least one VFSS subscore's change from baseline |
Countries
France
Outcome results
None listed