Effect of Intranasal Oxytocin on Dysphagia in Children and Adolescents With Prader-Willi Syndrome

Study Overview
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Brief Summary
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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.

Official Title
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Effect of Intranasal Oxytocin on Dysphagia Related to Oropharyngo-oesophageal Dysmotility in Children and Adolescents With Prader-Willi Syndrome: a Phase 2B Study

Conditions
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Prader-Willi Syndrome

Intervention / Treatment
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* Drug: Oxytocin nasal spray
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: 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. Exclusion Criteria: 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; 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.

Ages Eligible for Study
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Minimum Age: 2 Years
Maximum Age: 17 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Other
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: OXYTOCIN nasal spray<br>intranasal administration of Oxytocin | Drug: Oxytocin nasal spray<br>* 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<br>* Other names: oxytocin;|
| Placebo Comparator: PLACEBO<br>intranasal administration of placebo | Drug: Placebo<br>* Study treatment will be administered intranasally daily for 12 weeks.<br>* Other names: placebo nasal spray;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| videofluoroscopic swallowing study (VFSS) score change | Percentage of patient with at least one VFSS subscore's change from baseline | after 12 weeks oxytocin (OT) / placebo (at V2) | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Prader-Willi syndrome, Oxytocin