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

Prader-Willi Syndrome Clinical Trial

— DYSMOT  

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT05298085
• Other study ID #: RC31/20/0518
• Status: Completed
• Phase: Phase 2/Phase 3
• Start date: April 5, 2022
• Est. completion date: April 11, 2023

Study information

• Verified date: May 2023
• Source: University Hospital, Toulouse
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 25
• Est. completion date: April 11, 2023
• Est. primary completion date: April 11, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 2 Years to 17 Years

Eligibility

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.

Related Conditions & MeSH terms

• Prader-Willi Syndrome
• Syndrome

Intervention

Drug:
• Oxytocin 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
• Placebo
Study treatment will be administered intranasally daily for 12 weeks.

Locations

Country	Name	City	State
France	Hopital Jeanne de Flandre	Lille
France	Hôpital Femme-Mère-enfant Groupement hospitalier Est	Lyon
France	Chu Rouen	Rouen
France	CHU de Toulouse	Toulouse

Sponsors (1)

Lead Sponsor	Collaborator
University Hospital, Toulouse

Country where clinical trial is conducted

France, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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)