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

Phase 2

Completed

• Conditions: Prader-Willi Syndrome
• Interventions: Drug: Oxytocin nasal spray; Drug: Placebo

• Registration Number: NCT05298085
• Lead Sponsor: University Hospital, Toulouse

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2022-03-17
• Study First Submitted QC: 2022-03-17
• Study First Posted: 2022-03-28
• Study Start: 2022-04-05
• Primary Completion: 2023-04-11
• Study Completion: 2023-04-11
• Status Verified: 2023-05
• Last Update Submitted: 2023-05-31
• Last Update Posted: 2023-06-01

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 25

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
OXYTOCIN nasal spray	Oxytocin nasal spray	intranasal administration of Oxytocin
PLACEBO	Placebo	intranasal administration of placebo

Primary Outcome Measures

Name	Time	Method
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

Trial Locations

Locations (4)

Hopital Jeanne de Flandre; 🇫🇷 Lille, France

Chu Rouen; 🇫🇷 Rouen, France

Hôpital Femme-Mère-enfant Groupement hospitalier Est; 🇫🇷 Lyon, France

CHU de Toulouse; 🇫🇷 Toulouse, France