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

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.

Study Overview

• Status: Completed
• Conditions: Prader-Willi Syndrome
• Intervention / Treatment: Drug: Oxytocin nasal spray; Drug: Placebo

• Study Type: Interventional
• Enrollment (Actual): 25
• Phase: Phase 2; Phase 3

Contacts and Locations

Study Locations

• France
  • Lille, France
    • Hôpital Jeanne de Flandre
  • Lyon, France
    • Hôpital Femme-Mère-enfant Groupement hospitalier Est
  • Rouen, France
    • CHU Rouen
  • Toulouse, France
    • Chu de Toulouse

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 2 years to 17 years (Child)
• Accepts Healthy Volunteers: No

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Other
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

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

What is the study measuring?

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)

Collaborators and Investigators

• Sponsor: University Hospital, Toulouse
• Investigators: Principal Investigator: TAUBER Maithé, MD, University Hospital, Toulouse

Study record dates

Study Major Dates

• Study Start (Actual): April 5, 2022
• Primary Completion (Actual): April 11, 2023
• Study Completion (Actual): April 11, 2023

Study Registration Dates

• First Submitted: March 17, 2022
• First Submitted That Met QC Criteria: March 17, 2022
• First Posted (Actual): March 28, 2022

Study Record Updates

• Last Update Posted (Actual): June 1, 2023
• Last Update Submitted That Met QC Criteria: May 31, 2023
• Last Verified: May 1, 2023

More Information

Terms related to this study

• Keywords: Oxytocin; Prader-Willi syndrome
• Additional Relevant MeSH Terms: Pathologic Processes; Nervous System Diseases; Neurologic Manifestations; Neurobehavioral Manifestations; Disease; Congenital Abnormalities; Overnutrition; Nutrition Disorders; Overweight; Genetic Diseases, Inborn; Intellectual Disability; Abnormalities, Multiple; Chromosome Disorders; Obesity; Syndrome; Prader-Willi Syndrome; Physiological Effects of Drugs; Reproductive Control Agents; Oxytocics; Oxytocin
• Other Study ID Numbers: RC31/20/0518

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No