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

Sponsor
University Hospital, Toulouse (Other)
Overall Status
Recruiting
CT.gov ID
NCT05298085
Collaborator
(none)
36
Enrollment
1
Location
2
Arms
32.9
Anticipated Duration (Months)
1.1
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

This phase 3 is designed to test the effectiveness of intranasal Oxytocin on Prader Willi Syndrome (PWS).

This is a prospective, multicentre, randomised, double-blind, Phase 3 clinical study planned to include around 36 PWS patients aged 2-17 years and 5 months.

Condition or Disease Intervention/Treatment Phase
  • Drug: Oxytocin nasal spray
  • Drug: Placebo
 Phase 3

Study Design

Study Type:
Interventional
Anticipated Enrollment :
36 participants
Allocation:
Randomized
Intervention Model:
Parallel Assignment
Masking:
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose:
Other
Official Title:
Effect of Intranasal Oxytocin on Dysphagia Related to Oropharyngo-oesophageal Dysmotility in Children and Adolescents With Prader-Willi Syndrome: a Phase 3 Study
Actual Study Start Date :
Apr 5, 2022
Anticipated Primary Completion Date :
Apr 1, 2023
Anticipated Study Completion Date :
Dec 31, 2024

Arms and Interventions

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

    		•

    Outcome Measures

    Primary Outcome Measures

    1. 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

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    2 Years to 17 Years
    Sexes Eligible for Study:
    All
    Accepts 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:

    13. Inability to give parents (or legal representatives) expert medical information;

    14. No coverage by a social security regime.

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 CHU de Toulouse Toulouse France

    Sponsors and Collaborators

    • University Hospital, Toulouse

    Investigators

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

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    University Hospital, Toulouse
    ClinicalTrials.gov Identifier:
    NCT05298085
    Other Study ID Numbers:
    • RC31/20/0518
    First Posted:
    Mar 28, 2022
    Last Update Posted:
    May 19, 2022
    Last Verified:
    May 1, 2022
    Individual Participant Data (IPD) Sharing Statement:
    No
    Plan to Share IPD:
    No
    Studies a U.S. FDA-regulated Drug Product:
    Yes
    Studies a U.S. FDA-regulated Device Product:
    No
    Keywords provided by University Hospital, Toulouse
    Prader-Willi syndrome
    Oxytocin
    Additional relevant MeSH terms:
    Prader-Willi Syndrome
    Syndrome
    Oxytocin

    Study Results

    No Results Posted as of May 19, 2022