NCT05298085

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
25

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Apr 2022

Shorter than P25 for phase_2

Geographic Reach
1 country

4 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

March 17, 2022

Completed
11 days until next milestone

First Posted

Study publicly available on registry

March 28, 2022

Completed
8 days until next milestone

Study Start

First participant enrolled

April 5, 2022

Completed
1 year until next milestone

Primary Completion

Last participant's last visit for primary outcome

April 11, 2023

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

April 11, 2023

Completed
Last Updated

June 1, 2023

Status Verified

May 1, 2023

Enrollment Period

1 year

First QC Date

March 17, 2022

Last Update Submit

May 31, 2023

Conditions

Prader-Willi Syndrome

Keywords

Prader-Willi syndromeOxytocin

Outcome Measures

Primary Outcomes (1)

  • 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)

Study Arms (2)

OXYTOCIN nasal spray

EXPERIMENTAL

intranasal administration of Oxytocin

Drug: Oxytocin nasal spray

PLACEBO

PLACEBO COMPARATOR

intranasal administration of placebo

Drug: Placebo

Interventions

Oxytocin nasal sprayDRUG

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

Also known as: oxytocin
OXYTOCIN nasal spray
PlaceboDRUG

Study treatment will be administered intranasally daily for 12 weeks.

Also known as: placebo nasal spray
PLACEBO

Eligibility Criteria

Age2 Years - 17 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • Genetically confirmed diagnosis of PWS.
  • Parents (or legal representative) have signed the informed consent form and are willing to comply with all study procedures.

You may not qualify if:

  • 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;
  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (4)

Hopital Jeanne de Flandre

Lille, France

Location

Hôpital Femme-Mère-enfant Groupement hospitalier Est

Lyon, France

Location

Chu Rouen

Rouen, France

Location

CHU de Toulouse

Toulouse, France

Location

MeSH Terms

Conditions

Prader-Willi Syndrome

Interventions

Oxytocin

Condition Hierarchy (Ancestors)

Intellectual DisabilityNeurobehavioral ManifestationsNeurologic ManifestationsNervous System DiseasesAbnormalities, MultipleCongenital AbnormalitiesCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesChromosome DisordersGenetic Diseases, InbornImprinting DisordersObesityOverweightOvernutritionNutrition DisordersNutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Pituitary Hormones, PosteriorPituitary HormonesPeptide HormonesHormonesHormones, Hormone Substitutes, and Hormone AntagonistsPeptidesAmino Acids, Peptides, and Proteins

Study Officials

  • TAUBER Maithé, MD

    University Hospital, Toulouse

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
OTHER
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

March 17, 2022

First Posted

March 28, 2022

Study Start

April 5, 2022

Primary Completion

April 11, 2023

Study Completion

April 11, 2023

Last Updated

June 1, 2023

Record last verified: 2023-05

Data Sharing

IPD Sharing
Will not share

Locations

FR(4)