Intranasal administration of oxytocin in children with Prader-Willi syndrome

Phase 1

• Conditions: Prader-Willi syndrome; MedDRA version: 20.0Level: PTClassification code 10036476Term: Prader-Willi syndromeSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Hormonal diseases [C19]

• Registration Number: EUCTR2017-003423-30-NL
• Lead Sponsor: Dutch Growth Research Foundation

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2017-10-04
• Last Update Posted: 8 January 2018

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 33

Inclusion Criteria

In order to be eligible to participate in this study, a subject must meet all of the following criteria:
- Genetically confirmed diagnosis of Prader-Willi syndrome
- Children aged 3 to 10.99 years
- Informed consent
- Currently on growth hormone treatment for at least 1 year
- Behavioural characteristics such as reduced social reciprocity and interaction, repetitive behaviour or temper tantrums, and/or be in nutritional phase 2b or 3 according to Miller (increased interest in food, hyperphagia)
Are the trial subjects under 18? yes
Number of subjects for this age range: 33
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

A potential subject who meets any of the following criteria will be excluded from participation in this study:
- Severe psychiatric problems
- Non-cooperative behaviour
- Allergic reactions or hypersensitivity for oxytocin
- Serious illness
- Cardiac abnormalities
- Extremely low dietary intake or less than required itnake acoording to WHO
- Medication to reduce weight (fat)

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To evaluate the effects of oxytocin on social behaviour in children with Prader-Willi syndrome aged 3-11 years;Secondary Objective: - To evaluate the effects on appetite, satiety, food intake and food seeking behaviour in children with PWS aged 3-11 years<br>- To evaluate oxytocin levels in blood and saliva samples before and during intransal oxytocin treatment<br>- To evaluate if there is a difference in effect between once or twice daily adminsitration<br>- To evaluate effects in relation to: BMI and body composition by DXA, IGF-1 and IGF-BP3-levels, genetic subtype;Primary end point(s): Changes in social behaviour assessed by:<br>- Repetitive Behavior Scale-Revised<br>- Social Responsiveness-Scale;Timepoint(s) of evaluation of this end point: Day 1 and 3, 4, 7 and 13 months

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): Change in: <br>- Clinical Global Impression scale<br>- Quality of life (DUX25 and DUXPWS)<br>- Social Behaviour (VISK, Oxytocin questionnaire revised)<br>- Hyperphagia (Hyperphagia questionnaire Dykens)<br>- Reading the Mind in the Eyes test, child version<br>- Body Composition (Anthropometric measurements, BMI and DXA-scan)<br>- Social and food related behaviour (diary)<br>- Food intake (diary)<br>- Laboratory parameters (oxytocin in saliva and blood)<br>- Safety parameters (laboratory parameters and medical assessments);Timepoint(s) of evaluation of this end point: Day 1 and 3, 4, 7 and 13 months