Intranasal administration of oxytocin in children with Prader-Willi syndrome

• Register: EUCTR
• Last refreshed on: 8 January 2018
• Main ID: EUCTR2017-003423-30-NL
• Date of registration: 04/10/2017
• Prospective Registration: Yes
• Primary sponsor: Dutch Growth Research Foundation
• Public title: Intranasal administration of oxytocin in children with Prader-Willi syndrome
• Scientific title: Randomized, double-blind, placebo-controlled oxytocin and dose-response trial in children with Prader-Willi syndrome. Effects on social behaviour.
• Date of first enrolment: 12/12/2017
• Target sample size: 33
• Recruitment status: Authorised-recruitment may be ongoing or finished
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2017-003423-30
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes Randomised: yes Open: no Single blind: no Double blind: yes Parallel group: no Cross over: yes Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: yes Other: no Number of treatment arms in the trial: 2
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): yes Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV): no

Countries of recruitment

Netherlands

Contacts

• Name: Project leader Prader-Willi study
• Affiliation: Dutch Growth Research Foundation

• Name: Project leader Prader-Willi study
• Affiliation: Dutch Growth Research Foundation

Key inclusion & exclusion criteria

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)


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Prader-Willi syndrome
MedDRA version: 20.0 Level: PT Classification code 10036476 Term: Prader-Willi syndrome System Organ Class: 10010331 - Congenital, familial and genetic disorders

Therapeutic area: Diseases [C] - Hormonal diseases [C19]

Intervention(s)

Trade Name: Syntocinon nasal spray
Product Name: oxytocin
Product Code: oxytocin
Pharmaceutical Form: Nasal spray
Pharmaceutical form of the placebo: Nasal spray
Route of administration of the placebo: Intranasal use (Noncurrent)

Primary Outcome(s)

Main Objective: To evaluate the effects of oxytocin on social behaviour in children with Prader-Willi syndrome aged 3-11 years

Primary end point(s): Changes in social behaviour assessed by:
- Repetitive Behavior Scale-Revised
- Social Responsiveness-Scale

Secondary Objective: - To evaluate the effects on appetite, satiety, food intake and food seeking behaviour in children with PWS aged 3-11 years
- To evaluate oxytocin levels in blood and saliva samples before and during intransal oxytocin treatment
- To evaluate if there is a difference in effect between once or twice daily adminsitration
- To evaluate effects in relation to: BMI and body composition by DXA, IGF-1 and IGF-BP3-levels, genetic subtype

Timepoint(s) of evaluation of this end point: Day 1 and 3, 4, 7 and 13 months

Secondary Outcome(s)

Secondary end point(s): Change in:
- Clinical Global Impression scale
- Quality of life (DUX25 and DUXPWS)
- Social Behaviour (VISK, Oxytocin questionnaire revised)
- Hyperphagia (Hyperphagia questionnaire Dykens)
- Reading the Mind in the Eyes test, child version
- Body Composition (Anthropometric measurements, BMI and DXA-scan)
- Social and food related behaviour (diary)
- Food intake (diary)
- Laboratory parameters (oxytocin in saliva and blood)
- Safety parameters (laboratory parameters and medical assessments)

Timepoint(s) of evaluation of this end point: Day 1 and 3, 4, 7 and 13 months

Secondary ID(s)

2017-RCT-OX

Source(s) of Monetary Support

Dutch Growth Research Foundation