Intranasal administration of oxytocin in children with Prader-Willi Syndrome

• Register: EUCTR
• Last refreshed on: 23 January 2017
• Main ID: EUCTR2016-003820-22-NL
• Date of registration: 12/10/2016
• Prospective Registration: Yes
• Primary sponsor: Dutch Growth Research Foundation
• Public title: Intranasal administration of oxytocin in children with Prader-Willi Syndrome
• Scientific title: Intranasal administration of oxytocin in children with Prader-Willi Syndrome. A randomized, open-label, cross-over trial of different treatment regimens of oxytocin administration. Effects on eating behaviour and social behaviour. - Intranasal administration of oxytocin in children with PWS
• Date of first enrolment: 22/12/2016
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2016-003820-22
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no Randomised: yes Open: yes Single blind: no Double blind: no Parallel group: no Cross over: yes Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: no
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no 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
- Age between 3 and 16 years
- Currently on growth hormone treatment for at least 1 year
- Behavioural problems (for example temper tantrums and autistic behaviour) and/or be in nutritional phase 2b or 3 according to Miller.

For fMRI: age > 6 years
Are the trial subjects under 18? yes
Number of subjects for this age range: 44
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:
- Severe psychiatric problems
- Non-cooperatieve behaviour
- Allergic reactions or hypersensitivity for oxytocin
- Serious illness
- Cardiac abnormalities
- Extremely low dietary intake or less than minimal required intake 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: 19.0 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

Primary Outcome(s)

Secondary Objective: To evaluate oxytocin levels in blood and saliva samples before, during and after intranasal oxytocin treatment in different doses and frequencies in children with PWS
- To evaluate the effects of long-term intranasal oxytocin administration in different doses and frequencies in relation to: BMI, IGF-1 and IGF-BP3 levels, fMRI (BOLD response)

Main Objective: - To evaluate the effects of long-term intranasal oxytocin on social behaviour in children with PWS
- To evaluate the effects of long-term intranasal oxytocin administration on appetite, satiety, food intake and food seeking behaviour in children with PWS
- To investigate if there is a difference in the efficacy of oxytocin if adminstered in different doses and frequencies

Timepoint(s) of evaluation of this end point: Studygroup 1:
Day 1: baseline assessments in the hospital, first administration of oxytocin followed by observation period and instruction of administration
Day 2-month 6: administration of oxytocin one/twice a day every day at home
- 6 months: hospital visit and assessments as mentioned on day 1
- Mont 6-month 7: wash-out period
- 7 months: parents fill out a questionnaire at home. Start other dosing regimen.
Month 7-month 13: administration of oxytocin once/twice daily every day at home
13 months: hospital visit and assessment as mentioned on day 1

For studygroup 2 assessments will be done on day 1 (hospital), at 3 months (hospital), at 4 months (questionnaire at home), 7 months (hospital)

Primary end point(s): Changes in social and food related behaviour assessed by:
- Oxytocin Study Questionnaire
- VISK

Secondary Outcome(s)

Secondary end point(s): Change in:
- Body composition
- Quality of life
- Hyperphagia questionnaire
- Theory of Mind test
- Diary concerning social and food related behaviour
- fMRI (>6 years old)
- Laboratory parameters
- Safety parameters

Timepoint(s) of evaluation of this end point: Studygroup 1:
Day 1: baseline assessments in the hospital, first administration of oxytocin followed by observation period and instruction of administration
Day 2-month 6: administration of oxytocin one/twice a day every day at home
- 6 months: hospital visit and assessments as mentioned on day 1
- Mont 6-month 7: wash-out period
- 7 months: parents fill out a questionnaire at home. Start other dosing regimen.
Month 7-month 13: administration of oxytocin once/twice daily every day at home
13 months: hospital visit and assessment as mentioned on day 1

For studygroup 2 assessments will be done on day 1 (hospital), at 3 months (hospital), at 4 months (questionnaire at home), 7 months (hospital)

Secondary ID(s)

2016-Longterm-OX

Source(s) of Monetary Support

Dutch Growth Research Foundation