To evaluate the effects of long-term intranasal oxytocin on social behaviour in children with PWS and to compare the effects of different doses and frequencies of oxytocin administration
ID
Source
Brief title
Condition
- Chromosomal abnormalities, gene alterations and gene variants
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
The effect of different dosing regimens of intranasal oxytocin administration
on changes in social behaviour assessed by the oxytocin study questionnaire.
Secondary outcome
The effect of different dosing regimens of intranasal oxytocin administration
on changes in:
- Body composition (anthropometric measurements, BMI and DXA-scan)
- Quality of life (DUX25 and DUXPWS)
- Hyperphagia questionnaire (hyperphagia questionnaire Dykens)
- Theory of Mind (TOM test)
- Change in social and eating behaviour (diary)
- fMRI (BOLD responses during a task with food pictures
- Laboratory parameters (oxytocin in saliva and blood)
- Safety parameters (laboratory parameters and medical assessments).
Background summary
Patients with PWS have behavioural problems and are at risk for morbid obesity.
Several studies demonstrated hypothalamic and oxytocinergic dysfunction in
patients with PWS. The number of oxytocin-expressing neurons in the PVN of
patients with PWS is significantly decreased with 42%. Recent studies in humans
found positive effects of oxytocin on weight balance and social behaviour. The
oxytocin system is a promising target for therapeutic interventions, especially
in aberration in social function and obesity cotnrol. A pilot study with
intranasal oxytocin administration in adults with PWS and our previous study in
children with PWS aged 6 to 11 years showed positive effects on social
behaviour.
The aim of this study is to investigate the effects of different doses and
frequencies of oxytocin administration.
Study objective
To evaluate the effects of long-term intranasal oxytocin on social behaviour in
children with PWS and to compare the effects of different doses and frequencies
of oxytocin administration
Study design
Randomized cross-over open-label intervention study.
Intervention
Cross-over intervention with intranasal oxytocin in different dosing regimens
depending on study group and body surface
Study burden and risks
Burden: administration of intranasal oxytocin once a day, twice a day or every
other day during 3-6 months. Three hospital visits with a blood sample,
DXA-scan and psychological test and in children >6 years of age a fMRI. A short
diary about social and eating behaviour has to be filled out daily by parents.
Risks: we do not expect any side effects or adverse events during oxytocin
administration.
Patients and their parents are highly motivated to participate in this study
because of the major impact of hyperphagia and social problems on the daily
life of patients and their families
Westzeedijk 106
Rotterdam 3016AH
NL
Westzeedijk 106
Rotterdam 3016AH
NL
Listed location countries
Age
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
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)
Design
Recruitment
Medical products/devices used
Followed up by the following (possibly more current) registration
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Other (possibly less up-to-date) registrations in this register
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In other registers
| Register | ID |
|---|---|
| EudraCT | EUCTR2016-003820-22-NL |
| CCMO | NL59264.078.16 |