Prader-Willi Syndrome
Conditions
Keywords
Prader-Willi, Oxytocin, Communication skills, Oral skills, Brain activity, Hormonal disease
Brief summary
The objective of this study is to collect data on tolerance and effects of early treatment with oxytocin in children with Prader Willi Syndrome aged from 3 to 4 years and to compare these infants with not treated age-matched infants with Prader Willi Syndrome.
Detailed description
In accordance with recommendations of regulatory authorities, we want to collect long term data of patients treated with oxytocin before the age of 6 months. Moreover clinical observations of these infants support long term effects on communication skills, global development and behaviour.
Interventions
DRUGOxytocin
Infant included in the ancient study (repeated administrations of oxytocin in infants with Prader Willi Syndrome aged from 0 to 6 months)
OTHERControl
Not treated.
Sponsors
University Hospital, Toulouse
Study design
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
3 Years to 4 Years
Healthy volunteers
No
Inclusion criteria
* Infants with Prader Willi Syndrome (genetic diagnosis confirmed) * For treated group : infant included in the ancient study * For not treated group: infant never treated with oxytocin
Exclusion criteria
* Subject involved in another search including an exclusion period still in progress at the time of inclusion. * Impossibility to give parents or legal guardian informed information * No coverage by a Social Security scheme * Refusal of parents or legal representative to sign consent. If a patient has a contraindication to Magnetic resonance imaging, it may be included in the study but Magnetic resonance imaging will not be performed.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Evaluation of communication skills. | Day 1 | Assessed by Vineland-II scale. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Evaluation of global development. | Day 2 and 3 | Assessed by Bayley Scales of Infant and Toddler Development. |
| Evaluation of adaptative behavior composite and 3 domains : Daily living skills, Socialization, Motor skills. | Day 1 | Assessed by Vineland-II scale. |
| Evaluation of behavioral troubles. | Day 1 | Assessed by Child Behaviour Check List questionnaire. |
| Evaluation of brain activity. | Day 3 | Assessed by a morphological Magnetic resonance imaging, a resting functional Magnetic resonance imaging. |
| Evaluation of plasma levels of ghrelin and other peptides involved in feeding behaviour or energy metabolism. | Day 1 | Circulating levels of acylated and non-acylated ghrelin and some peptides and neuropeptides involved in appetite regulation (leptin, cortisol, insulin, Glucagon like peptide-1, pancreatic polypeptide, orexin A, alpha-melanocyte stimulating hormone...). |
| Evaluation of orality and eating behaviour. | Day 2 | Assessed by: * A questionnaire on eating behavior. * An oral evaluation, which combines a clinical examination carried out by the reference center physician, and the assessment of eating behavior during the meal. * The fluoroscopy of swallowing. |
Countries
France
Outcome results
None listed