Follow-up of Prader Willi Syndrome Infants Treated by Oxytocin and Comparison With Not-treated Infants.

Not Applicable

Completed

• Conditions: Prader-Willi Syndrome
• Interventions: Drug: Oxytocin; Other: Control

• Registration Number: NCT03081832
• Lead Sponsor: University Hospital, Toulouse

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.

Study Timeline

• Study Start: 2017-01
• Study First Submitted: 2017-03-10
• Study First Submitted QC: 2017-03-10
• Study First Posted: 2017-03-16
• Primary Completion: 2018-05
• Study Completion: 2018-12
• Status Verified: 2019-01
• Last Update Submitted: 2019-01-29
• Last Update Posted: 2019-01-30

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 34

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Oxytocin	Oxytocin	Groups of children with Prader Willi Syndrome treated by oxytocin for 7 days during their first 6 months of life.
Control	Control	Groups of children with Prader Willi Syndrome not treated by oxytocin for 7 days during their first 6 months of life.

Primary Outcome Measures

Name	Time	Method
Evaluation of communication skills.	Day 1	Assessed by Vineland-II scale.

Secondary Outcome Measures

Name	Time	Method
Evaluation of global development.	Day 2 and 3	Assessed by Bayley Scales of Infant and Toddler Development.
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.
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 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.

Trial Locations

Locations (1)

Centre de référence du syndrome de Prader-Willi Hôpital des Enfants; 🇫🇷 Toulouse, France