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

University Hospital, Toulouse34 enrolled

Overview

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.

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 Type

INTERVENTIONAL

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Prader-Willi Syndrome

Interventions

OxytocinDRUG

Infant included in the ancient study (repeated administrations of oxytocin in infants with Prader Willi Syndrome aged from 0 to 6 months)

ControlOTHER

Not treated.

Eligibility

Sex: ALLMin age: 3 YearsMax age: 4 Years

Medical Language ↔ Plain English

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.

Locations (1)

Centre de référence du syndrome de Prader-Willi Hôpital des Enfants

Toulouse, France

Outcomes

Primary Outcomes

Evaluation of communication skills.

Assessed by Vineland-II scale.

Time frame: Day 1

Secondary Outcomes

Evaluation of adaptative behavior composite and 3 domains : "Daily living skills", "Socialization", "Motor skills".

Assessed by Vineland-II scale.

Time frame: Day 1

Evaluation of behavioral troubles.

Assessed by Child Behaviour Check List questionnaire.

Time frame: Day 1

Evaluation of global development.

Assessed by Bayley Scales of Infant and Toddler Development.

Time frame: Day 2 and 3

Evaluation of orality and eating behaviour.

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.

Time frame: Day 2

Evaluation of brain activity.

Assessed by a morphological Magnetic resonance imaging, a resting functional Magnetic resonance imaging.

Time frame: Day 3

Evaluation of plasma levels of ghrelin and other peptides involved in feeding behaviour or energy metabolism.

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...).

Time frame: Day 1

Data from ClinicalTrials.gov

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