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


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.

Full Title of Study: “Long Term Evaluation of Infants Aged From 3 to 4 Years Old Included in the Ancient Study (Repeated Administrations of Oxytocin in Infants With Prader Willi Syndrome Aged From 0 to 6 Months) and Comparison With Not Treated and Age-matched Prader Willi Syndrome Infants (OT2SUITE)”

Study Type

  • Study Type: Interventional
  • Study Design
    • Allocation: Non-Randomized
    • Intervention Model: Parallel Assignment
    • Primary Purpose: Treatment
    • Masking: None (Open Label)
  • Study Primary Completion Date: May 2018

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.


  • Drug: Oxytocin
    • Infant included in the ancient study (repeated administrations of oxytocin in infants with Prader Willi Syndrome aged from 0 to 6 months)
  • Other: Control
    • Not treated.

Arms, Groups and Cohorts

  • Experimental: Oxytocin
    • Groups of children with Prader Willi Syndrome treated by oxytocin for 7 days during their first 6 months of life.
  • Experimental: Control
    • Groups of children with Prader Willi Syndrome not treated by oxytocin for 7 days during their first 6 months of life.

Clinical Trial Outcome Measures

Primary Measures

  • Evaluation of communication skills.
    • Time Frame: Day 1
    • Assessed by Vineland-II scale.

Secondary Measures

  • Evaluation of adaptative behavior composite and 3 domains : “Daily living skills”, “Socialization”, “Motor skills”.
    • Time Frame: Day 1
    • Assessed by Vineland-II scale.
  • Evaluation of behavioral troubles.
    • Time Frame: Day 1
    • Assessed by Child Behaviour Check List questionnaire.
  • Evaluation of global development.
    • Time Frame: Day 2 and 3
    • Assessed by Bayley Scales of Infant and Toddler Development.
  • Evaluation of orality and eating behaviour.
    • Time Frame: 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.
    • Time Frame: 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.
    • Time Frame: 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…).

Participating in This Clinical Trial

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.

Gender Eligibility: All

Minimum Age: 3 Years

Maximum Age: 4 Years

Are Healthy Volunteers Accepted: No

Investigator Details

  • Lead Sponsor
    • University Hospital, Toulouse
  • Provider of Information About this Clinical Study
    • Sponsor
  • Overall Official(s)
    • Maïthé Tauber, Pr, Principal Investigator, Centre de référence du syndrome de Prader-Willi- CHU Toulouse


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