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

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.

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.

Interventions

  • 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

References

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Meziane H, Schaller F, Bauer S, Villard C, Matarazzo V, Riet F, Guillon G, Lafitte D, Desarmenien MG, Tauber M, Muscatelli F. An Early Postnatal Oxytocin Treatment Prevents Social and Learning Deficits in Adult Mice Deficient for Magel2, a Gene Involved in Prader-Willi Syndrome and Autism. Biol Psychiatry. 2015 Jul 15;78(2):85-94. doi: 10.1016/j.biopsych.2014.11.010. Epub 2014 Nov 20.

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