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

NCT03081832 | Completed

• 2 other identifiers: RC31/16/84072016-A01348-43
• Study Type: interventional
• Target: 34
• Locations: 1 country
• Sites: 1

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.

Conditions

Prader-Willi Syndrome

Keywords

Prader-Willi; Oxytocin; Communication skills; Oral skills; Brain activity; Hormonal disease

Outcome Measures

Primary Outcomes (1)

• Evaluation of communication skills.

  Assessed by Vineland-II scale.

  Day 1

Secondary Outcomes (6)

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

  Day 1

• Evaluation of behavioral troubles.

  Day 1

• Evaluation of global development.

  Day 2 and 3

• Evaluation of orality and eating behaviour.

  Day 2

• Evaluation of brain activity.

  Day 3

Study Arms (2)

Oxytocin

EXPERIMENTAL

Groups of children with Prader Willi Syndrome treated by oxytocin for 7 days during their first 6 months of life.

Drug: Oxytocin

Control

EXPERIMENTAL

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

Other: Control

Interventions

Oxytocin DRUG

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

Oxytocin

Control OTHER

Not treated.

Control

Eligibility Criteria

• Age: 3 Years - 4 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• University Hospital, Toulouse: lead

Study Sites (1)

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

Toulouse, 31059, France

Location

Related Publications (21)

• Amico JA, Vollmer RR, Cai HM, Miedlar JA, Rinaman L. Enhanced initial and sustained intake of sucrose solution in mice with an oxytocin gene deletion. Am J Physiol Regul Integr Comp Physiol. 2005 Dec;289(6):R1798-806. doi: 10.1152/ajpregu.00558.2005. Epub 2005 Sep 8.

  PMID: 16150836 BACKGROUND

• Arletti R, Benelli A, Bertolini A. Oxytocin inhibits food and fluid intake in rats. Physiol Behav. 1990 Dec;48(6):825-30. doi: 10.1016/0031-9384(90)90234-u.

  PMID: 2087513 BACKGROUND

• Billings LB, Spero JA, Vollmer RR, Amico JA. Oxytocin null mice ingest enhanced amounts of sweet solutions during light and dark cycles and during repeated shaker stress. Behav Brain Res. 2006 Jul 15;171(1):134-41. doi: 10.1016/j.bbr.2006.03.028. Epub 2006 May 4.

  PMID: 16677726 BACKGROUND

• Bittel DC, Kibiryeva N, Sell SM, Strong TV, Butler MG. Whole genome microarray analysis of gene expression in Prader-Willi syndrome. Am J Med Genet A. 2007 Mar 1;143A(5):430-42. doi: 10.1002/ajmg.a.31606.

  PMID: 17236194 BACKGROUND

• Dykens EM, Lee E, Roof E. Prader-Willi syndrome and autism spectrum disorders: an evolving story. J Neurodev Disord. 2011 Sep;3(3):225-37. doi: 10.1007/s11689-011-9092-5. Epub 2011 Aug 20.

  PMID: 21858456 BACKGROUND

• Dykens EM, Maxwell MA, Pantino E, Kossler R, Roof E. Assessment of hyperphagia in Prader-Willi syndrome. Obesity (Silver Spring). 2007 Jul;15(7):1816-26. doi: 10.1038/oby.2007.216.

  PMID: 17636101 BACKGROUND

• Dykens EM, Roof E, Hunt-Hawkins H. Cognitive and adaptive advantages of growth hormone treatment in children with Prader-Willi syndrome. J Child Psychol Psychiatry. 2017 Jan;58(1):64-74. doi: 10.1111/jcpp.12601. Epub 2016 Aug 2.

  PMID: 27481444 BACKGROUND

• Goldstone AP, Holland AJ, Butler JV, Whittington JE. Appetite hormones and the transition to hyperphagia in children with Prader-Willi syndrome. Int J Obes (Lond). 2012 Dec;36(12):1564-70. doi: 10.1038/ijo.2011.274. Epub 2012 Jan 24.

  PMID: 22270375 BACKGROUND

• Guastella AJ, Einfeld SL, Gray KM, Rinehart NJ, Tonge BJ, Lambert TJ, Hickie IB. Intranasal oxytocin improves emotion recognition for youth with autism spectrum disorders. Biol Psychiatry. 2010 Apr 1;67(7):692-4. doi: 10.1016/j.biopsych.2009.09.020. Epub 2009 Nov 7.

  PMID: 19897177 BACKGROUND

• Hall SS, Lightbody AA, McCarthy BE, Parker KJ, Reiss AL. Effects of intranasal oxytocin on social anxiety in males with fragile X syndrome. Psychoneuroendocrinology. 2012 Apr;37(4):509-18. doi: 10.1016/j.psyneuen.2011.07.020. Epub 2011 Aug 20.

  PMID: 21862226 BACKGROUND

• Kosfeld M, Heinrichs M, Zak PJ, Fischbacher U, Fehr E. Oxytocin increases trust in humans. Nature. 2005 Jun 2;435(7042):673-6. doi: 10.1038/nature03701.

  PMID: 15931222 BACKGROUND

• Lischke A, Gamer M, Berger C, Grossmann A, Hauenstein K, Heinrichs M, Herpertz SC, Domes G. Oxytocin increases amygdala reactivity to threatening scenes in females. Psychoneuroendocrinology. 2012 Sep;37(9):1431-8. doi: 10.1016/j.psyneuen.2012.01.011. Epub 2012 Feb 23.

  PMID: 22365820 BACKGROUND

• McDonald NA, Kuzmiski JB, Naderi N, Schwab Y, Pittman QJ. Endogenous modulators of synaptic transmission: cannabinoid regulation in the supraoptic nucleus. Prog Brain Res. 2008;170:129-36. doi: 10.1016/S0079-6123(08)00412-3.

  PMID: 18655878 BACKGROUND

• 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.

  PMID: 25599930 BACKGROUND

• Miller JL, Lynn CH, Driscoll DC, Goldstone AP, Gold JA, Kimonis V, Dykens E, Butler MG, Shuster JJ, Driscoll DJ. Nutritional phases in Prader-Willi syndrome. Am J Med Genet A. 2011 May;155A(5):1040-9. doi: 10.1002/ajmg.a.33951. Epub 2011 Apr 4.

  PMID: 21465655 BACKGROUND

• Salles J, Strelnikov K, Carine M, Denise T, Laurier V, Molinas C, Tauber M, Barone P. Deficits in voice and multisensory processing in patients with Prader-Willi syndrome. Neuropsychologia. 2016 May;85:137-47. doi: 10.1016/j.neuropsychologia.2016.03.015. Epub 2016 Mar 16.

  PMID: 26994593 BACKGROUND

• Schaller F, Watrin F, Sturny R, Massacrier A, Szepetowski P, Muscatelli F. A single postnatal injection of oxytocin rescues the lethal feeding behaviour in mouse newborns deficient for the imprinted Magel2 gene. Hum Mol Genet. 2010 Dec 15;19(24):4895-905. doi: 10.1093/hmg/ddq424. Epub 2010 Sep 28.

  PMID: 20876615 BACKGROUND

• Skokauskas N, Sweeny E, Meehan J, Gallagher L. Mental health problems in children with prader-willi syndrome. J Can Acad Child Adolesc Psychiatry. 2012 Aug;21(3):194-203.

  PMID: 22876265 BACKGROUND

• Swaab DF, Purba JS, Hofman MA. Alterations in the hypothalamic paraventricular nucleus and its oxytocin neurons (putative satiety cells) in Prader-Willi syndrome: a study of five cases. J Clin Endocrinol Metab. 1995 Feb;80(2):573-9. doi: 10.1210/jcem.80.2.7852523.

  PMID: 7852523 BACKGROUND

• van Lieshout CF, de Meyer RE, Curfs LM, Koot HM, Fryns JP. Problem behaviors and personality of children and adolescents with Prader-Willi syndrome. J Pediatr Psychol. 1998 Apr;23(2):111-20. doi: 10.1093/jpepsy/23.2.111.

  PMID: 9585637 BACKGROUND

• Valette M, Diene G, Glattard M, Cortadellas J, Molinas C, Faye S, Benvegnu G, Boulanouar K, Payoux P, Salles JP, Arnaud C, Cabal S, Tauber M. Early oxytocin treatment in infants with Prader-Willi syndrome is safe and is associated with better endocrine, metabolic and behavioral outcomes. Orphanet J Rare Dis. 2025 Mar 1;20(1):96. doi: 10.1186/s13023-025-03560-3.

  PMID: 40025514 DERIVED

MeSH Terms

Conditions

Prader-Willi Syndrome

Interventions

Oxytocin

Condition Hierarchy (Ancestors)

Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Nervous System Diseases; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Chromosome Disorders; Genetic Diseases, Inborn; Imprinting Disorders; Obesity; Overweight; Overnutrition; Nutrition Disorders; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Pituitary Hormones, Posterior; Pituitary Hormones; Peptide Hormones; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Peptides; Amino Acids, Peptides, and Proteins

Study Officials

• Maïthé Tauber, Pr

  Centre de référence du syndrome de Prader-Willi- CHU Toulouse

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 10, 2017
• First Posted: March 16, 2017
• Study Start: January 1, 2017
• Primary Completion: May 1, 2018
• Study Completion: December 1, 2018
• Last Updated: January 30, 2019

Record last verified: 2019-01

Data Sharing

• IPD Sharing: Will not share

Locations

FR (1)