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

Prader-Willi Syndrome Clinical Trial

— OT2SUITE  

Official title:

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)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03081832
• Other study ID #: RC31/16/8407
• Secondary ID: 2016-A01348-43
• Status: Completed
• Phase: N/A
• Start date: January 2017
• Est. completion date: December 2018

Study information

• Verified date: January 2019
• Source: University Hospital, Toulouse
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 34
• Est. completion date: December 2018
• Est. primary completion date: May 2018
• Accepts healthy volunteers: No
• Gender: All
• Age group: 3 Years to 4 Years

Eligibility

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.

Related Conditions & MeSH terms

• Prader-Willi Syndrome
• Syndrome

Intervention

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.

Locations

Country	Name	City	State
France	Centre de référence du syndrome de Prader-Willi Hôpital des Enfants	Toulouse

Sponsors (1)

Lead Sponsor	Collaborator
University Hospital, Toulouse

Country where clinical trial is conducted

France, 

References & Publications (20)

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. Epub 2005 Sep 8. — View Citation

Arletti R, Benelli A, Bertolini A. Oxytocin inhibits food and fluid intake in rats. Physiol Behav. 1990 Dec;48(6):825-30. — View Citation

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. Epub 2006 May 4. — View Citation

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. — View Citation

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. — View Citation

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. — View Citation

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. — View Citation

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. — View Citation

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. — View Citation

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. — View Citation

Kosfeld M, Heinrichs M, Zak PJ, Fischbacher U, Fehr E. Oxytocin increases trust in humans. Nature. 2005 Jun 2;435(7042):673-6. — View Citation

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. — View Citation

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. Review. — View Citation

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. — View Citation

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. — View Citation

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. — View Citation

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. — View Citation

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. — View Citation

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. — View Citation

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. — View Citation

* Note: There are 20 references in all — Click here to view all references

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Evaluation of communication skills.	Assessed by Vineland-II scale.	Day 1
Secondary	Evaluation of adaptative behavior composite and 3 domains : "Daily living skills", "Socialization", "Motor skills".	Assessed by Vineland-II scale.	Day 1
Secondary	Evaluation of behavioral troubles.	Assessed by Child Behaviour Check List questionnaire.	Day 1
Secondary	Evaluation of global development.	Assessed by Bayley Scales of Infant and Toddler Development.	Day 2 and 3
Secondary	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.	Day 2
Secondary	Evaluation of brain activity.	Assessed by a morphological Magnetic resonance imaging, a resting functional Magnetic resonance imaging.	Day 3
Secondary	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...).	Day 1