Evaluation of Tolerance, Suckling and Food Intake After Repeated Nasals Administrations of Oxytocin in PWS Infants
Status:
Completed
Trial end date:
2014-07-01
Target enrollment:
Participant gender:
Summary
The Prader-Willi syndrome (PWS) includes severe neonatal hypotonia with impaired suckling
leading to failure to thrive in the most severe cases, subsequently followed by an early
onset of morbid obesity with hyperphagia and deficit of satiety, combined with other
endocrine dysfunction probably due to hypothalamic dysfunction. The pathophysiological
mechanism of the occurrence of the 2 main nutritional phases of PWS is unknown. Swaab
reported a deficit in the oxytocin (OT)-producing neurons of the paraventricular nucleus in
the brain of these patients. In addition of its well-known anorexigenic effect, OT is
involved in establishing and maintaining social codes. Moreover in a PWS mouse model
generated from a MAGEL2 KO gene a single OT injection at 5 hr of life prevent the early death
observed in 50 % of the new born mice by recovering normal suckling. Interestingly this
effect is no longer observed if OT injection takes place later. Our hypothesis is that early
administration of OT in babies with PWS may improve suckling and possibly infant-mother
interactions. In our recent study (manuscript in preparation), we have shown that a single
intranasal administration of OT is well tolerated. This escalating dose study is designed to
evaluate the tolerance of repeated intranasal administration of OT in 3 steps (4IU every
other day, 4 IU daily, 4IU twice daily) in babies younger than 5 months with PWS.