Phase 2 Study: Intranasal Oxytocin for Treatment of Infants with Prader-Willi Syndrome in Nutritional Phase 1a
Principal Investigator: Jennifer Miller, MD
Pediatric Endocrinology, University of Florida
Individuals with Prader-Willi syndrome (PWS) experience several nutritional phases through their lifespan. Initially, there is severe neonatal hypotonia with decreased appetite and impaired suck necessitating assisted feeding (nutritional phase 1a) (Miller et al, 2011). Nutritional phase 1a is characterized by the need for assisted feeding. The feeding can typically be accomplished using a special nipple/bottle, placement of a nasogastric tube, or placement of a gastrostomy tube.However, this is a dangerous time for infants with PWS, and has a high rate of morbidity and mortality due to aspiration and respiratory failure (Nagai et al, 2005). Feeding and swallowing skill development parallels psychosocial milestones of homeostasis, attachment, and separation/individuation. Mothers of infants with PWS often describe a feeling of lack of attachment or lack of bonding with their child due to the lack of suckling and responsiveness of the children.
In an open-label study, oxytocin nasal spray has been shown to be well-tolerated in infants with PWS and to improve feeding and social skills (Tauber et al, 2017). In this study we want to test whether daily oxytocin nasal spray will perform superiorly to placebo in improving suck, swallow, feeding, and maternal bonding in individuals with PWS who are in nutritional phase 1a. The results of this study will help determine if a larger, future trial should be done to consider intranasal oxytocin as standard of care for individuals with PWS in nutritional phase 1a. Clinical trials of intranasal oxytocin for food-related behaviors in older children will be forthcoming in the future.