Intranasal Oxytocin Trial Investigates Appetite and Social Behaviors in Prader-Willi Syndrome

Individuals with Prader-Willi syndrome (PWS) often experience severe challenges including an insatiable appetite leading to obesity, and significant social behavior deficits. These issues are thought to stem from a fundamental neurobiological problem: a deficit of oxytocin-producing neurons and reduced oxytocin receptor (OTR) gene function. Current management primarily focuses on symptomatic treatment and behavioral interventions, which often fall short in addressing the core neurodevelopmental and metabolic dysregulation. Investigating oxytocin administration directly targets this hypothesized underlying deficiency, offering a potential novel therapeutic avenue for these debilitating symptoms.

This Phase 2 clinical trial aims to assess the safety and efficacy of intranasal oxytocin (IN-OT) in infants and children diagnosed with Prader-Willi syndrome (PWS) who are in nutritional phase 1a. The study's primary objective is to compare the change from baseline to the morning of day 6 in suck and swallow competency following IN-OT administration. Additionally, the trial will investigate whether intranasal oxytocin causes any side effects and if it alters appetite or social behaviors in this vulnerable pediatric population. Specific dosing, frequency, and control arm details were not provided in the abstract, but a Phase 2 trial typically involves a placebo comparator.

This record describes the design and objectives of an ongoing clinical trial, not its completed results. Therefore, no findings regarding the efficacy or safety of intranasal oxytocin in Prader-Willi syndrome are available from this abstract. The study is designed to answer key questions regarding potential side effects and the impact of oxytocin administration on appetite and social behaviors in children with PWS, with a primary endpoint focused on suck and swallow competency.