Guanfacine Extended Release for the Reduction of Aggression and Self-injurious Behavior Associated With Prader-Willi Syndrome

Prader‐Willi syndrome is a genetic disorder due to loss of function of specific genes. Innewborns, symptoms include weak muscles, poor feeding, and slow development. Beginning inchildhood, the person becomes constantly hungry, which often leads to obesity and type 2diabetes. Aggression, oppositional behavior, and temper tantrums frequently occur inpatients with PWS. PWS also has a high prevalence of self‐injury, repetitive behavior,impulsivity, over‐activity, and mild to moderate learning disability.Guanfacine Extended Release (GXR), the investigational drug in this study would be thefirst study to evaluate the drug in patients with Prader Willi Syndrome."Investigational" means it is not approved by the Food and Drug Administration (FDA) totreat Prader Willi Syndrome. However, Guanfacine Extended Released (GXR) is an FDAapproved drug used to treat children and adolescents with hypertension and attentiondeficit hyperactivity disorder (ADHD). GXR is thought to respond to parts of the brainthat lead to strengthening working memory, reducing distraction, improving attention andimpulse control. GXR is generally considered safe for children as long as it is usedaccording to the dosing instructions (up to 4mg) of a qualified medical professional.This randomized, double‐blind, placebo‐controlled clinical trial aims to determinewhether guanfacine extended release (GXR) reduces aggression and self‐injury compared toplacebo in individuals with PWS with moderate to severe aggressive and/or self‐injuriousbehavior. In addition, GXR's tolerability will be assessed by systematically evaluatingand documenting adverse events.