Prader-Willi Syndrome Clinical Trial
Official title:
Dose Clinical Trial of Guanfacine Extended Release for the Reduction of Aggression and Self-injuries Behavior Associated With Prader-Willi Syndrome
This is a placebo-controlled clinical trial to assess the utility of Guanfacine Extended Release (GXR) in the management of patients with Prader Willi Syndrome (PWS) who have significant aggression or self-injury. The purpose of this trial is to establish the safety of GXR with a specific focus on metabolic effects.
Prader-Willi syndrome is a genetic disorder due to loss of function of specific genes. In
newborns, symptoms include weak muscles, poor feeding, and slow development. Beginning in
childhood, the person becomes constantly hungry, which often leads to obesity and type 2
diabetes. Also, mild to moderate learning disability and behavioral problems are typical.
Guanfacine Extended Release (GXR), the investigational drug in this study would be the first
study to evaluate the drug in patients with Prader Willi Syndrome. "Investigational" means it
is not approved by the Food and Drug Administration (FDA) to treat Prader Willi Syndrome.
However, Guanfacine Extended Released (GXR) is an FDA approved drug used to treat children
and adolescents with hypertension and attention deficit hyperactivity disorder (ADHD). GXR is
thought to respond to parts of the brain that lead to strengthening working memory, reducing
distraction, improving attention and impulse control. GXR is generally considered safe for
children as long as it is used according to the dosing instructions (up to 4mg) of a
qualified medical professional.
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