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

Obesity Clinical Trial

Official title: A Double Blind, Placebo Controlled, Fixed-Flexible Dose Clinical Trial of Guanfacine Extended Release for the Reduction of Aggression and Self-injurious Behavior Associated With Prader-Willi Syndrome

Status Recruiting

Clinical Trial Summary

This is a placebo-controlled clinical trial to assess whether Guanfacine Extended Release (GXR) reduces aggression and self injurious behavior in individuals with Prader Willi Syndrome (PWS). In addition, the study will establish the safety of GXR with a specific focus on metabolic effects.

Clinical Trial Description

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. Aggression, oppositional behavior, and temper tantrums frequently occur in patients 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 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.

This randomized, double-blind, placebo-controlled clinical trial aims to determine whether guanfacine extended release (GXR) reduces aggression and self-injury compared to placebo in individuals with PWS with moderate to severe aggressive and/or self-injurious behavior. In addition, GXR's tolerability will be assessed by systematically evaluating and documenting adverse events. ;

Related Conditions & MeSH terms

• Abnormalities, Multiple
• Aggression
• Behavioral Symptoms
• Chromosome Disorders
• Congenital Abnormalities
• Disease
• Genetic Diseases, Inborn
• Intellectual Disability
• Molecular Mechanisms of Pharmacological Action
• Nervous System Diseases
• Neurobehavioral Manifestations
• Neurologic Manifestations
• Neurotransmitter Agents
• Nutrition Disorders
• Obesity
• Overnutrition
• Pathologic Processes
• Physiological Effects of Drugs
• Prader-Willi Syndrome
• Self-Injurious Behavior
• Skin-Picking
• Syndrome

• NCT number: NCT05657860
• Study type: Interventional
• Source: Maimonides Medical Center
• Contact: Theresa Jacob, PhD, MPH
• Phone: 718-283-8170
• Email: tjacob@maimonidesmed.org
• Status: Recruiting
• Phase: Phase 4
• Start date: December 17, 2020
• Completion date: January 18, 2024