NCT05657860

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

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: Completed
• Phase: Phase 4
• Study type: Interventional
• Enrollment: 15 (actual)
• Sponsor: Maimonides Medical Center · Academic / Other
• Sex: All
• Age: 6 Years – 35 Years
• Healthy volunteers: Not accepted

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.

Detailed 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.

Conditions

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

Interventions

Type	Name	Description
DRUG	Guanfacine Extended Release	Initial dose for all participants will be 1mg per day. If the medication is well tolerated, the dose can be raised to 2 mg until day 28 and increased to 3 mg for the remaining 4 weeks in the trial. The dose schedule will not be fixed., the treating clinician can delay a planned increase or lower the dose to manage adverse effects. At week 8 timepoint, the study will be unblinded.and subjects will continue treatment for 8 weeks.
OTHER	Placebo	Placebo will be administered concurrently with GXR during trials.

Timeline

• Start date: 2020-12-17
• Primary completion: 2023-12-16
• Completion: 2023-12-16
• First posted: 2022-12-20
• Last updated: 2024-08-19

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study