Ignite Creation Date:
2025-12-25 @ 2:03 AM
Ignite Modification Date:
2025-12-26 @ 2:19 AM
Study NCT ID:
NCT05657860
Status:
COMPLETED
Last Update Posted:
2024-08-19
First Post:
2022-12-12
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Guanfacine Extended Release for the Reduction of Aggression and Self-injurious Behavior Associated With Prader-Willi Syndrome
Sponsor:
Maimonides Medical Center
Organization:
Study Overview
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:
COMPLETED
Status Verified Date:
2024-08
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
PWS-GXR
Brief 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.
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.
Study Oversight
Has Oversight DMC:
True
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
True
Is an FDA AA801 Violation?: