Dose Clinical Trial of Guanfacine Extended Release for the Reduction of Aggression and Self-injuries Behavior Associated With Prader-Willi Syndrome
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|ClinicalTrials.gov Identifier: NCT04066088|
Recruitment Status : Not yet recruiting
First Posted : August 26, 2019
Last Update Posted : August 27, 2019
|Condition or disease||Intervention/treatment||Phase|
|Prader-Willi Syndrome||Other: Placebo Drug: Guanfacine extended release (GXR)||Phase 4|
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.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||33 participants|
|Intervention Model:||Crossover Assignment|
|Masking:||Double (Participant, Investigator)|
|Official Title:||Dose Clinical Trial of Guanfacine Extended Release for the Reduction of Aggression and Self-injuries Behavior Associated With Prader-Willi Syndrome|
|Estimated Study Start Date :||December 2019|
|Estimated Primary Completion Date :||March 30, 2020|
|Estimated Study Completion Date :||March 30, 2020|
|Sham Comparator: Placebo||
Placebo will be administered same times as GXR
Immediately following the 8-week blinded randomized trial, an 8-week open-label continuation phase will be pursued to further define efficacy and tolerability of GXR, and to establish its safety with specific focus on metabolic profile.
Drug: Guanfacine extended release (GXR)
The starting dose for all subjects will be 1 mg per day. If the medication is well-tolerated, the dose can be raised to 2 mg until day 28 and increased to 3mg 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, the study will be unblinded and subjects will continue treatment for 8 more weeks.
- CGI-I scale rating [ Time Frame: 19 Weeks ]A positive clinical response will be determined by a rating of 1 or 2 (Very much/Much improved) on the CGI-I scale at the end of the blinded trial.
- Aberrant Behavior Checklist [ Time Frame: 19 Weeks ]Consists of 2 subscales; irritability (15 items) and hyperactivity/noncompliance (16 items).
- Self-Injury Trauma scale [ Time Frame: 19 Weeks ]SHI scores of 5 or greater were found to be indicative of borderline personality disorder. Part 1 is ranking based on the number of wounds 1=one would (common in a mild SIB but rare in a severe case) 2=two or four wounds (common) and 3=five or more wounds (rare). Injury severity is scored on a subjective basis with labels such as "mild" "moderate" and "severe" accompanied by descriptions of the observed state of the anatomy. Part 3 is the Estimate of Current Risk.
- Modified Overt Aggression Scale [ Time Frame: 19 Weeks ]four-part behavior rating scale used to evaluate and document the "frequency and severity" of aggressive episodes. The rating scale is made up of four categories; verbal aggression, aggression against objects, aggression against self, and aggression against others
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04066088
|Contact: Nirmal Tejwani||212 598 6599||Nirmal.Tejwani@nyulangone.org|
|United States, New York|
|NYU Langone Health||Not yet recruiting|
|New York, New York, United States, 10016|
|Contact: Nirmal Tejwani 212-598-6599 Nirmal.Tejwani@nyulangone.org|
|Principal Investigator: Deepan Singh, MD|
|Principal Investigator:||Deepan Singh, MD||New York Langone Health|