Study Purpose
Guanfacine Extended Release (GXR), is an FDA approved drug used to treat children and adolescents with hypertension and attention deficit hyperactivity disorder (ADHD).This will be the first study to evaluate the drug in patients with Prader Willi Syndrome.
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 of a qualified medical professional.
Maimonides Medical Center is seeking volunteers between the ages of 6 and 35 years old diagnosed with Prader-Willi Syndrome (PWS) to participate in a clinical research study to see whether or not the study drug works to reduce irritability, aggression, and skin picking/rectal picking behaviors seen in PWS.
This study has been amended and only the first visit is required to be in-person. The remaining visits can all be conducted virtually.
This study has been funded by the Foundation for Prader-Willi Research.
Dr. Deepan Singh discusses Guanfacine and his clinical trial in the video below.
Recruitment Criteria
Study Type: Randomized, double-blind, placebo-controlled, crossover
Eligible Ages: 6 to 35 years old
Other Criteria: diagnosis of Prader-Willi Syndrome
Trial Details
Phase: 4
Duration: 19 weeks
Status: recruiting
Lead Sponsor: FPWR
Countries: United States
Additional Details: Travel compensation will be provided for in-person visits. Only 3 in-person visits are required for completing the study.
Eligibility Criteria
Inclusion Criteria:
- 6 and 35 years of age
- diagnosis of PWS confirmed by genetic testing.
- rating of moderate or above on the Clinical Global Impression- Severity Scale will be required for entry.
Exclusion Criteria:
- Subjects with a positive pregnancy test, swallowing difficulty, and/or presenting with active psychosis or mania will be excluded.
- Individuals with pre-existing, clinically significant bradycardia (< 8 years: <64 bpm; 8 to 12 years: <59 bpm; 12 to 16 years: <53 bpm) or hypotension, defined as 5th percentile for height and gender,26 will be excluded from the study.
- Subjects receiving antipsychotic medications due to a documented history of psychosis or bipolar disorder will be allowed to continue taking the medication without dosage modification.
- Growth hormone, thyroid hormone replacement treatment, and non-psychiatric medicines will be allowed to continue.
- N-Acetyl Cysteine and anticonvulsant medication (only if prescribed for seizures) will be allowed to continue, with specific instructions to not make any dosage changes during the clinical trial.
This Study is enrolling at the following sites:
Maimonides Medical Center, Brooklyn, NY
Principal Investigator: Deepan Singh, MD
Contact: Psychiatry Research Division: (718) 283-8170 or psychresearch@maimonidesmed.org
Eligible participants will receive study related medical examinations, research medication and laboratory tests at no cost, while they are participating in this clinical research study.
