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

Launched by MAIMONIDES MEDICAL CENTER · Dec 12, 2022

Keywords

ClinConnect Summary

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 th...

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • Diagnosis of PWS confirmed by genetic testing documentation
• • Rating of moderate or above on the Clinical Global Impression- Severity Scale
• Exclusion Criteria:
• • Subjects with positive pregnancy test, swallowing difficulty, and/or presenting with active psychosis or mania will be excluded
• • Subjects currently taking guanfacine extended release
• • Patients with lactose intolerance
• • 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.

Trial Officials