Clinical trial NCT04066088: Dose Clinical Trial of Guanfacine Extended Release for the Reduction of Aggression and Self-injuries Behavior Associated With Prader-Willi Syndrome
Official title: Dose Clinical Trial of Guanfacine Extended Release for the Reduction of Aggression and Self-injuries Behavior Associated With Prader-Willi Syndrome
Study type: INTERVENTIONAL
Overall status: WITHDRAWN
Phase: PHASE4
Conditions: Prader-Willi Syndrome
Lead sponsor: NYU Langone Health
Sponsor class: OTHER
Collaborators: name: Winthrop University Hospital
First posted: 2019-08-26
Enrollment: 0
Sex: ALL
Minimum age (years): 6
Maximum age (years): 35
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: CROSSOVER
Primary purpose: TREATMENT

Brief summary: This is a placebo-controlled clinical trial to assess the utility of Guanfacine Extended Release (GXR) in the management of patients with Prader Willi Syndrome (PWS) who have significant aggression or self-injury. The purpose of this trial is to 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. 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.

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.

Primary outcomes:
measure: CGI-I scale rating
timeFrame: 19 Weeks
Secondary outcomes:
measure: Aberrant Behavior Checklist
timeFrame: 19 Weeks
measure: Self-Injury Trauma scale
timeFrame: 19 Weeks
measure: Modified Overt Aggression Scale
timeFrame: 19 Weeks
Other outcomes:
Locations:
city: New York
country: United States
facility: NYU Langone Health
state: New York
Study officials:
affiliation: New York Langone Health
name: Deepan Singh, MD
role: PRINCIPAL_INVESTIGATOR
References: