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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
Sponsor:
Collaborator:
Winthrop University Hospital
Information provided by (Responsible Party):
NYU Langone Health

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.

Condition or disease Intervention/treatment Phase
Prader-Willi Syndrome Other: Placebo Drug: Guanfacine extended release (GXR) Phase 4

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.


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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 33 participants
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
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


Arm Intervention/treatment
Sham Comparator: Placebo Other: Placebo
Placebo will be administered same times as GXR

Experimental: 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.




Primary Outcome Measures :
  1. 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.


Secondary Outcome Measures :
  1. Aberrant Behavior Checklist [ Time Frame: 19 Weeks ]
    Consists of 2 subscales; irritability (15 items) and hyperactivity/noncompliance (16 items).

  2. 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.

  3. 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.[1] The rating scale is made up of four categories; verbal aggression, aggression against objects, aggression against self, and aggression against others



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Ages Eligible for Study:   6 Years to 35 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04066088


Contacts
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Contact: Nirmal Tejwani 212 598 6599 Nirmal.Tejwani@nyulangone.org

Locations
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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         
Sponsors and Collaborators
NYU Langone Health
Winthrop University Hospital
Investigators
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Principal Investigator: Deepan Singh, MD New York Langone Health

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Responsible Party: NYU Langone Health
ClinicalTrials.gov Identifier: NCT04066088     History of Changes
Other Study ID Numbers: 19-00936
First Posted: August 26, 2019    Key Record Dates
Last Update Posted: August 27, 2019
Last Verified: August 2019

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Prader-Willi Syndrome
Guanfacine
Syndrome
Aggression
Self-Injurious Behavior
Disease
Pathologic Processes
Behavioral Symptoms
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
Abnormalities, Multiple
Congenital Abnormalities
Chromosome Disorders
Genetic Diseases, Inborn
Obesity
Overnutrition
Nutrition Disorders
Antihypertensive Agents
Adrenergic alpha-2 Receptor Agonists
Adrenergic alpha-Agonists
Adrenergic Agonists
Adrenergic Agents
Neurotransmitter Agents
Molecular Mechanisms of Pharmacological Action
Physiological Effects of Drugs