Back to resultsDose Clinical Trial of Guanfacine Extended Release for the Reduction of Aggression and Self-injuries Behavior Associated With Prader-Willi Syndrome
Primary Purpose
Prader-Willi Syndrome
Status
Withdrawn
Phase
Phase 4
Locations
United States
Study Type
Interventional
Intervention
Placebo
Guanfacine extended release (GXR)
Sponsored by
About this trial
This is an interventional treatment trial for Prader-Willi Syndrome
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.
Sites / Locations
- NYU Langone Health
Arms of the Study
Arm 1
Arm 2
Arm Type
Sham Comparator
Experimental
Arm Label
Placebo
GXR
Arm Description
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.
Outcomes
Primary Outcome Measures
CGI-I scale rating
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
Aberrant Behavior Checklist
Consists of 2 subscales; irritability (15 items) and hyperactivity/noncompliance (16 items).
Self-Injury Trauma scale
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
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
Full Information
NCT ID
NCT04066088
First Posted
August 21, 2019
Last Updated
August 24, 2020
Sponsor
NYU Langone Health
Collaborators
Winthrop University Hospital
1. Study Identification
Unique Protocol Identification Number
NCT04066088
Brief Title
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
2. Study Status
Record Verification Date
August 2020
Overall Recruitment Status
Withdrawn
Why Stopped
PI departure from institution
Study Start Date
December 1, 2019 (Actual)
Primary Completion Date
August 21, 2020 (Actual)
Study Completion Date
August 21, 2020 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
NYU Langone Health
Collaborators
Winthrop University Hospital
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Prader-Willi Syndrome
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Crossover Assignment
Masking
ParticipantInvestigator
Allocation
Randomized
Enrollment
0 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Placebo
Arm Type
Sham Comparator
Arm Title
GXR
Arm Type
Experimental
Arm Description
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.
Intervention Type
Other
Intervention Name(s)
Placebo
Intervention Description
Placebo will be administered same times as GXR
Intervention Type
Drug
Intervention Name(s)
Guanfacine extended release (GXR)
Intervention Description
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 Measure Information:
Title
CGI-I scale rating
Description
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.
Time Frame
19 Weeks
Secondary Outcome Measure Information:
Title
Aberrant Behavior Checklist
Description
Consists of 2 subscales; irritability (15 items) and hyperactivity/noncompliance (16 items).
Time Frame
19 Weeks
Title
Self-Injury Trauma scale
Description
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.
Time Frame
19 Weeks
Title
Modified Overt Aggression Scale
Description
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
Time Frame
19 Weeks
10. Eligibility
Sex
All
Minimum Age & Unit of Time
6 Years
Maximum Age & Unit of Time
35 Years
Accepts Healthy Volunteers
No
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Deepan Singh, MD
Organizational Affiliation
New York Langone Health
Official's Role
Principal Investigator
Facility Information:
Facility Name
NYU Langone Health
City
New York
State/Province
New York
ZIP/Postal Code
10016
Country
United States
12. IPD Sharing Statement
Learn more about this trial
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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