Back to resultsRisperidone for the Treatment of Huntington's Disease Involuntary Movements
Primary Purpose
Huntington Disease, Chorea
Status
Suspended
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Risperidone
BioStamp nPoint device
Sponsored by
About this trial
This is an interventional treatment trial for Huntington Disease
Eligibility Criteria
Inclusion Criteria:
- Manifest HD (Diagnostic Confidence Level 4 + CAG repeat ≥ 37 or family history of HD)
- UHDRS Total Maximal Chorea (TMC) ≥ 8
- UHDRS Total Functional Capacity ≥ 5
- Subject willing and able to provide written informed consent OR legally authorized representative provides written informed consent and subject provides assent*
- Between 18 and 65 years of age
Exclusion Criteria:
- Use of antipsychotic, levodopa, dopamine agonist, monoamine oxidase inhibitor or other disallowed medication in the 30 days prior to the baseline visit (see Section 4.2.5)*
- Prior non-response to risperidone or intolerability to risperidone (in the investigator's opinion)
- Allergy or hypersensitivity to risperidone
- Dysphagia that in the investigator's opinion would preclude participation in the study
- Active suicidal ideation or psychiatric condition that in the investigator's opinion would preclude study participation
- QTc > 460 msec for women and QTc > 450 msec for men on 12-lead EKG
- History of cardiac arrhythmia or congenital long QT syndrome
- Significant renal impairment (creatinine clearance < 30 mL/min as estimated by the Cockgroft-Gault formula) or hepatic impairment (AST or ALT > 2.5 times upper limit of normal OR alkaline phosphatase or total bilirubin > 2 times upper limit of normal)
- Active drug or alcohol abuse or dependence
- Pregnant or breast-feeding
- Any contraindication to MRI (e.g. pacemakers, aneurysm clips, metallic prostheses, shrapnel fragments, claustrophobia)
- History of active (clinically significant) skin disorder that would interfere with sensor adherence
- History of allergic response to adhesives
- Pacemaker, AICD, or other implantable stimulator
- Use of an investigational drug in the 30 days prior to the baseline visit
- Inability to complete study activities, as determined by the study team
- Clinically significant parkinsonism as determined by expert investigator assessment
Sites / Locations
- URMC Neurology; 919 Westfall Rd, Building C, Suite 100
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Risperidone
Arm Description
Participants will initiate risperidone 0.5 mg nightly the day after the baseline visit. Dose assessment will occur at pre-specified intervals during the titration phase (week 2, 3, 4, 6, 7). The investigator will increase the dose by 0.5 mg at the week 2, week 3, week 4, and week 6 visits until either optimal chorea benefit has been obtained, an intolerable adverse event occurs, or the maximum allowable dose (3.0 mg) is reached.
Outcomes
Primary Outcome Measures
mean Unified Huntington's Disease (HD) Rating Scale Total Maximal Chorea (UHDRS TMC) score
The UHDRS is a validated assessment of HD. The complete total maximal chorea score is a subset of the overall motor assessment and measures maximal chorea with scores from 0 to 4 with higher scores indicating more chorea.
Secondary Outcome Measures
mean Unified Huntington's Disease (HD)Rating Scale total scores
The total UHDRS measures motor, cognitive, behavioral, functional, and total functional capacity. The score ranges from 0 to 161. Higher total scores indicate worse health outcomes.
mean Epworth Sleepiness Scale (ESS)
This tool measures excessive daytime sleepiness with higher scores indicating worse health outcomes. The scale range is 0 to 24.
mean Barnes Akathisia Scale
This tool measures drug-induced akathisia by objective observation and subjective questions. The scale range is 0 to 12 with higher scores indicating akathisia.
mean clinical global impression of change (CGI)
This tool is a observer-rated scale that measures impression of severity and change. It is rated on a 7 point scale from 1(very much improved) to 7 (very much worse). Higher score indicates no change.
mean patient global impression of change
This tool is a patient related scale that measures impression of change on a 7 point scale from 1 (very much improved) to 7 (very much worse). A higher score indicates no change.
Chorea Index as measured by BioStamp nPoint device
The average amount of chorea measured using the BioStamp nPoint device will be determined. Higher score indicates greater degree of chorea.
Q-Motor (quantitative motor) assessments of chorea
The Q-motor tool uses force transducers and a grip device to measure chorea completing four different tasks that assess fine motor finger and foot tapping speed, pronation/supination and gripping strength.
Short Problem Behavior Assessment form (Short PBA-S)
This tool measures different behavioural problems which are rated for both severity and frequency on a 5 point scale; severity and frequency ratings are then multiplied to provide an overall score for each symptom. The range is 0 to 132. Higher scores indicate worse health outcomes.
Columbia Suicide Severity Rating Scale( C-SSRS)
The number of participants expressing suicidal ideations will be determined by answering yes to any of the following questions: Have you wished you were dead or wished you could go to sleep and not wake up?, Have you actually had any thoughts of killing yourself?, Have you been thinking about how you might do this?, Have you had these thoughts and had some intention of acting on them?, Have you started to work out or worked out the details of how to kill yourself? Do you intend to carry out this plan? All questions require only a yes or no response. There is no numerical scoring or rating.
mean Apathy Scale
This tool measures the presence and severity of apathy. The range is 0 to 42 with higher scores indicating worse health outcomes
mean hospital anxiety and depression scale
This is a self-reported scale that measures anxiety and depression and ranges from 0 to 21 with a lower score indicating better health outcomes.
mean Montreal Cognitive assessment
The Montreal Cognitive assessment (MoCA) measures mild cognitive dysfunction. The total possible score is 30 points; a score of 26 or above is considered normal.
mean Unified Huntington's Disease(HD) Rating Scale Independence rating
the UHDRS component measures level of current independence. It ranges from 10 to 100 with higher scores indicating greater degree of independence.
Full Information
NCT ID
NCT04201834
First Posted
December 13, 2019
Last Updated
January 9, 2023
Sponsor
University of Rochester
1. Study Identification
Unique Protocol Identification Number
NCT04201834
Brief Title
Risperidone for the Treatment of Huntington's Disease Involuntary Movements
Official Title
Risperidone for the Treatment of Huntington's Disease Chorea
Study Type
Interventional
2. Study Status
Record Verification Date
January 2023
Overall Recruitment Status
Suspended
Why Stopped
Temporarily on pause due to updating protocol and other study measurements and design
Study Start Date
August 13, 2020 (Actual)
Primary Completion Date
December 30, 2023 (Anticipated)
Study Completion Date
December 30, 2023 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
University of Rochester
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
Yes
Device Product Not Approved or Cleared by U.S. FDA
Yes
Product Manufactured in and Exported from the U.S.
No
Data Monitoring Committee
No
5. Study Description
Brief Summary
The purpose of this study is to assess the safety and benefit of risperidone for the treatment of chorea (involuntary movements) in Huntington's disease. Risperidone is commonly used in clinical practice to treat chorea, however, it has not been approved by the Food and Drug Administration (FDA) to treat chorea. This study will examine 1) whether the investigators see MRI changes with risperidone treatment and 2) whether sensors applied to the participants body can measure chorea and detect changes in chorea.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Huntington Disease, Chorea
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Masking Description
Outcomes assessor will be masked to the visit.
Allocation
N/A
Enrollment
12 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Risperidone
Arm Type
Experimental
Arm Description
Participants will initiate risperidone 0.5 mg nightly the day after the baseline visit. Dose assessment will occur at pre-specified intervals during the titration phase (week 2, 3, 4, 6, 7). The investigator will increase the dose by 0.5 mg at the week 2, week 3, week 4, and week 6 visits until either optimal chorea benefit has been obtained, an intolerable adverse event occurs, or the maximum allowable dose (3.0 mg) is reached.
Intervention Type
Drug
Intervention Name(s)
Risperidone
Intervention Description
capsule or tablet, 0.5 mg
Intervention Type
Device
Intervention Name(s)
BioStamp nPoint device
Intervention Description
MC10 has developed the BioStamp nPointTM, a FDA 510(k) cleared medical device. This multimodal, reusable and rechargeable biosensor uses flexible and stretchable electronics to enable unobtrusive wear on the body and monitoring in the home. The sensors have accelerometry, gyroscopy, and ECG/EMG capabilities. A docking station enables wireless recharging and data collection.
Primary Outcome Measure Information:
Title
mean Unified Huntington's Disease (HD) Rating Scale Total Maximal Chorea (UHDRS TMC) score
Description
The UHDRS is a validated assessment of HD. The complete total maximal chorea score is a subset of the overall motor assessment and measures maximal chorea with scores from 0 to 4 with higher scores indicating more chorea.
Time Frame
week 12
Secondary Outcome Measure Information:
Title
mean Unified Huntington's Disease (HD)Rating Scale total scores
Description
The total UHDRS measures motor, cognitive, behavioral, functional, and total functional capacity. The score ranges from 0 to 161. Higher total scores indicate worse health outcomes.
Time Frame
Screening to week 12
Title
mean Epworth Sleepiness Scale (ESS)
Description
This tool measures excessive daytime sleepiness with higher scores indicating worse health outcomes. The scale range is 0 to 24.
Time Frame
Baseline to week 12
Title
mean Barnes Akathisia Scale
Description
This tool measures drug-induced akathisia by objective observation and subjective questions. The scale range is 0 to 12 with higher scores indicating akathisia.
Time Frame
Baseline to week 12
Title
mean clinical global impression of change (CGI)
Description
This tool is a observer-rated scale that measures impression of severity and change. It is rated on a 7 point scale from 1(very much improved) to 7 (very much worse). Higher score indicates no change.
Time Frame
Baseline to week 12
Title
mean patient global impression of change
Description
This tool is a patient related scale that measures impression of change on a 7 point scale from 1 (very much improved) to 7 (very much worse). A higher score indicates no change.
Time Frame
Baseline to week 12
Title
Chorea Index as measured by BioStamp nPoint device
Description
The average amount of chorea measured using the BioStamp nPoint device will be determined. Higher score indicates greater degree of chorea.
Time Frame
Screening to week 8
Title
Q-Motor (quantitative motor) assessments of chorea
Description
The Q-motor tool uses force transducers and a grip device to measure chorea completing four different tasks that assess fine motor finger and foot tapping speed, pronation/supination and gripping strength.
Time Frame
Baseline to week 8
Title
Short Problem Behavior Assessment form (Short PBA-S)
Description
This tool measures different behavioural problems which are rated for both severity and frequency on a 5 point scale; severity and frequency ratings are then multiplied to provide an overall score for each symptom. The range is 0 to 132. Higher scores indicate worse health outcomes.
Time Frame
Baseline to week 12
Title
Columbia Suicide Severity Rating Scale( C-SSRS)
Description
The number of participants expressing suicidal ideations will be determined by answering yes to any of the following questions: Have you wished you were dead or wished you could go to sleep and not wake up?, Have you actually had any thoughts of killing yourself?, Have you been thinking about how you might do this?, Have you had these thoughts and had some intention of acting on them?, Have you started to work out or worked out the details of how to kill yourself? Do you intend to carry out this plan? All questions require only a yes or no response. There is no numerical scoring or rating.
Time Frame
Baseline to week 12
Title
mean Apathy Scale
Description
This tool measures the presence and severity of apathy. The range is 0 to 42 with higher scores indicating worse health outcomes
Time Frame
Baseline to week 12
Title
mean hospital anxiety and depression scale
Description
This is a self-reported scale that measures anxiety and depression and ranges from 0 to 21 with a lower score indicating better health outcomes.
Time Frame
Baseline to week 12
Title
mean Montreal Cognitive assessment
Description
The Montreal Cognitive assessment (MoCA) measures mild cognitive dysfunction. The total possible score is 30 points; a score of 26 or above is considered normal.
Time Frame
Baseline visit only
Title
mean Unified Huntington's Disease(HD) Rating Scale Independence rating
Description
the UHDRS component measures level of current independence. It ranges from 10 to 100 with higher scores indicating greater degree of independence.
Time Frame
screening to week 12
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Manifest HD (Diagnostic Confidence Level 4 + CAG repeat ≥ 37 or family history of HD)
UHDRS Total Maximal Chorea (TMC) ≥ 8
UHDRS Total Functional Capacity ≥ 5
Subject willing and able to provide written informed consent OR legally authorized representative provides written informed consent and subject provides assent*
Between 18 and 65 years of age
Exclusion Criteria:
Use of antipsychotic, levodopa, dopamine agonist, monoamine oxidase inhibitor or other disallowed medication in the 30 days prior to the baseline visit (see Section 4.2.5)*
Prior non-response to risperidone or intolerability to risperidone (in the investigator's opinion)
Allergy or hypersensitivity to risperidone
Dysphagia that in the investigator's opinion would preclude participation in the study
Active suicidal ideation or psychiatric condition that in the investigator's opinion would preclude study participation
QTc > 460 msec for women and QTc > 450 msec for men on 12-lead EKG
History of cardiac arrhythmia or congenital long QT syndrome
Significant renal impairment (creatinine clearance < 30 mL/min as estimated by the Cockgroft-Gault formula) or hepatic impairment (AST or ALT > 2.5 times upper limit of normal OR alkaline phosphatase or total bilirubin > 2 times upper limit of normal)
Active drug or alcohol abuse or dependence
Pregnant or breast-feeding
Any contraindication to MRI (e.g. pacemakers, aneurysm clips, metallic prostheses, shrapnel fragments, claustrophobia)
History of active (clinically significant) skin disorder that would interfere with sensor adherence
History of allergic response to adhesives
Pacemaker, AICD, or other implantable stimulator
Use of an investigational drug in the 30 days prior to the baseline visit
Inability to complete study activities, as determined by the study team
Clinically significant parkinsonism as determined by expert investigator assessment
Facility Information:
Facility Name
URMC Neurology; 919 Westfall Rd, Building C, Suite 100
City
Rochester
State/Province
New York
ZIP/Postal Code
14618
Country
United States
12. IPD Sharing Statement
Plan to Share IPD
Undecided
Learn more about this trial
Risperidone for the Treatment of Huntington's Disease Involuntary Movements
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