Development of the Virtual Unified Huntington's Disease Rating Scale

Study Overview
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Brief Summary
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The Huntington Study Group currently holds the registered trademark for the Unified Huntington's Disease Rating Scale. The UHDRS® is a research tool which has been developed by the HSG to provide a uniform assessment of the clinical features and course of HD. The UHDRS® has undergone extensive reliability and validity testing and has been used as a major outcome measure by the HSG in controlled clinical trials. The purpose of this research study is to determine if a standard HD assessment (the UHDRS®) is as reliable when conducted virtually as it is when conducted at an in-person visit (vUHDRS)

Detailed Description
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The UHDRS® is comprised of five components: Motor Assessment Cognitive Assessment Behavioral Assessment Independence Scale Functional Assessment Total Functional Capacity (TFC) This observational study seeks to characterize and compare in-person versus TeleVisits. Fifty percent of participants will be provided computer equipment for their remote assessment and the other 50% will use their existing home-based equipment for this purpose. TeleVisit is a method for participants to connect to the research study team remotely, via video and audio connection either on their computer or iPad.

Official Title
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Development of the Virtual Unified Huntington's Disease Rating Scale

Conditions
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Huntington Disease

Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Are male or female and 18 years or older at the time of signing the informed consent Are able and willing to provide written informed consent Are English speaking Currently exhibit motor signs and symptoms of Huntington Disease Have a genetic diagnosis of HD with an expanded CAG repeat (greater than or equal to 36) in the huntingtin (HTT) gene at or before Screening/Baseline visit from a validated laboratory Have oral and written communication skills that are sufficient/adequate to complete all cognitive assessments Are ambulatory, but may use any assistive device or require help Are able to maintain stable medications for 30 days prior to Screening/Baseline through the second in-person visit Have a study partner available to help with technology and set-up and to attend all study visits Have the ability to get email on the device used for the study Exclusion Criteria: Are unable to complete cognitive or functional assessments due to inability to communicate Are unable to obtain satisfactory internet connection (i.e., internet connection must be stable and support the audio and video needs without freezing or interruption) at home or designated location, even with provided equipment Have inadequate space at home to assess 10 steps for gait assessments Have active suicidal ideation endorsed with a score of 4 (Active Suicidal Ideation with Some Intent to Act, without Specific Plan) if the ideation occurred within 1 year of Screening or 5 (Active Suicidal Ideation with Specific Plan and Intent) on the C-SSRS if the attempt or acts were performed within 1 year of Screening/Baseline, or participants who, in the opinion of the Site Investigator (SI), present a serious risk of suicide. Have received gene therapy or an investigational drug within 30 days or 5 half-lives, whichever is longer, of the Screening/Baseline visit or plan to use an investigational drug during this study.

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Personal Equipment<br>Participants assigned to use technology they currently own | |
| HSG Equipment<br>Participants provided technology by the sponsor | |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Unified Huntington's Disease Rating Scale | Total Motor Score: Minimum Score = 0 Maximum Score = 124 Higher scores indicate more impaired motor functioning | from the TeleVisit to the second In-Person Visit, up to 6 weeks | 
| Unified Huntington's Disease Rating Scale | Total Functional Capacity: Minimum Score = 0 Maximum Score = 13 Greater scores indicate higher functioning | from the TeleVisit to the second In-Person Visit, up to 6 weeks | 
| United Huntington's Disease Rating Scale | Independence Scale Minimum Score = 0 Maximum Score = 100 Greater scores indicate greater independence | from the TeleVisit to the second In-Person Visit, up to 6 weeks | 
| United Huntington's Disease Rating Scale | Functional Assessment Minimum Score = 0 Maximum Score = 25 Higher scores indicate greater functionality | from TeleVisit to the second In-Person Visit, up to 6 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Safety of conducting the UHDRS remotely | Reported adverse events | from initial informed consent and continue until 30 days following the Participant's completion of the study | 
| Unified Huntington's Disease Rating Scale | Cognitive Function: Verbal Fluency minimum = 0, maximum is based upon participant responses Symbol Digit Modality (SDMT) minimum = 0, maximum = 110 Stroop minimum = 0, maximum is based upon participant responses Higher scores indicate greater cognitive function | from the TeleVisit to the second In-Person Visit, up to 6 weeks | 
| Unified Huntington's Disease Rating Scale | Total Motor Score Minimum Score = 0 Maximum Score = 124 Higher scores indicate more impaired motor functioning | Week 2, and Week 0 or Week 4 | 
| virtual Unified Huntington's Disease Rating Scale | Feasibility of completing the vUHDRS™ using participant personal equipment vs. Huntington Study Group provided equipment. | from first In-Person visit through Follow-Up, up to 6 weeks | 
| Unified Huntington's Disease Rating Scale | Behavior minimum = 0, maximum = 88 Higher scores indicate more behavioral symptoms | from the TeleVisit to the second In-Person Visit, up to 6 weeks |