Development of the Virtual Unified Huntington's Disease Rating Scale
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 60
- 试验地点
- 17
- 主要终点
- Unified Huntington's Disease Rating Scale
研究概览
简要总结
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)
详细描述
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.
研究设计
- 研究类型
- Observational
- 观察模型
- Case Control
- 时间视角
- Prospective
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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
排除标准
- •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.
结局指标
主要结局
Unified Huntington's Disease Rating Scale
时间窗: from the TeleVisit to the second In-Person Visit, up to 6 weeks
Total Functional Capacity: Minimum Score = 0 Maximum Score = 13 Greater scores indicate higher functioning
United Huntington's Disease Rating Scale
时间窗: from TeleVisit to the second In-Person Visit, up to 6 weeks
Functional Assessment Minimum Score = 0 Maximum Score = 25 Higher scores indicate greater functionality
次要结局
- Unified Huntington's Disease Rating Scale(from the TeleVisit to the second In-Person Visit, up to 6 weeks)
- virtual Unified Huntington's Disease Rating Scale(from first In-Person visit through Follow-Up, up to 6 weeks)
- Safety of conducting the UHDRS remotely(from initial informed consent and continue until 30 days following the Participant's completion of the study)