Clinical trial NCT05748288: Development of the Virtual Unified Huntington's Disease Rating Scale
Official title: Development of the Virtual Unified Huntington's Disease Rating Scale
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Huntington Disease
Lead sponsor: Huntington Study Group
Sponsor class: NETWORK
First posted: 2023-02-28
Enrollment: 60
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: CASE_CONTROL
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Participants will be enrolled from clinics with physicians who are members of the Huntington Study Group

Brief summary: 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: 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.

Eligibility criteria: Inclusion Criteria:

1. Are male or female and 18 years or older at the time of signing the informed consent
2. Are able and willing to provide written informed consent
3. Are English speaking
4. Currently exhibit motor signs and symptoms of Huntington Disease
5. 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
6. Have oral and written communication skills that are sufficient/adequate to complete all cognitive assessments
7. Are ambulatory, but may use any assistive device or require help
8. Are able to maintain stable medications for 30 days prior to Screening/Baseline through the second in-person visit
9. Have a study partner available to help with technology and set-up and to attend all study visits
10. Have the ability to get email on the device used for the study

Exclusion Criteria:

1. Are unable to complete cognitive or functional assessments due to inability to communicate
2. 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
3. Have inadequate space at home to assess 10 steps for gait assessments
4. 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.
5. 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.

Primary outcomes:
measure: Unified Huntington's Disease Rating Scale
timeFrame: from the TeleVisit to the second In-Person Visit, up to 6 weeks
measure: Unified Huntington's Disease Rating Scale
timeFrame: from the TeleVisit to the second In-Person Visit, up to 6 weeks
measure: United Huntington's Disease Rating Scale
timeFrame: from the TeleVisit to the second In-Person Visit, up to 6 weeks
measure: United Huntington's Disease Rating Scale
timeFrame: from TeleVisit to the second In-Person Visit, up to 6 weeks
Secondary outcomes:
measure: Safety of conducting the UHDRS remotely
timeFrame: from initial informed consent and continue until 30 days following the Participant's completion of the study
measure: Unified Huntington's Disease Rating Scale
timeFrame: from the TeleVisit to the second In-Person Visit, up to 6 weeks
measure: Unified Huntington's Disease Rating Scale
timeFrame: Week 2, and Week 0 or Week 4
measure: virtual Unified Huntington's Disease Rating Scale
timeFrame: from first In-Person visit through Follow-Up, up to 6 weeks
measure: Unified Huntington's Disease Rating Scale
timeFrame: from the TeleVisit to the second In-Person Visit, up to 6 weeks
Other outcomes:
measure: Participant Satisfaction
timeFrame: from first In-Person visit through Follow-Up, up to 6 weeks
Locations:
city: Birmingham
country: United States
facility: University of Alabama at Birmingham, Department of Neurology
state: Alabama
city: Little Rock
country: United States
facility: University of Arkansas for Medical Sciences
state: Arkansas
city: La Jolla
country: United States
facility: University of California, San Diego
state: California
city: Washington
country: United States
facility: Georgetown University
state: District of Columbia
city: Tampa
country: United States
facility: University of South Florida, Huntington Disease Center of Excellence
state: Florida
city: Chicago
country: United States
facility: Northwestern University Medical Center, Department of Neurology
state: Illinois
city: Chicago
country: United States
facility: Rush University Medical Center
state: Illinois
city: Indianapolis
country: United States
facility: Indiana University School of Medicine
state: Indiana
city: Wichita
country: United States
facility: Hereditary Neurological Disease Center
state: Kansas
city: Boston
country: United States
facility: Beth Israel Deaconess Medical Center
state: Massachusetts
city: Charlestown
country: United States
facility: Massachusetts General Hospital
state: Massachusetts
city: Saint Louis
country: United States
facility: Washington University
state: Missouri
city: Fargo
country: United States
facility: Sanford Brain and Spine Clinic
state: North Dakota
city: Columbus
country: United States
facility: Ohio State University Medical Center
state: Ohio
city: Pittsburgh
country: United States
facility: University of Pittsburgh
state: Pennsylvania
city: Houston
country: United States
facility: University of Texas, Health Science Center
state: Texas
city: Burlington
country: United States
facility: University of Vermont Medical Center
state: Vermont
Study officials:
affiliation: Huntington Study Group
name: Samuel Frank, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Huntington Study Group
name: Jody Goldstein
role: STUDY_DIRECTOR
References: