FuRST 2.0 Cognitive Pre-Testing

Study Overview
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Brief Summary
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The FuRST 2.0 scale is being developed as a Patient Reported Outcome (PRO) with information coming directly from the Huntington's Disease Gene Expansion Carrier (HDGEC) and companion through self-report. The purpose of this study is to identify real or potential comprehension or usage problems with questionnaire items or response options. Through a process of structured cognitive de-briefing with HDGEC participants and companions, independently, followed by qualitative analysis, the final phrasing of the individual items and response options for the scale will be generated. Depending on the results of the first round of cognitive pre-testing, additional rounds of cognitive pre-testing may be required.

Official Title
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FuRST 2.0: Cognitive Pre-Testing Study for a New Functional Rating Scale for Use in Huntington's Disease

Conditions
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Huntington's Disease

Intervention / Treatment
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* Behavioral: FuRST 2.0


Participation Criteria
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Eligibility Criteria
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Main criteria for inclusion: HDGEC participant must be a participant in Enroll-HD (NCT No.: NCT01574053) At least 18 years of age Must be fluent in English and had his primary education in English Must be willing and able to provide written informed consent Pre-Manifest HDGECs Criteria 1-4, and: CAG length greater than or equal to 40 Disease Burden Score greater than or equal to 250 (calculated by the equation: [CAGn-35.5] X age) UHDRS Diagnostic Confidence Level (DCL) < 3 At least five Pre-Manifest HDGEC participants should have a companion who is willing to participate in this study and complete the scale independently. Early-Manifest (Stage 1&2) HDGECs Criteria 1-4, and: CAG length greater than or equal to 36 DCL=4 UHDRS Total Functional Capacity (TFC) ≥7 Participants whose companion is willing to participate in this study and complete the scale independently Main criteria for exclusion: Significant cognitive or any other impairment sufficient to interfere with study associated tasks as judged by the study Investigator or the Investigator's designee Currently participating in a clinical trial involving an investigational medicinal product

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 |
| --- | --- |
| Pre-Manifest HDGEC Participant<br> | Behavioral: FuRST 2.0<br>* Cognitive Pre-testing of new Functional Rating Scale<br>|
| Early-Manifest HDGEC Participant<br> | Behavioral: FuRST 2.0<br>* Cognitive Pre-testing of new Functional Rating Scale<br>|
| Corresponding HDGEC participant Companion<br> | Behavioral: FuRST 2.0<br>* Cognitive Pre-testing of new Functional Rating Scale<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Participants' comprehension of the FuRST 2.0 rating scale measured by qualitative analysis | The primary assessment will use qualitative analysis of the cognitive interview to determine the usability of the FuRST 2.0 scale in the HD population. | 8 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| The difference between the ratings of the HDGEC participants and the companions measured by the FuRST 2.0 rating scale | The secondary outcome measure will be evaluated using the Kolmogorov-Smirnov test | 8 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Huntington's disease, functional rating scale, cognitive pre-testing, patient reported outcome (PRO), HDGEC participant at Pre-Manifest stage, HDGEC participant at Early-Manifest stage, Corresponding HDGEC participant companion