Huntington's Disease
Conditions
Keywords
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
Brief summary
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.
Interventions
BEHAVIORALFuRST 2.0
Cognitive Pre-testing of new Functional Rating Scale
Sponsors
Dr. Glenn T. Stebbins (Rush University Medical Center )
The University of Texas Health Science Center, Houston
Nancy LaPelle
CHDI Foundation, Inc.
Study design
Observational model
OTHER
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
Main criteria for inclusion: 1. HDGEC participant must be a participant in Enroll-HD (NCT No.: NCT01574053) 2. At least 18 years of age 3. Must be fluent in English and had his primary education in English 4. Must be willing and able to provide written informed consent Pre-Manifest HDGECs Criteria 1-4, and: 1. CAG length greater than or equal to 40 2. Disease Burden Score greater than or equal to 250 (calculated by the equation: \[CAGn-35.5\] X age) 3. UHDRS Diagnostic Confidence Level (DCL) \< 3 4. 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: 1. CAG length greater than or equal to 36 2. DCL=4 3. UHDRS Total Functional Capacity (TFC) ≥7 4. Participants whose companion is willing to participate in this study and complete the scale independently Main criteria for exclusion: 1. Significant cognitive or any other impairment sufficient to interfere with study associated tasks as judged by the study Investigator or the Investigator's designee 2. Currently participating in a clinical trial involving an investigational medicinal product
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Participants' comprehension of the FuRST 2.0 rating scale measured by qualitative analysis | 8 months | 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| The difference between the ratings of the HDGEC participants and the companions measured by the FuRST 2.0 rating scale | 8 months | The secondary outcome measure will be evaluated using the Kolmogorov-Smirnov test |
Countries
Canada, United States
Outcome results
None listed