FuRST 2.0 Cognitive Pre-Testing

CHDI Foundation, Inc.

Status: Completed

Conditions: Huntington's Disease

Treatments: Behavioral: FuRST 2.0

Study type: Observational

Funder types: Other

Identifiers

NCT02881931

C-000316

Details and patient eligibility

About

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.

Enrollment: 75 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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

Trial design

75 participants in 3 patient groups

Pre-Manifest HDGEC Participant

Treatment:

Behavioral: FuRST 2.0

Early-Manifest HDGEC Participant

Treatment:

Behavioral: FuRST 2.0

Corresponding HDGEC participant Companion

Treatment:

Behavioral: FuRST 2.0