FuRST 2.0 Cognitive Pre-Testing

Status:	Completed
Conditions:	Neurology
Therapuetic Areas:	Neurology
Healthy:	No
Age Range:	18 - Any
Updated:	8/9/2017
Start Date:	February 28, 2017
End Date:	July 28, 2017

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

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.

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

We found this trial at

sites

Hereditary Neurology Disease Centre, Inc.

3223 North Webb Road
Wichita, Kansas 67226

Principal Investigator: William Mallonee

from

Wichita, KS