Clinical trial NCT02881931: FuRST 2.0 Cognitive Pre-Testing
Official title: FuRST 2.0: Cognitive Pre-Testing Study for a New Functional Rating Scale for Use in Huntington's Disease
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Huntington's Disease
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
Lead sponsor: CHDI Foundation, Inc.
Sponsor class: OTHER
Collaborators: name: Dr. Glenn T. Stebbins (Rush University Medical Center )
name: The University of Texas Health Science Center, Houston
name: Nancy LaPelle
First posted: 2016-08-29
Enrollment: 75
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: OTHER
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Participants: Individuals of either gender, equal to or greater than 18 years of age. HDGEC Pre-Manifest and Early-Manifest participants (see inclusion criteria) will be recruited from English speaking Enroll-HD sites.

Companions: Available companions for HDGEC Pre-Manifest participants will be encouraged to participate (at least five Pre-Manifest's companions are required). Companions will be identified for HDGEC Early-Manifest participants and asked to participate. Companion's participation for HDGEC Early-Manifest participant is mandatory as specified in the inclusion criteria.

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.


Eligibility 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

Primary outcomes:
measure: Participants' comprehension of the FuRST 2.0 rating scale measured by qualitative analysis
timeFrame: 8 months
Secondary outcomes:
measure: The difference between the ratings of the HDGEC participants and the companions measured by the FuRST 2.0 rating scale
timeFrame: 8 months
Other outcomes:
Locations:
city: Englewood
country: United States
facility: Rocky Mountain Movement Disorders Center, P.C.
state: Colorado
city: Wichita
country: United States
facility: Hereditary Neurology Disease Centre, Inc.
state: Kansas
city: New York
country: United States
facility: Columbia University
state: New York
city: Toronto
country: Canada
facility: CENTRE FOR MOVEMENT DISORDERS (Neuropharm Consulting)
state: Ontario
Study officials:
References: