FuRST 2.0 Cognitive Pre-Testing

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.

Study Overview

• Status: Completed
• Conditions: Huntington's Disease
• Intervention / Treatment: Behavioral: FuRST 2.0

• Study Type: Observational
• Enrollment (Actual): 75

Contacts and Locations

Study Locations

• Canada
  • Ontario
    • Toronto, Ontario, Canada, M3B 2S7
      • Centre for Movement Disorders (Neuropharm Consulting)
• United States
  • Colorado
    • Englewood, Colorado, United States, 80113
      • Rocky Mountain Movement Disorders Center, P.C.
  • Kansas
    • Wichita, Kansas, United States, 67226
      • Hereditary Neurology Disease Centre, Inc.
  • New York
    • New York, New York, United States, 10032
      • Columbia University

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 14 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• 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.

Description

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

Study Plan

How is the study designed?

Design Details

• Observational Models: Other
• Time Perspectives: Prospective

Number of groups / cohorts

3

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Pre-Manifest HDGEC Participant	Behavioral: FuRST 2.0; Cognitive Pre-testing of new Functional Rating Scale
Early-Manifest HDGEC Participant	Behavioral: FuRST 2.0; Cognitive Pre-testing of new Functional Rating Scale
Corresponding HDGEC participant Companion	Behavioral: FuRST 2.0; Cognitive Pre-testing of new Functional Rating Scale

What is the study measuring?

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

Collaborators and Investigators

• Sponsor: CHDI Foundation, Inc.
• Collaborators: The University of Texas Health Science Center, Houston; Dr. Glenn T. Stebbins (Rush University Medical Center ); Nancy LaPelle

Study record dates

Study Major Dates

• Study Start (Actual): February 28, 2017
• Primary Completion (Actual): July 28, 2017
• Study Completion (Actual): July 28, 2017

Study Registration Dates

• First Submitted: August 12, 2016
• First Submitted That Met QC Criteria: August 24, 2016
• First Posted (Estimate): August 29, 2016

Study Record Updates

• Last Update Posted (Actual): August 8, 2017
• Last Update Submitted That Met QC Criteria: August 7, 2017
• Last Verified: August 1, 2017

More Information

Terms related to this study

• 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
• Additional Relevant MeSH Terms: Mental Disorders; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Neurocognitive Disorders; Genetic Diseases, Inborn; Basal Ganglia Diseases; Movement Disorders; Neurodegenerative Diseases; Dyskinesias; Heredodegenerative Disorders, Nervous System; Dementia; Cognition Disorders; Chorea; Huntington Disease
• Other Study ID Numbers: C-000316

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: Undecided