NCT03709173

Brief Summary

The study is a single, cross-sectional cognitive interview of FuRST 2.0, functional rating scale, administered to forty Huntington's Disease Gene Expansion Carriers (HDGECs) and potentially, their companions (the companion's participation is optional in this study). The scale will be tested as a patient reported outcome (PRO) in that the information will come directly from the HDGEC participant or the HDGEC participant together with his/her companion through self-report. The purpose is to identify real or potential comprehension or usage problems with scale items, response options, instructions and disclaimer statement, which are all components of the FuRST 2.0 scale. Through a structured cognitive interview with the HDGEC participants or the HDGEC participants together with their companions, followed by qualitative analysis, the final phrasing of the individual scale items, response options, instructions and disclaimer statement for the scale will be generated. Depending on the results of this study, an additional round of cognitive pre-testing may be required in a separate study.

Trial Health

90
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
52

participants targeted

Target at P25-P50 for all trials

Timeline
Completed

Started Aug 2019

Shorter than P25 for all trials

Geographic Reach
2 countries

4 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

September 25, 2018

Completed
22 days until next milestone

First Posted

Study publicly available on registry

October 17, 2018

Completed
10 months until next milestone

Study Start

First participant enrolled

August 2, 2019

Completed
3 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 25, 2019

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

October 25, 2019

Completed
Last Updated

October 29, 2019

Status Verified

October 1, 2019

Enrollment Period

3 months

First QC Date

September 25, 2018

Last Update Submit

October 28, 2019

Conditions

Outcome Measures

Primary Outcomes (1)

  • Participant's 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. Note: The instructions, items, response options and disclaimer statement are all part of the FuRST 2.0 scale.

    7 months

Study Arms (4)

Premanifest HDGEC participants

Behavioral: FuRST 2.0

Early-manifest HDGEC participants

Behavioral: FuRST 2.0

Companions of Premanifest HDGEC

Behavioral: FuRST 2.0

Companions of Early-manifest

Behavioral: FuRST 2.0

Interventions

FuRST 2.0BEHAVIORAL

Round 2 of cognitive pre-testing for a new functional rating scale

Companions of Early-manifestCompanions of Premanifest HDGECEarly-manifest HDGEC participantsPremanifest HDGEC participants

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

An HDGEC: Premanifest and early-manifest HDGEC who is also a participant in Enroll-HD and meets the protocol selection criteria for the HDGEC. HDGEC participants will be recruited from English speaking Enroll-HD sites. A Companion: A person who, in his/her opinion, has sufficient interaction and knowledge of the HDGEC participant's capabilities and daily activities, is acceptable to the HDGEC participant and the site principal investigator (site PI) or site PI's designee and meets the protocol selection criteria for the companion. The companion's participation is optional in this study.

You may qualify if:

  • Identified as an active participant in Enroll-HD (participants who have completed their last onsite Enroll-HD visit within approximately 15 months)
  • At least 18 years of age
  • Fluent in English and had his/her primary education in English
  • Able and willing to provide critical feedback (per site PI or site PI's designee discretion)
  • Willing and able to provide written informed consent
  • Cytosine, Adenine, Guanine (CAG) length ≥ 40
  • Disease burden score (DBS) ≥ 250
  • Diagnostic confidence level (DCL) ≤ 3
  • Identified as an active participant in Enroll-HD (participants who have completed their last onsite Enroll-HD visit within approximately 15 months)
  • At least 18 years of age
  • Fluent in English and had his/her primary education in English
  • Able and willing to provide critical feedback (per site PI or site PI's designee discretion)
  • Willing and able to provide written informed consent
  • CAG length ≥ 36
  • DCL= 4
  • +6 more criteria

You may not qualify if:

  • Significant cognitive or any other impairment sufficient to interfere with study associated tasks as judged by the site PI or the site PI's designee

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (4)

Rocky Mountain Movement Disorders Center, P.C.

Englewood, Colorado, 80113, United States

Location

Hereditary Neurological Disease Centre, Inc.

Wichita, Kansas, 67226, United States

Location

Columbia University

New York, New York, 10032, United States

Location

Centre for Movement Disorders (Neuropharm Consulting)

Toronto, Ontario, M3B 2S7, Canada

Location

MeSH Terms

Conditions

Huntington Disease

Condition Hierarchy (Ancestors)

Basal Ganglia DiseasesBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesDementiaChoreaDyskinesiasMovement DisordersHeredodegenerative Disorders, Nervous SystemNeurodegenerative DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesCognition DisordersNeurocognitive DisordersMental Disorders

Study Design

Study Type
observational
Observational Model
OTHER
Time Perspective
CROSS SECTIONAL
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

September 25, 2018

First Posted

October 17, 2018

Study Start

August 2, 2019

Primary Completion

October 25, 2019

Study Completion

October 25, 2019

Last Updated

October 29, 2019

Record last verified: 2019-10

Locations