Clinical trial NCT04370470: Development of Assessments for Later Stage HD
Official title: Development of Assessments for Later Stage Huntington's Disease: UHDRS Structured Interview of Function and HD Clinical Status Questionnaire
Study type: OBSERVATIONAL
Overall status: UNKNOWN
Conditions: Huntington Disease
Keywords: CHDI
Huntington Disease
Huntington's Disease
Huntington's Disease Later Stage
Lead sponsor: CHDI Foundation, Inc.
Sponsor class: OTHER
First posted: 2020-05-01
Enrollment: 340
Sex: ALL
Minimum age (years): 20
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: OTHER
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: Manifest HDGEC Participants: Manifest HDGEC Participants must be participants in the Enroll-HD study. Manifest HDGEC Participants will be recruited from English-speaking study sites participating in the Enroll-HD study.

Companion Participants: Companions Participants will be identified for Manifest HDGEC Participants and asked to participate. A Companion Participant's participation for a Manifest HDGEC participant is mandatory.

Brief summary: Later Stage HD Assessments (LSA) is an observational, multinational study aiming at developing two assessments that can be used to measure critical milestones and events during the later stages of Huntington's disease (HD). An important aspect of the evaluation will be to assess whether the assessments can be administered to a companion either in-person or remotely (i.e. by phone contact with the companion). Therefore, these assessments will be evaluated for their internal consistency, reliability and validity. Once established, these assessments may be incorporated into a large scale, global observational study of HD and/or other HD clinical studies as well as use them for planning clinical trials.

Detailed description: The primary objective of this study is to evaluate the internal consistency, reliability and validity of two assessments: (1) UHDRS Structured Interview of Function (SIF) that incorporates the UHDRS Total Functional Capacity, Functional Assessment Scale, and Independence Scale (UHDRS TFC, FAS and IS), and (2) the HD Clinical Status Questionnaire (HDCSQ). There will be two study parts: Part 1 will measure the performance of the UHDRS SIF assessment compared to the original UHDRS TFC, FAS and IS using a cross-over design by collecting data from the Manifest HD gene expansion carrier participants (Manifest HDGEC Participants) and their companions (Companion Participants) via an in-person baseline visit and a follow-up visit by phone. Part 2 will assess the UHDRS SIF and the HDCSQ using a cross-sectional design by collecting data from the Companion Participants via a follow-up visit by phone. Participants of Part 1 are eligible to participate in Part 2.

Eligibility criteria: Inclusion Criteria:

Participants must meet all the following inclusion criteria to participate in this study:

All Participants

1. Individual of either gender
2. Fluent in English
3. Must be willing and able to provide informed consent or have a legal representative willing and able to provide assent

Manifest HDGEC Participants

1. Must be an active participant in the Enroll-HD study. An active participant is defined as a participant who has successfully enrolled in the Enroll-HD study and completed the Enroll-HD study baseline visit.
2. Age≥20 years
3. CAG repeat length≥36
4. DCL=4
5. Inclusion score of 16 at the time of the participant's most recent Enroll-HD study visit (for inclusion score calculation, see Appendix A - Inclusion Score Calculation) Companion Participants

1. Age≥18 years 2. A person who, in his/her opinion, has sufficient interface and knowledge of the Manifest HDGEC Participant's capabilities and daily activities 3. Acceptable to the Manifest HDGEC Participant and the Site Investigator or the Site Investigator's designees

Exclusion Criteria:

People who meet the following criteria will be excluded from participating in this study:

1. Individuals with choreic movement disorders in the context of a laboratory verified non-expansion mutation for the Huntingtin gene
2. Manifest HDGEC Participants with Juvenile-onset HD (rater estimate of age of onset \<20 years old)

Primary outcomes:
measure: The UHDRS SIF ratings
timeFrame: 3 weeks
measure: Clinimetric properties of the UHDRS SIF
timeFrame: 3 weeks
Secondary outcomes:
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: The Board of Trustees of the University of Alabama for the University of Alabama at Birmingham
state: Alabama
city: La Jolla
country: United States
facility: Regents of the University of California, San Diego
state: California
city: Los Angeles
country: United States
facility: Regents of the University of California, Los Angeles
state: California
city: Englewood
country: United States
facility: Rocky Mountain Movement Disorders Center, P.C
state: Colorado
city: Chicago
country: United States
facility: Rush Universiity Medical Center
state: Illinois
city: Wichita
country: United States
facility: Hereditary Neurological Disease Centre, Inc.
state: Kansas
city: Saint Louis
country: United States
facility: Washington University
state: Missouri
city: Nashville
country: United States
facility: Vanderbilt University Medical Center
state: Tennessee
city: Seattle
country: United States
facility: University of Washington
state: Washington
city: Cambridge
country: United Kingdom
facility: The Chancellor, Masters and Scholars of the University of Cambridge
state: Cambridgeshire
city: Wales
country: United Kingdom
facility: Cardiff University
state: Cardiff
city: Exeter
country: United Kingdom
facility: Royal Devon and Exeter NHS Foundation Trust
state: Devon
city: Poole
country: United Kingdom
facility: Poole Hospital NHS
state: Dorset
city: Leicester
country: United Kingdom
facility: Leicestershire Partnership NHS Trust
state: Leicestershire
city: Oxford
country: United Kingdom
facility: Oxford University Hospitals NHS Foundation Trust
state: Leicester
city: Newcastle Upon Tyne
country: United Kingdom
facility: Northumberland, Tyne &Wear NHS Foundation Trust of St. Nicholas Hospital
state: Tyne & Wear
city: Birmingham
country: United Kingdom
facility: Birmingham and Solihull Mental Health NHS Foundation
state: West Midlands
city: Leeds
country: United Kingdom
facility: Leeds Teaching Hospitals NHS Trust
state: Yorkshire
city: Plymouth
country: United Kingdom
facility: University Hospitals Plymouth NHS Trust
city: Sheffield
country: United Kingdom
facility: Sheffield Children's NHS Foundation Trust
Study officials:
References: