Development of Assessments for Later Stage HD

Huntington Disease Clinical Trial

— LSA  

Official title:

Development of Assessments for Later Stage Huntington's Disease: UHDRS Structured Interview of Function and HD Clinical Status Questionnaire

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT04370470
• Other study ID #: C-000619
• Status: Not yet recruiting
• Start date: September 2020
• Est. completion date: March 2022

Study information

• Verified date: January 2020
• Source: CHDI Foundation, Inc.
• Contact: Jamie Levy
• Phone: +1-609-945-9600
• Email: jamie.levey[@]enroll-hd.org
• Is FDA regulated: No
• Study type: Observational [Patient Registry]

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 340
• Est. completion date: March 2022
• Est. primary completion date: March 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: 20 Years and older

Eligibility

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)

Related Conditions & MeSH terms

• Huntington Disease

Locations

Country	Name	City	State
United Kingdom	Birmingham and Solihull Mental Health NHS Foundation	Birmingham	West Midlands
United Kingdom	The Chancellor, Masters and Scholars of the University of Cambridge	Cambridge	Cambridgeshire
United Kingdom	Royal Devon and Exeter NHS Foundation Trust	Exeter	Devon
United Kingdom	Leeds Teaching Hospitals NHS Trust	Leeds	Yorkshire
United Kingdom	Leicestershire Partnership NHS Trust	Leicester	Leicestershire
United Kingdom	Northumberland, Tyne &Wear NHS Foundation Trust of St. Nicholas Hospital	Newcastle Upon Tyne	Tyne & Wear
United Kingdom	Oxford University Hospitals NHS Foundation Trust	Oxford	Leicester
United Kingdom	University Hospitals Plymouth NHS Trust	Plymouth
United Kingdom	Poole Hospital NHS	Poole	Dorset
United Kingdom	Sheffield Children's NHS Foundation Trust	Sheffield
United Kingdom	Cardiff University	Wales	Cardiff
United States	The Board of Trustees of the University of Alabama for the University of Alabama at Birmingham	Birmingham	Alabama
United States	Rush Universiity Medical Center	Chicago	Illinois
United States	Rocky Mountain Movement Disorders Center, P.C	Englewood	Colorado
United States	Regents of the University of California, San Diego	La Jolla	California
United States	Regents of the University of California, Los Angeles	Los Angeles	California
United States	Vanderbilt University Medical Center	Nashville	Tennessee
United States	Washington University	Saint Louis	Missouri
United States	University of Washington	Seattle	Washington
United States	Hereditary Neurological Disease Centre, Inc.	Wichita	Kansas

Sponsors (1)

Lead Sponsor	Collaborator
CHDI Foundation, Inc.

Countries where clinical trial is conducted

United States,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	The UHDRS SIF ratings	The equivalence of the original UHDRS function scales (TFC, FAS and IS) and the UHDRS SIF will be evaluated using a cross-over design study, with two baseline measurements and one follow up measurement. We will use ICC (Intraclass Correlation Coefficient) and Lin's correlation coefficient will be used to measure the agreement between raters across Participants.	3 weeks
Primary	Clinimetric properties of the UHDRS SIF	To assess the clinimetric properties of the UHDRS SIF we will use Classical Test Theory (CTT) and Item Response Theory (IRT)	3 weeks