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Clinical Trial Details — Status: Not yet recruiting

Administrative data

NCT number NCT04370470
Other study ID # C-000619
Secondary ID
Status Not yet recruiting
Phase
First received
Last updated
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
Health authority
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)

Study Design


Related Conditions & MeSH terms


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
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