Development of Assessments for Later Stage HD (LSA)

April 28, 2020 updated by: CHDI Foundation, Inc.

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

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.

Study Overview

Status

Unknown

Conditions

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.

Study Type

Observational

Enrollment (Anticipated)

340

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • United Kingdom
      • Plymouth, United Kingdom, PL6 8BQ
        • University Hospitals Plymouth NHS Trust
        • Contact:
        • Principal Investigator:
          • Rupert Noad, MD
      • Sheffield, United Kingdom, S5 7AU
        • Sheffield Children's NHS Foundation Trust
        • Contact:
        • Principal Investigator:
          • Oliver Quarrell
    • Cambridgeshire
      • Cambridge, Cambridgeshire, United Kingdom, CB2 0PY
        • The Chancellor, Masters and Scholars of the University of Cambridge
        • Contact:
        • Principal Investigator:
          • Roger Barker, MD
    • Cardiff
      • Wales, Cardiff, United Kingdom, CF144XN
        • Cardiff University
    • Devon
      • Exeter, Devon, United Kingdom, EX2 5DW
        • Royal Devon and Exeter NHS Foundation Trust
        • Contact:
        • Principal Investigator:
          • Timothy Harrower, MD
    • Dorset
      • Poole, Dorset, United Kingdom, BH15 2JB
    • Leicester
      • Oxford, Leicester, United Kingdom, OX3 9DU
        • Oxford University Hospitals NHS Foundation Trust
        • Contact:
        • Contact:
        • Principal Investigator:
          • Richard Armstrong, MD
    • Leicestershire
      • Leicester, Leicestershire, United Kingdom, LE2 2PL
    • Tyne & Wear
      • Newcastle Upon Tyne, Tyne & Wear, United Kingdom, NE6 4QD
        • Northumberland, Tyne &Wear NHS Foundation Trust of St. Nicholas Hospital
        • Principal Investigator:
          • Suresh Komati, MD
        • Contact:
    • West Midlands
      • Birmingham, West Midlands, United Kingdom, B152FG
        • Birmingham and Solihull Mental Health NHS Foundation
        • Contact:
        • Principal Investigator:
          • Hugh Rickards, MD
    • Yorkshire
      • Leeds, Yorkshire, United Kingdom, LS7 4SA
        • Leeds Teaching Hospitals NHS Trust
        • Contact:
        • Principal Investigator:
          • Emma Hobson, MD
  • United States
    • Alabama
      • Birmingham, Alabama, United States, 35294
        • The Board of Trustees of the University of Alabama for the University of Alabama at Birmingham
        • Contact:
        • Principal Investigator:
          • Victor Sung, MD
    • California
      • La Jolla, California, United States, 92037
        • Regents of the University of California, San Diego
        • Contact:
        • Principal Investigator:
          • Jody Corey-Bloom, MD
      • Los Angeles, California, United States, 90095
        • Regents of the University of California, Los Angeles
        • Contact:
        • Principal Investigator:
          • Susan Perlman, MD
    • Colorado
      • Englewood, Colorado, United States, 80113
        • Rocky Mountain Movement Disorders Center, P.C
        • Contact:
    • Illinois
      • Chicago, Illinois, United States, 60612
        • Rush Universiity Medical Center
        • Contact:
        • Principal Investigator:
          • Deborah Hall, MD
    • Kansas
      • Wichita, Kansas, United States, 67226
        • Hereditary Neurological Disease Centre, Inc.
        • Contact:
        • Principal Investigator:
          • William Mallonee, MD
    • Missouri
      • Saint Louis, Missouri, United States, 63110
        • Washington University
        • Contact:
        • Principal Investigator:
          • Joel S Perlmutter
    • Tennessee
      • Nashville, Tennessee, United States, 37212
        • Vanderbilt University Medical Center
        • Contact:
        • Principal Investigator:
          • Daniel O Claassen, MD
    • Washington
      • Seattle, Washington, United States, 98195
        • University of Washington
        • Contact:
        • Principal Investigator:
          • Anny Lin, MD

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

20 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

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.

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Other
  • Time Perspectives: Prospective

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
The UHDRS SIF ratings
Time Frame: 3 weeks
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
Clinimetric properties of the UHDRS SIF
Time Frame: 3 weeks
To assess the clinimetric properties of the UHDRS SIF we will use Classical Test Theory (CTT) and Item Response Theory (IRT)
3 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

CHDI Foundation, Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Anticipated)

September 1, 2020

Primary Completion (Anticipated)

March 1, 2022

Study Completion (Anticipated)

March 1, 2022

Study Registration Dates

First Submitted

April 24, 2020

First Submitted That Met QC Criteria

April 28, 2020

First Posted (Actual)

May 1, 2020

Study Record Updates

Last Update Posted (Actual)

May 1, 2020

Last Update Submitted That Met QC Criteria

April 28, 2020

Last Verified

January 1, 2020

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • C-000619

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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