Development of Assessments for Later Stage HD

NCT ID: NCT04370470

Last Updated: 2020-05-01

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

UNKNOWN

Total Enrollment

340 participants

Study Classification

OBSERVATIONAL

Study Start Date

2020-09-30

Study Completion Date

2022-03-31

Brief Summary

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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

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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.

Conditions

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Huntington Disease

Study Design

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Observational Model Type

OTHER

Study Time Perspective

PROSPECTIVE

Eligibility Criteria

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Inclusion Criteria

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)
Minimum Eligible Age

20 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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CHDI Foundation, Inc.

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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The Board of Trustees of the University of Alabama for the University of Alabama at Birmingham

Birmingham, Alabama, United States

Site Status

Regents of the University of California, San Diego

La Jolla, California, United States

Site Status

Regents of the University of California, Los Angeles

Los Angeles, California, United States

Site Status

Rocky Mountain Movement Disorders Center, P.C

Englewood, Colorado, United States

Site Status

Rush Universiity Medical Center

Chicago, Illinois, United States

Site Status

Hereditary Neurological Disease Centre, Inc.

Wichita, Kansas, United States

Site Status

Washington University

St Louis, Missouri, United States

Site Status

Vanderbilt University Medical Center

Nashville, Tennessee, United States

Site Status

University of Washington

Seattle, Washington, United States

Site Status

The Chancellor, Masters and Scholars of the University of Cambridge

Cambridge, Cambridgeshire, United Kingdom

Site Status

Cardiff University

Wales, Cardiff, United Kingdom

Site Status

Royal Devon and Exeter NHS Foundation Trust

Exeter, Devon, United Kingdom

Site Status

Poole Hospital NHS

Poole, Dorset, United Kingdom

Site Status

Leicestershire Partnership NHS Trust

Leicester, Leicestershire, United Kingdom

Site Status

Oxford University Hospitals NHS Foundation Trust

Oxford, Leicester, United Kingdom

Site Status

Northumberland, Tyne &Wear NHS Foundation Trust of St. Nicholas Hospital

Newcastle upon Tyne, Tyne & Wear, United Kingdom

Site Status

Birmingham and Solihull Mental Health NHS Foundation

Birmingham, West Midlands, United Kingdom

Site Status

Leeds Teaching Hospitals NHS Trust

Leeds, Yorkshire, United Kingdom

Site Status

University Hospitals Plymouth NHS Trust

Plymouth, , United Kingdom

Site Status

Sheffield Children's NHS Foundation Trust

Sheffield, , United Kingdom

Site Status

Countries

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United States United Kingdom

Central Contacts

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Jamie Levy

Role: CONTACT

+1-609-945-9600

Facility Contacts

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Jenna Smith

Role: primary

205-996-2807

Chase Snell

Role: primary

858-249-0569

Aaron Fisher

Role: primary

310-206-8153

Jessica Jaynes

Role: primary

303-357-5456

Alice Negron

Role: primary

Gregory Suter

Role: primary

316-609-3020

Stacey K Barton

Role: primary

314-362-3471

Elizabeth Huitz

Role: primary

615-936-1007

Leigh Beth Latham

Role: primary

206-685-6602

Priya Rogers

Role: primary

01223 331160

Sarah Irvine

Role: primary

01392406979

Annemieke Fox

Role: primary

Dawn Freire-Patino

Role: primary

01162951996

Zara Skitt

Role: primary

01865231556

Sarsha Wilson

Role: backup

0186521080

Sarah Edwards

Role: primary

441912467392

Ellice Parkinson

Role: primary

01213014337

Callum Schofield

Role: primary

01133924679

Jemma Inches

Role: primary

0175243845

Pauline Bayliss

Role: primary

0114 2260828

Other Identifiers

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C-000619

Identifier Type: -

Identifier Source: org_study_id

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