Development of Assessments for Later Stage HD
LSA
1 other identifier
observational
340
2 countries
20
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for all trials
Started Sep 2020
20 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
April 24, 2020
CompletedFirst Posted
Study publicly available on registry
May 1, 2020
CompletedStudy Start
First participant enrolled
September 1, 2020
CompletedPrimary Completion
Last participant's last visit for primary outcome
March 1, 2022
CompletedStudy Completion
Last participant's last visit for all outcomes
March 1, 2022
CompletedMay 1, 2020
January 1, 2020
1.5 years
April 24, 2020
April 28, 2020
Conditions
Keywords
Outcome Measures
Primary Outcomes (2)
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
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
Eligibility Criteria
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.
You may qualify if:
- All Participants
- Individual of either gender
- Fluent in English
- Must be willing and able to provide informed consent or have a legal representative willing and able to provide assent
- Manifest HDGEC Participants
- 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.
- Age≥20 years
- CAG repeat length≥36
- DCL=4
- \. 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
You may not qualify if:
- People who meet the following criteria will be excluded from participating in this study:
- Individuals with choreic movement disorders in the context of a laboratory verified non-expansion mutation for the Huntingtin gene
- Manifest HDGEC Participants with Juvenile-onset HD (rater estimate of age of onset \<20 years old)
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (20)
The Board of Trustees of the University of Alabama for the University of Alabama at Birmingham
Birmingham, Alabama, 35294, United States
Regents of the University of California, San Diego
La Jolla, California, 92037, United States
Regents of the University of California, Los Angeles
Los Angeles, California, 90095, United States
Rocky Mountain Movement Disorders Center, P.C
Englewood, Colorado, 80113, United States
Rush Universiity Medical Center
Chicago, Illinois, 60612, United States
Hereditary Neurological Disease Centre, Inc.
Wichita, Kansas, 67226, United States
Washington University
St Louis, Missouri, 63110, United States
Vanderbilt University Medical Center
Nashville, Tennessee, 37212, United States
University of Washington
Seattle, Washington, 98195, United States
The Chancellor, Masters and Scholars of the University of Cambridge
Cambridge, Cambridgeshire, CB2 0PY, United Kingdom
Cardiff University
Wales, Cardiff, CF144XN, United Kingdom
Royal Devon and Exeter NHS Foundation Trust
Exeter, Devon, EX2 5DW, United Kingdom
Poole Hospital NHS
Poole, Dorset, BH15 2JB, United Kingdom
Leicestershire Partnership NHS Trust
Leicester, Leicestershire, LE2 2PL, United Kingdom
Oxford University Hospitals NHS Foundation Trust
Oxford, Leicester, OX3 9DU, United Kingdom
Northumberland, Tyne &Wear NHS Foundation Trust of St. Nicholas Hospital
Newcastle upon Tyne, Tyne & Wear, NE6 4QD, United Kingdom
Birmingham and Solihull Mental Health NHS Foundation
Birmingham, West Midlands, B152FG, United Kingdom
Leeds Teaching Hospitals NHS Trust
Leeds, Yorkshire, LS7 4SA, United Kingdom
University Hospitals Plymouth NHS Trust
Plymouth, PL6 8BQ, United Kingdom
Sheffield Children's NHS Foundation Trust
Sheffield, S5 7AU, United Kingdom
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Central Study Contacts
Study Design
- Study Type
- observational
- Observational Model
- OTHER
- Time Perspective
- PROSPECTIVE
- Target Duration
- 21 Days
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
April 24, 2020
First Posted
May 1, 2020
Study Start
September 1, 2020
Primary Completion
March 1, 2022
Study Completion
March 1, 2022
Last Updated
May 1, 2020
Record last verified: 2020-01