Huntington DiseaseLSANCT04370470
Development of Assessments for Later Stage HD
An observational study of Huntington Disease, run by CHDI Foundation, Inc. at 20 sites in 2 countries.
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). Read more about this study
- Who is it for?
- Ages 20 years and older
- Conditions the study is for
- Huntington Disease
- How many people
- 340 participants
- Where
- 20 sitesin 2 countries
- Study dates
- Sep 2020to Mar 2022
- How long will I take part?
- 21 Days
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 20 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
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.
Who may be able to join
- Participants must meet all the following inclusion criteria to participate in this study:
- 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
Show all 12Show fewer
- 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
- 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
Who cannot join
- 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)
Trial locations and contacts
20 sites in 2 countries.
20 sites in 2 countries
United Kingdom11 sites
Birmingham and Solihull Mental Health NHS Foundation
Birmingham, West Midlands, B152FG
Ellice Parkinson, 01213014337, ellice.parkinsons@nhs.net
Hugh Rickards, MD
The Chancellor, Masters and Scholars of the University of Cambridge
Cambridge, Cambridgeshire, CB2 0PY
Priya Rogers, 01223 331160, pr466@cam.ac.uk
Roger Barker, MD
Royal Devon and Exeter NHS Foundation Trust
Exeter, Devon, EX2 5DW
Sarah Irvine, 01392406979, sarah.irvine@nhs.net
Timothy Harrower, MD
Leeds Teaching Hospitals NHS Trust
Leeds, Yorkshire, LS7 4SA
Callum Schofield, 01133924679, callum.schofield@nhs.net
Emma Hobson, MD
Leicestershire Partnership NHS Trust
Leicester, Leicestershire, LE2 2PL
Dawn Freire-Patino, 01162951996, dawn.freire-patino@leicspart.nhs.uk
Reza Kiani, MD
Northumberland, Tyne &Wear NHS Foundation Trust of St. Nicholas Hospital
Newcastle upon Tyne, Tyne & Wear, NE6 4QD
Sarah Edwards, 441912467392, sarah.edwards@ntw.nhs.uk
Suresh Komati, MD
Oxford University Hospitals NHS Foundation Trust
Oxford, Leicester, OX3 9DU
Zara Skitt, 01865231556, zara.skitt@ouh.nhs.uk
Sarsha Wilson, 0186521080, sarsha.wilson@ouh.nhs.uk
University Hospitals Plymouth NHS Trust
Plymouth, PL6 8BQ
Jemma Inches, 0175243845, jinches@nhs.net
Rupert Noad, MD
Poole Hospital NHS
Poole, Dorset, BH15 2JB
Annemieke Fox, annemieke.fox@poole.nhs.uk
John Burn, MD
Sheffield Children's NHS Foundation Trust
Sheffield, S5 7AU
Pauline Bayliss, 0114 2260828, pauline.bayliss@sch.nhs.uk
Oliver Quarrell
Cardiff University
Wales, Cardiff, CF144XN
United States9 sites
The Board of Trustees of the University of Alabama for the University of Alabama at Birmingham
Birmingham, Alabama, 35294
Jenna Smith, 205-996-2807, jltidwell@uabmc.edu
Victor Sung, MD
Rush Universiity Medical Center
Chicago, Illinois, 60612
Alice Negron, alice.negron@rush.edu
Deborah Hall, MD
Rocky Mountain Movement Disorders Center, P.C
Englewood, Colorado, 80113
Jessica Jaynes, 303-357-5456, jaynes@kumarneuro.com
Rajeev Kumar, MD
Regents of the University of California, San Diego
La Jolla, California, 92037
Chase Snell, 858-249-0569, cmsnell@ucsd.edu
Jody Corey-Bloom, MD
Regents of the University of California, Los Angeles
Los Angeles, California, 90095
Aaron Fisher, 310-206-8153, adfisher@mednet.ucla.edu
Susan Perlman, MD
Vanderbilt University Medical Center
Nashville, Tennessee, 37212
Elizabeth Huitz, 615-936-1007, elizabeth.huitz@vumc.org
Daniel O Claassen, MD
University of Washington
Seattle, Washington, 98195
Leigh Beth Latham, 206-685-6602, btlatham@uw.edu
Anny Lin, MD
Washington University
St Louis, Missouri, 63110
Stacey K Barton, 314-362-3471, bartons@wustl.edu
Joel S Perlmutter
Hereditary Neurological Disease Centre, Inc.
Wichita, Kansas, 67226
Gregory Suter, 316-609-3020, hndcentre@aol.com
William Mallonee, MD
About this study
Official title: 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.
Read the 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.
- Conditions
- Huntington Disease
- Keywords
- CHDI, Huntington Disease, Huntington's Disease, Huntington's Disease Later Stage
- Medical subject headings
- Huntington Disease
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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)
Measured: 3 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 May 2020
- Study startSep 2020 (estimated)
- Primary completionMar 2022 (estimated)
- Study completionMar 2022 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Huntington DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Other
- Time perspective
- Prospective
- Enrolment
- 340 participants (estimated)
- Target duration
- 21 Days
Sponsor and investigators
- Lead sponsor
- CHDI Foundation, Inc.
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04370470
- Sponsor's protocol ID
- C-000619
- Acronym
- LSA
- Status last verified
- Jan 2020
- First submitted
- 24 Apr 2020
- Last updated
- 1 May 2020
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 1 May 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Development of Assessments for Later Stage Huntington's Disease: UHDRS Structured Interview of Function and HD Clinical Status Questionnaire
- ClinicalTrials.gov ID
- NCT04370470
- Acronym / sponsor ID
- LSA / C-000619
- Conditions
- Huntington Disease
- Keywords
- CHDI, Huntington Disease, Huntington's Disease, Huntington's Disease Later Stage
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 340 participants (estimated)
- Lead sponsor
- CHDI Foundation, Inc.University, hospital or other
- Study start
- Sep 2020 (estimated)
- Primary completion
- Mar 2022 (estimated)
- Study completion
- Mar 2022 (estimated)
- First posted
- 1 May 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
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 design
- Observational model
- Other
- Time perspective
- Prospective
- Target follow-up
- 21 Days
Who may qualify
- Ages
- 20 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- 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.
Key inclusion criteria (12 listed)
- 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
Key exclusion criteria (3 listed)
- 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)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
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.
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)
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 May 2020.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
20 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Huntington Disease, run by CHDI Foundation, Inc. at 20 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 12 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 1 May 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 20 sites in United Kingdom, United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Jamie Levy (+1-609-945-9600, jamie.levey@enroll-hd.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The UHDRS SIF ratings (time frame: 3 weeks). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 May 2020.