Working…
COVID-19 is an emerging, rapidly evolving situation.
Get the latest public health information from CDC: https://www.coronavirus.gov.

Get the latest research information from NIH: https://www.nih.gov/coronavirus.
ClinicalTrials.gov
ClinicalTrials.gov Menu

Development of Assessments for Later Stage HD (LSA)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04370470
Recruitment Status : Not yet recruiting
First Posted : May 1, 2020
Last Update Posted : May 1, 2020
Sponsor:
Information provided by (Responsible Party):
CHDI Foundation, Inc.

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.

Condition or disease
Huntington Disease

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.

Layout table for study information
Study Type : Observational [Patient Registry]
Estimated Enrollment : 340 participants
Observational Model: Other
Time Perspective: Prospective
Target Follow-Up Duration: 21 Days
Official Title: Development of Assessments for Later Stage Huntington's Disease: UHDRS Structured Interview of Function and HD Clinical Status Questionnaire
Estimated Study Start Date : September 2020
Estimated Primary Completion Date : March 2022
Estimated Study Completion Date : March 2022

Resource links provided by the National Library of Medicine





Primary Outcome Measures :
  1. 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.

  2. 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)



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   20 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.

Criteria

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)

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04370470


Contacts
Layout table for location contacts
Contact: Jamie Levy +1-609-945-9600 jamie.levey@enroll-hd.org

Locations
Show Show 20 study locations
Sponsors and Collaborators
CHDI Foundation, Inc.
Layout table for additonal information
Responsible Party: CHDI Foundation, Inc.
ClinicalTrials.gov Identifier: NCT04370470    
Other Study ID Numbers: C-000619
First Posted: May 1, 2020    Key Record Dates
Last Update Posted: May 1, 2020
Last Verified: January 2020

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by CHDI Foundation, Inc.:
CHDI
Huntington Disease
Huntington's Disease
Huntington's Disease Later Stage
Additional relevant MeSH terms:
Layout table for MeSH terms
Huntington Disease
Basal Ganglia Diseases
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Dementia
Chorea
Dyskinesias
Movement Disorders
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Cognition Disorders
Neurocognitive Disorders
Mental Disorders