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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT03628235
Other study ID # C-001117-1
Secondary ID
Status Completed
Phase
First received
Last updated
Start date June 21, 2020
Est. completion date June 21, 2022

Study information

Verified date September 2022
Source CHDI Foundation, Inc.
Contact n/a
Is FDA regulated No
Health authority
Study type Observational

Clinical Trial Summary

Huntington's disease (HD) is a rare, inherited and progressive neurodegenerative disorder for which hallmark symptoms include movement disorders, loss of cognitive faculties and psychiatric disturbances. With the progression of the disease, patients require increasing level of medical care, caregiver support, and long-term care, which lead to substantial burden of illness. Very little data are available on the direct or indirect costs for HD. The direct medical costs of HD in the US have been summarized from retrospective commercial and Medicaid claims data analysis. The indirect and out-of-pocket costs of HD in the US have not been quantified. This study will help to bridge these gaps. This study is a single-assessment, cross-sectional online survey administered to Huntington disease gene expansion carriers (HDGECs) and companions of HDGECs by HD stage to understand the indirect and out-of-pocket costs of Huntington's disease in the US.


Description:

The survey collects the following data from each HDGEC and companion: 1. Participant demographics (for HDGECs and companion) 2. Informal care received (for HDGECs only) 3. Out of pocket HD costs (for HDGECs and companion) 4. Current employment status (for HDGECs and companion) 5. Non-work and work care giving time that companion spends taking care of HDGEC (for companion only) 6. Work Productivity and Activity Impairment (WPAI) Questionnaire (for HDGECs and companion)


Recruitment information / eligibility

Status Completed
Enrollment 233
Est. completion date June 21, 2022
Est. primary completion date June 21, 2022
Accepts healthy volunteers No
Gender All
Age group 21 Years and older
Eligibility Inclusion Criteria for HDGECs: - Identified as an active participant in Enroll-HD (participants who have completed their last on site Enroll-HD visit within approximately 15 months) - Able to provide online or written informed consent. A LAR for HDGEC can provide written consent, if applicable - Identified as a participant with clinically diagnosed HD and has a CAG length = 40 - Site PI has access to HDGEC's medical records for the purpose of completing the Resource Utilization Questionnaire - Able to speak and understand both verbal and written English - Has adult onset of disease (disease symptoms manifested at age 20 years or older) - Has access to a computer/tablet/smartphone (if compatible) and internet services Stage classification of HDGECs: 1. Early stage HDGEC: CAG length = 40; DCL = 4, TFC = 11 2. Middle stage HDGEC: CAG length = 40; DCL = 4, 7 = TFC = 10 3. Late stage HDGEC: CAG length = 40; DCL = 4, 0 = TFC = 6 Exclusion Criteria for HDGECs: HDGECs will be excluded from the study if they have significant cognitive or any other impairment sufficient to interfere with study associated tasks as judged by the Site PI or Site PI's designee (exceptions will be made if a companion/LAR completes the survey on their behalf). Inclusion criteria for companions: - Able to provide online or written informed consent - Identified as the primary provider (unpaid) of supportive care of an HDGEC (HDGEC must be an Enroll-HD participant and have a CAG length = 40 and have adult onset of disease (disease symptoms manifested at age 20 years or older)) - Able to speak and understand both verbal and written English - Aged 21 years or older at time of survey - Has access to a computer/tablet/smartphone (if compatible) and internet services and has the technical skills required to complete an online survey Exclusion Criteria for companions: Companions will be excluded from the study if they are identified as paid or salaried caregivers; however, the HDGECs for whom they care may still be included in the study.

Study Design


Related Conditions & MeSH terms


Intervention

Behavioral:
Online survey
Online survey to capture indirect and out of pocket costs of Huntington's Disease in HDGECs and companions

Locations

Country Name City State
United States The Ohio State University Columbus Ohio
United States Rocky Mountain Movement Disorder, P.C. Englewood Colorado
United States University of Texas Health Science Center at Houston Houston Texas
United States Vanderbilt University Medical Center Nashville Tennessee
United States Columbia University Medical Center New York New York
United States St. Joseph's Hospital and Medical Center Phoenix Arizona
United States University of Pittsburgh Pittsburgh Pennsylvania
United States Washington University Saint Louis Missouri
United States University of Washington Seattle Washington
United States University of South Florida Tampa Florida
United States Hereditary Neurological Disease Centre, Inc. Wichita Kansas

Sponsors (2)

Lead Sponsor Collaborator
CHDI Foundation, Inc. IQVIA Company

Country where clinical trial is conducted

United States, 

Outcome

Type Measure Description Time frame Safety issue
Primary Quantify the indirect costs associated with productivity loss for HDGECs and companions Productivity loss is measured by annual lost wages and it is collected in the survey's employment questionnaires and WPAI 12 months
Primary Quantify out-of-pocket expenses for HDGECs and companions The indirect costs associated with the total annual out-of-pocket expenses. 12 months
Primary Identify the indirect costs associated with informal care received for HDGECs The total informal care time received during a typical week 12 months
Primary Quantify the indirect costs associated with non-work caregiving time for companions The indirect costs associated with the annual opportunity costs of companion 12 months
Secondary Identify the reasons for HDGEC and companion's unemployment or underemployment 12 months
Secondary The proportion of Dual Role Companions, differentiated by HD stage of the companion 12 months
Secondary To understand traditional disease staging system based on total functional capacity (TFC) For HDGECs and Companions: Understand how the traditional disease staging system, based on the TFC score, relates to a staging system based on resource utilization questionnaire and comorbid conditions developed in Divino et al. (2013) 12 months
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