HD-Charge: Indirect and Out-of-Pocket Costs of Huntington's Disease in the United States

September 19, 2022 updated by: CHDI Foundation, Inc.

Indirect and Out-of-Pocket Costs of Huntington's Disease in the United States

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.

Study Overview

Status

Completed

Intervention / Treatment

Detailed 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)

Study Type

Observational

Enrollment (Actual)

233

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

    • Arizona
      • Phoenix, Arizona, United States, 85013
        • St. Joseph's Hospital and Medical Center
    • Colorado
      • Englewood, Colorado, United States, 80113
        • Rocky Mountain Movement Disorder, P.C.
    • Florida
      • Tampa, Florida, United States, 33612
        • University of South Florida
    • Kansas
      • Wichita, Kansas, United States, 66226
        • Hereditary Neurological Disease Centre, Inc.
    • Missouri
      • Saint Louis, Missouri, United States, 63110
        • Washington University
    • New York
      • New York, New York, United States, 10032
        • Columbia University Medical Center
    • Ohio
      • Columbus, Ohio, United States, 43210
        • The Ohio State University
    • Pennsylvania
      • Pittsburgh, Pennsylvania, United States, 15213
        • University of Pittsburgh
    • Tennessee
      • Nashville, Tennessee, United States, 37212
        • Vanderbilt University Medical Center
    • Texas
      • Houston, Texas, United States, 77030
        • University of Texas Health Science Center at Houston
    • Washington
      • Seattle, Washington, United States, 98115
        • University of Washington

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

21 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

Study Population

HDGECs: Participant in Enroll-HD with clinically diagnosed HD and a cytosine, adenine, guanine (CAG) length ≥ 40, who meets the protocol inclusion criteria and none of the exclusion criteria.

Companions: Primary provider (unpaid) of supportive care for an HDGEC (this HDGEC must be an active participant of Enroll-HD Study, have a CAG ≥ 40, have an adult onset of disease (disease symptoms manifested at age 20 years or older), and a DCL=4)), who meets the protocol's companion inclusion criteria and none of the exclusion criteria.

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Other
  • Time Perspectives: Prospective

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
Early stage HDGECs
CAG length ≥ 40; DCL = 4, TFC ≥ 11
Online survey to capture indirect and out of pocket costs of Huntington's Disease in HDGECs and companions
Middle stage HDGECs
CAG length ≥ 40; DCL = 4, 7 ≤ TFC ≤ 10
Online survey to capture indirect and out of pocket costs of Huntington's Disease in HDGECs and companions
Late stage HDGECs
CAG length ≥ 40; DCL = 4, 0 ≤ TFC ≤ 6
Online survey to capture indirect and out of pocket costs of Huntington's Disease in HDGECs and companions
Companions of early stage HDGECs
Online survey to capture indirect and out of pocket costs of Huntington's Disease in HDGECs and companions
Companions of middle stage HDGECs
Online survey to capture indirect and out of pocket costs of Huntington's Disease in HDGECs and companions
Companions of late stage HDGECs
Online survey to capture indirect and out of pocket costs of Huntington's Disease in HDGECs and companions

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Quantify the indirect costs associated with productivity loss for HDGECs and companions
Time Frame: 12 months
Productivity loss is measured by annual lost wages and it is collected in the survey's employment questionnaires and WPAI
12 months
Quantify out-of-pocket expenses for HDGECs and companions
Time Frame: 12 months
The indirect costs associated with the total annual out-of-pocket expenses.
12 months
Identify the indirect costs associated with informal care received for HDGECs
Time Frame: 12 months
The total informal care time received during a typical week
12 months
Quantify the indirect costs associated with non-work caregiving time for companions
Time Frame: 12 months
The indirect costs associated with the annual opportunity costs of companion
12 months

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Identify the reasons for HDGEC and companion's unemployment or underemployment
Time Frame: 12 months
12 months
The proportion of Dual Role Companions, differentiated by HD stage of the companion
Time Frame: 12 months
12 months
To understand traditional disease staging system based on total functional capacity (TFC)
Time Frame: 12 months
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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Collaborators

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

June 21, 2020

Primary Completion (Actual)

June 21, 2022

Study Completion (Actual)

June 21, 2022

Study Registration Dates

First Submitted

May 29, 2018

First Submitted That Met QC Criteria

August 9, 2018

First Posted (Actual)

August 14, 2018

Study Record Updates

Last Update Posted (Actual)

September 21, 2022

Last Update Submitted That Met QC Criteria

September 19, 2022

Last Verified

September 1, 2022

More Information

Terms related to this study

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

Undecided

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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