Huntington's DiseaseNCT03628235
HD-Charge: Indirect and Out-of-Pocket Costs of Huntington's Disease in the United States
An observational study of Huntington's Disease, run by CHDI Foundation, Inc. at 11 sites in United States.
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. Read more about this study
- Who is it for?
- Ages 21 years and older
- Conditions the study is for
- Huntington's Disease
- What is being tested?
- Online survey
- Type of treatment
- Lifestyle or therapy programme
- How many people
- 233 participants
- Where
- 11 sitesUnited States
- Study dates
- 21 Jun 2020to 21 Jun 2022
- 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 21 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
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.
Who may be able to join
- 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)
Show all 16Show fewer
- Has access to a computer/tablet/smartphone (if compatible) and internet services
- Stage classification of HDGECs:
- Early stage HDGEC: CAG length ≥ 40; DCL = 4, TFC ≥ 11
- Middle stage HDGEC: CAG length ≥ 40; DCL = 4, 7 ≤ TFC ≤ 10
- Late stage HDGEC: CAG length ≥ 40; DCL = 4, 0 ≤ TFC ≤ 6
- 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
Who cannot join
- 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).
- 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.
Trial locations and contacts
11 sites in 1 country.
11 sites in 1 country
United States11 sites
The Ohio State University
Columbus, Ohio, 43210
Rocky Mountain Movement Disorder, P.C.
Englewood, Colorado, 80113
University of Texas Health Science Center at Houston
Houston, Texas, 77030
Vanderbilt University Medical Center
Nashville, Tennessee, 37212
Columbia University Medical Center
New York, New York, 10032
St. Joseph's Hospital and Medical Center
Phoenix, Arizona, 85013
University of Pittsburgh
Pittsburgh, Pennsylvania, 15213
University of Washington
Seattle, Washington, 98115
Washington University
St Louis, Missouri, 63110
University of South Florida
Tampa, Florida, 33612
Hereditary Neurological Disease Centre, Inc.
Wichita, Kansas, 66226
About this study
Official title: 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.
Read the detailed description
The survey collects the following data from each HDGEC and companion:
- Participant demographics (for HDGECs and companion)
- Informal care received (for HDGECs only)
- Out of pocket HD costs (for HDGECs and companion)
- Current employment status (for HDGECs and companion)
- Non-work and work care giving time that companion spends taking care of HDGEC (for companion only)
- Work Productivity and Activity Impairment (WPAI) Questionnaire (for HDGECs and companion)
- Conditions
- Huntington's Disease
- Being tested
- Online survey
- Keywords
- online survey, indirect costs, out-of-pocket costs, HD, Huntington's disease, companions, Enroll-HD
- Medical subject headings
- Huntington Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Online surveyBehavioural
Online survey to capture indirect and out of pocket costs of Huntington's Disease in HDGECs and companions
| Study group | Role | What they receive |
|---|---|---|
| Early stage HDGECs | CAG length ≥ 40; DCL = 4, TFC ≥ 11 Receives: Behavioral: Online survey | |
| Middle stage HDGECs | CAG length ≥ 40; DCL = 4, 7 ≤ TFC ≤ 10 Receives: Behavioral: Online survey | |
| Late stage HDGECs | CAG length ≥ 40; DCL = 4, 0 ≤ TFC ≤ 6 Receives: Behavioral: Online survey | |
| Companions of early stage HDGECs | Not described Receives: Behavioral: Online survey | |
| Companions of middle stage HDGECs | Not described Receives: Behavioral: Online survey | |
| Companions of late stage HDGECs | Not described Receives: Behavioral: Online survey |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 12 months
Quantify out-of-pocket expenses for HDGECs and companions
The indirect costs associated with the total annual out-of-pocket expenses.
Measured: 12 months
Identify the indirect costs associated with informal care received for HDGECs
The total informal care time received during a typical week
Measured: 12 months
Quantify the indirect costs associated with non-work caregiving time for companions
The indirect costs associated with the annual opportunity costs of companion
Measured: 12 months
Secondary outcomes (3)
Identify the reasons for HDGEC and companion's unemployment or underemployment
Measured: 12 months
The proportion of Dual Role Companions, differentiated by HD stage of the companion
Measured: 12 months
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)
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Aug 2018
- Study start21 Jun 2020
- Primary completion21 Jun 2022
- Study completion21 Jun 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Huntington's 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
- 233 participants (actual)
Sponsor and investigators
- Lead sponsor
- CHDI Foundation, Inc.
- Sponsor type
- University, hospital or other
- Collaborators
- IQVIA Company
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03628235
- Sponsor's protocol ID
- C-001117-1
- Status last verified
- Sep 2022
- First submitted
- 29 May 2018
- Last updated
- 21 Sep 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 21 Sep 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Indirect and Out-of-Pocket Costs of Huntington's Disease in the United States
- ClinicalTrials.gov ID
- NCT03628235
- Acronym / sponsor ID
- C-001117-1
- Conditions
- Huntington's Disease
- Keywords
- online survey, indirect costs, out-of-pocket costs, HD, Huntington's disease, companions, Enroll-HD
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 233 participants (actual)
- Lead sponsor
- CHDI Foundation, Inc.University, hospital or other
- Collaborators
- IQVIA Company
- Study start
- 21 Jun 2020 (actual)
- Primary completion
- 21 Jun 2022 (actual)
- Study completion
- 21 Jun 2022 (actual)
- First posted
- 14 Aug 2018 (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.
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.
Detailed description
The survey collects the following data from each HDGEC and companion:
- Participant demographics (for HDGECs and companion)
- Informal care received (for HDGECs only)
- Out of pocket HD costs (for HDGECs and companion)
- Current employment status (for HDGECs and companion)
- Non-work and work care giving time that companion spends taking care of HDGEC (for companion only)
- Work Productivity and Activity Impairment (WPAI) Questionnaire (for HDGECs and companion)
Study design
- Observational model
- Other
- Time perspective
- Prospective
- Number of arms
- 6
| Arm | Type | Receives |
|---|---|---|
| Early stage HDGECs | – | Behavioral: Online surveyCAG length ≥ 40; DCL = 4, TFC ≥ 11 |
| Middle stage HDGECs | – | Behavioral: Online surveyCAG length ≥ 40; DCL = 4, 7 ≤ TFC ≤ 10 |
| Late stage HDGECs | – | Behavioral: Online surveyCAG length ≥ 40; DCL = 4, 0 ≤ TFC ≤ 6 |
| Companions of early stage HDGECs | – | Behavioral: Online survey |
| Companions of middle stage HDGECs | – | Behavioral: Online survey |
| Companions of late stage HDGECs | – | Behavioral: Online survey |
Who may qualify
- Ages
- 21 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- 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.
Key inclusion criteria (16 listed)
- 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
Key exclusion criteria (2 listed)
- 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).
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Online surveyOnline survey to capture indirect and out of pocket costs of Huntington's Disease in HDGECs and companions | Behavioural | Companions of early stage HDGECs, Companions of late stage HDGECs, Companions of middle stage HDGECs, Early stage HDGECs, Late stage HDGECs, Middle stage HDGECs |
Primary outcome
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
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.
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
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
3 secondary outcomes
Identify the reasons for HDGEC and companion's unemployment or underemployment
Time frame: 12 months
The proportion of Dual Role Companions, differentiated by HD stage of the companion
Time frame: 12 months
To understand traditional disease staging system based on total functional capacity (TFC)
Time frame: 12 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Sep 2022.
- Individual participant dataSharing planned: Undecided
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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.
The record lists no contact. Use the official record to reach the sponsor.
11 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Huntington's Disease, run by CHDI Foundation, Inc. at 11 sites in United States.
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 16 inclusion and 2 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: finished, as of the sponsor's last update on 21 Sep 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 11 sites in 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Quantify the indirect costs associated with productivity loss for HDGECs and companions (time frame: 12 months). The study lists 4 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 21 Sep 2022.