NCT03628235

Brief 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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
233

participants targeted

Target at P75+ for all trials

Timeline
Completed

Started Jun 2020

Geographic Reach
1 country

11 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

May 29, 2018

Completed
3 months until next milestone

First Posted

Study publicly available on registry

August 14, 2018

Completed
1.9 years until next milestone

Study Start

First participant enrolled

June 21, 2020

Completed
2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 21, 2022

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

June 21, 2022

Completed
Last Updated

September 21, 2022

Status Verified

September 1, 2022

Enrollment Period

2 years

First QC Date

May 29, 2018

Last Update Submit

September 19, 2022

Conditions

Keywords

online surveyindirect costsout-of-pocket costsHDHuntington's diseasecompanionsEnroll-HD

Outcome Measures

Primary Outcomes (4)

  • 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

  • Quantify out-of-pocket expenses for HDGECs and companions

    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

    The total informal care time received during a typical week

    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

    12 months

Secondary Outcomes (3)

  • Identify the reasons for HDGEC and companion's unemployment or underemployment

    12 months

  • The proportion of Dual Role Companions, differentiated by HD stage of the companion

    12 months

  • To understand traditional disease staging system based on total functional capacity (TFC)

    12 months

Study Arms (6)

Early stage HDGECs

CAG length ≥ 40; DCL = 4, TFC ≥ 11

Behavioral: Online survey

Middle stage HDGECs

CAG length ≥ 40; DCL = 4, 7 ≤ TFC ≤ 10

Behavioral: Online survey

Late stage HDGECs

CAG length ≥ 40; DCL = 4, 0 ≤ TFC ≤ 6

Behavioral: Online survey

Companions of early stage HDGECs

Behavioral: Online survey

Companions of middle stage HDGECs

Behavioral: Online survey

Companions of late stage HDGECs

Behavioral: Online survey

Interventions

Online surveyBEHAVIORAL

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

Companions of early stage HDGECsCompanions of late stage HDGECsCompanions of middle stage HDGECsEarly stage HDGECsLate stage HDGECsMiddle stage HDGECs

Eligibility Criteria

Age21 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)
Sampling MethodNon-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.

You may qualify if:

  • 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:
  • 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

You may not qualify if:

  • 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).
  • 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
  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (11)

St. Joseph's Hospital and Medical Center

Phoenix, Arizona, 85013, United States

Location

Rocky Mountain Movement Disorder, P.C.

Englewood, Colorado, 80113, United States

Location

University of South Florida

Tampa, Florida, 33612, United States

Location

Hereditary Neurological Disease Centre, Inc.

Wichita, Kansas, 66226, United States

Location

Washington University

St Louis, Missouri, 63110, United States

Location

Columbia University Medical Center

New York, New York, 10032, United States

Location

The Ohio State University

Columbus, Ohio, 43210, United States

Location

University of Pittsburgh

Pittsburgh, Pennsylvania, 15213, United States

Location

Vanderbilt University Medical Center

Nashville, Tennessee, 37212, United States

Location

University of Texas Health Science Center at Houston

Houston, Texas, 77030, United States

Location

University of Washington

Seattle, Washington, 98115, United States

Location

MeSH Terms

Conditions

Huntington Disease

Condition Hierarchy (Ancestors)

Basal Ganglia DiseasesBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesDementiaChoreaDyskinesiasMovement DisordersHeredodegenerative Disorders, Nervous SystemNeurodegenerative DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesCognition DisordersNeurocognitive DisordersMental Disorders

Study Design

Study Type
observational
Observational Model
OTHER
Time Perspective
PROSPECTIVE
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

May 29, 2018

First Posted

August 14, 2018

Study Start

June 21, 2020

Primary Completion

June 21, 2022

Study Completion

June 21, 2022

Last Updated

September 21, 2022

Record last verified: 2022-09

Locations