HEALTHE-RND
Research type
Research Study
Full title
European project title European eHealth Care Model for Rare Neurodegenerative Diseases: Development of HD-specific outcome measures UK study title Development and validation of multilingual, multinational HD specific need-based quality of life assessment tools: HD value assessment study
IRAS ID
276728
Contact name
Ramona Moldovan
Contact email
Sponsor organisation
Galen Research Ltd.
Clinicaltrials.gov Identifier
20/PR/0719, REC Reference
Duration of Study in the UK
1 years, 6 months, 1 days
Research summary
Huntington's Disease (HD) is a neurodegenerative disorder caused by an inherited defect in a single gene. HD is characterised by progressive motor dysfunction, cognitive decline and psychiatric disturbance. HD has a major impact on the lives of patients and their families. There is currently no cure, so management involves regular monitoring and treatment for problems as and when they occur. In order to determine whether various treatments improve the patient’s quality of life, it is necessary to produce scientific evidence.
The aim of the study is to develop and validate four quality of life instruments specific to HD for the following four groups: (1) pre-manifest (preHD) and (2) manifest carriers (mHD) of the HD expansion mutation as well as (3) companions (meaning informal caregivers being spouses or partners) of preHD and of (4) mHD carriers of the HD expansion mutation.
This will be done by combining qualitative and quantitative methodologies to develop four disease specific Patient Reported Outcome Measures (PROMs). The content of these measures will be derived entirely from patient and companions' interviews, thus ensuring that the issues covered are of importance and relevance to patients with HD. In subsequent stages of the study new groups of patients will evaluate the questionnaires in terms of its ease of completion, relevance to patients, completeness, accuracy and validity.
The new measure will then be used in studies to evaluate the effectiveness of treatments and to find out how best to provide treatment services. It is also intended that the questionnaire will be used in routine assessments to see whether patients are responding to their treatments.
REC name
London - West London & GTAC Research Ethics Committee
REC reference
20/PR/0719
Date of REC Opinion
10 Feb 2021
REC opinion
Further Information Favourable Opinion