R-PROMS
Research type
Research Study
Full title
A feasibility study of Remote Patient-Reported Outcome measures in Mitochondrial disease and Spinocerebellar ataxias
IRAS ID
331433
Contact name
Yi Shiau Ng
Contact email
Clinicaltrials.gov Identifier
N/A, N/A
Duration of Study in the UK
1 years, 5 months, 30 days
Research summary
Ataxia is a condition that occurs due to damage to the cerebellum (the part of the brain controlling balance and movement).
Symptoms include difficulties with balance and walking, lack of limb co-ordination, slurred speech, abnormal eye movement and cognition issues. Such symptoms can have a significant negative impact on the lives of those affected.
Ataxia can have a number of causes including conditions such as Mitochondrial Disease (MD) and Spinocerebellar Ataxias (SCA).
Being able to measure and monitor the symptoms of ataxia is essential in managing such conditions and in the development of future treatments.
Lack of local specialist centres mean that people with rare diseases such as MD and SCA often need to travel large distances to access specialised care for their condition. This makes accessing care and participating in clinical research challenging especially for individuals with mobility problems.
A potential solution to address these challenges is remote assessment of ataxia symptoms. Since the COVID-19 pandemic, use of remote monitoring solutions and remote delivery of clinical research assessments (de-centralised clinical trials) has increased.
In this study we plan to investigate the use of remote assessments of ataxia symptoms in participants with MD and SCA.
Participants will complete a number of assessments of ataxia symptoms, speech, cognition and quality of life remotely via an online system. The results will be analysed to determine the usability of such assessments and to compare the results from data gathered during ‘traditional’ in person assessments.
Each participant will be seen in-person at the start of the study and will then complete remote assessments every two months for a period of one year.
This study is based on the SCA-Remote project undertaken by Monash University, Australia. The infrastructure (for online assessments) already developed for SCA-Remote, will be used.
REC name
South West - Cornwall & Plymouth Research Ethics Committee
REC reference
23/SW/0126
Date of REC Opinion
23 Oct 2023
REC opinion
Favourable Opinion