MOVE FSHD
Research type
Research Study
Full title
Motor Outcomes to Validate Evaluations in FSHD (MOVE FSHD)
IRAS ID
313951
Contact name
Channa Hewamadduma
Contact email
Sponsor organisation
Sheffield Teaching Hospital NHS Foundation Trust
Clinicaltrials.gov Identifier
Duration of Study in the UK
4 years, 0 months, 0 days
Research summary
Facio-scapulo humeral dystrophy (FSHD) is one of the most common forms of muscular dystrophy, affecting children and adults. FSHD has a devastating impact on persons quality of life causing loss of mobility and arm function, with 20% of patients requiring a wheelchair as the disease progresses.
Currently there is no treatment or cure, however, there are several new treatments being developed. To ensure that these treatments are effective a better understanding of the natural history of FSHD is needed.
This study will be a natural history study over three years, studying changes to function over this period. This will be measured using several existing and innovative ‘outcome measures’ in a group of forty FSHD patients. We will assess muscle strength as a marker of disease change. We will collect patient reported changes in health. The information will inform the development of new outcome measures for clinical trials and provide a comprehensive understanding of a UK FSHD cohort and identify what changes are important to them. Conducting this project in the UK will help to establish a new generation of FSHD researchers.
Participants will be recruited through the Sheffield Teaching Hospitals Neuromuscular clinic and asked to perform these assessments at their usual annual clinic appointment. This will require a small amount of additional time in clinic over the three years.
Some participants will also be asked whether they would be willing to complete some remote assessments, to help better understand what can be reliably measured remotely. Should remote monitoring prove successful, it is hoped patients clinical visits can be better tailored to address patient’s needs, as their medical team will have more accurate and up to date information prior to each annual appointment.
The study is being funded via a charitable donation from a patient with FSHD to the Sheffield Hospitals Charity.
REC name
East of Scotland Research Ethics Service REC 1
REC reference
22/ES/0048
Date of REC Opinion
13 Dec 2022
REC opinion
Further Information Favourable Opinion