DT4RD
Research type
Research Study
Full title
Feasibility, validation and application of digital tools for the follow-up of neuromuscular patient mobility in daily living.
IRAS ID
325337
Contact name
Volker Straub
Contact email
Sponsor organisation
Association Institut de Myologie
Clinicaltrials.gov Identifier
Duration of Study in the UK
1 years, 6 months, 1 days
Research summary
The main objective is to develop a proof of concept of non-invasive tools for measuring rare disease patient mobility in daily living and test their feasibility and benefits and relationship to clinical measures conducted in the hospital. The approach is to propose a toolbox based on the following assessments: mobility, strength, range of motion, fatigue, pulmonary function, pain, cardiac, balance, ataxia, falls, tremor, acceptability of study and device utility. We hypothesise that measurements at home may be equivalent and / or relatable to measurements at the hospital owing to the facts that they are more frequent, less fatiguing and stressful for the patients and more related to real-life situations. Digital medicine products are evidence-based tools that support health research and medical practice. The ability to demonstrate reliability and meaningfulness for clinical trials should eventually translate into clinical use. The DT4RD protocol for the development of non-invasive tools for measuring rare disease patient mobility in daily living is aiming to help addressing this high unmet need. The DT4RD protocol benefits from the existing global setting and expertise in rare disease of the DT4RD consortium and the support from patient advocacy organisation ensuring that the outcome of the project is fit for regulatory purposes. DT4RD will also support rare disease patients moving towards decentralised clinical trials to assure their safety as patients and trial participants. The study will involve 40 participants aged between 12 and 60 years with a molecular-proven neuromuscular disorder and impaired ambulation. The primary objective is to develop and validate a non-invasive toolbox for measuring neuromuscular rare disease patient mobility at home by comparing the same or comparable measurements performed at home with those performed at the hospital and secondary objectives are to explore the feasibility of continuous, periodic and supervised assessment methods at home.
REC name
East Midlands - Nottingham 1 Research Ethics Committee
REC reference
23/EM/0134
Date of REC Opinion
22 Jun 2023
REC opinion
Unfavourable Opinion