KINEDMD study: developing an activity monitoring biomarker

Update: study extended into 2021/22. Please get in touch via research [at] duchenne.org.uk if you would like further information or are interested in taking part in the study, so we can put you in touch with the team at GOSH.

The KineDMD study overseen by Imperial College London in collaboration with Great Ormond Street Hospital initially recruited 21 Duchenne muscular dystrophy patients and 17 healthy control subjects to participate for 12 months. Both ambulant and non-ambulant boys with Duchenne aged 6-17 are eligible to take part. The study is non-mutation specific, and does not require any muscle biopsies or MRI. Boys are assessed at Great Ormond Street Hospital (GOSH). The study has now been extended and is recruiting.

KineDMD was recently featured on ITV’s Little Heroes TV show. Click here to read more.

During this 12 month study, participants wear a sensor suit on selected days (during clinical assessments at GOSH) allowing it to measure how their body interacts with the world around them. They also wear fitness tracker bracelets in the form of Apple watches throughout the trial, which will collect data on everyday movements at home/school.

Children are assessed at the National Institute for Health Research (NIHR) Clinical Research Facility at GOSH, overseen by Professor Thomas Voit and Dr Valeria Ricotti. The GOSH team work with chief investigator Dr Aldo Faisal from the Departments of Computing and Bioengineering at Imperial College London.

The bodysuit and tracker sensors feeds data back in real time, allowing the team to use artificial intelligence to make sense of the data patterns. From there, doctors are able to monitor disease progression with more precision. Should this study be successful, it will cut down on the amount of time taken to test new treatments, which will drive down the costs of future clinical trials.

Background:

The DRF has funded Dr Aldo Faisal’s team at Imperial College London to develop a novel biomarker to track the progression of Duchenne muscular dystrophy. The study will be called ‘KineDMD’. As part of the study, both ambulant and non-ambulant Duchenne patients will wear sensors in the form of four Apple watches (one on each wrist and one on each ankle) to monitor their daily activities and movements outside a clinical setting. Dr Faisal’s expertise is in developing a novel algorithm to analyse the data gathered from the sensor devices. On three occasions in the year the participants will then put on a sensor bodysuit just for their clinic appointment, in order to evaluate the link between the data gathered at home and their progress in clinic.

One of the major challenges in Duchenne drug development is linking measureable clinical trial endpoints with actual quality of life benefits. While there is a measurable and predictable decline in function during the course of the disease, each boy declines in a unique way, which can be hard to measure through classic functional tests like the six-minute walk test. This vital research aims to complement or even replace classic clinical endpoints, more sensitively capturing functional changes, and increasing the number of boys and young men eligible for clinical trials. This study will be carried out as a clinical natural history study at the Clinical Research Facility of Great Ormond Street Hospital Trust, overseen by Professor Thomas Voit.