KINEDMD study: developing an activity monitoring biomarkerUsing novel algorithms to chart daily activities of ambulant and non-ambulant boys
The KineDMD study overseen by Imperial College London has been extended and is open for recruitment. The initial study recruited 16 Duchenne muscular dystrophy patients and 10 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).
To find out more about participating in this study, please contact us so that we can put you in touch with the team at GOSH.
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.
The DRF has awarded £320,000 to 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.
£320,000 in 2017-19
£270,000 in 2019-21
Learn more about this ground-breaking technology
The development and approval of disease-modifying treatments is often slowed down by the fact that it takes a long time to determine if a treatment works well or not for a group of patients. Many gold-standard methods for tracking disease progression rely on judgements ‘by eye’ in the clinic. This is especially true in ‘slow’ degenerative disorders such as Duchenne. We have now the unique opportunity to translate our artificial intelligence technology to the monitoring of Duchenne progression. We will give Duchenne boys wearable sensors (in the form of Apple watches), which will capture their arm and leg movement where it matters most to them: at home and in school. This movement data is collected discreetly, 24/7, and will be analysed by our artificial intelligence technology. This is a bit like having a personal neurologist looking after you day and night, day after day, who can notice even subtle changes in movement ability. We hope that these novel approaches will significantly improve the accuracy and reduce the time it takes to detect disease progression, potentially reducing the duration of future clinical trials and the cost of treatment development. We are delighted to have a forward looking clinical partner in Great Ormond Street’s Clinical Research Facility, with whom we will be working closely.Dr Aldo Faisal
Why are we supporting this project?
Despite the significant acceleration of therapeutic developments in the field of Duchenne in the last few years, a number of issues remain to be solved and require dedicated support and attention. We need better and more reliable outcome measures for boys with Duchenne, which is an area that has been insufficiently explored in the field so far. Characterisation of better outcome measures will enable the field to monitor disease progression and benefits of any therapeutic strategies in a more coherent and reliable manner. The newly funded projects supported by the Duchenne Research Fund will address those shortcomings and I am confident that the successful completion of those will make seminal contributions to the field of Duchenne.Dr Ronald Cohn
View our other newly funded research projects.
Find out how you can help us beat Duchenne.