Duchenne UK announces exciting new project to fund the development of digital technology, to help develop and evaluate potential new approaches to measuring efficacy and safety in clinical trials.

Duchenne UK is very pleased to be working with Dr. Elin Haf Davies to develop her award-winning digital measuring device that could be used in forthcoming clinical studies, evaluating the safety and efficacy of potential new medicines for Duchenne muscular dystrophy (DMD).

As part of our commitment to support research at every stage of the clinical development pipeline, we are supporting the development of aparito, which uses a combination of a wearable device (for example a wristband that monitors number of steps taken in a day) with a mobile phone App, to monitor patient activity. aparito can monitor patients in real time and real life, outside of the hospital clinic. 

The wearable device monitors physical function and the disease-specific app captures information relevant to the impact that DMD has on the patients’ lives. The app can record specific events that are important for Duchenne patients, such as falls or near falls or missed school days.  

The app also records medication taken, prescribed or bought over the counter, and visits to health professionals. Patient reported outcomes (PROs) are also captured in the form of short questionnaires.  

Real-world data, reported by patients themselves, are more frequently being used as endpoints in clinical trials.  This type of data can help with understanding the impact of DMD, and any potential treatment, on patients’ lives. 

Duchenne UK is now planning a small feasibility study that will recruit around 10 DMD patients via two UK centres to help us develop aparito for DMD.  Monitoring patients for up to three months will provide sufficient data to identify the most appropriate events and PROs to include in aparito for DMD.  It will also provide data about the adherence and usability of the wearable device. The data generated from our study will be sufficient to produce a publication that will outline the findings, and make future recommendations about best practice use of mHealth and wearable devices in DMD.

aparito was one of three companies recently shortlisted under the ‘health’ category for the national digital agenda impact awards (www.digital-agenda.co.uk). These awards celebrate digital innovations that improve peoples’ lives and the world around us.