A novel device called ActiMyo® has been proposed for its use as an outcome measure in ambulant and non-ambulant Duchenne trials. ActiMyo offers the opportunity to conceptualise shorter studies with much less patients compared with the currently available outcome measures. It is a portable device capable of providing information registered through an accelerometer, a magnetometer and gyroscope. A further validation of ActiMyo in a large sample including both patients and healthy volunteers will provide essential information of how this tool would work if used as primary outcome in pivotal studies.

The purpose of this project is to generate normalized standard scales for control subjects based on the data gathered from 80 healthy children (6-17 years old).

Following 12 months of enrolment, subjects will carry the device during 4 weeks twice within a 12 months interval. A total of 12 ActiMyo® devices will be allocated to this study, which will allow enrolling the expected number of subjects over a 12 months period.

The project will be conducted in Liège neuromuscular center, in Belgium. Belgian regulation allows recruitment of healthy subjects in school. The recruitment strategy is to propose two schools take part in the project as part of their pedagogic project. We will propose at first a lecture and debate in each class about research and Duchenne muscular dystrophy, and ask the children and parents to take part in the study. This study will, of course, be validated by the Ethics committee. We believe that this approach will be highly efficient for recruiting participants from different age groups, and in addition help to generate conscience about the importance and the need of research in Duchenne.

Dr Laurent Servais, Chief Investigator in this project and Key Opinion Leader in Duchenne said: “In this context, identifying robust, objective, precise, reliable and reproducible follow-up tools, that objectively measure patient´s motor capacity, and thus serve to analyse and compare the response to emerging treatments, is needed. Then, the clinical development plan in DMD will be shorted, cheaper, and the risk of false-negative trials will decrease.  The whole Actimyo team is very excited to work with the support of action Duchenne on this project.”

The Director of Research at Action Duchenne added, “We are delighted to fund this pivotal work in validating a potential outcome measure for Duchenne trials, which may be applicable to all ages of the Duchenne spectrum. This is in sum reflects the ethos of Action Duchenne in meeting the needs of the whole community and also collaborating internationally to drive translational Duchenne research and clinical trials forward.”