Alex Johnson: What trial is Jack taking part in?

I am often asked whether Jack is taking part in a clinical trial, and if so, which one.

Yesterday, Jack enrolled into his fifth clinical trial. This sounds exciting. But this is actually the fifth trial he is taking part in where there is no chance of him getting any direct benefit from taking part. There’s no potentially disease modifying drug involved that he could benefit from.

Instead, he’s taking part in a trial to help us try and understand more about Duchenne and the disease progression.

The next question people often ask me is, ‘why do I keep enrolling him into these studies, if they won’t benefit him or make him better’?

For me the answer is simple: the more that I learn about Duchenne, the more I realise how complex and challenging this disease is, and how there are so many areas within it that we still know so little about.

I realise this is a strange statement. People think that this is a well characterised disease – and parts of it are: But we still don’t know for instance, why some children with DMD are stronger than others and why some respond better to treatments than others.

The latest study we have enrolled in is at Alder Hey children’s hospital. It’s being led by Professor Francesco Muntoni and his team at Great Ormond Street Hospital and will involve collecting DNA samples from Jack as well as collecting clinical outcome data from the North Star database to understand how the genetic profile of people with DMD determines their level of muscular ability.

Jack’s also part way through another study to test a wearable device that monitors patients remotely. The project is called Aparito and you can see him wearing his trendy Aparito watch in the photo above. The project will hopefully allow for the delivery of meaningful, relevant, and real-time data between patients and clinicians in a way that actively supports and enhances diagnosis, treatment and drug development. The watch is paired with an app on my phone and records patient reported outcome-measures (PROs) and events.

This study is being funded by Duchenne UK. We hope that it will play a pivotal role in clinical trials by reducing the lengthy, costly and often challenging process. This, I believe, will be valuable.

Jack really enjoys taking part in these trials. There is very little burden on him or on us as a family. He loves the staff at the Clinical Research Facility and we appreciate the care he gets through taking part in the studies. He knows taking part in these trials won’t help make him better, but the data he helps generate might help create a better understanding of the disease and bring treatments faster to the Duchenne community.  

If you or your child has Duchenne and is asked to take part in a study like the ones outlined above, please do consider it. Sometimes it might not be the interventional drug study you want to take part in, but by taking part in studies like Jack has done we can collect information we need to help understand the disease and help develop better treatments for people with Duchenne Muscular Dystrophy.

Published on 11 January 2017

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