Being the parent of a child on a Clinical Trial

Put at its most blunt, Duchenne is a terminal, progressive muscle-wasting condition. Every person living with it follows the same journey, some follow it faster or slower than others, but it always ends in the same place.

On the surface of it, clinical trials for Duchenne seem like hope, a lifeline, given the seriousness of the condition it feels like you have no choice but to take part.

When we enrolled Jack on the clinical trial, we hoped that it would give him access to a medicine that might slow the relentless progression of Duchenne. While we had hope, we also had very real fears about him trying an untested medicine. Little was known about how that medicine would work in children living with Duchenne.

Clinical trial drugs can and do have side effects. What if he was to suffer from side effects or the drug made his condition worse? In the past few years, we have seen two brave Duchenne boys lose their lives while on trials. These have been heart-breaking losses for our community and my heart goes out to those families who bravely put their children forward.

We want a treatment to preserve Jack’s quality of life, but we don’t want to hurt him in the process.

That’s why the past 18 months have been tough at times. We watch Jack take a medicine everyday and wonder what it’s doing to him on the inside. Our lives are filled with hope but also questions and uncertainty.

Jack has been incredible while on the trial. He takes it all in his stride with a big smile on his face and willingly complies with every test - there have literally been hundreds. His dad can’t even get into an MRI scanner, but I have lost count of how many times Jack has been in one and has never complained once. And, he does all this knowing the medicine might not work. I feel like I say it all the time, but he really is my hero. He really does want to find a treatment to stop the suffering Duchenne causes him, his loved ones and his friends living with Duchenne.

We are lucky to have a great clinical trial team caring for Jack at Alder Hey. They really do go above and beyond to make sure he’s happy, well cared for and monitored continuously. Jack considers them to be his best mates, which always amazes me as, every time he sees them, they must carry out bloods and a host of other tests on him.

We have not been able to share very much about Jack’s clinical trial journey over the past 18 months due to the confidentiality of the first stage of the trial but, over the next few months, he wants to share with you some of his experiences.

We are grateful to everyone who has helped us to fund the DMD Hub. It has given hundreds of children like Jack the chance to take part in clinical trials across the UK. Clinical trials give families hope but we also realise how hard it can be for them. It is a rollercoaster ride of emotion, but we realise that Jack is one of the lucky ones who’s got the chance to get on the ride. There are still too many who haven’t.

We set up the DMD Hub to ensure all patients with Duchenne, both children and adults, have access to clinical research trials.

The DMD Hub is a network of trial sites with trained staff who are funded to carry out clinical trials for Duchenne.

• Joining Jack has contributed £378,054.46 towards the £3 million Duchenne UK has committed to the DMD Hub to date.
• This has created 34 Duchenne dedicated posts of doctors, nurses, physiotherapists and clinical trial co-ordinators across the UK.
• There have been 437 more boys on clinical trials since its launch in 2015.
• There are now 11 DMD Hub clinical trial sites across the UK.
• And 20 clinical trials are now ongoing in UK with eight new trials opening in 2022 and nine trials in scoping.

You can read more about our work with the DMD Hub, along with our plans to make sure everyone living with Duchenne has a chance to take part in a clinical trial, HERE