Emma: "I would urge anyone to take part in a clinical trial"

My son Alex is now 12 years old and was diagnosed with Duchenne shortly before his 4th birthday.

Since diagnosis, I have been closely monitoring clinical trials, and when the PF-06252616 Domagrozumab phase 2 trial came up, we asked if Alex would be eligible to take part.  Luckily he was and he was the first boy in the UK and only the 6th boy in the world to be enrolled.  This meant that we had to attend quite a few appointments in the run-up to the trial start period and Alex got to find out lots of information about what would be required from him.  He couldn’t wait to start!

Alex took part in this trial to evaluate the safety and efficient of PF-06252616, a myostatin inhibitor for 2 years.  We had no idea if Alex was on the drug in the first year, but we were guaranteed to have the drug the following year.  Alex is still taking part in the extension trial and has been attending monthly visits to Newcastle Royal Infirmary since we started back in 2016.  We love visiting the team, headed up by Michela Guglieri, they make our visits feel very special.  Alex has been an absolute super star throughout, taking monthly drug infusions without any trouble whatsoever.  He’s also had lots of MRI scans, heart scans and tests to check everything is okay.  Pfizer covers the costs of our travel and accommodation which helps us financially.

What we love most about taking part in this clinical trial, is that Alex is seen by specialists in the field of Duchenne every single month – it gives us so much reassurance knowing that his care is looked after so closely.  We would never had received this level of care without taking part in the trial. I always have someone to ask questions about any changes in his ability.  We’ve met some great friends too and we love Newcastle – it’s like our second home.  We will continue to stay on the extension trial until we are told that he can no longer be on it.

It gives Alex so much hope for his future – he believes the drug is working, which is fine by me. He does know that there are no guarantees, but as long as he feels positive about things I can only see the trial benefiting him.

I would urge anyone to take part in a clinical trial, if anything it will help us all better understand Duchenne and ultimately help bring about new treatments for all children.  I would say not to let concerns worry you, just speak with the trial co-ordinator and take things from there. Without clinical trials, we cannot expect new drugs to become available, we must take part to help this generation – and certainly the next.

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