Parents have written about their experiences with participating in clinical trials.
We would like to thank the parents who have contributed.
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 world to be enrolled. This meant that we had to attend quite a few appointments in the run up 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 absolutely 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.
In my experience, it can be tempting to jump at the chance of getting your child onto a drug trial, though there are definitely things to consider-
Are you happy with all the extra hospital visits on top of your child’s routine appointments? Will it impact on any siblings? Can you afford time off work, childcare for other children, treats for being brave etc?
Can you be strong when your child is upset or scared about blood tests or other procedures? Would you be prepared to stop any supplements you give your child, even if they ended up taking a placebo?
We decided we were happy enrolling our son onto a trial and are definitely glad we did.
Of course, a massive plus of taking part in a trial is the chance of accessing a new potential treatment before it’s available. Another advantage is the extra checks our son has, meaning more opportunities to pick up any potential problems.
For us as parents, we feel that we’re ‘doing our bit’ to help defeat Duchenne. If it doesn’t directly help our son it could ultimately help future generations, for us that’s what makes taking part in a clinical trial worthwhile.
I am often asked whether Jack is taking part in a clinical trial and, if so, which one.
Yesterday, Jack enrolled in his fifth clinical trial. This sounds exciting. But for all five of these trials, there is no chance of him getting any direct benefit from taking part. There’s no potentially disease modifying drug involved that could improve his health.
Instead, he’s taking part in a trial to help us try to 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 little about.
I realise this is a strange statement. People think that this is a well understood 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. It 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 of collecting data. 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 generates 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.
Connor, who’s 8 years old has participated in two clinical trials now.
The first was a 1b trial. It was quite hard going with lots of intrusive tests and overnight stays for two weeks.
The second was a phase 2 trial, so not quite so intense. We had quite a few visits and a few overnight stays in the year he participated.
Connor had two muscle biopsies which were a little larger than we expected. He was out of hospital the same day. The pain wasn’t an issue afterwards for him and they both healed nicely with no trouble.
He has been an absolute trooper in taking part, hardly ever moaned and took everything in his stride.
We had a few difficulties with giving blood but his advice would be no cream or spray, choose the bounciest vein, drink lots of water beforehand and keep the area warm.
He is now on the extension phase of the trial so there are fewer visits now. We go to Great Ormond Street Hospital every six months for the trial and have a home nurse visit occasionally.
All our expenses have been paid for including hotel stays, meals, drinks and tickets, which is excellent and definitely helps financially.
We’ve made some amazing friends in the nurses, doctors, parents and patients with Duchenne. Connor used to look forward to the popcorn and movie nights with his new friends in the clinical research facility.
We are extremely proud of Connor and the way he has handled it all for the past few years. He also likes the fact he’s helping the scientists to find a new medicine to help all the kids out there with poorly muscles. He’s our hero!
Without families and the children with Duchenne participating in the clinical trials, we wouldn’t be as close as we are now in finding a possible treatment. So our advice would be get enrolling, there’s not a lot to lose and lots to gain……let’s find the drug that works!