Talking trials: ‘It’s about making the best decision for your family’

Hi Esther! Can you tell us more about why you’re interested in clinical trials? 

My son Joshua is eight and was diagnosed with CF at three and a half weeks old. We noticed a huge change in him when he started taking Orkambi. He had a lot more energy and grew fewer bugs. He moved onto Kaftrio about a year ago. We saw another big change, this time it was sustained weight gain, which had previously been a challenge. 

We feel very lucky. The burden of treatment, worry and stress has massively reduced since Kaftrio and our outlook now is that he will have a healthy life. Without clinical trials we wouldn’t have these life-changing advancements to treatments in CF seen over the past 20 years. Whether following the progress or taking part, it’s exciting to see how ideas can become medication in the hands of patients making real differences to CF lives.

What does Joshua think about trials? 

Although we haven’t been offered a clinical trial yet, Joshua has some understanding of trials, for example, he knows Kaftrio was ‘tried and tested'. He is inspired by science, he likes to know how drugs work in the body and how they help CF.

Joshua is someone who likes to help others. He would like to take part in the right trial, not just for his own benefit but for the greater good of everyone with CF. He likes the idea of participating as it helps everyone in the CF community. 

How do you find out about trials? 

Cystic Fibrosis Trust has a Trials Tracker which allows you to see what trials are taking place and whether they are suitable for you or your child. I’ve also signed up to various CF groups online where upcoming trials and trial results are discussed.

Find a clinical trial

What would you consider before taking part in a trial?

The main thing we would consider when being approached about a clinical trial would be whether he needed to come off Kaftrio to take part. I’d want to see strong data from earlier trials showing the drug worked better than Kaftrio. 

Other considerations would be what sort of procedures will be involved and time out of school. I know some trials now include home monitoring, which would be great as it would mean fewer hospital visits. We would be willing to travel to another centre that wasn’t our regular CF centre to take part in a trial if it was something that was important to us. I think reimbursement for the inconvenience of taking part in a trial is important, especially for things like travel costs.

Deciding to take part in a trial or not is about making the best decision for your family. Sometimes it comes down to timing and saying no isn’t the wrong thing if that is the right decision for your family.

Talking to your child about clinical trials 

Having conversations about clinical trials can help your child learn more about CF, feel okay about asking questions and think about what part they could play in future medical developments. Parents of children with CF and a CF psychologist have helped us put together advice on talking to your children about trials.

You can also use our Junior Agents Comic Book to help introduce your child to the idea of clinical trials. This comic book is aimed at primary-aged children and explains what clinical trials are, what taking part might involve and how getting involved could help children with CF like them. 

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