Spotlight on CF clinical trials: How you can get involved
Why are clinical trials important?
Clinical trials are a fundamental part of the process to develop and bring new medicines to people with CF. All new medicines have to be investigated in carefully designed clinical trials before they’re made available on the NHS for prescription. Clinical trials are research studies. They involve participants giving their consent to take part in a trial to potentially receive a new treatment for the duration of a trial. During a trial, researchers will carefully monitor the health of trial participants to make sure the new treatment is having the desired effect and is also safe. Without clinical trials, even everyday medicines like paracetamol or ibuprofen wouldn’t be available for us to use.
Why take part in research?
Taking part in research can be a rewarding experience for many people. In some instances, it offers people the chance to access potentially lifechanging new treatments many months or even years before they are available on the NHS. In recent years, clinical trials have given many people early access to CFTR modulators like Kaftrio. Of course, taking part in a trial doesn’t guarantee that you will receive a new medication, and as we know, not all new medications work for everyone. But there can be other benefits of taking part too. For some people, being a part of a research study is helpful to find out more about their health, since the extra clinic visits and additional investigations can provide a richer insight into what’s going on with your own health.
On a wider level, volunteering to take part in research is not only potentially helping yourself, but it can benefit others in the community too, by allowing new treatments to be thoroughly investigated for safety and effectiveness and helping to bring positive changes to routine clinical care.
What’s it like taking part in a clinical trial?
Candice, who lives with CF, shares her experiences of using the CF Trials Tracker and what taking part in a clinical trial was like. “When I first heard about the CF Trials Tracker from my CF clinical care team, I went onto Cystic Fibrosis Trust’s website to browse the Trials Tracker and found a few trials I was interested in. I had a look at these trials to see whether they were running at my centre, and one was. I got in touch with my consultant and spoke about the possibility of taking part in the trial. One of the clinical research nurse specialists then got in touch with me to discuss the trial in more detail and help me decide whether I wanted to take part.
“I felt like I was able to contribute something positive toward the advancement of treatment for people with cystic fibrosis. On a personal level, my health was closely monitored during this time, so I felt like my health improved and I had better insight.”
Tiffany, mum of 4-year-old Joseph, who has CF, tells us about her family’s experience of travelling 260km to take part in a clinical trial of ivacaftor, which she discovered through the Trust’s Clinical Trials newsletter.
“Our youngest son, Joseph, was diagnosed with CF from the heel prick test taken soon after birth. Neither of us were aware that we were carriers of the CF gene. I can still vividly remember when we were told that Joseph was pancreatic sufficient. It felt like a small victory.
“Soon after, we signed up to CF START, a trial that’s looking at the safest and most effective way to treat infants diagnosed with CF with antibiotics. We happened to find out about a clinical trial when we received the Trust’s Clinical Trials newsletter. The email featured an article about a trial for the CFTR modulator ivacaftor, which was recruiting children under 2 with Joseph’s genotype (G551D).
“We travelled 260km to take part in the trial at Alder Hey Children’s Hospital. We would have travelled to the other side of the world if we had to.
“We were very lucky as my husband had sufficient flexibility work-wise to enable us all to travel to all the appointments. Thomas, our eldest, was in nursery and was happy to go on a fortnightly adventure! The trial team did everything for us, and I mean everything - hotel, travel expenses, snacks, lunch at the hospital, it was all pre-arranged. We were even met at the hotel on arrival with anything we might have needed at the following morning’s appointment. The trial team’s coordinator was brilliant and really took away all of the stress.
“We were very excited to meet the clinical trials team at Alder Hey. We had spoken on the telephone a number of times prior to our first appointment and had already been given the chance to ask any questions we had. The team are all experts in their field and we felt very privileged to be receiving the very best treatment on offer for our beloved boy. We met some wonderful people during the trial, most of whom we’re still in contact with today.
“To have Joseph’s life-changing medication early, and to be a part of a trial to give a little back on behalf of all the families impacted by this condition, was an easy decision for us. The day we heard that Joseph was accepted on the trial was one of the best days of our lives.”
How is the Trust supporting clinical trials?
Through the CTAP programme, which we launched in 2017 with the support of the Cystic Fibrosis Foundation, we have brought together a network of 27 NHS CF centres who we have funded with specialist trial coordinators to oversee the running of CF clinical trials in the UK. Our national team of CF trial coordinators also act as a point of contact for the CF community to talk to about taking part in clinical trials, helping people with CF and their families find the right trial opportunity for them. Collectively, our network of CTAP centres have supported over 40 CF research studies and helped over 1,000 babies, children and adults take part in a clinical trial since CTAP launched.
As part of the CTAP programme, we’ve also created resources and online information about clinical trials, along with the CF Trials Tracker database. All of this information forms our CF Clinical Trials Digital Hub, a platform which aims to help children, adults and parents learn about, and take part in a variety of clinical trials across the UK. Our Hub is there to help ensure the UK CF community are at the forefront of new and transformative therapies being developed for CF.
Our CF Trials Tracker database is a unique tool which supports the CF community to keep track of CF trials opening in the UK, while also helping people find the right trial opportunity for themselves or a family member to take part in locally or further afield. Trials are searchable by age, location, and the type of medication being trialled. Each trial listed on the Trials Tracker includes a summary about the trial, along with information about who can take part and which CF centres are running the study. There is also a contact form for each trial to help put people in direct contact with clinical teams to request more information on taking part in a particular trial. This may be their local team or a team at a different CF centre who are running the trial.
Why is the Trials Tracker important?
The Trials Tracker empowers people with CF and their families to take the lead in finding their own trial opportunities. This means they don’t always have to be reliant on their CF team getting in touch. They’re also able to find and sign up for trials taking place across the country – so they’re not restricted to only taking part in trials at their local CF centre.
How can I find out more?
For more information on the Trials Accelerator and how you can get involved in clinical trials, visit our Clinical Trials Hub, or browse upcoming clinical trials on our CF Trials Tracker.
Together, we can all play a part in the race for effective treatments for everybody with cystic fibrosis.
Cystic fibrosis (CF) is a genetic condition which causes sticky mucus to build up in the lungs and digestive system. It affects more than 10,800 people in the UK. One in 25 of us carries the faulty gene that causes it, usually without knowing.
Since 1964, we've supported people with cystic fibrosis to live longer, healthier lives -and we won’t stop until everyone can live without limits imposed by CF.
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