Clinical trials stories
Four adults with cystic fibrosis (CF) who took part in some of the CF clinical trials that led to improved treatments widely in use today, featured in our clinical trials booklet. Here you can find out more about their experiences.
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I have always been very interested in clinical trials, and have had the opportunity to participate in several trials. The first was trialling the hormone Grehlin. If I’m honest, this trial was pretty hard. I didn’t feel I had all the facts to hand before I took part. My second trial was much easier and I felt much more informed.
I also took part in a home monitoring study; I monitored my lung function, oxygen levels, weight and sputum and uploaded these by phone. I wore a fitness tracker to measure daily movement and I also recorded how I was feeling. This helped me become much more aware of my own health, which I found very empowering. It didn’t impact on my day and I found myself trying to beat my own results!
I’d encourage people to be pro-active and speak to their CF centres if they want to find out what trials are available to them. I think clinical trials give people the power to be in charge of their own health – a chance to help themselves and others – especially for people who cannot participate themselves. My experience of clinical trials has made me more in tune with my own health, and overall that is something I have found very engaging.
Over the years I have been involved with many studies, surveys and trials. I have undoubtedly benefited from advances in CF care brought about by clinical trials, and I feel it is the least I can do to give something back.
I have been lucky to have a CF consultant who was particularly focussed on CF research, and have always made it known to my CF team that I was keen to participate in clinical trials.
I was a case study for the TOBI podhaler in December 2011, a treatment which is in use today. In the mid-90s I took part in the Ciprofloxacin trial – then a new antibiotic, it is also now in everyday use over 20 years later.
More recently, I was fortunate enough to meet the criteria for the Kalydeco trial, having one df508 mutation and S497R. I am now on the full dose of the drug and my lung function is over 90%.
I’ve been lucky to have benefited from my own participation in trials and from wider developments in CF medicine. My further participation in trials, as well as fundraising for research, feels like I can continue to help future generations. I was incredibly proud to be invited as a keynote speaker to talk about my experiences at the Cystic Fibrosis Trust’s 50th Anniversary Gala, which poignantly coincided with my own 50th birthday.
I have taken part in a number of clinical trials, and have always made sure my CF team were aware of my interest in taking part. I am quite regimented about sticking to my treatment so I think my CF team views me as a good candidate!
I took part in a trial for DNase when I was a teenager. The DNase trial showed the treatment was effective, and I’m still benefitting from it today. Now I want to do all I can to help the younger generations living with cystic fibrosis.
My experiences of taking part in clinical trials have been largely positive, and I think there are a lot of advantages in taking part. Often, you will be seen by the best expert clinicians and researchers, and much closer attention will be paid to your overall health. It’s a chance to learn much more about your CF and become more expert in your condition. I also quite enjoyed the novelty of attending clinic out of choice rather than necessity!
Where I can’t participate, I can still get involved in clinical trials. Feedback from the CF community will help to set achievable expectations for fitting daily treatments around a trial study and the considerations of the impact on family life and other commitments.
It’s important you let your team know of your views on trials, and that you’d like to be kept in mind. About three years ago I was approached by the research nurse at my CF Centre about an ivacaftor (Kalydeco) trial. Following that, I took part in the AlgiPharma trial, also at my CF Centre, and am currently involved in the SmartCareCF trial. I’d definitely be up for taking part in more trials in future.
At a clinic appointment I attended when I was about 16, the day before a careers fair, I recall the doctor telling me not to worry about a career and just to enjoy life. I’m pleased to say that I’ve reached my forties and have been able to both enjoy life and have a fulfilling career, as well as the joy of starting a family. I hope that I’ll be around to see my son grow up and follow his dreams in life – he currently wants to be an artist (or a superhero!). With continuing advances in treatments, brought about through clinical trials, that reality is becoming more and more the norm.
When I was younger I was involved in various studies; Azithromycin study, I-neb nebuliser study, Creon 10,000 study and more recently the Gene Therapy Study as well as and various smaller clinical studies which involved providing lung function, sputum and blood samples.
I knew that safety studies had been conducted before a trial takes place, so I never felt there was too much to be concerned about. However, there were times when I hadn’t taken on board what would be involved; having done one trial, I assumed others would follow suit, but there are differences.
Even signing a consent form, you aren’t obliged to participate. Clinical trials won’t affect your care either.
Taking part in clinical trials really helped improve my understanding of my condition. For example with the gene therapy trial, I was a given a peak flow metre, which I had to do every week and record my results. Due to this closer monitoring, I was more aware of when I was having a dip in my health, and could let my team know. Trials also gave me access to newer treatments, which benefited my health. Another plus for me was the sense of feeling useful, and helping the wider community of people with cystic fibrosis.