The decision to join a clinical trial is an individual and personal one. Benefits and risks of participating, as well as the time commitment required, must be considered. For the Donaldson family, this decision has been a positive one.
You may remember the Donaldson family and their two daughters, Isobel and Ruby, who have cystic fibrosis (CF) and who created their own fundraising campaign during Easter of 2016. Entitled ‘Crack A Cure’, this community fundraiser stretched across the nation, not only raising considerable funds for future research and service provision, but creating awareness with each Facebook post.
It is with this drive and enthusiasm that the family tackle life with CF. Recently we had the great pleasure of interviewing Isobel, almost 12, Ruby, 9 and mum, Christine, to gain some insight into their experience participating in a clinical trial.
Please explain your main reasons for participating in a clinical trial.
For Christine and her husband, Gerard, participating in a clinical trial had always been a big part of their journey. With both girls being involved in the AREST CF (Australian Respiratory Early Surveillance Team for Cystic Fibrosis) program from diagnosis, the benefits of being on a trial rang loud and clear. Not only did it provide invaluable information in regards to the girls’ health status and management, but it was contributing to future research and treatments. From this time, Christine and Gerard made it known to the CF team that they would be happy for the girls to be considered for future trials.
Isobel and Ruby have been involved in a few trials over the years. Christine stated that she is comforted in knowing that by the time clinical trials come to Australia, they have already had outcomes in other centres across the world and safety standards have been clearly established and tested. Another important factor considered is that the clinical trial is not too invasive.
What clinical trial are the girls currently on and how did you go about joining this trial?
Isobel and Ruby are part of a study involving the drug Orkambi® for people with CF homozygous for the F508del-CFTR mutation. As mentioned earlier, we made it quite clear to the girls’ CF team that we were interested in being involved in trials. We also made the message clear to the staff at the Telethon Kids Institute (TKI) as not only would it assist with research, but hopefully, the girls would access new treatments before they were readily available.
Please explain the selection process
Our CF team and members of the team at TKI identified the girls as a possibility to be selected for the Orkambi® drug trial as they appeared to fit the strict selection criteria. With this, and being aware of our interest to participate in clinical trials, they gave us a call. From here, the girls were brought in to the Children’s Clinical Research Facility (CCRF) at Princess Margaret Hospital (PMH) for further assessment to clearly identify if they qualified to be included.
The selection process involved many tests such as blood tests, sweat tests and lung function. Ruby and Isobel were successful in qualifying and were part of the first group of children in WA to participate.
How long is the trial?
The trial is for a period of 2 years. The first six months was a ‘blind trial’ which means we were not told if the girls were on the Orkambi® or a placebo.
What did the trial involve?
At first, fortnightly blood tests were required. Now the girls have 6-8 weekly assessments which involve blood tests, spirometry and ECGs (electrocardiography). Sweat tests are required every 4-5 months. Interestingly though, Isobel doesn’t produce enough sweat, so is now exempt from this test.
Eye testing is also a requirement. An eye test is performed prior to commencement of the trial and twice during the course of the trial.
How has this impacted your lives?
The more frequent testing at the beginning of the trial obviously required more frequent trips to the hospital and to TKI, but has always been manageable. The assessments take about an hour. With appointments now being 6-8 weekly, it is much easier these days.
Honestly, the impact has been a positive one. When considering the time commitment, travel time, time off school and expenses involved; with both girls experiencing an improvement in health, this has not been an issue. Isobel, who would normally be hospitalised frequently, has only had one admission in 2 years and Ruby has kept well too. So, less stress and manageable appointments.
Please discuss the benefits of the trial, personally and in regards to future advances (e.g. did you feel any different? were there any improvements in your health?)
Ruby: I don’t really feel any different. I can now cough up mucus when I do my physio though. Before the trial I always had a dry cough and couldn’t cough anything up. My appetite has improved too. My last admission was about 5 years ago.
Isobel: I am not in hospital much now. I have an appetite and have gained 20% of my body weight. I now weigh 36kg and am growing well. Before the trial I always struggled to gain weight and I was frequently unwell. I can now really appreciate the feeling of wellbeing.
Christine: Being part of this trial has given us hope and optimism; it is a game changer. To watch the girls with their increased appetite and energy levels, no longer looking at the necessity for a port or a PEG, it’s just amazing. One of the parents at netball made a comment about how much energy Isobel had running around the court!
Another benefit is being able to address Isobel’s needle phobia. Isobel has worked closely with the Clinical Psychologist from PMH to assist with the blood tests required on the trial, and this has helped immensely. Needling is no longer such a fear for Isobel.
Being on the trial means we are in regular contact with the researchers and clinicians. For example, if there is a change in one of the girl’s cough we are required to inform them. This provides the opportunity for different medications to be prescribed straight away if required.
A big positive of the trial is the impact it has had on our family. With the girl’s improved health, we are all less stressed. Our sons, Jackson and Angus are no longer having to manage while we juggle lengthy hospital stays and all that came with the girls being so unwell, with family life. It is just so much easier on everyone now. We are all enjoying the ‘feeling of wellbeing’.
Would you participate in other trials in the future?
Definitely, yes. We would be happy to be part of trials in the future. We are hoping that Orkambi® will hold us until new drugs come through. The benefits of all the research is going to be amazing.
Do you have anything that you would like to share about your experience on the trial?
Ruby: Having CF gets easier as you get older and I am happy to have had a chance to be on a trial. Just remember to take your tablets!
Good luck!!!
Isobel: Being on a trial helps. Do it!!!
Christine: Clinical trials are so beneficial. I know some parents have found that if there are no obvious improvements they question the benefits. For us, we could see the positives straight away in Isobel, with Ruby it wasn’t so apparent. We just understand the drugs are doing good things.
We are hoping that Vertex’s submission for reimbursement for Orkambi® will be approved by the Pharmaceutical Benefits Advisory Committee (PBAC). If not, let’s hope the girls can stay on Orkambi® on compassionate grounds.
Reference:
http://www.cflivesmatters.org.au/Clinical-Trials/Clinical-Trials-101
Article from RED Magazine, Edition 4 2017.