Robert - Interview 26

Brief Outline: Robert has always taken part in clinical trials where possible. These have ranged from simple one-off collection of breath samples to having a dose of gene therapy, having a bronchoscopy either side of the dosing and the visits to the hospital.

Background: Robert, aged 22, was diagnosed with cystic fibrosis soon after birth. He recently graduated from the University of Oxford in Mathematics and Statistics and would like a career in Medical Statistics. He starts work as a Maths teacher very soon.

More about me...

Robert, aged 22, was diagnosed with cystic fibrosis soon after birth. He recently graduated from the University of Oxford in Mathematics and Statistics and would like to pursue a career in Medical Statistics for clinical trials. He is currently preparing to start work as a Maths teacher. Since early childhood, Robert has taken part in many clinical trials ranging from simple one-off collection of breath samples to having a dose of gene therapy, having a bronchoscopy either side of the dosing and visits to the hospital. He is currently involved in a Phase 1/Phase 2 trial for gene therapy, and expects to progress to the Phase III multi-dose double-blind trial when it starts next year; 2011.

Robert talks about all his varied experiences including those that have been quite intensive. Despite recent health concerns that may be the result of taking part in some of the trials he is very keen to continue. Robert understands the importance of clinical trials and research to help improve treatment and care for future generations. He acknowledges the personal benefits of taking part and also the social and medical gains. He feels proud to be part of helping medicine move forward to help others with cystic fibrosis.

In improving his experience Robert says, “It might be nice to know that our contribution has been worthwhile.... it’s always satisfying to know that it has worked.” Robert would also like a website where young people who have taken part in clinical trials can communicate.

 

Robert describes the various phases of clinical trials.

So a Phase 1 trial is where the, is the riskiest one but that’s tested on people without the, normally tested on people without the condition. So that would just be to see if the drug is safe to deliver, and that’s done by any volunteer that wants to do it. The Phase 2 is then as similar to Phase 1 but they already know it’s probably safe to administer. And that’s done on a small group of people with the condition that they’re looking at and that’s to see if there are any side effects with the dosing or what’s the best method to do the dosing, how effective does it seem to be. And then once, and then Phase 3 is a much wider scale trial which is where, usually double blind and that’s where it’s to really see if across a large group of people with the condition whether they can see a general clinical benefit or not. And then Phase 4 is sort of ongoing, ongoing monitoring, of the drug once it’s been approved from the stage 3 just to make sure there’s no long term side effect that can’t be measured, you know, if there’s, if over 10 years something happens you can’t measure that in the earlier trials. 

Robert understands the importance of clinical trials and research to improve the treatment and care of future generations diagnosed with cystic fibrosis.

I’ve always taken part in clinical trials for as long as I can remember as a child I didn’t necessarily understand why I had to get on an exercise bike and go on it for 30 minutes while something was attached to me or why they wanted a breath sample. But as I grew older I began to realise the importance of medical research and clinical trials to directly benefit me. And when developing that point of view I saw how important it was that I had taken part in these trials and also it gave me the motivation to want to carry on in talking part in them. particularly when I think that the difference in the quality of life for people with cystic fibrosis now as opposed to when I was born is vastly different and so much better and solely due to medical research. And so I feel that because I’d benefitted from it then in a way I owe something back, you know, to future generations to help in whatever way I can.

There have been a few times where I’ve, it’s not been easy. As a child I was very needle phobic so and there was at least one occasion where I was asked to take part in a clinical trial and I didn’t because it would have meant having more blood tests and that was something I couldn’t really cope with back then. But now I’m older and I’ve learnt to deal with that then that’s not been a problem anymore.
 

Sometimes a clinical trial may show that a drug or other treatment has little benefit but this can be valuable information says Robert.

Well I would always remember that you know, things would be explained to me but I perhaps then just thought oh this is something else I’ve got to do for the hospital check up and maybe didn’t really understand what I was doing was a voluntary thing contributing to medical science. But you know, I, I trust my mother that she always made sure I was doing something that well hopefully that she would think that if I could have made that choice myself I would have wanted to do and as such when I’ve got older I have chosen to make those decisions. And yes from what I remember the ones I did when I was younger usually weren’t very involved as I say just be collecting sweat samples while I cycled on a bike or taking a sample of my breath to freeze it. Whereas when I’ve got older much had more involved ones like this gene therapy dose and I’ve also done, I did a clinical trial they had to stick a, a probe up my nose and then measured the measured the potential difference in the cells in my nose and so you had to sort of sit there with this tube in there while this liquid was dripping out of it. And in fact on that one I remember the, the doctor then said to me “I know you might not want to do this but could you come back and do it again in a couple of hour’s time?” And I wasn’t doing anything I had a travel card in London and so I went “Yes okay I’ll just go off for a bit and come back” so. So I like to think that I do give as much as I can.

 

And these are all clinical trials to do with cystic fibrosis and helping other people?

 

Yes and I’ve and, you know, sometimes I have seen a direct benefit from it, almost. I took part in one study where it was assessing whether a particular steroid flixotide, whether, it was assessing, it was known to have a good beneficial effect in people with asthma but it wasn’t really known if it was for people with Cystic Fibrosis. And as it happened in that trial I was on the placebo and there was no change so as a result of taking part in that trial I was able to come off a steroid that I was on effectively needlessly. So I have seen direct benefits in that way but then also sometimes it’s longer term like the one I’ve just told you about with the dripping through the nose because that’s run by one of the doctors at the hospital and I’ve seen him subsequently and he said, “Oh yes we actually found some good results and it’s been written up in a paper and it should end up in a journal”. So it’s nice to know that what I’ve taken part in has worked and it is making progress.

 

To take part in a Phase 2 trial that is at the forefront of medical science is really exciting and Robert is keen to continue.

Yes well the different trials are, have different purposes, so all the ones, so the one I did where I ended up on a placebo that was the double blind one to see, that’s were “Oh we know this drug is safe we just want to see if it has benefit”. Whereas the pilot study was specifically to see is the delivery of this drug safe, are there side effects how efficient does it seem to be and that. So that was on a smaller group of people and so from that point of view was reasonably, it was quite ground breaking actually and it was, it was exciting to take part in something that was virtually the forefront of medical science. Because it was using a different delivery for the gene therapy rather than a viral vector which most gene therapy uses it, it was it was using a different delivery mechanism which might mean it’s viable for multiple doses. 

In Robert's view when you are approached to take part in a trial by a doctor or nurse that you know, it can make the decision to take part (or not) easier.

Yes well with, so with the trials where it’s been with doctors I already know then obviously that’s been much easier because I know that they’ve looked after me before and that everything’s been fine. So I know I can trust them. But yes I guess with the other ones you just have to, I think it depends on how involved the trial is. So like this one that took my breath was a man I’d never met before and was, you know, all I know is my breath has been taken away and frozen somewhere [laughter] so, but then I have to think well it’s just a sample of my breath. Whereas, you know, if that man had wanted to approach me for the gene therapy one I might had, because that’s had much greater involvement I might have thought more about it whereas when you’re approached for an involved trial by someone you know and that yes I think that makes it easier. 

Robert urges others to see past any side effects or discomfort involved in trials and take part because the benefits to science are so great. He was also pleased to get access to a new gene therapy.

I think a barrier might be, so as I said when I was younger we didn’t do anything where I had to have another blood test. So if there’s a particular procedure involved that you don’t really want to do then you’re very free to say no. But even if it’s something that only, you know, that only mildly inconveniences you, like I had to have these bronchoscopies, you know, it’s not exactly a fun thing to have but you think well it’s worth it to put myself through that for the benefit that the trial gains from it. Or I mean there was another, with the pilot study they had this worst test ever, they had like a wire pipe cleaner brush that they’d stick in your nose and go whoosh and take it out. And it’s really, really uncomfortable and oh it was horrible but in doing so they got a million epithelial cells from each nostril doing it so, and the doctor doing it said “I know these are horrible we’ve done them on, we’ve done them on ourselves as well” but the amount of data we get from it is incredible. So I think perhaps sometimes maybe for young people to try and see past the, you know, slight uncomfort they may have to go through for the greater gain that it brings yes. And I think as, as a facilitator as I said being a young person on the gene therapy trial if I’ve got a chance of being one of the first people to have gene therapy and then to be on that drug then that’s a, a really great opportunity for my health. So even though that’s a yes it’s not a financial incentive it’s still a really good incentive to take part. 

Robert was pleased to have the opportunity to try a new gene therapy in a Phase 1 trial. It was also scientifically exciting, and a chance to give something back.

Well I’ll answer the first with respect of the leg rash that was actually, interestingly I had a very last appointment with them that day and then the rash turned up in the evening. So technically I had finished the trial by that point when the side effects happened. But with the losing my voice, I did wonder do I want to put myself through another bronchoscopy. And then I think I possibly took the approach of well it can’t do any more damage than it’s already done so I may as well carry on and I’ve got this far. And there was also because on the pilot study it wasn’t double blind because that was a sort of Phase 1/ Phase2 trial. So it was on a small cohort but we all knew we were definitely having the gene therapy. And so to have the opportunity to have a dose of gene therapy was, you know, even if it might not work, its full effect is still well it’s exciting scientifically but also a great opportunity medically for myself to get that.

 

So is that a personal benefit for you?

 

Yes I think so and also if so if on the multi dose trial, you know, if everything goes well and then it all progresses and your through to the end then by being someone that’s taken part in the trial you will then have preference to be on the drug once it’s finally finished and released. And so that, that’s always the benefit you can hope for as well.

 

Is that an incentive or motivation to take part, personal?

 

I think it is partly yes. Yes as I say as far as this gene therapy trial I think it’s partly being what I said earlier about wanting to give back and contribute for future generations but then also partly the chance to end up being on gene therapy from the very beginning, you know. As soon as I can and yes.

 

Robert was not eligible to take part in one study because at the time he was taking part in another study. He doesn't really see why it would have mattered.

Well luckily in this case I think I was doing the one that I would rather have done. But I guess there could be situations where you are doing a trial and then another one comes up that you’re really interested in and you want to do that one but you can’t and then you think, “Oh shall I pull out of the one I’m doing just so I can do this other one” and then. But yes the only time I can remember being told I was ineligible was because I was on another trial and that, in fact there was a very weird situation because the trial I was on wasn’t giving any drugs, this was the run-in study they were just taking observations and measurements. And this, and then the other trial I was approached for was just collecting breath samples. So neither trial would have been administering any kind of drug. They both would have been just taking observations. But it was still, you weren’t allowed to do both, which I didn’t quite understand, but I did what I was told. 

Having a computer to randomly allocate you to a group makes the trial fair. Robert was allocated to receive the 'placebo' and as a result of taking part he was able to stop taking a steroid, which he was quite pleased about.

And these are all clinical trials to do with cystic fibrosis and helping other people?

 

Yes and I’ve and, you know, sometimes I have seen a direct benefit from it, almost. I took part in one study where it was assessing whether a particular steroid flixotide, whether, it was assessing, it was known to have a good beneficial effect in people with asthma but it wasn’t really known if it was for people with Cystic Fibrosis. And as it happened in that trial I was on the placebo and there was no change so as a result of taking part in that trial I was able to come off a steroid that I was on effectively needlessly. So I have seen direct benefits in that way but then also sometimes it’s longer term like the one I’ve just told you about with the dripping through the nose because that’s run by one of the doctors at the hospital and I’ve seen him subsequently and he said “Oh yes we actually found some good results and it’s been written up in a paper and it should end up in a journal”. So it’s nice to know that what I’ve taken part in has worked and it is making progress.

 

Okay and what do you understand by the terms like you mentioned double blind and placebo, what do you understand?

 

So placebo is where, well in a, in a double blind trial which is where no one not even the doctor running the trial knows which person is actually taking the drug and which person is just taking an inert substance and, which is the placebo. Because it’s not actually the drug but everyone thinks that they might be taking the drug and so that means that you could see, you’ve got some people actually on the drug and some people not actually on the drug and you can make a direct comparison. And because no one knows who is on which then, then that makes it a fair trial.

 

And did you know did you have any idea when you were going through what you were on?

 

No I didn’t, no. I hoped I was on the placebo because then I might get to come off the steroids [laughter]. And as I say not even the doctor running the trial knew. I think they’d got a computer to randomly generate it and then you know, it tells them how this person has to have inhaler number 234 and then you only find out afterwards whether you are on the placebo or not but at the time no one knows.

 

Although Robert understood the need to be monitored and having extra tests, there were times when it was quite intensive; he often had to stay in overnight for observation.

So the run in study for the multi dose trial is just one appointment every four to six months I think so that’s just occasionally going in and they do this whole load of tests. And the idea is that they’ve done it over 18 months so they can then plot your progress but that’s just once every four to six months. And in fact on more than one occasion with those I’d managed to coincide them with my regular outpatient visits as well. Because they do a lot of similar tests so it just meant a longer day in the hospital when I got everything done. But the pilot study with the single dose of gene therapy that was quite involved so over the space of a month I think I went in six or seven times. And the bronchoscopy, there were two, two bronchoscopies for which you have a general anaesthetic and I had to take an overnight bag, to be prepared to stay overnight if I needed to. And likewise the day when I actually had the dose of gene therapy I had to be prepared to possibly stay overnight, so those three were all full days in the hospital. Whereas the other visits were, were just for taking results and measurements from you. But, and it was, they weren’t evenly spaced either as well. So for instance, so I had the day of the dosing and then the next appointment was two days later to see what the very short term effect is and then there’s one, you know, three days after that and then they got further and further apart but it meant there was, you know, one week that was very intensive with time in hospital. 

Payment for healthy people to take part might be appropriate, but helping others was incentive enough for Robert. However, now that he is starting work he may have to consider payment for time off work in the future.

Have you been had any payment or you’ve had travelling expenses?

 

Yes the, they’ve all paid travel expenses and so when I had to go down to a hospital for this overnight thing we got travel and then you know you where allowed to spend this much money on dinner and they would reimburse you for that. So, so yes they, because I know with, with other clinical trials where you’re a healthy person volunteering you do it for money but for the Cystic Fibrosis ones as I said I’m doing it to help people like me and hopefully directly myself in some cases so that’s incentive enough for me.

 

But where there any payments at all, did they offer payment, you know, sometimes they offer payment at the end or something?

 

No I don’t think I ever had.

 

Do you think that might be important for young people to have that? Or does it interfere with that kind of... bit of a dilemma?

 

Well at the moment I, it hasn’t been an issue for me but say now entering the world of work, because it’s not, it’s not only that you’ve got to travel there or you’ve got to stay somewhere. But now it’s, well I would have to, you know, it’s the time commitment which as a student or young person, you’ve got more time you can commit that’s free. But now I think it might make a difference if I did have to miss work to do it whether there was any incentive to do that.

 

Robert wasn't prepared for some of the side effects he experienced, and is unsure if one was a result of the gene therapy or something else.

Because the bronchoscopies were they’re a relatively routine procedure as it were. I didn’t really think oh yes there’s a risk I might lose my voice from this because I thought "Oh bronchoscopies people have those all the time don’t they". And so then it was a surprise when afterwards I just couldn’t talk and then, you know, it went on and on and then eventually my voice came back as I say my singing hasn’t really been the same since. And that has been a shame but well the best I can do is to try and get my, you know, try and practice more singing and get it back. But yes that wasn’t something I was prepared for. Whereas with the, with the rash on my legs and the meningitis scare that was something which it was about a month after I’d had the dose which is why it’s so unlikely that it was to do with that. But that was one I was perhaps more prepared for, you know, when I was on the way to the hospital the first thing I did was to ring the trial co-ordinator to let them know just in case it was to do with that. And so that was something perhaps I was more prepared for that there might be some unexplained effect but , you know the losing my voice I wasn’t really prepared for.

 

But did they sort of inform you, I mean in all of these did you; you must have had some information about them?

 

Yes.

 

And was those sort of side effects explained in there that the side effects....?

 

Yes with, I mean the leg rash thing was such a rare thing I think that sort of was probably encompassed on the consent form under there might be some rare unexplained things that happen but it’s not very likely.

 

Like with the voice?

 

With the voice I don’t really remember, I think they said that it was likely you would be coughing a lot after you come round from the general anaesthetic and that you would get a very sore throat from it. But to lose a voice for, it was about a month was actually very unusual and so I did tell them as it was happening, oh well it’s not come back and they, and they did show concern and try to suggest things to help but yes it was quite extreme.

 

Yes, yes. I mean how did you feel at the time was it a concern?

 

Well sort of for the first week or two I just thought oh yes it will pass but then by, you know, sort of the third week when it’s not getting any better, you think “Oh is my voice ever going to come back, what’s happened”. And then it did steadily come back. And in fact one of these weeks I was on holiday with some friends in Belgium and it did make it a bit harder on that holiday when, you know, you have to really strain to make yourself heard. And so I guess it, yes it wasn’t so fun on the holiday not being able to talk so much with my friends but yes but otherwise it was just a thing that I had to just sit tight and wait for it to pass.

 

But you were still keen to continue taking part?

 

Yes yes I think I am. Because in fact that, the losing the voice was only after the first bronchoscopy, the second one wasn’t so bad so it might just be that that first time something happened that.

 

Robert had considered withdrawing because the intervention was unpleasant, but knowing it was going to help him and others with cystic fibrosis gave him the motivation to continue.

Well I’ll answer the first with respect of the leg rash that was actually, interestingly I had a very last appointment with them that day and then the rash turned up in the evening. So technically I had finished the trial by that point when the side effects happened. But with the losing my voice, I did wonder do I want to put myself through another bronchospcopy. And then I think I possibly took the approach of well it can’t do any more damage than it’s already done so I may as well carry on and I’ve got this far. And there was also because on the pilot study it wasn’t double blind because that was a sort of Phase 1/ Phase2 trial. So it was on a small cohort but we all knew we were definitely having the gene therapy. And so to have the opportunity to have a dose of gene therapy was, you know, even if it might not work, its full effect is still well it’s exciting scientifically but also a great opportunity medically for myself to get that.

 

So is that a personal benefit for you?

 

Yes I think so and also if so if on the multi dose trial, you know, if everything goes well and then it all progresses and your through to the end then by being someone that’s taken part in the trial you will then have preference to be on the drug once it’s finally finished and released. And so that, that’s always the benefit you can hope for as well.

 

Is that an incentive or motivation to take part, personal?

 

I think it is partly yes. Yes as I say as far as this gene therapy trial I think it’s partly being what I said earlier about wanting to give back and contribute for future generations but then also partly the chance to end up being on gene therapy from the very beginning, you know. As soon as I can and yes.

 

Although researchers do try to keep you informed, it is a good idea to take an interest in what they are doing and get involved; having studied science and maths Robert often asked questions.

Did you have any results from all of these studies that you have?

 

I think I could have had, I think I could have had them if I asked for them I think with most of them sort of they wait until they’ve finished with it and it’s written up in a paper. But they always, they always try and make sure you’re as informed as possible and that you can, not exactly get involved but you can learn as much about what you’re doing as possible. And as I said because I did science A Levels and a maths degree I do take an interest in what they’re doing.

 

So you’re more likely to ask?

 

Yes, yes I mean I, for the, for the run in study I did up to this multi dose one, I had to go down to [area] overnight for a, some mucosal clearance scan and it was really cool. They got me to breath in radioactive isotope, where it, it was quite safe. It had a short half life but they then sort of took three, they sort of took an x-ray like that then basically take an x-ray like that and then, and then doing all of them all the way round they then built a 3D model of my lungs on the screen based on where these radioactive isotopes where, all the different angles and I mean it was just incredible to see it on the screen. And I mentioned I was doing a maths degree so they got the guy who designed the programme to come and have a chat with me and I got to learn about how the program was taking the data from the x-rays to build this model of my lungs.

 

Knowing he has taken part in trials that help others with cystic fibrosis is satisfying for Robert. He knows it takes a long time to publish results, but it would be nice to get some feedback closer to the end of trials.

Yes, well I guess one thing I might have liked, I don’t know how practical this is, is that so I said with the nose dripping one, I then saw the doctor and he said “Oh yes we got good results”. But that was so long after actually doing the trial I’d sort of almost forgotten about it. So I mean whether you might have to sign a confidentiality thing I don’t know but whether you could just be told thank you for taking part in the trial it seems we’ve actually found something. Because, because the publications process in academia is a long and drawn out that by the time we get to see anything that’s happened it’s always been such a big delay afterwards. And as I say you almost sort of forget what you’ve done. So it might be nice to know that your contribution has been worthwhile and maybe has found something interesting a bit closer to when you do it yes.

 

I think that’s important and a lot of people have said that, to have those results in a way so that you know, yes it was worthwhile, and just because you want to know.

 

Yes, yes and because, you know, when you’re doing something to, that you’re hoping has a benefit and find something useful then it is it’s always satisfying to know that it has worked.

 

Health professionals should try harder says Robert to get young people excited about the aims of the trial and engage their interest.

I think even if you can, when trying to recruit someone explain, well first of all what, really if you can try and excite them about the aim of what the trial is then if you can engage their interest then that’s half of the battle won, you know. So that this trial that I wasn’t eligible for sounded really cool because it was just measuring glucose levels of your breath and the eventual aim was to try and make it so that they could just have like a breathalyser that measured your glucose and would predict whether you were about to have, you know, whether you were about to get ill or have an infection and to try and catch the infections earlier and so, you know. The idea that you’re contributing towards a cool breathalyser thing that sounded really exciting. So I think that yes if you can try and excite them about what the aim of the trail is then that will encourage more people to do it and, you know, because then they’ll think oh, you know, “One day I’ll be able to tell people oh I helped, you know, I was part of the trial that did that”. Because that’s what with the gene therapy thing if it all works and get to say “Yes I was on the trial that pioneered gene therapy for Cystic Fibrosis” and that will be.....

 

Cool.

 

Yes [laughter].

 

Taking part in a trial can be a lonely experience, but Robert feels that sharing experiences of taking part in clinical trials is something young people might find helpful.

Yes, well actually on the subject of a website, it might be nice to have one anyway to talk to fellow people and say “Oh I did this trial did you do that one?” Because it’s, it is interesting to talk about it with other people and in particular if they have been through the same experiments that you have then you can talk about them together. But again I don’t know how sort of allowed that is from the point of view of the trial.

 

I think the only thing I would say is actually going back to what I literally just said about the maybe having a site to talk to other people who have taken part in clinical trials. Because obviously, you know, I can talk to my mum, my brother, my friends but I’m the only one that’s actually done the trial and been part of it and, you know, in that sense it’s sort of lonely in that no one else has been through it and really knows what I did or understands what it’s like going through yes. So it might be nice to be in touch with other people to share experiences.

 

I think that’s a good idea actually because it can be very isolating.

 

Yes and as I said though even though I talk to, you know, lot’s of my friends who studied science at University, they all get very interested in what I’ve done and the science behind it. But I’m the one that lived through it as it were, so yes it might be nice to yes and I mean that’s even on a more general point of just having Cystic Fibrosis, you know, I’ve got lots of people that care about me and that understand and I can talk to but that’s different from having someone else properly in the same boat as you that you can have, share that thing with.

 

There may be no immediate benefit from taking part in a trial says Robert, but you may be helping yourself and others diagnosed with the same condition in future.

Yes, well I guess I’d say I don’t know I guess to try and, even if it’s a trial that you might not benefit so much from, to try and see past that and see, you know, as a person with Cystic Fibrosis, you know, we are a community as it were. And to look out for your fellow people in that community and the people yet to come in future generations so because you can’t get a person without Cystic Fibrosis to do a trial that needs a person with Cystic Fibrosis. So it’s a responsibility in a way yes. 

Young people starting work may have to reconsider taking part in other trials.

It will be interesting now that because before I’ve always been a student so, you know. If I take part in a trial then, you know, say something does go wrong then you can always take a year out or, you know re sit an exam or whatever. Whereas now I’m moving into the world of work so I’ve, well I have an extra, being a teacher as well, I’ll have to factor that into my decision of what trials to take part in and how involved they are and what the risks might be.