Clinical trials: Parents’ experiences

 The second trial that we were signed up to, I can’t remember the name of it. And that was to look at preterm babies under a kilo, so we, you know, fitted that perfectly, and to look at the use of antibiotics to reduce sepsis. So our understanding of it was that sepsis is a kind of a, really a primary cause for neonatal mortality and it’s a, a big concern for any neonate, and we want to do what we can to minimise that risk as parents. So it was a randomised trial where you were either picked to have the antibiotic or you weren’t. It’s used in paediatric medicine and has been extremely successful, so it was now looking at whether it could be applicable and transferable to a neonatal unit. Everyone was really positive about this and the vibes we got was that it was a really important trial and it was great to be part of. And it would be great actually if we got given the antibiotic because we had a very small low birth weight baby who, as everyone led us to believe, would benefit from this. So we were quite happy to do that, because actually from all the reading and all the background material I’d done, you know, sepsis seemed to be a huge, huge risk, especially to us. So actually if we could do anything to minimise that risk then, you know, that was great. So we signed up for this, and we were given the antibiotic. Now we were approached by a consultant and also a registrar as well, who explained very, very well what we, what we had to do. So I don’t feel that we didn’t get given enough information. I felt that there, there, there was a lot of information there. And our child was given the antibiotic and everyone was very pleased about that. I didn’t really think too, too much about that. Now we knew as part of that trial they were also going to follow up development at two years and then again at five years. Looking at dosage, we were given good information and the vibes we got that, was that it was a really, was really good that our child had, had been picked to, to have the antibiotic. And I was aware in the neonatal unit of other parents being invited to take part in, in the trials as well and also aware when people weren’t picked to, to have the antibiotic as well.

 Seeing about different dosages for children who were born less gestational periods than there was, because Callum was born at 33 weeks, but was the size of a 28 week baby. And for children who haven’t caught up, they’re giving them growth hormones to see, but they’re trying to see as well as if, because at the moment they don’t give children under five growth hormones. And they wanted to see as well is if a child was born small, if it would benefit them to giving them earlier on, so they caught up a bit quicker, so then it wouldn’t be when they get to school that they start all this. They would have done it, so they’re looking into what is the best age and what is the best time to start the growth hormones.

 Okay, with last year’s extreme anxiety which I feel was enhanced by media coverage of the swine flu threat we were interested my husband and I when our hospital Trust e-mailed some employees to see if they had any children who would be willing to take part in swine flu vaccine trials. Because of our concerns with our son who is asthmatic we thought that might be an idea to get him vaccinated prior to these vaccines being released because at that time we weren’t sure when they were actually going to be released. So we answered the e-mail and we were contacted again and asked to take our son who was at that time 11 to a children’s hospital locally where he was seen and assessed for suitability to take part in these trials. It was explained to us there were two different types of vaccine and they were being trialled to decide which would be taken on board to be given to the population at large. We had it explained to us that the vaccines had different constituents and we were talked through those. One had a mercury derivative; were we happy with this; there was no threat etc.etc. So we understood that.

 Because our child has never caught up in height and weight, we were suitable should we want to go ahead with growth hormone treatment, which we opted to. And at that stage, once we said yes, we were then asked whether we’d take part in a clinical trial. Now that, you know, that was a slightly harder decision in many respects to make because it has been costly in terms of time really, which is why we’ve decided not to go any further with it. So our child has growth hormone treatment, treatment which is inject, he’s injected with growth hormone daily to, to, to make him grow at a normal rate. And in the first year there’s some uncertainty as to the best dosage, and models are different in America as they are from Europe. So we were part of a trial to see which dose is the best really. Now as it happens we were given the lowest dose, which is great, great for us. And so far it looks like children do very well in their first year on the lowest dose actually and don’t need to be put on a higher dose. So we went into that with a little bit of anticipation because obviously I don’t know what dose before, we, you know, what we were going to be given and I don’t, you know, like most parents you don’t want to inject your child with some synthetic growth hormone if, if, if you don’t need to. 

 The purpose was mainly to see if, I think they were getting a lot of hospital admissions of cardiac children who had contracted RSV [Respiratory Syncytial Virus]. And so they were trying to see if it could help, the vaccine could help children, cardiac children by well preventing them from getting, contracting RSV. Whereas they at that stage it had only been licensed for children I think with cystic fibrosis. And it had only purely been licensed for lung drugs rather than for cardiac. And I think they were trying to, very much driven by the cardiologists that if they, their patients could also get access then, especially for the, it appears that the sort of danger period is the 18 month, first 18 months. So they could have the drug for first, you know, get access and get, stay healthy for the first 18 months, their side effects would be less.

 Well, it, it has, it, it, the, both the drugs that are being trialed are currently used for childhood migraine, but they’d never done a study. And it can also, they’ve, they’ve linked, see, the other side that didn’t want me to do it, childhood migraine they’ve found is linked to epilepsy. So treatments that are given for migraine are actually also used for epilepsy in children. Which, the symptoms are slurred speech, slowness, and I didn’t want him to fall behind in school. And that’s why initially I said no to medication in 2008. But I think because they got so severe, so I don’t really think we had much choice. We had to try something. Toby was going to be put on one of these medications anyway, so we thought, “May as well go ahead with the trial.” If they can get more feedback from parents and children, anything can help.

 Nearly all medicines are tested in adults before they’re, they’re tried in children. And it is true of all such research that the safety of the people taking part in research is the most important standard that we are all holding to. And great care is undertaken to provide a plan of the research, called a protocol that puts safety first. And the governing principles around the clinical research are even stronger than those that relate to clinical care for instance in, in, in the NHS. That is, the standards of research practice have to be even safer and more stringent to protect the safety of participants, be they adult or child.

 I mean we weren’t offered into the CHIP Trial [Control of Hyperglycemia in Paediatrics] until the second bout of being in hospital really so, we came home, had a week and a half at home, started to deteriorate again, back into [hospital] which was quite straight forward at the time. She was in for a week suffered a massive cardiac arrest, shut down for six minutes completely, back into Intensive Care again. Doctors weren’t too sort of positive to begin with. She seemed to be, sort of struggling to, to maintain blood pressure etcetera, etcetera. So really about as poorly as you could possibly be I guess, at the time. Because she was intubated so on a ventilator, there’s a number of hospitals across the U.K. that are running this CHIP Trial. The idea being that in adults of a similar survey for want of a better term in adults, and they found that in adults if you, if you maintain a very close observation on blood sugar levels and maintain that very tightly as opposed to let it go quite a broad way before giving insulin, it increases the recovery rate. So they recover from Intensive Care much quicker. So we were approached by the doctors to consider whether we wanted to put Chloe forward to be a part of that trial. 

 The clinical trial that Matthew was part of was to test or prove the efficacy of these Ketogenic diets. There are two versions of the Ketogenic diet. There is the classical Ketogenic diet and there is the MCT version, which is the Medium Change Triglyceride diet which is where there, there are different types of fat given. So you’d have long-chain fats more for the classical diet, and then the medium chain. So with these two versions, they were testing which one was better because there was a lot of information out there. They were saying that maybe the MCT diet wasn’t as good.

 And then the trial was, she had to, as soon as she got up of a morning, for three mornings, she had to chew cotton wool. And we put it in a bag and put it in, in the freezer in a special box, because the trial was about seeing if they could get levels of cortisol from children just chewing the cotton wool rather than doing a blood test. But in order to find that out they obviously have to do a blood test after the three days to see what the levels are. So we did that for three days. On the third day we took Sarah to the hospital, where she met the nurse and a lovely doctor as well who, who sat with her and chatted. And they had all sorts of games and books and things for her, to, to distract her. And they did, I think it’s called a synacthen test, a long syn...

And do you know what the purpose of the trial was? Do you know why you were invited?

 Yes, yes, I think we got a big, we got a pack there and then I think, if we said yes, I think. Saskia came; I think that’s when Saskia came away with a big file full of lots of information. And my understanding was, thinking back, that the trial was to help them decide whether on diagnosis as a child, I think it’s going to be for, Saskia’s category was sort of teenage because by that time she was 12½, so I think she was in the sort of, that juvenile rather than young child, what support is given on diagnosis. Is it this big pack that she then got, which has got all the information and is child friendly, that’s written for children? Or do we do it in the way we’ve, they’ve done it where, they did give us a pack and some information at the start and then drip-fed some of the information through the diabetic nurse? But I don’t really know how it will be completely different, because I’m assuming you would still get support of the diabetic nurse, and this is just an information pack.

 And I mean when we first agreed to do the trial, it, I mean I think the fact that it was all to do with information and it wasn’t to do with changing her treatment, her medicines or anything like that, it was just to do with information. And I guess as a result of that maybe we didn’t think about it as much as we might have done if it had been, you know, there’d have been more impact if it had been a change in treatment or sort of a new treatment that was coming out. But even so, I mean when they said that it would be a random selection as to whether we would get the new pack of information or the old pack of information, and we were actually selected for the old, for the old pack of information. So in actual fact nothing changed for us in the way that anything was happening with my daughter’s treatment or the information even that was given. I mean that was a bit disappointing because you always think if there’s something new coming along then it might be useful to, to, to see it. Because I guess if as a result of the trial it’s decided that’s not the best approach, then we’ll never see that. But then it’s not the best approach, is it? They’ve made that decision based on the research. So I think that, that’s fine. And, you know, initially when they said that we would get the standard information, then, you know, it was a bit, “Oh, what a shame.” But it’s, I think it’s, it, it’s worked out fine. And we’ve not seen any differences. So, you know, there’s, there’s not been any impact from that point of view. And it has just been answering the, the questions on general every day, day-to-day stuff really.