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.

There was a follow-up at two years and then a follow-up at five years to look at development. So we had a two-year follow-up in [hospital name] actually. And then we got the results of those all printed out and all written up and posted to us. We had copies to give to our GP. And then the trial itself, they were brilliant at communicating actually. So you get, I think kind of two, yes, probably every six months a newsletter comes out, you know, about the number of people who’ve been recruited, what’s going on, what they thinks’ happening. They even send us Christmas cards I do believe. I think they might have stopped that now. But it was, the communication, you know, know, was really very good. And actually what they found with the antibiotics is that it doesn’t make any difference whatsoever. So that antibiotic is not going to be used in neonatal medicine. So that was quite surprising actually, because that wasn’t what we’d been led to believe. So that’s quite an interesting turn on, you know, why you need clinical trials actually to, even though everyone had a good feeling and it had been very successful in paediatric medicine, for, for neonates that wasn’t applicable. So that was quite interesting. So we had the antibiotics for nothing. But never mind. I’ll put that down to, that’s just the way it goes. That was the purpose of it.

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.

Well there were two vaccine’s and the way I understood it they were seeing which gave the best immunity and then deciding if that was the one that would be given to the population at large. And we knew there were two companies involved and they were looking at those particular companies.

I mean did that have an influence in as well as taking part that actually it was quite open in what was happening and who was behind the vaccines. [Yes.] Yes, that there were no other companies.

Oh absolutely yes, yes. I think, to be honest I can’t remember exactly what they said but I knew the names of both of the companies and I knew that they produced vaccines so I felt that they wouldn’t be bringing them to human use if they hadn’t looked at them really carefully.

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.

So that was a little bit more, I can’t quite see the benefit for u but I can see the bigger picture in that, you know, ten years down the line this needs to be more finely tuned as a treatment. And although growth hormone, as far as I understand, has been used in the UK for quite a while, it still, well, certainly it still feels a little bit embryonic at, at, at times. And, you know, for example the best dose for a 4-year-old isn’t standardised. So that was a bit harder. And we went into it thinking, Well, actually if we’ve signed up to thi which we have, because we signed up to injecting him with growth hormone till he’s 18, it’s a big commitment
And that was a hard decision to make because the only benefit is that our child is going to grow and not be extremely small in size and height. You know, there’s nothing medically wrong with leaving him small but, you know, we have decided to do it re-, really from a social point of view. So then to take part in a trial whereby it felt, you know, slightly like these dosage levels have still, you know, still need tweaking and it seems a little bit uncertain was much more, you have to think of a bigger picture as I say. So ten years down the line it will be really nice for children going through this to know, I don’t know, the medium level of, you know, that’s where you need to be or, you know, and, and for people to have a much bigger picture. And my feeling is, and it might be wrong, there are not that many children, you know, go, you, through the process we have. Most do catch up. So these small for, you know, small for, small for gestational age children, there aren’t that many of them about, although I might be wrong. So actually it’s really important that they try and catch us and get us to be part of it.

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.

And certainly where I had worked at that point, I often worked in the infectious diseases unit in and knew cystic fibrosis people had come in with it and, you know, that once you get it it’s quite a severe virus and that people who immune are compromised get it. So I think for us from that point of view because, I think had he been a surgeon or something he might not have been that caring. But his main thing was if she can get, even if she only has a 50% chance of getting the vaccine it’s got to help her. So, you know, so that was my main motivation I suppose.

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.

I think I’d, I’d just say that the medication is currently in use anyway. They just want to see how effective it is. The placebo isn’t a medication. But the only way that they can get anywhere is to research it. Throw the placebo in as well to see if it makes any difference. Because a lot of things psychologically, I think, pop a pill in, I think you feel better. But, yes, basically just that. it’s, I don’t think it’s a, a, a major thing, putting forward yourself or your child for a trial if the medication is already in use. it’s just collecting data. But I think its data that needs collecting just so they can help people in the future.

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.

There are real concerns about undertaking research in children. A feeling that, that that is perhaps unethical to do that. That children shouldn’t be experimented upon is the sort of thing one might hear or read about. And families and young people and children themselves and health professionals working with children are equally extremely sensitive that the research that is done has to be done under strict ethical guidance. And there are very clear ways, in which independent ethics committees and boards can check and supervise that the research practice is correct. I think there is an important statement to make about the ethics of research in children, and that is that it is being done with a purpose and the purpose is to improve the treatment and improve the knowledge and the safety of medicines for children. And overall about half the medicines that are used in children have not had proper evidence and are being used in what is called off-label or in an unlicensed way compared to the proper license that has been granted for adult use. And there is an ethical dilemma between continuing a situation in which we have untested and unproven medications for children against the other side of the coin, as it were, in terms of testing these medicines in children in proper situations to improve the evidence base and give us a better set of therapies for children.

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.

Nurse from the CHIP Team came in, went through with the Intensive Care nurses in terms of what they needed, needed to do with her. She only really needed I think two lots of insulin because she was doing quite well anyway. But they had two ways of doing this, they randomly select whether she’s going to be on tight control or normal control. They came back and they said she would be on tight control. Then she came out of Intensive Care. They did a follow up with her on, back on HDU ward, High Dependency Unit, and, you know the nurses since then maintain contact to check on how Chloe’s been doing. Unfortunately she’s deteriorated again; decided she wanted to go back into Intensive Care again, possibly because she got some addiction to Midazolam. I think she’s in love with it; I’ve got a vat of it here for when she comes home eventually. But I mean the information supplied was excellent, you know the support from the doctors and the nurses involved was, was superb. And the plan now is to do a follow up progress, really to keep a diary for the next twelve months on, whether she has any medication, what medication she has is for, if she’s back in hospital for any reason. And they’ll send us a questionnaire in twelve months in terms of how she’s been in herself, how she’s been medically. What intervention she’s needed if any, what our thoughts are etcetera. It seems to be quite detailed, well thought out, well prepared, well put forward.

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.

Now in order to prove the efficacy of the diet overall they had a control group for three months. So when you went in on your initial appointment you were randomised, by a computer to either the control group or the diet group. So the control group would have to fill in all the forms, and do all the seizure diaries and everything else, but and they, no treatment would change for three months. And then eventually they would start either one of the diets, and they would be randomised to either the MCT or the classical. Now Matthew wasn’t randomised to the control group so he didn’t have to wait three months to start the diet. And he was randomised by the computer programme that did it. And he was randomised straight to the classical Ketogenic diet.

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…

Short?

or short synacthen test, where they, they put, put a line, they took blood from Sarah and they then put a drug into her. [Is that right?] Which is meant to, to get her adrenal glands going; which, which should, we were told that the numbers that they were looking for, they were saying when they did the initial blood test she should have a level of about 100 and when they put the drug in it should go up to over 500, and, and they, they tested this all the time That was on the, the Tuesday afternoon and they said again, This is just to put your mind at rest that there’s nothing wrong with her But first thing on the Wednesday morning we got a phone call to say that she actually had the lowest test results that they’d ever seen. Her results at any point didn’t get any higher than 50. Which, they were meant to get to over 500, 600. And so there was, we had to pick up medication for her urgently at the hospital because she, she was desperately low on cortisol and she wasn’t producing any herself. And that, at that point we then got referred to another lovely consultant who, who’s now looked after us all the time because she has got quite severe adrenal insufficiency. And, and so we’re, we’re on a learning curve now. But we were told that it was only thanks to doing that trial that we found out. Because her, basically her life was in danger and we had no idea until we’d done this trial what was wrong with her.

I can’t say it was explained. I mean the main drive that I think was given to us was the fact that it may save children having to have blood tests. If we can, if we can do it this way, you know, we can give a child a cotton bud to chew for two minutes, get that tested rather than a blood test, then that in itself, particularly with Sarah’s experience of blood tests, seemed good enough and persuasive enough for us to continue.

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

Well, it was to monitor how Liam, like my son, was doing on like the like obviously the NovoMix 30, the insulin they give them and how he reacts to it and like certain stuff that makes him high, low, all different type of things like that.

So was it something new? A new type of injection or insulin?

No. But it’s because obviously he was just only diagnosed it’s to see how he was reacting to it at the time. Because obviously not everyone’s the same. So obviously they had to see if he was, obvious-, because there’s type 1, and type 2 where he might have been able to have the tablets, but he’s not, he’s type 1, so he has to have injections. So it was just to see how everything was going, to keep a monitor on him, I think, to keep an eye on, on him. And also make sure I’m learning at the same time. Because obviously you can’t just go, Oh, your son’s got diabetes. Go home. So some, you have to have training. And every, personally, everything that went on, I thought was brilliant. Like the hospital were absolutely, they were brilliant.

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.

So it’s kind of looking at which is the best way to –

Yes, inform a-, adolescents I think and support them with the diagnosis. Because the, the research that we fill in is very much from the basis of the child. So Saskia fills in how she feels and I fill in how I think she feels a little bit, or how I feel about her.

Yes, I think they’re thinking of giving it straight away, seemed to be the impression. Which is fine, because you can choose when you read it, can’t you? You do, the problem with diabetes is you need a lot of information. So it’s all very well saying, Well, we’ll just drip-feed it and . . You need a lot of information because it’s quite complicated. So if something happens and you haven’t had that piece of information that’s more of a worry. So we had a diabetic nurse who came who was a big support and was very good. But I’m the sort of person who would just be like, if I knew that they were going through a process, which I can look back and see they were now, I’ll see you here with Saskia firs do you know what I mean? And we’ll do.. I’m more the sort of, I don’t need that hand-holding. I’m more, Tell me everythin because I’d worry if I don’t know everything. I’d like to know everything and then I’ll deal with it. Everybody’s different, aren’t they? I think they go through a process of support. Because I’m not sure everybody, you know, we manage it well, Saskia’s very well controlled because it’s, to us it’s very very important, her physical health, and I’m not sure it, she, everybody is as controlled as she is, do you know what I mean?

I mean I know from, you know, friends and things who’ve got illnesses where, you know, a normal clinical trial I would think about was where you’re testing something new in, in comparison to something, so you might get given a placebo or you might be on a new drug and then they would make a judgement based on the results of that. So I did think, Oh, I’m not quite sure how this works But now talking to you it’s obviously, it, it’s reminded me what they said at the very beginning about comparing the treatment options as opposed to anything drug related.

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.