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Clinical trials: Parents’ experiences

Clinical trials: Parents’ experiences

Understanding allocation (randomisation) to a treatment comparison group

In Phase 1 and Phase 2 trials everyone taking part will get the treatment being evaluated. (See also 'Why do we have clinical trials in children and young people?’) However, many trials compare a new treatment with the standard or usual treatment by comparing two groups of people. People in one group are given the new treatment. Those in the other group are given a standard or existing treatment. (Sometimes you may hear the new treatment group called the ‘experimental group’, ‘trial group’ or ‘intervention group’. This can be confusing, as all the groups, including the control group, are part of the trial, and people in the control group may also be given an intervention, in the form of the standard treatment or placebo.)

To make sure that each group contains a similar mix of people, many trials are ‘randomised’. This means that people are allocated at random to one of the groups in the trial, often by using a computer programme. When people are randomised they have an equal chance of being in either trial group, and their personal characteristics are not taken into account when they are allocated. Random allocation helps ensure that two very similar groups of patients will be compared, so if one group does better than another, it is likely to be because the treatments being compared have different effects, and not because of differences between the people in the groups.

If no standard treatment is available, the control group may not be given any specific treatment, or may be given a placebo. A placebo is a treatment with no active ingredient, which is designed to appear very like the treatment being tested. By comparing people’s responses to the placebo and to the treatment being tested, researchers can tell whether the treatment is having any real benefit, rather than patients simply feeling better because ‘something is being done’.

There are several ways in which the results of trials can be made as reliable and accurate as possible. One of these is to make the trial a ‘blind trial’. In a blind trial the participants are not told which group they are in. This is because if they knew which treatment they were getting, it might influence how they felt or reported their symptoms. Some trials are double-blind, which means that neither participants nor the doctors and others treating them know which people are getting which treatments. This also avoids the doctors’ hopes and expectations influencing the results of the trial.

Helena, a senior research nurse has a lot of experience of clinical trials and explains what some...

Helena, a senior research nurse has a lot of experience of clinical trials and explains what some...

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Yes some terms that we use are blinded which means that you don’t know what treatment is, the patient’s having. There is single blinded where just the family don’t know, double blinded where the family and the researchers at site don’t know. Randomised means where a patient is randomly allocated one arm of the treatment of the study or another. And there can be two arms to the treatment, there can be three arms and they are randomly allocated. There are lots of different ways that they do like and how they’ll be done electronically or it can be done by sending a fax and someone not related to the study, here at site, randomises them and picks the next consecutive envelope, as simple as that or enters it onto a spreadsheet and it randomly allocates them.

Placebo, there’s some studies what we call placebo controlled and some patients get a placebo. A lot of families like it explained as a dummy drug so it’s a drug that isn’t a drug. It can be a lactose tablet or something else. And it will always be made to look exactly the same as the actual active drug so that people don’t know. So if, if we had a girl and a boy both on the study they couldn’t look at the drug and say “Oh well you’re on different than me”. They would always look the same and the tablets would look the same as each other, liquid would look the same as each other and everything would look the same as each other. And the idea of a placebo is, is it that the active ingredient is helping the patient and the symptoms and the quality of life or is the fact that they feel that having something is helping them and that’s, it’s very interesting to see the placebo effect.

Helena gives some examples of the questions parents ask when their child is taking...

Helena gives some examples of the questions parents ask when their child is taking...

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And one thing about being randomised and being blinded the questions that the parents ask is ‘when will I know what I’ve been on?’ Now for some studies they will and today I’ve seen a patient for a study who I’m about to send to the sponsor to request the patient to be un-blinded at the end of the study which has been approved by ethics. Because the child’s been on a treatment for a whole year and if they don’t know what they’ve been on, now we’ve stopped study drug what shall we put them on? So if they’ve been very well for a whole year then it would be wrong really for them not to know. So when they go back to their GP they would be back to where they were a year ago on control with the treatment so that’s something that we always explain to parents or sometimes parents ask when will I ever know what the child’s been on. And for some studies they never will, but for others they do and that’s so that we can get them on the right treatment at the end which is ethically the right thing to do really for that type of study.

Some of the parents we talked to had enrolled their children in a double-blind, randomised placebo controlled drug trial. This means that children were allocated randomly to either trial group and neither parents nor doctors or others treating them knew which group their children were in. However, all the parents we talked to were willing to accept this, though some were still hoping their child would get the new drug in case there was a chance their child would benefit. Some parents who had been in other types of trial were not sure they would be willing to enter a placebo-controlled trial.

Dr William vant Hoff explains about the importance of randomised trials.

Dr William vant Hoff explains about the importance of randomised trials.

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Sex: Male

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In many trials or studies of medicines, part of the proper practice is that the participants, in this case children are not aware whether they’re taking the new medicine or perhaps a dummy medicine called placebo or another medicine. Because if they were aware or their doctors were aware which one they were taking, this might influence the results and give a false answer, which none of us want. And so in many trials like this the term blinding is used and this refers to the patients, the families and/or doctors not knowing which of the medicines the child is on. Is it the new medicine or is it the comparator medicine? Now these are very useful and, and in a way the gold standard to provide the best research answer. But thereafter longer-term studies can be done when the medicine is known to the family and to the doctor. This is a, a, a phase of a trial that we call open-label, in which they are aware which medicine they’re having and may continue to take a medicine if it’s been shown to have a benefit.

During her stay on a neonatal unit, Catherine enrolled her son as a control participant in a study on gut deformities in unwell premature babies. She reflects on her own experience of being invited to join a placebo-controlled trial. She says that making a decision to take part in such a trial can be difficult, depending on the situation you are in.

Enrolling your child in a clinical trial when you dont know what treatment they will be given is...

Enrolling your child in a clinical trial when you dont know what treatment they will be given is...

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Age at interview: 27

Sex: Female

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One of the studies I was going to be, participate, in before we knew about my genetic problem was a progesterone trial when you when you got pregnant you’d be given progesterone. My issue with that really was I’d had three miscarriages by then and my issue with that was that if I was in the, the placebo group I wasn’t getting the progesterone and when you’re talking about whether it’s going to help you keep a baby that’s sort of a huge, it’s not like you’re taking a you’re taking a drug to see if you think you know does it work against aspirin. It’s like if I have this drug I might keep the baby and if I don’t I might lose it and if I’m in the placebo group I won’t know and I, and that would have been a really tough call to make. As it was I wasn’t accepted because I found I had an underlying condition. So I guess it all depends, it’s like if you were saying if he had cancer and you wanted to give him chemotherapy or a placebo group why would you want your child to not have the treatment because you were in the placebo group and you don’t know and then it it’s really tough isn’t it. I guess you really have to think about each thing as you come up to it; it’s not something you can just say we’d be in or we wouldn’t be in. It would depend on the circumstances.

Jo’s son is in a drug trial for the treatment of migraine in children. Not knowing whether her son is on a drug or a placebo has been difficult at times. Jo won’t know what her son has been taking until the end of the trial, but as she says there is nothing to lose because “You don’t know which one is going to come out on top”. She is pleased they are taking part if it can offer some help to her son and other children.

At the beginning of the trial Jos son showed signs of mood swings. She immediately told the...

At the beginning of the trial Jos son showed signs of mood swings. She immediately told the...

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Age at interview: 28

Sex: Female

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I think it’s, it’s a constant worry as well. You’ve put them on something you don’t really. I know you can read up on the leaflets and stuff, but you don’t know what they’re on. So when, like when Dan started having mood swings we didn’t know whether it, we don’t know if he, he was, if he was on a placebo or not. We still don’t know that. We won’t know for eight weeks, for another eight weeks.

But I think that’s, obviously it’s part of the trial and you have to not know, but it made it difficult if he was ill or, like I say, when he was moody and he was very lethargic. And Dan’s not like that. He’s a rugby player and he’s very… So for the first, when he was on the high dosage of the tablets for the first six weeks, he was, wasn’t his normal self and it was a little bit worrying. But we didn’t know what to, what was contributing to it. So, I don’t know, it was just a little bit of a tense time for everybody, I think. And also hoping that it would work. And then we were a little bit, I felt a little bit dismayed because we were hoping… the Doctor described it as a good phase. Dan was in a good phase when he came off the tablets because he hadn’t had a migraine in, in a month. And as soon as he came off them, for him to have a full-blown one where he was pretty much out of it for two days, and it was a little bit, I think, disheartening for everybody. That it kind of hit home that maybe if he was on something, which it seemed, you know, he may have been, that he’s going to end up taking tablets until an unspecified date. So it’s a bit, a bit much.

Yes, we, I know, I know that placebo means just, it could be candy for anything, it could be sweets. It does, it just if sometimes the brain thinks it’s getting help, then the body heals itself. Some people believe that. The other two drugs that were on it were, we were assured that they, they were well-used drugs. The trial in itself, we’d been through a lot with Dan’s migraines and there was no one, there was never any solution or never anyone there to help. And I think when we sat down and explained it to Dan, one of the, one of the reasons we used or explained to him as a child would be that, “If you do this and, and other children do this, then in a couple of years’ time maybe, maybe little boys and girls won’t have to suffer with headaches like you’ve done.” [um] So that’s how we explained it to him and that’s probably how I’d explain it to, to somebody else who asked, “Why would you choose to put him on a trial?”

I know for definite that Dan’s, the nurses and the doctor didn’t know what he was on. They still don’t know what he’s on now. I think that probably made it difficult for them as well in the fact that when I rang up to tell them that Dan’s moods seemed to have changed and we weren’t sure whether it was because he’d changed schools or because of his age or because, and of course they don’t know whether he’s on something or not. So I don’t know whether, if he hadn’t have, I think that they don’t know because if he hadn’t have been on something, and if he’d been on the placebo, they would have said, “No, we, no, we’ll just leave him on that. It’ll be fine.” And maybe I would have known that he wasn’t. I don’t know. I can’t, it’s not my field to say whether or not the doctors and nurses should know what he’s on. They, but they seem to have coped fine with not, with not knowing what he was on.

As the trial has gone on, Jo would now be surprised if her son had been given the placebo, but...

As the trial has gone on, Jo would now be surprised if her son had been given the placebo, but...

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Age at interview: 28

Sex: Female

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I’ve been grateful for the help that we got off the nurses and doctors, and we’ve been reassured it will continue anyway and, plus the fact that the trial is being done so that they’re, they’re trying to, they are trying to find the best drug all round for children. So regardless of whether or not he was on a tablet in the first place, he may be put on one if they feel that that’s the best. And then they’ll monitor him. And we know that they’re going to make sure that they find what’s right for Dan. But I don’t know. I think it would be strange if he’d been on it, if I’m honest. I think I’d be quite shocked at this point, if he’d been on it. Especially, at first we were very unsure whether or not he was on it. His migraines were a lot less than they, than they normally were. But for him to have one in such close proximity to him coming off the tablets, I think would be quite a shock. But it’s a possibility, and if it is it means that he had a really good six months. So we’ve, you know, we only had three migraines in six months. Which is really good. So either way there’s no losing, I don’t think, in this situation. He may be, he may have been on one of the drugs, but the other one still may suit him better. You don’t know what the trial is going to, it may show. You don’t know which one is going to come out on top, if either of them.

Randomised trials are done when we don’t know which treatment is best, in other words when the relative merits and disadvantages of different treatments are uncertain. It is important to realise that, on average, new treatments are as likely to turn out worse as they are to turn out better than existing treatments. This means that, going in a trial, everyone, regardless of which of the treatment groups the computer allocates them to, must have similar chances of a good outcome. If, in spite of the treatment uncertainties that the trial has been designed to address, people would strongly prefer one of the treatments being compared, they should not volunteer for the trial.

Lucinda’s son is also taking part in a double blind randomised placebo controlled drug trial for the treatment of migraine in children. Although this is the first time they have tested the drug in children, knowing that the drug was used to treat adults and children was reassuring to Lucinda. If it had been a new or untested drug she would not have agreed for her son to take part. Her son received his own information about the trial and the doctors talked to him to ensure that he understood about the different groups. (See also ‘Involving children in decisions’.)

A placebo isnt medication, although believing that it is may make patients feel better, but it...

A placebo isnt medication, although believing that it is may make patients feel better, but it...

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Age at interview: 37

Sex: Female

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Yes, we don’t know what Toby’s actually trialing at the moment. We don’t know what he’s on. Obviously a placebo does nothing for you. It doesn’t help at all, but it also doesn’t hinder. So that’s why I was quite happy to go on it. But, no, we don’t know what he’s on. I can’t really tell. Because they have asked if I can tell if there’s a difference. I can’t really tell at the moment.

I think 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, it’s, I don’t think it’s 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 it’s data that needs collecting just so they can help people in the future.

Lucindas son had his own information and signed his own assent form to say he was happy to take...

Lucindas son had his own information and signed his own assent form to say he was happy to take...

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Age at interview: 37

Sex: Female

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Well, I think it’s good, like you say that he has his own information. And, and did it come, I mean was it all words or were there words and pictures?

No, it was, it was all words, but it was, wasn’t clinical, scientific. It was

How did they explain the groups in the information, that there would be…?

Just that, they did say that like they were testing two medications and they, again they put the names of the medications, they were in bold. And then they said the placebo and they explained what the placebo was. And it just said, “You won’t know what you’re going to be on. But if you’re still happy, sign this.” And he had to sign his own consent form. So, yes, it was a big thing for him.

How did they explain a placebo?

Just that it wasn’t medication.

Lauras daughter took part in randomised trial on the prevention of eczema in babies. Although...

Lauras daughter took part in randomised trial on the prevention of eczema in babies. Although...

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Age at interview: 32

Sex: Female

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They’d be notified by the hospital when the baby was born, they’d contact us, make an appointment and come and see us. At which point we would be put randomly either into a control, control group where we wouldn’t be given any creams to apply or the group that were going to be given creams and if that was going to be the case then we would be given an option of three different types of emollients. So there was sort of a thick Vaseliney type cream, one that was more like the Diprobase that we use for our elder, elder child that’s a, a lighter cream, and then sunflower oil. And all of these had been they had been chosen because they shouldn’t create an allergic reaction and they shouldn’t in themselves be able to create a problem with eczema. And the idea being that potentially if you use one of these regularly at least every day and after bathing that you might be able to just help the skin’s barrier a little bit and it might prevent the onset of eczema. However, there is of course a very slight risk that if you are doing that and you are creating an artificial barrier, it’s not allowing your baby’s own skin barrier to develop in its own way.

So it seemed to us to be minimal risk and we thought, “Okay, well, we’ll take part in the study.” You got a, a Boots voucher for £15, which was quite nice, whether you took, whether you were in the control group who were not applying the provided emollients or whether, whether you were, whether you were using them. And this voucher was so that you could go to Boots and get soap-free bath things and any creams that you wanted, I suppose, if you were in the control group. And we thought, “Well, there’s nothing to lose here. There’s, and there’s potentially everything to gain.” So we’ve been taking part in that study now. We chose the sunflower oil because we thought that might be useful for baby massage as well. So it was a nice thing to do, spend a bit of time doing and make it into a bit of a feature, [that’s our baby] make it into, make it into a feature rather than an ordeal. And once we got into the hang of it and we got a bit of a routine going, and we lay out the towels and we oil her and she laughs and rolls around and it’s quite pleasant really.

The people that are involved have, have always been very nice, explained things extremely clearly. They’re always on the end of the phone if we’ve got a question and, and it’s been a very pleasant experience. We’ve not finished yet. We don’t finish until Beth is six months old. And we have to keep applying the emollient every day until then, whereupon she will be seen by a consultant, I believe, and they will check for any signs of eczema. And this, this person won’t know whether she’s been in the control group or the group who have been applying the emollient. So they will be able to say without prejudice what, whether they think that she, she has developed any eczema or that is, is likely to or there are any signs at all.

Because of her elder daughters experience of eczema, Laura would have been disappointed if her...

Because of her elder daughters experience of eczema, Laura would have been disappointed if her...

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Age at interview: 32

Sex: Female

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And I think as well if I’d been in the control group and not applying the emollient every day, I think that I would have been a little bit disappointed then as well. Because that would just have simply repeated what we’d done with our eldest child in a, in many respects. And I think that would have been a, a bit of a shame. So I feel at least we’ve had a go and done everything we possibly could.

I did, yes. It, it was a simple, it was done by computer. And they, they came to see me after the baby had been born, within the first couple of weeks, because obviously you, you’ve to start as soon as possible but not, not in a, not in an, a way that imposes on you. So they, they didn’t come within the first week, for example, because you, it takes a bit of time to settle with a baby. But as soon as it’s reasonable and within the first three weeks I think it was, had to be, they came round to see us, and from here made a phone call to see whether we would be in the control group or not. And it apparently came back randomly selected that we would be taking part in the study.

And did they say at that point, obviously you’re using the cream, so you know which cream, so you know which cream?

Well, they, at that point, that’s when they, they say, “Well, which of these creams?” And they allowed me to test them, and my husband was here as well, so that was quite nice. So we could make the decision together. So we tested them on our, our skins to see whether we, how we felt about them and, and, and made our choice. And we use the sunflower oil.

So there, how many creams were there?

There were three. So there was a Vaseliney type texture one, and a lighter cream, and then the sunflower oil.

An oil? So there were, so there were creams and an oil?

Yes. Because there might be a difference, I suppose, within the study with which emollient that you use as well as if you use one at all.

I was just wondering how they worked that with the, with going in, with the three groups.

I’m not sure. I don’t know. I, I did say, the, the Vaseliney one, the very thick one didn’t, it just was so gloopy and unpleasant in my opinion, and I did say, “How many people have chosen this?” and she said, “Not many.” So I don’t know, I don’t know how they do that. But I suppose they’ll be able to get a more random cross-section with that if, if the study continues. And if this is just to get some initial results then I, then that makes sense.

And you say, and the, obviously the nurses will know which one you were on. You were there, were, they were, when you picked it? But the, who’s looking at it doesn’t know?

No, the consultant has no idea whether we’ve used anything or, of what we have used, which one.

Yes, it was. That, and I think that’s important. In a study where you, where it’s, you’re going to put something on your child, I think it’s important that you’re comfortable with, with what it is.

Linda had also enrolled her daughter, soon after she was born with a serious heart condition, in a randomised trial that was double blind and placebo controlled. Linda couldn’t quite remember all the details but does remember the medication was marked ‘trial drug’. Linda was happy to take part as the drug had already been tested. She still does not know for certain which treatment her daughter received, but she is fairly certain that her daughter had the active treatment because she has done so well. Linda described this as like a lottery; others used terms such as ‘flipping a coin’ or ‘drawing it out of a hat’ to describe the way the decision is made.

Randomisation was a bit like a lottery. She does not know if her daughter got the actual drug,...

Randomisation was a bit like a lottery. She does not know if her daughter got the actual drug,...

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Age at interview: 43

Sex: Female

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A placebo, they probably did but I forget now what kind. They did say that some of the children would be receiving placebos some, they said it was, is it a double blind, does that mean, that rings a bell as where some children will be getting placebo, some will be getting the actual drug. They themselves didn’t know what was, it was all in I think, not, I don’t mean unmarked files but it wasn’t marked placebo. It wasn’t marked with the medication it was marked trial drug and you had to complete a diary for about 18 months afterwards to make sure that the child, to sort of say if they’d had any chest, it was all specifically respiratory problems, whether they had any colds, sniffles, coughs.

Well because it was a tested drug that didn’t bother me. It was more that I was sort of, a bit like the lottery were I was hoping she was getting the proper drug but knowing that even if she got the placebo it’s better than her not, you know. The odds were still there that she was getting the proper drug and if she didn’t take part in the trial she wouldn’t be given anything. So at least I was increasing her odds of getting the proper drug rather than her, you know, if she didn’t take part in the trial there would be no chance, this way at least she had a chance that was.

Sometimes when people do not experience a change in symptoms they assume they must be taking the placebo. However, this is not necessarily the case, as not all drugs work on all patients in the same way. Equally, people who are taking a placebo may sometimes experience improvements in their health. This is known as the placebo effect or placebo response. We still do not understand exactly why this happens, but it seems that believing a treatment will help can result in real changes. This is why new drugs are often compared against a placebo – if the patients on the drug do significantly better than those taking the placebo, it suggests the new drug really has an effect. If people were only given the new drug, we could not be sure if improvements were simply due to a placebo effect.

Parents we talked to understood that they would not be choosing which treatment they would receive. Alison has experience of enrolling her son in different clinical trials in neonatal care and when he was older, a growth hormone trial. She understands that the decision is made by the trial design which treatment your child will get.

When you enrol your child in a randomised trial, you have to be prepared to accept whichever...

When you enrol your child in a randomised trial, you have to be prepared to accept whichever...

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Age at interview: 39

Sex: Female

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You have no control over that. You know, as far as I was, I think it’s a computer isn’t it that does it. And so there you go; the decision is made for you. You just make the decision on whether or not you’re going to be happy with the outcome. So almost the outcome has to be irrelevant, because you’ve got to be able to sit with whatever the outcome is. And I think that’s what you need to focus on. “How do I feel if it’s a weight-related dose?” or, you know, “How, how do I feel…?” And I think providing you’re okay with that, then it’s….

Paul remembers there being different groups, but cant remember what they were, he was happy for...

Paul remembers there being different groups, but cant remember what they were, he was happy for...

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Age at interview: 29

Sex: Male

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So did they mention any sort of being in a group, you know, whether you’d be chosen to be in one group or another group? Did they mention that? Do you remember?

I’m not sure, see, I’m not too sure. I do remember groups but I don’t know if it was like putting like us there and them there, if you know what I mean. See, you did have, you did have groups, yes. I think it might have been. I’m not too sure.

It might have been a control…?

That’s the control group, yes, I did. That, that rings a bell but, as I say, it’s been that long, it’s sort of... The only things that stick in your head is everything else what I’ve got to do for my son. And that’s the, like the, try to keep on top of his needles, his finger strips, his insulin, making sure I’ve got everything and like not, it never runs out and everything’s like still in date because you’re only allowed it out the fridge for three, I think it’s three months and then obviously it’s no good after that. So you do have to make sure you’re on top of everything.

So it’s, more important is his care?

Well, yes, because you’re concentrating, see, anything else, you’re concentrating more on that. So it sort of, it sort of sticks in the back of your head, but it’s obviously, it’s, unless you read it or someone tells you, it won’t pop up into your head because you’re focused on the one thing. And, which is the main thing, which is obviously his care, to make sure everything’s all right for him.

And would it matter, did it matter to you that if you were in the control group and weren’t in the intervention, does that, did that make a difference in the decision at all?

Well, to be honest, I’d have to say no, because everything that, like everything that’s gone on I’ve, I’ve thought was spot on. It’s helped me; it’s really helped me a lot. So I couldn’t, I couldn’t say nothing bad about it. Every, everything to me is brilliant. Because, you know, he’s, I think my son’s been diagnosed now for two, two and a half years, and I haven’t had a problem on that in two and a half years. So to me that’s, that’s good. And it’s really, it’s really helped like. Personally I was scared at first at, like about learning of, you know, it’s a lot for you to take in. And then you’re giving him injections, doing his finger pricks, all stuff like that, and his food. But, to be honest, I didn’t think I’d be able to do it. And I did. It, they really did help. See, it, I, like obviously when he first got diagnosed and we were still in the hospital, that’s when you’re thinking, “Oh, I have to go home and do this.” But obviously they show you there first before you go home. And, to be honest, when they showed me I didn’t have a problem then about going home. It’s just at first I was thinking about it, going, “I’m going to have to take him home and do this. He’s going to be doing this forever.” But that’s before obviously I learnt it in there. And it, to be honest, it was brilliant. Like it all, it all helped me and I’ve had, I haven’t had a problem. So to me, they done good for me. They done brilliant.

Rachel enrolled her three children to a swine flu vaccine trial. She didn’t mind not having a choice which vaccine her children were given. However, she feels that some people may have a preference. It is important that when parents agree to enrol their children to take part in any randomised trial they understand that they may not get their preference.

Randomsiation is a bit like flipping a coin, but it is an important part of the scientific...

Randomsiation is a bit like flipping a coin, but it is an important part of the scientific...

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Age at interview: 35

Sex: Female

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But I would see it as being; I think it depends totally on what the trial is because obviously if it’s randomised then there’s always the element of chance. You never know which particular arm you’re going to be in. And it’s important everyone who’s, joins that trial is as equal as possible and as balanced as possible, so that differences can be determined from the treatments or whatever it is that’s being investigated. Yes, it was randomised, but it was just two vaccines that were being used. So it was which vaccine the child got basically.

We did know which one we got actually. Yes, it wasn’t blinded. So I did know. But, yes, I knew I had no choice in which one it would be.

And would it have mattered at all?

No, because I didn’t know anything about them. I quite liked the fact that the children had different ones. The two of them had the same, and one of them didn’t. I thought that was quite interesting to see. And I had a very slight inclination towards one or the other, but there wasn’t any logical reason for that. I have no idea why. I just remember the Baxter one, not being quite so keen on that one. But I have no reason why. I think it was probably something in the literature had possibly hinted that there was usually a, less of a reaction for the other one. I can’t even remember what it’s called.

I think really it just; a trial is just a way of being very scientific about something. So it’s not about looking at hunches or perhaps a couple of people do one thing. I mean we’re all very prone to think because we know one person that this has happened to, that that’s, that’s what happens. And that’s not what happens. In order to, in order to make a recommendation as to what people should do, we have to have good evidence to, to say, “Well, this is what people do. This is what the average person does.” And we can only do that in an unbiased way by using trial data. And part of that is that the two different groups of people have no other differences between them, other than one was randomised to something and one was randomised to something else. So I think the fact that you’ve got a group of people who are all starting off in the same place, and as large a group of people as possible, is really important. And that randomisation is just like flipping a coin really. You have no choice in it. It’s just one thing or the other. And that’s, the most important part of the trial is that there is no choice. If there was a choice, it wouldn’t be a randomised trial. So you might as well be going on hunches again. It’s because people have different views, and different people may have different views about one thing and choose something over something else, so.

Ann described randomisation as pot luck and trial and error.

Ann described randomisation as pot luck and trial and error.

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Age at interview: 43

Sex: Female

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Did they talk about all the randomisation and that and things like that?

Oh, yes, yes, yes, they did.

And what did you, what do you understand about that?

It was just basically that there’s different ways to treat it, and there’s no right way to treat. So if they can treat different people in different ways and then they can find out which is the happy medium. And, fingers crossed, they can use the one treatment for everybody. And hopefully they’ll find the right solution to it.

Did they explain that she might be in one group or another group and how that would work?

Yes, yes.

How did they explain that to you?

They just said that there was a couple of ways of treating it and they just randomly pick one. And, fingers crossed, it’ll work for Emily. And if it doesn’t, they’ll try something else.

And did you mind that, being randomised in to --?

No, no, because at the end of the day whatever treatment they try, it’s all trial and error, with any illness. You can’t say, “Right, this treatment will suit you and that treatment will suit that person.” It doesn’t always work that way. It’s all hit and miss.

What do those terms mean to you?What does randomisation mean to you, if you had to explain it, to another parent?

Pot luck. You just, you just, you just go, just go with the flow. At the end of the day if they randomly pick a treatment for you. All right, it might not be the best treatment for you. And they can change it. But it’s best if it gets, everything is trial and error.

Some parents we talked to had a preference for which group their children would be in, even though they knew they might not get their preference. Lisa’s son was born prematurely, weighing 2lb12oz. When he was 4 years old, she agreed to enrol him in a growth hormone trial in which he is still participating. She explains that the aim of the trial was to find the best dosage for different children.

Lisas son was allocated to the group with the highest dose of growth hormone. She thought the...

Lisas son was allocated to the group with the highest dose of growth hormone. She thought the...

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Age at interview: 37

Sex: Female

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With the growth hormones they have different dosage they give different children, and different strengths which they get on and then they look at which children have done differently on the strengths.

Did you know which strength you were on? What Callum was on?

We were told that he was on the highest strength because they had three different strengths and we just got told he was on the highest strength.

So you got, did you, did they talk about being randomised into?

I think they, like the first year he was on higher then the second year was something else, and then like the third year was I think like random. And he’s now gone on it because we’ve just re, because he’s done his three years, they’ve asked if we wanted to continue. So he’s now continuing and that’s to, the growth hormone to the level of what he’s missing, so he’s now on the dose which he is missing himself.

It’s just, I just usually say like the clinical trial bit is just like where they’re actually put, it’s a bit like number crunching, that they’re actually researching to find out more, to see what they can do to help other people and other children. So that they’re actually looking at each child, and then feeding in the information to see how they’ve developed, to see what they can do to make the medication and everything more effective overall to other people.

And if you’d have been put into a lower dose would, would you have minded? That would’ve been, you know, were you pleased to be in the higher dose, did it make a difference?

Well I thought the higher dose would have made a more difference, because for Callum being that he was so small, I thought the higher dose would give him a quicker boost to get him a bit bigger where he should be, and it has so,

But some parents might be on the lower dose?

And I suppose it all depends because with the children as well, they throughout, even if they are on whatever dose, a lower sort of thing, they’re always taking their growth hormone anyway. So, they do tweak within the dose limit’s as well because they have to be on a certain dosage to make it work so, I suppose even if you’re on the lower they would tweak with what your child, because if they’re not producing growth hormone they’ve got to have some to grow. So they wouldn’t be putting them on a dose which isn’t actually going to be any, not beneficial for them because whatever dose they are on it’s going to be beneficial. So they’re not going to give them doses what aren’t going to work.

Emma enrolled her son to a clinical trial to test different diets for children with epilepsy.

Emma understood why some parents would be upset to be in the control group.

Emma understood why some parents would be upset to be in the control group.

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Age at interview: 42

Sex: Female

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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.

But the families that came along after that were going on the clinical trial; I think they had a more difficult time. They were like, they had to go through things in more detail, and some of them were very disappointed that they went to the control group. And I could understand that, because they’ve got children who are very sick with seizures, that are having a lot, and they don’t want to wait another three months. You know, they’ve got in their heads, “It’s a new treatment we want to start it now.” So that was frustrating for some. And it was kind of coaching them through that. I was quite lucky.

With some of the parents that were frustrated about going into the control group, that, I was quite fortunate because I was able to chat with them on the phone and go, “Listen, this is important because this could affect the results, and this is why. You still will get the diet, but in the meantime prepare yourselves. Prepare,” you know.

And I basically had written up everything that I did before the diet. What I felt useful, what I’d not, and that’s what I put on the site, and that’s what I sort of coached parents through. So I was I was luckier than most because I knew what it would entail.

Yeah because as you say some of the other families I spoke to, they were in the control. And then some weren’t happy with the version of the diet they got. That was the other problem. Because they’d read a lot of literature, or, or the book that had come out from the Americans who don’t use the MCT version of the diet, and only had had bad experiences of it with earlier trials where children had had stomach problems and this kind of thing. But actually when you look closely it was because too much of the MCT was being given, they were giving it in too high a percentage. Whereas part of the trial, they were starting it off at a much lower percentage so you weren’t getting as many problems. Well not nearly as many problems.

So some of them had this fixed idea that the classical diet was the best diet, and that’s the one they wanted. So when they got randomised to the MCT version, again some of them got quite disappointed and I have to admit when Matthew got randomised to the classical, I was of that mind set as well and I thought, “Yes, I’ve got the classical, it’s the best one.” It wasn’t necessarily the best one, but it was just the one we knew the most about that had been used in America a lot. And I have to say after three years on the classical diet I changed Matthew over to the MCT diet and it was a doddle. It was really easy to use and Matthew loved it and it wasn’t a problem at all. And now I very much advocate both diets. And the MCT one is great for older kids and teenagers because it actually allows far more carbohydrates because of the different fats that you give, and how it’s metabolised.

In Emma’s case, children would be randomised to one of the two diets or a control group (though the children in the control group would also get one of the diets after 3 months).

Vicky and her daughter took part in a randomised trial on the management of diabetes in children and young people. Although they were happy to take part, she admits that they were both a little disappointed to be in the standard treatment group.

If you think there is a new and better treatment then you want it, but until they have done a...

If you think there is a new and better treatment then you want it, but until they have done a...

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Age at interview: 39

Sex: Female

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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, 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 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’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 worked out fine. And we’ve not seen any differences. So, you know, there’s not been any impact from that point of view. And it has just been answering the, the questions on general everyday, day-to-day stuff really.

Was, did your daughter understand that, that bit, you know, that she wasn’t getting something?

Yes, yes, and I think, well, I think she was, yes, she was disappointed, yes. Because, well, I don’t know what I imagine the new information to be like or the new way of delivering it. I don’t know. But, you know, you expect it to be all singing and dancing.

What is the standard information, how do they…?

Well, I don’t know, because that’s what, we’ve already had it. I mean we go to an appointment and we basically just have a conversation with the consultant and the diabetic nurse about how things have been going, and they pass on any information that is new that they’ve discovered. For example, there was a new book which is all to do with carb counting, which the diabetic nurse told us about. And so it’s just like that really, just sort of in a conversation style.

Vicky was a little disappointed her daughter was allocated to the standard treatment group.

Vicky was a little disappointed her daughter was allocated to the standard treatment group.

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Age at interview: 39

Sex: Female

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I don’t know whether they explained it. But I think, well, my understanding is that obviously they’ve got to pick some people who will get the new pack of information, or the information in a new way, and there have got to be some who, who continue with it the existing way, obviously to view what the results of the outcome of the study are. And I think the researcher went away and did something on a computer and it came up with a, with a magic number and, “Oh, right, you know, you’re going with the…” So I’m presuming that it was just a randomiser in that it just randomly chose whether we were going to be yes or no.

You were slightly disappointed that you didn’t get the –

Yes.

the new pack?

A lot of the terms used in trials are quite complex and closely related to each other. Below are some brief definitions as a reminder of how they fit together. You may like to look at the UK Clinical Research Collaboration booklet on Understanding Clinical Trials and chapter 3 of ‘Testing Treatments’

Control group – a comparison group in which people often get a standard treatment. If no standard treatment exists the control group receives no specific treatment or a Placebo. Comparing the results of people in the control group with people in the experimental group helps assess whether there are differences between the new treatment and the control treatment.

Randomisation – allocating people at random to one group or another, so that each group contains a similar mix of people. Random allocation helps ensure we are comparing two very similar groups of patients, so if one group does better than another, it is very likely to be because the treatments being compared have different effects, and not because of differences between the people in the groups.

Placebo – is a treatment with no active ingredient which is designed to appear very similar to the treatment being tested. By comparing people’s responses to the placebo and to the treatment being tested, researchers can tell whether the treatment provides any additional benefit.

Double blind – trying to ensure neither patients nor doctors or others treating you know which treatment each person is getting, so that this knowledge does not influence how patients feel or how doctors interact with their patients or interpret the results.

Last reviewed September 2018.
Last updated July 2015.

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