Clinical trials & medical research (young people)
Different types of trials
Clinical trials cover a broad range of different types of research and are carried out in a number of stages - see our introductory explanation in ‘Why do we have clinical trials in children and young people?’ Here we focus on the different types of treatments and interventions which can be involved.
Examples of how clinical trials can help:
• Prevent illness by testing a vaccine
• Detect or diagnose illnesses by testing a scan or blood test
• Treat illness by testing a new medicine
• Find out how best to provide psychological support
• Find out how people can control symptoms or improve their quality of life by testing how a particular diet affects a condition
Preventive trials' look for better ways to prevent disease in people who have never had the disease or to prevent a disease from returning. These approaches may include medicines, vitamins, vaccines, minerals, or lifestyle changes.
Sophie, aged 23, explained why she wanted to take part in a research to help prevent the onset of diabetes in people with cystic fibrosis. Ruby and Joanna talked about taking part in trials to find the best treatment to help prevent thinning of bones in people who take steroids.
Sophie took part in research to help stop people with cystic fibrosis developing diabetes. She says it is really important to detect changes in blood sugar levels early.
So now that I’m a patient at [Hospital] and all my care is overseen by the specialist team there. One of the doctors asked if I would be interested in taking part in some research about blood sugar levels in people with cystic fibrosis. I’m in a position where hopefully I won’t develop diabetes because of the nature of my condition. I’m actually pancreatic sufficient but there is a chance that if I do become insufficient that I can develop diabetes. But in people with cystic fibrosis that are pancreatic insufficient, which is 90% of patients, the blood sugar levels can fluctuate and can lead to developing diabetes. And they’ve found that when a person’s having problems with the blood sugar levels, like when they’re too high or when they’re too low, it can affect their whole their health in general so it can affect their chest it can affect their lung function and it can affect their weight and it can affect many things. So it’s really important to be able to monitor blood sugar levels carefully and so people are receiving the right treatment. There is a test called the Oral Glucose Tolerance Test which is used commonly to, throughout the World, to diagnose diabetes. But this wasn’t developed with CF patients in mind it was developed in, for general diabetes, whereas CF related diabetes is slightly different. So, they’ve been looking at other ways of how to detect changes in blood sugar levels particularly in people with cystic fibrosis.
And something has been developed to help monitor blood sugar levels more carefully and it’s called a Continual Blood Glucose Monitor System. And the study which I took part in required me to wear this system it’s just like a little monitor for four days. So I was told briefly about it and I was given an information sheet explaining about the study which I read and I could ask any questions that I liked.
Ruby took part in a randomised placebo controlled trial on finding the best treatment to prevent...
The trial will compare calcium with the trial drugs to see which one works. Joanna is hoping that...
Both Will and Buddy took part in vaccine trials. Will took part in a swine flu vaccine trial and says “They were just seeing which one was the best on that they could use out on the public and stuff.” Buddy took part in a meningitis C vaccine trial and says' “It wasn’t really too bad. Because she came and talked to me about like what she was doing and it didn’t hurt. I had one vaccination and they took some blood”.
Drug trials' may be testing whether a new drug has any major side effects, or whether it works better than an existing treatment; but they may also test timing (when or how often to give a drug) or dosage (how much of the drug is needed to be effective). Drug trials are probably the most familiar type of trial to many people. Trials can also be used to test whether giving a treatment in a different way will make it more effective or reduce any side effects.
Lois is taking part in a randomised trial on improving the treatment for Grave’s disease, which usually affects women but is rare in teenage girls and even rarer in males. Grave’s disease means that Lois has an overactive thyroid gland and she has to take daily medication to help control it.
Taking part in a trial made no difference to Lois, she was taking medication anyway. She is...
Lauren took part in a randomised trial on the side effects of a background insulin. She didnt...
Alexander understands that the trial was to find out any side effects of the drug. He has...
Graham is taking part in a clinical trial on the treatment of children and young people with Hodgkin’s lymphoma. Graham was unsure of the purpose of the trial but understood that it was to find out if treatment is just as successful by replacing one drug with another drug that is less likely to cause infertility or early menopause, and to find out what the long term effects are of each drug on fertility.
Taking part in the trial didnt seem any different to the normal treatment says Graham.
To take part in a Phase 2 trial that is at the forefront of medical science is really exciting...
Yes well the different trials are, have different purposes, so all the ones, so the one I did where I ended up on a placebo that was the double blind one to see, that’s were “Oh we know this drug is safe we just want to see if it has benefit”. Whereas the pilot study was specifically to see is the delivery of this drug safe, are there side effects how efficient does it seem to be and that. So that was on a smaller group of people and so from that point of view was reasonably, it was quite ground breaking actually and it was, it was exciting to take part in something that was virtually the forefront of medical science. Because it was using a different delivery for the gene therapy rather than a viral vector which most gene therapy uses it, it was it was using a different delivery mechanism which might mean it’s viable for multiple doses.
Dr William vant Hoff says ethics of research in children is being done with the purpose to...
Nearly all medicines are tested in adults before 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 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 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, a better, a better set of therapies for children.
Not all randomised trials are drug trials. They may also be testing other types of care, such as different levels of monitoring, the effect of different types of diet, or the effectiveness of different forms of screening e.g.
Screening trials: detect or diagnose illnesses, for example, by using a scan or blood test.
Diagnostic trials: conducted to find better tests or procedures for diagnosing a particular disease or condition.
There is also growing interest in testing different ways of giving people health information, to see which is most helpful to them in making decisions or understanding and managing their condition.
Information and quality of life interventions' to help people learn about their condition and help them to self-manage their health better and improve comfort and quality of life. Interventions may include finding the best approaches to help people control their symptoms or improve their quality of life by testing how a particular diet affects a condition.
Sophie (aged 12) was randomised to take part in a one day trial to develop an intervention for...
Joe took part in a trial to assess home versus hospital management at diagnosis of childhood...
Well, they explained what they were going to do in the trial. They explained like about diabetes. And they said, “The trial is just to see how you cope with like, with diabetes.” And in hospital I think it was better because if I needed any, if I had any questions I could have asked them and stuff. But I think if it, if I had the trial at home I would have been able to like, you know, deal with it on my own more, instead of like asking people.
So was there a choice?
No. The organisers, they choose for you.
They choose for you? So you could you have gone home at all?
could have gone home. But I think it’s like they randomly choose people to stay in hospital or go home. And I was chosen to stay in hospital.
I was going to say, I was wondering whether you were randomised. And did they mention about the, explain about the, that grouping in those groups to you? Why they were having those?
Yes, they said, they mentioned that they wanted to see how people dealt with it in different like situations. Like when I was in hospital, if like, you know, at home, to see how people coped with it more. If you got put in hospital it was just for the doctors to help you really and stuff.
And how did that feel, you know, when you were, you sort of, you were diagnosed and then you were, then you were asked to go in to a trial? How did you feel at that time? Because it must have been a bit overwhelming?
Yes, I was really overwhelmed, I was shocked. And my mum was shocked as well. And it was it was upsetting when I first found that out I had diabetes. But then I thought, “Well, it’s like, it’s a speed bump and not a stop sign. So it’s not going to stop me.” But then the trial, I just, I felt happy to be in the trial because it’s to help me and to help other people, research and stuff. So, yes, I was happy I got put in hospital though, because I was a bit shocked at the time.
And, like you say, one of the reasons you wanted to take part was to help yourself. In what way do you think it was going to help you and help research?
I think it would just help me to understand diabetes and like how to deal with it more, because I was really shocked when I found out. And it did, the research is, because I’ve been through the, you know, the shock of having diabetes, and when other people get diagnosed it’s just like unexpected and stuff. And the research will help people deal with it I think.
Other types of trials can include those that find the best surgical interventions and trials which compare the frequency or intensity of a treatment, such as radiotherapy regimens and even Psychological therapies' which help to find out how best to provide psychological support.
Some young people we interviewed were invited to take part in non-randomised trials - see ‘Other types of medical research’. These types of trials are also important in improving treatments and advancing knowledge.
Last reviewed March 2017.
Last updated July 2014.