Clinical trials are research studies involving people. They test whether particular treatments are safe and how well they work. We need to know:
- Does a treatment work?
- Does it work better than other treatments?
- Does it have any side effects?
Clinical trials are designed to answer these questions to improve the health and quality of life for patients, including children. Until well-designed trials have been carried out, we simply do not have enough evidence to know if a treatment is both effective and safe. Without trials, there is a risk that people will be given treatments which do not work and which may be harmful.
Experimenting and testing have long been a part of medicine, and there are many different types of trial. (See below for an explanation of Phase 1, Phase 2, Phase 3, and randomised trials.) Randomised trials are generally recognised as the best way to compare different approaches to preventing and treating illness. They can be used to compare drugs and other types of treatment across a range of conditions.
Dr William van’t Hoff was co-director of medicines for Children Research Network in 2011. William talks about the importance of clinical trials in children and young people, the different phases of research, and the ethics of conducting research in children.
Clinical research in children is essential in order to improve the treatment of children.
It is important that medicines for children are carefully checked within the right age group that…
Children and young people may need different treatments from adults because they are at a different stage of development. Clinical trials in children are therefore essential to ensure they receive appropriate, safe, and effective treatments and care. There are guidelines to protect children who take part in clinical trials of medicines in the UK and throughout the European Union (EU).
Dr William vant Hoff says ethics of research in children is being done with the purpose to…
Helena, in her role as a senior research nurse, ensures parents understand what a clinical trial…
Phase 1 and Phase 2 trials
Clinical trials are carried out in several stages, or phases. When a new treatment is developed, such as a new cancer drug, it will be tried first in a few people to get an idea of how safe it is. They may be healthy volunteers, who are given a compensation payment for taking part, or they may be people who are ill, perhaps people who have already tried all the usual treatments. This is called a Phase 1 trial. At this stage the treatment is usually given to all those taking part and is not being compared against other treatments.
Alisons son has completed a Phase 1 trial on gene therapy for cystic fibrosis.
At Phase 2 the researchers will know more about the treatment. The aim of a Phase 2 trial is to test the new treatment, such as a drug, in a larger group of people to better measure safety and side effects and see if there are signs of positive effects in patients. A Phase 2 trial may or may not involve comparison with another treatment. However, the numbers of people included are still too small to give firm evidence of whether the treatment works or that any change is not just happening by chance. This is why Phase 3 trials are needed.
Phase 3, randomised trials
Phase 3 trials are usually large. They include hundreds or even thousands of patients. They often compare the effects of new treatments or drugs with standard treatments, if there are any. They provide more reliable evidence about whether newer treatments are better, or worse than existing treatments, and firmer evidence about how common and serious any short-term side effects are. Almost all Phase 3 trials are randomised trials. In a randomised trial, some people are given the new treatment; others are given an existing, standard treatment. If there is no standard treatment, the new treatment may be compared with a placebo. Some randomised trials may compare more than two groups, and some may be blind.
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.
Jo’s son is taking part in a randomised placebo-controlled trial that is double-blind. Neither Jo nor the doctors or others treating her son know which drug he is taking.
Jos son is taking part in a randomised placebo controlled trial of a drug for migraine in…
As Helena says, information given to parents about a trial should include an explanation of all…
Most medical advances, improved treatments and care have been possible because adults and children have participated in clinical trials.
Advances in neonatal care have been possible thanks to the parents and their babies who have…
Advances in medical care for children have resulted from children participating in clinical trials.
Alison talks about the improvements in the care and treatment of cystic fibrosis. Alison acknowledges that although her son may not receive any immediate benefit from participating in trials, she is hopeful that the results will be of benefit to him and others with cystic fibrosis in the future.
Clinical trials have helped to improve the treatment for cystic fibrosis, but this often takes a…
Clinical trials are carried out when we do not know whether a treatment works, or which treatment works best. Sometimes a clinical trial may show that a drug or other treatment has little benefit, and this can be valuable information. Alison talks about a trial her son took part in to test the use of antibiotics to reduce sepsis in very premature babies.
The trial was unable to show any beneficial effect. This is still an important finding for the…
Childrens research has made a difference in improving their health, treatment and care.
Emma talks about what she has learned from her experience in supporting her son through a clinical trial. Emma feels that doctors sometimes criticise interventions like diets for not being evidence-based yet themselves use treatments that are not evidence-based. Emma enrolled her son in a randomised trial to test two versions of the ketogenic diet, for epilepsy. Emma is dedicated to supporting medical research into the causes and cures for epilepsies.
More research is needed into diets for children with epilepsy as well as research into the causes…
The National Institute for Health provides a research support structure to help families and…
Kathryn’s daughter took part in a randomised trial on managing diabetes in young people with diabetes. She feels it is important that children and young people participate in trials so that their feelings and views are heard. She says, ‘I think sometimes with the younger end, they don’t actually have a voice sometimes. They don’t feel like they can actually say how they feel in a lot of circumstances, in situations really. So it’s good that they’re actually asking these kids how they feel about things.’
Clinical trials are important to help improve understanding of how treatments work and how people…
You can find more information on clinical trials in resources.