Clinical trials are a core part of the way care is delivered for children with cancer, and are often international because of the small numbers involved. Treatment follows specifically designed protocols or, in a small number of cases where there is no clinical trial, a national UK guideline.
We are still learning the best ways to treat childhood cancer and clinical trials allow us to test new treatments and ways of controlling symptoms, or to investigate new ways of preventing or diagnosing cancer. It is largely because of clinical trials that such outstanding progress has been made in the treatment of children's cancer over the last few decades.
Your child's doctor will be aware of all of the clinical trials available and whether your child is eligible to join one. If your child joins a trial, they will receive their treatment at their designated hospital as normal.
There are numerous research studies and clinical trials open at any one time and each one has its own aims.
For example:
- test new treatments, such as new chemotherapy drugs
- look at new combinations of existing treatments, or change the way they’re given, to make them more effective or reduce side effects
- compare the effectiveness of drugs used to control symptoms
- find out how cancer treatments work
- look at improving the quality of life and services for children and young people who have been treated for cancer
- look at the biology of cancer and its treatment
- find out which treatments are the most cost-effective
Trials are the only reliable way to find out if a different type of surgery, chemotherapy, radiotherapy or other treatment is better than what is already available.
There are three different kinds (known as phases) of trial. Each phase aims to find out something different about the new treatment or procedure.
Phase I trials
These test possible new treatments on people for the first time and is the earliest stage of testing new drugs on humans. Any new drugs will already have been tested in the laboratory or in animals. These trials are carried out in a very small number of patients, usually those with advanced cancer and who have had all available standard treatment.
They help us to find the correct dose and look at any possible side effects.
Phase II trials
These trials test to see whether a treatment is likely to be effective at the dose(s) chosen. They are not always about introducing a new drug but sometimes testing whether an existing therapy can be given in a different way to be more effective. They will again involve a relatively small number of patients.
Phase III trials
If the results from Phase I and Phase II trials suggest a drug or treatment is both safe and effective, it will then be compared with the current or 'standard' treatment in a Phase III trial. These trials involve larger number of patients and usually run for much longer that Phase I and II trials. As the number of children with cancer is relatively small, there is often strong international collaboration with overseas research organisations so children across many countries can take part in a clinical trial.
Your child's medical team will talk to you about taking part in a clinical trial and will answer any questions you have.
Taking part is completely voluntary and you will be given plenty of time to decide if it is right for your child. You can also join a trial, and then withdraw if you change your mind at a later date.
Is it safe?
The safety of patients in clinical trials is of the utmost importance. All trial protocols are reviewed and approved by ethics and regulatory committees. All trials are reviewed on an ongoing basis. If there are any concerns about the safety or efffectiveness of the treatment, the trial may be stopped. It is very rare that this would happen.
What are the benefits of taking part in a clinical trial?
- Your child may receive a new treatment that is only available in a trial
- Your treatment will be the same wherever you live in the country
- Experts in your child's particular tumour type will have worked together to develop the trial protocol
- Emphasis on patient safety and your child will be monitored closely
- Sometimes there may be no direct benefit for you or your child but the results of the trial may help doctors to improve cancer treatments for future patients.
What are the disadvantages of taking part in a clinical trial?
- You may have to make more hospital visits
- You may have more tests carried out
- The new treatment, although expected to be better, may not actually be better
- You may experience side effects that you or your doctor are not expecting, but you will be closely monitored for these.
Who runs the trials?
The majority of clinical trials for childhood cancer in the UK are coordinated by a specialist Cancer Research UK Clinical Trials Unit at the University of Birmingham. The UK has an early phase trials paediatric network via the Experimental Cancer Medicine Centre Network (ECMC). Early phase trials may not be opened in all main hospitals so your child may need to travel to access specific trials.
What if my child cannot join a clinical trial?
Sometimes, clinical trials are not available for your child's tumour. This may be because a recent trial has just finished, or because the tumour is very rare. In these cases, you can expect your doctors and nurses to offer treatment which is agreed to be the most appropriate, using guidelines which have been prepared by experts across the country. CCLG is an important organisation which helps to produce these guidelines.
