What are clinical trials?
Clinical trials test new treatments or approaches to see whether they are safe to use and effective. These treatments will have been developed in a laboratory before they start being trialled with patients. Most treatments have to go through a number of different trials before they are judged to be safe and effective and to be used as a regular treatment for patients. Most of the treatments and medicines you get from a doctor or a pharmacy will have been developed through this process.
What are early stage or Phase I trials?
Early stage trials are also called Phase I trials and these are the first step in testing new treatments in patients. These phase I trials are usually testing experimental treatments, that work in the laboratory but it’s not yet known if they will work in patients. These trials look first at whether a treatment is safe, but also for signs that it might be effective in patients in the future. Phase I trials are usually small (often less than 30 patients), but because we treat patients one by one and observe them closely they can take one or two years to complete. These trials are usually only done in specialist centres like NIHR Leeds CRF, and each centre has to be ‘approved’ to do each individual trial.
What are the other stages of trials?
If a Phase I trial shows that a treatment is safe to use and may be effective, the treatment will then go into larger Phase II trials, involving more patients, which will look more closely at whether it does work as intended for the disease in question.
If the Phase II trial confirms a treatment is safe and effective we need to test whether it is better than the current standard treatment. This is done in a Phase III trial in which patients are allocated to receive either the new treatment or the current standard treatment. These Phase III trials often involve many hundreds of patients and last for several years.
Many Phase II and Phase III trials do not need to be carried out in a CRF. Often, however, these trials include additional blood tests or special tests, such as scans, to check whether a treatment is working. These extra tests will often be organised or carried out through the CRF.
Are there risks in taking part?
Each clinical trial is designed to reduce or eliminate any risk to patients and this is carefully checked by the body that has to approve the trial before it goes ahead, called an Ethics Committee. Every trial is different and so the risks for each one will vary. In general, as a new treatment progresses through Phase I to Phase II then Phase III trials experience with the treatment increases and our understanding of possible risk improves. Any potential risks will be explained to you in advance by the CRF team, so you can understand these fully before you decide if you want to take part.
Our doctors and research nurses monitor all the patients who take part in our trials very carefully to detect any side effects and changes. If anything happens that isn’t expected, our medical staff will act quickly. Our research facilities are based within the various sites of Leeds Teaching Hospitals, to ensure that if patients taking part in our trials need any other kind of medical attention, they will get that attention quickly and easily.
If you are taking part in a clinical trial, you should tell us about any illnesses or changes in your body that you notice, as these may be linked to the treatment you’re receiving.
Will I see any benefits?
Most of the treatments we are testing in Phase I trials at our facility are still at a very early stage of development and have not yet been proven to work. So there is no guarantee that they will have a positive effect on the disease for which you are being treated.
Treatments being tested in Phase II and Phase III trials will have already shown signs of working in patients, so those taking part in such trials may well benefit.
However, when you take part in a research study, you have more contact with medical staff than you normally do and there will be opportunities to gain information about your medical condition, which may help you to manage it better. Although you are likely to spend longer in hospital than if you were simply receiving routine treatment, many patients find this additional, one-to-one care reassuring.
Although the research might not help you personally, it may provide vital information that will help people in the future. Many patients feel a sense of satisfaction from taking part in our trials, because they feel they are doing something important to help others dealing with the same disease.
What does taking part in a trial involve?
What you will be asked to do, and the time involved, will depend on what is being investigated in the trial. Every trial is different, but our team will give you all the information you need to make an informed choice as to whether to take part, including how long the study is expected to last and what will be expected of you.
Most of our trials only involve out-patient treatment, which means you’ll come to one of our research clinics to be treated, before going home the same day. For some trials, your treatment may occasionally need to go on for longer, which might involve an overnight stay.
We’ll talk through with you how many visits might be needed, how long they might be and help you with travel and accommodation arrangements. Leeds Teaching Hospitals has a hotel at St James’s, for patients and their families who are undergoing treatment, including those taking part in clinical trials. Parking charges are also refunded for patients taking part in clinical trials.
Before you take part in a trial, you will be asked to sign a consent form, to show that you understand what is being asked of you and to confirm you agree to take part. You should not give your consent if you are unclear about any aspect of the trial and what is involved. Don’t be afraid to ask as many questions as you need to.
What questions should I ask?
Anything and everything! Never feel embarrassed to ask questions, as we want to make sure you fully understand all aspects of the trial and what it involves before you sign the consent form to take part.
First of all, we would encourage you to ask the doctors and nurses looking after you whether there are any clinical trials you might be able to take part in. You can also look for clinical trials on the internet and through the charities that support research.
Once you find a possible trial, there are some questions below that you might want to ask:
- What is the aim of the study?
- How/why was I chosen?
- Does the research involve collecting samples of my cells or tissues? If yes, what will happen with them and can they be used for other research projects?
- How long will the research take and what will I have to do?
- What will be different to the treatment or care I would normally receive?
- If you are testing a new drug, how many people have taken it before?
- Are there any possible side effects?
- What happens if something goes wrong?
- Who can I talk to if I have any questions, concerns or problems?
- Will I need to take time off work?
Do you provide financial support?
Taking part in a trial is voluntary, so we don’t usually pay patients for their time. However, taking part in a trial is likely to involve more visits to hospital than would be the case with normal treatment, so we do help with these extra costs. We can reimburse your travel costs, including parking charges. We can also help with accommodation costs for patients who require an overnight stay for treatment on consecutive days.
What if I change my mind?
Before you go onto a trial you will be given spoken and written information, have time to think about what you have been told and speak with friends and family as well as being given the chance to ask questions. If you agree, you will then sign an informed consent form.
If at any time before you go on the trial, or whilst on the trial, you have doubts or questions please speak to the research team. If you want to come off the trial you are entirely free to do so. Just say you would like to you stop; you don’t need to say why.
Are paediatric clinical trials available?
Yes – we work closely with the Leeds Children’s Hospital on paediatric clinical trials. To help us explain this work and design better trials, we have two patient research ambassadors and we’re also working with the Children’s Hospital Youth Forum. Although these trials cover different phases of research, it’s important to understand that a Phase I trial in children is very different from a Phase I trial in adults since the drugs have usually been used in adult trials previously.