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Clinical trials are medical research studies that involve people. They are a vital step in improving care and treatment for people living with Waldenstrom’s macroglobulinaemia.
There are a number of clinical trials in the UK that involve people living with WM. Here we’ll discuss what clinical trials are and how you can get involved in them.
All treatments must be thoroughly tested before they can be used by healthcare professionals. First of all, researchers will test medicines in a laboratory and if the results from these tests are promising, they’ll start to carefully test them on people in clinical trials. This is an important step in developing new treatments, as it helps us to understand how the treatment behaves in people.
Clinical trials help researchers find out if a new treatment is safe and effective in treating a certain disease, including what side effects it might have, how it affects a person’s quality of life and whether it is more effective than the treatments that are already available.
Clinical trials also study things other than new treatments such as:
These clinical trials are just as important as discovering new treatments, as they help us to better our understanding of how WM works so researchers and doctors can find new treatments and improve care.
Not all clinical trials show that a new treatment or procedure is better than what is already used. This doesn’t mean a trial has failed, as it has given researchers and doctors important information about how best to treat a disease – something that will still benefit people living with it.
Clinical trials that look at treatments are split into different stages, called ‘phases’. These are numbered 1-4, with 1 being the very earliest stage of studying a treatment, and 4 being a trial that takes place after a treatment has been licensed.
Looks at how safe a treatment is. This will often involve testing a low dose of the treatment to see what dosage is safe, look for any side effects, and learn how the medicine reacts in the body (including if it treats the disease). Not all Phase 1 trials make it to Phase 2.
Usually, trials stick to one phase. Sometimes trials might straddle two or more phases.
When this happens, they will be referred to as Phase 1/2 or 2/3 etc.
The best place to start is to talk to your doctor about clinical trials. They’ll be able to tell you if there are any clinical trials taking in new participants, and if it’s right for you.
You don’t need to be out of treatment options to join a clinical trial. In fact, some clinical trials are not suitable for people who are at this stage. It’s always good to talk to your doctor about clinical trials whatever stage of treatment you’re at. When your multi-disciplinary team (MDT) meet to discuss your treatment, they will also talk about clinical trials and any ones that are open and suitable for you.
If you want to inform yourself about potential options, you can use clinical trial databases. Lymphoma Action and Cancer Research UK both have searchable databases that list clinical trials.
These tools can be useful to learn more about available clinical trials, but you usually have to be referred by your doctor to join them. You can print off any information you find to discuss it with your doctor.
Not everyone can join a clinical trial. Each trial will have its own set of entry requirements, called eligibility criteria. Everyone who takes part must meet these criteria, so that researchers can be sure the participants are as similar as possible, and any differences in results are down to the differences on the trial and nothing else.
Criteria can include:
These criteria are very strict. Your doctor will be able to use your records and blood test results to tell you if you meet the criteria. Unfortunately, if you don’t meet them, there’s nothing you or your doctor can do to get you onto the trial. This can be difficult to hear, especially if you have been told you don’t have any other treatment options. You can find more about what happens if you don’t have any treatment options left, and how to find support here.
If you meet the eligibility criteria, you can then join the trial. The process to join a clinical trial can take a long time and is usually managed by a research nurse or doctor. They will guide you through the process, giving you information about what the trial involves, what you can expect as a participant and any risks or side effects of being on the trial.
Being a part of a clinical trial is slightly different to having normal treatment. Here are some things you might want to consider or talk to your healthcare team about before joining a trial:
Access to experts
The researchers and healthcare professionals carrying out clinical trials will have all the most up to date information about Waldenstrom’s macroglobulinaemia and people in your situation, meaning you might have access to more expert information and care than at your normal hospital.
Good medical care
Clinical trials often look at side effects and the way the treatment is behaving in your body, which leads to closer monitoring and a longer follow up time. This could reassure you that you are being well looked after, although it may mean more visits to hospital.
Travel
Not all trials run in every hospital, and you might find you need to go to a different hospital than usual. This could mean travelling longer distances. On top of this, you might find you’ll need to visit the hospital more times than normal.
Additional monitoring
Most Waldenstrom’s macroglobulinaemia trials involve more tests than you may be used to, including CT scans and bone marrow samples, alongside blood tests. These are often part of a screening process that happens before you join the trial, and during the trial itself. Many trials will have periods of follow-up, so that researchers can continue to monitor how you’ve responded to treatment.
Accessing newest treatment
Some clinical trials are studying brand new treatments that you wouldn’t be able to access anywhere else. This can be exciting for some people, especially if you have run out of treatment options. It might worry other people, who are maybe nervous about trying out a new medicine for the first time. Remember that the medicine has already gone through rigorous testing to get this far.
Randomisation
Some trials will be comparing a new treatment against the best standard treatment. This means a portion of the trial participants will be given the standard treatment and not the new treatment. You won’t be able to choose which group you are put in. Some trials will use randomisation if they are comparing treatments. This is when a computer randomly selects participants and puts them in groups. Although it can be disappointing to learn that you’re in the standard treatment group, remember that a new treatment doesn’t necessarily mean it will be better and you will still receive excellent care.
Blinded trials and placebos
Sometimes a trial will be ‘blinded’ so you and even your doctor won’t know what group you are in. This is done to make sure that the results of the trial aren’t affected by participants thinking they’re taking new medication. Trials may also use placebos. These are ‘fake’ treatments that have no active medicine in them. Placebos are used for the same reasons as blinded trials. Both blinded trials and placebos can add an element of uncertainty to taking part, and some people find not knowing what treatment they’re taking can make them anxious. Remember that no matter how you’re participating in a trial, you will receive a very high standard of care and there is no certainty that one treatment is better than another.
Risks
All clinical trials come with risks, as they are set up to answer questions about a treatment’s safety, side effects, and effectiveness at treating a certain disease. Although researchers will have ideas, even they don’t know for sure how the trial will go. For example, trials may be halted if treatment has side effects that researchers didn’t expect. Your research nurse or doctor will be able to talk through the risks for the trial you’re joining, and you can ask them questions or share concerns at any time. Phase 1 trials carry the most risk than in other stages, as researchers know less about how the treatment will work in people.
Helping others
Being a part of a trial doesn’t just potentially help you, it helps everyone affected by Waldenstrom’s macroglobulinaemia. Current treatments simply wouldn’t be available if it wasn’t for people like you volunteering for trials in the past. Even if the trial doesn’t benefit you, the questions it helps researchers answer will benefit a great many people with WM in the future.
You can find clinical trials for people living with WM on clinical trial database like Lymphoma Action’s Trials and Cancer Research UK’s database
Clinicaltrials.gov can also be a useful resource, although it is international so you might not be able to join some of the trials you find.
The number of trials available is always changing, as some will close to applications as others open
