Clinical trials

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.

What are clinical trials?

Clinical trial phases

How can I join a trial?

What can I expect?

What clinical trials are available?

What are
clinical trials?

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:

Finding better or faster ways to diagnose a disease
Comparing existing treatments to see which work best
Looking at how a disease develops and progresses
Studying existing treatments to see how they affect people in the long term

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 trial phases

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.

Why not start treatment straightaway?

When you’re first diagnosed with WM, being told you don’t need immediate treatment may surprise, or even worry, you. You might think that it must be better to kill the cancer cells before they can increase more. However active monitoring is a safe and recommended way to care for people with slow-growing cancers like WM. There isn’t any evidence to show that being treated earlier has any benefits. Treatment itself can have some harsh side effects, which can be long lasting. By putting you on active monitoring, your doctor is saving treatment for when it can have the maximum impact. If your doctor sees signs that your WM is worsening, they will discuss treatment options with you immediately. However, many people stay on active monitoring for years, meaning they can lead full lives without the side effects of needless treatment.
What happens on active monitoring?

You’ll have regular check-ups with your healthcare team, where you’ll have blood tests and a chance to talk about how you’re feeling and any symptoms or concerns you have. Normally these are face-to-face at the hospital, but since the COVID-19 pandemic, many check-ups have moved to video call or telephone. This doesn’t affect how you are monitored, just the way you speak to your healthcare team. Your blood tests and symptoms will help your doctor understand how your WM is behaving, and whether it’s time to start treatment or not. The check-ups also mean your doctor can pick up on any other conditions, for example anaemia, and ensure you get the right treatment for this. It’s important to raise concerns or mention any symptoms, however minor, at your check-up as your healthcare team might want to investigate these further. When you’re first diagnosed, these check-ups may be more frequent – for example, every 3 months – but as time goes by and if your WM remains stable, the check-ups may become less frequent. Some people who have been on active monitoring for years might have one check-up a year.
Is active monitoring safe?

Yes. It is the recommended way to care for people with WM that isn’t growing and who either don’t have symptoms or whose symptoms are mild.

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.

Can I join a trial?

Female doctor talking with a male patient at a hospital

How can I join a
clinical trial?

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:

Type of cancer – some trials will include people with different types of cancer, whereas others will only accept people with one type of cancer e.g. WM
Stage of cancer – this refers to how far your WM has progressed
Previous treatment – some trials might be looking for people who have no previous treatment, or people who have or have not already had a specific type of treatment
Age – this is often quite broad (e.g. over 18 years old) but researchers can make it more specific
General health – researchers will measure your general health on a scale of 0 (fully active) to 4 (in a bed or chair all the time). Most trials will be looking for people with a certain number on this scale
Other medical conditions – any other conditions you have may affect the results of the trial or might mean that the treatment being tested isn’t suitable for you
Life expectancy – some trials will only accept people with a longer life expectancy. This is because they will be looking into longer term effects of the treatment

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.

What can I expect?

What can I expect when taking
part in a clinical trial?

Why not start treatment straightaway?

When you’re first diagnosed with WM, being told you don’t need immediate treatment may surprise, or even worry, you. You might think that it must be better to kill the cancer cells before they can increase more. However active monitoring is a safe and recommended way to care for people with slow-growing cancers like WM. There isn’t any evidence to show that being treated earlier has any benefits. Treatment itself can have some harsh side effects, which can be long lasting. By putting you on active monitoring, your doctor is saving treatment for when it can have the maximum impact. If your doctor sees signs that your WM is worsening, they will discuss treatment options with you immediately. However, many people stay on active monitoring for years, meaning they can lead full lives without the side effects of needless treatment.
What happens on active monitoring?

You’ll have regular check-ups with your healthcare team, where you’ll have blood tests and a chance to talk about how you’re feeling and any symptoms or concerns you have. Normally these are face-to-face at the hospital, but since the COVID-19 pandemic, many check-ups have moved to video call or telephone. This doesn’t affect how you are monitored, just the way you speak to your healthcare team. Your blood tests and symptoms will help your doctor understand how your WM is behaving, and whether it’s time to start treatment or not. The check-ups also mean your doctor can pick up on any other conditions, for example anaemia, and ensure you get the right treatment for this. It’s important to raise concerns or mention any symptoms, however minor, at your check-up as your healthcare team might want to investigate these further. When you’re first diagnosed, these check-ups may be more frequent – for example, every 3 months – but as time goes by and if your WM remains stable, the check-ups may become less frequent. Some people who have been on active monitoring for years might have one check-up a year.
Is active monitoring safe?

Yes. It is the recommended way to care for people with WM that isn’t growing and who either don’t have symptoms or whose symptoms are mild.

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: