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Understanding Clinical Trials: 10 Questions

IKCC Trials 1

All cancer treatments available today have been proven in clinical trials. Clinical trials are research studies that test the idea that a new treatment or other healthcare intervention is better than the existing drug or practice. While it may be natural to assume that the new treatment is better, this may not be the case. It may be worse in terms of its effect on the cancer, its side effects, costs, or inconvenience to you. On the other hand, it might truly be better and become established as the new standard of care.

When patients take part in clinical trials, there is always some uncertainty as to whether the new treatment is better or worse than the standard therapy. Clinical trials are the only way forward to improve cancer treatment. For patients, joining a clinical trial is a big decision and there are several factors you need to think about as you make this decision.

This section explains how trials are run, how you might take part in a clinical trial and how a trial might help you.  

 

Question 1. What is a clinical trial?

A clinical trial is an experiment that tests an idea. It could be an idea about how to give a treatment, for example, if a new treatment is safe or how well it works. The treatment being tested can be a drug, a new way of giving radiation or performing an operation, or something as simple as a different way of caring for patients. Sometimes the clinical trial tests a combination of drugs or treatments that have not been tested before to see if two treatments combined are better than one.

At any time there are many trials testing new cancer treatments occurring all around the world. These trials cover many different types of cancer and include thousands of patients. All of the drugs we can use to treat kidney cancer have been tested in clinical trials at some point. In kidney cancer, researchers are also working to determine which treatments work best for which patients.

Every new drug has to be rigorously tested and must pass through pre-clinical research and several “phases” of clinical trials before it can be prescribed to patients. This process can take many years from start to finish.

 

Question 2. Why can’t I just take the new treatment?

We all want the best possible treatment for our family and ourselves. When a “new” medication is mentioned in the newspaper, or on the TV, we take interest, especially if someone we love is currently living with cancer. More generally we all hope that better treatment options might be available to us should the need ever arise. One question that people often ask is “why can’t I just take the new treatment?”

What treatment is offered in a clinical trial depends on the phase of the clinical trial, and the design of the trial. In phase I and some phase II trials, the new treatment is usually the only one given. In some phase II trials and almost all phase III trials, you might be “randomised” to take either the standard treatment or the new treatment.

Why do clinical trials randomise treatments?

“Randomised” means that once you choose to take part in the trial, and the team caring for you has checked that you fit all the criteria required for the trial, neither you nor your doctor has a choice of what treatment you take. While not as crude as flipping a coin, a computer randomly assigns patients to the different treatments being tested.

This can be very frustrating, as people hope that they will get the new medication rather than the standard, but sometimes only half of patients will “win” this bet. This has been particularly challenging as the immuno-oncology drugs offer a treatment with a completely different mode of action to standard treatments like chemotherapy and targeted therapies, and the very real hope is that even if these standard treatments have failed, then immuno-oncology drugs might succeed.

Will I get the new treatment either way? (Is there cross-over?)

Some well-designed trials allow patients to “cross-over” to the new treatment from the standard treatment if/when it fails. This allows the drug to be well tested, but also offers every patient the opportunity to benefit from the novel therapy.

If the trial does not allow cross-over, and you could take the standard treatment elsewhere, then there are still good reasons to stay on the trial. First and foremost your participation will serve to prove the benefit (or not) of the new treatment versus the standard treatment. Every treatment we have ever been able to prove works for patients has needed brave and generous people to remain on trials.

What if I don't receive the newer treatment?

While the quality of care provided by your doctor will be the highest they can deliver within your health care system, patients on clinical trials routinely experience an even higher quality of care than patients not on clinical trials. This is likely because they have the doctors and nurses watching them so closely, that problems get discovered and addressed even faster than in people not on clinical trials.

Question 3. Is a clinical trial right for me?

A clinical trial can be a very good option for treatment if you are suffering cancer. The most obvious reason for considering a clinical trial is obviously to find a treatment that will be best for you. Many patients join a trial hoping that the treatment they can get on a trial is better than the standard that is currently available. However it’s important to remember that you may or may not benefit personally from being part of a trial. You may not get the “new” treatment, or it may not work for you, or you may not tolerate whichever treatment you are assigned.

Is a clinical trial right for meYou can expect some things if you choose to take part in a clinical trial. Being treated on a clinical trial is likely to result in close observation and care from a highly skilled and specialized team. Your care taking the standard treatment on a clinical trial may indeed be better than in routine clinical practice. In addition, you will experience a great deal of personal satisfaction from having contributed to the body of human knowledge, of being one of the people that helps test new ideas for the treatment of cancer. Clinical trials are essential to find out more about diseases and how to treat them. So if you do take part in a trial, any information collected during the trial will inform decisions about the best way to treat future patients.

The decision to take part in a trial should not be taken lightly. Some people find the added information and complexity of a clinical trial too confusing, frightening or confronting. Some people don’t like the idea of their treatment being “randomly” imposed (though again, this is a coin-toss between “good treatment” and “hopefully even better treatment”; not win or lose). Some people would rather that their doctor or family decide what treatment is best for them. If you are offered a clinical trial, you do not have to take it. Your treatment will not suffer, your doctors will not disapprove. It is entirely your choice whether to participate.
 
If you are thinking of joining a trial, you can ask for the following information to help you decide.
  • An up-to-date assessment of your condition.
  • Detailed information about the trial, including what treatment is being tested and what taking part could mean for you.
  • What alternative treatments or trials are available.
If you would like more suggestions about what questions to ask your doctor before taking part in a trial, please click here.

Question 4. Am I right for a clinical trial?

It is not always possible to join a trial, even if you want to. When researchers design clinical trials, they usually have a strict set of rules to help them choose who can take part. For example, If a patient’s tumours are too small, it can make it hard to tell if the treatment is working or not. If patients are too sick then it may not be possible to safely give the treatment for fear of side effects.

You might not be eligible to take part in the trial if:
  • your general health is not good enough (you are too unwell)
  • you have had previous treatment for your kidney cancer that could affect the results of the trial
  • you have other diseases (e.g. diabetes mellitus or auto-immune diseases) which could make you more likely to have side-effects or affect the results of the trial
  • you have had another kind of cancer at some time in the past
During the “screening” period, the team will perform a number of tests to make sure you fit all the criteria for the trial. Sometimes, this testing can turn up new findings that mean you are no longer eligible for the trial.
 
If you are excluded from a trial, you should be told why by your doctor or one of the trial doctors.
Sometimes there are logistical reasons for you to be excluded from a trial (e.g. your country is not taking part). In rare cases, it might be possible for you to travel to a different country to take part. Your doctor should be able to help you explore this option.

Question 5. What are the benefits of taking part in a trial?

Benefits of a trialWhen deciding if you want to take part in a trial, it is important to realise that the new treatment might work for you, but it might not. Depending upon the trial, you might be randomised to a control arm to receive the standard treatment. When there are two arms of a trial, as the patient you cannot simply choose which treatment you would prefer.

Apart from access to the new treatment, there are other benefits to taking part in a trial.
  • You will be seen by doctors and nurses who are experts in treating your condition, in hospitals that have the best facilities.
  • The health of people who take part in clinical trials is usually monitored more closely than the health of patients who do not take part in trials.
  • You are making a valuable contribution to medical research.
  • You are taking control of the treatment of your illness.

Question 6. What are the risks of taking part in a clinical trial?

Risks of trialsThere are several risks to consider before joining a clinical trial.

The new treatment might cause unpredictable or serious side effects. In some cases, these can be permanent. This is particularly relevant if you want to join a trial that is the first to test a drug in humans (a phase I trial). Despite the hopes of the doctors running the trial, the new treatment might not work as well as the treatments that are already available, or it might not work at all for you.

There may be financial costs to consider too, like travel and accommodation costs, or the cost of you or a caregiver taking time off work to go to the trial clinic. You will not be paid money to take part in a clinical trial, and likewise while the trial should not place undue costs on you, it is very rare for the trial to reimburse any costs incurred. If you are worried about taking part in a clinical trial, you should talk to your doctor. To help you gather the facts you need to make your decision, we have written a list of questions to ask your doctor. These can be found here.

 

Question 7. Why can’t I get on to a clinical trial?

Why can't I get on a trialClinical trials need patients to take part to be successful. Unfortunately, the number of people taking part in trials is low in many countries. An American study found that about 1 in 3 trials close early because they cannot find enough people to take part. Conversely it can be hard to find a clinical trial that is useful to you.

There are many reasons why people are not able to take part in clinical trials. Some common reasons are:
  • no suitable trial is available
  • not being told about the trial by their doctor
  • worries about being given placebo treatment (placebos are rarely used in kidney cancer trials)
  • logistical problems (e.g. not being able to get to a treatment centre easily or not being able to take extra time off work)
  • the trial has very strict rules for who can take part and they are not eligible
  • simply not wanting to take part.

Question 8. Blind? What do all these words and phrases mean?

In the following we are going to explain words and phrases often used in the context of clinical trials. Many of those are also used by clinical researchers and within descriptions of clinical trials.

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Just click on the word to get the explanation:

Adjuvant therapy

If a cancer has been completely removed during an operation, there is still a chance that it can recur, due to tiny seeds from the cancer that spread. Adjuvant therapy adds to surgery, but trying to prevent this process and reduce the chance of a cancer coming back.

Best supportive care

This means a patient receives treatment to alleviate their symptoms, but no specific treatment against the cancer. This may include pain relief and other palliative measures.

Blinded

Blinding means concealing the allocation of which treatment the patient is taking. The power of persuasion is very strong, and people can often feel much better for taking a treatment, any treatment. In many trials patients are therefore “blinded” to which treatment they are taking.

Control arm

The group of patients who take the standard therapy, or if no therapy is proven useful in this situation, then patients may take a placebo, or best supportive care.

Double-blind study

Neither the doctor nor the patient knows if they are in the treatment or control group.

First-line therapy

This is the ‘go-to’ treatment that most people would take first when a disease is diagnosed. It is often the strongest and most effective treatment.

Open-label study

Both the doctor and the patient know which treatment group the patient is in.

Placebo

IKCC Pictograms 9 webA placebo is a dummy treatment that contains no active drug. It might be a capsule or pill, it might be a bag of saline. Sometimes just thinking you are being treated can make you feel better. This is a common psychological phenomenon called the placebo effect. Because there are now effective treatments for kidney cancers, placebos are very rarely used in kidney cancer trials. If you are thinking of taking part in a trial with a placebo group, you need to think about how you will feel if you find out at the end of the trial that you were given the placebo. Some trials give the new treatment to the placebo group after the trial has ended, or swap the treatment and placebo groups during the trial. So even if you are in the placebo group at first, you might still get the new treatment later on. When you ask about a clinical trial, be sure to ask about whether any patients will receive a placebo.

Randomisation

The process of randomly choosing which treatment group a patient is put into. This is normally done by a computer.

Screening

Before being allowed to start a trial, patients are always asked to undergo “screening” to ensure that their disease and general health is well understood and matches the criteria of the trial. Screening might be as simple as a blood test, or could include MRI, CT or PET scans. Unfortunately sometimes a biopsy of the cancer is also required, and even if you have only had scans recently, you might need to repeat these scans again.

Second-line therapy

This is the treatment given to patients if the first-line therapy does not work for them. If the second-line therapy doesn’t work, then a third-line therapy will be tried, then a fourth-line therapy, and so on.

Single-blind study

The doctor knows which treatment group the patient is in, but the patient does not.

Staging/Restaging

To assess whether treatment is helping patients or not, scans are taken at intervals to find out if patients’ cancers are bigger, smaller or the same size. Some clinical trials demand that a spot of cancer in a particular part of the body is measured at many times during the trial to assess treatment. These staging, and re-staging scans are therefore very important to assess the effectiveness of the treatment and to advise patients whether to stay on treatment or take something else.

Treatment/investigational/experimental arm

The group of patients who take the new treatment being tested.

 

Question 9. How do I find out more about clinical trials?

How to find out moreThere is a lot of useful information about clinical trials available online and through kidney cancer patient organisations. Local organisations may best know which trials are open in your region. For a list of kidney cancer organisations, see here.

Useful sources include:
  • the healthcare system in your country (e.g. the NHS Choices website in the UK)
  • teaching hospitals
  • medical research charities
  • patient groups
  • research programmes.

 

The following websites have some useful general information about clinical trials.

Please note that these websites are rarely up to date with the status of each trial, so be sure to call the hospital listed if you are investigating a clinical trial option.

Question 10. How do I find a clinical trial for ME?

This website has a list of major immuno-oncology (IO) trials for kidney cancer.

trial for me

To find out more about kidney cancer trials that might be right for you, you could try the following sources and ideas for more information.

  • Ask one of your doctors if they know of any trials that are suitable for you.
  • Ask about trials that might be available in other hospitals that you could travel to.
    • Call the hospital and ask to speak to “cancer clinical trials” or “oncology clinical trials”, then ask to speak to the “clinical trial coordinator” for kidney cancers or genitourinary cancers
  • Contact kidney cancer patient organizations in your country, or international organisations like the International Kidney Cancer Coalition.
  • Investigate country-specific or regional urology or kidney research networks or associations
  • Search clinical trial online databases; for example
  • Contact pharmaceutical companies themselves. Please be aware that pharmaceutical companies can only offer you information about the subset of clinical trials that they are conducting. For a broader view of clinical trial opportunities, contact the patient organization in your country. If you are not aware of a patient organisation in your country, please contact the International Kidney Cancer Coalition who may be able to put you in touch: This email address is being protected from spambots. You need JavaScript enabled to view it.