We're here for you if you want to talk

0808 2080 888

[email protected]

Clinical Trials Information Hub

We're here for you if you want to talk

0808 2080 888

[email protected]

Understanding clinical trials

All new drugs and treatments are thoroughly tested before they’re made available to patients. Following tests in a laboratory, they’re tested on people. Research studies involving testing new drugs and treatments on people are called clinical trials.

What are clinical trials and why are they important?

Clinical trials are important, because they’re the only way to develop new treatments – and improve existing ones – for you and other people with blood cancer. Researchers can compare the effects of new drugs and treatments to find out whether they work better than the current treatment used.

Clinical trials may test:

  • new drugs that have passed safety studies
  • medical equipment
  • new combinations of current treatments, and
  • different ways of giving treatments.

The goal of clinical trials is to find better and kinder treatments for people with blood cancer.

Although the outcome for people with blood cancer continues to get better, there’s still a lot more to be done to improve treatments and quality of life.

Clinical trials are also important to find out :

  • whether new treatments are safe
  • What side effects a treatment may cause (and how to manage them).

If clinical trials aren’t carried out there’s a risk that people could be given treatments that don’t work or are harmful.

An overview of clinical trials

There are several phases that all new drugs or treatments must go through before they are made widely available.

Clinical trials are divided into phases. The early phases look at the safety and the side effects of a new drug. Later phases test whether a new treatment is better than existing treatments.

Pronounced Phase "One", these trials test the safety and side effects of a new treatment. People who take part in phase I trials (up to 30 people) often have advanced cancers and have usually tried all the treatment options available to them. The first few patients to take part in the trial are given a very small dose of the drug. If they respond well, the next group have a slightly higher dose. The dose is gradually increased with each group. The researchers monitor the effect of the drug until they find the best dose to give. This is called a dose escalation study.

Pronounced Phase "Two", these trials test the new treatment on a larger group of people (more than 100) to find out more about the side effects, safety of the new treatment, and the effects on the cancer. These trials can last several weeks or months, and sometimes they can continue for years if the person is responding to treatment.

Pronounced Phase "Three", these trials involve more people (hundreds or thousands) and compare new treatments with the current treatment available longer-term.

Pronounced Phase "Four", these trials trials are carried out after a new drug has been shown to work and has been licensed to be used (a licence means the medicine can be made available on prescription). These trials aim to find out how well the drug works when it’s used on more people, the long-term benefits and risks, and more about the possible rare side effects.

Types of clinical trials

There are several different types of clinical trials or studies, in this section we break down some of the most common trial types. 

In open label trials, both you and the researchers know the treatment you’re having. These trials usually compare two very similar treatments to test which treatment is the best, and usually happen in the early phases.

If you take part in a randomised clinical trial, you’ll usually be put randomly into either:

  • the treatment group – where you’ll be given the treatment being trialled, or
  • the control group – where you’ll be given the existing standard treatment, or a placebo (a treatment that has no effect) if no proven standard treatment exists. A placebo is only used if outside of the trial, there is no other treatment option available. This could be because the person has already tried all the available treatments. Nobody in the trial would be given a placebo if there was any other treatment option available to them, as this would be unethical.

If you take part in a randomised trial (most phase 3 and some phase 2 trials are randomised) a computer programme is usually used to randomly select which group you will be in. Your personal details, such as age, sex and state of health, are taken into account so that the groups in the trial are as similar as possible. This helps to make sure that the results of the trial are reliable and accurate.

A blind trial is a trial where the people taking part don’t know which treatment they are getting. You might get the new treatment. Or you could receive the standard treatment. So that you can’t tell which treatment you are having, everyone taking part will receive identical injections, tablets, or other forms of treatment.

In a double blind trial, you won’t know which group you’re in, and neither will the researchers, until the end of the trial. This makes sure that the results are more reliable as they will not be influenced by your expectations or the researchers’ expectations.

Woman talking on the phone with headphones and computer.

Get help and support

Find out how our Clinical Trials Support Service can help you navigate through the entire clinical trial process.

Clinical Trials Support Service

We're here for you if you want to talk

0808 2080 888

[email protected]