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Clinical Trials: what are they?

written by

Leukaemia Care, Charity

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Clinical trials are part of clinical research. They often include comparing a new drug therapy with a standard chemotherapy that is currently being used to treat a certain type of cancer. The aim of cancer research is to improve cancer therapies and develop new effective treatments that are safe for humans.

There are many different types of clinical trials and research studies. Some of the studies that are available for patients with a blood cancer use specific blood tests and bone marrow results at diagnosis, and also for monitoring the response to treatment. Blood and bone marrow tests are taken at the start of the study and are tested again during and after treatment to assess the patient’s response to their chemotherapy.

In cancer treatment, particularly leukaemia, MRD (minimal residual disease) testing has several important roles in determining whether treatment has eradicated the cancer or whether traces remain. Blood and bone marrow tests are taken to compare the efficacy of different treatments, monitoring patient response to treatment, as well as detecting recurrence of the leukaemia or other blood cancer.

A lot of clinical trials will be interventional studies, which means these studies may be comparing types of treatments, devices for drug delivery, or will look at a patient’s quality of life with questionnaires. It is important to find out how new therapies impact upon a patient’s life, such as how many times does a patient need to attend hospital visits, and also look at how the treatment affects your daily activities.

Observational studies, on the other hand, involve asking patients for their permission to enter into a clinical trial that is collecting information about how one group of patients are treated, or how often they are seen in clinic, for example. Often, clinical trials will also ask for patients to provide a blood test or mouth swab that can be stored in the lab for future research studies. This will then be used to help find out more about a specific blood cancer.

Types of studies

All clinical trials are thoroughly tested, with drug therapies first tested in the laboratory. Following this, the next stage is for the trial to be carefully studied in people; this is referred to as ‘bench to bedside’.

There are three main phases for clinical trials to be tested at, along with one further phase monitoring licensed drugs:

Phase 1 trials are early phase studies. They recruit small numbers of patients and separate them into groups. Each group of patients, called a cohort, are given a trial drug and monitored closely for side effects, as well as how the drug dose works and affects the body. The information that is collected in the Phase 1 trial will be essential in order to take the potential new drug through to its next phase.

Phase 2 studies allow more patients to enter into a study, but are limited to some treatment centres or hospitals. The aim of Phase 2 is to again look at the best dose to use, as well as to monitor side effects and how to manage them. Often in Phase 2 trials the new drug may be compared to another treatment already in use, or a placebo (dummy drug/without any active drug). This is to see if the new therapy shows some promising effect or benefit, and can safely go forward to be tested in Phase 3 clinical studies.

Phase 3 studies are trials that involve large numbers of patients and are carried out in many treatment centres and hospitals. The aim of the Phase 3 trial is to compare the new drug therapy against the current gold standard to see if this drug is more effective. These studies have lots of research questions to answer and may include having access to new novel therapies or comparing chemotherapies that are currently available.

Lastly, Phase 4 is a study that involves a drug when it has been licenced for a specific cancer to be further monitored for side effects and long term safety information.

Some of the studies may compare targeted immunotherapies and monoclonal antibodies. This may include being allocated more treatment as maintenance when the standard practice is to stop after a set number of treatment courses. It is important patients make an informed decision and discuss the treatment options and possible entry into a clinical trial with their Doctors and specialist research teams.

Why are trials important?

Clinical trials and clinical studies are essential in helping to improve our current and future cancer treatments. They allow access to new and novel therapies and techniques that will give more knowledge and a better understanding of how blood cancers are diagnosed and behave. It is important that we continue to make progress and learn more by monitoring low levels of disease at the molecular level, as this knowledge may help inform us of when patients need further therapy. With more choice and new novel agents becoming available, it is important to understand where best to use these new drug therapies up front or as maintenance in order to make the most of our new treatments in the most effective way possible.

How to find a clinical trial?

Open data platform –

Please note: not all studies run at all hospitals and you will need to discuss available trials/studies at local centres with specialist team doctors and clinical nurse specialists.

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