Clinical Trials – What are they?

You may be asked to take part in a clinical trial by your physician as part of your treatment.

Or - you may want to ask your physician or specialist nurse whether a particular trial might be available as part of your ongoing care for MDS.

Patients need to be aware of clinical trials and need to be well informed by their physician or nurse about the various points to consider before agreeing to enter a clinical trial (risk/benefits, regular travelling to the trial hospital, travel costs, understanding the consent forms, care after the trial ends, etc).

It is important to know all these things and have answers to all your questions before you become involved in a clinical trial.

MDS Factsheet: Clinical Trials

Information for patients and family members

What is a clinical trial ?

Clinical trials are the process by which new treatments are tested, evaluated, and the evidence gathered so that the decisions can be made about changing standard practice.

Trials aim to find out if a new treatment or procedure:

  • Is safe
  • Has side effects
  • Works better than the currently used treatment
  • Helps you feel better
  • Might be a cure for a condition

There are 4 phases of clinical trials. Each treatment being tested has to go through all these phases before it can be used.

Phase 1 trials are often 'first-in-man' - treatments which have never been tried on human patients before. Drug trials will be looking for safety information, side effects, and considering dosage issues. Numbers recruited will be small, sometimes no more than 10-15 patients. Phase 1 trials are done under rigorously controlled conditions with intensive monitoring.

Phase 2 trials examine which diseases respond to the new treatment, and will be comparing dosages, assessing side effects and looking at outcomes. The numbers of patients recruited may be quite small (50 or so) but the data gathered will be the basis for later large scale trials. Occasionally Phase 2 trials are randomised so that alternative doses or methods of giving the new therapy can be compared.

Phase 3 trials are most often 'randomised controlled trials' (or RCTs). Here a standard therapy is compared with one or more new ones - if it gives better results, it may become the new standard treatment. Patients are randomly selected to receive one of the treatments, usually selected by computer to ensure there can be no bias. These trials are mostly large scale and can have thousands of patients in them, although with a rare condition like Myelodysplastic Syndromes the numbers are more likely to be in the hundreds.

Phase 4 trials are carried out after a drug has been licensed - they collect information about side effects, safety and the long term risks and benefits of a drug by continuing surveillance of patients on the treatment. This may be how rare side effects are identified

Different types of trials

The researchers may look at the impact a treatment has on you personally as well as the treatment’s clinical benefits - for example, how often you have to travel to the hospital, or whether you are able to lead a full and normal life. Studies of impact and side effects are sometimes called quality of life studies. Most well planned trials include a quality of life study.

Usually, a new treatment has to go through a few phase 3 clinical trials before doctors are confident enough to accept it as the new standard treatment. One good trial result could happen by chance or because a trial was not very well designed. This is not likely if several trials have the same results. Satisfactory results in a number of clinical trials are essential before a new treatment can be recommended by a regulatory body such as NICE.

Not all clinical trials will result in new and better treatment. Some will discover that the treatment being tested does not work, or is no better than an existing, established treatment. A trial might find that a new treatment has side effects that are much worse, or no less, than with existing treatments. But this information is also useful for researchers and doctors, and in the end for patients.

Trials databases

The UK Clinical Research Collaboration has produced a booklet on "Understanding Clinical Trials", which explains what clinical trials are and how and why they are carried out. It is designed to answer the many questions people may have when deciding whether to take part in a trial.

Phase 1 and 2 trials are most likely to be at individual hospitals, or a very small number of hospitals. Phase 3 trials in MDS are often developed as pan-European trials through the EORTC (European Organisation for Research and Treatment of Cancer) and may be available in many UK treatment centres.

To find which trials are currently open in MDS follow the links below. No log-in is required in any of these databases. You may need to use the search facilities offered. Where necessary use the search term 'MDS' or ‘Myelodysplastic Syndromes’ to keep the widest options available.

You will find trials involving research about all 3 types of blood cells affected by MDS: Red blood cells (Erythocytes), white blood cells (Neutrophils) and platelets (Thrombocytes).

Cancer Help UK has a 'plain English' database of clinical trials in the UK maintained by Cancer Research UK, as well as very comprehensive information regarding trials generally.

European (EORTC) Phase 3 trials (includes those open in the UK): here

This last link is the US database of all clinical trials taking place anywhere in the world – including the UK: here

Other aspects to consider

As patients and patient support groups, we also have the ability to shape the future development of clinical trials.

We could request the publication of all clinical trials (currently only 40% of trial results are published).

We could ask that patients who participate in clinical trials and benefit from the drugs be given that treatment for as long as they need it – not just for the time of the trial.

Please contact us if you have any general questions about clinical trials.

Please always consult your doctor or nurse about any decisions regarding your treatment.

Trials Recruiting Now

Read more information about current trials, eligibility criteria and what they are trying to achieve. Always discuss trial information with your own physician.

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