Information about Clinical Trials

March 2019

Increasingly Haemochromatosis UK and haemochromatosis patients are taking an interest in the world of medical research. Just what does the future hold? Prospects for alternative treatments and treatment pathways are just beginning to emerge. Here we take a look at the world of the clinical trial.

Clinical trials for new drug treatments

For now, there are three current treatments to reduce iron levels for people with haemochromatosis: venesection, chelation therapy, and erythrocytapheresis. Of these, venesection is by far the most widely used treatment. Potential new drugs or medical devices may provide an avenue for treating iron overload or its effects. We’re aware that new drugs for GH are in development, so if venesection is not an option for you, or if you find it difficult, new treatments may offer a welcome alternative.  For those of you being treated with chelation therapy or erythrocytapheresis, a new drug treatment may also offer you choice.

During 2019, HUK trustees are developing ethical policies to guide our activities and this will include, for example, how we respond to clinical trials for new treatments. In the meantime, in this article, we briefly describe the process by which new drugs are tested in volunteers before they are approved.  If you’re interested in new treatments, or if you’re thinking about volunteering for a drug trial, we hope you’ll find this helpful.

This is a brief introduction only.  It’s not intended to cover all aspects of drug trials, or all of the issues to be thought about before taking part in a drug trial.

Testing new drugs

All new drugs are tested for safety and effectiveness before they can be licensed or used in the NHS.  This usually takes many years. The pharmaceutical industry first develops and tests new drugs in animals in laboratory studies. If a new drug looks promising at this point, the next stage is to test the drugs in people in clinical trials. Sometimes, drugs already approved to treat one condition, are found to be beneficial for other conditions.  In this case, the time to test and get the drug into use can be much shorter.

Although pharmaceutical companies sponsor/fund research for new drugs, they are not allowed to test the drugs in people. This must be done by independent clinical research teams under strict ethical rules. In the UK, the organisation responsible for overseeing clinical drug trials is the Medicines and Healthcare products Regulatory Agency (MHRA).

Before any drug can be tested in humans in the UK, the sponsors must apply for ethical approval from a local research ethics committee (REC). REC members include clinicians, scientists, and patient representatives.  Their role is to ensure that there are good scientific reasons to conduct the research.  Secondly, they look carefully to see that the rights of anyone taking part in the research are protected.  This includes, for example, making sure potential volunteer participants are given information about the research in a way that helps them to make an informed decision. The information should include details of the known risks as well as potential benefits.  No drug comes without the potential for harm, so this is very important.  Individuals need to be able to weigh up the risk of harm against the potential for benefit before deciding whether or not to take part in a clinical trial.

Phases of clinical trials

Usually there are a number of phases to research into new drug treatments.


Drug is tested in an animal or cells that mimic the condition for which it is being developed. The aim is to find out if the drug has the potential to treat the condition. Although human volunteers are not involved in this stage, the results of this preclinical testing are the basis of approval for clinical trials in people.

Phase 1

Drug is tested in a small number of people who are healthy / who do not have the condition (in some trials people with terminal disease may be included; also, people with the condition might be included if only a few people are expected to be recruited for a trial). The aim is to see if the drug is safe, and to see what impact (for example, side effects) the drug has on the body with different doses.

Phase 2

Drug is tested in a small number of people who do have the condition. The aim is to test the safety of the drug in people who do have the condition, to see what impact it has on the body, and to see if it is effective in treating the condition.

Phase 3

Drug is tested in a larger number of people with the condition. The aim is to test the safety of the drug in people who do have the condition, to see what impact it has on the body, and to see if it is effective. Usually at this point the drug is tested against an existing treatment or an inert substance (placebo) to compare the effects of each.

Phase 4

Sometimes a phase 4 trial is conducted with many thousands of participants to see if the drug is safe and effective in the ‘real world’.

Medical devices are also tested in trials and are subject to strict controls.  If devices are adapting existing technology, however, (e.g. a different type of needle), approval might not be needed.

You can read more about clinical trials at these links


You’ll notice that in Phase 3 trials, drugs are compared to placebos.  Volunteer participants (and clinicians treating the volunteers) will not know if the individual has been given the drug or the placebo.  This is important to ensure that the real effects of the drug can be measured. However, it does mean you might not receive the new drug being tested. In some cases, when the benefits of a new drug are so powerful, the trial is effectively ended as it would not be considered ethical to deprive people in the placebo group of the treatment.

Even if you have the condition for which a new drug is being tested, you might not be eligible to take part.  Eligibility is often restricted by age, other health conditions or treatments, and so on.  This is to ensure that the effects of the drug being tested can be more easily identified and are not impacted by other factors (e.g. other treatments being used).

Finding out if a trial has ethical approval

If a clinical trial has received UK ethical approval, you should be able to find it on the NHS Health Research Authority website at  If you type haemochromatosis in the search box, you will see a list of research, including any drug trials, approved in the UK, together with information on the investigators. Sometimes, after getting ethical approval for a study, the trial investigators request amendments to the original approval. You can ask the investigators if this is the case and they must provide you with an up-to-date version of the approved trial information before you take part.

Those are the key contact points if you are thinking about volunteering in a clinical drug trial.  If you are eligible and interested in taking part, you will be given a Patient Information Sheet (PIS).  Do take time read and think about the information in the PIS, and discuss it with your GP and/or your consultant before you make your decision.

What if you don’t wish to take part

Expressing an interest in a clinical trial does not mean you are obliged to take part. If you choose not to proceed after expressing interest, you do not have to provide a reason for your decision.  If you do take part, you have the right to withdraw from a trial at any point in time.

Be well informed

From a legal standpoint, anyone taking part in a clinical trial must give informed consent.  The clinical trial team are obliged to describe what the trial is about, including risks and potential benefits, in a way that can easily be understood.

At HUK, we are keen to inform our members and the wider GH community about treatments. While we don’t actively promote any individual company conducting a clinical trial, we hope that this short guide, directing you to the expert sources of information, will help you to find out what you need to know about new treatments in development.

Later this year, we will be represented at The European Iron Club (a group of scientists with an interest in iron metabolism including GH) Congress in Germany.  We expect to learn more about current research including development of new drugs and we will share information about that with you in due course.

Our advice is to ‘be well informed’ and discuss the Patient Information Sheet about the trial with your GP and/or consultant before making your decision.