Clinical Trials

New treatment trials in thalassaemia

What are clinical trials?

A clinical trial is a type of project designed to investigate the effect of a treatment or intervention on human health. Whilst interventions may be new medicines or medical devices to help treat thalassaemia, it can sometimes can also take the form in lifestyle or behavioural changes.

Clinical trials are designed to investigate whether the intervention is both safe and effective in making a positive change to thalassaemia.

Why are clinical trials needed?

According to the NHS, Clinical trials help health care professionals understand how to treat a particular condition. It may benefit you, or others like you, in the future. If you take part in a clinical trial, you may be one of the first people to benefit from a new treatment.

However, there is also a chance that the new treatment turns out to be no better, or worse, than the standard treatment for thalassaemia.

Without clinical trials, treatments for conditions like thalassaemia, diabetes and others would never have been developed.

Clinical trials are the only way to ensure that new treatments are safe and effective.


What happens in a clinical trial?

All clinical trials of new medicines or devices go through a series of phases to test whether they’re safe and whether they work. In every clinical trial, the new treatment will be tested against another called a control.  A control will either be a placebo (inactive drug) or a standard treatment already in use. This is to test whether or not the treatment is safe and effective.

A clinical trial will usually consist of four phases: 

Phase 1 trials: 

Researchers trial the drug in a small number of individuals (human volunteers) for the first time, who may be healthy volunteers, are given the medicine. 

Researchers test for side effects and calculate what the right dose might be to use in treatment. 

Researchers start with small doses and only increase the dose if the volunteers do not experience any side effects, or if they only experience minor side effects.  

Phase 2 trials: 

Ill individuals are chosen in larger group to test the new medicine. This is to get a better idea of its effects in the short term. 

Phase 3 trials: 

Involve several thousand patients and carried out on medicines that have passed phases 1 and 2. 

The medicine is tested in this phase and compared against an existing treatment or a placebo to see if it’s better in practice and if it has important side effects. 

Trials often last a year or more. 

Phase 4 trials: 

The safety, side effects and effectiveness of the medicine continue to be studied while it’s being used in practice. All medicines that have passed all the previous stages and have been given marketing licences (means the medicine is available on prescription) will pass through this phase. 

Not required for every medicine. 

NHS Clinical trials information

Should I take part?

Deciding whether or not to take part in a clinical trial is a personal decision; you should never feel pressured to take part.

People living with thalassaemia who have participated told us that it’s really useful to get as much information as possible about the trial, so you can make an informed decision.

You should take as much time as you need to decide whether or not to take part. You may also find it useful to talk about it with your family and friends, especially any possible impact on your personal life, work/school and family commitments.

It is important to be aware that you would not necessarily receive a new treatment by participating in a trial – you may receive standard treatment or a placebo, but this still often contributes to a positive experience by helping identify the best treatment for yourself and others.



Informed Consent

Informed consent is a process to make sure that potential participants in clinical trials:

  • Can ask questions and get answers before, during and after the trial
  • Fully understand the nature and purpose of the trial
  • Fully understand what the trial will involve
  • Are informed about potential benefits to health, risks and inconveniences
  • Only agree to take part willingly
  • Remain informed throughout their period of participation

This process starts when your thalassaemia doctor or researcher introduces details of the clinical trial to you. Following initial discussions, you will be given information to take home to read and consider further with family or friends, who may also attend meetings with you. You should feel confident that you have enough information to help you decide whether you want to take part in the trial, and that you have been given enough time to consider all the information and what the trial will mean to you in practical terms, such as extra appointments and tests.

You can’t be entered into a trial without giving your written consent. Where clinical trials involve children the consent process is also different and will be fully explained by the thalassaemia doctor or researcher you talk to about the trial.

If you decide to take part, you will be given a consent form to sign to say that you understand the information and give your consent willingly – you should retain a copy of this form for your own records.

Informed consent is an ongoing process throughout your period of participation in a trial.

Researchers should continue to provide information, answer your questions and advise if any new relevant information comes to light during the trial.

You can reconsider your participation at any point, or even withdraw without the need to give a reason.

Your decision must be respected and will not affect your ongoing treatment for thalassaemia.

If you have any concerns or would like to talk to someone about the trial, you can always contact our team and we will be happy to talk to you or even connect to someone who has participated in clinical trials.

It is important to be aware that you would not necessarily receive a new treatment by participating in a trial – you may receive standard treatment or a placebo, but this still often contributes to a positive experience by helping identify the best treatment for yourself and others.

Current Clinical Trials in the UK