Around 1 in 5 people diagnosed with cancer in the UK take part in a clinical trial.
A study looking at the drug deferasirox in people with myelodysplastic syndromes (De Iron)
Please note - this trial is no longer recruiting patients. We hope to add results when they are available.
This study is looking at using the iron reducing drug deferasirox (Exjade) before iron levels in the body get too high, rather than the usual way of giving it once these levels have been reached.
But because blood is rich in iron, having lots of transfusions may cause a build up of iron that is not needed. Doctors call this iron overload. Although iron is a very important part of the blood, too much iron may affect body organs, including the liver and heart.
Doctors in this study want to test a drug called deferasirox in people with MDS. Deferasirox works by binding to the iron in your blood and removing it from the body in your urine or stools. It is already used in people with iron overload. But in this study doctors want to see if giving it early is better than waiting for iron levels to rise. The aims of this study are to see
- How well deferasirox works
- How safe it is
- How well people can cope with the drug
Who can enter
You may be able to enter this study if you have MDS and are in one of the following situations
- You have been told that your MDS is low risk, or low to moderate risk of turning into leukaemia (International Prognostic Scoring System (IPSS) Low or Intermediate 1) and you have not had any treatment, or medication to treat your MDS hasn’t worked, or has now stopped working
- You have been told by your doctor that your MDS is moderate to high risk of turning into leukaemia (IPSS Intermediate 2) and tests show that fewer than 10 in every 100 (10%) cells in your bone marrow are immature cells called blasts, and you have no
chromosomeproblems and the number of cells in your blood have stayed about the same since you were diagnosed - you can ask your doctor about this
To enter this study you must also
- Have a low number of red blood cells (anaemia) and have needed a
blood transfusionwith at least 2 bags (units) of blood in the last 8 weeks
- Have more iron in your blood than is normal, but not enough to have iron overload– you can ask your doctor about this
- Have satisfactory kidney function and other blood test results
- Be willing to use reliable contraception during the study and for 3 months after if there is any chance that you or your partner could become pregnant
- Be at least 18 years old
You cannot enter this study if
- You have had drug treatment for your MDS in the last 8 weeks
- You have had a bleeding episode in the last 8 weeks
- You have had any other cancer in the last 3 years, apart from successfully treated carcinoma in situ of the cervix, basal cell skin cancer, cancer cells in just your bowel lining or prostate cancer that is contained in the prostate gland (localised), and is being controlled with hormone therapy
- You have had any experimental medication as part of a clinical trial in the last 6 weeks
- Your doctor is worried about your use of alcohol or drugs
- You have an infection
- You have HIV
- You are pregnant or breastfeeding
- You have any other condition that would make you unwell if you took part, or affect the results of the study – you can ask your doctor about this
This study will recruit up to 54 people. Everyone taking part will take deferasirox tablets once a day for up to 12 months. You dissolve deferasirox in water or orange or apple juice. You take it on an empty stomach, at least 30 minutes before food.
You give up to 4 blood samples for the study during the year. The team will look at these to see how much iron is in your blood during treatment.
The team may ask if you would be willing to have some MRI scans for the study. These will look at your body organs before the study and again at the end of the study, or if the iron in your blood increases. The MRI scans will be able to show how much iron has built up in your body since you started the treatment.
Before you start the study you will see the doctor and have some tests. These tests include
- Physical examination
- Hearing and eye test (if needed)
- Blood tests
- MRI scan (if you agree)
During the study you see the doctor and have a blood test
- Once a week for the first month
- Once a month for the rest of the year
If you agreed to have the MRI scans for the study, you will also have one at the end of the study, or if your iron levels rise during the study.
When you finish the study, you will see the doctor about 30 days later, so that you can tell the doctor about any possible side effects you may have had since finishing the study treatment.
Common side effects of deferasirox include
- Feeling and being sick
- Tummy pain
- Headache and dizziness
- Skin rash
- Short term changes to the way your liver and kidneys work
- No longer being able to hear high pitched sounds
- Changes to your sight, making things look cloudy
The team are not sure if the hearing and sight changes get better with time. But you can talk to them about this or any of the other possible side effects if you are worried.
How to join a clinical trial
Please note: In order to join a trial you will need to discuss it with your doctor, unless otherwise specified.
Dr Dominic Culligan
NIHR Clinical Research Network: Cancer
University of Birmingham