A study looking at treatment for low risk myelodysplastic syndromes (REPAIR-MDS)

Cancer type:

Blood cancers
Myelodysplastic syndrome (MDS)




Phase 2

This study is comparing 2 treatments for myelodysplastic syndromes to increase the number of blood cells Open a glossary item

Myelodysplastic syndromes (MDS) are a group of blood cancers. This group of conditions causes a drop in the number of blood cells. 

This study is for people who:

  • have low risk MDS
  • can’t have a drug called erythropoietin (EPO Open a glossary item), it didn’t work or it has stopped working 
  • have low blood counts Open a glossary item

More about this trial

Myelodysplastic syndromes are also called myelodysplasia or MDS for short. In some people, MDS can develop into acute myeloid leukaemia Open a glossary item. This study is for people whose MDS has a very low, low or moderate  risk of turning into leukaemia.

You might have erythropoietin (EPO) to treat MDS. It helps your body to make more red blood cells Open a glossary item. This works for some people but sometimes it stops working or doesn’t work at all. There are also very few treatments for people with low neutrophils Open a glossary item and low platelets Open a glossary item. So doctors are looking for treatments to help people who have low blood counts. 

They are looking at drugs used for some other illnesses and other cancers to see if they help. In this study, researchers are looking at a combination of treatment called VBaP. This includes:

  • sodium valproate – an epilepsy drug 
  • bezafibrate – a drug to treat high cholesterol
  • medroxyprogesterone – a drug used in other cancers to increase the number of blood cells. It is also a type of contraceptive pill Open a glossary item.

The researchers are comparing VBaP with another drug called danazol. 

We know from earlier research that bezafibrate and medroxyprogesterone increased the number of blood cells in people who have acute myeloid leukaemia. Doctors think that adding sodium valproate to these 2 drugs could increase the number of blood cells even more. 

Danazol is a treatment to help people with low blood counts. It stops the cells in the bone marrow from ageing. This allows them to make blood cells for longer. Newer studies suggest it could work well for people with MDS.

The main aims of the study are to find out:

  • how well treatment works to improve low blood counts
  • if treatment reduces the need for blood transfusions
  • if treatment increases the length of time people live 
  • how treatment affects quality of life  Open a glossary item

Who can enter

The following bullet points are a summary of the entry conditions for this study. Talk to your doctor or the study team if you are unsure about any of these. They will be able to advise you. 

Who can take part

You may be able to join this study if all of the following apply. You:

  • have low risk myelodysplastic syndrome. Your doctor will know this. 
  • have had low levels of red blood cells (anaemia Open a glossary item), low levels of platelets  Open a glossary itemor low levels of neutrophils Open a glossary item for at least 16 weeks. And you have symptoms of low blood levels such as bleeding or infections. Your doctor checks these levels when you join the study to see you are suitable to take part.  
  • have satisfactory results for all your other blood tests
  • can’t have erythropoietin (EPO), it didn’t work or it stopped working 
  • must be well enough to be up and about for at least some of each day, even if you need help looking after yourself (performance status 0, 1, 2 or 3)
  • are at least 18 years old 

Who can’t take part

Cancer related 
You cannot join this study if any of these apply. You:

  • are having treatment for low risk MDS
  • have a chromosome Open a glossary item change called del 5q. Your doctor will know this. 
  • have had a bone marrow transplant with somebody else’s cells (allogeneic transplant Open a glossary item)
  • have had erythropoietin (EPO) or G-CSF Open a glossary item within 16 weeks of joining the study 
  • have already been put into a treatment group for this study even if you didn’t have treatment 
  • are having treatment for another cancer 
  • have prostate cancer
  • have taken part in another clinical trial using a drug or device in the last 16 weeks

Medical conditions
You cannot join this trial if any of these apply. You:

  • are taking sodium valproate, carbamazepine or phenytoin for epilepsy 
  • have liver problems  Open a glossary item
  • are taking a fibrate medication. Your doctor will know this. 
  • have moderate to severe heart problems Open a glossary item
  • are taking a medication called a statin to prevent further heart problems, strokes or circulation problems. You might be able to take part if you are taking a statin but have never had a heart problem or stroke.
  • are taking medication to thin your blood. You can take part of you are taking a blood thinner called warfarin. 
  • have had a blood clot in in the past, for example in your leg or lung
  • have HIV
  • have a condition called acute porphyria

You cannot join this study if any of these apply. You:

  • can’t have any of the treatments in this study or you are allergic or sensitive to them
  • are pregnant, there is any chance you could become pregnant or you are breastfeeding 

Trial design

This phase 2 study is taking place in the UK. The team need about 120 people to take part. 

It is a randomised study. You are put into a group by a computer. Neither you nor your doctor will be able to decide which group you are in. There are 2 treatment groups. 

You have 1 of the following:

  • sodium valproate, bezafibrate and medroxyprogesterone (VBaP)
  • danazol

How you have treatment
You start on a low dose of treatment. Your doctor will slowly increase the dose if treatment is working and the side effects aren’t too bad. 

Sodium valproate is a tablet. You take them twice a day.

Bezafibrate is a tablet. You take them 3 times a day. 

Medroxyprogesterone is a tablet. You take them twice a day.  

Danazol is a tablet. You take them 3 times a day. 

You have treatment for up to one year as long as it is working and the side effects aren’t too bad. 

Samples for research 
The researchers will ask for some extra samples of bone marrow Open a glossary item when you have a bone marrow test done. They will also ask to take some extra blood samples. You have these at the same time as your routine blood tests.

They plan to use the samples to see how treatment affects the blood and bone marrow. 

You can say no to giving these samples for research and still take part in the main study. 

Quality of life
The study team ask you to fill out a questionnaire:

  • before you start treatment
  • at set times during treatment
  • at set times after treatment 

The questionnaire asks about side effects and how you’ve been feeling. This is called a quality of life study.

The study team ask you to complete a diary to record when you take your medication and how many tablets you take. This helps them to check you are taking it correctly. It also helps you to know what dose you should be taking. 

Hospital visits

You see the doctor and have some tests before you can take part. These include:

You have regular blood tests and check ups with your doctor. You will be asked to have another bone marrow test:

  • 6 months after you start treatment and at
  • 12 months

The team call you about 4 to 6 weeks after you finish treatment to see how you are. 

Side effects

The study team monitor you during treatment and afterwards. Contact your advice line or tell your doctor or nurse if you have any side effects.

The most common side effects of sodium valproate include:

The most common side effects of bezafibrate include:

The most common side effects of medroxyprogesterone include:

  • headache
  • tummy (abdominal) pain
  • anxiety
  • breast pain or tenderness
  • changes in skin colour
  • making low amounts of urine 
  • difficulty swallowing
  • dizziness or light headedness
  • sweating a lot 
  • high blood sugars
  • swelling due to fluid retention

The possible side effects of danazol include:

  • changes to how your liver works 
  • muscle aches
  • high blood pressure
  • skin changes
  • feeling sick
  • blurred vision
  • low mood
  • your body not responding well to insulin 
  • flushing
  • risk of blood clots, but this is rare


Great Yarmouth

Recruitment start:

Recruitment end:

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.

Please note - unless we state otherwise in the summary, you need to talk to your doctor about joining a trial.

Chief Investigator

Dr Stephen Jenkins 
Professor Janet Dunn 
Dr Manoj Raghavan 

Supported by

Blood Cancer UK
The Dudley Group NHS Foundation Trust
University of Warwick
The University of Birmingham
MDS UK Patient Support Group

Other information

You can see a map of where this study is taking place on the REPAIR-MDS trial website.

If you have questions about the trial please contact our cancer information nurses

Freephone 0808 800 4040

Last review date

CRUK internal database number:


Please note - unless we state otherwise in the summary, you need to talk to your doctor about joining a trial.

Last reviewed:

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