Repair MDS trial

Repair MDS Trial

A new study is being coordinated by the University of Warwick clinical trials unit and jointly sponsored with the Dudley Group of Hospitals NHSFT. The study is now available for patients who have been diagnosed with Myelodysplastic Syndrome (MDS).  The study is taking place throughout the UK and is funded by Blood Cancer UK. It will be open in around 30 hospitals and aims to recruit 120 patients. The purpose of the study is to compare two different types of treatment to discover if either of the treatments will have a positive impact upon our patients’ quality of life and blood transfusion requirements.

Who is the study For?

Blood cells are normally produced by special cells in the bone marrow, called stem cells. MDS causes changes in these stem cells making them ’abnormal’ or ‘dysplastic’.  These abnormal or dysplastic stem cells can then no longer do their job properly which means that normal blood cells are no longer produced, or too few of them are made.

MDS can go on to become leukaemia (Acute Myeloid Leukaemia) but this doesn’t always happen.  This study is for patients with a type of MDS which has a lower risk of progressing to leukaemia according to the IPSS-R (the International Prognostic Scoring System used to measure the ‘grade’ of MDS)

Some patients with MDS can be treated with a drug called Erythropoietin (EPO). This can work very well for some patients and can reduce the need for blood transfusions. However, EPO doesn’t work for all patients, or it might work for a while and then stop working. Also, at the moment, there are very few treatments for patients with low white cell counts and low platelets. The study is looking for new ways to treat these issues in MDS, especially in this group of patients for whom EPO doesn’t seem to work. If you are eligible for the MDS study your Haematology team will be able to give you more information about where this study is taking place and how you may be able to take part.

Repair-MDS is looking at two study treatment groups, both using drugs which are already widely used for other illnesses or purposes and been shown to be safe for those patients. The Repair-MDS will assess whether these drugs can also be used to safely treat patients with MDS. Many drugs used to treat cancer were originally used to treat other illnesses. The term used for this type of treatment classed as Repurposed drugs/medication.

What Treatments are being Studied?

Treatment Group 1: Sodium Valproate (V), Bezafibrate (Ba), Medroxyprogesterone (P). This treatment is called ‘VBaP’. Bezafibrate and Medroxyprogesterone were used in an earlier trial (BaP) in acute myeloid leukaemia patients. Several people responded with improved blood cell production. The research team’s laboratory has shown that the addition of valproate at low doses could make BaP work better.

Treatment Group 2: Danazol. This drug has been used for many years in patients with low blood counts, but newer studies suggest it may work particularly well in MDS.

Patients who are wishing to take part in this study may have an improvement in their blood counts, reduce the need for transfusions and therefore reduce the frequency of their hospital visits. It may also improve an individual’s quality of life – for example, patients may feel less tired. Furthermore, the information from this study may also help to improve the treatment of people with MDS in the future.

Prior to taking part in the study information will be given both verbally and in writing with a patient information sheet which gives an outline of the study and all that it entails. Patients who take part in the study should have time to read the information and have a clear understanding of what is required at each stage of the study prior to giving written consent.

Once you and your consultant and Haematologist have discussed the study and you are eligible to be registered for the study there are two stages that have to be completed before the start of the trial treatments.

Baseline Assessment Period

The next stage is called the ‘Baseline Assessment Period’.  This is a very important part of the study and will take between 4 and 16 weeks to complete. Once you are receiving study treatment the research team can look back at this information and compare how you are doing with how you were doing in the baseline assessment period, to assess whether the study treatment is working for you.

The Baseline Assessment Period can take up to 16 weeks and allows the research team to understand how your type of MDS is affecting your blood cells. Patients will be monitored carefully during this baseline period to see how many blood transfusions are needed, have there been any infections or problems with bleeding. During this period all patients who have been registered into the study will also need to go through some assessments and appointments. These will include a bone marrow test, blood samples, a heart tracing (ECG) and an Ultrasound Scan to look at the Liver and Spleen. Other investigations will include a physical exam in clinic, Blood pressure, pulse and temperature recordings before going ahead with the study.

The tests during the Baseline Assessment Period will decide if an individual is definitely eligible to take part in the study. If eligible, the patient can then go ahead to the next part of the study – randomisation.

Randomisation is a process which decides which of the two study groups you go into – Group 1 or Group 2. Neither you nor your doctor will be able to choose which group you go into. Once the treatment has been allocated patients will be informed which group, they are receiving, a discussion with the research team will be given so that each patient is aware of how to take their medication, how it will affect them and how to contact the clinical team with local contact numbers for advice.

Patients on the study treatments will be reviewed carefully by your local haematology and the study team to see how well the treatment is working, patients will be seen in clinic every 2 weeks for the first few months to look for any side effects from the treatment. During the clinic appointments patients will have their blood counts completed along with Blood pressure, temperature, & pulse recordings. The research team will complete a patient medication diary and ask if patients will complete a questionnaire about their quality of life during the medication.

Further tests and investigations may be required at specific timepoints during the study to monitor how effective each trial treatment is for both arms of the study. Full explanations about the study will be given by the research team to ensure that the patients who take part in the study have the right information to be able to make an informed decisions with their clinical team.