REPAIR-MDS – a ground breaking clinical trial for low/intermediate risk MDS3 Aug. 2023
A new diagnosis of low/intermediate risk MDS can be shocking – hearing you have cancer, that it's life limiting and that there are very few treatments available, may well be distressing. Often, the only real option for patients is to return home and 'watch and wait' until the next appointment, usually three months away. The REPAIR-MDS trial offers a chance to get involved in one of two new experimental treatment options.
What is the REPAIR-MDS trial?
REPAIR-MDS is a ground-breaking clinical trial that aims to improve the symptoms and overall quality of life for patients with low/intermediate risk Myelodysplastic Syndromes. Patients will be put randomly into one of two treatment groups below. Both groups will be using drugs which are already used safely for other diseases (Repurposed drugs).
Treatment Group 1 VBaP - Sodium Valproate (V), Bezafibrate (Ba), Medroxyprogesterone (P)
Treatment Group 2 : Danazol.
The study is trying to find out if these ‘repurposed’ drugs can be used to treat MDS patients and to improve their blood counts, reduce their need for transfusions, improve their quality of life, and prolong their survival.
Who can participate?
Patients aged 18 years and over diagnosed with lower-risk MDS, who have either not been suitable for erythropoietin injections (EPO), have not responded or stopped responding to EPO, or they have a low neutrophil and/or platelet cell count.
Where are the trial sites?
The trial is taking place throughout the UK, is coordinated by the University of Warwick’s Clinical Trials Unit and will be open in around 30 hospitals and aims to recruit 120 patients.
Follow the link to the REPAIR-MDS website for a regularly updated list of REPAIR-MDS trial sites
What if my hospital isn't a trial site, is there a way I can participate?
Please let your clinical team know that you are willing to consider any relevant trials. As a patient you can ask your clinical team to review you for any clinical trial and consider opening the trial to recruitment at your hospital. If this is not possible, as not every trial or study is right for every hospital, you can ask for a referral to a recruiting centre where the trial/study is open to recruitment for consideration. This can be using established referral pathways, or outside of your usual NHS Trust partnerships if you decide is it is feasible and something you wish to pursue.
What's the trial duration?
September 2020 - June 2025
To find out more about what's involved, who to contact, and get more information, visit the trial website below.
REPAIR-MDS Central Management Team
A brief introduction to REPAIR-MDS from Co-Chief Investigator Dr Manoj Raghavan