BSH 2023 | Insights into the REPAIR-MDS trial: danazol vs VBaP in lower-risk MDS

So, this is myself and Steve Jenkin who is the other clinical chief investigator, we’re running a national trial now on low-risk MDS. So these are patients without the higher risk problems, but basically looking at improvement in cytopenias and looking at reductions in transfusion dependence. So, this is the first national randomized trial we’ve had in low-risk MDS. We’ve got about 14/15 sites open and we’re trying to look for more sites. We’re very keen to try and open up centers, particularly in the larger district general hospitals, which is where most of these patients lie. They’re not at the tertiary centers and they won’t be willing to travel so far. They’re a difficult population because they’ve got co-morbidities to try and recruit, but they’re an underserved population. There are no treatment options for most of them and therefore we really want to look at trying to help these patients out. It’s a randomized trial looking at danazol, which is a drug that’s been around for many decades, being used in other conditions with cytopenias and some evidence of them working in MDS. So, this is really looking at whether that can work and improve the telomere length of these patients so, we’ll be looking at that aspect. This drug combination called VBap or [sodium] valproate, bezafibrate and medroxyprogesterone which has been used in some Phase II trials in AML, some patients have got benefit from it and improved in terms of improving differentiation. So we wanted to see whether this combination can actually improve cytopenias in these patients working on reactive oxygen species and we’ll see whether that can do that. But this is a difficult group to try and recruit. They all have these co-morbidities. We’ve seen in the past when we’ve had Phase II trials in low-risk MDS, they’re really difficult because patients are unwilling to travel, or they have got other problems that stops them from getting into the trials. So we need all the support we can get to try and get these trials open and prove that we can actually get these trials working so the industry might then be willing to try and give us further drugs that might be potentially or benefit to these patients.