General Updates | Investigating venetoclax in children with R/R AML: a sub-trial of the PedAL master trial

We had to get all the investigators together and agree on endpoints and a data dictionary and also agree on what we would consider an internationally agreed upon standard of care therapy. And then we had to get data for that therapy. And we were able to do that. And with partnering with AbbVie, who is developing venetoclax, we were able to start what is a registrational trial. And it’s a single trial in all parts of the world. So we have a single database, a single trial, and we are able to test whether the addition of venetoclax adds to standard of care in terms of remission and eventually in terms of survival. And so this is a registrational trial for the use of venetoclax. The positive part is we are accruing patients in Japan, in Europe, in Canada, in Australia, in the United States. So really in developed countries around the world. 

The part that still tells us we need to do more is this is eight years since venetoclax was approved for adult acute myeloid leukemia. So we know the system is still broken. This is a good step in the right direction. And we hope will give us a new standard of care or at least an agreed upon standard of care that will give us a database and a starting point for new therapies for pediatrics. 

This is a randomized trial. And so there are no data points to share right now because this is going to be… the only thing we know is that the DSMB has said it’s safe to continue the trial. And so, no, we will not have data until after the trial is completed. But we are more than halfway through the accrual. So we feel confident that this will be able to continue. And it’s really important to do. 

Another point to make about this is venetoclax is available off-label in the United States. So the investigators here are at a disadvantage because this is a randomized trial. They could just give their patients venetoclax and they might. They have a 50 percent chance of not getting venetoclax. And so in Europe and in other parts of the world, venetoclax is not available off-label. And so we see where this time difference between adult approval and pediatric approval actually harms patients in pediatrics from answering these important questions about whether it’s useful to use these drugs in the pediatric leukemias. And so just another point to say, we’re very happy that we’re accruing patients. But it’s a lot of learnings that I think are very important for understanding what we need to change in the system.

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