A Study to Evaluate Axatilimab Versus Best Available Therapy in Participants With Chronic Graft Versus Host Disease After at Least 2 Prior Lines of Systemic Therapy

To be considered for this study, you need to be at least 12 years old and have moderate to severe chronic graft-versus-host-disease (cGVHD) that still needs treatment with medicines that suppress your immune system. You should also have tried at least two other types of treatment for your cGVHD before, including steroids and ruxolitinib.

You must have had a stem cell transplant in the past, regardless of the donor type or source. You'll also need to be willing to be treated with one of several standard 'Best Available Therapy' options if you are assigned to that group. If you are currently taking steroids, your dose needs to have been steady for at least two weeks before starting the study.

There are also some reasons you wouldn't be able to join the study. For example, if you've had more than one stem cell transplant (though a previous autologous transplant, using your own cells, is okay). You also can't be suffering from a relapse of your original blood condition or have severe kidney or liver problems. Certain other medical conditions, like active muscle inflammation or a history of severe pancreas inflammation, would also exclude you.

If you decide to take part in this study, you will either receive the new medicine, axatilimab, or one of the standard 'Best Available Therapy' options. Which treatment you receive will be decided randomly, like flipping a coin. You will have regular visits to the clinic for check-ups, blood tests, and other assessments to monitor your health and how well the treatment is working. This will involve physical examinations and potentially other tests to track your cGVHD.

The specific schedule of visits and assessments will be explained in detail by the study team, but generally, it involves ongoing monitoring throughout the treatment period. After your main treatment, there will likely be a follow-up period to see how you are doing in the longer term. The total duration of your participation in the study will depend on which treatment you receive and how you respond, but it will involve a commitment over several months, potentially longer.