A Phase 2 Study of Axatilimab Plus Ruxolitinib in Adults and Children With Chronic Graft-Versus-Host Disease

Full Title

A Phase 2, Open-Label, Randomized, Multicenter Study to Evaluate the Safety and Efficacy of Axatilimab in Combination With Ruxolitinib in Participants With Newly Diagnosed Chronic Graft-Versus-Host Disease

Purpose

With chronic graft-versus-host disease (cGVHD), healthy stem cells from a donor see the recipient’s healthy cells as foreign and attack. This commonly happens about 100 days after a stem cell transplant, but it can happen at any time.

Researchers want to see if a steroid-free approach using ruxolitinib, with or without axatilimab, works well to treat cGVHD. Ruxolitinib blocks JAK proteins, which may reduce inflammation (swelling) and immune system reactions related to GVHD. Axatilimab blocks a protein called CSF-1R that controls white blood cells which play a role in GVHD.

If you join this study, you will be randomly assigned to get one of these treatments:

• Axatilimab and ruxolitinib. Axatilimab is given intravenously (by vein) and ruxolitinib is taken orally (by mouth).
• Ruxolitinib alone.
• Corticosteroids alone (methylprednisolone or prednisone, taken orally).

Who Can Join

To join this study, there are a few conditions. You or your child must:

• Have moderate to severe cGVHD that has not yet been treated.
• Be able to walk and do routine activities for more than half the time you are awake.
• Be age 12 or older.

Contact

For more information or to see if you or your child can join this study, please call Dr. Doris Ponce’s office at 646-608-3739.