A Phase I/II Study of Ibrutinib in Children and Young Adults with Chronic Graft-versus-Host Disease


Full Title

Phase 1/2 Dose Finding, Safety and Efficacy Study of Ibrutinib in Pediatric Subjects with Chronic Graft Versus Host Disease (cGVHD)


Graft-versus-host disease (GVHD) is a potential complication that can occur when someone receives stem cells from a donor. It happens when white blood cells called T cells from the donor attack the tissues of the patient. Acute GVHD occurs shortly after the transplant, usually within three months. Chronic GVHD develops more slowly and occurs later.

The purpose of this study is to find the highest dose of ibrutinib that can be given safely in children with chronic GVHD (part A). Researchers also want to see how children’s and young adults’ bodies process the drug and see if it is effective against chronic GVHD (part B).

Ibrutinib blocks a protein called Bruton’s tyrosine kinase (BTK); this protein is believed to help cells multiply and survive. Blocking BTK may shut down the immune cells that cause chronic GVHD. Ibrutinib has been approved by the FDA to treat a number of blood cancers and other diseases, including chronic GVHD, in adults whose disease does not respond to one or more treatments. Ibrutinib has not been approved to treat chronic GVHD in children or in adults newly diagnosed with chronic GVHD. It is taken orally (by mouth).


To be eligible for this study, patients must meet several criteria, including but not limited to the following:

  • Patients in part A must have moderate or severe chronic GVHD after the failure of systemic therapy, and must be at least age 1 year but under age 12.
  • Patients in part B must have moderate or severe chronic GVHD that is new or persists despite prior treatment, and must be at least age 1 year but under age 22.

For more information about this study and to inquire about eligibility, please contact Dr. Susan Prockop at 212-639-6715.





Disease Status

Newly Diagnosed & Relapsed/Refractory