A Phase III Study Comparing Three Different Regimens to Prevent Chronic Graft-versus-Host Disease

Full Title

A Randomized, Multi-Center, Phase III Trial of Calcineurin Inhibitor-Free Interventions for Prevention of Graft-versus-Host Disease (BMT CTN 1301)

Purpose

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, and the drugs tacrolimus and methotrexate are typically used to prevent it. Chronic GVHD develops more slowly and occurs later than acute GVHD, but it is not prevented as well by tacrolimus and methotrexate.

Doctors are therefore seeking better ways to prevent chronic GVHD. In this study, patients receiving stem cells from a donor (“allogeneic transplantation”) as treatment for acute leukemia or myelodysplasia will be randomly assigned to receive one of three treatments designed to prevent chronic GVHD:


  1. CD34-selcted peripheral blood stem cell transplant, in which certain T cells have been removed from the donor’s stem cells

  2. Bone marrow transplant followed by treatment with the drug cyclophosphamide

  3. Bone marrow transplant followed by the conventional GVHD prevention therapy of tacrolimus and methotrexate.

Researchers will compare the first two investigational approaches with the third standard approach to see which is most effective.

Eligibility

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

  • Patients must have acute leukemia or myelodysplasia and be candidates for allogeneic stem cell transplantation.
  • Patients must have a related or unrelated donor.
  • This study is for patients aged 1-65 years.

For more information and to inquire about eligibility for this study, please contact Dr. Miguel-Angel Perales at 212-639-8682.

Protocol

14-263

Phase

III

Investigator

Locations