A Phase II Study of Stem Cell Transplantation Using Treosulfan Conditioning to Treat Bone Marrow Failure


Full Title

Hematopoietic Cell Transplantation Using Treosulfan-Based Conditioning for the Treatment of Bone Marrow Failure Diseases (BMT CTN) (NMDP)


Chemotherapy is given as part of a “conditioning regimen” before a person with bone marrow failure receives a stem cell transplant. In this study, researchers want to see if adding the chemotherapy drug treosulfan to the conditioning regimen will cause fewer complications and result in a more successful transplant than standard chemotherapy without treosulfan. Other studies using treosulfan show that it causes less damage to organs while being strong enough for transplanted stem cells to grow.

Participants in this study will receive treosulfan with the drugs fludarabine and rATG before stem cell transplantation. These medications are given intravenously (by vein).

Who Can Join

To be eligible for this study, patients must meet several requirements, including:

  • Participants must have bone marrow failure and be planning to have a stem cell transplant from a donor.
  • This study is for people ages 1-49.

For more information and to ask about eligibility for this study, please contact the office of Dr. Maria Cancio at 212-639-2446.




Phase II (phase 2)



ClinicalTrials.gov ID