Congenital Amegakaryocytic Thrombocytopenia clinical trials at UCSF
1 research study open to eligible people
Congenital amegakaryocytic thrombocytopenia is a rare condition that causes low levels of platelets in the blood. If left untreated, it can cause severe bleeding and other complications. UCSF is currently recruiting participants for a clinical trial testing a new treatment for bone marrow failure diseases, including this condition. The trial will test whether treosulfan, fludarabine, and rabbit antithymocyte globulin can help patients with this and other disorders receive successful blood or bone marrow transplants.
Treosulfan-Based Conditioning Regimen Before a Blood or Bone Marrow Transplant for the Treatment of Bone Marrow Failure Diseases (BMT CTN 1904)
open to eligible people ages 1-49
This phase II trial tests whether treosulfan, fludarabine, and rabbit antithymocyte globulin (rATG) work when given before a blood or bone marrow transplant (conditioning regimen) to cause fewer complications for patients with bone marrow failure diseases. Chemotherapy drugs, such as treosulfan, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Fludarabine may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. rATG is used to decrease the body's immune response and may improve bone marrow function and increase blood cell counts. Adding treosulfan to a conditioning regimen with fludarabine and rATG may result in patients having less severe complications after a blood or bone marrow transplant.
San Francisco, California and other locations
Our lead scientists for Congenital Amegakaryocytic Thrombocytopenia research studies include Kristin Shimano, MD.