Shwachman-Diamond Syndrome clinical trials at UCSF
1 research study open to eligible people
Shwachman-Diamond Syndrome is a rare genetic disease that affects the pancreas and bones. UCSF is currently recruiting for a clinical trial for a treatment that helps improve bone marrow function. The trial tests whether treosulfan, fludarabine, and rabbit antithymocyte globulin can properly prepare patients for a blood or bone marrow transplant.
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 Shwachman-Diamond Syndrome research studies include Kristin Shimano, MD.