Shwachman-Diamond Syndrome clinical trials at UCSF

1 research study open to eligible people

Shwachman-Diamond syndrome is a rare disorder that affects the bone marrow and pancreas. UCSF is studying a new treatment using treosulfan and fludarabine before a bone marrow transplant. This trial aims to improve outcomes for patients with bone marrow failure diseases.

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  • 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 .

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