A Phase IB/II Study of Vismodegib in Patients with Persistent or Recurrent Acute Myelogenous Leukemia or Myelodysplastic Syndromes

Full Title
A Phase IB/II Study to Evaluate the Safety and Efficacy of Vismodegib in Relapsed/Refractory Acute Myelogenous Leukemia (AML) and Relapsed/Refractory High-Risk Myelodysplastic Syndrome (MDS)
Disease Status

Vismodegib is a drug approved for a form of skin cancer called basal cell carcinoma. It works by targeting a protein pathway called the “hedgehog” pathway. Vismodegib inhibits SMO, a protein which fuels the growth of some cancers. It is a capsule that is taken orally (by mouth).

In this study, researchers are assessing the safety and effectiveness of vismodegib in patients with acute myelogenous leukemia (AML) or myelodysplastic syndromes (MDS) that have persisted or returned despite other treatment. Laboratory studies have shown that vismodegib may kill leukemia cells by blocking the hedgehog pathway, which is important for leukemia growth.


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

  • Patients must have AML or MDS that has persisted or returned despite other treatment.
  • At least 2 weeks must have passed since completion of prior treatment and entry into the study.
  • Patients may not have had prior therapy that included a drug that targets the hedgehog pathway.
  • Patients must be able to be ambulatory for more than half of their normal waking hours.
  • This study is open to patients age 18 and older.

For more information about this study and to inquire about eligibility, please contact Dr. Martin Tallman at 212-639-3842.

Hematology: Myelodysplastic Syndrome
Leukemia: Acute Myelocytic Leukemia
Leukemia: Acute Myelogenous Leukemia