A Phase II Study of Selinexor (KPT-330) in Patients with Persistent Myelodysplastic Syndromes

Full Title

A Phase II Study of the Selective Inhibitor of Nuclear Export (SINE) Selinexor (KPT-330) in Patients with Myelodysplastic Syndromes


The standard therapy for myelodysplastic syndromes (MDS) includes the drugs decitabine and 5-azacytidine. There are no other standard treatments available for patients whose MDS does not improve with these drugs. Doctors are therefore seeking more effective therapies.

Tumor suppressor proteins work inside normal cells to prevent the uncontrolled cell growth that can lead to cancer. Some cancer cells thrive by getting rid of tumor suppressor proteins. Selinexor (also known as KPT-330) is an investigational drug that works by trapping tumor suppressor proteins inside cancer cells, causing them to die or stop growing.

In this study, researchers want to see if selinexor can improve blood counts and bone marrow function in patients with MDS that is not responding to treatment with decitabine or 5-azacytidine. Selinexor is taken orally (by mouth).


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

  • Patients must have MDS that is not responding to treatment with decitabine or 5-azacytidine.
  • At least 3 weeks must pass between the completion of prior treatment and entry into the study.
  • Patients must be able to walk and do routine activities for more than half of their normal waking hours.
  • This study is for patients age 18 and older.

For more information about this study and to inquire about eligibility, please contact Dr. Virginia Klimek at 212-639-6519.