A Phase I Study of Selinexor to Treat Persistent and Recurrent Brain Tumors and Other Solid Tumors in Children, Teens, and Young Adults


Full Title

A Phase 1 Study of Selinexor (KPT-330, IND #125052), A Selective XPO1 Inhibitor, in Recurrent and Refractory Pediatric Solid Tumors, Including CNS Tumors (ADVL1414) (CIRB)


Proteins that suppress tumor growth work inside the nucleus of normal cells to turn genes on and off and thereby prevent the uncontrolled cell growth that can lead to cancer. Some cancer cells thrive by pumping the tumor suppressor proteins out of the nucleus, where they can no longer work. Selinexor is a drug that works by trapping tumor suppressor proteins inside the nucleus of cancer cells, causing them to die or stop growing. It is used to treat multiple myeloma and a type of lymphoma in adults.

Researchers want to know if selinexor is safe to use in children, teens, and young adults with cancer. In this study, they want to find the best dose of selinexor to use safely in young patients with brain and spinal cord tumors and other solid tumors that continue to grow or have come back despite prior treatment. Selinexor is taken orally (by mouth).


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

  • This study is for patients ages 1-21 years.
  • Patients must have a brain tumor (such as high-grade glioma), spinal cord tumor, or other solid tumor that has continued to grow or came back after previous treatment.
  • Patients must recover from the serious side effects of prior therapies before receiving selinexor.
  • Patients must be able to walk and do routine activities for more than half of their normal waking hours.

For more information about this study and to inquire about eligibility, please contact Dr. Julia Glade Bender at 212-639-2972.





Disease Status

Relapsed or Refractory