A Phase I Study of Pomalidomide in Children with Recurrent or Progressive Brain or Spine Tumors

Full Title

A Phase I Trial of Pomalidomide for Children with Recurrent, Progressive or Refractory CNS Tumors (PBTC-043)


Pomalidomide is a drug used to treat adults with a cancer called multiple myeloma. Doctors believe it could be active against brain tumors because it may block or change substances these tumors produce that help them survive and grow. It may also help the immune system fight the tumor.

In this study, researchers want to find the highest dose of pomalidomide that can be given safely in children with brain and spine tumors that have come back or continued to grow despite prior treatment. Pomalidomide is a capsule that 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 be at least 3 years old and under age 21.
  • Patient must have a brain or spine tumor that has come back or continued to grow despite other treatments.
  • Patients should recover from the serious side effects of prior treatments before entering the study.

For more information about this study and to inquire about eligibility, please contact Dr. Kevin De Braganca at 212-639-3449.





Disease Status

Relapsed or Refractory