A Phase I Study of Carfilzomib plus Cyclophosphamide and Etoposide for Children with Recurrent or Persistent Solid Tumors and Leukemias


Full Title

Phase I Study of Carfilzomib in Combination with Cyclophosphamide and Etoposide For Children with Relapsed and Refractory Solid Tumors and Leukemias


The purpose of this study is to evaluate the safety of different doses of carfilzomib in children and young adults with recurrent or persistent cancers when given in combination with cyclophosphamide and etoposide. Carfilzomib is used to treat multiple myeloma in adults; its use in this study is considered investigational. Cyclophosphamide and etoposide are standard treatments for children and young adults with cancer.

Carfilzomib is a type of drug called a proteasome inhibitor. It works by preventing the breakdown of abnormal proteins in cells, causing these cells to die. Cancer cells are more sensitive to these effects than normal cells. In laboratory studies, the effects of carfilzomib on cancer cells were enhanced when used in combination with etoposide. Similar effects have been seen with bortezomib, another proteasome inhibitor, when given with cyclophosphamide. All three drugs in this study are given intravenously (by vein).


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

  • This study is for patients age 6 months to 29 years.
  • Patients must have either leukemia that has come back two or more times OR a solid tumor that has come back or continued to grow despite standard therapy (or for which no standard treatments exist).
  • Patients with primary or metastatic brain tumors (except lymphoma) may not participate.
  • Patients must 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 1-833-MSK-KIDS.





Disease Status

Relapsed or Refractory