A Phase I Study of Venetoclax in Children and Young Adults with Persistent or Recurrent Cancers


Full Title

A Phase 1 Study of the Safety and Pharmacokinetics of Venetoclax in Pediatric and Young Adult Patients with Relapsed or Refractory Malignancies


The purpose of this study is to find the highest dose of the anticancer drug venetoclax that can be given safely in children and young adults with cancer that has come back or continued to grow despite prior treatment. Venetoclax is approved for treating chronic lymphocytic leukemia in adults; its use in this study is considered investigational.

Venetoclax blocks a protein called Bcl-2, a protein that helps cancer cells survive and resist the effects of anticancer treatments. By blocking Bcl-2, venetoclax could kill cancer cells and/or make them more vulnerable to the effects of other cancer treatments. It 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 under age 25 years.
  • Patients must have cancer (except primary or metastatic brain cancer) that came back or continued to grow despite prior treatment. Examples include leukemia and neuroblastoma.
  • Patients must recover from the serious side effects of previous 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