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Venetoclax M13-833 (hematologic/solid tumors)

Clinical study of the safety and efficacy of the drug venetoclax in children and young adults.
Who can enter
  • Children and young adults with cancer who relapsed or did not respond to treatment (refractory), and who have no other treatment options.
  • Age: <25 years.

The goal of this study is to find out how safe and effective the new agent venetoclax is for the treatment of different types of childhood cancer.


Cancer cells can contain excessive amounts of a specific protein, called BCL-2. This protein helps cancer cells to survive. The drug venetoclax blocks BCL-2, so that the cancer cells are no longer protected from dying.

Venetoclax has been approved (registered) for the treatment of adults with chronic lymphocytic leukemia (CLL) with a specific chromosomal abnormality (17p). Currently, this drug is being tested in other types of cancer in adults, including acute myeloid leukemia (AML), non-Hodgkin lymphoma (NHL), and multiple myeloma (MM). The first results indicate that venetoclax is active in all these types of cancer.

Venetoclax has not yet been investigated in children. In this study we will examine whether children and young adults respond to the treatment with venetoclax, and which side effects can occur due to the treatment. In addition, we will measure in the blood how much of the investigative agent is taken up by the body at different time points after administration (this is called pharmacokinetics). We will also determine how safe and effective the new agent is in combination with chemotherapy.

In order to participate in a study please refer to your/your child’s doctor.

Last reviewed

February 21, 2022