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Idasanutlin (acute leukemia/solid tumors)

Clinical study of idasanutlin in combination with chemotherapy or venetoclax in children who have unresponsive or recurrent acute leukemia or solid tumors.
Who can enter
  • Children who have unresponsive or recurrent acute leukemia or solid tumors
  • Age: younger than 18 years


The goal of this study is to determine the best dose of idasanutlin for children and young adults when it is given in combination with chemotherapy or another medicine called venetoclax. Another purpose is to assess the effect and side effects of idasanutlin in combination with chemotherapy or venetoclax.


Idasanutlin is an experimental medicine. This means that health authorities have not yet approved idasanutlin for use in children or adults. Laboratory evidence supports the activity of idasanutlin in pediatric cancers, and idasanutlin has been investigated in more than 500 adult patients; however, this is the first study of idasanutlin in pediatric patients.

Idasanutlin is not a chemotherapy. Idasanutlin is a targeted inhibitor of a protein called MDM2, which is sometimes found in higher levels in cancer cells. Targeted cancer therapies are designed to target a specific protein in a cancer cell that may play a role in promoting a cancer’s resistance to treatment. This means that inhibiting MDM2 with idasanutlin is expected to promote the death of the cancer cells and may help other medicines to work better to treat the cancer cells.

Venetoclax has been approved for the treatment of adult patients with chronic lymphocytic leukemia and acute myeloid leukemia, but not for children. Venetoclax is a medicine which inhibits a protein called BCL-2, which is expected to have an effect on tumor growth and responsiveness to chemotherapy. In this study, venetoclax is also a study medicine. This means that health authorities have still not approved venetoclax for the treatment of children, also not in combination with idasanutlin.

Venetoclax and the chemotherapies in this study have been studied previously in pediatrics, and there is support for the activity of these treatment combinations from data in the laboratory. However, this is the first evaluation of these treatment combinations in pediatric patients.

In order to participate in a study please refer to your/your child’s doctor.
For international patients: please feel welcome to contact our International Office.

Last reviewed

September 19, 2023