A Phase I Study of Idasanutlin Alone or with Chemotherapy in Pediatric and Young Adult Patients with Recurrent or Persistent Solid Tumors


Full Title

A Phase I/II Multicenter, Open-Label, Multi-Arm study Evaluating the Safety, Tolerability, Pharmacokinetics, and Preliminary Activity of Idasanutlin in Combination with Either Chemotherapy or Venetoclax in the Treatment of Pediatric and Young Adult Patients with Relapsed/Refractory Acute Leukemias or Solid Tumors


The purpose of this study is to find the highest dose of the investigational drug idasanutlin that can be given safely alone or combined with chemotherapy in children and young adults with childhood cancers that have come back or continued to grow despite prior treatment. Idasanutlin works by blocking a protein on cancer cells called MDM2, which cancer cells need to grow.

Patients in this study will receive idasanutlin alone or in combination with cyclophosphamide and topotecan, which are standard chemotherapy drugs. Idasanutlin is taken orally (by mouth) and the chemotherapy drugs are given intravenously (by vein).

Who Can Join

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

  • Patients must have neuroblastoma or another solid tumor that has continued to grow or came back despite previous therapy.
  • Patients with brain tumors may not participate.
  • Patients should recover from the serious side effects of prior treatments before entering the study.
  • This study is for patients under age 30.

For more information about this study and to inquire about eligibility, please contact 1-833-MSK-KIDS.




Phase I/II (phases 1 and 2 combined)

Disease Status

Relapsed or Refractory


ClinicalTrials.gov ID