A Phase I Study of Entrectinib in Children and Adolescents with Persistent or Recurrent Solid Tumors in the Body or Brain

Full Title

A Phase 1/2, Open-Label, Dose-Escalation and Expansion Study of Entrectinib (RXDX-101) in Children and Adolescents with No Curative First-Line Treatment Option, Recurrent or Refractory Solid Tumors and Primary CNS Tumors, With or Without TRK, ROS1, or ALK Fusions


Entrectinib is an investigational drug that interferes with cancer growth in tumors containing certain genetic mutations. It has been evaluated in adults with cancer. In this study, researchers are seeking to find the best dose of entrectinib to use in children and adolescents with solid tumors in the body or brain that have come back or continued to grow despite prior treatment. Entrectinib is taken orally (by mouth).


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

  • Patients must be at least 2 years of age and less than age 22.
  • Patient must have neuroblastoma, brain cancer, or a solid tumor elsewhere in the body that came back or continued to grow despite previous treatment.
  • Patients must recover from the serious side effects of prior therapies before entering the study.

For more information about this study and to inquire about eligibility, please contact Dr. Ellen Basu at 212-639-5204.





Disease Status

Relapsed or Refractory