A Phase I/II Study of CX-4945 (a "Sonic Hedgehog Pathway Inhibitor) in Patients with Recurrent Sonic Hedgehog-type Medulloblastoma


Full Title

PBTC-053: A Pediatric Brain Tumor Consortium Phase I/II and Surgical Study of CX-4945 in Patients with Recurrent SHH Medulloblastoma (CIRB)


The purpose of this study is to assess the safety and effectiveness of the investigational drug CX-4945 (also called silmitasertib) in patients with a brain tumor called medulloblastoma that has come back after prior treatment. There are multiple parts to this study.

In the phase I part of the study, researchers will find the highest dose of CX-4945 that can be given safely in children with recurrent medulloblastoma. In the phase II part, researchers will assess the safety of CX-4045 in older adolescents and adults and see if the drug can shrink or stop tumor growth. A surgical study will include children and adults; it will determine how much CX-4945 is found in tumor tissue that is removed during surgery and see if the drug works against a specific target found in the tumor.

CX-4945 works by inhibiting certain enzymes that drive cancer growth. It is taken orally (by mouth).

Who Can Join

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

  • Patients must have a type of medulloblastoma called sonic hedgehog (SHH) that has come back after treatment.
  • Patients in the phase I part of the study must be age 3-18 and be skeletally immature (bones still growing).
  • Patients in the phase II part of the study must be skeletally mature (bones finished growing).
  • Patients in the surgical study must be at least 3 years of age and have an operable tumor.
  • All patients must have recovered from the serious side effects of prior treatment before entering the study.

For more information about this study and to inquire about eligibility, please contact Dr. Sameer Farouk Sait at 212-639-3973.




Phase I/II (phases 1 and 2 combined)

Disease Status

Relapsed or Refractory



ClinicalTrials.gov ID