A Phase I Study of HER2-Directed CAR T-Cell Therapy for People with Recurrent or Persistent Ependymoma


Full Title

Phase 1 Trial of Autologous HER2-specific CAR T cells in Pediatric Patients with Refractory or Recurrent Ependymoma (PBTC-059) (CIRB)


CAR T-cell therapy is a type of immunotherapy. With CAR T-cell therapy, white blood cells called T cells are removed from the patient, altered in the laboratory to recognize a protein on the patient’s cancer cells, multiplied to larger numbers, and returned to the patient to find and destroy cancer cells. The treatment is made from the modified T cells.

In this study, researchers are evaluating a CAR T-cell therapy directed toward a protein on cancer cells called HER2. They are assessing this treatment in children, adolescents, and young adults with a brain tumor called an ependymoma that has come back or continued to grow despite prior treatment.

Before patients receive the CAR T cells, they will have “conditioning” chemotherapy with fludarabine and cyclophosphamide chemotherapy to suppress the immune system and help prepare the body for receiving the CAR T cells. The treatments in this study are given intravenously (by vein).

Who Can Join

To be eligible for this study, patients must meet several requirements, including:

  • Participants must have an ependymoma that has come back or continued to grow despite treatment.
  • At least 4 weeks must pass since the completion of prior therapies and receipt of the study treatment, and patients must have recovered from any serious side effects.
  • Patients must be able to walk and do routine activities for more than half of their normal waking hours.
  • This study is for people ages 1-22.


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




Phase I (phase 1)

Disease Status

Relapsed or Refractory



ClinicalTrials.gov ID