Title: Repurposing ibrutinib for chemo-immunotherapy in a phase 1b study of ibrutinib with indoximod plus metronomic cyclophosphamide and etoposide for pediatric patients with brain cancer

Principal Investigator: Theodore S. Johnson, MD, PhD, of Augusta University in Georgia

Description: Researchers are conducting a phase 1 trial to determine the safety and efficacy of ibrutinib in combination with indoximod, cyclophosphamide, and etoposide for pediatric and young adult patients with relapsed or refractory ependymoma, medulloblastoma, primitive neuroectodermal tumor (PNET), or glioblastoma.

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Patients will receive the 4-drug oral regimen for a maximum of 12 cycles. Cycles are a minimum of 28 days.

Primary outcome measures include objective response rate and the incidence of regimen-limiting toxicity to determine the recommended phase 2 dose of ibrutinib. Key secondary outcome measures include adverse events, progression-free survival, and overall survival.

To be eligible for this study, patients must be 12 to 25 years of age. They must also have documented progressive disease and a histologically proven diagnosis of ependymoma, medulloblastoma, PNET, or glioblastoma. For a full list of eligibility criteria, please see the reference.

Status: Recruiting

This study is sponsored by Theodore S. Johnson in collaboration with Augusta University.


ClinicalTrials.gov. Chemo-immunotherapy using ibrutinib plus indoximod for patients with pediatric brain cancer. NCT05106296. Accessed June 17, 2022.