Ibrutinib + Indoximod

Indoximod will be taken by mouth twice daily, throughout each treatment cycle.

For Regimen A, Ibrutinib will be taken by mouth once daily, on days 1-21 of each treatment cycle.

The GCC2020 trial is a prospective open-label phase 1 trial to determine the best safe dose of the BTK-inhibitor ibrutinib to use in combination with previously studied chemo-immunotherapy regimens comprised of the investigational IDO-inhibitor indoximod plus oral palliative chemotherapy for participants, age 6 to 25 years, with relapsed or refractory primary brain cancer.

Recent lab-based discoveries suggest that IDO (indoleamine 2,3-dioxygenase) and BTK (Bruton's tyrosine Kinase) form a closely linked metabolic checkpoint in tumor-associated antigen-presenting cells.

The central clinical hypothesis for the GCC2020 study is that combining ibrutinib (BTK-inhibitor) with indoximod (IDO-inhibitor) during chemotherapy will synergistically enhance anti-tumor immune responses, leading to improvement in clinical response with manageable overlapping toxicity.

Live record on ClinicalTrials.gov →

• Patients must have prior documented progressive or refractory disease with histologically proven initial diagnosis of ependymoma, medulloblastoma, glioblastoma, or another type of primary cancer of the central nervous system with no curative conventional therapy options available.
• Metastatic disease is acceptable.
• Patients must have MRI confirmation (with and without gadolinium contrast) of current active disease.
• Patients must be able to swallow pills.
• Lansky or Karnofsky performance status score must be ≥ 50%.

Inclusion criteria as written on the registry, trimmed. The full list, with exclusions, is on the live record.