A Phase 2 Pivotal Study of BCB-276, a B7-H3-Specific CAR T Cell Locoregional Immunotherapy for Diffuse Intrinsic Pontine Glioma
This study will evaluate BCB-276, an investigational B7-H3-targeted Chimeric Antigen Receptor (CAR) T cell therapy, in children and young adults with diffuse intrinsic pontine glioma (DIPG). DIPG is a rare and aggressive brain tumor with limited treatment options. CAR T cell therapy uses a patient's own immune cells that are changed in a laboratory to recognize and attack cancer cells. The purpose of this study is to determine whether BCB-276, when given after completion of standard radiation therapy, is safe and can improve survival for patients with DIPG. To participate, individuals must be between 1 and 26 years of age when they join the study, have a diagnosis of DIPG, and enroll for treatment within 6 weeks of completing initial radiation therapy. Participants must not have received prior anti-cancer therapy beyond radiation with or without temozolomide prior to joining this study. BCB-276 is administered intraventricularly (into the fluid around the brain), which requires placement of a catheter for treatment. BCB-276 is given every 2 weeks at a research center over a period of several months (approximately 7-8 months). Participation includes travel to a study site, procedures to support treatment administration, sample collection, and ongoing monitoring for safety and effectiveness, with follow-up visits lasting up to about 2 years.
• Participants must be aged 1 and ≤ 26 years and weigh ≥10kg.
• Diagnosis of Diffuse Intrinsic Pontine Glioma (DIPG) based on imaging, with or without biopsy confirmation consistent with high-grade glioma or diffuse midline glioma
• Able to tolerate leukapheresis and other study procedures in the opinion of the Investigator.
• Central Nervous System (CNS) reservoir catheter present prior to first dose of study drug.
• Participant must have completed standard radiation therapy within 6 weeks of enrollment for participation.
• Performance Status of ≥ 60; mild to moderate restriction or better. Lansky (under 16 years of age) or Karnofsky (16 years of age or older).
• Adequate organ function and overall clinical status to participate, including stable or improving neurologic symptoms.
• Participants of childbearing/fathering potential must agree to use highly effective contraception from the time of enrollment through 12 months following the last T cell infusion.
• Participants with ventriculoperitoneal (VP) shunts need to have a programable system and be able to tolerate temporary adjustment of the shunt required for study treatment.
• Participant must meet all other health and safety criteria defined in the study protocol.
• Participant and/or authorized legal representative willing to provide consent/assent for study participation, including participation in the 15-year long term follow up period.