PEPN2415, A Phase I Study to Assess the Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of AZD1390 (NSC# 852149, IND# 172675) when Combined with Focal Radiation in Pediatric Patients with High Grade Glioma
PEPN2415
What is the goal of the study?
This study aims to improve the outcomes for patients with HGGs, including DIPG, which currently have dismal survival rates and are resistant to conventional therapies. ATM kinase is a key regulator of DNA damage response and a rational radiosensitization target. AZD1390 has demonstrated favorable pharmacokinetics and CNS penetrance in preclinical and adult phase 1 studies, making it a promising candidate for pediatric translation. By targeting ATM, this trial aims to enhance the efficacy of RT without increasing systemic toxicity. The results may inform future studies and may establish a novel therapeutic backbone for pediatric HGGs.
Who can participate in the study?
Newly diagnosed HGG/DMG including DIPG ages 1-18 (cohort A-B), 1-22 (Cohort C-D), excluding spinal tumors and metastatic disease