PNOC022: A Combination Therapy Trial using an Adaptive Platform Design for Children and Young Adults with Diffuse Midline Gliomas (DMGs) including Diffuse Intrinsic Pontine Gliomas (DIPGs) at Initial Diagnosis, Post-Radiation Therapy and at Time of Progression
PNOC022
What is the goal of the study?
Despite years of research and much knowledge gained about the underlying biology of diffuse midline gliomas (DMGs) – especially those with H3K27M mutations – little to no progress has been made to improve outcomes for these patients. The reasons for the ongoing failures are multi-factorial but include (a) limited preclinical data from a variety of model systems and validation in multiple laboratories, (b) limited knowledge about the blood brain barrier (BBB) penetration and (c) lack of multi-agent therapy plans. Within this clinical trial, we aim to address these key issues by working with several laboratories around the world to select the most promising combination therapy strategies, leveraging a target validation design to confirm BBB penetration and by offering each participants combination therapy approach. Given the limited treatment options for participants with DMGs, we also aim to offer trial enrollment at different disease stages – newly-diagnosed, after completion of radiation therapy, and at time of progression.
Who can participate in the study?
Please contact the study team listed below to learn more.