Clinical trials for diffuse intrinsic pontine glioma: the current state of affairs.

To date, 95 clinical trials investigating DIPG with specific emphasis have been registered on ClinicalTrials.gov. There were only a small number of trials that had study results available, and they uniformly reported non-significant improvement to prognosis. Given the rarity and lethality of DIPG, which limits the accumulation of large cohorts, our results mandate the need for more robust, systematic clinical trial design to minimize redundancies and maximize yield in the future.