Abstract Background and aims Despite advances in molecular characterization, CNS tumours remain the leading cause of cancer-related mortality in children, underscoring the need for more clinical research. This study analyses the current clinical trial landscape for the two most common paediatric CNS tumour entities: gliomas and embryonal tumours. Methods A comprehensive analysis was conducted using ClinicalTrials.gov dataset with the following keywords: high-grade glioma, low-grade glioma, diffuse midline glioma, diffuse intrinsic pontine glioma, CNS embryonal tumors, medulloblastoma, and atypical teratoid/rhabdoid tumor. All trials involving these tumor entities and initiated since January 1, 2010, were included. Trial characteristics were extracted and analyzed using descriptive statistics. Results As of the data cutoff point on January 1, 2026, 379 clinical trials were identified, including 238 glioma, 71 CNS embryonal tumor, and 70 multi-entity trials. Most trials were interventional (89.5%) and treatment-oriented (85.8%). Among these trials 283 were early phase with only 18 phase 3 trials. Trials were evenly distributed between multicenter (53.9%) and single-center designs (46.1%). At the time of analysis, 181 trials were active, 118 had been completed, and 53 were terminated, with slow recruitment accounting for 19.61% of terminations. Randomization was applied in only 48 trials. Outcome reporting was limited, with results available for 70 trials. All studies reporting outcomes were interventional, while no observational studies reported results (20.64% vs 0%, p = 0.001). Multicenter trials demonstrated higher rates of result reporting compared with single-center trials (25.87% vs 9.88%, p = 0.001). Importantly, only 39 trials exclusively enrolled pediatric patients (ages 0–17). Conclusions Despite the biological distinctiveness of pediatric CNS tumors, most trials enroll both pediatric and adult populations. Even among pediatric studies, few are tumor-specific, complicating result interpretation. The predominance of early-phase, non-randomized designs, along with slow recruitment and limited reporting, underscores the urgent need for multicenter, pediatric-focused, disease-specific trials to improve outcomes in pediatric CNS tumors.
Hoveyan et al. (Tue,) studied this question.