试验设计、招募和数据报告方面的限制影响了癌症临床试验中药物评价的效率和可靠性。这些问题在神经肿瘤学中已被认识到，但尚未得到全面评估。我们对成人干预性神经肿瘤学试验进行了半自动化调查，研究设计、干预措施、结果和数据可用性趋势。试验是通过使用临床试验.gov中的原发性恶性中枢神经系统肿瘤分类术语进行程序选择的。回归分析评估了设计和招募趋势；效应大小分析利用调查生存率进行建模。 在评估了3038种试验后，大多数试验是非盲的（92％），单组（65％），非随机的（51％），研究胶质母细胞瘤（47％）或其他胶质瘤。大多数试验报告了基本设计要素，并随时间增加而增加（OR = 1.24，p <0.00001）。评估生存结果的试验估计假设干预措施具有大效应大小。42％的试验已完成；其中38％未达到招募目标，招募难度随时间恶化（R = -0.94，p <0.00001），对美国和非美国试验的招募均如此（OR = 0.5，p <0.00001）。28％的已完成试验报告了部分结果，美国试验报告更多（34.6％）比非美国试验（9.3％，p <0.00001）报告更多。已完成试验报告生存结果的15-23％检测到了疗效信号。 低随机化率、控制使用不足和效应大小的过高估计，特别是在早期试验中尤为显著，影响结果的普遍性。不完善的设计可能是由于招募挑战而产生的，强调了合作努力和新设计的必要性。有限的结果报告凸显了激励数据报告和协调以实现历史数据驱动研究的必要性。由牛津大学出版社代表神经肿瘤学协会2023年出版。此作品由美国政府雇员撰写，属于公共领域。

Limitations in trial design, accrual, and data reporting impact efficient and reliable drug evaluation in cancer clinical trials. These concerns have been recognized in neuro-oncology but have not been comprehensively evaluated. We conducted a semi-automated survey of adult interventional neuro-oncology trials, examining design, interventions, outcomes, and data availability trends.Trials were selected programmatically from ClinicalTrials.gov using primary malignant CNS tumor classification terms. Regression analyses assessed design and accrual trends; effect size analysis utilized survival rates among trials investigating survival.Of 3038 reviewed trials, most were non-blinded (92%), single-group (65%), non-randomized (51%), and studied glioblastomas (47%) or other gliomas. Basic design elements were reported by most trials, with reporting increasing over time (OR=1.24, p<0.00001). Trials assessing survival outcomes were estimated to assume large effect sizes of interventions when powering their designs. 42% of trials were completed; of these, 38% failed to meet their enrollment target, with worse accrual over time (R=-0.94, p<0.00001) and for US versus non-US based trials (OR=0.5, p<0.00001). 28% of completed trials reported partial results, with greater reporting for US (34.6%) versus non-US based trials (9.3%, p<0.00001). Efficacy signals were detected by 15-23% of completed trials reporting survival outcomes.Low randomization rates, underutilization of controls, and overestimation of effect size, particularly pronounced in early-phase trials, impede generalizability of results. Suboptimal designs may be driven by accrual challenges, underscoring the need for cooperative efforts and novel designs. The limited results reporting highlights the need to incentivize data reporting and harmonization to enable historical-data driven studies.Published by Oxford University Press on behalf of the Society for Neuro-Oncology 2023. This work is written by (a) US Government employee(s) and is in the public domain in the US.