Stanford University School of Medicine, Stanford, CA, United States of America; Department of Radiation Oncology, Massachusetts General Hospital, Boston, MA, United States of America.
Stanford University School of Medicine, Stanford, CA, United States of America; Division of Urology, Department of Surgery, Brigham and Women's Hospital, Boston, MA, United States of America.
J Neurol Sci. 2021 Sep 15;428:117579. doi: 10.1016/j.jns.2021.117579. Epub 2021 Jul 14.
Increasing neurological disease burden and advancing treatment options require clinical trials to expand the evidence base of clinical care. We aimed to characterize neurology clinical trials registered between October 2007 and April 2018 and identify features associated with early discontinuation and results reporting.
We compared 16,994 neurology (9.4%) and 163,714 non-neurology comparison trials registered to ClinicalTrials.gov. Trials therapeutic focus within neurology was assigned via combination programmatic and manual review. We performed descriptive analyses of trial characteristics, cox regression of early discontinuation, and multivariable logistic regression for results reporting within 3 years of completion.
Most neurology trials were academic-funded (58.5%) followed by industry (31.9%) and US-government (9.6%). Neurology trials focused more on treatment than prevention compared to non-neurology studies. Of neurology trials, 11.3% discontinued early, and 32.2% of completed trials reported results by April 30, 2018. In multivariable analysis accounting for time-to-event, neurology trials were at lower risk of discontinuation than non-neurology trials (adjusted hazard 0.83, p < 0.0001). Both academic and government-funded trials had greater risk of discontinuation than industry (adjusted hazard 0.57 and 0.46, respectively). Among completed trials, government-funded studies (adjusted odds ratio 2.12, p < 0.0001) had highest odds of results reporting while academic trials reported less (adjusted odds ratio 0.51, p < 0.0001).
Funding source is associated with trial characteristics and outcomes in neurology. Improvements in trial completion and timely dissemination of results remain urgent goals for the field.
神经疾病负担的增加和治疗选择的进步需要临床试验来扩大临床护理的证据基础。我们旨在描述 2007 年 10 月至 2018 年 4 月期间注册的神经科临床试验，并确定与早期终止和结果报告相关的特征。
我们比较了在 ClinicalTrials.gov 注册的 16994 项神经科（9.4%）和 163714 项非神经科对照试验。通过程序和手动相结合的方法审查试验治疗重点。我们对试验特征进行描述性分析，对早期终止进行 Cox 回归，对完成后 3 年内的结果报告进行多变量逻辑回归。
大多数神经科试验是由学术机构资助的（58.5%），其次是工业界（31.9%）和美国政府（9.6%）。与非神经科研究相比，神经科试验更侧重于治疗而非预防。在神经科试验中，有 11.3%提前终止，在已完成的试验中，有 32.2%在 2018 年 4 月 30 日前报告了结果。在多变量分析中，考虑到时间事件，神经科试验的终止风险低于非神经科试验（调整后的危险比 0.83，p<0.0001）。与工业界相比，学术和政府资助的试验的终止风险更高（调整后的危险比分别为 0.57 和 0.46）。在已完成的试验中，政府资助的研究（调整后的优势比 2.12，p<0.0001）报告结果的可能性最高，而学术试验报告的结果较少（调整后的优势比 0.51，p<0.0001）。
资金来源与神经科临床试验的特点和结果有关。提高试验完成率和及时传播结果仍然是该领域的紧迫目标。
