Klas Katarzyna, Strzebonska Karolina, Buedo Paola, Włodarczyk Alicja, Gordon Samuel, Kaszuba Paulina, Polak Maciej, Waligora Marcin
Faculty of Health Sciences, Research Ethics in Medicine Study Group (REMEDY), Jagiellonian University Medical College, Krakow, Poland.
Doctoral School of Medical and Health Sciences, Jagiellonian University Medical College, Krakow, Poland.
PLoS One. 2024 Dec 3;19(12):e0312797. doi: 10.1371/journal.pone.0312797. eCollection 2024.
Seamless clinical trials have received much attention as a possible way to expedite drug development. The growing importance of seamless design can be seen in oncology research, especially in the early stages of drug development. Our objective is to examine the basic characteristics of seamless early-phase oncology trials registered on the ClinicalTrials.gov database and to determine their results reporting rates. We also aim to identify factors associated with results reporting.
Cross-sectional study. We defined seamless early-phase trials as either those registered as Phase 1/2 or Phase 1 with planned expansion cohort(s). Using the ClinicalTrials.gov registry, we searched for interventional cancer clinical trials with primary completion date (PCD) between 2016 and 2020. After trial selection, we performed manual data extraction based on the trial record description and the results posted in the trial registry. We used logistic regression to search for predictors of results reporting. Protocol: https://osf.io/m346x/.
We included 1051 seamless early-phase oncology trials reported as completed (PCD) between 2016 and 2020. We provided descriptive statistics including the number of patients enrolled, study start date, primary completion date, funding, type of intervention, cancer type, design details, type of endpoints, recruitment regions, and number of trial sites. Overall, only 34.7% trials reported results on ClinicalTrials.gov. The results reporting rates for 24 months was 24.0%. The overall reporting rate for Phase 1/2 studies was over three times higher than for seamless Phase 1.
Our study provides cross-sectional data on seamless early-phase oncology trials registered on ClinicalTrials.gov. We highlight the challenges of the evolving clinical trial design landscape and the problem of missing results in the seamless design context, which raises serious ethical concerns. Efforts should be made to adapt the functionality of the ClinicalTrials.gov database to emerging clinical trial models.
无缝临床试验作为加快药物研发的一种可能方式受到了广泛关注。无缝设计在肿瘤学研究中日益重要，尤其是在药物研发的早期阶段。我们的目的是研究在ClinicalTrials.gov数据库中注册的无缝早期肿瘤学试验的基本特征，并确定其结果报告率。我们还旨在识别与结果报告相关的因素。
横断面研究。我们将无缝早期试验定义为注册为1/2期或有计划扩展队列的1期试验。利用ClinicalTrials.gov注册库，我们搜索了主要完成日期（PCD）在2016年至2020年之间的介入性癌症临床试验。试验筛选后，我们根据试验记录描述和试验注册库中公布的结果进行手动数据提取。我们使用逻辑回归来寻找结果报告的预测因素。方案：https://osf.io/m346x/。
我们纳入了2016年至2020年期间报告已完成（PCD）的1051项无缝早期肿瘤学试验。我们提供了描述性统计数据，包括入组患者数量、研究开始日期、主要完成日期、资金、干预类型、癌症类型、设计细节、终点类型、招募地区和试验站点数量。总体而言，只有34.7%的试验在ClinicalTrials.gov上报告了结果。24个月时的结果报告率为24.0%。1/2期研究的总体报告率比无缝1期高出三倍多。
我们的研究提供了在ClinicalTrials.gov上注册的无缝早期肿瘤学试验的横断面数据。我们强调了不断演变的临床试验设计格局带来的挑战以及无缝设计背景下结果缺失的问题，这引发了严重的伦理问题。应努力使ClinicalTrials.gov数据库的功能适应新兴的临床试验模式。
