We present the rationale and design of a master protocol study that clarifies the effectiveness and safety of Chinese herbal formulas on Qi-stagnation and blood-stasis pattern(QBP).Three randomized controlled trials(R...We present the rationale and design of a master protocol study that clarifies the effectiveness and safety of Chinese herbal formulas on Qi-stagnation and blood-stasis pattern(QBP).Three randomized controlled trials(RCTs)and real-world observational studies.Based on three registry cohorts of stable angina,tension-type headache and primary dysmenorrhea,patients with QBP will be enrolled in RCTs to receive either Xuefu Zhuyu(血府逐瘀,XFZY)oral liquid or a placebo,while patients with non-QBP will be enrolled in the observational studies and experience follow-up.1414 patients(RCTs:574;observational studies:840)will be recruited at seven centers in China over a 3-year period.The primary outcome is the visual analog scale of pain intensity.Adverse events will also be reported.The analysis will be undertaken separately in each sub-study,and then an overall analysis combining multiple subgroups will be performed to comprehensively investigate the effect of XFZY oral liquid.This study will provide high-quality evidence of XFZY oral liquid for QBP patients and show a paradigm of post-marketing evaluation of the effectiveness and safety for Chinese medicine following the notion of the pattern dominating different disease research models.展开更多
This review examines the clinical trial designs in rare mutation tumors,defined as mutations occurring in≤5%of specific cancer types.It discusses molecular characteristics,the impact of histological types,and the com...This review examines the clinical trial designs in rare mutation tumors,defined as mutations occurring in≤5%of specific cancer types.It discusses molecular characteristics,the impact of histological types,and the complexities of trial conduct due to tumor heterogeneity and limited patient numbers.The article explores accelerated approval pathways,adaptive trial methods like basket and telescope designs,and the use of surrogate endpoints.It underscores the significance of international collaboration and technological advancements in advancing research for these tumors.The review concludes that rare mutation tumor presents a novel prospective for oncological researches and facilitate the precision treatment of tumors.展开更多
基金the National Key Research and Development Program of China:Research on“the Pattern Dominating Disease”of Postmarket Evaluation on Two Classic Chinese Herbal Formulas based on Basket Design(No.2018YFC1707407)。
文摘We present the rationale and design of a master protocol study that clarifies the effectiveness and safety of Chinese herbal formulas on Qi-stagnation and blood-stasis pattern(QBP).Three randomized controlled trials(RCTs)and real-world observational studies.Based on three registry cohorts of stable angina,tension-type headache and primary dysmenorrhea,patients with QBP will be enrolled in RCTs to receive either Xuefu Zhuyu(血府逐瘀,XFZY)oral liquid or a placebo,while patients with non-QBP will be enrolled in the observational studies and experience follow-up.1414 patients(RCTs:574;observational studies:840)will be recruited at seven centers in China over a 3-year period.The primary outcome is the visual analog scale of pain intensity.Adverse events will also be reported.The analysis will be undertaken separately in each sub-study,and then an overall analysis combining multiple subgroups will be performed to comprehensively investigate the effect of XFZY oral liquid.This study will provide high-quality evidence of XFZY oral liquid for QBP patients and show a paradigm of post-marketing evaluation of the effectiveness and safety for Chinese medicine following the notion of the pattern dominating different disease research models.
基金supported by the CAMS Innovation Fund for Medical Sciences(CIFMS)under Award Number 2023-I2M-3-012 to Y.B.
文摘This review examines the clinical trial designs in rare mutation tumors,defined as mutations occurring in≤5%of specific cancer types.It discusses molecular characteristics,the impact of histological types,and the complexities of trial conduct due to tumor heterogeneity and limited patient numbers.The article explores accelerated approval pathways,adaptive trial methods like basket and telescope designs,and the use of surrogate endpoints.It underscores the significance of international collaboration and technological advancements in advancing research for these tumors.The review concludes that rare mutation tumor presents a novel prospective for oncological researches and facilitate the precision treatment of tumors.
