目的 以双黄升白口服液Ⅱ期临床试验设计方案为例，探索中成药治疗肺癌化疗后中性粒细胞（ANC）减少的临床试验方案的设计，为治疗肿瘤化疗后出现ANC减少的中药新药研发提供参考。方法 双黄升白口服液Ⅱ期临床试验设计方案：以晚期肺癌或肺癌术后化疗导致1、2级ANC减少的精气两亏证患者为研究对象，采用随机、双盲、安慰剂平行对照、多中心的临床试验方法，将240例患者以1∶1比例分到试验组和对照组，试验组予双黄升白口服液、对照组予双黄升白口服液模拟剂，疗程为21 d。以ANC维持与复常为主要疗效指标；将ANC和白细胞（WBC）计数减少的发生率、ANC曲线下面积、粒细胞减少性发热（FN）的发生率、重组人粒细胞集落刺激因子（rhG-CSF）注射液使用量、中医证候疗效改变以及FACT-L量表评分变化作为次要疗效指标；以不良事件作为安全性指标。结果 双黄升白口服液Ⅱ期临床试验设计方案将疾病共性规律与患者个体特征相结合，在方法学上病证结合评定疗效，客观评价药物的有效性与安全性，具备实用性和科学性。结论 该方案既体现了中医学治疗思维和疗效优势，又适应了循证理念，成果具有外推性，为规范临床试验方法和推动中医药走向国际公认化做出了有益探索。

Objective To explore the design of the clinical trial protocol of Chinese traditional medicine for the treatment of absolute neutrophil count (ANC) reduction after chemotherapy for lung cancer by taking the design protocol of phase II clinical trial of Shuanghuang Shengbai Oral Liquid as an example, so as to provide reference for the development of new Chinese medicine for the treatment of neutropenia after chemotherapy. Methods The design scheme of phase II clinical trial of Shuanghuang Shengbai Oral Liquid: Patients with advanced lung cancer or postoperative chemotherapy for lung cancer resulting in reduction of grade 1 or 2 ANC with deficiency of both essence and qi were taken as the study subjects, and a total of 240 patients were divided into the experimental group and the control group in a ratio of 1∶1 by adopting a randomized, double-blind, placebo-parallel-controlled, multicentre clinical trial method, and the experimental group was given Shuanghuang Shengbai Oral Liquid, while the control group was given the simulant Shuanghuang Shengbai Oral Liquid, and the course of treatment was 21 days. ANC maintenance and recurrence were the main therapeutic indexes. The incidence of ANC and decreased white blood cell count, the area under ANC curve, the incidence of granulocytopenic fever, the amount of rhG-CSF injection, the change of TCM syndrome effect and the change of FACT-L scale score were taken as the secondary efficacy indicators. Adverse events were used as safety indicators. Results The design of phase II clinical trial of Shuanghuang Shengbai Oral Liquid combines the common law of disease with the individual characteristics of patients, and evaluates the therapeutic effect by combining disease and syndrome in the methodology to objectively evaluate the effectiveness and safety of the drug, which is practical and scientific. Conclusion The protocol embodies the therapeutic thinking and efficacy advantages of Chinese medicine while adapting to evidence-based concepts, and the results are extrapolated, making a useful exploration for standardizing the methodology of clinical trials and promoting Chinese medicine towards international recognition.

国家科技重大专项项目-重大新药创制专项（2019ZX09201004-002-001）;国家中医药管理局高水平中医药重点学科建设项目中医血液病学（zyyzdxk-2023268）;北京中医药大学东直门医院（国家中医药管理局下拨）—临床研究和成果转化能力提升试点项目（DZMG-ZJXY-23014）;北京中医药大学揭榜挂帅重点项目（2024-JYB-JBZD-007）