Department of Hepatic Surgery VI, Eastern Hepatobiliary Surgery Hospital, Second Military Medical University, Changhai Rd No.225, Shanghai, 200438, China.
Department of Cancer Center, First Hospital of Jilin University, Xinmin Rd No71, Changchun, 130021, Jilin Province, China.
Trials. 2019 Apr 29;20(1):245. doi: 10.1186/s13063-019-3349-9.
Among patients with hepatocellular carcinoma (HCC), 85% of patients have an advanced disease stage at diagnosis and curative therapies cannot be performed. Prognosis has been quite poor as until recently there was no proven effective chemotherapy. Our group found that all-trans-retinoic acid (ATRA) could improve the efficacy of platinum in HCC in vivo and in vitro, thus we wish to validate the efficiency of ATRA in clinical practice.
This is a double-blinded, 1:1 randomized, controlled, multicenter clinical trial. Three hundred and sixty-eight patients with HCC and extrahepatic metastases will receive palliative chemotherapy at the Eastern Hepatobiliary Surgery Hospital, First Hospital of Jilin University and Fujian Provincial Cancer Hospital. Subjects will be randomly assigned to one of the two arms, either ATRA + oxaliplatin + 5-fluorouracil/leucovorin (FOLFOX4) or FOLFOX4 alone. ATRA 20 mg will be given orally three times/day for 3 days prior to the initiation of FOLFOX4. ATRA will be discontinued at the end of FOLFOX4.
Overall survival rate is the primary endpoint. Secondary endpoints are time to progression according to the modified response evaluation criteria in solid tumors (mRECIST) criteria, acute and chronic adverse events, and quality of life.
Chinese Clinical Trial Registry, ChiCTR-IIR-17012916 . Registered on 9 October 2017.
在肝细胞癌（HCC）患者中，85%的患者在诊断时处于晚期，无法进行治愈性治疗。由于直到最近还没有经过证实的有效化疗方法，因此预后一直很差。我们的研究小组发现全反式维甲酸（ATRA）可以提高 HCC 体内和体外铂类治疗的疗效，因此我们希望验证 ATRA 在临床实践中的疗效。
这是一项双盲、1:1 随机、对照、多中心临床试验。368 例 HCC 伴肝外转移患者将在东方肝胆外科医院、吉林大学第一医院和福建省肿瘤医院接受姑息性化疗。受试者将被随机分配到以下两个治疗组之一，即 ATRA+奥沙利铂+5-氟尿嘧啶/亚叶酸钙（FOLFOX4）或 FOLFOX4 单药治疗。在开始 FOLFOX4 前，ATRA 20mg 将口服，每日 3 次，连续 3 天。在 FOLFOX4 结束时，将停止使用 ATRA。
总生存率是主要终点。次要终点是根据实体瘤改良疗效评价标准（mRECIST）的无进展生存期、急性和慢性不良事件以及生活质量。
中国临床试验注册中心，ChiCTR-IIR-17012916。注册于 2017 年 10 月 9 日。
