Chu Quincy Siu-Chung, Sangha Randeep, Spratlin Jennifer, Vos Larissa J, Mackey John R, McEwan Alexander J B, Venner Peter, Michelakis Evangelos D
Department of Oncology, University of Alberta and Division of Medical Oncology, Cross Cancer Institute, 11560 University Avenue, Edmonton, AB, T6G 1Z2, Canada,
Invest New Drugs. 2015 Jun;33(3):603-10. doi: 10.1007/s10637-015-0221-y. Epub 2015 Mar 13.
Purpose Preclinical evidence suggests dichloroacetate (DCA) can reverse the Warburg effect and inhibit growth in cancer models. This phase 1 study was undertaken to assess the safety, recommended phase 2 dose (RP2D), and pharmacokinetic (PK) profile of oral DCA in patients with advanced solid tumors. Patients and Methods Twenty-four patients with advanced solid malignancies were enrolled using a standard 3 + 3 protocol at a starting dose of 6.25 mg/kg twice daily (BID). Treatment on 28 days cycles was continued until progression, toxicity, or consent withdrawal. PK samples were collected on days 1 and 15 of cycle 1, and day 1 of subsequent cycles. PET imaging ((18) F-FDG uptake) was investigated as a potential biomarker of response. Results Twenty-three evaluable patients were treated with DCA at two doses: 6.25 mg/kg and 12.5 mg/kg BID (median of 2 cycles each). No DLTs occurred in the 6.25 mg/kg BID cohort so the dose was escalated. Three of seven patients had DLTs (fatigue, vomiting, diarrhea) at 12.5 mg/kg BID. Thirteen additional patients were treated at 6.25 mg/kg BID. Most toxicities were grade 1-2 with the most common being fatigue, neuropathy and nausea. No responses were observed and eight patients had stable disease. The DCA PK profile in cancer patients was consistent with previously published data. There was high variability in PK values and neuropathy among patients. Progressive increase in DCA trough levels and a trend towards decreased (18) F-FDG uptake with length of DCA therapy was observed. Conclusions The RP2D of oral DCA is 6.25 mg/kg BID. Toxicities will require careful monitoring in future trials.
目的 临床前证据表明二氯乙酸(DCA)可逆转瓦伯格效应并抑制癌症模型中的肿瘤生长。本1期研究旨在评估口服DCA在晚期实体瘤患者中的安全性、推荐的2期剂量(RP2D)和药代动力学(PK)特征。 患者与方法 采用标准的3 + 3方案,纳入24例晚期实体恶性肿瘤患者,起始剂量为6.25 mg/kg,每日两次(BID)。以28天为周期持续治疗,直至疾病进展、出现毒性反应或患者撤回同意书。在第1周期的第1天和第15天以及后续周期的第1天采集PK样本。研究正电子发射断层扫描(PET)成像((18)F-FDG摄取)作为反应的潜在生物标志物。 结果 23例可评估患者接受了两种剂量的DCA治疗:6.25 mg/kg和12.5 mg/kg BID(各剂量组的中位治疗周期均为2个周期)。6.25 mg/kg BID剂量组未发生剂量限制毒性(DLT),因此该剂量得以递增。12.5 mg/kg BID剂量组的7例患者中有3例出现DLT(疲劳、呕吐、腹泻)。另外13例患者接受6.25 mg/kg BID剂量治疗。大多数毒性反应为1 - 2级,最常见的是疲劳、神经病变和恶心。未观察到缓解情况,8例患者疾病稳定。癌症患者的DCA PK特征与先前发表的数据一致。患者之间的PK值和神经病变存在高度变异性。观察到随着DCA治疗时间延长,DCA谷浓度逐渐升高,(18)F-FDG摄取有下降趋势。 结论 口服DCA的RP2D为6.25 mg/kg BID。在未来试验中需要仔细监测毒性反应。
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