METHODS: The RamuNET study is an investigator-initiated multicenter prospective single-arm trial to evaluate the efficacy of ramucirumab in combination with dacarbazine (DTIC) over a period of at least 6 months. Patients with progressive well-differentiated and metastatic pancreatic neuroendocrine tumors are eligible. The study aims to include 45 patients over a period of 24 months with a minimum follow-up of 24 months. The primary endpoint is disease control after 6 months. Secondary endpoints include progression-free survival, biochemical response, overall survival, quality of life and toxicity. Based on the hypothesis that 80% of the patients can achieve a disease control after 6 months, the sample size calculation follows an exact binomial single-stage design. H0: p < =p0 = 60% versus H1: p > =p1 = 80%, alpha = 0.05, beta = 0.1.
CONCLUSIONS: This study investigates a new therapeutic approach using the combination of cytotoxic and targeted antiangiogenic therapy in advanced PNET. If positive, this trial will be the basis for a randomized two-arm study to investigate the combination of ramucirumab and DTIC against other established therapies in PNET.
BACKGROUND: EudraCT: 2017-001207-68 . Date of registration: 2018.01.03.
方法:RamuNET研究是一项由研究者发起的多中心前瞻性单臂试验,用于评估雷莫西单抗联合达卡巴嗪(DTIC)在至少6个月的时间内的疗效。患有进行性高分化和转移性胰腺神经内分泌肿瘤的患者是合格的。该研究旨在包括45名患者,为期24个月,最少随访24个月。主要终点是6个月后的疾病控制。次要终点包括无进展生存期,生化反应,总生存率,生活质量和毒性。基于假设80%的患者可以在6个月后实现疾病控制,样本量计算遵循精确的二项式单级设计。H0:p<=p0=60%与H1:p>=p1=80%,α=0.05,β=0.1。
结论:本研究探讨了在晚期PNET中使用细胞毒性和靶向抗血管生成疗法的联合治疗方法。如果是积极的,这项试验将成为一项随机双臂研究的基础,该研究旨在研究雷莫西单抗和DTIC联合治疗对PNET中其他既定治疗的效果.
背景:EudraCT:2017-001207-68。注册日期:2018.01.03。