NET-02 试验方案：一项多中心、随机、平行组、开放性、二期、单阶段选择试验，旨在评估脂质体伊立替康（nal-IRI）联合 5-氟尿嘧啶（5-FU）/亚叶酸或多西他赛作为进展性低分化肺外神经内分泌癌（NEC）二线治疗的疗效。
NET-02 trial protocol: a multicentre, randomised, parallel group, open-label, phase II, single-stage selection trial of liposomal irinotecan (nal-IRI) and 5-fluorouracil (5-FU)/folinic acid or docetaxel as second-line therapy in patients with progressive poorly differentiated extrapulmonary neuroendocrine carcinoma (NEC).
机构信息
Clinical Trials Research Unit, Leeds Institute of Clinical Trials Research, University of Leeds, Leeds, West Yorkshire, UK.
Department of Oncology, Weston Park Hospital, Sheffield, Sheffield, UK.
出版信息
BMJ Open. 2020 Feb 5;10(2):e034527. doi: 10.1136/bmjopen-2019-034527.
INTRODUCTION
Poorly differentiated (PD), extrapulmonary (EP), neuroendocrine carcinomas (NECs) are rare but aggressive neuroendocrine neoplasms. First-line treatment for advanced disease is an etoposide and platinum-based chemotherapy combination. There is no established second-line treatment for patients with PD-EP-NEC, and this is an area of unmet need.
METHODS AND ANALYSIS
NET-02 is a UK, multicentre, randomised (1:1), parallel group, open-label, phase II, single-stage selection trial of liposomal irinotecan (nal-IRI)/5-fluorouracil (5-FU)/folinic acid or docetaxel as second-line therapy in patients with progressive PD-EP-NEC. One hundred and two eligible participants will be randomised to receive either nal-IRI/5-FU/folinic acid or docetaxel. The primary objective is to determine the 6-month progression-free survival (PFS) rate. The secondary objectives of this study are to determine PFS, overall survival, objective response rate, toxicity, quality of life and whether neuron-specific enolase is predictive of treatment response. If either treatment is found to have a 6-month PFS rate of at least 25%, that treatment will be considered for a phase III trial. If both treatments meet this target, prespecified selection criteria will be applied to establish which treatment to take forward.
ETHICS AND DISSEMINATION
This study has ethical approval from the Greater Manchester Central Research Ethics Committee (reference no. 18/NW/0031) and clinical trial authorisation from the Medicine and Healthcare Products Regulatory Agency. Results will be published in peer-reviewed journals and uploaded to the European Union Clinical Trials Register.
TRIAL REGISTRATION NUMBERS
ISRCTN10996604, NCT03837977, EudraCT Number: 2017-002453-11.
简介
低分化（PD）、肺外（EP）神经内分泌癌（NEC）较为罕见，但属于侵袭性神经内分泌肿瘤。晚期疾病的一线治疗是依托泊苷和铂类化疗联合治疗。对于 PD-EP-NEC 患者，目前尚无既定的二线治疗方法，这是一个未满足的需求领域。
方法和分析
NET-02 是一项英国多中心、随机（1:1）、平行组、开放性、二期、单阶段选择试验，旨在评估脂质体伊立替康（nal-IRI）/5-氟尿嘧啶（5-FU）/亚叶酸或多西他赛作为进展性 PD-EP-NEC 二线治疗的疗效。102 名符合条件的患者将被随机分为 nal-IRI/5-FU/亚叶酸或多西他赛组。主要终点是确定 6 个月无进展生存期（PFS）率。该研究的次要目标是确定 PFS、总生存期、客观缓解率、毒性、生活质量以及神经元特异性烯醇化酶是否可预测治疗反应。如果任何一种治疗方案的 6 个月 PFS 率至少为 25%，则认为该治疗方案具有进入三期试验的潜力。如果两种治疗方案均达到这一目标，则将应用预设选择标准以确定哪种治疗方案继续推进。
伦理和传播
该研究已获得大曼彻斯特中部研究伦理委员会（参考号 18/NW/0031）的伦理批准和药品和保健品管理局的临床试验授权。研究结果将发表在同行评议的期刊上，并上传至欧洲临床试验注册中心。
试验注册号
ISRCTN83130137、NCT03837977、EudraCT 编号：2017-002453-11。
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