Background:
Neuroendocrine carcinomas (NEC WHO grade 3) are rare and aggressive cancers. There are no randomised trials to date to establish standard therapy for advanced gastrointestinal (GI) NECs. Etoposide and carboplatin are used extrapolating from small cell lung cancer data. Paclitaxel is also active in NECs however there is no data on the role of nab-paclitaxel. NABNEC aims to establish if the carboplatin and nab-paclitaxel combination is an effective and tolerable treatment for advanced GI-NECs and to enhance our understanding of biological and imaging characteristics of NECs.
Trial design:
NABNEC is a randomised phase II trial of adults with non-resectable GI-NECs (Ki 67 ≥ 20%). Arm A (n = 47) IV nab-paclitaxel 100 mg/m2 on Day (D) 1 weekly and IV carboplatin AUC = 5 on D1, 3 weekly; Arm B (n = 23) IV etoposide 100mg/m2 on D1-3, 3 weekly and IV carboplatin AUC = 5 on D1, 3 weekly, to continue until disease progression or unacceptable toxicity. Primary endpoint: objective response rate (RR) by RECIST 1.1 at 6 months. A total sample size of 70 patients with a 2:1 randomisation (intervention to control) will have 80% power with 95% confidence to rule out a 30% RR in favour of a clinically relevant RR of 50%, which would justify further investigation. Secondary endpoints: progression free survival, overall survival, adverse events by NCI-CTCAE V4.03 and quality of life (EORTC QLQC30, QLQ-GINET21 questionnaires). Translational endpoints include1) blood and tissue biomarkers (prognostic and/or predictive) correlated with clinical endpoints including circulating tumour cells, mutation profile (whole exome sequencing), DNA methylation profile; 2) Correlation of 18-fluoro-deoxyglucose positron emission tomography (FDG-PET) to early response and other clinical endpoints. NABNEC has opened at 10 of 20 planned study sites in Australia and New Zealand and is enrolling patients. ANZCTR # 12616000958482