Proof of concept and design of an externally controlled trial for patients with gastroenteropancreatic neuroendocrine carcinomas based on the randomised phase II BEVANEC trial

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Introduction: Neuroendocrine carcinomas of digestive or unknown primary trials are challenging to perform because of disease rarity. BEVANEC trial randomised 126 patients to FOLFIRI +/- bevacizumab in second line, over 5 years, and reported similar overall survival (OS) in both arms.

Aim(s): To demonstrate that BEVANEC could have been led with an external control arm (ECA).

Materials and methods: BEVANEC-EMU was emulated as a prospective single arm trial of FOLFIRI + bevacizumab [data from BEVANEC experimental arm (EA)] vs. an ECA of FOLFIRI from real-world data (RWD). The inclusion criteria and primary endpoints were similar as those of BEVANEC.

Conference:

Presenting Author: Hadoux J

Authors: Ganame S, Walter T, Durand A, Lièvre A, Tougeron D,

Keywords: Neuroendocrine carcinoma, synthetic control arm, external control arm, new clinical trial design, randomisation algorithm, real-world data, invers probability weighting,