Outcomes
toxicity
DISEASE
An interim analysis was planned to evaluate the toxicity and benefit of treatment for the first 20 consecutive patients. If >50% of the patients experienced dose-limiting toxicity, a trial amendment with revised study drug dosing would be performed. The study would be terminated for lack of efficacy if <60% had at best non-progressive disease after 6 weeks of treatment.The primary endpoint was disease control rate (DCR) at 6 months, equivalent to non-progressive disease at 6 months according to RECIST version 1.1. In the Nordic NEC study, DCR at 6 months was 38% for a similar patient population (Ki-67 20–55% and PS 0–1) and the use of temozolomide-based chemotherapy as second-line treatment in GEP-NEC resulted in a general disease control rate of 72% at 2 months, and was even higher if Ki-67 was <60% [