Study outcomes
toxicity, PD, LNA-i-miR-221
ADVERSE EVENT, DISEASE
The primary study endpoint was safety of LNA-i-miR-221, with assessment of the MTD, and of the RP2D. The MTD was defined as the dose below the dose level at which at least 2 out of 6 patients experienced dose-limiting toxicity (DLT).The study exploratory endpoints were PK and PD profiles of LNA-i-miR-221, and preliminary investigation of anti-tumor activity, disease control and efficacy.The NCI-Common Terminology Criteria for Adverse Events (CTCAE; version 4.03, 2010) was used to assess the severity of AEs (grade 1–grade 5). An AE was classified as DLT if the AE was graded 3 or 4 and assessed as having a potential relationship to LNA-i-miR-221 administration. An independent Safety Review Committee (SRC) reviewed all safety data on an ongoing basis. Dose-escalation to the next dose was permitted only after approval by the SRC.