Patupilone for the treatment of recurrent/progressive brain metastases in patients (pts) with non-small cell lung cancer (NSCLC): An open-label phase II study

2033 Background: Preclinical data have shown that patupilone, a novel microtubule stabilizer, crosses the intact blood brain barrier in mice, rats and dogs and has antitumor activity in brain tumor models. This study evaluates patupilone's efficacy in NSCLC pts with brain metastases and progression after chemotherapy, surgery and/or radiation to the brain. Methods: This open-label, single-arm, multicenter, phase II, Simon 2-stage (St) study (25 pts per St) uses a multinomial design (response (R), early progression (EP)). Primary efficacy endpoints were a combination of EP (PD or death before cycle 1 Day 21) and R (progression free at 9 wks). Criteria to proceed to St 2 were >3 R and <11 EP in 25 pts. Eligible pts had confirmed NSCLC and ≥1 recurrent, measurable intracranial lesion ≥2 cm. Patupilone was given as 10 mg/m2 in a single 20-min IV infusion every 3 wks. Results: So far, 25 pts in St I and 2 pts in St II (median age 62 yrs, range 40–74, 59% male) have received 78 cycles (median 2 cycles). All pts...