Trial/Year | Design | BRCA1/2 mutation | Primary end point | Randomisation | Treatment arms | Patients in each arm | Outcome |
Andrew Tutt, 2010 [14] | Non-randomised, sequential cohort design, multicentre, phase II | Yes | ORR | NA | 400 mg bid 100 mg bid | 27 27 | ORR was higher in 400 mg dosage cohort than in 100 mg dosage cohort |
M. William Audeh, 2010 [13] | Non-randomised, prospective, multicentre, phase II | Yes | ORR | NA | 400 mg bid 100 mg bid | 33 24 | ORRs were confirmed and duration of response in the two cohorts (33% vs. 13%) |
Karen A. Gelmon, 2011 [18] | Non-randomised, open-label, multicentre, phase II | With or without | ORR | NA | Breast cancer 400 mg bid Ovarian cancer 400 mg bid | 26
65 | Confirmed objective responses were reported in patients with ovarian carcinoma (29%), particularly in BRCA mutation cohort |
Stan B. Kaye. 2012 [19] | Randomized, prospective, open-label, multicenter, phase II | Yes | PFS | 1:1:1 | 200 mg bid 400 mg bid PLD 50 mg/m2 | 32 32 33 | There was no statistically significant difference in PFS (p = 0.66) |
Jonathan Ledermann, 2014 [12] [23] | Randomized, double-blind, phase 2 study | With or without | PFS | 1:1 | 400 mg bid Placebo | 136 129 | PFS was significantly longer with olaparib than with placebo (p < 0.001) |
Bella Kaufman, 2015 [20] | Non-randomized, prospective, multicenter, phase II | Yes | Tumor response rate | NA | Breast cancer 400 mg bid Ovarian cancer 400 mg bid Pancreatic cancer 400 mg bid Prostate cancer 400 mg bid other | 62
193
23
8
12 | Tumor response rate was 31.1% and 12.9% of patients with ovarian and breast cancers, respectively |