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