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. Author manuscript; available in PMC: 2016 Aug 15.
Published in final edited form as: Expert Opin Ther Targets. 2015 Oct 6;20(3):341–359. doi: 10.1517/14728222.2016.1094057

Table 1.

Clinical trials assessing the role of targeted agents in pancreatic cancer.

Study Study design Targeting agent Molecular
mechanism
Outcomes
Rinehart et al. [16] Phase II clinical trial in multiple
types of advanced cancers
CI-1040 alone Oral MEK
inhibitor
No significant clinical benefit (no
objective responses)
Infante et al. [17] Phase II randomized clinical trial
in advance stage pancreatic
cancer
Trametinib
(GSK1120212)
combined with
gemcitabine
Oral MEK
inhibitor
No significant clinical benefit
(95% CI, 0.67 – 1.44
p = 0.453)
Philip et al. [27] Phase III randomized clinical trial
in advanced stage pancreatic
cancer
Cetuximab combined
with gemcitabine
EGFR monoclonal
antibody
No significant clinical benefit
(HR; = 1.06; 95% CI,
0.91 – 1.23; p = 0.23)
Moore et al. [28] Phase III randomized clinical trial
in advanced stage pancreatic
cancer
Erlotinib combined
with gemcitabine
EGFR small
molecule tyrosine
kinase inhibitor
Superior to gemcitabine alone
[HR of 0.77 (95% CI
0.64 – 0.92; p = 0.004)]
Wolpin et al. [41] Phase II trial in gemcitabine-
resistant pancreatic cancer
Everolimus Mammalian target
of rapamycin
(mTOR) inhibitor
No complete or partial
responses
Daud et al. [106] Phase I trial in multiple
advanced stage cancers
MK-8776 ±
gemcitabine
Chk1 inhibitor Two PR’s and 13 stable disease
in N = 30
Bramhall et al. [125] Phase III double blind, placebo-
controlled randomized trial in
pancreatic cancer
Marimastat combined
with gemcitabine
MMP inhibitor No survival benefit in
experimental arm vs
gemcitabine alone (165.5 days
vs 164 days p = 0.95)
Moore et al. [126] Phase III randomized clinical trial
in advanced stage pancreatic
cancer
Bay12 – 9566 MMP inhibitor Gemcitabine superior to
Bay12 – 9566 (6.59 months vs
3.74 months p < 0.001)
Kim et al. [154] A single arm pilot clinical trial in
advanced stage pancreatic
cancer
Vismodegib combined
with gemcitabine
SHH inhibitor Progression free survival and OS;
2.8 and 5.3 months respectively
Ross et al.
(NCT01130142)
Randomized clinical trial in
advanced stage pancreatic
cancer
Saridegib combined
with gemcitabine
SHH inhibitor Study suspended due to
progressive disease in
experimental arm