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Evaluation of Phase II Trial Design in Advanced Pancreatic Cancer

Tang, Monica MMed*; Chen, Julia MBBS*; Goldstein, David FRACP; Links, Matthew PhD*; Lord, Sarah MSc; Marschner, Ian PhD; Simes, Robert J. MD; Lee, Chee K. PhD*,‡

doi: 10.1097/MPA.0000000000001429
Original Articles

Objectives We evaluated how well phase II trials in locally advanced and metastatic pancreatic cancer (LAMPC) meet current recommendations for trial design.

Methods We conducted a systematic review of phase II first-line treatment trial for LAMPC. We assessed baseline characteristics, type of comparison, and primary end point to examine adherence to the National Cancer Institute recommendations for trial design.

Results We identified 148 studies (180 treatment arms, 7505 participants). Forty-seven (32%) studies adhered to none of the 5 evaluated National Cancer Institute recommendations, 62 (42%) followed 1, 31 (21%) followed 2, and 8 (5%) followed 3 recommendations. Studies varied with respect to the proportion of patients with good performance status (range, 0%–80%) and locally advanced disease (range, 14%–100%). Eighty-two (55%) studies concluded that investigational agents should progress to phase III testing; of these, 24 (16%) had documented phase III trials. Three (8%) phase III trials demonstrated clinically meaningful improvements for investigational agents. One of 38 phase II trials that investigated biological investigational agents was enriched for a biomarker.

Conclusions Phase II trials do not conform well to current recommendations for trial design in LAMPC.

From the *Department of Medical Oncology, St George Hospital

Department of Medical Oncology, Prince of Wales Hospital

NHMRC Clinical Trials Centre, University of Sydney, Sydney, Australia.

Received for publication May 19, 2019; accepted September 19, 2019.

Address correspondence to: Chee K. Lee, PhD, NHMRC Clinical Trials Centre, University of Sydney, Medical Foundation Building, Level 4-6, 92-94 Parramatta Rd, Camperdown NSW 2050, Australia (e-mail:

The authors reported no nonfinancial support.

M.T. received travel support from Roche, outside the submitted work. D.G. reports board membership of Sababa Medical and receives research grants from Amgen, Pfizer, and Celgene, outside the submitted work. M.L. reports consultancy fees from Leximed, outside the submitted work. I.M. receives research grants from Janssen-Cilag, outside the submitted work. R.J.S. reports research funding from the National Health and Medical Research Council (Australia). For the remaining authors, none were declared.

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