Evaluation of Phase II Trial Design in Advanced Pancreatic Cancer

Monica Tang; Julia Chen; David Goldstein; Matthew Links; Sarah Lord; Ian Marschner; Robert J. Simes; Chee K. Lee

doi:10.1097/MPA.0000000000001429

Evaluation of Phase II Trial Design in Advanced Pancreatic Cancer

Monica Tang, Julia Chen, David Goldstein, Matthew Links, Sarah Lord, Ian Marschner, Robert J. Simes, Chee K. Lee^*

^*Corresponding author for this work

Research output: Contribution to journal › Article › Research › peer-review

2 Citations (Scopus)

Abstract

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.

Original language	English
Pages (from-to)	1274-1284
Number of pages	11
Journal	Pancreas
Volume	48
Issue number	10
DOIs	https://doi.org/10.1097/MPA.0000000000001429
Publication status	Published - 1 Nov 2019
Externally published	Yes

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10.1097/MPA.0000000000001429

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@article{d03a7d8bfdc14250bf6a9d1f20d79718,

title = "Evaluation of Phase II Trial Design in Advanced Pancreatic Cancer",

abstract = "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.",

author = "Monica Tang and Julia Chen and David Goldstein and Matthew Links and Sarah Lord and Ian Marschner and Simes, {Robert J.} and Lee, {Chee K.}",

year = "2019",

month = nov,

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doi = "10.1097/MPA.0000000000001429",

language = "English",

volume = "48",

pages = "1274--1284",

journal = "Pancreas",

issn = "0885-3177",

publisher = "Lippincott Williams and Wilkins",

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TY - JOUR

T1 - Evaluation of Phase II Trial Design in Advanced Pancreatic Cancer

AU - Tang, Monica

AU - Chen, Julia

AU - Goldstein, David

AU - Links, Matthew

AU - Lord, Sarah

AU - Marschner, Ian

AU - Simes, Robert J.

AU - Lee, Chee K.

PY - 2019/11/1

Y1 - 2019/11/1

N2 - 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.

AB - 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.

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U2 - 10.1097/MPA.0000000000001429

DO - 10.1097/MPA.0000000000001429

M3 - Article

C2 - 31688590

AN - SCOPUS:85074549209

SN - 0885-3177

VL - 48

SP - 1274

EP - 1284

JO - Pancreas

JF - Pancreas

IS - 10

ER -

Evaluation of Phase II Trial Design in Advanced Pancreatic Cancer

Abstract

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