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.

journal_name

Pancreas

journal_title

Pancreas

authors

Tang M,Chen J,Goldstein D,Links M,Lord S,Marschner I,Simes RJ,Lee CK

doi

10.1097/MPA.0000000000001429

subject

Has Abstract

pub_date

2019-11-01 00:00:00

pages

1274-1284

issue

10

eissn

0885-3177

issn

1536-4828

pii

00006676-201911000-00009

journal_volume

48

pub_type

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