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Randomized Phase II Cancer Clinical Trials

By Sin-Ho Jung

Chapman and Hall/CRC – 2013 – 244 pages

Series: Chapman & Hall/CRC Biostatistics Series

Purchasing Options:

  • Add to CartHardback: $89.95
    978-1-43-987185-0
    May 2nd 2013

Description

In cancer research, a traditional phase II trial is designed as a single-arm trial that compares the experimental therapy to a historical control. This simple trial design has led to several adverse issues, including increased false positivity of phase II trial results and negative phase III trials. To rectify these problems, oncologists and biostatisticians have begun to use a randomized phase II trial that compares an experimental therapy with a prospective control therapy.

Randomized Phase II Cancer Clinical Trials explains how to properly select and accurately use diverse statistical methods for designing and analyzing phase II trials. The author first reviews the statistical methods for single-arm phase II trials since some methodologies for randomized phase II trials stem from single-arm phase II trials and many phase II cancer clinical trials still use single-arm designs. The book then presents methods for randomized phase II trials and describes statistical methods for both single-arm and randomized phase II trials. Although the text focuses on phase II cancer clinical trials, the statistical methods covered can also be used (with minor modifications) in phase II trials for other diseases and in phase III cancer clinical trials.

Suitable for cancer clinicians and biostatisticians, this book shows how randomized phase II trials with a prospective control resolve the shortcomings of traditional single-arm phase II trials. It provides readers with numerous statistical design and analysis methods for randomized phase II trials in oncology.

Reviews

"… an excellent book covering various practical phase II designs with their associated statistical methods for cancer clinical trials. It is an asset to statistical professionals who work at industry or academia and would like to learn more about or improve their understanding of phase II trial design and methods for cancer clinical trials. The book can also be used as a text book for graduate student with statistical major."

—Steven Sun, Janssen Research Development, New Jersey, USA

Contents

Introduction

Single-Arm Phase II Trial Designs

Single-Stage Designs

Two-Stage Designs

Two-Stage Designs with Both Upper and Lower Stopping Values

Inference on the Binomial Probability in Single-Arm Multistage Clinical Trials

Point Estimation

Confidence Intervals

P-Values

When Realized Sample Size Is Different from That Specified in Design

Single-Arm Phase II Clinical Trials with Time-to-Event Endpoints

A Test Based on Median Survival Time

Maximum Likelihood Method for Exponential Distribution

One-Sample Log-Rank Test

Two-Stage Trials Using One-Sample Log-Rank Test

Binomial Testing on t-Year Survival Probability

Single-Arm Phase II Trials with Heterogeneous Patient Populations: Binary and Survival Outcomes

Binary Outcome Case

Survival Outcome Case: Stratified One-Sample Log-Rank Test

Randomized Phase II Trials for Selection: No Prospective Control Arms

With a Historical Control

When No Historical Control Exists

Extension to More Than Two Arms

Randomized Phase II Cancer Clinical Trials with a Prospective Control on Binary Endpoints (I): Two-Sample Binomial Test

Two-Sample Binomial Test

Two-Stage Designs with Both Upper and Lower Stopping Values

Discussions

Randomized Phase II Cancer Clinical Trials with a Prospective Control on Binary Endpoints (II): Fisher’s Exact Test

Single-Stage Design

Two-Stage Design

Extensions

Discussions

Randomized Phase II Trials with Heterogeneous Patient Populations: Stratified Fisher’s Exact Test

Single-Stage Stratified Fisher’s Exact Test

Two-Stage Designs with an Interim Futility Test

Randomized Phase II Clinical Trials Based on Survival Endpoints: Two-Sample Log-Rank Test

Two-Sample Log-Rank Test

Two-Stage Log-Rank Test

Stratified Two-Sample Log-Rank Test for Single-Stage Designs

Some Flexible Phase II Clinical Trial Designs

Comparing Survival Distributions under General Hypothesis Testing

Randomized Phase II Trials for Comparing Maintenance Therapies

Index

References appear at the end of each chapter.

Author Bio

Sin-Ho Jung is a professor of biostatistics and bioinformatics at Duke University School of Medicine. He earned a PhD from the University of Wisconsin-Madison. His research interests include clinical trial design, survival analysis, longitudinal data analysis, clustered data analysis, ROC curve analysis, and microarray studies.

Name: Randomized Phase II Cancer Clinical Trials (Hardback)Chapman and Hall/CRC 
Description: By Sin-Ho Jung. In cancer research, a traditional phase II trial is designed as a single-arm trial that compares the experimental therapy to a historical control. This simple trial design has led to several adverse issues, including increased false positivity of phase...
Categories: Clinical Implementation, Oncology, Statistics for the Biological Sciences