St. Jude researchers propose phase II clinical trial design and software

Scientists from St. Jude Children’s Research Hospital have proposed a new method for designing phase II clinical trials and have created a corresponding software to implement the approach.

The work was recently detailed in the journal Pharmaceutical Statistics. The corresponding authors are Haitao Pan, Ph.D., of the St. Jude Department of Biostatistics, and Jianrong Wu, Ph.D. of the Markey Cancer Center at University of Kentucky.

“In rare cancers, like pediatric cancers, using historical data is a pragmatic approach for speeding up clinical trials,” Pan said. “The proposed method focuses on a kind of phase II trial with characteristics of a single-arm trial, using historical control data and a survival endpoint.”

In recent years, drug development in oncology has broadened from mainly considering randomized controlled trials to subtype-specific clinical trials. The latter often relies on historical controls, Pan said.

“Though the randomized controlled trial is the established gold standard for clinical research, in recent years there has been an increasing number of single-arm studies, particularly in the areas of oncology with phase I expansion cohorts, as well as in rare diseases,” Pan said. “Such an approach is better suited for cases where a randomized trial would be unethical or impractical, and for smaller trials to facilitate quicker access to novel therapies for patients.”

Co-author Chia-Wei Hsu of St. Jude Biostatistics, said, “We present an event-driven approach for Bayesian one-stage and two-stage single-arm phase II trial designs. The theoretical and empirical findings will help investigators who want to design a trial using the Bayesian approach and meanwhile to attain the frequentist operating characteristics which are often required by the regulatory agencies.”

The software they developed, called BayesDesign, can be accessed at no cost by researchers via R CRAN for convenient use of the proposed methods at https://CRAN.R-project.org/package=BayesDesign.

The study was funded by the National Cancer Institute (CA177558) and ALSAC, the fundraising and awareness organization of St. Jude.