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A sequential phase II/III trial for binary outcomes.

B E Storer1

  • 1Department of Statistics, University of Wisconsin, Medical Science Center, Madison 53706.

Statistics in Medicine
|March 1, 1990
PubMed
Summary
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This study presents a two-stage clinical trial design for binary outcomes. It allows early stopping in the experimental arm without affecting the overall type I error rate, though sample size may increase.

Area of Science:

  • Clinical Trials Methodology
  • Biostatistics
  • Experimental Design

Background:

  • Randomized clinical trials (RCTs) are crucial for evaluating new treatments.
  • Binary outcomes are common in clinical research, necessitating specific statistical approaches.
  • Early stopping rules can improve trial efficiency but require careful design.

Purpose of the Study:

  • To introduce a straightforward two-stage design for randomized clinical trials with binary outcomes.
  • To incorporate an early stopping criterion focused solely on the experimental arm's efficacy.
  • To assess the impact of this design on statistical properties like type I error and power.

Main Methods:

  • The proposed design utilizes a two-stage approach for randomized clinical trials.

Related Experiment Videos

  • An early stopping rule is implemented, specifically evaluating the experimental arm against an external efficacy standard.
  • The stopping rules are analogous to those employed in Phase II clinical trial designs.

    • Main Results:

    • The two-stage design maintains the overall type I error rate.
    • Early stopping is applied only to the experimental arm.
    • A modest loss in statistical power may occur, necessitating an increased sample size.

    Conclusions:

    • This simple two-stage design offers a method for early evaluation of experimental arms in RCTs.
    • The design effectively controls the type I error rate.
    • Researchers must consider the trade-off between early stopping and potential power reduction when increasing sample size.