Phase II designs for anticancer botanicals and supplements

Area of Science:

• Oncology
• Clinical Trial Design
• Pharmacology

Background:

• Traditional phase II trials assess anticancer agents using tumor response (e.g., 50% size reduction).
• Anticancer botanicals and supplements may not cause rapid tumor regression but could improve survival.
• Existing phase II designs are not optimal for evaluating botanicals and supplements based on survival endpoints.

Purpose of the Study:

• To present a novel phase II trial design for evaluating anticancer botanicals and supplements.
• To establish a methodology for using historical data to predict and assess patient outcomes.
• To determine if a new agent warrants further investigation in phase III trials based on survival benefits.

Main Methods:

• Utilizes historical data to create a statistical prediction model for patient outcomes.
• Adjusts predictions for sampling variation and patient case mix.
• Compares predicted survival outcomes with observed results in the phase II study cohort.

Main Results:

• The proposed design allows for the evaluation of survival endpoints, such as 6-month progression-free survival.
• It provides a framework for comparing observed patient outcomes against statistically predicted results.
• The approach facilitates the identification of promising agents for further clinical development.

Conclusions:

• The presented phase II design is suitable for anticancer botanicals and supplements, focusing on survival rather than tumor response.
• This method appropriately uses historical data to assess the efficacy of novel agents.
• Positive results from this design indicate the potential for agents to advance to phase III randomized controlled trials.