Related Experiment Video
Updated: May 11, 2026
04:53
A Clinical Trial Assessing the Safety, Efficacy, and Delivery of Olive-Oil-Based Three-Chamber Bags for Parenteral Nutrition
Published on: September 20, 2019
Designs for randomized phase II clinical trials with two treatment arms.
Wei Hou1, Myron N Chang, Sin-Ho Jung
1Department of Preventive Medicine, Stony Brook University Medical Center, Stony Brook, NY 11794-8036 USA. wei.hou@stonybrookmedicine.edu
Statistics in Medicine
|May 1, 2013
Summary
This study introduces new designs for randomized phase II clinical trials to assess cancer treatment response rates. The methods precisely define error rates and powers, aiding in selecting effective therapies.
Area of Science:
- Clinical Trials
- Biostatistics
- Oncology
Background:
- Phase II clinical trials commonly use responder/non-responder status as a primary endpoint.
- Randomized phase II trials aim to evaluate novel anticancer treatments against a baseline response rate.
- Selecting promising treatments for further study is a key objective.
Purpose of the Study:
- To propose novel single-stage and two-stage designs for randomized phase II clinical trials.
- To precisely define and control Type I error rates and statistical power for treatment selection.
- To provide a computational tool for designing trials that meet specific statistical requirements.
Main Methods:
- Development of precise statistical definitions for Type I error rates and power.
- Implementation of a program to accurately compute these error rates and powers.
- Application of proposed designs to a real-world randomized phase II trial.
Main Results:
- The proposed designs allow for exact computation of error rates and powers.
- Numerous design examples are provided to meet pre-defined statistical criteria.
- The methodology was successfully applied to a trial involving relapsed non-Hodgkin's disease.
Conclusions:
- The developed designs offer a robust framework for randomized phase II anticancer trials.
- The computational tool facilitates the selection of optimal trial designs based on desired error rates and power.
- This approach enhances the efficiency and reliability of early-phase cancer treatment evaluation.
Keywords:
average sample sizeminimizing the maximum sample sizepowersingle-stage designthresholdtwo-stage designtype I error rateMore Related Videos
Related Concept Videos
01:11Clinical Trials: Overview
Clinical development focuses on how the drug will interact with the human body and encompasses four key phases of clinical trials, each serving a specific purpose in assessing the safety and effectiveness of new drugs. These phases overlap and build upon one another. Phase I involves a small group of healthy volunteers (typically 20-80 individuals) or, in cases where significant toxicity is expected, patients with the targeted disease, such as cancer or AIDS. The volunteers are tested for...
01:16Clinical Trials
Clinical trials are prospective experimental studies conducted on humans to determine the safety and efficacy of treatments, drugs, diet methods, and medical devices. Using statistics in clinical trials enables researchers to derive reasonable and accurate conclusions from the collected data, allowing them to make wise decisions in uncertain situations. In medical research, statistical methods are crucial for preventing errors and bias.
There are four phases in a clinical trial. A phase one...
There are four phases in a clinical trial. A phase one...
01:15Bioequivalence Experimental Study Designs: Repeated Measures, Cross-Over, Carry-Over, and Latin Square Designs
Bioequivalence experimental study designs play a pivotal role in testing the effectiveness of various treatments. Key among these are the repeated measures, cross-over, carry-over, and Latin square designs. In the repeated measures design, each subject receives all treatments, allowing for temporal comparisons. This type of design is useful in reducing variability but requires careful planning to avoid bias.The cross-over design, an economical method, involves sequential administration of...
01:20Bioequivalence Experimental Study Designs: Completely Randomized and Randomized Block Designs
Bioequivalence experimental study designs are crucial methodologies used in evaluating and comparing the bioavailability of different drug products. These designs are categorized into various types: completely randomized, randomized block, repeated measures, cross and carry-over, and Latin square designs.Completely randomized designs involve randomly allocating treatments to all subjects participating in the experiment. This allocation is achieved by assigning unique random numbers to subjects...
01:16Crossover Experiments
Crossover experiments, also called the repeated-measurements design, is a study design in which all experimental units are exposed to all treatments in different periods. Crossover experiments are generally used in psychology, the pharmaceutical industry, agriculture, and medicine.
Crossover designs are performed even with smaller sample sizes since the samples can act as their controls. These are better than simple randomized trials since patients are exposed to all the treatments.
Crossover designs are performed even with smaller sample sizes since the samples can act as their controls. These are better than simple randomized trials since patients are exposed to all the treatments.
01:28Dosage Regimens: Designs and Approaches
Designing a dosage regimen, which refers to the manner of drug administration, is a complex process involving the selection of drug dose, route, and frequency. This process is underpinned by pharmacokinetic parameters derived from tests and population averages. These parameters are then tailored to patient-specific variables such as diagnosis, demographics, and allergy status. Once therapy commences, therapeutic response monitoring is critical and achieved through clinical and physical...