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Phase I clinical trial designs in oncology: A systematic literature review from 2020 to 2022
Ning Li1,2, Xitong Zhou3, Donglin Yan2
1Department of Biostatistics, College of Public Health, University of Kentucky, Lexington, KY, USA.
Background:
Phase I clinical trials aim to find the highest dose of a novel drug that may be administrated safely without having serious adverse effects. Model-based designs have recently become popular in dose-finding procedures. Our objective is to provide an overview of phase I clinical trials in oncology.
Methods:
A retrospective analysis of phase I clinical trials in oncology was performed by using the PubMed database between January 1, 2020, and December 31, 2022. We extracted all papers with the inclusion of trials in oncology and kept only those in which dose escalation or/ and dose expansion were conducted. We also compared the study parameters, design parameters, and patient parameters between industry-sponsored studies and academia-sponsored research.
Result:
Among the 1450 papers retrieved, 256 trials described phase I clinical trials in oncology. Overall, 71.1% of trials were done with a single study cohort, 56.64% of trials collected a group of at least 20 study volunteers, 55.1% were sponsored by industry, and 99.2% of trials had less than 10 patients who experienced DLTs.The traditional 3 + 3 (73.85%) was still the most prevailing method for the dose-escalation approach. More than 50% of the trials did not reach MTDs. Industry-sponsored study enrolled more patients in dose-escalation trials with benefits of continental cooperation. Compared to previous findings, the usage of model-based design increased to about 10%, and the percentage of traditional 3 + 3 design decreased to 74%.
Conclusions:
Phase I traditional 3 + 3 designs perform well, but there is still room for development in novel model-based dose-escalation designs in clinical practice.
Insights
Phase I clinical trials in oncology are evolving. While the traditional 3+3 design remains common, model-based designs are increasing, offering potential improvements for safe dose escalation.
Area of Science:
- Oncology
- Clinical Pharmacology
- Biostatistics
Background:
- Phase I clinical trials are crucial for determining the maximum tolerated dose (MTD) of novel cancer drugs.
- Model-based designs are gaining traction in dose-finding studies.
- This study provides an overview of current practices in Phase I oncology trials.
Purpose of the Study:
- To analyze trends and methodologies in Phase I oncology clinical trials.
- To compare industry-sponsored versus academia-sponsored research in this domain.
Main Methods:
- Retrospective analysis of Phase I oncology trials published between January 2020 and December 2022.
- Data extraction from PubMed focusing on dose escalation/expansion studies.
- Comparison of study, design, and patient parameters between industry and academic sponsorships.
Main Results:
- 256 Phase I oncology trials were analyzed; 71.1% used a single cohort.
- The traditional 3+3 design was used in 73.85% of trials, though its use decreased to 74%.
- Model-based designs increased to approximately 10%; over 50% of trials did not reach MTDs.
Conclusions:
- Traditional 3+3 designs are still prevalent but show a slight decrease in usage.
- There is a growing adoption of model-based designs in Phase I oncology trials.
- Further development of novel model-based dose-escalation designs is warranted for clinical practice.
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