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The Targeted Agent and Profiling Utilization Registry Study: A pragmatic clinical trial
Pam K Mangat1, Elizabeth Garrett-Mayer1, Jacqueline K Perez1
1American Society of Clinical Oncology, Alexandria, VA, USA.
Clinical Trials (London, England)
|July 25, 2023
Summary
The Targeted Agent and Profiling Utilization Registry Study demonstrates that pragmatic, multi-center basket trials can identify targeted therapy signals in advanced cancer patients. This approach generates clinically useful, generalizable evidence with minimal patient and site burden.
Area of Science:
- Oncology
- Clinical Trial Design
- Precision Medicine
Background:
- Pragmatic clinical trials are essential for real-world evidence generation.
- The American Society of Clinical Oncology's Targeted Agent and Profiling Utilization Registry Study (NCT02693535) serves as a model for pragmatic trial design.
Purpose of the Study:
- To explore the conceptual framework of pragmatism in clinical trials.
- To identify drug activity signals for Food and Drug Administration (FDA)-approved drugs used off-label in advanced cancer.
- To educate community cancer centers on genomic testing and precision medicine.
Main Methods:
- A pragmatic, non-randomized, phase II, multi-center basket clinical trial design.
- Matching FDA-approved drugs to pre-specified genomic targets in patients with advanced cancer.
- Utilizing usual care conditions to ensure generalizability of results.
Main Results:
- Demonstrates the feasibility of conducting large, multi-center precision medicine basket trials in community practice.
- Generates clinically useful information on off-label targeted therapies.
- Minimal burden on patients and clinical trial sites.
Conclusions:
- Pragmatic trial principles can be applied across various evidence-generation strategies.
- The Targeted Agent and Profiling Utilization Registry Study exemplifies a successful pragmatic approach.
- This model yields generalizable results applicable to real-world clinical practice.
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