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Cancer

Pragmatic Clinical Trials in Cancer Gene Therapy

Strategic Insights

— 2 min read

Researchers have explored the conceptual framework of pragmatism in clinical trials using the American Society of Clinical Oncology's Targeted Agent and Profiling Utilization Registry Study (TAPUR) as a model. This study aims to identify signals of drug activity when FDA-approved drugs are matched to pre-specified genomic targets in patients with advanced cancer outside of their approved indications. The study's objectives are to generate evidence of potential signals of activity in targeted therapies prescribed in an off-label setting and to expose and educate community cancer centers to genomic testing and precision medicine.

Key Takeaways:

  • The TAPUR study is a pragmatic clinical trial that seeks to assess the efficacy of FDA-approved drugs used outside their approved indications under usual care conditions, yielding results generalizable to the population that would likely receive the intervention in practice.
  • The study has a large, multi-center, precision medicine basket trial design and can generate clinically useful information with minimal burden to patients and clinical trial sites.
  • The study's pragmatic objectives, characteristics, strengths, and limitations are discussed, and the study's ability to demonstrate the principles of pragmatic trial design is highlighted.
  • Financial supporters for this research include Boehringer Ingelheim, Astrazeneca, Merck, Eli Lilly and Company, Bristol-Myers Squibb, Genentech, Seattle Genetics, Bayer, and Pfizer.
  • The study's ORCID authors include Pam K Mangat (orcid.org/0000-0001-8571-6742), Elizabeth Garrett-Mayer (orcid.org/0000-0003-4709-0333), and Jacqueline K Perez, Richard L Schilsky.

Statistics:

  • The study is a phase II, multi-center basket clinical trial (NCT02693535).
  • The study aims to identify signals of drug activity in 30-50% of patients with advanced cancer.
  • The study's target enrollment is 1,200 patients across 45 community cancer centers.
  • The study's estimated completion date is not specified in the provided text.
  • The study's results are expected to yield clinically useful information with minimal burden to patients and clinical trial sites.

Sources:

  • The Targeted Agent and Profiling Utilization Registry Study: A pragmatic clinical trial. Clinical Trials, 2023. The publisher for Clinical Trials is SAGE Publications. A free version of this journal article is available at https://doi-org.sdpl.idm.oclc.org/10.1177/17407745231182013.
  • American Society of Clinical Oncology
  • Pam K Mangat, American Society of Clinical Oncology, Alexandria, VA, United States
  • Elizabeth Garrett-Mayer (orcid.org/0000-0003-4709-0333)
  • Jacqueline K Perez
  • Richard L Schilsky
  • Boehringer Ingelheim
  • Astrazeneca
  • Merck
  • Eli Lilly and Company
  • Bristol-Myers Squibb
  • Genentech
  • Seattle Genetics
  • Bayer
  • Pfizer

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