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Evaluating the Effectiveness of Cancer Drug Sensitization In Vitro and In Vivo
Published on: February 6, 2015
The changing face of phase 1 cancer clinical trials: new challenges in study requirements
Barbara S Craft1, Razelle Kurzrock, Xiudong Lei
1Division of Cancer Medicine, The University of Texas M. D. Anderson Cancer Center, Houston, Texas, USA.
Abstract:
Phase 1 studies in cancer have changed in recent years. Now, with the advent of new, less toxic, targeted agents, more patients may be candidates for new drug studies earlier in the course of their disease. It is to the advantage of the members of the oncology community to know more regarding the details and requirements for participation in early-phase clinical trials so they can advocate for their patients and help them decide when such trials may be an appropriate choice. To examine the work intensity of early phase cancer clinical trials, the authors of this report compared the study requirements of phase 1 and 2 protocols. Five parameters were studied as a surrogate of study complexity-the number of physical examinations, vital sign determinations, electrocardiograms (ECGs), nonpharmacokinetic laboratory tests, and pharmacokinetic (PK) sampling-in the first 4 weeks of protocol in 90 studies (49 phase 1 studies and 41 phase 2 studies). From July 2004 through March 2007, there were 49 phase 1 trials in the phase 1 Program, 9 phase 2 studies that were conducted by physicians appointed in that program, and 32 phase 2 trials with accessible data in the Department of Thoracic/Head & Neck Medical Oncology. In the phase 1 trials versus the phase 2 trials, there were significantly more (P < .05) physical examinations (mean +/- standard error, 3.16 +/- 0.24 vs 2.22 +/- 0.13), vital sign determinations (5.63 +/- 0.61 vs 2.80 +/- 0.26), ECGs (4.36 +/- 1.16 vs 0.80 +/- 0.17), nonpharmacokinetic laboratory tests (18.08 +/- 1.31 vs 10.12 +/- 0.65), and PK sampling (15.14 +/- 1.79 vs 1.02 +/- 0.53). These values also differed significantly (P < .005 for each) when the median values were compared in nonparametric tests. Although both phase 1 and phase 2 trials had substantial study requirements, those for the phase 1 studies were significantly higher. The successful conduct of early-phase clinical trials requires significant research infrastructure.
Insights
Early-phase cancer clinical trials, particularly phase 1 studies, involve more intensive requirements than phase 2 trials. Understanding these demands is crucial for oncologists to effectively guide patients toward appropriate early-phase cancer drug studies.
Area of Science:
- Oncology
- Clinical Pharmacology
- Clinical Trial Design
Background:
- Recent advancements in targeted cancer therapies have increased patient eligibility for early-phase drug studies.
- Understanding the requirements of early-phase clinical trials is essential for oncologists to advocate for patients.
- The complexity and workload of early-phase cancer trials need to be quantified to inform trial design and resource allocation.
Purpose of the Study:
- To compare the work intensity of phase 1 and phase 2 early-phase cancer clinical trials.
- To evaluate study requirements as a surrogate for complexity in early-phase cancer trials.
Main Methods:
- A comparative analysis of 90 early-phase cancer clinical trial protocols (49 phase 1, 41 phase 2) was conducted.
- Five parameters were assessed as surrogates of study complexity within the first four weeks of protocol initiation: physical examinations, vital sign determinations, electrocardiograms (ECGs), non-pharmacokinetic laboratory tests, and pharmacokinetic (PK) sampling.
- Data were collected from July 2004 to March 2007.
Main Results:
- Phase 1 cancer trials demonstrated significantly higher requirements across all studied parameters compared to phase 2 trials (P < .05).
- Specific parameters showing significant differences included physical examinations (3.16 vs 2.22), vital sign determinations (5.63 vs 2.80), ECGs (4.36 vs 0.80), non-PK lab tests (18.08 vs 10.12), and PK sampling (15.14 vs 1.02).
- Median value comparisons using nonparametric tests also revealed significant differences (P < .005).
Conclusions:
- Early-phase cancer clinical trials, especially phase 1 studies, impose substantially higher demands on patients and research infrastructure than phase 2 trials.
- The findings underscore the need for robust research infrastructure to successfully conduct early-phase clinical trials.
- Oncologists should be aware of these intensified requirements when considering early-phase trial enrollment for their patients.
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