ArticleCancer2018
Statistical justification of expansion cohorts in phase 1 cancer trials.
Article in Cancer, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT04525859 (Direct Injection of Poly-ICLC), which is not on this map. Cited by 4 papers.
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Direct Injection of Poly-ICLC (Hiltonol®) Vaccine In Malignant Pleural Mesothelioma
Who cites it
4 citing papers in PubMed.
- TODO: A Triple-Outcome Double-Criterion Optimal Design for Dose Monitoring-and-Optimization in Multi-Dose Randomized Trials.Statistics in medicine · 2025Article
- A Road Map for Designing Phase I Clinical Trials of Radiotherapy-Novel Agent Combinations.Clinical cancer research : an official journal of the American Association for Cancer Research · 2022Review
- Pharmacokinetic-Pharmacometabolomic Approach in Early-Phase Clinical Trials: A Way Forward for Targeted Therapy in Type 2 Diabetes.Pharmaceutics · 2022Article
- Flexible, rule-based dose escalation: The cohort-sequence design.Contemporary clinical trials communications · 2020Article
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Authors and funding
5 authors.
Funding
Abstract
backgroundPhase I cancer trials increasingly incorporate dose-expansion cohorts (DECs), reflecting a growing demand to acquire more information about investigational drugs. Protocols commonly fail to provide a sample-size justification or analysis plan for the DEC. In this study, we develop a statistical framework for the design of DECs.
methodsWe assume the maximum tolerated dose (MTD) for the investigational drug has been identified in the dose-escalation stage of the trial. We use the 80% lower confidence bound and the 90% upper confidence bound for the response and toxicity rates, respectively, as decision thresholds for the dose-expansion stage. We calculate the operating characteristics with reference to prespecified minimum effective response rates and maximum safe DLT rates.
resultsWe apply our framework to specify a system of DEC plans. The design comprises three components: 1) the number of subjects enrolled at the MTD, 2) the minimum number of responses necessary to indicate provisional drug efficacy, and 3) the maximum number of dose-limiting toxicities (DLTs) permitted to indicate drug safety. We demonstrate our method in an application to a cancer immunotherapy trial.
conclusionsOur simple and practical tool enables creation of DEC designs that appropriately address the safety and efficacy objectives of the trial.
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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.