ArticleStatistics in medicine2017
Sequential monitoring of Phase I dose expansion cohorts.
Article in Statistics in medicine, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 11 papers.
What it found
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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.
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Who cites it
11 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
- CONSORT-DEFINE explanation and elaboration: recommendations for enhancing reporting quality and impact of early phase dose-finding clinical trials.EClinicalMedicine · 2025Article
- SPIRIT-DEFINE explanation and elaboration: recommendations for enhancing quality and impact of early phase dose-finding clinical trials protocols.EClinicalMedicine · 2025Article
- Comparison Between Simultaneous and Sequential Utilization of Safety and Efficacy for Optimal Dose Determination in Bayesian Model-Assisted Designs.Therapeutic innovation & regulatory science · 2023Article
- Controlled amplification in oncology dose-finding trials.Contemporary clinical trials · 2023Article
- Phase I clinical trials in adoptive T-cell therapies.Journal of the Royal Statistical Society. Series C, Applied statistics · 2021Article
- Clinical pharmacology applications in clinical drug development and clinical care: A focus on Saudi Arabia.Saudi pharmaceutical journal : SPJ : the official publication of the Saudi Pharmaceutical Society · 2020Review
- How to design a dose-finding study using the continual reassessment method.BMC medical research methodology · 2019Article
- Article
- Dimension of model parameter space and operating characteristics in adaptive dose-finding studies.Statistics in medicine · 2016Article
- Dose expansion cohorts in Phase I trials.Statistics in biopharmaceutical researchArticle
Corrections and comments
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Authors and funding
2 authors.
Funding
Abstract
A relatively recent development in the design of Phase I dose-finding studies is the inclusion of expansion cohort(s), that is, the inclusion of several more patients at a level considered to be the maximum tolerated dose established at the conclusion of the 'pure' Phase I part. Little attention has been given to the additional statistical analysis, including design considerations, that we might wish to consider for this more involved design. For instance, how can we best make use of new information that may confirm or may tend to contradict the estimate of the maximum tolerated dose based on the dose escalation phase. Those patients included during the dose expansion phase may possess different eligibility criteria. During the expansion phase, we will also wish to have an eye on any evidence of efficacy, an aspect that clearly distinguishes such studies from the classical Phase I study. Here, we present a methodology that enables us to continue the monitoring of safety in the dose expansion cohort while simultaneously trying to assess efficacy and, in particular, which disease types may be the most promising to take forward for further study. The most elementary problem is where we only wish to take account of further toxicity information obtained during the dose expansion cohort, and where the initial design was model based or the standard 3+3. More complex set-ups also involve efficacy and the presence of subgroups. Copyright © 2016 John Wiley & Sons, Ltd.
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