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The Generalized 3+3 (G3+3) Design for Phase I Dose-Finding Trials

Ji, Y.; Zhang, Y.; Ji, A. L.

2024-08-19 oncology
10.1101/2024.08.18.24312178
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PURPOSEWe propose and demonstrate the feasibility and desirability of a novel model-free dose-finding design for phase I clinical trials. METHODSThe Generalized 3+3 (G3+3) design uses a set of simple rules summarized as follows: For 3 or 6 patients at a dose, apply the 3+3 design for making dosing decisions. For other numbers, if the observed toxicity rate (OTR) is less than 0.2, escalate to the next higher dose; if the OTR is greater than 0.29, de-escalate to the next lower dose; otherwise, stay at the current dose. RESULTSThe G3+3 design is the only design that can replicate the decisions of the 3+3 design for 3 or 6 patients among the popular designs compared like BOIN and i3+3. G3+3 generates desirable decisions when the number of patients treated is not 3 or 6, like the popular designs. Computer simulation verifies the superior operating characteristics of the G3+3 design. CONCLUSIONThe G3+3 design generalizes the popular 3+3 design so that desirable decisions can be made for any number of patients at a dose. G3+3 does not rely on statistical models, is simple and transparent, and can be implemented without a software tool. Therefore, it is expected to facilitate and enhance modern phase I dose-finding trials and early-phase drug development.

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