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Immunotherapies and targeted therapies have gained popularity due to their promising therapeutic effects across multiple treatment areas. The focus of early phase dose-finding clinical trials has shifted from finding the maximum tolerated…

统计方法学 · 统计学 2023-12-27 Hao Sun , Jieqi Tu

The primary goal of a two-stage Phase I/II trial is to identify the optimal dose for the following large-scale Phase III trial. Recently, Phase I dose-finding designs have shifted from identifying the maximum tolerated dose (MTD) to the…

统计方法学 · 统计学 2025-01-16 Hao Sun , Jerry Li

Oncology dose-finding trials are shifting from identifying the maximum tolerated dose (MTD) to determining the optimal biological dose (OBD), driven by the need for efficient methods that consider both toxicity and efficacy. This is…

统计方法学 · 统计学 2025-04-01 Hao Sun , Jieqi Tu , Revathi Ananthakrishnan , Eunhee Kim

In Phase I/II dose-finding trials, the objective is to find the Optimal Biological Dose (OBD), a dose that is both safe and efficacious that maximises some optimality criterion based on safety and efficacy. This is further complicated when…

应用统计 · 统计学 2022-03-31 Helen Barnett , Oliver Boix , Dimitris Kontos , Thomas Jaki

Phase I dose escalation trials in oncology generally aim to find the maximum tolerated dose (MTD). However, with the advent of molecular targeted therapies and antibody drug conjugates, dose limiting toxicities are less frequently observed,…

统计方法学 · 统计学 2025-08-19 Ayon Mukherjee , Jonathan L. Moscovici , Zheng Liu

The purpose of a phase I dose-finding clinical trial is to investigate the toxicity profiles of various doses for a new drug and identify the maximum tolerated dose. Over the past three decades, various dose-finding designs have been…

统计方法学 · 统计学 2021-11-25 Yunshan Duan , Shijie Yuan , Yuan Ji , Peter Mueller

The landscape of dose-finding designs for phase I clinical trials is rapidly shifting in the recent years, noticeably marked by the emergence of interval-based designs. We categorize them as the iDesigns and the IB-Designs. The iDesigns are…

统计方法学 · 统计学 2017-06-15 Yuan Ji , Shengjie Yang

The US Food and Drug Administration (FDA) launched Project Optimus and issued guidance to reform dose-finding and selection trials, shifting the paradigm from identifying the maximum tolerable dose (MTD) to determining the optimal…

统计方法学 · 统计学 2025-09-16 Kai Chen , Yixuan Zhao , Kentaro Takeda , Ying Yuan

In oncology dose-finding trials, due to staggered enrollment, it might be desirable to make dose-assignment decisions in real-time in the presence of pending toxicity outcomes, for example, when the dose-limiting toxicity is late-onset.…

统计方法学 · 统计学 2023-03-13 Tianjian Zhou , Yuan Ji

The primary objective of phase I cancer clinical trials is to evaluate the safety of a new experimental treatment and to find the maximum tolerated dose (MTD). We show that the MTD estimation problem can be regarded as a level set…

机器学习 · 统计学 2025-04-15 Keiichiro Seno , Kota Matsui , Shogo Iwazaki , Yu Inatsu , Shion Takeno , Shigeyuki Matsui

In the development of new cancer treatment, an essential step is to determine the maximum tolerated dose (MTD) via phase I clinical trials. Generally speaking, phase I trial designs can be classified as either model-based or algorithm-based…

应用统计 · 统计学 2022-03-02 Huaqing Jin , Wenbin Du , Guosheng Yin

Purpose: The early identification of maximum tolerated dose (MTD) in phase I trial leads to faster progression to a phase II trial or an expansion cohort to confirm efficacy. Methods: We propose a novel adaptive design for identifying MTD…

统计方法学 · 统计学 2021-10-07 Masahiro Kojima

An objective of phase I dose-finding trials is to find the maximum tolerated dose; the dose with a particular risk of toxicity. Frequently, this risk is assessed across the first cycle of therapy. However, in oncology, a course of treatment…

应用统计 · 统计学 2021-05-03 Helen Barnett , Oliver Boix , Dimintris Kontos , Thomas Jaki

Traditionally, the major objective in phase I trials is to identify a working-dose for subsequent studies, whereas the major endpoint in phase II and III trials is treatment efficacy. The dose sought is typically referred to as the maximum…

统计方法学 · 统计学 2016-08-14 Mourad Tighiouart , André Rogatko

We propose a Bayesian optimal phase 2 design for jointly monitoring efficacy and toxicity, referred to as BOP2-TE, to improve the operating characteristics of the BOP2 design proposed by Zhou et al. (2017). BOP2-TE utilizes a…

统计方法学 · 统计学 2024-08-13 Kai Chen , Heng Zhou , J. Jack Lee , Ying Yuan

Phase I oncology trials aim to identify a safe dose - often the maximum tolerated dose (MTD) - for subsequent studies. Conventional designs focus on population-level toxicity modeling, with recent attention on leveraging pharmacokinetic…

应用统计 · 统计学 2026-01-13 Kyong Ju Lee , Yuan Ji

Purpose: During discussions at the Data Science Roundtable meeting in Japan, there were instances where the adoption of the BOIN design was declined, attributed to the extension of study duration and increased sample size in comparison to…

定量方法 · 定量生物学 2023-09-19 Masahiro Kojima , Wu Wende , Henry Zhao

Cohort-based enrollment can slow down dose-finding trials since the outcomes of the previous cohort must be fully evaluated before the next cohort can be enrolled. This results in frequent suspension of patient enrollment. The issue is…

应用统计 · 统计学 2020-01-01 Tianjian Zhou , Wentian Guo , Yuan Ji

In phase I dose escalation studies for dual-agent combinations, at least one drug often has an established monotherapy dose. Consequently, substantial prior clinical safety data often exist for one or more monotherapies, allowing the study…

统计方法学 · 统计学 2026-05-07 Yuxuan Chen , Haiming Zhou , Keiko Nakajima , Philip He

The initiation of dose optimization has driven a paradigm shift in oncology clinical trials to determine the optimal biological dose (OBD). Early-phase trials with randomized doses can facilitate additional investigation of the identified…

统计方法学 · 统计学 2025-02-27 Gina DAngelo , Guannan Chen , Di Ran
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