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相关论文: A Seamless Phase II/III Design with Dose Optimizat…

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Sector duration optimization (SDO) is a problem arising in treatment planning for stereotactic radiosurgery on Gamma Knife. Given a set of isocenter locations, SDO aims to select collimator size configurations and irradiation times thereof…

最优化与控制 · 数学 2022-05-24 Oylum Şeker , Mucahit Cevik , Merve Bodur , Young Lee-Bartlett , Mark Ruschin

In oncology phase I trials, model-assisted designs have been increasingly adopted because they enable adaptive yet operationally simple dose adjustment based on accumulating safety data, leading to a paradigm shift in dose-escalation…

统计方法学 · 统计学 2026-02-23 Kana Yamada , Hisato Sunami , Kentaro Takeda , Keisuke Hanada , Masahiro Kojima

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

Adaptive Phase 2/3 designs hold great promise in contemporary oncology drug development, especially when limited data from Phase 1 dose-finding is insufficient for identifying an optimal dose. However, there is a general concern about…

应用统计 · 统计学 2025-02-25 Cong Chen , Mo Huang

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

The use of drug combinations in clinical trials is increasingly common during the last years since a more favorable therapeutic response may be obtained by combining drugs. In phase I clinical trials, most of the existing methodology…

统计方法学 · 统计学 2020-02-17 José L. Jiménez , Sungjin Kim , Mourad Tighiouart

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

In this article, we propose a phase I-II design in two stages for the combination of molecularly targeted therapies. The design is motivated by a published case study that combines a MEK and a PIK3CA inhibitors; a setting in which higher…

统计方法学 · 统计学 2025-05-21 José L. Jiménez , Mourad Tighiouart

With the development of novel therapies such as molecularly targeted agents and immunotherapy, the maximum tolerated dose paradigm that "more is better" does not necessarily hold anymore. In this context, doses and schedules of novel…

统计方法学 · 统计学 2025-11-24 Anaïs Andrillon , Sandrine Micallef , Moreno Ursino , Pavel Mozgunov , Marie-Karelle Riviere

Dose-finding trials are a key component of the drug development process and rely on a statistical design to help inform dosing decisions. Triallists wishing to choose a design require knowledge of operating characteristics of competing…

统计计算 · 统计学 2025-03-11 Michael Sweeting , Daniel Slade , Dan Jackson , Kristian Brock

We propose a new integrated phase I/II trial design to identify the most efficacious dose combination that also satisfies certain safety requirements for drug-combination trials. We first take a Bayesian copula-type model for dose finding…

应用统计 · 统计学 2011-08-09 Ying Yuan , Guosheng Yin

Conventionally, a first-in-human phase I trial in healthy volunteers aims to confirm the safety of a drug in humans. In such situations, volunteers should not suffer from any safety issues and simple algorithm-based dose-escalation schemes…

Dose-escalation trials in oncology drug development still today typically aim to identify 1-size-fits-all dose recommendations, as arbitrary quantiles of the toxicity thresholds evident in patient samples. In the late 1990s efforts to…

范畴论 · 数学 2025-07-03 David C. Norris

Due to the high cost and high failure rate of Phase III trials, seamless Phase II/III designs are more and more popular to trial efficiency. A potential attraction of Phase II/III design is to allow a randomized proof-of-concept stage prior…

应用统计 · 统计学 2022-06-28 Guanhong Miao , Jason J. Z. Liao , Jing Yang , Keaven Anderson

Phase I-II cancer clinical trial designs are intended to accelerate drug development. In cases where efficacy cannot be ascertained in a short period of time, it is common to divide the study in two stages: i) a first stage in which dose is…

统计方法学 · 统计学 2022-12-13 José L. Jiménez , Mourad Tighiouart

Optimal design of a Phase I cancer trial can be formulated as a stochastic optimization problem. By making use of recent advances in approximate dynamic programming to tackle the problem, we develop an approximation of the Bayesian optimal…

统计方法学 · 统计学 2010-12-01 Jay Bartroff , Tze Leung Lai

Phase I clinical trials are designed to test the safety (non-toxicity) of drugs and find the maximum tolerated dose (MTD). This task becomes significantly more challenging when multiple-drug dose-combinations (DC) are involved, due to the…

机器学习 · 计算机科学 2021-01-27 Hyun-Suk Lee , Cong Shen , William Zame , Jang-Won Lee , Mihaela van der Schaar

Modern clinical trials and cohort studies gather low-cost data on all participants but may have limited resources to assess expensive exposures such as biomarkers or genomic data. When interest lies in associations involving expensive…

统计方法学 · 统计学 2026-05-27 Yunbi Nam , Nathan I. Shapiro , Eric P. Schmidt , Wesley H. Self , Ran Tao , Jonathan S. Schildcrout

Optimizing doses for multiple indications is challenging. The pooled approach of finding a single optimal biological dose (OBD) for all indications ignores that dose-response or dose-toxicity curves may differ between indications, resulting…

统计方法学 · 统计学 2024-10-07 Shuqi Wang , Peter F. Thall , Kentaro Takeda , Ying Yuan

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