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Phase I dose-finding studies aim at identifying the maximal tolerated dose (MTD). It is not uncommon that several dose-finding studies are conducted, although often with some variation in the administration mode or dose panel. For instance,…

Quantitative Methods · Quantitative Biology 2021-03-24 Moreno Ursino , Christian Röver , Sarah Zohar , Tim Friede

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…

Methodology · Statistics 2020-02-17 José L. Jiménez , Sungjin Kim , Mourad Tighiouart

Model-assisted designs have garnered significant attention in recent years due to their high accuracy in identifying the maximum tolerated dose (MTD) and their operational simplicity. To identify the MTD, they employ estimated dose limiting…

Applications · Statistics 2025-08-19 Rentaro Wakayama , Tomotaka Momozaki , Shuji Ando

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…

Methodology · Statistics 2022-12-13 José L. Jiménez , Mourad Tighiouart

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…

Applications · Statistics 2021-05-03 Helen Barnett , Oliver Boix , Dimintris Kontos , Thomas Jaki

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…

Machine Learning · Statistics 2025-04-15 Keiichiro Seno , Kota Matsui , Shogo Iwazaki , Yu Inatsu , Shion Takeno , Shigeyuki Matsui

Dose-finding clinical trials in oncology aim to determine the maximum tolerated dose (MTD) of a new drug, generally defined by the proportion of patients with short-term dose-limiting toxicities (DLTs). Model-based approaches for such phase…

Methodology · Statistics 2020-12-08 Moreno Ursino , Lucie Biard , Sylvie Chevret

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…

Methodology · Statistics 2016-08-14 Mourad Tighiouart , André Rogatko

The primary objective of Phase I oncology trials is to assess the safety and tolerability of novel therapeutics. Conventional dose escalation methods identify the maximum tolerated dose (MTD) based on dose-limiting toxicity (DLT). However,…

Methodology · Statistics 2024-09-02 Yunlong Yang , 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…

Applications · Statistics 2026-01-13 Kyong Ju Lee , Yuan Ji

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…

Applications · Statistics 2022-03-02 Huaqing Jin , Wenbin Du , Guosheng Yin

Phase I dose-finding trials in oncology seek to find the maximum tolerated dose (MTD) of a drug under a specific schedule. Evaluating drug-schedules aims at improving treatment safety while maintaining efficacy. However, while we can…

Background: Phase I trials desire to identify the maximum tolerated dose (MTD) early and proceed quickly to an expansion cohort or phase II trial for efficacy. We propose an early completion method based on multiple dosages to accelerate…

Quantitative Methods · Quantitative Biology 2021-10-04 Masahiro Kojima

Drug combination trials are increasingly common nowadays in clinical research. However, very few methods have been developed to consider toxicity attributions in the dose escalation process. We are motivated by a trial in which the…

Methodology · Statistics 2018-08-23 Jose L. Jimenez , Mourad Tighiouart , Mauro Gasparini

Combination of several anti-cancer treatments has typically been presumed to have enhanced drug activity. Motivated by a real clinical trial, this paper considers phase I-II dose finding designs for dual-agent combinations, where one main…

Methodology · Statistics 2023-05-09 José L. Jiménez , Haiyan Zheng

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…

Machine Learning · Computer Science 2021-01-27 Hyun-Suk Lee , Cong Shen , William Zame , Jang-Won Lee , Mihaela van der Schaar

An early phase clinical trial is the first step in evaluating the effects in humans of a potential new anti-disease agent or combination of agents. Usually called "phase I" or "phase I/II" trials, these experiments typically have the…

Methodology · Statistics 2010-12-01 Peter F. Thall

Traditional dose selection for oncology registration trials typically employs a one- or two-step single maximum tolerated dose (MTD) approach. However, this approach may not be appropriate for molecularly targeted therapy that tends to have…

Methodology · Statistics 2023-09-28 Jason J. Z. Liao , Ekaterine Asatiani , Qingyang Liu , Kevin Hou

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,…

Methodology · Statistics 2025-08-19 Ayon Mukherjee , Jonathan L. Moscovici , Zheng Liu

Phase I dose-finding trials are increasingly challenging as the relationship between efficacy and toxicity of new compounds (or combination of them) becomes more complex. Despite this, most commonly used methods in practice focus on…

Machine Learning · Computer Science 2020-06-16 Cong Shen , Zhiyang Wang , Sofia S. Villar , Mihaela van der Schaar
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