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Related papers: Dual-criterion Dose Finding Designs Based on Dose-…

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Traditional phase I dose finding cancer clinical trial designs aim to determine the maximum tolerated dose (MTD) of the investigational cytotoxic agent based on a single toxicity outcome, assuming a monotone dose-response relationship.…

Methodology · Statistics 2024-11-14 Hao Sun , Hsin-Yu Lin , Jieqi Tu , Revathi Ananthakrishnan , Eunhee Kim

The long-term efficacy of targeted therapeutics for cancer treatment can be significantly limited by the type of therapy and development of drug resistance, inter alia. Experimental studies indicate that the factors enhancing acquisition of…

Tissues and Organs · Quantitative Biology 2021-02-16 Joseph Malinzi , Kevin Bosire Basita , Sara Padidar , Henry A. Adeola

Treatment of cancer has rapidly evolved over time in quite dramatic ways, for example from chemotherapies, targeted therapies to immunotherapies and chimeric antigen receptor T-cells. Nonetheless, the basic design of early phase I trials in…

Applications · Statistics 2024-12-04 Lukas Andreas Widmer , Sebastian Weber , Yunnan Xu , Hans-Jochen Weber

Circulating tumour DNA (ctDNA) detection of molecular residual disease (MRD) in solid tumours correlates strongly with patient outcomes and is being adopted as a new clinical standard. ctDNA levels are known to correlate with tumor volume,…

Other Quantitative Biology · Quantitative Biology 2023-11-02 Dan Stetson , Paul Labrousse , Hugh Russell , David Shera , Chris Abbosh , Brian Dougherty , J. Carl Barrett , Darren Hodgson , James Hadfield

FDA's Project Optimus initiative for oncology drug development emphasizes selecting a dose that optimizes both efficacy and safety. When an inferentially adaptive Phase 2/3 design with dose selection is implemented to comply with the…

Applications · Statistics 2024-12-12 Cong Chen , Mo Huang , Xuekui Zhang

We developed a study design for rare disease clinical trials (RDTs) that efficiently evaluate treatments, promotes access to new treatments during treatment development, and optimizes healthcare resource utilization for future treatment…

Applications · Statistics 2016-07-04 Jian Yong , Sohaib H. Mohammad , Yan Yuan

Phase 1-2 designs provide a methodological advance over phase 1 designs for dose finding by using both clinical response and toxicity. A phase 1-2 trial still may fail to select a truly optimal dose. because early response is not a perfect…

Applications · Statistics 2024-04-03 Cheng-Han Yang , Peter F. Thall , Ruitao Lin

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.…

Methodology · Statistics 2023-03-13 Tianjian Zhou , Yuan Ji

Topology optimization (TO) serves as a widely applied structural design approach to tackle various engineering problems. Nevertheless, sensitivity-based TO methods usually struggle with solving strongly nonlinear optimization problems. By…

Machine Learning · Computer Science 2025-06-16 Jun Yang , Shintaro Yamasaki

Dose-finding clinical trials in oncology aim to estimate the maximum tolerated dose (MTD), based on safety traditionally obtained from the clinician's perspective. While the collection of patient-reported outcomes (PROs) has been advocated…

Applications · Statistics 2023-04-03 Anaïs Andrillon , Lucie Biard , Shing M. Lee

Delineating and planning with respect to regions suspected to contain microscopic tumor cells is an inherently uncertain task in radiotherapy. The recently proposed \textit{clinical target distribution} (CTD) is an alternative to the…

Medical Physics · Physics 2023-04-26 Ivar Bengtsson , Anders Forsgren , Albin Fredriksson

Although optimal control theory has been used for the theoretical study of anti-cancerous drugs scheduling optimization, with the aim of reducing the primary tumor volume, the effect on metastases is often ignored. Here, we use a previously…

Tissues and Organs · Quantitative Biology 2013-06-21 Sebastien Benzekry , Philip Hahnfeldt

Purpose: Various dose calculation algorithms are available for radiation therapy for cancer patients. However, these algorithms are faced with the tradeoff between efficiency and accuracy. The fast algorithms are generally less accurate,…

Medical Physics · Physics 2020-07-01 Yixun Xing , Dan Nguyen , Weiguo Lu , Ming Yang , Steve Jiang

A common problem faced in clinical studies is that of estimating the effect of the most effective (e.g., the one having the largest mean) treatment among $k~(\geq2)$ available treatments. The most effective treatment is adjudged based on…

Statistics Theory · Mathematics 2022-09-20 Masihuddin , Neeraj Misra

The widespread application of Artificial Intelligence (AI) techniques has significantly influenced the development of new therapeutic agents. These computational methods can be used to design and predict the properties of generated…

Neural and Evolutionary Computing · Computer Science 2024-08-21 Arthur Cerveira , Frederico Kremer , Darling de Andrade Lourenço , Ulisses B Corrêa

In parametric Bayesian designs of early phase cancer clinical trials with drug combinations exploring a discrete set of partially ordered doses, several authors claimed that there is no added value in including an interaction term to model…

Methodology · Statistics 2022-08-12 Mourad Tighiouart , José L. Jiménez , Marcio A. Diniz , André Rogatko

Nonlinear regression models addressing both efficacy and toxicity outcomes are increasingly used in dose-finding trials, such as in pharmaceutical drug development. However, research on related experimental design problems for corresponding…

Methodology · Statistics 2016-01-06 Holger Dette , Katrin Kettelhake , Kirsten Schorning , Weng Kee Wong , Frank Bretz

We propose Locally Optimal Restricted Designs (LORDs) for phase I/II dose-finding studies that focus on both efficacy and toxicity outcomes. As an illustrative application, we find various LORDs for a 4-parameter continuation-ratio (CR)…

Methodology · Statistics 2025-09-09 Oleksandr Sverdlov , Yevgen Ryeznik , Weng Kee Wong

We propose a rule-based statistical design for combination dose-finding trials with two agents. The Ci3+3 design is an extension of the i3+3 design with simple decision rules comparing the observed toxicity rates and equivalence intervals…

Applications · Statistics 2023-03-29 Shijie Yuan , Tianjian Zhou , Yawen Lin , Yuan Ji

The primary goal of dose allocation in phase I trials is to minimize patient exposure to subtherapeutic or excessively toxic doses, while accurately recommending a phase II dose that is as close as possible to the maximum tolerated dose…

Methodology · Statistics 2025-03-21 Jiapeng Xu , Dehua Bi , Shenghua Kelly Fan , Bee Leng Lee , Ying Lu