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As the availability of omics data has increased in the last few years, more multi-omics data have been generated, that is, high-dimensional molecular data consisting of several types such as genomic, transcriptomic, or proteomic data, all…

Genomics · Quantitative Biology 2023-02-09 Roman Hornung , Frederik Ludwigs , Jonas Hagenberg , Anne-Laure Boulesteix

Recently, the strategy for dose optimization in oncology has shifted to conduct Phase 2 randomized controlled trials with multiple doses. Optimal biologic dose selection from Phase 1 trial data to determine candidate doses for Phase 2…

Methodology · Statistics 2023-02-14 Masahiro Kojima

Problem Definition. Increasing costs of healthcare highlight the importance of effective disease prevention. However, decision models for allocating preventive care are lacking. Methodology/Results. In this paper, we develop a data-driven…

Machine Learning · Computer Science 2023-08-15 Mathias Kraus , Stefan Feuerriegel , Maytal Saar-Tsechansky

Combination drug therapies hold significant promise for enhancing treatment efficacy, particularly in fields such as oncology, immunotherapy, and infectious diseases. However, designing clinical trials for these regimens poses unique…

Methodology · Statistics 2025-06-04 Nan Miles Xi , Man Mandy Jin , Lin Wang , Xin Huang

In the era of precision medicine, more and more clinical trials are now driven or guided by biomarkers, which are patient characteristics objectively measured and evaluated as indicators of normal biological processes, pathogenic processes,…

Methodology · Statistics 2024-01-02 Hong Zhang , Jie Pu , Shibing Deng , Satrajit Roychoudhury , Haitao Chu , Douglas Robinson

An individualized dose rule recommends a dose level within a continuous safe dose range based on patient level information such as physical conditions, genetic factors and medication histories. Traditionally, personalized dose finding…

Methodology · Statistics 2020-07-21 Liangyu Zhu , Wenbin Lu , Michael R. Kosorok , Rui Song

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…

Methodology · Statistics 2026-05-07 Yuxuan Chen , Haiming Zhou , Keiko Nakajima , Philip He

We propose the BayesDose-Framework, a Bayesian approach for fast and accurate dose prediction in proton therapy. Our framework is based on a previously published deterministic LSTM model and is trained and evaluated on simulated beamlet…

Medical Physics · Physics 2023-07-04 Luke Voss , Ahmad Neishabouri , Tim Ortkamp , Andrea Mairani , Niklas Wahl

At the hospital, the dispersion of information regarding anti-cancer treatment makes it difficult to extract. We proposed a solution capable of identifying dates, drugs and their temporal relationship within free-text oncology reports with…

Quantitative Methods · Quantitative Biology 2024-10-15 Valentin Pohyer , Elizabeth Fabre , Stéphane Oudard , Laure Fournier , Bastien Rance

Evaluating the influence of continuous covariates, like exposure time or dose, on a response variable is a pivotal objective in the assessment of a compound's effect, particularly when determining toxicity in pre-clinical research or…

Methodology · Statistics 2026-04-16 Lucia Ameis , Niklas Hagemann , Kathrin Möllenhoff

An important tool to evaluate the performance of any design is an optimal benchmark proposed by O'Quigley and others (2002, Biostatistics 3(1), 51-56) that provides an upper bound on the performance of a design under a given scenario. The…

Statistics Theory · Mathematics 2018-03-06 Pavel Mozgunov , Thomas Jaki , Xavier Paoletti

In this paper we consider two-stage adaptive dose-response study designs, where the study design is changed at an interim analysis based on the information collected so far. In a simulation study, two approaches will be compared for these…

Methodology · Statistics 2016-02-08 Emma McCallum , Björn Bornkamp

We extend conformal inference to general settings that allow for time series data. Our proposal is developed as a randomization method and accounts for potential serial dependence by including block structures in the permutation scheme. As…

Machine Learning · Statistics 2019-07-09 Victor Chernozhukov , Kaspar Wuthrich , Yinchu Zhu

We propose a frequentist adaptive phase 2 trial design to evaluate the safety and efficacy of three treatment regimens (doses) compared to placebo for four types of helminth (worm) infections. This trial will be carried out in four…

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

Oncology drug development starts with a dose escalation phase to find the maximal tolerable dose (MTD). Dose limiting toxicity (DLT) is the primary endpoint for dose escalation phase. Traditionally, model-based dose escalation trial designs…

Applications · Statistics 2024-04-18 Arnab Kumar Maity , Satrajit Roy Chowdhury , Ray Li , Lada Markovtsova , Roberto Bugarini

Chemotherapy is one of the primary modalities of cancer treatment. Chemotherapy drug administration is a complex problem that often requires expensive clinical trials to evaluate potential regimens. One way to alleviate this burden and…

Optimization and Control · Mathematics 2021-11-04 Temitayo Ajayi , Seyedmohammadhossein Hosseinian , Andrew J. Schaefer , Clifton D. Fuller

Broadening eligibility criteria in cancer trials has been advocated to represent the true patient population more accurately. While the advantages are clear in terms of generalizability and recruitment, novel dose-finding designs are needed…

Applications · Statistics 2023-01-12 Rebecca B. Silva , Bin Cheng , Richard D. Carvajal , Shing M. Lee

We propose a virtual clinical trial for assessing the safety and efficacy of closed-loop diabetes treatments prior to an actual clinical trial. Such virtual trials enable rapid and risk-free pretrial testing of algorithms, and they can be…

Optimization and Control · Mathematics 2022-05-04 Tobias K. S. Ritschel , Asbjørn Thode Reenberg , John Bagterp Jørgensen

We consider the problem of estimating a dose-response curve. Continuous treatments arise often in practice, e.g. in the form of time spent on an operation, distance traveled to a location or dosage of a drug. Letting $A$ denote a continuous…

Methodology · Statistics 2026-04-14 Matteo Bonvini , Edward H. Kennedy