Incorporating covariates information in adaptive clinical trials for precision medicine

2021 ◽  
Author(s):  
Wanying Zhao ◽  
Wei Ma ◽  
Fan Wang ◽  
Feifang Hu
Keyword(s):  
Clinical Trials ◽  
Precision Medicine ◽  
Adaptive Clinical Trials

scholarly journals Bayesian adaptive design of early-phase clinical trials for precision medicine based on cancer biomarkers

2021 ◽  
Vol 0 (0) ◽  
Author(s):  
Shinjo Yada
Keyword(s):  
Neural Network ◽  
Clinical Trials ◽  
Precision Medicine ◽  
Early Phase ◽  
Patient Specific ◽  
Specific Gene ◽  
Cancer Type ◽  
Background Characteristics ◽  
Number Of Patients ◽  
Early Phase Clinical Trials

Abstract Cancer tissue samples obtained via biopsy or surgery were examined for specific gene mutations by genetic testing to inform treatment. Precision medicine, which considers not only the cancer type and location, but also the genetic information, environment, and lifestyle of each patient, can be applied for disease prevention and treatment in individual patients. The number of patient-specific characteristics, including biomarkers, has been increasing with time; these characteristics are highly correlated with outcomes. The number of patients at the beginning of early-phase clinical trials is often limited. Moreover, it is challenging to estimate parameters of models that include baseline characteristics as covariates such as biomarkers. To overcome these issues and promote personalized medicine, we propose a dose-finding method that considers patient background characteristics, including biomarkers, using a model for phase I/II oncology trials. We built a Bayesian neural network with input variables of dose, biomarkers, and interactions between dose and biomarkers and output variables of efficacy outcomes for each patient. We trained the neural network to select the optimal dose based on all background characteristics of a patient. Simulation analysis showed that the probability of selecting the desirable dose was higher using the proposed method than that using the naïve method.

scholarly journals A simple solution to the inadequacy of asymptotic likelihood-based inference for response-adaptive clinical trials

2021 ◽  
Author(s):  
Alessandro Baldi Antognini ◽  
Marco Novelli ◽  
Maroussa Zagoraiou
Keyword(s):  
Clinical Trials ◽  
Primary Outcome ◽  
Wald Test ◽  
Simple Solution ◽  
Nuisance Parameters ◽  
Simulation Studies ◽  
Fundamental Properties ◽  
Adaptive Clinical Trials ◽  
Treatment Comparisons ◽  
Practical Recommendations

AbstractThe present paper discusses drawbacks and limitations of likelihood-based inference in sequential clinical trials for treatment comparisons managed via Response-Adaptive Randomization. Taking into account the most common statistical models for the primary outcome—namely binary, Poisson, exponential and normal data—we derive the conditions under which (i) the classical confidence intervals degenerate and (ii) the Wald test becomes inconsistent and strongly affected by the nuisance parameters, also displaying a non monotonic power. To overcome these drawbacks, we provide a very simple solution that could preserve the fundamental properties of likelihood-based inference. Several illustrative examples and simulation studies are presented in order to confirm the relevance of our results and provide some practical recommendations.

Sample size re-estimation in group-sequential response-adaptive clinical trials

2003 ◽  
Vol 22 (24) ◽  
pp. 3843-3857 ◽  
Author(s):  
Caroline C. Morgan
Keyword(s):  
Clinical Trials ◽  
Sample Size ◽  
Group Sequential ◽  
Adaptive Clinical Trials

scholarly journals Critical concepts in adaptive clinical trials

2018 ◽  
Vol Volume 10 ◽  
pp. 343-351 ◽  
Author(s):  
Jay JH Park ◽  
Kristian Thorlund ◽  
Edward J Mills
Keyword(s):  
Clinical Trials ◽  
Adaptive Clinical Trials

The need for precision medicine clinical trials in childhood asthma: rationale and design of the PUFFIN trial

2017 ◽  
Vol 18 (4) ◽  
pp. 393-401 ◽  
Author(s):  
Susanne JH Vijverberg ◽  
Mariëlle W Pijnenburg ◽  
Anke M Hövels ◽  
Gerard H Koppelman ◽  
Anke-Hilse Maitland-van der Zee
Keyword(s):  
Clinical Trials ◽  
Precision Medicine ◽  
Childhood Asthma

scholarly journals Transforming clinical trials in rheumatology: towards patient-centric precision medicine

2020 ◽  
Vol 16 (10) ◽  
pp. 590-599 ◽  
Author(s):  
Costantino Pitzalis ◽  
Ernest H. S. Choy ◽  
Maya H. Buch
Keyword(s):  
Clinical Trials ◽  
Precision Medicine ◽  
Patient Centric

Adaptive clinical trials in tuberculosis: applications, challenges and solutions

2015 ◽  
Vol 19 (6) ◽  
pp. 626-634 ◽  
Author(s):  
G. R. Davies ◽  
P. P. J. Phillips ◽  
T. Jaki
Keyword(s):  
Clinical Trials ◽  
Adaptive Clinical Trials
Sign in / Sign up

Export Citation Format

Share Document