20 papers · ranked by Valyu relevance
Guanhong Miao, Jason J. Z. Liao, Jing Yang, Keaven M. Anderson
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 to committing to the full cost of the Phase III trial. Population selection…
Ruyi Liu, Fan Li, Denise Esserman, Mary Ryan
The two-stage preference design (TSPD) enables the inference for treatment efficacy while allowing for incorporation of patient preference to treatment. It can provide unbiased estimates for selection and preference effects, where a selection effect occurs when patients who prefer one treatment respond differently than…
David S. Robertson, Babak Choodari‐Oskooei, Munya Dimairo, Laura Flight + 2 more
'Laura Flight' 'Philip Pallmann' 'Thomas Jaki'] In adaptive clinical trials, the conventional end-of-trial point estimate of a treatment effect is prone to bias, that is, a systematic tendency to deviate from its true value. As stated in recent FDA guidance on adaptive designs, it is desirable to report estimates of…
Stephen Schüürhuis, Gernot Wassmer, Meinhard Kieser, Friedrich Pahlke + 2 more
Background In group-sequential designs, it is typically assumed that there is no time gap between patient enrollment and outcome measurement in clinical trials. However, in practice, there is usually a lag between the two time points. This can affect the statistical analysis of the data, especially in trials with…
Zhangyi He, Suzie Cro, Laurent Billot
Group sequential designs (GSDs) are well established and the most commonly used adaptive design in confirmatory clinical trials with interim analyses. However, they remain underutilised, and their implementation involves unique theoretical and practical decisions that demand careful consideration to optimise…
Long-Hao Xu, Tobias Mütze, Frank Konietschke, Tim Friede
In group sequential designs, where several data looks are conducted for early stopping, we generally assume the vector of test statistics from the sequential analyses follows (at least approximately or asymptotially) a multivariate normal distribution. However, it is well-known that test statistics for which an…
Fraser Lewis
A group sequential clinical trial design can be an attractive option when planning a pivotal trial as this approach has the ability to stop the trial early for success, whilst also being well accepted from a regulatory review perspective. Compared to a single stage design there are more moving parts to consider and…
Nick R. Parsons, Nigel Stallard, Helen Parsons, Aminul Haque + 11 more
'Martin Underwood' 'James Mason' 'Iftekhar Khan' 'Matthew L. Costa' 'Damian R. Griffin' 'James Griffin' 'David J. Beard' 'Jonathan A. Cook' 'Loretta Davies' 'Jemma Hudson' 'Andrew Metcalfe'] Background Assessing the long term effects of many surgical interventions tested in pragmatic RCTs may require extended periods…
Guanhong Miao, Jason J. Z. Liao, Jing Yang, Keaven Anderson
Background Due to the high cost and high failure rate of Phase III trials where a classical group sequential design (GSD) is usually used, seamless Phase II/III designs are more and more popular to improve trial efficiency. A potential attraction of Phase II/III design is to allow a randomized proof-of-concept stage…
Sonja Zehetmayer, Martin Posch, Franz Koenig
When testing multiple hypotheses, a suitable error rate should be controlled even in exploratory trials. Conventional methods to control the False Discovery Rate assume that all p-values are available at the time point of test decision. In platform trials, however, treatment arms enter and leave the trial at different…
John P. Veillette, Letitia Ho, Howard C. Nusbaum
Cognitive neuroscientists have been grappling with two related experimental design problems. First, the complexity of neuroimaging data (e.g. often hundreds of thousands of correlated measurements) and analysis pipelines demands bespoke, non-parametric statistical tests for valid inference, and these tests often lack…
Mengyan Zhang, Maciej Bartosz Holowko, Huw Hayman Zumpe, Cheng Soon Ong
Optimisation of gene expression levels is an essential part of the organism design process. Fine control of this process can be achieved through engineering transcription and translation control elements, including the ribosome binding site (RBS). Unfortunately, design of specific genetic parts can still be challenging…
Qi Zhang, Chang Liu, Stephen Wu, Ryo Yoshida
In the last few years, de novo molecular design using machine learning has made great technical progress but its practical deployment has not been as successful. This is mostly owing to the cost and technical difficulty of synthesizing such computationally designed molecules. To overcome such barriers, various methods…
Laura Etfer, James Wason, Michael J. Grayling
binary outcome data Authors: ['Laura Etfer' 'James Wason' 'Michael J. Grayling'] Introduction: A number of recent articles have investigated the optimal design of stepped-wedge cluster randomised trials. Each of these has focused on the case of normally distributed outcomes, analysed using a linear mixed model. This is…
Authors not listed
Incorporating non-natural amino acids (NNAAs) into peptides enhances therapeutic properties, including binding affinity, metabolic stability, and in vivo half-life time. The pursuit of novel NNAAs for improved peptide designs faces the challenge of effective synthesis. Solid-Phase Peptide Synthesis (SPPS) is an…
Gavin Hsu, A. Duke Shereen, Leonardo G. Cohen, Lucas C. Parra
Motor learning experiments with transcranial direct current stimulation (tDCS) at 2mA have produced mixed results. We hypothesize that tDCS will boost motor learning provided sufficiently high field intensity on the motor cortex. In a single-blinded, between-subject design, 72 healthy right-handed participants received…
Helena Wright-Wieckowski, Kate Wilmut, Katja Kornysheva
Research suggests that motor difficulties in Developmental Coordination Disorder (DCD) are related to altered motor sequence planning, but it is unclear which mechanisms are affected, particularly in adults. This study addresses that gap by examining how the order of upcoming movements during the planning of skilled…
Helena Wright-Wieckowski, Jason Friedman, Joseph M. Galea, Katja Kornysheva
Results in human and non-human primates have shown that elements of a movement sequence are pre-ordered in parallel competitively before execution, a process known as competitive queueing (CQ). However, it is unclear whether the preplanning of individual movements persists in continuous skilled actions that involve…
Authors not listed
Sequence is the critical determinant of macromolecular function, yet current polymer design approaches often optimize monomer composition and ratios while ignoring sequence. This creates poorly defined design spaces for active learning that miss the vast combinatorial landscape of sequence possibilities. We introduce…
Babu Bassa
In this communication the author describes a software tool named "ChameleonSort". The software program, developed by the present author is useful in the sorting of biological sequence variants like those accumulating mutations while diverging from the common ancestors. Examples include viral protein variants, protein…