7 citations · 25 across the 10 of their papers we have counts for
11 papers
Estimands and estimation in trials with time trends
Marta Bofill Roig, Ekkehard Glimm, Kelly Van Lancker +1
In platform trials, model-based approaches typically estimate treatment effects conditional on calendar time. However, scientific and regulatory interest often lies in treatment ef…
An Adaptive Phase II Trial Design for Dose Selection and Addition in Microfilarial Infections
Sonja Zehetmayer, Marta Bofill Roig, Fabrice Lotola Mougeni +3
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)…
Design Considerations for a Phase II platform trial in Major Depressive Disorder
Michaela Maria Freitag, Dario Zocholl, Elias Laurin Meyer +5
Major Depressive Disorder (MDD) is one of the most common causes of disability worldwide. Unfortunately, about one-third of patients do not benefit sufficiently from available trea…
An exhaustive ADDIS principle for online FWER control
Lasse Fischer, Marta Bofill Roig, Werner Brannath
In this paper we consider online multiple testing with familywise error rate (FWER) control, where the probability of committing at least one type I error shall remain under contro…
NCC: An R-package for analysis and simulation of platform trials with non-concurrent controls
Pavla Krotka, Katharina Hees, Peter Jacko +3
Platform trials evaluate the efficacy of multiple treatments, allowing for late entry of the experimental arms and enabling efficiency gains by sharing controls. The power of indiv…
Design of Trials with Composite Endpoints with the R Package CompAREdesign
Jordi Cortés Martinez, Marta Bofill Roig, Guadalupe Gómez Melis
Composite endpoints are widely used as primary endpoints in clinical trials. Designing trials with time-to-event endpoints can be particularly challenging because the proportional…
The Online Closure Principle
Lasse Fischer, Marta Bofill Roig, Werner Brannath
The closure principle is fundamental in multiple testing and has been used to derive many efficient procedures with familywise error rate control. However, it is often unsuitable f…
Adaptive clinical trial designs with blinded selection of binary composite endpoints and sample size reassessment
Marta Bofill Roig, Guadalupe Gómez Melis, Martin Posch +1
For randomized clinical trials where a single, primary, binary endpoint would require unfeasibly large sample sizes, composite endpoints are widely chosen as the primary endpoint.…
Design of phase III trials with long-term survival outcomes based on short-term binary results
Marta Bofill Roig, Yu Shen, Guadalupe Gómez Melis
Pathologic complete response (pCR) is a common primary endpoint for a phase II trial or even accelerated approval of neoadjuvant cancer therapy. If granted, a two-arm confirmatory…
A class of two-sample nonparametric statistics for binary and time-to-event outcomes
Marta Bofill Roig, Guadalupe Gómez Melis
We propose a class of two-sample statistics for testing the equality of proportions and the equality of survival functions. We build our proposal on a weighted combination of a sco…
Decision tool and Sample Size Calculator for Composite Endpoints
Marta Bofill Roig, Jordi Cortés Martínez, Guadalupe Gómez Melis
Summary points: - This article considers the combination of two binary or two time-to-event endpoints to form the primary composite endpoint for leading a trial. - It discusses the…