Failed Experiment ReportOpen accessMedicine· cited by 19

Chest computed tomography outcomes in a randomized clinical trial in cystic fibrosis: Lessons learned from the first ataluren phase 3 study

Harm A.W.M. Tiddens; Eleni‐Rosalina Andrinopoulou; Joe McIntosh; J.S. Elborn; Eitan Kerem; N. Bouma; J. Bosch; Mariette Kemner-van de Corput · 2020 · PLoS ONE

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Abstract

A phase 3 randomized double blind controlled, trial in 238 people with cystic fibrosis (CF) and at least one nonsense mutation (nmCF) investigated the effect of ataluren on FEV1. The study was of 48 weeks duration and failed to meet its primary endpoint. Unexpectedly, while FEV1 declined, chest computed tomography (CT) scores using the Brody-II score as secondary outcome measures did not show progression in the placebo group. Based on this observation it was concluded that the role of CT scans in CF randomized clinical trials was limited. However, more sensitive scoring systems were developed

Abstract by Harm A.W.M. Tiddens; Eleni‐Rosalina Andrinopoulou; Joe McIntosh; J.S. Elborn; Eitan Kerem; N. Bouma; J. Bosch; Mariette Kemner-van de Corput, PLoS ONE (2020) — licensed CC BY 4.0.

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