作者
Giovanni Monteleone, Markus F Neurath, Sandro Ardizzone, Antonio Di Sabatino, Massimo C Fantini, Fabiana Castiglione, Maria L Scribano, Alessandro Armuzzi, Flavio Caprioli, Giacomo C Sturniolo, Francesca Rogai, Maurizio Vecchi, Raja Atreya, Fabrizio Bossa, Sara Onali, Maria Fichera, Gino R Corazza, Livia Biancone, Vincenzo Savarino, Roberta Pica, Ambrogio Orlando, Francesco Pallone
发表日期
2015/3/19
期刊
New England Journal of Medicine
卷号
372
期号
12
页码范围
1104-1113
出版商
Massachusetts Medical Society
简介
Background
Crohn’s disease–related inflammation is characterized by reduced activity of the immunosuppressive cytokine transforming growth factor β1 (TGF-β1) due to high levels of SMAD7, an inhibitor of TGF-β1 signaling. Preclinical studies and a phase 1 study have shown that an oral SMAD7 antisense oligonucleotide, mongersen, targets ileal and colonic SMAD7.
Methods
In a double-blind, placebo-controlled, phase 2 trial, we evaluated the efficacy of mongersen for the treatment of persons with active Crohn’s disease. Patients were randomly assigned to receive 10, 40, or 160 mg of mongersen or placebo per day for 2 weeks. The primary outcomes were clinical remission at day 15, defined as a Crohn’s Disease Activity Index (CDAI) score of less than 150, with maintenance of remission for at least 2 weeks, and the safety of mongersen treatment. A secondary outcome was clinical response (defined as a …
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G Monteleone, MF Neurath, S Ardizzone, A Di Sabatino… - New England Journal of Medicine, 2015