Nirogacestat, a gamma-Secretase Inhibitor for Desmoid Tumors
Mené sur 142 patients atteints d'une tumeur desmoïde, cet essai international de phase III évalue l'efficacité, du point de vue de la survie sans progression, du nirogacestat (un inhibiteur de la gamma sécrétase)
New England Journal of Medicine, Volume 388, Numéro 10, Page 898-912, 2023, résumé
Résumé en anglais
BACKGROUND : Desmoid tumors are rare, locally aggressive, highly recurrent soft-tissue tumors without approved treatments.
METHODS : We conducted a phase 3, international, double-blind, randomized, placebo-controlled trial of nirogacestat in adults with progressing desmoid tumors according to the Response Evaluation Criteria in Solid Tumors, version 1.1. Patients were assigned in a 1:1 ratio to receive the oral
