Nirogacestat, a γ-Secretase Inhibitor for Desmoid Tumors

M. Gounder, Mrinal; Ratan, Ravin; Alcindor, Thierry; Schöffski, Patrick; T.A. van der Graaf, Winette; A. Wilky, Breelyn; F. Riedel, Richard; Lim, Allison; Mary Smith, L.; Moody, Stephanie; Attia, Steven; P. Chawla, Sant; Z. D’Amato, Gina; Federman, Noah; Merriam, Priscilla; A. Van Tine, Brian; Vincenzi, Bruno; Benson, Charlotte; Q. Bui, Nam; Chugh, Rashmi; Tinoco, Gabriel; A. Charlson, John; Dileo, Palma; Hartner, Lee; Lapeire, Lore; Mazzeo, F; Palmerini, Emanuela; Reichardt, Peter; Stacchiotti, Silvia; H. Bailey, Howard; Burgess, Melissa; M. Coté, Gregory; E. Davis, Lara; A. Deshpande, Hari; Gelderblom, Hans; Grignani, Giovanni; T. Loggers, Elizabeth; Philip, Tony; G. Pressey, Joseph; Kummar, Shivaani; Kasper, Bernd

doi:10.1056/nejmoa2210140

Public

Nirogacestat, a γ-Secretase Inhibitor for Desmoid Tumors

Published in New England Journal of Medicine • Mar 8, 2023

Authors:

Mrinal M. Gounder

Ravin Ratan

Thierry Alcindor

Abstract

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 Resp...

Subject

Placebo

Medicine

Hazard ratio

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