Nirogacestat, a γ-Secretase Inhibitor for Desmoid Tumors
Memorial Sloan Kettering Cancer Center · Cornell University · +52 more institutions
Abstract
Desmoid tumors are rare, locally aggressive, highly recurrent soft-tissue tumors without approved treatments.
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 γ-secretase inhibitor nirogacestat (150 mg) or placebo twice daily. The primary end point was progression-free survival.
Citation impact
- FWCI
- 53.50
- Percentile
- 100%
- References
- 39
Authors
41- MMMrinal M. GounderCorresponding
Memorial Sloan Kettering Cancer Center, Cornell University
- RRRavin Ratan
Memorial Sloan Kettering Cancer Center, The University of Texas MD Anderson Cancer Center, Cornell University
- TAThierry Alcindor
Memorial Sloan Kettering Cancer Center, Cornell University, McGill University
- PSPatrick Schöffski
Memorial Sloan Kettering Cancer Center, Cornell University, KU Leuven
- WTWinette T.A. van der Graaf
Memorial Sloan Kettering Cancer Center, Cornell University, The Netherlands Cancer Institute
Topics & keywords
- Placebo
- Medicine
- Hazard ratio
- Internal medicine
- Nausea
- Adverse effect
- Gastroenterology
- Clinical endpoint
- Good health and well-being