Multinational phase 3 trial of nirogacestat for desmoid tumors
Dr. Mrinal Gounder
Desmoid tumors are rare, locally aggressive, highly recurrent soft-tissue tumors without approved treatments. Nirogacestat is an oral γ-secretase inhibitor. A multinational phase 3 trial evaluated nirogacestat for progressive desmoid tumors in adults. Patients were assigned in a 1:1 ratio to receive nirogacestat or placebo twice daily. Nirogacestat was associated with significant benefits with respect to progression-free survival, objective response, pain, symptom burden, physical functioning, role functioning, and health-related quality of life (quick take video summary).
