Nirogacestat, a γ-Secretase Inhibitor for Desmoid Tumors.

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 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.

Results

From May 2019 through August 2020, a total of 70 patients were assigned to receive nirogacestat and 72 to receive placebo. Nirogacestat had a significant progression-free survival benefit over placebo (hazard ratio for disease progression

SUBMITTER: Gounder M 

PROVIDER: S-EPMC11225596 | biostudies-literature | 2023 Mar

REPOSITORIES: biostudies-literature