Data source
tumor, ICH, DT/AF, Tumors, intra-abdominal, fibromatosis
DESMOID, TUMOR, TUMORS, ONCOLOGY, FIBROMATOSIS
Analyses were conducted using blinded data from the Phase 3, double-blind, randomized, placebo-controlled study of nirogacestat in adults with desmoid tumor/aggressive fibromatosis (DT/AF) (DeFi; NCT03785964). Patients were administered nirogacestat or placebo tablets twice daily continuously in 28-day cycles. Eligible patients were ≥ 18 years old, with histologically confirmed DT/AF that progressed by ≥ 20% per Response Evaluation Criteria In Solid Tumors (RECIST) v1.1 within 12 months of screening, and an Eastern Cooperative Oncology Group (ECOG) Performance Status (PS) score ≤ 2. A 1:1 randomization allocation was stratified based on tumor location: intra-abdominal (including mesentery/pelvis) and extra-abdominal (including head/neck, para-spinal, extremities, abdominal wall, chest wall, and other). DeFi was conducted in accordance with Good Clinical Practice; all study materials were approved by the appropriate ethics body at each participating center. Informed consent was obtained prior to any study procedures and follows ICH GCP/CFR guidelines. Consent included the collection and use of NIR-DT-301 study data, which were used for the validation of the GODDESS