Nirogacestat for Adults With Desmoid Tumor/Aggressive Fibromatosis (DT/AF) — DeFi  

Desmoid Tumor Clinical Trial

Official title: A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Trial of Nirogacestat Versus Placebo in Adult Patients With Progressing Desmoid Tumors/Aggressive Fibromatosis (DT/AF)

Status Active, not recruiting

Clinical Trial Summary

This study evaluates nirogacestat (PF-03084014) in the treatment of desmoid tumor/aggressive fibromatosis (DT/AF). In the double-blind phase, half of the participants will receive nirogacestat while the other half will receive placebo. Once participants are eligible to roll into the open-label phase, they will receive nirogacestat.

Clinical Trial Description

Desmoid tumors, also referred to as aggressive fibromatosis, are rare, locally invasive, slow growing soft tissue tumors. Although considered benign because of their inability to metastasize, desmoid tumors can cause significant morbidity and occasionally mortality in patients.

Nirogacestat (PF-03084014) is a potent, small molecule, selective, reversible, noncompetitive inhibitor of γ-secretase (GS) with a potential antitumor activity.

Nirogacestat is being investigated for the treatment of desmoid tumors due to its ability to bind to GS, blocking proteolytic activation of Notch receptors. Previous clinical study data have shown that Notch signaling plays an important role in cancer development. Hence, inhibition of Notch signaling is an important strategy for therapeutic treatment. ;

Related Conditions & MeSH terms

• Aggression
• Aggressive Fibromatosis
• Desmoid Tumor
• Fibroma

• NCT number: NCT03785964
• Study type: Interventional
• Source: SpringWorks Therapeutics, Inc.
• Status: Active, not recruiting
• Phase: Phase 3
• Start date: April 17, 2019
• Completion date: December 31, 2024