NCT03785964

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

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: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 142 (actual)
• Sponsor: SpringWorks Therapeutics, Inc. · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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.

Detailed 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.

Conditions

• Desmoid Tumor
• Aggressive Fibromatosis

Interventions

Type	Name	Description
DRUG	Nirogacestat oral tablet	Nirogacestat tablet
DRUG	Placebo Oral Tablet	Sugar pill manufactured to mimic nirogacestat 50 mg tablet

Timeline

• Start date: 2019-04-17
• Primary completion: 2022-04-07
• Completion: 2024-10-17
• First posted: 2018-12-24
• Last updated: 2025-01-31
• Results posted: 2024-06-12

Locations

52 sites across 7 countries: United States, Belgium, Canada, Germany, Italy, Netherlands, United Kingdom

Regulatory

• FDA-regulated drug study