Last updated on June 2019

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


Brief description of study

This study evaluates nirogacestat in the treatment of desmoid tumor/aggressive fibromatosis (DT/AF). Half of the participants will receive nirogacestat while the other half will receive placebo.

Detailed Study 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 -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.

Clinical Study Identifier: NCT03785964

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SpringWorks' Site

Berlin, Germany
5.34miles
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Recruitment Status: Open


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