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The Desmoid Tumor Research Foundation

March 2025

A study to learn if nirogacetat works and is safe for adult participants with desmoid tumors: a plain language summary of the DeFi study

Author Information

Bernd Kasper a, Jeanne Whiting b*, Dana Lawson c, Marlene Portnoy b, Kathrin Schuster d, Christina Baumgarten d, Ravin Ratan a, Allison Lim f*, Sunny Chof *, Shivaani Kummar g & Mrinal Gounder h

aUniversity of Heidelberg, Mannheim University Medical Center, Mannheim Cancer Center, Sarcoma Unit, Mannheim, Germany; bThe Desmoid Tumor Research Foundation, USA; cPatient Author; dSarcoma Patient Advocacy Global Network, Diessen am Ammersee, Germany; eThe Department of Sarcoma Medical Oncology, Division of Cancer Medicine, University of Texas M.D. Anderson Cancer Center, Houston, TX, USA; fSpringWorks Therapeutics, Inc., Stamford, CT, USA; gThe Knight Cancer Institute, Oregon Health and Science University, Portland, OR, USA; hSarcoma Medical Oncology, Department of Medicine, Memorial Sloan Kettering Cancer Center, and Weill Cornell Medical College, New York, NY, USA

Publication

Future Oncology

Summary

This is a plain langauge summary of the DeFi study, published in The New England Journal of Medicine in March 2023. In the DeFi study, researchers were studying if the drug called nirogacestat (Ogsiveo®) could slow disease progression and improve disease symptoms. The study included 142 adults with progressing desmoid tumors.

View Abstract