A Phase III Study of Nirogacestat versus Placebo in People with Worsening Desmoid Tumors or Aggressive Fibromatosis


Full 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)


Desmoid tumors (DT) and aggressive fibromatosis (AF) are abnormal growths in the tissue that provides strength and flexibility to structures like bones, ligaments, and muscles (connective tissue). In this study, researchers are evaluating the effectiveness of the investigational drug nirogacestat in patients with DT or AF that is getting worse.

Nirogacestat blocks gamma secretase, a protein that is important for cancer cell growth and survival. Researchers think that nirogacestat may prevent the growth of DT and AF by blocking important pathways that promote their growth. Patients in this study will be randomly assigned to receive either nirogacestat or a placebo (inactive pill), and the researchers will compare the two groups.


To be eligible for this study, patients must meet several criteria, including but not limited to the following:

  • Patients must have DT or AF that has grown by 20 percent or more within the year prior to entering the study.
  • Patients must be able to walk and do routine activities for more than half of their normal waking hours.
  • This study is for patients age 18 and older.

For more information about this study and to inquire about eligibility, please contact Dr. Mrinal Gounder at 646-888-4167.





Disease Status

Newly Diagnosed & Relapsed/Refractory