A Phase II Study of Nirogacestat to Treat Children and Adolescents with Inoperable Desmoid Tumors


Full Title

A Safety, Pharmacokinetic and Efficacy Study of a y-Secretase Inhibitor, Nirogacestat (PF-03084014, IND 146375), in Children and Adolescents with Progressive, Surgically Unresectable Desmoid Tumors (ARST1921) (CIRB)


The purpose of this study is to assess the safety and effectiveness of the investigational drug nirogacestat in children and adolescents with desmoid tumors that persist despite prior treatment and cannot be surgically removed. Nirogacestat works by inhibiting an enzyme called gamma-secretase, which is involved in the growth of desmoid tumors. It is taken orally (by mouth).


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

  • Patients must have aggressive fibromatosis or a desmoid tumor that continues to grow despite treatment and cannot be removed during surgery.
  • Patients should recover from the serious side effects of previous therapies before receiving nirogacestat.
  • This study is for patients over 1 year of age and under age 18.

For more information about this study and to inquire about eligibility, please contact 1-833-MSK-KIDS.