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

Contact:

NCT Number:

Protocol:

AAAT5220

Study Status:

Active/Enrolling

Population:

Pediatrics

Phase:

II

This is a Phase 2 study of a new cancer fighting drug called Nirogacestat in children and adolescents with relapsed desmoid tumor that cannot be completely removed by surgery. All patients will receive Nirogacestat by mouth twice a day at a dose of 90 mg/m2 (maximum per dose 150 mg). The overall goals of this study are to find out what effects, good and/or bad, Nirogacestat has on children and adolescents with desmoid tumors as well as how well it works in treating desmoid tumor. In addition, we want to see how the body of a child or adolescent processes the drug.

Are you Eligible? (Inclusion Criteria)

  • Patient must be > 12 months and < 18 years of age at the time of enrollment.
  • Patient must have existing or recurrent desmoid tumor that cannot be removed by surgery and has gotten bigger in size within the last 6 months.
  • Patient must have received at least one prior treatment (other than surgery) for desmoid tumor.
  • Patient must also meet all other eligibility criteria as outlined in the study protocol.

Specialty Area(s)

Childhood and Adolescent Cancers (Pediatric)

Principal Investigator

Profile Headshot
  • Section Head of Oncology, Division of Hematology, Oncology, and Stem Cell Transplant

Trial Location(s)

CUIMC/Herbert Irving Pavilion
161 Fort Washington Avenue
New York, NY 10032