Study of Nirogacestat in Children and Adolescents With Progressive, Surgically Unresectable Desmoid Tumors
A Safety, Pharmacokinetic and Efficacy Study of a γ-Secretase Inhibitor, Nirogacestat, in Children and Adolescents With Progressive, Surgically Unresectable Desmoid Tumors
About
Protocol Description
In this study, we want to look at how well nirogacestat works and its side effects when given to children and adolescents. Nirogacestat has been used in a small number of adult patients and approximately five children with desmoid tumor. The drug has been well tolerated and in some cases stopped the tumor from growing.
COG# ARST1921
Eligibility Criteria
Inclusion Criteria
- Patients must have existing or recurrent desmoid tumor that is deemed not amenable to surgery without significant morbidity and progressed by ≥ 10% as assessed by RECIST within the 6-month period prior to study enrollment.
- Patients must have had histologic verification of the desmoid tumor.
- Patient must have received at least one prior course of systemic therapy for desmoid tumor.
- Patients must have adequate bone marrow, renal, liver and cardiac function as defined in the protocol.
- Patients must be able to swallow tablets. Tablets must not be crushed or chewed.
Exclusion Criteria
- Patients with gastrointestinal conditions that might predispose for drug intolerability or poor drug absorption
- Known active infection with hepatitis B, hepatitis C or HIV
- Pregnant or lactating female patients
See Protocol Section 3 for a complete comprehensive list of inclusion and exclusion criteria.
Primary Investigator
Dr. Glenn Edwards is the section chief of Pediatric Hematology/Oncology, as well as the Children's Oncology Group's principal investigator for Carilion Clinic. He has over 30 years of clinical experience and is board certified by the American Board of Pediatrics in Pediatrics and Pediatric Hematology/Oncology. He completed his fellowship at Walter Reed Army Medical Center and completed his internship and residency at Tripler Army Medical Center.
Contact Information
Wendy McCarty, C.C.R.P.
Clinical Research Coordinator II
