Evaluation of the Response and Non-response of Nirogacestat in Desmoid Tumors- Clinical Study
M.D. Anderson Cancer Center
Summary
To learn about the safety and effects of an investigational drug called nirogacestat when given to participants with a desmoid tumor/aggressive fibromatosis
Description
Primary Objectives: • To identify biomarkers associated with response and non-response to nirogacestat in participants with desmoid tumors (DT). Secondary Objectives: * To assess the 12-month progression-free survival (PFS of participants with DT who receive nirogacestat at 150 mg or 100 mg BID. * To assess MRI volumetric and functional parameters associated with response and non-response to nirogacestat in participants with DT. * To evaluate the histopathological changes in DT biopsy specimens. * To evaluate the safety and tolerability of nirogacestat in participants with DT. Exploratory…
Eligibility
- Age range
- 18+ years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: This study will enroll approximately 40 participants diagnosed with DT. Participants must meet the following eligibility criteria to be enrolled: 1. Age ≥ 18 years. Individuals younger than 18 years old are excluded. More than 99% of the population evaluated at MDACC with a diagnosis of DT is older than 18. Sixty-three percent of the population with a diagnosis of DT evaluated at MDACC are 10 - 54 years old. The remaining 37 percent are older than 54 years old. Additionally, most of the robust data related to dosing or adverse event data currently available on the use of…
Interventions
- DrugNirogacestat
Given by PO
Location
- M D Anderson Cancer CenterHouston, Texas