This study is being conducted to study how nirogacestat may affect the ovarian function of adult premenopausal women with progressing desmoid tumors/aggressive fibromatosis.
Desmoid tumors, also referred to as aggressive fibromatosis, are rare, locally invasive, slow growing soft tissue tumors. Although considered benign because of their inability to metastasize, desmoid tumors can cause significant morbidity and occasionally mortality in patients. Nirogacestat is a tumor inhibitor that works by slowing or stopping the growth of tumor cells. Nirogacestat is a tablet taken by mouth and has been approved in the USA for adult patients with progressing desmoid tumors who require systemic treatment. This is an open-label study to characterize the incidence and ovarian function recovery rates of ovarian toxicity (OT) events and to evaluate the efficacy, safety, and tolerability of nirogacestat in postpubertal and premenopausal females with desmoid tumors (DT).
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
50
Nirogacestat oral tablet
Cliniques Universitaires Saint-Luc (CUSL)
Brussels, Belgium
Ovarian function recovery rate of ovarian toxicity (OT) treatment-emergent adverse events (TEAEs)
Ovarian function recovery is defined as achieving the resumption of ≥2 consecutive menstrual periods and an FSH level \<30 mIU/mL with concomitant estradiol \<80 pg/mL OR resumption of ≥2 consecutive menstrual periods and AMH level within normal range adjusted for age and pretreatment baseline OR a positive serum β-HCG pregnancy test.
Time frame: Up to 24 cycles (each cycle is 28 days) of treatment and up to 2 years in the Clinical Follow-up Period
Incidence of OT TEAEs
OT is any new onset amenorrhea lasting ≥3 consecutive menstrual periods, FSH level ≥30 mIU/mL and a negative β-HCG pregnancy test.
Time frame: Up to 24 cycles (each cycle is 28 days) of treatment and up to 2 years in the Clinical Follow-up Period
Time to ovarian function recovery in participants with a TEAE of OT
Time to ovarian function recovery is evaluated in participants who had a TEAE of OT and is defined as the time it takes to resolve.
Time frame: Up to 24 cycles (each cycle is 28 days) of treatment and up to 2 years in the Clinical Follow-up Period
The incidence of adverse events (AEs) according to toxicities graded by National Cancer Institute (NCI) Common Technology Criteria for Adverse Events (CTCAE) Version 5
Time frame: Up to 24 cycles (each cycle is 28 days) of treatment and up to 2 years in the Clinical Follow-up Period
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