A Single-arm, Open-label Phase 4 Study of Nirogacestat in Adult Premenopausal Females with Desmoid Tumors/Aggressive Fibromatosis (DT/AF)

Condition studied

DESMOID TUMOURS/AGGRESSIVE FIBROMATOSIS (DT/AF)

Investigational medicinal product(s)

Eligibility

Primary endpoint

Ovarian function recovery rate of OT TEAEs. Ovarian function recovery is defined as achieving the resumption of ≥2 consecutive menstrual periods and an follicle stimulating hormone (FSH) level <30 mIU/mL with concomitant estradiol <80 pg/mL, OR resumption of ≥2 consecutive menstrual periods and anti-mullerian hormone (AMH) level within normal range adjusted for age and pretreatment baseline, OR a positive serum beta-human chorionic gonadotropin (β-HCG) pregnancy test.

Endpoint detail

1.Incidence of OT TEAEs. OT is defined as new onset amenorrhea lasting ≥3 consecutive menstrual periods, FSH level ≥30 mIU/mL, AND a negative β-HCG pregnancy test., 2.Time to ovarian function recovery in participants with a TEAE of OT., 3.Safety endpoints will include incidence of TEAEs, changes in laboratory parameters including hormones, vital signs, and physical examination findings. Tolerability will be assessed according to toxicities graded by National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) v5.0.