What is nirogacestat studying for desmoid tumors?

Nirogacestat is being studied to see how it affects ovarian function and to evaluate its safety and tolerability in adult premenopausal women with progressing desmoid tumors. The research aims to understand the drug's impact on ovarian toxicity while monitoring tumor response. This study is sponsored by SpringWorks Therapeutics, Inc.

Who may be eligible for this desmoid tumor clinical trial?

Adult females aged 18 to 40 who are premenopausal with regular menstrual cycles and have a confirmed diagnosis of progressing desmoid tumor or aggressive fibromatosis may be eligible. Participants should not be using hormonal contraception or certain other treatments. Please review eligibility criteria for more details.

Is there any cost to participate in this desmoid tumor clinical trial?

Study-related care, including the study drug and all trial visits, is provided at no cost. However, personal travel expenses and other costs are not guaranteed to be covered and may vary depending on individual circumstances.

Actively Recruiting

Phase 4

Age: 18Years - 40Years

FEMALE

ID07176689

Study of Nirogacestat Treatment in Adult Premenopausal Women with Desmoid Tumors or Aggressive Fibromatosis

Led by SpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, Germany · Updated on 2026-07-17

Participants Needed

Research Sites

N/A

Total Duration

AI-Summary

What this Trial Is About

Researchers are evaluating how nirogacestat may affect ovarian function in adult premenopausal women who have progressing desmoid tumors or aggressive fibromatosis. Desmoid tumors are rare, slow-growing soft tissue tumors that do not spread to other parts of the body but can cause serious health problems. This open-label phase 4 study aims to understand the incidence and recovery rate of ovarian toxicity caused by nirogacestat while also assessing the drugs safety and tolerability in this population. Participants will take nirogacestat tablets orally at a dose of 150 mg twice daily. The study includes up to 24 treatment cycles, each lasting 28 days, followed by a clinical follow-up period of up to 2 years. During this time, researchers will monitor ovarian function recovery and other health effects related to ovarian toxicity and drug safety. Throughout the study, participants will undergo regular evaluations including blood tests to measure hormone levels, completion of menstrual diaries, and electronic patient-reported outcomes. Researchers will track ovarian function recovery rates, the occurrence and timing of ovarian toxicity events, and any adverse events using standardized criteria. The total participation time may extend up to 2 years after treatment for thorough monitoring and safety assessments.

CONDITIONS

Brief Title

Nirogacestat in Premenopausal Females With Desmoid Tumor/Aggressive Fibromatosis (DT/AF)

Who Can Participate

Age: 18Years - 40Years

FEMALE

Eligibility Criteria

You may qualify if you...

Female participants aged 18 to 40 years, postpubertal and premenopausal at baseline
Premenopausal defined by estradiol >30 pg/mL, FSH <40 IU/L, and regular menstrual cycles (21 to 35 days) for at least 3 cycles before consent
Use of one highly effective non-hormonal contraceptive method
Negative pregnancy test before first dose and not breastfeeding
Agreement to not harvest or donate eggs for at least 90 days before and during the study
Histologically confirmed desmoid tumor/aggressive fibromatosis with symptomatic or progressive disease requiring systemic treatment
Eastern Cooperative Oncology Group (ECOG) performance status ≤2 at screening
Adequate organ and bone marrow function

You will not qualify if you...

Known malabsorption syndrome or gastrointestinal conditions that impair nirogacestat absorption
Significant cardiac disease or events within 6 months before consent (e.g., severe heart failure, myocardial infarction)
History of lymphoma, leukemia, or malignancy within 5 years except certain treated skin or bladder cancers without metastasis
Known hepatic impairment
Prior or current treatment with gamma secretase inhibitors or anti-Notch antibody therapy
Use of tyrosine kinase inhibitors or investigational treatment for desmoid tumor within 28 days before first dose
Use or anticipated use of strong/moderate CYP3A4 inhibitors or inducers within 14 days before first dose
History of polycystic ovary syndrome, hypothalamic amenorrhea, severe endometriosis of ovaries, or conditions linked to early menopause
Use of hormonal contraception or ovarian suppression within 90 days before first dose
Heavy tobacco smoking history (≥20 pack years) or current smoking >1 pack per day
Severe acute or chronic medical or psychiatric conditions within 1 year before consent
Inability to comply with study procedures including menstrual diary and hormone blood draws timed to menstrual cycle

Research Team

US Medical Information

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Study of Nirogacestat Treatment in Adult Premenopausal Women with Desmoid Tumors or Aggressive Fibromatosis

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AI-Summary

What this Trial Is About

Brief Title

Who Can Participate

Eligibility Criteria

Research Team

Not the Right Trial for You?

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Who Can Participate

Eligibility Criteria