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Study of Nirogacestat in adult premenopausal women with desmoid tumors and aggressive fibromatosis to evaluate ovarian function recovery

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This study focuses on desmoid tumors/aggressive fibromatosis (DT/AF), which are rare growths that develop in soft tissues. The study will test a medication called nirogacestat (also known as PF-03084014) that comes in tablet form and is taken by mouth. This medicine will be given to adult women before menopause who have these tumors.

The main purpose of this research is to understand how the treatment affects ovarian function in women. The study will look at whether and how quickly normal menstrual cycles and hormone levels return after treatment. Participants will take the medication daily for up to 24 months.

During the study, doctors will monitor participants' overall health, including regular blood tests to check hormone levels and track menstrual cycles. The medication dose can be up to 300 milligrams per day. This is an open-label study, which means all participants will receive the active medication, and there is no placebo group.

The research process

The trial runs in 4 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Initial assessment

    Your eligibility for the study will be evaluated based on specific criteria, including being a female aged 18-40 years and having confirmed desmoid tumor/aggressive fibromatosis (DT/AF) that requires treatment.

    A physical examination and laboratory tests will be performed to assess your organ function and overall health status.

    Your menstrual status and hormone levels will be checked to confirm premenopausal status.

  2. Step 2

    Treatment phase

    You will receive Nirogacestat tablets for oral use.

    During treatment, regular assessments will monitor your menstrual cycle and hormone levels, including FSH (follicle stimulating hormone), estradiol, and AMH (anti-mullerian hormone).

    Your overall health status and any side effects will be monitored using standardized criteria.

  3. Step 3

    Monitoring phase

    Regular check-ups will track changes in your menstrual cycle.

    Blood tests will measure hormone levels to assess ovarian function.

    Any changes in your health status or new symptoms will be documented and evaluated.

  4. Step 4

    Follow-up period

    After completing treatment, you will continue to be monitored for ovarian function recovery.

    Recovery is confirmed by either the return of regular menstrual periods (at least 2 consecutive cycles) with specific hormone levels, or a positive pregnancy test.

    The study will continue until September 2028.

Who can join the trial?

8 criteria

  • Must be a female between 18 and 40 years old who has gone through puberty and still has regular menstrual cycles at the start of the study
  • Must not be pregnant or breastfeeding and must either: - Practice complete abstinence or use highly effective birth control - Not have donated eggs in the past 90 days and agree not to donate eggs during the study
  • Must have a confirmed diagnosis of desmoid tumor/aggressive fibromatosis that is causing symptoms or getting worse, requiring treatment
  • If previously treated with chemotherapy or radiation, must still have regular menstrual cycles at least 2 weeks after completing treatment. Cannot participate if previously received treatments that could harm the ovaries or radiation to the pelvic area
  • Must have good daily physical function (rated as ECOG score of 2 or less, meaning able to care for self but may be unable to work)
  • Must have properly functioning organs and adequate bone marrow function (related to blood cell production)
  • Must be able to swallow tablets and have no digestive conditions that would affect how medicine is absorbed
  • Must be able to understand and sign the consent form and follow all study requirements

Who cannot join the trial?

10 criteria

  • Patients who are male (only females can participate)
  • Patients who are under 18 years old
  • Patients who are not diagnosed with desmoid tumors or aggressive fibromatosis (DT/AF)
  • Patients who are unable to provide informed consent
  • Patients who do not have ovarian function (due to previous surgery or other medical conditions)
  • Patients who are currently pregnant or breastfeeding
  • Patients with known allergies or hypersensitivity to the study medications
  • Patients with serious medical conditions that could interfere with study participation
  • Patients who are participating in other clinical trials
  • Patients with psychiatric disorders that could affect their ability to comply with the study requirements
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Investigated drugs

Nirogacestat is a medication that works by blocking certain proteins involved in cell growth. It is being studied in adult premenopausal women who have desmoid tumors or aggressive fibromatosis, which are rare types of soft tissue tumors. The medication is taken orally and is designed to help control tumor growth. This study specifically looks at how the medication might affect ovarian function in women of childbearing age.

What is already known about the treatment

Nirogacestat – A novel gamma secretase inhibitor administered orally that is being investigated for the treatment of desmoid tumors and aggressive fibromatosis (DT/AF). This medication works by blocking the gamma secretase enzyme, which plays a crucial role in the Notch signaling pathway involved in tumor growth. It belongs to the class of targeted therapy medications and represents a promising treatment option for patients with these rare soft tissue tumors, particularly in premenopausal women where treatment options have been limited. The drug is currently being evaluated in Phase 4 clinical trials to better understand its effects on ovarian function and potential reproductive implications.

Investigated diseases

Desmoid Tumors/Aggressive Fibromatosis (DT/AF) – A rare condition characterized by the growth of non-cancerous tumors in the soft tissues of the body. These tumors develop from connective tissue cells and can occur in various parts of the body, including the abdomen, shoulders, arms, and legs. While not malignant, these tumors can grow aggressively and invade surrounding tissues and organs. DT/AF tumors typically develop slowly but can be unpredictable in their growth pattern. The condition can occur at any age but is more common in young adults, particularly women of childbearing age.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2024-515215-21-00Protocol codeNIR-DT-401Estimated enrolment50 patientsSponsorSpringworks Therapeutics Inc.

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