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 study about?

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.

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 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 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 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 Study?

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 Study?

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

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country	Status
Hospital Universitario Y Politecnico La Fe	Valencia	Spain
Universitaetsklinikum Heidelberg AöR	Heidelberg	Germany

Other Sites

Site Name	City	Country
Istituto Ortopedico Rizzoli	Bologna	Italy
IRCCS Istituto Nazionale Tumori Fondazione Pascale	Naples	Italy
Hospital General Universitario Gregorio Maranon	Madrid	Spain
Hospital Universitario 12 De Octubre	Madrid	Spain
Universita’ Campus Bio-medico Di Roma	Rome	Italy
Istituto Di Candiolo Fondazione Del Piemonte Per Loncologia IRCCS	Candiolo	Italy
Hospital Universitario Fundacion Jimenez Diaz	Madrid	Spain
Netherlands Cancer Institute	Amsterdam	The Netherlands
Fondazione I.R.C.C.S. Istituto Neurologico Besta	Milan	Italy
Cfvrlmwgt Ughrartjdcgwty Sevgehkzo	Woluwe-Saint-Lambert	Belgium
Lbfpe Upjyieoyulrj Mvieqmn Cwvuqwc (woajy	Leiden	The Netherlands
Ixubhsdh Cmhblu Dzceybhqxerhuhqei	L'hospitalet De Llobregat	Spain
Ichnotnv Reneosvuf Ppw Lr Szqlqt Doi Txruef Drzy Amfuljv Ixkc Sqnfjq	Meldola	Italy
Eclfxxe Upqkuyowztst Mvpabfo Cczgsjy Rghkmbuwi (qhpxgnx Mqz	Rotterdam	The Netherlands
Azfhcjw Umfrg Sfmsaccfq Lpwihq Dv Bmtuczb	Bologna	Italy
Hvwxzyum Dp Ll Sgqxu Cbti I Slek Pve	Barcelona	Spain
Ffxyqckdn Pext Ly Imqxzzstvoxzg Bzpvyyvrd Dgv Hgdcsvpf Usanqgvoxuotu Ly Pfp	Madrid	Spain

Trial status

Country	Status	Recruitment Start
Belgium	Recruiting	30.09.2025
Germany	Recruiting	30.09.2025
Italy	Recruiting	30.09.2025
Spain	Recruiting	30.09.2025
The Netherlands	Recruiting	30.09.2025

Trial locations

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.

Investigated diseases:

Desmoid tumour

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 ID:

2024-515215-21-00

Protocol code:

NIR-DT-401

Trial Phase:

Therapeutic confirmatory (Phase III)

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

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?