Nirogacestat for premenopausal women with desmoid tumors

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This open-label Phase 4 trial gives oral nirogacestat to postpubertal, premenopausal women with symptomatic or progressive desmoid tumors to measure ovarian toxicity events and the rates of ovarian function recovery. Participants must meet baseline hormone and menstrual criteria and have adequate organ function and ECOG ≤2. The study will also collect safety, tolerability, and efficacy data while monitoring reproductive hormones and menstrual patterns over time. Sites are located in Europe and the protocol includes regular in-person visits and laboratory assessments.

Why it matters

Who can join: the full rules

Inclusion Criteria:

* Participant is female, postpubertal aged ≥18 and ≤40 years of age at the time of signing the informed consent and premenopausal at baseline. Premenopausal is defined as meeting all of the following: Estradiol \>30 pg/mL. Follicle-stimulating hormone (FSH) \<40 IU/L. Regular menses (e.g., menstrual cycle length of 21 to 35 days) for at least 3 menstrual cycles prior to signing informed consent
* Participant uses 1 highly effective non-hormonal contraceptive method, has a negative pregnancy test prior to first dose of study treatment), is not breastfeeding, agrees to not harvest or donate eggs for at least 90 days prior to and during the study
* Participant has histologically confirmed DT/AF with symptomatic or progressive disease requiring systemic treatment
* Participant has an Eastern Cooperative Oncology Group (ECOG) performance status ≤2 at screening
* Participant has adequate organ and bone marrow function.

Exclusion Criteria:

* Participant has known malabsorption syndrome or preexisting gastrointestinal conditions that may impair absorption of nirogacestat
* Participant has experienced any of the following within 6 months of signing informed consent: clinically significant cardiac disease (New York Heart Association Class III or IV), myocardial infarction, severe/unstable angina, coronary/peripheral artery bypass graft, symptomatic congestive heart failure, cerebrovascular accident, transient ischemic attack, or symptomatic pulmonary embolism.
* Participant has had lymphoma, leukemia, or any malignancy within the past 5 years at the time of informed consent, except for any locally recurring cancer that has been treated curatively (e.g., resected basal or squamous cell skin cancer, superficial bladder cancer, carcinoma in situ of the cervix or breast), with no evidence of metastatic disease for 3 years at the time of informed consent.
* Participant has known hepatic impairment
* Participant previously received or is currently receiving gamma secretase inhibitors or anti-Notch antibody therapy
* Participant is currently using any treatment for DT/AF including tyrosine kinase inhibitors (TKIs) or any investigational treatment 28 days (or 5 half-lives, whichever is longer) prior to the first dose of study treatment
* Participant is currently using or anticipates using food or drugs that are known strong/moderate cytochrome P450 (CYP) 3A4 inhibitors, or strong CYP3A inducers within 14 days prior to the first dose of study treatment.
* Participant has a history of polycystic ovary syndrome, hypothalamic amenorrhea, severe endometriosis involving ovaries, family history of primary ovarian insufficiency, any chromosomal abnormality, mutation, gene variant or medical condition associated with early/premature menopause, including a history of OT while on a TKI
* Participant is currently using or has used hormonal contraception or ovarian suppression within 90 days prior to first dose of study treatment
* Participant has a history of heavy tobacco smoking (≥20 pack years) or is a current smoker (\>1 pack per day)
* Participant has experienced other severe acute or chronic medical or psychiatric conditions within 1 year of signing informed consent.
* Participant is unable to comply with study related procedures (including, but not limited to, the completion of a menstrual diary and electronic patient-reported outcomes and ability to return to clinic for hormone level blood draws timed to the menstrual cycle (days 1-5)

Where it's running

• Cliniques Universitaires Saint-Luc (CUSL)
  Brussels, Belgium Recruiting
• Universitaetsmedizin Mannheim
  Mannheim, Germany Recruiting
• IRCCS Istituto Romagnolo per lo Studio dei Tumori "Dino Amadori" - IRST S.r.l.
  Meldola, Forli-Cesena, Italy Recruiting
• La Fondazione e l'Istituto di Candiolo
  Candiolo, Torino, Italy Recruiting
• IRCCS Azienda Ospedaliero-Universitaria di Bologna - Policlinico di Sant Orsola
  Bologna, Italy Recruiting
• IRCCS Istituto Ortopedico Rizzoli di Bologna
  Bologna, Italy Recruiting

Study contacts

• Study coordinator: US Medical Information
• Email: eMediUSA@emdserono.com
• Phone: 888-275-7376

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Treatment for adults with desmoid tumors

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• Comparing cryoablation and medical therapy for desmoid tumors after watchful waiting

  This study is testing whether cryoablation works better than medical therapy for people with desmoid tumors that have gotten worse after watching them for a while.

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• Treatment of Desmoid Tumors with Nirogacestat and Cryoablation

  This study is testing if a combination of a new oral medication called nirogacestat and a freezing treatment can help people with growing or painful desmoid tumors.

  Recruiting Phase 2 Stanford University · Palo Alto, California · NCT05949099

• Comparing in-person and remote monitoring for cancer follow-up

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• Using chemoembolization to treat desmoid fibromatosis

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• Comparing imaging techniques for predicting desmoid tumor progression after cryoablation

  This study is testing if a new imaging method can better predict how desmoid tumors change after cryoablation treatment compared to standard methods.

  Recruiting Istituto Ortopedico Rizzoli · Bologna · NCT06224283

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.