A Phase 4 interventional study of Nirogacestat in Desmoid Tumor and Aggressive Fibromatosis, sponsored by SpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, Germany. Recruiting at 23 sites in 6 countries. Open to female participants aged 18 Years to 40 Years. Per ClinicalTrials.gov, last updated 2026-11-09.
Sponsored by SpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, Germany (part of Merck KGaA, Darmstadt, Germany) · Phase 4, Interventional, and Treatment
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This study is being conducted to study how nirogacestat may affect the ovarian function of adult premenopausal women with progressing desmoid tumors/aggressive fibromatosis.
Desmoid tumors, also referred to as aggressive fibromatosis, are rare, locally invasive, slow growing soft tissue tumors. Although considered benign because of their inability to metastasize, desmoid tumors can cause significant morbidity and occasionally mortality in patients.
Nirogacestat is a tumor inhibitor that works by slowing or stopping the growth of tumor cells. Nirogacestat is a tablet taken by mouth and has been approved in the USA for adult patients with progressing desmoid tumors who require systemic treatment.
This is an open-label study to characterize the incidence and ovarian function recovery rates of ovarian toxicity (OT) events and to evaluate the efficacy, safety, and tolerability of nirogacestat in postpubertal and premenopausal females with desmoid tumors (DT).
71 studies on the registry are indexed under Desmoid Tumors; 21 are open to participants now.
This study's planned enrollment of 50 is above the median of 44 across 47 interventional studies indexed under Desmoid Tumors.
Browse Desmoid Tumors studies →SpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, Germany is the lead sponsor of 14 studies on the registry; 6 are open to participants now.
Of its 5 completed or terminated interventional studies of FDA-regulated products, 3 (60%) have results posted.
First submitted after SpringWorks Therapeutics, Inc. became part of Merck KGaA, Darmstadt, Germany.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
The diagnosis was confirmed by looking at a sample under a microscope: tissue from a biopsy or surgery (histology), or cells from a fluid or needle sample (cytology).
Your pathology report is the document that shows this.
A scale of how much an illness limits everyday activity. For people being assessed to join a study, scores run from 0 (fully active) to 4; lower is more active.
Studies most often ask for 0–1 or 0–2. Your doctor gives the score; the WHO scale uses the same steps. The full scale also has 5, which records death.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
The New York Heart Association’s four classes of how much heart failure limits physical activity.
Studies set their own limit — many outside heart failure leave out class III–IV, while heart failure studies may ask for a particular class. Check the class this study names.
A “mini-stroke”: temporary stroke-like symptoms caused by reduced blood flow to part of the brain, spinal cord or eye, without signs of a new stroke on a scan.
Stroke symptoms need emergency care at once, even if they go away.
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Cancer that has spread from where it began to other parts of the body. “Metastatic” cancer is cancer that has spread this way.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
CYP3A is a group of enzymes, mainly CYP3A4, in the liver and gut that break down many medicines. Some medicines, supplements and foods (grapefruit, St John’s wort) slow them down or speed them up, which changes how much of a study drug stays in the body.
Studies list them so they can be avoided or switched; bring a list of everything you take, including supplements.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Histologically or cytologically confirmed
The diagnosis was confirmed by looking at a sample under a microscope: tissue from a biopsy or surgery (histology), or cells from a fluid or needle sample (cytology).
Your pathology report is the document that shows this.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ECOG performance status
A scale of how much an illness limits everyday activity. For people being assessed to join a study, scores run from 0 (fully active) to 4; lower is more active.
Studies most often ask for 0–1 or 0–2. Your doctor gives the score; the WHO scale uses the same steps. The full scale also has 5, which records death.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
NYHA class (heart failure)
The New York Heart Association’s four classes of how much heart failure limits physical activity.
Studies set their own limit — many outside heart failure leave out class III–IV, while heart failure studies may ask for a particular class. Check the class this study names.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
TIA (transient ischaemic attack)
A “mini-stroke”: temporary stroke-like symptoms caused by reduced blood flow to part of the brain, spinal cord or eye, without signs of a new stroke on a scan.
Stroke symptoms need emergency care at once, even if they go away.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cancer (malignancy)
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Metastases
Cancer that has spread from where it began to other parts of the body. “Metastatic” cancer is cancer that has spread this way.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
CYP3A (CYP3A4) inhibitors and inducers
CYP3A is a group of enzymes, mainly CYP3A4, in the liver and gut that break down many medicines. Some medicines, supplements and foods (grapefruit, St John’s wort) slow them down or speed them up, which changes how much of a study drug stays in the body.
Studies list them so they can be avoided or switched; bring a list of everything you take, including supplements.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Nirogacestat 150 mg by mouth, twice daily
Drug: Nirogacestat
Nirogacestat oral tablet
Also known as: PF-03084014, Ogsiveo
Ovarian function recovery rate of ovarian toxicity (OT) treatment-emergent adverse events (TEAEs)
Ovarian function recovery is defined as achieving the resumption of ≥2 consecutive menstrual periods and an FSH level \<30 mIU/mL with concomitant estradiol \<80 pg/mL OR resumption of ≥2 consecutive menstrual periods and AMH level within normal range adjusted for age and pretreatment baseline OR a positive serum β-HCG pregnancy test.
Time frame: Up to 24 cycles (each cycle is 28 days) of treatment and up to 2 years in the Clinical Follow-up Period
Incidence of OT TEAEs
OT is any new onset amenorrhea lasting ≥3 consecutive menstrual periods, FSH level ≥30 mIU/mL and a negative β-HCG pregnancy test.
Time frame: Up to 24 cycles (each cycle is 28 days) of treatment and up to 2 years in the Clinical Follow-up Period
Time to ovarian function recovery in participants with a TEAE of OT
Time to ovarian function recovery is evaluated in participants who had a TEAE of OT and is defined as the time it takes to resolve.
Time frame: Up to 24 cycles (each cycle is 28 days) of treatment and up to 2 years in the Clinical Follow-up Period
The incidence of adverse events (AEs) according to toxicities graded by National Cancer Institute (NCI) Common Technology Criteria for Adverse Events (CTCAE) Version 5
Time frame: Up to 24 cycles (each cycle is 28 days) of treatment and up to 2 years in the Clinical Follow-up Period
Objective Response Rate (ORR)
Proportion of participants with Complete Response (CR) and Partial Response (PR) using Response Evaluation Criteria in Solid Tumors (RECIST) v1.1 criteria
Time frame: First day of every 3 cycles (each cycle is 28 days) for up to 24 cycles of treatment and up to 1 year in the Clinical Follow-up Period
Duration of Response (DoR) for participants whose best response is CR or PR
DoR is defined as the duration from the time of first assessment of CR or PR response (per RECIST v1.1) to the first date of disease progression or death (whichever comes first).
Time frame: First day of every 3 cycles (each cycle is 28 days) until disease progression is observed or death, whichever comes first, up to 24 cycles of treatment and up to 1 year in the Clinical Follow-up Period
Disease Control Rate (DCR) for participants whose best response is CR, PR or Stable Disease (SD)
DCR is defined as the proportion of participants who have a best response of CR, PR, or SD.
Time frame: First day of every 3 cycles (each cycle is 28 days) for up to 24 cycles of treatment and up to 1 year in the Clinical Follow-up Period
Duration of Disease Control (DoDC) for participants whose best response is CR, PR or SD
DoDC is defined as the duration from the start of study treatment until first date of disease progression or death (whichever comes first).
Time frame: First day of every 3 cycles (each cycle is 28 days) until disease progression is observed or death, whichever comes first, up to 24 cycles of treatment and up to 1 year in the Clinical Follow-up Period
Progression Free Survival (PFS)
PFS is defined at the start of study treatment until the date of assessment of progression or death by any cause. Progression will be determined radiographically using RESIST v1.1 performed by a local radiologist.
Time frame: First day of every 3 cycles (each cycle is 28 days) until disease progression is observed or death, whichever comes first, up to 24 cycles of treatment and up to 1 year in the Clinical Follow-up Period
Changes in tumor volume from baseline assessed by MRI volumetric analysis performed locally
Time frame: First day of every 3 cycles (each cycle is 28 days) up to 24 cycles and up to 1 year in the Clinical Follow-up Period
Change in T2 hyperintensity baseline assessed by MRI analysis performed locally
Time frame: First day of every 3 cycles (each cycle is 28 days) up to 24 cycles and up to 1 year in the Clinical Follow-up Period
Characterization of serum nirogacestat concentrations assessed in sparse pharmacokinetic (PK) sampling (pre-dose and 1-hour post dose at Cycle 1 Day 1 only) and trough PK sampling (pre-dose at treatment period visits after Cycle 1 Day 1
Time frame: First day of every 3 cycles (each cycle is 28 days) up to 24 cycles of treatment
Pain severity using Brief Pain Index - Short Form (BPI-SF), question #3
BPI question #3 will measure worst pain in the last 24 hours using an 11-point numeric scale from 0 (no pain) to 10 (pain as bad as you can imagine)
Time frame: From 7 days prior to date of first dose of treatment through end of Cycle 2 (each cycle is 28 days), assessed up to 9 weeks
Patient experience of desmoid tumors and nirogacestat
Two (2) optional 60-minute qualitative interviews using open-ended exploratory questions to elicit spontaneous responses will be transcribed and analyzed in an inductive/deductive method to identify themes in the data
Time frame: One interview will occur following treatment completion (on average approximately 2 years after study entry) and a second interview will occur at study completion (on average approximately 3 years after study entry)
Plan to share: Yes — IPD supporting publicly available results will be evaluated for sharing.
Supporting information: Study protocol, Statistical analysis plan, Clinical study report, Analytic code
Time frame: IPD from completed trials will be publicly available within 6 months after all of the following events: Approval of a product/new indication by major authorities (FDA, EMA, PMDA if requested) with no pending submissions Public results via primary manuscript or trial registry disclosure Legal authority to share data Privacy protections are in place If approval is not sought or development is globally discontinued, data will be publicly available within 18 months after global trial completion. Further information on how to request data can be found on our website bit.ly/IPD21
Access criteria: Qualified researchers may propose access to IPD from sponsored trials via https://vivli.org/members/ourmembers/
Data sharing details ↗ · vivli.org
Started Sep 2025 (actual). Primary completion is estimated for Feb 2031, 65 months after the start.
How these figures are worked out, and which studies are left out of them.
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
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Studies of the drug tested here: Nirogacestat
SpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, Germany →