Desmoid TumorPhase 2NCT07170644
A Study of Nirogacestat in Japanese Adults With Desmoid Tumors/Aggressive Fibromatosis (DT/AF)
A Phase 2 study testing Nirogacestat oral tablet in people with Desmoid Tumor and Aggressive Fibromatosis, run by SpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, Germany at 3 sites in Japan.
Trial summary
This study is being conducted to characterize the efficacy and safety of nirogacestat in Japanese adults with progressing desmoid tumors/aggressive fibromatosis. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Desmoid Tumor, Aggressive Fibromatosis
- What is being tested?
- Nirogacestat oral tablet
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 3 sitesJapan
- Study dates
- 8 Aug 2025to 9 Oct 2029
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Participant is aged ≥18 at the time of signing the informed consent.
- Participant has histologically confirmed DT (by local pathologist prior to informed consent) that has progressed by ≥20% as measured by RECIST v1.1 within 12 months of the screening visit scan.
- Participant has:
- Treatment-naive, measurably progressing DT that is deemed not amenable to surgery without the risk of significant morbidity; OR
- Recurrent, measurably progressing DT following at least 1 line of therapy; OR
- Refractory, measurably progressing DT following at least 1 line of therapy.
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- Participant agreed to provide archival or new tumor tissue for re-confirmation of disease.
- Participant has a DT tumor where continued PD will not result in immediate significant risk to the participant.
- Participants who are receiving chronic NSAIDs as treatment for conditions other than DT must be receiving them prior to documented DT progressive disease (inclusion criterion 2) and on a stable dose for at least 28 days prior to the first dose of study treatment.
- Participant has an Eastern Cooperative Oncology Group (ECOG) performance status ≤2 at screening
- Participant has adequate organ and bone marrow function
Who cannot join
- 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 severe 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
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- 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 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 electronic patient-reported outcomes)
Trial locations and contacts
3 sites in 1 country.
Japan3 sites
National Cancer Center Hospital
Chuo Ku, Tokyo, 104-0045
Nagoya University Hospital
Nagoya, Aichi-ken, 466-8560
Osaka International Cancer Institute
Osaka, 541-8567
About this study
Official title: A Single-arm, Open-label Phase 2 Study of Nirogacestat in Adult Japanese Patients With Progressing Desmoid Tumors/Aggressive Fibromatosis (DT/AF)
This study is being conducted to characterize the efficacy and safety of nirogacestat in Japanese adults with progressing desmoid tumors/aggressive fibromatosis.
Read the detailed description
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 efficacy and safety of nirogacestat in Japanese adults with progressing desmoid tumors/aggressive fibromatosis (DT).
- Conditions
- Desmoid Tumor, Aggressive Fibromatosis
- Being tested
- Nirogacestat oral tablet
- Keywords
- Nirogacestat, PF-03084014, GSI, gamma secretase inhibitor, notch pathway, Ogsiveo
- Medical subject headings
- Desmoid Tumors
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Nirogacestat oral tabletDrug
Also called PF-03084014, Ogsiveo
Nirogacestat tablet
| Study group | Role | What they receive |
|---|---|---|
| Nirogacestat | Experimental | Nirogacestat 150 mg by mouth, twice daily Receives: Drug: Nirogacestat oral tablet |
What is being measured
The primary outcome is the main question the study is designed to answer.
Objective response rate (ORR)
Objective response rate (ORR), defined as the proportion of participants with confirmed complete response (CR) + partial response (PR) assessed by independent Central Imaging Review using RECIST v1.1 (Eisenhauer 2009).
Measured: On the first day of every 3 cycles (each cycle is 28 days) until disease progression is observed or death, assessed up to approximately 3 years.
Secondary outcomes (6)
Duration of response (DoR)
Duration of response (DoR), defined as the time from the first objective response (CR or PR) that is subsequently confirmed until the first occurrence of disease progression (radiographic or clinical) by RECIST v1.1 or death by any cause (whichever occurs first).
Measured: On the first day of every 3 cycles (each cycle is 28 days) until disease progression is observed or death, assessed up to approximately 3 years.
Time to Response (TTR)
Time to Response (TTR), defined as the time interval between the first dose of study treatment and the first date of objective response that is subsequently confirmed.
Measured: On the first day of every 3 cycles (each cycle is 28 days) until disease progression is observed or death, assessed up to approximately 3 years.
Safety endpoints will include incidence of treatment-emergent adverse events (TEAEs), changes in laboratory parameters, vital signs, physical examination findings, and ECGs.
Tolerability will be assessed according to toxicities graded by NCI CTCAE v5.0.
Measured: On the first day of every 3 cycles (each cycle is 28 days) until disease progression is observed or death, assessed up to approximately 5 years.
Progression Free Survival (PFS)
Progression Free Survival (PFS) is defined as the time from the start of study treatment until the date of assessment of progression or death by any cause. Progression will be determined radiographically using RECIST v1.1 (Eisenhauer 2009) or clinically as assessed by the investigator.
Clinical progression is defined as the onset or worsening of symptoms resulting in a global deterioration of health status causing the permanent discontinuation from study treatment and the initiation of emergent treatment (e.g., radiotherapy, surgery, or systemic therapy, including chemotherapy or tyrosine kinase inhibitors) for DT.
Measured: On the first day of every 3 cycles (each cycle is 28 days) until disease progression is observed or death, assessed up to approximately 3 years.
Symptoms and impacts will be assessed by evaluating change from baseline at Cycle 10 on the following patient reported outcomes (PROs):
- Brief Pain Inventory (BPI) short form
- European Organization for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire-Core 30 (QLQ-C30)
Measured: On the first day of every 3 cycles (each cycle is 28 days) until disease progression is observed or death, assessed up to approximately 3 years.
Characterize nirogacestat serum concentrations.
Measured: On the first day of the first cycle and of every third cycles (each cycle is 28 days) until disease progression is observed or death, assessed up to approximately 3 years.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Sep 2025
- Study start8 Aug 2025
- Primary completion28 May 2027 (estimated)
- Study completion9 Oct 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Desmoid TumorStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (actual)
Sponsor and investigators
- Lead sponsor
- SpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, Germany
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Responsible, Study director, SpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, Germany
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07170644
- Sponsor's protocol ID
- NIR-DT-202
- Other IDs
- jRCT2031250269
- Status last verified
- Sep 2026
- First submitted
- 31 Jul 2025
- Last updated
- 15 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: IPD supporting publicly available results will be evaluated for sharing.
Study at a glance
From the registry record, last updated by the sponsor on 15 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Single-arm, Open-label Phase 2 Study of Nirogacestat in Adult Japanese Patients With Progressing Desmoid Tumors/Aggressive Fibromatosis (DT/AF)
- ClinicalTrials.gov ID
- NCT07170644
- Acronym / sponsor ID
- NIR-DT-202
- Conditions
- Desmoid Tumor, Aggressive Fibromatosis
- Keywords
- Nirogacestat, PF-03084014, GSI, gamma secretase inhibitor, notch pathway, Ogsiveo
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 20 participants (actual)
- Lead sponsor
- SpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, GermanyIndustry (pharma, biotech, devices)
- Study start
- 8 Aug 2025 (actual)
- Primary completion
- 28 May 2027 (estimated)
- Study completion
- 9 Oct 2029 (estimated)
- First posted
- 12 Sep 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study is being conducted to characterize the efficacy and safety of nirogacestat in Japanese adults with progressing desmoid tumors/aggressive fibromatosis.
Detailed description
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 efficacy and safety of nirogacestat in Japanese adults with progressing desmoid tumors/aggressive fibromatosis (DT).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Nirogacestat | Experimental | Drug: Nirogacestat oral tabletNirogacestat 150 mg by mouth, twice daily |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (11 listed)
- Participant is aged ≥18 at the time of signing the informed consent.
- Participant has histologically confirmed DT (by local pathologist prior to informed consent) that has progressed by ≥20% as measured by RECIST v1.1 within 12 months of the screening visit scan.
- Participant has:
- Participant agreed to provide archival or new tumor tissue for re-confirmation of disease.
- Participant has a DT tumor where continued PD will not result in immediate significant risk to the participant.
Key exclusion criteria (9 listed)
- 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 severe hepatic impairment
- Participant previously received or is currently receiving gamma secretase inhibitors or anti-Notch antibody therapy
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Nirogacestat oral tabletAlso: PF-03084014, OgsiveoNirogacestat tablet | Drug | Nirogacestat |
Primary outcome
Objective response rate (ORR)
Time frame: On the first day of every 3 cycles (each cycle is 28 days) until disease progression is observed or death, assessed up to approximately 3 years.
Objective response rate (ORR), defined as the proportion of participants with confirmed complete response (CR) + partial response (PR) assessed by independent Central Imaging Review using RECIST v1.1 (Eisenhauer 2009).
6 secondary outcomes
Duration of response (DoR)
Time frame: On the first day of every 3 cycles (each cycle is 28 days) until disease progression is observed or death, assessed up to approximately 3 years.
Time to Response (TTR)
Time frame: On the first day of every 3 cycles (each cycle is 28 days) until disease progression is observed or death, assessed up to approximately 3 years.
Safety endpoints will include incidence of treatment-emergent adverse events (TEAEs), changes in laboratory parameters, vital signs, physical examination findings, and ECGs.
Time frame: On the first day of every 3 cycles (each cycle is 28 days) until disease progression is observed or death, assessed up to approximately 5 years.
Progression Free Survival (PFS)
Time frame: On the first day of every 3 cycles (each cycle is 28 days) until disease progression is observed or death, assessed up to approximately 3 years.
Symptoms and impacts will be assessed by evaluating change from baseline at Cycle 10 on the following patient reported outcomes (PROs):
Time frame: On the first day of every 3 cycles (each cycle is 28 days) until disease progression is observed or death, assessed up to approximately 3 years.
Characterize nirogacestat serum concentrations.
Time frame: On the first day of the first cycle and of every third cycles (each cycle is 28 days) until disease progression is observed or death, assessed up to approximately 3 years.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Sep 2026.
- Trial Awareness and Transparency websiteLink provided by the sponsor.
- Medical Information Location Map - Med Info ContactsLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. IPD supporting publicly available results will be evaluated for sharing.
Related trials for Desmoid Tumor
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FOG-001 in Locally Advanced or Metastatic Solid Tumors
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A Study of a New Drug, Nirogacestat, for Treating Desmoid Tumors That Cannot be Removed by Surgery
Desmoid Fibromatosis, Recurrent Desmoid Fibromatosis
Enrolling by invitationObservational study
Patient-reported Outcomes Following Percutaneous Ablation of Desmoid Tumors
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Medical Responsible study director
SpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, Germany
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Nirogacestat oral tablet in people with Desmoid Tumor and Aggressive Fibromatosis, run by SpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, Germany at 3 sites in Japan.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 11 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 15 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in Japan. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Objective response rate (ORR) (time frame: On the first day of every 3 cycles (each cycle is 28 days) until disease progression is observed or death, assessed up to approximately 3 years.). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 Sep 2026.