Status:
RECRUITING
A Study of Nirogacestat in Japanese Adults With Desmoid Tumors/Aggressive Fibromatosis (DT/AF)
Lead Sponsor:
SpringWorks Therapeutics, Inc.
Conditions:
Desmoid Tumor
Aggressive Fibromatosis
Eligibility:
All Genders
18+ years
Phase:
PHASE2
Brief Summary
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 ...
Eligibility Criteria
Inclusion
- Key
- 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.
- 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
- Key
Exclusion
- 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
- 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)
Key Trial Info
Start Date :
August 8 2025
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
December 31 2030
Estimated Enrollment :
18 Patients enrolled
Trial Details
Trial ID
NCT07170644
Start Date
August 8 2025
End Date
December 31 2030
Last Update
December 30 2025
Active Locations (3)
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1
Nagoya University Hospital
Nagoya, Aichi-ken, Japan, 466-8560
2
National Cancer Center Hospital
Chuo Ku, Tokyo, Japan, 104-0045
3
Osaka Prefectural Hospital Organization Osaka International Cancer Institute
Osaka, Japan, 541-8567