New pill shows promise for rare tumor in japanese patients
NCT ID NCT07170644
First seen Jun 27, 2026 · Last updated Sep 16, 2026 · Updated 4 times
Summary
This phase 2 study tests the drug nirogacestat in 20 Japanese adults with desmoid tumors that are growing. The goal is to see if the drug can shrink tumors or stop them from getting worse. Participants take nirogacestat as an oral tablet, and researchers will monitor tumor response and side effects.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- nirogacestat (oral tablet)
- What this could lead to
- If successful, nirogacestat could offer a new treatment option for Japanese patients with desmoid tumors, potentially shrinking tumors and delaying progression without surgery.
- What could go wrong
- This is a small, early-phase study with only 20 participants, so results may not apply to all patients. The drug may cause side effects and may not work for everyone.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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20 people
The number who actually took part.
- Started
-
Aug 2025
- Expected to finish
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Oct 2029
An estimate. End dates often move.
- Lead sponsor
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A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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18 years and older
- Sex
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Anyone
- Healthy volunteers
-
Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Key Inclusion Criteria: * 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: 1. Treatment-naive, measurably progressing DT that is deemed not amenable to surgery without the risk of significant morbidity; OR 2. Recurrent, measurably progressing DT following at least 1 line of therapy; OR 3. 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 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 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)
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Nagoya University Hospital
Nagoya, Aichi-ken, 466-8560, Japan
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National Cancer Center Hospital
Chuo Ku, Tokyo, 104-0045, Japan
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Osaka International Cancer Institute
Osaka, 541-8567, Japan
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Other studies related to the condition(s) this trial covers.
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