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New pill shows promise for rare joint tumor in early trial
NCT ID NCT07499362
First seen Jun 26, 2026 · Last updated Sep 11, 2026 · Updated 6 times
Summary
This phase 2 trial tests a daily oral drug called pimicotinib in 20 Japanese adults with tenosynovial giant cell tumor (TGCT) that cannot be removed by surgery. The study aims to see if the drug is safe, how it moves through the body, and whether it shrinks tumors. Participants take the drug in 28-day cycles until their disease worsens or side effects become unacceptable.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- pimicotinib (a drug taken by mouth once daily)
- What this could lead to
- If successful, this could provide a new treatment option for people with TGCT that cannot be surgically removed, potentially shrinking tumors and easing symptoms.
- What could go wrong
- This is a small, early-phase study (20 participants) with no placebo group. It only tests one dose and focuses on Japanese patients, so results may not apply broadly. Side effects and lack of efficacy are possible.
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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About 20 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Mar 2026
- Expected to finish
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Nov 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
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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.
Inclusion Criteria: * Japanese participants with a diagnosis of TGCT that has been histologically confirmed at the local laboratory and is unresectable (that is \[i.e.\] it is located in a complex anatomical site, extensively invasive, and cannot be completely resected; or a surgical operation may cause dysfunction or serious complications). Symptomatic disease because of active TGCT, defined as a worst pain of greater than or equal to \[\>=\] 4 within 2 weeks prior to enrollment (based on scale of 0 to 10, with 10 representing "pain as bad as you can imagine"), and/or a worst stiffness of \>= 4 within 2 weeks prior to first dose of pimicotinib (based on a scale of 0 to 10, with 10 representing "stiffness as bad as you can imagine") * Eastern Cooperative Oncology Group (ECOG) Performance Status less than or equal to \[\<=\] 1 * Participants with adequate hepatic, renal hematologic functions * Other protocol defined inclusion criteria may apply Exclusion Criteria: * Known additional malignancy that required active treatment and may affect the participant's participation in the study or affect the outcome of the study as assessed by the Investigator. Exceptions include cured basal cell carcinoma of skin, squamous cell carcinoma of skin, and other carcinoma in situ * Serious gastrointestinal bleeding within 3 months of first dose of pimicotinib or factors that significantly affected the absorption of oral drug, such as inability to take oral medication or significant nausea and vomiting, malabsorption, external bile duct drainage, massive small-bowel resection, etc. * Impaired cardiac function or clinically significant cardiac disease, including any one of the following: New York Heart Association (NYHA) class III or IV heart disease, active ischemia or any other uncontrolled cardiac condition such as angina pectoris, clinically significant cardiac arrhythmia requiring therapy, uncontrolled hypertension or congestive heart failure; prolongation of the rate corrected QT interval based on repeated demonstration of QT interval corrected using Fridericia's formula (QTcF) greater than (\>) 480 milliseconds (ms), or history of long QT interval corrected (QTc) syndrome; Left ventricular ejection fraction (LVEF) less than (\<) 50 percent (%) or below the lower limit of normal, whichever is higher * Cerebrovascular accident/stroke (within 6 months of first dose of pimicotinib) * Other protocol defined exclusion criteria may apply
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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.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The places running it
4 sites. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
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Kanazawa University Hospital
RECRUITINGKanazawa, Japan
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Kyushu University Hospital - 300173484
RECRUITINGFukuoka, Japan
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Nagoya University Hospital - 300176284
RECRUITINGNagoya, Japan
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National Cancer Center Hospital
RECRUITINGChūōku, Japan
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- New pill for tough tumors enters human testing
- New pill could shrink rare joint tumors without surgery
- New drug hope for rare joint tumor patients who Can't have surgery
- Leukemia drug shows promise against rare joint tumors
- New drug shows promise for rare joint tumor in early trial
- New drug could shrink rare joint tumors without surgery