Pimicotinib for Japanese people with tenosynovial giant cell tumor (J-MANEUVER)
Phase 2, Single-arm Study to Investigate the Tolerability, Pharmacokinetics, Efficacy, and Safety of Pimicotinib in Japanese Participants With Tenosynovial Giant Cell Tumor
This trial will test whether pimicotinib is safe, reaches effective blood levels, and improves symptoms in Japanese participants with unresectable tenosynovial giant cell tumor.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Enrollment | 20 (estimated) |
| Ages | 18 Years and up |
| Sex | All |
| Sponsor | Merck Healthcare KGaA, Darmstadt, Germany, an affiliate of Merck KGaA, Darmstadt, Germany Industry-sponsored |
| Drugs / interventions | pimicotinib |
| Locations | 1 site (Fukuoka) |
| Trial ID | NCT07499362 on ClinicalTrials.gov |
What this trial studies
This Phase 2 interventional trial gives oral pimicotinib, a CSF-1R inhibitor, to Japanese adults with histologically confirmed, unresectable tenosynovial giant cell tumor. The study includes a safety run-in cohort followed by an expansion cohort to characterize tolerability, pharmacokinetics, efficacy, and overall safety. Eligible participants are symptomatic (worst pain or stiffness ≥4), have ECOG performance status 0–1, and adequate organ function, and they attend regular clinic visits for imaging, labs, and symptom assessments. Primary outcomes focus on tolerability and PK, with secondary outcomes measuring tumor response and changes in symptoms and function.
Who should consider this trial
Good fit: Ideal candidates are Japanese adults with histologically confirmed, unresectable TGCT who are symptomatic (worst pain or stiffness ≥4), have ECOG 0–1, and meet blood and organ function requirements.
Not a fit: Patients with resectable or asymptomatic TGCT, poor performance status, significant organ dysfunction, or who do not meet eligibility criteria are unlikely to benefit from this study.
Why it matters
Potential benefit: If successful, pimicotinib could reduce tumor-related pain and stiffness and may delay or avoid the need for surgery in patients with unresectable TGCT.
How similar studies have performed: Other CSF-1R inhibitors such as pexidartinib have produced tumor shrinkage and symptom improvement in TGCT, so the mechanism has clinical precedent, though pimicotinib is less studied in Japanese patients.
Eligibility criteria
Show full inclusion / exclusion criteria
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
Where this trial is running
Fukuoka
- Kyushu University Hospital - 300173484 — Fukuoka, Japan (Recruiting)
Study contacts
- Study coordinator: Communication Center
- Email: service@emdgroup.com
- Phone: +49 6151 72 5200
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.