An Exploratory Clinical Trial of Vebreltinib for the Treatment of Patients With Locally Advanced or Metastatic Clear Cell Sarcoma
This is a prospective, cohort, multicenter study. Cohort 1 is treatment group. All eligible subjects will receive Vebreltinib (200 mg bid po) after signing the informed consent and meeting the inclusion/exclusion criteria, until disease progression, intolerable toxicity, or death. Subjects will undergo MET abnormality testing after enrollment, including MET amplification or MET protein overexpression. Cohort 2 is external reference group. Subjects diagnosed with CCS and met the inclusion criteria but refused to enter Cohort1 will receive the standard treatment decided by investigators. These subjects will receive follow-up. The efficacy and safety data will be collected. Imaging evaluation will be performed using RECIST v1.1, with CT or MR plain scans every two months (±7 days) until disease recurrence or death. During the study, subjects will receive safety follow-up, and survival follow-up will be conducted every two months after treatment, which can be done by telephone interview for approximately 3 years after treatment ends.
• Age ≥12 years old, male or female;
• ECOG PS score 0-2;
• Histopathologically confirmed unresectable locally advanced or metastatic clear cell sarcoma;
• Previous surgery, radiotherapy, or chemotherapy is allowed; patients who have used PD1/PDL1 for 4 months without effect are allowed to continue using the same PD1/PDL1 drug;
• At least 1 measurable lesion according to RECIST1.1 criteria;
• Normal function of major organs, meeting the following criteria within 7 days before treatment:① Hematology examination criteria must be met (without blood transfusion or blood products, and without correction by G-CSF or other hematopoietic stimulating factors within 14 days):a) Hemoglobin (HB) ≥90g/L; b) Absolute neutrophil count (ANC) ≥1.5×10⁹/L; c) Platelets (PLT) ≥100×10⁹/L; ② Biochemical examination must meet the following criteria: a) Total bilirubin (TBIL) ≤1.5× upper limit of normal (ULN); b) Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤2.5×ULN, and if with liver metastasis，ALT and AST ≤5×ULN; c) Serum creatinine (Cr) ≤1.5×ULN or creatinine clearance (CCr) ≥60ml/min; ③ Urine protein \<2+, and 24h urine protein quantitation shows protein ≤1g; ④ Coagulation function: INR \<2.0 and APTT ≤1.5×ULN ⑤ Doppler ultrasound assessment: Left ventricular ejection fraction (LVEF) ≥ lower limit of normal (60%) ⑥ Thyroid function: TSH ≤ upper limit of normal (ULN); if abnormal, T3 and T4 levels should be considered, and subjects can be enrolled if T3 and T4 levels are normal;
• Male or female of childbearing potential agree to use reliable contraception during treatment and for at least 12 months after the last study drug administration.
• Consent to sign the informed consent form, good compliance, and cooperation with follow-up.