A Multicenter, Open-Label, Phase IIa Clinical Study Evaluating the Preliminary Efficacy and Safety of Sodium Porphyrin Injection Combined With Photodynamic Therapy Plus Gemcitabine and Cisplatin Chemotherapy in Patients With Advanced Extrahepatic Cholangiocarcinoma With Biliary Obstruction
Design: Multicenter, open-label, Single Group Study Population: Patients with locally advanced or recurrent metastatic extrahepatic cholangiocarcinoma who are inoperable or unwilling to undergo surgery. Primary Research
Objective: To preliminarily evaluate the efficacy of photodynamic therapy (PDT) with sodium protoporphyrin combined with gemcitabine and cisplatin (GC regimen) chemotherapy in patients with advanced extrahepatic cholangiocarcinoma complicated by biliary obstruction. Secondary Study Objective: To preliminarily evaluate the safety and tolerability of photodynamic therapy (PDT) with heme porphyrin sodium combined with gemcitabine and cisplatin (GC regimen) chemotherapy in patients with advanced extrahepatic cholangiocarcinoma with biliary obstruction. Primary endpoint: 6-month overall survival rate (6m-OS rate)
• Subjects must meet all of the following inclusion criteria to be enrolled in this study:
‣ All subjects or their legal representatives must voluntarily sign an ethics committee-approved informed consent form in writing prior to initiating any screening procedures;
⁃ Age ≥ 18 years, no gender restrictions;
⁃ Patients with histologically or cytologically confirmed, locally advanced or recurrent metastatic extrahepatic cholangiocarcinoma who are inoperable or unwilling to undergo surgery;
⁃ No prior systemic therapy during the recurrent or metastatic stage;
⁃ Presence of obstructive lesions in the extrahepatic bile ducts, with at least one measurable lesion outside the bile ducts according to the Response Evaluation Criteria in Solid Tumors (RECIST) 1.1;
⁃ Subjects with an ECOG performance status score of 0 or 1 (see Appendix 3 for details);
⁃ Subjects with an expected survival of ≥3 months;
⁃ Subjects with adequate organ and bone marrow function meeting the following laboratory criteria:
• Bone marrow function: Absolute neutrophil count (ANC) ≥1.5 × 10⁹/L (1500/mm³) ; platelets ≥100 × 10⁹/L (1 × 10⁵/mm³); hemoglobin ≥9.0 g/dL (no treatment for bone marrow suppression, such as GCS-F, EPO, or blood transfusion, within 14 days prior to screening laboratory tests);
∙ Renal Function: Creatinine clearance (Ccr) ≥50 mL/min (calculated using the Cockcroft-Gault formula; see Appendix 4);
∙ Coagulation Function: International Normalized Ratio (INR) ≤1.5 × ULN; Activated Partial Thromboplastin Time (APTT) ≤1.5 × ULN;
∙ Echocardiogram: Left ventricular ejection fraction (LVEF) ≥50%;
∙ 12-lead electrocardiogram: Fridericia-corrected QT interval (QTcF) \<470 msec (Appendix 4);
⁃ Recovered from all prior treatments with toxicities resolved to Grade 1 or below;
‣ Female subjects of childbearing potential and all male subjects must agree to use highly effective contraception during the trial and for 6 months after the last dose of sodium porphyrin. contraceptive gel, contraceptive film, intrauterine device, oral or injectable contraceptives, subcutaneous implant, etc.), and women of childbearing potential must have a negative pregnancy test result within ≤7 days prior to study drug administration.