Cholangiocarcinoma is bile duct cancer, an adenocarcinoma arising from the biliary epithelium that can occur intrahepatically or extrahepatically. It is often diagnosed at advanced stages and generally has poor prognosis.
A phase one trial of RSO-021 achieved disease control in 67% of relapsed mesothelioma patients. Other advances include daraxonrasib for NRAS-driven melanoma, an aneuploidy biomarker for PARP inhibitor response in childhood cancers, and new research into a cancer target and cholangiocarcinoma treatment.
FDA approved zenocutuzumab-zbco (Bizengri) for NRG1 fusion-positive cholangiocarcinoma on May 8, 2026. The eNRGy trial showed a 36.8% ORR. It also received priority review, breakthrough, and orphan designations.
The FDA has granted Priority Review for lirafugratinib, an FGFR2 inhibitor for second-line cholangiocarcinoma treatment, with a PDUFA date of September 27, 2026. Clinical trial data showed a 46.5% objective response rate and 11.8-month median duration of response. The therapy demonstrated a manageable safety profile with lower rates of common FGFR inhibitor side effects.
Mayo Clinic is pursuing two distinct nanoparticle-based cancer treatments: milk-derived nanoparticles for targeted gene therapy in bile duct cancer, and magnetic nanoparticle hyperthermia for metastatic solid tumors.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT07738939 |
IKS04 Regimen in Advanced Solid Tumors That Express CA242 |
NOT_YET_RECRUITING | PHASE1 |
| NCT07729917 |
Precision Integrated Strategies and Efficacy Evaluation for Biliary Tract Cancers: An Umbrella Platform Study |
NOT_YET_RECRUITING | PHASE2 |
| NCT07714538 |
Breath Research Narrow Validation for Gastrointestinal Cancer Detection |
NOT_YET_RECRUITING | |
| NCT07704021 |
Safety and Efficacy of EUS-guided Gallbladder Drainage Under Endoscopist-directed Conscious Sedation |
COMPLETED | |
| NCT07673341 |
[18F]FTT Positron Emission Tomography/Computed Tomography to Predict Treatment Response in Patients Scheduled to Receive Gemcitabine, Cisplatin, and Durvalumab for Newly Diagnosed Cholangiocarcinoma |
NOT_YET_RECRUITING | PHASE2 |
| NCT07641036 |
Dual-Target HER2/CEA CAR-NK Cells in Advanced Biliary Tract Cancer |
RECRUITING | PHASE1/PHASE2 |
| NCT07623642 |
A Phase II Study of GV20-0251 in Combination With Anti-PD-1 Monoclonal Antibodies in Patients With Unresectable, Locally Advanced, or Metastatic Solid Tumors. |
NOT_YET_RECRUITING | PHASE2 |
| NCT07620080 |
EYEMAX® Versus SPYGLASS™ DS for Biliary Stricture Diagnosis |
NOT_YET_RECRUITING | NA |
| NCT07619937 |
Efficacy and Safety of Hepatic Arterial Infusion Chemotherapy Combined With Tislelizumab and Regorafenib as First-Line Therapy for Advanced Cholangiocarcinoma |
COMPLETED | PHASE2 |
| NCT07598318 |
Study of Becotatug Vedotin Added to Standard Treatment for Advanced Bile Duct Cancer With EGFR Mutations |
NOT_YET_RECRUITING | PHASE2/PHASE3 |
