| Biomarker | Variant | ESCAT | Evidence | Clinical significance | Drugs | Sources |
|---|---|---|---|---|---|---|
| BIO-FGFR2 | BICC1-FGFR2 fusion (most common partner ~30-40% of FGFR2-fusion cholangio) | IA | BICC1-FGFR2 fusion is the most common FGFR2 fusion partner in intrahepatic cholangiocarcinoma. Treatment is identical to gene-level FGFR2-fusion: pemigatinib (FIGHT-202) and futibatinib (FOENIX-CCA2). Fusion-partner identity does not currently modify TKI selection. | pemigatinib monotherapy futibatinib monotherapy |
| |
| BIO-FGFR2 | fusion / rearrangement (BICC1-FGFR2 most common; ~10-15% intrahepatic cholangio) | IA | FGFR2 fusion in intrahepatic cholangiocarcinoma (~10-15%): pemigatinib (FIGHT-202, Abou-Alfa Lancet Oncol 2020 — ORR 36%, mDOR 9.1 mo) and futibatinib (FOENIX-CCA2, Goyal NEJM 2023 — ORR 42%, mPFS 9.0 mo) are FDA-approved for previously-treated FGFR2-fusion cholangio. Futibatinib is irreversible and has activity against pemigatinib-resistance gatekeeper mutations. | pemigatinib monotherapy (2L+ post-gemcitabine/cisplatin) futibatinib monotherapy (2L+; preferred after pemigatinib resistance) |
|
IND-CHOLANGIO-2L-FGFR2-FUSION-PEMIGATINIB| ID | Name | Priority | Category | Where to order | Needed for |
|---|---|---|---|---|---|
| TEST-CBC | Complete Blood Count with Differential | Critical | lab | — | aggressive |
| TEST-CMP | Comprehensive Metabolic Panel | Critical | lab | — | aggressive |
| TEST-CT-CHEST-ABDOMEN-PELVIS | CT chest + abdomen + pelvis with IV contrast | Critical | imaging | — | aggressive |
| TEST-LFT | Liver Function Tests (ALT, AST, bilirubin, ALP, GGT, albumin) | Critical | lab | — | aggressive |
| TEST-ECG | Electrocardiogram | Standard | clinical_assessment | — | aggressive |
| TEST-NGS-COMPREHENSIVE | Comprehensive NGS tumor panel (DNA + RNA, ≥300 genes) | Desired | histology | CSD Lab: M065 | desired (aggressive) |
| # | Owner | Topic | Action |
|---|---|---|---|
| 1 | hematologist | Staging / disease burden | What is the current LDH? Marker of tumor burden and transformation. |
| 2 | molecular_geneticist | Biomarker status | What is the status of IDH1 / IDH2 mutation status (BIO-IDH-MUTATION)? It is required by track(s): IND-CHOLANGIO-2L-IDH1-IVOSIDENIB. Expected value: IDH1 R132 hotspot mutation positive (R132C / R132L / R132G / R132H / R132S; tumor NGS preferred; ctDNA acceptable when tumor tissue insufficient). IDH2 mutations NOT eligible.. |
| 3 | pathologist | Biomarker status | What is the status of HER2 status (solid tumors — gastric/GEJ/CRC scoring) (BIO-HER2-SOLID)? It is required by track(s): IND-CHOLANGIO-2L-HER2-ZANIDATAMAB. Expected value: HER2 IHC 3+ OR (IHC 2+ AND ISH amplified, HER2/CEP17 ratio ≥2.0); gastric-style scoring (Hofmann 2008 — basolateral/lateral membranous staining ≥10%). |
| 4 | clinical_pharmacist | Specialist review | Chemoimmunotherapy regimen — drug-drug interactions, dose adjustments, premedication. |
| Missing biomarker | Label | MDT owner | Default track | Required by | Next action |
|---|---|---|---|---|---|
BIO-HER2-SOLID | HER2 status (solid tumors — gastric/GEJ/CRC scoring) | pathologist | no | IND-CHOLANGIO-2L-HER2-ZANIDATAMAB | Verify result, method, specimen, and report date before sign-off. Expected/constraint: HER2 IHC 3+ OR (IHC 2+ AND ISH amplified, HER2/CEP17 ratio ≥2.0); gastric-style scoring (Hofmann 2008 — basolateral/lateral membranous staining ≥10%) |
BIO-IDH-MUTATION | IDH1 / IDH2 mutation status | molecular_geneticist | no | IND-CHOLANGIO-2L-IDH1-IVOSIDENIB | Verify result, method, specimen, and report date before sign-off. Expected/constraint: IDH1 R132 hotspot mutation positive (R132C / R132L / R132G / R132H / R132S; tumor NGS preferred; ctDNA acceptable when tumor tissue insufficient). IDH2 mutations NOT eligible. |
| Specialist | skill_id | Version | Last reviewed | Sign-offs | Domain |
|---|---|---|---|---|---|
| Cellular therapy specialist (CAR-T) | cellular_therapy_specialist | v0.1.0 | 2026-04-25 | 0 | cellular_therapy |
| Clinical pharmacist | clinical_pharmacist | v0.1.0 | 2026-04-25 | 0 | clinical_pharmacy |
| Hematologist / oncohematologist | hematologist | v0.1.0 | 2026-04-25 | 0 | hematology_oncology |
| Hematopathologist (lymphoma / leukemia / myeloma) | hematopathologist | v0.1.0 | 2026-04-25 | 0 | hematopathology |
| Infectious disease / hepatology | infectious_disease_hepatology | v0.1.0 | 2026-04-25 | 0 | infectious_diseases |
| Medical oncologist (solid-tumor chemotherapist) | medical_oncologist | v0.1.0 | 2026-04-25 | 0 | solid_oncology |
| Molecular geneticist / molecular oncologist | molecular_geneticist | v0.1.0 | 2026-04-25 | 0 | molecular_oncology |
| Palliative care | palliative_care | v0.1.0 | 2026-04-25 | 0 | palliative_care |
| Pathologist (general) | pathologist | v0.1.0 | 2026-04-25 | 0 | pathology |
| Primary care / family physician | primary_care | v0.1.0 | 2026-04-25 | 0 | primary_care |
| Psycho-oncologist | psychologist | v0.1.0 | 2026-04-25 | 0 | psychosocial |
| Radiation oncologist | radiation_oncologist | v0.1.0 | 2026-04-25 | 0 | radiation_oncology |
| Radiologist | radiologist | v0.1.0 | 2026-04-25 | 0 | diagnostic_imaging |
| Social worker / case manager | social_worker_case_manager | v0.1.0 | 2026-04-25 | 0 | psychosocial |
| Surgical oncologist | surgical_oncologist | v0.1.0 | 2026-04-25 | 0 | surgical_oncology |
| Transplant specialist (BMT) | transplant_specialist | v0.1.0 | 2026-04-25 | 0 | cellular_therapy |
| NCT | Title | Phase | Status | Sponsor | UA | Signals | Eligibility (excerpt) |
|---|---|---|---|---|---|---|---|
| NCT05010681 | Lenvatinib Plus Sintilimab in Patients With Immune Checkpoint Inhibitor Previously Treated Advanced Liver Cancer | PHASE2 | RECRUITING | Fudan University | — | Small N (<50) Surrogate endpoint only Single country | |
| NCT04753996 | Characterization of Biliary Cell-derived Organoids From Bile of PSC and Non-PSC Patients | N/A | RECRUITING | Mayo Clinic | — | Single country | |
| NCT06298968 | Combined Therapy Using Gemcitabine and Cisplatin Chemotherapy, Lenvatinib and Adebrelimab for Patients With Advanced and Unresectable Intrahepatic Cholangiocarcinoma | PHASE2 | RECRUITING | Nanfang Hospital, Southern Medical University | — | Small N (<50) Surrogate endpoint only Single country | |
| NCT06208462 | Neoadjuvant Therapy of HAIC(GEMOX) Combined With Adebrelimab and Lenvatinib for Resectable Intrahepatic Cholangiocarcinoma With High-risk Recurrence Factors | PHASE2 | RECRUITING | The First Affiliated Hospital with Nanjing Medical University | — | Small N (<50) Single country | |
| NCT06192797 | Combined HAIC, Lenvatinib and Pucotenlimab As Conversion Therapy for Unresectable Intrahepatic Cholangiocarcinoma | PHASE2 | RECRUITING | Tongji Hospital | — | Small N (<50) Single country | |
| NCT07105852 | Exploring the Safety and Efficacy of Sacituzumab Tirumotecan Combined With Pucotenlimab in the Treatment of Advanced Cholangiocarcinoma | PHASE2 | RECRUITING | Sir Run Run Shaw Hospital | — | Surrogate endpoint only Single country | |
| NCT06342414 | An Exosome-Based Liquid Biopsy for the Differential Diagnosis of Primary Liver Cancer | N/A | RECRUITING | City of Hope Medical Center | — | — | |
| NCT06925516 | A Trial of Apatinib and Adebrelimab in Combination With Chemotherapy in Patients With Unresectable ICC | PHASE2 | RECRUITING | Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University | — | Small N (<50) Surrogate endpoint only Single country | |
| NCT06964425 | Raman Spectroscopy in the Diagnosis of Extrahepatic Cholangiocarcinoma - a Pilot Study | NA | RECRUITING | University Hospital Olomouc | — | Small N (<50) Single country | |
| NCT06583993 | A Clinical Trial Evaluating the Safety and Efficacy of Intravenous HNF4α srRNA in Treating Advanced ICC Patients | EARLY_PHASE1 | RECRUITING | Shanghai Changzheng Hospital | — | Small N (<50) Single country |
Verify recruitment status directly with the trial site. ctgov data can lag behind current UA-site status.
| Option | UA registration | NSZU | Cost orientation | Access pathway |
|---|---|---|---|---|
| Aggressive plan Pemigatinib monotherapy (FIGHT-202) — 2L+ FGFR2-fusion cholangiocarcinoma (REG-PEMIGATINIB-CHOLANGIO) 1/1 component drug(s) not registered in Ukraine +1 | ✗ not registered | ✗ out-of-pocket | ₴-? — verify pathway | not recorded |
| Aggressive plan Ivosidenib monotherapy (ClarIDHy) — 2L+ IDH1-mutated cholangiocarcinoma (REG-IVOSIDENIB-CHOLANGIO) 1/1 component drug(s) not registered in Ukraine +1 | ✗ not registered | ✗ out-of-pocket | ₴-? — verify pathway | not recorded |
| Aggressive plan Zanidatamab — 2L+ HER2-amplified biliary tract cancer (HERIZON-BTC-01) (REG-ZANIDATAMAB) 1/1 component drug(s) not registered in Ukraine +1 | ✗ not registered | ✗ out-of-pocket | ₴-? — verify pathway | not recorded |
| Standard plan Gemcitabine + cisplatin (advanced biliary tract cancer, 1L — ABC-02) (REG-GEMCITABINE-CISPLATIN-CHOLANGIO) | ✓ registered | ✓ covered | ₴-? — verify pathway | NSZU formulary |
| Trial · NCT05010681 Lenvatinib Plus Sintilimab in Patients With Immune Checkpoint Inhibitor Previously Treated Advanced Liver Cancer No UA site listed — international referral required | — unknown | — unknown | self-pay: ₴0/course | Trial sponsor |
| Trial · NCT04753996 Characterization of Biliary Cell-derived Organoids From Bile of PSC and Non-PSC Patients No UA site listed — international referral required | — unknown | — unknown | self-pay: ₴0/course | Trial sponsor |
| Trial · NCT06298968 Combined Therapy Using Gemcitabine and Cisplatin Chemotherapy, Lenvatinib and Adebrelimab for Patients With Advanced and Unresectable Intrahepatic Cholangiocarcinoma No UA site listed — international referral required | — unknown | — unknown | self-pay: ₴0/course | Trial sponsor |
| Trial · NCT06208462 Neoadjuvant Therapy of HAIC(GEMOX) Combined With Adebrelimab and Lenvatinib for Resectable Intrahepatic Cholangiocarcinoma With High-risk Recurrence Factors No UA site listed — international referral required | — unknown | — unknown | self-pay: ₴0/course | Trial sponsor |
| Trial · NCT06192797 Combined HAIC, Lenvatinib and Pucotenlimab As Conversion Therapy for Unresectable Intrahepatic Cholangiocarcinoma No UA site listed — international referral required | — unknown | — unknown | self-pay: ₴0/course | Trial sponsor |
| Trial · NCT07105852 Exploring the Safety and Efficacy of Sacituzumab Tirumotecan Combined With Pucotenlimab in the Treatment of Advanced Cholangiocarcinoma No UA site listed — international referral required | — unknown | — unknown | self-pay: ₴0/course | Trial sponsor |
| Trial · NCT06342414 An Exosome-Based Liquid Biopsy for the Differential Diagnosis of Primary Liver Cancer No UA site listed — international referral required | — unknown | — unknown | self-pay: ₴0/course | Trial sponsor |
| Trial · NCT06925516 A Trial of Apatinib and Adebrelimab in Combination With Chemotherapy in Patients With Unresectable ICC No UA site listed — international referral required | — unknown | — unknown | self-pay: ₴0/course | Trial sponsor |
| Trial · NCT06964425 Raman Spectroscopy in the Diagnosis of Extrahepatic Cholangiocarcinoma - a Pilot Study No UA site listed — international referral required | — unknown | — unknown | self-pay: ₴0/course | Trial sponsor |
| Trial · NCT06583993 A Clinical Trial Evaluating the Safety and Efficacy of Intravenous HNF4α srRNA in Treating Advanced ICC Patients No UA site listed — international referral required | — unknown | — unknown | self-pay: ₴0/course | Trial sponsor |
Cost information is orientation. Verify with a specific pharmacy / foundation / trial site. Status updated: 2026-07-15.