Where bile duct cancer treatment is heading

Cholangiocarcinoma is cancer of the bile ducts, about 8,000 to 12,000 US cases a year. Most are found late. Immunotherapy with chemotherapy is the first treatment, and about 4 in 10 tumors have a gene change (FGFR2, IDH1, HER2 and others) with a matching drug.

• 308studies open or running
• 19phase 3 trials
• 2,158papers published this year
• 40NIH-funded projects

1. Now

   Nov 2026

   Immunotherapy doubles 3-year survivalHappened 14.6% alive at 3 years

   Durvalumab with chemotherapy: 14.6% alive at 3 years vs 6.9%.

   14.6%: alive at 3 years with durvalumab, vs 6.9%.

   • TOPAZ-1 3-year results

   Third FGFR2 drug approvedHappened 46% tumors shrank

   Lirafugratinib shrank tumors in 46% of people with FGFR2 changes; approved September 2026.

   46%: of tumors shrank.

   • Lirafugratinib approval

   Treatment before surgeryHappened 18.0 mo without relapse

   In a Chinese trial, pre-surgery therapy doubled time without relapse to 18.0 months.

   18.0 mo: without relapse with treatment before surgery, vs 8.7.

   • GOLP trial

2. 6 months

   by May 2027

   Tinengotinib after FGFR drugs stop workingPossible

   A 200-person phase 3 for FGFR-altered tumors that grew on earlier FGFR drugs finishes December 2026.

   • FIRST-308 trial

3. 12 months

   by Nov 2027

   More people tested for gene changesLikely

   With three FGFR2 drugs and HER2 and IDH1 options, tumor gene testing at diagnosis matters more.

   • Lirafugratinib approval

4. 3 years

   by late 2029

   Pre-surgery treatment tested in EuropePossible 18.0 mo without relapse

   PURITY (300 people, Italy) and NEODISCO (150, Netherlands) test treatment before surgery.

   18.0 mo: without relapse with treatment before surgery, vs 8.7.

   • GOLP trial
   • PURITY trial
   • NEODISCO trial

   HER2 drug moves to first treatmentPossible

   Zanidatamab, approved in 2024 after chemotherapy, is in a phase 3 trial as part of first treatment.

   • MD Anderson on zanidatamab

5. 5 years

   by late 2031

   Earlier detection for people at riskLong shot

   Mayo Clinic's NIH-funded program studies earlier detection and new treatments for liver and bile duct cancers.

   • Mayo Clinic hepatobiliary program

The research paths

Borrowed from other cancers

Open to join now

• Removable tumors, Italy

  Chemotherapy before surgery

  Phase 3 · 19 sites

  Locations and eligibility →

• Removable tumors, Netherlands

  Pembrolizumab with chemotherapy around surgery

  Phase 3 · 1 site

  Locations and eligibility →

• FGFR2 fusion

  Futibatinib

  Phase 2 · 65 sites

  Locations and eligibility →

See every recruiting Bile duct cancer trial →

When the trials finish

Completion dates the trials registered. Results usually follow 3 to 12 months later.

Knowledge on the way

Not treatments yet: detection, causes and how care is delivered. Mostly NIH-funded.

• AreaQuestionFunded throughNews likely
• Program Mayo Clinic research program on liver and bile duct cancersMayo Clinic · NIH Funded through Aug 2029 News likely Ongoing

What just happened

Standard treatment today

• Can be removedSurgery, then capecitabine for 6 months; treatment before surgery is being tested.
• Advanced, first treatmentGemcitabine and cisplatin with durvalumab or pembrolizumab.
• Gene change foundFGFR2: pemigatinib, futibatinib, lirafugratinib. IDH1: ivosidenib. HER2: zanidatamab.
• No gene change, after first treatmentFOLFOX chemotherapy or a clinical trial.

See Bile duct cancer trials recruiting now →