Current Cholangiocarcinoma Trials

Listed here are the clinical trials for those with cholangiocarcinoma (bile duct cancer) or gall bladder cancer, currently open and recruiting in the UK.

Current Cholangiocarcinoma Trials

Wherever possible links for further information have been included to follow up for more information and to check eligibility.  Each trial has specific eligibility criteria, so if you are interested in taking part in one, please discuss with those running the trial (contact details are given where possible) and/or your consultant.

Please note it is not appropriate for AMMF to give medical advice or recommendations, and all details provided are for information purposes only.

Molecular Profiling and Targeted Therapies

Current scientific advances mean that cancer treatments are moving towards those more specifically targeting the tumour ‘drivers’, and ways to inhibit these drivers. Progress is beginning to be made in the understanding of cholangiocarcinoma and of genomic or molecular profiling, with trials being carried out to find target agents, in the search for better and more effective treatments.

Several of the clinical trials listed here are looking at targeted therapies and will require molecular profiling to be carried out first to see if a patient has the particular mutation or fusion which the therapy will act on.

For a short animated film explaining “Cholangiocarcinoma and the importance of molecular testing” and a booklet, “ Molecular testing in cholangiocarcinoma”, click here

Pemigatinib – first targeted therapy to be approved for cholangiocarcinoma: In July 2021 NICE approved pemigatinib (Pemazyre®) for those with cholangiocarcinoma who have an FGFR2 fusion. Approval by the Scottish Medicines Consortium (SMC) followed in February 2022.  

This approval should give eligible patients in England, Wales and Scotland access to an alternative to chemotherapy in the 2nd line, but importantly should mean molecular profiling becomes mandated as standard of care, opening the door for all those diagnosed with cholangiocarcinoma to have molecular testing carried out.  

Read More


The ProvIDHe study is an early access Phase 3b study of ivosidenib in patients with pretreated locally advanced or metastatic cholangiocarcinoma and is now open in the UK.

This study is evaluating how safe and effective the targeted therapy drug, ivosidenib, is in people previously treated, locally advanced cholangiocarcinoma.

Eligibility criteria includes:

  • A diagnosis of nonresectable or metastatic cholangiocarcinoma (CCA), not eligible for curative-intent resection, transplantation, or ablative therapies
  • A documented IDH1, R132C, R132L, R132G, R132H, or R132S gene-mutated disease
  • Have tried at least one prior type of systemic therapy for CCA, and have recovered from any side effects
Read More


February 2023 AMMF understands that the Phase 2 RXC004 (PORCUPINE2) study has now closed to recruitment.  

April 2023: We understand that the revised Phase 3 study for those with biliary tract cancer – cholangiocarcinoma, gallbladder or ampullary cancer – using RXC004 + pembrolizumab, ie, RXC004 with the addition of an immunotherapy, is now open in some centres and that there will be no patient pre-selection required in terms of molecular profile.

If you are interested in this study, please speak to your consultant about this.  

We will further update the information on the Phase 2 and Phase 3 studies as soon as we can. 

A Phase 2 Study to Assess RXC004 Efficacy in Advanced Solid Tumours After Progression on Standard of Care (SoC) Therapy.

This second-line study is open for molecularly unselected patients with advanced biliary tract cancer, ie, cholangiocarcinoma, gallbladder or ampullary cancer, who have developed disease progression after first-line chemotherapy only.

It is also open to those with pancreatic cancer who have a specific mutation, RNF43, in their tumour.

Read More


A First-in-Human Study of Highly Selective FGFR2 Inhibitor, RLY-4008, in Patients with intrahepatic Cholangiocarcinoma and Other Advanced Solid Tumors

This is a Phase 1/2, open-label, first-in-human study designed to evaluate the safety, tolerability, PK, PDy*, and antineoplastic activity** of RLY-4008, a potent and highly selective FGFR2 inhibitor, in patients with unresectable or metastatic CCA and other solid tumors. The study consists of 3 parts: a dose escalation (Part 1), a dose expansion (Part 2), and an extension (Part 3).

*PK and PDY = Pharmacokinetics (PK) represents “what the body does to the drug,” whereas pharmacodynamics (PD) can be defined as “what the drug does to the body,” or, more specifically, to target cell-types, tissues, or organs.

** antineoplastic activity = Blocking the formation of neoplasms (growths that may become cancer).

Read More


FIGHT-302: Phase 3 Study of First-line Pemigatinib Versus Gemcitabine Plus Cisplatin for Cholangiocarcinoma With FGFR2 Fusions or Rearrangements.

This study will compare chemotherapy (CisGem) with an FGFR-inhibitor in FGFR-altered patients and will require prior analysis of a tumour biopsy to determine that there is the appropriate FGFR-alteration.

Read More


A Phase II, Single Arm Study of BGJ398 (infigratinib) in patients with advanced cholangiocarcinoma.

Full title: “A Phase II Multicenter, Single Arm Study of Oral BGJ398 in Adult Patients With Advanced or Metastatic Cholangiocarcinoma With FGFR2 Gene Fusions or Other FGFR Genetic Alterations Who Failed or Are Intolerant to Platinum-based Chemotherapy”

Read More


A Phase 2 Study of Erdafitinib in patients with Advanced Solid Tumours and FGFR Gene Alterations

This study seeks to evaluate the efficacy of erdafitinib in terms of overall response rate in participants with advanced solid tumours with FGFR (fibroblast growth factor receptor) mutations and gene fusions.

Erdafitinib is a selective and potent pan FGFR 1-4 inhibitor with demonstrated clinical activity in those with metastatic urothelial cancer and cholangiocarcinoma who have alterations in the FGFR pathway.

Read More


Although this study is now closed to recruitment, Zanidatamab (also know as ZW25) is available for eligible CCA patients under an Expanded Access/Compassionate Use Program.

Read More


DETERMINE is the first UK national precision medicine trial in rare cancers*, testing a range of therapies specifically targeting key genetic changes in cancer cells.

A non-randomised trial, DETERMINE will explore whether treatments approved for use in some cancer types can target identifiable genetic alterations in other types. And provide new treatments options for those who might not have any left.

This could include those with a BRAF V600 mutation-positive cholangiocarcinoma, or with HER2 amplification or activating mutations.

For further details, please see the Read More page. 

* Rare is defined generally as incidence fewer than 6 cases in 100,000 patients, or common cancers with rare alterations

Read More

Biliary Tract Cancer Virtual Tissue Bank – BTC VTB

Biliary tract cancers include cholangiocarcinoma (intrahepatic, perihilar, and extrahepatic), gallbladder cancer and ampullary cancer

The aim of the Biliary Tract Cancer Virtual Tissue Bank (BTC VTB) is to create a national tissue bank of human tissue samples (e.g. blood, tissue) from patients who had a biliary tract cancer and who have taken part in particular clinical trials. 

The samples will be stored in two locations in Manchester and London and the management of the samples will be run by UCL Cancer Trials Centre in London. 

Biliary tract cancers are quite rare with poor outcomes and it is often difficult to obtain large amounts of tissue, which makes this tissue bank extremely valuable.  Our aim is to make samples available for researchers so that more research can be done in biliary tract cancers which will, hopefully, help future patients. If we are better able to understand these cancers then the correct treatments can be used in clinical trials, which will then be used in clinic if successful.  

For more information:

Trials in set up

For information on trials that are currently in set up, please click here

Current trials no longer recruiting

For information on trials that are no longer recruiting, but are still live, please click here

National Institute for Health Research

The NIHR (National Institute for Health Research) “Be Part of Research” website has details of all UK clinical trials. Visit: and search for the name or number of the trial you are interested in.

Cancer Research UK also lists clinical trials for bile duct cancer (cholangiocarcinoma).  To see this, click here

For information on current treatments for cholangiocarcinoma, click here

August 2023