ActiveCancerNIHR-supported projectDigestion, Kidneys & Other Organs
Molecular targeted maintenance therapy versus standard of care in advanced biliary cancer: an international, randomised, controlled, open-label, platform phase 3 trial
Recipient organisationNIHR Cambridge Biomedical Research Centre
NIHR supportRecorded as supported by this research centre
PeriodMar 2025 — May 2028
In plain English
AI plain-English summary
A new clinical trial will test whether drugs tailored to a patient’s tumour DNA can keep advanced biliary cancer from progressing, rather than relying on standard chemotherapy alone. Biliary tract cancers are aggressive and often diagnosed late. Only one in five patients can have surgery, and five-year survival remains poor. Roughly half of these tumours carry specific genetic mutations that might be targetable with existing drugs. The SAFIR-ABC10 trial aims to find out whether matching those mutations to molecular therapies—after an initial 12-week course of chemotherapy—extends the time before the cancer worsens, compared with continuing the same chemotherapy. If the experimental arm proves superior, it could shift the standard of care for advanced biliary cancer from a one-size-fits-all chemotherapy regimen to a genetically guided maintenance strategy. That would mean longer disease control for patients whose tumours harbour targetable mutations, without the cumulative toxicity of ongoing chemotherapy. The trial is international and platform-based, meaning it can incorporate new drugs and mutation types as evidence evolves, potentially accelerating access to precision treatments for a cancer that currently has few options beyond first-line chemotherapy.
View original technical description
Biliary tract cancers (BTC) are a heterogenous group of malignancies of the bile ducts and gallbladder. The incidence of BTC,especially cholangiocarcinoma (CCA),has risen steadily over the past 30 years. BTCs are common worldwide and are the fifth most common cancer in the developing world. Surgery is currently the only curative treatment but only 1 in 5 cases can be surgically removed and the proportion of patients who survive after 5 years from being diagnosed remains poor. Recently,it was demonstrated that roughly half of BTCs feature a very clear set of changes in the genetic material (Jusakul,2017). In previous studies it was observed how some patients derived a clear clinical benefit from treatments that were targeting these specific changes (genetic mutations) with a medical approach called matching molecular targeted therapies (MTT) (Verlingue,2017a). These observations suggest that MTTs may be a useful strategy in advanced biliary tract cancers (ABC). SAFIR-ABC10 will recruit patients with ABC (cholangiocarcinoma or gallbladder adenocarcinoma) who will receive the chemotherapy approved treatment for this disease,which is a combination of two drugs: Cisplatin and Gemcitabine. During the first 12 weeks of treatment,the genetic mutations of these patients will be identified by taking a small piece of tumour tissue (archival or fresh biopsy if archival is unavailable) and analyse the DNA inside the cells. Depending on the type of genetic alterations that the patient displays,they will be treated with different drugs,which are designed to target that specific change in their tumour cells and for this reason are called “matching molecular therapies”. Patients who,by the end of the 12 weeks of treatment,have not experienced a worsening of their cancer or have not had significant toxicity from their treatment,will be randomly allocated to one of the two following groups: • Experimental arm: patients will be treated with MTT • Control arm: patients will be treated with the chemotherapy combination they have already received (Cisplatin + Gemcitabine)
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