Combination treatment for advanced biliary tract cancer

A Phase II Study to Evaluate the Safety and the Efficacy of GEN-001 in Combination With Pembrolizumab for Patients With Advanced Refractory Biliary Tract Cancer

This study is testing a new combination treatment for patients with advanced biliary tract cancer who haven't responded to other therapies to see if it can help them.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 148 (estimated)
• Ages: 19 Years and up
• Sex: All
• Sponsor: Genome & Company (Industry-sponsored)
• Drugs / interventions: pembrolizumab
• Locations: 6 sites (Seoul and 5 other locations)
• Trial ID: NCT05998447 on ClinicalTrials.gov

What this trial studies

This phase II, multicenter, open-label study evaluates the safety and efficacy of GEN-001 in combination with pembrolizumab, and potentially with mFOLFOX, for patients with advanced refractory biliary tract cancer who have not responded to prior therapies. The study includes a safety run-in phase before the main trial to assess initial safety outcomes. Eligible patients must have a confirmed diagnosis of unresectable, recurrent, or metastatic biliary tract adenocarcinoma and have progressed after one or two standard treatments.

Who should consider this trial

Good fit: Ideal candidates are adults aged 19 and older with advanced biliary tract adenocarcinoma that is unresectable or metastatic.

Not a fit: Patients who have previously received certain immune checkpoint inhibitors or those with specific immune-related adverse events may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a new therapeutic option for patients with advanced biliary tract cancer who have limited treatment choices.

How similar studies have performed: While there have been studies on similar combinations, this specific approach is novel and has not been extensively tested in this patient population.

Eligibility criteria

Inclusion Criteria:

* The patient (or legally acceptable representative if applicable) provides written informed consent for the trial.
* Be ≥19 years of age on day of signing informed consent.
* Patient with histologically or cytologically confirmed diagnosis of unresectable, recurrent, or metastatic advanced biliary tract adenocarcinoma of the gallbladder or biliary tree (either intrahepatic or extrahepatic cholangiocarcinoma)

Exclusion Criteria:

* A WOCBP who has a positive urine pregnancy test (within 72 hours) prior to treatment. If the urine test is positive or cannot be confirmed as negative, a serum pregnancy test will be required.
* Has received prior therapy with an anti-PD-1, anti-PD-L1, or anti-PD-L2 agent or with an agent directed to another stimulatory or co-inhibitory T-cell receptor (Cohort 1 only), and was discontinued from the previous IO therapy due to a Grade 3 or higher immune-related AE (irAE) (Cohort 2 and Cohort 3).
* Is currently participating and receiving study treatments or has participated in a study of an investigational agent and received the study therapy or has used an investigational device within 4 weeks prior to the first dose of study treatment.
* Has had an allogeneic tissue/solid organ transplan

Where this trial is running

Seoul and 5 other locations

• Seoul National University Hospital — Seoul, Korea, Republic of (Recruiting)
• Severance Hospital — Seoul, Korea, Republic of (Recruiting)
• Asan Medical Center — Seoul, Korea, Republic of (Recruiting)
• Samsung Medical Center. — Seoul, Korea, Republic of (Recruiting)
• Korea University Guro Hospital — Seoul, Korea, Republic of (Recruiting)
• Ajou University Medical Center — Suwon, Korea, Republic of (Recruiting)

Study contacts

• Study coordinator: Clinical Development Team

Conditions Biliary Tract Cancer