GEN-001 Plus Pembrolizumab for Patients With Advanced Refractory 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 is a phase II, multicenter, open-label study with a safety run-in to evaluate the safety and efficacy of GEN-001 in combination with pembrolizumab or in combination with pembrolizumab and mFOLFOX for patients with advanced refractory BTC who have progressed after 1 or 2 prior standard therapy and are not candidates for any other standard therapy. The safety run-in phase will be conducted before the main study phase

Study Overview

• Status: Terminated
• Conditions: Biliary Tract Cancer
• Intervention / Treatment: Drug: GEN-001; Drug: Pembrolizumab; Drug: mFOLFOX

• Study Type: Interventional
• Enrollment (Actual): 10
• Phase: Phase 2

Contacts and Locations

Study Locations

• South Korea
  • Suwon, South Korea, 16499
    • Ajou University Medical Center
  • Seoul
    • Seoul, Seoul, South Korea, 03080
      • Seoul National University Hospital
    • Seoul, Seoul, South Korea, 08308
      • Korea University Guro Hospital
    • Seoul, Seoul, South Korea, 05505
      • Asan Medical Center
    • Seoul, Seoul, South Korea, 03722
      • Severance Hospital
    • Seoul, Seoul, South Korea, 06351
      • Samsung Medical Center.

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: GEN-001 with pembrolizumab or GEN-001 with pembrolizumab and mFOLFOX; Drug: GEN-001 Drug: pembrolizumab Drug: mFOLFOX	Drug: GEN-001; The capsules taken by mouth. Each capsule will contain ≥ 3x10^11 colony-forming units (CFU); Drug: Pembrolizumab; 200 mg given by intravenous (IV) infusion once every 3 weeks; Other Names: KEYTRUDA®; Drug: mFOLFOX; mFOLFOX given by intravenous (IV) once every 2 weeks for only cohort 3

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
The recommended Phase 2 dose (RP2D) of GEN-001 in patients with advanced refractory biliary tract cancer (BTC), when administered as combined with pembrolizumab or as combined with pembrolizumab and mFOLFOX.	Incidence of dose-limiting toxicity (DLT)	1 years
Objective Response Rate (ORR) per Response Evaluation Criteria in Solid Tumors (RECIST) v1.1 by the Investigator.	The assessment of the anti-tumor activity per cohort	1 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence of Adverse Event (AE)s and laboratory abnormalities per National Cancer Institute Common Terminology Criteria for AEs (NCI CTCAE) v.5.0. cohort.	The assessment the overall safety and tolerability per cohort.	1 years
Duration of Response (DoR)	the time from the first documentation of objective tumor response to the first documentation of objective tumor progression or to death due to any cause, whichever occurs first	1 years
Progression-free Survival (PFS)	the time from the start date of treatment to the date of PD by RECIST v1.1 or death due to any cause, whichever occurs first	1 years
Overall Survival (OS)	the time from the start date of treatment to the date of death	1 years

Collaborators and Investigators

• Sponsor: Genome & Company
• Collaborators: Merck Sharp & Dohme LLC

Study record dates

Study Major Dates

• Study Start (Actual): September 18, 2023
• Primary Completion (Actual): November 26, 2024
• Study Completion (Actual): November 26, 2024

Study Registration Dates

• First Submitted: August 1, 2023
• First Submitted That Met QC Criteria: August 16, 2023
• First Posted (Actual): August 21, 2023

Study Record Updates

• Last Update Posted (Actual): December 16, 2025
• Last Update Submitted That Met QC Criteria: December 8, 2025
• Last Verified: December 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Neoplasms by Site; Neoplasms; Digestive System Neoplasms; Digestive System Diseases; Biliary Tract Diseases; Biliary Tract Neoplasms; Molecular Mechanisms of Pharmacological Action; Antineoplastic Agents; Antineoplastic Agents, Immunological; Immune Checkpoint Inhibitors; pembrolizumab
• Other Study ID Numbers: [GNC] GEN001-202; KEYNOTE-D86 (Other Identifier: Merck Sharp & Dohme LLC); MK-3475-D86 (Other Identifier: Merck Sharp & Dohme LLC)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No