GEN-001 Plus Pembrolizumab for Patients With Advanced Refractory Biliary Tract Cancer

NCT05998447 | Terminated Phase 2 DMC

• 3 other identifiers: [GNC] GEN001-202KEYNOTE-D86MK-3475-D86
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 6

Brief Summary

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

Conditions

Biliary Tract Cancer

Outcome Measures

Primary Outcomes (2)

• 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 Outcomes (4)

• Incidence of Adverse Event (AE)s and laboratory abnormalities per National Cancer Institute Common Terminology Criteria for AEs (NCI CTCAE) v.5.0. cohort.

  1 years

• Duration of Response (DoR)

  1 years

• Progression-free Survival (PFS)

  1 years

• Overall Survival (OS)

  1 years

Study Arms (1)

GEN-001 with pembrolizumab or GEN-001 with pembrolizumab and mFOLFOX

EXPERIMENTAL

Drug: GEN-001 Drug: pembrolizumab Drug: mFOLFOX

Drug: GEN-001; Drug: Pembrolizumab; Drug: mFOLFOX

Interventions

Pembrolizumab DRUG

200 mg given by intravenous (IV) infusion once every 3 weeks

Also known as: KEYTRUDA®

GEN-001 with pembrolizumab or GEN-001 with pembrolizumab and mFOLFOX

mFOLFOX DRUG

mFOLFOX given by intravenous (IV) once every 2 weeks for only cohort 3

GEN-001 with pembrolizumab or GEN-001 with pembrolizumab and mFOLFOX

GEN-001 DRUG

The capsules taken by mouth. Each capsule will contain ≥ 3x10\^11 colony-forming units (CFU)

GEN-001 with pembrolizumab or GEN-001 with pembrolizumab and mFOLFOX

Eligibility Criteria

• Age: 19 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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)

You may not qualify if:

• 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

Sponsors & Collaborators

• Merck Sharp & Dohme LLC: collaborator
• Genome & Company: lead

Study Sites (6)

Seoul National University Hospital

Seoul, Seoul, 03080, South Korea

Location

Severance Hospital

Seoul, Seoul, 03722, South Korea

Location

Asan Medical Center

Seoul, Seoul, 05505, South Korea

Location

Samsung Medical Center.

Seoul, Seoul, 06351, South Korea

Location

Korea University Guro Hospital

Seoul, Seoul, 08308, South Korea

Location

Ajou University Medical Center

Suwon, 16499, South Korea

Location

MeSH Terms

Conditions

Biliary Tract Neoplasms

Interventions

pembrolizumab

Condition Hierarchy (Ancestors)

Digestive System Neoplasms; Neoplasms by Site; Neoplasms; Biliary Tract Diseases; Digestive System Diseases

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 1, 2023
• First Posted: August 21, 2023
• Study Start: September 18, 2023
• Primary Completion: November 26, 2024
• Study Completion: November 26, 2024
• Last Updated: December 16, 2025

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will not share

Locations

KR (6)