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Biliary Tract CancerPhase 2NCT05998447

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

A Phase 2 study testing GEN-001, Pembrolizumab and mFOLFOX in people with Biliary Tract Cancer, run by Genome & Company at 6 sites in South Korea.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial 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… Read more about this study

Who is it for?
Ages 19 years and older
Conditions the study is for
Biliary Tract Cancer
What is being tested?
GEN-001, Pembrolizumab and mFOLFOX
Could I receive a placebo?
No, everyone receives an active treatment
How many people
10 participants
Where
6 sitesSouth Korea
Study dates
18 Sep 2023to 26 Nov 2024
Phase
Phase 2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 19 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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)

Who cannot join

  • 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

Trial locations and contacts

6 sites in 1 country.

6 sites in 1 country

South Korea6 sites

  • Seoul National University Hospital

    Seoul, Seoul, 03080

  • Severance Hospital

    Seoul, Seoul, 03722

  • Asan Medical Center

    Seoul, Seoul, 05505

  • Samsung Medical Center.

    Seoul, Seoul, 06351

  • Korea University Guro Hospital

    Seoul, Seoul, 08308

  • Ajou University Medical Center

    Suwon, 16499

Other trials in South Korea

About this study

Official title: 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

Being tested
GEN-001, Pembrolizumab, mFOLFOX
Medical subject headings
Biliary Tract Neoplasms

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • GEN-001Drug

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

  • PembrolizumabDrug

    Also called KEYTRUDA®

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

  • mFOLFOXDrug

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

Study groups in NCT05998447
Study groupRoleWhat they receive
GEN-001 with pembrolizumab or GEN-001 with pembrolizumab and mFOLFOXExperimentalDrug: GEN-001 Drug: pembrolizumab Drug: mFOLFOX
Receives: Drug: GEN-001; Drug: Pembrolizumab; Drug: mFOLFOX

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. 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)

    Measured: 1 years

  2. 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

    Measured: 1 years

Secondary outcomes (4)

  1. 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.

    Measured: 1 years

  2. 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

    Measured: 1 years

  3. 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

    Measured: 1 years

  4. Overall Survival (OS)

    the time from the start date of treatment to the date of death

    Measured: 1 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted21 Aug 2023
  2. Study start18 Sep 2023
  3. Primary completion26 Nov 2024
  4. Study completion26 Nov 2024

Why it stopped: Sponsor decision

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
10 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05998447
Sponsor's protocol ID
[GNC] GEN001-202
Other IDs
KEYNOTE-D86, MK-3475-D86
Status last verified
Dec 2025
First submitted
1 Aug 2023
Last updated
16 Dec 2025
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing GEN-001, Pembrolizumab and mFOLFOX in people with Biliary Tract Cancer, run by Genome & Company at 6 sites in South Korea.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 3 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: stopped early, as of the sponsor's last update on 16 Dec 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 6 sites in South Korea. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study was stopped early (reason given: Sponsor decision). Results are not available in the source record.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

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. (time frame: 1 years). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05998447.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Dec 2025.

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