Biliary Tract Cancer
Conditions
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
Interventions
Sponsors
Merck Sharp & Dohme LLC
Genome & Company
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
19 Years to No maximum
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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. | 1 years | Incidence of dose-limiting toxicity (DLT) |
| Objective Response Rate (ORR) per Response Evaluation Criteria in Solid Tumors (RECIST) v1.1 by the Investigator. | 1 years | The assessment of the anti-tumor activity per cohort |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| 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 | The assessment the overall safety and tolerability per cohort. |
| Duration of Response (DoR) | 1 years | 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 |
| Progression-free Survival (PFS) | 1 years | 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 |
| Overall Survival (OS) | 1 years | the time from the start date of treatment to the date of death |
Countries
South Korea
Outcome results
None listed