The purpose of this study is to build on the efficacy of the GEMOX regimen by adding Sorafenib in the treatment of Biliary Tract Cancer. Since there is no data on the combination of these three agents, the investigators plan to evaluate the safety in a run-in phase I portion in order to define the recommended phase II dose (RPTD). The phase II trial will enroll 40 patients at the RPTD level within 2 years in order to provide a preliminary estimate of progression-free survival (primary endpoint of the trial) in the target population.
The purpose of this study is to build on the efficacy of the GEMOX regimen by adding Sorafenib in the treatment of Biliary Tract Cancer. Since there are no data on the combination of these three agents, the investigators plan to evaluate the safety in a run-in phase I portion in order to define the recommended phase II dose (RPTD). The phase II trial will enroll 40 patients at the RPTD level within 2 years in order to provide a preliminary estimate of progression-free survival (primary endpoint of the trial) in the target population.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
9
Intravenously (IV) on Day 1 of each 14 day cycle, until progression or unacceptable toxicity develops.
Intravenously (IV) on Day 2 of each 14 day cycle, until progression or unacceptable toxicity develops.
Orally, twice daily for each 14-day cycle, until progression or unacceptable toxicity develops.
University of Miami
Miami, Florida, United States
Phase I: Recommended Phase II Dose (RPTD) of the Combination of Sorafenib and GEMOX in Patients With Advanced Biliary Tract Cancer (BTC).
Establish the recommended phase II dose (RPTD) of the combination of sorafenib and GEMOX in patients with advanced biliary tract cancer (BTC).
Time frame: First two 14-day Phase I cycles
Phase II: Obtain an Estimate of the 9-month Progression-free Survival Rate in Patients With Advanced BTC Receiving the RPTD of the Combination Sorafenib and GEMOX.
Rate of study participants achieving progression-free survival at 9 months post-initiation of study therapy at RPTD. Progression-Free Survival (PFS) is defined as the time elapsed from the start of treatment to the date of documented progression or death, whichever comes first. For surviving patients without progression who begin alternative treatment, PFS will be censored at the last date of documented progression-free status prior to starting alternative treatment. Similarly, losses to follow up will be censored at the last date of documented progression-free status.
Time frame: 9 Months
Phase II: Estimate Overall Response Rate and Clinical Benefit Rate.
Overall response rate \[CR + PR\]. Clinical Benefit Rate \[Complete Response (CR) + Partial Response (PR) + Stable Disease (SD)\] per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0).
Time frame: About 9 Months
Phase II: Estimate Overall Survival
Overall survival is defined as the time elapsed from the start of treatment until death. For surviving patients, follow-up will be censored at the date of last contact.
Time frame: Start of treatment until death or date of last contact
Phase II: Further Evaluate the Safety of the Proposed Combination
Rate of study participants experiencing toxicity after receiving study therapy at the recommended Phase 2 Dose (RPTD).
Time frame: About 9 Months
Phase II: Explore Biomarkers of Response to the Combination
A study of the correlation between biomarker levels and response to RPTD study therapy. Blood samples for biomarker analysis are collected at baseline and on day 1 of Cycles 2 onward
Time frame: Baseline, Day 1 of Cycle 2 and subsequent cycles, about 9 Months
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