A Randomized, Multicenter, Open-label, Phase 3 Study to Compare the Efficacy and Safety of GEMOX(Gemcitabine/Oxaliplatin) vs XELOX(Xeloda/Oxaliplatin) in Advanced Biliary Tract Carcinoma
In patients with advanced BTC(biliary tract cancer), either gemcitabine-based, 5-FU-based
chemotherapy or clinical trial is recommended as first-line treatment. According to ABC-02
trial, as compared with gemcitabine alone, cisplatin plus gemcitabine was associated with a
significant survival advantage without the addition of substantial toxicity. Cisplatin plus
gemcitabine is an appropriate option for the treatment of patients with advanced biliary
cancer. (ClinicalTrials.gov number, NCT00262769.) Recent metaanalysis , analyzed 104
phase II and III trials comprising 2810 BTC patients and found that gemcitabine combined
with platinum compounds such as cisplatin or oxaliplatin had superior response rate and
survival when compared with gemcitabine alone. The metaanalysis concluded the combination of
gemcitabine and cisplatin or oxaliplatin to be the reference arm for future clinical trials.
Meanwhile, oxaliplatin (l-OHP), an alkylating diaminocyclohexane platinum derivate, has been
noted to display a marked cytotoxic synergism in combination with fluoropyrimidines against
a variety of solid human tumour cells . Based on these information, Nehls et al. 
conducted a prospective phase II study of oxaliplatin plus 5-FU/folinic acid in biliary
system adenocarcinomas, and the disease control rate (responses and stable disease (SD)) was
56%, and the median OS was 9.5 months. To improve efficacy and to offer a more convenient
treatment option for patients by reducing clinical visits and avoiding indwelling devices,
they prospectively investigated the activity and toxicity profile of three-weekly
intravenous oxaliplatin plus oral capecitabine (XELOX), and concluded that the XELOX regimen
was a well-tolerated and active treatment option for advanced BTC .
Given a lack of prospective, direct, comparison between XELOX and GEMOX regimens in advanced
BTC, we propose a randomized phase III trial of GEMOX (gemcitabine/oxaliplatin) vs XELOX
(capecitabine/oxaliplatin) in metastatic or unresectable BTC patients.
Allocation: Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment
Progression free survival of GEMOX vs XELOX
reference 6 months PFS 50% (GEMOX arm), noninferiority 6 months PFS 35% (XELOX arm), 1:1 randomization, accrual 24 months, 6 months follow-up after the last patient registry.
6 months PFS
Korea: Food and Drug Administration