Trial of Therapeutic Vaccine in Patients With Cholangiocarcinoma
Trial of Therapeutic Vaccine in Patients With Cholangiocarcinoma
Cholangiocarcinoma (CCA) is a malignant neoplasm originating from the epithelial cells lining
 the intra- or extrahepatic biliary ducts. It is the second-most common liver cancer, after
 hepatocellular carcinoma (HCC). About 6,000 people in the United States develop bile duct
 cancer each year. One-year survival is less than 25% and no effective and safe systemic
 treatments are currently available. Last year the completion of open-label phase 2 trial
 (NCT02256514) of hepcortespenlisimut-L (V5) has been reported, which has shown that two-third
 of Mongolian patients with advanced HCC had a favorable clinical response, including complete
 remissions and with overall survival over 90% after 1 year. So far a few patients with CCA
 were treated with V5, but it appeared that their response rate was somewhat inferior to
 patients with HCC since two (both with hemochromatosis) out six patients died within 6
 months. In one patient who had improved clinically, the improvement was correlated with
 decrease in CA19-9 tumor marker, but no marker profile information is available in regard to
 other CCA patients. As V5 tablets are made from pooled blood of patients with HCC, in theory,
 they will be not very useful to patients with CCA. The goal of this project is to manufacture
 an immunotherapeutic formulation made from pooled heat- and chemically-inactivated blood from
 donors with CCA and initiate pilot open-label trial in 20 cholangiocarcinoma patients. This
 clinical trial will be conducted in collaboration with the National Cancer Center.
Upon obtaining regulatory and ethical approvals the Phase II single-arm study will be
 initiated at the NCC involving 20 patients with confirmed CCA diagnosis. The trial will be
 short, it will last only 2 months, but this will be sufficient to gauge the safety and
 efficacy. Only those patients who have higher than normal levels of CA19-9 tumor marker will
 be enrolled, which will serve as a surrogate marker in a manner alpha fetoprotein (AFP) has
 been used as a predictor of clinical response in HCC patients. Additional primary endpoints
 will be overall survival and changes in tumor burden, with secondary endpoints being liver
 function tests and changes in quality of life.
Inclusion Criteria:

 -  only those positive for CA19.9

 Exclusion Criteria:

 -  pregnant and lactating females