Trial of Therapeutic Vaccine in Patients With Cholangiocarcinoma

Trial of Therapeutic Vaccine in Patients With Cholangiocarcinoma

Invitation only.

Purpose
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.

Condition Intervention Phase
Cholangiocarcinoma
Biological: Oral therapeutic vaccine V3-X to treat cholangiocarcinoma
Phase 1
Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Intervention Model Description:
one arm open label study to last 2 months
Masking: No masking
Primary Purpose: Treatment
Official Title: Open Label Trial of Therapeutic Vaccine in Patients With Cholangiocarcinoma

Resource links provided by NLM:

Genetics Home Reference related topics: cholangiocarcinoma
U.S. FDA Resources

Further study details as provided by Immunitor LLC:

Primary Outcome Measures:
Changes in CA19.9 tumor marker induced by daily dose of oral vaccine V3-X of cholangiocarcinoma [ Time Frame: 2 months ]
open label trial of once daily tablet of V3-X vaccine

Secondary Outcome Measures:
safety of vaccine [ Time Frame: 2 months ]
toxicity or adverse side effects, such as diarrhea and vomiting, we would have graded them according to accepted standards, e.g., NCI CTEP CTCAE.

Estimated Enrollment: 20
Anticipated Study Start Date: February 20, 2017
Estimated Study Completion Date: April 20, 2019
Estimated Primary Completion Date: February 20, 2019 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Single pill of V3-X vaccine administered once daily
One pill of oral therapeutic vaccine V3-X administered to patients with cholangiocarcinoma for two months and changes in CA19.9 tumor marker from baseline levels versus post-treatment levels will be assessed as correlates of changes in tumor burden
Biological: Oral therapeutic vaccine V3-X to treat cholangiocarcinoma
One single pill of V3-X vaccine administered once per day to patients with cholangiocarcinoma

Detailed Description:
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.
Eligibility

Ages Eligible for Study: Child, Adult, Senior
Sexes Eligible for Study: All
Accepts Healthy Volunteers: No
Criteria
Inclusion Criteria:

only those positive for CA19.9
Exclusion Criteria:

pregnant and lactating females
Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT03042182

Locations
Mongolia
Immunitor LLC
Ulaanbaatar, CA, Mongolia, 13838
Sponsors and Collaborators
Immunitor LLC
Investigators
Study Director: aldar bourinbaiar, MD/PhD Immunitor LLC
More Information

Responsible Party: Immunitor LLC
ClinicalTrials.gov Identifier: NCT03042182 History of Changes
Other Study ID Numbers: 03
Study First Received: January 26, 2017
Last Updated: February 1, 2017