Study to Test the Benefit and Safety of GM-CT-01 in Combination With 5-FU to Treat Bile Duct and Gall Bladder Cancer

Phase 2, Multi-center, Open-label Trial to Evaluate Efficacy and Safety of GM-CT-01 in Combination With 5-FU as First Line Chemotherapy in Patients With Advanced Biliary Cancer

The purpose of this clinical trial is to determine whether the combination of the established chemotherapeutic agent 5-fluorouracil(5-FU) and the large carbohydrate molecule GM-CT-01 is beneficial in treating advanced gall bladder and bile duct cancer.

Determine the overall response rate (ORR) defined as complete response (CR)rate plus partial response (PR) rate using Response Evaluation Criteria in Solid Tumors (RECIST), as well as the stable disease (SD) rate in subjects with unresectable, locally advanced or metastatic cholangiocarcinoma or other biliary tract tumors treated with GM-CT-01 plus 5-Fluorouracil (DAVFU) at doses of 280 mg/m2 and 600 mg/m2, respectively, during cycles of 4 consecutive days of treatment followed by a 24-day follow-up period.

Inclusion Criteria:

1. 18 years of age or older.
2. Histologically or cytologically documented carcinoma primary to the intra- or extra-hepatic biliary system or gall bladder with clinical and/or radiologic evidence of unresectable, locally advanced or metastatic disease.
3. Prior neo-adjuvant or adjuvant therapy (including radiation therapy) is allowed provided it was completed at least 4 weeks before documented disease recurrence or metastasis.
4. Discontinuation of radiation therapy at least 3 weeks prior to Day 1 of Cycle 1. Radiation therapy will not be allowed while a subject is on study except for palliative radiation therapy to non-target lesions administered following consultation with the Medical Monitor.
5. Prior surgery must have been completed at least 14 days prior to Day 1 of Cycle 1.
6. Nne or more measurable target lesion(s) according to RECIST. Measurable lesion(s) must be outside of previous radiation field or demonstrate clear radiographic progression on serial imaging if within previous treatment field.
7. ECOG performance status less than or equal to 2.
8. Life expectancy greater or equal to 3 months.

Exclusion Criteria:

1. Central nervous system metastasis.
2. Bony metastasis as the sole metastasis.
3. Received prior chemotherapy or anti-angiogenesis agents including bevacizumab or erbitux or radiation therapy other than in a neo-adjuvant or adjuvant setting. No concomitant chemotherapy, anti-tumor biologic therapy, or radiation therapy is allowed.
4. If nitrosoureas or mitomycin C were used as neo-adjuvant or adjuvant therapy, then at least 6 weeks should have elapsed prior to treatment with DAVFU.
5. Active infection that requires treatment with systemic antibiotic, anti- fungal. or anti-viral therapy.
6. Congestive heart failure (Class III or IV in the NYHA functional classification system) or any other medical condition that would preclude the IV administration of up to approximately 200 mL of fluid over 30-60 minutes.
7. Unresolved biliary tract obstruction.
8. Known or clinically suspected infection with HIV.
9. Subject has a known intolerance to 5- FU.