Phase 2, Randomized, Double-Blind, Placebo-Controlled of the Efficacy and Safety of CF102 in Hepatocellular Carcinoma (HCC)

Inclusion Criteria

• Males and females at least 18 years of age.
• Diagnosis of HCC:
• For subjects without underlying cirrhosis at the time of diagnosis, diagnosis of HCC documented by cytology and/or histology.
• For subjects with underlying cirrhosis at the time of diagnosis, diagnosis of HCC established according to the American Association for the Study of Liver Diseases Practice Guideline algorithm (Appendix E).
• HCC is advanced, ie, treatment-refractory or metastatic, and no standard therapies are expected to be curative.
• Receipt of 1 previous systemic drug therapy for at least 3 weeks and withdrawal from treatment due either to intolerability or to radiographic disease progression. If treatment was withdrawn due to intolerability manifested as a Grade 3 or 4 event by National Cancer Institute Common Toxicity Criteria for Adverse Events (CTCAE v4.0), less than 3 weeks of continuous prior administration prior to withdrawal is acceptable (see also Exclusion Criterion #3).
• Prior systemic treatment was discontinued for at least 2 weeks prior to the Baseline Visit.
• Eastern Cooperative Oncology Group (ECOG) performance status (PS) of ≤ 2 (Appendix B).
• Cirrhosis classified as Child-Pugh Class B (Appendix C).
• The following laboratory values must be documented within 3 days prior to the first dose of study drug:
• Absolute neutrophil count (ANC) ≥ 1.5 × 109/L
• Platelet count ≥ 75 × 109/L
• Serum creatinine ≤ 2.0 mg/dL
• Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤ 5 × the upper limit of normal (ULN)
• Total bilirubin ≤ 3.0 mg/dL
• Serum albumin ≥ 2.8 g/dL
• Prothrombin time (PT) no greater than 6 seconds longer than control.
• Life expectancy of ≥ 6 weeks.

Exclusion Criteria

• Receipt of no, or of >1, prior systemic drug therapies for HCC.
• Receipt of systemic cancer therapy, immunomodulatory drug therapy, immunosuppressive therapy, or corticosteroids > 20 mg/day prednisone or equivalent within 14 days prior to the Baseline Visit or concurrently during the trial.
• Presence of an acute or chronic toxicity of prior chemotherapy that has not resolved to ≤ Grade 1, as determined by CTCAE v 4.0.
• Locoregional treatment within 4 weeks prior to the Baseline Visit.
• Major surgery or radiation therapy within 4 weeks prior to the Baseline Visit.
• Use of any investigational agent within 4 weeks prior to the Baseline Visit.
• Child-Pugh Class A or C cirrhosis, or hepatic encephalopathy.
• Occurrence of esophageal or other gastrointestinal hemorrhage requiring transfusion within 4 weeks prior to the Baseline Visit.
• Active bacterial, viral, or fungal infection requiring systemic therapy or operative or radiological intervention.
• Known human immunodeficiency virus- or acquired immunodeficiency syndrome-related illness.
• Liver transplant.
• Active malignancy other than HCC.
• Uncontrolled arterial hypertension or congestive heart failure (New York Heart Association Classification 3 or 4) (Appendix B).
• Angina, myocardial infarction, cerebrovascular accident, coronary/peripheral artery bypass graft surgery, transient ischemic attack, or pulmonary embolism within 3 months prior to initiation of study drug.
• History of or ongoing cardiac dysrhythmias requiring treatment, atrial fibrillation of any grade, or persistent prolongation of the QTc (Fridericia) interval to > 450 msec for males or > 470 msec for females.
• Pregnant or lactating female.
• Any severe, acute, or chronic medical or psychiatric condition, or laboratory abnormality that may increase the risk associated with trial participation or study drug administration; may interfere with the informed consent process and/or with compliance with the requirements of the trial; or may interfere with the interpretation of trial results and, in the Investigator's opinion, would make the subject inappropriate for entry into this trial.