Dose-escalation Study to Investigate the Safety, PK, and PD of ISU304/CB2679d in Hemophilia B Patients

Inclusion Criteria

• Previously treated male patients with moderate or severe hemophilia B (documented FIX activity ≤ 2% and exposed to any FIX product for ≥ 150 exposure days (estimated) at the time of screening)
• Patients must be 12 to 65 years old at the time of screening
• Patients who have discontinued a previously treated FIX product at least 4 days prior to the administration of investigational product
• HIV negative, or if HIV positive with a CD4 count > 200/μL (documented < 200 particles/μL or ≤ 400,000 copies/mL) at the time of screening
• Voluntary consent to participate in the study

Exclusion Criteria

• Patients with a history or a family history of FIX inhibitors
• Patients with FIX inhibitors (positive result for BeneFIX or ISU304 from inhibitor tests) at the time of screening
• Patients who have a history of thromboembolic events (myocardial infarction, cerebrovascular disease, venous thrombosis, etc.)
• Patients with known hypersensitivity, allergy, or anaphylaxis to any FIX product or hamster protein
• Patients receiving treatment with a FIX product or a bypass agent within 4 half-lives for the agent used (at least 96 hours) prior to the administration of the investigational product
• Patients who have been exposed to long-term administration of immunomodulating agents or immunosuppressants such as α-INF or adrenocortical hormones over the past 3 months or who are currently receiving or planning to receive such treatment during the study period
• Patients who have been administered vaccines during the period of 6 months prior to the administration of the investigational product or plan to receive vaccines during the study period
• Patients with any other co-existing bleeding disorder (Von Willebrand disease, etc.)
• Patients with positive D-dimer results (≥ 0.5 μg/mL) at the time of screening
• Patients with platelet counts less than 100,000/μL at the time of screening
• Patients with ALT, AST levels 5 times greater than upper normal limit or total bilirubin, serum creatinine levels 2 times greater than upper normal limit at the time of screening
• Active hepatitis patients who are HBs Ag positive or anti-HCV Ab positive at the time of screening
• Patients scheduled for surgery during the study period
• Patients participated in another study within 30 days before screening or scheduled to participate in any other study during the study period