Trial of AAV5-hFIX in Severe or Moderately Severe Hemophilia B

Inclusion Criteria

• Male
• Age ≥ 18 years
• Patients with congenital hemophilia B classified as one of the following:
• Known severe FIX deficiency with plasma FIX activity level 2 times upper normal limit
• aspartate aminotransferase > 2 times upper normal limit
• total bilirubin > 2 times upper normal limit
• alkaline phosphatase > 2 times upper normal limit
• creatinine > 1.5 times upper normal limit
• Positive HIV serological test at Visit 1, not controlled with anti-viral therapy as shown by cluster of differentiation 4+ counts ≤ 200 per μL or by a viral load of >200 copies per mL (measured by the central laboratory)
• Active infection with Hepatitis B or C virus as reflected by Hepatitis B Surface Antigen (HBsAg), Hepatitis B extracellular Antigen (HBeAg), Hepatitis B Virus DeoxyriboNucleic Acid (HBV DNA) or Hepatitis C Virus RiboNucleic Acid (HCV RNA) positivity, respectively, at Visit 1 (measured by the central laboratory).
• History of Hepatitis B or C exposure, currently controlled by antiviral therapy
• Any coagulation disorder other than hemophilia B
• Thrombocytopenia, defined as a platelet count below 50 × 10E9 / L, at Visit 1 (measured by the central laboratory)
• Body mass index < 16 or ≥ 35 kg/m2
• Planned surgery for the initial 6 months after IMP administration in this trial
• Previous arterial or venous thrombotic event (e.g. acute myocardial infarction, cerebrovascular disease and venous thrombosis)
• Active severe infection or any other significant concurrent, uncontrolled medical condition including, but not limited to, renal, hepatic, haematological, gastrointestinal, endocrine, pulmonary, neurological, cerebral or psychiatric disease, alcoholism, drug dependency or any other psychological disorder evaluated by the investigator to interfere with adherence to the protocol procedures or with the degree of tolerance to the IMP
• Known significant medical condition including disseminated intravascular coagulation, fibrinolysis and liver fibrosis which, in the opinion of the investigator, may confound, contraindicate or limit the interpretation of either safety or efficacy data
• Known history of an allergic reaction or anaphylaxis to FIX products
• Known uncontrolled allergic conditions or allergy/hypersensitivity to any component of the IMP excipients
• Previous gene therapy treatment and/or previous participation in a gene therapy clinical trial
• Receipt of an experimental agent within 60 days prior to Visit 1
• Current participation or anticipated participation within one year after IMP administration in this trial in any other interventional clinical trial involving drugs or devices.