None listed
Conditions
Interventions
Drug, Genetic : The study treatment, a single infusion of PF 06838435, a gene transfer agent, will be administered on Day 1 at a dose of 5 × 1011 vector genomes/kg of body weight. The safety and effi
Sponsors
Kyung Hee University Hospital at Gangdong
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: 1. Participants must have completed at least 6 months of routine FIX prophylaxis therapy during the lead in study (C0371004) prior to providing consent at the screening visit for this study. 2. Participants who have documented moderately severe to severe hemophilia B, defined as FIX:C ≤2%. 3. Participants must agree to suspend prophylaxis therapy for hemophilia B after administration of the IP. FIX replacement therapy is allowed as needed (see Section 6.5.1). 4. Acceptable screening laboratory values as follows: • Hemoglobin ≥11 g/dL; • Platelets ≥100,000 cells/µL; • Creatinine ≤2.0 mg/dL. Sex 5. Male. Contraceptive use by men should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies. Male participants are eligible to participate if they agree to the following for at least time required for 3 consecutive ejaculate samples to test negative for vector shedding: • Refrain from donating sperm. PLUS either: • Be abstinent from heterosexual or homosexual intercourse as their preferred and usual lifestyle (abstinent on a long term and persistent basis) and agree to remain abstinent. OR • Must agree to use contraception/barrier as detailed below: • Agree to use male condom when engaging in any activity that allows for passage of ejaculate to another person Informed Consent 6. Capable of giving signed informed consent as described in Appendix 1 which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol.
Exclusion criteria
Exclusion criteria: 1. Anti AAV Spark100 neutralizing antibodies (nAb) titer ≥1:1 (ie, positive for nAb), performed by a central laboratory during screening. 2. Prior history of inhibitor to FIX or positive inhibitor testing as measured by the central laboratory ≥0.6 Bethesda Units (BU) during screening. Clinical signs or symptoms of decreased response to FIX. 3. Known hypersensitivity to FIX replacement product or intravenous immunoglobulin administration. 4. History of chronic infection or other chronic disease that investigator deems as an unacceptable risk. 5. Any concurrent clinically significant major disease or condition that the investigator deems unsuitable for participation or other acute or chronic medical or psychiatric condition including recent (within the past year) or active suicidal ideation or behavior (including alcoholism) or laboratory abnormality that may increase the risk associated with study participation or may interfere with the interpretation of study results and, in the judgment of the investigator, would make the participant inappropriate for entry into this study. 6. Alanine transaminase (ALT), aspartate aminotransferase (AST), alkaline phosphatase (ALP) >2 × upper limit of normal (ULN), based on central laboratory results. 7. Bilirubin >1.5 × ULN (isolated bilirubin >1.5 × ULN is acceptable if bilirubin is fractionated and direct bilirubin 8 kPa units, Fibro Test/FibroSURE >0.48* or AST to Platelet Ratio Index (APRI) >1. In the investigator’s opinion, if there is concern regarding the FibroTest results due to a confounding medical history (eg, proteinuria can impact FibroTest result), the investigator can perform a different assessment of liver fibrosis (eg, FibroScan or APRI) during the screening period. *Please note: if a participan
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Non inferiority on annualized bleeding rate (ABR) for total bleeds (treated and untreated) from Week 12 to Month 15 versus standard of care (SOC) FIX prophylaxis replacement regimen, comparing pre-and post-IP infusion. | — |
Secondary
| Measure | Time frame |
|---|---|
| Non-inferiority on ABR for treated bleeds from Week 12 to Month 15 versus SOC FIX prophylaxis replacement regimen, comparing pre- and post-IP infusion. | — |
Countries
Korea, Republic of
Contacts
Public ContactHaim Choi
Kyung Hee University Hospital at Gangdong
Outcome results
None listed