A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B
- Conditions
- Hemophilia B
- Registration Number
- NCT03861273
- Lead Sponsor
- Pfizer
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Active, not recruiting
- Sex
- Male
- Target Recruitment
- Not specified
Inclusion Criteria<br><br> - Males who completed 6 months of Factor IX prophylaxis therapy during the lead-in<br> study (C0371004) prior to providing consent at the screening visit for this study.<br><br> - Documented moderately severe to severe hemophilia B (Factor IX activity < =2%)<br><br> - Previous experience with FIX therapy (=>50 documented exposure days to a FIX protein<br> product)<br><br> - Suspension of prophylaxis therapy for hemophilia B after administration of the study<br> drug<br><br> - Laboratory values (hemoglobin, platelets and creatinine) within study specified<br> limits<br><br> - Agree to contraception until components of the drug are eliminated from their body<br><br> - Capable of giving signed informed consent<br><br>Exclusion Criteria<br><br> - Anti-AAVRh74var neutralizing antibodies (nAb) titer above the established threshold<br> (ie, positive for nAb).<br><br> - History of inhibitor to Factor IX or inhibitor detected during screening. Clinical<br> signs or symptoms of decreased response to Factor IX<br><br> - Hypersensitivity to Factor IX replacement product or IV immunoglobulin<br> administration<br><br> - History of chronic infection or other chronic disease<br><br> - Any conditions associated with increased thromboembolic risk<br><br> - Concurrent clinically significant major disease or condition unsuitable for<br> participation and/or may interfere with the interpretation of study results<br><br> - Laboratory values at screening visit that are abnormal or outside acceptable study<br> limits<br><br> - Current unstable liver or biliary disease<br><br> - Currently on antiviral therapy for hepatitis B or C<br><br> - Planned surgical procedure requiring Factor IX surgical prophylactic factor<br> treatment 15 months from screening visit<br><br> - Use of restricted therapies (e.g., blood products, acetylsalicylic acid [aspirin] or<br> ibuprofen, other investigational therapy, and by-passing agents)<br><br> - Previously dosed in a gene therapy research trial at any time or in an<br> interventional clinical study within 12 weeks of screening visit<br><br> - Active hepatitis B or C; hepatitis B surface antigen, hepatitis B virus<br> deoxyribonucleic acid positivity, or hepatitis C virus ribonucleic acid positivity<br><br> - Significant liver disease<br><br> - Serological evidence of HIV1 or HIV2 infection with either CD4+ cell count <=200 mm3<br> and/or a viral load >20 copies/mL<br><br> - Study and sponsor staff involved in the conduct of the study and their families<br><br> - Unable to comply with study procedures<br><br> - Sensitivity to heparin or heparin induced thrombocytopenia<br><br> - Sensitivity to any of the study interventions, or components thereof, or drug or<br> other allergy
Not provided
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Annualized Bleeding Rate (ABR) for Total Bleeds (Treated and Untreated) From Week 12 to Month 15
- Secondary Outcome Measures
Name Time Method