Status:
ACTIVE_NOT_RECRUITING
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
Lead Sponsor:
Pfizer
Conditions:
Hemophilia B
Eligibility:
MALE
18-65 years
Phase:
PHASE3
Brief Summary
This study will evaluate the efficacy and safety of PF-06838435 (a gene therapy drug) in adult male participants with moderately severe to severe hemophilia B (participants that have a Factor IX circu...
Eligibility Criteria
Inclusion
- Inclusion Criteria
- Males who completed 6 months of Factor IX prophylaxis therapy during the lead-in study (C0371004) prior to providing consent at the screening visit for this study.
- Documented moderately severe to severe hemophilia B (Factor IX activity \< =2%)
- Previous experience with FIX therapy (=\>50 documented exposure days to a FIX protein product)
- Suspension of prophylaxis therapy for hemophilia B after administration of the study drug
- Laboratory values (hemoglobin, platelets and creatinine) within study specified limits
- Agree to contraception until components of the drug are eliminated from their body
- Capable of giving signed informed consent
- Exclusion Criteria
- Anti-AAVRh74var neutralizing antibodies (nAb) titer above the established threshold (ie, positive for nAb).
- History of inhibitor to Factor IX or inhibitor detected during screening. Clinical signs or symptoms of decreased response to Factor IX
- Hypersensitivity to Factor IX replacement product or IV immunoglobulin administration
- History of chronic infection or other chronic disease
- Any conditions associated with increased thromboembolic risk
- Concurrent clinically significant major disease or condition unsuitable for participation and/or may interfere with the interpretation of study results
- Laboratory values at screening visit that are abnormal or outside acceptable study limits
- Current unstable liver or biliary disease
- Currently on antiviral therapy for hepatitis B or C
- Planned surgical procedure requiring Factor IX surgical prophylactic factor treatment 15 months from screening visit
- Use of restricted therapies (e.g., blood products, acetylsalicylic acid \[aspirin\] or ibuprofen, other investigational therapy, and by-passing agents)
- Previously dosed in a gene therapy research trial at any time or in an interventional clinical study within 12 weeks of screening visit
- Active hepatitis B or C; hepatitis B surface antigen, hepatitis B virus deoxyribonucleic acid positivity, or hepatitis C virus ribonucleic acid positivity
- Significant liver disease
- Serological evidence of HIV1 or HIV2 infection with either CD4+ cell count \<=200 mm3 and/or a viral load \>20 copies/mL
- Study and sponsor staff involved in the conduct of the study and their families
- Unable to comply with study procedures
- Sensitivity to heparin or heparin induced thrombocytopenia
- Sensitivity to any of the study interventions, or components thereof, or drug or other allergy
Exclusion
Key Trial Info
Start Date :
July 29 2019
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
February 25 2031
Estimated Enrollment :
51 Patients enrolled
Trial Details
Trial ID
NCT03861273
Start Date
July 29 2019
End Date
February 25 2031
Last Update
September 30 2025
Active Locations (60)
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1
University of California, San Francisco - Clinical Research Center
San Francisco, California, United States, 94143
2
University of California, San Francisco - Outpatient Hematology Clinic
San Francisco, California, United States, 94143
3
Hemophilia and Thrombosis Center at the University of Colorado Anschutz Medical Campus
Aurora, Colorado, United States, 80045
4
Indiana Hemophilia & Thrombosis Center, Inc.
Indianapolis, Indiana, United States, 46260