Clinical Trials List
2019-09-01 - 2027-12-31
Phase III
Recruiting3
Terminated2
ICD-10D67
Hereditary factor IX deficiency
ICD-9286.1
Congenital factor IX disorder
PHASE 3, OPEN LABEL, SINGLE ARM STUDY TO EVALUATE EFFICACY AND SAFETY OF FIX GENE TRANSFER WITH PF-06838435 (RAAV-SPARK100-HFIX-PADUA) IN ADULT MALE PARTICIPANTS WITH MODERATELY SEVERE TO SEVERE HEMOPHILIA B (FIX:C ≤ 2%) (BENEGENE-2)
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Sponsor
Pfizer
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Trial scale
Multi-Regional Multi-Center
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Update
2025/08/20
Investigators and Locations
Co-Principal Investigator
- 施銘洋 Division of Pediatrics
The Actual Total Number of Participants Enrolled
0 Recruiting
The Actual Total Number of Participants Enrolled
0 Recruiting
Co-Principal Investigator
- Pei-Chin Lin Division of Pediatrics
- 許琬宜 Division of Pediatrics
The Actual Total Number of Participants Enrolled
0 Recruiting
The Actual Total Number of Participants Enrolled
0 Terminated
Co-Principal Investigator
- 巫康熙 Division of Pediatrics
The Actual Total Number of Participants Enrolled
0 Terminated
Condition/Disease
Objectives
Test Drug
Active Ingredient
Dosage Form
Dosage
Endpoints
Annualized bleeding rate (ABR) [ Time Frame: First 12 months post PF 06838435 infusion ]
Vector derived FIX:C level [ Time Frame: Week 12 to 12 months post PF 06838435 infusion ]
Inclution Criteria
Males who completed 6 months of routine 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%)
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-AAV Spark100 neutralizing antibodies (nAb) titer >= 1:1.
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 intravenous immunoglobulin administration
History of chronic infection or other chronic disease
Any concurrent clinically significant major disease or condition
Laboratory values at screening visit that are abnormal or outside acceptable study limits
Significant and/or unstable liver disease, biliary disease, significant liver fibrosis
Planned surgical procedure requiring Factor IX surgical prophylactic factor treatment 12 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 (HBsAg), hepatitis B virus (HBV) deoxyribonucleic acid (DNA) positivity, or hepatitis C virus (HCV) ribonucleic acid (RNA) positivity
Serological evidence of human immunodeficiency virus HIV-1 or HIV-2 with Cluster of Differentiation 4 positive (CD4+) cell count ≤200 mm3 and/or viral load >20 copies/mL
Study and sponsor staff and their families involved in the conduct of the study
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
The Estimated Number of Participants
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Taiwan
8 participants
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Global
50 participants
