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
a study on Hemophilia
Summary
- Eligibility
- for males ages 18-65 (full criteria)
- Location
- at San Francisco, California and other locations
- Dates
- study startedestimated completion
Description
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 circulating activity of 2% or less). The gene therapy is designed to introduce genetic material into cells to compensate for missing or non-functioning Factor IX. Eligible study participants will have completed a minimum 6 months of routine Factor IX prophylaxis therapy during the lead in study (C0371004). Participants will be dosed once (intravenously) and will be evaluated over the course of 6 years. The main objectives of the study are to compare the annualized bleeding rate [ABR] of the gene therapy to routine prophylaxis from the lead-in study and to evaluate the impact that it may have on participant's Factor IX circulating activity [FIX:C].
Official Title
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)
Keywords
Hemophilia B PF 06838435 Hemophilia Factor IX FIX Gene Therapy Infusion rAAV Spark100 hFIX Padua BeneGene 2 Hematological Diseases Annualized bleeding rate (ABR) Hemophilia A PF-06838435/ fidanacogene elaparvovec
Eligibility
You can join if…
Open to males ages 18-65
- 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
You CAN'T join if...
- 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
Locations
- University of California, San Francisco - Investigational Drug Service (IDS) Pharmacy
accepting new patients
San Francisco California 94143-0622 United States - University of California, San Francisco - Clinical Research Center
accepting new patients
San Francisco California 94143 United States - University of California, San Francisco - Outpatient Hematology Clinic
accepting new patients
San Francisco California 94143 United States
Details
- Status
- accepting new patients
- Start Date
- Completion Date
- (estimated)
- Sponsor
- Pfizer
- Links
- To obtain contact information for a study center near you, click here.
- ID
- NCT03861273
- Phase
- Phase 3
- Study Type
- Interventional
- Last Updated
Frequently Asked Questions
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If you do not hear from the study team, please call 888-689-8273 and tell them you’re interested in study number NCT03861273.