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 (BENEGENE-2)
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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].
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)
Actual Study Start Date :
July 29, 2019
Estimated Primary Completion Date :
July 23, 2021
Estimated Study Completion Date :
July 21, 2026
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Layout table for eligibility information
Ages Eligible for Study:
18 Years to 65 Years (Adult, Older Adult)
Sexes Eligible for Study:
Gender Based Eligibility:
Accepts Healthy Volunteers:
Males who completed 6 months of routine Factor IX prophylaxis therapy during the lead in study (C0371004) and have > = 50 documented exposure days to a FIX protein product such as recombinant, plasma-derived or extended half-life FIX product
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
Anti-AAV Spark100 neutralizing antibodies (nAb) titer, above the established threshold of 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
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD:
Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.