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Gene Therapy Trial Report

Summary

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


NCTID NCT03861273 (View at clinicaltrials.gov)
Description
Development Status Marketing Discontinued
Indication Hemophilia B
Disease Ontology Term DOID:12259
Compound Name BEQVEZ
Compound Alias fidanacogene elaparvovec, PF-06838435
Sponsor Pfizer
Funder Type Industry
Recruitment Status
Active not recruiting
Enrollment Count 51 (ACTUAL)
Results Posted View Results

Therapy Information


Target Gene/Variant F9-R338L
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Functional gene replacement
Route of Administration Intravenous
Drug Product Type Viral vector
Target Tissue/Cell Liver
Delivery System Viral transduction
Vector Type AAVrh74
Editor Type none
Dose 1 Phase 2: 5E11 vg/kg (n=15)
Dose 2 Phase 3: 5E11 vg/kg (n=45)
Dose 3 Approved dose: 5E11 vg/kg

Study Record Dates


Current Stage Phase3
Submit Date 2019-03-01
Completion Date 2031-02-25
Last Update 🔄 2026-03-16

Participation Criteria


Eligible Age 18 Years - 65 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study MALE
Eligibility Criteria
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
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 🔄 65
Locations United States,Japan,United Kingdom,Spain,Greece,Saudi Arabia,Canada,South Korea,Sweden,Taiwan,Turkey (Türkiye),Brazil,Italy,Australia,France,Germany

Regulatory Information


Has US IND True
FDA Designations Breakthrough Therapy, Orphan Drug Designation, Regenerative Medicine Advanced Therapy
Recent Updates FDA approved 4/25/24, Price/treatment $3.5M; Pfizer will no longer market Beqvez

Resources/Links