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Clinical Trial Report
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
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 objective of the study will evaluate the annualized bleeding rate \[ABR\] for participants treated with gene therapy versus standard of care (SOC) therapy (FIX prophylaxis replacement regimen).
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Development Status
Approved (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
Results Posted
View Results
Therapy Information
Target Gene/Variant
F9R338L
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
5E11 vg/kg
Dose 2
Dose 3
Dose 4
Dose 5
Study Record Dates
Current Stage
Phase3
Submit Date
2019-03-01
Completion Date
2031-02-25
Last Update
2025-04-17
Participation Criteria
Eligible Age
18 Years - 65 Years
Standard Ages
Adult, Older adult
Sexes Eligible for Study
MALE
Locations
No.of Trial Sites
60
Locations
United States,Japan,United Kingdom,Spain,Greece,Saudi Arabia,Canada,Sweden,Turkey,Taiwan,Brazil,Korea, Republic of,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
Clinical Publications
Field Study and Correlative Studies of Factor IX Variant FIX-R338L in Participants Treated with Fidanacogene Elaparvovec
Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant
Gene Therapy with Fidanacogene Elaparvovec in Adults with Hemophilia B
Factor IX assay discrepancies in the setting of liver gene therapy using a hyperfunctional variant factor IX-Padua
News and Press Releases
Pfizer discontinues hemophilia treatment Beqvez, emptying its gene therapy portfolio
Preclinical Publications
AAV liver expression of FIX-Padua prevents and eradicates FIX inhibitor without increasing thrombogenicity in hemophilia B dogs and mice
Protocol
Clinical Trial Protocol
FDA Approval Documents
Statistical Analysis Plan
Related NCTID
Phase 1: NCT01620801
Phase 2: NCT02484092
Phase 2: NCT03307980
Long Term Follow-Up: NCT05568719
Long Term Follow-Up: NCT06634836