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Gene Therapy Trial Browser
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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Indication
Hemophilia B
Compound Name
BEQVEZ/fidanacogene elaparvovec
Sponsor
Pfizer
Funder Type
Industry
Status
Active not recruiting
Enrollment Count
51
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
2031-01-09
Last Update
2024-12-03
Participation Criteria
Eligible Age
18 Years - 65 Years
Standard Ages
Adult, Older adult
Eligible Sex
MALE
Locations
No.of Trial Sites
64
Locations
United States,Japan,United Kingdom,Spain,Greece,Saudi Arabia,Canada,Sweden,Turkey,Belgium,Taiwan,Brazil,Korea, Republic of,Italy,Australia,France,Germany
Regulatory Information
Has US IND
True
Recent Updates
FDA approved 4/25/24, Price/treatment $3.5M
Resources/Links
Clinical Publications
Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant
Factor IX assay discrepancies in the setting of liver gene therapy using a hyperfunctional variant factor IX-Padua
Gene Therapy with Fidanacogene Elaparvovec in Adults with Hemophilia B
Field Study and Correlative Studies of Factor IX Variant FIX-R338L in Participants Treated with Fidanacogene Elaparvovec
Preclinical Publications
AAV liver expression of FIX-Padua prevents and eradicates FIX inhibitor without increasing thrombogenicity in hemophilia B dogs and mice
Protocol
Statistical Analysis Plan
FDA Approval Documents
Clinical Trial Protocol