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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
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