Contact SCGE




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
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