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Gene Therapy Trial Report

Summary

A Study to Evaluate the Safety and Efficacy of VGB-R04 in Adult Hemophilia B Patients

NCTID NCT05441553 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Hemophilia B
Disease Ontology Term DOID:12259
Compound Name VGB-R04
Sponsor Shanghai Vitalgen BioPharma Co., Ltd.
Funder Type Industry
Recruitment Status
Unknown
Enrollment Count 26 (ESTIMATED)
Results Posted Not Available

Therapy Information

Target Gene/Variant F9
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
Delivery System Viral transduction
Vector Type AAV8
Editor Type none
Dose 1 Undisclosed dose (ranging from 4E11 - 2E12 vg/kg)
Dose 2
Dose 3
Dose 4
Dose 5

Study Record Dates

Current Stage Phase1, Phase2
Submit Date 2022-06-15
Completion Date 2025-01
Last Update 2022-07-01

Participation Criteria

Eligible Age 18 Years - 65 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study MALE

Locations

No.of Trial Sites 1
Locations China

Regulatory Information

Has US IND False
FDA Designations Orphan Drug Designation
Recent Updates Granted ODD in December 2021

Resources/Links

News and Press Releases
Related NCTID