Contact SCGE
Your email
Message
Send
SCGE Consortium Home
|
About SCGE TCDC
|
Contact Us
|
License
Home
Gene Therapy Trial Browser
Clinical Trial Report
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
A multicenter, open, non-randomized, phase I/II, two-phase clinical study. The dose exploration phase was phase I, and the dose extension phase was phase II.
(Show More)
Indication
Hemophilia B
Compound Name
VGB-R04
Sponsor
Shanghai Vitalgen BioPharma Co., Ltd.
Funder Type
Industry
Status
Not yet recruiting
Enrollment Count
26
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
Dose 1
Undisclosed dose (ranging from 4x10^11 - 2x10^12 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
Eligible Sex
MALE
Locations
No.of Trial Sites
1
Locations
China
Regulatory Information
Has US IND
False
Recent Updates
Granted ODD in December 2021
Resources/Links
Patents
WO2023280323A1
WO2023072181A1