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Clinical Trial Summary

An Open-Label, Non-Randomized, uncontrolled, single-dose pilot study of VGB-R04 in subjects with Hemophilia B.


Clinical Trial Description

Hemophilia B is a genetic bleeding disorder caused by pathogenic variants (eg, mutations, deletion) in the FIX gene. HB patients have frequent and potentially life-threatening bleeding and often develop progressive physical disability and pain from chronic haemarthropathy. Current replacement therapy needs regular treatment in the life-long time, bringing heavy economic and social burdens. VGB-R04 is a novel AAV vector carrying a high specific activity factor IX variant. This study is intended to evaluate the safety, tolerability and kinetics of a single IV infusion of VGB-R04. All subjects in this study will provide informed consent and then undergo screening assessments up to 6 weeks before administration of VGB-R04. All subjects will undergo 52(±2) weeks of safety observation and will be encouraged to enroll in an extension study to evaluate the long-term safety of VGB-R04 for a total of five years. ;


Study Design


Related Conditions & MeSH terms


NCT number NCT05152732
Study type Interventional
Source Institute of Hematology & Blood Diseases Hospital
Contact Lei Zhang, Doctor
Phone +86-13502118379
Email zhanglei1@ihcams.as.cn
Status Recruiting
Phase Early Phase 1
Start date December 28, 2021
Completion date December 2025

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