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Clinical Trial Summary

This is a prospective, open, multicenter, observational lead-in study,to collect prospective efficacy and safety data of current replacement therapy in adult hemophilia B patients.


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 is a prospective, open, multicenter, observational lead-in study. To evaluate the efficacy and safety of current treatment regiments in patients with moderate to severe HB (FIX:C activity ≤2%). The efficacy and safety data collected in this study may be used as baseline data for subsequent related clinical trials of VGB-R04. All subjects in this study will provide informed consent and then undergo screening assessments up to 4 weeks. All subjects will undergo 26 weeks of efficacy and safety observation .Eligible subjects will be invited to a key clinical study(phase 2 study)of VGB-R04,and then encouraged to enroll in an Long-term follow-up study to evaluate the long-term safety of VGB-R04 for a total of five years. ;


Study Design


Related Conditions & MeSH terms


NCT number NCT05442528
Study type Observational
Source Shanghai Vitalgen BioPharma Co., Ltd.
Contact Gui Cao
Phone 15156012172
Email g.cao@vitalgen.com
Status Not yet recruiting
Phase
Start date August 2022
Completion date December 2024

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