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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.
Full 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.
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Inclusion criteria
Male ≥18 years and ≤65years of age;
Confirmed diagnosis of hemophilia B (baseline FIX activity ≤ 2% of normal);
At least 50 days exposure history to FIX;
Currently receiving FIX Prophylaxis therapy or on-demand treatment to prevent bleeding;
Have acceptable laboratory values:
No measurable factor IX inhibitor as assessed by the central laboratory and have no prior history of inhibitors to factor IX protein;
Exclusion criteria
Have significant underlying liver disease within the past 6 months prior to or at Screening, including but not limited to:
Have anti-VGB-R04 neutralizing antibody titers ≥1:5;
Evidence of severe infection disease, i.e., human immunodeficiency virus (HIV) infection, syphilis, tuberculosis, etc.;
Novel coronavirus infection occurred in the 6 weeks prior to entry into the group
Evidence of active hepatitis B virus infection (HBsAg positive) or hepatitis C virus infection (HCV-RNA positive);
Evidence of malignant tumours or those with a previous history of malignant tumours;
Have a history of chronic infection or other chronic diseases that the Investigator considers to constitute an unacceptable risk;
Any immunodeficiency;
planned surgery may be required within one year;
Past thromboembolic events (arterial or venous thromboembolic events);
Hypertensive patients with poor blood pressure control (systolic blood pressure ≥150 mmHg or diastolic blood pressure ≥90mmHg after antihypertensive drug treatment);
20 participants in 1 patient group
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Central trial contact
Gui Cao
Data sourced from clinicaltrials.gov
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