Six-month lead-in Study to Collect Prospective Efficacy and Safety Data of Current Replacement Therapy in Adult Hemophilia B Patients -- An Observational Survey Analysis Study
试验速览
- 阶段
- 不适用
- 状态
- 尚未招募
- 入组人数
- 20
- 主要终点
- Annualized bleeding rate changes from baseline
研究概览
简要总结
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.
详细描述
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.
研究设计
- 研究类型
- Observational
- 观察模型
- Case Only
- 时间视角
- Prospective
入排标准
- 年龄范围
- 18 Years 至 65 Years（Adult, Older Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •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:
- •Hemoglobin ≥110 g/L;
- •Platelets ≥100×109 /L;
- •AST, ALT, alkaline phosphatase ≤2×upper limit of normal (ULN) at the testing laboratory;
- •Bilirubin ≤3× ULN ;
- •Creatinine ≤1.5× ULN.
- •No measurable factor IX inhibitor as assessed by the central laboratory and have no prior history of inhibitors to factor IX protein;
排除标准
- •Have significant underlying liver disease within the past 6 months prior to or at Screening, including but not limited to:
- •Preexisting diagnosis of portal hypertension;
- •Splenomegaly;
- •Encephalopathy;
- •Reduction of serum albumin;
- •Evidence of significant liver fibrosis;
- •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);
结局指标
主要结局
Annualized bleeding rate changes from baseline
时间窗: Baseline up to Week 26
The number of bleeding episodes per participant will be recorded, and the annualized number of bleeding episodes.
次要结局
- Total number of annualized bleeding(Baseline up to Week 26)
- Annualized FIX consumption changes from baseline(Baseline up to Week 26)
- FIX:C activity level.(Baseline up to Week 26)
- Number of target joints(Baseline up to Week 26)
- The incidence of adverse Events of special interest(Baseline up to Week 26)