A PHASE 3, NON-INVESTIGATIONAL PRODUCT, MULTI COUNTRY COHORT STUDY TO DESCRIBE THE LONG-TERM SAFETY AND EFFECTIVENESS OF A PRIOR SINGLE-DOSE TREATMENT WITH INVESTIGATIVE GIROCTOCOGENE FITELPARVOVEC OR FIDANACOGENE ELAPARVOVEC IN PARTICIPANTS WITH HEMOPHILIA A OR HEMOPHILIA B, RESPECTIVELY
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 发起方
- Pfizer
- 入组人数
- 173
- 试验地点
- 35
- 主要终点
- Incidence of factor inhibitor development
研究概览
简要总结
A study to learn about the long-term safety and efficacy of giroctocogene fitelparvovec or fidanacogene elaparvovec in patients with hemophilia A or hemophilia B respectively, who have received treatment through prior participation in a Pfizer-sponsored clinical trial. Data collection and participant visits will be based on standard of care.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Other
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Only participants who received investigational giroctocogene fitelparvovec or fidanacogene eleparvovec and were enrolled in a Pfizer-sponsored study (C0371002, C0371003, C0371005, C3731001, C3731003) are eligible.
排除标准
- 未提供
研究组 & 干预措施
Hemophilia A / giroctocogene fitelparvovec
Participants have received treatment with giroctocogene fitelparvovec in a previous study and are not receiving any investigational product in this study
干预措施: Testing of hepatic AAV Vector integration (Diagnostic Test)
Hemophilia B / fidanacogene elaparvovec
Participants have received treatment with fidanacogene elaparvovec in a previous study and are not receiving any investigational product in this study
干预措施: Testing of hepatic AAV Vector integration (Diagnostic Test)
结局指标
主要结局
Incidence of factor inhibitor development
时间窗: Day 1 to 10 years
FIX inhibitor development was defined as an inhibitor titer \>= 0.6 Bethesda units per milliliter (BU/mL).
Incidence of liver abnormalities
时间窗: Day 1 to 10 years
Factor activity level
时间窗: Day 1 to 10 years
Factor activity level will be reported. Factor levels may be measured using different assay methods including a one-stage assay or by chromogenic substrate assay and a second one-stage assay.
Incidence of thromboembolic events
时间窗: Day 1 to 10 years
Incidence of hepatic malignancy
时间窗: Day 1 to 10 years
次要结局
- Consumption of exogenous factor (excluding infusions related to surgery)(Day 1 to 10 years)
- Incidence of Non-hepatic malignancy(Day 1 to 10 years)
- All cause mortality(Day 1 to 10 years)
- AIR of exogenous factor (excluding infusions related to surgery)(Day 1 to 10 years)
- Incidence of Auto-immune disorders(Day 1 to 10 years)
- Incidence of and time from vector infusion to resumption of prophylaxis(Day 1 to 10 years)
- EQ-5D-5L dimension and VAS scores(Day 1 to 10 years)
- Total ABR (treated or untreated; (excluding bleeds related to surgery)(Day 1 to 10 years)
- Incidence of SAEs(Day 1 to 10 years)