A Multi-Center Evaluation of the Long-Term Safety and Efficacy of Spark-sponsored Gene Therapies in Males With Hemophilia A
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 入组人数
- 29
- 试验地点
- 12
- 主要终点
- Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)
研究概览
简要总结
This long-term follow-up study will continue to evaluate the long-term safety and efficacy of SPK-8011 and SPK-8016 in males with hemophilia A, who have received a single intravenous administration of SPK-8011 or SPK-8016 in any Spark-sponsored SPK-8011 or SPK-8016 study.
详细描述
This study will follow patients with hemophilia A, who have received a single intravenous administration of SPK-8011 or SPK-8016 in any prior Spark-sponsored SPK-8011 or SPK-8016 study. Participants will be followed for up to a total of 10 years post infusion (including the time on the dosing study).
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
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
- 未提供
排除标准
- 未提供
结局指标
主要结局
Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)
时间窗: Up to 9 years
Number of Participants with Adverse Events of Special Interest (AESIs)
时间窗: Up to 9 years
Incidence of Coagulation Factor VIII (FVIII) Inhibitor Development
时间窗: Up to 9 years
Number of Participants with Clinical Laboratory Abnormalities
时间窗: Up to 9 years
Number of Participants with Vital Sign Abnormalities
时间窗: Up to 9 years
Number of Participants with Physical Exam Abnormalities
时间窗: Up to 9 years
次要结局
未报告次要终点