A Long-Term Follow-up Study to Evaluate the Safety, Tolerability, and Efficacy of Adeno-Associated Virus (AAV) rh10-Mediated Gene Transfer of Human Factor IX in Adults With Moderate/Severe to Severe Hemophilia B
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 6
- 试验地点
- 5
- 主要终点
- Incidence of adverse events and serious adverse events by dosing group
研究概览
简要总结
A long-term follow-up study to evaluate the safety, tolerability, and efficacy of DTX101 in adult males with moderate/severe to severe hemophilia B.
详细描述
Hemophilia B is an X-linked recessive genetic bleeding disorder caused by mutations in the factor IX (FIX) gene. FIX is produced in the liver and is critical for fibrin clot formation. Hemophilia B is characterized by frequent, spontaneous internal bleeding that can lead to chronic arthropathy (joint damage), intracranial hemorrhage, and even death. In patients with moderate/severe to severe hemophilia B, the majority of bleeding episodes occur in the joints and, if not treated, lead to debilitating damage and a decreased quality of life.
Study 101HEMB02 is a long-term follow-up study to evaluate the safety, tolerability, and efficacy of AAVrh10-mediated gene therapy of human FIX in subjects with moderate/severe to severe hemophilia B. The primary objective of the study is to determine the long-term safety and efficacy of DTX101 following a single IV infusion (administered during Study 101HEMB01) in adults with moderate/severe to severe hemophilia B.
This study was previously posted by Dimension Therapeutics, which has been acquired by Ultragenyx.
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
- •Willing and able to provide written informed consent.
- •Completed the Week 52 visit in Study 101HEMB
- •Willing to stop prophylactic treatment with recombinant FIX at specified time points during the study if medically acceptable.
- •Willing, able, and committed to comply with scheduled study site visits, study procedures, and requirements.
排除标准
- •Planned or current participation in another interventional clinical study that may confound the efficacy or safety evaluation of DTX101 during the duration of this study.
- •Any clinically significant medical condition that, in the opinion of the investigator, would pose a risk to subject safety or would impede the study
结局指标
主要结局
Incidence of adverse events and serious adverse events by dosing group
时间窗: 208 weeks
Change from baseline in FIX activity level
时间窗: 208 weeks
次要结局
- Number of bleeding episodes requiring recombinant FIX infusion(208 weeks)