NCT03876301已完成不适用
A Multi-Center, Observational Study in Males With Hemophilia A
适应症
干预措施
相关药物
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 25
- 试验地点
- 11
- 主要终点
- Number of bleeding events, annualized
研究概览
简要总结
The aim of this prospective, observational study is to establish a dataset on the frequency of bleeding events, as well as other characteristics of bleeding events and FVIII infusions, in patients with clinically severe hemophilia A receiving prophylactic FVIII replacement therapy as standard of care. The data collected from this study may assist in providing baseline information for comparison to the Spark's investigational hemophilia A gene therapy in future Phase 3 studies.
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
- •Able and willing to provide written informed consent.
- •Males ≥18 years of age.
- •Clinically severe hemophilia A
- •Previous exposure to FVIII therapy
- •No prior history of hypersensitivity or anaphylaxis associated with an FVIII or intravenous immunoglobulin administration.
- •No measurable inhibitor against FVIII
- •Willing to participate and receive treatment in a future Spark hemophilia A gene therapy study.
排除标准
- •Documented active hepatitis B or C within the past 12 months of Screening
- •Currently on antiviral therapy to treat hepatitis B or C;
- •Documented significant liver disease within the past 6 months of Screening
- •Have serological evidence of HIV-1 or HIV-2
- •Anti-AAV-Spark 200 neutralizing titers ≥1:1
- •Previously received SPK-8011;
- •Previously dosed with any investigational or approved gene therapy product at any time or treated with an investigational drug within the last 12 weeks;
- •Planned surgical procedure in the next 12 months requiring FVIII prophylactic treatment.
- •Any history of chronic infection or other chronic disease, concurrent clinically significant major disease (such as liver abnormalities or type I diabetes) including active malignancy, except for non-melanoma skin cancer, any other condition or any other unspecified reasons that, in opinion of the Investigator or Sponsor, makes the participant unsuitable for participation and dosing in a future clinical study for Spark's hemophilia A gene therapy.
- •Unable or unwilling to comply with the schedule of visits and/or study assessments described in the protocol.
研究组 & 干预措施
Observational Cohort
Adult males with clinically severe hemophilia A, who are negative for neutralizing antibody (NAb) to AAV-Spark200
干预措施: Standard of Care FVIII Replacement therapy (Drug)
结局指标
主要结局
Number of bleeding events, annualized
时间窗: 12 months
Annualized bleeding rate (ABR)
次要结局
- Dose and total FVIIII consumption(12 months)
- Annualized number of infusions (AIR)(12 months)
研究者
研究点 (11)
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