Screening/Observational Protocol to Determine Patient Eligibility for Inclusion in AAV Gene Therapy Clinical Trials (ECLIPSE)
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 37
- 试验地点
- 56
- 主要终点
- Factor IX replacement therapy consumption
研究概览
简要总结
Freeline is developing adeno-associated virus (AAV) vector based gene therapies for a number of diseases and is actively advancing a programme in Haemophilia B (HB). This study aims to collect prospective data to characterise bleeding events and Factor IX (FIX) concentrate consumption in HB patients that can be used as baseline for participants who elect to participate in a subsequent Freeline gene therapy study. The study will also screen participants for antibodies to a novel AAV vector to assess their suitability for inclusion in a Freeline gene therapy treatment study.
详细描述
This is a prospective study to collect baseline disease characteristics in patients with HB that are required to establish eligibility for treatment with a novel AAV gene therapy in a subsequent Freeline gene therapy treatment study.
Participants providing consent will attend an enrolment visit to complete eligibility evaluations, collect health and demographic information and receive instruction for completing the study diary. Participants will then complete a diary recording bleeding episodes and FIX usage until they enrol into a gene therapy treatment study. A blood sample will be drawn at a convenient timepoint during the study to assess the participant's AAV neutralising antibody (NAb) status.
No treatment intervention will occur as part of this study.
研究设计
- 研究类型
- Observational
- 观察模型
- Case Only
- 时间视角
- Other
入排标准
- 年龄范围
- 16 Years 至 —（Child, 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
- •Documented evidence of liver fibrosis and/ or liver dysfunction
- •Prior treatment with a gene transfer medicinal product.
- •Known presence or history of neutralising anti-human FIX antibodies (inhibitors)
- •Previously established serological evidence of HIV-1
- •Documented active hepatitis B or C, and HBsAg or HCV RNA viral load positivity, respectively, or currently on antiviral therapy for hepatitis B or C
- •Participants at high risk of thromboembolic events (history of arterial or venous thromboembolism
- •Known coagulation disorder other than Haemophilia B
- •Known history of an allergic reaction or anaphylaxis to Factor IX products or known uncontrolled allergic conditions
- •Known history of allergy to corticosteroids or to tacrolimus or any other macrolide
- •Known medical condition that would require chronic administration of corticosteroids (excluding topical formulations)
- •History of alcohol or drug dependence.
- •Planned surgical procedure within the next 12 months requiring prophylactic FIX treatment.
- •Known active severe infection (including documented COVID-19 infection), or any other significant concurrent, uncontrolled medical condition evaluated by the investigator to interfere with adherence to the protocol procedures or with tolerance to gene therapy in a future treatment study including, but not limited to, renal, hepatic, cardiovascular, opthalmological, hematological, immunological, gastrointestinal, endocrine, pulmonary, neurological, cerebral or psychiatric disease, malignancy or any other psychological disorder.
结局指标
主要结局
Factor IX replacement therapy consumption
时间窗: 6 - 24 months
Factor IX replacement therapy data recorded during the study
Bleeding episodes
时间窗: 6 - 24 months
Bleeding episode data recorded during the study
次要结局
- To characterise baseline clinical parameters related to Haemophilia B.(6 - 24 months)
- To screen Haemophilia B patients for neutralising antibodies to a novel AAV vector (AAVS3).(6 - 24 months)