2024-513901-30-00招募中3 期
Long-term Follow-up of Subjects With Sickle Cell Disease Treated With Ex Vivo Gene Therapy Using Autologous Hematopoietic Stem Cells Transduced With a Lentiviral Vector
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 3
- 试验地点
- 1
- 主要终点
- The number of subjects with immune-related AEs (e.g., autoimmune disorders, graft-versus-host disease, opportunistic infections, HIV)
研究概览
简要总结
- Evaluate long-term safety of treatment with lovocel (lovotibeglogene autotemcel, also known as bb1111 or LentiGlobin BB305 Drug Product for Sickle Cell Disease) in subjects with sickle cell disease (SCD)
- Evaluate long-term efficacy of treatment with bb1111 in subjects with SCD
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 0 years 至 64 years（0-17 Years, 18-64 Years）
- 接受健康志愿者
- 是
入选标准
- •Provision of written informed consent for this study by subject, or as applicable, subject's parent(s)/ legal guardian(s)
- •Treated with drug product for therapy of SCD in a bluebird bio-sponsored clinical study
排除标准
- •There are no exclusion criteria for this study.
结局指标
主要结局
The number of subjects with immune-related AEs (e.g., autoimmune disorders, graft-versus-host disease, opportunistic infections, HIV)
The number of subjects with immune-related AEs (e.g., autoimmune disorders, graft-versus-host disease, opportunistic infections, HIV)
The number of subjects with new or worsening hematologic disorders
The number of subjects with new or worsening hematologic disorders
The number of subjects with new or worsening neurologic disorders
The number of subjects with new or worsening neurologic disorders
The number of subjects with malignancies
The number of subjects with malignancies
次要结局
- Vaso-Occlusive Events Endpoints: The proportion of subjects with complete resolution of severe VOEs (sVOE-CR) over time through Year 15
- The proportion of subjects with complete resolution of VOEs (VOE-CR) over time through Year 15
- Annualized number of severe VOEs over time through Year 15
- Annualized number of VOEs over time through Year 15
- Change from parent study baseline in annualized number of severe VOEs over time through Year 15
- Hematologic Endpoints: Assessment of the following over time post-drug product infusion through Year 15: − total Hb − non-transfused total Hb − HbS percentage of non-transfused total Hb − HbAT87Q percentage of non-transfused total Hb − non-HbS percentage of non-transfused total Hb
- Change from parent study baseline through Year 15 in the following hemolysis markers: absolute reticulocyte count, % reticulocytes/erythrocytes, total bilirubin, indirect bilirubin, haptoglobin, and lactate dehydrogenase
- Change from parent study baseline through Year 15 in the following markers of iron stores: serum ferritin and liver iron content
研究者
Clinical Trial Information
Scientific
Bluebird Bio Inc.
研究点 (1)
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