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
试验速览
- 阶段
- 不适用
- 状态
- Enrolling By Invitation
- 发起方
- bluebird bio
- 入组人数
- 85
- 试验地点
- 16
- 主要终点
- Number of subjects with immune-related AEs (e.g., autoimmune disorders, GVHD, opportunistic infections, HIV)
研究概览
简要总结
This is a multi-center, long-term safety and efficacy follow-up study for subjects with sickle cell disease who have been treated with ex vivo gene therapy drug product in bluebird bio-sponsored clinical studies. After completing the parent clinical study (approximately 2 years), eligible subjects will be followed for an additional 13 years for a total of 15 years post-drug product infusion. No investigational drug product will be administered in the study.
研究设计
- 研究类型
- Observational
- 观察模型
- Case Only
- 时间视角
- Prospective
入排标准
- 年龄范围
- 2 Years 至 53 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
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 sickle cell disease 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
Number of subjects with immune-related AEs (e.g., autoimmune disorders, GVHD, opportunistic infections, HIV)
时间窗: Through 15 years post-drug product infusion
Number of subjects with malignancies
时间窗: Through 15 years post-drug product infusion
Number of subjects with new or worsening hematologic disorders
时间窗: Through 15 years post-drug product infusion
Number of subjects with new or worsening neurologic disorders
时间窗: Through 15 years post-drug product infusion
次要结局
- Annualized number of VOEs over time through Year 15(Through 15 years post-drug product infusion)
- Assessment of HbS percentage of non-transfused total Hb over time post-drug product infusion through Year 15(Through 15 years post-drug product infusion)
- Assessment of non-HbS percentage of non-transfused total Hb over time post-drug product infusion through Year 15(Through 15 years post-drug product infusion)
- Proportion of subjects with complete resolution of severe VOEs (sVOE-CR) over time through Year 15(Through 15 years post-drug product infusion)
- Annualized number of severe VOEs over time through Year 15(Through 15 years post-drug product infusion)
- Assessment of total Hb over time post-drug product infusion through Year 15(Through 15 years post-drug product infusion)
- Assessment of non-transfused total Hb over time post-drug product infusion through Year 15(Through 15 years post-drug product infusion)
- Proportion of subjects with complete resolution of VOEs (VOE-CR) over time through Year 15(Through 15 years post-drug product infusion)
- Change from parent study baseline through Year 15 in hemolysis markers(Through 15 years post-drug product infusion)
- Change from parent study baseline in annualized number of severe VOEs over time through Year 15(Through 15 years post-drug product infusion)
- Assessment of HbAT87Q percentage of non-transfused total Hb over time post-drug product infusion through Year 15(Through 15 years post-drug product infusion)
- Change from parent study baseline through Year 15 in markers of iron stores(15 years post-drug product infusion)