An Observational Long-term Safety and Efficacy Follow-up Study After Ex-vivo Gene Therapy With BIVV003 in Participants With Severe Sickle Cell Disease (SCD) or With ST-400 in Participants With Transfusion-dependent Beta-thalassemia (TDT) With Autologous Hematopoietic Stem Cell Transplant
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 入组人数
- 8
- 试验地点
- 7
- 主要终点
- Adverse Events
研究概览
简要总结
Primary Objectives:
Long-term safety of BIVV003 in participants with severe sickle cell disease (SCD) and ST- 400 in participants with transfusion-dependent beta-thalassemia (TDT)
Secondary Objectives:
- Long-term efficacy of the biological treatment effect of BIVV003 in SCD
- Long-term efficacy of the clinical treatment effect of BIVV003 on SCD-related clinical events
- Long-term efficacy of the biological treatment effect of ST-400 in TDT
- Long-term efficacy of the clinical treatment effect of ST-400 in TDT
详细描述
The total study duration is up to 15 years of follow-up post BIVV003 and/or ST-400 infusion.
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 45 Years（Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Received treatment with BIVV003 or ST-400 in one of the parent studies (ACT16222, ST- 400-01) or any future studies with BIVV003
- •Capable of giving signed informed consent (and if applicable assent)
排除标准
- •Unable to comply with study visit schedule or study procedures
- •Any other reason that, in the opinion of the Investigator or Medical Monitor, would render the participant unsuitable for participation in the study The above information is not intended to contain all considerations relevant to a potential participation in a clinical trial.
研究组 & 干预措施
BIVV003 Cohort
All participants treated in parent and future studies with BIVV003
干预措施: BIVV003 (Drug)
ST-400 Cohort
All participants treated in parent studies with ST-400
干预措施: ST-400 (Drug)
结局指标
主要结局
Adverse Events
时间窗: Up to 15 years
Number of participants with serious adverse events and adverse events related to BIVV003 or ST-400, including new malignancy, new incidence or exacerbation of neurologic or rheumatologic or autoimmune or hematologic disorder, or new incidence of infection potentially related to BIVV003 or ST-400
Overall Survival
时间窗: Up to 15 years
Duration from first dose of study medication to death
次要结局
- Change in hemoglobin levels(Up to 15 years)
- Change in hemolysis markers(Up to 15 years)
- Frequency of severe vaso-occlusive crises(Up to 15 years)
- Frequency and severity of SCD-related clinical events(Up to 15 years)
- Red blood cell transfusions(Up to 15 years)