Long-term Follow-up of Subjects With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 入组人数
- 64
- 试验地点
- 23
- 主要终点
- Number of participants with new or worsening neurologic disorders
研究概览
简要总结
This is a multi-center, long-term safety and efficacy follow-up study for participants with cerebral adrenoleukodystrophy (CALD) who have received Lenti-D Drug Product (eli-cel) in a parent clinical study (Study ALD-102 or Study ALD-104).
After completing a parent clinical study (approximately 2 years), eligible participants 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 this 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
- — 至 19 Years（Child, Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Provision of written informed consent for this study by the participant or participant's parent(s)/ legal guardian(s) and written informed assent by participant, if applicable
- •Have received eli-cel in a parent clinical study
排除标准
- •There are no exclusion criteria for this study
研究组 & 干预措施
Long-term followup
Participants who have received Lenti-D Drug Product in a parent clinical study (bluebird bio-sponsored clinical studies ALD-102 and ALD-104) and who meet the eligibility criteria for the Study LTF-304 will be followed in this long-term followup study for 13 years (for a total of 15 years of follow-up after drug product infusion in the parent studies).
干预措施: No interventional drug product utilized in this follow-up study (Genetic)
结局指标
主要结局
Number of participants with new or worsening neurologic disorders
时间窗: 15 years post-drug-product infusion
Major functional disability (MFD)-free survival
时间窗: 15 years post-drug-product infusion
The MFDs are loss of communication, cortical blindness, tube feeding, total incontinence, wheelchair dependence, complete loss of voluntary movement.
Number of participants with malignancies
时间窗: 15 years post-drug-product infusion
Number of participants who experience graft versus host disease (GVHD)
时间窗: 15 years post-drug-product infusion
Number of participants with new or worsening hematologic disorders
时间窗: 15 years post-drug-product infusion
Number of participants with immune-related adverse events (AEs)
时间窗: 15 years post-drug-product infusion
次要结局
- Number of participants who undergo subsequent stem cell transplantation(15 years post-drug-product infusion)
- Change from baseline in neurological function score (NFS)(15 years post-drug-product infusion)
- Number of participants without gadolinium enhancement (GdE) status on magnetic resonance imaging (MRI)(15 years post-drug-product infusion)