Methodology Study to Investigate the Utility of Retroviral Insertion Site Analysis in Samples From Subjects Treated With Strimvelis Gene Therapy.
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 入组人数
- 15
- 试验地点
- 1
- 主要终点
- To assess the precision of S-EPTS/LM-PCR methodology for RIS analysis using control insertion site DNA
研究概览
简要总结
This study is a post approval commitment to evaluate the accuracy and precision of retroviral insertion site (RIS) analysis and its utility for investigating and predicting the potential for insertional oncogenesis in subjects treated with Strimvelis.
详细描述
This non-interventional, retrospective, methodology study will use peripheral blood and bone marrow samples previously taken from subjects treated with Strimvelis. The study does not require subjects to undergo any further treatment, intervention or blood withdrawal.
The objective of this methodology study is to evaluate the accuracy and precision of shearing extension primer tag selection ligation-mediated polymerase chain reaction (S-EPTS/LM-PCR) for RIS analysis and its utility for investigating and predicting the potential for insertional oncogenesis in subjects previously treated with Strimvelis for severe combined immunodeficiency (SCID) due to adenosine deaminase (ADA) deficiency.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Retrospective
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
结局指标
主要结局
To assess the precision of S-EPTS/LM-PCR methodology for RIS analysis using control insertion site DNA
时间窗: Retrospective sample analysis.
Precision will be determined by the variability (%CV) of the abundance data.
To assess the accuracy of S-EPTS/LM-PCR methodology for RIS using control insertion site DNA
时间窗: Retrospective sample analysis.
Accuracy will be determined based on the difference between the mean retrieved abundance and the expected abundance of control DNA.
次要结局
未报告次要终点