Long-Term Follow-Up of Subjects Treated With Seattle Children's Therapeutics Gene Therapy Products
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 500
- 试验地点
- 1
- 主要终点
- Incidence of delayed adverse events
研究概览
简要总结
Subjects exposed to gene therapy products (GTPs) may be at risk for delayed or long-term adverse events. This is a long-term follow-up (LTFU) protocol designed to evaluate the long-term safety of Seattle Children's Therapeutics (SCTx) GTPs and incorporates in monitoring guidance from the U.S. Food and Drug Administration (FDA). Subjects will be followed for up to 15 years starting from the date of the most recent infusion of an SCTx GTP. Subjects planning to receive, or who have received, at least one infusion of an SCTx GTP will be offered participation in this LTFU study. Subjects enrolled in this study will have safety assessments and laboratory evaluations performed at scheduled intervals for each unique SCTx GTP received. No treatment is administered in this LTFU study.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Enrolled in an SCTx GTP clinical trial;
- •Received or planned to receive an SCTx GTP;
- •Subject and/or legally authorized representative have provided consent/assent for study participation.
排除标准
- 未提供
结局指标
主要结局
Incidence of delayed adverse events
时间窗: 15 years post-final infusion of SCTx GTP
The type, frequency, severity, and duration of adverse events considered related to the SCTx gene therapy product (GTP) the participant received.
Long-term persistence of SCTx Gene Therapy Products
时间窗: 15 years post-final infusion
Monitor for long-term persistence of SCTx GTPs.
Long-term clinical outcomes following treatment with SCTx GTP
时间窗: 15 years post-final infusion
Death from any cause and first relapse following an initial remission
次要结局
未报告次要终点
研究者
Colleen Annesley
SCTx Medical Director
Seattle Children's Hospital