Congenital Athymia Patient Registry of RETHYMIC
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 75
- 试验地点
- 1
- 主要终点
- Flow cytometry including total and naïve CD3, CD4, and CD8 counts
研究概览
简要总结
This Congenital Athymia Patient Registry is an observational exposure-based registry study. It uses a prospective cohort design to follow patients who have been treated with RETHYMIC.
Clinical studies conducted with investigational RETHYMIC showed that treatment can result in immune reconstitution and prolong life. This treatment-based registry is being conducted to learn more about the reconstitution process following treatment and the impact of treatment on longer-term survival and the occurrence of adverse events of special interest (AESI).
详细描述
This Congenital Athymia Patient Registry is an observational exposure-based registry study. It uses a prospective cohort design to follow patients who have been treated with RETHYMIC.
Clinical studies conducted with investigational RETHYMIC showed that treatment can result in immune reconstitution and prolong life. This treatment-based registry is being conducted to learn more about the reconstitution process following treatment and the impact of treatment on longer-term survival and the occurrence of adverse events of special interest (AESI).
Currently, there is a single site where patients are being treated with RETHYMIC. The Investigator will be responsible for recruiting patients who have recently had (i.e., within the past 60 days) or are scheduled to have this treatment within 30 days and obtaining signed informed consent (and assent as applicable). The Investigator will collect baseline medical history and clinical data information for each enrolled subject. The start of follow-up (day 1) begins on the day following treatment surgery. Clinical data and flow cytometry test results (when conducted) will be abstracted from the medical records by Clinical Staff at baseline and during follow-up at predefined intervals.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Other
入排标准
- 年龄范围
- 0 Years 至 21 Years(Child, Adult)
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Pediatric patients diagnosed with Congenital Athymia:
- •Who are scheduled for implantation with RETHYMIC within the next 30 days, or who were treated with RETHYMIC within the previous 60 days.
- •Who have provided written informed consent directly, or written informed consent has been provided by the patient, the parent, or legal guardian.
排除标准
- •Written informed consent cannot be obtained.
研究组 & 干预措施
Main cohort
All eligible patients
干预措施: Cultured Thymus Tissue (Biological)
Secondary cohort
All eligible patients who survived 1 year post treatment
干预措施: Cultured Thymus Tissue (Biological)
结局指标
主要结局
Flow cytometry including total and naïve CD3, CD4, and CD8 counts
时间窗: Immediately after the intervention/procedure/surgery
Vital Status
时间窗: 12 months post treatment with RETHYMIC.
次要结局
- AESI classified by MedDRA coding, severity and grade(Immediately after the intervention/procedure/surgery)
