A Phase I Study of Autologous Mesenchymal Stromal Cell Coated Fistula Plug in Patients With Fistulizing Crohn's Disease: Pediatric Sub-study
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- Mayo Clinic
- 入组人数
- 1
- 试验地点
- 1
- 主要终点
- Number of participants with treatment-related adverse events (safety and toxicity)
研究概览
简要总结
The purpose of this study is to determine the safety of using an autologous mesenchymal stromal cell (MSC) coated fistula plug in people with fistulizing Crohn's disease. Autologous means these cells to coat the plug come from the patient.
详细描述
The investigators propose to study the safety of autologous mesenchymal stromal cell transfer using a biomatrix (the Gore Bio-A Fistula Plug) in a Phase I study using a single dose of 20 million cells. 5 patients (age 12 to 17 years) with Crohn's perianal fistulas will be enrolled.
Subjects will undergo standard adjuvant therapy including drainage of infection and placement of a draining seton. Six weeks post placement of the draining seton, the seton will be replaced with the MSC loaded Gore fistula plug as per current clinical practice. The subjects will be subsequently followed for fistula response and closure for 24 months. This is an autologous product derived from the patient and used only for the same patient.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 12 Years 至 17 Years（Child）
- 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
研究组 & 干预措施
MSC-AFP
This is a single treatment group study. All patients will receive treatment of a stem cell coated fistula plug.
干预措施: MSC-AFP (Drug)
结局指标
主要结局
Number of participants with treatment-related adverse events (safety and toxicity)
时间窗: 2 months to approximately at 24 months
Participants will have a health assessment and blood work done at each study visit to monitor for adverse events.
次要结局
- Number of participants with response to the treatment regarding cessation of drainage from the treated fistula.(2 months to approximately at 24 months)
- Number of participants with radiographic response to the treatment regarding the treated fistula.(Week 2, Week 8, and Week 24)
研究者
Michael C. Stephens
Sponsor-Investigator
Mayo Clinic