A Phase I Study of Autologous Mesenchymal Stromal Cell Coated Fistula Plug in Patients With Rectovaginal Fistulizing Crohn's Disease
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- Mayo Clinic
- 入组人数
- 5
- 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 rectovaginal fistulizing Crohn's disease. This is an autologous product derived from the patient and used only for the same patient. Participants will be in this study for two years. There is potential to continue to monitor participants' progress with regular visits as part of standard of care. All study visits take place at Mayo Clinic and Rochester, MN.
The study visit schedule is as follows:
Visit 1 (Week -6) - Screening visit: exam under anesthesia and surgery to assess eligibility of fistula tract, take fat biopsy, if eligible, and fecal diversion.
Visit 2 (Week 0; Day 0), exam under anesthesia for stem cell coated fistula plug placement Visit 3 (Week 0; Day 1) Visit 4 (Week 2; Month 1) Visit 5 (Week 4; Month 1) Visit 6 (Week 8; Month 2) Visit 7 (Week 12; Month 3) Visit 8 (Week 24; Month 6) Visit 9 (Week 52; Month 12). Visit 10 (Week 104, Month 24)
详细描述
The Investigators propose to study the safety of autologous mesenchymal stromal cell transfer using a biomatrix (the Gore Bio-A174; Fistula Plug) in a Phase I study using a single dose of 20 million cells. Fifteen adult patients with refractory, rectovaginal fistulizing Crohn's disease will be enrolled. Participants will undergo standard adjuvant therapy including drainage of infection and placement of a draining seton with continuation of pre-existing anti-Crohn's therapy. If the participant is not currently diverted, then participants will undergo a laparoscopic diversion with a loop ileostomy. Six weeks post placement of the draining seton, the seton will be replaced with the MSC loaded Gore Bio-A fistula plug as per current clinical practice. The participants 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.
Participants will be screened at outpatient clinic visits and interested qualified participants will be offered participation in the trial and consented. At the first study visit (Visit 1; Screening visit), the patient will be evaluated and assessed. Six weeks post placement of the draining seton, the seton will be replaced with the MSC loaded Gore Bio-A fistula plug. Participants will return on: Day 1, Week 2, Week 4, Week 8, Week 12, Week 24, Week 52, and Week 104.
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
- 18 Years 至 65 Years（Adult, Older Adult）
- 性别
- Female
- 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 Single Treatment Group
Eligible patients will be treated, single treatment group, no placebo arm.
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: 0-24 months
Primary Outcome Measure: 1.Number of participants with treatment-related adverse events (safety and toxicity). Participants will have a health assessment and blood work measured at each study visit to monitor for adverse events, such as worsening of the perinal fistulizing disease, abnormal laboratory values, or significant abnormalities in physical examination. The Outcome Measure will be the number of Participants With Abnormal Laboratory Values and/or Adverse Events That Are Related to Treatment
次要结局
- Number of participants with response to the treatment regarding potential cessation of drainage from their fistula.(3-24 months)
研究者
Eric J. Dozois, M.D.
Principal Investigator
Mayo Clinic