A Phase I Study of Autologous Mesenchymal Stromal Cell Coated Fistula Plug in Patients With Rectovaginal Fistulizing Crohn's Disease
Overview
- Phase
- Phase 1
- Intervention
- MSC-AFP
- Conditions
- Fistula Vagina
- Sponsor
- Mayo Clinic
- Enrollment
- 5
- Locations
- 1
- Primary Endpoint
- Number of participants with treatment-related adverse events (safety and toxicity).
- Status
- Completed
- Last Updated
- 5 years ago
Overview
Brief Summary
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)
Detailed Description
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.
Investigators
Eric J. Dozois, M.D.
Principal Investigator
Mayo Clinic
Eligibility Criteria
Inclusion Criteria
- Not provided
Exclusion Criteria
- Not provided
Arms & Interventions
MSC-AFP Single Treatment Group
Eligible patients will be treated, single treatment group, no placebo arm.
Intervention: MSC-AFP
Outcomes
Primary Outcomes
Number of participants with treatment-related adverse events (safety and toxicity).
Time Frame: 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
Secondary Outcomes
- Number of participants with response to the treatment regarding potential cessation of drainage from their fistula.(3-24 months)