A Phase I Study of Autologous Mesenchymal Stromal Cell Coated Fistula Plug in Patients With Fistulizing Crohn's Disease: Pediatric Sub-study
Overview
- Phase
- Phase 1
- Intervention
- MSC-AFP
- Conditions
- Fistula in Ano
- Sponsor
- Mayo Clinic
- Enrollment
- 1
- Locations
- 1
- Primary Endpoint
- Number of participants with treatment-related adverse events (safety and toxicity)
- Status
- Completed
- Last Updated
- 2 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 fistulizing Crohn's disease. Autologous means these cells to coat the plug come from the patient.
Detailed Description
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.
Investigators
Michael C. Stephens
Sponsor-Investigator
Mayo Clinic
Eligibility Criteria
Inclusion Criteria
- Not provided
Exclusion Criteria
- Not provided
Arms & Interventions
MSC-AFP
This is a single treatment group study. All patients will receive treatment of a stem cell coated fistula plug.
Intervention: MSC-AFP
Outcomes
Primary Outcomes
Number of participants with treatment-related adverse events (safety and toxicity)
Time Frame: 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.
Secondary Outcomes
- 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)