A Phase I Study of Autologous Mesenchymal Stromal Cell Coated Fistula Plug in Patients With High, Transsphincteric Cryptoglandular Perianal Fistulas
Overview
- Phase
- Phase 1
- Intervention
- MSC-AFP
- Conditions
- Perianal Fistula
- Sponsor
- Mayo Clinic
- Enrollment
- 15
- Locations
- 1
- Primary Endpoint
- Number of participants with treatment-related adverse events (safety and toxicity).
- Status
- Completed
- Last Updated
- 6 years ago
Overview
Brief Summary
The investigators propose to study the safety of autologous mesenchymal stromal cell transfer using a biomatrix (the Gore Fistula Plug) in a Phase I study using a single dose of 20 million cells. 15 adult patients, ages 18 years and older with cryptoglandular 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; 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.
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. 15 adult patients (age \> 18 years) with cryptoglandular 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. Study visits are Day 1, Week 2, Week 4, Week 8, Week 12, Week 24, Week 52, and Week 104. This is an autologous product derived from the patient and used only for the same patient.
Investigators
Eric J. Dozois, M.D.
MD
Mayo Clinic
Eligibility Criteria
Inclusion Criteria
- Not provided
Exclusion Criteria
- Not provided
Arms & Interventions
MSC-AFP
Single Treatment Group
Intervention: MSC-AFP
Outcomes
Primary Outcomes
Number of participants with treatment-related adverse events (safety and toxicity).
Time Frame: 2-24 months
Participants will have a health assessment and blood work measured at each study visit to monitor for adverse events, such as worsening of the cryptoglandular 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.(2-24 months)