Re-treatment From a Phase I Study of MSC-AFP in Patients With Perianal Fistulas

Phase 1

Completed

• Conditions: Crohn's Perianal Fistula; Perianal Fistula; Cryptoglandular Perianal Fistula
• Interventions: Drug: MSC-AFP

• Registration Number: NCT03209700
• Lead Sponsor: William A. Faubion, M.D.

Brief Summary

This study is an extension to re-treat partial and non-responders from the previously approved Phase 1 MCS-AFP protocols IRB #12-009716 (Crohn's Disease perianal fistulas) and 15-003200 (cryptoglandular perianal fistulas).

Detailed Description

This study is an extension to re-treat partial and non-responders from the previously approved Phase 1 MCS-AFP protocols IRB #12-009716 (Crohn's Disease perianal fistulas) and 15-003200 (cryptoglandular perianal fistulas). This study will enroll patients that have persistent symptomatic perianal disease despite being treated with an MSC coated fistula plug, and also treat patients that have had recurrence of their perianal fistula.

As per the previously approval treatment under IND 15356, patients will have a single affected fistula treated, which is the same fistula that was treated in the original protocol. The matrix for delivering the cells is a Gore Bio-A Fistula Plug.

Subjects will be screened at outpatient clinic visits and interested qualified subjects will be offered participation in the trial and consented. At the first study visit (Visit 1; Screening visit), the patient will be evaluated and assessment will be made if an EUA is clinically necessary to assess the fistula. As this is a re-treatment trial, patients with incomplete response to initial plug placement typically have close follow up, recent MRI imaging, and may have a seton in place. If an EUA is clinically indicated, this would be by a colorectal surgeon for drainage of sepsis and placement of a seton as part of the standard clinical care for perianal fistula.

Patients will return on: Day 1, Week 4, Week 12, and Week 24.

Study Timeline

• Study First Submitted: 2017-07-03
• Study First Submitted QC: 2017-07-03
• Study First Posted: 2017-07-06
• Study Start: 2017-07-20
• Status Verified: 2019-12
• Primary Completion: 2019-12-10
• Study Completion: 2019-12-10
• Last Update Submitted: 2019-12-31
• Last Update Posted: 2020-01-03

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 7

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
MSC-AFP Single Treatment Group	MSC-AFP	Eligible patients will be treated, single treatment group, no placebo arm

Primary Outcome Measures

Name	Time	Method
Number of participants with treatment-related adverse events (safety and toxicity).	6-12 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 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 Outcome Measures

Name	Time	Method
Number of participants with response to the treatment regarding potential cessation of drainage from their fistula.	6 months	Participants will have a clinical assessment of fistula drainage. Participants will be assessed during an office examination if their fistula is draining or not. The Outcome Measure will be the presence or absence of fistula drainage.

Trial Locations

Locations (1)

Mayo Clinic in Minnesota; 🇺🇸 Rochester, Minnesota, United States