Pediatric MSC-AFP Sub-study for Crohn's Fistula

Phase 1

Completed

• Conditions: Fistula in Ano; Crohn Disease
• Interventions: Drug: MSC-AFP

• Registration Number: NCT03449069
• Lead Sponsor: Mayo Clinic

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.

Study Timeline

• Study First Submitted: 2018-02-22
• Study First Submitted QC: 2018-02-22
• Study First Posted: 2018-02-28
• Study Start: 2018-07-23
• Primary Completion: 2023-12-31
• Study Completion: 2023-12-31
• Status Verified: 2024-03
• Last Update Submitted: 2024-03-11
• Last Update Posted: 2024-03-13

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 1

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
MSC-AFP	MSC-AFP	This is a single treatment group study. All patients will receive treatment of a stem cell coated fistula plug.

Primary Outcome Measures

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

Name	Time	Method
Number of participants with response to the treatment regarding cessation of drainage from the treated fistula.	2 months to approximately at 24 months	Participants will have a clinical assessment of fistula drainage at each study visit.
Number of participants with radiographic response to the treatment regarding the treated fistula.	Week 2, Week 8, and Week 24	Participants will have an assessment of healing radiographically by magnetic resonance imaging (MRI) at Week 2 visit, Week 8, and Week 24.

Trial Locations

Locations (1)

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