Stem Cell Fistula Plug in Perianal Crohn's Disease (MSC-AFP)

A Phase 1 Study of Autologous Mesenchymal Stromal Cell Coated Fistula Plug in Patients With Fistulizing Crohn's Disease.

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. Twenty adult patients (age 18 years or older) with refractory, complicated perianal fistulizing Crohn's disease will be enrolled. Subjects will undergo standard adjuvant therapy including drainage of infection and placement of a draining seton with continuation of pre-existing anti-Crohn's therapy. 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 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 Overview

• Status: Completed
• Conditions: Perianal Crohn's Disease
• Intervention / Treatment: Drug: MSC-AFP

• Study Type: Interventional
• Enrollment (Actual): 20
• Phase: Phase 1

Contacts and Locations

Study Locations

• United States
  • Minnesota
    • Rochester, Minnesota, United States, 55905
      • Mayo Clinic

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 65 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria

• Males and females 18-65 years of age.
• Residents of the United States.
• Crohn's disease with single or multiple draining complex perianal fistulae (definition as below) for at least three months despite standard therapy (definition below).
• Concurrent therapies with corticosteroids, 5-ASA drugs, thiopurines, MTX, antibiotics, and anti-TNF therapy are permitted.
• All patients should have undergone a colonoscopy in last 12 months to rule out malignant or premalignant condition
• Have no contraindications to MR evaluations: e.g. pacemaker or magnetically active metal fragments, claustrophobia
• Ability to comply with protocol
• Competent and able to provide written informed consent
• Must have failed standard medical therapy including anti-TNF agents

Exclusion Criteria

• Inability to give informed consent.
• Clinically significant medical conditions within the six months before administration of MSCs: e.g. myocardial infarction, active angina, congestive heart failure or other conditions that would, in the opinion of the investigators, compromise the safety of the patient.
• Specific exclusions: Evidence of hepatitis B, C, or HIV
• History of cancer including melanoma (with the exception of localized skin cancers)
• Investigational drug within thirty (30) days of baseline
• A resident outside the United States
• Pregnant or breast feeding.
• History of clinically significant auto-immunity (other than Crohn's disease) or any previous example of fat-directed autoimmunity
• Previous allergic reaction to a perianal fistula plug.
• If liposuction is not technically feasible
• Allergic to local anesthetics
• Pregnant patients or trying to become pregnant.
• Non-enterocutaneous tracts (ie recto-vaginal, entero-vesicular)

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Other: 1; Only 1 arm: treatment with MSC-AFP	Drug: MSC-AFP

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
To determine the safety and toxicity of using autologous MSC coated fistula plug in patients with fistulizing Crohn's Disease.	The primary endpoint of this study is to determine the safety and feasibility of using adipose derived, autologous mesenchymal stromal cells (MSC) bound to the Gore® Bio-A® Fistula Plug for treatment of refractory CD perianal fistulae.	2-24 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
To assess in preliminary fashion the response of fistula healing induced by the GORE plug containing MSC	To assess in preliminary fashion the response of fistula healing induced by the GORE plug containing MSC i) Clinically by drainage assessment and ii) Radiographically by MRI, the gold standard test for assessment of healing.	2-24 months

Collaborators and Investigators

• Sponsor: William A. Faubion, M.D.
• Investigators: Principal Investigator: William Faubion, MD, Mayo Clinic

Publications and helpful links

General Publications

• Dietz AB, Dozois EJ, Fletcher JG, Butler GW, Radel D, Lightner AL, Dave M, Friton J, Nair A, Camilleri ET, Dudakovic A, van Wijnen AJ, Faubion WA. Autologous Mesenchymal Stem Cells, Applied in a Bioabsorbable Matrix, for Treatment of Perianal Fistulas in Patients With Crohn's Disease. Gastroenterology. 2017 Jul;153(1):59-62.e2. doi: 10.1053/j.gastro.2017.04.001. Epub 2017 Apr 9.

Helpful Links

• Mayo Clinic Clinical Trials

Study record dates

Study Major Dates

• Study Start (Actual): July 1, 2013
• Primary Completion (Actual): December 1, 2019
• Study Completion (Actual): December 1, 2019

Study Registration Dates

• First Submitted: July 31, 2013
• First Submitted That Met QC Criteria: August 1, 2013
• First Posted (Estimate): August 5, 2013

Study Record Updates

• Last Update Posted (Actual): January 3, 2020
• Last Update Submitted That Met QC Criteria: December 31, 2019
• Last Verified: December 1, 2019

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Digestive System Diseases; Gastrointestinal Diseases; Gastroenteritis; Intestinal Diseases; Pathological Conditions, Anatomical; Inflammatory Bowel Diseases; Fistula; Crohn Disease
• Other Study ID Numbers: 12-009716