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

Primary Purpose

Perianal Crohn's Disease

Status
Completed
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
MSC-AFP
Sponsored by
William A. Faubion, M.D.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Perianal Crohn's Disease

Eligibility Criteria

18 Years - 65 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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)

Sites / Locations

  • Mayo Clinic

Arms of the Study

Arm 1

Arm Type

Other

Arm Label

1

Arm Description

Only 1 arm: treatment with MSC-AFP

Outcomes

Primary Outcome Measures

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.

Secondary Outcome Measures

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.

Full Information

First Posted
July 31, 2013
Last Updated
December 31, 2019
Sponsor
William A. Faubion, M.D.
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1. Study Identification

Unique Protocol Identification Number
NCT01915927
Brief Title
Stem Cell Fistula Plug in Perianal Crohn's Disease
Acronym
MSC-AFP
Official Title
A Phase 1 Study of Autologous Mesenchymal Stromal Cell Coated Fistula Plug in Patients With Fistulizing Crohn's Disease.
Study Type
Interventional

2. Study Status

Record Verification Date
December 2019
Overall Recruitment Status
Completed
Study Start Date
July 2013 (Actual)
Primary Completion Date
December 2019 (Actual)
Study Completion Date
December 2019 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor-Investigator
Name of the Sponsor
William A. Faubion, M.D.

4. Oversight

Data Monitoring Committee
No

5. Study Description

Brief Summary
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Perianal Crohn's Disease

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
20 (Actual)

8. Arms, Groups, and Interventions

Arm Title
1
Arm Type
Other
Arm Description
Only 1 arm: treatment with MSC-AFP
Intervention Type
Drug
Intervention Name(s)
MSC-AFP
Primary Outcome Measure Information:
Title
To determine the safety and toxicity of using autologous MSC coated fistula plug in patients with fistulizing Crohn's Disease.
Description
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.
Time Frame
2-24 months
Secondary Outcome Measure Information:
Title
To assess in preliminary fashion the response of fistula healing induced by the GORE plug containing MSC
Description
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.
Time Frame
2-24 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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)
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
William Faubion, MD
Organizational Affiliation
Mayo Clinic
Official's Role
Principal Investigator
Facility Information:
Facility Name
Mayo Clinic
City
Rochester
State/Province
Minnesota
ZIP/Postal Code
55905
Country
United States

12. IPD Sharing Statement

Citations:
PubMed Identifier
28400193
Citation
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.
Results Reference
background
Links:
URL
https://www.mayo.edu/research/clinical-trials
Description
Mayo Clinic Clinical Trials

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Stem Cell Fistula Plug in Perianal Crohn's Disease

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