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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01915927
Recruitment Status : Active, not recruiting
First Posted : August 5, 2013
Last Update Posted : November 22, 2017
Information provided by (Responsible Party):
William A. Faubion, M.D., Mayo Clinic

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.

Condition or disease Intervention/treatment Phase
Perianal Crohn's Disease Drug: MSC-AFP Phase 1

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 20 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 1 Study of Autologous Mesenchymal Stromal Cell Coated Fistula Plug in Patients With Fistulizing Crohn's Disease.
Actual Study Start Date : July 2013
Estimated Primary Completion Date : December 2018
Estimated Study Completion Date : December 2019

Resource links provided by the National Library of Medicine

Arm Intervention/treatment
Only 1 arm: treatment with MSC-AFP

Primary Outcome Measures :
  1. To determine the safety and toxicity of using autologous MSC coated fistula plug in patients with fistulizing Crohn's Disease. [ Time Frame: 2-24 months ]
    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 :
  1. To assess in preliminary fashion the response of fistula healing induced by the GORE plug containing MSC [ Time Frame: 2-24 months ]
    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.

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Ages Eligible for Study:   18 Years to 65 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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)

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01915927

United States, Minnesota
Mayo Clinic
Rochester, Minnesota, United States, 55905
Sponsors and Collaborators
William A. Faubion, M.D.
Principal Investigator: William Faubion, MD Mayo Clinic

Responsible Party: William A. Faubion, M.D., PI, Mayo Clinic Identifier: NCT01915927     History of Changes
Other Study ID Numbers: 12-009716
First Posted: August 5, 2013    Key Record Dates
Last Update Posted: November 22, 2017
Last Verified: November 2017

Additional relevant MeSH terms:
Crohn Disease
Inflammatory Bowel Diseases
Gastrointestinal Diseases
Digestive System Diseases
Intestinal Diseases
Pathological Conditions, Anatomical