Stem Cell Fistula Plug in Cryptoglandular Perianal Fistulas (MSC-AFP)

A Phase I Study of Autologous Mesenchymal Stromal Cell Coated Fistula Plug in Patients With High, Transsphincteric Cryptoglandular Perianal Fistulas

The investigators propose to study the safety of autologous mesenchymal stromal cell transfer using a biomatrix (the Gore Fistula Plug) in a Phase I study using a single dose of 20 million cells. 15 adult patients, ages 18 years and older with cryptoglandular 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; 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 Fistula; Cryptoglandular Perianal Fistula
• Intervention / Treatment: Drug: MSC-AFP

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. 15 adult patients (age > 18 years) with cryptoglandular 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. Study visits are Day 1, Week 2, Week 4, Week 8, Week 12, Week 24, Week 52, and Week 104. This is an autologous product derived from the patient and used only for the same patient.

• Study Type: Interventional
• Enrollment (Actual): 15
• Phase: Phase 1

Contacts and Locations

Study Locations

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

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

1. Males and females 18-65 years of age.
2. Residents of the United States.
3. Single-tract, transsphincteric anal fistula of cryptoglandular origin. Primary fistulas (no previous surgical treatment) and those who have failed previous surgical repairs
4. Have no contraindications to MR evaluations: e.g. pacemaker or magnetically active metal fragments, claustrophobia
5. Ability to comply with protocol
6. Competent and able to provide written informed consent

Exclusion Criteria

1. Inability to give informed consent.
2. 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.
3. Specific exclusions; Evidence of hepatitis B, C, or HIV
4. History of cancer including melanoma (with the exception of localized skin cancers)
5. Investigational drug within thirty (30) days of baseline
6. A resident outside the United States
7. Pregnant or breast feeding.
8. History of clinically significant auto-immunity (e.g. Crohn's disease) or any previous example of fat-directed autoimmunity
9. Previous allergic reaction to a perianal fistula plug.
10. If liposuction is not technically feasible
11. Allergic to local anesthetics
12. Pregnant patients or trying to become pregnant.
13. Non-enterocutaneous tracts (i.e. recto-vaginal, entero-vesicular)
14. Multi-tract, suprasphincteric and extrasphincteric fistula tract extensions
15. Active local infection associated with the fistula

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: MSC-AFP; Single Treatment Group	Drug: MSC-AFP; Eligible patients will be treated with a fistula plug that has been coated with autologous mesenchymal stromal cell.; Other Names: mesenchymal stromal cell coated fistula plug

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of participants with treatment-related adverse events (safety and toxicity).	Participants will have a health assessment and blood work measured at each study visit to monitor for adverse events, such as worsening of the cryptoglandular 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	2-24 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of participants with response to the treatment regarding potential cessation of drainage from their fistula.	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.	2-24 months

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of participants with response to the treatment regarding their score based on the Cleveland Clinic Florida (Wexner) fecal incontinence questionnaire.	Participants will have an assessment with the Cleveland Clinic Florida (Wexner) fecal incontinence questionnaire. Participants have the questionnaire administered within one week before plug implantation and at the 6 month and 12 months follow up visits. The Outcome Measure will be the how the participants' score change over time.	6-12 months
Number of participants with radiographic healing response to the treatment.	Participants will have an assessment of healing radiographically by magnetic resonance imaging (MRI). Participants' fistula tracts will be assessed during a MRI if their fistula is healing. The Outcome Measure will be the closure or persistence of fistula tract.	2-24 months

Collaborators and Investigators

• Sponsor: Mayo Clinic
• Investigators: Principal Investigator: Eric J Dozois, MD, Mayo Clinic

Publications and helpful links

Helpful Links

• Mayo Clinic Clinical Trials

Study record dates

Study Major Dates

• Study Start: January 1, 2016
• Primary Completion (Actual): September 1, 2019
• Study Completion (Actual): September 1, 2019

Study Registration Dates

• First Submitted: October 21, 2015
• First Submitted That Met QC Criteria: October 26, 2015
• First Posted (Estimate): October 28, 2015

Study Record Updates

• Last Update Posted (Actual): December 6, 2019
• Last Update Submitted That Met QC Criteria: December 3, 2019
• Last Verified: December 1, 2019

More Information

Terms related to this study

• Keywords: perianal fistula; cryptoglandular; cryptoglandular perianal fistulas
• Additional Relevant MeSH Terms: Digestive System Diseases; Gastrointestinal Diseases; Intestinal Diseases; Pathological Conditions, Anatomical; Rectal Diseases; Intestinal Fistula; Digestive System Fistula; Fistula; Rectal Fistula
• Other Study ID Numbers: 15-003200