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

Study Overview
=================
Brief Summary
-----------------
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.

Detailed Description
-----------------
The investigators propose to study the safety of autologous mesenchymal stromal cell transfer using a biomatrix (the Gore&#174; Bio-A&#174; Fistula Plug) in a Phase I study using a single dose of 20 million cells. 15 adult patients (age &gt; 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&#174; 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.

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

Conditions
-----------------
Perianal Fistula, Cryptoglandular Perianal Fistula

Intervention / Treatment
-----------------
* Drug: MSC-AFP


Participation Criteria
=================
Eligibility Criteria
-----------------
Inclusion Criteria Males and females 18-65 years of age. Residents of the United States. Single-tract, transsphincteric anal fistula of cryptoglandular origin. Primary fistulas (no previous surgical treatment) and those who have failed previous surgical repairs 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 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 (e.g. 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 (i.e. recto-vaginal, entero-vesicular) Multi-tract, suprasphincteric and extrasphincteric fistula tract extensions Active local infection associated with the fistula

Ages Eligible for Study
-----------------
Minimum Age: 18 Years
Maximum Age: 65 Years

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Other: MSC-AFP<br>Single Treatment Group | Drug: MSC-AFP<br>* Eligible patients will be treated with a fistula plug that has been coated with autologous mesenchymal stromal cell.<br>* 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 | 

 Terms related to the study
=================
Keywords Provided by Centre Hospitalier Valida
-----------------
cryptoglandular, perianal fistula, cryptoglandular perianal fistulas