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

Primary Purpose

Perianal Fistula, Cryptoglandular Perianal Fistula

Status

Completed

Phase

Phase 1

Locations

United States

Study Type

Interventional

Intervention

MSC-AFP

About this trial

This is an interventional treatment trial for Perianal Fistula focused on measuring cryptoglandular, perianal fistula, cryptoglandular perianal fistulas

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.
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

Sites / Locations

Mayo Clinic in Rochester

Arms of the Study

Arm 1

Arm Type

Other

Arm Label

MSC-AFP

Arm Description

Single Treatment Group

Outcomes

Primary Outcome Measures

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

Secondary Outcome Measures

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.

Full Information

NCT ID

NCT02589119

First Posted

October 21, 2015

Last Updated

December 3, 2019

Sponsor

Mayo Clinic

1. Study Identification

Unique Protocol Identification Number

NCT02589119

Brief Title

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

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

December 2019

Overall Recruitment Status

Completed

Study Start Date

January 2016 (undefined)

Primary Completion Date

September 2019 (Actual)

Study Completion Date

September 2019 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Principal Investigator

Name of the Sponsor

Mayo Clinic

4. Oversight

Data Monitoring Committee

Yes

5. Study Description

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® 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Perianal Fistula, Cryptoglandular Perianal Fistula

Keywords

cryptoglandular, perianal fistula, cryptoglandular perianal fistulas

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

15 (Actual)

8. Arms, Groups, and Interventions

Arm Title

MSC-AFP

Arm Type

Other

Arm Description

Single Treatment Group

Intervention Type

Drug

Intervention Name(s)

MSC-AFP

Other Intervention Name(s)

mesenchymal stromal cell coated fistula plug

Intervention Description

Eligible patients will be treated with a fistula plug that has been coated with autologous mesenchymal stromal cell.

Primary Outcome Measure Information:

Title

Number of participants with treatment-related adverse events (safety and toxicity).

Description

Time Frame

2-24 months

Secondary Outcome Measure Information:

Title

Number of participants with response to the treatment regarding potential cessation of drainage from their fistula.

Description

Time Frame

2-24 months

Other Pre-specified Outcome Measures:

Title

Number of participants with response to the treatment regarding their score based on the Cleveland Clinic Florida (Wexner) fecal incontinence questionnaire.

Description

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.

Time Frame

6-12 months

Title

Number of participants with radiographic healing response to the treatment.

Description

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.

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

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

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Eric J Dozois, MD

Organizational Affiliation

Mayo Clinic

Official's Role

Principal Investigator

Facility Information:

Facility Name

Mayo Clinic in Rochester

City

Rochester

State/Province

Minnesota

ZIP/Postal Code

55905

Country

United States

12. IPD Sharing Statement

Links:

URL

https://www.mayo.edu/research/clinical-trials

Description

Mayo Clinic Clinical Trials

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

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