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Stem Cell Coated Fistula Plug in Patients With Crohn's RVF

Primary Purpose

Fistula Vagina, Crohn Disease

Status

Completed

Phase

Phase 1

Locations

United States

Study Type

Interventional

Intervention

MSC-AFP

About this trial

This is an interventional treatment trial for Fistula Vagina

Eligibility Criteria

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

Inclusion Criteria

Females 18-65 years of age.
Residents of the United States.
Crohn's disease with single or multiple draining complex rectovaginal fistulae for at least three months despite standard therapy
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
Currently with diverting ileostomy or accepting of diverting ileostomy at time of stem cell loaded plug placement.

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.
Allergic to local anesthetics
Pregnant patients or trying to become pregnant.
entero-vesicular or multiple concurrent perianal tracts

Sites / Locations

Mayo Clinic in Rochester

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

MSC-AFP Single Treatment Group

Arm Description

Eligible patients will be treated, single treatment group, no placebo arm.

Outcomes

Primary Outcome Measures

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

Primary Outcome Measure: 1.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 perinal 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

NCT03220243

First Posted

July 14, 2017

Last Updated

October 28, 2020

Sponsor

Mayo Clinic

1. Study Identification

Unique Protocol Identification Number

NCT03220243

Brief Title

Stem Cell Coated Fistula Plug in Patients With Crohn's RVF

Official Title

A Phase I Study of Autologous Mesenchymal Stromal Cell Coated Fistula Plug in Patients With Rectovaginal Fistulizing Crohn's Disease

Study Type

Interventional

2. Study Status

Record Verification Date

October 2020

Overall Recruitment Status

Completed

Study Start Date

October 19, 2017 (Actual)

Primary Completion Date

September 20, 2020 (Actual)

Study Completion Date

September 20, 2020 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Principal Investigator

Name of the Sponsor

Mayo Clinic

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

5. Study Description

Brief Summary

The purpose of this study is to determine the safety of using an autologous mesenchymal stromal cell (MSC) coated fistula plug in people with rectovaginal fistulizing Crohn's disease. This is an autologous product derived from the patient and used only for the same patient. Participants will be in this study for two years. There is potential to continue to monitor participants' progress with regular visits as part of standard of care. All study visits take place at Mayo Clinic and Rochester, MN. The study visit schedule is as follows: Visit 1 (Week -6) - Screening visit: exam under anesthesia and surgery to assess eligibility of fistula tract, take fat biopsy, if eligible, and fecal diversion. Visit 2 (Week 0; Day 0), exam under anesthesia for stem cell coated fistula plug placement Visit 3 (Week 0; Day 1) Visit 4 (Week 2; Month 1) Visit 5 (Week 4; Month 1) Visit 6 (Week 8; Month 2) Visit 7 (Week 12; Month 3) Visit 8 (Week 24; Month 6) Visit 9 (Week 52; Month 12). Visit 10 (Week 104, Month 24)

Detailed Description

The Investigators propose to study the safety of autologous mesenchymal stromal cell transfer using a biomatrix (the Gore Bio-A174; Fistula Plug) in a Phase I study using a single dose of 20 million cells. Fifteen adult patients with refractory, rectovaginal fistulizing Crohn's disease will be enrolled. Participants will undergo standard adjuvant therapy including drainage of infection and placement of a draining seton with continuation of pre-existing anti-Crohn's therapy. If the participant is not currently diverted, then participants will undergo a laparoscopic diversion with a loop ileostomy. 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 participants 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. Participants will be screened at outpatient clinic visits and interested qualified participants will be offered participation in the trial and consented. At the first study visit (Visit 1; Screening visit), the patient will be evaluated and assessed. Six weeks post placement of the draining seton, the seton will be replaced with the MSC loaded Gore Bio-A fistula plug. Participants will return on: Day 1, Week 2, Week 4, Week 8, Week 12, Week 24, Week 52, and Week 104.

6. Conditions and Keywords

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

Fistula Vagina, Crohn 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

5 (Actual)

8. Arms, Groups, and Interventions

Arm Title

MSC-AFP Single Treatment Group

Arm Type

Experimental

Arm Description

Eligible patients will be treated, single treatment group, no placebo arm.

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 cells.

Primary Outcome Measure Information:

Title

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

Description

Time Frame

0-24 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

3-24 months

10. Eligibility

Sex

Female

Gender Based

Yes

Gender Eligibility Description

Rectovaginal fistula, females only eligible

Minimum Age & Unit of Time

18 Years

Maximum Age & Unit of Time

65 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria Females 18-65 years of age. Residents of the United States. Crohn's disease with single or multiple draining complex rectovaginal fistulae for at least three months despite standard therapy 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 Currently with diverting ileostomy or accepting of diverting ileostomy at time of stem cell loaded plug placement. 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. Allergic to local anesthetics Pregnant patients or trying to become pregnant. entero-vesicular or multiple concurrent perianal tracts

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Amy L Lightner

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

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Stem Cell Coated Fistula Plug in Patients With Crohn's RVF

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