Re-treatment From a Phase I Study of MSC-AFP in Patients With Perianal Fistulas

NCT ID: NCT03209700

Last Updated: 2020-01-03

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1
• Total Enrollment: 7 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2017-07-20
• Study Completion Date: 2019-12-10

Brief Summary

This study is an extension to re-treat partial and non-responders from the previously approved Phase 1 MCS-AFP protocols IRB #12-009716 (Crohn's Disease perianal fistulas) and 15-003200 (cryptoglandular perianal fistulas).

Detailed Description

This study is an extension to re-treat partial and non-responders from the previously approved Phase 1 MCS-AFP protocols IRB #12-009716 (Crohn's Disease perianal fistulas) and 15-003200 (cryptoglandular perianal fistulas). This study will enroll patients that have persistent symptomatic perianal disease despite being treated with an MSC coated fistula plug, and also treat patients that have had recurrence of their perianal fistula.

As per the previously approval treatment under IND 15356, patients will have a single affected fistula treated, which is the same fistula that was treated in the original protocol. The matrix for delivering the cells is a Gore Bio-A Fistula Plug.

Subjects will be screened at outpatient clinic visits and interested qualified subjects will be offered participation in the trial and consented. At the first study visit (Visit 1; Screening visit), the patient will be evaluated and assessment will be made if an EUA is clinically necessary to assess the fistula. As this is a re-treatment trial, patients with incomplete response to initial plug placement typically have close follow up, recent MRI imaging, and may have a seton in place. If an EUA is clinically indicated, this would be by a colorectal surgeon for drainage of sepsis and placement of a seton as part of the standard clinical care for perianal fistula.

Patients will return on: Day 1, Week 4, Week 12, and Week 24.

Conditions

Perianal Fistula; Cryptoglandular Perianal Fistula; Crohn's Perianal Fistula

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

MSC-AFP Single Treatment Group

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

Group Type: EXPERIMENTAL

MSC-AFP

Intervention Type: DRUG

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

Interventions

MSC-AFP

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

Intervention Type: DRUG

Other Intervention Names

mesenchymal stromal cell coated fistula plug

Eligibility Criteria

Inclusion Criteria

1. Males and females 18-65 years of age.

2. Residents of the United States.

3. Single draining perianal fistula for at least three months despite standard therapy

4. Concurrent therapies are permitted (such as antibiotics, corticosteroids, thiopurines).

5. Have no contraindications to MR evaluations: e.g. pacemaker or magnetically active metal fragments, claustrophobia

6. Ability to comply with protocol

7. Competent and able to provide written informed consent

8. Patients that were treated with an MSC-coated fistula plug in the study IRB#12-009716 or IRB#15-003200

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. Previous allergic reaction to a perianal fistula plug.

8. Allergic to local anesthetics

9. Pregnant patients or trying to become pregnant or breast feeding.

10. Non-enterocutaneous tracts (i.e. recto-vaginal, entero-vesicular)

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 65 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

William A. Faubion, M.D.

OTHER

Sponsor Role: lead

Responsible Party

William A. Faubion, M.D.

William A. Faubion, M.D.

Responsibility Role: SPONSOR_INVESTIGATOR

Principal Investigators

William A Faubion, M.D.

Role: PRINCIPAL_INVESTIGATOR

Mayo Clinic

Eric J Dozois, M.D.

Role: PRINCIPAL_INVESTIGATOR

Mayo Clinic

Locations

Mayo Clinic in Minnesota

Rochester, Minnesota, United States

Countries

United States

Other Identifiers

17-004503

Identifier Type: -

Identifier Source: org_study_id