Clinical trial NCT03209700: Re-treatment From a Phase I Study of MSC-AFP in Patients With Perianal Fistulas
Official title: Re-treatment for Partial or Non-responders for a Phase I Study of Autologous Mesenchymal Stromal Cell Coated Fistula Plug in Patients With Perianal Fistulas
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Perianal Fistula
Cryptoglandular Perianal Fistula
Crohn's Perianal Fistula
Lead sponsor: Mayo Clinic
Sponsor class: OTHER
First posted: 2017-07-06
Enrollment: 7
Sex: ALL
Minimum age (years): 18
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

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)

Primary outcomes:
measure: Number of participants with treatment-related adverse events (safety and toxicity).
timeFrame: 6-12 months
Secondary outcomes:
measure: Number of participants with response to the treatment regarding potential cessation of drainage from their fistula.
timeFrame: 6 months
Other outcomes:
Locations:
city: Rochester
country: United States
facility: Mayo Clinic in Minnesota
state: Minnesota
Study officials:
affiliation: Mayo Clinic
name: William A Faubion, M.D.
role: PRINCIPAL_INVESTIGATOR
affiliation: Mayo Clinic
name: Eric J Dozois, M.D.
role: PRINCIPAL_INVESTIGATOR
References: