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

NCT02589119 | Completed Phase 1 DMC

• 1 other identifier: 15-003200
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 1

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.

Conditions

Perianal Fistula; Cryptoglandular Perianal Fistula

Keywords

cryptoglandular; perianal fistula; cryptoglandular perianal fistulas

Outcome Measures

Primary Outcomes (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 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 Outcomes (1)

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

  2-24 months

Other Outcomes (2)

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

  6-12 months

• Number of participants with radiographic healing response to the treatment.

  2-24 months

Study Arms (1)

MSC-AFP

OTHER

Single Treatment Group

Drug: MSC-AFP

Interventions

MSC-AFP DRUG

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

Also known as: mesenchymal stromal cell coated fistula plug

MSC-AFP

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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.
• 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

Sponsors & Collaborators

• Mayo Clinic: lead

Study Sites (1)

Mayo Clinic in Rochester

Rochester, Minnesota, 55905, United States

Location

Related Links

• Mayo Clinic Clinical Trials

Study Officials

• Eric J Dozois, MD

  Mayo Clinic

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: MD

Study Record Dates

• First Submitted: October 21, 2015
• First Posted: October 28, 2015
• Study Start: January 1, 2016
• Primary Completion: September 1, 2019
• Study Completion: September 1, 2019
• Last Updated: December 6, 2019

Record last verified: 2019-12

Locations

US (1)