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

NCT03209700 | Completed Phase 1 FDA Drug

• 1 other identifier: 17-004503
• Study Type: interventional
• Target: 7
• Locations: 1 country
• Sites: 1

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).

Conditions

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

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 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

  6-12 months

Secondary Outcomes (1)

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

  6 months

Study Arms (1)

MSC-AFP Single Treatment Group

EXPERIMENTAL

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

Drug: MSC-AFP

Interventions

MSC-AFP DRUG

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

Also known as: mesenchymal stromal cell coated fistula plug

MSC-AFP Single Treatment Group

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 draining perianal fistula for at least three months despite standard therapy
• Concurrent therapies are permitted (such as antibiotics, corticosteroids, thiopurines).
• 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
• Patients that were treated with an MSC-coated fistula plug in the study IRB#12-009716 or IRB#15-003200

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
• Previous allergic reaction to a perianal fistula plug.
• Allergic to local anesthetics
• Pregnant patients or trying to become pregnant or breast feeding.
• Non-enterocutaneous tracts (i.e. recto-vaginal, entero-vesicular)

Sponsors & Collaborators

• William A. Faubion, M.D.: lead

Study Sites (1)

Mayo Clinic in Minnesota

Rochester, Minnesota, 55905, United States

Location

Study Officials

• William A Faubion, M.D.

  Mayo Clinic

  PRINCIPAL INVESTIGATOR

• Eric J Dozois, M.D.

  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: SPONSOR INVESTIGATOR
• PI Title: William A. Faubion, M.D.

Study Record Dates

• First Submitted: July 3, 2017
• First Posted: July 6, 2017
• Study Start: July 20, 2017
• Primary Completion: December 10, 2019
• Study Completion: December 10, 2019
• Last Updated: January 3, 2020

Record last verified: 2019-12

Locations

US (1)