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

NCT03220243 | Completed Phase 1 FDA Drug

• 1 other identifier: 17-002892
• Study Type: interventional
• Target: 5
• Locations: 1 country
• Sites: 1

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)

Conditions

Fistula Vagina; Crohn Disease

Outcome Measures

Primary Outcomes (1)

• 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

  0-24 months

Secondary Outcomes (1)

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

  3-24 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
• Gender Eligibility Details: Rectovaginal fistula, females only eligible
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

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 (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

Sponsors & Collaborators

• Mayo Clinic: lead

Study Sites (1)

Mayo Clinic in Rochester

Rochester, Minnesota, 55905, United States

Location

MeSH Terms

Conditions

Vaginal Fistula; Crohn Disease

Condition Hierarchy (Ancestors)

Vaginal Diseases; Genital Diseases, Female; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Genital Diseases; Fistula; Pathological Conditions, Anatomical; Pathological Conditions, Signs and Symptoms; Inflammatory Bowel Diseases; Gastroenteritis; Gastrointestinal Diseases; Digestive System Diseases; Intestinal Diseases

Study Officials

• Amy L Lightner

  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: Principal Investigator

Study Record Dates

• First Submitted: July 14, 2017
• First Posted: July 18, 2017
• Study Start: October 19, 2017
• Primary Completion: September 20, 2020
• Study Completion: September 20, 2020
• Last Updated: October 30, 2020

Record last verified: 2020-10

Locations

US (1)