Mesenchymal Stem Cells for the Treatment of Pouch Fistulas in Crohn's

• Register: ClinicalTrials.gov
• Last refreshed on: 19 April 2022
• Main ID: NCT04073472
• Date of registration: 23/08/2019
• Prospective Registration: Yes
• Primary sponsor: The Cleveland Clinic
• Public title: Mesenchymal Stem Cells for the Treatment of Pouch Fistulas in Crohn's IPAAF
• Scientific title: A Phase I Study of Allogeneic Bone Marrow Derived Mesenchymal Stem Cells for the Treatment of Ileal Anal Anastomosis and Ileal Pouch Fistulas in the Setting of Crohn's Disease of the Pouch
• Date of first enrolment: June 1, 2021
• Target sample size: 0
• Recruitment status: Withdrawn
• URL: https://clinicaltrials.gov/show/NCT04073472
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 1

Countries of recruitment

United States

Contacts

• Name: Amy Lightner, MD
• Affiliation: The Cleveland Clinic

Key inclusion & exclusion criteria

Inclusion Criteria

1. Men and women 18-75 years of age who have undergone an ileal pouch anal anastomosis at
least 6 months prior who have developed a clinical diagnosis of Crohn's disease of the
pouch as determined by a combination of clinical symptoms, pouchoscopy with biopsy,
enterography.

2. Single and multi-tract (up to 2 internal and 3 external openings) fistula tract
arising from the ileal pouch, ileal anal anastomosis, or anal canal distal to
anastomosis that travels to the perianal skin, perineal body, or vagina. Patients with
fistulas that arise from the pouch, anastomosis, or anal canal distal to the
anastomosis will both be included in enrollment.

1. Acceptable internal openings and tract locations for the fistula to arise from
include the ileal pouch body, the pouch anal anastomosis, and the anal canal
distal to the anastomosis.

2. Acceptable external openings and tract locations for the fistula to arise from
include the perianal skin, perineal body, and/or the vaginal wall.

3. Concurrent Crohn's related therapies with stable doses (>3 months) corticosteroids,
5-ASA drugs, immunomodulators, anti-TNF therapy, anti-integrin and anti-interleukin
are permitted.

4. Have failed conventional medical therapies described above, defined as a lack of
response to systemic immune suppression (e.g. azathioprine, methotrexate,
6-mercaptopurine) or biologic (e.g. anti-TNF, anti-integrin, anti-interleukin)
therapies to treat fistulizing CD for at least 3 months

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

6. Competent and able to provide written informed consent

7. Ability to comply with protocol.

Exclusion Criteria

1. Inability to give informed consent.

2. Severe antibiotic refractory pouchitis

3. Severe cuffitis refractory to antibiotics

4. Change in medical management for CD in the previous 2 months or changes anticipated in
the next 2 months

5. 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.

6. Specific exclusions;

1. HIV

2. Hepatitis B or C

3. Abnormal CBC at screening

4. Abnormal AST or ALT at screening

7. History of cancer including melanoma (with the exception of localized skin cancers)

8. Investigational drug within thirty (30) days of baseline

9. Pregnant or breast feeding or trying to become pregnant

10. Branching fistula tract that has > 2 internal openings or 3 external openings,

1. Patients with greater than 3 blind/branching tracts are excluded

2. Fistula tracts on the left and/or right side are allowed

11. Allergic to local anesthetics

12. Unwilling to agree to use acceptable contraception methods during participation in
study

13. Patients with a non-abscessed chronic cavity will not be included in enrollment

14. Known allergy to DMSO solution



Age minimum: 18 Years
Age maximum: 75 Years
Gender: All

Health Condition(s) or Problem(s) studied

Fistula

Pouch, Ileal

Crohn's Disease

Anal Fistula

Pouches, Ileoanal

Intervention(s)

Drug: mesenchymal stem cells (MSCs)

Primary Outcome(s)

Safety and Feasibility: Number Of Adverse Events [Time Frame: Baseline, Drug administration Visit, Day 1, 1 week, 2 weeks, 1 month, 2 months, 3 month, 6 month, 12 month after each MSCs injection]

Secondary Outcome(s)

Clinical Healing [Time Frame: Baseline, 1 week, 2 weeks, 1 month, 2 months, 3 month, 6 month, 12 month after each 100% cessation of drainage on both clinical exam with deep palpation and per patient MSCs injection]

Radiographic Healing [Time Frame: Baseline, 1 week, 2 weeks, 1 month, 2 months, 3 month, 6 month, 12 month after each 100% cessation of drainage on both clinical exam with deep palpation and per patient MSCs injection]

Source(s) of Monetary Support

Please refer to primary and secondary sponsors