Study of Mesenchymal Stem Cells for Pediatric Perianal Fistulizing Crohn's Disease

• Status: Recruiting
• Trial end date: 2023-04-01
• Target enrollment: 0
• Participant gender: All

Summary

This study plans to enroll 10 patients aged 13-17 years of age with refractory perianal fistulizing disease. Patients will be treated by direct injection to the fistula tract(s) with 75 million allogeneic bone marrow derived mesenchymal stem cells at baseline and again after 3 months if not completely healed.

• Phase: Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Amy Lightner

Criteria

Inclusion Criteria

1. Males and females aged 13-17 with a diagnosis of Crohn's disease for at least six months duration.

2. Single and Multi-tract Perianal fistula, with or without previous failed surgical repair.

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

4. Ability to comply with protocol

5. Competent and able to provide written informed consent

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

7. Agree to use birth control or abstinence to avoid pregnancy during the study

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:

1. Hepatitis B or C

2. HIV

3. Abnormal AST or ALT at screening

4. History of colon cancer in the past two years, or treatment for other cancers within the last 6 months.

5. Investigational drug within one month of treatment

6. Pregnant or breast feeding or trying to become pregnant.

7. Presence of a rectovaginal or perineal body fistula

8. Change in Crohn's immunosuppressive regimen within the 2 months prior to enrollment

9. Uncontrolled intestinal Crohn's disease which will require escalation for medical therapy or surgery within 2 months of enrollment

10. Severe anal canal disease that is stenotic and requires dilation

11. Female participant unwilling to agree to use acceptable contraception methods during participation in study