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

Amy Lightner10 enrolled

Overview

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.

Crohn's disease (CD), a chronic transmural inflammatory disease of the gastrointestinal tract, continues to increase in incidence for unknown reasons. According to population based studies, at least 26% of patients with CD will develop perianal fistulas in the first two decades following diagnosis, particularly those with colonic and rectal involvement. These patients experience significant morbidity due to pain, persistent drainage, recurrent perianal sepsis, and ongoing need to access medical care resulting in increased costs and impaired quality of life. Onset of Crohn's disease in childhood is associated with even more aggressive perianal fistula development, with fistulas occurring in as many as 20-31% of children within 5-7 years after Crohn's disease diagnosis. Based on national estimates of pediatric Crohn's disease prevalence, this suggests that there are more than 10,000 children with perianal fistulas due to Crohn's disease in the United States. This study plans to enroll 10 patients (aged 13-17 years) with refractory perianal fistulizing disease. The next step in management for these patients would be a mucosal tissue flap, temporary stoma, or proctectomy with permanent ostomy. Patients will be treated by direct injection of 75 million allogeneic bone marrow derived mesenchymal stem cells at baseline and again after 3 months if not completely healed. Patients will be followed for a total of 12 months post initial injection.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Perianal Fistula Due to Crohn's Disease (Disorder)

Interventions

Mesenchymal stem cellsDRUG

Direct injection of 75 million allogeneic bone marrow derived mesenchymal stem cells at baseline and again after 3 months if not completely healed

Eligibility

Sex: ALLMin age: 13 YearsMax age: 17 Years

Medical Language ↔ Plain English

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 subject. 3. Specific exclusions: 1. Hepatitis B or C 2. HIV 3. Abnormal AST or ALT at screening (defined as \>/+2x ULN) 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

Locations (1)

Cleveland Clinic

Cleveland, Ohio, United States

RECRUITING

Outcomes

Primary Outcomes

Treatment related adverse events

Number of participants with treatment related adverse events post-injection of 75 million allogeneic bone marrow derived MSC's for the treatment of perianal fistula(s) in the setting of Crohn's disease as assessed by protocol

Time frame: Month 3

Treatment related adverse events

Time frame: Month 12

Secondary Outcomes

Complete clinical healing

Number of participants with complete clinical healing post-injection of 75 million allogeneic bone marrow derived MSC's for the treatment of perianal fistula(s) in the setting of Crohn's disease. Complete Healing is defined as: Radiographic Healing: MRI with an absence of a fluid collection \>2 cm in 3 of 3 dimensions, lack of edema, inflammation or sign of active inflammatory response. A remnant scar of a fistula tract may remain Clinical Healing: 100% cessation of drainage on both clinical exam with deep palpation and per patient report and epithelization of the external fistula opening

Time frame: Month 3

Complete clinical healing

Time frame: Month 12

Central Contacts

Allison Bayles

CONTACT

216-444-0887 ibdstemcelltherapy@ccf.org

Alex VanDenBossche, BSN

CONTACT

215-370=0307 ibdstemcelltherapy@ccf.org

Data from ClinicalTrials.gov

This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.