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

Phase 1

• Conditions: Perianal Fistula Due to Crohn's Disease (Disorder)
• Interventions: Drug: Mesenchymal stem cells

• Registration Number: NCT04791878
• Lead Sponsor: Amy Lightner

Brief 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.

Detailed Description

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 Timeline

• Study First Submitted: 2021-03-06
• Study First Submitted QC: 2021-03-09
• Study First Posted: 2021-03-10
• Study Start: 2021-04-01
• Status Verified: 2022-04
• Last Update Submitted: 2022-04-01
• Last Update Posted: 2022-04-05
• Primary Completion: 2023-04-01
• Study Completion: 2023-04-01

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 10

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Mesenchymal stem cells	Mesenchymal stem cells	allogeneic bone marrow derived mesenchymal stem cells

Primary Outcome Measures

Name	Time	Method
Treatment related adverse events	Month 12	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

Secondary Outcome Measures

Name	Time	Method
Complete clinical healing	Month 12	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
Lack of response	Month 12	Number of participants with lack of response 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; Lack of response is defined as: Radiographic and Clinical healing which does not meet the threshold for Partial Healing
Worsening of disease	Month 12	Number of participants with worsening of disease 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; Worsening disease is defined as: Radiographic: MRI with a fluid collection \>2 cm in 2 of 3 dimensions, edema, inflammation or sign of active inflammatory response. An increased number of tracts may be seen, or increased branching from the primary tract,; Clinical: Increased drainage per patient report and on clinical exam
Partial clinical healing	Month 12	Number of participants with partial 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; Partial clinical healing is defined as: Radiographic Healing: MRI with an absence of a fluid collection \>2 cm in 2 of 3 dimensions, lack of edema, inflammation or sign of active inflammatory response. A remnant scar of a fistula tract may remain; Clinical healing: Greater than or equal to 50 % cessation of drainage on both clinical exam with deep palpation and per patient report and epithelization of the external fistula opening

Trial Locations

Locations (1)

Cleveland Clinic; 🇺🇸 Cleveland, Ohio, United States