NCT01144962

Dose-escalating Therapeutic Study of Allogeneic Bone Marrow Derived Mesenchymal Stem Cells for the Treatment of Fistulas in Patients With Refractory Perianal Crohn's Disease

Allogeneic Bone Marrow Derived Mesenchymal Stem Cells for the Treatment of Fistulas in Patients With Refractory Perianal Crohn's Disease

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 21 (actual)
• Sponsor: Leiden University Medical Center · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

In a dose escalation study we will determine the safety and preliminary efficacy of allogeneic bone marrow mesenchymal stem cells (bmMSCs) in the induction of response for active fistulizing Crohn's Disease (CD).

Detailed description

Despite the introduction of anti-TNFa (tumor necrosis factor alpha) therapy, perianal disease still accounts for a high rate of morbidity in patients diagnosed with CD. Recently, a phase II multicenter randomized study was reported showing that expanded adipose tissue derived mesenchymal stem cells (atMSCs) in combination with fibrin glue was an effective and safe treatment for complex perianal fistula. However, dose escalation of allogeneic bone marrow (bm) MSCs for the local treatment of perianal fistulas has not been studied. In this study, three escalating doses will be tested in a total of three cohorts. MSC implantation will be preceded by surgical localization, curettage of the fistulous tract and closure of the internal opening. Per cohort, patients will be randomized in a 5:2 fashion to receive either 10x10\^6 (cohort 1), 30x10\^6 (cohort 2) or 90x10\^6 (cohort 3) bmMSCs or no cells (control group). The primary endpoint will be assessed at week 12: i) the number of adverse and serious adverse events and ii) a reduction in the number of draining fistulas, which is defined as absence of discharge and absence of collections of ≥2 cm directly related to the treated fistulas tracts as measured by MRI.

Conditions

• Crohn's Disease
• Fistula

Interventions

Type	Name	Description
PROCEDURE	Localization, curettage of the fistulous tract and closure of the internal opening without MSC injection.	Patients will undergo surgical localization, curettage of the fistulous tract and closure of the internal opening, without injection of MSCs.
PROCEDURE	Localization, curettage of the fistulous tract and closure of the internal opening with local MSC injection.	Patients will undergo surgical localization, curettage of the fistulous tract and closure of the internal opening, with local injection of indicated dose of MSCs

Timeline

• Start date: 2010-06-01
• Primary completion: 2014-09-01
• Completion: 2014-12-01
• First posted: 2010-06-16
• Last updated: 2014-12-30

Locations

1 site across 1 country: Netherlands