NCT01378390

Safety and Efficacy of Adipose-Derived Stem Cells to Treat Complex Perianal Fistulas Patients With Crohn's Disease

Randomized, Single-blind, Placebo Controlled Multicenter Phase III Study to Assess the Efficacy and Safety of Expanded Autologous Adipose-derived Stem Cells (ASCs) (CX-401), for Treatment of Complex Perianal Fistulas in Perianal Crohn's Disease

• Status: Terminated
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 56 (actual)
• Sponsor: Tigenix S.A.U. · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to determine safety and efficacy of eASCs (expanded adult stem cells) for treatment of complex perianal fistulas in patients with Crohn's disease.

Detailed description

Complex perianal fistulas are a source of great distress for suffers. In Crohn's disease, options are limited, and surgery is often associated with incontinence and recurrence. The biological properties of stem cells derived from adult tissues make them candidates for the treatment of pathologies requiring tissue regeneration or in diseases where the healing process is altered. A proof-of-concept study, a phase 1 study and a phase 2 study \[Clinicaltrials.gov identifier: NCT00115466\] have all suggested that this approach is promising, even in patients whose fistula is associated with Crohn's disease. The present multicenter, placebo-controlled, phase 3 study aims to confirm the efficacy and safety shown in the previous phase 1 and phase 2 studies in the treatment of complex perianal fistulas in patients with Crohn's disease after 24 weeks from initial administration. Fistula closure is defined as absence of suppuration through the external orifice with complete re-epithelization of the external orifice and absence of collections \>2cm directly related to the fistula tract treated, as measured by MRI.

Conditions

• Complex Perianal Fistula
• Crohn Disease

Interventions

Type	Name	Description
DRUG	Expanded autologous adipose-derived adult stem cells (eASCs)	Administration will be preceded by localization and closure of the internal opening. Cell treatment injection will be performed following Major Ambulatory Surgery standards. Patients will receive an intralesional dose of 20 million cells at baseline. 50% of the cell suspension will be placed into the fistula walls of the internal opening, with the remaining 50% being injected across the wall tracts of the target fistula. Patients without healing (complete fistula closure) at week 12 will receive a second dose of 40 million cells, using the same treatment approach.
DRUG	Placebo	Administration will be preceded by localization and closure of the internal opening following Major Ambulatory Surgery standards. Subjects will be receiving one treatment cycle with placebo consisting of intralesional instillation of 5 ml saline solution. Patients without healing (complete fistula closure) following week 12 assessment will continue to receive standard of care until the primary assessment of fistula closure at week 24. Patients with incomplete fistula closure at week 24 will be offered the possibility of participating in a separate open-label, single arm study using the ASCs derived from the liposuction procedure.

Timeline

• Start date: 2008-12-01
• Primary completion: 2010-02-01
• Completion: 2010-02-01
• First posted: 2011-06-22
• Last updated: 2019-04-12

Locations

12 sites across 3 countries: Austria, Netherlands, Spain