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.
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.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
SINGLE
Enrollment
56
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.
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.
University of Vienna
Vienna, Austria
Academic Medical Center
Amsterdam, Netherlands
Hospital Germans Trias i Pujol
Badalona, Barcelona, Spain
Percentage of subjects in whom the external openings of the treated perianal fistula have closed
Time frame: 24 weeks
Percentage of subjects in whom the external openings of the treated perianal fistula have closed
Time frame: 12 weeks
Changes over time in the Perianal Disease Activity Index
Time frame: 4, 10, 12, 16, 22 and 24 weeks
Changes over time in the Crohn's Disease Activity Index
Time frame: 10 and 22 weeks
Changes over time in the MRI Score of Severity (MSS)
Time frame: 12 and 24 weeks
Quality of life assessment using the SF-36 questionnaire
Time frame: 12 and 24 weeks
Percentage of subjects for whom surgery is avoided
Time frame: 24 weeks
Adverse events
Time frame: 4, 10, 12, 16, 22 and 24 weeks
Clinically relevant variations in vital signs
Time frame: 4, 10, 12, 16, 22 and 24 weeks
Clinically relevant variations in laboratory tests
Time frame: 4, 10, 12, 16, 22 and 24 weeks
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Hospital de Sagunto
Sagunto, Valencia, Spain
Hospital Reina Sofia
Córdoba, Spain
Complejo Hospitalario de León
Léon, Spain
Hospital Universitario de la Princesa
Madrid, Spain
Hospital Gregorio Marañón
Madrid, Spain
Hospital Ramón y Cajal
Madrid, Spain
Hospital Puerta de Hierro
Madrid, Spain
...and 2 more locations