Safety and Efficacy of Adipose-Derived Stem Cells to Treat Complex Perianal Fistulas Patients With Crohn's Disease (FATT)
|Complex Perianal Fistula Crohn Disease||Drug: Expanded autologous adipose-derived adult stem cells (eASCs) Drug: Placebo||Phase 3|
|Study Design:||Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Single Blind (Outcomes Assessor)
Primary Purpose: Treatment
|Official Title:||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|
- 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 ]
|Study Start Date:||December 2008|
|Study Completion Date:||February 2010|
|Primary Completion Date:||February 2010 (Final data collection date for primary outcome measure)|
Intralesional dose of 20 million cells at baseline with a possible second administration of 40 million cells in case of incomplete fistula closure following week 12 assessment.
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 Surgey standards. Patients will receive an intralesional dose of 20 million cells at baseline. 50% of the cell susupension 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.
Sham Comparator: Placebo
Instillation of saline solution into the fistulous tract, following identical tract preparation process as for the investigational treatment group.
Administration will be preceded by localization and closure of the internal opening following Major Ambulatory Surgey 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.
Other Name: Normale saline solution
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.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01378390
|University of Vienna|
|Academic Medical Center|
|Hospital Germans Trias i Pujol|
|Badalona, Barcelona, Spain, 08916|
|Hospital de Sagunto|
|Sagunto, Valencia, Spain, 46520|
|Hospital Reina Sofia|
|Córdoba, Spain, 14004|
|Complejo Hospitalario de León|
|Léon, Spain, 24071|
|Hospital Universitario de la Princesa|
|Madrid, Spain, 28006|
|Hospital Gregorio Marañón|
|Madrid, Spain, 28007|
|Hospital Ramón y Cajal|
|Madrid, Spain, 28034|
|Hospital Puerta de Hierro|
|Madrid, Spain, 28035|
|Hospital Clínico San Carlos|
|Madrid, Spain, 28040|
|Complejo Hospitalario de Pontevedra|
|Pontevedra, Spain, 36001|
|Principal Investigator:||Damián García-Olmo, MD||General Surgery Department, Hospital Universitario La Paz|