Clinical trial NCT01020825: Long-term Safety and Efficacy of Adipose-derived Stem Cells to Treat Complex Perianal Fistulas in Patients Participating in the FATT-1 Randomized Controlled Trial
Official title: A Prospective Study for the Assessment of the Long-term Safety and Efficacy of Cx401 in Patients Taking Part in the FATT-1 Trial
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Complex Perianal Fistula
Keywords: Complex perianal fistula
Adipose-derived adult stem cells
Fibrin glue
Lead sponsor: Takeda
Sponsor class: INDUSTRY
First posted: 2009-11-26
Enrollment: 148
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: OTHER
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients that received at least one treatment dose in study Cx401/FATT1

Brief summary: The purpose of this extension is to investigate and confirm the long-term (6 months) safety and efficacy of the preceding FATT-1 trial \[ClinicalTrials.gov identifier: NTC00475410\], which studied patients with perianal fistula treated having received adipose-derived adult stem cell (ASC)and/or fibrin glue.

Detailed description: Complex perianal fistulas are a source of great distress for suffers. Treatment 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.

This study aims to evaluate the safety and efficacy of patients having participated within a preceding multicenter, placebo-controlled, phase 3 study \[ClinicalTrials.gov identifier: NTC00475410\]. The present extension aims to collect safety and efficacy data for up to 12 month 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.

Eligibility criteria: Inclusion Criteria:

* At least 1 dose of the treatment assigned in the FATT-1 trial
* Informed consent

Exclusion Criteria:

* Other experimental drugs other than Cx401 during the follow-up period

Primary outcomes:
measure: Cumulative incidence of adverse events (clinical or laboratory) attributed to the study therapy in the preceding FATT-1 randomized trial (CX401 or fibrin glue)
timeFrame: 6 months (since last visit in FATT-1 trial)
Secondary outcomes:
measure: Closure of the fistula (defined as suppuration through the external opening of the fistula spontaneously and on pressure, complete re-epithelization of the external opening in the clinical evaluation and absence of collections >2 cm in MRI)
timeFrame: 6 months (since last visit of FATT-1 trial)
Other outcomes:
Locations:
city: Terrasa
country: Spain
facility: Hospital Mutua de Terrasa
state: Barcelona
city: Santander
country: Spain
facility: Hospital Universitario Marqués de Valdecilla
state: Cantabria
city: Fuenlabrada
country: Spain
facility: Hospital de Fuenlabrada
state: Madrid
city: Sagunto
country: Spain
facility: Hospital de Sagunto
state: Valencia
city: Girona
country: Spain
facility: Hospital Doctor Josep Trueta
city: Madrid
country: Spain
facility: Hospital Gregorio Marañón
city: Madrid
country: Spain
facility: Hospital Clínico San Carlos
city: Madrid
country: Spain
facility: Hospital 12 de Octubre
city: Madrid
country: Spain
facility: Hospital Universitario La Paz
city: Pamplona
country: Spain
facility: Clínica Universitaria Navarra
city: Salamanca
country: Spain
facility: Hospital Universitario de Salamanca
city: Seville
country: Spain
facility: Hospital Universitario Nuestra Señora de Valme
city: Tarragona
country: Spain
facility: Hospital Universitari Joan XXIII
city: Valencia
country: Spain
facility: Hospital General Univeritario de Valencia
city: Zaragoza
country: Spain
facility: Hospital Clínico Universitario "Lozano Blesa"
Study officials:
affiliation: General Surgery Department, Hospital Universitario La Paz
name: Damián García-Olmo, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Garcia-Olmo D, Garcia-Arranz M, Garcia LG, Cuellar ES, Blanco IF, Prianes LA, Montes JA, Pinto FL, Marcos DH, Garcia-Sancho L. Autologous stem cell transplantation for treatment of rectovaginal fistula in perianal Crohn's disease: a new cell-based therapy. Int J Colorectal Dis. 2003 Sep;18(5):451-4. doi: 10.1007/s00384-003-0490-3. Epub 2003 May 20.
citation: Garcia-Olmo D, Garcia-Arranz M, Herreros D, Pascual I, Peiro C, Rodriguez-Montes JA. A phase I clinical trial of the treatment of Crohn's fistula by adipose mesenchymal stem cell transplantation. Dis Colon Rectum. 2005 Jul;48(7):1416-23. doi: 10.1007/s10350-005-0052-6.
citation: Garcia-Olmo D, Herreros D, Pascual M, Pascual I, De-La-Quintana P, Trebol J, Garcia-Arranz M. Treatment of enterocutaneous fistula in Crohn's Disease with adipose-derived stem cells: a comparison of protocols with and without cell expansion. Int J Colorectal Dis. 2009 Jan;24(1):27-30. doi: 10.1007/s00384-008-0559-0. Epub 2008 Aug 12.
citation: Garcia-Olmo D, Garcia-Arranz M, Herreros D. Expanded adipose-derived stem cells for the treatment of complex perianal fistula including Crohn's disease. Expert Opin Biol Ther. 2008 Sep;8(9):1417-23. doi: 10.1517/14712598.8.9.1417.
citation: Herreros MD, Garcia-Arranz M, Guadalajara H, De-La-Quintana P, Garcia-Olmo D; FATT Collaborative Group. Autologous expanded adipose-derived stem cells for the treatment of complex cryptoglandular perianal fistulas: a phase III randomized clinical trial (FATT 1: fistula Advanced Therapy Trial 1) and long-term evaluation. Dis Colon Rectum. 2012 Jul;55(7):762-72. doi: 10.1097/DCR.0b013e318255364a.