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

Study Overview
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Brief Summary
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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
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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.

Official Title
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A Prospective Study for the Assessment of the Long-term Safety and Efficacy of Cx401 in Patients Taking Part in the FATT-1 Trial

Conditions
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Complex Perianal Fistula

Intervention / Treatment
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* Drug: ASCs
* Drug: Fibrin glue
* Drug: ASCs


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| ASCs<br>Patients randomized to experimental treatment (ASC transplantation) in the FATT-1 randomized controlled trial [ClinicalTrials.gov identifier: NTC00475410] | Drug: ASCs<br>* Intralesional injection of adult-stem-cells at a dose of 20 and 40 million.<br>* Other names: Ontaril®;|
| Fibrin glue<br>Patients randomized to the control treatment (application of fibrin glue) in the FATT-1 randomized controlled trial [ClinicalTrials.gov identifier: NTC00475410] | Drug: Fibrin glue<br>* After curettage, the fistulous tract was sealed with fibrin glue.<br>|
| ASCs + Fibrin Glue<br>Patients randomized to the control treatment (application of fibrin glue) + intralesional injection of ASCs in the FATT-1 randomized controlled trial [ClinicalTrials.gov identifier: NTC00475410] | Drug: ASCs<br>* Intralesional injection of adult-stem-cells at a dose of 20 and 40 million in combination with fibrin glue.<br>* Other names: Ontaril®;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Cumulative incidence of adverse events (clinical or laboratory) attributed to the study therapy in the preceding FATT-1 randomized trial (CX401 or fibrin glue) |  | 6 months (since last visit in FATT-1 trial) | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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) |  | 6 months (since last visit of FATT-1 trial) | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Complex perianal fistula, Adipose-derived adult stem cells, Fibrin glue