Stem Cell Therapy: a Compassionate Use Program in Perianal Fistula (RESCeCUF)

Retrospective Evaluation of Stem Cell Therapy: a Compassionate Use Program in Perianal Fistula.

This observational study aims at assessing the safety and efficacy profiles of mesenchymal stem cell, in different formats (SVF, autologous, allogenic), to treat complex perianal fistula according to the Spanish national Compassionate-use law .

Study Overview

• Status: Completed
• Conditions: Perianal Fistula
• Intervention / Treatment: Procedure: Mesenchymal Stem Cell injection

Detailed Description

Under controlled circumstances, and approved by European and Spanish laws, a Compassionate-use Program allows the use of stem-cell therapy for patients with non-healing diseases, mostly complex fistula-in-ano, who do not meet criteria to be included in a clinical trial. Candidates had previously undergone multiple surgical interventions that had failed. The intervention consisted of surgery (with closure of the internal opening or a surgical flap performance), followed by stem cells injection. Three types of cells were used for implant: stromal vascular fraction, autologous expanded adipose-derived or allogenic adipose derived stem cells.

Healing was evaluated at 6th month follow-up. Outcome was classified as partial response or healing. Relapse was evaluated 1 year later. Maximum follow up period was 48 months.

• Study Type: Observational
• Enrollment (Actual): 52

Contacts and Locations

Study Locations

• Spain
  • Madrid, Spain, 28040
    • Instituto Investigación Sanitario Fundación Jiménez Díaz

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 24 years to 70 years (ADULT, OLDER_ADULT)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

We present an observational study, including 52 cases (53% male and 47% female) treated by a compassionate use ASC program. The mean age was 45 years and ranged from 24 to 69 years. There were 42 perianal fistulas, 7 rectovaginal fistulas, 1 urethrorectal fistula, 1 sacral fistula and 1 hidradenitis suppurativa. All the cases had previous failed surgeries. Thirteen of fifty-two (25%) of the cases presented fecal incontinence at the moment of enrollment. 5/52 (9,6%) of the cases presented anal stenosis and 11/52 (21%) had a scaring anus.

Description

Inclusion Criteria:

• patients with complex perianal fistula
• patients who didn't meet criteria of the clinical trial (CT) in development
• foreign patients, who were not allowed to be included in the CT
• patients included in some CT control arms
• failure treatment in patients included in a CT treatment arm as a retreatment

Exclusion Criteria:

• not signature informed consent

Study Plan

How is the study designed?

Number of groups / cohorts

3

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Stromal Vascular fraction; 31 patients were treated with one injection of Stromal Vascular Fraction from adipose tissue obtained by liposuction. Procedure: curettage, closure of the internal opening (IO) and SVF injection in IO (50%) and fistula tract (50%)	Procedure: Mesenchymal Stem Cell injection; curettage, closure of the internal opening (IO) and cell injection in IO (50%) and fistula tract (50%)
Autologous mesenchymal stem cells; 9 patients were treated with one injection of autologous mesenchymal stem cells from adipose tissue. Procedure: curettage, closure of the internal opening (IO) and autologous cell injection in IO (50%) and fistula tract (50%)	Procedure: Mesenchymal Stem Cell injection; curettage, closure of the internal opening (IO) and cell injection in IO (50%) and fistula tract (50%)
Allogenic mesenchymal stem cells; 12 patients were treated with one injection of allogenic mesenchymal stem cells of healthy donors: Procedure: curettage, closure of the internal opening (IO) and allogenic cell injection in IO (50%) and fistula tract (50%)	Procedure: Mesenchymal Stem Cell injection; curettage, closure of the internal opening (IO) and cell injection in IO (50%) and fistula tract (50%)

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of patients with fistula healed	Complete closure of the fistula and epithelization of the external orifice	12 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of adversus events	general adversus events	12 months

Collaborators and Investigators

• Sponsor: Instituto de Investigación Sanitaria de la Fundación Jiménez Díaz
• Investigators: Principal Investigator: Damian Garcia-Olmo, Prof., Instituto de Investigación Sanitaria Fundación Jimenez Díaz

Study record dates

Study Major Dates

• Study Start (ACTUAL): April 1, 2014
• Primary Completion (ACTUAL): January 26, 2018
• Study Completion (ACTUAL): July 26, 2018

Study Registration Dates

• First Submitted: October 26, 2018
• First Submitted That Met QC Criteria: October 29, 2018
• First Posted (ACTUAL): October 31, 2018

Study Record Updates

• Last Update Posted (ACTUAL): November 13, 2018
• Last Update Submitted That Met QC Criteria: November 8, 2018
• Last Verified: October 1, 2018

More Information

Terms related to this study

• Keywords: mesenchymal stem cells; compassionate use; Perianal fistula
• Additional Relevant MeSH Terms: Digestive System Diseases; Gastrointestinal Diseases; Intestinal Diseases; Pathological Conditions, Anatomical; Rectal Diseases; Intestinal Fistula; Digestive System Fistula; Fistula; Rectal Fistula
• Other Study ID Numbers: EO166-18_FJD

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No