Study of the 12-month Efficacy of Stem Cell Injection in Crohn's Disease With Complex Ano-perineal Fistula (ECESMAC)

October 31, 2022 updated by: Groupe Hospitalier Paris Saint Joseph

Ano-perineal (or ano-rectal) fistulas are a frequent and major complication of Crohn's disease. Indeed, they indicate a greater severity of the disease. In addition, they have a major impact on the quality of life of patients. Finally, these fistulas are difficult to cure, especially when they are complex. Indeed, medical treatment with anti-TNF drugs can only cure them in one third of cases. Various obturation treatments have been proposed in addition to medical treatment such as advancement flap, injection of biological glue, placement of a plug, intersphincter ligation of the fistulous path or laser but with still insufficient healing rates.

The injection of mesenchymal stem cells of adipose origin was first proposed for this indication in 2003. Since then, numerous studies have been published on the subject. Most importantly, an international multicenter randomized double-blind phase 3 controlled trial demonstrated the superiority of allogeneic stem cells over placebo with a therapeutic effect maintained at one year follow-up. The trial involved selected patients with Crohn's disease and one or more complex ano-perineal fistulas refractory to conventional treatments. This treatment has a high cost and its practical modalities are constraining (manufacturing, delivery, constrained time between the factory and the injection). However, this study is the first to have demonstrated in such a rigorous way the effectiveness of a local treatment on complex ano-perineal fistulas of Crohn's disease. These allogeneic mesenchymal stem cells have therefore been granted marketing authorization in Europe and have been commercialized in several European countries including France during the summer of 2020 under the trade name ALOFISEL®. They are indicated for complex ano-perineal fistulas that have not responded to at least one conventional biological treatment, in the context of inactive or less active Crohn's disease.

The main objective of the study is to evaluate the rate of "deep" remission (i.e. clinical and radiological) at 12 months of follow-up in the first patients treated in France with ALOFISIEL® after its marketing. The evaluation is performed at 12 months because it seems that the result is consolidated after this period.

Study Overview

Status

Active, not recruiting

Conditions

Study Type

Observational

Enrollment (Actual)

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

France
- - Paris, France, 75014
    - Groupe Hospitalier Paris Saint-Joseph
  - Rennes, France
    - CHU de Rennes

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (ADULT, OLDER_ADULT)

Accepts Healthy Volunteers

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

Study Population

Patient with inactive or minimally active Crohn's disease associated with one or more complex ano-perineal fistula(s) that has not responded to at least one conventional biologic therapy, managed for their fistula(s) by mesenchymal stem cell injection between July 1, 2020 and July 30, 2021 at one of the participating centers.

Description

Inclusion Criteria:

Patient whose age ≥ 18 years
Patient with inactive or minimally active Crohn's disease associated with one or more complex ano-perineal fistula(s) that has not responded to at least one conventional biologic therapy
Patients managed for their fistula(s) by mesenchymal stem cell injection between July 1, 2020 and July 30, 2021 at one of the participating centers
Patients who understand the French language

Exclusion Criteria:

Patients under guardianship or curatorship
Minor patients
Pregnant women
Patient deprived of liberty
Patient under court protection
Patients who object to the use of their data for this research
Patients with a simple anal fistula
Patients with anorecto-vaginal fistula
Patients with anal canal stenosis that cannot be crossed by the index finger
Patients who have not previously received medical treatment (biotherapy +/- immunosuppressant)

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Deep remission rates Time Frame: Month 12	This outcome corresponds to the Rate of patients in deep remission at 12 months defined by the combination of: absence of pain and oozing closure of the secondary orifice(s) with the absence of discharge on digital pressure absence of abscesses absence of a collection of more than 20 mm on MRI.	Month 12

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
12-month clinical remission rate only Time Frame: Month 12	This outcome corresponds to the rate of patients in clinical remission alone at 12 months defined by the combination an absence of pain and oozing closure of the secondary orifice(s) with the absence of discharge on digital pressure absence of abscesses	Month 12
Primary clinical failure rate Time Frame: Month 3	This outcome corresponds to the rate of patients with primary treatment failure defined as non-closure of secondary orifice(s) with persistent flow on digital pressure at 3-month follow-up.	Month 3
Evolution of the severity of the disease at 12 months Time Frame: Month 12	This outcome corresponds to the complication rates and types at 12 months.	Month 12

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Groupe Hospitalier Paris Saint Joseph

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (ACTUAL)

July 2, 2021

Primary Completion (ACTUAL)

September 2, 2022

Study Completion (ANTICIPATED)

December 31, 2024

Study Registration Dates

First Submitted

December 31, 2021

First Submitted That Met QC Criteria