Efficacy of Cx601 (Darvadstrocel) for the Treatment of Perianal Fistulizing Crohn's Disease

Efficacy of Cx601 (Darvadstrocel) for the Treatment of Perianal Fistulizing Crohn's Disease - a Prospective Nationwide Multicentre Study

The use of mesenchymal stem cells is considered a novel and promising therapeutic option for patients with perianal fistulizing Crohn's disease. However, due to limited clinical data, this multicentre, nationwide study aimed to assess its clinical efficacy in closing complex anal fistula.

Study Overview

• Status: Completed
• Conditions: Crohn Disease; Fistula Perianal
• Intervention / Treatment: Drug: Darvadstrocel

Detailed Description

Fifteen applications in 14 patients (3 male, 11 female) with complex anal fistulas treated in three tertiary hospitals in Austria were included between October 2018 and April 2021. Injection of 120 million allogeneic expanded adipose-derived mesenchymal stem cells (Cx601 - Darvadstrocel, Alofisel®) was performed in each patient. Closure of the external fistula opening without secretion by finger compression was defined as success.

• Study Type: Observational
• Enrollment (Actual): 14

Contacts and Locations

Study Locations

• Austria
  • Vienna, Austria, 1090
    • Medical University of Vienna

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 14 years to 86 years (Adult, Older Adult)
• Accepts Healthy Volunteers: N/A
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

14 patients with Crohn's disease and complex fistula got enrolled in this study.

Description

Inclusion Criteria:

• patients aged 18 and older who had a non-active or mildly active luminal CD with complex fistula with a maximum of two internal and three external fistulas

Exclusion Criteria:

• rectovaginal fistulas
• rectal and/or anal stenosis
• active proctitis
• diverting stomas
• an abscess (<2cm) that was not drained at the fistula preperation visit

Study Plan

How is the study designed?

Design Details

• Observational Models: Case-Only
• Time Perspectives: Prospective

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
patients with Crohn's disease and complex perianal fistula; 14 patients with Crohn's disease refractory to standard treatment for complex perianal fistula got enrolled in this study.	Drug: Darvadstrocel; Injection of 120 million allogeneic expanded adipose-derived mesenchymal stem cells around the fistula opening and around the fistula tract; Other Names: Alofisel

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Fistula Closure	The study's primary endpoint was established at week 52 with clinically assessed fistula closure.	1 year

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Evaluation of perianal disease	At every follow up visit the perianal disease was evaluated using the PDAI (Perianal disease activity index)	1 year
Evaluation of Crohn's disease	Crohn's disease was evaluated by using the HBI (Harvey Bradshaw Index)	1 year

Collaborators and Investigators

• Sponsor: Medical University of Vienna
• Investigators: Study Chair: Stefan Riss, Prof.MD, Medical University of Vienna, Head of Pelvic Floor surgery

Study record dates

Study Major Dates

• Study Start (Actual): October 1, 2018
• Primary Completion (Actual): December 1, 2021
• Study Completion (Actual): December 1, 2021

Study Registration Dates

• First Submitted: April 3, 2022
• First Submitted That Met QC Criteria: April 3, 2022
• First Posted (Actual): April 11, 2022

Study Record Updates

• Last Update Posted (Actual): April 11, 2022
• Last Update Submitted That Met QC Criteria: April 3, 2022
• Last Verified: April 1, 2022

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Digestive System Diseases; Gastrointestinal Diseases; Gastroenteritis; Intestinal Diseases; Pathological Conditions, Anatomical; Inflammatory Bowel Diseases; Fistula; Crohn Disease
• Other Study ID Numbers: 1682/2018

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No
• IPD Plan Description: IPD can be shared just in anonymised form in case of an ethical agreement with other researches.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No