Darvadstrocel for Crohn's Fistula in the Realworld

Use of Darvadstrocel (Allogenic Stem Cell Therapy) for Crohn's Fistula in Real Clinical Practice: the National Project to Implement Mesenchymal Stem Cell for the Treatment of Perianal Crohn's Fistula (the PRIME Study)

Background Perianal fistula may affect 15-50% of patients with Crohn's disease (CD). Treatment is complex, requiring a multidisciplinary approach. Darvadstrocel (allogeneic mesenchymal cells obtained from lipoaspirates) was approved in 2018 by the European Medicines Agency and Spanish Agency of Medicines and Medical Products as a treatment for fistulas in CD. Recent European Crohn's and Colitis Organisation (ECCO) andSpanish Working Group on Crohn's Disease and Ulcerative Colitis (GETECCU) guidelines state that darvadstrocel is effective with a favourable safety profile,with a strong level of evidence (level 2).With this study we want to see the real efficacy of darvadstrocel in a Spanish population after 6 months of follow-up.

Study Overview

• Status: Completed
• Conditions: Crohn's Fistula
• Intervention / Treatment: Drug: Darvadstrocel

Detailed Description

Methods: The criteria for the Spanish National Health System to fund darvadstrocel treatment are: 1)complex fistula in a patient with CD; 2)failure of conventional and anti-tumour necrosis factor treatment; and 3)absence of collection s>2cm confirmed by pelvic MRI scan at the time of surgery. From November 2019 to April 2022, 73 patients were treated with darvadstrocel at 14Spanish centres and evaluated clinically and radiologically 6 months after treatment.

• Study Type: Observational
• Enrollment (Actual): 73

Contacts and Locations

Study Locations

• Spain
  • Madrid, Spain, 28040
    • Fundacion Jimenez Diaz

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Probability Sample

Study Population

All patients with complex anorectal fistulas associated with CD treated with darvadstrocel in the 14 dedicated centres from 29 November 2019 to 21 April2022 will be enrolled in the study.

Description

Inclusion Criteria:

• 1)complex fistula in a patient with CD
• 2)failure of conventional and anti-tumour necrosis factor treatment
• 3)absence of collection s>2cm confirmed by pelvic MRI scan at the time of surgery

Exclusion Criteria:

• Patients with a diagnosis of rectovaginal fistula, rectal stenosis or severe active proctitis were excluded

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Group of patients treated with darvadstrocel	Drug: Darvadstrocel; expanded allogeneic adipose-derived mesenchymal stem cell therapy for the treatment of complex perianal fistulas

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Participants with clinical remission	Clinical remission was defined as closure of all previously draining external openings treated with darvadstrocel, despite gentle finger compression	6-month
Number of Participants with clinical response	Clinical response was defined as closure of ≥50% of previously draining external openings treated with darvadstrocel, despite gentle finger compression	6-month
Number of Participants with clinical radiographic healing	Complete radiographic healing was defined as an MRI result with no fluid collection >2cm in all dimensions, no oedema and no inflammation nor sign of active inflammatory response;a remnant fistula tract scar may remain	6-month
Number of Participants with combined clinical-radiological response	Combined clinical-radiological response, a new concept, was used to describe patients who achieved both clinical remission and complete radiological healing.	6-month

Collaborators and Investigators

• Sponsor: Dolores Herreros Marcos
• Investigators: Principal Investigator: Dolores Herreros, Hospital Fundacion Jimenez Diaz

Study record dates

Study Major Dates

• Study Start (Actual): November 29, 2019
• Primary Completion (Actual): April 21, 2022
• Study Completion (Actual): April 21, 2022

Study Registration Dates

• First Submitted: February 10, 2023
• First Submitted That Met QC Criteria: February 21, 2023
• First Posted (Estimate): February 23, 2023

Study Record Updates

• Last Update Posted (Estimate): February 23, 2023
• Last Update Submitted That Met QC Criteria: February 21, 2023
• Last Verified: February 1, 2023

More Information

Terms related to this study

• Keywords: Fistula-in-ano; Crohn's disease; Cell therapy; Compassionate use; Adipose-derived stem cells
• Additional Relevant MeSH Terms: Pathological Conditions, Anatomical; Fistula
• Other Study ID Numbers: Darvastrocel Prime Study 001

Drug and device information, study documents

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