Clinical trial NCT05742100: Darvadstrocel for Crohn's Fistula in the Realworld
Official title: 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)
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Crohn's Fistula
Keywords: Adipose-derived stem cells
Cell therapy
Compassionate use
Crohn's disease
Fistula-in-ano
Lead sponsor: Instituto de Investigación Sanitaria de la Fundación Jiménez Díaz
Sponsor class: OTHER
First posted: 2023-02-23
Enrollment: 73
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: RETROSPECTIVE
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.

Brief summary: 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.

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.

Eligibility criteria: 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

Primary outcomes:
measure: Number of Participants with clinical remission
timeFrame: 6-month
measure: Number of Participants with clinical response
timeFrame: 6-month
measure: Number of Participants with clinical radiographic healing
timeFrame: 6-month
measure: Number of Participants with combined clinical-radiological response
timeFrame: 6-month
Secondary outcomes:
Other outcomes:
Locations:
city: Madrid
country: Spain
facility: Fundacion Jimenez Diaz
Study officials:
affiliation: Hospital Fundación Jiménez Diaz
name: Dolores Herreros
role: PRINCIPAL_INVESTIGATOR
References: