Alofisel-5003: An Observational Post-Marketing Registry on the Effectiveness and Safety of Darvadstrocel in Patients With Crohn's Disease and Complex Perianal Fistulas (INSPIRE)

Study type

• Study type:
  Non-interventional study
• Scope of the study:
  Effectiveness study (incl. comparative)

Non-interventional study

• Non-interventional study design:
  Other
• Non-interventional study design, other:
  Registry study

Study drug and medical condition

• Study drug International non-proprietary name (INN) or common name:
  DARVADSTROCEL
• Medical condition to be studied:
  Anal fistula

  Crohn's disease

Population studied

• Age groups:
  Adults (18 to < 46 years)

  Adults (46 to < 65 years)

  Adults (65 to < 75 years)

  Adults (75 to < 85 years)

  Adults (85 years and over)
• Estimated number of subjects:
  800

Study design details

• Main study objective:
  The primary objective of the study is to evaluate the real-world clinical effectiveness and safety of darvadstrocel in patients with Crohn’s disease (CD) with complex perianal fistulas for a duration of 36 months with the primary evaluation at six months.
• Outcomes:
  The primary outcomes are clinical response and clinical remission. Clinical response is defined as a reduction of at least 50 percent (%) in the number of draining perianal fistula treated with darvadstrocel compared to baseline. Clinical remission is defined as a reduction of all draining perianal fistulae treated with darvadstrocel compared to baseline. The secondary outcomes includes evaluation of combined remission, relapse of perianal fistula and new perianal abscess fistula treated with darvadstrocel, surgical procedures and post-surgery complication status, and clinical/patient assessment of disease activity using subcomponent symptom scores from the Perianal Disease Activity Index (PDAI) and Harvey-Bradshaw Index (HBI).
• Data analysis plan:
  The analysis will be basically descriptive. Continuous variables will be described by mean, standard deviation (SD), median, interquartile range (IQR), minimum, maximum, number of known and number of unknown observations. When relevant, 2-sided 95% confidence intervals (CI) will be provided. Categorical variables will be described by frequency and percentages (n, %). Percentages will be calculated using the most appropriate denominator, in some cases missing data will be included as a separate category depending on the nature of the variable. In all tables with percentages, the denominator will be reported. Subgroup presentations of the outcomes in terms of continuous and categorical variables and survival curves will be provided as appropriate.