INSPECT: A retrospectIve chart review study evaluating the longer-term effectiveness of darvadstrocel in patients who completed ADMIRE-CD

Study type

• Study topic:
  Human medicinal product

  Disease /health condition
• Study type:
  Non-interventional study
• Scope of the study:
  Effectiveness study (incl. comparative)
• Data collection methods:
  Secondary use of data

Non-interventional study

• Non-interventional study design:
  Other
• Non-interventional study design, other:
  Multi-centre, retrospective chart review study

Study drug and medical condition

• Study drug International non-proprietary name (INN) or common name:
  DARVADSTROCEL
• Medical condition to be studied:
  Crohn's disease

  Anal fistula

Population studied

• Short description of the study population:
  Patients with Crohn’s disease (CD), who received darvadstrocel or placebo and completed at least 52 weeks of follow-up in the ‘Adipose Derived Mesenchymal Stem Cell for Induction of Remission in Perianal Fistulising Crohn’s Disease (ADMIRE-CD)’ clinical trial.; Patients were excluded if they did not provide informed consent or if there was no information on patient care within the 104 weeks INSPECT study period.
• 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)
• Special population of interest:
  Immunocompromised
• Estimated number of subjects:
  131

Study design details

• Main study objective:
  To evaluate the following over 52 and 104 weeks post-trial completion: time in remission and proportion of patients who sustained remission, incidence of relapse, new fistula occurrence and response, remission and relapse status of new onset fistula, disease activity, medical treatment patterns, surgical and non-surgical procedures and healthcare resource use, AESIs.
• Outcomes:
  The primary outcomes include clinical response, clinical remission, perianal abscess, rescue medication for perianal fistulising disease, relapse of perianal fistula, development of new perianal fistula, perianal and luminal disease activity, treatment patterns, surgical and non-surgical procedures, medical healthcare resource utilization and AESIs.
• Data analysis plan:
  A statistical analysis plan will be finalized prior to any data analyses being performed defining all analytic populations and definitions of derived variables. Analyses will be reported in descriptive manner and compare outcomes between study arms (darvadstrocel versus control). Data from the trial will not be re-analysed and will be directly integrated from clinical trial datasets. Variables from medical records will be collected to assess outcomes during 104-weeks chart review study period. Continuous data will be described by mean, standard deviation, median, interquartile range, minimum, maximum, and number of unknown/missing observations and categorical variables will be described by frequency and percentages(when applicable, 2-sided 95 % confidence interval CI will be provided). Time-to-event data will be analysed using survival analysis techniques. Regression will be used to analyse potential predictors of and/or variables associated with response and remission.