Alofisel-4001: Postauthorization Safety Study of the Long-Term Safety and Efficacy of Repeat Administration of Darvadstrocel in Patients With Crohn’s Disease and Complex Perianal Fistula

Study type

• Study topic:
  Human medicinal product
• Study type:
  Clinical trial

Clinical trials

• Clinical trial randomisation:
  Non-randomised clinical trial

Study drug and medical condition

• Name of medicine:
  ALOFISEL
• Medical condition to be studied:
  Crohn's disease
• Additional medical condition(s):
  Complex Perianal Fistula

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:
  50

Study design details

• Main study objective:
  To evaluate the long-term safety of repeat administration of darvadstrocel in subjects with CD and complex perianal fistula by evaluation of adverse events (AEs), serious adverse events (SAEs), adverse events of special interest (AESIs), and pregnancy.
• Outcomes:
  Primary outcomes of the study are assessments of safety parameters through treatment-emergent adverse events (TEAEs), TE serious AEs, Pregnancy, TEAE of special interests- (Immunogenicity/alloimmune reactions, Hypersensitivity, Transmission of infectious agents, Tumorgenicity (applying to malignant tumors only), Ectopic tissue formation, Medication errors), Secondary outcomes included combined remission, clinical remission, clinical response, relapse and time to relapse, new perianal abscess and Change in scores of discharge and pain items of Perianal Disease Activity Index (PDAI) score after darvadstrocel repeat administration.
• Data analysis plan:
  Count and percentage of participants with AEs, SAEs and AESIs will be summarized by system organ class and preferred term using MedDRA terminology for the overall study population and for relevant subgroups. ; All efficacy outcomes will be summarized by visit, as applicable. Proportion of participants with each of following outcomes, along with 95% 2-sided confidence intervals will be provided by visit:1) Clinical remission, 2) Clinical response,3) Combined remission,4) Reopening of any of the treated external openings with active drainage,5) New perianal abscess in treated fistula. In addition, at Weeks 24, 52, 104, and 156, the proportion of participants who changed in status since the previous assessment will be provided. Change from baseline in PDAI discharge and pain subscores will be summarized descriptively by visit. Among participants who achieve combined probability of relapse across time will be estimated using Kaplan-Meier estimator.