TK011: Prospective, non-interventional, post-authorisation safety study (PASS) of Zalmoxis prescribed in patients undergoing haploidentical hematopoietic stem cell transplantation for high-risk hematological malignancies

Study type

• Study type:
  Non-interventional study
• Scope of the study:
  Assessment of risk minimisation measure implementation or effectiveness

  Effectiveness study (incl. comparative)

Study drug and medical condition

• Name of medicine:
  ZALMOXIS
• Medical condition to be studied:
  Stem cell transplant

Population studied

• Age groups:
  • Adult and elderly population (≥18 years)

    • Adults (18 to < 65 years)

      • Adults (18 to < 46 years)
      • Adults (46 to < 65 years)
    • Elderly (≥ 65 years)

      • Adults (65 to < 75 years)
      • Adults (75 to < 85 years)
      • Adults (85 years and over)
• Estimated number of subjects:
  240

Study design details

• Main study objective:
  To characterize and determine the incidence of events of interest identified as important or potentially important risks in pts who receive Zalmoxis after haploidentical transplantation in a post-marketing setting and placing into context with the background incidence of these events in a non-randomized, concurrent control group of pts undergoing haploidentical transplantation without Zalmoxis
• Outcomes:
  GvHD, severe systemic infection, CMV/EBV, febrile neutropenia, hepatic failure, development of RCR, second cancer, development of immunological events, DMSO-related AEs, concomitant administration of ganciclovir, valganciclovir or immunosuppressive therapy and related AEs, treatment failure of ganciclovir for GvHD control, donor site reaction (local and/or systemic) and any AEs related to Zalmoxis
• Data analysis plan:
  Mean, standard deviation, median, range, quartiles (for continuous data), and counts and percentages (for categorical data) will be calculated for baseline donor/patient/disease-related characteristics and treatments. The overall AE incidence will be summarized in terms of patient counts, percentages and 95% confidence intervals (CIs). Incidence will be computed as the number of patients with event onset in the interval divided by the number of patients in the ITT population. Adverse events will be classified using the MedDRA classification system. The severity of the toxicities will be graded according to the National Cancer Institute common toxicity criteria for adverse events (NCI-CTCAE) version 4.02 whenever possible. Frequency of AEs will be tabulated by MedDRA system organ class and preferred term. In the by-patient analysis, a patient having the same event more than once will be counted only once. AEs will be summarized by worst grade