Adakveo (crizanlizumab) PRegnancy outcomes Intensive Monitoring (PRIM)

Study type

• Study topic:
  Disease /health condition

  Human medicinal product
• Study type:
  Non-interventional study
• Scope of the study:
  Safety study (incl. comparative)
• Data collection methods:
  Secondary use of data

Non-interventional study

• Non-interventional study design:
  Cohort

  Other
• Non-interventional study design, other:
  Intensive monitoring schemes

Study drug and medical condition

• Medicinal product name:
  ADAKVEO
• Study drug International non-proprietary name (INN) or common name:
  CRIZANLIZUMAB
• Anatomical Therapeutic Chemical (ATC) code:
  (B06AX01) crizanlizumab

  crizanlizumab
• Medical condition to be studied:
  Sickle cell disease

Population studied

• Short description of the study population:
  All prospective and retrospective pregnancy cases exposed to crizanlizumab during pregnancy or 105 days before LMP reported to the Novartis global safety database were eligible for the PRIM program.
• Age groups:
  • Adolescents (12 to < 18 years)
  • Adults (18 to < 46 years)
  • Adults (46 to < 65 years)
• Special population of interest:
  Pregnant women
• Estimated number of subjects:
  500

Study design details

• Study design:
  The crizanlizumab PRIM study is a non-interventional study (NIS) based on secondary use of data from a set of targeted follow-up (TFU) checklists with structured follow-up on pregnancies reported to the Novartis global safety database. This study is single-arm, descriptive in nature
• Main study objective:
  The primary objective was to estimate the proportion of pregnancies resulting in fetal loss (intrauterine death resulting in stillbirth, spontaneous abortion, or induced termination), among pregnant women exposed to crizanlizumab within 105 days prior to LMP or at any time during pregnancy
• Outcomes:
  Fetal loss (intrauterine death resulting in stillbirth, spontaneous abortion, or induced termination), Congenital malformations
• Data analysis plan:
  Data analysis will focus on the prospectively reported pregnancy cases. Descriptive analysis will be performed for prospective pregnancy cases including case disposition and maternal characteristics by providing the number and percentage of pregnancies in each category. Distributions of continuous variables will be summarized with means, standard deviations, medians, and range. Categorical variables will be summarized with proportions. Numbers and proportions (with 95% confidence intervals) for pregnancy outcomes will be reported. Adverse outcomes will be examined by trimester of exposure and concomitant exposure to hydroxyurea/hydroxycarbamide. Case details will be provided separately for retrospective cases.