A Prospective, Non-interventional, Observational Post-authorisation Study to Assess the Late-effects of Patients who have received Unituxin (ALIVIO)

Study type

• Study topic:
  Disease /health condition

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

  Effectiveness study (incl. comparative)
• Data collection methods:
  Secondary use of data

Non-interventional study

• Non-interventional study design:
  Cohort

  Other
• Non-interventional study design, other:
  Prospective, observational cohort registry

Study drug and medical condition

• Anatomical Therapeutic Chemical (ATC) code:
  (L01XC16) dinutuximab beta

  dinutuximab beta
• Medical condition to be studied:
  Neuroblastoma

Population studied

• Short description of the study population:
  High-risk neuroblastoma patient for whom a decision has been made to commence Unituxin immunotherapy.; Patients who are participating in study DIV-NB-401, a Post-marketing Study to Further Assess the Immunogenicity and Safety of Unituxin in High-Risk Neuroblastoma Patients werel also eligible to participate in this study.
• Age groups:
  Infants and toddlers (28 days – 23 months)

  Children (2 to < 12 years)

  Adolescents (12 to < 18 years)
• Special population of interest:
  Other
• Special population of interest, other:
  Neuroblastoma patients
• Estimated number of subjects:
  590

Study design details

• Main study objective:
  To evaluate the late-effects associated with the central and peripheral nervous system, growth and endocrine development, auditory, visual, hepatic, renal, lower urinary tract, cardiac, respiratory, skeletal, second and secondary malignancies in patients who received Unituxin for neuroblastoma and remain event-free for at least 5 years since the start of Unituxin immunotherapy.
• Outcomes:
  The late effects associated with the relevant systems and second/secondary malignancies will be analysed in the Safety Set of patients by comparing the characteristics at 12 months to 5 years tothe characteristics at baseline using descriptive statistics. Event-free survival (EFS) and Overall Survival (OS) will be assessed every 6 months for up to 5 years following treatment. EFS will be calculated as the time from start of Unituxin until the first occurrence of the events of relapse, PD, secondary malignancy, or death. If no event occurs, the date of last contact will be used. OS will be calculated as the time from start Unituxin until death.
• Data analysis plan:
  All analyses will be performed using SAS for Windows statistical software using validated implementations of each application or SAS custom programming. The data from all centres will be combined, so that an adequate number of patients will be available for analyses. Summary tabulations will be presented that will display the number ofobservations, mean, standard deviation, median, minimum and maximum for continuous variables, and the number and percentage per category for categorical or ordered categorical data. In addition, two sided 95% confidence intervals will be calculated for all outcomes when possible.