A Post-Authorisation Safety Study Patient Registry of patients with high-risk neuroblastoma being treated with the monoclonal antibody dinutuximab beta (EUSA DB 0001)

Study type

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

  Drug utilisation

  Effectiveness study (incl. comparative)

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Name of medicine:
  QARZIBA
• Name of medicine, other:
  Qarziba 4.5 mg/mL concentrate for solution for infusion
• Medical condition to be studied:
  Neuroblastoma

Population studied

• Age groups:
  Infants and toddlers (28 days – 23 months)

  Children (2 to < 12 years)

  Adolescents (12 to < 18 years)
• Estimated number of subjects:
  125

Study design details

• Main study objective:
  The efficacy and safety of dinutuximab beta will further be evaluated in this registry that will provide information on survival, QOL/Burden of care, pain severity and incidence of neurotoxicity, visual impairment, capillary leak syndrome, cardiovascular events, hypersensitivity reactions and longterm safety.
• Outcomes:
  - Pain severity and use of analgesics during treatment - Incidence of neurotoxicity, visual impairment, capillary leak syndrome, cardiovascular events and hypersensitivity reactions. - Long term safety, - Progression Free Survival (PFS) in patients treated with dinutuximab beta. - Event Free Survival (EFS) in patients treated with dinutuximab beta - Overall Survival (OS) in patients treated with dinutuximab beta
• Data analysis plan:
  The safety analysis set, containing all patients treated with dinutuximab beta at least once will be considered for safety and efficacy analyses. Data will be presented in individual listings and summarized ? if appropriate over time - according to their data type: - Continuous data by mean, standard deviation, minimum, median, maximum - Qualitative (nominal) data by absolute and/or relative frequencies - Time to event (death or progression of disease) using Kaplan Meier methods - Endpoints addressing primary and secondary analysis will also include 95% CI including the Clopper Pearson method for binomial, log-log transform for survival. Patient listings of efficacy outcome will be provided separately for the different patient subgroups (relapsed, refractory, first line). Efficacy tables will be repeated for the different patient subsets (relapsed, refractory, first line) and overall. Analyses will be performed by visit/ time point, if not stated otherwise.