A Patient Registry of patients with high-risk neuroblastoma being treated with the monoclonal antibody dinutuximab beta

Study type

• Study topic:
  Disease /health condition

  Human medicinal product
• Study type:
  Non-interventional study
• Scope of the study:
  Drug utilisation

  Effectiveness study (incl. comparative)

  Safety study (incl. comparative)
• Data collection methods:
  Primary data collection

Non-interventional study

• Non-interventional study design:
  Other
• Non-interventional study design, other:
  Patient Registry

Study drug and medical condition

• Medicinal product name:
  QARZIBA
• Medicinal product name, other:
  Qarziba 4.5 mg/mL concentrate for solution for infusion
• Study drug International non-proprietary name (INN) or common name:
  DINUTUXIMAB BETA
• Medical condition to be studied:
  Neuroblastoma

Population studied

• Short description of the study population:
  Patients with high-risk neuroblastoma being treated with the monoclonal antibody dinutuximab beta
• 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

• Study design:
  Long-term non-interventional, multi-national, observational and prospective registry
• Main study objective:
  The efficacy and safety of dinutuximab beta will further be evaluated in this registry that will provide information on pain severity and use of analgesics during treatment, incidence of neurotoxicity, visual impairment, capillary leak syndrome, cardiovascular events, hypersensitivity reactions and long-term safety and survival.
• 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. ; - Confidence intervals (CIs) for continuous outcome variables were calculated based on normal distribution assumption.; 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.; For time to event analyses censoring information will be used for statistical analyses. ; Prior treatments for neuroblastoma (induction chemotherapy and high dose chemotherapy) and used analgesics during treatment were coded using the most recent version of WHO-DRUG.; AEs, prior surgeries, and relevant medical history were coded using MedDRA v23.1.;