Study type

Study type

Non-interventional study

Scope of the study

Effectiveness study (incl. comparative)
Safety study (incl. comparative)
Non-interventional study

Non-interventional study design

Cohort
Study drug and medical condition

Name of medicine

QARZIBA

Study drug International non-proprietary name (INN) or common name

DINUTUXIMAB BETA

Medical condition to be studied

Neuroblastoma

Additional medical condition(s)

High-Risk 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

150
Study design details

Main study objective

This is a multi-center, observational, ambispective cohort study with the aim of evaluating the long-term effectiveness and safety of dinutuximab beta or conventional treatment as first-line maintenance therapy in patients with HR NB. The primary endpoint of this study is measured by the 3-year rwEFS rate of patients received dinutuximab beta or conventional treatment.

Outcomes

Title: 3-year real-world event free survival (rwEFS) rate. The survival curves for rwEFS will be plotted using the Kaplan-Meier method, and the 3-year rwEFS rate will be estimated. The two-sided 95% CIs will be calculated using the Greenwood formula. Real world event free survival (rwEFS) is defined as the time from first dose of maintenance therapy to the date of earliest occurrence, 2-year Real-World Event-Free Survival (rwEFS) rate 1-year Real-World Event-Free Survival (rwEFS) rate 3-year overall survival（OS）rate Percentage of Participants With an Adverse Event (AE), Serious AE, or Death Due to an AE

Data analysis plan

There are two stages for this study: 1. Case Collection Stage: Collecting eligible patients in 10 study centers and using the electronic data collection system to record demographic, clinical characteristics, treatment, concomitant medication and safety information. This will be from when they received dinutuximab beta until two years after. The case information is reported retrospectively and after recruitment, the data is collected at the site prospectively. 2. Follow Up Stage: all patients will be followed up with for at least two years to collection endpoint data.