Post-Marketing Assessment of Immunogenicity and Safety of Unituxin® in High-Risk Neuroblastoma Patients

Study Overview
=================
Brief Summary
-----------------
The purpose of this study was to assess the incidence of human anti-chimeric antibody (HACA) in high-risk neuroblastoma patients treated with Unituxin combination therapy.

Detailed Description
-----------------
This was a multi-center, non-randomized, open-label study in patients with high-risk neuroblastoma to assess the immunogenicity, safety and tolerability of Unituxin combination therapy. Patients enrolled in the study were prescribed Unituxin for the treatment of high-risk neuroblastoma.

Official Title
-----------------
A Post-Marketing Study to Further Assess the Immunogenicity and Safety of Unituxin® in High-Risk Neuroblastoma Patients

Conditions
-----------------
Neuroblastoma

Intervention / Treatment
-----------------
* Drug: Dinutuximab


Participation Criteria
=================
Eligibility Criteria
-----------------
Inclusion Criteria: Patient had been diagnosed with high-risk neuroblastoma. Patient had been prescribed Unituxin and plans to start Unituxin therapy within 30 days of study entry. Patient started Unituxin therapy no later than 200 days after Autologous Stem Cell Transplantation (ASCT). Written informed consent / assent was obtained in accordance with institutional and International Conference on Harmonisation (ICH) guidelines. Exclusion Criteria: Patient had received prior anti-disialoganglioside (anti-GD2) antibody therapy. Patient had participated in an investigational clinical trial with tumor therapeutic intent within 30 days of informed consent. Patient underwent Autologous Stem Cell Transplantation (ASCT) more than 200 days prior to receiving Unituxin therapy.

Ages Eligible for Study
-----------------
Minimum Age: 1 Year
Maximum Age: 18 Years

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Dinutuximab administered for 5 cycles<br>High-risk neuroblastoma patient treated with Unituxin as standard of care | Drug: Dinutuximab<br>* Unituxin was administered along with cytokines according to the prescribing information<br>* Other names: Unituxin®;|

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| To Determine the Incidence of Human Anti-chimeric Antibody (HACA) in High-risk Neuroblastoma Patients Treated With Unituxin Combination Therapy. | Seven blood samples were collected at the following time points for the evaluation of HACA levels: Course 1- Prior to the first Unituxin infusion Course 2- Prior to the first Unituxin infusion Course 3- Prior to the first Unituxin infusion Course 4- Prior to the first Unituxin infusion Course 5- Prior to the first Unituxin infusion Course 6- Prior to the first dose of 13-cis-retinoic acid (RA) Study termination (approximately 2 weeks following the final 13-cis-retinoic acid dose) | Approximately 6 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| To Determine the Incidence of Targeted Immune-related Adverse Events (AEs) During Treatment With Dinutuximab Combination Therapy in High-risk Neuroblastoma Subjects. | The incidence of targeted immune-related adverse events (AEs) during treatment with dinutuximab combination therapy in high-risk neuroblastoma subjects were summarized and listed. | Approximately 6 months | 
| To Determine the Incidence of Neutralizing Antibody (NAb) in Patients With Human Anti-chimeric Antibody (HACA) Positive Samples. | Incidence of neutralizing antibody (NAb) in patients with human anti-chimeric antibody (HACA) positive samples was summarized and listed. | Approximately 6 months | 
| To Determine the Effect of HACA on Dinutuximab Plasma Concentrations. | Ten blood samples were collected at the following time points for the evaluation of dinutuximab plasma concentrations: Course 1- Prior to the first Unituxin infusion Course 2- Prior to the first Unituxin infusion Course 3- Prior to the first Unituxin infusion Course 4- Prior to the first Unituxin infusion Course 5- Prior to the first Unituxin infusion Course 6- Prior to the first dose of 13-cis-retinoic acid (RA) Study termination (approximately 2 weeks following the final 13-cis-retinoic acid dose). An additional 3 blood samples were obtained for the evaluation of dinutuximab plasma concentrations. Each of these blood samples was obtained immediately following the fourth dinutuximab infusion in Courses 1, 3, and 5. | Approximately 6 months |