Evaluation of the Safety and Pharmacokinetics of Dinutuximab Beta as Maintenance Therapy in Chinese Patients With High-risk Neuroblastoma

Study Overview
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Brief Summary
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This is an open-label, multi-center, single-arm, Phase 1 study. The purpose of this study is for evaluating the safety and pharmacokinetics of dinutuximab beta as maintenance therapy in Chinese patients with high-risk neuroblastoma

Official Title
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An Open-Label, Multi-Center, Single-Arm, Phase 1 Study Evaluating the Safety and Pharmacokinetics of Dinutuximab Beta as Maintenance Therapy in Chinese Patients With High-Risk Neuroblastoma

Conditions
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High-risk Neuroblastoma

Intervention / Treatment
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* Drug: Dinutuximab Beta
* Drug: 13 cis retinoic acid


Participation Criteria
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Eligibility Criteria
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Signed informed consent form (ICF) and ability to comply with study requirements Age ≥ 12 months at consent Diagnosis of high-risk neuroblastoma according to the International Neuroblastoma Staging System (INSS) criteria. Patients who have previously received induction chemotherapy and achieved a partial or complete response followed by myeloablative therapy and stem cell transplantation. Stem cell transplantation should be completed within 120 days of dinutuximab beta first administration Exclusion Criteria: Hypersensitivity to ≥ 1 component of dinutuximab beta antibody or against mouse proteins Actively progressive disease (not stabilized) or recurrent disease at the time of inclusion into the study Previous treatment with anti-GD2 antibody before enrolling in this study Note: Other protocol defined Inclusion/Exclusion criteria may apply

Ages Eligible for Study
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Minimum Age: 12 Months

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Dinutuximab beta cohort<br>Patients who received dinutuximab beta as maintenance therapy | Drug: Dinutuximab Beta<br>* Administered via intravenous infusion<br>Drug: 13 cis retinoic acid<br>* Administered orally<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of participants with adverse events (AEs) | Number of participants with treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs) | Up to 2 years | 
| Area under the serum-concentration-time curve (AUC) of dinutuximab beta |  | approximately 20 weeks | 
| Maximum observed serum concentration (Cmax) of dinutuximab beta |  | approximately 20 weeks | 
| Minimum observed serum concentration (Cmin) of dinutuximab beta |  | approximately 20 weeks | 
| Time to reach maximum observed serum concentration (Tmax) of dinutuximab beta |  | approximately20 weeks | 
| Apparent terminal elimination half life (t1/2) of dinutuximab beta |  | approximately 20 weeks |