A Post-Authorisation Safety Study Patient Registry of Patients With High-risk Neuroblastoma Being Treated With the Monoclonal Antibody Dinutuximab Beta
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- 入组人数
- 125
- 试验地点
- 13
- 主要终点
- Number of participants using analgesics during treatment with dinutuximab beta
研究概览
简要总结
This is a non-interventional, multi-national, observational, prospective patient registry to further evaluate the effectiveness and safety of dinutuximab beta - a monoclonal immunoglobulin G 1 (IgG1) antibody, to obtain information on survival, pain severity and incidence of neuro-toxicity, visual impairment, capillary leak syndrome, cardiovascular events, hypersensitivity reactions and long-term safety.
详细描述
Rationale and Background:
Neuroblastoma, is the most common extra-cranial solid tumour in children. Most patients with neuroblastoma are diagnosed under the age of 5 years and most present with metastatic disease and/or high-risk features. Despite the introduction of novel treatment strategies, including high-dose chemotherapy followed by autologous stem cell transplantation (ASCT), the outcome of these patients remains poor.
Dinutuximab beta is a chimeric monoclonal immunoglobulin G 1 (IgG1) antibody that is specifically directed against the carbohydrate moiety of disialoganglioside antigen (GD2), which is overexpressed on neuroblastoma cells. By binding to neuroblastoma cells, dinutuximab beta can induce both complement dependent cytotoxicity (CDC) and antibody dependent cell-mediated cytotoxicity (ADCC).
The efficacy of dinutuximab beta has been evaluated in a randomised controlled trial comparing the administration of dinutuximab beta with or without interleukin 2 (IL-2) in the first-line treatment of patients with high-risk neuroblastoma and in two singlearm studies in the relapsed/refractory setting. The efficacy and safety of dinutuximab beta will further be evaluated in this registry that will provide information on survival, pain severity and incidence of neurotoxicity, visual impairment, capillary leak syndrome, cardiovascular events, hypersensitivity reactions and long-term safety.
Study Design:
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 1 Year 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients meeting the following criteria will be considered for inclusion into the registry:
- •Patients diagnosed with high-risk neuroblastoma and starting treatment with commercially available dinutuximab beta OR
- •Patients diagnosed with high-risk neuroblastoma and starting treatment with dinutuximab beta in a clinical trial where dinutuximab beta is provided according to the country/regional marketing authorisation AND
- •Appropriate consent/assent has been obtained for participation in the registry with a willingness to be followed up for up to 10 years.
排除标准
- •Patient will not be eligible for inclusion if the following criterion applies:
- •Patients commencing dinutuximab beta within a clinical trial where the product is being provided outside of the country/regional marketing authorisation OR
- •Appropriate consent/assent has not been obtained for participation in the registry or patient/legal representative is not willing for the patient be followed up for up to 10 years.
结局指标
主要结局
Number of participants using analgesics during treatment with dinutuximab beta
时间窗: First dose of dinutuximab beta to the end of the last 35 day course of the 5th cycle of treatment (each cycle is 35 days)
Use of analgesics during the period of first dose of dinutuximab beta to end of last 35 day course of 5th cycle of treatment
Incidence of neurotoxicity, visual impairment, capillary leak syndrome, cardiovascular events and hypersensitivity reactions
时间窗: First dose of dinutuximab beta to the end of the last 35 day course of the 5th cycle of treatment (each cycle is 35 days)
Incidence of neurotoxicity, visual impairment, capillary leak syndrome, cardiovascular events and hypersensitivity reactions up to the end of the last 35 day course of 5th cycle of treatment
Assessment of the severity of pain experienced by participants during treatment with dinutuximab beta
时间窗: First dose of dinutuximab beta to the end of the last 35 day course of the 5th cycle of treatment (each cycle is 35 days)
Assessment of pain severity experienced by participants during the period of first dose of dinutuximab beta to the end of last 35 day course of 5th cycle of treatment
Number of participants experiencing serious adverse events (SAEs) and adverse drug reactions (ADRs) during treatment with dinutuximab beta
时间窗: First dose of dinutuximab beta to the end of the last 35 day course of the 5th cycle of treatment (each cycle is 35 days)
Number of participants experiencing serious adverse events (SAEs) and adverse drug reactions (ADRs) following the end of the last 35 day course of 5th cycle of treatment
次要结局
- Overall Survival (OS)(First dose of dinutuximab beta to the end of the last 35 day course of the 5th cycle of treatment (each cycle is 35 days))
- Event Free Survival (EFS)(First dose of dinutuximab beta to the end of the last 35 day course of the 5th cycle of treatment (each cycle is 35 days))
- Progression free survival (PFS)(First dose of dinutuximab beta to the end of the last 35 day course of the 5th cycle of treatment (each cycle is 35 days))