Multinational European Trial for Children With the Opsoclonus Myoclonus Syndrome / Dancing Eye Syndrome
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 102
- 试验地点
- 36
- 主要终点
- The response to treatment schedule as defined by the percentage of patients with disappearance of all symptoms.
研究概览
简要总结
The OMS/DES study is a multinational European Trial for Children with the Opsoclonus Myoclonus Syndrome / Dancing Eye Syndrome.
This trial brought on the way by specialists of the EPNS (European Paediatric Neurology Society), the GPOH (Gesellschaft für Pädiatrische Hematologic und Oncologie) and the SIOPEN (SIOP (International Society Oncology Pediatric) Europe Neuroblastoma).
This protocol will investigate an escalating treatment schedule starting with a corticosteroid standard treatment with dexamethasone pulses (first step), which is followed, if response has been inadequate after 3 months of treatment, by the addition of CP (second step) and, if still no sufficient improvement, by the replacement of CP by Rituximab (third step). Treatment intensification is decided on the basis of standardized scoring of OMS/DES severity.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 6 Months 至 8 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Children with newly diagnosed OMS/DES either NB-pos or NB-neg.
- •Three out of the following four components are necessary for the diagnosis of OMS/DES:
- •Opsoclonus or ocular flutter (but not nystagmus)
- •Ataxia and/or myoclonus
- •Behavioural change and/or sleep disturbance
- •Neuroblastoma The diagnosis of OMS/DES may be difficult in some patients. Opsoclonus, in particular, may be intermittent or late in onset. A video example will be available at www.dancingeyes.org.uk. If uncertain, please contact the national coordinator for support in interpreting clinical features.
- •Age 6 months or over up to less than 8 years (< 8th birthday) The date of diagnosis of OMS/DES is the date on which a doctor confirms the condition to be OMS/DES. The date of symptom onset needs also to be documented.
- •Treatment start with the standard corticosteroid treatment with dexamethasone pulses as proposed by the guidelines given in this trial protocol (see 11.10, page 71).
- •In patients with presumed NB-neg OMS/DES, neuroblastoma must be excluded according the guidelines of this trial (see chapter 4.4.1.4, page 30, and appendix 11.9, page 70)
- •Documented informed consent for treatment and enrolment in the trial by parents / legal representatives.
排除标准
- •Patients with opsoclonus, myoclonus or ataxia caused by other identified disease (e.g. current active CNS infection, neurometabolic disorder or demyelination).
- •An identified viral precursor is not an exclusion criterion.
- •prior or parallel use of chemotherapy (other than required for treatment of the neuroblastoma)
- •Corticoid steroid for OMS/DES or other reasons lasting 14 days or more immediately before treatment start according the standard treatment proposed (treatment with corticosteroids for less than 14 days will be allowed)
- •contre-indication of use of one of the experimental study drug (cf Summary of Product Characteristics used in this study)
研究组 & 干预措施
Dexamethasone Cyclophosphamide Rituximab
干预措施: Dexamethasone acetate (Drug)
Dexamethasone Cyclophosphamide Rituximab
干预措施: dexamethasone and cyclophosphamide (Drug)
Dexamethasone Cyclophosphamide Rituximab
干预措施: dexamethasone and rituximab (Drug)
结局指标
主要结局
The response to treatment schedule as defined by the percentage of patients with disappearance of all symptoms.
时间窗: at 48 weeks after treatment start
次要结局
未报告次要终点