Phase II Study on Mannan Binding Lectin (MBL) Substitution in MBL-Deficient Children With Chemotherapy-Induced Neutropenia
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 12
- 试验地点
- 2
- 主要终点
- pharmacokinetics of MBL
研究概览
简要总结
The pharmacokinetics, and clinical and biological effects of MBL replacement therapy in MBL-deficient children during chemotherapy-induced neutropenia were studied.
详细描述
Mannan Binding Lectin (MBL) is a member of the lectin pathway of the complement system and plays an important role in the innate immune system. MBL replacement in MBL-deficient children with chemotherapy-induced neutropenia represents a new approach to lower the risk of febrile episodes, of hospital admission, of prolonged use of intravenous antibiotics and of severe infections.
The aim of the Phase II study is to find evidence for the correct prediction of plasma levels of MBL necessary for clinical effects and biological efficacy, to confirm the dosage regimen needed to reach the required MBL plasma levels, and reconfirm the safety and lack of side-effects.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Prevention
- 盲法
- None
入排标准
- 年龄范围
- — 至 12 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Children ages 0 - 12 years, during chemotherapy, and expected to become neutropenic
- •MBL deficiency by genotype or phenotype (< 100 ng/ml)
- •Informed consent and assent of patient and/or legal representative
排除标准
- •Inability or unwillingness to comply with the protocol or likely inability to complete the study period
- •Known allergic reactions to MBL and other human plasma products
- •Participation in other investigational drug studies within the last month
- •Clinically relevant abnormalities in: serum immunoglobulins IgG, IgA, IgM; blood counts; complement factors measured by AP50, CH50; urine protein and cell counts; serum creatinine and liver enzymes, as routinely determined for regular patient care.
研究组 & 干预措施
A
MBL until the patient's absolute neutrophil count (ANC) is above 500/microL blood.
干预措施: Mannan Binding Lectin (MBL) (Drug)
A
MBL until the patient's absolute neutrophil count (ANC) is above 500/microL blood.
干预措施: Mannan Binding Lectin (Drug)
结局指标
主要结局
pharmacokinetics of MBL
时间窗: until the patient's absolute neutrophil count (ANC) is above 500/uL blood.
次要结局
- days of fever(until the patient's absolute neutrophil count (ANC) is above 500/uL blood.)
- days of hospital admission(until the patient's absolute neutrophil count (ANC) is above 500/uL blood.)
- use of antibiotics or antifungal medication(until the patient's absolute neutrophil count (ANC) is above 500/uL blood.)
- number and type of infections(until the patient's absolute neutrophil count (ANC) is above 500/uL blood.)
- MBL-dependent opsonizing capacity in vitro(until the patient's absolute neutrophil count (ANC) is above 500/uL blood.)
- safety and incidence of side effects(until the patient's absolute neutrophil count (ANC) is above 500/uL blood.)