Safety and Efficacy of Allogeneic Umbilical Cord Blood Therapy Combined With Erythropoietin in Children With Cerebral Palsy: a Double-blind, Randomized, Placebo-controlled Clinical Trial
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 92
- 试验地点
- 2
- 主要终点
- Changes in Standardized Gross Motor Function
研究概览
简要总结
This randomized controlled study aims to evaluate the efficacy and safety of allogeneic umbilical cord blood therapy combined with erythropoietin for children with cerebral palsy.
详细描述
Cerebral palsy (CP) is a group of neurodevelopmental conditions with abnormal movement and posture resulted from a non-progressive cerebral disturbance. It is the most common cause of motor disability in childhood. Most therapies are palliative rather than restorative. Umbilical cord blood (UCB) and erythropoetin (EPO) may be used as restorative approach for children with CP.
Many experimental animal studies have revealed that UCB is beneficial to improve and repair neurological injuries. EPO is also known to have neuroprotective effects.
Based on animal studies and some clinical trials, UCB is suggested as a potential therapy for children with CP. EPO is combined to add synergistic effects to UCB therapy.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 10 Months 至 6 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosed with cerebral palsy
- •Age of ≥10 months and ≤6 years
- •Mismatch in HLA-A, B, and DR ≤2, and total nucleated cell count ≥3x107/kg. If the cell count is less than given values, more than 1 unit could be used.
- •Hemoglobin ≤13.6 g/dL
- •Decision of participation in the study by and acquisition of informed consent from the subject's representative
- •Willingness and ability to be hospitalized according to the schedule specified in the protocol and continue the study for 12 months after study entry
排除标准
- •Current aspiration pneumonia
- •Known genetic disease
- •History of hypersensitivity reaction to any study drugs pertinent to the study
- •History of participation in any other study with stem cell
- •Prior treatment with EPO within 3 months prior to study entry
- •Known coagulopathy with family history of thrombosis or medical history of recurrent thrombosis
- •Patient with severe seizure disease who has clinical convulsion despite combination therapy with 3 or more agents
- •Uncontrolled hypertension defined as systolic blood pressure >115 mmHg and/or diastolic blood pressure >70 mmHg
- •Hepatic impairment defined as asparate aminotransferase (AST) >55 IU/L and/or alanine aminotransferase (ALT) >45 IU/L
- •Renal impairment defined as creatinine (Cr) ≥1.2 mg/dL
- •Absolute neutrophil count ≤500/dL
- •Presence of diagnosed or suspected malignant tumor and/or hematologic malignancy
- •Non-compliance with study visits specified in the protocol or unwillingness of care-giver due to lack of understanding of the patient
结局指标
主要结局
Changes in Standardized Gross Motor Function
时间窗: Baseline - 1 month - 3 months - 6 months - 12months
GMFM (Gross Motor Function Measure) is a standardized measurement tool for assessing gross motor function consisting of sub-scales; lying \& rolling, sitting, crawling \& kneeling, standing, walking, running \& jumping (range: 0\~100, higher value means better gross motor function).
Changes in Motor Performance
时间窗: Baseline - 1 month - 3 months - 6 months - 12 months
GMPM (Gross Motor Performance Measure) is a standardized measurement tool for assessing quality of movement regarding 3 properties of 5 ones; alignment, coordination, dissociated movement, stability, and weight shift (range: 0\~100, higher value means better motor quality).
Changes in Motor Neurodevelopmental Outcome
时间窗: Baseline - 1 month - 3 months - 6 months - 12 months
Korean version of Bayley Scale of Infant Development-II (K-BSID-II) Motor Scale (range: 0\~112; worst: 0, best: 112)
Changes in Cognitive Neurodevelopmental Outcome
时间窗: Baseline - 1 month - 3 months - 6 months - 12 months
Korean version of Bayley Scale of Infant Development-II (K-BSID-II) Mental Scale (range: 0\~178; worst: 0, best: 178)
次要结局
- Changes in Visual Perception Test(Baseline - 1 month - 3 months - 6 months - 12 months)
- Changes in Spasticity(Baseline - 1 month - 3 months - 6 months - 12 months)
- Changes in Muscle Strength(Baseline - 1 month - 3 months - 6 months - 12 months)
- Changes in Brain MRI(Baseline - 12 months)
- Number of adverse events and participants with those adverse events(12 months)
- Changes in Gross Motor Function Classification System(Baseline - 1 month - 3 months - 6 months - 12 months)
- Changes in Functional Independence in Daily Activities(Baseline - 1 month - 3 months - 6 months - 12 months)
- Changes in Dynamic Component of Spasticity(Baseline - 1 month - 3 months - 6 months - 12 months)
- Changes in Functional Performance in Daily Activities(Baseline - 1 month - 3 months - 6 months - 12 months)
- Changes in Upper Extremity Function(Baseline - 1 month - 3 months - 6 months - 12 months)
- Changes in Selective Movement of Lower Extremity(Baseline - 1 month - 3 months - 6 months - 12 months)
- Changes in Brain 18F-FDG PET(Baseline - 12 months)
- Changes in EEG(Baseline - 12 months)
- Changes in EP(Baseline - 12 months)
研究者
MinYoung Kim, MD, PhD
Professor of CHA University, M.D., Ph.D.
Bundang CHA Hospital