Treatment of Children With Cerebral Palsy With Autologous Umbilical Cord Blood, a Pilot Study
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 72
- 试验地点
- 1
- 主要终点
- Functional assessment
研究概览
简要总结
This study is a phase II, prospective, double blind, placebo-controlled study of the efficacy of autologous umbilical cord blood infusion.
The study population will consist of 72 children ages 2 months to 12 years with cerebral palsy. The population will be randomly assigned to 2 groups, 36 children in each group. The study group be treated by cord blood in the beginning of the study and the control group by placebo product. The study population will be stratified to reduce variance 3 groups by age: 2-12 months / 1-6 years / 6-12 years The study will consist of 4 stages Stage 1: initial assessment by physiotherapist and occupational therapist / treatment by cord blood or placebo / blood work before and after treatment Stage 2: at stage 1 + 3 months assessment by physiotherapist and occupational therapist Stage 3: at stage 1 + 6 months assessment by physiotherapist and occupational therapist / cross-over treatment by cord blood or placebo / blood work before and after treatment Stage 4: at stage 1 + 12 months assessment by physiotherapist and occupational therapist The primary outcome is improvement motor skills six months after treatment at stage 3
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)
盲法说明
The randomization will be done by external source and the assignment will disclosed to cord bank bank only. They will produce either a cord blood or placebo unit which will be completely covered. Each unit will have its own index number that will be documented by the research coordinator. Neither the researcher or the family will know the nature of the unit.
入排标准
- 年龄范围
- 2 Months 至 12 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age ≥ 2 month and ≤ 12 years.
- •Performance status for children older than 2 months and younger than 12 months including all the following (Gross Motor Function Classification Score is less indicative before 12 months):
- •Abnormal General Movements (in infants 2-6 months)
- •Abnormal Hammersmith Infant Neurological Examination
- •Performance status for children older than 12 months
- •Bilateral spastic cerebral palsy (diplegia or quadraplegia): Gross Motor Function Classification Score levels I - IV
- •Spastic hemiplegia: Gross Motor Function Classification Score levels I - IV. A subject classified as GMFCS level I with significant upper extremity impairment will be eligible if the affected upper extremity is used as an assist only.
- •Bilateral hypotonic cerebral palsy (diplegia or quadraplegia): Gross Motor Function Classification Score levels I - IV.
- •An abnormal brain MRI suggestive of an acquired etiology (and not genetic etiology or brain malformation).
- •Autologous umbilical cord blood available at a private or public cord blood bank with a minimum total nucleated cell dose of ≥ 2 x 10e7 cells/kilogram.
- •Parental consent.
排除标准
- •Autism and autistic spectrum disorders without motor disability.
- •Hypsarrhythmia.
- •Intractable seizures causing epileptic encephalopathy.
- •Evidence of a progressive neurologic disease.
- •Known HIV or uncontrolled bacterial, fungal, or viral infections.
- •Impaired renal or liver function as determined by serum creatinine >1.5mg/dL and/or total bilirubin >1.3mg/dL.
- •Head circumference >3 standard deviations below the mean for age.
- •Known genetic disease or phenotypic evidence of a genetic disease on physical examination.
- •Requires ventilatory support, including home ventilator
- •Surgical procedure or botulinum toxin injection from 6 months prior to the study and during the time of the study
- •Patient's medical condition does not permit safe travel.
- •Previously received any form of cellular therapy.
- •Autologous umbilical cord blood unit has any of the following:
- •Total nuclear cell dose < 2 x 10e7 cells/kilogram
- •Positive maternal infectious disease markers (except CMV)
- •Evidence of infectious contamination of the cord blood unit
- •Lack of a test sample to confirm identity
- •Evidence of a genetic disease
- •Unable to obtain parental consent.
结局指标
主要结局
Functional assessment
时间窗: 6 months
Pediatric Evaluation of Disability Evaluation . Score 0-100 higher score better functionality Pediatric Evaluation of Disability Inventory-Computer Adaptive Test (PEDI-CAT)
Motor developmental improvement
时间窗: 6 months
Gross Motor Function Measurement - 66 (GMFM-66). All score are normalized with mean 100 and STD of 15. High score is better than low score .
次要结局
- Functional assessment(6 months)
研究者
Dr. Omer Bar-Yosef
M.D.-Ph.D. Peadiatric Neurology and Child Development. Deputy Director The Edmond and Lily Safra Children's Hospital
Sheba Medical Center