Umbilical Cord Blood Therapy for Cerebral Palsy

Study Overview
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Brief Summary
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This randomized controlled study aims to evaluate the efficacy of umbilical cord blood therapy for children with cerebral palsy.

Detailed Description
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Cerebral palsy is a disorder of movement and posture resulted from a non-progressive lesion or injury of the immature brain. It is a leading cause of childhood onset disability. Many experimental animal studies have revealed that umbilical cord blood is useful to repair neurological injury in brain. On the basis of many experimental studies, umbilical cord blood is suggested as a potential therapy for cerebral palsy.

Official Title
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Umbilical Cord Blood Therapy for Cerebral Palsy: a Randomized,Double-blind, Placebo-controlled Trial

Conditions
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Cerebral Palsy

Intervention / Treatment
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* Biological: Umbilical Cord Blood Infusion
* Other: Placebo Umbilical Cord Blood
* Other: Active Rehabilitation


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Cerebral Palsy with abnormal muscle tone Gross Motor Function Classification System (GMFCS): I, II, III, IV, V Willing to comply with all study procedure Exclusion Criteria: Medical instability including pneumonia or renal function at enrollment Presence of known genetic disease Presence of drug hypersensitivity which is related to this study remedy Poor cooperation of guardian,including inactive attitude for rehabilitation and visits for follow-up Decision by the principal investigator when there are unexpected events including brain surgery, that may affect the outcome

Ages Eligible for Study
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Minimum Age: 6 Months
Maximum Age: 20 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Umbilical Cord Blood and Rehabilitation<br>Allogeneic Umbilical Cord Blood infusion and Active Rehabilitation | Biological: Umbilical Cord Blood Infusion<br>* The subjects will be undertaken allogeneic umbilical cord blood infusion intravenously or intraarterially under non-myeloablative immunosuppression.<br>* Other names: Donated Umbilical Cord Blood Units from Affiliated Cord Blood Bank;Other: Active Rehabilitation<br>* All subjects should participate in active rehabilitation. They will receive two physical and occupational therapy sessions per day. Post discharge, each participant should continue to receive rehabilitation therapy at least 3 days per week until the study completion.<br>|
| Active Comparator: Placebo Umbilical Cord Blood and Rehabilitation<br>Placebo Umbilical Cord Blood infusion and Active Rehabilitation | Other: Placebo Umbilical Cord Blood<br>* Placebo Umbilical Cord Blood that resembles cord blood in appearance was designed.<br>Other: Active Rehabilitation<br>* All subjects should participate in active rehabilitation. They will receive two physical and occupational therapy sessions per day. Post discharge, each participant should continue to receive rehabilitation therapy at least 3 days per week until the study completion.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Changes in Motor Performance | GMPM (Gross Motor Performance Measure) as 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). We will report GMPM scores at each assessment time points. | Baseline - 1 month - 3 months | 
| Changes in Standardized Gross Motor Function | GMFM (Gross Motor Function Measure) as 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). We will report GMFM scores at each assessment time points. | Baseline - 1 month - 3 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Changes in Cognitive Neurodevelopmental Outcome | Korean version of Bayley Scale of Infant Development-II (K-BSID-II) Mental Scales (higher value means better mental function: 0 - worst, 178 - best). We will report K-BSID-II Mental Scale raw scores at each assessment time points. | Baseline - 1 month - 3 months | 
| Changes in Motor Neurodevelopmental Outcome | Korean version of Bayley Scale of Infant Development-II (K-BSID-II) Motor Scales (higher value means better motor function: 0 - worst, 112 - best). We will report K-BSID-II Motor Scale raw scores at each assessment time points. | Baseline - 1 month - 3 months | 
| Changes in Functional Independence in Daily Activities | WeeFIM (Functional Independence Measure for Children) measures functional independence in daily activities. WeeFIM contains 18 items and each item is ranked from complete dependence (scored as 1) to complete independence (scored as 7). The range is from 18 to 126 and higher scores mean more independent performance in daily activities. We will report total WeeFIM scores measured at each assessment time points. | Baseline - 1 month - 3 months | 
| Changes in Visual Perception Test | We will evaluate visual perception function with one of three measures: DTVP (Developmental Test of Visual Perception), MVPT (Motor-free Visual Perception Test), and VMI (Visual-Motor Integration, Visual Perception and Motor Coordination). All can be scored as percentile rank from 0 to 100. Higher values mean better visual perception ability. | Baseline - 1 month - 3 months | 
| Changes in Muscle Strength | Summation of MMT (manual muscle strength test score): summated scores of the manual muscle strength test (zero=0, trace=1, poor=2, fair=3, good=4, normal=5) for flexors, extensors, abductors, and adductors of bilateral shoulder and hip joints; flexors and extensors of bilateral elbow, wrist, and knee; dorsiflexors and plantar flexors of the ankles (range: 0   160) Higher scores mean better muscle strength. Categories of outcome table will be summation of MMT scores measured at each assessment time point. | Baseline - 1 month - 3 months | 
| Changes in Functional Performance in Daily Activities | Pediatric Evaluation of Disability Inventory (PEDI) for assessing functional performance in daily activities in children (All values are adjusted and higher value means better functional performance, 0 - worst, 100 - best). We will report 2 scales and 3 domains of each scale: a Functional Skill Scale (FSS) and a Caregiver Assistance Scale (CAS) which are divided respectively into 3 domains: self care, mobility, and social function. Categories of outcome table will be each domain scores measured at each assessment time point. | Baseline - 1 month - 3 months | 
| Changes in Brain Glucose Metabolism Using by Brain 18F-FDG PET | 18F-FDG PET imaging will be underwent twice prior to and then 2 weeks post-treatment. All scans will be reviewed by a nuclear physician. Spatial pre-processing and statistical analyses will be done using SPM8 implanted in Matlab to compare differences in regional brain glucose metabolism between groups and differences between pre- and post-therapy imaging data. We will reported increased areas and decreased areas of glucose metabolism in two groups. | Baseline - 2 weeks | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Cerebral Palsy, Umbilical Cord Blood, Rehabilitation