Umbilical Cord Blood Therapy for Cerebral Palsy

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

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

Study Overview

• Status: Completed
• Conditions: Cerebral Palsy
• Intervention / Treatment: Other: Active Rehabilitation; Biological: Umbilical Cord Blood Infusion; Other: Placebo Umbilical Cord Blood

Detailed Description

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.

• Study Type: Interventional
• Enrollment (Actual): 37
• Phase: Phase 2

Contacts and Locations

Study Locations

• Korea, Republic of
  • Gyeonggi-do
    • Seongnam-si, Gyeonggi-do, Korea, Republic of, 463-712
      • CHA Bundang Medical Center, CHA University

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 6 months to 20 years (Child, Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Umbilical Cord Blood and Rehabilitation; Allogeneic Umbilical Cord Blood infusion and Active Rehabilitation	Other: Active Rehabilitation; 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.; Biological: Umbilical Cord Blood Infusion; The subjects will be undertaken allogeneic umbilical cord blood infusion intravenously or intraarterially under non-myeloablative immunosuppression.; Other Names: Donated Umbilical Cord Blood Units from Affiliated Cord Blood Bank
Active Comparator: Placebo Umbilical Cord Blood and Rehabilitation; Placebo Umbilical Cord Blood infusion and Active Rehabilitation	Other: Active Rehabilitation; 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.; Other: Placebo Umbilical Cord Blood; Placebo Umbilical Cord Blood that resembles cord blood in appearance was designed.

What is the study measuring?

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 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 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 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

Collaborators and Investigators

• Sponsor: MinYoung Kim, M.D.

Study record dates

Study Major Dates

• Study Start: February 1, 2012
• Primary Completion (Actual): July 1, 2012
• Study Completion (Actual): July 1, 2012

Study Registration Dates

• First Submitted: February 3, 2012
• First Submitted That Met QC Criteria: February 3, 2012
• First Posted (Estimate): February 8, 2012

Study Record Updates

• Last Update Posted (Estimate): July 18, 2012
• Last Update Submitted That Met QC Criteria: July 17, 2012
• Last Verified: July 1, 2012

More Information

Terms related to this study

• Keywords: Rehabilitation; Cerebral Palsy; Umbilical Cord Blood
• Additional Relevant MeSH Terms: Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Neurologic Manifestations; Brain Damage, Chronic; Cerebral Palsy; Paralysis
• Other Study ID Numbers: CPUCBRCT