Back to resultsUmbilical Cord Blood Therapy for Cerebral Palsy
Primary Purpose
Cerebral Palsy
Status
Completed
Phase
Phase 2
Locations
Korea, Republic of
Study Type
Interventional
Intervention
Umbilical Cord Blood Infusion
Placebo Umbilical Cord Blood
Active Rehabilitation
Sponsored by
About this trial
This is an interventional treatment trial for Cerebral Palsy focused on measuring Cerebral Palsy, Umbilical Cord Blood, Rehabilitation
Eligibility Criteria
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
Sites / Locations
- CHA Bundang Medical Center, CHA University
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Active Comparator
Arm Label
Umbilical Cord Blood and Rehabilitation
Placebo Umbilical Cord Blood and Rehabilitation
Arm Description
Allogeneic Umbilical Cord Blood infusion and Active Rehabilitation
Placebo Umbilical Cord Blood infusion and Active Rehabilitation
Outcomes
Primary Outcome Measures
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.
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.
Secondary Outcome Measures
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.
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.
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.
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.
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.
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.
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.
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT01528436
Brief Title
Umbilical Cord Blood Therapy for Cerebral Palsy
Official Title
Umbilical Cord Blood Therapy for Cerebral Palsy: a Randomized,Double-blind, Placebo-controlled Trial
Study Type
Interventional
2. Study Status
Record Verification Date
July 2012
Overall Recruitment Status
Completed
Study Start Date
February 2012 (undefined)
Primary Completion Date
July 2012 (Actual)
Study Completion Date
July 2012 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor-Investigator
Name of the Sponsor
MinYoung Kim, M.D.
4. Oversight
Data Monitoring Committee
No
5. Study Description
Brief Summary
This randomized controlled study aims to evaluate the efficacy of umbilical cord blood therapy for children with cerebral palsy.
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Cerebral Palsy
Keywords
Cerebral Palsy, Umbilical Cord Blood, Rehabilitation
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
37 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Umbilical Cord Blood and Rehabilitation
Arm Type
Experimental
Arm Description
Allogeneic Umbilical Cord Blood infusion and Active Rehabilitation
Arm Title
Placebo Umbilical Cord Blood and Rehabilitation
Arm Type
Active Comparator
Arm Description
Placebo Umbilical Cord Blood infusion and Active Rehabilitation
Intervention Type
Biological
Intervention Name(s)
Umbilical Cord Blood Infusion
Other Intervention Name(s)
Donated Umbilical Cord Blood Units from Affiliated Cord Blood Bank
Intervention Description
The subjects will be undertaken allogeneic umbilical cord blood infusion intravenously or intraarterially under non-myeloablative immunosuppression.
Intervention Type
Other
Intervention Name(s)
Placebo Umbilical Cord Blood
Intervention Description
Placebo Umbilical Cord Blood that resembles cord blood in appearance was designed.
Intervention Type
Other
Intervention Name(s)
Active Rehabilitation
Intervention Description
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.
Primary Outcome Measure Information:
Title
Changes in Motor Performance
Description
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.
Time Frame
Baseline - 1 month - 3 months
Title
Changes in Standardized Gross Motor Function
Description
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.
Time Frame
Baseline - 1 month - 3 months
Secondary Outcome Measure Information:
Title
Changes in Cognitive Neurodevelopmental Outcome
Description
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.
Time Frame
Baseline - 1 month - 3 months
Title
Changes in Motor Neurodevelopmental Outcome
Description
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.
Time Frame
Baseline - 1 month - 3 months
Title
Changes in Functional Independence in Daily Activities
Description
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.
Time Frame
Baseline - 1 month - 3 months
Title
Changes in Visual Perception Test
Description
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.
Time Frame
Baseline - 1 month - 3 months
Title
Changes in Muscle Strength
Description
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.
Time Frame
Baseline - 1 month - 3 months
Title
Changes in Functional Performance in Daily Activities
Description
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.
Time Frame
Baseline - 1 month - 3 months
Title
Changes in Brain Glucose Metabolism Using by Brain 18F-FDG PET
Description
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.
Time Frame
Baseline - 2 weeks
10. Eligibility
Sex
All
Minimum Age & Unit of Time
6 Months
Maximum Age & Unit of Time
20 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Minyoung Kim, M.D., Ph.D.
Organizational Affiliation
CHA University
Official's Role
Principal Investigator
Facility Information:
Facility Name
CHA Bundang Medical Center, CHA University
City
Seongnam-si
State/Province
Gyeonggi-do
ZIP/Postal Code
463-712
Country
Korea, Republic of
12. IPD Sharing Statement
Learn more about this trial
Umbilical Cord Blood Therapy for Cerebral Palsy
We'll reach out to this number within 24 hrs