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Allogenic Umbilical Cord Blood and Erythropoietin Combination Therapy for Cerebral Palsy

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT01193660
Recruitment Status : Completed
First Posted : September 2, 2010
Results First Posted : March 1, 2012
Last Update Posted : November 24, 2020
Sponsor:
Information provided by (Responsible Party):
Sung Kwang Medical Foundation

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition Cerebral Palsy
Interventions Biological: Umbilical Cord Blood Infusion
Drug: Erythropoietin Injection
Other: Active Rehabilitation
Other: Placebo Umbilical Cord Blood
Other: Placebo Erythropoietin
Enrollment 105
Recruitment Details Enrollment Period: May 31, 2010 through April 9, 2011 All participants were recruited at CHA Bundang Medical Center.
Pre-assignment Details We tried to recruit 106 subjects. Finally 105 patients were enrolled at the beginning of the overall study and none of the participants were washed out before assignment to groups.
Arm/Group Title Umbilical Cord Blood & Erythropoietin & Rehabilitation Erythropoietin & Rehabilitation Only Rehabilitation
Hide Arm/Group Description Allogeneic umbilical cord blood infusion (total nucleated cells > 3x10^7/kg intravenously), Erythropoietin injection (twice a week for 4 weeks with the dosage of 500 IU/kg for 2 times intravenously and 250 IU/kg subcutaneously for 6 times), and active rehabilitation Erythropoietin injection (twice a week for 4 weeks with the dosage of 500 IU/kg for 2 times intravenously and 250 IU/kg subcutaneously for 6 times),and active rehabilitation Active rehabilitation
Period Title: Overall Study
Started 35 36 34
Completed 31 33 32
Not Completed 4 3 2
Reason Not Completed
Withdrawal by Subject             2             3             1
Death             1             0             0
Physician Decision             1             0             1
Arm/Group Title Umbilical Cord Blood & Erythropoietin & Rehabilitation Erythropoietin & Rehabilitation Only Rehabilitation Total
Hide Arm/Group Description Allogeneic umbilical cord blood infusion (total nucleated cells > 3x10^7/kg intravenously), Erythropoietin injection (twice a week for 4 weeks with the dosage of 500 IU/kg for 2 times intravenously and 250 IU/kg subcutaneously for 6 times), and active rehabilitation Erythropoietin injection (twice a week for 4 weeks with the dosage of 500 IU/kg for 2 times intravenously and 250 IU/kg subcutaneously for 6 times),and active rehabilitation Active rehabilitation Total of all reporting groups
Overall Number of Baseline Participants 35 36 34 105
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 35 participants 36 participants 34 participants 105 participants
<=18 years
35
 100.0%
36
 100.0%
34
 100.0%
105
 100.0%
Between 18 and 65 years
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
>=65 years
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Months
Number Analyzed 35 participants 36 participants 34 participants 105 participants
45.71  (18.91) 50.05  (24.45) 46.0  (19.58) 47.30  (21.07)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 35 participants 36 participants 34 participants 105 participants
Female
13
  37.1%
11
  30.6%
9
  26.5%
33
  31.4%
Male
22
  62.9%
25
  69.4%
25
  73.5%
72
  68.6%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
Korea, Republic of Number Analyzed 35 participants 36 participants 34 participants 105 participants
35 36 34 105
1.Primary Outcome
Title Changes in Motor Performance
Hide 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 reported changes of GMPM score between each assessment time points. Categories of outcome table are baseline and values of just subtracting the latter raw scores from the former ones.
Time Frame Baseline -1 month - 3 months - 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
Intention to treat
Arm/Group Title Umbilical Cord Blood & Erythropoietin & Rehabilitation Erythropoietin & Rehabilitation Only Rehabilitation
Hide Arm/Group Description:
Allogeneic umbilical cord blood infusion (total nucleated cells > 3x10^7/kg intravenously), Erythropoietin injection (twice a week for 4 weeks with the dosage of 500 IU/kg for 2 times intravenously and 250 IU/kg subcutaneously for 6 times), and active rehabilitation
Erythropoietin injection (twice a week for 4 weeks with the dosage of 500 IU/kg for 2 times intravenously and 250 IU/kg subcutaneously for 6 times),and active rehabilitation
Active rehabilitation
Overall Number of Participants Analyzed 31 33 32
Mean (Standard Error)
Unit of Measure: units on a scale
Baseline 34.5  (2.7) 38.2  (2.8) 35.5  (2.9)
1 month - Baseline 7.00  (1.3) 4.5  (0.6) 6.0  (1.0)
3 months - Baseline 11.5  (1.5) 7.5  (0.8) 8.1  (1.2)
6 months - Baseline 14.5  (1.8) 9.2  (0.8) 9.6  (1.2)
3 months - 1 month 4.5  (0.7) 2.9  (0.5) 2.1  (0.6)
6 months - 1 month 7.5  (0.9) 4.7  (0.7) 3.6  (0.7)
6 months - 3 months 3.1  (0.6) 1.7  (0.5) 1.5  (0.4)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Umbilical Cord Blood & Erythropoietin & Rehabilitation, Erythropoietin & Rehabilitation, Only Rehabilitation
Comments In our analysis, the null hypothesis is that the effects of 3 experimental groups are same, and the alternative hypothesis is that the therapeutic effect of combination intervention Group (Umbilical Cord Blood + Erythropoietin + Rehabilitation) is higher than that of either Erythropoietin + Rehabilitation or Rehabilitation Group. This is a pilot study and power calculation was not applicable. The sample size of each group is over 30 and is considered to follow approximately normal distribution.
Type of Statistical Test Superiority or Other (legacy)
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.05
Comments [Not Specified]
Method Repeated Measure ANOVA
Comments [Not Specified]
Method of Estimation Estimation Parameter Wilks' Lambda (group*visit effect)
Estimated Value 2.59
Estimation Comments [Not Specified]
2.Primary Outcome
Title Changes in Standardized Gross Motor Function
Hide 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 reported changes of GMFM between each assessment time points. Categories of outcome table are baseline and values of just subtracting the latter raw scores from the former ones.
Time Frame Baseline - 1 month - 3 months - 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
Intention to treat
Arm/Group Title Umbilical Cord Blood & Erythropoietin & Rehabilitation Erythropoietin & Rehabilitation Only Rehabilitation
Hide Arm/Group Description:
Allogeneic umbilical cord blood infusion (total nucleated cells > 3x10^7/kg intravenously), Erythropoietin injection (twice a week for 4 weeks with the dosage of 500 IU/kg for 2 times intravenously and 250 IU/kg subcutaneously for 6 times), and active rehabilitation
Erythropoietin injection (twice a week for 4 weeks with the dosage of 500 IU/kg for 2 times intravenously and 250 IU/kg subcutaneously for 6 times),and active rehabilitation
Active rehabilitation
Overall Number of Participants Analyzed 31 33 32
Mean (Standard Error)
Unit of Measure: units on a scale
Baseline 36.0  (4.7) 42.6  (4.9) 36.8  (4.5)
1 month - Baseline 3.7  (0.4) 4.3  (0.5) 4.6  (0.6)
3 months - Baseline 6.5  (0.9) 6.8  (0.8) 6.4  (0.7)
6 months - Baseline 9.1  (1.2) 9.0  (1.1) 7.8  (0.9)
3 months - 1 month 2.9  (0.8) 2.5  (0.5) 1.8  (0.4)
6 months - 1 month 5.4  (1.2) 4.7  (0.9) 3.1  (0.6)
6 months - 3 months 2.6  (0.5) 2.2  (0.6) 1.3  (0.4)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Umbilical Cord Blood & Erythropoietin & Rehabilitation, Erythropoietin & Rehabilitation, Only Rehabilitation
Comments In our analysis, the null hypothesis is that the effects of 3 experimental groups are same, and the alternative hypothesis is that the therapeutic effect of combination intervention Group (Umbilical Cord Blood + Erythropoietin + Rehabilitation) is higher than that of either Erythropoietin + Rehabilitation or Rehabilitation Group. This is a pilot study and power calculation was not applicable. The sample size of each group is over 30 and is considered to follow approximately normal distribution.
Type of Statistical Test Superiority or Other (legacy)
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.05
Comments [Not Specified]
Method Repeated Measure ANOVA
Comments [Not Specified]
Method of Estimation Estimation Parameter interaction of group and visit
Estimated Value 0.90
Estimation Comments [Not Specified]
3.Secondary Outcome
Title Changes in Cognitive Neurodevelopmental Outcome
Hide 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 reported changes of BSID-II Mental Scale raw score between each assessment time points. Categories of outcome data are values of subtracting the latter scores from the former ones.
Time Frame Baseline -1 month - 3 months - 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
Intention to treat
Arm/Group Title Umbilical Cord Blood & Erythropoietin & Rehabilitation Erythropoietin & Rehabilitation Only Rehabilitation
Hide Arm/Group Description:
Allogeneic umbilical cord blood infusion (total nucleated cells > 3x10^7/kg intravenously), Erythropoietin injection (twice a week for 4 weeks with the dosage of 500 IU/kg for 2 times intravenously and 250 IU/kg subcutaneously for 6 times), and active rehabilitation
Erythropoietin injection (twice a week for 4 weeks with the dosage of 500 IU/kg for 2 times intravenously and 250 IU/kg subcutaneously for 6 times),and active rehabilitation
Active rehabilitation
Overall Number of Participants Analyzed 31 33 32
Mean (Standard Error)
Unit of Measure: units on a scale
BSID-II Mental Scale: Baseline 94.1  (9.1) 105.9  (8.9) 86.6  (9.0)
BSID-II Mental Scale: 1 month - Baseline 8.2  (1.3) 3.4  (0.5) 3.3  (0.6)
BSID-II Mental Scale: 3 months - Baseline 12.0  (1.4) 7.4  (0.9) 5.8  (0.8)
BSID-II Mental Scale: 6 months - Baseline 17.6  (1.8) 11.5  (1.3) 9.9  (1.6)
BSID-II Mental Scale: 3 months - 1 month 3.8  (0.8) 4.0  (0.7) 2.5  (0.7)
BSID-II Mental Scale: 6 months - 1 month 9.4  (1.4) 8.1  (1.3) 6.6  (1.4)
BSID-II Mental Scale: 6 months - 3 months 5.6  (1.2) 4.1  (0.8) 4.1  (1.0)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Umbilical Cord Blood & Erythropoietin & Rehabilitation, Erythropoietin & Rehabilitation, Only Rehabilitation
Comments The null hypothesis is that in terms of K-BSID-II MENTAL Scale, the effects of 3 groups are same, and the alternative one is that the therapeutic effect of combination intervention Group (Umbilical Cord Blood + Erythropoietin + Rehabilitation) is higher than that of either Erythropoietin + Rehabilitation or Rehabilitation Group. This is a pilot study and power calculation was not applicable. The sample size of each group is over 30 and is considered to follow approximately normal distribution.
Type of Statistical Test Superiority or Other (legacy)
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.05
Comments [Not Specified]
Method Repeated Measure ANOVA
Comments [Not Specified]
Method of Estimation Estimation Parameter Wilks' Lambda (group*visit effect)
Estimated Value 3.94
Estimation Comments [Not Specified]
4.Secondary Outcome
Title Changes in Motor Neurodevelopmental Outcome
Hide Description Korean version of Bayley Scale of Infant Development-II (K-BSID-II) Motor Scales (higher value means better motor function: 0 - worst, 111 - best). We reported changes of BSID-II Motor Scale raw score between each assessment time points. Categories of outcome data are values of subtracting the latter scores from the former ones.
Time Frame Baseline - 1 month - 3 months - 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
Intention to treat
Arm/Group Title Umbilical Cord Blood & Erythropoietin & Rehabilitation Erythropoietin & Rehabilitation Only Rehabilitation
Hide Arm/Group Description:
Allogeneic umbilical cord blood infusion (total nucleated cells > 3x10^7/kg intravenously), Erythropoietin injection (twice a week for 4 weeks with the dosage of 500 IU/kg for 2 times intravenously and 250 IU/kg subcutaneously for 6 times), and active rehabilitation
Erythropoietin injection (twice a week for 4 weeks with the dosage of 500 IU/kg for 2 times intravenously and 250 IU/kg subcutaneously for 6 times),and active rehabilitation
Active rehabilitation
Overall Number of Participants Analyzed 31 33 32
Mean (Standard Error)
Unit of Measure: units on a scale
BSID-II Motor Scale: Baseline 45.2  (4.7) 53.3  (5.0) 45.5  (4.6)
BSID-II Motor Scale: 1 month - Baseline 5.0  (1.5) 5.2  (2.1) 2.7  (0.6)
BSID-II Motor Scale: 3 months - Baseline 9.5  (1.9) 6.8  (2.1) 4.3  (0.8)
BSID-II Motor Scale: 6 months - Baseline 11.7  (2.0) 7.6  (2.2) 5.2  (0.9)
BSID-II Motor Scale: 3 months - 1 month 4.5  (1.0) 1.6  (0.5) 1.5  (0.4)
BSID-II Motor Scale: 6 months - 1 month 6.7  (1.3) 2.5  (0.6) 2.5  (0.5)
BSID-II Motor Scale: 6 months - 3 months 2.2  (0.6) 0.9  (0.4) 1.0  (0.4)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Umbilical Cord Blood & Erythropoietin & Rehabilitation, Erythropoietin & Rehabilitation, Only Rehabilitation
Comments The null hypothesis is that in terms of K-BSID-II MOTOR Scale, the effects of 3 groups are same, and the alternative one is that the therapeutic effect of combination intervention Group (Umbilical Cord Blood + Erythropoietin + Rehabilitation) is higher than that of either Erythropoietin + Rehabilitation or Rehabilitation Group. This is a pilot study and power calculation was not applicable. The sample size of each group is over 30 and is considered to follow approximately normal distribution.
Type of Statistical Test Superiority or Other (legacy)
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.05
Comments [Not Specified]
Method Repeated Measure ANOVA
Comments [Not Specified]
Method of Estimation Estimation Parameter Wilks' Lambda (group*visit effect)
Estimated Value 2.70
Estimation Comments [Not Specified]
5.Secondary Outcome
Title Changes in Brain MRI
Hide Description Changes on brain Diffusion Tensor Image (DTI); DTI provides quantitative information about the microscopic integrity of white matter. White matter normally possesses a high degree of diffusion anisotropy than gray matter. We can measure fractional anisotropy (FA) value in DTI imaging and it ranges from 0 to 1. Higher FA value of a certain region of interest means the area has more integrity of white matter.
Time Frame Baseline - 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Umbilical Cord Blood & Erythropoietin & Rehabilitation Erythropoietin & Rehabilitation Only Rehabilitation
Hide Arm/Group Description:
Allogeneic umbilical cord blood infusion (total nucleated cells > 3x10^7/kg intravenously), Erythropoietin injection (twice a week for 4 weeks with the dosage of 500 IU/kg for 2 times intravenously and 250 IU/kg subcutaneously for 6 times), and active rehabilitation
Erythropoietin injection (twice a week for 4 weeks with the dosage of 500 IU/kg for 2 times intravenously and 250 IU/kg subcutaneously for 6 times),and active rehabilitation
Active rehabilitation
Overall Number of Participants Analyzed 30 31 29
Mean (Standard Error)
Unit of Measure: units on a scale
Anterior portion of Rt. posterior Internal Capsule 0.03  (0.01) 0.01  (0.01) 0.01  (0.01)
Posterior portion of Rt posterior Internal Capsule 0.04  (0.01) 0.02  (0.01) 0.02  (0.02)
Anterior portion of Lt. posterior Internal Capsule 0.03  (0.01) 0.02  (0.01) 0.04  (0.02)
Posterior portion of Lt posterior Internal Capsule 0.05  (0.02) 0.04  (0.02) 0.05  (0.02)
6.Secondary Outcome
Title Comparison of Changes in Brain Glucose Metabolism Using by Brain 18F-FDG PET: Increased and Decreased Areas of Brain Glucose Metabolism
Hide Description 18F-FDG PET imaging was performed twice prior to and then 2 weeks post-treatment. Ninety slices of each emission image were obtained, and all scans were reviewed by a nuclear physician. Spatial pre-processing and statistical analyses were performed 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 reported increased areas and decreased areas of glucose metabolism in three groups. We defined that "1" refers to INCREASED areas, "-1", DECREASED areas and "0", just NO CHANGE.
Time Frame Baseline - 2 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Intention to treat
Arm/Group Title Umbilical Cord Blood & Erythropoietin & Rehabilitation Erythropoietin & Rehabilitation Only Rehabilitation
Hide Arm/Group Description:
Allogeneic umbilical cord blood infusion (total nucleated cells > 3x10^7/kg intravenously), Erythropoietin injection (twice a week for 4 weeks with the dosage of 500 IU/kg for 2 times intravenously and 250 IU/kg subcutaneously for 6 times), and active rehabilitation
Erythropoietin injection (twice a week for 4 weeks with the dosage of 500 IU/kg for 2 times intravenously and 250 IU/kg subcutaneously for 6 times),and active rehabilitation
Active rehabilitation
Overall Number of Participants Analyzed 31 33 32
Measure Type: Number
Unit of Measure: units on a scale
Rt. Lentiform Nucleus, Gray Matter, Putamen 1 0 0
Lt. Frontal Lobe, Medial Frontal Gyrus 1 0 0
Lt. Sub-lobar, Insula 1 0 -1
Lt. Lentiform Nucleus, Gray Matter, Putamen 1 0 0
Lt.Lentiform Nucleus, Gray Matter, Globus Pallidus 1 0 0
Lt. Midbrain (Thalamus) 1 0 0
Rt. Parietal Lobe, Precuneus 1 0 0
Lt. Temporal Lobe, Middle Temporal Gyrus 1 0 -1
Rt. Parietal Lobe, Pre- and Post-central Gyrus 1 0 0
Rt. Frontal Lobe, Paracentral Lobule 0 1 0
Rt. Limbic Lobe, Anterior Cingulate 0 1 0
Lt. Frontal Lobe, Precentral Gyrus 0 1 0
Lt. Lentiform Nucleus, Putamen 0 1 0
Rt. Cerebellar Anterior Lobe, Culmen 0 1 1
Lt. Cerebellar Posterior Lobe, Cerebellar Tonsil 0 1 1
Rt. Frontal Lobe, Orbital Gyrus 0 1 1
Rt. Occipital Lobe, Middle Occipital Gyrus -1 0 0
Lt. Limbic Lobe, Parahippocampal Gyrus -1 0 0
Rt. Limbic Lobe, Parahippocampal Gyrus -1 0 -1
Rt Cerebellum, Posterior Lobe, Declive 0 -1 0
Rt Cerebellum, Anterior Lobe, Culmen, Occipital 0 -1 0
Lt Cerebellum, Posterior Lobe, Declive, Occipital 0 -1 0
Lt. Frontal Lobe, Middle Frontal Gyrus 0 0 -1
Rt. Frontal Lobe, Middle Frontal Gyrus 0 0 -1
Rt. Frontal Lobe, Sub-gyral 0 0 -1
Lt. Temporal Lobe, Fusiform Gyrus 0 0 -1
Lt. Temporal Lobe, Inferior Temporal Gyrus 0 0 -1
Rt. Temporal Lobe, Middle Temporal Gyrus 0 0 -1
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Umbilical Cord Blood & Erythropoietin & Rehabilitation, Erythropoietin & Rehabilitation, Only Rehabilitation
Comments In our analysis, the null hypothesis is that the effects of three experimental groups are same each other, and the alternative hypothesis is that the therapeutic effect of combination intervention Group (Umbilical Cord Blood + Erythropoietin + Rehabilitation) has much higher than that of either Erythropoietin + Rehabilitation Group or Rehabilitation Group. This study is a pilot study and therefore, power calculation was not applicable in our study. The sample size of each group is more than 30.
Type of Statistical Test Superiority or Other (legacy)
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.05
Comments The baseline and post-therapy data of each group were compared using paired t-test statistics.
Method t-test, 2 sided
Comments Voxels with an uncorrected p-value of <0.05 were considered significant, and an extent threshold Ke of 100 voxels was set by SPM implanted in Matlab.
7.Secondary Outcome
Title Changes in Functional Performance in Daily Activities
Hide 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 reported here 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 are each domain scores measured at each assessment time point.
Time Frame Baseline -1 month - 3 months - 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
Intention to treat
Arm/Group Title Umbilical Cord Blood & Erythropoietin & Rehabilitation Erythropoietin & Rehabilitation Only Rehabilitation
Hide Arm/Group Description:
Allogeneic umbilical cord blood infusion (total nucleated cells > 3x10^7/kg intravenously), Erythropoietin injection (twice a week for 4 weeks with the dosage of 500 IU/kg for 2 times intravenously and 250 IU/kg subcutaneously for 6 times), and active rehabilitation
Erythropoietin injection (twice a week for 4 weeks with the dosage of 500 IU/kg for 2 times intravenously and 250 IU/kg subcutaneously for 6 times),and active rehabilitation
Active rehabilitation
Overall Number of Participants Analyzed 31 33 32
Mean (Standard Error)
Unit of Measure: units on a scale
Self care of FSS: Baseline 39.1  (2.5) 38.2  (2.7) 37.4  (2.2)
Self care of FSS: 1 month 42.2  (2.2) 40.4  (2.9) 40.0  (2.2)
Self care of FSS: 3 months 44.7  (2.4) 43.5  (2.9) 41.7  (2.3)
Self care of FSS: 6 months 46.5  (2.5) 45.0  (2.8) 42.8  (2.3)
Mobility of FSS: Baseline 24.8  (3.7) 29.2  (4.3) 24.9  (3.7)
Mobility of FSS: 1 month 28.1  (3.9) 32.4  (4.3) 27.9  (3.8)
Mobility of FSS: 3 months 31.7  (4.3) 34.6  (4.3) 29.3  (3.9)
Mobility of FSS: 6 months 34.6  (4.3) 36.6  (4.4) 31.4  (3.9)
Social function of FSS: Baseline 40.0  (3.5) 40.1  (3.3) 34.8  (3.5)
Social function of FSS: 1 month 42.8  (3.6) 42.5  (3.3) 37.5  (3.6)
Social function of FSS: 3 months 46.4  (3.9) 45.8  (3.3) 40.0  (3.5)
Social function of FSS: 6 months 49.1  (3.6) 47.8  (3.4) 42.3  (3.9)
Self care of CAS: Baseline 18.0  (4.1) 17.9  (3.7) 15.3  (3.2)
Self care of CAS: 1 month 20.1  (4.0) 21.1  (3.8) 19.0  (3.7)
Self care of CAS: 3 months 27.0  (4.3) 24.2  (3.9) 21.5  (3.7)
Self care of CAS: 6 months 29.4  (4.0) 26.0  (4.1) 23.1  (3.9)
Mobility of CAS: Baseline 14.6  (4.1) 19.7  (4.8) 13.5  (4.0)
Mobility of CAS: 1 month 19.6  (4.5) 22.6  (4.9) 15.6  (4.2)
Moblity of CAS: 3 months 22.5  (5.1) 26.0  (5.3) 19.3  (4.5)
Mobility of CAS: 6 months 24.9  (5.5) 29.2  (5.4) 24.0  (4.8)
Social function of CAS: Baseline 23.0  (5.2) 24.7  (3.9) 18.2  (4.4)
Social function of CAS: 1 month 28.5  (5.6) 30.0  (4.5) 21.1  (4.8)
Social function of CAS: 3 months 34.8  (6.0) 34.2  (4.8) 24.2  (5.0)
Social function of CAS: 6 months 38.4  (6.2) 36.2  (4.6) 27.6  (5.3)
8.Secondary Outcome
Title Changes in Functional Independence in Daily Activities
Hide 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. Categories of outcome table are total WeeFIM scores measured at each assessment time point.
Time Frame Baseline - 1 month - 3 months - 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
Intention to treat
Arm/Group Title Umbilical Cord Blood & Erythropoietin & Rehabilitation Erythropoietin & Rehabilitation Only Rehabilitation
Hide Arm/Group Description:
Allogeneic umbilical cord blood infusion (total nucleated cells > 3x10^7/kg intravenously), Erythropoietin injection (twice a week for 4 weeks with the dosage of 500 IU/kg for 2 times intravenously and 250 IU/kg subcutaneously for 6 times), and active rehabilitation
Erythropoietin injection (twice a week for 4 weeks with the dosage of 500 IU/kg for 2 times intravenously and 250 IU/kg subcutaneously for 6 times),and active rehabilitation
Active rehabilitation
Overall Number of Participants Analyzed 31 33 32
Mean (Standard Error)
Unit of Measure: units on a scale
Baseline 34.1  (3.8) 36.6  (3.7) 31.4  (2.9)
1 month 35.6  (4.1) 37.5  (3.8) 33.2  (3.1)
3 months 38.7  (4.7) 38.5  (4.1) 37.1  (3.8)
6 months 41.1  (4.8) 40.3  (4.2) 37.7  (4.1)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Umbilical Cord Blood & Erythropoietin & Rehabilitation, Erythropoietin & Rehabilitation, Only Rehabilitation
Comments In our analysis, the null hypothesis is that the effects of 3 experimental groups are same, and the alternative hypothesis is that the therapeutic effect of combination intervention Group (Umbilical Cord Blood + Erythropoietin + Rehabilitation) is higher than that of either Erythropoietin + Rehabilitation or Rehabilitation Group. This is a pilot study and power calculation was not applicable. The sample size of each group is over 30 and is considered to follow approximately normal distribution.
Type of Statistical Test Superiority or Other (legacy)
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.05
Comments [Not Specified]
Method Repeated Measure ANOVA
Comments [Not Specified]
Method of Estimation Estimation Parameter Wilks' Lambda (group*visit effect)
Estimated Value 1.279
Estimation Comments [Not Specified]
9.Secondary Outcome
Title Changes in Muscle Strength
Hide 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 score means better muscle strength. Categories of outcome table are summation of MMT scores measured at each assessment time point.
Time Frame Baseline - 1 month - 3 months - 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
Intention to treat
Arm/Group Title Umbilical Cord Blood & Erythropoietin & Rehabilitation Erythropoietin & Rehabilitation Only Rehabilitation
Hide Arm/Group Description:
Allogeneic umbilical cord blood infusion (total nucleated cells > 3x10^7/kg intravenously), Erythropoietin injection (twice a week for 4 weeks with the dosage of 500 IU/kg for 2 times intravenously and 250 IU/kg subcutaneously for 6 times), and active rehabilitation
Erythropoietin injection (twice a week for 4 weeks with the dosage of 500 IU/kg for 2 times intravenously and 250 IU/kg subcutaneously for 6 times),and active rehabilitation
Active rehabilitation
Overall Number of Participants Analyzed 31 33 32
Mean (Standard Error)
Unit of Measure: units on a scale
Baseline 100.7  (5.5) 104.8  (4.4) 100.3  (4.6)
1 month 105.0  (4.9) 106.8  (4.5) 101.8  (4.6)
3 months 107.8  (4.4) 107.7  (4.5) 103.6  (4.6)
6 months 109.9  (4.6) 109.3  (4.5) 104.3  (4.6)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Umbilical Cord Blood & Erythropoietin & Rehabilitation, Erythropoietin & Rehabilitation, Only Rehabilitation
Comments In our analysis, the null hypothesis is that the effects of 3 experimental groups are same, and the alternative hypothesis is that the therapeutic effect of combination intervention Group (Umbilical Cord Blood + Erythropoietin + Rehabilitation) is higher than that of either Erythropoietin + Rehabilitation or Rehabilitation Group. This is a pilot study and power calculation was not applicable. The sample size of each group is over 30 and is considered to follow approximately normal distribution.
Type of Statistical Test Superiority or Other (legacy)
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.05
Comments [Not Specified]
Method Repeated Measure ANOVA
Comments [Not Specified]
Method of Estimation Estimation Parameter Wilks' Lambda (group*visit effect)
Estimated Value 0.996
Estimation Comments [Not Specified]
10.Secondary Outcome
Title Changes in Hand Function
Hide Description QUEST (Quality of Upper Extremity Skills Test) as a standardized measurement tool for assessing hand function consisting of sub-scales; dissociated movement, grasps, weight bearing, and protective extension. These are standardized to range from zero (or below zero in grasp section) to 100 and higher values mean better hand function. We reported QUEST differences between each assessment times.
Time Frame Baseline - 1 month - 3 months - 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
Intention to treat
Arm/Group Title Umbilical Cord Blood & Erythropoietin & Rehabilitation Erythropoietin & Rehabilitation Only Rehabilitation
Hide Arm/Group Description:
Allogeneic umbilical cord blood infusion (total nucleated cells > 3x10^7/kg intravenously), Erythropoietin injection (twice a week for 4 weeks with the dosage of 500 IU/kg for 2 times intravenously and 250 IU/kg subcutaneously for 6 times), and active rehabilitation
Erythropoietin injection (twice a week for 4 weeks with the dosage of 500 IU/kg for 2 times intravenously and 250 IU/kg subcutaneously for 6 times),and active rehabilitation
Active rehabilitation
Overall Number of Participants Analyzed 31 33 32
Mean (Standard Error)
Unit of Measure: units on a scale
Baseline 36.0  (6.4) 52.8  (4.9) 43.0  (5.5)
1 month - Baseline 5.0  (1.4) 5.7  (1.7) 6.6  (1.4)
3 months - Baseline 11.1  (2.2) 8.8  (2.0) 10.5  (1.8)
6 months - Baseline 13.9  (2.6) 10.7  (2.2) 13.0  (2.4)
3 months - 1 month 5.7  (1.6) 3.1  (1.0) 3.5  (1.7)
6 months - 1 month 8.4  (2.0) 5.1  (1.3) 6.4  (2.1)
6 months - 3 months 2.8  (0.8) 2.4  (0.8) 3.1  (1.2)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Umbilical Cord Blood & Erythropoietin & Rehabilitation, Erythropoietin & Rehabilitation, Only Rehabilitation
Comments In our analysis, the null hypothesis is that the effects of 3 experimental groups are same, and the alternative hypothesis is that the therapeutic effect of combination intervention Group (Umbilical Cord Blood + Erythropoietin + Rehabilitation) is higher than that of either Erythropoietin + Rehabilitation or Rehabilitation Group. This is a pilot study and power calculation was not applicable. The sample size of each group is over 30 and is considered to follow approximately normal distribution.
Type of Statistical Test Superiority or Other (legacy)
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.05
Comments [Not Specified]
Method Repeated Measure ANOVA
Comments [Not Specified]
Method of Estimation Estimation Parameter Wilks' Lambda (group*visit effect)
Estimated Value 0.56
Estimation Comments [Not Specified]
11.Secondary Outcome
Title Number of Participants With Serious Adverse Events as a Measure of Safety,Which Are Related to Umbilical Cord Blood, Erythropoietin, or Immunosuppressant
Hide Description The number of patients with serious adverse events within each group; Serious adverse events were defined as any event that resulted in death, was life-threatening, required hospitalization or prolonged the hospital stay, or was otherwise serious in the judgment of the investigator.
Time Frame 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Umbilical Cord Blood & Erythropoietin & Rehabilitation Erythropoietin & Rehabilitation Only Rehabilitation
Hide Arm/Group Description:
Allogeneic umbilical cord blood infusion (total nucleated cells > 3x10^7/kg intravenously), Erythropoietin injection (twice a week for 4 weeks with the dosage of 500 IU/kg for 2 times intravenously and 250 IU/kg subcutaneously for 6 times), and active rehabilitation
Erythropoietin injection (twice a week for 4 weeks with the dosage of 500 IU/kg for 2 times intravenously and 250 IU/kg subcutaneously for 6 times),and active rehabilitation
Active rehabilitation
Overall Number of Participants Analyzed 31 33 32
Measure Type: Number
Unit of Measure: participants
3 3 3
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Umbilical Cord Blood & Erythropoietin & Rehabilitation, Erythropoietin & Rehabilitation, Only Rehabilitation
Comments [Not Specified]
Type of Statistical Test Superiority or Other (legacy)
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.05
Comments [Not Specified]
Method Fisher Exact
Comments We compared the ratio of participants with a certain adverse event (AE) and without the AE between three groups using Fisher Exact test.
Time Frame Baseline - 1 month - 3 months - 6 months
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Umbilical Cord Blood & Erythropoietin & Rehabilitation Erythropoietin & Rehabilitation Only Rehabilitation
Hide Arm/Group Description Allogeneic umbilical cord blood infusion (total nucleated cells > 3x10^7/kg intravenously), Erythropoietin injection (twice a week for 4 weeks with the dosage of 500 IU/kg for 2 times intravenously and 250 IU/kg subcutaneously for 6 times), and active rehabilitation Erythropoietin injection (twice a week for 4 weeks with the dosage of 500 IU/kg for 2 times intravenously and 250 IU/kg subcutaneously for 6 times),and active rehabilitation Active rehabilitation
All-Cause Mortality
Umbilical Cord Blood & Erythropoietin & Rehabilitation Erythropoietin & Rehabilitation Only Rehabilitation
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   --/--      --/--      --/--    
Hide Serious Adverse Events
Umbilical Cord Blood & Erythropoietin & Rehabilitation Erythropoietin & Rehabilitation Only Rehabilitation
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   3/35 (8.57%)      3/6 (50.00%)      3/8 (37.50%)    
Nervous system disorders       
Seizure † 1  0/35 (0.00%)  0 1/6 (16.67%)  1 0/8 (0.00%)  0
Renal and urinary disorders       
Urinary tract infection † 1  0/35 (0.00%)  0 0/6 (0.00%)  0 1/8 (12.50%)  1
Respiratory, thoracic and mediastinal disorders       
Pneumonia † 1  1/35 (2.86%)  1 2/6 (33.33%)  3 1/8 (12.50%)  1
Influenza † 1  1/35 (2.86%)  1 0/6 (0.00%)  0 1/8 (12.50%)  1
Death † 1 [1]  1/35 (2.86%)  1 0/6 (0.00%)  0 0/8 (0.00%)  0
†
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 14.1
[1]
One death at 14 wks post-transplantation. She was a 25 mo-old with spastic quadriplegia from profound hypoxia. She was unable to control her head and showed poor oral function. She had been medically stable. The cause was supposed to be suffocation.
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Umbilical Cord Blood & Erythropoietin & Rehabilitation Erythropoietin & Rehabilitation Only Rehabilitation
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   30/35 (85.71%)      29/36 (80.56%)      32/34 (94.12%)    
Blood and lymphatic system disorders       
Anemia † 1  1/35 (2.86%)  1 0/0  0 0/18 (0.00%)  0
Ear and labyrinth disorders       
Otitis media acute † 1  1/35 (2.86%)  1 1/14 (7.14%)  1 0/18 (0.00%)  0
Eye disorders       
Conjunctival irritations † 1  0/35 (0.00%)  0 1/14 (7.14%)  1 1/18 (5.56%)  2
Gastrointestinal disorders       
Constipation † 1  5/35 (14.29%)  5 4/14 (28.57%)  4 5/18 (27.78%)  5
Dyspepsia † 1  5/35 (14.29%)  5 2/36 (5.56%)  2 2/18 (11.11%)  2
Nausea, vomiting † 1  6/35 (17.14%)  6 5/36 (13.89%)  5 2/18 (11.11%)  2
Diarrhea † 1  6/35 (17.14%)  6 2/36 (5.56%)  2 2/18 (11.11%)  2
Colitis † 1  0/35 (0.00%)  0 1/14 (7.14%)  1 2/18 (11.11%)  2
Anorexia † 1  5/35 (14.29%)  5 2/36 (5.56%)  2 1/18 (5.56%)  1
Infections and infestations       
Herpangina † 1  0/35 (0.00%)  0 2/14 (14.29%)  2 1/18 (5.56%)  1
Fever † 1  12/35 (34.29%)  12 4/36 (11.11%)  4 8/18 (44.44%)  8
Nervous system disorders       
Seizure † 1  1/35 (2.86%)  1 3/36 (8.33%)  6 3/18 (16.67%)  7
Febrile convulsioin † 1  2/35 (5.71%)  3 1/14 (7.14%)  1 0/18 (0.00%)  0
Psychiatric disorders       
Irritability † 1  4/35 (11.43%)  4 0/14 (0.00%)  0 0/18 (0.00%)  0
Insomnia † 1  0/35 (0.00%)  0 1/36 (2.78%)  1 1/18 (5.56%)  1
Respiratory, thoracic and mediastinal disorders       
Bronchitis † 1  4/35 (11.43%)  4 4/14 (28.57%)  4 3/18 (16.67%)  3
Pneumonia † 1  6/35 (17.14%)  6 0/14 (0.00%)  0 0/18 (0.00%)  0
Upper respiratory tract infection † 1  18/35 (51.43%)  20 19/36 (52.78%)  23 21/34 (61.76%)  24
Hypoxia † 1 [1]  3/35 (8.57%)  3 1/36 (2.78%)  1 1/18 (5.56%)  1
Skin and subcutaneous tissue disorders       
Dermatitis † 1  0/35 (0.00%)  0 2/36 (5.56%)  2 2/18 (11.11%)  2
Urticaria † 1  2/35 (5.71%)  2 1/14 (7.14%)  1 4/18 (22.22%)  4
Hirsuitism † 1  2/35 (5.71%)  2 0/0  0 0/0  0
Alopecia † 1  1/35 (2.86%)  1 0/0  0 0/0  0
†
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 14.1
[1]
Temporary O2 desaturation(89-91%) at the end of UCB infusion in UCB group; O2 desaturation(91%) shortly during sedation for PET in EPO group; O2 desaturation(71-95%) intermittently during 47 min maybe from postictal respiratory depression in control
[Not Specified]
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Minyoung Kim, MD, PhD
Organization: CHA Bundang Medical Center, CHA University
Phone: 82-31-780-1872
EMail: kmin@cha.ac.kr
Layout table for additonal information
Responsible Party: Sung Kwang Medical Foundation
ClinicalTrials.gov Identifier: NCT01193660    
Other Study ID Numbers: RCTUBSC
PBC09-095 ( Other Identifier: IRB of CHA Bundang Medical Center )
First Submitted: August 29, 2010
First Posted: September 2, 2010
Results First Submitted: October 11, 2011
Results First Posted: March 1, 2012
Last Update Posted: November 24, 2020