Allogenic Umbilical Cord Blood and Erythropoietin Combination Therapy for Cerebral Palsy

This study has been completed.

Sponsor:

Information provided by (Responsible Party):

Sung Kwang Medical Foundation

ClinicalTrials.gov Identifier:

NCT01193660

First received: August 29, 2010

Last updated: March 1, 2012

Last verified: March 2012

History of Changes

Results First Received: October 11, 2011

Study Type:	Interventional
Study Design:	Allocation: Randomized; Endpoint Classification: Safety/Efficacy Study; Intervention Model: Parallel Assignment; Masking: Double Blind (Subject, Caregiver, 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

Participant Flow

Hide Participant Flow

Recruitment Details

Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
Enrollment Period: May 31, 2010 through April 9, 2011 All participants were recruited at CHA Bundang Medical Center.

Pre-Assignment Details

Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
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.

Reporting Groups

	Description
Umbilical Cord Blood & Erythropoietin & Rehabilitation	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 & 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
Only Rehabilitation	Active rehabilitation

Participant Flow: Overall Study

	Umbilical Cord Blood & Erythropoietin & Rehabilitation	Erythropoietin & Rehabilitation	Only Rehabilitation
STARTED	35	36	34
COMPLETED	31	33	32
NOT COMPLETED	4	3	2
Withdrawal by Subject	2	3	1
Death	1	0	0
Physician Decision	1	0	1

Baseline Characteristics

Show Baseline Characteristics

Outcome Measures

Hide All Outcome Measures

1. Primary:

Changes in Motor Performance [ Time Frame: Baseline -1 month - 3 months - 6 months ]

Measure Type	Primary
Measure Title	Changes in Motor Performance
Measure 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
Safety Issue	No

Population Description

Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
Intention to treat

Reporting Groups

	Description
Umbilical Cord Blood & Erythropoietin & Rehabilitation	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 & 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
Only Rehabilitation	Active rehabilitation

Measured Values

	Umbilical Cord Blood & Erythropoietin & Rehabilitation	Erythropoietin & Rehabilitation	Only Rehabilitation
Number of Participants Analyzed [units: participants]	31	33	32
Changes in Motor Performance [units: units on a scale] Mean ± Standard Error
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

Statistical Analysis 1 for Changes in Motor Performance

Groups ^[1]	All groups
Method ^[2]	Repeated Measure ANOVA
P Value ^[3]	<0.05
Wilks' Lambda (group*visit effect) ^[4]	2.59

[1]	Additional details about the analysis, such as null hypothesis and power calculation:
	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.
[2]	Other relevant information, such as adjustments or degrees of freedom:
	No text entered.
[3]	Additional information, such as whether or not the p-value is adjusted for multiple comparisons and the a priori threshold for statistical significance:
	No text entered.
[4]	Other relevant estimation information:
	No text entered.

2. Primary:

Changes in Standardized Gross Motor Function [ Time Frame: Baseline - 1 month - 3 months - 6 months ]

Measure Type	Primary
Measure Title	Changes in Standardized Gross Motor Function
Measure 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
Safety Issue	No

Population Description

Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
Intention to treat

Reporting Groups

	Description
Umbilical Cord Blood & Erythropoietin & Rehabilitation	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 & 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
Only Rehabilitation	Active rehabilitation

Measured Values

	Umbilical Cord Blood & Erythropoietin & Rehabilitation	Erythropoietin & Rehabilitation	Only Rehabilitation
Number of Participants Analyzed [units: participants]	31	33	32
Changes in Standardized Gross Motor Function [units: units on a scale] Mean ± Standard Error
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

Statistical Analysis 1 for Changes in Standardized Gross Motor Function

Groups ^[1]	All groups
Method ^[2]	Repeated Measure ANOVA
P Value ^[3]	<0.05
interaction of group and visit ^[4]	0.90

[1]	Additional details about the analysis, such as null hypothesis and power calculation:
	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.
[2]	Other relevant information, such as adjustments or degrees of freedom:
	No text entered.
[3]	Additional information, such as whether or not the p-value is adjusted for multiple comparisons and the a priori threshold for statistical significance:
	No text entered.
[4]	Other relevant estimation information:
	No text entered.

3. Secondary:

Changes in Cognitive Neurodevelopmental Outcome [ Time Frame: Baseline -1 month - 3 months - 6 months ]

Measure Type	Secondary
Measure Title	Changes in Cognitive Neurodevelopmental Outcome
Measure 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
Safety Issue	No

Population Description

Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
Intention to treat

Reporting Groups

	Description
Umbilical Cord Blood & Erythropoietin & Rehabilitation	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 & 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
Only Rehabilitation	Active rehabilitation

Measured Values

	Umbilical Cord Blood & Erythropoietin & Rehabilitation	Erythropoietin & Rehabilitation	Only Rehabilitation
Number of Participants Analyzed [units: participants]	31	33	32
Changes in Cognitive Neurodevelopmental Outcome [units: units on a scale] Mean ± Standard Error
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

Statistical Analysis 1 for Changes in Cognitive Neurodevelopmental Outcome

Groups ^[1]	All groups
Method ^[2]	Repeated Measure ANOVA
P Value ^[3]	<0.05
Wilks' Lambda (group*visit effect) ^[4]	3.94

[1]	Additional details about the analysis, such as null hypothesis and power calculation:
	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.
[2]	Other relevant information, such as adjustments or degrees of freedom:
	No text entered.
[3]	Additional information, such as whether or not the p-value is adjusted for multiple comparisons and the a priori threshold for statistical significance:
	No text entered.
[4]	Other relevant estimation information:
	No text entered.

4. Secondary:

Changes in Motor Neurodevelopmental Outcome [ Time Frame: Baseline - 1 month - 3 months - 6 months ]

Measure Type	Secondary
Measure Title	Changes in Motor Neurodevelopmental Outcome
Measure 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
Safety Issue	No

Population Description

Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
Intention to treat

Reporting Groups

	Description
Umbilical Cord Blood & Erythropoietin & Rehabilitation	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 & 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
Only Rehabilitation	Active rehabilitation

Measured Values

	Umbilical Cord Blood & Erythropoietin & Rehabilitation	Erythropoietin & Rehabilitation	Only Rehabilitation
Number of Participants Analyzed [units: participants]	31	33	32
Changes in Motor Neurodevelopmental Outcome [units: units on a scale] Mean ± Standard Error
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

Statistical Analysis 1 for Changes in Motor Neurodevelopmental Outcome

Groups ^[1]	All groups
Method ^[2]	Repeated Measure ANOVA
P Value ^[3]	<0.05
Wilks' Lambda (group*visit effect) ^[4]	2.70

[1]	Additional details about the analysis, such as null hypothesis and power calculation:
	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.
[2]	Other relevant information, such as adjustments or degrees of freedom:
	No text entered.
[3]	Additional information, such as whether or not the p-value is adjusted for multiple comparisons and the a priori threshold for statistical significance:
	No text entered.
[4]	Other relevant estimation information:
	No text entered.

5. Secondary:

Changes in Brain MRI [ Time Frame: Baseline - 6 months ]

Measure Type	Secondary
Measure Title	Changes in Brain MRI
Measure 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
Safety Issue	No

Population Description

Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups

	Description
Umbilical Cord Blood & Erythropoietin & Rehabilitation	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 & 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
Only Rehabilitation	Active rehabilitation

Measured Values

	Umbilical Cord Blood & Erythropoietin & Rehabilitation	Erythropoietin & Rehabilitation	Only Rehabilitation
Number of Participants Analyzed [units: participants]	30	31	29
Changes in Brain MRI [units: units on a scale] Mean ± Standard Error
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

No statistical analysis provided for Changes in Brain MRI

6. Secondary:

Comparison of Changes in Brain Glucose Metabolism Using by Brain 18F-FDG PET: Increased and Decreased Areas of Brain Glucose Metabolism [ Time Frame: Baseline - 2 weeks ]

Measure Type	Secondary
Measure Title	Comparison of Changes in Brain Glucose Metabolism Using by Brain 18F-FDG PET: Increased and Decreased Areas of Brain Glucose Metabolism
Measure 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
Safety Issue	No

Population Description

Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
Intention to treat

Reporting Groups

	Description
Umbilical Cord Blood & Erythropoietin & Rehabilitation	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 & 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
Only Rehabilitation	Active rehabilitation

Measured Values

	Umbilical Cord Blood & Erythropoietin & Rehabilitation	Erythropoietin & Rehabilitation	Only Rehabilitation
Number of Participants Analyzed [units: participants]	31	33	32
Comparison of Changes in Brain Glucose Metabolism Using by Brain 18F-FDG PET: Increased and Decreased Areas of Brain Glucose Metabolism [units: 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	0
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	0
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	0
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
Lt. Sub-lobar, Insula	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
Lt. Temporal Lobe, Middle Temporal Gyrus	0	0	-1
Rt. Temporal Lobe, Middle Temporal Gyrus	0	0	-1
Rt. Limbic Lobe, Anterior Cingulate	0	0	-1

Statistical Analysis 1 for Comparison of Changes in Brain Glucose Metabolism Using by Brain 18F-FDG PET: Increased and Decreased Areas of Brain Glucose Metabolism

Groups ^[1]	All groups
Method ^[2]	t-test, 2 sided
P Value ^[3]	0.05

[1]	Additional details about the analysis, such as null hypothesis and power calculation:
	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.
[2]	Other relevant information, such as adjustments or degrees of freedom:
	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.
[3]	Additional information, such as whether or not the p-value is adjusted for multiple comparisons and the a priori threshold for statistical significance:
	The baseline and post-therapy data of each group were compared using paired t-test statistics.

7. Secondary:

Changes in Functional Performance in Daily Activities [ Time Frame: Baseline -1 month - 3 months - 6 months ]

Measure Type	Secondary
Measure Title	Changes in Functional Performance in Daily Activities
Measure 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
Safety Issue	No

Population Description

Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
Intention to treat

Reporting Groups

	Description
Umbilical Cord Blood & Erythropoietin & Rehabilitation	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 & 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
Only Rehabilitation	Active rehabilitation

Measured Values

	Umbilical Cord Blood & Erythropoietin & Rehabilitation	Erythropoietin & Rehabilitation	Only Rehabilitation
Number of Participants Analyzed [units: participants]	31	33	32
Changes in Functional Performance in Daily Activities [units: units on a scale] Mean ± Standard Error
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

No statistical analysis provided for Changes in Functional Performance in Daily Activities

8. Secondary:

Changes in Functional Independence in Daily Activities [ Time Frame: Baseline - 1 month - 3 months - 6 months ]

Measure Type	Secondary
Measure Title	Changes in Functional Independence in Daily Activities
Measure 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
Safety Issue	No

Population Description

Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
Intention to treat

Reporting Groups

	Description
Umbilical Cord Blood & Erythropoietin & Rehabilitation	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 & 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
Only Rehabilitation	Active rehabilitation

Measured Values

	Umbilical Cord Blood & Erythropoietin & Rehabilitation	Erythropoietin & Rehabilitation	Only Rehabilitation
Number of Participants Analyzed [units: participants]	31	33	32
Changes in Functional Independence in Daily Activities [units: units on a scale] Mean ± Standard Error
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

Statistical Analysis 1 for Changes in Functional Independence in Daily Activities

Groups ^[1]	All groups
Method ^[2]	Repeated Measure ANOVA
P Value ^[3]	<0.05
Wilks' Lambda (group*visit effect) ^[4]	1.279

[1]	Additional details about the analysis, such as null hypothesis and power calculation:
	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.
[2]	Other relevant information, such as adjustments or degrees of freedom:
	No text entered.
[3]	Additional information, such as whether or not the p-value is adjusted for multiple comparisons and the a priori threshold for statistical significance:
	No text entered.
[4]	Other relevant estimation information:
	No text entered.

9. Secondary:

Changes in Muscle Strength [ Time Frame: Baseline - 1 month - 3 months - 6 months ]

Measure Type	Secondary
Measure Title	Changes in Muscle Strength
Measure 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
Safety Issue	No

Population Description

Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
Intention to treat

Reporting Groups

	Description
Umbilical Cord Blood & Erythropoietin & Rehabilitation	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 & 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
Only Rehabilitation	Active rehabilitation

Measured Values

	Umbilical Cord Blood & Erythropoietin & Rehabilitation	Erythropoietin & Rehabilitation	Only Rehabilitation
Number of Participants Analyzed [units: participants]	31	33	32
Changes in Muscle Strength [units: units on a scale] Mean ± Standard Error
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

Statistical Analysis 1 for Changes in Muscle Strength

Groups ^[1]	All groups
Method ^[2]	Repeated Measure ANOVA
P Value ^[3]	<0.05
Wilks' Lambda (group*visit effect) ^[4]	0.996

[1]	Additional details about the analysis, such as null hypothesis and power calculation:
	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.
[2]	Other relevant information, such as adjustments or degrees of freedom:
	No text entered.
[3]	Additional information, such as whether or not the p-value is adjusted for multiple comparisons and the a priori threshold for statistical significance:
	No text entered.
[4]	Other relevant estimation information:
	No text entered.

10. Secondary:

Changes in Hand Function [ Time Frame: Baseline - 1 month - 3 months - 6 months ]

Measure Type	Secondary
Measure Title	Changes in Hand Function
Measure 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
Safety Issue	No

Population Description

Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
Intention to treat

Reporting Groups

	Description
Umbilical Cord Blood & Erythropoietin & Rehabilitation	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 & 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
Only Rehabilitation	Active rehabilitation

Measured Values

	Umbilical Cord Blood & Erythropoietin & Rehabilitation	Erythropoietin & Rehabilitation	Only Rehabilitation
Number of Participants Analyzed [units: participants]	31	33	32
Changes in Hand Function [units: units on a scale] Mean ± Standard Error
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

Statistical Analysis 1 for Changes in Hand Function

Groups ^[1]	All groups
Method ^[2]	Repeated Measure ANOVA
P Value ^[3]	<0.05
Wilks' Lambda (group*visit effect) ^[4]	0.56

[1]	Additional details about the analysis, such as null hypothesis and power calculation:
	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.
[2]	Other relevant information, such as adjustments or degrees of freedom:
	No text entered.
[3]	Additional information, such as whether or not the p-value is adjusted for multiple comparisons and the a priori threshold for statistical significance:
	No text entered.
[4]	Other relevant estimation information:
	No text entered.

11. Secondary:

Number of Participants With Serious Adverse Events as a Measure of Safety,Which Are Related to Umbilical Cord Blood, Erythropoietin, or Immunosuppressant [ Time Frame: 6 months ]

Measure Type	Secondary
Measure Title	Number of Participants With Serious Adverse Events as a Measure of Safety,Which Are Related to Umbilical Cord Blood, Erythropoietin, or Immunosuppressant
Measure 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
Safety Issue	Yes

Population Description

Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups

	Description
Umbilical Cord Blood & Erythropoietin & Rehabilitation	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 & 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
Only Rehabilitation	Active rehabilitation

Measured Values

	Umbilical Cord Blood & Erythropoietin & Rehabilitation	Erythropoietin & Rehabilitation	Only Rehabilitation
Number of Participants Analyzed [units: participants]	31	33	32
Number of Participants With Serious Adverse Events as a Measure of Safety,Which Are Related to Umbilical Cord Blood, Erythropoietin, or Immunosuppressant [units: participants]	3	3	3

Statistical Analysis 1 for Number of Participants With Serious Adverse Events as a Measure of Safety,Which Are Related to Umbilical Cord Blood, Erythropoietin, or Immunosuppressant

Groups ^[1]	All groups
Method ^[2]	Fisher Exact
P Value ^[3]	<0.05

[1]	Additional details about the analysis, such as null hypothesis and power calculation:
	No text entered.
[2]	Other relevant information, such as adjustments or degrees of freedom:
	We compared the ratio of participants with a certain adverse event (AE) and without the AE between three groups using Fisher Exact test.
[3]	Additional information, such as whether or not the p-value is adjusted for multiple comparisons and the a priori threshold for statistical significance:
	No text entered.

Serious Adverse Events

Show Serious Adverse Events

Other Adverse Events

Show Other Adverse Events

More Information

Hide More Information

Certain Agreements:

All Principal Investigators ARE employed by the organization sponsoring the study.

Limitations and Caveats

Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
No text entered.

Results Point of Contact:

Name/Title: Minyoung Kim, MD, PhD
Organization: CHA Bundang Medical Center, CHA University
phone: 82-31-780-1872
e-mail: kmin@cha.ac.kr

No publications provided

Responsible Party:	Sung Kwang Medical Foundation
ClinicalTrials.gov Identifier:	NCT01193660 History of Changes
Other Study ID Numbers:	RCTUBSC, PBC09-095
Study First Received:	August 29, 2010
Results First Received:	October 11, 2011
Last Updated:	March 1, 2012
Health Authority:	Korea: Institutional Review Board

To Top