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

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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

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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
Total	Total of all reporting groups

Baseline Measures

	Umbilical Cord Blood & Erythropoietin & Rehabilitation	Erythropoietin & Rehabilitation	Only Rehabilitation	Total
Number of Participants [units: participants]	35	36	34	105
Age [units: participants]
<=18 years	35	36	34	105
Between 18 and 65 years	0	0	0	0
>=65 years	0	0	0	0
Age [units: months] Mean ± Standard Deviation	45.71 ± 18.91	50.05 ± 24.45	46.0 ± 19.58	47.30 ± 21.07
Gender [units: participants]
Female	13	11	9	33
Male	22	25	25	72
Region of Enrollment [units: participants]
Korea, Republic of	35	36	34	105

Outcome Measures

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1. Primary:

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

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2. Primary:

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

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3. Secondary:

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

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4. Secondary:

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

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5. Secondary:

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

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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 ]

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7. Secondary:

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

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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 ]

Show Outcome Measure 8

9. Secondary:

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

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10. Secondary:

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

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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 method 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 ]

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Serious Adverse Events

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Other Adverse Events

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Limitations and Caveats

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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
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