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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 ]
  Show Outcome Measure 1

2.  Primary:   Changes in Standardized Gross Motor Function   [ Time Frame: Baseline - 1 month - 3 months - 6 months ]
  Show Outcome Measure 2

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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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 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.



5.  Secondary:   Changes in Brain MRI   [ Time Frame: Baseline - 6 months ]
  Show Outcome Measure 5

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 ]
  Show Outcome Measure 6

7.  Secondary:   Changes in Functional Performance in Daily Activities   [ Time Frame: Baseline -1 month - 3 months - 6 months ]
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8.  Secondary:   Changes in Functional Independence in Daily Activities   [ Time Frame: Baseline - 1 month - 3 months - 6 months ]
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9.  Secondary:   Changes in Muscle Strength   [ Time Frame: Baseline - 1 month - 3 months - 6 months ]
  Show Outcome Measure 9

10.  Secondary:   Changes in Hand Function   [ Time Frame: Baseline - 1 month - 3 months - 6 months ]
  Show Outcome Measure 10

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 ]
  Show Outcome Measure 11


  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
No text entered.


  More Information
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Certain Agreements:  
All Principal Investigators ARE employed by the organization sponsoring the study.


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



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