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
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 ]

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

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

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

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

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 ]

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

8.  Secondary:   Changes in Functional Independence in Daily Activities   [ Time Frame: Baseline - 1 month - 3 months - 6 months ]
  Hide Outcome Measure 8

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 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.



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

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

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 ]


  Serious Adverse Events


  Other Adverse Events


  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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