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Allogenic Umbilical Cord Blood and Erythropoietin Combination Therapy for Cerebral Palsy

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ClinicalTrials.gov Identifier: NCT01193660
Recruitment Status : Completed
First Posted : September 2, 2010
Results First Posted : March 1, 2012
Last Update Posted : March 5, 2012
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

  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

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 
Overall Participants Analyzed 
[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

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 ]
  Show Outcome Measure 3

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

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  

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 
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
Statistical Test Type [2] Superiority or Other
Statistical Method [3] Repeated Measure ANOVA
P Value [4] <0.05
Wilks' Lambda (group*visit effect) [5] 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] Details of power calculation, definition of non-inferiority margin, and other key parameters:
  No text entered.
[3] Other relevant method information, such as adjustments or degrees of freedom:
  No text entered.
[4] 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.
[5] 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 ]
  Show Outcome Measure 7

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 ]
  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


  Other Adverse Events


  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.


  More Information

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 )
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: March 5, 2012