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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
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


  Outcome Measures
  Show All Outcome Measures

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 ]

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 ]
  Hide Outcome Measure 6

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 method 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 ]

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

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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