Allogenic Umbilical Cord Blood and Erythropoietin Combination Therapy for Cerebral Palsy
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| ClinicalTrials.gov Identifier: NCT01193660 |
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Recruitment Status
:
Completed
First Posted
: September 2, 2010
Results First Posted
: March 1, 2012
Last Update Posted
: March 5, 2012
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Sponsor:
Sung Kwang Medical Foundation
Information provided by (Responsible Party):
Sung Kwang Medical Foundation
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| Study Type: | Interventional |
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| 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 |
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| 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 |
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| 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 |
Show Baseline Characteristics
Outcome Measures
| 1. Primary: | Changes in Motor Performance [ Time Frame: Baseline -1 month - 3 months - 6 months ] |
Hide Outcome Measure 1
| Measure Type | Primary |
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| Measure Title | Changes in Motor Performance |
| Measure Description | GMPM (Gross Motor Performance Measure) as a standardized measurement tool for assessing quality of movement regarding 3 properties of 5 ones; alignment, coordination, dissociated movement, stability, and weight shift (range: 0~100, Higher value means better motor quality). We reported changes of GMPM score between each assessment time points. Categories of outcome table are baseline and values of just subtracting the latter raw 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. |
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| 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 | |
|---|---|---|---|
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Participants Analyzed [Units: Participants] |
31 | 33 | 32 |
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Changes in Motor Performance [Units: Units on a scale] Mean (Standard Error) |
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| Baseline | 34.5 (2.7) | 38.2 (2.8) | 35.5 (2.9) |
| 1 month - Baseline | 7.00 (1.3) | 4.5 (0.6) | 6.0 (1.0) |
| 3 months - Baseline | 11.5 (1.5) | 7.5 (0.8) | 8.1 (1.2) |
| 6 months - Baseline | 14.5 (1.8) | 9.2 (0.8) | 9.6 (1.2) |
| 3 months - 1 month | 4.5 (0.7) | 2.9 (0.5) | 2.1 (0.6) |
| 6 months - 1 month | 7.5 (0.9) | 4.7 (0.7) | 3.6 (0.7) |
| 6 months - 3 months | 3.1 (0.6) | 1.7 (0.5) | 1.5 (0.4) |
Statistical Analysis 1 for Changes in Motor Performance
| Groups [1] | All groups |
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| Statistical Test Type [2] | Superiority or Other |
| Statistical Method [3] | Repeated Measure ANOVA |
| P Value [4] | <0.05 |
| Wilks' Lambda (group*visit effect) [5] | 2.59 |
| [1] | Additional details about the analysis, such as null hypothesis and power calculation: |
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| 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] | Details of power calculation, definition of non-inferiority margin, and other key parameters: |
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| [3] | Other relevant method information, such as adjustments or degrees of freedom: |
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| [4] | Additional information, such as whether or not the p-value is adjusted for multiple comparisons and the a priori threshold for statistical significance: |
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| [5] | Other relevant estimation information: |
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| 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 ] |
| 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 ] |
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 |
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