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A Randomized Study of Autologous Umbilical Cord Blood Reinfusion in Children With Cerebral Palsy

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ClinicalTrials.gov Identifier: NCT01147653
Recruitment Status : Completed
First Posted : June 22, 2010
Results First Posted : June 12, 2017
Last Update Posted : November 30, 2017
Sponsor:
Collaborator:
The Robertson Foundation
Information provided by (Responsible Party):
Joanne Kurtzberg, MD, Duke University

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Crossover Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Conditions Cerebral Palsy
CP
Spastic Cerebral Palsy
Interventions Biological: Autologous UCB Reinfusion
Other: Placebo
Enrollment 63
Recruitment Details  
Pre-assignment Details Participants were enrolled and randomized on the same day.
Arm/Group Title Autologous UCB Reinfusion First, Then Placebo Placebo First, Then Autologous UCB Reinfusion
Hide Arm/Group Description Subjects receive autologous umbilical cord blood at Baseline, then Placebo at Year 1. Subjects receive Placebo at Baseline, then autologous umbilical cord blood at Year 1.
Period Title: Year 1
Started 32 31
Completed 32 31
Not Completed 0 0
Period Title: Year 2
Started 32 31
Completed 30 28
Not Completed 2 3
Reason Not Completed
Withdrawal by Subject             2             3
Arm/Group Title Autologous UCB First, Then Placebo Placebo First, Then Autologous UCB Reinfusion Total
Hide Arm/Group Description Subjects receive autologous umbilical cord blood at Baseline, then Placebo at Year 1. Subjects receive Placebo at Baseline, then autologous umbilical cord blood at Year 1. Total of all reporting groups
Overall Number of Baseline Participants 32 31 63
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Median (Full Range)
Unit of measure:  Years
Number Analyzed 32 participants 31 participants 63 participants
2.1
(1.1 to 6.2)
2.3
(1.1 to 7.0)
2.3
(1.1 to 7.0)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 32 participants 31 participants 63 participants
Female
12
  37.5%
9
  29.0%
21
  33.3%
Male
20
  62.5%
22
  71.0%
42
  66.7%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 32 participants 31 participants 63 participants
Hispanic or Latino
3
   9.4%
4
  12.9%
7
  11.1%
Not Hispanic or Latino
29
  90.6%
27
  87.1%
56
  88.9%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 32 participants 31 participants 63 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
Asian
4
  12.5%
3
   9.7%
7
  11.1%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
1
   3.1%
0
   0.0%
1
   1.6%
White
27
  84.4%
28
  90.3%
55
  87.3%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
GMFCS Level   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 32 participants 31 participants 63 participants
Level I
11
  34.4%
6
  19.4%
17
  27.0%
Level II
9
  28.1%
15
  48.4%
24
  38.1%
Level III
4
  12.5%
4
  12.9%
8
  12.7%
Level IV
8
  25.0%
6
  19.4%
14
  22.2%
[1]
Measure Description: The Gross Motor Function Classification System (GMFCS) classifies gross motor function of children with cerebral palsy into Levels I (least disabled) to V (most disabled). For example, children at Level I are able to walk at home, in school, and in the community and can climb stairs without a railing. At Level II children may walk with assistance and can climb stairs using a railing. At Level III children walk with handheld assistive devices for short distances but use a wheelchair for long distance mobility. Mobility at Levels IV and V require physical assistance or powered devices.
Type of Cerebral Palsy  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 32 participants 31 participants 63 participants
Spastic Diplegia
6
  18.8%
6
  19.4%
12
  19.0%
Spastic Hemiplegia
15
  46.9%
15
  48.4%
30
  47.6%
Spastic Quadriplegia
10
  31.3%
7
  22.6%
17
  27.0%
Hypotonic Quadriplegia
1
   3.1%
3
   9.7%
4
   6.3%
1.Primary Outcome
Title Change in Gross Motor Function Measure 66 (GMFM-66) Score
Hide Description Change in Gross Motor Function Measure 66 (GMFM-66) Score from Baseline to Year 1. The GMFM-66 is a clinical tool used to evaluate gross motor function in children with cerebral palsy and is scored using a propriety software program called the Gross Motor Ability Estimator (GMAE) that produces an interval level continuous score ranging from 0 to 100. Higher scores indicate better motor function. A negative change in GMFM-66 score indicates a reduction in motor function, a positive change indicates improvement in motor function, and zero indicates no change in motor function.
Time Frame Baseline to Year 1
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
All enrolled patients were analyzed as randomized from Baseline to Year 1.
Arm/Group Title Autologous UCB Reinfusion First, Then Placebo Placebo First, Then Autologous UCB Reinfusion
Hide Arm/Group Description:
Subjects receive their autologous umbilical cord blood cells at Baseline, than placebo at Year 1.
Subjects receive placebo at Baseline, then autologous umbilical cord blood cell reinfusion at Year 1.
Overall Number of Participants Analyzed 32 31
Mean (Standard Deviation)
Unit of Measure: units on a scale
7.5  (6.8) 6.9  (5.6)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Autologous UCB Reinfusion First, Then Placebo, Placebo First, Then Autologous UCB Reinfusion
Comments H0: The true mean 1-year change in GMFM-66 is the same on Autologous Umbilical Cord Blood and Placebo
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.72
Comments [Not Specified]
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Final Values)
Estimated Value 0.6
Confidence Interval (2-Sided) 95%
-2.6 to 3.7
Estimation Comments [Not Specified]
2.Secondary Outcome
Title Change in Peabody Gross Motor Quotient From Baseline to Year 1
Hide Description The Peabody Developmental Motor Scales - Second Edition (PDMS-2) measures gross and fine motor skills in children from birth through five years of age. The Gross Motor Quotient score from the PDMS-2 was used in this study to evaluate gross motor function. The Gross Motor Quotient measures the ability to use large muscle systems for locomotion, maintain a stable posture when not moving, and throw/catch objects. The range of possible scores is 41 to 164. High scores indicate better gross motor function. Lower scores indicate less gross motor function ability. The change in Gross Motor Quotient from Baseline to Year 1 was evaluated in this study. Positive numbers indicate an increase in gross motor ability, negative numbers indicate decreases in gross motor function, and a zero indicates no change.
Time Frame Baseline to Year 1
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Participants outside of the applicable age range for the PDMS-2 at Baseline or Year 1 were excluded from the analysis.
Arm/Group Title Autologous Umbilical Cord Blood Reinfusion Placebo First, Then Autologous UCB Reinfusion
Hide Arm/Group Description:
Subjects receive their autologous umbilical cord blood cells at Baseline, than placebo at Year 1.
Subjects receive placebo at Baseline, then autologous umbilical cord blood cell reinfusion at Year 1.
Overall Number of Participants Analyzed 24 26
Median (Full Range)
Unit of Measure: units on a scale
1
(-10 to 20)
-0.5
(-19 to 15)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Autologous Umbilical Cord Blood Reinfusion, Placebo First, Then Autologous UCB Reinfusion
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.39
Comments [Not Specified]
Method Wilcoxon (Mann-Whitney)
Comments [Not Specified]
3.Secondary Outcome
Title Change in IT-QOL Questionnaire Score
Hide Description The Infant and Toddler Quality of Life Questionnaire (IT-QOL) was utilized for children ages one to three years at study entry.This 97-item questionnaire is completed by the parents and covers 12 concepts related to the physical, mental, and social well being of the child and the impact of their illness on the family. Scores range from 0 (worst health) to 100 (best health). The change from Baseline to Year 1 is summarized here for each of the 12 items on the questionnaire. Negative values indicate a decline in quality of life over time, positive values indicate an improvement, and zero indicates no change.
Time Frame Baseline to Year 1
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
The analysis includes patients whose parents reported scores on each item at both Baseline and Year 1 and whose children met the age criteria for the questionnaire at both time points.
Arm/Group Title Autologous UCB Reinfusion First,Then Placebo Placebo First, Then Autologous UCB Reinfusion
Hide Arm/Group Description:

Subjects receive their autologous umbilical cord blood cells at Baseline, than placebo at Year 1.

Autologous UCB Reinfusion: Autologous umbilical cord blood (UCB) reinfusion

Placebo: Placebo

Subjects receive placebo at Baseline, then autologous umbilical cord blood cell reinfusion at Year 1.

Autologous UCB Reinfusion: Autologous umbilical cord blood (UCB) reinfusion

Placebo: Placebo

Overall Number of Participants Analyzed 24 21
Median (Full Range)
Unit of Measure: units on a scale
Behavior
4.5
(-13 to 20)
3
(-15 to 20)
Bodily Pain/Discomfort
0
(-42 to 42)
8
(-25 to 25)
Family Cohesion
0
(-40 to 40)
0
(-25 to 25)
General Behavior
4
(-35 to 29)
2
(-21 to 28)
General Health Perceptions
0
(-23 to 21)
0
(-19 to 25)
Global Behavior
0
(-40 to 55)
0
(-55 to 30)
Growth and Development
7.5
(-22 to 35)
3
(-28 to 30)
Overall Health
0
(-30 to 30)
0
(-40 to 25)
Parental Impact-Emotional
18
(-14 to 50)
0
(-21 to 53)
Parental Impact-Time
2
(-22 to 43)
0
(-43 to 47)
Physical Abilities
5.50
(-15 to 35)
6
(-33 to 34)
Temperament and Moods
4
(-20 to 25)
2
(-8 to 30)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Autologous UCB Reinfusion First,Then Placebo, Placebo First, Then Autologous UCB Reinfusion
Comments H0: The 12-month Behavior change score is identically distributed in patients assigned Autologous UCB Reinfusion First and Placebo First.
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.473
Comments [Not Specified]
Method Wilcoxon (Mann-Whitney)
Comments [Not Specified]
Show Statistical Analysis 2 Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Autologous UCB Reinfusion First,Then Placebo, Placebo First, Then Autologous UCB Reinfusion
Comments H0: The 12-month Bodily Pain/Discomfort change score is identically distributed in patients assigned Autologous UCB Reinfusion First and Placebo First.
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.377
Comments [Not Specified]
Method Wilcoxon (Mann-Whitney)
Comments [Not Specified]
Show Statistical Analysis 3 Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Autologous UCB Reinfusion First,Then Placebo, Placebo First, Then Autologous UCB Reinfusion
Comments H0: The 12-month Family Cohesion change score is identically distributed in patients assigned Autologous UCB Reinfusion First and Placebo First.
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.844
Comments [Not Specified]
Method Wilcoxon (Mann-Whitney)
Comments [Not Specified]
Show Statistical Analysis 4 Hide Statistical Analysis 4
Statistical Analysis Overview Comparison Group Selection Autologous UCB Reinfusion First,Then Placebo, Placebo First, Then Autologous UCB Reinfusion
Comments H0: The 12-month General Behavior change score is identically distributed in patients assigned Autologous UCB Reinfusion First and Placebo First.
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.322
Comments [Not Specified]
Method Wilcoxon (Mann-Whitney)
Comments [Not Specified]
Show Statistical Analysis 5 Hide Statistical Analysis 5
Statistical Analysis Overview Comparison Group Selection Autologous UCB Reinfusion First,Then Placebo, Placebo First, Then Autologous UCB Reinfusion
Comments H0: The 12-month General Health Perceptions change score is identically distributed in patients assigned Autologous UCB Reinfusion First and Placebo First.
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.927
Comments [Not Specified]
Method Wilcoxon (Mann-Whitney)
Comments [Not Specified]
Show Statistical Analysis 6 Hide Statistical Analysis 6
Statistical Analysis Overview Comparison Group Selection Autologous UCB Reinfusion First,Then Placebo, Placebo First, Then Autologous UCB Reinfusion
Comments H0: The 12-month Global Behavior change score is identically distributed in patients assigned Autologous UCB Reinfusion First and Placebo First.
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.199
Comments [Not Specified]
Method Wilcoxon (Mann-Whitney)
Comments [Not Specified]
Show Statistical Analysis 7 Hide Statistical Analysis 7
Statistical Analysis Overview Comparison Group Selection Autologous UCB Reinfusion First,Then Placebo, Placebo First, Then Autologous UCB Reinfusion
Comments H0: The 12-month Growth and Development change score is identically distributed in patients assigned Autologous UCB Reinfusion First and Placebo First.
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.294
Comments [Not Specified]
Method Wilcoxon (Mann-Whitney)
Comments [Not Specified]
Show Statistical Analysis 8 Hide Statistical Analysis 8
Statistical Analysis Overview Comparison Group Selection Autologous UCB Reinfusion First,Then Placebo, Placebo First, Then Autologous UCB Reinfusion
Comments H0: The 12-month Overall Health change score is identically distributed in patients assigned Autologous UCB Reinfusion First and Placebo First.
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.726
Comments [Not Specified]
Method Wilcoxon (Mann-Whitney)
Comments [Not Specified]
Show Statistical Analysis 9 Hide Statistical Analysis 9
Statistical Analysis Overview Comparison Group Selection Autologous UCB Reinfusion First,Then Placebo, Placebo First, Then Autologous UCB Reinfusion
Comments H0: The 12-month Parental Impact-Emotional change score is identically distributed in patients assigned Autologous UCB Reinfusion First and Placebo First.
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.315
Comments [Not Specified]
Method Wilcoxon (Mann-Whitney)
Comments [Not Specified]
Show Statistical Analysis 10 Hide Statistical Analysis 10
Statistical Analysis Overview Comparison Group Selection Autologous UCB Reinfusion First,Then Placebo, Placebo First, Then Autologous UCB Reinfusion
Comments H0: The 12-month Parental Impact-Time change score is identically distributed in patients assigned Autologous UCB Reinfusion First and Placebo First.
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.396
Comments [Not Specified]
Method Wilcoxon (Mann-Whitney)
Comments [Not Specified]
Show Statistical Analysis 11 Hide Statistical Analysis 11
Statistical Analysis Overview Comparison Group Selection Autologous UCB Reinfusion First,Then Placebo, Placebo First, Then Autologous UCB Reinfusion
Comments H0: The 12-month Physical Abilities change score is identically distributed in patients assigned Autologous UCB Reinfusion First and Placebo First.
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.381
Comments [Not Specified]
Method Wilcoxon (Mann-Whitney)
Comments [Not Specified]
Show Statistical Analysis 12 Hide Statistical Analysis 12
Statistical Analysis Overview Comparison Group Selection Autologous UCB Reinfusion First,Then Placebo, Placebo First, Then Autologous UCB Reinfusion
Comments H0: The 12-month Temperament and Moods change score is identically distributed in patients assigned Autologous UCB Reinfusion First and Placebo First.
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.869
Comments [Not Specified]
Method Wilcoxon (Mann-Whitney)
Comments [Not Specified]
4.Secondary Outcome
Title Change in CP-QOL Score
Hide Description Children age four years or older at study entry were assessed using the disease-specific “CP-QOL Child” assessment tool as completed by a parent. The CP-QOL Child primary-caregiver proxy form is designed for children 4 – 12 with cerebral palsy and contains 66 items which assess physical, emotional, and social well being as well as access to services and acceptance by others. Scores are summarized in seven topic areas. Scores in each area range from 0 (worst health) to 100 (best health). The change score from Baseline to Year 1 is summarized here for each item. Negative scores indicated a decrease in quality of life, positive scores indicate an increase and zero indicates no change.
Time Frame Baseline, Year 1
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
The analysis includes patients whose parents reported scores on each item at both Baseline and Year 1 and whose children met the age criteria for the questionnaire at both time points.
Arm/Group Title Autologous UCB Reinfusion First, Then Placebo Placebo First, Then Autologous UCB Reinfusion
Hide Arm/Group Description:
Subjects receive autologous umbilical cord blood at Baseline, then Placebo at Year 1.
Subjects receive Placebo at Baseline, then autologous umbilical cord blood at Year 1.
Overall Number of Participants Analyzed 6 5
Median (Full Range)
Unit of Measure: units on a scale
Access to services
-13
(-32 to 12)
15
(-13 to 28)
Emotional well being and self esteem
-8.5
(-30 to 4)
10
(-13 to 90)
Family Health
-3.5
(-37 to 3)
0
(-18 to 19)
Functioning
-11.5
(-22 to 6)
3
(-14 to 30)
Pain and impact of disability
7
(-17 to 17)
-5
(-22 to 3)
Participation and physical health
-3
(-20 to 12)
17
(-20 to 35)
Social wellbeing and acceptance
-4
(-11 to 4)
3
(-17 to 12)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Autologous UCB Reinfusion First, Then Placebo, Placebo First, Then Autologous UCB Reinfusion
Comments H0: The 12 month Access to Services change score is identically distributed in patients assigned Autologous Cord Blood Reinfusion First and Placebo First.
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.055
Comments [Not Specified]
Method Wilcoxon (Mann-Whitney)
Comments [Not Specified]
Show Statistical Analysis 2 Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Autologous UCB Reinfusion First, Then Placebo, Placebo First, Then Autologous UCB Reinfusion
Comments H0: The 12-month 2 Emotional Well Being and Self Esteem change score is identically distributed in patients assigned Autologous UCB Reinfusion First and Placebo First.
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.121
Comments [Not Specified]
Method Wilcoxon (Mann-Whitney)
Comments [Not Specified]
Show Statistical Analysis 3 Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Autologous UCB Reinfusion First, Then Placebo, Placebo First, Then Autologous UCB Reinfusion
Comments H0: The 12-month 2 Family Health change score is identically distributed in patients assigned Autologous UCB Reinfusion First and Placebo First.
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.647
Comments [Not Specified]
Method Wilcoxon (Mann-Whitney)
Comments [Not Specified]
Show Statistical Analysis 4 Hide Statistical Analysis 4
Statistical Analysis Overview Comparison Group Selection Autologous UCB Reinfusion First, Then Placebo, Placebo First, Then Autologous UCB Reinfusion
Comments H0: The 12-month Functioning change score is identically distributed in patients assigned Autologous UCB Reinfusion First and Placebo First.
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.170
Comments [Not Specified]
Method Wilcoxon (Mann-Whitney)
Comments [Not Specified]
Show Statistical Analysis 5 Hide Statistical Analysis 5
Statistical Analysis Overview Comparison Group Selection Autologous UCB Reinfusion First, Then Placebo, Placebo First, Then Autologous UCB Reinfusion
Comments H0: The 12-month Pain and Impact of Disability change score is identically distributed in patients assigned Autologous UCB Reinfusion First and Placebo First.
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.410
Comments [Not Specified]
Method Wilcoxon (Mann-Whitney)
Comments [Not Specified]
Show Statistical Analysis 6 Hide Statistical Analysis 6
Statistical Analysis Overview Comparison Group Selection Autologous UCB Reinfusion First, Then Placebo, Placebo First, Then Autologous UCB Reinfusion
Comments H0: The 12-month Participation and Physical Health change score is identically distributed in patients assigned Autologous UCB Reinfusion First and Placebo First.
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.200
Comments [Not Specified]
Method Wilcoxon (Mann-Whitney)
Comments [Not Specified]
Show Statistical Analysis 7 Hide Statistical Analysis 7
Statistical Analysis Overview Comparison Group Selection Autologous UCB Reinfusion First, Then Placebo, Placebo First, Then Autologous UCB Reinfusion
Comments H0: The 12-month Social wellbeing and acceptance change score is identically distributed in patients assigned Autologous UCB Reinfusion First and Placebo First.
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.225
Comments [Not Specified]
Method Wilcoxon (Mann-Whitney)
Comments [Not Specified]
5.Secondary Outcome
Title Change in Whole Brain Connectivity Measured by Diffusion Tensor Magnetic Resonance Imaging (MRI)
Hide Description Change in number of connections in the brain as measured by diffusion tensor magnetic resonance imaging (MRI). Changes in connectivity are normalized to white matter volume of the brain. A positive number indicates an increase in connections, a negative number indicates a decrease, and zero indicates no change. The number of connections is expressed in terms of 10e5. For example, a change of 1 indicates an increase of 1x10e5 or 100,000 connections.
Time Frame Baseline to Year 1
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Patients without substantial morphologic brain abnormalities that prevented accurate anatomical image parcellation.
Arm/Group Title Autologous UCB Reinfusion First, Then Placebo Placebo First, Then Autologous UCB Reinfusion
Hide Arm/Group Description:
Subjects receive their autologous umbilical cord blood cells at Baseline, than placebo at Year 1.
Subjects receive placebo at Baseline, then autologous umbilical cord blood cell reinfusion at Year 1.
Overall Number of Participants Analyzed 23 15
Median (Full Range)
Unit of Measure: Number of connections x10e5
0.3
(-0.5 to 0.6)
0.2
(-0.5 to 0.6)
6.Secondary Outcome
Title Change in Loes Score of Functional MRI From Baseline to Year 1 and From Year 1 to Year 2
Hide Description No data were collected from this procedure because enrolled subjects who were eligible to receive fMRI were unable to comply with the procedure.
Time Frame Baseline, Year 1, Year 2
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
No data was available.
Arm/Group Title Autologous UCB Reinfusion First,Then Placebo Placebo First, Then Autologous UCB Reinfusion
Hide Arm/Group Description:
Subjects receive their autologous umbilical cord blood cells first, than placebo at Year 1.
Subjects receive placebo first, then autologous umbilical cord blood cell reinfusion at Year 1.
Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
7.Secondary Outcome
Title Correlation Between Clinical Response and RNA Expression of Neural, Endothelial and Inflammatory Cytokines Measured by RNA Arrays in Cord Blood Cells Given to These Patients.
Hide Description Various pre-selected neural, angiogenic, and anti-inflammatory markers expressed by UCB cells and clinical response will be evaluated.
Time Frame 2 years
Outcome Measure Data Not Reported
8.Secondary Outcome
Title Change in Bayley Scales of Infant and Toddler Development-III, Cognitive Composite From Baseline to Year 1
Hide Description Cognitive function was assessed in English-speaking study participants using one of three different tools depending on the age of the patient at the time of assessment: The Bayley Scales of Infant and Toddler Development, Third Edition (Bayley-III), the Wechsler Intelligence Scale for Children (WISC-IV), and the Wechsler Preschool and Primary Scale of Intelligence (WPPSI-III). Some patients were assessed with different tools at subsequent visits as they aged during the conduct of the trial. The Bayley-III is designed to assess developmental functioning of infants and toddlers. Scores for the Cognitive Composite range from 1 to 19 and results in the range of 8 to 12 are considered average. The outcome measure reported here is the change in Cognitive Composite between Baseline to Year 1. Positive numbers indicate increases in cognitive functioning, negative numbers indicate a decrease, and zero indicates no change.
Time Frame Baseline to Year 1
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
The Bayley III is an age-specific test and was used for 42 patients at Baseline. A total of 27 of these patients were also scored with the Bayley III at Year 1. The results here represent change from Baseline to Year 1 in the 27 participants assessed with the Bayley III at both time points.
Arm/Group Title Autologous UCB Reinfusion First,Then Placebo Placebo First, Then Autologous UCB Reinfusion
Hide Arm/Group Description:
Subjects receive their autologous umbilical cord blood cells at Baseline, than placebo at Year 1.
Subjects receive placebo at Baseline, then autologous umbilical cord blood cell reinfusion at Year 1.
Overall Number of Participants Analyzed 14 13
Mean (Standard Error)
Unit of Measure: units on a scale
3.2  (3.0) 8.1  (3.3)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Autologous UCB Reinfusion First,Then Placebo, Placebo First, Then Autologous UCB Reinfusion
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.290
Comments [Not Specified]
Method t-test, 2 sided
Comments [Not Specified]
9.Secondary Outcome
Title Change in Wechsler Preschool and Primary Scale of Intelligence (WPPSI) III Full Scale Intelligence Quotient (IQ) for Younger Children (Ages 2 Years & 6 Months to 3 Years & 11 Months) From Baseline to Year 1.
Hide Description Cognitive function was assessed in English-speaking study participants using one of three different tools depending on the age of the patient at the time of assessment: The Bayley Scales of Infant and Toddler Development, Third Edition (Bayley-III), the Wechsler Intelligence Scale for Children (WISC-IV), and the Wechsler Preschool and Primary Scale of Intelligence (WPPSI-III). Some patients were assessed with different tools at subsequent visits as they aged during the conduct of the trial. There are two versions of the WPPSI-III for two different age ranges: 2 years & 6 months to 3 years & 11 months, and 4 years to 7 years & 3 months. The Full Scale IQ is calculated for both age ranges and provides a continuous score with an average of 100 and a standard deviation of 15. Change from Baseline to Year 1 was evaluated, with positive numbers indicating an increase in cognitive ability, negative numbers indicating a decrease in cognitive ability, and zero indicating no change.
Time Frame Baseline to Year 1
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Change scores were evaluable in only 2 subjects who were assessed with the WPPSI-III at both Baseline and Year 1. One subject was randomized to Autologous Cord Blood and the other to Placebo.
Arm/Group Title Autologous UCB Reinfusion First,Then Placebo Placebo First, Then Autologous UCB Reinfusion
Hide Arm/Group Description:
Subjects receive their autologous umbilical cord blood cells at Baseline, than placebo at Year 1.
Subjects receive placebo at Baseline, then autologous umbilical cord blood cell reinfusion at Year 1.
Overall Number of Participants Analyzed 1 1
Measure Type: Number
Unit of Measure: units on a scale
14 -8
10.Secondary Outcome
Title Change in the Wechsler Intelligence Scale for Children (WISC-IV) From Baseline to Year 1.
Hide Description Cognitive function was assessed in English-speaking study participants using one of three different tools depending on the age of the patient at the time of assessment: The Bayley Scales of Infant and Toddler Development, Third Edition (Bayley-III), the Wechsler Intelligence Scale for Children (WISC-IV), and the Wechsler Preschool and Primary Scale of Intelligence (WPPSI-III). Some patients were assessed with different tools at subsequent visits as they aged during the conduct of the trial. The WISC-IV is designed for children 6 years 0 months to 16 years 11 months. This study used the Full Scale IQ, which ranges from 45 to 155 with a mean of 100 and standard deviation of 15. Higher scores indicate stronger cognitive function. Scores between 90 and 110 are considered to be within the range of average IQ.
Time Frame Baseline to Year 1
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Only one patient in this trial was evaluated with the WISC-IV and thus change over time could not be evaluated using this outcome measure. The single value for this patient is reported here.
Arm/Group Title Autologous UCB Reinfusion First,Then Placebo Placebo First, Then Autologous UCB Reinfusion
Hide Arm/Group Description:
Subjects receive their autologous umbilical cord blood cells first, than placebo at Year 1.
Subjects receive placebo first, then autologous umbilical cord blood cell reinfusion at Year 1.
Overall Number of Participants Analyzed 0 1
Measure Type: Number
Unit of Measure: Full Scale IQ Points
103
11.Secondary Outcome
Title Change in Cognitive Z-Score From Baseline to Year 1
Hide Description Because patients in this study were evaluated with different cognitive assessments based on their age at the time of assessment (The Bayley-III Cognitive Composite, the WPSSI-III Full Scale IQ, and the WISC-IV Full Scale IQ Composite), with some patients being assessed using different tools at subsequent visits during the trial, a method for combining the assessments was employed to evaluate change in cognitive function over time in as many patients as possible. A cognitive Z-score was calculated for each participant at Baseline and Year 1 by adjusting each score by the relevant assessments’ population mean and standard deviation. The Z-scores represent the distance from the population mean, as measured by standard deviations. The analysis presented here summarizes the change in Z-score between Baseline and Year 1. A positive number indicates an increase in cognitive function, a negative number indicates a decrease, and zero indicates no change.
Time Frame Baseline to Year 1
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
When using all available cognitive assessments, the change in cognitive Z-score was available for 42 of the 63 participants.
Arm/Group Title Autologous UCB Reinfusion First,Then Placebo Placebo First, Then Autologous UCB Reinfusion
Hide Arm/Group Description:
Subjects receive their autologous umbilical cord blood cells at Baseline, than Placebo at Year 1.
Subjects receive Placebo at Baseline, then autologous umbilical cord blood cell reinfusion at Year 1.
Overall Number of Participants Analyzed 21 21
Mean (Standard Error)
Unit of Measure: Z scores
0.2  (0.2) 0.3  (0.2)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Autologous UCB Reinfusion First,Then Placebo, Placebo First, Then Autologous UCB Reinfusion
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.697
Comments [Not Specified]
Method t-test, 2 sided
Comments [Not Specified]
12.Secondary Outcome
Title Change in Assisting Hand Assessment (AHA) Score From Baseline to Year 1
Hide Description The Assisting Hand Assessment (AHA) measures the use of hemiplegic cerebral palsy patients' involved hand in tasks involving two hands. The test is valid for ages 18 months to 12 years. The score is an interval scale ranging from 22 to 88 with higher numbers indicating more effective use of the affected hand in performance of bimanual tasks. Change in this score was evaluate between Baseline and Year 1. Positive numbers indicate more effective use of the affected hand, negative numbers indicate a reduction in the effective use of the affected hand, and a zero indicates no change.
Time Frame Baseline to Year 1
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Participants who were less than 18 months old, or had diplegia or quadriplegia were excluded. For those with unspecified typography, AHA scores were excluded if scores on either the Modified Ashworth Scale or Barry-Albright Dystonia scale indicated neither or both of the upper extremities were involved.
Arm/Group Title Autologous UCB Reinfusion First,Then Placebo Placebo First, Then Autologous UCB Reinfusion
Hide Arm/Group Description:
Subjects receive their autologous umbilical cord blood cells at Baseline, than placebo at Year 1.
Subjects receive placebo at Baseline, then autologous umbilical cord blood cell reinfusion at Year 1.
Overall Number of Participants Analyzed 9 10
Mean (Standard Error)
Unit of Measure: units on a scale
5.4  (2.1) 5.1  (2.3)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Autologous UCB Reinfusion First,Then Placebo, Placebo First, Then Autologous UCB Reinfusion
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.912
Comments [Not Specified]
Method t-test, 2 sided
Comments [Not Specified]
13.Secondary Outcome
Title Change in Pediatric Evaluation of Disability (PEDI) Self Care Score
Hide Description The Pediatric Evaluation of Disability is used to evaluate functional skills in children aged 6 months to 7 years in three areas: Self Care, Mobility, and Social Function. The score in each area can range from 10-90. Higher scores indicate higher function. The change from Baseline to Year 1 in the Self Care score is presented here. Positive scores indicate increased function, negative scores indicate a decrease, and zero indicates no change.
Time Frame Baseline to Year 1
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Patients were excluded if change scores were not observable due to missing data at Baseline or Year 1.
Arm/Group Title Autologous Umbilical Cord Blood Reinfusion Placebo First, Then Autologous UCB Reinfusion
Hide Arm/Group Description:
Subjects receive their autologous umbilical cord blood cells at Baseline, than placebo at Year 1.
Subjects receive placebo at Baseline, then autologous umbilical cord blood cell reinfusion at Year 1.
Overall Number of Participants Analyzed 31 29
Mean (Standard Error)
Unit of Measure: units on a scale
-3.7  (2) -2.2  (1.4)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Autologous Umbilical Cord Blood Reinfusion, Placebo First, Then Autologous UCB Reinfusion
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.544
Comments [Not Specified]
Method t-test, 2 sided
Comments [Not Specified]
14.Secondary Outcome
Title Change in Pediatric Evaluation of Disability (PEDI) Mobility Score
Hide Description The Pediatric Evaluation of Disability is used to evaluate functional skills in children aged 6 months to 7 years in three areas: Self Care, Mobility, and Social Function. The score in each area can range from 10-90. Higher scores indicate higher function. The change from Baseline to Year 1 in the Mobility score is presented here. Positive scores indicate increased function, negative scores indicate a decrease, and zero indicates no change.
Time Frame Baseline to Year 1
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Patients were excluded if change scores were not observable due to missing data at Baseline or Year 1.
Arm/Group Title Autologous Umbilical Cord Blood Reinfusion Placebo First, Then Autologous UCB Reinfusion
Hide Arm/Group Description:
Subjects receive their autologous umbilical cord blood cells at Baseline, than placebo at Year 1.
Subjects receive placebo at Baseline, then autologous umbilical cord blood cell reinfusion at Year 1.
Overall Number of Participants Analyzed 31 29
Median (Full Range)
Unit of Measure: units on a scale
0
(-21.81 to 19.80)
0
(-18.50 to 13.21)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Autologous Umbilical Cord Blood Reinfusion, Placebo First, Then Autologous UCB Reinfusion
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.76
Comments [Not Specified]
Method Wilcoxon (Mann-Whitney)
Comments [Not Specified]
15.Secondary Outcome
Title Change in Pediatric Evaluation of Disability (PEDI) Social Function Score
Hide Description The Pediatric Evaluation of Disability is used to evaluate functional skills in children aged 6 months to 7 years in three areas: Self Care, Mobility, and Social Function. The score in each area can range from 10-90. Higher scores indicate higher function. The change from Baseline to Year 1 in the Social Function score is presented here. Positive scores indicate increased function, negative scores indicate a decrease, and zero indicates no change.
Time Frame Baseline to Year 1
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Patients were excluded if change scores were not observable due to missing data at Baseline or Year 1.
Arm/Group Title Autologous Umbilical Cord Blood Reinfusion Placebo First, Then Autologous UCB Reinfusion
Hide Arm/Group Description:
Subjects receive their autologous umbilical cord blood cells at Baseline, than placebo at Year 1.
Subjects receive placebo at Baseline, then autologous umbilical cord blood cell reinfusion at Year 1.
Overall Number of Participants Analyzed 31 29
Mean (Standard Error)
Unit of Measure: units on a scale
-0.5  (2.0) -1.3  (1.8)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Autologous Umbilical Cord Blood Reinfusion, Placebo First, Then Autologous UCB Reinfusion
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.76
Comments [Not Specified]
Method t-test, 2 sided
Comments [Not Specified]
16.Secondary Outcome
Title Change in Child Behavior Checklist (CBCL) Z-score Internalizing Problems From Baseline to Year 1
Hide Description Two versions of the CBCL exist for children ages 1.5 to 5 years and ages 6-18 years. The CBCL evaluates internalizing and externalizing behaviors and total problems using 99-item assessments that are scored on an ordinal scale as 0 for “not true of the child,” 1 for “somewhat or sometimes true of the child,” and 2 for “very true or often true of the child” based on behavior in the preceding two months. Scores are expressed on a standard normal distribution with mean 50 and standard deviation 10. Z scores were created for analysis. A Z-score represents the distance from the population mean in terms of the number of standard deviations. The change in Z-score from Baseline to Year 1 was calculated for each patient. A positive number indicates an increase in the behavior, a negative number indicates a decrease in the behavior, and a zero indicates no change.
Time Frame Baseline to Year 1
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Evaluations done outside the age ranges specified for the assessments were excluded, the majority being assessments that were done on patients who were not yet 1.5 years of age.
Arm/Group Title Autologous UCB Reinfusion First,Then Placebo Placebo First, Then Autologous UCB Reinfusion
Hide Arm/Group Description:
Subjects receive their autologous umbilical cord blood cells at Baseline, than placebo at Year 1.
Subjects receive placebo at Baseline, then autologous umbilical cord blood cell reinfusion at Year 1.
Overall Number of Participants Analyzed 23 22
Median (Full Range)
Unit of Measure: units on a scale
-0.6
(-1.8 to 2.5)
0.0
(-2.2 to 1.0)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Autologous UCB Reinfusion First,Then Placebo, Placebo First, Then Autologous UCB Reinfusion
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.176
Comments [Not Specified]
Method Wilcoxon (Mann-Whitney)
Comments [Not Specified]
17.Secondary Outcome
Title Change in Child Behavior Checklist (CBCL) Z-score Externalizing Problems From Baseline to Year 1
Hide Description Two versions of the CBCL exist for children ages 1.5 to 5 years and ages 6-18 years. The CBCL evaluates internalizing and externalizing behaviors and total problems using 99-item assessments that are scored on an ordinal scale as 0 for “not true of the child,” 1 for “somewhat or sometimes true of the child,” and 2 for “very true or often true of the child” based on behavior in the preceding two months. Scores are expressed on a standard normal distribution with mean 50 and standard deviation 10. Z scores were created for analysis. A Z-score represents the distance from the population mean in terms of the number of standard deviations. The change in Z-score from Baseline to Year 1 was calculated for each patient. A positive number indicates an increase in the behavior, a negative number indicates a decrease in the behavior, and a zero indicates no change.
Time Frame Baseline to Year 1
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Evaluations done outside the age ranges specified for the assessments were excluded, the majority being assessments that were done on patients who were not yet 1.5 years of age.
Arm/Group Title Autologous UCB Reinfusion First,Then Placebo Placebo First, Then Autologous UCB Reinfusion
Hide Arm/Group Description:
Subjects receive their autologous umbilical cord blood cells at Baseline, than placebo at Year 1.
Subjects receive placebo at Baseline, then autologous umbilical cord blood cell reinfusion at Year 1.
Overall Number of Participants Analyzed 23 22
Mean (Standard Error)
Unit of Measure: units on a scale
-0.2  (0.2) 0.1  (0.1)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Autologous UCB Reinfusion First,Then Placebo, Placebo First, Then Autologous UCB Reinfusion
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.099
Comments [Not Specified]
Method t-test, 2 sided
Comments [Not Specified]
18.Secondary Outcome
Title Change in Child Behavior Checklist (CBCL) Z-score Total Problems From Baseline to Year 1
Hide Description Two versions of the CBCL exist for children ages 1.5 to 5 years and ages 6-18 years. The CBCL evaluates internalizing and externalizing behaviors and total problems using 99-item assessments that are scored on an ordinal scale as 0 for “not true of the child,” 1 for “somewhat or sometimes true of the child,” and 2 for “very true or often true of the child” based on behavior in the preceding two months. Scores are expressed on a standard normal distribution with mean 50 and standard deviation 10. Z scores were created for analysis. A Z-score represents the distance from the population mean in terms of the number of standard deviations. The change in Z-score from Baseline to Year 1 was calculated for each patient. A positive number indicates an increase in the behavior, a negative number indicates a decrease in the behavior, and a zero indicates no change.
Time Frame Baseline to Year 1
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Evaluations done outside the age ranges specified for the assessments were excluded, the majority being assessments that were done on patients who were not yet 1.5 years of age.
Arm/Group Title Autologous UCB Reinfusion First,Then Placebo Placebo First, Then Autologous UCB Reinfusion
Hide Arm/Group Description:
Subjects receive their autologous umbilical cord blood cells at Baseline, than placebo at Year 1.
Subjects receive placebo at Baseline, then autologous umbilical cord blood cell reinfusion at Year 1.
Overall Number of Participants Analyzed 23 22
Median (Full Range)
Unit of Measure: units on a scale
-0.3
(-2.0 to 2.8)
0.1
(-1.9 to 1.2)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Autologous UCB Reinfusion First,Then Placebo, Placebo First, Then Autologous UCB Reinfusion
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.131
Comments [Not Specified]
Method Wilcoxon (Mann-Whitney)
Comments [Not Specified]
19.Secondary Outcome
Title Change in Parental Distress From Baseline to Year 1
Hide Description

Parent stress was evaluated with the Parenting Stress Index – Short Form for children aged 0-12 years, which measures stress in three domains: Parental Distress, Parent-Child Dysfunctional Interaction, and Difficult Child. Results in each domain are expressed as percentiles. Scores from the 15th-80th percentile are considered to be within the normal range.

Scores at or above the 85th percentile considered high distress. Scores greater than the 89th percentile indicate clinically significant levels of distress. Analysis focused on changes in percentile scores between Baseline and Year 1. Positive numbers represent an increase in distress, negative numbers represent a decrease in distress, and zero indicates no change.

Time Frame Baseline to Year 1
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
No participants were outside the age range for this test but change scores could not be calculated for some participants due either to missing data at the Baseline or Year 1 visit.
Arm/Group Title Autologous UCB Reinfusion First,Then Placebo Placebo First, Then Autologous UCB Reinfusion
Hide Arm/Group Description:
Subjects receive their autologous umbilical cord blood cells at Baseline, than placebo at Year 1.
Subjects receive placebo at Baseline, then autologous umbilical cord blood cell reinfusion at Year 1.
Overall Number of Participants Analyzed 28 29
Mean (Standard Error)
Unit of Measure: Change in percentile score
1.0  (4.5) -3.6  (4.6)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Autologous UCB Reinfusion First,Then Placebo, Placebo First, Then Autologous UCB Reinfusion
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.478
Comments [Not Specified]
Method t-test, 2 sided
Comments [Not Specified]
20.Secondary Outcome
Title Change in Parent-Child Dysfunctional Interaction From Baseline to Year 1
Hide Description Parent stress was evaluated with the Parenting Stress Index - Short Form for children aged 0-12 years, which measures stress in three domains: Parental Distress, Parent-Child Dysfunctional Interaction, and Difficult Child. Results in each domain are expressed as percentiles. Scores from the 15th-80th percentile are considered to be within the normal range. Scores at or above the 85th percentile considered high distress. Scores greater than the 89th percentile indicate clinically significant levels of distress. Analysis focused on changes in percentile scores between Baseline and Year 1. Positive numbers represent an increase in distress, negative numbers represent a decrease in distress, and zero indicates no change.
Time Frame Baseline to Year 1
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
No participants were outside the age range for this test but change scores could not be calculated for some participants due either to missing data at the Baseline or Year 1 visit.
Arm/Group Title Autologous UCB Reinfusion First,Then Placebo Placebo First, Then Autologous UCB Reinfusion
Hide Arm/Group Description:
Subjects receive their autologous umbilical cord blood cells at Baseline, than placebo at Year 1.
Subjects receive placebo at Baseline, then autologous umbilical cord blood cell reinfusion at Year 1.
Overall Number of Participants Analyzed 28 29
Median (Full Range)
Unit of Measure: units on a scale
0.0
(-35.0 to 45.0)
-10.0
(-56.0 to 41.0)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Autologous UCB Reinfusion First,Then Placebo, Placebo First, Then Autologous UCB Reinfusion
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.029
Comments [Not Specified]
Method Wilcoxon (Mann-Whitney)
Comments [Not Specified]
21.Secondary Outcome
Title Change in Difficult Child Score From Baseline to Year 1
Hide Description Parent stress was evaluated with the Parenting Stress Index - Short Form for children aged 0-12 years, which measures stress in three domains: Parental Distress, Parent-Child Dysfunctional Interaction, and Difficult Child. Results in each domain are expressed as percentiles. Scores from the 15th-80th percentile are considered to be within the normal range. Scores at or above the 85th percentile considered high distress. Scores greater than the 89th percentile indicate clinically significant levels of distress. Analysis focused on changes in percentile scores between Baseline and Year 1. Positive numbers represent an increase in distress, negative numbers represent a decrease in distress, and zero indicates no change.
Time Frame Baseline to Year 1
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
No participants were outside the age range for this test but change scores could not be calculated for some participants due either to missing data at the Baseline or Year 1 visit.
Arm/Group Title Autologous UCB Reinfusion First,Then Placebo Placebo First, Then Autologous UCB Reinfusion
Hide Arm/Group Description:
Subjects receive their autologous umbilical cord blood cells at Baseline, than placebo at Year 1.
Subjects receive placebo at Baseline, then autologous umbilical cord blood cell reinfusion at Year 1.
Overall Number of Participants Analyzed 28 29
Mean (Standard Error)
Unit of Measure: units on a scale
-0.2  (4.8) 6.9  (5.0)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Autologous UCB Reinfusion First,Then Placebo, Placebo First, Then Autologous UCB Reinfusion
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.310
Comments [Not Specified]
Method t-test, 2 sided
Comments [Not Specified]
22.Secondary Outcome
Title Modified Ashworth Scale at Baseline
Hide Description The Modified Ashworth Scale uses a 6 point scale (range 0, 1, 1+, 2, 3, or 4) to measure spasticity in 5 body regions (central, right upper extremity, left upper extremity, right lower extremity, and left lower extremity). Scores of 0 indicate no increase in muscle tone whereas a score of 4 indicates rigidity in flexion or extension.
Time Frame Baseline
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Patients are classified by their maximum score, regardless of body region.
Arm/Group Title Autologous UCB Reinfusion First, Then Placebo Placebo First, Then Autologous UCB Reinfusion
Hide Arm/Group Description:
Subjects receive their autologous umbilical cord blood cells at Baseline, than placebo at Year 1.
Subjects receive placebo at Baseline, then autologous umbilical cord blood cell reinfusion at Year 1.
Overall Number of Participants Analyzed 30 30
Measure Type: Count of Participants
Unit of Measure: Participants
0 @ Baseline
2
   6.7%
3
  10.0%
1 @ Baseline
4
  13.3%
3
  10.0%
1+ @ Baseline
5
  16.7%
3
  10.0%
2 @ Baseline
11
  36.7%
11
  36.7%
3 @ Baseline
8
  26.7%
8
  26.7%
5 @ Baseline
0
   0.0%
2
   6.7%
23.Secondary Outcome
Title Modified Ashworth Scale at Year 1
Hide Description The Modified Ashworth Scale uses a 6 point scale (range 0, 1, 1+, 2, 3, or 4) to measure spasticity in 5 body regions (central, right upper extremity, left upper extremity, right lower extremity, and left lower extremity). Scores of 0 indicate no increase in muscle tone whereas a score of 4 indicates rigidity in flexion or extension.
Time Frame Year 1
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Patients are classified by their maximum score, regardless of body region.
Arm/Group Title Autologous UCB Reinfusion First, Then Placebo Placebo First, Then Autologous UCB Reinfusion
Hide Arm/Group Description:
Subjects receive their autologous umbilical cord blood cells at Baseline, than placebo at Year 1.
Subjects receive placebo at Baseline, then autologous umbilical cord blood cell reinfusion at Year 1.
Overall Number of Participants Analyzed 31 30
Measure Type: Count of Participants
Unit of Measure: Participants
0 @ Year 1
1
   3.2%
1
   3.3%
1 @ Year 1
3
   9.7%
5
  16.7%
1+ @ Year 1
6
  19.4%
8
  26.7%
2 @ Year 1
10
  32.3%
9
  30.0%
3 @ Year 1
10
  32.3%
6
  20.0%
4 @ Year 1
1
   3.2%
1
   3.3%
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Autologous UCB Reinfusion First, Then Placebo, Placebo First, Then Autologous UCB Reinfusion
Comments H0: The population distribution of Modified Ashworth Scale scores is the same at 1-year post-infusion with autologous cord blood and placebo.
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.233
Comments [Not Specified]
Method Wilcoxon (Mann-Whitney)
Comments [Not Specified]
24.Secondary Outcome
Title Change in Bruininks-Oseretsky-2 Total Motor Composite From Baseline to Year 1
Hide Description The Bruininks-Oseretsky Test of Motor Proficiency, Second Edition, (BOT-2) evaluates motor function in four areas: stability, mobility, strength, coordination, and object manipulation. A Total Motor Composite is then calculated and expressed on a normal distribution with mean 50 and standard deviation of 10. Higher scores indicate better motor function. The BOT-2 Total Motor Composite was used to measure motor function in children at age 6 in this study. The study intended to evaluate change in the BOT-2 Total Motor Composite from Baseline to Year 1, where positive change indicates improvement in motor function, negative change indicates decrease in motor function, and zero indicates no change.
Time Frame Baseline to Year 1
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
The BOT-2 Total Motor Composite was available at Baseline and Year 1 in only 1 subject. The change score for that subject is reported here.
Arm/Group Title Autologous Umbilical Cord Blood Reinfusion Placebo First, Then Autologous UCB Reinfusion
Hide Arm/Group Description:
Subjects receive their autologous umbilical cord blood cells at Baseline, than placebo at Year 1.
Subjects receive placebo at Baseline, then autologous umbilical cord blood cell reinfusion at Year 1.
Overall Number of Participants Analyzed 1 0
Measure Type: Number
Unit of Measure: units on a scale
-1
25.Secondary Outcome
Title Parent Experience of Child Illness
Hide Description This outcome measure was not used in this study because it had not been validated for children with Cerebral Palsy.
Time Frame Baseline to Year 1
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Autologous Umbilical Cord Blood Reinfusion Placebo First, Then Autologous UCB Reinfusion
Hide Arm/Group Description:
Subjects receive their autologous umbilical cord blood cells at Baseline, than placebo at Year 1.
Subjects receive placebo at Baseline, then autologous umbilical cord blood cell reinfusion at Year 1.
Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
26.Other Pre-specified Outcome
Title Change in Barry-Albright Dystonia Total Score From Baseline to Year 1
Hide Description The Barry-Albright Dystonia Scale measures generalized dystonia in eight body regions (eyes, mouth, neck, trunk, and the four extremities) using an ordinal scale (0=no dystonia, 1=slight dystonia, 2=mild dystonia, 3=moderate dystonia, and 4=severe dystonia). Individual scores for each region are summed to obtain a total score. The total score can range from 0 to 32 and higher scores indicate an overall greater degree of dystonia. The change in Barry-Albright Dystonia Total Score was evaluated from Baseline to Year 1. Positive numbers indicate increasing dystonia, negative numbers indicate a decrease in dystonia, and zero indicates no change.
Time Frame Baseline to Year 1
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Autologous UCB Reinfusion First,Then Placebo Placebo First, Then Autologous UCB Reinfusion
Hide Arm/Group Description:
Subjects receive their autologous umbilical cord blood cells at Baseline, than placebo at Year 1.
Subjects receive placebo at Baseline, then autologous umbilical cord blood cell reinfusion at Year 1.
Overall Number of Participants Analyzed 32 31
Median (Full Range)
Unit of Measure: units on a scale
0.0
(-12.0 to 10.0)
0.0
(-11.0 to 28.0)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Autologous UCB Reinfusion First,Then Placebo, Placebo First, Then Autologous UCB Reinfusion
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.762
Comments [Not Specified]
Method Wilcoxon (Mann-Whitney)
Comments [Not Specified]
27.Post-Hoc Outcome
Title Gross Motor Function Measure 66 (GMFM-66) Change Score at 1 Year by Infused Dose
Hide Description Gross Motor Function Measure 66 (GMFM-66) Change Score at 1 Year. Analyzed by infused dose, above or below the median. The GMFM-66 is a clinical tool used to evaluate gross motor function in children with cerebral palsy and is scored using a propriety software program called the Gross Motor Ability Estimator (GMAE) that produces an interval level continuous score ranging from 0 to 100. Higher scores indicate better motor function. A negative change in GMFM-66 score indicates a reduction in motor function, a positive change indicates improvement in motor function, and zero indicates no change in motor function.
Time Frame 1 Year
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Patients randomized to Autologous Umbilical Cord Blood were divided into two groups for this analysis: Low and High dose. The median dose infused in these 32 patients was used as the cut point to define Low and High doses: 1.98x10e7 total nucleated cells per kilogram of patient weight (TNCC/kg).
Arm/Group Title Autologous UCB Reinfusion First, Then Placebo Placebo First, Then Autologous UCB Reinfusion
Hide Arm/Group Description:
Subjects receive their autologous umbilical cord blood cells at Baseline, than placebo at Year 1.
Subjects receive placebo at Baseline, then autologous umbilical cord blood cell reinfusion at Year 1.
Overall Number of Participants Analyzed 32 31
Median (Full Range)
Unit of Measure: units on a scale
All Number Analyzed 32 participants 31 participants
6.5
(-3 to 19)
6.0
(-5 to 19)
Low Infused Dose Number Analyzed 16 participants 0 participants
4
(-3 to 19)
High Infused Dose Number Analyzed 16 participants 0 participants
8.5
(0 to 19)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Autologous UCB Reinfusion First, Then Placebo
Comments H0: The population distribution of the 1-year GMFM-66 change score is the same for patients infused with Low and High doses of autologous umbilical cord blood.
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.05
Comments [Not Specified]
Method Wilcoxon (Mann-Whitney)
Comments [Not Specified]
Show Statistical Analysis 2 Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Autologous UCB Reinfusion First, Then Placebo, Placebo First, Then Autologous UCB Reinfusion
Comments H0: The population distribution of the 1-year GMFM-66 change score is the same for patients infused with High doses of autologous umbilical cord blood and Placebo.
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.15
Comments [Not Specified]
Method Wilcoxon (Mann-Whitney)
Comments [Not Specified]
Show Statistical Analysis 3 Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Autologous UCB Reinfusion First, Then Placebo, Placebo First, Then Autologous UCB Reinfusion
Comments H0: The population distribution of the 1-year GMFM-66 change score is the same for patients infused with Low doses of autologous umbilical cord blood and placebo.
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.21
Comments [Not Specified]
Method Wilcoxon (Mann-Whitney)
Comments [Not Specified]
28.Post-Hoc Outcome
Title Observed Minus Expected GMFM-66 Change Score of All Subjects Receiving Autologous UCB and Who Are Greater Than or Equal to 2 Years of Age
Hide Description Validated functional curves were used to identify the expected 1-year change in GMFM-66 score given each child's age and Gross Motor Function Classification System (GMFCS) Level at baseline. The difference between observed change (in this study) and expected change was then calculated for each participant. The threshold defining Low/High dose is the median dose infused in all 63 enrolled patients, 2x10e7 TNCC/kg.
Time Frame 1 year post-infusion with autologous umbilical cord blood
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Hide Analysis Population Description
Patients who received cord blood at baseline and 1 year post-infusion with Placebo, and who are >=2 years old (to allow calculation of expected GMFM-66 change scores from published data). An additional 6 are excluded because change scores were not observable due to subject withdrawal (5) or inability to comply due to a broken leg (1).
Arm/Group Title Autologous Umbilical Cord Blood Reinfusion
Hide Arm/Group Description:
Patients will be randomized to receive their autologous umbilical cord blood cells first or placebo first. Subjects will receive both infusions but will be randomized and blinded by which they are receiving first and second.
Overall Number of Participants Analyzed 46
Median (Full Range)
Unit of Measure: units on a scale
All Number Analyzed 46 participants
1.1
(-8.1 to 15.4)
Low Infused Dose Number Analyzed 23 participants
-1.1
(-8.1 to 12.9)
High Infused Dose Number Analyzed 23 participants
3.6
(-5.6 to 15.4)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Autologous Umbilical Cord Blood Reinfusion
Comments H0: The population distribution of differences between observed and expected GMFM-66 change scores at 1-year post-infusion is the same for patients receiving Low and High doses of autologous umbilical cord blood.
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.01
Comments [Not Specified]
Method Wilcoxon (Mann-Whitney)
Comments [Not Specified]
29.Post-Hoc Outcome
Title Peabody Gross Motor Quotient Change Score 1 Year After Receiving Autologous UCB.
Hide Description The Peabody Developmental Motor Scales - Second Edition (PDMS-2), Gross Motor Quotient change score was calculated for each patient at 1 year after infusion with autologous umbilical cord blood. Analyzed by infused dose, above or below the median. The Gross Motor Quotient score from the PDMS-2 was used in this study to evaluate gross motor function. The Gross Motor Quotient measures the ability to use large muscle systems for locomotion, maintain a stable posture when not moving, and throw/catch objects. The range of possible scores is 41 to 164. High scores indicate better gross motor function. Lower scores indicate less gross motor function ability.
Time Frame 1 year post-infusion with autologous umbilical cord blood
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Hide Analysis Population Description
This group combines patients who received Autologous Umbilical Cord Blood at baseline or 1 year post-infusion with Placebo, and who are within the age range specified for the PDMS-2. The threshold defining Low/High dose is the median dose infused in all 63 enrolled patients, 2x10e7 TNCC/kg.
Arm/Group Title Autologous Umbilical Cord Blood Reinfusion
Hide Arm/Group Description:
Patients will be randomized to receive their autologous umbilical cord blood cells first or placebo first. Subjects will receive both infusions but will be randomized and blinded by which they are receiving first and second.
Overall Number of Participants Analyzed 47
Median (Full Range)
Unit of Measure: units on a scale
Low Infused Dose Number Analyzed 25 participants
0
(-10 to 15)
High Infused Dose Number Analyzed 22 participants
3
(-8 to 20)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Autologous Umbilical Cord Blood Reinfusion
Comments [Not Specified]
Type of Statistical Test Superiority
Comments H0: The population distribution of the Peabody Gross Motor Quotient change score 1 year post-infusion with autologous umbilical cord blood is the same in patients receiving a Low infused dose and a High infused dose.
Statistical Test of Hypothesis P-Value 0.03
Comments [Not Specified]
Method Wilcoxon (Mann-Whitney)
Comments [Not Specified]
30.Post-Hoc Outcome
Title Change in Whole Brain Connectivity Measured by Diffusion Tensor Magnetic Resonance Imaging (MRI) of All Subjects 1 Year Post-infusion With Autologous UCB
Hide Description Change in whole brain connectivity measured by diffusion tensor magnetic resonance imaging (MRI). Changes in connectivity are normalized to white matter volume.
Time Frame 1 year post-infusion with autologous umbilical cord blood
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Hide Analysis Population Description
This group combines patients who received Autologous Umbilical Cord Blood whether at baseline or 1 year post-infusion with Placebo and is limited to patients without morphologic brain abnormalities that prevented accurate anatomical image parcellation. Low/High dose is defined as the median dose infused in all 63 enrolled patients, 2x10e7 TNCC/kg.
Arm/Group Title Autologous Umbilical Cord Blood Reinfusion
Hide Arm/Group Description:

All participants will be treated with autologous cord blood reinfusion, but the time course will vary between groups and participants will be blinded to the order in which they receive infusions.

Autologous Umbilical Cord Blood or Placebo: All participants will be treated with autologous cord blood reinfusion, but the time course will vary between groups and participants will be blinded to the order in which they receive infusions. Patients will be randomized to receive their autologous umbilical cord blood cells first or placebo first. Subjects will receive both infusions but will be randomized and blinded by which they are receiving first and second.

Overall Number of Participants Analyzed 38
Mean (Standard Deviation)
Unit of Measure: Number of connections x10e5
Low Infused Dose Number Analyzed 19 participants
0.10  (0.32)
High Infused Dose Number Analyzed 19 participants
0.30  (0.24)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Autologous Umbilical Cord Blood Reinfusion
Comments H0: The true mean 1-year change in normalized whole brain connectivity is the same in patients infused with Low and High doses of autologous umbilical cord blood.
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.04
Comments [Not Specified]
Method Wilcoxon (Mann-Whitney)
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Final Values)
Estimated Value 0.20
Confidence Interval (2-Sided) 95%
0.01 to 0.38
Estimation Comments [Not Specified]
Time Frame 2 years
Adverse Event Reporting Description Since there is no washout period for autologous umbilical cord blood transfusions, the only adverse events included in the placebo arm were those events that occurred in subjects that had not yet received cord blood. All other adverse events that occurred in subjects that had been exposed to cord blood at either Baseline or Year 1 were included in the "Autologous Umbilical Cord Blood" arm.
 
Arm/Group Title Autologous Umbilical Cord Blood Reinfusion Placebo
Hide Arm/Group Description

All participants will be treated with autologous cord blood reinfusion, but the time course will vary between groups and participants will be blinded to the order in which they receive infusions.

Autologous Umbilical Cord Blood or Placebo: All participants will be treated with autologous cord blood reinfusion, but the time course will vary between groups and participants will be blinded to the order in which they receive infusions. Patients will be randomized to receive their autologous umbilical cord blood cells first or placebo first. Subjects will receive both infusions but will be randomized and blinded by which they are receiving first and second.

All participants will be treated with autologous cord blood reinfusion, but the time course will vary between groups and participants will be blinded to the order in which they receive infusions.

Autologous Umbilical Cord Blood or Placebo: All participants will be treated with autologous cord blood reinfusion, but the time course will vary between groups and participants will be blinded to the order in which they receive infusions. Patients will be randomized to receive their autologous umbilical cord blood cells first or placebo first. Subjects will receive both infusions but will be randomized and blinded by which they are receiving first and second.

All-Cause Mortality
Autologous Umbilical Cord Blood Reinfusion Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   0/63 (0.00%)      0/63 (0.00%)    
Show Serious Adverse Events Hide Serious Adverse Events
Autologous Umbilical Cord Blood Reinfusion Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   0/63 (0.00%)      1/63 (1.59%)    
Endocrine disorders     
Flushed in face with hypersensitivity rash on trunk * [1]  0/63 (0.00%)  0 1/63 (1.59%)  1
*
Indicates events were collected by non-systematic assessment
[1]
Subject developed polydipsia, polyuria, and hyperglycemia the evening after infusion and was seen in the ED. Subject was diagnosed with type I diabetes (mother also has type I diabetes). This was reported to Medwatch.
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Autologous Umbilical Cord Blood Reinfusion Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   24/63 (38.10%)      4/63 (6.35%)    
Congenital, familial and genetic disorders     
Diagnosed with Cockayne syndrome *  1/63 (1.59%)  0/63 (0.00%)  0
Gastrointestinal disorders     
Stomach pain from hydronephrotic dysplastic kidney *  1/63 (1.59%)  0/63 (0.00%)  0
G tube fell out *  1/63 (1.59%)  0/63 (0.00%)  0
G tube placement *  0/63 (0.00%)  0 1/63 (1.59%) 
General disorders     
Fever of 102 3 days post. Negative culture. No treatment-resolved in 3 days. *  1/63 (1.59%)  0/63 (0.00%)  0
Adverse behavioral rxn to Benadryl given infusion day- crying *  1/63 (1.59%)  0/63 (0.00%)  0
Fever for 2 days prior to infusion-given Tylenol *  1/63 (1.59%)  0/63 (0.00%)  0
Immune system disorders     
Flushed in face with hypersensitivity rash on trunk. Sats 92%. No wheezing. Benadryl given *  0/63 (0.00%)  0 1/63 (1.59%) 
Agitation and mild conjunctivitis of right eye following infusion. * [1]  1/63 (1.59%)  0/63 (0.00%)  0
Infections and infestations     
Tactile temp overnight. Left otitis media *  0/63 (0.00%)  0 1/63 (1.59%) 
Pneumonia-hospitalized 10 days *  1/63 (1.59%)  0/63 (0.00%)  0
Flu *  1/63 (1.59%)  0/63 (0.00%)  0
Withdrawn during strep throat and sinus infection- EEG showed spikes , but no epileptiform activity *  1/63 (1.59%)  0/63 (0.00%)  0
Fever 2 weeks post infusion-found to be viral laryngitis *  1/63 (1.59%)  0/63 (0.00%)  0
Admitted to hospital- Flu *  1/63 (1.59%)  0/63 (0.00%)  0
Cough/Cold requiring antibiotics *  1/63 (1.59%)  0/63 (0.00%)  0
Pneumonia requiring antibiotics *  1/63 (1.59%)  0/63 (0.00%)  0
Ear infection, cough, and fever 2 wks post infusion *  1/63 (1.59%)  0/63 (0.00%)  0
Ear infection *  1/63 (1.59%)  0/63 (0.00%)  0
Diagnosed with walking pneumonia prior to final visit- on antibiotics *  0/63 (0.00%)  0 1/63 (1.59%) 
Metabolism and nutrition disorders     
Admitted to ER with polyuria and polydipsia * [2]  1/63 (1.59%)  0/63 (0.00%)  0
Musculoskeletal and connective tissue disorders     
Fractured left foot *  1/63 (1.59%)  0/63 (0.00%)  0
Right foot fracture- cast required *  1/63 (1.59%)  0/63 (0.00%)  0
Nervous system disorders     
Febrile seizure-isolated, not treated *  1/63 (1.59%)  0/63 (0.00%)  0
High fever and febrile seizure associated with viral infection at home 2 weeks post infusion *  1/63 (1.59%)  0/63 (0.00%)  0
Febrile Seizure *  1/63 (1.59%)  0/63 (0.00%)  0
2 seizures in interval- due to virus- on anti-seizure med *  1/63 (1.59%)  0/63 (0.00%)  0
Renal and urinary disorders     
Painful urination and spotting 1 wk post infusion. * [3]  1/63 (1.59%)  0/63 (0.00%)  0
Respiratory, thoracic and mediastinal disorders     
Developed cough and sats fell to 70 toward end of MRI- received blow by O2 *  1/63 (1.59%)  0/63 (0.00%)  0
Briefly hospitalized for bronchiolitis and dehydration- treated with rocephin and rehydration *  1/63 (1.59%)  0/63 (0.00%)  0
Inhaler needed for respiratory wheezing *  1/63 (1.59%)  0/63 (0.00%)  0
Skin and subcutaneous tissue disorders     
Rash on abdomen and legs. No itching or welps. No treatment- resolved next day *  0/63 (0.00%)  0 1/63 (1.59%) 
*
Indicates events were collected by non-systematic assessment
[1]
Returned to baseline after being given Benadryl.
[2]
Highest blood sugar 757. Received fluid and insulin- Diabetes Dx
[3]
Seen by urgent care-abx started, stopped when cultures neg.
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Name/Title: Jesse Troy, PhD
Organization: Duke University School of Medicine, Division of Pediatric Blood and Marrow Transplantation
Responsible Party: Joanne Kurtzberg, MD, Duke University
ClinicalTrials.gov Identifier: NCT01147653     History of Changes
Other Study ID Numbers: Pro00017801
First Submitted: June 17, 2010
First Posted: June 22, 2010
Results First Submitted: March 15, 2017
Results First Posted: June 12, 2017
Last Update Posted: November 30, 2017