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Cord Blood Infusion for Children With Autism Spectrum Disorder (Duke ACT)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02847182
Recruitment Status : Completed
First Posted : July 28, 2016
Results First Posted : December 26, 2019
Last Update Posted : June 9, 2020
Sponsor:
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 Autism Spectrum Disorder
ASD
Autism
PDD
Interventions Biological: Cord Blood Infusion
Biological: Placebo
Enrollment 180
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Cord Blood Infusion, Then Placebo Infusion Placebo Infusion, Then Cord Blood Infusion
Hide Arm/Group Description

Subjects will be randomized to receive a cord blood infusion at the baseline visit, followed by a placebo infusion at 6 months. The cord blood will be autologous (if available) or unrelated cord blood. The placebo is an acellular media product similar in both appearance and odor.

Cord Blood Infusion

Placebo

Subjects will be randomized to receive a placebo infusion at the baseline visit, followed by a cord blood infusion at 6 months. The placebo is an acellular media product similar in both appearance and odor. The cord blood will be autologous (if available) or unrelated cord blood.

Cord Blood Infusion

Placebo

Period Title: Overall Study
Started 119 61
First Intervention (6 Months) 119 61
Second Intervention (6 Months) 61 119
Completed 119 60
Not Completed 0 1
Reason Not Completed
Withdrawal by Subject             0             1
Arm/Group Title Cord Blood Infusion, Then Placebo Infusion Placebo Infusion, Then Cord Blood Infusion Total
Hide Arm/Group Description

Subjects will be randomized to receive a cord blood infusion at the baseline visit, followed by a placebo infusion at 6 months. The cord blood will be autologous (if available) or unrelated cord blood. The placebo is an acellular media product similar in both appearance and odor.

Cord Blood Infusion

Placebo

Subjects will be randomized to receive a placebo infusion at the baseline visit, followed by a cord blood infusion at 6 months. The placebo is an acellular media product similar in both appearance and odor. The cord blood will be autologous (if available) or unrelated cord blood.

Cord Blood Infusion

Placebo

Total of all reporting groups
Overall Number of Baseline Participants 119 61 180
Hide Baseline Analysis Population Description
The protocol-specified pilot subjects, both randomized to placebo, were also mistakenly enrolled several days after their 8th birthday, in violation of the age-related inclusion criteria.
Age, Continuous  
Median (Full Range)
Unit of measure:  Years
Number Analyzed 119 participants 61 participants 180 participants
5.30
(2.39 to 8.00)
5.24
(2.31 to 8.13)
5.29
(2.31 to 8.13)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 119 participants 61 participants 180 participants
Female
21
  17.6%
16
  26.2%
37
  20.6%
Male
98
  82.4%
45
  73.8%
143
  79.4%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 119 participants 61 participants 180 participants
Hispanic or Latino
26
  21.8%
6
   9.8%
32
  17.8%
Not Hispanic or Latino
93
  78.2%
55
  90.2%
148
  82.2%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
Race/Ethnicity, Customized  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 119 participants 61 participants 180 participants
Non-White
24
  20.2%
17
  27.9%
41
  22.8%
White
95
  79.8%
44
  72.1%
139
  77.2%
Region of Enrollment  
Measure Type: Count of Participants
Unit of measure:  Participants
United States Number Analyzed 119 participants 61 participants 180 participants
119
 100.0%
61
 100.0%
180
 100.0%
1.Primary Outcome
Title Change in Social Communication as Measured by the Vineland Adaptive Behavior Scales, Third Edition (VABS-3)
Hide Description The Vineland Adaptive Behavior Scales, Third Edition (VABS-3) Socialization domain standard score has mean=100 and standard deviation=15 (range: 20-140). Higher scores indicate better developed adaptive social behavior. The change in the Socialization domain standard score was calculated for each participant from Baseline to Month 6. Changes in the Socialization standard score are indicative of skill acquisition relative to chronologically aged peers of the same sex. Thus, a zero (no change) represents change consistent with what is expected. An increase represents acquisition of more skills over time than would be expected. Participants who experience a decrease in Socialization standard score may still have acquired skills although not at the rate expected based on their age and sex.
Time Frame Baseline, 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
Four subjects randomized to placebo were not included in the efficacy analyses. Two were pre-specified exclusions (defined as pilot subjects in the protocol) and two were found ineligible after enrollment.
Arm/Group Title Cord Blood Infusion - 6 Months Placebo Infusion - 6 Months
Hide Arm/Group Description:

Subjects will be randomized to receive a cord blood infusion at the baseline visit. The cord blood will be autologous (if available) or unrelated cord blood.

Cord Blood Infusion

Subjects will be randomized to receive a placebo infusion at the baseline visit. The placebo is an acellular media product similar in both appearance and odor.

Placebo

Overall Number of Participants Analyzed 119 57
Mean (Standard Deviation)
Unit of Measure: score on a scale
3.13  (8.76) 1.98  (8.41)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Cord Blood Infusion - 6 Months, Placebo Infusion - 6 Months
Comments Null hypothesis: The mean of the 6-month change in VABS-II Socialization Subscale Standard Score is the same for the Cord Blood and Placebo groups.
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.83
Comments [Not Specified]
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Final Values)
Estimated Value 1.14
Confidence Interval (2-Sided) 95%
-1.58 to 3.86
Estimation Comments [Not Specified]
2.Secondary Outcome
Title Change in Vineland Socialization Domain Raw Score
Hide Description There are 3 raw scores within the Socialization domain of the VABS-3. These are the Interpersonal Relationships Raw Score (range: 0-86), the Play and Leisure Raw Score (range: 0-72), and the Coping Skills Raw Score (range: 0-66). Higher numbers on all three scores reflect better functioning in each area. Each raw score is the sum of the item scores in the respective subdomain of Socialization skills. The items are scored as follows: 2=usually present, 1=sometimes present, 0= never present. The item scores are assigned by a trained interviewer who interviews the parent of the child participating in the study. The change in raw score was calculated for each participant from Baseline to Month 6. Positive scores indicate improvement over time whereas negative scores indicate worsening, and zero indicates no change. The scores are not norm-referenced.
Time Frame Baseline, 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
Four subjects randomized to placebo were not included in the efficacy analyses. Two were pre-specified exclusions (defined as pilot subjects in the protocol) and two were found ineligible after enrollment.
Arm/Group Title Cord Blood Infusion - 6 Months Placebo Infusion - 6 Months
Hide Arm/Group Description:

Subjects will be randomized to receive a cord blood infusion at the baseline visit. The cord blood will be autologous (if available) or unrelated cord blood.

Cord Blood Infusion

Subjects will be randomized to receive a placebo infusion at the baseline visit. The placebo is an acellular media product similar in both appearance and odor.

Placebo

Overall Number of Participants Analyzed 119 57
Mean (Standard Deviation)
Unit of Measure: score on a scale
Interpersonal Relationships Raw Score 5.40  (9.13) 4.11  (9.42)
Play and Leisure Raw Score 4.76  (9.84) 3.09  (7.68)
Coping Skills Raw Score 3.82  (7.54) 2.91  (7.21)
3.Secondary Outcome
Title Change in Vineland Socialization Domain Age Equivalent
Hide Description There are 3 age equivalent scores within the Socialization domain of the VABS-3: the Interpersonal Relationships Age Equivalent, the Play and Leisure Age Equivalent, and the Coping Sills Age Equivalent. An individual participant's age equivalent represents the chronological age (in years:months) at which their score would be considered normative. The age equivalent ranges are 0:0-22:0, 0:0-20:0, and 2:0-22:0 for the Interpersonal Relationships, Play and Leisure and Coping Skills age equivalents, respectively. The change in this age equivalent was calculated for each participant from Baseline to Month 6 and expressed as a number of months. Positive scores indicate increases in the age equivalent of the participant's social communication skills over time and are considered an improvement. Negative scores indicate decreases in the age equivalent of the participant's social communication skills and are considered worsening, and zero indicates no change.
Time Frame Baseline, 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
Four subjects randomized to placebo were not included in the efficacy analyses. Two were pre-specified exclusions (defined as pilot subjects in the protocol) and two were found ineligible after enrollment.
Arm/Group Title Cord Blood Infusion - 6 Months Placebo Infusion - 6 Months
Hide Arm/Group Description:

Subjects will be randomized to receive a cord blood infusion at the baseline visit. The cord blood will be autologous (if available) or unrelated cord blood.

Cord Blood Infusion

Subjects will be randomized to receive a placebo infusion at the baseline visit. The placebo is an acellular media product similar in both appearance and odor.

Placebo

Overall Number of Participants Analyzed 119 57
Mean (Standard Deviation)
Unit of Measure: months
Interpersonal Relationships Age Equivalent Score 0.37  (0.72) 0.19  (0.79)
Play and Leisure Age Equivalent Score 0.41  (1.17) 0.30  (1.21)
Coping Skills Age Equivalent Score 0.26  (0.77) 0.18  (0.89)
4.Secondary Outcome
Title Change in Pervasive Developmental Disorder Behavior Inventory (PDD-BI) Composite Standard Score (Parent Questionnaire)
Hide Description The PDD-BI is an informant-based rating scale that assesses problem behaviors as well as appropriate social, language, and learning/memory skills. The PDD-BI assesses both social impairments and development of pro-social skills that are integral to improved reciprocal social behavior. The PDD-BI renders T scores (mean=50, standard deviation=10) based on comparisons to a standardized ASD population. The Autism Composite T score ranges from 10-100. The typical child with autism scores between 40-60.Higher scores indicate more severe autism symptoms and lower scores reflect milder symptoms. Change in this score from Baseline to Month 6 was calculated for each participant. Negative change scores indicate improvement in autism symptoms over time whereas positive scores indicate worsening of symptoms, and zero indicates no change in symptoms.
Time Frame Baseline, 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
Four subjects randomized to placebo were not included in the efficacy analyses. Two were pre-specified exclusions (defined as pilot subjects in the protocol) and two were found ineligible after enrollment.
Arm/Group Title Cord Blood Infusion - 6 Months Placebo Infusion - 6 Months
Hide Arm/Group Description:

Subjects will be randomized to receive a cord blood infusion at the baseline visit. The cord blood will be autologous (if available) or unrelated cord blood.

Cord Blood Infusion

Subjects will be randomized to receive a placebo infusion at the baseline visit. The placebo is an acellular media product similar in both appearance and odor.

Placebo

Overall Number of Participants Analyzed 119 57
Mean (Standard Deviation)
Unit of Measure: score on a scale
-6.65  (10.44) -6.12  (8.82)
5.Secondary Outcome
Title Change in Clinical Global Impressions - Severity of Illness (CGI-S) Score, Clinician Assessment
Hide Description The CGI-S is a 7 point scale completed at the baseline and 6-month visits that requires the clinician to rate the severity of the participant's symptoms of autism at the time of assessment, relative to the clinician's past experience with participants who have the same diagnosis. There are 3 CGI-S scores: the Social Communication Score, the Restricted and Repetitive Behaviors Score, and the Overall Score. The clinician's rates the severity of autism symptoms - 1, normal, no symptoms; 2, borderline level of symptoms; 3, mild symptoms; 4, moderate symptoms; 5, marked symptoms; 6, severe symptoms; or 7, extremely severe symptoms. Increases in the change score represent increases in symptom severity, decreases in the change score indicate improvement, and zero indicates no change.
Time Frame Baseline, 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
Four subjects randomized to placebo were not included in the efficacy analyses. Two were pre-specified exclusions (defined as pilot subjects in the protocol) and two were found ineligible after enrollment. Two additional subjects were excluded from the Cord Blood group because they were missing assessments.
Arm/Group Title Cord Blood Infusion - 6 Months Placebo Infusion - 6 Months
Hide Arm/Group Description:

Subjects will be randomized to receive a cord blood infusion at the baseline visit. The cord blood will be autologous (if available) or unrelated cord blood.

Cord Blood Infusion

Subjects will be randomized to receive a placebo infusion at the baseline visit. The placebo is an acellular media product similar in both appearance and odor.

Placebo

Overall Number of Participants Analyzed 117 57
Mean (Standard Deviation)
Unit of Measure: score on a scale
Social Communication Score -0.11  (0.58) -0.19  (0.52)
Restricted, Repetitive Behaviors Score -0.25  (0.73) -0.18  (0.73)
Overall Score -0.15  (0.57) -0.26  (0.58)
6.Secondary Outcome
Title Clinical Global Impressions - Global Improvement (CGI-I) Score, Clinician Assessment
Hide Description The CGI-I is a 7 point scale that requires the clinician to assess how much the participant's autism symptoms have improved or worsened relative to a baseline assessment. The symptoms are rated as: 1, very much improved; 2, much improved; 3, minimally improved; 4, no change; 5, minimally worse; 6, much worse; or 7, very much worse. There are three separate CGI-I ratings: social communicative functioning, restricted/repetitive interests and behaviors, and overall improvement.
Time Frame Baseline, 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
Four subjects randomized to placebo were not included in the efficacy analyses. Two were pre-specified exclusions (defined as pilot subjects in the protocol) and two were found ineligible after enrollment. Two additional subjects were excluded from the Cord Blood group because they were missing assessments.
Arm/Group Title Cord Blood Infusion - 6 Months Placebo Infusion - 6 Months
Hide Arm/Group Description:

Subjects will be randomized to receive a cord blood infusion at the baseline visit. The cord blood will be autologous (if available) or unrelated cord blood.

Cord Blood Infusion

Subjects will be randomized to receive a placebo infusion at the baseline visit. The placebo is an acellular media product similar in both appearance and odor.

Placebo

Overall Number of Participants Analyzed 117 57
Measure Type: Count of Participants
Unit of Measure: Participants
Social Communicative Functioning 1=Very Much Improved
0
   0.0%
0
   0.0%
2=Much Improved
26
  22.2%
9
  15.8%
3=Minimally Improved
30
  25.6%
16
  28.1%
4=No Change
58
  49.6%
31
  54.4%
5=Minimally Worse
3
   2.6%
1
   1.8%
6=Much Worse
0
   0.0%
0
   0.0%
Restricted/repetitive Interests and Behaviors 1=Very Much Improved
1
   0.9%
0
   0.0%
2=Much Improved
17
  14.5%
11
  19.3%
3=Minimally Improved
29
  24.8%
10
  17.5%
4=No Change
59
  50.4%
29
  50.9%
5=Minimally Worse
10
   8.5%
7
  12.3%
6=Much Worse
1
   0.9%
0
   0.0%
Overall Score 1=Very Much Improved
1
   0.9%
0
   0.0%
2=Much Improved
26
  22.2%
11
  19.3%
3=Minimally Improved
37
  31.6%
18
  31.6%
4=No Change
47
  40.2%
27
  47.4%
5=Minimally Worse
6
   5.1%
1
   1.8%
6=Much Worse
0
   0.0%
0
   0.0%
7.Secondary Outcome
Title Change in Expressive One-Word Picture Vocabulary Test (Clinician Assessment)
Hide Description This Expressive One-Word Picture Vocabulary Test is a standardized evaluation of the child's expressive one-word vocabulary by a trained clinician. It tests an individual's ability to name, with one word, objects, actions, and concepts when presented with color illustrations. Higher EOWPVT standard scores reflect a better vocabulary. The minimum score is age-dependent (years:months) as follows. For a child age 2:0 to 2:1 the minimum is 65; age 2:2-2:3 (min=62); age 2:4-2:5 (min=60); age 2:6-2:7 (min=58); age 2:8-2:9 (min=57); age 2:10-2:11 (min=56); age 3:0 and older (min=55). The maximum possible score across all ages is 145. The change in score from Baseline to Month 6 was the outcome measure. Increases reflect increases in vocabulary skills, decreasing reflect decreases in vocabulary skills, and zero reflects no change.
Time Frame Baseline, 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
Four subjects randomized to placebo were not included in the efficacy analyses. Two were pre-specified exclusions (defined as pilot subjects in the protocol) and two were found ineligible after enrollment. Three additional subjects were excluded from the Cord Blood group because they were missing assessments.
Arm/Group Title Cord Blood Infusion - 6 Months Placebo Infusion - 6 Months
Hide Arm/Group Description:

Subjects will be randomized to receive a cord blood infusion at the baseline visit. The cord blood will be autologous (if available) or unrelated cord blood.

Cord Blood Infusion

Subjects will be randomized to receive a placebo infusion at the baseline visit. The placebo is an acellular media product similar in both appearance and odor.

Placebo

Overall Number of Participants Analyzed 116 57
Mean (Standard Deviation)
Unit of Measure: score on a scale
0.89  (7.48) 1.88  (10.18)
8.Secondary Outcome
Title Change in Vineland Adaptive Behavior Scales II (VABS-II) Communication Subscale Standard Score
Hide Description The VABS-II measures adaptive functioning in socialization, communication, daily living, and motor skills. The Communication subscale standard score is derived by summing norm-referenced (by age group and sex) v-scale scores (mean=15, standard deviation=3) from the Receptive, Expressive, and Written communication subdomains and standardizing this sum to a normal distribution with mean=100 and standard deviation=15. Changes in the Communication standard score are indicative of skill acquisition relative to chronologically aged peers of the same sex. Thus, a zero (no change) represents change consistent with what is expected. An increase represents acquisition of more skills over time than would be expected. Participants who experience a decrease in Communication standard score may still have acquired skills although not at the rate expected based on their age and sex.
Time Frame Baseline, 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
Four subjects randomized to placebo were not included in the efficacy analyses. Two were pre-specified exclusions (defined as pilot subjects in the protocol) and two were found ineligible after enrollment.
Arm/Group Title Cord Blood Infusion - 6 Months Placebo Infusion - 6 Months
Hide Arm/Group Description:

Subjects will be randomized to receive a cord blood infusion at the baseline visit. The cord blood will be autologous (if available) or unrelated cord blood.

Cord Blood Infusion

Subjects will be randomized to receive a placebo infusion at the baseline visit. The placebo is an acellular media product similar in both appearance and odor.

Placebo

Overall Number of Participants Analyzed 119 57
Mean (Standard Deviation)
Unit of Measure: score on a scale
2.87  (8.95) 2.81  (9.68)
9.Secondary Outcome
Title Change in Vineland Adaptive Behavior Scales II (VABS-II) Daily Living Subscale Standard Score
Hide Description The VABS-II measures adaptive functioning in socialization, communication, daily living, and motor skills. The Daily Living standard score is derived by summing norm-referenced (by age group and sex) v-scale scores (mean=15, standard deviation=3) from the Personal, Domestic and Community subdomains and standardizing this sum to a normal distribution with mean=100 and standard deviation=15. Changes in the Daily Living standard score are indicative of skill acquisition relative to chronologically aged peers of the same sex. Thus, a zero (no change) represents change consistent with what is expected. An increase represents acquisition of more skills over time than would be expected. Participants who experience a decrease in Daily Living standard score may still have acquired skills although not at the rate expected based on their age and sex.
Time Frame Baseline, 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
Four subjects randomized to placebo were not included in the efficacy analyses. Two were pre-specified exclusions (defined as pilot subjects in the protocol) and two were found ineligible after enrollment.
Arm/Group Title Cord Blood Infusion - 6 Months Placebo Infusion - 6 Months
Hide Arm/Group Description:

Subjects will be randomized to receive a cord blood infusion at the baseline visit. The cord blood will be autologous (if available) or unrelated cord blood.

Cord Blood Infusion

Subjects will be randomized to receive a placebo infusion at the baseline visit. The placebo is an acellular media product similar in both appearance and odor.

Placebo

Overall Number of Participants Analyzed 119 57
Mean (Standard Deviation)
Unit of Measure: score on a scale
2.21  (6.89) 2.49  (7.41)
10.Secondary Outcome
Title Change in Vineland Adaptive Behavior Scales II (VABS-II) Composite Score
Hide Description The Vineland Adaptive Behavior Scales II (VABS-II) measures adaptive functioning in socialization, communication, daily living, and motor skills. The Adaptive Behavior Composite provides an overall summary of adaptive behavior across all of the domains. Each participant's score is standardized to a normal distribution with mean=100 and standard deviation=15. Positive scores indicate an increase in the Adaptive Behavior Composite Score over time whereas negative scores indicate decrease in the Adaptive Behavior Composite Score, and zero indicates no change in the Adaptive Behavior Composite Score.
Time Frame Baseline, 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
Four subjects randomized to placebo were not included in the efficacy analyses. Two were pre-specified exclusions (defined as pilot subjects in the protocol) and two were found ineligible after enrollment.
Arm/Group Title Cord Blood Infusion - 6 Months Placebo Infusion - 6 Months
Hide Arm/Group Description:

Subjects will be randomized to receive a cord blood infusion at the baseline visit. The cord blood will be autologous (if available) or unrelated cord blood.

Cord Blood Infusion

Subjects will be randomized to receive a placebo infusion at the baseline visit. The placebo is an acellular media product similar in both appearance and odor.

Placebo

Overall Number of Participants Analyzed 119 57
Mean (Standard Deviation)
Unit of Measure: score on a scale
2.34  (5.93) 2.09  (6.05)
11.Secondary Outcome
Title Change in Pervasive Developmental Disorder Behavior Inventory (PDD-BI) Repetitive, Ritualistic and Pragmatic Problems T-Score
Hide Description The PDD-BI is an informant-based rating scale that assesses problem behaviors as well as appropriate social, language, and learning/memory skills. The Repetitive, Ritualistic and Pragmatic Problems T-Score (mean=50, standard deviation=10) measures a broad range of behavioral problems associated with autism. The score ranges from 26-100 in patients aged 2-8 years. Higher values indicate increasing levels of severity. The change in this score from Baseline to Month 6 was calculated for each participant. Increases in the change score reflect worsening of problem behaviors, decreases indicate improvement, and zero indicates no change.
Time Frame Baseline, Month 6
Hide Outcome Measure Data
Hide Analysis Population Description
Four subjects randomized to placebo were not included in the efficacy analyses. Two were pre-specified exclusions (defined as pilot subjects in the protocol) and two were found ineligible after enrollment.
Arm/Group Title Cord Blood Infusion - 6 Months Placebo Infusion - 6 Months
Hide Arm/Group Description:

Subjects will be randomized to receive a cord blood infusion at the baseline visit. The cord blood will be autologous (if available) or unrelated cord blood.

Cord Blood Infusion

Subjects will be randomized to receive a placebo infusion at the baseline visit. The placebo is an acellular media product similar in both appearance and odor.

Placebo

Overall Number of Participants Analyzed 119 57
Mean (Standard Deviation)
Unit of Measure: T score
-5.50  (10.38) -5.18  (8.47)
12.Secondary Outcome
Title Change in PDD-BI Approach/Withdrawal Problems T-Score
Hide Description The PDD-BI is an informant-based rating scale that assesses problem behaviors as well as appropriate social, language, and learning/memory skills. The Approach/Withdrawal Problems T-Score (mean=50, standard deviation=10) measures a broad range of behavioral problems associated with autism. The score ranges from 27-100 in patients aged 2-8 years. Higher values indicate increasing levels of severity. The change in this score from Baseline to Month 6 was calculated for each participant. Increases in the change score reflect worsening of problem behaviors, decreases indicate improvement, and zero indicates no change.
Time Frame Baseline, Month 6
Hide Outcome Measure Data
Hide Analysis Population Description
Four subjects randomized to placebo were not included in the efficacy analyses. Two were pre-specified exclusions (defined as pilot subjects in the protocol) and two were found ineligible after enrollment.
Arm/Group Title Cord Blood Infusion - 6 Months Placebo Infusion - 6 Months
Hide Arm/Group Description:

Subjects will be randomized to receive a cord blood infusion at the baseline visit. The cord blood will be autologous (if available) or unrelated cord blood.

Cord Blood Infusion

Subjects will be randomized to receive a placebo infusion at the baseline visit. The placebo is an acellular media product similar in both appearance and odor.

Placebo

Overall Number of Participants Analyzed 119 57
Mean (Standard Deviation)
Unit of Measure: T score
-5.42  (10.60) -4.86  (8.09)
13.Secondary Outcome
Title Change in PDD-BI Sensory/Perceptual Approach Behaviors T-Score
Hide Description The PDD-BI is an informant-based rating scale that assesses problem behaviors as well as appropriate social, language, and learning/memory skills. The Sensory/Perceptual Approach Behaviors T-score (mean=50, standard deviation=10) includes behaviors that are largely non-communicative and involve approach toward asocial stimuli. The score ranges from 31 to 86 in patients aged 2-8 years. Higher values indicate increasing levels of severity. The change in this score from Baseline to Month 6 was calculated for each participant. Increases in the change score reflect worsening of problem behaviors, decreases indicate improvement, and zero indicates no change.
Time Frame Baseline, Month 6
Hide Outcome Measure Data
Hide Analysis Population Description
Four subjects randomized to placebo were not included in the efficacy analyses. Two were pre-specified exclusions (defined as pilot subjects in the protocol) and two were found ineligible after enrollment.
Arm/Group Title Cord Blood Infusion - 6 Months Placebo Infusion - 6 Months
Hide Arm/Group Description:

Subjects will be randomized to receive a cord blood infusion at the baseline visit. The cord blood will be autologous (if available) or unrelated cord blood.

Cord Blood Infusion

Subjects will be randomized to receive a placebo infusion at the baseline visit. The placebo is an acellular media product similar in both appearance and odor.

Placebo

Overall Number of Participants Analyzed 119 57
Mean (Standard Deviation)
Unit of Measure: T score
-4.13  (6.80) -2.75  (6.66)
14.Secondary Outcome
Title Change in PDD-BI Ritualisms/Resistance to Change T-Score
Hide Description The PDD-BI is an informant-based rating scale that assesses problem behaviors as well as appropriate social, language, and learning/memory skills. The Ritualisms/Resistance to Change T-Score (mean=50, standard deviation=10) describes behaviors that communicate the child's desires to carry out rituals or to communicate dissatisfaction with a change in the environment or routine. The score ranges from 34 to 100 for participants aged 2-8 years. Higher values indicate increasing levels of severity. The change in this score from Baseline to Month 6 was calculated for each participant. Increases in the change score reflect worsening of problem behaviors, decreases indicate improvement, and zero indicates no change.
Time Frame Baseline, Month 6
Hide Outcome Measure Data
Hide Analysis Population Description
Four subjects randomized to placebo were not included in the efficacy analyses. Two were pre-specified exclusions (defined as pilot subjects in the protocol) and two were found ineligible after enrollment.
Arm/Group Title Cord Blood Infusion - 6 Months Placebo Infusion - 6 Months
Hide Arm/Group Description:

Subjects will be randomized to receive a cord blood infusion at the baseline visit. The cord blood will be autologous (if available) or unrelated cord blood.

Cord Blood Infusion

Subjects will be randomized to receive a placebo infusion at the baseline visit. The placebo is an acellular media product similar in both appearance and odor.

Placebo

Overall Number of Participants Analyzed 119 57
Mean (Standard Deviation)
Unit of Measure: T score
-5.66  (7.62) -5.00  (8.61)
15.Secondary Outcome
Title Change in PDD-BI Social Pragmatic Problems T-Score
Hide Description The PDD-BI is an informant-based rating scale that assesses problem behaviors as well as appropriate social, language, and learning/memory skills. The Social Pragmatic Problems T-Score (mean=50, standard deviation=10) measures the difficulties children with autism have in either reacting to the approaches of others, understanding social conventions, or initiating social interactions with others. The score ranges from 29 to 100 for participants aged 2-8 years. Higher values indicate increasing levels of severity. The change in this score from Baseline to Month 6 was calculated for each participant. Increases in the change score reflect worsening of problem behaviors, decreases indicate improvement, and zero indicates no change.
Time Frame Baseline, Month 6
Hide Outcome Measure Data
Hide Analysis Population Description
Four subjects randomized to placebo were not included in the efficacy analyses. Two were pre-specified exclusions (defined as pilot subjects in the protocol) and two were found ineligible after enrollment.
Arm/Group Title Cord Blood Infusion - 6 Months Placebo Infusion - 6 Months
Hide Arm/Group Description:

Subjects will be randomized to receive a cord blood infusion at the baseline visit. The cord blood will be autologous (if available) or unrelated cord blood.

Cord Blood Infusion

Subjects will be randomized to receive a placebo infusion at the baseline visit. The placebo is an acellular media product similar in both appearance and odor.

Placebo

Overall Number of Participants Analyzed 119 57
Mean (Standard Deviation)
Unit of Measure: T score
-3.66  (11.36) -3.33  (8.78)
16.Secondary Outcome
Title Change in PDD-BI Semantic/Pragmatic Problems T-Score
Hide Description The PDD-BI is an informant-based rating scale that assesses problem behaviors as well as appropriate social, language, and learning/memory skills. The Semantic/Pragmatic Problems T-Score (mean=50, standard deviation=10) assesses the difficulties children with autism have in using spoken language to indicate comprehension, communicate meaning, respond to the interests of others, and sustain a conversation. The score ranges from 34 to 100 for participants aged 2-8 years. Higher values indicate increasing levels of severity. The change in this score from Baseline to Month 6 was calculated for each participant. Increases in the change score reflect worsening of problem behaviors, decreases indicate improvement, and zero indicates no change.
Time Frame Baseline, Month 6
Hide Outcome Measure Data
Hide Analysis Population Description
Four subjects randomized to placebo were not included in the efficacy analyses. Two were pre-specified exclusions (defined as pilot subjects in the protocol) and two were found ineligible after enrollment.
Arm/Group Title Cord Blood Infusion - 6 Months Placebo Infusion - 6 Months
Hide Arm/Group Description:

Subjects will be randomized to receive a cord blood infusion at the baseline visit. The cord blood will be autologous (if available) or unrelated cord blood.

Cord Blood Infusion

Subjects will be randomized to receive a placebo infusion at the baseline visit. The placebo is an acellular media product similar in both appearance and odor.

Placebo

Overall Number of Participants Analyzed 119 57
Mean (Standard Deviation)
Unit of Measure: T score
-3.82  (7.51) -3.68  (7.90)
17.Secondary Outcome
Title Change in PDD-BI Arousal Regulation Problems T-Score
Hide Description The PDD-BI is an informant-based rating scale that assesses problem behaviors as well as appropriate social, language, and learning/memory skills. The Arousal Regulation Problems T-Score (mean=50, standard deviation=10) measures behaviors that are largely non-communicative or unresponsive and reflect emotional constriction, the apparent seeking of kinesthetic sensation, and, in the parent version, difficulty with sleep regulation. The score ranges from 26 to 77 for participants aged 2-8 years. Higher values indicate increasing levels of severity. The change in this score from Baseline to Month 6 was calculated for each participant. Increases in the change score reflect worsening of problem behaviors, decreases indicate improvement, and zero indicates no change.
Time Frame Baseline, Month 6
Hide Outcome Measure Data
Hide Analysis Population Description
Four subjects randomized to placebo were not included in the efficacy analyses. Two were pre-specified exclusions (defined as pilot subjects in the protocol) and two were found ineligible after enrollment.
Arm/Group Title Cord Blood Infusion - 6 Months Placebo Infusion - 6 Months
Hide Arm/Group Description:

Subjects will be randomized to receive a cord blood infusion at the baseline visit. The cord blood will be autologous (if available) or unrelated cord blood.

Cord Blood Infusion

Subjects will be randomized to receive a placebo infusion at the baseline visit. The placebo is an acellular media product similar in both appearance and odor.

Placebo

Overall Number of Participants Analyzed 119 57
Mean (Standard Deviation)
Unit of Measure: T score
-4.34  (7.49) -5.02  (7.28)
18.Secondary Outcome
Title Change in PDD-BI Specific Fears T-Score
Hide Description The PDD-BI is an informant-based rating scale that assesses problem behaviors as well as appropriate social, language, and learning/memory skills. The Specific Fears T-Score (mean=50, standard deviation=10) measures behaviors that communicate the fears and anxieties associated with withdrawal from social or asocial stimuli. The score ranges from 36 to 100 for participants aged 2-8 years. Higher values indicate increasing levels of severity. The change in this score from Baseline to Month 6 was calculated for each participant. Increases in the change score reflect worsening of problem behaviors, decreases indicate improvement, and zero indicates no change.
Time Frame Baseline, Month 6
Hide Outcome Measure Data
Hide Analysis Population Description
Four subjects randomized to placebo were not included in the efficacy analyses. Two were pre-specified exclusions (defined as pilot subjects in the protocol) and two were found ineligible after enrollment.
Arm/Group Title Cord Blood Infusion - 6 Months Placebo Infusion - 6 Months
Hide Arm/Group Description:

Subjects will be randomized to receive a cord blood infusion at the baseline visit. The cord blood will be autologous (if available) or unrelated cord blood.

Cord Blood Infusion

Subjects will be randomized to receive a placebo infusion at the baseline visit. The placebo is an acellular media product similar in both appearance and odor.

Placebo

Overall Number of Participants Analyzed 119 57
Mean (Standard Deviation)
Unit of Measure: T score
-4.24  (8.81) -3.00  (8.82)
19.Secondary Outcome
Title Change in PDD-BI Aggressiveness T-Score
Hide Description The PDD-BI is an informant-based rating scale that assesses problem behaviors as well as appropriate social, language, and learning/memory skills. The Aggressiveness T-Score (mean=50, standard deviation=10) assesses the aggressive approach toward self or others, as well as the negative mood changes that are often associated with such behaviors. The score ranges from 36 to 100 for participants aged 2-8 years. Higher values indicate increasing levels of severity. The change in this score from Baseline to Month 6 was calculated for each participant. Increases in the change score reflect worsening of problem behaviors, decreases indicate improvement, and zero indicates no change.
Time Frame Baseline, Month 6
Hide Outcome Measure Data
Hide Analysis Population Description
Four subjects randomized to placebo were not included in the efficacy analyses. Two were pre-specified exclusions (defined as pilot subjects in the protocol) and two were found ineligible after enrollment.
Arm/Group Title Cord Blood Infusion - 6 Months Placebo Infusion - 6 Months
Hide Arm/Group Description:

Subjects will be randomized to receive a cord blood infusion at the baseline visit. The cord blood will be autologous (if available) or unrelated cord blood.

Cord Blood Infusion

Subjects will be randomized to receive a placebo infusion at the baseline visit. The placebo is an acellular media product similar in both appearance and odor.

Placebo

Overall Number of Participants Analyzed 119 57
Mean (Standard Deviation)
Unit of Measure: T score
-4.31  (11.41) -1.79  (9.07)
20.Secondary Outcome
Title Change in PDD-BI Expressive Social Communication Abilities T-Score
Hide Description The PDD-BI is an informant-based rating scale that assesses problem behaviors as well as appropriate social, language, and learning/memory skills. The Expressive Social Communication Abilities T-Score (mean=50, standard deviation=10) measures a broad range of social communication skills affected by autism. The score ranges from 20 to 100 for participants aged 2-8 years. Higher values indicate increasing levels of competence. The change in this score from Baseline to Month 6 was calculated for each participant. Increases in the change score reflect improvement, decreases indicate worsening, and zero indicates no change.
Time Frame Baseline, Month 6
Hide Outcome Measure Data
Hide Analysis Population Description
Four subjects randomized to placebo were not included in the efficacy analyses. Two were pre-specified exclusions (defined as pilot subjects in the protocol) and two were found ineligible after enrollment.
Arm/Group Title Cord Blood Infusion - 6 Months Placebo Infusion - 6 Months
Hide Arm/Group Description:

Subjects will be randomized to receive a cord blood infusion at the baseline visit. The cord blood will be autologous (if available) or unrelated cord blood.

Cord Blood Infusion

Subjects will be randomized to receive a placebo infusion at the baseline visit. The placebo is an acellular media product similar in both appearance and odor.

Placebo

Overall Number of Participants Analyzed 119 57
Mean (Standard Deviation)
Unit of Measure: T score
3.20  (5.34) 2.74  (5.55)
21.Secondary Outcome
Title Change in PDD-BI Receptive/Expressive Social Communication Ability T-Score
Hide Description The PDD-BI is an informant-based rating scale that assesses problem behaviors as well as appropriate social, language, and learning/memory skills. The Receptive/Expressive Social Communication Ability T-Score (mean=50, standard deviation=10) measures a broad range of social communication skills affected by autism. The score ranges from 20 to 100 for participants aged 2-8 years. Higher values indicate increasing levels of competence. The change in this score from Baseline to Month 6 was calculated for each participant. Increases in the change score reflect improvement, decreases indicate worsening, and zero indicates no change.
Time Frame Baseline, Month 6
Hide Outcome Measure Data
Hide Analysis Population Description
Four subjects randomized to placebo were not included in the efficacy analyses. Two were pre-specified exclusions (defined as pilot subjects in the protocol) and two were found ineligible after enrollment.
Arm/Group Title Cord Blood Infusion - 6 Months Placebo Infusion - 6 Months
Hide Arm/Group Description:

Subjects will be randomized to receive a cord blood infusion at the baseline visit. The cord blood will be autologous (if available) or unrelated cord blood.

Cord Blood Infusion

Subjects will be randomized to receive a placebo infusion at the baseline visit. The placebo is an acellular media product similar in both appearance and odor.

Placebo

Overall Number of Participants Analyzed 119 57
Mean (Standard Deviation)
Unit of Measure: T score
2.99  (5.09) 2.28  (5.16)
22.Secondary Outcome
Title Change in PDD-BI Social Approach Behaviors T-Score
Hide Description The PDD-BI is an informant-based rating scale that assesses problem behaviors as well as appropriate social, language, and learning/memory skills. The Social Approach Behaviors T-Score (mean=50, standard deviation=10) assesses those social communication skills that are notoriously difficult for children with autism (e.g., eye contact, joint attention, effective use of gesture, imaginative skills). The score ranges from 14 to 93 for participants aged 2-8 years. Higher values indicate increasing levels of competence. The change in this score from Baseline to Month 6 was calculated for each participant. Increases in the change score reflect improvement, decreases indicate worsening, and zero indicates no change.
Time Frame Baseline, Month 6
Hide Outcome Measure Data
Hide Analysis Population Description
Four subjects randomized to placebo were not included in the efficacy analyses. Two were pre-specified exclusions (defined as pilot subjects in the protocol) and two were found ineligible after enrollment.
Arm/Group Title Cord Blood Infusion - 6 Months Placebo Infusion - 6 Months
Hide Arm/Group Description:

Subjects will be randomized to receive a cord blood infusion at the baseline visit. The cord blood will be autologous (if available) or unrelated cord blood.

Cord Blood Infusion

Subjects will be randomized to receive a placebo infusion at the baseline visit. The placebo is an acellular media product similar in both appearance and odor.

Placebo

Overall Number of Participants Analyzed 119 57
Mean (Standard Deviation)
Unit of Measure: T score
3.92  (6.46) 3.23  (5.99)
23.Secondary Outcome
Title Change in PDD-BI Expressive Language T-Score
Hide Description The PDD-BI is an informant-based rating scale that assesses problem behaviors as well as appropriate social, language, and learning/memory skills. The Expressive Language T-Score (mean=50, standard deviation=10) assesses the ability of the child to speak the sounds associated with the English language and to use words and sentences that indicate his or her competence with grammar, tone of voice, and the pragmatic aspects of communicating with others. The score ranges from 28 to 100 for participants aged 2-8 years. Higher values indicate increasing levels of competence. The change in this score from Baseline to Month 6 was calculated for each participant. Increases in the change score reflect improvement, decreases indicate worsening, and zero indicates no change.
Time Frame Baseline, Month 6
Hide Outcome Measure Data
Hide Analysis Population Description
Four subjects randomized to placebo were not included in the efficacy analyses. Two were pre-specified exclusions (defined as pilot subjects in the protocol) and two were found ineligible after enrollment.
Arm/Group Title Cord Blood Infusion - 6 Months Placebo Infusion - 6 Months
Hide Arm/Group Description:

Subjects will be randomized to receive a cord blood infusion at the baseline visit. The cord blood will be autologous (if available) or unrelated cord blood.

Cord Blood Infusion

Subjects will be randomized to receive a placebo infusion at the baseline visit. The placebo is an acellular media product similar in both appearance and odor.

Placebo

Overall Number of Participants Analyzed 119 57
Mean (Standard Deviation)
Unit of Measure: T score
2.03  (4.92) 1.86  (5.07)
24.Secondary Outcome
Title Change in PDD-BI Learning, Memory, and Receptive Language T-Score
Hide Description The PDD-BI is an informant-based rating scale that assesses problem behaviors as well as appropriate social, language, and learning/memory skills. The Learning, Memory, and Receptive Language T-Score (mean=50, standard deviation=10) assesses two areas of variable competence in children with autism: (a) memory and (b) receptive language. The score ranges from 22 to 88 for participants aged 2-8 years. Higher values indicate increasing levels of competence. The change in this score from Baseline to Month 6 was calculated for each participant. Increases in the change score reflect improvement, decreases indicate worsening, and zero indicates no change.
Time Frame Baseline, Month 6
Hide Outcome Measure Data
Hide Analysis Population Description
Four subjects randomized to placebo were not included in the efficacy analyses. Two were pre-specified exclusions (defined as pilot subjects in the protocol) and two were found ineligible after enrollment.
Arm/Group Title Cord Blood Infusion - 6 Months Placebo Infusion - 6 Months
Hide Arm/Group Description:

Subjects will be randomized to receive a cord blood infusion at the baseline visit. The cord blood will be autologous (if available) or unrelated cord blood.

Cord Blood Infusion

Subjects will be randomized to receive a placebo infusion at the baseline visit. The placebo is an acellular media product similar in both appearance and odor.

Placebo

Overall Number of Participants Analyzed 119 57
Mean (Standard Deviation)
Unit of Measure: T score
2.02  (5.45) 1.05  (5.37)
25.Secondary Outcome
Title Number of Participants With Infusion Reactions
Hide Description [Not Specified]
Time Frame 12 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Cord Blood Infusion - 12 Months Placebo Infusion - 12 Months
Hide Arm/Group Description:

Subjects will be randomized to receive a cord blood infusion at the baseline visit. The cord blood will be autologous (if available) or unrelated cord blood.

Cord Blood Infusion

Subjects will be randomized to receive a placebo infusion at the baseline visit. The placebo is an acellular media product similar in both appearance and odor.

Placebo

Overall Number of Participants Analyzed 61 119
Measure Type: Count of Participants
Unit of Measure: Participants
2
   3.3%
1
   0.8%
26.Secondary Outcome
Title Severity of Infusion Reactions
Hide Description Grade/severity will be assessed according to CTCAE v4.0 guidelines
Time Frame 12 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Cord Blood Infusion - 12 Months Placebo Infusion - 12 Months
Hide Arm/Group Description:

Subjects will be randomized to receive a cord blood infusion at the baseline visit. The cord blood will be autologous (if available) or unrelated cord blood.

Cord Blood Infusion

Subjects will be randomized to receive a placebo infusion at the baseline visit. The placebo is an acellular media product similar in both appearance and odor.

Placebo

Overall Number of Participants Analyzed 61 119
Measure Type: Count of Participants
Unit of Measure: Participants
Mild
2
   3.3%
0
   0.0%
Moderate
0
   0.0%
1
   0.8%
Severe
0
   0.0%
0
   0.0%
27.Secondary Outcome
Title Number of Participants With Product-related Infections
Hide Description [Not Specified]
Time Frame 12 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Cord Blood Infusion - 12 Months Placebo Infusion - 12 Months
Hide Arm/Group Description:

Subjects will be randomized to receive a cord blood infusion at the baseline visit. The cord blood will be autologous (if available) or unrelated cord blood.

Cord Blood Infusion

Subjects will be randomized to receive a placebo infusion at the baseline visit. The placebo is an acellular media product similar in both appearance and odor.

Placebo

Overall Number of Participants Analyzed 61 119
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
0
   0.0%
28.Secondary Outcome
Title Severity of Product-related Infections
Hide Description Grade/severity will be assessed according to CTCAE v4.0 guidelines
Time Frame 12 months
Hide Outcome Measure Data
Hide Analysis Population Description
There were no product-related infections in the study.
Arm/Group Title Cord Blood Infusion - 12 Months Placebo Infusion - 12 Months
Hide Arm/Group Description:

Subjects will be randomized to receive a cord blood infusion at the baseline visit. The cord blood will be autologous (if available) or unrelated cord blood.

Cord Blood Infusion

Subjects will be randomized to receive a placebo infusion at the baseline visit. The placebo is an acellular media product similar in both appearance and odor.

Placebo

Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
29.Secondary Outcome
Title Evidence of Alloimmunization Via Anti-HLA (Human Leukocyte Antigen) and Anti-RBC (Red Blood Cell) Antibodies and Nonspecific Markers of Systemic Inflammation (ESR, CRP)
Hide Description [Not Specified]
Time Frame 12 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Cord Blood Infusion - 12 Months Placebo Infusion - 12 Months
Hide Arm/Group Description:

Subjects will be randomized to receive a cord blood infusion at the baseline visit. The cord blood will be autologous (if available) or unrelated cord blood.

Cord Blood Infusion

Subjects will be randomized to receive a placebo infusion at the baseline visit. The placebo is an acellular media product similar in both appearance and odor.

Placebo

Overall Number of Participants Analyzed 61 119
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
0
   0.0%
30.Secondary Outcome
Title Incidence of Graft vs. Host Disease
Hide Description [Not Specified]
Time Frame 12 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Cord Blood Infusion - 12 Months Placebo Infusion - 12 Months
Hide Arm/Group Description:

Subjects will be randomized to receive a cord blood infusion at the baseline visit. The cord blood will be autologous (if available) or unrelated cord blood.

Cord Blood Infusion

Subjects will be randomized to receive a placebo infusion at the baseline visit. The placebo is an acellular media product similar in both appearance and odor.

Placebo

Overall Number of Participants Analyzed 61 119
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
0
   0.0%
31.Secondary Outcome
Title Severity of Graft vs. Host Disease
Hide Description Grade/severity will be assessed according to Common Terminology Criteria for Adverse Events (CTCAE) v4.0 guidelines.
Time Frame 12 months
Hide Outcome Measure Data
Hide Analysis Population Description
There were no cases of graft vs. host disease in the study.
Arm/Group Title Cord Blood Infusion - 12 Months Placebo Infusion - 12 Months
Hide Arm/Group Description:

Subjects will be randomized to receive a cord blood infusion at the baseline visit. The cord blood will be autologous (if available) or unrelated cord blood.

Cord Blood Infusion

Subjects will be randomized to receive a placebo infusion at the baseline visit. The placebo is an acellular media product similar in both appearance and odor.

Placebo

Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
32.Secondary Outcome
Title Incidence of Unexpected Adverse Events, by Relation to Study Product
Hide Description [Not Specified]
Time Frame 6 months,12 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Cord Blood Infusion - 6 Months Placebo Infusion - 6 Months Cord Blood Infusion - 12 Months Placebo Infusion - 12 Months
Hide Arm/Group Description:

Subjects will be randomized to receive a cord blood infusion at the baseline visit. The cord blood will be autologous (if available) or unrelated cord blood.

Cord Blood Infusion

Subjects will be randomized to receive a placebo infusion at the baseline visit. The placebo is an acellular media product similar in both appearance and odor.

Placebo

Subjects will be randomized to receive a cord blood infusion at the baseline visit. The cord blood will be autologous (if available) or unrelated cord blood.

Cord Blood Infusion

Subjects will be randomized to receive a placebo infusion at the baseline visit. The placebo is an acellular media product similar in both appearance and odor.

Placebo

Overall Number of Participants Analyzed 119 61 61 119
Measure Type: Count of Participants
Unit of Measure: Participants
Related
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Unrelated
5
   4.2%
1
   1.6%
0
   0.0%
2
   1.7%
33.Secondary Outcome
Title Severity of Unexpected Adverse Events, by Relation to Study Product
Hide Description Grade/severity will be assessed according to CTCAE v4.0 guidelines
Time Frame 6 months, 12 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Cord Blood Infusion - 6 Months Placebo Infusion - 6 Months Cord Blood Infusion - 12 Months Placebo Infusion - 12 Months
Hide Arm/Group Description:

Subjects will be randomized to receive a cord blood infusion at the baseline visit. The cord blood will be autologous (if available) or unrelated cord blood.

Cord Blood Infusion

Subjects will be randomized to receive a placebo infusion at the baseline visit. The placebo is an acellular media product similar in both appearance and odor.

Placebo

Subjects will be randomized to receive a cord blood infusion at the baseline visit. The cord blood will be autologous (if available) or unrelated cord blood.

Cord Blood Infusion

Subjects will be randomized to receive a placebo infusion at the baseline visit. The placebo is an acellular media product similar in both appearance and odor.

Placebo

Overall Number of Participants Analyzed 119 61 61 119
Measure Type: Count of Participants
Unit of Measure: Participants
Mild : Related
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Mild : Unrelated
2
   1.7%
0
   0.0%
0
   0.0%
2
   1.7%
Moderate : Related
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Moderate : Unrelated
3
   2.5%
1
   1.6%
0
   0.0%
0
   0.0%
Severe : Related
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Severe : Unrelated
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Time Frame 6 months and 12 months
Adverse Event Reporting Description Adverse events collected on Safety Population (all 180 participants). Participants were randomized to receive a single infusion of umbilical cord blood or placebo and evaluated at six months. Participants then received a second infusion in a blinded fashion (placebo for participants initially randomized to cord blood, and cord blood for participants initially randomized to placebo). Participants were then followed for an additional 6 months for safety data collection.
 
Arm/Group Title Cord Blood Infusion - 6 Months Placebo Infusion - 6 Months Cord Blood Infusion - 12 Months Placebo Infusion - 12 Months
Hide Arm/Group Description

Subjects will be randomized to receive a cord blood infusion at the baseline visit. The cord blood will be autologous (if available) or unrelated cord blood.

Cord Blood Infusion

Subjects will be randomized to receive a placebo infusion at the baseline visit. The placebo is an acellular media product similar in both appearance and odor.

Placebo

Subjects will be randomized to receive a cord blood infusion at the baseline visit. The cord blood will be autologous (if available) or unrelated cord blood.

Cord Blood Infusion

Subjects will be randomized to receive a placebo infusion at the baseline visit. The placebo is an acellular media product similar in both appearance and odor.

Placebo

All-Cause Mortality
Cord Blood Infusion - 6 Months Placebo Infusion - 6 Months Cord Blood Infusion - 12 Months Placebo Infusion - 12 Months
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/119 (0.00%)   0/61 (0.00%)   0/61 (0.00%)   0/119 (0.00%) 
Hide Serious Adverse Events
Cord Blood Infusion - 6 Months Placebo Infusion - 6 Months Cord Blood Infusion - 12 Months Placebo Infusion - 12 Months
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   3/119 (2.52%)   3/61 (4.92%)   3/61 (4.92%)   2/119 (1.68%) 
Infections and infestations         
Gastroenteritis * 1  0/119 (0.00%)  1/61 (1.64%)  1/61 (1.64%)  0/119 (0.00%) 
Injury, poisoning and procedural complications         
Concussion * 1  1/119 (0.84%)  0/61 (0.00%)  0/61 (0.00%)  1/119 (0.84%) 
Metabolism and nutrition disorders         
Dehydration * 1  1/119 (0.84%)  1/61 (1.64%)  1/61 (1.64%)  1/119 (0.84%) 
Psychiatric disorders         
Aggression * 1  0/119 (0.00%)  1/61 (1.64%)  1/61 (1.64%)  0/119 (0.00%) 
Pediatric autoimmune neuropsychiatric disorders associated with streptococcal infection * 1  1/119 (0.84%)  0/61 (0.00%)  0/61 (0.00%)  0/119 (0.00%) 
1
Term from vocabulary, MedDRA
*
Indicates events were collected by non-systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Cord Blood Infusion - 6 Months Placebo Infusion - 6 Months Cord Blood Infusion - 12 Months Placebo Infusion - 12 Months
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   97/119 (81.51%)   51/61 (83.61%)   41/61 (67.21%)   83/119 (69.75%) 
Ear and labyrinth disorders         
Ear pain * 1  1/119 (0.84%)  0/61 (0.00%)  1/61 (1.64%)  0/119 (0.00%) 
Ear discomfort * 1  0/119 (0.00%)  0/61 (0.00%)  0/61 (0.00%)  1/119 (0.84%) 
Hypoacusis * 1  0/119 (0.00%)  0/61 (0.00%)  0/61 (0.00%)  1/119 (0.84%) 
Eye disorders         
Excessive eye blinking * 1  1/119 (0.84%)  1/61 (1.64%)  0/61 (0.00%)  0/119 (0.00%) 
Eye pain * 1  1/119 (0.84%)  0/61 (0.00%)  0/61 (0.00%)  0/119 (0.00%) 
Photophobia * 1  1/119 (0.84%)  0/61 (0.00%)  0/61 (0.00%)  0/119 (0.00%) 
Strabismus * 1  1/119 (0.84%)  0/61 (0.00%)  0/61 (0.00%)  0/119 (0.00%) 
Eye inflammation * 1  0/119 (0.00%)  0/61 (0.00%)  0/61 (0.00%)  1/119 (0.84%) 
Visual impairment * 1  0/119 (0.00%)  0/61 (0.00%)  0/61 (0.00%)  1/119 (0.84%) 
Gastrointestinal disorders         
Abdominal pain upper * 1  1/119 (0.84%)  0/61 (0.00%)  1/61 (1.64%)  0/119 (0.00%) 
Anal incontinence * 1  2/119 (1.68%)  1/61 (1.64%)  0/61 (0.00%)  0/119 (0.00%) 
Constipation * 1  2/119 (1.68%)  1/61 (1.64%)  1/61 (1.64%)  3/119 (2.52%) 
Dental caries * 1  2/119 (1.68%)  0/61 (0.00%)  0/61 (0.00%)  2/119 (1.68%) 
Diarrhea * 1  5/119 (4.20%)  3/61 (4.92%)  3/61 (4.92%)  2/119 (1.68%) 
Dyspepsia * 1  2/119 (1.68%)  0/61 (0.00%)  0/61 (0.00%)  0/119 (0.00%) 
Flatulence * 1  1/119 (0.84%)  0/61 (0.00%)  0/61 (0.00%)  0/119 (0.00%) 
Food poisoning * 1  1/119 (0.84%)  0/61 (0.00%)  0/61 (0.00%)  0/119 (0.00%) 
Frequent bowel movements * 1  1/119 (0.84%)  0/61 (0.00%)  2/61 (3.28%)  0/119 (0.00%) 
Gastroesophageal reflux disease * 1  2/119 (1.68%)  0/61 (0.00%)  0/61 (0.00%)  2/119 (1.68%) 
Nausea * 1  1/119 (0.84%)  0/61 (0.00%)  1/61 (1.64%)  0/119 (0.00%) 
Toothache * 1  1/119 (0.84%)  0/61 (0.00%)  0/61 (0.00%)  0/119 (0.00%) 
Vomiting * 1  5/119 (4.20%)  2/61 (3.28%)  2/61 (3.28%)  2/119 (1.68%) 
Gastrointestinal dyskinetic disorders * 1  0/119 (0.00%)  0/61 (0.00%)  0/61 (0.00%)  1/119 (0.84%) 
General disorders         
Gait disturbance * 1  0/119 (0.00%)  1/61 (1.64%)  0/61 (0.00%)  0/119 (0.00%) 
Fatigue * 1  3/119 (2.52%)  0/61 (0.00%)  0/61 (0.00%)  0/119 (0.00%) 
Influenza like illness * 1  1/119 (0.84%)  2/61 (3.28%)  0/61 (0.00%)  1/119 (0.84%) 
Pyrexia * 1  11/119 (9.24%)  8/61 (13.11%)  8/61 (13.11%)  5/119 (4.20%) 
Thirst * 1  2/119 (1.68%)  0/61 (0.00%)  0/61 (0.00%)  1/119 (0.84%) 
Immune system disorders         
Food allergy * 1  1/119 (0.84%)  0/61 (0.00%)  0/61 (0.00%)  0/119 (0.00%) 
Hypersensitivity * 1  4/119 (3.36%)  3/61 (4.92%)  1/61 (1.64%)  9/119 (7.56%) 
Drug hypersensitivity * 1  0/119 (0.00%)  0/61 (0.00%)  0/61 (0.00%)  1/119 (0.84%) 
Infections and infestations         
Candida infection * 1  0/119 (0.00%)  1/61 (1.64%)  0/61 (0.00%)  0/119 (0.00%) 
Infection * 1  0/119 (0.00%)  1/61 (1.64%)  0/61 (0.00%)  0/119 (0.00%) 
Infectious mononucleosis * 1  0/119 (0.00%)  1/61 (1.64%)  0/61 (0.00%)  0/119 (0.00%) 
Lung infection * 1  0/119 (0.00%)  1/61 (1.64%)  0/61 (0.00%)  0/119 (0.00%) 
Molluscum contagiosum * 1  0/119 (0.00%)  1/61 (1.64%)  0/61 (0.00%)  0/119 (0.00%) 
Otitis externa * 1  0/119 (0.00%)  1/61 (1.64%)  0/61 (0.00%)  0/119 (0.00%) 
Tooth abscess * 1  0/119 (0.00%)  1/61 (1.64%)  1/61 (1.64%)  0/119 (0.00%) 
Body tinea * 1  2/119 (1.68%)  0/61 (0.00%)  0/61 (0.00%)  0/119 (0.00%) 
Conjunctivitis * 1  5/119 (4.20%)  1/61 (1.64%)  0/61 (0.00%)  2/119 (1.68%) 
Croup infectious * 1  1/119 (0.84%)  1/61 (1.64%)  0/61 (0.00%)  2/119 (1.68%) 
Ear infection * 1  10/119 (8.40%)  4/61 (6.56%)  2/61 (3.28%)  7/119 (5.88%) 
Erythema infection * 1  1/119 (0.84%)  0/61 (0.00%)  0/61 (0.00%)  0/119 (0.00%) 
Fungal infection * 1  2/119 (1.68%)  0/61 (0.00%)  0/61 (0.00%)  0/119 (0.00%) 
Gastroenteritis viral * 1  6/119 (5.04%)  3/61 (4.92%)  2/61 (3.28%)  6/119 (5.04%) 
Gastrointestinal infection * 1  1/119 (0.84%)  1/61 (1.64%)  1/61 (1.64%)  1/119 (0.84%) 
Hand-foot-and-mouth disease * 1  2/119 (1.68%)  0/61 (0.00%)  2/61 (3.28%)  1/119 (0.84%) 
Influenza * 1  8/119 (6.72%)  2/61 (3.28%)  2/61 (3.28%)  3/119 (2.52%) 
Nasopharyngitis * 1  2/119 (1.68%)  4/61 (6.56%)  0/61 (0.00%)  4/119 (3.36%) 
Otitis media * 1  6/119 (5.04%)  3/61 (4.92%)  0/61 (0.00%)  2/119 (1.68%) 
Overgrowth fungal * 1  1/119 (0.84%)  0/61 (0.00%)  0/61 (0.00%)  0/119 (0.00%) 
Pharyngitis Streptococcal * 1  10/119 (8.40%)  1/61 (1.64%)  2/61 (3.28%)  6/119 (5.04%) 
Pneumonia * 1  1/119 (0.84%)  0/61 (0.00%)  0/61 (0.00%)  0/119 (0.00%) 
Scarlet fever * 1  1/119 (0.84%)  1/61 (1.64%)  0/61 (0.00%)  0/119 (0.00%) 
Sinusitis * 1  1/119 (0.84%)  0/61 (0.00%)  1/61 (1.64%)  3/119 (2.52%) 
Streptococcal infection * 1  2/119 (1.68%)  5/61 (8.20%)  1/61 (1.64%)  2/119 (1.68%) 
Upper respiratory tract infection * 1  21/119 (17.65%)  10/61 (16.39%)  2/61 (3.28%)  11/119 (9.24%) 
Urinary tract infection * 1  2/119 (1.68%)  0/61 (0.00%)  0/61 (0.00%)  0/119 (0.00%) 
Viral infection * 1  2/119 (1.68%)  1/61 (1.64%)  0/61 (0.00%)  1/119 (0.84%) 
Oral herpes * 1  0/119 (0.00%)  0/61 (0.00%)  1/61 (1.64%)  0/119 (0.00%) 
Bronchitis * 1  0/119 (0.00%)  0/61 (0.00%)  0/61 (0.00%)  1/119 (0.84%) 
Injury, poisoning and procedural complications         
Clavicle fracture * 1  0/119 (0.00%)  1/61 (1.64%)  0/61 (0.00%)  0/119 (0.00%) 
Epiphyseal fracture * 1  0/119 (0.00%)  1/61 (1.64%)  0/61 (0.00%)  0/119 (0.00%) 
Allergic transfusion reaction * 1  7/119 (5.88%)  1/61 (1.64%)  2/61 (3.28%)  1/119 (0.84%) 
Fall * 1  1/119 (0.84%)  1/61 (1.64%)  0/61 (0.00%)  0/119 (0.00%) 
Infusion related reaction * 1  3/119 (2.52%)  1/61 (1.64%)  0/61 (0.00%)  0/119 (0.00%) 
Facial bones fracture * 1  0/119 (0.00%)  0/61 (0.00%)  1/61 (1.64%)  0/119 (0.00%) 
Wrist fracture * 1  0/119 (0.00%)  0/61 (0.00%)  1/61 (1.64%)  1/119 (0.84%) 
Investigations         
Blood urine present * 1  1/119 (0.84%)  0/61 (0.00%)  0/61 (0.00%)  0/119 (0.00%) 
Laboratory test abnormal * 1  4/119 (3.36%)  0/61 (0.00%)  2/61 (3.28%)  14/119 (11.76%) 
Vitamin D decreased * 1  2/119 (1.68%)  0/61 (0.00%)  0/61 (0.00%)  1/119 (0.84%) 
Streptococcus test positive * 1  0/119 (0.00%)  0/61 (0.00%)  1/61 (1.64%)  0/119 (0.00%) 
Cardiac murmur * 1  0/119 (0.00%)  0/61 (0.00%)  0/61 (0.00%)  1/119 (0.84%) 
Weight increased * 1  0/119 (0.00%)  0/61 (0.00%)  0/61 (0.00%)  2/119 (1.68%) 
Metabolism and nutrition disorders         
Appetite disorder * 1  1/119 (0.84%)  0/61 (0.00%)  0/61 (0.00%)  0/119 (0.00%) 
Decreased appetite * 1  2/119 (1.68%)  0/61 (0.00%)  1/61 (1.64%)  1/119 (0.84%) 
Cow's milk intolerance * 1  0/119 (0.00%)  0/61 (0.00%)  0/61 (0.00%)  1/119 (0.84%) 
Food refusal * 1  0/119 (0.00%)  0/61 (0.00%)  0/61 (0.00%)  1/119 (0.84%) 
Gluten sensitivity * 1  0/119 (0.00%)  0/61 (0.00%)  0/61 (0.00%)  1/119 (0.84%) 
Increased appetite * 1  0/119 (0.00%)  0/61 (0.00%)  0/61 (0.00%)  3/119 (2.52%) 
Musculoskeletal and connective tissue disorders         
Arthralgia * 1  0/119 (0.00%)  1/61 (1.64%)  0/61 (0.00%)  0/119 (0.00%) 
Joint laxity * 1  1/119 (0.84%)  0/61 (0.00%)  0/61 (0.00%)  0/119 (0.00%) 
Muscle rigidity * 1  6/119 (5.04%)  4/61 (6.56%)  1/61 (1.64%)  2/119 (1.68%) 
Toe walking * 1  1/119 (0.84%)  0/61 (0.00%)  0/61 (0.00%)  1/119 (0.84%) 
Neoplasms benign, malignant and unspecified (incl cysts and polyps)         
Skin papilloma * 1  1/119 (0.84%)  0/61 (0.00%)  0/61 (0.00%)  2/119 (1.68%) 
Nervous system disorders         
Restless legs syndrome * 1  0/119 (0.00%)  1/61 (1.64%)  0/61 (0.00%)  0/119 (0.00%) 
Disturbance in attention * 1  4/119 (3.36%)  2/61 (3.28%)  1/61 (1.64%)  1/119 (0.84%) 
Headache * 1  3/119 (2.52%)  2/61 (3.28%)  0/61 (0.00%)  1/119 (0.84%) 
Lethargy * 1  1/119 (0.84%)  0/61 (0.00%)  0/61 (0.00%)  1/119 (0.84%) 
Poor quality sleep * 1  1/119 (0.84%)  0/61 (0.00%)  0/61 (0.00%)  0/119 (0.00%) 
Psychomotor hyperactivity * 1  5/119 (4.20%)  5/61 (8.20%)  2/61 (3.28%)  6/119 (5.04%) 
Repetitive speech * 1  1/119 (0.84%)  1/61 (1.64%)  0/61 (0.00%)  1/119 (0.84%) 
Seizure * 1  1/119 (0.84%)  0/61 (0.00%)  0/61 (0.00%)  0/119 (0.00%) 
Sensory processing disorder * 1  10/119 (8.40%)  5/61 (8.20%)  2/61 (3.28%)  2/119 (1.68%) 
Somnolence * 1  1/119 (0.84%)  0/61 (0.00%)  0/61 (0.00%)  1/119 (0.84%) 
Syncope * 1  1/119 (0.84%)  0/61 (0.00%)  0/61 (0.00%)  0/119 (0.00%) 
Sensory disturbance * 1  0/119 (0.00%)  0/61 (0.00%)  1/61 (1.64%)  0/119 (0.00%) 
Speech disorder * 1  0/119 (0.00%)  0/61 (0.00%)  0/61 (0.00%)  1/119 (0.84%) 
Psychiatric disorders         
Bipolar disorder * 1  0/119 (0.00%)  1/61 (1.64%)  0/61 (0.00%)  0/119 (0.00%) 
Distractibility * 1  0/119 (0.00%)  1/61 (1.64%)  0/61 (0.00%)  0/119 (0.00%) 
Dysphemia * 1  0/119 (0.00%)  1/61 (1.64%)  0/61 (0.00%)  0/119 (0.00%) 
Affect lability * 1  9/119 (7.56%)  7/61 (11.48%)  0/61 (0.00%)  9/119 (7.56%) 
Aggression * 1  22/119 (18.49%)  12/61 (19.67%)  6/61 (9.84%)  14/119 (11.76%) 
Agitation * 1  13/119 (10.92%)  7/61 (11.48%)  6/61 (9.84%)  7/119 (5.88%) 
Anxiety * 1  1/119 (0.84%)  2/61 (3.28%)  1/61 (1.64%)  2/119 (1.68%) 
Apathy * 1  4/119 (3.36%)  1/61 (1.64%)  0/61 (0.00%)  1/119 (0.84%) 
Attention deficit/hyperactivity disorder * 1  1/119 (0.84%)  0/61 (0.00%)  0/61 (0.00%)  0/119 (0.00%) 
Confusional state * 1  1/119 (0.84%)  0/61 (0.00%)  0/61 (0.00%)  0/119 (0.00%) 
Defiant behavior * 1  10/119 (8.40%)  4/61 (6.56%)  4/61 (6.56%)  7/119 (5.88%) 
Depressed mood * 1  3/119 (2.52%)  0/61 (0.00%)  1/61 (1.64%)  0/119 (0.00%) 
Echolalia * 1  3/119 (2.52%)  0/61 (0.00%)  0/61 (0.00%)  1/119 (0.84%) 
Encopresis * 1  1/119 (0.84%)  0/61 (0.00%)  0/61 (0.00%)  0/119 (0.00%) 
Enuresis * 1  1/119 (0.84%)  0/61 (0.00%)  0/61 (0.00%)  1/119 (0.84%) 
Food aversion * 1  1/119 (0.84%)  0/61 (0.00%)  0/61 (0.00%)  0/119 (0.00%) 
Frustration tolerance decreased * 1  4/119 (3.36%)  2/61 (3.28%)  2/61 (3.28%)  4/119 (3.36%) 
Impulsive behavior * 1  3/119 (2.52%)  3/61 (4.92%)  1/61 (1.64%)  1/119 (0.84%) 
Insomnia * 1  11/119 (9.24%)  2/61 (3.28%)  5/61 (8.20%)  6/119 (5.04%) 
Intentional self-injury * 1  4/119 (3.36%)  3/61 (4.92%)  2/61 (3.28%)  1/119 (0.84%) 
Irritability * 1  6/119 (5.04%)  3/61 (4.92%)  0/61 (0.00%)  3/119 (2.52%) 
Negativism * 1  3/119 (2.52%)  2/61 (3.28%)  1/61 (1.64%)  0/119 (0.00%) 
Pediatric autoimmune neuropsychiatric disorders associated with streptococcal infection * 1  1/119 (0.84%)  0/61 (0.00%)  0/61 (0.00%)  0/119 (0.00%) 
Separation anxiety disorder * 1  1/119 (0.84%)  0/61 (0.00%)  0/61 (0.00%)  0/119 (0.00%) 
Sleep disorder * 1  1/119 (0.84%)  0/61 (0.00%)  0/61 (0.00%)  0/119 (0.00%) 
Social avoidant behavior * 1  1/119 (0.84%)  1/61 (1.64%)  0/61 (0.00%)  0/119 (0.00%) 
Stereotypy * 1  16/119 (13.45%)  6/61 (9.84%)  1/61 (1.64%)  6/119 (5.04%) 
Tic * 1  2/119 (1.68%)  1/61 (1.64%)  1/61 (1.64%)  2/119 (1.68%) 
Aversion * 1  0/119 (0.00%)  0/61 (0.00%)  1/61 (1.64%)  0/119 (0.00%) 
Restlessness * 1  0/119 (0.00%)  0/61 (0.00%)  1/61 (1.64%)  0/119 (0.00%) 
Reproductive system and breast disorders         
Nipple swelling * 1  0/119 (0.00%)  0/61 (0.00%)  0/61 (0.00%)  1/119 (0.84%) 
Respiratory, thoracic and mediastinal disorders         
Rhinorrhea * 1  0/119 (0.00%)  2/61 (3.28%)  1/61 (1.64%)  0/119 (0.00%) 
Asthma * 1  2/119 (1.68%)  0/61 (0.00%)  0/61 (0.00%)  0/119 (0.00%) 
Epistaxis * 1  5/119 (4.20%)  1/61 (1.64%)  1/61 (1.64%)  3/119 (2.52%) 
Hypoxia * 1  1/119 (0.84%)  0/61 (0.00%)  0/61 (0.00%)  0/119 (0.00%) 
Cough * 1  0/119 (0.00%)  0/61 (0.00%)  1/61 (1.64%)  4/119 (3.36%) 
Oropharyngeal pain * 1  0/119 (0.00%)  0/61 (0.00%)  1/61 (1.64%)  0/119 (0.00%) 
Nasal congestion * 1  0/119 (0.00%)  0/61 (0.00%)  0/61 (0.00%)  1/119 (0.84%) 
Sinus congestion * 1  0/119 (0.00%)  0/61 (0.00%)  0/61 (0.00%)  1/119 (0.84%) 
Skin and subcutaneous tissue disorders         
Dermatitis diaper * 1  0/119 (0.00%)  1/61 (1.64%)  0/61 (0.00%)  0/119 (0.00%) 
Eczema * 1  2/119 (1.68%)  3/61 (4.92%)  0/61 (0.00%)  3/119 (2.52%) 
Pruritus * 1  1/119 (0.84%)  0/61 (0.00%)  0/61 (0.00%)  0/119 (0.00%) 
Rash * 1  4/119 (3.36%)  2/61 (3.28%)  3/61 (4.92%)  4/119 (3.36%) 
Rash papular * 1  1/119 (0.84%)  0/61 (0.00%)  0/61 (0.00%)  0/119 (0.00%) 
Urticaria * 1  2/119 (1.68%)  0/61 (0.00%)  0/61 (0.00%)  0/119 (0.00%) 
Alopecia * 1  0/119 (0.00%)  0/61 (0.00%)  0/61 (0.00%)  1/119 (0.84%) 
Dermatitis contact * 1  0/119 (0.00%)  0/61 (0.00%)  0/61 (0.00%)  1/119 (0.84%) 
Surgical and medical procedures         
Adenoidectomy * 1  0/119 (0.00%)  1/61 (1.64%)  0/61 (0.00%)  1/119 (0.84%) 
Ear tube removal * 1  0/119 (0.00%)  1/61 (1.64%)  0/61 (0.00%)  0/119 (0.00%) 
Tonsillectomy * 1  0/119 (0.00%)  1/61 (1.64%)  0/61 (0.00%)  0/119 (0.00%) 
Myringotomy * 1  0/119 (0.00%)  0/61 (0.00%)  0/61 (0.00%)  1/119 (0.84%) 
Tooth repair * 1  0/119 (0.00%)  0/61 (0.00%)  0/61 (0.00%)  1/119 (0.84%) 
Tooth extraction * 1  0/119 (0.00%)  0/61 (0.00%)  0/61 (0.00%)  1/119 (0.84%) 
Vascular disorders         
Hypotension * 1  1/119 (0.84%)  0/61 (0.00%)  0/61 (0.00%)  0/119 (0.00%) 
Pallor * 1  1/119 (0.84%)  0/61 (0.00%)  0/61 (0.00%)  0/119 (0.00%) 
1
Term from vocabulary, MedDRA
*
Indicates events were collected by non-systematic assessment
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Jesse D. Troy, PhD, MPH
Organization: Duke University
Phone: 919-668-1102
EMail: cordbloodtherapyinfo@dm.duke.edu
Layout table for additonal information
Responsible Party: Joanne Kurtzberg, MD, Duke University
ClinicalTrials.gov Identifier: NCT02847182    
Other Study ID Numbers: Pro00070514
First Submitted: July 25, 2016
First Posted: July 28, 2016
Results First Submitted: August 20, 2019
Results First Posted: December 26, 2019
Last Update Posted: June 9, 2020