Trial Outcomes & Findings for Cord Blood Infusion for Children With Autism Spectrum Disorder (NCT NCT02847182)

Last Updated: 2020-06-09

Results Overview

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.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

180 participants

Primary outcome timeframe

Baseline, 6 months

Results posted on

2020-06-09

Participant Flow

Participant milestones

Participant milestones
Measure	Cord Blood Infusion, Then Placebo Infusion 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	Placebo Infusion, Then Cord Blood Infusion 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
Overall Study STARTED	119	61
Overall Study First Intervention (6 Months)	119	61
Overall Study Second Intervention (6 Months)	61	119
Overall Study COMPLETED	119	60
Overall Study NOT COMPLETED	0	1

Reasons for withdrawal

Reasons for withdrawal
Measure	Cord Blood Infusion, Then Placebo Infusion 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	Placebo Infusion, Then Cord Blood Infusion 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
Overall Study Withdrawal by Subject	0	1

Baseline Characteristics

Cord Blood Infusion for Children With Autism Spectrum Disorder

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Cord Blood Infusion, Then Placebo Infusion n=119 Participants 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	Placebo Infusion, Then Cord Blood Infusion n=61 Participants 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 n=180 Participants Total of all reporting groups
Age, Continuous	5.30 years n=5 Participants	5.24 years n=7 Participants	5.29 years n=5 Participants
Sex: Female, Male Female	21 Participants n=5 Participants	16 Participants n=7 Participants	37 Participants n=5 Participants
Sex: Female, Male Male	98 Participants n=5 Participants	45 Participants n=7 Participants	143 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	26 Participants n=5 Participants	6 Participants n=7 Participants	32 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	93 Participants n=5 Participants	55 Participants n=7 Participants	148 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race/Ethnicity, Customized Non-White	24 Participants n=5 Participants	17 Participants n=7 Participants	41 Participants n=5 Participants
Race/Ethnicity, Customized White	95 Participants n=5 Participants	44 Participants n=7 Participants	139 Participants n=5 Participants
Region of Enrollment United States	119 Participants n=5 Participants	61 Participants n=7 Participants	180 Participants n=5 Participants

PRIMARY outcome

Timeframe: Baseline, 6 months

Outcome measures

Outcome measures
Measure	Cord Blood Infusion - 6 Months n=119 Participants 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	Placebo Infusion - 6 Months n=57 Participants 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	Cord Blood Infusion - 12 Months 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	Placebo Infusion - 12 Months 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
Change in Social Communication as Measured by the Vineland Adaptive Behavior Scales, Third Edition (VABS-3)	3.13 score on a scale Standard Deviation 8.76	1.98 score on a scale Standard Deviation 8.41	—	—

SECONDARY outcome

Timeframe: Baseline, 6 months

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.

Outcome measures

Outcome measures
Measure	Cord Blood Infusion - 6 Months n=119 Participants 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	Placebo Infusion - 6 Months n=57 Participants 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	Cord Blood Infusion - 12 Months 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	Placebo Infusion - 12 Months 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
Change in Vineland Socialization Domain Raw Score Interpersonal Relationships Raw Score	5.40 score on a scale Standard Deviation 9.13	4.11 score on a scale Standard Deviation 9.42	—	—
Change in Vineland Socialization Domain Raw Score Play and Leisure Raw Score	4.76 score on a scale Standard Deviation 9.84	3.09 score on a scale Standard Deviation 7.68	—	—
Change in Vineland Socialization Domain Raw Score Coping Skills Raw Score	3.82 score on a scale Standard Deviation 7.54	2.91 score on a scale Standard Deviation 7.21	—	—

SECONDARY outcome

Timeframe: Baseline, 6 months

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.

Outcome measures

Outcome measures
Measure	Cord Blood Infusion - 6 Months n=119 Participants 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	Placebo Infusion - 6 Months n=57 Participants 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	Cord Blood Infusion - 12 Months 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	Placebo Infusion - 12 Months 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
Change in Vineland Socialization Domain Age Equivalent Play and Leisure Age Equivalent Score	0.41 months Standard Deviation 1.17	0.30 months Standard Deviation 1.21	—	—
Change in Vineland Socialization Domain Age Equivalent Coping Skills Age Equivalent Score	0.26 months Standard Deviation 0.77	0.18 months Standard Deviation 0.89	—	—
Change in Vineland Socialization Domain Age Equivalent Interpersonal Relationships Age Equivalent Score	0.37 months Standard Deviation 0.72	0.19 months Standard Deviation 0.79	—	—

SECONDARY outcome

Timeframe: Baseline, 6 months

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.

Outcome measures

Outcome measures
Measure	Cord Blood Infusion - 6 Months n=119 Participants 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	Placebo Infusion - 6 Months n=57 Participants 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	Cord Blood Infusion - 12 Months 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	Placebo Infusion - 12 Months 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
Change in Pervasive Developmental Disorder Behavior Inventory (PDD-BI) Composite Standard Score (Parent Questionnaire)	-6.65 score on a scale Standard Deviation 10.44	-6.12 score on a scale Standard Deviation 8.82	—	—

SECONDARY outcome

Timeframe: Baseline, 6 months

Population: 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.

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.

Outcome measures

Outcome measures
Measure	Cord Blood Infusion - 6 Months n=117 Participants 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	Placebo Infusion - 6 Months n=57 Participants 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	Cord Blood Infusion - 12 Months 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	Placebo Infusion - 12 Months 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
Change in Clinical Global Impressions - Severity of Illness (CGI-S) Score, Clinician Assessment Social Communication Score	-0.11 score on a scale Standard Deviation 0.58	-0.19 score on a scale Standard Deviation 0.52	—	—
Change in Clinical Global Impressions - Severity of Illness (CGI-S) Score, Clinician Assessment Restricted, Repetitive Behaviors Score	-0.25 score on a scale Standard Deviation 0.73	-0.18 score on a scale Standard Deviation 0.73	—	—
Change in Clinical Global Impressions - Severity of Illness (CGI-S) Score, Clinician Assessment Overall Score	-0.15 score on a scale Standard Deviation 0.57	-0.26 score on a scale Standard Deviation 0.58	—	—

SECONDARY outcome

Timeframe: Baseline, 6 months

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.

Outcome measures

Outcome measures
Measure	Cord Blood Infusion - 6 Months n=117 Participants 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	Placebo Infusion - 6 Months n=57 Participants 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	Cord Blood Infusion - 12 Months 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	Placebo Infusion - 12 Months 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
Clinical Global Impressions - Global Improvement (CGI-I) Score, Clinician Assessment Overall Score · 5=Minimally Worse	6 Participants	1 Participants	—	—
Clinical Global Impressions - Global Improvement (CGI-I) Score, Clinician Assessment Social Communicative Functioning · 1=Very Much Improved	0 Participants	0 Participants	—	—
Clinical Global Impressions - Global Improvement (CGI-I) Score, Clinician Assessment Social Communicative Functioning · 2=Much Improved	26 Participants	9 Participants	—	—
Clinical Global Impressions - Global Improvement (CGI-I) Score, Clinician Assessment Social Communicative Functioning · 3=Minimally Improved	30 Participants	16 Participants	—	—
Clinical Global Impressions - Global Improvement (CGI-I) Score, Clinician Assessment Social Communicative Functioning · 4=No Change	58 Participants	31 Participants	—	—
Clinical Global Impressions - Global Improvement (CGI-I) Score, Clinician Assessment Social Communicative Functioning · 5=Minimally Worse	3 Participants	1 Participants	—	—
Clinical Global Impressions - Global Improvement (CGI-I) Score, Clinician Assessment Social Communicative Functioning · 6=Much Worse	0 Participants	0 Participants	—	—
Clinical Global Impressions - Global Improvement (CGI-I) Score, Clinician Assessment Restricted/repetitive Interests and Behaviors · 1=Very Much Improved	1 Participants	0 Participants	—	—
Clinical Global Impressions - Global Improvement (CGI-I) Score, Clinician Assessment Restricted/repetitive Interests and Behaviors · 2=Much Improved	17 Participants	11 Participants	—	—
Clinical Global Impressions - Global Improvement (CGI-I) Score, Clinician Assessment Restricted/repetitive Interests and Behaviors · 3=Minimally Improved	29 Participants	10 Participants	—	—
Clinical Global Impressions - Global Improvement (CGI-I) Score, Clinician Assessment Restricted/repetitive Interests and Behaviors · 4=No Change	59 Participants	29 Participants	—	—
Clinical Global Impressions - Global Improvement (CGI-I) Score, Clinician Assessment Restricted/repetitive Interests and Behaviors · 5=Minimally Worse	10 Participants	7 Participants	—	—
Clinical Global Impressions - Global Improvement (CGI-I) Score, Clinician Assessment Restricted/repetitive Interests and Behaviors · 6=Much Worse	1 Participants	0 Participants	—	—
Clinical Global Impressions - Global Improvement (CGI-I) Score, Clinician Assessment Overall Score · 1=Very Much Improved	1 Participants	0 Participants	—	—
Clinical Global Impressions - Global Improvement (CGI-I) Score, Clinician Assessment Overall Score · 2=Much Improved	26 Participants	11 Participants	—	—
Clinical Global Impressions - Global Improvement (CGI-I) Score, Clinician Assessment Overall Score · 3=Minimally Improved	37 Participants	18 Participants	—	—
Clinical Global Impressions - Global Improvement (CGI-I) Score, Clinician Assessment Overall Score · 4=No Change	47 Participants	27 Participants	—	—
Clinical Global Impressions - Global Improvement (CGI-I) Score, Clinician Assessment Overall Score · 6=Much Worse	0 Participants	0 Participants	—	—

SECONDARY outcome

Timeframe: Baseline, 6 months

Population: 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.

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.

Outcome measures

Outcome measures
Measure	Cord Blood Infusion - 6 Months n=116 Participants 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	Placebo Infusion - 6 Months n=57 Participants 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	Cord Blood Infusion - 12 Months 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	Placebo Infusion - 12 Months 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
Change in Expressive One-Word Picture Vocabulary Test (Clinician Assessment)	0.89 score on a scale Standard Deviation 7.48	1.88 score on a scale Standard Deviation 10.18	—	—

SECONDARY outcome

Timeframe: Baseline, 6 months

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.

Outcome measures

Outcome measures
Measure	Cord Blood Infusion - 6 Months n=119 Participants 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	Placebo Infusion - 6 Months n=57 Participants 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	Cord Blood Infusion - 12 Months 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	Placebo Infusion - 12 Months 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
Change in Vineland Adaptive Behavior Scales II (VABS-II) Communication Subscale Standard Score	2.87 score on a scale Standard Deviation 8.95	2.81 score on a scale Standard Deviation 9.68	—	—

SECONDARY outcome

Timeframe: Baseline, 6 months

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.

Outcome measures

Outcome measures
Measure	Cord Blood Infusion - 6 Months n=119 Participants 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	Placebo Infusion - 6 Months n=57 Participants 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	Cord Blood Infusion - 12 Months 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	Placebo Infusion - 12 Months 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
Change in Vineland Adaptive Behavior Scales II (VABS-II) Daily Living Subscale Standard Score	2.21 score on a scale Standard Deviation 6.89	2.49 score on a scale Standard Deviation 7.41	—	—

SECONDARY outcome

Timeframe: Baseline, 6 months

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.

Outcome measures

Outcome measures
Measure	Cord Blood Infusion - 6 Months n=119 Participants 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	Placebo Infusion - 6 Months n=57 Participants 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	Cord Blood Infusion - 12 Months 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	Placebo Infusion - 12 Months 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
Change in Vineland Adaptive Behavior Scales II (VABS-II) Composite Score	2.34 score on a scale Standard Deviation 5.93	2.09 score on a scale Standard Deviation 6.05	—	—

SECONDARY outcome

Timeframe: Baseline, Month 6

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.

Outcome measures

Outcome measures
Measure	Cord Blood Infusion - 6 Months n=119 Participants 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	Placebo Infusion - 6 Months n=57 Participants 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	Cord Blood Infusion - 12 Months 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	Placebo Infusion - 12 Months 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
Change in Pervasive Developmental Disorder Behavior Inventory (PDD-BI) Repetitive, Ritualistic and Pragmatic Problems T-Score	-5.50 T score Standard Deviation 10.38	-5.18 T score Standard Deviation 8.47	—	—

SECONDARY outcome

Timeframe: Baseline, Month 6

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.

Outcome measures

Outcome measures
Measure	Cord Blood Infusion - 6 Months n=119 Participants 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	Placebo Infusion - 6 Months n=57 Participants 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	Cord Blood Infusion - 12 Months 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	Placebo Infusion - 12 Months 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
Change in PDD-BI Approach/Withdrawal Problems T-Score	-5.42 T score Standard Deviation 10.60	-4.86 T score Standard Deviation 8.09	—	—

SECONDARY outcome

Timeframe: Baseline, Month 6

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.

Outcome measures

Outcome measures
Measure	Cord Blood Infusion - 6 Months n=119 Participants 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	Placebo Infusion - 6 Months n=57 Participants 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	Cord Blood Infusion - 12 Months 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	Placebo Infusion - 12 Months 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
Change in PDD-BI Sensory/Perceptual Approach Behaviors T-Score	-4.13 T score Standard Deviation 6.80	-2.75 T score Standard Deviation 6.66	—	—

SECONDARY outcome

Timeframe: Baseline, Month 6

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.

Outcome measures

Outcome measures
Measure	Cord Blood Infusion - 6 Months n=119 Participants 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	Placebo Infusion - 6 Months n=57 Participants 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	Cord Blood Infusion - 12 Months 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	Placebo Infusion - 12 Months 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
Change in PDD-BI Ritualisms/Resistance to Change T-Score	-5.66 T score Standard Deviation 7.62	-5.00 T score Standard Deviation 8.61	—	—

SECONDARY outcome

Timeframe: Baseline, Month 6

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.

Outcome measures

Outcome measures
Measure	Cord Blood Infusion - 6 Months n=119 Participants 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	Placebo Infusion - 6 Months n=57 Participants 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	Cord Blood Infusion - 12 Months 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	Placebo Infusion - 12 Months 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
Change in PDD-BI Social Pragmatic Problems T-Score	-3.66 T score Standard Deviation 11.36	-3.33 T score Standard Deviation 8.78	—	—

SECONDARY outcome

Timeframe: Baseline, Month 6

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.

Outcome measures

Outcome measures
Measure	Cord Blood Infusion - 6 Months n=119 Participants 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	Placebo Infusion - 6 Months n=57 Participants 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	Cord Blood Infusion - 12 Months 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	Placebo Infusion - 12 Months 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
Change in PDD-BI Semantic/Pragmatic Problems T-Score	-3.82 T score Standard Deviation 7.51	-3.68 T score Standard Deviation 7.90	—	—

SECONDARY outcome

Timeframe: Baseline, Month 6

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.

Outcome measures

Outcome measures
Measure	Cord Blood Infusion - 6 Months n=119 Participants 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	Placebo Infusion - 6 Months n=57 Participants 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	Cord Blood Infusion - 12 Months 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	Placebo Infusion - 12 Months 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
Change in PDD-BI Arousal Regulation Problems T-Score	-4.34 T score Standard Deviation 7.49	-5.02 T score Standard Deviation 7.28	—	—

SECONDARY outcome

Timeframe: Baseline, Month 6

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.

Outcome measures

Outcome measures
Measure	Cord Blood Infusion - 6 Months n=119 Participants 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	Placebo Infusion - 6 Months n=57 Participants 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	Cord Blood Infusion - 12 Months 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	Placebo Infusion - 12 Months 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
Change in PDD-BI Specific Fears T-Score	-4.24 T score Standard Deviation 8.81	-3.00 T score Standard Deviation 8.82	—	—

SECONDARY outcome

Timeframe: Baseline, Month 6

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.

Outcome measures

Outcome measures
Measure	Cord Blood Infusion - 6 Months n=119 Participants 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	Placebo Infusion - 6 Months n=57 Participants 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	Cord Blood Infusion - 12 Months 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	Placebo Infusion - 12 Months 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
Change in PDD-BI Aggressiveness T-Score	-4.31 T score Standard Deviation 11.41	-1.79 T score Standard Deviation 9.07	—	—

SECONDARY outcome

Timeframe: Baseline, Month 6

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.

Outcome measures

Outcome measures
Measure	Cord Blood Infusion - 6 Months n=119 Participants 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	Placebo Infusion - 6 Months n=57 Participants 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	Cord Blood Infusion - 12 Months 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	Placebo Infusion - 12 Months 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
Change in PDD-BI Expressive Social Communication Abilities T-Score	3.20 T score Standard Deviation 5.34	2.74 T score Standard Deviation 5.55	—	—

SECONDARY outcome

Timeframe: Baseline, Month 6

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.

Outcome measures

Outcome measures
Measure	Cord Blood Infusion - 6 Months n=119 Participants 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	Placebo Infusion - 6 Months n=57 Participants 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	Cord Blood Infusion - 12 Months 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	Placebo Infusion - 12 Months 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
Change in PDD-BI Receptive/Expressive Social Communication Ability T-Score	2.99 T score Standard Deviation 5.09	2.28 T score Standard Deviation 5.16	—	—

SECONDARY outcome

Timeframe: Baseline, Month 6

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.

Outcome measures

Outcome measures
Measure	Cord Blood Infusion - 6 Months n=119 Participants 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	Placebo Infusion - 6 Months n=57 Participants 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	Cord Blood Infusion - 12 Months 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	Placebo Infusion - 12 Months 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
Change in PDD-BI Social Approach Behaviors T-Score	3.92 T score Standard Deviation 6.46	3.23 T score Standard Deviation 5.99	—	—

SECONDARY outcome

Timeframe: Baseline, Month 6

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.

Outcome measures

Outcome measures
Measure	Cord Blood Infusion - 6 Months n=119 Participants 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	Placebo Infusion - 6 Months n=57 Participants 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	Cord Blood Infusion - 12 Months 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	Placebo Infusion - 12 Months 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
Change in PDD-BI Expressive Language T-Score	2.03 T score Standard Deviation 4.92	1.86 T score Standard Deviation 5.07	—	—

SECONDARY outcome

Timeframe: Baseline, Month 6

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.

Outcome measures

Outcome measures
Measure	Cord Blood Infusion - 6 Months n=119 Participants 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	Placebo Infusion - 6 Months n=57 Participants 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	Cord Blood Infusion - 12 Months 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	Placebo Infusion - 12 Months 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
Change in PDD-BI Learning, Memory, and Receptive Language T-Score	2.02 T score Standard Deviation 5.45	1.05 T score Standard Deviation 5.37	—	—

SECONDARY outcome

Timeframe: 12 months

Outcome measures

Outcome measures
Measure	Cord Blood Infusion - 6 Months n=61 Participants 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	Placebo Infusion - 6 Months n=119 Participants 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	Cord Blood Infusion - 12 Months 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	Placebo Infusion - 12 Months 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
Number of Participants With Infusion Reactions	2 Participants	1 Participants	—	—

SECONDARY outcome

Timeframe: 12 months

Grade/severity will be assessed according to CTCAE v4.0 guidelines

Outcome measures

Outcome measures
Measure	Cord Blood Infusion - 6 Months n=61 Participants 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	Placebo Infusion - 6 Months n=119 Participants 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	Cord Blood Infusion - 12 Months 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	Placebo Infusion - 12 Months 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
Severity of Infusion Reactions Mild	2 Participants	0 Participants	—	—
Severity of Infusion Reactions Moderate	0 Participants	1 Participants	—	—
Severity of Infusion Reactions Severe	0 Participants	0 Participants	—	—

SECONDARY outcome

Timeframe: 12 months

Outcome measures

Outcome measures
Measure	Cord Blood Infusion - 6 Months n=61 Participants 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	Placebo Infusion - 6 Months n=119 Participants 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	Cord Blood Infusion - 12 Months 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	Placebo Infusion - 12 Months 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
Number of Participants With Product-related Infections	0 Participants	0 Participants	—	—

SECONDARY outcome

Timeframe: 12 months

Population: There were no product-related infections in the study.

Grade/severity will be assessed according to CTCAE v4.0 guidelines

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 12 months

Outcome measures

Outcome measures
Measure	Cord Blood Infusion - 6 Months n=61 Participants 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	Placebo Infusion - 6 Months n=119 Participants 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	Cord Blood Infusion - 12 Months 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	Placebo Infusion - 12 Months 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
Evidence of Alloimmunization Via Anti-HLA (Human Leukocyte Antigen) and Anti-RBC (Red Blood Cell) Antibodies and Nonspecific Markers of Systemic Inflammation (ESR, CRP)	0 Participants	0 Participants	—	—

SECONDARY outcome

Timeframe: 12 months

Outcome measures

Outcome measures
Measure	Cord Blood Infusion - 6 Months n=61 Participants 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	Placebo Infusion - 6 Months n=119 Participants 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	Cord Blood Infusion - 12 Months 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	Placebo Infusion - 12 Months 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
Incidence of Graft vs. Host Disease	0 Participants	0 Participants	—	—

SECONDARY outcome

Timeframe: 12 months

Population: There were no cases of graft vs. host disease in the study.

Grade/severity will be assessed according to Common Terminology Criteria for Adverse Events (CTCAE) v4.0 guidelines.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 6 months,12 months

Outcome measures

Outcome measures
Measure	Cord Blood Infusion - 6 Months n=119 Participants 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	Placebo Infusion - 6 Months n=61 Participants 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	Cord Blood Infusion - 12 Months n=61 Participants 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	Placebo Infusion - 12 Months n=119 Participants 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
Incidence of Unexpected Adverse Events, by Relation to Study Product Related	0 Participants	0 Participants	0 Participants	0 Participants
Incidence of Unexpected Adverse Events, by Relation to Study Product Unrelated	5 Participants	1 Participants	0 Participants	2 Participants

SECONDARY outcome

Timeframe: 6 months, 12 months

Grade/severity will be assessed according to CTCAE v4.0 guidelines

Outcome measures

Outcome measures
Measure	Cord Blood Infusion - 6 Months n=119 Participants 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	Placebo Infusion - 6 Months n=61 Participants 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	Cord Blood Infusion - 12 Months n=61 Participants 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	Placebo Infusion - 12 Months n=119 Participants 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
Severity of Unexpected Adverse Events, by Relation to Study Product Mild : Related	0 Participants	0 Participants	0 Participants	0 Participants
Severity of Unexpected Adverse Events, by Relation to Study Product Mild : Unrelated	2 Participants	0 Participants	0 Participants	2 Participants
Severity of Unexpected Adverse Events, by Relation to Study Product Moderate : Related	0 Participants	0 Participants	0 Participants	0 Participants
Severity of Unexpected Adverse Events, by Relation to Study Product Moderate : Unrelated	3 Participants	1 Participants	0 Participants	0 Participants
Severity of Unexpected Adverse Events, by Relation to Study Product Severe : Related	0 Participants	0 Participants	0 Participants	0 Participants
Severity of Unexpected Adverse Events, by Relation to Study Product Severe : Unrelated	0 Participants	0 Participants	0 Participants	0 Participants

Adverse Events

Cord Blood Infusion - 6 Months

Serious events: 3 serious events

Other events: 97 other events

Deaths: 0 deaths

Placebo Infusion - 6 Months

Serious events: 3 serious events

Other events: 51 other events

Deaths: 0 deaths

Cord Blood Infusion - 12 Months

Serious events: 3 serious events

Other events: 41 other events

Deaths: 0 deaths

Placebo Infusion - 12 Months

Serious events: 2 serious events

Other events: 83 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Cord Blood Infusion - 6 Months n=119 participants at risk 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	Placebo Infusion - 6 Months n=61 participants at risk 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	Cord Blood Infusion - 12 Months n=61 participants at risk 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	Placebo Infusion - 12 Months n=119 participants at risk 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
Infections and infestations Gastroenteritis	0.00% 0/119 • 6 months and 12 months 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.	1.6% 1/61 • 6 months and 12 months 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.	1.6% 1/61 • 6 months and 12 months 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.	0.00% 0/119 • 6 months and 12 months 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.
Metabolism and nutrition disorders Dehydration	0.84% 1/119 • 6 months and 12 months 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.	1.6% 1/61 • 6 months and 12 months 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.	1.6% 1/61 • 6 months and 12 months 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.	0.84% 1/119 • 6 months and 12 months 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.
Psychiatric disorders Aggression	0.00% 0/119 • 6 months and 12 months 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.	1.6% 1/61 • 6 months and 12 months 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.	1.6% 1/61 • 6 months and 12 months 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.	0.00% 0/119 • 6 months and 12 months 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.
Psychiatric disorders Pediatric autoimmune neuropsychiatric disorders associated with streptococcal infection	0.84% 1/119 • 6 months and 12 months 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.	0.00% 0/61 • 6 months and 12 months 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.	0.00% 0/61 • 6 months and 12 months 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.	0.00% 0/119 • 6 months and 12 months 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.
Injury, poisoning and procedural complications Concussion	0.84% 1/119 • 6 months and 12 months 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.	0.00% 0/61 • 6 months and 12 months 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.	0.00% 0/61 • 6 months and 12 months 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.	0.84% 1/119 • 6 months and 12 months 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.

Other adverse events

Additional Information

Jesse D. Troy, PhD, MPH

Duke University

Phone: 919-668-1102

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place