Trial · NCT01147653

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

NCT01147653, Phase 2, Completed, Joanne Kurtzberg, MD; results first posted 2017-06-12

3 sourcestrials-2026-11-12-0.5.0

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ConductScience Library. A Randomized Study of Autologous Umbilical Cord Blood Reinfusion in Children With Cerebral Palsy. Trials, record NCT01147653. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01147653

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: A Randomized Study of Autologous Umbilical Cord Blood Reinfusion in Children With Cerebral Palsy
NCT number: NCT01147653
Status: Completed
Phases: Phase 2
Enrollment: 63 participants, actual
Lead sponsor: Joanne Kurtzberg, MD
Conditions: Cerebral Palsy, CP, Spastic Cerebral Palsy
Interventions: Autologous UCB Reinfusion, Placebo
First posted: 2010-06-22
Last updated: 2026-05-06
Results first posted: 2017-06-12

Source ClinicalTrials.gov

Registered outcomes26

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Change in Gross Motor Function Measure 66 (GMFM-66) Score	Baseline to Year 1	Change in Gross Motor Function Measure 66 (GMFM-66) Score from Baseline to Year 1. The GMFM-66 is a clinical tool used to evaluate gross motor function in children with cerebral palsy and is scored using a propriety software program called the Gross Motor Ability Estimator (GMAE) that produces an interval level continuous score ranging from 0 to 100. Higher scores indicate better motor function. A negative change in GMFM-66 score indicates a reduction in motor function, a positive change indicates improvement in motor function, and zero indicates no change in motor function.
Secondary	Change in Peabody Gross Motor Quotient From Baseline to Year 1	Baseline to Year 1	The Peabody Developmental Motor Scales - Second Edition (PDMS-2) measures gross and fine motor skills in children from birth through five years of age. The Gross Motor Quotient score from the PDMS-2 was used in this study to evaluate gross motor function. The Gross Motor Quotient measures the ability to use large muscle systems for locomotion, maintain a stable posture when not moving, and throw/catch objects. The range of possible scores is 41 to 164. High scores indicate better gross motor function. Lower scores indicate less gross motor function ability. The change in Gross Motor Quotient from Baseline to Year 1 was evaluated in this study. Positive numbers indicate an increase in gross motor ability, negative numbers indicate decreases in gross motor function, and a zero indicates no change.
Secondary	Change in IT-QOL Questionnaire Score	Baseline to Year 1	The Infant and Toddler Quality of Life Questionnaire (IT-QOL) was utilized for children ages one to three years at study entry.This 97-item questionnaire is completed by the parents and covers 12 concepts related to the physical, mental, and social well being of the child and the impact of their illness on the family. Scores range from 0 (worst health) to 100 (best health). The change from Baseline to Year 1 is summarized here for each of the 12 items on the questionnaire. Negative values indicate a decline in quality of life over time, positive values indicate an improvement, and zero indicates no change.
Secondary	Change in CP-QOL Score	Baseline, Year 1	Children age four years or older at study entry were assessed using the disease-specific "CP-QOL Child" assessment tool as completed by a parent. The CP-QOL Child primary-caregiver proxy form is designed for children 4 - 12 with cerebral palsy and contains 66 items which assess physical, emotional, and social well being as well as access to services and acceptance by others. Scores are summarized in seven topic areas. Scores in each area range from 0 (worst health) to 100 (best health). The change score from Baseline to Year 1 is summarized here for each item. Negative scores indicated a decrease in quality of life, positive scores indicate an increase and zero indicates no change.
Secondary	Change in Whole Brain Connectivity Measured by Diffusion Tensor Magnetic Resonance Imaging (MRI)	Baseline to Year 1	Change in number of connections in the brain as measured by diffusion tensor magnetic resonance imaging (MRI). Changes in connectivity are normalized to white matter volume of the brain. A positive number indicates an increase in connections, a negative number indicates a decrease, and zero indicates no change. The number of connections is expressed in terms of 10e5. For example, a change of 1 indicates an increase of 1x10e5 or 100,000 connections.
Secondary	Change in Loes Score of Functional MRI From Baseline to Year 1 and From Year 1 to Year 2	Baseline, Year 1, Year 2	No data were collected from this procedure because enrolled subjects who were eligible to receive fMRI were unable to comply with the procedure.
Secondary	Change in Bayley Scales of Infant and Toddler Development-III, Cognitive Composite From Baseline to Year 1	Baseline to Year 1	Cognitive function was assessed in English-speaking study participants using one of three different tools depending on the age of the patient at the time of assessment: The Bayley Scales of Infant and Toddler Development, Third Edition (Bayley-III), the Wechsler Intelligence Scale for Children (WISC-IV), and the Wechsler Preschool and Primary Scale of Intelligence (WPPSI-III). Some patients were assessed with different tools at subsequent visits as they aged during the conduct of the trial. The Bayley-III is designed to assess developmental functioning of infants and toddlers. Scores for the Cognitive Composite range from 1 to 19 and results in the range of 8 to 12 are considered average. The outcome measure reported here is the change in Cognitive Composite between Baseline to Year 1. Positive numbers indicate increases in cognitive functioning, negative numbers indicate a decrease, and zero indicates no change.
Secondary	Change in Wechsler Preschool and Primary Scale of Intelligence (WPPSI) III Full Scale Intelligence Quotient (IQ) for Younger Children (Ages 2 Years & 6 Months to 3 Years & 11 Months) From Baseline to Year 1.	Baseline to Year 1	Cognitive function was assessed in English-speaking study participants using one of three different tools depending on the age of the patient at the time of assessment: The Bayley Scales of Infant and Toddler Development, Third Edition (Bayley-III), the Wechsler Intelligence Scale for Children (WISC-IV), and the Wechsler Preschool and Primary Scale of Intelligence (WPPSI-III). Some patients were assessed with different tools at subsequent visits as they aged during the conduct of the trial. There are two versions of the WPPSI-III for two different age ranges: 2 years \& 6 months to 3 years \& 11 months, and 4 years to 7 years \& 3 months. The Full Scale IQ is calculated for both age ranges and provides a continuous score with an average of 100 and a standard deviation of 15. Change from Baseline to Year 1 was evaluated, with positive numbers indicating an increase in cognitive ability, negative numbers indicating a decrease in cognitive ability, and zero indicating no change.
Secondary	Change in the Wechsler Intelligence Scale for Children (WISC-IV) From Baseline to Year 1.	Baseline to Year 1	Cognitive function was assessed in English-speaking study participants using one of three different tools depending on the age of the patient at the time of assessment: The Bayley Scales of Infant and Toddler Development, Third Edition (Bayley-III), the Wechsler Intelligence Scale for Children (WISC-IV), and the Wechsler Preschool and Primary Scale of Intelligence (WPPSI-III). Some patients were assessed with different tools at subsequent visits as they aged during the conduct of the trial. The WISC-IV is designed for children 6 years 0 months to 16 years 11 months. This study used the Full Scale IQ, which ranges from 45 to 155 with a mean of 100 and standard deviation of 15. Higher scores indicate stronger cognitive function. Scores between 90 and 110 are considered to be within the range of average IQ.
Secondary	Change in Cognitive Z-Score From Baseline to Year 1	Baseline to Year 1	Because patients in this study were evaluated with different cognitive assessments based on their age at the time of assessment (The Bayley-III Cognitive Composite, the WPSSI-III Full Scale IQ, and the WISC-IV Full Scale IQ Composite), with some patients being assessed using different tools at subsequent visits during the trial, a method for combining the assessments was employed to evaluate change in cognitive function over time in as many patients as possible. A cognitive Z-score was calculated for each participant at Baseline and Year 1 by adjusting each score by the relevant assessments' population mean and standard deviation. The Z-scores represent the distance from the population mean, as measured by standard deviations. The analysis presented here summarizes the change in Z-score between Baseline and Year 1. A positive number indicates an increase in cognitive function, a negative number indicates a decrease, and zero indicates no change.

1 to 10 of 26Source ClinicalTrials.gov

See all 26 registered outcomes

Outcomes30

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Change in Gross Motor Function Measure 66 (GMFM-66) Score	Baseline to Year 1	All enrolled patients were analyzed as randomized from Baseline to Year 1.	units on a scale	MEAN
SECONDARY	Change in Peabody Gross Motor Quotient From Baseline to Year 1	Baseline to Year 1	Participants outside of the applicable age range for the PDMS-2 at Baseline or Year 1 were excluded from the analysis.	units on a scale	MEDIAN
SECONDARY	Change in IT-QOL Questionnaire Score	Baseline to Year 1	The analysis includes patients whose parents reported scores on each item at both Baseline and Year 1 and whose children met the age criteria for the questionnaire at both time points.	units on a scale	MEDIAN
SECONDARY	Change in CP-QOL Score	Baseline, Year 1	The analysis includes patients whose parents reported scores on each item at both Baseline and Year 1 and whose children met the age criteria for the questionnaire at both time points.	units on a scale	MEDIAN
SECONDARY	Change in Whole Brain Connectivity Measured by Diffusion Tensor Magnetic Resonance Imaging (MRI)	Baseline to Year 1	Patients without substantial morphologic brain abnormalities that prevented accurate anatomical image parcellation.	Number of connections x10e5	MEDIAN
SECONDARY	Change in Loes Score of Functional MRI From Baseline to Year 1 and From Year 1 to Year 2	Baseline, Year 1, Year 2	No data was available.
SECONDARY	Change in Bayley Scales of Infant and Toddler Development-III, Cognitive Composite From Baseline to Year 1	Baseline to Year 1	The Bayley III is an age-specific test and was used for 42 patients at Baseline. A total of 27 of these patients were also scored with the Bayley III at Year 1. The results here represent change from Baseline to Year 1 in the 27 participants assessed with the Bayley III at both time points.	units on a scale	MEAN
SECONDARY	Change in Wechsler Preschool and Primary Scale of Intelligence (WPPSI) III Full Scale Intelligence Quotient (IQ) for Younger Children (Ages 2 Years & 6 Months to 3 Years & 11 Months) From Baseline to Year 1.	Baseline to Year 1	Change scores were evaluable in only 2 subjects who were assessed with the WPPSI-III at both Baseline and Year 1. One subject was randomized to Autologous Cord Blood and the other to Placebo.	units on a scale	NUMBER
SECONDARY	Change in the Wechsler Intelligence Scale for Children (WISC-IV) From Baseline to Year 1.	Baseline to Year 1	Only one patient in this trial was evaluated with the WISC-IV and thus change over time could not be evaluated using this outcome measure. The single value for this patient is reported here.	Full Scale IQ Points	NUMBER
SECONDARY	Change in Cognitive Z-Score From Baseline to Year 1	Baseline to Year 1	When using all available cognitive assessments, the change in cognitive Z-score was available for 42 of the 63 participants.	Z scores	MEAN

1 to 10 of 30Source ClinicalTrials.gov

See all 30 outcomes

Results by group109

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Category	Value	Spread	Units
Change in Gross Motor Function Measure 66 (GMFM-66) Score	Autologous UCB Reinfusion First, Then Placebo		7.5	Standard Deviation 6.8	units on a scale
Change in Gross Motor Function Measure 66 (GMFM-66) Score	Placebo First, Then Autologous UCB Reinfusion		6.9	Standard Deviation 5.6	units on a scale
Change in Peabody Gross Motor Quotient From Baseline to Year 1	Autologous Umbilical Cord Blood Reinfusion		1	Full Range -10 to 20	units on a scale
Change in Peabody Gross Motor Quotient From Baseline to Year 1	Placebo First, Then Autologous UCB Reinfusion		-0.5	Full Range -19 to 15	units on a scale
Change in IT-QOL Questionnaire Score	Autologous UCB Reinfusion First,Then Placebo	Behavior	4.5	Full Range -13 to 20	units on a scale
Change in IT-QOL Questionnaire Score	Placebo First, Then Autologous UCB Reinfusion	Behavior	3	Full Range -15 to 20	units on a scale
Change in IT-QOL Questionnaire Score	Autologous UCB Reinfusion First,Then Placebo	Bodily Pain/Discomfort	0	Full Range -42 to 42	units on a scale
Change in IT-QOL Questionnaire Score	Placebo First, Then Autologous UCB Reinfusion	Bodily Pain/Discomfort	8	Full Range -25 to 25	units on a scale
Change in IT-QOL Questionnaire Score	Autologous UCB Reinfusion First,Then Placebo	Family Cohesion	0	Full Range -40 to 40	units on a scale
Change in IT-QOL Questionnaire Score	Placebo First, Then Autologous UCB Reinfusion	Family Cohesion	0	Full Range -25 to 25	units on a scale

1 to 10 of 109Source ClinicalTrials.gov

See all 109 results by group

Participant flow12

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Year 1	STARTED	Autologous UCB Reinfusion First, Then Placebo	32
Year 1	STARTED	Placebo First, Then Autologous UCB Reinfusion	31
Year 1	COMPLETED	Autologous UCB Reinfusion First, Then Placebo	32
Year 1	COMPLETED	Placebo First, Then Autologous UCB Reinfusion	31
Year 1	NOT COMPLETED	Autologous UCB Reinfusion First, Then Placebo	0
Year 1	NOT COMPLETED	Placebo First, Then Autologous UCB Reinfusion	0
Year 2	STARTED	Autologous UCB Reinfusion First, Then Placebo	32
Year 2	STARTED	Placebo First, Then Autologous UCB Reinfusion	31
Year 2	COMPLETED	Autologous UCB Reinfusion First, Then Placebo	30
Year 2	COMPLETED	Placebo First, Then Autologous UCB Reinfusion	28

1 to 10 of 12Source ClinicalTrials.gov

See all 12 participant flow

Adverse events66

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk	Events
other	Flushed in face with hypersensitivity rash on trunk. Sats 92%. No wheezing. Benadryl given	Immune system disorders	Autologous Umbilical Cord Blood Reinfusion	0	63	0
other	Flushed in face with hypersensitivity rash on trunk. Sats 92%. No wheezing. Benadryl given	Immune system disorders	Placebo	1	63
other	Tactile temp overnight. Left otitis media	Infections and infestations	Autologous Umbilical Cord Blood Reinfusion	0	63	0
other	Tactile temp overnight. Left otitis media	Infections and infestations	Placebo	1	63
other	Painful urination and spotting 1 wk post infusion.	Renal and urinary disorders	Autologous Umbilical Cord Blood Reinfusion	1	63
other	Painful urination and spotting 1 wk post infusion.	Renal and urinary disorders	Placebo	0	63	0
other	Admitted to ER with polyuria and polydipsia	Metabolism and nutrition disorders	Autologous Umbilical Cord Blood Reinfusion	1	63
other	Admitted to ER with polyuria and polydipsia	Metabolism and nutrition disorders	Placebo	0	63	0
other	Rash on abdomen and legs. No itching or welps. No treatment- resolved next day	Skin and subcutaneous tissue disorders	Autologous Umbilical Cord Blood Reinfusion	0	63	0
other	Rash on abdomen and legs. No itching or welps. No treatment- resolved next day	Skin and subcutaneous tissue disorders	Placebo	1	63

1 to 10 of 66Source ClinicalTrials.gov

See all 66 adverse events

Adverse event totals6

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Autologous Umbilical Cord Blood Reinfusion	0	63
Total, all-cause mortality	Placebo	0	63
Total, other adverse events	Autologous Umbilical Cord Blood Reinfusion	24	63
Total, other adverse events	Placebo	4	63
Total, serious adverse events	Autologous Umbilical Cord Blood Reinfusion	0	63
Total, serious adverse events	Placebo	1	63

Source ClinicalTrials.gov

Papers1

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as
29080265	DERIVED

Source PubMed

Grants0

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

None in this release.

Trial comparisons2

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

Comparison	Source
Autologous Umbilical Cord Blood Reinfusion vs Placebo: deaths from any cause	Trial comparisons
Autologous Umbilical Cord Blood Reinfusion vs Placebo: serious adverse events	Trial comparisons

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

Read this trial from software.

The record and each of its lists are JSON, twenty rows a page. No key.

GET https://conductscience.org/api/trials/v1/NCT01147653

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API reference For AI assistants