The purpose of this study is to compare the safety and effectiveness of two types of stem cells,(either banked cord blood or bone marrow), in children between the ages of 2 to 10 years with CP. 15 children with banked cord blood at CBR and 15 children without banked cord blood will be enrolled into the study. The study involves one baseline/treatment visit and 3 follow-up visits at 6 months, 12 months, and 2 years. Five children in each group will be randomized to a placebo control group at the baseline/treatment visit. Parents will not be told if their child received stem cells or a placebo until the 12 month follow-up visit. At that time parents may elect to have their child receive the stem cell treatment; either bone marrow harvest or umbilical cord blood if banked with CBR. All study visits will be conducted at the UTHealth Medical School and Children's Memorial Hermann Hospital in Houston, Texas. As of 1/21/2014 we have met our enrollment limit for children without banked cord blood undergoing bone marrow harvest for stem cells.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Enrollment
20
Autologous umbilical cord blood banked with the Cord Blood Registry.
A total of 10 children (5 from each cohort) will be randomized to a placebo infusion at the baseline visit and then have the opportunity to cross-over to stem cell treatment at the 1yr. visit.
Autologous stem cells from bone marrow harvest.
UTHealth, Medical School, Dept. of Pediatric Surgery
Houston, Texas, United States
Safety as Assessed by Number of Participants With In-hospital Infusion Toxicity
In-hospital infusion toxicity includes hemodynamic, pulmonary, hepatic, renal, or neurologic complications.
Time frame: 24 hours after infusion
Long-term Safety
Long-term safety as assessed by number of participants who developed new mass lesions or other pathological structural changes or had worsening neurological status
Time frame: from the time of infusion to 1 year after infusion
Number of Participants With an Improvement in White Matter Integrity.
Number of participants with an improvement in white matter integrity as measured by diffusion tensor imaging (DTI) magnetic resonance imaging (MRI) reconstruction of corticospinal tract (CST) radial diffusivity (RD). Improvement is defined as radial diffusivity (RD) decreased in at least one corticospinal tract (CST) reconstruction.
Time frame: from baseline to 1 year after infusion
Gross Motor Function Classification Score (GMFM-66)
Interval age adjusted scaled scores from 0 to 100 with 0 reflecting a child with low motor ability and 100 indicating high motor ability.
Time frame: baseline before infusion
Gross Motor Function Classification Score (GMFM-66)
Interval age adjusted scaled scores from 0 to 100 with 0 reflecting a child with low motor ability and 100 indicating high motor ability.
Time frame: 1 year after infusion
Gross Motor Function Classification Score (GMFM-88)
Ordinal age adjusted scaled raw and percent scores from 0 to 100 with 0 reflecting a child with low motor ability and 100 indicating high motor ability.
Time frame: baseline before infusion
Gross Motor Function Classification Score (GMFM-88)
Ordinal age adjusted scaled raw and percent scores from 0 to 100 with 0 reflecting a child with low motor ability and 100 indicating high motor ability.
Time frame: 1 year after infusion
Score on Vineland Adaptive Behavior Scales (VABS-2) - Communication
Standard score ranging from 20 to 140 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.
Time frame: baseline before infusion
Score on Vineland Adaptive Behavior Scales (VABS-2) - Communication
Standard score ranging from 20 to 140 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.
Time frame: 1 year after infusion
Score on Vineland Adaptive Behavior Scales (VABS-2) - Daily Living
Standard score ranging from 20 to 140 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.
Time frame: baseline before infusion
Score on Vineland Adaptive Behavior Scales (VABS-2) - Daily Living
Standard score ranging from 20 to 140 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.
Time frame: 1 year after infusion
Score on Vineland Adaptive Behavior Scales (VABS-2) - Social
Standard score ranging from 20 to 140 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.
Time frame: baseline before infusion
Score on Vineland Adaptive Behavior Scales (VABS-2) - Social
Standard score ranging from 20 to 140 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.
Time frame: 1 year after infusion
Score on Vineland Adaptive Behavior Scales (VABS-2) - Motor
Standard score ranging from 20 to 140 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.
Time frame: baseline before infusion
Score on Vineland Adaptive Behavior Scales (VABS-2) - Motor
Standard score ranging from 20 to 140 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.
Time frame: 1 year after infusion
Score on Pediatric Evaluation of Disability Inventory - Self-Care
Standard scores ranging from 0 to 100 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.
Time frame: baseline before infusion
Score on Pediatric Evaluation of Disability Inventory - Self-Care
Standard scores ranging from 0 to 100 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.
Time frame: 1 year after infusion
Score on Pediatric Evaluation of Disability Inventory - Mobility
Standard scores ranging from 0 to 100 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.
Time frame: baseline before infusion
Score on Pediatric Evaluation of Disability Inventory - Mobility
Standard scores ranging from 0 to 100 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.
Time frame: 1 year after infusion
Score on Pediatric Evaluation of Disability Inventory - Social
Standard scores ranging from 0 to 100 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.
Time frame: baseline before infusion
Score on Pediatric Evaluation of Disability Inventory - Social
Standard scores ranging from 0 to 100 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.
Time frame: 1 year after infusion
Score on A Developmental NEuroPSYchological Assessment (NEPSY-II) - Learning and Memory Memory Subtest - Recall
Scales scores from 1 to 19 with higher scores indicate better outcomes and lower scores indicate poorer outcomes.
Time frame: baseline before infusion
Score on A Developmental NEuroPSYchological Assessment (NEPSY-II) - Learning and Memory Memory Subtest - Recall
Scales scores from 1 to 19 with higher scores indicate better outcomes and lower scores indicate poorer outcomes.
Time frame: 1 year after infusion
Score on A Developmental NEuroPSYchological Assessment (NEPSY-II) - Learning and Memory Memory Subtest - Free/Cued
Scales scores from 1 to 19 with higher scores indicate better outcomes and lower scores indicate poorer outcomes.
Time frame: baseline before infusion
Score on A Developmental NEuroPSYchological Assessment (NEPSY-II) - Learning and Memory Memory Subtest - Free/Cued
Scales scores from 1 to 19 with higher scores indicate better outcomes and lower scores indicate poorer outcomes.
Time frame: 1 year after infusion
Expressive and Receptive Vocabulary as Assessed by Score on the Peabody Picture Vocabulary Test (PPVT-4)
Standard score ranging from 20 to 160. Higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.
Time frame: baseline before infusion
Expressive and Receptive Vocabulary as Assessed by Score on the Peabody Picture Vocabulary Test (PPVT-4)
Standard score ranging from 20 to 160. Higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.
Time frame: 1 year after infusion
Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Family
Standard scores ranging from 0 to 100 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.
Time frame: baseline before infusion
Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Family
Standard scores ranging from 0 to 100 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.
Time frame: 1 year after infusion
Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Social
Standard scores ranging from 0 to 100 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.
Time frame: baseline before infusion
Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Social
Standard scores ranging from 0 to 100 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.
Time frame: 1 year after infusion
Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Feelings
Standard scores ranging from 0 to 100 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.
Time frame: baseline before infusion
Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Feelings
Standard scores ranging from 0 to 100 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.
Time frame: 1 year after infusion
Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Participation
Standard scores ranging from 0 to 100 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.
Time frame: baseline before infusion
Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Participation
Standard scores ranging from 0 to 100 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.
Time frame: 1 year after infusion
Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Emotional
Standard scores ranging from 0 to 100 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.
Time frame: baseline before infusion
Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Emotional
Standard scores ranging from 0 to 100 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.
Time frame: 1 year after infusion
Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Access
Standard scores ranging from 0 to 100 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.
Time frame: baseline before infusion
Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Access
Standard scores ranging from 0 to 100 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.
Time frame: 1 year after infusion
Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Pain
Standard scores ranging from 0 to 100 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.
Time frame: baseline before infusion
Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Pain
Standard scores ranging from 0 to 100 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.
Time frame: 1 year after infusion
Visual-Spatial Processing as Assessed by Score on the Motor-Free Visual Perception Test (MVPT-3)
Standard scores ranging from 55 to 145 with higher scores indicating better outcomes and lower scores poorer outcomes.
Time frame: baseline before infusion
Visual-Spatial Processing as Assessed by Score on the Motor-Free Visual Perception Test (MVPT-3)
Standard scores ranging from 55 to 145 with higher scores indicating better outcomes and lower scores poorer outcomes.
Time frame: 1 year after infusion
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