Cerebral Palsy
Conditions
Keywords
Cerebral Palsy, Brain Injury, Stem Cells, Mononuclear Cells, Bone Marrow, Umbilical Cord Blood
Brief summary
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.
Interventions
BIOLOGICALumbilical cord blood (hUCB) cells
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.
BIOLOGICALbone marrow derived mononuclear cells (BMMNCs)
Autologous stem cells from bone marrow harvest.
Sponsors
Cord Blood Registry, Inc.
Let's Cure CP Foundation
Mission Connect, a program of TIRR Foundation
The University of Texas Health Science Center, Houston
Study design
Allocation
RANDOMIZED
Intervention model
CROSSOVER
Primary purpose
TREATMENT
Masking
TRIPLE (Subject, Caregiver, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
2 Years to 10 Years
Healthy volunteers
No
Inclusion criteria
1. Children with diagnosis of Cerebral Palsy (spastic CP due to periventricular white matter damage or neonatal brain injury from perinatal stroke or intra-ventricular hemorrhage) 2. Gross Motor Function Classification Score level II-V 3. Ages 24 months to 10 years 4. English speaking, if verbal 5. Ability to travel to Houston for treatment and follow-up -
Exclusion criteria
1. Known history of: * Intractable seizures * Traumatic brain injury * Genetic disorder (as demonstrated by newborn screening or genetic diagnostic testing) * Recently treated or current infection * Renal insufficiency or altered renal function (as defined by serum creatinine \> 1.5 mg/dl at screening) * Hepatic disease or altered liver function (as defined by SGPT \> 150 U/L \[non-contusion related\], and/or T. Bilirubin \>1.3 mg/dL at screening) * HIV+ (as demonstrated by positive blood test) * Immunosuppression (as defined by WBC \<3,000 cells/ml at screening) * Infectious related neurological injury * Sensitivity to Ethylene Oxide (EtO) \[found in fumigants and disinfectants\] 2. If Athetoid CP diagnosis, other etiologies such as degenerative, mitochondrial, and metabolic disorders must be excluded, and the outcome assessments must be able to be conducted to assess for potential treatment effects 3. Normal brain MRI 4. Evidence of acute illness at the time of infusion, such as, but not limited to, fever (temperature \> 37.5 C), vomiting, diarrhea, wheezing or crackles 5. Progressing neurological disease (as defined by Batten Disease, Leukodystrophies, Metabolic disorders, Mitochondrial disorders, Neurotransmitter disorders) 6. Microcephaly, macrocephaly, cortical malformations, genetic disorders of dysgenesis brain malformations due to infection or metabolic disorders 7. Pulmonary disease requiring ventilator support 8. If hUCB candidate, banked cord cells totaling \<10 million/kg 9. If hUCB candidate, any positive maternal infectious disease test (Hepatitis A, Hepatitis B, HIV 1, HIV 2, HTLV 1, HTLV 2, and Syphilis) 10. If hUCB candidate, cord blood sample contamination 11. Participation in a concurrent intervention study 12. Unwillingness to return for follow-up visits 13. Contraindications to MRI 14. Any patient that the investigators feel in their opinion the study intervention is unlikely to benefit the patient will be a screen failure. 15. Any patients who are currently or has previously been enrolled in a clinical stem cell study.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Safety as Assessed by Number of Participants With In-hospital Infusion Toxicity | 24 hours after infusion | In-hospital infusion toxicity includes hemodynamic, pulmonary, hepatic, renal, or neurologic complications. |
| Long-term Safety | from the time of infusion to 1 year after infusion | Long-term safety as assessed by number of participants who developed new mass lesions or other pathological structural changes or had worsening neurological status |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Gross Motor Function Classification Score (GMFM-88) | baseline before infusion | 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. |
| Score on A Developmental NEuroPSYchological Assessment (NEPSY-II) - Learning and Memory Memory Subtest - Free/Cued | baseline before infusion | Scales scores from 1 to 19 with higher scores indicate better outcomes and lower scores indicate poorer outcomes. |
| Score on Vineland Adaptive Behavior Scales (VABS-2) - Communication | baseline before infusion | 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. |
| Score on Vineland Adaptive Behavior Scales (VABS-2) - Daily Living | baseline before infusion | 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. |
| Score on Vineland Adaptive Behavior Scales (VABS-2) - Social | baseline before infusion | 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. |
| Score on Vineland Adaptive Behavior Scales (VABS-2) - Motor | baseline before infusion | 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. |
| Score on Pediatric Evaluation of Disability Inventory - Self-Care | baseline before infusion | 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. |
| Score on Pediatric Evaluation of Disability Inventory - Mobility | baseline before infusion | 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. |
| Score on Pediatric Evaluation of Disability Inventory - Social | baseline before infusion | 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. |
| Number of Participants With an Improvement in White Matter Integrity. | from baseline to 1 year after infusion | 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. |
| Expressive and Receptive Vocabulary as Assessed by Score on the Peabody Picture Vocabulary Test (PPVT-4) | baseline before infusion | 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. |
| Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Family | baseline before infusion | 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. |
| Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Social | baseline before infusion | 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. |
| Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Feelings | baseline before infusion | 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. |
| Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Participation | baseline before infusion | 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. |
| Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Emotional | baseline before infusion | 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. |
| Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Access | baseline before infusion | 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. |
| Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Pain | baseline before infusion | 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. |
| Visual-Spatial Processing as Assessed by Score on the Motor-Free Visual Perception Test (MVPT-3) | baseline before infusion | Standard scores ranging from 55 to 145 with higher scores indicating better outcomes and lower scores poorer outcomes. |
| Score on A Developmental NEuroPSYchological Assessment (NEPSY-II) - Learning and Memory Memory Subtest - Recall | baseline before infusion | Scales scores from 1 to 19 with higher scores indicate better outcomes and lower scores indicate poorer outcomes. |
| Gross Motor Function Classification Score (GMFM-66) | baseline before infusion | Interval age adjusted scaled scores from 0 to 100 with 0 reflecting a child with low motor ability and 100 indicating high motor ability. |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Umbilical Cord Blood (UCB) Cells Children who have banked UCB with CBR will receive an umbilical cord blood stem cell infusion at the baseline/treatment visit.
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry. | 5 |
| Bone Marrow-derived Mononuclear Cells (BMMNCs) Children in the BMMNC group will undergo bone marrow harvest and stem cell infusion at the baseline/treatment visit.
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest. | 15 |
| Total | 20 |
Baseline characteristics
| Characteristic | Umbilical Cord Blood (UCB) Cells | Bone Marrow-derived Mononuclear Cells (BMMNCs) | Total |
|---|---|---|---|
| Age, Categorical <=18 years | 5 Participants | 15 Participants | 20 Participants |
| Age, Categorical >=65 years | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical Between 18 and 65 years | 0 Participants | 0 Participants | 0 Participants |
| Age, Continuous | 4 years STANDARD_DEVIATION 2.12 | 6.13 years STANDARD_DEVIATION 2.92 | 5.6 years STANDARD_DEVIATION 2.85 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 1 Participants | 0 Participants | 1 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 4 Participants | 15 Participants | 19 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants | 2 Participants | 2 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) More than one race | 1 Participants | 0 Participants | 1 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) White | 4 Participants | 13 Participants | 17 Participants |
| Region of Enrollment Italy | 0 Participants | 1 Participants | 1 Participants |
| Region of Enrollment United Kingdom | 0 Participants | 1 Participants | 1 Participants |
| Region of Enrollment United States | 5 Participants | 13 Participants | 18 Participants |
| Sex: Female, Male Female | 2 Participants | 6 Participants | 8 Participants |
| Sex: Female, Male Male | 3 Participants | 9 Participants | 12 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk |
|---|---|---|---|
| deaths Total, all-cause mortality | 0 / 5 | 0 / 15 | 0 / 7 |
| other Total, other adverse events | 1 / 5 | 8 / 15 | 3 / 7 |
| serious Total, serious adverse events | 2 / 5 | 3 / 15 | 0 / 7 |
Outcome results
Primary
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
Population: Two in the BMMNC arm were lost to follow up.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Umbilical Cord Blood (UCB) Cells | Long-term Safety | 0 Participants |
| Bone Marrow-derived Mononuclear Cells (BMMNCs) | Long-term Safety | 0 Participants |
| Saline Infusion (Placebo) | Long-term Safety | 0 Participants |
Primary
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
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Umbilical Cord Blood (UCB) Cells | Safety as Assessed by Number of Participants With In-hospital Infusion Toxicity | 0 Participants |
| Bone Marrow-derived Mononuclear Cells (BMMNCs) | Safety as Assessed by Number of Participants With In-hospital Infusion Toxicity | 0 Participants |
| Saline Infusion (Placebo) | Safety as Assessed by Number of Participants With In-hospital Infusion Toxicity | 0 Participants |
Secondary
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
Population: Only 6 subjects were testable at this time point.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Umbilical Cord Blood (UCB) Cells | Expressive and Receptive Vocabulary as Assessed by Score on the Peabody Picture Vocabulary Test (PPVT-4) | 56.5 score on a scale | Standard Deviation 21.92 |
| Bone Marrow-derived Mononuclear Cells (BMMNCs) | Expressive and Receptive Vocabulary as Assessed by Score on the Peabody Picture Vocabulary Test (PPVT-4) | 86 score on a scale | Standard Deviation 11.58 |
Secondary
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
Population: Only 7 subjects were testable with this instrument.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Umbilical Cord Blood (UCB) Cells | Expressive and Receptive Vocabulary as Assessed by Score on the Peabody Picture Vocabulary Test (PPVT-4) | 80 score on a scale | Standard Deviation 26.87 |
| Bone Marrow-derived Mononuclear Cells (BMMNCs) | Expressive and Receptive Vocabulary as Assessed by Score on the Peabody Picture Vocabulary Test (PPVT-4) | 85 score on a scale | Standard Deviation 13.34 |
| Saline Infusion (Placebo) | Expressive and Receptive Vocabulary as Assessed by Score on the Peabody Picture Vocabulary Test (PPVT-4) | 89 score on a scale | Standard Deviation 0 |
Secondary
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
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Umbilical Cord Blood (UCB) Cells | Gross Motor Function Classification Score (GMFM-66) | 43.17 score on a scale | Standard Deviation 19.8 |
| Bone Marrow-derived Mononuclear Cells (BMMNCs) | Gross Motor Function Classification Score (GMFM-66) | 25.61 score on a scale | Standard Deviation 12.83 |
| Saline Infusion (Placebo) | Gross Motor Function Classification Score (GMFM-66) | 23.24 score on a scale | Standard Deviation 15.15 |
Secondary
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
Population: Two in the BMMNC arm were lost to follow up.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Umbilical Cord Blood (UCB) Cells | Gross Motor Function Classification Score (GMFM-66) | 36.5 score on a scale | Standard Deviation 24.1 |
| Bone Marrow-derived Mononuclear Cells (BMMNCs) | Gross Motor Function Classification Score (GMFM-66) | 25.95 score on a scale | Standard Deviation 13.88 |
Secondary
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
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Umbilical Cord Blood (UCB) Cells | Gross Motor Function Classification Score (GMFM-88) | 50.67 score on a scale | Standard Deviation 37.5 |
| Bone Marrow-derived Mononuclear Cells (BMMNCs) | Gross Motor Function Classification Score (GMFM-88) | 24.72 score on a scale | Standard Deviation 20.06 |
| Saline Infusion (Placebo) | Gross Motor Function Classification Score (GMFM-88) | 18.06 score on a scale | Standard Deviation 21.56 |
Secondary
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
Population: Two in the BMMNC arm were lost to follow up.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Umbilical Cord Blood (UCB) Cells | Gross Motor Function Classification Score (GMFM-88) | 34.77 score on a scale | Standard Deviation 40.38 |
| Bone Marrow-derived Mononuclear Cells (BMMNCs) | Gross Motor Function Classification Score (GMFM-88) | 25.92 score on a scale | Standard Deviation 19.83 |
Secondary
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
Population: Two in the BMMNC arm were lost to follow up.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Umbilical Cord Blood (UCB) Cells | Number of Participants With an Improvement in White Matter Integrity. | 5 Participants |
| Bone Marrow-derived Mononuclear Cells (BMMNCs) | Number of Participants With an Improvement in White Matter Integrity. | 9 Participants |
| Saline Infusion (Placebo) | Number of Participants With an Improvement in White Matter Integrity. | 0 Participants |
Secondary
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
Population: Only 3 subjects were testable at this time point.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Bone Marrow-derived Mononuclear Cells (BMMNCs) | Score on A Developmental NEuroPSYchological Assessment (NEPSY-II) - Learning and Memory Memory Subtest - Free/Cued | 4 score on a scale | Standard Deviation 0 |
Secondary
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
Population: Only 4 subjects were testable at this time point.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Bone Marrow-derived Mononuclear Cells (BMMNCs) | Score on A Developmental NEuroPSYchological Assessment (NEPSY-II) - Learning and Memory Memory Subtest - Free/Cued | 5.67 score on a scale | Standard Deviation 1.53 |
| Saline Infusion (Placebo) | Score on A Developmental NEuroPSYchological Assessment (NEPSY-II) - Learning and Memory Memory Subtest - Free/Cued | 6 score on a scale | Standard Deviation 0 |
Secondary
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
Population: Data were not collected for the 5 in the UCB cell arm and 4 in the placebo arm. Untestable with this instrument.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Bone Marrow-derived Mononuclear Cells (BMMNCs) | Score on A Developmental NEuroPSYchological Assessment (NEPSY-II) - Learning and Memory Memory Subtest - Recall | 8.33 score on a scale | Standard Deviation 0.58 |
| Saline Infusion (Placebo) | Score on A Developmental NEuroPSYchological Assessment (NEPSY-II) - Learning and Memory Memory Subtest - Recall | 6 score on a scale | Standard Deviation 0 |
Secondary
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
Population: Only 4 subjects were testable at this time point.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Umbilical Cord Blood (UCB) Cells | Score on A Developmental NEuroPSYchological Assessment (NEPSY-II) - Learning and Memory Memory Subtest - Recall | 6 score on a scale | Standard Deviation 0 |
| Bone Marrow-derived Mononuclear Cells (BMMNCs) | Score on A Developmental NEuroPSYchological Assessment (NEPSY-II) - Learning and Memory Memory Subtest - Recall | 5 score on a scale | Standard Deviation 1 |
Secondary
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
Population: Only 8 subjects were testable with this instrument.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Umbilical Cord Blood (UCB) Cells | Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Access | 47.5 score on a scale | Standard Deviation 0 |
| Bone Marrow-derived Mononuclear Cells (BMMNCs) | Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Access | 63.75 score on a scale | Standard Deviation 10.31 |
| Saline Infusion (Placebo) | Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Access | 80.83 score on a scale | Standard Deviation 17.02 |
Secondary
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
Population: Two in the BMMNC arm were lost to follow up. Five subjects were untestable at this time point.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Umbilical Cord Blood (UCB) Cells | Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Access | 72.5 score on a scale | Standard Deviation 29.01 |
| Bone Marrow-derived Mononuclear Cells (BMMNCs) | Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Access | 65.5 score on a scale | Standard Deviation 10.26 |
Secondary
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
Population: Two in the BMMNC arm were lost to follow up. Five other subjects were untestable at this time point.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Umbilical Cord Blood (UCB) Cells | Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Emotional | 78.12 score on a scale | Standard Deviation 15.73 |
| Bone Marrow-derived Mononuclear Cells (BMMNCs) | Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Emotional | 70.83 score on a scale | Standard Deviation 8.56 |
Secondary
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
Population: Only 8 subjects were testable with this instrument.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Umbilical Cord Blood (UCB) Cells | Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Emotional | 85.42 score on a scale | Standard Deviation 0 |
| Bone Marrow-derived Mononuclear Cells (BMMNCs) | Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Emotional | 75.52 score on a scale | Standard Deviation 5.98 |
| Saline Infusion (Placebo) | Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Emotional | 69.44 score on a scale | Standard Deviation 6.7 |
Secondary
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
Population: Two in the BMMNC arm were lost to follow up.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Umbilical Cord Blood (UCB) Cells | Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Family | 79.69 score on a scale | Standard Deviation 22.89 |
| Bone Marrow-derived Mononuclear Cells (BMMNCs) | Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Family | 72.81 score on a scale | Standard Deviation 17.74 |
Secondary
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
Population: Only 7 subjects were testable with this instrument.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Umbilical Cord Blood (UCB) Cells | Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Family | 87.5 score on a scale | Standard Deviation 0 |
| Bone Marrow-derived Mononuclear Cells (BMMNCs) | Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Family | 75.78 score on a scale | Standard Deviation 15.39 |
| Saline Infusion (Placebo) | Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Family | 75 score on a scale | Standard Deviation 36.36 |
Secondary
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
Population: 13 subjects were testable with this instrument.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Bone Marrow-derived Mononuclear Cells (BMMNCs) | Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Feelings | 60.16 score on a scale | Standard Deviation 6.1 |
| Saline Infusion (Placebo) | Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Feelings | 48.96 score on a scale | Standard Deviation 2.76 |
Secondary
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
Population: Two in the BMMNC arm were lost to follow up. Five subjects were untestable.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Umbilical Cord Blood (UCB) Cells | Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Feelings | 67.01 score on a scale | Standard Deviation 26.85 |
| Bone Marrow-derived Mononuclear Cells (BMMNCs) | Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Feelings | 55.73 score on a scale | Standard Deviation 9.68 |
Secondary
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
Population: Only 7 subjects were testable with this instrument.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Umbilical Cord Blood (UCB) Cells | Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Pain | 3.13 score on a scale | Standard Deviation 0 |
| Bone Marrow-derived Mononuclear Cells (BMMNCs) | Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Pain | 40.63 score on a scale | Standard Deviation 20.53 |
| Saline Infusion (Placebo) | Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Pain | 35.94 score on a scale | Standard Deviation 19.88 |
Secondary
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
Population: Two in the BMMNC arm were lost to follow up. Eight subjects were untestable at this time point.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Umbilical Cord Blood (UCB) Cells | Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Pain | 22.39 score on a scale | Standard Deviation 20.82 |
| Bone Marrow-derived Mononuclear Cells (BMMNCs) | Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Pain | 34.38 score on a scale | Standard Deviation 21.26 |
Secondary
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
Population: Only 8 subjects were testable with this instrument.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Umbilical Cord Blood (UCB) Cells | Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Participation | 72.73 score on a scale | Standard Deviation 0 |
| Bone Marrow-derived Mononuclear Cells (BMMNCs) | Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Participation | 56.82 score on a scale | Standard Deviation 8.25 |
| Saline Infusion (Placebo) | Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Participation | 56.82 score on a scale | Standard Deviation 17.71 |
Secondary
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
Population: Two in the BMMNC arm were lost to follow up. Five subjects were untestable at this time point.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Umbilical Cord Blood (UCB) Cells | Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Participation | 69.88 score on a scale | Standard Deviation 18.57 |
| Bone Marrow-derived Mononuclear Cells (BMMNCs) | Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Participation | 55.81 score on a scale | Standard Deviation 8.74 |
Secondary
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
Population: Two in the BMMNC arm were lost to follow up. Five subjects were untestable.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Umbilical Cord Blood (UCB) Cells | Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Social | 87.7 score on a scale | Standard Deviation 8.67 |
| Bone Marrow-derived Mononuclear Cells (BMMNCs) | Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Social | 74.77 score on a scale | Standard Deviation 9.21 |
Secondary
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
Population: Only 8 subjects were testable with this instrument
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Umbilical Cord Blood (UCB) Cells | Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Social | 88.64 score on a scale | Standard Deviation 0 |
| Bone Marrow-derived Mononuclear Cells (BMMNCs) | Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Social | 75.57 score on a scale | Standard Deviation 9.75 |
| Saline Infusion (Placebo) | Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Social | 83.71 score on a scale | Standard Deviation 15.01 |
Secondary
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
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Umbilical Cord Blood (UCB) Cells | Score on Pediatric Evaluation of Disability Inventory - Mobility | 46.83 score on a scale | Standard Deviation 27.62 |
| Bone Marrow-derived Mononuclear Cells (BMMNCs) | Score on Pediatric Evaluation of Disability Inventory - Mobility | 33.31 score on a scale | Standard Deviation 11.91 |
| Saline Infusion (Placebo) | Score on Pediatric Evaluation of Disability Inventory - Mobility | 26.54 score on a scale | Standard Deviation 12.88 |
Secondary
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
Population: Two in the BMMNC arm were lost to follow up.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Umbilical Cord Blood (UCB) Cells | Score on Pediatric Evaluation of Disability Inventory - Mobility | 39.66 score on a scale | Standard Deviation 36.36 |
| Bone Marrow-derived Mononuclear Cells (BMMNCs) | Score on Pediatric Evaluation of Disability Inventory - Mobility | 34.16 score on a scale | Standard Deviation 14.8 |
Secondary
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
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Umbilical Cord Blood (UCB) Cells | Score on Pediatric Evaluation of Disability Inventory - Self-Care | 35.4 score on a scale | Standard Deviation 12.29 |
| Bone Marrow-derived Mononuclear Cells (BMMNCs) | Score on Pediatric Evaluation of Disability Inventory - Self-Care | 43.31 score on a scale | Standard Deviation 13.04 |
| Saline Infusion (Placebo) | Score on Pediatric Evaluation of Disability Inventory - Self-Care | 27.36 score on a scale | Standard Deviation 9.35 |
Secondary
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
Population: Two in the BMMNC arm were lost to follow up.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Umbilical Cord Blood (UCB) Cells | Score on Pediatric Evaluation of Disability Inventory - Self-Care | 36.28 score on a scale | Standard Deviation 13.97 |
| Bone Marrow-derived Mononuclear Cells (BMMNCs) | Score on Pediatric Evaluation of Disability Inventory - Self-Care | 37.13 score on a scale | Standard Deviation 16.35 |
Secondary
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
Population: Two in the BMMNC arm were lost to follow up.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Umbilical Cord Blood (UCB) Cells | Score on Pediatric Evaluation of Disability Inventory - Social | 44.54 score on a scale | Standard Deviation 9.29 |
| Bone Marrow-derived Mononuclear Cells (BMMNCs) | Score on Pediatric Evaluation of Disability Inventory - Social | 47.32 score on a scale | Standard Deviation 14.82 |
Secondary
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
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Umbilical Cord Blood (UCB) Cells | Score on Pediatric Evaluation of Disability Inventory - Social | 46.9 score on a scale | Standard Deviation 7.82 |
| Bone Marrow-derived Mononuclear Cells (BMMNCs) | Score on Pediatric Evaluation of Disability Inventory - Social | 52 score on a scale | Standard Deviation 12.02 |
| Saline Infusion (Placebo) | Score on Pediatric Evaluation of Disability Inventory - Social | 34.12 score on a scale | Standard Deviation 16.15 |
Secondary
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
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Umbilical Cord Blood (UCB) Cells | Score on Vineland Adaptive Behavior Scales (VABS-2) - Communication | 79 score on a scale | Standard Deviation 16.09 |
| Bone Marrow-derived Mononuclear Cells (BMMNCs) | Score on Vineland Adaptive Behavior Scales (VABS-2) - Communication | 67.56 score on a scale | Standard Deviation 21.04 |
| Saline Infusion (Placebo) | Score on Vineland Adaptive Behavior Scales (VABS-2) - Communication | 52.86 score on a scale | Standard Deviation 15.95 |
Secondary
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
Population: Two in the BMMNC arm were lost to follow up.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Umbilical Cord Blood (UCB) Cells | Score on Vineland Adaptive Behavior Scales (VABS-2) - Communication | 64.4 score on a scale | Standard Deviation 18.96 |
| Bone Marrow-derived Mononuclear Cells (BMMNCs) | Score on Vineland Adaptive Behavior Scales (VABS-2) - Communication | 62.23 score on a scale | Standard Deviation 20.79 |
Secondary
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
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Umbilical Cord Blood (UCB) Cells | Score on Vineland Adaptive Behavior Scales (VABS-2) - Daily Living | 67 score on a scale | Standard Deviation 16.09 |
| Bone Marrow-derived Mononuclear Cells (BMMNCs) | Score on Vineland Adaptive Behavior Scales (VABS-2) - Daily Living | 57.44 score on a scale | Standard Deviation 9.58 |
| Saline Infusion (Placebo) | Score on Vineland Adaptive Behavior Scales (VABS-2) - Daily Living | 49.14 score on a scale | Standard Deviation 8.95 |
Secondary
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
Population: Two in the BMMNC arm were lost to follow up.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Umbilical Cord Blood (UCB) Cells | Score on Vineland Adaptive Behavior Scales (VABS-2) - Daily Living | 63 score on a scale | Standard Deviation 23.16 |
| Bone Marrow-derived Mononuclear Cells (BMMNCs) | Score on Vineland Adaptive Behavior Scales (VABS-2) - Daily Living | 52.54 score on a scale | Standard Deviation 13.2 |
Secondary
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
Population: 9 subjects were untestable.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Umbilical Cord Blood (UCB) Cells | Score on Vineland Adaptive Behavior Scales (VABS-2) - Motor | 58.67 score on a scale | Standard Deviation 15.5 |
| Bone Marrow-derived Mononuclear Cells (BMMNCs) | Score on Vineland Adaptive Behavior Scales (VABS-2) - Motor | 43 score on a scale | Standard Deviation 11.75 |
| Saline Infusion (Placebo) | Score on Vineland Adaptive Behavior Scales (VABS-2) - Motor | 38.5 score on a scale | Standard Deviation 14.62 |
Secondary
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
Population: Two in the BMMNC arm were lost to follow up. Nine subjects were untestable at this time point.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Umbilical Cord Blood (UCB) Cells | Score on Vineland Adaptive Behavior Scales (VABS-2) - Motor | 52.33 score on a scale | Standard Deviation 16.8 |
| Bone Marrow-derived Mononuclear Cells (BMMNCs) | Score on Vineland Adaptive Behavior Scales (VABS-2) - Motor | 40.67 score on a scale | Standard Deviation 11.24 |
Secondary
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
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Umbilical Cord Blood (UCB) Cells | Score on Vineland Adaptive Behavior Scales (VABS-2) - Social | 83 score on a scale | Standard Deviation 13 |
| Bone Marrow-derived Mononuclear Cells (BMMNCs) | Score on Vineland Adaptive Behavior Scales (VABS-2) - Social | 68.89 score on a scale | Standard Deviation 19.59 |
| Saline Infusion (Placebo) | Score on Vineland Adaptive Behavior Scales (VABS-2) - Social | 57.71 score on a scale | Standard Deviation 7.13 |
Secondary
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
Population: Two in the BMMNC arm were lost to follow up. One BMMNC subject was untestable at this time point.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Umbilical Cord Blood (UCB) Cells | Score on Vineland Adaptive Behavior Scales (VABS-2) - Social | 65 score on a scale | Standard Deviation 16 |
| Bone Marrow-derived Mononuclear Cells (BMMNCs) | Score on Vineland Adaptive Behavior Scales (VABS-2) - Social | 60.75 score on a scale | Standard Deviation 16.84 |
Secondary
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
Population: Data were not collected for the 5 in the UCB cell arm.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Bone Marrow-derived Mononuclear Cells (BMMNCs) | Visual-Spatial Processing as Assessed by Score on the Motor-Free Visual Perception Test (MVPT-3) | 63 score on a scale | Standard Deviation 11.31 |
Secondary
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
Population: Only three subjects were testable with this instrument.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Umbilical Cord Blood (UCB) Cells | Visual-Spatial Processing as Assessed by Score on the Motor-Free Visual Perception Test (MVPT-3) | 55 score on a scale | Standard Deviation 0 |
| Bone Marrow-derived Mononuclear Cells (BMMNCs) | Visual-Spatial Processing as Assessed by Score on the Motor-Free Visual Perception Test (MVPT-3) | 55 score on a scale | Standard Deviation 0 |