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Safety and Effectiveness of Banked Cord Blood or Bone Marrow Stem Cells in Children With Cerebral Palsy (CP). (ACT for CP)

Primary Purpose

Cerebral Palsy

Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
umbilical cord blood (hUCB) cells
Saline Infusion (Placebo)
bone marrow derived mononuclear cells (BMMNCs)
Sponsored by
The University of Texas Health Science Center, Houston
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Cerebral Palsy focused on measuring Cerebral Palsy, Brain Injury, Stem Cells, Mononuclear Cells, Bone Marrow, Umbilical Cord Blood

Eligibility Criteria

2 Years - 10 Years (Child)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • UTHealth, Medical School, Dept. of Pediatric Surgery

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm Type

Experimental

Experimental

Experimental

Experimental

Arm Label

umbilical cord blood (UCB) cells

bone marrow-derived mononuclear cells (BMMNCs)

saline infusion (placebo), then umbilical cord blood (UCB) cells

saline infusion (placebo), then bone marrow-derived mononuclear cells (BMMNCs)

Arm Description

Children who have banked UCB with CBR will receive an umbilical cord blood stem cell infusion at the baseline/treatment visit.

Children in the BMMNC group will undergo bone marrow harvest and stem cell infusion at the baseline/treatment visit.

Five children in each group will be randomly assigned to receive an inactive substance (placebo) at the baseline/treatment visit. Parents will be given the opportunity to cross-over to either the umbilical cord blood or bone marrow harvest group at the one year visit.

Outcomes

Primary Outcome Measures

Safety as Assessed by Number of Participants With In-hospital Infusion Toxicity
In-hospital infusion toxicity includes hemodynamic, pulmonary, hepatic, renal, or neurologic complications.
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

Secondary Outcome Measures

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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.

Full Information

First Posted
November 4, 2013
Last Updated
October 27, 2022
Sponsor
The University of Texas Health Science Center, Houston
Collaborators
Cord Blood Registry (CBR), Let's Cure CP Foundation, Mission Connect, a program of TIRR Foundation
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1. Study Identification

Unique Protocol Identification Number
NCT01988584
Brief Title
Safety and Effectiveness of Banked Cord Blood or Bone Marrow Stem Cells in Children With Cerebral Palsy (CP).
Acronym
ACT for CP
Official Title
Autologous Cell Therapies for Cerebral Palsy-Chronic (ACT for CP)
Study Type
Interventional

2. Study Status

Record Verification Date
October 2022
Overall Recruitment Status
Completed
Study Start Date
November 2013 (Actual)
Primary Completion Date
February 21, 2018 (Actual)
Study Completion Date
February 21, 2018 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
The University of Texas Health Science Center, Houston
Collaborators
Cord Blood Registry (CBR), Let's Cure CP Foundation, Mission Connect, a program of TIRR Foundation

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Cerebral Palsy
Keywords
Cerebral Palsy, Brain Injury, Stem Cells, Mononuclear Cells, Bone Marrow, Umbilical Cord Blood

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Crossover Assignment
Masking
ParticipantCare ProviderOutcomes Assessor
Allocation
Randomized
Enrollment
20 (Actual)

8. Arms, Groups, and Interventions

Arm Title
umbilical cord blood (UCB) cells
Arm Type
Experimental
Arm Description
Children who have banked UCB with CBR will receive an umbilical cord blood stem cell infusion at the baseline/treatment visit.
Arm Title
bone marrow-derived mononuclear cells (BMMNCs)
Arm Type
Experimental
Arm Description
Children in the BMMNC group will undergo bone marrow harvest and stem cell infusion at the baseline/treatment visit.
Arm Title
saline infusion (placebo), then umbilical cord blood (UCB) cells
Arm Type
Experimental
Arm Description
Five children in each group will be randomly assigned to receive an inactive substance (placebo) at the baseline/treatment visit. Parents will be given the opportunity to cross-over to either the umbilical cord blood or bone marrow harvest group at the one year visit.
Arm Title
saline infusion (placebo), then bone marrow-derived mononuclear cells (BMMNCs)
Arm Type
Experimental
Intervention Type
Biological
Intervention Name(s)
umbilical cord blood (hUCB) cells
Other Intervention Name(s)
Autolgous Stem Cells
Intervention Description
Autologous umbilical cord blood banked with the Cord Blood Registry.
Intervention Type
Drug
Intervention Name(s)
Saline Infusion (Placebo)
Intervention Description
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.
Intervention Type
Biological
Intervention Name(s)
bone marrow derived mononuclear cells (BMMNCs)
Other Intervention Name(s)
Autologous Stem Cells
Intervention Description
Autologous stem cells from bone marrow harvest.
Primary Outcome Measure Information:
Title
Safety as Assessed by Number of Participants With In-hospital Infusion Toxicity
Description
In-hospital infusion toxicity includes hemodynamic, pulmonary, hepatic, renal, or neurologic complications.
Time Frame
24 hours after infusion
Title
Long-term Safety
Description
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
Secondary Outcome Measure Information:
Title
Number of Participants With an Improvement in White Matter Integrity.
Description
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
Title
Gross Motor Function Classification Score (GMFM-66)
Description
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
Title
Gross Motor Function Classification Score (GMFM-66)
Description
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
Title
Gross Motor Function Classification Score (GMFM-88)
Description
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
Title
Gross Motor Function Classification Score (GMFM-88)
Description
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
Title
Score on Vineland Adaptive Behavior Scales (VABS-2) - Communication
Description
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
Title
Score on Vineland Adaptive Behavior Scales (VABS-2) - Communication
Description
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
Title
Score on Vineland Adaptive Behavior Scales (VABS-2) - Daily Living
Description
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
Title
Score on Vineland Adaptive Behavior Scales (VABS-2) - Daily Living
Description
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
Title
Score on Vineland Adaptive Behavior Scales (VABS-2) - Social
Description
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
Title
Score on Vineland Adaptive Behavior Scales (VABS-2) - Social
Description
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
Title
Score on Vineland Adaptive Behavior Scales (VABS-2) - Motor
Description
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
Title
Score on Vineland Adaptive Behavior Scales (VABS-2) - Motor
Description
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
Title
Score on Pediatric Evaluation of Disability Inventory - Self-Care
Description
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
Title
Score on Pediatric Evaluation of Disability Inventory - Self-Care
Description
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
Title
Score on Pediatric Evaluation of Disability Inventory - Mobility
Description
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
Title
Score on Pediatric Evaluation of Disability Inventory - Mobility
Description
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
Title
Score on Pediatric Evaluation of Disability Inventory - Social
Description
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
Title
Score on Pediatric Evaluation of Disability Inventory - Social
Description
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
Title
Score on A Developmental NEuroPSYchological Assessment (NEPSY-II) - Learning and Memory Memory Subtest - Recall
Description
Scales scores from 1 to 19 with higher scores indicate better outcomes and lower scores indicate poorer outcomes.
Time Frame
baseline before infusion
Title
Score on A Developmental NEuroPSYchological Assessment (NEPSY-II) - Learning and Memory Memory Subtest - Recall
Description
Scales scores from 1 to 19 with higher scores indicate better outcomes and lower scores indicate poorer outcomes.
Time Frame
1 year after infusion
Title
Score on A Developmental NEuroPSYchological Assessment (NEPSY-II) - Learning and Memory Memory Subtest - Free/Cued
Description
Scales scores from 1 to 19 with higher scores indicate better outcomes and lower scores indicate poorer outcomes.
Time Frame
baseline before infusion
Title
Score on A Developmental NEuroPSYchological Assessment (NEPSY-II) - Learning and Memory Memory Subtest - Free/Cued
Description
Scales scores from 1 to 19 with higher scores indicate better outcomes and lower scores indicate poorer outcomes.
Time Frame
1 year after infusion
Title
Expressive and Receptive Vocabulary as Assessed by Score on the Peabody Picture Vocabulary Test (PPVT-4)
Description
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
Title
Expressive and Receptive Vocabulary as Assessed by Score on the Peabody Picture Vocabulary Test (PPVT-4)
Description
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
Title
Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Family
Description
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
Title
Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Family
Description
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
Title
Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Social
Description
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
Title
Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Social
Description
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
Title
Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Feelings
Description
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
Title
Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Feelings
Description
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
Title
Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Participation
Description
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
Title
Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Participation
Description
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
Title
Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Emotional
Description
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
Title
Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Emotional
Description
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
Title
Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Access
Description
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
Title
Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Access
Description
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
Title
Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Pain
Description
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
Title
Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Pain
Description
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
Title
Visual-Spatial Processing as Assessed by Score on the Motor-Free Visual Perception Test (MVPT-3)
Description
Standard scores ranging from 55 to 145 with higher scores indicating better outcomes and lower scores poorer outcomes.
Time Frame
baseline before infusion
Title
Visual-Spatial Processing as Assessed by Score on the Motor-Free Visual Perception Test (MVPT-3)
Description
Standard scores ranging from 55 to 145 with higher scores indicating better outcomes and lower scores poorer outcomes.
Time Frame
1 year after infusion

10. Eligibility

Sex
All
Minimum Age & Unit of Time
2 Years
Maximum Age & Unit of Time
10 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: 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) Gross Motor Function Classification Score level II-V Ages 24 months to 10 years English speaking, if verbal Ability to travel to Houston for treatment and follow-up - Exclusion Criteria: 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] 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 Normal brain MRI 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 Progressing neurological disease (as defined by Batten Disease, Leukodystrophies, Metabolic disorders, Mitochondrial disorders, Neurotransmitter disorders) Microcephaly, macrocephaly, cortical malformations, genetic disorders of dysgenesis brain malformations due to infection or metabolic disorders Pulmonary disease requiring ventilator support If hUCB candidate, banked cord cells totaling <10 million/kg If hUCB candidate, any positive maternal infectious disease test (Hepatitis A, Hepatitis B, HIV 1, HIV 2, HTLV 1, HTLV 2, and Syphilis) If hUCB candidate, cord blood sample contamination Participation in a concurrent intervention study Unwillingness to return for follow-up visits Contraindications to MRI Any patient that the investigators feel in their opinion the study intervention is unlikely to benefit the patient will be a screen failure. Any patients who are currently or has previously been enrolled in a clinical stem cell study.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Charles S Cox, MD
Organizational Affiliation
UTHealth, Medical School, Dept. of Pediatric Surgery
Official's Role
Principal Investigator
Facility Information:
Facility Name
UTHealth, Medical School, Dept. of Pediatric Surgery
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States

12. IPD Sharing Statement

Learn more about this trial

Safety and Effectiveness of Banked Cord Blood or Bone Marrow Stem Cells in Children With Cerebral Palsy (CP).

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