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Completed

NCT Number: NCT01988584

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

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.

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Key information

Conditions

Cerebral Palsy Brain Damage, Chronic Brain Diseases Brain Injuries Central Nervous System Diseases Craniocerebral Trauma Nervous System Diseases Trauma, Nervous System Wounds and Injuries

Age range

2 year–10 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

UTHealth, Medical School, Dept. of Pediatric Surgery

Houston, Texas, 77030, United States

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

umbilical cord blood (hUCB) cells

Biological

Autologous umbilical cord blood banked with the Cord Blood Registry.

Other names: Autolgous Stem Cells

Saline infusion (placebo)

Drug

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.

bone marrow derived mononuclear cells (BMMNCs)

Biological

Autologous stem cells from bone marrow harvest.

Other names: Autologous Stem Cells

Primary outcomes

  1. Safety as Assessed by Number of Participants With In-hospital Infusion Toxicity

    Time frame: 24 hours after infusion

    In-hospital infusion toxicity includes hemodynamic, pulmonary, hepatic, renal, or neurologic complications.

  2. Long-term Safety

    Time frame: 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 outcomes

  1. Number of Participants With an Improvement in White Matter Integrity.

    Time frame: 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.

  2. Gross Motor Function Classification Score (GMFM-66)

    Time frame: 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.

  3. Gross Motor Function Classification Score (GMFM-66)

    Time frame: 1 year after 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.

  4. Gross Motor Function Classification Score (GMFM-88)

    Time frame: 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.

  5. Gross Motor Function Classification Score (GMFM-88)

    Time frame: 1 year after 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.

  6. Score on Vineland Adaptive Behavior Scales (VABS-2) - Communication

    Time frame: 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.

  7. Score on Vineland Adaptive Behavior Scales (VABS-2) - Communication

    Time frame: 1 year after 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.

  8. Score on Vineland Adaptive Behavior Scales (VABS-2) - Daily Living

    Time frame: 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.

  9. Score on Vineland Adaptive Behavior Scales (VABS-2) - Daily Living

    Time frame: 1 year after 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.

  10. Score on Vineland Adaptive Behavior Scales (VABS-2) - Social

    Time frame: 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.

  11. Score on Vineland Adaptive Behavior Scales (VABS-2) - Social

    Time frame: 1 year after 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.

  12. Score on Vineland Adaptive Behavior Scales (VABS-2) - Motor

    Time frame: 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.

  13. Score on Vineland Adaptive Behavior Scales (VABS-2) - Motor

    Time frame: 1 year after 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.

  14. Score on Pediatric Evaluation of Disability Inventory - Self-Care

    Time frame: 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.

  15. Score on Pediatric Evaluation of Disability Inventory - Self-Care

    Time frame: 1 year after 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.

  16. Score on Pediatric Evaluation of Disability Inventory - Mobility

    Time frame: 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.

  17. Score on Pediatric Evaluation of Disability Inventory - Mobility

    Time frame: 1 year after 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.

  18. Score on Pediatric Evaluation of Disability Inventory - Social

    Time frame: 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.

  19. Score on Pediatric Evaluation of Disability Inventory - Social

    Time frame: 1 year after 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.

  20. Score on A Developmental NEuroPSYchological Assessment (NEPSY-II) - Learning and Memory Memory Subtest - Recall

    Time frame: baseline before infusion

    Scales scores from 1 to 19 with higher scores indicate better outcomes and lower scores indicate poorer outcomes.

  21. Score on A Developmental NEuroPSYchological Assessment (NEPSY-II) - Learning and Memory Memory Subtest - Recall

    Time frame: 1 year after infusion

    Scales scores from 1 to 19 with higher scores indicate better outcomes and lower scores indicate poorer outcomes.

  22. Score on A Developmental NEuroPSYchological Assessment (NEPSY-II) - Learning and Memory Memory Subtest - Free/Cued

    Time frame: baseline before infusion

    Scales scores from 1 to 19 with higher scores indicate better outcomes and lower scores indicate poorer outcomes.

  23. Score on A Developmental NEuroPSYchological Assessment (NEPSY-II) - Learning and Memory Memory Subtest - Free/Cued

    Time frame: 1 year after infusion

    Scales scores from 1 to 19 with higher scores indicate better outcomes and lower scores indicate poorer outcomes.

  24. Expressive and Receptive Vocabulary as Assessed by Score on the Peabody Picture Vocabulary Test (PPVT-4)

    Time frame: 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.

  25. Expressive and Receptive Vocabulary as Assessed by Score on the Peabody Picture Vocabulary Test (PPVT-4)

    Time frame: 1 year after 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.

  26. Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Family

    Time frame: 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.

  27. Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Family

    Time frame: 1 year after 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.

  28. Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Social

    Time frame: 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.

  29. Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Social

    Time frame: 1 year after 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.

  30. Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Feelings

    Time frame: 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.

  31. Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Feelings

    Time frame: 1 year after 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.

  32. Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Participation

    Time frame: 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.

  33. Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Participation

    Time frame: 1 year after 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.

  34. Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Emotional

    Time frame: 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.

  35. Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Emotional

    Time frame: 1 year after 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.

  36. Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Access

    Time frame: 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.

  37. Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Access

    Time frame: 1 year after 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.

  38. Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Pain

    Time frame: 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.

  39. Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Pain

    Time frame: 1 year after 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.

  40. Visual-Spatial Processing as Assessed by Score on the Motor-Free Visual Perception Test (MVPT-3)

    Time frame: baseline before infusion

    Standard scores ranging from 55 to 145 with higher scores indicating better outcomes and lower scores poorer outcomes.

  41. Visual-Spatial Processing as Assessed by Score on the Motor-Free Visual Perception Test (MVPT-3)

    Time frame: 1 year after infusion

    Standard scores ranging from 55 to 145 with higher scores indicating better outcomes and lower scores poorer outcomes.

Sponsors and collaborators

Lead sponsor

The University of Texas Health Science Center, Houston

Other

Collaborators

  • Cord Blood Registry, Inc.
  • Let's Cure CP Foundation
  • Mission Connect, a program of TIRR Foundation

Registry information

Official study title

Autologous Cell Therapies for Cerebral Palsy-Chronic (ACT for CP)

Acronym: ACT for CP

Important dates

Study start
2013
Primary completion
2018
Study completion
2018
First posted
Nov 20, 2013
Registry last updated
Nov 21, 2022

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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