Clinical trial NCT01988584: Safety and Effectiveness of Banked Cord Blood or Bone Marrow Stem Cells in Children With Cerebral Palsy (CP).
Official title: Autologous Cell Therapies for Cerebral Palsy-Chronic (ACT for CP)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Cerebral Palsy
Keywords: Cerebral Palsy
Brain Injury
Stem Cells
Mononuclear Cells
Bone Marrow
Umbilical Cord Blood
Lead sponsor: The University of Texas Health Science Center, Houston
Sponsor class: OTHER
Collaborators: name: Cord Blood Registry, Inc.
name: Let's Cure CP Foundation
name: Mission Connect, a program of TIRR Foundation
First posted: 2013-11-20
Enrollment: 20
Sex: ALL
Minimum age (years): 2
Maximum age (years): 10
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: CROSSOVER
Primary purpose: TREATMENT

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.


Eligibility criteria: 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.

Primary outcomes:
measure: Safety as Assessed by Number of Participants With In-hospital Infusion Toxicity
timeFrame: 24 hours after infusion
measure: Long-term Safety
timeFrame: from the time of infusion to 1 year after infusion
Secondary outcomes:
measure: Number of Participants With an Improvement in White Matter Integrity.
timeFrame: from baseline to 1 year after infusion
measure: Gross Motor Function Classification Score (GMFM-66)
timeFrame: baseline before infusion
measure: Gross Motor Function Classification Score (GMFM-66)
timeFrame: 1 year after infusion
measure: Gross Motor Function Classification Score (GMFM-88)
timeFrame: baseline before infusion
measure: Gross Motor Function Classification Score (GMFM-88)
timeFrame: 1 year after infusion
measure: Score on Vineland Adaptive Behavior Scales (VABS-2) - Communication
timeFrame: baseline before infusion
measure: Score on Vineland Adaptive Behavior Scales (VABS-2) - Communication
timeFrame: 1 year after infusion
measure: Score on Vineland Adaptive Behavior Scales (VABS-2) - Daily Living
timeFrame: baseline before infusion
measure: Score on Vineland Adaptive Behavior Scales (VABS-2) - Daily Living
timeFrame: 1 year after infusion
measure: Score on Vineland Adaptive Behavior Scales (VABS-2) - Social
timeFrame: baseline before infusion
measure: Score on Vineland Adaptive Behavior Scales (VABS-2) - Social
timeFrame: 1 year after infusion
measure: Score on Vineland Adaptive Behavior Scales (VABS-2) - Motor
timeFrame: baseline before infusion
measure: Score on Vineland Adaptive Behavior Scales (VABS-2) - Motor
timeFrame: 1 year after infusion
measure: Score on Pediatric Evaluation of Disability Inventory - Self-Care
timeFrame: baseline before infusion
measure: Score on Pediatric Evaluation of Disability Inventory - Self-Care
timeFrame: 1 year after infusion
measure: Score on Pediatric Evaluation of Disability Inventory - Mobility
timeFrame: baseline before infusion
measure: Score on Pediatric Evaluation of Disability Inventory - Mobility
timeFrame: 1 year after infusion
measure: Score on Pediatric Evaluation of Disability Inventory - Social
timeFrame: baseline before infusion
measure: Score on Pediatric Evaluation of Disability Inventory - Social
timeFrame: 1 year after infusion
measure: Score on A Developmental NEuroPSYchological Assessment (NEPSY-II) - Learning and Memory Memory Subtest - Recall
timeFrame: baseline before infusion
measure: Score on A Developmental NEuroPSYchological Assessment (NEPSY-II) - Learning and Memory Memory Subtest - Recall
timeFrame: 1 year after infusion
measure: Score on A Developmental NEuroPSYchological Assessment (NEPSY-II) - Learning and Memory Memory Subtest - Free/Cued
timeFrame: baseline before infusion
measure: Score on A Developmental NEuroPSYchological Assessment (NEPSY-II) - Learning and Memory Memory Subtest - Free/Cued
timeFrame: 1 year after infusion
measure: Expressive and Receptive Vocabulary as Assessed by Score on the Peabody Picture Vocabulary Test (PPVT-4)
timeFrame: baseline before infusion
measure: Expressive and Receptive Vocabulary as Assessed by Score on the Peabody Picture Vocabulary Test (PPVT-4)
timeFrame: 1 year after infusion
measure: Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Family
timeFrame: baseline before infusion
measure: Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Family
timeFrame: 1 year after infusion
measure: Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Social
timeFrame: baseline before infusion
measure: Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Social
timeFrame: 1 year after infusion
measure: Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Feelings
timeFrame: baseline before infusion
measure: Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Feelings
timeFrame: 1 year after infusion
measure: Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Participation
timeFrame: baseline before infusion
measure: Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Participation
timeFrame: 1 year after infusion
measure: Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Emotional
timeFrame: baseline before infusion
measure: Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Emotional
timeFrame: 1 year after infusion
measure: Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Access
timeFrame: baseline before infusion
measure: Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Access
timeFrame: 1 year after infusion
measure: Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Pain
timeFrame: baseline before infusion
measure: Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Pain
timeFrame: 1 year after infusion
measure: Visual-Spatial Processing as Assessed by Score on the Motor-Free Visual Perception Test (MVPT-3)
timeFrame: baseline before infusion
measure: Visual-Spatial Processing as Assessed by Score on the Motor-Free Visual Perception Test (MVPT-3)
timeFrame: 1 year after infusion
Other outcomes:
Locations:
city: Houston
country: United States
facility: UTHealth, Medical School, Dept. of Pediatric Surgery
state: Texas
Study officials:
affiliation: UTHealth, Medical School, Dept. of Pediatric Surgery
name: Charles S Cox, MD
role: PRINCIPAL_INVESTIGATOR
References: