Phase 2
N=20
Safety and Effectiveness of Banked Cord Blood or Bone Marrow Stem Cells in Children With Cerebral Palsy (CP).
Cerebral Palsy
Bottom Line
View on ClinicalTrials.gov: NCT01988584 ↗Enrolled (actual)
20
Serious AEs
18.5%
Results posted
Nov 2022
Primary outcome: Primary: Safety as Assessed by Number of Participants With In-hospital Infusion Toxicity — 0; 0; 0 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- umbilical cord blood (hUCB) cells (Biological); Saline Infusion (Placebo) (Drug); bone marrow derived mononuclear cells (BMMNCs) (Biological)
- Age
- Pediatric · 2+ yrs
- Sex
- All
- Sponsor
- The University of Texas Health Science Center, Houston
- Primary completion
- Feb 2018
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Safety as Assessed by Number of Participants With In-hospital Infusion Toxicity |
0; 0; 0 | — |
| PRIMARY Long-term Safety |
0; 0; 0 | — |
| SECONDARY Number of Participants With an Improvement in White Matter Integrity. |
5; 9; 0 | — |
| SECONDARY Gross Motor Function Classification Score (GMFM-66) |
36.5; 25.95 | — |
| SECONDARY Gross Motor Function Classification Score (GMFM-66) |
36.5; 25.95 | — |
| SECONDARY Gross Motor Function Classification Score (GMFM-88) |
34.77; 25.92 | — |
| SECONDARY Gross Motor Function Classification Score (GMFM-88) |
34.77; 25.92 | — |
| SECONDARY Score on Vineland Adaptive Behavior Scales (VABS-2) - Communication |
64.4; 62.23 | — |
| SECONDARY Score on Vineland Adaptive Behavior Scales (VABS-2) - Communication |
64.4; 62.23 | — |
| SECONDARY Score on Vineland Adaptive Behavior Scales (VABS-2) - Daily Living |
63; 52.54 | — |
| SECONDARY Score on Vineland Adaptive Behavior Scales (VABS-2) - Daily Living |
63; 52.54 | — |
| SECONDARY Score on Vineland Adaptive Behavior Scales (VABS-2) - Social |
65; 60.75 | — |
| SECONDARY Score on Vineland Adaptive Behavior Scales (VABS-2) - Social |
65; 60.75 | — |
| SECONDARY Score on Vineland Adaptive Behavior Scales (VABS-2) - Motor |
52.33; 40.67 | — |
| SECONDARY Score on Vineland Adaptive Behavior Scales (VABS-2) - Motor |
52.33; 40.67 | — |
| SECONDARY Score on Pediatric Evaluation of Disability Inventory - Self-Care |
36.28; 37.13 | — |
| SECONDARY Score on Pediatric Evaluation of Disability Inventory - Self-Care |
36.28; 37.13 | — |
| SECONDARY Score on Pediatric Evaluation of Disability Inventory - Mobility |
39.66; 34.16 | — |
| SECONDARY Score on Pediatric Evaluation of Disability Inventory - Mobility |
39.66; 34.16 | — |
| SECONDARY Score on Pediatric Evaluation of Disability Inventory - Social |
44.54; 47.32 | — |
| SECONDARY Score on Pediatric Evaluation of Disability Inventory - Social |
44.54; 47.32 | — |
| SECONDARY Score on A Developmental NEuroPSYchological Assessment (NEPSY-II) - Learning and Memory Memory Subtest - Recall |
6; 5 | — |
| SECONDARY Score on A Developmental NEuroPSYchological Assessment (NEPSY-II) - Learning and Memory Memory Subtest - Recall |
6; 5 | — |
| SECONDARY Score on A Developmental NEuroPSYchological Assessment (NEPSY-II) - Learning and Memory Memory Subtest - Free/Cued |
4 | — |
| SECONDARY Score on A Developmental NEuroPSYchological Assessment (NEPSY-II) - Learning and Memory Memory Subtest - Free/Cued |
4 | — |
| SECONDARY Expressive and Receptive Vocabulary as Assessed by Score on the Peabody Picture Vocabulary Test (PPVT-4) |
56.5; 86 | — |
| SECONDARY Expressive and Receptive Vocabulary as Assessed by Score on the Peabody Picture Vocabulary Test (PPVT-4) |
56.5; 86 | — |
| SECONDARY Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Family |
79.69; 72.81 | — |
| SECONDARY Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Family |
79.69; 72.81 | — |
| SECONDARY Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Social |
87.7; 74.77 | — |
| SECONDARY Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Social |
87.7; 74.77 | — |
| SECONDARY Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Feelings |
67.01; 55.73 | — |
| SECONDARY Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Feelings |
67.01; 55.73 | — |
| SECONDARY Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Participation |
69.88; 55.81 | — |
| SECONDARY Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Participation |
69.88; 55.81 | — |
| SECONDARY Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Emotional |
78.12; 70.83 | — |
| SECONDARY Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Emotional |
78.12; 70.83 | — |
| SECONDARY Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Access |
72.5; 65.5 | — |
| SECONDARY Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Access |
72.5; 65.5 | — |
| SECONDARY Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Pain |
22.39; 34.38 | — |
| SECONDARY Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Pain |
22.39; 34.38 | — |
| SECONDARY Visual-Spatial Processing as Assessed by Score on the Motor-Free Visual Perception Test (MVPT-3) |
63 | — |
| SECONDARY Visual-Spatial Processing as Assessed by Score on the Motor-Free Visual Perception Test (MVPT-3) |
63 | — |
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
- 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 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.
Data sourced from ClinicalTrials.gov (NCT01988584). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.