Phase 2
N=63
A Randomized Study of Autologous Umbilical Cord Blood Reinfusion in Children With Cerebral Palsy
Cerebral Palsy · CP · Spastic Cerebral Palsy
Bottom Line
View on ClinicalTrials.gov: NCT01147653 ↗Enrolled (actual)
63
Serious AEs
0.8%
Results posted
Jun 2017
Primary outcome: Primary: Change in Gross Motor Function Measure 66 (GMFM-66) Score — 7.5; 6.9 units on a scale — p=0.72
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Autologous UCB Reinfusion (Biological); Placebo (Other)
- Age
- Pediatric · 0+ yrs
- Sex
- All
- Sponsor
- Joanne Kurtzberg, MD
- Primary completion
- Mar 2016
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change in Gross Motor Function Measure 66 (GMFM-66) Score |
7.5; 6.9 | 0.72 |
| SECONDARY Change in Peabody Gross Motor Quotient From Baseline to Year 1 |
1; -0.5 | 0.39 |
| SECONDARY Change in IT-QOL Questionnaire Score |
4.5; 3; 0; 8; 0; 0 | 0.473 |
| SECONDARY Change in CP-QOL Score |
-13; 15; -8.5; 10; -3.5; 0 | 0.055 |
| SECONDARY Change in Whole Brain Connectivity Measured by Diffusion Tensor Magnetic Resonance Imaging (MRI) |
0.3; 0.2 | — |
| SECONDARY Change in Loes Score of Functional MRI From Baseline to Year 1 and From Year 1 to Year 2 |
— | — |
| SECONDARY Change in Bayley Scales of Infant and Toddler Development-III, Cognitive Composite From Baseline to Year 1 |
3.2; 8.1 | 0.290 |
| SECONDARY Change in Wechsler Preschool and Primary Scale of Intelligence (WPPSI) III Full Scale Intelligence Quotient (IQ) for Younger Children (Ages 2 Years & 6 Months to 3 Years & 11 Months) From Baseline to Year 1. |
14; -8 | — |
| SECONDARY Change in the Wechsler Intelligence Scale for Children (WISC-IV) From Baseline to Year 1. |
103 | — |
| SECONDARY Change in Cognitive Z-Score From Baseline to Year 1 |
0.2; 0.3 | 0.697 |
| SECONDARY Change in Assisting Hand Assessment (AHA) Score From Baseline to Year 1 |
5.4; 5.1 | 0.912 |
| SECONDARY Change in Pediatric Evaluation of Disability (PEDI) Self Care Score |
-3.7; -2.2 | 0.544 |
| SECONDARY Change in Pediatric Evaluation of Disability (PEDI) Mobility Score |
0; 0 | 0.76 |
| SECONDARY Change in Pediatric Evaluation of Disability (PEDI) Social Function Score |
-0.5; -1.3 | 0.76 |
| SECONDARY Change in Child Behavior Checklist (CBCL) Z-score Internalizing Problems From Baseline to Year 1 |
-0.6; 0.0 | 0.176 |
| SECONDARY Change in Child Behavior Checklist (CBCL) Z-score Externalizing Problems From Baseline to Year 1 |
-0.2; 0.1 | 0.099 |
| SECONDARY Change in Child Behavior Checklist (CBCL) Z-score Total Problems From Baseline to Year 1 |
-0.3; 0.1 | 0.131 |
| SECONDARY Change in Parental Distress From Baseline to Year 1 |
1.0; -3.6 | 0.478 |
| SECONDARY Change in Parent-Child Dysfunctional Interaction From Baseline to Year 1 |
0.0; -10.0 | 0.029 sig |
| SECONDARY Change in Difficult Child Score From Baseline to Year 1 |
-0.2; 6.9 | 0.310 |
| SECONDARY Modified Ashworth Scale at Baseline |
2; 3; 4; 3; 5; 3 | — |
| SECONDARY Modified Ashworth Scale at Year 1 |
1; 1; 3; 5; 6; 8 | 0.233 |
| SECONDARY Change in Bruininks-Oseretsky-2 Total Motor Composite From Baseline to Year 1 |
-1 | — |
| SECONDARY Parent Experience of Child Illness (PECI) |
— | — |
| SECONDARY Correlation Between Clinical Response and RNA Expression of Neural, Endothelial and Inflammatory Cytokines Measured by RNA Arrays in Cord Blood Cells Given to These Patients. |
— | — |
Summary
The purpose of this study is to determine the efficacy of a single intravenous infusion of autologous umbilical cord blood (UCB) for the treatment of pediatric patients with spastic cerebral palsy.
Eligibility Criteria
Inclusion Criteria
- Age ≥ 12 months and ≤ 6 years
- Diagnosis: Spastic cerebral palsy with diplegia, hemiplegia, or quadraplegia.
- Performance status:
Gross Motor Function (GMF) Classification Score levels II - IV or GMF Score leve I, age >/= 2 years Spastic hemiplegia: GMF Score II-IV or minimal functional capabilities in the affected upper extremity. A subject classified as GMFCS level I with significant upper extremity impairment will be eligible if the affected upper extremity is used as an assist only. An eligibility committee will meet to review the child's records and determine eligibility.
Bilateral hypotonic CP (diplegia or quadriplegia): GMF Score II-IV and an abnormal brain MRI suggestive of an acquired etiology (versus a genetic etiology or brain malformation).
- Autologous umbilical cord blood available at a private or public cord blood bank with a minimum total nucleated cell dose of ≥ 1 x 107 cells/kilogram.
- Parental consent.
Exclusion Criteria
- Athetoid cerebral palsy.
- Autism and autistic spectrum disorders without motor disability.
- Hypsarrhythmia.
- Intractable seizures causing epileptic encephalopathy.
- Evidence of a progressive neurologic disease.
- Known HIV or uncontrolled bacterial, fungal, or viral infections.
- Impaired renal or liver function as determined by serum creatinine >1.5mg/dL and/or total bilirubin >1.3mg/dL.
- Head circumference >3 standard deviations below the mean for age.
- Known genetic disease or phenotypic evidence of a genetic disease on physical examination.
- Concurrent genetic or acquired disease or comorbidity(ies) that could require a future allogeneic stem cell transplant.
- Requires ventilatory support, including home ventilator, CPAP, BiPAP, or supplemental oxygen.
- Patient's medical condition does not permit safe travel.
- Previously received any form of cellular therapy.
- Autologous umbilical cord blood unit has any of the following:
- Total nuclear cell dose < 1 x 107 cells/kilogram
- Positive maternal infectious disease markers (except CMV)
- Evidence of infectious contamination of the cord blood unit
- Lack of a test sample to confirm identity
- Evidence of a genetic disease
- Unable to obtain parental consent.
Data sourced from ClinicalTrials.gov (NCT01147653). 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.