Cerebral Palsy
Conditions
Keywords
cerebral palsy, autologous umbilical cord blood
Brief summary
This study is a phase II, prospective, double blind, placebo-controlled study of the efficacy of autologous umbilical cord blood infusion. The study population will consist of 72 children ages 2 months to 12 years with cerebral palsy. The population will be randomly assigned to 2 groups, 36 children in each group. The study group be treated by cord blood in the beginning of the study and the control group by placebo product. The study population will be stratified to reduce variance 3 groups by age: 2-12 months / 1-6 years / 6-12 years The study will consist of 4 stages Stage 1: initial assessment by physiotherapist and occupational therapist / treatment by cord blood or placebo / blood work before and after treatment Stage 2: at stage 1 + 3 months assessment by physiotherapist and occupational therapist Stage 3: at stage 1 + 6 months assessment by physiotherapist and occupational therapist / cross-over treatment by cord blood or placebo / blood work before and after treatment Stage 4: at stage 1 + 12 months assessment by physiotherapist and occupational therapist The primary outcome is improvement motor skills six months after treatment at stage 3
Interventions
single dose of an autologous umbilical cord blood transfusion
OTHERplacebo
The placebo product will consist of the standard ingredients of the acellular content of the UCB unit. It will consist of 20 ml Dextran (Plander 40.000 - 50g/500ml, solution for infusion) and 20 ml of human Albumin 5% (solution for infusion). The volume of placebo product will be 40 ml
Sponsors
Sheba Medical Center
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Masking description
The randomization will be done by external source and the assignment will disclosed to cord bank bank only. They will produce either a cord blood or placebo unit which will be completely covered. Each unit will have its own index number that will be documented by the research coordinator. Neither the researcher or the family will know the nature of the unit.
Intervention model description
This study is a phase II, prospective, double blind, placebo-control study of the efficacy of autologous umbilical cord blood infusion. The study population will consist of 72 children ages 2 months to 12 years with cerebral palsy. The population will be randomly assigned to 2 groups, the study group be treated by cord blood in the beginning of the study and the control group by placebo product. The study population will be stratified to reduce variance 3 groups by age: 2-12 months / 1-6 years / 6-12 years The study will consist of 4 stages Stage 1: initial assessment by physiotherapist and occupational therapist / treatment by cord blood or placebo / blood work before and after treatment Stage 2: at stage 1 + 3 months assessment by physiotherapist and occupational therapist Stage 3: at stage 1 + 6 months assessment by physiotherapist and occupational therapist
Eligibility
Sex/Gender
ALL
Age
2 Months to 12 Years
Healthy volunteers
No
Inclusion criteria
* Age ≥ 2 month and ≤ 12 years. * Performance status for children older than 2 months and younger than 12 months including all the following (Gross Motor Function Classification Score is less indicative before 12 months): * Abnormal General Movements (in infants 2-6 months) * Abnormal Hammersmith Infant Neurological Examination * Performance status for children older than 12 months * Bilateral spastic cerebral palsy (diplegia or quadraplegia): Gross Motor Function Classification Score levels I - IV * Spastic hemiplegia: Gross Motor Function Classification Score levels I - IV. 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. * Bilateral hypotonic cerebral palsy (diplegia or quadraplegia): Gross Motor Function Classification Score levels I - IV. * An abnormal brain MRI suggestive of an acquired etiology (and not genetic etiology or brain malformation). * 4\. Autologous umbilical cord blood available at a private or public cord blood bank with a minimum total nucleated cell dose of ≥ 2 x 10e7 cells/kilogram. * 5\. Parental consent.
Exclusion criteria
* 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. * Requires ventilatory support, including home ventilator * Surgical procedure or botulinum toxin injection from 6 months prior to the study and during the time of the study * 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 \< 2 x 10e7 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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Motor developmental improvement | 6 months | Gross Motor Function Measurement - 66 (GMFM-66). All score are normalized with mean 100 and STD of 15. High score is better than low score . |
| Functional assessment | 6 months | Pediatric Evaluation of Disability Evaluation . Score 0-100 higher score better functionality Pediatric Evaluation of Disability Inventory-Computer Adaptive Test (PEDI-CAT) |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Functional assessment | 6 months | Adaptive Behavior Assessment System, Third Edition (ABAS-3). All score are normalized with mean 100 and STD of 10. High score is better than low score . |
Countries
Israel
Contacts
Primary ContactOmer Bar-Yosef, MD.PHD
Outcome results
None listed