Treatment of Children With Cerebral Palsy With Autologous Umbilical Cord Blood, a Pilot Study

Overview

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

Study Type

  • Study Type: Interventional
  • Study Design
    • Allocation: Randomized
    • Intervention Model: Parallel Assignment
    • Primary Purpose: Treatment
    • Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
  • Study Primary Completion Date: December 2024

Interventions

  • Other: Autologous umbilical cord blood transfusion
    • single dose of an autologous umbilical cord blood transfusion
  • Other: placebo
    • 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

Arms, Groups and Cohorts

  • Experimental: group 1
  • Placebo Comparator: group 2

Clinical Trial Outcome Measures

Primary Measures

  • Motor developmental improvement
    • Time Frame: 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 .
  • Motor developmental improvement
    • Time Frame: 6 months
    • Peabody developmental motor scales-second edition (PDMS-2). All score are normalized with mean 100 and STD 10. High score is better
  • Functional assessment
    • Time Frame: 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 Measures

  • Functional assessment
    • Time Frame: 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 .
  • Functional assessment
    • Time Frame: 6 months
    • Vineland Adaptive Behavior Scales-Second Edition (VINELAND-II). All score are normalized with mean 100 and STD of 15. High score is better than low score .

Participating in This Clinical Trial

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.

Gender Eligibility: All

Minimum Age: 2 Months

Maximum Age: 12 Years

Are Healthy Volunteers Accepted: No

Investigator Details

  • Lead Sponsor
    • Sheba Medical Center
  • Provider of Information About this Clinical Study
    • Principal Investigator: Dr. Omer Bar-Yosef, M.D.-Ph.D. Peadiatric Neurology and Child Development. Deputy Director The Edmond and Lily Safra Children’s Hospital – Sheba Medical Center
  • Overall Official(s)
    • Omer Bar-Yosef, MD.PHD, Study Director, Sheba Medical Center
  • Overall Contact(s)
    • Omer Bar-Yosef, MD.PHD, 972-35302895, Omer.BarYosef@sheba.health.gov.il

References

Bax M, Goldstein M, Rosenbaum P, Leviton A, Paneth N, Dan B, Jacobsson B, Damiano D; Executive Committee for the Definition of Cerebral Palsy. Proposed definition and classification of cerebral palsy, April 2005. Dev Med Child Neurol. 2005 Aug;47(8):571-6. doi: 10.1017/s001216220500112x.

Colver A, Fairhurst C, Pharoah PO. Cerebral palsy. Lancet. 2014 Apr 5;383(9924):1240-9. doi: 10.1016/S0140-6736(13)61835-8. Epub 2013 Nov 20.

Clark SL, Hankins GD. Temporal and demographic trends in cerebral palsy–fact and fiction. Am J Obstet Gynecol. 2003 Mar;188(3):628-33. doi: 10.1067/mob.2003.204.

Clinical trials entries are delivered from the US National Institutes of Health and are not reviewed separately by this site. Please see the identifier information above for retrieving further details from the government database.

At TrialBulletin.com, we keep tabs on over 200,000 clinical trials in the US and abroad, using medical data supplied directly by the US National Institutes of Health. Please see the About and Contact page for details.