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

Cerebral Palsy Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT04243408
• Other study ID #: SHEBA-18-5102-OBY-CTIL
• Status: Recruiting
• Phase: Phase 2
• Start date: July 8, 2020
• Est. completion date: December 2024

Study information

• Verified date: January 2021
• Source: Sheba Medical Center
• Contact: Omer Bar-Yosef, MD.PHD
• Phone: 972-35302895
• Email: Omer.BarYosef[@]sheba.health.gov.il
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 72
• Est. completion date: December 2024
• Est. primary completion date: December 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 2 Months to 12 Years

Eligibility

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.

Related Conditions & MeSH terms

• Cerebral Palsy
• Paralysis

Intervention

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

Locations

Country	Name	City	State
Israel	Chaim Seba Medical Center	Ramat Gan

Sponsors (1)

Lead Sponsor	Collaborator
Sheba Medical Center

Country where clinical trial is conducted

Israel, 

References & Publications (3)

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. Review. — View Citation

Clark SL, Hankins GD. Temporal and demographic trends in cerebral palsy--fact and fiction. Am J Obstet Gynecol. 2003 Mar;188(3):628-33. Review. — View Citation

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. Review. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Motor developmental improvement	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 .	6 months
Primary	Motor developmental improvement	Peabody developmental motor scales-second edition (PDMS-2). All score are normalized with mean 100 and STD 10. High score is better	6 months
Primary	Functional assessment	Pediatric Evaluation of Disability Evaluation . Score 0-100 higher score better functionality Pediatric Evaluation of Disability Inventory-Computer Adaptive Test (PEDI-CAT)	6 months
Secondary	Functional assessment	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 .	6 months
Secondary	Functional assessment	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 .	6 months