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

NCT04243408 | Unknown Phase 2 DMC

• 1 other identifier: SHEBA-18-5102-OBY-CTIL
• Study Type: interventional
• Target: 72
• Locations: 1 country
• Sites: 1

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

Conditions

Cerebral Palsy

Keywords

cerebral palsy, autologous umbilical cord blood

Outcome Measures

Primary Outcomes (3)

• 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

• 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

• 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 Outcomes (2)

• Functional assessment

  6 months

• Functional assessment

  6 months

Study Arms (2)

group 1

EXPERIMENTAL

Other: Autologous umbilical cord blood transfusion

group 2

PLACEBO COMPARATOR

Other: placebo

Interventions

Autologous umbilical cord blood transfusion OTHER

single dose of an autologous umbilical cord blood transfusion

group 1

placebo OTHER

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

group 2

Eligibility Criteria

• Age: 2 Months - 12 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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).
• \. 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.
• \. Parental consent.

You may not qualify if:

• 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)

Sponsors & Collaborators

• Sheba Medical Center: lead

Study Sites (1)

Chaim Seba Medical Center

Ramat Gan, Israel

RECRUITING

Related 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. doi: 10.1017/s001216220500112x.

  PMID: 16108461 BACKGROUND

• 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.

  PMID: 24268104 BACKGROUND

• 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.

  PMID: 12634632 BACKGROUND

MeSH Terms

Conditions

Cerebral Palsy

Condition Hierarchy (Ancestors)

Brain Damage, Chronic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases

Study Officials

• Omer Bar-Yosef, MD.PHD

  Sheba Medical Center

  STUDY DIRECTOR

Central Study Contacts

Omer Bar-Yosef, MD.PHD

CONTACT

972-35302895; Omer.BarYosef@sheba.health.gov.il

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: 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.
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER GOV
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: M.D.-Ph.D. Peadiatric Neurology and Child Development. Deputy Director The Edmond and Lily Safra Children's Hospital

Model Details: 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

Study Record Dates

• First Submitted: January 15, 2020
• First Posted: January 28, 2020
• Study Start: July 8, 2020
• Primary Completion: December 1, 2024
• Study Completion: December 1, 2024
• Last Updated: January 20, 2021

Record last verified: 2021-01

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR
• Time Frame: within 2 years from the end of data collection

Locations

IL (1)