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

Primary Purpose

Cerebral Palsy

Status
Recruiting
Phase
Phase 2
Locations
Israel
Study Type
Interventional
Intervention
Autologous umbilical cord blood transfusion
placebo
Sponsored by
Sheba Medical Center
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Cerebral Palsy focused on measuring cerebral palsy, autologous umbilical cord blood

Eligibility Criteria

2 Months - 12 Years (Child)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • Chaim Seba Medical CenterRecruiting

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

group 1

group 2

Arm Description

Outcomes

Primary Outcome Measures

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 .
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
Functional assessment
Pediatric Evaluation of Disability Evaluation . Score 0-100 higher score better functionality Pediatric Evaluation of Disability Inventory-Computer Adaptive Test (PEDI-CAT)

Secondary Outcome Measures

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 .
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 .

Full Information

First Posted
January 15, 2020
Last Updated
January 17, 2021
Sponsor
Sheba Medical Center
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1. Study Identification

Unique Protocol Identification Number
NCT04243408
Brief Title
Treatment of Children With Cerebral Palsy With Autologous Umbilical Cord Blood, a Pilot Study
Official Title
Treatment of Children With Cerebral Palsy With Autologous Umbilical Cord Blood, a Pilot Study
Study Type
Interventional

2. Study Status

Record Verification Date
January 2021
Overall Recruitment Status
Recruiting
Study Start Date
July 8, 2020 (Actual)
Primary Completion Date
December 2024 (Anticipated)
Study Completion Date
December 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Sheba Medical Center

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Product Manufactured in and Exported from the U.S.
No
Data Monitoring Committee
Yes

5. Study Description

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Cerebral Palsy
Keywords
cerebral palsy, autologous umbilical cord blood

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
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
Masking
ParticipantCare ProviderInvestigatorOutcomes 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.
Allocation
Randomized
Enrollment
72 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
group 1
Arm Type
Experimental
Arm Title
group 2
Arm Type
Placebo Comparator
Intervention Type
Other
Intervention Name(s)
Autologous umbilical cord blood transfusion
Intervention Description
single dose of an autologous umbilical cord blood transfusion
Intervention Type
Other
Intervention Name(s)
placebo
Intervention Description
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
Primary Outcome Measure Information:
Title
Motor developmental improvement
Description
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 .
Time Frame
6 months
Title
Motor developmental improvement
Description
Peabody developmental motor scales-second edition (PDMS-2). All score are normalized with mean 100 and STD 10. High score is better
Time Frame
6 months
Title
Functional assessment
Description
Pediatric Evaluation of Disability Evaluation . Score 0-100 higher score better functionality Pediatric Evaluation of Disability Inventory-Computer Adaptive Test (PEDI-CAT)
Time Frame
6 months
Secondary Outcome Measure Information:
Title
Functional assessment
Description
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 .
Time Frame
6 months
Title
Functional assessment
Description
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 .
Time Frame
6 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
2 Months
Maximum Age & Unit of Time
12 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Omer Bar-Yosef, MD.PHD
Phone
972-35302895
Email
Omer.BarYosef@sheba.health.gov.il
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Omer Bar-Yosef, MD.PHD
Organizational Affiliation
Sheba Medical Center
Official's Role
Study Director
Facility Information:
Facility Name
Chaim Seba Medical Center
City
Ramat Gan
Country
Israel
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Omer Bar-Yosef

12. IPD Sharing Statement

Plan to Share IPD
Yes
IPD Sharing Plan Description
data set of the baseline of the children, the quantity of cord blood transfusion they received and the clinical followup information.
IPD Sharing Time Frame
within 2 years from the end of data collection
Citations:
PubMed Identifier
16108461
Citation
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.
Results Reference
background
PubMed Identifier
24268104
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.
Results Reference
background
PubMed Identifier
12634632
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. doi: 10.1067/mob.2003.204.
Results Reference
background

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

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