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

NCT ID: NCT04243408

Last Updated: 2021-01-20

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

UNKNOWN

Clinical Phase

PHASE2

Total Enrollment

72 participants

Study Classification

INTERVENTIONAL

Study Start Date

2020-07-08

Study Completion Date

2024-12-31

Brief Summary

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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

Detailed Description

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Conditions

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Cerebral Palsy

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

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
Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors
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.

Study Groups

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group 1

Group Type EXPERIMENTAL

Autologous umbilical cord blood transfusion

Intervention Type OTHER

single dose of an autologous umbilical cord blood transfusion

group 2

Group Type PLACEBO_COMPARATOR

placebo

Intervention Type 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

Interventions

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Autologous umbilical cord blood transfusion

single dose of an autologous umbilical cord blood transfusion

Intervention Type 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

Intervention Type OTHER

Eligibility Criteria

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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.
Minimum Eligible Age

2 Months

Maximum Eligible Age

12 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Sheba Medical Center

OTHER_GOV

Sponsor Role lead

Responsible Party

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Dr. Omer Bar-Yosef

M.D.-Ph.D. Peadiatric Neurology and Child Development. Deputy Director The Edmond and Lily Safra Children's Hospital

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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Omer Bar-Yosef, MD.PHD

Role: STUDY_DIRECTOR

Sheba Medical Center

Locations

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Chaim Seba Medical Center

Ramat Gan, , Israel

Site Status RECRUITING

Countries

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Israel

Central Contacts

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Omer Bar-Yosef, MD.PHD

Role: CONTACT

972-35302895

Facility Contacts

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Omer Bar-Yosef

Role: primary

References

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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.

Reference Type BACKGROUND
PMID: 16108461 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 24268104 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 12634632 (View on PubMed)

Other Identifiers

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SHEBA-18-5102-OBY-CTIL

Identifier Type: -

Identifier Source: org_study_id

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