Clinical trial NCT04243408: 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
Overall status: UNKNOWN
Phase: PHASE2
Conditions: Cerebral Palsy
Keywords: cerebral palsy, autologous umbilical cord blood
Lead sponsor: Sheba Medical Center
Sponsor class: OTHER_GOV
First posted: 2020-01-28
Enrollment: 72
Sex: ALL
Minimum age (years): 2
Maximum age (years): 12
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention 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
Primary purpose: TREATMENT

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


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.

Primary outcomes:
measure: Motor developmental improvement
timeFrame: 6 months
measure: Motor developmental improvement
timeFrame: 6 months
measure: Functional assessment
timeFrame: 6 months
Secondary outcomes:
measure: Functional assessment
timeFrame: 6 months
measure: Functional assessment
timeFrame: 6 months
Other outcomes:
Locations:
city: Ramat Gan
country: Israel
facility: Chaim Seba Medical Center
Study officials:
affiliation: Sheba Medical Center
name: Omer Bar-Yosef, MD.PHD
role: STUDY_DIRECTOR
References:
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.
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.
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.