A Randomized Study of Autologous Umbilical Cord Blood Reinfusion in Children With Cerebral Palsy
Condition: Cerebral Palsy; CP; Spastic Cerebral Palsy
MeSH terms: Muscle Spasticity; Paralysis; Cerebral Palsy
Intervention: Autologous UCB Reinfusion; Placebo
Intervention description: Autologous umbilical cord blood (UCB) reinfusion; Placebo
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Crossover Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Enrollment: 63.0
Sex: All
Minimum age: 12 Months
Maximum age: 6 Years
Healthy volunteers: No
Keywords: Cerebral Palsy; CP; Spastic Cerebral Palsy; Cord Blood; Umbilical Cord Blood; Autologous Cord Blood

Summary:

      The purpose of this study is to determine the efficacy of a single intravenous infusion of
      autologous umbilical cord blood (UCB) for the treatment of pediatric patients with spastic
      cerebral palsy.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Age ≥ 12 months and ≤ 6 years

          -  Diagnosis: Spastic cerebral palsy with diplegia, hemiplegia, or quadraplegia.

          -  Performance status:

        Gross Motor Function (GMF) Classification Score levels II - IV or GMF Score leve I, age >/=
        2 years Spastic hemiplegia: GMF Score II-IV or minimal functional capabilities in the
        affected upper extremity. 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. An eligibility committee will meet to review the child's records and determine
        eligibility.

        Bilateral hypotonic CP (diplegia or quadriplegia): GMF Score II-IV and an abnormal brain
        MRI suggestive of an acquired etiology (versus a 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 ≥ 1 x 107 cells/kilogram.

          -  Parental consent.

        Exclusion Criteria:

          -  Athetoid cerebral palsy.

          -  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.

          -  Concurrent genetic or acquired disease or comorbidity(ies) that could require a future
             allogeneic stem cell transplant.

          -  Requires ventilatory support, including home ventilator, CPAP, BiPAP, or supplemental
             oxygen.

          -  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:

               1. Total nuclear cell dose < 1 x 107 cells/kilogram

               2. Positive maternal infectious disease markers (except CMV)

               3. Evidence of infectious contamination of the cord blood unit

               4. Lack of a test sample to confirm identity

               5. Evidence of a genetic disease

          -  Unable to obtain parental consent.