Assessment of the Safety of Allogeneic Umbilical Cord Blood Infusions in Children With Cerebral Palsy

This study is a phase I, prospective, open-label trial designed to assess the safety of a
single allogeneic sibling cord blood infusion in young children with cerebral palsy. Children
ages one to six years with uncomplicated cerebral palsy and an available HLA matched or
haploidentical, qualified, sibling cord blood unit will be eligible to participate. All
participants will receive a single intravenous infusion of allogeneic sibling cord blood. All
participants will have an initial clinical evaluation to verify the diagnosis of cerebral
palsy and determine eligibility. The main endpoint is safety, for which acute infusion
reactions as well as incidence of infections and graft versus host disease will be assessed.
Functional outcome measures, described below, will be assessed at baseline and six months
post sibling cord blood infusion and described.

Inclusion Criteria:

1. Age ≥12 months and ≤ 6 years at the time of CB infusion.

2. Diagnosis: Cerebral palsy with diplegia, hemiplegia, or quadriplegia.

3. Performance status:

- Bilateral cerebral palsy (diplegia or quadraplegia):

Gross Motor Function Classification Score levels II - IV, or Gross Motor Function
Classification Score level I, age ≥ 2 years

- Hemiplegia: Gross Motor Function Classification Score levels 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.

4. Review of brain imaging (obtained as standard of care prior to study entry) does not
suggest a genetic condition or brain malformation.

5. Suitably matched sibling donor CB unit (see section 6.2 for matching details)
available at a private or public cord blood bank with a minimum total nucleated cell
dose of ≥ 2.5 x 107 cells/kilogram.

6. Legal authorized representative consent.

Exclusion Criteria:

1. Available qualified autologous cord blood unit

2. Autism and autistic spectrum disorders without motor disability.

3. Hypsarrhythmia.

4. Intractable seizures causing epileptic encephalopathy.

5. Evidence of a progressive neurologic disease.

6. Has an active, uncontrolled systemic infection or documentation of HIV+ status.

7. Known genetic disease or phenotypic evidence of a genetic disease on physical exam.

8. Concurrent genetic or acquired disease or comorbidity(ies) that could require a future
allogeneic stem cell transplant.

9. Requires ventilatory support, including home ventilator, CPAP, BiPAP, or supplemental
oxygen.

10. Impaired renal or liver function as determined by serum creatinine >1.5mg/dL and/or
total bilirubin >1.3mg/dL except in patients with known Gilbert's disease.

11. Possible immunosuppression, defined as WBC <3,000 cells/mL or absolute lymphocyte
count (ALC) below normal for age with abnormal T-cell subsets.

12. Patient's medical condition does not permit safe travel.

13. Previously received any form of cellular therapy.