Safety and Effectiveness of Banked Cord Blood or Bone Marrow Stem Cells in Children With Cerebral Palsy (CP).
Condition: Cerebral Palsy
MeSH terms: Paralysis; Cerebral Palsy
Intervention: umbilical cord blood (hUCB) cells; Saline Infusion (Placebo); bone marrow derived mononuclear cells (BMMNCs)
Intervention description: Autologous umbilical cord blood banked with the Cord Blood Registry.; A total of 10 children (5 from each cohort) will be randomized to a placebo infusion at the baseline visit and then have the opportunity to cross-over to stem cell treatment at the 1yr. visit.; Autologous stem cells from bone marrow harvest.
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Crossover Assignment
Masking: Triple (Participant, Care Provider, Outcomes Assessor)
Enrollment: 20.0
Sex: All
Minimum age: 2 Years
Maximum age: 10 Years
Healthy volunteers: No
Keywords: Cerebral Palsy; Brain Injury; Stem Cells; Mononuclear Cells; Bone Marrow; Umbilical Cord Blood

Summary:

      The purpose of this study is to compare the safety and effectiveness of two types of stem
      cells,(either banked cord blood or bone marrow), in children between the ages of 2 to 10
      years with CP. 15 children with banked cord blood at CBR and 15 children without banked cord
      blood will be enrolled into the study. The study involves one baseline/treatment visit and 3
      follow-up visits at 6 months, 12 months, and 2 years. Five children in each group will be
      randomized to a placebo control group at the baseline/treatment visit. Parents will not be
      told if their child received stem cells or a placebo until the 12 month follow-up visit. At
      that time parents may elect to have their child receive the stem cell treatment; either bone
      marrow harvest or umbilical cord blood if banked with CBR. All study visits will be conducted
      at the UTHealth Medical School and Children's Memorial Hermann Hospital in Houston, Texas.

      As of 1/21/2014 we have met our enrollment limit for children without banked cord blood
      undergoing bone marrow harvest for stem cells.
    

Eligibility criteria:

        Inclusion Criteria:

          1. Children with diagnosis of Cerebral Palsy (spastic CP due to periventricular white
             matter damage or neonatal brain injury from perinatal stroke or intra-ventricular
             hemorrhage)

          2. Gross Motor Function Classification Score level II-V

          3. Ages 24 months to 10 years

          4. English speaking, if verbal

          5. Ability to travel to Houston for treatment and follow-up -

        Exclusion Criteria:

          1. Known history of:

               -  Intractable seizures

               -  Traumatic brain injury

               -  Genetic disorder (as demonstrated by newborn screening or genetic diagnostic
                  testing)

               -  Recently treated or current infection

               -  Renal insufficiency or altered renal function (as defined by serum creatinine >
                  1.5 mg/dl at screening)

               -  Hepatic disease or altered liver function (as defined by SGPT > 150 U/L
                  [non-contusion related], and/or T. Bilirubin >1.3 mg/dL at screening)

               -  HIV+ (as demonstrated by positive blood test)

               -  Immunosuppression (as defined by WBC <3,000 cells/ml at screening)

               -  Infectious related neurological injury

               -  Sensitivity to Ethylene Oxide (EtO) [found in fumigants and disinfectants]

          2. If Athetoid CP diagnosis, other etiologies such as degenerative, mitochondrial, and
             metabolic disorders must be excluded, and the outcome assessments must be able to be
             conducted to assess for potential treatment effects

          3. Normal brain MRI

          4. Evidence of acute illness at the time of infusion, such as, but not limited to, fever
             (temperature > 37.5 C), vomiting, diarrhea, wheezing or crackles

          5. Progressing neurological disease (as defined by Batten Disease, Leukodystrophies,
             Metabolic disorders, Mitochondrial disorders, Neurotransmitter disorders)

          6. Microcephaly, macrocephaly, cortical malformations, genetic disorders of dysgenesis
             brain malformations due to infection or metabolic disorders

          7. Pulmonary disease requiring ventilator support

          8. If hUCB candidate, banked cord cells totaling <10 million/kg

          9. If hUCB candidate, any positive maternal infectious disease test (Hepatitis A,
             Hepatitis B, HIV 1, HIV 2, HTLV 1, HTLV 2, and Syphilis)

         10. If hUCB candidate, cord blood sample contamination

         11. Participation in a concurrent intervention study

         12. Unwillingness to return for follow-up visits

         13. Contraindications to MRI

         14. Any patient that the investigators feel in their opinion the study intervention is
             unlikely to benefit the patient will be a screen failure.

         15. Any patients who are currently or has previously been enrolled in a clinical stem cell
             study.