Safety and Effectiveness of Banked Cord Blood or Bone Marrow Stem Cells in Children With Cerebral Palsy (CP). (ACT for CP)

The University of Texas System (UT)

Status and phase: Completed; Phase 2

Conditions: Cerebral Palsy

Treatments: Biological: bone marrow derived mononuclear cells (BMMNCs); Drug: Saline Infusion (Placebo); Biological: umbilical cord blood (hUCB) cells

Study type: Interventional

Funder types: Other

Identifiers

NCT01988584

HSC-MS-12-0876

Details and patient eligibility

About

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.

Enrollment: 20 patients

Sex: All

Ages: 2 to 10 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Crossover Assignment

Masking: Triple Blind

---

20 participants in 4 patient groups

umbilical cord blood (UCB) cells

Experimental group

Description:

Children who have banked UCB with CBR will receive an umbilical cord blood stem cell infusion at the baseline/treatment visit.

Treatment:

Biological: umbilical cord blood (hUCB) cells

bone marrow-derived mononuclear cells (BMMNCs)

Experimental group

Description:

Children in the BMMNC group will undergo bone marrow harvest and stem cell infusion at the baseline/treatment visit.

Treatment:

Biological: bone marrow derived mononuclear cells (BMMNCs)

saline infusion (placebo), then umbilical cord blood (UCB) cells

Experimental group

Description:

Five children in each group will be randomly assigned to receive an inactive substance (placebo) at the baseline/treatment visit. Parents will be given the opportunity to cross-over to either the umbilical cord blood or bone marrow harvest group at the one year visit.

Treatment:

Biological: umbilical cord blood (hUCB) cells

Drug: Saline Infusion (Placebo)

saline infusion (placebo), then bone marrow-derived mononuclear cells (BMMNCs)

Experimental group

Treatment:

Drug: Saline Infusion (Placebo)

Biological: bone marrow derived mononuclear cells (BMMNCs)