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

NCT ID: NCT01988584

Last Updated: 2022-11-21

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 20 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2013-11-30
• Study Completion Date: 2018-02-21

Brief 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.

Conditions

Cerebral Palsy

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: CROSSOVER
• Primary Study Purpose: TREATMENT
• Blinding Strategy: TRIPLE

Study Groups

umbilical cord blood (UCB) cells

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

Group Type: EXPERIMENTAL

umbilical cord blood (hUCB) cells

Intervention Type: BIOLOGICAL

Autologous umbilical cord blood banked with the Cord Blood Registry.

bone marrow-derived mononuclear cells (BMMNCs)

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

Group Type: EXPERIMENTAL

bone marrow derived mononuclear cells (BMMNCs)

Intervention Type: BIOLOGICAL

Autologous stem cells from bone marrow harvest.

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

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.

Group Type: EXPERIMENTAL

umbilical cord blood (hUCB) cells

Intervention Type: BIOLOGICAL

Autologous umbilical cord blood banked with the Cord Blood Registry.

Saline Infusion (Placebo)

Intervention Type: DRUG

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.

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

Group Type: EXPERIMENTAL

Saline Infusion (Placebo)

Intervention Type: DRUG

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.

bone marrow derived mononuclear cells (BMMNCs)

Intervention Type: BIOLOGICAL

Autologous stem cells from bone marrow harvest.

Interventions

umbilical cord blood (hUCB) cells

Autologous umbilical cord blood banked with the Cord Blood Registry.

Intervention Type: BIOLOGICAL

Saline Infusion (Placebo)

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.

Intervention Type: DRUG

bone marrow derived mononuclear cells (BMMNCs)

Autologous stem cells from bone marrow harvest.

Intervention Type: BIOLOGICAL

Other Intervention Names

Autolgous Stem Cells; Autologous 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.

• Minimum Eligible Age: 2 Years
• Maximum Eligible Age: 10 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Cord Blood Registry, Inc.

INDUSTRY

Sponsor Role: collaborator

Let's Cure CP Foundation

OTHER

Sponsor Role: collaborator

Mission Connect, a program of TIRR Foundation

UNKNOWN

Sponsor Role: collaborator

The University of Texas Health Science Center, Houston

OTHER

Sponsor Role: lead

Responsible Party

Charles Cox

The Children's Fund Distinguished Professor, Department of Pediatric Surgery

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Charles S Cox, MD

Role: PRINCIPAL_INVESTIGATOR

UTHealth, Medical School, Dept. of Pediatric Surgery

Locations

UTHealth, Medical School, Dept. of Pediatric Surgery

Houston, Texas, United States

Countries

United States

Provided Documents

Document Type: Study Protocol and Statistical Analysis Plan

View Document

Other Identifiers

HSC-MS-12-0876

Identifier Type: -

Identifier Source: org_study_id