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

This study is currently recruiting participants. (see Contacts and Locations)
Verified January 2014 by The University of Texas Health Science Center, Houston
Sponsor:
Collaborators:
Cord Blood Registry, Inc.
Let's Cure CP Foundation
TIRR Foundation
Information provided by (Responsible Party):
Charles Cox, The University of Texas Health Science Center, Houston
ClinicalTrials.gov Identifier:
NCT01988584
First received: November 4, 2013
Last updated: January 21, 2014
Last verified: January 2014
  Purpose

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 morrow 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.


Condition Intervention Phase
Cerebral Palsy
Biological: Autologous Stem Cells
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Crossover Assignment
Masking: Double Blind (Subject, Caregiver, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Autologous Cell Therapies for Cerebral Palsy-Chronic (ACT for CP)

Resource links provided by NLM:


Further study details as provided by The University of Texas Health Science Center, Houston:

Primary Outcome Measures:
  • To determine if autologous cells using either BMMNCs or hUCBs are safe to administer in children with CP by assessing change at multiple time points. [ Time Frame: All study visits from baseline to the end of study visit at year 2. ] [ Designated as safety issue: Yes ]
    1. In-hospital infusion toxicity: pulmonary and hepatic function; new seizures, hemorrhagic lesions or ischemic lesions on imaging. (Composite Outcome Measure)
    2. Long-term safety: development of new mass lesions or other pathological structural changes; worsening neurological status. (Composite Outcome Measure)


Secondary Outcome Measures:
  • To determine if late functional outcomes are improved following the administration of autologous cells compared with patients in the control group. [ Time Frame: Follow-up visits at 6 and 12 months, and the end of study year 2 visit. ] [ Designated as safety issue: No ]
    1. Detailed analysis of MRI's done at baseline and follow-up visits. Specific white matter tract analysis will be identified at baseline MRI and correlated with motor function studies as the primary lesion of interest. Total volumes and specific tract lesions will be analyzed and correlated with functional outcomes.
    2. The following functional outcomes studies will be performed at baseline, 6 months, 1 year after infusion, and 2 year after infusion.

      • Gross Motor Function Measures
      • Psychological Assessment Tests


Estimated Enrollment: 30
Study Start Date: November 2013
Estimated Primary Completion Date: November 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Active Comparator: Umbilical Cord Blood (UCB) Arm
Children who have banked UCB with CBR will receive an umbilical cord blood stem cell infusion at the baseline/treatment visit.
Biological: Autologous Stem Cells
Other Name: Autolgous stem cells; from either banked umbilical cord blood or bone morrow harvest.
Active Comparator: Bone Marrow Stem Cells (BMMNC's)
Children in the BMMNC group will undergo bone marrow harvest and stem cell infusion at the baseline/treatment visit.
Biological: Autologous Stem Cells
Other Name: Autolgous stem cells; from either banked umbilical cord blood or bone morrow harvest.
Placebo Comparator: Placebo (inactive substance) Group
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.

  Eligibility

Ages Eligible for Study:   2 Years to 10 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01988584

Contacts
Contact: Steven Kosmach, MSN, RN, CCRC 713.500.7329 steven.kosmach@uth.tmc.edu
Contact: Fernando Jimenez, MS, RN 713.500.7395 fernando.jimenez@uth.tmc.edu

Locations
United States, Texas
UTHealth, Medical School, Dept. of Pediatric Surgery Recruiting
Houston, Texas, United States, 77030
Contact: Steven Kosmach, MSN, RN, CCRC    713.500.7329    steven.kosmach@uth.tmc.edu   
Principal Investigator: Charles S Cox, MD         
Sponsors and Collaborators
The University of Texas Health Science Center, Houston
Cord Blood Registry, Inc.
Let's Cure CP Foundation
TIRR Foundation
Investigators
Principal Investigator: Charles S Cox, MD UTHealth, Medical School, Dept. of Pediatric Surgery
  More Information

No publications provided

Responsible Party: Charles Cox, The Children's Fund Distinguished Professor, Department of Pediatric Surgery, The University of Texas Health Science Center, Houston
ClinicalTrials.gov Identifier: NCT01988584     History of Changes
Other Study ID Numbers: HSC-MS-12-0876
Study First Received: November 4, 2013
Last Updated: January 21, 2014
Health Authority: United States: Food and Drug Administration

Keywords provided by The University of Texas Health Science Center, Houston:
Cerebral Palsy
Brain Injury
Stem Cells
Mononuclear Cells
Bone Morrow
Umbilical Cord Blood

Additional relevant MeSH terms:
Cerebral Palsy
Paralysis
Brain Damage, Chronic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Neurologic Manifestations
Signs and Symptoms

ClinicalTrials.gov processed this record on July 24, 2014