Safety and Effectiveness of Cord Blood Stem Cell Infusion for the Treatment of Cerebral Palsy in Children

This study is currently recruiting participants. (see Contacts and Locations)
Verified March 2014 by Georgia Regents University
Sponsor:
Information provided by (Responsible Party):
James E. Carroll, Georgia Regents University
ClinicalTrials.gov Identifier:
NCT01072370
First received: February 16, 2010
Last updated: March 24, 2014
Last verified: March 2014

February 16, 2010
March 24, 2014
January 2010
March 2015   (final data collection date for primary outcome measure)
Confirm the safety of autologous cord blood infusion in children with cerebral palsy by repeated follow-up over one year with clinical and laboratory evaluations. [ Time Frame: 1 year ] [ Designated as safety issue: Yes ]
Same as current
Complete list of historical versions of study NCT01072370 on ClinicalTrials.gov Archive Site
Confirm the efficacy of autologous cord blood infusion in children with cerebral palsy using patient questionnaire and standardized Gross Motor Function Measure evaluation. [ Time Frame: 3-4 months ] [ Designated as safety issue: No ]
Same as current
Not Provided
Not Provided
 
Safety and Effectiveness of Cord Blood Stem Cell Infusion for the Treatment of Cerebral Palsy in Children
A Placebo-Controlled, Observer-Blinded, Crossover Study to Evaluate the Safety and Effectiveness of a Single, Autologous, Cord Blood Stem Cell Infusion for the Treatment of Cerebral Palsy in Children

The purpose of this study is to test the safety and effectiveness of a cord blood infusion in children who have motor disability due to cerebral palsy (CP). The subjects will be children whose parents have saved their infant's cord blood, who have non-progressive motor disability, and whose parents intend to have a cord blood infusion.

The purpose of this study is to conduct an observer-blinded crossover investigation of the safety and efficacy of autologous cord blood infusion in children who demonstrate non-progressive motor disability due to brain dysfunction (commonly called cerebral palsy) and who do not have an apparent disorder of brain development or obstructive hydrocephalus. The degree of delay in motor development will be such that the children are unable to sit independently by 12 months of age or unable to walk independently by 18 months of age. However, because the diagnosis is one of exclusion, we will enroll patients only after they have reached two years of age. By this age, it is likely other conditions would be excluded. As the GMFCS was developed for children up to 12 years of age, the maximum age of recruitment will be 12 years. Any level of cerebral palsy severity will be allowed. The subjects will be children whose parents have saved their infants cord blood, who have clinical evidence of a non-progressive motor disability, and whose parents intend to have a cord blood infusion.

Interventional
Phase 1
Phase 2
Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Crossover Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Cerebral Palsy
  • Biological: Cord Blood Infusion
    red-cell depleted, mononuclear cell enriched cord blood unit prepared for infusion
    Other Name: Stem cell infusion
  • Biological: Intravenous Sham
    intravenous infusion of 5% dextrose, ¼ normal saline solution
    Other Name: Placebo
  • Active Comparator: Treatment Group 1
    Intervention: Biological: Cord Blood Infusion
  • Sham Comparator: Treatment Group 2
    Intervention: Biological: Intravenous Sham
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
40
July 2015
March 2015   (final data collection date for primary outcome measure)

Inclusion Criteria:

  • Must be more than 1 year of age and less than 12 years of age at the time of screening for inclusion in the study.
  • Clinical evidence of a non-progressive motor disability due to brain dysfunction. The subjects will not have the ability to sit independently by one year of age or the ability to walk by 18 months of age.
  • Have stored umbilical cord blood with CBR that meets all selection and testing criteria.
  • Willing to comply with all study procedures.
  • The nucleated cells available in the cord blood sample stored at CBR must exceed 1 X 107 cells per kg body weight. (Note: Because cord blood collection has been in process for about 16 years but widely for far less than that period, the age of most subjects likely will be considerably less than 12 years of age. Due to the amount due of cord blood available for most subjects, the body weight of subjects usually will not exceed 25 kg).
  • The patients must be seizure-free or seizures adequately controlled. If there is a suspicion of seizures and EEG should be done prior to inclusion.

Exclusion Criteria:

  • Have complicating medical issues that would interfere with blood drawing, such as venous access so limited that success is unlikely
  • Presence of obstructive hydrocephalus.
  • Presence of progressive neurological disease.
  • Presence of significant defect of brain development, such as schizencephaly or agenesis of corpus callosum
  • Presence of known chromosomal anomaly
  • Presence of major congenital anomaly
  • Severe intrauterine growth restriction (birth weight less than 1800 grams)
  • Cord blood viability <60%
  • Positive infectious disease markers from mother's blood or cord blood at the time of collection.
  • Evidence of illness on planned infusion date (such as but not limited to fever >38.5, vomiting, diarrhea, wheezing, or crackles)
  • Pregnancy
  • Use of immunosuppressive drugs
  • Evidence of known genetic disorder
  • Impaired hepatic or renal function
Both
1 Year to 12 Years
No
Contact: Kimberly R Gray, BBA, CCRP 706-721-1870 kigray@gru.edu
United States
 
NCT01072370
ACBSC09
Yes
James E. Carroll, Georgia Regents University
Georgia Regents University
Not Provided
Study Chair: James E Carroll, M.D. Georgia Regents University
Georgia Regents University
March 2014

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP