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

Tracking Information
First Submitted Date ICMJE	February 16, 2010
First Posted Date ICMJE	February 22, 2010
Last Update Posted Date	January 18, 2018
Study Start Date ICMJE	January 2010
Estimated Primary Completion Date	March 2018 (Final data collection date for primary outcome measure)
Current Primary Outcome Measures ICMJE; (submitted: February 18, 2010)	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 ]
Original Primary Outcome Measures ICMJE	Same as current
Change History	Complete list of historical versions of study NCT01072370 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures ICMJE; (submitted: February 18, 2010)	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 ]
Original Secondary Outcome Measures ICMJE	Same as current
Current Other Outcome Measures ICMJE	Not Provided
Original Other Outcome Measures ICMJE	Not Provided
Descriptive Information
Brief Title ICMJE	Safety and Effectiveness of Cord Blood Stem Cell Infusion for the Treatment of Cerebral Palsy in Children
Official Title ICMJE	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
Brief Summary	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.
Detailed Description	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 Gross Motor Function Classification System (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.
Study Type ICMJE	Interventional
Study Phase	Phase 1; Phase 2
Study Design ICMJE	Allocation: Randomized; Intervention Model: Crossover Assignment; Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor); Primary Purpose: Treatment
Condition ICMJE	Cerebral Palsy
Intervention ICMJE	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
Study Arms	Active Comparator: Treatment Group 1 Intervention: Biological: Cord Blood Infusion; Sham Comparator: Treatment Group 2 Intervention: Biological: Intravenous Sham
Publications *	Not Provided
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
Recruitment Information
Recruitment Status ICMJE	Recruiting
Estimated Enrollment ICMJE; (submitted: February 18, 2010)	40
Original Estimated Enrollment ICMJE	Same as current
Estimated Study Completion Date	July 2018
Estimated Primary Completion Date	March 2018 (Final data collection date for primary outcome measure)
Eligibility Criteria ICMJE	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 Cord Blood Registry (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
Sex/Gender	Sexes Eligible for Study: All
Ages	1 Year to 12 Years (Child)
Accepts Healthy Volunteers	No
Contacts ICMJE	Contact: Kimberly R Gray, BBA, CCRP 706-721-1870 kigray@augusta.edu
Listed Location Countries ICMJE	United States
Removed Location Countries
Administrative Information
NCT Number ICMJE	NCT01072370
Other Study ID Numbers ICMJE	ACBSC09
Has Data Monitoring Committee	Yes
U.S. FDA-regulated Product	Not Provided
IPD Sharing Statement	Not Provided
Responsible Party	James E. Carroll, Augusta University
Study Sponsor ICMJE	Augusta University
Collaborators ICMJE	Not Provided
Investigators ICMJE	Study Chair: James E Carroll, M.D. Augusta University
PRS Account	Augusta University
Verification Date	January 2018
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP