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

Sponsor:	Georgia Health Sciences University
Information provided by:	Georgia Health Sciences University
ClinicalTrials.gov Identifier:	NCT01072370

Purpose

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.

Condition	Intervention	Phase
Cerebral Palsy	Biological: Cord Blood Infusion Biological: Intravenous Sham	Phase 1 Phase 2

Study Type:	Interventional
Study Design:	Allocation: Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Crossover Assignment Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor) Primary Purpose: Treatment
Official Title:	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

Resource links provided by NLM:

MedlinePlus related topics: Cerebral Palsy Paralysis

U.S. FDA Resources

Further study details as provided by Georgia Health Sciences University:

Primary Outcome Measures:

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 ]

Secondary Outcome Measures:

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 ]

Estimated Enrollment:	40
Study Start Date:	January 2010
Estimated Study Completion Date:	February 2013
Estimated Primary Completion Date:	February 2012 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
Active Comparator: Treatment Group 1	Biological: Cord Blood Infusion red-cell depleted, mononuclear cell enriched cord blood unit prepared for infusion Other Name: Stem cell infusion
Sham Comparator: Treatment Group 2	Biological: Intravenous Sham intravenous infusion of 5% dextrose, ¼ normal saline solution Other Name: Placebo

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 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.

Eligibility

Ages Eligible for Study:	1 Year to 12 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

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

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT01072370

Contacts

Contact: Kimberly R Gray, BBA, CCRP

706-721-3600

kgray@georgiahealth.edu

Locations

United States, Georgia
Medical College of Georgia	Recruiting
Augusta, Georgia, United States, 30912
Contact: James E Carroll, M.D. 706-721-3371 jcarroll@georgiahealth.edu
Sub-Investigator: Roger A Vega, M.D.
Principal Investigator: James E Carroll, M.D.
Sub-Investigator: Elizabeth Sekul, M.D.
Sub-Investigator: Lloyd O Cook, M.D.
Sub-Investigator: Roni Bollag, M.D., Ph.D.
Sub-Investigator: Suzanne Strickland, MD
Sub-Investigator: Afshin Ameri, MD

Sponsors and Collaborators

Georgia Health Sciences University

Investigators

Study Chair:

James E Carroll, M.D.

Georgia Health Sciences University

More Information

Additional Information:

Cord Blood Registry This link exits the ClinicalTrials.gov site

Medical College of Georgia Department of Neurology This link exits the ClinicalTrials.gov site

No publications provided

Responsible Party:	James E. Carroll, M.D., Medical College of Georgia
ClinicalTrials.gov Identifier:	NCT01072370 History of Changes
Other Study ID Numbers:	ACBSC09
Study First Received:	February 16, 2010
Last Updated:	February 1, 2011
Health Authority:	United States: Food and Drug Administration

Keywords provided by Georgia Health Sciences University:

Umbilical Cord Blood
Stem Cells
Autologous Blood Transfusion
Cerebral Palsy

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 May 23, 2012