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 August 2016 by Augusta University

Sponsor:

Information provided by (Responsible Party):

James E. Carroll, Georgia Regents University

ClinicalTrials.gov Identifier:

NCT01072370

First received: February 16, 2010

Last updated: August 9, 2016

Last verified: August 2016

History of Changes

Tracking Information

First Received Date ^ICMJE

February 16, 2010

Last Updated Date

August 9, 2016

Start Date ^ICMJE

January 2010

Estimated Primary Completion Date

March 2018 (final data collection date for primary outcome measure)

Current Primary Outcome Measures ^ICMJE

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 ]

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

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 ]

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
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Crossover Assignment
Masking: Double Blind (Subject, Caregiver, 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 Arm (s)

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

Estimated 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

Gender

Both

Ages

1 Year to 12 Years (Child)

Accepts Healthy Volunteers

Contacts ^ICMJE

Contact: Kimberly R Gray, BBA, CCRP

706-721-1870

kigray@gru.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

Plan to Share Data

Not Provided

IPD Description

Not Provided

Responsible Party

James E. Carroll, Georgia Regents University

Study Sponsor ^ICMJE

Augusta University

Collaborators ^ICMJE

Not Provided

Investigators ^ICMJE

Study Chair:

James E Carroll, M.D.

Augusta University

Information Provided By

Augusta University

Verification Date

August 2016

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

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