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

NCT ID: NCT01072370

Last Updated: 2022-10-07

Basic Information

• Recruitment Status: WITHDRAWN
• Clinical Phase: PHASE1/PHASE2
• Study Classification: INTERVENTIONAL
• Study Start Date: 2010-01-31
• Study Completion Date: 2019-02-28

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.

Conditions

Cerebral Palsy

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: CROSSOVER
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

Treatment Group 1

Group Type: ACTIVE_COMPARATOR

Cord Blood Infusion

Intervention Type: BIOLOGICAL

red-cell depleted, mononuclear cell enriched cord blood unit prepared for infusion

Treatment Group 2

Group Type: SHAM_COMPARATOR

Intravenous Sham

Intervention Type: BIOLOGICAL

intravenous infusion of 5% dextrose, ¼ normal saline solution

Interventions

Cord Blood Infusion

red-cell depleted, mononuclear cell enriched cord blood unit prepared for infusion

Intervention Type: BIOLOGICAL

Intravenous Sham

intravenous infusion of 5% dextrose, ¼ normal saline solution

Intervention Type: BIOLOGICAL

Other Intervention Names

Stem cell infusion; Placebo

Eligibility 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 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

• Minimum Eligible Age: 1 Year
• Maximum Eligible Age: 12 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Augusta University

OTHER

Sponsor Role: lead

Responsible Party

James E. Carroll

Professor and Chief, Child Neurology

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

James E Carroll, M.D.

Role: STUDY_CHAIR

Augusta University

Locations

Augusta University

Augusta, Georgia, United States

Countries

United States

Related Links

http://www.augusta.edu

Augusta University

Other Identifiers

ACBSC09

Identifier Type: -

Identifier Source: org_study_id