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

October 6, 2022 updated by: James E. Carroll, Augusta University

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.

Study Overview

Status

Withdrawn

Conditions

Intervention / Treatment

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

Interventional

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Georgia
      • Augusta, Georgia, United States, 30912
        • Augusta University

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

1 year to 12 years (Child)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Crossover Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Active Comparator: Treatment Group 1
Biological: Cord Blood Infusion
red-cell depleted, mononuclear cell enriched cord blood unit prepared for infusion
Other Names:
  • Stem cell infusion
Sham Comparator: Treatment Group 2
Biological: Intravenous Sham
intravenous infusion of 5% dextrose, ¼ normal saline solution
Other Names:
  • Placebo

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
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
1 year

Secondary Outcome Measures

Outcome Measure
Time Frame
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
3-4 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Augusta University

Investigators

  • Study Chair: James E Carroll, M.D., Augusta University

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

January 1, 2010

Primary Completion (Actual)

February 28, 2019

Study Completion (Actual)

February 28, 2019

Study Registration Dates

First Submitted

February 16, 2010

First Submitted That Met QC Criteria

February 18, 2010

First Posted (Estimate)

February 22, 2010

Study Record Updates

Last Update Posted (Actual)

October 7, 2022

Last Update Submitted That Met QC Criteria

October 6, 2022

Last Verified

October 1, 2022

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ACBSC09

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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