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

Primary Purpose

Cerebral Palsy

Status

Withdrawn

Phase

Phase 1

Locations

United States

Study Type

Interventional

Intervention

Cord Blood Infusion

Intravenous Sham

About this trial

This is an interventional treatment trial for Cerebral Palsy focused on measuring Umbilical Cord Blood, Stem Cells, Autologous Blood Transfusion, Cerebral Palsy

Eligibility Criteria

1 Year - 12 Years (Child)All SexesDoes not accept healthy volunteers

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

Sites / Locations

Augusta University

Arms of the Study

Arm 1

Arm 2

Arm Type

Active Comparator

Sham Comparator

Arm Label

Treatment Group 1

Treatment Group 2

Arm Description

Outcomes

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.

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.

Full Information

NCT ID

NCT01072370

First Posted

February 16, 2010

Last Updated

October 6, 2022

Sponsor

Augusta University

1. Study Identification

Unique Protocol Identification Number

NCT01072370

Brief Title

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

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

Study Type

Interventional

2. Study Status

Record Verification Date

October 2022

Overall Recruitment Status

Withdrawn

Study Start Date

January 2010 (undefined)

Primary Completion Date

February 28, 2019 (Actual)

Study Completion Date

February 28, 2019 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Principal Investigator

Name of the Sponsor

Augusta University

4. Oversight

Data Monitoring Committee

Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Cerebral Palsy

Keywords

Umbilical Cord Blood, Stem Cells, Autologous Blood Transfusion, Cerebral Palsy

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1, Phase 2

Interventional Study Model

Crossover Assignment

Masking

ParticipantCare ProviderInvestigatorOutcomes Assessor

Allocation

Randomized

Enrollment

0 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Treatment Group 1

Arm Type

Active Comparator

Arm Title

Treatment Group 2

Arm Type

Sham Comparator

Intervention Type

Biological

Intervention Name(s)

Cord Blood Infusion

Other Intervention Name(s)

Stem cell infusion

Intervention Description

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

Intervention Type

Biological

Intervention Name(s)

Intravenous Sham

Other Intervention Name(s)

Placebo

Intervention Description

intravenous infusion of 5% dextrose, ¼ normal saline solution

Primary Outcome Measure Information:

Title

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

Secondary Outcome Measure Information:

Title

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

10. Eligibility

Sex

All

Minimum Age & Unit of Time

1 Year

Maximum Age & Unit of Time

12 Years

Accepts Healthy Volunteers

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

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

James E Carroll, M.D.

Organizational Affiliation

Augusta University

Official's Role

Study Chair

Facility Information:

Facility Name

Augusta University

City

Augusta

State/Province

Georgia

ZIP/Postal Code

30912

Country

United States

12. IPD Sharing Statement

Links:

URL

http://www.augusta.edu

Description

Augusta University

URL

http://www.cordblood.com

Description

Cord Blood Registry

Learn more about this trial

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

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