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

NCT01072370 | Withdrawn Phase 1 DMC

• 1 other identifier: ACBSC09
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 1

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.

Conditions

Cerebral Palsy

Keywords

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

Outcome Measures

Primary Outcomes (1)

• 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.

  1 year

Secondary Outcomes (1)

• Confirm the efficacy of autologous cord blood infusion in children with cerebral palsy using patient questionnaire and standardized Gross Motor Function Measure evaluation.

  3-4 months

Study Arms (2)

Treatment Group 1

ACTIVE COMPARATOR

Biological: Cord Blood Infusion

Treatment Group 2

SHAM COMPARATOR

Biological: Intravenous Sham

Interventions

Cord Blood Infusion BIOLOGICAL

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

Also known as: Stem cell infusion

Treatment Group 1

Intravenous Sham BIOLOGICAL

intravenous infusion of 5% dextrose, ¼ normal saline solution

Also known as: Placebo

Treatment Group 2

Eligibility Criteria

• Age: 1 Year - 12 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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).

You may not qualify if:

• 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

Sponsors & Collaborators

• Augusta University: lead

Study Sites (1)

Augusta University

Augusta, Georgia, 30912, United States

Location

Related Links

• Augusta University

MeSH Terms

Conditions

Cerebral Palsy

Condition Hierarchy (Ancestors)

Brain Damage, Chronic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases

Study Officials

• James E Carroll, M.D.

  Augusta University

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor and Chief, Child Neurology

Study Record Dates

• First Submitted: February 16, 2010
• First Posted: February 22, 2010
• Study Start: January 1, 2010
• Primary Completion: February 28, 2019
• Study Completion: February 28, 2019
• Last Updated: October 7, 2022

Record last verified: 2022-10

Locations

US (1)