A Randomized Study of Autologous Umbilical Cord Blood Reinfusion in Children With Cerebral Palsy

NCT ID: NCT01147653

Last Updated: 2023-01-04

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 63 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2010-06-30
• Study Completion Date: 2016-03-31

Brief Summary

The purpose of this study is to determine the efficacy of a single intravenous infusion of autologous umbilical cord blood (UCB) for the treatment of pediatric patients with spastic cerebral palsy.

Detailed Description

Cerebral palsy results from in utero or perinatal injury to the developing brain, often through stroke, hypoxic insult or hemorrhage. Currently available treatments for patients with cerebral palsy are supportive, but not curative. Umbilical cord blood (UCB) has been shown to lessen the clinical and radiographic impact of hypoxic brain injury and stroke in animal models. UCB also engrafts and differentiates in brain, facilitating neural cell repair, in animal models and human patients with inborn errors of metabolism undergoing allogeneic, unrelated donor UCB transplantation. We hypothesize that, in the setting of brain injury, infusion of autologous UCB will facilitate neural cell repair resulting in improved function in pediatric patients with cerebral palsy.

Conditions

Cerebral Palsy; CP; Spastic Cerebral Palsy

Study Design

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

Study Groups

Autologous UCB Reinfusion First,Then Placebo

Subjects receive their autologous umbilical cord blood cells at Baseline, than placebo at Year 1.

Group Type: ACTIVE_COMPARATOR

Autologous UCB Reinfusion

Intervention Type: BIOLOGICAL

Autologous umbilical cord blood (UCB) reinfusion

Placebo

Intervention Type: OTHER

Placebo

Placebo First, Then Autologous UCB Reinfusion

Subjects receive placebo at Baseline, then autologous umbilical cord blood cell reinfusion at Year 1.

Group Type: PLACEBO_COMPARATOR

Autologous UCB Reinfusion

Intervention Type: BIOLOGICAL

Autologous umbilical cord blood (UCB) reinfusion

Placebo

Intervention Type: OTHER

Placebo

Interventions

Autologous UCB Reinfusion

Autologous umbilical cord blood (UCB) reinfusion

Intervention Type: BIOLOGICAL

Placebo

Placebo

Intervention Type: OTHER

Eligibility Criteria

Inclusion Criteria

• Age ≥ 12 months and ≤ 6 years
• Diagnosis: Spastic cerebral palsy with diplegia, hemiplegia, or quadraplegia.
• Performance status:

Gross Motor Function (GMF) Classification Score levels II - IV or GMF Score leve I, age >/= 2 years Spastic hemiplegia: GMF Score II-IV or minimal functional capabilities in the affected upper extremity. A subject classified as GMFCS level I with significant upper extremity impairment will be eligible if the affected upper extremity is used as an assist only. An eligibility committee will meet to review the child's records and determine eligibility.

Bilateral hypotonic CP (diplegia or quadriplegia): GMF Score II-IV and an abnormal brain MRI suggestive of an acquired etiology (versus a genetic etiology or brain malformation).

• Autologous umbilical cord blood available at a private or public cord blood bank with a minimum total nucleated cell dose of ≥ 1 x 107 cells/kilogram.
• Parental consent.

Exclusion Criteria

• Athetoid cerebral palsy.
• Autism and autistic spectrum disorders without motor disability.
• Hypsarrhythmia.
• Intractable seizures causing epileptic encephalopathy.
• Evidence of a progressive neurologic disease.
• Known HIV or uncontrolled bacterial, fungal, or viral infections.
• Impaired renal or liver function as determined by serum creatinine >1.5mg/dL and/or total bilirubin >1.3mg/dL.
• Head circumference >3 standard deviations below the mean for age.
• Known genetic disease or phenotypic evidence of a genetic disease on physical examination.
• Concurrent genetic or acquired disease or comorbidity(ies) that could require a future allogeneic stem cell transplant.
• Requires ventilatory support, including home ventilator, CPAP, BiPAP, or supplemental oxygen.
• Patient's medical condition does not permit safe travel.
• Previously received any form of cellular therapy.
• Autologous umbilical cord blood unit has any of the following:

1. Total nuclear cell dose < 1 x 107 cells/kilogram

2. Positive maternal infectious disease markers (except CMV)

3. Evidence of infectious contamination of the cord blood unit

4. Lack of a test sample to confirm identity

5. Evidence of a genetic disease

• Unable to obtain parental consent.

• Minimum Eligible Age: 12 Months
• Maximum Eligible Age: 6 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

The Robertson Foundation

OTHER

Sponsor Role: collaborator

Joanne Kurtzberg, MD

OTHER

Sponsor Role: lead

Responsible Party

Joanne Kurtzberg, MD

Professor of Pediatrics

Responsibility Role: SPONSOR_INVESTIGATOR

Principal Investigators

Joanne Kurtzberg, MD

Role: PRINCIPAL_INVESTIGATOR

Duke University

Locations

Duke University Medical Center

Durham, North Carolina, United States

Countries

United States

References

Sun JM, Song AW, Case LE, Mikati MA, Gustafson KE, Simmons R, Goldstein R, Petry J, McLaughlin C, Waters-Pick B, Chen LW, Wease S, Blackwell B, Worley G, Troy J, Kurtzberg J. Effect of Autologous Cord Blood Infusion on Motor Function and Brain Connectivity in Young Children with Cerebral Palsy: A Randomized, Placebo-Controlled Trial. Stem Cells Transl Med. 2017 Dec;6(12):2071-2078. doi: 10.1002/sctm.17-0102. Epub 2017 Oct 28.

Reference Type: DERIVED

PMID: 29080265 (View on PubMed)

Other Identifiers

Pro00017801

Identifier Type: -

Identifier Source: org_study_id