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

NCT01147653 | Completed Phase 2 Results

• 1 other identifier: Pro00017801
• Study Type: interventional
• Target: 63
• Locations: 1 country
• Sites: 1

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.

Conditions

Cerebral Palsy; CP; Spastic Cerebral Palsy

Keywords

Cerebral Palsy; CP; Spastic Cerebral Palsy; Cord Blood; Umbilical Cord Blood; Autologous Cord Blood

Outcome Measures

Primary Outcomes (1)

• Change in Gross Motor Function Measure 66 (GMFM-66) Score

  Change in Gross Motor Function Measure 66 (GMFM-66) Score from Baseline to Year 1. The GMFM-66 is a clinical tool used to evaluate gross motor function in children with cerebral palsy and is scored using a propriety software program called the Gross Motor Ability Estimator (GMAE) that produces an interval level continuous score ranging from 0 to 100. Higher scores indicate better motor function. A negative change in GMFM-66 score indicates a reduction in motor function, a positive change indicates improvement in motor function, and zero indicates no change in motor function.

  Baseline to Year 1

Secondary Outcomes (24)

• Change in Peabody Gross Motor Quotient From Baseline to Year 1

  Baseline to Year 1

• Change in IT-QOL Questionnaire Score

  Baseline to Year 1

• Change in CP-QOL Score

  Baseline, Year 1

• Change in Whole Brain Connectivity Measured by Diffusion Tensor Magnetic Resonance Imaging (MRI)

  Baseline to Year 1

• Change in Loes Score of Functional MRI From Baseline to Year 1 and From Year 1 to Year 2

  Baseline, Year 1, Year 2

Other Outcomes (1)

• Change in Barry-Albright Dystonia Total Score From Baseline to Year 1

  Baseline to Year 1

Study Arms (2)

Autologous UCB Reinfusion First,Then Placebo

ACTIVE COMPARATOR

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

Biological: Autologous UCB Reinfusion; Other: Placebo

Placebo First, Then Autologous UCB Reinfusion

PLACEBO COMPARATOR

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

Biological: Autologous UCB Reinfusion; Other: Placebo

Interventions

Autologous UCB Reinfusion BIOLOGICAL

Autologous umbilical cord blood (UCB) reinfusion

Autologous UCB Reinfusion First,Then Placebo; Placebo First, Then Autologous UCB Reinfusion

Placebo OTHER

Placebo

Autologous UCB Reinfusion First,Then Placebo; Placebo First, Then Autologous UCB Reinfusion

Eligibility Criteria

• Age: 12 Months - 6 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

• 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:
• Total nuclear cell dose \< 1 x 107 cells/kilogram

Sponsors & Collaborators

• Joanne Kurtzberg, MD: lead
• The Robertson Foundation: collaborator

Study Sites (1)

Duke University Medical Center

Durham, North Carolina, 27705, United States

Location

Related Publications (1)

• 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.

  PMID: 29080265 DERIVED

MeSH Terms

Conditions

Cerebral Palsy

Condition Hierarchy (Ancestors)

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

Results Point of Contact

• Title: Jesse Troy, PhD
• Organization: Duke University School of Medicine, Division of Pediatric Blood and Marrow Transplantation

cordbloodtherapyinfo@dm.duke.edu

Study Officials

• Joanne Kurtzberg, MD

  Duke University

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Professor of Pediatrics

Study Record Dates

• First Submitted: June 17, 2010
• First Posted: June 22, 2010
• Study Start: June 1, 2010
• Primary Completion: March 1, 2016
• Study Completion: March 1, 2016
• Last Updated: May 6, 2026
• Results First Posted: June 12, 2017

Record last verified: 2022-12

Locations

US (1)