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Trial record 1 of 1 for:    NCT02599207
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Assessment of the Safety of Allogeneic Umbilical Cord Blood Infusions in Children With Cerebral Palsy

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ClinicalTrials.gov Identifier: NCT02599207
Recruitment Status : Completed
First Posted : November 6, 2015
Last Update Posted : December 4, 2019
Sponsor:
Information provided by (Responsible Party):
Joanne Kurtzberg, MD, Duke University

Tracking Information
First Submitted Date  ICMJE November 2, 2015
First Posted Date  ICMJE November 6, 2015
Last Update Posted Date December 4, 2019
Study Start Date  ICMJE November 2015
Actual Primary Completion Date October 2016   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: November 5, 2015)
Assessment for infusion reactions, infections, graft versus host disease or any other adverse events [ Time Frame: 6 months ]
The primary endpoint of this study is safety which will be evaluated by assessing the incidence of acute infusion reactions, infections, graft versus host disease.
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: November 5, 2015)
  • Assessment for improvement in gross motor function [ Time Frame: 6 months ]
    Assessment of improvement in gross motor function using validated tools.
  • Assessment for improvement in fine motor function [ Time Frame: 6 months ]
    Assessment of fine motor function using validated tools.
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Assessment of the Safety of Allogeneic Umbilical Cord Blood Infusions in Children With Cerebral Palsy
Official Title  ICMJE Assessment of the Safety of Allogeneic Umbilical Cord Blood Infusions in Children With Cerebral Palsy
Brief Summary This study is a single site, phase I, prospective study of the safety of intravenous sibling cord blood infusion in 15 children ages 1-6 years with Cerebral Palsy (CP). All subjects will be treated with sibling cord blood cells. The first six will receive cord blood cells from an HLA-matched sibling. The following nine subjects will receive cord blood cells from an HLA-mismatched (≥3/6 match) or matched sibling. The duration of study participation will be six months from the time of the cord blood infusion.
Detailed Description This study is a phase I, prospective, open-label trial designed to assess the safety of a single allogeneic sibling cord blood infusion in young children with cerebral palsy. Children ages one to six years with uncomplicated cerebral palsy and an available HLA matched or haploidentical, qualified, sibling cord blood unit will be eligible to participate. All participants will receive a single intravenous infusion of allogeneic sibling cord blood. All participants will have an initial clinical evaluation to verify the diagnosis of cerebral palsy and determine eligibility. The main endpoint is safety, for which acute infusion reactions as well as incidence of infections and graft versus host disease will be assessed. Functional outcome measures, described below, will be assessed at baseline and six months post sibling cord blood infusion and described.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Cerebral Palsy
Intervention  ICMJE Biological: sibling umbilical cord blood
All subjects will receive a single infusion of allogeneic sibling cord blood. The first six subjects will receive cord blood cells from an HLA-matched sibling. The following 9 subjects will receive cord blood cells from an HLA-mismatched (≥3/6 match) or matched sibling. Six of the 15 subjects must be treated with haplo-identical sibling CB. Duration of study participation will be six months from the time of CB infusion.
Study Arms  ICMJE
  • Experimental: Matched related umbilical cord blood
    Six subjects will receive an infusion of HLA matched sibling umbilical cord blood cells.
    Intervention: Biological: sibling umbilical cord blood
  • Experimental: Mismatched related umbilical cord blood
    Nine subjects will receive an infusion of HLA-mismatched (≥3/6 match) or matched sibling umbilical cord blood cells.
    Intervention: Biological: sibling umbilical cord blood
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: November 5, 2015)
15
Original Estimated Enrollment  ICMJE Same as current
Actual Study Completion Date  ICMJE July 2018
Actual Primary Completion Date October 2016   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  1. Age ≥12 months and ≤ 6 years at the time of CB infusion.
  2. Diagnosis: Cerebral palsy with diplegia, hemiplegia, or quadriplegia.
  3. Performance status:

    • Bilateral cerebral palsy (diplegia or quadraplegia):

    Gross Motor Function Classification Score levels II - IV, or Gross Motor Function Classification Score level I, age ≥ 2 years

    • Hemiplegia: Gross Motor Function Classification Score levels 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.
  4. Review of brain imaging (obtained as standard of care prior to study entry) does not suggest a genetic condition or brain malformation.
  5. Suitably matched sibling donor CB unit (see section 6.2 for matching details) available at a private or public cord blood bank with a minimum total nucleated cell dose of ≥ 2.5 x 107 cells/kilogram.
  6. Legal authorized representative consent.

Exclusion Criteria:

  1. Available qualified autologous cord blood unit
  2. Autism and autistic spectrum disorders without motor disability.
  3. Hypsarrhythmia.
  4. Intractable seizures causing epileptic encephalopathy.
  5. Evidence of a progressive neurologic disease.
  6. Has an active, uncontrolled systemic infection or documentation of HIV+ status.
  7. Known genetic disease or phenotypic evidence of a genetic disease on physical exam.
  8. Concurrent genetic or acquired disease or comorbidity(ies) that could require a future allogeneic stem cell transplant.
  9. Requires ventilatory support, including home ventilator, CPAP, BiPAP, or supplemental oxygen.
  10. Impaired renal or liver function as determined by serum creatinine >1.5mg/dL and/or total bilirubin >1.3mg/dL except in patients with known Gilbert's disease.
  11. Possible immunosuppression, defined as WBC <3,000 cells/mL or absolute lymphocyte count (ALC) below normal for age with abnormal T-cell subsets.
  12. Patient's medical condition does not permit safe travel.
  13. Previously received any form of cellular therapy.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 1 Year to 6 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02599207
Other Study ID Numbers  ICMJE Pro00065043
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Joanne Kurtzberg, MD, Duke University
Study Sponsor  ICMJE Joanne Kurtzberg, MD
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Joanne Kurtzberg, MD Duke University
Principal Investigator: Jessica Sun, MD Duke University
PRS Account Duke University
Verification Date December 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP