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Trial record 5 of 22 for:    umbilical cord cerebral palsy

Assessment of the Safety of Allogeneic Umbilical Cord Blood Infusions in Children With Cerebral Palsy

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Joanne Kurtzberg, MD, Duke University Medical Center
ClinicalTrials.gov Identifier:
NCT02599207
First received: November 2, 2015
Last updated: June 27, 2017
Last verified: June 2017
  Purpose
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.

Condition Intervention Phase
Cerebral Palsy Biological: sibling umbilical cord blood Phase 1

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Assessment of the Safety of Allogeneic Umbilical Cord Blood Infusions in Children With Cerebral Palsy

Resource links provided by NLM:


Further study details as provided by Joanne Kurtzberg, MD, Duke University Medical Center:

Primary Outcome Measures:
  • 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.


Secondary Outcome Measures:
  • 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.


Enrollment: 15
Study Start Date: November 2015
Estimated Study Completion Date: May 2018
Primary Completion Date: October 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Matched related umbilical cord blood
Six subjects will receive an infusion of HLA matched sibling umbilical cord blood cells.
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.
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.
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.

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.
  Eligibility

Ages Eligible for Study:   1 Year to 6 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02599207

Locations
United States, North Carolina
Duke University Medical Center
Durham, North Carolina, United States, 27705
Sponsors and Collaborators
Joanne Kurtzberg, MD
Investigators
Principal Investigator: Joanne Kurtzberg, MD Duke University
Principal Investigator: Jessica Sun, MD Duke University
  More Information

Responsible Party: Joanne Kurtzberg, MD, Professor of Pediatrics, Duke University Medical Center
ClinicalTrials.gov Identifier: NCT02599207     History of Changes
Other Study ID Numbers: Pro00065043
Study First Received: November 2, 2015
Last Updated: June 27, 2017

Additional relevant MeSH terms:
Paralysis
Cerebral Palsy
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms
Brain Damage, Chronic
Brain Diseases
Central Nervous System Diseases

ClinicalTrials.gov processed this record on August 18, 2017