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Expanded Access Protocol: Umbilical Cord Blood Infusions for Children With Brain Injuries

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ClinicalTrials.gov Identifier: NCT03327467
Expanded Access Status : Available
First Posted : October 31, 2017
Last Update Posted : March 16, 2018
Sponsor:
Information provided by (Responsible Party):
Joanne Kurtzberg, MD, Duke University

Brief Summary:

The objective of the study is to enable access to sibling or autologous umbilical cord blood (UCB) infusions for children with various brain disorders.

The use of UCB in this fashion is based on safety and efficacy data from prior and ongoing clinical trials at Duke University Medical Center in over 700 patients with these diagnoses infused with autologous or sibling UCB over the past decade.


Condition or disease Intervention/treatment
Autism Spectrum Disorder Autism Cerebral Palsy Hydrocephalus Apraxia of Speech Hypoxia Ischemia, Cerebral Drowning; Anoxia Biological: Cord Blood Infusion

Detailed Description:

The primary purpose of this protocol is to enable access to sibling or autologous umbilical cord blood for children with various brain disorders. Children with cerebral palsy, hydrocephalus, hypoxic brain injury, stroke, apraxia, autism and other brain injuries will be eligible if they do not qualify for or are unable to participate in another active clinical trial at Duke. Children with qualifying partially or fully matched sibling or autologous cord blood units will be eligible. The family will be enrolled on the screening protocol, "A Research Database and Screening Protocol for Children with Brain Injuries Potentially Undergoing Cellular Therapy or Other Clinical Trials," Pro00063563. Information may be obtained under this protocol including but not limited to: diagnostic information, genetic testing, videos and pictures of the child, cord blood report, etc. The PI or her designee will review this information to determine if the child might be eligible for a cord blood infusion(s) on this expanded access protocol.

If eligible, the child must come to Duke with a parent or legal guardian for clinical evaluations and infusion of the umbilical cord blood unit. Parents will be required to participate in remote follow-up phone calls and be willing to complete questionnaires for safety follow-up.


Study Type : Expanded Access
Expanded Access Type : Treatment IND/Protocol
Official Title: Expanded Access Protocol: Umbilical Cord Blood Infusions for Children With Brain Injuries



Intervention Details:
  • Biological: Cord Blood Infusion
    IV infusion of umbilical cord infusion (sibling or autologous)

Information from the National Library of Medicine

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Ages Eligible for Study:   up to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Criteria

Inclusion Criteria:

  1. Have a qualifying sibling or autologous cord blood unit
  2. Be < 18 years of age at the time of consent.
  3. Have given written informed consent according to FDA guidelines (or consent of parent/legal guardian as applicable).
  4. Have baseline blood counts and basic chemistries within normal for age.
  5. Have a normal absolute lymphocyte count (ALC) for age on baseline blood count and differential.

Exclusion Criteria:

  1. Documented HIV or Hepatitis or other disease transmittable through the blood.
  2. A cord blood unit that fails to meet specifications
  3. Refusal of consent
  4. Uncontrolled seizure disorder
  5. Uncontrolled infection
  6. History of an immune deficiency
  7. History of treatment with chemo or immunosuppressive therapy
  8. History of previous allogeneic cell therapy
  9. Need for mechanical ventilation or chronic O2 support
  10. History of inability to protect the airway
  11. Eligibility for an active clinical trial of cellular therapies at Duke

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03327467


Contacts
Contact: Hildy Donner 8448002673 cordbloodtherapyinfo@dm.duke.edu
Contact: Jayne Cash cordbloodtherapyinfo@dm.duke.edu

Sponsors and Collaborators
Joanne Kurtzberg, MD
Investigators
Principal Investigator: Joanne Kurtzberg Duke University
Principal Investigator: Jessica Sun Duke University

Responsible Party: Joanne Kurtzberg, MD, Professor of Pediatrics, Duke University
ClinicalTrials.gov Identifier: NCT03327467     History of Changes
Other Study ID Numbers: Pro00083888
First Posted: October 31, 2017    Key Record Dates
Last Update Posted: March 16, 2018
Last Verified: March 2018

Additional relevant MeSH terms:
Hydrocephalus
Brain Injuries
Ischemia
Autistic Disorder
Cerebral Palsy
Autism Spectrum Disorder
Hypoxia
Apraxias
Drowning
Brain Ischemia
Hypoxia-Ischemia, Brain
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Craniocerebral Trauma
Trauma, Nervous System
Wounds and Injuries
Pathologic Processes
Child Development Disorders, Pervasive
Neurodevelopmental Disorders
Mental Disorders
Brain Damage, Chronic
Signs and Symptoms, Respiratory
Signs and Symptoms
Psychomotor Disorders
Neurobehavioral Manifestations
Neurologic Manifestations
Death
Cerebrovascular Disorders
Vascular Diseases