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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT03327467
Expanded Access Status : Available
First Posted : October 31, 2017
Last Update Posted : February 28, 2022
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 autism, apraxia, cerebral palsy, hydrocephalus, hypoxic brain injury, stroke, 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.

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

Resource links provided by the National Library of Medicine

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

Information from the National Library of Medicine

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, Learn About Clinical Studies.

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Ages Eligible for Study:   up to 26 Years   (Child, Adult)
Sexes Eligible for Study:   All

Inclusion Criteria:

  1. Have a qualifying sibling or autologous cord blood unit
  2. Be < 26 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. Diagnosed with a genetic or metabolic disorder related to the neurologic condition
  7. History of an immune deficiency
  8. History of treatment with chemo or immunosuppressive therapy
  9. History of previous allogeneic cell therapy. If previously enrolled on another Duke cell therapy protocol the subject can still be considered for enrollment on this study
  10. Need for mechanical ventilation or chronic O2 support
  11. History of inability to protect the airway
  12. 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 identifier (NCT number): NCT03327467

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Contact: Lettie Moore 9196681102
Contact: Jayne Cash

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United States, North Carolina
Duke University Medical Center Available
Durham, North Carolina, United States, 27705
Contact: Lettie Moore   
Contact: Jayne Cash   
United States, Texas
MD Anderson Available
Houston, Texas, United States, 77030
Contact: LaTarsha Williams    713-563-0524   
Principal Investigator: Kris Mahadeo, MD         
Sponsors and Collaborators
Joanne Kurtzberg, MD
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Principal Investigator: Joanne Kurtzberg Duke University
Principal Investigator: Jessica Sun Duke University
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Responsible Party: Joanne Kurtzberg, MD, Professor of Pediatrics, Duke University Identifier: NCT03327467    
Other Study ID Numbers: Pro00083888
First Posted: October 31, 2017    Key Record Dates
Last Update Posted: February 28, 2022
Last Verified: February 2022
Additional relevant MeSH terms:
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Brain Injuries
Cerebral Palsy
Brain Ischemia
Hypoxia-Ischemia, Brain
Autistic Disorder
Autism Spectrum Disorder
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Craniocerebral Trauma
Trauma, Nervous System
Wounds and Injuries
Child Development Disorders, Pervasive
Neurodevelopmental Disorders
Mental Disorders
Pathologic Processes
Brain Damage, Chronic
Signs and Symptoms, Respiratory
Psychomotor Disorders
Neurobehavioral Manifestations
Neurologic Manifestations
Cerebrovascular Disorders
Vascular Diseases
Cardiovascular Diseases
Hypoxia, Brain