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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.
 
ClinicalTrials.gov Identifier: NCT03327467
Expanded Access Status : Available
First Posted : October 31, 2017
Last Update Posted : December 5, 2022
Sponsor:
Information provided by (Responsible Party):
Joanne Kurtzberg, MD, Duke University

Study Description
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Brief Summary:
The purpose of this protocol is to enable access to intravenous infusions of banked autologous (a person's own) or sibling umbilical cord blood (CB) for children with various brain disorders. This is an expanded access protocol intended for patients who are unable to participate in a clinical trial involving their own or their sibling's cord blood. Children with cerebral palsy, congenital hydrocephalus, apraxia, stroke, hypoxic brain injury and related conditions will be eligible if they have normal immune function and do not qualify for, have previously participated in, or are unable to participate in an active cell therapy clinical trial at Duke Medicine. For the purpose of this protocol the term children refers to patients less than 26 years of age. The cord blood is thawed and then administered as an intravenous infusion. Recipients do not receive chemotherapy or immunosuppression. The mechanism of action is through paracrine signaling of cord blood monocytes inducing endogenous cells to repair existing damage.

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

Detailed Description:

This protocol is designed to enable access to intravenous infusions of banked autologous (a person's own) or sibling umbilical cord blood (CB) for children with various brain disorders. Children with cerebral palsy, congenital hydrocephalus, apraxia, stroke, hypoxic brain injury and related conditions will be eligible if they have normal immune function and do not qualify for, have previously participated in, or are unable to participate in an active cell therapy clinical trial at Duke Medicine. For the purpose of this protocol the term children refers to patients less than 26 years of age. Cord blood is administered as a cellular infusion without prior treatment with chemotherapy or immunosuppression. The use of CB 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 over the past decade. The mechanism of action is through paracrine signaling of cord blood monocytes inducing endogenous cells to repair existing damage. Of note, CB is thawed, washed and infused through a peripheral IV without prior or post-infusion immunosuppression.

Initially, the participant 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 about the participant's health and eligibility will be obtained under this protocol and may include records such as diagnostic information, genetic testing, videos, pictures of the child, and a cord blood report. The study team will review these records to determine if the child and the cord blood unit are eligible. For all cord blood infusions, the unit must meet certain cell count, sterility, and viability criteria. For sibling cord blood infusions, the intended recipient and their sibling must be at least a half HLA match.

If eligible, the participant will come to Duke with a parent or legal guardian for clinical evaluations and infusion of the umbilical cord blood unit. Typically, medical evaluations are done on day one of the visit and the infusion is given on day two. Parents will be required to participate in remote follow-up phone calls and be willing to complete questionnaires for safety follow ups. Safety questionnaires will be sent by email at one and two years after the infusion.

Study Design
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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


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

Patients with documented cerebral palsy, hypoxic brain injury, stroke, hydrocephalus, apraxia or other brain injuries will be eligible. In addition, patients who have competed participation in a prior Duke cell therapy study (CP-AC, Duke ACT, CPSib, ACCeNT-CP, TACT, IMPACT) and who have an eligible/qualified autologous or sibling cord blood unit are eligible. To be eligible, patients must:

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 outside of participation in a Duke clinical trial
  10. Need for mechanical ventilation or chronic O2 support
  11. Unstable airway
  12. Eligible for an active clinical trial of cellular therapy at Duke. If previously enrolled on another Duke cell therapy protocol the subject can still be considered for enrollment on this study after the follow-up period for the clinical trial is completed.
  13. Pregnant or breastfeeding

Umbilical Cord Blood Criteria

Patient enrollment is dependent on the availability of a banked unit of autologous or full or partially (at least half) matched allogeneic sibling CB that has been stored at a private or public bank and meets the following criteria:

Precryopreservation:

  1. TNCC ≥2x107/kg
  2. Sterility cultures performed and negative
  3. Viability ≥70%
  4. At least haploidentical HLA match for sibling units
  5. Donor screening testing performed and negative

CBU Test sample

  1. Segment or test vial available
  2. Identity confirmed via HLA typing of test sample and donor
  3. Viability testing recommended, but not required
Contacts and Locations
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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
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Contact: Sydney Crane, RN 9196681102 cordbloodtherapyinfo@dm.duke.edu
Contact: Lettie Moore cordbloodtherapyinfo@dm.duke.edu

Locations
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United States, North Carolina
Duke University Medical Center Available
Durham, North Carolina, United States, 27705
Contact: Sydney Crane, RN       cordbloodtherapyinfo@dm.duke.edu   
Contact: Lettie Moore       cordbloodtherapyinfo@dm.duke.edu   
United States, Texas
MD Anderson Available
Houston, Texas, United States, 77030
Contact: LaTarsha Williams    713-563-0524    ldwilliams1@mdanderson.org   
Principal Investigator: Kris Mahadeo, MD         
Sponsors and Collaborators
Joanne Kurtzberg, MD
Investigators
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Principal Investigator: Joanne Kurtzberg Duke University
Principal Investigator: Jessica Sun Duke University
More Information
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Responsible Party: Joanne Kurtzberg, MD, Professor of Pediatrics, Duke University
ClinicalTrials.gov Identifier: NCT03327467    
Other Study ID Numbers: Pro00083888
First Posted: October 31, 2017    Key Record Dates
Last Update Posted: December 5, 2022
Last Verified: December 2022
Additional relevant MeSH terms:
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Brain Injuries
Cerebral Palsy
Hydrocephalus
Apraxias
Brain Ischemia
Hypoxia-Ischemia, Brain
Hypoxia
Drowning
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Craniocerebral Trauma
Trauma, Nervous System
 Wounds and Injuries
Pathologic Processes
Brain Damage, Chronic
Signs and Symptoms, Respiratory
Psychomotor Disorders
Neurobehavioral Manifestations
Neurologic Manifestations
Death
Cerebrovascular Disorders
Vascular Diseases
Cardiovascular Diseases
Hypoxia, Brain