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

January 22, 2026 updated by: Joanne Kurtzberg, MD
This protocol is designed to enable access to intravenous infusions of banked umbilical cord blood (CB), that is thawed and not more than minimally manipulated, 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 mechanism of action is through paracrine signaling of cord blood monocytes inducing endogenous cells to repair existing damage.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Available

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Detailed Description

The primary purpose of this protocol is to enable access to autologous (a person's own) or allogeneic (someone else's) umbilical cord blood infusion for children with various brain disorders. Children with cerebral palsy, hydrocephalus, hypoxic brain injury, stroke, apraxia, and other brain injuries may be eligible if they do not qualify for or are unable to participate in another active clinical trial at Duke. For the purpose of this protocol the term children refers to patients less than 26 years of age. Cord blood infusions are still being studied to determine if they are effective in helping to treat certain types of brain injuries.

If being seen at Duke, 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". 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 and unrelated donor cord blood infusions, the intended recipient and their sibling must be at least a half HLA match, and in the case of an unrelated donor, a 4/6 match.

If eligible, the participant will come to the hospital 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. On the day of infusion, CB cells will be thawed and transported to the Pediatric clinic for infusion to the participant under the supervision of the treatment team and/or clinic staff. A peripheral IV will be placed. Some children may require numbing cream and/or intranasal or oral versed prior to IV placement. Prior to the infusion of cells, premedications (Benadryl, Solumedrol) will be administered. The CB will be infused over approximately 5-15 minutes using standard practices. The participant will receive maintenance IV fluids and be observed in the clinic for a minimum of thirty minutes after the infusion. Participants will be discharged from clinic after at least thirty minutes, providing all vital signs are at their baseline and they are awake, taking oral fluids, and asymptomatic with no evidence of toxicity.

If a participant has evidence of illness on the day of planned infusion, including but not limited to fever, vomiting, diarrhea, or respiratory distress, the infusion will be postponed.

In the event that a participant develops signs or symptoms of anaphylaxis including rash, difficulty breathing, cough, wheezing, or vomiting during their CB infusion, the infusion will be terminated or slowed and appropriate medical therapy initiated.

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.

Required Evaluations:

  1. Screening:

    1. blood counts, blood chemistries, participant HLA typing, blood type (for females receiving allogeneic CB units)
    2. confirmatory HLA typing of the cord blood unit

  2. Baseline Visit:

    1. History and Physical Exam
    2. Pregnancy test (blood test for menstruating females)
    3. blood counts, blood chemistries, if not performed during screening or within 12 months prior to the baseline visit
    4. infectious disease testing, blood type, test for antibodies against donor blood type (for those receiving allogenic cord blood)

  3. Safety Assessments

    1. phone call the day after the infusion
    2. email safety survey at 1 and 2 years after the infusion

Multiple CB infusions may be administered from the same or two unique CB donors, but subsequent infusions will depend upon the quality and availability of an autologous or sibling cord blood unit and the need to limit exposure of the participant to new HLA antigens.

  1. Donor source(s):

    A participant may receive an infusion from a maximum of two non-self donors in the following combinations:

    • Two sibling donors
    • One sibling and one unrelated donor (in any sequence)

    If a participant receives an infusion from a second non-self donor, the second sibling or unrelated donor must be a full 6/6 HLA match with either the recipient and/or the first non-self donor.

    Two unique unrelated donors is NOT permissible.

  2. Each cord blood unit used for infusion must meet the cord blood and donor screening criteria.
  3. A single cord blood unit may be used for multiple sequential infusions if there are enough cells available.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Expanded Access

Expanded Access Type

Expanded Access Type

Describes the category of expanded access under U.S. Food and Drug Administration (FDA) regulations. There are three types of expanded access:
  • Individual Patients: Allows a single patient, with a serious disease or condition who cannot participate in a clinical trial, access to a drug or biological product that has not been approved by the FDA. This category also includes access in an emergency situation.
  • Intermediate-size Population: Allows more than one patient (but generally fewer patients than through a Treatment IND/Protocol) access to a drug or biological product that has not been approved by the FDA. This type of expanded access is used when multiple patients with the same disease or condition seek access to a specific drug or biological product that has not been approved by the FDA.
  • Treatment IND/Protocol: Allows a large, widespread population access to a drug or biological product that has not been approved by the FDA. This type of expanded access can only be provided if the product is already being developed for marketing for the same use as the expanded access use.
  • Treatment IND/Protocol

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Contact Backup

Study Locations

  • United States
    • New Jersey
      • New Brunswick, New Jersey, United States, 08901
        • Available
        • Rutgers Cancer Institutute
    • North Carolina
      • Durham, North Carolina, United States, 27705
        • Available
        • Duke University Medical Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

No older than 22 years (Child, Adult)

Accepts Healthy Volunteers

N/A

Description

Under this protocol, participants may be eligible for infusion of autologous, sibling, and/or unrelated donor CB cells (Duke only). Some eligibility criteria vary based on CB source. For all participants, eligibility is predicated on the availability of a qualifying CB unit To be eligible, patients must:

Inclusion Criteria:

  1. Age at Consent:

    1. Autologous: 0-26 years
    2. Sibling: 6 months - 26 years
    3. Unrelated Donor (Duke only): 6 months - 26 years

  2. Diagnosis

    1. Autologous and Sibling: Cerebral palsy, Hypoxic brain injury, Stroke, Hydrocephalus, Apraxia (without autism), Other brain injury
    2. Unrelated Donor (Duke only): Motor impairment secondary to: Hypoxic brain injury, Periventricular leukomalacia, Stroke/bleed, Congenital hydrocephalus

  3. Functional Status:

    1. Autologous and Sibling: See exclusion criteria
    2. Unrelated Donor (Duke only): GMFCS level I-IV (age ≥2)
  4. No clinically significant abnormalities in blood counts and basic chemistries for age.

  5. Absolute Lymphocyte Count:

    • 1200 for African American participants
    • 1500 for all other participants

  6. Availability of a qualified autologous or Sibling CB Unit

    1. Autologous or Sibling: available
    2. Unrelated Donor (Duke only): N/A
  7. Written informed consent obtained from the parent or legal guardian.

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 participant may still be considered for enrollment on this study after the follow-up period for the clinical trial is completed.
  13. Cerebral palsy secondary to infection, congenital or otherwise, without another known cause
  14. Pregnant or breastfeeding
  15. Treated with autologous cellular therapy through a non-intravenous route (ie. intrathecally) within one year prior to enrollment.

Umbilical Cord Blood Criteria

Participant enrollment is dependent on the availability of a banked unit of autologous or sibling CB that has been stored at a private or public bank, or, if enrolling at Duke, availability of a publicly banked unrelated donor unit. Units must meet 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 and at least a 4/6 HLA match for unrelated donor 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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Joanne Kurtzberg, MD

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Principal Investigator: Joanne Kurtzberg, Duke University
  • Principal Investigator: Jessica Sun, Duke University

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
October 25, 2017

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
October 25, 2017

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
October 31, 2017

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
January 23, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
January 22, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
August 1, 2025

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • Pro00083888

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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