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

Sponsor

Joanne Kurtzberg, MD (Other)

Overall Status

Available

CT.gov ID

NCT03327467

Collaborator

(none)

Locations

Study Details

Study Description

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	Phase
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.

Study Design

Study Type:

Expanded Access

Official Title:

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

Outcome Measures

Primary Outcome Measures

Eligibility Criteria

Criteria

Ages Eligible for Study:

N/A to 26 Years

Sexes Eligible for Study:

All

Inclusion Criteria:

Have a qualifying sibling or autologous cord blood unit
Be < 26 years of age at the time of consent.
Have given written informed consent according to FDA guidelines (or consent of parent/legal guardian as applicable).
Have baseline blood counts and basic chemistries within normal for age.
Have a normal absolute lymphocyte count (ALC) for age on baseline blood count and differential.

Exclusion Criteria:

Documented HIV or Hepatitis or other disease transmittable through the blood.
A cord blood unit that fails to meet specifications
Refusal of consent
Uncontrolled seizure disorder
Uncontrolled infection
Diagnosed with a genetic or metabolic disorder related to the neurologic condition
History of an immune deficiency
History of treatment with chemo or immunosuppressive therapy
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
Need for mechanical ventilation or chronic O2 support
History of inability to protect the airway
Eligibility for an active clinical trial of cellular therapies at Duke