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

Pro00083888

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.

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Under this protocol, participants may be eligible for infusion of autologous, sibling, and/or unrelated donor CB cells. 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: 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: 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: GMFCS level I-IV (age ≥2) 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. 6. Availability of a qualified autologous or Sibling CB Unit 1. Autologous or Sibling: available 2. Unrelated Donor: not available 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. Pregnant or breastfeeding 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 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

{'studyType': 'EXPANDED_ACCESS', 'expandedAccessTypes': {'treatment': True}}

2024-06-07