Cord Blood Infusion for Children With Autism Spectrum Disorder

Inclusion Criteria

• Age ≥ 2 years to ≤ 7 years (7 years, 364 days) at the time of visit 1
• Confirmed clinical DSM-5 diagnosis of Autism Spectrum Disorder using the DSM-5 Checklist
• Fragile X testing performed and negative
• Available and qualified umbilical cord blood unit with a minimum banked total nucleated cell dose of ≥ 2.5 x 107 cells/kg that meets criteria outlined in Section 6.0, either:
• Autologous umbilical cord blood unit OR
• ≥4/6 HLA-matched and ABO/Rh-matched allogeneic unrelated umbilical cord blood unit from the Carolinas Cord Blood Bank
• Stable on current psychiatric medication regimen (dose and dosing schedule) for at least 2 months prior to infusion of study product
• Normal absolute lymphocyte count (≥1500/uL)
• Participant and parent/guardian are English speaking
• Able to travel to Duke University two times (baseline and 6 months post-baseline), and parent/guardian is able to participate in interim surveys and interviews
• Parental consent

Exclusion Criteria

• General:
• Review of medical records indicates ASD diagnosis not likely
• Known diagnosis of any of the following coexisting psychiatric conditions: depression, bipolar disorder, schizophrenia, obsessive compulsive disorder, Tourette syndrome
• Screening data suggests that participant would not be able to comply with the requirements of the study procedures, including study outcome measures, as assessed by the study team
• Family is unwilling or unable to commit to participation in all study-related assessments, including follow up for approximately 12 months
• Sibling is enrolled in this (DukeACT) study
• Genetic:
• Records indicate that child has a known genetic syndrome such as (but not limited to) Fragile X syndrome, neurofibromatosis, Rett syndrome, tuberous sclerosis, PTEN mutation, cystic fibrosis, muscular dystrophy b. Known pathogenic copy number variation (CNV) associated with ASD (e.g., 16p11.2, 15q13.2, 2q13.3)
• Infectious:
• Known active central nervous system infection
• Evidence of uncontrolled infection based on records or clinical assessment
• HIV positivity
• Medical:
• Known metabolic disorder
• Known mitochondrial dysfunction
• History of unstable epilepsy or uncontrolled seizure disorder, infantile spasms, Lennox Gastaut syndrome, Dravet syndrome, or other similar chronic seizure disorder
• Active malignancy or prior malignancy that was treated with chemotherapy
• History of a primary immunodeficiency disorder
• History of autoimmune cytopenias (i.e., ITP, AIHA)
• Coexisting medical condition that would place the child at increased risk for complications of sedation or other study procedures
• Concurrent genetic or acquired disease or comorbidity(ies) that could require a future stem cell transplant
• Significant sensory (e.g., blindness, deafness, uncorrected hearing impairment) or motor (e.g., cerebral palsy) impairment
• Impaired renal or liver function as determined by serum creatinine >1.5mg/dL or total bilirubin >1.3mg/dL, except in patients with known Gilbert's disease
• Significant hematologic abnormalities defined as: Hemoglobin 2 weeks, and no systemic steroids within 3 months prior to enrollment. Topical and inhaled steroids are permitted.