Cord Blood Infusion for Children With Autism Spectrum Disorder

Inclusion Criteria:

1. Age ≥ 2 years to ≤ 7 years (7 years, 364 days) at the time of visit 1

2. Confirmed clinical DSM-5 diagnosis of Autism Spectrum Disorder using the DSM-5
Checklist

3. Fragile X testing performed and negative

4. 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

5. Stable on current psychiatric medication regimen (dose and dosing schedule) for at
least 2 months prior to infusion of study product

6. Normal absolute lymphocyte count (≥1500/uL)

7. Participant and parent/guardian are English speaking

8. 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

9. Parental consent

Exclusion Criteria:

1. 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

2. 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)

3. Infectious:

- Known active central nervous system infection

- Evidence of uncontrolled infection based on records or clinical assessment

- HIV positivity

4. 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 thatwould 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 <10.0 g/dL, White
blood count < 3,000 cells/mL, absolute lymphocyte count <1000/uL, Platelets <150
x 10e9/uL

- Evidence of clinically relevant physical dysmorphology indicative of a genetic
syndrome as assessed by the PIs or other investigators, including a medical
geneticist or psychiatrists trained in identifying dysmorphic features associated
with neurodevelopmental conditions

5. Current/Prior Therapy:

- History of prior cell therapy

- Current or prior use of immune globulins or other anti-inflammatory medications
with the exception of non steroidal anti-inflammatory medications

- Current or prior immunosuppressive therapy

- No systemic steroid therapy that has lasted >2 weeks, and no systemic steroids
within 3 months prior to enrollment. Topical and inhaled steroids are permitted.