Treatment of Children With Autistic Spectrum Disorder With Autologous Umbilical Cord Blood, a Pilot Study

This is an interventional treatment trial for Autistic Spectrum Disorder focused on measuring Autistic Spectrum Disorder, autologous umblical cord blood Read More

Inclusion Criteria:

• Age ≥ 1.5 years to ≤ 12 years (11 years, 364 days) at the time of visit 1
• Confirmed clinical DSM-5 diagnosis of Autism Spectrum Disorder using the DSM-5 criteria
• Fragile X testing performed and negative
• Available and qualified umbilical cord blood unit with a minimum banked total nucleated cell dose of ≥ 2 x 10e7 cells/kg
• 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)
• Able to travel to Sheba Medical Center University three times (baseline, 6 and 12 months post-baseline), and parent/guardian is able to participate in interim surveys and interviews monthly
• 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
  • 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

• 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
  • Known pathogenic copy number variation (CNV) associated with ASD (e.g., 16p11.2, 15q13.2, 2q13.3)

• Infectious:

  • Known active CNS 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, Lennox Gastaut syndrome, Dravet syndrome, or other similar epileptic encephalopathy
  • 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
  • Evidence of clinically relevant physical dysmorphology indicative of a genetic syndrome as assessed by the PIs or other investigators, including a medical geneticist and psychiatrists trained in identifying dysmporphic features associated with neurodevelopmental conditions.

• Current/Prior Therapy:

  • History of prior cell therapy
  • Current or prior use of IVIG or other anti-inflammatory medications with the exception of NSAIDs
  • 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.