Open Label Phase I hCT-MSC in Toddlers With Autism Spectrum Disorder
Condition: Autism Spectrum Disorder
MeSH terms: Autistic Disorder; Autism Spectrum Disorder; Child Development Disorders, Pervasive
Study type: Interventional
Phase: Phase 1

Summary: 
      This is a single site, prospective study of one intravenous infusion of human umbilical cord
      tissue-derived mesenchymal stromal cells (hCT-MSC) in toddlers with autism spectrum disorder
      (ASD). Toddlers 18 to 48 months of age with a confirmed diagnosis of ASD will be eligible to
      participate. Diagnosis will be confirmed at the time of the eligibility visit at the Duke
      Center for Autism and Brain Development. All participants will receive a single intravenous
      dose of 2x106/kg hCT-MSC per kilogram at baseline. Assessments will be conducted at baseline
      and 6 months, with remote follow-up assessments at 12 months.
    

Detailed description: 
      The primary purpose of this study is to evaluate safety and feasibility. Safety assessments
      include monitoring of acute infusion reactions, adverse events, incidence of infections, and
      markers of alloimmunization. Clinical outcome measures will also be described. A key clinical
      outcome measeure is the change in social communication abilities from baseline to 6 months
      based on the Joint Engagement Rating Inventory (JERI), a commonly-used and well-validated
      coding system for rating the quality and quantity of social communication skills in toddlers
      with and without ASD.91 JERI coding rates social communication abilities on a 1 to 7 scale
      and factors in both the quantity and quality of skills. The total joint engagement score as
      well as ratings on all JERI subscales that comprise the total score will be described.

      Other clinical endpoints will include the PDD Behavior Inventory (PDDBI) autism composite
      score, the mean of the Socialization Subscale Standard Score and Communication Subscale
      Standard Score on the Vineland Adaptive Behavior Scales (VABS-3), the Clinical Global
      Impression Scale (CGI) - Severity and Improvement Scales, the Communicative Development
      Inventories (CDI-2): Words & Sentences subscales, attention abilities via eye-tracking, and
      brain activity.

      Exploratory clinical endpoints will include autism symptoms measured by an app that elicits
      and records autism symptoms on an iPad (SenseToKnow), Autism Diagnostic Observation Scale
      (ADOS-2) Calibrated Severity Score (overall, social affect, and repetitive behavior), PDD
      Behavior Inventory (PDDBI) Subscales, and VABS-3 Standard Score and age equivalent for the
      following subscales: Socialization, Communication, and Daily Living and the Standard Score
      and age equivalent for the VABS-3 Adaptive Behavior Composite.

      Safety and VABS-3 assessments will also be conducted remotely at three and 12 months.
      Duration of study participation will be 12 months from the time of the baseline infusion.
    

Intervention name(s): hCT-MSC infusion
Intervention type(s): Biological
Intervention description(s): This is a single site, phase I, open-label, prospective study of one intravenous infusion of human umbilical cord tissue-derived mesenchymal stromal cells (hCT-MSC) in 12 toddlers 18 to 48 months of age with autism spectrum disorder (ASD).
Intervention model: Single Group Assignment
Masking: None (Open Label)
Primary purpose: Treatment
Keywords: Autism Spectrum Disorder; ASD; Autism; PDD