Phase 1
Completed N=12
Open Label Phase I hCT-MSC in Toddlers With Autism Spectrum Disorder
Source: ClinicalTrials.gov NCT04294290 ↗Enrolled (actual)
12
Serious AEs
0.0%
Results posted
Feb 2025
Primary outcomePrimary: Safety of hCT-MSC Infusion as Measured by Total Number of Infusion Reactions — 1 infusion reactions
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.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Safety of hCT-MSC Infusion as Measured by Total Number of Infusion Reactions |
1 | — |
| PRIMARY Safety of hCT-MSC Infusion as Measured by Severity of Infusion Reactions |
1 | — |
| PRIMARY Safety of hCT-MSC Infusion as Measured by Total Number of Product-related Infections |
— | — |
| PRIMARY Safety of hCT-MSC Infusion as Measured by Severity of Product-related Infections |
— | — |
| PRIMARY Safety of hCT-MSC Infusion as Measured by Number of Participants With Evidence of Alloimmunization Via Anti-HLA Antibodies |
2 | — |
| PRIMARY Safety of hCT-MSC Infusion as Measured by Total Number of Graft vs. Host Disease (GVHD) Instances |
— | — |
| PRIMARY Safety of hCT-MSC Infusion as Measured by Severity of Graft vs. Host Disease (GVHD) |
— | — |
| PRIMARY Safety of hCT-MSC Infusion as Measured by Total Number of Unexpected Adverse Events |
— | — |
| PRIMARY Safety of hCT-MSC Infusion as Measured by Severity of Unexpected Adverse Events |
— | — |
| PRIMARY Change in Pervasive Developmental Disorder Behavior Inventory Autism Composite Score (PDDBI) |
-5.00 | — |
| PRIMARY Change in VABS-3 (Vineland Adaptive Behavior Scales) Socialization and Communication Composite Score |
3.83 | — |
| PRIMARY Number of Participants Who Improved (Much Improved or Minimally Improved) on the Clinical Global Impression Scale (CGI) |
8 | — |
| PRIMARY Change in Communicative Development Inventories (CDI-2) |
75.92 | — |
| PRIMARY Change in Attention Abilities as Assessed Via Eye-tracking |
-0.03 | — |
| PRIMARY Change in Brain Activity as Measured by EEG |
— | — |
Eligibility Criteria
Inclusion Criteria
- Age ≥ 18 months to ≤ 48 months (48 months, 29 days) at the time of consent
- Confirmed clinical DSM-5 diagnosis of Autism Spectrum Disorder using the DSM-5 Checklist as informed by the Autism Diagnostic Observation Schedule - 2.
- Fragile X testing performed and negative; CMA and/or whole exome sequencing performed and results not linked to autism diagnosis
- Stable on current psychoactive 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 for two multi-day visits (baseline and six months) 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
- Screening data suggests that participant would not be able to comply with the requirements of the study procedures as assessed by the study team
- Family is unwilling or unable to commit to participation in all study-related assessments, including protocol follow up
- Sibling is enrolled in this (Duke hCT-MSC) 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 or a genetic mutation known to be associated with ASD
- Known pathogenic mutation or 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
- Known 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 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.
Data sourced from ClinicalTrials.gov (NCT04294290). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.