hCT-MSC in Children With Autism Spectrum Disorder (NCT04089579) | Clinical Trial Compass
CompletedPhase 2
hCT-MSC in Children With Autism Spectrum Disorder
United States137 participantsStarted 2020-10-12
Plain-language summary
The purpose of this Phase II study is to determine the efficacy of human umbilical cord tissue-derived mesenchymal stromal cells (hCT-MSC) for improving social communication abilities in children with autism spectrum disorder (ASD).
Who can participate
Age range
4 Years – 11 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Age ≥ 4 years to \< 12 years (11 years, 364 days) at the time of consent
. Confirmed clinical DSM-5 diagnosis of Autism Spectrum Disorder using the DSM-5 Checklist as informed by the Brief Observation of Symptoms of Autism (BOSA) and the Autism Diagnostic Interview-Revised (ADI-R)
. Fragile X testing performed and negative; CMA and/or whole exome sequencing performed and results not linked to autism diagnosis
. 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 (≥1200/uL for African American participants and ≥1500/uL for all other participants)
. GAI ≥ 65 via cognitive testing by study personnel
. Participant and parent/guardian are English speaking
. Able to travel to Duke University two times (baseline, six months), and parent/guardian is able to participate in interim surveys and interviews
Exclusion criteria
. General:
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Change on the Average Socialization and Communication Subscale Standard Scores on the Vineland Behavior Scales (VABS-3)
. Review of medical records and/or screening assessments indicates ASD diagnosis and/or GAI \> 65 not confident
. Known diagnosis of any of the following coexisting psychiatric conditions: depression, bipolar disorder, schizophrenia, obsessive compulsive disorder associated with bipolar disorder, Tourette syndrome
. 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 IMPACT) 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 defect definitively known to be associated with ASD