137 Participants Needed

hCT-MSC for Autism Spectrum Disorder

(IMPACT Trial)

AB
KH
Overseen ByKerry Hoyle
Prior Safety DataThis treatment has passed at least one previous human trial

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial is testing if special cells from umbilical cords can help children with autism improve their social communication skills by potentially enhancing brain function. Previous studies have shown that umbilical cord blood infusions are safe and may improve social functioning and communication abilities in children with autism.

Do I need to stop my current medications for the trial?

The trial requires that participants have been stable on their current psychiatric medications for at least 2 months before starting the study, so you do not need to stop taking them.

Is the use of human umbilical cord tissue mesenchymal stromal cells (hCT-MSCs) safe for humans?

Studies have shown that hCT-MSCs, used in children with autism spectrum disorder, are generally safe and well-tolerated, with only mild or moderate short-term side effects like agitation during infusion. Some participants developed antibodies, but these did not cause any health issues.12345

How does the hCT-MSC treatment for autism differ from other treatments?

The hCT-MSC treatment for autism is unique because it uses mesenchymal stromal cells derived from human umbilical cord tissue to potentially reduce neuroinflammation, which is thought to contribute to autism symptoms. This approach is different from standard behavioral interventions and focuses on modulating immune responses, offering a novel biological treatment option for autism.12346

What data supports the effectiveness of the treatment Cord Tissue Mesenchymal Stromal Cells for Autism Spectrum Disorder?

Some studies suggest that mesenchymal stromal cells (MSCs) from umbilical cord tissue may help reduce inflammation, which is linked to autism symptoms. In small trials, some children with autism showed improvement in specific measures after receiving MSCs, but more research is needed to confirm these findings.13457

Who Is on the Research Team?

BS

Beth Shaz, MD

Principal Investigator

Duke University

LF

Lauren Franz, MBChB

Principal Investigator

Duke University

Are You a Good Fit for This Trial?

This trial is for children aged 4 to less than 12 years with Autism Spectrum Disorder (ASD), who have a normal immune cell count, no genetic conditions linked to ASD, and are stable on current psychiatric medications. They must be English-speaking and able to visit Duke University twice. Children with known genetic syndromes, metabolic disorders, significant sensory or motor impairments, or unstable medical conditions cannot participate.

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
See 6 more

Exclusion Criteria

The child has a diagnosed genetic syndrome like Fragile X syndrome, neurofibromatosis, Rett syndrome, tuberous sclerosis, PTEN mutation, cystic fibrosis, muscular dystrophy, or a known genetic defect linked to autism spectrum disorder (ASD).
You have a known disorder that affects how your body processes and uses nutrients.
You have a condition that weakens your immune system since birth.
See 24 more

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive either a single infusion of hCT-MSC or placebo at baseline, followed by a second infusion at six months

6 months
2 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment, including assessments for adverse events and changes in social communication skills

6 months
2 visits (in-person)

What Are the Treatments Tested in This Trial?

Interventions

  • Cord Tissue Mesenchymal Stromal Cells
Trial Overview The study tests the effectiveness of human umbilical cord tissue-derived mesenchymal stromal cells (hCT-MSC) versus a placebo in improving social communication skills in children with ASD. It's a Phase II trial where participants will receive either the hCT-MSC treatment or a placebo infusion.
How Is the Trial Designed?
2Treatment groups
Experimental Treatment
Placebo Group
Group I: MSCExperimental Treatment1 Intervention
Group II: Placebo InfusionPlacebo Group1 Intervention

Find a Clinic Near You

Who Is Running the Clinical Trial?

Joanne Kurtzberg, MD

Lead Sponsor

Trials
19
Recruited
710+

Cryo-Cell International

Collaborator

Trials
1
Recruited
140+

The Marcus Foundation

Collaborator

Trials
19
Recruited
2,200+

Citations

Infusion of human umbilical cord tissue mesenchymal stromal cells in children with autism spectrum disorder. [2021]
Transplantation of human cord blood mononuclear cells and umbilical cord-derived mesenchymal stem cells in autism. [2021]
Stem cell therapy for autism. [2018]
Mesenchymal stem cells in autism spectrum and neurodevelopmental disorders: pitfalls and potential promises. [2019]
Allogeneic Human Umbilical Cord Mesenchymal Stem Cells for the Treatment of Autism Spectrum Disorder in Children: Safety Profile and Effect on Cytokine Levels. [2021]
Efficacy of fetal stem cell transplantation in autism spectrum disorders: an open-labeled pilot study. [2014]
Intrathecal autologous bone marrow stem cell therapy in children with autism: A randomized controlled trial. [2021]
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