hCT-MSC for Autism Spectrum Disorder

(IMPACT Trial)

Recruiting in Durham (>99 mi)

Overseen By Kerry Hoyle

Age: < 18

Sex: Any

Trial Phase: Phase 2

Sponsor: Joanne Kurtzberg, MD

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 4

Duration: 6 months

137 Participants Needed

Trial Summary

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.

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. ^{1 2 3 4 5}

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. ^{1 2 3 5 6}

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. ^{1 2 3 6 7}

Research Team

Beth Shaz, MD

Principal Investigator

Duke University

Lauren Franz, MBChB

Principal Investigator

Duke University

Eligibility Criteria

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

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.

Timeline

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)

Treatment Details

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.

Participant Groups

Group I: MSC Experimental Treatment 1 Intervention

One dose of 6x10e6 cells/kg administered intravenously.

Group II: Placebo Infusion Placebo Group 1 Intervention

Placebo infusion

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+

References

1. England pubmed.ncbi.nlm.nih.gov

Infusion of human umbilical cord tissue mesenchymal stromal cells in children with autism spectrum disorder. [2021]

2. England pubmed.ncbi.nlm.nih.gov

Transplantation of human cord blood mononuclear cells and umbilical cord-derived mesenchymal stem cells in autism. [2021]

3. England pubmed.ncbi.nlm.nih.gov

Stem cell therapy for autism. [2018]

4. England pubmed.ncbi.nlm.nih.gov

Mesenchymal stem cells in autism spectrum and neurodevelopmental disorders: pitfalls and potential promises. [2019]

5. England pubmed.ncbi.nlm.nih.gov

Allogeneic Human Umbilical Cord Mesenchymal Stem Cells for the Treatment of Autism Spectrum Disorder in Children: Safety Profile and Effect on Cytokine Levels. [2021]

6. United States pubmed.ncbi.nlm.nih.gov

Efficacy of fetal stem cell transplantation in autism spectrum disorders: an open-labeled pilot study. [2014]

7. Australia pubmed.ncbi.nlm.nih.gov

Intrathecal autologous bone marrow stem cell therapy in children with autism: A randomized controlled trial. [2021]