hCT-MSC for Autism Spectrum Disorder

(IMPACT Trial)

Overseen By Kerry Hoyle

Age: < 18

Sex: Any

Trial Phase: Phase 2

Sponsor: Joanne Kurtzberg, MD

Must not be taking: IVIG, Anti-inflammatories, Immunosuppressives, others

Disqualifiers: Depression, Bipolar, Schizophrenia, others

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

Compensation: Varies

Number of Visits: 4

Duration: 6 months

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores whether Cord Tissue Mesenchymal Stromal Cells from umbilical cords can improve social communication in children with autism. The trial includes two groups: one receiving the cell treatment and another receiving a placebo (an inactive substance). It suits children diagnosed with autism who have been stable on their current medication for at least two months. Participants must travel to Duke University for assessments and have a parent available for surveys and interviews. As a Phase 2 trial, the research measures the treatment's effectiveness in an initial, smaller group, allowing participants to contribute to important early findings.

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 there any evidence suggesting that this treatment is likely to be safe for humans?

Research has shown that using cells from human umbilical cord tissue is generally safe and well-tolerated. In past studies, young children received these cells through an IV without serious safety issues. For instance, one study found that this treatment was safe and feasible for children. Another study showed that the infusions were not only safe but also improved symptoms in some children.

Adults have also safely received these treatments. In studies with adults, a single IV dose was well-tolerated. Overall, these studies have reported no major safety concerns, suggesting that the treatment is safe for humans. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for autism?

Researchers are excited about using Cord Tissue Mesenchymal Stromal Cells (MSC) for Autism Spectrum Disorder because this treatment introduces a novel approach by utilizing stem cells to potentially repair and support neural networks in the brain. Unlike standard treatments, which often focus on behavioral therapies and medications to manage symptoms, MSCs have the potential to address the underlying biological aspects of autism by promoting neurogenesis and reducing inflammation. This innovative method could offer a more comprehensive and possibly transformative impact on the condition, providing hope for improved outcomes beyond symptom management.

What evidence suggests that this treatment might be an effective treatment for autism spectrum disorder?

Research has shown that cells from human umbilical cord tissue, known as mesenchymal stromal cells (MSCs), might improve the main symptoms of autism spectrum disorder (ASD). In earlier studies, children with ASD who received these cells demonstrated better social communication skills. Some research suggests that MSCs could reduce inflammation in the brain, which is thought to be linked to ASD. Early findings also indicate that these cells might help repair or protect brain cells. While more research is needed, these early results are promising for using MSCs as a treatment for ASD. In this trial, participants will receive either an MSC infusion or a placebo infusion to evaluate the effectiveness of MSCs in treating ASD. ^{1 2 3 6 7}

Who Is on the Research Team?

Beth Shaz, MD

Principal Investigator

Duke University

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

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 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?

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+

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT03099239

hCT-MSCs for Children With Autism Spectrum Disorder ... ASD-specific outcome measures, described below, will be assessed at baseline and six months from baseline and results will be described. Official Title. A Phase ...

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC9294632/

Possible Effect of the use of Mesenchymal Stromal Cells in the ... Possible effect of the use of mesenchymal stromal cells in the treatment of autism spectrum disorders: a review

3. marcuscenter.duke.edu marcuscenter.duke.edu/clinical-trials/current-trials

Current Trials | Duke Marcus Center for Cellular Cures The purpose of this double blinded Phase II study is to determine the efficacy of human umbilical cord tissue-derived mesenchymal stromal cells (hCT-MSC), ...

4. stemcellsjournals.onlinelibrary.wiley.com stemcellsjournals.onlinelibrary.wiley.com/doi/full/10.1002/sctm.19-0434

Infusion of human umbilical cord tissue mesenchymal stromal ... The ability of hCT-MSC to improve core symptoms of autism is being studied in a subsequent randomized, placebo-controlled clinical trial. 1 ...

5. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12130910/

Mesenchymal Stromal Cell Treatment Alleviates Autism ... Allogeneic mesenchymal stem cells might offer an attractive innovative modality for some children with ASD and may prove a promising therapy for children with ...

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC6766688/

Allogeneic Human Umbilical Cord Mesenchymal Stem ... Umbilical cord mesenchymal stem cell infusions were safe and generally well tolerated. Forty percent of children showed notable improvements of symptoms.

7. clinicaltrials.gov clinicaltrials.gov/study/NCT04484077

hCT-MSC Infusion in Adults With Autism Spectrum Disorder The purpose of the study is to determine the safety and tolerability of a single intravenous dose of Human Umbilical Cord Tissue Derived Mesenchymal Stromal ...