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AutismPhase 2IMPACTNCT04089579

hCT-MSC in Children With Autism Spectrum Disorder

A Phase 2 study testing Cord Tissue Mesenchymal Stromal Cells in people with Autism and Autism Spectrum Disorder, run by Joanne Kurtzberg, MD at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial 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). Read more about this study

Who is it for?
Ages 4 to 11 years
Conditions the study is for
Autism, Autism Spectrum Disorder
What is being tested?
Cord Tissue Mesenchymal Stromal Cells
Type of treatment
Injection or infusion
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
137 participants
Where
1 siteUnited States
Study dates
12 Oct 2020to 1 Feb 2024
Phase
Phase 2
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 4 to 11 years
  • All sexes
  • No healthy volunteers
  • Child

Who may be able to join

  • 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
Show all 9Show fewer
  • 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
  • Parental/guardian consent from at least one parent/guardian

Who cannot join

  • General:
  • 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
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  • 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
  • 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
  • Exposure to COVID-19 in the preceding 14 days or positive COVID-19 test in the previous 28 days. Subjects with a past history of infection with COVID-19 must be symptom-free for 14 days prior to the initial visit.
  • 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 <10.0 g/dL, Platelets <150 x 10e9/uL, WBC <3,000 cells/mL, ALC <1200/uL for African Americans or <1500/uL for all other participants.
  • Evidence of clinically relevant physical dysmorphology indicative of a genetic syndrome as assessed by the PIs or other investigators, including a medical geneticist and psychiatrists trained in identifying dysmorphic features associated with neurodevelopmental conditions.
  • Current/Prior Therapy:
  • Availability of a banked, qualified autologous cord blood unit or parents deferred use of qualified, autologous cord blood unit b. History of prior cell therapy c. Current or prior use of IVIG or other anti-inflammatory medications with the exception of NSAIDs d. Current or prior immunosuppressive therapy i. No systemic steroid therapy that has lasted >2 weeks, and no systemic steroids within 3 months prior to enrollment. Topical and inhaled steroids are permitted.

Trial locations and contacts

1 site in 1 country.

United States1 site
  • Duke University Medical Center

    Durham, North Carolina, 27705

Other trials in United States

About this study

Official title: A Phase II Study of hCT-MSC, an Umbilical Cord-Derived Mesenchymal Stromal Cell Product, in Children With Autism Spectrum Disorder

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).

Read the detailed description

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), administered in two different dosing strategies, in children with autism spectrum disorder (ASD).

This study will be enrolling children with ASD, aging 4-11 years of age. Qualifying subjects will undergo neuropsychological evaluation, EEG testing, eye tracking, CVA assessments, and infusion of study product. Subjects will be randomized to one of two study arms; 1) a single infusion of 6.0x106 cells/Kg at baseline, followed by a blinded placebo infusion at six months or, 2) Placebo infusion at baseline, followed by an intravenous dose of 6x106 cells/Kg at six months. The second infusion was included primarily as an incentive to ensure that all participants ultimately receive an infusion of MSC, to reduce the risk of unblinding, and to encourage study completion. Efficacy outcome measures were only evaluated 6 months before the second infusion. Safety outcomes were assessed during the entire 12 months of the study.

The primary endpoint of this study is the change in social communication skill from baseline to six months. The potential risks associated with infusion of MSCs include a reaction to the product (rash, shortness of breath, wheezing, difficulty breathing, hypotension, swelling around the mouth, throat or eyes, tachycardia, diaphoresis), transmission of infection, and HLA sensitization.

Being tested
Cord Tissue Mesenchymal Stromal Cells, Placebo Infusion
Keywords
Autism, Autism Spectrum Disorder, Stem cell, Mesenchymal Stromal Cells
Medical subject headings
Autistic Disorder, Autism Spectrum Disorder

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Cord Tissue Mesenchymal Stromal CellsBiological

    Human Umbilical Cord Tissue Derived Mesenchymal Stromal Cells (hCT-MSC), isolated and expanded from umbilical cord tissue from allogeneic unrelated donors. One dose of 6x10e6 cells/kg administered intravenously.

  • Placebo InfusionOther

    Placebo comparative infusion

Study groups in NCT04089579
Study groupRoleWhat they receive
MSCExperimentalOne dose of 6x10e6 cells/kg administered intravenously.
Receives: Biological: Cord Tissue Mesenchymal Stromal Cells
Placebo InfusionPlacebo comparatorPlacebo infusion
Receives: Other: Placebo Infusion

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Change on the Average Socialization and Communication Subscale Standard Scores on the Vineland Behavior Scales (VABS-3)

    The primary outcome measure is the mean of the Socialization and Communication Subscale Standard Scores on the Vineland Adaptive Behavior Scales (VABS-3) from the Comprehensive Interview form. The primary endpoint is the change in this outcome measure from baseline to six months before the second infusion. A positive change in the scores indicates an improvement in socialization and communication. A standard score of 100 is the mean with a standard deviation of 15 points. A score of 100 should be understood as being similar to the typical population of the same age. Scores greater than or equal to 86 are considered adequate or above adequate. Domain scores less than or equal to 85 are considered moderately low to low and indicate the patient has a significant skill deficit when compared with similarly aged peers.

    Measured: Baseline, 6 months

Secondary outcomes (5)
  1. Change in VABS-3 (Vineland Adaptive Behavior Scales) Socialization Standard Score

    The change in the Socialization Subscale Standard Scores on the Vineland Adaptive Behavior Scales (VABS-3) from baseline to six months. Higher Socialization Standard scores indicate greater socialization. A positive change in the scores indicates an improvement in socialization. A standard score of 100 is the mean with a standard deviation of 15 points. A score of 100 should be understood as being similar to the typical population of the same age. Scores greater than or equal to 86 are considered adequate or above adequate. Domain scores less than or equal to 85 are considered moderately low to low and indicate the patient has a significant skill deficit when compared with similarly aged peers.

    Measured: Baseline, 6 months

  2. Change in VABS-3 Communication Standard Score

    The change in the Communication Standard Scores on the Vineland Adaptive Behavior Scales (VABS-3) from the Comprehensive Interview form from baseline to six months before the second infusion. Higher scores indicate greater communication. A positive change in the scores indicates an improvement in communication. A standard score of 100 is the mean with a standard deviation of 15 points. A score of 100 should be understood as being similar to the typical population of the same age. Scores greater than or equal to 86 are considered adequate or above adequate. Domain scores less than or equal to 85 are considered moderately low to low and indicate the patient has a significant skill deficit when compared with similarly aged peers.

    Measured: Baseline, 6 months

  3. CGI-S (Clinical Global Impression - Severity of Illness) Overall Score

    The CGI-S Overall Score is a 7-point scale that requires the clinician to rate the severity of the participant's overall functioning and symptoms of autism at the time of assessment, relative to the clinician's experience with participants who have the same diagnosis. The clinician rates the severity of autism symptoms - 1, normal, no symptoms; 2, borderline level of symptoms; 3, mild symptoms; 4, moderate symptoms; 5, marked symptoms; 6, severe symptoms; or 7, extremely severe symptoms. The higher ratings indicate greater severity of overall functioning impairment.

    Measured: 6 months

  4. CGI-I (Clinical Global Impression - Improvement) Overall Score

    The CGI-I Overall Score is a 7-point scale that requires the clinician to assess how much the participant's autism overall functioning and symptoms have improved or worsened relative to a baseline assessment. The symptoms are rated as: 1, very much improved; 2, much improved; 3, minimally improved; 4, no change; 5, minimally worse; 6, much worse; or 7, very much worse. The lower scores indicate greater improvement.

    Measured: 6 months

  5. Change in the Pediatric Quality of Life (PedsQL) Total Scale Score

    The PedsQL 4.0 Generic Core Scales is a 5-minute parent questionnaire that measures the child's functioning in the dimensions of physical, emotional, social, and school. The items use a Likert rating scale from 0 (Never) to 4 (Almost Always). This 0-4 scale is then transformed to 0=100, 1=75, 2=50, 3=25, and 4=0 for a reverse score. The Total Scale Score is then computed as the sum of all the items over the number of items answered on all the Scales for a total score range of 0 to 100. Higher scores indicate a better quality of life.

    Measured: Baseline, 6 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted13 Sep 2019
  2. Study start12 Oct 2020
  3. Primary completion2 May 2023
  4. Study completion1 Feb 2024

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Model description
This study is a phase II, prospective, randomized, blinded clinical trial designed to assess the efficacy of intravenous dosing of hCT-MSC for improving social communication abilities in young children with ASD. All participants will ultimately be treated with hCT-MSC. Participants randomized to arm A will each receive a single intravenous dose of 6x106 hCT-MSC per kilogram at baseline, followed by a placebo infusion at six months. Participants randomized to arm B will each receive a placebo infusion at baseline, followed by an intravenous dose of 6x106 hCT-MSC per kilogram at six months.
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
137 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04089579
Sponsor's protocol ID
Pro00113011
Other IDs
Pro00102894
Acronym
IMPACT
Status last verified
May 2026
First submitted
12 Sep 2019
Last updated
24 Jun 2026
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Publications

  • Chatham CH, Taylor KI, Charman T, Liogier D'ardhuy X, Eule E, Fedele A, Hardan AY, Loth E, Murtagh L, Del Valle Rubido M, San Jose Caceres A, Sevigny J, Sikich L, Snyder L, Tillmann JE, Ventola PE, Walton-Bowen KL, Wang PP, Willgoss T, Bolognani F. Adaptive behavior in autism: Minimal clinically important differences on the Vineland-II. Autism Res. 2018 Feb;11(2):270-283. doi: 10.1002/aur.1874. Epub 2017 Sep 21.

    PubMed 28941213

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Cord Tissue Mesenchymal Stromal Cells in people with Autism and Autism Spectrum Disorder, run by Joanne Kurtzberg, MD at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 9 inclusion and 29 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 24 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 24 Jun 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Change on the Average Socialization and Communication Subscale Standard Scores on the Vineland Behavior Scales (VABS-3) (time frame: Baseline, 6 months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04089579, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Jun 2026.

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