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.
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
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- 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
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.
- Conditions
- Autism, Autism Spectrum Disorder
- 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 group | Role | What they receive |
|---|---|---|
| MSC | Experimental | One dose of 6x10e6 cells/kg administered intravenously. Receives: Biological: Cord Tissue Mesenchymal Stromal Cells |
| Placebo Infusion | Placebo comparator | Placebo infusion Receives: Other: Placebo Infusion |
What is being measured
The primary outcome is the main question the study is designed to answer.
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)
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
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
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
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
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.
- First posted13 Sep 2019
- Study start12 Oct 2020
- Primary completion2 May 2023
- Study completion1 Feb 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for AutismStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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)
Sponsor and investigators
- Lead sponsor
- Joanne Kurtzberg, MD
- Sponsor type
- University, hospital or other
- Collaborators
- The Marcus Foundation, Cryo-Cell International
- Responsible party
- Sponsor-investigator, Joanne Kurtzberg, MD, Duke University
- Investigators
- Beth Shaz, MD, Principal investigator, Duke UniversityLauren Franz, MBChB, Principal investigator, Duke University
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
- Results posted
- Yes, on ClinicalTrials.gov
- 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
Study at a glance
From the registry record, last updated by the sponsor on 24 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Study of hCT-MSC, an Umbilical Cord-Derived Mesenchymal Stromal Cell Product, in Children With Autism Spectrum Disorder
- ClinicalTrials.gov ID
- NCT04089579
- Acronym / sponsor ID
- IMPACT / Pro00113011
- Conditions
- Autism, Autism Spectrum Disorder
- Keywords
- Autism, Autism Spectrum Disorder, Stem cell, Mesenchymal Stromal Cells
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 137 participants (actual)
- Lead sponsor
- Joanne Kurtzberg, MDUniversity, hospital or other
- Collaborators
- The Marcus Foundation, Cryo-Cell International
- Study start
- 12 Oct 2020 (actual)
- Primary completion
- 2 May 2023 (actual)
- Study completion
- 1 Feb 2024 (actual)
- First posted
- 13 Sep 2019 (actual)
- Results
- Posted 24 Jun 2026
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
- 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.
| Arm | Type | Receives |
|---|---|---|
| MSC | Experimental | Biological: Cord Tissue Mesenchymal Stromal CellsOne dose of 6x10e6 cells/kg administered intravenously. |
| Placebo Infusion | Placebo comparator | Other: Placebo InfusionPlacebo infusion |
Who may qualify
- Ages
- 4 to 11 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (9 listed)
- 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)
Key exclusion criteria (29 listed)
- General:
- Genetic:
- Infectious:
- Medical:
- Current/Prior Therapy:
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Cord Tissue Mesenchymal Stromal CellsHuman 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. | Biological | MSC |
| Placebo InfusionPlacebo comparative infusion | Other | Placebo Infusion |
Primary outcome
Change on the Average Socialization and Communication Subscale Standard Scores on the Vineland Behavior Scales (VABS-3)
Time frame: Baseline, 6 months
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.
5 secondary outcomes
Change in VABS-3 (Vineland Adaptive Behavior Scales) Socialization Standard Score
Time frame: Baseline, 6 months
Change in VABS-3 Communication Standard Score
Time frame: Baseline, 6 months
CGI-S (Clinical Global Impression - Severity of Illness) Overall Score
Time frame: 6 months
CGI-I (Clinical Global Impression - Improvement) Overall Score
Time frame: 6 months
Change in the Pediatric Quality of Life (PedsQL) Total Scale Score
Time frame: Baseline, 6 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Jun 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 28941213Chatham 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.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Beth Shaz, MD principal investigator
Duke University
Lauren Franz, MBChB principal investigator
Duke University
1 site in 1 country: see every site and its status.
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.