Autism Spectrum DisorderPhase 1TACTNCT04294290
Open Label Phase I hCT-MSC in Toddlers With Autism Spectrum Disorder
A Phase 1 study testing hCT-MSC infusion in people with Autism Spectrum Disorder, run by Duke University at 1 site in United States.
Trial summary
This is a single site, prospective study of one intravenous infusion of human umbilical cord tissue-derived mesenchymal stromal cells (hCT-MSC) in toddlers with autism spectrum disorder (ASD). Toddlers 18 to 48 months of age with a confirmed diagnosis of ASD will be eligible to participate. Read more about this study
- Who is it for?
- Ages 18 to 48 months
- Conditions the study is for
- Autism Spectrum Disorder
- What is being tested?
- hCT-MSC infusion
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 1 siteUnited States
- Study dates
- 24 Feb 2021to 17 Aug 2022
- Phase
- Phase 1
- 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 18 to 48 months
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Age ≥ 18 months to ≤ 48 months (48 months, 29 days) at the time of consent
- Confirmed clinical DSM-5 diagnosis of Autism Spectrum Disorder using the DSM-5 Checklist as informed by the Autism Diagnostic Observation Schedule - 2.
- Fragile X testing performed and negative; CMA and/or whole exome sequencing performed and results not linked to autism diagnosis
- Stable on current psychoactive medication regimen (dose and dosing schedule) for at least 2 months prior to infusion of study product
- Normal absolute lymphocyte count (≥1500/uL)
- Participant and parent/guardian are English speaking
- Able to travel to Duke University for two multi-day visits (baseline and six months) and parent/guardian is able to participate in interim surveys and interviews
- Parental consent
Who cannot join
- General:
- Review of medical records indicates ASD diagnosis not likely
- 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 hCT-MSC) study
- Genetic:
Show all 27Show fewer
- 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 mutation 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
- 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, WBC < 3,000 cells/mL, ALC <1000/uL, Platelets <150 x 10e9/uL
- Known clinically relevant physical dysmorphology associated with neurodevelopmental conditions.
- Current/Prior Therapy:
- History of prior cell therapy b. Current or prior use of IVIG or other anti-inflammatory medications with the exception of NSAIDs c. 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: AN OPEN LABEL PHASE I STUDY OF hCT-MSC, AN UMBILICAL CORD-DERIVED MESENCHYMAL STROMAL CELL PRODUCT IN YOUNG CHILDREN WITH AUTISM SPECTRUM DISORDER
This is a single site, prospective study of one intravenous infusion of human umbilical cord tissue-derived mesenchymal stromal cells (hCT-MSC) in toddlers with autism spectrum disorder (ASD). Toddlers 18 to 48 months of age with a confirmed diagnosis of ASD will be eligible to participate. Diagnosis will be confirmed at the time of the eligibility visit at the Duke Center for Autism and Brain Development. All participants will receive a single intravenous dose of 2x106/kg hCT-MSC per kilogram at baseline. Assessments will be conducted at baseline and 6 months, with remote follow-up assessments at 12 months.
Read the detailed description
The primary purpose of this study is to evaluate safety and feasibility. Safety assessments include monitoring of acute infusion reactions, adverse events, incidence of infections, and markers of alloimmunization. Clinical outcome measures will also be described. A key clinical outcome measeure is the change in social communication abilities from baseline to 6 months based on the Joint Engagement Rating Inventory (JERI), a commonly-used and well-validated coding system for rating the quality and quantity of social communication skills in toddlers with and without ASD.91 JERI coding rates social communication abilities on a 1 to 7 scale and factors in both the quantity and quality of skills. The total joint engagement score as well as ratings on all JERI subscales that comprise the total score will be described.
Other clinical endpoints will include the PDD Behavior Inventory (PDDBI) autism composite score, the mean of the Socialization Subscale Standard Score and Communication Subscale Standard Score on the Vineland Adaptive Behavior Scales (VABS-3), the Clinical Global Impression Scale (CGI) - Severity and Improvement Scales, the Communicative Development Inventories (CDI-2): Words \& Sentences subscales, attention abilities via eye-tracking, and brain activity.
Exploratory clinical endpoints will include autism symptoms measured by an app that elicits and records autism symptoms on an iPad (SenseToKnow), Autism Diagnostic Observation Scale (ADOS-2) Calibrated Severity Score (overall, social affect, and repetitive behavior), PDD Behavior Inventory (PDDBI) Subscales, and VABS-3 Standard Score and age equivalent for the following subscales: Socialization, Communication, and Daily Living and the Standard Score and age equivalent for the VABS-3 Adaptive Behavior Composite.
Safety and VABS-3 assessments will also be conducted remotely at three and 12 months. Duration of study participation will be 12 months from the time of the baseline infusion.
- Conditions
- Autism Spectrum Disorder
- Being tested
- hCT-MSC infusion
- Keywords
- Autism Spectrum Disorder, ASD, Autism, PDD
- Medical subject headings
- Autism Spectrum Disorder, Autistic Disorder
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
hCT-MSC infusionBiological
This is a single site, phase I, open-label, prospective study of one intravenous infusion of human umbilical cord tissue-derived mesenchymal stromal cells (hCT-MSC) in 12 toddlers 18 to 48 months of age with autism spectrum disorder (ASD).
| Study group | Role | What they receive |
|---|---|---|
| hCT-MSC infusion | Experimental | hCT-MSC infusion Receives: Biological: hCT-MSC infusion |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety of hCT-MSC Infusion as Measured by Total Number of Infusion Reactions
Measured: 1 year
Safety of hCT-MSC Infusion as Measured by Severity of Infusion Reactions
CTCAE is used to measure severity: 1= Mild, 2 = Moderate, 3 = Severe, 4 = Life-threatening, 5 = Death.
Measured: 1 year
Safety of hCT-MSC Infusion as Measured by Total Number of Product-related Infections
Measured: 1 year
Safety of hCT-MSC Infusion as Measured by Severity of Product-related Infections
CTCAE is used to measure severity: 1= Mild, 2 = Moderate, 3 = Severe, 4 = Life-threatening, 5 = Death.
Measured: 1 year
Safety of hCT-MSC Infusion as Measured by Number of Participants With Evidence of Alloimmunization Via Anti-HLA Antibodies
Measured: 1 year
Safety of hCT-MSC Infusion as Measured by Total Number of Graft vs. Host Disease (GVHD) Instances
Measured: 1 year
Safety of hCT-MSC Infusion as Measured by Severity of Graft vs. Host Disease (GVHD)
CTCAE is used to measure severity: 1= Mild, 2 = Moderate, 3 = Severe, 4 = Life-threatening, 5 = Death.
Measured: 1 year
Safety of hCT-MSC Infusion as Measured by Total Number of Unexpected Adverse Events
Measured: 1 year
Safety of hCT-MSC Infusion as Measured by Severity of Unexpected Adverse Events
CTCAE is used to measure severity: 1= Mild, 2 = Moderate, 3 = Severe, 4 = Life-threatening, 5 = Death.
Measured: 1 year
Change in Pervasive Developmental Disorder Behavior Inventory Autism Composite Score (PDDBI)
6-month T score minus Baseline T score. PDDBI is designed to assess problem behaviors and social, language, and learning or memory skills of children who have been diagnosed with autism spectrum disorder. Raw scores are converted to T scores with a mean of 50 and standard deviation of 10, with a possible range of 10-100. Higher scores represent more severe problem behaviors. Therefore, a negative change in score indicates improvement.
Measured: Baseline, 6 months
Change in VABS-3 (Vineland Adaptive Behavior Scales) Socialization and Communication Composite Score
The change in the average of the Communication and Socialization Standard Subscale Scores on the Vineland Adaptive Behavior Scales (VABS-3) from the Comprehensive Interview form from baseline to six months. Higher scores indicate greater communication and/or socialization, with a range of 20 to 160 (mean = 100). A positive change in the scores indicates an improvement in communication and/or socialization.
Measured: Baseline, 6 months
Number of Participants Who Improved (Much Improved or Minimally Improved) on the Clinical Global Impression Scale (CGI)
The number of participants who scored a 1 (very much improved), 2 (much improved), or 3 (minimally improved) on the Clinical Global Impression Scale (CGI-I). The CGI-I score ranges from 1 to 7, where a lower score indicates greater improvement.
Measured: Baseline, 6 months
Change in Communicative Development Inventories (CDI-2)
6-month number of words produced minus baseline number of words produced. A positive change indicates improvement.
Measured: Baseline, 6 months
Change in Attention Abilities as Assessed Via Eye-tracking
6-month percentage of gaze directed towards social stimulus minus baseline percentage of gaze directed towards social stimulus. A negative change indicates improvement.
Measured: Baseline, 6 months
Change in Brain Activity as Measured by EEG
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 posted4 Mar 2020
- Study start24 Feb 2021
- Primary completion17 Aug 2022
- Study completion17 Aug 2022
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.
- Other trials for Autism Spectrum DisorderStudies of the same condition, open and under way.
- More trials run by Duke UniversityThe sponsor's other registered studies.
- 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 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- Duke University
- Sponsor type
- University, hospital or other
- Collaborators
- The Marcus Foundation
- Responsible party
- Principal investigator, Joanne Kurtzberg, MD, Duke University
- Investigators
- Joanne Kurtzberg, MD, Principal investigator, Duke HealthGeraldine Dawson, PhD, Principal investigator, Duke HealthJessica Sun, MD, Principal investigator, Duke Health
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04294290
- Sponsor's protocol ID
- Pro00104460
- Acronym
- TACT
- Status last verified
- Feb 2025
- First submitted
- 2 Mar 2020
- Last updated
- 24 Feb 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 Feb 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- AN OPEN LABEL PHASE I STUDY OF hCT-MSC, AN UMBILICAL CORD-DERIVED MESENCHYMAL STROMAL CELL PRODUCT IN YOUNG CHILDREN WITH AUTISM SPECTRUM DISORDER
- ClinicalTrials.gov ID
- NCT04294290
- Acronym / sponsor ID
- TACT / Pro00104460
- Conditions
- Autism Spectrum Disorder
- Keywords
- Autism Spectrum Disorder, ASD, Autism, PDD
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 12 participants (actual)
- Lead sponsor
- Duke UniversityUniversity, hospital or other
- Collaborators
- The Marcus Foundation
- Study start
- 24 Feb 2021 (actual)
- Primary completion
- 17 Aug 2022 (actual)
- Study completion
- 17 Aug 2022 (actual)
- First posted
- 4 Mar 2020 (actual)
- Results
- Posted 24 Feb 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a single site, prospective study of one intravenous infusion of human umbilical cord tissue-derived mesenchymal stromal cells (hCT-MSC) in toddlers with autism spectrum disorder (ASD). Toddlers 18 to 48 months of age with a confirmed diagnosis of ASD will be eligible to participate. Diagnosis will be confirmed at the time of the eligibility visit at the Duke Center for Autism and Brain Development. All participants will receive a single intravenous dose of 2x106/kg hCT-MSC per kilogram at baseline. Assessments will be conducted at baseline and 6 months, with remote follow-up assessments at 12 months.
Detailed description
The primary purpose of this study is to evaluate safety and feasibility. Safety assessments include monitoring of acute infusion reactions, adverse events, incidence of infections, and markers of alloimmunization. Clinical outcome measures will also be described. A key clinical outcome measeure is the change in social communication abilities from baseline to 6 months based on the Joint Engagement Rating Inventory (JERI), a commonly-used and well-validated coding system for rating the quality and quantity of social communication skills in toddlers with and without ASD.91 JERI coding rates social communication abilities on a 1 to 7 scale and factors in both the quantity and quality of skills. The total joint engagement score as well as ratings on all JERI subscales that comprise the total score will be described.
Other clinical endpoints will include the PDD Behavior Inventory (PDDBI) autism composite score, the mean of the Socialization Subscale Standard Score and Communication Subscale Standard Score on the Vineland Adaptive Behavior Scales (VABS-3), the Clinical Global Impression Scale (CGI) - Severity and Improvement Scales, the Communicative Development Inventories (CDI-2): Words \& Sentences subscales, attention abilities via eye-tracking, and brain activity.
Exploratory clinical endpoints will include autism symptoms measured by an app that elicits and records autism symptoms on an iPad (SenseToKnow), Autism Diagnostic Observation Scale (ADOS-2) Calibrated Severity Score (overall, social affect, and repetitive behavior), PDD Behavior Inventory (PDDBI) Subscales, and VABS-3 Standard Score and age equivalent for the following subscales: Socialization, Communication, and Daily Living and the Standard Score and age equivalent for the VABS-3 Adaptive Behavior Composite.
Safety and VABS-3 assessments will also be conducted remotely at three and 12 months. Duration of study participation will be 12 months from the time of the baseline infusion.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| hCT-MSC infusion | Experimental | Biological: hCT-MSC infusionhCT-MSC infusion |
Who may qualify
- Ages
- 18 to 48 months
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (8 listed)
- Age ≥ 18 months to ≤ 48 months (48 months, 29 days) at the time of consent
- Confirmed clinical DSM-5 diagnosis of Autism Spectrum Disorder using the DSM-5 Checklist as informed by the Autism Diagnostic Observation Schedule - 2.
- Fragile X testing performed and negative; CMA and/or whole exome sequencing performed and results not linked to autism diagnosis
- Stable on current psychoactive medication regimen (dose and dosing schedule) for at least 2 months prior to infusion of study product
- Normal absolute lymphocyte count (≥1500/uL)
Key exclusion criteria (27 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 |
|---|---|---|
| hCT-MSC infusionThis is a single site, phase I, open-label, prospective study of one intravenous infusion of human umbilical cord tissue-derived mesenchymal stromal cells (hCT-MSC) in 12 toddlers 18 to 48 months of age with autism spectrum disorder (ASD). | Biological | hCT-MSC infusion |
Primary outcome
Safety of hCT-MSC Infusion as Measured by Total Number of Infusion Reactions
Time frame: 1 year
Safety of hCT-MSC Infusion as Measured by Severity of Infusion Reactions
Time frame: 1 year
CTCAE is used to measure severity: 1= Mild, 2 = Moderate, 3 = Severe, 4 = Life-threatening, 5 = Death.
Safety of hCT-MSC Infusion as Measured by Total Number of Product-related Infections
Time frame: 1 year
Safety of hCT-MSC Infusion as Measured by Severity of Product-related Infections
Time frame: 1 year
CTCAE is used to measure severity: 1= Mild, 2 = Moderate, 3 = Severe, 4 = Life-threatening, 5 = Death.
Safety of hCT-MSC Infusion as Measured by Number of Participants With Evidence of Alloimmunization Via Anti-HLA Antibodies
Time frame: 1 year
Safety of hCT-MSC Infusion as Measured by Total Number of Graft vs. Host Disease (GVHD) Instances
Time frame: 1 year
Safety of hCT-MSC Infusion as Measured by Severity of Graft vs. Host Disease (GVHD)
Time frame: 1 year
CTCAE is used to measure severity: 1= Mild, 2 = Moderate, 3 = Severe, 4 = Life-threatening, 5 = Death.
Safety of hCT-MSC Infusion as Measured by Total Number of Unexpected Adverse Events
Time frame: 1 year
Safety of hCT-MSC Infusion as Measured by Severity of Unexpected Adverse Events
Time frame: 1 year
CTCAE is used to measure severity: 1= Mild, 2 = Moderate, 3 = Severe, 4 = Life-threatening, 5 = Death.
Change in Pervasive Developmental Disorder Behavior Inventory Autism Composite Score (PDDBI)
Time frame: Baseline, 6 months
6-month T score minus Baseline T score. PDDBI is designed to assess problem behaviors and social, language, and learning or memory skills of children who have been diagnosed with autism spectrum disorder. Raw scores are converted to T scores with a mean of 50 and standard deviation of 10, with a possible range of 10-100. Higher scores represent more severe problem behaviors. Therefore, a negative change in score indicates improvement.
Change in VABS-3 (Vineland Adaptive Behavior Scales) Socialization and Communication Composite Score
Time frame: Baseline, 6 months
The change in the average of the Communication and Socialization Standard Subscale Scores on the Vineland Adaptive Behavior Scales (VABS-3) from the Comprehensive Interview form from baseline to six months. Higher scores indicate greater communication and/or socialization, with a range of 20 to 160 (mean = 100). A positive change in the scores indicates an improvement in communication and/or socialization.
Number of Participants Who Improved (Much Improved or Minimally Improved) on the Clinical Global Impression Scale (CGI)
Time frame: Baseline, 6 months
The number of participants who scored a 1 (very much improved), 2 (much improved), or 3 (minimally improved) on the Clinical Global Impression Scale (CGI-I). The CGI-I score ranges from 1 to 7, where a lower score indicates greater improvement.
Change in Communicative Development Inventories (CDI-2)
Time frame: Baseline, 6 months
6-month number of words produced minus baseline number of words produced. A positive change indicates improvement.
Change in Attention Abilities as Assessed Via Eye-tracking
Time frame: Baseline, 6 months
6-month percentage of gaze directed towards social stimulus minus baseline percentage of gaze directed towards social stimulus. A negative change indicates improvement.
Change in Brain Activity as Measured by EEG
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 Feb 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- 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.
Joanne Kurtzberg, MD principal investigator
Duke Health
Geraldine Dawson, PhD principal investigator
Duke Health
Jessica Sun, MD principal investigator
Duke Health
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing hCT-MSC infusion in people with Autism Spectrum Disorder, run by Duke University 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 8 inclusion and 27 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 Feb 2025. 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. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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 Feb 2025). 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?
Safety of hCT-MSC Infusion as Measured by Total Number of Infusion Reactions (time frame: 1 year). The study lists 15 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 Feb 2025.