Autism Spectrum DisorderPhase 1AIMsNCT04484077
hCT-MSC Infusion in Adults With Autism Spectrum Disorder
A Phase 1 study testing hCT-MSC in people with Autism Spectrum Disorder and Autism, run by Joanne Kurtzberg, MD at 1 site in United States.
Trial summary
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 Cells (hCT-MSC) in adults with autism spectrum disorder (ASD). hCT-MSC is a cell product isolated from umbilical cord tissue. Read more about this study
- Who is it for?
- Ages 18 to 34 years
- Conditions the study is for
- Autism Spectrum Disorder, Autism
- What is being tested?
- hCT-MSC
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 8 participants
- Where
- 1 siteUnited States
- Study dates
- 24 Jan 2022to 29 Apr 2024
- Phase
- Phase 1
- Are there results?
- None posted in the registry
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 34 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Age ≥18 to < 35 years (34 years, 364 days) at the time of consent
- Confirmed pre-existing diagnosis of ASD in the individual's educational and/or medical record
- 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, with no intention of changing or beginning new psychiatric medications or behavioral treatments during the duration of the study.
- Normal absolute lymphocyte count (≥1200/uL for African American participants and ≥1500/uL for all other participants)
- General Ability Index of ≥70, confirmed through cognitive testing completed by study personnel to establish eligibility
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- Participant and Parent are English speaking
- Able to travel to Duke University for baseline visit.
- Participant has a parent who spends four or more hours a week with the participant, who is able and willing to participate in study visits and interim surveys and interviews
- Informed consent of the participant and parent (participants must have legal authority regarding their own medical care and a parent or legally authorized representative may not consent on their behalf)
Who cannot join
- General:
- Review of medical records indicates ASD diagnosis and IQ > 70 unlikely.
- Known or suspected diagnosis of any of the following coexisting psychiatric conditions: bipolar disorder, schizophrenia, Tourette syndrome, and/or any co-occurring psychiatric disorder that the investigator believes would interfere with accurate completion of study instruments and/or current (within the past year) evidence of suicidality as assessed by an interview with participant and/or parent which includes the Columbia Suicide Severity Rating Scale, and a review of responses on the Behavioral and Symptom Identification Scale (BASIS-24).
- Screening data or in-person evaluations suggest that participant would not be able to comply with the requirements of the study procedures as assessed by the study team
- Parent and/or participant is unwilling or unable to commit to participation in all study-related assessments, including protocol follow up
- Sibling is enrolled in this (Duke AIMs) study
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- Genetic:
- Records indicate that the participant 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 participant 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 participants 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 pregnancy and unwillingness to use adequate birth control for 3 months after the final study product infusion.
- Current/Prior Therapy:
- Availability of a banked, qualified autologous cord blood unit or parent 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 >5 days within 4 weeks, 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, 27710
About this study
Official title: A PHASE I STUDY OF hCT-MSC, AN UMBILICAL CORD-DERIVED MESENCHYMAL STROMAL CELL PRODUCT, 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 Cells (hCT-MSC) in adults with autism spectrum disorder (ASD). hCT-MSC is a cell product isolated from umbilical cord tissue. The cells from the cord tissue are processed and expanded in the laboratory and then infused intravenously in a single dose per participant. Participants will be ages 18-35 years, with ASD and a full-scale IQ >70 without an identified genetic cause of autism. Participants will have an in-person baseline visit and remote follow up visits at 6 and 12 months. In addition to the primary endpoints evaluating safety, the study will evaluate changes in social communications skills after hCT-MSC administration.
Read the detailed description
This is a prospective, open label, phase one study to determine the safety and tolerability of a single intravenous dose of Human Umbilical Cord Tissue Derived Mesenchymal Stromal Cells (hCT-MSC) in adults with autism spectrum disorder (ASD). hCT-MSCs are manufactured from umbilical cord tissue donated by healthy mothers delivering full term babies via Cesarean Section. The cells are extracted from the cord tissue, expanded and cryopreserved (frozen). One dose of 2x10^6 cells/kg (maximum of 10 x 10^7) will be administered intravenously to each participant in this study.
It is hypothesized that immune dysregulation and/or abnormal neuronal connectivity that adversely affects normal brain development may cause core symptomatology observed in individuals with ASD. Mesenchymal stromal cells (MSC) have demonstrated a multitude of immunomodulatory effects which are thought to be carried out via paracrine and trophic signaling. While MSCs modulate the immune response, MSCs themselves have low immunogenicity (the body does not have a strong immune reaction against them) and they do not permanently engraft in the recipient.
Adults ages 18 to less than 35 years with ASD will be eligible to participate. All participants will have a screening visit that includes clinical evaluations to verify cognitive abilities and confirmation of eligibility.
One dose of 2x10^6 cells/kg (maximum of 10 x 10^7) will be administered intravenously to each participant at a baseline visit. Participants will be admitted to the infusion center on the day of their baseline visit and vital signs (heart rate, blood pressure, temperature, respiratory rate) will be measured. Participants may request anti-anxiety medication prior to IV placement if desired. A peripheral IV will be placed and prior to the infusion, premedications (Benadryl, Solumedrol, 0.5mg/kg each) will be administered. The hCT-MSCs product will be administered intravenously over 30-60 minutes. Pulse oximetry will be monitored continuously throughout the infusion and IV fluid maintenance will be given. Participants will be discharged after 1 hour, providing all vital signs are at their baseline and they are asymptomatic with no evidence of toxicity. Participants will be evaluated the day after the infusion to assess for any infusion-related adverse reactions or complications. A phone call or email will be done to assess safety of the infusion 7-10 days after the infusion. Remote follow up will be conducted 6 months and one year after infusion. The Medical and Behavioral History Questionnaire assessing adverse events will be obtained at baseline, 6 and 12-months.
The main endpoint is safety, and acute infusion reactions, incidence of infections, and markers of alloimmunization will be assessed. Social communication will be assessed at baseline and 6 months post product administration.
- Conditions
- Autism Spectrum Disorder, Autism
- Being tested
- hCT-MSC
- Keywords
- autism, MSC, hCT-MSC, mesenchymal stromal cells
- 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-MSCBiological
2x10^6 hCT-MSC/kg suspended in plasmalyte-A, 5% HSA, and residual DMSO/dextran administered intravenously over 30-60 minutes via syringe pump
| Study group | Role | What they receive |
|---|---|---|
| hCT-MSC infusion | Experimental | A single, intravenous infusion of hCT-MSCs. Targeted dose is 2x10^6 cells/kg with a maximum dose of 10 x 10^7 cells/kg. Receives: Biological: hCT-MSC |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of infusion reactions
cumulative incidence as measured by clinical examination and patient interview
Measured: Baseline through 10 days post infusion
Incidence of product-related infections
cumulative incidence as measured by patient interview and questionnaire
Measured: Baseline through 12 months
Evidence of formation of anti-HLA antibodies
change from baseline to 6 and 12 months post infusion as measured by PRA testing
Measured: Baseline, 6 months, 12 months
Incidence of graft vs. host disease
cumulative incidence as measured by patient interview and questionnaire
Measured: Baseline through 12 months
Incidence of unexpected adverse events, by severity and relation to study
cumulative incidence as measured by patient questionnaire and clinical labs
Measured: Baseline through 12 months
Secondary outcomes (1)
The Vineland Adaptive Behavior Scale Interview, 3rd Edition, Comprehensive interview form
Change from baseline to six months in the Combined Socialization Standard Score based on parent report. Scores range from 20 to 140. Higher scores indicate a higher functioning level
Measured: Baseline and 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Jul 2020
- Study start24 Jan 2022
- Primary completion29 Apr 2024
- Study completion29 Apr 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Autism Spectrum DisorderStudies 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 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 8 participants (actual)
Sponsor and investigators
- Lead sponsor
- Joanne Kurtzberg, MD
- Sponsor type
- University, hospital or other
- Collaborators
- The Marcus Foundation
- Responsible party
- Sponsor-investigator, Joanne Kurtzberg, MD, Duke University
- Investigators
- Jessica Sun, MD, Principal investigator, Duke UniversityJoanne Kurtzberg, MD, Principal investigator, Duke University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04484077
- Sponsor's protocol ID
- PRO00104691
- Acronym
- AIMs
- Status last verified
- Jul 2024
- First submitted
- 16 Jul 2020
- Last updated
- 23 Jul 2024
- Results posted
- No
- 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 23 Jul 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A PHASE I STUDY OF hCT-MSC, AN UMBILICAL CORD-DERIVED MESENCHYMAL STROMAL CELL PRODUCT, IN ADULTS WITH AUTISM SPECTRUM DISORDER
- ClinicalTrials.gov ID
- NCT04484077
- Acronym / sponsor ID
- AIMs / PRO00104691
- Conditions
- Autism Spectrum Disorder, Autism
- Keywords
- autism, MSC, hCT-MSC, mesenchymal stromal cells
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 8 participants (actual)
- Lead sponsor
- Joanne Kurtzberg, MDUniversity, hospital or other
- Collaborators
- The Marcus Foundation
- Study start
- 24 Jan 2022 (actual)
- Primary completion
- 29 Apr 2024 (actual)
- Study completion
- 29 Apr 2024 (actual)
- First posted
- 23 Jul 2020 (actual)
- Results
- Not posted in the source record
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 the study is to determine the safety and tolerability of a single intravenous dose of Human Umbilical Cord Tissue Derived Mesenchymal Stromal Cells (hCT-MSC) in adults with autism spectrum disorder (ASD). hCT-MSC is a cell product isolated from umbilical cord tissue. The cells from the cord tissue are processed and expanded in the laboratory and then infused intravenously in a single dose per participant. Participants will be ages 18-35 years, with ASD and a full-scale IQ >70 without an identified genetic cause of autism. Participants will have an in-person baseline visit and remote follow up visits at 6 and 12 months. In addition to the primary endpoints evaluating safety, the study will evaluate changes in social communications skills after hCT-MSC administration.
Detailed description
This is a prospective, open label, phase one study to determine the safety and tolerability of a single intravenous dose of Human Umbilical Cord Tissue Derived Mesenchymal Stromal Cells (hCT-MSC) in adults with autism spectrum disorder (ASD). hCT-MSCs are manufactured from umbilical cord tissue donated by healthy mothers delivering full term babies via Cesarean Section. The cells are extracted from the cord tissue, expanded and cryopreserved (frozen). One dose of 2x10^6 cells/kg (maximum of 10 x 10^7) will be administered intravenously to each participant in this study.
It is hypothesized that immune dysregulation and/or abnormal neuronal connectivity that adversely affects normal brain development may cause core symptomatology observed in individuals with ASD. Mesenchymal stromal cells (MSC) have demonstrated a multitude of immunomodulatory effects which are thought to be carried out via paracrine and trophic signaling. While MSCs modulate the immune response, MSCs themselves have low immunogenicity (the body does not have a strong immune reaction against them) and they do not permanently engraft in the recipient.
Adults ages 18 to less than 35 years with ASD will be eligible to participate. All participants will have a screening visit that includes clinical evaluations to verify cognitive abilities and confirmation of eligibility.
One dose of 2x10^6 cells/kg (maximum of 10 x 10^7) will be administered intravenously to each participant at a baseline visit. Participants will be admitted to the infusion center on the day of their baseline visit and vital signs (heart rate, blood pressure, temperature, respiratory rate) will be measured. Participants may request anti-anxiety medication prior to IV placement if desired. A peripheral IV will be placed and prior to the infusion, premedications (Benadryl, Solumedrol, 0.5mg/kg each) will be administered. The hCT-MSCs product will be administered intravenously over 30-60 minutes. Pulse oximetry will be monitored continuously throughout the infusion and IV fluid maintenance will be given. Participants will be discharged after 1 hour, providing all vital signs are at their baseline and they are asymptomatic with no evidence of toxicity. Participants will be evaluated the day after the infusion to assess for any infusion-related adverse reactions or complications. A phone call or email will be done to assess safety of the infusion 7-10 days after the infusion. Remote follow up will be conducted 6 months and one year after infusion. The Medical and Behavioral History Questionnaire assessing adverse events will be obtained at baseline, 6 and 12-months.
The main endpoint is safety, and acute infusion reactions, incidence of infections, and markers of alloimmunization will be assessed. Social communication will be assessed at baseline and 6 months post product administration.
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-MSCA single, intravenous infusion of hCT-MSCs. Targeted dose is 2x10^6 cells/kg with a maximum dose of 10 x 10^7 cells/kg. |
Who may qualify
- Ages
- 18 to 34 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (10 listed)
- Age ≥18 to < 35 years (34 years, 364 days) at the time of consent
- Confirmed pre-existing diagnosis of ASD in the individual's educational and/or medical record
- 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, with no intention of changing or beginning new psychiatric medications or behavioral treatments during the duration of the study.
- Normal absolute lymphocyte count (≥1200/uL for African American participants and ≥1500/uL for all other participants)
Key exclusion criteria (30 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-MSC2x10^6 hCT-MSC/kg suspended in plasmalyte-A, 5% HSA, and residual DMSO/dextran administered intravenously over 30-60 minutes via syringe pump | Biological | hCT-MSC infusion |
Primary outcome
Incidence of infusion reactions
Time frame: Baseline through 10 days post infusion
cumulative incidence as measured by clinical examination and patient interview
Incidence of product-related infections
Time frame: Baseline through 12 months
cumulative incidence as measured by patient interview and questionnaire
Evidence of formation of anti-HLA antibodies
Time frame: Baseline, 6 months, 12 months
change from baseline to 6 and 12 months post infusion as measured by PRA testing
Incidence of graft vs. host disease
Time frame: Baseline through 12 months
cumulative incidence as measured by patient interview and questionnaire
Incidence of unexpected adverse events, by severity and relation to study
Time frame: Baseline through 12 months
cumulative incidence as measured by patient questionnaire and clinical labs
1 secondary outcome
The Vineland Adaptive Behavior Scale Interview, 3rd Edition, Comprehensive interview form
Time frame: Baseline and 6 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Jul 2024.
- 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.
Jessica Sun, MD principal investigator
Duke University
Joanne Kurtzberg, MD 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 1 study testing hCT-MSC in people with Autism Spectrum Disorder and Autism, 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 10 inclusion and 30 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 23 Jul 2024. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Incidence of infusion reactions (time frame: Baseline through 10 days post infusion). The study lists 5 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 23 Jul 2024; AzTrials last refreshed its copy on 7 Nov 2026.