AutismPhase 1hCT-MSCsNCT03099239
hCT-MSCs for Children With Autism Spectrum Disorder (ASD)
A Phase 1 study testing hCT-MSC infusion 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 1 study is to determine the safety of one, two, and three intravenous infusions of human umbilical cord tissue-derived mesenchymal stromal cells (hCT-MSC), administered every two months, in children with autism spectrum disorder (ASD). Read more about this study
- Who is it for?
- Ages 2 to 11 years
- Conditions the study is for
- Autism, Autism Spectrum Disorder, ASD
- 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
- 6 Jun 2017to 10 Jun 2019
- 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 2 to 11 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Age ≥ 2 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 with a moderate severity level of ASD as reflected by SRS score ≥ 66 and CGI-S severity score of ≥ 4.
- 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 (≥1500/uL)
- Participant and parent/guardian are English speaking
- Able to travel to Duke University up to four times (baseline, every two months for subsequent infusions, and 6 months after initial infusion), 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
- 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 hCT-MSC) study
Show all 29Show fewer
- 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
- Evaluation by geneticist (performed locally as standard of care or remotely by the study geneticist via review of available data - minimally medical records, photos, Fragile X and CMA testing) indicates a genetic cause for ASD.
- Infectious:
- Known active CNS infection
- Evidence of uncontrolled infection based on records or clinical assessment
- Known HIV positivity
- Medical:
- Known metabolic disorder
- Known abnormal thyroid function (patients with treated hypothyroidism with a normal TSH may be included)
- 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
- 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:
- 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: A Phase I Study of hCT-MSC, An Umbilical Cord-Derived Mesenchymal Stromal Cell Product, in Children With Autism Spectrum Disorder
The purpose of this Phase 1 study is to determine the safety of one, two, and three intravenous infusions of human umbilical cord tissue-derived mesenchymal stromal cells (hCT-MSC), administered every two months, in children with autism spectrum disorder (ASD).
Read the detailed description
This study is a phase I, prospective, open-label trial designed to assess the safety of one, two, and three intravenous doses of hCT-MSC in young children with ASD. Children ages two to 11 years with ASD will be eligible to participate. All participants will receive intravenous infusion(s) of CTCs. The first cohort of three patients will receive a single dose. If there are no safety concerns, the second cohort of three patients will receive two doses, given two months apart. The third cohort will consist of six patients, each of whom will receive three hCT-MSC infusions with a two-month interval between doses. All participants will have an initial clinical evaluation to verify the diagnosis of ASD and confirm protocol eligibility. The main endpoint is safety, for which acute infusion reactions and incidence of infections will be assessed. ASD-specific outcome measures, described below, will be assessed at baseline and six months from baseline and results will be described.
- Conditions
- Autism, Autism Spectrum Disorder, ASD
- Being tested
- hCT-MSC infusion
- 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.
hCT-MSC infusionBiological
hCT-MSCs are a product of allogeneic cells manufactured from digested umbilical cord tissue that is expanded in culture, cryopreserved and banked.
| Study group | Role | What they receive |
|---|---|---|
| Single hCT-MSC infusion | Experimental | Subjects 1-3 will receive a single infusion of hCT-MSCs. Receives: Biological: hCT-MSC infusion |
| Two hCT-MSC infusions | Experimental | Subjects 4-6 will receive two infusions of hCT-MSCs. Receives: Biological: hCT-MSC infusion |
| Three hCT-MSC infusions | Experimental | Subjects 6-12 will receive three infusions of hCT-MSCs. Receives: Biological: hCT-MSC infusion |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of Infusion reactions
Patients will be assessed for infusion reactions.
Measured: Assessed for a significant change at the time of each infusion, 24 hours after each infusion, 7-10 days after each infusion, 6 and 12 months after the final infusion.
Incidence of Infections
Patients will be assessed for infections.
Measured: Assessed for a significant change at the time of each infusion, 24 hours after each infusion, 7-10 days after each infusion, 6 and 12 months after the final infusion.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Apr 2017
- Study start6 Jun 2017
- Primary completion10 Jun 2019
- Study completion10 Jun 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 12 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
- Joanne Kurtzberg, MD, Principal investigator, Duke UniversityGeraldine Dawson, PhD, Principal investigator, Duke UniversityJessica Sun, MD, Principal investigator, Duke University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03099239
- Sponsor's protocol ID
- Pro00079421
- Acronym
- hCT-MSCs
- Status last verified
- Dec 2019
- First submitted
- 24 Mar 2017
- Last updated
- 3 Dec 2019
- 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 3 Dec 2019. 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 Children With Autism Spectrum Disorder
- ClinicalTrials.gov ID
- NCT03099239
- Acronym / sponsor ID
- hCT-MSCs / Pro00079421
- Conditions
- Autism, Autism Spectrum Disorder, ASD
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 12 participants (actual)
- Lead sponsor
- Joanne Kurtzberg, MDUniversity, hospital or other
- Collaborators
- The Marcus Foundation
- Study start
- 6 Jun 2017 (actual)
- Primary completion
- 10 Jun 2019 (actual)
- Study completion
- 10 Jun 2019 (actual)
- First posted
- 4 Apr 2017 (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 this Phase 1 study is to determine the safety of one, two, and three intravenous infusions of human umbilical cord tissue-derived mesenchymal stromal cells (hCT-MSC), administered every two months, in children with autism spectrum disorder (ASD).
Detailed description
This study is a phase I, prospective, open-label trial designed to assess the safety of one, two, and three intravenous doses of hCT-MSC in young children with ASD. Children ages two to 11 years with ASD will be eligible to participate. All participants will receive intravenous infusion(s) of CTCs. The first cohort of three patients will receive a single dose. If there are no safety concerns, the second cohort of three patients will receive two doses, given two months apart. The third cohort will consist of six patients, each of whom will receive three hCT-MSC infusions with a two-month interval between doses. All participants will have an initial clinical evaluation to verify the diagnosis of ASD and confirm protocol eligibility. The main endpoint is safety, for which acute infusion reactions and incidence of infections will be assessed. ASD-specific outcome measures, described below, will be assessed at baseline and six months from baseline and results will be described.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Single hCT-MSC infusion | Experimental | Biological: hCT-MSC infusionSubjects 1-3 will receive a single infusion of hCT-MSCs. |
| Two hCT-MSC infusions | Experimental | Biological: hCT-MSC infusionSubjects 4-6 will receive two infusions of hCT-MSCs. |
| Three hCT-MSC infusions | Experimental | Biological: hCT-MSC infusionSubjects 6-12 will receive three infusions of hCT-MSCs. |
Who may qualify
- Ages
- 2 to 11 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (8 listed)
- Age ≥ 2 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 with a moderate severity level of ASD as reflected by SRS score ≥ 66 and CGI-S severity score of ≥ 4.
- 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 (≥1500/uL)
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 |
|---|---|---|
| hCT-MSC infusionhCT-MSCs are a product of allogeneic cells manufactured from digested umbilical cord tissue that is expanded in culture, cryopreserved and banked. | Biological | Single hCT-MSC infusion, Three hCT-MSC infusions, Two hCT-MSC infusions |
Primary outcome
Incidence of Infusion reactions
Time frame: Assessed for a significant change at the time of each infusion, 24 hours after each infusion, 7-10 days after each infusion, 6 and 12 months after the final infusion.
Patients will be assessed for infusion reactions.
Incidence of Infections
Time frame: Assessed for a significant change at the time of each infusion, 24 hours after each infusion, 7-10 days after each infusion, 6 and 12 months after the final infusion.
Patients will be assessed for infections.
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 3 Dec 2019.
- 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 University
Geraldine Dawson, PhD principal investigator
Duke University
Jessica Sun, 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 infusion 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 8 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 3 Dec 2019. 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: Assessed for a significant change at the time of each infusion, 24 hours after each infusion, 7-10 days after each infusion, 6 and 12 months after the final infusion.). The study lists 2 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 3 Dec 2019.