AutismPhase 1/2NCT02192749
Allogeneic Umbilical Cord Mesenchymal Stem Cell Therapy for Autism
A Phase 1/2 study testing Umbilical cord mesenchymal stem cells in people with Autism, run by Translational Biosciences at 1 site in Panama.
Trial summary
Allogeneic (not from the subject) human umbilical cord tissue-derived stem cells administered intravenously (IV) in a series of 4 infusions every 3 months over the course of one year is safe and will induce a therapeutic effect in autism patients. Read more about this study
- Who is it for?
- Ages 6 to 16 years
- Conditions the study is for
- Autism
- What is being tested?
- Umbilical cord mesenchymal stem cells
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 sitePanama
- Study dates
- Jul 2014to Aug 2017
- Phase
- Phase 1/2
- 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 6 to 16 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Male or Female
- Ages 6 to 16
- Diagnostic and Statistical Manual of Mental Disorders (DSM IV) diagnosis of autism confirmed by Autism Diagnostic Observation Schedule (ADOS) and/or Autism Diagnostic Interview-Revised (ADI-R)
- No anticipated changes in treatment for the study duration (e.g., diet, nutrients)
- No additional biomedical treatments started 6 weeks prior to enrollment
- No changes in dietary management for 3 months prior to enrollment
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- Ambulatory or require minimum support walking, per parent
- Able to sit still for 5 minutes or longer with a preferred toy item, per parent
- Adequate vision and hearing for the purposes of test administration, per parent
- Adequate arm-hand-finger coordination (i.e., able to point) for learning and cognitive tasks used in outcome measurement, per parent
- Stable and controlled mental disorder
- Under the care of a caregiver willing to participate by attending regularly scheduled appointments and completing the necessary measures
- Normal heavy metals test for lead and mercury levels performed within 30 days of first stem cell infusion
- Must provide name and specialty of specialist who has made Autism Spectrum Disorder (ASD) diagnosis
- Adequate financial means to cover $7,200 (US Dollars) plus travel expenses
Who cannot join
- Significant prematurity at birth (less than 32 weeks gestation); or birth weight significantly below normal for gestational age (SGA - small for gestational age)
- mental retardation
- seizure disorder
- auto-immune conditions
- history of head trauma and other neurological or medical conditions
- Abnormal heavy metals test for lead and mercury performed within 30 days of first stem cell infusion
- Prior stem cell therapy of any kind
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Open, Prospective Trial of Treatment of Autism Spectrum Disorders (ASD) Using Intravenous Infusion of Umbilical Cord Tissue Mesenchymal Stem Cells (UC-MSC)
Allogeneic (not from the subject) human umbilical cord tissue-derived stem cells administered intravenously (IV) in a series of 4 infusions every 3 months over the course of one year is safe and will induce a therapeutic effect in autism patients.
- Conditions
- Autism
- Being tested
- Umbilical cord mesenchymal stem cells
- Keywords
- autism, umbilical cord, mesenchymal, stem cells
- Medical subject headings
- Autistic Disorder
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Umbilical cord mesenchymal stem cellsBiological
| Study group | Role | What they receive |
|---|---|---|
| Umbilical cord mesenchymal stem cells | Experimental | Not described Receives: Biological: Umbilical cord mesenchymal stem cells |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of participants with adverse events
Measured: 89 weeks
Secondary outcomes (4)
Number of participants with a change in disability as measured by the Autism Treatment Evaluation Checklist (ATEC)
Measured: 13 weeks, 25 weeks, 37 weeks, 49 weeks, 89 weeks
Number of participants with a change in disability as measured by the Childhood Autism Rating Scale (CARS)
Measured: 13 weeks, 25 weeks, 37 weeks, 49 weeks, 89 weeks
Change from baseline macrophage-derived chemokine (MDC)
Measured: 13 weeks, 25 weeks, 37 weeks, 49 weeks, 89 weeks
Change from baseline thymus and activation-regulated chemokine (TARC)
Measured: 13 weeks, 25 weeks, 37 weeks, 49 weeks, 89 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Jul 2014 (estimated)
- Study startJul 2014
- Primary completionAug 2017
- Study completionAug 2017
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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (actual)
Sponsor and investigators
- Lead sponsor
- Translational Biosciences
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Nelson Novarro, MD, Principal investigatorJorge Paz-Rodriguez, MD, Principal investigator, Translational Biosciences / Stem Cell Institute Panama
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02192749
- Sponsor's protocol ID
- TBS-UCMSC-ASD001
- Status last verified
- Oct 2017
- First submitted
- 12 Jul 2014
- Last updated
- 13 Oct 2017
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 13 Oct 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Open, Prospective Trial of Treatment of Autism Spectrum Disorders (ASD) Using Intravenous Infusion of Umbilical Cord Tissue Mesenchymal Stem Cells (UC-MSC)
- ClinicalTrials.gov ID
- NCT02192749
- Acronym / sponsor ID
- TBS-UCMSC-ASD001
- Conditions
- Autism
- Keywords
- autism, umbilical cord, mesenchymal, stem cells
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 20 participants (actual)
- Lead sponsor
- Translational BiosciencesIndustry (pharma, biotech, devices)
- Study start
- Jul 2014
- Primary completion
- Aug 2017 (actual)
- Study completion
- Aug 2017 (actual)
- First posted
- 17 Jul 2014 (estimated)
- 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.
Allogeneic (not from the subject) human umbilical cord tissue-derived stem cells administered intravenously (IV) in a series of 4 infusions every 3 months over the course of one year is safe and will induce a therapeutic effect in autism patients.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Umbilical cord mesenchymal stem cells | Experimental | Biological: Umbilical cord mesenchymal stem cells |
Who may qualify
- Ages
- 6 to 16 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (15 listed)
- Male or Female
- Ages 6 to 16
- Diagnostic and Statistical Manual of Mental Disorders (DSM IV) diagnosis of autism confirmed by Autism Diagnostic Observation Schedule (ADOS) and/or Autism Diagnostic Interview-Revised (ADI-R)
- No anticipated changes in treatment for the study duration (e.g., diet, nutrients)
- No additional biomedical treatments started 6 weeks prior to enrollment
Key exclusion criteria (7 listed)
- Significant prematurity at birth (less than 32 weeks gestation); or birth weight significantly below normal for gestational age (SGA - small for gestational age)
- mental retardation
- seizure disorder
- auto-immune conditions
- history of head trauma and other neurological or medical conditions
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 |
|---|---|---|
| Umbilical cord mesenchymal stem cells | Biological | Umbilical cord mesenchymal stem cells |
Primary outcome
Number of participants with adverse events
Time frame: 89 weeks
4 secondary outcomes
Number of participants with a change in disability as measured by the Autism Treatment Evaluation Checklist (ATEC)
Time frame: 13 weeks, 25 weeks, 37 weeks, 49 weeks, 89 weeks
Number of participants with a change in disability as measured by the Childhood Autism Rating Scale (CARS)
Time frame: 13 weeks, 25 weeks, 37 weeks, 49 weeks, 89 weeks
Change from baseline macrophage-derived chemokine (MDC)
Time frame: 13 weeks, 25 weeks, 37 weeks, 49 weeks, 89 weeks
Change from baseline thymus and activation-regulated chemokine (TARC)
Time frame: 13 weeks, 25 weeks, 37 weeks, 49 weeks, 89 weeks
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 13 Oct 2017.
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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.
Nelson Novarro, MD principal investigator
Jorge Paz-Rodriguez, MD principal investigator
Translational Biosciences / Stem Cell Institute Panama
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Umbilical cord mesenchymal stem cells in people with Autism, run by Translational Biosciences at 1 site in Panama.
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 15 inclusion and 7 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 13 Oct 2017. 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 Panama. 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 combined 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.
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?
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?
Number of participants with adverse events (time frame: 89 weeks). 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 13 Oct 2017.