Allogeneic Umbilical Cord Mesenchymal Stem Cell Therapy for Autism
Primary Purpose
Autism
Status
Completed
Phase
Phase 1
Locations
Panama
Study Type
Interventional
Intervention
Umbilical cord mesenchymal stem cells
Sponsored by
About this trial
This is an interventional treatment trial for Autism focused on measuring autism, umbilical cord, mesenchymal, stem cells
Eligibility Criteria
Inclusion Criteria:
- 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
- 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
Exclusion Criteria:
- 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
Sites / Locations
- Stem Cell Institute
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Umbilical cord mesenchymal stem cells
Arm Description
Outcomes
Primary Outcome Measures
Number of participants with adverse events
Secondary Outcome Measures
Number of participants with a change in disability as measured by the Autism Treatment Evaluation Checklist (ATEC)
Number of participants with a change in disability as measured by the Childhood Autism Rating Scale (CARS)
Change from baseline macrophage-derived chemokine (MDC)
Change from baseline thymus and activation-regulated chemokine (TARC)
Full Information
NCT ID
NCT02192749
First Posted
July 12, 2014
Last Updated
October 12, 2017
Sponsor
Translational Biosciences
1. Study Identification
Unique Protocol Identification Number
NCT02192749
Brief Title
Allogeneic Umbilical Cord Mesenchymal Stem Cell Therapy for Autism
Official Title
Open, Prospective Trial of Treatment of Autism Spectrum Disorders (ASD) Using Intravenous Infusion of Umbilical Cord Tissue Mesenchymal Stem Cells (UC-MSC)
Study Type
Interventional
2. Study Status
Record Verification Date
October 2017
Overall Recruitment Status
Completed
Study Start Date
July 2014 (undefined)
Primary Completion Date
August 2017 (Actual)
Study Completion Date
August 2017 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Translational Biosciences
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
Brief 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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Autism
Keywords
autism, umbilical cord, mesenchymal, stem cells
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
20 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Umbilical cord mesenchymal stem cells
Arm Type
Experimental
Intervention Type
Biological
Intervention Name(s)
Umbilical cord mesenchymal stem cells
Primary Outcome Measure Information:
Title
Number of participants with adverse events
Time Frame
89 weeks
Secondary Outcome Measure Information:
Title
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
Title
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
Title
Change from baseline macrophage-derived chemokine (MDC)
Time Frame
13 weeks, 25 weeks, 37 weeks, 49 weeks, 89 weeks
Title
Change from baseline thymus and activation-regulated chemokine (TARC)
Time Frame
13 weeks, 25 weeks, 37 weeks, 49 weeks, 89 weeks
10. Eligibility
Sex
All
Minimum Age & Unit of Time
6 Years
Maximum Age & Unit of Time
16 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
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
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
Exclusion Criteria:
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Nelson Novarro, MD
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Jorge Paz-Rodriguez, MD
Organizational Affiliation
Translational Biosciences / Stem Cell Institute Panama
Official's Role
Principal Investigator
Facility Information:
Facility Name
Stem Cell Institute
City
Panama City
Country
Panama
12. IPD Sharing Statement
Learn more about this trial
Allogeneic Umbilical Cord Mesenchymal Stem Cell Therapy for Autism
We'll reach out to this number within 24 hrs