Clinical trial NCT02192749: 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
Overall status: COMPLETED
Phase: PHASE1
PHASE2
Conditions: Autism
Keywords: autism
umbilical cord
mesenchymal
stem cells
Lead sponsor: Translational Biosciences
Sponsor class: INDUSTRY
First posted: 2014-07-17
Enrollment: 20
Sex: ALL
Minimum age (years): 6
Maximum age (years): 16
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


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

Primary outcomes:
measure: Number of participants with adverse events
timeFrame: 89 weeks
Secondary outcomes:
measure: Number of participants with a change in disability as measured by the Autism Treatment Evaluation Checklist (ATEC)
timeFrame: 13 weeks, 25 weeks, 37 weeks, 49 weeks, 89 weeks
measure: Number of participants with a change in disability as measured by the Childhood Autism Rating Scale (CARS)
timeFrame: 13 weeks, 25 weeks, 37 weeks, 49 weeks, 89 weeks
measure: Change from baseline macrophage-derived chemokine (MDC)
timeFrame: 13 weeks, 25 weeks, 37 weeks, 49 weeks, 89 weeks
measure: Change from baseline thymus and activation-regulated chemokine (TARC)
timeFrame: 13 weeks, 25 weeks, 37 weeks, 49 weeks, 89 weeks
Other outcomes:
Locations:
city: Panama City
country: Panama
facility: Stem Cell Institute
Study officials:
name: Nelson Novarro, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Translational Biosciences / Stem Cell Institute Panama
name: Jorge Paz-Rodriguez, MD
role: PRINCIPAL_INVESTIGATOR
References: