Allogeneic Umbilical Cord Mesenchymal Stem Cell Therapy for Autism

Phase 1

Completed

• Conditions: Autism
• Interventions: Biological: Umbilical cord mesenchymal stem cells

• Registration Number: NCT02192749
• Lead Sponsor: Translational Biosciences

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2014-07
• Study First Submitted: 2014-07-12
• Study First Submitted QC: 2014-07-15
• Study First Posted: 2014-07-17
• Primary Completion: 2017-08
• Study Completion: 2017-08
• Status Verified: 2017-10
• Last Update Submitted: 2017-10-12
• Last Update Posted: 2017-10-13

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 20

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Umbilical cord mesenchymal stem cells	Umbilical cord mesenchymal stem cells	-

Primary Outcome Measures

Name	Time	Method
Number of participants with adverse events	89 weeks

Secondary Outcome Measures

Name	Time	Method
Change from baseline thymus and activation-regulated chemokine (TARC)	13 weeks, 25 weeks, 37 weeks, 49 weeks, 89 weeks
Number of participants with a change in disability as measured by the Autism Treatment Evaluation Checklist (ATEC)	13 weeks, 25 weeks, 37 weeks, 49 weeks, 89 weeks
Change from baseline macrophage-derived chemokine (MDC)	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)	13 weeks, 25 weeks, 37 weeks, 49 weeks, 89 weeks

Trial Locations

Locations (1)

Stem Cell Institute; 🇵🇦 Panama City, Panama